Press Release: Santhera Announces Completion of First 6-Month Period of Pivotal VISION-DMD Trial with Vamorolone in Duchenne Muscular Dystrophy

Pratteln, Switzerland, March 3, 2021 -- Santhera Pharmaceuticals (SIX:

SANN) announces that the last patient has completed the last visit for

the first period of the placebo-controlled pivotal VISION-DMD study with

vamorolone in patients with Duchenne muscular dystrophy (DMD), conducted

by partner ReveraGen Biopharma Inc. Subject to a positive 6-month

topline data readout of this first study phase, this could allow for a

regulatory submission to the US FDA in Q1-2022 with the potential to

offer an alternative to current standard of care in DMD.

The 48-week Phase 2b VISION-DMD study is designed as a pivotal trial to

demonstrate efficacy and safety of vamorolone administered orally at

doses of 2.0 mg/kg/day and 6.0 mg/kg/day versus prednisone 0.75

mg/kg/day and placebo in ambulant boys aged 4 to <7 years with DMD [1].

Efficacy outcome measures are motor function and strength outcomes with

Time to Stand test (TTSTAND) as the primary study endpoint. Additional

analyses compare safety and tolerability between the vamorolone dose

groups, placebo and prednisone. In the now completed 24-week, placebo-

and active-controlled treatment period, patients were randomized to

receive vamorolone 2.0 mg/kg/day, vamorolone 6.0 mg/kg/day, prednisone

0.75 mg/kg/day or matching placebo. For the second treatment period of

another 24 weeks of continued study conduct, patients who previously

received prednisone or placebo have been randomized and will be switched

to one of two doses of vamorolone (2.0 or 6.0 mg/kg/day). This treatment

period where all patients receive vamorolone is evaluating the

persistence of effect in the longer term. In addition to efficacy, the

study aims to confirm the differentiated safety and 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 as a chronic therapy.

"We are delighted about having achieved this important milestone and are

looking forward to announcing the topline 6-month results of this

pivotal study together with Santhera," said Eric Hoffman, PhD, President

and CEO at ReveraGen BioPharma. "The use of glucocorticoids, despite

having proven benefits in the treatment of DMD, is severely limited due

to side effects and poor tolerability. Our expectation is that

vamorolone will have the benefits but avoids many of the tolerability

issues that limit the use of this standard of care. Our thanks go out to

the study participants, their families and healthcare professionals who,

in the midst of the COVID-19 pandemic, are enabling us to advance this

pivotal study as intended."

"Based on previously established data, we believe that vamorolone has

the potential to become a foundational therapy in DMD for patients

irrespective of the underlying gene mutation and a promising alternative

to existing corticosteroids," noted Dario Eklund, CEO of Santhera. "Our

organization is whole heartedly dedicated to bringing this novel therapy

to patients who are hoping for a DMD therapy with fewer treatment

limiting side effects, making it suitable for longer term administration

and also improving quality of life."

In the currently completed studies, a total of 48 patients have received

various doses of vamorolone; of which 41 patients have been treated and

evaluated for a period of 2.5 years. Aggregate clinical data from these

open label studies in DMD published to date showed sustained efficacy

and clinical improvement with vamorolone across multiple endpoints [2].

Additionally, vamorolone did not show stunting of growth seen with

deflazacort and prednisone, and also showed fewer physician-reported

adverse events such as mood disturbance, excessive hair growth, and

Cushingoid appearance [2].

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 [3-6]. 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. In the

pivotal Phase 2b VISION-DMD trial [1], the last patient has completed

the last visit of the 24-week, placebo- and active-controlled treatment

period and topline 6-month data are expected in Q2-2021, paving the way

for a US NDA submission in Q1-2022. Vamorolone has been granted Orphan

Drug status in the US and in Europe, and has received Fast Track and

Rare Pediatric Disease designations by the US FDA and Promising

Innovative Medicine (PIM) status from the UK MHRA.

Vamorolone was discovered by US-based ReveraGen BioPharma, Inc. and is

being developed in collaboration with Santhera, which owns worldwide

rights to the drug candidate in all indications. The vamorolone

development program has received funding from several international

non-profit foundations and patient organizations, the US National

Institutes of Health, the US Department of Defense and the European

Commission's Horizon 2020 program.

