Bio Roundup: ASCO Abstracts, Migraine Drug Prices & “Blueprint” Fallout

Xconomy National — 

There’s nothing abstract about a 20 percent jump in a company’s stock price. But yeah, that happened to Loxo Oncology when it released data previews for its American Society for Clinical Oncology presentations; the meeting starts in two weeks in Chicago.

Wednesday was ASCO abstract day, and we’ll round up the headliners below, all with caveats that these early peeks are not peer-reviewed and could be updated in a couple weeks. It’s even worth asking whether they’re worth writing about at all.

We also have the latest on the Trump administration’s finger-wagging attempts to rein in drug prices; a raft of biotech fundraising and dealmaking; and (with, ahem, apologies to Le Show) the apologies of the week from the Broad Institute’s Eric Lander and former Novartis CEO Joe Jimenez.


—Loxo Oncology (NASDAQ: LOXO) boosted investor belief that it will benefit from the FDA’s acceptance that cancer’s genetic profile, not its tissue of origin, is a key to drug development. Loxo’s ASCO abstract updated a Phase 1 study of LOXO-292, which attacks cancers with RET mutations. Shares were up nearly 20 percent at Thursday’s market close. Loxo’s success was a blow to Blueprint Medicines (NASDAQ: BPMC), also working on a RET-mutation drug that it updated last month. Its shares were down 7 percent.

—Jounce Therapeutics (NASDAQ: JNCE) was down nearly 35 percent on very preliminary Phase 1 data for its homegrown JTX-2011, testing it both on its own and combined with nivolumab (Opdivo). The news also dragged down shares of Celgene (NASDAQ: CELG), which paid $261 million in cash and stock to partner with Jounce on the drug in 2016.

—Another abstract bummer was Syndax Pharmaceuticals (NASDAQ: SNDX), which updated Phase 2 study data for its etinostat combined with pembrolizumab (Keytruda). Only 11 percent of 57 lung cancer patients showed a response to the combo, but Syndax said a subset of patients with a specific biomarker showed a better response rate of 29 percent (4 of 14). Investors were not convinced and sent shares down nearly 23 percent.

—Quick abstract hits: Nektar Therapeutics (NASDAQ: NKTR) slipped more than 7.5 percent because its NKTR-214, combined with nivolumab, showed lower response rates than previously reported… A Chinese research team is comparing the two approved CAR-T cell therapies head-to-head in patients with acute lymphoblastic leukemia and give the advantage to Novartis’s tisagenlecleucel (Kymriah).


— President Trump’s “blueprint” to lower prescription drug prices, unveiled last week, didn’t scare drug makers. In fact, investors boosted their shares as well as those of drug-buying middlemen. This week, the administration’s health officials added more detail. The FDA posted a “shame list” of companies that game the generic-drug system, with Celgene the top offender. And Health and Human Services secretary Alex Azar, a former Eli Lilly (NYSE: LLY) executive, threatened other measures, saying he’s “tired” of pharma’s excuses.

—The FDA approved Amgen’s (NASDAQ: AMGN) erenumab (Aimovig), the first of a new wave of drugs meant to reduce the frequency of migraines. Amgen priced the drug at $575 per month, or roughly $6,900 per patient, per year.

—In the runup to an FDA decision on erenumab, physicians and industry observers discussed with Xconomy the potential cost of the new migraine drugs, and who they might be best suited for.

—The FDA approved lofexidine (Lucemyra), the first non-opioid treatment for opioid withdrawal symptoms.

—The agency also criticized research firm IQVIA Holdings (NYSE: IQV) for faulty opioid data, saying the firm overestimated prescription fentanyl sales in the U.S.


—Broad Institute president and founder Eric Lander apologized Monday for a weekend birthday toast he gave to 90-year-old Nobel laureate James Watson, a co-discoverer of DNA’s double-helix structure. Watson has made a series of offensive remarks over the years, sometimes in the context of scientific talks. In his apology, Lander admitted having been the direct target of Watson’s anti-Semitism and called his views “abhorrent.”

—Turmoil for Novartis (NYSE: NVS) continued, with its top lawyer stepping down and taking blame for the $1.2 million it paid to Donald Trump’s lawyer Michael Cohen for healthcare consulting. Forbes interviewed former Novartis CEO Joe Jimenez, on whose watch the Cohen contract was signed.

—One man who’s never sorry: biotech mega-investor Bob Nelsen of Arch Venture Partners. Here’s Nelsen’s recent conversation with Xconomy editor in chief Bob Buderi.


—Until recently, IDO inhibitors were seen as key to expanding the reach of cancer immunotherapy. But the field has collapsed. The latest: Roche has officially terminated a wide-ranging 2014 deal with NewLink Genetics (NASDAQ: NLNK) to co-develop several IDO blockers.

—Pfizer (NYSE: PFE) finally won approval for its biosimilar (read: nearly generic) version of the longtime blockbuster anemia drug Epogen.

—San Diego’s aTyr Pharma (NASDAQ: LIFE) will cut 30 percent of its workforce and focus on ATYR1923, an early-stage drug candidate for interstitial lung disease.

—Eleven Biotherapeutics has changed its name to Sesen Bio (NASDAQ: SESN). Eleven went public in 2013 on the promise of a dry eye disease drug that later failed two clinical trials. It’s now developing a bladder cancer drug in Phase 3.

—Worrisome safety signals have caused Johnson & Johnson to stop testing of an experimental Alzheimer’s drug in late-stage clinical trials. The news marks the latest setback for a class of Alzheimer’s drugs known as BACE inhibitors.


—Beam Therapeutics of Cambridge, MA, launched with $87 million in new venture cash to pursue medicines based on base editing, a new version of gene modification based on the CRISPR system that Beam founders David Liu, Keith Joung, and Feng Zhang have helped create.

—FogPharma’s $66 million Series B round seems like standard fare as biotechs rake in tons of cash. But a closer look shows academic-turned-executive Greg Verdine has hand-picked investors to let him to keep strategic control of the growing startup.

—Eli Lilly shelled out $110 million to acquire AurKa Pharma, whose cancer drug program was licensed from Lilly two years ago.

—Illumina (NASDAQ: ILMN) paid $100 million for San Diego startup Edico Genome, whose software and microchips help DNA sequencers map genetic information more quickly.

—Celsius Therapeutics launched with a $65 million Series A round from Third Rock Ventures, GV, and others. The Cambridge, MA, company is using single-cell genomics to discover new drug targets for autoimmune disease and cancer.

—Accent Therapeutics unveiled $40 million in financing that the Cambridge company will use to discover drugs meant to fine-tune the activity of a cell’s genes.

—Cambridge, MA-based EIP Pharma raised a $20.5 million Series B to push an experimental Alzheimer’s disease drug licensed from Vertex Pharmaceuticals (NASDAQ: VRTX) through a Phase 2b trial.

—New York-based MeriaGTx filed for an IPO to continue early-stage clinical testing of its gene therapies for rare eye diseases.

Ben Fidler and Frank Vinluan contributed to this report.