Positive safety data for retinitis pigmentosa drug

Amarantus Bioscience announced positive ocular toxicology data for the intravitreal administration of MANF (mesencephalic astrocyte-derived neurotrophic factor). The company hopes to develop MANF for orphan diseases such as retinitis pigmentosa.

"The results seen in this ocular tolerance study are encouraging for an initial clinical focus on MANF in orphan ocular diseases,” said Gerald E. Commissiong, president and CEO of Amarantus. “This data will support the preparation of our orphan drug designation application with the FDA for MANF in one or more ocular disorders in the fourth quarter, including retinitis pigmentosa."

The ocular tolerance study evaluated the safety of a single intravitreal administration of MANF in pigmented rabbits over a 15 day period at dosing levels expected to be above therapeutically relevant dosing levels in human. The drug was well tolerated both macroscopically and microscopically. No treatment or administration-related effects were observed on body weight, clinical observations or ophthalmic examinations.

MANF is believed to have broad potential because it is a naturally-occurring protein produced by the body for the purpose of reducing and preventing apoptosis in response to injury or disease, via the unfolded protein response of the endoplasmic reticulum. By manufacturing MANF and administering it to the body, Amarantus is seeking to use a regenerative medicine approach to assist the body with higher quantities of MANF when needed.