Savara abandons drug behind last year’s reverse merger with Mast

(Connor Wells via Unsplash)

Texas biotech Savara has stopped development of an inhaled drug for heart failure after a failed phase 2 trial, sparking a fall in its share price.

The INDIE study of Aironite (AIR001)—an inhaled formulation of sodium nitrite—was being tested to see whether it could provide symptomatic relief to heart failure patients with preserved ejection fraction (HFpEF), a 3 million-strong group of patients in the U.S. alone that has been particularly poorly served when it comes to new drug treatments.

Aironite wasn’t able to meet the primary objective of increasing exercise capacity in these patients in the trial, however, and Savara now says it “does not plan to support any new development” of the drug. The results of the 105-patient study were presented yesterday at the American College of Cardiology (ACC) conference in Orlando, Florida.

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Barry Borlaug, M.D., professor of medicine at Mayo Clinic in Rochester, Minnesota, and principal investigator for the study, told attendees at the ACC conference that the drug also failed to improve daily activity levels, severity of symptoms or quality of life in patients. The trial could have been affected by adherence issues, he suggested, as patients needed to receive the drug via nebulizer for 10 minutes three times a day, a regimen he described as “arduous.”

“We are disappointed that this treatment strategy failed to live up to the promise it had shown in preliminary studies,” said Borlaug. “We need to redouble our efforts to find an effective approach to treating this patient population, which represents a huge unmet public health need.”

It’s a particularly disappointing turn of events for Savara, as Aironite was the main asset behind its reverse merger with Mast Therapeutics last year—which was itself struggling to cope with the failure of sickle cell drug vepoloxamer and was running out of cash.

The deal did provide Savara with a shortcut to a public listing, but that has made the latest setback all the more visible, with shares in the company down around 10% at the time of writing, having been down almost a quarter at one point.

At the time, Savara Chairman and CEO Rob Neville described the reverse merger as “transformative,” adding a new respiratory asset with potential in large numbers of patients to a pipeline that until that point had been focused on rare and orphan indications.

Savara’s pipeline includes in-house inhaled antibiotic AeroVanc (vancomycin) for resistant infections in cystic fibrosis patients, as well as Molgradex (recombinant human GM-CSF) for rare respiratory disease pulmonary alveolar proteinosis, which it acquired after taking over Denmark’s Serendex Pharma in 2016. The biotech has previously suggested these could have $1 billion sales potential, which could explain the moderate share price decline.

"These results were obviously disappointing, but we hope the study will nevertheless serve to increase the understanding of the disease mechanisms in HFpEF, and perhaps help develop other treatment concepts for this difficult clinical condition,” commented Neville in the statement.