{ "categories": { "approved_treatments": [ { "drug_name": "Riluzole", "evidence": "The pivotal trial studied 959 people. A Cochrane synthesis estimated roughly two to three months of median survival benefit in the trial populations; that average is not a prediction for an individual.", "evidence_date": "2021-07-01", "id": "riluzole", "india_note": "Ask the prescribing team and a licensed pharmacy about the appropriate formulation, current supply and a written price quotation.", "limitations": [ "It does not regenerate motor neurons. Treatment choice, formulation and monitoring belong in a discussion with the treating neurologist." ], "name": "Riluzole", "source_ids": [ "riluzole-trial", "riluzole-review" ], "sources": [ "https://pubmed.ncbi.nlm.nih.gov/8676624/", "https://www.cochrane.org/evidence/CD001447_riluzole-amyotrophic-lateral-sclerosis-alsmotor-neuron-disease-mnd" ], "status": "Established ALS treatment", "status_kind": "approved", "subtitle": "Rilutek · oral formulations", "summary": "An oral treatment with randomized evidence of a modest survival benefit in ALS." }, { "drug_name": "Edaravone", "evidence": "FDA describes evidence of less decline in daily functioning in a six-month trial. Oral approval used comparable drug exposure to the IV product. This supports slowing decline in the studied population, not recovery of lost function.", "evidence_date": "2022-05-12", "id": "edaravone", "india_note": "Verify the exact product and route locally. US approval of Radicava ORS does not establish its routine availability in India.", "limitations": [ "Limited evidence in a particular disease stage does not prove that every person outside the trial criteria cannot benefit. Avoid blanket exclusions based on wheelchair or ventilator use." ], "name": "Edaravone", "source_ids": [ "edaravone-fda" ], "sources": [ "https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-oral-form-treatment-adults-amyotrophic-lateral-sclerosis-als" ], "status": "US approval: IV and oral", "status_kind": "approved", "subtitle": "Radicava · Radicava ORS", "summary": "Edaravone is approved in the US for ALS. The oral suspension provides an alternative to intravenous administration and can also be given through a feeding tube." }, { "drug_name": "Tofersen", "evidence": "The 28-week VALOR trial did not establish a significant primary clinical benefit. Longer follow-up associated earlier treatment with less decline, but the open-label comparison has limitations. Clinical benefit remains under confirmatory study.", "evidence_date": "2025-12-22", "id": "tofersen", "india_note": "Confirm the genetic result with a specialist. Local authorization, supply, import permission and funding must be checked for the individual patient.", "limitations": [ "A biomarker response is not proof of a cure or predictable recovery. Intrathecal treatment requires a specialist team. Investigation in non-SOD1 ALS does not expand the approved indication." ], "name": "Tofersen", "source_ids": [ "tofersen-fda", "tofersen-longterm" ], "sources": [ "https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-treatment-amyotrophic-lateral-sclerosis-associated-mutation-sod1-gene", "https://jamanetwork.com/journals/jamaneurology/fullarticle/2843130" ], "status": "US accelerated approval", "status_kind": "approved", "subtitle": "Qalsody · SOD1-targeted antisense therapy", "summary": "Approved for adults with ALS and a SOD1 mutation. The FDA decision was based on lowering blood neurofilament light (NfL), a marker of nerve injury." }, { "drug_name": "High-dose mecobalamin", "evidence": "JETALS found less ALSFRS-R decline over 16 weeks in a selected early-stage Japanese population. The PMDA review supports the indication while documenting the studied population and safety requirements.", "evidence_date": "2024-10-16", "id": "mecobalamin", "india_note": "Japanese authorization does not mean approval or reliable supply in India. Discuss product identity and any lawful access route with the clinical team.", "limitations": [ "Do not generalize the short trial to all stages or substitute ordinary supplements. Rozebalamin is mecobalamin (E0302), not MT-1186." ], "name": "High-dose mecobalamin", "source_ids": [ "rozebalamin-pmda", "rozebalamin-approval", "jetals" ], "sources": [ "https://www.pmda.go.jp/files/000277177.pdf", "https://www.eisai.com/news/2024/pdf/enews202475pdf.pdf", "https://jamanetwork.com/journals/jamaneurology/fullarticle/2792228" ], "status": "Japan-specific approval", "status_kind": "approved", "subtitle": "Rozebalamin · development code E0302", "summary": "Japan approved this injectable methylcobalamin formulation for slowing functional impairment in ALS. It is distinct from routine vitamin B12 supplementation." } ], "clinical_trials": [ { "evidence": "The registry was updated on 9 September 2026. It describes a randomized, blinded, placebo-controlled trial assessing safety, tolerability and early signals after intrathecal administration.", "evidence_date": "2026-09-09", "id": "trcn1023", "india_note": "No Indian site is listed in the cited record. Recruitment elsewhere does not guarantee eligibility, travel support or access from India.", "limitations": [ "No results are posted. A newly recruiting trial is an opportunity to test a hypothesis, not evidence of clinical benefit." ], "name": "TRCN-1023", "registry": { "countries": [ "Germany", "Netherlands", "United Kingdom" ], "id": "NCT07674667", "phase": [ "PHASE1", "PHASE2" ], "results_posted": false, "status": "RECRUITING", "updated_on": "2026-09-09", "url": "https://clinicaltrials.gov/study/NCT07674667" }, "source_ids": [ "NCT07674667" ], "sources": [ "https://clinicaltrials.gov/study/NCT07674667" ], "status": "Recruiting", "status_kind": "recruiting", "subtitle": "FUNCtion ALS · UNC13A restoration", "summary": "A new Phase 1/2 study evaluates a medicine intended to restore UNC13A function in ALS.", "trial_name": "TRCN-1023" }, { "evidence": "First enrollment was announced on 30 March 2026; the registry still lists recruiting. The earlier HEALEY trial did not meet its primary endpoint. Subgroup observations motivated a more focused confirmatory trial.", "evidence_date": "2026-08-25", "id": "pridopidine", "india_note": "No Indian site is listed in the cited record. Recruitment elsewhere does not guarantee eligibility, travel support or access from India.", "limitations": [ "Subgroup findings are hypothesis-generating. PREVAiLS has not yet established efficacy or supported ALS marketing approval." ], "name": "Pridopidine", "registry": { "countries": [ "Belgium", "Canada", "France", "Germany", "Ireland", "Israel", "Italy", "Netherlands", "Poland", "Spain", "Sweden", "United Kingdom", "United States" ], "id": "NCT07322003", "phase": [ "PHASE3" ], "results_posted": false, "status": "RECRUITING", "updated_on": "2026-08-25", "url": "https://clinicaltrials.gov/study/NCT07322003" }, "source_ids": [ "prevails", "pridopidine-rct", "NCT07322003" ], "sources": [ "https://news.prilenia.com/press-releases/press-release-details/2026/Prilenia-and-Ferrer-Announce-First-Participant-Enrolled-in-the-PREVAiLS-Phase-3-Study-of-Pridopidine-in-ALS/default.aspx", "https://jamanetwork.com/journals/jama/fullarticle/2830509", "https://clinicaltrials.gov/study/NCT07322003" ], "status": "Recruiting", "status_kind": "recruiting", "subtitle": "PREVAiLS · sigma-1 receptor agonist", "summary": "The Phase 3 PREVAiLS study tests pridopidine in people with early, rapidly progressive ALS.", "trial_name": "Pridopidine" }, { "evidence": "Clene’s 14 August update targets an accelerated-approval application in early Q4 2026. Its HEALEY trial did not show significant benefit on the primary 24-week progression endpoint. Later biomarker and survival analyses underpin the planned submission.", "evidence_date": "2026-08-14", "id": "cnm-au8", "india_note": "No routine Indian access or local trial site is established by these sources. Expanded access is distinct from marketing approval.", "limitations": [ "A planned application, FDA meeting or exploratory analysis is not approval. RESTORE-ALS should not be described as enrolling without a verified launch." ], "name": "CNM-Au8", "source_ids": [ "clene-update", "cnm-rct" ], "sources": [ "https://www.sec.gov/Archives/edgar/data/1822791/000143774926027837/ex_967804.htm", "https://jamanetwork.com/journals/jama/fullarticle/2830508" ], "status": "Application planned", "status_kind": "planned", "subtitle": "Clene · gold nanocrystal suspension", "summary": "An investigational therapy targeting cellular energy pathways; CNM-Au8 remains under development for ALS.", "trial_name": "CNM-Au8" }, { "evidence": "The August 2026 sponsor update describes preparations to open the study; the registry lists not yet recruiting. An FDA-agreed Special Protocol Assessment concerns trial design and does not establish that the treatment works.", "evidence_date": "2026-08-14", "id": "nurown", "india_note": "No Indian site is listed in the cited record. Recruitment elsewhere does not guarantee eligibility, travel support or access from India.", "limitations": [ "The new trial has no posted results. Do not present NurOwn as an approved stem-cell treatment or imply that protocol agreement guarantees approval." ], "name": "NurOwn / debamestrocel", "registry": { "countries": [ "United States" ], "id": "NCT06973629", "phase": [ "PHASE3" ], "results_posted": false, "status": "NOT_YET_RECRUITING", "updated_on": "2025-05-22", "url": "https://clinicaltrials.gov/study/NCT06973629" }, "source_ids": [ "nurown-update", "NCT06973629" ], "sources": [ "https://ir.brainstorm-cell.com/2026-08-14-BrainStorm-Cell-Therapeutics-Announces-Second-Quarter-2026-Financial-Results-and-Provides-Corporate-Update", "https://clinicaltrials.gov/study/NCT06973629" ], "status": "Not yet recruiting", "status_kind": "planned", "subtitle": "ENDURANCE · autologous cell therapy", "summary": "ENDURANCE is the planned confirmatory study of cells prepared from a participant’s own bone marrow.", "trial_name": "NurOwn / debamestrocel" }, { "evidence": "The registry lists not yet recruiting. In April 2026 AB Science described the ALS study as not yet started and a temporary European trial halt. EMA’s earlier refusal cited unreliable study data and insufficiently demonstrated benefit.", "evidence_date": "2026-04-16", "id": "masitinib", "india_note": "No Indian site is listed in the cited record. Recruitment elsewhere does not guarantee eligibility, travel support or access from India.", "limitations": [ "Post-hoc or preprint survival analyses do not overturn