M0002

Advanced compound for ascites

M0002, a compound which is tested for the treatment of ascites, is planned to enter a Phase IIb study in the first half of 2010. The compound is an aquaretic, or next generation diuretic, which is developed as a once daily oral treatment.

Ascites is a condition characterised by the accumulation of fluid in the abdomen cavity in patients with impaired functioning of the liver due to conditions such as hepatitis C, hepatitis B, alcoholism or other diseases such as cancer.

Treatment for ascites with current diuretics is not always effective (especially in patients with tense and refractory ascites).

This leads to significant mortality and morbidity and the need for invasive procedures to remove fluid, or liver transplantation as a last resort.

Potential market

With approximately one million patients in the US and Europe not getting adequate relief from diuretics, Movetis believe that there is a need for a more effective treatment. M0002 is an orally-active selective vasopressin-2 receptor antagonist and a member of a new class of compounds, known as aquaretics, which inhibit water re-absorption in the kidneys. Thus the compound induces free-water clearance without any loss of sodium.

It works by specifically inhibiting vasopressin, which promotes the re-absorption of water in the kidneys. By doing this, V2 antagonists stimulate the excretion of water, without disturbing the physiological process of electrolyte re-absorption in the kidneys. In contrast to diuretics, the current standard of care for moderate or non-tense ascites, M0002 is less likely to be associated with potential electrolyte imbalances and the related life-threatening complications.

Development status

The compound is believed to be effective in patients with tense and refractory ascites, where diuretics fail to provide sufficient relief. In addition, preliminary data suggests that M0002 has a favourable drug-drug interaction profile in likely contrast to other V2 antagonists in development. The early data also suggest a favourable cardiovascular profile. Based on M0002’s novel mechanism of action, we believe that, for the longer term, this class of compounds may have the potential to treat a variety of stages of ascites, alone or in combination with diuretics.


In terms of testing, M0002 is expected to progress into a Phase IIb study, following positive results from a Phase IIa multiple-dosing trial. Movetis is considering sharing some of the future development cost and risk of M0002 with a development and commercial partner.

Commercial rights

Movetis has exclusive worldwide rights for the drug candidate for all indications. For the markets outside Europe, Movetis is considering out-licensing rights to a partner.