CLEVELAND, March 18, 2024 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO) today reported financial results for the full year of 2023 and provided an update on progress toward achieving key corporate objectives.

克利夫兰，2024年3月18日（环球通讯社）--Abeona Therapeutics Inc。（纳斯达克：ABEO）今天公布了2023年全年的财务业绩，并提供了实现关键企业目标的最新进展。

Abeona also announced today that the U.S. Food and Drug Administration (FDA) has completed a Pre-License Inspection (PLI) of its Cleveland, Ohio manufacturing facility related to the Company’s Biologics License Application (BLA) for pz-cel (prademagene zamikeracel) for recessive dystrophic epidermolysis bullosa (RDEB).

Abeona今天还宣布，美国食品和药物管理局（FDA）已完成其俄亥俄州克利夫兰制造工厂的许可前检查（PLI），该工厂与该公司针对pz-cel（prademagene zamikeracel）的隐性营养不良性大疱性表皮松解症（RDEB）的生物制剂许可证申请（BLA）有关。

During the inspection, the FDA reviewed the facilities, systems, and processes at the Cleveland site. The FDA also observed the manufacturing process for pz-cel, as well as performance of in-process and release assays. The two-week PLI, which was conducted by five FDA inspectors, concluded on March 1, 2024.

在检查期间，FDA审查了克利夫兰工厂的设施、系统和流程。FDA还观察了pz-cel的制造过程，以及过程中和释放测定的性能。由五名FDA检查员进行的为期两周的物价指数于2024年3月1日结束。

Upon completion of the inspection, a Form 483 was issued with observations related to process controls. On March 15, 2024, the Company submitted a response to the FDA, outlining already implemented and ongoing steps toward resolution that follow FDA guidance provided during the audit. In addition, the FDA has completed the clinical study site inspections of Stanford University School of Medicine in Palo Alto, CA and University of Massachusets Medical School in Worcester, MA, both of which enrolled subjects in the pivotal Phase 3 VIITAL™ study supporting the pz-cel BLA, with no Form 483 observations noted.

检查完成后，发布了一份表格483，其中包含与过程控制有关的意见。2024年3月15日，该公司向美国食品和药物管理局提交了一份回复，概述了按照审计期间提供的美国食品和药物管理局指南已经实施和正在进行的解决步骤。此外，FDA已经完成了对加利福尼亚州帕洛阿尔托的斯坦福大学医学院和马萨诸塞州伍斯特的马萨丘塞茨大学医学院的临床研究现场检查，这两所大学都参加了支持pz cel-BLA的关键性3期VIITAL™研究，没有注意到483表观察结果。

The FDA’s review of Abeona’s pz-cel BLA is ongoing, with a target Prescription Drug User Fee Act (PDUFA) date of May 25, 2024, and the FDA does not currently plan to conduct an Advisory Committee..

FDA正在对Abeona的pz cel BLA进行审查，目标处方药使用费法案（PDUFA）日期为2024年5月25日，FDA目前不计划成立咨询委员会。。

“We appreciate the FDA’s collaborative conduct of the PLI, and look forward to working with them through the remainder of the BLA review.” said Vish Seshadri, Chief Executive Officer of Abeona.

Abeona首席执行官维什·塞沙德里（Vish Seshadri）表示：“我们赞赏FDA对PLI的合作行为，并期待在BLA审查的其余部分与他们合作。”。

Fourth Quarter and Recent Progress

第四季度和最新进展

Pz-cel for RDEB

RDEB的Pz cel

In November 2023, the FDA accepted and granted Priority Review for the Company’s BLA for pz-cel for RDEB with a target PDUFA date of May 25, 2024. The FDA also advised that it does not plan to convene an Advisory Committee meeting to discuss the pz-cel application.

2023年11月，美国食品和药物管理局接受并批准了该公司针对RDEB的pz cel BLA的优先审查，目标PDUFA日期为2024年5月25日。FDA还表示，它不打算召开咨询委员会会议来讨论pz cel申请。

In January 2024, the FDA completed both a Bioresearch Monitoring (BIMO) inspection of Abeona and the Mid-Cycle Meeting regarding the pz-cel BLA. Following completion of the BIMO inspection, the FDA inspector did not issue any observations or FDA Form 483s. At the BLA Mid-Cycle Review meeting, the FDA reaffirmed that it does not currently plan to convene an Advisory Committee for pz-cel.

