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Malaysian Reserve
5 days ago
- Business
- Malaysian Reserve
Progressive Supranuclear Palsy Market on Upward Trajectory During the Forecast Period (2025-2034) with Emerging Immunotherapies
The progressive supranuclear palsy market is expected to witness steady growth due to increasing disease awareness, advancements in diagnostic tools, and ongoing clinical research focused on disease-modifying therapies. Rising prevalence of neurodegenerative disorders, coupled with the aging global population, is driving the demand for novel treatments. LAS VEGAS, Aug 7, 2025 /PRNewswire/ — DelveInsight's Progressive Supranuclear Palsy Market Insights report includes a comprehensive understanding of current treatment practices, progressive supranuclear palsy emerging drugs, market share of individual therapies, and current and forecasted market size from 2020 to 2034, segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan]. Key Takeaways from the Progressive Supranuclear Palsy Market Report According to DelveInsight's analysis, the market size for progressive supranuclear palsy in the 7MM is expected to grow significantly by 2034. The United States accounted for the highest progressive supranuclear palsy treatment market size in 7MM in 2024, in comparison to the other major markets, i.e., EU4 countries (Germany, France, Italy, and Spain), the United Kingdom, and Japan. Approximately one in every 100,000 individuals over the age of 60 is affected by progressive supranuclear palsy. Among all the phenotype-specific cases of PSP, Richardson's syndrome accounted for more than 50% of cases in 2024. Leading progressive supranuclear palsy companies developing emerging therapies, such as Amylyx Pharmaceuticals, AlzProtect, Transposon Therapeutics, Ferrer, Asceneuron, UCB Biopharma, Novartis, and others, are developing new progressive supranuclear palsy treatment drugs that can be available in the progressive supranuclear palsy market in the coming years. The promising progressive supranuclear palsy therapies in the pipeline include AMX0035, AZP2006, TPN-101, FNP-223/ASN90, Bepranemab (UCB0107), NIO752, and others. Discover the progressive supranuclear palsy new treatment @ New Treatments for Progressive Supranuclear Palsy Progressive Supranuclear Palsy Market Dynamics The progressive supranuclear palsy market dynamics are expected to change in the coming years. The progressive supranuclear palsy market is driven by increasing disease awareness, advancements in diagnostic technologies, and growing research efforts to develop disease-modifying therapies. Rising prevalence of neurodegenerative disorders, particularly among the aging population, is further fueling the demand for effective PSP treatment options. Strategic collaborations between pharmaceutical companies and research institutions are accelerating drug discovery and clinical trials, while regulatory incentives such as orphan drug designations are encouraging innovation in this rare disease space. Additionally, the growing adoption of advanced imaging techniques and biomarker-based diagnostics is enabling early and accurate diagnosis, creating a favorable environment for therapeutic interventions and market growth. As potential therapies are being investigated for the treatment of progressive supranuclear palsy, it is safe to predict that the treatment space will significantly impact the progressive supranuclear palsy market during the forecast period. Moreover, the anticipated introduction of emerging therapies with improved efficacy and a further improvement in the diagnosis rate are expected to drive the growth of the progressive supranuclear palsy market in the 7MM. However, several factors may impede the growth of the progressive supranuclear palsy market. A major challenge is the limited understanding of the disease's underlying pathophysiology, which complicates the identification of viable therapeutic targets. The small patient population and the rarity of PSP restrict the scope of clinical trials, leading to high costs, longer timelines, and recruitment difficulties. Additionally, the absence of disease-modifying treatments and reliance on symptomatic therapies limit market growth, as current treatment options provide only minimal clinical benefit. Regulatory hurdles, stringent approval processes, and a high failure rate in late-stage clinical trials further exacerbate the challenges. Moreover, the lack of robust diagnostic tools and biomarkers often delays early diagnosis, reducing the potential for timely interventions and effective disease management. Progressive Supranuclear Palsy Treatment Market At present, no approved medications or standardized clinical guidelines exist specifically for the treatment of progressive supranuclear palsy. Management focuses on alleviating symptoms, providing supportive care, and enhancing the patient's quality of life. Drugs commonly prescribed for Parkinson's disease, such as levodopa, may offer some relief from motor symptoms like rigidity, tremors, and bradykinesia, but their effectiveness in PSP is generally