Full Press Release Details
Therapeutics Reports Second Quarter Financial Results and Business Updates
patients dosed in RDEB, MPS IIIA and MPS IIIB clinical programs
alignment with CHMP on plans for EU registration of ABO-102 for MPS IIIA
from RDEB and MPS III programs presented at recent medical meetings
leadership team with appointment of experienced Chief Commercial Officer and two independent Board members
call on Tuesday, August 11, 2020 at 8:30 a.m. ET
YORK and CLEVELAND, August 10, 2020 - Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader in gene and cell
therapy, today announced financial results for the second quarter 2020 and recent business progress.
the past several months, Abeona has delivered on our goals in clinical development, manufacturing, and regulatory affairs toward
bringing urgently needed treatments to patients with RDEB and MPS III," said Jo o Siffert, M.D., Chief Executive
Officer of Abeona. "Notably, new patients have been treated in our RDEB and MPS IIIA clinical programs, and we expect additional
patient enrollment across our clinical programs in the coming weeks. Concurrent with the increased clinical activities, we resumed
internal manufacturing operations at our Cleveland campus in June. We have made significant advancements in process development
for retrovirus and AAV manufacturing in-house, anticipated to start in late-2020 and early-2021, respectively. In addition, we
recently reached general alignment with the CHMP on our proposed path toward a European marketing authorization application for
ABO-102 in MPS IIIA, anticipated in 2023. Looking ahead to the potential commercial launches of EB-101 in late-2022 and ABO-102
the following year, we have also strengthened our leadership team, bringing on board a Chief Commercial Officer with significant
relevant experience and expertise."
Quarter and Recent Highlights
(Autologous, Gene-Corrected Cell Therapy)
| A second patient was treated in Abeona's EB-101 pivotal Phase 3 VIITAL study for recessive dystrophic epidermolysis bullosa (RDEB) after enrollment resumed in June 2020 following a pause due to the COVID-19 pandemic. Additional patients are expected to be treated in the coming weeks, with completion of enrollment in the VIITAL study expected in early-2021. | |
| Two posters were presented at the Society for Pediatric Dermatology 45 th Annual Meeting. The first poster highlighted data showing that EB-101 treatment of large RDEB wounds resulted in up to five years of durable healing, which was associated with long-term pain relief. A separate poster characterized the significant disease burden of RDEB on patients and their families. |
and ABO-101 (AAV-based Gene Therapies)
| In July 2020, Abeona held a kick-off meeting under the European Medicines Agency's (EMA) PRIority MEdicines (PRIME) program, which included members of the Committee for Advanced Therapies (CAT) and the Committee for Medicinal Products for Human Use (CHMP) of the EMA. The Company presented its plan toward registration of ABO-102 for MPS IIIA (Sanfilippo syndrome type A), taking advantage of the PRIME designation that offers a path for accelerated assessment of promising therapies targeting unmet medical needs. Based on the PRIME meeting, along with previous input from the CHMP and the Pediatric Committee (PDCO) of the EMA, Abeona anticipates submitting a marketing authorization application for EU conditional approval of ABO-102 for MPS IIIA in 2023. The Company continues to seek guidance from the FDA on the U.S. regulatory path for ABO-102 in MPS IIIA, but acknowledges that the FDA is currently focused on matters related to COVID-19 and other life-threatening conditions, as reflected in its issued guidance in May 2020. | |
| Total enrollment to date in the ABO-102 Transpher A study for MPS IIIA is 17 patients, including 11 patients dosed in cohort 3. Total enrollment to date in the ABO-101 Transpher B study for MPS IIIB (Sanfilippo syndrome type B) is 9 patients, including 2 patients dosed in cohort 3. Abeona anticipates completing enrollment in both the Transpher A and Transpher B studies by the end of 2020. | |
| Updated positive interim data from the Transpher A and Transpher B studies were presented at the American Society of Gene & Cell Therapy 23rd Annual Meeting. The findings support previously reported data showing preservation of neurocognitive skills among three young patients treated in dose cohort 3 of the Transpher A study. Improvements in multiple disease-specific biomarkers, denoting clear biologic effects, and a favorable safety profile in MPS IIIA and MPS IIIB patients after treatment with ABO-102 and ABO-101, respectively, were observed in both studies. |
Quarter Financial Results
cash equivalents and short-term investments totaled $107.9 million as of June 30, 2020, compared to $129.3 million as of December
31, 2019. Net cash used in operating activities was $9.5 million for the second quarter 2020.
