CAMBRIDGE, Mass. - Amylyx Pharmaceuticals Inc. (NASDAQ: AMLX) announced today that interim data from its Phase 2 HELIOS clinical trial suggest that its drug AMX0035 has shown potential benefits in adults with Wolfram syndrome, a rare genetic disorder.
The study included eight participants who have completed 24 weeks of treatment, with results indicating improvements in pancreatic function and glycemic control, as well as some improvements in vision.
The primary efficacy endpoint of the trial focused on changes in C-peptide levels, a marker of pancreatic beta cell function and glycemic control. The interim analysis, based on data as of March 5, 2024, revealed that on average, participants experienced an increase in total C-peptide response, with seven out of eight participants showing a reduction in time to peak C-peptide response, which typically increases due to the disease's progression.
Additionally, the majority of participants reported better control of blood sugar levels, as evidenced by improvements in Hemoglobin A1C (HbA1c) and time in target glucose range. Notably, one participant's vision improved from legally blind to legally sighted.
All eight participants met the prespecified responder criteria, demonstrating either improvement or stabilization of disease according to both the Patient Reported Global Impression of Change (PGIC) and the Clinician Reported Global Impression of Change (CGIC) scales. The drug was generally well-tolerated, with the most common adverse event being diarrhea.
Dr. Fumihiko Urano, Principal Investigator of the HELIOS trial, emphasized the significance of these findings for the Wolfram syndrome community, given the typically worsening outcomes over time. The interim results are seen as encouraging for addressing the urgent need for disease-modifying treatments.
AMX0035 targets endoplasmic reticulum (ER) stress and mitochondrial dysfunction, which are believed to play a role in cell death and neurodegeneration in Wolfram syndrome. The HELIOS trial is part of a collaboration with the Wolfram syndrome community, following strong preclinical data.
Amylyx plans to engage with regulatory authorities to discuss the development path for AMX0035 in Wolfram syndrome. Topline data for all 12 participants at Week 24 is expected in the second half of this year.
The company will present the interim data in a webcast today at 1:30 p.m. Eastern Time. This news is based on a press release statement.
InvestingPro Insights
In light of the recent clinical advancements reported by Amylyx Pharmaceuticals Inc. (NASDAQ: AMLX), the company's financial metrics provide a broader context for investors evaluating its market potential. The current adjusted market capitalization of Amylyx stands at $180.98 million, reflecting the market's valuation of the company in the wake of its ongoing clinical trials and research developments.
With an impressive revenue growth rate of 1612.94% for the last twelve months as of Q4 2023, Amylyx has demonstrated a significant increase in its financial performance. This growth is further highlighted by a quarterly revenue increase of 395.54% in Q4 2023. The company's ability to drastically increase revenue is a positive indicator for potential investors, especially in the context of its recent clinical trial successes.
Amylyx's financial health is underscored by a strong gross profit margin of 59.66% for the same period, suggesting that the company is not only increasing its revenue but also maintaining profitability in its operations. This is a crucial factor for investors considering the long-term sustainability of Amylyx's business model as it continues to invest in research and development.
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