On Thursday, H.C. Wainwright adjusted its price target for NASDAQ:RGNX, Regenxbio Inc (NASDAQ:RGNX)., to $36 from the previous $40, while reaffirming a Buy rating on the stock. This revision follows the release of promising functional data from an early-stage clinical trial for a Duchenne muscular dystrophy (DMD) treatment.
Earlier this week, Regenxbio disclosed the first functional results from its Phase 1/2 AFFINITY DUCHENNE trial evaluating RGX-202, a therapy for DMD. The data encompassed findings from five subjects, including 12-month outcomes from three patients at dose level 1, aged between 4 and 10 years, and 9-month outcomes from two patients at dose level 2, aged 8 and 12 years. The results indicated either stability or improvement in patient function as assessed by the North Star Ambulatory Assessment and timed function tests, compared to matched external natural history controls.
In light of the new data, H.C. Wainwright has increased the probability of approval for RGX-202 from 35% to 45%. Despite the positive clinical outcomes, the firm has moderated its peak sales projections for RGX-202 due to the reported sales of Elevidys, which amounted to $181 million in the third quarter of 2024.
The estimated market value of Regenxbio is now approximately $1.80 billion, or $36 per share. H.C. Wainwright's revised price target reflects these updated assessments while the Buy rating indicates continued confidence in the stock's potential. The firm's analyst emphasized the significance of the functional improvements observed in the DMD patients treated with RGX-202, underscoring the therapy's potential impact on the disease's treatment landscape.
In other recent news, biotechnology firm Regenxbio Inc. has disclosed its financial and operating results for the third quarter of 2024. The firm has also announced promising initial data from its Phase 1/2 trial of Duchenne Muscular Dystrophy gene therapy, RGX-202. The trial revealed functional improvements in patients, a significant achievement given the older age of the trial participants. Regenxbio has also reached a regulatory milestone by aligning with the FDA on using microdystrophin expression as a surrogate biomarker, positioning RGX-202 potentially two years away from market entry.
Raymond (NS:RYMD) James has reaffirmed its Outperform rating and $18.00 price target for Regenxbio, citing the initial trial results as grounds for continued support. Meanwhile, Morgan Stanley (NYSE:MS) has resumed coverage of Regenxbio, assigning an Overweight rating to the stock and highlighting the potential of gene therapy in treating VEGF-mediated diseases. Despite concerns about commercial viability due to subretinal delivery method challenges, the firm expressed cautious optimism about Regenxbio's advancements in gene therapy.
InvestingPro Insights
Recent InvestingPro data provides additional context to Regenxbio's financial situation and market performance. The company's market capitalization stands at $477.12 million, significantly lower than H.C. Wainwright's estimated market value of $1.80 billion. This discrepancy suggests potential upside if the company's DMD treatment continues to show promise.
InvestingPro Tips highlight that Regenxbio holds more cash than debt on its balance sheet, which could be crucial for funding ongoing clinical trials and potential commercialization of RGX-202. However, the company is quickly burning through cash, a common characteristic of biotech firms in the development stage.
The stock has taken a significant hit recently, with a 13.55% decline in the past week and a 51.61% drop over the last year. This aligns with the analyst's price target adjustment and may reflect market uncertainty about the company's prospects.
For investors seeking a more comprehensive analysis, InvestingPro offers 9 additional tips for Regenxbio, providing a deeper understanding of the company's financial health and market position.
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