As previously reported, UBS analyst Carter Gould downgraded Vertex to Neutral from Buy, arguing that the valuation of its cystic fibrosis franchise appears "full" following the run-up in the stock on the heels of the company's data from Phase 1 and Phase 2 studies of three different triple combination regimen. Gould raised his price target on Vertex shares to $174 from $135, as he sees visibility for the CF franchise to approach $8B in revenue by the end of the 2020s.

Vertex Pharmaceuticals announced important advancements across its suzetrigine pain program, which has the potential to be the first new class of medicine for acute and neuropathic pain in more than two decades. Suzetrigine is an oral selective NaV1.8 pain signal inhibitor. Following the positive Phase 3 results in acute pain announced in January, the FDA has granted a rolling New Drug Application (NDA) submission for suzetrigine in moderate-to-severe acute pain. Vertex has started the rolling submission process and is on track to complete the submission in the second quarter of 2024. Suzetrigine was previously granted FDA Fast Track and Breakthrough Therapy designations in moderate-to-severe acute pain. In neuropathic pain, Vertex released positive results from its Phase 2 study in December 2023 and recently completed a successful end-of-phase 2 meeting with the FDA. Vertex is now preparing to initiate a Phase 3 pivotal program of suzetrigine in patients with DPN in 2H 2024. In addition, the FDA recently granted suzetrigine Breakthrough Therapy designation for the treatment of pain associated with DPN. The Phase 3 program will include two identical 12-week randomized, double-blind, placebo-controlled studies evaluating the efficacy and safety of suzetrigine in patients with DPN. The primary endpoint for both studies will be the change from baseline in weekly average of daily pain intensity on the numeric pain rating scale, or NPRS, assessed at Week 12 compared to placebo. Both studies will also include a key secondary endpoint of change from baseline in the weekly average of daily pain intensity on the NPRS at Week 12 compared to pregabalin. Approximately 1,100 patients are expected to enroll in each Phase 3 study. After completing participation in the randomized controlled studies, patients may roll over into an open-label study to evaluate the long-term safety and effectiveness of suzetrigine in DPN. Additionally, Vertex continues to enroll its Phase 2 study of suzetrigine in patients with lumbosacral radiculopathy, or LSR, which is pain caused by impairment or injury to nerve roots in the area of the lumbar spine. The company is on track to complete enrollment in the Phase 2 LSR study by the end of the year.