Announcement • Jun 27

X4 Pharmaceuticals, Inc. Announces Positive Interim Clinical Data from Ongoing Six-Month Phase 2 Trial of Mavorixafor in Chronic Neutropenia (Cn) and Initiation of Pivotal Phase 3 Cn Trial

X4 Pharmaceuticals, Inc. announced positive new clinical data from its ongoing Phase 2 clinical trial evaluating the safety and efficacy of mavorixafor, an oral CXCR4 antagonist, in the treatment of people with chronic neutropenia (CN). An interim analysis of data from the ongoing six-month study showed that once-daily oral mavorixafor was generally well tolerated and durably increased participants’ absolute neutrophil counts (ANC) both as a monotherapy and in combination with stable doses of injectable granulocyte colony-stimulating factor (G-CSF), the only therapy approved in the U.S. for severe chronic neutropenia. The company also announced that it is currently screening patients for enrollment into its global, pivotal Phase 3 clinical trial, the 4WARD study, evaluating the efficacy, safety, and tolerability of oral, once-daily mavorixafor (with or without stable doses of G-CSF) in people with congenital, acquired primary autoimmune, or idiopathic CN who are experiencing recurrent and/or serious infections. The 52-week trial is a randomized, double-blind, placebo-controlled, multicenter study aiming to enroll 150 participants. The Phase 2 study of mavorixafor is a six-month, open-label clinical trial that enrolled a total of 23 participants diagnosed with idiopathic, congenital, or cyclic CN. The interim analysis included results from the two treatment groups in the study (mavorixafor monotherapy and mavorixafor with stable-dose G-CSF) that most closely mirror the participant population of the newly initiated Phase 3 4WARD trial. Fifteen participants were enrolled across these two groups and, as of the May 14, 2024 interim analysis data cut-off date, seven had completed the study, and five remain ongoing. Data from a third treatment group of eight participants receiving mavorixafor and dose-adjusted G-CSF are expected to be presented later this year. The mavorixafor monotherapy group included 10 participants and the mavorixafor with stable-dose G-CSF group included five participants. As of the data cut-off date, findings from the interim analysis show: 100% (6/6) of evaluable participants who had completed the six-month study achieved target ANC increase (ANC >500 cells/µL) at Months 3 and 6 on once-daily mavorixafor therapy with or without stable-dose G-CSF. Participants on mavorixafor monotherapy achieved mean ANC levels above the lower limit of normal for CN (=1,500 cells/µL) at Month 3 (n=8) and Month 6 (n=3). Mavorixafor monotherapy also durably increased ANC in participants with severe CN (ANC<500 cells/µL at baseline), achieving mean ANC of ~800-1,000 cells/µL (ANC range targeted by experts) at Months 1, 3, and 6 (n=5, 3, and 2, respectively). Participants on mavorixafor in combination with stable-dose G-CSF experienced increases in mean ANC of >1,000 cells/µL at Months 1, 3, and 6 (n=4, 4, and 3, respectively) versus baseline. Across the 23 participants enrolled in the study, mavorixafor was generally well tolerated as a monotherapy and in combination with G-CSF, with no drug-related serious adverse events reported, as of the interim analysis data cut-off date. Of the 23 participants, three discontinued due to non-serious adverse events. The overall safety profile remains consistent with previous clinical studies. The Phase 1b/Phase 2 clinical trial (NCT04154488) is a proof-of-concept, open-label, multicenter study designed to assess the safety and tolerability of oral mavorixafor, with or without injectable G-CSF, in participants with chronic neutropenic disorders, including idiopathic, cyclic, and congenital neutropenia. In the Phase 1b portion of the study, participants received one dose of oral mavorixafor and were assessed for magnitude of absolute neutrophil count (ANC) response and tolerability. In this initial portion of the study, 100% of participants (n=25) responded to treatment and mavorixafor was generally well tolerated alone or dosed concurrently with G-CSF. The ongoing Phase 2 portion of the trial (n=23 fully enrolled) is assessing the safety, tolerability, and the impact on participants’ neutropenia of oral, once-daily mavorixafor with and without concurrent injectable G-CSF therapy over a six-month period.

