BioAxone's supporting science in the area of spinal cord injury has received attention from perhaps the most famous research proponent in that area: actor Christopher Reeve.
Privately held BioAxone, of Montreal, is based on at least 12 years of research by its chief scientific officer and co-founder, Lisa McKerracher, who was awarded the Christopher Reeve Research Medal in 2000 for her work in the area.
Spinal cord injuries might be "as complex as AIDS," said BioAxone President and CEO Pierre Caouette, who co-founded the private company with McKerracher in 2000. "We don't see a single type of drug to be the answer for all [spinal cord injuries]." But what might come to fruition ultimately are combination therapies that work for such injuries, he said.
McKerracher has focused her attention on the subject through years of research, resulting in BioAxone having three products in development that target the Rho signaling pathway, which the company said is important for regulating the cellular process involved in neuronal trauma and cancer metastasis.
"Rho as a signal is indicated in so many different diseases, so to be able to control activation or inactivation is becoming a very hot subject," said Caouette.
The company is developing Cethrin for acute spinal cord injury. The product needs to be given to a patient within 48 hours of injury, but BioAxone hopes to develop the compound so that it can be given even after patients have begun functioning in a wheelchair, Caouette said. The company said that it expects to ask the FDA for orphan drug status for the product. Research is still early, though, as BioAxone expects to complete toxicity studies with Cethrin this summer and file an investigational new drug application in the fall.
"The biggest one [goal of] the company is to get this to the people on the street as soon as possible," Caouette said.
Its second compound, Rhostatin, is in preclinical cancer testing. The company said its research indicates that Rhostatin has three blocking actions on cancer cells: it stops uncontrolled proliferation, blocks cell migration and inhibits angiogenesis. BioAxone is in the process of selecting the most appropriate cancer indication for animal testing.
With the small molecule BA-1016, the company said its research has shown that Rho is abnormally activated in ischemia, which would indicate that Rho might be an important target for neuroprotection following stroke and in neurodegenerative diseases. BA-1016 is being developed for indications in both neuroscience and cancer, and optimization of the molecule is in progress.
BioAxone's business strategy is to outlicense its products for specialized indications beyond the area of neuroscience but to retain rights in Canada. Certain potential products would be developed to Phase II in order to increase its value to potential buyers, the company said.
In March, BioAxone negotiated its first license, valued at about $30 million, with a U.S. biotech company, the name of which Caouette wouldn't reveal for competitive reasons.
The company might go fund raising this fall, looking to raise $15 million.
"Every week or two weeks we get calls from investors," he said. "I don't see any problem there at all."
To date, the company has raised about C$12 million (US$8.7 million). Also, he said the company has had some interest from big pharma companies. If BioAxone raises funds this fall, Caouette hopes to double its current 27 employees by next year. Earlier this year, BioAxone appointed Nigel Brown as chairman. Brown, who is senior vice president of business planning, technologies and markets for MDS Pharma Services, of Montreal, brings valuable experience to the company, since he has been involved in taking products from the preclinical stage onward, Caouette said.
Serendipity may come naturally to BioAxone. Caouette and McKerracher met while sailing and married a year after founding the company. The money that McKerracher received as a result of the Christopher Reeve Research Medal in 2000 she funneled into the company. They invested about C$250,000 to start the company and MedTech Partners Inc., of Ontario, infused C$600,000 of seed financing.
In July 2002, BioAxone completed a C$11.5 million financing with help from T2C2/Bio2000, Fonds FTQ, Investissement Desjardins and Innovatech, all of Montreal.
So, the company looks forward to moving ahead, particularly if it can create a product that could help thousands with spinal cord injuries.
"We all dream of making a difference in the medical world," Caouette said, but noted, "We also have to be realistic."