Aviadobio Ltd. has entered a potential $2.18 billion license and commercialization agreement for its frontotemporal dementia gene therapy, AVB-101, with Astellas Pharma Inc. Astellas is making a $20 million equity investment in London-based Aviadobio and will pay up to $30 million up front in advance of deciding whether or not to exercise the exclusive option to worldwide rights. If it takes up the option, the Japanese pharma will make subsequent license fee and milestone payments of up to $2.18 billion.

Mestag’s activated fibroblast approach draws Merck in $1.9B deal

Mestag Therapeutics Ltd. has sealed a potential $1.9 billion agreement with Merck & Co. Inc., in which it will apply its expertise in activated fibroblasts to identify novel targets for inflammatory diseases. The pharma company has the option to license one or more targets, up to a prespecified number, and will take on all subsequent discovery, development and commercialization work. Cambridge, U.K.-based Mestag is getting an up-front payment and access fees; reaching the $1.9 billion headline figure will depend on products reaching the market. The deal rests on the company’s activated fibroblast technology with which it models the pathogenic role of fibroblasts in disease.

Sage stumbles in Alzheimer’s while awaiting Huntington’s data

Bad news has buffeted Sage Therapeutics Inc. twice in the past few months. Its placebo-controlled phase II Lightwave study of dalzanemdor in Alzheimer's disease has missed the primary outcome measure, prompting the company to stop development of the NMDA receptor positive allosteric modulator in the indication. Sage is awaiting top-line phase II data of dalzanemdor in Huntington’s disease before year’s end. In late July, the company stumbled in a phase II study of Zurzuvae (zuranolone), a neuroactive steroid designed to function as a positive allosteric modulator of gamma-aminobutyric acid A receptors, in treating essential tremor. Sage stock had sagged about 25% before the market opened but had rallied by midday to $6.75 a share, a rise of half a percentage point.

Make-or-break adcom next step for Stealth’s Barth syndrome drug

By now, Stealth Biotherapeutics Inc. had hoped the U.S. FDA would have approved its lead candidate, elamipretide. Instead, it’s headed to a meet-up Thursday with the agency’s Cardiovascular and Renal Drugs Advisory Committee. The discussion and vote at that meeting could be make-or-break for patients with Barth syndrome, an ultra-rare debilitating mitochondrial disease that has no approved therapies. The rareness of the disease, which afflicts 230 to 250 males worldwide, has complicated development of the treatment. Consequently, elamipretide has been caught in what Stealth has described as a “regulatory churn” for the past five years, with its development program and review handed off from one FDA division to another at least three times. The conflicting guidance from those divisions on what’s needed to confirm results has raised the question of whether approval under the FDA’s current regulatory scheme is even possible with such a small patient population.

Protein design: if you can’t find ’em, make ’em

David Baker, director of the Institute for Protein Design at the University of Washington School of Medicine, is a pioneer in protein design. His contributions have been recognized with countless awards, and now, a place among the 2024 Clarivate Citation Laureates. Baker’s lab has developed several open-source software applications for nanotechnology and biomedicine. With these methods, scientists build new proteins that bind to drug targets and block them or activate cellular signals.

Wuhan YZY, CTTQ ink $143M China bispecific antibody deal

Wuhan YZY Biopharma Co. Ltd. is out-licensing lead candidate M-701, a CD3/EpCAM bispecific antibody, to Chia Tai Tianqing Pharmaceutical Group Co. Ltd. (CTTQ) for China rights in a deal worth up to $143 million. Under the deal, Sino Biopharmaceutical Ltd. subsidiary CTTQ is granted an exclusive license to develop, register, manufacture and commercialize M-701 within mainland China. Under the terms, Lianyungang, China-based CTTQ will pay YZY Biopharma an up-front payment of ¥315 million (US$44.68 million) and sales-based milestone payments up to ¥700 million, plus royalties.

2024 Physics Nobel goes for foundations of AI

It’s hard to know where to start in describing the biopharma applications of the 2024 Nobel Prize in Physics. It was awarded to John Hopfield and Geoffrey Hinton “for foundational discoveries and inventions that enable machine learning with artificial neural networks.” Neural networks are the computer programs that form the basis for machine learning, which itself is a subset of artificial intelligence.

BioWorld Insider podcast: Gene and cell therapies will propel innovation, says Astellas CCO

Gene and cell therapies will drive innovation for the next 10 years, Claus Zieler, the chief commercial officer at Astellas Pharma Inc., said in this episode of the BioWorld Insider podcast. Developers are on the cusp of breakthroughs because a gene can now be replaced “and that means we can potentially cure a disease rather than intervening in a disease.” Zieler also shared his thoughts on creating sustainable health care in aging societies, the innovation cycle gap between the U.S. and Europe, and how collaboration between business and government is critical in forging ahead in this wide-ranging discussion.

Also in the news

Astrazeneca, Avillion, Bergenbio, Biofrontera, Biovaxys, Bridge Medicines, Camp4, Cytodyn, Galecto, GE Healthcare, Hansa, Insignis, Johnson & Johnson, Kalvista, Kezar, Kronos, KSQ, Onkure, Opthea, Optinose, Rege Nephro, Reneo, Scholar Rock, Tourmaline, Ultragenyx, Upstream, Virios, Wex