Project to Advance Push-Button, Single-Dose Inhaler to Treat CF Lung Infections

Pharmaxis has entered into a research collaboration with the Woolcock Institute of Medical Research in Sydney, Australia, to develop an inhalation therapy that combines Pharmaxis’ Orbital Inhaler with a dry powder formulation of the antibiotic tobramycin. The resulting therapy is intended for patients with cystic fibrosis (CF).

Supported by a $421,545 grant from The National Health and Medical Research Council (NHMRC), the work will be led by Professor Paul Young, director of the Woolcock Institute and head of Respiratory Technology. Pharmaxis aims to create a commercial-ready product to be further tested in late-stage clinical trials, its chief executive officer, Gary Phillips, said in a press release.

The Orbital inhaler is designed to deliver high-doses of dry powder antibiotics directly into the respiratory tract from a single dosing, push-button unit. It is designed to be disposable, eliminating the risk of re-infection for users.

“While antibiotic treatment options for patients with cystic fibrosis has come a long way, the use of innovative devices such as the Orbital will be a significant step forward,” Young said. “Currently, CF patients have to grapple with loading multiple drug-containing capsules into their devices when taking their daily dose of antibiotics. With a lack of flexibility … patients may encounter tolerability and cough issues along with logistical issues … The Orbital circumvents these problems by providing a press-button, single use device containing the whole antibiotic dose that the patient can inhale over a number of breaths that are suitable to them.

“This approach would not only improve the quality of life for CF sufferers but mark a revolutionary way in which we deliver antibiotics for CF,” he said.

A Phase 1 study of the inhaler’s use in healthy subjects showed that that large amounts of dry powder (up to 118 mg) could be administered in one inhalation without compromising safety or tolerability, the company reported.

Orbital can deliver single antibiotic powder doses, starting at 50 mg, in a series of sequential inhalations to treat lung infections in CF patients.

“Australia is at the leading edge of CF treatment. We have around 3200 people with CF in Australia, and each will need to receive regular antibiotic treatment for lung infection, occurring due to the thick mucosal secretion build-up in the lung. If these are not treated effectively, progressive lung deterioration occurs,” said Scott Bell, a member of the research team, with The Prince Charles Hospital and QIMR Berghofer Medical Research Institute in Brisbane. “The ability to deliver antibiotics locally, using the Orbital device, overcomes a number of challenges that we are facing in the clinic, and this clinical trial is likely to pave the way to better health outcomes and quality of life.”

Pharmaxis has also developed Bronchitol, a dry powder mannitol which is inhaled twice daily using a small handheld device. Bronchitol is currently approved in Europe, Russia and Australia for the treatment of certain CF patients to help them clear mucus from their lungs. The company is working toward regulatory approval in the United States.