Sami Corwin, an analyst at William Blair, maintained his Outperform rating on Rocket Pharmaceuticals Inc. (NASDAQ:RCKT) on May 27 with no price target, despite the company’s recent difficulties. The biotechnology company’s trial for the gene therapy medicine RP-A501 was clinically halted and a patient tragically passed away.
Corwin stated that the delay was required due to the capillary leak syndrome (CLS) safety event, which had never been observed with RP-A501 or comparable treatments before. Rather than the AAV9 vector utilized in the treatment, Corwin expressed hope that the serious adverse event could be related to the recent addition of a C3 inhibitor to the immunosuppressive protocol. Only individuals who received the C3 inhibitor had CLS cases documented, the analyst said.
Although the occurrences of thrombotic microangiopathy (TMA) are alarming, the analyst also pointed out that the afflicted patients are making a full recovery. Despite the RCKT stock’s price tanking by around 80% year-to-date, Corwin thinks that investors are primarily concerned about the safety of the program and when the clinical hold would be lifted. He went on to say that Rocket’s other clinical cardiovascular program, RP-A601, which employs a different AAV vector and does not include the C3 inhibitor, remains unaffected, implying that the recent occurrences shouldn’t impact how this distinct program is viewed.
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