Camp4 Therapeutics (NasdaqGM:CAMP) FY Conference Transcript

Camp4 Therapeutics FY Conference Summary Company Overview - Company: Camp4 Therapeutics (NasdaqGM:CAMP) - Industry: Biotechnology, focusing on genetic disorders and CNS diseases - Key Product: CMP-002, an antisense oligonucleotide targeting SYNGAP1-related disorders Core Insights and Arguments 1. Flagship Program: Camp4 is developing CMP-002 for SYNGAP1-related disorders, a genetic haploinsufficiency CNS disorder with no approved treatments. This program aims to be the first disease-modifying therapy for SYNGAP patients, with tens of thousands affected globally [2][3][34]. 2. Platform Technology: The company utilizes a proprietary WRAP platform to identify and drug regulatory RNAs, allowing for selective increases in protein expression. This platform is designed to address various haploinsufficient diseases, including developmental epileptic encephalopathies (DEEs) [4][5][6]. 3. Preclinical Data: Strong preclinical data supports the efficacy of CMP-002, showing potential for significant disease modification. The program is currently completing GLP-tox studies, with plans to enter clinical trials later in the year [3][8][27]. 4. Market Opportunity: Camp4 estimates at least 20,000 patients in the U.S. and EU5 with SYNGAP1, with only about 25% diagnosed. Increased awareness and diagnostic capabilities are expected to drive this number higher [15][17]. 5. Collaborations: The company has formed a partnership with GSK to explore additional targets in CNS and kidney diseases, providing non-dilutive capital and expanding the application of its platform [9][60][61]. Important but Overlooked Content 1. Patient-Centric Approach: The presentation emphasized the impact of SYNGAP1 on patients and their families, highlighting the emotional and physical challenges faced by caregivers [10][11][12]. 2. Regulatory Strategy: Camp4 is engaging with regulatory agencies to facilitate a smooth transition into clinical trials, with a focus on rapid enrollment and diverse endpoints to demonstrate efficacy [28][30][50]. 3. Financial Stability: The company is funded through the end of 2027, with additional financing available upon achieving regulatory milestones, ensuring a stable runway for development [61]. Future Directions - Clinical Trials: Plans to initiate a global phase one-two study for CMP-002, focusing on various endpoints including seizures, sleep, motor function, and cognitive development [28][29][30]. - Expansion of Pipeline: Camp4 aims to leverage its platform to develop additional programs targeting other haploinsufficient diseases, with expectations to announce new initiatives later in the year [34]. - Long-Term Vision: The company aspires to become a leader in the field, drawing parallels to successful companies like Alnylam, while maintaining a strong focus on patient needs and innovative therapies [34].