Dynacure Announces New Program, DYN201, for Hereditary Spastic Paraplegia (HSP), a Rare Neurodegenerative Disease

Dynacure acquires intellectual property from Paris Brain Institute to support DYN201 development

STRASBOURG, France and PHILADELPHIA, Oct. 13, 2020 /PRNewswire/ -- Dynacure, a clinical-stage drug development company focused on improving the lives of patients with rare and orphan disorders, announced today that they have entered into a license agreement with the Paris Brain Institute to acquire intellectual property rights related to the development of the Company's second pipeline program, DYN201 for the treatment of Hereditary Spastic Paraplegia (HSP).

Hereditary Spastic Paraplegias are a group of neurodegenerative diseases characterized by spasticity and motor dysfunction and affecting approximately 5 out of every 100,000 people. The only available treatments for these diseases are muscle relaxants, which influence the muscle rigidity or spasticity but do not prevent or delay disease progression. Furthermore, some forms of HSP are associated with cognitive deficits that are not improved by current treatments. There is a strong unmet need for treatment of these diseases.

"We are very pleased to collaborate with the Paris Brain Institute and in particular Professor Frederic Darios and Professor Giovanni Stevanin, global experts in Hereditary Spastic Paraplegias," said Frederic Legros (PhD), Chief Operating Officer of Dynacure. "With the aim of building Dynacure's pipeline, this second program demonstrates our ability to identify promising academic research that is establishing pathological drivers of rare diseases that will support further preclinical and clinical research in HSP."

The researchers at the Paris Brain Institute identified mutations in the SPG11 gene cause the most prevalent form of autosomal recessive HSP (HSP type 11) in 2008 (Stevanin, G. et al. 2008). Mutations in the causal gene SPG11 are associated with thin corpus callosum, cognitive decline and lower motor neuron degeneration (Brain, 131, 772-784). Dynacure is advancing DYN201, a preclinical program for the treatment of HSP type 11, which strengthens the Company's focus on the development of therapies for rare neurological indications.

"We are pleased to see one of our early-stage, fundamental discoveries in rare diseases be translated into an exciting drug development program. The collaborative nature of scientific interactions between both the Dynacure and Paris Brain Institute teams have been key to reaching this point," said Alexis Genin, Head of Research & Technology Development at Paris Brain Institute.

About Dynacure

Dynacure is a clinical-stage drug development company focused on improving the lives of patients with rare and orphan diseases. The Dynacure team leverages its proven track record in rare disease drug development to build a pipeline of novel drugs. Dynacure is developing DYN101, an investigational antisense medicine designed to modulate the expression of dynamin 2 for the treatment of Myotubular and Centronuclear Myopathies, in strategic collaboration with Ionis Pharmaceuticals. Dynacure is also building a complementary research portfolio targeting other orphan disorders, including its DYN201 program for the treatment of Hereditary Spastic Paraplegias Type 11 (HSP11).

The Company is headquartered in Strasbourg, France with a corporate office in Philadelphia, PA, USA. Dynacure's investors are Andera Partners, Bpifrance Large Venture, Bpifrance through its FABS and Fonds Biothérapies Innovantes et Maladies Rares funds, Idinvest, Ionis Pharmaceuticals, Kurma Partners, Perceptive Advisors, Pontifax and funds managed by Tekla Capital Management LLC.

About Paris Brain Institute

Created in 2010, the Paris Brain Institute is a scientific and medical research centre of international excellence, located in Paris in the heart of the Pitié-Salpêtrière Hospital. Its innovative model brings together patients, doctors, researchers and entrepreneurs in one place with a common goal: to understand the brain and accelerate the discovery of new treatments for nervous system diseases. The Institute includes a network of more than 700 researchers and clinicians, 10 cutting-edge technological platforms, 1 clinical investigation centre and more than 1,000m² for the incubation of start-ups. Since 2017, it has also been Station F's health partner, giving it a competitive advantage in the field of connected healthcare. This year, the Paris Brain Institute celebrates its tenth anniversary. For more information, please visit www.dynacure.com

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SOURCE Dynacure

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