limited and short-lived. Dopamine agonists, including amantadine and ropinirole, can be used alone or in combination with levodopa, though they provide only modest benefits. Beyond medications, a multidisciplinary care approach is crucial. Physical therapy aids in improving mobility and balance, occupational therapy assists with daily activities, and speech and swallowing therapy helps manage communication and feeding challenges. These supportive strategies play a key role in preserving the best possible quality of life for PSP patients. To know more about progressive supranuclear palsy treatment options, visit @ Approved Progressive Supranuclear Palsy Drugs Progressive Supranuclear Palsy Pipeline Therapies and Key Companies The emerging pipeline for PSP includes AZP2006 (AlzProtect), AMX0035 (Amylyx Pharmaceuticals), TPN-101 (Transposon Therapeutics), FNP-223 (Ferrer), and others. AZP2006 is an orally administered small molecule featuring a unique mechanism of action. It acts through a neurotrophic factor, offering enhanced neuroprotective effects combined with anti-inflammatory activity in the nervous system. This compound shows significant therapeutic promise for addressing the underlying pathological mechanisms of progressive supranuclear palsy (PSP) and other tau-related disorders, including Alzheimer's disease. In September 2024, the company announced the successful completion of a Phase IIa clinical trial of AZP2006 for PSP treatment. Throughout a 3-month Phase IIa study in PSP patients, the drug demonstrated promising clinical and biomarker efficacy signals. These findings were further corroborated by results from a 6-month open-label extension study, strengthening its therapeutic potential. AZP2006 has also been granted Orphan Drug Designation by both the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA). In April 2025, the Progressive Supranuclear Palsy (PSP) Trial Platform (PTP), led by Drs. Adam Boxer (University of California, San Francisco), Irene Litvan (University of California, San Diego), Julio Rojas (UCSF), and Anne-Marie Wills (Massachusetts General Hospital), selected two promising drug candidates, Axon Neuroscience's AADvac1 and Alzprotect's AZP2006, as the first compounds to be tested under the platform trial. This initiative aims to accelerate the development of effective treatments for PSP, a rare and fatal neurodegenerative disorder. AMX0035 is an oral, fixed-dose combination therapy composed of sodium phenylbutyrate and taurursodiol (also known as ursodoxicoltaurine). It has a strong mechanistic rationale for PSP, targeting critical upstream pathways involved in disease pathogenesis, such as the unfolded protein response and mitochondrial dysfunction. Additionally, AMX0035 has been shown to lower p-tau levels in Alzheimer's disease studies. Currently, AMX0035 is being evaluated in a global Phase IIb/III (ORION) clinical trial, which aims to determine its efficacy, safety, and tolerability in PSP patients. The trial is expected to conclude by April 2026, with Amylyx Pharmaceuticals planning to release safety and efficacy data from the unblinded Phase IIb portion of the study in Q3 2025. The anticipated launch of these emerging therapies are poised to transform the progressive supranuclear palsy market landscape in the coming years. As these cutting-edge therapies continue to mature and gain regulatory approval, they are expected to reshape the progressive supranuclear palsy market landscape, offering new standards of care and unlocking opportunities for medical innovation and economic growth. Discover more about progressive supranuclear palsy marketed and pipeline drugs @ Progressive Supranuclear Palsy Clinical Trials Recent Developments in the Progressive Supranuclear Palsy Market In June 2025, Ferrer announced that the US FDA has granted the Fast Track designation to FNP-223 for treatment of patients with progressive supranuclear palsy (PSP). In April 2025, AlzProtect's AZP2006 was chosen as one of the initial two therapies to be tested in the newly launched national Phase II Platform Trial for Progressive Supranuclear Palsy (PSP) in the United States. Funded by the National Institute on Aging (NIH), the trial aims to fast-track the development of effective treatments for PSP. Progressive Supranuclear Palsy Overview Progressive supranuclear palsy is a rare neurodegenerative disorder that affects movement, balance, speech, and eye movements. It is caused by the accumulation of abnormal tau protein in certain areas of the brain, leading to the degeneration of nerve cells. While the exact cause of PSP is not fully understood, it is believed to involve a combination of genetic and environmental factors. PSP typically affects individuals over the age of 60 and progresses gradually over time. The symptoms of PSP include difficulties with balance and frequent falls, stiffness and slow movements similar to Parkinson's disease, and problems with eye movements, particularly the inability to move the eyes up and down. Other symptoms may include speech and swallowing difficulties, personality changes, irritability, depression, and