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Taysha Gene Therapies, Inc. (TSHA)·Q3 2025 Earnings Summary

Executive Summary

  • Q3 2025 revenue was $0, missing S&P Global consensus of $1.38M*, while EPS was $(0.09), essentially in line/slightly better than the $(0.088) consensus*; net loss widened year over year to $32.7M as R&D scaled ahead of the pivotal program .
  • Strategic/regulatory momentum: FDA granted Breakthrough Therapy Designation for TSHA‑102 and aligned on the REVEAL pivotal protocol and SAP, including a 6‑month interim analysis that may serve as the BLA basis, potentially accelerating filing by ≥2 quarters .
  • Clinical update: Part A data continued to show robust efficacy and tolerability (100% responders on developmental milestones; no treatment‑related SAEs/DLTs), and first patient dosing in the pivotal is scheduled for Q4 2025, with broad site interest suggesting 3–6 month enrollment timing for 15 patients .
  • Balance sheet/capacity: Cash and equivalents were $297.3M (runway into 2028). TSHA increased ATM capacity to $212M and regained full global rights to TSHA‑102, enhancing strategic flexibility and funding options .

What Went Well and What Went Wrong

  • What Went Well

    • Breakthrough Therapy designation granted; FDA alignment on pivotal protocol/SAP with a 6‑month interim that may support BLA filing, providing a potentially faster regulatory path .
    • Clinical data robustness: 100% response rate across 10 Part A patients on natural‑history‑defined developmental milestones; 83% responder rate at 6 months in high‑dose cohort deepening to 100% by 9 months; no treatment‑related SAEs/DLTs across 12 patients as of Oct 2025 cut‑off .
    • Management tone and demand: “Demand to be in the trial is exceptionally high,” with >15 Centers of Excellence and potential 3–6 month enrollment; “no patient left behind” ambition as additional skills improve activities of daily living (ADLs) .

  • What Went Wrong

    • Revenue miss: reported Q3 revenue was $0 vs S&P Global consensus $1.38M*, creating a headline top‑line miss despite EPS in‑line/slightly better .
    • Operating spend ramp: R&D rose to $25.7M (vs $14.9M y/y) driven by PPQ manufacturing, REVEAL activities, and headcount; net loss widened to $32.7M (vs $25.5M y/y) .
    • Limited financial guidance: no product revenue guidance; commercialization timelines remain contingent on pivotal execution/BLA acceptance, with ex‑US strategy intentionally staged after U.S. focus .

Financial Results

Metric ($USD, Millions except per-share)Q3 2024Q2 2025Q3 2025
Revenue1.79 1.99 0.00
R&D Expense14.95 20.14 25.75
G&A Expense7.90 8.60 8.28
Net Loss25.52 26.88 32.73
Diluted EPS(0.10) (0.09) (0.09)
Cash & Equivalents (period end)—312.76 297.34

Vs Estimates (S&P Global)

MetricQ3 2025 ConsensusQ3 2025 Actual
Revenue ($M)1.38*0.00
EPS ($)(0.088)*(0.087) ≈ (0.09)

Values marked with * retrieved from S&P Global.

KPIs and Operating Metrics

KPIQ3 2025
Breakthrough Therapy Designation (TSHA‑102)Granted by FDA (Sept)
Pivotal Protocol & SAP (6‑mo interim)Finalized with FDA; interim may serve as BLA basis
Part A Efficacy100% responders (10/10); 83% at 6 months → 100% at 9 months (high dose)
Part A SafetyNo treatment‑related SAEs/DLTs across 12 pts (Oct 2025 cut)
Pivotal DosingFirst patient scheduled Q4 2025
Cash RunwayInto 2028

Guidance Changes

MetricPeriodPrevious GuidanceCurrent GuidanceChange
First patient dosing (pivotal)Q4 2025Enrollment “anticipated to begin in Q4 2025” (Q2 2025) First dosing scheduled Q4 2025 Firmed/specified
BLA timing pathwayPivotal interimInterim analysis contemplated (Q2) 6‑month interim may serve as BLA basis; could accelerate filing by ≥2 quarters Strengthened; potential acceleration
Cash runwayMulti‑yearInto 2028 (Q2 2025) Into 2028 (Q3 2025) Maintained
Younger cohort (2–<6 yrs)Label strategyFDA alignment on extrapolation approach (prior) Safety‑focused study with efficacy extrapolated from 6+; aim for 2+ label at approval Maintained/clarified
Ex‑US/Europe—Working to enable; focus U.S. first (Q2) Same prioritization; Europe enabled after further data/policy clarity Maintained
Capital markets—Public follow‑on $230M completed (May) ATM increased to $212M capacity; removed one agent Increased flexibility

