CAMP4 Therapeutics Initiates GLP Toxicology Studies for CMP-SYNGAP-01
CAMP4 Therapeutics (Nasdaq: CAMP) has initiated GLP toxicology studies for CMP-SYNGAP-01, their lead product candidate targeting SYNGAP1-related disorders. The company aims to submit a clinical trial application that could lead to a Phase 1/2 clinical trial beginning in H2 2026.
Preclinical studies have shown promising results, with CMP-SYNGAP-01 successfully restoring SYNGAP1 protein levels in mouse models and increasing protein levels in relevant brain regions of non-human primates. SYNGAP1-related disorders, affecting 0.5% to 1.0% of intellectual disability cases, currently lack FDA-approved disease-modifying treatments.
CAMP4 Therapeutics (Nasdaq: CAMP) ha avviato studi tossicologici GLP per CMP-SYNGAP-01, il loro candidato principale mirato ai disturbi legati a SYNGAP1. L'azienda punta a presentare una domanda di trial clinico che potrebbe portare a uno studio clinico di fase 1/2 che potrebbe iniziare nella seconda metà del 2026.
Gli studi preclinici hanno mostrato risultati incoraggianti, con CMP-SYNGAP-01 che ha ripristinato con successo i livelli di proteina SYNGAP1 in modelli murini e aumentando i livelli proteici nelle regioni cerebrali rilevanti dei primati non umani. I disturbi legati a SYNGAP1, che interessano lo 0,5% - 1,0% dei casi di disabilità intellettiva, attualmente non dispongono di trattamenti modificanti la malattia approvati dalla FDA.
CAMP4 Therapeutics (Nasdaq: CAMP) ha iniciado estudios GLP de toxicología para CMP-SYNGAP-01, su candidato principal dirigido a trastornos relacionados con SYNGAP1. La empresa tiene como objetivo presentar una solicitud de ensayo clínico que podría conducir a un ensayo clínico de fase 1/2 que podría comenzar en la segunda mitad de 2026.
Los estudios preclínicos han mostrado resultados prometedores, con que CMP-SYNGAP-01 ha restaurado con éxito los niveles de proteína SYNGAP1 en modelos de ratón y aumentado los niveles de proteína en regiones cerebrales relevantes de primates no humanos. Los trastornos relacionados con SYNGAP1, que afligen entre el 0,5% y 1,0% de los casos de discapacidad intelectual, actualmente no cuentan con tratamientos modificadores de la enfermedad aprobados por la FDA.
CAMP4 Therapeutics(Nasdaq: CAMP)은 SYNGAP1 관련 질환을 표적으로 하는 리드 후보 CMP-SYNGAP-01에 대한 GLP 독성학 연구를 시작했습니다. 회사는 임상시험 신청서를 제출하여 1/2상 임상시험으로 이어질 수 있으며, 이는 2026년 하반기에 시작될 수 있습니다.
전임상 연구는 고무적인 결과를 보여 주었으며, CMP-SYNGAP-01은 쥐 모델에서 SYNGAP1 단백질 수치를 성공적으로 회복하고 비인간 영장류의 관련 뇌 영역에서 단백질 수치를 증가시켰습니다. SYNGAP1 관련 질환은 지적장애 사례의 약 0.5%에서 1.0%에 영향을 미치며, 현재 FDA 승인 질병 수정 치료제가 없습니다.
CAMP4 Therapeutics (Nasdaq : CAMP) a lancé des études toxicologiques GLP pour CMP-SYNGAP-01, leur candidat principal ciblant les troubles liés à SYNGAP1. L’entreprise vise à déposer une demande d’essai clinique qui pourrait conduire à un essai clinique de phase 1/2 débutant au cours de la deuxième moitié de 2026.
Les études précliniques ont montré des résultats prometteurs, CMP-SYNGAP-01 ayant réussi à restaurer les niveaux de protéine SYNGAP1 chez des modèles murins et à augmenter les niveaux de protéines dans des régions cérébrales pertinentes chez des primates non humains. Les troubles liés à SYNGAP1, touchant entre 0,5% et 1,0% des cas de handicap intellectuel, n’ont actuellement pas de traitements modificateurs de la maladie approuvés par la FDA.
