Sign in

You're signed outSign in or to get full access.

TT

Tenaya Therapeutics, Inc. (TNYA)·Q4 2024 Earnings Summary

Executive Summary

  • Q4 2024 showed continued cost discipline with R&D ($18.69M) and G&A ($5.96M) down year over year, narrowing net loss to $23.84M ($0.28/share) vs. $29.94M ($0.40/share) a year ago; sequentially, both OpEx and net loss also improved from Q3 2024 ($26.71M OpEx; $25.63M net loss) .
  • Liquidity strengthened post-quarter via a $48.9M offering, extending cash runway to mid-2026 (vs. prior guidance of 2H 2025); cash at 12/31/24 was $61.4M (down from $79.5M at 9/30/24) before the raise .
  • Pipeline momentum: TN-201 (MYBPC3-HCM) Cohort 2 dosing underway with two participants treated and ACC late-breaker for Cohort 1; TN-401 (PKP2-ARVC) Cohort 1 enrolling, aided by an $8M CIRM grant; initial TN-401 data targeted for 2H25 .
  • Management emphasized early but “meaningful” TN-201 signals (robust cardiac transduction, rising mRNA and protein over time; manageable liver enzyme elevations) and set 2025 as a “data-rich” year—key stock catalysts are ACC late-breaker (Mar 31, 2025), MyPEAK-1 Cohort 2 initial data (2H25), and RIDGE-1 initial data (2H25) .

What Went Well and What Went Wrong

What Went Well

  • Safety/profile and dose escalation: DSMB clearance to escalate TN-201 to 6E13 vg/kg; Cohort 2 dosing initiated; enzyme elevations were transient/steroid-responsive; no cardiac toxicities/TMA observed .
  • Biological proof-of-concept: robust cardiac transduction at 3E13 vg/kg (VCN >2 at 8 weeks) with TN-201 mRNA and MyBP-C protein increasing over time (e.g., Patient 1 RNA +50% from week 8 to 52; protein +3% from week 8 to 52) supporting durable expression .
  • Funding runway extended and non-dilutive support: $48.9M net financing (Mar-2025) extends runway to mid-2026; $8M CIRM grant supports TN-401 clinical costs .

Quotes:

  • “These initial data derisk the safety of TN-201… and offer a promising potential read-through to our AAV9-based…TN-401” – CEO Faraz Ali .
  • “Throughout 2024… advances… positioned us for a data-rich 2025.” – CEO Faraz Ali .

What Went Wrong

  • Cash burn continues (pre-revenue): cash fell to $61.4M at 12/31/24 (from $79.5M at 9/30/24 and $104.6M at 12/31/23), necessitating capital raise (post-quarter) .
  • Biomarker/clinical signals mixed at this early look: NT‑proBNP remained elevated for Patient 1 at week 52; several endpoints are stable or not yet interpretable; larger and higher-dose cohorts needed to clarify efficacy .
  • Interest income declined QoQ and YoY, reflecting lower cash balances (Q4: $0.81M vs. Q3: $1.08M; Q4’23: $1.47M), modestly increasing net loss pressure absent revenue .

Financial Results

MetricQ4 2023Q3 2024Q4 2024
R&D Expense ($USD Millions)$22.865 $20.350 $18.688
G&A Expense ($USD Millions)$8.581 $6.361 $5.964
Total Operating Expenses ($USD Millions)$31.446 $26.711 $24.652
Interest Income ($USD Millions)$1.470 $1.080 $0.812
Net Loss ($USD Millions)$(29.935) $(25.634) $(23.836)
Diluted EPS ($)$(0.40) $(0.30) $(0.28)
Weighted Avg Shares (Millions)74.098 86.163 86.406
Cash, Cash Equivalents & Marketable Securities ($USD Millions)$104.642 (12/31/23) $79.469 (9/30/24) $61.446 (12/31/24)

Notes:

  • No product revenue reported; income statement presentation shows operating expenses and loss from operations without revenue line items .

