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Beam Therapeutics Inc. (BEAM)·Q1 2025 Earnings Summary

Executive Summary

  • Q1 2025 was operationally strong but financially light versus consensus: license & collaboration revenue of $7.47M and EPS of -$1.24 versus S&P Global consensus of $14.68M* and -$1.17*, respectively (miss on both) . Values retrieved from S&P Global.*
  • Strategic momentum accelerated: first patient dosed in BEAM-301 (GSDIa), Part A fourth cohort initiated and U.S. IND cleared for BEAM-302 (AATD), and updated BEAM-101 (SCD) data accepted for EHA June; cash runway extended into 2028 following ~$500M financing .
  • BEAM-302 delivered first-ever clinical genetic correction in AATD with 60 mg cohort achieving mean total AAT 12.4µM at Day 28 (above 11µM protective threshold) and up to 78% reduction in mutant Z-AAT; FDA granted RMAT designation, enabling enhanced regulatory dialogue ahead of H2’25 data update .
  • Stock reaction catalysts: H2’25 BEAM-302 updated data; EHA June BEAM-101 update; BEAM-302 Part B start; BEAM-103 Phase 1 HV initiation by YE’25; extended funding runway de-risks near-term execution .

What Went Well and What Went Wrong

What Went Well

  • BEAM-302 clinical PoC in AATD with dose-dependent increases in total and functional AAT and reductions in Z-AAT; 60 mg cohort reached 12.4µM total AAT at Day 28 (above protective threshold) and up to 78% Z-AAT reduction; additional biomarker data showed corrected M‑AAT ~91% of total at Day 28 in 60 mg cohort .
  • Platform and regulatory de-risking: U.S. IND clearance and RMAT designation for BEAM-302, plus initiation of 75 mg (fourth) cohort; H2’25 data update planned .
  • Capital and runway: ~$500M registered direct financing extended cash runway to 2028, supporting BEAM-101, ESCAPE, BEAM-301, and BEAM-302 milestones . “This significant clinical progress is supported by our strong financial foundation…extends our projected cash runway into 2028” — CEO John Evans .

What Went Wrong

  • Financials came in below consensus: revenue $7.47M vs $14.68M* and EPS -$1.24 vs -$1.17* for Q1 2025 (misses) . Values retrieved from S&P Global.*
  • Operating spend rose year over year: R&D $98.8M vs $84.8M (+16.5%); G&A $27.9M vs $26.7M; total operating expenses $126.8M vs $111.5M; net loss widened to $109.3M (from $98.7M) .
  • No Q1 2025 earnings call transcript available to clarify drivers/variability of collaboration revenue; management context instead derived from company press releases and the May RBC conference discussion .

Financial Results

Historical performance (oldest → newest)

MetricQ3 2024Q4 2024Q1 2025
License & Collaboration Revenue ($M)$14.27 $30.07 $7.47
Research & Development Expense ($M)$94.26 $101.44 $98.82
General & Administrative Expense ($M)$26.52 $28.66 $27.94
Total Operating Expenses ($M)$120.77 $130.10 $126.76
Loss from Operations ($M)$(106.50) $(100.04) $(119.29)
Net Loss ($M)$(96.67) $(90.35) $(109.27)
Net Loss per Share ($)$(1.17) $(1.09) $(1.24)
Cash, Cash Equivalents & Marketable Securities (Period-End, $B)$0.926 $0.851 $1.220

Q1 2025 vs S&P Global consensus

MetricQ1 2025 ActualQ1 2025 Consensus
Revenue ($M)$7.47 $14.68*
EPS ($)$(1.24) $(1.17)*

Values retrieved from S&P Global.*

Notes:

  • All dollar amounts reflect reported figures (license & collaboration revenue recognized; no product revenue) .

Guidance Changes

MetricPeriodPrevious GuidanceCurrent GuidanceChange
Cash runwayMulti‑yearFund operations into 2027 (as of 12/31/24) Fund operations into 2028 (post $500M financing) Raised
BEAM-302 data updates (Part A)H2 2025Initial data 1H 2025 (delivered March/April) Further data at medical conference in H2 2025 New event (post initial)
BEAM-302 Part B first patientH2 2025—First patient dosing planned H2 2025 New
BEAM-301 first patientEarly 2025Dosing expected early 2025 First patient dosed in U.S. Phase 1/2 Achieved
BEAM-101 BEACON dosingMid‑2025Dose 30 patients by mid‑2025 On track to dose 30 patients by mid‑2025; updated data at EHA June Maintained
BEAM-103 Phase 1 HV startYE 2025Phase 1 enabling in 2024; HV by YE 2025 HV trial initiation by YE 2025 Maintained
BEAM-302 U.S. IND / RMAT2025—U.S. IND cleared; RMAT designation granted New regulatory milestones

Earnings Call Themes & Trends

Note: No Q1 2025 earnings call transcript was available. Current-period commentary reflects the Q1 press release and May 20, 2025 RBC conference discussion.

