Taysha Gene Therapies, Inc.

DALLAS, April 05, 2024 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a clinical-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of severe monogenic diseases of the central nervous system (CNS), today announced that, on April 1, 2024, the Compensation Committee of Taysha's Board of Directors granted three new employees, in the aggregate, options to purchase 140,000 shares of the Company's common stock in connection with their employment. The stock options were granted under the Taysha Gene Therapies, Inc. 2023 Inducement Plan as an inducement material to the individuals entering employment with Taysha in accordance with Nasdaq Listing Rule 5635(c)(4). The stock options have an exercise price of $2.84 per share, which is equal to the closing price of Taysha's common stock on the date of grant. The stock option has a 10-year term and will vest over four years, with 25% of the option vesting on the first anniversary of the vesting commencement date and the remaining 75% of the option vesting in equal monthly installments over the 36 months thereafter. Vesting of the stock option is subject to such employee's continued service to Taysha on each vesting date. About Taysha Gene TherapiesTaysha Gene Therapies (Nasdaq: TSHA) is a clinical-stage biotechnology company focused on advancing adeno-associated virus (AAV)-based gene therapies for severe monogenic diseases of the central nervous system. Its lead clinical program TSHA-102 is in development for Rett syndrome, a rare neurodevelopmental disorder with no approved disease-modifying therapies that address the genetic root cause of the disease. With a singular focus on developing transformative medicines, Taysha aims to address severe unmet medical needs and dramatically improve the lives of patients and their caregivers. The Company’s management team has proven experience in gene therapy development and commercialization. Taysha leverages this experience, its manufacturing process and a clinically and commercially proven AAV9 capsid in an effort to rapidly translate treatments from bench to bedside. For more information, please visit www.tayshagtx.com. Company Contact:Hayleigh Collins Director, Head of Corporate Communications and Investor RelationsTaysha Gene Therapies, Inc.hcollins@tayshagtx.com Media Contact:Carolyn HawleyInizio EvokeCarolyn.hawley@inizioevoke.com

Data from first adult patient in REVEAL Phase 1/2 trial showed TSHA-102 (low dose, 5.7x1014 total vg) was well-tolerated with no treatment-emergent SAEs as of 35-week assessment, with sustained improvement across key efficacy measures at decreased steroid levels and new improvement in RSBQ at month six Data from second adult patient showed TSHA-102 (low dose, 5.7x1014 total vg) was well-tolerated with no treatment-emergent SAEs as of 19-week assessment, with sustained improvement across key efficacy measures, significantly reduced seizure events and new improvement in R-MBA at week 12 Principal Investigator observed sustained and new improvements across multiple clinical domains following completion of steroid taper for patient one through 35-weeks post-treatment and for patient two through 19-weeks post-treatment at decreased steroid levels Received Independent Data Monitoring Committee approval of Company’s request to proceed to early advancement to cohort two (high dose, 1x1015 total vg) in REVEAL adolescent and adult trial, and approval to dose second pediatric patient in cohort one (low dose, 5.7x1014 total vg) in REVEAL pediatric trial Initial data from cohort one (low dose, 5.7x1014 total vg) in REVEAL pediatric trial expected mid-2024; initial data from cohort two (high dose, 1x1015 total vg) in both trials (adolescent/adult and pediatric) expected in 2H 2024 Conference call and live webcast today at 4:30 PM Eastern Time DALLAS, March 19, 2024 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (Nasdaq: TSHA) (Taysha or the Company), a clinical-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of severe monogenic diseases of the central nervous system (CNS), today reported financial results for the full-year ended December 31, 2023, and provided corporate and clinical updates. “We are highly encouraged by the safety profile and durable response reported in the longer-term data from the low dose cohort in our REVEAL adolescent and adult trial. Importantly, following completion of the steroid taper for the first patient and at decreased steroid levels for the second patient, both patients showed sustained improvements across multiple clinical domains, as well as new improvements compared to earlier post-treatment