VRTX Vertex Pharmaceuticals Incorporated
Upcoming Catalysts
| Drug | Stage | Catalyst Date |
|---|---|---|
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Elexacaftor, tezacaftor and ivacaftor
Children ages 6 to 11 years who have two F508del mutations and one F508del mutation and one minimal function mutation
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Phase 3
Phase 3
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Drug Pipeline
| Drug | Stage | Notes |
|---|---|---|
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CTX001
Beta-thalassemia
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Phase 1/2
Phase 1/2
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Phase 1/2 data presented at EHA June 12, 2020. Two patients are transfusion independent at 5 and 15 months after infusion.
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CTX001
Sickle cell disease
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Phase 1/2
Phase 1/2
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Phase 1/2 data presented at EHA June 12, 2020. Patient is free of vaso-occlusive crises at 9 months after CTX001 infusion.
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VX-147
Focal segmental glomerulosclerosis (FSGS)
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Phase 2
Phase 2
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Phase 2 trial has been initiated - noted April 29, 2020.
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VX-814
alpha-1 antitrypsin (AAT) deficiency
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Phase 2
Phase 2
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Phase 2 trial has temporarily paused screening and enrolment - April 29, 2020.
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VX-864
Alpha-1 antitrypsin (AAT) deficiency
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Phase 1
Phase 1
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Phase 1 trial ongoing.
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VX-561 and VX-121
Cystic Fibrosis
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Phase 2
Phase 2
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Phase 2 combo trial initiated May 2019.
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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
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Approved
Approved
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FDA Approval announced October 21, 2019.
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Tezacaftor / ivacaftor
Cystic fibrosis - Two Copies of the F508del Mutation
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Approved
Approved
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Label expanded to now include all patients over 6 years old - June 21, 2019.
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VX-659 in combination with tezacaftor and ivacaftor
Cystic fibrosis (CF) who have one F508del mutation and one minimal function mutation (F508del/Min)
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Phase 3
Phase 3
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Phase 3 positive data released November 27, 2018. 14% improvement in ppFEV1 cf placebo.
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VX-150
Neuropathic pain
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Phase 2
Phase 2
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Phase 2 data released December 18, 2018. Primary endpoint met.
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VX-659 in combination with tezacaftor and ivacaftor
Cystic Fibrosis Who Have Two Copies of the F508del Mutation
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Phase 3
Phase 3
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Phase 3 positive data released November 27, 2018. 10% improvement in ppFEV1 cf placebo.
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VX-561
Cystic fibrosis who have a gating mutation
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Phase 2
Phase 2
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Phase 2 monotherapy trial initiated 2Q 2019.
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Ivacaftor
Cystic Fibrosis (6-12 mths)
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Approved
Approved
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FDA Approval announced April 30, 2019.
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Lumacaftor and ivacaftor
Cystic fibrosis (CF) ages 1-2.
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Approved
Approved
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Approval announced August 15, 2018.
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Ivacaftor
Cystic fibrosis (CF) ages 2 to 5.
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Approved
Approved
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FDA Approval announced August 7, 2018.
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ORKAMBI
Cystic fibrosis (CF) ages 6-11 who have F508del mutation
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Approved
Approved
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Approved September 28, 2016.
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VX-661
Cystic fibrosis - one copy of the F508del mutation and a second mutation that results in minimal CFTR Function
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Phase 3
Phase 3
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Phase 3 trial terminated August 2016
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Tezacaftor (VX-661) / ivacaftor
Cystic fibrosis - one copy of the F508del mutation and a second mutation that results in a gating mutation
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Phase 3
Phase 3
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Phase 3 data released October 25, 2017 - primary endpoint not met.
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KALYDECO (ivacaftor)
Children ages 2 to 5 with cystic fibrosis who have the G551D or one of the eight additional gating mutations
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Approved
Approved
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Approved March 17, 2015.