cognitive decline. Unlike Parkinson's disease, tremors are uncommon in PSP, which helps in differentiating the two conditions. Diagnosis of PSP is challenging as there is no specific test to confirm the condition. It is primarily diagnosed based on clinical symptoms, neurological examinations, and the exclusion of other similar conditions. Brain imaging techniques, such as MRI, may reveal characteristic brain changes like midbrain shrinkage, which supports the diagnosis. Early recognition of eye movement abnormalities and postural instability is crucial for an accurate diagnosis. Progressive Supranuclear Palsy Epidemiology Segmentation The progressive supranuclear palsy epidemiology section provides insights into the historical and current progressive supranuclear palsy patient pool and forecasted trends for the 7MM. It helps recognize the causes of current and forecasted patient trends by exploring numerous studies and views of key opinion leaders. The progressive supranuclear palsy market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM, segmented into: Total Prevalent Cases of PSP Total Diagnosed Prevalent Cases of PSP Gender-specific Diagnosed Prevalent Cases of PSP Phenotype-specific Diagnosed Prevalent Cases of PSP Comorbidity Associated Cases with PSP Progressive Supranuclear Palsy Market Report Metrics Details Study Period 2020–2034 Coverage 7MM [The United States, the EU4 (Germany, France, Italy, and Spain) and The United Kingdom, and Japan]. Key Progressive Supranuclear Palsy Companies Amylyx Pharmaceuticals, AlzProtect, Transposon Therapeutics, Ferrer, Asceneuron, UCB Biopharma, Novartis, and others Key Progressive Supranuclear Palsy Therapies AMX0035, AZP2006, TPN-101, FNP-223/ASN90, Bepranemab (UCB0107), NIO752, and others Scope of the Progressive Supranuclear Palsy Market Report Therapeutic Assessment: Progressive Supranuclear Palsy current marketed and emerging therapies Progressive Supranuclear Palsy Market Dynamics: Key Market Forecast Assumptions of Emerging Progressive Supranuclear Palsy Drugs and Market Outlook Competitive Intelligence Analysis: SWOT analysis and Market entry strategies Unmet Needs, KOL's views, Analyst's views, Progressive Supranuclear Palsy Market Access and Reimbursement Download the report to understand which factors are driving progressive supranuclear palsy therapeutics market trends @ Progressive Supranuclear Palsy Market Trends Table of Contents 1. Progressive Supranuclear Palsy Market Key Insights 2. Progressive Supranuclear Palsy Market Report Introduction 3. Progressive Supranuclear Palsy Market Overview at a Glance 4. Progressive Supranuclear Palsy Market Executive Summary 5. Disease Background and Overview 6. Progressive Supranuclear Palsy Treatment and Management 7. Progressive Supranuclear Palsy Epidemiology and Patient Population 8. Patient Journey 9. Progressive Supranuclear Palsy Marketed Drugs 10. Progressive Supranuclear Palsy Emerging Drugs 11. Seven Major Progressive Supranuclear Palsy Market Analysis 12. Progressive Supranuclear Palsy Market Outlook 13. Potential of Current and Emerging Therapies 14. KOL Views 15. Unmet Needs 16. SWOT Analysis Related Reports Progressive Supranuclear Palsy Pipeline Progressive Supranuclear Palsy Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key PSP companies, including Amylyx Pharmaceuticals, Transposon Therapeutics, Inc., Ferrer International, Novartis Pharmaceuticals, TauC3 Biologics, among others. Parkinson's Disease Market Parkinson's Disease Market Insights, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key Parkinson's disease companies, including UCB Biopharma SRL, Novartis, Annovis Bio, Supernus Pharmaceuticals, Inc., Britannia Pharmaceutical, Pharma Two B, Mitsubishi Tanabe Pharma (NeuroDerm), AbbVie, Cerevel Therapeutics, LLC, among others. Alzheimer's Disease Market Alzheimer's Disease Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key Alzheimer's disease companies including AB Science, Alzheon Inc., AriBio Co., Ltd., AgeneBio, Inc., Anavex Life Sciences Corp., Annovis Bio, Inc., Cerecin, BioVie, Cassava Sciences, Novo Nordisk, Eli Lilly, Neurim Pharmaceuticals, Suven Life Sciences, Bristol-Myers Squibb, Karuna Therapeutics, T3D Therapeutics, Inc., Lexeo Therapeutics, Axsome Therapeutics, Inc., Araclon Biotech S.L., Eisai Co., Ltd., TauRx Therapeutics, TrueBinding, Inc., AC Immune SA, Johnson & Johnson, Longeveron Inc., Vaccinex Inc., IGC Pharma LLC, among others. Dementia Market Dementia Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key dementia companies including Eisai Co., Ltd., Eli Lilly and Company, Novartis AG, DAIICHI SANKYO COMPANY, LIMITED, AbbVie Inc., Lundbeck, Biogen, Cipla, among others. 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. Contact UsShruti Thakur info@ + Logo: View original content:
Business Wire
5 days ago
- Business
- Business Wire
Amylyx Pharmaceuticals Reports Second Quarter 2025 Financial Results
CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Amylyx Pharmaceuticals, Inc. (Nasdaq: AMLX) ('Amylyx' or the 'Company') today reported financial and business results for the second quarter ended June 30, 2025. 'As we look ahead to the second half of the year and into 2026, we remain encouraged by the strength of our pipeline and the continued momentum towards our clinical milestones,' said Joshua Cohen and Justin Klee, Co-CEOs of Amylyx. 'We expect to complete recruitment in the pivotal Phase 3 LUCIDITY trial of avexitide in 2025, with topline data anticipated in the first half of 2026. In the third quarter, we expect to share an unblinded analysis from the Phase 2b portion of our ORION trial of AMX0035 in progressive supranuclear palsy, for which we have set a high bar and will inform our decision regarding advancing to the Phase 3 portion of the trial. We also look forward to providing an update on our Wolfram syndrome program later this year, building on previous long-term Week 48 data from our Phase 2 HELIOS trial. In ALS, we were pleased to receive FDA Fast Track designation for AMX0114, and we anticipate early cohort data from the ongoing Phase 1 LUMINA trial later this year. We remain focused on disciplined execution across our programs.' Second Quarter and Recent Updates: Amylyx presented new exploratory analyses from the Phase 2 PREVENT and Phase 2b clinical trials of avexitide, a glucagon-like peptide-1 (GLP-1) receptor antagonist with U.S. Food and Drug Administration (FDA) Breakthrough Designation, for the treatment of post-bariatric hypoglycemia (PBH) at the Endocrine Society's annual meeting (ENDO 2025) in July 2025. The Phase 2 PREVENT trial evaluated avexitide in PBH following Roux-en-Y gastric bypass (RYGB) surgery, and the Phase 2b trial evaluated avexitide in PBH following a variety of upper GI surgeries, including RYGB, sleeve gastrectomy, esophagectomy, Nissen fundoplication, and gastrectomy. 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 (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. The 45 mg twice daily, 30 mg twice daily, and 60 mg once daily dose regimens all likewise demonstrated consistent reductions in composite rate of Level 2 and Level 3 hypoglycemic events. 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. Avexitide was generally well tolerated, with a favorable safety profile replicated across clinical trials. Amylyx presented new long-term Week 48 data from the Phase 2 open-label HELIOS clinical trial of AMX0035 (sodium phenylbutyrate [PB] and taurursodiol [TURSO, also known as ursodoxicoltaurine]) in adults living with Wolfram syndrome at the Joint Congress of the European Society for Pediatric Endocrinology and the European Society of Endocrinology in May 2025. Consistent with previously presented data on the primary efficacy outcome of improvement in pancreatic beta cell function, as measured by C-peptide response to a mixed-meal tolerance test at Week 24, treatment with AMX0035 through Week 48 of the HELIOS trial demonstrated continued and sustained improvement in pancreatic beta cell function. Long-term Week 48 results also showed sustained improvements or stabilization in multiple outcomes related to disease progression, including in glycemic control, as measured by hemoglobin A1c and time in target glucose range assessed by continuous glucose monitoring, as well as visual acuity. All participants with available measurements met the responder criteria, defined as either improvement or no change, on both the Patient Global Impression of Change and Clinician Global Impression of Change at Weeks 24 and 48, indicating stability or improvement in their Wolfram syndrome-related symptoms. Results from qualitative on-study interviews further supported the potential positive impact of AMX0035 on symptom burden. Safety data were consistent with prior studies of AMX0035. Amylyx received FDA Fast Track designation for AMX0114, an investigational antisense oligonucleotide targeting knockdown of calpain-2, for people living with amyotrophic lateral sclerosis (ALS) in June 2025. Under the FDA's Fast Track Designation, AMX0114 is eligible for more frequent meetings and communications with the FDA, as well as Priority Review if relevant criteria continue to be met. Upcoming Expected Milestones: Completion of recruitment for the pivotal Phase 3 LUCIDITY clinical trial of avexitide in PBH following RYGB surgery expected in 2025, with a data readout anticipated in the first half of 2026 and, if approved, commercial launch anticipated in 2027. LUCIDITY is a multicenter, randomized, double-blind, placebo-controlled Phase 3 clinical trial evaluating the efficacy and safety of avexitide in approximately 75 participants at approximately 20 sites in the U.S. LUCIDITY is evaluating the FDA-agreed-upon primary outcome of reduction in the composite of Level 2 and Level 3 hypoglycemic events through Week 16. Unblinded analysis of the Phase 2b portion of the Phase 2b/3 ORION trial evaluating AMX0035 for progressive supranuclear palsy (PSP) expected in the third quarter of 2025. ORION is an operationally seamless Phase 2b/3 clinical trial in people living with PSP. The Phase 2b portion was fully enrolled in January 2025 with a total of 139 participants randomized. Phase 2b