Cambridge, MA-based Amylyx Pharmaceuticals Inc. (NASDAQ:AMLX) today announced encouraging outcomes from its Phase 2 HELIOS trial evaluating AMX0035 for Wolfram syndrome treatment, a rare genetic condition. The trial demonstrated improvements in pancreatic function and other health markers among participants.
The HELIOS study, which included 12 adults, revealed that after 24 weeks of treatment with AMX0035, patients showed an increase in C-peptide response, the study's primary efficacy endpoint, indicating better pancreatic function.
This contrasts with the expected decline due to the disease's progression. Secondary endpoints, such as hemoglobin A1c levels, glucose range, and visual acuity, also showed stabilization or improvements.
In addition to the 24-week data, longer-term results for participants at Week 36 and Week 48 suggested sustained benefits. Notably, the safety profile of AMX0035 was consistent with previous data, with the most common adverse effect being mild or moderate diarrhea and no serious adverse events related to treatment reported.
AMX0035, a combination of sodium phenylbutyrate and taurursodiol, has received Orphan Drug Designation for Wolfram syndrome treatment by the FDA and the European Commission. With these positive findings, Amylyx plans to engage with the FDA and other stakeholders to shape a Phase 3 program, with updates expected in 2025.
The HELIOS trial data is particularly significant for the estimated 3,000 people in the U.S. affected by Wolfram syndrome, a condition that leads to a host of progressive symptoms and complications. Amylyx's efforts could potentially lead to a new treatment option for those affected by this rare disease.
The information in this article is based on a press release statement.
In other recent news, Amylyx Pharmaceuticals disclosed its Q2 2024 financial results, declaring a net loss of $72.7 million. The company also revealed a robust cash position of $309.8 million, which is anticipated to sustain operations until 2026.
A significant development from Amylyx was the acquisition of Avexitide, a treatment for hyperinsulinemic hypoglycemia that has received FDA breakthrough therapy designation. Amylyx plans to initiate Phase 3 development for Avexitide in the coming year.
The company also provided updates on other key programs, including treatments for Alzheimer's disease, axonal degeneration, and various other conditions. In addition to these developments, Amylyx is maintaining a focus on current assets while remaining open to potential pipeline additions.
These are the recent highlights from Amylyx Pharmaceuticals.
InvestingPro Insights
Amylyx Pharmaceuticals' promising HELIOS trial results come at a crucial time for the company. According to InvestingPro data, AMLX has experienced significant revenue growth, with a 55.71% increase in the last twelve months as of Q2 2024. This growth aligns with the company's progress in developing treatments for rare diseases like Wolfram syndrome.
However, investors should note that despite the revenue growth, Amylyx is currently operating at a loss, with an adjusted operating income of -$177.76 million in the same period. This is not uncommon for biotech companies in the development stage, as they often invest heavily in research and clinical trials before achieving profitability.
InvestingPro Tips highlight that Amylyx's stock is trading below its InvestingPro Fair Value of $4.87, suggesting potential upside. Additionally, the company has shown strong price momentum, with a 92.45% price return over the last three months. These factors could indicate growing investor confidence in Amylyx's pipeline and recent trial results.
For those interested in a deeper analysis, InvestingPro offers 11 additional tips for Amylyx Pharmaceuticals, providing a more comprehensive view of the company's financial health and market position.
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