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C19061501-F

Generic Name
C19061501-F
Clinical Trials
Related News

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

• 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

• 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.

Sanofi's Q3 Sales Surge 15.7% Driven by Vaccines and Dupixent; 2024 EPS Guidance Raised

• Sanofi reported a robust 15.7% sales growth in Q3, fueled by strong vaccine sales and the continued success of Dupixent, with sales up 23.8% to €3,476 million. • Pharma launches, including ALTUVIIIO, Nexviazyme, and Rezurock, saw a significant 67.1% increase, contributing €727 million to the overall sales growth. • The company's pipeline advanced with four regulatory approvals, including Dupixent for COPD in the US and China, and four positive Phase 3 data readouts, including tolebrutinib for nrSPMS. • Based on strong Q3 business performance, Sanofi has raised its 2024 business EPS guidance to at least a low single-digit percentage growth at CER.

Sanofi and Denali Terminate Phase 2 Multiple Sclerosis Trial of RIPK1 Inhibitor Oditrasertib After Disappointing Results

• Sanofi and Denali Therapeutics have discontinued the Phase 2 K2 clinical trial of oditrasertib in multiple sclerosis due to its failure to meet primary and key secondary endpoints. • The trial assessed oditrasertib's impact on neurofilament light chain levels in relapsing-remitting, secondary progressive, and primary progressive MS patients, but did not demonstrate significant reduction. • This setback follows a previous failure of oditrasertib in a Phase 2 trial for amyotrophic lateral sclerosis, leading to the termination of its development for that indication. • Despite the challenges, Sanofi and Denali continue to collaborate on other RIPK1 inhibitors, including eclitasertib, currently in Phase 2 development for ulcerative colitis.

Sanofi and Denali's RIPK1 Inhibitor Fails in Phase 2 ALS Trial

• Sanofi and Denali Therapeutics' investigational drug SAR443820 (DNL788), a RIPK1 inhibitor, did not meet the primary endpoint in a Phase 2 clinical trial for amyotrophic lateral sclerosis (ALS). • The trial assessed the change in the ALS Functional Rating Scale-Revised (ALSFRS-R) as the primary outcome, with Sanofi planning to present detailed efficacy and safety results at a future scientific meeting. • Despite the setback in ALS, the Phase 2 study of SAR443820 in multiple sclerosis (MS) is ongoing, and a 52-week open-label extension study in ALS will further evaluate the drug's safety and efficacy. • The failure raises questions about RIPK1 inhibition as a therapeutic strategy for ALS, impacting other companies like Eli Lilly, which has a partnership with Rigel Pharmaceuticals for RIPK1-blocking molecules.

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

• 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.
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