Business Wire

Vertex Advances VX-548 in Acute and Neuropathic Pain

Share

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that following the positive Phase 2 results earlier this year and having reached agreement on the design of the pivotal development program with the U.S. Food and Drug Administration (FDA), Vertex plans to advance the selective NaV1.8 inhibitor VX-548 into Phase 3 clinical trials in the fourth quarter of 2022. Vertex also intends to initiate a Phase 2 dose-ranging study of VX-548 in neuropathic pain by the end of this year. In addition, the FDA has granted VX-548 Breakthrough Therapy Designation for the treatment of moderate-to-severe acute pain.

The Phase 3 program will include two randomized, double-blind, placebo-controlled studies evaluating the efficacy and safety of VX-548 for moderate to severe acute pain following bunionectomy or abdominoplasty surgery. Both studies will also include a hydrocodone bitartrate/acetaminophen treatment arm. A third, single-arm study will evaluate the safety and effectiveness of VX-548 for up to 14 days across multiple other types of moderate to severe acute pain.

“We are very pleased to complete our discussions with the FDA and reach agreement on the design of the pivotal development program for acute pain,” said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex. “VX-548 has demonstrated a positive benefit-risk profile in Phase 2 and we are working with urgency to bring forward a medicine that delivers effective pain control without addictive potential to patients who are waiting.”

About the VX-548 Pivotal Program in Acute Pain
The Phase 3 program will consist of two randomized, double-blind, placebo-controlled studies evaluating the efficacy and safety of VX-548 (100 mg first dose, followed by 50 mg every 12 hours) for acute pain after bunionectomy or abdominoplasty. The primary endpoint in both studies will be the time-weighted Sum of the Pain Intensity Difference over the first 48 hours of treatment (SPID48), as recorded on the 11-point Numeric Pain Rating Scale (NPRS) compared to placebo. A key secondary endpoint in both studies will be the SPID48 of VX-548 compared to hydrocodone bitartrate/acetaminophen.

The Phase 3 program will also include a single-arm study evaluating the safety and effectiveness of VX-548 in multiple other types of moderate to severe acute pain. In this study, patients will be treated for up to 14 days.

About the VX-548 Phase 2 Study in Neuropathic Pain
The Phase 2 study will be a randomized, double-blind, active-controlled, dose-ranging study evaluating the efficacy and safety of VX-548 in people with painful diabetic peripheral neuropathy. The primary endpoint is the change from baseline in the weekly average of daily pain intensity on a numeric pain rating scale (NPRS) at Week 12.

About FDA Breakthrough Therapy Designation
The FDA's Breakthrough Therapy Designation is intended to expedite development and review of medicines that aim to address a serious condition with preliminary clinical evidence indicating that the drug may demonstrate substantial improvement over existing treatments on one or more clinically significant endpoints. The Breakthrough Therapy Designation was granted to VX-548 based on clinical evidence from the Phase 2 proof-of-concept studies demonstrating the potential of VX-548 to treat moderate to severe acute pain.

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 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.

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, cell and genetic therapies in other serious diseases where it has deep insight into causal human biology, including sickle cell disease, beta thalassemia, APOL1-mediated kidney disease, pain, type 1 diabetes, alpha-1 antitrypsin deficiency and Duchenne muscular dystrophy.

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 12 consecutive years on Science magazine's Top Employers list and one of the 2021 Seramount (formerly Working Mother Media) 100 Best Companies. 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 made by Dr. Carmen Bozic in this press release, and statements regarding the advancement of VX-548 into pivotal development for acute pain and to initiate Phase 2 study of VX-548 for neuropathic pain, the potential benefits of VX-548, the anticipated timelines and dosing associated with ongoing and future clinical trials, study designs, including expectations on patient enrollment, expectations regarding efficacy endpoints, and the expedited development of VX-548 resulting from the FDA’s Breakthrough Therapy Designation. 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 the trial may not be completed in the expected timeframe, or at all, 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, and other risks listed under the heading “Risk Factors” in Vertex's most recent annual report filed with the Securities and Exchange Commission (SEC) and available through the company's website at www.vrtx.com and on the SEC’s website at www.sec.gov. 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)

To view this piece of content from cts.businesswire.com, please give your consent at the top of this page.

Contact information

Vertex Pharmaceuticals Incorporated
Investors:
InvestorInfo@vrtx.com
or
Miroslava Minkova: +1 617-341-6135

Media:
mediainfo@vrtx.com
or
U.S.: +1 617-341-6992
or
Heather Nichols: +1 617-839-3607
or
International: +44 20 3204 5275

About Business Wire

For more than 50 years, Business Wire has been the global leader in press release distribution and regulatory disclosure.

