Who knew healthcare could be so complicated? This week, President Trump and the GOP saw first hand, as a feverish rally to get their healthcare bill through the House of Representatives on Thursday stalled before a vote. Elsewhere in Washington DC, drug pricing continued to be a political football. Two well known congressional drug pricing critics launched a preemptive strike on PTC Therapeutics over its soon to be launched steroid for Duchenne muscular dystrophy, deflazacort (Emflaza), while Trump, during a Monday speech in Louisville, again vowed to introduce drug pricing legislation in the near future.
The rest of the week’s biotech news featured some long-awaited data from Amgen, a coming investigation into the orphan drug program, an update on one of the initiatives to help close the gender gap in life sciences, and much more. Let’s get it all rolling below.
—The House of Representatives delayed its vote on the American Health Care Act, the White House-backed replacement for the Affordable Care Act, also known as Obamacare. According to multiple reports, President Trump met with conservative GOP “Freedom Caucus” House members, and separately with moderate GOP members, but the negotiations didn’t bear enough fruit to hold a vote Thursday.
—At the American College of Cardiology’s annual meeting, Amgen (NASDAQ: AMGN) revealed details of a massive study showing that its cholesterol lowering drug, evolocumab (Repatha), reduced heart attacks and strokes. The company wants insurers to loosen access to the drug, which has struggled in the marketplace. Cardiologists Xconomy spoke with were lukewarm on the drug’s prospects.
Rare Disease Developments
—Congressional drug price hawks Sen. Bernie Sanders (I-VT) and Rep. Elijah Cummings (D-MD) urged PTC Therapeutics (NASDAQ: PTCT) to charge about $1,000 a year for deflazacort, a steroid that helps treat Duchenne. That’s the cost of importing the drug from the U.K. and Canada. PTC paid $140 million for deflazacort rights last week from Marathon Pharmaceuticals—which had intended to charge $89,000 per year, inciting public backlash.
—More on Duchenne: The FDA said Akashi Therapeutics can resume testing its experimental DMD drug, HT-100, a year after a patient died in a clinical study. CEO Marc Blaustein described to Xconomy the findings of Akashi’s investigation and how it intends to press forward.
—Kaiser Health News reported that the Government Accountability Office will investigate drug industry abuse of the Orphan Drug Act, which gives drug makers patent extensions and tax breaks for developing rare disease treatments.
Cancer Ups and Downs
—After a second clinical trial failure, Cerulean Pharma (NASDAQ: CERU) sold its cancer drugs and founding technology for a total of just $7.5 million and announced plans to merge with a privately held women’s health company, Dare Bioscience.
—Eli Lilly (NYSE: LLY) said its breast cancer drug abemaciclib met its main goal in the first of two Phase 3 trials and plans to file for FDA approval in the second quarter. Lilly is trying to catch up to Pfizer and Novartis, which have both already won approval of similar breast cancer drugs, known as CDK4/6 inhibitors.
—Pfizer and partner EMD Serono won FDA approval of avelumab (Bavencio), a treatment for a form of skin cancer called merkel cell carcinoma. The drug has become the fourth of a class of cancer immunotherapy drugs called checkpoint inhibitors to hit the U.S. market.
Suits and Cases
—Biogen (NASDAQ: BIIB) beat back a patent challenge to its multiple sclerosis pill, dimethyl fumarate (Tecfidera), from hedge fund manager Kyle Bass. Biogen still, however, awaits a decision on another patent suit from Forward Pharma (NASDAQ: FWP), the results of which could determine whether Biogen owes the Danish firm royalties on the drug’s sales going forward.
—Regeneron Pharmaceuticals (NASDAQ: REGN) and partner Sanofi preemptively filed a suit against rival Amgen hoping to show patents covering eczema and asthma drug dupilumab (Dupixent) don’t infringe on a patent held by Amgen for a failed, similar type of drug. The companies are already in a patent fight over their rival PCSK9-blocking heart drugs, evolocumab and alirocumab (Praluent).
Real and Rumored Deals
——Boston-based PureTech Health licensed two drug candidates from Novartis and funneled them into a new startup, ResTORbio, which will develop drugs that target a complex of proteins called mTORC1. Novartis gets an equity stake in the startup. ResTORbio, meanwhile, isn’t the only Boston area startup targeting mTORC1; Navitor Pharmaceuticals, backed by Johnson & Johnson, GlaxoSmithKline and others, is also in the mix.
—San Ramon, CA-based BioClin Therapeutics raised $30 million in a Series B round that the company will use to do more clinical testing of its experimental bladder cancer treatment.
—With health insurers increasingly looking to base reimbursement on patient outcomes, New York health IT startup Aetion raised $11 million for software that finds evidence about the value and effectiveness of medical treatment.
—SutroVax raised $64 million in a Series B round that the Foster City, CA-based biotech will apply toward clinical trials testing its conjugate vaccine for pneuomococcal infections.
—Citing an unnamed source, FiercePharma reported that Sanofi is in talks to buy Flexion Therapeutics (NASDAQ: FLXN), developer of a long-acting steroid for osteoarthritic knee pain, for more than $1 billion.
—Google’s secretive anti-aging drug division, Calico, struck a five-year research pact with Cambridge startup C4 Therapeutics to develop drugs for cancer and other diseases. Financial terms weren’t disclosed.
—Biotech’s gender gap on boards and in executive suites is well documented. One response is a new mentoring network, CSweetener, started by Bay Area VC Lisa Suennen. She unveiled it last fall, and it now counts more than 100 each of women subscribers and volunteer mentors.
—Phase 3 clinical trial results for a Nektar Therapeutics (NASDAQ: NKTR) opioid showed the drug reduced pain without causing a high, suggesting that the San Francisco biotech might be able to offer a painkiller that doesn’t add to the growing problem of addiction to prescription medications.
—Former National Institutes of Health Director Harold Varmus, Tri-Institutional Therapeutics Discovery Institute director Mike Foley, and others will be headlining our latest biotech event, “New York Biotech Seizes the Momentum,” which will take place on May 31. You can grab your tickets here.
Frank Vinluan and Alex Lash contributed to this report