Roche’s risdiplam achieves primary endpoint in infant-related spinal muscular atrophy trial
Roche announced that its risdiplam has achieved primary endpoint in the FIREFISH study in infants with type 1 spinal muscular atrophy.
Roche announced that its risdiplam has achieved primary endpoint in the FIREFISH study in infants with type 1 spinal muscular atrophy.
Verrica Pharmaceuticals announced the presentation of positive data from a post-hoc pooled analysis of the pivotal Phase 3 CAMP trials evaluating the safety and efficacy of VP-102 (cantharidin 0.7% topical solution), Verrica’s lead product candidate for the treatment of molluscum contagiosum (molluscum).
Innovation Pharmaceuticals, a clinical stage pharmaceutical company, announced that the company has received agreement from the study’s Dose Escalation Committee (DEC) to progress dosing to the second cohort in its Phase 1 trial to investigate the use of delayed release tablets for colonic delivery of Brilacidin in healthy volunteers.
Neuroptika, a privately held biotechnology company focused on the development of novel regenerative treatments for ophthalmic diseases, announced that the first patient was enrolled in a Phase 2 clinical trial of NRO-1 for the treatment of dry eye disease.
VBI Vaccines has reported top-line data from CONSTANT, the second pivotal Phase 3 study, assessing lot-to-lot manufacturing consistency of Sci-B-Vac, the company’s trivalent hepatitis B (HBV) vaccine, and comparing safety and immunogenicity of Sci-B-Vac to GlaxoSmithKline’s currently-marketed HBV vaccine, Engerix-B.
Catabasis Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company, and Duchenne UK, a charity that seeks to fund and accelerate treatments and a cure for Duchenne muscular dystrophy (DMD), have entered into a partnership for a Phase 2 trial of edasalonexent, a novel NF-kB inhibitor, in non-ambulatory DMD patients.
Veristat, a full-service CRO, announced that it has completed the acquisition of The Clinical Trial Company, a global Contract Research Organization headquartered in Europe.
Exelixis announced a collaboration agreement with Roche to evaluate cabozantinib (CABOMETYX), Exelixis’ small molecule inhibitor of receptor tyrosine kinases, in combination with atezolizumab (TECENTRIQ), Roche’s PD-L1 immune checkpoint inhibitor, in patients with locally advanced or metastatic solid tumours.
Arcus Biosciences announced a clinical collaboration with Genentech, a member of the Roche group, for the evaluation of novel combinations with AB928, Arcus’s dual antagonist of adenosine receptors A2aR and A2bR, for colorectal (CRC) and pancreatic (PDAC) cancers.
Amylyx Pharmaceuticals, Inc., a pharmaceutical company focused on developing new treatments for amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases, and the Sean M. Healey & AMG Center for ALS at Mass General today announced that AMX0035 demonstrated a significant treatment benefit for people with ALS in the CENTAUR study.