Bio Roundup: CRISPR Tests, Parkinson’s Questions, Opioid Bills & More

Xconomy National — 

Politics is more partisan than ever these days, but the opioid crisis might be one of the few issues where elected officials find common ground. This week, members of Congress exercised a bipartisan effort in both chambers to advance dozens of measures proposing various approaches to fighting the opioid epidemic.

A Senate committee voted unanimously to send the “Opioid Response Act of 2018” to the chamber floor for a vote. In the House of Representatives, an Energy and Commerce subcommittee advanced dozens of opioid crisis bills to the full committee. Some measures address the crisis by expanding treatment. Others aim to limit access to addictive pain-killing pills. House members hope to bring opioid bills to a vote in their chamber by Memorial Day.

In other news, a new CRISPR startup made its debut, doctors raised some questions about the benefit of new Parkinson’s drugs, and a proposed pharma mega merger inched closer to a deal. Let’s recap those stories and more in this week’s biotech roundup.


—Two experimental drugs from Acorda Therapeutics (NASDAQ: ACOR) and Sunovion Pharmaceuticals—both under FDA review, or close to it—could provide Parkinson’s disease patients with much-needed rescue therapies when their medications stop working. But experts Xconomy spoke with questioned how much additional benefit the drugs will offer compared to levodopa, and want to see if Parkinson’s patients, with their impaired movement, will actually be able to use the drugs when needed.

—The American Academy of Neurology’s annual meeting marked the latest forum for a few important updates on cutting-edge treatments for the rare genetic disease spinal muscular atrophy. AveXis (NASDAQ: AVXS), which could bring a gene therapy for SMA to market next year, gave people the first look from a key clinical study and updated a previous one. And Biogen (NASDAQ: BIIB), which brought the first approved SMA drug, nusinersen (Spinraza), to market, revealed the drug’s latest sales numbers and offered a look at an ongoing open-label extension study.

—Separately, Biogen will pay $1 billion to expand the alliance with Ionis Pharmaceuticals (NASDAQ: IONS) that led to nusinersen. The two companies will now try to develop RNA drugs for a variety of neurological diseases.

—Sage Therapeutics (NASDAQ: SAGE) has filed for FDA approval of brexanlone, which could become the first drug marketed specifically for women with postpartum depression.

—At a Congressional hearing, FDA commissioner Scott Gottlieb said that Nuplazid, a treatment for Parkinson’s psychosis the FDA approved in 2016 despite safety concerns, was under review. The drug could be linked to hundreds of deaths.


—Can CRISPR make disease testing faster and cheaper? Mammoth Biosciences, a spinout from the UC Berkeley lab of Jennifer Doudna talked up its plans to develop home kits for infectious disease and more. Researchers at the Broad Institute updated their own technology, called SHERLOCK, that pioneer Feng Zhang, virus hunter Pardis Sabeti, and others are tuning to use to track Zika, dengue, and other microbial threats.

—A rheumatoid arthritis drug developed by Eli Lilly (NYSE: LLY) won the recommendation of an FDA advisory panel, but only at the lower dose. If Lilly can’t bring the higher dose of baricitinib (Olumiant) to the market, analysts say its commercial prospects will be more limited.

—The FDA placed a partial hold on tazemetostat, the experimental lead drug of Cambridge, MA-based Epizyme (NASDAQ: EPZM), after a pediatric patient developed a secondary cancer.

—Takeda Pharmaceutical bumped up its cash and stock bid for rare diseases drug developer Shire (NASDAQ: SHPG) to $64 billion. Under takeover rules in the U.K., Takeda has until May 8 to make a binding offer, though that deadline could be extended.

—Former Sarepta Therapeutics (NASDAQ: SRPT) CEO Chris Garabedian spoke to a group of scientists and researchers at the University of Texas Health Science Center in San Antonio, looking for new projects to put into Xontogeny, his Boston startup accelerator. David Holley has more on Xontogeny and Garabedian’s talk at UT here.

—AstraZeneca (NYSE: AZN) reported that its immunotherapy combination, durvalumab (Imfinzi) and tremelimumab, failed a Phase 3 trial in lung cancer. In the ARCTIC study, the combo was tested against chemotherapy in non-small cell lung cancer patients who had failed two prior treatments and whose tumors express very little, if any, of the protein PD-L1.


Revolution Medicines raised $56 million in Series B financing as the Redwood City, CA, company sheds its earlier efforts in antifungal development and turns its focus to cancer drugs.

—Cedilla Therapeutics debuted this week with $56 million from Third Rock Ventures. The Cambridge, MA, biotech is developing drugs that target disease-causing proteins.

—Rare disease drug developer Rallybio launched with $37 million from 5am Ventures, Canaan Partners, and New Leaf Ventures.

—Two RNA interference drug developers, Alnylam Pharmaceuticals (NASDAQ: ALNY) and Dicerna Pharmaceuticals (NASDAQ: DRNA), settled a lawsuit in which Alnylam had charged Dicerna with stealing trade secrets. Dicerna agreed to pay Alnylam $2 million up front, 983,208 shares of Dicerna stock, and $13 million in payouts over the next four years. Here’s more from Reuters.

—Zymeworks (NYSE: ZYME) and Celgene (NASDAQ: CELG) amended a drug development pact from 2014. The revised agreement expands the number of drugs the partners could develop from eight to 10, and bumps up the potential payments to Zymeworks to as much as $1.64 billion.

—Corvidia Therapeutics, of Waltham, MA, raised a $60 million Series B round to advance a drug being developed for a genetic subset of people with chronic kidney disease.

Ben Fidler and Alex Lash contributed to this report.

Photo by Flick user Dave Winer via a Creative Commons license