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

Cerevance Announces Presentation of Preclinical Study Results at the Society for Neuroscience Conference

Data demonstrated C101248 is a promising inhibitor of THIK-1, an emerging target in Alzheimer’s disease.
THIK-1 was identified using Cerevance’s proprietary NETSseq platform.

 Boston, MA – November 15, 2022 – Cerevance, a private, clinical-stage drug discovery and development company focused on central nervous system (CNS) diseases, announced the presentation of preclinical study results during the poster session held November 14th at the Society for Neuroscience (SFN) 2022 conference in San Diego, California.

“We are incredibly pleased to have identified not only a new target for the treatment of Alzheimer’s disease, but also a means to inhibit inflammatory signaling associated with THIK-1 in cellular models,” said Mark Carlton, Ph.D., chief scientific officer of Cerevance.  “NETSseq is a powerful tool to identify previously unelucidated drug targets for the development of novel therapies.”

The presentation entitled “Identification of THIK-1 as a Therapeutic Target For Alzheimer’s Disease: Characterization of a Selective and Novel Blocker,” demonstrated the power of the proprietary Nuclear Enriched Transcript Sort sequencing (NETSseq) platform in identifying a novel target.

Key highlights of the presentation include:

  • Using NETSseq, tandem pore domain halothane-inhibited potassium channel 1 (THIK-1) demonstrated significantly restricted gene expression in microglia which is increased in Alzheimer’s diseased human brain samples.
  • C101248, Cerevance’s novel inhibitor, demonstrated a concentration-dependent inhibition of the protein in human and mouse cells expressing THIK-1 with similar potency and maximum effect as measured by thalium assays and patch-clamp experiments.
  • Inhibition of THIK-1 with C101248 resulted in reduced activity of the NLRP3 inflammasome and a reduction of the cytokine IL-1β, key components in the development of neuroinflammation in Alzheimer’s disease.
  • Overall, the data demonstrate the promise of blocking the target THIK-1 with molecules such as C101248 for the treatment of Alzheimer’s disease.

Cerevance’s proprietary NETSseq approach enables the company to comprehensively profile specific brain cell types – including both neurons and glial cells – in mature human brain tissue. The approach involves using antibodies against nuclear proteins (e.g., transcription factors), endoplasmic reticulum proteins and membrane proteins, as well as RNA probes against any cell-type-specific transcripts. These probes tag specific cell types in brain tissue to enable the sorting of nuclei using fluorescence-activated cell sorting (FACS).

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 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

Cerevance to Present at the Society for Neuroscience (SFN) 2022

Boston, MA – November 7, 2022 – Cerevance, a private, clinical-stage drug discovery and development company focused on central nervous system (CNS) diseases, today announced a poster presentation at the Society for Neuroscience (SFN) 2022 conference, to be held in San Diego, California and virtually on November 12-16, 2022.

Presentation Details

Title:  Identification of THIK-1 as a therapeutic target for Alzheimer’s disease and characterization of a selective and novel blocker

Presentation Number:  366.05

Presenter:  Madhurima Dey, Neuroscientist, Cell Biology and Target Validation
Session Title:  Microglia and Neurodegeneration
Presentation Time:  Monday, November 14, 2022, 1:00 PM – 2:00 PM PT

For more information and registration details, see the SFN website here.

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 selective novel target proteins that are either over- or under-expressed in diseased brains. Partnering with over 20 brain banks and evaluating more than 11,000 human post-mortem brain tissue samples, Cerevance is advancing a robust pipeline of targeted treatments for patients with neurodegenerative diseases, including Parkinson’s disease 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

Cerevance Appoints Ottavio V. Vitolo, M.D., as Chief Medical Officer

Boston, MA – October 3, 2022 – Cerevance, a private, clinical-stage drug discovery and development
company focused on central nervous system (CNS) diseases, today announced the appointment of
Ottavio V. Vitolo, M.D. as chief medical officer. Dr. Vitolo brings more than 20 years of experience in
neuroscience research and drug development. He joins Cerevance to lead the global clinical
development and regulatory operations.

“We are thrilled to welcome Ottavio whose expertise will help advance our broad portfolio of innovative
treatments for neurodegenerative diseases,” said Craig Thompson, chief executive officer of Cerevance.
“His diverse and extensive experience in translational and clinical research will be an invaluable asset as
we strategically advance our pipeline into and through the clinic.”

