Proneuron's Therapy For Post-Spinal Cord Injury Gets Orphan
Drug Status
Dow Jones Venture Wire, September 14, 2004
Gene/Cell Therapies
Proneuron Biotechnologies Inc., a developer of central
nervous system treatments through enhanced immune response, said
its incubated macrophage therapy for acute spinal cord injuries
received orphan drug status from the Food and Drug
Administration.
An orphan drug is one that treats a rare
disease and a limited amount of the population - fewer than
200,000 people according to the FDA. Orphan drug status provides
seven years of U.S. market exclusivity.
Proneuron estimates
around 11,000 new spinal cord injuries occur in the U.S. each
year.
Chief Executive Nir Nimrodi said the development of this
therapy is a response to the question, "How can we properly
boost the immune system in the CNS following an injury [to the
spinal cord]?" The treatment involves the extraction of
macrophages from a patient and the co-incubation of the
extracted cells with molecules from the patient's skin. This
alters the phenotype of macrophages to exhibit regenerative
qualities similar to skin tissue. The macrophages are then
injected back into the patient, and have "neuroprotective"
effects.
A multi-site Phase II study is currently underway for
the therapy, and Nimrodi expects enrollment for the study to be
complete during the first quarter of 2005.
By properly
stimulating the immune system, you can allow a certain level of
repair within the central nervous system," Nimrodi said of
Proneuron's mission, adding that the immune system is not as
active in the CNS as it is in other tissues.
Also in the
company's pipeline is an antigen that helps boost T-cell
activity in the brain following the development of acute trauma
or chronic disorders such as glaucoma and Alzheimer's disease.
The compound, partnered with Teva Pharmaceuticals, is currently
in Phase I/II trials for glaucoma. Nimrodi said Proneuron has
already received $20 million in equity investments and up-front
and milestone payments from the deal, and is entitled to receive
additional milestone and royalty payments.
Nimrodi expects a
third treatment, which can "harness the immune system by
down-regulating the activity of regulatory T-cells," and
therefore unleash an immune response, to enter the clinic in
early 2005. In preclinical studies, he said it has shown results
in treating stroke and other acute CNS diseases.
Los
Angeles-based Proneuron recently acquired a $5 million
convertible note in July from Tamir Fishman Ventures. It has
raised $25 million of funding from private investors, including
Hudson Venture Partners, Giza Venture Capital, Israel Infinity
Venture Capital, J.P. Morgan Capital, Mezam Capital Funds
Management and Pitango Venture Capital.
Nimrodi said the
company is not actively seeking additional funding at this time.
Proneuron has 45 employees.
