Sign in

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

CT

CAPRICOR THERAPEUTICS, INC. (CAPR)·Q1 2025 Earnings Summary

Executive Summary

  • Q1 2025 printed zero revenue and a wider net loss of $24.4M ($0.53 EPS) as prior milestone revenues were fully recognized in FY 2024; cash, cash equivalents and marketable securities were $144.8M, with runway guided into 2027 .
  • The quarter missed Wall Street on both revenue ($0 vs $3.16M est*) and EPS (-$0.53 vs -$0.32 est*); Q4 2024 had been a revenue and EPS beat (actual $11.13M vs $9.87M est*, EPS -$0.16 vs -$0.17 est*) .
  • Regulatory trajectory strengthened: FDA mid-cycle review identified no significant deficiencies; advisory committee confirmed ahead of Aug 31, 2025 PDUFA, and pre-licensing inspection (PLI) scheduled in the coming weeks—key stock catalysts into summer .
  • Commercial readiness advanced: appointment of CMO Michael Binks (Pfizer/GSK veteran), expansion of GMP space in San Diego, and NS Pharma’s U.S. team (125 FTEs) preparing for launch; management reiterated a potential $80M milestone and Priority Review Voucher monetization post-approval .

What Went Well and What Went Wrong

What Went Well

  • FDA review milestones on-track: “no significant deficiencies” at mid-cycle; AdCom confirmed; PDUFA Aug 31, 2025—“We remain on track” .
  • Strengthened leadership: “Appointed Michael Binks, M.D., as Chief Medical Officer,” bringing deep rare disease experience to guide medical affairs and commercialization .
  • Cash runway into 2027 with strategic optionality: ~$145M cash/marketable securities; management highlighted potential non-dilutive cash from $80M milestone + PRV sale “well over $200M” .

Quote: “We continue to have active dialogue with the FDA... and we remain on track with our PDUFA target action date of August 31, 2025.”

What Went Wrong

  • Non-commercial quarter with no recognized revenue: Q1 revenue was $0 as FY 2024 fully recognized the $50M upfront/milestones—optics drove a larger GAAP loss .
  • Operating expense growth ahead of launch readiness: OpEx rose to ~$25.0M from $15.2M YoY; net loss widened to $24.4M vs $9.8M in Q1 2024 .
  • Consensus misses: Revenue miss ($0 vs $3.16M est*) and EPS miss (-$0.53 vs -$0.32 est*) in Q1; underscores timing mismatch between spend ramp and revenue recognition while awaiting approval .

Financial Results

Quarterly Results (oldest → newest)

MetricQ3 2024Q4 2024Q1 2025
Revenue ($USD)$2.262M $11.131M $0
Diluted EPS ($USD)-$0.38 -$0.16 -$0.53
Total Operating Expenses ($USD)$15.273M $18.828M $24.983M
Net Loss ($USD)-$12.557M -$7.117M -$24.392M

Year-over-Year (Q1 comparison)

MetricQ1 2024Q1 2025
Revenue ($USD)$4.907M $0
Diluted EPS ($USD)-$0.31 -$0.53
Total Operating Expenses ($USD)$15.173M $24.983M
Net Loss ($USD)-$9.794M -$24.392M

Estimates vs Actuals

MetricQ3 2024 Est*Q3 2024 ActualQ4 2024 Est*Q4 2024 ActualQ1 2025 Est*Q1 2025 Actual
Revenue ($USD)$3.568M*$2.262M $9.870M*$11.131M $3.159M*$0
Primary EPS ($USD)-$0.358*-$0.38 -$0.17*-$0.16 -$0.32*-$0.53

Estimates retrieved from S&P Global.*

KPIs and Balance Highlights

KPIQ3 2024Q4 2024Q1 2025
Cash, cash equivalents & marketable securities ($USD)~$85M (as of Sep 30, 2024) $151.5M (Dec 31, 2024) $144.8M (Mar 31, 2025)
Financial runwayInto 2027 Into 2027 Into 2027
U.S. milestone receipts—$10M triggered Dec 2024 $10M received Jan 2025

Note: Margin metrics are not meaningful in Q1 due to $0 revenue; company is pre-commercial with milestone/licensing revenue mix .

Guidance Changes

MetricPeriodPrevious GuidanceCurrent GuidanceChange
Cash runwayThrough 2027Into 2027 (Q4 2024) Into 2027 (Q1 2025) Maintained
Regulatory timeline2025Priority Review; PDUFA H2’25 (Q3 2024) PDUFA Aug 31, 2025; AdCom confirmed; mid-cycle “no significant deficiencies” Clarified/strengthened
Manufacturing capacity2025–2026San Diego GMP operational; planning expansion (Q4 2024) Lease amended for added GMP space; expansion underway, targeted mid–late 2026 Raised capacity outlook
Commercial readiness2025NS Pharma ~125 FTE preparing; payer feedback positive (Q4 2024) Continued launch prep; medical leadership strengthened (CMO) Enhanced execution plans

