Bio Roundup: RNAi’s Big Day, CAR-T For Kids, Drugs From Fungi & More

Xconomy National — 

It’s been a year of biomedical milestones in the U.S., including the first approval of a CAR-T cellular immunotherapy, the first smartphone app to treat substance abuse, and the first approval of a cancer drug based on genetic signature instead of the tumor’s organ of origin.

Add to the list the first successful Phase 3 trial for an RNA interference drug, likely leading to a first ever FDA review.

We might not need to go far into the future to look back at 2017 and call it a year when medicine began to significantly change. Perhaps we can even say it now—and there are still three months to go.

While you ponder the big picture, don’t forget to catch up on the week’s headlines below. It’s roundup time.


—For the first time, a medicine using RNA interference is headed for an FDA review. Alnylam Pharmaceuticals (NASDAQ: ALNY) reported positive Phase 3 results for patisiran, a drug for the rare disease transthyretin amyloidosis, and will now file for approval in U.S. and Europe. The news marks a turning point in the quest to turn RNAi, a biological process discovered in 1998, into a drug making method.

—In the wake of the first-ever approval of a CAR-T cell therapy, tisagenlecleucel (Kymriah) from Novartis (NYSE: NVS), Xconomy reported on a second, similar CAR-T, also for kids with recurring leukemia. This other CAR-T, from Seattle Children’s Hospital, has also produced stunning results and some cures, but instead of pushing for FDA approval and commercial status, Seattle Children’s researchers have embarked upon a riskier, more ambitious development path.

—A race is underway to mine the genomes of fungi to discover drugs. LifeMine Therapeutics, a startup co-founded by Harvard University chemical biologist Greg Verdine, just raised $55 million for the field’s biggest bet to date, but other efforts in California and elsewhere are ramping up as well.


—A last-ditch effort to repeal the Affordable Care Act, aka Obamacare, could reach a Senate vote next week. Written mainly by Lindsey Graham (R-SC) and Bill Cassidy (R-LA), the bill would turn federal Medicaid funding into block grants that give states flexibility—and in some cases, a lot less money—to craft their own insurance systems. Groups representing insurers, doctors, nurses, patients, and hospitals have all blasted the bill for several reasons, including more Americans uninsured than under the current system and the possibility that people with pre-existing conditions will be charged exorbitant premiums.

—Republicans are pushing Graham-Cassidy without a substantive hearing, and likely without a Congressional Budget Office evaluation, to meet the end-of-September deadline to pass legislation with only 50 votes in the Senate. The effort has killed, for now, the bipartisan efforts of Senate HELP committee leaders Lamar Alexander (R-TN) and Patty Murray (D-WA) to craft a narrow bill with several short-term fixes for the ACA.


—A new report from MassBio details gender inequality in the Boston-area biotech sector and shows the gap between the perceptions of male and female employees. While the rank and file are split evenly between men and women, the gender gap widens to 76 percent men and 24 percent women in “C suite” jobs, and is even more stark in the boardroom.

—Teresa Spehar, the former VP of intellectual property at Craig Venter’s Synthetic Genomics, has filed suit against the San Diego company, alleging a “boys club” atmosphere that discriminated against her and other female employees. The charges range from unequal pay to exclusion from key decision-making to crude comments by Venter himself. (As Xconomy reported last December, Venter is well known for his abrasive personality.)

—The Justice Department has charged Steve Burrill with 26 counts of fraud and tax evasion for alleged pilfering of his eponymous biotech venture funds. The charges could lead to 30 years in prison and hundreds of thousands of dollars in fines. The unraveling began in 2014 with a lawsuit filed by former Burrill & Co. employees who found that money had “gone missing.”


—Rodin Therapeutics raised $27 million in funding, a round that comes less than a year after an option-to-buy deal the neurological drug startups signed with Biogen (NASDAQ: BIIB) quietly fizzled.

—Flagship Ventures unveiled Kaleido Bioscience, incubating since 2015, and named ex-Cubist CEO Mike Bonney the top executive. Kaleido has already raised $65 million and has multiple microbiome-based therapies in clinical testing.

—Boston biotech accelerator Xontogeny announced its first company is Landos Biopharma, a Virginia Tech spinout that will use a $10 million Series A round to bring an experimental Crohn’s disease drug into clinical trials.

—Disarm Therapeutics of Cambridge, MA, raised $30 million in a Series A round to develop drugs that address an underlying cause of nerve damage in conditions such as multiple sclerosis.

—Less than a month after RegenxBio (NASDAQ: RGNX) reached an $86 million agreement to acquire Cambridge gene therapy developer Dimension Therapeutics (NASDAQ: DMTX), Ultragenyx (NASDAQ: RARE) has stepped in with a competing bid. Dimension says it will review the roughly $138 million cash offer from the Novato, CA, company.


—Reeling from safety problems that forced it to scrap all of its drugs in human clinical testing, Arrowhead Pharmaceuticals (NASDAQ: ARWR) is looking to a new RNAi drug delivery technology to turn its fortunes around.

—Shares of Intercept Pharmaceuticals (NASDAQ: ICPT) fell 24 percent after the FDA sent a letter to clinicians warning of instances of serious liver problems and deaths in patients taking obeticholic acid (Ocaliva), which is approved for primary biliary cholangitis and is being tested in non-alcoholic steatohepatitis.

—Two former GlaxoSmithKline (NYSE: GSK) executives are taking on new roles in biotech investing. Andrew Witty, who was GSK’s CEO for nine years, has joined Hatteras Ventures of Durham, NC, as a venture partner. Former GSK R&D executive Moncef Slaoui is now a partner at Medicxi, an investment firm based in Europe.

—Nabriva Therapeutics (NASDAQ: NBRV) reported positive results from the first of two Phase 3 trials that are testing Nabriva’s antibiotic against drug-resistant bacterial pneumonia infections.

—Shares of Menlo Park, CA-based Versartis (NASDAQ: VSAR) plummeted more than 84 percent after somavaratan—an engineered, long-acting version of human growth hormone—failed a Phase 3 trial in pediatric growth hormone deficiency.

—CRISPR-Cas9 gene editing pioneer Feng Zhang won the $500,000 Lemelson-MIT prize for “big ideas that change fields,” as STAT reports here.

—At a workshop at the National Academies of Sciences, Engineering, and Medicine, top FDA drug evaluator Janet Woodcock said the clinical trials system is “broken” and needs to be updated, Regulatory Focus reported.

—In a blog on his company’s website, Express Scripts (NYSE: ESRX) chief medical officer Steve Miller wrote that the health care system “isn’t set up” to handle a high-priced, one-time treatment like gene therapy, which is nearing its first U.S. approval. Miller wrote that gene therapies will require novel payment systems, and many ideas—like paying for a treatment over time, forming risk pools, and financing one-time payments—are on the table.

Alex Lash and Frank Vinluan contributed to this report.

Photo courtesy of flickr user UCL Mathematical and Physical Sciences via a Creative Commons 2.0 license.