the regulatory refusal or establish an approved treatment." ], "name": "Masitinib", "registry": { "countries": [ "Greece" ], "id": "NCT07174492", "phase": [ "PHASE3" ], "results_posted": false, "status": "NOT_YET_RECRUITING", "updated_on": "2025-10-03", "url": "https://clinicaltrials.gov/study/NCT07174492" }, "source_ids": [ "masitinib-ema", "masitinib-update", "NCT07174492" ], "sources": [ "https://www.ema.europa.eu/en/medicines/human/EPAR/masitinib-ab-science", "https://www.ab-science.com/wp-content/uploads/2026/04/Clinical-Program-Update-April-2026-VENG.pdf", "https://clinicaltrials.gov/study/NCT07174492" ], "status": "Not yet recruiting", "status_kind": "planned", "subtitle": "AB23005 · add-on to standard care", "summary": "A confirmatory Phase 3 study is registered for this investigational kinase inhibitor.", "trial_name": "Masitinib" }, { "evidence": "The July 2026 registry lists active follow-up with enrollment closed and no results posted. The study evaluates efficacy and safety of lowering FUS protein.", "evidence_date": "2026-07-21", "id": "ulefnersen", "india_note": "No Indian site is listed in the cited record. Recruitment elsewhere does not guarantee eligibility, travel support or access from India.", "limitations": [ "This genetically defined program cannot be generalized to all ALS. An anticipated readout is not a completed positive result." ], "name": "Ulefnersen / ION363", "registry": { "countries": [ "Belgium", "Brazil", "Canada", "Germany", "Ireland", "Italy", "Japan", "Netherlands", "Poland", "South Korea", "Spain", "Sweden", "Switzerland", "Taiwan", "United Kingdom", "United States" ], "id": "NCT04768972", "phase": [ "PHASE3" ], "results_posted": false, "status": "ACTIVE_NOT_RECRUITING", "updated_on": "2026-07-21", "url": "https://clinicaltrials.gov/study/NCT04768972" }, "source_ids": [ "NCT04768972" ], "sources": [ "https://clinicaltrials.gov/study/NCT04768972" ], "status": "Active, not recruiting", "status_kind": "active", "subtitle": "FUSION · FUS-targeted antisense therapy", "summary": "A Phase 3 trial in ALS associated with pathogenic FUS mutations.", "trial_name": "Ulefnersen / ION363" }, { "evidence": "QurAlis reported interim target-engagement and biomarker findings in February 2026, with an overall trend and subgroup signals in function. The sponsor calls ANQUR Phase 1/2; the registry classifies it Phase 1 and lists enrollment closed.", "evidence_date": "2026-05-15", "id": "qrl201", "india_note": "No Indian site is listed in the cited record. Recruitment elsewhere does not guarantee eligibility, travel support or access from India.", "limitations": [ "Interim and subgroup results are not confirmatory efficacy. A proposed 2027 Phase 3 program remains a plan." ], "name": "QRL-201", "registry": { "countries": [ "Belgium", "Canada", "Germany", "Ireland", "Netherlands", "United Kingdom" ], "id": "NCT05633459", "phase": [ "PHASE1" ], "results_posted": false, "status": "ACTIVE_NOT_RECRUITING", "updated_on": "2026-05-15", "url": "https://clinicaltrials.gov/study/NCT05633459" }, "source_ids": [ "qrl201-update", "NCT05633459" ], "sources": [ "https://www.quralis.com/news/quralis-demonstrates-effects-on-disease-progression-and-target-engagement-in-anqur-clinical-trial-of-qrl-201-a-first-in-class-precision-medicine-in-development-for-sporadic-als/", "https://clinicaltrials.gov/study/NCT05633459" ], "status": "Active, not recruiting", "status_kind": "active", "subtitle": "ANQUR · STMN2 restoration", "summary": "An antisense approach intended to restore stathmin-2 expression affected by TDP-43 dysfunction.", "trial_name": "QRL-201" }, { "evidence": "On 31 August 2026 NeuroSense announced plans to optimize PARAGON into a smaller, shorter pivotal study, subject to FDA alignment. A Canadian submission is targeted for December 2026. Neither milestone should be described as completed.", "evidence_date": "2026-08-31", "id": "primec", "india_note": "An Indian approval, launch date or price is not established by this development announcement.", "limitations": [ "Earlier clinical and biomarker signals require confirmation. The formulation is not interchangeable with self-combining its component medicines." ], "name": "PrimeC", "source_ids": [ "primec-update" ], "sources": [ "https://neurosense.investorroom.com/2026-08-31-NeuroSense-Unveils-Accelerated,-Capital-Efficient-Strategy-to-Advance-PrimeC-Toward-Potential-Approval-in-ALS" ], "status": "Pivotal study planned", "status_kind": "planned", "subtitle": "NeuroSense · extended-release combination", "summary": "An investigational fixed-dose formulation of ciprofloxacin and celecoxib.", "trial_name": "PrimeC" }, { "evidence": "The August update reports continuing enrollment and targets topline data in Q1 2027. The sponsor describes ALSTARS as Phase 2; ClinicalTrials.gov lists Phase 2/3. Both descriptions refer to the same study.", "evidence_date": "2026-08-11", "id": "coya302", "india_note": "No Indian site is listed in the cited record. Recruitment elsewhere does not guarantee eligibility, travel support or access from India.", "limitations": [ "No randomized efficacy results are posted. Trial phase and Fast Track designation are not evidence of approval." ], "name": "COYA 302", "registry": { "countries": [ "Canada", "United States" ], "id": "NCT07161999", "phase": [ "PHASE2", "PHASE3" ], "results_posted": false, "status": "RECRUITING", "updated_on": "2026-08-06", "url": "https://clinicaltrials.gov/study/NCT07161999" }, "source_ids": [ "coya-update", "NCT07161999" ], "sources": [ "https://ir.coyatherapeutics.com/news/news-details/2026/Coya-Therapeutics-Reports-Second-Quarter-2026-Financial-Results-and-Provides-a-Corporate-Update/default.aspx", "https://clinicaltrials.gov/study/NCT07161999" ], "status": "Recruiting", "status_kind": "recruiting", "subtitle": "ALSTARS · immune regulation", "summary": "A combination biologic strategy intended to enhance regulatory T-cell activity.", "trial_name": "COYA 302" }, { "evidence": "The HEALEY trial center reports enrollment complete, consistent with the July 2026 registry status. The Phase 2/3 cohort is now in follow-up; no efficacy results are posted.", "evidence_date": "2026-07-20", "id": "nuz001", "india_note": "No Indian site is listed in the cited record. Recruitment elsewhere does not guarantee eligibility, travel support or access from India.", "limitations": [ "Small earlier safety studies and open-label observations do not establish slowing of ALS progression." ], "name": "NUZ-001", "registry": { "countries": [ "United States" ], "id": "NCT07410806", "phase": [ "PHASE2", "PHASE3" ], "results_posted": false, "status": "ACTIVE_NOT_RECRUITING", "updated_on": "2026-07-20", "url": "https://clinicaltrials.gov/study/NCT07410806" }, "source_ids": [ "healey", "NCT07410806" ], "sources": [ "https://research.massgeneralbrigham.org/en/institutes-centers/neuroscience-institute/department-of-neurology/sean-healey-amg-center-for-als/healey-als-platform-trial", "https://clinicaltrials.gov/study/NCT07410806" ], "status": "Active, not recruiting", "status_kind": "active", "subtitle": "HEALEY Regimen I · S-monepantel", "summary": "An investigational oral medicine designed to support clearance of harmful cellular proteins.", "trial_name": "NUZ-001" }, { "evidence": "The early study is registered as completed. The sponsor reported Phase 2a findings in November 2025. A separate US expanded-access protocol is listed as available (NCT07088159).", "evidence_date": "2026-07-17", "id": "spg302", "india_note": "The US expanded-access protocol is not an Indian approval or a guarantee that an overseas patient can join.", "limitations": [ "Early functional observations and access outside a trial do not prove synapse regeneration or reliable functional recovery in people with ALS." ], "name": "SPG302 / tazbentatol", "registry": { "countries": [ "Australia" ], "id": "NCT05882695", "phase": [ "PHASE1" ], "results_posted": false, "status": "COMPLETED", "updated_on": "2025-06-29", "url": "https://clinicaltrials.gov/study/NCT05882695" }, "source_ids": [ "spg302-update", "NCT05882695", "NCT07088159" ], "sources": [ "https://spinogenix.com/press-release/spinogenix-presents-topline-phase-2a-clinical-trial-results-for-spg302-a-first-in-class-als-treatment/", "https://clinicaltrials.gov/study/NCT05882695", "https://clinicaltrials.gov/study/NCT07088159" ], "status": "Early study completed", "status_kind": "active", "subtitle": "Spinogenix · synaptic function", "summary": "A small molecule being studied for effects on synaptic function; clinical benefit in ALS remains unconfirmed.", "trial_name": "SPG302 / tazbentatol" }, { "evidence": "Alchemab announced a first-in-human Phase 1 trial on 9 September 2025. The program was licensed to Lilly. No later ALS efficacy result was established in this review.", "evidence_date": "2025-09-09", "id": "atlx1282", "india_note": "No Indian trial site or commercial access is verified. Corporate presence in India does not establish future access.", "limitations": [ "A licensing agreement does not validate efficacy. Current patient recruitment and a Phase 2 start date have not been verified." ], "name": "ATLX-1282", "source_ids": [ "atlx-update" ], "sources": [ "https://www.prnewswire.com/news-releases/alchemab-therapeutics-initiates-phase-1-clinical-trial-of-atlx-1282-and-announces-series-a-financing-extension-302549523.html" ], "status": "Early clinical development", "status_kind": "active", "subtitle": "Alchemab / Lilly · antibody program", "summary": "A therapeutic antibody program derived from research into biological resilience.", "trial_name": "ATLX-1282" }, { "evidence": "June 2026 preliminary results cover 82 randomized participants. Axoltis reported exploratory functional and biomarker trends; the cited early NfL comparison was not statistically significant.", "evidence_date": "2026-06-24", "id": "nx210c", "india_note": "No Indian site is listed in the cited record. Recruitment elsewhere does not guarantee eligibility, travel support or access from India.", "limitations": [ "Preliminary conference findings need the full controlled analysis. Biomarker trends must not be presented as proven survival benefit." ], "name": "NX210c", "registry": { "countries": [ "France" ], "id": "NCT06365216", "phase": [ "PHASE2" ], "results_posted": false, "status": "ACTIVE_NOT_RECRUITING", "updated_on": "2025-12-24", "url": "https://clinicaltrials.gov/study/NCT06365216" }, "source_ids": [ "nx210-update", "NCT06365216" ], "sources": [ "https://www.axoltis.com/2026/06/24/encals-2026-european-network-to-cure-als/", "https://clinicaltrials.gov/study/NCT06365216" ], "status": "Active, not recruiting", "status_kind": "active", "subtitle": "SEALS · neuroprotective peptide", "summary": "A Phase 2 study of an intravenous peptide approach to neuroprotection and blood–brain barrier function.", "trial_name": "NX210c" }, { "evidence": "The ALS-specific record NCT07118319 lists recruiting and no posted results. Separate XS228 spinal-cord-injury trials must not be used as evidence of benefit in ALS.", "evidence_date": "2025-11-21", "id": "xs228", "india_note": "No Indian site is listed in the cited record. Recruitment elsewhere does not guarantee eligibility, travel support or access from India.", "limitations": [ "Safety, integration of transplanted cells and clinical benefit remain experimental. There is no established restoration of lost motor neurons or function." ], "name": "XS-228 / XS228CN", "registry": { "countries": [ "China" ], "id": "NCT07118319", "phase": [ "PHASE1" ], "results_posted": false, "status": "RECRUITING", "updated_on": "2025-11-21", "url": "https://clinicaltrials.gov/study/NCT07118319" }, "source_ids": [ "NCT07118319" ], "sources": [ "https://clinicaltrials.gov/study/NCT07118319" ], "status": "Recruiting", "status_kind": "recruiting", "subtitle": "XellSmart · iPSC-derived neural progenitor cells", "summary": "A Phase 1 ALS study in China evaluates induced-pluripotent-stem-cell-derived motor neuron progenitor cells.", "trial_name": "XS-228 / XS228CN" }, { "evidence": "Amylyx’s June 2026 update reported no drug-related serious adverse events in the lowest-dose cohort, while the measured biomarkers remained near baseline. Higher-dose cohorts are being evaluated.", "evidence_date": "2026-06-23", "id": "amx0114", "india_note": "No Indian site is listed in the cited record. Recruitment elsewhere does not guarantee eligibility, travel support or access from India.", "limitations": [ "Tolerability at a low dose is not evidence of clinical efficacy. Dose-escalation findings are still early." ], "name": "AMX0114", "registry": { "countries": [ "Canada", "United States" ], "id": "NCT06665165", "phase": [ "PHASE1" ], "results_posted": false, "status": "RECRUITING", "updated_on": "2026-04-14", "url": "https://clinicaltrials.gov/study/NCT06665165" }, "source_ids": [ "amx0114-update", "NCT06665165" ], "sources": [ "https://investors.amylyx.com/news-releases/news-release-details/amylyx-pharmaceuticals-present-data-first-human-dose-ranging", "https://clinicaltrials.gov/study/NCT06665165" ], "status": "Recruiting", "status_kind": "recruiting", "subtitle": "LUMINA · calpain-2 antisense therapy", "summary": "A Phase 1 study of an antisense medicine targeting calpain-2.", "trial_name": "AMX0114" }, { "evidence": "Nura Bio announced the first ALS participant dosed in June 2026. The Phase 1b/2a program measures safety, drug exposure and biomarkers; registry sites are in Australia and Canada.", "evidence_date": "2026-06-22", "id": "nb4746", "india_note": "No Indian site is listed in the cited record. Recruitment elsewhere does not guarantee eligibility, travel support or access from India.", "limitations": [ "The biological rationale and healthy-volunteer findings do not yet demonstrate benefit in ALS. This program is distinct from preclinical ASHA-624." ], "name": "NB-4746", "registry": { "countries": [ "Australia", "Canada" ], "id": "NCT07369076", "phase": [ "PHASE1", "PHASE2" ], "results_posted": false, "status": "RECRUITING", "updated_on": "2026-06-22", "url": "https://clinicaltrials.gov/study/NCT07369076" }, "source_ids": [ "nb4746-update", "NCT07369076" ], "sources": [ "https://www.globenewswire.com/news-release/2026/06/22/3315110/0/en/nura-bio-completes-73-8m-series-b-financing-and-advances-pipeline-of-sarm1-inhibitors.html", "https://clinicaltrials.gov/study/NCT07369076" ], "status": "Recruiting", "status_kind": "recruiting", "subtitle": "Nura Bio · SARM1 inhibitor", "summary": "SARM1 inhibition has now entered ALS patient testing with this oral investigational drug.", "trial_name": "NB-4746" }, { "evidence": "Celosia announced first dosing in March 2026 at Macquarie University Hospital. The Phase 1b study is open-label and focuses on safety and tolerability. The recruiting registry record was last updated in February.", "evidence_date": "2026-03-23", "id": "ctx1000", "india_note": "No Indian site is listed in the cited record. Recruitment elsewhere does not guarantee eligibility, travel support or access from India.", "limitations": [ "The registry is older than the dosing announcement: confirm current enrollment directly. Reversal in animal models is not evidence of reversal in people." ], "name": "CTx1000", "registry": { "countries": [ "Australia" ], "id": "NCT07401121", "phase": [ "PHASE1" ], "results_posted": false, "status": "RECRUITING", "updated_on": "2026-02-10", "url": "https://clinicaltrials.gov/study/NCT07401121" }, "source_ids": [ "ctx1000-update", "NCT07401121" ], "sources": [ "https://www.prnewswire.com/news-releases/celosia-therapeutics-announces-first-patient-dosed-in-phase-1b-clinical-trial-of-ctx1000-in-amyotrophic-lateral-sclerosis-302721815.html", "https://clinicaltrials.gov/study/NCT07401121" ], "status": "Recruiting in cited record", "status_kind": "recruiting", "subtitle": "KOANEWA · TDP-43-directed gene therapy", "summary": "An early gene-therapy study intended to clear pathological TDP-43.", "trial_name": "CTx1000" }, { "evidence": "The published first study involved six participants and examined safety, exposure and preliminary biological or clinical observations. A separate Phase 1 trial is registered as recruiting.", "evidence_date": "2026-07-15", "id": "rag17", "india_note": "No Indian site is listed in the cited record. Recruitment elsewhere does not guarantee eligibility, travel support or access from India.", "limitations": [ "Six participants without a definitive controlled efficacy comparison cannot establish survival benefit or superiority to tofersen. Enrollment status needs confirmation because the cited registry update is older." ], "name": "RAG-17", "registry": { "countries": [ "China" ], "id": "NCT06556394", "phase": [ "PHASE1" ], "results_posted": false, "status": "RECRUITING", "updated_on": "2025-01-08", "url": "https://clinicaltrials.gov/study/NCT06556394" }, "source_ids": [ "rag17-paper", "NCT06556394" ], "sources": [ "https://www.nature.com/articles/s41591-026-04491-7", "https://clinicaltrials.gov/study/NCT06556394" ], "status": "Phase 1 evidence", "status_kind": "active", "subtitle": "SOD1-targeted siRNA", "summary": "A July 2026 Nature Medicine paper reports early human testing of an RNA-silencing approach for SOD1 ALS.", "trial_name": "RAG-17" }, { "evidence": "The 1 September 2026 registry update lists recruiting, with multiple countries participating and no results posted. This is an investigational ALS program associated with an Indian-origin sponsor.", "evidence_date": "2026-09-01", "id": "usnoflast", "india_note": "No Indian site is listed in the cited record. Recruitment elsewhere does not guarantee eligibility, travel support or access from India.", "limitations": [ "Sponsor origin does not mean a trial is taking place in India or that the medicine is approved there." ], "name": "Usnoflast", "registry": { "countries": [ "Australia", "Belgium", "Canada", "France", "Germany", "Ireland", "Italy", "Netherlands", "Poland", "Spain", "Sweden", "United States" ], "id": "NCT07023835", "phase": [ "PHASE2" ], "results_posted": false, "status": "RECRUITING", "updated_on": "2026-09-01", "url": "https://clinicaltrials.gov/study/NCT07023835" }, "source_ids": [ "NCT07023835" ], "sources": [ "https://clinicaltrials.gov/study/NCT07023835" ], "status": "Recruiting", "status_kind": "recruiting", "subtitle": "UNITE-ALS · Zydus", "summary": "A Phase 2b randomized study of an oral NLRP3 inflammasome inhibitor.", "trial_name": "Usnoflast" }, { "evidence": "The July 2026 registry lists ongoing recruitment and evaluates safety and pharmacological effects of an RNA-interference approach.", "evidence_date": "2026-07-22", "id": "aln-sod", "india_note": "No Indian site is listed in the cited record. Recruitment elsewhere does not guarantee eligibility, travel support or access from India.", "limitations": [ "No results are posted. This is a separate investigational product from approved tofersen and from RAG-17." ], "name": "ALN-SOD", "registry": { "countries": [ "Australia", "Belgium", "Canada", "Germany", "Japan", "Poland", "South Korea", "Sweden", "Taiwan" ], "id": "NCT06351592", "phase": [ "PHASE1", "PHASE2" ], "results_posted": false, "status": "RECRUITING", "updated_on": "2026-07-22", "url": "https://clinicaltrials.gov/study/NCT06351592" }, "source_ids": [ "NCT06351592" ], "sources": [ "https://clinicaltrials.gov/study/NCT06351592" ], "status": "Recruiting", "status_kind": "recruiting", "subtitle": "SOD1-targeted RNA interference", "summary": "A first-in-human Phase 1/2 study for adults with a SOD1 mutation.", "trial_name": "ALN-SOD" }, { "evidence": "The registry updated on 10 September 2026 lists recruiting at sites in Europe and the US. It is an open-label ascending-dose study with clinical and biomarker assessments.", "evidence_date": "2026-09-10", "id": "vtx002", "india_note": "No Indian site is listed in the cited record. Recruitment elsewhere does not guarantee eligibility, travel support or access from India.", "limitations": [ "No results are posted. Small uncontrolled gene-therapy studies establish early safety information, not a proven ALS treatment. The older sponsor mechanism page still says preclinical; use the dated registry for current trial status." ], "name": "VTx-002", "registry": { "countries": [ "Belgium", "Netherlands", "United Kingdom", "United States" ], "id": "NCT07287397", "phase": [ "PHASE1", "PHASE2" ], "results_posted": false, "status": "RECRUITING", "updated_on": "2026-09-10", "url": "https://clinicaltrials.gov/study/NCT07287397" }, "source_ids": [ "vectory-mechanism", "NCT07287397" ], "sources": [ "https://vectorytx.squarespace.com/vtx002-for-als", "https://clinicaltrials.gov/study/NCT07287397" ], "status": "Recruiting", "status_kind": "recruiting", "subtitle": "PIONEER-ALS · gene therapy", "summary": "A first-in-human Phase 1/2 program using a vectorized antibody approach directed at TDP-43 pathology.", "trial_name": "VTx-002" }, { "evidence": "The September 2026 registry lists recruiting in the US. The study evaluates safety, tolerability and pharmacodynamic effects after intrathecal