REGENXBIO Reports Fourth Quarter and Full-Year 2017 Financial Results and Recent Operational Highlights
- Completed dosing of third cohort in RGX-314 Phase I clinical trial for wet AMD
- Continuing dosing of second cohort in RGX-501 Phase I/II clinical trial for HoFH
- Anticipate presenting topline data from RGX-314 and RGX-501 clinical trials in late 2018
- Expect to initiate dosing in clinical trials for RGX-111 for MPS I and RGX-121 for MPS II in mid-2018
$176 million in cash, cash equivalents and marketable securities as ofDecember 31, 2017
“In 2017, we significantly advanced our AAV gene therapy pipeline, which now consists of 12 active clinical stage programs, including four internal programs, as we seek to improve treatment options using our NAV Technology Platform,” said
Recent Operational Highlights
- In
February 2018 ,REGENXBIO announced the completion of dosing of the third cohort in the Phase I clinical trial for RGX-314 for the treatment of wet age-related macular degeneration (wet AMD). A total of 18 patients have been treated in the clinical trial to date.REGENXBIO expects to present topline data from the RGX-314 clinical trial in late 2018, which will include both primary and secondary endpoint data. - In
February 2018 ,REGENXBIO dosed the second patient in the second cohort, and fifth patient overall, with a single administration of RGX-501 in the Phase I/II clinical trial for the treatment of homozygous familial hypercholesterolemia (HoFH).REGENXBIO expects to present topline data from the RGX-501 clinical trial in late 2018, which will include both primary and secondary endpoint data. - Site activation is continuing in the Phase I clinical trial evaluating RGX-111 for the treatment of Mucopolysaccharidosis Type I (MPS I). Patient recruitment is anticipated to begin in the first quarter of 2018, with the first patient expected to be dosed in mid-2018.
- Site activation is continuing in the Phase I/II clinical trial evaluating RGX-121 for the treatment of Mucopolysaccharidosis Type II (MPS II). Patient recruitment is anticipated to begin in the first quarter of 2018, with the first patient expected to be dosed in mid-2018.
- In
January 2018 ,REGENXBIO andAveXis, Inc. announced an amendment to their license agreement for the development and commercialization of treatments for spinal muscular atrophy (SMA). Under the terms of the amended agreement,REGENXBIO could receive up to$260 million , including an upfront payment of$80 million ,$60 million in additional guaranteed annual payments and potential commercial milestone payments of up to$120 million , andAveXis acquired exclusive rights to the entire NAV Technology Platform for the development of treatments for SMA. Additionally, the amended agreement permits assignment byAveXis upon a change of control without REGENXBIO’s consent. - In
January 2018 ,REGENXBIO entered into an agreement with FUJIFILM Diosynth Biotechnologies which secures access to dedicated cGMP suite capacity and resources capable of manufacturing REGENXBIO’s lead product candidates at up to 2,000L scale in support of global development and commercialization.
As of
- In
January 2018 ,AveXis announced that it will initiate screening for remaining patients in the pivotal trial of AVXS-101 for SMA Type I following review of preliminary data from the first three patients. AVXS-101 uses the NAV AAV9 vector. - In
January 2018 ,Audentes Therapeutics, Inc. announced positive interim data from the first dose cohort in the Phase I/II clinical trial evaluating AT132 for the treatment of X-linked myotubular myopathy. AT132 uses the NAV AAV8 vector. - In
February 2018 ,Audentes Therapeutics announced dosing of the first patient in the Phase I/II clinical trial evaluating AT342 for the treatment of Crigler-Najjar Syndrome. AT342 uses the NAV AAV8 vector.
Financial Results
Cash, cash equivalents and marketable securities were
Revenues were
Total operating expenses were
Net loss was
Financial Guidance
About REGENXBIO Inc.
Forward-Looking Statements
This press release includes “forward-looking statements,” within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. These statements express a belief, expectation or intention and are generally accompanied by words that convey projected future events or outcomes such as “believe,” “may,” “will,” “estimate,” “continue,” “anticipate,” “design,” “intend,” “expect,” “could,” “plan,” “potential,” “predict,” “seek,” “should,” “would” or by variations of such words or by similar expressions. The forward-looking statements include statements relating to, among other things, REGENXBIO’s future operations, clinical trials, costs and cash flow.
