Could long non-coding RNAs be the key to developing organ-specific antifibrotic drugs that only mediate their effects in disease-related contexts? That’s the intriguing hypothesis that Haya Therapeutics SA has set out to explore, and its lead program, in heart failure caused by non-obstructive hypertrophic cardiomyopathy, is now in IND-enabling studies. A first clinical trial is pencilled in for late 2024 or early 2025.
By next June or July, Swedish firm Atrogi AB expects to have data from a first-in-human phase Ia/Ib trial of its novel beta-2 adrenergic receptor agonist, ATR-258, which is in development for type 2 diabetes. The study has completed single ascending and multiple ascending-dose arms in 52 healthy volunteers and recently started recruiting 24 patients onto the phase Ib portion.
Sysnav Healthcare SA and Roche Holding AG entered a new collaboration to develop digital endpoints for use in clinical trials of therapies for a range of neuromuscular disorders. The alliance combines Sysnav’s expertise in wearable technologies and movement evaluation with Roche’s clinical experience. It builds on an existing collaboration that led to the qualification of the world’s first digital endpoint, for evaluating therapies in development for Duchenne muscular dystrophy (DMD). They now aim to put that experience to work in widening its application to other disorders in which movement is a key parameter.
Nextpoint Therapeutics Inc. closed an $80 million series B round to move two novel immune checkpoint inhibitor programs into clinical development in the coming year. The Cambridge, Mass.-based firm is planting a flag in an area of immune signaling that has been largely overlooked by drug developers until now.
Aethon Therapeutics Inc. closed a $30 million series A round to develop a novel antibody-based therapeutic strategy for cancer, which relies on the concomitant use of small-molecule covalent inhibitors that form peptide-drug conjugates or beacons.
For European biotechnology, 2022 was a year of contraction. Disclosed equity investments in European firms engaged in the discovery and development of therapeutics totaled $6.782 billion, down 55% on the previous year’s record-breaking tally of $15.193 billion. Last year’s tally is the worst performance since 2017 and is well below the totals achieved during the two years immediately preceding the onset of the COVID-19 pandemic, which triggered a boom in biotech investing.
Belharra Therapeutics Inc., an early stage firm which is pioneering a new approach to chemoproteomics, has already found an influential backer for the distinctive way it systematically probes the interactions between small-molecule ligands and the proteins to which they bind.
Shares in Dutch RNA editing specialist Proqr Therapeutics NV (NASDAQ:PRQR) surged by as much as 88% Dec. 22 news that Eli Lilly and Co. is substantially expanding an existing preclinical alliance. It is paying Proqr an initial $75 million, which consists of an up-front payment and equity investment, a potential $50 million option fee should it decide to widen the scope of the partnership even further, and up to $2.5 billion in new research, development, and commercialization milestones. Proqr would also receive royalties on any resulting product sales.
Anocca AB raised €25 million (US$26.5 million) in venture debt financing from the European Investment Bank to maintain its progress toward the clinic. “We’re quickly moving towards regulatory filings next year,” CEO and co-founder Reagan Jarvis told BioWorld. The company aims to start its first clinical trial in 2024.
Dialing down the immune response remains at the heart of myriad drug development efforts in autoimmune disease. Targeting cytokines, such as tumor necrosis factor alpha or interleukin-12 (IL-12) and IL-23, IL-6, or IL-17, or modulating immune cell trafficking by targeting sphingosine-1-phosphate receptor or integrins, are therapeutic mainstays. But chronic immunosuppression and all its attendant safety concerns is the price that many autoimmune disease patients pay to remain in remission.
