Bio Roundup: Drug Price Debates, NY Bio Blooms & CEOs Take Social Stand

Xconomy National — 

In these politically divided times, Americans this week found near universal agreement on one thing: A celestial event that plunges a summer day into temporary darkness is a captivating experience.

The totality of the solar eclipse lasted less than 3 minutes. If you missed it, you’ll have to wait until 2024 for the next one. Those of us who stepped outside with eclipse glasses, pin-hole projectors, or even colanders found a brief respite from the news.

But developments in health policy and life sciences march onward. This week saw some skirmishes in the drug price debate, new plans to expand the biotech startup presence in the Big Apple, and heartfelt reflection from two life science CEOs weighing in on the role that executives have, speaking out on policy and social matters. Let’s get to this week’s roundup—no special eyewear needed.

Policy, pricing & politics

—When the U.S. Senate returns from its summer recess after Labor Day, the Health, Education, Labor, and Pensions committee will hold healthcare reform hearings Sept. 6 and 7 and try to find what HELP chair Lamar Alexander (R-TN) calls a “bipartisan way to get a limited result that actually helps people.” Democrat and Republican governors and state insurance commissioners will testify. Here’s a Q&A with Alexander that dives into details of insurance coverage in his state and beyond.

—The running fight over the price of the cholesterol-fighting drug evolocumab (Repatha) played out this week in the Journal of the American Medical Association. UCSF researchers said the drug needs a 70 percent cut to be cost-effective, using data that evolocumab owner Amgen (NASDAQ: AMGN) published earlier this year. A separate study, funded and supported by Amgen, said the current $14,500 list price was cost-effective in some situations. Each side criticized the other’s study methods.

—Patient advocate (and cancer patient) David Mitchell has gotten the attention of Novartis CEO Joe Jimenez, whose company should receive a historic FDA nod for its CAR-T therapy this fall. Mitchell is pressing the point that Novartis benefited from taxpayer support for early R&D, and it should think hard about how it sets the therapy’s price. Mitchell spoke to FiercePharma here.

—Biotech CEOs Jeremy Levin and Steve Holtzman were disgusted with the violent events in Charlottesville, VA, and the aftermath. Last week, they expressed their disappointment with the muted response from the life sciences industry. Xconomy spoke with Levin and Holtzman about the responsibility of executives to stand against policies and values they detest.

Clinical developments

—The FDA has scheduled a Sept. 28 advisory panel to weigh the merits of ataluren (Translarna), the Duchenne muscular dystrophy drug that PTC Therapeutics (NASDAQ: PTCT) filed for approval of “over protest” of the agency.

—Alkermes (NASDAQ: ALKS) began submitting an approval application for its experimental depression drug, ALKS-5461, a closely watched therapy that had mixed results in a group of Phase 3 tests. The application should be complete by the end of 2017.

—Ultragenyx Pharmaceutical (NASDAQ: RARE) stopped work on its experimental treatment for GNE myopathy, a disease that leads to progressive muscle atrophy and weakness. The Novato, CA, company’s drug failed to beat a placebo in a Phase 3 clinical trial.

—The FDA approved amantadine (Gocovri) a drug developed by Emeryville, CA-based Adamas Pharmaceuticals (NASDAQ: ADMS) to treat the involuntary and unpredictable movements experienced by patients who have Parkinson’s disease.

Biotech moves

—Bloomberg reported that Paratek Pharmaceuticals (NASDAQ:PRTK), which could file for approval of a broad-spectrum antibiotic next year, is considering a sale. Shares climbed more than 27 percent.

—Seattle’s Omeros (NASDAQ: OMER) responded this week to a STAT article that contained “inaccuracies,” the company said. The article, and this follow-on, amplifies questions raised by a short seller group that uses the online pseudonym “Art Doyle.” Omeros is suing the group for defamation.

—Just a week after a new lab space facility was christened on the West Side of Manhattan, Xconomy learned of new plans in the works for what would be known as the New York Life Sciences and Biotechnology Center, a research complex planned for construction next year near the East River.

—GNS Healthcare, a Cambridge, MA, startup whose software uses modeling and machine-learning techniques to try to improve healthcare, raised $6 million in funding led by Amgen Ventures.

—Longevity Fund, a venture capital firm that invests in startups working on anti-aging therapies, raised $22 million to support companies developing potential treatments for heart disease, arthritis, and Alzheimer’s disease, according to the Wall Street Journal.

…And the award could go to…

—The inaugural Xconomy Awards gala is a month away. This week, we took a look at the finalists in the Commitment to Diversity category.

Alex Lash and Ben Fidler contributed to this report.

Photo by Flickr user David Moulton via a Creative Commons license. Photo has been cropped to fit Xconomy’s publishing system standards.