X4 Pharmaceuticals Reports Second Quarter 2022 Financial Results and Provides Corporate Update
Highlights notable upcoming milestones including data from Phase 1b trial in chronic neutropenia, expected in late September, and results from global, pivotal Phase 3 trial in WHIM syndrome, expected in 4Q 2022
Announces additional positive data from Phase 1b trial in Waldenström’s Macroglobulinemia supportive of a potential partnership to advance clinical development
Conference call to be hosted today at
“With the completion of our recent financing and the sharpening of our strategic focus on chronic neutropenic disorders, we believe that we are well positioned to deliver significant future value to both patients and shareholders as we approach key milestones in the second half of this year,” said
Recent Highlights & Anticipated Upcoming Milestones
- Completed a private investment in public equity (PIPE) financing, raising gross proceeds of approximately
$56 million; the financing included participation from new and existing investors.
- Entered into an amendment to the company’s loan and security agreement with Hercules Capital to extend the interest-only period of its loan facility by up to twelve months (into 2024), subject to achieving certain financial and business milestones.
- Announced a strategic re-prioritization of resources towards advancing mavorixafor in chronic neutropenic disorder indications, including WHIM syndrome, while progressing oncology programs only upon completion of strategic partnership(s); the announced strategic update was also inclusive of cost-cutting initiatives estimated to support extension of X4’s cash runway into the third quarter of 2023.
- Presented research data at the
June 2022meeting of the European Hematology Association(EHA) further supporting mechanism and market potential of mavorixafor across of range of clinical indications.
- Planned late
September 2022presentation of data from the fully enrolled Phase 1b trial evaluating mavorixafor for the treatment of chronic neutropenic disorders, including congenital, idiopathic, and cyclic neutropenia.
- Planned fourth quarter 2022 presentation of results from the global, pivotal Phase 3 clinical trial of mavorixafor for the treatment of patients with WHIM syndrome; data anticipated to support a submission for
U.S.regulatory approval early in the second half of 2023.
Positive Data Update from Phase 1b Trial in Waldenström’s Macroglobulinemia (WM)
- The Phase 1b, open-label, multicenter, single-arm study is evaluating the safety and efficacy of mavorixafor in combination with the BTK inhibitor ibrutinib in adult patients (either treatment naïve or relapsed/refractory) with the rare B-cell lymphoma Waldenström’s macroglobulinemia (WM) and confirmed MYD88 and CXCR4 genetic mutations.
- A total of 16 patients were enrolled in the study; all were dosed with oral, once-daily doses of ibrutinib (420 mg) and escalating doses (200 mg, 400 mg, 600 mg) of oral mavorixafor, also once daily.
- As of
June 2022, 10 of 12 evaluable patients (83%) achieved a major response (MR) to therapy, or a greater than 50% reduction in serum IgM from baseline:
- In relapsed/refractory patients, 8 of 9 (89%) achieved a MR
- In treatment-naïve patients, MR was seen in all patients escalated to >200 mg of mavorixafor
- Adding mavorixafor to ibrutinib was associated with a higher MR rate at 9, 12, and 24 months compared to previously reported MR rates achieved with ibrutinib monotherapy.
- In addition, patients achieved elevations in absolute neutrophil count (ANC), with no neutropenic events reported; patients also experienced fewer infections over time with chronic dosing.
- No major safety signals had been identified in the trial as of the data cut-off; mavorixafor in combination with ibrutinib showed a safety profile similar to ibrutinib monotherapy (N=16, including 8 patients escalated to the 600 mg dose of mavorixafor).
- In June, mavorixafor was granted Orphan Drug Designation by the
U.S. Food & Drug Administrationfor the treatment of patients with WM, regardless of CXCR4 mutation status.
- The Phase 1b clinical trial is expected to be completed in the fourth quarter of 2022; further clinical studies in WM will now be subject to completing a strategic partnership.
Second Quarter 2022 Financial Results
- Cash, Cash Equivalents & Restricted Cash: X4 had
$48.7 millionin cash, cash equivalents, and restricted cash as of June 30, 2022. On June 30, 2022, the company announced a private equity financing (PIPE) of approximately $56 millionbefore deducting offering costs. The financing closed on July 6, 2022, and proceeds are not reflected in the cash on hand as of June 30, 2022. Including funds raised in this offering, the company’s recently announced cost-cutting measures, and recent amendments to its loan and security agreement with Hercules, X4 believes that it has sufficient funds to support company operations into the third quarter of 2023.
- Research and Development (R&D) Expenses were
$13.8 millionfor the second quarter of 2022 as compared to $13.2 millionfor the comparable period in 2021. R&D expenses include $0.7 millionand $0.8 millionof certain non-cash expenses for the second quarter of 2022 and 2021, respectively.