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Lumacaftor and ivacaftor
Cystic fibrosis (CF) ages 12 and older who have two copies of the F508del mutation
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Approved
Approved
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Approved July 2, 2015.
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KALYDECO (ivacaftor)
Cystic fibrosis (CF) ages 2 and older who have one of 23 residual function mutations.
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CRL
CRL
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CRL issued February 5, 2016.
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KALYDECO
Cystic fibrosis (CF) ages 6 and older who have the R117H mutation
|
Approved
Approved
|
Approved December 29, 2014.
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Latest News
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-CF patients in England will be among the first in Europe to benefit from access to KAFTRIO®, if the medicine is approved by the European Commission-
Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today announced that it has expanded its reimbursement agreement with NHS England for Vertex's cystic fibrosis medicines to include KAFTRIO® (ivacaftor/tezacaftor/elexacaftor), in a combination regimen with KALYDECO® (ivacaftor) 150 mg, ahead of the medicine's anticipated approval by the European Commission.
The new expanded agreement includes reimbursed access to Vertex's currently licensed medicines — KALYDECO® (ivacaftor), ORKAMBI® (lumacaftor/ivacaftor) and SYMKEVI® (tezacaftor/ivacaftor), as well as the triple combination therapy if approved — and any future additional licensed indications for all of these medicines.
Reshma Kewalramani, M.D., Chief Executive Officer and President at Vertex, said, "I'm pleased that NHS England has recognized the value of KAFTRIO, and that Vertex and NHS England have been able to work quickly, collaboratively and flexibly to expand the existing reimbursement agreement to include the triple combination therapy in advance of the medicine being licenced. This will ensure that eligible patients in England will be among the first in Europe to benefit from access to this innovative medicine upon approval."
The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) recently adopted a positive opinion for KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with KALYDECO® (ivacaftor) 150 mg to treat people with cystic fibrosis (CF) ages 12 and older with one F508del mutation and one minimal function mutation (F/MF) or two F508del mutations (F/F) in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
As part of the agreement with NHS England, Vertex has committed to submit ORKAMBI®, SYMKEVI® and KAFTRIO® to the National Institute for Health and Care Excellence (NICE) within an agreed upon timetable, allowing for a period of real-world data collection on the medicines.
Vertex will be working closely with the authorities in Northern Ireland, Wales and Scotland with the aim of securing an equivalent agreement in those countries as soon as possible.
Vertex's CF medicines are reimbursed in more than 20 countries around the world including Australia, France, Germany, the Republic of Ireland, Italy, Switzerland, Spain, Denmark, the UK and the U.S.
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 KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in a Combination Regimen With KALYDECO® (ivacaftor)
KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with KALYDECO® (ivacaftor) is an investigational medicine developed for the treatment of cystic fibrosis (CF) in patients ages 12 years and older who have at least one copy of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. It is designed to increase the quantity and function of the F508del-CFTR protein at the cell surface. It recently received a CHMP positive opinion, with the EU license expected over the next few months.
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, 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 10 consecutive years on Science magazine's Top Employers list and top five on the 2019 Best Employers for Diversity list by Forbes. For company updates and to learn more about Vertex's history of innovation, visit https://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, the statements by Dr. Kewalramani in this press release and statements regarding our expectations for the patient population that will be able to access Vertex's medicines, the timing of such access, our commitment to submit ORKAMBI, SYMKEVI and KAFTRIO to NICE for a period of real-world data collection and our plans to secure additional agreements with other countries. 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 obtaining approval for and commercializing our medicines in Europe, and other risks listed under 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. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.(VRTX-GEN)
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– If granted Marketing Authorization, people ages 12 and older in Europe who have one F508del mutation and one minimal function mutation will for the first time be able to benefit from a medicine that treats the underlying cause of the disease –
– People 12 years of age and older who have two F508del mutations also will be eligible for the new triple combination regimen –
Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today announced that the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion for KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with KALYDECO® (ivacaftor) 150 mg to treat people with cystic fibrosis (CF) ages 12 and older with one F508del mutation and one minimal function mutation (F/MF) or two F508del mutations (F/F) in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. If approved, up to 10,000 people in Europe with CF ages 12 and older who have one F508del mutation and one minimal function mutation would be newly eligible for a medicine that targets the disease- causing protein defect. Additionally, people 12 years of age and older who have two F508del mutations and who are currently eligible for one of Vertex's other EMA-approved cystic fibrosis medicines would be eligible for the new triple combination regimen.