Seeking Alpha • Sep 19

X4 Pharmaceuticals: Upcoming Catalysts

Summary X4 Pharmaceuticals is advancing Mavorixafor, a once-daily oral dose, first in class, small molecule antagonist of the CXCR4 receptor in WHIM syndrome and in chronic neutropenia disorders. On Sept 27, topline results will be presented from a Phase 1b trial testing Mavorixafor with or without G-CSF in patients with chronic neutropenia including severe congenital, idiopathic or cyclic. More importantly, in Q4, X4 plans to release results from its global Phase 3 pivotal trial in patients with WHIM syndrome (a rare disease caused by a genetic mutation). Binary outcome. If Ph 3 WHIM trial results successful, possible NDA filing in mid-23; potential FDA approval and Priority Review Voucher in H1 2024; commercially launch in the U.S. X4's market cap approx. $100M, approx. $100M cash (or equiv.), $33MM in secured debt. Sufficient cash to Q3 '23. 51M outstanding $1.095 warrants create a share price overhang. X4 Pharmaceuticals Logo (X4 Website) Headquartered in Boston, MA with research facilities in Vienna, Austria, X4 Pharmaceuticals, Inc. (XFOR) is a late-stage clinical biotech company focused on the discovery and development of CXCR4 targeted small molecule therapeutics to address diseases of the immune system including chronic neutropenic disorders and certain types of cancer. X4' lead drug, Mavorixafor, is an orally administered, first-in-class, small molecule antagonist of the CXCR4 receptor being advanced in a number of clinical trials, with two upcoming significant near-term clinical trial readouts; from a Phase 1b trial on September 27th, and more importantly the results from its pivotal Phase 3 trial in Q4. With approximately 86.5 million shares outstanding (including pre-funded warrants) X4 recently issued 51 million warrants as part of a $55 million financing which closed in early July, which may put a lid on X4's share price for some time. X4 also has $33 million in secured debt which contains some onerous terms. This is not an investment for everyone, but there are some very sophisticated biotech investors, such as Bain Capital, NEA, Orbimed, Lumira and Acorn, who all participated in the recent $55 million private placement. Notwithstanding some issues and the binary aspect of the investment in X4, I thought the upside was sufficient at current share price levels to buy a small X4 share position last week. X4 trades at approximately $1.10 per share which works out to X4 having a market cap of under $100M. The company has an equal amount of cash, or equivalent, and believes that it has sufficient resources to operate to Q3 2023, subject to satisfying terms set out in its secured loan agreement with Hercules Capital, discussed in more detail below. While its upcoming Phase 1b trial results in chronic neutropenia are important, its most significant upcoming event is the read-out from its pivotal Phase 3 trial in WHIM Syndrome expected in Q4. The Company and investors are optimistic that the results will be favourable. If the Phase 3 WHIM Syndrome clinical results meet its endpoints, there could be a significant upside for X4 investors over the next few years. An NDA for Mavorixafor could be filed in mid-2023, and FDA approval as well as a Priority Review Voucher (worth in excess of $100 Million) could be obtained in H1 2024. Commercial sales in the US could begin in 2024. The Company believes it has patent protection to 2038 and beyond. WHIM Syndrome is a serious and debilitating rare disease with a large unmet need. Potential peak sales for Mavorixafor in WHIM Syndrome in the U.S. is in the $200 to $300 million range (my estimate), and European approval could follow 6 to 12 months later. On the other hand, if the Phase 3 WHIM trial results fail to meet their primary endpoints etc. or are otherwise disappointing, it could spell trouble for the company. We should know by year-end. This article sets out my analysis. Founders of X4 X4's five founders are an impressive group with significant biotech credentials. The list includes its current CEO Dr. Paula Ragan, Ph.D. (M.I.T), as well as the late Henri Termeer (who passed away in 2017). Mr. Termeer was the past Chairman, President and CEO of Genzyme Corporation (bought by Sanofi), as well as a former board member of the Massachusetts Institute of Technology Corporation and the Board of Fellows of Harvard Medical School. Senior Management Team X4's senior