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CAMP4 Presents Translational Data from SYNGAP1-Related Disorders Program Showcasing Increased Protein in Non-Human Primates and Reviews Preclinical and Detailed Single Ascending Dose Safety Data from Urea Cycle Disorders Program at the 28th American Society of Gene and Cell Therapy Annual Meeting

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CAMP4 Therapeutics (NASDAQ: CAMP) presented promising data from two key programs at the 28th ASGCT Annual Meeting. For their SYNGAP1-related disorders program, CMP-SYNGAP-01 demonstrated increased SYNGAP1 protein levels in both haploinsufficient mice and non-human primates, with successful rescue of behavioral phenotypes. In mice, the treatment restored protein levels to near normal range and improved motor and spatial learning defects. In NHPs, biweekly intrathecal injections achieved a ~1.5-fold protein increase across relevant brain regions. For their Urea Cycle Disorders (UCDs) program, Phase 1 clinical trial data of CMP-CPS-001 showed favorable safety profiles in healthy volunteers. The trial enrolled 48 participants across four SAD cohorts, with no serious adverse events reported. Preclinical data showed promising results in both mice and NHPs, with up to 40% increase in ureagenesis in NHPs and dose-dependent reductions in ammonia levels in mice.
CAMP4 Therapeutics (NASDAQ: CAMP) ha presentato dati promettenti da due programmi chiave al 28° Meeting Annuale ASGCT. Per il loro programma sui disturbi correlati a SYNGAP1, CMP-SYNGAP-01 ha mostrato un aumento dei livelli di proteina SYNGAP1 sia nei topi con insufficienza genica che nelle scimmie non umane, con un recupero efficace dei fenotipi comportamentali. Nei topi, il trattamento ha riportato i livelli proteici quasi alla normalità e ha migliorato i difetti motori e di apprendimento spaziale. Nelle scimmie non umane, le iniezioni intratecali bisettimanali hanno ottenuto un aumento di circa 1,5 volte della proteina nelle aree cerebrali rilevanti. Per il loro programma sui disturbi del ciclo dell'urea (UCD), i dati della fase 1 della sperimentazione clinica di CMP-CPS-001 hanno mostrato un profilo di sicurezza favorevole in volontari sani. Lo studio ha arruolato 48 partecipanti in quattro coorti SAD, senza eventi avversi gravi segnalati. I dati preclinici hanno mostrato risultati promettenti sia nei topi che nelle scimmie non umane, con un aumento fino al 40% della ureagenesi nelle scimmie e riduzioni dose-dipendenti dei livelli di ammoniaca nei topi.
CAMP4 Therapeutics (NASDAQ: CAMP) presentó datos prometedores de dos programas clave en la 28ª Reunión Anual de ASGCT. Para su programa relacionado con trastornos de SYNGAP1, CMP-SYNGAP-01 demostró un aumento en los niveles de proteína SYNGAP1 tanto en ratones haploinsuficientes como en primates no humanos, con una recuperación exitosa de los fenotipos conductuales. En ratones, el tratamiento restauró los niveles de proteína a un rango casi normal y mejoró los defectos motores y de aprendizaje espacial. En primates no humanos, las inyecciones intratecales quincenales lograron un aumento de aproximadamente 1,5 veces en las regiones cerebrales relevantes. Para su programa de trastornos del ciclo de la urea (UCD), los datos del ensayo clínico de fase 1 de CMP-CPS-001 mostraron perfiles de seguridad favorables en voluntarios sanos. El ensayo incluyó a 48 participantes en cuatro cohortes SAD, sin eventos adversos graves reportados. Los datos preclínicos mostraron resultados prometedores tanto en ratones como en primates no humanos, con hasta un 40% de aumento en la ureogénesis en primates y reducciones dependientes de la dosis en los niveles de amoníaco en ratones.