The CHMP positive opinion was based on the results of two global Phase 3 studies in people with CF: a 24-week placebo-controlled study in people ages 12 years and older with one F508del mutation and one minimal function mutation and a 4-week head-to-head study of the triple combination therapy in comparison to tezacaftor/ivacaftor in people ages 12 years and older with two F508del mutations. Both Phase 3 studies showed statistically significant and clinically meaningful improvements in lung function (percent predicted forced expiratory volume in one second; ppFEV1), which was the primary endpoint, and in all key secondary endpoints. In both studies, the ivacaftor/tezacaftor/elexacaftor plus ivacaftor combination regimen was generally well tolerated.
"The clinical data for ivacaftor/tezacaftor/elexacaftor plus ivacaftor in people with CF ages 12 years and older with an F/F or F/MF genotype are unprecedented. In addition to improvements in lung function, the data have shown improvements in multiple important outcome measures, including quality of life as measured by the CFQ-R respiratory domain score," said Professor Marcus A. Mall, M.D., Head of Department of Pediatric Pulmonology, Immunology and Critical Care Medicine at Charité University Medical Center Berlin. "Both the clinical and patient communities are excited that more people with CF will be able to benefit from CFTR modulators."
"We are delighted to have received this positive opinion from CHMP. If approved, this would be the first CFTR modulator for people with one F508del mutation and one minimal function mutation and would bring additional benefit to people with two F508del mutations," said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex. "This milestone brings us one step closer to delivering this innovative CF medicine to those who are waiting, and toward our ultimate goal of providing a therapeutic option for every person with this rare and devastating disease."
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 KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in a Combination Regimen With KALYDECO® (ivacaftor)
KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with KALYDECO® (ivacaftor) is an investigational medicine developed for the treatment of cystic fibrosis (CF) in patients ages 12 years and older who have at least one copy of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. KAFTRIO® is designed to increase the quantity and function of the F508del-CFTR protein at the cell surface. The EU submission for KAFTRIO® was supported by positive results of two global Phase 3 studies in people ages 12 years and older with CF: a 24-week Phase 3 study in 403 people with one F508del mutation and one minimal function mutation (F/MF) and a four-week Phase 3 study in 107 people with two F508del mutations (F/F).
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, 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 10 consecutive years on Science magazine's Top Employers list and top five on the 2019 Best Employers for Diversity list by Forbes.
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. Mall and Dr. Bozic in this press release, and statements regarding our expectations for the approval of KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with KALYDECO® (ivacaftor) in Europe, and our expectations regarding the eligible patient population 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 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 registration or further development of its compounds due to safety, efficacy or other reasons, risks related to obtaining approval for and commercializing medicines in Europe, and other risks listed under 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. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.
(VRTX-GEN)
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-Vertex Foundation Announces $1.5 Million Gift to Boston University's New Center for Antiracist Research-
-Biotechnology-focused curriculum established in partnership with Year Up-
-Additional commitments made to STEAM education-
Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today announced that Vertex and the Vertex Foundation, a nonprofit charitable foundation, have committed $4 million over three years to fighting racism and injustice in America and around the world.
The donations are an extension of Vertex's long-standing commitment to equity, diversity and inclusion and the existing work of the Vertex Foundation to promote these values in its communities through education, innovation and health programs.
