Latest news with #Amylyx
Globe and Mail
4 days ago
- Health
- Globe and Mail
Amyotrophic Lateral Sclerosis Pipeline Insight 2025: 75+ Companies Accelerating Innovation Across Genetic and Neuroinflammatory Targets
The ALS pipeline is gaining momentum, with over 75 companies advancing R&D across key targets like SOD1 mutations, TDP-43, C9orf72 expansions, and neuroinflammation. Approaches include ASOs, gene therapies, mitochondrial modulators, neuroprotective agents, and immune biologics. A major milestone was the FDA's 2023 accelerated approval of Biogen and Ionis's Tofersen for SOD1-ALS. DelveInsight's ' Amyotrophic Lateral Sclerosis (ALS) – Pipeline Insight, 2025 ' offers a detailed analysis of the evolving therapeutic landscape for ALS, a fatal neurodegenerative disease characterized by progressive loss of motor neurons, leading to muscle weakness, paralysis, and eventually respiratory failure. With most patients succumbing to the disease within 3–5 years of symptom onset, there remains a critical unmet need for effective disease-modifying treatments. Several late-stage candidates are progressing through global Phase II/III studies, including Amylyx's AMX0035, which received full FDA approval in September 2022 as RELYVRIO, and is now being evaluated in confirmatory trials. Additionally, investigational programs from companies like Clene Nanomedicine, QurAlis, Wave Life Sciences, and BrainStorm Cell Therapeutics are advancing promising assets in both sporadic and familial ALS subpopulations, often with orphan drug or fast-track designations. The regulatory landscape continues to support innovation in ALS with flexible pathways and growing biomarker guidance, including neurofilament light chain (NfL) levels, digital endpoints, and functional scores. Patient advocacy, real-world evidence generation, and adaptive trial designs are also helping de-risk development strategies and accelerate access. As 2025 unfolds, the ALS pipeline reflects a shift from symptomatic care to precision medicine and disease modification, driven by robust collaboration among academia, biotech, regulatory bodies, and patient communities. With over 80 therapies in the pipeline, the future of ALS treatment is poised for transformative change. Key Takeaways from the Amyotrophic Lateral Sclerosis Pipeline Report • DelveInsight's amyotrophic lateral sclerosis pipeline analysis depicts a strong space with 75+ active players working to develop 80+ pipeline drugs for amyotrophic lateral sclerosis treatment. • The leading amyotrophic lateral sclerosis companies include Ionis Pharmaceuticals, 1ST Biotherapeutics, Scholar Rock, Revalesio, QurAlis Corporation, Sanofi, MediciNova, Helixmith, Verge Genomics, UCB, and others are evaluating their lead assets to improve the amyotrophic lateral sclerosis treatment landscape. • Key amyotrophic lateral sclerosis pipeline therapies in various stages of development include ION363, FB418, SRK-015, RNS60, QRL-201, SAR443820, MN-166, VM202, VRG-50635, Zilucoplan, and others. • In July 2025, Neurizon Therapeutics submitted a formal response to the FDA addressing the clinical hold on its IND application for NUZ-001, its lead ALS therapy. • In July 2025, Klotho Neurosciences received FDA Orphan Drug Designation for KLTO-202 ( its novel gene therapy targeting ALS. • In June 2025, Amylyx Pharmaceuticals, Inc. (NASDAQ: AMLX) announced that the FDA granted Fast Track designation to AMX0114, an investigational antisense oligonucleotide targeting calpain-2 for treating ALS. • In May 2025, BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI) announced FDA clearance to begin its Phase IIIb trial of NurOwn® for treating amyotrophic lateral sclerosis (ALS). • In May 2025, Neuralink announced that it received the FDA's " breakthrough" designation for its device aimed at restoring communication for individuals with severe speech impairment. The device, designed to assist patients with conditions like ALS, stroke, spinal cord injury, cerebral palsy, and multiple sclerosis, represents a significant step forward in neurotechnology. • In March 2025, DiagnaMed Holdings Corp. announced a significant milestone in rare disease research, receiving Orphan Drug Designation (ODD) from the FDA for molecular hydrogen in the treatment of ALS. • In January 2025, the FDA granted approval for Zydus Lifesciences to proceed with a randomized Phase IIb trial of its oral NLRP3 inflammasome inhibitor, Usnoflast, for the treatment of ALS. Request a sample and discover the recent breakthroughs happening in the amyotrophic lateral sclerosis pipeline landscape. Amyotrophic Lateral Sclerosis Overview Amyotrophic Lateral Sclerosis (ALS) is a rare, progressive neurodegenerative disorder that affects nerve