Company Product Description Indication Status
Phase I
Bluesky Immunotherapies GmbH, of Vienna DelNS HPV 16 Interferon-inducing viral vector HPV 16 tumors Tolerability of the drug was confirmed; removal of HPV 16 infection and the elimination of tumors will be assessed later
Sarepta Therapeutics, of Cambridge, Mass. SRP-9003 Gene therapy expressing beta-sarcoglycan (beta-SG) Limb-girdle muscular dystrophy type 2E In the phase I/II study, 24 months after treatment with the 1.85×10^13-vg/kg dose, mean intensity of transduced beta-SG was 35%, compared to 47% at day 60; mean NSAD improvement from baseline of 5.7 points at 18 months was sustained through 24 months; 60 days after treatment with the 7.41×10^13-vg/kg dose, mean intensity expression of beta-SG was 73%; after 12 months, mean NSAD improvement from baseline was 4 points, compared to 3.7 at 6 months
Phase II
Amyndas Pharmaceuticals Inc., of Athens, Greece AMY-101 Complement C3 inhibitor Periodontal inflammation and gingivitis Treatment once a week for 3 weeks reduced bleeding (p<0.001) and gingival inflammation index (p<0.001); benefit was maintained for at least 90 days
Phase III
Clovis Oncology Inc., of Boulder, Colo. Rubraca (rucaparib) Inhibitor of PARP1, PARP2 and PARP3 Advanced, relapsed ovarian cancer and a deleterious BRCA mutation after 2 or more lines of chemotherapy In the Ariel 4 study, median progression-free survival was 7.4 months for Rubraca compared to 5.7 months for chemotherapy (HR-0.64, p=0.001)
Idera Pharmaceuticals Inc., of Exton, Pa. Tilsotolimod  Toll-like receptor 9 agonist Anti-PD-1 refractory advanced melanoma In the Illuminate-301 study, tilsotolimod plus Yervoy (ipilimumab, Bristol Myers Squibb Co.) produced an objective response rate of 8.8% compared to 8.6% for Yervoy alone; disease control rate was 34.5% for the combination and 27.2% for Yervoy alone
Incyte Corp., of Wilmington, Del. Jakafi (ruxolitinib) JAK1/JAK2 inhibitor COVID-19-associated acute respiratory distress syndrome In the Devent study, overall survival through day 29 was 55.2% for Jakafi compared to 74.3% for placebo (p=0.0280) in the 5-mg arm and 51.8% for Jakafi compared to 69.6% for placebo (p=0.0292) in the 15-mg arm
Merck & Co. Inc., of Kenilworth, N.J., and Eisai Co., Ltd., of Tokyo Lenvima (lenvatinib) and Keytruda (pembrolizumab) Receptor tyrosine kinase inhibitor and monoclonal antibody targeting PD-1 Advanced, metastatic or recurrent endometrial cancer following 1 prior platinum-based regimen  In Study 309/Keynote-775, the combination reduced the risk of disease progression or death by 44% (p<0.0001), with a median progression-free survival of 7.2 months compared to 3.8 months for chemotherapy; overall survival for the combination was 18.3 months compared to 11.4 months for chemotherapy (p<0.0001)


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