Penwest Pharmaceuticals Co., as part of a relatively new strategy to target niche neurological diseases, gained rights from Edison Pharmaceuticals Inc. to technology being developed for treating mitochondrial disorders.

Penwest gained exclusive worldwide rights to Edison's EPI-A0001 in all fields of use, and plans to take the product into clinical development next year for treating mitochondrial respiratory chain diseases, said Jennifer Good, president and CEO of Penwest. The indications involve neurological disorders resulting from defects in cellular energy metabolism.

Most of the focus at Penwest, of Danbury, Conn., has been on applying its formulations and delivery technologies to new and existing drugs. One example is Opana ER, an oxymorphone-based pain product formulated with Penwest's extended-release technology that Endo Pharmaceuticals Inc. launched last year.

Good told BioWorld Today the plan at Penwest is to continue its reformulation and delivery work while separately building a portfolio of new chemical entities targeting neurological conditions, a strategy the company embarked on about a year ago.

Edison, of San Jose, Calif., is entitled to an up-front payment and a $1 million loan from Penwest, which also will provide research funding over the next 18 months. Those payments could total $7.5 million, with the majority going toward sponsored research, Good said.

Penwest then would have an option to extend the reach an additional 18 months. As part of the deal, it also got exclusive rights to develop a second drug candidate from Edison during the sponsored-research term. Edison is entitled to certain undisclosed additional option payments, as well as milestone and royalty payments.

Guy Miller, chairman and CEO of Edison, told BioWorld Today there is a lot of interest now in the area of antioxidants and neurology, with the question being whether anyone is "going to crack the code on converting an antioxidant into a drug. We at Edison have a really keen understanding of how antioxidants work and how to convert them into drugs.

"We teamed with Penwest because they have a very clear set of skills in neurology and drug development," Miller said. "We believe the combination of Penwest's capital and their passioned research and development team, and Edison's skills in redox drugs, is a winning formula to crack the code that people have been chiseling at for the better part of 20 years."

Edison was founded in 2005 with an initial plan to develop the EPI-A0001 compound series for orphan respiratory chain diseases, such as Friedreich's ataxia, Leber's hereditary optic neuropathy, coenzyme Q(10) deficiency and MELAS syndrome, or mitochondrial encephalopathy, lactic acidosis and stroke-like symptoms.

All such diseases involve genetic changes in enzymes that affect mitochondrial function. EPI-A0001 is an analogue of CoQ(10) designed to have improved drug-like properties.

Edison described the respiratory chain, named because the last link of the chain involves oxygen consumption, as an energy currency exchange device. Its platform focuses on redox medicinal chemistry, an oxidation-reduction approach.

Miller said Edison was approached by five prospective partners, with Penwest being "by far the most competent in terms of having a cogent strategy" for bringing technologies in, moving drugs forward and building a commercialization infrastructure.

He said the deal was not done because Edison needed the money, as evidenced by its plan to announce a Series B financing round next week.

Instead, he said, the deal was done because working with Penwest was the best way to move the programs forward.

Edison has forged relationships, and received grant funding, from a number of nonprofit groups, including the Muscular Dystrophy Association, Friedreich's Ataxia Research Alliance and Seek A Miracle.

The technology also may have applicability beyond orphan respiratory chain diseases. Edison last year partnered with Los Angeles-based CHDI Inc., a nonprofit organization that supports efforts in Huntington's disease. They plan to develop analogues of CoQ(10) targeted to reach the brain and address the mitochondrial component of Huntington's disease.

Miller said in addition to other neurodegenerative diseases, Edison - along now with Penwest - plans to study the technology in metabolic syndromes, inflammatory diseases and ischemic conditions.