- Selling, General, and Administrative Expenses (SG&A) were
$6.7 millionfor the second quarter of 2022 as compared to $5.8 millionfor the comparable period in 2021. SG&A expenses include $0.8 millionand $1.0 millionof certain non-cash expenses for the second quarter of 2022 and 2021, respectively.
- Net Loss: X4 reported a net loss of
$21.2 millionfor the second quarter of 2022, as compared to $19.6 millionfor the comparable period in 2021. Net losses include $1.5 millionand $1.8 millionof certain non-cash expenses for the second quarter of 2022 and 2021, respectively.
Conference Call and Webcast
X4 will host a conference call and webcast today at
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. These statements may be identified by the words “may,” “will,” “could,” “would,” “should,” “expect,” “plan,” “anticipate,” “intend,” “believe,” “estimate,” “predict,” “project,” “potential,” “continue,” “target,” or other similar terms or expressions that concern X4's expectations, strategy, plans, or intentions. Forward-looking statements include, without limitation, statements regarding the clinical development and therapeutic potential of mavorixafor; the anticipated achievement of upcoming clinical milestones; the expected availability, content, and timing of clinical trial data; anticipated regulatory milestones; and the company’s cash runway. Any forward-looking statements in this press release are based on management's current expectations and beliefs. Actual events or results may differ materially from those expressed or implied by any forward-looking statements contained herein, including, without limitation, on account of uncertainties inherent in the initiation and completion of preclinical studies and clinical trials and clinical development; the risk that trials and studies may be delayed, including, but not limited to, as a result of the effects of the ongoing COVID-19 pandemic, and may not have satisfactory outcomes; the risk that the outcomes of preclinical studies or earlier clinical trials will not be predictive of later clinical trial results; the risk that initial or interim results from a clinical trial may not be predictive of the final results of the trial or the results of future trials; the potential adverse effects arising from the testing or use of mavorixafor or other product candidates; the risks related to X4’s ability to raise additional capital, and other risks and uncertainties, including those described in the section entitled “Risk Factors” in X4’s Quarterly Report on Form 10-Q filed with the
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS
(in thousands, except per share amounts)
|Three Months Ended||Six Months Ended|
|Research and development||$||13,821||$||13,193||27,934||25,297|
|Selling, general and administrative||6,749||5,804||14,413||11,636|
|Gain on sale of non-financial asset||—||—||(509||)||—|
|Total operating expenses||20,570||18,997||41,838||36,933|
|Loss from operations||(20,570||)||(18,997||)||(41,838||)||(36,933||)|
|Other expense, net||(638||)||(635||)||(1,312||)||(1,369||)|
|Loss before provision for income taxes||(21,208||)||(19,632||)||(43,150||)||(38,302||)|
|Provision for income taxes||4||6||27||12|
|Deemed dividend due to Class B warrant price reset||—||—||(2,259||)||(8,239||)|
|Net loss attributable to common stockholders||$||(21,212||)||$||(19,638||)||$||(45,436||)||$||(46,553||)|
|Net loss per share attributable to common stockholders- basic and diluted||$||(0.60||)||$||(0.74||)||$||(1.31||)||$||(1.97||)|
|Weighted average common shares outstanding-basic and diluted||35,437||26,527||34,592||23,655|
CONDENSED CONSOLIDATED STATEMENTS OF CASH FLOWS
|Six months ended
|Adjustments to reconcile net loss to net cash used in operating activities||4,083||4,345|
|Changes in operating assets and liabilities||391||(3,672||)|
|Net cash used in operating activities||(38,703||)||(37,641||)|
|Net cash used in investing activities||(60||)||(582||)|
|Net cash provided by financing activities||4,609||54,117|
|Impact of foreign exchange on cash, cash equivalents and restricted cash||(271||)||(103||)|
|Net (decrease) increase in cash, cash equivalents and restricted cash||(34,425||)||15,791|
|Cash, cash equivalents and restricted cash at beginning of period||83,108||80,702|
|Cash, cash equivalents and restricted cash at end of period||$||48,683||$||96,493|
CONSOLIDATED BALANCE SHEETS
|Cash and cash equivalents||$||47,378||$||81,787|
|Research and development incentive receivable||843||747|
|Prepaid expenses and other current assets||4,364||5,344|
|Total current assets||52,585||87,878|
|Property and equipment, net||1,309||1,514|
|Current portion of lease liability||1,136||1,075|
|Current portion of long-term debt||8,607||795|
|Total current liabilities||22,002||14,023|
|Long-term debt, including accretion, net of discount||24,490||33,139|
|Total stockholders' equity||30,073||64,412|
|Total liabilities and stockholders' equity||$||81,019||$||117,176|
Source: X4 Pharmaceuticals