Amarantus Bioscience Holdings Inc (AMBS)

[Solantey]

Amarantus Receives Orphan Drug Designation for MANF in Retinitis Pigmentosa

http://bionapcfa.blogspot.com

From the article:

Companies that are marketing drugs targeting ocular diseases include Regeneron, Sanofi, Novartis/Alcon, Pfizer, Roche, Bayer, and Santen. We believe any one of these companies may be interested in an early-stage research collaboration with Amarantus around MANF. Beyond Orphan indications, MANF may also show utility in Glaucoma. Glaucoma affects approximately 4 million people in the U.S. alone. It's a $3 billion market. Data presented at AVRO and TOD provide the basis for moving MANF into human clinical studies in 2015. This creates a very interesting development pathway for Amarantus and a potential development partner - i.e. secure market exclusivity and aggressive pricing with Orphan Drug indications such as RP and RAO, then expand the approval to blockbuster markets like Glaucoma.

The company is currently wrapping up translational development and preclinical toxicology work on MANF prior to filing the first investigational new drug (IND) application to being human clinical studies next year. If Phase 1 clinical studies go well with MANF in RP, we expect the company to expand research into new indications and seek a larger development and commercialization partner that would come with a handsome upfront payment and backend milestones and royalties.