Santhera announces the launch of an early access program in China for AGAMREE® by its partner Sperogenix

Pratteln, Switzerland, June 10, 2024 – Santhera Pharmaceuticals (SIX: SANN) announces that its partner Sperogenix Therapeutics has launched a paid 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 PAE for AGAMREE based on local policies, AGAMREE’s existing overseas approvals (US, EU, UK), and capacity demonstrated to address urgent clinical needs in DMD, where approved treatments are currently unavailable in China. The PAE started in the Bo’ao Lecheng pilot area, 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 filing of a New Drug Application (NDA) for AGAMREE in DMD for patients aged 4 years and older, making it part of both the Priority Review and Breakthrough Therapy Program. Subject to a positive outcome of the review, approval could be obtained by the first quarter of 2025.

Duchenne muscular dystrophy is a rare neuromuscular disease that affects approximately 70,000 patients in China. Currently, there are no drugs approved to treat DMD in China, leaving an unmet medical need and a significant therapeutic gap, especially considering the increasing diagnosis rates that allow more patients to access to specialized treatment centers.

According to the licensing agreement between the companies, first announced in January 2022, Sperogenix holds exclusive rights to develop and commercialize AGAMREE in DMD and all other rare disease indications for China. Santhera supplies therapeutic drugs to Sperogenix for PAE as well as commercialization. Sperogenix will pay Santhera double-digit royalties on net product sales (including for EAP) and additional revenue-dependent milestones on commercial sales.

About AGAMREE® (vamorolone)
AGAMREE is a new drug whose mode of action relies on binding to the same receptor as glucocorticoids but modifies its downstream activity. Additionally, it is not a substrate for 11-β-hydroxysteroid dehydrogenase (11β-HSD) enzymes which may be responsible for local drug amplification and corticosteroid-associated toxicity in local tissues (1-4 ). This mechanism has shown the potential to “decouple” effectiveness from steroid safety concerns and AGAMREE is therefore positioned as a dissociative anti-inflammatory medication and an alternative to existing corticosteroids, the current standard of care in children and adolescents with of DMD (1-4).

In the pivotal VISION-DMD study, AGAMREE met the primary endpoint, withdrawal time (TTSTAND) compared to placebo (p = 0.002) at 24 weeks of treatment and showed a good safety profile and tolerability (1, 4). The most commonly reported side effects were cushingoid features, vomiting, weight gain, and irritability. Side effects were generally mild to moderate in intensity.

Currently available data show that AGAMREE, unlike corticosteroids, has no growth restrictions (5) and no negative effects on bone metabolism as demonstrated by normal serum markers of bone formation and bone resorption (6).

AGAMREE (vamorolone), an orphan drug, is approved for use in the United States (Prescribing Information), the European Union (Summary of Product Characteristics), and the United Kingdom.

The references:
(1) Dang UJ et al. (2024) Neurology 2024;102:e208112. doi.org/10.1212/WNL.0000000000208112. Link.
(2) Guglieri M et al (2022). JAMA Neurol. 2022;79(10):1005-1014. doi:10.1001/jamaneurol.2022.2480. Link.
(3) Liu X et al (2020). Proc Natl Acad Sci USA 117:24285-24293
(4) Heier CR et al (2019). Life Sciences Alliance DOI: 10.26508
(5) Ward et al., WMS 2022, FP.27 – Poster 71. Link.
(6) Hasham et al., MDA 2022 poster presentation. Link.

About Duchenne muscular dystrophy
Duchenne muscular dystrophy (DMD) is a rare hereditary disease linked to the X chromosome, which almost exclusively affects men. DMD is characterized by inflammation present at or shortly after birth. Inflammation leads to muscle fibrosis and manifests clinically as progressive muscle degeneration and weakness. The major stages of the disease are loss of walking, loss of self-feeding, initiation of assisted ventilation and development of cardiomyopathy. DMD reduces life expectancy before the fourth decade due to respiratory and/or cardiac failure. Corticosteroids are currently the standard of care for the treatment of DMD.

About Sperogenix
Sperogenix Therapeutics is a platform company dedicated to the development and commercialization of treatments for rare diseases in China. With priority therapeutic areas such as neuromuscular diseases and hereditary metabolic diseases, Sperogenix is dedicated to establishing an innovative business model suitable for the field of rare diseases in China, to provide affordable and reliable products and services to doctors and Chinese patients. Sperogenix was founded in 2019 and is backed by blue-chip investors in the biopharmaceutical industry, including Lilly Asia Ventures, Morningside Ventures and Prosperico Ventures. www.sperogenix.com.

About Santhera
Santhera Pharmaceuticals (SIX: SANN) is a Swiss pharmaceutical company specializing in the development and commercialization of innovative medicines for the treatment of rare neuromuscular and pulmonary diseases with significant unmet medical needs. The Company holds an exclusive license from ReveraGen for all indications worldwide for AGAMREE® (vamorolone), a dissociative steroid with a novel mode of action, which was investigated in a pivotal study conducted with for patients with Duchenne muscular dystrophy (DMD) as an alternative to standard corticosteroids. . AGAMREE for the treatment of DMD is approved in the US by the Food and Drug Administration (FDA), in the EU by the European Medicines Agency (EMA), and in the UK by the Medicines Regulatory Agency and health products (MHRA). Santhera licensed the rights to AGAMREE for North America to Catalyst Pharmaceuticals, Inc. and for China to Sperogenix Therapeutics. For more information, please visit www.santhera.com.

AGAMREE® is a trademark of Santhera Pharmaceuticals.

For more information please contact:
[email protected] or
Eva Kalias, Head of Investor Relations and Communications
Telephone: +41 79 875 27 80
[email protected]

Disclaimer / Forward-Looking Statements
This communication does not constitute an offer or invitation to subscribe for or purchase securities of Santhera Pharmaceuticals Holding AG. This publication may contain certain forward-looking statements regarding 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 in connection with any contract or investment decision. The Company undertakes no obligation to update these forward-looking statements.

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