Voyager Therapeutics(US:VYGR)2023-03-07 20:09Financial Data and Key Metrics Changes - As of December 31, 2022, Voyager reported cash, cash equivalents, and marketable securities of $118.8 million, with a pro forma cash position of approximately $320 million after factoring in recent collaborations [16][17]. - The company expects its cash position to be sufficient to meet planned operating expenses and capital expenditure requirements into 2025 [17]. Business Line Data and Key Metrics Changes - Voyager's pipeline includes four programs advancing through late research, with two wholly-owned programs: a humanized anti-tau antibody for Alzheimer's disease and an SOD1 gene silencing program for ALS [19][20]. - The GBA1 gene therapy for Parkinson's disease and another gene therapy program for Friedreich's ataxia are being advanced in collaboration with Neurocrine, which is fully funding both programs through Phase 1 [21]. Market Data and Key Metrics Changes - The strategic collaboration with Neurocrine provided $175 million upfront and up to $4.2 billion in potential milestones for rights to the GBA1 gene therapy program and three additional gene therapy programs [10][12]. - Novartis exercised options for capsids against two neurologic disease targets, triggering $25 million in option exercise fees and eligibility for up to $600 million in associated milestone payments [13][14]. Company Strategy and Development Direction - Voyager focuses on three pillars: the TRACER capsid discovery platform, a CNS pipeline targeting diseases with high unmet needs, and strategic partnerships to enable neurogenetic medicine [6][10]. - The company aims to advance wholly-owned programs while leveraging non-dilutive financing from partnerships to support its pipeline [10][17]. Management's Comments on Operating Environment and Future Outlook - Management acknowledged the transformation Voyager underwent in 2022, emphasizing advancements in their pipeline and collaborations despite challenging market conditions [6][10]. - The company plans to file INDs for its anti-tau antibody and SOD1 gene therapy programs in 2024, with ongoing efforts to break through barriers in gene therapy and neurology [23][17]. Other Important Information - Voyager is exploring delivery modalities beyond AAV, including coding the LMP and fusing it to ASL or siRNA [34]. - The company is actively evaluating siRNA gene silencing approaches for Alzheimer's disease, targeting intracellular tau [22]. Q&A Session Summary Question: Discussion on TRACER capsids and IND timelines - Management indicated that the IND for the SOD1 ALS program is planned for 2024, and it may be the first capsid in the clinic from the TRACER platform [26]. Question: Non-human primate procurement issues - Management acknowledged the industry-wide challenges in procuring non-human primates but reported no current delays in their programs [29]. Question: Receptor X and delivery modalities - The identified receptor X is sufficient for delivery, and the company is exploring other delivery modalities [34]. Question: Collaboration with Neurocrine - Rights for targets not opted into by Pfizer and Novartis revert to Voyager, allowing the company to pursue those targets independently [31]. Question: GBA1 program and co-development options - Voyager views partnering as essential for common diseases like Parkinson's and is excited about the GBA1 program's potential [47]. Question: Neurofilament light chain as a surrogate marker - Management believes neurofilament can be a reliable surrogate marker for efficacy, which could shorten the approval pathway if accepted by the FDA [58]. Question: TRACER capsids studies by Pfizer and Novartis - Both companies conducted their own evaluations of TRACER capsids before exercising options, providing valuable insights to Voyager [61].