X4 Pharmaceuticals Reports Third Quarter 2020 Financial Results and Provides Corporate Update
WHIM Phase 2 data published in ‘Blood’ - the
Granted Fast Track Designation by FDA for mavorixafor in WHIM syndrome
Key leadership appointments including CSO and Board Director
Conference call today at
“The third quarter was highlighted by the publication of our positive Phase 2 safety and efficacy data for mavorixafor in WHIM syndrome in the prestigious journal, Blood, which, we believe, continues to demonstrate the significant potential of our lead candidate to treat this patient population,” said
Recent Highlights
- Published comprehensive data from the Phase 2 and open label extension study of mavorixafor in WHIM syndrome in Blood, the official journal of the
American Society of Hematology , expanding on previously presented data. New results included long-term patient-level data regarding the effect on neutrophils and lymphocytes and the effect of increasing doses of mavorixafor on total white blood cell counts and monocytes. In addition, the manuscript provided the most up-to-date pharmacokinetic data and presented a detailed analysis of the clinical improvements in infection rates and warts demonstrated with extended mavorixafor treatment.
- Granted Fast Track Designation by the FDA for mavorixafor in WHIM syndrome. Through the Fast Track program, X4 will be eligible for more frequent meetings with the FDA to discuss mavorixafor’s development plan, protocols and clinical data that would support its potential approval for WHIM. Mavorixafor was previously granted Breakthrough Therapy Designation by the FDA, as well as Orphan Drug status by the FDA and the European Commission for the treatment of WHIM syndrome
- Appointed
Art Taveras , Ph.D., as Chief Scientific Officer.Dr. Taveras is a seasoned Chief Scientific Officer with experience in the discovery of novel, next-generation CXCR2 antagonists for the treatment of cancer. Previously,Dr. Taveras was VP, Small Molecule Drug Discovery at Biogen, President and CSO of ShangPharma ChemPartners, and CSO of CoMET Therapeutics. With his expertise in chemokine-related chemistries,Dr. Taveras is well suited to lead X4’s R&D initiatives and its continued evolution into a global rare disease company.
- Expanded Board of Directors through the appointment of biopharmaceutical industry veteran,
Alison Lawton .Ms. Lawton strengthens the board’s drug development, manufacturing and commercialization experience and brings a unique understanding of X4’s core scientific and corporate goals, having previously served as a consulting Chief Operating Officer to the company and as a member of X4’s corporate advisory board.Ms. Lawton had an over 20 year tenure at Genzyme Corporation (now Sanofi Genzyme) including Senior Vice President and General Manager of Sanofi Biosurgery, a $750 million business and led global functional organizations, including regulatory affairs and quality systems, public policy, health outcomes and strategic pricing, product safety and risk management. Ms. Lawton most recently served as CEO of Kaleido Biosciences and currently serves as an independent director on the Board of ProQR Therapeutics NV.
Third Quarter 2020 Financial Results
- Cash, Cash Equivalents & Restricted Cash: X4 had
$90.7 million in cash, cash equivalents and restricted cash, as ofSeptember 30, 2020 . X4 continues to expect that its cash and cash equivalents will fund company operations into early 2022. Note this guidance does not include the$25 million in potential and contingent proceeds from our Hercules Debt Facility or any cash exercise proceeds from investors holding our outstanding warrants.
- Research and Development Expenses were
$11.4 million for the third quarter endedSeptember 30, 2020 , as compared to$8.6 million for the comparable period in 2019. R&D expenses include$1.0 million of certain non-cash expenses for the quarter endedSeptember 30, 2020 .
- General and Administrative Expenses were
$5.6 million for the third quarter endedSeptember 30, 2020 , as compared to$4.4 million for the comparable period in 2019. G&A expenses include$1.4 million of certain non-cash expenses for the quarter endedSeptember 30, 2020 .
- Net Loss: X4 reported a net loss of
$17.4 million for the third quarter endedSeptember 30, 2020 , as compared to a net loss of$17.7 million for the comparable period in 2019. Net loss includes$2.5 million of certain non-cash expenses for the quarter endedSeptember 30, 2020 . Net loss in the third quarter of 2019 included a$4.0 million loss on the sale of non-financial assets.