Dr. Vitolo most recently served as chief medical officer and global head of R&D at Alcyone Therapeutics.
Prior to this position, Dr. Vitolo served as senior vice president, head of R&D and chief medical officer at
Relmada Therapeutics Inc. While at Relmada, he critically contributed to the company’s growth and
strategy, including leading the successful initiation and completion of dextromethadone clinical studies.
Earlier in his career, Dr. Vitolo was vice president of clinical development at Homology Medicines and
held positions of increasing responsibilities at Pfizer, including senior director and head of
neuromuscular clinical research, research project lead, global clinical lead and medical director in the
neuroscience research unit.

Dr. Vitolo is a trained and practicing neuropsychiatrist. After receiving his medical degree from the
University of Rome La Sapienza, he completed his psychiatry residency at Washington University in St.
Louis and completed a fellowship in behavioral neurology and neuropsychiatry at Brigham and Women’s
Hospital and Massachusetts General Hospital. Dr. Vitolo also has a Master of Medical Sciences degree
from Harvard Medical School.

Dr. Vitolo stated, “I am delighted to join Cerevance at such a pivotal time in the company’s history. The
potential of the NETSseq target discovery platform combined with the broad pipeline creates a
tremendous opportunity to bring several novel and potentially transformative therapies to patients.”

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 highly selectively identify novel target
proteins that are either over- or under-expressed in diseased brains. Partnering with over 20 brain banks
and evaluating more than 11,000 human post-mortem brain tissue samples, Cerevance is advancing a
robust pipeline of targeted treatments for patients with neurodegenerative diseases, including
Parkinson’s disease 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

Cerevance Establishes Strategic Research Collaboration with Merck for the Discovery of Novel Targets in Alzheimer’s Disease

Cerevance to concurrently out-license one discovery-stage program

Boston, MA – AUGUST 9, 2022 – Cerevance, a private, clinical-stage drug discovery and development
company focused on central nervous system (CNS) diseases, today announced a multi-year strategic
research collaboration with Merck, known as MSD outside the United States and Canada, to identify novel
targets for Alzheimer’s disease utilizing Cerevance’s proprietary Nuclear Enriched Transcript Sort
sequencing (NETSseq) technology platform. Cerevance will concurrently out-license one discovery-stage
program to Merck as part of the collaboration.

Under the terms of the agreement, Cerevance will receive a $25 million upfront payment and is eligible
to receive development and commercial milestone payments totaling approximately $1.1B, in addition to
potential royalties on sales of approved products derived from the collaboration.

“The establishment of this collaboration with Merck, which comes on the heels of our positive Phase 2
data for CVN424 in patients with Parkinson’s disease, represents a significant milestone for Cerevance
and reinforces the promise of our NETSseq technology platform,” said Mark Carlton, Ph.D., chief scientific
officer of Cerevance. “We believe we are well-positioned to identify novel targets for neurodegenerative
diseases and look forward to collaborating with Merck to potentially bring forward transformative
therapeutics for patients with Alzheimer’s disease.”

“Progress in our understanding of the biology of neurodegenerative diseases continues to reveal
compelling new mechanisms for potential therapeutic intervention,” said Jason M. Uslaner, Ph.D., vice
president and head of neuroscience discovery, Merck Research Laboratories. “We look forward to
advancing the discovery program as well as taking advantage of the NETSseq platform to identify new
targets with the team at Cerevance.”

To date, Cerevance’s NETSseq technology platform has isolated and allowed for analysis of specific cell
populations in thousands of post-mortem, healthy and diseased human brain tissue samples across a
range of ages and brain regions. These analyses of human brain tissue can expose biological pathways
underlying neurodegenerative and psychiatric diseases that would be difficult to see in animal models or
differentiated human stem cells. As a result, Cerevance’s platform can reveal novel therapeutic targets
that can be modulated to correct neural circuitry or slow the disease process.

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 highly selectively identify novel target
proteins that are either over- or under-expressed in diseased brains. Partnering with over 20 brain banks
and evaluating more than 11,000 human post-mortem brain tissue samples, Cerevance is advancing a
robust pipeline of targeted treatments for patients with neurodegenerative diseases, including
Parkinson’s disease 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

Cerevance to Present During the 5th Annual LSX World Congress USA

Boston, MA – June 16, 2022 – Cerevance, a private, clinical-stage drug discovery and development company focused on central nervous system (CNS) diseases, today announced that Carrie Cook, chief business officer (CBO) of Cerevance, will participate in a fireside chat during the 5th Annual LSX World Congress USA being held in Boston, MA, June 21-22. Ms. Cook will share her thoughts around the evolving mergers and acquisitions (M&A) landscape, including how to prepare for a successful merger and how to best integrate teams and cultures from both parties involved.

