Bayer and PPL Therapeutics are to collaborate in developing PPL's recombinant alpha-1 antitrypsin (AAT) for treating AAT deficiency-related emphysema and cystic fibrosis. The companies announced this week that they would initially collaborate on a phase III efficacy study in AAT deficiency and then in the manufacture of an aerosol formulation of the recombinant AAT, for delivery by nebuliser.
PPL's AAT is produced from the milk of transgenic animals that have been given the human gene for AAT.
An inherited deficiency of AAT predisposes to premature development of emphysema. Treatment with replacement AAT can protect against lung tissue destruction in such cases. In cystic fibrosis, AAT is not a cure but it might reduce symptoms of the disease by inhibiting locally produced elastase enzyme in the lung.
Bayer already produces a plasma-derived AAT product (Prolastin). Prolastin, which is given intravenously, is currently the only product available for chronic AAT replacement therapy.
"For Bayer, this agreement is the first step in greatly increasing AAT supply and convenience to patients. Bayer's commitment to this therapeutic category, combined with PPL's recombinant AAT technology, is a natural collaboration for future advances," said Mr Jan Turek (general manager, Bayer's biological product global business unit). He said that they were hoping to begin the phase III trial at the end of 2000.