Biotech Roundup: CRISPR’s 1st Trial, IPO Insiders, Zika & More

Xconomy National — 

It’s a week for the history books. The United Kingdom voted to leave the European Union last night, triggering worldwide financial fears and, perhaps, a breakup of the U.K. itself. Closer to home, Cleveland (pictured) celebrated LeBron James & the Cavaliers’ historic comeback that brought the city its first sports title in over 50 years.

Slightly fewer people paid attention to another historic vote: An NIH panel approved plans for what could be the first-ever human clinical trial of CRISPR-Cas9, the gene editing system that has been taking the scientific world by storm over the past few years. The FDA still has to clear the study, but if it does, it’s sure to become one of the most closely watched scientific events in more than a decade. There’s plenty more news to chew on as well, from the latest developments in the Zika battle to biotech’s sigh of relief when Medicare spending cuts were avoided (for now). Let’s get right to it.

—University of Pennsylvania School of Medicine researchers are planning a cancer trial that could be the first ever to use the gene editing system CRISPR-Cas9 to create a human therapy. The trial plan passed muster with a federal oversight committee earlier this week, but it still must gain FDA approval to begin. The work is being funded by Internet mogul Sean Parker’s new cancer immunotherapy institute, which launched earlier this year with $250 million earmarked for work at Penn and five other medical centers.

—IBM’s Watson Health group said that a commercial product could arrive in 2017 that uses machine learning to digest patient scans, medical records, and other information to help doctors with care decisions.

—Selecta Biosciences (NASDAQ: SELB) of Watertown, MA, became the latest biotech not just to go public this week, but to lean on insiders for help. The company raised $70 million in its IPO, but before the deal current shareholders offered to buy $40 million, or close to 60 percent, of IPO shares. That makes it one of the more extreme recent cases of insider buying in an offering.

—There were two major developments in the fight against Zika virus, which has begun to affect children born in the U.S. First, on Monday, the FDA cleared Plymouth Meeting, PA-based Inovio Pharmaceuticals (NASDAQ: INO) to begin the first clinical trial for an experimental Zika vaccine (check out CNBC’s interview of CEO Joseph Kim for more). Then, in the early hours Thursday, the U.S. House of Representatives passed a $1.1 billion emergency funding bill to fight the virus. The White House had requested $1.9 billion and slammed the House bill, in part because it diverts unspent money that was earmarked for Affordable Care Act administration and the fight against Ebola. Democrats say they will filibuster in the Senate.

—The Associated Press reported that surgeon Paolo Macchiarini faces involuntary manslaughter charges in Sweden related to two patients who died after surgeries he conducted to implant artificial tracheas to replace their damaged windpipes. The work of Macchiarini, who reportedly denied the allegations, led to the formation of Holliston, MA-based Biostage (NASDAQ: BSTG), which was known as Harvard Apparatus Regenerative Technology up until March. Biostage cut ties with the surgeon in 2014.

—Cambridge, MA-based Epizyme (NASDAQ: EPZM) released interim data from the most important trial in its nine-year history, a Phase 2 study of cancer drug tazemetostat. Epizyme has only enrolled a fraction of the patients it aims to, and treated most of them for just a handful of months, so it’s tough to draw any conclusions so far. Epizyme will have more data later this year. Still, shares slumped about 4 percent this week.

—Biotech stocks got a small boost Wednesday when mandatory Medicare spending cuts were avoided, thanks to a slower rise in costs than expected. If cuts had been triggered, the details would have been decided by the Independent Payment Advisory Board, which was put in place by the Affordable Care Act.

—Austin, TX-based Savara Pharmaceuticals bought a Danish drug developer, Serendex Pharmaceuticals, to nab two inhaled experimental therapies. Savara now aims to pull together a funding round to support a future IPO, David Holley reports.

—Funding news: Annexon Biosciences of South San Francisco, CA, raised a $44 million Series B round led by New Enterprise Associates to move its neurodegeneration treatments toward clinical trials…Basel and Cambridge-based CRISPR Therapeutics added $38 million to its previously announced Series B round, bringing the total financing to about $140 million. CRISPR added several new investors in the round, such as Wellington Management, New Leaf Venture Partners, and Franklin Templeton Investments…Boston-based Paratek Pharmaceuticals (NASDAQ: PRTK) raised $55 million in a stock offering after a successful Phase 3 trial last week…Cambridge-based IFM Therapeutics raised $27 million from Atlas Venture, Novartis, and Abingworth. The company is led by Gary Glick, the former founder of Lycera, which inked an option-to-buy deal with Celgene last year.

—EndoCyte (NASDAQ: ECYT) of West Lafayette, IN, is saying goodbye to founder and CEO Ron Ellis. Ellis stepped down suddenly this week and will take home nearly $1 million in severance. Chief operating officer Mike Sherman was named his successor.

Stat profiled Rachel Haurwitz, the CEO of Berkeley, CA-based Caribou Biosciences, which was co-founded by CRISPR-Cas9 pioneer Jennifer Doudna.

—The 2016 Blavatnik Awards for Young Scientists went to Harvard University astronomer David Charbonneau, Scripps Research Institute chemistry professor Phil Baran, and Michael Rape of the University of California, Berkeley, for his research on a process called ubiquitylation that marks unwanted proteins for destruction within a cell. Rape is a co-founder of San Francisco biotech Nurix. Each award winner gets a $250,000 cash prize.

—Cambridge-based Eleven Biotherapeutics (NASDAQ: EBIO) will ax 14 employees, or 70 percent of its existing workforce, to save cash as it continues to evaluate its next steps. Eleven, whose eye drug failed two Phase 3 trials last year, recently sold off one of only two remaining drug candidates in its pipeline.

—Celgene (NASDAQ: CELG) paid a total of $50 million to form a 10-year research consortium with the University of Pennsylvania, Columbia University Medical Center, Johns Hopkins University, and the Icahn School of Medicine at Mount Sinai. The institutions will work on cancer drugs, and Celgene will get an option to license them.

—Warsaw, IN-based OrthoPediatrics filed for an IPO to help support sales expansion and new product development. The company sells orthopedic devices used in surgeries on children.

Photo of the Cleveland Cavaliers’ victory parade courtesy of FirstEnergy via Creative Commons.

Alex Lash contributed to this report