Presentation Details:

Title: “Expert Insight Fireside chat: Merging of teams, cultures and assets – when M&A makes sense and how to prepare”
Date: Wednesday, June 22, 2022
Time: 3:30 p.m. ET

Speakers:

  • Carrie Cook – CBO of Cerevance
  • Douglas Fambrough – Former CEO of Dicerna
  • Chuck Triano – CFO of Xalud Therapeutics
  • Ruta Laukien – Managing Partner of Graybella Capital

For more information and registration details, see the LSX website here.

About Cerevance

Cerevance is a private pharmaceutical company whose lead therapeutic, CVN424, a first-in-class compound acting on a novel target, recently demonstrated significant and clinically meaningful efficacy in a 135-patient phase 2 study in Parkinson’s Disease. The company uses its proprietary NETSseq platform to highly selectively identify novel target proteins that are either over- or under-expressed. Partnering with over 20 brain banks and evaluating more than 11,000 brain tissue samples, Cerevance is advancing therapeutics with novel mechanisms of action for diseases such as Parkinson’s, amyotrophic lateral sclerosis (ALS), and Alzheimer’s. 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

Cerevance to Participate in Panel Discussion During the 2022 BIO International Convention

Boston, MA – June 8, 2022 – Cerevance, a private, clinical-stage drug discovery and development company focused on central nervous system (CNS) diseases, today announced that Craig Thompson, chief executive officer of Cerevance, will participate in a panel discussion during the 2022 BIO International Convention being held in San Diego, CA, June 13-16. Mr. Thompson will present at the panel titled, “CNS drug development: Do biomarkers hold the future for patients?” and will share his insights on the future of neurological drug development alongside other leading CNS experts in the life sciences industry.

Panel Details:

Title: “CNS drug development: Do biomarkers hold the future for patients?”
Moderator: Allison DeAngelis, STAT News Reporter
Date: Wednesday, June 15, 2022
Time: 1:45 p.m. PT – 2:45 p.m. PT

Panelists:

  • Craig Thompson, M.B.A. – CEO of Cerevance
  • Dolo Diaz, Ph.D. – VP, Development Sciences of Denali Therapeutics
  • Dhivya Venkat – CEO of Esya Labs
  • Amit Etkin, M.D., Ph.D. – Co-Founder and CEO of Alto Neuroscience

For more information and registration details, see the BIO website here.

About Cerevance

Cerevance is a private pharmaceutical company whose lead therapeutic, CVN424, a first-in-class compound acting on a novel target, recently demonstrated significant and clinically meaningful efficacy in a 135-patient phase 2 study in Parkinson’s Disease. The company uses its proprietary NETSseq platform to highly selectively identify novel target proteins that are either over- or under-expressed. Partnering with over 20 brain banks and evaluating more than 11,000 brain tissue samples, Cerevance is advancing therapeutics with novel mechanisms of action for diseases such as Parkinson’s, amyotrophic lateral sclerosis (ALS), and Alzheimer’s. 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

Cerevance Announces Leadership Transition to Implement Next Phase of Growth in Advancing Novel Therapeutics for Brain Diseases

Craig Thompson Appointed Chief Executive Officer
Founder Brad Margus Assumes Role of Executive Chairman of the Board

Boston, MA – April 19, 2022 – Cerevance, a private, clinical-stage drug discovery and development company focused on central nervous system diseases, today announced the appointment of Craig Thompson to chief executive officer. Mr. Thompson brings over 25 years of commercial and marketing leadership experience in the biotech and pharmaceutical industries to the company. Brad Margus will transition from CEO to Executive Chairman of the Board at the end of April.

“It’s been a privilege to lead our talented team over the last six years. Together, we have scaled a powerful target discovery platform, assembled a top-tier group of investors and advanced a portfolio of novel therapeutics to clinical proof of concept,” said Mr. Margus. “Now, Craig’s robust track record of executing later-stage clinical development and leading commercialization of pharmaceutical products will ensure that Cerevance succeeds in its next phase of growth. I look forward to supporting the company as Executive Chairman going forward.”

“I’m thrilled to be joining the Cerevance team as CEO,” said Mr. Thompson. “The company is on the cusp of translating exciting new therapeutic targets into potential clinical treatments that hold real promise for millions of people suffering from neurological and psychiatric diseases. The data from our recent CVN-424 Phase 2 clinical study in Parkinson’s disease validates our development platform.”

