• Neurologist and Neuroscientist with Deep Expertise in Orphan and CNS Drug Development Across Disease Areas

    AavantiBio, a gene therapy company focused on transforming the lives of patients with rare genetic diseases, today announced the appointment of Christopher Wright, M.D., Ph.D., as Chief Medical Officer. A neurologist and neuroscientist, Dr. Wright brings to AavantiBio more than 20 years of medical research and drug development experience in specialty and CNS orphan diseases, including cystic fibrosis, dementias, epilepsy, mitochondrial diseases, and sickle cell disease.

    An accomplished scientific and medical leader in both the academic and biopharmaceutical communities, Dr. Wright most recently served as Senior Vice President and Chief…

    • Neurologist and Neuroscientist with Deep Expertise in Orphan and CNS Drug Development Across Disease Areas

    AavantiBio, a gene therapy company focused on transforming the lives of patients with rare genetic diseases, today announced the appointment of Christopher Wright, M.D., Ph.D., as Chief Medical Officer. A neurologist and neuroscientist, Dr. Wright brings to AavantiBio more than 20 years of medical research and drug development experience in specialty and CNS orphan diseases, including cystic fibrosis, dementias, epilepsy, mitochondrial diseases, and sickle cell disease.

    An accomplished scientific and medical leader in both the academic and biopharmaceutical communities, Dr. Wright most recently served as Senior Vice President and Chief Medical Officer of Cyclerion Therapeutics (NASDAQ:CYCN), where he led global development functions across therapeutic areas, including clinical development and operations, regulatory affairs, patient safety, quality, and pharmaceutical development. Previously, he led the global development organization at Ironwood Pharmaceuticals (NASDAQ:IRWD), including responsibility for advancing the late stage and life cycle gastrointestinal and soluble guanylate cyclase stimulator programs. Earlier in his career, Dr. Wright spent seven years in senior medical and clinical roles at Vertex Pharmaceuticals (NASDAQ:VRTX) including Senior Vice President, Medicines Development and Affairs, where he oversaw the development of ORKAMBI® through Phase 3, and the successful development and rapid approval of KALYDECO®, a lifechanging cystic fibrosis therapy, by the FDA, EMA and other regulatory authorities. Dr. Wright also played an important role in the global development and approval of INCIVEK® for hepatitis C.

    "We are thrilled to welcome Chris to AavantiBio as a key member of the senior leadership team who shares our commitment to deep science, innovation, and collaboration," said Bo Cumbo, Chief Executive Officer of AavantiBio. "Chris and I previously worked together at Vertex. I know his extensive background in CNS diseases and specialized experience in orphan drug development, along with his unique perspective as a physician and scientist, will be essential to our mission of bringing transformative therapies to patients with rare genetic diseases."

    "As a neurologist personally committed to improving patient care, I have been deeply impressed with the innovative approach backed by sophisticated science that AavantiBio is employing to develop potentially life-changing treatments for those suffering from serious neurological diseases," said Wright. "I'm excited to partner with this experienced team to advance the development of the Company's lead program in Friedreich's Ataxia and broader pipeline of gene therapies to patients who need them most."

    Dr. Wright earned a A.B. in biochemical sciences from Harvard University, an M.D. and MMSc. from Harvard Medical School and a Ph.D. in neurosciences from Vrije University in the Netherlands. Previously, he was an associate professor of neurology at Harvard Medical School and a graduate of and advisor to the Harvard-MIT Clinical Investigator Training Program. For 20 years, Dr. Wright was a practicing neurologist at Brigham and Women's Hospital in Boston, Mass. He currently serves as a Clinical Advisory Board member at Cyclerion Therapeutics and on the Advisory Board of Clora Inc.

    Earlier this month, AavantiBio announced the appointments of Douglas J. Swirsky as Chief Financial Officer and Treasurer, and Ty Howton as Chief Operating Officer and General Counsel.

    About AavantiBio, Inc.

    AavantiBio is a gene therapy company backed by a premier syndicate of life sciences investors including Perceptive Advisors, Bain Capital Life Sciences, and RA Capital Management, who led the company's recent $107 million Series A financing. Headquartered in Cambridge, Massachusetts, AavantiBio is advancing a diversified gene therapy pipeline in areas of high unmet medical need, including a lead program in Friedreich's Ataxia, a rare inherited genetic disease that causes cardiac and central nervous system dysfunction. The company benefits from strategic partnerships with the University of Florida's renowned Powell Gene Therapy Center and the MDA Care Center at UF Health where AavantiBio's co-founders and renowned gene therapy researchers Barry Byrne, M.D., Ph.D. and Manuela Corti, P.T., Ph.D maintain their research and clinical practices. Learn more at www.aavantibio.com.

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  1. - With this approval approximately 750 people living with cystic fibrosis in Australia will be newly eligible for a CFTR modulator therapy -

    Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today announced that the Australian Therapeutic Goods Administration (TGA) has approved the use of TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) for people with cystic fibrosis (CF) ages 12 years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, the most common CF-causing mutation worldwide. Up to 90% of people living with CF worldwide have at least one F508del mutation.

    "We are delighted the TGA has approved TRIKAFTA for eligible people living with CF in Australia and…

    - With this approval approximately 750 people living with cystic fibrosis in Australia will be newly eligible for a CFTR modulator therapy -

    Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today announced that the Australian Therapeutic Goods Administration (TGA) has approved the use of TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) for people with cystic fibrosis (CF) ages 12 years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, the most common CF-causing mutation worldwide. Up to 90% of people living with CF worldwide have at least one F508del mutation.

    "We are delighted the TGA has approved TRIKAFTA for eligible people living with CF in Australia and will continue working with the Australian government to bring this important medicine to patients as quickly as possible," said Reshma Kewalramani, M.D., Chief Executive Officer and President, Vertex. "It is our goal to develop and provide treatments for all people with CF around the world, and today is another significant milestone on that journey."

    CF affects approximately 3,500 people in Australia. It is caused by a defective and/or missing CFTR protein resulting from mutations in the CFTR gene.

    "Cystic fibrosis is a complex, progressive, devastating disease that causes severe damage to the lungs, digestive system and other organs in the body. It is a condition that significantly affects not only the patient, but also those who care for them, with people living with cystic fibrosis spending multiple hours every day on treatment and requiring daily care from a family member or loved one," said Professor John Wilson AM, Head, Cystic Fibrosis Service, Alfred Health. "The approval of any new treatment option for people living with cystic fibrosis is always welcome news. This new treatment is for patients ages 12 years and older with at least one F508del mutation and means more patients can potentially benefit from a medicine that targets the underlying cause of the disease, for the first time."

    The TGA approval of TRIKAFTA was based on the results of four global Phase 3 studies, which included multiple trial sites and patients from Australia.

    About Cystic Fibrosis

    Cystic Fibrosis (CF) is a rare, life-shortening genetic disease affecting more than 80,000 people globally. CF is a progressive, multi-system disease that affects the lungs, liver, GI tract, sinuses, sweat glands, pancreas and reproductive tract. CF is caused by a defective and/or missing CFTR protein resulting from certain mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF. While there are many different types of CFTR mutations that can cause the disease, the vast majority of all people with CF have at least one F508del mutation. These mutations, which can be determined by a genetic test, or genotyping test, lead to CF by creating non-working and/or too few CFTR proteins at the cell surface. The defective function and/or absence of CFTR protein results in poor flow of salt and water into and out of the cells in a number of organs. In the lungs, this leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage in many patients that eventually leads to death. The median age of death is in the early 30s.

    About TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor)

    In Australia, TRIKAFTA® is indicated for the treatment of cystic fibrosis (CF) in patients ages 12 years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

    The complete product information (PI) can be found on www.tga.gov.au.

    About Vertex

    Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) — a rare, life-threatening genetic disease — and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational small molecule medicines in other serious diseases where it has deep insight into causal human biology, including pain, alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases. In addition, Vertex has a rapidly expanding pipeline of cell and genetic therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.

    Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London, UK. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including 11 consecutive years on Science magazine's Top Employers list and a best place to work for LGBTQ equality by the Human Rights Campaign.

    Special Note Regarding Forward-Looking Statements

    This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, statements made by Reshma Kewalramani, M.D., and Professor John Wilsonin this press release, and statements regarding our expectations for the eligible patient population in Australia, including patients not previously eligible for treatment with a CFTR modulator. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of factors that could cause actual events or results to differ materially from those indicated by such forward-looking statements. Those risks and uncertainties include, among other things, that data from the company's development programs may not support a license extension for TRIKAFTA in Australia, and other risks listed under the heading "Risk Factors" in Vertex's annual report filed with the Securities and Exchange Commission and available through the company's website at www.vrtx.com and on the SEC's website at www.sec.gov. You should not place undue reliance on these statements. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

    (VRTX-GEN)

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  2. Advancing Clinical Development of Next Generation Diabetic Macular Edema (DME) Therapies – with Novel MoAs Beyond anti-VEGF

    Recruiting Phase 2 study (‘KALAHARI') evaluating THR-149, a potent plasma kallikrein inhibitor in DME – On track for Part A data by mid-year

    Positive data from Phase 1 study evaluating THR-687, potential best in class pan-RGD integrin antagonist in DME – On track to start Phase 2 by mid-year

    Highlights
                  

    • Industry-leading pipeline targeting a total potential market opportunity of $12 billion in retinal vascular disorders
       
    • First patients dosed in Phase 2 study (‘KALAHARI') evaluating multiple injections of THR-149, a potent plasma kallikrein inhibitor, for the treatment of DME. Part A data…

    Advancing Clinical Development of Next Generation Diabetic Macular Edema (DME) Therapies – with Novel MoAs Beyond anti-VEGF

    Recruiting Phase 2 study (‘KALAHARI') evaluating THR-149, a potent plasma kallikrein inhibitor in DME – On track for Part A data by mid-year

    Positive data from Phase 1 study evaluating THR-687, potential best in class pan-RGD integrin antagonist in DME – On track to start Phase 2 by mid-year

    Highlights

                  

    • Industry-leading pipeline targeting a total potential market opportunity of $12 billion in retinal vascular disorders

       
    • First patients dosed in Phase 2 study (‘KALAHARI') evaluating multiple injections of THR-149, a potent plasma kallikrein inhibitor, for the treatment of DME. Part A data expected by mid-year

       
    • THR-687, a pan-RGD integrin antagonist, Phase 2 DME study planned to start by mid-year, following positive Phase 1 data released in 2020

       
    • The Company has strengthened the management team with the appointments of Tom Graney, CFA, based in Boston as CFO, Grace Chang M.D., PhD, based in LA as CMO, and Professor Alan Stitt, PhD as CSO.

       
    • At the end of December 2020, Oxurion had cash, cash equivalents & investments of €24.8 million, allowing the Company to execute on its business plans through the end of Q3 2021.  The Company is in advanced discussions with potential investors in order to increase its cash position.

    Leuven, Belgium, Boston, MA, US – March 17, 2021 – 08.00 PM CET Oxurion NV (Euronext Brussels: OXUR), a biopharmaceutical company developing next generation standard-of-care ophthalmic therapies, with an initial focus on diabetic macular edema (DME), today issues its business and financial update for the twelve-month period ending December 31, 2020.

    Oxurion is focused on developing an industry leading DME franchise based on novel therapies. These new drug candidates, which have novel modes of action, largely independent of anti-VEGF pathways, have been designed to improve visual outcomes for all DME patients and to deliver other important clinical benefits.

    DME is a significant and growing global healthcare problem and the major cause of vision loss in persons with diabetes worldwide. Global prevalence of DME is estimated to be 28 million people in 2019.1 The current market value for DME treatments globally is estimated to be approximately $4.5 billion.

    The Company is advancing its pipeline of innovative clinical drug candidates for treating DME. Oxurion's clinical development pipeline consists of two novel product candidates with different and complementary modes of action:   

    • THR-149 is a potential first in class plasma kallikrein inhibitor with the possibility to become the treatment of choice for DME patients who respond sub-optimally to anti-VEGF therapy. 

       
    • THR-687 is a potential best in class small molecule pan-RGD integrin antagonist being developed to treat DME with the possibility to become the standard of care for most DME patients.

    By advancing both of these exciting new drug candidates, with differentiated modes of action, Oxurion expects to bring much needed innovation and improved clinical outcomes to patients with DME.

    Beyond DME, THR-687 also has development possibilities in additional vascular retinal disorders including for wet Age-related Macular Degeneration (wet AMD) and retinal vein occlusion (RVO), thereby potentially allowing the Company to tap into a broader therapeutic market with a current combined estimated annual value of $12 billion.

    Patrik De Haes, M.D., CEO of Oxurion, commented:

    "The last 12 months have seen further important progress towards Oxurion's goal of building the industry leading DME franchise based on bringing much needed innovation to this significant market opportunity. Over this period, we have advanced the clinical development of our lead assets and been successful in making a number of significant senior management appointments.

    In September 2020, we dosed the first patients in the Phase 2 KALAHARI study of our potential first in class plasma kallikrein inhibitor THR-149. These patients with DME have sub-optimally responded to previous anti-VEGF. This two-part study is an important step in bringing THR-149 to patients and is designed to support our goal of positioning THR-149 as the treatment of choice for the large number of DME patients who have a sub-optimal response to anti-VEGF therapy. The KALAHARI study is progressing as planned, despite the widespread COVID-19 pandemic, and we anticipate announcing Part A dose selection data in mid-2021.

    In 2020 we announced positive and highly promising Phase 1 results with THR-687. The data confirmed that this potential best in class pan-RGD integrin antagonist has the profile to become the standard of care for the majority of DME patients by replacing anti-VEGFs as the mainstay of DME therapy. We have carried out additional multiple dose preclinical studies to support the next phase of clinical development and remain on track to start a Phase 2 study in mid 2021. We are very excited by the potential for THR-687 given it has the possibility to be developed for other significant vascular retinal disorders including wet AMD and RVO.

    We have also taken important steps to strengthen our management team. I am pleased to welcome Grace Chang, M.D. as our Chief Medical Officer, Tom Graney, C.F.A. as Chief Financial Officer, and more recently Professor Alan Stitt, Ph.D., as Chief Scientific Officer. We are delighted to be able to attract individuals with such great industry standing to Oxurion as we seek to advance our lead assets through the clinic towards commercialization and in parallel expand our pipeline of differentiated drug candidates. I am confident that we have a team that can deliver results, and with both Tom and Grace being based in the US, we are starting to build the transatlantic organization we need to deliver on our global ambition.

    Oxurion remains focused on its strategy to deliver significant benefits to DME patients and other retinal vascular disorders globally as well as value to our shareholders. These goals are based on successfully advancing THR-149 and THR-687 which together have the potential to provide innovative tailored therapeutic solutions that deliver much improved clinical outcomes to all DME patients, and in the case of THR-687, also the broader market for retinal vascular disorders."

    Diabetes, Diabetic Retinopathy, Diabetic Macular Edema – a global and growing health concern

    Diabetic macular edema (DME) is a result of diabetes caused by fluid accumulation in the macula (central part of the retina), due to leaking blood vessels, leading to swelling of the macular area due to the increased permeability of the vessels resulting in the loss of vision.

    DME is caused by another complication of diabetes, called diabetic retinopathy (DR), in which blood vessels in the eye are damaged, allowing fluid to escape. DR is the presence and characteristic evolution of typical retinal microvascular lesions in an individual with diabetes. DR is a chronic, progressive, sight-threatening, and life-altering disease, and is the leading cause of vision loss in working-age adults (20-65 years). DME can occur at any stage in the development of DR.

    DR and DME are a growing public health concerns due to the rapid growth in the number of people with diabetes globally. More than one in three people living with diabetes will develop some form of DR in their lifetime, and twenty percent of those will have some vision-threatening form of the disease such as DME.

    The current market value for DME treatments has been estimated to be approximately $4.5 billion. Along with the development of diabetes as a global health issue, prevalence numbers of DME are expected to raise for the foreseeable future.  

    Oxurion DME franchise addressing unmet medical need

    The market for DME therapies is currently dominated by anti-VEGFs, which are the standard of care.

    However, anti-VEGFs have been shown to deliver sub-optimal results in a significant portion of the patient population.  Approximately forty percent of DME patients have an unsatisfactory visual response with anti-VEGF therapy, and in many cases anti-VEGFs fail to achieve a clinically meaningful visual improvement. 

    Moreover, despite the significant success of anti-VEGFs, there will always be a need from both physicians and patients for improved therapies, not only to expand treatment capabilities for the forty percent of DME patients who respond sub-optimally to anti-VGEFs, but equally to deliver:

    • Faster onset of action
    • Better therapeutic effect in terms of visual function, best corrected visual acuity (BCVA), and response rate (proportion of patients)
    • Longer duration of response allowing extended treatment intervals
    • Improved convenience of treatment through a simpler dosing regimen

    The above requirements are driving the development of THR-149 and THR-687 to meet specific unmet needs in this market so that these novel compounds could become the new standard of care for patients with DME.

    Oxurion's emerging DME franchise will be based on the successful development of THR-149 and THR-687, two novel therapeutics with different validated modes of action designed for specific complementary target patient groups. 

    Oxurion is confident that with both THR-149 and THR-687 it has the potential to be able to provide new tailored therapeutic solutions that deliver improved clinical outcomes to all DME patients.

    THR-149 – a plasma kallikrein inhibitor for treatment of DME: currently recruiting patients in Phase 2 Part A

    In September 2020, Oxurion announced that the first patient had been treated in the Phase 2 KALAHARI study evaluating THR-149 for treatment of DME.

    THR-149 is a novel plasma kallikrein inhibitor being developed as a potential new standard of care for the forty of DME patients who respond sub-optimally to anti-VEGF therapy.

    THR-149 acts through inhibition of the plasma kallikrein-kinin (PKaI-Kinin) system, a validated target for DME.

    The first part (Part A) of our Phase 2 study will evaluate 3 dose levels of multiple injections of THR-149 in patients with DME to select the optimal dosing regimen based on safety and efficacy. Initial data (from Part A) is expected in mid-2021.

    In Part B of the study, planned to start in H2 2021, the dosing regimen selected in Part A will be compared to the current anti-VEGF standard of care in the form of aflibercept (Eylea) in terms of its ability to improve BCVA. Topline data from Part B is planned to become available in the first half of 2023.

    A positive Phase 1 study with THR-149 showed that it:

    • Is well-tolerated and safe. No dose-limiting toxicities nor drug-related serious adverse events were reported at any of the dosages evaluated in the study.

       
    • Delivered promising efficacy results, particularly improvements in the patient's BCVA. There was a rapid onset of action observed from Day 1, with an increasing average improvement in BCVA of up to 7.5 letters at Day 14.

       
    • Importantly, this activity was maintained with an average improvement in BCVA of 6.5 letters at Day 90 following a single injection of THR-149.

    This novel drug candidate was generated using Bicycle Therapeutics' Bicycles® technology platform. 

    THR-687 - a small molecule pan-RGD integrin antagonist for the treatment of DME:

    Positive Phase 1 Results with THR-687 for the treatment of DME – Phase 2 program planned to start in mid-2021

    Oxurion is developing THR-687, a potential best in class pan-RGD integrin antagonist, to preserve vision in a broad range of patients with DME as first line therapy. Inhibition of integrins is a validated target in DME.

    Topline data in 2020 from the Phase 1 trial showed that THR-687:

    • Is well-tolerated and safe with no dose-limiting toxicities. No serious adverse events were reported at any of the doses evaluated in the study.

       
    • The study also looked at efficacy including changes to the patient's BCVA. Across all doses, a rapid onset of action as measured by mean BCVA change was observed from Day 1 with an increase of 3.1 letters, which further improved to 9.2 letters at Day 30.

       
    • This activity was maintained with a mean BCVA improvement of 8.3 letters at Day 90 following a single injection of THR-687.

       
    • A clear dose response was seen in terms of BCVA with the highest dose of THR-687 delivering a mean BCVA Improvement of 11 letters at Day 14, with a peak improvement of 12.5 letters at Day 90. 

       
    • In addition, a peak mean central subfield thickness (CST) decrease of 106 µm was observed at Day 14 with the highest dose of THR-687.

    Data from this positive Phase 1 study with THR-687 were presented by a leading retina expert at the Bascom Palmer Eye Institute Angiogenesis, Exudation, and Degeneration 2020 Meeting in Miami (US).

    Oxurion is preparing a Phase 2 study with THR-687. The team has carried out additional multiple dose pre-clinical studies and is in the process of completing the IND (Investigational New Drug) submission ahead of the planned start of the Phase 2 study in mid-2021.

    The planned Phase 2 study will be a multiple dose study in two parts, the first Part A to select the optimal dose of THR-687 and the second Part B to compare this selected dose to aflibercept. Dose selection following Part A is anticipated in the first half of 2022 with topline data from Part B planned for the second half of 2023.

    Oxurion preclinical data presentation on THR-687 and dry AMD

    Oxurion presented new preclinical data on THR-687 and dry AMD at the EURETINA 2020 Virtual Meeting in October 2020. The European Society of Retina Specialists (EURETINA) was established over 20 years ago and hosts the leading annual European retinal congress which now attracts over 5,000 global vitreoretinal and macular specialists.

    • The first presentation (title: THR-687, a potent pan-RGD integrin antagonist, holds promise as next-generation therapy for diabetic macular edema) confirmed THR-687 as a promising drug candidate for the treatment of vision-threatening retinal pathologies such as diabetic retinopathy (DR) and DME.

       
    • The second presentation (title: Characterization of the acute rat model of sodium iodate-induced dry age-related macular degeneration) reported data from a new preclinical model for testing and validation of drug candidates for different stages of dry AMD using complementary read-outs.

    Details of the abstracts can be found on the EURETINA 2020 Virtual website: https://www.euretina.org/congress/amsterdam-2020/virtual-2020-freepapers/

    Oxurion Virtual R&D Day

    On October 15, 2020 Oxurion held a virtual R&D day for analysts and investors, highlighting its innovative drug candidates for next generation DME therapy. This included presentations from leading KOLs on THR-149 and THR-687 clinical data and our ongoing and future clinical development strategies for these assets. The presenting KOLs were:

    • For THR-149: Ramin Tadayoni, M.D., Ph.D., Professor of ophthalmology at University of Paris, Head of the Ophthalmology Departments at Lariboisière, St Louis and Rothschild Foundation Hospitals in Paris, France

       
    • For THR-687: Arshad Khanani, M.D., M.A., Managing Partner, Director of Clinical Research, Director of Fellowship at Sierra Eye Associates, and Clinical Associate Professor at the University of Nevada, Reno, US.