assessments, which supports the transformative potential of TSHA-102,” said Sean P. Nolan, Chairman and Chief Executive Officer of Taysha. “These continued improvements in both adult patients with advanced stage four Rett syndrome and the initial clinical data from the first pediatric patient were reviewed by the Independent Data Monitoring Committee (IDMC) and enabled us to proceed to earlier dose escalation in the adolescent and adult trial, which will expedite and further inform our clinical development and regulatory strategy for the dose expansion portion of the studies. With this progress, we believe we are well-positioned to focus on generating clinical data in a broad range of ages and stages of patients with Rett syndrome across multiple geographies this year.” Dr. Elsa Rossignol, M.D., FRCP, FAAP, Associate Professor in Neuroscience and Pediatrics at the Université de Montréal, and Principal Investigator of the REVEAL trial at the CHU Sainte-Justine added, “Both adult patients treated with TSHA-102 showed sustained and new improvements across key areas of disease impacting activities of daily living, including multiple aspects of autonomic function, social communication, motor skills, and experienced stabilized or significantly reduced seizures. The first patient sustained improvements at week 35 post-treatment after the completion of her steroid taper, with restored movement in her legs, the gained ability to sit unassisted for the first time in over a decade and gained function in her non-dominant hand. She has also sustained improvements in breathing dysrhythmia and sleep quality and duration, including the gained ability to sleep through the night for the first time in 20 years. Notably, she has vastly increased interest in social communication and activities at week 35 compared to earlier post-treatment assessments. She is more alert and socially interactive, with increased vocalizations and enhanced ability to use an eye-driven communication device. The second patient showed sustained improvements at decreased steroid levels at week 19 post-treatment, including reduced hand stereotypies for the first time since regression at age three, and sustained improvements in breathing dysrhythmia, including hyperventilation and reduced apneic spells. As of week 19 post-treatment, she’s experienced a significant reduction in seizures at a lower dose of anti-seizure medication. Collectively, these continued improvements in both adult patients with different genetic mutation severity and phenotypic expression are encouraging and support the potential of TSHA-102 to bring meaningful change to the lives of patients and their caregivers.” Data Summary from Cohort One (Low Dose, 5.7x1014 total vg) of the REVEAL Phase 1/2 Adolescent and Adult Trial TSHA-102 in Rett syndrome: a self-complementary intrathecally delivered AAV9 gene transfer therapy in clinical evaluation for Rett syndrome, a rare genetic neurodevelopmental disorder caused by mutations in the X-linked MECP2 gene. TSHA-102 utilizes a novel miRARE technology designed to mediate levels of MECP2 in the CNS on a cell-by-cell basis without risk of overexpression. The safety and preliminary efficacy of TSHA-102 are being evaluated in female patients aged 12-years and older with Rett syndrome due to MECP2 loss-of-function mutation in the REVEAL Phase 1/2 adolescent and adult trial, a first-in-human, open-label, randomized, dose-escalation and dose-expansion study taking place in Canada and the United States (U.S.). Dose escalation will evaluate two dose levels of TSHA-102 sequentially. The maximum tolerated dose (MTD) or maximum administered dose (MAD) established in Part A will then be administered during dose expansion in Part B of the study. Results from the first patient (large MECP2 deletion; associated with severe phenotype) and second patient (missense MECP2 mutation; associated with milder phenotype) with late motor deterioration stage four Rett syndrome dosed with TSHA-102 in the low dose cohort (5.7x1014 total vg): Generally well-tolerated with no treatment-emergent serious adverse events (SAEs) as of 35-week assessment post-treatment for patient one and 19-week assessment post-treatment for patient twoSustained and new improvements observed across multiple clinical domains, as of 35-weeks post-treatment for patient one following completion of steroid taper at week 33, and 19-weeks post-treatment for patient two at decreased steroid levels (taper began at week 17), based on clinical observations by the Principal Investigator (PI), including: Autonomic function: improved breathing patterns, sleep