REGENXBIO Reports First Quarter 2016 Financial Results and Recent Operational Highlights
- On track to initiate a Phase I/II clinical trial for RGX-501 for the treatment of homozygous familial hypercholesterolemia in the first half of 2016
- Progressing toward filing an IND with the
U.S. Food and Drug Administration for RGX-111 for the treatment of Mucopolysaccharidosis Type I in the first half of 2016; expect to initiate a Phase I/II clinical trial in the third quarter of 2016 - First quarter 2016 cash burn of
$7.8 million , with cash, cash equivalents and marketable securities of$208.6 million as ofMarch 31, 2016 ; the Company continues to expect full year cash burn between$60 million and $70 million
“During the first quarter of 2016, we made significant progress toward this year’s goals of initiating two clinical studies and filing an Investigational New Drug application to support the initiation of a third study,” said
Recent Operational Highlights
REGENXBIO and trial sponsor theUniversity of Pennsylvania (Penn ) are recruiting participants in the Phase I/II clinical trial of RGX-501 for the treatment of homozygous familial hypercholesterolemia (HoFH). The Company entered into agreements withAdvanced BioScience Laboratories, Inc. (ABL), to support the transfer, process scale-up and production of RGX-501 for future use in clinical trials. The initial RGX-501 study drug to be used in the Phase I/II clinical trial was manufactured under a resource grant supported by theNational Institutes of Health (NIH) and awarded toPenn. - The Company is completing the initial production of RGX-111 for the treatment of Mucopolysaccharidosis Type I (MPS I) with
WuXi AppTec, Inc. , and is preparing for regulatory filing submissions in the U.S. andCanada to support the planned Phase I/II clinical trial for RGX-111. - The Company had meetings with regulatory authorities to discuss filing submissions for RGX-314 for the treatment of wet age-related macular degeneration (wet AMD), and RGX-121 for the treatment of Mucopolysaccharidosis Type II (MPS II). Preclinical data used in these meetings are being presented this week at the
American Society of Gene & Cell Therapy 19th Annual Meeting.REGENXBIO plans to file an Investigational New Drug application (IND) for RGX-314 in the second half of 2016, and an IND for RGX-121 in the first half of 2017. REGENXBIO expanded the organization to 63 full-time employees as ofMay 2, 2016 , including the following key executives dedicated to corporate initiatives and the progression of its lead programs:
Laura Coruzzi , Ph.D., J.D., Senior Vice President, Intellectual Property- More than 30 years of experience in all aspects of life sciences patent law.
- Previous positions include Partner in the intellectual property practice at
Jones Day and Partner in the biotechnology group atPennie & Edmonds, LLP .
Kimberly Sloan , Senior Vice President, Human Resources- More than 20 years of experience in domestic and global human resources.
- Previous positions include Vice President, Human Resources at
Biocon / MTF, and senior leadership roles at Princeton Financial Systems andDun & Bradstreet .
Rickey Reinhardt , M.D., Ph.D., Vice President,Clinical Research & Development - More than 20 years of experience in global clinical development.
- Previous positions include Vice President of Translational Medicine and Clinical Development in the rare disease unit at GlaxoSmithKline, and Vice President of
Global Molecule Development at Covance.
Financial Results
- Cash, cash equivalents and marketable securities as of
March 31, 2016 were$208.6 million , compared to$216.4 million as ofDecember 31, 2015 . - Revenues were
$0.4 million for the quarter endedMarch 31, 2016 , compared to$0.6 million for the quarter endedMarch 31, 2015 . - Total operating expenses were
$11.6 million for the quarter endedMarch 31, 2016 , compared to$4.6 million for the quarter endedMarch 31, 2015 . - Net loss was
$10.8 million , or$0.41 net loss per basic and diluted share, for the quarter endedMarch 31, 2016 , compared to$4.0 million , or$0.94 net loss per basic and diluted share, for the quarter endedMarch 31, 2015 .
Guidance
- The Company continues to expect full year 2016 cash burn to be between
$60 million and $70 million .
