Sign in

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

IP

Inozyme Pharma, Inc. (INZY)·Q1 2024 Earnings Summary

Executive Summary

  • Q1 2024 delivered clean execution: cash/investments of $166.2M with runway into Q4 2025, while OpEx rose as clinical programs advanced; no revenue recognized, net loss was $23.3M or $0.38/share (vs. $17.4M or $0.40/share in Q1 2023) .
  • Clinical catalysts remain stacked: interim ENERGY‑1 (ENPP1 infants) 2H 2024, SEAPORT‑1 (ESKD/hemodialysis) interim Q4 2024, ENERGY‑3 (ENPP1 pediatrics) topline mid‑2025; ABCC6 pediatric pivotal planned for Q1 2025 (regulatory review/funding dependent) .
  • Strategy pivot crystallized in ABCC6: focus on pediatric population with high stroke risk; adult Phase 1/2 showed favorable safety with vascular and ophthalmic signals, supporting path to a pivotal pediatric trial and potential AA/CMA using stroke‑predictive imaging endpoints .
  • Street estimates were unavailable via S&P Global for Q1 2024, so beat/miss analysis cannot be provided; INZY is pre‑revenue and results hinge on OpEx/clinical milestones rather than top‑line/EPS beats (S&P Global estimates unavailable).

What Went Well and What Went Wrong

What Went Well

  • Adult ABCC6 Phase 1/2 met study objectives: no serious/severe AEs; cIMT stabilization/decrease (7/8 evaluable), increased choroidal thickness (7/8), and improved PROs (C‑GIC 9/9; P‑GIC 7/9), supporting biologic activity in vascular/retinal disease .
  • ENPP1 adult program durability: favorable safety/immunogenicity maintained through 48 weeks; biomarkers (↓FGF‑23; ↑BSAP; ↓CTX) and once‑weekly cohort PK/PPi support weekly dosing across future trials .
  • Balance sheet strength maintained: $166.2M in cash/investments as of 3/31/24; runway into Q4 2025 reaffirmed, de‑risking near‑term milestone execution .

What Went Wrong

  • Higher R&D spend drove wider loss: R&D rose to $19.1M (+61% YoY), lifting total OpEx to $24.3M and net loss to $23.3M; this reflects accelerated development, though no explicit Q1 attribution was provided (FY23 drivers included CMC and clinical costs) .
  • No earnings call transcript for Q1 2024; investor color instead came from the April 8 special call (topline data briefing), limiting quarter‑specific Q&A on OpEx cadence/runway sensitivities .
  • Estimate benchmarking absent: S&P Global consensus for revenue/EPS not available for Q1 2024, precluding standard beat/miss framing (S&P Global estimates unavailable).

Financial Results

P&L snapshot (USD Millions, except per share)

MetricQ1 2023Q3 2023Q1 2024
R&D Expense ($M)$11.86 $13.34 $19.11
G&A Expense ($M)$6.51 $4.73 $5.23
Total Operating Expenses ($M)$18.37 $18.07 $24.35
Net Loss ($M)$17.40 $16.64 $23.35
Diluted EPS ($)$(0.40) $(0.29) $(0.38)

Balance sheet and liquidity (USD Millions)

MetricSep 30, 2023Dec 31, 2023Mar 31, 2024
Cash, Cash Equivalents & Investments$192.44 $188.59 $166.15
Total Assets$205.70 $200.85 $176.94
Total Liabilities$45.49 $60.37 $58.11
Stockholders’ Equity$160.21 $140.48 $118.84

Notes:

  • No product revenue reported; Statements of Operations list only operating expenses with loss from operations equal to total OpEx (implying no recognized revenue) .
  • Runway: “into Q4 2025” maintained on both 12/31/23 and 3/31/24 snapshots .

Guidance Changes

MetricPeriodPrevious GuidanceCurrent GuidanceChange
Cash runwayAs of 12/31/23 → forwardFund operations into Q4 2025 Fund operations into Q4 2025 Maintained
ENERGY‑2 (ENPP1 infants)Start2H 2024 2H 2024 Maintained
ENERGY‑1 (ENPP1 infants)Interim data2H 2024 2H 2024 Maintained
ENERGY‑3 (ENPP1 pediatrics)ToplineMid‑2025 Mid‑2025 Maintained
ABCC6 pivotal (pediatric)StartQ1 2025 (Phase 3; earlier disclosures didn’t specify pediatrics) Q1 2025, pediatric focus (regulatory/funding dependent) Maintained; scope clarified to pediatric
SEAPORT‑1 (ESKD/hemodialysis)Interim dataQ4 2024 Q4 2024 Maintained

