Latest news with #Ionis
Globe and Mail
3 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 Wire
16-07-2025
- Business
- Business Wire
Ionis to hold second quarter 2025 financial results webcast
CARLSBAD, Calif.--(BUSINESS WIRE)--Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced today that it will host a live webcast on Wednesday, July 30 th at 11:30 a.m. Eastern Time to discuss its second quarter 2025 financial results and progress on key programs. The webcast may be accessed at A replay will be available for a limited time at the same address. About Ionis Pharmaceuticals, Inc. For three decades, Ionis has invented medicines that bring better futures to people with serious diseases. Ionis currently has six marketed medicines and a leading pipeline in neurology, cardiology, and select areas of high patient need. As the pioneer in RNA-targeted medicines, Ionis continues to drive innovation in RNA therapies in addition to advancing new approaches in gene editing. A deep understanding of disease biology and industry-leading technology propels our work, coupled with a passion and urgency to deliver life-changing advances for patients. To learn more about Ionis, visit and follow us on X (Twitter), LinkedIn and Instagram.
Business Wire
12-06-2025
- Business
- Business Wire
Ionis announces Dr. Richard Geary, chief development officer, to retire; Dr. Holly Kordasiewicz to assume role in January 2026
CARLSBAD, Calif.--(BUSINESS WIRE)-- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that Richard Geary, Ph.D., executive vice president and chief development officer, will retire effective January 2026 and that Holly Kordasiewicz, Ph.D., currently senior vice president, neurology, will succeed him in the role. 'Richard has made tremendous contributions to Ionis over his 30-year tenure during which he spearheaded dozens of development programs and brought six innovative medicines through regulatory approvals, including TRYNGOLZA, our first independent commercial medicine. We thank him for his leadership and unwavering dedication to patients in need,' said Brett P. Monia, Ph.D., chief executive officer, Ionis. 'Holly is a deeply respected and innovative leader who will champion our vision to bring a steady cadence of transformational medicines to people in need and support Ionis' continued growth as a fully integrated biotechnology company. Her broad experience across research and development, strong operational and management skills, and deep neurology expertise, position Holly to successfully guide our portfolio, which includes eight wholly owned neurology medicines in clinical development.' Dr. Kordasiewicz has 20 years of experience in research and development and joined Ionis in 2011. An expert in neurology drug development, she currently oversees Ionis' industry-leading neurology program, including medicines for Alexander disease, Angelman syndrome, Alzheimer's disease, prion disease and multiple system atrophy. Dr. Kordasiewicz also played an integral role in the discovery and development of our Biogen-partnered programs QALSODY ® (tofersen) for a genetic form of amyotrophic lateral sclerosis and IONIS-MAPT Rx (BIIB080), a promising Alzheimer's disease medicine in Phase 2 development. Dr. Kordasiewicz holds a B.A. from SUNY-Geneseo and a Ph.D. in Neuroscience from the University of Minnesota. She completed postdoctoral work at the University of Minnesota and the University of California San Diego. Dr. Geary joined Ionis in 1995 and has been involved in nearly every aspect of discovery and development. Under Dr. Geary's leadership, Ionis submitted more than 50 investigational new drug applications to regulatory agencies and achieved six FDA and EU approvals, including TRYNGOLZA ® (olezarsen). Dr. Geary will serve as a strategic consultant to Ionis throughout 2026 to support a seamless transition. About Ionis Pharmaceuticals, Inc. For three decades, Ionis has invented medicines that bring better futures to people with serious diseases. Ionis currently has six marketed medicines and a leading pipeline in neurology, cardiology and select areas of high patient need. As the pioneer in RNA-targeted medicines, Ionis continues to drive innovation in RNA therapies in addition to advancing new approaches in gene editing. A deep understanding of disease biology and industry-leading technology propels our work, coupled with a passion and urgency to deliver life-changing advances for patients. To learn more about Ionis, visit and follow us on X (Twitter), LinkedIn and Instagram. Ionis Forward-looking Statements This press release includes forward-looking statements regarding Ionis' business and the therapeutic and commercial potential of our commercial medicines, additional medicines in development and technologies. Any statement describing Ionis' goals, expectations, financial or other projections, intentions or beliefs is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to certain risks and uncertainties including those inherent in the process of discovering, developing and commercializing medicines that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such medicines. Ionis' forward-looking statements also involve assumptions that, if they never materialize or prove correct, could cause its results to differ materially from those expressed or implied by such forward-looking statements. Although Ionis' forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Ionis. Except as required by law, we undertake no obligation to update any forward-looking statements for any reason. As a result, you are cautioned not to rely on these forward-looking statements. These and other risks concerning Ionis' programs are described in additional detail in Ionis' annual report on Form 10-K for the year ended December 31, 2024, and most recent Form 10-Q, which are on file with the Securities and Exchange Commission. Copies of these and other documents are available from the Company. In this press release, unless the context requires otherwise, "Ionis," "Company," "we," "our" and "us" all refer to Ionis Pharmaceuticals and its subsidiaries. Ionis Pharmaceuticals ® and TRYNGOLZA ® are trademarks of Ionis Pharmaceuticals, Inc. QALSODY ® is a registered trademark of Biogen.
