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Vertex Pharmaceuticals is a global biotechnology company dedicated to developing transformative medicines for serious diseases, particularly in specialty markets . The company has developed four approved medicines that address the underlying cause of cystic fibrosis (CF), a genetic disease, and one approved therapy for severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT), both of which are life-shortening blood disorders . Vertex is also advancing its pipeline with clinical-stage programs targeting various conditions, including acute and neuropathic pain, APOL1-mediated kidney disease, and type 1 diabetes, among others .
- Cystic Fibrosis Medicines - Develops and markets treatments that address the underlying cause of cystic fibrosis, with TRIKAFTA/KAFTRIO being the leading product used by nearly three-quarters of CF patients in North America, Europe, and Australia .
- Gene-Edited Cell Therapy (CASGEVY) - Offers a CRISPR/Cas9 gene-edited cell therapy for severe sickle cell disease and transfusion-dependent beta thalassemia, approved in several regions including the U.S. and Europe .
- Clinical-Stage Programs - Advances a pipeline of clinical-stage programs targeting acute and neuropathic pain, APOL1-mediated kidney disease, type 1 diabetes, and other conditions .
| Name | Position | External Roles | Short Bio | |
|---|---|---|---|---|
Jeffrey M. Leiden ExecutiveBoard | Executive Chairman | Board Member at Massachusetts Mutual Life Insurance Company, Odyssey Therapeutics, Casana | Jeffrey M. Leiden, M.D., Ph.D., has been with Vertex since 2009, leading the company through significant growth and expansion into new disease areas. | |
Reshma Kewalramani ExecutiveBoard | President, CEO, and Director | None | Reshma Kewalramani, M.D., has been with Vertex since 2017, leading significant milestones including the approval of multiple therapies and pipeline expansion. | View Report → |
Charles F. Wagner, Jr. Executive | Executive Vice President and CFO | Board Member at The TJX Companies, Inc. | Charles F. Wagner, Jr. joined Vertex in 2019, overseeing financial strategy and operations. | |
Duncan J. McKechnie Executive | Senior Vice President, Head of North America Commercial | None | Duncan J. McKechnie has been with Vertex for approximately 12 years, overseeing successful launches of CF medicines and other therapies. | |
Jonathan Biller Executive | Executive Vice President and CLO | None | Jonathan Biller has been with Vertex since 2022, serving as the Chief Legal Officer. | |
Kristen C. Ambrose Executive | Senior Vice President and CAO | None | Kristen C. Ambrose joined Vertex in 2021, leading accounting, tax, and treasury functions. | |
Stuart A. Arbuckle Executive | Executive Vice President and COO | Board Member at Rhythm Pharmaceuticals Inc., ImmunoGen, Inc. | Stuart A. Arbuckle has been with Vertex since 2012, leading the launch of all Vertex CF medicines and overseeing global commercial strategy. | |
Alan Garber Board | Director | Board Member at Exelixis, Inc., Interim President of Harvard University | Alan Garber has been a Director at Vertex since 2017, contributing his expertise in healthcare economics and policy. | |
Bruce I. Sachs Board | Director | General Partner at Charles River Ventures | Bruce I. Sachs has been a Director at Vertex since 1998, providing business leadership and financial expertise. | |
Diana McKenzie Board | Director | Board Member at MetLife Inc., agilon health, inc., Special Advisor to Brighton Park Capital, Board Member at Paradox AI | Diana McKenzie has been a Director at Vertex since 2020, recognized for her expertise in technology innovation and corporate leadership. | |
Jennifer Schneider Board | Director | Co-Founder and CEO of Homeward Health, Inc. | Jennifer Schneider has been a Director at Vertex since 2025, with extensive experience in healthcare technology and leadership. | |
Lloyd Carney Board | Director | Board Member at Visa Inc., Grid Dynamics Holdings Inc., Ambassador/Special Investment Envoy for Technology Jamaica, Chancellor of the University of Technology, Jamaica | Lloyd Carney has been a Director at Vertex since 2019, bringing expertise from his roles in technology and leadership. | |
Michel Lagarde Board | Director | Executive Vice President and COO at Thermo Fisher Scientific Inc. | Michel Lagarde joined the Vertex board in 2023, bringing experience from his leadership roles in the pharmaceutical industry. | |
Nancy A. Thornberry Board | Director | Board Member at Denali Therapeutics, Inc., Schrödinger, Inc. | Nancy Thornberry joined the Vertex board in 2023, with over 30 years of experience in drug discovery and development. | |
Sangeeta N. Bhatia Board | Director | Professor at MIT, Co-Founder of several biotech companies | Sangeeta N. Bhatia has been a Director at Vertex since 2015, providing insights from her extensive experience in biomedical engineering and medical research. | |
Suketu Upadhyay Board | Director | Executive Vice President and CFO at Zimmer Biomet Holdings, Inc. | Suketu Upadhyay has been a Director at Vertex since 2022, contributing financial expertise from his roles in the healthcare industry. |
- With suzetrigine entering a highly competitive pain management market, how confident are you in securing timely and favorable payer reimbursement, especially given payers are concerned about the budget impact due to the large number of acute pain patients?
- Considering the potential availability of cost-effective generic opioids and non-opioid alternatives, how do you plan to price suzetrigine to demonstrate its value proposition to payers and patients while remaining competitive?
- Despite the significant need for non-opioid pain treatments, how will you drive physician adoption of suzetrigine given the entrenched prescribing habits and possible skepticism towards new therapies in acute pain management?
- Given that povetacicept's Phase III RAINIER trial involves post-dose monitoring, what specific regulatory strategies are you implementing to ensure approval for at-home monthly dosing, and could this requirement impact your projected launch timeline?
