This morning, Prana Biotechnology (PRAN) announced that the European Medicines Agency’s Committee for Orphan Medicinal Products has adopted a positive opinion recommending designation of PBT2 for the treatment of Huntington disease as an orphan medicinal product to the European Commission. In September last year the US Food and Drug Administration granted PBT2 orphan drug status for the treatment of Huntington disease.
Huntington’s disease is a neurodegenerative genetic disorder that affects muscle coordination and leads to cognitive decline and behavioral symptoms. The Huntington’s Outreach Project for Education estimates there are between 40-100 cases of Huntington disease per million people in Europe.
Prana’s PBT2 has also entered Phase trials as a novel therapy for Alzheimer’s. Top line results for the Phase 2 “IMAGINE” trial of PBT2 in patients with Alzheimer’s disease were released in March 2014. Furthermore, its PBT434 has been advanced as a treatment for Parkinson’s disease, with research funded by the Michael J Fox Foundation. PBT434 has advanced through discovery, chemistry and screening and is currently in the final stage of preclinical testing. There is currently no cure for Parkinson’s disease but a range of treatments do exist, although they can cost up to $1,500 per year for medication and $100,000 for therapeutic surgery. Drug treatments work by restoring dopamine levels in the brain, but are known to have side effects including involuntary movements and compulsive behavior.
Other companies like Auspex Pharmaceuticals Inc (ASPX) and Cellular Dynamics International, Inc. (ICEL) have also focused efforts on Huntington’s Disease and other neurological related therapies. Significantly higher in price and in further phase trials as compared to Prana, these stocks have seen significant increases in price over the last few months. Auspex, for example, has more than doubled since December. According to several reports from last March, in the latest acquisition in the pharmaceuticals industry, Teva (TEVA) will pay $101 a share for Auspex. Including debt, the deal is worth $3.5 billion, and the boards of both companies have approved it.
Further, Cellular Dynamics has also realized a “double down” in price after Japanese giant Fujifilm (FUJIY), a photography hardware manufacturer that branched into biotech, agreed to pay $307 million in cash, or $16.50 a share, for Cellular Dynamics. The company has developed a technology platform for industrial-scale production of human cells. According to reports, Fujifilm plans to combine the company with its own regenerative-medicine technology and expand its drug-discovery services.
Cellular Dynamics initially gained after it went public at 12 in July 2013, but dropped to near 5 by early this year. Current share prices for ICEL have been hovering between $16.40 and $16.90.