cells in the brain and spinal cord. It leads to the loss of muscle control, eventually impacting the ability to speak, move, eat, and breathe. ALS is often classified into sporadic (the most common form) and familial types, with the latter being linked to genetic mutations. Although the exact cause of ALS is unknown in most cases, environmental and genetic factors are believed to contribute to its development. Currently, there is no cure for ALS, but several treatment options aim to slow disease progression and manage symptoms. FDA-approved drugs like riluzole and edaravone offer modest benefits in extending survival or preserving function. Ongoing research is focused on gene therapies, neuroprotective agents, and immune-targeting approaches to better address the underlying mechanisms of the disease and improve patient outcomes. Find out more about amyotrophic lateral sclerosis medication at Amyotrophic Lateral Sclerosis Treatment Analysis: Drug Profile MN-166: MediciNova MN-166 is an orally administered small molecule glial attenuator that reduces neuroinflammation by suppressing pro-inflammatory cytokines such as IL-1β, TNF-α, and IL-6, while potentially increasing the anti-inflammatory cytokine IL-10. It also functions as a toll-like receptor 4 (TLR4) antagonist, contributing further to its anti-inflammatory effects. MN-166 is currently in Phase II/III clinical trials for the treatment of ALS. RNS60: Revalesio RNS60 is designed to offer disease-modifying and potentially regenerative effects in neurological disorders. It works by activating signaling pathways that enhance mitochondrial function and reduce inflammation, offering neuroprotection and immune modulation. The FDA has granted RNS60 both Orphan Drug and Fast Track status for ALS. It is currently in Phase II clinical trials. VM202: Helixmith VM202 is a gene therapy aimed at tissue regeneration by promoting the production of hepatocyte growth factor (HGF) at the site of injection along peripheral nerves. HGF supports nerve protection, neuron growth, and muscle atrophy prevention. The FDA has granted VM202 Orphan Drug and Fast Track designations. It is being evaluated in Phase II trials for ALS treatment. Learn more about the novel and emerging amyotrophic lateral sclerosis pipeline therapies. By Product Type • Mono • Combination • Mono/Combination. By Stage • Late-stage products (Phase III) • Mid-stage products (Phase II) • Early-stage product (Phase I) along with the details of • Pre-clinical and Discovery stage candidates • Discontinued & Inactive candidates By Route of Administration • Intravenous • Subcutaneous • Oral • Intramuscular By Molecule Type • Monoclonal antibody • Small molecule • Peptide Scope of the Amyotrophic Lateral Sclerosis Pipeline Report • Coverage: Global • Key Amyotrophic Lateral Sclerosis Companies: Ionis Pharmaceuticals, 1ST Biotherapeutics, Scholar Rock, Revalesio, QurAlis Corporation, Sanofi, MediciNova, Helixmith, Verge Genomics, UCB, and others. • Key Amyotrophic Lateral Sclerosis Pipeline Therapies: ION363, FB418, SRK-015, RNS60, QRL-201, SAR443820, MN-166, VM202, VRG-50635, Zilucoplan, and others. Explore detailed insights on drugs used in the treatment of amyotrophic lateral sclerosis here. Table of Contents 1. Introduction 2. Executive Summary 3. Amyotrophic Lateral Sclerosis Pipeline: Overview 4. Analytical Perspective In-depth Commercial Assessment 5. Amyotrophic Lateral Sclerosis Pipeline Therapeutics 6. Amyotrophic Lateral Sclerosis Pipeline: Late-Stage Products (Phase III) 7. Amyotrophic Lateral Sclerosis Pipeline: Mid-Stage Products (Phase II) 8. Amyotrophic Lateral Sclerosis Pipeline: Early Stage Products (Phase I) 9. Therapeutic Assessment 10. Inactive Products 11. Company-University Collaborations (Licensing/Partnering) Analysis 12. Key Companies 13. Key Products 14. Unmet Needs 15. Market Drivers and Barriers 16. Future Perspectives and Conclusion 17. Analyst Views 18. Appendix About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform, PharmDelve. Media Contact Company Name: DelveInsight Contact Person: Jatin Vimal Email: Send Email Phone: +14699457679 Address: 304 S. Jones Blvd #2432 City: Las Vegas State: Nevada Country: United States Website:
Business Insider
14-07-2025
- Health
- Business Insider
Amylyx presents new analyses from Phase 2, Phase 2b trials of avexitide