administration.", "evidence_date": "2026-09-01", "id": "ins1202", "india_note": "No Indian site is listed in the cited record. Recruitment elsewhere does not guarantee eligibility, travel support or access from India.", "limitations": [ "There are no posted efficacy results. Eligibility is protocol-specific and must be confirmed by the study team." ], "name": "INS1202", "registry": { "countries": [ "United States" ], "id": "NCT07290062", "phase": [ "PHASE1" ], "results_posted": false, "status": "RECRUITING", "updated_on": "2026-09-01", "url": "https://clinicaltrials.gov/study/NCT07290062" }, "source_ids": [ "NCT07290062" ], "sources": [ "https://clinicaltrials.gov/study/NCT07290062" ], "status": "Recruiting", "status_kind": "recruiting", "subtitle": "Insmed · investigational genetic therapy", "summary": "A single-dose Phase 1 study in adults with SOD1 ALS or ALS without a known ALS-related genetic mutation.", "trial_name": "INS1202" }, { "evidence": "The registry updated in June 2026 lists a US Phase 2 study and excludes SOD1- and FUS-associated disease. This is a research question with no posted results.", "evidence_date": "2026-06-25", "id": "tofersen-nonsod1", "india_note": "No Indian site is listed in the cited record. Recruitment elsewhere does not guarantee eligibility, travel support or access from India.", "limitations": [ "The FDA-approved indication remains SOD1 ALS. This trial is not evidence to prescribe tofersen broadly for sporadic ALS." ], "name": "Tofersen in non-SOD1 ALS", "registry": { "countries": [ "United States" ], "id": "NCT07294144", "phase": [ "PHASE2" ], "results_posted": false, "status": "RECRUITING", "updated_on": "2026-06-25", "url": "https://clinicaltrials.gov/study/NCT07294144" }, "source_ids": [ "NCT07294144" ], "sources": [ "https://clinicaltrials.gov/study/NCT07294144" ], "status": "Recruiting", "status_kind": "recruiting", "subtitle": "Investigator-led Phase 2 study", "summary": "An exploratory trial tests whether tofersen has biological or clinical effects beyond genetically confirmed SOD1 ALS.", "trial_name": "Tofersen in non-SOD1 ALS" }, { "evidence": "The April 2026 sponsor report describes two-year survival analyses and a planned Phase 3 trial. The long-term comparisons include selected participants who did or did not enter the extension. The registry still says recruiting, which should not be read as enrollment in the planned pivotal study.", "evidence_date": "2026-06-16", "id": "dazucorilant", "india_note": "No Indian site is listed in the cited record. Recruitment elsewhere does not guarantee eligibility, travel support or access from India.", "limitations": [ "Survival signals after a failed primary endpoint need confirmation, especially with selection into an extension. No results posted in a registry does not mean results have not been reported elsewhere. Confirm any enrollment directly." ], "name": "Dazucorilant", "registry": { "countries": [ "Belgium", "Canada", "France", "Germany", "Ireland", "Netherlands", "Poland", "Spain", "United Kingdom", "United States" ], "id": "NCT05407324", "phase": [ "PHASE2" ], "results_posted": false, "status": "RECRUITING", "updated_on": "2026-06-16", "url": "https://clinicaltrials.gov/study/NCT05407324" }, "source_ids": [ "dazucorilant-update", "NCT05407324" ], "sources": [ "https://ir.corcept.com/news-releases/news-release-details/corcept-phase-2-study-dazucorilant-demonstrates-two-year-overall/", "https://clinicaltrials.gov/study/NCT05407324" ], "status": "Phase 3 planned", "status_kind": "planned", "subtitle": "DAZALS · glucocorticoid receptor modulation", "summary": "DAZALS missed its primary functional endpoint. Corcept is pursuing further study after secondary and exploratory survival findings.", "trial_name": "Dazucorilant" }, { "evidence": "Leal announced first dosing in June 2026. The August registry lists a blinded, placebo-controlled dose-escalation study assessing safety, tolerability and pharmacological effects.", "evidence_date": "2026-08-18", "id": "ltx002", "india_note": "No Indian site is listed in the cited record. Recruitment elsewhere does not guarantee eligibility, travel support or access from India.", "limitations": [ "The rationale extends to sporadic and genetic ALS, but clinical benefit has not been established. This is a first-in-human program with no posted results." ], "name": "LTX-002", "registry": { "countries": [ "Germany", "Italy", "Netherlands", "Sweden" ], "id": "NCT07660614", "phase": [ "PHASE1", "PHASE2" ], "results_posted": false, "status": "RECRUITING", "updated_on": "2026-08-18", "url": "https://clinicaltrials.gov/study/NCT07660614" }, "source_ids": [ "ltx002-update", "NCT07660614" ], "sources": [ "https://www.prnewswire.com/news-releases/leal-therapeutics-doses-first-patient-in-neurals-phase-12-trial-of-ltx-002-for-amyotrophic-lateral-sclerosis-als-302807297.html", "https://clinicaltrials.gov/study/NCT07660614" ], "status": "Recruiting", "status_kind": "recruiting", "subtitle": "NeurALS · SPTLC1-targeted antisense therapy", "summary": "A new Phase 1/2 study investigates an RNA-targeted approach to abnormal lipid metabolism in ALS.", "trial_name": "LTX-002" } ], "communication_technology": [ { "category": "PCEye · TD I-Series · TD Pilot", "evidence": "Manufacturer documentation distinguishes a computer eye tracker from integrated Windows and iPad-based speech devices. Selection depends on positioning, vision, fatigue and the person’s preferred communication system.", "evidence_date": null, "form_factor": "PCEye · TD I-Series · TD Pilot", "how_it_works": "Manufacturer documentation distinguishes a computer eye tracker from integrated Windows and iPad-based speech devices. Selection depends on positioning, vision, fatigue and the person’s preferred communication system.", "id": "tobii", "india_availability": { "available": "Confirm locally", "how_to_get": "Request a local assessment and written quotation covering hardware, mount, software, warranty, repairs and language support." }, "india_note": "Request a local assessment and written quotation covering hardware, mount, software, warranty, repairs and language support.", "limitations": [ "Product documentation describes capabilities, not a guarantee of individual performance. Assess with an AAC professional and trial the exact setup." ], "name": "Tobii Dynavox AAC", "realistic_expectations": "Supports communication; it does not treat motor neuron degeneration.", "solution_name": "Tobii Dynavox AAC", "source_ids": [ "tobii-aac" ], "sources": [ "https://downloads.tobiidynavox.com/Conditions/ALS/CI_Sheet/ALS_CI-Sheet_A4_TD_en-INT.pdf" ], "status": "Assistive communication", "status_kind": "support", "subtitle": "PCEye · TD I-Series · TD Pilot", "summary": "Dedicated eye-gaze and speech-generating products can provide communication and computer access as movement changes.", "what_it_is": "Dedicated eye-gaze and speech-generating products can provide communication and computer access as movement changes." }, { "category": "Wearable eye-tracking communication", "evidence": "An ALS feasibility study is registered, while current manufacturer documentation also describes a bedside hospital platform. Product configurations and clinical use cases must be distinguished.", "evidence_date": "2019-08-28", "form_factor": "Wearable eye-tracking communication", "how_it_works": "An ALS feasibility study is registered, while current manufacturer documentation also describes a bedside hospital platform. Product configurations and clinical use cases must be distinguished.", "id": "eyecontrol", "india_availability": { "available": "Confirm locally", "how_to_get": "Current Indian distribution, home-use suitability and support have not been verified; contact the clinical team and manufacturer before purchase." }, "india_note": "Current Indian distribution, home-use suitability and support have not been verified; contact the clinical team and manufacturer before purchase.", "limitations": [ "A completed feasibility study does not guarantee reliable use for every person with advanced ALS. Verify the specific product and evidence with the supplier." ], "name": "EyeControl", "realistic_expectations": "Supports communication; it does not treat motor neuron degeneration.", "solution_name": "EyeControl", "source_ids": [ "eyecontrol", "NCT02891629" ], "sources": [ "https://www.eyecontrol.co.il/howitworks", "https://clinicaltrials.gov/study/NCT02891629" ], "status": "Specialist assessment needed", "status_kind": "support", "subtitle": "Wearable eye-tracking communication", "summary": "A wearable, screen-free approach uses eye movements with audio feedback to communicate needs.", "what_it_is": "A wearable, screen-free approach uses eye movements with audio feedback to communicate needs." }, { "category": "Open-source Windows communication software", "evidence": "The maintainers explicitly removed support for Tobii Eye Tracker 5, EyeX and 4C in OptiKey 4. Legacy OptiKey 3.2.x supports these older use cases.", "evidence_date": "2025-05-14", "form_factor": "Open-source Windows communication software", "how_it_works": "The maintainers explicitly removed support for Tobii Eye Tracker 5, EyeX and 4C in OptiKey 4. Legacy OptiKey 3.2.x supports these older use cases.", "id": "optikey", "india_availability": { "available": "Confirm locally", "how_to_get": "Assess the existing hardware first. Budget for setup and assistance even where the software is free." }, "india_note": "Assess the existing hardware first. Budget for setup and assistance even where the software is free.", "limitations": [ "Do not assume the latest OptiKey works with the Tobii Eye Tracker 5 setup elsewhere in this project. Verify hardware, driver, Windows and application versions together." ], "name": "OptiKey with an eye tracker", "realistic_expectations": "Supports communication; it does not treat motor neuron degeneration.", "solution_name": "OptiKey with an eye tracker", "source_ids": [ "optikey-versions" ], "sources": [ "https://github.com/OptiKey/OptiKey/wiki/Optikey-versions" ], "status": "Version compatibility matters", "status_kind": "support", "subtitle": "Open-source Windows communication software", "summary": "OptiKey supports typing, speech output and computer control using compatible input devices.", "what_it_is": "OptiKey supports typing, speech output and computer control using compatible input devices." } ], "experimental_communication_tech": [ { "cost": "No verified current quotation.", "critical_limitation": "Building a usable communication interface requires software, calibration/positioning work and individual