Shares of Sarepta Therapeutics Inc. (NASDAQ:SRPT) tumbled 49%, or $83.56, to trade midday at $85.39 after the Cambridge, Mass.-based firm made public data from the first part of Study 102, testing the safety, efficacy and tolerability of a single dose of SRP-9001 (rAAVrh74.MHCK7.micro-dystrophin) in 41 patients with Duchenne muscular dystrophy (DMD). A gene transfer therapy intended to deliver its microdystrophin-encoding gene to muscle tissue for the targeted production of the microdystrophin protein, SRP-9001 met the primary biological endpoint of microdystrophin protein expression at 12 weeks post-treatment, as measured by western blot, in DMD participants vs. placebo. Patients on active drug showed an increase in North Star Ambulatory Assessment (NSAA) total score compared to placebo at 48 weeks, but results of the phase II experiment fell short of statistical significance on the primary functional endpoint of improvement in the NSAA total score vs. placebo at after 48 weeks.

Chimerix acquires Oncoceutics in a $78M stock/cash deal

Chimerix Inc., of Durham, N.C., has acquired privately held Oncoceutics Inc., bringing ONC-201, a small-molecule dopamine receptor D2 antagonist and caseinolytic protease agonist for treating recurrent gliomas harboring the H3 K27M mutation, to Chimerix. The oral therapeutic is in a registrational clinical trial, with data expected in 2021. Chimerix will pay Oncoceutics shareholders $78 million, $39 million in Chimerix stock and $39 million in cash. They also could earn development, regulatory and sales milestones of up to $360 million across three development programs and royalties on combined sales of ONC-201 and ONC-206, a DRD2 antagonist and ClpP agonist. Chimerix stock (NASDAQ:CMRX) was having a solid outing on the market as shares were up 59% at midday.

Lilly looks to Blacksmith to Forge new metalloenzyme portfolio

Forge Therapeutics Inc. spinout Blacksmith Medicines Inc. said Eli Lilly and Co. has committed up to $300 million in milestone payments to back its creation of five new immuno-oncology and inflammation-focused medicines targeting human metalloenzymes. Terms of the agreement also included an up-front payment for Blacksmith and investment by Lilly in the San Diego-based company in exchange for a convertible note. Following the discovery work, Lilly has rights to continue R&D and commercialize the assets if warranted. Forge and its partner, Evotec AG, launched Blacksmith in 2017.

Terns closes $87M series C financing to advance NASH pipeline

HONG KONG – U.S. and China-based Terns Pharmaceuticals Inc. closed an $87 million series C financing round that will pave the way for the company’s pipeline of candidates to treat nonalcoholic steatohepatitis (NASH). Terns’ pipeline is focused on differentiated monotherapies and combination therapies with clinically validated mechanisms of action, CEO Senthil Sundaram told BioWorld. The company plans to use the proceeds of its latest fundraising round to advance clinical trials for three candidates across its NASH pipeline, including the ongoing phase IIa LIFT study in approximately 96 patients of TERN-101, a liver-directed non-bile acid farnesoid X receptor agonist. Data are expected in the second half of 2021.

IPOs, SPACs, venture capital: The endless financing options of 2020

When uncertainty strikes, survival instincts flourish. That is exactly how the biopharma industry weathered 2020 and the global spread of the devastating SARS-CoV-2 virus. “The theme for 2020 was, ‘If the capital is there, take it. It’s an uncertain future’,” said Gabriel Cavazos, managing director in investment banking at SVB Leerink. A panel of financial and company executives agreed, during an advanced session of the virtual Biotech Showcase 2021, that “2020 was a phenomenal year across the board,” Cavazos said. “Nobody expected us to be as productive as we were.”

Theolytics advancing evolution of oncolytic viruses with $6.8M series A

LONDON – Theolytics Ltd. raised $6.8 million in a series A round to further develop its platform technology and advance the lead oncolytic virus program toward the clinic. “We are set up for 2021 and beyond,” said Charlotte Casebourne, CEO. “We have one of the largest libraries of oncolytic viruses and are progressing programs across a number of different indications,” she told BioWorld. Rather than trial and error cell culture experiments, the viruses in the library have been generated through an accelerated Darwinian selection approach that depends on the fact that the fittest viruses can outcompete their brethren when exposed to a specific niche.

Also in the news

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