quality/duration and circulation (patient one), and improved breathing patterns and circulation (patient two)Socialization/Communication: improved social interest, vocalization and ability to use eye-driven communication device (patient one), and improved social interest (patient two)Motor skills: improved hand function and gained ability to sit unassisted and move legs (patient one), and improved hand stereotypies (patient two)Seizures: stable seizure events (patient one), and significantly reduced seizure events (patient two) Seizure Diary showed stable seizure events at lower levels of anti-seizure medication relative to baseline through 35-weeks post-treatment in patient one, and significantly reduced seizure events with lower levels of anti-seizure medication relative to baseline through 19-weeks post-treatment in patient two, based on caregiver-reported medical history Clinical improvements seen across key efficacy measures in both patients include: Patient one at six-month assessment: Sustained improvement in Clinical Global Impression–Improvement (CGI-I), Clinical Global Impression–Severity (CGI-S), Parental Global Impressions–Improvement (PGI-I), Revised Motor Behavior Assessment (R-MBA), Rett Syndrome Hand Function Scale (RSHFS) and Seizure Diaries, at decreased steroid levels, with new improvement in Rett Syndrome Behavior Questionnaire (RSBQ)Patient two at 12-week assessment: Sustained improvement in CGI-I, PGI-I and RSBQ, with new improvement in R-MBA and Seizure Diaries Figure accompanying this announcement is available here. Recent Corporate and Program Highlights Strengthened clinical and regulatory leadership with the promotion of Meredith Schultz, M.D., M.S., to Chief Medical Officer and Rumana Haque-Ahmed to Chief Regulatory Officer, reporting to Sukumar Nagendran, M.D., President and Head of R&D. Dr. Schultz is a board-certified, licensed pediatric neurologist experienced in treating patients with Rett syndrome and leading gene therapy clinical trials. She brings more than 17 years of clinical experience and will lead the Company’s clinical development, clinical operations, medical affairs and safety activities. Rumana Haque-Ahmed brings nearly 30 years of experience in regulatory strategy and product development in the biopharmaceutical space. She will continue to lead the Company’s regulatory affairs department and initiatives. REVEAL Phase 1/2 Adolescent and Adult Trial (Canada and U.S.): Completed dosing in cohort one (low dose, n=2) of 5.7x1014 total vg.Received IDMC approval of the Company’s request to dose escalate immediately, enabling early advancement to cohort two (high dose, n=3) of 1x1015 total vg.Announced expansion of ongoing trial in Canada into the U.S. and initiated site activation. REVEAL Phase 1/2 Pediatric Trial (U.S. and United Kingdom (U.K.)): Received IDMC approval to dose the second pediatric patient in cohort one (low dose, n=3) of 5.7x1014 total vg following review of initial clinical data from the six-week post-treatment assessment.Received authorization from U.K. Medicines and Healthcare Products Regulatory Agency (MHRA) of Clinical Trial Application (CTA) for TSHA-102, enabling expansion of ongoing trial in U.S. into the U.K.Received Innovative Licensing and Access Pathway (ILAP) designation for TSHA-102 from U.K. MHRA. The ILAP aims to facilitate patient access to novel treatments by accelerating time to market through opportunities for enhanced engagements with U.K. regulatory authorities and other stakeholders. Anticipated 2024 Milestones REVEAL Adolescent and Adult Trial Dosing of the first patient in cohort two (high dose, n=3) of 1x1015 total vg expected in the second quarter of 2024.Initial safety and efficacy data from cohort two expected in the second half of 2024. REVEAL Pediatric Trial Dosing of the second patient in cohort one (low dose, n=3) of 5.7x1014 total vg expected in the first quarter of 2024.Initial safety and efficacy data from cohort one expected in mid-2024.Initial safety and efficacy data from cohort two (high dose, n=3) of 1x1015 total vg expected in the second half of 2024. Full-Year 2023 Financial Highlights Revenue: Revenue for the full year ended December 31, 2023, was $15.5 million compared to $2.5 million for the full year ended December 31, 2022, as revenue was derived entirely from the Company’s Option Agreement with Audentes Therapeutics, Inc. (d/b/a Astellas Gene Therapies). The increase in revenue is primarily a result of Rett syndrome research and development activities performed in 2023. Research and Development Expenses: Research and development expenses were $56.8 million for the