CAMP4 Therapeutics (Nasdaq: CAMP) hat GLP-Tox-Studien für CMP-SYNGAP-01, ihren führenden Produktkandidaten, der auf SYNGAP1-bezogene Störungen abzielt, eingeleitet. Das Unternehmen beabsichtigt, einen Antrag auf eine klinische Prüfung einzureichen, der zu einer Phase-1/2-Studie führen könnte, die in der zweiten Hälfte 2026 beginnt.
Vorgängliche Studien haben vielversprechende Ergebnisse gezeigt, da CMP-SYNGAP-01 erfolgreich SYNGAP1-Protein in Mausmodellen wiederherstellte und die Proteinspiegel in relevanten Hirnregionen von Nicht-Meforigen erhöht. SYNGAP1-bezogene Störungen betreffen zwischen 0,5% und 1,0% der Fälle intellektueller Behinderung und es gibt derzeit keine FDA-anerkannten krankheitsmodifizierenden Behandlungen.
CMC4 Therapeutics (Nasdaq: CAMP) بدأت دراسات سمية GLP لـ CMP-SYNGAP-01، مرشحها الرائد المستهدف للاضطرابات المرتبطة بـ SYNGAP1. تهدف الشركة إلى تقديم طلب تجربة سريرية قد يقود إلى تجربة سريرية من المرحلة 1/2 تبدأ في النصف الثاني من 2026.
أظهرت الدراسات قبل السريرية نتائج واعدة، حيث نجح CMP-SYNGAP-01 في استعادة مستويات بروتين SYNGAP1 في نماذج فأرية ورفع مستويات البروتين في المناطق الدماغية ذات الصلة لدى الرئيسيات غير البشرية. الاضطرابات المرتبطة بـ SYNGAP1، التي تؤثر على 0.5% إلى 1.0% من حالات الإعاقة الذهنية، لا يوجد لها حتى الآن علاجات مغيرة للمرض معتمدة من FDA.
CAMP4 Therapeutics（纳斯达克代码：CAMP） 已为 CMP-SYNGAP-01 开始 GLP 毒理学研究，他们的领先候选产品，靶向 SYNGAP1 相关疾病。公司目标提交一份临床试验申请，可能使在 2026 年下半年 启动的 Ⅰ/Ⅱ 期临床试验 成为现实。
前临床研究显示出有希望的结果，CMP-SYNGAP-01 已在小鼠模型中成功恢复 SYNGAP1 蛋白水平，并在非人灵长类动物的大脑相关区域中提高了蛋白水平。SYNGAP1 相关疾病影响约 0.5% 到 1.0% 的智力障碍病例，目前尚无 FDA 批准的可改变疾病进程的治疗方法。
- Strong preclinical results showing protein level restoration in mouse models
- Successful protein increase demonstrated in non-human primate brain regions
- Targeting a significant market with 0.5-1.0% of intellectual disability cases
- Advancing towards first-in-human clinical trials with clear timeline
- Clinical trials won't begin until second half of 2026
- No current FDA-approved treatments, indicating potential regulatory challenges
- Complex development pathway for neurodevelopmental disorders
Insights
CAMP4's advance to GLP toxicology testing represents meaningful pipeline progress toward clinical trials in an underserved neurological condition.
CAMP4 Therapeutics has achieved a significant regulatory milestone with the initiation of GLP toxicology studies for CMP-SYNGAP-01, their lead candidate targeting SYNGAP1-related disorders. This development represents a critical step in the preclinical pathway, positioning the company for a potential clinical trial application that could enable first-in-human testing by H2 2026.
The preclinical data package appears compelling, with evidence of target engagement and efficacy in both mouse models and non-human primates - specifically demonstrating SYNGAP1 protein restoration in brain regions relevant to human disease. This cross-species translation strengthens the scientific rationale for advancing to human studies.
SYNGAP1-related disorders represent a significant unmet medical need within the neurodevelopmental disease space. As a condition potentially responsible for 0.5-1.0% of intellectual disability cases, particularly those with comorbid epilepsy, the addressable patient population may be substantially larger than current estimates suggest. The haploinsufficiency mechanism (reduced protein levels) aligns perfectly with CAMP4's regulatory RNA platform designed to upregulate gene expression.
