Amylyx Pharmaceuticals
🇺🇸United States
- Country
- 🇺🇸United States
- Ownership
- Public
- Established
- 2014-01-01
- Employees
- 384
- Market Cap
- $145M
- Website
- http://www.amylyx.com
Pharma Leadership Shuffle: Key Executive Moves in Early 2025 Reshape Industry Landscape
• The pharmaceutical industry has experienced significant leadership changes in early 2025, with major transitions at companies including Pfizer, Boehringer Ingelheim, and Takeda.
• Former FDA CDER Director Patricia Cavazzoni's move to Pfizer as Chief Medical Officer has sparked "revolving door" criticisms, highlighting ongoing concerns about regulatory-industry transitions.
• Several biotechnology firms have strengthened their executive teams with specialized expertise in clinical development, particularly in areas like stroke treatment, antibody-drug conjugates, and neuropsychiatric therapies.
Eli Lilly and Alchemab Forge $415M Partnership for Novel ALS Antibody Therapy
• Eli Lilly has entered a $415 million licensing agreement with UK-based Alchemab Therapeutics for ATLX-1282, a first-in-class antibody therapy for amyotrophic lateral sclerosis (ALS).
• The novel antibody was discovered using Alchemab's AI-powered platform that identified protective antibodies in individuals with genetic mutations for frontotemporal dementia (FTD) who remained healthy into old age.
• Alchemab will conduct early Phase 1 trials before Lilly takes over further development and commercialization, leveraging its expertise in neurological conditions to advance the therapy.
FDA Approves VALTOCO Nasal Spray for Seizure Clusters in Children Ages 2-5
• The FDA has approved Neurelis' VALTOCO (diazepam nasal spray) for treating seizure clusters in patients as young as 2 years old, expanding its previous indication to include younger children.
• Clinical trials demonstrated VALTOCO's safety and efficacy in children aged 2-5, with 31 of 35 enrolled patients completing the 180-day safety period and no treatment-related serious adverse events reported.
• The nasal spray formulation provides a significant alternative to rectal diazepam, addressing a major unmet need for caregivers managing seizures in young children.
Plus Therapeutics Receives FDA Orphan Drug Designation for Novel Lung Cancer Metastasis Treatment
• Plus Therapeutics has secured FDA Orphan Drug Designation for Rhenium (186Re) Obisbemeda, targeting leptomeningeal metastases in lung cancer patients.
• The designation marks a significant milestone in addressing the critical unmet need for effective treatments for lung cancer that has spread to the central nervous system.
• The company's stock responded positively to the announcement, with shares surging 71% following the FDA's decision.
FDA Grants Historic Approval to Biogen's Qalsody for Rare Genetic Form of ALS
• Biogen's Qalsody (tofersen) receives FDA accelerated approval as the first therapy for SOD1-mutated ALS, marking a significant breakthrough in genetic-specific ALS treatment.
• The approval is based on the drug's ability to reduce neurofilament light chain (NfL) biomarker levels by 55% over 28 weeks, representing the first use of a blood biomarker for neurological drug approval.
• Biogen plans to launch Qalsody within a week, with patient out-of-pocket costs capped at $50 per month, while conducting the confirmatory ATLAS trial through 2027.
Coya Therapeutics Advances Treg-Enhancing Therapy for ALS into Phase 2b Trial
• Coya Therapeutics is set to initiate a Phase 2b trial for Coya-302 in 2025, an innovative approach to treating amyotrophic lateral sclerosis (ALS) by enhancing regulatory T cells (Tregs).
• Coya-302 aims to reduce neuroinflammation, a key factor in ALS progression, potentially slowing down the disease via a combination therapy approach.
• With recent financial backing and a commercialization partnership with Dr. Reddy’s, Coya is well-positioned to advance its pipeline and address the significant unmet need in the ALS market.
• Coya's broader pipeline includes preclinical Treg-based therapies for Alzheimer’s and Parkinson’s diseases, highlighting its focus on addressing neuroinflammation across multiple neurodegenerative conditions.
