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CAPRICOR THERAPEUTICS, INC. (CAPR)·Q4 2024 Earnings Summary

Executive Summary

  • Q4 2024 delivered a revenue and EPS beat versus S&P Global consensus, driven by milestone recognition and steady ratable revenue from the Nippon Shinyaku agreement; revenue was $11.13M and diluted EPS was -$0.16, both ahead of consensus . Estimates marked with * retrieved from S&P Global.*
  • The FDA accepted CAPR’s BLA for deramiocel with Priority Review and set a PDUFA target action date of August 31, 2025, sharpening the regulatory catalyst path laid out in Q3 (previously “2H25”) .
  • Cash, cash equivalents and marketable securities ended Q4 at $151.5M; with a $10M post-quarter milestone receipt and expected $80M on approval plus potential PRV monetization, management underscored runway into 2027 and robust financing options for commercialization and pipeline expansion .
  • Manufacturing capacity expansion (additional 25,000 sq ft) targets 2,000–3,000 patients per year by mid-2026, supporting anticipated launch demand; current San Diego facility supports ~250–500 patients per year for early commercialization .
  • Near-term stock reaction catalysts: AdCom determination (if any), PLI inspection timing, EMA engagement (with Orphan + ATMP designations), and Project NextGen milestones for StealthX vaccine; core narrative centers on first-in-class treatment for DMD cardiomyopathy and reimbursement readiness with NS Pharma .

What Went Well and What Went Wrong

  • What Went Well

    • BLA accepted with Priority Review; PDUFA set for 8/31/2025, solidifying a clear regulatory timeline . Quote: “We continue to work diligently towards our August 31, 2025 action date…preparing for pre-approval licensure inspection and potential commercial launch with Nippon Shinyaku” .
    • Q4 revenue/EPS beat vs consensus, aided by recognition of the $10M second development milestone and ratable revenue from the NS agreement . Quote: “Revenues for the fourth quarter of 2024 were approximately $11.1 million… ratable recognition of the $40.0 million… and the recognition of the $10.0 million second development milestone payment” .
    • Strengthened balance sheet and expansion plan: $151.5M year-end cash; pro forma ~$161.5M after $10M milestone; capacity expansion to 2,000–3,000 patients by mid-2026 .

  • What Went Wrong

    • Operating expenses rose materially YoY as CAPR accelerated R&D and commercialization build-out: Q4 total OpEx $18.8M vs $13.4M in Q4 2023, expanding losses .
    • Q3 showed a notable revenue shortfall versus consensus ($2.26M actual vs ~$3.57M est) and a larger EPS loss (-$0.38 actual vs -$0.358 est), highlighting quarter-to-quarter variability prior to Q4 momentum . Estimates marked with * retrieved from S&P Global.*
    • Q2 cash runway had been guided only into Q1 2025 before subsequent capital raises and milestone timing extended runway; this earlier constraint underscored dependence on external financing and milestones prior to strengthening the balance sheet later in the year .

Financial Results

Quarterly Actuals vs Consensus (oldest → newest)

MetricQ2 2024Q3 2024Q4 2024
Revenue ($USD) Actual$3,971,438 $2,261,642 $11,130,509
Revenue Consensus Mean ($USD)*$3,664,000*$3,568,330*$9,870,000*
Diluted EPS Actual ($USD)-$0.35 -$0.38 -$0.16
Primary EPS Consensus Mean ($USD)*-$0.356*-$0.35833*-$0.17*

Estimates marked with * retrieved from S&P Global.

  • Q4 2024 beat: Revenue +$1.26M vs consensus; EPS +$0.01 vs consensus (beats). Q3 2024 miss: Revenue -$1.31M; EPS -$0.02 (misses). Q2 2024 beat: Revenue +$0.31M; EPS +$0.006 (beats). Estimates marked with * retrieved from S&P Global.

Operating Performance

MetricQ2 2024Q3 2024Q4 2024
Total Operating Expenses ($USD)$15,562,657 $15,271,522 $18,828,350
EBIT / Loss from Operations ($USD)-$11,591,219 -$13,009,880 -$7,697,841
Net Loss ($USD)-$10,999,782 -$12,556,728 -$7,116,603
Net Loss Margin (%)-277.0% -555.3% -64.0%

Note: Net loss margin is derived from cited net loss and revenue values.

