CSL progresses therapies for haemophilia

CSL has presented promising results from a new group of therapies for the treatment of haemophilia at the International Society of Thrombosis and Haemostasis congress in Amsterdam this week.

Haemophilia is an inherited disorder caused by the failure to produce certain clotting factors required for blood clotting.

People with haemophilia suffer prolonged or spontaneous internal bleeding, especially in the muscles and joints, which can lead to severe arthritis, chronic pain and disability. Clotting factor replacement therapy is required up to three times a week to reduce these bleeding episodes.

CSL has a long history of developing factor replacement therapies, derived from human plasma, for patients who are factor deficient.

Over the past several years, CSL has been developing recombinant versions of several clotting factors including some fused with the plasma protein albumin. Trials of these albumin fusion clotting factors have shown the technology extends the effect of the treatment.

“We are very encouraged by the results of our clinical studies, which aim to develop longer-acting and more effective products for people with haemophilia,” said Dr Russell Basser, CSL Senior Vice President, Global Clinical Research and Development.

“The investigation of novel ways to extend the life of recombinant clotting factors in patients may result in a significant reduction in the number of weekly infusions for people with haemophilia.”

Further clinical studies are being planned for other recombinant clotting factors in development and are expected to be the first therapies produced in CSL Behring’s new Biotechnology Facility in Broadmeadows, Melbourne.

“This world-class facility is the largest of its kind in Australia and was constructed to support the late stage development of novel recombinant therapies in our R&D pipeline,” said General Manager of CSL Behring Australia, Dr Simon Green.

The recently completed facility is currently producing test batches of recombinant proteins and is on track to start manufacture of clinical trial material early next year.

A solid R&D pipeline bodes well for new Managing Director and Chief Executive Officer Paul Perreault who took up the helm at CSL from 1 July 2013. Previous chief executive Brian McNamee retired at the end of last month.

CSL shares were trading at $61.25 at about 10 am on Thursday.