2024年1月，FDA完成了对Abeona的生物研究监测（BIMO）检查和关于pz cel BLA的中期会议。完成BIMO检查后，FDA检查员没有发布任何观察结果或FDA表格483s。在BLA中期审查会议上，FDA重申目前不计划为pz cel召开咨询委员会。

In addition, the FDA advised that Risk Evaluation and Mitigation Strategies (REMS) are not anticipated for the pz-cel BLA at this time, though review is ongoing, and reconfirmed the target PDUFA date of May 25, 2024..

此外，美国食品和药物管理局建议，目前预计pz cel BLA不会进行风险评估和缓解策略（REMS），尽管正在进行审查，并再次确认PDUFA的目标日期为2024年5月25日。。

In March 2024, Abeona received a written Establishment Inspection Report (EIR), the formal report from the FDA regarding the BIMO inspection. The results of the inspection determined there were no deficiencies with the monitoring or management of study sites, IP accountability, retention of study site documents, or safety oversight..

2024年3月，Abeona收到了一份书面机构检查报告（EIR），这是FDA关于BIMO检查的正式报告。检查结果表明，在研究地点的监测或管理、知识产权责任、保留研究地点文件或安全监督方面没有缺陷。。

U.S. commercial launch preparations for pz-cel

U、 pz cel的美国商业发射准备

Abeona continues to advance key commercial activities in preparation for a potential U.S. launch for pz-cel, including onboarding discussions with EB treatment sites, payer engagement, hiring key commercial roles, and conducting market research.

Abeona继续推进关键商业活动，为pz cel在美国的潜在推出做准备，包括与EB治疗网站的入职讨论、付款人参与、雇佣关键商业角色以及进行市场研究。

The Company recently completed payer market research that supports reimbursement coverage of pz-cel at a price commensurate with the value of approved gene therapies that offer years of durable benefit following a one-time application.

该公司最近完成了付款人市场研究，该研究支持pz-cel的报销范围，其价格与经批准的基因疗法的价值相当，该疗法在一次性应用后可提供多年的持久益处。

Corporate highlights

企业亮点

In January 2024, Abeona entered a $50 million credit facility with the Avenue Venture Opportunities Fund, L.P. The credit agreement includes a first tranche of $20 million, which was funded in January 2024, a second tranche of $10 million of committed capital, and an option for an additional $20 million upon satisfaction of certain terms and conditions.

2024年1月，Abeona与Avenue Venture Opportunities Fund，L.P.签订了5000万美元的信贷协议。该信贷协议包括2024年1月出资的第一批2000万美元，第二批1000万美元的承诺资本，以及在满足某些条款和条件后额外获得2000万美元的期权。

Proceeds from the facility are intended to support the Company’s ongoing preparations for launch and commercialization in anticipation of a potential FDA approval of pz-cel..

该设施的收益旨在支持该公司正在进行的上市和商业化准备，以期获得FDA对pz cel的潜在批准。。

Full Year 2023 Financial Results and Cash Runway Guidance

2023年全年财务业绩和现金跑道指南

Cash, cash equivalents, restricted cash and short-term investments totaled $52.6 million as of December 31, 2023, compared to $52.5 million as of December 31, 2022. Net cash used in operating activities was $37.0 million for the full year of 2023, compared to $43.5 million in the full year of 2022.

截至2023年12月31日，现金、现金等价物、限制性现金和短期投资总额为5260万美元，而截至2022年12月31日为5250万美元。2023年全年用于经营活动的净现金为3700万美元，而2022年全年为4350万美元。

In January 2024, Abeona received a first tranche of $20 million as part of the credit facility with Avenue Venture Opportunities Fund, L.P. Abeona estimates that its current cash and cash equivalents, restricted cash and short-term investments, as well as the credit facility with Avenue Venture Opportunities Fund, L.P, are sufficient resources to fund operations into the first quarter of 2025 before accounting for any revenue from commercial sales of pz-cel, if approved, or proceeds from the sale of a Priority Review Voucher, if awarded by the FDA..

