AMO Pharma raises $25 million in private equity financing

AMO Pharma Limited ("AMO Pharma"), a privately held biopharmaceutical company focusing on debilitating diseases with limited or no treatment options, announced today that the company has raised $25 million in private equity financing from Woodford Investment Management ("Woodford"). This initial investment came from the Woodford Patient Capital Trust (WPCT). Funding will support Company efforts to advance two clinical-stage pipeline assets targeting rare muscular and central nervous system disorders. The financing will also be used to expand the AMO Pharma pipeline through acquisition of additional development-stage products.

"Following our successful acquisition of two promising compounds, our financing with Woodford provides us with the resources we need to continue to rapidly advance our clinical development programs while also positioning us to further expand our pipeline. Our current development programs focus on two devastating rare diseases – myotonic dystrophy and fragile X syndrome – both of which have limited treatment options and represent significant commercial opportunities in drug development," said Michael Snape, chief executive officer and a founding director of AMO Pharma. "We are especially pleased that the team at Woodford, a global leader in life science investments, has recognized the strong potential for success in both our assets and our business model at AMO Pharma."

In early 2016, AMO Pharma plans to initiate a Phase II clinical trial for the company's lead product candidate, AMO-02, in the treatment of a severe form of myotonic dystrophy (DM) known as DM1 or Steinert's disease. Myotonic dystrophy is a genetic disorder that causes skeletal and muscle weakness and dysfunction, cognitive deficits, as well as symptoms affecting other organs in the body. There are currently no approved therapies to treat DM1. In cellular and animal models of DM1 myotonic and Duchenne muscular dystrophy, as well as in muscle biopsies from patients, activity of glycogen synthase kinase 3 beta (GSK3β) has been shown to increase. AMO-02 is an inhibitor of GSK3β that has demonstrated pre-clinical efficacy in transgenic models and reversal of muscle cell deficits in patient tissue ex vivo.

AMO Pharma also plans to initiate a clinical program in fragile X syndrome with a second product candidate, AMO-01. According to the FRAXA Research Foundation, fragile X syndrome is the most common inherited cause of autism and intellectual disabilities. AMO-01 is an inhibitor of the ras-extracellular signal-regulated kinase pathway (Ras-ERK). In pre-clinical efficacy studies, AMO-01 rescued the neuronal phenotype of the Fmr1 knockout transgenic mouse model of fragile X syndrome, further supporting the hypothesis that inhibition of the Ras-ERK cascade may have therapeutic benefit in this disease.

Lisa Wittmer, chief operating officer and head of portfolio development at AMO Pharma, elaborated, "Drug developers must be able to identify, de-risk and rapidly advance promising therapies that address significant unmet needs. The team and business model developed by AMO Pharma have the clear potential to deliver important new therapies to treat diseases in underserved patient populations in the years ahead. We plan to advance our first two programs while aggressively acquiring additional high value assets attractive for development and partnering or commercialization."