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Taysha Gene Therapies(TSHA) - 2024 Q2 - Earnings Call Transcript
TSHATaysha Gene Therapies(TSHA)2024-08-12 16:08
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Financial Data and Key Metrics Changes - Research and development expenses decreased to 15.1millionforQ22024from15.1 million for Q2 2024 from 19.8 million in Q2 2023, primarily due to a milestone fee payable in the prior year [18] - General and administrative expenses increased to 7.3millioninQ22024from7.3 million in Q2 2024 from 6 million in Q2 2023, attributed to higher noncash stock-based compensation and consulting fees [19] - Net loss for Q2 2024 was 20.9millionor20.9 million or 0.09 per share, compared to a net loss of 24.6millionor24.6 million or 0.38 per share in Q2 2023 [19] - As of June 30, 2024, the company had 172.7millionincashandcashequivalents,withexpectationstosupportoperationsintoQ42026[19]BusinessLineDataandKeyMetricsChanges−TheTSHA−102programforRettSyndromeshowedpromisingsafetyandefficacydatafromthelowdosecohortinongoingclinicaltrials[7][11]−ThecompanyinitiatedthehighdosecohortandexpandeditspediatrictrialintoCanada,indicatingprogressinclinicalevaluations[7][10]MarketDataandKeyMetricsChanges−RettSyndromeaffectsanestimated15,000to20,000patientsintheU.S.,EU,andUK,withnoapproveddisease−modifyingtherapiescurrentlyavailable[8]CompanyStrategyandDevelopmentDirection−ThecompanyaimstodevelopatransformativetherapeuticoptionforallpatientssufferingfromRettSyndrome,focusingonclinicaltrialexecutionanddatacollection[8][21]−Thestrategyincludesadvancingtohighdosecohortsbasedonencouragingpreliminarydatafromlowdosecohorts,whichisexpectedtoinformregulatorydiscussions[12][21]Management′sCommentsonOperatingEnvironmentandFutureOutlook−ManagementexpressedconfidenceinthesafetyprofileandclinicaleffectsofTSHA−102,emphasizingthepotentialtoaddresssignificantunmetmedicalneedsinRettSyndrome[20][21]−Thecompanyplanstoreportsafetyandefficacydatafrombothlowandhighdosecohortsinthefirsthalfof2025,indicatingacommitmenttotransparencyanddata−drivendecision−making[14][21]OtherImportantInformation−Thecompanycompletedapublicfollow−onoffering,resultinginnetproceedsof172.7 million in cash and cash equivalents, with expectations to support operations into Q4 2026 [19] Business Line Data and Key Metrics Changes - The TSHA-102 program for Rett Syndrome showed promising safety and efficacy data from the low dose cohort in ongoing clinical trials [7][11] - The company initiated the high dose cohort and expanded its pediatric trial into Canada, indicating progress in clinical evaluations [7][10] Market Data and Key Metrics Changes - Rett Syndrome affects an estimated 15,000 to 20,000 patients in the U.S., EU, and UK, with no approved disease-modifying therapies currently available [8] Company Strategy and Development Direction - The company aims to develop a transformative therapeutic option for all patients suffering from Rett Syndrome, focusing on clinical trial execution and data collection [8][21] - The strategy includes advancing to high dose cohorts based on encouraging preliminary data from low dose cohorts, which is expected to inform regulatory discussions [12][21] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the safety profile and clinical effects of TSHA-102, emphasizing the potential to address significant unmet medical needs in Rett Syndrome [20][21] - The company plans to report safety and efficacy data from both low and high dose cohorts in the first half of 2025, indicating a commitment to transparency and data-driven decision-making [14][21] Other Important Information - The company completed a public follow-on offering, resulting in net proceeds of 76.8 million, which is expected to extend the cash runway into Q4 2026 [13] Q&A Session Summary Question: How will the high-dose data be measured for dose response? - Management indicated that preclinical data showed improvements at high doses and that consistent effects across clinical domains are expected [22] Question: What is the status of regulatory discussions? - The company has an upcoming Type B meeting with regulatory authorities to align on trial design and data sharing [26] Question: Will the timeline for data impact the timeline for FDA meetings? - Management stated that they aim to complete dosing and natural history analysis before engaging with the FDA, with flexibility in the timeline [42] Question: What are the baseline characteristics of high-dose patients? - Management noted that baseline characteristics are consistent with Rett Syndrome, with common clinical features expected across patients [48] Question: Are there any updates on safety events related to the immunosuppressive regimen? - Management confirmed that there have been no new significant adverse events reported and that discussions regarding the dosing stagger are ongoing [52]