EXOME

all the information, none of the junk | biotech • healthcare • life sciences

Biotech Roundup: ASCO Ahead, CAMP4’s Cash, Ambien Tweeting & More

Xconomy National — 

For the next five days, the oncology community’s attention will focus on Chicago. The Windy City is the home for the American Society of Clinical Oncology’s annual meeting, and it kicks off today.

Many ASCO attendees will zero in on potential cancer drug combinations. They’re seen as the key to expanding the reach of caner immunotherapy, which still works on just a small fraction of patients. Among other news, expect a post-mortem analysis of a once-promising immunotherapy combination that included so-called IDO inhibitors, which were at the top of the cancer combo hopefuls list just a short time ago. The April clinical trial failure of an immunotherapy-IDO combo set off a cascade of activity as companies halted or curtailed work on their own IDO inhibitors.

In other news this week, the FDA temporarily stopped plans for the first clinical trial testing a CRISPR-cas9 gene editing therapy, the biotech IPO queue added more companies, and a pharma giant schooled the Twitterverse on the side effect profile of its sleep drug. Let’s round up this week’s headlines in biotech and pharma news.

CANCER CONCERNS

—The rise and fall of a class of drugs known as IDO inhibitors serves as a cautionary tale this weekend at the American Society of Clinical Oncology’s annual meeting, when all the new “can’t miss” cancer immunotherapy combinations are trotted out. Xconomy spoke with cancer experts about the lessons learned from the IDO story, and the changes they’d like to see the biopharma industry make as a result.

—Speaking of cancer immunotherapy combinations, this week brought more mixed results. A Roche regimen of immunotherapy and chemotherapy succeeded in another study in advanced lung cancer patients. But Johnson & Johnson and partner Genmab announced plans to discontinue or roll back several combination studies due to safety problems.

—More on the eve of ASCO: Xconomy examined the promise of cancer drugs targeted at a tumor’s genetic profile regardless of its location in the body. The era of tissue-agnostic cancer medicine, which began last year with an FDA approval, isn’t likely to blossom soon. Read on to find out why.

PHARMA FINANCING (AND ONE SETTLEMENT)

—Cortexyme of South San Francisco, CA, raised a $76 million B round from Sequoia Capital, Verily Life Sciences, and others. The cash will go toward Phase 2 studies of its antibacterial drug meant to combat Alzheimer’s disease.

—Shattuck Labs, an Austin, TX, company developing immunotherapies for cancer and inflammatory disorders, raised $46 million and will start clinical trials next year.

Propeller Health raised $20 million to expand its digital technologies, initially developed for respiratory diseases, to potentially include immunology, diabetes, and migraine.

Eidos Therapeutics of San Francisco and Avrobio of Cambridge joined the IPO queue, unveiling plans to raise cash to finance more clinical tests of their respective rare disease drugs.

—Neuro-drug developer Denali Therapeutics (NASDAQ: DNLI) exercised its option to acquire F-Star Gamma, a company that has developed technology to deliver therapies across the blood-brain barrier. South San Francisco, CA-based Denali paid $24 million up front for F-Star, and could be on the hook for up to $447 million in downstream payments.

—A months-long legal fight between the Arcturus Therapeutics (NASDAQ: ARCT) board and the company’s former chief executive has ended. The four directors who fired former CEO Joseph Payne have resigned as part of a settlement agreement, and Payne was reinstated as president and CEO of the San Diego biotech.

DRUG DEVELOPMENTS

—The FDA placed a clinical hold on an application to test a sickle cell disease treatment developed with CRISPR-Cas9 gene editing technology. Vertex Pharmaceuticals (NASDAQ: VRTX) and CRISPR Therapeutics (NASDAQ: CRSP) said the FDA notified the partners the hold relates to “resolution of certain questions” the regulator will ask as part of its review.

—The FDA will decide whether to approve Loxo Oncology’s (NASDAQ: LOXO) larotrectinib by Nov. 26. The drug has a chance to become the second-ever cancer therapy approved for all tumors with a specific genetic signature, regardless of where they originated.

—Another possible milestone approval comes from Sage Therapeutics (NASDAQ: SAGE). By Dec. 19, the FDA could approve brexanolone, which would make it the first-ever drug marketed specifically for postpartum depression.

—Shares of Madrigal Pharmaceuticals (NASDAQ: MDGL) soared 144 percent to $265.61 after the company released updated, 36-week results from a Phase 2 study of MDGL-3196, an experimental drug for nonalcoholic steatohepatitis (NASH). The results appear to put the Pennsylvania company in the thick of the race among companies developing treatments for the fatty liver disease. One analyst told Reuters Madrigal had “the best result we’ve seen across all the NASH studies so far to date.”

—Nektar Therapeutics (NASDAQ: NKTR) filed for FDA approval of NKTR-181 as a treatment for low back pain. The Nektar opioid was developed to offer pain relief without the addictive properties of other treatments in the drug class.

—Women’s health company TherapeuticsMD (NASDAQ: TXMD) won FDA approval for estradiol (Imvexxy), a treatment for vaginal pain during sex. The FDA nod comes a year after the regulator rejected the drug, citing the Boca Raton, FL, company’s lack of long-term data.

—TV star Roseanne Barr blamed her racist tweet about Valerie Jarrett, an Obama adviser, on the sleep drug Ambien. Ambien manufacturer Sanofi checked Ambien’s label and tweeted back that racism isn’t among the listed side effects.

THIS WEEK IN HEALTH POLICY

—“Right to try,” a bill that would allow terminally ill patients to seek access to drugs not yet approved by the FDA (though it does not compel pharmaceutical companies to provide them), was signed into law. President Trump said pharma companies would respond with “voluntary massive” price cuts, but he offered no further details on why that would happen or how such cuts would follow the signing of the new law.

—Virginia legislators voted to expand Medicaid, the government-funded health program for the poor. Here are more details from The Hill.

Ben Fidler and Alex Lash contributed to this report.