On Thursday, Stifel maintained a Buy rating on Regenxbio Inc. (NASDAQ:RGNX) and increased the price target to $40 from the previous $35. This adjustment comes after the latest updates from the company's AFFINITY-DUCHENNE Phase 1/2 trial for RGX-202, a potential treatment for Duchenne muscular dystrophy.
The recent data from the trial revealed a significant step-up in mean expression levels, especially from Dose Level 2, where RGX-202 showed a 75.7% expression in a 12.1-year-old patient. There was also a notable reduction in creatinine kinase, a marker of muscle injury, by 77%, alongside early indications of strength and functional improvements.
The safety profile of RGX-202 remains solid, with follow-ups ranging from seven weeks to 11 months, and most patients have completed their post-administration prophylactic regimens. These findings are particularly promising as they demonstrate RGX-202's unique ability to target muscles and generate strong microdystrophin expression across a broad patient demographic, including older patients who may have more advanced disease progression.
Regenxbio plans to finalize the Dose Level 2 results in the second quarter of 2024. The company aims to determine the appropriate dose for pivotal studies by mid-2024, using microdystrophin expression as a basis for a Biologics License Application (BLA) filing. This timeline suggests a significant advancement in the development of RGX-202 for treating Duchenne muscular dystrophy.
InvestingPro Insights
Following the positive outlook from Stifel on Regenxbio Inc. (NASDAQ:RGNX), InvestingPro data and insights offer a deeper financial perspective on the company's current status. With a market capitalization of approximately $1.04 billion, Regenxbio is navigating through challenging financial metrics. The company's revenue in the last twelve months as of Q4 2023 stood at $90.24 million, which reflects a concerning decline of nearly 20%. Moreover, the gross profit margin during the same period was significantly negative at -195.29%, indicating operational challenges.
Despite the promising clinical trial results for RGX-202, InvestingPro Tips highlight several areas of financial concern. Regenxbio is quickly burning through cash, and analysts have recently revised their earnings expectations downwards for the upcoming period. Additionally, the company's stock appears to be in overbought territory according to the Relative Strength Index (RSI). However, it's worth noting that the company has seen a strong return over the past week, month, and three months, with price upticks indicating investor optimism in the short term.
Investors interested in the detailed financial health and future projections for Regenxbio can access further InvestingPro Tips at https://www.investing.com/pro/RGNX. There are 12 additional tips available on InvestingPro, and users can apply the coupon code PRONEWS24 to receive an extra 10% off a yearly or biyearly Pro and Pro+ subscription. This offer provides an opportunity for investors to gain comprehensive insights into Regenxbio's financials and make informed decisions based on a blend of clinical progress and financial analysis.
This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.