full year ended December 31, 2023, compared to $91.2 million for the full year ended December 31, 2022. The decrease was due to reduced research and development headcount, lower research and development manufacturing expenses and a reduction in third-party research and development consulting fees, mainly related to pre-clinical studies and IND-enabling toxicology studies. General and Administrative Expenses: General and administrative expenses were $30.0 million for the full year ended December 31, 2023, compared to $37.4 million for the full year ended December 31, 2022. The decrease was primarily attributable to a reduction in compensation expenses as a result of lower headcount and reduced corporate insurance and consulting expenses. Net loss: Net loss for the full year ended December 31, 2023, was $111.6 million, or $0.96 per share, as compared to a net loss of $166.0 million, or $3.78 per share, for the full year ended December 31, 2022. The net loss includes a non-recurring and non-cash expense of $34.5 million related to the change in fair value from the pre-funded warrants as a result of the August 2023 private placement financing. Cash and cash equivalents: As of December 31, 2023, Taysha had $143.9 million in cash and cash equivalents. The Company continues to expect that its current cash resources will support planned operating expenses and capital requirements into 2026. Conference Call and Webcast InformationTaysha management will hold a conference call and webcast today at 4:30 p.m. ET to review its financial and operating results and to provide corporate and clinical updates. The dial-in number for the conference call is 877-407-0792 (U.S./Canada) or 201-689-8263 (international). The conference ID for all callers is 13744574. The live webcast and replay may be accessed by visiting Taysha’s website at https://ir.tayshagtx.com/news-events/events-presentations. An archived version of the webcast will be available on the website for 30 days. About TSHA-102TSHA-102 is a self-complementary intrathecally delivered AAV9 investigational gene transfer therapy in clinical evaluation for Rett syndrome. Designed as a one-time treatment, TSHA-102 aims to address the genetic root cause of the disease by delivering a functional form of MECP2 to cells in the CNS. TSHA-102 utilizes a novel miRNA-Responsive Auto-Regulatory Element (miRARE) technology designed to mediate levels of MECP2 in the CNS on a cell-by-cell basis without risk of overexpression. TSHA-102 has received Fast Track designation and Orphan Drug and Rare Pediatric Disease designations from the FDA and has been granted Orphan Drug designation from the European Commission. TSHA-102 has also received Innovative Licensing and Access Pathway designation from the U.K. Medicines and Healthcare Products Regulatory Agency. About Rett SyndromeRett syndrome is a rare neurodevelopmental disorder caused by mutations in the X-linked MECP2 gene encoding methyl CpG-binding protein 2 (MeCP2), which is essential for regulating neuronal and synaptic function in the brain. The disorder is characterized by loss of communication and hand function, slowing and/or regression of development, motor and respiratory impairment, seizures, intellectual disabilities and shortened life expectancy. Rett syndrome progression is divided into four key stages, beginning with early onset stagnation at 6 to 18 months of age followed by rapid regression, plateau and late motor deterioration. Rett syndrome primarily occurs in females and is one of the most common genetic causes of severe intellectual disability. Currently, there are no approved disease-modifying therapies that treat the genetic root cause of the disease. Rett syndrome caused by a pathogenic/likely pathogenic MECP2 mutation is estimated to affect between 15,000 and 20,000 patients in the U.S., EU, and U.K. About Taysha Gene TherapiesTaysha Gene Therapies (Nasdaq: TSHA) is a clinical-stage biotechnology company focused on advancing adeno-associated virus (AAV)-based gene therapies for severe monogenic diseases of the central nervous system. Its lead clinical program TSHA-102 is in development for Rett syndrome, a rare neurodevelopmental disorder with no approved disease-modifying therapies that address the genetic root cause of the disease. With a singular focus on developing transformative medicines, Taysha aims to address severe unmet medical needs and dramatically improve the lives of patients and their caregivers. The Company’s management team has proven experience in gene therapy development and commercialization. Taysha leverages this experience, its manufacturing process and a clinically and commercially proven AAV9 capsid in an effort to rapidly translate treatments from bench to bedside. For more information, please visit www.tayshagtx.com. Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as “anticipates,” “believes,” “expects,” “intends,” “projects,” “plans,” and “future” or similar expressions are intended to identify forward-looking statements. Forward-looking statements include statements concerning the potential of TSHA-102, including the reproducibility and durability of any favorable results initially seen in patient dosed to date in clinical trials, and our other product candidates, to positively impact quality of life and alter the course of disease in the patients we seek to treat, our research, development and regulatory plans for our product candidates, including the timing of initiating additional trials and reporting data from our clinical trials, the potential for these product candidates to receive regulatory approval from the FDA or equivalent foreign regulatory agencies, and our current cash resources supporting our planned operating expenses and capital requirements into 2026. Forward-looking statements are based on management’s current expectations and are subject to various risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forward-looking statements. Accordingly, these forward-looking statements do not constitute guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements. Risks regarding our business are described in detail in our Securities and Exchange Commission (“SEC”) filings, including in our Annual Report on Form 10-K for the full-year ended December 31, 2023, which is available on the SEC’s website at www.sec.gov. Additional information will be made available in other filings that we make from time to time with the SEC. These forward-looking statements speak only as of the date hereof, and we disclaim any obligation to update these statements except as may be required by law. Taysha Gene Therapies, Inc.Condensed Consolidated Statements of Operations(in thousands, except share and per share data) For the YearEnded December 31, 2023 2022 Revenue$15,451 $2,502 Operating expenses: Research and development 56,778 91,169 General and administrative 30,047 37,360 Impairment of long-lived assets 1,065 36,420 Total operating expenses 87,890 164,949 Loss from operations (72,439) (162,447)Other income (expense): Change in fair value of warrant liability (34,718) — Loss on debt extinguishment (1,398) — Change in fair value of term loan (1,538) — Interest income 3,572 249 Interest expense (4,998) (3,798)Other expense (47) (18)Total other expense, net (39,127) (3,567)Net loss$ (111,566) $ (166,014)Net loss per common share, basic and diluted$(0.96) $(3.78)Weighted average common shares outstanding, basic and diluted 116,121,482 43,952,015 Taysha Gene Therapies, Inc.Condensed Consolidated Balance Sheet Data(in thousands, except share and per share data) December 31,2023 December 31,2022ASSETS Current assets: Cash and cash equivalents$143,940 $87,880 Restricted cash 449 — Prepaid expenses and other current assets 3,479 8,537 Assets held for sale 2,000 — Total current assets 149,868 96,417 Restricted cash 2,151 2,637 Property, plant and equipment, net 10,826 14,963 Operating lease right-of-use assets 9,582 10,943 Other non-current assets 304 1,316 Total assets$ 172,731 $ 126,276 LIABILITIES AND STOCKHOLDERS' EQUITY Current liabilities: Accounts payable$6,366 $10,946 Accrued expenses and other current liabilities 12,284 18,287 Deferred revenue 18,106 33,557 Total current liabilities 36,756 62,790 Term loan, net 40,508 37,967 Operating lease liability, net of current portion 18,953 20,440 Other non-current liabilities 1,577 4,130 Total liabilities 97,794 125,327 Stockholders' equity Preferred stock, $0.00001 par value per share; 10,000,000 shares authorized and no shares issued and outstanding as of December 31, 2023 and December 31, 2022 — — Common stock, $0.00001 par value per share; 400,000,000 shares authorized and 186,960,193 issued and outstanding as of December 31, 2023, and 200,000,000 shares authorized and 63,207,507 issued and outstanding as of December 31, 2022 2 1 Additional paid-in capital 587,942 402,389 Accumulated deficit (513,007) (401,441)Total stockholders’ equity 74,937 949 Total liabilities and stockholders' equity$ 172,731 $ 126,276 Company Contact:Hayleigh Collins Director, Head of Corporate Communications and Investor RelationsTaysha Gene Therapies, Inc.hcollins@tayshagtx.com Media Contact:Carolyn HawleyInizio EvokeCarolyn.hawley@inizioevoke.com A photo accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/466835b0-a81c-400a-af61-1395051604ec

