Otsuka heads to FDA as Ionis-partnered ALS drug hits primary phase 3 endpoint

Live updates

1. 1d ago

   Otsuka and Ionis Target FDA Submission for ALS Drug

   Otsuka Pharmaceutical and Ionis Pharmaceuticals are heading to the FDA after their drug ulefnersen met its primary phase three endpoint in the FUSION trial for FUS-associated amyotrophic lateral sclerosis. The treatment could become the first disease-modifying option for this rare genetic subtype. Meanwhile, Swiss biotech AL-S Pharma appointed Harry Welten as chief executive in October 2026 and outlined plans for a phase three trial of AP-101 in sporadic and SOD1-associated ALS for 2027. AL-S Pharma was founded in 2016 by Neurimmune and TVM Capital Life Science.

   Why it matters

   Amyotrophic lateral sclerosis drug development continues to advance across distinct genetic and sporadic patient populations. Companies are targeting specific disease mechanisms, such as misfolded SOD1 proteins and FUS mutations. Regulatory filings for targeted therapies mark a shift toward treating precise biological subtypes of the disease.

   What is confirmed

   • Otsuka Pharmaceutical and Ionis Pharmaceuticals' ulefnersen met its primary phase 3 endpoint in the FUSION trial for FUS-associated amyotrophic lateral sclerosis.
   • An Early Access Program was created for eligible patients following the trial results.
   • AL-S Pharma is a Swiss biotech founded in 2016 by Neurimmune and TVM Capital Life Science.
   • AL-S Pharma named finance executive Harry Welten chief executive in October 2026.
   • AL-S Pharma outlined the design of AP101-03, a planned phase 3 trial of AP-101 in sporadic and SOD1-associated ALS, at the 2026 NEALS Annual Meeting.

   What to watch next

   • Otsuka and Ionis regulatory submission to the FDA for ulefnersen
   • AL-S Pharma confirmatory Phase III trial for AP-101 in 2027
   Sources used for this update (3)

   1. www.neurologylive.com — Neuromuscular | Clinical | NeurologyLive - Clinical Neurology ...
   2. www.neurologylive.com — AL-S Pharma Details Planned Phase 3 Trial Design for AP-101 ...
   3. www.thepharmaletter.com — AL-S Pharma | The Pharmaletter
   confidence 100%
2. 7d ago

   Otsuka heads to FDA as Ionis-partnered ALS drug hits primary phase 3 endpoint

   Otsuka Pharmaceutical and Ionis Pharmaceuticals' ulefnersen met its primary phase 3 endpoint in the FUSION trial for FUS-associated amyotrophic lateral sclerosis. This could become the first disease-modifying option for this rare genetic subtype of ALS. An Early Access Program has been created for eligible patients. The companies are moving toward regulatory submission.

   Why it matters

   The development of ulefnersen is significant as it targets a specific genetic subtype of ALS, offering potential hope for patients with this rare condition. Currently, treatment options for ALS are limited, and a disease-modifying therapy would represent a major advancement. The partnership between Otsuka and Ionis has led to this promising result, and their next steps will be crucial in bringing this treatment to market.

   What is confirmed

   • Otsuka Pharmaceutical and Ionis Pharmaceuticals' ulefnersen met its primary phase 3 endpoint in the FUSION trial for FUS-associated amyotrophic lateral sclerosis.
   • An Early Access Program has been created for eligible patients.

   What to watch next

   • FDA review and potential approval of ulefnersen
   • regulatory submission details and timeline
   • availability and uptake of ulefnersen through the Early Access Program
   Sources used for this update (5)

   1. www.pdufa.bio — FDA Decisions Archive: 485 Tracked, Updated Daily | pdufa.bio
   2. www.distillintelligence.com — Latest Otsuka Holdings (Otsuka) News & Announcements
   3. stockanalysis.com — Ionis Pharmaceuticals (FRA:ISI) Stock Price, Chart & News
   4. ctv.veeva.com — A Trial of the Efficacy and Safety of SEP-363856 in Acutely ...
   5. pharmaphorum.com — Treating mechanisms, not symptoms: ALS R&D today
   confidence 100%
3. 12d ago

   Otsuka's ALS drug hits phase 3 endpoint, heads to FDA

   Otsuka Pharmaceutical and Ionis Pharmaceuticals' ulefnersen met its primary phase 3 endpoint in the FUSION trial for FUS-associated amyotrophic lateral sclerosis. This could become the first disease-modifying option for this rare genetic subtype of ALS. An Early Access Program has been created for eligible patients. The companies are moving toward regulatory submission.