FDA Lifts Clinical Hold on Amylyx's AMX0114 Phase 1 ALS Trial, Paving Way for LUMINA Study
• The FDA has lifted the clinical hold on Amylyx Pharmaceuticals' Phase 1 trial of AMX0114, an antisense oligonucleotide for ALS treatment.
• Amylyx is now cleared to initiate screening and enrollment at U.S. sites for the Phase 1 LUMINA trial, expected to begin in Canada in early 2025.
• The LUMINA trial will assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of AMX0114 in 48 adults with ALS.
• AMX0114 targets calpain-2, a protein involved in axonal degeneration, with preclinical data showing improved neuronal survival.
FDA Places Clinical Hold on Atara Biotherapeutics' Cell Therapy Programs Due to Manufacturing Concerns
• The FDA has placed a clinical hold on Atara Biotherapeutics' EBVALLO (tabelecleucel) program and ATA3219, pausing new patient enrollment.
• The hold is linked to GMP compliance issues at a third-party manufacturing facility, as identified in the Complete Response Letter for EBVALLO.
• Existing patients who may benefit clinically can continue treatment, while Atara collaborates with the FDA to resolve the manufacturing issues.
• Atara is working with the FDA to address the issues and aims to resubmit the BLA for EBVALLO, potentially securing approval within six months of resubmission.
Denali Therapeutics' DNL343 Fails to Meet Primary Endpoint in HEALEY ALS Platform Trial
• Denali Therapeutics' DNL343, an eIF2B agonist, did not meet the primary endpoint of slowing ALS disease progression in a Phase 2/3 trial.
• The HEALEY ALS Platform Trial's Regimen G assessed DNL343's impact on disease severity and survival over 24 weeks compared to placebo.
• While DNL343 was safe and well-tolerated, key secondary endpoints like muscle strength and respiratory function showed no significant difference.
• Further analyses, including biomarker assessments, are planned for 2025 to explore potential subgroup benefits and long-term effects.
Amylyx Pharmaceuticals Appoints Dan Monahan as Chief Commercial Officer to Spearhead Avexitide Launch
• Amylyx Pharmaceuticals has appointed Dan Monahan as Chief Commercial Officer to lead the commercialization of their product portfolio.
• Monahan will focus on the potential approval and launch of avexitide, a GLP-1 receptor antagonist for post-bariatric hypoglycemia (PBH).
• Amylyx plans to initiate the Phase 3 LUCIDITY trial of avexitide in Q1 2025, with data expected in 2026, to address the unmet need for PBH treatment.
• Monahan brings over 20 years of experience in the biopharmaceutical industry, including successful launches at Otsuka, Novartis, and Sanofi.
Top Neurology Podcasts of 2024 Highlight Breakthroughs in MS, Alzheimer's, and ALS Research
NeurologyLive's Mind Moments podcast featured groundbreaking discussions on updated MS diagnostic criteria, advances in Alzheimer's treatment, and developments in ALS therapeutics throughout 2024. The most popular episodes covered critical topics including the new McDonald criteria for MS diagnosis, emerging RNA therapeutics for Alzheimer's disease, and insights from the PHOENIX trial in ALS.
PrimeC Shows Promise in ALS Treatment: 53% Extension in Complication-Free Survival in Phase 2b Trial
NeuroSense's PrimeC demonstrated significant benefits in ALS treatment during a Phase 2b clinical trial, extending complication-free survival by 53% compared to placebo. The therapy also showed improvements in patients' quality of life, leading to discussions with U.S. regulators about Phase 3 trials and potential early approval in Canada.
Dazucorilant Fails Primary Endpoint in Phase 2 ALS Trial but Shows Survival Benefit
• Corcept Therapeutics' dazucorilant did not meet the primary endpoint in the DAZALS Phase 2 trial for amyotrophic lateral sclerosis (ALS).
• The study, involving 249 patients, aimed to slow the decline in motor skills as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R).
• A statistically significant survival benefit was observed in the 300 mg dazucorilant arm compared to placebo (p-value: 0.02) over the 24-week study.
• An open-label extension study is ongoing to assess long-term survival, with results expected in March 2025, and complete DAZALS results will be presented at a future medical conference.
