CAMP4 Therapeutics (Nasdaq: CAMP) highlights new CMP-002 seizure data and trial plan
Filing Impact
Filing Sentiment
Form Type
8-K
Rhea-AI Filing Summary
CAMP4 Therapeutics Corporation filed an 8-K to share a corporate update and investor materials highlighting new preclinical data for CMP-002 in SYNGAP1-related disorder. The company presented these findings and an updated slide deck on May 14, 2026.
In a SYNGAP1 haploinsufficient mouse model with chemically induced seizures, a single dose of CMP-002 produced statistically significant improvements in both seizure threshold and seizure severity. These results build on earlier motor and behavioral data, suggesting CMP-002 may address multiple neurological symptoms by restoring SYNGAP1 protein toward healthy levels.
The company describes SYNGAP1-related disorder as a rare central nervous system disease affecting over 10,000 people in the U.S., with high rates of intellectual disability and epilepsy and no approved disease-modifying therapies. CAMP4 expects to advance CMP-002, an antisense oligonucleotide designed to upregulate SYNGAP1, into a global Phase 1/2 clinical trial in the second half of 2026.
Positive
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Insights
Preclinical seizure data support CMP-002’s potential but remain early-stage.
CAMP4 Therapeutics reports that CMP-002 improved seizure threshold and severity in a SYNGAP1 haploinsufficient mouse model using a pentylenetetrazol induction paradigm. This extends prior preclinical evidence of motor and behavioral benefit and supports CMP-002 as a broad, disease-modifying candidate for SYNGAP1-related disorder.
The candidate is an antisense oligonucleotide targeting regulatory RNA to boost SYNGAP1 expression, fitting CAMP4’s platform strategy to amplify healthy protein. The disorder has substantial unmet need and no disease-modifying therapies, which makes a successful program potentially meaningful, but these results are limited to animal models.
The company plans a global Phase 1/2 clinical trial in the second half of 2026, which will begin to test safety and early signals in patients. Actual impact will depend on human safety, pharmacology, and whether seizure and neurodevelopmental benefits translate beyond preclinical models.
8-K Event Classification
2 items: 7.01, 9.01
2 items
Item 7.01
Regulation FD Disclosure
Disclosure
Material non-public information disclosed under Regulation Fair Disclosure, often investor presentations or guidance.
Item 9.01
Financial Statements and Exhibits
Exhibits
Financial statements, pro forma financial information, and exhibit attachments filed with this report.
Key Figures
SYNGAP1 protein level in disorder: approximately 50% of normal
U.S. SYNGAP1 patient count: over 10,000 individuals
Epilepsy prevalence in SYNGAP1: approximately 85%
+4 more
7 metrics
SYNGAP1 protein level in disorder
approximately 50% of normal
Described level in SYNGAP1-related disorder patients
U.S. SYNGAP1 patient count
over 10,000 individuals
Estimated number of affected individuals in the United States
Epilepsy prevalence in SYNGAP1
approximately 85%
Proportion of SYNGAP1-related disorder patients with epilepsy
Behavioral problems prevalence
approximately 70%
Proportion of SYNGAP1-related disorder patients with severe behavioral problems
Sleep problems prevalence
approximately 60%
Proportion of SYNGAP1-related disorder patients with sleep problems
Non-verbal patients share
approximately 30%
Proportion of SYNGAP1-related disorder patients who are non-verbal
Planned Phase 1/2 start
second half of 2026
Expected timing to advance CMP-002 into a clinical trial
Key Terms
antisense oligonucleotide, regulatory RNA, haploinsufficient, disease-modifying therapies, +2 more
6 terms
antisense oligonucleotide medical
"CMP-002, the Company's lead investigational antisense oligonucleotide (ASO) therapeutic candidate for SYNGAP1-related disorder"
An antisense oligonucleotide is a small piece of synthetic genetic material designed to attach to specific molecules in the body’s cells, effectively blocking or modifying how genes are expressed. This technology is important because it can be used to develop targeted treatments for certain diseases, which may influence the value of biotech companies and the broader healthcare sector. Its development reflects advances in personalized medicine and gene-based therapies.
regulatory RNA medical
"our therapeutic ASO drug candidates target regulatory RNAs (regRNAs), which act locally on transcription factors"
Regulatory RNA are natural or engineered RNA molecules that control when and how much a gene is turned on, acting like dimmer switches or thermostats for cells rather than making proteins themselves. Investors watch them because therapies and diagnostics that target these RNAs can change disease processes, create new drug classes, or unlock patents and approvals—events that can rapidly affect a biotech company's value and market prospects.
haploinsufficient medical
"genes underlying haploinsufficient and recessive partial loss-of-function disorders, of which there are more than 1,200"
A haploinsufficient gene is one where losing or disabling just one of the two inherited copies causes a loss of normal function, because the remaining copy cannot make enough product to keep things working. This matters to investors because such genes often drive disease biology, influence how well drugs or genetic therapies must restore function, affect diagnostic and regulatory strategies, and can change the size and risk of a treatment’s potential market—like a two-engine plane that can’t fly properly on one engine.
disease-modifying therapies medical
"There are currently no approved disease-modifying therapies for patients living with SYNGAP1."
