VRTX Vertex Pharmaceuticals Incorporated

216.35
-0.74  -0%
Previous Close 217.09
Open 215.62
52 Week Low 202.565
52 Week High 306.08
Market Cap $56,005,587,921
Shares 258,865,671
Float 258,720,629
Enterprise Value $50,171,977,517
Volume 932,971
Av. Daily Volume 1,634,923
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Upcoming Catalysts

Drug Stage Catalyst Date
TRIKAFTA (elexacaftor/tezacaftor/ivacaftor and ivacaftor)
Children (6-11 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
PDUFA priority review
PDUFA priority review
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CTX001
Beta-thalassemia
Phase 1/2
Phase 1/2
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CTX001
Sickle cell disease
Phase 1/2
Phase 1/2
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VX-864
Alpha-1 antitrypsin (AAT) deficiency
Phase 2
Phase 2
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VX-147
Focal segmental glomerulosclerosis (FSGS)
Phase 2
Phase 2
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Drug Pipeline

Drug Stage Notes
VX-880
Type 1 Diabetes
Phase 1/2
Phase 1/2
Phase 1/2 trial initiation announced March 10, 2021.
Elexacaftor, tezacaftor and ivacaftor
Cystic fibrosis
Approved
Approved
FDA approval announced December 21, 2020.
VX-814
alpha-1 antitrypsin (AAT) deficiency
Phase 2
Phase 2
Phase 2 trial and development to be discontinued due to safety.
VX-445 in combination with tezacaftor and ivacaftor
Cystic fibrosis (CF) who have one copy of the F508del mutation and one minimal function mutation and patients with two copies of the F508del mutation
Approved
Approved
FDA Approval announced October 21, 2019.
Tezacaftor / ivacaftor
Cystic fibrosis - Two Copies of the F508del Mutation
Approved
Approved
Label expanded to now include all patients over 6 years old - June 21, 2019.
Ivacaftor
Cystic Fibrosis (6-12 mths)
Approved
Approved
FDA Approval announced April 30, 2019.
Lumacaftor and ivacaftor
Cystic fibrosis (CF) ages 1-2.
Approved
Approved
Approval announced August 15, 2018.
Ivacaftor
Cystic fibrosis (CF) ages 2 to 5.
Approved
Approved
FDA Approval announced August 7, 2018.
ORKAMBI
Cystic fibrosis (CF) ages 6-11 who have F508del mutation
Approved
Approved
Approved September 28, 2016.
Tezacaftor (VX-661) / ivacaftor
Cystic fibrosis - one copy of the F508del mutation and a second mutation that results in a gating mutation
Phase 3
Phase 3
Phase 3 data released October 25, 2017 - primary endpoint not met.
VX-661
Cystic fibrosis - one copy of the F508del mutation and a second mutation that results in minimal CFTR Function
Phase 3
Phase 3
Phase 3 trial terminated August 2016
KALYDECO (ivacaftor)
Children ages 2 to 5 with cystic fibrosis who have the G551D or one of the eight additional gating mutations
Approved
Approved
Approved March 17, 2015.
Lumacaftor and ivacaftor
Cystic fibrosis (CF) ages 12 and older who have two copies of the F508del mutation
Approved
Approved
Approved July 2, 2015.
KALYDECO (ivacaftor)
Cystic fibrosis (CF) ages 2 and older who have one of 23 residual function mutations.
CRL
CRL
CRL issued February 5, 2016.
KALYDECO
Cystic fibrosis (CF) ages 6 and older who have the R117H mutation
Approved
Approved
Approved December 29, 2014.

Latest News

  1. Concert Pharmaceuticals, Inc. (NASDAQ:CNCE) today announced that Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) has purchased the potential future milestones under the companies' 2017 asset purchase agreement relating to VX-561 (deutivacaftor) for $32 million.

    "This transaction provided an opportunity to secure non-dilutive capital and strengthens our balance sheet as we continue to advance CTP-543, our lead asset for alopecia areata, through its Phase 3 program," stated Roger Tung, Ph.D., President and Chief Executive Officer of Concert Pharmaceuticals. "By receiving these proceeds, we now expect our cash, cash equivalents and investments to fund the Company into the second quarter of 2022."

    Under the asset purchase agreement, Vertex…

    Concert Pharmaceuticals, Inc. (NASDAQ:CNCE) today announced that Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) has purchased the potential future milestones under the companies' 2017 asset purchase agreement relating to VX-561 (deutivacaftor) for $32 million.

    "This transaction provided an opportunity to secure non-dilutive capital and strengthens our balance sheet as we continue to advance CTP-543, our lead asset for alopecia areata, through its Phase 3 program," stated Roger Tung, Ph.D., President and Chief Executive Officer of Concert Pharmaceuticals. "By receiving these proceeds, we now expect our cash, cash equivalents and investments to fund the Company into the second quarter of 2022."