Amylyx (AMLX) Pharmaceuticals announced the presentation of new exploratory analyses from the Phase 2 PREVENT and Phase 2b clinical trials of avexitide, an investigational, first-in-class glucagon-like peptide-1, GLP-1, receptor antagonist for the treatment of post-bariatric hypoglycemia, PBH, at the Endocrine Society's annual meeting, ENDO 2025. In the Phase 2b trial, avexitide 90 mg once daily, the dose being evaluated in the pivotal Phase 3 LUCIDITY trial, led to a 64% least-squares mean reduction vs. baseline in the composite rate of Level 2 and Level 3 hypoglycemic events in PBH, with more than half of the participants experiencing no events during the treatment period. The FDA-agreed-upon primary endpoint of LUCIDITY is reduction in the composite of Level 2 and Level 3 hypoglycemic events. Consistent reductions in composite rate of Level 2 and Level 3 hypoglycemic events also were seen with avexitide 45 mg twice daily studied in the Phase 2b trial and avexitide 30 mg twice daily and 60 mg once daily studied in the Phase 2 PREVENT trial. New pharmacokinetic and pharmacodynamic data were also presented demonstrating continuous pharmacologic activity of the 90 mg once daily dose regimen for a 24-hour period. Elevate Your Investing Strategy: Take advantage of TipRanks Premium at 50% off! Unlock powerful investing tools, advanced data, and expert analyst insights to help you invest with confidence. Make smarter investment decisions with , delivered to your inbox every week.
Associated Press
14-07-2025
- Health
- Associated Press
Amylyx Pharmaceuticals Presents New Exploratory Analyses from Phase 2 and Phase 2b Clinical Trials of Avexitide in Post-Bariatric Hypoglycemia at ENDO 2025
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Jul 13, 2025-- Amylyx Pharmaceuticals, Inc. (NASDAQ: AMLX) ('Amylyx' or the 'Company') today announced the presentation of new exploratory analyses from the Phase 2 PREVENT and Phase 2b clinical trials of avexitide, an investigational, first-in-class glucagon-like peptide-1 (GLP-1) receptor antagonist for the treatment of post-bariatric hypoglycemia (PBH) at the Endocrine Society's annual meeting (ENDO 2025). In the Phase 2b trial, avexitide 90 mg once daily, the dose being evaluated in the pivotal Phase 3 LUCIDITY trial, led to a 64% least-squares (LS) mean reduction (p=0.0031) vs. baseline in the composite rate of Level 2 and Level 3 hypoglycemic events in PBH, with more than half of the participants experiencing no events during the treatment period. LUCIDITY is a multicenter, randomized, double-blind, placebo-controlled Phase 3 clinical trial evaluating the efficacy and safety of avexitide in approximately 75 participants with PBH following Roux-en-Y gastric bypass surgery. The FDA-agreed-upon primary endpoint of LUCIDITY is reduction in the composite of Level 2 and Level 3 hypoglycemic events. Consistent reductions in composite rate of Level 2 and Level 3 hypoglycemic events also were seen with avexitide 45 mg twice daily studied in the Phase 2b trial and avexitide 30 mg twice daily and 60 mg once daily studied in the Phase 2 PREVENT trial. New pharmacokinetic (PK) and pharmacodynamic (PD) data were also presented demonstrating continuous pharmacologic activity of the 90 mg once daily dose regimen for a 24-hour period. 'Post-bariatric hypoglycemia can profoundly disrupt daily life, requiring individuals to carefully manage meals, social interactions, and routines, often while living in fear of their next hypoglycemic event. The new analysis presented at ENDO 2025 continues to support that avexitide may significantly reduce the frequency of these events,' said Marilyn Tan, MD, FACE, Principal Investigator of the LUCIDITY trial and Clinical Associate Professor at Stanford University. Camille L. Bedrosian, MD, Chief Medical Officer of Amylyx, added, 'Post-bariatric hypoglycemia is a serious and underrecognized condition with no FDA-approved treatments. The data presented show that, in an exploratory analysis from the Phase 2 PREVENT and Phase 2b clinical trials, avexitide significantly reduced the composite rate of Level 2 and 3 hypoglycemic events, including at the 90 mg once daily dose that is being studied in our pivotal Phase 3 LUCIDITY trial. We are particularly encouraged that over half of participants did not experience Level 2 or Level 3 hypoglycemic events during the treatment period. In addition, the pharmacokinetic and pharmacodynamic data demonstrated continuous pharmacologic activity of avexitide 90 mg once daily dose over 24 hours. We continue to be encouraged by avexitide's potential to deliver consistent, meaningful benefit to people living with PBH.' The population PK and PD analyses presented at ENDO 2025 demonstrated that avexitide 90 mg once daily maintained consistent GLP-1 receptor inhibition from morning to midnight and between doses. In vitro potency studies showed an IC₅₀ of approximately 20-30 nM (70-100 ng/mL), indicating robust target inhibition even in the presence of significant levels of GLP-1. PK modeling demonstrated that avexitide plasma levels exceeded IC₅₀ for a full 24-hour period. LUCIDITY