Balance Sheet KPIs

KPIQ2 2024Q3 2024Q4 2024
Cash, Cash Equivalents & Marketable Securities ($USD)$29,462,030 $85,028,624 $151,515,877

Pro forma cash including $10M milestone post year-end: ~$161.5M (as discussed on call) .

Revenue Drivers (qualitative context)

  • Ratable recognition of $40M (upfront + first development milestone) and recognition of $10M second development milestone following BLA submission in Dec 2024 .

Guidance Changes

MetricPeriodPrevious GuidanceCurrent GuidanceChange
Cash RunwayQ2 2024 → Q3/Q4 2024“Sufficient into Q1 2025” “Sufficient into 2027” Raised materially
Regulatory TimingQ3 2024 → Q4 2024“Potential PDUFA in 2H25” PDUFA target action date: Aug 31, 2025 Clarified/confirmed
BLA StatusQ2 2024 → Q4 2024Pre-BLA positive; rolling BLA initiation planned; completion by YE24 BLA accepted; Priority Review granted Advanced to review
Manufacturing CapacityQ3/Q4 2024San Diego facility supports ~250–500 pts/year Expanded lease (+25k sq ft); target 2,000–3,000 pts/year by mid-2026 Raised capacity plan
Milestones/PRVQ2/Q3 → Q4 2024~$700M US milestones incl. ~$90M to approval $10M milestone received in Jan 2025; expected $80M on approval; PRV rights to sell/transfer Clarified near-term cash events
Europe PathQ3 2024 → Q4 2024Binding term sheet with NS; potential $1.5B milestones EMA Orphan + ATMP granted; EMA meeting planned Q2 2025; definitive EU agreement under negotiation Progressed regulatory; deal timing TBD
StealthX Vaccine (Project NextGen)Q3 → Q4 2024/Q1–Q2 2025Deliver to NIAID in Q1 2025; initiate Phase 1 in Q1 2025 NIAID planning regulatory approval in Q2 2025; study initiation soon thereafter Timeline refined

Earnings Call Themes & Trends

TopicQ2 2024 (Previous Mentions)Q3 2024Q4 2024 (Current Period)Trend
Regulatory/BLAPositive pre-BLA; rolling BLA plan; explore full vs accelerated paths Filing strategy: cardiomyopathy first, combine HOPE-3 cohorts post-approval for label expansion BLA accepted; Priority Review; PDUFA 8/31/2025; preparing for potential AdCom and PLI De-risking, advancing
ManufacturingSan Diego facility operational; comparability affirmed by FDA Scale-up plans; modular clean rooms; shelf-life aids stockpiling Lease expansion +25k sq ft; capacity to 2,000–3,000 patients by mid-2026 Capacity increasing
Commercial/NS PharmaFocus on launch readiness; US milestones (~$700M) NS team ~125; rapid adoption expected; 100 OLE transfers at launch Active payer engagement; 50–60% of US DMD eligible; revenue share 30–50% Launch preparation intensifying
ReimbursementN/APositive payer discussions anticipated Payer surveys favorable; reimbursement aligned with exon skippers Positive momentum
Europe/EMAPursuing partners; late-stage EU discussions Term sheet with NS; planning global supply EMA Orphan + ATMP; EMA meeting Q2 2025; EU definitive agreement under negotiation Regulatory progress
Safety vs Gene TherapyN/AN/A>700 infusions; mild manageable reactions; simpler infusion vs gene therapy Safety profile reiterated
HOPE-3 / Label ExpansionCohorts fully enrolled; top-line planned Q4 2024 Combine cohorts post-approval to expand skeletal muscle label FDA has not requested HOPE-3 for current BLA; may support ex-US authorizations Label expansion plan clarified
StealthX Exosome ProgramPreclinical progress; manufacturing scale-up; Project NextGen IND planning for therapeutic exosome; NIAID-funded Phase 1 vaccine Manufacturing underway; regulatory approval Q2 2025; first-in-human expected thereafter Advancing

Management Commentary

  • “We continue to work diligently towards our August 31, 2025 action date…preparing for pre-approval licensure inspection and preparing for potential commercial launch with our partner Nippon Shinyaku” .
  • “Our BLA is supported with data from…HOPE-2 placebo-controlled trial and the HOPE-2 open label extension (OLE) trial compared to natural history data… The results demonstrated statistically significant and clinically relevant improvements in cardiac function up to three-years after treatment” .
  • “Our current GMP compliant facility…can support approximately 250 to 500 patients per year… we have leased an additional 25,000 square feet…taking our manufacturing capacity to approximately 2,000 to 3,000 patients per year once completed” .
  • “If we receive FDA approval, we will be slated to receive an additional $80 million milestone payment… and… a priority review voucher… These non-dilutive cash infusions could total well over $200 million” .