    The virtual R&D day was highly successful, attended by approximately 100 participants, mainly analysts and investors.  

    Key Management Appointments

    Tom Graney, CFA, appointed Chief Financial Officer

    In October, Oxurion announced the appointment of Tom Graney, CFA as its Chief Financial Officer (effective October 14, 2020) to succeed Dominique Vanfleteren.

    He is based in Boston, MA, US.

    Tom has over 25 years' experience in senior finance, strategy and operational roles including capital raising, corporate development, and audit. Before joining Oxurion he served as CFO at Generation Bio (NASDAQ:GBIO), a non-viral gene therapy company based in Cambridge, MA, where he led all of the company's financial operations.

    Prior to joining Generation Bio, Tom was Senior Vice President (SVP) and CFO at Vertex Pharmaceuticals (NASDAQ:VRTX), one of the world's most highly valued commercial stage biotech companies, with a multi-billion-dollar turnover. At Vertex Tom was responsible for financial strategy and operations including finance, accounting, and internal audit functions.

    Prior to Vertex, he was the CFO and senior vice president, finance, and corporate strategy at Ironwood Pharmaceuticals (NASDAQ:IRWD), a commercial stage GI-focused biotech company. Before joining Ironwood, Tom spent 20 years with Johnson & Johnson, serving in various roles in the US and abroad, including being Worldwide VP of Finance and CFO of Ethicon, a major medical device company and VP and CFO of Janssen Pharmaceuticals NA, a major pharmaceutical company in North America.

    Grace Chang, M.D., Ph.D. appointed Chief Medical Officer

    In August, Oxurion appointed Grace Chang, M.D., Ph.D. as its Chief Medical Officer (effective August 1, 2020).  She is responsible for leading the Company's clinical programs for both THR-687 and THR-149 as Oxurion looks to build a world-leading DME franchise that could provide much improved therapeutic solutions for all DME patients.

    Dr Chang is a board-certified ophthalmologist and practicing vitreoretinal surgeon with deep expertise in ophthalmic drug research and development.

    Dr Chang is also currently an adjunct Clinical Associate Professor in the Department of Ophthalmology, Vitreoretinal Service at the University of Southern California in Los Angeles.

    Professor Alan Stitt, Ph.D. appointed Chief Scientific Officer

    Effective January 19, 2021, Professor Alan Stitt, Ph.D. was appointed Chief Scientific Officer (CSO) of Oxurion NV. This appointment follows the retirement of the former CSO Jean Feyen, PhD, who has served in this position since joining the Company in 2013. Dr. Feyen will remain available to the Company during a transition period to support Professor Stitt and the rest of the preclinical development team. 

    Professor Stitt is the Chair of Experimental Ophthalmology at Queen's University of Belfast and is internationally known for his research in ophthalmology, particularly in basic science relating to the pathogenesis of retinal diseases, especially diabetic retinopathy, and age-related macular degeneration. He has also been awarded many accolades for his research including a Royal Society Merit Award, election to membership of the Royal Irish Academy (RIA) and Fellowship of the Association for Research in Vision & Ophthalmology (ARVO).

    Beyond his research programs, Alan contributes significantly to the international academic community by serving on advisory boards and grant panels and has a range of editor and editorial board memberships in the ophthalmology are­­na. Going forward, Alan will continue to perform his University duties and affiliations on a part-time basis.

    Other appointments

    Following the tragic passing of Oxurion's Chief Legal Officer and Corporate Secretary/Global Head of Corporate Development, Claude Sander, in December 2019, Oxurion appointed Kathleen Paisley as Chief Legal Officer and Michaël Dillen as Chief Corporate Development Officer and Corporate Secretary.

    Kathleen Paisley is an accomplished lawyer with more than 25 years' experience in major law firms practicing in Brussels, London and The Hague, including as a partner engaged in life sciences at US-based international law firms and Ambos Lawyers in Brussels. She is a US national who is qualified in New York and Washington DC, and earned her JD from the Yale Law School, as well as an MBA in Finance, Bachelor of Sciences and has passed the Certified Public Accountancy exam.

    Michaël Dillen joined the Company from Mithra Pharmaceuticals SA where he was Company Secretary and Vice President of Corporate Development. Prior to Mithra, he was Senior Legal Counsel at Terumo Corporation. His experience includes corporate development, legal, regulatory, and company secretary activities, for pharmaceutical companies as well as at leading law firms. He holds law degrees from the University of Antwerp and Queen Mary University of London, and a business degree from Solvay Brussels School.

    ONCURIOUS - Exciting Progress with Solid Tumor Pipeline Announced

    Oncurious is developing next-generation immuno-oncology drugs targeting a broad spectrum of cancers. Oncurious is a majority owned subsidiary of Oxurion. The remainder of the shares in the company are owned by VIB, a leading life sciences research institute based in Flanders, Belgium.

    Oncurious scientists, in collaboration with world-class immuno-oncology experts in T cell and endothelial cell biology – Prof. Dr. Gabriele Bergers (VIB-KU Leuven), Prof. Dr. Massimiliano Mazzone (VIB-KU Leuven) and Prof. Dr. Jo Van Ginderachter (VIB-VUB), and the drug discovery unit at VIB, are building a pipeline of proprietary investigational I-O therapies with distinct modes of action.

    The team has discovered a potent and diverse panel of leads targeting human CCR8, has reached preclinical proof of concept and is entering the final lead optimization stages nearing preclinical candidate selection. Oncurious is accelerating its efforts towards initiation of preclinical development of the therapeutic antibody program in early 2021.

    Oncurious' CCR8 leads have been generated using an antibody technology platform that has been validated and used for more than a decade to generate high quality binders against G-protein coupled receptors. Molecules discovered using this technology were tested in several preclinical tumor models, and showed that targeting CCR8, depleted Tregs specifically in the tumor microenvironment and resulted in strong anti-tumor responses in monotherapy as well as in combination with anti-PD1. The treatments led to the establishment of immunological memory.

    In addition to the anti-CCR8 program, Oncurious is focusing on two other programs aimed at boosting anti-tumor T cell influx and activity in immune excluded tumors. Exclusion of T cells is an immunosuppressive mechanism commonly used by cancers to evade the immune system and as such is an attractive target for new therapeutic modalities.

    ONCURIOUS – Update TB-403 study in Pediatric Subjects with Medulloblastoma

    Data from the Phase 1, Open-Label, Multicenter, Dose Escalation Study of TB-403 in Pediatric Subjects with Relapsed or Refractory Medulloblastoma are scheduled for presentation at the AACR (American Association for Cancer Research) Annual Meeting on Saturday April 10, 2021.

    Financial Results (unaudited)

    Total revenue amounted to €2.1 million in 2020 compared to €3.9 million in 2019.

    The Company reported a gross profit of €1.5 million in 2020. This compares to a gross profit of €1.7 million in 2019.

    R&D expenses in 2020 were €22.1 million compared to €25.7 million in 2019. R&D expenses were mainly related to preclinical activities as well as clinical activities in THR-687 and THR-149. The 2020 figure included a milestone payment of €2.0 million related to the development of THR-149. Government grants and income from recharge of costs are deducted from the research and development expenses.

    Selling and marketing expenses were €3.3 million in 2020. This compares to €7.0 million in 2019.

    General and administrative expenses decreased from €6.3 million in 2019 to €5.5 million in 2020.

    In 2020, Oxurion made a loss for the year of €28.6 million, compared to a loss for the year in 2019 of €52.1 million resulting in negative diluted earnings per share of €0.75 euro in 2020 versus €1.36 euro in 2019.

    The Oxurion cash position (including investments) at the end of 2020 amounted to €24.8 million. This compares to €52.9 million (including investments) at the end of 2019.

    Oxurion believes that its cash position will allow it to execute on its business plans through the end of Q3 2021 given the cost reductions it has made.  The Company is therefore in advanced discussions with potential investors and other sources of capital to secure the necessary funding to deliver on its further plans, as well as continuing to manage its cash position carefully.

    The financial results included in this press release remain unaudited. Audited financial results and the Annual Report 2020 for the period ending December 31, 2020, will be published by April 5, 2021, on the Company's website.

    END

    For further information please contact:

    Oxurion NV

    Wouter Piepers,

    Global Head of Investor Relations

    & Corporate Communications

    Tel: +32 16 75 13 10 / +32 478 33 56 32





     
    EU

    Citigate Dewe Rogerson

    David Dible/ Sylvie Berrebi/Frazer Hall

    Tel: +44 20 7638 9571





     



    US

    Westwicke, an ICR Company

    Christopher Brinzey

    Tel: +1 617 835 9304

    About Oxurion

    Oxurion (Euronext Brussels: OXUR) is a biopharmaceutical company developing next generation standard of care ophthalmic therapies, which are designed to better preserve vision in patients. Our initial focus is in diabetic macular edema (DME), the leading cause of vision loss in diabetic patients worldwide.

    Oxurion is aiming to build the leading global franchise in the treatment of DME, based on the successful development of its two novel therapeutics:

    • THR-149, a plasma kallikrein inhibitor being developed as a potential new standard of care for DME patients who respond sub-optimally to anti-VEGF therapy. THR-149 has shown positive topline Phase 1 results for the treatment of DME. The Company is currently conducting a Phase 2 clinical trial evaluating multiple injections of THR-149 in DME patients who previously responded sub-optimally to anti-VEGF therapy. THR-149 was developed in conjunction with Bicycle Therapeutics PLC (NASDAQ:BCYC).

       
    • THR-687 is a pan-RGD integrin inhibitor, that is initially being developed as a potential new standard of care for the majority of first line DME patients. Positive topline results in a Phase 1 clinical study assessing THR-687 as a treatment for DME were announced in January 2020. THR-687 is expected to enter a Phase 2 clinical trial by mid-2021 after receiving regulatory approval. THR-687 is an optimized compound derived from a broader library of integrin inhibitors in-licensed from Galapagos NV ((Euronext &, NASDAQ:GLPG).

    Oxurion is headquartered in Leuven, Belgium, and is listed on the Euronext Brussels exchange under the symbol OXUR. More information is available at www.oxurion.com.

    Important information about forward-looking statements

    Certain statements in this press release may be considered "forward-looking". Such forward-looking statements are based on current expectations, and, accordingly, entail and are influenced by various risks and uncertainties. The Company therefore cannot provide any assurance that such forward-looking statements will materialize and does not assume an obligation to update or revise any forward-looking statement, whether as a result of new information, future events, or any other reason. Additional information concerning risks and uncertainties affecting the business and other factors that could cause actual results to differ materially from any forward-looking statement is contained in the Company's Annual Report. This press release does not constitute an offer or invitation for the sale or purchase of securities or assets of Oxurion in any jurisdiction.  No securities of Oxurion may be offered or sold within the United States without registration under the U.S. Securities Act of 1933, as amended, or in compliance with an exemption therefrom, and in accordance with any applicable U.S. state securities laws.



     

    Unaudited Consolidated statement of profit and loss

    In '000 euro (for the year ended 31 December) 2020 2019
         
    Income 2,078 3,946
    Sales 2,000 3,820
    Income from royalties 78 126
    Cost of sales -550 -2,259
    Gross profit 1,528 1,687
    Research and development expenses -22,053 -25,709
    General and administrative expenses -5,489 -6,324
    Selling expenses -3,252 -6,955
    Other operating income 777 2,022
    Other operating expense -6 -4
    Impairment losses -125 -16,891
    Operating result -28,620 -52,174
    Finance income 468 495
    Finance expense -408 -407
    Result before income tax -28,560 -52,086
    Taxes 0 -17
    Result of the year -28,560 -52,103
         
    Attributable to:    
    Equity holders of the company -28,012 -51,827
    Non-controlling interest -548 -276
         
    Result per share    
    Basic earnings / loss (-) per share (euro) -0.75 -1.36
    Diluted earnings / loss (-) per share (euro) -0.75 -1.36



    In '000 euro (as at 31 December) 2020 2019
    Result of the year -28,560 -52,103
    Exchange differences on translation of foreign operations and remeasurement DBP -424 -342
    Other comprehensive income, net of income tax -424 -342
    Other comprehensive income that will not be reclassified to profit or loss -424 -342
    Total comprehensive loss (-) / income for the year -28,984 -52,445
    Attributable to:    
    Equity holders of the company -28,436 -52,169
    Non-controlling interest -548 -276



     

    Unaudited Consolidated statement of financial position

    In '000 euro (as at 31 December) 2020 2019
         
    ASSETS    
    Property, plant and equipment 230 340
    Right-of-use assets 1,069 2,212
    Intangible assets 2,127 1,982
    Other non-current assets 96 96
    Non-current tax credit 3,708 3,385
    Non-current assets 7,230 8,015
    Inventories 85 20
    Trade and other receivables 1,451 3,592
    Current tax receivables 719 467
    Investments 288 10,444
    Cash and cash equivalents 24,511 42,492
    Current assets 27,054 57,015
    Total assets 34,284 65,030
         
    EQUITY AND LIABILITIES    
    Share capital 44,913 100,644
    Share premium 0 0
    Cumulative translation differences -1,039 -615
    Other reserves -6,133 -12,122
    Retained earnings -12,561 -34,747
    Equity attributable to equity holders of the company 25,180 53,160
    Non-controlling interest -132 146
    Total equity 25,048 53,306
    Lease liabilities 447 1,335
    Employee benefit liabilities 1,096 801
    Non-current liabilities 1,543 2,136
    Trade payables 4,377 4,725
    Lease liabilities 649 898
    Other short-term liabilities 2,667 3,965
    Current liabilities 7,693 9,588
    Total equity and liabilities 34,284 65,030



     

    Unaudited Consolidated statement of cash flows

    In '000 euro (for the year ended 31 December) 2020 2019
         
    Cash flows from operating activities    
    Loss for the period -28,560 -52,103
    Finance expense 408 407
    Finance income -468 -495
    Depreciation of property, plant and equipment 194 330
    Amortization and impairment of intangible assets 125 18,468
    Amortization of right-of-use assets 916 864
    Gain on sale of property, plant and equipment -7 0
    Equity settled share-based payment transactions 458 440
    Decrease in trade and other receivables including tax receivables and inventories 1,501 1,082
    Increase / Decrease (-) in short-term liabilities -1,646 -432
    Net cash flows generated / used (-) in operating activities -27,079 -31,439
         
    Cash flows from investing activities    
    Disposal of property, plant and equipment (following a sale) 35 77
    Decrease / Increase (-) in investments 10,154 10,033
    Interest received and similar income -6 4
    Purchase of property, plant and equipment -119 -133
    Purchase / divestment (-) of other non-current assets 0 31
    Net cash flows generated / used (-) in investing activities 10,064 10,012
         
    Cash flows from financing activities    
    Principal paid on lease liabilities -903 -843
    Interest paid on lease liabilities -16 -24
    Paid interests -12 -10
    Net cash flows used (-) / generated in financing activities -931 -877
         
    Net change in cash and cash equivalents -17,946 -22,304
    Net cash and cash equivalents at the beginning of the period 42,492 64,652
    Effect of exchange rate fluctuations -35 144
    Net cash and cash equivalents at the end of the period 24,511 42,492



     

    Unaudited Consolidated statement of changes in equity

      Share capital Share premium Cumulative translation differences and revaluation reserve Other reserves Retained earnings Attributable to equity holders of the company Non-controlling interest Total
    Balance as at 1 January 2019 137,564 13 -273 -12,563 -19,853 104,888 422 105,310
    Result of the year 2019 0 0 0 0 -51,827 -51,827 -276 -52,103
    Change to foreign currency translation difference and revaluation reserve 0 0 -342 0 0 -342 0 -342
    Net change in fair value of investments 0 0 0 1 0 1 0 1
    Capital decrease -36,920 -13 0 0 36,933 0 0 0
    Share-based payment transactions 0 0 0 440 0 440 0 440
    Balance as at 31 December 2019 100,644 0 -615 -12,122 -34,747 53,160 146 53,306
                     
    Balance as at 1 January 2020 100,644 0 -615 -12,122 -34,747 53,160 146 53,306
    Result of the year 2020 0 0 0 0 -28,012 -28,012 -548 -28,560
    Change to foreign currency translation difference and revaluation reserve 0 0 -424 0 0 -424 0 -424
    Net change in fair value of investments 0 0 0 -2 0 -2 0 -2
    Issue of ordinary shares 0 0 0 0 0 0 270 270
    Capital decrease -55,731 0 0 5,533 50,198 0 0 0
    Share-based payment transactions 0 0 0 458 0 458 0 458
    Balance as at 31 December 2020 44,913 0 -1,039 -6,133 -12,561 25,180 -132 25,048










     

    1 Estimated and based on International Diabetes Federation. IDF Diabetes Atlas, 9th edn. Brussels, Belgium: International Diabetes Federation, 2019. http://www.diabetesatlas.org; Yau JW et al. Diabetes Care 2012;35(3):556-564; Thomas RL et al. Diabetes Res Clin Pract 2019;157:107840





     

     

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  3. Industry Veteran Brings Over 20 Years of Commercial, Operational, and Legal Experience

    AavantiBio, a gene therapy company focused on transforming the lives of patients with rare genetic diseases, today announced the appointment of Ty Howton as Chief Operating Officer and General Counsel. A veteran life sciences executive, Mr. Howton brings to AavantiBio more than two decades of strategic, operational, and legal experience in the biopharmaceutical industry.

    In his most recent position, Mr. Howton served as Executive Vice President, General Counsel and Corporate Secretary of Sarepta Therapeutics (NASDAQ:SRPT), where he led Company's global legal and compliance functions and oversaw quality assurance and information technology. Previously, he…

    Industry Veteran Brings Over 20 Years of Commercial, Operational, and Legal Experience

    AavantiBio, a gene therapy company focused on transforming the lives of patients with rare genetic diseases, today announced the appointment of Ty Howton as Chief Operating Officer and General Counsel. A veteran life sciences executive, Mr. Howton brings to AavantiBio more than two decades of strategic, operational, and legal experience in the biopharmaceutical industry.

    In his most recent position, Mr. Howton served as Executive Vice President, General Counsel and Corporate Secretary of Sarepta Therapeutics (NASDAQ:SRPT), where he led Company's global legal and compliance functions and oversaw quality assurance and information technology. Previously, he served as Senior Vice President, Chief Legal Officer, and as a member of the executive team at Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX), where he had general management responsibilities and oversaw all aspects of the legal and compliance departments globally, including risk management. Prior to joining Vertex, Mr. Howton worked at Genentech, Inc., where he served in a variety of legal roles before becoming the company's chief healthcare compliance officer. Earlier in his career, he was a member of the Sidley Austin LLP corporate healthcare practice where he advised on corporate transactions involving life science companies and provided regulatory counsel.

    "Ty brings extensive industry leadership experience along with a diverse professional skill set, and we are ecstatic to welcome him at this exciting time in our growth journey," said Bo Cumbo, Chief Executive Officer of AavantiBio. "As a key member of the leadership team, he will be integrally involved in determining and executing on our corporate strategy as we begin to scale our commercial and manufacturing operations to bring a competitive pipeline of gene therapies to patients who need them most."

    Mr. Howton's appointment comes on the heels of Douglas J. Swirsky joining the Company as Chief Financial Officer and Treasurer.

    "Having spent the majority of my career at biotech companies with missions focused on patient care and innovation, I'm thrilled to be joining a company like AavantiBio that is taking such a unique approach to transforming the lives of patients with rare genetic diseases," said Howton. "I look forward to working closely with Bo and the team to advance the Company's strong pipeline of programs towards the clinic so we can help these patients with significant unmet needs."

    Mr. Howton currently serves as a member of the Board of Directors of Make-A-Wish® Massachusetts and Rhode Island. He holds a BA from Yale University and a JD from Northwestern University School of Law.

    About AavantiBio, Inc.

    AavantiBio is a gene therapy company backed by a premier syndicate of life sciences investors including Perceptive Advisors, Bain Capital Life Sciences, and RA Capital Management, who led the company's recent $107 million Series A financing. Headquartered in Cambridge, Massachusetts, AavantiBio is advancing a diversified gene therapy pipeline in areas of high unmet medical need, including a lead program in Friedreich's Ataxia (FA), a rare inherited genetic disease that causes cardiac and central nervous system dysfunction. The company benefits from strategic partnerships with the University of Florida's renowned Powell Gene Therapy Center and the MDA Care Center at UF Health where AavantiBio's co-founders and renowned gene therapy researchers Barry Byrne, M.D., Ph.D. and Manuela Corti, P.T., Ph.D maintain their research and clinical practices. Learn more at www.aavantibio.com.

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  4. VX-880 is the first investigational stem cell-derived therapy utilizing fully differentiated, insulin-producing pancreatic islet cells for the treatment of type 1 diabetes –

    VX-880 is the first and only pancreatic islet replacement therapy known to receive Fast Track Designation –

    Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for VX-880 and that the company has initiated a clinical trial for VX-880 in patients who have type 1 diabetes (T1D) with severe hypoglycemia and impaired hypoglycemic awareness.

    "This program has its roots in the groundbreaking work that began in Dr. Doug Melton's lab, progressed at Semma Therapeutics, and has…

    VX-880 is the first investigational stem cell-derived therapy utilizing fully differentiated, insulin-producing pancreatic islet cells for the treatment of type 1 diabetes –

    VX-880 is the first and only pancreatic islet replacement therapy known to receive Fast Track Designation –

    Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for VX-880 and that the company has initiated a clinical trial for VX-880 in patients who have type 1 diabetes (T1D) with severe hypoglycemia and impaired hypoglycemic awareness.

    "This program has its roots in the groundbreaking work that began in Dr. Doug Melton's lab, progressed at Semma Therapeutics, and has been accelerated and brought to the clinic by the team at Vertex," said Bastiano Sanna, Ph.D., Executive Vice President and Chief of Cell and Genetic Therapies at Vertex. "Ours is the only approach that produces fully differentiated and fully functional insulin-secreting pancreatic islets. We are very pleased to have received FDA's Fast Track Designation, which facilitates the development and expedites the review of drugs that treat serious conditions and fill an unmet medical need. We continue to work with urgency to bring this innovative therapy to patients."

    "It's a remarkable time for T1D research efforts worldwide, as this investigational treatment enters the clinic," said Camillo Ricordi, M.D., Professor of Surgery, Director of the Diabetes Research Institute (DRI) and the Cell Transplant Center at the University of Miami Miller School of Medicine, and Steering Committee Chair for the VX-880 clinical trial. "The field's experience with the limited cadaveric islet transplants available, where some patients have experienced prolonged insulin independence for years, provides important proof-of-concept for the potential of cell therapy to be transformative for patients living with T1D."