    Under the asset purchase agreement, Vertex acquired worldwide development and commercialization rights to VX-561 (formerly known as CTP-656) and other assets related to the treatment of cystic fibrosis. VX-561 is an investigational cystic fibrosis transmembrane conductance regulator (CFTR) potentiator that has the potential to be used as part of future once-daily combination regimens of CFTR modulators that treat the underlying cause of cystic fibrosis. In 2017, Concert received a one-time cash payment of $160 million upon closing the asset purchase, with the potential for $90 million in future milestones. Following receipt of the $32 million, no further milestone obligations remain.

    Chestnut Securities, Inc. acted as exclusive financial advisor to Concert in this transaction.

    About Concert

    Concert Pharmaceuticals is a clinical stage biopharmaceutical company that is developing small molecule drugs that it discovered through the application of its DCE Platform® (deuterated chemical entity platform). Selective incorporation of deuterium into known molecules has the potential, on a case-by-case basis, to provide better pharmacokinetic or metabolic properties, thereby enhancing their clinical safety, tolerability or efficacy. Concert's lead product candidate is in late-stage development for the treatment of alopecia areata, a serious autoimmune dermatological condition. Concert is also assessing a number of earlier-stage pipeline candidates. For more information please visit www.concertpharma.com or follow us on Twitter at @ConcertPharma or on LinkedIn.

    Cautionary Note on Forward Looking Statements

    Any statements in this press release about our future expectations, plans and prospects, including, among others, statements about our expectations regarding the sufficiency of our cash, cash equivalents and investments to fund our operations, and any other statements containing the words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would" and similar expressions, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: the uncertainties inherent in the initiation, timing and design of future clinical trials, the availability and timing of data from ongoing and future clinical trials and the results of such trials, whether preliminary results from a clinical trial will be predictive of the final results of that trial or whether results of early clinical trials will be indicative of the results of later clinical trials, expectations for regulatory approvals, availability of funding sufficient for our foreseeable and unforeseeable operating expenses and capital expenditure requirements, expectations with respect to the protection of our intellectual property afforded by our patents and other factors discussed in the "Risk Factors" section of our most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission and in other filings that we make with the Securities and Exchange Commission. In addition, any forward-looking statements included in this press release represent our views only as of the date of this release and should not be relied upon as representing our views as of any subsequent date. We specifically disclaim any obligation to update any forward-looking statements included in this press release.

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  2. Patrik De Haes M.D. becomes Non-executive Chairman

    Leuven, BE and Boston, MA, US May 17, 20217:30 AM CETOxurion NV (Euronext Brussels: OXUR), a biopharmaceutical company developing next generation standard of care ophthalmic therapies, with a clinical stage portfolio in retinal vascular disorders, announces in a planned succession, the promotion of Tom Graney, CFA from Chief Financial Officer (CFO) to Chief Executive Officer (CEO) of the company. He takes over from Dr. Patrik De Haes, who after 14 years as CEO, has decided to move away from day-to-day management of the company to become the Non-executive Chairman of Oxurion. Thomas Clay, the outgoing Chairman of Oxurion, will remain on the Board as a Non-executive Director of the company…

    Patrik De Haes M.D. becomes Non-executive Chairman

    Leuven, BE and Boston, MA, US May 17, 20217:30 AM CETOxurion NV (Euronext Brussels: OXUR), a biopharmaceutical company developing next generation standard of care ophthalmic therapies, with a clinical stage portfolio in retinal vascular disorders, announces in a planned succession, the promotion of Tom Graney, CFA from Chief Financial Officer (CFO) to Chief Executive Officer (CEO) of the company. He takes over from Dr. Patrik De Haes, who after 14 years as CEO, has decided to move away from day-to-day management of the company to become the Non-executive Chairman of Oxurion. Thomas Clay, the outgoing Chairman of Oxurion, will remain on the Board as a Non-executive Director of the company.

    Mr. Graney has extensive capital markets and business development experience in biotech as the CFO of several successful public companies. This includes having raised >$500M in capital for highly innovative companies like Oxurion and completing a number of value creating business development transactions. Tom also has an impressive track record of developing high performing teams and talent everywhere he has worked both in the United States and Europe.

    Tom's transatlantic experience in creating significant company and shareholder value makes him uniquely qualified to lead Oxurion as its next CEO as it moves its highly differentiated assets through clinical development.

    Tom joined Oxurion as CFO in October 2020, and has over 25 years' experience in senior finance, strategy, business development and operational roles including most recently as CFO of Generation Bio (NASDAQ:GBIO), and before that CFO of Vertex Pharmaceuticals (NASDAQ:VRTX), and CFO and Head of Corporate Strategy at Ironwood Pharmaceuticals (NASDAQ:IRWD). Earlier in his career he spent 20 years with Johnson and Johnson, including CFO roles at Ethicon, Tibotec and Janssen. Tom also serves on the board of AC Immune (NASDAQ:ACIU).

