San Diego-based Retrophin Inc. is taking over Orphan Technologies Ltd., of Lexington, Mass., to bring aboard OT-58 for the treatment of classical homocystinuria. The enzyme replacement therapy is being evaluated in phase I/II development against the disease, a rare metabolic disorder characterized by elevated levels of plasma homocysteine that can lead to stroke and heart attacks, ophthalmologic and skeletal complications, as well as developmental delay. Retrophin will make an up-front payment of $90 million, and Orphan shareholders could collect as much as $427 million more if development and commercial milestones are hit. Retrophin is also on the hook for a tiered mid-single-digit royalty on net sales of OT-58 in the U.S. and Europe, plus a milestone payment if a pediatric rare disease voucher is granted.

Ultragenyx and Solid firm up a Duchenne collaboration

Ultragenyx Pharmaceutical Inc., of Novato, Calif., and Solid Biosciences Inc., of Cambridge, Mass., will collaborate on developing and commercializing new gene therapies for treating Duchenne muscular dystrophy. Ultragenyx is investing $40 million in Solid, which saw its own Duchenne program held back by a clinical hold that was lifted Oct. 1 after nearly a year in limbo. Ultragenyx also agreed to pay up to $255 million in milestone payments in the deal. Ultragenyx receives an exclusive license for any product expressing Solid’s microdystrophin construct from AAV8 and variants thereof in clade E for treating Duchenne and other diseases resulting from lack of functional dystrophin, including Becker muscular dystrophy. Solid’s stock (NASDAQ:SLDB) was trading 3.5% upward at midday, while Ultragenyx stock (NASDAQ:RARE) dropped nearly a full percentage point.

Sirnaomics secures $105M series D, preps for potential IPO

RNAi therapeutics developer Sirnaomics Inc. has wrapped up a $105 million series D financing that will support ongoing development of its cancer and fibrotic disease programs plus early stage programs targeting metabolic disease and viral infections. But with both new and existing investors jumping in, the company is also preparing for something bigger: its next phase of growth, including a near-term IPO, CEO Patrick Lu said.

Immuneonco bags $25M in series B for CD47 antibodies development

HONG KONG – Shanghai, China-based Immuneonco Biopharmaceuticals Co. Ltd. bagged $25 million in series B financing in October. “The financing was led by Lilly Asia Ventures, with participation from Lyfe Capital and Shanghai Milestone Asset Management. Existing investor Licheng Capital also participated,” Wenzhi Tian, CEO at Immuneonco, told BioWorld. The funding will mainly support the companies’ immunotherapies, which include IMM-01 and IMM-0306. Phase I trials are underway for both products.

Azura Ophthalmics raises $20M series C for registration study in meibomian gland dysfunction

PERTH, Australia – Azura Ophthalmics Ltd., a company with operations in Melbourne, Australia, and Tel Aviv, Israel, raised a $20 million series C round with existing investors, including Brandon Capital’s Medical Research Commercialization Fund (MRCF), Orbimed, TPG Biotech and Ganot Capital. The funds will allow Azura to advance its lead candidate, AZR-MD-001, into a registration study for the treatment of Meibomian gland dysfunction, which is the leading cause of dry eye disease and affects more than 30 million adults in the U.S. alone.

Artificial intelligence extending biopharma drug research

The pace at which companies are integrating the sophisticated tools of artificial intelligence (AI) and machine learning into their early stage drug discovery and development programs is accelerating as evidenced by the creation of new dedicated AI-focused biopharma companies and the eagerness of drug developers to partner with AI firms. A panel at the BIO Investment Forum delved into the impact of AI on the sector and its ability to assist scientists uncover the intricacies of disease mechanisms and lead them toward novel drug targets.

As M&As lag other years, biopharma deals hit records 

With more than two months left in 2020, it is imminently clear that the $147.3 billion in biopharma projected deal values will likely rise above last year’s record of $160.3 billion, considering the average value per month more than covers the gap. Mergers and acquisitions are currently tracking in fifth place over the past seven years with projected values at $134.7 billion. The 1,630 deals, including licensing, collaborations and joint ventures, are already on top, above the 2019 record of 1,608 deals. 

Indonesia waffles over vaccine administration as halal concerns grow

HONG KONG – Even with vaccine administration just around the corner, Indonesian President Joko Widodo has warned his ministers to not rush the launch of COVID-19 vaccines over mounting public concerns about whether those vaccines would be certified halal, or permissible under Islamic law. Indonesia, the country with the world’s largest Muslim population, was set to start administering vaccines for the novel coronavirus as early as next month.

Also in the news

Akebia, Aldeyra, Allakos, Allarity, Ascendis, Astrazeneca, Aurinia, Bayer, Brickell, Bristol Myers Squibb, Curevac, Evgen, Foghorn, Gilead Sciences, Goldfinch, Immunomedics, Immutep, Innate, Innocan, Iterum, KBP Biosciences, Kezar Life Sciences, Milestone, Nicox, Orbus, Orphazyme, Phasebio, Phosplatin, Promis Neurosciences, Sensyne Health, Sumitomo Dainippon Pharma Oncology