testing. Do not equate raw gaze streaming with ready-to-use AAC.", "evidence": "The official real-time API can stream data to custom applications on the same network. That capability enables prototyping but does not establish clinical reliability for ALS.", "evidence_date": null, "id": "neon", "india_note": "Consider established AAC assessment first. A custom integration needs sustained technical support and a backup communication method.", "limitations": [ "Building a usable communication interface requires software, calibration/positioning work and individual testing. Do not equate raw gaze streaming with ready-to-use AAC." ], "name": "Pupil Labs Neon integrations", "recommendation": "Consider established AAC assessment first. A custom integration needs sustained technical support and a backup communication method.", "risk_level": "Research / individual assessment", "source_ids": [ "neon-api" ], "sources": [ "https://docs.pupil-labs.com/neon/data-collection/ecosystem-overview/" ], "status": "Research hardware", "status_kind": "experimental", "subtitle": "Wearable gaze data · custom software", "summary": "Neon provides gaze and other eye signals for researchers and developers; it is not a complete ALS communication system.", "technology": "Pupil Labs Neon integrations", "what_it_is": "Neon provides gaze and other eye signals for researchers and developers; it is not a complete ALS communication system.", "who_can_try": "Subject to individual assessment or formal study eligibility." }, { "cost": "No verified current quotation.", "critical_limitation": "It is head tracking, not eye tracking. Weakness, fatigue and positioning can limit use; these tools are not established replacements for eye-gaze AAC.", "evidence": "Enable Viacam’s documentation describes camera and tracking-area configuration. This is an access method for someone who can comfortably produce the required movements.", "evidence_date": null, "id": "head-tracking", "india_note": "Trial comfort and accuracy with an assistive-technology professional. Maintain a simple communication backup.", "limitations": [ "It is head tracking, not eye tracking. Weakness, fatigue and positioning can limit use; these tools are not established replacements for eye-gaze AAC." ], "name": "Webcam head tracking", "recommendation": "Trial comfort and accuracy with an assistive-technology professional. Maintain a simple communication backup.", "risk_level": "Research / individual assessment", "source_ids": [ "eviacam" ], "sources": [ "https://eviacam.crea-si.com/help/en/wizard.htm" ], "status": "Individual assessment", "status_kind": "experimental", "subtitle": "Enable Viacam · alternative computer access", "summary": "Head-tracking software can move a cursor using movements observed by a webcam.", "technology": "Webcam head tracking", "what_it_is": "Head-tracking software can move a cursor using movements observed by a webcam.", "who_can_try": "Subject to individual assessment or formal study eligibility." }, { "cost": "No verified current quotation.", "critical_limitation": "Do not present a prototype, price estimate or working demo as reliable daily communication for a person with ALS.", "evidence": "No ALS-specific clinical validation or supported complete communication product was established for the older page’s proposed DIY setup. The Pupil Labs API documentation supports only the general feasibility of custom data integrations.", "evidence_date": null, "id": "diy-tracking", "india_note": "Keep prototypes optional and supervised. They should not replace a dependable way to express urgent needs.", "limitations": [ "Do not present a prototype, price estimate or working demo as reliable daily communication for a person with ALS." ], "name": "DIY marker and smartphone tracking", "recommendation": "Keep prototypes optional and supervised. They should not replace a dependable way to express urgent needs.", "risk_level": "Research / individual assessment", "source_ids": [ "neon-api" ], "sources": [ "https://docs.pupil-labs.com/neon/data-collection/ecosystem-overview/" ], "status": "ALS evidence not established", "status_kind": "experimental", "subtitle": "Prototype concepts · including AprilTag setups", "summary": "Marker-based and smartphone experiments should be treated as engineering prototypes.", "technology": "DIY marker and smartphone tracking", "what_it_is": "Marker-based and smartphone experiments should be treated as engineering prototypes.", "who_can_try": "Subject to individual assessment or formal study eligibility." }, { "cost": "No verified current quotation.", "critical_limitation": "Small participant numbers, implanted hardware and intensive specialist support limit generalization. These systems are not established routine home products.", "evidence": "UC Davis reported real-time voice synthesis in June 2025 in a participant with ALS. This is a communication milestone rather than restoration of natural speech muscles.", "evidence_date": "2025-06-11", "id": "speech-bci", "india_note": "Access is through formal research eligibility. Maintain practical AAC planning alongside interest in BCI research.", "limitations": [ "Small participant numbers, implanted hardware and intensive specialist support limit generalization. These systems are not established routine home products." ], "name": "Brain-to-voice neuroprostheses", "recommendation": "Access is through formal research eligibility. Maintain practical AAC planning alongside interest in BCI research.", "risk_level": "Research / individual assessment", "source_ids": [ "speech-bci" ], "sources": [ "https://health.ucdavis.edu/chpr/news/headlines/first-of-its-kind-technology-helps-man-with-als-speak-in-real-time/2025/06" ], "status": "Early human research", "status_kind": "experimental", "subtitle": "Implanted brain–computer interfaces", "summary": "Research systems have enabled an individual with ALS to produce synthetic speech directly from recorded brain activity.", "technology": "Brain-to-voice neuroprostheses", "what_it_is": "Research systems have enabled an individual with ALS to produce synthetic speech directly from recorded brain activity.", "who_can_try": "Subject to individual assessment or formal study eligibility." }, { "cost": "No verified current quotation.", "critical_limitation": "A research authorization is not commercial approval. Surgery, long follow-up and strict eligibility distinguish this from buying an eye tracker.", "evidence": "The trial registry lists recruiting in the US and includes people with severe speech or movement impairment. Safety and device feasibility are central objectives.", "evidence_date": "2026-04-14", "id": "connexus", "india_note": "No Indian study site is listed. Contact a formal research center about eligibility rather than relying on commercial availability claims.", "limitations": [ "A research authorization is not commercial approval. Surgery, long follow-up and strict eligibility distinguish this from buying an eye tracker." ], "name": "Connexus / Connect-One", "recommendation": "No Indian study site is listed. Contact a formal research center about eligibility rather than relying on commercial availability claims.", "registry": { "countries": [ "United States" ], "id": "NCT07357428", "phase": [ "NA" ], "results_posted": false, "status": "RECRUITING", "updated_on": "2026-04-14", "url": "https://clinicaltrials.gov/study/NCT07357428" }, "risk_level": "Research / individual assessment", "source_ids": [ "NCT07357428" ], "sources": [ "https://clinicaltrials.gov/study/NCT07357428" ], "status": "Recruiting feasibility study", "status_kind": "experimental", "subtitle": "Paradromics · implanted BCI", "summary": "A 2026 early-feasibility study evaluates an implanted interface for communication and computer control.", "technology": "Connexus / Connect-One", "what_it_is": "A 2026 early-feasibility study evaluates an implanted interface for communication and computer control.", "who_can_try": "Subject to individual assessment or formal study eligibility." } ], "failed_discontinued_trials": [ { "evidence": "Calico reported in January 2025 that neither the primary nor exploratory higher dose met the primary endpoint. Key secondary endpoints for the primary dose also were not significantly different from placebo.", "evidence_date": "2025-01-06", "id": "fosigotifator", "india_note": "", "limitations": [ "Exploratory findings cannot be presented as an approved benefit. A negative trial does not, by itself, prove every future study of the mechanism must fail." ], "name": "Fosigotifator", "source_ids": [ "fosigotifator-results" ], "sources": [ "https://www.calicolabs.com/press/calico-statement-on-fosigotifator-in-healey-als-platform-trial/" ], "status": "Primary endpoint not met", "status_kind": "stopped", "subtitle": "ABBV-CLS-7262 · HEALEY Regimen F", "summary": "The tested eIF2B activator did not establish efficacy in the HEALEY study.", "trial_name": "Fosigotifator" }, { "evidence": "The January 2025 announcement reports that the primary combined function/survival endpoint and key strength and respiratory secondary endpoints were not met at 24 weeks.", "evidence_date": "2025-01-06", "id": "dnl343", "india_note": "", "limitations": [ "Safety or a biological rationale cannot substitute for a demonstrated clinical effect." ], "name": "DNL343", "source_ids": [ "dnl343-results" ], "sources": [ "https://investors.denalitherapeutics.com/node/10826/pdf" ], "status": "Primary endpoint not met", "status_kind": "stopped", "subtitle": "HEALEY Regimen G · eIF2B activation", "summary": "DNL343 did not demonstrate slowing of ALS progression in the tested regimen.", "trial_name": "DNL343" }, { "evidence": "The May 2024 announcement reports target lowering without evidence sufficient to support continued development for ALS.", "evidence_date": "2024-05-16", "id": "biib105", "india_note": "", "limitations": [ "Lowering a target protein alone does not establish that patients function better or live longer." ], "name": "BIIB105 / ION541", "source_ids": [ "biib105-results" ], "sources": [ "https://investors.biogen.com/news-releases/news-release-details/biogen-and-ionis-announce-topline-phase-12-study-results" ], "status": "Development discontinued", "status_kind": "stopped", "subtitle": "ALSpire · ataxin-2 antisense therapy", "summary": "Biogen and Ionis ended development following the Phase 1/2 ALSpire study.", "trial_name": "BIIB105 / ION541" }, { "evidence": "The study registry records termination for lack of efficacy. This entry concerns the ALS study, not results from