Company’s small molecule OGA inhibitors have shown potential to improve protein aggregation in Alzheimer’s, Parkinson’s and ALS.
Swiss biotech Asceneuron has announced the successful completion of an oversubscribed $100 million Series C financing round to advance its pipeline of treatments for neurodegenerative conditions. The developer of small molecule OGA (O-GlcNAcase) inhibitors targeting tau protein aggregation says the new funding will propel its lead candidate into Phase 2 clinical trials for the treatment of Alzheimer’s.
In neurodegenerative diseases, the build-up of the tau protein in the brain results in tangles that disrupt the structure and function of neurons, ultimately leading to conditions including Alzheimer’s, Parkinson’s and amyotrophic lateral sclerosis (ALS). With a pipeline that focuses on therapies that can penetrate the brain fully, Asceneuron says its drugs have produced promising preclinical results in improving the pathologies of both tau and α-synuclein, another protein linked to Parkinson’s.
The company’s lead asset, ASN51, is an orally administered small molecule drug designed to inhibit the OGA enzyme, which is implicated in the aggregation of tau proteins and the progression of Alzheimer’s disease. The mechanism has also shown potential in preventing protein aggregation in other neurodegenerative diseases, including Parkinson’s and ALS.
Barbara Angehrn Pavik, CEO of Asceneuron
According to Asceneuron, multiple Phase 1 clinical trials have demonstrated that ASN51 can achieve full central nervous system uptake and high OGA enzyme occupancy, and the company now plans to initiate its first Phase 2 clinical trial for ASN51 later this year.
“This high caliber life sciences investor syndicate further validates the potential of our OGA inhibitor pipeline and leadership in the field of tauopathies,” said Barbara Angehrn Pavik, CEO of Asceneuron. “We are excited to advance our lead asset ASN51 into Phase 2 clinical development, recognizing its potential to significantly expand treatment options for patients with Alzheimer’s disease.”
The financing round was led by Novo Holdings, with new investments from EQT Life Sciences’ LSP Dementia Fund, OrbiMed, and SR One.
“Alzheimer’s disease is undergoing a transformational moment… We are now witnessing the approvals of the first disease modifying antibody based injectable therapies,” said Novo Holdings’ Dr Naveed Siddiqi. “Asceneuron’s innovative oral small molecule drug targeting intracellular tau offers the potential for a paradigm shift in the way this neurodegenerative disease is treated.”
Existing investors, including M Ventures, Sofinnova Partners, GSK Equities Investments Limited, and Johnson & Johnson Innovation, also participated.
“Asceneuron has successfully developed highly differentiated oral OGA inhibitors from pre-clinical development to where they are today, entering Phase 2 development in patients with Alzheimer’s disease,” said Dr Hakan Goker, of M Ventures. “ASN51 holds great promise as a next generation treatment for Alzheimer’s disease in addition to addressing other neurodegenerative diseases, including Parkinson’s disease and amyotrophic lateral sclerosis.”
Photograph: ktsimage/Envato
