Courier crosses the blood-brain barrier, and innovative therapy for ALS enters phase 2 clinical trials

▎WuXi AppTec Content Team Editor Recently, Denali Therapeutics announced that its partner, Sanofi, has started a Phase 2 clinical trial to test the efficacy and safety of SAR443820 (DNL788) in the treatment of amyotrophic lateral sclerosis (ALS)
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SAR443820 is a central nervous system (CNS) penetrating small molecule inhibitor of RIPK1
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ALS, also known as ALS, has an average lifespan of about 2 to 5 years, and there is currently no cure for the disease
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RIPK1 is a key signaling protein in the tumor necrosis factor (TNF) receptor signaling pathway, which regulates inflammation and cell death
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Elevated RIPK1 activity in the brain drives neuroinflammation and necrosis, and it is thought to be associated with a variety of CNS and peripheral diseases
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In preclinical models of ALS and multiple sclerosis (MS), inhibition of RIPK1 activity can delay disease progression
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Image source: Denali's official website Sanofi and Denali reached a collaboration to develop RIPK1 inhibitors in October 2018
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Sanofi will lead the Phase 1 and 2 clinical development of SAR443820 in ALS and MS, and together with Denali will conduct Phase 3 clinical trials of SAR443820 in ALS, MS and Alzheimer's disease
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In a phase 1 clinical trial in healthy volunteers, SAR443820 demonstrated potent interactions with its target at well-tolerated doses
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Based on these results, Sanofi has decided to initiate a Phase 2 clinical trial in ALS patients and plans to initiate a Phase 2 clinical trial in MS patients
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The US FDA has granted Fast Track designation to SAR443820 for the treatment of ALS
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"The initiation of this Phase 2 clinical trial marks an important milestone for
the SAR443820 development program and our collaborative program with Sanofi's RIPK1 inhibitor ," said Denali Chief Medical Officer Dr.
Carole Ho.
"We are delighted that Denali's pipeline for ALS has gained This progress .
Denali is committed to working with the ALS patient community towards the common goal of developing life-saving therapies for patients .
” Reference: [1] Denali Therapeutics Announces Achievement of RIPK1 Milestone for Phase 2 Clinical Trial Initiation in ALS by Sanofi .
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