Proneuron’s ProCord Receives FDA Orphan Drug Designation

ProCord (autologous incubated macrophage therapy), an experimental procedure for complete spinal cord injury, is currently being tested in an international, multicenter, randomized-controlled Phase II trial in the U.S. and Israel

Los Angeles, California, September 13, 2004 ---Proneuron Biotechnologies (www.proneuron.com) announced today that the FDA has granted an orphan drug designation to ProCord, autologous incubated macrophage therapy, to improve the motor and sensory neurological outcome in cases of acute spinal cord injury.

An orphan drug status is granted by The FDA Office of Orphan Products Development (OOPD) (http://www.fda.gov/orphan/oda.htm) to promote the development of products that demonstrate promise for the diagnosis and/or treatment of rare diseases or conditions. In order to qualify as an orphan drug, a product must target conditions that affect less than 200,000 people a year in the U.S. and that should offer substantially improved therapeutic benefit as compared to current medical regimens. Orphan drug designation provides for seven years of market exclusivity in the U.S. to the first entity that obtains marketing approval in the designated indication. Other benefits to the sponsor include tax credits for the costs of clinical research, reduced filing fees and preferential pre-filing regulatory guidance.

In the U.S. alone, there are an estimated 11,000 new spinal cord injuries per year. Causes include motor vehicle accidents, acts of violence, falls, sports and other occurrences. SCI trauma usually results in paralysis and other significant morbidity factors are common, including respiratory failure, and bladder and bowel dysfunction.

Proneuron is currently engaged in an international, multi-center, randomized-controlled Phase II study of ProCord in the U.S. and Israel. Patients are currently being enrolled at the following sites: Craig Hospital in Colorado, Kessler Institute for Rehabilitation with UMDNJ (Newark), New Jersey, The Mount Sinai Medical Center in New York, the Shepherd Center in Georgia and the Chaim Sheba Medical Center in Israel.

ProCord is a result of the research efforts of Professor Michal Schwartz of the Weizmann Institute of Science, who first proposed that specially treated macrophages (a certain type of white blood cell) could stimulate a wound-healing immune reaction in an immune privileged area like the central nervous system.

“It is an important milestone that the product of this research has been granted an orphan drug status by the FDA and we shall continue to rigorously investigate its potential in the current Phase II study for complete spinal cord injury,” said Proneuron CEO Mr. Nir Nimrodi.

The ProCord clinical trial is open to patients who meet eligibility criteria, including but not limited to ASIA Grade A, C5-T11, within 14 days of injury. This is a randomized-controlled clinical trial. Patients found eligible for the study are randomly assigned to either a treatment or control group, two treatment patients for every one control patient. Control patients do not undergo the procedure. All control and treatment patients receive standard spinal cord injury rehabilitation and follow-up testing for one year. It is crucial that trial site investigators are notified of a potential candidate within a few days of their spinal cord injury in order to give ample time for the clinical staff to confirm patient eligibility and complete enrollment within the 14-day window of the clinical trial.

Patient, Immediate Family of Patient and/or Physician inquiries:

24 hour a day Patient Recruitment Center:
Email: clinical.trial@proneuron.com
Telephone: 1 866 539 0767 (U.S. toll free) or 1 506 652 3486.
Fax: 1 866-214-7078
*Callers outside of the U.S., please use standard international dialing code

About Proneuron Biotechnologies

Proneuron is a privately held biopharmaceutical company developing products for the medical treatment of spinal cord injuries and other disorders of the central nervous system. Its products are based on proprietary technology for modulating the interaction between the nervous and immune systems. The company's products are currently being evaluated in several clinical studies located in the U.S., Belgium and Israel. These include: an independently managed, international, multi-center, randomized-controlled Phase II trial of ProCord, autologous incubated macrophages, an experimental procedure for spinal cord injuries and a Phase II program of Cop-1 for the treatment of Glaucoma and other neurodegenerative diseases. The latter is being managed by Teva as part of the Proneuron’s strategic collaboration for development and commercialization of Cop-1 for various neurodegenerative indications. The Company is also developing PN277 for the treatment of additional neurological diseases.

Press inquires: Marjie Hadad, Media Liaison, Proneuron, Marjie.hadad@proneuron.com; +972-54-536-5220.