Pratteln, Switzerland, June 10, 2024 - Santhera Pharmaceuticals (SIX: SANN)
announces that its partner Sperogenix Therapeutics has launched a paid-for Early
Access Program (EAP) for AGAMREE® (vamorolone) in China for patients with
Duchenne muscular dystrophy (DMD).
In April 2024, the Hainan Medical Products Administration (HMPA) authorized the
EAP for AGAMREE based on local policies, AGAMREE's existing overseas approvals
(U.S., EU, UK) and its demonstrated ability to address urgent clinical needs in
DMD, where approved treatments are currently unavailable in China. The EAP has
started in the Bo'ao Lecheng Pilot Zone, located in Hainan Province, in mid-May,
when the first patients were treated with AGAMREE.
In March 2024, the National Medical Products Administration (NMPA) accepted the
new drug application (NDA) filing for AGAMREE in DMD for patients aged 4 years
and older, incorporating it into both the Priority Review Program and the
Breakthrough Therapy Program. Subject to a positive review outcome, approval
could be obtained by Q1-2025.
Duchenne muscular dystrophy is a rare neuromuscular disease affecting about
70,000 patients in China. Currently, there is no approved drug to treat DMD in
China, leaving a high unmet medical need and therapeutic gap, especially
considering the increasing diagnosis rates that enable more patients to access
specialized treatment centers.
According to the license agreement between the companies, first announced in
January 202 (https://www.santhera.com/assets/files/press-releases/2022-
01-04_SperogenixLicense_e_final_220104_064153.pdf)2, Sperogenix holds exclusive
development and commercialization rights to AGAMREE in DMD and all other rare
disease indications for China. Santhera is supplying treatment medication to
Sperogenix for the EAP as well as for commercialization. Sperogenix will pay
Santhera double-digit percentage royalties on net product sales (including for
the EAP) and additional revenue-dependent milestones on commercial sales.
About AGAMREE® (vamorolone)
AGAMREE is a novel drug with a mode of action based on binding to the same
receptor as glucocorticoids but modifying its downstream activity. Moreover, it
is not a substrate for the 11-?-hydroxysteroid dehydrogenase (11?-HSD) enzymes
that may be responsible for local drug amplification and corticosteroid-
associated toxicity in local tissues (1-4). This mechanism has shown the
potential to 'dissociate' efficacy from steroid safety concerns and therefore
AGAMREE is positioned as a dissociative anti-inflammatory drug and an
alternative to existing corticosteroids, the current standard of care in
children and adolescent patients with DMD (1-4).
In the pivotal VISION-DMD study, AGAMREE met the primary endpoint Time to Stand
(TTSTAND) velocity versus placebo (p=0.002) at 24 weeks of treatment and showed
a good safety and tolerability profile (1, 4). The most commonly reported side
effects were cushingoid features, vomiting, weight increase and irritability.
Side effects were generally of mild to moderate severity.
Currently available data show that AGAMREE, unlike corticosteroids, has no
restriction of growth (5) and no negative effects on bone metabolism as
demonstrated by normal bone formation and bone resorption serum markers (6).
AGAMREE (vamorolone), an orphan medicinal product, is approved for use in the
United States (Prescribing Information (https://agamree.com/pdf/agamree-
pi.pdf)), the European Union (Summary of Product Characteristics
(https://www.ema.europa.eu/en/documents/product-information/agamree-epar-
product-information_en.pdf)) and the United Kingdom.
References:
(1) Dang UJ et al. (2024) Neurology 2024;102:e208112.
doi.org/10.1212/WNL.0000000000208112. Link
(https://www.neurology.org/doi/pdf/10.1212/WNL.0000000000208112).
(2) Guglieri M et al (2022). JAMA Neurol. 2022;79(10):1005-1014.
doi:10.1001/jamaneurol.2022.2480. Link
(https://jamanetwork.com/journals/jamaneurology/fullarticle/2795868?utm_campaign
=articlePDF&utm_medium=articlePDFlink&utm_source=articlePDF&utm_content=jamaneur
ol.2022.2480).
(3) Liu X et al (2020). Proc Natl Acad Sci USA 117:24285-24293
(4) Heier CR et al (2019). Life Science Alliance DOI: 10.26508
(5) Ward et al., WMS 2022, FP.27 - Poster 71. Link
(https://www.santhera.com/assets/files/content/scientific-literature/P71-
SAN1122002_WMS_Height_Poster_30x42_v6.09534-FINAL-cm3.pdf).
(6) Hasham et al., MDA 2022 Poster presentation. Link
(https://www.santhera.com/assets/files/content/scientific-literature/SAN1122001-
PDN-switch-Poster_14Mar22.pdf).
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. Corticosteroids are the
current standard of care for the treatment of DMD.
About Sperogenix
Sperogenix Therapeutics is a platform company dedicated to developing and
commercializing rare disease therapeutics in China. With prioritized therapeutic
areas such as neuromuscular diseases and inherited metabolic diseases,
Sperogenix is dedicated to establishing an innovative commercial model tailored
to the China rare disease field, in order to provide affordable and reliable
products and services to Chinese physicians and patients. Sperogenix was founded
in 2019 and is backed by biopharma industry blue chip investors including Lilly
Asia Ventures, Morningside Ventures and Prosperico Ventures. www.sperogenix.com.
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. The
Company has an exclusive license from ReveraGen for all indications worldwide to
AGAMREE® (vamorolone), a dissociative steroid with novel mode of action, which
was investigated in a pivotal study in patients with Duchenne muscular dystrophy
(DMD) as an alternative to standard corticosteroids. AGAMREE for the treatment
of DMD is approved in the U.S. by the Food and Drug Administration (FDA), in the
EU by the European Medicines Agency (EMA), and in the UK by the Medicines and
Healthcare products Regulatory Agency (MHRA). Santhera has out-licensed rights
to AGAMREE for North America to Catalyst Pharmaceuticals, Inc. and for China to
Sperogenix Therapeutics. For further information, please visit www.santhera.com
(http://www.santhera.com).
AGAMREE® is a trademark of Santhera Pharmaceuticals.
For further information please contact:
public-relations@santhera.com (mailto:public-relations@santhera.com) or
Eva Kalias, Head Investor Relations & Communications
Phone: +41 79 875 27 80
eva.kalias@santhera.com
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.
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