Company Product Description Indication Status
Agios Pharmaceuticals Inc., of Cambridge, Mass. Tibsovo (ivosidenib)  IDH1 gene inhibitor Myelodysplastic syndrome FDA granted breakthrough status for treatment of adults with relapsed or refractory MDS with a susceptible IDH1 mutation; already FDA-approved for AML
Dicerna Pharmaceuticals Inc., of Lexington, Mass. DCR-A1AT SERPINA1 gene inhibitor Alpha-1 antitrypsin deficiency EC granted orphan status for treatment of congenital A1AT
Eiger Biopharmaceuticals Inc., of Palo Alto, Calif. Lonafarnib Ras GTPase inhibitor; protein farnesyltransferase inhibitor Hutchinson Gilford progeria syndrome Initiated rolling NDA filing for treatment of HGPS and progeroid laminopathies
Immunogen Inc., of Waltham, Mass. Mirvetuximab soravtansine Folate receptor alpha antagonist; tubulin receptor antagonist Ovary tumor FDA said a new single-arm study in platinum-resistant ovarian cancer could support accelerated approval, so the company is starting pivotal monotherapy trial in women with FRα-high platinum-resistant ovarian cancer who've been previously treated with Avastin (bevacizumab)
Onconova Therapeutics Inc., of Newtown, Pa. and Pint Pharma GmbH, of Vienna Rigosertib Non-ATP-binding inhibitor of cyclin-dependent kinase Myelodysplastic syndrome Brazilian health authority approved start of phase III Inspire trial in Brazil with I.V. rigosertib in higher-risk MDS
Pfizer Inc., of New York, and Astellas Pharma Inc., of Tokyo Xtandi (enzalutamide) Androgen receptor inhibitor Prostate tumor FDA approved sNDA, extending label to include men with non-metastatic castration-resistant prostate cancer
Pharmamar SA, of Madrid Lurbinectedin TAT protein inhibitor; RNA polymerase II inhibitor Small-cell lung cancer Submitted NDA for lurbinectedin in treatment of people with relapsed SCLC; seeking accelerated approval
Prevail Therapeutics Inc., of New York PR-006 GRN gene stimulator Frontotemporal dementia FDA granted orphan status for FDT with a GRN mutation
Transthera Biosciences Co. Ltd., of Nanjing, China TT-00920 PDE9 inhibitor Congestive heart failure FDA approved IND; company will start phase I trial in early 2020

Notes

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

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