X4 Pharmaceuticals Reports Fourth Quarter and Full Year 2020 Financial Results and Provides Corporate Update
2021 expected to be a year of key value-driving catalysts as company advances multiple clinical programs with lead candidate mavorixafor
Company anticipates completing full enrollment in WHIM Phase 3 trial in 2021 and presenting clinical data from the ongoing Phase 1b Waldenström’s trial throughout the year
Conference call today at
“As many companies did, we encountered a number of unexpected challenges in 2020, most notably the COVID-19 pandemic. However, I could not be prouder of our accomplishments, as we remained steadfast in our commitment to advance our lead candidate, mavorixafor, in a number of rare disease indications,” said
“On the clinical front, we continue to expect top-line data from our Phase 3 WHIM trial in 2022 and, as our Phase 1b Waldenström’s trial is progressing well, we continue to expect initial data from that study in the first half of 2021, with a more fulsome dataset expected towards the end of 2021. We also anticipate initial data from our SCN Phase 1b trial in 2021.”
Recent Highlights
- Received Fast Track and Rare Pediatric Designations (RPD) from the FDA for Mavorixafor for the treatment of WHIM Syndrome: These FDA designations further recognize WHIM as a serious condition with a clear unmet need in both adult and pediatric populations.
- Through the Fast Track program, X4 will be eligible for more frequent meetings with the FDA to discuss the drug’s development plan, protocols and clinical data that would support mavorixafor’s potential approval for WHIM.
- Under the RPD program, a sponsor who receives an approval for a drug for a “rare pediatric disease” and a Fast Track designation may qualify for a voucher that can be redeemed to receive a priority review by the FDA for any subsequent marketing application for a different product. Such a voucher is transferable and may be sold.
- 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.
- Strengthened Leadership Team: During the Fourth Quarter of 2020:
Diego Cadavid , M.D., joined as Chief Medical Officer, bringing more than 20 years of industry experience and having led multiple programs through all phases of clinical development, including small molecules and biologics for the treatment of rare and immunological diseases.Art Taveras , Ph.D., joined as Chief Scientific Officer with more than 30 years of experience leading small molecule research and development programs focused on cancer, dysregulated immune disorders, neurodegeneration, and metabolic diseases.Alison Lawton was appointed to the company’s Board of Directors, bringing more than 30 years of experience across a full spectrum of drug development and commercial roles and having previously served on X4’s corporate advisory board and as consulting Chief Operating Officer.
Fourth Quarter 2020 Financial Results
- Cash, Cash Equivalents & Restricted Cash: X4 had
$80.7 million in cash, cash equivalents and restricted cash as ofDecember 31, 2020 . X4 continues to expect that its cash and cash equivalents will fund company operations into 2022.
- Research and Development Expenses were
$12.3 million and$41.9 million for the fourth quarter and full year endedDecember 31, 2020 , respectively, as compared to$7.1 million and$30.2 million for the comparable periods in 2019, respectively. R&D expenses include$0.6 million and$2.3 million of certain non-cash expenses for the fourth quarter and fully year endedDecember 31, 2020 , respectively.
- General and Administrative Expenses were
$5.4 million and$20.9 million for the fourth quarter and full year endedDecember 31, 2020 , respectively, as compared to$3.9 million and$17.6 million for the comparable periods in 2019, respectively. G&A expenses include$0.8 million and$3.1 million of certain non-cash expenses for the fourth quarter and full year endedDecember 31, 2020 , respectively.