- FDA completed Pre-License Inspection of Abeona’s Cleveland manufacturing facility, as well as inspection of clinical trial sites for pz-cel pivotal Phase 3 VIITAL™ study -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. 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). 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. 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. 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. “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. Fourth Quarter and Recent Progress Pz-cel for RDEB 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.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. 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.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. U.S. commercial launch preparations for pz-cel 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.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. Corporate highlights 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. 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. Full Year 2023 Financial Results and Cash Runway Guidance 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. 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. 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. 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. 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. Conference Call Details 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. The archived webcast replay will be available for 30 days following the call. About 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. 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. 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. 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; 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. 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 developments or otherwise, except as required by the federal securities laws. ABEONA THERAPEUTICS INC. AND SUBSIDIARIESConsolidated Statements of Operations and Comprehensive Loss(In thousands, except share and per share amounts) For the years ended December 31, 2023 2022 Revenues: License and other revenues $3,500 $1,414 Expenses: Royalties 1,605 450 Research and development 31,091 28,965 General and administrative 19,004 17,256 Impairment of licensed technology — 1,355 Loss/(gain) on operating lease right-of-use assets (1,065) 2,511 Impairment of construction-in-progress — 1,792 Total expenses 50,635 52,329 Loss from operations (47,135) (50,915) Interest income 2,117 431 Interest expense (418) (736)Change in fair value of warrant liabilities (11,695) 11,383 Other income 2,943 141 Net loss $(54,188) $(39,696)Deemed dividends related to Series A and Series B Convertible Redeemable Preferred Stock — (3,782)Net loss attributable to Common Shareholders $(54,188) $(43,478) Basic and diluted loss per common share $(2.53) $(5.53) Weighted average number of common shares outstanding – basic and diluted 21,380,476 7,861,515 Other comprehensive income (loss): Change in unrealized gains (losses) related to available-for-sale debt securities 34 (99)Foreign currency translation adjustments 29 (3)Comprehensive loss $(54,125) $(43,580) ABEONA THERAPEUTICS INC. AND SUBSIDIARIESConsolidated Balance Sheets(In thousands, except share and per share amounts) December 31, 2023 December 31, 2022 ASSETS Current assets: Cash and cash equivalents$14,473 $14,217 Short-term investments 37,753 37,932 Restricted cash 338 338 Other receivables 2,444 188 Prepaid expenses and other current assets 729 424 Total current assets 55,737 53,099 Property and equipment, net 3,533 5,741 Operating lease right-of-use assets 4,455 5,331 Other assets 277 43 Total assets$64,002 $64,214 LIABILITIES AND STOCKHOLDERS' EQUITY Current liabilities: Accounts payable$1,858 $1,811 Accrued expenses 5,985 3,991 Current portion of operating lease liability 998 1,773 Current portion of payable to licensor 4,580 — Other current liabilities 1 204 Total current liabilities 13,422 7,779 Payable to licensor — 4,163 Long-term operating lease liabilities 4,402 5,854 Warrant liabilities 31,352 19,657 Total liabilities 49,176 37,453 Commitments and contingencies Stockholders' equity: 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 — — 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 265 177 Additional paid-in capital 764,151 722,049 Accumulated deficit (749,524) (695,336)Accumulated other comprehensive loss (66) (129)Total stockholders' equity 14,826 26,761 Total liabilities and stockholders' equity$64,002 $64,214