Santhera will hold a conference call / webcast today at 16:00 CEST, 15:00 BST, 10:00 EDT. Details are at the end of this statement.
- Vamorolone demonstrates strong efficacy across primary and secondary endpoints in both doses of 6 mg/kg/day and 2 mg/kg/day
- Primary endpoint Time to Stand (TTSTAND) velocity is met for vamorolone 6 mg/kg/day versus placebo (p=0.002)
- Secondary endpoints for Six-Minute Walk (6MWT) and Time to Run/Walk 10 meters (TTRW) tests achieve statistical significance versus placebo
- Study confirmed good safety and tolerability profile of vamorolone for both doses
- Santhera plans to file a New Drug Application (NDA) with the
US FDA in Q1-2022, requesting priority review
Pratteln,
VISION-DMD is a pivotal Phase 2b study designed to demonstrate efficacy and safety of vamorolone compared to placebo and prednisone (active control) in the treatment of DMD. In the first 24-week double-blind period, of which the topline data readout is presented here, 121 ambulant boys aged 4 to <7 years with DMD were randomized to receive vamorolone (low dose 2 mg/kg/day or high dose 6 mg/kg/day) or prednisone (0.75 mg/kg/day) or placebo. A second period of 24 weeks, where all participants receive vamorolone treatment on either of the two dose levels, will continue to capture additional longer term safety and tolerability data.
Vamorolone demonstrates efficacy across primary and secondary endpoints and over a broad dose range
The study met its primary endpoint of superiority in change of time to stand from supine positioning to standing (TTSTAND) velocity with vamorolone 6 mg/kg/day versus placebo (p=0.002) with a treatment difference of 0.06 [95% CI: 0.02–0.10] rises/second from baseline. This corresponds to a clinically relevant improvement in TTSTAND in the vamorolone 6 mg/kg/day group from 6.0 to 4.6 seconds and a corresponding deterioration in the placebo group from 5.4 to 5.5 seconds. The study also demonstrated superiority of vamorolone versus placebo across multiple secondary endpoints which include (in the order of pre-defined hierarchy): TTSTAND velocity for 2 mg/kg/day (p=0.02), 6MWT for 6 mg/kg/day (p=0.003) and 2 mg/kg/day (p=0.009), TTRW for 6 mg/kg/day (p=0.002). The clinical results establish the efficacy of vamorolone over a wide, threefold dose range from 2 to 6 mg/kg/day. No statistically significant differences were observed between vamorolone 6 mg/kg/day and prednisone across the above endpoints.
Vamorolone showed a favorable safety and tolerability profile over prednisone
The study completion rate at 24 weeks was 94% (or 114 of 121 participants). Vamorolone at both doses of 2 and 6 mg/kg/day showed a favorable safety and tolerability profile. In the vamorolone groups, no grade 3 or higher treatment emergent adverse events (TEAEs) or adverse events leading to study discontinuation were observed. The total number of TEAEs was lower in the vamorolone 2 mg/kg/day (events n=96) and 6 mg/kg/day (n=91) groups compared to prednisone (n=120). In a prespecified analysis of clinically relevant adverse events (moderate, severe, serious or leading to discontinuation due to safety), defined for the EMA and conducted by Santhera, vamorolone 6 mg/kg/day was significantly superior to prednisone (n=6 vs n=19, p=0.02).
