Avidity Biosciences, Inc. (RNA)·Q4 2024 Earnings Summary

Executive Summary

• Q4 2024 collaboration revenue was $3.0M, net loss was $102.3M, and EPS was $(0.80); cash, cash equivalents and marketable securities ended the year at ~$1.50B, providing substantial runway for 2025 execution .
• Company reiterated 2025 catalysts and commercial preparations: del-zota (DMD44) BLA submission targeted for year-end 2025 following FDA confirmation of the accelerated approval path; DM1 Phase 3 HARBOR enrollment completion mid-2025; del-brax (FSHD) global Phase 3 design and registrational initiation in Q2 2025 .
• The AOC platform narrative strengthened with 2024 readouts across all three clinical programs and next‑generation technology showing up to 30‑fold improvements in delivery in preclinical studies, supporting long-term product and pipeline durability .
• Wall Street consensus (S&P Global) for Q4 2024 EPS and revenue was unavailable at time of query; estimate comparison will be updated when accessible (consensus unavailable via S&P Global).

What Went Well and What Went Wrong

What Went Well

• FDA confirmed del-zota’s accelerated approval path and that the EXPLORE44 clinical package could support a BLA filing; BLA targeted for year-end 2025, de-risking regulatory trajectory in DMD44 .
• Strong balance sheet (~$1.5B cash at 12/31/24) to fund global commercial buildout and pipeline expansion; management emphasized transitioning to next stage with infrastructure development in 2025 .
• Positive 2024 readouts across programs: del-zota increased near full-length dystrophin to 25% of normal with robust exon 44 skipping; del-brax showed >50% reductions in DUX4-regulated genes and biomarker improvements; del-desiran long‑term data showed reversal of DM1 progression across multiple endpoints .
  • “Successful readouts from our three clinical-stage programs in 2024 demonstrate the consistent and reproducible data of our AOC platform…” — Sarah Boyce, CEO .

What Went Wrong

• Operating expenses stepped up sharply: R&D of $95.6M (+81% YoY) and G&A of $28.3M (+76% YoY) in Q4, driving a larger quarterly operating loss of $121.0M and net loss of $102.3M .
• Collaboration revenue remains modest ($3.0M in Q4), with no product revenue yet; the P&L is dominated by R&D as registrational work scales .
• Consensus estimate data from S&P Global was not available to assess beats/misses; limits timely comparison vs Street expectations (consensus unavailable via S&P Global).

Financial Results

Balance Sheet snapshot (period-end):

Estimates vs actuals (S&P Global consensus):

• Consensus unavailable at time of query; no comparison vs estimates can be provided (consensus unavailable via S&P Global).

KPIs and operational milestones:

• EXPLORE44-OLE enrollment complete to support del-zota BLA; topline EXPLORE44 in Q1 2025 and OLE topline in Q4 2025 planned .
• DM1 HARBOR Phase 3 enrollment completion targeted mid-2025; MARINA-OLE long-term update Q4 2025 .
• FSHD FORTITUDE biomarker cohort completion, topline, and registrational initiation targeted Q2 2025; accelerated approval alignment sought .

Guidance Changes

Earnings Call Themes & Trends

Note: No Q4 2024 earnings call transcript was available in the document catalog; themes reflect company-issued materials.

Management Commentary

• “We are extending our leadership position in the rare neuromuscular space as we plan to submit our first BLA for an AOC and prepare for three potential successive product launches…” — Sarah Boyce, President & CEO .
• “Our strong balance sheet with approximately $1.5 billion at the end of 2024 allows us to continue to expedite our global commercial infrastructure development… We are transitioning to the next stage…” — Mike MacLean, CFO .
• “The FDA confirmed the accelerated approval path is available for del-zota and that the clinical data package from the EXPLORE44 program could support a BLA filing.” — Company statement .

Q&A Highlights

• No Q4 2024 earnings call transcript was available in the document catalog; Q&A themes and clarifications cannot be assessed at this time.

Estimates Context

• S&P Global consensus for Q4 2024 EPS and revenue was unavailable due to access constraints; as a result, beats/misses vs the Street cannot be determined here (consensus unavailable via S&P Global).
• Given sizable opex increases and lack of product revenue, Street models likely emphasize cash runway and regulatory timing; updates may be needed as 2025 clinical and regulatory milestones materialize .

Key Takeaways for Investors

• Cash runway remains a core asset (~$1.5B), enabling simultaneous advancement of three rare neuromuscular programs and commercial readiness activities in 2025–26 .
• Regulatory de-risking in DMD44: FDA accelerated approval path confirmed for del-zota; BLA submission targeted for year-end 2025, with OLE enrollment complete to support filing .
• Multiple near-term catalysts in 2025: EXPLORE44 topline (Q1), del-brax biomarker cohort topline and Phase 3 design alignment (Q2), HARBOR enrollment completion (mid-2025), EXPLORE44‑OLE topline (Q4) .
• Platform momentum: Next‑gen AOC tech with up to 30x preclinical delivery improvement and first precision cardiology candidates (PRKAG2 and PLN) broaden long-term optionality .
• Expense step-up reflects registrational execution; monitor quarterly opex trajectory and any signals on commercial build pacing vs key readouts .
• With consensus unavailable, trading setups likely hinge on event path: regulatory updates, Phase 3 design alignment, and toplines—particularly del‑zota Q1 and del‑brax Q2 2025, which can be stock-moving catalysts .