   Why it matters

   The development of ulefnersen is significant as it addresses a critical need for treatments for FUS-associated ALS, a rare and severe form of the disease. If approved, it could provide a new treatment option for patients with this specific subtype of ALS. The partnership between Otsuka and Ionis has been focused on bringing this therapy to market. The FDA submission is a key step in this process.

   What is confirmed

   • Otsuka Pharmaceutical and Ionis Pharmaceuticals' ulefnersen met its primary phase 3 endpoint in the FUSION trial for FUS-associated amyotrophic lateral sclerosis.
   • An Early Access Program has been created for eligible patients.

   What to watch next

   • FDA approval decision
   • regulatory submission details
   • availability of ulefnersen through the Early Access Program
   Sources used for this update (4)

   1. www.pharmabiz.com — Otsuka presents final phase 3 VISIONARY results at ASN ...
   2. patientworthy.com — Otsuka’s Phase 3 VISIONARY Study Suggests Voyxact May Alter ...
   3. www.otsuka.com — Investor Relations | Otsuka Holdings Co., Ltd.
   4. www.biospace.com — Ionis partner Roche announces new Phase 3 data showing ...
   confidence 100%
4. 15d ago

   Otsuka's ALS drug hits phase 3 endpoint, FDA submission next

   Otsuka Pharmaceutical and Ionis Pharmaceuticals' ulefnersen met its primary phase 3 endpoint in the FUSION trial for FUS-associated amyotrophic lateral sclerosis. This could become the first disease-modifying option for this rare genetic subtype of ALS. An Early Access Program has been created for eligible patients. The companies are moving toward regulatory submission.

   Why it matters

   The FUSION trial's success marks a significant milestone in the development of treatments for amyotrophic lateral sclerosis. ALS is a progressive neurological disease with limited treatment options. The availability of ulefnersen could provide new hope for patients with FUS-associated ALS. Otsuka and Ionis are collaborating on the development of this RNA-targeted therapy.

   What is confirmed

   • Otsuka Pharmaceutical and Ionis Pharmaceuticals' ulefnersen met its primary phase 3 endpoint in the FUSION trial for FUS-associated amyotrophic lateral sclerosis.
   • Ulefnersen could become the first disease-modifying option for FUS-associated ALS.
   • Otsuka has created an Early Access Program for eligible patients.

   What to watch next

   • FDA submission and approval for ulefnersen
   • results from ongoing trials for VOYXACT and zipalertinib
   Sources used for this update (3)

   1. www.otsuka.co.jp — 2026｜News Releases | Otsuka Pharmaceutical Co., Ltd.
   2. www.otsuka-us.com — Otsuka Presents Landmark Final Phase 3 VISIONARY Results at ...
   3. www.marketscreener.com — Otsuka's VOYXACT Sustains Kidney Function in Phase 3 of Trial
   confidence 100%
5. 21d ago

   Otsuka prepares FDA submission for ulefnersen following phase 3 success

   Otsuka Pharmaceutical and Ionis Pharmaceuticals are moving toward regulatory submission for ulefnersen after the drug met its primary phase 3 endpoint in the FUSION trial. The RNA-targeted therapy demonstrated significant benefit for FUS-associated amyotrophic lateral sclerosis. This treatment could become the first disease-modifying option for this rare genetic subtype of ALS. To provide immediate availability, Otsuka has created an Early Access Program for eligible patients. Separately, Taiho Pharmaceutical has started a US application for zipalertinib used with chemotherapy for lung cancer.

   Why it matters

   FUS-associated ALS is a rare genetic form of the disease with no current disease-modifying treatments. Ulefnersen targets the RNA to slow progression. Success in this trial marks a potential shift in how this specific subtype is managed clinically.

   What is confirmed

   • Ulefnersen met its primary phase 3 endpoint in the FUSION trial.
   • The drug showed significant benefit for FUS-associated amyotrophic lateral sclerosis.
   • Otsuka established an Early Access Program for eligible patients.
   • Taiho Pharmaceutical initiated a US application for zipalertinib combined with chemotherapy for lung cancer.