Disease-modifying therapies are treatments intended to change the underlying course of a chronic illness by slowing, stopping, or reversing the biological process that causes harm, rather than only relieving symptoms. For investors they matter because therapies that alter disease progression can reach more patients, support longer-term use and higher pricing, and shift risk/reward profiles in development and approval — like repairing a building’s foundation instead of patching visible cracks.
Phase 1/2 clinical trial medical
"CAMP4 expects to advance CMP-002 into a Phase 1/2 clinical trial in individuals with SYNGAP1-related disorder in the second half of 2026."
A phase 1/2 clinical trial is an early human study that combines first-in-people safety and dosing checks (phase 1) with an initial look at whether the treatment appears to work (phase 2). Think of it as a short test drive where researchers both confirm the product won’t cause serious harm and gather early signs it could be effective; for investors, successful results reduce risk and can unlock value-creating milestones like larger trials or regulatory discussions.
forward-looking statements regulatory
"This press release contains forward-looking statements which involve risks, uncertainties and contingencies"
Forward-looking statements are predictions or plans that companies share about what they expect to happen in the future, like estimating sales or profits. They matter because they help investors understand a company's outlook, but since they are based on guesses and assumptions, they can sometimes be wrong.
UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
FORM 8-K
CURRENT REPORT
Pursuant to Section 13 or 15(d)
of the Securities Exchange Act of 1934
Date of Report (Date of earliest event reported): May 14, 2026
(Exact name of registrant as specified in its charter)
| (State or other jurisdiction of incorporation) | (Commission File Number) | (IRS Employer Identification No.) | ||||||||||||
| (Address of principal executive offices) | (Zip Code) | |||||||||||||
(Registrant’s telephone number, including area code): (617 ) 651-8867
Not Applicable
(Former name or former address, if changed since last report)
Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions (see General Instruction A.2. below):
| Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425) | |||||
| Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12) | |||||
| Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b)) | |||||
| Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c)) | |||||
Securities registered pursuant to Section 12(b) of the Act:
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Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (§240.12b-2 of this chapter).
Emerging growth company x
If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ¨
Item 7.01 Regulation FD Disclosure.
On May 14, 2026, CAMP4 Therapeutics Corporation (the “Company”) issued a press releases titled “CAMP4 Therapeutics to Present New Preclinical Data Demonstrating CMP-002 Improves Seizure Threshold and Severity in a Model of SYNGAP1-Related Disorder,” a copy of which is furnished as Exhibit 99.1 hereto.
On May 14, 2026, the Company also updated its corporate slide presentation, a copy of which is furnished as Exhibit 99.2 hereto.
The information in this Item 7.01 and Exhibits 99.1 and 99.2 attached hereto is being furnished and shall not be deemed “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the “Exchange Act”) or otherwise subject to the liabilities of that Section, nor shall it be deemed incorporated by reference into any registration statement or other filing under the Securities Act of 1933, as amended (the “Securities Act”), or the Exchange Act, except as shall be expressly set forth by specific reference in such a filing.
Item 9.01 Financial Statements and Exhibits.
(d) Exhibits
| Exhibit No. | Description | |||||||
| 99.1 | Press release issued by CAMP4 Therapeutics Corporation on May 14, 2026. | |||||||
| 99.2 | Slide presentation, dated May 2026. | |||||||
| 104 | Cover Page Interactive Data File (embedded within the Inline XBRL document). | |||||||
SIGNATURES
Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.
| CAMP4 THERAPEUTICS CORPORATION | ||||||||
| By: | /s/ Josh Mandel-Brehm | |||||||
| Name: Josh Mandel-Brehm | ||||||||
| Title: President and Chief Executive Officer | ||||||||
Date: May 14, 2026
CAMP4 Therapeutics to Present New Preclinical Data Demonstrating CMP-002 Improves Seizure Threshold and Severity in a Model of SYNGAP1-Related Disorder
CMP-002 administration resulted in a statistically significant improvement in seizure phenotypes and parameters in a SYNGAP1 haploinsufficient mouse model
Results build upon prior preclinical evidence of neurodevelopmental benefit and suggest the potential for broader therapeutic impact
CAMBRIDGE, Mass., May 14, 2026 (GLOBE NEWSWIRE) -- CAMP4 Therapeutics Corporation ("CAMP4" or "the Company") (Nasdaq: CAMP), a clinical-stage biopharmaceutical company developing a pipeline of regulatory RNA-targeting therapeutics designed to upregulate gene expression with the goal of restoring healthy protein levels to treat a broad range of genetic diseases, today announced the presentation of new preclinical data for CMP-002, the Company's lead investigational antisense oligonucleotide (ASO) therapeutic candidate for SYNGAP1-related disorder (SRD), at the TIDES Oligonucleotide & Peptide Therapeutics conference on May 14, 2026.
