Neil Woodford gambles $25M on a rare genetic disease startup

Neil Woodford

Neil Woodford's Patient Capital Trust has blessed the British-American biotech hybrid AMO Pharma with a $25 million investment to help fund development of a small pipeline of therapies for rare genetic disorders.

AMO was set up by Michael Snape, a faculty member at Case Western who has put two drugs in the clinic at the upstart. The most advanced is AMO-02, which is being studied as a new treatment for myotonic dystrophy, with additional potential in CNS, neuromuscular and cancer indications. A Phase II for that therapy is expected to get started in early 2016. There's also a Phase I/II trial for its 01 drug, which is designed to treat Fragile X syndrome, a tough target that has defied earlier attempts by other biotechs.

AMO-02 blocks GSK3β, posting encouraging results for the company in preclinical animal and tissue studies, with evidence of a reversal of muscle cell deficits in patient tissue ex vivo.

Industry observers have been paying close attention to Woodford's biotech investment strategy. Backed with a sterling reputation in the U.K. investment circles, he's been able to raise well over $1 billion to start backing biopharma companies. And he's taken a transatlantic approach, looking to British as well as American companies.

AMO, which has offices in Philadelphia and London, evidently was launched back in February of this year, according to the website. And there are plans in the works for the company to acquire more drugs for its pipeline, according to their very first press release.

- here's the release