Phase 2 trial
Scholar Rock’s SMA drug helps Zepbound patients retain muscle in phase 2 trial
Scholar Rock; apitegromab; SMA drug; Zepbound; tirzepatide; muscle preservation; obesity; weight loss; phase 2 trial; lean mass; myostatin inhibitor
FDA Rejects Stealth BioTherapeutics’ Rare Disease Drug but Leaves Door Open for Accelerated Approval
FDA rejection; Stealth BioTherapeutics; elamipretide; Barth syndrome; rare disease; accelerated approval; muscle strength endpoint; phase 2 trial; regulatory delay
Tourmaline Bio’s Heart Drug Pacibekitug Shows Promise Against Cardiovascular Inflammation, Targets Competition with Novo, CSL
Tourmaline Bio; heart drug; pacibekitug; cardiovascular inflammation; inflammation markers; Novo Nordisk; CSL; Phase 2 trial; FDA; IL-6
Beacon’s rare eye disease gene therapy signals vision function improvements in early phase 2 data
XLRP, gene therapy, laru-zova, visual function, rare eye disease, Phase 2 trial, DAWN trial, vision improvement, low light vision
BioCity’s SC0062 Shows Promise in Phase 2 Trial for Diabetic Kidney Disease
SC0062, endothelin receptor type A (ETA) antagonist, diabetic kidney disease (DKD), proteinuria reduction, phase 2 trial, BioCity Biopharma
AlgoTx’s ATX01 Shows Promise Despite Missing Primary Endpoint in Phase 2 CIPN Trial
AlgoTx, ATX01, chemotherapy-induced peripheral neuropathy (CIPN), Phase 2 trial, placebo effect, non-opioid pain relief, topical amitriptyline
Kura Oncology and Kyowa Kirin Report Positive Phase 2 Results for Ziftomenib in AML, Prepare for FDA Submission
Ziftomenib, AML, NPM1-mutant, Phase 2 trial, KOMET-001, FDA submission, Kura Oncology, Kyowa Kirin
LB-102 Shows Promise as Potential First-in-Class Benzamide Antipsychotic for Schizophrenia
LB-102, schizophrenia, Phase 2 trial, benzamide antipsychotic, efficacy, safety
LB-102 Shows Promise as Potential First-in-Class Benzamide Antipsychotic for Schizophrenia in US
LB-102, schizophrenia, Phase 2 trial, benzamide antipsychotic, efficacy, safety
LB-102: Promising Phase 2 Results Revive Benzamide Antipsychotics for Schizophrenia Treatment in the US
LB-102, schizophrenia, benzamide antipsychotics, Phase 2 trial, PANSS scores, safety profile, once-daily dosing