2024年1月，作为Avenue Venture Opportunities Fund，L.P.信贷安排的一部分，Abeona收到了第一批2000万美元。Abeona估计其当前现金和现金等价物、受限现金和短期投资，以及Avenue Venture Opportunities Fund，L.P.的信贷安排，在考虑pz cel商业销售收入（如果批准）或优先审查凭证销售收入（如果FDA授予）之前，有足够的资源为2025年第一季度的运营提供资金。。

License and other revenues for the year ended December 31, 2023 were $3.5 million, as compared to $1.4 million for the same period of 2022. The revenues in both years primarily represent clinical milestone payments under a licensing agreement with Taysha Gene Therapies for investigational AAV-based gene therapy for Rett syndrome..

截至2023年12月31日止年度的许可证和其他收入为350万美元，而2022年同期为140万美元。这两年的收入主要是根据与Taysha Gene Therapes签订的许可协议，为Rett综合征的基于AAV的研究性基因治疗支付的临床里程碑付款。。

Research and development expenses for the full year ended December 31, 2023 were $31.1 million, compared to $29.0 million for the full year ended December 31, 2022, primarily a result of increased headcount related to BLA activities. General and administrative expenses were $19.0 million for the full year ended December 31, 2023, compared to $17.3 million for the year ended December 31, 2022, primarily a result of increased headcount for the potential launch of pz-cel.

截至2023年12月31日的全年研发费用为3110万美元，而截至2022年12月31日的全年研发费用为2900万美元，这主要是由于与BLA活动相关的人数增加所致。截至2023年12月31日的全年一般和管理费用为1900万美元，而截至2022年12月31日的全年为1730万美元，这主要是由于pz cel的潜在推出增加了员工人数。

Net loss attributable to common shareholders for the full year ended December 31, 2023 was $54.2 million, or $2.53 loss per common share as compared to $43.5 million, or $5.53 loss per common share, for the full year of 2022..

截至2023年12月31日的全年归属于普通股股东的净亏损为5420万美元，即每股普通股亏损2.53美元，而2022年全年为4350万美元，即每股普通股亏损5.53美元。。

Conference Call Details

电话会议详细信息

Abeona Therapeutics will host a conference call and webcast today, March 18, 2024, at 8:30 a.m. ET. To access the call, dial 888-506-0062 (U.S. toll-free) or 973-528-0011 (international) and Entry Code: 428606 five minutes prior to the start of the call. A live, listen-only webcast and archived replay of the call can be accessed at https://investors.abeonatherapeutics.com/events.

Abeona Therapeutics将于今天2024年3月18日美国东部时间上午8:30主持电话会议和网络广播。要拨打电话，请在通话开始前五分钟拨打888-506-0062（美国免费电话）或973-528-0011（国际电话），输入代码：428606。您可以通过以下网址访问实时的、仅供收听的网络广播和存档的重播：https://investors.abeonatherapeutics.com/events.

The archived webcast replay will be available for 30 days following the call..

通话结束后，存档的网络广播重播将持续30天。。

About Abeona Therapeutics

关于Abeona Therapeutics

Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing cell and gene therapies for serious diseases. The U.S. FDA has accepted and granted Priority Review with a PDUFA target action date of May 25, 2024 for the Biologics License Application for pz-cel (prademagene zamikeracel), Abeona’s investigational autologous, COL7A1 gene-corrected epidermal sheets currently in development for recessive dystrophic epidermolysis bullosa.

Abeona Therapeutics Inc.是一家临床阶段的生物制药公司，开发针对严重疾病的细胞和基因疗法。美国食品和药物管理局（FDA）已接受并批准了pz-cel（prademagene zamikeracel）生物制剂许可证申请的PDUFA目标行动日期为2024年5月25日的优先审查，pz-cel是Abeona的研究性自体COL7A1基因校正表皮片，目前正在开发用于隐性营养不良性大疱性表皮松解症。

The Company’s fully integrated cell and gene therapy cGMP manufacturing facility served as the manufacturing site for pz-cel used in its Phase 3 VIITAL™ trial, and is capable of supporting commercial production of pz-cel upon FDA approval. The Company’s development portfolio also features AAV-based gene therapies for ophthalmic diseases with high unmet medical need.