management team is led by Dr. Paula Ragan whose past experience also includes a senior role at Genzyme (where she had significant involvement in rare diseases). X4 Management Team (X4 August 2022 Corporate Presentation) X4's board of directors includes a very experienced group. For more details, the Company's website includes a short biography of each director found here. Lead Drug Mavorixafor X4's lead drug Mavorixafor is an orally administered, first-in-class, small molecule antagonist of the CXCR4 receptor, which according to X4's August 2022 presentation, "enables the mobilization of immune cells from the bone marrow to the blood to improve immune system function." X4's pipeline is set out in the slide below. X4 Pipeline and Upcoming Milestones (X4 August 2022 corporate presentation) Mavorixafor is being advanced in a number of clinical trials including: 1. WHIM Syndrome (Warts, Hypogammaglobulinemia, Infections and Myelokathexis), an immunodeficiency disease and sub-type of chronic neutropenia. X4 is currently in the completion stages of a global Phase 3 pivotal trial for WHIM Syndrome; 2. Chronic neutropenia disorders including subtypes Congenital, Cyclic and Idiopathic in which X4 is fully enrolled in a Phase 1b trial; and 3. Waldenstrom's Macroglobulinemia ("WM"), a rare form of lymphoma (considered a type of non-Hodgkin's lymphoma) in which a person's bone marrow produces too many abnormal white blood cells; currently in Phase 1b trials. On August 4, 2022, X4 released some very promising Phase 1b trial results in WB (updated September 12). Mavorixafor was also awarded Orphan Drug designation by the FDA for WM disease regardless of CXCR4 genetic mutation. Note, that X4 has recently announced its strategic decision to focus its resources on advancing Mavorixafor in only WHIM Syndrome and Chronic Neutropenia, and will proceed with this WM oncology and other assets only through partnering these assets going forward, although it will complete the Phase 1b WM trial (currently being finalized). Upcoming Catalysts: X4's two upcoming catalysts are: a. the presentation and discussion of the results from its open-label, multi-center Phase 1b clinical trial (ClinicalTrials.gov Identifier: NCT04154488) evaluating Mavorixafor, in 25 chronic neutropenia patients with idiopathic, cyclic, and congenital neutropenia. The presentation is scheduled for September 27, 2022 in the form of an X4 investor webinar. For more details on what type of information will be presented at the September 27 webcast, see X4's August 31, 2022 press release here; and b. the readout of X4's pivotal Phase 3 4WHIM trial (ClinicalTrials.gov Identifier NCT03995108) of once-daily, oral Mavorixafor in individuals aged 12 and older with WHIM Syndrome, is expected to be released in Q4 (no specific date currently provided). The outcome of this pivotal Phase 3 WHIM trial is the most significant upcoming catalyst for the Company as success could lead to Mavorixafor filing an NDA in Q3 2023, obtaining FDA approval and commercial sales beginning in 2024 in addition to a potential Priority Review Voucher ("PRV") which can be sold for $100 million or more (upon FDA approval). The proceeds from the PRV could provide a form of non-dilutive funding for the Company in time for the Company's commercial launch in WHIM Syndrome in 2024. X4: Significant Near-Term Catalysts (X4 August 2022 corporate presentation) As X4 sets out in its website, chronic neutropenic diseases are caused by a dysregulated immune system, and patients who have these diseases suffer chronic immunosuppression, which in turn causes severe and/or life-threatening infections throughout their lifetime. CXCR4 Plays Key Role in Immune Cells (X4 August 2022 corporate presentation) Chronic neutropenic diseases are characterized by low white blood cell counts (cytopenia) and/or dysregulated or dysfunctional immune cells. According to X4 and summarized in the slides below, the CXCR4 receptor plays a key role in "the expansion, maturation and mobilization of immune cells, and its dysfunction can cause a range of chronic neutropenic disorders." As discussed in X4's slides below, the root cause of WHIM Syndrome is the "over-signalling of CXCR4, driven by CXCR4 genetic mutations, that impact all white blood cells". X4 believes that Mavorixafor can "increase the mobilization of white blood cells, including neutrophils, from bone marrow to bloodstream". Root Cause of WHIM