July 25 Clinical Quick Takes: J&J’s ponesimod hits MS endpoint; plus a CRISPR first, Genkyotex and Aeglea

Janssen’s ponesimod tops Aubagio in Phase III
The Janssen Pharmaceuticals unit of Johnson & Johnson (NYSE:JNJ) said it plans to submit NDAs to FDA and EMA this year after reporting ponesimod met the primary endpoint of improving annualized relapse rate vs. Aubagio teriflunomide in the Phase III OPTIMUM trial to treat relapsing multiple sclerosis. The S1PR1 modulator also met most secondary endpoints. Johnson & Johnson gained ponesimod in its 2017 acquisition of Actelion Ltd. (see "Actelion, Take Two").

First in vivo CRISPR trial for LCA10 under way
Allergan plc (NYSE:AGN) and Editas Medicine Inc. (NASDAQ:EDIT) have begun enrolling patients in the Phase I/II Brilliance trial of AGN-151587 (EDIT-101) to treat Leber congenital amaurosis type 10, which the companies said is the world’s first clinical trial of an in vivo CRISPR-based therapy. The partners plan to evaluate the therapy targeting the CEP290 gene in 18 adult and pediatric patients with LCA10 and expect to dose the first patient this half. Vertex Pharmaceuticals Inc. (NASDAQ:VRTX) and CRISPR Therapeutics AG (NASDAQ:CRSP) began the first clinical trial of a CRISPR gene therapy in September (see "Vertex Becomes First U.S. Company to Enter Clinic with CRISPR Therapy")...