Experimental Drug Seems to Slow Respiratory Decline in DMD

Update (Sept. 19, 2012):This story was updated to reflect the fact that the phase 3 trial of idebenone in Duchenne MD remains open and that new sites are now participating.

Santhera Pharmaceuticals presented clinical data Oct. 21, 2011, indicating that the experimental drug idebenone (brand name Catena) modestly slows the decline in some measurements of respiratory function in people who have Duchenne muscular dystrophy (DMD).

The company, headquartered in Liestal, Switzerland, presented the data at the 16th International Congress of the World Muscle Society in Portugal.

Catena is a small-molecule therapy and is administered orally. It's thought to work by helping preserve function in the energy-producing parts of cells known as mitochondria. This, in turn, could protect cells from a type of damage known as oxidative stress.

The new data are based on clinical observations of 19 trial participants in the company's phase 2, two-year, open-label extension study of Catena in DMD, called DELPHI-E. The results are in agreement with and confirm data generated in a phase 2, one-year study called DELPHI, in which an idebenone group and placebo group were compared. Both studies were conducted in Belgium.

Efficacy was measured as change from baseline in the respiratory function tests peak expiratory flow (PEF), forced vital capacity (FVC) and maximum inspiratory mouth pressures (MIP). Preliminary analysis reported in May 2011 indicated that the rates of decline in PEF and MIP were slower when participants received Catena during the extension study, as compared to a period where they did not receive Catena. There was no difference in the rate of decline in FVC measurements, on or off Catena.

As of November 2011, a Santhera-sponsored phase 3 trial of Catena in DMD was under way in Philadelpia and several European sites. To learn more, see Phase 3 trial of idebenone in Duchenne MD still open.

As of September 2012, the phase 3 trial remains open and has expanded to several additional U.S. and non-U.S. sites. See Phase 3 Study of Idebenone in Duchenne Muscular Dystrophy (DELOS); or enter NCT01027884 in the search box at ClinicalTrials.gov.

As of Sept. 19, the Seattle site is not yet posted on ClinicalTrials.gov website. Contact information for that site is:

United States, Washington state
Seattle Children's Hospital
Seattle, Washington, United States
Contact: Ana Kobayashi, ana.kobayashi@seattlechildrens.org
Principal Investigator: Susan Apkon, MD

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