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, WHIM, Waldenström’s macroglobulinemia, SCN or X4's other product candidates or programs, and fast track designation. 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)
(unaudited)
Three Months Ended | Nine Months Ended | ||||||||||||||
2020 | 2019 | 2020 | 2019 | ||||||||||||
License revenue | $ | — | $ | — | $ | 3,000 | $ | — | |||||||
Operating expenses: | |||||||||||||||
Research and development | 11,381 | 8,589 | 29,634 | 23,098 | |||||||||||
General and administrative | 5,599 | 4,383 | 15,585 | 13,726 | |||||||||||
Loss on transfer of non-financial assets | — | 4,004 | — | 4,004 | |||||||||||
Total operating expenses | 16,980 | 16,976 | 45,219 | 40,828 | |||||||||||
Loss from operations | (16,980 | ) | (16,976 | ) | (42,219 | ) | (40,828 | ) | |||||||
Other expense, net | (469 | ) | (738 | ) | (1,364 | ) | (1,142 | ) | |||||||
Loss before provision for income taxes | (17,449 | ) | (17,714 | ) | (43,583 | ) | (41,970 | ) | |||||||
Provision for income taxes | — | — | 148 | — | |||||||||||
Net loss | (17,449 | ) | (17,714 | ) | (43,731 | ) | (41,970 | ) | |||||||
Adjustments related to convertible preferred stock | — | — | — | (592 | ) | ||||||||||
Net loss attributable to common stockholders | $ | (17,449 | ) | $ | (17,714 | ) | $ | (43,731 | ) | $ | (42,562 | ) | |||
Net loss per share attributable to common stockholders- basic and diluted | $ | (0.87 | ) | $ | (1.22 | ) | $ | (2.18 | ) | $ | (4.31 | ) | |||
Weighted average common shares outstanding-basic and diluted | 20,085 | 14,562 | 20,035 | 9,866 |
CONDENSED CONSOLIDATED STATEMENTS OF CASH FLOWS
(in thousands)
(unaudited)
Nine months ended |
|||||||
2020 | 2019 | ||||||
Net loss | $ | (43,731 | ) | $ | (41,970 | ) | |
Adjustments to reconcile net loss to net cash used in operating activities | 5,198 | 7,132 | |||||
Changes in operating assets and liabilities | (2,781 | ) | (3,976 | ) | |||
Net cash used in operating activities | (41,314 | ) | (38,814 | ) | |||
Net cash (used in) provided by investing activities | (1,060 | ) | 27,211 | ||||
Net cash provided by financing activities | 4,765 | 80,377 | |||||
Impact of foreign exchange on cash, cash equivalents and restricted cash | 240 | (266 | ) | ||||
Net (decrease) increase in cash, cash equivalents and restricted cash | $ | (37,369 | ) | $ | 68,508 | ||
Cash, cash equivalents and restricted cash at beginning of period | $ | 128,086 | $ | 8,498 | |||
Cash, cash equivalents and restricted cash at end of period | $ | 90,717 | $ | 77,006 |
CONDENSED CONSOLIDATED BALANCE SHEETS
(in thousands)
(unaudited)
Current assets: | |||||||
Cash and cash equivalents | $ | 88,812 | $ | 126,184 | |||
Research and development incentive receivable | 629 | 1,998 | |||||
Prepaid expenses and other current assets | 4,472 | 1,096 | |||||
Total current assets | 93,913 | 129,278 | |||||
Property and equipment, net | 1,334 | 403 | |||||
27,109 | 27,109 | ||||||
Right-of-use assets | 8,222 | 1,959 | |||||
Other assets | 2,548 | 1,949 | |||||
Total assets | $ | 133,126 | $ | 160,698 | |||
Current liabilities: | |||||||
Accounts payable | $ | 3,891 | $ | 2,088 | |||
Accrued expenses | 8,532 | 6,461 | |||||
Current portion of lease liability | 756 | 898 | |||||
Total current liabilities | 13,179 | 9,447 | |||||
Long-term debt, including accretion, net of discount | 25,537 | 20,097 | |||||
Lease liabilities | 4,677 | 1,918 | |||||
Other liabilities | 25 | 16 | |||||
Total liabilities | 43,418 | 31,478 | |||||
Total stockholders' equity | 89,708 | 129,220 | |||||
Total liabilities and stockholders' equity | $ | 133,126 | $ | 160,698 |
Investors and Media:
Managing Director
daniel@lifesciadvisors.com
(617) 430-7576
Senior Account Executive
mroucomolina@lifescicomms.com
Source: X4 Pharmaceuticals