Bio Roundup: Wilson’s Warning, Amazon’s Health Biz, Moderna Cash & More

Xconomy National — 

The State of the Union address was…. long. At one hour and 20 minutes, President Trump fell about 9 minutes shy of the high mark. But those who stayed tuned in heard comments about several health policy matters.

The president credited the FDA for a record number of generic drug approvals in 2017, which PwC notes is part of a trend that began three years ago. President Trump also said he supported “right to try,” allowing terminally ill patients try experimental drugs. The FDA’s expanded access program already lets patients receive drugs that have not yet been approved. If the president wants a stronger federal policy, he’ll have to overcome resistance from his own FDA commissioner. Scott Gottlieb has pointed to shortcomings of “right to try” legislation proposed in Congress.

In non-State of the Union news this week, a biotech company raised a whopping $500 million, gene therapy leader James Wilson shook up the sector, and Amazon revealed its foray into healthcare. Alexa, what else happened this week?


—In the State of the Union address, President Trump once again ramped up his rhetoric against high drug prices, calling price cuts “one of my greatest priorities… Prices will come down.” Pharma stocks fell, even though Trump’s previous pledges, which date back to the presidential campaign, have come to naught.

—Brenda Fitzgerald, the director of the Centers for Disease Control and Prevention, stepped down this week after Politico reported that she had bought tobacco stocks and had other financial conflicts while in office. Fitzgerald said her financial manager made the purchases without her knowledge.

—The Securities and Exchange Commission has sued “one or possibly many” unknown stock traders due to “highly suspicious trading” just ahead of French drug maker Sanofi’s recent $11.6 billion buyout of Bioverativ (NASDAQ: BIVV). The SEC said the traders—who made their purchases through foreign brokerage firms—gained $4.9 million in profits.


—Secretive Moderna Therapeutics unveiled a $500 million round of financing, yet another massive funding haul that boosts the Cambridge, MA, company’s balance sheet to $1.4 billion.

—Amazon (NASDAQ: AMZN), Berkshire Hathaway, and JPMorgan Chase (NYSE: JPM) said they would form a joint venture to provide their employees with better healthcare. The partners said the new firm’s initial focus would be “technology solutions,” but there were few other details.

—Continuing a string of buyouts, Sanofi (NYSE: SNYpaid $4.8 billion for Belgian biotech Ablynx, which had previously rejected a bid from Novo Nordisk. Ablynx is developing antibody drugs for rare diseases such as acquired thrombotic thrombocytopenic purpura.

BridgeBio Pharma launched its latest startup, QED Therapeutics, around a drug Novartis (NYSE: NVS) has previously tested as a treatment for bile duct cancer. Palo Alto, CA-based QED plans to continue testing that drug for cancer, but it will also develop it as a treatment for a rare disorder that leads to dwarfism.

—Seattle Genetics (NASDAQ: SGEN) agreed to buy Cascadian Therapeutics (NASDAQ: CASC) and its experimental cancer drug tucatinib for $614 million. It’s the end of a long, rocky road for Cascadian, once known as Biomira and later Oncothyreon.

San Francisco startup Engine Biosciences raised $10 million in seed funding to develop an artificial intelligence-based drug discovery technology.

—LRVHealth, formerly known as Long River Ventures, said it wants to raise $100 million for its fourth investment fund.

—Partner Therapeutics, a new Boston startup formed by former Merrimack Pharmaceuticals CEO Robert Mulroy and ex-Seragon Pharmaceuticals chief medical officer Debasish Roychowdhury, raised $60 million and bought an immune system boosting drug from Sanofi.

Section 32 founder Bill Maris talked to Xconomy about his venture capital firm’s strategy for investing in companies focused on the convergence of information technology and life sciences.


—James Wilson and University of Pennsylvania colleagues published a paper with troubling preclinical results from high doses of a certain type of gene therapy delivered into the bloodstream. The paper raised questions about several programs that are in or not far from human trials. Xconomy spoke with Wilson, a pioneer in the gene therapy field, and several experts about the paper’s implications.

—Cambridge-based Avrobio nabbed $60 million in Series B funding to finance Phase 2 studies of its gene therapy for Fabry disease.

—Voyager Therapeutics (NASDAQ: VYGR) is looking for a new CEO. Current chief executive Steven Paul plans to move to a new role as executive science advisor to the Cambridge-based gene therapy developer. Paul will keep his seat on Voyager’s board.


Sunovion Pharmaceuticals touted positive results from a Phase 3 study of experimental Parkinson’s disease drug APL-130277, setting up a possible commercial battle with Acorda Therapeutics (NASDAQ: ACOR).

—Vertex Pharmaceuticals (NASDAQ: VRTX) disclosed which “triple combination” cystic fibrosis regimens it has chosen for Phase 3 studies. If successful, these regimens could slow the progression of CF for up to 90 percent of patients, perhaps twice the number Vertex medicines are able to treat today. Here’s more from STAT.

—The FDA rejected an inhalable version of antibiotic ciprofloxacin that Aradigm (NASDAQ: ARDM) developed to treat chronic lung infections. The regulator told the Hayward, CA, company it needs to conduct another Phase 3 study before reapplying.

—The agency is also set to begin reviewing Alnylam Pharmaceutials’s (NASDAQ: ALNY) patisiran, which could become the first-ever marketed RNA interference medicine. The FDA will decide on the drug by Aug. 11.

—Sage Therapeutics (NASDAQ: SAGE) touted positive early-stage results from experimental SAGE-217 for insomnia. The results are noteworthy as Sage is developing SAGE-217 for a variety of neurological disorders, among them major depressive disorder and Parkinson’s disease.

—Bellicum Therapeutics (NASDAQ: BLCM) said its most advanced drug candidate has been put on hold by the FDA after three participants suffered brain injuries in a clinical study.

—In a cash-saving move, San Diego biotech Vical (NASDAQ: VICL) announced a corporate restructuring that will cut its staff by 54 percent. The shakeup follows the Phase 3 failure of a vaccine for cytomegalovirus, which Vical was testing in partnership with Japan-based Astellas Pharma.

Ben Fidler and Alex Lash contributed to this report.

Image by Flickr user Kevin Dooley via a Creative Commons license