Bio Roundup: Clinical Shirkers, AMD Worriers, ASH Fallers & More

Xconomy National — 

Our previous roundup came out last week before news broke that Susan Lindquist, a highly decorated and thoughtful researcher into the inner workings of proteins, passed away. She was 67.

First at the University of Chicago and then MIT’s Whitehead Institute, Lindquist helped biologists see that all kinds of organisms—including ourselves—share errors of protein folding and can shed light onto human disease. She pursued many inquiries through experiments with yeast, what she called a “unified principle of life.” One of her most recent projects was a biotech startup in concert with industry veteran Tony Coles (pictured with Lindquist, above). They aimed to put yeast experiments at the center of drug discovery for neurological diseases.

Our condolences go out to Lindquist’s friends and family. The next time you open a beer, a bottle of wine, or break bread, salute the humble yeast that helped make it, and give a thought to Lindquist, who saw a world of possibility in that yeast. On to the roundup…


—Drugs that treat a common form of blindness in the elderly, known as age-related macular degeneration, aren’t just big business. Their high cost has also put eye doctors under the spotlight of insurers and Medicare, which is why the prospect of a new combination therapy from Ophthotech (NASDAQ: OPHT) has many of them on edge.

—California’s Proposition 61, which aims to control pharmaceutical spending, hasn’t had quite the same polling attention as the presidential race. But a new poll this week shows support has dropped sharply since the summer as drug companies have poured near-record amounts into an anti-61 campaign.

—Boston startup creator Third Rock Ventures closed a $616 million fund, its largest to date, and also made former Eleven Biotherapeutics CEO Abbie Celniker the second female partner in its 10-year history. Former Cubist CEO Mike Bonney, who was named a partner in January, is no longer involved in day to day operations, co-founder Bob Tepper told Xconomy.


—A new database from the University of Oxford exposes the industry, government, and academic researchers who aren’t publishing their clinical trial results. Since 2006, 45 percent of all trials registered on the U.S. site have been completed without publishing results. The worst offender is Sanofi, with two-thirds of its trials missing results. At the other end of the spectrum is Shire, with a perfect 96-for-96 record. Other academic groups have also built databases and scoring systems around trial data transparency.

—Flatiron Health and Foundation Medicine (NASDAQ: FMI) have unveiled a new database that holds the medical records and tumor DNA profiles of 20,000 cancer patients and counting. The firms will sell the data, stripped of patient identities, to drug companies looking for insights as they develop new cancer drugs or plan clinical trials.

—The Mayo Clinic of Rochester, MN, reported that a misdiagnosis of a heart problem called long QT syndrome led to an unnecessary implant for a young boy. He and his family received genetic tests after the boy’s brother died in his sleep. The false positives came from a misreading of the test results. The Mayo Clinic cardiologist behind the report published a study earlier this year about long QT misdiagnosis leading to improper defibrillator implants.

—Three New York research institutions, two venture firms, and Japanese pharma Takeda joined to form Bridge Medicines, an initiative to take drug projects from New York’s Tri-Institutional Therapeutics Discovery Institute and funnel them into New York-based biotech startups. Given the city’s dearth of lab space, however, it’s unclear where those startups will go.


—The new wave of so-called PCSK9 drugs to treat high cholesterol, once expected to flood the market, have barely caused a ripple. One latecomer, bococizumab, won’t even reach approval. Its owner Pfizer (NYSE: PFE) shelved it this week, citing poor Phase 3 data and pricing pressures on the two approved PCSK9 drugs. Pfizer also reported worse-than-expected earnings.

—Last Friday, development partners Regeneron Pharmaceuticals (NASDAQ: REGN) and Sanofi (NYSE: SNY) said the FDA delayed its approval decision on their experimental immune-disease treatment sarilumab because of manufacturing problems. The companies were expecting a decision by October 30.

—Doctors testing a combination of two immunotherapy checkpoint inhibitors, nivolumab (Opdivo) and ipilimumab (Yervoy), reported in the New England Journal of Medicine that two melanoma patients in the trial died of heart problems. The drugs unleashed the patients’ immune systems to fight their cancer, but the doctors suspect their immune cells attacked the heart, as well.

—Shares of antibiotic maker Cempra (NASDAQ: CEMP) were crushed this week after FDA staff raised concerns about Cempra’s pneumonia treatment solithromycin (Solithera) causing liver damage. The firm is going before a panel of FDA advisors Friday.

—In the latest twist in the controversial approval saga of Sarepta Therapeutics’s (NASDAQ: SRPT) Duchenne muscular dystrophy drug eteplirsen (Exondys 51), the FDA released a slew of internal documents and e-mails regarding its review of the treatment. The documents shed even more light on the scientific struggle within the agency over eteplirsen and the power play by top drug evaluator Janet Woodcock, whom, one of her FDA rivals on the matter claimed, “provided no cogent rationale” for why Sarepta’s data was “reasonably likely to predict clinical benefit.”


—The American Society of Hematology released data abstracts ahead of its annual meeting next month. One notable abstract divulged new gene therapy data from Bluebird Bio (NASDAQ: BLUE) that show both the potential and the limitations of the therapy for beta-thalassemia and sickle cell disease, and shed light on the company’s recent upgrade to its manufacturing technology.

—Shares of Spark Therapeutics (NASDAQ: ONCE) fell 13 percent after the company disclosed in its ASH abstract that one hemophilia patient receiving its experimental gene therapy required steroids to stifle an immune reaction to the treatment. Immune reactions have occurred with other hemophilia gene therapies without serious problems, but it was a first for Spark.

—Several other biotechs released ASH abstract data, and the net effect was a drop in most stocks. The Nasdaq Biotechnology Index (NASDAQ: IBB) fell about two percent Thursday.


—Nimbus Therapeutics of Cambridge, MA, earned a $200 million milestone from Gilead Sciences (NASDAQ: GILD), which bought rights to Nimbus’s early-stage treatment for NASH, a type of fatty liver disease, in April. Nimbus could earn $600 million more if all goes well. The companies did not say what triggered this week’s milestone payment.

—A few months after AbbVie ended a partnership with Infinity Pharmaceuticals on blood cancer drug duvelisib, Boston-based Verastem (NASDAQ: VSTM) grabbed rights to that drug without paying a cent up front and promising only $28 million in future milestones. The tiny deal terms reflect both duvelisib’s struggles and the declining interest in so-called PI3 kinase inhibitors, once a hot commodity in the cancer drug field.

—Boulder, CO-based Miragen Therapeutics will go public via a reverse merger with diagnostics developer Signal Genetics (NASDAQ: SGNL). Miragen’s investors will put $40 million into the company in company alongside the deal.

—Hampton, NJ, and Needham, MA-based Celldex Therapeutics (NASDAQ: CLDX) paid $62.5 million upfront, all in stock, for privately held Kolltan Pharmaceuticals.

Ben Fidler contributed to this report

Photo of Tony Coles and Sue Lindquist at Xconomy’s “What’s Hot In Boston Biotech” event, April 2015. Courtesy of Keith Spiro Photography.