In a move that Sarepta Therapeutics Inc.’s president and CEO, Doug Ingram, called “transformational” for the company, Sarepta granted Roche Holding AG exclusive commercial rights outside the U.S. for SRP-9001, its gene therapy for Duchenne muscular dystrophy (DMD). In exchange, Sarepta, of Cambridge, Mass., receives $1.15 billion up front and as an equity investment, up to $1.7 billion in regulatory and sales milestones and royalties on net sales, which are expected to be in the midteens. The two companies will equally share the global development expenses, while Sarepta retains all of SRP-9001’s rights in the U.S. China, Ingram said in this morning’s investor conference call, was likely a clear target for Roche, of Basel, Switzerland, noting there are 60,000 DMD patients in China and that “outside the U.S., every hour of every day, a child is dying of DMD.”

FDA brings good tidings, with new drugs for breast cancer, insomnia and schizophrenia

Three new U.S. drug approvals, one accelerated for need, have handed year-end victories to drugmakers Daiichi Sankyo Co. Ltd., Astrazeneca plc, Eisai Inc. and Intra-Cellular Therapies Inc. Daiichi's Enhertu (trastuzumab deruxtecan), the subject of a $6.9 billion deal with Astrazeneca, won accelerated approval for the third-line treatment of adults with unresectable or metastatic HER2-positive breast cancer. Eisai's Dayvigo (lemborexant) was approved to treat insomnia. Intra-Cellular's Caplyta (lumateperone) was approved to treat schizophrenia.

Tracon buys rights to subcutaneous PD-L1 antibody from China’s 3D Medicines and Alphamab 

BEIJING – U.S. biotech Tracon Pharmaceuticals Inc., of San Diego, has in-licensed from Chinese drugmakers 3D Medicines (Beijing) Co. Ltd. and Jiangsu Alphamab Biopharmaceuticals Co. Ltd. the rights to develop envafolimab, a subcutaneous PD-L1 antibody, to treat soft tissue sarcoma in North America. Envafolimab was invented by Alphamab and is being co-developed with 3D Medicines. Known as KN-035 in Alphamab’s pipeline, envafolimab is potentially the world’s first subcutaneously injectable PD-L1 inhibitor to qualify as a best-in-class drug. Under the terms, Tracon will develop and commercialize envafolimab for its orphan indication. The license, which is exclusive and nontransferable, covers the U.S., Canada, Mexico and each of their dependent territories, where most of the clinical work will take place.  

Australia unveils new companion diagnostics regulatory framework

Following lengthy consultations with industry, Australia’s Therapeutic Goods Administration has released its new regulatory framework for in vitro companion diagnostics (IVD CDx) that becomes effective in February. The new framework provides a 30-month grace period for tests that already appear in the Australian Register of Therapeutic Goods, but any new CDx must be immediately in compliance as of the Feb. 1, 2020, deadline.

Newco news: Gene therapy startup Dinaqor ‘building a highway to the human heart’

DUBLIN – Compared to other indication areas, gene therapy for cardiac disease is still at the “caveman” stage, according to Dinaqor AG CEO and Chairman Johannes Holzmeister. The Pfäffikon, Switzerland-based firm aims to drag it into the modern era by applying a novel locoregional delivery system to its adeno-associated virus 9 (AAV9) vector technology, in order to achieve high levels of transduction and high levels of selective transgene expression in cardiac cells. The company recently entered a strategic collaboration with Basel-based CDMO Lonza AG on gene therapy process and analytical development, which will underpin its entry into clinical development for cardiac myosin-binding protein-C (MyBPC3) cardiomyopathies. It plans to conduct clinical trials in the U.S. and Europe.

Also in the news

AB Science, Acorda, Akari, Astrazeneca, Beigene, Biomarin, Contrafect, Cytodyn, Eisai, Fibrogen, Fortress Biotech, Intra-Cellular, J&J, Juvisé, Leo Pharma, Monopar, Neoleukin, Oncology Pharma, Oncosec, Phathom, Progenics, Sangamo, Sanofi, Seattle Genetics, Sosei, Synthorx, Taris, TG Therapeutics, Theravance, Trevena, Viiv Healthcare, Xoma