On the heels of the marketing OK in Europe, Protalix Biotherapeutics Inc. and the Chiesi Group’s global rare diseases unit scored approval of Elfabrio (pegunigalsidase alfa-iwxj) from the U.S. FDA for adults with Fabry disease. Read More
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. Read More
How grave they might be remains unknown, but regulatory questions have surfaced in briefing documents related to the soon-to-happen panel meeting on Sarepta Therapeutics Inc.’s gene transfer therapy delandistrogene moxeparvovec in Duchenne muscular dystrophy (DMD). The U.S. FDA’s Cellular, Tissue and Gene Therapies Advisory Committee will meet May 12 to discuss the compound, also known as SRP-9001. Read More
Barely a month and a half after its radioconjugate approach landed a potential $1.7 billion deal with Novartis AG, Bicycle Therapeutics plc drew another big radiopharma player to the table, signing a collaboration agreement with Bayer AG to use Bicycle’s peptide technology to discover and develop radiotherapies against cancer targets. Terms are similar to the Novartis deal, with Bayer paying $45 million up front and Bicycle eligible for development and commercial milestones totaling up to $1.7 billion, and underline the growing interest in the radiopharma space. Read More
Despite hitting a phase III study’s primary and key secondary endpoints for treating diabetic kidney disease, Reata Pharmaceuticals Inc. is discontinuing its bardoxolone programs for the indication. The data that disappointed Reata were, after three years of treatment, finding no separation in end-stage renal disease events between the bardoxolone and placebo groups. Read More
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. Read More
When it comes to CAR T therapies, South Korea is trailing behind the U.S. and China, but the South Korean government sees cell and gene therapies as a space where the country can draw international investors, speakers said during the Bio Korea 2023 conference in Seoul on May 10. Read More
The human genome, the sequence that represents the DNA of our species, was built with a single individual as a model. This all-in-one standard didn’t include the gene variations that make us different or explain why some people develop certain diseases. Four simultaneous studies from the Human Pangenome Reference Consortium have published a sequence based on 47 individuals, beginning to capture the genetic diversity that defines humans. Read More
New hires and promotions in the biopharma industry, including: Alia, Cerevel, CNS, Hutchmed, Immodulon, Impel, Jasper, Kalivir, Neurotrauma, Nutcracker, Sonalasense, Wugen. Read More
Regulatory snapshots, including global drug submissions and approvals, clinical trial approvals and other regulatory decisions and designations: Abbvie, Acrivon, Adaptive, Alphamab, Astrazeneca, Catalyst, Freya, Genentech, Radiopharm, Rocket, Salarius. Read More