KPIs and Operational Metrics

KPIQ2 2024Q3 2024Q4 2024 / Subsequent Update
MyPEAK-1 Cohort 1 (TN-201 @ 3E13 vg/kg)Screening/enrollment ongoing First 3 patients dosed; DSMB endorsed escalation ACC late-breaker accepted; additional Cohort 1 data Mar 31, 2025
MyPEAK-1 Cohort 2 (TN-201 @ 6E13 vg/kg)—DSMB recommended escalate Dosing initiated; 2 participants dosed; enrollment completion 1H25
RIDGE-1 (TN-401)Planned to start dosing Q4 2024 Sites activated First patient dosed; Cohort 1 completion 1H25; initial data 2H25
Natural History (MyClimb/RIDGE)Ongoing Ongoing MyClimb >200 enrolled; RIDGE >100 enrolled; data in 2H25 (MyClimb) / 1H25 (RIDGE)

Guidance Changes

MetricPeriodPrevious GuidanceCurrent GuidanceChange
Cash RunwayCompany operationsInto 2H 2025 Into mid-2026 (post $48.9M net offering) Raised/Extended
MyPEAK-1 Cohort 1 data timingCohort 1Initial data Dec 2024 Additional Cohort 1 data at ACC Mar 31, 2025 Clarified follow-up
MyPEAK-1 Cohort 2EnrollmentDSMB endorsed escalation; enroll beginning Q4’24/Q1’25 Enrollment completion in 1H25 Timed
MyPEAK-1 Cohort 2Initial dataNot previously guidedInitial Cohort 2 data in 2H25 New
RIDGE-1 (TN-401)EnrollmentStart dosing Q4 2024 Cohort 1 completion 1H25 Timed
RIDGE-1 (TN-401)Initial data2025 (initial) 2H25 (initial Cohort 1 data) Narrowed window
Non-interventional studiesMyClimb/RIDGEOngoing MyClimb data 2H25; RIDGE data 1H25 New timing detail

Earnings Call Themes & Trends

TopicPrevious Mentions (Q-2 and Q-1)Current Period (Q4 2024 context)Trend
TN-201 safety and dose escalationDSMB endorsed escalation to 6E13; broadened criteria Enzyme elevations transient/steroid-managed; no cardiac toxicities/TMA; Cohort 2 dosing underway Positive / advancing
Cardiac delivery/expression—Robust transduction (VCN >2 at week 8), TN‑201 mRNA up 50% by 1 year; protein +3% from 8 to 52 weeks (Patient 1) De-risking biology
RIDGE-1 progressSites activated; planned Q4’24 dosing First patient dosed; Cohort 1 completion 1H25; initial data 2H25; $8M CIRM grant Advancing
Manufacturing readinessResearch/manufacturing posters; platform advances Internal cGMP; 1,000L scale; Phase Ib supply on hand De-risked
Financing runway$99.3M cash (6/30); runway into 2H25 $61.4M cash (12/31) plus $48.9M raise extends runway to mid-2026 Improved duration
Regulatory/designations/IPTN‑201 RPDD; patents for PKP2 program TN‑301 HFpEF method patent to 2040 Supportive

Management Commentary

  • CEO framing: “These initial data derisk the safety of TN-201… provide us with increased confidence for the continued success of this ongoing Phase Ib/II trial of TN-201 and offer a promising potential read-through to… TN-401” .
  • CMO on safety: “TN-201 was generally well tolerated… no cardiac toxicities… elevations in liver enzymes… well managed with corticosteroids” .
  • CMO on expression: “Clear evidence of RNA expression… 50% increase by week 52 in Patient 1… accompanied by an increase in protein level over time” .
  • CEO on 2025: “We are excited to share substantially more data in 2025, including longer follow-ups… and initial results for Cohort 2… 2025 will also be an important year for our TN‑401 program” .
  • CFO/Finance update (press release): “Recent $48.9 Million financing extends cash runway into mid-2026” .