Boston University Center for Antiracist Research
As part of this commitment, the Vertex Foundation will donate $1.5 million, allocated over three years, to help establish the new Boston University Center for Antiracist Research. Under the leadership of Founding Director Ibram X. Kendi, the Center will study and develop new ways to understand, explain and solve seemingly intractable problems of racial inequity and injustice. As the first corporate foundation partner for the Center, the Vertex Foundation's contribution will support and advance this critical mission.
The Vertex Foundation's donation will support the Center's research and data collection to identify innovative policies that may reduce or eliminate racial disparities, including health disparities, in the United States. In order to extend the Center's reach beyond the University and spark both dialogue and action within the broader community, the Foundation's gift will also support the establishment of an annual public symposium led by the Center on wide-ranging topics relating to antiracism.
President Robert A. Brown of Boston University said, "The creation of the Center for Antiracist Research under the leadership of Ibram X. Kendi is a milestone in Boston University's commitment to lead in the research that leads to policy changes to end systemic racism in the United States. We are very grateful to Vertex and the Vertex Foundation both for their financial support and their thought partnership as we take on one of the great societal inequities of our time."
"At Vertex, we have a strong and long-standing commitment to creating a diverse and inclusive workplace and to promoting social justice and economic and educational opportunities within our communities. Today's announcement reflects our ongoing commitment to address the roots of systemic racism and injustice within our communities and broader society," said Reshma Kewalramani, M.D., Chief Executive Officer and President of Vertex. "We are proud to serve as an ally to the Boston University Center for Antiracist Research. Today's gift to Boston University from the Vertex Foundation, as well as additional donations to the City of Boston, Year Up and other important organizations from Vertex, are extensions of our long-term commitment to address these issues head-on in our communities, with our employees and for the patients we serve."
"I know that the launch of the Center at BU has been a bright spot for many people and I think this gift will brighten it that much more," said Founding Director Ibram X. Kendi, one of the nation's leading scholars on racism and a best-selling author. "Vertex and the Vertex Foundation realize the importance and seriousness of our work, and they are taking the lead."
Year Up
The Vertex corporate office is making a new multiyear commitment to Year Up to establish the first-ever biotechnology curriculum to prepare young adults for future careers in research, development and medicine. Year Up's mission is to close the Opportunity Divide by ensuring that young adults gain the skills, experiences and support that will empower them to reach their potential through careers and higher education.
"Year Up looks forward to partnering with Vertex to create career pathways in biotech for our students, the majority of whom are Black or Hispanic/Latino," said Year Up Founder and CEO Gerald Chertavian. "By enabling young people of color to launch careers in more high-growth industries, we are driving real change on behalf of social justice and racial equity within corporate America."
In addition to this commitment, Vertex is also enhancing its support of its long-standing partners including:
- Boston Private Industry Council, which works at the intersection of business and community interests to connect Boston residents to promising career pathways, while creating a diverse talent pipeline for local employers. Funding will focus on reaching out to students who have not connected with their high schools during the first phase of remote teaching and learning.
- Bottom Line, which helps low-income and first-generation-to-college students get to and through college. They are committed to building strong connections with students, providing them with individual support, and ensuring they have the guidance they need to persist and earn a college degree and successfully launch a career. Bottom Line provides mentorship and support to our Vertex Science Leaders Scholarship program, a fully funded four-year scholarship, plus mentorship with a Vertex scientist and access to a college internship.
- Biomedical Science Careers Program (BSCP), a program that aims at increasing the representation of underrepresented minority and disadvantaged individuals in all facets of science and medicine while helping health care institutions, biopharma/biotechnology firms, educational institutions, professional organizations and private industry members meet their need for a diverse workforce. The primary objective of all BSCP activities is to identify, inform, support and provide mentoring for academically outstanding students/fellows, particularly African American, Hispanic/Latino and American Indian/Alaska Native individuals.
