Denali, Sanofi start with dosing patients in Phase 1 testing DNL747 in patients with ALS

Denali Therapeutics and Sanofi in partnership have started dosing patients in a clinical Phase 1b study of DNL747 in patients with ALS.

Carole Ho, Chief Medical Officer of Denali, said that Phase 1 data in healthy volunteer subjects demonstrating DNL747’s excellent CNS penetration is a good ground for evaluating DNL747 in ALS patients. She said that the primary purpose of this Phase 1b study is to gain additional safety and biomarker data in ALS patients to support dose selection. Hi sadi that the results from this study will inform decisions by Denali and Sanofi on further clinical testing of DNL747, including potential registrational trials.

RIPK1, receptor-interacting serine/threonine-protein kinase 1, is a critical signaling protein in the TNF receptor pathway, which regulates inflammation and cell death in tissues throughout the body. Denali, together with its partner Sanofi, is investigating several molecules targeting RIPK1 for multiple indications, including DNL747 for ALS.

To remind, Sanofi will make an upfront cash payment to Denali of $125 million, with future development and commercial milestone payments that could exceed $1 billion. Sanofi and Denali will share commercial profits and losses from DNL747 in the U.S. and China equally, while Denali will receive a royalty from Sanofi for other territories for DNL747 and worldwide for DNL758.