Infrequent – Talk Bio

Amylyx Discontinues Rare Disease Program After Relyvrio Fails to Show Benefit Over Placebo

6 days ago talkbio0Tagged AMX0035, Amylyx, Amyotrophic Lateral Sclerosis, Clinical trial failure, Discontinuation (procedure), disease program, Infrequent, Placebos, Progressive, Relyvrio, Supranuclear paralysis

Amylyx; Relyvrio; AMX0035; progressive supranuclear palsy; ALS; clinical trial failure; rare disease program; placebo; discontinuation

Regeneron Reports Phase 3 Success for Cemdisiran in Rare Disease Generalized Myasthenia Gravis

1 week ago talkbio0Tagged Alnylam, cemdisiran, Infrequent, Myasthenia Gravis, Generalized, Regeneron, regulatory submission, RNA drug, RNA, Small Interfering, United States

Regeneron; Phase 3 trial; cemdisiran; generalized myasthenia gravis; rare disease; RNA drug; siRNA; Alnylam Pharmaceuticals; U.S. regulatory submission

Stealth BioTherapeutics Resubmits Third FDA Application for Rare Disease Drug

2 weeks ago talkbio0Tagged 3-Methylglutaconic aciduria type 2, access program, Drug Approval, Elamipretide, Infrequent, resubmission, Stealth BioTherapeutics, United States Food and Drug Administration

Stealth BioTherapeutics; FDA resubmission; rare disease; Barth syndrome; elamipretide; drug approval; expanded access program

FDA Extends PDUFA Date for Regenxbio’s Hunter Syndrome Gene Therapy RGX-121

2 weeks ago talkbio0Tagged clemidsogene lanparvovec, Deferred, gene therapy, IDS gene, Infrequent, Mucopolysaccharidosis II, PDUFA, Regenxbio, RGX-121, United States Food and Drug Administration

FDA delay; PDUFA extension; Regenxbio; Hunter syndrome; clemidsogene lanparvovec; RGX-121; gene therapy; Mucopolysaccharidosis II; IDS gene; rare disease

FDA Grants Full Approval to Precigen’s Papzimeos for Rare Disease RRP

3 weeks ago talkbio0Tagged Approved, Episodic, gene therapy, Immunotherapy, Infrequent, orphan drug designation, Papzimeos, Precigen, Recurrent respiratory papillomatosis, RRP, United States Food and Drug Administration

Precigen; Papzimeos; FDA approval; recurrent respiratory papillomatosis (RRP); gene therapy; immunotherapy; rare disease; Orphan Drug designation

Novartis antibody ianalumab clinches another Phase 3 win in rare blood disease (ITP)

3 weeks ago talkbio0Tagged Autoimmune, Autoimmune thrombocytopenia, B-cell depletion, ianalumab, Infrequent, Inosine Triphosphate, Novartis, Phase 3, platelet levels, Psychological inhibition, regulatory submissions, Sjögren 's disease, TNFRSF13C protein, human, treatment failure, VAY736

Novartis; ianalumab; VAY736; primary immune thrombocytopenia; ITP; rare disease; Phase 3; time to treatment failure; safe platelet levels; eltrombopag; autoimmune; B-cell depletion; BAFF-R inhibition; Sjögren’s disease; regulatory submissions

Y-mAbs Therapeutics Acquired by SERB Pharmaceuticals in $412 Million Buyout Following Strategic Review

4 weeks ago talkbio0Tagged all-cash tender, Danyelza, Infrequent, Merger, Neoplasms, pharmaceutical acquisition, Serbs, strategic review, Y-mAbs

Y-mAbs Therapeutics; SERB Pharmaceuticals; buyout; Danyelza; rare oncology; merger; pharmaceutical acquisition; strategic review; all-cash tender offer

AstraZeneca Amyloidosis Drug Fails Phase 3 Trials but Shows Potential in Subgroup

2 months ago talkbio0Tagged Alexion, Amyloidosis, Anselamimab, AstraZeneca, Clinical trial failure, Drug Development, Infrequent, prespecified, Primary amyloidosis, Subgroup A Nepoviruses

AstraZeneca; amyloidosis; anselamimab; Phase 3 trial; clinical trial failure; prespecified subgroup; rare diseases; Alexion; AL amyloidosis; drug development

AstraZeneca’s Alexion Signs $825M AAV Capsid Deal with JCR Pharma

2 months ago talkbio0Tagged AAVS1 gene, Alexion, AstraZeneca, Capsid, gene therapy, genomic medicines, Infrequent, JCR, JUST-AAV, Licensing Deal

AstraZeneca; Alexion; JCR Pharmaceuticals; JUST-AAV; gene therapy; AAV capsid; rare disease; licensing deal; genomic medicines

Regeneron Leads $66M Series B for Actio Biosciences’ Rare Disease Therapeutics

3 months ago talkbio0Tagged ABS-0871, ABS-1230, Actio, Biosciences, Charcot-Marie-Tooth Disease, Clinical Trials, Drugs, Orphan, Epilepsy, Infrequent, Inhibitor, KCNT1 gene, Regeneron, Series B financing, TRPV4 protein, human

Regeneron; Actio Biosciences; rare diseases; Series B financing; KCNT1 inhibitor; TRPV4 inhibitor; ABS-1230; ABS-0871; Charcot-Marie-Tooth disease; epilepsy; orphan drug; clinical trials