The current treatment landscape consists solely of symptom management through supportive therapies and non-specific anti-seizure medications, with no approved disease-modifying options. This creates a substantial market opportunity for CAMP4 if they can demonstrate clinical efficacy through their protein restoration approach.
While this milestone is promising, investors should note that clinical trials remain at least a year away, with substantial development and regulatory hurdles still ahead. The complex nature of neurological disorders and the challenges of delivering therapeutics across the blood-brain barrier will be important factors to monitor as this program progresses toward the clinic.
CAMBRIDGE, Mass., Oct. 01, 2025 (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 initiation of toxicology studies conducted under Good Laboratory Practice (GLP) standards for its lead product candidate, CMP-SYNGAP-01. These studies will support the Company’s planned submission of a clinical trial application, which could enable the initiation of a first-in-human Phase 1/2 clinical trial in SYNGAP1-related disorders as early as the second half of 2026.
“CMP-SYNGAP-01 has demonstrated robust preclinical activity, both restoring SYNGAP1 protein levels to improve phenotypes in a mouse model and increasing SYNGAP1 protein in brain regions of non-human primates that are affected in human disease,” said Daniel Tardiff, Ph.D., Chief Scientific Officer of CAMP4. “Our preclinical data gives us confidence in the potential of CMP-SYNGAP-01 to translate into meaningful clinical benefit for patients living with SYNGAP1-related disorders, and advancing our candidate through GLP toxicology studies marks a critical step towards the clinic.”
SYNGAP1-related disorders are a group of neurodevelopmental conditions caused by pathogenic variants in the SYNGAP1 gene that result in a haploinsufficient state, reducing SYNGAP protein levels by up to
Today, there are no FDA-approved, disease-modifying therapies for SYNGAP1-related disorders, with treatment often limited to supportive physical, occupational, and speech therapy. A combination of non-specific anti-seizure drugs and other medications may be prescribed, though SYNGAP1-related disorders have proven difficult to control with available therapeutics.
About CMP-SYNGAP-01
CMP-SYNGAP-01 is an investigational, novel approach that targets the SYNGAP1 gene at the transcriptional level to restore SYNGAP function and improve symptoms by utilizing an intrathecally delivered antisense oligonucleotide. Upregulation of SYNGAP1 gene expression may increase SYNGAP protein levels in amounts sufficient to yield therapeutic benefit. Preclinical studies have demonstrated a dose-dependent increase in SYNGAP mRNA levels accompanied by an increase in SYNGAP protein expression.
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.
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 potential for GLP toxicology studies to generate regulatory-compliant data to support a clinical trial in SYNGAP1-related disorders; expectations regarding the timing to advance the Company’s SYNGAP1 program into a Phase 1/2 clinical trial; and the therapeutic potential of CMP-SYNGAP-01; and the incidence and prevalence of SYNGAP1-related disorders. 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 Company’s limited operating history, incurrence of substantial losses since the Company’s inception and anticipation of incurring substantial and increasing losses for the foreseeable future; the Company’s need for substantial additional financing to achieve the Company’s goals; the uncertainty of 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; delays or difficulties in the enrollment and dosing of patients in clinical trials; the impact of any significant adverse events or undesirable side effects caused by the Company’s product candidates; potential competition, including from large and specialty pharmaceutical and biotechnology companies; the Company’s ability to realize the benefits of the Company’s current or future collaborations or licensing arrangements and ability to successfully consummate future partnerships; the Company’s ability to obtain regulatory approval to commercialize any product candidate in the United States or any other jurisdiction, and the risk that any such approval may be for a more narrow indication than the Company seeks; 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; the Company’s ability to grow the Company’s organization, and manage the Company’s growth and expansion of the Company’s operations; 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 product candidates or any future product candidates the Company may develop; the Company’s reliance on third parties to conduct the Company’s preclinical studies and clinical trials; the Company’s compliance with the Company’s obligations under the licenses granted to the Company by others, for the rights to develop and commercialize the Company’s product candidates; risks related to the operations of the Company’s suppliers; 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, 2024 and Quarterly Report on Form 10-Q for the quarter ended June 30, 2025, 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:
Jason Braco, Ph.D.
LifeSci Communications
jbraco@lifescicomms.com
FAQ
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