    The first clinical trial sites at the University of Miami Health System, the University of Pennsylvania and Massachusetts General Hospital are open for enrollment, and additional sites will be activated this year. To learn more visit clinicaltrials.gov.

    About VX-880

    VX-880, formerly known as STx-02, is an investigational allogeneic human stem cell-derived islet cell therapy that is being evaluated for patients who have T1D with impaired hypoglycemic awareness and severe hypoglycemia. VX-880 has the potential to restore the body's ability to regulate glucose levels by restoring pancreatic islet cell function, including insulin production.

    The VX-880 clinical trial will involve an infusion of fully differentiated, functional islet cells, as well as the chronic administration of concomitant immunosuppressive therapy, to protect the islet cells from immune rejection.

    About the Phase 1/2 Clinical Trial

    The clinical trial is a Phase 1/2, single-arm, open-label study in patients who have T1D with impaired hypoglycemic awareness and severe hypoglycemia. This will be a sequential, multi-part clinical trial to evaluate the safety and efficacy of different doses of VX-880. Approximately 17 patients will be enrolled in the clinical trial.

    About Type 1 Diabetes

    T1D results from the autoimmune destruction of insulin-producing islet cells in the pancreas, leading to loss of insulin production and impairment of blood glucose control. The absence of insulin leads to abnormalities in how the body processes nutrients, leading to high blood glucose levels. High blood glucose can lead to diabetic ketoacidosis and over time, to complications such as kidney disease/failure, eye disease (including vision loss), heart disease, stroke, nerve damage and even death.

    Due to the limitations and complexities of insulin delivery systems, it can be difficult to achieve and maintain balance in glucose control in patients with T1D. Hypoglycemia often results because of the difficulty in balancing the different factors that impact glucose levels, including insulin, diet and exercise. Hypoglycemia remains a critical limiting factor in glycemic management, and severe hypoglycemia can cause loss of consciousness, coma, seizures, injury, and can be fatal. Over time, patients with T1D can develop impaired awareness of hypoglycemia, meaning they are no longer able to perceive the early signs of a hypoglycemic event, which can be dangerous and result in life threatening events.

    There are currently limited treatment options beyond insulin for the management of T1D.

    About Vertex

    Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) — a rare, life-threatening genetic disease — and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational small molecule medicines in other serious diseases where it has deep insight into causal human biology, including pain, alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases. In addition, Vertex has a rapidly expanding pipeline of cell and genetic therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.

    Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including 11 consecutive years on Science magazine's Top Employers list and a best place to work for LGBTQ equality by the Human Rights Campaign. For company updates and to learn more about Vertex's history of innovation, visit www.vrtx.com or follow us on Facebook, Twitter, LinkedIn, YouTube and Instagram.

    Special Note Regarding Forward-Looking Statements

    This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, statements made by Dr. Bastiano Sanna and Dr. Camillo Ricordi in this press release, statements regarding the development, plans and expectations for our T1D pipeline program, including our Phase 1/2 clinical trial in people with T1D, statements regarding patient enrollment, dosing and activation of new sites, statements regarding potential clinical trial results and anticipated benefits of VX-880, and our plans to provide further updates on our T1D pipeline program. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that the FDA may not approve our IND, that data from a limited number of patients may not be indicative of final clinical trial results, that data from the company's development programs may not support registration or further development due to safety, efficacy or other reasons, that the COVID-19 pandemic may impact the status or progress of our clinical trial of VX-880, and other risks listed under the heading "Risk Factors" in Vertex's most recent annual report filed with the Securities and Exchange Commission at www.sec.gov and available through the company's website at www.vrtx.com. You should not place undue reliance on these statements. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

    (VRTX-GEN)

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  5. Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today announced that management will present at the Cowen Health Care Conference on Tuesday, March 2, 2021 at 9:50 a.m. ET.

    The audio portion of management's remarks will be available live through Vertex's website, www.vrtx.com in the "Investors" section under the "News and Events" page. A replay of the conference webcast will be archived on the company's website.

    About Vertex

    Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) — a rare, life-threatening genetic disease — and has several ongoing…

    Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today announced that management will present at the Cowen Health Care Conference on Tuesday, March 2, 2021 at 9:50 a.m. ET.

    The audio portion of management's remarks will be available live through Vertex's website, www.vrtx.com in the "Investors" section under the "News and Events" page. A replay of the conference webcast will be archived on the company's website.

    About Vertex

    Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) — a rare, life-threatening genetic disease — and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational small molecule medicines in other serious diseases where it has deep insight into causal human biology, including pain, alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases. In addition, Vertex has a rapidly expanding pipeline of genetic and cell therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.

    Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including 11 consecutive years on Science magazine's Top Employers list and a best place to work for LGBTQ equality by the Human Rights Campaign. For company updates and to learn more about Vertex's history of innovation, visit www.vrtx.com or follow us on Facebook, Twitter, LinkedIn, YouTube and Instagram.

    (VRTX-WEB)

    View Full Article Hide Full Article
  6. WARREN, N.J., Feb. 09, 2021 (GLOBE NEWSWIRE) -- Aquestive Therapeutics, Inc. (NASDAQ:AQST), a pharmaceutical company focused on developing and commercializing differentiated products that address patients' unmet needs and solve therapeutic problems, today announced the appointment of Julie Krop, M.D., Chief Medical Officer of Freeline Therapeutics (NASDAQ:FRLN), and Marco Taglietti, M.D., Director, President and Chief Executive Officer of SCYNEXIS (NASDAQ:SCYX), to the Board of Directors of the Company effective February 10, 2021. Aquestive also announced the resignation of Douglas K. Bratton from the Board of Directors after more than 17 years of service. Aquestive's Board of Directors will now be comprised of eight Directors, seven of whom…

    WARREN, N.J., Feb. 09, 2021 (GLOBE NEWSWIRE) -- Aquestive Therapeutics, Inc. (NASDAQ:AQST), a pharmaceutical company focused on developing and commercializing differentiated products that address patients' unmet needs and solve therapeutic problems, today announced the appointment of Julie Krop, M.D., Chief Medical Officer of Freeline Therapeutics (NASDAQ:FRLN), and Marco Taglietti, M.D., Director, President and Chief Executive Officer of SCYNEXIS (NASDAQ:SCYX), to the Board of Directors of the Company effective February 10, 2021. Aquestive also announced the resignation of Douglas K. Bratton from the Board of Directors after more than 17 years of service. Aquestive's Board of Directors will now be comprised of eight Directors, seven of whom are independent. Dr. Krop will serve as a member of the Board's Nominating and Corporate Governance Committee and Dr. Taglietti will serve as a member of the Board's Audit Committee.

    "We are delighted to welcome Julie and Marco as new independent directors to the Aquestive Board of Directors," said Santo Costa, Aquestive's Chairman of the Board. "Their significant expertise in the pharmaceutical and biotechnology industry and impressive backgrounds complement our Board of Directors' skills and experiences. We are confident they will provide valuable perspectives as we continue to execute our strategy and enhance value for the Company's shareholders."

    Dr. Krop stated, "I am thrilled to join the Board of Directors of Aquestive and look forward to working with the Board and the leadership team to advance the Company's mission of providing novel alternatives to invasively administered standard of care therapies. I am particularly excited about helping management guide Aquestive's proprietary orally administered epinephrine through clinical development. This novel formulation of epinephrine has the potential to eliminate the burden of intramuscular or subcutaneous injections for patients at risk of anaphylaxis due to severe allergies."

    Dr. Taglietti commented, "Aquestive is at an important stage of its evolution and I am delighted to join the Board of Directors during this exciting time. I look forward to contributing to Aquestive's continued growth and success as the Company advances its Libervant™ (diazepam) Buccal Film application through approval and executes on its innovative development activities with the potential to change patients' lives."

    "Julie and Marco join Aquestive at an exciting time as we continue to focus on developing and bringing to market valuable products in the CNS and allergy spaces. Julie and Marco's extensive collective experience in leading clinical development, regulatory strategies and commercialization of key value assets is a great addition and complement to the skills already present on our board," remarked Keith Kendall, Director, President and Chief Executive Officer of Aquestive. "We've experienced significant growth and strengthened our capabilities as a commercial pharmaceutical company under Doug's leadership as the original Chairman of the Board and then director. I would like to thank him for his contributions and expert guidance that have well positioned Aquestive for continued momentum and success."

    "On behalf of the Board of Directors and the Company, I would like to thank Doug for his more than 17 years of service to our Board, including as former Chairman, in guiding the Company from its start-up phase to a public commercial pharmaceutical company and leader in the film-based therapeutics industry," said Mr. Costa.

    "It has been a great privilege to serve as a director and former Chairman of Aquestive's Board of Directors, the members of which I hold in very high regard. I'd like to thank Keith, his team, and the Board for many exciting and satisfying years, and look forward to watching them accomplish even more great things for Aquestive in the months and years to come," stated Mr. Bratton.

    About Douglas K. Bratton

    Mr. Bratton has served as a member of the Company's Board of Directors since January 2004 and was the Chairman of the Board from January 2004 until August 2018. Mr. Bratton is the Founder, President and Chief Investment Officer of Crestline Investors, an institutional alternative investment management firm. He has been an investment professional specializing in alternative asset strategies since 1983 and has managed assets on behalf of the Bass family of Fort Worth, Texas since 1988.

    About Julie Krop, M.D.

    Dr. Krop is a seasoned biotech executive with more than two decades of experience successfully designing and executing clinical development programs from early stage development all the way through FDA approval. She has held senior leadership roles across clinical development, regulatory affairs, clinical operations, pharmacovigilance, medical affairs and program management during her career in the pharmaceutical and biotechnology industry. She currently serves as the Chief Medical Officer of Freeline Therapeutics (NASDAQ:FRLN). Prior to assuming her position with Freeline Therapeutics, Dr. Krop was the Chief Medical Officer and Executive Vice President, Development, at AMAG Pharmaceuticals (NASDAQ:AMAG) from 2015 to 2020. From 2012 to 2015, Dr. Krop served as the Vice President, Clinical Development for Vertex Pharmaceuticals (NASDAQ:VRTX). In addition, Dr. Krop was Vice President, Clinical Development and Regulatory Affairs for Stryker Biotech (NYSE:SYK) from 2006 to 2012. Dr. Krop received a B.A. from Brown University and her M.D. from Brown University School of Medicine. She completed her fellowship in the Department of Endocrinology at the Johns Hopkins University School of Medicine where she was also a Robert Wood Johnson Foundation Clinical Scholar.

    About Marco Taglietti, M.D.

    Dr. Taglietti has more than three decades of experience in the pharmaceutical and biotechnology industry. He currently serves as a Director and President and Chief Executive Officer of SCYNEXIS Inc. (NASDAQ:SCYX). Prior to joining SCYNEXIS, Dr. Taglietti held various executive positions with Forest Laboratories (now AbbVie (NYSE: ABBV)) from 2007 until 2014, including President, Forest Research Institute, Chief Medical Officer and Executive Corporate Vice President, Research & Development. Dr. Taglietti was also the Senior Vice President, Head of Global Research and Development for Stiefel Laboratories, Inc. (now a GlaxoSmithKline company) from 2004 until 2007 and served in a number of executive positions from 1992 to 2004 with Schering-Plough Research Institute, including Vice President, Clinical Research Anti-Infectives, CNS, Dermatology and Endocrinology. From 1987 until 1992, Dr. Taglietti served in a number of executive positions with Marion Merrell Dow Research Institute, including as the European Product Team Leader – Anti-Infectives. Dr. Taglietti previously served on the boards of directors of Delcath (NASDAQ:DCTH) from 2014 to 2020 and NephroGenex (NASDAQ:NRX) from 2014 to 2017. Dr. Taglietti also served as a director of Stiefel International, Ltd., a private company, from 2004 to 2007 and a director of TransCelerate BioPharma, a non-profit pharma coalition dedicated to streamlining and accelerating the research and development of innovative new therapies, from 2013 to 2014. Since 2011, Dr. Taglietti has served on the board of directors of BioNJ, a life sciences trade association in New Jersey. In addition, Dr. Taglietti served on the board of directors of HINJ, Health Institute of New Jersey, a trade association for the leading research-based biopharmaceutical and medical technology companies in New Jersey, from 2011 to 2014, and is currently on the boards of directors of Orchestra of St. Luke, a New York City based orchestra, and American Foundation for Suicide Prevention, the largest non-profit organization dedicated to saving lives and bringing hope to those affected by suicide. Dr. Taglietti received his Degree in Medicine from the University of Pavia, Italy.

    About Aquestive Therapeutics

    Aquestive Therapeutics is a pharmaceutical company that applies innovative technology to solve therapeutic problems and improve medicines for patients. The Company has commercialized one internally-developed proprietary product to date, Sympazan® (clobazam) oral film, has a commercial proprietary product pipeline focused on the treatment of diseases of the central nervous system, or CNS, and other unmet needs, and is developing orally administered complex molecules to provide alternatives to invasively administered standard of care therapies. The Company also collaborates with other pharmaceutical companies to bring new molecules to market using proprietary, best-in-class technologies, like PharmFilm®, and has proven capabilities for drug development and commercialization.

    Forward-Looking Statements

    Certain statements in this press release are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as "believe," "anticipate," "plan," "expect," "estimate," "intend," "may," "will," or the negative of those terms, and similar expressions, are intended to identify forward-looking statements. These forward-looking statements include, but are not limited to, statements regarding the advancement of Libervant and other product candidates through the regulatory and development pipeline; and business strategies, market opportunities, and other statements that are not historical facts. These forward-looking statements are subject to the uncertain impact of the COVID-19 global pandemic on our business including with respect to our clinical trials including site initiation, patient enrollment and timing and adequacy of clinical trials; on regulatory submissions and regulatory reviews and approvals of our product candidates; pharmaceutical ingredient and other raw materials supply chain, manufacture, and distribution; sale of and demand for our products; our liquidity and availability of capital resources; customer demand for our products and services; customers' ability to pay for goods and services; and ongoing availability of an appropriate labor force and skilled professionals. Given these uncertainties, the Company is unable to provide assurance that operations can be maintained as planned prior to the COVID-19 pandemic.

    These forward-looking statements are based on our current expectations and beliefs and are subject to a number of risks and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. Such risks and uncertainties include, but are not limited to, risks associated with the Company's development work, including any delays or changes to the timing, cost and success of our product development activities and clinical trials and plans for AQST-108 and our other drug candidates; risk of delays in FDA approval of our drug candidate Libervant and AQST-108 and our other drug candidates or failure to receive approval; ability to address the concerns identified in the FDA's Complete Response Letter dated September 25, 2020 regarding the New Drug Application for Libervant; risk of our ability to demonstrate to the FDA "clinical superiority" within the meaning of the FDA regulations of Libervant relative to FDA-approved diazepam rectal gel and nasal spray products including by establishing a major contribution to patient care within the meaning of FDA regulations relative to the approved products as well as risks related to other potential pathways or positions which are or may in the future be advanced to the FDA to overcome the seven year orphan drug exclusivity granted by the FDA for the approved nasal spray product of a competitor in the U.S. and there can be no assurance that we will be successful; risk that a competitor obtains FDA orphan drug exclusivity for a product with the same active moiety as any of our other drug products for which we are seeking FDA approval and that such earlier approved competitor orphan drug blocks such other product candidates in the U.S. for seven years for the same indication; risk inherent in commercializing a new product (including technology risks, financial risks, market risks and implementation risks and regulatory limitations); risks and uncertainties concerning the royalty and other revenue stream of the KYNMOBI™ monetization transaction, achievement of royalty targets worldwide or in any jurisdiction and certain other commercial targets required for contingent payments under the monetization transaction, and of sufficiency of net proceeds of the monetization transaction after satisfaction of and compliance with 12.5% Senior Notes obligations, as applicable, and for funding the Company's operations; risk of development of our sales and marketing capabilities; risk of legal costs associated with and the outcome of our patent litigation challenging third party at risk generic sale of our proprietary products; risk of sufficient capital and cash resources, including access to available debt and equity financing and revenues from operations, to satisfy all of our short-term and longer term cash requirements and other cash needs, at the times and in the amounts needed; risk of failure to satisfy all financial and other debt covenants and of any default; our and our competitors' orphan drug approval and resulting drug exclusivity for our products or products of our competitors; short-term and long-term liquidity and cash requirements, cash funding and cash burn; risk related to government claims against Indivior for which we license, manufacture and sell Suboxone® and which accounts for the substantial part of our current operating revenues; risk associated with Indivior's cessation of production of its authorized generic buprenorphine naloxone film product, including the impact from loss of orders for the authorized generic product and risk of eroding market share for Suboxone and risk of sunsetting product; risks related to the outsourcing of certain marketing and other operational and staff functions to third parties; risk of the rate and degree of market acceptance of our product and product candidates; the success of any competing products, including generics; risk of the size and growth of our product markets; risks of compliance with all FDA and other governmental and customer requirements for our manufacturing facilities; risks associated with intellectual property rights and infringement claims relating to the Company's products; risk of unexpected patent developments; the impact of existing and future legislation and regulatory provisions on product exclusivity; legislation or regulatory actions affecting pharmaceutical product pricing, reimbursement or access; claims and risks that may arise regarding the safety or efficacy of the Company's products and product candidates; risk of loss of significant customers; risks related to legal proceedings, including patent infringement, investigative and antitrust litigation matters; changes in government laws and regulations; risk of product recalls and withdrawals; uncertainties related to general economic, political, business, industry, regulatory and market conditions and other unusual items; and other uncertainties affecting the Company described in the "Risk Factors" section and in other sections included in our Annual Report on Form 10 K, in our Quarterly Reports on Form 10-Q, and in our Current Reports on Form 8-K filed with the Securities Exchange Commission (SEC). Given those uncertainties, you should not place undue reliance on these forward-looking statements, which speak only as of the date made. All subsequent forward-looking statements attributable to us or any person acting on our behalf are expressly qualified in their entirety by this cautionary statement. The Company assumes no obligation to update forward-looking statements or outlook or guidance after the date of this press release whether as a result of new information, future events or otherwise, except as may be required by applicable law.

    PharmFilm®, Sympazan® and the Aquestive logo are registered trademarks of Aquestive Therapeutics, Inc. All other registered trademarks referenced herein are the property of their respective owners.

    Investor inquiries:

    Westwicke, an ICR Company

    Stephanie Carrington



    646-277-1282

     



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  7. -Full-year 2020 GAAP product revenues of $6.20 billion-

    -Full-year 2020 non-GAAP product revenues of $6.20 billion, a 55% increase compared to full-year 2019-

    -Company provides full-year 2021 product revenue guidance of $6.7 to $6.9 billion-

    Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today reported consolidated financial results for the full year and fourth quarter ended December 31, 2020 and provided full-year 2021 financial guidance.

    "Our achievements in 2020 were marked by a significant increase in the number of people treated with the triple combination in the U.S. and the EU. It was also a year marked by meaningful pipeline advancement. We now have clinical programs in seven disease areas, spanning multiple modalities including…

    -Full-year 2020 GAAP product revenues of $6.20 billion-

    -Full-year 2020 non-GAAP product revenues of $6.20 billion, a 55% increase compared to full-year 2019-

    -Company provides full-year 2021 product revenue guidance of $6.7 to $6.9 billion-

    Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today reported consolidated financial results for the full year and fourth quarter ended December 31, 2020 and provided full-year 2021 financial guidance.

    "Our achievements in 2020 were marked by a significant increase in the number of people treated with the triple combination in the U.S. and the EU. It was also a year marked by meaningful pipeline advancement. We now have clinical programs in seven disease areas, spanning multiple modalities including small molecules for alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases, gene editing for sickle cell disease and beta thalassemia, and cell therapy for type 1 diabetes," said Reshma Kewalramani, M.D., Chief Executive Officer and President of Vertex. "As we enter 2021, we look forward to treating more CF patients and reaching important R&D milestones in multiple additional diseases which will fuel our growth this year and for many years to come."

    Fourth-Quarter and Full-Year 2020 Financial Highlights

     

    Three Months Ended December 31,

     

    %

     

    Twelve Months Ended December 31,

     

    %

     

    2020

     

    2019

     

    Change

     

    2020

     

    2019

     

    Change

     

    (in millions, except per share amounts)

    GAAP Product revenues, net

    $

    1,627

     

     

    $

    1,413

     

     

    15

    %

     

    $

    6,203

     

     

    $

    4,161

     

     

    49

    %

    Non-GAAP Product revenues, net (1)

    $

    1,627

     

     

    $

    1,257

     

     

    29

    %

     

    $

    6,203

     

     

    $

    4,005

     

     

    55

    %

     

     

     

     

     

     

     

     

     

     

     

     

     

     

     

     

    GAAP Operating income

    $

    746

     

     

    $

    551

     

     

    35

    %

     

    $

    2,856

     

     

    $

    1,198

     

     

    139

    %

    Non-GAAP Operating income

    $

    887

     

     

    $

    593

     

     

    50

    %

     

    $

    3,491

     

     

    $

    1,786

     

     

    95

    %

     

     

     

     

     

     

     

     

     

     

     

     

     

     

     

     

    GAAP Net income

    $

    604

     

     

    $

    583

     

     

    4

    %

     

    $

    2,712

     

     

    $

    1,177

     

     

    130

    %

    Non-GAAP Net income

    $

    661

     

     

    $

    444

     

     

    49

    %

     

    $

    2,719

     

     

    $

    1,389

     

     

    96

    %

     

     

     

     

     

     

     

     

     

     

     

     

     

     

     

     

    GAAP Net income per share - diluted

    $

    2.30

     

     

    $

    2.23

     

     

    3

    %

     

    $

    10.29

     

     

    $

    4.51

     

     

    128

    %

    Non-GAAP Net income per share - diluted

    $

    2.51

     

     

    $

    1.70

     

     

    48

    %

     

    $

    10.32

     

     

    $

    5.33

     

     

    94

    %

    Full-Year 2020 Results

     

    Twelve Months Ended December 31,

     

    2020

     

    2019

     

    (in millions)

    GAAP Product revenues, net

    $

    6,203

     

     

    $

    4,161

     

     

     

     

     

     

     

    Non-GAAP Product revenues, net (1)

    $

    6,203

     

     

    $

    4,005

     

    TRIKAFTA/KAFTRIO

    $

    3,864

     

     

    $

    420

     

    SYMDEKO/SYMKEVI

    $

    629

     

     

    $

    1,418

     

    ORKAMBI

    $

    908

     

     

    $

    1,176

     

    KALYDECO

    $

    803

     

     

    $

    991

     

    GAAP and Non-GAAP product revenues increased 49% and 55%, respectively, compared to 2019, primarily driven by the uptake of TRIKAFTA in the U.S., KAFTRIO in Europe and our other medicines outside the U.S. following the completion of several significant reimbursement agreements. Net product revenues in 2020 were $4.8 billion in the U.S. and $1.4 billion outside the U.S.