and development (R&D) spending was $6.1 million for the second quarter of 2020 and $12.9 million for the six months ended
June 30, 2020, compared to $16.3 million and $28.0 million in the comparable periods in 2019. The decrease in R&D expenses
was primarily due to decreased manufacturing, clinical and non-clinical development activities arising from the effects of the
COVID-19 pandemic, and cost savings associated with the decision to internally manufacture retrovirus for the EB-101 program.
and administrative (G&A) expenses were $5.5 million for the second quarter of 2020 and $12.0 million for the six months ended
June 30, 2020, compared to $5.6 million and $11.3 million in the comparable periods in 2019. The increase in G&A expenses
for the six months ended June 30, 2020 was largely due to increases in salary and related costs partially offset by decreased
loss was $13.0 million for the second quarter of 2020 and $61.2 million for the six months ended June 30, 2020, compared to net
loss of $23.9 million and $42.5 million for the comparable periods in 2019.
Therapeutics will host a conference call and webcast tomorrow, Tuesday, August 11, 2020 at 8:30 a.m. ET, to discuss its second
quarter 2020 financial results and provide an update on the company's business. To access the call, dial 844-369-8770 (U.S.
toll-free) or 862-298-0840 (international) and provide conference ID 18965539 five minutes prior to the start of the call. A live,
listen-only webcast and archived replay of the call can be accessed on the Investors & Media section of Abeona's website
at www.abeonatherapeutics.com. The archived webcast replay will be available for 30 days following the call.
Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene and cell therapies for serious diseases. Abeona's
clinical programs include EB-101, its autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa in
Phase 3 development, as well as ABO-102 and ABO-101, novel AAV-based gene therapies for Sanfilippo syndrome types A and B (MPS
IIIA and MPS IIIB), respectively, in Phase 1/2 development. The Company's portfolio of AAV-based gene therapies also features
ABO-202 and ABO-201 for CLN1 disease and CLN3 disease, respectively. Abeona's novel, next-generation AIM capsids
have shown potential to improve tropism profiles for a variety of devastating diseases. Abeona's fully functional, gene
and cell therapy GMP manufacturing facility produces EB-101 for the pivotal Phase 3 VIITAL study and is capable of clinical
and commercial production of AAV-based gene therapies. For more information, visit www.abeonatherapeutics.com.
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.
These statements include statements about the Company's clinical trials and its products and product candidates, future
regulatory interactions with regulatory authorities, as well as the Company's goals and objectives. We have attempted to
identify forward looking statements by such terminology as "may," "will," "believe," "estimate,"
"expect," and similar 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 potential impacts of the COVID-19 pandemic on our business, operations, and financial condition,
continued interest in our rare disease portfolio, our ability to enroll patients in clinical trials, the outcome of any future
meetings with the U.S. Food and Drug Administration or other regulatory agencies, the impact of competition, the ability to secure
licenses for any technology that may be necessary to commercialize our products, the ability to achieve or obtain necessary regulatory
approvals, the impact of changes in the financial markets and global economic conditions, risks associated with data analysis
and reporting, and other risks as may be detailed from time to time in the Company's Annual Reports on Form 10-K and quarterly
reports on Form 10-Q and other periodic reports filed by the Company with the Securities and Exchange Commission. The Company
undertakes no obligation to revise the forward-looking statements or to update them to reflect events or circumstances occurring
after the date of this presentation, whether as a result of new information, future developments or otherwise, except as required
by the federal securities laws.
