New top-line data from Scholar Rock Holding Group’s phase II Topaz trial of apitegromab (SRK-015) in patients with type 2 and type 3 spinal muscular atrophy generated enough proof-of-concept results for the company to plan on initiating a phase III for later this year. But the 12-month data didn’t stop the Cambridge, Mass.-based company’s stock (NASDAQ:SRRK) from struggling mightily on April 6 as shares closed 20.3% lower at $35.97 each.

Nevro Corp.'s spinal cord stimulation system cut diabetic neuropathy pain in half or more in 85% of patients in a study published on April 5 in JAMA Neurology. Patients receiving the stimulation treatment delivered by the Senza system experienced an average reduction in pain of 76% at six months in the largest clinical largest trial to date to evaluate the use of spinal cord stimulation (SCS) in painful diabetic neuropathy (PDN).

Full rights to TAK-169, a CD38-targeted engineered toxin body that Takeda Pharmaceuticals Co. and Molecular Templates Inc. have been developing, are going to Molecular Templates as is the therapy’s clinical development.

Acadia Pharmaceuticals Inc. President Serge Stankovic said the company was “kept completely in [the] dark” by the FDA regarding specifics about what the agency found wrong a month ago with the sNDA for Nuplazid (pimavanserin) – until, that is, the agency’s complete response letter (CRL) landed on the firm’s desk.

BERLIN – Lindis Blood Care GmbH launched a multicenter clinical EU certification study for Catuvab, which is used to eliminate tumor cells from surgical blood. The experiment, dubbed Remove, aims to confirm that Catuvab can reliably remove epithelial cell adhesion molecules (EPCAM)-positive tumor cells from patient blood during cancer surgery.

At the end of March, JAMA Ophthalmology’s publication of first results from the NIH-sponsored, 328-patient trial with Regeneron Pharmaceuticals Inc.’s VEGF inhibitor, Eylea (aflibercept), in non-proliferative diabetic retinopathy bolstered investor hopes for wider use. But competitors loom for the compound, first approved in November 2011 for wet age-related macular degeneration.

Following the company’s COVID-19 manufacturing misstep, Emergent Biosolutions Inc. continued its struggle to rebalance as top-line data from a phase III study it participated in failed to hit its primary endpoints with statistically significant results. The data showed that adding anti-SARS-CoV-2 hyperimmunoglobulin to standard of care, inclusive of remdesivir, for hospitalized adult COVID-19 patients with symptoms for less than 12 days failed to provide clinical benefit when compared to standard of care plus placebo. The global, multicenter double-blind, placebo-controlled randomized study assessed the safety and efficacy of four immunoglobulin candidates, the SARS-CoV-2 immune globulin intravenous (human) (COVID-HIG) plus standard of care.

LONDON – A new route to the approval of drugs promises to smooth the path from clinical trials to approval, reimbursement and commissioning in the National Health Service, through closer and earlier collaboration between the U.K. Medicines and Healthcare products Regulatory Agency (MHRA) and health technology assessment bodies.