Q&A Highlights

  • Transaminase elevations and mitigation: First patient reached Grade 3 (~≥5x ULN) late in steroid taper; increased monitoring enabled earlier steroid intervention in later patients; no bilirubin elevation or hepatic damage .
  • Dosing strategy: Cohort 2 will dose sequentially per FDA-aligned protocol for first three patients; expansion at one or both dose levels remains flexible after initial Cohort 2 data .
  • Expression scaling: At low end of dose–response, 2x dose can yield >2x expression; team prioritizes protein output but set no fixed numeric target given inter-patient variability .
  • Endpoints/MRI: Current endpoints mirror approved HCM programs and peer gene therapies; MRI may be incorporated where feasible as eligibility now allows non-ICD patients (less artifact) .
  • Manufacturing: Internal cGMP with 1,000L scale; sufficient Phase Ib drug made; future scale contingent on clinical trajectory .
  • Pediatrics: High unmet need in severe pediatric MYBPC3-HCM; MyClimb data to inform strategy and potential engagement with FDA for accelerated pathways .

Estimates Context

  • S&P Global consensus (EPS, revenue, EBITDA) for Q4 2024 was not available to retrieve at the time of analysis due to data access limits; as a pre-revenue biotech, revenue/EPS estimates are not widely published and are less relevant to near-term valuation drivers for TNYA. As a result, no vs-consensus comparisons are shown (S&P Global retrieval unavailable).
  • Where estimates may adjust: 2025 clinical timing clarity (ACC late-breaker; Cohort 2 and RIDGE-1 milestones) and extended runway could affect expense and cash burn estimates, as well as probability-of-success and timing assumptions in pipeline models .

Key Takeaways for Investors

  • 2025 is the pivotal year: additional Cohort 1 data (ACC), first look at high-dose Cohort 2 (2H25), and initial TN‑401 data (2H25) are the principal catalysts likely to drive the stock .
  • Early TN‑201 biopsy data de-risk delivery and expression; higher-dose cohort should test for greater expression and potential strengthening of clinical signals, the key to broader re-rating .
  • Safety manageable thus far (no cardiac toxicities/TMA; steroid-responsive LFTs), supporting continued dose escalation and broader eligibility—critical for trial velocity .
  • Cash runway into mid‑2026, bolstered by $48.9M raise and $8M CIRM grant, reduces near-term financing overhang through key data readouts .
  • Watch for MRI incorporation and endpoint refinement; LVMI and functional endpoints could be central to regulatory dialogues given peer precedents .
  • Medium-term thesis: If Cohort 2 strengthens protein/clinical signals, pathway to dose expansion and registrational planning (including pediatric strategy leveraging MyClimb) becomes more tangible .
  • Risk factors: pre-revenue profile, small early dataset with mixed clinical signals to date, biomarker variability, and gene therapy class risks (immune management, durability) warrant position sizing discipline .

Additional Relevant Press Releases (Q4 2024 context)

  • TN‑201 MyPEAK-1 update (DSMB endorsement, expanded eligibility; Oct 17, 2024) .
  • TN‑401 first patient dosed (RIDGE‑1; Nov 25, 2024) .
  • MyPEAK-1 Cohort 1 initial data (Dec 17, 2024) .

Prior Two Quarters (for trend)

  • Q3 2024: R&D $20.35M; G&A $6.36M; OpEx $26.71M; net loss $25.63M ($0.30); cash $79.47M; runway guided into 2H25 .
  • Q2 2024: R&D $22.65M; G&A $8.17M; OpEx $30.82M; net loss $29.43M ($0.34); cash $99.30M; runway guided into 2H25; SVB credit facility established .

Sources: Company press releases, Form 8-K exhibits, and management/transcript remarks as cited above .