the separate multiple-sclerosis program.", "evidence_date": "2025-03-21", "id": "sar443820", "india_note": "", "limitations": [ "Evidence from a different disease cannot be used to imply efficacy in ALS." ], "name": "SAR443820 / DNL788", "registry": { "countries": [ "Belgium", "Canada", "China", "France", "Germany", "Italy", "Japan", "Netherlands", "Poland", "Spain", "Sweden", "United Kingdom", "United States" ], "id": "NCT05237284", "phase": [ "PHASE2" ], "results_posted": true, "status": "TERMINATED", "updated_on": "2025-03-21", "url": "https://clinicaltrials.gov/study/NCT05237284" }, "source_ids": [ "NCT05237284" ], "sources": [ "https://clinicaltrials.gov/study/NCT05237284" ], "status": "Trial terminated", "status_kind": "stopped", "subtitle": "HIMALAYA · RIPK1 inhibitor", "summary": "The HIMALAYA Phase 2 program did not meet its ALS efficacy objective.", "trial_name": "SAR443820 / DNL788" }, { "evidence": "The published randomized study did not establish benefit on ALS progression.", "evidence_date": "2025-02-17", "id": "zilucoplan", "india_note": "", "limitations": [ "Approval or benefit in another condition does not imply an ALS indication." ], "name": "Zilucoplan", "source_ids": [ "zilucoplan-results" ], "sources": [ "https://jamanetwork.com/journals/jamanetworkopen/fullarticle/2830151" ], "status": "Stopped for futility", "status_kind": "stopped", "subtitle": "HEALEY Regimen A · complement C5", "summary": "The HEALEY trial stopped this regimen after a prespecified futility analysis.", "trial_name": "Zilucoplan" }, { "evidence": "Neither the prespecified primary endpoint nor the secondary endpoints differed significantly between active treatment and placebo.", "evidence_date": "2025-02-17", "id": "verdiperstat", "india_note": "", "limitations": [ "This negative result belongs alongside other unsuccessful programs, rather than in a list of proven options." ], "name": "Verdiperstat", "source_ids": [ "verdiperstat-results" ], "sources": [ "https://jamanetwork.com/journals/jamaneurology/fullarticle/2830248" ], "status": "Primary endpoint not met", "status_kind": "stopped", "subtitle": "HEALEY Regimen B · myeloperoxidase", "summary": "The published randomized HEALEY study did not demonstrate slowing of ALS.", "trial_name": "Verdiperstat" }, { "evidence": "The 2025 Lancet Neurology publication found no significant benefit in secondary clinical or biomarker measures either.", "evidence_date": "2025-06", "id": "trehalose", "india_note": "", "limitations": [ "Laboratory effects on protein clearance do not establish clinical benefit, and oral supplements are not equivalent to the trial intervention." ], "name": "Trehalose", "source_ids": [ "trehalose-results" ], "sources": [ "https://pubmed.ncbi.nlm.nih.gov/40409314/" ], "status": "No demonstrated benefit", "status_kind": "stopped", "subtitle": "HEALEY Regimen E · autophagy", "summary": "The intravenous trehalose regimen did not show evidence of slower ALS progression than placebo.", "trial_name": "Trehalose" }, { "evidence": "The published trial found no reduction in neurofilament and no clinical benefit relative to placebo.", "evidence_date": "2024-08", "id": "biib078", "india_note": "", "limitations": [ "This result applies to this product and study; other approaches to C9ORF72 biology remain under investigation." ], "name": "BIIB078", "source_ids": [ "biib078-results" ], "sources": [ "https://pubmed.ncbi.nlm.nih.gov/39059407/" ], "status": "Development discontinued", "status_kind": "stopped", "subtitle": "C9ORF72-targeted antisense therapy", "summary": "Development ended after the Phase 1 randomized study in C9ORF72-associated ALS.", "trial_name": "BIIB078" } ], "india_research": [ { "evidence": "The funder describes an India–UK collaboration involving NIMHANS, with genetic analysis and longitudinal samples. This is research into disease risk and biology, not a treatment trial.", "evidence_date": "2025-05-13", "id": "india-genetics", "india_note": "Ask the treating neurology center about genetic counseling, clinically appropriate testing and any currently open research protocol.", "limitations": [ "Research participation is not a guarantee of free clinical genetic testing, a diagnosis or access to a targeted medicine." ], "name": "ALS genetics in South Asia", "source_ids": [ "india-gwas" ], "sources": [ "https://targetals.org/news/unlocking-als-risk-in-south-asian-populations/" ], "status": "Observational research", "status_kind": "support", "subtitle": "NIMHANS · India–UK collaboration", "summary": "India’s participation in a Target ALS-funded genome-wide association study broadens the ancestry represented in ALS genetics." }, { "evidence": "The 2025 Global Natural History / Longitudinal Biofluid Study poster identifies AIIMS New Delhi and NIMHANS among participating institutions. Target ALS’s January 2026 update also describes tissue and genomic research resources.", "evidence_date": "2026-01-23", "id": "india-biofluids", "india_note": "Confirm the exact protocol and recruitment status directly with the institution before arranging travel.", "limitations": [ "Institutional involvement does not prove that a specific clinic is recruiting today or providing experimental treatment." ], "name": "Longitudinal samples and natural history", "source_ids": [ "india-biofluids", "research-cores" ], "sources": [ "https://targetals.org/wp-content/uploads/2025/12/TALS_AM25_Biofluids_V4_Final.pdf", "https://targetals.org/news/target-als-research-cores-empowering-global-als-discovery/" ], "status": "Research infrastructure", "status_kind": "support", "subtitle": "Target ALS · AIIMS New Delhi · NIMHANS", "summary": "Following participants over time can connect molecular changes with clinical progression." }, { "evidence": "CDSCO provides a personal-use import process: Form 12A is the application and Form 12B is the permit. Permission, prescription, product supply and clinical suitability require separate confirmation.", "evidence_date": "2015-05-21", "id": "india-access", "india_note": "Work with the treating team and licensed supply channels. Obtain written confirmation before committing to treatment-related travel or expense.", "limitations": [ "Import permission is not Indian marketing approval or evidence that a medicine works. This page does not certify current prices, reimbursement or supply." ], "name": "Treatment and trial access from India", "source_ids": [ "cdsco-import" ], "sources": [ "https://www.cdsco.gov.in/opencms/opencms/en/consumer/Drugs-for-Personal-Use/" ], "status": "Check case by case", "status_kind": "support", "subtitle": "Approval · enrollment · personal import", "summary": "A treatment’s approval abroad, a company’s Indian presence and trial eligibility are separate questions." } ], "preclinical_research": [ { "evidence": "The identified funder announcement supports preclinical studies in an inherited optic-nerve disease. It does not establish an ALS clinical trial or human ALS efficacy.", "evidence_date": "2026-06-22", "id": "asha624", "india_note": "Research only; no verified clinical access for ASHA-624.", "limitations": [ "Do not equate promising work in another disease model with an available ALS therapy. NB-4746 is the distinct SARM1 program now in ALS clinical testing." ], "name": "ASHA-624 and axon protection", "research_area": "ASHA-624 and axon protection", "source_ids": [ "asha624", "nb4746-update" ], "sources": [ "https://www.advanciumhealth.org/news-and-resources/advancium-health-network-catalyzes-research-on-an-innovative-sarm1-inhibitor-in-a-rare-eye-disease", "https://www.globenewswire.com/news-release/2026/06/22/3315110/0/en/nura-bio-completes-73-8m-series-b-financing-and-advances-pipeline-of-sarm1-inhibitors.html" ], "status": "Preclinical", "status_kind": "preclinical", "subtitle": "SARM1 biology", "summary": "ASHA-624 is a molecular-glue strategy designed to hold SARM1 inactive and protect axons." }, { "evidence": "A 2025 Nature Communications study used a high-fidelity Cas13 system in experimental models of C9ORF72-linked disease and reduced molecular abnormalities.", "evidence_date": "2025-01-08", "id": "crispr", "india_note": "No approved CRISPR treatment for ALS is established by this study.", "limitations": [ "This is laboratory and animal-model evidence. Delivery, off-target effects and long-term safety need evaluation before claims of human benefit." ], "name": "RNA-targeting CRISPR", "research_area": "RNA-targeting CRISPR", "source_ids": [ "crispr" ], "sources": [ "https://www.nature.com/articles/s41467-024-55548-5" ], "status": "Preclinical", "status_kind": "preclinical", "subtitle": "C9ORF72-linked ALS / FTD", "summary": "Researchers are investigating precise removal of disease-associated repeat RNA while preserving normal gene activity." }, { "evidence": "The Science paper reports rescue of disease-related phenotypes in models by blocking repeat-associated translation without removing the repeat RNA.", "evidence_date": "2026-02-05", "id": "ran-translation", "india_note": "A research finding, not a clinical treatment or enrollment offer.", "limitations": [ "Mechanistic advances can identify future targets, but do not demonstrate an effective medicine in patients." ], "name": "Blocking toxic repeat translation", "research_area": "Blocking toxic repeat translation", "source_ids": [ "ran-translation" ], "sources": [ "https://pubmed.ncbi.nlm.nih.gov/41643021/" ], "status": "Preclinical", "status_kind": "preclinical", "subtitle": "C9ORF72-linked ALS / FTD", "summary": "A 2026 study separates the toxicity of repeat-derived proteins from the effects of the repeat RNA itself." }, { "evidence": "Dewpoint selected a development candidate in January 2026 on the basis of cell and animal-model findings.", "evidence_date": "2026-01-08", "id": "tdp43-condensates", "india_note": "No clinical access is established by the cited preclinical program.", "limitations": [ "A development candidate is not a tested or approved ALS treatment. No human efficacy result is established in the cited announcement." ], "name": "TDP-43 condensate modulation", "research_area": "TDP-43 condensate modulation", "source_ids": [ "dewpoint" ], "sources": [ "https://dewpointx.com/dewpoint-therapeutics-announces-tdp-43-condensate-modulator-development-candidate-for-treatment-of-als-and-related-neurodegenerative-diseases/" ], "status": "Development candidate", "status_kind": "preclinical", "subtitle": "Dewpoint Therapeutics", "summary": "Small molecules are being developed to correct abnormal TDP-43 condensates and restore RNA-processing