Epidermolysis Bullosa Dystrophica

- 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

BARCELONA, Spain & WATERTOWN, Mass.--( BUSINESS WIRE )-- Almirall, S.A. (BME:ALM) and Eloxx Pharmaceuticals, Inc. (OTC: ELOX) announced today that the companies have entered into an exclusive license agreement for the asset ZKN-013 . Under the agreement, Almirall obtains global rights to develop and commercialize ZKN-013, including for the use in orphan dermatological diseases. ZKN-013 is a potentially promising oral, nonsense mutation readthrough drug, which enables the host cells to produce functional proteins which counteract the root cause of these rare dermatological and potentially other diseases. The drug candidate is expected to shortly enter into Phase I development in healthy volunteers. As part of this agreement, Eloxx will receive an upfront of $3 million, and additional payments throughout the potential development phases, including regulatory and sales milestones of up to $470 million, as well as tiered royalties based on any potential future global sales. " We are very excited about this agreement with Almirall to develop and distribute ZKN-013, our lead TURBO-ZM™ based molecule, as we believe it has the potential to have a significant impact on the treatment of these painful and debilitating diseases ,” said Sumit Aggarwal, President and Chief Executive Officer of Eloxx . “ In addition to advancing development of ZKN-013, this agreement will allow Eloxx to remain focused on fully maximizing the potential of ELX-02 in rare kidney diseases and continue funded discovery efforts on our TURBO-ZM™ platform .” “ This license agreement is aligned with Almirall’s R&D strategy to develop novel treatments to help people with dermatological conditions, including rare diseases ” said Karl Ziegelbauer, Executive Vice President R&D and Chief Scientific Officer of Almirall . “ We look forward to progressing the development of ZKN-013 to find a potentially impactful solution for patients suffering from rare and devastating diseases caused by nonsense mutations .” RDEB /JEB are rare skin diseases characterized by defects in the Collagen7 gene which is essential for the correct formation of the skin structure and barrier function. ZKN-013 has demonstrated robust functional preclinical activity in RDEB/JEB patient cells and in APCmin (multiple intestinal neoplasia) mice. The studies demonstrated that ZKN-013 induces the production of functional, full-length COL7 in RDEB patient cells. ZKN-013 is also being developed for the treatment of FAP patients with nonsense mutations characterized by proliferation of colon polyps and progression to colon cancer. FAP is a rare GI disease with patients progressing to colon cancer caused by mutations in the APC gene. ZKN-013 is Eloxx’s lead TURBO-ZM based molecule. Eloxx's TURBO-ZM platform uses chemistry technology to develop novel Ribosome Modulating Agents to target the human ribosome to develop new potential therapeutics. Ribosomes form the translation machinery that generates functional proteins from genetic sequences; modulating the ribosome subunits provides a therapeutic approach to address a number of different diseases. About Eloxx Pharmaceuticals Eloxx Pharmaceuticals, Inc. is engaged in the science of ribosome modulation, leveraging its innovative TURBO-ZM™ chemistry technology platform in an effort to develop novel Ribosome Modulating Agents (RMAs) and its library of Eukaryotic Ribosome Selective Glycosides (ERSGs). Eloxx’s lead investigational product candidate, ELX-02, is a small molecule drug candidate designed to restore production of full-length functional proteins. ELX-02 is in Phase 2 clinical development for the treatment of Alport syndrome in patients with nonsense mutations. For more information, please visit www.eloxxpharma.com . About Almirall Almirall is a global pharmaceutical company dedicated to medical dermatology. We closely collaborate with leading scientists, healthcare professionals, and patients to deliver our purpose: to transform the patients' world by helping them realize their hopes and dreams for a healthy life . We are at the forefront of science to deliver ground-breaking, differentiated medical dermatology innovations that address patients´ needs. Almirall, founded in 1944 and headquartered in Barcelona, is publicly traded on the Spanish Stock Exchange (ticker: ALM). Almirall (total revenue in 2023: €898.8 MM, 1900 employees globally) has a direct presence in 21 countries and affiliates in over 70 others. For more information, please visit almirall.com Eloxx Forward-looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements other than statements of historical facts contained in this press release, including without limitation, statements regarding the potential future payments and future benefits under the license agreement, achievement of key milestones under the license agreement and the expected timeline for clinical development and efficacy of ZKN-013 are forward-looking statements. Forward-looking statements can be identified by the words “aim,” “may,” “will,” “would,” “should,” “expect,” “explore,” “plan,” “anticipate,” “could,” “intend,” “target,” “project,” “contemplate,” “believe,” “estimate,” “predict,” “potential,” “seeks,” or “continue” or the negative of these terms similar expressions, although not all forward-looking statements contain these words. Forward-looking statements are based on management's current plans, estimates, assumptions and projections based on information currently available to us. Forward-looking statements are subject to known and unknown risks, uncertainties and assumptions, and actual results or outcomes may differ materially from those expressed or implied in the forward-looking statements due to various important factors, including, but not limited to: our ability, or our licensees’ ability, to progress any product candidates in preclinical or clinical trials; the uncertainty of clinical trial results and the fact that positive results from preclinical studies are not always indicative of positive clinical results; the scope, rate and progress of our, and our licensees’ preclinical studies and clinical trials and other research and development activities; the competition for patient enrollment from drug candidates in development;; our ability to obtain the capital necessary to fund our operations; the cost of filing, prosecuting, defending and enforcing any patent claims and other intellectual property rights; our ability to obtain financing in the future through product licensing, public or private equity or debt financing or otherwise; our ability to meet the continued listing requirements of the Nasdaq Capital Market; general business conditions, regulatory environment, competition and market for our products; and business ability and judgment of personnel, and the availability of qualified personnel and other important factors discussed under the caption “Risk Factors” in our Quarterly Report on Form 10-Q for the quarterly period ended September 30, 2023, as any such factors may be updated from time to time in our other filings with the SEC, accessible on the SEC’s website at www.sec.gov and the “Financials & Filings” page of our website at https://investors.eloxxpharma.com/financials-filings . All forward-looking statements speak only as of the date of this press release and, except as required by applicable law, we have no obligation to update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise. Almirall Legal warning This document includes only summary information and is not intended to be exhaustive. The facts, figures and opinions contained in this document, in addition to the historical ones, are "forward-looking statements". These statements are based on the information currently available and the best estimates and assumptions that the Company considers reasonable. These statements involve risks and uncertainties beyond the control of the Company. Therefore, actual results may differ materially from those declared by such forward-looking statements. The Company expressly waives any obligation to revise or update any forward-looking statements, goals or estimates contained in this document to reflect any changes in the assumptions, events or circumstances on which such forward-looking statements are based, unless required by the applicable law.

