Company Product Description Indication Status
Acasti Pharma Inc., of Laval, Quebec Capre Omega-3 phospholipid Hypertriglyceridemia Meeting request submitted to FDA to discuss phase III Trilogy 1 study, which missed statistical significance for primary endpoint, and gain alignment on data interpretation; FDA input also will be sought on proposed revisions to prespecified statistical analysis plan for still-blinded Trilogy 2 trial and potential plan to pool data from Trilogy studies to support NDA filing; meeting expected in second half of June 2020
Aveo Pharmaceuticals Inc. (Aveo Oncology), of Cambridge, Mass. Fotivda (tivozanib) VEGF receptor-tyrosine kinase inhibitor Renal cell carcinoma NDA submitted to FDA to treat individuals with relapsed/refractory disease
Concert Pharmaceuticals Inc., of Lexington, Mass. CTP-543 (deuterium-modified ruxolitinib) JAK1/JAK2 tyrosine kinase inhibitor Alopecia areata Completed end-of-phase II meeting with FDA, reaching agreement on planned phase III program to include 2 trials in adults at sites in the U.S., Canada and Europe to evaluate Severity of Alopecia Tool score after 24 weeks of dosing in participants with moderate to severe disease
Cytodyn Inc., of Vancouver, Wash. Leronlimab CCR5 antagonist COVID-19 infection Additional clinical trial protocol filed with FDA to treat individuals with severe disease under same FDA-approved IND as recently initiated phase II trial in those with mild to moderate disease; new protocol is for 342 participants, double-blinded with 2-to-1 ratio, with primary endpoint of mortality rate at 14 days
Diurnal Group plc, of London Chronocort (modified release hydrocortisone) Glucocorticoid receptor agonist Congenital adrenal hyperplasia MAA passed validation with EMA
Faron Pharmaceuticals Oy, of Turku, Finland Interferon-beta-1a (intravenous; also Traumakine) Interferon-beta ligand COVID-19-related acute respiratory distress syndrome Regulatory approval granted for global Remap-Cap (Randomized, Embedded, Multifactorial Adaptive Platform Trial for Community-Acquired Pneumonia) program that includes Faron candidate, with primary outcome of 90-day all-cause mortality
LFB SA, of  Paris Sevenfact  Coagulation factor VIIa (recombinant)-njcw Hemophilia A and B FDA approved use to treat and control bleeding episodes in adults and adolescents 12 and older with inhibitors
Mallinckrodt plc, of Staines-upon-Thames, U.K., and Novoteris LLC, of Garden Grove, Calif. Thiolanox  Nitric oxide inhalant COVID-19-associated lung complications Health Canada's Therapeutic Products Directorate cleared joint pilot trial assessing Novoteris' inhaled nitric oxide delivery device and Mallinckrodt's high-concentration nitric oxide gas for inhalation canisters; recruitment expected to begin within days at Vancouver Coastal Health Authority facilities
Mesoblast Ltd., of Sydney Ryoncil (remestemcel-L) Mesenchymal stem cell therapy Graft-vs.-host disease FDA accepted BLA filing to treat children with steroid-refractory acute disease, granting priority review and setting Sept. 30, 2020, PDUFA date
Orchard Therapeutics plc, of London OTL-200 ARSA gene stimulator Metachromatic leukodystrophy Despite continuing engagement with EMA on submitted MAA under accelerated assessment, decision likely to be postponed due to COVID-19 pandemic; potential launch in Europe now expected in first half of 2021; BLA submission to FDA also postponed to first half of 2021
Orchard Therapeutics plc, of London OTL-101 (simoladagene autotemcel) ADA gene stimulator Severe combined immunodeficiency COVID-19-related clinical and manufacturing impacts expected to delay initiation of rolling BLA to FDA, previously planned for first half of 2020
Y-mabs Therapeutics Inc., of New York Naxitamab Anti-GD2 3F8 monoclonal antibody Neuroblastoma Rolling BLA submission to FDA completed for treatment of those with  relapsed/refractory high-risk disease

Notes

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

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