LONDON ¿ PPL Therapeutics plc announced positive data in a second Phase II trial of transgenically produced Bile Salt Stimulated Lipase (tgBSSL), showing improved fat absorption in cystic fibrosis patients with chronic pancreatic insufficiency.
Ron James, managing director, said, ¿BSSL is PPL¿s second product in advanced clinical trials. The results announced today are encouraging and will form part of the ongoing discussions with our licensing partner, AstraZeneca.¿
The results show that fat absorption increased in relation to the dose of enzyme and that the tgBSSL dose response effect was statistically significant. Fat absorption with tgBSSL at the highest dose was statistically better than no treatment. However, a certain amount of patient-to-patient variability was observed and in some cases fat absorption with tgBSSL was less than that achieved with Creon, the current treatment, which is derived from pig pancreas. PPL says this result is not surprising because CF patients can suffer from severe fat digestion problems, relative to other patient groups with pancreatic insufficiency, and thus are difficult to treat.
Neither Creon nor tgBSSL restored fat absorption to within the normal range. Further clinical trials are planned.
Creon has a low activity, which means patients have to take lots of tablets, and PPL hopes that a transgenic product will overcome this problem. Poor fat absorption is also common in pre-term infants, slowing growth rates and increasing the time they have to spend in intensive care. Because of the high number of tablets needed to achieve an effect, Creon is not suitable for treating babies, and PPL is investigating tgBSSL in this indication. The company, based in Edinburgh, Scotland, intends to produce a highly purified version of tgBSSL for use in this population.
¿PPL¿s transgenic BSSL clearly promotes fat absorption in CF patients suffering from debilitating pancreatic insufficiency,¿ James said. ¿Work remains to be done on dosing levels that may call for individual tailoring in this patient group as is the normal practice with current therapies.
¿We continue to believe that the larger commercial opportunity for PPL is in premature infants, but the need for an effective and acceptable product for adult patients is clear, and both programs remain on target,¿ he said.