Yahoo
12-06-2025
- Business
- Yahoo
Ionis doses first subject in Phase III trial of Angelman syndrome therapy
Ionis Pharmaceuticals has dosed the first subject in the global, randomised Phase III REVEAL trial of investigational ION582, aimed at treating individuals with Angelman syndrome (AS), a rare neurodevelopmental disorder. The placebo-controlled, double-blind trial will recruit nearly 200 AS adults and children with a maternal UBE3A gene deletion or mutation. Over a 52-week treatment period, subjects will be randomised in a 2:1 ratio and given either the therapy or a placebo. Quarterly doses of either 40mg or 80mg of ION582 will be administered to those in the active treatment groups. After the initial treatment phase, eligible subjects will have the opportunity to enter a long-term extension segment of the trial, where the therapy will be administered for up to two years. The primary goal of the trial is to assess improvements in expressive communication using the Bayley Scales for Infant and Toddler Development-4 (Bayley-4), a direct clinician-administered assessment tool. Expressive communication deficits are notably the most burdensome symptoms for caregivers of individuals with AS. The trial will also evaluate secondary endpoints, including overall disease severity, communication, cognition, motor functioning, sleep, and daily living skills, as well as other exploratory endpoints. ION582 has previously shown promise in the multiple ascending dose (MAD) portion of the Phase I/II open-label HALOS trial, demonstrating clinical improvement across various functional domains such as cognition, motor function, and communication. The RNA-targeted antisense medicine, ION582, works by inhibiting the expression of the UBE3A antisense transcript (UBE3A-ATS) to potentially increase the UBE3A protein production, to treat AS. Ionis Pharmaceuticals neurology senior vice-president Holly Kordasiewicz: 'This placebo-controlled study will evaluate ION582 in both children and adults with either UBE3A deletion or mutation and builds on earlier encouraging findings from our Phase I/II HALOS study. 'With ION582, we continue to advance our leading wholly owned neurology pipeline, which includes eight medicines in clinical development across a range of rare and more prevalent diseases.' Last year, Ionis announced the design for the Phase III trial of ION582 for the treatment of AS, following discussions with the US Food and Drug Administration. "Ionis doses first subject in Phase III trial of Angelman syndrome therapy" was originally created and published by Clinical Trials Arena, a GlobalData owned brand. The information on this site has been included in good faith for general informational purposes only. It is not intended to amount to advice on which you should rely, and we give no representation, warranty or guarantee, whether express or implied as to its accuracy or completeness. You must obtain professional or specialist advice before taking, or refraining from, any action on the basis of the content on our site. 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
Business Wire
11-06-2025
- Business
- Business Wire
First patient dosed in pivotal Phase 3 REVEAL clinical study of ION582 in Angelman syndrome
CARLSBAD, Calif.--(BUSINESS WIRE)-- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that the first participant has been dosed in the global Phase 3 REVEAL study, which is designed to evaluate the efficacy and safety of ION582, an investigational medicine for the treatment of people living with Angelman syndrome (AS), a serious and rare neurodevelopmental disorder that leads to significant physical and cognitive impairments. 'Dosing the first person with Angelman syndrome in our pivotal REVEAL study marks an important milestone for this underserved community, who currently have no approved disease modifying treatments,' said Holly Kordasiewicz, Ph.D., senior vice president of neurology, Ionis. 'This placebo-controlled study will evaluate ION582 in both children and adults with either UBE3A deletion or mutation and builds on earlier encouraging findings from our Phase 1/2 HALOS study. This milestone reflects our commitment to those impacted by Angelman syndrome and builds on our legacy of innovation in neurology including the development of SPINRAZA and QALSODY. With ION582, we continue to advance our leading wholly owned neurology pipeline, which includes eight medicines in clinical development across a range of rare and more prevalent diseases.' REVEAL (NCT06914609) is a global, randomized, double-blind, placebo-controlled Phase 3 study that will enroll approximately 200 children and adults with AS that have a maternal UBE3A gene deletion or mutation. During the 52-week treatment period, participants will be randomized 2:1 to receive either ION582 or placebo. Participants in the active treatment groups will receive quarterly 40 mg or 80 mg doses of ION582. Following the treatment period, eligible participants will transition into the long-term extension portion of the study where all participants will receive ION582 for up to 2 