Company Product Description Indication Status
Phase I
Aileron Therapeutics Inc., of Watertown, Mass. ALRN-6924 Dual MDM2/MDMX inhibitor Myelopreservation Enrolled first small-cell lung cancer patient in open-label phase Ib schedule optimization part of ongoing phase Ib/II trial, evaluating ALRN-6924’s potential to protect patients against chemotherapy-induced toxicities
Intra-Cellular Therapies Inc., of New York ITI-214 PDE1 inhibitor Heart failure with reduced ejection fraction In phase I/II study of single ascending doses vs. placebo in 35 participants with chronic systolic disease, cardiac output was improved through increased heart contractility and decreased vascular resistance
TFF Pharmaceuticals Inc., of Austin, Texas Tacrolimus Dry powder formulated with TFF's Thin Film Freezing platform Lung transplant rejection Initiated study to assess safety, tolerability and pharmacokinetics of direct-to-lung delivery of a dry-powder inhaled tacrolimus product for prevention of lung transplant rejection
TFF Pharmaceuticals Inc., of Austin, Texas Voriconazole Dry powder formulated with TFF's Thin Film Freezing platform Invasive pulmonary aspergillosis Top-line data indicated safety of single doses of voriconazole inhalation powder up to 80-mg/dose in healthy subjects for direct-to-lung delivery
Phase II
Immutep Ltd., of Sydney Eftilagimod alpha (IMP-321) HLA class II antigen stimulator Non-small-cell lung cancer Final participant with first-line disease dosed in stage 2 of part A of Tacti-002 combination trial with Keytruda (pembrolizumab, Merck & Co. Inc.); additional data expected in second half of 2020
KBP Biosciences Co. Ltd., of Princeton, N.J. KBP-5074 Mineralocorticoid receptor antagonist Hypertension Design of Block CKD trial published in Hypertension; ongoing phase IIb study recently completed enrollment of participants with advanced chronic kidney disease, with primary efficacy endpoint of change in trough cuff seated systolic blood pressure from baseline to day 84 of each dose (0.25 mg, 0.5 mg) vs. placebo; top-line data expected in fourth quarter of 2020
Revimmune Inc., unit of Revimmune SAS, of Paris CYT-107 IL-7 receptor agonist COVID-19 infection Launched Iliad trial to assess therapy's ability to increase number of immune T cells and correct immune exhaustion associated with infection
Phase III
9 Meters Biopharma Inc., of Raleigh, N.C. Larazotide Zonulin inhibitor Celiac disease After consultation with FDA, modified definition of primary endpoint in ongoing trial; validated instrument used for primary assessment remains Celiac Disease Patient-Reported Outcome but definition of primary endpoint now continuous variable instead of responder analysis, with study population reduced to 525 participants from 630 while maintaining 90% statistical powering; interim analysis still anticipated during first half of 2021 and top-line data in second half of 2021
Achieve Life Sciences Inc., of Seattle Cytisinicline Nicotinic ACh receptor alpha 4/beta 2 subunit modulator Nicotine dependence Top-line results from New Zealand Rauora noninferiority trial vs. varenicline (Chantix, Pfizer Inc.) in 679 participants met primary endpoint, showing study drug plus behavioral support was at least as effective as comparator plus behavioral support at 6 months, measured by biochemically confirmed continuous abstinence rates, with fewer reported adverse events; final results submitted for presentation at Society for Research on Nicotine and Tobacco Europe annual meeting in September 2020
Biocardia Inc., of San Carlos, Calif. Cardiamp cell therapy  Autologous bone marrow-derived stem cell therapy + Helix device Heart failure Pivotal trial resumed enrollment following pause due to COVID-19 pandemic
Cytodyn Inc., of Vancouver, Wash. Leronlimab CCR5 antagonist  COVID-19 infection Completed memorandum of understanding with Coordinating Commission of the NIH and High Specialty Hospitals of Mexico to conduct trial in severe and critically ill individuals; Cytodyn to supply study drug at its expense, with parties collaborating on study protocol
Immunovant Inc., of New York IMVT-1401 Dual IgG receptor FcRn large subunit p51 antagonist/modulator Myasthenia gravis Phase III development accelerated; FDA input will be sought on design and conduct of pivotal program
Iterum Therapeutics plc, of Dublin Sulopenem Penem beta-lactam antibiotic Uncomplicated urinary tract infections In Sure1 trial, study drug achieved primary endpoint in participants with baseline pathogens resistant to quinolones, showing statistically significant superiority to ciprofloxacin (p <0.001); in those with organisms susceptible to quinolones, study drug missed second primary endpoint of noninferiority to ciprofloxacin, attributed to rate of asymptomatic bacteriuria post treatment
Kiniksa Pharmaceuticals Ltd., of Hamilton, Bermuda Rilonacept Dual interleukin-1 alpha/beta ligand inhibitor Pericarditis Pivotal Rhapsody trial met primary endpoint of time-to-first adjudicated disease recurrence in randomized withdrawal period with high statistical significance; median (95% CI) time to recurrence for study drug could not be estimated due to low number in treatment arm vs. median time to recurrence of 8.6 [4-11.7] weeks for placebo (p<0.0001); major secondary efficacy endpoints in randomized withdrawal period also were highly statistically significant
Montreal Heart Institute, of Montreal Colchicine  Microtubule function inhibitor COVID-19  Colcorona trial in non-hospitalized adult patients passed futility test in interim analysis and will continue; seeks to determine if colchicine may prevent inflammatory storm observed in adults suffering from serious complications linked to infection
Tonix Pharmaceuticals Holding Corp., of New York TNX-102 (cyclobenzaprine hydrochloride, sublingual) 5-HT 2a receptor antagonist; alpha 2 adrenoceptor antagonist Fibromyalgia Pivotal Relief trial expected to reach full enrollment ahead of schedule, with top-line results expected in fourth quarter of 2020 rather than first quarter of 2021; optional interim analysis of first 50% of randomized participants evaluable for efficacy will be conducted, with results expected in September 2020
Phase IV
Medicure Inc., of Winnipeg, Manitoba Aggrastat (tirofiban hydrochloride) GP IIb IIIa antagonist Acute coronary syndrome Data from investigator-sponsored Fabolus-Faster trial, published in Circulation, showed Aggrastat achieved superior inhibition of platelet aggregation at 30 minutes vs. comparator cangrelor (Kengreal, Chiesi Farmaceutici SpA) (95%±9% vs. 34.1%±22.5%; p<0.001)

Notes

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

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