Bio Roundup: Alzheimer’s Puzzle, ESMO Assessed, Drug Prices & More

Xconomy National — 

In the biotech investment and research communities this week, much of the attention was turned to Europe.

Munich, Germany, hosted the annual conference of the European Society for Medical Oncology, where cancer drug developers unveiled the latest clinical data for experimental immunotherapies and drugs that treat the disease based on a tumor’s genetic signature. Also this week, the Clinical Trials on Alzheimer’s Disease conference took place in Barcelona, Spain. As with another Alzheimer’s meeting back in July, much of the interest there focused on data from a drug developed by Japanese pharmaceutical company Eisai and partner Biogen that has left some scientists and investors puzzled.

In other news, President Trump announced a new drug pricing initiative, a big pharma player made a surprising bet, a big biotech announced a price cut, and several new startups formed with drugs shelved by larger companies. Those headlines and more below.


Eisai presented more data from its BAN2401 Alzheimer’s program, which it co-owns with Biogen (NASDAQ: BIIB). Instead of clearing up controversy over its data release in July, the fresh information only raised more questions.

—Biogen also disclosed that two Phase 2 clinical trials failed: one testing experimental drug for lupus, and another studying a potential treatment for sciatic nerve pain. The lupus drug is part of an alliance with UCB, while the pain drug, still being developed for trigeminal neuralgia, comes from Biogen’s $200 million buyout of Convergence Pharmaceuticals in 2015.

—President Trump announced a plan to lower the prices of some drugs covered by Medicare, a proposal that was greeted coolly by PhRMA, the pharmaceutical industry trade group.

—The European Society of Medical Oncology’s annual meeting came and went this past week, and Xconomy rounded up the key headlines, which included immunotherapy advances in breast and lung cancer and a brewing battle among two developers aiming for “tissue agnostic” drug approvals.

——Amgen (NASDAQ: AMGN) announced a 60 percent cut to the list price of its cholesterol lowering drug evolocumab (Repatha), which has struggled commercially amid pushback from insurers. The move comes months after Regeneron vowed to cut the net price—as in, after discounts and rebates to payers—of its rival drug alirocumab (Praluent) if it could get concessions from insurers.

—Pfizer (NYSE: PFE) and Eli Lilly (NYSE: LLY) released encouraging early Phase 3 results showing that their experimental drug tanezumab reduced pain in patients with knee or hip problems. The companies are competing against partners Regeneron Pharmaceuticals (NASDAQ: REGN) and Teva Pharmaceutical (NYSE: TEVA) to win the first FDA approval of a non-opioid treatment for osteoarthritis pain.

—Bristol-Myers Squibb (NYSE: BMY) disclosed another setback to its immunotherapy strategy in lung cancer, as the FDA extended, by three months, a review of a two-drug regimen the company has been touting for the disease.

—Shares of Mirati Therapeutics (NASDAQ: MRTX) fell 15 percent after the company disclosed data from a Phase 2 study testing its experimental cancer drug, sitravatinib, in tandem with Bristol’s immunotherapy nivolumab in lung cancer patients.

—Synergy Pharmaceuticals (NASDAQ: SGYP) shares plunged 66 percent after the company said it hasn’t found a buyer or come to a deal with its lender, CRG Servicing, to avoid defaulting on its debt. Synergy, which sells a drug for constipation called plecanatide (Trulance), could file for bankruptcy if it can’t reach a new funding deal.

—Less than a year after Stat reported on sexual misconduct allegations against healthcare investor Sam Isaly, Isaly filed a defamation lawsuit against Stat’s owner, the Boston Globe, and the reporter who wrote the story. Isaly left OrbiMed Advisors, the firm he founded, in April. Here’s more from Bloomberg.


—Stoke Therapeutics, a startup developing RNA drugs and led by former Sarepta Therapeutics (NASDAQ: SRPT) CEO Ed Kaye, raised a $90 million Series B and could go public next year. The company is developing what Kaye terms the “Spinraza of genetic epilepsy,” a reference to the spinal muscular atrophy drug invented by Stoke’s scientific founder Adrian Krainer.

—Synthego of Menlo Park, CA, raised $110 million in a Series C round to expand its gene-editing toolkit business, particularly its “Engineered Cells” product, in which Synthego uses CRISPR to produce custom-edited cells for researchers. Matt Porteus of Stanford University, a cofounder of CRISPR Therapeutics, joined the Synthego advisory board.

—China based Innovent Biologics raised $421 million in its IPO, the biggest of the year in Hong Kong and the fourth under new rules implemented this year meant to to woo more early-stage drug developers to the exchange.


—Pharma giant AbbVie (NYSE: ABBV) surprisingly bought a group of cystic fibrosis drugs from Galapagos NV for $45 million just as those treatments posted data that some Wall Street analysts deemed disappointing. AbbVie aims to challenge CF market leader Vertex Pharmaceuticals (NASDAQ: VRTX), which has three drugs for the lung disease already on the market and more potentially on the way.

—Alexion Pharmaceuticals (NASDAQ: ALXN) paid Dicerna Pharmaceuticals (NASDAQ: DRNA) $22 million up front and made a $15 million equity investment in the company. The two will co-develop RNA interference drugs that impact a part of the immune system called the complement system.

—Genoptix, a Carlsbad, CA-based cancer testing laboratory, was acquired by NeoGenomics in a $125 million cash and stock deal.

— AstraZeneca (NYSE: AZN) added more cancer immunotherapies to its pipeline with a deal to acquire a group of drugs from France-based Innate Pharma. In exchange, Innate gets U.S. and European rights to an AstraZeneca drug that was recently approved by the FDA as a treatment for hairy cell leukemia.


—San Francisco-based 89Bio launched with $60 million in funding and a lead drug candidate for non-alcoholic steatohepatitis acquired from Teva.

Qpex Biopharma emerged with $33 million in financing and a preclinical anti-infectives portfolio acquired from The Medicines Company (NASDAQ: MDCO).

—Bain Capital put $350 million into a new startup, Cerevel Therapeutics, that will develop neuroscience drugs that Pfizer shelved when it stopped investing in the field earlier this year. Bain and Pfizer similarly teamed to funnel stalled drugs into another startup, Springworks Therapeutics, last year. Here’s more from CNBC.


—Don’t miss this look back at our latest biotech event, “Boston’s Life Science Disruptors,” which included some candid stories and anecdotes from the founders of Beam Therapeutics, Arrakis Therapeutics, and Celsius Therapeutics.

Ben Fidler and Alex Lash contributed to this report.

Photo by Flickr user xiquinhosilva via a Creative Commons license