As previously published, in open-label studies of 2.5 years duration (113 patient years), vamorolone did not show stunting of growth as reported with conventional corticosteroids. Particularly noteworthy, this was validated in the current 24-week double-blind study where vamorolone 6 mg/kg/day versus prednisone 0.75 mg/kg/day showed a significant difference in growth velocity (p=0.02). “Stunting of growth is a major concern of families and patients treated with corticosteroids, and we are delighted to see this superiority of vamorolone proven in this double-blind study,” said Paula Clemens, MD, study Co-Chair, and Vice Chair of VA Affairs and Professor of Neurology,
“Today’s news is a tremendous milestone for patients and Santhera as we further advance vamorolone as a foundational treatment option in DMD. The treatment effect translates into the potential to delay disease progression by about two years and indicates disease modifying potential of vamorolone,” said
“We are thrilled about the positive results of the VISION-DMD study as it represents a culmination over a decade of scientific research,” said
“The strength of evidence for both efficacy and safety of vamorolone over such a wide dose range from 2 to 6 mg/kg/day allows clinicians to individually tailor treatment of Duchenne patients by starting at the higher 6 mg/kg/day dose of vamorolone with equivalent efficacy to daily prednisone and titrate the dose according to how well the treatment is tolerated whilst maintaining optimal efficacy. I am enthusiastic that this approach may allow patients to avoid side effects that currently lead to discontinuing steroid treatment, meaning they are able stay on for longer,” said
Santhera plans to submit a New Drug Application (NDA) in the US in Q1-2022, requesting a priority review based on the fast track designation granted by the FDA. The VISION-DMD study continues to 48 weeks and will, subject to a positive outcome, deliver data for the submission of a marketing authorization application in
Vamorolone has been granted Orphan Drug status in the US and in
Santhera Webcast
Santhera will hold a conference call with webcast today at
Webcast: click here http://public.viavid.com/index.php?id=145140
Conference call: dial one of the following numbers (Conference ID 13720219):
Toll Free: +1-877-407-9716
Toll/International: +1-201-493-6779
Replay: a webcast replay of the conference will be available shortly after the call at the above link.
Slides: the slide presentation will be available after the webcast at https://www.santhera.com/investors-and-media/news-and-media-center/company-presentations.
About VISION-DMD
VISION-DMD is a 48-week Phase 2b study designed as a pivotal trial to demonstrate efficacy and safety of vamorolone (2 and 6 mg/kg/day) versus prednisone (0.75 mg/kg/day) and placebo in 121 ambulant boys aged 4 to <7 years with Duchenne muscular dystrophy (DMD). The topline results cover data from the first period of 24 weeks of the study where vamorolone is compared to placebo (FDA-prerequisite for filing an NDA) and prednisone. The primary endpoint of the study is TTSTAND velocity at 24 weeks comparing the 6 mg/kg/day dose of vamorolone to placebo. TTSTAND velocity measures the speed at which patients are able to stand up from lying in a supine position and is a strong and recognized marker for muscle function. Secondary efficacy outcome measures include TTSTAND velocity for vamorolone at the lower dose of 2 mg/kg/day, Six-Minute Walk (6MWT) and Time to Run/Walk 10 meters (TTRW) tests at 24 weeks. The VISION-DMD study continues to completion at 48 weeks. During the second period of this 48-week study, all participants receive vamorolone. Participants from the placebo and prednisone arms are randomized to either the 2 or 6 mg/kg/day dose of vamorolone and the current vamorolone arms continue on their existing dose. The final data readout is expected in Q4-2021. In addition to efficacy, the study aims to confirm the favorable tolerability profile of vamorolone with the potential to offer an alternative to current standard of care. Although glucocorticoids are part of the current care recommendations for DMD, their adverse effect profile limits their use.
About Vamorolone
Vamorolone is a first-in-class dissociative steroid which retains the anti-inflammatory activity of corticosteroids while decreasing the deleterious side effects. As such, 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.
Vamorolone was discovered by US-based
About Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is a rare inherited X-chromosome-linked disease, which almost exclusively affects males. DMD is characterized by inflammation which is present at birth or shortly thereafter. Inflammation leads to fibrosis of muscle and is clinically manifested by progressive muscle degeneration and weakness. Major milestones in the disease are the loss of ambulation, the loss of self-feeding, the start of assisted ventilation, and the development of cardiomyopathy. DMD reduces life expectancy to before the fourth decade due to respiratory and/or cardiac failure.
About Santhera
Raxone® is a trademark 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 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
# # #
Attachment
- 2021 06 01_PR_VamTopline_e_final
Source:
2021 GlobeNewswire, Inc., source