    Tom has an MBA in finance, marketing and international business from the Leonard N. Stern School of Business at New York University and a BS in accounting from the University of Delaware. He is also a CFA charterholder.

    Tom Graney, the incoming CEO of Oxurion, said, "I am very excited and honored to become the CEO of Oxurion as we build out our plan to create an important ophthalmic company grounded in exceptional science. I am confident that with THR-149 and THR-687, two highly promising and differentiated new drug candidates, coupled with our extremely talented team, Oxurion has the potential to successfully address a broad range of retinal vascular disorders. This would allow us to generate significant value for patients, payors and shareholders as we look to disrupt and grow the $12 billion market for retinal vascular disorders."

    Patrik De Haes M.D., the incoming Chairman of Oxurion, said, "I am looking forward to supporting Tom and the rest of the Oxurion team in my new role as Chairman as we deliver a number of important clinical development milestones in the months ahead. I am confident that our drug candidates that have the potential to make a significant difference to the lives of the large number of patients with sight threatening disease, who require better treatment options." 

    Thomas Clay, outgoing Chairman of Oxurion, commented, "I would like to thank Patrik for his high level of commitment and leadership to Oxurion over the past 14 years and his desire to continue as Chairman of the company. Oxurion is well placed for future success given its industry-leading pipeline of next generation standard of care ophthalmic therapies and a highly experienced team. I look forward to working with Tom, Patrik and the rest of the board as a Non-executive Director of Oxurion as we look to realize a significant opportunity to bring value to patients and shareholders as we progress the clinical development of THR-149 and THR-687 in diabetic macular edema and other important retinal vascular disorders."

    END

    For further information please contact:

    Oxurion NV

    Wouter Piepers,

    Global Head of Investor Relations

    & Corporate Communications

    Tel: +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 with retinal vascular disorders including diabetic macular edema (DME), the leading cause of vision loss in diabetic patients worldwide as well as other conditions, including wet age-related macular degeneration (AMD) and retinal vein occlusion (RVO).

    Oxurion is aiming to build the leading global franchise in the treatment of retinal vascular disorders 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 the 40% of DME patients who respond suboptimally 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 suboptimally to anti-VEGF therapy.



    • THR-687 is a pan-RGD integrin antagonist that is initially being developed as a potential first line therapy for DME patients. Positive topline results in a Phase 1 clinical study assessing THR-687 as a treatment for DME were announced in 2020. THR-687 is expected to enter a Phase 2 clinical trial in mid-2021. THR-687, also has the potential to deliver improved treatment outcomes for patients with wet AMD and RVO.



    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.



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  3. CAMBRIDGE, Mass. and ZUG, Switzerland and BOSTON, May 12, 2021 (GLOBE NEWSWIRE) -- Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) and CRISPR Therapeutics (NASDAQ:CRSP) today announced two abstracts detailing updated data from the ongoing CTX001 clinical trials have been accepted for presentation during the European Hematology Association (EHA) 2021 Virtual Congress.

    Abstract #EP736 entitled "CTX001 for Sickle Cell Disease: Safety and Efficacy Results from the Ongoing CLIMB SCD-121 Study of Autologous Crispr-Cas9-Modified CD34+ Hematopoietic Stem and Progenitor Cells," will be made available on the virtual platform as an e-poster Friday, June 11 at 9:00 CEST. The abstract posted online today includes data on patients with severe sickle…

    CAMBRIDGE, Mass. and ZUG, Switzerland and BOSTON, May 12, 2021 (GLOBE NEWSWIRE) -- Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) and CRISPR Therapeutics (NASDAQ:CRSP) today announced two abstracts detailing updated data from the ongoing CTX001 clinical trials have been accepted for presentation during the European Hematology Association (EHA) 2021 Virtual Congress.

    Abstract #EP736 entitled "CTX001 for Sickle Cell Disease: Safety and Efficacy Results from the Ongoing CLIMB SCD-121 Study of Autologous Crispr-Cas9-Modified CD34+ Hematopoietic Stem and Progenitor Cells," will be made available on the virtual platform as an e-poster Friday, June 11 at 9:00 CEST. The abstract posted online today includes data on patients with severe sickle cell disease with more than 3 months of follow-up, as of the interim data cut on January 28, 2021. Data will be updated and information on additional patients will be included for the congress.

    Abstract #EP733 entitled "CTX001 for Transfusion-Dependent Β-Thalassemia: Safety and Efficacy Results from the Ongoing CLIMB Thal-111 Study of Autologous Crispr-Cas9-Modified CD34+ Hematopoietic Stem and Progenitor Cells," will be made available on the virtual platform as an e-poster Friday, June 11 at 9:00 CEST. The abstract posted online today includes data on patients with transfusion-dependent beta thalassemia (TDT) with more than 3 months of follow-up, including patients with the most severe genotypes, as of the interim data cut on January 21, 2021. Data will be updated and information on additional patients will be included for the congress.