Additional Vertex Foundation Commitments
The Vertex Foundation has longstanding commitments to organizations that promote education, innovation and health, especially those that focus on underrepresented minorities and girls/women. The Foundation is augmenting those commitments with additional donations to the following organizations in their work to support racial and social justice:
- City Year, an existing partner of the Foundation, is a national organization committed to long-term partnerships with students in systemically under-resourced communities and schools. Through education they are working to advance and develop leaders who will create a more just and equitable future for all. The Vertex Foundation's support will help City Year serve more than 226,000 students in 350 schools across the country by addressing educational inequities that harm communities of color.
- Equal Justice Works, which facilitates opportunities for law students and lawyers to engage in public service and bring lasting change to underserved communities across the country. This year, the Vertex Foundation funded its first Equal Justice Works Fellow, helping Equal Justice Works expand in Boston. This additional commitment will support one additional Fellow in Boston and another Fellow in California, both beginning in 2021.
The Foundation is also doubling its match for employees' charitable donations to eligible organizations that support social and racial justice.
About the Vertex Foundation
The Vertex Foundation is a nonprofit charitable foundation. It seeks to improve the lives of people with serious diseases and in its communities through education, innovation and health programs. Established in 2017, the Foundation is a long-term source of charitable giving and is part of Vertex Pharmaceuticals' corporate giving commitment. To learn more about the Vertex Foundation, visit https://www.vrtx.com/responsibility/vertex-foundation.
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, 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 10 consecutive years on Science magazine's Top Employers list and top five on the 2019 Best Employers for Diversity list by Forbes. 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)
View source version on businesswire.com: https://www.businesswire.com/news/home/20200625005512/en/
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-Beta thalassemia: Two patients are transfusion independent at 5 and 15 months after CTX001 infusion; data demonstrate clinical proof-of-concept for CTX001 in transfusion-dependent beta thalassemia-
-Sickle cell disease: Patient is free of vaso-occlusive crises at 9 months after CTX001 infusion-
-Five patients with beta thalassemia and two patients with sickle cell disease have been treated to date with CTX001 and all have successfully engrafted-
ZUG, Switzerland and CAMBRIDGE, Mass. and BOSTON, June 12, 2020 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (NASDAQ:CRSP) and Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today announced new clinical data for CTX001, an investigational CRISPR/Cas9 gene-editing therapy, from the CLIMB-111 and CLIMB-121 Phase 1/2 trials in transfusion-dependent beta thalassemia (TDT) and severe sickle cell disease (SCD), and highlighted recent progress in the CTX001 development program. These data were presented during an oral presentation at the European Hematology Association (EHA) virtual congress by Dr. Selim Corbacioglu, Professor of Pediatrics and the Chair of Pediatric Hematology, Oncology, and Stem Cell Transplantation, Regensburg University Hospital, Regensburg, Germany.
CLIMB-111 Trial in Transfusion-Dependent Beta Thalassemia Updated Results
Data presented today at EHA demonstrate clinical proof-of-concept for CTX001 in TDT. Data include longer-duration follow-up data for the first patient with TDT treated with CTX001 and new data for the second TDT patient treated. CRISPR Therapeutics and Vertex announced initial data for the first TDT patient in November of 2019.Patient 1 with TDT has the β0/IVS-I-110 genotype, which is associated with a severe phenotype similar to β0/β0, and had a transfusion requirement of 34 units of packed red blood cells per year (annualized rate during the two years prior to consenting for the trial) before enrolling in the clinical trial. As previously reported, the patient achieved neutrophil engraftment 33 days after CTX001 infusion and platelet engraftment 37 days after infusion. After CTX001 infusion, two serious adverse events (SAEs) occurred, neither of which the principal investigator (PI) considered related to CTX001: pneumonia in the presence of neutropenia, and veno-occlusive liver disease attributed to busulfan conditioning; both subsequently resolved. New data presented today show that at 15 months after CTX001 infusion, the patient was transfusion independent and had total hemoglobin levels of 14.2 g/dL, fetal hemoglobin of 13.5 g/dL, and F-cells (erythrocytes expressing fetal hemoglobin) of 100.0%. Bone marrow allelic editing was 78.1% at 6 months and 76.1% at one year.