The new data demonstrate that CMP-002 administration produced a statistically significant improvement in both seizure threshold and severity of chemically-induced tonic-clonic seizures in mice haploinsufficient for SYNGAP1.
"SYNGAP1-related disorder is characterized by a constellation of neurological symptoms, of which seizures are among the most common, resulting in a devastating burden on patients and their families," said Daniel Tardiff, PhD, Chief Scientific Officer of CAMP4. "Our prior work established that CMP-002 can meaningfully restore motor and behavioral function in preclinical models, and these new seizure data are an important extension of that story. Given this evidence, we believe that by restoring SYNGAP1 protein towards healthy levels, CMP-002 may address a broad range of symptoms that define this disease. We look forward to sharing these findings with the broader oligonucleotide therapeutics community at TIDES."
Because SYNGAP1 haploinsufficient mice do not exhibit spontaneous and readily countable seizures, the study employed a seizure induction model using pentylenetetrazol (PTZ). PTZ is a GABA receptor antagonist that increases excitatory signaling and induces seizures. In the new study, PTZ was administered to induce tonic-clonic seizures one month after administration of CMP-002 to juvenile mice. SYNGAP1 haploinsufficient mice experienced a greater seizure burden compared to wild-type mice, while intervention with a single dose of CMP-002 led to a statistically significant resistance to the onset of tonic-clonic seizures following repeated PTZ administration, as well as a statistically significant decrease in seizure severity.
Together, these data suggest that restoring SYNGAP1 protein toward healthy levels with CMP-002 may improve both the neurodevelopmental and seizure phenotypes that define SRD, supporting the potential of CMP-002 to provide broad disease-modifying benefit.
CAMP4 expects to advance CMP-002 into a Phase 1/2 clinical trial in individuals with SYNGAP1-related disorder in the second half of 2026.
About CAMP4 Therapeutics
CAMP4 is developing disease-modifying treatments for a broad range of genetic diseases where amplifying healthy protein may offer therapeutic benefits. Our approach amplifies mRNA by harnessing a fundamental mechanism of how genes are controlled. To amplify mRNA, our therapeutic ASO drug candidates target regulatory RNAs (regRNAs), which act locally on transcription factors and are the master regulators of gene expression. CAMP4's proprietary RAP Platform® enables the mapping of regRNAs and generation of therapeutic candidates designed to target the regRNAs associated with genes underlying haploinsufficient and recessive partial loss-of-function disorders, of which there are more than 1,200, in which a modest increase in protein expression may have the potential to be clinically meaningful. For more information, visit camp4tx.com.
About SYNGAP1-Related Disorder
SYNGAP1-related disorder (also referred to as SYNGAP1) is a rare, haploinsufficient CNS disorder caused by mutations in the SYNGAP1 gene, resulting in approximately 50% of normal SYNGAP protein levels. The condition affects over 10,000 individuals in the United States and is characterized by intellectual disability in 100% of patients, epilepsy in approximately 85%, severe behavioral problems in approximately 70%, sleep problems in approximately 60%, and limited communication, with approximately 30% of patients being non-verbal. There are currently no approved disease-modifying therapies for patients living with SYNGAP1.
About CMP-002
CMP-002 is CAMP4's lead investigational ASO therapeutic candidate designed to bind to a SYNGAP1-specific regRNA to increase SYNGAP1 gene expression and restore SYNGAP protein toward near wild-type levels. Administered intrathecally, CMP-002 has demonstrated dose-dependent increases in SYNGAP protein expression in patient-derived neurons, reversal of disease-relevant behavioral phenotypes in a humanized haploinsufficient mouse model, statistically significant improvement of seizure phenotypes and parameters in a chemically induced seizure mouse model, and broad brain distribution with significant SYNGAP protein upregulation in non-human primates. The Company expects to initiate a global Phase 1/2 clinical trial in SYNGAP1 patients in 2H 2026.
About TIDES Oligonucleotide & Peptide Therapeutics
TIDES is a leading annual conference focused on the development, manufacturing, and clinical advancement of oligonucleotide and peptide therapeutics. The conference convenes scientists, clinicians, and industry leaders to accelerate the translation of these modalities from discovery to the clinic.
