Amylyx Pharmaceuticals Stock Price, News & Analysis

Amylyx Pharmaceuticals (NASDAQ: AMLX) announced positive topline results from its Phase 2 HELIOS clinical trial of AMX0035 in adults with Wolfram syndrome. The trial showed improvement in pancreatic function after 24 weeks of treatment, contrary to expected disease progression. Improvements or stabilization were observed across all secondary endpoints, including glycemic control, vision, and patient- and clinician-reported impressions of disease burden.

Key findings include:

• Sustained improvement over time in longer-term data for participants completing Week 36 and Week 48 assessments
• AMX0035 was generally well-tolerated
• Amylyx plans to meet with the FDA to inform a Phase 3 program, with an update expected in 2025

The results suggest AMX0035 has the potential to favorably change the trajectory of Wolfram syndrome, a rare, progressive disease with no approved treatment options.

Amylyx Pharmaceuticals (Nasdaq: AMLX) has announced a virtual webcast on October 17, 2024 at 1:30 p.m. ET to discuss topline results from the HELIOS Phase 2 trial of AMX0035 for the investigational treatment of Wolfram syndrome. The webcast will feature management and Dr. Fumihiko Urano, the principal investigator of the HELIOS trial and a professor at Washington University School of Medicine in St. Louis.

The presentation follows the release of data at the International Society for Pediatric and Adolescent Diabetes 50th Annual Congress in Lisbon, Portugal. The live webcast will be accessible on the company's website and available for replay for 90 days. Dr. Urano, a leading expert in Wolfram syndrome, directs the Wolfram Syndrome Clinic and International Registry & Clinical Study at Washington University.

Amylyx Pharmaceuticals (Nasdaq: AMLX) has announced its participation in three upcoming investor conferences in New York City:

• Morgan Stanley 22nd Annual Global Healthcare Conference on September 5, 2024, at 1:50pm ET
• Baird 2024 Global Healthcare Conference on September 10, 2024, at 3:45pm ET
• H.C. Wainwright 26th Annual Global Investment Conference on September 11, 2024, at 8:30am ET

Live webcasts of these events will be available on the company's website under the 'Events and Presentations' section. The recordings will remain accessible for 90 days after each event, providing investors with extended access to Amylyx's presentations and discussions.

Amylyx Pharmaceuticals (Nasdaq: AMLX) has published exploratory analyses from the Phase 2 PEGASUS trial, showing encouraging effects of AMX0035 on cerebrospinal fluid (CSF) biomarkers in Alzheimer's disease (AD) patients. The study, published in Alzheimer's & Dementia: Translational Research & Clinical Interventions, suggests that AMX0035 engages multiple pathological pathways related to neurodegeneration.

Key findings include:

• Reduced levels of p-tau181 and total tau compared to placebo
• Decreased levels of synaptic and neuronal degeneration biomarkers (neurogranin and FABP3)
• Reduced levels of YKL-40, a biomarker associated with cortical volume loss and cognitive decline

These results support the potential of AMX0035 in treating neurodegenerative diseases associated with tau dysfunction, including progressive supranuclear palsy (PSP), for which the ORION trial is ongoing.

Amylyx Pharmaceuticals (Nasdaq: AMLX) reported Q2 2024 financial results.

The company expanded its pipeline with avexitide, a Phase 3-ready GLP-1 receptor antagonist expected to enter Phase 3 in Q1 2025 for post-bariatric hypoglycemia (PBH) and congenital hyperinsulinism.

Updated Phase 2 HELIOS trial data for AMX0035 in Wolfram syndrome will be presented at ISPAD 2024.

Amylyx plans a multiple ascending dose trial of AMX0114 for ALS in H2 2024 and expects interim data for AMX0035 in progressive supranuclear palsy by mid-2025.

Financially, Q2 net product revenue was $(1.0) million due to adjustments, down from $98.2 million in Q2 2023. Cost of sales rose to $7.4 million. R&D expenses decreased to $23.3 million, SG&A expenses fell to $21.6 million, but restructuring expenses reached $22.9 million. Amylyx posted a net loss of $72.7 million, compared to a net income of $22.1 million in Q2 2023.

Cash and equivalents were $309.8 million, projecting a runway into 2026.

Amylyx Pharmaceuticals (Nasdaq: AMLX) has announced that it will report its second quarter 2024 financial results on Thursday, August 8, 2024. The company's senior management team will host a conference call and audio webcast at 8:00 a.m. ET to discuss the financial results and provide company updates. Investors and interested parties can access the call by dialing +1 (800)-836-8184 (U.S. & Canada) or +1 (646)-357-8785 (international). A live audio webcast will also be available on the company's website under the Investor section. The webcast will be archived and available for replay for 90 days following the event.

Amylyx Pharmaceuticals (NASDAQ: AMLX) has received Orphan Drug Designation from the European Commission for AMX0035 to treat Wolfram syndrome. This follows a similar designation by the FDA in 2020. Wolfram syndrome is a rare, progressive disease characterized by childhood-onset diabetes and neurodegeneration, with no approved treatments. Interim data from the Phase 2 HELIOS trial showed AMX0035 improved pancreatic function and glycemic control in 8 participants at Week 24. The drug was generally well-tolerated, with patients reporting disease improvement or stabilization. Topline data for all 12 participants is expected in fall 2024.

Amylyx Pharmaceuticals has acquired avexitide, a first-in-class GLP-1 receptor antagonist, from Eiger BioPharmaceuticals for $35.1 million. Avexitide is designed to treat hyperinsulinemic hypoglycemia and has received FDA Breakthrough Therapy Designation for post-bariatric hypoglycemia (PBH) and congenital hyperinsulinism (HI). The Phase 3 trial for PBH is set to begin in Q1 2025, with results expected in 2026. Two Phase 2 trials demonstrated significant reductions in hypoglycemic events, and avexitide showed a favorable safety profile. Amylyx will incorporate avexitide into its existing pipeline, bolstering its focus on treating endocrine and neuroscience disorders.

Amylyx Pharmaceuticals (NASDAQ: AMLX) announced that it will host a conference call and webcast on Wednesday, July 10, 2024, at 8:00 a.m. ET to discuss its acquisition of avexitide from Eiger BioPharmaceuticals.

The conference call can be accessed by dialing +1 (800) 836-8184 in the U.S. and Canada or +1 (646) 357-8785 internationally, 10 minutes before the start time, and by asking to join the Amylyx Pharmaceuticals call.

A live audio webcast will be available in the Investor section of the Amylyx website and can be replayed for 90 days post-event.