About Abeona Therapeutics

Abeona Therapeutics Inc. (Abeona), a clinical-stage biopharmaceutical company, engages in developing cell and gene therapies for life-threatening diseases. The company's lead clinical program is EB-101, an autologous, engineered cell therapy in development for recessive dystrophic epidermolysis bullosa (RDEB). EB-101 has been granted Orphan Drug and Rare Pediatric Disease (RPD) designations by the U.S. Food and Drug Administration (FDA) and Orphan Drug Designation by the European Medicines Agency (EMA). The company plans to continue the development of adeno-associated virus (AAV)-based gene therapies designed to treat ophthalmic diseases with high unmet medical need using the novel AIM capsid platform that it has exclusively licensed from the University of North Carolina at Chapel Hill (UNC), and internal AAV vector research programs. Abeona's novel, next-generation AAV capsids are being evaluated to improve tropism profiles for a variety of devastating diseases. Strategy The key elements of the company's strategy include advancing and commercializing its late-stage clinical cell and gene therapy programs with a focus on life-threatening diseases; developing novel in-vivo gene therapies using AIM capsid technology; leveraging its leadership position in commercial-scale cell and gene therapy manufacturing; establishing additional cell and gene therapy franchises and adjacencies through in-licensing and strategic partnerships; and maintaining and growing its IP portfolio. Developing Next-Generation Cell and Gene Therapy EB-101 for the Treatment of RDEB The company expects EB-101 could be a treatment option for toughest to treat RDEB wounds. EB-101 has shown durable healing and associated pain reduction in the company's VIITAL phase 3 trial in large and/or chronic wounds that carry the highest burden, including the need for frequent dressing changes, pain, pruritus, risk of infection, and developing skin cancer. EB-101 is an autologous, engineered cell therapy in which a functioning COL7A1 gene is inserted into a patient's own skin cells (keratinocytes) using a retrovirus. The keratinocytes are then transplanted back to the patient to restore Type VII collagen expression and skin function. Results from a completed Phase ½ study that enrolled 7 patients with large and chronic RDEB wounds at Stanford University showed that EB-101 was well-tolerated and resulted in significant and durable wound healing (Siprashvili, Z., et al., 2016), with up to eight years of follow-up (So. Y, Nazaraoff, et al., Orphanet Journal Rare Disease 2022). On November 3, 2022, the company announced positive topline data from VIITAL study. The pivotal Phase 3 VIITAL study evaluated the efficacy, safety and tolerability of EB-101 in 43 large chronic wound pairs in 11 subjects with RDEB. The VIITAL study met its two co-primary efficacy endpoints demonstrating statistically significant, clinically meaningful improvements in wound healing and pain reduction in large chronic RDEB wounds. EB-101 was shown to be well-tolerated with no serious treatment-related adverse events observed, consistent with past clinical experience. Based on these positive topline results, the company intends to submit a Biologics License Application (BLA) for EB-101 to the FDA by mid-2023. EB-101 has been granted Regenerative Medicine Advanced Therapy (RMAT), Breakthrough Therapy, Orphan Drug and RPD designations by the by the FDA, as well as Orphan Drug designation by the EMA. The company has continued to prepare its current Good Manufacturing Practice (cGMP) commercial facility in Cleveland for manufacturing EB-101 to support its planned BLA filing. EB-101 study drug product for all the company's VIITAL study participants has been manufactured at its Cleveland facility. ABO-503 for the Treatment of X-linked Retinoschisis (XLRS). ABO-503, composed of a functional human RS1 packaged in the novel AIM capsid AAV204, has shown preclinical efficacy following delivery to the retina in a mouse model of XLRS. Preclinical studies have demonstrated robust RS1 expression in the retina, improved cone photoreceptor density and overall photoreceptor cell survival, as well as a restoration of outer retina architecture. The company submitted a pre-IND (Investigational New Drug) meeting request with the FDA in March 2023. ABO-504 for the Treatment of Stargardt Disease Abeona's internal research and development team developed ABO-504, which is designed to efficiently reconstitute the full-length ABCA4 gene by implementing a dual AAV vector strategy using the Cre-LoxP recombinase system. In May 2021, at the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting, Abeona reported preclinical data demonstrating the ability of the dual AAV vector system to produce full length ABCA4 protein in cell culture. Recent proof-of-concept studies have extended these findings by showing expression of ABCA4 mRNA and full-length ABCA4 protein in the retina of subretinally dosed abca4-/- knockout mice, at levels similar to endogenous ABCA4 in wild-type animals. ABO-505 for the Treatment of Autosomal Dominant Optic Atrophy (ADOA) ABO-505 is designed to express a functional copy of human Opa1 in the retina following para-retinal injection. ABO-505 aims to take advantage of the robust optic nerve and retinal ganglion cell (RGC) transduction ability of AAV204 to deliver its genetic payload to the cells most affected by ADOA. New preclinical data with ABO-503, ABO-504 and ABO-505 have been submitted for presentation at a future medical meeting in the second quarter of 2023. Gene Therapy Treatments anchored in AIM Vector Platform In 2016, the company licensed a library of novel AAV capsids from UNC. The AIM vector system is a platform of AAV capsids capable of widespread central nervous system gene transfer and can be used to confer high transduction efficiency for various therapeutic indications. In partnership with academic institutions, the company's own scientific research teams have identified vectors within the AIM capsid library showing strong potential to successfully target and reach the central nervous system, as well as ocular, lung, muscle, liver, and other tissues. Based on continuing research by the company and its research partners, it has observed improvements in gene delivery to specific tissues compared to available AAV technology. AIM vectors also have the potential for redosing subjects who previously received certain AAV gene therapy or subjects who have pre-existing antibodies to naturally occurring AAV serotypes. Strategic