Lausanne, SWITZERLAND and San Francisco, CA, USA, 4 May 2022 – Asceneuron SA, a clinical stage company dedicated to targeting the root causes of neurodegenerative diseases, today announces that it has been awarded a second grant from The Michael J. Fox Foundation for Parkinson’s Research (MJFF) to accelerate the study of its clinical stage O-GlcNAcase (OGA) inhibitor ASN51 as a potential new treatment for Parkinson’s disease (PD).

Parkinson’s disease is caused by a loss of dopamine producing nerve cells in the substantia nigra. This leads to a reduction in the signalling mediated by the neurotransmitter dopamine in the brain causing the characteristic motoric dysfunction in PD. An estimated seven to 10 million people worldwide suffer from PD. Although there is no current cure for PD, Asceneuron is discovering and developing therapeutics for the high unmet medical needs of patients experiencing neurodegenerative disorders.
 

The grant will fund a preclinical proof-of-concept study to assess the disease-modifying properties of Asceneuron’s OGA inhibitor ASN51 in a preclinical model of inherited PD. The genetic model is characterized by the overexpression of a-synuclein harbouring the A53T mutation known to cause early-onset, familial PD in humans. Aggregated forms of a-synuclein are the main component of the characteristic Lewy body pathology and thus thought to be causative of the loss of dopaminergic neurons in PD.
 

The aim of this study is to extend previously published findings demonstrating a reduction of motor impairment with Asceneuron’s OGA inhibitors (e.g., Permanne et al., ACS Chem. Neurosci. 2022) to this alternative genetic disease model. The results will further interrogate the mechanism of action of OGA inhibitors with respect to a-synuclein toxicity and aggregation and provide support for the clinical exploration of ASN51 in PD patients. The project will begin immediately, with results expected in the fourth quarter of 2022.
 

Dirk Beher, Chief Executive Officer, Co-Founder of Asceneuron, commented: “We are pleased to have been awarded further funding support from The Michael J. Fox Foundation to progress research on our next generation O-GlcNAcase inhibitor ASN51. ASN51 has the potential to make a meaningful impact on patients suffering with Parkinson’s disease to improve quality of life and slow progression. We are proud to receive the continued support from a foundation that contributes meaningfully towards development of improved therapies for those living with Parkinson’s disease.”
 

“The Michael J. Fox Foundation is dedicated to unlocking the complex biology of Parkinson’s disease and unearth novel breakthrough treatments to improve patient lives and slow disease progression,” said Luis Oliveira, PhD, Senior Associate Director of Research Programs, MJFF. “We are pleased to award Asceneuron a grant to support further research in the O-GlcNAcase inhibitor, ASN51, as a potential modifier of a-synuclein toxicity.”

• ASN90, an O-GlcNAcase inhibitor, is a new chemical entity at clinical stage and a promising candidate for the treatment of PSP, a disorder with a high unmet medical need

• PSP is a rare neurological condition that causes severe problems with walking, balance, speech, swallowing and vision. As the disease progresses, it causes severe disability within three to five years of onset1

• ASN90 in PSP is ready to start phase II of clinical development

Barcelona (Spain) / Lausanne (Switzerland), February 22nd, 2023 – Spanish pharmaceutical company Ferrer and Swiss clinical stage biotechnology company Asceneuron announce the signing of a licensing agreement in which Ferrer obtains the exclusive worldwide rights to develop and commercialize ASN90 in progressive supranuclear palsy (PSP), an orphan, tau-related disease with a high unmet medical need¹.

Both Ferrer and Asceneuron are pleased that this announcement occurs in close occurrence with Rare Disease Day (February 28). This agreement underscores the commitments both companies have undertaken to improve the lives of patients with a rare disease, such as PSP.

The terms of the agreement include an upfront fee and multiple development, regulatory and commercial milestone payments. Asceneuron is also eligible to receive tiered double-digit royalties on worldwide net sales of ASN90. 

Mario Rovirosa, Ferrer’s Chief Executive Officer, stated: “This is yet another meaningful example of how Ferrer moves forward guided by its purpose of making a positive impact in society. As a B Corp company, we are proud of our people’s commitment to delivering the best possible solutions for those in need.”

Dirk Beher, Chief Executive Officer and Co-Founder of Asceneuron, commented: “This licensing agreement with Ferrer is important news for patients suffering from PSP and their families. Asceneuron, as a neurodegeneration-focused company, will continue expanding the application of OGA mechanism in other diseases in addition to developing further programs in some of the largest unmet medical need indications in neurology.”

Oscar Pérez, Chief Business Development and Global Alliances Officer at Ferrer, added: “Researching a cure for such a devastating condition that lacks available therapeutic options fits perfectly with Ferrer’s mission to develop transformative treatments. We are seeking to generate life-changing solutions for people with PSP. ASN90 is a very promising addition to our growing portfolio in neurological disorders, making Ferrer an increasingly relevant player in the field of neuroscience.”

Ferrer will now conduct a phase II clinical study to determine the efficacy and optimal dose range of ASN90.
SVB Securities acted as exclusive financial advisor to Asceneuron.