was informed by data from five PBH clinical trials of avexitide showing consistent, dose-dependent effects, including statistically significant and clinically meaningful reductions in hypoglycemic events. Avexitide was generally well-tolerated, with a favorable safety profile replicated across clinical trials. Completion of recruitment for LUCIDITY is expected in 2025, with a data readout anticipated in the first half of 2026 and, if approved, commercial launch anticipated in 2027. The presentation and posters are available on the ' Presentations ' tab of the Amylyx website. Webcast Information Amylyx will host an investor event today, July 13, 2025, at 6:00 p.m. PT / 9:00 p.m. ET in San Francisco to discuss post-bariatric hypoglycemia and avexitide. A live webcast of the presentation and Q&A portion of the event can be accessed under 'Events and Presentations' in the Investor section of the Company's website, The webcast will be archived and available for replay for 90 days following the event. About Avexitide Avexitide is an investigational, first-in-class glucagon-like peptide-1 (GLP-1) receptor antagonist that has been evaluated in five Phase 1 and Phase 2 clinical trials for post-bariatric hypoglycemia (PBH) and has also been studied in congenital hyperinsulinism (HI). The U.S. Food and Drug Administration (FDA) has granted avexitide Breakthrough Therapy Designation for both indications, Rare Pediatric Disease Designation in congenital HI, and Orphan Drug Designation for the treatment of hyperinsulinemic hypoglycemia (which includes PBH and congenital HI). Avexitide is designed to bind to the GLP-1 receptor on pancreatic islet beta cells and inhibit the effect of GLP-1 to mitigate hypoglycemia by decreasing insulin secretion and stabilizing blood glucose levels. In PBH, excessive GLP-1 can lead to the hypersecretion of insulin and subsequent debilitating hypoglycemic events. In two Phase 2 PBH clinical trials, avexitide demonstrated highly statistically significant reductions in hypoglycemic events. These events can lead to autonomic and neuroglycopenic symptoms that can have a devastating impact on daily living. About Post-Bariatric Hypoglycemia (PBH) Post-bariatric hypoglycemia (PBH) is a condition that is estimated to affect approximately 8% of people in the U.S. who have undergone the two most common types of bariatric surgery, sleeve gastrectomy and Roux-en-Y gastric bypass (approximately 160,000 people in the U.S.). PBH is thought to be caused by an excessive glucagon-like peptide-1 (GLP-1) response leading to hypoglycemia and impaired quality of life. PBH can cause debilitating hypoglycemic events associated with inadequate supply of glucose to the brain, known as neuroglycopenia. Clinical manifestations can include impaired cognition, loss of consciousness, and seizures. PBH is also associated with a high degree of disability that can result in major disruptions to independent living. There are no approved therapies for PBH. About the LUCIDITY Trial LUCIDITY ( NCT06747468 ) is an approximately 75-participant, multicenter, randomized, double-blind, placebo-controlled Phase 3 clinical trial evaluating the efficacy and safety of avexitide in participants with PBH following Roux-en-Y gastric bypass (RYGB) surgery. The Phase 3 trial will be conducted at approximately 20 sites in the U.S. Participants will be randomized 3:2 to receive either 90 mg of avexitide subcutaneously once daily or placebo. The trial includes an up to six-week screening period, including a three-week run-in period, and a 16-week double-blind treatment period. Participants who complete the double-blind period will be eligible to enter an open-label extension (OLE) period with a duration of 32 weeks. The primary efficacy objective of LUCIDITY will evaluate the FDA-agreed upon primary outcome of reduction in the composite of Level 2 and Level 3 hypoglycemic events through Week 16. Safety and tolerability will also be evaluated. About Amylyx Pharmaceuticals At Amylyx, our mission is to usher in a new era of treating diseases with high unmet needs. Where others see challenges, we see opportunities that we pursue with urgency, rigorous science, and unwavering commitment to the communities we serve. We are currently focused on three investigational therapies across several neurodegenerative and endocrine diseases in which we believe they can make the greatest impact. For more information, visit and follow us on LinkedIn and X. For investors, please visit Forward-Looking Statements Statements contained in this press release regarding matters that are not historical facts are 'forward-looking statements' within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Such statements include, but are