FDA opens up Bellerophon's gas therapy for COVID-19, stock rockets

A patient in a hospital bed on a ventilator
Nitric oxide gas previously showed affects against the novel coronavirus' genetically similar predecessor, the strain behind the SARS outbreak of the early 2000s. (Getty/sudok1)

Using a regulatory pathway that allows patients with life-threatening conditions to use unapproved therapies, the FDA has granted “emergency expanded access” to Bellerophon Therapeutics’ inhaled nitric oxide delivery system for treating the novel coronavirus. 

Also known as compassionate use, the agency’s policy is typically administered on a per-patient basis—at times when clinical trials are infeasible, the benefits are seen to outweigh the risks, and there are no available alternatives. 

According to Bellerophon, the FDA’s decision now opens up its INOpulse system for use at high-doses in ventilated COVID-19 patients, immediately. The Friday morning announcement rocketed the company’s stock price over 550% in premarket trading.

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Inhaled nitric oxide acts as a vasodilator, relaxing the lungs’ muscles and blood vessels, and is used in hospitals worldwide. Bellerophon has been researching its use to combat potentially harmful high blood pressure in the lungs among patients with chronic obstructive pulmonary disease, pulmonary fibrosis, interstitial lung disease and sarcoidosis.

The compound has also been tested as a potential treatment for the novel coronavirus’ closely related predecessor—the strain behind the SARS outbreak in the early 2000s. Previous studies showed that nitric oxide slowed down the replication of that virus and reduced the need for long-term ventilator support. 

According to Bellerophon, the genetic similarities between the two coronaviruses “support the potential for [inhaled nitric oxide] to provide meaningful benefit for patients infected with COVID-19” and inhibit the progression of upper respiratory tract symptoms. 

“Based on currently available data and its significant role in the immune response, we believe INOpulse has the potential to be a safe and effective treatment for COVID-19,” said CEO Fabian Tenenbaum. “INOpulse technology utilizes targeted pulsatile delivery of inhaled nitric oxide, providing important antiviral potential, as well as improved arterial oxygenation.” 

“Importantly, INOpulse is designed to treat patients in the outpatient setting, which may be critical in helping combat the further spread of the virus and significantly alleviate the mounting impact on hospitals and intensive care units,” Tenenbaum added.

Prior to the outbreak, Bellerophon had been building up inventory of its INOpulse device for a planned, large phase 3 trial in pulmonary fibrosis—which the company is currently looking to divert to hospitals in need, Tenenbaum told FierceMedTech in an interview.

Additionally, about a month ago, Bellerophon completed a safety trial in healthy volunteers showing its high-dose paradigm could be tolerated, at levels similar to those used against the earlier SARS outbreak, he said. The FDA is also currently reviewing a research protocol for a new clinical trial against COVID-19 to explore the device's next steps in development.

The FDA’s access decision comes less than 24 hours after President Donald Trump pointed to compassionate use as one of the ways the administration is responding to the spread of the virus.

“Disseminating information about promising off-label uses of drugs we already have, investigating their effectiveness, and pursuing other therapeutics will help give American healthcare providers the tools they need to save lives,” HHS Secretary Alex Azar said in an FDA statement on the agency’s work to facilitate development of investigational treatments such as the malaria drug chloroquine and Gilead’s remdesivir antiviral, which is also available under compassionate use. 

“While there are no FDA-approved therapeutics or drugs to treat, cure or prevent COVID-19, there are several FDA-approved treatments that may help ease the symptoms from a supportive care perspective,” the agency said March 19.

The new emergency expanded access pathway allows doctors to request use of the INOpulse for multiple patients at once, without including their names or other personally identifying details, Tenenbaum said.

Inhaled nitric oxide has also been pursued as a COVID-19 therapy by several others, and is currently undergoing clinical trials in China—both as a potential treatment for patients and as a preventive measure for healthcare providers—with studies sponsored by Massachusetts General Hospital and the Chinese University of Hong Kong.

Mallinckrodt, for one, has been exploring (PDF) its use against the disease in coordination with the FDA, NIH and BARDA, and previously markets the gas as a treatment for neonates suffering from respiratory failure.

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