    GAAP and Non-GAAP net income increased compared to 2019, largely driven by strong growth in product revenues.

    Cash, cash equivalents and marketable securities as of December 31, 2020 were $6.7 billion, an increase of approximately $2.9 billion compared to $3.8 billion as of December 31, 2019 driven by strong revenue and profitability.

    Full-Year 2020 Expenses

     

    Twelve Months Ended December 31,

     

    2020

     

    2019

     

    (in millions)

    Combined GAAP R&D and SG&A expenses

    $

    2,600

     

     

    $

    2,413

     

    Combined Non-GAAP R&D and SG&A expenses

    $

    1,981

     

     

    $

    1,694

     

     

     

     

     

     

     

    GAAP R&D expense

    $

    1,830

     

     

    $

    1,755

     

    Non-GAAP R&D expense

    $

    1,372

     

     

    $

    1,171

     

     

     

     

     

     

     

    GAAP SG&A expense

    $

    770

     

     

    $

    658

     

    Non-GAAP SG&A expense

    $

    609

     

     

    $

    523

     

     

     

     

     

     

     

    GAAP income taxes

    $

    405

     

     

    $

    218

     

    Non-GAAP income taxes

    $

    721

     

     

    $

    397

     

     

     

     

     

     

     

    GAAP effective tax rate

     

    13

    %

     

     

    16

    %

    Non-GAAP effective tax rate (2)

     

    21

    %

     

     

    22

    %

    Combined GAAP and Non-GAAP R&D and SG&A expenses increased compared to 2019, primarily due to the incremental investment to support the global use of Vertex's medicines and the expansion of Vertex's pipeline in CF and other disease areas.

    GAAP and Non-GAAP income taxes increased compared to 2019 primarily due to Vertex's increased operating income. Refer to the "Supplemental Income Tax Information" section for discussion of the cash versus non-cash components of Vertex's provision for income taxes.

    Fourth-Quarter 2020 Results

     

    Three Months Ended December 31,

     

    2020

     

    2019

     

    (in millions)

    GAAP Product revenues, net

    $

    1,627

     

     

    $

    1,413

     

     

     

     

     

     

     

    Non-GAAP Product revenues, net (1)

    $

    1,627

     

     

    $

    1,257

     

    TRIKAFTA/KAFTRIO

    $

    1,091

     

     

    $

    420

     

    SYMDEKO/SYMKEVI

    $

    128

     

     

    $

    332

     

    ORKAMBI

    $

    215

     

     

    $

    270

     

    KALYDECO

    $

    193

     

     

    $

    236

     

    GAAP and Non-GAAP product revenues increased 15% and 29%, respectively, compared to the fourth quarter of 2019, primarily driven by the uptake of TRIKAFTA in the U.S., KAFTRIO in Europe and our other medicines outside the U.S. following the completion of several significant reimbursement agreements.

    GAAP and non-GAAP net income increased compared to the fourth quarter of 2019, largely driven by strong growth in total product revenues.

    Fourth-Quarter 2020 Expenses

     

    Three Months Ended December 31,

     

    2020

     

    2019

     

    (in millions)

    Combined GAAP R&D and SG&A expenses

    $

    678

     

     

    $

    675

     

    Combined Non-GAAP R&D and SG&A expenses

    $

    539

     

     

    $

    496

     

     

     

     

     

     

     

    GAAP R&D expense

    $

    467

     

     

    $

    480

     

    Non-GAAP R&D expense

    $

    364

     

     

    $

    337

     

     

     

     

     

     

     

    GAAP SG&A expense

    $

    212

     

     

    $

    195

     

    Non-GAAP SG&A expense

    $

    175

     

     

    $

    159

     

     

     

     

     

     

     

    GAAP income taxes

    $

    284

     

     

    $

    94

     

    Non-GAAP income taxes

    $

    198

     

     

    $

    145

     

     

     

     

     

     

     

    GAAP effective tax rate

     

    32

    %

     

     

    14

    %

    Non-GAAP effective tax rate (2)

     

    23

    %

     

     

    25

    %

     

     

     

     

     

     

    Combined GAAP R&D and SG&A expenses were similar to the fourth quarter of 2019.

    Combined Non-GAAP R&D and SG&A expenses increased compared to the fourth quarter of 2019, primarily due to the incremental investment to support the global use of Vertex's medicines and the expansion of Vertex's pipeline in CF and other disease areas.

    GAAP and Non-GAAP income taxes increased compared to the fourth quarter of 2019 primarily due to Vertex's increased operating income. Refer to the "Supplemental Income Tax Information" section for discussion of the cash versus non-cash components of Vertex's provision for income taxes.

    Full-Year 2021 Financial Guidance

    Vertex today provided its full-year 2021 financial guidance. Product revenue guidance is primarily based on:

    • The continued strong performance of TRIKAFTA in the U.S. and KAFTRIO in certain European countries
    • The launch of medicines in the U.S. for rare mutations following approval in December 2020 and the approval of TRIKAFTA for children with CF ages 6-11 in the U.S. expected mid-year
    • Countries where patients currently have access or reimbursement

    Vertex's guidance is summarized below:

     

     

    FY 2021

     

     

     

    Product revenues

     

    $6.7 to 6.9 billion

     

     

     

    Combined GAAP R&D and SG&A expenses (3)

     

    $2.9 to 3.05 billion

    Combined Non-GAAP R&D and SG&A expenses (3)

     

    $2.25 to 2.3 billion

    Non-GAAP effective tax rate

     

    21% to 22%

    Key Business Highlights

    Cystic Fibrosis (CF) R&D pipeline

    Vertex expects to increase the number of CF patients eligible to take our medicines and thereby continue to grow our CF business. Important progress has been made in increasing the number of eligible patients and expanding approval and access for our medicines to additional geographies and age groups.

    TRIKAFTA/KAFTRIO (elexacaftor, tezacaftor and ivacaftor)

    • The U.S. Food and Drug Administration (FDA) expanded the eligibility for TRIKAFTA to include people with CF ages 12 and older with certain mutations that are responsive to TRIKAFTA based on in vitro data. SYMDEKO and KALYDECO also received approvals to include additional responsive mutations in people with CF ages 6 and older and ages 4 months and older, respectively.
    • Swissmedic, the Swiss Agency for Therapeutic Products, granted marketing authorization and a reimbursement agreement was reached for TRIKAFTA in Switzerland in people with CF ages 12 and older with two F508del mutations or one F508del mutation and one minimal function mutation.
    • The U.S. FDA accepted a supplemental New Drug Application (sNDA) for TRIKAFTA for the treatment of children with CF ages 6 to 11 who have at least one F508del mutation or have certain mutations that are responsive to TRIKAFTA based on in vitro data. The FDA granted Priority Review of the sNDA and assigned a Prescription Drug User Fee Act (PDUFA) target action date of June 8, 2021.
    • Health Canada accepted a New Drug Submission for Priority Review for TRIKAFTA for the treatment of people with CF ages 12 years and older.

    SYMDEKO/SYMKEVI (tezacaftor and ivacaftor)

    • The European Commission (EC) granted approval of the label extension for SYMKEVI to include people with CF ages 6 years and older with two copies of the F508del mutation or one copy of the F508del mutation and certain residual function mutations.

    KALYDECO (ivacaftor)

    • The EC granted approval of the label extension for KALYDECO to include the treatment of infants with CF ages 4 months and older who have R117H or certain gating mutations.

    R&D pipeline outside of CF

    Vertex continues to progress a broad pipeline of potentially transformative small molecule, cell and genetic therapies aimed at serious diseases. Recent and anticipated progress for key pipeline programs is noted below:

    Beta Thalassemia and Sickle Cell Disease

    • Vertex and its partner CRISPR Therapeutics are evaluating the use of an ex vivo CRISPR gene-edited therapy for the treatment of transfusion-dependent beta thalassemia (TDT) and sickle cell disease (SCD). This approach aims to edit a person's hematopoietic stem cells to produce fetal hemoglobin in red blood cells, which has the potential to reduce or eliminate symptoms associated with disease.
    • Enrollment and dosing are ongoing in the clinical studies for CTX001. More than 20 patients have been dosed with CTX001 across both studies to date. Completion of enrollment in both studies is expected in 2021.

    Alpha-1 Antitrypsin (AAT) Deficiency

    • Vertex is evaluating multiple compounds with the potential to correct the misfolding of Z-AAT protein in the liver, in order to increase the levels of functional AAT in the blood. Misfolded Z-AAT protein is the root cause of AAT deficiency.
    • Enrollment is ongoing in a Phase 2 proof-of-concept study for the Z-AAT corrector, VX-864. Data from this study is expected in the first half of 2021.

    APOL1-mediated Kidney Diseases

    • Vertex is evaluating the potential for inhibitors of APOL1 function to reduce proteinuria in people with serious kidney diseases, including focal segmental glomerulosclerosis (FSGS).
    • Enrollment is ongoing in a Phase 2 proof-of-concept study designed to evaluate the reduction in proteinuria in people with APOL1-mediated FSGS after treatment with VX-147. Data from this study is expected in 2021.

    Type 1 Diabetes (T1D)

    • Vertex is developing a cell therapy designed to replace insulin-producing islet cells in people with T1D. Two opportunities exist for the transplant of these functional islets into patients: 1) transplantation of islet cells alone, using immunosuppression to protect the implanted cells and 2) implantation of the islet cells inside a novel immunoprotective device.
    • The U.S. FDA cleared the Investigational New Drug Application (IND) for VX-880, the islet cells alone program. Vertex expects to initiate a Phase 1/2 clinical trial in the first half of 2021.

    Investments in External Innovation

    • Skyhawk Therapeutics and Vertex established a strategic collaboration to discover and develop novel small molecules that modulate RNA splicing for the treatment of serious diseases.

    Non-GAAP Financial Measures

    In this press release, Vertex's financial results and financial guidance are provided in accordance with accounting principles generally accepted in the United States (GAAP) and using certain non-GAAP financial measures. In particular, non-GAAP financial results and guidance exclude from Vertex's pre-tax income (i) stock-based compensation expense, (ii) an adjustment to product revenues and related cost of sales to reflect the conclusion of the early access program for ORKAMBI in France in the fourth quarter of 2019, (iii) revenues and expenses related to collaboration agreements, (iv) gains or losses related to the fair value of the company's strategic investments, (v) increases or decreases in the fair value of contingent consideration, (vi) acquisition-related costs and (vii) other adjustments. The company's non-GAAP financial results also exclude from its provision for income taxes the estimated tax impact related to its non-GAAP adjustments to pre-tax income described above and certain discrete items. These results should not be viewed as a substitute for the company's GAAP results and are provided as a complement to results provided in accordance with GAAP. Management believes these non-GAAP financial measures help indicate underlying trends in the company's business, are important in comparing current results with prior period results and provide additional information regarding the company's financial position that the company believes is helpful to an understanding of its ongoing business. Management also uses these non-GAAP financial measures to establish budgets and operational goals that are communicated internally and externally, to manage the company's business and to evaluate its performance. The company adjusts, where appropriate, for both revenues and expenses in order to reflect the company's operations. The company's calculation of non-GAAP financial measures likely differs from the calculations used by other companies. A reconciliation of the GAAP financial results to non-GAAP financial results is included in the attached financial information.

    The company provides guidance regarding combined R&D and SG&A expenses and effective tax rate on a non-GAAP basis. The guidance regarding combined GAAP R&D and SG&A expenses does not include estimates associated with any potential future business development activities. The company does not provide a GAAP effective tax rate because it is unable to forecast with reasonable certainty the impact of excess tax benefits related to stock-based compensation and the possibility of certain discrete items, which could be material.

     

    Vertex Pharmaceuticals Incorporated

    Fourth-Quarter Results

    Consolidated Statements of Operations

    (in thousands, except per share amounts)

    (unaudited)

     

     

    Three Months Ended December 31,

     

    Twelve Months Ended December 31,

     

    2020

     

    2019

     

    2020

     

    2019

    Revenues:

     

     

     

     

     

     

     

    Product revenues, net

    $

    1,626,920

     

     

    $

    1,413,265

     

     

    $

    6,202,783

     

     

    $

    4,160,726

     

    Collaboration and royalty revenues

    900

     

     

     

     

    2,900

     

     

    2,095

     

    Total revenues

    1,627,820

     

     

    1,413,265

     

     

    6,205,683

     

     

    4,162,821

     

    Costs and expenses:

     

     

     

     

     

     

     

    Cost of sales

    203,101

     

     

    185,012

     

     

    736,300

     

     

    547,758

     

    Research and development expenses

    466,584

     

     

    480,011

     

     

    1,829,537

     

     

    1,754,540

     

    Sales, general and administrative expenses

    211,843

     

     

    195,277

     

     

    770,456

     

     

    658,498

     

    Change in fair value of contingent consideration

    500

     

     

    1,500

     

     

    13,100

     

     

    4,459

     

    Total costs and expenses

    882,028

     

     

    861,800

     

     

    3,349,393

     

     

    2,965,255

     

    Income from operations

    745,792

     

     

    551,465

     

     

    2,856,290

     

     

    1,197,566

     

    Interest income

    2,320

    12,359

     

     

    22,239

     

     

    63,678

     

    Interest expense

    (16,288

    )

     

    (14,249

    )

     

    (58,151

    )

     

    (58,502

    )

    Other income, net (4)

    156,799

     

     

    127,375

     

     

    296,420

     

     

    192,177

     

    Income before provision for income taxes

    888,623

     

     

    676,950

     

     

    3,116,798

     

     

    1,394,919

     

    Provision for income taxes

    284,433

     

    93,716

     

     

    405,151

     

     

    218,109

     

    Net income

    $

    604,190

     

     

    $

    583,234

     

     

    $

    2,711,647

     

     

    $

    1,176,810

     

     

     

     

     

     

     

     

     

    Net income per common share:

     

     

     

     

     

     

     

    Basic

    $

    2.32

     

     

    $

    2.26

     

     

    $

    10.44

     

     

    $

    4.58

     

    Diluted

    $

    2.30

     

     

    $

    2.23

     

     

    $

    10.29

     

     

    $

    4.51

     

    Shares used in per share calculations:

     

     

     

     

     

     

     

    Basic

    260,038

     

     

    258,003

     

     

    259,841

     

     

    256,728

     

    Diluted

    263,106

     

     

    262,108

     

     

    263,396

     

     

    260,673

     

     

    Reconciliation of GAAP to Non-GAAP Net Income

    Fourth-Quarter Results

    (in thousands, except per share amounts)

    (unaudited)

     

     

    Three Months Ended December 31,

     

    Twelve Months Ended December 31,

     

    2020

     

    2019

     

    2020

     

    2019

    GAAP net income

    $

    604,190

     

     

    $

    583,234

     

     

    $

    2,711,647

     

     

    $

    1,176,810

     

    Stock-based compensation expense

    97,027

     

     

    91,591

     

     

    429,461

     

     

    360,489

     

    ORKAMBI Adjustment (1)

     

     

    (140,854

    )

     

     

     

    (140,854

    )

    Increase in fair value of strategic investments (4)

    (171,071

    )

     

    (128,734

    )

     

    (311,937

    )

     

    (197,596

    )

    Increase in fair value of contingent consideration (5)

    500

     

     

    1,500

     

     

    13,100

     

     

    4,459

     

    Collaborative revenues and expenses (6)

    40,400

     

     

    56,057

     

     

    181,700

     

     

    318,343

     

    Acquisition-related costs (7)

    2,820

     

     

    33,181

     

     

    10,682

     

     

    45,871

     

    Total non-GAAP adjustments to pre-tax income

    (30,324

    )

     

    (87,259

    )

     

    323,006

     

     

    390,712

     

    Tax adjustments (2)

    86,728

     

     

    (51,627

    )

     

    (315,455

    )

     

    (178,578

    )

    Non-GAAP net income

    $

    660,594

     

     

    $

    444,348

     

     

    $

    2,719,198

     

     

    $

    1,388,944

     

     

     

     

     

     

     

     

     

    Net income per diluted common share:

     

     

     

     

     

     

     

    GAAP

    $

    2.30

     

     

    $

    2.23

     

     

    $

    10.29

     

     

    $

    4.51

     

    Non-GAAP

    $

    2.51

     

     

    $

    1.70

     

     

    $

    10.32

     

     

    $

    5.33

     

    Shares used in diluted per share calculations:

     

     

     

     

     

     

     

    GAAP and Non-GAAP

    263,106

     

     

    262,108

     

     

    263,396

     

     

    260,673

     

     

    Reconciliation of GAAP to Non-GAAP Revenues and Expenses

    Fourth-Quarter Results

    (in thousands)

    (unaudited)

     

     

    Three Months Ended December 31,

     

    Twelve Months Ended December 31,

     

    2020

     

    2019

     

    2020

     

    2019

    GAAP total revenues

    $

    1,627,820

     

     

    $

    1,413,265

     

     

    $

    6,205,683

     

     

    $

    4,162,821

     

    ORKAMBI Adjustment (1)

     

     

    (155,773

    )

     

     

     

    (155,773

    )

    Collaborative revenues

    (900

    )

     

     

     

    (2,900

    )

     

    (158

    )

    Non-GAAP total revenues

    $

    1,626,920

     

     

    $

    1,257,492

     

     

    $

    6,202,783

     

     

    $

    4,006,890

     

     

     

     

     

     

     

     

     

     

    Three Months Ended December 31,

     

    Twelve Months Ended December 31,

     

    2020

     

    2019

     

    2020

     

    2019

    GAAP cost of sales

    $

    203,101

     

     

    $

    185,012

     

     

    $

    736,300

     

     

    $

    547,758

     

    ORKAMBI Adjustment (1)

     

     

    (14,919

    )

     

     

     

    (14,919

    )

    Stock-based compensation expense

    (1,581

    )

     

    (1,397

    )

     

    (5,579

    )

     

    (5,575

    )

    Non-GAAP cost of sales

    $

    201,520

     

     

    $

    168,696

     

     

    $

    730,721

     

     

    $

    527,264

     

     

     

     

     

     

     

     

     

    GAAP research and development expenses

    $

    466,584

     

     

    $

    480,011

     

     

    $

    1,829,537

     

     

    $

    1,754,540

     

    Stock-based compensation expense

    (58,958

    )

     

    (56,707

    )

     

    (262,690

    )

     

    (224,558

    )

    Collaborative expenses (6)

    (41,300

    )

     

    (56,057

    )

     

    (184,600

    )

     

    (318,501

    )

    Acquisition-related costs (7)

    (2,820

    )

     

    (30,461

    )

     

    (10,229

    )

     

    (40,583

    )

    Non-GAAP research and development expenses

    $

    363,506

     

     

    $

    336,786

     

     

    $

    1,372,018

     

     

    $

    1,170,898

     

     

     

     

     

     

     

     

     

    GAAP sales, general and administrative expenses

    $

    211,843

     

     

    $

    195,277

     

     

    $

    770,456

     

     

    $

    658,498

     

    Stock-based compensation expense

    (36,488

    )

     

    (33,487

    )

     

    (161,192

    )

     

    (130,356

    )

    Acquisition-related costs (7)

     

     

    (2,720

    )

     

    (453

    )

     

    (5,288

    )

    Non-GAAP sales, general and administrative expenses

    $

    175,355

     

     

    $

    159,070

     

     

    $

    608,811

     

     

    $

    522,854

     

     

     

     

     

     

     

     

     

    Combined non-GAAP R&D and SG&A expenses

    $

    538,861

     

     

    $

    495,856

     

     

    $

    1,980,829

     

     

    $

    1,693,752

     

     

     

     

     

     

     

     

     

     

    Three Months Ended December 31,

     

    Twelve Months Ended December 31,

     

    2020

     

    2019

     

    2020

     

    2019

    GAAP other income, net

    $

    156,799

     

     

    $

    127,375

     

     

    $

    296,420

     

     

    $

    192,177

     

    Increase in fair value of strategic investments (4)

    (171,071

    )

     

    (128,734

    )

     

    (311,937

    )

     

    (197,596

    )

    Non-GAAP other expense, net

    $

    (14,272

    )

     

    $

    (1,359

    )

     

    $

    (15,517

    )

     

    $

    (5,419

    )

     

     

     

     

     

     

     

     

    GAAP provision for income taxes

    $

    284,433

     

     

    $

    93,716

     

     

    $

    405,151

     

     

    $

    218,109

     

    Tax adjustments (2)

    (86,728

    )

     

    51,627

     

     

    315,455

     

     

    178,578

     

    Non-GAAP provision for income taxes (8)

    $

    197,705

     

     

    $

    145,343

     

     

    $

    720,606

     

     

    $

    396,687

     

     

    Condensed Consolidated Balance Sheets

    (in thousands)

    (unaudited)

     

     

    December 31, 2020

     

    December 31, 2019

    Assets

     

     

     

    Cash, cash equivalents and marketable securities

    $

    6,658,897

     

     

    $

    3,808,294

     

    Accounts receivable, net

    885,352

     

     

    633,518

     

    Inventories

    280,777

     

     

    167,502

     

    Property and equipment, net

    958,534

     

     

    745,080

     

    Goodwill and intangible assets

    1,402,158

     

     

    1,402,158

     

    Deferred tax assets

    882,779

     

     

    1,190,815

     

    Other assets

    683,311

     

     

    371,098

     

    Total assets

    $

    11,751,808

     

     

    $

    8,318,465

     

     

     

     

     

    Liabilities and Shareholders' Equity

     

     

     

    Accounts payable and accrued expenses

    $

    1,560,110

     

     

    $

    1,204,522

     

    Finance lease liabilities

    581,476

     

     

    577,371

     

    Contingent consideration

    189,600

     

     

    176,500

     

    Other liabilities

    733,807

     

     

    274,828

     

    Shareholders' equity

    8,686,815

     

     

    6,085,244

     

    Total liabilities and shareholders' equity

    $

    11,751,808

     

     

    $

    8,318,465

     

     

     

     

     

    Common shares outstanding

    259,890

     

     

    258,993

     

     

    Supplemental Income Tax Information

    (in thousands, except percentages)

    (unaudited)

     

     

    Three Months Ended December 31,

     

    Twelve Months Ended December 31,

     

    2020

     

    2019

     

    2020

     

    2019

    Components of provision for income taxes related to:

     

     

     

     

     

     

     

     

     

     

     

     

    Cash paid or accrued for income taxes

    $

    122,975

     

     

    $

    9,185

     

     

    $

    168,322

     

     

    $

    32,138

     

    Benefits from income taxes due to discrete tax items (2)

    10,034

     

     

     

     

    (287,565

    )

     

     

    Provision for income taxes offset by net operating losses and credits (8)

    151,424

     

     

    84,531

     

     

    524,394

     

     

    185,971

     

    GAAP provision for income taxes (8)

    $

    284,433

     

     

    $

    93,716

     

     

    $

    405,151

     

     

    $

    218,109

     

     

     

     

     

     

     

     

     

    Cash paid or accrued for income taxes

    $

    122,975

     

     

    $

    9,185

     

     

    $

    168,322

     

     

    $

    32,138

     

    Adjustments to pre-tax income

    (76,694

    )

     

    51,627

     

     

    27,890

     

     

    178,578

     

    Provision for income taxes offset by net operating losses and credits (8)

    151,424

     

     

    84,531

     

     

    524,394

     

     

    185,971

     

    Non-GAAP provision for income taxes (8)

    $

    197,705

     

     

    $

    145,343

     

     

    $

    720,606

     

     

    $

    396,687

     

     

     

     

     

     

     

     

     

     

     

     

     

     

     

     

     

    Effective tax rate reconciliation:

     

     

     

     

     

     

     

    GAAP effective tax rate

    32

    %

     

    14

    %

     

    13

    %

     

    16

    %

    Impact of GAAP to Non-GAAP adjustments

    (9

    )%

     

    11

    %

     

    8

    %

     

    6

    %

    Non-GAAP effective tax rate

    23

    %

     

    25

    %

     

    21

    %

     

    22

    %

    Notes and Explanations

    1: "ORKAMBI adjustment" in the company's Reconciliation of GAAP to Non-GAAP Net Income in the three and twelve months ended December 31, 2019 included an adjustment to net product revenues and cost of sales related to the conclusion of the early access program for ORKAMBI in France in the fourth quarter of 2019. The company had previously recognized a portion of net product revenues related to ORKAMBI distributed through the early access program in France. As a result, the company recognized an adjustment to increase net product revenues by $155.8 million and cost of sales by $14.9 million, which related to prior period shipments of ORKAMBI distributed through the early access program in France. The company excluded the adjustment to net product revenues and cost of sales from its Non-GAAP measures for the three and twelve months ended December 31, 2019.