not limited to, Amylyx' expectations regarding: the potential of avexitide as a treatment for PBH; expectations regarding the timing for recruitment completion and topline data readout of the Phase 3 LUCIDITY trial of avexitide in PBH; and expectations regarding timing for potential commercialization of avexitide. Any forward-looking statements in this press release and related comments in the Company's earnings conference call are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. Risks that contribute to the uncertain nature of the forward-looking statements include: the success, cost, and timing of Amylyx' program development activities; Amylyx' ability to execute on its regulatory development plans and expectations regarding the timing of results from its planned data announcements and initiation of clinical studies; the risk that early-stage results may not reflect later-stage results; Amylyx' ability to fund operations, and the impact that global macroeconomic uncertainty, geopolitical instability, and public health events will have on Amylyx' operations, as well as the risks and uncertainties set forth in Amylyx' United States Securities and Exchange Commission (SEC) filings, including Amylyx' Annual Report on Form 10-K for the year ended December 31, 2024, and subsequent filings with the SEC. All forward-looking statements contained in this press release and related comments in our earnings conference call speak only as of the date on which they were made. Amylyx undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law. View source version on CONTACT: Media Amylyx Media Team (857) 320-6191 [email protected] Lindsey Allen (857) 320-6244 [email protected] KEYWORD: UNITED STATES NORTH AMERICA MASSACHUSETTS INDUSTRY KEYWORD: HEALTH CLINICAL TRIALS RESEARCH PHARMACEUTICAL SCIENCE BIOTECHNOLOGY SOURCE: Amylyx Pharmaceuticals, Inc. Copyright Business Wire 2025. PUB: 07/13/2025 05:00 PM/DISC: 07/13/2025 05:00 PM
Business Wire
13-07-2025
- Health
- Business Wire
Amylyx Pharmaceuticals Presents New Exploratory Analyses from Phase 2 and Phase 2b Clinical Trials of Avexitide in Post-Bariatric Hypoglycemia at ENDO 2025
CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Amylyx Pharmaceuticals, Inc. (NASDAQ: AMLX) ('Amylyx' or the 'Company') today announced the presentation of new exploratory analyses from the Phase 2 PREVENT and Phase 2b clinical trials of avexitide, an investigational, first-in-class glucagon-like peptide-1 (GLP-1) receptor antagonist for the treatment of post-bariatric hypoglycemia (PBH) at the Endocrine Society's annual meeting (ENDO 2025). In the Phase 2b trial, avexitide 90 mg once daily, the dose being evaluated in the pivotal Phase 3 LUCIDITY trial, led to a 64% least-squares (LS) mean reduction (p=0.0031) vs. baseline in the composite rate of Level 2 and Level 3 hypoglycemic events in PBH, with more than half of the participants experiencing no events during the treatment period. LUCIDITY is a multicenter, randomized, double-blind, placebo-controlled Phase 3 clinical trial evaluating the efficacy and safety of avexitide in approximately 75 participants with PBH following Roux-en-Y gastric bypass surgery. The FDA-agreed-upon primary endpoint of LUCIDITY is reduction in the composite of Level 2 and Level 3 hypoglycemic events. Consistent reductions in composite rate of Level 2 and Level 3 hypoglycemic events also were seen with avexitide 45 mg twice daily studied in the Phase 2b trial and avexitide 30 mg twice daily and 60 mg once daily studied in the Phase 2 PREVENT trial. New pharmacokinetic (PK) and pharmacodynamic (PD) data were also presented demonstrating continuous pharmacologic activity of the 90 mg once daily dose regimen for a 24-hour period. 'Post-bariatric hypoglycemia can profoundly disrupt daily life, requiring individuals to carefully manage meals, social interactions, and routines, often while living in fear of their next hypoglycemic event. The new analysis presented at ENDO 2025 continues to support that avexitide may significantly reduce the frequency of these events,' said Marilyn Tan, MD, FACE, Principal Investigator of the LUCIDITY trial and Clinical Associate Professor at Stanford University. Camille L. Bedrosian, MD, Chief Medical Officer of Amylyx, added, 'Post-bariatric hypoglycemia is a serious and underrecognized condition with no FDA-approved treatments. The data presented show that, in an exploratory analysis from the Phase 2 PREVENT and Phase 2b clinical trials, avexitide significantly reduced the composite rate of Level 2 and 3 hypoglycemic events, including at the 90 mg once daily dose that is being studied in our pivotal Phase 3 LUCIDITY trial. We are particularly encouraged that over half of participants did