Santhera Acquires Global Rights to DMD Therapy Vamorolone

Santhera Pharmaceuticals has obtained the worldwide rights to vamorolone (VBP15), an experimental therapy for Duchenne muscular dystrophy (DMD), the company announced.

The company has reached an agreement with ReveraGen Biopharma, the original developer of vamorolone, and Actelion Pharmaceuticals’ spin-off Idorsia, which had acquired an option to in-license vamorolone from ReveraGen. Santhera now holds the rights to vamorolone for all indications, including DMD.

“We are excited about the license transfer of vamorolone to Santhera. Our decision to exercise the option now has been driven by a combination of factors including the availability of encouraging clinical efficacy and safety data with vamorolone,” Dario Eklund, CEO of Santhera, said in a press release.

Vamolorone is an anti-inflammatory steroid treatment for young DMD patients who maintain respiratory function. The medicine seeks to sustain muscle function and prolong the ability to walk without assistance (ambulation), without some of the side effects commonly associated with corticosteroids — the current standard of care in children and adolescents with DMD — such as prednisone and Emflaza (deflazacort).

The therapy has received orphan drug and rare pediatric disease designations, and was granted fast track status by the U.S. Food and Drug Administration (FDA).

The Phase 2b VISION-DMD (VBP15-004) trial (NCT03439670) is comparing Vamorolone’s efficacy and safety to prednisone and a placebo in boys with DMD. The study is currently recruiting approximately 120 participants, ages 4 to 7, in countries around the world; full enrollment is expected soon. More information on the 33 study sites is available here.

The results from the first six months of treatment are expected by April 2021, after a delay due to the COVID-19 pandemic. If positive, the data could be used to support a submission to the FDA still in 2021 seeking regulatory approval of vamorolone.

Data from preclinical and clinical studies have shown that vamolorone improves the muscle function of DMD patients in a dose-dependent manner, without the detrimental side effects of corticosteroids.

“Our work to date clearly shows that vamorolone not only holds the potential to become a new standard of care for patients with DMD but also could benefit patients in a number of other inflammatory diseases,” said Eric Hoffman, PhD, vice president of research at ReveraGen.

Santhera also is developing Puldysa (idebenone), an oral treatment for respiratory symptoms associated with DMD in patients not taking glucocorticoids.

Treatment with Puldysa reduced the loss of respiratory function in DMD patients who discontinued glucocorticoids, delaying the need for assisted ventilation by three years, as compared with a placebo, data from the Phase 3 DELOS trial (NCT01027884) showed.

Based on the data from DELOS and additional Phase 2 and Phase 3 trials, Santhera submitted a request to the European Medicines Agency (EMA) seeking approval of Puldysa in the European Union.

That request is currently under review by EMA’s Committee for Medicinal Products for Human Use. A decision on whether to recommend the treatment is expected by the end of this year.

Santhera expects vamorolone and Puldysa to complement each other in addressing the medical needs of people with DMD from early to late disease stages, irrespective of the patients’ age.

“We believe that having two promising, complementary, late stage assets for DMD in our pipeline will enable increased access to potentially transformative treatments for a wider patient population,” Eklund said. “We are grateful to Idorsia, our anchor shareholder, for enabling early access to the license, highlighting its confidence in Santhera as the best suited company to bring vamorolone to patients.”

Under the terms of the agreement, Santhera will pay monthly installments to fund the development of vamorolone, and will support a new drug application to the FDA based on the six-month results of VISION-DMD.

According to the rare pediatric disease designation from the FDA, ReveraGen may receive a priority review voucher upon approval of vamorolone for DMD. In that case, Santhera would receive part of any potential proceeds from the priority review voucher.

“We are delighted about the revised contractual arrangement and being able to work directly with Santhera as the licensee for vamorolone,” Hoffman said. “Santhera’s experience in both development of DMD drug candidates and the commercialization of a rare disease product positions it well to bring vamorolone to patients.”