Researcher Study Non-Opioid Pain Medication for Diabetic Neuropathy

On the tail of their win as the first FDA-approved CRISPR-based gene editing therapy, Vertex Pharmaceuticals announced the results of a phase 2 clinical trial for VX-548, a non-opioid pain management drug for patients with diabetic peripheral neuropathy.

The company’s press release, published on December 13, 2023, identified the drug as an investigational oral medication that inhibits NaV voltage-gated sodium channels with a high selectivity for NaV1.8. NaV1.8 is thought to mediate pain signaling in the peripheral nervous system. Previously, the company conducted studies validating it as a genetic target for pain management.

Throughout the phase 2 clinical trial, the researcher evaluated the change in average daily pain using the 11-point Numeric Pain Rating Scale (NPRS), where a 0 indicated no pain and a 10 indicated the worst pain. Patients with diabetic peripheral neuropathy were asked to rate their baseline pain and then randomized into four arms. Three groups, including the high-, mid-, and low-dose groups, were experimental, receiving 69, 46, or 23 mg of VX-548, respectively, once daily. The final group acted as a control group and was given 100 mg of pregabalin three times per day.

At the end of the 12-week experimental period, all four groups were asked to rate their pain on the NPRS scale again. The control group reported an average pain reduction of 2.09 points. Comparatively, the experimental groups achieved more significant pain relief, averaging 2.26-, 2.11-, and 2.18-point pain reductions for the high-, mid-, and low-dose groups.

“We are very pleased with these results, which add to the body of safety and efficacy data for VX-548 and provide further validation of the analgesic effects of NaV1.8 inhibitors,” said Carmen Bozic, MD, Executive Vice President of Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex, in the press release. “Given the favorable benefit/risk profile of VX-548 seen in this study, we are working with urgency to advance this investigational non-opioid pain medicine into phase 3 in painful diabetic neuropathy with the goal of changing the standard of care for neuropathic pain, where treatment options are limited. In addition, our phase 3 studies of VX-548 in acute pain are on track to read out in the first quarter of 2024.”

Moving forward, the company has begun a second phase 2 study on the drug in patients with painful lumbosacral radiculopathy. In the meantime, it continues exploring the drug for acute pain in a phase 3 clinical trial.