CAMP4 Therapeutics, Inc.: Initiation of Research Coverage

William Blair initiated research coverage of CAMP4 Therapeutics, Inc. (CAMP $10.05). Camp4 is pioneering therapies for rare diseases by harnessing recently discovered regulatory RNA (regRNA) to modulate epigenetic expression of genetic targets.

Analyst Myles Minter, Ph.D. said, "Camp4 is an RNA therapeutics company that develops therapies for rare diseases. Building on the discovery of regRNA, Camp4 has developed a proprietary RAP platform to identify specific regRNAs targetable in specific disease states. The company's proprietary EPIC machine learning model integrates multiple DNA- and RNA-based sequencing datasets to identify such genes and regRNAs, and then uses a screening approach to identify specific antisense oligonucleotides, or ASOs, that will serve to boost gene expression. Thus, through the company's discovery and technology platforms, Camp4 can enact titratable changes to gene production."

Dr. Minter continued, "The company's lead asset, CMP-CPS-001, is targeting a regRNA that modulates expression of the Cps1 gene for the treatment of urea cycle disorders. Following positive preclinical studies, Camp4 is conducting a Phase I study for CMP-CPS-001 in healthy volunteers with data from the single ascending dose portion expected in the first quarter of 2025, which will focus on safety. Data from the multiple ascending dose portion of the study should read out in the second half of the year, which will provide key biomarker data, including ureagenesis. Pending positive results from the Phase I study, the company expects to initiate a Phase II/III pivotal study for CMP-CPS-001 in 2026 and would be positioned as a disease-modifying therapy. Camp4's second clinical asset targets the regRNA-controlling expression of the SYNGAP1 gene, which can cause SYNGAP1-RD, a group of neurodevelopmental conditions. Camp4's SYNGAP1-targeting asset will be targeted to the central nervous system via intrathecal delivery, and the company is preparing for preclinical proof-of-concept studies and GLP toxicology in 2025."

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William Blair or an affiliate received compensation for investment banking services or non-investment-banking services from CAMP4 Therapeutics, Inc. within the last 12 months. CAMP4 Therapeutics, Inc. is or was, within the last 12 months, an investment banking client of William Blair and/or one or more of its affiliates.

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Current Ratings Distribution (as of 11/4/24)

Coverage Universe
Outperform (Buy): 72%
Market Perform (Hold): 27%
Underperform (Sell): 1%

Inv. Banking Relationships*
Outperform (Buy): 8%
Market Perform (Hold): 1%
Underperform (Sell): 0%

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