Ironwood Pharmaceuticals Inc., of Cambridge, Mass., amended its collaboration agreement with Astrazeneca plc, of Cambridge, U.K., for the development and commercialization of Linzess (linaclotide) in China. Under the terms of the amended agreement, Astrazeneca obtained exclusive rights to develop, manufacture and commercialize linaclotide in China and will be responsible for all expenses associated with that work. In return, Ironwood will receive up to $125 million, including non-contingent payments totaling $35 million paid in three installments through 2024 and up to $90 million in commercial milestone payments contingent on the achievement of certain net sales targets. Additionally, Ironwood will receive royalties beginning in the mid-single-digit percent and increasing up to 20% based on annual net sales of Linzess in China. Ironwood will no longer jointly fund the development and commercialization of linaclotide or share in the net profit from sales in China. Linzess was approved by the National Medical Products Administration for adults with irritable bowel syndrome with constipation in China in January and is expected to be launched in the market by year-end. The original collaboration between Ironwood and Astrazeneca for linaclotide was established in 2012.

Massachusetts General Hospital, of Boston, said it is launching the first platform trial for amyotrophic lateral sclerosis with three therapies and plans to add two more soon. The experimental treatments included in the study include zilucoplan, a small macrocyclic peptide inhibitor of complement component 5, developed by Ra Pharmaceuticals Inc., of Cambridge, Mass.; verdiperstat, an oral myeloperoxidase inhibitor, developed by Biohaven Pharmaceutical Holding Co. Ltd., of New Haven, Conn.; bioenergetic nanocatalysis (CNM-Au8, nanocrystalline gold) developed by Clene Nanomedicine Inc., of Salt Lake City; pridopidine, a selective S1R agonist, developed by Prilenia Therapeutics Development Ltd., of Columbus, Ohio; and IC14 immunotherapy, developed by Implicit Bioscience Ltd., of Seattle. Partial financial support for the treatments is being provided by the family and friends of Sean Healey, executive chairman of the global asset management company Affiliated Managers Group Inc. (AMG) and the AMG Charitable foundation.

Ose Immunotherapeutics SA, of Nantes, France, said it received a €5.4 million (US$6 million) payment from Bpifrance, triggered by completion of development milestones related to its collaborative program, EFFI-CLIN, focused on testing BI-765063, a SIRPalpha antagonist and myeloid checkpoint inhibitor. The milestone was triggered by reaching several steps, including initiation of studies characterizing the SIRPalpha/CD47 axis and determining preclinical efficacy, completion of regulatory preclinical toxicology studies, manufacturing of GMP-compliant batches and development of a tool used to characterize the immune profile and biomarkers found in patients. BI-765063, which is being developed in partnership with Boehringer Ingelheim GmbH, of Ingelheim, Germany, is in phase I testing in advanced solid tumors. (See BioWorld, April 5, 2018.)

Proof-of-concept data for London-based Storm Therapeutics Ltd.'s RNA modifying enzymes for cancer treatment demonstrated that small-molecule inhibition of methyltransferase like 3 (METTL3) produces the same phenotype and validates METTL3 as a druggable target for cancer. Storm has identified small-molecule inhibitors of METTL3 that are orally bioavailable and show pronounced antitumor efficacy in physiologically relevant, proof-of-concept animal models of acute myeloid leukemia. The data were presented at St. Catherine's College in Cambridge, U.K. Storm recently closed a $38.1 million series A round. (See BioWorld, May 22, 2019.)

Titan Pharmaceuticals Inc., of South San Francisco, received $6.1 million in second-year funding from the NIH's National Institute on Drug abuse for nonclinical development of a Proneura-based, six-month implantable formulation of nalmefene, an opioid antagonist, designed to prevent opioid addiction relapses after detoxification. The award funds the completion of implant formulation development, cGMP manufacturing and nonclinical studies which, if successful, are expected to support Titan's submission of a nalmefene six-month implant NDA.

Verantos Inc., of Palo Alto, Calif., Amgen Inc., of Thousand Oaks, Calif., and the Stanford University School of Medicine have created a study that proposes a definition of and approaches to standards needed for advanced regulatory-grade evidence in cardiovascular medicine. The group assessed several sources of data, including structured and unstructured electronic health records data. For example, sensitivity for myocardial infarction was 29.8% in structured data vs. 90.4% in unstructured data using artificial intelligence. That suggests that for a study with myocardial infarction as an exclusion criterion, using the wrong data source and not measuring sensitivity could result in 70% of patients with previous heart attacks inadvertently being included in the study without any reviewer being aware. The study was published in the Journal of American Medical Informatics Association.

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