Bio Roundup: A Gene Editing 1st, China Rises, Schenkein Steps Away

Xconomy National — 

If you’re still recovering from a Labor Day hangover or busy searching for the identity of the anonymous writer of the anti-Trump op-ed for the New York Times, you may have missed a few biomedical firsts.

This week brought the first sliver of human data from an in-body gene editing procedure, and the results were decidedly undecided (more on that below). China also continued its push in biomedicine with regulators there stamping the first approval of a drug discovered and developed in China.

Elsewhere, top biotech executives on the East and West Coasts are on the move, the Theranos saga concluded, and a new nonprofit venture emerged to combat high drug prices. Let’s get the roundup rolling.


—Bay Area-based Sangamo Therapeutics (NASDAQ: SGMO) released the first results from an in-human study of gene editing. The Phase 1 study is not yet complete; four patients with the rare Hunter syndrome (aka MPS II) showed mixed results. One measure showed evidence that the gene editing, using Sangamo’s proprietary zinc-finger nuclease technology, was working as promised, but another measure showed no activity. Investors weren’t impressed and as of midday Thursday had knocked Sangamo shares down 33 percent.

—The National Medicinal Products Administration of China approved fruquintinib (Elunate), a drug from Hutchison China MediTech for colorectal cancer. The approval is the latest step in China’s ascension as an international biotech power. Here’s more from Reuters.


—David Schenkein, the longtime CEO of Agios Pharmaceuticals (NASDAQ: AGIO), will step aside next year after nearly a decade. He transformed a high-risk startup into a public biotech with over 500 employees and two drugs on the market. Schenkein will become executive chairman and be replaced by ex-Celgene executive Jacqualyn Fouse.

—Separately, Agios and Celgene (NASDAQ: CELG) ended an alliance on experimental cancer drug AG-881, which the two have been co-developing for gliomas and solid tumors. Agios now has full rights to the drug.

—Former Kleiner Perkins managing partner Beth Seidenberg surfaced with new venture firm Westlake Village BioPartners, which she will lead with outgoing Amgen (NASDAQ: AMGN) R&D executive Sean Harper. While Westlake will invest its $320 million fund in companies throughout the U.S., Seidenberg says the firm also aims to raise the profile of the Southern California biotech community.

—After a long, sordid saga, disgraced diagnostics company Theranos is dissolving, according to an email to shareholders obtained by the Wall Street Journal.

—GlaxoSmithKline (NYSE: GSK) will cut 650 U.S. jobs, primarily in sales.

—Akcea Therapeutics (NASDAQ: AKCA) will lay off 10 percent of its workforce following the FDA rejection of volanesorsen, its drug for the ultra-rare disease familiarl chylomicronemia syndrome.

—Ovid Therapeutics (NASDAQ: OVID) chief business and financial officer and former analyst Yaron Werber is returning to Wall Street to become a managing director and senior biotech analyst at Cowen and Co.


STAT profiles U.S. Senate candidate and former Celgene CEO Bob Hugin, asking if a Republican pharma executive can overcome American anger about high drug prices and win in a blue state.

—Several states began their argument in a Texas court that the Affordable Care Act should be scrapped.


—Seven organizations representing 500 U.S. hospitals have teamed to create Civica Rx, a nonprofit  generic drug maker. The move is meant to battle high drug prices and manufacturing shortages. CNBC has more here.

—NHS England, Britain’s national healthcare system, cut a deal with Novartis to make the CAR-T cell therapy Kymriah available to children with an aggressive form of leukemia, though specifics weren’t disclosed. Britain’s drug pricing watchdog, NICE, rejected a different CAR-T treatment from Gilead Sciences (NASDAQ: GILD), citing its price.


—Liquid biopsy maker Guardant Health filed paperwork for a $100 million IPO. The firm, which has been selling its lead product since 2014, brought in nearly $50 million in 2017 revenue and incurred a net loss of $83 million. The SoftBank Group is Guardant’s top shareholder, with a 39 percent stake.

—Harvard spinout 28-7 Therapeutics launched with a $65 million Series A round that it will use to develop small molecule cancer drugs that target microRNAs.

—4D Molecular Therapeutics raised $90 million in Series B financing as the Emeryville, CA, company prepares to begin testing a gene therapy for the rare eye disorder choroideremia.

—With a new $80 million round and an atypical close-knit relationship with patient foundations, Fulcrum Therapeutics, of Cambridge, aims to file for an IPO next year, CEO Robert Gould told Xconomy.

—PhaseBio Pharmaceuticals, of Malvern, PA, raised a $34 million Series D to continue developing drugs for rare cardiopulmonary diseases like pulmonary arterial hypertension.

Pfizer spinout Springworks Therapeutics named former Alexion Pharmaceuticals executive Saqib Islam its CEO and cut a deal with BeiGene (NASDAQ: BGNE) to begin testing two experimental cancer drugs in tandem against solid tumors. Springworks president and co-founder Lara Sullivan left the company in May, according to a spokesperson.

—Biohaven Pharmaceuticals (NYSE: BHVN) grabbed rights to an experimental drug for multiple systems atrophy, a rare neurodegenerative disease, from AstraZeneca (NYSE: AZN) for an undisclosed sum. It plans to start a Phase 3 trial.


—Shares of Arrowhead Pharmaceuticals (NASDAQ: ARWR) climbed 37 percent on data from an early stage study of an experimental RNA interference drug for hepatitis B.

—Bluebird Bio (NASDAQ: BLUE) updated results from a late-stage study of experimental gene therapy Lenti-D for the rare brain disorder cerebral adrenoleukodystrophy. Bluebird also reached agreements with U.S. and European regulators on  data to support Lenti-D approval.

—Teva Pharmaceutical (NYSE: TEVA) returned to Active Biotech (NASDAQ Stockholm: ACTI) the development rights for neurodegenerative drug laquinimod. The move follows the drug’s clinical failures in multiple sclerosis last year and Huntington’s disease in July.

—Shares of Waltham, MA-based Histogenics (NASDAQ: HSGX) plummeted 72 percent after the firm’s cell therapy Neocort failed a Phase 3 trial. Histogenics says it still intends to meet with the FDA to discuss potential approval.

—Privately held CAR-T developer Eureka Therapeutics said its liver cancer treatment had promising early-stage results, with tumors shrinking in three of six patients. The study was held in China; Emeryville, CA-based Eureka plans a U.S. trial next year, according to Bloomberg.

—Shares of Netherlands-based ProQR Therapeutics (NASDAQ: [[tkcker:PRQR]]) doubled after early-stage results from an experimental RNA-based drug for Leber’s congenital amaurosis 10 (LCA10), a rare, inherited type of vision loss. A late-stage trial will begin next year. Editas Medicine (NASDAQ: EDIT) is developing a rival gene editing treatment for LCA10.

—The FDA will extend by three months its review of Roche/Genentech’s four-drug regimen for lung cancer that includes the Roche drugs atezolizumab (Tecentriq) and bevacizumab (Avastin).


—Thanks to everyone who attended our sold out Xconomy Awards Gala this past week. You can see the award winners here, and stay tuned for a slide show.

Frank Vinluan and Alex Lash contributed to this report.