Mr. Thompson most recently served as President and CEO at Neurana Pharmaceuticals, a private company focused on the treatment of neuromuscular conditions. Prior to Neurana, he served as President and CEO of Anthera Pharmaceuticals, and before that, as chief operating officer of Tetraphase Pharmaceuticals, where he oversaw the development and implementation of the marketing, access, reimbursement and sales strategy, as well as business development and commercial manufacturing. Prior to Tetraphase, he was chief commercial officer for Trius Therapeutics, which was acquired by Cubist Pharmaceuticals for more than $700 million. Earlier in his career, Mr. Thompson served in various commercial and marketing roles of increasing responsibility at Pfizer and Merck. He holds a Bachelor of Commerce degree from McMaster University and an M.B.A. from the University of Notre Dame.

About Cerevance

Cerevance is a private pharmaceutical company whose lead program, CVN424, a first-in-class compound acting on a novel target, recently demonstrated significant and meaningful efficacy in a 135-patient phase 2 study in Parkinson’s Disease. The company uses its NETSseq transcriptional and epigenetic profiling platform to examine neuronal and glial cell populations in thousands of human donor brain tissue samples obtained from over 20 brain bank partners around the world. Then, drawing on its comprehensive understanding of the molecular characteristics of specific cell types, Cerevance is advancing a pipeline of therapeutic programs against novel targets that address the selective cellular vulnerability of neurodegenerative disease, reduce neuroinflammation without perturbing the peripheral immune system, and normalize dysfunctional circuits that are at the root of many motor and behavioral disorders.

Contacts

Cerevance:
Robert Middlebrook, +1-408-220-5722

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

Cerevance Reports Positive Phase 2 Clinical Trial Results with CVN424, a Parkinson’s Disease Drug Working Through a New Mechanism

Demonstrates the Power of Cerevance’s Deep Understanding of Specific Human Brain Cell Types

Boston, MA – March 31, 2022 – Cerevance, a private, clinical-stage drug discovery and development company focused on central nervous system diseases, today announced the completion of its Phase 2 clinical trial of CVN424, the company’s first-in-class, once-a-day, orally-delivered compound in development for the treatment of Parkinson’s disease. Beyond meeting safety objectives, the drug achieved a significant and meaningful, dose-dependent reduction of “OFF time,” which refers to periods of the day when Parkinson’s symptoms recur despite medication. It also had an encouraging side-effect profile.

CVN424 was evaluated in a randomized, double-blind, placebo-controlled multicenter Phase 2 study at two dose levels in Parkinson’s disease patients with motor fluctuations. Approximately 135 subjects with Parkinson’s disease, on a stable dosage of levodopa and other Parkinson’s medications but with at least two hours or more per day of average OFF time, were enrolled. Following baseline safety and efficacy assessments, subjects were randomized to receive once-daily doses of low-dose CVN424, high-dose CVN424 or matching placebo for four weeks.

At the high dose, CVN424 showed a 1.3-hour improvement in OFF time compared to placebo (p=0.042) at four weeks. This was accompanied by an increase in ON time without Troublesome Dyskinesia, without a meaningful worsening of ON time with Troublesome Dyskinesia. Efficacy improved at four weeks versus at two weeks, and daytime sleepiness as measured by the Epworth Sleepiness Scale was reduced compared to placebo, differentiating it from most Parkinson’s disease drugs used as adjuncts to levodopa. At the lower dose, CVN424 also demonstrated a meaningful improvement in OFF time and ON time without Troublesome Dyskinesia compared to placebo. The most common adverse reactions were nausea, vomiting and headache, occurring in two subjects (4%) each at the higher dose. All other adverse reactions occurred in one subject or less.

“We are delighted to report these results which we believe demonstrate that CVN424 can provide a significant improvement for patients, with little exacerbation of dopaminergic side effects,” said Brad Margus, chief executive officer of Cerevance. “We look forward to rapidly advancing CVN424 into several larger clinical studies aimed at obtaining regulatory approval.”

Mark Carlton, Ph.D., chief scientific officer of Cerevance, added, “CVN424’s positive results demonstrate the power of the deep, cell-type-specific transcriptional and epigenetic data we are generating by applying our NETSseq platform technology to thousands of post-mortem human brain tissue samples. These data also increase the confidence we have in our pipeline of additional programs against novel targets identified by our approach.”

