Company Product Description Indication Status
Fulcrum Therapeutics Inc., of Cambridge, Mass. Losmapimod Oral p38α/β MAPK inhibitor COVID-19 Received early notification from FDA that it may proceed with initiating phase III trial in higher-risk hospitalized adults with COVID-19; Losvid trial will enroll about 400 patients; primary endpoint is proportion who progress to death or respiratory failure by day 28; an interim analysis will be conducted in the fourth quarter of 2020 for futility, and top-line data are expected in the first quarter of 2021
Global Blood Therapeutics Inc., of South San Francisco Oxbryta (voxelotor) Inhibits hemoglobin polymerization Hemolytic anemia in sickle cell disease Based on discussion with EMA, plans to submit MAA by mid-2021, seeking approval for use in patients 12 and older; company also intends to initiate early access program in Europe prior to potential marketing authorization
Hemoshear Therapeutics Inc., of Charlottesville, Va. HST-5040 Oral, small molecule Methylmalonic acidemia and propionic acidemia FDA cleared IND for phase II study
Imara Inc., of Boston IMR-687 Small-molecule inhibitor of PDE9 Beta-thalassemia FDA granted orphan designation
Innate Pharma SA, of Paris Lacutamab (IPH-4102) Anti-KIR3DL2 humanized cytotoxicity-inducing antibody Advanced T-cell lymphomas FDA lifted partial clinical hold placed on phase II Tellomak trial, based on quality assessment of new GMP-certified batch manufactured for clinical development program; recruitment can now resume for new patients with relapsed/refractory Sézary syndrome and mycosis fungoides who have received at least 2 prior systemic therapies  
Mayne Pharma Group Ltd., of Adelaide, Australia E4/DRSP  Combined oral pill containing 15 mg estetrol and 3 mg drospirenone Contraception NDA accepted for review by the FDA; decision expected in first half of 2021
Medigene AG, of Martinsried, Germany MDG-1021 T-cell receptor-modified T-cell immunotherapy targeting antigen HA-1 Relapsed or persistent blood cancers  Received approval from Dutch regulators to begin phase I study enrolling patients after allogeneic hematopoietic stem cell transplantation
Neurorx Inc., of Radnor, Pa., and Relief Therapeutics Holding AG, of Geneva RLF-100 Synthetic form of human vasoactive intestinal peptide COVID-19 FDA granted fast track designation for treatment of acute lung injury/acute respiratory distress syndrome associated with COVID-19
Novartis AG, of Basel, Switzerland Entresto (sacubitril/valsartan) Inhibits neprilysin and blocks angiotensin II type-I receptor Heart failure with preserved ejection fraction  FDA accepted supplemental NDA; decision expected in first half of 2021
Philogen SpA, of Siena, Italy Nidlegy (bifikafusp alfa and onfekafusp alfa) Combination of immunocytokines Melanoma and nonmelananoma skin cancers EMA agreed to request for marketing the product as a combination pack for use as neoadjuvant intralesional treatment for resectable stage III melanoma and locally advanced, not metastatic nonmelanoma skin cancers


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

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