X4 Pharmaceuticals Reports Third Quarter 2019 Financial Results and Recent Business Highlights
Received orphan drug designation from the
Reported positive data from Phase 2a clinical trial of mavorixafor in combination with axitinib in advanced clear cell renal cell carcinoma patients
Hosts investment community conference call at
“Our core focus remains to advance our clinical programs with mavorixafor across our three lead rare disease indications. We are proud to report that we now have orphan drug designation in
Clinical Development Update
In
X4 commenced U.S. patient enrollment and is activating additional sites globally in the pivotal Phase 3 global clinical trial of mavorixafor in WHIM syndrome, a rare, inherited, primary immunodeficiency disease caused by genetic mutations in the CXCR4 receptor gene. The 52-week trial is designed to enroll 18 to 28 subjects in approximately 20 countries, followed by an open-label extension trial.
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Business Update
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Third Quarter 2019 Financial Highlights
Cash Position: As of
Research and Development Expenses (R&D): R&D expenses were
General and Administrative Expenses (G&A): G&A expenses were
Loss on Transfer of Non-Financial Assets: During the third quarter of 2019, X4 transferred to third parties the rights to develop and commercialize the programs underlying its in-process research and development (IPR&D) intangible assets, which were obtained in its merger with Arsanis. Accordingly, X4 recorded a
Net Loss: Net loss was
X4 expects current cash and cash equivalents to be sufficient to fund operations into the first half of 2021.
Conference Call and Webcast Information
X4 will host a conference call and webcast on
About Mavorixafor
X4 Pharmaceuticals’ lead product candidate, mavorixafor (X4P-001), is a potential first-in-class, once-daily, oral inhibitor of CXCR4, currently in a Phase 3 clinical trial or the treatment of WHIM syndrome, a rare, inherited, primary immunodeficiency disease caused by genetic mutations in the CXCR4 receptor gene. Mavorixafor has demonstrated proof-of-concept in WHIM syndrome in a Phase 2 clinical trial, including clinically meaningful increases in neutrophil and lymphocyte biomarker counts, as well as a trend of reduction in infection rates and wart burden, and a favorable safety profile. Mavorixafor was designated orphan drug status by the
About
Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. The words “may,” “will,” “could,” “would,” “should,” “expect,” “plan,” “anticipate,” “intend,” “believe,” “estimate,” “predict,” “project,” “potential,” “continue,” “target” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Forward-looking statements include, but are not limited to, statements regarding X4’s plans for the development of mavorixafor (X4P-001) or any of X4’s other product candidates or programs, including the clinical trials X4 plans to initiate; the design, rate of patient enrollment and clinical site initiation for its clinical trials; the potential benefits of mavorixafor, including as a treatment for advanced renal cell carcinoma, WHIM, Waldenström’s macroglobulinemia, or Severe Congenital Neutropenia; the safety, durability or efficacy of mavorixafor; X4’s plans to announce future trial results; X4's ability to fund its future operations; and X4’s plans to partner with current and future third-parties. These statements are subject to various risks and uncertainties, actual results could differ materially from those projected and X4 cautions investors not to place undue reliance on the forward-looking statements in this press release. These risks and uncertainties include, without limitation, the risk that trials and studies may be delayed and may not have satisfactory outcomes, potential adverse effects arising from the testing or use of mavorixafor or other product candidates, the risk that prior results, such as signals of safety, activity or durability of effect, observed from preclinical studies or clinical trials will not be replicated or will not continue in ongoing or future studies or trials involving X4’s product candidates, the risk that X4 will be unable to identify collaboration partners or realize benefits from any existing or future collaborations, and the risk that costs required to develop mavorixafor or other product candidates or to expand X4’s operations will be higher than anticipated. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, the risks and uncertainties described in the section entitled “Risk Factors” in X4’s most recent Annual Report on Form 10-K filed with the