Earnings Call Themes & Trends

TopicPrevious Mentions (Q-2: Q3 2024; Q-1: Q4 2024)Current Period (Q1 2025)Trend
BLA/RegulatoryFiling for full approval in cardiomyopathy; Priority Review expected Mid-cycle “no significant deficiencies”; AdCom confirmed; PDUFA 8/31/25 Improving momentum
AdComPrepared if needed; collaborative FDA interactions AdCom intent confirmed; multiple mock AdComs completed Firming to event
Manufacturing/PLISan Diego GMP operational; expansion plan to 2–3k patients; PLI set for Q2 PLI “coming up this quarter within the next few weeks” Execution nearing
Commercial readiness & reimbursementNS Pharma 125 FTE; positive payer surveys; initial 100 OLE patients to transition Continued coordination; >100 patients expected to transition post-approval Building readiness
Europe strategyTerm sheet with NS; evaluating EMA path; designations obtained Negotiations ongoing; Capricor engaging directly with EMA; decisioning on best path Evolving approach
StealthX vaccine (Project NextGen)Manufacturing underway; NIAID to conduct Phase 1 Phase 1 planned Q3 2025 pending NIAID clearance Advancing
HOPE-3 strategyCombine cohorts for post-approval label expansion; potential ex-U.S. Fully enrolled in U.S.; potential international expansion; timing contingent on regulatory outcomes Optimization
PRV/MilestonesPriority Review implies PRV; potential $1.5B EU milestones if executed Plan to sell PRV; $80M approval milestone from NS Monetization clarity

Management Commentary

  • “Deramiocel is the only therapeutic that has been shown to be effective in slowing the decline in ejection fraction... We are asking for approval for a therapy that has been shown to be generally safe and effective for the treatment of DMD cardiomyopathy” — Linda Marbán, CEO .
  • “We aim to have over 100 patients transition from clinical to commercial products following potential BLA approval” .
  • “Our San Diego GMP facility is fully staffed and operational and is currently producing doses... expansion to be operational mid-to late 2026” .
  • “Our cash balance totals approximately $145 million... If we receive FDA approval, we will be slated to receive an $80 million milestone payment... and a Priority Review Voucher... non-dilutive cash infusions could potentially total well over $200 million” .

Q&A Highlights

  • Regulatory/PLI cadence: Pre-licensing inspection “coming up this quarter within the next few weeks”; multiple mock AdComs completed; mid-cycle flagged no substantive issues .
  • Europe commercialization: Active EMA engagement by Capricor; NS negotiations extended; considering alternatives to maximize economics .
  • Endpoint framing: LV ejection fraction treated as an outcome measure in rare pediatric disease context; strong real-world natural history comparator supports efficacy .
  • Label/use and safety profile: Intended open use alongside exon skippers/gene therapies; infusion center administration with mitigated hypersensitivity; no complex gene therapy care team required .
  • PRV monetization: Plan to sell PRV post-approval to strengthen balance sheet; approval triggers $80M NS milestone .

Estimates Context

  • Q1 2025: Miss on revenue ($0 vs $3.16M est*) and Miss on EPS (-$0.53 vs -$0.32 est*), driven by the completion of revenue recognition on prior milestones in FY 2024 and continued OpEx ramp for launch readiness .
  • Q4 2024: Beat on revenue ($11.13M vs $9.87M est*) and slight Beat on EPS (-$0.16 vs -$0.17 est*), reflecting milestone recognition timing late in 2024 .
  • Q3 2024: Misses on both revenue and EPS (actual $2.26M and -$0.38 vs $3.57M and -$0.358 est*), consistent with ratable milestone accounting .

Estimates retrieved from S&P Global.*

Key Takeaways for Investors

  • Regulatory de-risking: Mid-cycle “no significant deficiencies,” AdCom confirmed, and PLI imminent—these create tangible catalysts into June–August; headline risk is AdCom tone/outcome .
  • Pre-commercial spend vs optics: Q1 losses reflect launch build and non-recognition of milestones; watch for AdCom/PDUFA to pivot narrative from GAAP losses to commercialization milestones .
  • Commercial readiness: NS Pharma’s infrastructure and >100 OLE patient transitions should support a smoother ramp; payer feedback has been favorable given the cardiomyopathy unmet need .
  • Manufacturing scalability: Current capacity to support early demand, with expansion to 2–3k patients by mid–late 2026; PLI outcome is a critical gating event .
  • Balance sheet resilience: ~$145M cash and potential >$200M non-dilutive inflows (PRV sale + NS milestone) provide flexibility to invest in label expansion (HOPE-3, Becker) and exosome pipeline .
  • European strategy optionality: Active EMA engagement alongside NS negotiations could optimize ex-U.S. economics and timelines; monitor for definitive agreement terms .
  • Trading setup: AdCom scheduling disclosure and briefing documents will likely drive volatility; a constructive panel and clean PLI would be material positives heading into the Aug 31 PDUFA .