The coming months represent a crucial phase in the development of Selection Inc., a U.S.-German firm that is tackling a target in autoimmune disease that has evaded the best efforts of many other drug developers.
Lonza Group Ltd. has acquired Synaffix BV, the developer of a widely partnered set of tools for generating antibody-drug conjugates (ADCs), for €100 million (US$108 million) up front plus up to €60 million more in milestones.
Coave Therapeutics aims to move its lead gene therapy program, CTx-PDE6b, for a form of retinitis pigmentosa (RP) into a pivotal trial in 2025 on the back of a phase I/II study, which uncovered a preliminary efficacy signal in one patient subgroup.
Lack of efficacy brought the development of two investigational agents for amyotrophic lateral sclerosis (ASL) to a halt over the past week. On May 23, Wave Life Sciences Inc. disclosed that its stereopure antisense oligonucleotide WVE-004 failed to demonstrate clinical benefit after 24 weeks of treatment on a phase Ib/IIa trial in familial ALS patients or frontotemporal dementia patients. And on May 25, Apellis Pharmaceuticals Inc. and its partner, Swedish Orphan Biovitrum International AB, said that pegcetacoplan failed to meet its primary endpoint of a one-year phase II trial in patients with sporadic disease.
Boundless Bio Inc. raised $100 million in a series C round to continue its highly distinctive approach to cancer therapy, which is based on disrupting the functioning of highly amplified oncogenic extrachromosomal DNA fragments.
Nido Biosciences Inc. emerged from stealth by unveiling $109 million in series A and B equity funding and a clinical-stage program in spinal and bulbar muscular atrophy.
Diogenx SAS raised €27.5 million (US$30.4 million) in a series A round to move a novel therapy for type 1 diabetes into clinical development. The Marseille, France-based company is building on the research of co-founder Patrick Collombat, an expert in beta-cell regeneration, who is based at the Insitute of Biology Valrose and the University Côte d’Azur, in Nice. Its lead drug candidate comprises a recombinant R-spondin protein, which acts on the Wnt/beta-catenin signal pathway to boost replication of endogenous functioning beta-cells.
Swedish Orphan Biovitrum (Sobi) AB is offering a hefty 95% premium to shareholders in CTI Biopharma Corp., as it tables a $9.10-per-share cash offer for the company, which implies a total equity valuation of $1.7 billion. Shares in Seattle-based CTI (NASDAQ:CTIC) had closed May 9 at $4.82 but surged 85% to close at $8.93 during trading May 10. The premium calculation is based on CTI’s 30-day volume-weighted average trading price of $4.67 prior to the deal announcement.
By the end of this year, it will become apparent whether Morphosys AG has executed one of the biotechnology industry’s boldest pivots in recent years or has instead blown $1.7 billion of investor cash on a dud. The day of reckoning is coming a little sooner than expected for the Planegg, Germany-based firm, as it has completed recruitment in a phase III trial of pelabresib in first-line myelofibrosis ahead of schedule. Top-line data from the study are now expected before year-end, instead of early 2024.
Integrated Biosciences Inc., an early-stage startup that is combining synthetic biology and machine learning in the hunt for drugs that tackle cell senescence, has demonstrated its capabilities in a newly published study in Nature Aging on May 4, 2023, which employed artificial intelligence to identify three novel compounds that are highly selective for Bcl-2 and that exhibit favorable medicinal chemistry profiles.
