Amarna Therapeutics secures additional €5 ($5.7) million to advance its transformative gene therapy platform towards clinical trials

- €5 million financing provided by existing investors C4 holding BV and Flerie Invest AB, as well as the Netherlands Enterprise Agency (RVO)

- Will fund progress of hemophilia B program towards first-in-human clinical study

- Dutch biopharmaceutical CDMO HALIX selected to produce clinical trial materials

News provided by

Amarna Therapeutics

Dec 01, 2021, 08:32 ET

LEIDEN, Netherlands, Dec. 1, 2021 /PRNewswire/ -- Amarna Therapeutics, a privately-held biotechnology company developing transformative gene therapies in a range of rare and prevalent diseases, today announced that it has secured a further €4 million from existing investors C4 holding BV and Flerie Invest AB, as well as € 1 million of additional innovation credit funding from RVO, the Netherlands Enterprise Agency. The funds will enable Amarna to advance its lead gene therapy for the treatment of hemophilia B towards a first-in-human clinical trial, and to progress its R&D pipeline in selected autoimmune diseases and chronic inflammation.

This new capital complements a €10 million funding from the same investors and RVO in October 2019, recognizing the progress the company has made in advancing its lead project towards the clinic, and demonstrating the strong potential of Amarna's platform in other highly prevalent disease areas. The latter includes the generation of promising in vitro and in vivo results, showing the potential to induce immune tolerance in autoimmune diseases, as well as to downregulate infection in chronic inflammatory indications.

Broadening pipeline through SVec gene delivery platform

Amarna is uniquely positioned to develop a broad pipeline of safe effective and durable gene therapies by leveraging the intrinsic non-immunogenicity features of its proprietary in vivo SV40-mediated gene delivery platform, called SVec. The company's lead product, AMA005, aims to restore the blood clotting process in hemophilia B patients. The company is currently completing IND enabling studies and has initiated manufacturing with the aim of starting a Phase 1/2a trial in approximately two years. For this, Amarna has recently engaged HALIX, a leading biopharmaceutical contract development and manufacturing organization (CDMO), to produce the clinical trial material. Technology transfer of Amarna's production process to HALIX's GMP manufacturing facility in the Leiden Bio Science Park in the Netherlands, is currently ongoing.

In parallel with AMA005, Amarna is progressing its R&D pipeline of selected autoimmune and chronic inflammation programs towards proof-of-principle in order to maximize the therapeutic potential of its SVec gene delivery platform beyond gene replacement.

Steen Klysner, CEO of Amarna Therapeutics commented:

"As Amarna is entering an exciting next phase, we would like to thank both our existing investors and RVO for their continued support, which enables us to advance our lead gene therapy towards the clinic and progress our broader R&D pipeline for the treatment of autoimmune and chronic inflammation towards pre-clinical proof-of-principle."

"Moreover, we are pleased to have partnered with renowned Dutch CDMO HALIX for the production of our first clinical batches, providing an optimal setting for process transfer and collaboration, due to its location in our close proximity in Leiden."

About Hemophilia B

Hemophilia B is an X-linked blood clotting disorder causing easy bruising and bleeding due to an inherited mutation of the F9 gene, resulting in a deficiency of coagulation factor IX (FIX) in the blood. Present treatment regimens include regular intravenous infusion of FIX and/or blood transfusions. While clinical proof-of-concept has been demonstrated for vector-mediated correction of FIX activity, current approaches are hampered by safety and efficacy issues related to pre-existing and amplified immune responses, thereby providing a significant opportunity for a safe, non-immunogenic gene therapy product with the potential to durably cure all hemophilia B patients.

About Amarna Therapeutics

Amarna Therapeutics is a privately held, pre-clinical biotech company developing transformative, potentially curative gene therapies for a range of rare and prevalent diseases. The company is pioneering the development of gene therapies based on simian virus 40 (SV40), a macaque polyoma virus to which humans are immunologically naïve. Amarna has created a proprietary production cell line (SuperVero) that for the first time makes it possible to produce SV40-derived vectors suitable for therapeutic use. The company's fully integrated gene therapy platform combining SuperVero with its genetically engineered SVec viral vector is truly unique in its intrinsic capability to deliver transgenes without eliciting immune responses to the vector nor to the transgene product. Leveraging this 'natural' non-immunogenicity feature of its gene delivery system in humans, Amarna is developing a broad pipeline of safe effective and durable gene therapies focused on genetic disorders, autoimmune diseases and chronic inflammation. Founded in 2008, Amarna's head office is based in Leiden Bio Science Park, the Netherlands, with a research subsidiary located in Seville, Spain.

More information on www.amarnatherapeutics.com

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For further inquiries please contact:

Amarna Therapeutics
Steen Klysner, CEO
E-mail: [email protected]

LifeSpring Life Sciences Communication, Amsterdam
Léon Melens
Tel: +31 6 538 16 427
E-mail: [email protected]

SOURCE Amarna Therapeutics