Syndrome: Over-signaling of CXCR4 (X4 August 2022 Corporate Presentation) X4: Dysregulated Immune System (X4 August 2022 corporate presentation) While there is no treatment approved specifically for WHIM Syndrome, the current protocol is to treat disease symptoms; antibiotics are prescribed for acute infection and multiple daily injections of granulocyte-colony stimulating factor ("G-CSF") for those with severe neutropenia. Unfortunately, daily injections of G-CSF are often not tolerated well by patients or are discontinued because of the inconvenience/discomfort from taking multiple daily injections. X4 believes that Mavorixafor will provide a safe and efficacious treatment for WHIM and other neutropenia-related diseases and if approved, has the potential of becoming the new standard of care in the treatment of all chronic neutropenia related diseases including WHIM Syndrome. X4's Regulatory Designations (WHIM Syndrome): X4 has been awarded various regulatory designations for Mavorixafor in the treatment of WHIM including: Breakthrough Therapy Designation in the US Fast Track Designation Rare Pediatric Disease Designation (making it eligible for the Priority Review Voucher potentially worth $100 million or more) Orphan Drug Status in the US and Europe Mavorixafor: Realizing potential of CXCR4 Antagonism in oral capsule (X4 August 2022 corporate presentation) Phase 2 WHIM Trial Demonstrates Activity Across Phase 3 Endpoints Looking at X4's earlier Phase 2 WHIM results, X4 is cautiously optimistic that its pivotal Phase 3 WHIM trial will succeed. Phase 2 WHIM trial results showed a 600% increase in neutrophil counts and a greater than 80% reduction in annualized infections. Furthermore, the WHIM patients who continued on in the open-label extension phase of the Phase 2 trial showed continued improvement as seen in X4's slides below. X4 August 2022 Corporate presentation Phase 2 WHIM Open Label Extension: Continued Improvements Across Patients In the Phase 2 WHIM Open Label extension trial, there was a continued benefit among WHIM patients as summarized by X4 in the slide below. WHIM Phase 2 Open Label Extension: Continued Improvements Across Patients (X4 August 2022 Corporate Presentation) Design of WHIM Global Pivotal Phase 3 Trial X4's global pivotal Phase 3 WHIM trial design has two arms; 14 WHIM patients who received daily dosing of Mavorixafor, and a 14 WHIM patient placebo group. Ultimately, the trial was overenrolled and now has 31 patients. The full details of the Phase 3 WHIM clinical trial, in 23 clinical sites in multiple countries in patients ages 12 and over, including the US, Europe and elsewhere, are set out on the clinicaltrials.gov website (ClinicalTrials.gov Identifier: NCT03995108) which was updated on September 14, 2022. The Phase 3 WHIM trial consists of a Randomized Placebo-controlled period (52 weeks) followed by an open-label period in which any participant may continue to receive Mavorixafor (if available in the jurisdiction, until the trial terminates or the drug is approved commercially). In order to be enrolled in this Phase 3 trial, the patient has to have, among other criteria, a "genotype-confirmed mutation of chemokine (C-X-C motif) receptor 4 (CXCR4) consistent with WHIM phenotype". Patients enrolled in the Phase 3 trial are treated for 52 weeks, with absolute neutrophil count measurements being the primary endpoint being measured at Weeks 13, 26, 39 and 52. There are also 52 secondary endpoints listed including infections, wart burdens, quality of life assessments and other measurements. Dosing is 400 mg per day for patients weighting over 50 kg and 200 mg once daily for those under 50 Kg and under 18 years of age. For greater details see the clinicaltrials.gov website (ClinicalTrials.gov Identifier: NCT03995108). During our recent discussion, X4's CEO indicated that X4 has had ongoing rigorous discussions with the FDA (because of its Breakthrough Therapy designation) during which the FDA has indicated that, assuming the clinical trial endpoints are met, the data from the Phase 3 trial will be sufficient to warrant full FDA approval for Mavorixafor in the treatment of WHIM for patients 12 years old and older. Design of Global Phase 3 WHIM Trial (age 12 plus) (X4 August 2022 Corporate presentation) Size of the WHIM Market X4's CEO estimates the WHIM patient population to be in the 1,000 to 3,500 range in the U.S. and probably at least double