Orphan Drug Candidate ALT-07 

The Alitair Pharmaceutical strategy is to acquire late-stage orphan drug products already approved for therapeutic use outside the United States. This in–licensing-based approach is extremely efficient. In our experience, a typical European-approved product can move rapidly to a late-stage Phase II trial and sometimes a Phase III trial in the United States. To this end, we in-licensed ALT-07 (doxofylline), a drug that is used to treat respiratory diseases. As an Orphan Drug, the product will hold a 7-year exclusivity period, at a minimum.

ALT-07 (doxofylline) possesses many characteristics that make it an exciting alternative to current therapies in the orphan respiratory disease space.  ALT-07 is administered orally, so it has much higher levels of compliance and adherence when compared to the current, predominantly inhaled treatments. Fundamentally, ALT-07 is safer and better tolerated than inhaled long-acting beta agonists (LABAs) and inhaled cortico-steroids (ICS), which are currently the mainstay of orphan respiratory disease treatment. Recent studies regarding the use of long-acting beta agonists have suggested that in rare cases severe asthma attacks and deaths may be associated with their use, resulting in a black box warning for LABA medications. Additionally, there is evidence that inhaled steroids may be associated with growth retardation in children and osteoporosis in adults.  ALT-07 delivers both bronchodilation and anti-inflammatory effects in one oral drug without these side effects. We were granted orphan drug designation for ALT-07 for the treatment of bronchiectasis in the United States.