Bio Roundup: DIY Biohacking, PCSK9 Data, Solid Setback & More

Xconomy National — 

Drug prices are top of mind for many patients. Some of them have to cut back on basic needs just to pay for treatments that seem to rise in price each year. That made a recent announcement by Regeneron Pharmaceuticals particularly unusual.

The Tarrytown, NY, drugmaker plans to cut the price of its cholesterol-lowering drug alirocumab (Praluent) if it can get concessions from payers. Regeneron made the move after data from a heart study suggested its drug might reduce the risk of death in those who have recently suffered a heart attack or stroke, and are at high risk of having another one.

Alirocumab is part of class of cholesterol-lowering drugs whose high list prices—around $14,000 per year—have led to significant pushback from payers. Regeneron hopes to change things by lowering its drug’s price to align with an assessment from an independent nonprofit organization, the Institute for Clinical and Economic Review. It’s the second time Regeneron has taken ICER’s input into account with a pricing decision, but these instances are outliers. It remains to be seen whether the gambit will work and drive adoption of alirocumab, or whether other drugmakers will follow suit.

In other news this week, a leading “biohacker” explained his approach to do-it-yourself biotech, the FDA stopped a study of a gene therapy for Duchenne muscular dystrophy, and investors kept on pouring money into immuno-oncology. Let’s get to the rest of the week’s news.


—Regeneron Pharmaceuticals (NASDAQ: REGN) and partner Sanofi (NYSE: SNY) unveiled data from their massive cardiovascular study Odyssey Outcomes and unexpectedly cut the price of their cholesterol-lowering medicine alirocumab (Praluent). Regeneron reported the drug cut the risk of death for some patients. The results came with caveats, but cardiologists told Xconomy that insurers might be more willing to cover the drug.

—Xconomy’s Corie Lok reported that a few patient advocate groups are wading into the fight over high drug prices, even at the risk of upsetting pharma companies that donate to their cause.

—Bay Area biohacker Josiah Zayner has attracted attention with his self-experimentation, including a public injection of CRISPR-Cas9. Xconomy’s Alex Lash profiled Zayner, who is rethinking his own role—in public, as usual—on the do-it-yourself biotech frontier.

—The Securities and Exchange Commission revealed that it charged Theranos and its two top executives with “elaborate, years-long fraud.” CEO Elizabeth Holmes has settled the charges in part by paying a $500,000 fine and surrendering her majority equity stake. Former president Ramesh Balwani will go to trial.

—The saga of former pharmaceuticals executive Martin Shkreli concluded with a seven-year prison sentence handed down for securities fraud.

—The FDA suspended a clinical test of a gene therapy from Solid Biosciences (NASDAQ: SLDB), the latest sign of trouble for the firm. The company reported that the first Duchenne muscular dystrophy patient dosed in the study was hospitalized for several days. Solid shares plummeted 64 percent.


—A year after GlaxoSmithKline (NYSE: GSK) bailed on a partnership with Ionis Pharmaceuticals (NASDAQ: IONS) for a transthyretin amyloidosis drug, Ionis sent the drug, inotersen, to spinout Akcea Therapeutics (NASDAQ: AKCA). Like a rival drug, patisiran, inotersen is under FDA review.

—Biogen (NASDAQ: BIIB) paid $150 million up front to acquire a Pfizer (NYSE: PFE) schizophrenia drug ready to start Phase 2 studies.

—Arcus Biosciences (NYSE: RCUS) raised $120 million in an initial public stock offering. Proceeds will finance clinical testing of the Hayward, CA, company’s two lead cancer immunotherapies.

Crinetics closed a $63.5 million round of financing to support clinical testing of a treatment for acromegaly, a rare hormone disorder.

—Humacyte closed a $75 million Series C round that the Research Triangle Park, NC, company will use to continue Phase 3 studies of its bioengineered blood vessels and to complete a bioprocessing system for manufacturing them.

—Flagship Pioneering launched Cambridge, MA-based Foghorn Therapeutics with $50 million in funding. Forbes has more on the startup, which is founded by 32-year-old Dana-Farber Cancer Institute professor Cigall Kodach and aims to develop drugs that control gene expression in cells.

—Orexigen Therapeutics (NASDAQ: OREX) filed for bankruptcy with plans to sell itself, and its weight loss pill Contrave, at an open auction in May. Orexigen was about to breach a debt deal because 2017 sales of Contrave didn’t reach $100 million.

—Ideaya Biosciences of South San Francisco, CA, raised $94 million to bring its cancer drugs into human testing. The Series B round included “crossover investors,” entities that invest in both public and private companies, laying the groundwork for a potential IPO.


—In a surprise, the House of Representatives rejected a bill that would give terminally ill patients more access to experimental drugs. The so-called right-to-try movement is staunchly opposed by patient advocate groups and the drug industry, which lobbied hard against the bill.

— Norbert Bischofberger, chief medical officer of Gilead Sciences (NASDAQ: GILD), is transitioning to retirement after nearly 30 years with the Foster City, CA, company. Gilead says he’ll step away from his executive post in April and leave the company in July.

—Sanofi chose not to gain rights to an experimental Alnylam RNA interference medicine, lumisiran, for the rare kidney disease primary hyperoxaluria type 1. The two streamlined a wide-ranging alliance on a variety of RNA interference drugs in January.

—Sarepta Therapeutics (NASDAQ: SRPT) will file for accelerated approval of its second Duchenne muscular dystrophy drug, golodirsen, by the end of the year. The FDA approved Sarepta’s first drug, eteplirsen (Exondys 51) even though it hadn’t yet proven to benefit patients.

—Intra-Cellular Therapies (NASDAQ: ITCI) said after a meeting with the FDA the two have agreed “on the content and timing” of an approval filing for experimental schizophrenia drug lumateperone, which has had mixed results in clinical testing. Intra-Cellular should complete the application by mid-year.

—The FDA halted a study testing a combination of Advaxis’ (NASDAQ: ADXS) experimental cancer vaccine axalimogene filolisbac and AstraZeneca’s immunotherapy durvalumab (Imfinzi) after a patient died of respiratory failure.

—Auris Medical’s (NASDAQ: EARS) tinnitus drug Keyzilen failed a Phase 3 study, the second late-stage failure for the Switzerland-based company’s drug.

—There’s a new enzyme for the CRISPR-Cas13 gene-editing system. Arbor Biotechnologies published its discovery of Cas13d in the journal Molecular Cell, saying the enzyme could be used in RNA manipulation and highly sensitive diagnostics.

—Alexion Pharmaceuticals (NASDAQ: ALXN) announced positive preliminary results from a Phase 3 study testing its drug ALXN210 in paroxysmal nocturnal hemoglobinuria, a rare blood disorder.

Ben Fidler and Alex Lash contributed to this report.

Photo by Flickr user Brandon Giesbrecht via a Creative Commons license.