that number in the rest of the world. She also indicated that based upon her own experience (when she held a senior position at Genzyme in rare diseases), she would not be surprised if the actual number of WHIM syndrome patients in the U.S. turned out to be significantly higher once an effective treatment for WHIM was approved and on the market (such that diagnosing WHIM patients would be a meaningful exercise and genetic testing for such suspected patients paid for by X4). At this point however it is simply speculation. If and when Mavoraxifor for WHIM is approved by the FDA in the US, it will likely be approved in Europe about 6 to 12 months later. As part of their commercial strategy to market Mavoraxifor for WHIM, X4 plans to create WHIM Centres for Excellence, provide ongoing education to medical professionals, work with WHIM patient advocacy groups and also pay for genetic testing to help diagnose WHIM Syndrome patients. In discussing proposed pricing for Mavoraxifor in the U.S., if approved, while the pricing has not yet been finalized, it is anticipated that for a rare disease such as WHIM, pricing of Mavoraxifor, is expected in the range of $200,000 to $400,000 per annum in the US. Analysts covering X4 have used an average of about $300,000 annual pricing in modelling Mavoraxifor's future revenues. If X4 manages to take most of the WHIM market in the U.S. (which if approved, is likely), that could generate peak annual sales of between $200 to $300 million (assuming 1,000 WHIM patients in the U.S. at $200,000 to $300,000 per annum) less the usual discounts. Clearly identifying and expanding the number of WHIM patients, will potentially increase sales revenue projections (assuming diagnosis, prescription and insurance coverage). Sales in Europe and the rest of the world could double that figure (assuming that those markets aren't licensed out under license/distribution agreements in which case other forms of revenues will be expected). As well, if Mavoraxifor is approved for WHIM Syndrome in the U.S. and Europe, expansion to Japan, China and other parts of the world could also follow although more likely those ex-US jurisdictions would be the subject of licensing agreements (another source of potential non-dilutive financing and revenues for X4). Chronic Neutropenia Beyond WHIM Syndrome - Phase 1B trial On September 27th, X4 will be providing a corporate update and updated clinical trial data from its Phase 1b clinical trial with 25 patients being treated for Chronic Neutropenia or "CN", including severe congenital, idiopathic or cyclic CN. Severe CN conditions can flare up a few times per year, with very severe and frequent infections, which may require hospitalization, IV administered antibiotics and potentially severe illness or even death. In order to manage chronic severe CN, current treatments are unsatisfactory such as once or twice daily injections of G-CSF which are not always well tolerated or tolerated at all and which can have serious side effects. The only other alternative is a bone marrow transplant which is painful, inconvenient and expensive. Chronic Neutropenia patients have a very low neutrophil count so potential treatments try to improve the absolute neutrophil count ("ANC") as well as increases in white blood cells, lymphocyte and monocyte counts. Initial Phase 1 studies involving Mavorixafor and CN showed some promising results including data presented at ASH in 2021. As can be seen in the slide below, a single combination dose of Mavorixafor in combination with G-CSF compared to a single dose of G-CSF alone, resulted in a 2 fold increase in Absolute Neutrophil Count, in addition to increases in total white blood cells, absolute lymphocyte counts and absolute monocyte counts. See X4's slide below summarizing its ASH2021 results for more details. ASH 2021: Mavorixafor impact on white blood cells in CN patients (X4 August 2022 corporate presentation) Chronic Neutropenia Beyond WHIM Syndrome (X4 August 2022 Corporate Presentation) Current Treatments for CN have Significant Limitations (X4 August 2022 Corporate presentation) 25% of CN Patients in US experience Serious Infection Events Each Yr. (X4 August 2022 Corporate presentation) X4 estimates that its target market in the US for chronic neutropenia patients are those who suffer more than two serious infection events ("SIEs") per year (approximately 5,000 patients i.e. 25% of all CN patients). An