Catabasis lifted by early-stage muscular dystrophy data, but bigger test is coming soon

Top-line results from a Phase II trial in boys with DMD are due shortly(Pixabay)

Just months after writing off a cholesterol drug, Catabasis is back with the first data on its Duchenne muscular dystrophy candidate.

Edasalonexent (CAT-1004) is an oral NF-kB inhibitor and—unlike other DMD therapies such as Sarepta's injectable Exondys 51 (eteplirsen) which was approved last year for a specific genetically-defined group of patients—could potentially be used in patients regardless of their underlying mutation, says the company.

The phase 1 trial showed that Catabasis' drug was found to be safe when given to adults and reduced levels of NF-kb, an inflammatory mediator that the company believes is activated in the very early stages of DMD and is critical to the progressive muscle degeneration seen in the disease.  The data are published in the Journal of Clinical Pharmacology.

So far so good, but a bigger test for the biotech will come when it reports top-line results from a phase 2 trial in boys with DMD—called MoveDMD—in the next few weeks. Earlier this month, the company reported preclinical data showing that the drug could interrupt the disease process in two animal models (dog and mouse).

Catabasis is hoping for a broad approval that could see its drug being used in combination with Exondys 51 and other DMD drug candidates, and last September agreed to test alongside Sarepta's drug. At the time, Catabasis' chief executive Jill Milne said that while edasalonexent has potential as a monotherapy, "we think there is benefit to exploring innovative ways to make the most meaningful difference in this devastating disease."

Last June, Catabasis shares were decimated after the company was forced to abandon a mid-stage program for former lead drug CAT-2054 in patients with high cholesterol levels.

The latest edasalonexent data drove shares up 20% and "support edasalonexent development in DMD and potentially other diseases," said Catabasis' chief medical officer Joanne Donovan, who added the Phase II results in DMD patients should be ready "in the first half of Q1 2017."

That's actually a little later than hoped, as Catabasis had previously indicated it hoped to have top-line data available in time for the JP Morgan conference last week. The biotech previously said positive data could allow the company to move quickly ahead with a phase 3 trial, likely before the end of 2017.

Catabasis is also developing the NF-kB inhibitor for Friedreich’s ataxia, amyotrophic lateral sclerosis and cystic fibrosis.