CNM-Au8
- Generic Name
- CNM-Au8
- Drug Type
- Biotech
China Approves First Stem Cell Therapy and Global Advances in Cell & Gene Therapies
China has approved its first stem cell therapy, marking a significant milestone in the field of cell and gene therapies. Meanwhile, global efforts are underway to improve regulatory pathways, access, and manufacturing methods for these innovative treatments, with particular focus on rare diseases and the challenges of regulatory convergence.
Denali Therapeutics' DNL343 Fails to Meet Primary Endpoint in HEALEY ALS Platform Trial
• Denali Therapeutics' DNL343, an eIF2B agonist, did not meet the primary endpoint of slowing ALS disease progression in a Phase 2/3 trial.
• The HEALEY ALS Platform Trial's Regimen G assessed DNL343's impact on disease severity and survival over 24 weeks compared to placebo.
• While DNL343 was safe and well-tolerated, key secondary endpoints like muscle strength and respiratory function showed no significant difference.
• Further analyses, including biomarker assessments, are planned for 2025 to explore potential subgroup benefits and long-term effects.
FDA Roundup: Approvals, Breakthrough Designations, and Clinical Holds in Neurology and Rare Diseases
• The FDA granted breakthrough therapy designation to STK-001 for Dravet syndrome, highlighting its potential to improve upon current treatments by restoring NaV1.1 protein levels.
• Tolebrutinib received breakthrough therapy designation for non-relapsing secondary progressive multiple sclerosis based on phase 3 trial results showing delayed disability progression.
• The FDA placed a clinical hold on PepGen’s PGN-EDO51 phase 2 study for Duchenne muscular dystrophy, pending further clarification from the agency.
Clene's CNM-Au8 Receives FDA Guidance for Accelerated Approval in ALS Treatment
• Clene Inc. has received FDA guidance for a potential accelerated approval pathway for CNM-Au8 in treating amyotrophic lateral sclerosis (ALS).
• The FDA recommends leveraging additional Neurofilament Light (NfL) data from expanded access programs and the HEALEY ALS Platform Trial.
• Clene plans to submit a New Drug Application (NDA) in mid-2025 after completing NfL biomarker analyses and will initiate a Phase 3 trial.
• Clinical data shows CNM-Au8 has a strong safety profile and potential survival benefits, including a 78% risk reduction in time to death.
uniQure's AMT-130 Receives FDA Nod for Accelerated Approval Pathway in Huntington's Disease
• The FDA has agreed that existing Phase I/II data for uniQure's AMT-130, compared to external controls, can support a Biologics License Application for accelerated approval.
• The composite Unified Huntington’s Disease Rating Scale (cUHDRS) is accepted by the FDA as an intermediate clinical endpoint for AMT-130.
• Reductions in neurofilament light chain (NfL) levels in cerebrospinal fluid may serve as supportive evidence of AMT-130's therapeutic benefit.
• uniQure plans to engage with the FDA in early 2025 to discuss statistical analysis and CMC requirements for AMT-130.
HEALEY ALS Platform Trial: Streamlining Drug Development for Amyotrophic Lateral Sclerosis
• The HEALEY ALS Platform Trial is pioneering a new approach to accelerate the development of effective treatments for amyotrophic lateral sclerosis (ALS).
• By testing multiple drugs simultaneously using a shared infrastructure, the platform trial significantly reduces the time and cost associated with traditional clinical trials.
• Initial results from the platform have led to two drugs, CNM-Au8 and pridopidine, advancing to Phase 3 testing based on promising trends in secondary outcome measures and biomarker data.
• The collaborative effort unites patients, clinicians, scientists, and industry partners, fostering innovation and improving access to care across a network of over 70 sites.
Multiple Sclerosis Pipeline Shows Promise with Novel Therapies in Development
• The multiple sclerosis (MS) therapeutic landscape is expanding, with over 80 active pipeline therapies currently in development by more than 75 companies.
• Recent clinical trials have yielded mixed results, with some therapies showing promise in specific MS subtypes, such as non-relapsing secondary progressive MS (nrSPMS).
• Regulatory milestones have been achieved, including FDA approval for new formulations and fast-track designations for therapies targeting progressive MS.
• Emerging therapies in the MS pipeline include monoclonal antibodies, oral treatments, and CAR-T cell therapies, offering diverse mechanisms of action and routes of administration.
Clene to Meet with FDA to Discuss Accelerated Approval for CNM-Au8 in ALS
• Clene will meet with the FDA to discuss accelerated approval of CNM-Au8 for amyotrophic lateral sclerosis (ALS).
• The meeting will involve senior FDA leadership and experts to discuss ALS biomarkers, clinical endpoints, and survival data related to CNM-Au8.
• CNM-Au8, a gold nanoparticle suspension, aims to support nerve cell energy needs and has shown potential in slowing ALS progression.
• Clene is seeking accelerated approval based on data demonstrating CNM-Au8 significantly lowers neurofilament light chain (NfL) levels, a biomarker of nerve cell damage.
Clene to Meet with FDA to Discuss CNM-Au8 for ALS
• Clene Inc. will meet with the FDA before the end of November 2024 to discuss CNM-Au8's development for amyotrophic lateral sclerosis (ALS).
• The FDA agreed to re-evaluate Clene's submission under the accelerated approval pathway after initial concerns regarding the briefing package.
• Clene has accumulated over 700 patient-years of safety data for CNM-Au8, showing no significant safety concerns or trends.
• No serious adverse events related to CNM-Au8 treatment have been identified by clinical trial investigators to date.
ALS Clinical Trial Landscape: Investigational Therapies Targeting Sporadic ALS
• Several clinical trials are underway for sporadic ALS, targeting TDP-43 pathology, STMN2 expression, and neuroinflammation.
• Biogen's BIIB-105, AbbVie/Calico's ABBV-CLS-7262, Denali's DNL-343, and QurAlis' QRL-201 are among the therapies currently being evaluated in clinical trials.
• Prilenia's Pridopidine and Clene's CNM-Au8 have completed Phase 2 trials, showing some positive trends in motor function and survival benefits, respectively.
• The FDA is set to make a decision on Brainstorm Cell's NurOwn, a mesenchymal stem cell therapy, with an advisory committee meeting scheduled for September 27th.
New Drug Development Pipeline Shows Promise for ALS Treatment with 53 Candidates Under Investigation
• A comprehensive review of ALS drug development reveals 53 new drug candidates in clinical trials between 2020-2022, with 13 compounds advancing to Phase 3 trials and showing promising therapeutic potential.
• Five drugs demonstrated particularly strong efficacy: high-dose methylcobalamin, masitinib, AMX0035, CNM-Au8, and tofersen, with AMX0035 receiving FDA approval as the third treatment for ALS.
• The success of clinical trials is influenced by patient population homogeneity, observation duration, and analysis strategies, highlighting the importance of refined trial design in ALS drug development.
Drug Development Updates
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