On Monday, Stifel, a financial services company, maintained its Buy rating and $40.00 stock price target for Regenxbio Inc. (NASDAQ:RGNX). This affirmation follows the announcement of an expanded clinical trial for a Duchenne muscular dystrophy (DMD) gene therapy.
Regenxbio revealed the extension of its Phase 1/2 AFFINITY DUCHENNE trial to include a new cohort of younger patients. This group will consist of up to five individuals between the ages of 1 and 3, who will be administered RGX-202 at Dose Level 2. This is the same dosage planned for the pivotal study phase.
The inclusion of a younger demographic is a notable development in DMD treatment research, as it aims to preserve muscle function before significant deterioration occurs. Traditionally, this age group has been excluded from such studies due to potential safety concerns.
The U.S. Food and Drug Administration (FDA) has approved the cohort expansion, signaling a vote of confidence in the safety of RGX-202. To date, no major adverse events (AEs) have been reported with the use of the therapy. Moreover, the FDA has confirmed that an accelerated development pathway for RGX-202 remains a possibility.
In other recent news, REGENXBIO Inc. has made significant strides in its operations. The biotechnology firm announced the expansion of its Phase I/II AFFINITY DUCHENNE trial, which includes a new cohort to assess the gene therapy RGX-202. The company also reported a productive first quarter with total revenue of $15.6 million and a net loss of $63.3 million.
In addition, REGENXBIO has appointed Curran Simpson as the new President and Chief Executive Officer, and it is on track to submit a rolling Biologics License Application (BLA) for RGX-121, a gene therapy candidate for the treatment of Mucopolysaccharidosis Type II (MPS II).
Analyst firms H.C. Wainwright and RBC Capital have maintained their positive ratings on REGENXBIO's stock, citing the company's strategic positioning in the market and progress of its drug candidates. These recent developments highlight the company's ongoing efforts in advancing its gene therapy pipeline.
InvestingPro Insights
Following Stifel's endorsement of Regenxbio Inc. (NASDAQ:RGNX), current financial metrics from InvestingPro provide additional context to investors considering the company's stock. Regenxbio's market capitalization stands at $640.82 million, reflecting the market's valuation of the company amidst its ongoing clinical trials and research activities.
Notably, the company's price to book ratio over the last twelve months as of Q1 2024 is 1.63, suggesting that the stock may be valued reasonably in relation to the company's book value.
InvestingPro Tips indicate that Regenxbio holds more cash than debt on its balance sheet, which could be a sign of financial stability and potential for funding future research without incurring significant debt. Moreover, the stock's Relative Strength Index (RSI) suggests it is in oversold territory, which might attract investors looking for potential rebound opportunities.
These insights, alongside the fact that analysts have revised their earnings downwards and anticipate the company will not be profitable this year, paint a complex picture for potential investors. For those interested in a deeper analysis, there are over 10 additional InvestingPro Tips available at: https://www.investing.com/pro/RGNX. To access these valuable insights, use the coupon code PRONEWS24 to get an additional 10% off a yearly or biyearly Pro and Pro+ subscription.
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