Patient 2 with TDT has the β0/IVS-II-745 genotype and had a transfusion requirement of 61 units of packed red blood cells per year (annualized rate during the two years prior to consenting for the trial) before enrolling in the clinical trial. The patient achieved neutrophil engraftment 36 days after CTX001 infusion and platelet engraftment 34 days after infusion. After CTX001 infusion, two SAEs occurred, neither of which the PI considered related to CTX001: pneumonia and an upper respiratory tract infection; both subsequently resolved. At 5 months after CTX001 infusion, the patient was transfusion independent and had total hemoglobin levels of 12.5 g/dL, fetal hemoglobin of 12.2 g/dL, and F-cells (erythrocytes expressing fetal hemoglobin) of 99.4%.
Hemoglobin data over time are presented for Patient 1 and Patient 2 below.
Figure 1 accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/35581299-d683-44b0-a75e-7a1a9b9fe9eb
CLIMB-121 Trial in Severe Sickle Cell Disease Updated Results
Data presented today at EHA reflect longer-duration follow-up data for the first patient with SCD treated with CTX001. CRISPR Therapeutics and Vertex announced initial data for this first SCD patient in November of 2019.Patient 1 with SCD experienced seven vaso-occlusive crises (VOCs) and five packed red blood cell transfusions per year (annualized rate during the two years prior to consenting for the trial) before enrolling in the clinical trial. As previously reported, the patient achieved neutrophil and platelet engraftment 30 days after CTX001 infusion. After CTX001 infusion, three SAEs occurred, none of which the PI considered related to CTX001: sepsis in the presence of neutropenia, cholelithiasis and abdominal pain; all subsequently resolved. New data presented today show that at 9 months after CTX001 infusion, the patient was free of VOCs, was transfusion independent and had total hemoglobin levels of 11.8 g/dL, 46.1% fetal hemoglobin, and F-cells (erythrocytes expressing fetal hemoglobin) of 99.7%. Bone marrow allelic editing was 81.4% at 6 months. Figure 2 presents the hemoglobin data over time for this patient.
Figure 2 accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/7610c5bd-25c8-4f5b-be86-8bc16ed57eb1
"With these new data, we are beginning to see early evidence of the potential durability of benefit from treatment with CTX001, as well as consistency of the therapeutic effect across patients," said Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics. "These highly encouraging early data represent one more step toward delivering on the promise and potential of CRISPR/Cas9 therapies as a new class of potentially transformative medicines to treat serious diseases."
"The data announced today are remarkable, including the demonstration of clinical proof-of-concept in TDT," said Reshma Kewalramani, M.D., Chief Executive Officer and President of Vertex. "While these are still early days, these data mark another important milestone for this program and for the field of gene editing. The results presented at this medical conference add to results previously shared demonstrating that CRISPR/Cas9 gene editing has the potential to be a curative therapy for severe genetic diseases like sickle cell and beta thalassemia."
"In my 25 years of caring for children and young adults facing both sickle cell disease and beta thalassemia, I have seen how these diseases can adversely affect patients' lives in very significant ways," said Dr. Haydar Frangoul, Medical Director of Pediatric Hematology and Oncology at Sarah Cannon Research Institute, HCA Healthcare's TriStar Centennial Medical Center and senior author of the abstract presented at the EHA virtual congress. "I am encouraged by the preliminary results, which demonstrate, in essence, a functional cure for patients with beta thalassemia and sickle cell disease."
Recent Progress in the Phase 1/2 Clinical Trials
CLIMB-111 for TDT has dosed a total of 5 patients, and all patients have successfully engrafted. The trial is also now open for concurrent dosing after successful dosing and engraftment of the first two patients. Additionally, CLIMB-111 has been expanded to allow enrollment of β0/β0 patients and is in the process of being expanded to allow enrollment of pediatric patients ages 12 years or older.CLIMB-121 for SCD has dosed a total of 2 patients and both patients have successfully engrafted. The trial is also now open for concurrent dosing after successful dosing and engraftment of these first two patients.
