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Investing.com -- Shares of PTC (NASDAQ:PTC) Therapeutics, Inc. (NASDAQ:PTCT) fell 17% following the release of results from their Phase 2 PIVOT-HD study of PTC518 in patients with Huntington’s disease. Despite meeting its primary endpoint and exhibiting a favorable safety profile, the market response was negative, reflecting skepticism about the drug’s potential for accelerated approval.
The study, which focused on Stage 2 and Stage 3 Huntington’s disease patients, showed a significant reduction in blood Huntingtin protein levels at Week 12, with the 12-month data indicating dose-dependent lowering of the protein and trends across clinical scales. CEO Matthew B. Klein expressed optimism about the drug’s potential and mentioned ongoing discussions about next steps, including the possibility of accelerated approval.
However, the stock’s decline suggests investor concerns, particularly regarding the less pronounced effects in Stage 3 patients and the absence of definitive supportive data for an association between mutant huntingtin reduction and clinical outcomes, which is a key consideration for the FDA’s accelerated approval pathway.
Analysts have echoed this sentiment. Joseph P. Schwartz from Leerink Partners reiterated a Market Perform rating with a $52.00 price target, noting that the Street does not seem convinced by PTCT’s evidence of associations necessary for accelerated approval. He also highlighted that the data call into question whether PTC Therapeutics and its partner will be able to avoid a large, lengthy Phase 3 trial.
TD Cowen’s Joseph Thome remarked on the dose-dependent lowering of mutant huntingtin protein observed in the study but pointed out that the functional data need more time to mature, making a near-term accelerated approval pathway less likely. Thome also noted that while the data did not meet expectations for functional measures at 12 months, there is still support for further investigation, particularly in Stage 2 patients.
On the call, PTCT highlighted dose-dependent reductions in mutant huntingtin and changes in clinical outcomes as evidence for these associations, but the market’s reaction and questions raised during the call indicate a lack of consensus on the drug’s pathway forward.
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