Earnings Call Themes & Trends

TopicPrevious Mentions (Q3 2023 and FY 2023)Current Period (Q1 2024)Trend
ENPP1 regulatory path (PPI endpoint)PPI primary (US), co‑primary (EU) in ENERGY‑3; clear path to filings with robust PPi/clinical data Adult data supportive; weekly dosing cohort validates PK/PPi; pediatric pivotal enrollment pace emphasized ; mid‑2025 topline reiterated Consistent; execution on plan
ABCC6 strategy & endpointsPhase 3 planning noted; timelines evolving across disclosures Adult Phase 1/2 showed vascular/retinal signals (cIMT, choroid, PROs); pediatric focus with potential AA/CMA based on stroke‑predictive imaging; pivotal planned Q1 2025 Sharpened pediatric focus; de‑risked by adult data
Pediatric ABCC6 burdenNoted disease burden; framing buildingNatural history shows 7/12 with stroke or at‑risk; severe morbidity early in life; robust patient‑finding via claims and KOLs Elevated urgency; clearer prevalence signal
Calciphylaxis programASN/ECTS data linked low PPi to outcomes; SEAPORT‑1 initiation plan SEAPORT‑1 underway; interim in Q4 2024; approach to subsequent outcomes study shaping On track
Cash runway/capitalInto Q4 2025 reiterated (Q3 and FY) Into Q4 2025 reaffirmed Stable

Management Commentary

  • “We were extremely pleased to see preliminary evidence of improved vascular health with INZ‑701 treatment in adults with ABCC6 Deficiency… We look forward to working with regulators to establish a path to approval for our ABCC6 Deficiency program.” — CEO Douglas A. Treco .
  • “Phase I/II trial of INZ‑701 in adults with ABCC6 Deficiency successfully met all study objectives… improvements in vascular pathology, visual function and patient‑reported outcomes” — Special Call remarks .
  • “Once‑weekly dosing cohort showed the expected PK profile and supports the use of weekly dosing… favorable safety and immunogenicity with most patients essentially antibody‑free at week 48.” — Special Call .

Q&A Highlights

  • ABCC6 pediatric pivotal/AA path: Company aims for a randomized trial using an imaging metric predictive of ischemic stroke for AA/CMA, then transition all to drug and follow for outcomes; a composite clinical endpoint route is also under evaluation to potentially enable full approval without AA .
  • Dosing strategy: Weekly dosing preferred; plan to develop flat adult dosing; ENERGY‑3 uses 2.4 mg/kg weekly in pediatrics given faster metabolism .
  • Visual endpoints: VFQ‑25 captured earlier functional impact vs BCVA; BCVA improved in 2/7 with no declines; choroidal thickness increase correlates with VFQ‑25 improvements; endpoint selection to be discussed with regulators .
  • Pediatric prevalence and identification: Multimodal efforts (claims analysis, KOLs, advocacy) identified ~1,300 likely US pediatric ABCC6 patients across four symptom clusters; early access program being set up to gather data .

Estimates Context

  • S&P Global consensus was unavailable for Q1 2024 (EPS and revenue), so we cannot provide beat/miss analysis for the quarter (S&P Global estimates unavailable).
  • As a pre‑revenue company, quarterly comps hinge on OpEx cadence and clinical milestones rather than top‑line variances; Q1 showed expected OpEx step‑up consistent with advancing programs .

Key Takeaways for Investors

  • Near‑term catalysts intact: ENERGY‑1 interim (2H 2024), SEAPORT‑1 interim (Q4 2024), ENERGY‑3 topline (mid‑2025), ABCC6 pediatric pivotal start (Q1 2025) — potential multi‑program de‑risking over next 6–12 months .
  • ABCC6 narrative improved: adult signals across cIMT, choroid thickness, and PROs plus pediatric natural history data strengthen the case for a pediatric‑first pivotal with imaging‑based AA/CMA strategy .
  • ENPP1 execution: once‑weekly dosing supported; biomarker durability and PROs sustained through 48 weeks, validating the platform and simplifying real‑world dosing .
  • Runway through key readouts: Cash/investments of $166.2M and runway into Q4 2025 provide line‑of‑sight through 2024/25 data events, reducing near‑term financing overhang .
  • Watch OpEx trajectory: R&D step‑up reflects pipeline momentum; continued discipline will matter as pivotal studies initiate and manufacturing scales .
  • Regulatory inflection points: FDA/EMA engagements on ABCC6 endpoints and pediatric pivotal design are potential stock catalysts; clarity on composite endpoints vs AA could influence timelines and perceived risk .
  • Trading setup: With multiple 2H24/2025 catalysts and preserved runway, shares may trade on clinical update cadence; downside risks center on trial execution, regulatory endpoint acceptance, and capital needs beyond Q4 2025 .

Appendix: Additional Quantitative Disclosures

Selected per‑share and share count

MetricQ1 2023Q3 2023Q1 2024
Weighted‑avg shares (basic/diluted)43,720,578 56,758,395 61,772,279
Net loss per share ($)$(0.40) $(0.29) $(0.38)