Bio Roundup: Drug Prices, CRISPR Caveats, Rubius IPO Pop & More

Xconomy National — 

After nearly two years of all smoke, no fire around President Trump’s promise to curtail drug prices, there’s finally some glimmer of action. Pfizer said last week it would halt its price hikes, and Novartis and Merck took similar actions this week. But we still don’t know the details of the administration’s drug-pricing policy, if there is one beyond the blueprint released in May. And we don’t know if the moves by Pfizer, Novartis, and Merck are just for optics, or indicative of deeper industry compromise. As we’ll outline more below, Novartis’s capitulation comes only after new revelations about its 2017 dalliance with Trump’s right-hand man, Michael Cohen.

What else? Another study emerged with questions about the risks of editing human genes with CRISPR-Cas9; Bill Gates is putting his millions into an effort to find better Alzheimer’s diagnostics; the biggest biotech IPO of the year so far went to a company that’s reengineering red blood cells; and Amgen, Pfizer, and Eli Lilly all have their fingers crossed with potential FDA approvals looming.

You don’t need to pay a fixer for access to the latest information. Just dive with us into this week’s roundup.


—Novartis (NYSE: NVS) said this week it would not raise U.S. prices mid-year, having already raised prices on 75 drugs in January. The announcement came after a Senate Democrat report charged last week that Novartis’s $1.2 million consulting deal with President Trump’s personal attorney and self-described fixer Michael Cohen led to more interactions than the company first admitted. The report noted that Cohen and then-CEO Joe Jimenez traded email, and Jimenez offered some industry-friendly ideas that later appeared in the administration’s drug-price blueprint in May.

—Novartis followed the lead of Pfizer (NYSE: PFE), which cancelled its midyear price hikes last week after pressure from Trump. Pfizer’s pledge wasn’t good enough for Sen. Tammy Baldwin (D-WI), who wrote a letter this week asking the company to “permanently roll back prices.”

—Next up was Merck (NYSE: MRK), which vowed to no longer increase the “average net price across [its] portfolio by more than inflation annually.” Merck also said it will cut the price of hepatitis C drug elbasvir/grazoprevir (Zepatier)—which generated $1.66 billion in 2017, or just 4 percent of Merck’s $40.12 billion in sales, and whose revenue is declining—by 60 percent, and “several other medicines” that have already lost patent protection by 10 percent. Here’s more from the New York Times.

—The FDA formed a working group to consider the possibility of importing drugs in certain cases, such when “sudden, significant price increases…close off channels of availability.” Here’s more from Forbes.

—Martin Shkreli’s former company, Vyera Pharmaceuticals (once known as Turing Pharmaceuticals), is losing cash and considering a name change, STAT reports. When known as Turing, the company bought an old drug for a rare infection and gained national infamy for jacking up its price by 5,000 percent.


—A paper published in Science Translational Medicine suggested that CRISPR-Cas9 gene editing, in humans, might alter segments of DNA that it shouldn’t touch. While the paper has plenty of caveats, it is the latest academic study to warn of possible unintended side effects of CRISPR. Shares of CRISPR drug developers Editas Medicine (NASDAQ: EDIT), Intellia Therapeutics (NASDAQ: NTLA), and CRISPR Therapeutics (NASDAQ: CRSP) all fell about 6 to 7 percent.

—Bill Gates and Alzheimer’s Drug Discovery Foundation co-founder Leonard Lauder are backing a new initiative, the Diagnostics Accelerator, meant to help find biomarkers for early detection of Alzheimer’s.


—With new clinical data, Amgen (NASDAQ: AMGN) resubmitted its application for osteoporosis drug romosozumab (Evenity). The FDA rejected the drug in 2017 due to concerns about heart risks.

—Pfizer and Eli Lilly (NYSE: LLY) reported positive results from a Phase 3 study of tanezumab, a member of a class of pain drugs called NGF inhibitors that have been stymied by safety issues over the years. The companies reported some patients experienced rapidly progressing osteoarthritis, a side effect that has led the FDA to suspend testing of previous NGF blockers.

—Roche handed Japanese rights to entrectinib—a targeted cancer drug it acquired when it bought Ignyta for $1.7 billionto Chugai Pharmaceutical for an undisclosed sum.

—Roche also disclosed that its experimental oral flu treatment baloxavir marboxil succeeded in the second of two Phase 3 studies. The drug is currently under FDA review, with a decision expected in late December.