CAMP4 Therapeutics(NASDAQ: CAMP)는 제28회 ASGCT 연례회의에서 두 가지 주요 프로그램의 유망한 데이터를 발표했습니다. SYNGAP1 관련 질환 프로그램에서 CMP-SYNGAP-01은 유전자 결핍 생쥐와 비인간 영장류 모두에서 SYNGAP1 단백질 수치를 증가시켰으며, 행동 표현형의 성공적인 회복을 보였습니다. 생쥐에서는 치료가 단백질 수치를 거의 정상 범위로 회복시키고 운동 및 공간 학습 결함을 개선했습니다. 비인간 영장류에서는 격주로 시행한 척수강 내 주사가 관련 뇌 영역에서 약 1.5배 단백질 증가를 달성했습니다. 요소회로질환(UCD) 프로그램에서는 CMP-CPS-001의 1상 임상시험 데이터가 건강한 지원자에서 우수한 안전성 프로파일을 보여주었습니다. 시험에는 4개의 SAD 코호트에 걸쳐 48명의 참가자가 등록되었으며, 심각한 이상 반응은 보고되지 않았습니다. 전임상 데이터는 생쥐와 비인간 영장류 모두에서 유망한 결과를 나타냈으며, 비인간 영장류에서는 요소 생성이 최대 40% 증가했고, 생쥐에서는 용량 의존적으로 암모니아 수치가 감소했습니다.
CAMP4 Therapeutics (NASDAQ : CAMP) a présenté des données prometteuses issues de deux programmes clés lors de la 28e réunion annuelle de l'ASGCT. Pour leur programme sur les troubles liés à SYNGAP1, CMP-SYNGAP-01 a démontré une augmentation des niveaux de protéine SYNGAP1 chez des souris haploinsuffisantes ainsi que chez des primates non humains, avec une restauration réussie des phénotypes comportementaux. Chez les souris, le traitement a ramené les niveaux de protéine à une plage proche de la normale et a amélioré les défauts moteurs et d'apprentissage spatial. Chez les primates non humains, des injections intrathécales bihebdomadaires ont permis une augmentation d'environ 1,5 fois de la protéine dans les régions cérébrales concernées. Pour leur programme sur les troubles du cycle de l'urée (UCD), les données de l'essai clinique de phase 1 de CMP-CPS-001 ont montré un profil de sécurité favorable chez des volontaires sains. L'essai a inclus 48 participants répartis en quatre cohortes SAD, sans événements indésirables graves rapportés. Les données précliniques ont montré des résultats prometteurs chez les souris et les primates non humains, avec une augmentation allant jusqu'à 40 % de l'uréagenèse chez les primates et des réductions dépendantes de la dose des niveaux d'ammoniaque chez les souris.
CAMP4 Therapeutics (NASDAQ: CAMP) präsentierte vielversprechende Daten aus zwei wichtigen Programmen auf dem 28. ASGCT-Jahrestreffen. Für ihr SYNGAP1-bezogenes Störungsprogramm zeigte CMP-SYNGAP-01 erhöhte SYNGAP1-Proteine bei haploinsuffizienten Mäusen und nicht-menschlichen Primaten, mit erfolgreicher Wiederherstellung der Verhaltensphänotypen. Bei Mäusen stellte die Behandlung die Proteinspiegel nahezu auf Normalniveau wieder her und verbesserte motorische und räumliche Lernstörungen. Bei nicht-menschlichen Primaten führten zweiwöchentliche intrathekale Injektionen zu einer etwa 1,5-fachen Erhöhung des Proteins in relevanten Hirnregionen. Für ihr Programm zu Harnstoffzyklus-Störungen (UCDs) zeigten Daten der Phase-1-Studie von CMP-CPS-001 ein günstiges Sicherheitsprofil bei gesunden Freiwilligen. Die Studie umfasste 48 Teilnehmer in vier SAD-Kohorten, ohne schwerwiegende unerwünschte Ereignisse. Präklinische Daten zeigten vielversprechende Ergebnisse sowohl bei Mäusen als auch bei nicht-menschlichen Primaten, mit bis zu 40 % Steigerung der Ureagenese bei Primaten und dosisabhängigen Reduktionen der Ammoniakspiegel bei Mäusen.
Positive
  • CMP-SYNGAP-01 successfully restored SYNGAP1 protein levels to near normal range in mice
  • CMP-SYNGAP-01 achieved ~1.5-fold protein increase in NHP brain regions
  • Phase 1 trial of CMP-CPS-001 demonstrated favorable safety profile with no serious adverse events
  • Preclinical data showed up to 40% increase in ureagenesis in NHPs
  • Both drug candidates were well-tolerated in studies
Negative
  • None.

Insights

CAMP4's dual pipeline progress shows promising data for SYNGAP1 and UCD programs with positive safety profiles and mechanism validation.

