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. |
