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X4 Pharmaceuticals, Inc (XFOR)·Q4 2024 Earnings Summary

Executive Summary

  • Q4 2024 delivered accelerating XOLREMDI ramp and operational focus: product revenue rose to $1.43M (up >150% Q/Q) as the U.S. WHIM launch progressed; pro forma liquidity extended into 1H 2026 following the Norgine upfront and cost restructuring .
  • Clinical execution advanced: the Phase 3 4WARD CN trial is activated at ~90% of sites; protocol was refined with FDA/EMA input (focus on ANC <1,000 and a uniform ≥500 cells/μL ANC increase component), with full enrollment expected in 3Q–4Q 2025 and top-line data in 2H 2026 .
  • Versus S&P Global consensus, Q4 revenue beat ($1.43M vs $1.06M*) while S&P “Primary EPS” missed (-6.00 vs -4.73*), noting methodology/share-base differences vs GAAP diluted EPS (-$0.20) reported by the company .
  • Catalysts: protocol refinement and enrollment progress in CN, EMA MAA validation, ex-U.S. partnerships (Norgine upfront €28.5M; taiba rare in MENA), and restructuring driving $30–35M in annualized savings support the path to 4WARD readout and WHIM uptake in 2025–2026 .

What Went Well and What Went Wrong

What Went Well

  • Commercial uptake improved: net product revenue reached $1.43M in Q4 (vs $0.56M in Q3), reflecting increasing prescriber engagement and patient services; management cited payer receptivity and no discounting through specialty pharmacy .
  • Strategic and financial positioning strengthened: Norgine license delivered €28.5M upfront and up to €226M in milestones (double‑digit tiered royalties), while restructuring reduces annual spend by $30–35M, extending cash runway into 1H 2026 pro forma .
  • CN program de-risking: refined 4WARD protocol (moderate/severe ANC, uniform ANC endpoint) and ~90% site activation bolster confidence in achieving infection benefit and operational timelines (full enrollment 3Q–4Q 2025; top‑line 2H 2026) .

What Went Wrong

  • Losses widened with scale-up: Q4 net loss was -$39.8M vs -$19.1M LY, driven by higher R&D ($21.7M) and SG&A ($15.1M) as commercialization and CN Phase 3 advanced .
  • Enrollment timing modestly pushed: guidance moved from “mid-2025 fully enrolled” (Q3) to “3Q–4Q 2025,” reflecting operational prudence and protocol refinements, though management does not expect material impact from excluding mild ANC patients .
  • EPS optics vs S&P methodology: S&P “Primary EPS” showed a larger loss than consensus (-6.00 vs -4.73*), diverging from company GAAP diluted EPS (-$0.20); investors should note metric methodology and share-base differences .

Financial Results

Revenue, EPS, and margins vs prior periods and consensus

MetricQ4 2023Q2 2024Q3 2024Q4 2024Q4 2024 Cons. Est.
Product Revenue, net ($USD)$0 $0.563M $0.560M $1.434M $1.0647M*
Cost of Revenue ($USD)$0 $0.268M $0.227M $0.302M —
Gross Profit ($USD)$0 $0.295M (calc) $0.333M (calc) $1.132M (calc) —
Gross Margin %—52.4% (calc) 59.5% (calc) 79.0% (calc) —
GAAP Diluted EPS ($)-$0.10 $0.45 -$0.18 -$0.20 —
S&P Primary EPS (actual/est)———-6.00*-4.73*

Notes: Gross Profit and Gross Margin % are computed from reported revenue and cost of revenue (citations reference source figures). Asterisked values are from S&P Global. Values retrieved from S&P Global.

Operating expenses and cash

MetricQ4 2023Q2 2024Q3 2024Q4 2024
R&D Expense ($USD)$15.3M $20.9M $19.2M $21.7M
SG&A Expense ($USD)$9.9M $13.3M $15.7M $15.1M
Net Loss ($USD)-$19.1M $90.8M (PRV-driven) -$36.7M -$39.8M
Cash, Equivalents & Securities (period-end)$147.3M (12/31/23) $210.6M (6/30/24) $178.2M Assets; $135.8M cash-like (9/30/24) $102.8M (12/31/24)

KPI snapshot

KPIQ2 2024Q3 2024Q4 2024 / Early 2025
4WARD Sites ActivatedInitiated ~40% initiated ~90% activated
4WARD Enrollment Guidance—Fully enroll mid-2025 Fully enroll 3Q–4Q 2025; top-line 2H 2026
EMA MAA (WHIM)Plan to submit by early 2025 Expect submit by early 2025 MAA validated; decision 1H 2026
Ex-U.S. Partnerships——Norgine (€28.5M upfront; milestones/royalties) ; taiba rare (MENA)

Guidance Changes

MetricPeriodPrevious GuidanceCurrent GuidanceChange
4WARD full enrollmentCN Phase 3Mid-2025 3Q–4Q 2025 Lowered (timing pushed modestly)
4WARD top-lineCN Phase 3Not specified2H 2026 New specific timeline
Site activationCN Phase 3~40% sites initiated (Nov-24) ~90% activated (Mar-25) Raised
Cash runwayCorporateInto late 2025 (Sep-24) Into 1H 2026 (pro forma with Norgine upfront & restructuring) Raised
Annualized spendingCorporate—Decrease by $30–35M New (cost reduction)
U.S. priceXOLREMDI—~7% price increase in early 2025 New
Revenue/OpEx guidance2025Not providedNot providing 2025 sales forecast Maintained (no formal guidance)

