Voyager Therapeutics(VYGR) - 2022 Q3 - Earnings Call Transcript

Financial Data and Key Metrics Changes - Voyager Therapeutics reported cash, cash equivalents, and marketable securities of $132 million as of September 30, 2022, which is expected to be sufficient to meet planned operating expenses and capital expenditures into 2024 [16][17] - The company recognized $10 million in revenue and $30 million in deferred revenue in Q3 2022 due to Pfizer exercising their capsid option [13] Business Line Data and Key Metrics Changes - The TRACER capsids discovery platform is generating breakthrough capsids, with over 20 million variants evaluated, leading to more than 100-fold higher trans-gene expression in the brain compared to conventional AAV9 capsids [9][10] - Voyager's pipeline includes three prioritized programs targeting Alzheimer’s disease, Parkinson’s disease, and ALS, with IND filings expected in 2024 and 2025 [15][30] Market Data and Key Metrics Changes - The company is targeting serious life-threatening diseases, with a focus on leveraging biomarkers for efficient proof of biology [15] - The collaboration with Neurocrine for gene therapy development is currently funded through Phase 1, with Voyager having the option to co-develop and co-commercialize the asset [21] Company Strategy and Development Direction - Voyager aims to break through barriers in gene therapy and neurology, focusing on delivery challenges and safety issues associated with larger molecules [6][7] - The company is open to partnerships on capsids and programs, indicating ongoing discussions with multiple parties [45] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the ability to translate preclinical results into human clinical trials, particularly with the novel capsids derived from the TRACER platform [11][12] - The company anticipates identifying lead candidates for its pipeline programs by the first half of 2023, with ongoing updates expected as they advance towards INDs [34] Other Important Information - Voyager appointed new members to its Board and executive team, including Dr. Kitty Mackey and Peter Pfreundschuh [33] - The company has no debt obligations to third parties, enhancing its financial stability [18] Q&A Session Summary Question: Development candidate selection and capsid strategy - Management indicated that capsid profiles are tailored for each program, aiming to select the best capsid for optimal patient treatment [38][39] Question: Future partnership appetite - Management confirmed ongoing conversations with multiple parties and expressed openness to partnerships on capsids and programs [45] Question: Measures for selecting lead candidates in GBA1 and SOD1 therapies - The team is evaluating vector distribution, expression levels, and off-target issues in nonhuman primates to inform candidate selection [49][50] Question: Predictive nature of NHP models - Management acknowledged that while NHP models are the best available, human trials are necessary for definitive results [52][56] Question: CNS gene therapy guidance impact - Management noted that the FDA's guidance emphasizes safety and therapeutic window, which aligns with their development approach [72][73] Question: Cash runway and potential milestones - Management highlighted that potential milestone payments from partnerships could extend the cash runway into 2024 [76][78] Question: R&D expense direction for 2023 - Management indicated that R&D expenses for 2023 are expected to follow a similar trajectory as 2022, with a focus on prudent capital investment [81][82] Question: Importance of DRG de-targeting in capsids - Management confirmed that de-targeting is crucial to minimize toxicity while enhancing CNS delivery [85] Question: Immunogenicity of novel capsids - Management stated that they can test the immunogenicity of novel capsids against pre-existing antibodies in patients [89] Question: Safety concerns regarding identified receptors - Management indicated that there are currently no known safety concerns related to the identified receptor for BBB penetration [106]