Potential for Blockbuster Drug Status
Analysts anticipate that if VX-548 gains approval from regulators, it could become a blockbuster drug with annual sales surpassing $1 billion. Vertex is now focused on pushing the drug into a late-stage trial, a crucial step closer to regulatory approval. Additionally, the company is conducting late-stage studies to assess its effectiveness in treating acute pain, with data expected in the first quarter of next year. Acute pain results from various factors, such as injury, surgery, or illness.
Expanding Market Potential
Vertex executives are optimistic about VX-548’s market potential, projecting it to be a multi-billion dollar product for acute pain as well as chronic nerve pain in diabetes patients. These expectations have contributed to the significant increase in Vertex’s share price, which has risen nearly 40% this year. The recent approval of the first-ever gene-editing therapy for sickle cell disease, developed by Vertex and CRISPR Therapeutics, has also had a positive impact on the company’s stock.
Mid-Stage Trial Details and Results
The phase-two trial, spanning 12 weeks and involving approximately 160 patients with diabetic peripheral neuropathy, demonstrated the drug’s effectiveness in reducing pain intensity. The condition, caused by high blood sugar levels in diabetes patients, damages peripheral nerves and can lead to mild to severe pain, numbness, and other complications.
Using an 11-point scale to measure pain intensity, with 10 being the most severe pain possible, the high, mid, and low doses of VX-548 resulted in average pain intensity reductions of 2.26, 2.11, and 2.18 points, respectively. Vertex reported that the drug was generally well tolerated, with most adverse events being mild or moderate. The trial also compared VX-548 with pregabalin, an approved non-opioid therapy for nerve pain and seizures, affirming the painkiller’s efficacy.
Investors have responded positively to these trial results as they meet or exceed expectations, validating the potential effectiveness of Vertex’s painkiller candidate.