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Bio Roundup: GOP Drama, Immunotherapy Setbacks, Pharma Revamps

Xconomy National — 

Two failed cancer studies this week provided a fresh reminder how little researchers and clinicians understand about immunotherapy’s vagaries: why it works for some people and not for others.

The results, in lung cancer and head-and neck cancer, add to recent failures in multiple myeloma and bladder cancer and splash cold water on a sector that, according to one measure, has been lukewarm for some time.

We’ve got the immunotherapy news, the latest on the GOP healthcare drama, and much more below. Let’s get right to it.

ON CAPITOL HILL…

—The Senate reopened debate to replace Obamacare with…no one was quite sure at the beginning of the week, but by early Friday morning had rejected three different plans. After shooting down a straight repeal of Obamacare as well as a replacement plan called the Better Care Reconciliation Act, the Senate voted against a so-called “skinny repeal,” that seemed to get rid of the individual mandate, abolish a requirement for employers with more than 50 workers to provide healthcare for five years, defund Planned Parenthood for one year, and more.

IMMUNOTHERAPY FAILURES

—AstraZeneca reported disappointing results from a closely watched 1,100-patient clinical trial called MYSTIC,  which tested a combination of two of its cancer immunotherapies in newly diagnosed lung cancer patients. The news affected the fortunes of several big immunotherapy players: shares of AstraZeneca and Bristol-Myers Squibb both fell, while Merck shares climbed. Separately, AstraZeneca and Merck inked a multi-billion dollar pact to co-develop  several of their cancer drugs.

—Merck had bad news of its own. A Phase 3 study of its pembrolizumab (Keytruda) in head-and-neck cancer failed to show a survival benefit over standard chemotherapy. The FDA approved pembrolizumab last year for head-and-neck cancer based on tumor shrinkage, but the approval required future evidence of patients living longer. Pembrolizumab is currently in two other head-and-neck cancer trials; it’s unclear if data from those studies could influence the FDA’s decision.

RARE DISEASE SUCCESSES

—-Biogen (NASDAQ: BIIB) reported $203 million in quarterly sales of the closely watched spinal muscular atrophy drug nusinersen (Spinraza), well beyond consensus estimates, but executives warned on a conference call that sales could slow near-term. The company also hinted at potential future deals for neuroscience assets, as well as job cuts in a plan to “streamline operations.”

—Following the best quarterly results to date for its Duchenne muscular dystrophy drug eteplirsen (Exondys 51) and a roughly 20 percent rise in shares, Sarepta Therapeutics (NASDAQ: SRPT) raised $325 million in a stock offering.

FDA STOPS AND STARTS

—Watertown, MA-based Tetraphase Pharmaceuticals (NASDAQ: TTPH) plans to seek FDA approval of its experimental antibiotic, eravacycline, after positive results from its latest Phase 3 study.

—Indianapolis-based Eli Lilly (NYSE: LLY) announced that a resubmitted FDA application for rheumatoid arthritis drug baricitinib would be delayed by at least 18 months. The FDA rejected the drug in April due to safety concerns, and the regulator now wants the company to run another trial.

DEAL OR NO DEAL

—Eli Lilly is paying $150 million up front for rights to NKTR-358, an early-stage compound from with San Francisco-based Nektar Therapeutics being developed for autoimmune disorders.

—Tesaro (NASDAQ: TSRO) agreed to license partial rights to its ovarian cancer drug niraparib (Zejula) to Takeda, a deal that pays the Waltham, MA, biotech $100 million up front and potentially $340 million overall.

—Effector Therapeutics of San Diego raised a $38.6 million Series C round to develop an experimental cancer immunotherapy.

—South San Francisco, CA-based Kezar Life Sciences raised a $50 million Series B round to continue developing a potential drug for autoimmune disorders currently in Phase 1 testing.

—Seattle’s Cyrus Biotechnology, a spinout from David Baker’s protein design lab at the University of Washington, raised an $8 million Series A round led by Trinity Ventures. Cyrus makes modeling software that biopharmas and others use to design protein-based drugs from scratch.

—Rodeo Therapeutics, the latest Seattle-based Accelerator Corp. startup, raised a $5.9 million Series A round to develop regenerative medicine therapies.

SETTLEMENTS & REVAMPS

—Summit, NJ-based Celgene (NASDAQ: CELG) paid $280 million to settle a fraud suit over tactics it allegedly used to promote two of its cancer drugs for unapproved uses. Here’s more in the New York Times.

–As part of a strategic restructuring, troubled Alexion Pharmaceuticals (NYSE: ALXN) ended partnerships with two Moderna Therapeutics, Blueprint Medicines (NASDAQ: BPMC), and Arbutus Biopharma (NASDAQ: ABUS).

—New GlaxoSmithKline (NYSE: GSK) CEO Emma Walmsley outlined a vast revamp that will see the British pharma dump more than 30 drug programs and potentially shed its rare disease business.

THIS WEEK IN CRISPR

—The University of California filed a court brief this week, the first detail from its appeal of a decision that in February gave the Broad Institute the upper hand in the long-running battle to determine who invented the breakthrough gene-editing technology CRISPR-Cas9.

—In other CRISPR news, MIT Technology Review reported a group of researchers in Oregon have become the first in the U.S. to try to use CRISPR-Cas9 to make genetically modified embryos.

Alex Lash and Frank Vinluan contributed to this report.

Photo courtesy of flickr user Gage Skidmore via a Creative Commons 2.0 license.