X4 Pharmaceuticals Reports Second Quarter 2020 Financial Results and Provides Corporate Update
Presentation of positive clinical data at EHA 2020 supporting the ongoing Phase 3 trial in WHIM syndrome
Initial data expected later this year from Phase 1b clinical trial in Waldenström’s macroglobulinemia
Conference call today at
“Despite the challenges posed by the ongoing COVID-19 pandemic, we achieved significant progress during the second quarter, benefiting from two key events related to the WHIM syndrome indication for mavorixafor, while continuing to advance our program in the Waldenström’s indication,” said
“As we await these important results,”
Recent Highlights
- Increased Guidance on Prevalence of WHIM at Virtual Analyst Day, based on in-depth, internal market research indicating the range of diagnosed and undiagnosed WHIM patients in
the United States to be greater than 3,500, a significant increase from the prior estimate of approximately 1,000 patients diagnosed with WHIM syndrome. - Presented Positive Data from the Phase 2 Open-Label Extension Study of Mavorixafor in WHIM Syndrome at EHA 2020, supporting the selection of 400 mg once-daily and time above threshold for absolute neutrophil counts (TATANC) as the dose and primary endpoint in the Phase 3 trial, respectively, and long-term favorable tolerability. At the median follow-up of 16.5 months, data revealed sustained, dose-dependent increases in WBC (white blood cells), ANC (absolute neutrophil count), and ALC (absolute lymphocyte count), with higher doses of mavorixafor shown to increase the TATANC at least 4.5-fold versus lower doses. In patients treated for at least 6 months, mavorixafor also significantly decreased the yearly rate of infections versus the 12 months prior to treatment and effected a 75% reduction in cutaneous warts versus baseline.
- Promoted
Renato Skerlj , Ph.D., to the position of Chief Scientific Officer.Dr. Skerlj , one of the scientific founders of X4 and co-inventor of mavorixafor, has more than 25 years of experience leading the discovery and development of disease-modifying small molecule drugs to treat genetically defined rare diseases. In this expanded role,Dr. Skerlj leads all research and non-clinical development functions at X4, overseeing operations at the company’sVienna, Austria research facility, as well as the company’s efforts to advance and expand its pipeline targeting additional rare diseases.
Second Quarter 2020 Financial Results
- Cash, Cash Equivalents & Restricted Cash: X4 had
$105.6 million in cash, cash equivalents and restricted cash, as ofJune 30, 2020 . X4 continues to expect that its cash and cash equivalents will fund company operations into early 2022. In addition, X4 continues to have$25 million of potential borrowing capacity under its amended credit agreement with Hercules. - Research and Development Expenses were
$9.3 million for the second quarter endedJune 30, 2020 , as compared to$8.9 million for the comparable period in 2019. - General and Administrative Expenses were
$5.3 million for the second quarter endedJune 30, 2020 , as compared to$4.6 million for the comparable period in 2019. - Net Loss: X4 reported a net loss of
$15.1 million for the second quarter endedJune 30, 2020 , as compared to a net loss of$13.4 million for the comparable period in 2019.