X4 PHARMACEUTICALS, INC. | ||||||||||||||||
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS | ||||||||||||||||
(in thousands, except per share amounts) | ||||||||||||||||
(Unaudited) | ||||||||||||||||
Three Months Ended September 30 |
Nine Months Ended September 30 |
|||||||||||||||
2019 |
2018 |
2019 |
2018 |
|||||||||||||
Operating expenses: | ||||||||||||||||
Research and development |
|
8,589 |
|
|
6,158 |
|
|
23,098 |
|
|
15,657 |
|
||||
General and administrative |
|
4,383 |
|
|
2,387 |
|
|
13,726 |
|
|
5,374 |
|
||||
Loss on transfer of non-financial assets |
|
4,004 |
|
— |
|
4,004 |
|
— | ||||||||
Total operating expenses |
|
16,976 |
|
|
8,545 |
|
|
40,828 |
|
|
21,031 |
|
||||
Loss from operations |
|
(16,976 |
) |
|
(8,545 |
) |
|
(40,828 |
) |
|
(21,031 |
) |
||||
Other income (expense), net |
|
(738 |
) |
|
(57 |
) |
|
(1,142 |
) |
|
(972 |
) |
||||
Net loss |
|
(17,714 |
) |
|
(8,602 |
) |
|
(41,970 |
) |
|
(22,003 |
) |
||||
Adjustments related to convertible preferred stock | — |
|
(756 |
) |
|
(592 |
) |
|
(2,266 |
) |
||||||
Net loss attributable to common stockholders |
$ |
(17,714 |
) |
$ |
(9,358 |
) |
$ |
(42,562 |
) |
$ |
(24,269 |
) |
||||
Net loss per share attributable to common stockholders—basic and diluted |
$ |
(1.22 |
) |
$ |
(20.39 |
) |
$ |
(4.31 |
) |
$ |
(52.92 |
) |
||||
Weighted average common shares outstanding—basic and diluted |
|
14,562 |
|
|
459 |
|
|
9,866 |
|
|
459 |
|
X4 PHARMACEUTICALS, INC. | |||||||
CONDENSED CONSOLIDATED STATEMENTS OF CASH FLOWS | |||||||
(in thousands) | |||||||
(Unaudited) | |||||||
Three Months Ended |
Nine Months Ended |
||||||
September 30, 2019 |
September 30, 2019 |
||||||
Net loss |
$ |
(17,714 |
) |
$ |
(41,970 |
) |
|
Adjustments to reconcile net loss to net cash used in operating activities (non-cash items) (1) |
|
5,675 |
|
|
7,132 |
|
|
Changes in operating assets and liabilities |
|
(733 |
) |
|
(3,976 |
) |
|
Net cash used in operating activities |
|
(12,772 |
) |
|
(38,814 |
) |
|
Net cash provided by investing activities |
|
815 |
|
|
27,211 |
|
|
Net cash (used in) provided by financing activities |
|
(6,414 |
) |
|
80,377 |
|
|
Impact of foreign exchange on cash and restricted cash |
|
(264 |
) |
|
(266 |
) |
|
Net (decrease) increase in cash, cash equivalents and restricted cash |
|
(18,635 |
) |
|
68,508 |
|
|
Cash, cash equivalents and restricted cash at beginning of period |
|
95,641 |
|
|
8,498 |
|
|
Cash, cash equivalents and restricted cash at end of period |
$ |
77,006 |
|
$ |
77,006 |
|
|
(1) Primarily related to a $4.0 million loss on the transfer of non-financial assets during the third quarter of 2019. |
X4 PHARMACEUTICALS, INC. | |||||||
CONDENSED CONSOLIDATED BALANCE SHEETS | |||||||
(in thousands) | |||||||
(Unaudited) | |||||||
September 30, 2019 |
December 31, 2018 |
||||||
Assets | |||||||
Current assets: | |||||||
Cash and cash equivalents |
$ |
76,251 |
$ |
8,134 |
|
||
Research and development incentive receivable |
|
1,730 |
|
— | |||
Prepaid expenses and other current assets |
|
1,234 |
|
|
1,205 |
|
|
Total current assets |
|
79,215 |
|
|
9,339 |
|
|
Property and equipment, net |
|
348 |
|
|
241 |
|
|
Goodwill |
|
27,109 |
|
— | |||
Right-of-use assets |
|
2,110 |
|
— | |||
Restricted cash |
|
755 |
|
|
364 |
|
|
Total assets |
$ |
109,537 |
|
$ |
9,944 |
|
|
Liabilities, convertible preferred stock, redeemable common stock and stockholders’ equity (deficit) | |||||||
Current liabilities: | |||||||
Accounts payable, accrued expenses and other current liabilities |
$ |
8,279 |
|
$ |
6,220 |
|
|
Current portion of long-term debt, net of discount | — |
|
1,687 |
|
|||
Total current liabilities |
|
8,279 |
|
|
7,907 |
|
|
Preferred stock warrant liability | — |
|
4,947 |
|
|||
Long-term debt, net |
|
19,986 |
|
|
8,145 |
|
|
Lease and other non-current liabilities |
|
2,165 |
|
|
622 |
|
|
Total liabilities |
|
30,430 |
|
|
21,621 |
|
|
Convertible preferred stock | — |
|
64,675 |
|
|||
Redeemable common stock | — |
|
734 |
|
|||
Total stockholders’ equity (deficit) |
|
79,107 |
|
|
(77,086 |
) |
|
Total liabilities, convertible preferred stock, redeemable common stock and stockholders’ equity (deficit) |
$ |
109,537 |
|
$ |
9,944 |
View source version on businesswire.com: https://www.businesswire.com/news/home/20191107005118/en/
Source:
Investors:
Stephanie Carrington
Westwicke, an ICR company
646-277-1282
Stephanie.Carrington@icrinc.com
Media:
Darcie Robinson
Westwicke, an ICR company
203-919-7905
Darcie.Robinson@icrinc.com