   What to watch next

   • FDA decision on the ulefnersen regulatory submission
   • Clinical outcome data from the Early Access Program
   • FDA review status of the zipalertinib application
   Sources used for this update (2)

   1. www.otsuka.com — Otsuka Group News - Otsuka Holdings Co., Ltd.
   2. stockanalysis.com — Otsuka Holdings (OTSKF) Stock Price & Overview
   confidence 100%
6. 23d ago

   Otsuka Moves Ulefnersen Toward FDA Following FUSION Trial Success

   Otsuka Pharmaceutical and Ionis Pharmaceuticals are advancing ulefnersen toward a regulatory submission after the RNA-targeted therapy met its primary phase 3 endpoint in the FUSION trial. The treatment showed a significant benefit across a combined measure for FUS-associated amyotrophic lateral sclerosis, establishing its potential as the first disease-modifying option for this rare genetic subtype. Otsuka also established an Early Access Program for eligible patients, while subsidiary Taiho Pharmaceutical initiated a US application submission for zipalertinib combined with chemotherapy in lung cancer treatment.

   Why it matters

   The FUSION trial success marks a potential breakthrough for patients suffering from FUS-associated amyotrophic lateral sclerosis by targeting the underlying genetic mechanism of the disease. Advancing ulefnersen toward FDA submission represents a crucial operational step for Otsuka and Ionis as they seek to deliver the first disease-modifying therapy for this specific patient population. Concurrently, the separate oncology pipeline progress by Taiho Pharmaceutical broadens the scope of the corporate group's regulatory activities in the United States.

   What is confirmed

   • Otsuka Pharmaceutical and Ionis Pharmaceuticals are advancing ulefnersen toward an FDA submission after the RNA-targeted therapy successfully met its primary phase 3 endpoint in the FUSION trial.
   • The treatment demonstrated significant benefit across a combined measure for FUS-associated amyotrophic lateral sclerosis.
   • Otsuka established an Early Access Program for eligible patients.
   • Otsuka subsidiary Taiho Pharmaceutical initiated a US application submission for zipalertinib combined with chemotherapy as a lung cancer treatment.

   What to watch next

   • Official FDA receipt and filing acceptance of the ulefnersen regulatory application
   • Further clinical safety and efficacy data releases concerning the FUSION trial and long-term access programs
   Sources used for this update (4)

   1. www.fda.gov — What's New Related to Drugs | FDA - U.S. Food and Drug ...
   2. ctv.veeva.com — A Study of JNT-517 in Participants With Phenylketonuria (PKU)
   3. ctv.veeva.com — A Long-Term Study of JNT-517 in Participants With Phenylketonuria
   4. www.pdufa.bio — PDUFA Dates 2026: FDA Decision Calendar, Updated Daily ...
   confidence 100%
7. 26d ago

   Otsuka Targets FDA Review for Ionis-Partnered ALS Drug

   Otsuka Pharmaceutical and Ionis Pharmaceuticals are advancing ulefnersen toward an FDA submission after the RNA-targeted therapy successfully met its primary phase 3 endpoint in the FUSION trial for FUS-associated amyotrophic lateral sclerosis. The treatment demonstrated significant benefit across a combined measure, establishing its potential as the first disease-modifying option for this rare genetic subtype. Otsuka also established an Early Access Program for eligible patients. Meanwhile, Otsuka subsidiary Taiho Pharmaceutical initiated a US application submission for zipalertinib combined with chemotherapy as a lung cancer treatment.

   Why it matters

   Ulefnersen addresses FUS-associated amyotrophic lateral sclerosis, representing a crucial step for patients suffering from this rare genetic subtype. Otsuka and Ionis are working to secure regulatory approval to make the therapy widely available. Alongside this neurodegenerative drug candidate, the corporate group is expanding its oncology pipeline through Taiho Pharmaceutical.

   What is confirmed

   • Otsuka Pharmaceutical and Ionis Pharmaceuticals are advancing ulefnersen toward an FDA submission after it met its primary phase 3 endpoint in the FUSION trial.
   • Ulefnersen is an RNA-targeted therapy intended for FUS-associated amyotrophic lateral sclerosis.
   • Otsuka established an Early Access Program for eligible patients.
   • Taiho Pharmaceutical has initiated a US application submission for zipalertinib combined with chemotherapy as a lung cancer treatment.