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

Faster Recoveries, Improved Security: HTEC and G2 Risk Solutions Join Forces to Advance Bankruptcy Management28.3.2024 19:02:00 EET | Press release

HTEC, a global digital consulting, software engineering, and product development company, is announcing a strategic collaboration with G2 Risk Solutions (G2RS), the preeminent provider of end-to-end bankruptcy technology solutions. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20240328622816/en/ HTEC & G2 Risk Solutions Partnership (Photo: Business Wire) Under the agreement, G2RS will engage HTEC to incorporate its leading-edge technology into G2RS’ premier bankruptcy management platform. As the creators of the most comprehensive national bankruptcy database and the first bankruptcy management portal, G2RS is an industry pioneer. By adding HTEC’s premium engineering to the G2RS technology stack, G2RS will offer new and innovative functionalities to help clients expedite financial recoveries, increase data protection, and reduce operational inefficiencies in the bankruptcy management life cycle. “We are thrilled to be a part o

LambdaTest Launches The Phoenix Project, an Employee Resource Group for Women28.3.2024 18:00:00 EET | Press release

LambdaTest, a leading cloud-based unified testing platform, announced the launch of The Phoenix Project, an Employee Resource Group (ERG) dedicated to supporting and promoting the success of its female employees. “LambdaTest is committed to fostering a diverse, inclusive, and equitable workplace where all employees feel valued and empowered to reach their full potential,” said Chandini Chopra, VP of People and Culture at LambdaTest. “The Phoenix Project is a critical step towards achieving this goal by providing a platform for professional development, networking, and mentorship, especially for our women employees.” The Phoenix Project Aims To: Offer professional development workshops, networking opportunities, and dedicated time off for women to participate in ERG initiatives. Provide mentorship programs connecting senior female employees with mentees. Promote leadership development among women within the company. Create a sense of community for women in the workplace. Leadership and

The reopening of the Yokohama Museum of Art adds another innovative and distinctive venue for events in Yokohama28.3.2024 18:00:00 EET | Press release

After extensive renovations, the iconic Yokohama Museum of Art has reopened its doors to visitors from all over the world. The museum’s reopening paves the way to new and closer collaboration with international events in Yokohama. This extraordinary venue is the ideal setting for a one-of-a-kind gathering, in part, supported by the Yokohama Convention & Visitors Bureau (hereinafter YCVB). The Yokohama Museum of Art Founded in 1989, the Yokohama Museum of Art collects over 14,000 works of art that reflect the many facets of life in Yokohama—past, present, and future. The reopening of the museum coincides with the Yokohama Triennale, an international exhibition featuring contemporary artists whose decentralized exhibits turn the entire metropolis into an art museum. The 8th Yokohama Triennale is being held from March 15 to June 9, 2024, with the Yokohama Museum of Art being a pivotal venue for the exhibition. With the museum firmly back on the map as Yokohama’s leading artistic venue, YC

Eight new sustainability-themed experiences—exclusive to Yokohama—for convention participants28.3.2024 18:00:00 EET | Press release

In a survey conducted by Yokohama Convention & Visitors Bureau (hereinafter YCVB), many convention attendees expressed particular interest in Yokohama’s culture, sightseeing spots, and local cuisine. In response, YCVB has developed eight new Yokohama Tours for event attendees to enjoy during their free time in Yokohama, keeping in line with our ethos of sustainability. Noge Area Bar-Hopping Tour, complete with guide Known affectionately by locals as Yokohama’s kitchen, Noge is a bustling nightlife spot that is home to over 600 izakaya (Japanese-style pubs). A knowledgeable local guide will lead participants to all the hidden gems in Noge, where they can indulge in distinctive, delicious pub-style food and drink. With easy on-foot accessibility, participants can rest assured this fun night out won’t impact their carbon footprint. Soto Zen Tour of Sojiji, head temple of the Soto Zen school As a prominent Zen sect in Japan, Soto Zen provides one of the best Zen wellness experiences. Durin

Nordson EFD Releases New 3-Axis Automated Fluid Dispensing Systems28.3.2024 17:00:00 EET | Press release

Nordson EFD , a Nordson company (NASDAQ: NDSN) and leading precision fluid dispensing systems manufacturer is proud to announce the new GVPlus and PROX families of automated fluid dispensing products. Both robotic solutions share a focus on motion, workspace, repeatability, payload, setup, and vision technology enhancements. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20240305726899/en/ The GV Series offers market-leading dimensional positional accuracy and deposit placement repeatability. (Photo: Business Wire) The new GVPlus automated fluid dispensing solution offers improved repeatability, bigger payloads with simplified setup requirements, and superior vision capabilities. Repeatability is now best-in-class at 8 μm, improving repeatability to ±0.008 mm. Setting up the robot is easier due to a new dual mounting flange that enables a tool payload of up to 4.5 kg (10 lbs). When paired with the working area of 400 mm x 400

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
HiddenA line styled icon from Orion Icon Library.Eye