functions." } ], "symptom_management": [ { "drug_name": "Dextromethorphan / quinidine", "evidence": "The FDA indication addresses pseudobulbar affect. It is not an approval for slowing ALS progression or extending survival.", "evidence_date": "2010-10-29", "id": "nuedexta", "india_note": "Local availability and alternatives should be discussed with the neurologist; no current Indian price or supply guarantee is established here.", "limitations": [ "Suitability and interactions need a prescribing clinician. Symptom benefit should not be counted as disease modification." ], "name": "Dextromethorphan / quinidine", "source_ids": [ "nuedexta-fda" ], "sources": [ "https://www.accessdata.fda.gov/drugsatfda_docs/appletter/2010/021879s000ltr.pdf" ], "status": "Symptom treatment", "status_kind": "support", "subtitle": "Nuedexta · pseudobulbar affect", "summary": "US-approved for pseudobulbar affect: episodes of involuntary laughing or crying that can occur in neurological conditions, including ALS." } ], "withdrawn_treatments": [ { "drug_name": "Relyvrio / Albrioza", "evidence": "Amylyx announced withdrawal in April 2024 following PHOENIX, which did not meet its prespecified primary and secondary endpoints.", "evidence_date": "2024-04-04", "id": "relyvrio", "india_note": "Do not present this as a currently marketed ALS treatment or recommend purchasing a substitute combination.", "limitations": [ "Earlier approval and positive small-study results do not outweigh a failed confirmatory trial. Current care should be discussed with the treating clinician." ], "name": "Relyvrio / Albrioza", "source_ids": [ "relyvrio-withdrawal" ], "sources": [ "https://investors.amylyx.com/news-releases/news-release-details/amylyx-pharmaceuticals-announces-formal-intention-remove" ], "status": "Withdrawn from market", "status_kind": "stopped", "subtitle": "AMX0035 · sodium phenylbutyrate / taurursodiol", "summary": "The medicine was removed from the US and Canadian markets after its confirmatory trial failed." } ] }, "coverage": "A curated review of all previously included programs, with selected important additions. It is not an exhaustive registry of every ALS study worldwide.", "data_sources": [ "Lacomblez et al. · The Lancet: Dose-ranging study of riluzole in amyotrophic lateral sclerosis", "Cochrane: Riluzole for ALS / motor neuron disease", "FDA: FDA approves oral edaravone for adults with ALS", "FDA: Approval of tofersen for ALS associated with a SOD1 mutation", "Miller et al. · JAMA Neurology: Long-Term Tofersen in SOD1 Amyotrophic Lateral Sclerosis", "PMDA / MHLW: Rozebalamin (mecobalamin): review and deliberation report", "Oki et al. · JAMA Neurology: Efficacy and Safety of Ultrahigh-Dose Methylcobalamin in Early-Stage ALS", "FDA: Nuedexta approval letter: pseudobulbar affect", "European Medicines Agency: Masitinib AB Science: refusal of marketing authorisation", "AB Science: Clinical program update: planned AB23005 and temporary European trial halt", "Prilenia and Ferrer: First participant enrolled in the PREVAiLS Phase 3 study", "HEALEY ALS Platform Trial · JAMA: Pridopidine in Amyotrophic Lateral Sclerosis", "Clene · SEC exhibit: Second quarter 2026 results and CNM-Au8 regulatory update", "HEALEY ALS Platform Trial · JAMA: CNM-Au8 in Amyotrophic Lateral Sclerosis", "BrainStorm Cell Therapeutics: Second quarter 2026 results and ENDURANCE preparation", "QurAlis: Interim ANQUR data for QRL-201", "NeuroSense: Revised strategy for PrimeC and planned PARAGON study", "Coya Therapeutics: Second quarter 2026 results and ALSTARS update", "Mass General Brigham: HEALEY ALS Platform Trial: study drugs and enrollment", "Spinogenix: Topline Phase 2a clinical trial results for SPG302", "Alchemab Therapeutics · PR Newswire: Initiation of Phase 1 clinical trial of ATLX-1282", "Axoltis Pharma: SEALS preliminary results at ENCALS 2026", "Amylyx Pharmaceuticals: LUMINA cohort and biomarker update at ENCALS", "Nura Bio · GlobeNewswire: First ALS participant dosed with NB-4746", "Celosia Therapeutics · PR Newswire: First patient dosed in the Phase 1b CTx1000 trial", "Chen et al. · Nature Medicine: Oligonucleotide–siRNA conjugate for SOD1 ALS: a phase 1 trial", "Amylyx Pharmaceuticals: Formal intention to remove RELYVRIO / ALBRIOZA from the market", "Calico: Fosigotifator HEALEY trial results", "Denali Therapeutics: Topline results for HEALEY Regimen G (DNL343)", "Biogen and Ionis: BIIB105 development discontinued after ALSpire", "Paganoni et al. · JAMA Network Open: Efficacy and Safety of Zilucoplan in ALS", "HEALEY ALS Platform Trial · JAMA Neurology: Verdiperstat in ALS: results from the randomized HEALEY trial", "HEALEY ALS Platform Trial · The Lancet Neurology: Safety and efficacy of trehalose in ALS", "van den Berg et al. · The Lancet Neurology: BIIB078 in C9orf72-associated ALS: phase 1 study", "Advancium Health Network: Preclinical ASHA-624 research in autosomal dominant optic atrophy", "McCallister et al. · Nature Communications: High-fidelity CRISPR-Cas13 for C9ORF72-linked ALS/FTD", "Science · PubMed: Blocking RAN translation without altering repeat RNAs rescues C9ORF72-related ALS and FTD phenotypes", "Dewpoint Therapeutics: TDP-43 condensate modulator development candidate", "Target ALS: Unlocking ALS risk in South Asian populations", "Target ALS: Global Natural History / Longitudinal Biofluid Study poster", "Target ALS: Research cores: global discovery and long-read sequencing", "CDSCO: Drugs for personal use: Form 12A, Form 12B and import procedure", "Tobii Dynavox: Assistive communication solutions for ALS", "EyeControl: How the eye-tracking communication platform works", "OptiKey maintainers: Optikey versions and eye-tracker compatibility", "Pupil Labs: Neon ecosystem and real-time API", "Enable Viacam: Configuration wizard and head tracking", "UC Davis Health: Real-time speech neuroprosthesis in a participant with ALS", "Eisai: Rozebalamin approved in Japan on 24 September 2024", "VectorY Therapeutics: VTx-002 vectorized antibody mechanism", "Corcept Therapeutics: Two-year DAZALS results and planned Phase 3 study", "Leal Therapeutics · PR Newswire: First participant dosed in NeurALS Phase 1/2", "Trace Neuroscience, Inc. · ClinicalTrials.gov: FUNCtion ALS: Aiming to Restore UNC13A Function in People Living With ALS", "Prilenia · ClinicalTrials.gov: Pridopidine Phase 3 Study to Evaluate Efficacy and Safety in ALS", "Brainstorm-Cell Therapeutics · ClinicalTrials.gov: Efficacy and Safety of MSC-NTF (NurOwn) in Participants With Early Symptomatic ALS and Moderate Disease Presentation in ALS (ENDURANCE STUDY)", "AB Science · ClinicalTrials.gov: Efficacy and Safety of Masitinib in Combination With SoC Versus Placebo in the Treatment of ALS Patients", "Ionis Pharmaceuticals, Inc. · ClinicalTrials.gov: FUSION: A Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of ION363 in Amyotrophic Lateral Sclerosis Participants With Fused in Sarcoma Mutations (FUS-ALS)", "QurAlis Corporation · ClinicalTrials.gov: A Study Evaluating the Safety and Tolerability of QRL-201 in ALS", "Coya Therapeutics · ClinicalTrials.gov: Study of COYA 302 for the Treatment of ALS", "Merit E. Cudkowicz, MD · ClinicalTrials.gov: HEALEY ALS Platform Trial - Regimen I NUZ-001", "Spinogenix · ClinicalTrials.gov: Study of SPG302 in Healthy Volunteers and ALS Participants", "Spinogenix · ClinicalTrials.gov: Intermediate-size Patient Population Expanded Access Protocol", "Axoltis Pharma · ClinicalTrials.gov: ALS Phase II Study of NX210c", "XellSmart Bio-Pharmaceutical (Suzhou) Co., Ltd. · ClinicalTrials.gov: The Safety, Tolerability and Preliminary Efficacy of Derived Motor Neuron Progenitor Cells (XS228CN) in Subjects With Amyotrophic Lateral Sclerosis", "Amylyx Pharmaceuticals Inc. · ClinicalTrials.gov: AMX0114 in Adult Participants With Amyotrophic Lateral Sclerosis", "Nura Bio · ClinicalTrials.gov: A Clinical Trial to Evaluate NB-4746 in Participants With Amyotrophic Lateral Sclerosis.", "Celosia Therapeutics Pty Ltd · ClinicalTrials.gov: Safety and Tolerability Study of CTx1000 In Participants With Amyotrophic Lateral Sclerosis", "Ractigen Therapeutics. · ClinicalTrials.gov: A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of RAG-17 in Subjects With Amyotrophic Lateral Sclerosis (ALS) With Superoxide Dismutase Type 1 (SOD1) Gene Mutation", "Zydus Therapeutics Inc. · ClinicalTrials.gov: Usnoflast Neuromuscular Investigation for Treatment Efficacy in Amyotrophic Lateral Sclerosis", "Regeneron Pharmaceuticals · ClinicalTrials.gov: First in Human (FIH) Study of ALN-SOD in Adult Participants With Amyotrophic Lateral Sclerosis Associated With Mutation in the SOD1 Gene (SOD1-ALS)", "Vector Y Therapeutics · ClinicalTrials.gov: Study is to Assess the Safety and Tolerability of VTx-002 in Participants With ALS", "Insmed Gene Therapy LLC · ClinicalTrials.gov: A Study to Investigate the Safety and Pharmacodynamics of a Single Intrathecal Injection (IT) of INS1202 in Participants With Amyotrophic Lateral Sclerosis (ALS)", "Washington University School of Medicine · ClinicalTrials.gov: Tofersen in Non-SOD1 ALS", "Corcept Therapeutics · ClinicalTrials.gov: Dazucorilant in Patients With Amyotrophic Lateral Sclerosis", "Leal Therapeutics, Inc · ClinicalTrials.gov: A Study of LTX-002 in Adult Participants With Amyotrophic Lateral Sclerosis", "Sanofi · ClinicalTrials.gov: Phase 2 Study for SAR443820 in Participants With Amyotrophic Lateral Sclerosis (ALS)", "Eyefree Assisting Communication Ltd · ClinicalTrials.gov: Safety and Feasibility of the EyeControl Device", "Paradromics · ClinicalTrials.gov: Connect-One: Early Feasibility Study of Connexus® Brain-Computer Interface (BCI)" ], "highlights": [ { "date": "2026-09-09", "entry_id": "trcn1023", "label": "New study", "source_id": "NCT07674667", "text": "FUNCtion ALS is listed as recruiting in the latest registry record.", "title": "UNC13A restoration enters the trial landscape" }, { "date": "2026-08-31", "entry_id": "primec", "label": "Development update", "source_id": "primec-update", "text": "The proposed PARAGON redesign still requires FDA alignment.", "title": "PrimeC revises its pivotal study plan" }, { "date": "2026-08-14", "entry_id": "cnm-au8", "label": "Regulatory plans", "source_id": "clene-update", "text": "Clene targets early Q4 2026; a submission is not an approval.", "title": "CNM-Au8 application remains planned" } ], "homepage_entries": [ "tofersen", "pridopidine", "cnm-au8", "nurown", "masitinib" ], "last_updated": "2026-09-10", "methodology": [ "Evidence cutoff: 10 September 2026. Sources were retrieved