Already de-risked through EU approvals, FDA interactions, and Cystic Fibrosis Foundation support
US Patients with Cystic Fibrosis Infections Lack Good Options
Cystic fibrosis (CF) is a genetic disease leading to progressive lung inflammation & damage. Impaired host defense results in chronic lower airway bacterial infection, most commonly by Pseudomonas aeruginosa (PA) and Staphylococcus aureus. PA is linked to greater airway inflammation and is a major etiologic factor in disease progression and overall decline in health. Chronic PA infection results in a vicious cycle of infection and inflammation, which causes airway surface damage, airway plugging, which progressively leads to lung function decline and reduced survival. Strategies to administer anti-pseudomonal antibiotics to the airways have been a cornerstone of CF clinical care for many years. PA remains most prevalent bacterial pathogen in CF in adults, and that most closely linked to worse patient outcomes: chronic PA infection is the leading cause of exacerbations, lung function decline and mortality in CF.
Infections will remain a major problem in CF post-CFTR modulator era as patients live longer. In 2020, 57% of CF patients were over 18 years old, compared to only 32% in 1990. The US CF Foundation estimates that by age 25, >30% of patients have moderate to severe lung disease, increasing to >50% by age 35. Most of these patients are colonized with PA and require regular courses of therapy to manage these infections. That continuing need combined with poor tolerance of long-term tobramycin (otoxicity leading to hearing loss, diminished efficacy) and the fact that CFTR modulators only appear to help clear infections in earlier-stage patients leaves a clear unmet need that ColiFin can fill.
ColiFin fills a critical unmet need in treating CF lung infections for the numerous patients who after long-term tobramycin usage see reduced efficacy & tolerance.
ColiFin® is an inhaled therapeutic for the management of chronic infections in cystic fibrosis (“CF”) patients. Although CFTR modulator therapy has transformed the lives of CF patients, it has also expanded the need for a product like ColiFin® since the overwhelming majority of adult CF patients have irreversible lung damage causing them to suffer from chronic infections despite CFTR modulator therapy. Indeed and counterintuitively, it is expected that because CFTR modulator therapy will extend the lives of CF patients, the actual pool of CF patients needing a product like ColiFin® will increase significantly over the next 20-30 years. Spexis in-licensed worldwide ex-European rights to ColiFin® from PARI GmbH, a world-leader in nebulized drug delivery. ColiFin already is approved and generates significant sales in the EU. Spexis has negotiated a single P3 study pathway to approval with the U.S. FDA and holds both Orphan Drug and QIDP designations, granting it 12 years of U.S. market exclusivity from the time of U.S. approval. ColiFin® also has significant commercial opportunity in non-CF bronchiectasis and COPD, which represent markets many times larger than the CF market. We are seeking investors and corporate partners to support our P3 program and commercialize ColiFin® in North America and in other markets.
ColiFin® Clinical Development Plan
The FDA has given a “Study may Proceed” letter to initiate a Phase 3 program for ColiFin® in adult and adolescent subjects with cystic fibrosis and chronic PA lung infection to support a future US marketing authorization. The clinical development of ColiFin® is supported by an FDA Orphan Drug Designation for treatment of respiratory infection in patients with cystic fibrosis, Qualified Infectious Disease Product (QIDP) Designation for ColiFin® for the treatment of PA lung infections in CF patients, and Fast Track Designation. We plan initiate the ColiFin® COPILOT P3 study in CF patients in 1Q23.
Expanded Access Statement
ColiFin® is an investigational medication currently under development. There are alternative approved treatment options currently available for patients with Cystic Fibrosis. Physicians and healthcare providers are encouraged to consider these approved alternatives as the primary treatment options for their patients.
Spexis is committed to establishing an Expanded Access Program (EAP) to provide limited access to ColiFin® to eligible patients who have exhausted all available approved alternatives. Physicians or healthcare providers interested in applying for access to should contact email@example.com. Our primary goal is to offer hope and support to those in need while maintaining the highest standards of ethical and regulatory compliance.