Licensing Agreements The company has out-licensed certain clinical and research programs, including for the treatment of Sanfilippo syndrome type A (MPS IIIA) to Ultragenyx Pharmaceutical Inc. (Ultragenyx), and for CLN1 disease (infantile Batten disease) and Rett syndrome to Taysha Gene Therapies, Inc. (Taysha). Licensed Technologies and Intellectual Property Recessive Dystrophic Epidermolysis Bullosa To support its EB franchise, the company has licensed a patent family from Stanford University covering EB-101 and its use in the treatment of RDEB. Patents covering the company's investigational EB-101 product have been granted by the European Patent Office (EP3400287B1) and in other geographical regions, and are expected to expire in early 2037. Patent applications remain pending in the United States which, if granted, would be expected to expire in 2037. The company has also filed United States patent applications directed to the packaging and transport of EB-101, which, if granted, are not expected to expire before 2040. The company may also rely on the additional protection afforded by data exclusivity (12 years for biologics like EB-101), other market exclusivity, such as orphan drug exclusivity, and patent term extensions, where applicable. AIM Capsids The company has an exclusive license to an international patent family from UNC covering novel AAV capsids (AIM capsids) that may potentially be used to deliver a wide variety of therapeutic transgenes to human cells to treat genetic diseases. National stage applications directed to the AIM capsids have been filed in the United States, Europe and other geographical regions. The first U.S. patent in this patent family, U.S. Patent No. 10,532,110 (the 110 Patent), was issued to UNC on January 14, 2020. The '110 Patent is entitled to 352 days of patent term adjustment, making its projected expiration date November 6, 2036. The second U.S. patent in this patent family, U.S. Patent No. 10,561,743 (the 743 Patent), was issued to UNC on February 18, 2020. The 743 Patent is expected to expire on November 20, 2035. A third U.S. patent in this patent family, U.S. Patent No. 11,491,242 (the 242 Patent) issued on November 8, 2022. The 242 Patent is entitled to 429 days of patent term adjustment and will not expire before January 22, 2037. The company has exclusive rights to these patents under its license with UNC. The company also owns a second patent family directed to certain AAV capsids and have filed national stage applications in the United States, Europe and other geographical regions. Patents issuing from these applications are not expected to expire before 2039. CLN1 Disease (Infantile Batten Disease) The company has also licensed from UNC rights to two patent families directed to treating CLN1 disease (also known as infantile Batten disease). The first patent family is directed to optimized CLN1 genes and expression cassettes for use in treating CLN1 disease, which has applications pending in the United States, Europe, and other geographical regions. One U.S. patent in the first patent family, U.S. Patent No. 11,504,435 (the 435 Patent), was issued to UNC on November 22, 2022. The 435 Patent is entitled to 578 days of patent term adjustment, making its projected expiration date January 12, 2039. The second patent family is directed to treating CLN1 disease using a combination of intrathecal and intravenous administrations, which has applications pending in the United States, Europe and other geographical regions. Patents issuing from applications in the second patent family are not expected to expire before 2040. The company has entered into agreements exclusively sublicensing these two CLN1 patent families to Taysha Gene Therapies. Rett Syndrome The company has licensed rights to patent families from both UNC and the University of Edinburgh relating to gene therapy for the treatment of Rett Syndrome. The patent family licensed from UNC at Chapel Hill are directed to viral genomes designed to regulate expression of the MeCP2 gene, which is mutated in patients with Rett Syndrome. This family has pending applications in the United States, Europe and other geographical regions. Patents issuing from these applications are not expected to expire before 2039. The patent families licensed from the University of Edinburgh are directed to expression cassettes for MeCP2 polypeptides and to synthetic MeCP2 polypeptides. The patent family directed to MeCP2 expression cassettes has pending applications in the United States, Europe and other geographical regions. The patent family directed to synthetic MeCP2 polypeptides has pending applications in the United States and other geographical regions. Patents issuing from applications in the Edinburgh patent families are not expected to expire before 2038. In October 2020, the company entered into an agreement exclusively sublicensing these UNC and University of Edinburgh patent rights to Taysha Gene Therapies. Multipartite AAV Delivery of Large Transgenes The company has filed a PCT application (PCT/US2021/041527) directed to multipartite delivery of large transgenes using AAV vectors. The company is filing national stage applications in the United States, Europe and other geographical regions. Patents issuing from these applications are not expected to expire before 2041. New AAV Capsids and Ophthalmic Disease Treatment via Para-retinal AAV Administration The company owns a pending PCT application (PCT/US2022/029797) directed to novel AAV capsid proteins and treating ophthalmic diseases via para-retinal administration of AAV vectors. Patents issuing from future national stage applications of this PCT application are not expected to expire before 2042. Treatment of Dominant Optic Atrophy and X-linked Retinoschisis The company owns a pending U.S. provisional application directed to compositions and methods for treating dominant optic atrophy and x-linked retinoschisis. Research and Development For the year ended December 31, 2022, the company's total research and development expenses were $28.9 million. Regulation In addition to the foregoing, state and federal laws regarding environmental protection and hazardous substances, including the Occupational Safety and Health Act, the Resource Conservation and Recovery Act and the Toxic Substances Control Act, affect the company's business. History The company was formerly known as Access Pharmaceuticals, Inc. and changed its name to PlasmaTech Biopharmaceuticals, Inc. in 2014. Further, it changed its name to Abeona Therapeutics Inc. in 2015.