TopicPrevious Mentions (Q3’24)Previous Mentions (Q4’24)Current Period (Q1’25)Trend
In vivo base editing (BEAM-302, AATD)First cohort dosing completed; initial data expected 2025 Initial data expected 1H25 PoC achieved; 60 mg cohort >11µM total AAT; U.S. IND cleared; 75 mg cohort initiated; RMAT granted; further H2’25 data Accelerating; de-risked platform/regulatory
BEAM-301 (GSDIa)IND open; dosing expected early 2025 Site activation; dosing early 2025 First patient dosed Transition to clinical execution
BEAM-101 (SCD)35 enrolled/8 dosed; initial data highlighted; “best-in-class” potential versus trait-like HbF/S profile Adult enrollment target achieved; data mid‑2025 EHA June updated data; on track to dose 30 by mid‑2025 Steady progress to registrational potential
ESCAPE platform (non-genotoxic conditioning)NHP PoC; robust HbF with antibody-only conditioning Phase 1 enabling studies ongoing BEAM-103 HV trial by YE’25; management positioning as market-expanding evolution Moving toward clinic
Capital/RunwayRunway into 2027 Reiterated into 2027 $500M financing; runway into 2028 Improved
Regulatory path (AATD)——RMAT designation; plan for accelerated pathways discussion; biomarker strategy context (AAT, Z‑AAT, CT densitometry) Clearer pathway emerging

Management Commentary

  • “We achieved a historic milestone with BEAM-302, delivering the first-ever clinical genetic correction of a disease-causing mutation for alpha-1 antitrypsin deficiency…initiating the fourth cohort…securing U.S. FDA clearance for our IND” — John Evans, CEO .
  • On AATD strategy and endpoints: “We’re fixing the disease at its root cause… composite biomarkers (total/functional AAT up, Z‑AAT down) support accelerated paths; CT densitometry favored for lung longer-term” — RBC conference .
  • On platform/LNP safety: “It’s a fundamentally different LNP… very well tolerated… de-risked now for the rest of our platform” — RBC conference .
  • On market evolution in SCD: “Wave 1 therapies effective for severe patients; ESCAPE could expand and ultimately replace Wave 1 if equally effective” — RBC conference .

Q&A Highlights

  • Regulatory acceleration for BEAM-302: With RMAT, Beam plans a “new conversation” with FDA leveraging biomarker correction (total/functional AAT, Z‑AAT) and exploring confirmatory functional endpoints (e.g., liver biopsy aggregates, CT densitometry) .
  • Dosing strategy: Targeting higher fold changes with 75 mg to further lift total AAT; emphasis on fold-change from baseline and durable, physiologic regulation post-editing .
  • Safety/translatability: Differentiated LNP from prior industry issues; clean tolerability profile supports dose escalation and portability to BEAM-301 and future in vivo programs .
  • SCD portfolio strategy: Maintain BEAM-101 momentum for severe patients while advancing ESCAPE to broaden eligibility and potentially shift standard of care longer term .

Estimates Context

  • Q1 2025 results vs S&P Global consensus: Revenue $7.47M vs $14.68M*; EPS -$1.24 vs -$1.17* (misses on both). Values retrieved from S&P Global.*
  • Implications: Given collaboration-driven revenue, estimate dispersion can be high; no product revenue yet. Continued clinical/regulatory momentum (BEAM-302 PoC, RMAT) may shift investor focus toward data/catalysts while estimates adjust to collaboration timing .

Key Takeaways for Investors

  • Near-term catalyst stack is unusually rich: BEAM-101 updated data at EHA (June), BEAM-302 H2’25 update, BEAM-302 Part B start, and BEAM-103 HV initiation by YE’25 .
  • BEAM-302 PoC plus RMAT materially de-risks the in vivo franchise and clarifies a potential accelerated regulatory pathway anchored in composite biochemical correction, with functional follow-ups planned .
  • Financing extends runway into 2028, reducing capital risk through the next major readouts and potential pivotal planning .
  • Despite Q1 financial misses versus consensus, the stock narrative is driven by clinical execution and platform validation; watch for 75 mg BEAM-302 cohort data quality/signals in H2 .
  • In SCD, BEAM-101 continues progressing toward a potential registrational package, while ESCAPE could expand the market and improve the risk–benefit profile over time .
  • Execution risks: collaboration revenue variability; clinical and regulatory execution for accelerated approval pathways; manufacturing scale-up for ex vivo and in vivo programs .

KPIs and Program Status (selected)

KPI / ProgramStatus Q3’24Status Q4’24Status Q1’25
BEAM-302 (AATD)First cohort dosing complete; data 2025 planned Initial data expected 1H25 PoC achieved; 75 mg cohort initiated; U.S. IND cleared; RMAT granted; further data H2’25
BEAM-301 (GSDIa)IND open; dosing early 2025 expected Site activation ongoing First patient dosed
BEAM-101 (SCD, BEACON)35 enrolled; 8 dosed Adult enrollment target achieved; data mid‑2025 EHA June updated data; on track to 30 dosed by mid‑2025
Cash runwayInto 2027 Into 2027 Into 2028 post $500M raise

Citations for all data points are provided inline above. Values marked with an asterisk () are from S&P Global consensus estimates; Values retrieved from S&P Global.