Pharmaceutical R&D Highlights of 2024: GLP-1s, CRISPR, and More
• GLP-1 receptor agonists like semaglutide and tirzepatide showed potential beyond diabetes and weight loss, including cardiovascular and mental health benefits.
• CRISPR gene editing entered the market with Casgevy for sickle cell disease and β-thalassemia, though uptake has been slow due to complex treatment requirements.
• Protein-folding algorithms developed by Nobel laureates revolutionized biochemical research and found applications in pharmaceutical drug discovery.
• Antibody-drug conjugates (ADCs) saw major investment, with firms focusing on novel linker chemistries to enhance their efficacy and target specificity.
Amylyx Announces Phase 3 LUCIDITY Trial for Avexitide in Post-Bariatric Hypoglycemia
• Amylyx Pharmaceuticals initiates Phase 3 LUCIDITY trial for avexitide, a GLP-1 receptor antagonist, targeting post-bariatric hypoglycemia (PBH).
• The trial aims to reduce hypoglycemia events, with participant dosing expected in Q1 2025 and topline results anticipated in 2026.
• LUCIDITY is a multicenter, randomized, double-blind, placebo-controlled study involving 75 participants across 20 U.S. sites.
• Avexitide has received FDA Breakthrough Therapy and Orphan Drug Designations, highlighting the unmet need in PBH treatment.
Amylyx Pharmaceuticals Advances Pipeline with Positive Data and Strategic Acquisitions
• Amylyx reported positive topline data from its Phase 2 HELIOS trial of AMX0035 in Wolfram syndrome, showing improvement or stabilization across key disease measures.
• The company is on track to initiate a Phase 3 program for avexitide in post-bariatric hypoglycemia (PBH) in Q1 2025, following its acquisition and Breakthrough Therapy Designation.
• Amylyx plans to begin the Phase 1 LUMINA clinical trial of AMX0114 in people living with ALS by the end of 2024 or in early 2025 in Canada.
• With $234.4 million in cash reserves, Amylyx anticipates funding operations into 2026, supporting its pipeline development in neurodegenerative diseases and endocrine conditions.
Alzheimer's Clinical Trial Update: New Data on Tau-Targeting Drugs and More
• Eisai presented Phase 1/2 data on E2814, an anti-tau drug, showing reduced levels of disease biomarkers in participants with mild to moderate genetic Alzheimer’s.
• Roche terminated its agreement with UCB for beprenemab, an experimental tau-targeting antibody, after Phase 2 trial results showed it did not slow cognitive decline.
• A Phase 2 trial of intranasal insulin and empagliflozin showed that insulin alone was associated with a small improvement in cognitive test performance in early Alzheimer’s patients.
AMX0035 Shows Sustained Improvements in Wolfram Syndrome Patients in Phase 2 Trial
• Amylyx Pharmaceuticals' AMX0035 demonstrated sustained improvements in pancreatic function in adults with Wolfram syndrome in a Phase 2 trial.
• The HELIOS trial showed improvements in C-peptide response, glycemic control, and visual acuity over 24 weeks of treatment with AMX0035.
• Longer-term data up to 48 weeks indicated sustained improvements in C-peptide and HbA1c levels, suggesting potential for disease trajectory modification.
• AMX0035 was generally well-tolerated, with a safety profile consistent with prior data, supporting further investigation in a Phase 3 program.
Intellia Therapeutics Initiates Phase 3 Trial of NTLA-2002 for Hereditary Angioedema
• Intellia Therapeutics has dosed the first patient in its Phase 3 HAELO trial evaluating NTLA-2002 for hereditary angioedema (HAE).
• The HAELO trial is a global, randomized, double-blind, placebo-controlled study involving 60 adults with Type I or Type II HAE.
• NTLA-2002, a single-dose CRISPR-based therapy, targets the _KLKB1_ gene to reduce plasma kallikrein activity and prevent HAE attacks.
• Intellia anticipates completing enrollment in the second half of 2025 and plans for a potential U.S. launch in 2027, pending regulatory approval.
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