Japanese firm in discussions with regulator as high-need novel therapy for spinocerebellar degeneration misses endpoint but shows promise in pooled analysis.

With top-line data showing efficacy in type 1 SMA patients, risdiplam may be able to obtain a label that would address significantly more patients than Novartis’ Zolgensma. Final efficacy data will show how drug’s efficacy compares to Biogen’s Spinraza.

HanAll and Daewoong remain upbeat on dry eye candidate HL036 and are gearing up for a second Phase III US study despite the first missing its primary endpoint in just-released top-line results.

Chi-Med’s surufatinib has come up trumps in a second trial in neuroendocrine tumors, after the monitoring committee recommended an early halt following achievement of its PFS target in the pancreatic setting.

Stock market reaction to the first day’s news from the 38th J.P. Morgan Healthcare conference in San Francisco was negative, probably because expectations for a continuation of December’s bumper M&A bonanza were too high.

Among the Korean firms presenting at J.P. Morgan, Hanmi laid out its strategy for obesity and other core pipeline assets, Celltrion focused on its expanding biosimilar pipeline and China plans and LG Chem unveiled new R&D progress.

Accelerated approval in epithelial sarcoma may de-risk the firm’s goals for approval of Tazverik in follicular lymphoma and other larger indications.

The European Commission has backed J&J's blockbuster to treat children with psoriasis but the drug has fallen foul of NICE for ulcerative colitis.

Once a pariah because of its price gouging, the new approval represents a change of tack from Horizon.

Mayzent is the first oral treatment specifically indicated for patients with secondary-progressive multiple sclerosis and while it is expected to be a blockbuster, the company tells Scrip that educational programs for physicians are a priority.

Pipeline Watch is a weekly snapshot of selected late-stage clinical trial events and approvals announced by pharmaceutical and biotech companies at medical and industry conferences, in financial and company presentations, and in company releases and statements.