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Bio Roundup: Travel Ban Block, Gilead Shock, Dean Kamen Talk & More

Xconomy National — 

The biotech community pushed back against President Donald Trump’s order to block travelers and refugees this week, and not much later, a federal court did, too. Patient advocates have scored big wins with recent approvals of rare disease treatments, but they’re not as happy with insurers’ coverage decisions or with Trump’s promises of massive FDA deregulation. All this, plus heart-stopping patent fights, drug-price intrigue, a regenerative chat with inventor Dean Kamen, and more. Let’s get to the roundup.

BAN IN THE U.S.A.

—A federal appeals court upheld a freeze of the Trump administration’s temporary ban on travelers from seven Muslim-majority countries and all refugees. The question of the ban’s legality will likely be decided by the Supreme Court, but the administration as of late Thursday had not announced an appeal.

—165 U.S. biotech leaders signed a letter published in Nature Biotechnology voicing “deep concern and opposition” to the travel ban, claiming it would cause significant damage to the nation’s life sciences sector.

—Xconomy also heard from biotech CEOs in Louisville, KY, and San Diego about the travel ban. Both are immigrants to the U.S., and they discussed their personal views as well as the ban’s potential effect on their businesses.

AROUND OUR NETWORK

—Patient advocates have pushed for faster, more flexible drug reviews from the FDA, and they’ve tallied recent successes. But Trump’s promises of deep deregulation at the agency don’t sit as well with several advocates Xconomy interviewed this week.

—Xconomy’s Jeff Buchanan spoke with entrepreneur and inventor Dean Kamen about leading the recently announced Advanced Regenerative Manufacturing Institute, which has gotten some $294 million to advance regenerative medicine and tissue engineering.

—Ardsley, NY-based Acorda Therapeutics (NASDAQ: ACOR) said its CVT-301, an experimental, inhalable form of the Parkinson’s disease drug levodopa, succeeded in a Phase 3 trial. Acorda could file for FDA approval this year.

—Humana ((NYSE: HMN) became the latest major U.S. insurer to place limits on coverage for Biogen’s (NASDAQ: BIIB) nusinersen (Spinraza), the first-ever approved spinal muscular atrophy drug. Like its rival Anthem, Humana deemed nusinersen “medically necessary” only for patients with type 1 SMA and instituted a pay-for-performance model.

MORE FROM THE DRUG-PRICE FRONT

—Once public enemy No. 1 for the price tag of its hepatitis C drug sofosbuvir (Sovaldi), Gilead Sciences (NASDAQ: GILD) has seen its hep C business and its share price tumble, accentuated this week by lower-than-expected projections as the pool of new patients shrinks and competition eats away prices. The slowdown prompted The Street’s Adam Feuerstein to wonder if curing dangerous diseases, while great for patients, might be bad for business.

—Kaleo gained attention last year for bringing to market an alternative to Mylan’s EpiPen injector and its controversial price tag. But Kaleo is now under fire from U.S. senators for its own price hike of an emergency injection that first responders give to opioid overdose victims.

—Express Scripts (NYSE: ESRX), a top pharmacy benefit manager (PBM)—a negotiator of drug prices for employers and health plans—touted its own role in keeping drug costs down. It was the latest salvo in the fight over who’s more to blame for high drug prices—PBMs like Express Scripts and their behind-the-scenes rebates, or drug companies who set high prices.

—The punch-up between PBMs and drug makers is important because of the threat of the Trump administration giving Medicare its own drug-price negotiating power. Asked to clarify comments Trump made last week to pharma executives, White House spokesman Sean Spicer said this week the president is committed to Medicare negotiation.

KEEP ON TRUMPIN’

—The man who will oversee the administration’s Medicare plans, whatever they turn out to be, is new Health and Human Services secretary Tom Price. Price was confirmed by the Senate late Thursday despite fresh revelations this week about his conflicts of interest related to his healthcare stock trading as a member of Congress.

—After that meeting with Trump, Eli Lilly (NYSE: LLY) CEO David Ricks was bullish on job creation. This week, Lilly said it would cut about three percent of its 7,000 R&D staff. The cuts come on top of nearly 500 sales jobs Lilly shed in the wake of last fall’s failure of the Alzheimer’s drug solanezumab.

STUDIES, FAILURES, AND APPROVALS

—Amgen (NASDAQ: AMGN) won FDA approval for etelcalcetide (Parsabiv), a treatment for secondary hyperparathyroidism, which affects chronic kidney disease patients.

—Idalopirdine, an experimental Alzheimer’s drug from Danish firm Lundbeck, failed two Phase 3 trials, adding yet another late-stage Alzheimer’s treatment to the scrap heap.

—French firm Cellectis (NASDAQ: CLLS) got a green light to start the first-ever trial of an “off the shelf” CAR-T cell therapy in the U.S., meaning it would use cells sourced from donors instead of a patient’s own cells. Cellectis said it would begin the trial for two types of blood cancer by mid-2017.

—Marathon Pharmaceuticals of Northbrook, IL, won FDA approval of deflazacort (Emflaza) for Duchenne muscular dystrophy. Deflazacort, a steroid, is now the second therapy approved for Duchenne in the U.S. Unlike Sarepta Therapeutics’s (NASDAQ: SRPT) eteplirsen (Exondys 51), deflazacort is approved for all DMD patients, not just a subset. Already approved in Canada and Europe for other uses, deflazacort will likely compete with the steroid prednisone, already used off-label to slow muscle damage in Duchenne patients. Marathon reportedly priced the drug at $89,000 per year.

IN OTHER NEWS

—An appeals court allowed Regeneron Pharmaceuticals (NASDAQ: REGN) and partner Sanofi (NYSE: SNY) to keep selling their anti-cholesterol drug alirocumab (Praluent) while the court considers a recent patent ruling against Regeneron/Sanofi and in favor of Amgen, which sells the rival drug evolocumab (Repatha). A decision is expected this year.

—Reuters reported that that Waltham, MA-based Tesaro (NASDAQ: TSRO), which could win approval this year of a type of cancer drug called a “PARP” blocker, is being courted by “several drugmakers” and is weighing its options with investment banks.

—Also according to Reuters, a former Merrimack Pharmaceuticals “(NASDAQ: MACK) employee was arrested on charges of insider trading he allegedly carried out with an ex-employee of another biotech firm, Akebia Therapeutics (NASDSAQ: AKBA).

Image by Dave Winer via a Creative Commons 2.0 license.

Ben Fidler contributed to this report