On Thursday, H.C. Wainwright maintained a Buy rating and a stock price target of $39.00 on Regenxbio Inc. (NASDAQ:RGNX), following the announcement of new long-term data from the CAMPSIITE trial of RGX-121. The study, which evaluates the treatment for MPS II or Hunter syndrome, showed an 85% median reduction in cerebrospinal fluid levels of heparan sulfate D2S6, approaching normal levels and sustaining for up to two years.
The pivotal phase of the CAMPSIITE trial met its primary endpoint with RGX-121 treatment resulting in decreased levels of D2S6 below the maximum attenuated disease levels at 16 weeks, with a statistically significant p-value of 0.00016. Moreover, 80% of patients at the pivotal dose level were enzyme replacement therapy-free at their last time point, recorded up to more than 18 months post-dosing.
The trial, which included 25 patients, found RGX-121 to be well tolerated. The most common treatment-emergent adverse events (TEAEs) were vomiting, experienced by 73.3% of participants, and symptoms like pyrexia, cough, and gastroenteritis in 60% of participants. The majority of these TEAEs were classified as mild to moderate in severity, and no TEAEs led to discontinuation of the study.
Regenxbio's management has indicated that the submission of a rolling Biologics License Application (BLA) is on track for the third quarter of 2024. A confirmatory trial is anticipated to begin enrollment in the second half of 2025, ahead of potential FDA approval under the accelerated approval pathway. The firm also highlighted the possibility of Regenxbio receiving a Priority Review Voucher following potential approval of RGX-121.
The reiteration of the Buy rating and price target by H.C. Wainwright reflects the firm's positive outlook on Regenxbio's prospects following the recent trial results. The company's progress towards regulatory approval appears to be moving forward as planned, with key milestones expected in the coming years.
In other recent news, REGENXBIO Inc. has made significant strides in its gene therapy programs. The biotechnology firm's CAMPSIITE trial of RGX-121, a potential gene therapy for Mucopolysaccharidosis Type II (MPS II), also known as Hunter syndrome, showed promising results.
The data revealed sustained reductions in a key biomarker of brain disease and a significant number of patients being able to discontinue standard enzyme replacement therapy. The company is preparing for a rolling Biologics License Application (BLA) submission in the third quarter of 2024, which could lead to a Priority Review Voucher in 2025.
In addition, REGENXBIO reported a solid financial position during its Q2 2024 earnings call, with $327 million in cash, cash equivalents, and marketable securities, which is expected to support operations into 2026. The company also discussed advancements in other priority programs, including gene therapies targeting Duchenne muscular dystrophy (DMD), wet age-related macular degeneration (wet AMD (NASDAQ:AMD)), and diabetic macular edema (DME).
These are recent developments that highlight the company's progress in gene therapy research and development. The firm's commitment to advancing its gene therapy pipeline is underscored by strong clinical data and strategic regulatory plans. However, it's important to note that these findings and plans are based on the company's own statements and the analysis of independent analysts.
InvestingPro Insights
The latest insights from InvestingPro reveal a mixed financial landscape for Regenxbio Inc. (NASDAQ:RGNX). On the positive side, the company holds more cash than debt on its balance sheet, which may provide a cushion for operational needs and investment in research and development. Moreover, liquid assets exceed short-term obligations, suggesting a degree of financial stability in the near term. This financial positioning is crucial as the company advances towards the submission of a rolling Biologics License Application for RGX-121.
However, the financial metrics also highlight some challenges. Regenxbio is not currently profitable, and analysts do not expect profitability within this year. The company's gross profit margin is notably weak, with a negative margin over the last twelve months as of Q2 2024. The stock has experienced a significant decline over the past six months, reflecting investor concerns. These financial considerations are essential for investors to weigh against the clinical progress and long-term potential of RGX-121.
For those seeking a deeper dive into Regenxbio's financial health, InvestingPro offers additional insights and metrics. There are currently 8 more InvestingPro Tips available on their platform, which can provide investors with a more comprehensive understanding of the company's financial and operational standing. Visit InvestingPro for Regenxbio for further details and to inform your investment decisions.
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