ROCKVILLE, Md. - REGENXBIO Inc. (NASDAQ:RGNX), a biotechnology firm, today announced the expansion of its Phase I/II AFFINITY DUCHENNE trial. The trial now includes a new cohort for boys aged 1-3 with Duchenne muscular dystrophy (Duchenne) to assess the investigational gene therapy RGX-202.
RGX-202 is designed to deliver a novel microdystrophin gene, which includes the C-Terminal domain, aiming to closely mimic the naturally occurring dystrophin gene. The trial's expansion will enroll up to five patients in this age group at the pivotal dose level.
An end-of-Phase II meeting with the FDA is scheduled for late July to finalize the pivotal trial design, with the company planning to include data from all patients dosed at level 2 in its Biologics License Application (BLA) submission. REGENXBIO anticipates using the accelerated approval pathway, citing an ongoing unmet need for differentiated treatments in Duchenne.
The company remains on track to initiate the pivotal trial between late Q3 and early Q4 of 2024, with the goal of expediting the development of RGX-202 towards commercialization. Initial strength and functional assessment data from the trial are expected in the second half of 2024.
The AFFINITY DUCHENNE trial has been designed in consultation with the Duchenne community, including a prophylactic immunosuppression regimen to mitigate potential immune responses. The trial will assess safety, tolerability, and efficacy of a one-time intravenous dose of RGX-202 in patients aged 1-11 with Duchenne.
RGX-202 has demonstrated a strong safety profile and positive microdystrophin data in the trial so far, with no drug-related serious adverse events reported in five patients aged 4.4 to 12.1. Early results indicate increases in RGX-202 microdystrophin expression and reductions in serum CK levels, suggesting clinical improvement.
Duchenne is a severe muscle disease affecting 1 in every 3,500 to 5,000 male births annually. It leads to progressive muscle degradation, loss of movement, and premature death.
This news is based on a press release statement from REGENXBIO Inc.
In other recent news, Regenxbio Inc. has seen significant advancements in its operations. The company's drug candidate RGX-121 for the treatment of mucopolysaccharidosis type II (MPS II) has made progress, with H.C. Wainwright maintaining a Buy rating and a $38.00 price target. The FDA agreed with Regenxbio's proposal to use cerebrospinal fluid levels of heparan sulfate D2S6 as a surrogate endpoint, potentially supporting the accelerated approval of RGX-121.
Regenxbio also announced a leadership transition, with Curran Simpson assuming the role of President and Chief Executive Officer. Despite potential outcomes from the upcoming PDUFA decision for a competing drug by Sarepta Therapeutics (NASDAQ:SRPT), RBC Capital maintained its Outperform rating on Regenxbio's stock, citing the company's strategic positioning in the market.
Regenxbio 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. The company's CAMPSIITE trial successfully met its primary endpoint, leading to a significant reduction in cerebrospinal fluid levels of D2S6 at 16 weeks with RGX-121 treatment.
These are the latest developments in the company's operations.
InvestingPro Insights
As REGENXBIO Inc. (NASDAQ:RGNX) continues to advance its gene therapy RGX-202 through clinical trials, investors and stakeholders are closely monitoring the company's financial and operational metrics. InvestingPro data reveals key insights into the company's market position and financial health that could influence its ability to sustain research and development efforts.
InvestingPro Data indicates that REGENXBIO holds a market capitalization of $592.06 million USD, which reflects the market's valuation of the company in light of its current and future prospects. Despite the company's innovative strides in gene therapy, its P/E Ratio stands at -2.06, and the adjusted P/E Ratio for the last twelve months as of Q1 2024 is -2.32, suggesting that the company is not currently profitable. Furthermore, the Revenue Growth for the same period shows a decline of -20.9%, highlighting challenges in generating increased revenue streams.
Among the InvestingPro Tips, two particularly relevant points for investors to consider are:
1. REGENXBIO's balance sheet shows more cash than debt, providing a cushion for ongoing research and operational expenses. This is a positive sign for the company's financial stability and its ability to fund future development phases of RGX-202.
2. The Relative Strength Index (RSI) suggests the stock is in oversold territory. This technical indicator might interest potential investors looking for entry points, especially considering the company's strategic efforts to bring RGX-202 to market.
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