“For more than 50 years, physicians have relied on therapeutics that work by directly increasing dopaminergic signaling,” said Karl Kieburtz, M.D., President of Clintrex, a group of clinical development and regulatory experts who have collectively led more than 75 international multicenter clinical trials of treatments for movement disorders and neurodegenerative diseases involving tens of thousands of patients. “This new mechanism holds great promise for treating the motor fluctuations eventually experienced by all Parkinson’s disease patients, as well as potential for treating patients in the earlier stages of the disease.”

Cerevance intends to pursue additional Phase 2/3 clinical studies with CVN424 to establish a clear path to regulatory approval of the drug for adjunctive therapy. In parallel, the company will evaluate the drug’s promise as stand-alone treatment for recently diagnosed patients not yet treated with levodopa.

About Cerevance

Cerevance, a private, clinical-stage pharmaceutical company focused on brain diseases, is applying a new technology called NETSseq to reveal transcriptional and epigenetic differences between specific cell types in post-mortem human brain tissue acquired from donors whose ages varied from eight to 104 years old. NETSseq profiles neuronal and glial cell populations at depths not possible with other approaches, generating unprecedented data sets and insights. The company has thus far partnered with more than 20 brain banks around the world to assemble a growing collection of more than 10,000 clinically annotated samples from healthy and diseased donors. By applying NETSseq to specific cell types critical to circuits disrupted by disease and comparing vulnerable and resilient cell populations, Cerevance’s scientists have begun identifying targets and advancing a pipeline of novel therapeutics that modulate them to treat CNS diseases.

Contacts

Cerevance:
Robert Middlebrook, +1-408-220-5722

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

Cerevance Doses First Subjects in Phase 1 Clinical Trial of CVN766

Study will evaluate safety and pharmacokinetics of potential treatment for psychiatric disorders

Boston, MA – January 25, 2022 – Cerevance, a private, clinical-stage drug discovery and development company focused on central nervous system diseases, today announced the dosing of the first human subjects in a Phase 1 clinical trial of its compound, CVN766.

CVN766 is a potent and highly selective small molecule antagonist of the orexin 1 receptor (Ox1R), a target with genetic links to panic disorder that is expressed in areas of the brain important for regulating fear, anxiety, reward and emotion. When stimulated, orexigenic neurons cause anxiety, while antagonizing Ox1R reduces this effect. CVN766 was found to be effective in various animal models of psychiatric diseases, including reversing nicotine-induced stress in rodents and acute stress behaviors in non-human primate models of panic.

“Increases of orexin levels in cerebral spinal fluid and plasma have been seen in patients with panic disorder and in a subset of patients with schizophrenia. Because of these phenomena, we believe there may be opportunities to stratify patients and also to employ these potential biomarkers to monitor treatment responses,” said Nicola Brice, Ph.D., director of neuroscience at Cerevance.

Cerevance chief business officer, Carrie Cook, noted, “We believe CVN766 may have an advantage over other Ox1R antagonists currently in development, given its extremely high selectivity against orexin 2.”  Cook added, “While dual orexin antagonists have previously been developed for sleep aid, we believe that CVN766’s selectivity against solely Ox1R could prove therapeutic in a range of psychiatric disorders.”

The Phase 1, randomized, double-blind, placebo-controlled trial will assess the safety, tolerability and pharmacokinetics of escalating single and multiple doses of CVN766 in healthy subjects. The company expects to complete the Phase 1 study by year-end.

Cerevance is continuing to actively enroll patients in the Phase 1 study. To learn more about this trial, please visit www.clinicaltrials.gov.

About Cerevance

Cerevance, a private, clinical-stage pharmaceutical company focused on brain diseases, is applying a new technology called NETSseq to reveal transcriptional and epigenetic differences between specific cell types in post-mortem human brain tissue acquired from donors whose ages varied from eight to 104 years old.   NETSseq profiles neuronal and glial cell populations at depths not possible with other approaches, generating unprecedented data sets and insights. The company has thus far partnered with more than 20 brain banks around the world to assemble a growing collection of more than 10,000 clinically annotated samples from healthy and diseased donors. By applying NETSseq to specific cell types critical to circuits disrupted by disease and comparing vulnerable and resilient cell populations, Cerevance’s scientists have begun identifying targets and advancing a pipeline of novel therapeutics that modulate them to treat CNS diseases.

Contacts

Cerevance:
Robert Middlebrook, +1-408-220-5722

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