    The accepted abstracts are now available online on the EHA website https://library.ehaweb.org/eha/#!*menu=6*browseby=8*sortby=2*media=3*ce_id=2035*label=21989*ot_id=25562*marker=1286.

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

    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. Earlier results from these ongoing trials were published as a Brief Report in The New England Journal of Medicine in January of 2021.

    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, as well as Priority Medicines (PRIME) designation from the European Medicines Agency (EMA), for both TDT and SCD.

    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 CLIMB-131

    This is a long-term, open-label trial to evaluate the safety and efficacy of CTX001 in patients who received CTX001 in CLIMB-111 or CLIMB-121. The trial is designed to follow participants for up to 15 years after CTX001 infusion.

    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 Vertex-CRISPR Collaboration

    Vertex and CRISPR Therapeutics 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. Under a recently amended collaboration agreement, Vertex will lead global development, manufacturing and commercialization of CTX001 and split program costs and profits worldwide 60/40 with CRISPR Therapeutics. This amendment is subject to customary closing conditions and clearances, including clearance under the Hart-Scott Rodino Antitrust Improvements Act.

    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.

    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, our plans and expectations to present clinical data from the ongoing CTX001 clinical trials during the EHA Virtual Congress, expectations regarding the abstracts that will be made available on the virtual platform, the expectation that data will be updated for the conference, the potential benefits of CTX001, our plans and expectations for our clinical trials and pipeline products, 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 patient enrollment, and our expectations regarding the transaction contemplated by the amended collaboration agreement with CRISPR, including satisfaction of closing conditions and antitrust clearances, and the future activities of the parties pursuant to the amended collaboration agreement. 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 and/or 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, or other reasons, 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 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)

    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 safety, efficacy and clinical progress of CRISPR Therapeutics' various clinical programs, including CTX001, including expectations regarding the abstracts that will be made available on the virtual platform and the clinical data that are being presented from the ongoing CTX001 clinical trials during the EHA Virtual Congress; (ii) the timing of the potential closing of the transaction contemplated by the amended collaboration agreement, future activities of the parties pursuant to the collaboration and the potential benefits of CRISPR Therapeutics' collaboration with Vertex; 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, 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: 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 or future trial results; the potential that CTX001 clinical trial results may not be favorable or may not support registration or further development; that future competitive or other market factors may adversely affect the commercial potential for CTX001; the transaction contemplated by the amended collaboration agreement is subject to certain closing conditions, including the expiration of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act; CRISPR Therapeutics may not realize the potential benefits of the collaboration with Vertex; potential impacts due to the coronavirus pandemic, such as to the timing and progress of clinical trials; the potential 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 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.

    Vertex Pharmaceuticals Incorporated

    Investors:

    Michael Partridge, +1 617-341-6108

    or

    Brenda Eustace, +1 617-341-6187

    Or

    Manisha Pai, +1 617-429-6891

    Media:



    or

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

    or

    Heather Nichols: +1 617-839-3607

    or

    International: +44 20 3204 5275

    CRISPR Therapeutics

    Investors:

    Susan Kim, +1 617-307-7503

    Media:

    Rachel Eides, +1-617-315-4493



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  4. Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today announced that management will present at the Bank of America Securities 2021 Virtual Health Care Conference on Thursday, May 13, 2021 at 10:15 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…

    Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today announced that management will present at the Bank of America Securities 2021 Virtual Health Care Conference on Thursday, May 13, 2021 at 10:15 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)

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  5. -Product revenues of $1.72 billion, a 14% increase compared to Q1 2020-

    - Company advancing clinical programs in six additional diseases beyond cystic fibrosis-

    -Multiple Phase 2 proof-of-concept study results expected in 2021 -

    Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today reported consolidated financial results for the first quarter ended March 31, 2021 and reiterated full-year 2021 guidance for product revenues.

    "In CF, our goal is that all eligible patients have access to and can benefit from CFTR modulators. In the first quarter, we continued to make significant progress towards this goal, and in so doing again delivered strong revenue and earnings growth," said Reshma Kewalramani, M.D., Chief Executive Officer and President…

    -Product revenues of $1.72 billion, a 14% increase compared to Q1 2020-

    - Company advancing clinical programs in six additional diseases beyond cystic fibrosis-

    -Multiple Phase 2 proof-of-concept study results expected in 2021 -

    Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today reported consolidated financial results for the first quarter ended March 31, 2021 and reiterated full-year 2021 guidance for product revenues.

    "In CF, our goal is that all eligible patients have access to and can benefit from CFTR modulators. In the first quarter, we continued to make significant progress towards this goal, and in so doing again delivered strong revenue and earnings growth," said Reshma Kewalramani, M.D., Chief Executive Officer and President of Vertex. "Beyond CF, we have also seen continued significant progress across our broad pipeline, including advancement of VX-548 to Phase 2 in acute pain, initiation of the Phase 1/2 clinical trial with VX-880 in type 1 diabetes and completion of enrollment and dosing in our Phase 2 proof-of-concept study with the AAT corrector, VX-864. The recent amendment of our agreement with CRISPR Therapeutics for the CTX001 program further enhances our leadership position in cell and genetic therapies and we look forward to completing enrollment of our ongoing trials for CTX001 in sickle cell disease and beta thalassemia this year and bringing this first-in-class treatment to patients with these devastating diseases as soon as possible."