not experience Level 2 or Level 3 hypoglycemic events during the treatment period. In addition, the pharmacokinetic and pharmacodynamic data demonstrated continuous pharmacologic activity of avexitide 90 mg once daily dose over 24 hours. We continue to be encouraged by avexitide's potential to deliver consistent, meaningful benefit to people living with PBH.' The population PK and PD analyses presented at ENDO 2025 demonstrated that avexitide 90 mg once daily maintained consistent GLP-1 receptor inhibition from morning to midnight and between doses. In vitro potency studies showed an IC₅₀ of approximately 20-30 nM (70-100 ng/mL), indicating robust target inhibition even in the presence of significant levels of GLP-1. PK modeling demonstrated that avexitide plasma levels exceeded IC₅₀ for a full 24-hour period. LUCIDITY was informed by data from five PBH clinical trials of avexitide showing consistent, dose-dependent effects, including statistically significant and clinically meaningful reductions in hypoglycemic events. Avexitide was generally well-tolerated, with a favorable safety profile replicated across clinical trials. Completion of recruitment for LUCIDITY is expected in 2025, with a data readout anticipated in the first half of 2026 and, if approved, commercial launch anticipated in 2027. The presentation and posters are available on the ' Presentations ' tab of the Amylyx website. Webcast Information Amylyx will host an investor event today, July 13, 2025, at 6:00 p.m. PT / 9:00 p.m. ET in San Francisco to discuss post-bariatric hypoglycemia and avexitide. A live webcast of the presentation and Q&A portion of the event can be accessed under 'Events and Presentations' in the Investor section of the Company's website, The webcast will be archived and available for replay for 90 days following the event. About Avexitide Avexitide is an investigational, first-in-class glucagon-like peptide-1 (GLP-1) receptor antagonist that has been evaluated in five Phase 1 and Phase 2 clinical trials for post-bariatric hypoglycemia (PBH) and has also been studied in congenital hyperinsulinism (HI). The U.S. Food and Drug Administration (FDA) has granted avexitide Breakthrough Therapy Designation for both indications, Rare Pediatric Disease Designation in congenital HI, and Orphan Drug Designation for the treatment of hyperinsulinemic hypoglycemia (which includes PBH and congenital HI). Avexitide is designed to bind to the GLP-1 receptor on pancreatic islet beta cells and inhibit the effect of GLP-1 to mitigate hypoglycemia by decreasing insulin secretion and stabilizing blood glucose levels. In PBH, excessive GLP-1 can lead to the hypersecretion of insulin and subsequent debilitating hypoglycemic events. In two Phase 2 PBH clinical trials, avexitide demonstrated highly statistically significant reductions in hypoglycemic events. These events can lead to autonomic and neuroglycopenic symptoms that can have a devastating impact on daily living. About Post-Bariatric Hypoglycemia (PBH) Post-bariatric hypoglycemia (PBH) is a condition that is estimated to affect approximately 8% of people in the U.S. who have undergone the two most common types of bariatric surgery, sleeve gastrectomy and Roux-en-Y gastric bypass (approximately 160,000 people in the U.S.). PBH is thought to be caused by an excessive glucagon-like peptide-1 (GLP-1) response leading to hypoglycemia and impaired quality of life. PBH can cause debilitating hypoglycemic events associated with inadequate supply of glucose to the brain, known as neuroglycopenia. Clinical manifestations can include impaired cognition, loss of consciousness, and seizures. PBH is also associated with a high degree of disability that can result in major disruptions to independent living. There are no approved therapies for PBH. About the LUCIDITY Trial LUCIDITY (NCT06747468) is an approximately 75-participant, multicenter, randomized, double-blind, placebo-controlled Phase 3 clinical trial evaluating the efficacy and safety of avexitide in participants with PBH following Roux-en-Y gastric bypass (RYGB) surgery. The Phase 3 trial will be conducted at approximately 20 sites in the U.S. Participants will be randomized 3:2 to receive either 90 mg of avexitide subcutaneously once daily or placebo. The trial includes an up to six-week screening period, including a three-week run-in period, and a 16-week double-blind treatment period. Participants who complete the double-blind period will be eligible to enter an open-label extension (OLE) period with a duration of 32 weeks. The primary efficacy objective of LUCIDITY will evaluate the FDA-agreed upon primary outcome of reduction in the composite of Level 2 and Level 3 hypoglycemic events through Week 