Vertex Announces Publication of VX-548 Positive Phase 2, Proof-of-Concept Results in New England Journal of Medicine
- Strong efficacy and favorable benefit-risk profile demonstrated in Phase 2 trials for VX-548
- Breakthrough Therapy and Fast Track designations granted for moderate to severe acute pain
- Potential to bring an effective, non-opioid therapeutic option to patients suffering from acute pain
- None.
- First published manuscript of VX-548 preclinical and clinical trial results in a peer-reviewed journal -
“VX-548 preclinical studies demonstrated high selectivity and potency for the NaV1.8 sodium channel which translated into Phase 2 clinical results with strong efficacy, a favorable benefit-risk profile and promise in the treatment of acute pain,” said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex. “Our Phase 3 trials for VX-548 are well underway, and we look forward to the potential of bringing an effective, non-opioid therapeutic option to patients suffering from moderate to severe acute pain.”
“Acute pain is frequently treated with opioids that come with significant safety and tolerability concerns, including sedation, respiratory depression and the potential for misuse and addiction,” said Scott Weiner, M.D., M.P.H., co-author of the NEJM manuscript, Associate Professor of Emergency Medicine at Harvard Medical School and Attending Emergency Physician in the Department of Emergency Medicine at Brigham and Women’s Hospital. “There has not been a new class of pain medicine in decades, and we urgently need new treatment options to help the millions of people affected by pain each year in the
Vertex expects to complete the pivotal program in late 2023 and share results from these studies in late 2023 or early 2024. VX-548 has been granted Breakthrough Therapy and Fast Track designations in the
About VX-548
VX-548 is an oral, selective NaV1.8 inhibitor that is highly selective for NaV1.8 relative to other NaV channels. NaV1.8 is a voltage-gated sodium channel that plays a critical role in pain signaling in the peripheral nervous system. NaV1.8 is a genetically validated target for the treatment of pain, and Vertex has previously demonstrated clinical proof-of-concept with a small molecule investigational treatment targeting NaV1.8 in multiple pain indications including acute pain, neuropathic pain and musculoskeletal pain. Vertex’s approach is to selectively inhibit NaV1.8 using small molecules with the objective of creating a new class of medicines that have the potential to provide superior relief of pain without the limitations of opioids, including their addictive potential. VX-548 is one of the most recent molecules to enter clinical development from Vertex’s portfolio of NaV1.8 inhibitors.
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 clinical pipeline of investigational small molecule, mRNA, cell and genetic therapies (including gene editing) in other serious diseases where it has deep insight into causal human biology, including sickle cell disease, beta thalassemia, APOL1-mediated kidney disease, acute and neuropathic pain, type 1 diabetes and alpha-1 antitrypsin deficiency.
Founded in 1989 in
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 Carmen Bozic, M.D., and Scott Weiner, M.D., M.P.H., in this press release, and statements regarding the potential benefits of VX-548 as a treatment for patients with acute pain, expectations that the pivotal program will complete in late 2023, plans to share results from these studies in late 2023 or early 2024, and our expectations for VX-548. 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 studies may not be indicative of final clinical trial results, 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, that data may not be available on the anticipated timeline, or at all, that our development programs may experience delays, and other risks listed under the heading “Risk Factors” in Vertex's most recent annual report and subsequent filings 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.
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