Earnings Call Themes & Trends

TopicPrevious Mentions (Q1–Q2 2025)Current Period (Q3 2025)Trend
Regulatory alignmentRMAT-driven FDA alignment; IND/CTA amendments; site activation (Q2) Breakthrough designation; finalized protocol/SAP; 6‑mo interim as BLA basis Positive momentum
Efficacy durability100% responders in Part A; stronger high‑dose kinetics; multi‑domain gains (Q2) 83% → 100% high‑dose response by 9 months; added supplemental analyses of additional skills Reinforced/expanding
SafetyNo treatment‑related SAEs/DLTs across 12 pts (Q2) Remains generally well tolerated; no treatment‑related SAEs/DLTs (Oct cut) Steady/positive
Pivotal executionSite activation; Q4 enrollment anticipated (Q2) First dosing scheduled Q4; strong demand; 3–6 month enrollment possible Advancing
Younger cohort (2–<6)Extrapolation approach aligned with FDA (Q2) Aim for 2+ label; dosing likely mid‑2026; safety focus, efficacy extrapolated Clarity on timeline
Commercial readinessFinancing and CMC readiness (Q2) Chief Commercial Officer hired; payer‑relevant endpoints focus; large patient opportunity Building capability
Ex‑US strategyScientific advice planning; focus U.S. (Q2) Continue enabling U.K./EU; staging after U.S. focus Unchanged

Management Commentary

  • “The receipt of FDA Breakthrough Therapy designation… and finalized alignment with FDA on our REVEAL pivotal trial protocol and SAP… allows us to focus on executing our REVEAL pivotal trial and advancing towards BLA submission with clarity and confidence.” — CEO Sean Nolan .
  • “In addition to the developmental milestones achieved… patients consistently gained multiple additional skills… across autonomic function, communication, fine motor, and gross motor areas.” — President/Head of R&D Sukumar Nagendran .
  • “Research and development expenses were $25.7 million… Net loss… was $32.7 million, or $0.09 per share… cash and cash equivalents $297.3 million… we expect… cash resources will support… into 2028.” — CFO Kamran Alam .

Q&A Highlights

  • Enrollment dynamics: >15 Centers of Excellence, many with 100+ patients; best‑case enrollment of 15 pivotal patients in ~3 months, more conservatively 3–6 months; some sites can dose multiple patients in staggered fashion .
  • Interim/BLA threshold: FDA aligned on responder definition and 33% response‑rate success threshold (5/15 patients); 6‑month interim will be assessed after all patients reach 6 months; minimal alpha loss; methodology designed for objectivity via blinded central video raters .
  • Younger cohort label: Target approval with a broad “2+” label via efficacy extrapolation from ≥6 group; safety study initiation likely mid‑2026 pending FDA alignment .
  • Commercial framing: Payers value functional gains over small changes in scales (e.g., CGI); TSHA intends to highlight milestone‑based ADL improvements; leadership team with Zolgensma/Esbriet track records .
  • Ex‑U.S.: U.S. remains priority; enabling discussions with EU/U.K. continue, with timing paced by data/policy .

Estimates Context

  • Q3 2025: EPS of $(0.09) was essentially in line/slightly better than S&P Global consensus $(0.088); revenue of $0 missed the $1.38M consensus. Street models may reduce near‑term collaboration/other revenue assumptions and modestly increase 2025 operating expense run‑rate given PPQ/pivotal scale‑up .
    Values marked with * retrieved from S&P Global.

Key Takeaways for Investors

  • Regulatory de‑risking and potential timeline pull‑forward: Breakthrough designation and an FDA‑aligned 6‑month interim that may serve as BLA basis are meaningful accelerants/catalysts into 2026 .
  • Efficacy signal is strong and payer‑relevant: 100% responder rate in Part A with video‑adjudicated functional milestones, plus multi‑domain ADL improvements; no treatment‑related SAEs/DLTs to date .
  • Near‑term catalyst path: Q4 2025 pivotal first dosing; robust site demand suggests fast enrollment; longer‑term Part A update in H1 2026; interim analysis timing depends on enrollment pace .
  • Financing capacity/runway: ~$297M cash into 2028 and a $212M ATM increase provide flexibility to fund pivotal and pre‑commercial build‑out; regained TSHA‑102 global rights improve strategic optionality .
  • Risk factors: Zero revenue near‑term and rising R&D/PPQ spend widen losses; BLA path depends on successful pivotal execution, objective video adjudication, and FDA acceptance of interim data .
  • Trading setup: Stock likely sensitive to enrollment cadence, any safety updates, and clarity on interim read timing; upside on smooth execution/regulatory engagement, downside if timelines slip or data diverge.

Citations: Earnings call transcript (Q3 2025) –; 8‑K and press release (Q3 2025) –; 8‑K and press release (Q2 2025) –; earnings call transcript (Q2 2025) –; 8‑K and press release (Q1 2025) –. Values marked with * retrieved from S&P Global.