About
Forward Looking Statements
This press release contains "forward-looking statements," within the meaning of the Private Securities Litigation Reform Act of 1995, regarding, among other things,
All forward-looking statements contained in this press release are expressly qualified by the cautionary statements contained or referred to herein.
| REGENXBIO INC. | ||||||||
| BALANCE SHEETS | ||||||||
| (unaudited) | ||||||||
| (in thousands, except per share data) | ||||||||
| March 31, | December 31, | |||||||
| 2016 | 2015 | |||||||
| Assets | ||||||||
| Current assets | ||||||||
| Cash and cash equivalents | $ | 35,511 | $ | 54,116 | ||||
| Marketable securities | 65,544 | 60,025 | ||||||
| Accounts receivable | 2,036 | 2,136 | ||||||
| Prepaid expenses | 1,189 | 1,020 | ||||||
| Other current assets | 1,300 | 851 | ||||||
| Total current assets | 105,580 | 118,148 | ||||||
| Marketable securities | 107,553 | 102,226 | ||||||
| Property and equipment, net | 1,328 | 538 | ||||||
| Cost method investments | 300 | 300 | ||||||
| Other assets | 223 | 168 | ||||||
| Total assets | $ | 214,984 | $ | 221,380 | ||||
| Liabilities and Stockholders’ Equity | ||||||||
| Current liabilities | ||||||||
| Accounts payable | $ | 1,113 | $ | 1,014 | ||||
| Accrued expenses and other current liabilities | 4,633 | 3,198 | ||||||
| Advance payments | 35 | 127 | ||||||
| Total current liabilities | 5,781 | 4,339 | ||||||
| Deferred rent, net of current portion | 602 | 233 | ||||||
| Total liabilities | 6,383 | 4,572 | ||||||
| Stockholders’ equity | 208,601 | 216,808 | ||||||
| Total liabilities and stockholders’ equity | $ | 214,984 | $ | 221,380 | ||||
| REGENXBIO INC. | ||||||||
| STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS | ||||||||
| (unaudited) | ||||||||
| (in thousands, except per share data) | ||||||||
| Three Months Ended March 31, | ||||||||
| 2016 | 2015 | |||||||
| Revenues | ||||||||
| License revenue | $ | 328 | $ | 100 | ||||
| Reagent sales | 59 | 104 | ||||||
| Grant revenue | 6 | 440 | ||||||
| Total revenues | 393 | 644 | ||||||
| Expenses | ||||||||
| Costs of revenues | ||||||||
| Licensing costs (including amounts to related parties) | 66 | 20 | ||||||
| Costs of reagent sales (including amounts to related parties) | 30 | 33 | ||||||
| Research and development (including amounts to related parties) | 6,183 | 2,791 | ||||||
| General and administrative (including amounts to related parties) | 5,479 | 1,716 | ||||||
| Other operating expenses (income) | (114 | ) | 77 | |||||
| Total operating expenses | 11,644 | 4,637 | ||||||
| Loss from operations | (11,251 | ) | (3,993 | ) | ||||
| Other Income (Expense) | ||||||||
| Investment income | 483 | 2 | ||||||
| Interest expense | — | (20 | ) | |||||
| Total other income (expense) | 483 | (18 | ) | |||||
| Net loss | $ | (10,768 | ) | $ | (4,011 | ) | ||
| Other Comprehensive Income | ||||||||
| Unrealized gain on available-for-sale securities | 994 | — | ||||||
| Total other comprehensive income | 994 | — | ||||||
| Comprehensive loss | $ | (9,774 | ) | $ | (4,011 | ) | ||
| Reconciliation of net loss to net loss applicable to common stockholders | ||||||||
| Net loss | $ | (10,768 | ) | $ | (4,011 | ) | ||
| Net decretion and dividends on convertible preferred stock | — | 755 | ||||||
| Net gain on extinguishment of convertible preferred stock | — | 759 | ||||||
| Net loss applicable to common stockholders | $ | (10,768 | ) | $ | (2,497 | ) | ||
| Basic and diluted net loss per common share | $ | (0.41 | ) | $ | (0.94 | ) | ||
| Weighted-average basic and diluted common shares | 26,327 | 2,645 | ||||||
CONTACT: InvestorsElizabeth Broder , 646-378-2945 ebroder@troutgroup.com MediaLaura Bagby , 312-448-8098 lbagby@6degreespr.com