SIE is defined by X4 as an infection that requires hospitalization, IV antibiotics and/or results in disability or death. Presumably, if Mavorixifor obtains FDA approval in WHIM, and if the Phase 1b trial results are encouraging in chronic neutropenia, the odds of obtaining approval for Mavorixafor in all types of chronic neutropenia increase significantly. X4 anticipates that it will have greater clarity on the path forward for Mavorixafor in the non-WHIM CN indications next year. X4 Pharmaceuticals, Inc.: Corporate Summary XFOR data by YCharts Symbol: XFOR (Nasdaq): 52-week share price high and low- $6.18 to $0.86 Share price close of September 14, 2022: $1.12 Market Cap: $91,750,000 assuming 86,516,563 common shares outstanding (including the 13,276,279 pre-funded June 30, 2022 $55 million private placement financing warrants described below, as well as an additional 4.6 million previously issued pre-funded warrants, which I am including as equivalent to common shares for the purpose of this calculation) Common Shares outstanding as of June 20, 2022 - 30,991,198 (Source: X4 Form 10-Q, filed August 4, 2022) X4 Financial Snapshot (X4 August 2022 Corporate Presentation) Cash: Pro-forma of $101 Million following June 30th, 2022 financing. See X4's Slide above. $55 Million Private Placement announced June 30, 2022 (closed July 6, 2022) Unit price of $1.095, under which 37,649,086 common shares were issued, 13,276,279 pre-funded warrants were issued to purchase 13,276,279 common shares at an exercise price of $0.001; and accompanying 50,925,365 warrants were issued with an exercise price of $1.095, with a 60 month expiry period. (Source: X4 Form 10-Q filed with the SEC on August 4, 2022) Summary of Estimated Common Shares (or equivalent pre-funded Warrants) 86,516,563 common shares (including 13,276,279 pre-funded Warrants from the June 30th financing, as well as 4.6 million pre-funded Warrants from earlier financing, which I am treating as common shares for the purposes of calculating market cap and most other purposes) (Foregoing calculation confirmed by X4's CFO by email) 50,925,365 warrants outstanding (from the June 30 private placement) with an exercise price of $1.095 (expiring on or about July 6, 2027) Additional Warrants outstanding: 3.9 million Class A warrants have a 2024 expiry and $13.20 exercise price; 5.4 million Class B warrants have a $1.50 exercise price and an effective expiry date 30 days post Phase 3 WHIM data (which is expected to be released in Q4).(Source: X4 Form 10-Q and email confirmation from X4's CFO) Stock Options Outstanding as of June 30, 2022: 1,746,638 options at a weighted average exercise price of $8.59 and 7.5 years before expiry (Source: X4 Form 10-Q) Long Term Secured Debt: Hercules Loan Agreement, as Amended: $33,097,000 outstanding as of June 30, 2022 to Hercules Capital Inc. ("Hercules") with an effective annual rate as of June 30 of 10.9% per annum. Subject to meeting certain milestone events, the monthly loan payments are interest only until February 1, 2023 (possibly extended to August 1, 2023 or possibly extended to February 1, 2024 as described below); subject to certain amended terms, thereafter monthly interest and principal payments are to be made; the Hercules loan matures on July 1, 2024. (Source: X4 Form 10-Q, Note 7). On June 30, 2022, X4 entered into Amendment No. 4 to the Hercules Loan Agreement which added two milestones: the first was to extend the interest-only payments from February 1, 2023 to August 1, 2023, provided X4 raised $50 million (achieved) and achieve Performance Milestone III as defined in the Hercules Loan Agreement but not disclosed in the Form 10-Q. (Source: Note 7 of Form 10-Q). To achieve the second milestone which would extend the interest-only monthly payment portion of the Hercules loan to February 1, 2024 the Company has to raise an additional $25,000,000 and file a New Drug Application for Mavorixafor for the treatment of WHIM syndrome. In addition, there is a requirement under the Hercules Loan Agreement that X4 maintain a minimum cash balance of $30,000,000 (in which Hercules has a first position security interest security). The $30,000,000 cash minimum can be reduced to $20,000,000 following the achievement of Performance Milestone III. If and when X4 files a New Drug Application for Mavorixafor for the treatment of WHIM syndrome, that covenant is extinguished. (Source: Note 7, Form 10-Q)

