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Press Release: Vamorolone Designated Promising Innovative Medicine (PIM) for Treatment of Duchenne Muscular Dystrophy by the UK MHRA

| Quelle: Dow Jones Newsw... | Lesedauer etwa 5 min. | Text vorlesen Stop Pause Fortsetzen

Pratteln, Switzerland, October 21, 2019 Santhera Pharmaceuticals
(SIX: SANN) announces that the UK's Medicines and Healthcare Products
Regulatory Agency (MHRA) has informed ReveraGen BioPharma about having
designated vamorolone as Promising Innovative Medicine (PIM) for the
treatment of Duchenne muscular dystrophy (DMD).

"We congratulate ReveraGen on this success and are excited about the PIM
designation as it further validates the potential of vamorolone as an
innovative treatment approach addressing the high unmet medical need in
young patients with DMD," said Thomas Meier, PhD, CEO of Santhera.

The PIM designation indicates that the UK MHRA considers vamorolone a
promising candidate for the Early Access to Medicines Scheme (EAMS). In
the UK, the EAMS, of which PIM is the first step, aims to give patients
with life threatening or seriously debilitating conditions access to
medicines that do not yet have a marketing authorization when there is a
clear unmet medical need.

Vamorolone is a first-in-class steroidal anti-inflammatory
investigational drug in development as treatment for DMD. Data from
non-clinical and clinical studies indicated that vamorolone treatment
results in a persistent improvement of muscle function with less adverse
effects typically reported for traditional corticosteroids [1-6].

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.

About Vamorolone first-in-class dissociative steroid

Vamorolone is a first-in-class drug candidate that binds to the same
receptors as corticosteroids but modifies the downstream activity of the
receptors. This has the potential to 'dissociate' efficacy from typical
steroid safety concerns and therefore could replace existing
corticosteroids, the current standard of care in children and adolescent
patients with DMD. There is significant unmet medical need in this
patient group as high dose corticosteroids have severe systemic side
effects that detract from treatment compliance and patient quality of

The currently ongoing 48-week Phase IIb VISION-DMD study (VBP15-004;
NCT03439670) is designed as a pivotal trial to demonstrate efficacy and
safety of vamorolone compared with prednisone and placebo in 120 boys
aged 4 to <7 with DMD that have not yet been treated with
corticosteroids. For more information:

Vamorolone is being developed by US-based ReveraGen BioPharma Inc. with
participation in funding and design of studies by several international
non-profit foundations, the US National Institutes of Health, the US
Department of Defense and the European Commission's Horizon 2020
program. In November 2018, Santhera acquired from Idorsia the option to
an exclusive sub-license to vamorolone in all indications and all
countries worldwide (except Japan and South Korea).

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 is building a Duchenne muscular dystrophy (DMD)
product portfolio to treat patients irrespective of causative mutations,
disease stage or age. A marketing authorization application for
Puldysa(R) (idebenone) is currently under review by the European
Medicines Agency. Santhera has an option to license vamorolone, a
first-in-class dissociative steroid currently investigated in a pivotal
study in patients with DMD to replace standard corticosteroids. The
clinical stage pipeline also includes POL6014 to treat cystic fibrosis
(CF) and other neutrophilic pulmonary diseases, as well as omigapil and
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

Raxone(R) and Puldysa(R) are trademarks 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 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).


[1] Hoffman EP et al. (2019). Vamorolone trial in Duchenne muscular
dystrophy shows dose-related improvement of muscle function. Neurology
93(13):e1312-e1323. doi:10.1212/WNL.0000000000008168

[2] Hoffman EP et al. (2018). Phase 1 trial of vamorolone, a
first-in-class steroid, shows improvements in side effects via
biomarkers bridged to clinical outcomes. Steroids 134: 43-52.

[3] Conklin LS et al. (2018). Phase IIa trial in Duchenne muscular
dystrophy shows vamorolone is a first-in-class dissociative steroidal
anti-inflammatory drug. Pharmacol Res
. 13 6:14 0-150. doi: 10.1016/j.phrs.2018.09.007.

[4] Mavroudis PD et al. (2019). Population pharmacokinetics of
vamorolone (VBP15) in healthy men and boys with Duchenne muscular
dystrophy. J Clin Pharmacol
. 59(7):979-988. doi: 10.1002/jcph.1388.

[5] Heier CR at al. (2013). VBP15, a novel antiinflammatory and
membranestabilizer, improves muscular dystrophy without side effects.
EMBO Mol Med 5: 15691585

[6] Heier CR et al. (2019). Vamorolone targets dual nuclear receptors to
treat inflammation and dystrophic cardiomyopathy. Life Science Alliance
DOI 10.26508/lsa.201800186

For further information please contact:


public-relations@santhera.com or

Eva Kalias, Head External Communications

Phone: +41 79 875 27 80



Eric Hoffman, PhD, CEO

Phone: +1 240-672-0295


Disclaimer / Forward-looking statements

This communication does not constitute an offer or invitation to
subscribe for or purchase any securities of Santhera Pharmaceuticals
Holding AG. This publication may contain certain forward-looking
statements concerning the Company and its business. Such statements
involve certain risks, uncertainties and other factors which could cause
the actual results, financial condition, performance or achievements of
the Company to be materially different from those expressed or implied
by such statements. Readers should therefore not place undue reliance on
these statements, particularly not in connection with any contract or
investment decision. The Company disclaims any obligation to update
these forward-looking statements.

# # #


2019 10 21_PIM_Vamorolone_e_final

(END) Dow Jones Newswires

October 21, 2019 01:00 ET ( 05:00 GMT)

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