Leerink Partners analyst Joseph Schwartz has reiterated their bullish stance on RARE stock, giving a Buy rating yesterday.
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Joseph Schwartz’s rating is based on Ultragenyx’s strategic progress with their gene therapy, DTX401, which targets Glycogen Storage Disease Type Ia (GSDIa). The company has initiated a rolling Biologics License Application (BLA) submission to the FDA, indicating a significant step towards commercialization. The therapy has shown promising results in reducing daily cornstarch intake, a major burden for GSDIa patients, with pivotal data revealing substantial reductions over time. Additionally, Ultragenyx’s decision to bring DTX401 manufacturing in-house reflects their commitment to addressing any potential regulatory challenges proactively. Schwartz also notes the potential market size, with approximately 6,000 GSDIa patients in developed regions, and projects gross peak sales of around $460 million in the US and EU, with a 60% probability of success.
While investor attention is currently more focused on the setrusumab program for osteogenesis imperfecta, Schwartz believes that DTX401’s progress could garner more recognition as it advances. The ongoing developments and the strategic approach taken by Ultragenyx support the Buy rating, as the company is well-positioned to address unmet medical needs and capitalize on market opportunities.
In another report released yesterday, William Blair also maintained a Buy rating on the stock with a $0.00 price target.
Based on the recent corporate insider activity of 44 insiders, corporate insider sentiment is negative on the stock. This means that over the past quarter there has been an increase of insiders selling their shares of RARE in relation to earlier this year.