   What to watch next

   • Official FDA acceptance of the ulefnersen submission
   • Regulatory updates regarding the zipalertinib combination application
   Sources used for this update (14)

   1. www.biospace.com — Neuroscience - BioSpace
   2. www.marketbeat.com — Ionis Pharmaceuticals (IONS) FDA Approvals - MarketBeat
   3. www.biospace.com — Press Releases - BioSpace
   4. www.marketscreener.com — Otsuka Holdings Co., Ltd.: Official Announcements - Stock ...
   5. www.marketscreener.com — Latest news about Otsuka Holdings Co., Ltd. - MarketScreener
   6. www.otsuka-us.com — Home | Otsuka US
   7. otsuka-us.com — Products | Otsuka US
   8. en.wikipedia.org — Otsuka Pharmaceutical - Wikipedia
   9. www.otsuka.co.jp — Otsuka Pharmaceutical Co., Ltd.
   10. www.otsuka.com — Otsuka Holdings Co., Ltd.
   11. www.otsuka-america.com — Otsuka America
   12. www.medical.otsuka-us.com — Home Page | Otsuka
   confidence 100%
8. 33d ago

   Otsuka Moves Forward with Regulatory Submissions

   Otsuka Pharmaceutical and Ionis Pharmaceuticals are advancing ulefnersen toward an FDA submission after the RNA-targeted therapy successfully met its primary phase 3 endpoint in the FUSION trial for FUS-associated amyotrophic lateral sclerosis. The treatment showed significant benefit across a combined measure and holds potential as the first disease-modifying option for this rare genetic subtype. Additionally, Otsuka established an Early Access Program for eligible patients. Meanwhile, Otsuka subsidiary Taiho Pharmaceutical has initiated a US application submission for zipalertinib combined with chemotherapy as a lung cancer treatment.

   Why it matters

   The FUSION trial success marks a major milestone for patients with FUS-associated amyotrophic lateral sclerosis, a rare and severe genetic condition that previously lacked disease-modifying therapies. Otsuka and Ionis are positioning ulefnersen for regulatory approval while simultaneously expanding patient access through an Early Access Program. In a separate development, Otsuka subsidiary Taiho Pharmaceutical is pursuing a US drug application for zipalertinib in lung cancer treatment.

   What is confirmed

   • Otsuka Pharmaceutical and Ionis Pharmaceuticals reported positive phase 3 results for ulefnersen in the FUSION trial targeting FUS-associated amyotrophic lateral sclerosis.
   • Ulefnersen met its primary endpoint, demonstrating a significant benefit across a combined measure and displaying a favorable safety and tolerability profile.
   • Otsuka is preparing an FDA submission for ulefnersen and has established an Early Access Program for eligible patients.
   • Otsuka subsidiary Taiho Pharmaceutical started the US submission of a new drug application for zipalertinib in combination with chemotherapy.

   What to watch next

   • FDA review decisions regarding the ulefnersen and zipalertinib submissions.
   • Updates on patient enrollment in the ulefnersen Early Access Program.
   Sources used for this update (2)

   1. stockanalysis.com — Ionis Pharmaceuticals (IONS) Stock Price & Overview
   2. nl.marketscreener.com — Dochteronderneming Otsuka start indiening Amerikaanse ...
   confidence 100%
9. 35d ago

   Otsuka and Ionis Report Phase 3 Win for FUS-ALS Drug

   Otsuka Pharmaceutical and Ionis Pharmaceuticals reported positive phase 3 results for ulefnersen in the FUSION trial targeting FUS-associated amyotrophic lateral sclerosis. The RNA-targeted therapy met its primary endpoint, demonstrating a significant benefit across a combined measure. The drug previously met its primary endpoint in the phase 3 study, displayed a favorable safety and tolerability profile, and positioned itself as the first potential disease-modifying treatment for this rare genetic subtype. Otsuka is now preparing an FDA submission and has established an Early Access Program for eligible patients.

   Why it matters

   FUS-associated amyotrophic lateral sclerosis is a rare genetic subtype of the disease that currently lacks any approved treatments. The success of this antisense oligonucleotide therapy marks a significant milestone for RNA medicine in rare disease families. Otsuka and Ionis are moving toward regulatory review following the positive data from the Fusion study.

   What is confirmed

   • Ulefnersen met its primary endpoint in the phase 3 FUSION trial for FUS-associated amyotrophic lateral sclerosis.
   • Otsuka Pharmaceutical and Ionis Pharmaceuticals reported positive phase 3 results from the Fusion study.