Conference Call and Webcast
The Company will host a conference call and webcast today at
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. 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 anticipated and potential impact of the COVID-19 pandemic on X4’s business and operations, including the timing of its ongoing clinical trials; X4’s borrowing capacity under credit agreements; X4’s plans for clinical development of mavorixafor, including the timing of completion and results of its global Phase 3 clinical trial in patients with WHIM syndrome, its Phase 1b clinical trial in combination with ibrutinib in patients with Waldenström’s macroglobulinemia, and its Phase 1b clinical trial as monotherapy in patients with Severe Congenital Neutropenia; the expected timing of guidance and data disclosures on X4’s current clinical trials; and estimates regarding the WHIM patient population and potential market opportunity. 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, the risks and uncertainties 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 | ||||||||||||||
2020 | 2019 | 2020 | 2019 | ||||||||||||
License revenue | $ | - | $ | - | $ | 3,000 | $ | - | |||||||
Operating expenses: | |||||||||||||||
Research and development | 9,342 | 8,854 | 18,253 | 14,509 | |||||||||||
General and administrative | 5,316 | 4,560 | 9,986 | 9,343 | |||||||||||
Total operating expenses | 14,658 | 13,414 | 28,239 | 23,852 | |||||||||||
Loss from operations | (14,658 | ) | (13,414 | ) | (25,239 | ) | (23,852 | ) | |||||||
Other income (expense), net | (486 | ) | 31 | (895 | ) | (404 | ) | ||||||||
Loss before provision for income taxes | (15,144 | ) | (13,383 | ) | (26,134 | ) | (24,256 | ) | |||||||
Provision for income taxes | - | - | 148 | - | |||||||||||
Net loss | (15,144 | ) | (13,383 | ) | (26,282 | ) | (24,256 | ) | |||||||
Adjustments related to convertible preferred stock | - | - | - | (592 | ) | ||||||||||
Net loss attributable to common stockholders | $ | (15,144 | ) | $ | (13,383 | ) | $ | 26,282 | $ | (24,848 | ) | ||||
Net loss per share attributable to common stockholders- basic and diluted | $ | (0.76 | ) | $ | (1.02 | ) | $ | (1.31 | ) | $ | (3.32 | ) | |||
Weighted average common shares outstanding-basic and diluted | 20,032 | 13,177 | 20,016 | 7,479 | |||||||||||
CONDENSED CONSOLIDATED STATEMENTS OF CASH FLOWS | |||||||
(in thousands) | |||||||
Six months ended |
|||||||
2020 | 2019 | ||||||
Net loss | $ | (26,282 | ) | $ | (24,256 | ) | |
Adjustments to reconcile net loss to net cash used in operating activities | 2,591 | 1,457 | |||||
Changes in operating assets and liabilities | (3,295 | ) | (3,243 | ) | |||
Net cash used in operating activities | (26,986 | ) | (26,042 | ) | |||
Net cash (used in) provided by investing activities | (564 | ) | 26,396 | ||||
Net cash provided by financing activities | 5,049 | 86,791 | |||||
Impact of foreign exchange on cash, cash equivalents and restricted cash | 60 | (2 | ) | ||||
Net (decrease) increase in cash, cash equivalents and restricted cash | $ | (22,441 | ) | $ | 87,143 | ||
Cash, cash equivalents and restricted cash at beginning of period | $ | 128,086 | $ | 8,498 | |||
Cash, cash equivalents and restricted cash at end of period | $ | 105,645 | $ | 95,641 | |||
CONDENSED CONSOLIDATED BALANCE SHEETS | |||||||
(in thousands) | |||||||
Current assets: | |||||||
Cash and cash equivalents | $ | 103,744 | $ | 126,184 | |||
Research and development incentive receivable | 509 | 1,998 | |||||
Prepaid expenses and other current assets | 5,634 | 1,096 | |||||
Total current assets | 109,887 | 129,278 | |||||
Property and equipment, net | 421 | 403 | |||||
27,109 | 27,109 | ||||||
Right-of-use assets | 1,644 | 1,959 | |||||
Other assets | 3,473 | 1,949 | |||||
Total assets | $ | 142,534 | $ | 160,698 | |||
Current liabilities: | |||||||
Accounts payable | $ | 1,838 | $ | 2,088 | |||
Accrued expenses | 8,003 | 6,461 | |||||
Current portion of lease liabilities | 940 | 898 | |||||
Total current liabilities | 10,781 | 9,447 | |||||
Long-term debt, including accretion, net of discount | 25,398 | 20,097 | |||||
Lease liabilities | 1,445 | 1,918 | |||||
Other liabilities | 26 | 16 | |||||
Total liabilities | 37,650 | 31,478 | |||||
Total stockholders' equity | 104,884 | 129,220 | |||||
Total liabilities and stockholders' equity | $ | 142,534 | $ | 160,698 | |||
Investors and Media:
Director,
Candice.Ellis@x4pharma.com
Source: X4 Pharmaceuticals