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X4 Pharmaceuticals, Inc (XFOR)·Q3 2024 Earnings Summary

Executive Summary

  • Q3 2024: First full quarter post-U.S. launch of XOLREMDI; net product revenue was $0.56M, essentially flat vs Q2 ($0.56M), with a net loss of $36.7M (EPS $(0.18)); YoY revenue improved from $0, but expenses and warrant remeasurement drove a larger loss YoY .
  • CN (chronic neutropenia) program advanced: Final Phase 2 data showed durable ANC increases and clinician-driven G-CSF dose reductions (mean −52% at Month 3; −70% at Month 6) while keeping mean ANC in normal range—bolstering confidence in the pivotal Phase 3 4WARD trial .
  • Commercial execution for WHIM: 3,400 targeted HCPs engaged; market research indicates >75% awareness of WHIM and >80% likely prescribers for XOLREMDI; >150M lives covered—foundation for 2025 ramp as disease education converts to screening and treatment .
  • Operational outlook maintained: 4WARD full enrollment still expected mid-2025; EMA MAA for WHIM “by early 2025”; cash runway into late 2025 (ex-FY25 sales) remains intact—no sales guidance provided yet .

What Went Well and What Went Wrong

What Went Well

  • CN Phase 2 success strengthens Phase 3 outlook: Mavorixafor monotherapy durably raised mean ANC into the normal range (Month 3 and 6) and enabled meaningful G-CSF cutbacks in combination cohorts (mean −52% at Month 3; −70% at Month 6) with ANC maintained in normal range; three participants discontinued G-CSF entirely by Month 6 .
  • WHIM launch foundations: X4 engaged all 3,400 targeted immunologists/hematologists; HCP awareness >75% and >80% of surveyed likely prescribers would consider XOLREMDI; >150M lives covered—management emphasized these are building blocks for acceleration in 2025 .
  • Management confidence and tone: “We could not be more pleased with the results from the six-month Phase 2 study of mavorixafor in CN…[they] increase our confidence in a positive outcome for our ongoing pivotal Phase 3 4WARD trial,” (CEO Paula Ragan) .

What Went Wrong

  • Revenue flat QoQ post-initial stocking: Net product revenue was $0.56M in both Q2 and Q3; management noted Q2 included some specialty pharmacy stocking as is typical at launch, framing Q3 as early in the awareness-to-screening conversion cycle .
  • Higher SG&A YoY as launch investments scale: SG&A rose to $15.7M from $8.1M a year ago; R&D remained elevated at $19.2M (vs $19.1M YoY) to support the CN program and other initiatives .
  • GAAP volatility from non-cash items: Q3 net loss of $36.7M included a $1.9M non-cash loss from warrant remeasurement; prior-year quarter benefited from a $25.2M non-cash gain—masking underlying operating progress .

Financial Results

P&L Summary (USD Millions, except per-share)

MetricQ3 2023Q1 2024Q2 2024Q3 2024
Net Product Revenue$0.00 $0.00 (no prior-period product revenue) $0.56 $0.56
Cost of Revenue$0.00 $0.00 (no prior-period cost of revenue) $0.27 $0.23
R&D$19.08 $19.85 $20.91 $19.17
SG&A$8.13 $17.44 $13.28 $15.66
Net Income (Loss)$(2.31) $(51.77) $90.83 $(36.70)
Diluted EPS$(0.01) $(0.26) $0.45 $(0.18)

Notes: Q2 2024 net income benefited from a $105M PRV sale and a $20.2M non-cash warrant gain .

Balance Sheet and Liquidity

MetricQ1 2024Q2 2024Q3 2024
Cash, Cash Equivalents, Restricted Cash, and ST Marketable Securities$81.6 $169.5 $135.8
Cash Runway (Mgmt view)into 2025 into late 2025 into late 2025 (ex-future XOLREMDI revenue)

KPIs and Operating Metrics

KPIQ3 2024
Targeted HCPs engaged (initial cohort)3,400 engaged
HCP awareness of WHIM>75%
Likely prescribers considering XOLREMDI>80%
Covered lives>150M
4WARD regulatory/site progress~85% country approvals; ~40% sites initiated
Cost of revenue detailIncludes ~$0.2M in license costs (royalties/milestones amortized)

Guidance Changes

MetricPeriodPrevious GuidanceCurrent GuidanceChange
4WARD CN Phase 3 full enrollmentMid-2025On track for mid-2025 On track for mid-2025 Maintained
EMA MAA submission for WHIMBy early 2025By early 2025 By early 2025 Maintained
Cash runwayLate 2025Into late 2025 Into late 2025; excludes future XOLREMDI revenue Maintained
FY25 Sales Guidance2025To be provided later Not yet providing Maintained (none)

