Clinical Trials
30
17 active
Approvals
3
Total approvals
Agencies
2
Regulatory bodies
Founded
2014
Active, not recruiting
17
56.7%
Approved For Marketing
1
3.3%
Available
1
3.3%
Completed
6
20.0%
Recruiting
5
16.7%
- Novartis discontinued development of VHB937 (lifonebart) after the Phase 2 ASTRALS trial in early-stage ALS missed both primary and secondary endpoints. - The 251-patient trial tested the TREM2-stabilizing monoclonal antibody or placebo for 40 weeks, with no numerical data released by the company. - The setback adds to a difficult pipeline stretch for Novartis, following Phase 3 misses for pelacarsen and del-desiran and halted rap-cel studies. - Detailed ASTRALS findings are scheduled for presentation at the 37th International Symposium on ALS/MND in Amsterdam on December 9-11, 2026.
- No disease-modifying therapies currently exist for Parkinson's disease, with only three of 84 Phase 2 and 3 DMT studies registered over the last decade testing combination approaches. - A Cure Parkinson's workshop in June 2025 explored how combination therapies, proven in oncology and infectious disease, could address the molecular complexity and patient heterogeneity of PD. - Case studies including Auvelity (dextromethorphan-bupropion) and AMX0035 (sodium phenylbutyrate-TUDCA) illustrate the mechanistic, regulatory, and patenting considerations for developing combination treatments. - Workshop attendees emphasized early regulatory engagement, patient stratification, and multi-arm platform trials as key strategies for advancing combination DMTs in PD.
- Amylyx Pharmaceuticals has nominated AMX0318, a novel long-acting GLP-1 receptor antagonist, as a development candidate for post-bariatric hypoglycemia and other rare diseases. - The compound was identified through collaboration with Gubra A/S using their AI-driven streaMLine platform and demonstrated robust chemical stability, strong in vitro potency, and favorable pharmacokinetic properties. - IND-enabling studies are expected to begin in 2026 with an IND submission targeted for 2027, while Amylyx continues its Phase 3 LUCIDITY trial of avexitide. - Under the collaboration terms, Gubra is eligible for over $50 million in development milestones plus mid-single digit royalties, with $4 million in immediate milestone payments.
- Amylyx Pharmaceuticals presented early safety and tolerability data from the Phase I LUMINA trial of AMX0114, an antisense oligonucleotide targeting calpain-2 for ALS treatment. - The first cohort of 12 participants showed AMX0114 was generally well-tolerated with no treatment-related serious adverse events reported. - Based on these positive safety results, Amylyx plans to begin enrolling the second cohort in Canada in December and in the US in January 2026. - The company expects biomarker data from the first cohort, including neurofilament light chain levels, in the first half of 2026.
- Amylyx Pharmaceuticals' AMX0035 failed to meet primary or secondary endpoints in a mid-stage trial for progressive supranuclear palsy, leading to discontinuation of the study and related extension trial. - The company will continue developing AMX0035 for Wolfram syndrome while prioritizing a late-stage study of a blood sugar-adjusting medicine acquired in summer 2024. - AMX0035 previously received U.S. approval for ALS treatment but was withdrawn from the market in 2024, eliminating Amylyx's main revenue source. - The company expects its cash runway to extend through the end of next year despite the setback.
- Amylyx Pharmaceuticals has discontinued the ORION program of AMX0035 for progressive supranuclear palsy after the drug failed to show differences compared to placebo on primary or secondary outcomes at Week 24. - The Phase 2b trial, which targeted 110 patients, assessed disease progression as the primary endpoint, with AMX0035 also failing to outperform placebo in motor aspects of daily living activities. - Despite the setback, Amylyx will continue developing AMX0035 for Wolfram syndrome and maintains focus on its Phase 3 LUCIDITY trial of avexitide for post-bariatric hypoglycemia, with enrollment completion expected in 2025. - The company expects its cash runway to extend through the end of 2026 and will also progress AMX0114 development in ALS with early cohort data expected in 2025.
- The HDAC inhibitors market across seven major markets is expected to surge significantly by 2034, driven by innovation in cancer therapies and expanding applications beyond traditional hematologic malignancies. - Several next-generation HDAC inhibitors are advancing through clinical trials, including Abexinostat in Phase III for renal cell carcinoma and Quisinostat in Phase II for uveal melanoma. - Recent market entrants include DUVYZAT for Duchenne muscular dystrophy and HIYASTA for T-cell lymphomas, demonstrating the therapeutic potential of HDAC inhibitors beyond oncology applications. - The market faces challenges from toxicity issues and competition from newer targeted therapies, but combination approaches with immune checkpoint inhibitors and precision oncology trends are creating new growth opportunities.
- The progressive supranuclear palsy (PSP) market is experiencing significant growth driven by emerging therapies including AZP2006 and AMX0035, which are advancing through clinical trials with promising results. - AZP2006 from AlzProtect demonstrated positive clinical and biomarker efficacy signals in a Phase IIa trial and has received orphan drug designation from both FDA and EMA. - AMX0035 from Amylyx Pharmaceuticals is currently being evaluated in a global Phase IIb/III ORION trial, with safety and efficacy data expected in Q3 2025. - Strategic collaborations and regulatory incentives are accelerating drug development, while improved diagnostic capabilities are creating favorable conditions for therapeutic interventions in this rare neurodegenerative disorder.
- DelveInsight's analysis reveals 4+ key companies are developing 6+ therapies for congenital hyperinsulinism, with emerging treatments including CRN-04777, HM 15136, RZ358, and dasiglucagon expected to significantly impact the market. - Recent regulatory developments include FDA's Breakthrough Therapy Designation for RZ358 in January 2025 and removal of clinical holds in September 2024, while Zealand Pharma received a Complete Response Letter for dasiglucagon in October 2024. - The congenital hyperinsulinism market is anticipated to grow with significant CAGR during 2020-2034, driven by increasing disease awareness and research activities, though challenges remain with high treatment costs and diagnostic complexities.
- Biogen's Qalsody (tofersen) receives FDA accelerated approval as the first therapy for SOD1-mutated ALS, marking a significant breakthrough in genetic-specific ALS treatment. - The approval is based on the drug's ability to reduce neurofilament light chain (NfL) biomarker levels by 55% over 28 weeks, representing the first use of a blood biomarker for neurological drug approval. - Biogen plans to launch Qalsody within a week, with patient out-of-pocket costs capped at $50 per month, while conducting the confirmatory ATLAS trial through 2027.
