AOP Orphan Pharmaceuticals AG to acquire Selisistat

A clinical stage drug candidate for the treatment of Huntington's disease (HD)

AOP Orphan Pharmaceuticals AG announced today that it has acquired Selisistat, a highly selective SIRT1 inhibitor, which in experimental studies has previously shown potential disease modifying properties in a number of neuro-degenerative diseases. In vitro data furthermore suggest that Selisistat could have the potential to act as a chemical inducer to generate functionally active neurons.

The drug candidate has clinically been tested in Phase I (dose finding and tolerability in humans) and in Phase II (therapeutic effectiveness in a limited patient number) HD studies. Selisistat proved to be safe and tolerable. Due to the chronic nature of HD a proof of efficacy, however, will require another controlled long term Phase III study.

Apart from being a potential disease modifying treatment for neuro-degenerative diseases, the SIRT1 Inhibitor Selisistat may be of therapeutic value in a number of indications amongst them, leukemia, cancer, sepsis, and prevention of organ transplant rejection.

"Development of meaningful drugs for rare diseases does come with significant risks," said Dr. Rudolf Widmann, CEO of AOP Orphan. "Overcoming the hurdles of proving efficacy and safety in order to obtain regulatory approval nowadays is not enough anymore to bring a drug to patients in need of it. The discrepancy of making affordable drugs in an environment of accumulative administrative sophistication frequently causes research to stall. This is a regretful development for patients, physicians, pharmaceutical industry and payers."

AOP Orphan has decided to maintain the chances of the promising drug candidate Selisistat and will, after thorough evaluation of existing data and consultation with medical experts and regulators, look for partners to take up the challenge to resume Selisistat development in potential indications, because, as stated by Dr. Widmann: "Abandoning hope is not an option unless lack of efficacy or unacceptable toxicity mandate the search for alternative molecules."

About Huntington's Disease
HD disease is a hereditary brain disorder which usually manifests between age of 30 and 50 and does to various degrees affect a person´s memory, emotions, and movement. The disease symptoms are essentially caused by the accumulation of "faulty proteins in their nerve cells, which cannot be degraded and thereby cause cell damage. Existing drug therapies are merely symptomatic but Selisistat may have disease modifying properties by helping cells to reduce the toxic burden of faulty proteins and mitigate HD progression.

About AOP Orphan
AOP Orphan is a multinational company with headquarters in Vienna, Austria, focusing on clinical research, development and distribution of medicines for rare and complex diseases. The company also provides individualized and customized services to meet and accommodate the needs of physicians and patients across Central Europe, the Middle East & Asia. Currently AOP Orphan is concentrating on orphan and complex diseases in Hematology & Oncology, Cardiology & Pulmonology, and Neurology/Psychiatry & Gastroenterology.

Press contact
AOP Orphan Pharmaceuticals AG
Dr. Georg Fischer
Chief of Corporate Development
Tel: + 43 (0)1 503 72 44 15
Fax: + 43 (0)1 503 72 44 5
Email: georg.fischer(at)

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