The need-to-know this morning

• Novartis shares were down as much as 10% after the company announced a key neuromuscular disease drug failed in a Phase 3 study, just days after a major cardiovascular drug missed the mark in its pivotal trial.
• AstraZeneca detailed the positive results of two Phase 3 studies of tozorakimab in COPD, with "tozo," as the drug is known, now positioned for blockbuster sales pending approval.
• A once-daily pill developed by Pharvaris reduced the frequency of swelling attacks by 83% in patients with hereditary angioedema — achieving the goal of a Phase 3 study intended to demonstrate the prophylatic drug's ability to prevent such attacks from happening. 
• Structure Therapeutics reported study results from its oral obesity drugs targeting amylin and GLP-1.

Oncology

Patients in need of RevMed's drug struggle to get it

The FDA approval of Revolution Medicines’ pancreatic cancer drug Rasonque last month is expected to dramatically expand access to a treatment that nearly doubled median overall survival. But for some patients in desperate need of the therapy, the decision has actually complicated access, STAT’s Angus Chen writes.

The approval immediately shut down the company’s expanded access program — and both insurers and pharmacies still needed weeks or longer to get the newly approved drug into their systems. Patients like Juan Solano, who has metastatic pancreatic cancer, were left stranded in the bureaucratic gap.

“It was a gut-wrenching process of phone calls and hurdles. Sixty-one phone calls in 48 hours,” his wife, Kelsey, told STAT. “Trying to figure out what needs to happen, and who do I need to talk to, figure out a pathway that my husband can get the medicine [through].”

They ultimately paid $9,286.67 for a seven-day supply, before Revolution Medicines and Aetna intervened and offered a free 30-day supply and insurance coverage for a year. That said, the pain he’d been feeling the past year and a half suddenly vanished after three days on the drug.

“And have a little bit more of him. Get him back a little bit. He’s a triathlete. Or he was,” she said. “I know it’s not a cure, but a median survival of 13.2 months. That is a lot of meaningful time.”

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pulmonary hypertension

Roivant drug delivers impressive mid-stage results

Roivant said this morning that its experimental pulmonary hypertension drug mosliciguat delivered better-than-expected results in a 135-patient Phase 2 study, STAT’s Adam Feuerstein writes. It cut pulmonary vascular resistance by 56% versus placebo, and also significantly improved walking distance as well as a reduction inNt-proBNP, a biomarker of heart failure.

The once-daily inhaled drug is being developed for pulmonary hypertension associated with interstitial lung disease. Right now, the four-times-daily drug Tyvaso from United Therapeutics is the top-selling treatment. Roivant CEO Matt Gline told STAT the magnitude of the benefit exceeded the company’s expectations: “I did not expect in my wildest dreams that we would reach [statistical significance] on a six-minute walk test, but we did and with a phenomenal absolute number.”

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oncology

Inhibrx injects promise into failed cancer target

An experimental immunotherapy from Inhibrx improved head-and-neck cancer responses when combined with Keytruda in a small trial, STAT’s Jason Mast writes. Nearly half the patients taking the INBRX-106 combination therapy saw their tumors shrink considerably, versus just a quarter on Keytroda alone — though the difference was not statistically significant.

The strongest results came in the 19 patients with HPV-positive tumors: 80% responded to the combination, versus a third on Keytruda, and nearly a third had no visible signs of cancer.

This suggests that targeting OX40, a receptor found on T cells that’s been pursued unsuccessfully by Pfizer, Genentech, GSK, AstraZeneca, and others, might indeed have promise.

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