efficacy and safety data from an unblinded analysis with Week 24 data from all participants will be used to inform a go/no-go decision on the Phase 3 portion of the trial. Update on the AMX0035 Wolfram syndrome program expected in 2025. In 2024, Amylyx reported positive topline results from the Company's Phase 2 HELIOS trial, an open-label study of 12 adult participants. At Week 24, stabilization or improvement was demonstrated across all key clinical measures, including pancreatic function, glycemic control, and vision, including the trial's primary efficacy outcome of improvement in pancreatic function, as measured by C-peptide response to a mixed-meal tolerance test at Week 24. In May, long-term Week 48 data from HELIOS were presented at the Joint Congress of the European Society for Pediatric Endocrinology and the European Society of Endocrinology. These data demonstrated that treatment with AMX0035 led to continued sustained stabilization or improvement. These results and discussions with FDA are informing the design of a Phase 3 trial of AMX0035 in Wolfram syndrome. Early cohort data from the Phase 1 LUMINA clinical trial of AMX0114 in ALS expected in 2025. LUMINA is a multinational, randomized, double-blind, placebo-controlled, multiple ascending dose clinical trial designed to evaluate the safety and biological activity of AMX0114. The trial will also assess ALS biomarkers, including changes from baseline in neurofilament light (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. Financial Results for the Second Quarter Ended June 30, 2025 R&D Expenses: Research and development expenses for the second quarter of 2025 were $27.2 million, compared to $23.3 million for the same period in 2024. The increase was primarily due to the clinical development of avexitide in PBH and AMX0035 in PSP, offset by a decrease in spending on AMX0035 in ALS. Research and development expenses include $2.0 million of stock-based compensation expense for the quarter, compared to $2.4 million of stock-based compensation expense for the same period in 2024. SG&A Expenses: Selling, general, and administrative expenses for the second quarter of 2025 were $15.6 million, compared to $21.6 million for the same period in 2024. The decrease was primarily due to a decrease in payroll and personnel-related costs and a decrease in consulting, professional services, and other expenses. Selling, general, and administrative expenses include $5.4 million of stock-based compensation expense for the quarter, compared to $7.1 million for the same period in 2024. Net Loss: Net loss for the three months ended June 30, 2025 was $41.4 million, or $0.46 per share, compared to net loss of $72.7 million, or $1.07 per share for the same period in 2024. Cash Position: Cash, cash equivalents, and marketable securities were $180.8 million at June 30, 2025, compared to $204.1 million at March 31, 2025. Based on its current operating plans, Amylyx expects its cash runway to be through the end of 2026. Investor Conference Call Information Amylyx' management team will host a conference call today, August 7, 2025, at 8:00 a.m. ET to discuss financial results and provide an update on the business. To access the conference call, please dial +1 (800)-836-8184 (U.S. & Canada) or +1 (646)-357-8785 (international) at least 10 minutes prior to the start time and ask to be joined into the Amylyx Pharmaceuticals call. A live audio webcast of the call will be available 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. Available Information We periodically provide other information for investors on our corporate website, and our investor relations website, This includes press releases and other information about financial performance, information on corporate governance, and details related to our annual meeting of stockholders. We intend to use our website as a means of disclosing material non-public information and for complying with our disclosure obligations under Regulation FD. Accordingly, investors should monitor our website, in addition to following the Company's press releases, SEC filings, and public conference calls and webcasts. 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 is being 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; expectations regarding timing for potential commercialization of avexitide; expectations regarding the potential of AMX0035 (sodium phenylbutyrate and taurursodiol) as a treatment for Wolfram syndrome and PSP or other neurodegenerative diseases and planned updates to those programs; planned discussions with the FDA related to AMX0035 for the treatment of Wolfram syndrome; expectations regarding the timing of the announcement of interim results from the Company's Phase 2b/3 ORION trial of AMX0035 for the treatment of PSP; the potential for AMX0114 as a treatment for ALS, the expected timeline for data readout of the Phase 1 LUMINA clinical trial, and expectation for regulatory action; and Amylyx' expectations regarding its financial performance, cash runway and longer-term strategy. 