X4 Pharmaceuticals Reports First Quarter Financial Results and Provides Corporate Update
Company continues to make significant clinical progress with lead candidate mavorixafor
across multiple rare disease indications
Enrollment update on Phase 3 WHIM trial expected in mid-2021
Initial data from ongoing Phase 1b trial in Waldenström’s macroglobulinemia to be presented at
EHA Meeting in
Conference call today at
“We are excited by the strong progress we have achieved to date in 2021 in the development of mavorixafor across multiple rare disease indications,” said
Recent Highlights
- Mavorixafor Phase 1b Data in Waldenström’s to be Presented at EHA 2021. X4 has received notification of acceptance of an abstract for a poster presentation at this year’s annual congress of the
European Hematology Association (EHA), taking place virtually fromJune 9-17, 2021 . The abstract and poster will include initial clinical safety and efficacy data from the ongoing Phase 1b clinical trial of mavorixafor in combination with ibrutinib in the treatment of a subset of Waldenström’s macroglobulinemia patients with mutations to both the MYD88 and CXCR4 genes. - Enrollment Update in 4WHIM Clinical Trial Expected to be Announced in Mid-2021. The 4WHIM Phase 3 trial is a randomized, double-blind, placebo-controlled, multicenter study designed to evaluate the safety and efficacy of mavorixafor in approximately 18-28 genetically confirmed WHIM patients over the course of a 52-week study. The primary endpoint for the trial will compare the level of circulating neutrophils relative to a clinically meaningful threshold in response to treatment with mavorixafor versus placebo. Secondary endpoints will assess infection rates, wart burden, and markers of immune system function and quality of life among others. Based on the current rate of enrollment, X4 expects to provide an important enrollment update on the trial in the second or third quarter of 2021.
- Raised
$55.0 Million in an At-the-Market PIPE Financing. InMarch 2021 , X4 priced a$55 million at-the-market private placement financing at$8.70 per common share. The financing transaction included participation from leading biotechnology investors that are both new and existing X4 shareholders. The financing extended the company’s guidance on its expected cash runway into the fourth quarter of 2022.
First Quarter 2021 Financial Results
- Cash, Cash Equivalents & Restricted Cash: X4 had
$116.7 million in cash, cash equivalents and restricted cash as ofMarch 31, 2021 . X4 expects that its cash and cash equivalents will fund company operations into the fourth quarter of 2022. - Research and Development Expenses were
$12.1 million for the first quarter endedMarch 31, 2021 , as compared to$8.9 million for the comparable period in 2020. R&D expenses include$0.6 million and$0.2 million of certain non-cash expenses for the quarters endedMarch 31, 2021 and 2020, respectively. - General and Administrative Expenses were
$5 .8 million for the first quarter endedMarch 31, 2021 , as compared to$4.7 million for the comparable period in 2020. G&A expenses include$0.7 million and$0.4 million of certain non-cash expenses for the quarters endedMarch 31, 2021 and 2020, respectively. - Net Loss: X4 reported a net loss of
$18.7 million for the quarter endedMarch 31, 2021 , as compared to a net loss of$11.1 million for the comparable period in 2020. Net losses include$1.3 million and$0.6 million of certain non-cash expenses for the quarters endedMarch 31, 2021 and 2020, respectively.
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 clinical development of mavorixafor and X4’s other product candidates or programs 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, the risks and uncertainties described in the section entitled “Risk Factors” in X4’s Annual Report on Form 10-K filed with the
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS
(in thousands, except per share amounts)
(unaudited)
Three Months Ended | |||||||||
2021 | 2020 | ||||||||
License revenue | $ | — | $ | 3,000 | |||||
Operating expenses: | |||||||||
Research and development | 12,104 | 8,911 | |||||||
General and administrative | 5,832 | 4,670 | |||||||
Total operating expenses | 17,936 | 13,581 | |||||||
Loss from operations | (17,936 | ) | (10,581 | ) | |||||
Other expense, net | (734 | ) | (409 | ) | |||||
Loss before provision for income taxes | (18,670 | ) | (10,990 | ) | |||||
Provision for income taxes | 6 | 148 | |||||||
Net loss | (18,676 | ) | (11,138 | ) | |||||
Deemed dividend due to Class B warrant price reset | (8,239 | ) | — | ||||||
Net loss attributable to common stockholders | $ | (26,915 | ) | $ | (11,138 | ) | |||
Net loss per share attributable to common stockholders- basic and diluted | $ | (1.30 | ) | $ | (0.56 | ) | |||
Weighted average common shares outstanding-basic and diluted | 20,751 | 20,014 | |||||||
CONDENSED CONSOLIDATED STATEMENTS OF CASH FLOWS
(in thousands)
(unaudited)
Three months ended |
|||||||||
2021 | 2020 | ||||||||
Net loss | $ | (18,676 | ) | $ | (11,138 | ) | |||
Adjustments to reconcile net loss to net cash used in operating activities | 1,955 | 1,158 | |||||||
Changes in operating assets and liabilities | (1,723 | ) | (5,547 | ) | |||||
Net cash used in operating activities | (18,444 | ) | (15,527 | ) | |||||
Net cash used in provided by investing activities | (496 | ) | (555 | ) | |||||
Net cash provided by financing activities | 55,041 | 4,984 | |||||||
Impact of foreign exchange on cash, cash equivalents and restricted cash | (143 | ) | (34 | ) | |||||
Net increase (decrease) in cash, cash equivalents and restricted cash | 35,958 | (11,132 | ) | ||||||
Cash, cash equivalents and restricted cash at beginning of period | 80,702 | 128,086 | |||||||
Cash, cash equivalents and restricted cash at end of period | $ | 116,660 | $ | 116,954 | |||||
CONSOLIDATED BALANCE SHEETS
(in thousands)
(unaudited)
Current assets: | |||||||||
Cash and cash equivalents | $ | 114,945 | $ | 78,708 | |||||
Research and development incentive receivable | 768 | 917 | |||||||
Prepaid expenses and other current assets | 4,246 | 3,682 | |||||||
Total current assets | 119,959 | 83,307 | |||||||
Property and equipment, net | 1,788 | 1,237 | |||||||
27,109 | 27,109 | ||||||||
Right-of-use assets | 9,788 | 7,960 | |||||||
Other assets | 2,391 | 3,258 | |||||||
Total assets | $ | 161,035 | $ | 122,871 | |||||
Current liabilities: | |||||||||
Accounts payable | $ | 3,091 | $ | 3,144 | |||||
Accrued expenses | 10,706 | 8,018 | |||||||
Current portion of lease liability | 875 | 786 | |||||||
Total current liabilities | 14,672 | 11,948 | |||||||
Long-term debt, including accretion, net of discount | 33,356 | 33,178 | |||||||
Lease liabilities | 5,579 | 4,484 | |||||||
Other liabilities | 491 | 462 | |||||||
Total liabilities | 54,098 | 50,072 | |||||||
Redeemable common shares | 1,875 | — | |||||||
Total stockholders’ equity | 105,062 | 72,799 | |||||||
Total liabilities, redeemable common shares and stockholders' equity | $ | 161,035 | $ | 122,871 | |||||
Contacts:
Managing Director
LifeSci Advisors
daniel@lifesciadvisors.com
(617) 430-7576
Senior Account Executive
mroucomolina@lifescicomms.com
Source: X4 Pharmaceuticals