    First-Quarter 2021 Financial Highlights

     

    Three Months Ended March 31,

     

    %

     

    2021

     

    2020

     

    Change

     

    (in millions, except per share amounts)

    Product revenues, net

    $

    1,723

     

     

     

    $

    1,515

     

     

     

    14

    %

    TRIKAFTA/KAFTRIO

    $

    1,193

     

     

     

    $

    895

     

     

     

     

    SYMDEKO/SYMKEVI

    $

    125

     

     

     

    $

    173

     

     

     

     

    ORKAMBI

    $

    219

     

     

     

    $

    234

     

     

     

     

    KALYDECO

    $

    186

     

     

     

    $

    213

     

     

     

     

     

     

     

     

     

     

     

     

     

     

    GAAP Operating income

    $

    888

     

     

     

    $

    720

     

     

     

    23

    %

    Non-GAAP Operating income

    $

    1,003

     

     

     

    $

    877

     

     

     

    14

    %

     

     

     

     

     

     

     

     

     

     

    GAAP Net income

    $

    653

     

     

     

    $

    603

     

     

     

    8

    %

    Non-GAAP Net income

    $

    781

     

     

     

    $

    674

     

     

     

    16

    %

     

     

     

     

     

     

     

     

     

     

    GAAP Net income per share - diluted

    $

    2.49

     

     

     

    $

    2.29

     

     

     

    9

    %

    Non-GAAP Net income per share - diluted

    $

    2.98

     

     

     

    $

    2.56

     

     

     

    16

    %

    Product revenues increased 14% compared to the first quarter of 2020, primarily driven by the uptake of KAFTRIO in Europe and continued performance of TRIKAFTA in the U.S. Net product revenues in the first quarter of 2021 increased 6% to $1.25 billion in the U.S. and increased 43% to $470 million outside the U.S., compared to the prior year.

    GAAP and non-GAAP net income increased compared to the first quarter of 2020, largely driven by strong growth in total product revenues.

    Cash, cash equivalents and marketable securities as of March 31, 2021 were $6.9 billion, an increase of $265 million compared to $6.7 billion as of December 31, 2020 primarily driven by strong revenue and profitability offset by the repurchase of our common stock authorized under our stock repurchase plan.

    First-Quarter 2021 Expenses

     

    Three Months Ended March 31,

     

    2021

     

    2020

     

    (in millions)

    Combined GAAP R&D and SG&A expenses

    $

    648

     

     

    $

    631

     

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

    $

    530

     

     

    $

    477

     

     

     

     

     

     

     

     

     

    GAAP R&D expenses

    $

    456

     

     

    $

    449

     

    Non-GAAP R&D expenses

    $

    379

     

     

    $

    337

     

     

     

     

     

     

     

     

     

    GAAP SG&A expenses

    $

    192

     

     

    $

    182

     

    Non-GAAP SG&A expenses

    $

    151

     

     

    $

    140

     

     

     

     

     

     

     

     

     

    GAAP income taxes (1)

    $

    168

     

     

    $

    55

     

    Non-GAAP income taxes

    $

    207

     

     

    $

    184

     

     

     

     

     

     

     

     

     

    GAAP effective tax rate

     

    20

    %

    8

    %

    Non-GAAP effective tax rate (1)

     

    21

    %

     

     

    21

    %

     

     

     

     

     

     

     

     

    Combined GAAP and Non-GAAP R&D and SG&A expenses increased compared to the first quarter of 2020, primarily due to the expansion of Vertex's pipeline in CF and other disease areas and incremental investment to support the global launches of Vertex's medicines.

    GAAP income taxes and the GAAP effective tax rate increased compared to the first quarter of 2020 due a non-recurring discrete tax benefit recognized in the first quarter of 2020 and Vertex's increased operating income.

    Non-GAAP income taxes increased compared to the first quarter of 2020 primarily due to Vertex's increased operating income.

    Full-Year 2021 Financial Guidance

    Vertex today reiterated its full-year 2021 financial guidance, except for its expectations for combined GAAP R&D and SG&A expenses, which increased by $900 million as a result of Vertex's amended collaboration with CRISPR announced in April. Vertex's guidance is summarized below:

     

    Current FY 2021

     

    Previous FY 2021

     

     

     

     

    Product revenues

    Unchanged

     

    $6.7 to 6.9 billion

     

     

     

     

    Combined GAAP R&D and SG&A expenses (2)

    $3.8 to 3.95 billion

     

    $2.9 to 3.05 billion

    Combined Non-GAAP R&D and SG&A expenses (2)

    Unchanged

     

    $2.25 to 2.3 billion

    Non-GAAP effective tax rate

    Unchanged

     

    21% to 22%

    Key Business Highlights

    Cystic Fibrosis (CF)

    Vertex anticipates that achieving new approvals and entering into additional reimbursement agreements for our current CFTR modulators will increase the number of CF patients treated with our medicines and continue to grow our CF business in the years ahead.

