1st Patient Dosed in Pilot Trial of Vamorolone for Becker MD

Medication expected to work like corticosteroids but with fewer side effects

Lindsey Shapiro, PhD avatar

by Lindsey Shapiro, PhD |

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The first patient has been dosed in a pilot study evaluating vamorolone as a potential treatment for Becker muscular dystrophy (BMD).

The steroid medication has been under development by ReveraGen BioPharma as a possible therapy for other types of muscular dystrophy.

Now, a Phase 2 trial (NCT05166109) will evaluate the safety, tolerability, and exploratory efficacy of vamorolone against a placebo in up to 39 males with BMD, ages 18-64.

The study plans to enroll patients at sites in Pittsburgh, Pennsylvania, and Padova, Italy. Recruitment is listed as active at the study center in Pittsburgh.

“There are currently no approved drugs for BMD in any country, and [few] drugs in clinical development for this indication in the USA or Europe,” Eric Hoffman, PhD, president and CEO of ReveraGen, said in a press release.

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Meanwhile, ReveraGen and Santhera Pharmaceuticals — which acquired global rights to vamorolone in 2020 — are planning to seek the therapy’s approval in the U.S. for Duchenne muscular dystrophy (DMD).

Vamorolone development for MDs

The two companies are basing that approval application, to the U.S. Food and Drug Administration (FDA), on positive findings from another Phase 2 trial.

“Vamorolone has shown efficacy in the pivotal VISION-DMD study in Duchenne muscular dystrophy (DMD), a more severe but related disease, and, based on these findings and its mechanisms of action, this developmental compound may show a benefit in BMD,” Hoffman said.

Corticosteroids are the standard of care for muscular dystrophy patients, given their ability to manage inflammation and strengthen muscles. However, these medications often are accompanied by a number of side effects, including excessive weight gain, behavioral changes, and high blood pressure.

Vamorolone is a steroid medication that targets the same receptors as corticosteroids do, but interacts differently with them. In doing so, it’s thought to maintain a similar anti-inflammatory profile as corticosteroids, but with fewer accompanying side effects.

“The treatment of BMD is lacking standard of care recommendations albeit there is a high unmet medical need, and about one fifth of BMD patients use some chronic steroid dosing which is often not well tolerated,” said Shabir Hasham, MD, chief medical officer and head of global medical affairs at Santhera.

“Vamorolone could address some important safety concerns that may lead to poor tolerability or early treatment discontinuation, and thus may potentially represent a novel therapeutic approach to the treatment of BMD as a chronic therapy,” Hasham said.

Preclinical data have shown that vamorolone can increase levels of dystrophin, a protein partially functional in BMD, by suppressing certain inflammatory molecules, the companies reported.

The VISION-DMD trial (NCT03439670), completed in August 2021, evaluated vamorolone in 121 boys with DMD, ages 4–6.

Participants were randomly assigned to receive vamorolone, prednisone — a standard corticosteroid — or a placebo for 24 weeks, or about six months. After the first six months, boys using prednisolone or the placebo were switched to vamorolone for another 24 weeks.

Top-line trial results showed that vamorolone demonstrated a good safety profile and led to functional improvements after six months. The trial’s primary goal of a significant change in the Time to Stand (TTSTAND) velocity, a marker of muscle function, was met.

A 6 mg/kg daily oral dose of vamorolone performed similarly to prednisone in terms of efficacy, but was significantly superior to the corticosteroid in terms of side effects.

In the second six-month phase, boys who remained on the treatment maintained or experienced a further improvement in muscle function for up to 48 weeks. Those who switched from prednisone experienced a reduction in side effects, reversal of stunted growth, and stabilization of body fat, which had increased when using prednisone.

In the pilot study in BMD, participants will be randomly assigned, in a 2:1 ratio, to receive vamorolone or a placebo for 24 weeks.

Vamorolone will be given as an oral suspension at a dose of 500 mg daily, or 250 mg if the patient weighs less than 50 kg (110 lbs).

The therapy’s pharmacokinetics, or movement into, through, and out of the body, will be measured as a secondary outcome. Another secondary outcome is its pharmacodynamics, or biochemical effects on the body.

Efficacy will be evaluated in an exploratory manner, and will include the Time to Run/Walk Test and the North Star Ambulatory Assessment; both evaluate motor function. The NeuroQOL scales will be used to assess health-related quality of life.

Funding for the trial comes in part from the FDA under a $1.2 million “Clinical Studies of Orphan Products Addressing Unmet Needs of Rare Diseases (R01)” grant, a funding opportunity for rare disease research.

Additional grant support comes from the National Institutes of Health (NIH) and the Foundation to Eradicate Duchenne.