    2: In the three and twelve months ended December 31, 2020 and 2019, "Tax adjustments" primarily related to the estimated income taxes related to non-GAAP adjustments to pre-tax income including (i) stock-based compensation (including an adjustment for excess tax benefits related to stock-based compensation), (ii) increases or decreases in the fair value of the company's strategic investments and (iii) collaborative payments. In the twelve months ended December 31, 2020, "Tax adjustments" also included non-recurring discrete benefits to the company's provision for income taxes, such as the transfer of intellectual property rights to the company's U.K. entity, of approximately $287.6 million that the company excluded from its Non-GAAP measures.

    3: The difference between the company's full-year 2021 combined GAAP R&D and SG&A expenses and combined non-GAAP R&D and SG&A expenses guidance relates primarily to $430 million to $500 million of stock-based compensation expense and $200 million to $250 million of R&D expenses related to existing collaboration agreements. The guidance regarding combined GAAP R&D and SG&A expenses does not include estimates associated with any potential future business development activities.

    4: "Other income, net" includes gains related to changes in the fair value of the company's strategic investments and from sales of certain investments.

    5: During the three and twelve months ended December 31, 2020 and 2019, the increase in the fair value of contingent consideration relates to potential payments to Exonics Therapeutics' former equity holders.

    6: "Collaborative revenues and expenses" in the three and twelve months ended December 31, 2020 and 2019 primarily related to collaborative upfront and milestone payments.

    7: "Acquisition-related costs" in the three and twelve months ended December 31, 2020 and 2019 related to costs associated with the company's acquisitions of Semma Therapeutics and Exonics.

    8: The company records a provision for income taxes on its pre-tax income using an effective tax rate approximating statutory rates. Since the company released its valuation allowance on the majority of its net operating losses and other deferred tax assets as of December 31, 2018, its tax provision has included a significant non-cash charge due to the company's ability to offset its pre-tax income against previously benefited net operating losses and credits. As of December 31, 2019, the company had federal net operating losses and credits that were available to offset future pre-tax income. The company utilized substantially all of its remaining federal net operating losses in 2020. As a result, a larger portion of the company's tax provision will represent a cash expense in future periods, subject to continued utilization of certain tax credits.

    Note: Amounts may not foot due to rounding.

    About Vertex

    Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) — a rare, life-threatening genetic disease — and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational small molecule medicines in other serious diseases where it has deep insight into causal human biology, including pain, alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases. In addition, Vertex has a rapidly expanding pipeline of genetic and cell therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.

    Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including 11 consecutive years on Science magazine's Top Employers list and a best place to work for LGBTQ equality by the Human Rights Campaign. For company updates and to learn more about Vertex's history of innovation, visit www.vrtx.com or follow us on Facebook, Twitter, LinkedIn, YouTube and Instagram.

    Special Note Regarding Forward-Looking Statements

    This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, Dr. Kewalramani's statements in this press release, the information provided regarding future financial performance and operations, the section captioned "Full-Year 2021 Financial Guidance" and statements regarding (i) anticipated regulatory filings and data submissions, (ii) anticipated future label expansions, (iii) the expectations, development plans and anticipated timelines for the company's medicines, drug candidates and pipeline programs, including collaborations with third parties, (iv) expectations for the collaborations with CRISPR, including expectations regarding completion of enrollment, (v) expectations for uptake of and expanded access to the company's medicines, including additional reimbursement agreements, and (vi) anticipated investment in internal and external innovation. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that the company's expectations regarding its 2021 product revenues, expenses and effective tax rates may be incorrect (including because one or more of the company's assumptions underlying its expectations may not be realized), that COVID-19 may have different or more significant impacts on the company's business or operations than the company currently expects, data from preclinical testing or early clinical trials, especially if based on a limited number of patients, may not be indicative of final results, that the FDA may not approve VX-880 on a timely basis, or at all, that data from the company's development programs may not support registration or further development of its potential medicines in a timely manner, or at all, due to safety, efficacy or other reasons, and other risks listed under the heading "Risk Factors" in Vertex's annual report and subsequent quarterly reports filed with the Securities and Exchange Commission and available through the company's website at www.vrtx.com and on the SEC's website at www.sec.gov. You should not place undue reliance on these statements. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

    Conference Call and Webcast

    The company will host a conference call and webcast today at 4:30 p.m. ET. To access the call, please dial (866) 501-1537 (U.S.) or +1 (720) 545-0001 (International). The conference call will be webcast live and a link to the webcast can be accessed through Vertex's website at www.vrtx.com in the "Investors" section under "Events and Presentations." To ensure a timely connection, it is recommended that users register at least 15 minutes prior to the scheduled webcast. An archived webcast will be available on the company's website.

    (VRTX-E)

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  8. - Vertex will initiate a Phase 1/2 clinical trial in first half of 2021 -

    - VX-880 is the first stem cell-derived therapy evaluating fully differentiated pancreatic islet cells for the treatment of T1D -

    Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today announced that the U.S. Food and Drug Administration (FDA) has cleared the IND, enabling the company to proceed with initiating a clinical trial for VX-880, an investigational stem cell-derived, fully differentiated pancreatic islet cell therapy to treat T1D. Vertex plans to initiate a Phase 1/2 clinical trial in the first half of 2021 in patients who have T1D with impaired hypoglycemic awareness and severe hypoglycemia.

    "As we celebrate the 100th anniversary of the discovery of insulin…

    - Vertex will initiate a Phase 1/2 clinical trial in first half of 2021 -

    - VX-880 is the first stem cell-derived therapy evaluating fully differentiated pancreatic islet cells for the treatment of T1D -

    Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today announced that the U.S. Food and Drug Administration (FDA) has cleared the IND, enabling the company to proceed with initiating a clinical trial for VX-880, an investigational stem cell-derived, fully differentiated pancreatic islet cell therapy to treat T1D. Vertex plans to initiate a Phase 1/2 clinical trial in the first half of 2021 in patients who have T1D with impaired hypoglycemic awareness and severe hypoglycemia.

    "As we celebrate the 100th anniversary of the discovery of insulin this year, we are excited to bring a first-in-class cell therapy to the clinic with the potential to meaningfully impact people living with T1D," said Bastiano Sanna, Ph.D., Executive Vice President and Chief of Cell and Genetic Therapies at Vertex. "We look forward to getting our clinical program underway and testing our unique approach of replacing pancreatic islet cells, which are destroyed in people with type 1 diabetes, with our stem cell-derived fully differentiated insulin-producing pancreatic islet cells."

    About VX-880

    VX-880, formerly known as STx-02, is an investigational allogeneic human stem cell-derived islet cell therapy that is being evaluated for patients who have T1D with impaired hypoglycemic awareness and severe hypoglycemia. VX-880 has the potential to restore the body's ability to regulate glucose levels by restoring pancreatic islet cell function, including insulin production.

    The VX-880 clinical trial will involve an infusion of fully differentiated, functional islet cells, as well as the chronic administration of concomitant immunosuppressive therapy, to protect the islet cells from immune rejection.

    About the Phase 1/2 Clinical Trial

    The clinical trial is a Phase 1/2, single-arm, open-label study in subjects who have T1D with impaired hypoglycemic awareness and severe hypoglycemia. This will be a sequential, multi-part clinical trial to evaluate the safety and efficacy of different doses of VX-880. Approximately 17 patients will be enrolled in the clinical trial.

    About Type 1 Diabetes

    T1D results from the autoimmune destruction of insulin-producing islet cells in the pancreas, leading to loss of insulin production and impairment of blood glucose control. The absence of insulin leads to abnormalities in how the body processes nutrients, leading to high blood glucose levels. High blood glucose can lead to diabetic ketoacidosis and over time, to complications such as kidney disease/failure, eye disease (including vision loss), heart disease, stroke, nerve damage and even death. Due to the limitations and complexities of insulin delivery systems, it can be difficult to achieve and maintain balance in glucose control in patients with T1D. Hypoglycemia remains a critical limiting factor in glycemic management, and severe hypoglycemia can cause loss of consciousness, coma, seizures, injury, and can be fatal.

    There are currently limited treatment options beyond insulin for the management of T1D.

    About Vertex

    Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) — a rare, life-threatening genetic disease — and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational small molecule medicines in other serious diseases where it has deep insight into causal human biology, including pain, alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases. In addition, Vertex has a rapidly expanding pipeline of cell and genetic therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.

    Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including 11 consecutive years on Science magazine's Top Employers list and a best place to work for LGBTQ equality by the Human Rights Campaign. For company updates and to learn more about Vertex's history of innovation, visit www.vrtx.com or follow us on Facebook, Twitter, LinkedIn, YouTube and Instagram.

    Special Note Regarding Forward-Looking Statements

    This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, statements made by Bastiano Sanna, Ph.D., in this press release, statements regarding the development, plans and expectations for our T1D pipeline program, including our plans to initiate a Phase 1/2 clinical trial in people with T1D and expected timeline of our clinical trials, statements regarding patient enrollment and dosing, statements regarding potential clinical trial results and anticipated benefits of VX-880, and our plans to provide further updates on our T1D pipeline program. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that the FDA may not approve our IND, that data from a limited number of patients may not be indicative of final clinical trial results, that data from the company's development programs may not support registration or further development due to safety, efficacy or other reasons, that the COVID-19 pandemic may impact the status or progress of our clinical trials, and other risks listed under the heading "Risk Factors" in Vertex's most recent annual report and subsequent quarterly reports filed with the Securities and Exchange Commission at www.sec.gov and available through the company's website at www.vrtx.com. You should not place undue reliance on these statements. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

    (VRTX-GEN)

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  9. -FDA grants Priority Review of the application and sets a PDUFA target action date of June 8, 2021-

    Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today announced that the U.S. Food and Drug Administration (FDA) has accepted its supplemental New Drug Application (sNDA) to expand the use of TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) to include children ages 6 through 11 years old who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or a mutation in the CFTR gene that is responsive based on in vitro data. The FDA has granted Priority Review of the sNDA and assigned a Prescription Drug User Fee Act (PDUFA) target action date of June 8, 2021. The submission was supported…

    -FDA grants Priority Review of the application and sets a PDUFA target action date of June 8, 2021-

    Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today announced that the U.S. Food and Drug Administration (FDA) has accepted its supplemental New Drug Application (sNDA) to expand the use of TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) to include children ages 6 through 11 years old who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or a mutation in the CFTR gene that is responsive based on in vitro data. The FDA has granted Priority Review of the sNDA and assigned a Prescription Drug User Fee Act (PDUFA) target action date of June 8, 2021. The submission was supported by data from a global Phase 3 study of TRIKAFTA in children ages 6 through 11 years old with cystic fibrosis (CF) who have either two copies of the F508del mutation or one copy of the F508del mutation and one minimal function mutation.

    "If approved for this expanded use, we will have the opportunity to treat the underlying cause of the disease earlier in life with TRIKAFTA and potentially benefit approximately 1,500 additional children with CF," said Carmen Bozic, M.D., Executive Vice President and Chief Medical Officer at Vertex. "Since our initial approval of TRIKAFTA in 2019, we have continued to work tirelessly to bring this medicine to those waiting as quickly as possible. We look forward to working with the Agency as they review the application over the course of the coming months."

    Vertex plans to submit a Marketing Authorization Application (MAA) variation for the triple combination in the European Union in the first half of 2021 for children ages 6 through 11. Global regulatory filings in additional markets, including Canada and Australia, are planned in the coming months for this age group.

    About Cystic Fibrosis

    Cystic Fibrosis (CF) is a rare, life-shortening genetic disease affecting more than 80,000 people globally. CF is a progressive, multi-system disease that affects the lungs, liver, GI tract, sinuses, sweat glands, pancreas and reproductive tract. CF is caused by a defective and/or missing CFTR protein resulting from certain mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF. While there are many different types of CFTR mutations that can cause the disease, the vast majority of all people with CF have at least one F508del mutation. These mutations, which can be determined by a genetic test, or genotyping test, lead to CF by creating non-working and/or too few CFTR proteins at the cell surface. The defective function and/or absence of CFTR protein results in poor flow of salt and water into and out of the cells in a number of organs. In the lungs, this leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage in many patients that eventually leads to death. The median age of death is in the early 30s.

    INDICATION AND IMPORTANT SAFETY INFORMATION FOR TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor)

    What is TRIKAFTA?

    TRIKAFTA is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients aged 12 years and older who have at least one copy of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or another mutation that is responsive to treatment with TRIKAFTA. Patients should talk to their doctor to learn if they have an indicated CF gene mutation. It is not known if TRIKAFTA is safe and effective in children under 12 years of age.

    Patients should not take TRIKAFTA if they take certain medicines or herbal supplements, such as: the antibiotics rifampin or rifabutin; seizure medications such as phenobarbital, carbamazepine, or phenytoin; or St. John's wort.

    Before taking TRIKAFTA, patients should tell their doctor about all of their medical conditions, including if they: have kidney problems; have or have had liver problems; are pregnant or plan to become pregnant because it is not known if TRIKAFTA will harm an unborn baby; or are breastfeeding or planning to breastfeed because it is not known if TRIKAFTA passes into breast milk.

    TRIKAFTA may affect the way other medicines work, and other medicines may affect how TRIKAFTA works. Therefore, the dose of TRIKAFTA may need to be adjusted when taken with certain medications. Patients should especially tell their doctor if they take antifungal medications such as ketoconazole, itraconazole, posaconazole, voriconazole, or fluconazole; or antibiotics such as telithromycin, clarithromycin, or erythromycin.

    TRIKAFTA can cause dizziness in some people who take it. Patients should not drive a car, operate machinery, or do anything that needs them to be alert until they know how TRIKAFTA affects them.

    Patients should avoid food or drink that contains grapefruit while they are taking TRIKAFTA.

    TRIKAFTA can cause serious side effects, including:

    High liver enzymes in the blood, which is a common side effect in people treated with TRIKAFTA. The patient's doctor will do blood tests to check their liver before they start TRIKAFTA, every 3 months during the first year of taking TRIKAFTA, and every year while taking TRIKAFTA. Patients should call their doctor right away if they have any of the following symptoms of liver problems: pain or discomfort in the upper right stomach (abdominal) area; yellowing of the skin or the white part of the eyes; loss of appetite; nausea or vomiting; dark, amber-colored urine.

    Abnormality of the eye lens (cataract) in some children and adolescents treated with TRIKAFTA. If the patient is a child or adolescent, their doctor should perform eye examinations before and during treatment with TRIKAFTA to look for cataracts.

    The most common side effects of TRIKAFTA include headache, diarrhea, upper respiratory tract infection (common cold) including stuffy and runny nose, stomach (abdominal) pain, inflamed sinuses, increase in liver enzymes, increase in a certain blood enzyme called creatine phosphokinase, rash, flu (influenza), and increase in blood bilirubin.

    These are not all the possible side effects of TRIKAFTA. Please click the product link to see the full Prescribing Information for TRIKAFTA.

    About Vertex

    Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) — a rare, life-threatening genetic disease — and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational small molecule medicines in other serious diseases where it has deep insight into causal human biology, including pain, alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases. In addition, Vertex has a rapidly expanding pipeline of genetic and cell therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.

    Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including 11 consecutive years on Science magazine's Top Employers list and a best place to work for LGBTQ equality by the Human Rights Campaign. For company updates and to learn more about Vertex's history of innovation, visit www.vrtx.com or follow us on Facebook, Twitter, LinkedIn, YouTube and Instagram.

    Special Note Regarding Forward-Looking Statements

    This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, statements made by Dr. Carmen Bozic in this press release, statements regarding our plans to submit an MAA in the EU and other global regulatory filings in additional markets, our expectations regarding the number of patients newly eligible for TRIKAFTA, and statements regarding the potential benefits of TRIKAFTA. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that the sNDA to expand the use of TRIKAFTA to include children ages 6 through 11 could not be approved on a timely basis, or at all, that data from the company's development programs may not support registration or further development of its compounds due to safety, efficacy or other reasons, and other risks listed under the heading "Risk Factors" in Vertex's most recent annual report and subsequent quarterly reports filed with the Securities and Exchange Commission (SEC) and available through the company's website at www.vrtx.com and on the SEC's website at www.sec.gov. You should not place undue reliance on these statements. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

    (VRTX-GEN)

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  10. Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) will report its fourth-quarter 2020 financial results on Monday, February 1, 2021 after the financial markets close. The company will host a conference call and webcast at 4:30 p.m. ET. To access the call, please dial (866) 501-1537 (U.S.) or +1 (720) 545-0001 (International).

    The conference call will be webcast live and a link to the webcast can be accessed through Vertex's website at www.vrtx.com in the "Investors" section. To ensure a timely connection, it is recommended that participants register at least 15 minutes prior to the scheduled webcast. An archived webcast will be available on the company's website.

    About Vertex

    Vertex is a global biotechnology company that invests in scientific…

    Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) will report its fourth-quarter 2020 financial results on Monday, February 1, 2021 after the financial markets close. The company will host a conference call and webcast at 4:30 p.m. ET. To access the call, please dial (866) 501-1537 (U.S.) or +1 (720) 545-0001 (International).

    The conference call will be webcast live and a link to the webcast can be accessed through Vertex's website at www.vrtx.com in the "Investors" section. To ensure a timely connection, it is recommended that participants register at least 15 minutes prior to the scheduled webcast. An archived webcast will be available on the company's website.

    About Vertex

    Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) — a rare, life-threatening genetic disease — and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational small molecule medicines in other serious diseases where it has deep insight into causal human biology, including pain, alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases. In addition, Vertex has a rapidly expanding pipeline of genetic and cell therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.

    Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including 11 consecutive years on Science magazine's Top Employers list and a best place to work for LGBTQ equality by the Human Rights Campaign. For company updates and to learn more about Vertex's history of innovation, visit www.vrtx.com or follow us on Facebook, Twitter, LinkedIn, YouTube and Instagram.

    (VRTX-WEB)

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  11. Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today announced that management will present at the J.P. Morgan Healthcare Conference on Monday, January 11, 2021 at 9:10 a.m. ET (6:10 a.m. PT).

    The audio portion of management's remarks can be accessed live through Vertex's website, www.vrtx.com, in the "Investors" section under the "News and Events" page. A replay of the conference webcast will be archived on the company's website.

    About Vertex

    Vertex is a global biotechnology company that invests in scientific innovation to create medicines for people with serious diseases. The company has multiple approved medicines that treat cystic fibrosis (CF) — a rare, life-threatening genetic disease — and has several ongoing clinical and research…

    Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today announced that management will present at the J.P. Morgan Healthcare Conference on Monday, January 11, 2021 at 9:10 a.m. ET (6:10 a.m. PT).

    The audio portion of management's remarks can be accessed live through Vertex's website, www.vrtx.com, in the "Investors" section under the "News and Events" page. A replay of the conference webcast will be archived on the company's website.

    About Vertex

    Vertex is a global biotechnology company that invests in scientific innovation to create medicines for people with serious diseases. The company has multiple approved medicines that treat cystic fibrosis (CF) — a rare, life-threatening genetic disease — and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational small molecule medicines in other serious diseases where it has deep insight into causal human biology, including pain, alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases. In addition, Vertex has a rapidly expanding pipeline of genetic and cell therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.

    Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including 11 consecutive years on Science magazine's Top Employers list and a best place to work for LGBTQ equality by the Human Rights Campaign. For company updates and to learn more about Vertex's history of innovation, visit www.vrtx.com

    (VRTX-WEB)

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  12. Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today announced its New Drug Submission for TRIKAFTA®, Vertex's investigational triple combination medicine, has been accepted for Priority Review by Health Canada for the treatment of cystic fibrosis (CF) in people ages 12 years and older.

    "We are pleased this submission has been accepted for Priority Review by Health Canada, and we anticipate this accelerated review process will enable access for patients as early as possible," said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex.

    With Priority Review, the conventional review timeline of 300 days is reduced to 180 days. The expected approval target by Health…

    Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today announced its New Drug Submission for TRIKAFTA®, Vertex's investigational triple combination medicine, has been accepted for Priority Review by Health Canada for the treatment of cystic fibrosis (CF) in people ages 12 years and older.

    "We are pleased this submission has been accepted for Priority Review by Health Canada, and we anticipate this accelerated review process will enable access for patients as early as possible," said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex.

