Pratteln,
This publication in the journal PLOS Medicine [1] provides peer-reviewed and detailed open-label data in patients with DMD treated for 18 months with vamorolone. A multi-center, open-label, 24-week trial (VBP15-003; [2, 3]) with a total 24-month long-term extension (VBP15-LTE; [4]) was conducted by the
“This long-term study showed significant continued clinical improvement of all outcomes measured over an 18-month follow-up period,” said
“Importantly, we also found that vamorolone did not show stunting of growth seen with deflazacort and prednisone, and vamorolone also showed fewer physician-reported adverse events such as mood disturbance, excessive hair growth, and Cushingoid appearance,” noted
DMD trial participants (4 to <7 years at entry) treated with 2.0 or 6.0 mg/kg/day vamorolone for the full 18-month period (n=23) showed clinical improvement of all motor outcomes from baseline to month 18 (time to stand velocity, p = 0.012 [95% CI 0.010, 0.068 event/second]; run/walk 10 meters velocity, p < 0.001 [95% CI 0.220, 0.491 meters/second]; climb 4 stairs velocity, p = 0.001 [95% CI 0.034, 0.105 event/second]; 6-minute walk test, p = 0.001 [95% CI 31.14, 93.38 meters]; North Star Ambulatory Assessment, p < 0.001 [95% CI 2.702, 6.662 points]). Outcomes in vamorolone-treated DMD patients (n = 46) were compared to group-matched participants in the CINRG Duchenne Natural History Study (corticosteroid-naïve, n = 19; corticosteroid-treated, n = 68) over a similar 18-month period. Time to stand was not significantly different between vamorolone-treated and corticosteroid-naïve participants (p = 0.088; least squares [LS] mean 0.042 [95% CI –0.007, 0.091]), but vamorolone-treated participants showed significant improvement compared to group-matched corticosteroid-naïve participants for run/walk 10 meters velocity (p = 0.003; LS mean 0.286 [95% CI 0.104, 0.469]) and climb 4 stairs velocity (p = 0.027; LS mean 0.059 [95% CI 0.007, 0.111]). The vamorolone-related improvements were similar in magnitude to corticosteroid-related improvements. Corticosteroid-treated participants showed stunting of growth, whereas vamorolone-treated trial participants did not (p < 0.001; LS mean 15.86 [95% CI 8.51, 23.22]). Physician-reported incidences of adverse events (AEs) for Cushingoid appearance, hirsutism, weight gain, and behavior change were less for vamorolone than published incidences for prednisone and deflazacort.
About Vamorolone
Vamorolone is a first-in-class drug candidate that binds to the same receptor as corticosteroids but modifies its downstream activity and as such is a dissociative partial agonist [5-8]. This mechanism has the potential to ‘dissociate’ efficacy from typical steroid safety concerns and therefore vamorolone could emerge as a promising alternative to existing corticosteroids, the current standard of care in children and adolescent patients with DMD. There is substantial unmet medical need in this patient group as high-dose corticosteroids have significant systemic side effects that diminish patient quality of life. The fully-enrolled, pivotal Phase 2b VISION-DMD trial (VBP15-004, [10, 11], https://vision-dmd.info/2b-trial-information) is currently being conducted at study sites across
Vamorolone was discovered by US-based
References:
[1] Smith E, et al. (2020). Efficacy and safety of vamorolone in Duchenne muscular dystrophy: an 18-month interim analysis of a non-randomized open-label extension study. PLOS Medicine, Link
[2] Clinicaltrials.gov: An Extension Study to Assess Vamorolone in Boys with Duchenne Muscular Dystrophy (DMD), Link
[3] Hoffman EP et al. (2019). Vamorolone trial in Duchenne muscular dystrophy shows dose-related improvement of muscle function. Neurology 93: e1312-e1323.
[4] Clinicaltrials.gov: Long-term Extension Study to Assess Vamorolone in Boys with Duchenne Muscular Dystrophy (DMD), Link
[5] Heier CR at al. (2013). VBP15, a novel anti‐inflammatory and membrane‐stabilizer, improves muscular dystrophy without side effects. EMBO Mol Med 5: 1569–1585.
[6] Reeves EKM, et al (2013) VBP15: preclinical characterization of a novel anti-inflammatory delta 9,11 steroid. Bioorg Med Chem 21(8):2241-2249
[7] Heier CR et al. (2019). Vamorolone targets dual nuclear receptors to treat inflammation and dystrophic cardiomyopathy. Life Science Alliance DOI 10.26508/lsa.201800186.
[8] Liu X et al. (2020). Disruption of a key ligand-H-bond network drives dissociative properties in vamorolone for Duchenne muscular dystrophy treatment.
[9] Press release “Santhera Exercises Option to Obtain Worldwide Rights to Vamorolone in Duchenne Muscular Dystrophy and All Other Indications”,
[10] Clinicaltrials.gov: A Study to Assess the Efficacy and Safety of Vamorolone in Boys with Duchenne Muscular Dystrophy (DMD), Link
[11] Press release “Santhera Announces Full Enrollment of ReveraGen’s Pivotal VISION-DMD Study with Vamorolone in Duchenne Muscular Dystrophy”,
About Santhera
Puldysa® and Raxone® are trademarks of
About ReveraGen BioPharma
ReveraGen was founded in 2008 to develop first-in-class dissociative steroidal drugs for Duchenne muscular dystrophy and other chronic inflammatory disorders. The development of ReveraGen’s lead compound, vamorolone, has also been supported through partnerships with foundations worldwide, including
For further information please contact:
Santhera
public-relations@santhera.com or
Phone: +41 79 875 27 80
eva.kalias@santhera.com
ReveraGen BioPharma
Phone: + 1 240-672-0295
eric.hoffman@reveragen.com
Disclaimer / Forward-looking statements
This communication does not constitute an offer or invitation to subscribe for or purchase any securities of
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Attachment
- 2020-09-22_LTE-vamorolone_e_final
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