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. AMYLYX PHARMACEUTICALS, INC. UNAUDITED (in thousands, except share and per share data) Three Months Ended June 30, Six Months Ended June 30, 2025 2024 2025 2024 Product revenue, net $ — $ (1,023 ) $ — $ 87,620 Operating expenses: Cost of sales — 8 — 5,953 Cost of sales - inventory impairment and loss on firm purchase commitments — 7,410 — 117,871 Research and development 27,217 23,347 49,336 59,955 Selling, general and administrative 15,640 21,647 31,324 79,406 Restructuring expenses — 22,851 — 22,851 Total operating expenses 42,857 75,263 80,660 286,036 Loss from operations (42,857 ) (76,286 ) (80,660 ) (198,416 ) Other income, net 1,414 3,586 3,310 7,165 Loss before income taxes (41,443 ) (72,700 ) (77,350 ) (191,251 ) Provision for income taxes — — — 242 Net loss $ (41,443 ) $ (72,700 ) $ (77,350 ) $ (191,493 ) Net loss per share — basic and diluted $ (0.46 ) $ (1.07 ) $ (0.88 ) $ (2.82 ) Weighted-average shares used in computing net loss per share — basic and diluted 89,138,568 68,024,929 87,427,345 67,939,642 Expand
Yahoo
23-06-2025
- Health
- Yahoo
Amyotrophic Lateral Sclerosis Treatment Market To Observe Growth at a CAGR of 6.1% by 2033
The global amyotrophic lateral sclerosis treatment market is undergoing gradual yet impactful transformation, driven by advancements in neurodegenerative disease research, gene-targeted therapies, and biomarkers innovation. Emerging therapeutic approaches, including antisense oligonucleotides and monoclonal antibodies, are enhancing precision treatment for genetically defined amyotrophic lateral sclerosis (ALS) subtypes, such as SOD1-linked ALS. Although ALS remains a rare condition, with an incidence of about 2 per 100,000 annually, it is receiving increased attention due to heightened awareness, improved diagnostic capabilities, and robust investment in R&D. The evolving treatment landscape is also benefiting from a growing emphasis on personalized medicine, which is reshaping disease management strategies for this devastating condition. Chicago, June 23, 2025 (GLOBE NEWSWIRE) -- The global amyotrophic lateral sclerosis treatment market is projected to grow from USD 724.5 million in 2024 to USD 1,234.5 million by 2033, reflecting a CAGR of 6.1% over the forecast period. This moderate yet steady expansion is supported by a rising aging population, advancements in disease-modifying therapies, and increasing prevalence rates. Although curative therapies remain elusive, the pipeline is increasingly populated with targeted investigational drugs, including small molecules, peptides, and biologics. Institutions and regulatory agencies are placing a growing focus on biomarker validation to accelerate clinical development and improve treatment outcomes. The recognition of biomarkers such as neurofilament light chain (NfL) as indicators of therapeutic response marks a pivotal step forward in streamlining ALS drug development. Download Free Sample Pages: The rising incidence of amyotrophic lateral sclerosis, particularly among adults aged 60 to 79, underscores the growing healthcare burden. In the U.S., the National ALS Registry estimated over 32,800 cases in 2022, with projections nearing 36,300 by 2030. The progressive nature and high mortality rate of ALS necessitate rapid intervention and proactive management. The predominance of sporadic ALS, comprising 90% of all cases, presents challenges in early detection and treatment targeting. Conversely, familial ALS, which accounts for roughly 10%, presents distinct genetic markers such as C9ORF72 and SOD1 mutations, offering a clearer path for precision therapies like Qalsody (Tofersen), which received FDA approval in 2023. Innovative drug approvals and clinical research are driving optimism. Treatments such as Riluzole, Edaravone, and more recently, gene-targeted agents like Tofersen and AMX0035, have brought incremental benefits. However, setbacks remains, for example; AMX0035 (Relyvrio) was withdrawn in 2024 following unsatisfactory phase III results. Nonetheless, therapeutic progress continues, with a growing number of trials exploring novel approaches, including stem cell-based interventions and gene editing technologies. The National Institutes of Health (NIH) has committed substantial funding, including a $25 million investment over five years, to accelerate ALS research and drug discovery efforts. Despite these advances, the amyotrophic lateral sclerosis treatment market continues to face notable challenges. The high cost of care, particularly in late-stage disease, imposes a significant financial burden. Treatment expenses in the U.S. can exceed $120,000 annually as the disease progresses, compounded by delayed diagnoses and complex care requirements. Furthermore, innovative gene therapies while promising carry a high price tags, as seen with Qalsody, which costs over $16,000 per 15 mL dose. Nevertheless, the amyotrophic lateral sclerosis treatment market holds promising potential, bolstered by biomarker innovation, gene-specific therapies, and a commitment to earlier diagnosis and intervention. With increasing efforts from academic research centers, pharmaceutical