CAMP4 Provides Corporate Updates and Highlights Key Upcoming Milestones
CAMP4 Therapeutics provided corporate updates and key objectives for 2025, highlighting significant progress in their clinical programs. The company completed its IPO, raising gross proceeds of $82.1 million. Their lead program, CMP-CPS-001 for urea cycle disorders (UCDs), demonstrated favorable safety results in the Single Ascending Dose (SAD) portion of Phase 1 study, with all treatment emergent adverse events being mild or moderate.
The company has completed dosing in the first two Multiple Ascending Dose (MAD) cohorts and initiated Cohort 3. The FDA granted both Rare Pediatric Disease Designation and Orphan Drug Designation to CMP-CPS-001. CAMP4 also entered a strategic research collaboration with BioMarin and was added to the Russell 2000® Index. Key upcoming milestones include MAD safety and biomarker data expected in 2H 2025, and the initiation of a new discovery program targeting GBA1 regRNA for Parkinson's disease treatment.
CAMP4 Therapeutics ha fornito aggiornamenti aziendali e obiettivi chiave per il 2025, evidenziando progressi significativi nei loro programmi clinici. L'azienda ha completato la sua IPO, raccogliendo proventi lordi di 82,1 milioni di dollari. Il loro programma principale, CMP-CPS-001 per i disturbi del ciclo dell'urea (UCD), ha mostrato risultati di sicurezza favorevoli nella parte di Dose Ascendente Singola (SAD) dello studio di Fase 1, con tutti gli eventi avversi emergenti dal trattamento che sono stati lievi o moderati.
L'azienda ha completato la somministrazione nelle prime due coorti di Dose Ascendente Multipla (MAD) ed ha iniziato la Coorte 3. La FDA ha concesso sia la Designazione di Malattia Pediatrica Rara che la Designazione di Farmaco Orfano a CMP-CPS-001. CAMP4 ha anche avviato una collaborazione di ricerca strategica con BioMarin ed è stata inserita nell'indice Russell 2000®. I principali traguardi imminenti includono dati di sicurezza e biomarker MAD attesi nel secondo semestre del 2025, e l'avvio di un nuovo programma di scoperta mirato al GBA1 regRNA per il trattamento della malattia di Parkinson.
CAMP4 Therapeutics proporcionó actualizaciones corporativas y objetivos clave para 2025, resaltando avances significativos en sus programas clínicos. La empresa completó su IPO, recaudando ingresos brutos de 82,1 millones de dólares. Su programa principal, CMP-CPS-001 para trastornos del ciclo de la urea (UCD), demostró resultados de seguridad favorables en la parte de Dosis Ascendente Única (SAD) del estudio de Fase 1, con todos los eventos adversos emergentes del tratamiento siendo leves o moderados.
La compañía ha completado la dosificación en las primeras dos cohortes de Dosis Ascendente Múltiple (MAD) e inició la Cohorte 3. La FDA otorgó tanto la Designación de Enfermedad Pediátrica Rara como la Designación de Medicamento Huérfano a CMP-CPS-001. CAMP4 también entró en una colaboración de investigación estratégica con BioMarin y fue añadida al índice Russell 2000®. Los hitos clave que se esperan incluyen datos de seguridad y biomarcadores de MAD esperados en la segunda mitad de 2025, y el inicio de un nuevo programa de descubrimiento dirigido a GBA1 regRNA para el tratamiento de la enfermedad de Parkinson.
CAMP4 Therapeutics는 2025년을 위한 기업 업데이트와 주요 목표를 제공하며 임상 프로그램에서의 중요한 진전을 강조했습니다. 이 회사는 IPO를 완료하고 8210만 달러의 총 수익을 올렸습니다. 그들의 주요 프로그램인 CMP-CPS-001는 요소 주기 장애(UCD)에 대한 것으로, 1상 연구의 단일 상승 용량(SAD) 부분에서 안전성 결과가 유리하다는 것을 보여주었으며, 모든 치료 유발 부작용은 경미하거나 중간 정도였습니다.
