Company Product Description Indication Status
Phase I
Biohaven Pharmaceutical Holding Co. Ltd., of New Haven, Conn. Zavegepant (formerly BHV-3500)  CGRP receptor antagonist Migraine; non-migraine indications Following IND clearance by FDA, dosing initiated to assess safety and pharmacokinetics of oral formulations 
Boehringer Ingelheim GmbH, of Ingelheim, Germany BI-905711  Bispecific antibody targeting TNF-related apoptosis-inducing ligand receptor 2 and cadherin 17 Gastrointestinal cancers Started study
Corvus Pharmaceuticals Inc., of Burlingame, Calif. CPI-006  Monoclonal antibody targeting CD73 COVID-19 Interim data from the first 2 cohorts showed antibodies against the SARS-CoV-2 trimeric spike and/or receptor binding domain increased in 8 of 8 evaluable patients within 7 days of a single infusion of low doses of CPI-006; antibody titers increased out to 28 days post treatment
Diffusion Pharmaceuticals Inc., of Charlottesville, Va. Trans sodium crocetinate (TSC) Increases oxygen availability Hospitalized COVID-19 Dosed first 2 of 24 patients in the phase Ib study testing safety and tolerability of TSC; study will also measure arterial blood oxygenation
Forte Biosciences Inc., of Torrance, Calif. FB-401 Roseomonas mucosa biotherapeutic Atopic dermatitis Data published in Science Translational Medicine showed 90% of the 20 pediatric patients treated with FB-401 had at least a 50% improvement in the Eczema Activity and Severity Index (EASI-50); 100% of the 9 patients with moderate to severe disease achieved EASI-50; mean improvement in the EASI score was 77%; pruritus improved by an average 58%
Gemini Therapeutics Inc., of Cambridge, Mass. GEM-103 Complement factor H  Dry age-related macular degeneration and central geographic atrophy No dose-limiting toxicities were observed in 4 ascending doses; GEM-103 prolonged supraphysiologic complement factor H
Histogen Inc., of San Diego HST-001  Hair stimulating complex enriched for growth factors including KGF, VEGF and follistatin Androgenic alopecia Completed dosing of patients in phase Ib/IIa study; top-line data expected in the fourth quarter of 2020
Imcheck Therapeutics SAS, of Marseille, France ICT-01 Monoclonal antibody targeting BTN3A Hematologic malignancies and solid tumors Independent safety review committee for the phase I/IIa Eviction study recommended escalating the dose and starting enrollment in a cohort testing the monotherapy in hematologic malignancies and a cohort testing ICT-01 plus pembrolizumab in solid tumors
Recce Pharmaceuticals Ltd., of Sydney RECCE-327 Antibiotic Healthy volunteers (eventually Escherichia coli and
infections)
Selected Cmax Clinical Research to conduct the 48-subject study testing the safety, tolerability, pharmacokinetics and pharmacodynamic profile of RECCE-327
Sanofi SA, of Paris, and Swedish Orphan Biovitrum (Sobi) AB, of Stockholm  BIVV-001 Long-lasting factor VIII Severe hemophilia A In the phase I/IIa Exten-A study published in The New England Journal of Medicine, 65 IU/kg dose of BIVV-001 had a half-life of 43 hours compared to 13 hours for recombinant factor VIII (rFVIII); 25 IU/kg dose had a half-life of 38 hours compared to 9 hours for rFVIII
Sutro Biopharma Inc., of South San Francisco STRO-002  Antibody-drug conjugate targeting folate receptor alpha Ovarian cancer Overall response rate was 24% for 33 evaluable patients treated with 2.9 mg/kg and higher dose levels
Phase II
Anavex Life Sciences Corp., of New York Anavex2-73 (blarcamesine) Activates sigma-1 receptor Rett syndrome Completed the 31-patient study; top-line data expected in the fourth quarter of 2020
Avillion LLP , of London Sonelokinab (M-1095) Anti-IL-17 A/F nanobody Moderate to severe chronic plaque psoriasis Study met its primary endpoint based on Investigator's Global Assessment at week 12 and secondary endpoints of Psoriasis Area and Severity Index (PASI) 75, PASI 90 and PASI 100 at week 12 (p<0.001 for all); data to be presented at an upcoming conference
Corcept Therapeutics Inc., of Menlo Park, Calif. Miricorilant  Glucocorticoid receptor modulator and mineralocorticoid receptor antagonist Obese patients with schizophrenia and long-standing antipsychotic-induced weight gain Enrolled first of 150 patients in the Gratitude II study comparing 2 dose levels of the drug to placebo; primary endpoint is reduction in body weight
Gemini Therapeutics Inc., of Cambridge, Mass. GEM-103 Complement factor H  Geographic atrophy secondary to dry age-related macular degeneration Started enrollment of up to 80 patients in the phase IIa Regatta study testing monthly or every other month dosing
Haliodx SAS, of Marseille, France Keytruda (pembrolizumab), Xelox (capecitabine plus oxaliplatin) and Avastin (bevacizumab) Anti-PD-1 antibody, chemotherapy and anti-VEGF antibody Microsatellite stable/proficient mismatch repair system metastatic colorectal cancer with high immune infiltrate Immunoscore will be used to decide patient eligibility in the Xelox study being run by the Francophone Federation of Digestive Oncology
