Verge Genomics Inc. has entered a second big AI deal with a large drug company. Privately held Verge will receive up to $42 million, including up-front, equity and near-term payments from Alexion, Astrazeneca Rare Disease, to identify multiple targets for rare neurodegenerative and neuromuscular diseases. The deal could top out at $840 million. There also is potential for downstream royalties.
The interleukin-17 (IL-17) family is involved in innate immunity regulation and plays a crucial role in inflammatory and autoimmune disorders by sustaining inflammation during the onset and progression of diseases such as rheumatoid arthritis (RA), psoriasis or inflammatory bowel disease (IBD). Researchers from the University of Naples have reported on a new biological entity, Ab-IPL-IL-17, that displays potent anti-inflammatory activity in preclinical testing.
While an “unexpected placebo effect” marred its primary endpoint, the pivotal phase II/III study testing AMO-02 (tideglusib) showed clinically significant benefits across a range of functional and objective assessments, according to developer Amo Pharma Ltd., which is prepping to meet with regulators to discuss potential approval for use in children and adolescents with congenital myotonic dystrophy type 1 (CDM1), an ultra-rare subtype of myotonic dystrophy type 1 for which no treatment options are available.
Astrazeneca AB has synthesized IL-1 receptor-associated kinase 4 (IRAK4) inhibitors reported to be useful for the treatment of cancer, systemic lupus erythematosus, gout, rheumatoid arthritis, psoriasis, Sjögren’s syndrome, myositis and atopic dermatitis, among others.
Boston Immune Technologies and Therapeutics Inc. (BITT) has been awarded funding by the National Institutes of Health (NIH) to support the preclinical development of BITT-101, its CD40 antagonist antibody for treating Sjögren’s syndrome. The award will help support steps toward an IND filing next year.
Safe Orthopaedics SAS reported results from a biomechanical study comparing its new surgical technique Sycamore – a pedicle-anchored implant – with standalone balloon kyphoplasty. The results published in summer 2023 in the Journal of Experimental Orthopaedics showed a “significant increase” in compression strength with patients using Sycamore.
Researchers at F. Hoffmann-La Roche Ltd. and Hoffmann-La Roche Inc. have synthesized benzimidazole derivatives acting as serine/threonine-protein kinase SIK2 (QIK) and/or SIK1 (SNF1LK) and/or SIK3 (QSK) inhibitors reported to be useful for the treatment of rheumatoid arthritis, juvenile idiopathic arthritis, nonalcoholic steatohepatitis, primary sclerosing cholangitis, inflammatory bowel disease, atherosclerosis, type 2 diabetes and glomerulonephritis, among others.
Researchers working at the Lady Davis Institute, Jewish General Hospital, McGill University, have combined whole-exome sequencing (WES) from nearly 300,000 samples from the multiancestry UK Biobank, with ultrasound-derived heel estimated bone mineral density (eBMD) and genome-wide association (GWAS) data to identify potential future therapeutic targets for patients with osteoporosis.
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