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Global New Drug R&D express:Lexicon, Govorestat (AT-007) , Milvexian, Dyne Therapeutics DYNE-101

1 Lexicon Announces FDA Approval of INPEFA™ (sotagliflozin) for Treatment of Heart Failure

Lexicon Pharmaceuticals announced that the U.S. Food and Drug Administration (FDA) has approved INPEFA™ (sotagliflozin), a once-daily oral tablet to reduce the risk of cardiovascular death, hospitalization for heart failure, and urgent heart failure visit in adults with heart failure or type 2 diabetes mellitus, chronic kidney disease, and other cardiovascular risk factors.

2 Apellis Reports Top-Line Results from Phase 2 MERIDIAN Study in ALS

Apellis Pharmaceuticals announced that the Phase 2 MERIDIAN study investigating systemic pegcetacoplan for the treatment of amyotrophic lateral sclerosis (ALS) did not meet its primary endpoint of the Combined Assessment of Function and Survival (CAFS) rank score at Week 52. The study also did not meet key secondary efficacy endpoints. Based on the lack of efficacy, Apellis and Sobi plan to discontinue development of systemic pegcetacoplan for ALS. In April, Apellis and Sobi discontinued treatment in the open-label portion of the study, following a recommendation from an independent data monitoring committee.

3 Govorestat (AT-007) Receives Orphan Medicinal Product Designation from the EMA for Treatment of SORD Deficiency

Applied Therapeutic announced that AT-007 (govorestat) has been granted orphan medicinal product designation by the European Medicines Agency (EMA) for treatment of Sorbitol Dehydrogenase (SORD) Deficiency. Additionally, the Company announced that new data has been published in the Journal of Clinical Investigation on govorestat treatment in models of SORD Deficiency. Govorestat is a central nervous system (CNS) penetrant Aldose Reductase inhibitor (ARI) in development for the treatment of several rare neurological diseases, including Galactosemia, SORD Deficiency, and PMM2-CDG.

4 Milvexian Granted U.S. FDA Fast Track Designation for All Three Indications Under Evaluation in Phase 3 Librexia Program: Ischemic Stroke, Acute Coronary Syndrome and Atrial Fibrillation

Bristol Myers Squibb in collaboration with Janssen Pharmaceuticals announced that all three prospective indications for milvexian, an investigational oral factor XIa (FXIa) inhibitor, have now been granted Fast Track Designation by the U.S. Food and Drug Administration (FDA). The designations cover all three indication-seeking studies within the Phase 3 Librexia development program (Librexia STROKE, Librexia ACS and Librexia AF), which are all dosing patients. The Librexia program is unrivaled as the most comprehensive FXIa clinical development program to date and will provide extensive data from nearly 50,000 patients.

5 Dyne Therapeutics Receives European Medicines Agency (EMA) Orphan Drug Designation for DYNE-101

Dyne Therapeutics announced that the European Medicines Agency (EMA) has granted orphan drug designation for DYNE-101. DYNE-101 is being evaluated in the Phase 1/2 ACHIEVE global clinical trial in adults with myotonic dystrophy type 1 (DM1). DYNE-101 consists of an antigen-binding fragment antibody (Fab) conjugated to an antisense oligonucleotide (ASO) and is designed to enable targeted muscle tissue delivery with the goal of reducing toxic DMPK RNA in the nucleus, releasing splicing proteins, allowing normal mRNA processing and translation of normal proteins, and potentially stopping or reversing the disease.

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