Therapy Areas: Hereditary Disorders
Positive Phase 2, Proof-of-Concept Results for VX-548 Published in New England Journal of Medicine
10 August 2023 - - US-based Vertex Pharmaceuticals Inc. (NASDAQ: VRTX) has announced publication in the New England Journal of Medicine (NEJM) of preclinical data and the results from its Phase 2 proof-of-concept trials evaluating treatment with the selective, oral NaV1.8 inhibitor VX-548 for acute pain following abdominoplasty and bunionectomy surgeries.

Vertex said it 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 US for moderate to severe acute pain.

In addition, Vertex has initiated a Phase 2 dose-ranging study of VX-548 in neuropathic pain.

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.

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 -- 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 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 13 consecutive years on Science magazine's Top Employers list and one of Fortune's 100 Best Companies to Work For.
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