Bio Roundup: Nationwide’s Rise, a Surgeon’s Quest, CRISPR USA & More

Xconomy National — 

Persistence and perseverance were the themes of two of our top stories this week.

First there’s the story of Nationwide Children’s Hospital. The Columbus, Ohio, hospital chose to invest in gene therapy when the field was in its darkest days. After some ups and downs, the team it put in place helped advance some of the most promising gene therapies currently in human testing—and put Nationwide in the national spotlight as one of those treatments nears FDA approval.

Then there’s Suzanne Ildstad. A longtime transplant surgeon, Ildstad cobbled together enough money through grant funding to start a company and develop a cell therapy for organ transplant patients. After some stops and starts and nearly 20 years of waiting, she is finally on the verge of a test that could lead to a regulatory approval if all goes well.

You don’t have to persevere any longer to get to the roundup, though. This week’s mashup of biopharma deals, data, regulatory news, and more are just ahead.


—Over the past two decades, Nationwide Children’s Hospital has quietly gone from a small regional facility with barely any in-house research of its own to a leader in the booming field of gene therapy. Xconomy profiled Nationwide’s rise through the ranks, which could soon culminate with the US approval of the spinal muscular atrophy gene therapy Zolgensma.

—Human testing of CRISPR-based gene editing technology has officially come to the US. This week, the first two patients in a study at the University of Pennsylvania were given an experimental cancer treatment tweaked with CRISPR. Federal oversight groups cleared the study, funded by the Parker Institute for Cancer Immunotherapy, in 2016. Here’s more from NPR.

—Blackstone Life Sciences led a $100 million investment into a startup, Talaris Therapeutics, developing a cell therapy meant to help free kidney transplant patients from the need for chronic immunosuppressive drugs. The financing marks the latest turn in a nearly two decade long quest by Talaris’s scientific founder and transplant surgeon Suzanne Ildstad, who saw her work, once licensed to Novartis (NYSE: NVS), stall when the Swiss pharma dissolved its cell and gene therapy division.

—Alnylam Pharmaceuticals (NASDAQ: ALNY) set out at a big medical meeting to bolster its case for givosiran, which could soon become just the second-ever FDA approved RNA interference medicine. Xconomy spoke with clinicians about the prospects for the drug, which is being developed for a debilitating genetic disease known as acute hepatic porphyria.


—The US Supreme Court nixed a long-running effort by Allergan (NYSE: AGN) to protect the patents protecting its best-selling glaucoma drug cyclosporine (Restasis) by transferring their rights to a Native American tribe. Elsewhere, two proxy advisory firms recommended that Allergan shareholders reject a bid by activist shareholder Appaloosa Management to split the roles of CEO and chairman, titles both held by Brent Saunders.

—The FDA approved Plenity, a hydrogel capsule that PureTech Health-backed Gelesis developed to help people feel full and eat less.

—Adverum Biotechnologies (NASDAQ: ADVM) said that the FDA had temporarily suspended a Phase 1 study of its experimental gene therapy for age-related macular degeneration earlier this month due to questions about the company’s manufacturing process. Adverum responded to the FDA last week.

—The National Institute for Health and Care Excellence (NICE), the UK’s drug-pricing authority, reversed course and recommended the use of inotersen (Tegsedi), an Akcea Therapeutics (NASDAQ: AKCA) drug for the rare genetic disease hereditary transthyretin amyloidosis. NICE changed its tune after Akcea dropped the drug’s price.

—Less than one year after Achaogen (NASDAQ: AKAO) won FDA approval for an antibiotic for complicated urinary tract infections, the South San Francisco company filed for bankruptcy to sell its assets.


—Contract drug manufacturer Calatent (NYSE: CTLT) paid $1.2 billion for Paragon Bio Services, which makes viral “vectors” companies use to deliver gene therapies. The deal comes less than a month after Thermo Fisher (NYSE: TMO) bought Brammer Bio, another vector developer, for $1.7 billion.

—Insitro and Gilead Sciences (NASDAQ: GILD) kicked off a three-year partnership aiming to use the startup’s AI technology to discover new drugs to treat the liver disorder NASH. Gilead is paying South San Francisco-based Insitro $15 million up front.

—In other AI drug discovery news, Mountain View, CA-based twoXAR will work with South Korea’s SK Biopharmaceuticals to develop new treatments for lung cancer. No financial terms were disclosed.

