CAMP4 Therapeutics Targets 2028 for Key SYNGAP1 Trial Data

Key Points

• CAMP4 expects initial CMP-002 Phase 1/2 data in the first half of 2028, after results from all three dosing cohorts are available; the company may discuss findings sooner while aiming to preserve the study blind.
• The trial will test the SYNGAP1 treatment in patients ages 2–18, with three doses over three months and six months of follow-up. Preclinical studies showed increased gene expression and symptom reversal in mice, but clinical benefit in patients has not been established.
• CAMP4 is also advancing a SHANK3 program for Phelan-McDermid syndrome, aiming to select a development candidate next year, and has a GSK collaboration covering two undisclosed diseases.

CAMP4 Therapeutics NASDAQ: CAMP expects data from its Phase 1/2 study of CMP-002, its experimental treatment for SYNGAP1-related disease, in the first half of 2028, CEO Josh Mandel-Brehm said during a company presentation.

The company plans to wait until results from all three dosing cohorts are available, although it could discuss findings earlier if it sees something sooner. Mandel-Brehm said CAMP4 wants to maintain the study’s blind while it proceeds.

CMP-002 is designed to increase expression of the SYNGAP1 gene in patients with a condition in which one of two gene copies is nonfunctional. Mandel-Brehm said the company’s platform uses antisense oligonucleotides to target regulatory RNAs and typically aims for a roughly twofold increase in expression of an associated protein-coding gene.

Study Design and Preclinical Findings

The Phase 1/2 trial is enrolling patients ages 2 to 18 with mutations that make one SYNGAP1 gene copy nonfunctional. Enrollment criteria include daily seizures and an inability to speak, Mandel-Brehm said.

The study will move directly into multiple ascending doses, with three dosing cohorts and a three-to-one randomization between treatment and control. Patients will receive three doses over three months, followed by six months of follow-up. The company also plans an open-label extension after identifying an optimal biological dose.

CAMP4 has approvals in Argentina, Australia and the U.K., as well as from the European Medicines Agency, and plans to use eight to 10 sites globally, Mandel-Brehm said. He added that the study will use patients’ baseline periods and natural history data alongside its control group.

In preclinical work, Mandel-Brehm said the company observed a twofold increase in expression from the remaining functional allele in patient-derived cells. He also said the drug candidate reversed disease symptoms in a humanized mouse model and produced a statistically significant increase in SYNGAP gene expression in primates.

Those findings have not established clinical benefit in patients. Mandel-Brehm said it is difficult to predict when changes might appear, but the study duration could be sufficient to detect movement in several areas affected by the disease. Assessments include seizures, EEGs and measures of development, daily functioning and motor function.

Delivery and Market Estimates

CMP-002 is administered intrathecally, or into the spinal fluid. Asked about potential toxicity associated with that delivery route, Mandel-Brehm said the candidate uses an established antisense chemistry and showed a large therapeutic index in preclinical work. He said CAMP4 is monitoring alternative delivery technologies but is focused on intrathecal administration for the program.

Mandel-Brehm estimated that at least 10,000 people in the U.S. have SYNGAP1-related disease and that approximately one-quarter are diagnosed. He said existing medications often have limited effects on patients’ symptoms.

SHANK3 and Other Programs

CAMP4 is also developing a program targeting SHANK3 for Phelan-McDermid syndrome, another condition associated with a nonfunctional gene copy. Mandel-Brehm said the company aims to select a development candidate for the program next year and believes it can achieve a twofold increase in expression.

He estimated that 45,000 people in the U.S. have Phelan-McDermid syndrome, while noting continued underdiagnosis. He also cautioned that the program does not necessarily have the same quality of preclinical model available for SYNGAP1 and that whether treatment could reverse symptoms remains to be demonstrated.

Beyond its internal pipeline, Mandel-Brehm said CAMP4 has a collaboration with GSK involving two undisclosed diseases, one affecting the central nervous system and the other the kidney. The company also intends to pursue additional collaborations and advance other programs it has not disclosed.

About CAMP4 Therapeutics (NASDAQ:CAMP)

CAMP4 Therapeutics Corporation is a clinical-stage biotechnology company focused on developing medicines that increase the production of disease-relevant proteins by activating the expression of specific genes. The company's approach is designed to address diseases caused by insufficient protein levels, including certain genetic disorders in which boosting activity from a functional gene copy may provide therapeutic benefit. CAMP4 uses its Regulatory Action Platform, or RAP, to identify regulatory elements that control RNA production and discover small-molecule compounds capable of increasing the expression of selected genes.

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