该公司的完全集成的细胞和基因治疗cGMP生产设施是其3期VIITAL™试验中使用的pz-cel的生产基地，并且能够在FDA批准后支持pz-cel的商业生产。该公司的开发组合还包括基于AAV的基因疗法，用于高度未满足医疗需求的眼科疾病。

Abeona’s novel, next-generation AAV capsids are being evaluated to improve tropism profiles for a variety of devastating diseases. For more information, visit www.abeonatherapeutics.com..

正在评估Abeona的新型下一代AAV衣壳，以改善各种破坏性疾病的趋向性。有关更多信息，请访问www.abeonatherapeutics.com。。

Forward-Looking Statements

前瞻性声明

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.

本新闻稿包含经修订的《1933年证券法》第27A节和经修订的《1934年证券交易法》第21E节所指的前瞻性声明，并涉及风险和不确定性。我们试图通过“可能”、“将”、“相信”、“预期”、“预期”、“打算”、“潜在”等术语以及类似的词语和表达（以及其他涉及未来事件、条件或情况的词语或表达）来识别前瞻性陈述，这些词语构成并旨在识别前瞻性陈述。

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; the FDA’s grant of a Priority Review Voucher; continued interest in our rare disease portfolio; our ability to enroll patients in clinical trials; the outcome of future meetings with and inspections from 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 Securities and Exchange Commission.

由于各种重要因素，众多风险和不确定性，包括但不限于我们向FDA提交pz cel生物制剂许可证申请的时间和结果，实际结果可能与此类前瞻性声明所示的结果存在重大差异；FDA授予优先审查凭证；对我们罕见疾病组合的持续兴趣；我们招募患者参加临床试验的能力；与FDA或其他监管机构（包括与临床前计划有关的机构）未来会议和检查的结果；获得或获得必要监管批准的能力；金融市场和全球经济状况的任何变化的影响；与数据分析和报告相关的风险；以及公司最新的10-K表年度报告以及随后提交给美国证券交易委员会的定期报告中披露的其他风险。

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 press release, whether as a result of new information, future developme.

公司没有义务修改前瞻性声明或更新前瞻性声明，以反映本新闻稿发布日期后发生的事件或情况，无论是由于新信息、未来发展。

ABEONA THERAPEUTICS INC. AND SUBSIDIARIES

ABEONA THERAPEUTICS INC.及其子公司

Consolidated Statements of Operations and Comprehensive Loss

合并经营报表和综合损失

(In thousands, except share and per share amounts)

（单位：千，股份和每股金额除外）

For the years ended December 31,

截至12月31日止年度，

2023

2023

2022

2022

Revenues:

收入：

License and other revenues

许可证和其他收入

$

$

3,500

3,500

$

$

1,414

1,414

Expenses:

费用：

Royalties

版税

1,605

1,605

450

450

Research and development

研究与开发

31,091

31,091

28,965

28,965

General and administrative

一般和行政

19,004

19,004

17,256

17,256

Impairment of licensed technology

许可技术减值

—

—

1,355

1,355

Loss/(gain) on operating lease right-of-use assets

经营租赁使用权资产损失/（收益）

(1,065

(1,065

)

)

2,511

2,511

Impairment of construction-in-progress

在建工程减值

—

—

1,792

1,792

Total expenses

总费用

50,635

50,635

52,329

52,329

Loss from operations

运营损失

(47,135

(47,135

)

)

(50,915

(50,915

)

)

Interest income

利息收入

2,117

2,117

431

431

Interest expense

利息支出

(418

(418

)

)

(736

(736

)

)

Change in fair value of warrant liabilities

权证负债公允价值变动

(11,695

(11,695

)

)

11,383

11,383

Other income

其他收入

2,943

2,943

141

141

Net loss

净损失

$

$

(54,188

(54,188

)

)

$

$

(39,696

(39,696

)

)

Deemed dividends related to Series A and Series B Convertible Redeemable Preferred Stock

与A系列和B系列可转换可赎回优先股相关的视同股息

—

—

(3,782

(3,782

)

)

Net loss attributable to Common Shareholders

归属于普通股股东的净亏损

$

$

(54,188

(54,188

)

)

$

$

(43,478

(43,478

)

)

Basic and diluted loss per common share

每股普通股基本损失和稀释损失

$

$

(2.53

(2.53

)

)