Seneca Biopharma Inc., of Germantown, Md. NSI-566  Neural stem cells  Chronic ischemic stroke Completed study in China; top-line data expected in the fourth quarter of 2020
VBL Therapeutics Ltd., of Tel Aviv, Israel VB-111 CD95/TNF receptor modulator  Metastatic colorectal cancer First 2 patients enrolled in the study testing VB-111 plus Opdivo (nivolumab, Bristol Myers Squibb Co.); primary endpoints are safety and tolerability as well as best overall response
Phase III
Arbor Pharmaceuticals LLC, of Atlanta AR-19 Immediate-release, amphetamine sulfate capsule Attention-deficit/hyperactivity disorder Pairwise comparisons using an analysis of least square means of differences in the Adult ADHD Investigator Symptom Rating Scale for AR-19 vs. placebo were -7.2 for the 20-mg dose and -7.3 for the 40-mg dose (p<0.001 for both)
Astrazeneca plc, of Cambridge, U.K. Fasenra (benralizumab) Monoclonal antibody targeting IL-5 Chronic rhinosinusitis with nasal polyps Ostro study met its primary endpoint of improvement in size of nasal polyps and in nasal blockage; data to be presented at an upcoming medical meeting
Bayer AG, of Leverkusen, Germany Nubeqa (darolutamide)  Androgen receptor inhibitor  Castration-resistant prostate cancer  New data from Aramis trial, published in The New England Journal of Medicine, showed study drug + androgen deprivation therapy (ADT) improved overall survival (secondary endpoint) in men with non-metastatic disease vs. placebo + ADT, with 31% reduction in risk of death (p=0.003); Nubeqa also delayed time to pain progression and first initiation of cytotoxic chemotherapy (both p<0.001) and time to first symptomatic skeletal event (p=0.005)
Ocular Therapeutix Inc., of Bedford, Mass. Dextenza (dexamethasone ophthalmic insert) Glucocorticoid receptor agonist Ocular inflammation; pain First of about 60 participants dosed in trial assessing study drug vs. prednisolone acetate suspension eye drops following cataract surgery in children ages 0 to 3 years
Roche Holding AG, of Basel, Switzerland Enspryng (satralizumab)  IL-6 receptor antagonist Neuromyelitis optica spectrum disorder In post-hoc analysis across double-blind periods of Sakura studies, risk of severe relapse in treated group reduced by 79% vs. placebo (5 of 27 [19%] vs. 12 of 34 [35%]); in separate pooled analysis, study drug reduced risk of relapse in combined double-blind period and open-label extension by 51% (p=0.002) vs. original placebo group
Satsuma Pharmaceuticals Inc., of South San Francisco STS-101 (dihydroergotamine nasal powder) 5-HT 1d receptor agonist Acute migraine Top-line data did not show statistically significant differences between 3.9-mg and 5.2-mg doses of study drug vs. placebo on co-primary endpoints of freedom from pain and most bothersome symptom at 2 hours post-administration
Takeda Pharmaceutical Co. Ltd., of Osaka, Japan Ninlaro (ixazomib) Proteasome inhibitor Multiple myeloma Tourmaline-MM2 trial evaluating study drug + lenalidomide/dexamethasone vs. placebo + lenalidomide/dexamethasone in newly diagnosed disease missed statistical significance and primary endpoint of progression-free survival (PFS), with 13.5-month increase in median PFS (35.3 months) for Ninlaro arm vs. placebo (21.8 months) (p=0.073)
Theratechnologies Inc., of Montreal Tesamorelin GHRH receptor agonist Nonalcoholic steatohepatitis Protocol filing planned for fourth quarter of 2020 for trial expected to enroll about 650 participants with fibrosis scores of 2 and 3 and NAFLD Activity Score of at least 4, including cohort of 50 people with HIV; enrollment expected to begin in first quarter of 2021
Vertex Pharmaceuticals Inc., of Boston Trikafta (elexacaftor/tezacaftor/ivacaftor + ivacaftor) CFTR modulator/stimulator Cystic fibrosis Study in 66 children ages 6 to 11 with 2 copies of F508del mutation or 1 copy of F508del mutation and 1 minimal function mutation met primary endpoint of safety and tolerability and showed clinically meaningful improvements across secondary efficacy endpoints, including ppFEV1, sweat chloride, Cystic Fibrosis Questionnaire Revised respiratory domain score and BMI through 24 weeks; sNDA submission to FDA planned in fourth quarter of 2020
Zogenix Inc., of Emeryville, Calif. Fintepla (fenfluramine) 5-HT 1d/2a receptor modulator Dravet syndrome Dosed at 0.7 mg/kg/day, study drug achieved 64.8% greater reduction in mean monthly convulsive seizures and dosed at 0.2 mg/kg/day, 49.9% greater reduction in mean monthly convulsive seizures vs. placebo (both p<0.0001); inclusion of participants in Japan will enable Study 3 to serve as pivotal trial for J-NDA submission, expected in 2021

Notes

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