CAMP4 Therapeutics Targets First SYNGAP1 Data in 2028 as Patient Dosing Nears

Key Points

• CAMP4 expects to begin dosing patients with its SYNGAP1 treatment, CMP-002, by year-end and report initial data in the first half of 2028. The Phase 1/2 trial will assess safety, biological dose and possible efficacy signals, but is not designed to establish efficacy; the company does not plan an interim readout.
• Preclinical studies showed increased SYNGAP1 expression and protein, with symptom improvements in a mouse model, but these results have not demonstrated benefit in patients. The company says it has clearance in several regions and is approaching the FDA.
• CAMP4 reported about $124.5 million in cash at the end of the third quarter, which it expects to fund operations through the end of 2028, including the SYNGAP1 readout and work on its SHANK3 and other programs.

CAMP4 Therapeutics NASDAQ: CAMP expects initial data from its SYNGAP1 clinical program in the first half of 2028, as the company prepares to begin dosing patients with a treatment designed to increase expression of a gene associated with a rare neurological disorder.

Chief Executive Officer Josh Mandel-Brehm discussed the program at a Guggenheim conference in Boston, where research analyst Delma Caiati said the company expects to dose its first patients by the end of the year. Mandel-Brehm said CAMP4 would provide progress updates but does not plan an interim safety or pharmacokinetic readout before the initial data.

“We want to maintain that blind as long as possible unless there’s a reason to not have it,” he said.

SYNGAP1 Trial Design

CAMP4 uses antisense oligonucleotides to target regulatory RNAs and increase gene expression. Its approach is aimed in part at diseases in which one of a patient’s two gene copies is not functional, resulting in insufficient protein production.

Mandel-Brehm said the company’s SYNGAP1 treatment, CMP-002, will be studied directly in patients in a global Phase 1/2 trial. The multiple-ascending-dose study will begin with three cohorts of at least eight patients each. Participants will have the opportunity to receive three doses over three months, followed by six months of monitoring.

The study includes a control arm with 3-to-1 randomization, a one-month baseline period and natural history data for comparison. Mandel-Brehm said the trial is intended to assess safety, find an optimal biological dose and look for efficacy signals; it is not powered to establish efficacy.

Researchers will measure seizures and brain activity using EEGs, along with cognitive and other clinical measures and sleep monitoring. Mandel-Brehm said an effect in any one of those areas could be meaningful for patients and support discussions with regulators.

The company has received regulatory clearance in Australia, Argentina, the U.K. and the EMEA, he said, and is approaching the U.S. Food and Drug Administration. He said it was too early to determine a potential approval pathway, though CAMP4 plans to explore accelerated approval with regulators.

Preclinical Results and Patient Population

Mandel-Brehm said CAMP4 observed a twofold increase in gene expression in patient-derived cells. In a humanized mouse model, he said, the treatment produced a dose-dependent increase in SYNGAP1 protein and reversed modeled symptoms involving learning, memory, cognition and anxiety, as well as an induced seizure state.

In primates, the clinical drug produced a statistically significant increase in gene expression at doses projected to be relevant in humans, he said. The drug was delivered intrathecally, the same route planned for the clinical trial. Those preclinical findings have not yet established a benefit in patients.

Mandel-Brehm estimated that at least 10,000 people in the United States have SYNGAP1 disorder, though he said it remains significantly underdiagnosed. He attributed improvements in diagnosis to greater awareness and the use of genetic screening and panels.

Pipeline, Partnerships and Cash

CAMP4 is also developing a SHANK3 program for Phelan-McDermid syndrome. Mandel-Brehm said the company chose it because of its biology, unmet need and similarities to SYNGAP1. He estimated that 45,000 people in the U.S. have the condition and said CAMP4 is working on additional, undisclosed programs.

The CEO also discussed a partnership with GSK, announced at the end of 2025, covering a large central nervous system disease and a kidney disease. CAMP4 is identifying regulatory RNAs and creating antisense oligonucleotides for the targets, after which GSK is expected to take over development. CAMP4 would receive milestones and royalties, he said.

Mandel-Brehm said CAMP4 had approximately $124.5 million at the end of the third quarter, which it expects to fund operations through the end of 2028. He said that runway would support the SYNGAP1 data readout and advancement of SHANK3 and other programs.

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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