LONDON - PPL Therapeutics Ltd. said it obtained positive results in a Phase II proof-of-concept trial of its transgenic bile salt stimulated lipase (BSSL) in the treatment of pancreatic insufficiency in patients suffering from cystic fibrosis and pancreatitis.
PPL's product, even at the lowest dose tested, was equally as effective as a standardized dose of Creon, the current leader in the digestive lipase market. PPL said its transgenic BSSL will have advantages over existing products that are derived from porcine and bovine pancreatic extracts.
The company said these products "are fairly unpalatable and have low activity, which means patients have to take large numbers of tablets daily - sometimes as many as 30 to 50. Patient compliance is poor."
In addition, PPL, of Edinburgh, Scotland, said there will be a significant market in the treatment of preterm infants for whom existing products are unsuitable. Newborn infants do not produce BSSL themselves, but it is a constituent of human milk. Existing lipase products are unsuitable for infants who cannot be breastfed.
PPL acquired the BSSL project from AstraZeneca plc, of London, which has the right to buy it back at the end of Phase II.
Ron James, managing director of PPL said, "We are very encouraged by these results. Transgenic BSSL clearly works, and the effectiveness of the lowest dose [0.2g] may well be a decisive advantage in the price-sensitive pancreatic insufficiency market.
"The larger commercial opportunity is in premature infants who do not get mother's milk, which naturally contains BSSL. They therefore do not efficiently absorb fats, some of which are believed to be very important to the growth and development of the baby."
A further small-scale trial is being planned to determine the lowest effective dose and see if it is necessary to coat BSSL to protect it from stomach acids.