Intra-Cellular Therapies submits sNDA to FDA for CAPLYTA for major depressive disorder
- Intra-Cellular Therapies has submitted a supplemental New Drug Application (sNDA) to the FDA for CAPLYTA as an adjunctive therapy for Major Depressive Disorder (MDD).
- The submission is based on positive results from Phase 3 Studies 501 and 502, which showed significant efficacy in reducing depression symptoms.
- CAPLYTA demonstrated a favorable safety profile, with common adverse events including dizziness, dry mouth, and fatigue.
- If approved, CAPLYTA would be indicated for three major psychiatric conditions, potentially benefiting over 30 million adults in the US.
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Arrakis Therapeutics unveils data for RNA-targeted small molecule drug program for myotonic dystrophy
- Arrakis Therapeutics presented preclinical data for its RNA-targeted small molecule (rSM) drug program aimed at treating myotonic dystrophy type 1 (DM1).
- The data demonstrated the reversal of myotonia by binding to the pathogenic CUG repeat RNA element, disrupting nuclear aggregates, and correcting splicing defects.
- Arrakis's proprietary methods enabled the design of small molecules targeting the trinucleotide repeat expansion in the mRNA of DMPK, which drives DM1 pathology.
- The methodologies used in the DM1 program are being applied to advance Arrakis's pipeline of rSM drug candidates for other diseases.
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