Forest Laboratories Inc and PAION GmbH have entered into an agreement for the development and marketing of PAION's product, desmoteplase, in the US and Canada, a release from Forest said.

Desmoteplase, a novel plasminogen activator, or blood clot-dissolving agent, is currently under development in the US and Europe for treatment of acute ischemic stroke.

Forest and PAION entered into the agreement on June 30, 2004 and Forest made an undisclosed upfront payment to PAION on that date. Under the agreement, PAION will receive milestone payments and a royalty based on sales, and Forest will fund all continuing clinical development activities for the US and Canadian markets. Forest will be responsible for regulatory and sales and marketing activities in the US and Canada and will have development and marketing rights to other indications of the product in these territories. PAION retains commercial rights in Europe, Japan and the rest of the world. Desmoteplase has several issued composition of matter patents, including some that do not expire in the US until 2015, with the potential for extensions.

PAION and Forest will be finalizing discussions with the US FDA regarding study protocols to serve as the basis for a new Biologics License Application (BLA) filing. It is anticipated that a phase IIb study will be initiated in the third calendar quarter of this year.

Positive results from a phase II study showed that the compound has the potential to treat patients up to nine hours after the onset of stroke symptoms. The only currently available clot-dissolving agent must be administered within three hours of symptom onset; however, the majority of stroke patients arrive at the hospital outside that treatment window. At present, only 11 per cent of ischemic stroke patients are eligible for the treatment and fewer than four per cent actually receive it. Desmoteplase, with a longer treatment window, could expand the number of patients who receive clot-dissolving therapy.

Desmoteplase was recently granted fast track status by the FDA, a designation granted for drugs that address an unmet medical need in life-threatening indications. Fast track designation allows the submission of portions of the application for approval in advance of the final section becoming available ("Rolling Biologics License Application"), and serves as the basis of an expedited review by the FDA, generally within six months of the filing date.

If the trials are successful, it is possible that a BLA for desmoteplase would be submitted to the FDA as early as 2007. Howard Solomon, chairman and CEO of Forest Laboratories, Inc said, "We are extremely pleased with our agreement with PAION GmbH. Desmoteplase is another late stage product to add to our pipeline of both late and early stage products. It is also our first biologic product, an important category of products that we have not participated in heretofore."

Wolfgang Soehngen, CEO of PAION commented, "The income from this agreement will secure the development for desmoteplase until approval. We have selected Forest for its proven development and regulatory expertise and its track record to successfully bring Central Nervous System products to market. We were especially impressed by the speed and pragmatism of decision making. The enthusiasm for this difficult indication from both the marketing and development colleagues, as well as the management at Forest, will be a key success factor for the collaboration. This is so important, since the successful development and launch of desmoteplase will require intensive educational efforts to increase stroke awareness and to overcome existing treatment barriers."

Desmoteplase, first in a new class of plasminogen activators, is a genetically engineered version of a clot-dissolving protein found in the saliva of the vampire bat Desmodus rotundus. It possesses high fibrin selectivity, allowing it to dissolve a clot locally without affecting the blood coagulation system, which is thought to potentially reduce the risk of intracranial bleeding (a common risk when administering blood clot-dissolvers) as compared to less fibrin-specific plasminogen activators.