Stablix Therapeutics Inc. has raised $63 million in series A funding to pioneer a novel class of small-molecule drugs, designed to selectively stabilize proteins that would otherwise undergo degradation in the ubiquitin-proteasome system (UPS). It represents what its founding investors consider the first company focused on inhibiting the UPS in a target-specific fashion.
DUBLIN – Ronopterin (VAS-203) failed to meet the primary endpoint of a phase III trial in traumatic brain injury (TBI), but developer Vasopharm GmbH is nevertheless convinced that it has an active drug on its hands. It has also received backing from the study’s data monitoring committee, which has provided, Vasopharm said, a “positive benefit-risk assessment” on the basis of the efficacy signals detected and the drug’s known safety profile.
DUBLIN – Shares in Santhera Pharmaceuticals Holding AG surged by as much as 71% June 1 on news that the high-dose arm of a phase IIb pivotal trial of vamorolone hit the primary endpoint of an improvement vs. placebo in the time-to-stand velocity attained by ambulatory boys with Duchenne muscular dystrophy (DMD).
Pieris Pharmaceuticals Inc. is banking $20 million up front and could receive up to $1.4 billion more in preclinical, clinical and commercial milestones from a multi-program collaboration and license agreement in respiratory disease and ophthalmology with Genentech.
DUBLIN – Abivax SA has started planning for a pivotal phase III program for its inflammatory bowel disease drug candidate, ABX-464, following a readout of top-line data from a phase IIb induction study in ulcerative colitis, in which the molecule attained the primary endpoint at all three dose levels tested. It demonstrated efficacy on a range of secondary endpoints, too, while its safety profile also appeared to be favorable.
DUBLIN – Skysona (elivaldogene autotemcel, Lenti-D), Bluebird Bio Inc.’s gene therapy for cerebral adrenoleukodystrophy, received a nod from the EMA’s Committee for Human Medicinal Products (CHMP) during its May meeting this week, paving the way for a formal European authorization in the coming weeks. It will constitute the first approval for the product. An FDA approval is some way behind – the company will not complete its BLA filing with the FDA until around midyear.
Where Ilaris (canakinumab) failed, can Kineret (anakinra) succeed? Swedish Orphan Biovitrum AB is reporting that an investigator-initiated phase III study of its interleukin-1 (IL-1) blocker, Kineret, in 594 hospitalized COVID-19 patients with moderate or severe pneumonia, who had a poor prognosis, uncovered what appear to be dramatic benefits.
DUBLIN – Numab Therapeutics AG has moved closer to the front rank of European antibody developers by raising CHF100 million (US$111 million) in a series C financing round to accelerate the development of NM21-1480, a trispecific antibody that acts both as a PD-L1 inhibitor and 4-1BB (CD137) agonist. It also contains a human serum albumin recognition group, to extend its circulatory half-life.
DUBLIN – Bergenbio ASA joins an expanding list of companies whose candidate drug has failed to attain the primary endpoint of a clinical trial in COVID-19.
Flare Therapeutics Inc. emerged from stealth mode with $82 million in series A financing and an ambitious agenda to systematically drug transcription factors associated with cancer and other neurological, genetic and immunological conditions.