회사는 첫 두 개의 다중 상승 용량(MAD) 코호트에서의 투약을 완료하고 3코호트를 시작했습니다. FDA는 CMP-CPS-001에 대해 희귀 소아 질환 지정 및 고아 약 지정 모두를 부여했습니다. CAMP4는 또한 BioMarin과 전략적 연구 협력을 시작했으며 Russell 2000® 지수에 추가되었습니다. 다가오는 주요 이정표로는 2025년 하반기에 예상되는 MAD 안전성 및 바이오마커 데이터와 파킨슨병 치료를 위한 GBA1 regRNA를 목표로 하는 새로운 발견 프로그램의 시작이 포함됩니다.
CAMP4 Therapeutics a fourni des mises à jour d'entreprise et des objectifs clés pour 2025, soulignant des progrès significatifs dans leurs programmes cliniques. L'entreprise a terminé son introduction en bourse, levant des fonds bruts de 82,1 millions de dollars. Leur programme phare, CMP-CPS-001 pour les troubles du cycle de l'urée (UCD), a montré des résultats de sécurité favorables dans la partie de dose unique ascendante (SAD) de l'étude de phase 1, tous les événements indésirables liés au traitement étant légers ou modérés.
L'entreprise a terminé la posologie dans les deux premières cohortes de dose multiple ascendante (MAD) et a initié la Cohorte 3. La FDA a accordé à CMP-CPS-001 une désignation de maladie pédiatrique rare et une désignation de médicament orphelin. CAMP4 a également engagé une collaboration de recherche stratégique avec BioMarin et a été inclus dans l'indice Russell 2000®. Les principales étapes à venir incluent des données de sécurité et des biomarqueurs MAD qui devraient être disponibles au deuxième semestre 2025, ainsi que le lancement d'un nouveau programme de découverte ciblant le GBA1 regRNA pour le traitement de la maladie de Parkinson.
CAMP4 Therapeutics hat Unternehmensupdates und wichtige Ziele für 2025 bereitgestellt und dabei erhebliche Fortschritte in seinen klinischen Programmen hervorgehoben. Das Unternehmen hat seine IPO abgeschlossen und bruttowert von 82,1 Millionen Dollar eingeworben. Ihr Hauptprogramm, CMP-CPS-001 für Harnstoffzyklusstörungen (UCDs), zeigte in der Phase-1-Studie während des Abschnitts mit der Einzelaufdosierung (SAD) positive Sicherheitsresultate, wobei alle behandlungsbedingten unerwünschten Ereignisse mild oder moderat waren.
Das Unternehmen hat die Dosierung in den ersten beiden Kohorten der mehrfachen Aufdosierung (MAD) abgeschlossen und die Kohorte 3 initiiert. Die FDA hat CMP-CPS-001 sowohl die Auszeichnung als seltene pädiatrische Erkrankung als auch die Auszeichnung als Arzneimittel für Waisen erteilt. CAMP4 trat außerdem eine strategische Forschungszusammenarbeit mit BioMarin ein und wurde in den Russell 2000®-Index aufgenommen. Wichtige bevorstehende Meilensteine umfassen die in der zweiten Hälfte von 2025 erwarteten Sicherheitsdaten und Biomarker der MAD sowie den Beginn eines neuen Entdeckungsprogramms, das auf GBA1 regRNA für die Behandlung der Parkinson-Krankheit abzielt.
- Successful IPO raising $82.1 million in gross proceeds
- Favorable safety results in Phase 1 SAD study of CMP-CPS-001
- FDA granted Rare Pediatric Disease and Orphan Drug Designations for CMP-CPS-001
- Strategic research collaboration secured with BioMarin
- Addition to Russell 2000® Index
- None.
