Response process – Talk Bio

FDA Rejection Derails PTC Therapeutics’ Rare Disease Hopeful

3 days ago talkbio0Tagged Clinical Trials, concerns, Effectiveness, factor IX, Failure (biologic function), Friedreich 's ataxia, Rare Diseases, Rejection, Response process, United States Food and Drug Administration, vatiquinone

PTC Therapeutics; FDA rejection; vatiquinone; Friedreich’s ataxia; rare disease; efficacy concerns; complete response letter; clinical trial failure

Response Pharma: RDX-002 Limits Weight Regain After GLP-1 Therapy – Positive Phase II Results Announced

1 week ago talkbio0Tagged cardiometabolic health, Discontinuation (procedure), Glucagon-Like Peptide 1, iMTP, Inhibitor, Obesity, Phase 2 Clinical Trials, postprandial, RDX-002, Response process, Triglycerides, weight regain

Response Pharmaceuticals; RDX-002; GLP-1 discontinuation; weight regain; obesity; Phase II clinical trial; postprandial triglycerides; cardiometabolic health; iMTP inhibitor

Spine BioPharma’s Phase 3 Back Pain Drug Fails to Meet Primary Endpoint, Attributes Miss to High Sham Response

3 weeks ago talkbio0Tagged Chronic low back pain, Clinical Trials, Intervertebral Disc Degeneration, primary endpoint, Response process, salicylhydroxamic acid, SB-01, Spine BioPharma

Spine BioPharma; Phase 3 trial; chronic low back pain; degenerative disc disease; SB-01; primary endpoint; sham response; clinical trial results

FDA Rejects Capricor’s Cell Therapy for Duchenne Muscular Dystrophy

1 month ago talkbio0Tagged Capricor Therapeutics, Cardiomyopathies, Cell Therapy, Clinical Data, Complete, deramiocel, HOPE-3 trial, Muscular Dystrophy, Duchenne, Phase 3, Rejection, Response process, United States Food and Drug Administration

FDA rejection; Capricor Therapeutics; deramiocel; Duchenne muscular dystrophy; DMD; cell therapy; cardiomyopathy; clinical data; Phase 3 HOPE-3 trial; Complete Response Letter

Analysts Downplay Intellia Adverse Event, but Gene Therapy Safety Remains Under Scrutiny

3 months ago talkbio0Tagged Adverse event, ATTR-CM, Event, Gene Modification, gene therapy, Intellia, liver transaminase, MAGNITUDE trial, Response process, Safety, Stock Decline

Intellia Therapeutics; gene therapy; safety event; adverse event; MAGNITUDE trial; liver transaminase; stock decline; ATTR-CM; gene editing; analyst response

FDA Rejects Milestone Pharmaceuticals’ Etripamil Nasal Spray for PSVT

5 months ago talkbio0Tagged Cardamyst, Complete, etripamil, Milestone, Paroxysmal supraventricular tachycardia, Rejection, Response process, United States Food and Drug Administration

Etripamil, Cardamyst, PSVT, FDA rejection, Complete Response Letter, manufacturing issues, Milestone Pharmaceuticals

Gilead’s Seladelpar Receives Conditional EU Approval for Primary Biliary Cholangitis Treatment

6 months ago talkbio0Tagged Biliary Cholangitis, Commission, Conditional, European (ethnic group), Gilead Sciences, Primary Biliary Cholangitis, Response process, seladelpar, ursodiol

Seladelpar, Gilead Sciences, primary biliary cholangitis, PBC, European Commission, conditional marketing authorization, RESPONSE trial, ursodeoxycholic acid (UDCA)