treatment News

Ingelheim am Rhein, Germany – Boehringer Ingelheim today announced that up to 83.0% of adults treated with survodutide (BI 456906) achieved a statistically significant improvement of metabolic dysfunction-associated steatohepatitis (MASH) versus placebo (18.2%) in a Phase II trial [response difference: 64.8% (CI 51.1% – 78.6%), p<0.0001]. The trial met its...
SOUTH SAN FRANCISCO, Calif. — Sutro Biopharma, Inc (NASDAQ: STRO), a clinical-stage oncology company pioneering site-specific and novel-format antibody drug conjugates (ADCs), today announced that the randomized portion (Part 2) of REFRαME-O1, the registration-directed trial of luveltamab tazevibulin (luvelta) in platinum-resistant ovarian cancer (PROC), is now open for enrollment, and...
PHILADELPHIA – SwanBio Therapeutics, a gene therapy company advancing AAV-based therapies for the treatment of devastating, genetically defined neurological conditions, today announced the initiation of the CYGNET study with the enrollment of the first two participants. CYGNET is a natural history study of adrenomyeloneuropathy (AMN), a form of adrenoleukodystrophy (ALD)...
UXBRIDGE, England — Norgine, a leading European specialty pharmaceutical company, is pleased to announce that Swissmedic has approved the registration of IFINWIL® (eflornithine) as monotherapy for the treatment of paediatric patients from one year with high-risk neuroblastoma (HRNB)1. “This milestone marks a further regulatory approval of IFINWIL® for high-risk neuroblastoma, following approvals in the US, Israel and Australia,” said Jörg Plessl,...
By Raffaele Pereno – PhD, MBA AMSTERDAM, the Netherlands – SynaptixBio, a UK-based biopharmaceutical company pioneering antisense oligonucleotide (ASO) therapies for ultra-rare neurological diseases, presented its groundbreaking work on TUBB4A-related leukodystrophy (also known as H-ABC) at the World Orphan Drug Congress (WODC) in Amsterdam. CheckOrphan spoke with SynaptixBio’s CEO, Dan...
WALTHAM, Mass. — Syndax Pharmaceuticals Inc. (Nasdaq: SNDX), a clinical stage biopharmaceutical company developing an innovative pipeline of cancer therapies, today announced completion of enrollment in the AUGMENT-101 pivotal trial cohort of patients with relapsed/refractory (R/R) mutant nucleophosmin (mNPM1) acute myeloid leukemia (AML). Topline data is expected in the fourth...
CAMBRIDGE, Mass. – Synlogic, Inc. (Nasdaq: SYBX), a clinical stage company bringing the transformative potential of synthetic biology to medicine, today announced that it has initiated the Phase 1 clinical trial of SYNB1934, an investigational Synthetic Biotic™ medicine for the treatment of Phenylketonuria (PKU). “We are delighted to have so quickly advanced...
CAMBRIDGE, Mass. – Synlogic, Inc. (Nasdaq: SYBX), a clinical stage company bringing the transformative potential of synthetic biology to medicine, today announced positive data from clinical studies evaluating both SYNB1618 and SYNB1934, investigational Synthetic Biotic™ medicines for the treatment of phenylketonuria (PKU). SYNB1618 demonstrated clinically meaningful reductions of phenylalanine (Phe) at several dose levels, across multiple time points, in...
CAMBRIDGE, Mass. — Synlogic, Inc. (Nasdaq: SYBX), a biopharmaceutical company advancing novel therapeutics to transform the care of serious diseases, announced today the publication of a manuscript detailing the development and activity of SYNB1353 in preclinical models and demonstrating safety, tolerability, and clinical proof of mechanism in healthy volunteers through...