BOSTON–(BUSINESS WIRE)–Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced multiple program updates ahead of upcoming investor meetings in January, including the company’s scheduled webcast from the 43rd Annual J.P. Morgan Healthcare Conference on Monday, January 13, 2025, at 10:30 a.m. ET/7:30 a.m. PT.
“2024 marked another year of excellent progress for Vertex, as we reached more people with CF than ever before, began a new era of commercial diversification, and advanced and broadened our clinical stage pipeline,” said Reshma Kewalramani, M.D., Chief Executive Officer and President of Vertex. “In 2025, we are poised to diversify our business further across multiple dimensions: our revenue, as we continue the launches of CASGEVY, ALYFTREK and potentially launch suzetrigine in acute pain; our pipeline, as we progress four potentially transformative medicines through pivotal trials; and our geographic footprint, as we expand both our commercial and clinical presence globally.”
Disease Areas with Approved Medicines
Cystic Fibrosis (CF)
Sickle Cell Disease (SCD) and Transfusion-Dependent Beta Thalassemia (TDT) – CASGEVY
Pipeline Disease Areas
Acute Pain
Peripheral Neuropathic Pain (PNP)
IgA Nephropathy (IgAN) and other B Cell-Mediated Diseases
APOL1-Mediated Kidney Disease (AMKD) – Inaxaplin (VX-147)
Type 1 Diabetes (T1D)
Myotonic Dystrophy Type 1 (DM1) – VX-670
Autosomal Dominant Polycystic Kidney Disease (ADPKD) – VX-407
J.P. Morgan Healthcare Conference Presentation and Webcast
Dr. Kewalramani will present at the 43rd Annual J.P. Morgan Healthcare Conference on Monday, January 13, 2025, at 10:30 a.m. ET/7:30 a.m. PT.
A live webcast of management’s remarks will be available through the Vertex 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 approved medicines that treat the underlying causes of multiple chronic, life-shortening genetic diseases — cystic fibrosis, sickle cell disease and transfusion-dependent beta thalassemia — and continues to advance clinical and research programs in these diseases. Vertex also has a robust clinical pipeline of investigational therapies across a range of modalities in other serious diseases where it has deep insight into causal human biology, including acute and neuropathic pain, APOL1-mediated kidney disease, IgA nephropathy, primary membranous nephropathy, autosomal dominant polycystic kidney disease, type 1 diabetes and myotonic dystrophy type 1.
Vertex was founded in 1989 and has its global headquarters in Boston, with international headquarters in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia, Latin America and the Middle East. Vertex is consistently recognized as one of the industry’s top places to work, including 14 consecutive years on Science magazine’s Top Employers list and one of Fortune’s 100 Best Companies to Work For. For company updates and to learn more about Vertex’s history of innovation, visit www.vrtx.com or follow us on LinkedIn, Facebook, Instagram, YouTube and Twitter/X.
Special Note Regarding Forward-Looking Statements
This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements by Reshma Kewalramani, M.D., and statements about our expectations for our CF program, including with respect to the diversification of Vertex’s business and expanding commercially and clinically across more geographies, commercial expectations for ALYFTREK, the expectation to have data from the Phase 1/2 study of VX-522, expectations that VX-522 may treat >5,000 people with CF, the company’s beliefs regarding CF epidemiology and market opportunities, expectations for the company’s agreement with CMS and resulting patient access to CASGEVY, expectations that suzetrigine in acute pain will be included on the list of treatments that qualify for add-on payments under the NOPAIN Act, plans to advance suzetrigine into pivotal development for painful LSR, expectations regarding povetacicept in IgAN, including completing enrollment in the interim analysis cohort in 2025 for potential accelerated approval, expectations for the collaboration with Zai Lab, including the future activities of the parties pursuant to the collaboration, expectations regarding inaxaplin in AMKD, including that the company will complete enrollment in the interim analysis cohort in 2025 for potential accelerated approval in the U.S., plans to initiate a Phase 2b open-label study of inaxaplin in patients with AMKD and diabetes or other co-morbidities and expanding the eligible patient population, expectations regarding completion of enrollment and dosing in the pivotal study evaluating zimislecel in 2025, expectations regarding the initial eligible patient population that will benefit from zimislecel, plans to work with urgency to advance zimislecel to be able to serve all patients with severe T1D, plans to develop additional therapies for T1D, plans to share data from the VX-264 Phase 1/2 study in 2025, plans to pursue alternative approaches to immunosuppression that could be used with zimislecel and other T1D product candidates, and expectations to advance VX-407 into a Phase 2 proof of concept study in people with ADPKD in 2025. 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 clinical trial data might not be available on the expected timeline, that the anticipated benefits and potential of Vertex’s collaboration with Zai Lab may not be achieved on the anticipated timeline, or at all, that data from the company’s research and development programs may not support registration or further development of its compounds due to safety, efficacy, and other risks, that our discussions with regulators may be delayed or cause delays in our pipeline programs, and other risks listed under the heading “Risk Factors” in Vertex’s most recent annual report and subsequent quarterly reports filed with the Securities and Exchange Commission at www.sec.gov and available through the company’s website at www.vrtx.com. You should not place undue reliance on these statements. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.
(VRTX-GEN)
Vertex Pharmaceuticals Incorporated
Investors:
InvestorInfo@vrtx.com
or
617-961-7163
Media:
mediainfo@vrtx.com
or
International: +44 20 3204 5275
or
U.S.: 617-341-6992
Vertex Pharmaceuticals Incorporated
Investors:
InvestorInfo@vrtx.com
or
617-961-7163
Media:
mediainfo@vrtx.com
or
International: +44 20 3204 5275
or
U.S.: 617-341-6992