Insights
The Phase 1 clinical trial results for CMP-CPS-001 demonstrate a promising safety profile in treating urea cycle disorders (UCDs). The completion of SAD cohorts with only Grade 1-2 adverse events aligns with expectations for liver-targeted ASOs. The progression to MAD Cohort 3 following Safety Review Committee approval indicates strong clinical momentum.
The <money>82.1 million</money> IPO proceeds provide runway for pipeline advancement. The dual FDA designations (Rare Pediatric Disease and Orphan Drug) enhance the program's regulatory pathway and market potential. The BioMarin collaboration validates CAMP4's regRNA platform technology.
Key catalysts include MAD data readout in H2 2025 and potential Phase 2/3 initiation in 2026. The expansion into Parkinson's disease through GBA1 targeting represents significant market opportunity beyond rare diseases.
The successful IPO raising <money>82.1 million</money> strengthens CAMP4's balance sheet and provides operational runway. Inclusion in the Russell 2000® Index increases institutional visibility and potential trading liquidity. The BioMarin partnership offers both validation and future revenue potential through milestone payments and royalties.
With a market cap of <money>102 million</money>, the company appears reasonably valued considering its clinical-stage assets and platform technology. The multiple shots on goal strategy across UCDs, neurodevelopmental disorders and Parkinson's disease diversifies risk while maintaining focus on rare diseases with high unmet needs.
The safety profile from the SAD portion is encouraging - headache and nausea as primary adverse events are manageable and typical for ASO therapeutics. The strategic expansion into GBA1-related Parkinson's disease leverages the platform's ability to upregulate gene expression, particularly relevant for loss-of-function mutations.
The completion of two MAD cohorts and progression to the third demonstrates methodical clinical development. The upcoming H2 2025 biomarker data will be important in validating the mechanism of action and determining optimal dosing for the pivotal trial. The addition of industry veterans John Maraganore and Rachel Meyers as advisors brings valuable development expertise.
- Data from Single Ascending Dose (SAD) portion of Phase 1 study of CMP-CPS-001 for the treatment of urea cycle disorders (UCDs) demonstrates favorable safety results
- Completed dosing in the first two Multiple Ascending Dose (MAD) cohorts, and initiated dosing in Cohort 3
- MAD safety and key study biomarker data expected in 2H 2025
- CAMP4 added to Russell 2000® Index
- Initiation of new discovery program targeting a GBA1 regRNA for the treatment of Parkinson’s disease (PD)
- Company to present at the 43rd Annual J.P. Morgan Healthcare Conference on Wednesday, January 15 at 3:45 p.m. PST
CAMBRIDGE, Mass., Jan. 07, 2025 (GLOBE NEWSWIRE) -- CAMP4 Therapeutics Corporation (“CAMP4”) (Nasdaq: CAMP), a clinical-stage biotechnology company developing regRNA-targeting antisense oligonucleotide (ASO) therapies to upregulate gene expression to restore healthy protein levels, today provided corporate updates and key objectives for 2025.
“2024 was a transformative year for CAMP4, highlighted by our successful IPO, and we are entering 2025 with tremendous momentum and a clear path towards delivering on our goals,” said Josh Mandel-Brehm, Chief Executive Officer of CAMP4. “We are very pleased with the clinical progress of our lead program CMP-CPS-001 for urea cycle disorders, which was granted Rare Pediatric Disease Designation and Orphan Drug Designation, validating the urgency of our novel regRNA-targeting ASOs. Additionally, our strategic partnership with BioMarin highlights confidence in our RAP platform. These achievements, combined with the proceeds from the IPO, position us to continue to progress our pipeline development and execute on key milestones this year.”
“We are pleased to report the safety data from the SAD portion of our Phase 1 study of CMP-CPS-001 for UCDs, showing that the drug has been well-tolerated,” said Dr. Yuri Maricich, Chief Medical Officer of CAMP4. “We expect 2025 to be a pivotal year as we anticipate reporting MAD safety and key biomarker data in the second half of 2025. Results from these studies could enable us to advance the CMP-CPS-001 program into a registrational Phase 2/3 trial in 2026.”