$

$

(5.53

(5.53

)

)

Weighted average number of common

公用工程的加权平均数

shares outstanding – basic and diluted

流通股–基本股和稀释股

21,380,476

21,380,476

7,861,515

7,861,515

Other comprehensive income (loss):

其他综合收益（损失）：

Change in unrealized gains (losses) related to available-for-sale debt securities

与可供出售债务证券相关的未实现收益（损失）变化

34

34

(99

(99

)

)

Foreign currency translation adjustments

外币折算调整

29

29

(3

(3

)

)

Comprehensive loss

综合损失

$

$

(54,125

(54,125

)

)

$

$

(43,580

(43,580

)

)

ABEONA THERAPEUTICS INC. AND SUBSIDIARIES

ABEONA THERAPEUTICS INC.及其子公司

Consolidated Balance Sheets

合并资产负债表

(In thousands, except share and per share amounts)

（单位：千，股份和每股金额除外）

December 31,

12月31日，

2023

2023

December 31,

12月31日，

2022

2022

ASSETS

资产

Current assets:

流动资产：

Cash and cash equivalents

现金及现金等价物

$

$

14,473

14,473

$

$

14,217

14,217

Short-term investments

短期投资

37,753

37,753

37,932

37,932

Restricted cash

受限现金

338

338

338

338

Other receivables

其他应收款

2,444

2,444

188

188

Prepaid expenses and other current assets

预付费用和其他流动资产

729

729

424

424

Total current assets

流动资产总额

55,737

55,737

53,099

53,099

Property and equipment, net

财产和设备净值

3,533

3,533

5,741

5,741

Operating lease right-of-use assets

经营租赁使用权资产

4,455

4,455

5,331

5,331

Other assets

其他资产

277

277

43

43

Total assets

总资产

$

$

64,002

64,002

$

$

64,214

64,214

LIABILITIES AND STOCKHOLDERS' EQUITY

负债和股东权益

Current liabilities:

流动负债：

Accounts payable

应付账款

$

$

1,858

1,858

$

$

1,811

1,811

Accrued expenses

应计费用

5,985

5,985

3,991

3,991

Current portion of operating lease liability

经营租赁负债的当前部分

998

998

1,773

1,773

Current portion of payable to licensor

4,580

4,580

—

—

Other current liabilities

其他流动负债

1

1

204

204

Total current liabilities

流动负债合计

13,422

13,422

7,779

7,779

Payable to licensor

应付给许可方

—

—

4,163

4,163

Long-term operating lease liabilities

长期经营租赁负债

4,402

4,402

5,854

5,854

Warrant liabilities

认股权证负债

31,352

31,352

19,657

19,657

Total liabilities

负债总额

49,176

49,176

37,453

37,453

Commitments and contingencies

承诺和或有事项

Stockholders' equity:

股东权益：

Preferred stock - $0.01 par value; authorized 2,000,000 shares; No shares issued and outstanding as of December 31, 2023 and December 31, 2022, respectively

优先股——面值0.01美元；授权2000000股；截至2023年12月31日和2022年12月31日，分别没有发行和流通的股份

—

—

—

—

Common stock - $0.01 par value; authorized 200,000,000 shares; 26,523,878 and 17,719,720 shares issued and outstanding as of December 31, 2023 and December 31, 2022, respectively

普通股——面值0.01美元；授权200000000股；截至2023年12月31日和2022年12月31日，分别发行26523878股和17719720股

265

265

177

177

Additional paid-in capital

额外实收资本

764,151

764,151

722,049

722,049

Accumulated deficit

累积赤字

(749,524

(749,524

)

)

(695,336

(695,336

)

)

Accumulated other comprehensive loss

累积其他综合损失

(66

(66

)

)

(129

(129

)

)

Total stockholders' equity

股东权益合计

14,826

14,826

26,761

26,761

Total liabilities and stockholders' equity

总负债和股东权益

$

$

64,002

64,002

$

$

64,214

64,214

Investor and Media Contact:

投资者和媒体联系人：

Greg Gin

格雷格·金

VP, Investor Relations and Corporate Communications

投资者关系和企业传播副总裁

Abeona Therapeutics

Abeona治疗学

ir@abeonatherapeutics.com

ir@abeonatherapeutics.com