The initial safety profile in these trials appears to be consistent with myeloablative busulfan conditioning and an autologous hematopoietic stem cell transplant.
In March 2020, clinical trial sites in the U.S. and Europe temporarily paused their elective hematopoietic stem cell transplant programs due to the COVID-19 pandemic, and as a result, CRISPR and Vertex temporarily paused conditioning and dosing in these trials. Enrollment, mobilization and drug product manufacturing in each trial remains ongoing. The companies are now in the process of re-initiating dosing with CTX001 at certain clinical trial sites. The CLIMB-111 and CLIMB-121 clinical trials are ongoing, and patients will be followed for 2 years following CTX001 infusion. The companies expect to provide additional data in the second half of 2020.
About CTX001
CTX001 is an investigational ex vivo CRISPR 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) from the U.S. FDA, Orphan Drug Designation from both the FDA and the European Medicines Agency (EMA), and Fast Track Designation from the FDA for both SCD and TDT.
CTX001 is being developed under a co-development and co-commercialization agreement between CRISPR Therapeutics and Vertex. CTX001 is the most advanced gene-editing approach in development for TDT and SCD.
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 18 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 18 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 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 status of clinical trials (including, without limitation, the expected timing of data releases and activities at clinical trial sites) related to product candidates under development by CRISPR Therapeutics and its collaborators, including expectations regarding the data that is being presented at the European Hematology Association's virtual congress; (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: potential impacts due to the coronavirus pandemic, such as the timing and progress of clinical trials; 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; 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, 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.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, 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 10 consecutive years on Science magazine's Top Employers list and top five on the 2019 Best Employers for Diversity list by Forbes. 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. Kulkarni, Dr. Kewalramani and Dr. Frangoul in this press release, and statements regarding our plans and expectations for our clinical trials and clinical trial sites, and our expectations regarding future data announcements. 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 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. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.(VRTX-GEN)
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- KALYDECO® (ivacaftor) is the first and only approved medicine in Europe to treat the underlying cause of cystic fibrosis in patients with the R117H mutation, the most common residual function mutation, in children as young as 6 months of age -
Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today announced that the European Commission has granted approval of the label extension for KALYDECO® (ivacaftor) to include the treatment of children and adolescents with cystic fibrosis (CF), ages 6 months and older and weighing at least 5 kg, who have the R117H mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene – the most common residual function mutation underlying CF.
"A little over eight years ago, KALYDECO® was approved as the first and only medicine to treat the underlying cause of cystic fibrosis in patients with specific mutations," said Reshma Kewalramani, M.D., Chief Executive Officer and President at Vertex. "Since then, it's been our goal to ensure that as many people with CF as possible are able to benefit from our treatments, and today's label extension means that approximately 500 young patients in Europe, who have long awaited a treatment option, are now eligible for KALYDECO®."
Now approved, KALYDECO® (ivacaftor) will be immediately available to additional eligible patients in Germany and shortly in countries where respective long-term reimbursement agreements have been previously secured. Vertex will work closely with all other relevant government authorities to secure access for eligible patients as quickly as possible.
In Europe, KALYDECO® (ivacaftor) is already approved for the treatment of people with CF ages 18 and older with the R117H mutation, and in infants 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, 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.
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, 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 10 consecutive years on Science magazine's Top Employers list and top five on the 2019 Best Employers for Diversity list by Forbes.
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. Kewalramani in this press release, and statements regarding our expectations for the availability of KALYDECO in Europe, and our plans for securing access to KALYDECO for eligible patients 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 annual report and subsequent quarterly reports filed with the Securities and Exchange Commission and available through the company's website at www.vrtx.com. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.
(VRTX-GEN)
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