Arrakis Therapeutics raised a $75 million Series B to build a pipeline of small molecule drugs that target RNA.

—Hatteras Venture Partners launched its sixth fund with an initial $94 million for seed and early-stage investments in life science startups.

—PureTech Health struck a research partnership with Boehringer Ingelheim to develop treatments for disorders associated with dysfunction of the connections between the brain, the immune system, and the gut.

—Nocion Therapeutics, of Cambridge, MA, started up with a $27 million Series A round to develop drugs meant to “silence” neurons that are involved in pain, inflammation, and other conditions, including chronic cough. Here’s more from STAT.

—Kintai Therapeutics emerged from stealth, revealing plans to develop small molecule drugs that tap into the biology of the gut to treat disease throughout the body.

—Fibrocell Science (NASDAQ: FCSC) landed $30 million up front in a collaboration with Castle Creek Pharmaceuticals to develop Fibrocell’s Phase 3-ready gene therapy for the rare, inherited skin disorder dystrophic epidermolysis bullosa.


—Hookipa Pharma (NASDAQ: HOOK) raised $84 million in an IPO that will support mid-stage tests of a drug for the prevention of cytomegalovirus infection.

—Turning Point Therapeutics (NASDAQ: TPTX) nabbed $166 million in an upsized IPO that will help it develop targeted cancer drugs.


—Xconomy profiled San Diego startup Amplyx, which is developing a treatment for infections of Candida auris, a fungus that has become increasingly resistant to other drugs.

—With US regulators set to potentially approve Novartis’s SMA gene therapy Zolgensma next month, the Swiss pharma presented new data at a medical meeting this week meant to support its use. Here’s more from Fierce Pharma.

—An experimental gene therapy licensed by Mustang Bio (NASDAQ: MBIO) from St. Jude Children’s Hospital cured eight infants with a form of a rare immune disorder known as X-linked severe combined immunodeficiency (The gene therapy Strimvelis, owned by Orchard Therapeutics, is approved for a different form of the disease). Bloomberg has more.

—Shares of WaVe Life Sciences (NASDAQ: WVE) fell roughly 24 percent after the company disclosed interim safety results from a Phase 1 study of an experimental RNA-based treatment, suvodirsen, for Duchenne muscular dystrophy. The problem: the side effects, seen in patients who got just one dose of the drug, were “disconcerting,” wrote Stifel analyst Paul Matteis in a research note. WaVe will start a late-stage study later this year.

—Johnson & Johnson (NYSE: JNJ) disclosed the details of a long-term study showing that its Type 2 diabetes drug canagliflozin (Invokana) helps lower the risk of kidney failure. Vantage has more on the results and their implications here.

—Clovis Oncology (NASDAQ: CLVS) scrapped a Phase 2 study of PARP inhibitor rucaparib (Rubraca) in bladder cancer after an early look at the data showed that the drug wasn’t effective.


—In a blog posted on Health Affairs, drug policy researchers Peter Bach and Mark Trusheim proposed abandoning the use of biosimilars to lower the prices of biologic drugs in favor of “post-exclusivity price regulations.” The post got the attention of ex-FDA commissioner Scott Gottlieb, who tweeted that it’s “far too early to throw in the towel on biosimilars,” which have struggled commercially.

—Amgen (NASDAQ: AMGN) said its osteoporosis drug romosozumab (Evenity) would cost $21,900 for a full, 12-month course of treatment. That price puts romosozumab on par with a rival drug sold by Radius Health (NASDAQ: RDUS).


—Xconomy posted a sneak peek at the agenda for our annual half-day life sciences event, “What’s Hot in Boston Biotech,” on May 21 at Bristol-Myers Squibb in Cambridge, MA. This year we’re featuring the gene therapy boom, the latest in Alzheimer’s disease research, the growing need for (and role of) diversity initiatives within the life sciences, community, and much more.


—Obsidian Therapeutics appointed Paul Wotton as CEO, succeeding part-time chief executive Michael Gilman. Gilman is now serving as the full-time CEO of Arrakis.

—Trudy Vanhove jumped from Jazz Pharmaceuticals (NASDAQ: JAZZ) to South San Francisco-based Surrozen to become chief medical officer.

Frank Vinluan contributed to this report.