- Recognizing the complexities and lengthy process involved in administering CASGEVY, what measures are you taking to overcome potential barriers to patient uptake and streamline the treatment journey to improve adoption rates?
Competitors mentioned in the company's latest 10K filing.
| Company | Description |
|---|---|
Orion Corporation | This company is pursuing clinical development of a selective NaV1.8 inhibitor in a Phase 1 clinical trial for acute and chronic pain indications. |
This company has NaV1.8/1.7 inhibitors in preclinical development for pain. | |
Grünenthal | This company is developing NaV1.8/1.7 inhibitors in preclinical stages for pain. |
CellTrans, Inc. | This company developed Lantidra, the first FDA-approved cadaveric islet therapy for the treatment of type 1 diabetes (T1D). |
This company is actively engaged in the research and development of cell therapies for T1D. | |
This company is developing cell therapies for T1D and is also investigating a Janus kinase inhibitor (baricitinib) in a Phase 2 study for APOL1-mediated kidney disease (AMKD). | |
This company is involved in the development of cell therapies for T1D. | |
This company is working on cell therapy approaches for T1D. | |
Seraxis, Inc. | This company is developing cell therapies for T1D. |
This company is engaged in the development of cell therapies for T1D. | |
This company has early programs developing APOL1-targeted assets for patients with AMKD. | |
Maze Therapeutics | This company is working on early programs for APOL1-targeted assets for AMKD. |
This company is developing APOL1-targeted assets for AMKD in early programs. | |
This company is developing sparsentan, which is under regulatory review for the treatment of focal segmental glomerulosclerosis (FSGS). | |
Dimerix | This company is conducting a pivotal study for QYTOVRA, a therapy for FSGS. |
Fair Therapeutics | This company is advancing CFTR modulators licensed from Yumanity Therapeutics, with plans for a Phase 3 trial in European CF patients with rare mutations of the CFTR gene. |
Sionna Therapeutics | This company has at least two CFTR modulators in Phase 1 development and several in preclinical development. |
This company is developing nucleic acid therapies for CF, targeting patients who cannot benefit from CFTR modulators. | |
ReCode Therapeutics, Inc. | This company is working on nucleic acid therapies for CF to address patients who cannot benefit from CFTR modulators. |
This company is developing nucleic acid therapies for CF. | |
Spirovant Sciences, Inc. | This company is pursuing nucleic acid therapies for CF. |
Boehringer Ingelheim International, GmbH | This company is working on nucleic acid therapies for CF. |
This company is developing nucleic acid therapies for CF. | |
SpliSense, Ltd. | This company is involved in the development of nucleic acid therapies for CF. |
This company has approved small molecule and biologic treatments for sickle cell disease (SCD) and beta thalassemia. | |
This company offers approved treatments for SCD and beta thalassemia. | |
This company, together with Merck & Co., provides approved treatments for SCD and beta thalassemia. | |
This company obtained FDA approval for its gene therapies Zynteglo for beta thalassemia and Lyfgenia for SCD. | |
This company is investigating a CRISPR gene-editing asset in clinical trials for SCD and beta thalassemia. |
Notable M&A activity and strategic investments in the past 3 years.
| Company | Year | Details |
|---|---|---|
Alpine Immune Sciences, Inc. | 2024 | Vertex acquired Alpine Immune Sciences, Inc. for approximately $5.0 billion in cash as an asset acquisition, with the lead molecule povetacicept driving significant in-process R&D expenses and additional post-acquisition costs recorded for unvested equity awards. |
ViaCyte | 2022 | Vertex acquired ViaCyte for $315.0 million, gaining stem cell-derived cell replacement capabilities including IP, tools, and a Phase 1/2 hypoimmune cell program that complements its type 1 diabetes research, with the transaction accounted for as a business combination. |
Catalyst Biosciences, Inc. | 2022 | Vertex acquired Catalyst Biosciences’ portfolio of protease medicines for $60 million in cash, securing assets such as the pre-clinical CB 2782-PEG for geographic atrophy in dry AMD, with the payment fully recognized as acquired in-process R&D expenses. |
Recent press releases and 8-K filings for VRTX.
- Total revenue reached $2.77 billion in Q1 2025 with a 3% year-over-year increase and 43% non-GAAP operating margins.
- Updated full-year revenue guidance raised to $11.85–$12B for 2025.
- Robust pipeline progress in CF therapies with key FDA-approved products including ALYFTREK (launched with noninferiority to TRIKAFTA and early U.S. launch), JOURNAVX, and advances in CASGEVY.
- US revenue grew by 9%, while ex-US revenue was impacted by an approximately $100M headwind due to issues in Russia.
- Strategic capital deployment executed with a share repurchase of approximately 930,000 shares for $425M.
- Vertex Pharmaceuticals Incorporated issued an 8-K update on March 28, 2025, outlining developments in its type 1 diabetes portfolio.
- The update covers Phase 1/2 clinical trial results for the VX-264 program, leading to its discontinuation from clinical development and the start of an impairment evaluation of related intangible assets.
- The company expects to incur a non-cash impairment charge of approximately $400 million to GAAP operating expenses in Q1 and full year 2025, with further details to be provided in its upcoming quarterly report.
- Vertex is advancing its diversified portfolio with key product launches, including ALYFTREK for cystic fibrosis and JOURNAVX for acute pain, with early payer coverage progress noted in New York and Arkansas.
- The company provided guidance that JOURNAVX revenue is included in the annual forecast of $11.75B to $12B, with volume ramping ahead of revenues in anticipation of broader coverage later in the year.
- Significant pipeline innovation was highlighted, including updates on VX-993 for pain management, as well as transformative cell therapy programs for type 1 diabetes (e.g., VX-880/ VX-264) and selective sodium channel inhibitors targeting NaV1.7/NaV1.8.