ADDED
As of March 4, 2026, there were 51,919,321 shares of the registrant s common stock, par value $0.0001 per share, outstanding.
Our lead product candidate, CMP-002, has the potential to be the first disease-modifying therapy for the treatment of synaptic Ras GTPase activating protein 1 ( SYNGAP1 )-related disorder, or SYNGAP1, a severe developmental and epileptic encephalopathy ( DEE ) characterized by seizures, developmental delays, and cognitive impairments.
SYNGAP1 is caused by haploinsufficiency of the SYNGAP1 gene, where mutation of one gene copy results in a reduction in functional SYNGAP protein levels of up to 50%.
While we believe that it remains underdiagnosed, we estimate that there are approximately 21,000 individuals living with SYNGAP1 in the United States and the five largest European markets.
There are no approved disease-modifying therapies for SYNGAP1.
CMP-002 is a novel, intrathecally delivered antisense oligonucleotide ( ASO ) designed to increase SYGNAP1 gene expression at the transcriptional level, which may increase SYNGAP protein levels in amounts sufficient to yield therapeutic benefit.
In preclinical studies, intracerebroventricular injection of CMP-002 restored SYNGAP protein levels to near normal range in haploinsufficient mice carrying a single copy of the human SYNGAP1 gene after a single dose and rescued motor defects and spatial learning defects following two doses.
In addition, biweekly intrathecal injections of CMP-002 in cynomolgus monkeys were well tolerated and significantly increased SYNGAP protein levels across multiple brain regions clinically relevant to the disease, with dose-linear increases of CMP-002 in disease-relevant brain regions.
We have initiated Good Laboratory Practice ( GLP ) toxicology studies for CMP-002 to support clinical trial applications and, pending successful completion and regulatory clearance, we intend to initiate a global Phase 1/2 clinical trial in individuals with SYNGAP1 as early as the second half of 2026.
Our product development efforts are enabled by our proprietary RAP Platform.
REMOVED
As of March 13, 2025, there were 20,161,072 shares of the registrant s common stock, par value $0.0001 per share, outstanding.
Our product candidates are in varying stages of preclinical and clinical development and we have not completed a clinical trial of any product candidate.
If these third parties do not successfully carry out their contractual duties, fail to comply with applicable regulatory requirements, or do not meet expected deadlines, our development programs may be delayed or subject to increased costs or we may be unable to obtain regulatory approval for or commercialize our product candidates; Prior to the completion of our initial public offering, there was no public market for our common stock.
An active, liquid, and orderly market for our common stock may not develop or be sustained, or we may in the future fail to satisfy the continued listing requirements of Nasdaq; and The trading price of the shares of our common stock could be highly volatile, and purchasers of our common stock could incur substantial losses.
Regulatory RNAs ( regRNAs ), play a central role in the regulation of every protein-coding gene by contributing to gene activation and suppression.
Our proprietary RNA Actuating Platform, or RAP Platform, allows us to rapidly and systematically identify and characterize the active regulatory elements controlling every expressed gene and tens of thousands of druggable enhancer and promoter regRNA sequences that control protein-coding genes.
Once a disease-associated target gene is identified, we apply our RAP Platform to identify the controlling regRNA and rapidly generate novel antisense oligonucleotide ( ASO ) candidates, which we also refer to as RNA Actuators.
We are currently focused on metabolic diseases and diseases of the central nervous system ( CNS ) with validated disease biology, and we believe our RAP Platform allows us to address a broad range of rare and prevalent genetic diseases in which a modest increase in protein expression has the potential to be clinically meaningful.
Based on our preclinical studies, we believe our lead product candidate, CMP-CPS-001, has the potential to be the first disease-modifying therapy for the treatment of the most prevalent urea cycle disorders ( UCDs ).
These preclinical studies also demonstrated that CMP-CPS-001 can increase the level of, or upregulate, the production of multiple enzymes responsible for converting ammonia into urea, potentially allowing us to address more than 85% of patients with UCDs, which we refer to as our pan-UCD approach.