MA-VERTEX-PHARMA
26.4.2021 13:31:06 CEST | Business Wire | Press release
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that it will advance the selective NaV1.8 inhibitor, VX-548, into Phase 2 clinical development with clinical trials in patients with both visceral and non-visceral types of acute pain. The decision to move to Phase 2 was based on supportive Phase 1 data in healthy volunteers, including safety, tolerability and pharmacokinetics (PK). In these studies, the molecule exhibited a favorable profile at doses considerably lower than those required with our previous NaV1.8 inhibitors.
NaV1.8 is a voltage-gated sodium channel that plays a critical role in pain signaling in the peripheral nervous system. NaV1.8 is genetically validated as a novel 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 acute pain without the limitations of opioids, including their addictive potential. VX-548 is the most recent molecule to enter clinical development from Vertex’s portfolio of NaV1.8 inhibitors.
“Vertex has previously validated the potential role of NaV1.8 inhibitors with positive proof-of-concept results in a variety of pain conditions,” said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex. “We are excited about VX-548, a potent NaV1.8 inhibitor, and we are moving forward to the Phase 2 proof-of-concept trials with urgency, as these studies are the key next step toward our ultimate goal of developing transformative medicines for the treatment of pain.”
About VX-548
VX-548 is an oral, selective NaV1.8 inhibitor that has completed Phase 1 studies. Based on a favorable pharmacokinetic, safety and tolerability profile, it will advance to Phase 2 studies in the second half of 2021.
About the Planned Phase 2 Studies in Acute Pain
The planned Phase 2 studies will be randomized, double-blind, placebo-controlled trials that will evaluate multiple doses of VX-548 in patients with acute pain following bunionectomy surgery or abdominoplasty. Both studies will also include a hydrocodone/acetaminophen arm. The primary endpoint in both the planned studies is the time-weighted Sum of the Pain Intensity Difference over the first 48 hours of treatment (SPID48).
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.
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 Dr. Carmen Bozic in this press release, and statements regarding our plans to advance VX-548 into Phase 2 clinical studies in the second half of 2021, the potential benefits of VX-548, and our plans, expectations and anticipated timelines for these clinical trials. 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 may not support registration or further development of its compounds due to safety, efficacy, or other reasons, that the COVID-19 pandemic may impact the status or progress of our clinical trials, 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)
View source version on businesswire.com: https://www.businesswire.com/news/home/20210426005435/en/
Link:
About Business Wire
Subscribe to releases from Business Wire
Subscribe to all the latest releases from Business Wire by registering your e-mail address below. You can unsubscribe at any time.
Latest releases from Business Wire
EVE Energy Showcases All-Scenario Energy Storage Solutions at The Smarter E Europe 20261.7.2026 03:45:00 CEST | Press release
EVE Energy unveiled its Mr. Big Family series, a 6.9+ MWh energy storage system, and all-scenario energy storage solutions at Intersolar Europe in Munich. Drawing on traceable large-cell technology, proven large-scale energy storage project delivery experience, and global delivery capabilities, the company is addressing Europe's diverse energy storage requirements across utility-scale, commercial & industrial (C&I ), and data center segments. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20260630889717/en/ EVE Energy showcases its Mr. Giant 3.0 6.9+ MWh energy storage system at The Smarter E Europe 2026 in Munich, Germany Advancing Large-Cell Technology with Global Project Validation As one of the first companies to focus on large-capacity energy storage cells, EVE Energy has iteratively upgraded its cell platform from 560 Ah and 628 Ah to 702 Ah, adhering to a stacking process route throughout. At the exhibition, the Mr. Gia
Bending Spoons S.p.A. announces pricing of initial public offering1.7.2026 01:56:00 CEST | Press release
