ORLANDO, Florida—The present and future of Santhera Pharmaceuticals’ lead drug candidate, vamorolone, were described at the CureDuchenne 2022 FUTURES National Conference by Mindy Cameron, the company’s patient advocacy lead.

Mindy started her presentation with her own personal experience with Duchenne muscular dystrophy (DMD) as the mother of a 21-year-old son living with the diagnosis.

Corticosteroids are the standard of care in DMD. This is because they can have a positive impact on disease progression and extend patients’ lifespan. However, they come with many side effects including developmental delays, bone fractures, behavioral changes, weight gain, and cataracts.


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Sharing her son’s own experience of long-term steroid use, Mindy said that he experienced every side effect mentioned above. This is the reason why better therapies are needed to manage the disease, Mindy explained. “I am really really excited about the potential of any new therapies,” she said.

Vamolorone has already been through phase 2a and phase 2b clinical trials. In total, the vamorolone program includes more than 200 patient-years of treatment exposure in 160 patients and many studies have been published about clinical evidence supporting the potential benefits of the treatment.

Mindy said that Santhera is now in the midst of a new drug application to the US Food and Drug Application and a marketing authorization application in Europe for the treatment of DMD.

Vamolorone is an experimental treatment engineered to uncouple antiinflammatory effects from adverse effects mediated by corticosteroids. Like corticosteroids, it inhibits the NF-κB pathway but unlike corticosteroids, it is not a substrate of 11-B hydroxysteroid dehydrogenase. The double bond in vamorolone’s chemical structure attenuates glucocorticoid receptor binding and may lead to less activation of genes associated with the side effects of corticosteroids.

Reference

Beyond dystrophin. Oral presentation at: CureDuchenne 2022 FUTURES National Conference; May 27-29, 2022; Orlando, FL.