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CAMBRIDGE, Mass. - Amylyx Pharmaceuticals, Inc. (NASDAQ: AMLX), a $408 million market cap biotech company whose stock has surged over 160% in the past year, has announced positive 48-week data from its Phase 2 HELIOS trial evaluating AMX0035 for the treatment of Wolfram syndrome, a rare, progressive neurodegenerative disorder. According to InvestingPro analysis, the company currently trades near its Fair Value. The data, presented at a recent congress in Copenhagen, Denmark, suggests sustained stabilization or improvement in disease progression markers, including pancreatic function, glycemic control, vision, and overall symptom burden.
The trial’s findings indicate that AMX0035, an oral combination of sodium phenylbutyrate and taurursodiol, continued to show sustained improvements or stabilization in glycemic control and visual acuity from Week 24 to Week 48. All participants with available measurements met responder criteria, reflecting stability or improvement in Wolfram syndrome-related symptoms. For detailed analysis of Amylyx’s drug development pipeline and market potential, InvestingPro subscribers can access comprehensive research reports and expert insights.
Safety data from the HELIOS trial were consistent with prior studies, with all adverse events being mild or moderate and no serious adverse events related to the treatment reported.
Fumihiko Urano, MD, PhD, Principal Investigator of the HELIOS trial, emphasized the consistency of the Week 48 results across multiple measures of disease progression. Camille L. Bedrosian, MD, Chief Medical Officer at Amylyx, stated that the long-term results reinforce the belief in the potential of AMX0035 to stabilize and improve key manifestations of Wolfram syndrome. With these findings, Amylyx is focused on working with the FDA to inform the design of a Phase 3 trial.
The HELIOS trial, which has received Orphan Drug Designation by the FDA and the European Commission, is designed to evaluate the safety and tolerability of AMX0035, as well as its effects on endocrinological, neurological, and ophthalmological function in adults with Wolfram syndrome.
With a strong Financial Health score of 2.27 (FAIR) and particularly robust cash flow metrics on InvestingPro, Amylyx has reported that it expects its cash runway to extend through the end of 2026. Analysts maintain a Strong Buy consensus with price targets ranging from $7 to $16. The company continues to focus on developing treatments for diseases with high unmet needs, including several neurodegenerative and endocrine diseases.
The information in this article is based on a press release statement from Amylyx Pharmaceuticals.
In other recent news, Amylyx Pharmaceuticals Inc. announced the commencement of a Phase 3 clinical trial for avexitide, aimed at treating post-bariatric hypoglycemia (PBH). The trial, named LUCIDITY, will evaluate the drug’s efficacy and safety in approximately 75 participants who have undergone Roux-en-Y gastric bypass surgery. Avexitide has shown promise in previous trials, earning FDA Breakthrough Therapy and Orphan Drug Designations. Meanwhile, Mizuho analysts upgraded Amylyx’s stock rating from Neutral to Outperform, doubling the price target from $3.00 to $7.00. This upgrade reflects Mizuho’s positive outlook on the company’s focus on avexitide and its research and development pipeline. The analysts have also reintroduced contributions from Amylyx’s AMX0035 for progressive supranuclear palsy and Wolfram Syndrome into their valuation. The shift to a discounted cash flow-based valuation methodology underscores confidence in Amylyx’s financial prospects. These developments indicate a strategic emphasis on advancing treatments for PBH and other conditions.
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