on 11 September 2026; dated developments after the cutoff are excluded.", "Regulators establish approved indications. Trial registries establish the recorded design, phase and recruitment status. Registry dates are shown because records can lag behind events.", "Published controlled results take precedence over subgroup claims. Sponsor announcements are labeled and used for operational milestones or explicitly preliminary findings.", "Undated manufacturer pages describe product capabilities only; they do not establish a dated approval or verified availability on the cutoff date.", "A biomarker measures a biological process. A change in NfL, TDP-43 or another marker is not automatically a demonstrated clinical benefit.", "No medical specialist sign-off is claimed. Treatment suitability, genetic findings, access and current recruitment should be confirmed with the relevant clinical team." ], "review_changes": [ "Replaced unsupported stage-by-duration rules and blanket treatment recommendations with study-population and evidence limitations.", "Separated Nuedexta symptom treatment from the four ALS-directed medicines reviewed here; clarified jurisdiction-specific approvals.", "Corrected Rozebalamin to mecobalamin / E0302; removed the incorrect MT-1186 identification.", "Corrected CNM-Au8, NurOwn, masitinib, PrimeC and NUZ-001 milestones using dated sources and registry records.", "Added newer UNC13A, SARM1, RNA, gene-therapy and communication studies; kept every original topic or a clearly explained successor entry.", "Made the previously hidden withdrawal, communication and experimental-technology content visible.", "Removed unsupported Indian price, import-access and future availability promises; corrected Form 12A versus Form 12B.", "Corrected OptiKey 4 versus legacy Tobii compatibility and removed unsourced device approval and superiority claims." ], "reviewed_on": "2026-09-11", "schema_version": 2, "sections": [ { "categories": [ "approved_treatments", "symptom_management" ], "description": "Jurisdiction and indication matter. ALS-directed medicines and treatment for pseudobulbar affect are shown separately.", "icon": "circle-check", "id": "approved", "label": "Approved medicines", "title": "Approved medicines" }, { "categories": [ "clinical_trials" ], "description": "Recruiting studies, follow-up cohorts and planned programs. A trial phase or regulatory designation does not establish efficacy.", "icon": "flask", "id": "trials", "label": "Clinical trials", "title": "Clinical trials & development" }, { "categories": [ "preclinical_research" ], "description": "Laboratory findings and development candidates. The path from a biological idea to a useful treatment remains uncertain.", "icon": "microscope", "id": "preclinical", "label": "Early science", "title": "Early science & discovery" }, { "categories": [ "withdrawn_treatments", "failed_discontinued_trials" ], "description": "Negative findings remain part of the evidence. A failed endpoint, a stopped program and a market withdrawal are different outcomes.", "icon": "circle-minus", "id": "stopped", "label": "Negative & withdrawn", "title": "Negative trials & withdrawn medicines" }, { "categories": [ "communication_technology" ], "description": "Assistive technology can support independence and expression. It does not slow ALS biology.", "icon": "comment", "id": "communication", "label": "Communication tools", "title": "Communication tools" }, { "categories": [ "experimental_communication_tech" ], "description": "Separate experimental interfaces and custom prototypes from established, individually assessed AAC.", "icon": "microchip", "id": "experimental", "label": "Emerging technology", "title": "Emerging communication technology" }, { "categories": [ "india_research" ], "description": "Local research participation and practical questions about genetics, trials and access to treatment.", "icon": "globe", "id": "india", "label": "India & access", "title": "India research & access" } ], "source_library": [ { "accessed_on": "2026-09-11", "id": "riluzole-trial", "kind": "Randomized trial", "published_on": "1996-05-25", "publisher": "Lacomblez et al. · The Lancet", "title": "Dose-ranging study of riluzole in amyotrophic lateral sclerosis", "url": "https://pubmed.ncbi.nlm.nih.gov/8676624/" }, { "accessed_on": "2026-09-11", "id": "riluzole-review", "kind": "Systematic review", "published_on": "2021-07-01", "publisher": "Cochrane", "title": "Riluzole for ALS / motor neuron disease", "url": "https://www.cochrane.org/evidence/CD001447_riluzole-amyotrophic-lateral-sclerosis-alsmotor-neuron-disease-mnd" }, { "accessed_on": "2026-09-11", "id": "edaravone-fda", "kind": "Regulator", "published_on": "2022-05-12", "publisher": "FDA", "title": "FDA approves oral edaravone for adults with ALS", "url": "https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-oral-form-treatment-adults-amyotrophic-lateral-sclerosis-als" }, { "accessed_on": "2026-09-11", "id": "tofersen-fda", "kind": "Regulator", "published_on": "2023-04-25", "publisher": "FDA", "title": "Approval of tofersen for ALS associated with a SOD1 mutation", "url": "https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-treatment-amyotrophic-lateral-sclerosis-associated-mutation-sod1-gene" }, { "accessed_on": "2026-09-11", "id": "tofersen-longterm", "kind": "Trial and open-label extension", "published_on": "2025-12-22", "publisher": "Miller et al. · JAMA Neurology", "title": "Long-Term Tofersen in SOD1 Amyotrophic Lateral Sclerosis", "url": "https://jamanetwork.com/journals/jamaneurology/fullarticle/2843130" }, { "accessed_on": "2026-09-11", "id": "rozebalamin-pmda", "kind": "Regulator", "published_on": "2024-09-03", "publisher": "PMDA / MHLW", "title": "Rozebalamin (mecobalamin): review and deliberation report", "url": "https://www.pmda.go.jp/files/000277177.pdf" }, { "accessed_on": "2026-09-11", "id": "jetals", "kind": "Randomized trial", "published_on": "2022-05-09", "publisher": "Oki et al. · JAMA Neurology", "title": "Efficacy and Safety of Ultrahigh-Dose Methylcobalamin in Early-Stage ALS", "url": "https://jamanetwork.com/journals/jamaneurology/fullarticle/2792228" }, { "accessed_on": "2026-09-11", "id": "nuedexta-fda", "kind": "Regulator", "published_on": "2010-10-29", "publisher": 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Lateral Sclerosis", "url": "https://jamanetwork.com/journals/jama/fullarticle/2830508" }, { "accessed_on": "2026-09-11", "id": "nurown-update", "kind": "Sponsor update", "published_on": "2026-08-14", "publisher": "BrainStorm Cell Therapeutics", "title": "Second quarter 2026 results and ENDURANCE preparation", "url": "https://ir.brainstorm-cell.com/2026-08-14-BrainStorm-Cell-Therapeutics-Announces-Second-Quarter-2026-Financial-Results-and-Provides-Corporate-Update" }, { "accessed_on": "2026-09-11", "id": "qrl201-update", "kind": "Sponsor update", "published_on": "2026-02-23", "publisher": "QurAlis", "title": "Interim ANQUR data for QRL-201", "url": "https://www.quralis.com/news/quralis-demonstrates-effects-on-disease-progression-and-target-engagement-in-anqur-clinical-trial-of-qrl-201-a-first-in-class-precision-medicine-in-development-for-sporadic-als/" }, { "accessed_on": "2026-09-11", "id": "primec-update", "kind": "Sponsor update", "published_on": "2026-08-31", "publisher": "NeuroSense", "title": "Revised strategy for PrimeC and planned PARAGON study", "url": "https://neurosense.investorroom.com/2026-08-31-NeuroSense-Unveils-Accelerated,-Capital-Efficient-Strategy-to-Advance-PrimeC-Toward-Potential-Approval-in-ALS" }, { "accessed_on": "2026-09-11", "id": "coya-update", "kind": "Sponsor update", "published_on": "2026-08-11", "publisher": "Coya Therapeutics", "title": "Second quarter 2026 results and ALSTARS update", "url": "https://ir.coyatherapeutics.com/news/news-details/2026/Coya-Therapeutics-Reports-Second-Quarter-2026-Financial-Results-and-Provides-a-Corporate-Update/default.aspx" }, { "accessed_on": "2026-09-11", "id": "healey", "kind": "Trial center", "published_on": null, "publisher": "Mass General Brigham", "title": "HEALEY ALS Platform Trial: study drugs and enrollment", "url": "https://research.massgeneralbrigham.org/en/institutes-centers/neuroscience-institute/department-of-neurology/sean-healey-amg-center-for-als/healey-als-platform-trial" }, 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"https://www.axoltis.com/2026/06/24/encals-2026-european-network-to-cure-als/" }, { "accessed_on": "2026-09-11", "id": "amx0114-update", "kind": "Sponsor update", "published_on": "2026-06-23", "publisher": "Amylyx Pharmaceuticals", "title": "LUMINA cohort and biomarker update at ENCALS", "url": "https://investors.amylyx.com/news-releases/news-release-details/amylyx-pharmaceuticals-present-data-first-human-dose-ranging" }, { "accessed_on": "2026-09-11", "id": "nb4746-update", "kind": "Sponsor update", "published_on": "2026-06-22", "publisher": "Nura Bio · GlobeNewswire", "title": "First ALS participant dosed with NB-4746", "url": "https://www.globenewswire.com/news-release/2026/06/22/3315110/0/en/nura-bio-completes-73-8m-series-b-financing-and-advances-pipeline-of-sarm1-inhibitors.html" }, { "accessed_on": "2026-09-11", "id": "ctx1000-update", "kind": "Sponsor update", "published_on": "2026-03-23", "publisher": "Celosia Therapeutics · PR Newswire", "title": "First patient dosed in the 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"publisher": "Calico", "title": "Fosigotifator HEALEY trial results", "url": "https://www.calicolabs.com/press/calico-statement-on-fosigotifator-in-healey-als-platform-trial/" }, { "accessed_on": "2026-09-11", "id": "dnl343-results", "kind": "Sponsor update", "published_on": "2025-01-06", "publisher": "Denali Therapeutics", "title": "Topline results for HEALEY Regimen G (DNL343)", "url": "https://investors.denalitherapeutics.com/node/10826/pdf" }, { "accessed_on": "2026-09-11", "id": "biib105-results", "kind": "Sponsor update", "published_on": "2024-05-16", "publisher": "Biogen and Ionis", "title": "BIIB105 development discontinued after ALSpire", "url": "https://investors.biogen.com/news-releases/news-release-details/biogen-and-ionis-announce-topline-phase-12-study-results" }, { "accessed_on": "2026-09-11", "id": "zilucoplan-results", "kind": "Randomized trial", "published_on": "2025-02-17", "publisher": "Paganoni et al. · JAMA Network Open", "title": "Efficacy and Safety of 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Brain-Computer Interface (BCI)", "url": "https://clinicaltrials.gov/study/NCT07357428" } ] }