Actinium Pharmaceuticals Inc. is receiving an up-front $35 million with a possible $417 million in regulatory and commercial milestones in its new license and supply agreement with Immedica Pharma AB, of Stockholm. Immedica is getting the exclusive European, Middle Eastern and North African rights to Iomab-B, an antibody radiation conjugate comprising apamistamab, a CD45-targeting antibody, and the radioisotope iodine-131. Actinium is eligible for royalties in the mid-20% range on net sales.
Bluebird Bio Inc. became the latest in a spate of gene therapy firms to disclose restructuring plans, as the company aims to save $160 million over the next two years, saying goodbye to about a third of its workforce. It’s the other shoe to drop after Cambridge, Mass.-based Bluebird rattled Wall Street with phraseology in the firm’s fourth-quarter earnings report March 4 that expressed “substantial doubt” regarding whether operations could go on.
Spotlight Therapeutics Inc., a company developing cell-targeted in vivo CRISPR gene editing biologics, has raised $36.5 million in series B financing to support advancement of its first-in-class immuno-oncology program and further applications of its in-house technology platform. The financing round was co-led by new investors GordonMD Global Investments and Epiq Capital Group, with participation from Magnetic Ventures, as well as existing investors GV, Emerson Collective and others.
Novartis AG, an early and active player in bringing gene therapies to market, has agreed to pay Voyager Therapeutics Inc. $54 million up front and up to $1.7 billion in fees and milestone payments for options to license up to five next-generation adeno-associated virus (AAV) capsids to use as gene therapy vectors for neurological diseases.
U.K. biotech Complement Therapeutics Ltd has come out of stealth mode with €5 million ($5.7 million) in seed funding to tackle complement-related diseases, initially targeting the currently untreatable condition geographic atrophy due to dry age-related macular degeneration.
LONDON – In the largest-ever series A for a Spanish biotech, Splicebio S.L. has raised €50 million (US$56.9 million) to apply its protein splicing technology to the delivery of large genes that do not fit into existing vectors. The company claims its approach will overcome the capacity constraints of adeno-associated viral vectors (AAVs), by splitting genes into parcels and reconstituting the proteins they express in vivo.
In one of its familiar U-turns, the U.K. National Institute for Health and Care Excellence (NICE) has recommended NHS England should fund a rare disease gene therapy from Orchard Therapeutics plc, considered to be the world’s most expensive drug. The list price for Libmeldy (atidarsagene autotemcel) in England and Wales is £2,875,000 (US$3.9 million), making it the most expensive drug that NICE has ever evaluated.
Sio Gene Therapies Inc. is retrenching the business as it terminated the company’s AXO-Lenti-PD license agreement with Oxford Biomedica plc for treating Parkinson’s disease. Also, Sio’s CEO, Pavan Cheruvu, is leaving the company. Sio said it is deprioritizing its Parkinson’s disease program due to several factors, including resource requirements and development timelines “to reach meaningful value inflection for the program and an increasingly challenging market and regulatory environment” for the indication.
Having unveiled more data from the ongoing, global phase III Gener8-1 study with Roctavian (valoctocogene roxaparvovec, also known as valrox), Biomarin Pharmaceutical Inc. remains on track to file a regulatory submission with the FDA in the second quarter of this year for the gene therapy to treat adults with severe hemophilia A. The EMA is already reviewing a marketing authorization application.
A raft of potentially high-value drug development collaborations, most for gene and RNA therapies, led the first day of the 40th Annual J.P. Morgan Healthcare Conference Monday. Pfizer Inc. enlisted Beam Therapeutic Inc. to advance in vivo base editing programs for up to $1.35 billion, while Bayer AG tapped Mammoth Biosciences Inc.’s in vivo CRISPR systems expertise in a potential $1 billion-plus deal. Selecta Biosciences Inc. inked a new $1.1 billion partnership with Ginkgo Bioworks Inc. to develop next-generation gene therapy capsids, while Acadia Pharmaceuticals Inc. agreed to pay Stoke Therapeutics Inc. as much as $967 million to develop RNA-based medicines. Work on new mRNA vaccines and therapies is also proceeding, with both Pfizer and Biontech SE announcing new collaborations in the space.
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