Abeona’s first-ever
FDA decision on priority review and acceptance of BLA for pz-cel (prademagene zamikeracel, formerly known as EB-101) in recessive dystrophic epidermolysis bullosa (RDEB) expected by
Initiated commercial readiness activities for potential
“In the third quarter, we made substantial progress in our evolution from a late-stage clinical development company to one with significant commercial opportunity,” said
Third Quarter and Recent Progress
Pz-cel for RDEB
- The proposed non-proprietary (generic) name prademagene zamikeracel (referred to as “pz-cel” going forward) was approved by the
World Health Organization as the International Nonproprietary Name for Abeona’s investigational autologous, COL7A1 gene-corrected epidermal sheets formerly known as EB-101. - In August, Abeona completed a positive pre-BLA meeting in which it reached alignment with the
U.S. Food and Drug Administration (FDA) that the pz-cel clinical efficacy and safety data appear adequate to support a BLA submission. The FDA also agreed that retroviral vector manufactured atAbeona andIndiana University appear comparable based on the data that Abeona provided in its briefing book. - In September, Abeona submitted a BLA to FDA seeking approval with priority review of pz-cel for RDEB. The FDA’s decision on BLA acceptance is typically made during the 60-day window following submission. If accepted with Priority Review, Abeona expects potential BLA approval in the second quarter of 2024.
Strengthened balance sheet, initiated
- Abeona raised
$25 million in a registered direct offering priced at-the-market with select existing institutional investors to primarily fund the Company’s initial commercial preparations in support of the potentialU.S. launch of pz-cel. - Appointed Madhav Vasanthavada, Ph.D., M.B.A. as Chief Commercial Officer and Head of Business Development. Dr. Vasanthavada brings over 20 years of diverse leadership experience in the life sciences industry with recent experience in launching autologous cell therapies at
Bristol Myers Squibb (BMS) and Celgene. - Started
U.S. commercial launch planning activities for pz-cel, including initiating onboarding discussions with EB treatment sites, payer engagement, and hiring key commercial roles.
Third Quarter Financial Results
Cash, cash equivalents, restricted cash and short-term investments totaled
Research and development expenses for the three months ended
Conference Call Details
About
Forward-Looking Statements
This press release contains certain statements that are forward-looking 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, and that involve risks and uncertainties. We have attempted to identify forward-looking statements by such terminology as “may,” “will,” “believe,” “anticipate,” “expect,” “intend,” “potential,” and similar words and expressions (as well as other words or expressions referencing future events, conditions or circumstances), which constitute and are intended to identify forward-looking statements. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, numerous risks and uncertainties, including but not limited to, the timing and outcome of our Biologics License Application submission to the FDA for pz-cel; continued interest in our rare disease portfolio; our ability to enroll patients in clinical trials; the outcome of future meetings with the FDA or other regulatory agencies, including those relating to preclinical programs; the ability to achieve or obtain necessary regulatory approvals; the impact of any changes in the financial markets and global economic conditions; risks associated with data analysis and reporting; and other risks disclosed in the Company’s most recent Annual Report on Form 10-K and subsequent periodic reports filed with the
Condensed Consolidated Statements of Operations and Comprehensive Loss
