The 2017 Nobel Prizes with a biomedical bent were announced this week. Three researchers shared the physiology and medicine prize for their work shining light on the circadian rhythms that govern the wake-and-sleep cycles of all organisms. That basic research is working its way into applications such as drugs that help regulate our body clocks.
Another academic trio won the chemistry Nobel for cool—very, very cool—electron microscopy. Their work has led to microscopic images of living molecules that are frozen in place without damage to their shape or structure.
Congratulations to all the prize winners. The week also brought plenty of high-profile data and a bounty of deals and dollars. Let’s get to the roundup.
—In Washington, four top Trump administration health officials testified about the ongoing opioid crisis. Vox’s Dylan Scott was at the hearing and noted how the facts of opioid addiction have shifted recently, and the public narrative—that most abuse starts with painkillers before moving to heroin—hasn’t caught up. A video replay of the hearing is here.
—Under FDA commissioner Scott Gottlieb, the agency has been on a drug-approval spree. The green lights extend to generics, which Gottlieb specifically targeted when he joined the agency. We have one quarter to go, and the number of generic approvals in 2017 has already topped the previous record, as RF Today reports.
IPOs AND HANDSHAKES
—Rhythm Pharmaceuticals (NASDAQ: RYTM), a Boston company developing treatments for rare metabolic orders that cause obesity, priced its upsized IPO at $17 per share. In first-day trading, the share price jumped more than 75 percent.
—To replenish its depleted pipeline, Endocyte (NASDAQ: ECYT) acquired global rights to a prostate cancer drug developed by German company ABX. West Lafayette, IN-based Endocyte now plans to talk with the FDA about a Phase 3 study for the compound. Shares have quadrupled in value this week to $5.69 apiece.
—Ultragenyx Pharmaceutical (NASDAQ: RARE) is set to acquire gene therapy developer Dimension Therapeutics (NASDAQ: DMTX) after raising its offer to $151 million. Novato, CA-based Ultragenyx plans to keep Dimension’s Cambridge, MA, site.
—Roivant Sciences invested $116 million in Canadian firm Arbutus Biopharma (NASDAQ: ABUS). As Forbes explains, the backing comes as hepatitis-B drug developer Arbutus battles Moderna Therapeutics over patent rights to drug delivery technology. Roivant’s subsidiary Axovant Sciences (NASDAQ: AXON) took a hit last week with the failure of a closely watched Alzheimer’s study.
—Amgen (NASDAQ: AMGN) is paying CytomX $40 million upfront and buying $20 million of its stock in an immuno-oncology partnership. If the partnership produces new cancer drugs, the South San Francisco, CA-based CytomX could earn up to $455 million more.
—In other partnering news, Cambridge biotech Nimbus Therapeutics signed on with Celgene (NASDAQ: CELG) to develop treatments for autoimmune disorders. The companies aren’t disclosing financial terms until Celgene acquires one of the drug programs.
—Flu treatment developer Visterra tacked on another $23.6 million to its Series C funding from the Bill & Melinda Gates Foundation and other investors.
STARTUPS AND LAUNCHES
—Cullinan Oncology raised $150 million in Series A financing that the Cambridge, MA, company will use to assemble a portfolio of cancer drug candidates.
—Waltham, MA-based Palleon Pharmaceuticals raised $48 million in a Series A round to develop drugs that target sugar molecules on tumor cells.
—KSQ Therapeutics of Cambridge, MA, debuted this week with $76 million in funding, 40 employees, and former Genzyme executive David Meeker as its CEO.
—Navire Pharma launched with $30 million in backing from BridgeBio Pharma and a research team from Houston’s MD Anderson Cancer Center that specializes in cancer drugs that attack an enzyme, SHP2, implicated in the growth of several cancers.
—CNBC reported that tech moguls Sean Parker and Peter Thiel are backing a stealthy T-cell therapy company called 3T Biosciences.
DATA, PLANS, AND MOVES
—In a Phase 2/3 study, a gene therapy from bluebird bio (NASDAQ: BLUE) has kept 15 of 17 boys with cerebral adrenoleukodystrophy—commonly known as Lorenzo’s Oil disease—alive and “free of major functional disability,” according to a New England Journal of Medicine paper. The median time from treatment was 29.4 months. The study, dubbed Starbeam, is ongoing.
—Amicus Therapeutics (NASDAQ: FOLD) plans to talk with regulators about a path forward for its Pompe disease treatment. The Cranbury, NJ, company released data from a small study that showed improved movement in patients who have the muscle-wasting disease.
—If CRISPR Therapeutics (NASDAQ: CRSP) manages to move its lead gene-editing drug, a treatment for beta-thalassemia, into clinical studies next year, a new CEO will be in charge. Founding CEO Rodger Novak is stepping aside, and current president and chief business officer Samarth Kulkarni will take the reins Dec. 1.
—A BioHaven (NYSE: BHVN) drug for spinocerebellar ataxia, a rare degenerative brain disorder, failed to beat a placebo in a clinical trial, sending shares of the New Haven, CT-based biotech down more than 28 percent.
—Belgian biotech Ablynx filed for a U.S. IPO right after reporting positive Phase 3 results for its drug caplacizumab, a treatment for a rare blood clotting disease.
—Acadia Pharmaceuticals of San Diego is pushing its drug pimavanserin (Nuplazid) into Phase 3 to study its effect on dementia-related psychosis after the FDA gave it a “breakthrough” designation. The new study will supercede Acadia’s previous plans to study pimavanserin in psychosis specific to Alzheimer’s patients. Pimavanserin is already approved for Parkinson’s-related psychosis.
—Motif Bio (NASDAQ: NTFB) reported positive Phase 3 results for its antibiotic iclaprim. The London firm said it would apply for marketing approval in the U.S.
—Coming off the failure of its C. difficile treatment, microbiome drug maker Seres Therapeutics (NASDAQ: MCRB) reported what it considered positive topline Phase 1b data from a 58-patient ulcerative colitis study. It wasn’t positive enough for investors, who drove the stock down nearly 25 percent.
Frank Vinluan contributed to this report.