REGENXBIO Reports First Quarter 2018 Financial Results and Recent Operational Highlights
"We are looking forward to continuing to demonstrate the potential of
Recent Operational Highlights
- In
February 2018 ,REGENXBIO announced the completion of dosing of the third cohort in the Phase I clinical trial of RGX-314 for the treatment of wet age-related macular degeneration (wet AMD). To date, 18 patients have been treated with RGX-314.REGENXBIO expects to present topline data from the RGX-314 clinical trial in late 2018, which will include both primary and secondary endpoint data. REGENXBIO completed dosing of the third patient in the second cohort in the Phase I/II clinical trial of RGX-501 for the treatment of homozygous familial hypercholesterolemia (HoFH). To date, six patients have been treated with RGX-501.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). Initiation of patient recruitment and dosing of the first patient in the clinical trial are expected in mid-2018.
- In
May 2018 ,REGENXBIO announced that theU.S. Food and Drug Administration had granted Fast Track designation for RGX-121. Site activation is continuing in the Phase I/II clinical trial evaluating RGX-121 for the treatment of Mucopolysaccharidosis Type II (MPS II). Initiation of patient recruitment and dosing of the first patient in the clinical trial are expected in mid-2018.
- In
January 2018 ,REGENXBIO andAveXis, Inc. amended 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 , of which$80 million was received inJanuary 2018 . In addition to the$80 million ,REGENXBIO will receive payments of$30 million on the first and second anniversaries of the agreement and is eligible to receive potential commercial milestone payments of up to$120 million . For any product developed for the treatment of SMA using the NAV AAV9 vector,REGENXBIO will continue to receive mid-single to low double-digit royalties on net sales, and for any product developed for the treatment of SMA using a NAV vector other than NAV AAV9,REGENXBIO will receive a low double-digit royalty on net sales. - In
April 2018 ,AveXis announced that it had entered into an agreement and plan of merger pursuant to which it will be acquired byNovartis AG for$218 per share or a total of approximately$8.7 billion in cash, pending certain closing conditions. In the event the transaction betweenAveXis andNovartis is completed,REGENXBIO expects to be entitled to receive accelerated license payments of$100 million as a result of the change of control ofAveXis . - In
April 2018 ,AveXis also announced that 11 patients had been enrolled in the pivotal trial for AVXS-101.AveXis reported that the six patients who were at least one-month post gene transfer were exhibiting motor function improvements that correlate to motor function improvements experienced in patients in the Phase I clinical trial of AVXS-101. In addition,AveXis announced that the first patient has been dosed in a Phase III trial evaluating AVXS-101 in pre-symptomatic patients with SMA Types 1, 2 and 3. - In
April 2018 ,Ultragenyx Pharmaceutical Inc. announced that the investigational new drug application is active for DTX401 for the treatment of glycogen storage disease type Ia. DTX401 uses the NAV AAV8 vector.
Financial Results
Cash, cash equivalents and marketable securities were
Revenues were
Research and development expenses were
General and administrative expenses were
Net income 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 INC. |
||||||||
CONSOLIDATED BALANCE SHEETS |
||||||||
(unaudited) |
||||||||
(in thousands, except per share data) |
||||||||
March 31, 2018 |
December 31, 2017 |
|||||||
Assets |
||||||||
Current assets |
||||||||
Cash and cash equivalents |
$ |
71,870 |
$ |
46,656 |
||||
Marketable securities |
157,997 |
114,122 |
||||||
Accounts receivable |
25,976 |
473 |
||||||
Prepaid expenses |
4,667 |
5,334 |
||||||
Other current assets |
2,208 |
1,412 |
||||||
Total current assets |
262,718 |
167,997 |
||||||
Marketable securities |
5,917 |
15,616 |
||||||
Accounts receivable |
32,645 |
— |
||||||
Property and equipment, net |
14,829 |
13,977 |
||||||
Restricted cash |
225 |
225 |
||||||
Other assets |
883 |
862 |
||||||
Total assets |
$ |
317,217 |
$ |
198,677 |
||||
Liabilities and Stockholders' Equity |
||||||||
Current liabilities |
||||||||
Accounts payable |
$ |
5,007 |
$ |
4,832 |
||||
Accrued expenses and other current liabilities |
9,869 |
9,605 |
||||||
Total current liabilities |
14,876 |
14,437 |
||||||
Deferred rent, net of current portion |
1,225 |
1,211 |
||||||
Other liabilities |
1,776 |
— |
||||||
Total liabilities |
17,877 |
15,648 |
||||||
Stockholders' equity |
||||||||
Preferred stock; $0.0001 par value; 10,000 shares authorized, |
— |
— |
||||||
Common stock; $0.0001 par value; 100,000 shares authorized |
3 |
3 |
||||||
Additional paid-in capital |
378,954 |
371,497 |
||||||
Accumulated other comprehensive loss |
(903) |
(715) |
||||||
Accumulated deficit |
(78,714) |
(187,756) |
||||||
Total stockholders' equity |
299,340 |
183,029 |
||||||
Total liabilities and stockholders' equity |
$ |
317,217 |
$ |
198,677 |
REGENXBIO INC. |
||||||||
CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE INCOME (LOSS) |
||||||||
(unaudited) |
||||||||
(in thousands, except per share data) |
||||||||
Three Months Ended March 31, |
||||||||
2018 |
2017 |
|||||||
Revenues |
||||||||
License revenue |
$ |
132,391 |
$ |
455 |
||||
Total revenues |
132,391 |
455 |
||||||
Expenses |
||||||||
Costs of revenues |
||||||||
Licensing costs |
2,408 |
91 |
||||||
Research and development |
19,550 |
16,619 |
||||||
General and administrative |
8,380 |
6,622 |
||||||
Other operating expenses |
28 |
45 |
||||||
Total operating expenses |
30,366 |
23,377 |
||||||
Income (loss) from operations |
102,025 |
(22,922) |
||||||
Other Income |
||||||||
Interest income from licensing |
1,355 |
— |
||||||
Investment income |
859 |
929 |
||||||
Total other income |
2,214 |
929 |
||||||
Net income (loss) |
$ |
104,239 |
$ |
(21,993) |
||||
Other Comprehensive Loss |
||||||||
Unrealized loss on available-for-sale securities, |
(188) |
(539) |
||||||
Total other comprehensive loss |
(188) |
(539) |
||||||
Comprehensive income (loss) |
$ |
104,051 |
$ |
(22,532) |
||||
Net income (loss) applicable to common stockholders |
$ |
104,239 |
$ |
(21,993) |
||||
Net income (loss) per share: |
||||||||
Basic |
$ |
3.30 |
$ |
(0.82) |
||||
Diluted |
$ |
3.04 |
$ |
(0.82) |
||||
Weighted-average common shares outstanding: |
||||||||
Basic |
31,632 |
26,673 |
||||||
Diluted |
34,275 |
26,673 |
CONTACT:
Investors
natalie@argotpartners.com
Media
apawluk@jpa.com
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