Bending Spoons S.p.A. (“Bending Spoons”), a leading technology company, today announces the pricing of its initial public offering (“IPO”) at $29.00 per share. A total of 57,971,015 ordinary shares are being offered, of which 34,398,640 shares are being offered by Bending Spoons and 23,572,375 shares are being offered by certain selling shareholders (the “Selling Shareholders”). Bending Spoons will not receive any proceeds from any sale of shares by the Selling Shareholders. The shares are expected to begin trading on the Nasdaq Global Select Market under the ticker symbol “BSP” on July 1, 2026. The offering is expected to close on July 2, 2026, subject to customary closing conditions. In addition, Bending Spoons and the Selling Shareholders granted the underwriters an option to purchase up to an additional 5,244,026 ordinary shares from Bending Spoons and up to an additional 3,451,626 ordinary shares from the Selling Shareholders at the initial public offering price, less underwriting
FDA Issues Modified Risk Tobacco Product Orders for 20 ZYN Nicotine Pouch Products30.6.2026 18:19:00 CEST | Press release
FDA’s decision makes ZYN the first nicotine pouch product to receive MRTP orders authorizing reduced-risk claims versus cigarettes Philip Morris International Inc. (PMI) (NYSE: PM) today announced that the U.S. Food and Drug Administration (FDA) issued Modified Risk Tobacco Product (MRTP) orders for 20 variants of ZYN nicotine pouch products. These are the first MRTP orders granted for nicotine pouches, allowing PMI U.S. to market the following claim for the authorized ZYN products: “Using ZYN instead of cigarettes puts you at a lower risk of mouth cancer, heart disease, lung cancer, stroke, emphysema, and chronic bronchitis.” “FDA’s decision is an important moment for the more than 45 million legal-age nicotine consumers in America,” saidStacey Kennedy, PMI U.S. CEO. “Today’s news ensures these adultshave access to accurate, science-based information, including FDA-authorized evidence that switching from cigarettes to ZYN reduces the risk of smoking-related diseases like heart disease
Caidya Announces Strategic Combination with Simbec-Orion Bridging Early Scientific Insight and Global Clinical Execution30.6.2026 17:00:00 CEST | Press release
Caidya today announced a strategic combination with Simbec-Orion designed to close the divide between early scientific insight and global clinical execution. The combination of Caidya and Simbec-Orion creates a differentiated specialty clinical CRO platform that enables programs to scale, maintaining focus, speed, and accountability. The strategic combination brings together complementary strengths to create a more complete development partner for innovative biopharma companies. With established operations across Europe, the Americas, APAC, and China, the combined organization provides meaningful expertise and execution capabilities in the regions that matter most. Simbec-Orion brings early-phase clinical pharmacology capabilities alongside deep therapeutic expertise for later stage complex oncology and rare disease trials, helping sponsors shape critical decisions early in the development lifecycle. Together, the organizations strengthen their ability to support complex, cross-border
Archer® Proves Purpose-Built AI Beats General-Purpose LLMs on Regulatory Change Management: 95% Verified Accuracy, 80x Faster, 92% Lower Cost30.6.2026 16:13:00 CEST | Press release
In a head-to-head benchmark, a leading general-purpose LLM was confidently wrong 35% of the time on regulatory dates. Archer Evolv™ shipped zero errors. For enterprises deploying AI in compliance, a wrong date is a missed deadline. The more dangerous failure is a wrong answer the model returns with high confidence, one that flows silently into a compliance calendar and is only discovered after the window has passed. Archer® today released results showing purpose-built AI beats a general-purpose large language model (LLM) on regulatory work, and it’s not close. This head-to-head test compared Archer’s purpose-built, vertical-specific AI and proprietary data sets against a leading general-purpose LLM, on a core compliance task: determining the publication, effective and comment-close dates of regulatory documents across six jurisdictions. General-purpose models are a genuine breakthrough, and this is no referendum on their quality. The question Archer set out to answer is narrower and mo
In our pressroom you can read all our latest releases, find our press contacts, images, documents and other relevant information about us.
Visit our pressroom