Snowflake Score
Valuation	1/6
Future Growth	2/6
Past Performance	0/6
Financial Health	5/6
Dividends	0/6

Historical stock prices
Current Share Price	US$3.94
52 Week High	US$4.83
52 Week Low	US$1.35
Beta	0.32
1 Month Change	-9.01%
3 Month Change	15.54%
1 Year Change	34.93%
3 Year Change	-93.75%
5 Year Change	-98.34%
Change since IPO	-99.14%

	XFOR	US Biotechs	US Market
7D	-11.1%	1.0%	1.1%
1Y	34.9%	40.3%	26.7%

XFOR volatility
XFOR Average Weekly Movement	11.9%
Biotechs Industry Average Movement	10.7%
Market Average Movement	7.2%
10% most volatile stocks in US Market	16.2%
10% least volatile stocks in US Market	3.1%

XFOR fundamental statistics
Market cap	US$377.28m
Earnings (TTM)	-US$99.72m
Revenue (TTM)	US$9.01m

XFOR income statement (TTM)
Revenue	US$9.01m
Cost of Revenue	US$1.64m
Gross Profit	US$7.38m
Other Expenses	US$107.10m
Earnings	-US$99.72m

Earnings per share (EPS)	-1.06
Gross Margin	81.82%
Net Profit Margin	-1,106.30%
Debt/Equity Ratio	45.4%

Data	Last Updated (UTC time)
Company Analysis	2026/05/15 21:51
End of Day Share Price	2026/05/15 00:00
Earnings	2026/03/31
Annual Earnings	2025/12/31

Package	Data	Timeframe	Example US Source ^*
Company Financials	10 years	Income statement Cash flow statement Balance sheet	SEC Form 10-K SEC Form 10-Q
Analyst Consensus Estimates	+3 years	Forecast financials Analyst price targets	Analyst Research Reports Blue Matrix
Market Prices	30 years	Stock prices Dividends, Splits and Actions	ICE Market Data SEC Form S-1
Ownership	10 years	Top shareholders Insider trading	SEC Form 4 SEC Form 13D
Management	10 years	Leadership team Board of directors	SEC Form 10-K SEC Form DEF 14A
Key Developments	10 years	Company announcements	SEC Form 8-K

Analyst	Institution
Leah Rush Cann	Brookline Capital Markets
Arlinda Lee	Canaccord Genuity
Michael Schmidt	Guggenheim Securities, LLC

X4 Pharmaceuticals (XFOR) Stock Overview

Rewards

Risk Analysis

Analyst Price Targets

Intensifying Trial Risks Will Hobble Returns Yet Open Global Potential

US Launch And EMA Approval Will Expand Global Reach

Rising Rare Disease Awareness And Precision Medicine Will Expand Access

Top Analyst Narratives

Intensifying Trial Risks Will Hobble Returns Yet Open Global Potential

US Launch And EMA Approval Will Expand Global Reach

Rising Rare Disease Awareness And Precision Medicine Will Expand Access

X4 Pharmaceuticals, Inc. Competitors

Verastem

Prelude Therapeutics

Autolus Therapeutics

Invivyd

Price History & Performance

Recent News & Updates

CFO, Treasurer & Corporate Secretary recently sold US$73k worth of stock

Consensus revenue estimates increase by 13%

New minor risk - Profitability

First quarter 2026 earnings: EPS exceeds analyst expectations while revenues lag behind

XFOR: Single CXCR4 Hematology Program Will Drive Future Upside Potential

X4 Pharmaceuticals Announces European Commission Approval of XOLREMDI (Mavorixafor) For WHIM Syndrome

Recent updates

CFO, Treasurer & Corporate Secretary recently sold US$73k worth of stock

Consensus revenue estimates increase by 13%

New minor risk - Profitability

First quarter 2026 earnings: EPS exceeds analyst expectations while revenues lag behind

XFOR: Single CXCR4 Hematology Program Will Drive Future Upside Potential

X4 Pharmaceuticals Announces European Commission Approval of XOLREMDI (Mavorixafor) For WHIM Syndrome