    With Priority Review, the conventional review timeline of 300 days is reduced to 180 days. The expected approval target by Health Canada is in the first half of 2021.

    About Cystic Fibrosis

    Cystic fibrosis (CF) is a rare, life-shortening genetic disease affecting approximately 75,000 people worldwide. CF is a progressive, multi-system disease that affects the lungs, liver, GI tract, sinuses, sweat glands, pancreas and reproductive tract. CF is caused by a defective and/or missing CFTR protein resulting from certain mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF. While there are many different types of CFTR mutations that can cause the disease, the vast majority of all people with CF have at least one F508del mutation. These mutations, which can be determined by a genetic test, or genotyping test, lead to CF by creating non-working and/or too few CFTR proteins at the cell surface. The defective function and/or absence of CFTR protein results in poor flow of salt and water into and out of the cells in a number of organs. In the lungs, this leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage in many patients that eventually leads to death. The median age of death is in the early 30s.

    About Vertex

    Vertex is a global biotechnology company that invests in scientific innovation to create medicines for people with serious diseases. The company has multiple approved medicines that treat cystic fibrosis (CF) — a rare, life- threatening genetic disease — and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational small molecule medicines in other serious diseases where it has deep insight into causal human biology, including pain, alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases. In addition, Vertex has a rapidly expanding pipeline of genetic and cell therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.

    Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including 11 consecutive years on Science magazine's Top Employers list and a best place to work for LGBTQ equality by the Human Rights Campaign. For company updates and to learn more about Vertex's history of innovation, visit www.vrtx.com or follow us on Facebook, Twitter, LinkedIn, YouTube and Instagram.

    Special Note Regarding Forward-Looking Statements

    This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, statements made by Carmen Bozic in this press release, including expectations for patient access to our medicine, and statements regarding the anticipated timing of the expected approval target by Health Canada. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that the New Drug Submission to Health Canada may not be approved in the expected timeline, or at all, that data from the company's development programs may not support registration or further development of its compounds due to safety, efficacy or other reasons, and other risks listed under the heading "Risk Factors" in Vertex's most recent annual report and subsequent quarterly reports filed with the Securities and Exchange Commission at www.sec.gov and available through the company's website at www.vrtx.com. You should not place undue reliance on these statements. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

    (VRTX-GEN)

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  13. BOSTON, Dec. 28, 2020 /CNW/ - Vertex Pharmaceuticals Incorporated  (NASDAQ:VRTX) today announced its New Drug Submission for TRIKAFTA®, Vertex's investigational triple combination medicine, has been accepted for Priority Review by Health Canada for the treatment of cystic fibrosis (CF) in people ages 12 years and older.

    "We are pleased this submission has been accepted for Priority Review by Health Canada, and we anticipate this accelerated review process will enable access for patients as early as possible," said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex.

    With Priority Review, the conventional review timeline of 300 days is reduced to 180 days. The expected approval target by Health Canada is in the first half of 2021.

    About Cystic Fibrosis

    Cystic fibrosis (CF) is a rare, life-shortening genetic disease affecting approximately 75,000 people worldwide. CF is a progressive, multi-system disease that affects the lungs, liver, GI tract, sinuses, sweat glands, pancreas and reproductive tract. CF is caused by a defective and/or missing cystic fibrosis transmembrane conductance regulator (CFTR) protein resulting from certain mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF. While there are many different types of CFTR mutations that can cause the disease, the vast majority of all people with CF have at least one F508del mutation. These mutations, which can be determined by a genetic test, or genotyping test, lead to CF by creating non-working and/or too few CFTR proteins at the cell surface. The defective function and/or absence of CFTR protein results in poor flow of salt and water into and out of the cells in a number of organs. In the lungs, this leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage in many patients that eventually leads to death. The median age of death is in the early 30s.

    About Vertex

    Vertex is a global biotechnology company that invests in scientific innovation to create medicines for people with serious diseases. The company has multiple approved medicines that treat cystic fibrosis (CF) — a rare, life-threatening genetic disease — and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational small molecule medicines in other serious diseases where it has deep insight into causal human biology, including pain, alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases. In addition, Vertex has a rapidly expanding pipeline of genetic and cell therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.

    Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including 11 consecutive years on Science magazine's Top Employers list and a best place to work for LGBTQ equality by the Human Rights Campaign. For company updates and to learn more about Vertex's history of innovation, visit www.vrtx.com

    Special Note Regarding Forward-looking Statements

    This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, statements made by Carmen Bozic in this press release, including expectations for patient access to our medicine, and statements regarding the anticipated timing of the expected approval target by Health Canada. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that the New Drug Submission to Health Canada may not be approved in the expected timeline, or at all, that data from the company's development programs may not support registration or further development of its compounds due to safety, efficacy or other reasons, and other risks listed under the heading "Risk Factors" in Vertex's most recent annual report and subsequent quarterly reports filed with the Securities and Exchange Commission at www.sec.gov and available through the company's website at www.vrtx.com. You should not place undue reliance on these statements. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

    (VRTX-GEN)

    SOURCE Vertex Pharmaceuticals (Canada) Inc.

    Cision View original content to download multimedia: http://www.newswire.ca/en/releases/archive/December2020/28/c9962.html

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  14. Vertex receives exclusive options to license a number of Skyhawk's product candidates directed at serious diseases

    Skyhawk receives $40 million upfront as well as potential milestones and royalty payments on future sales

    Skyhawk Therapeutics, Inc. and Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) announced today a strategic research collaboration and licensing agreement aimed at the discovery and development of novel small molecules that modulate RNA splicing for the treatment of serious diseases.

    This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20201222005032/en/

    "We believe that splice site modulation holds significant promise for the treatment of diseases which today have limited…

    Vertex receives exclusive options to license a number of Skyhawk's product candidates directed at serious diseases

    Skyhawk receives $40 million upfront as well as potential milestones and royalty payments on future sales

    Skyhawk Therapeutics, Inc. and Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) announced today a strategic research collaboration and licensing agreement aimed at the discovery and development of novel small molecules that modulate RNA splicing for the treatment of serious diseases.

    This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20201222005032/en/

    "We believe that splice site modulation holds significant promise for the treatment of diseases which today have limited or no therapeutic options," said Mark Bunnage, D.Phil., Senior Vice President and Site Head, Boston Research at Vertex. "This collaboration brings Skyhawk's innovative technology together with Vertex's research and development experience; and fits perfectly with our strategy of investing in new technologies that will help us transform multiple serious diseases."

    "We are excited to enter this collaboration with Vertex, a global leader in creating transformative medicines through serial innovation," said Bill Haney, Chief Executive Officer of Skyhawk. "In collaboration with the Vertex team, we look forward to using our SkySTAR™ platform to discover and develop novel small molecule therapeutics that modulate RNA splicing which have the potential to transform the lives of patients with serious diseases."

    Under the collaboration agreement, Vertex will pay Skyhawk $40 million upfront. Skyhawk will grant Vertex options to exclusively license worldwide intellectual property rights to candidates discovered and developed under the collaboration that are directed to program targets. Following Vertex's exercise of its options, Vertex will be responsible for further development and commercialization. Skyhawk is also eligible to receive up to $2.2 billion in potential milestone payments, as well as potential royalties on future sales.

    About Skyhawk Therapeutics

    Skyhawk develops and commercializes therapies using its novel SkySTAR™ platform, building small molecules that provide breakthrough treatments for patients. Skyhawk has productive collaborations across a broad range of disease areas ranging from neurodegenerative disease to oncology.

    For more information visit:

    www.skyhawktx.com

    https://www.linkedin.com/company/skyhawk-therapeutics/

    https://twitter.com/Skyhawk_Tx

    About Vertex Pharmaceuticals

    Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) — a rare, life-threatening genetic disease — and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational small molecule medicines in other serious diseases where it has deep insight into causal human biology, including pain, alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases. In addition, Vertex has a rapidly expanding pipeline of genetic and cell therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.

    Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including 11 consecutive years on Science magazine's Top Employers list and a best place to work for LGBTQ equality by the Human Rights Campaign. For company updates and to learn more about Vertex's history of innovation, visit www.vrtx.com or follow us on Facebook, Twitter, LinkedIn, YouTube and Instagram.

    Vertex/Skyhawk Forward-Looking Statements

    This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, statements by Mark Bunnage, D.Phil. and Bill Haney in this press release, statements about the potential benefits and results that may be achieved through Vertex's collaboration with Skyhawk, and statements regarding upfront and milestone payments and potential royalties on future sales. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that the anticipated benefits and potential of Vertex's collaboration with Skyhawk may not be achieved, that data may not support further development of the therapies subject to the collaboration due to safety, efficacy or other reasons, and other risks listed under the heading "Risk Factors" in Vertex's annual report and subsequent quarterly reports filed with the Securities and Exchange Commission (SEC) and available through Vertex's website at www.vrtx.com and on the SEC's website at www.sec.gov. You should not place undue reliance on these statements. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

    (VRTX-GEN)

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  15. - More than 600 people with certain rare CF mutations are now eligible for TRIKAFTA, SYMDEKO or KALYDECO -

    Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today announced the U.S. Food and Drug Administration (FDA) expanded the eligibility for TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) to include people with cystic fibrosis (CF) ages 12 years and older with certain mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that are responsive to TRIKAFTA based on in vitro data. SYMDEKO® (tezacaftor/ivacaftor and ivacaftor) and KALYDECO® (ivacaftor) also received approvals to include additional responsive mutations in people with CF ages 6 years and older and age 4 months and older, respectively. These…

    - More than 600 people with certain rare CF mutations are now eligible for TRIKAFTA, SYMDEKO or KALYDECO -

    Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today announced the U.S. Food and Drug Administration (FDA) expanded the eligibility for TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) to include people with cystic fibrosis (CF) ages 12 years and older with certain mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that are responsive to TRIKAFTA based on in vitro data. SYMDEKO® (tezacaftor/ivacaftor and ivacaftor) and KALYDECO® (ivacaftor) also received approvals to include additional responsive mutations in people with CF ages 6 years and older and age 4 months and older, respectively. These approvals allow more than 600 people with CF not previously eligible for these medicines an opportunity to potentially benefit from treatment that targets the underlying cause of their disease.

    "The approval for expanded use of three of our CF medicines based on our well-established in vitro model is a testament to the relentless commitment of our scientists to reach our goal of developing treatments for all people with CF," said Reshma Kewalramani, M.D., Chief Executive Officer and President, Vertex. "We remain as committed today to reaching every patient who might benefit from our medicines as when we first started out on this journey 20 years ago, and this important milestone now enables hundreds of people with CF access to a treatment option to address the underlying cause of their disease — many for the first time."

    TRIKAFTA was previously approved for people with at least one F508del mutation and is now approved for 177 additional mutations; SYMDEKO is now approved for 127 additional mutations, for a total of 154 SYMDEKO-responsive mutations; and KALYDECO is now approved for an additional 59 mutations, for a total of 97 KALYDECO-responsive mutations. In addition, for certain people with CF who are currently eligible for KALYDECO, this approval allows them to also be eligible for SYMDEKO or TRIKAFTA; and similarly, for those who are currently eligible for SYMDEKO, this approval allows them to also be eligible for TRIKAFTA.

    The full list of mutations for TRIKAFTA, SYMDEKO and KALYDECO can be found within the updated full Prescribing Information for each respective product. In addition, people with CF and their families can search eligibility for Vertex CF medicines through vertextreatments.com.

    About Cystic Fibrosis

    Cystic Fibrosis (CF) is a rare, life-shortening genetic disease affecting approximately 75,000 people worldwide. CF is a progressive, multi-system disease that affects the lungs, liver, GI tract, sinuses, sweat glands, pancreas and reproductive tract. CF is caused by a defective and/or missing CFTR protein resulting from certain mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF. While there are many different types of CFTR mutations that can cause the disease, the vast majority of all people with CF have at least one F508del mutation. These mutations, which can be determined by a genetic test, or genotyping test, lead to CF by creating non-working and/or too few CFTR proteins at the cell surface. The defective function and/or absence of CFTR protein results in poor flow of salt and water into and out of the cells in a number of organs. In the lungs, this leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage in many patients that eventually leads to death. The median age of death is in the early 30s.

    INDICATION AND IMPORTANT SAFETY INFORMATION FOR KALYDECO® (ivacaftor), SYMDEKO® (tezacaftor/ivacaftor and ivacaftor), and TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor)

    What is KALYDECO?

    KALYDECO is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients

    age 4 months and older who have at least one mutation in their CF gene that is responsive to KALYDECO. Patients should talk to their doctor to learn if they have an indicated CF gene mutation. It is not known if KALYDECO is safe and effective in children under 4 months of age.

    What is SYMDEKO?

    SYMDEKO is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients age 6 years and older who have two copies of the F508del mutation, or who have at least one mutation in the CF gene that is responsive to treatment with SYMDEKO. Patients should talk to their doctor to learn if they have an indicated CF gene mutation. It is not known if SYMDEKO is safe and effective in children under 6 years of age.

    What is TRIKAFTA?

    TRIKAFTA is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients aged 12 years and older who have at least one copy of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or another mutation that is responsive to treatment with TRIKAFTA. Patients should talk to their doctor to learn if they have an indicated CF gene mutation. It is not known if TRIKAFTA is safe and effective in children under 12 years of age.

    Patients should not take KALYDECO, SYMDEKO, or TRIKAFTA if they take certain medicines or herbal supplements, such as: the antibiotics rifampin or rifabutin; seizure medications such as phenobarbital, carbamazepine, or phenytoin; or St. John's wort.

    Before taking KALYDECO, SYMDEKO, or TRIKAFTA, patients should tell their doctor about all of their medical conditions, including if they: have kidney problems; have or have had liver problems; are pregnant or plan to become pregnant because it is not known if KALYDECO, SYMDEKO, or TRIKAFTA will harm an unborn baby; or are breastfeeding or planning to breastfeed because it is not known if KALYDECO, SYMDEKO, or TRIKAFTA passes into breast milk. Before taking KALYDECO, patients should tell their doctor if they drink grapefruit juice or eat grapefruit.

    KALYDECO, SYMDEKO, or TRIKAFTA may affect the way other medicines work, and other medicines may affect how KALYDECO, SYMDEKO, or TRIKAFTA work. Therefore, the dose of KALYDECO, SYMDEKO, or TRIKAFTA may need to be adjusted when taken with certain medications. Patients should especially tell their doctor if they take antifungal medications such as ketoconazole, itraconazole, posaconazole, voriconazole, or fluconazole; or antibiotics such as telithromycin, clarithromycin, or erythromycin.

    KALYDECO, SYMDEKO, or TRIKAFTA can cause dizziness in some people who take it. Patients should not drive a car, use machinery, or do anything that needs them to be alert until they know how KALYDECO, SYMDEKO, or TRIKAFTA affects them.

    Patients should avoid food or drink containing grapefruit while taking KALYDECO, SYMDEKO or TRIKAFTA.

    KALYDECO, SYMDEKO, and TRIKAFTA can cause serious side effects, such as:

    High liver enzymes in the blood have been reported in patients receiving KALYDECO, SYMDEKO, or TRIKAFTA. The patient's doctor will do blood tests to check their liver before starting treatment with KALYDECO, SYMDEKO, or TRIKAFTA, every 3 months during the first year of treatment, and every year while on treatment. Patients should call their doctor right away if they have any of the following symptoms of liver problems: pain or discomfort in the upper right stomach (abdominal) area; yellowing of their skin or the white part of their eyes; loss of appetite; nausea or vomiting; or dark, amber-colored urine.

    Abnormality of the eye lens (cataract) in some children and adolescents treated with KALYDECO, SYMDEKO, or TRIKAFTA. If the patient is a child or adolescent, their doctor should perform eye examinations before and during treatment with KALYDECO, SYMDEKO, or TRIKAFTA to look for cataracts.

    The most common side effects of KALYDECO include headache; upper respiratory tract infection (common cold), which includes sore throat, nasal or sinus congestion, and runny nose; stomach (abdominal) pain; diarrhea; rash; nausea; and dizziness.

    The most common side effects of SYMDEKO include headache, nausea, sinus congestion, and dizziness.

    The most common side effects of TRIKAFTA include headache, diarrhea, upper respiratory tract infection (common cold) including stuffy and runny nose, stomach (abdominal) pain, inflamed sinuses, increase in liver enzymes, increase in a certain blood enzyme called creatine phosphokinase, rash, flu (influenza), and increase in blood bilirubin.

    These are not all the possible side effects of KALYDECO, SYMDEKO, or TRIKAFTA. Please click product link to see the full Prescribing Information for KALYDECO, SYMDEKO or TRIKAFTA.

    About Vertex

    Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) — a rare, life-threatening genetic disease — and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational small molecule medicines in other serious diseases where it has deep insight into causal human biology, including pain, alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases. In addition, Vertex has a rapidly expanding pipeline of genetic and cell therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.

    Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including 11 consecutive years on Science magazine's Top Employers list and a best place to work for LGBTQ equality by the Human Rights Campaign. For company updates and to learn more about Vertex's history of innovation, visit www.vrtx.com or follow us on Facebook, Twitter, LinkedIn, YouTube and Instagram.

    Special Note Regarding Forward-Looking Statements

    This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, statements made by Dr. Reshma Kewalramani in this press release, statements regarding the eligible patient population for TRIKAFTA, SYMDEKO and KALYDECO, our expectations regarding the number of patients newly eligible for TRIKAFTA, SYMDEKO and KALYDECO, and statements regarding the potential benefits of TRIKAFTA, SYMDEKO and KALYDECO. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that data from the company's development programs may not support registration or further development of its compounds due to safety, efficacy or other reasons, and other risks listed under the heading "Risk Factors" in Vertex's most recent annual report and subsequent quarterly reports filed with the Securities and Exchange Commission (SEC) and available through the company's website at www.vrtx.com and on the SEC's website at www.sec.gov. You should not place undue reliance on these statements. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

    (VRTX-GEN)

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  16. - Beta thalassemia: All seven patients were transfusion independent with 3 to 18 months of follow-up after CTX001 infusion -

    - Sickle cell disease: All three patients were free of vaso-occlusive crises with 3 to 15 months of follow-up after CTX001 infusion -

    - Nineteen patients have been dosed with CTX001 across both programs -

    - The New England Journal of Medicine publishes CTX001 manuscript containing the first report of investigational use of CRISPR/Cas9-based gene editing to treat inherited diseases in humans -

    ZUG, Switzerland and CAMBRIDGE, Mass. and BOSTON, Dec. 05, 2020 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (NASDAQ:CRSP) and Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today announced…

    - Beta thalassemia: All seven patients were transfusion independent with 3 to 18 months of follow-up after CTX001 infusion -

    - Sickle cell disease: All three patients were free of vaso-occlusive crises with 3 to 15 months of follow-up after CTX001 infusion -

    - Nineteen patients have been dosed with CTX001 across both programs -

    - The New England Journal of Medicine publishes CTX001 manuscript containing the first report of investigational use of CRISPR/Cas9-based gene editing to treat inherited diseases in humans -

    ZUG, Switzerland and CAMBRIDGE, Mass. and BOSTON, Dec. 05, 2020 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (NASDAQ:CRSP) and Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today announced new data on a total of 10 patients treated with the investigational CRISPR/Cas9-based gene-editing therapy, CTX001, that show a consistent and sustained response to treatment. All seven patients with transfusion-dependent beta thalassemia (TDT), including three who have either a severe or b0/b0 genotype, were transfusion independent at last follow-up and all three patients with sickle cell disease (SCD) were free of vaso-occlusive crises (VOCs) from CTX001 infusion through last follow-up. These data will be presented during the Scientific Plenary at the annual ASH Meeting and Exposition on December 6, 2020. A summary of the results from the CLIMB-111 and CLIMB-121 Phase 1/2 clinical studies is provided below.

    The companies also announced that The New England Journal of Medicine (NEJM) has published an independently peer-reviewed article entitled "CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β Thalassemia." The article includes detailed information on the first patient with TDT enrolled in CLIMB-111 and the first patient with severe SCD enrolled in CLIMB-121, at 18 and 15 months of follow-up, respectively.

    CTX001 is being investigated in these two ongoing Phase 1/2 clinical trials as a potential one-time curative therapy for patients suffering from TDT and severe SCD.

    "We are pleased with the data presented at ASH, which demonstrate potential benefit and durability among a larger population of patients with transfusion-dependent beta thalassemia and sickle cell disease," said Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics. "Additionally, the NEJM case study is the first peer-reviewed journal publication for our CRISPR/Cas9 gene therapy, CTX001. Together this is further validation of the potential of CTX001 to become a best-in-class therapy. We plan to continue the rapid advancement of our clinical trials to bring these much-needed therapies to patients."

    "These are the first published results from CRISPR/Cas9 therapy in people with a genetic disease and represent an important milestone in medicine and for our collaboration with CRISPR Therapeutics. Most importantly, these data represent a critical step in our effort to bring transformative and potentially curative therapies to patients," said Reshma Kewalramani, M.D., Chief Executive Officer and President, Vertex. "With clinical proof-of-concept for both beta thalassemia and sickle cell disease and 19 patients dosed, we look forward to continued efforts to bring our investigational treatment to patients living with TDT and SCD as quickly as we can."

    "Our vision with this approach is to use the patient's own stem cells to provide a transformative treatment for these diseases, something almost unimaginable a few years ago," said Dr. Haydar Frangoul, M.D., Medical Director of Pediatric Hematology and Oncology at Sarah Cannon Research Institute, HCA Healthcare's TriStar Centennial Medical Center. "With these data in 10 patients, we can see the potential to fulfill this vision. With more data and longer duration of follow-up, we will hopefully confirm that we have a durable therapy that may transform the lives of many patients."

    CLIMB-111 Trial in TDT: Updated Results

    A total of 13 patients with TDT have been dosed with CTX001, including eight additional patients since the last update in June 2020.

    The seven patients with TDT reported at ASH are patients who had reached at least three months of follow-up after CTX001 dosing and therefore could be assessed for initial safety and efficacy. All seven patients showed a similar pattern of response, with rapid and sustained increases in total hemoglobin, fetal hemoglobin and transfusion independence at last analysis.

    All seven patients were transfusion independent with follow-up ranging from three to 18 months after CTX001 infusion, with normal to near normal total hemoglobin levels at last visit, including total hemoglobin from 9.7 to 14.1 g/dL and fetal hemoglobin from 40.9% to 97.7%.

    Bone marrow allelic editing data collected from four patients with six months of follow-up and from one patient with 12 months of follow-up after CTX001 infusion demonstrated a durable effect.

