Replimune's experimental melanoma treatment, RP1, has secured support from an FDA advisory panel for approval in combination with Bristol Myers Squibb's Opdivo for patients whose melanoma has progressed after other immunotherapies. This decision comes despite the FDA having rejected RP1 twice before and agency scientists raising concerns about the clarity and interpretability of the company's trial data, particularly regarding the individual contributions of RP1 and Opdivo. The panel ultimately voted 10-3 that the efficacy results from Replimune's key study were "evaluable and clinically meaningful," with some experts emphasizing the significant unmet need for this patient population.
FDA staff had argued that Replimune's trial design made it difficult to differentiate RP1's impact from Opdivo's, and that the survival analysis presented was unreliable. The trial itself lacked a control arm and primarily measured tumor shrinkage, with Replimune reporting about one-third of patients saw some tumor response and 15% achieved complete remission. However, panel members largely challenged the FDA's stance, highlighting the drug's impact in a difficult-to-treat group and the potential for a confirmatory trial, expected in late 2027, to provide further evidence.
The FDA's target action date for a final decision on RP1's approval was August 2. Following the advisory committee's positive vote, analysts at Wedbush anticipate FDA approval, though they noted the decision could still face delays. Leerink, upgrading Replimune to 'Outperform' from 'Market Perform' and raising its price target to $17 from $11, believes the FDA is now "boxed into approval" due to the strong advisory committee vote, the acknowledged unmet medical need, and the current political climate favoring faster access to therapies. Wedbush also noted Replimune's potential financing risk, with its cash runway only expected to fund operations into Q1 2027.
The approval would target an initial market of 150 clinics, expanding to 350 within six to nine months of launch. This marks a significant turnaround for Replimune, whose shares were up more than 100% in trading following the advisory panel's recommendation.
