Trophos SA Receives Grant From The Michael J. Fox Foundation To Evaluate Compounds In Parkinson's Disease Model

11 Feb 2008
http://www.medicalnewstoday.com/articles/96853.php

Trophos SA, a biopharmaceutical company specializing in the discovery and development of drugs for neurological disorders, announces that The Michael J. Fox Foundation (MJFF) has awarded the company a Therapeutics Development Initiative grant to pursue the evaluation of compounds preventing mitochondrial dysfunction to treat Parkinson's disease (PD).

The project aims to establish the ability of two Trophos compounds to arrest or prevent the early PD-like behavioral changes observed in a preclinical model overexpressing human alpha-synuclein. Mutations in alpha-synuclein cause familial PD and the protein accumulates in affected neurons in sporadic forms of the disease. The model has been extensively studied by Marie-Françoise Chesselet, MD, PhD, and her team at UCLA, who will conduct the project in collaboration with Trophos. Dr. Chesselet is the Charles Markham Professor of Neurology at UCLA and Chair of the Department of Neurobiology. She has an extensive background in the basic science of neurological disorders and is a world expert on the biology of central nervous system (CNS) diseases such as PD.

PD is caused by a broad pathology, including the death of brain dopaminergic neurons that are essential for controlling movement. While the exact cause of PD is not known, evidence suggests that mitochondrial malfunction occurs in stressed dopaminergic neurons. Several environmental toxins that produce symptoms remarkably like PD and several genes associated with familial PD lead to mitochondrial malfunction. The team at UCLA has documented early PD-like behavioral changes in the preclinical model overexpressing human alpha-synuclein.

Trophos has identified a family of compounds that enhance the survival of motor neurons in models of motor neuron degeneration. These compounds bind to mitochondrial proteins and thereby maintain mitochondrial function in cells subjected to various types of stress. The two Trophos compounds to be studied have been extensively characterized, and one of them, TRO19622, has already demonstrated a very good safety profile in human clinical trials. Should either compound show potential therapeutic benefit in the PD model, it could progress to clinical testing in patients with PD.

"We are very pleased that Trophos has been awarded funding under the Therapeutics Development Initiative for the preclinical evaluation of Trophos compounds in the PD model," said Rebecca Pruss, CSO at Trophos. "Evidence that a Trophos compound that targets mitochondria has a beneficial effect could establish a new therapeutic approach to the treatment of PD and potentially other neurodegenerative diseases."

"By funding industry partners directly, MJFF's TDI initiative helps us to optimize and advance the most promising treatments that might otherwise get stuck at the pre-clinical stage for lack of funding," said Katie Hood, CEO of The Michael J. Fox Foundation. "The grant to Trophos is an excellent example of how our capital, while comparatively modest, can serve as a 'carrot' to leverage companies' expertise and infrastructure and speed the development of treatments that could have an immense impact on patients' quality of life."

About Trophos

Trophos is a biopharmaceutical company committed to the discovery and development of novel therapeutic compounds to treat neurological disorders and other diseases with high unmet medical needs. The Trophos discovery strategy has enabled it to develop a proprietary portfolio of products, such as our lead product TRO19622 & drug candidate TRO40303, that confer a survival benefit upon both neuronal and non-neuronal cells. This is achieved through a mitochondria-based mechanism of action with a robust therapeutic rationale predicted to exhibit a therapeutic benefit in diseases such as neuropathic pain, ischemia-reperfusion injury and hepatotoxicity. The company is focusing its efforts on the orphan indicates ALS, SMA and Huntington's disease, while seeking to establish clinical proof of concept and partnerships in other indications, such as PD. The company is currently performing two clinical studies with TRO19622, a Phase IIa trial in painful diabetic neuropathy and a Phase Ib study in SMA.

Trophos was founded in 1999, is based in Marseille, France and currently has 32 employees.