Bio Roundup: Trump, Gottlieb, Gene Therapy Prices, IPO Hopes & More

Xconomy National — 

Welcome to the new year. What will it bring? Hopefully a lot less hatred, greed, stupidity, and angst than 2017. But we’re not holding our breath.

We emerged from the winter break pondering several of the year’s burning biopharma questions, including the volatility of the U.S. president and what it means for the biopharmaceutical business, a frequent target of his scattershot rants. Two other big questions we raised immediately became topics of conversation this week, thanks to Spark Therapeutics, which set the price for its gene therapy—the first ever approved in the U.S.—at $850,000.

We also covered gene therapy development news and the typical pre-J.P. Morgan funding-palooza. Let’s get right to it, the first roundup of 2018. See you in San Francisco next week—or maybe not, seeing what our friends on the East Coast are dealing with. Bomb cyclone? Polar vortex? Does anyone care that the Bay Area had a 4.4 magnitude temblor this week? Anyone? Hello? Oh well. It’s roundup time.


—Donald Trump? Scott Gottlieb? Seema Verma? Congress? Which federal officials will have the most influence over the drug industry in 2018? We ask that question and six more for the new year, about digital health, the microbiome, cancer diagnostics, the opioid crisis, gene therapy costs, and patient input into drug development.

—Spark Therapeutics (NASDAQ: ONCE) said its gene therapy for an ultra-rare type of vision loss will cost $850,000 for the one-time dose—an injection in each eye. Upfront, it is the most expensive drug in America. (Other drugs taken multiple times rack up higher costs.) Spark is offering a relatively small rebate if its drug doesn’t work within 30 months, and it wants the government to allow for installment payments and deeper rebates for patients who aren’t helped. Those prospects are far from guaranteed.

—Even with its progress, gene therapy has plenty of biological and medical problems. (Not to mention the affordability problem. See above.) That leaves an opening for startups like Generation Bio and Stoke Therapeutics—each debuted this week—to provide new solutions to control dosing, delivery, and potentially expand gene therapy’s reach.

—Making good on a priority Commissioner Scott Gottlieb flagged during his nomination hearing, the FDA unveiled new regulatory plans for generic drugs. Gottlieb says the new rules should boost competition and lower drug prices.


—The IPO window was open for many life science companies in 2017. Four biotechs hoping to continue the trend filed the paperwork to go public before the close of the year. On the first business day of the 2018, four more biotechs unveiled financing that totaled nearly $200 million.

—In another sign of the fervor for messenger RNA therapeutics—an unproven technology meant to help the body churn out its own therapeutic proteins—Germany’s BioNTech raised a monster $270 million Series A round.

—Meanwhile, former aTyr Pharma CEO John Mendlein joined BioNTech rival Moderna Therapeutics as president of corporate and product strategy.

—The venture capital cash continued flowing, as San Diego firm Expansion Therapeutics nabbed $55 million to join the race to develop pills that alter the activity of RNA inside cells and change the course of disease.

—Quick funding hits: Cambridge, MA-based Elstar Therapeutics raised $39 million to advance preclinical cancer immunotherapies… Neurogastrx of Boston raised $45 million to bring a drug to treat gastroparesis into clinical trials… Cambridge-based Scholar Rock bagged $47 million to get to its first clinical trial, a test of an experimental treatment for spinal muscular atrophy… French firm Enterome raised €32 million ($38 million) to push ahead with clinical trials in gastrointestinal disease and brain cancer… Personal Genome Diagnostics of Baltimore, MD, nabbed $75 million, the latest indication of investor belief in the commercial viability of tumor profiling tests… Gossamer Bio of San Diego emerged with $100 million in initial financing to in-license drugs addressing various diseases.


—Takeda agreed to acquire Belgian partner TiGenix and its experimental Crohn’s disease drug darvadstrocel in a buyout valued at about $626 million.

—In a separate deal, Takeda will pay Denali Therapeutics (NASDAQ: DNLI) $155 million up front in cash and stock to co-develop up to three drugs for neurodegenerative diseases.

—Kevin Lo, a former Facebook (NASDAQ: FB) executive who recently joined San Diego diagnostics startup Genalyte, talked to Xconomy about the company’s plans to upend lab testing with a cloud-connected device developed to produce faster test results.

—The day after Christmas, Mallinckrodt (NYSE: MNK) said it would buy Rockville, MD-based Sucampo Pharmaceuticals (NASDAQ: SCMP) for $1.2 billion for its constipation drug lubirprostone (Amitiza) and other assets.

—Seattle’s Adaptive Biotechnologies is teaming with Microsoft (NASDAQ: MSFT) to develop a blood test for a wide range of diseases. Microsoft is making an undisclosed investment in privately-held Adaptive.

—While corporate America cheered the tax cuts signed into law late last year, the medical device industry is bracing for a tax hike. The 2.3 percent excise tax on medical devices—written into the Affordable Care Act to fund the legislation but suspended for the past two years— resumed on Jan. 1.

—Research Triangle Park, NC-based Viamet Pharmaceuticals, which has scuttled two IPO attempts to finance late-stage testing of its lead antifungal drug, reached a deal to be acquired by investment firm NovaQuest Capital Management.

—Women’s health company OvaScience (NASDAQ: OVAS) of Waltham, MA, said it would slash its headcount in half to continue work on its clinical programs without the need for additional financing.

Ben Fidler and Frank Vinluan contributed to this report.