    The safety data from all seven patients were generally consistent with an autologous stem cell transplant and myeloablative conditioning. There were four serious adverse events (SAEs) considered related or possibly related to CTX001 reported in one patient: headache, hemophagocytic lymphohistiocytosis (HLH), acute respiratory distress syndrome and idiopathic pneumonia syndrome. All four SAEs occurred in the context of HLH and have resolved. The majority of non-serious adverse events were considered mild to moderate.

    CLIMB-121 Trial in Severe SCD: Updated Results

    A total of six patients with SCD have been dosed with CTX001, including four additional patients since the last update in June 2020.

    The three patients reported at ASH are patients who had reached at least three months of follow-up after CTX001 dosing and therefore could be assessed for initial safety and efficacy. All three patients showed a similar pattern of response, with rapid and sustained increases in total hemoglobin and fetal hemoglobin, as well as elimination of VOCs through last analysis.

    All three patients remained VOC-free with follow-up ranging from three to 15 months after CTX001 infusion and had hemoglobin levels in the normal to near normal range at last visit, including total hemoglobin from 11.5 to 13.2 g/dL and fetal hemoglobin levels from 31.3% to 48.0%.

    Bone marrow allelic editing data collected from one patient with six months of follow-up and from one patient with 12 months of follow-up after CTX001 infusion demonstrated a durable effect.

    The safety data from all three patients were generally consistent with an autologous stem cell transplant and myeloablative conditioning. There were no SAEs considered related to CTX001, and the majority of non-serious adverse events were considered mild to moderate.

    About CTX001

    CTX001 is an investigational, autologous, ex vivo CRISPR/Cas9 gene-edited therapy that is being evaluated for patients suffering from TDT or severe SCD, in which a patient's hematopoietic stem cells are edited to produce high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is a form of the oxygen-carrying hemoglobin that is naturally present at birth, which then switches to the adult form of hemoglobin. The elevation of HbF by CTX001 has the potential to alleviate transfusion requirements for patients with TDT and reduce painful and debilitating sickle crises for patients with SCD.

    Based on progress in this program to date, CTX001 has been granted Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the U.S. Food and Drug Administration (FDA) for both TDT and SCD. CTX001 has also been granted Orphan Drug Designation from the European Commission for both TDT and SCD, as well as Priority Medicines (PRIME) designation from the European Medicines Agency (EMA) for SCD.

    CTX001 is being developed under a co-development and co-commercialization agreement between CRISPR Therapeutics and Vertex. Among gene-editing approaches being investigated/evaluated for TDT and SCD, CTX001 is the furthest advanced in clinical development.

    About CLIMB-111

    The ongoing Phase 1/2 open-label trial, CLIMB-Thal-111, is designed to assess the safety and efficacy of a single dose of CTX001 in patients ages 12 to 35 with TDT. The trial will enroll up to 45 patients and follow patients for approximately two years after infusion. Each patient will be asked to participate in a long-term follow-up trial.

    About CLIMB-121

    The ongoing Phase 1/2 open-label trial, CLIMB-SCD-121, is designed to assess the safety and efficacy of a single dose of CTX001 in patients ages 12 to 35 with severe SCD. The trial will enroll up to 45 patients and follow patients for approximately two years after infusion. Each patient will be asked to participate in a long-term follow-up trial.

    About the Gene-Editing Process in These Trials

    Patients who enroll in these trials will have their own hematopoietic stem and progenitor cells collected from peripheral blood. The patient's cells will be edited using the CRISPR/Cas9 technology. The edited cells, CTX001, will then be infused back into the patient as part of a stem cell transplant, a process which involves, among other things, a patient being treated with myeloablative busulfan conditioning. Patients undergoing stem cell transplants may also encounter side effects (ranging from mild to severe) that are unrelated to the administration of CTX001. Patients will initially be monitored to determine when the edited cells begin to produce mature blood cells, a process known as engraftment. After engraftment, patients will continue to be monitored to track the impact of CTX001 on multiple measures of disease and for safety.

    About the CRISPR-Vertex Collaboration

    CRISPR Therapeutics and Vertex entered into a strategic research collaboration in 2015 focused on the use of CRISPR/Cas9 to discover and develop potential new treatments aimed at the underlying genetic causes of human disease. CTX001 represents the first potential treatment to emerge from the joint research program. CRISPR Therapeutics and Vertex will jointly develop and commercialize CTX001 and equally share all research and development costs and profits worldwide.

    About CRISPR Therapeutics

    CRISPR Therapeutics is a leading gene editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 platform. CRISPR/Cas9 is a revolutionary gene editing technology that allows for precise, directed changes to genomic DNA. CRISPR Therapeutics has established a portfolio of therapeutic programs across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine and rare diseases. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic collaborations with leading companies including Bayer, Vertex Pharmaceuticals and ViaCyte, Inc. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Cambridge, Massachusetts, and business offices in San Francisco, California and London, United Kingdom. For more information, please visit www.crisprtx.com.

    CRISPR Therapeutics Forward-Looking Statement

    This press release may contain a number of "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including statements made by Dr. Kulkarni, Dr. Kewalramani and Dr. Frangoul in this press release, as well as statements regarding CRISPR Therapeutics' expectations about any or all of the following: (i) the safety, efficacy and clinical progress of CRISPR Therapeutics' various clinical programs including CTX001; (ii) the status of clinical trials (including, without limitation, the expected timing of data releases) related to product candidates under development by CRISPR Therapeutics and its collaborators, including expectations regarding the data that are being presented in this press release, at the annual ASH Meeting and Exposition, and in the NEJM article; (iii) the expected benefits of CRISPR Therapeutics' collaborations; and (iv) the therapeutic value, development, and commercial potential of CRISPR/Cas9 gene editing technologies and therapies. Without limiting the foregoing, the words "believes," "anticipates," "plans," "expects" and similar expressions are intended to identify forward-looking statements. Although CRISPR Therapeutics believes that such statements are based on reasonable assumptions within the bounds of its knowledge of its business and operations, existing and prospective investors are cautioned that forward-looking statements are inherently uncertain, are neither promises nor guarantees and not to place undue reliance on such statements, which speak only as of the date they are made. Actual performance and results may differ materially from those projected or suggested in the forward-looking statements due to various risks and uncertainties. These risks and uncertainties include, among others: that preliminary data from any clinical trial and initial data from a limited number of patients (as is the case with CTX001 at this time) may not be indicative of final or future trial results; that CTX001 clinical trial results may not be favorable or may not support registration or further development; potential impacts due to the coronavirus pandemic, such as to the timing and progress of clinical trials; that future competitive or other market factors may adversely affect the commercial potential for CTX001; uncertainties regarding the intellectual property protection for CRISPR Therapeutics' technology; and those risks and uncertainties described under the heading "Risk Factors" in CRISPR Therapeutics' most recent annual report on Form 10-K, quarterly report on Form 10-Q, and in any other subsequent filings made by CRISPR Therapeutics with the U.S. Securities and Exchange Commission, which are available on the SEC's website at www.sec.gov. CRISPR Therapeutics disclaims any obligation or undertaking to update or revise any forward-looking statements contained in this press release, other than to the extent required by law.

    CRISPR THERAPEUTICS® word mark and design logo and CTX001™ are trademarks and registered trademarks of CRISPR Therapeutics AG. All other trademarks and registered trademarks are the property of their respective owners.

    About Vertex

    Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) — a rare, life-threatening genetic disease — and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational small molecule medicines in other serious diseases where it has deep insight into causal human biology, including pain, alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases. In addition, Vertex has a rapidly expanding pipeline of genetic and cell therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.

    Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including 11 consecutive years on Science magazine's Top Employers list and a best place to work for LGBTQ equality by the Human Rights Campaign. For company updates and to learn more about Vertex's history of innovation, visit www.vrtx.com or follow us on Facebook, Twitter, LinkedIn, YouTube and Instagram.

    Vertex Special Note Regarding Forward-Looking Statements

    This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, statements made by Dr. Samarth Kulkarni, Dr. Reshma Kewalramani and Dr. Haydar Frangoul in this press release and statements regarding the expectations and plans to present data at the annual ASH Meeting and Exposition, the development, including expected timeline for development, updated data on patients treated to date and new data on additional patients, and the potential benefits and curative therapy of CTX001, our plans and expectations for our clinical trials and clinical trial sites, including statements regarding patient enrollment, and the status of our clinical trials of our product candidates under development by us and our collaborators, including activities at the clinical trial sites and potential outcomes. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that data from a limited number of patients may not be indicative of final clinical trial results, that data from the company's development programs, including its programs with its collaborators, may not support registration or further development of its compounds due to safety, efficacy, or other reasons, that the COVID-19 pandemic may impact the status or progress of our clinical trials and clinical trial sites and the clinical trials and clinical trial sites of our collaborators, including patient enrollment, and other risks listed under the heading "Risk Factors" in Vertex's most recent annual report and subsequent quarterly reports filed with the Securities and Exchange Commission at www.sec.gov and available through the company's website at www.vrtx.com. You should not place undue reliance on these statements or the scientific data presented. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

    (VRTX-GEN)

    CRISPR Therapeutics Investor Contact:

    Susan Kim, +1 617-307-7503

    CRISPR Therapeutics Media Contact:

    Rachel Eides

    WCG on behalf of CRISPR

    +1 617-337-4167

    Vertex Pharmaceuticals Incorporated

    Investors:

    Michael Partridge, +1 617-341-6108

    or

    Zach Barber, +1 617-341-6470

    or

    Brenda Eustace, +1 617-341-6187

    Media:



    or

    U.S.: +1 617-341-6992

    or

    Heather Nichols: +1 617-839-3607

    or

    International: +44 20 3204 5275



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  17. ZUG, Switzerland and CAMBRIDGE, Mass. and BOSTON, Dec. 01, 2020 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (NASDAQ:CRSP) and Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today announced that the companies will host an investor webcast on December 9, 2020 at 8:00 a.m. ET to review clinical data presented during the Plenary Scientific Session at the annual ASH Meeting and Exposition from two ongoing Phase 1/2 clinical trials of the investigational CRISPR/Cas9 gene-editing therapy CTX001 in patients with sickle cell disease and beta thalassemia. The presentation will include speakers from Vertex and CRISPR Therapeutics as well as Haydar Frangoul M.D., Medical Director of Pediatric Hematology and Oncology at Sarah Cannon Research Institute…

    ZUG, Switzerland and CAMBRIDGE, Mass. and BOSTON, Dec. 01, 2020 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (NASDAQ:CRSP) and Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today announced that the companies will host an investor webcast on December 9, 2020 at 8:00 a.m. ET to review clinical data presented during the Plenary Scientific Session at the annual ASH Meeting and Exposition from two ongoing Phase 1/2 clinical trials of the investigational CRISPR/Cas9 gene-editing therapy CTX001 in patients with sickle cell disease and beta thalassemia. The presentation will include speakers from Vertex and CRISPR Therapeutics as well as Haydar Frangoul M.D., Medical Director of Pediatric Hematology and Oncology at Sarah Cannon Research Institute, HCA Healthcare's TriStar Centennial Medical Center, and a principal investigator in the CTX001 clinical studies.

    The conference call will be webcast live and a link to the webcast can be accessed on the CRISPR Therapeutics website at https://crisprtx.gcs-web.com/events in the Investors section under Events and Presentations and on the Vertex website at www.vrtx.com in the "Investors" section. To access the call via phone, please dial (866) 501-1537 (U.S.) or +1 (720) 545-0001 (International). To ensure a timely connection, it is recommended that users register at least 15 minutes prior to the scheduled webcast. An archived webcast will be available on the companies' websites for approximately 30 days.

    This meeting is not an official program of the ASH annual meeting.

    About the CRISPR-Vertex Collaboration

    CRISPR Therapeutics and Vertex entered into a strategic research collaboration in 2015 focused on the use of CRISPR/Cas9 to discover and develop potential new treatments aimed at the underlying genetic causes of human disease. CTX001 represents the first potential treatment to emerge from the joint research program. CRISPR Therapeutics and Vertex will jointly develop and commercialize CTX001 and equally share all research and development costs and profits worldwide.

    About CRISPR Therapeutics

    CRISPR Therapeutics is a leading gene editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 platform. CRISPR/Cas9 is a revolutionary gene editing technology that allows for precise, directed changes to genomic DNA. CRISPR Therapeutics has established a portfolio of therapeutic programs across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine and rare diseases. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic collaborations with leading companies including Bayer, Vertex Pharmaceuticals and ViaCyte, Inc. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Cambridge, Massachusetts, and business offices in San Francisco, California and London, United Kingdom. For more information, please visit www.crisprtx.com.

    CRISPR Therapeutics Forward-Looking Statement

    This press release may contain a number of "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including statements regarding CRISPR Therapeutics' expectations about plans to review data presented at the annual ASH Meeting and Exposition from two ongoing Phase 1/2 clinical trials of CTX001 in patients with sickle cell disease and beta thalassemia, as well as the anticipated speakers participating in the investor webcast. Although CRISPR Therapeutics believes that such statements are based on reasonable assumptions within the bounds of its knowledge of its business and operations, existing and prospective investors are cautioned that forward-looking statements are inherently uncertain and not to place undue reliance on such statements, which speak only as of the date they are made. Actual performance and results may differ materially from those projected or suggested in the forward-looking statements due to various risks and uncertainties. These risks and uncertainties include, among others: the potential that preliminary data from any clinical trial not to be indicative of final trial results; the potential that clinical trial results may not support registration or further development; uncertainties regarding the intellectual property protection for CRISPR Therapeutics' technology; and those risks and uncertainties described under the heading "Risk Factors" in CRISPR Therapeutics' most recent annual report on Form 10-K, quarterly report on Form 10-Q, and in any other subsequent filings made by CRISPR Therapeutics with the U.S. Securities and Exchange Commission, which are available on the SEC's website at www.sec.gov. CRISPR Therapeutics disclaims any obligation or undertaking to update or revise any forward-looking statements contained in this press release, other than to the extent required by law.

    CRISPR THERAPEUTICS® word mark and design logo and CTX001are trademarks and registered trademarks of CRISPR Therapeutics AG. All other trademarks and registered trademarks are the property of their respective owners.

    About Vertex

    Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) — a rare, life-threatening genetic disease — and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational small molecule medicines in other serious diseases where it has deep insight into causal human biology, including pain, alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases. In addition, Vertex has a rapidly expanding pipeline of genetic and cell therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.

    Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including 11 consecutive years on Science magazine's Top Employers list and a best place to work for LGBTQ equality by the Human Rights Campaign. For company updates and to learn more about Vertex's history of innovation, visit www.vrtx.com or follow us on Facebook, Twitter, LinkedIn, YouTube and Instagram.

    Vertex Special Note Regarding Forward-Looking Statements  

    This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, statements regarding the expectations and plans to review data presented at the annual ASH meeting and exposition from two ongoing Phase 1/2 clinical trials of the investigational CRISPR/Cas9 gene-editing therapy CTX001 in patients with sickle cell disease and beta thalassemia, and the anticipated speakers participating in the investor webcast. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, the potential for data from a limited number of patients may not to be indicative of final clinical trial results, that data from the company's development programs, including its programs with its collaborators, may not support registration or further development of its compounds due to safety, efficacy or other reasons, and other risks listed under "Risk Factors" in Vertex's most recent annual report and subsequent quarterly reports filed with the Securities and Exchange Commission and available through the company's website at www.vrtx.com. You should not place undue reliance on these statements or the scientific data presented. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

    (VRTX-GEN)

    CRISPR Therapeutics Investor Contact:

    Susan Kim, +1 617-307-7503

    CRISPR Therapeutics Media Contact:

    Rachel Eides

    WCG on behalf of CRISPR

    +1 617-337-4167

    Vertex Pharmaceuticals Incorporated

    Investors:

    Michael Partridge, +1 617-341-6108

    or

    Zach Barber, +1 617-341-6470

    or

    Brenda Eustace, +1 617-341-6187

    Media:



    or

    U.S.: +1 617-341-6992

    or

    Heather Nichols: +1 617-839-3607

    or

    International: +44 20 3204 5275

     



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  18. - The combination therapy is a new treatment option for CF patients who are homozygous for F508del -

    - The only medicine to treat the underlying cause of CF in this age group with one F508del mutation and one of 14 residual function mutations -

    Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today announced that the European Commission has granted approval of the label extension for SYMKEVI® (tezacaftor/ivacaftor) with KALYDECO® (ivacaftor), to include the treatment of cystic fibrosis (CF) in patients ages 6 years and older who have two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or one copy of the F508del mutation and one copy of one of 14 mutations in the CFTR gene that result…

    - The combination therapy is a new treatment option for CF patients who are homozygous for F508del -

    - The only medicine to treat the underlying cause of CF in this age group with one F508del mutation and one of 14 residual function mutations -

    Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today announced that the European Commission has granted approval of the label extension for SYMKEVI® (tezacaftor/ivacaftor) with KALYDECO® (ivacaftor), to include the treatment of cystic fibrosis (CF) in patients ages 6 years and older who have two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or one copy of the F508del mutation and one copy of one of 14 mutations in the CFTR gene that result in residual CFTR activity: P67L, R117C, L206W, R352Q, A455E, D579G, 711+3A→G, S945L, S977F, R1070W, D1152H, 2789+5G→A, 3272-26A→G, and 3849+10kbC→T.

    "With this approval, children with CF in Europe ages 6 to 11 years with the most common mutation, F508del, have a new treatment option and children with certain residual function mutations will, for the first time, have a treatment option available that addresses the underlying cause of their CF," said Reshma Kewalramani, M.D., Chief Executive Officer and President at Vertex. "Today's approval brings us closer to our ultimate goal of providing medicines for all people with CF."

    SYMKEVI® (tezacaftor/ivacaftor) with KALYDECO® (ivacaftor) will be immediately available to additional eligible patients in Germany and will be available shortly in countries that have entered into innovative long-term reimbursement agreements with Vertex, including the UK, Denmark and the Republic of Ireland. In all other countries, Vertex will work closely with relevant authorities in Europe to secure access for eligible patients.

    In Europe, SYMKEVI® (tezacaftor/ivacaftor) with KALYDECO® (ivacaftor) is already approved for the treatment of people with CF ages 12 years and older who have either two copies of the F508del mutation in the CFTR gene, or one copy of the F508del mutation and a copy of one of the following 14 mutations in which the CFTR gene results in residual CFTR protein activity: P67L, R117C, L206W, R352Q, A455E, D579G, 711+3A→G, S945L, S977F, R1070W, D1152H, 2789+5G→A, 3272-26A→G, and 3849+10kbC→T.

    About Cystic Fibrosis

    Cystic Fibrosis (CF) is a rare, life-shortening genetic disease affecting approximately 75,000 people worldwide. CF is a progressive, multi-system disease that affects the lungs, liver, GI tract, sinuses, sweat glands, pancreas and reproductive tract. CF is caused by a defective and/or missing CFTR protein resulting from certain mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF. While there are many different types of CFTR mutations that can cause the disease, the vast majority of all people with CF have at least one F508del mutation. These mutations, which can be determined by a genetic test, or genotyping test, lead to CF by creating non-working and/or too few CFTR proteins at the cell surface. The defective function and/or absence of CFTR protein results in poor flow of salt and water into and out of the cells in a number of organs. In the lungs, this leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage in many patients that eventually leads to death. The median age of death is in the early 30s.

    About SYMKEVI® in combination with KALYDECO®

    Some mutations result in CFTR protein that is not processed or folded normally within the cell, and that generally does not reach the cell surface. Tezacaftor is designed to address the trafficking and processing defect of the CFTR protein to enable it to reach the cell surface and ivacaftor is designed to enhance the function of the CFTR protein once it reaches the cell surface.

    For complete product information including dosing guidance, please see the Summary of Product Characteristics that can be found on www.ema.europa.eu.

    About Vertex

    Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) — a rare, life-threatening genetic disease — and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational small molecule medicines in other serious diseases where it has deep insight into causal human biology, including pain, alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases. In addition, Vertex has a rapidly expanding pipeline of genetic and cell therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.

    Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including 11 consecutive years on Science magazine's Top Employers list and a best place to work for LGBTQ equality by the Human Rights Campaign. For company updates and to learn more about Vertex's history of innovation, visit www.vrtx.com or follow us on Facebook, Twitter, LinkedIn, YouTube and Instagram.

    Special Note Regarding Forward-looking Statements

    This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, statements made by Dr. Reshma Kewalramani in this press release, statements regarding the eligible patient population in Europe, our expectations regarding the timing of access to SYMKEVI in combination with KALYDECO for eligible patients ages 6-11 years across countries in Europe, and our plans to secure access to SYMKEVI in combination with KALYDECO for additional eligible patients ages 6-11 years in Europe. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that data from the company's development programs may not support registration or further development of its compounds due to safety, efficacy or other reasons, risks related to commercializing SYMKEVI in combination with KALYDECO in Europe, and other risks listed under Risk Factors in Vertex's most recent annual report and subsequent quarterly reports filed with the Securities and Exchange Commission and available through the company's website at www.vrtx.com. You should not place undue reliance on these statements. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

    (VRTX-GEN)

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  19. Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today announced that it has been notified of an unsolicited "mini-tender" offer dated November 9, 2020, made by TRC Capital Investment Corporation, an Ontario, Canada, corporation to purchase up to 1,000,000 shares of Vertex common stock. TRC Capital's unsolicited "mini-tender" offer price of $210.00 per share is approximately 4.67% below the $220.28 per share closing price of Vertex's common stock on November 6, 2020, the last trading day prior to the commencement of the offer.

    Vertex does not endorse TRC Capital's offer and recommends that Vertex shareholders reject the offer and not tender their shares in response to TRC Capital's unsolicited offer. The "mini-tender" offer is at a price…

    Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today announced that it has been notified of an unsolicited "mini-tender" offer dated November 9, 2020, made by TRC Capital Investment Corporation, an Ontario, Canada, corporation to purchase up to 1,000,000 shares of Vertex common stock. TRC Capital's unsolicited "mini-tender" offer price of $210.00 per share is approximately 4.67% below the $220.28 per share closing price of Vertex's common stock on November 6, 2020, the last trading day prior to the commencement of the offer.

    Vertex does not endorse TRC Capital's offer and recommends that Vertex shareholders reject the offer and not tender their shares in response to TRC Capital's unsolicited offer. The "mini-tender" offer is at a price below the market price of shares of Vertex's common stock (as of today's date) and is subject to numerous conditions, including TRC Capital's ability to obtain financing necessary to consummate the offer. Vertex is not affiliated in any way with TRC Capital, the offer, or the offer documentation.

