Remix Therapeutics Inc. is getting $30 million up front in a potential $1 billion agreement with Roche AG aimed at developing small-molecule therapies designed to modulate RNA processing. The deal, which includes up to $12 million in near-term milestones, will center on Remix’s Remaster drug discovery platform, with the companies collaborating on discovery and preclinical activities, and Roche taking on development and commercialization of any resulting products. At the same time, Remix announced the closing of a $60 million financing, expected to support development of lead in-house program REM-422, set to move into the clinic this year in adenoid cystic carcinoma and acute myeloid leukemia/myelodysplastic syndromes.

No injunction for state’s epinephrine ‘affordability program’

One of the latest state laws to control prescription drug prices is an unconstitutional taking, a U.S. district judge said last week, but he refused to grant a preliminary injunction to shield manufacturers from the effects of Colorado’s new law while Teva Pharmaceuticals USA Inc.’s constitutional challenge moves through the courts. Effective Jan. 1, the law establishes what’s dubbed as an “affordability program,” setting a monthly $60 out-of-pocket cap for a two-pack of epinephrine autoinjectors such as Epipen and its generic equivalents. If a consumer is insured, the insurance company has to cover the rest of the cost of the emergency-use combination product. But with uninsured or underinsured consumers, the law requires the manufacturer to reimburse the pharmacy for its acquisition cost or resupply the product at no cost.

Medilink nets second $1B deal, out-licenses ADC to Roche

Three months out from its first $1 billion deal with Biontech SE for an antibody-drug conjugate (ADC), Suzhou, China-based Medilink Therapeutics Co. Ltd. clinched another potential $1 billion ADC deal, but this time with Roche Holding AG. Under the terms announced on Jan. 2, Basel, Switzerland-headquartered Roche will pay $50 million in up-front and near-term milestone payments to gain exclusive global rights to develop, manufacture and commercialize YL-211 – Medilink’s c-mesenchymal epithelial transition factor-targeting ADC for solid tumors. The deal could reach potentially $1 billion upon reaching additional development, regulatory and commercial milestones, along with potential royalties tiered on global annual net sales.

Anavex rallies a bit after phase II/III failure

Anavex Life Sciences Corp. stock (NASDAQ:AVXL) sunk 35% on Jan. 2 after a phase II/III study failure in Rett syndrome but had a next-day rebound of 12% at midday with shares going for $6.82 each. Top-line data from the randomized, double-blind, placebo-controlled study of the company’s lead asset, Anavex 2-73 (blarcamesine), showed improvement but did not reach statistical significance on the co-primary endpoint, the Rett Syndrome Behaviour Questionnaire. It missed the other co-primary endpoint, too, as measured on the Clinical Global Impression-Improvement scale. Anavex said “a large placebo effect” may have obscured the oral activator of the sigma-1 receptor’s impact on participants.

Tome buys Replace, gains DNA editing technology 

Genome editing specialist Tome Biosciences Inc. now has all the bases covered, after arriving on the scene in December with $213 million funding and three weeks later announcing the acquisition of fellow precision editing company, Replace Therapeutics Inc. for up to $185 million. The combination gives Tome technology with which to engineer changes ranging from tens of base pair edits to hundreds of thousands, at specific locations in the genome and without making any double-strand breaks. “[That] provides us with the flexibility to choose the right editing tools for the right indications, expanding the types of diseases that we have the potential to cure,” said Rahul Kakkar, president and CEO of Tome.

Phase III diabetes data positive for Sciwind’s GLP-1 ecnoglutide; obesity results expected soon

Sciwind Biosciences Co. Ltd.’s injectable glucagon-like peptide-1 (GLP-1) analog, ecnoglutide (XW-003), achieved positive top-line results in a phase III trial in Chinese adults with type 2 diabetes. A long-acting, cAMP signaling biased GLP-1 analog, ecnoglutide is being developed for treating type 2 diabetes and obesity. GLP-1 receptor agonists are increasingly gaining attention in the obesity therapy area. “We are very pleased to see the positive results from the first phase III clinical trial of ecnoglutide in China,” said Sciwind Biosciences CEO Hai Pan. 

Readouts in pair of dystrophy trials lift Dyne shares

Dyne Therapeutics Inc. offered initial positive data from the phase I/II Achieve trial with DYNE-101 in myotonic dystrophy type 1 and the same-stage study called Deliver with DYNE-251 in Duchenne muscular dystrophy who are amenable to exon 51 skipping. Favorable safety supports dose escalation, the firm said. Waltham, Mass.-based Dyne’s shares (NASDAQ:DYN) were trading midday at $15.36, up $2.29, or 17%.

BioWorld highlights the top biopharma trends of 2023

As industry gears up for the annual J.P. Morgan Healthcare conference to kick off 2024, take a moment to review the successes and challenges that defined 2023. It was a year when biopharma faced a tough capital market, as it settled into the new post-pandemic normal, and a year when drug pricing debates went global. But the industry continued to innovate, as always, celebrating advances in gene editing and introducing the world’s first two RSV vaccines. And for companies working on GLP-1 drugs or antibody-drug conjugates, 2023 was a very, very good year.

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