Orphan diseases on the viewfinder
Transmission electron microscopy image of mitochondria in heart muscle. © EPFL / CIME
Mitokyne, a start-up whose co-founder Johan Auwerx is a professor at EPFL, has just signed a 45 million dollars research agreement with Astellas, a Japanese pharmaceutical company. Based in Cambridge, near MIT, Mitokyne focuses on developing molecules for treating rare diseases that affect mitochondrial functions.
Nicknamed as the "cellular power plants", mitochondria have a fundamental role in metabolism. They are also involved in the aging processes and muscular performance.
At EPFL, the Laboratory of Integrated Systems Physiology (LISP) is specialized in the study of such cellular organelles. Its director, Johan Auwerx, is one of the co-founders of Mitokyne, a spin-off whose goal is to develop therapeutic molecules targeting mitochondria. He founded it together with three distinguished colleagues: Bob Horwitz (Nobel Prize) from MIT, Andrew Dillin from the University of California at Berkeley and Ron Evans (Lasker Award) from the Salk Institute for Biological Studies. The start-up mainly takes aim at orphan diseases related to mitochondrial dysfunction.
From extremely rare cases to a global scale
According to Johan Auwerx, "the interest in mitochondria and the rare diseases associated to them opens extremely interesting perspectives for developing drugs that could be able to tackle more common diseases." That is how drugs for lowering cholesterol – today’s best-selling medications – were developed. "Statins were originally developed to treat a very rare and violent form of hypercholesterolemia, said the researcher. Only afterwards were they prescribed for treating a less severe form of the disease – for a current turnover of 35 billion dollars."
Mitokyne’s objective is to fight mitochondrial related diseases, which can cause blindness, deafness, chronic muscle weakness or neurological disorders. "The molecules that we will develop for treating extreme forms of these diseases are very likely to also work on less severe ones or on types occurring at an advanced age, such as glaucoma, hearing loss, sarcopenia and Alzheimer’s and Parkinson's diseases, estimated Auwerx. Another advantage of this approach lies in the fact that the drug development process is faster in the case of orphan diseases, as regulations are more relaxed.
Roadmap to 775 million dollars
This "fast track" to new molecules development has attracted a heavyweight investor. Mitokyne had barely been officially registered in early October when it announced the signature of a research agreement with Astellas, a Japanese pharmaceutical company. The latter is investing 45 million dollars in Mitokyne’s takeoff over a 5 years period.
At the end of this term, if the company’s goals – that is, the development of three molecules – are met, Astellas and Mitokyne have already agreed to carry out a full acquisition for an amount up to 730 million dollars. "By establishing a roadmap up to the company’s "output" we are able to avoid wasting time and energy in fundraising and – possibly – in entering the Stock Market, said Auwerx. This is a completely original approach to venture capital investing. It is quite promising since from the outset it establishes a general overview for the company’s development as well as some form of security to its investors."