Company Product Description Indication Status
Phase I
Adverum Biotechnologies Inc., of Redwood City, Calif. ADVM-022 (AAV.7m8 vector encoding aflibercept) VEGF gene inhibitor Wet age-related macular degeneration First of 9 participants in cohort 4 of ongoing Optic trial dosed with single intravitreal injection of 6 x 10 ^11 vg/eye (same as cohort 1) and steroid eye drop prophylaxis for 6 weeks (same as cohort 3)
Harpoon Therapeutics Inc., of South San Francisco HPN-217 APRIL receptor/albumin/CD3 modulator Multiple myeloma First participant dosed in phase I/II trial enrolling those with relapsed/refractory disease; primary outcome measures of phase I portion include safety, tolerability, pharmacokinetics and determination of phase II dose; secondary endpoints include overall response rate, progression-free and overall survival and duration of response
Hua Medicine Ltd., of Shanghai Dorzagliatin Glucokinase stimulator Type 2 diabetes In combination HMM0112 study, co-administration of dorzagliatin (75 mg twice daily) and 25 mg once daily of SGLT-2 inhibitor empagliflozin (Jardiance, Boehringer Ingelheim GmbH/Eli Lilly and Co.) showed no impact on respective pharmacokinetic properties; following oral glucose tolerance test, combination treatment showed enhanced glucose lowering effect (AUEC: 279 h•mg/dL) over empagliflozin (AUEC: 452 h•mg/dL, p<0.01) or dorzagliatin (AUEC: 364 h•mg/dL, p<0.05) monotherapy
Phase II
Axsome Therapeutics Inc., of New York AXS-05 NMDA receptor antagonist Agitation Phase II/III Advance-1 trial in individuals with Alzheimer's disease met primary endpoint, showing statistically significant mean reduction in Cohen Mansfield Agitation Inventory (CMAI) total score vs. placebo at week 5, with mean reductions from baseline of 15.4 vs. 11.5 points, respectively (p=0.010), representing mean percentage reduction from baseline of 48% vs. 38%, respectively; improvement on CMAI total score was numerically superior to placebo starting at week 2 and achieved statistical significance at week 3 (p=0.007)
Italfarmaco SpA, of Milan Givinostat HDAC inhibitor Duchenne muscular dystrophy Follow-up analyses from ongoing long-term extension showed delay in disease progression in those treated with study drug and steroids for > 6 years compared to natural history study by Cooperative International Neuromuscular Research Group (CINRG); mean age of loss of ambulation in treated participants was 15.2 years vs. 13.4 years in CINRG study; yearly rate of decline of Forced Vital Capacity % Predicted and Peak Expiratory Flow % Predicted was 2.3% and 0%, respectively, in treated participants vs. 4% to 6% yearly rate of decline in both measures in natural history studies of patient population comparable to givinostat cohort
Sanofi SA, of Paris, and Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y. Kevzara (sarilumab) IL-6 receptor antagonist COVID-19 infection Phase II portion in individuals hospitalized with severe or critical respiratory illness showed study drug lowered C-reactive protein, meeting primary endpoint; based on differences between severe and critically ill participants and recommendation of independent data monitoring committee, ongoing phase III trial amended to enroll only critical patients and to compare only higher dose (400 mg) vs. placebo
Phase III
Avadel Pharmaceuticals plc, of Dublin FT-218 (sodium oxybate, micropump-controlled release) GABA B receptor agonist Narcolepsy Pivotal Rest-On trial showed highly statistically significant improvements vs. placebo on 3 co-primary endpoints of Maintenance of Wakefulness Test, Clinical Global Impression-Improvement and mean weekly cataplexy attacks at 3 tested doses (9 g, 7.5 g and 6 g)
CTI Biopharma Corp., of Seattle Pacritinib JAK2/IRAK1/CSF1R kinase inhibitor COVID-19 infection Pre-Vent trial initiated to assess study drug plus standard of care (SOC) vs. placebo plus SOC in 358 individuals, with or without cancer, hospitalized with severe infection; primary endpoint is proportion who progress to invasive mechanical ventilation and/or extracorporeal membrane oxygenation or die by day 28; data expected by year-end 2020
Italfarmaco SpA, of Milan Givinostat HDAC inhibitor Duchenne muscular dystrophy Based on interim imaging analysis that showed lower muscle fat infiltration with study drug vs. placebo after 12 months of dosing, independent data monitoring committee recommended continuation of Epydis trial; blinded sample size re-estimation supported reducing enrollment to 169 from 242 participants, which could close enrollment as early as July 2020 and provide final results in 2022
Myovant Sciences Ltd., of Basel, Switzerland Relugolix  Oral gonadotropin-releasing hormone receptor antagonist Uterine fibroids Abstracts with findings from Liberty 1 and 2 studies published in Obstetrics & Gynecology showed combination with estradiol and norethindrone acetate in women with heavy menstrual bleeding include pooled data showing 65% treated with combo reported no or minimal pain during menstrual days vs.19.3% for placebo; 44.6% of women in combo group reported no or minimum pain during non-menstrual days vs. 21.6% in placebo group; quality-of-life outcomes showed women in combo group experienced significant improvement in symptom severity from baseline to week 24 (both studies p < 0.0001) from 55.1 to 23.4 compared with 60.3 to 49.2 for placebo in Liberty 1, and from 59.1 to 21.7 compared with 59.2 to 45.1 for placebo in Liberty 2
Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y., and Sanofi SA, of Paris Libtayo (cemiplimab) Anti-PD-1 antibody Non-small-cell lung cancer Primary endpoint of overall survival was met in trial comparing Libtayo to platinum-doublet chemotherapy in patients with first-line locally advanced or metastatic disease that tested positive for PD-L1 in ≥50% of tumor cells; based on recommendation by independent data monitoring committee to stop the trial early, the study will be modified to allow all patients to receive Libtayo; regulatory submissions expected in U.S. and EU in 2020
Romark Laboratories LC, of Tampa, Fla. NT-300 (nitazoxanide extended-release tablets) Inhibits viral replication COVID-19 Plans to start 2 trials for prevention of COVID-19 and other viral respiratory illnesses in high-risk populations, including elderly residents of long-term care facilities and health care workers; in both studies, participants will receive either NT-300 or placebo for 6 weeks, and the primary endpoint is the rate of COVID-19 illness for NT-300 vs. placebo


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