Earnings Call Themes & Trends

TopicQ2 2024 (Q-2)Q3 2024 (Q-1)Q4 2024 (Current)Trend
CN Phase 3 design/execution4WARD initiated On track; ~40% sites; enroll mid-2025 ~90% sites; protocol refined (ANC<1,000; uniform ANC endpoint); enroll 3Q–4Q 2025; top-line 2H 2026 Improving operational readiness; timeline clarified
WHIM U.S. launchFirst sales; patient services active Growing awareness and screening Revenue acceleration; payer receptivity; no discounting; 7% price increase Early uptake building
Regulatory (EU)Plan EMA submission by early 2025 Expect submission by early 2025 MAA validated; decision 1H 2026 Progressing
Ex-U.S. strategy——Norgine EU/ANZ deal; taiba rare (MENA) Expanding global reach
R&D de-riskingPositive CN Phase 2 interim Positive CN Phase 2 full data Protocol refined with FDA/EMA input Heightened confidence
Cost discipline/cashPRV sale bolsters cash Runway into late 2025 Restructuring; runway into 1H 2026 (pro forma) Stronger runway

Management Commentary

  • “2024 was a transformative year… With the U.S. approval and launch of our first product, XOLREMDI… we are now a fully integrated company.” – CEO Paula Ragan .
  • “We expect full trial enrollment in the third or fourth quarter of this year and top-line trial data in the second half of 2026.” – CEO on 4WARD .
  • Patient impact: testimonials of “normally functioning immune system… significant improvement in skin infections” and “near‑normal ANC levels,” underscoring clinical benefit resonance in WHIM .
  • “We ended 2024 with just under $103 million… we believe we have sufficient funds to support company operations into the first half of 2026 [pro forma].” – CFO Adam Mostafa .
  • “We did take a slight price increase… 7% price increase” for XOLREMDI into 2025; “we’re not engaging in discounting at this time.” – Commercial leadership .

Q&A Highlights

  • 4WARD protocol tightening and enrollment: FDA/EMA alignment to focus on ANC<1,000; management does not expect material enrollment friction; timeline prudently shifted to 3Q–4Q 2025 .
  • Channel dynamics: inventory reflected specialty pharmacy stocking in Q4; no discernible discounting; payer coverage described as quick .
  • Pricing: ~7% price increase executed in early 2025 .
  • Patient metrics: management declined to disclose patient counts; described growing demand and adherence above typical daily oral benchmarks, supported by patient services and specialty pharmacy partnership .
  • Ex-U.S. agreements: Norgine/taiba arrangements structured without tight registration timing clauses; next major regulatory milestone is potential EU approval in 1H 2026 .

Estimates Context

  • Revenue: Q4 actual $1.434M vs S&P Global consensus $1.065M* — a clear beat as XOLREMDI adoption increased with broader prescriber engagement and patient support programs .
  • EPS: S&P “Primary EPS” actual -6.00 vs -4.73* consensus — a miss; note this metric differs from the company’s GAAP diluted EPS (-$0.20) due to methodology and share-base normalization used by S&P Global/IBES, particularly in a year with unusual items (e.g., PRV sale earlier in 2024) .
  • Forward adjustments: We expect Street models to modestly raise near-term WHIM revenue trajectories (given Q/Q acceleration) while maintaining conservative opex/cash burn assumptions ahead of 4WARD top-line in 2H 2026.
    Asterisked values are from S&P Global. Values retrieved from S&P Global.

Key Takeaways for Investors

  • XOLREMDI launch is gaining traction with Q4 revenue acceleration and supportive payer dynamics; no discounting and a 7% price increase should aid 2025 net revenue trajectory .
  • CN program remains the core value driver: ~90% site activation, protocol refinement with regulators, and clarified timelines set up a high‑conviction 2H 2026 readout; enrollment push to 3Q–4Q 2025 is prudent rather than problematic .
  • Balance sheet support from Norgine upfront and restructuring should bridge to key catalysts; runway into 1H 2026 (pro forma) reduces near-term financing overhang risk .
  • EU (WHIM) regulatory path validated; potential approval in 1H 2026 plus MENA partnership expands optionality beyond U.S. .
  • Watch for 2025 WHIM demand indicators (patient identification, refill adherence) and CN 4WARD screening/activation updates as near-term sentiment drivers .
  • Valuation sensitivity centers on CN success probability, U.S. WHIM ramp pacing, and capital needs if timelines slip; management messaging on payer access and adherence is incrementally positive .

Appendix: Source Documents

  • Q4 2024 8-K (Item 2.02) and financial tables .
  • Q4 2024 earnings press release .
  • Q4 2024 earnings call transcript (prepared remarks and Q&A) -.
  • Prior quarters: Q3 2024 PR/8-K - -; Q2 2024 PR -.
  • Early 2025 partner/EMA/restructuring PRs .