DNL343

Eli Lilly and Alchemab Forge $415M Partnership for Novel ALS Antibody Therapy

16 days ago

• Eli Lilly has entered a $415 million licensing agreement with UK-based Alchemab Therapeutics for ATLX-1282, a first-in-class antibody therapy for amyotrophic lateral sclerosis (ALS). • The novel antibody was discovered using Alchemab's AI-powered platform that identified protective antibodies in individuals with genetic mutations for frontotemporal dementia (FTD) who remained healthy into old age. • Alchemab will conduct early Phase 1 trials before Lilly takes over further development and commercialization, leveraging its expertise in neurological conditions to advance the therapy.

IGM Biosciences Halts Autoimmune Programs, Imvotamab and IGM-2644, Following Disappointing Phase 1b Results

4 months ago

• IGM Biosciences has stopped the development of imvotamab and IGM-2644 after Phase 1b trials showed insufficient B cell depletion in rheumatoid arthritis and lupus patients. • The company is reducing its workforce by 73% to preserve cash and is exploring strategic alternatives, including a potential sale or merger. • Interim data indicated that imvotamab, a CD20 x CD3 bispecific antibody, did not meet the company's criteria for success in B cell depletion. • The decision marks a strategic shift for IGM, which initially focused on cancer therapies before pivoting to autoimmune disease research in 2024.

Tenvie Therapeutics Launches with $200M for Neurodegeneration Drug R&D; Sana Biotechnology Achieves Landmark in Type 1 Diabetes Transplant

4 months ago

• Tenvie Therapeutics, backed by Arch Venture Partners and others, debuts with $200 million to develop drugs targeting neurodegenerative diseases. • Sana Biotechnology's technology enables successful pancreatic islet cell transplant in a Type 1 diabetes patient without immunosuppression. • Jasper Therapeutics shares mixed Phase 1b/2a data on briquilimab for chronic spontaneous urticaria, leading to a stock decline. • Stoke Therapeutics finalizes plans for a Phase 3 trial of its experimental drug for Dravet syndrome, with data expected by 2027.

Denali Therapeutics' Tividenofusp Alfa Receives FDA Breakthrough Therapy Designation for Hunter Syndrome

4 months ago

• Denali Therapeutics' tividenofusp alfa (DNL310) has received Breakthrough Therapy Designation from the FDA for Hunter syndrome (MPS II). • The designation aims to expedite the development and review of tividenofusp alfa, a potential improvement over existing therapies. • Denali plans to submit a Biologics License Application (BLA) in early 2025 for accelerated approval of tividenofusp alfa. • Tividenofusp alfa is designed to address both neurological and physical symptoms of Hunter syndrome by crossing the blood-brain barrier.

Denali Therapeutics' ALS Trial Fails to Meet Endpoints, BofA Adjusts Stock Target

5 months ago

• Denali Therapeutics' DNL343, an eIF2B inhibitor, failed to meet primary and key secondary endpoints in a Phase 2/3 trial for amyotrophic lateral sclerosis (ALS) at 24 weeks. • BofA Securities reduced Denali's stock target to $30 from $34, removing DNL343 from their valuation model due to the uncertainty of the drug's future development. • Despite the trial setback, BofA remains optimistic about Denali's brain delivery platform and strong financial position, suggesting potential buying opportunities. • Denali anticipates submitting a BLA for tividenofusp alfa, a Hunter syndrome treatment, in early 2025, potentially transitioning to a commercial organization later that year.

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.

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.

Denali Therapeutics Announces Positive Clinical Updates and Financial Results for Q1 2024

a year ago

• Denali Therapeutics reported positive two-year clinical data for tividenofusp alfa in MPS II, showing sustained normalization of heparan sulfate in CSF and improvements in clinical outcomes. • The company initiated a Phase 1/2 clinical trial for DNL126, an enzyme replacement therapy for children with MPS IIIA, with biomarker and safety data expected by the end of 2024. • Enrollment is complete in the Phase 2/3 HEALEY ALS Platform Trial for DNL343, an eIF2B activator being developed for the treatment of amyotrophic lateral sclerosis (ALS). • Denali completed a private investment in public equity (PIPE) financing with gross proceeds of $500 million and divested its preclinical small molecule portfolio.

Denali and Sanofi's ALS Drug, SAR443820, Fails to Meet Primary Endpoint in Phase 2 Trial

a year ago

• Denali Therapeutics and Sanofi's investigational drug SAR443820/DNL788 did not meet the primary endpoint in the Phase 2 HIMALAYA trial for amyotrophic lateral sclerosis (ALS). • The primary endpoint was measured by change in the ALS Functional Rating Scale-Revised (ALSFRS-R), a tool used to assess the severity and progression of ALS. • Sanofi plans to present detailed efficacy and safety results from the HIMALAYA study at an upcoming scientific forum, while continuing to evaluate SAR443820 in multiple sclerosis. • This setback marks another challenge in the development of effective treatments for ALS, a neurodegenerative disease affecting thousands in the United States.

ALS Clinical Trial Landscape: Investigational Therapies Targeting Sporadic ALS

2 years ago

• 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.

MIROCALS Trial Results: Low-Dose IL-2 Shows Potential Benefit for Subset of MND Patients

2 years ago

• The MIROCALS trial testing low-dose interleukin-2 (IL-2) for motor neurone disease (MND) failed to meet its primary endpoint for the overall population, with results published in The Lancet on May 9, 2025. • Post-hoc analysis revealed a potential benefit for patients with slower disease progression, identified by low neurofilament levels, showing 18% higher survival rates and 23% decreased functional decline compared to placebo. • The UK MND Clinical Studies Group is currently evaluating the complex findings to determine next steps, with three MND organizations collaborating to expedite access to proven treatments.

Advancements in Neurology: From Hunter Syndrome to Alzheimer's Disease

5 years ago

Recent studies and trials in neurology have shown promising results across a range of conditions, including Hunter syndrome, Alzheimer's disease, and Dravet syndrome. Innovations in treatment and diagnostic tools are paving the way for more effective management of these complex conditions.

Drug Development Updates