PPL Therapeutics Product Review on RecAAT

September 16, 2002

Recombinant Alpha-1-Antitrypsin ("recAAT")

Under the agreement, signed in August 2000, the clinical development of recAAT is being undertaken in collaboration with Bayer Corporation’s Biological Products Division ("Bayer"). Bayer manages and funds the clinical development and marketing of recAAT. In turn, PPL is responsible for exclusive supply of finished product. PPL and Bayer are due to conduct clinical trials for hereditary emphysema with the intention of commercialising an aerosol formulation of transgenically-produced recAAT worldwide. PPL and Bayer are also collaborating on the development of an aerosol recAAT for a second indication, cystic fibrosis (CF).

At the beginning of 2002 PPL and Bayer announced a delay to the recAAT programme in order to permit further work to be carried out on a number of issues that could have caused the higher number of drop outs than would have been expected in a Phase II study carried out by Bayer in 2001.

It was agreed that the companies would look at route of administration, dose, purity, and formulation related issues. PPL and Bayer have been particularly aggressive in pursuing the latter two aspects of this joint programme and have made significant progress in developing an extraction process capable of producing product of even greater purity. This work overcomes one of the technical hurdles to the programme moving forward and thereby significantly reducing the development risk. Bayer has contributed substantial technical expertise and scientific consultation and both parties are pleased with the results of this work and plan to further review progress in the next quarter.

It has always been the intention of the parties to develop AAT for the treatment of both hereditary emphysema and cystic fibrosis. PPL and Bayer remain committed to the license and launch of an aerosolised recAAT product. The developments reported above continue to support a launch date for the product in 2007.

For more information contact:
PPL Therapeutics plc:
April D'Arcy
Tel +44 (0)131 440 4777

Financial PR Enquiries:
Alistair Mackinnon-Musson
Philip Dennis
Hudson Sandler
Tel +44 (0)20 7796 4133

Notes to Editors:

  1. PPL Therapeutics is a biopharmaceutical company, which is one of the world’s leaders in the application of transgenic animal technologies to the development and production of human proteins for therapeutic and nutritional applications.
  2. PPL’s patented transgenic production of human proteins involves the introduction of copies of human DNA into the genetic material of another species. The resulting transgenic animals express the human gene product (protein) in the mammary gland allowing its collection and purification during lactation. The technique, which offers the opportunity to produce human proteins economically and in potentially unlimited quantities, is based on patented technology developed by the Roslin Institute in the 1980s for which PPL holds an exclusive licence.
  3. PPL’s three lead products are AAT, a fibrin sealant, and bile salt stimulated lipase (BSSL).
  4. PPL is the only company to offer a wide range of animals for transgenic protein production, including sheep, cows, rabbits, and pigs.
  5. Further information on PPL, its products, and technologies can be found at: www.ppl-therapeutics.com .