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
Andersen offentliggør regnskab for fjerde kvartal og helåret 202510.3.2026 19:52:00 CET | Pressemeddelelse
Andersen Group Inc. (NYSE: ANDG) ("Andersen"), en førende udbyder af uafhængig rådgivning inden for skat, vurdering og finans til private kunder og familiekontorer, virksomheder og fonde i USA, vil offentliggøre sine finansielle resultater for hele året og fjerde kvartal 2025 efter børslukketid tirsdag den 17. marts 2026. Andersens administrerende direktør og bestyrelsesformand, Mark L. Vorsatz, og Andersens økonomidirektør, Neal Livingston, vil afholde en telekonference for at fremlægge Andersens økonomiske resultater tirsdag den 17. marts 2026 kl. 17.00 ET. Deltagere kan følge webcasten på https://event.choruscall.com/mediaframe/webcast.html?webcastid=J3Hvslre. Et link til optagelsen af webcasten vil kunne findes på Andersens Investor Relations-webside på investor.andersen.com inden for et par timer efter begivenheden og vil være tilgængeligt på websiden i seks måneder. Om Andersen Andersen er en førende udbyder af uafhængig rådgivning inden for skat, vurdering og finans til private
Xtep-Sponsored Chinese Teenage Dancer Claims World Championship at Juste Debout Street Dance World Finals10.3.2026 19:46:00 CET | Press release
Xtep, a well-known sportswear brand from China, proudly announced that Xtep-sponsored Chinese teenage dancers delivered an electrifying performance at the Juste Debout Street Dance World Finals in Paris on Sunday, March 8, 2026, claiming top honors and showcasing a unique fusion of traditional Chinese culture with modern street dance. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20260310519224/en/ Chinese Teens Dance to Glory at Juste Debout World Finals Li Yongqiu, a 14-year-old from Southwestern China’s Chongqing Municipality, made history by winning the Global Championship in the Junior Dance Tour, becoming the third Chinese dancer to achieve this feat. His explosive power and technical mastery, honed through years of training that integrates Kungfu elements, captivated judges and audiences alike. The Chinese contingent's success didn't stop there. Zhang Xinlan and Zhang Chuyi secured a Top 8 finish in the Popping Adult D
ATLAS Infrastructure Invests in H2O America, Backs Long-Term Growth Strategy10.3.2026 18:29:00 CET | Press release
ATLAS anchors H2O America’s equity raise and establishes new substantial shareholdingUpsized transaction highlights strong market support for H2O’s long-term strategy and sector leading growth outlookH2O’s organic growth fully equity funded into 20281 ATLAS Infrastructure (“ATLAS”) is a specialist Global Listed Infrastructure investor managing funds on behalf of long-term infrastructure clients. Following the recent equity placement, ATLAS’s actively managed accounts hold ~10.8% voting and economic interest in H2O America (“H2O”). ATLAS was pleased to support H2Os long‑term strategy to invest in local water and wastewater utility operations through our participation in the recent equity raise. ATLAS recognises the disciplined approach from H2O management which focuses on organic investment in existing businesses alongside targeted and accretive transactions such as the acquisition of Quadvest which materially expands H2O’s regulated water footprint in a premium high‑growth jurisdiction
TIXiMED Announces Promising Preclinical Data: Oral TIX100 Prevents Weight Regain After GLP-1 Cessation in Obesity Model10.3.2026 18:22:00 CET | Press release
TIX100 prevented weight regain in mice after semaglutide discontinuation Supports potential role as oral weight maintenance therapy following GLP-1 Receptor Agonist (GLP-1) treatment, addressing a major challenge in long-term obesity management TIXiMED, Inc., a clinical-stage pharmaceutical company developing a novel oral therapy targeting thioredoxin-interacting protein (TXNIP) for diabetes and metabolic disorders, is pleased to highlight new research by investigators at the University of Alabama at Birmingham (UAB) Comprehensive Diabetes Center. The study demonstrates that its oral compound, TIX100, effectively prevents weight rebound following discontinuation of GLP-1 treatment in a mouse model of diet-induced obesity. In the study, mice treated with semaglutide experienced significant weight loss of over 13% body weight during a 2-week treatment period while continuing a high-fat diet. Upon discontinuation of semaglutide, untreated mice rapidly regained 14% of their body weight, mi
Svante and Integrated Packaging Company Advance U.S. Biogenic CDR Project to Feasibility10.3.2026 14:57:00 CET | Press release
The bioenergy with carbon capture (BECCS) project aims to remove more than 500,000 tonnes per year of biogenic CO2 emissions from a U.S. paper mill’s recovery boiler Svante Development Inc. is co-investing to advance the project The project will generate high-quality carbon dioxide removal (CDR) credits to market to Voluntary Carbon Market (VCM) buyers. Svante Technologies Inc. (“Svante”) announced today that its bioenergy with carbon capture and storage (BECCS) project at a paper mill in the Southeast U.S. has progressed to the feasibility study phase. The project is being developed in partnership with an integrated sustainable packaging company, following an extensive screening and pre-feasibility study conducted across several of the partner’s mills. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20260310319382/en/ During the feasibility study phase, the companies will complete further engineering and design activities, cos
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