    Key progress in 2021 includes:

    • New approval received for TRIKAFTA (elexacaftor/tezacaftor/ivacaftor and ivacaftor) in Australia for people with CF ages 12 years and older who have at least one F508del mutation.
    • Post-marketing application filed with the European Medicines Agency (EMA) for the expanded indication of KAFTRIO (elexacaftor/tezacaftor/ivacaftor and ivacaftor) to include children with CF ages 6 through 11 years.
    • TRIKAFTA/KAFTRIO is now approved and reimbursed or accessible in 12 countries outside the U.S., including Denmark, Germany, Ireland, Israel, Switzerland and the countries within the UK.

    R&D pipeline

    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

    • In April, Vertex and CRISPR Therapeutics amended their collaboration for the CTX001 programs in beta thalassemia and sickle cell disease. Under the terms of the revised agreement, Vertex will lead worldwide development, manufacturing and commercialization of CTX001. The revised agreement provides Vertex with 60% and CRISPR Therapeutics with 40% of program economics. At closing, CRISPR Therapeutics will receive a $900 million upfront payment with the potential for an additional $200 million milestone payment upon CTX001 regulatory approval.
    • The CTX001 program employs a non-viral ex vivo CRISPR gene-editing 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 the diseases.
    • Enrollment and dosing are ongoing in the clinical studies for CTX001 and more than 30 patients have now been dosed to date. Completion of enrollment in both studies is expected in 2021.
    • In April, the European Medicines Agency (EMA) granted Priority Medicines Designation (PRIME) to CTX001 for TDT. The program has previously 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 PRIME designation for SCD and Orphan Drug Designation from EMA for both TDT and SCD.

    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 systemic levels of functional AAT. Misfolded Z-AAT protein is the root cause of AAT deficiency and the Vertex small molecule corrector program targets both the liver and lung manifestations of the disease.
    • Patients enrolled in the Phase 2 proof-of-concept study for the Z-AAT corrector, VX-864, have completed the 28-day dosing period. The study includes a 28-day safety follow-up period which is ongoing, and results are expected in the second quarter of 2021.

    APOL1-mediated Kidney Diseases

    • Vertex is evaluating the potential of inhibitors of APOL1 function in people with APOL1-mediated 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. Results from this study are expected in the second half of 2021.

    Type 1 Diabetes (T1D)

    • Vertex is evaluating a cell therapy designed to replace insulin-producing islet cells in people with T1D. Vertex is pursuing two programs for the transplant of these fully-differentiated 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.
    • In March, the U.S. FDA granted Fast Track Designation and Vertex initiated a Phase 1/2 clinical trial for VX-880, the islet cells alone program, in people with T1D.

    Pain

    • Vertex is evaluating selective small molecule inhibitors of NaV1.8, a genetically validated, novel target for the treatment of pain, with the goal of preventing pain signals traveling from the sensory nerves to the central nervous system. Vertex has previously demonstrated clinical proof-of-concept with a small molecule investigational treatment targeting NaV1.8, VX-150, in multiple pain indications including acute pain, neuropathic pain and musculoskeletal pain.
    • VX-548, a selective NaV1.8 inhibitor, demonstrated favorable safety, tolerability and pharmacokinetic profiles in Phase 1 studies. In these studies, the molecule exhibited a favorable profile at doses considerably lower than those required with our previous NaV1.8 inhibitors.
    • VX-548 is expected to advance into Phase 2 proof-of-concept studies for acute pain in the second half of 2021.

    Investments in External Innovation

    • In April, we entered into a research collaboration with Obsidian Therapeutics, Inc., or Obsidian, aimed at the discovery of novel therapies that regulate gene-editing for the treatment of serious diseases. This collaboration enables us to leverage Obsidian's cytoDRiVE® platform technology to discover gene-editing medicines whose therapeutic activity can be precisely controlled using small molecules.