Earnings Call Themes & Trends

TopicPrevious Mentions (Q1 and Q2)Current Period (Q3)Trend
WHIM launch and awarenessQ1: Approval impact; building advocacy and HCP education . Q2: Launch underway; early stocking; focus on awareness; reimbursement processes smooth; patient services via PANTHERx/X4Connect .3,400 HCPs engaged; >75% awareness; >80% likely prescribers; >150M lives covered; focus on disease education and screening .Building foundation; awareness/screening improving; preparing for 2025 ramp.
CN Phase 2 data and Phase 3 designQ1: Defined success (≥+500 ANC cells/µL) and design; Phase 3 to enroll ~150 patients; endpoints: infection rate and ANC responder . Q2: Interim Phase 2 positive; Phase 3 initiated; success looks like replicating ANC improvements .Final Phase 2: durable ANC rises; G-CSF reductions −52% M3, −70% M6 while maintaining normal mean ANC; neutrophil functionality comparable to healthy donors in sub-study; Phase 3 tracking to full enrollment mid-2025 .Increased conviction; functional data de-risking; execution progressing.
Reimbursement and accessQ2: Prior auths and exceptions working smoothly; specialty pharmacy distribution .>150M covered lives; continued emphasis on payer policy and access .Stable-to-improving access environment.
Ex-U.S./EMAQ1: Planning EU submission late 2024/early 2025 . Q2: Expect EMA MAA by early 2025; exploring geographies leveraging U.S. approval .EMA MAA by early 2025 reiterated .Maintained timelines.
Financial runway and PRVQ1: Runway into 2025; plan to monetize PRV . Q2: PRV monetized ($105M); runway into late 2025 .Runway into late 2025 reiterated (ex-future sales) .Maintained.

Management Commentary

  • CEO on CN Phase 2: “We could not be more pleased with the results…[they] increase our confidence in a positive outcome for our ongoing pivotal Phase 3 4WARD trial” .
  • CEO on WHIM launch: “We are seeing our focus on disease education translate into growing awareness of and screening for WHIM…setting a strong foundation for further identifying and treating patients” .
  • R&D lead on functionality: “The percentage of functional neutrophil in the healthy donors… and congenital CN subset are essentially the same following 6 months of mavorixafor therapy” .
  • CEO on sales cadence: “Sales appear to be flat quarter-over-quarter…some of the sales in the second quarter were product stocking…typical during initial launch” .
  • CFO on cash runway: “We ended the third quarter…with cash and equivalents of almost $136 million…runway into late 2025” (ex-future revenue) .

Q&A Highlights

  • CN dose-reduction drivers: Physicians reduced G-CSF in ~75% of eligible patients based on robust ANC responses with mavorixafor; 25%+ reduction considered clinically meaningful by HCPs and patients; some taken fully off G-CSF by Month 6 .
  • Phase 3 endpoints and timing: 4WARD is powered (>90%) on infection-rate reduction with conservative effect-size assumptions; full enrollment expected mid-2025; tolerability and potential post-hoc analyses on G-CSF reduction benefits may follow .
  • WHIM commercialization detail: Patient-directed campaigns launched; focus on awareness and screening; specific patient counts and sales guidance not yet provided .
  • Reimbursement dynamics: Prior authorization/exception pathways functioning; X4Connect supports rapid access; distribution via PANTHERx Rare .

Estimates Context

  • S&P Global consensus estimates (EPS and revenue) were not retrievable at time of query due to data-access limits; therefore, we cannot provide an apples-to-apples print vs. Street for Q3 2024 at this time. As a result, “vs. estimates” comparisons are unavailable and should be treated as not reported for this quarter (S&P Global data unavailable).

Key Takeaways for Investors

  • CN read-across: Final Phase 2 data (ANC durability, neutrophil functionality, and G-CSF sparing) materially de-risk the infection-rate endpoint in Phase 3, raising probability of technical success and widening the addressable opportunity beyond WHIM .
  • Launch pacing: Q3 sales were flat vs Q2 as initial stocking normalized; leading indicators (HCP engagement, awareness, screening, coverage) improved—watch for conversion to patient starts and prescription durability in 2025 .
  • Expense profile: SG&A elevated vs. prior year as commercial infrastructure scales; sustainability of SG&A growth vs. revenue ramp is a 2025 focal point .
  • Balance sheet: ~$135.8M cash/marketable securities provides runway into late 2025 (ex-future sales), supporting commercial build and 4WARD execution without near-term financing, absent upside/downside from launch trajectory .
  • Milestone catalysts: EMA MAA submission (early 2025), 4WARD site ramp and enrollment updates (through 2025), increasing WHIM screening/prescriber conversion, and potential ex-U.S. partnerships .
  • Risk checks: Launch pacing (ultra-rare prevalence, diagnosis lag), payer adoption, manufacturing/supply continuity, and Phase 3 outcomes remain the principal stock drivers per risk disclosures .

Financials Detail (supplemental)

  • Q3 2024 cost of revenue included ~$0.2M of license costs (royalties/milestones amortized) .
  • Non-cash items: Q3 2024 included a $1.9M warrant remeasurement loss; Q2 2024 included a $20.2M warrant remeasurement gain and $105M PRV sale .

Appendix: Selected Additional Disclosures

  • 4WARD trial footprint: regulatory approvals in ~85% of targeted countries; ~40% of planned sites initiated as of Q3 call .
  • Monotherapy impact in severe CN: >2× increase in mean ANC sustained through six months in severe patients (<500 cells/µL baseline) .
  • Neutrophil functionality: % functional neutrophils in CN sub-study comparable to healthy donors after six months of dosing (phagocytosis and ROS assays) .