    TRC Capital has made many similar "mini-tender" offers for the shares of other companies. "Mini-tender" offers are designed to seek less than five percent of a company's outstanding shares, thereby avoiding many disclosure and procedural requirements of the U.S. Securities and Exchange Commission (SEC) because they are below the SEC's threshold to provide such disclosure and procedural protections for investors.

    The SEC has cautioned investors about "mini-tender" offers in an investor alert. The SEC noted that these offers "have been increasingly used to catch investors off guard" and that many investors who hear about "mini-tender" offers "surrender their securities without investigating the offer, assuming that the price offered includes the premium usually present in larger, traditional tender offers."

    To read more about the risks of "mini-tender" offers, please review the alert on the SEC's website at http://www.sec.gov/investor/pubs/minitend.htm.

    Like TRC Capital's other offers, this "mini-tender" puts individual investors at risk because they may sell their shares at a discount without so realizing. Vertex urges shareholders to obtain current stock quotes for their shares of Vertex common stock, review the terms and conditions to the offer, consult with their broker or financial adviser and exercise caution with respect to TRC Capital's "mini-tender" offer. Shareholders who have already tendered should consider the advisability of withdrawing their shares as permitted under TRC Capital's Offer to Purchase documents.

    Shareholders who have already tendered their shares may withdraw them at any time prior to the expiration of the offer, and if TRC Capital has not accepted a shareholder's shares for payment pursuant to the offer, that shareholder may withdraw them at any time after December 21, 2020, until TRC Capital accepts the shareholder's shares for payment, in accordance with TRC Capital's offering documents.. According to the offer documents, the offer is currently scheduled to expire at 12:01 a.m., New York City time, on December 9, 2020.

    Vertex encourages broker-dealers, as well as other market participants, to review the SEC's letter regarding broker-dealer "mini-tender" offer dissemination and disclosure at https://www.sec.gov/divisions/marketreg/minitenders/sia072401.htm and the NASD Notice to Members regarding guidance to members forwarding "mini-tender" offers to their customers, which can be found at https://www.finra.org/sites/default/files/NoticeDocument/p004221.pdf.

    Vertex requests that a copy of this news release be included with all distributions of materials relating to TRC Capital's "mini-tender" offer related to shares of Vertex's common stock.

    About Vertex

    Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) — a rare, life-threatening genetic disease — and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational small molecule medicines in other serious diseases where it has deep insight into causal human biology, including pain, alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases. In addition, Vertex has a rapidly expanding pipeline of genetic and cell therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.

    Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including 11 consecutive years on Science magazine's Top Employers list and a best place to work for LGBTQ equality by the Human Rights Campaign. For company updates and to learn more about Vertex's history of innovation, visit www.vrtx.com or follow us on Facebook, Twitter, LinkedIn, YouTube and Instagram.

    (VRTX-GEN)

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  20. Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today announced that management will present at the Jefferies Virtual London Health Care Conference on Thursday, November 19, 2020 at 9:05 a.m. ET (2:05 p.m. GMT).

    The audio portion of management's remarks will be available live through Vertex's website, www.vrtx.com in the "Investors" section under the "News and Events" page. A replay of the conference webcast will be archived on the company's website.

    About Vertex

    Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) — a rare, life-threatening genetic…

    Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today announced that management will present at the Jefferies Virtual London Health Care Conference on Thursday, November 19, 2020 at 9:05 a.m. ET (2:05 p.m. GMT).

    The audio portion of management's remarks will be available live through Vertex's website, www.vrtx.com in the "Investors" section under the "News and Events" page. A replay of the conference webcast will be archived on the company's website.

    About Vertex

    Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) — a rare, life-threatening genetic disease — and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational small molecule medicines in other serious diseases where it has deep insight into causal human biology, including pain, alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases. In addition, Vertex has a rapidly expanding pipeline of genetic and cell therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.

    Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including 11 consecutive years on Science magazine's Top Employers list and a best place to work for LGBTQ equality by the Human Rights Campaign. For company updates and to learn more about Vertex's history of innovation, visit www.vrtx.com or follow us on Facebook, Twitter, LinkedIn, YouTube and Instagram. (VRTX-WEB)

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  21. - Approval provides opportunity to treat the underlying cause of cystic fibrosis earlier than ever before in Europe -

    Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today announced that the European Commission has granted approval of the label extension for KALYDECO® (ivacaftor) granules to include the treatment of infants with cystic fibrosis (CF) ages 4 months and older and weighing at least 5 kg who have the R117H mutation or one of the following gating (class III) mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R.

    "Our very first CFTR modulator, KALYDECO, was first approved eight years ago, for certain CF patients ages 6 years and older…

    - Approval provides opportunity to treat the underlying cause of cystic fibrosis earlier than ever before in Europe -

    Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today announced that the European Commission has granted approval of the label extension for KALYDECO® (ivacaftor) granules to include the treatment of infants with cystic fibrosis (CF) ages 4 months and older and weighing at least 5 kg who have the R117H mutation or one of the following gating (class III) mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R.

    "Our very first CFTR modulator, KALYDECO, was first approved eight years ago, for certain CF patients ages 6 years and older. With today's approval, babies as young as 4 months are eligible and we believe early treatment is important in managing CF," said Reshma Kewalramani, M.D., Chief Executive Officer and President, Vertex. "Today's approval is a testament to our commitment to keep going until all people with CF have a treatment option."

    The label update is based on data from a cohort in the 24-week Phase 3 open-label safety study (ARRIVAL) consisting of six children with CF ages four months to less than six months who have eligible gating mutations.

    KALYDECO® (ivacaftor) will be now available to additional eligible patients in Germany and will be available shortly in countries that have entered into innovative long-term reimbursement agreements with Vertex, including the UK, Denmark and the Republic of Ireland. In all other countries, Vertex will work closely with relevant authorities in Europe to secure access for eligible patients.

    KALYDECO® (ivacaftor) is already approved in Europe for people with CF ages 6 months and older weighing at least 5 kg who have one of the following mutations in the CFTR gene: G551D, G1244E, G1349D, G178R, G551S, R117H, S1251N, S1255P, S549N or S549R.

    About Cystic Fibrosis

    Cystic Fibrosis (CF) is a rare, life-shortening genetic disease affecting approximately 75,000 people worldwide. CF is a progressive, multi-system disease that affects the lungs, liver, GI tract, sinuses, sweat glands, pancreas and reproductive tract. CF is caused by a defective and/or missing CFTR protein resulting from certain mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF. While there are many different types of CFTR mutations that can cause the disease, the vast majority of all people with CF have at least one F508del mutation. These mutations, which can be determined by a genetic test, or genotyping test, lead to CF by creating non-working and/or too few CFTR proteins at the cell surface. The defective function and/or absence of CFTR protein results in poor flow of salt and water into and out of the cells in a number of organs. In the lungs, this leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage in many patients that eventually leads to death. The median age of death is in the early 30s.

    About KALYDECO® (ivacaftor)

    Ivacaftor is the first medicine to treat the underlying cause of CF in people with specific mutations in the CFTR gene. Known as a CFTR potentiator, ivacaftor is an oral medicine designed to keep CFTR proteins at the cell surface open longer to improve the transport of salt and water across the cell membrane, which helps hydrate and clear mucus from the airways.

    For complete product information, please see the Summary of Product Characteristics that can be found on www.ema.europa.eu.

    About Vertex

    Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) — a rare, life-threatening genetic disease — and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational small molecule medicines in other serious diseases where it has deep insight into causal human biology, including pain, alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases. In addition, Vertex has a rapidly expanding pipeline of genetic and cell therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.

    Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including 11 consecutive years on Science magazine's Top Employers list and a best place to work for LGBTQ equality by the Human Rights Campaign. For company updates and to learn more about Vertex's history of innovation, visit www.vrtx.com or follow us on Facebook, Twitter, LinkedIn, YouTube and Instagram.

    Special Note Regarding Forward-looking Statements

    This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, statements made by Dr. Reshma Kewalramani in this press release, and statements regarding the eligible patient population in Europe, our expectations regarding the timing of access to KALYDECO for eligible patients four months of age and older across countries in Europe, and our plans to secure access to KALYDECO for additional eligible patients four months of age and older in Europe. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that data from the company's development programs may not support registration or further development of its compounds due to safety, efficacy or other reasons, risks related to commercializing KALYDECO in Europe, and other risks listed under Risk Factors in Vertex's most recent annual report and subsequent quarterly reports filed with the Securities and Exchange Commission and available through the company's website at www.vrtx.com. You should not place undue reliance on these statements. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

    (VRTX-GEN)

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  22. ZUG, Switzerland and CAMBRIDGE, Mass. and BOSTON, Nov. 04, 2020 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (NASDAQ:CRSP) and Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today announced data in seven patients from two ongoing Phase 1/2 clinical trials of the investigational CRISPR/Cas9 gene-editing therapy CTX001 in severe hemoglobinopathies has been accepted for an oral presentation during the Plenary Scientific Session at the annual ASH Meeting and Exposition, which will take place virtually from December 5-8, 2020. Haydar Frangoul, M.D., Medical Director of Pediatric Hematology and Oncology at Sarah Cannon Research Institute, HCA Healthcare's TriStar Centennial Medical Center, will deliver the presentation on behalf of all the authors…

    ZUG, Switzerland and CAMBRIDGE, Mass. and BOSTON, Nov. 04, 2020 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (NASDAQ:CRSP) and Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today announced data in seven patients from two ongoing Phase 1/2 clinical trials of the investigational CRISPR/Cas9 gene-editing therapy CTX001 in severe hemoglobinopathies has been accepted for an oral presentation during the Plenary Scientific Session at the annual ASH Meeting and Exposition, which will take place virtually from December 5-8, 2020. Haydar Frangoul, M.D., Medical Director of Pediatric Hematology and Oncology at Sarah Cannon Research Institute, HCA Healthcare's TriStar Centennial Medical Center, will deliver the presentation on behalf of all the authors on December 6, 2020.

    An abstract posted online today includes data from five patients with three months to 15 months of follow-up after CTX001 infusion in the ongoing Phase 1/2 CLIMB-111 trial in transfusion-dependent beta thalassemia (TDT) and data from two patients with three months and 12 months of follow-up in the ongoing Phase 1/2 CLIMB-121 trial in severe sickle cell disease (SCD). Additional data will be presented at ASH, including longer-duration follow-up data for the patients included in the abstract and data for additional patients with greater than three months of follow-up.

    CTX001 is being investigated in these two ongoing clinical trials as a potential one-time curative therapy for patients suffering from TDT and severe SCD.

    The accepted abstract is now available on the ASH conference website.

    About CTX001

    CTX001 is an investigational, autologous, ex vivo CRISPR/Cas9 gene-edited therapy that is being evaluated for patients suffering from TDT or severe SCD, in which a patient's hematopoietic stem cells are engineered to produce high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is a form of the oxygen-carrying hemoglobin that is naturally present at birth, which then switches to the adult form of hemoglobin. The elevation of HbF by CTX001 has the potential to alleviate transfusion requirements for TDT patients and reduce painful and debilitating sickle crises for SCD patients.

    Based on progress in this program to date, CTX001 has been granted Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the U.S. Food and Drug Administration (FDA). CTX001 has also been granted Orphan Drug Designation from the European Commission for both TDT and SCD, as well as Priority Medicines (PRIME) designation from the European Medicines Agency (EMA) for SCD.

    CTX001 is being developed under a co-development and co-commercialization agreement between CRISPR Therapeutics and Vertex. Among gene-editing approaches being investigated/evaluated for TDT and SCD, CTX001 is the furthest advanced in clinical development.

    About CLIMB-111

    The ongoing Phase 1/2 open-label trial, CLIMB-Thal-111, is designed to assess the safety and efficacy of a single dose of CTX001 in patients ages 12 to 35 with TDT. The trial will enroll up to 45 patients and follow patients for approximately two years after infusion. Each patient will be asked to participate in a long-term follow-up trial.

    About CLIMB-121

    The ongoing Phase 1/2 open-label trial, CLIMB-SCD-121, is designed to assess the safety and efficacy of a single dose of CTX001 in patients ages 12 to 35 with severe SCD. The trial will enroll up to 45 patients and follow patients for approximately two years after infusion. Each patient will be asked to participate in a long-term follow-up trial.

    About the Gene-Editing Process in These Trials

    Patients who enroll in these trials will have their own hematopoietic stem and progenitor cells collected from peripheral blood. The patient's cells will be edited using the CRISPR/Cas9 technology. The edited cells, CTX001, will then be infused back into the patient as part of a stem cell transplant, a process which involves, among other things, a patient being treated with myeloablative busulfan conditioning. Patients undergoing stem cell transplants may also encounter side effects (ranging from mild to severe) that are unrelated to the administration of CTX001. Patients will initially be monitored to determine when the edited cells begin to produce mature blood cells, a process known as engraftment. After engraftment, patients will continue to be monitored to track the impact of CTX001 on multiple measures of disease and for safety.

    About the CRISPR-Vertex Collaboration

    CRISPR Therapeutics and Vertex entered into a strategic research collaboration in 2015 focused on the use of CRISPR/Cas9 to discover and develop potential new treatments aimed at the underlying genetic causes of human disease. CTX001 represents the first potential treatment to emerge from the joint research program. CRISPR Therapeutics and Vertex will jointly develop and commercialize CTX001 and equally share all research and development costs and profits worldwide.

    About CRISPR Therapeutics

    CRISPR Therapeutics is a leading gene editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 platform. CRISPR/Cas9 is a revolutionary gene editing technology that allows for precise, directed changes to genomic DNA. CRISPR Therapeutics has established a portfolio of therapeutic programs across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine and rare diseases. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic collaborations with leading companies including Bayer, Vertex Pharmaceuticals and ViaCyte, Inc. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Cambridge, Massachusetts, and business offices in San Francisco, California and London, United Kingdom. For more information, please visit www.crisprtx.com.

    CRISPR Therapeutics Forward-Looking Statement

    This press release may contain a number of "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, as well as statements regarding CRISPR Therapeutics' expectations about any or all of the following: (i) the status of clinical trials (including, without limitation, the expected timing of data releases) related to product candidates under development by CRISPR Therapeutics and its collaborators, including expectations regarding the data and plans to present data at the annual ASH meeting and exposition; (ii) the expected benefits of CRISPR Therapeutics' collaborations; and (iii) the therapeutic value, development, and commercial potential of CRISPR/Cas9 gene editing technologies and therapies. Without limiting the foregoing, the words "believes," "anticipates," "plans," "expects" and similar expressions are intended to identify forward-looking statements. You are cautioned that forward-looking statements are inherently uncertain. Although CRISPR Therapeutics believes that such statements are based on reasonable assumptions within the bounds of its knowledge of its business and operations, forward-looking statements are neither promises nor guarantees and they are necessarily subject to a high degree of uncertainty and risk. Actual performance and results may differ materially from those projected or suggested in the forward-looking statements due to various risks and uncertainties. These risks and uncertainties include, among others: the potential for initial and preliminary data from any clinical trial and initial data from a limited number of patients (as is the case with CTX001 at this time) not to be indicative of final trial results; the potential that CTX001 clinical trial results may not be favorable; the potential impacts due to the coronavirus pandemic, such as the timing and progress of clinical trials; that future competitive or other market factors may adversely affect the commercial potential for CTX001; uncertainties regarding the intellectual property protection for CRISPR Therapeutics' technology and intellectual property belonging to third parties, and the outcome of proceedings (such as an interference, an opposition or a similar proceeding) involving all or any portion of such intellectual property; and those risks and uncertainties described under the heading "Risk Factors" in CRISPR Therapeutics' most recent annual report on Form 10-K, quarterly report on Form 10-Q, and in any other subsequent filings made by CRISPR Therapeutics with the U.S. Securities and Exchange Commission, which are available on the SEC's website at www.sec.gov. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date they are made. CRISPR Therapeutics disclaims any obligation or undertaking to update or revise any forward-looking statements contained in this press release, other than to the extent required by law.

    CRISPR THERAPEUTICS® word mark and design logo and CTX001™ are trademarks and registered trademarks of CRISPR Therapeutics AG. All other trademarks and registered trademarks are the property of their respective owners.

    About Vertex

    Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) — a rare, life-threatening genetic disease — and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational small molecule medicines in other serious diseases where it has deep insight into causal human biology, including pain, alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases. In addition, Vertex has a rapidly expanding pipeline of genetic and cell therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.

    Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including 11 consecutive years on Science magazine's Top Employers list and a best place to work for LGBTQ equality by the Human Rights Campaign. For company updates and to learn more about Vertex's history of innovation, visit www.vrtx.com or follow us on Facebook, Twitter, LinkedIn, YouTube and Instagram.

    Vertex Special Note Regarding Forward-Looking Statements

    This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, statements regarding the expectations and plans to present data at the annual ASH meeting and exposition, the development, including expected timeline for development, and potential benefits of CTX001, our plans and expectations for our clinical trials and clinical trial sites, and the status of our clinical trials of our product candidates under development by us and our collaborators, including activities at the clinical trial sites and potential outcomes. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that data from the company's development programs, including its programs with its collaborators, may not support registration or further development of its compounds due to safety, efficacy or other reasons, and other risks listed under Risk Factors in Vertex's most recent annual report and subsequent quarterly reports filed with the Securities and Exchange Commission and available through the company's website at www.vrtx.com. You should not place undue reliance on these statements or the scientific data presented. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

    (VRTX-GEN)

    CRISPR Therapeutics Investor Contact:

    Susan Kim, +1 617-307-7503

    CRISPR Therapeutics Media Contact:

    Rachel Eides

    WCG on behalf of CRISPR

    +1 617-337-4167

    Vertex Pharmaceuticals Incorporated

    Investors:

    Michael Partridge, +1 617-341-6108

    or

    Zach Barber, +1 617-341-6470

    or

    Brenda Eustace, +1 617-341-6187

    Media:



    or

    U.S.: +1 617-341-6992

    or

    Heather Nichols: +1 617-839-3607

    or

    International: +44 20 3204 5275

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  23. -Product revenues of $1.54 billion, a 62% increase compared to Q3 2019-

    -Company raises revenue guidance; now expects 2020 product revenues of $6.0 to $6.2 billion-

    Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today reported consolidated financial results for the third quarter ended September 30, 2020 and revised upward its full-year 2020 financial guidance for product revenues.

    "This has been another very strong quarter for Vertex with execution across our CF business and continued earnings and revenue growth," said Reshma Kewalramani, M.D., Chief Executive Officer and President of Vertex. "We are pleased with our progress toward treating all people with cystic fibrosis highlighted by several recent milestones: the early approval and…

    -Product revenues of $1.54 billion, a 62% increase compared to Q3 2019-

    -Company raises revenue guidance; now expects 2020 product revenues of $6.0 to $6.2 billion-

    Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today reported consolidated financial results for the third quarter ended September 30, 2020 and revised upward its full-year 2020 financial guidance for product revenues.

    "This has been another very strong quarter for Vertex with execution across our CF business and continued earnings and revenue growth," said Reshma Kewalramani, M.D., Chief Executive Officer and President of Vertex. "We are pleased with our progress toward treating all people with cystic fibrosis highlighted by several recent milestones: the early approval and encouraging start of the launch of KAFTRIO in the EU, positive TRIKAFTA data in children ages 6-11, and continued expansion of our medicines' labels as with our recent approval of KALYDECO for infants as young as 4 months of age."

    "As we extend our leadership in CF, we are also advancing a broad pipeline of innovative therapies," continued Dr. Kewalramani. "Our R&D strategy contemplates the high-risk nature of drug development and therefore includes a portfolio approach to each of our disease areas of interest. In AATD, while disappointed by the VX-814 outcome, we look forward to the VX-864 Phase 2 proof-of-concept data in the first half of 2021. Our pipeline spans multiple diseases, and multiple important clinical readouts are expected from now through the end of 2021, each of which we expect will hold transformative potential for patients and further growth for Vertex."

    Third-Quarter 2020 Financial Highlights

             

     

    Three Months Ended September 30,

     

    %

             

     

    2020

     

    2019

     

    Change

             

     

    (in millions, except per share amounts)

             

    Product revenues, net

    $

    1,536

     

     

     

    $

    950

     

     

     

    62%

             

    TRIKAFTA/KAFTRIO

    $

    960

     

     

     

    $

     

     

     

     

             

    SYMDEKO/SYMKEVI

    $

    156

     

     

     

    $

    404

     

     

     

     

             

    ORKAMBI

    $

    226

     

     

     

    $

    297

     

     

     

     

             

    KALYDECO

    $

    194

     

     

     

    $

    249

     

     

     

     

             

     

     

     

     

     

     

     

     

     

     

             

    GAAP Operating income

    $

    672

     

     

     

    $

    99

     

     

     

    577%

             

    Non-GAAP Operating income

    $

    854

     

     

     

    $

    403

     

     

     

    112%

             

     

     

     

     

     

     

     

     

     

     

             

    GAAP Net income

    $

    667

     

     

     

    $

    58

     

     

     

    1,060%

             

    Non-GAAP Net income

    $

    697

     

     

     

    $

    322

     

     

     

    117%

             

     

     

     

     

     

     

     

     

     

     

             

    GAAP Net income per share - diluted

    $

    2.53

     

     

     

    $

    0.22

     

     

     

    1,050%

             

    Non-GAAP Net income per share - diluted

    $

    2.64

     

     

     

    $

    1.23

     

     

     

    115%

    Product revenues increased 62% compared to the third quarter of 2019, primarily driven by the uptake of TRIKAFTA in the U.S. and the uptake of our medicines outside the U.S. following the completion of several significant reimbursement agreements.

    GAAP and non-GAAP net income increased compared to the third quarter of 2019, largely driven by strong growth in total product revenues.

    Cash, cash equivalents and marketable securities as of September 30, 2020 were $6.2 billion, an increase of approximately $2.3 billion compared to $3.8 billion as of December 31, 2019 driven by strong revenue and profitability.

    Third-Quarter 2020 Expenses

             

     

    Three Months Ended September 30,

             

     

    2020

     

    2019

             

     

    (in millions)

             

    Combined GAAP R&D and SG&A expenses

    $

    678

     

     

     

    $

    716

     

     

             

    Combined Non-GAAP R&D and SG&A expenses

    $

    497

     

     

     

    $

    416

     

     

             

     

     

     

     

     

     

     

     

             

    GAAP R&D expense

    $

    493

     

     

     

    $

    556

     

     

             

    Non-GAAP R&D expense

    $

    350

     

     

     

    $

    290

     

     

             

     

     

     

     

     

     

     

     

             

    GAAP SG&A expense

    $

    185