years. The primary endpoint is improvement in expressive communication as assessed by the Bayley Scales for Infant and Toddler Development-4 (Bayley-4), an objective and direct clinician-administered assessment of clinical functioning. Deficits in expressive communication are reported to be the symptoms most challenging to caregivers of people with AS. Secondary endpoints include overall disease severity, cognition, communication, sleep, motor functioning and daily living skills, in addition to other exploratory endpoints. In the completed multiple ascending dose (MAD) portion of the Phase 1/2 open-label HALOS study, ION582 provided consistent and encouraging clinical improvement on all functional domains including communication, cognition and motor function and showed favorable safety and tolerability at all dose levels in the study. For more information on the REVEAL clinical study, please visit and About ION582 ION582 is an investigational RNA-targeted antisense medicine designed to inhibit the expression of the UBE3A antisense transcript (UBE3A-ATS) and increase production of UBE3A protein, for the potential treatment of Angelman syndrome (AS). The U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) granted Orphan Drug designation to ION582. Additionally, the FDA granted Fast Track and Rare Pediatric designations to ION582. About Angelman Syndrome (AS) AS is a rare, genetic neurological disease that affects an estimated 1 in 21,000 people worldwide and is caused by the loss of function of the maternally inherited UBE3A gene. AS typically presents in infancy and is characterized by profound intellectual disability, balance issues, motor impairment and debilitating seizures. Most people with AS are unable to speak. Individuals with AS have a normal lifespan but require complete care from a caregiver. Some symptoms can be managed with existing medicines; however, there are no approved disease modifying therapies. About Ionis Neurology Ionis has been at the forefront of discovering and developing leading neurological disease medicines, including SPINRAZA ® (nusinersen), the first approved treatment for spinal muscular atrophy, WAINUA™ (eplontersen), a medicine to treat hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN), and QALSODY ® (tofersen) for SOD1-ALS. The clinical-stage portfolio includes 13 therapies, of which eight are wholly owned by Ionis. Ionis' investigational portfolio includes medicines for which there are few or no disease modifying treatments, such as rare diseases including Prion disease and Alexander disease and more common conditions such as Alzheimer's and Parkinson's disease. About Ionis Pharmaceuticals, Inc. For three decades, Ionis has invented medicines that bring better futures to people with serious diseases. Ionis currently has six marketed medicines and a leading pipeline in neurology, cardiology, and select areas of high patient need. As the pioneer in RNA-targeted medicines, Ionis continues to drive innovation in RNA therapies in addition to advancing new approaches in gene editing. A deep understanding of disease biology and industry-leading technology propels our work, coupled with a passion and urgency to deliver life-changing advances for patients. To learn more about Ionis, visit and follow us on X (Twitter), LinkedIn and Instagram. Ionis Forward-looking Statements This press release includes forward-looking statements regarding Ionis' business and the therapeutic and commercial potential of ION582, our commercial medicines, additional medicines in development and technologies. Any statement describing Ionis' goals, expectations, financial or other projections, intentions or beliefs is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to certain risks and uncertainties including those inherent in the process of discovering, developing and commercializing medicines that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such medicines. Ionis' forward-looking statements also involve assumptions that, if they never materialize or prove correct, could cause its results to differ materially from those expressed or implied by such forward-looking statements. Although Ionis' forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Ionis. Except as required by law, we undertake no obligation to update any forward-looking statements for any reason. As a result, you are cautioned not to rely on these forward-looking statements. These and other risks concerning Ionis' programs are described in additional detail in Ionis' annual report on Form 10-K for the year ended December 31, 2024, and most recent Form 10-Q, which are on file with the Securities and Exchange Commission. Copies of these and other documents are available from the Company. In this press release, unless the context requires otherwise, "Ionis," "Company," "we," "our" and "us" all refer to Ionis Pharmaceuticals and its subsidiaries. Ionis Pharmaceuticals ® is a trademark of Ionis Pharmaceuticals, Inc. SPINRAZA ® and QALSODY ® are registered trademarks of Biogen. WAINUA™ is a registered trademark of the AstraZeneca group of companies.
