- A Western Australian-led international clinical trial for a new cystic fibrosis drug has successfully completed a Phase 1b study in patients and is now proceeding into a Phase 2a study
- The locally developed drug is a novel inhaled antibiotic combination, which has already been shown to be effective in reducing infection and improving lung function in cystic fibrosis patients
- The Phase 2a study will be led in Australia by the Perth Children’s Hospital and involve up to 20 other sites in Australia and the USA
- Potential for the drug to be used to treat lung infections in other indications in both children and adults
Children with cystic fibrosis will be included in the next phase of a clinical trial of a promising new treatment pioneered in Western Australia aimed at boosting their immune responses to infections.
Originally developed by researchers at The Kids Research Institute Australia (The Kids) and Perth Children’s Hospital (PCH), the treatment, known as RSP-1502, uses an antibiotic booster to improve the effectiveness of treatment for Pseudomonas aeruginosa infections in people with cystic fibrosis.
Respirion Chief Scientific Officer Professor Barry Clements said a Phase 1b study of the trial had been successful. “We are now able to proceed into the Phase 2 study, which can now include children aged 12 years and older who have cystic fibrosis.
“With limited antibiotic options to address rising bacterial resistance, new options are urgently needed for people with cystic fibrosis and RSP-1502 represents a long-awaited breakthrough for this vulnerable population.”
The Phase 1b study was conducted under US FDA oversight and tested ascending doses of the RSP-1502 combination in cystic fibrosis patients, with the highest dose proceeding to be tested in a Phase 2a study in children and adults.
The follow-on Phase 2a study will test the efficacy of RSP-1502 in reducing bacterial infections and improving lung function, as well as examine how the combination reduces biomarkers of inflammation in the lung.
The Phase 2a study will involve PCH and eight other sites in Australia as well as additional sites in the USA, and enrol 40 children and adults with cystic fibrosis.
“We are very excited by the potential for RSP-1502 to solve the challenge of chronic lung infections in children and adults,” Professor Clements said. “This Phase 2a study will provide key data to support further development of the drug and represents a real inflection point for Respirion.”
About RSP-1502 and Cystic Fibrosis
An early WA-based pilot study using RSP-1502 to treat chronically infected CF patients was published in the industry leading journal[1] and demonstrated an average 16 per cent improvement in lung function - compared to 5 per cent for those patients using a standard of care antibiotic treatment. Enhanced lung function is vital for patients, enabling them to engage more in normal activities such as moving around, participating in sports, enjoying improved quality of life and ultimately, increasing their overall life expectancy.
Cystic fibrosis is a genetic disorder that occurs in about 1 in 4,000 births, where the defect predisposes patients as young as one to lung infections with bacteria including Pseudomonas aeruginosa. Once established, these infections are difficult to clear with existing treatment options, and lead to progressive lung damage, decreased lung function, and eventually respiratory failure and death. With few effective options for the treatment of chronic lung infections in people with cystic fibrosis, RSP-1502 represents a potential breakthrough for this vulnerable population.
The promising results from the Perth pilot study led to RSP-1502 being developed by spinout company Respirion Pharmaceuticals Pty Ltd, supported with funding from Brandon Capital through the Federal Government’s Biomedical Translation Fund and The Kids Research Institute Australia, as well as the US Cystic Fibrosis Foundation, Cure4CF, The Perth Children’s Hospital Foundation, CUREator and other local West Australian investors.
The experienced team behind Respirion were instrumental in developing the current standard of care for cystic fibrosis infections (tobramycin) and have taken a number of drugs successfully through FDA approval and commercialisation globally. Together, with advisers from the US Cystic Fibrosis Foundation and their Therapeutic Development Network, Respirion is now taking RSP-1502 into the Phase 2a efficacy study in Australia and the USA.
Next steps for RSP-1502
With the successful completion of the Phase 1b study, RSP-1502 is now proceeding into a Phase 2a efficacy study in 40 cystic fibrosis patients. Recruitment for the Phase 2a study is currently underway and interested patients can find a local site and additional information about the Phase 2a study by accessing https://clinicaltrials.gov/search?intr=RSP-1502.
This Phase 2a study will bring the new drug closer to receiving international regulatory approval, thereby providing people with cystic fibrosis access to a more effective treatment which, if started early in childhood could slow disease progression. RSP-1502 has broader potential in other chronic lung conditions including Non-CF Bronchiectasis and COPD where new and effective antibiotics are needed as there are no FDA approved inhaled antibiotics for these conditions. Respirion has plans to expand testing RSP-1502 to these conditions and is actively seeking new investment to help fund that expansion.
[1] Puvvadi R, Clements B. Tris-CaEDTA as an adjuvant with nebulised tobramycin in cystic fibrosis patients with Pseudomonas aeruginosa lung infections J Cyst Fibros. 2021 Mar 20(2):316-323
About Respirion Pharmaceuticals Pty Ltd
Founded in 2018, Respirion Pharmaceuticals is an early-stage biotechnology company focused on developing new treatments for respiratory disease. Respirion’s lead product (RSP-1502) is an inhaled combination therapy for the life-threatening disease Cystic Fibrosis, has shown promising results in an early clinical study carried out in conjunction with The Kids Research Institute Australia and the Western Australian Department of Health. The Company has partnered with Australia’s largest life science investment fund, the Medical Research Commercialisation Fund (managed by Brandon Capital), as well as the US Cystic Fibrosis Foundation, Cure4CF, The Kids Research Institute Australia and The Perth Children’s Hospital Foundation to conduct further clinical trials in Australia and the US.
About Brandon Capital
Brandon Capital is Australasia's leading life science venture capital firm, with a strong global presence supported by key partnerships and team members across the US and UK. Through collaboration and investment, we transform promising medical research breakthroughs into medical therapies that improve patients lives. We have supported more than 50 start-up companies to date, the majority of which were founded by us. From early-stage seed investment through to expansion capital, we support life science companies from proof-of-concept through to commercialisation. In ANZ, we source and create our deals through Brandon BioCatalyst (formerly the Medical Research Commercialisation Fund), a unique collaboration of Australian superannuation funds, CSL, the Australian and New Zealand governments, Australian state governments and more than 50 leading medical research institutes and research hospitals.
About the Perth Children’s Hospital Foundation
Perth Children’s Hospital Foundation proudly fuels the fight on the frontline of children’s health care, helping sick children get well and stay well. By working closely with those on the frontline, the Foundation has an in-depth understanding of what clinicians, researchers and families need to ensure we make a tangible impact where it counts most. We are the official and largest funder of PCH and the wider Child and Adolescent Health Service (CAHS) after Government and are the 5th largest philanthropic foundation in WA.