All eyes are on the nation’s capital this week, where the Senate released its version of the American Health Care Act (now called the Better Care Reconciliation Act) and the White House worked on an executive order to address drug prices. No one knows how to pay for high-priced medicines that could bring long-term relief or even cures; we checked with gene therapy maker Bluebird Bio about its latest experimental data and asked officials for their thoughts on pricing. There were plenty more clinical results and FDA news to round up, plus boatloads of startup funding. Let’s get right to it.
THIS WEEK IN WASHINGTON
—After secret deliberations that kept many of their own healthcare-savvy colleagues in the dark, the Senate GOP released its version of Trumpcare Thursday morning. It proposes to roll back Medicare, just like the House version of the bill, but more slowly. It keeps the Obamacare protections for people with pre-existing medical conditions, unlike the House bill. It raises the ceiling on how much more insurers could charge older people than younger people, same as the House bill. For a comprehensive comparison, click here.
—While much of the healthcare industry’s attention focused on the Senate bill, a separate behind-the-scenes effort was underway to write an executive order on drug prices. Though President Trump has pledged to rein in prices and famously said before his inauguration that drug companies are “getting away with murder,” experts told Kaiser Health News the White House’s draft proposals won’t ease patient costs and are actually “pharma-friendly recommendations.”
—As Thursday ended, biopharma indices were up more than 8 percent for the week.
WHAT DOES THE FDA SAY?
—New FDA commissioner Scott Gottlieb told Congress that the agency would soon offer new guidance for developers of cancer drugs that are “tumor-agnostic”—that is, based on the underlying genetic profile of the cancer, not the organ or tissue where the tumor is growing. The FDA has already shown openness to the idea with its approval last month of pembrolizumab (Keytruda) for cancers that share certain genetic abnormalities. Gottlieb also sidestepped questions about the Trump administration’s proposal to slash FDA’s budget and raise the fees drug and device makers pay to have their products reviewed.
—The antibiotic delafloxacin (Baxdela) received FDA approval to treat serious skin infections, the first drug approval for New Haven, CT-based Melinta Therapeutics.
—Neos Therapeutics received FDA approval for methylphenidate (Cotempla XR-ODT), the Grand Prairie, TX-based company’s extended-release tablet for attention deficit hyperactivity disorder.
TRIALS AND TRIBULATIONS
—The same researchers who showed in 2014 that the Alzheimer’s drug pipeline had a 99.6 percent failure rate released this week an analysis of the current pipeline. Among their findings: Only a small percentage of Phase 2 drugs are anti-amyloid treatments; current studies are aiming to recruit a “daunting” number of patients; and the slow pace of recruitment is a challenge for drugs that aim to change the underlying course of disease—70 percent of the pipeline.
—Bothell, WA-based Seattle Genetics (NASDAQ: SGEN) stopped a Phase 3 study of its acute myeloid leukemia drug after more patients died in the treatment arm than in the control group. The FDA has halted all trials involving the drug, according to a regulatory filing.
—Clovis Oncology (NASDAQ: CLVS) of Boulder, CO, reported Monday that rucaparib (Rubraca), already approved to treat ovarian cancer driven by the BRCA mutation, might also help women regardless of genetic profile. The firm will use its Phase 3 data to ask the FDA for the wider approval.
—Regeneron Pharmaceuticals (NASDAQ: REGN) of Tarrytown, NY, took a hit from Novartis, whose experimental drug to treat the eye disease AMD worked just as well as Regeneron’s aflibercept (Eylea) but required fewer injections in more than half the patients in head-to-head studies.
—Bluebird Bio (NASDAQ: BLUE) said the first results from its revamped LentiGlobin gene therapy product showed an improvement over the old process in a Phase 3 beta-thalassemia study, but the sample size was tiny: one patient on treatment for six months and two other patients for only two and three months.
—Nature Methods noted that study results it published on May 30, describing higher than expected off-target edits in a CRISPR-Cas9 gene editing experiment in mice, had attracted criticism, and that the journal editors were working on a response. The study, published as a letter to the editor, attracted a storm of criticism from companies developing CRISPR-Cas9 medicines and academics in the gene-editing field.
—Rubius Therapeutics of Cambridge, MA, reeled in $120 million in Series B funding to bring its engineered red blood cells into clinical trials as treatments for enzyme disorders and possibly other diseases.
—Cambridge-based Sigilon Therapeutics raised $23.5 million to finance clinical trials of an encapsulation technology that keeps the immune system from recognizing biological drugs as foreign bodies.
—Syntimmune landed $50 million in a Series B round. It aims to start clinical trials for two rare autoimmune disorders soon.
—Repare Therapeutics emerged from stealth with a $68 million Series A round that the company, split between Boston and Montreal, will use to discover drugs that fight cancer by disrupting tumor repair mechanisms.
U.S. map image from Dave Winer via Creative Commons 2.0.
Frank Vinluan contributed to this report.