16. Safety and tolerability will also be evaluated. About Amylyx Pharmaceuticals At Amylyx, our mission is to usher in a new era of treating diseases with high unmet needs. Where others see challenges, we see opportunities that we pursue with urgency, rigorous science, and unwavering commitment to the communities we serve. We are currently focused on three investigational therapies across several neurodegenerative and endocrine diseases in which we believe they can make the greatest impact. For more information, visit and follow us on LinkedIn and X. For investors, please visit Forward-Looking Statements Statements contained in this press release regarding matters that are not historical facts are 'forward-looking statements' within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Such statements include, but are not limited to, Amylyx' expectations regarding: the potential of avexitide as a treatment for PBH; expectations regarding the timing for recruitment completion and topline data readout of the Phase 3 LUCIDITY trial of avexitide in PBH; and expectations regarding timing for potential commercialization of avexitide. Any forward-looking statements in this press release and related comments in the Company's earnings conference call are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. Risks that contribute to the uncertain nature of the forward-looking statements include: the success, cost, and timing of Amylyx' program development activities; Amylyx' ability to execute on its regulatory development plans and expectations regarding the timing of results from its planned data announcements and initiation of clinical studies; the risk that early-stage results may not reflect later-stage results; Amylyx' ability to fund operations, and the impact that global macroeconomic uncertainty, geopolitical instability, and public health events will have on Amylyx' operations, as well as the risks and uncertainties set forth in Amylyx' United States Securities and Exchange Commission (SEC) filings, including Amylyx' Annual Report on Form 10-K for the year ended December 31, 2024, and subsequent filings with the SEC. All forward-looking statements contained in this press release and related comments in our earnings conference call speak only as of the date on which they were made. Amylyx undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law.
Yahoo
03-06-2025
- Business
- Yahoo
Amylyx Pharmaceuticals Receives U.S. FDA Fast Track Designation for AMX0114 for the Treatment of Amyotrophic Lateral Sclerosis
- AMX0114 is an Amylyx-developed antisense oligonucleotide designed to target calpain-2, a key contributor to the axonal degeneration pathway in ALS - Phase 1 LUMINA trial of AMX0114 underway; early cohort data expected in 2025 CAMBRIDGE, Mass., June 03, 2025--(BUSINESS WIRE)--Amylyx Pharmaceuticals, Inc. (NASDAQ: AMLX) ("Amylyx" or the "Company") today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to AMX0114, an investigational antisense oligonucleotide (ASO) targeting calpain-2 for the treatment of people living with amyotrophic lateral sclerosis (ALS). "Obtaining FDA Fast Track designation for AMX0114 is an important step forward in our mission to develop potential treatments for people living with ALS, a relentlessly progressive and fatal disease with limited therapeutic options," said Camille L. Bedrosian, MD, Chief Medical Officer at Amylyx. "This designation from the FDA recognizes both the seriousness of this devastating disorder and the encouraging preclinical evidence supporting AMX0114's potential to target calpain-2, which has been found to be an important contributor to axonal degeneration, a critical driver in ALS progression. We are committed to advancing AMX0114 as quickly and efficiently as possible, and we continue to anticipate early cohort data from the Phase 1 LUMINA clinical trial later this year. We look forward to continued interaction with the FDA as we work to expeditiously advance the development of AMX0114, with the ultimate goal of addressing the urgent, unmet needs of the ALS community." The FDA's Fast Track designation is designed to facilitate the development and expedited review of therapies that are intended to treat serious and life-threatening conditions and demonstrate the potential to address an unmet medical need. A therapy that receives Fast Track designation may be eligible for more frequent meetings and communications with the FDA, as well as Priority Review if relevant criteria continue to be met. Amylyx designed AMX0114 to target calpain-2, a calcium-activated protease. Peer-reviewed research has demonstrated that overactive calpain-2 activity may be an important driver of disease progression in ALS and other