companies, and patient advocacy groups, the field is gradually shifting from symptomatic care toward disease-modifying approaches. As regulatory pathways evolve and precision medicine takes hold, the market is expected to offer more effective and personalized treatments, transforming the outlook for patients living with amyotrophic lateral sclerosis (ALS).Market Forecast (2033) USD 1,234.5 million CAGR 6.1% Top Driver Increasing Incidence of Amyotrophic Lateral Sclerosis (ALS) Among the Aging Population Top Trend Emerging Biomarkers in Amyotrophic Lateral Sclerosis Treatment Top Challenge Elevated Cost of Treating Amyotrophic Lateral Sclerosis (ALS) Biomarker Innovation: Advancing Diagnostic Precision and Accelerating ALS Therapeutics Rapid strides in biomarker research are transforming the treatment paradigm for amyotrophic lateral sclerosis (ALS), ushering in a new era of precision diagnostics and targeted therapeutic strategies. Historically constrained by delayed diagnoses and limited tools for tracking disease progression, the ALS field is now benefitting from biomarker technologies that enhance early detection, improve patient monitoring, and streamline the development of novel therapies. A pivotal breakthrough came in 2023 when the U.S. Food and Drug Administration (FDA) granted accelerated approval to Tofersen (Qalsody), establishing neurofilament light chain (NfL) as a qualified response biomarker. Clinical data revealed that reductions in NfL were indicative of slower neurodegeneration, validating its role in assessing treatment effectiveness. Building on this momentum, researchers are expanding their focus to identify other biomarkers, including abnormal forms of the TDP-43 protein, strongly associated with ALS pathology. Early detection of these proteins in cerebrospinal fluid or blood may enable real-time disease monitoring and inform more individualized treatment plans. Public-sector institutions are playing a key role in driving this progress. The National Institute of Neurological Disorders and Stroke (NINDS) is actively funding initiatives aimed at validating emerging biomarkers, particularly to shorten diagnostic timelines for patients with atypical or early-stage ALS. These efforts are critical to improving prognostic accuracy and tailoring therapeutic approaches to each patient's disease trajectory. As biomarker science continues to evolve, its integration into amyotrophic lateral sclerosis treatment and research is poised to deliver meaningful clinical and commercial impact. From optimizing trial design to facilitating faster regulatory approvals, biomarkers are redefining how amyotrophic lateral sclerosis (ALS) is diagnosed and managed moving the field closer to a future of earlier intervention, personalized care, and more effective therapies Global Amyotrophic Lateral Sclerosis Treatment Market: Regional Drivers Shaping the Future of Neuromuscular Care The global amyotrophic lateral sclerosis treatment market is segmented into North America, South America, Europe, Asia-Pacific, and the Middle East & Africa, with North America maintaining a dominant position. In the United States alone, an estimated 30,000 individuals are currently living with ALS, underscoring the urgent need for effective therapeutic options. The region leads in research and development, with most ALS medications receiving initial regulatory approval there. Key organizations like the ALS Association play a pivotal role in advancing treatment efforts, supporting a wide range of initiatives including grant-funded stem cell therapy programs. Enhanced insurance coverage further facilitates access to care, mitigating the financial burden of high treatment costs. Additionally, comprehensive support systems such as the ALS Network offer a wide array of services, from equipment loans and clinic access to benefit assistance, advocacy, and peer support groups, ensuring holistic care for patients and caregivers. Canada also plays an important role, with the Canadian Neuromuscular Disease Registry (CNDR) collecting nationwide clinical data to improve care standards and treatment accessibility. Building on the foundations established in North America, Europe has emerged as a strong contributor to ALS research and patient care. Collaborative efforts such as the ENCALS and TRICALS work with more than 60 centers across the continent to expand clinical trial availability, though currently, less than 5% of amyotrophic lateral sclerosis (ALS) patients in Europe participate in such studies. These efforts reflect a regional commitment to improving early access to experimental therapies and fostering cross-border collaboration in amyotrophic lateral sclerosis (ALS) care. The momentum continues to build in the Asia-Pacific region, where countries are increasingly investing in infrastructure and professional training. Japan is establishing dedicated multidisciplinary ALS centers, while emerging economies like India are making notable progress. In 2023/2024, over 550 Indian physicians received certification through Advanced Life Support (ALS) programs delivered in partnership