Experimental Drug Seems to Slow Respiratory Decline in DMD

New data show that Catena (idebenone) has a modest effect on respiratory function, bolstering results from an earlier trial

by Amy Madsen on November 4, 2011 - 10:33am

Update (Dec. 2, 2013): In a Nov. 26, 2013, press release, Santhera Pharmaceuticals said the phase 3 trial of idebenone (brand names Catena and Raxone) in Duchenne muscular dystrophy (DMD) remains on track to deliver results during the second quarter of 2014 and is unaffected by recent regulatory developments related to drisapersen and eteplirsen, which are also drugs for DMD but which work by a completely different mechanism from that of idebenone and are being evaluated using different measurements.

Update (April 17, 2013): This story was updated to reflect that the phase 3 trial is ongoing but closed to new participants, and that the data safety monitoring board said it should continue.

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 April 2013, a Santhera-sponsored phase 3 trial of idebenone in DMD is ongoing but closed to new participants.

In an April 16, 2013, press release, Santhera announced that an independent data safety monitoring board (DSMB) had informed the company that this phase 3 study should continue as planned, since no safety issues have been detected and the trial has a "reasonable chance of achieving its primary endpoint for improving or delaying the loss of respiratory function in Duchene patients not using corticosteroids."

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