Company Product Description Indication Status
Alkermes plc, of Dublin ALKS-3831 (olanzapine + samidorphan) Atypical antipsychotic + opioid receptor modulator Schizophrenia; bipolar I disorder FDA issued complete response letter, citing need to resolve conditions related to tablet coating process at manufacturing facility
Apellis Pharmaceuticals Inc., of Waltham, Mass. Pegcetacoplan Targeted C3 therapy Paroxysmal nocturnal hemoglobinuria FDA accepted and granted priority review designation for NDA; PDUFA date is May 14, 2021
Azurrx Biopharma Inc., of Delray Beach, Fla. MS-1819 Lipase modulator Exocrine pancreatic insufficiency Protocol amendment expected to be submitted to FDA for phase IIb Option 2 trial in people with cystic fibrosis, requesting addition of study arm using immediate-release capsules to compare data with existing arm using delayed-release capsules and select optimal delivery method; trial on target to report top-line data in first quarter of 2021
Bristol Myers Squibb Co., of New York Lisocabtagene maraleucel B-lymphocyte antigen CD19 modulator Large B-cell lymphoma FDA said it could not complete its review of BLA to treat adults with relapsed/refractory disease prior to Nov. 16, 2020, PDUFA date; agency was unable to inspect third-party manufacturing facility in Texas during review cycle due to COVID-19-related travel restrictions; new PDUFA date not set
CNS Pharmaceuticals Inc., of Houston Berubicin Topoisomerase II inhibitor Glioblastoma IND filed with FDA for phase II trial in adults who failed first-line therapy; trial expected to begin in first quarter of 2021
Cytodyn Inc., of Vancouver, Wash. Leronlimab (PRO-140) CCR5 antagonist COVID-19 infection Protocol filed with FDA to conduct phase II trial in 102 people without detectable virus but with so-called long-hauler symptoms
Inovio, of Plymouth Meeting, Pa. INO-4800 Nucleic-acid based vaccine COVID-19 prophylaxis FDA cleared the phase II/III Innovate study comparing 2 dose levels of the vaccine to placebo to determine the best dose(s) to study in the phase III portion
Istari Oncology Inc., of Durham, N.C. PVS-RIPO Live-attenuated serotype 1 poliovirus vaccine Advanced melanoma FDA accepted IND for phase II Luminos-102 trial examining therapy alone or in combination with PD-1 inhibitor in treatment-refractory disease, expected to begin in first quarter of 2021; interim analysis expected when 20 participants have been randomized and treated for 3 months; efficacy measures include objective response rates by RECIST criteria, durability of responses and overall survival over 24 months
Kiniksa Pharmaceuticals Ltd., of Hamilton, Bermuda Vixarelimab Oncostatin M receptor subunit beta inhibitor Pruritus FDA granted breakthrough therapy designation for disease associated with prurigo nodularis
Moderna Inc., of Cambridge, Mass. mRNA-1273 mRNA vaccine COVID-19 EMA’s Committee for Medicinal Products for Human Use started a rolling review following confirmation of eligibility for submission on Oct. 14, 2020
Plx Pharma Inc., of Sparta, N.J. Vazalore 325 mg and Vazalore 81 mg Liquid-filled aspirin capsule Vascular disease FDA confirmed receipt of 2 CMC supplemental NDAs, setting estimated completion date for reviews for end of February 2021
Praxis Precision Medicines Inc., of Cambridge, Mass. PRAX-114 GABAA receptor PAM Major depressive disorder Received comments from FDA on full clinical hold of IND submission; agency proposed company conduct further toxicological investigation of the effect of PRAX-114 and its metabolites on fertility, reproduction and embryofetal development to support the planned trial; company believes results of ongoing standard fertility and reproductive studies, expected to be completed in first quarter of 2021, together with available toxicology package will satisfy the FDA request
Redhill Biopharma Ltd., of Tel Aviv, Israel RHB-107 (upamostat) Antiviral COVID-19 FDA cleared the IND for phase II/III study in patients with symptomatic COVID-19 who do not require hospitalization
UCB SA, of Brussels, Belgium Vimpat (lacosamide) Dihydropyrimidinase related protein 2 modulator; sodium channel modulator Primary generalized tonic-clonic seizures FDA approved Vimpat CV for use as adjunctive therapy in patients 4 and older and Vimpat injection for intravenous use in children 4 and older
Verrica Pharmaceuticals Inc., of West Chester, Penn. VP-102 (cantharidin, topical film-forming solution) Protein phosphatase 2A inhibitor Molluscum contagiosum  Received final meeting notes from FDA type A meeting to discuss resubmission of NDA; company reaffirmed expectations to resubmit NDA in first quarter of 2021
Viiv Healthcare Ltd., of London Cabotegravir  HIV integrase inhibitor HIV pre-exposure prophylaxis FDA granted breakthrough therapy designation for long-acting injectable cabotegravir

Notes

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