2024 Key Highlights
- Completed IPO of 6,820,000 shares of common stock at an initial public offering price of
$11.00 per share. Aggregate gross proceeds to CAMP4 were approximately$75.0 million . The underwriters also partially exercised their option to purchase an additional 643,762 shares of common stock for total offering gross proceeds of$82.1 million . - Advanced Phase 1 clinical trial of CMP-CPS-001 for UCDs. The Phase 1 study is a randomized, double-blind, and placebo-controlled study designed to evaluate the safety, tolerability, and pharmacokinetics of CMP-CPS-001 in 96 healthy volunteers.
- Completed planned interim analysis of all four SAD cohorts of the Phase 1 clinical trial of CMP-CPS-001 in 48 healthy volunteer participants. Safety results were favorable and consistent with the safety profile of approved liver-targeted ASOs, with all treatment emergent adverse events (TEAEs) being Grade 1 (mild) or Grade 2 (moderate). The two most common TEAEs across all cohorts were headache (six participants) and nausea (four participants). No safety trends of concern have been observed, and CMP-CPS-001 appears to be well-tolerated. Dosing is completed in the first two MAD cohorts, and the Safety Review Committee (SRC) has approved dose escalation to MAD Cohort 3, in which dosing has been initiated.
- The FDA granted Rare Pediatric Disease Designation and Orphan Drug Designation to CMP-CPS-001 for the treatment of UCDs.
- Entered strategic research collaboration agreement with BioMarin Pharmaceutical Inc., under which BioMarin has the right to select two targets identified by CAMP4’s RAP Platform to advance into clinical development.
- Company added to the Russell 2000® Index as part of the fourth quarter IPO additions.
- Appointed John Maraganore, Ph.D., and Rachel Meyers, Ph.D., as strategic advisors.
Expected Milestones in 2025
- MAD safety, pharmacokinetic and key pharmacodynamic biomarker data in healthy volunteers in the second half of 2025.
- GLP toxicity studies of lead ASO candidate for neurodevelopmental disorders caused by SYNGAP1 mutations to be initiated this year.
- Advance a new discovery program targeting a GBA1 regRNA to increase gene expression for the treatment of Parkinson’s disease (PD) caused by mutations in GBA1, with potential for application in sporadic PD.
- Company to focus on expanding its strategic partnerships to continue maximizing the value of its RAP platform.
About CAMP4 Therapeutics
CAMP4 is developing disease-modifying treatments for a broad range of rare and prevalent genetic diseases where increasing healthy protein levels may offer meaningful therapeutic benefits. Our approach allows for targeted gene upregulation by harnessing a fundamental mechanism of how genes are controlled. To increase gene expression, our therapeutic ASO drug candidates target 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. Learn more about us at www.CAMP4tx.com and follow us on LinkedIn and X.
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, objectives, expectations and intentions; the timing and results of ongoing and future clinical trials, including expectations on the timing of reporting MAD data from the CMP-CPS-001 trial and advancing the CMP-CPS-001 program into a registration-enabling trial; the timing to initiate GLP toxicity studies relating to CAMP4’s SYNGAP1 program; the timing to advance new discovery programs; its growth strategy; and cash runway guidance. 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 Quarterly Report on Form 10-Q for the quarter ended September 30, 2024, as well as other information we file 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:
Sandya von der Weid
LifeSci Advisors svonderweid@lifesciadvisors.com
Media:
Jason Braco, Ph.D.
LifeSci Communications
jbraco@lifescicomms.com
FAQ
What were the Phase 1 SAD study results for CAMP's CMP-CPS-001 in UCD treatment?
How much did CAMP4 raise in its 2024 IPO?
When will CAMP4 release MAD safety and biomarker data for CMP-CPS-001?
What new therapeutic area is CAMP4 expanding into for 2025?
What regulatory designations has CAMP received for CMP-CPS-001?