   What to watch next

   • Official FDA submission filing details by Otsuka
   • Regulatory review timeline and decisions
   • Updates on the Early Access Program for eligible patients
   Sources used for this update (5)

   1. www.biospace.com — Phase III Clinical Trials - BioSpace
   2. www.finanznachrichten.de — IONIS PHARMACEUTICALS INC
   3. generationhope.co — Ionis ASO Therapy Meets Phase 3 Goal in Rare FUS-ALS
   4. biobriefs.com — FDA decision risk-free? Otsuka and Ionis report phase III win ...
   5. touchneurology.com — Ulefnersen meets phase 3 endpoint in genetically defined ...
   confidence 100%
10. 41d ago

    Otsuka's Ionis-partnered ALS drug hits phase 3 endpoint, FDA submission next

    Otsuka Pharmaceutical and Ionis Pharmaceuticals' partnered drug ulefnersen met its primary endpoint in a phase 3 study for FUS-associated ALS, a rare genetic subtype with no approved treatments. The drug demonstrated a favorable safety and tolerability profile. Otsuka is preparing an FDA submission and has established an Early Access Program for eligible patients. This development positions ulefnersen as the first potential disease-modifying treatment for FUS-ALS.

    Why it matters

    FUS-ALS is a severe and rare subtype of amyotrophic lateral sclerosis, a progressive neurological disease. Current treatments for ALS primarily focus on managing symptoms, making ulefnersen a significant potential advancement. The partnership between Otsuka and Ionis leverages the strengths of both companies in developing RNA-targeted therapies.

    What is confirmed

    • Ulefnersen met its primary endpoint in a phase 3 study for FUS-associated ALS.
    • The phase 3 FUSION study demonstrated a favorable safety and tolerability profile for ulefnersen.
    • Otsuka is preparing an FDA submission for ulefnersen.
    • An Early Access Program for ulefnersen has been established for eligible FUS-ALS patients.

    What to watch next

    • FDA submission and review timeline for ulefnersen
    • Ulefnersen's potential approval and market launch
    • Long-term efficacy and safety data for ulefnersen
    Sources used for this update (3)

    1. biobriefs.com — Phase 3 win in FUS-ALS with antisense therapy | BioBriefs
    2. marketontology.com — Ionis reports positive Phase III trial results for partnered ...
    3. simplywall.st — Ionis Pharmaceuticals (IONS) Posted Two Positive Phase 3 ...
    confidence 100%
11. 46d ago

    Otsuka and Ionis move ulefnersen toward FDA approval after Phase 3 success

    Otsuka is preparing an FDA submission for ulefnersen following positive topline results from the Phase 3 FUSION study. The drug met its primary endpoint and demonstrated a favorable safety and tolerability profile. Developed in partnership with Ionis Pharmaceuticals, ulefnersen is positioned as the first potential disease modifying treatment for FUS-ALS. To provide immediate options for patients, Otsuka has established an Early Access Program for eligible individuals living with FUS-ALS while the formal regulatory process proceeds.

    Why it matters

    FUS-ALS is a specific form of amyotrophic lateral sclerosis. This development represents a shift toward targeted therapies for genetic subtypes of the disease. Regulatory approval would mark a significant milestone in treating a condition that currently lacks disease modifying options.

    What is confirmed

    • Ulefnersen met its primary endpoint in the Phase 3 FUSION study.
    • Otsuka and Ionis Pharmaceuticals partnered to develop the drug.
    • Ionis reported that ulefnersen has a favorable safety and tolerability profile.
    • Otsuka created an Early Access Program for eligible people with FUS-ALS.

    What to watch next

    • FDA decision on the ulefnersen application
    • Full publication of the FUSION study data
    Sources used for this update (6)

    1. Endpoints News — Amgen’s Phase 3 Sjögren’s win; Novartis’ $900M radioligand deal
    2. Fierce Biotech — Otsuka heads to FDA as Ionis-partnered ALS drug hits primary phase 3 endpoint
    3. Yahoo Finance — Ionis announces positive topline results from Phase 3 FUSION study of ulefnersen marking significant milestone in advancing first potential disease modifying treatment for FUS-ALS
    4. TradingView — Ionis Pharmaceuticals Says Ulefnersen Demonstrates Favorable Safety And Tolerability Profile
    5. BioSpace — Otsuka Establishes Ulefnersen Early Access Program (EAP) for Eligible People Living with FUS-ALS
    6. Yahoo — Otsuka and Ionis report positive data in Phase III FUSION trial of ulefnersen
    confidence 100%