Clene Nanomedicine, Inc.

Denali Therapeutics' DNL343 Fails to Meet Primary Endpoint in HEALEY ALS Platform Trial

5 months ago

• 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.

Clene's CNM-Au8 Receives FDA Guidance for Accelerated Approval in ALS Treatment

5 months ago

• 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.

HEALEY ALS Platform Trial: Streamlining Drug Development for Amyotrophic Lateral Sclerosis

6 months ago

• 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

6 months ago

• 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.