Director, Corporate Communications
| Abeona Therapeutics Inc. and Subsidiaries | ||||||||||||||||
| Condensed Consolidated Statements of Operations and Comprehensive Loss | ||||||||||||||||
| (unaudited) | ||||||||||||||||
| For the three months ended June 30, | For the six months ended June 30, | |||||||||||||||
| 2020 | 2019 | 2020 | 2019 | |||||||||||||
| Revenues | $ | - | $ | - | $ | - | $ | - | ||||||||
| Expenses: | ||||||||||||||||
| Research and development | 6,109,000 | 16,307,000 | 12,927,000 | 28,044,000 | ||||||||||||
| General and administrative | 5,538,000 | 5,612,000 | 11,950,000 | 11,271,000 | ||||||||||||
| Depreciation and amortization | 834,000 | 2,057,000 | 2,899,000 | 3,715,000 | ||||||||||||
| Licensed technology impairment charge | - | - | 32,916,000 | - | ||||||||||||
| Total expenses | 12,481,000 | 23,976,000 | 60,692,000 | 43,030,000 | ||||||||||||
| Loss from operations | (12,481,000 | ) | (23,976,000 | ) | (60,692,000 | ) | (43,030,000 | ) | ||||||||
| Interest and miscellaneous income | 271,000 | 52,000 | 923,000 | 551,000 | ||||||||||||
| Interest expense | (800,000 | ) | - | (1,400,000 | ) | - | ||||||||||
| Net loss | $ | (13,010,000 | ) | $ | (23,924,000 | ) | $ | (61,169,000 | ) | $ | (42,479,000 | ) | ||||
| Basic and diluted loss per common share | $ | (0.14 | ) | $ | (0.49 | ) | $ | (0.66 | ) | $ | (0.88 | ) | ||||
| Weighted average number of common shares outstanding - basic and diluted | 93,180,837 | 48,961,988 | 92,910,014 | 48,458,004 | ||||||||||||
| Other comprehensive income/(loss): | ||||||||||||||||
| Change in unrealized (losses) gains related to available-for-sale debt securities | (253,000 | ) | - | 133,000 | - | |||||||||||
| Comprehensive loss | $ | (13,263,000 | ) | $ | (23,924,000 | ) | $ | (61,036,000 | ) | $ | (42,479,000 | ) |
| Abeona Therapeutics Inc. and Subsidiaries | ||||||||
| Condensed Consolidated Balance Sheets | ||||||||
| (unaudited) | ||||||||
| June 30, 2020 | December 31, 2019 | |||||||
| ASSETS | ||||||||
| Current assets: | ||||||||
| Cash and cash equivalents | $ | 14,542,000 | $ | 129,258,000 | ||||
| Short-term investments | 93,337,000 | - | ||||||
| Prepaid expenses and other current assets | 924,000 | 3,132,000 | ||||||
| Total current assets | 108,803,000 | 132,390,000 | ||||||
| Property and equipment, net | 12,628,000 | 13,157,000 | ||||||
| Right-of-use lease assets | 7,551,000 | 8,047,000 | ||||||
| Licensed technology, net | 1,924,000 | 36,178,000 | ||||||
| Goodwill | 32,466,000 | 32,466,000 | ||||||
| Other assets and restricted cash | 1,006,000 | 1,144,000 | ||||||
| Total assets | $ | 164,378,000 | $ | 223,382,000 | ||||
| LIABILITIES AND STOCKHOLDERS' EQUITY | ||||||||
| Current liabilities: | ||||||||
| Accounts payable | $ | 1,979,000 | $ | 3,763,000 | ||||
| Accrued expenses | 3,017,000 | 5,543,000 | ||||||
| Loan payable | 1,758,000 | - | ||||||
| Current portion of lease liability | 1,706,000 | 1,699,000 | ||||||
| Payable to licensor | 28,800,000 | 27,400,000 | ||||||
| Deferred revenue | 296,000 | 296,000 | ||||||
| Total current liabilities | 37,556,000 | 38,701,000 | ||||||
| Long-term lease liabilities | 5,768,000 | 6,251,000 | ||||||
| Total liabilities | 43,324,000 | 44,952,000 | ||||||
| Commitments and contingencies | ||||||||
| Stockholders' equity: | ||||||||
| Common stock - $0.01 par value; authorized 200,000,000 shares; issued and outstanding 84,781,241 at June 30, 2020 and 83,622,135 at December 31, 2019 | 848,000 | 836,000 | ||||||
| Additional paid-in capital | 667,712,000 | 664,064,000 | ||||||
| Accumulated deficit | (547,639,000 | ) | (486,470,000 | ) | ||||
| Accumulated other comprehensive income | 133,000 | - | ||||||
| Total stockholders' equity | 121,054,000 | 178,430,000 | ||||||
| Total liabilities and stockholders' equity | $ | 164,378,000 | $ | 223,382,000 |