with the European Resuscitation Council (ERC). These internationally accredited training initiatives are elevating standards of care and expanding clinical capacity across the region. While advancements are more gradual in the Middle East and Africa, the region is showing signs of long-term potential. With healthcare infrastructure and resources still in development, increasing attention to awareness-building, professional training, and international cooperation indicates a growing foundation for future market expansion. As these efforts mature, the region may play a more prominent role in the global amyotrophic lateral sclerosis treatment landscape. Modify Report as Per Requirements: Recent Advancements and Key Players in the Amyotrophic Lateral Sclerosis Treatment Market Several prominent players, including MediciNova, Mitsubishi Tanabe Pharma, Ionis Pharmaceuticals, Alector, Sanofi, ITF Pharma, Aquisitive Therapeutics, GSK, Denali Therapeutics, and BrainStorm Cell Therapeutics, are actively shaping the future of amyotrophic lateral sclerosis treatment through advancements in neurodegenerative research and synaptic regeneration. These companies are driving forward clinical development, regulatory progress, and therapeutic innovation in the amyotrophic lateral sclerosis treatment landscape. In a significant regulatory milestone, on June 3rd, 2025, Spinogenix announced that the European Medicines Agency (EMA) granted Orphan Drug Designation (ODD) to its lead candidate, SPG302, for the treatment of amyotrophic lateral sclerosis (ALS). This followed the recent completion of a Phase 2 clinical trial in Australia (NCT06903286), highlighting SPG302's potential as the first synaptic regenerative therapy for ALS. Further advancing ALS research, Transposon Therapeutics announced on May 28th, 2025, positive Phase 2 results for its investigational therapy TPN-101 in C9orf72-related ALS, showing reductions in neurofilament light chain (NfL) and interleukin-6 (IL-6) levels. TPN-101 is slated to enter the HEALEY ALS Platform Trial in Q4 2025, signaling continued momentum in adaptive trial designs. Similarly, on April 7th, 2025, Immunity Pharma reported positive Phase 2a clinical trial results for its investigational amyotrophic lateral sclerosis treatment, IPL344. The data showed that IPL344 significantly slowed the progression of ALS, as measured by the ALSFRS-R, by 58–64% compared to a matched control group from the PRO-ACT database. Additionally, treatment with IPL344 led to a 27% reduction in neurofilament light chain (NfL) levels after just two months, suggesting a potential disease-modifying effect. In a key regional development, the Japanese Ministry of Health, Labor and Welfare approved QALSODY in December 2024, expanding global access to this innovative therapy and underscoring Japan's regulatory support for amyotrophic lateral sclerosis treatments. These advancements underscore the rapid progress in amyotrophic lateral sclerosis drug development, driven by novel mechanisms of action and supportive global regulatory pathways. As leading companies continue to invest in clinical research and therapeutic innovation, the ALS treatment landscape is poised for transformation, offering renewed hope to patients worldwide. Future Outlook: Evolving Amyotrophic Lateral Sclerosis Treatments and the Rise of Regenerative Approaches The future of amyotrophic lateral sclerosis treatment is poised for significant change, with ongoing advancements in genetic therapies and an early promise in stem cell research. While current market leaders like Riluzole, Edaravone, and Qalsody continue to support disease management; high treatment costs and limited efficacy underscore the urgent need for more effective, accessible options. Stem cell therapies, particularly those involving mesenchymal stem cells (MSCs), remain in the research phase but are showing encouraging results. In 2025, DVCStem reported that MSCs may help delay onset, slow progression, and extend lifespan in ALS patients. Similarly, Mitsubishi Chemical's CL2020 completed Phase 2 trials with strong safety outcomes, reinforcing confidence in cell-based interventions. Looking ahead, the amyotrophic lateral sclerosis treatment landscape is expected to shift toward more personalized, regenerative, and combination-based approaches. As clinical trials advance and GMP-certified manufacturing expands, stem cell therapies may become a viable addition to standard care. By 2030, greater integration of genetic targeting, cellular therapies, and streamlined delivery models could redefine ALS management and offer new hope to patients worldwide. Key Competitors Medicinova Mitsubishi Tanabe Pharma Ionis Alector Sanofi ITF Pharma Aquisitive Therapuetice GSK Denali Therapuetics BrainStrom Segmentation of Amyotrophic Lateral Sclerosis Treatment Market By Treatment Type Medication Therapy Riluzole Radicava Qalsody Stem Cell Therapy By Distribution Channel Hospital & Clinics Pharmacies By Region North America South America Europe Asia-Pacific Middle East and Africa Have Questions? 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