    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) revenues and expenses related to collaborative milestones and upfront payments, (iii) gains or losses related to the fair value of the company's strategic investments, (iv) increases or decreases in the fair value of contingent consideration, (v) acquisition-related costs and (vi) 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 guidance regarding its 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

    First-Quarter Results

    Consolidated Statements of Operations

    (in thousands, except per share amounts)

    (unaudited)

     

     

    Three Months Ended March 31,

     

    2021

     

    2020

    Revenues:

     

     

     

    Product revenues, net

    $

    1,723,305

     

     

    $

    1,515,107

     

    Other revenues

    1,000

     

     

     

    Total revenues

    1,724,305

     

     

    1,515,107

     

    Costs and expenses:

     

     

     

    Cost of sales

    192,329

     

     

    162,497

     

    Research and development expenses

    455,973

     

     

    448,528

     

    Sales, general and administrative expenses

    192,077

     

     

    182,258

     

    Change in fair value of contingent consideration

    (3,900

    )

     

    1,600

     

    Total costs and expenses

    836,479

     

     

    794,883

     

    Income from operations

    887,826

     

     

    720,224

     

    Interest income

    1,465

     

     

    12,576

     

    Interest expense

    (15,678

    )

     

    (14,136

    )

    Other expense, net (3)

    (52,653

    )

     

    (61,130

    )

    Income before provision for income taxes

    820,960

     

     

    657,534

     

    Provision for income taxes

    167,822

     

     

    54,781

     

    Net income

    $

    653,138

     

     

    $

    602,753

     

     

     

     

     

    Net income per common share:

     

     

     

    Basic

    $

    2.52

     

     

    $

    2.32

     

    Diluted

    $

    2.49

     

     

    $

    2.29

     

    Shares used in per share calculations:

     

     

     

    Basic

    259,369

     

     

    259,815

     

    Diluted

    261,916

     

     

    263,515

     

     

    Reconciliation of GAAP to Non-GAAP Net Income

    First-Quarter Results

    (in thousands, except per share amounts)

    (unaudited)

     

     

    Three Months Ended March 31,

     

    2021

     

    2020

    GAAP net income

    $

    653,138

     

     

    $

    602,753

     

    Stock-based compensation expense

    115,174

     

     

    115,706

     

    Decrease in fair value of strategic investments (3)

    52,295

     

     

    44,870

     

    (Decrease) increase in fair value of contingent consideration (4)

    (3,900

    )

     

    1,600

     

    Collaborative revenues and expenses (5)

    650

     

     

    36,250

     

    Acquisition-related costs (6)

    2,820

     

     

    2,883

     

    Total non-GAAP adjustments to pre-tax income

    167,039

     

     

    201,309

     

    Tax adjustments (1)

    (38,961

    )

     

    (129,608

    )

    Non-GAAP net income

    $

    781,216

     

     

    $

    674,454

     

     

     

     

     

    Net income per diluted common share:

     

     

     

    GAAP

    $

    2.49

     

     

    $

    2.29

     

    Non-GAAP

    $

    2.98

     

     

    $

    2.56

     

    Shares used in diluted per share calculations:

     

     

     

    GAAP and Non-GAAP

    261,916

     

     

    263,515

     

     

     

     

     

     

     

     

     

     

     

     

     

     

     

     

     

     

     

     

     

     

     

     

     

     

    Three Months Ended March 31,

     

    2021

     

    2020

    GAAP operating income

    $

    887,826

     

     

    $

    720,224

     

    Stock-based compensation expense

    115,174

     

     

    115,706

     

    (Decrease) increase in fair value of contingent consideration (4)

    (3,900

    )

     

    1,600

     

    Collaborative revenues and expenses (5)

    650

     

     

    36,250

     

    Acquisition-related costs (6)

    2,820

     

     

    2,883

     

    Non-GAAP operating income

    $

    1,002,570

     

     

    $

    876,663

     

     

    Reconciliation of GAAP to Non-GAAP Revenues and Expenses

    First-Quarter Results

    (in thousands)

    (unaudited)

     

     

    Three Months Ended March 31,

     

    2021

     

    2020

    GAAP total revenues

    $

    1,724,305

     

     

    $

    1,515,107

     

    Collaborative revenues

    (1,000

    )

     

     

    Non-GAAP total revenues

    $

    1,723,305

     

     

    $

    1,515,107

     

     

     

     

     

     

    Three Months Ended March 31,

     

    2021

     

    2020

    GAAP cost of sales

    $

    192,329

     

     

    $

    162,497

     

    Stock-based compensation expense

    (1,431

    )

     

    (1,361

    )

    Non-GAAP cost of sales

    $

    190,898

     

     

    $

    161,136

     

     

     

     

     

    GAAP research and development expenses

    $

    455,973

     

     

    $

    448,528

     

    Stock-based compensation expense

    (72,802

    )

     

    (72,687

    )

    Collaborative expenses (5)

    (1,650

    )

     

    (36,250

    )

    Acquisition-related costs (6)

    (2,820

    )

     

    (2,678

    )

    Non-GAAP research and development expenses

    $

    378,701

     

     

    $

    336,913

     

     

     

     

     

    GAAP sales, general and administrative expenses

    $

    192,077

     

     

    $

    182,258

     

    Stock-based compensation expense

    (40,941

    )

     

    (41,658

    )

    Acquisition-related costs (6)

     

     

    (205

    )

    Non-GAAP sales, general and administrative expenses

    $

    151,136

     

     

    $

    140,395

     

     

     

     

     

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

    $

    529,837

     

     

    $

    477,308

     

     

     

     

     

     

    Three Months Ended March 31,

     

