On Friday, Regenxbio Inc. (NASDAQ:RGNX) stock maintained its Buy rating and a $38.00 price target from H.C. Wainwright. This affirmation follows the company's recent announcement regarding the progress of its drug candidate RGX-121 for the treatment of mucopolysaccharidosis type II (MPS II), also known as Hunter Syndrome.
Earlier in the week, Regenxbio completed a pre-Biologics License Application (BLA) meeting with the U.S. Food and Drug Administration (FDA). The FDA has agreed with Regenxbio's proposal to use cerebrospinal fluid levels of heparan sulfate D2S6 as a surrogate endpoint, which could potentially support the accelerated approval of RGX-121.
The company is preparing to commence a rolling submission of the BLA in the third quarter of 2024. Additionally, an FDA inspection of Regenxbio's manufacturing innovation center is anticipated in the first half of 2025. The firm has confirmed the comparability of its commercial bulk drug and clinical trial material, produced using its proprietary NAVXpress platform process.
A confirmatory trial for RGX-121 is expected to start enrolling patients in the second half of 2025, ahead of the potential accelerated approval by the FDA. The pivotal phase of the CAMPSIITE trial has already met its primary endpoint, demonstrating a significant decrease in CSF levels of D2S6 after 16 weeks of treatment with RGX-121.
The successful development and potential approval of RGX-121 could also result in Regenxbio receiving a Priority Review Voucher, which is valued at approximately $100 million. This milestone, coupled with the drug's progress, underpins H.C. Wainwright's reiterated Buy rating and price target for Regenxbio's stock.
In other recent news, Regenxbio Inc. has made significant strides in its operations. The company 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), also known as Hunter syndrome. This submission process is expected to commence in the third quarter of 2024.
RBC Capital has maintained its Outperform rating on Regenxbio's stock, citing the company's strategic positioning in the market. The firm noted that regardless of potential outcomes from the upcoming PDUFA decision for a competing drug by Sarepta Therapeutics (NASDAQ:SRPT), Regenxbio is well-positioned to enter the market.
Regenxbio has also announced a leadership transition, with Curran Simpson stepping into the role of President and Chief Executive Officer. The company maintains its financial outlook, projecting operational funding into 2026.
H.C. Wainwright, an analyst firm, recently reaffirmed a Buy rating on Regenxbio's stock and increased the price target to $38.00. The company reported a productive first quarter with total revenue of $15.6 million and a net loss of $63.3 million, a decrease from the estimated $70.7 million loss.
These are the latest developments in the company's operations, providing investors with a glimpse into the company's recent activities and future plans.
InvestingPro Insights
As Regenxbio Inc. (NASDAQ:RGNX) advances its drug candidate RGX-121, it's crucial for investors to monitor the company's financial health and market performance. According to InvestingPro data, Regenxbio currently holds a market capitalization of $585.16 million, with a Price to Book ratio over the last twelve months as of Q1 2024 at 1.5. Despite challenges, the company's liquid assets surpass its short-term obligations, which is a positive sign for its financial stability.
InvestingPro Tips indicate that Regenxbio's stock is in oversold territory based on its Relative Strength Index (RSI), which could suggest a potential rebound. Additionally, the company is trading near its 52-week low. For investors considering a deeper analysis, there are additional InvestingPro Tips available, which can be found at: https://www.investing.com/pro/RGNX. These insights may help in assessing the risk and opportunity associated with Regenxbio's stock, especially in light of its recent developments.
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