Forward-Looking Statements
This press release contains forward-looking statements which involve risks, uncertainties and contingencies, many of which are beyond the control of the Company, which may cause actual results, performance, or achievements to differ materially from anticipated results, performance,
or achievements. All statements other than statements of historical facts contained in this press release are forward-looking statements. In some cases, you can identify forward-looking statements by terms such as "may," "will," "should," "expect," "plan," "anticipate," "could," "intend," "target," "project," "contemplate," "believe," "estimate," "predict," "potential" or "continue" or the negative of these terms or other similar expressions, although not all forward-looking statements contain these words. Forward-looking statements include, but are not limited to, statements concerning the anticipated timing to advance CMP-002 into a clinical trial and the therapeutic potential of the Company's platform technology and product candidates. The forward-looking statements in this press release speak only as of the date of this press release and are subject to a number of known and unknown risks, uncertainties and assumptions that could cause the Company's actual results to differ materially from those anticipated in the forward-looking statements, including, but not limited to: the uncertainty of preclinical and clinical development, which is lengthy and expensive, and characterized by uncertain outcomes, and risks related to additional costs or delays in completing, or failing to complete, the development and commercialization of the Company's current product candidates or any future product candidates; the Company's dependence on the services of the Company's senior management and other clinical and scientific personnel, and the Company's ability to retain these individuals or recruit additional management or clinical and scientific personnel; risks related to the manufacturing of the Company's product candidates, which is complex, and the risk that the Company's third-party manufacturers may encounter difficulties in production; the Company's ability to obtain and maintain sufficient intellectual property protection for the Company's platform technology and product candidates; and other risks and uncertainties described in the section "Risk Factors" in the Company's Annual Report on Form 10-K for the year ended December 31, 2025 and Quarterly Report on Form 10-Q for the quarter ended March 31, 2026, as well as other information the Company files with the Securities and Exchange Commission. The forward-looking statements in this press release are inherently uncertain and are not guarantees of future events. Because forward-looking statements are inherently subject to risks and uncertainties, some of which cannot be predicted or quantified and some of which are beyond the Company's control, you should not unduly rely on these forward-looking statements. The events and circumstances reflected in the forward-looking statements may not be achieved or occur and actual future results, levels of activity, performance and events and circumstances could differ materially from those projected in the forward-looking statements. Moreover, the Company operates in an evolving environment. New risks and uncertainties may emerge from time to time, and management cannot predict all risks and uncertainties. Investors, potential investors, and others should give careful consideration to these risks and uncertainties. Except as required by applicable law, the Company does not undertake to publicly update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise.
Contacts
Investor Relations:
Sara Michelmore
Milestone Advisors
sara@milestone-advisorsllc.com
Media:
Sofia Bermudez
LifeSci Communications
sbermudez@lifescicomms.com
FAQ
What did CAMP4 Therapeutics (CAMP) announce about CMP-002 in this 8-K?
CAMP4 announced new preclinical data showing CMP-002 improved seizure threshold and severity in a SYNGAP1 haploinsufficient mouse model using chemically induced seizures, and highlighted plans to advance the therapy into a global Phase 1/2 clinical trial in the second half of 2026.
How does CMP-002 work according to CAMP4 Therapeutics (CAMP)?
CMP-002 is an investigational antisense oligonucleotide designed to bind a SYNGAP1-specific regulatory RNA, increasing SYNGAP1 gene expression and restoring SYNGAP protein toward near wild-type levels, with the aim of delivering disease-modifying benefit in SYNGAP1-related disorder patients.
What preclinical results did CAMP4 (CAMP) report for CMP-002 in SYNGAP1 models?
CAMP4 reported dose-dependent increases in SYNGAP protein in patient-derived neurons, reversal of disease-relevant behavioral phenotypes in a humanized haploinsufficient mouse model, statistically significant improvement in seizure phenotypes in a chemically induced seizure model, and broad brain distribution with SYNGAP upregulation in non-human primates.
When does CAMP4 Therapeutics (CAMP) plan to start clinical trials for CMP-002?
CAMP4 expects to initiate a global Phase 1/2 clinical trial of CMP-002 in individuals with SYNGAP1-related disorder in the second half of 2026, moving the program from preclinical studies into first-in-human evaluation, subject to typical development and regulatory considerations.
What broader platform strategy does CAMP4 Therapeutics (CAMP) describe in this filing?
CAMP4 describes a platform that targets regulatory RNAs to amplify mRNA and increase healthy protein levels in haploinsufficient and recessive partial loss-of-function disorders, noting more than 1,200 such genetic diseases where modest protein increases may offer clinically meaningful benefit.