ROCKVILLE, Md. - REGENXBIO Inc. (NASDAQ:RGNX) today announced positive interim data from its Phase I/II trial for RGX-202, a gene therapy candidate for Duchenne muscular dystrophy (Duchenne). The treatment has shown high levels of microdystrophin expression in young patients, which is believed to be a crucial factor in alleviating the disease's progression.
The investigational one-time therapy, designed to deliver a novel microdystrophin gene, has demonstrated a clear dose response, with patients aged 5.8 and 8.5 showing expression levels at 77.2% and 46.5% of control, respectively, three months post-dosing. These results are consistent across all age groups treated and support the company's plans for seeking accelerated approval.
The AFFINITY DUCHENNE trial has reported no serious adverse events as of July 8, 2024. Seven patients who completed three-month assessments exhibited meaningful increases in microdystrophin expression and a reduction in serum creatinine kinase levels, suggesting clinical improvement.
REGENXBIO's proprietary suspension-based manufacturing process, NAVXpress™, used for producing RGX-202 at the company's Manufacturing Innovation Center, has been cited as capable of meeting full market demand with an annual production capacity of up to 2,500 doses.
The company anticipates completing enrollment for the dose level 2 expansion cohort in early Q3 2024 and has begun enrolling boys aged 1-3. The pivotal trial is expected to commence in Q4 2024, with initial strength and functional assessment data for both dose levels due in the second half of 2024.
RGX-202 is the only gene therapy in advanced development for Duchenne that encodes for the C-Terminal domain of dystrophin, which is associated with improved muscle repair and protection from contraction-induced stress. This therapy aims to address the genetic root of Duchenne, a severe muscle disease affecting 1 in 3,500 to 5,000 boys born worldwide, leading to muscle degeneration, loss of movement, and premature death.
This article is based on a press release statement from REGENXBIO Inc.
In other recent news, Regenxbio Inc. has made significant strides in its operations. The company's drug candidate RGX-121 for the treatment of mucopolysaccharidosis type II (MPS II) has made progress, with the FDA agreeing to use cerebrospinal fluid levels of heparan sulfate D2S6 as a surrogate endpoint. This could potentially support the accelerated approval of RGX-121.
Moreover, Regenxbio reported a total revenue of $15.6 million and a net loss of $63.3 million for the first quarter, a decrease from the estimated $70.7 million loss.
In the field of ocular gene therapy, the company has seen advancements and has also announced the expansion of its Phase I/II AFFINITY DUCHENNE trial for boys aged 1-3 with Duchenne muscular dystrophy. RBC Capital and H.C. Wainwright have maintained their positive ratings on the company's stock, noting its strategic positioning in the market and potential in the Duchenne muscular dystrophy market.
The company has also undergone a leadership transition with Curran Simpson assuming the role of President and Chief Executive Officer. These are recent developments at Regenxbio Inc., providing investors with a glimpse into the company's recent activities and future plans.
InvestingPro Insights
As REGENXBIO Inc. (NASDAQ:RGNX) continues to progress in its clinical trials for RGX-202, its financial health and market performance provide additional context for investors. Notably, the company holds more cash than debt on its balance sheet, which is a positive sign for its financial stability. This is complemented by the fact that REGENXBIO's liquid assets exceed its short-term obligations, suggesting a robust short-term financial position.
Still, investors should be aware of some challenges. Analysts have revised their earnings predictions downwards for the upcoming period, indicating potential concerns about the company's future earnings capacity. Moreover, REGENXBIO has been quickly burning through cash, which could raise questions about its long-term financial sustainability, especially given that analysts do not expect the company to be profitable this year.
From a market perspective, REGENXBIO has experienced a strong return over the last month, yet it's important to consider this in the context of its overall performance, which includes a -25.59% return over the past year. The company's market capitalization currently stands at 701.9 million USD, and it has a negative price-to-earnings (P/E) ratio of -2.52, reflecting its lack of profitability in the last twelve months.
For investors seeking a more comprehensive analysis, there are additional InvestingPro Tips available for REGENXBIO, which can offer deeper insights into the company's financial health and market potential. These tips can be accessed at InvestingPro.
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