    2021

     

    2020

    GAAP other expense, net

    $

    (52,653

    )

     

    $

    (61,130

    )

    Decrease in fair value of strategic investments (3)

    52,295

     

     

    44,870

     

    Non-GAAP other expense, net

    $

    (358

    )

     

    $

    (16,260

    )

     

     

     

     

    GAAP provision for income taxes

    $

    167,822

     

     

    $

    54,781

     

    Tax adjustments (1)

    38,961

     

     

    129,608

     

    Non-GAAP provision for income taxes (7)

    $

    206,783

     

     

    $

    184,389

     

    GAAP effective tax rate

    20

    %

    8

    %

    Non-GAAP effective tax rate (7)

    21

    %

    21

    %

     

    Condensed Consolidated Balance Sheets

    (in thousands)

    (unaudited)

     

     

    March 31, 2021

     

    December 31, 2020

    Assets

     

     

     

    Cash, cash equivalents and marketable securities

    $

    6,923,968

     

     

    $

    6,658,897

     

    Accounts receivable, net

    977,551

     

     

    885,352

     

    Inventories

    298,863

     

     

    280,777

     

    Property and equipment, net

    986,123

     

     

    958,534

     

    Goodwill and intangible assets

    1,402,158

     

     

    1,402,158

     

    Deferred tax assets

    815,890

     

     

    882,779

     

    Other assets

    710,506

     

     

    683,311

     

    Total assets

    $

    12,115,059

     

     

    $

    11,751,808

     

     

     

     

     

    Liabilities and Shareholders' Equity

     

     

     

    Accounts payable and accrued expenses

    $

    1,659,876

     

     

    $

    1,560,110

     

    Finance lease liabilities

    572,856

     

     

    581,476

     

    Contingent consideration

    185,700

     

     

    189,600

     

    Other liabilities

    716,373

     

     

    733,807

     

    Shareholders' equity

    8,980,254

     

     

    8,686,815

     

    Total liabilities and shareholders' equity

    $

    12,115,059

     

     

    $

    11,751,808

     

     

     

     

     

    Common shares outstanding

    258,829

     

     

    259,890

     

    Notes and Explanations

    1: In the three months ended March 31, 2021 and 2020, "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) decreases in the fair value of the company's strategic investments and (iii) collaborative milestone payments. In the three months ended March 31, 2020, "Tax adjustments" also included a non-recurring discrete benefit to the company's provision for income taxes of $50.4 million, relating to the write-off of a long-term intercompany receivable, that the company excluded from its Non-GAAP measures.

    2: The company's increased combined GAAP R&D and SG&A expenses guidance reflects the expected effect upon closing of the company's contemplated transaction with CRISPR, which was announced in April 2021. 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 $1.12 billion to $1.17 billion of R&D expenses related to existing and contemplated collaboration agreements and $430 million to $455 million of stock-based compensation expense. The guidance regarding combined GAAP R&D and SG&A expenses does not include estimates associated with any potential future business development activities other than the company's contemplated transaction with CRISPR.

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

    4: During the three months ended March 31, 2021 and 2020, the change in the fair value of contingent consideration relates to potential payments to Exonics Therapeutics' former equity holders.

    5: "Collaborative revenues and expenses" in the three months ended March 31, 2021 and 2020 primarily related to collaborative milestone payments.

    6: "Acquisition-related costs" in the three months ended March 31, 2021 and 2020 related to costs associated with the company's acquisition of Exonics Therapeutics in 2019.

    7: The company released its valuation allowance on the majority of its net operating losses and other deferred tax assets as of December 31, 2018. As of December 31, 2020, the company had utilized substantially all of its remaining federal net operating losses. As a result, a larger portion of the company's tax provision will represent a cash tax payable beginning in 2021, 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, data submissions, and approvals, (ii) anticipated future label expansions, (iii) the expectations, development plans and anticipated timelines for the company's medicines, drug candidates and pipeline programs, including clinical site activations, patient enrollment, and data availability, (iv) expectations for the collaborations with CRISPR, including expectations regarding completion of enrollment in the CTX001 clinical studies and anticipated benefits of the collaborations, (v) expectations for uptake of and expanded access to the company's medicines, including additional reimbursement agreements, (vi) expectations for an increase in the number of CF patients treated with our medicines, (vii) expectations for VX-548, including our plans to advance VX-548 into Phase 2 proof-of-concept studies in the second half of 2021, (viii) expectations for the transactions contemplated by the amended collaboration with CRISPR, including satisfaction of closing conditions, antitrust clearance, anticipated upfront and milestone payments to CRISPR, and anticipated future activities of the parties, (ix) expected effect on our expenses upon the closing of the transaction contemplated by the amended collaboration with CRISPR, and (x) 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, that the closing of the transaction contemplated by the amended collaboration with CRISPR may not occur in a timely manner, or at all, that 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 company may not realize the anticipated benefits from our collaborations with third parties, 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 5:30 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.

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