After receiving FDA accelerated approval for Vyondys 53 (golodirsen) in Duchenne muscular dystrophy (DMD) last week, Sarepta Therapeutics Inc. followed up with news that it had struck a major transaction with Roche Holding AG, granting the latter exclusive commercial rights outside the U.S. for SRP-9001 (AAVrh74.MHCK7.micro-dystrophin), its gene therapy for DMD. President and CEO Doug Ingram called the deal “transformational” and “the largest licensing transaction in cell or gene therapy history.”  Sarepta receives $1.15 billion, which includes an up-front payment and an equity investment, plus $1.7 billion allocated for potential downstream regulatory and sales milestones and royalties on net sales. 

Astellas Pharma Inc.’s deal to take over Xyphos Biosciences Inc. brings $120 million up front in an arrangement with an eventual value as high as $665 million. Astellas gains the ACCEL (Advanced Cellular Control through Engineered Ligands) platform and will turn Xyphos into a wholly owned subsidiary. The latter has targeted 2021 for its lead candidate to enter human trials in immuno-oncology. U.S. biotech Tracon Pharmaceuticals Inc. 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. 

Sanofi SA began its tender offer to acquire Synthorx Inc. for $68 per share in cash as part of the merger plan announced Dec. 7.  

Theravance Biopharma Ireland Ltd., a subsidiary of Theravance Biopharma Inc., and Pfizer Inc. said they entered a global license agreement for Theravance's preclinical program for skin-targeted, locally acting pan-JAK inhibitors that can be rapidly metabolized. Fortress Biotech Inc. said Baergic Bio Inc., its newly formed partner company, entered an exclusive worldwide licensing agreement with Astrazeneca plc for AZD-7325, an α2/3-subtype-selective GABAA positive allosteric modulator (PAM), as well as an agreement with Cincinnati Children’s Hospital Medical Center to advance clinical development in select central nervous system disorders. 

Newlink Genetics Corp. said it entered an exclusive worldwide license agreement with Ellipses Pharma Ltd. for the development of, and rights to commercialize, NLG-207 (formerly CRLX-101), a nanoparticle formulation of the topoisomerase 1 inhibitor camptothecin. 

Drug approvals 

The number of new molecular entities (NMEs) approved by the FDA this year jumped to 48 with the agency giving the green light to four new drugs including Daiichi's Enhertu (trastuzumab deruxtecan), which 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. Allergan’s Ubrelvy (ubrogepant) became the first of a relatively new class of drugs to be approved for the acute treatment of migraine. 

Financial transactions 

Although it was a shortened week, deals were completed, including: 

Abeona Therapeutics Inc., which closed its underwritten public offering, with a gross offering size of about $103.5 million. Axsome Therapeutics Inc. grossed $200.1 million from its public offering; Neoleukin Therapeutics Inc. generated $86.2 million; and Summit Therapeutics plc and TG Therapeutics Inc. each generated $50 million.  

Rarecyte Inc. reported picking up $22 million in a series F financing round led by Healthquest Capital. 

Word on the Street: BioWorld's favorite quotes of the week 

“As the most advanced single-domain antibody in immune-oncology with the advantage of a subcutaneous dosage, we are confident it will provide a valuable option for cancer patients.”
Ting Xu, CEO, Jiangsu Alphamab Biopharmaceuticals Co. Ltd., which licensed, together with 3D Medicines (Beijing) Co. Ltd., the North America rights to develop envafolimab, a subcutaneous PD-L1 antibody, to treat soft tissue sarcoma to Tracon Pharmaceuticals Inc. 

“GWAS have been done for a range of diseases over the past 15 years and has given huge insights into their genetic basis. But typically, they look at one disease at a time. What UK Biobank allows us to do is look at all diseases simultaneously.”
Gil McVean, professor of statistical genetics and director of the Big Data Institute at Oxford University, who is lead author of a study on the cross-disease components of genetic risk, published in Nature Genetics 

"CAR T cells, given our current state of knowledge, are very unlikely to impact solid tumors."
Steven Rosenberg, chief of surgery at the National Cancer Institute, speaking at the annual meeting of the American Association of Immunologists [Editor’s note: This quote was one drawn from our popular annual feature of industry opinions that paints a picture of the significant events that shaped 2019.]  

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