Rhenovia launches drug discovery programs in rare, orphan and neglected neurodegenerative diseases

Rhenovia Pharma, a leading biotech company in biosimulation applied to the discovery of new medications to treat neurodegenerative, neurological and psychiatric diseases, announces today that it has initiated drug research programs in rare, orphan and neglected diseases.

Rare diseases are defined as those affecting fewer than one in 2,000. Eighty per cent are caused by genetic defects but they also include rare forms of cancer, auto-immune disorders, congenital malformations, infectious diseases and intoxications. France’s Ministry of Health data suggest that there are nearly 7,000 rare diseases. Europe alone accounts for some 25 million sufferers.


No cures exist for most rare diseases. The only currently available treatments are those that improve the quality of life. The annual Rare Diseases Day, which took place this year on February 29, extends the international recognition of these diseases with the aim of improving diagnosis and treatment.

Rhenovia’s decision to expand its expertise to cover rare, orphan and neglected diseases is based on the need to respond to this pressing challenge. Its initial focus will be on Huntington’s disease (HD) and Duchenne muscular dystrophy (DMD).

As a first step, Rhenovia is building a new biosimulation platform (RHENOMS(TM) STRI) aimed at modeling the complex interplay between biological mechanisms in striatum, the brain region that is most affected in HD. HD is a fatal, rare neurodegenerative disease that is particularly difficult to treat because of the very broad spectrum of symptoms it causes, involving involuntary movement disorders, cognitive deficits and psychiatric manifestations.

”It is exactly because of this variety and often opposite syndromes that the biosimulation approach is probably the most appropriate strategy in the search for new treatments,” said Doctor Serge Bischoff, president and CEO of Rhenovia. “It will allow us to integrate the complexity of the biological systems affected by HD and to address the multifactorial nature of this disease.”

The objective of Rhenovia’s HD program is to provide new tools and solutions to optimize the Drug Discovery and Development (DD&D) process and accelerate the search for new treatment strategies and medications, not only for relieving HD patients from their symptoms, but also for modifying the course of their illness. A further objective is to consolidate Rhenovia’s own pipeline of drug candidates.

A second direction in Rhenovia’s rare, orphan and neglected disease program is the development of a modeling and simulation platform designed to better understand the basic mechanisms underlying cognitive impairment and mental affects associated with muscular dystrophies with a first focus on DMD.

“This choice is dictated by our privileged partnership with the French Association against Myopathies and also by the urgent medical need of DMD patients for specific treatments of syndromes linked to the deterioration of some brain functions,” added Bischoff. “Strengthening our efforts in the domain of neglected, and especially rare diseases, will not only contribute to a better understanding of the biological mechanisms and pathological manifestations underlying these diseases, but will also markedly consolidate the biosimulation technology mastered by Rhenovia.

“The huge progress made by Rhenovia’s neuroscientists, chemists and informatics, physics and mathematics engineers in the last two years means we can expect significant advances by simulating deficits of rare diseases to help to shed light on the neurodevelopmental (such as autism), neurodegenerative (such as Alzheimer’s) and psychiatric diseases (such as depression and schizophrenia) affecting broader patient populations.”

Rhenovia recently presented initial data on how the firm approaches HD syndromes by biosimulation at the seventh Annual Huntington’s Diseases Therapeutics Conference held in Palm Springs (California, USA). The poster entitled ‘Use of biosimulation to facilitate drug discovery in Huntington’s disease’ can be downloaded from Rhenovia’s website http://www.rhenovia.com section Publications.

About Rhenovia Pharma SAS

Rhenovia Pharma SAS is a biotechnology company that is specialized in the development of innovative solutions to improve the prevention and the treatment of Alzheimer’s and other diseases of the central and peripheral nervous systems. In the recent years, Rhenovia became a fully integrated Research and Development firm with technologies facilitating DD&D, an emerging pipeline of drug candidates, a prototype of a unique rate controlled transdermal delivery system, and a highly sensitive analytical technique of protein purification foreseen to be applied to the identification of biomarkers of drug efficacy. Rhenovia is a world leader in the biosimulation of nervous system mechanisms in support of the drug discovery process. Its unique nervous transmission modeling technology makes it possible to reproduce on a computer the cellular and molecular mechanisms involved in learning, memory, and a whole range of other impaired brain functions associated with neurological diseases such as epilepsy, neurodegenerative disorders like Alzheimer’s or Parkinson’s, and psychiatric indications such as schizophrenia and depression. Through its Rhenovia Drug Development Optimization Services (RHEDDOS) package, the company makes its technology available to biotechnology and pharmaceutical companies seeking to extend the life cycle of their medicines, develop new drug combinations, or identify and validate new targets for therapeutic compounds.

Rhenovia is based in Mulhouse, France and Cambridge, MA, USA, and employs 20 people in France, Switzerland, Germany and the USA. Its French headquarters were founded in 2007 for the purpose of developing new technologies for optimizing the drug discovery and development process in the biopharmaceutical industry. The company has raised total funding of some EUR 4.5 million to date through commercial contracts, French and European calls-for-projects and public tenders, as well as from investors (including Alsace Business Angels and SODIV, a regional venture capital fund), state agencies and seed capital funds, in addition to the equity capital provided by its founders. For further information, please visit: http://www.rhenovia.com

Mark Tidmarsh