| REGENXBIO INC. CONSOLIDATED BALANCE SHEETS (unaudited) (in thousands, except per share data) |
||||||||
| December 31, 2017 | December 31, 2016 | |||||||
| Assets | ||||||||
| Current assets | ||||||||
| Cash and cash equivalents | $ | 46,656 | $ | 24,840 | ||||
| Marketable securities | 114,122 | 64,714 | ||||||
| Accounts receivable | 473 | 1,032 | ||||||
| Prepaid expenses | 5,334 | 1,775 | ||||||
| Other current assets | 1,412 | 1,010 | ||||||
| Total current assets | 167,997 | 93,371 | ||||||
| Marketable securities | 15,616 | 69,412 | ||||||
| Property and equipment, net | 13,977 | 9,324 | ||||||
| Restricted cash | 225 | 225 | ||||||
| Other assets | 862 | 400 | ||||||
| Total assets | $ | 198,677 | $ | 172,732 | ||||
| Liabilities and Stockholders’ Equity | ||||||||
| Current liabilities | ||||||||
| Accounts payable | $ | 4,832 | $ | 1,543 | ||||
| Accrued expenses and other current liabilities | 9,605 | 8,126 | ||||||
| Total current liabilities | 14,437 | 9,669 | ||||||
| Deferred rent, net of current portion | 1,211 | 1,326 | ||||||
| Total liabilities | 15,648 | 10,995 | ||||||
| Stockholders’ equity | ||||||||
| Preferred stock; $0.0001 par value; 10,000 shares authorized, and no shares issued and outstanding at December 31, 2017 and December 31, 2016 |
— | — | ||||||
| Common stock; $0.0001 par value; 100,000 shares authorized at December 31, 2017 and December 31, 2016; 31,295 and 26,477 shares issued and outstanding at December 31, 2017 and December 31, 2016, respectively |
3 | 3 | ||||||
| Additional paid-in capital | 371,497 | 276,354 | ||||||
| Accumulated other comprehensive loss | (715 | ) | (33 | ) | ||||
| Accumulated deficit | (187,756 | ) | (114,587 | ) | ||||
| Total stockholders’ equity | 183,029 | 161,737 | ||||||
| Total liabilities and stockholders’ equity | $ | 198,677 | $ | 172,732 | ||||
| REGENXBIO INC. CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS (unaudited) (in thousands, except per share data) |
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| Three Months Ended December 31, | Years Ended December 31, | |||||||||||||||
| 2017 | 2016 | 2017 | 2016 | |||||||||||||
| Revenues | ||||||||||||||||
| License revenue | $ | 2,040 | $ | 1,665 | $ | 10,385 | $ | 4,303 | ||||||||
| Reagent sales | — | — | — | 213 | ||||||||||||
| Grant revenue | — | 31 | 8 | 73 | ||||||||||||
| Total revenues | 2,040 | 1,696 | 10,393 | 4,589 | ||||||||||||
| Expenses | ||||||||||||||||
| Costs of revenues | ||||||||||||||||
| Licensing costs | (382 | ) | 330 | 1,703 | 861 | |||||||||||
| Costs of reagent sales | — | — | 6 | 98 | ||||||||||||
| Research and development | 14,170 | 16,059 | 57,224 | 45,482 | ||||||||||||
| General and administrative | 4,808 | 5,742 | 27,229 | 23,590 | ||||||||||||
| Other operating expenses (income) | 42 | 34 | 116 | (102 | ) | |||||||||||
| Total operating expenses | 18,638 | 22,165 | 86,278 | 69,929 | ||||||||||||
| Loss from operations | (16,598 | ) | (20,469 | ) | (75,885 | ) | (65,340 | ) | ||||||||
| Other Income | ||||||||||||||||
| Investment income | 601 | 426 | 2,716 | 1,938 | ||||||||||||
| Total other income | 601 | 426 | 2,716 | 1,938 | ||||||||||||
| Loss before income taxes | (15,997 | ) | (20,043 | ) | (73,169 | ) | (63,402 | ) | ||||||||
| Income Tax Benefit | — | 435 | — | 435 | ||||||||||||
| Net loss | $ | (15,997 | ) | $ | (19,608 | ) | $ | (73,169 | ) | $ | (62,967 | ) | ||||
| Other Comprehensive Income (Loss) | ||||||||||||||||
| Unrealized gain (loss) on available-for-sale securities, net of reclassifications and income tax expense |
(161 | ) | (886 | ) | (682 | ) | 686 | |||||||||
| Total other comprehensive income (loss) | (161 | ) | (886 | ) | (682 | ) | 686 | |||||||||
| Comprehensive loss | $ | (16,158 | ) | $ | (20,494 | ) | $ | (73,851 | ) | $ | (62,281 | ) | ||||
| Basic and diluted net loss per common share | $ | (0.51 | ) | $ | (0.74 | ) | $ | (2.45 | ) | $ | (2.38 | ) | ||||
| Weighted-average basic and diluted common shares | 31,178 | 26,476 | 29,878 | 26,409 | ||||||||||||
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