BETHESDA, Md. -- Today, the Cystic Fibrosis Foundation announced its investment of $3.5 million in Pulmocide Limited, a U.K.-based biotech company, to develop an inhaled drug (opelconazole) to prevent Aspergillus fungal infections in lung transplant recipients. The investment is part of the CF Foundation’s Lung Transplant Initiative, a commitment to improve outcomes and support people with CF throughout the transplant journey. It also marks the CF Foundation’s first funding of a potential treatment specifically for people who have received a lung transplant.
The CF Foundation’s funding will support a multicenter Phase 2 study of opelconazole. The objective of the study is to assess the treatment’s safety and tolerability and the patient’s ability to complete the course of treatment when being used to prevent invasive pulmonary fungal infections in lung transplant recipients.
Although anyone who has received a lung transplant may be susceptible to an Aspergillus fungal infection, people with CF who get a lung transplant are at an even higher risk. They are more likely to have fungus present in their airways because of their cystic fibrosis, even before transplant, which can lead to an invasive fungal infection after transplant.
“This is exciting news for the entire CF community,” said Albert Faro, MD, vice president of clinical affairs for the CF Foundation. “We are hopeful not only that this potential treatment could help prevent lung transplant recipients from getting a devastating Aspergillus infection, but also that if successful, it could eventually help all people with CF who have chronic Aspergillus infections.”
Challenges of current treatment options
There are many challenges associated with current treatment options for people infected with Aspergillus. They may be hard to tolerate, which may lead many patients to discontinue use before they have completed the treatment’s course. Some current treatments may also interact with other common transplant medicines, resulting in potentially serious side effects.
The Phase 2 trial the Foundation is funding aims to see whether inhaled opelconazole will be well tolerated and less likely to have drug-drug interactions. As an inhaled treatment option, it has the potential for being administered at home, instead of at a transplant or CF care center.
This potential treatment could benefit all lung transplant recipients.
About the CF Foundation’s Lung Transplant Initiative
As part of the CF Foundation's commitment to helping people with CF wherever they are on their journey, the Foundation established the Lung Transplant Initiative in 2016 with the goal of maximizing the opportunity for transplant as a life-sustaining therapy and extending post-transplant survival. Since the initiative began, the Foundation has helped facilitate the creation of new clinical care guidelines, extended a key lung transplant study, worked to improve organ allocation for people with CF, developed a clinical database focused on advanced lung disease and lung transplant, awarded research grants, and funded a new lung transplant-specific biorepository and linked patient registry. The agreement with Pulmocide is the first clinical-stage lung transplant trial for the initiative.
Lung transplantation remains a vital treatment option for individuals with CF who have advanced lung disease. Post-transplant outcomes vary widely among people with cystic fibrosis, and the factors that drive these outcomes are not well understood. The Foundation's Lung Transplant Initiative aims to increase our understanding of these factors and drive therapeutic development to improve outcomes.
About the Cystic Fibrosis Foundation
The Cystic Fibrosis Foundation is the world's leader in the search for a cure for cystic fibrosis. The Foundation funds more CF research than any other organization, and nearly every CF drug available today was made possible because of Foundation support. Based in Bethesda, Md., the Foundation also supports and accredits a national care center network that has been recognized by the National Institutes of Health as a model of care for a chronic disease. The CF Foundation is a donor-supported nonprofit organization. For more information, visit cff.org.