|Alpine Immune Sciences Inc., of Seattle||AlPN-101||Dual T-cell co-stimulation inhibitor||Acute graft-vs.-host disease||FDA granted 2 orphan designations for prevention and treatment of acute GVHD|
|Biohaven Pharmaceutical Holding Co. Ltd., of New Haven, Conn.||Verdiperstat||Oral, brain-penetrant irreversible myeloperoxidase inhibitor||Multiple system atrophy||FDA granted fast track designation|
|Pharnext SA, of Paris||PXT-3003||Oral fixed-dose combination of baclofen, naltrexone and sorbitol||Charcot-Marie-Tooth disease type 1A||U.K.’s Medicine and Healthcare products Regulatory Agency granted promising innovative medicine designation for use in patients 16 and older|
|Rhythm Pharmaceuticals Inc., of Boston||Setmelanotide||Selective agonist of MC₄ receptor||Alstrom syndrome||FDA granted orphan designation|
|Sage Therapeutics Inc., of Cambridge, Mass.||Zuranolone||Positive allosteric modulator of GABAA receptor||Postpartum depression, major depression and major depressive syndrome||Following an FDA breakthrough therapy guidance meeting, identified 3 pathways supporting possible filing for approval to treat postpartum depression, acute treatment of major depressive disorder when co-initiated with a new antidepressant and as an episodic therapy in major depressive syndrome patients; 3 short-term clinical studies set to start in 2020|
|Synairgen plc, of Southampton, U.K.||SNG-001||Inhaled formulation of interferon-beta-1a||COVID-19||Received expedited approvals from U.K.’s Medicines and Healthcare products Regulatory Agency and Health Research Authority to conduct a clinical trial|
For more information about individual companies and/or products, see Cortellis.