Q&A Highlights

  • Reimbursement posture: Top 5 payer surveys were favorable; deramiocel addresses an unmet need (DMD cardiomyopathy) and is expected to be reimbursed consistent with exon skippers .
  • AdCom and HOPE-3: No indication yet of AdCom; FDA has not requested HOPE-3 for the current BLA; HOPE-3 intended for post-approval label expansion and ex-US authorizations .
  • Manufacturing scale-up cost/timing: New clean room build costs characterized as “very reasonable”; plans underway with prior clean room built for “just under a couple of million dollars” .
  • Safety and administration: ~700 infusions to date; mild hypersensitivity mitigated with premedication; infusion center model; markedly simpler care paradigm vs gene therapies .
  • Patient targeting: Greatest long-term stabilization seen when baseline LVEF ≥45%; early treatment emphasized; ~50–60% of US DMD population (~7,500) estimated eligible at launch .

Estimates Context

  • Q4 2024 beat: Revenue $11.13M vs $9.87M est; EPS -$0.16 vs -$0.17 est (beats). Q3 miss: Revenue $2.26M vs $3.57M est; EPS -$0.38 vs -$0.358 est (miss). Q2 beat: Revenue $3.97M vs $3.66M est; EPS -$0.35 vs -$0.356 est (beat). Estimates marked with * retrieved from S&P Global. Actuals cited above .
  • Implications: Q4 momentum and milestone recognition suggest upward adjustments to near-term revenue expectations; however, OpEx growth tied to commercialization and manufacturing scale warrants careful monitoring of operating loss trajectory .

Key Takeaways for Investors

  • The regulatory path is de-risking: Priority Review accepted, PDUFA 8/31/2025, and FDA clarity that HOPE-3 is not required for cardiomyopathy approval materially reduces near-term binary risk .
  • Commercial readiness is maturing: NS Pharma’s established Duchenne infrastructure (~125 personnel) plus payer receptivity and planned early OLE transitions (~100 patients) set up a potentially strong launch .
  • Capacity expansion addresses supply risk: Near-term capacity (250–500 pts/yr) bridges to mid-2026 expansion (2,000–3,000 pts/yr), aligning manufacturing scale with anticipated adoption .
  • Balance sheet can support launch and pipeline: $151.5M year-end cash, $10M post-year milestone, expected $80M on approval, and potential PRV monetization provide strategic flexibility without immediate dilution .
  • Narrative catalyst: First-in-class therapy for DMD cardiomyopathy where no approved options exist; EMA Orphan + ATMP status broadens ex-US optionality and potential exclusivity .
  • Trading implications (near term): Watch for AdCom decision, PLI inspection outcomes, and EMA interactions; Q4 beat may support sentiment, while elevated OpEx highlights execution discipline needed ahead of launch .
  • Medium-term thesis: Label expansion (skeletal muscle, Becker MD) and exosome platform (Project NextGen vaccine; targeted delivery) provide multi-pronged optionality beyond initial cardiomyopathy indication .

Additional Relevant Press Releases (Q4 2024)

  • Rolling BLA submission initiated; plan to complete by YE2024; Priority Review eligibility noted .
  • Positive 3-year HOPE-2 OLE data showing cardiac and skeletal muscle function preservation; supports BLA .
  • EMA granted Orphan Drug and ATMP designations for deramiocel (DMD), adding regulatory advantages and potential exclusivity .

Appendix: Prior Two Quarters’ Earnings Reference

  • Q3 2024: Revenue $2.26M; diluted EPS -$0.38; cash $85.0M; term sheet with NS for Europe; rolling BLA underway .
  • Q2 2024: Revenue $3.97M; diluted EPS -$0.35; cash $29.5M; positive pre-BLA meeting; HOPE-3 fully enrolled .

Citations: All document-based data and quotes are cited as [doc_id:chunk_idx]. Estimates marked with * retrieved from S&P Global.