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Bio Roundup: Award Winners, Lung Data, Dems on Drug Pricing & More

Xconomy National — 

For decades, advanced lung cancer was a quick death sentence. That’s no longer a given, thanks in part to the arrival of immunotherapy. If a medical meeting in Barcelona this week was any indication, more help could be on the way.

Targeted medicine isn’t new to lung cancer, but only a fraction of patients have the eligible mutations, such as EGFR and ALK, and thus have a chance to avoid chemotherapy and its toxic side effects. New drugs targeting other alterations, like KRAS-G12C and RET fusions, could expand that pool. A few of them continued to show promise in clinical studies this week.

There was plenty more to digest as the medical conference calendar kicked into high gear this week, along with the rest of the top life sciences headlines, so let’s get right to it.


—Treatment for lung cancer has changed dramatically the past few years, and more advances were presented this week at a medical meeting in Spain. Among them: targeted pills, immunotherapy-chemotherapy combinations, and a closely watched medicine from Amgen (NASDAQ: AMGN).

—In a Phase 3 study called IMpower110, the Roche immunotherapy atezolizumab (Tecentriq) fared better than chemotherapy in patients whose non-small cell lung cancer (NSCLC) has high levels of the protein PD-L1.

—Merck (NYSE: MRK) and Bristol-Myers Squibb (NYSE: BMY) had immunotherapy updates too. Merck’s pembrolizumab (Keytruda)-chemo combo helped NSCLC patients whose tumors didn’t have PD-L1; Bristol presented long-term survival numbers for patients on nivolumab (Opdivo).


—Biogen (NASDAQ: BIIB) and Alkermes (NASDAQ: ALKS) posted an update for diroximel fumarate, which they aim to show is a safer successor to Biogen’s multiple sclerosis (MS) pill Tecfidera. Less than 1 percent of the 888 patients in the Phase 3 study EVOLVE-MS-1 for a median of 18 months have dropped out thus far because of GI issues, a key statistic the companies are tracking.

—Novartis (NYSE: NVS) today is presenting results of a Phase 3 study showing that its monthly MS drug ofatumumab beat Sanofi’s teriflunomide (Aubagio) head to head.

—Moderna (NASDAQ: MRNA) reported encouraging Phase 1 results for cytomegalovirus and chikungunya virus infections, a small step forward for its closely watched experimental messenger RNA therapies. The biggest cause for concern: One volunteer reported cardiovascular problems. STAT has more.

—Shares of Adverum Biotechnologies (NASDAQ: ADVM) fell 31 percent after what analyst Mani Foroohar of SVB Leerink called “mixed signals” in an early trial of a gene therapy for wet age-related macular degeneration. At the study’s six-month mark, none of the six patients had yet received a rescue therapy. But vision, on average, also worsened.

—Shares of Tocagen (NASDAQ: TOCA) plummeted nearly 80 percent after its gene therapy for recurrent brain cancer failed a Phase 3 trial.

—Neurotrope (NASDAQ: NTRP) shares fell below the dollar mark after its Phase 2 Alzheimer’s drug Bryostatin-1 failed to show benefit over placebo.

—Aveo Oncology (NASDAQ: AVEO) updated survival data from a late-stage study of its tivozanib (Fotvida) in kidney cancer, briefly boosting shares 50 percent. The FDA first rejected Fotvida in 2013. It was approved in Europe in 2017, but Aveo warned earlier this year that the approval was at risk if long-term survival rates didn’t improve.

—The Acadia Pharmaceuticals (NASDAQ: ACAD) drug pimavanserin succeeded in a Phase 3 study in psychosis in dementia patients. The drug is already approved to treat psychosis in Parkinson’s patients.


—The winners of this year’s Lasker Awards, the top US biomedical prizes, went to the inventors of the breast cancer drug Herceptin, pioneers in the field of immunology, and a nonprofit expanding global access to vaccines.

—Xconomy hosted its annual Boston life science awards on Thursday night. Winners included Sage Therapeutics CEO Jeff Jonas, Cambridge, MA, startup IFM Therapeutics, Third Rock Ventures co-founder Mark Levin, and many more.


—Investors and Wall Street analysts mainly shrugged off leaked news of a House Democrat drug-pricing plan that would give Medicare negotiating power, penalize companies that refused to negotiate, base price caps on an international index, and more.

—The Trump administration called for a ban on flavored electronic cigarettes, a decision that follows reports of at least six deaths and respiratory illness in hundreds associated with vaping.

—Purdue Pharma reached a tentative deal to settle litigation with approximately 2,000 state and local governments that hold the company partly responsible for the nation’s opioid crisis. But some states are holding out, saying that the $10 billion to $12 billion settlement isn’t enough.


—Palforzia, an experimental peanut allergy drug from Aimmune Therapeutics (NASDAQ: AIMT), heads to an FDA advisory panel today. In briefing documents posted earlier this week, the agency didn’t question the drug’s effectiveness, but flagged some potential safety concerns seen in human testing.

—Joi Ito resigned as director of MIT Media Lab Saturday and stepped down from several boards including Boston startup creator PureTech Health, after a New Yorker report revealed more details about Ito’s ties to sex offender Jeffrey Epstein.

—Translate Bio (NASDAQ: TBIO) stopped work on MRT5201, an experimental mRNA drug for the rare metabolic disorder ornithine transcarbamylase deficiency. The FDA suspended the program in January.

—Previously sealed legal documents obtained by Reuters argued that Merck has downplayed side effects, like sexual dysfunction and depression, tied to its hair loss drug Propecia. Merck denies the allegations and told Reuters it “stands behind the safety and efficacy of Propecia.”


—Tony Coles, who recently relinquished his role as CEO of Yumanity Therapeutics, was named CEO of Cerevel Therapeutics, which spun out of Pfizer last year with a portfolio of drugs for neurodegenerative diseases.

—Trucode Gene Repair launched with $34 million to support development of a gene-editing technology from Yale University. The San Francisco-based startup says its approach may avoid the potential “off-target” effects of CRISPR-based gene editing.

—Platelet BioGenesis added $26 million to its Series A round to help develop technology to produce platelets from stem cells.

–Netherlands-based Pharvaris got $66 million to back Phase 1 testing of an experimental treatment for hereditary angioedema.

—OncoCell MDx joined the growing group of liquid biopsy startups with a $22.2 million Series B round.

—Six years after GlaxoSmithKline teamed with Avalon Ventures to launch celiac disease drug developer Sitari Pharmaceuticals, GSK bought the company outright. Terms weren’t disclosed.


—This week, the public markets welcomed 10x Genomics (NASDAQ: TXG), SpringWorks Therapeutics (NASDAQ: SWTX), and Satsuma Pharmaceuticals (NASDAQ: STSA). They raised $595 million combined.

Frequency Therapeutics, ADC Therapeutics, Aprea Therapeutics, and BioNTech also jumped into the IPO queue.


—Xconomy will hold a unique event on Oct. 17 at the Broad Institute, “Boston’s Life Science Disruptors,” featuring a candid, fireside chat with biotech pioneer Phil Sharp, a look at the rise of patient influence on the development of rare disease drugs, and more. You can grab your tickets here.


—Carol Addy was appointed chief medical officer of Genfit (NASDAQ:GNFT]])… John Furey left Spark Therapeutics (NASDAQ: ONCE) to become CEO of Imvax… and Monique Levy was named chief strategy officer of BlackThorn Therapeutics.

Frank Vinluan and Alex Lash contributed to this report.