—Roche’s Genentech unit also said a combination of its immunotherapy atezolizumab (Tecentriq) and chemotherapy succeeded in another non-small cell lung cancer study. But the results, as of yet, haven’t lead to a statistically significant survival benefit—positive news for Merck, whose combination regimen so far has bested its rivals in lung cancer.

—And the Swiss firm cut a deal with PureTech Health to use exosomes derived from milk to orally deliver complex drugs, like RNA therapies. Roche committed to pay PureTech “up to” $36 million, which includes an unspecified upfront cash payment. PureTech could see a variety of downstream payouts too.

—Allergan (NYSE: AGN) and partner Molecular Partners declared success in two Phase 3 studies of abicipar, an experimental drug for the “wet” form of age-related macular degeneration that could compete with drugs like aflibercept (Eylea) and ranibizumab (Lucentis). Analysts were critical of the data, however. Evercore ISI’s Umer Raffat called the results “underwhelming” and Leerink’s Geoffrey Porges felt abicipar only has a “modest chance” of approval and worse commercial prospects, particularly given the drug’s propensity to cause eye inflammation.

—Novartis paid $111 million up front to grab rights to an experimental eczema drug, MOR106, from MorphoSys and Galapagos NV.

—Sanofi (NYSE: SNY) paid Redwood City, CA, startup Revolution Medicines $50 million upfront to split rights to a group of experimental cancer medicines. Revolution recently switched its focus from antifungals to cancer.

—Johnson & Johnson’s (NYSE: JNJ) canagliflozin (Invokana) succeeded in a Phase 3 study of Type 2 diabetes patients with chronic kidney disease. The FDA also delayed a decision whether to expand canagliflozin’s diabetes label to include cardiovascular disease.

—Aveo Oncology (NASDAQ: AVEO) won’t report results from a Phase 3 study of kidney cancer drug tivozanib until the fourth quarter. Data had been expected in the third quarter. Shares fell more than 30 percent this week.


—The FDA announced plans to help get generic biologic drugs, called “biosimilars,” to market more quickly.

—The agency approved Johnson & Johnson’s HIV antiviral cocktail darunavir (Symtuza), which will now compete with similar, rival medicines from Gilead Sciences and GlaxoSmithKline.

—The FDA also greenlighted the first drug to treat smallpox, TPOXX, from New York-based SIGA Technologies.

—And the agency partially suspended testing of a Mersana Therapeutics (NASDAQ: MRSN) breast cancer drug after a patient died in clinical testing. Shares plunged more than 30 percent.


—Rubius Therapeutics (NASDAQ: RUBYraised $241 million in an upsized IPO, the largest biotech issue this year. The Cambridge firm says its lead red-cell therapeutic, a treatment for the rare disease phenylketonuria, could start clinical testing in 2019.

—Hotspot Therapeutics of Cambridge, MA, emerged with $45 million in financing to find drugs that target proteins in unconventional ways. The firm wants to develop treatments for liver disease and autoimmune disorders.

—Verge Genomics, a startup using a combination of machine learning and cutting-edge biology to find treatments for neurological diseases, raised a $32 million Series A round. Xconomy profiled its young CEO, Alice Zhang, in 2016.

—IFM Therapeutics spun out a subsidiary, IFM Tre, backed by a $31 million Series A financing round. The cash will support a trio preclinical drugs targeting inflammatory disorders.

—Roivant Sciences, the holding company run by Vivek Ramaswamy, formed its latest biotech. The company, Shanghai-based Sinovant Sciences, is developing a pipeline of medicines for Chinese and other Asian markets.

—Hong Kong and Rockville, MD-based Ascentage Pharma raised a $150 million Series C to continue developing drugs for cancer, hepatitis B, and age-related diseases.

—Neuraly, a Johns Hopkins Medicine spinout based in Germantown, MD, bagged a $36 million Series A to advance therapies for neurodegenerative diseases. Research underlying the startup was recently reported in Nature Medicine.

—Pliant Therapeutics of South San Francisco, CA, raised a $62 million Series B round to support tests of its lead drug as a treatment for liver and lung fibrosis.


—This week we unveiled the Xconomy Awards finalists for the Innovation at the Intersection category. The finalists all use a variety of methods outside of biology to tackle life sciences problems.

Alex Lash and Frank Vinluan contributed to this report.