XFOR: CXCR4 Program In WHIM And Chronic Neutropenia Will Drive Future Upside

XFOR: Mavorixafor Franchise Is Expected To Drive Future Upside Potential

X4 Pharmaceuticals, Inc., Annual General Meeting, May 11, 2026

XFOR: WHIM And Chronic Neutropenia Program Will Drive Future Upside

Full year 2025 earnings: EPS and revenues exceed analyst expectations

XFOR: EU WHIM Decision And Chronic Neutropenia Program Will Drive Upside Potential

X4 Pharmaceuticals Receives Positive Opinion from the Ema Chmp Recommending Approval of Mavorixafor in the Eu for Whim Syndrome

XFOR: Higher Fair Value Will Reflect Stronger Outlook Despite Margin Headwinds

XFOR: Fair Value View Will Balance Dilution Concerns And Execution Milestones

XFOR: Future Outlook Weighs October Financing Dilution Against Execution And Earnings Milestones

XFOR: Future Outlook Balances October Financing Dilution With Execution And Earnings Milestones

X4 Pharmaceuticals, Inc.'s (NASDAQ:XFOR) Shares May Have Run Too Fast Too Soon

XFOR Future Outlook Weighs Financing Dilution Against Long Term Commercial Upside

Price target increased by 8.5% to US$8.00

XFOR: Recent Financing Will Support Key Phase 3 Progress And Upside Potential

XFOR: Leadership Overhaul And Fresh Capital Will Drive Pipeline Progress

X4 Pharmaceuticals, Inc. has completed a Follow-on Equity Offering in the amount of $135.0233 million.

US Launch And EMA Approval Will Expand Global Reach

X4 Pharmaceuticals Announces Restructuring of Business Operations

US Launch And EMA Approval Will Expand Global Reach

High number of new directors

US Launch And EMA Approval Will Expand Global Reach

Price target decreased by 46% to US$23.25

X4 Pharmaceuticals, Inc. Announces Chief Financial Officer Changes, Effective August 12, 2025

New major risk - Shareholder dilution

X4 Pharmaceuticals, Inc. announced that it expects to receive $59.999985 million in funding

X4 Pharmaceuticals, Inc.'s (NASDAQ:XFOR) Revenues Are Not Doing Enough For Some Investors

New major risk - Financial position

New major risk - Market cap size

X4 Pharmaceuticals, Inc. has filed a Follow-on Equity Offering.

X4 Pharmaceuticals Presents Positive Phase 2 Chronic Neutropenia Trial Data in Poster Presentations at the 30th Annual Congress of the European Hematology Association (EHA)

X4 Pharmaceuticals Grants Fast Track Designation for Mavorixafor for the Treatment of Chronic Neutropenia by U.S. FDA

X4 Pharmaceuticals Announces Upcoming Presentation of Phase 2 Chronic Neutropenia Trial Data At the 30Th Annual Eha Congress

Consensus revenue estimates increase by 106%

Price target decreased by 43% to US$42.67

X4 Pharmaceuticals, Inc., Annual General Meeting, Jun 09, 2025

X4 Pharmaceuticals, Inc. to Report Q1, 2025 Results on May 01, 2025

US Launch And EMA Approval Will Expand Global Reach

X4 Pharmaceuticals, Inc. Auditor Raises 'Going Concern' Doubt

X4 Pharmaceuticals, Inc. to Report Q4, 2024 Results on Mar 25, 2025

X4 Pharmaceuticals Announces Strategic Restructuring to Drive Value and Maximize Opportunity for Mavorixafor in Chronic Neutropenia

New major risk - Financial position

Price target decreased by 8.9% to US$3.68

X4 Pharmaceuticals Announces Positive Results from Completed Six-Month Phase 2 Trial of Mavorixafor in Chronic Neutropenia (CN)

X4 Pharmaceuticals, Inc. to Report Q3, 2024 Results on Nov 13, 2024

CEO, President & Director notifies of intention to sell stock

New minor risk - Market cap size

X4 Pharmaceuticals Receives Non-Compliance Letter Regarding Nasdaq Listing Rule 5550(a)(2)

New major risk - Revenue and earnings growth