CAMP4 Therapeutics has presented compelling translational data for two key pipeline programs targeting genetic diseases with significant unmet needs. The company's regulatory RNA-targeting platform (RAP) shows promising progress in both neurological and metabolic disorders.

For the SYNGAP1-related disorders program, their candidate CMP-SYNGAP-01 demonstrated meaningful protein restoration in mouse models, rescuing key behavioral phenotypes. More importantly, the candidate showed approximately 150% increase in SYNGAP1 protein levels across multiple disease-relevant brain regions in non-human primates when administered intrathecally - a critical translational step towards human studies. This is particularly significant as SYNGAP1-related disorders currently have no approved disease-modifying treatments.

For their more advanced Urea Cycle Disorders (UCD) program, CAMP4 presented both preclinical data and Phase 1 clinical results. Their candidate CMP-CPS-001 showed dose-dependent ammonia reductions in mouse models and increased ureagenesis in non-human primates. The Phase 1 trial demonstrated a favorable safety profile across four single ascending dose cohorts with no serious adverse events. The pharmacokinetic data showed dose-dependent increases in exposure with greater than dose-proportional increases - a positive sign for potential efficacy at manageable dose levels.

The company has already completed dosing in multiple ascending dose cohorts and plans to expand testing into OTC (Ornithine Transcarbamylase) heterozygotes - patients with reduced urea cycle function. This represents a critical step toward evaluating therapeutic potential in the target patient population.

These results validate CAMP4's scientific approach of upregulating gene expression through targeting regulatory RNA to address haploinsufficiency disorders - a novel therapeutic modality with broad potential application across multiple genetic diseases with limited treatment options.

Haploinsufficient SYNGAP1 mice treated with CMP-SYNGAP-01 demonstrated an increase in SYNGAP1 protein levels; treatment rescued multiple SYNGAP1-dependent behavioral phenotypes

CMP-SYNGAP-01 administration led to a significant increase in SYNGAP1 protein levels in relevant brain regions in non-human primates (NHPs)

Patient safety and pharmacokinetic data from single ascending dose (SAD) cohorts of the first-in-human Phase 1 clinical trial of CMP-CPS-001 in healthy volunteers highlighted

CAMBRIDGE, Mass., May 16, 2025 (GLOBE NEWSWIRE) -- CAMP4 Therapeutics Corporation (“CAMP4”) (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 delivered three oral presentations on its SYNGAP1-related disorders and Urea Cycle Disorders (UCDs) programs and shared favorable safety and pharmacokinetics data from the ongoing Phase 1 trial of CMP-CPS-001 in healthy volunteers at the 28th Annual Meeting of the American Society of Gene and Cell Therapy, taking place in New Orleans, May 13 – 17, 2025.

“Patients living with SYNGAP1-related disorders and UCDs currently face a critical dearth of disease-modifying treatment options to manage their condition,” said Dan Tardiff, Ph.D., Senior Vice President, Head of Discovery at CAMP4. “These proof-of-mechanism data indicate CMP-SYNGAP-01 can restore SYNGAP1 protein levels to mitigate disease-relevant phenotypes in haploinsufficient mice and increase SYNGAP1 protein in disease-relevant brain regions in non-human primates when delivered by the clinical route of administration. Additionally, our UCD clinical candidate is well tolerated, and we are preparing to evaluate CMP-CPS-001 in OTC heterozygotes, a population with reduced urea cycle function. We’re excited to continue pioneering our novel approach of upregulating gene expression and addressing the unmet needs of many patients living with genetic diseases characterized by haploinsufficiency or recessive loss of function.”

Josh Mandel-Brehm, Chief Executive Officer of CAMP4, added, “These compelling data underscore the expansive potential of our RAP Platform to address a wide spectrum of genetic conditions, starting with neurologic and metabolic disorders. By pairing clinically validated antisense technologies with newly discovered regulatory RNA targets to upregulate gene expression, we have an opportunity to rapidly advance therapeutics for disorders characterized by insufficient protein production including SYNGAP1-related disorders, where there is significant unmet need for disease-modifying therapies. We look forward to progressing toward additional clinical trials and exploring strategic partnerships that can accelerate our development plans and deliver long-term value for patients and shareholders.”

