Company Product Description Indication Status
Phase I
Arcutis Biotherapeutics Inc., of Westlake Village, Calif. ARQ-252 Topical small-molecule JAK1 inhibitor Chronic hand eczema Started enrollment of phase IIb portion of phase I/IIb study; top-line data expected in second half of 2021
Calithera Biosciences Inc., of South San Francisco CB-280 Arginase inhibitor Cystic fibrosis Dosed first patients in phase Ib trial in CF and chronic airway infection; trial will test safety and optimal dose range of drug when added to existing CF therapies, including CFTR modulators
Catalyst Biosciences Inc., of South San Francisco MarzAA (marzeptacog alfa) Next-generation subcutaneous factor VIIa candidate Hemophilia Final data from MAA-102 trial demonstrated potential of MarzAA to rapidly achieve and maintain therapeutic levels to treat acute bleeding events in hemophilia and confirms dosing regimen chosen for upcoming phase III trial, Crimson 1
Freeline Therapeutics Ltd., of London FLT-180a AAV-based gene therapy Hemophilia B Further data from B-Amaze phase I/II trial showed, in the first, low-dose cohort (4.5 x1011), durable expression of FIX activity now seen for 2 years post dose, with average FIX level activity at 52 and 104 weeks of 38% and 38% FIX activity, respectively; durable FIX activity levels in normal range were seen with 7.5 x1011 and 9.75x1011 doses; latest cohort, at dose level 4 (9.75x1011), has been expanded to 4 patients, all of whom are showing encouraging FIX expression greater than 50% as of June 15, 2020
Junshi Biosciences Ltd., of Shanghai JS-016 SARS-CoV-2 neutralizing antibody COVID-19 Completed enrollment testing single dose in healthy subjects; no dose-limiting events observed as of July 12, 2020
Kodiak Sciences Inc., of Palo Alto, Calif. KSI-301 Intravitreal anti-VEGF antibody biopolymer conjugate Wet age-related macular degeneration, diabetic macular edema and retinal vein occlusion Additional phase Ib data in treatment-naïve patients showed 82% of wet AMD eyes and 76% of DME eyes were extended to 4 months or longer after last loading dose before receiving their first retreatment; 68% of wet AMD eyes achieved 6-month interval at least once during follow-up; in DME, with all patients having been followed for 6 months or longer after 3 initial loading doses (vs. 5 required with current standard of care), nearly half have yet to require retreatment
Nextcure Inc., of Beltsville, Md. NC-318 Immunomedicine against S15 Multiple cancer types Based on interim update for phase II portion of phase I/II monotherapy trial, company does not plan to advance non-small-cell lung cancer and ovarian cancer cohorts into stage II portion; continues to enroll in head and neck squamous cell carcinoma (1 confirmed partial response) and triple-negative breast cancer cohorts
Rocket Pharmaceuticals Inc., of New York RP-L301 Lentiviral vector-based gene therapy Pyruvate kinase deficiency First patient received treatment in open-label trial
Spark Therapeutics, of Philadelphia, a member of the Roche Group SPK-8011 Gene therapy Hemophilia A Updated phase I/II data showed, as of June 3, 2020, cutoff, results from 5 total participants in the 5x1011 vg/kg and 1×1012-dose cohorts and 7 in the 2×1012 vg/kg-dose cohort had acceptable safety profile, 91% reduction in annualized bleed rate, 96% reduction in FVIII infusions and stable and durable factor FVIII expression after between 2 and 3.3 years of follow-up
Phase II
Adverum Biotechnologies Inc., of Redwood City, Calif. ADVM-022 Gene therapy using vector capsid AAV.7m8 to carry an aflibercept coding Diabetic macular edema First patient randomized to Infinity trial; data expected in second half of 2021
Biosig Technologies Inc., of Westport, Conn., and subsidiary Viralclear Pharmaceuticals Inc. Merimepodib Broad-spectrum, oral antiviral COVID-19 Enrolled adults with advanced COVID-19
Summit Therapeutics plc, of Oxford, U.K. Ridinilazole Antibiotic C. difficile infection Data published in the American Journal of Physiology – Gastrointestinal and Liver Physiology demonstrated microbiome preservation resulted in gut environment expected to inhibit the growth of C. difficile; in contrast, vancomycin treatment resulted in gut environment that may more highly favor the growth of C. difficile; difference in gut environment could explain the roughly 60% relative reduction in recurrence observed in patients treated with ridinilazole over vancomycin in the trial
Trevi Therapeutics Inc., of New Haven, Conn. Haduvio (nalbuphine ER) Oral, extended-release formulation of mixed ĸ-opioid receptor agonist and µ-opioid receptor antagonist Severe pruritis in prurigo nodularis Completed prespecified size re-estimation analysis of ongoing Prism phase IIb/III study, with independent data monitoring committee recommending that study continue, with trial size increased from initial enrollment target of 240 to 360, which maintains statistical power for primary endpoint; top-line data expected in fourth quarter of 2021
Phase III
Avadel Pharmaceuticals plc, of Dublin FT-218 Once-nightly formulation of sodium oxybate Excessive daytime sleepiness and cataplexy in narcolepsy Dosed first patient in open-label extension/switch study
Bellerophon Therapeutics Inc., of Warren, N.J. Inopulse Inhaled nitric oxide therapy COVID-19 First patient initiated treatment; Covinox study will test therapy in patients who require supplemental oxygen; primary endpoint will assess proportion of subjects that had respiratory failure or mortality, which should allow trial to serve as a registrational study for approval
Celsion Corp., of Lawrenceville, N.J. Thermodox Lyso-thermosensitive liposomal doxorubicin Hepatocellular carcinoma  Received recommendation from independent data monitoring committee to consider stopping Optima trial testing combination with radiofrequency ablation, after second preplanned interim analysis found prespecified boundary for stopping the trial for futility of 0.900 was crossed, with actual value of 0.903
Chugai Pharmaceutical Co. Ltd., of Tokyo Hemlibra (emicizumab) Bispecific factor IXa- and factor X-directed antibody Hemophilia A Second interim analysis from Stasey study showed drug was effective with no new safety signals identified in adults and adolescents; additional results suggest people with hemophilia A with FVIII inhibitors who undergo certain minor surgeries while on treatment may not need additional preventative coagulation factor; of unplanned major surgeries (n=9), 8 were managed with prophylactic coagulation factor, 4 of which resulted in bleeds managed successfully with recombinant FVIIa
Genentech, of South San Francisco, a member of the Roche Group Tecentriq (atezolizumab) PD-L1-targeting antibody Ovarian cancer Imagyn050 study showed addition of Tecentriq to Avastin (bevacizumab), paclitaxel and carboplatin did not meet primary endpoint of progression-free survival for front-line treatment of women with newly diagnosed advanced-stage disease; data for overall survival co-primary endpoint are currently immature, and follow-up will continue until the next planned analysis

Notes

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