Company Product Description Indication Status
Phase I
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
Phase II
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
Phase III
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

Notes

For more information about individual companies and/or products, see Cortellis.

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