- Net Loss: X4 reported net losses of
$18.4 million and$62.1 million for the fourth quarter and full year endedDecember 31, 2020 as compared to net losses of$10.8 million and$52.8 million for the comparable periods in 2019, respectively. Net losses include$1.4 million and$5.4 million of certain non-cash expenses for the fourth quarter and full year endedDecember 31, 2020 , respectively. Net losses in the full year of 2019 included a$3.9 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 and X4's other product candidates or programs, the potential benefits resulting from Fast Track and Rare Pediatric Disease designations, 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 Quarterly Report on Form 10-Q filed with the
(Tables Follow)
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS
(in thousands, except per share amounts)
(unaudited)
Three Months Ended | Year Ended | ||||||||||||||
2020 | 2019 | 2020 | 2019 | ||||||||||||
License revenue | $ | — | $ | — | $ | 3,000 | $ | — | |||||||
Operating expenses: | |||||||||||||||
Research and development | 12,298 | 7,065 | 41,932 | 30,163 | |||||||||||
General and administrative | 5,357 | 3,914 | 20,942 | 17,640 | |||||||||||
Loss on transfer of non-financial assets | — | (104 | ) | — | 3,900 | ||||||||||
Total operating expenses | 17,655 | 10,875 | 62,874 | 51,703 | |||||||||||
Loss from operations | (17,655 | ) | (10,875 | ) | (59,874 | ) | (51,703 | ) | |||||||
Other income (expense), net | (745 | ) | 38 | (2,109 | ) | (1,104 | ) | ||||||||
Loss before provision for income taxes | (18,400 | ) | (10,837 | ) | (61,983 | ) | (52,807 | ) | |||||||
Provision for income taxes | — | — | 148 | — | |||||||||||
Net loss | (18,400 | ) | (10,837 | ) | (62,131 | ) | (52,807 | ) | |||||||
Adjustments related to convertible preferred stock | — | — | — | (592 | ) | ||||||||||
Net loss attributable to common stockholders | $ | (18,400 | ) | $ | (10,837 | ) | $ | (62,131 | ) | $ | (53,399 | ) | |||
Net loss per share attributable to common stockholders- basic and diluted | $ | (0.91 | ) | $ | (0.66 | ) | $ | (3.09 | ) | $ | (4.63 | ) | |||
Weighted average common shares outstanding-basic and diluted | 20,174 | 16,466 | 20,077 | 11,530 |
CONDENSED CONSOLIDATED STATEMENTS OF CASH FLOWS
(in thousands)
(unaudited)
Year ended |
|||||||
2020 | 2019 | ||||||
Net loss | $ | (62,131 | ) | $ | (52,807 | ) | |
Adjustments to reconcile net loss to net cash used in operating activities | 7,376 | 7,988 | |||||
Changes in operating assets and liabilities | (4,063 | ) | (3,236 | ) | |||
Net cash used in operating activities | (58,818 | ) | (48,055 | ) | |||
Net cash (used in) provided by investing activities | (1,362 | ) | 27,232 | ||||
Net cash provided by financing activities | 12,394 | 140,661 | |||||
Impact of foreign exchange on cash, cash equivalents and restricted cash | 402 | (250 | ) | ||||
Net (decrease) increase in cash, cash equivalents and restricted cash | (47,384 | ) | 119,588 | ||||
Cash, cash equivalents and restricted cash at beginning of period | 128,086 | 8,498 | |||||
Cash, cash equivalents and restricted cash at end of period | $ | 80,702 | $ | 128,086 |
CONSOLIDATED BALANCE SHEETS
(in thousands)
(unaudited)
Current assets: | |||||||
Cash and cash equivalents | $ | 78,708 | $ | 126,184 | |||
Research and development incentive receivable | 917 | 1,998 | |||||
Prepaid expenses and other current assets | 3,682 | 1,096 | |||||
Total current assets | 83,307 | 129,278 | |||||
Property and equipment, net | 1,237 | 403 | |||||
27,109 | 27,109 | ||||||
Right-of-use assets | 7,960 | 1,959 | |||||
Other assets | 3,258 | 1,949 | |||||
Total assets | $ | 122,871 | $ | 160,698 | |||
Current liabilities: | |||||||
Accounts payable | $ | 3,144 | $ | 2,088 | |||
Accrued expenses | 8,018 | 6,461 | |||||
Current portion of lease liability | 786 | 898 | |||||
Total current liabilities | 11,948 | 9,447 | |||||
Long-term debt, including accretion, net of discount | 33,178 | 20,097 | |||||
Lease liabilities | 4,484 | 1,918 | |||||
Other liabilities | 462 | 16 | |||||
Total liabilities | 50,072 | 31,478 | |||||
Total stockholders' equity | 72,799 | 129,220 | |||||
Total liabilities and stockholders' equity | $ | 122,871 | $ | 160,698 |
Contacts:
Managing Director
daniel@lifesciadvisors.com
(617) 430-7576
Senior Account Executive
mroucomolina@lifescicomms.com
Source: X4 Pharmaceuticals