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Cerevance Announces Presentation of Preclinical Data During the Cold Spring Harbor Laboratory (CSHL) Neurodegenerative Diseases – Biology and Therapeutics Conference and Publication in Neuropharmacology

Presentation highlights a novel target identified for Alzheimer’s disease using Cerevance’s proprietary NETSseq platform

Cerevance’s THIK-1 targeting inhibitor, C101248, demonstrated positive results in human and mouse cell based and electrophysiology assays relevant to neuroinflammation in neurodegenerative disease

 Data has been concurrently published in the peer-reviewed journal, Neuropharmacology

Boston, MA – November 30, 2022 – Cerevance, a private, clinical-stage drug discovery and development company focused on central nervous system (CNS) diseases, today announced the presentation of preclinical data supporting the use of the company’s Nuclear Enriched Sort sequencing (NETSseq) technology platform to identify novel, druggable targets involved in the regulation of neuroinflammation in Alzheimer’s disease (AD). The data will be presented during the CSHL Neurodegenerative Disease – Biology and Therapeutics Conference held at the Cold Spring Harbor Laboratory from November 30 – December 2, 2022.

In addition, this data has been published in the peer-reviewed journal Neuropharmacology. The article, titled, Characterisation of C101248: A novel selective THIK-1 channel inhibitor for the modulation of microglial NLRP3-inflammasome,” is available on the publisher’s website.

“The recent preclinical data and subsequent publication are incredibly important in further validating NETSseq’s ability to uncover never-before-targeted mechanisms in the treatment of Alzheimer’s disease,” said Mark Carlton, Ph.D., chief scientific officer of Cerevance. “CN101248, the first selective small molecule THIK-1 inhibitor, attacks neuroinflammation via THIK-1 and we have observed beneficial effects on neuroinflammation with this compound. Our development of CN101248 has demonstrated THIK-1’s involvement in inflammatory signaling in microglia, the brain’s immune cells, and serves as a promising avenue of further research.”

The presentation during the CSHL conference, titled, “Using Cerevance’s NETSseq platform to identify targets involved in neuroinflammation in Alzheimer’s disease,” highlights the ability of the company’s NETSseq platform to discover targets affecting inflammatory processes associated with AD.

Key highlights of the presentation include:

  • Tandem pore domain halothane-inhibited potassium channel 1 (THIK-1) demonstrated significantly higher gene expression in microglia in diseased human brain samples.
  • Increasing THIK-1 gene expression was shown to correspond with disease progression in AD.
  • C101248 demonstrated a selective and concentration-dependent inhibition of the THIK-1 protein in human and mouse cells.
  • In mouse microglia, inhibition of THIK-1 with C101248 attenuated activity of the NLRP3 inflammasome and corresponded with a reduction of IL-1β, key components in the development of inflammation in AD.
  • Overall, the data demonstrate the promise of C101248 as a candidate for the treatment of AD.

About Cerevance

Cerevance is a private pharmaceutical company whose lead therapeutic, CVN424, a first-in-class, oral, non-dopaminergic compound acting on a novel target (GPR6), recently demonstrated significant and clinically meaningful efficacy in a 135-patient Phase 2 study in patients with Parkinson’s disease. The company uses its proprietary NETSseq technology platform to identify highly selectively novel target proteins that are either specific to certain brain circuits or are over- or under-expressed in diseased brains. Partnering with over 25 brain banks and evaluating more than 12,000 human post-mortem brain tissue samples, Cerevance is advancing a robust pipeline of targeted treatments for patients with neurodegenerative diseases, including Parkinson’s disease, Amyotrophic Lateral Sclerosis and Alzheimer’s disease. For additional information, please visit www.cerevance.com.

Contacts

Cerevance:
Johnna Simoes, ir@cerevance.com

Media:
Andrew Mielach, amielach@lifescicomms.com, +1-646-876-5868

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