Taysha Gene Therapies(TSHA) - 2022 Q3 - Earnings Call Transcript

Financial Data and Key Metrics Changes - Research and development expenses decreased to $16.4 million for Q3 2022 from $39.5 million in Q3 2021, a reduction of $23.1 million attributed to lower costs in various areas [37] - General and administrative expenses were $8.7 million for Q3 2022, down from $11.2 million in Q3 2021, reflecting a decrease of approximately $2.5 million [38] - Net loss for Q3 2022 was $26.3 million or $0.64 per share, compared to a net loss of $51.2 million or $1.35 per share in Q3 2021 [39] - As of September 30, 2022, cash and cash equivalents stood at $34.3 million, excluding a recent $50 million investment from Astellas and $25.6 million from a follow-on offering [40] Business Line Data and Key Metrics Changes - The GAN program (TSHA-120) demonstrated clinically meaningful slowing of disease progression across all therapeutic dose cohorts, with effects observed up to five years post-dosing [21] - TSHA-102 for Rett syndrome is the first gene therapy in clinical development, with preclinical data showing significant improvements in survival and behavior in Rett knockout mice [28][30] Market Data and Key Metrics Changes - The partnership with Astellas includes a $50 million investment for 15% of Taysha's outstanding shares, highlighting the market opportunity for TSHA-120 and TSHA-102 [13] - The company anticipates updates on regulatory pathways and clinical data in early 2023, which could impact market perception and stock performance [19][42] Company Strategy and Development Direction - The strategic investment from Astellas is aimed at supporting the development of TSHA-120 for GAN and TSHA-102 for Rett syndrome, indicating a focus on gene therapy for serious genetic diseases [11][12] - The company plans to leverage Astellas' expertise in gene therapy to enhance its capabilities and expedite the development of its product candidates [16][18] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the upcoming FDA meeting to discuss the pathway to a BLA filing for TSHA-120, with expectations for regulatory updates in January 2023 [42] - The company is optimistic about the potential for its gene therapies to address significant unmet medical needs in neurodegenerative diseases [87] Other Important Information - The company has outlined possible scenarios for regulatory approval in Europe and the U.S., with a focus on achieving conditional and accelerated approvals based on existing data [25][26] - TSHA-102 has received orphan drug and rare pediatric disease designations from the FDA, enhancing its development prospects [34] Q&A Session Summary Question: Does Astellas have experience in manufacturing AAV9 ACE genetic medicine? - Management noted that while Astellas' experience is primarily with AAV8, the manufacturing processes are similar, allowing for effective collaboration [48][49] Question: What are the specified outcomes from the FDA meeting for Astellas to opt in? - Management indicated that Astellas is aware of the range of possible outcomes and is focused on the robustness of the data set rather than specific outcomes [51][53] Question: How will the registrational trial design differ from the ongoing Phase 1/2 in Rett syndrome? - Management stated that the registrational study will focus on the pediatric population, with similar assessment buckets but tailored endpoints for children [56][61] Question: What is the expected translatability of outcomes from adult patients to pediatrics? - Management explained that dosing for CNS delivered drugs is different, and after age four, the same dose is expected for both adults and children [67][69] Question: What is the bar for success in the initial clinical data for Rett syndrome? - Management emphasized that safety is paramount, and preliminary efficacy will be assessed across various endpoints, with expectations for better outcomes in children [72][78] Question: What triggers the start of the pediatric study? - Management indicated that patient experience and follow-up in the adult study will be the main triggers for initiating the pediatric study [84][87]