Key findings for each program are as follows:

SYNGAP1-related disorders program

  • In haploinsufficient mice carrying a single copy of the human SYNGAP1 gene, intracerebroventricular (ICV) injection of CMP-SYNGAP-01, a development candidate targeting a regulatory RNA sequence mapped to a SYNGAP1 gene regulatory region, resulted in:
    • Restored SYNGAP1 protein levels to near normal range after a single dose
    • Rescue of motor defects and spatial learning defects following two doses
  • In NHPs, biweekly intrathecal injections of CMP-SYNGAP-01 resulted in a ~1.5-fold increase in SYNGAP1 protein levels across multiple brain regions clinically relevant to the disease
    • Dose-linear increase in CMP-SYNGAP-01 in disease-relevant brain regions
    • CMP-SYNGAP-01 was well tolerated

UCD program

  • Preclinical data
    • In Otc-deficient mice, treatment with CMP-CPS-001 resulted in dose-dependent reductions in ammonia levels, which persisted for approximately 4 weeks
      • Increases in mRNA levels of additional enzymes of the urea cycle were observed, suggesting increased metabolic activity
    • In mice with humanized livers, administration of CMP-CPS-001 following an ammonia challenge resulted in increased ureagenesis and decreased ammonia levels
    • Administration of CMP-CPS-001 in NHPs resulted in up to 40% increase in ureagenesis, supporting the MOA to convert ammonia to urea
  • Phase 1 clinical data
    • 48 healthy adult participants were enrolled across four SAD cohorts and randomized 3:1 to a single subcutaneous dose of CMP-CPS-001, with 36 participants randomized to CMP-CPS-001
    • CMP-CPS-001 was well tolerated, with no evidence of a maximum tolerated dose and no safety trends of concern
    • All treatment-emergent adverse events (TEAEs) were Grade 1 (mild) or Grade 2 (moderate) with no serious or severe adverse events (AEs) or TEAEs and no participants discontinued study drug due to a TEAE
      • Most common TEAEs were headache (n=6) followed by nausea (n=4)
    • Pharmacokinetics
      • Dose-dependent increase in exposure (Cmax and AUC) with clear separation between dose levels
      • Greater than dose-proportional increase in exposure (Cmax and AUC0-24)
  • Study Update
    • Dosing complete in MAD Cohort 1 through Cohort 3
    • Anticipate expansion into OTC heterozygotes to assess safety and CMP-CPS-001 effect on ureagenesis in patients with evidence of reduced urea cycle function

The presentations can be accessed on the CAMP4 website at https://investors.camp4tx.com/news-events/presentations after the presentations.

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.

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 CAMP4’s plans and expectations regarding its ongoing Phase 1 clinical trial of CMP-CPS-001 and its expansion into a Phase 1b clinical trial of CMP-CPS-001; the anticipated timing and results of the company’s ongoing and future clinical trials, including expectations regarding the timing of reporting data from the CMP-CPS-001 clinical trials; the expected timing for the company’s initiation of GLP toxicity studies relating to CAMP4’s SYNGAP1 program; and the therapeutic potential of CAMP4’s 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 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 March 31, 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:

Kelly Gold, CFO
CAMP4 Therapeutics
kgold@camp4tx.com

Media:

Jason Braco, Ph.D.
LifeSci Communications
jbraco@lifescicomms.com


FAQ

What were the key results from CAMP4's SYNGAP1 program presentation at ASGCT 2025?

CMP-SYNGAP-01 restored SYNGAP1 protein levels to near normal range in mice and achieved a ~1.5-fold increase in protein levels across multiple brain regions in non-human primates, while demonstrating good tolerability.

How did CAMP's CMP-CPS-001 perform in the Phase 1 clinical trial for Urea Cycle Disorders?

The Phase 1 trial showed CMP-CPS-001 was well-tolerated across 48 participants, with only mild to moderate adverse events, no serious adverse events, and demonstrated dose-dependent increases in exposure.

What were the most common side effects reported in CAMP4's Phase 1 trial of CMP-CPS-001?

The most common treatment-emergent adverse events were headache (6 cases) and nausea (4 cases), all classified as Grade 1 (mild) or Grade 2 (moderate).

What is CAMP4 Therapeutics' approach to treating genetic diseases?

CAMP4 develops regulatory RNA-targeting therapeutics designed to upregulate gene expression to restore healthy protein levels in genetic diseases, particularly focusing on disorders characterized by haploinsufficiency or recessive loss of function.
Camp4 Therapeutics Corp.

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