References:

[1] VISION-DMD (VBP15-004) study information at

https://www.globenewswire.com/Tracker?data=fXvL9V_hAkyVU5ckUfkCBgYe2MmRiK8FI8e7sZTjJshE-y_kHX4QigKPWYMUxdu-uMBH_L6NE6MFE_o76OtxTFC45gjxtppBUpyAchxFH_NF3Halu1uWgTE_ei6nktDgDvSnxdDqZzu_UFHfRtlM9VDWdnifJzI_k-enHwSDRho=

ClinicalTrials.gov Identifier: NCT03439670 and study website

https://www.globenewswire.com/Tracker?data=lDvmuEFIMvgL0TNfh0Jxuxk5CjEiEeswtH1BHXZsYleifjGU0TCJFt3MCK7ts6TN4XrOPQ7BmQfQLunh-NQ-Zjg8_uHH4jWeQx-uGGRgxRsveo3P_b-fnCn51KhKbTI0RzIZwJ9-5U0k8SGEGKBZAA==

https://vision-dmd.info/2b-trial-information

[2] Smith E, et al. (2020). PLOS Medicine,

https://www.globenewswire.com/Tracker?data=ttRL4WFXv6QUKsLCOzJ4oGmKrfqegRXrOh8wIkFPXptyI2L4Z8VYfAuDDNtqyQJSq9jrgE_0-4cYvPh984E_raY0kTLY9oUEMxE7Gbb8fEA=

Link

[3] 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.

[4] Reeves EKM, et al (2013) VBP15: preclinical characterization of a

novel anti-inflammatory delta 9,11 steroid. Bioorg Med Chem

21(8):2241-2249

[5] Heier CR et al. (2019). Vamorolone targets dual nuclear receptors

to treat inflammation and dystrophic cardiomyopathy. Life Science

Alliance DOI 10.26508/lsa.201800186.

[6] Liu X et al. (2020). Disruption of a key ligand-H-bond network

drives dissociative properties in vamorolone for Duchenne muscular

dystrophy treatment. Proc Natl Acad Sci USA.

https://www.globenewswire.com/Tracker?data=ttRL4WFXv6QUKsLCOzJ4oD8TW6zIRRDSThe_fvPB_4hmIcbytap9JhRcv4RWF-1tsO_na3Ecp_e8uLXVgXfPsgt3jdarZMxKMUbxJ23G3o4=

Link

About Santhera

Santhera Pharmaceuticals (SIX: SANN) is a Swiss specialty pharmaceutical

company focused on the development and commercialization of innovative

medicines for rare neuromuscular and pulmonary diseases with high unmet

medical need. Santhera has an exclusive license for all indications

worldwide to vamorolone, a first-in-class dissociative steroid with

novel mode of action, currently investigated in a pivotal study in

patients with DMD as an alternative to standard corticosteroids. The

clinical stage pipeline also includes lonodelestat (POL6014) to treat

cystic fibros.is (CF) and other neutrophilic pulmonary diseases as well

as an exploratory gene therapy approach targeting congenital muscular

dystrophies. Santhera out-licensed ex-North American rights to its first

approved product, Raxone(R) (idebenone), for the treatment of Leber's

hereditary optic neuropathy (LHON) to Chiesi Group. For further

information, please visit

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www.santhera.com.

Raxone(R) is a trademark of Santhera Pharmaceuticals.

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 Muscular Dystrophy Association USA,

Parent Project Muscular Dystrophy, Foundation to Eradicate Duchenne,

Save Our Sons, JoiningJack, Action Duchenne, CureDuchenne, Ryan's Quest,

Alex's Wish, DuchenneUK, Pietro's Fight, Michael's Cause, and Duchenne

Research Fund. ReveraGen has also received generous support from the US

Department of Defense CDMRP, National Institutes of Health (NCATS, NINDS,

NIAMS), and European Commission (Horizons 2020).

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www.reveragen.com

For further information please contact:

Santhera

Santhera Pharmaceuticals Holding AG, Hohenrainstrasse 24, CH-4133

Pratteln

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public-relations@santhera.com or

Eva Kalias, Head External Communications

Phone: +41 79 875 27 80

eva.kalias@santhera.com

ReveraGen BioPharma

Eric Hoffman, PhD, President and CEO

Phone: + 1 240-672-0295

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eric.hoffman@reveragen.com

Disclaimer / Forward-looking statements

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