ZKN-013 has already demonstrated activity in RDEB/JEB patient cells in preclinical studies. Almirall is handing over $3 million for Eloxx Pharmaceuticals’ molecule aimed at a pair of rare skin diseases and potentially more just before the drug enters the clinic. The candidate in question is a so-called nonsense mutation readthrough drug called ZKN-013. Nonsense mutations stop DNA synthesis in its tracks, resulting in an incomplete protein that doesn't work. ZKN-013 is designed to enable host cells to produce functional proteins that counteract the root cause of certain dermatological diseases. Specifically, Eloxx has been targeting ZKN-013 at dystrophic epidermolysis bullosa (RDEB) and junctional epidermolysis bullosa (JEB). Both of these rare skin conditions are characterized by defects in the Collagen7 gene, which plays a vital role in the correct formation of the skin’s structure and ability to act as a barrier. In preclinical studies ZKN-013 has already demonstrated activity in RDEB/JEB patient cells, Almirall said in a March 13 release, including the production of functional, full-length Collagen7 in RDEB cells. As a result of these findings, Eloxx has been gearing up to enter ZKN-013 into a phase 1 clinical trial “shortly.” The biotech has also been investigating ZKN-013’s potential as a treatment for familial adenomatous polyposis, an inherited disorder characterized by cancer of the large intestine and rectum. In return for the global rights to the molecule, Eloxx will be in line for up to $470 million in potential milestone payments as well as tiered royalties on global sales. "We are very excited about this agreement with Almirall to develop and distribute ZKN-013, our lead TURBO-ZM based molecule, as we believe it has the potential to have a significant impact on the treatment of these painful and debilitating diseases,” Eloxx CEO Sumit Aggarwal said in the release.  “In addition to advancing development of ZKN-013, this agreement will allow Eloxx to remain focused on fully maximizing the potential of ELX-02 in rare kidney diseases and continue funded discovery efforts on our TURBO-ZM platform,” Aggarwal added.