neurodegenerative diseases by executing the degeneration of axons, the long tubular neuronal segments which carry signals from neurons to the muscle or other neurons. In preclinical studies, AMX0114 showed improved neuronal survival and reductions in extracellular neurofilament light (NfL) levels across multiple disease models. In April 2025, the Company announced the first participant was dosed in LUMINA, a multinational, randomized, double-blind, placebo-controlled, multiple ascending dose Phase 1 clinical trial designed to evaluate the safety and biological activity of AMX0114 in people living with ALS. LUMINA will also assess broadly researched ALS biomarkers, including changes from baseline in NfL levels. Approximately 48 participants will be randomized 3:1 to receive AMX0114 or placebo by intrathecal administration once every four weeks, for up to four doses. Amylyx expects early cohort data from LUMINA in 2025. About AMX0114 AMX0114 is an investigational antisense oligonucleotide (ASO) targeting calpain-2 (CAPN2) for the potential treatment of ALS. The U.S. Food and Drug Administration (FDA) has granted AMX0114 Fast Track designation for the treatment of ALS. In preclinical studies, treatment with AMX0114 resulted in potent, dose-dependent, and durable reduction in CAPN2 mRNA and calpain-2 protein levels in disease-relevant cell models of axonal degeneration. This translated to improved neuronal survival, including in a model of TDP-43 ALS, and reductions in extracellular neurofilament light (NfL) levels across multiple disease models and paradigms of neuronal injury. AMX0114 was well-tolerated in in vivo preclinical safety studies. About ALS Amyotrophic lateral sclerosis (ALS, also known as motor neuron disease) is a relentlessly progressive and fatal neurodegenerative disorder caused by motor neuron death in the brain and spinal cord. Motor neuron loss in ALS leads to deteriorating muscle function, the inability to move and speak, respiratory paralysis, and eventually, death. More than 90% of people with ALS have sporadic disease, showing no clear family history. About LUMINA The Phase 1 LUMINA clinical trial (NCT06665165) is a multinational, randomized, double-blind, placebo-controlled, multiple ascending dose trial evaluating the safety, tolerability, pharmacokinetics, and pharmacodynamics of AMX0114 in people living with ALS. LUMINA is anticipated to enroll approximately 48 adult participants. LUMINA will also assess broadly researched ALS biomarkers, including change from baseline in neurofilament light (NfL) levels. About Amylyx Pharmaceuticals At Amylyx, our mission is to usher in a new era of treating diseases with high unmet needs. Where others see challenges, we see opportunities that we pursue with urgency, rigorous science, and unwavering commitment to the communities we serve. We are currently focused on three investigational therapies across several neurodegenerative and endocrine diseases in which we believe they can make the greatest impact. For more information, visit and follow us on LinkedIn and X. For investors, please visit Forward-Looking Statements Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Such statements include, but are not limited to, Amylyx' expectations regarding: the potential for AMX0114 as a treatment for ALS, the expected timeline for data readout, and the expectation for regulatory action. Any forward-looking statements in this press release and related comments in the Company's earnings conference call are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. Risks that contribute to the uncertain nature of the forward-looking statements include: the success, cost, and timing of Amylyx' program development activities; Amylyx' ability to execute on its regulatory development plans and expectations regarding the timing of results from its planned data announcements and initiation of clinical studies; Amylyx' ability to fund operations, and the impact that global macroeconomic uncertainty, geopolitical instability, and public health events will have on Amylyx' operations, as well as the risks and uncertainties set forth in Amylyx' United States Securities and Exchange Commission (SEC) filings, including Amylyx' Annual Report on Form 10-K for the year ended December 31, 2024, and subsequent filings with the SEC. All forward-looking statements contained in this press release and related comments in our earnings conference call speak only as of the date on which they were made. Amylyx undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law. View source version on Contacts Media Amylyx Media Team(857) 320-6191amylyxmediateam@ Investors Lindsey Allen(857) 320-6244Investors@ Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
