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On Friday, Rocket Pharmaceuticals (NASDAQ:RCKT), currently trading at $6.73 with a market cap of $725 million, received a vote of confidence from Cantor Fitzgerald, as the firm reiterated its Overweight rating on the stock and increased the price target to $30.00, up from the previous target of $20.00. According to InvestingPro data, analysts maintain a strong buy consensus with price targets ranging from $13 to $55. The adjustment follows Rocket Pharmaceuticals’ report of preliminary data for its gene therapy candidate RP-A601, designed to treat PKP2-Arrhythmogenic Cardiomyopathy, a rare heart disease.
The encouraging early data from a cohort of three patients showed strong protein expression and early signs of clinical benefit. Notably, meaningful improvements were observed in right ventricular (RV) function, New York Heart Association (NYHA) Class, and Kansas City Cardiomyopathy Questionnaire-12 (KCCQ-12) scores. InvestingPro analysis shows the company maintains a strong balance sheet with more cash than debt and a healthy current ratio of 9.19, providing runway for continued clinical development. Additionally, two out of three patients experienced a roughly 60% reduction in premature ventricular contractions (PVCs), while the third patient showed improvements in other arrhythmia markers and significant enhancement in other cardiac parameters.
However, one patient did encounter severe side effects, including sepsis, pancreatitis, and liver function test (LFT) elevation, believed to be associated with the immune suppression regimen used in the therapy. Fortunately, due to the strong outcomes at the initial dose level of 8e13 vg/kg, further dose escalation has been deemed unnecessary, potentially reducing the risk of adverse effects related to the adeno-associated virus (AAV) used in the therapy.
Rocket Pharmaceuticals has faced challenges recently, with delays in its gene therapy portfolio causing investors to question the company’s future prospects and the broader investability of gene therapy firms. Despite these concerns, the latest data has provided a more optimistic view of Rocket Pharmaceuticals’ pipeline and its potential to advance toward a registration-enabling study. The company is currently evaluating the next phases of development for RP-A601. InvestingPro analysis indicates the stock is currently undervalued, though investors should note the company’s rapid cash burn rate and negative EBITDA of $260 million in the last twelve months. For deeper insights into RCKT’s financial health and growth prospects, including 8 additional ProTips and comprehensive valuation metrics, check out the full Pro Research Report available on InvestingPro.
In other recent news, Rocket Pharmaceuticals reported promising preliminary results from a Phase 1 clinical trial for its gene therapy, RP-A601, aimed at treating plakophilin-2 related arrhythmogenic cardiomyopathy. The trial showed the therapy was well-tolerated and associated with improved heart function and quality of life for patients. In related developments, BMO Capital Markets maintained its Outperform rating for Rocket Pharmaceuticals, with a target price of $50.00, citing potential positive updates on the company’s Danon Disease program. Furthermore, Cantor Fitzgerald reaffirmed its Overweight rating on Rocket Pharmaceuticals, expressing increased confidence in the company’s gene therapy program after a recent management webinar.
In executive news, Rocket Pharmaceuticals appointed Sarbani Chaudhuri as the new Chief Commercial & Medical (TASE:BLWV) Affairs Officer, bringing extensive experience from her previous roles at Johnson & Johnson and AstraZeneca (NASDAQ:AZN). Additionally, Rocket Pharmaceuticals was among several biotech firms experiencing a decline in stock value following the resignation of FDA’s top vaccine official, Peter Marks, which raised concerns about changes in the regulatory landscape. Despite these market fluctuations, analyst firms like BMO Capital Markets and Cantor Fitzgerald remain optimistic about the company’s future prospects.
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