Author name: Nasir

BridgeBio Pharma, Inc., a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, today announced updated data from its ongoing Phase 2 open-label extension (OLE) study of acoramidis (AG10) in patients with symptomatic transthyretin (TTR) amyloid cardiomyopathy (ATTR-CM). The results were featured in an oral presentation at the American College of Cardiology (ACC) Annual Scientific …

BridgeBio Pharma Presents Updated Results From Phase 2 Open-Label Extension Study of Acoramidis in Transthyretin Amyloid Cardiomyopathy (ATTR-CM) Read More »

Attralus, Inc., a clinical stage biopharmaceutical company developing transformative medicines to improve the lives of patients with systemic amyloidosis, today announced encouraging new clinical data from the University of Tennessee Graduate School of Medicine from its Phase 1/2 trial of iodine evuzamitide (124I-AT-01), the company’s pan-amyloid binding peptide in development as a radiotracer for the …

Positive New Data from Phase 1/2 Trial of AT-01 Presented at 2022 American College of Cardiology Annual Scientific Session Read More »

Alnylam Pharmaceuticals, Inc., the leading RNAi therapeutics company, today announced new positive results from an 18-month analysis of exploratory cardiac endpoints in the HELIOS-A Phase 3 study of vutrisiran, an investigational RNAi therapeutic in development for the treatment of transthyretin-mediated (ATTR) amyloidosis. The 18-month findings show that in a pre-defined cardiac subpopulation, treatment with vutrisiran …

Alnylam Presents New 18-Month Results From Exploratory Cardiac Endpoints in HELIOS-A Phase 3 Study of Investigational Vutrisiran Read More »

Alnylam Pharmaceuticals, Inc., the leading RNAi therapeutics company, announced that the U.S. Food and Drug Administration (FDA) approved AMVUTTRA™ (vutrisiran), an RNAi therapeutic administered via subcutaneous injection once every three months (quarterly) for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults. hATTR amyloidosis is a rare, inherited, rapidly progressive, and fatal …

Alnylam Announces FDA Approval of AMVUTTRA™ (Vutrisiran), an RNAi Therapeutic for the Treatment of the Polyneuropathy of Hereditary Transthyretin-Mediated Amyloidosis in Adults Read More »

Attralus, Inc., a clinical stage biopharmaceutical company developing transformative medicines to improve the lives of patients with systemic amyloidosis, announced encouraging new clinical data from the University of Tennessee Graduate School of Medicine from its Phase 1/2 trial of AT-01 (Iodine I-124 evuzamitide), the company’s pan-amyloid binding peptide in development as a radiotracer for the …

Positive New Data From Phase 1/2 Trial of AT-01 (Iodine I-124 Evuzamitide) Presented at 2022 Society of Nuclear Medicine & Molecular Imaging Annual Meeting Read More »

Positive high-level results from the NEURO-TTRansform Phase III trial in patients with hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN) showed AstraZeneca and Ionis’ eplontersen met its co-primary endpoints in a planned interim analysis at 35 weeks. In the trial, eplontersen reached a statistically significant and clinically meaningful change from baseline for its co-primary endpoint of percent change …

Eplontersen Met Co-Primary and Secondary Endpoints in Interim Analysis of the NEURO-TTRansform Phase III Trial for Hereditary Transthyretin-Mediated Amyloid Polyneuropathy (ATTRv-PN) Read More »

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced that the APOLLO-B Phase 3 study of patisiran, an investigational RNAi therapeutic in development for the treatment of transthyretin-mediated (ATTR) amyloidosis with cardiomyopathy, met the primary endpoint of change from baseline in the 6-Minute Walk Test (6-MWT) at 12 months compared to placebo …

Alnylam Reports Positive Topline Results From APOLLO-B Phase 3 Study of Patisiran in Patients With ATTR Amyloidosis With Cardiomyopathy Read More »

Prothena Presents Data Demonstrating Consistent Survival Benefit Observed with Birtamimab in Mayo Stage IV AL Amyloidosis Patients in Phase 3 VITAL Study at ASH 2022

Nexcella, Inc., a biopharmaceutical company engaged in the discovery and development of novel cell therapies for oncology and other indications, today announced that NXC-201 treatment continues to demonstrate 100% complete responses in a total of 6 relapsed/refractory AL amyloidosis patients. Clinical data published December 2022 in Clinical Cancer Research (https://doi.org/10.1158/1078-0432.CCR-22-0637) demonstrated 100% complete response rate; …

Nexcella, Inc. Announces Additional Positive NXC-201 Clinical Data Demonstrating 100% Complete Responses in Relapsed/Refractory AL Amyloidosis Patients, Duration of Response Not Yet Reached Read More »

CARLSBAD, Calif., April 24, 2023 /PRNewswire/ — Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that the Phase 3 NEURO-TTRansform study for AstraZeneca and Ionis’ eplontersen in patients with hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN) met all co-primary endpoints and secondary endpoints at 66 weeks versus an external placebo group. The positive results are being presented today …

Eplontersen halted ATTRv-PN disease progression and improved neuropathy impairment and quality of life in Phase 3 study through 66 weeks Read More »