|Biomarin Pharmaceutical Inc., of San Rafael, Calif.||Valoctocogene roxaparvovec||AAV gene therapy||Severe hemophilia A||Multiyear follow-up phase I/II data published in The New England Journal of Medicine demonstrated that a single infusion resulted in sustained, clinically relevant benefit, as measured by a substantial reduction in annualized rates of bleeding events and complete cessation of prophylactic factor VIII use in all 13 participants who had received 4e13vg/kg or 6e13 vg/kg|
|Sol-Gel Technologies Ltd., of Ness Ziona, Israel||SGT-210||Topical EGFR inhibitor||Punctate palmoplantar keratoderma type 1||Started proof-of-concept study; top-line data expected in first half of 2021|
|Akcea Therapeutics Inc., of Boston, a majority-owned affiliate of Ionis Pharmaceuticals Inc.||AKCEA-APO(a)-LRx1 (TQJ-230)||Antisense drug designed to inhibit production of apolipoprotein||Cardiovascular disease with elevated Lp(a)||Results published in The New England Journal of Medicine showed dose-dependent reductions in Lp(a) at all doses studied, with an up to 80% reduction at highest cumulative dose regimen; about 98% of patients on 80-mg monthly dose achieved Lp(a) levels below 125 nmol/L|
|Durect Corp., of Cupertino, Calif.||DUR-928||Sulfated oxysterol||Mild to moderate plaque psoriasis||Did not demonstrate benefit vs. placebo on primary analysis based on Investigator’s Global Assessment, or any secondary analyses; company does not plan to continue development in psoriasis|
|Gamida Cell Ltd., of Jerusalem||Omidubicel||Cell therapy||Bone marrow transplant in high-risk hematologic malignancies||Completed patient enrollment; top-line data expected in first half of 2020|
For more information about individual companies and/or products, see Cortellis.