(In thousands, except share and per share amounts)
(Unaudited)
For the three months ended | For the nine months ended | |||||||||||||||
2023 | 2022 | 2023 | 2022 | |||||||||||||
Revenues: | ||||||||||||||||
License and other revenues | $ | — | $ | — | $ | 3,500 | $ | 1,346 | ||||||||
Expenses: | ||||||||||||||||
Royalties | 30 | — | 1,605 | 350 | ||||||||||||
Research and development | 7,148 | 5,490 | 23,712 | 22,693 | ||||||||||||
General and administrative | 4,156 | 3,890 | 13,174 | 11,574 | ||||||||||||
Impairment of licensed technology | — | — | — | 1,355 | ||||||||||||
Loss/(gain) on right-of-use lease assets | — | — | (1,065 | ) | 1,561 | |||||||||||
Impairment of construction-in-progress | — | — | — | 1,792 | ||||||||||||
Total expenses | 11,334 | 9,380 | 37,426 | 39,325 | ||||||||||||
Loss from operations | (11,334 | ) | (9,380 | ) | (33,926 | ) | (37,979 | ) | ||||||||
Interest income | 593 | 72 | 1,374 | 110 | ||||||||||||
Interest expense | (105 | ) | (157 | ) | (309 | ) | (558 | ) | ||||||||
Change in fair value of warrant liabilities | (1,101 | ) | 3,050 | (7,465 | ) | 5,995 | ||||||||||
Other income (expense) | 111 | (19 | ) | 2,729 | (143 | ) | ||||||||||
Net loss | $ | (11,836 | ) | $ | (6,434 | ) | $ | (37,597 | ) | $ | (32,575 | ) | ||||
Deemed dividends related to Series A and Series B Convertible Redeemable Preferred Stock | — | — | — | (3,782 | ) | |||||||||||
Net loss attributable to Common Shareholders | $ | (11,836 | ) | $ | (6,434 | ) | $ | (37,597 | ) | $ | (36,357 | ) | ||||
Basic and diluted loss per common share | $ | (0.48 | ) | $ | (1.00 | ) | $ | (1.89 | ) | $ | (6.05 | ) | ||||
Weighted average number of common | ||||||||||||||||
shares outstanding – basic and diluted | 24,797,564 | 6,421,245 | 19,942,613 | 6,009,902 | ||||||||||||
Other comprehensive income (loss): | ||||||||||||||||
Change in unrealized gains (losses) related to available-for-sale debt securities | (33 | ) | (4 | ) | 1 | (11 | ) | |||||||||
Foreign currency translation adjustments | 29 | (6 | ) | 29 | (6 | ) | ||||||||||
Comprehensive loss | $ | (11,840 | ) | $ | (6,444 | ) | $ | (37,567 | ) | $ | (36,374 | ) | ||||
Condensed Consolidated Balance Sheets
(In thousands, except share and per share amounts)
(Unaudited) | |||||||
ASSETS | |||||||
Current assets: | |||||||
Cash and cash equivalents | $ | 4,712 | $ | 14,217 | |||
Short-term investments | 49,042 | 37,932 | |||||
Restricted cash | 338 | 338 | |||||
Other receivables | 2,209 | 188 | |||||
Prepaid expenses and other current assets | 963 | 424 | |||||
Total current assets | 57,264 | 53,099 | |||||
Property and equipment, net | 3,999 | 5,741 | |||||
Right-of-use lease assets | 4,685 | 5,331 | |||||
Other assets | 139 | 43 | |||||
Total assets | $ | 66,087 | $ | 64,214 | |||
LIABILITIES AND STOCKHOLDERS' EQUITY | |||||||
Current liabilities: | |||||||
Accounts payable | $ | 2,592 | $ | 1,811 | |||
Accrued expenses | 3,972 | 3,991 | |||||
Current portion of lease liability | 1,649 | 1,773 | |||||
Other current liabilities | 199 | 204 | |||||
Total current liabilities | 8,412 | 7,779 | |||||
Payable to licensor | 4,472 | 4,163 | |||||
Long-term lease liabilities | 4,043 | 5,854 | |||||
Warrant liabilities | 27,122 | 19,657 | |||||
Total liabilities | 44,049 | 37,453 | |||||
Commitments and contingencies | |||||||
Stockholders' equity: | |||||||
Preferred stock - | — | — | |||||
Common stock - | 247 | 177 | |||||
Additional paid-in capital | 754,823 | 722,049 | |||||
Accumulated deficit | (732,933 | ) | (695,336 | ) | |||
Accumulated other comprehensive loss | (99 | ) | (129 | ) | |||
Total stockholders' equity | 22,038 | 26,761 | |||||
Total liabilities and stockholders' equity | $ | 66,087 | $ | 64,214 | |||
Investor and Media Contact:Greg Gin VP, Investor Relations and Corporate CommunicationsAbeona Therapeutics ir@abeonatherapeutics.com
Source:
2023 GlobeNewswire, Inc., source