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Abeona Therapeutics Leveraging Innovations in Gene and Cell Therapy to Forge Ahead

18 Feb Abeona Therapeutics Leveraging Innovations in Gene and Cell Therapy to Forge Ahead

Abeona Therapeutics (NASDAQ: ABEO), a clinical-stage biopharmaceutical Company pioneering cell and gene-based therapies for rare genetic disease, announced positive interim data from two ongoing phase I/II clinical trials evaluating ABO-102 and ABO-101, indicated for the treatment of MPS IIIA and MPS IIIB. The study being conducted by the Abigail Wexner Research Institute (AWRI) at Nationwide Children’s Hospital showed neurocognitive development 18 months to two years in patients younger than 30 months, who were treated with ABO-102, the Companies lead gene therapy candidate for the treatment of Sanfilippo syndrome type A (MPS IIIA). The Company’s AAV9-based gene therapy technology, which forms the basis for ABO-102 and ABO-101, was licensed from Abigail Wexner Research Institute (AWRI) at Nationwide Children’s Hospital.

In addition the results demonstrated a reduction in cerebrospinal fluid (CSF), heparin sulfate (HS) and liver volume reductions were found to be stable two years after the treatment. The clinical study named “Transpher B” also improved numerous disease biomarkers, proving their biologic effect in patients with MPS IIIB. A total of 8 patients have been treated till date with both the therapies demonstrating a favourable safety profile and tolerability. Transpher A and B are open-label, dose-escalation, Phase I/II global clinical trial testing the efficacy and safety of ABO-102 and ABO-101 respectively.

Abeona Therapeutics (NASDAQ: ABEO)

Market Cap: $264.81M; Current Share Price: 3.17 USDChart
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João Siffert, M.D., Chief Executive Officer, commented

“In total, the new results continue to show that early treatment with ABO-102 can help preserve neurodevelopment in children with MPS IIIA. These data will inform our ongoing discussions with the FDA and EMA, as we work towards providing a regulatory update in the second quarter. “For ABO-101, the reductions in disease-specific biomarkers are encouraging and demonstrate a clear biologic effect, which parallels that seen in the MPS IIIA study. We look forward to enrolling the Transpher B study as expeditiously as possible.”

The data clearly shows that ABO-102 AAV9-based gene therapy is capable of delivering a functional copy of the SGSH gene to cells of the CNS and peripheral organs and can potentially help treat MPS IIIA patients, when administrated at a young age. Besides the U.S, the study is enrolling patients at clinical sites in Spain and Australia. In addition ABO-101, has not registered any severe adverse events.

Mucopolysaccharidosis type III (MPS III), also known as Sanfilippo syndrome, is caused by the mutations in the glucosamine (N-acetyl)-6-sulfatase (GNS), heparan-alpha-glucosaminide N-acetyltransferase (HGSNAT), N-acetyl-alpha-glucosaminidase (NAGLU) and N-sulfoglucosamine sulfohydrolase (SGSH) genes, which are responsible for the synthesis of large sugar molecules called glycosaminoglycans (GAGs). The dysfunction in any of these genes hinders the breakdown of heparan sulfate, leading to its accumulation inside the lysosomes.

This disease primarily affects the brain and the central nervous system and is manifested in early childhood in the form of behavioral problems, sleep disturbances, developmental regression, motor disability and seizures. Those affected with MPS III exhibit macrocephaly, umbilical or inguinal hernia and skeletal abnormalities.

According to the NIH, the estimated incidence of all forms of mucopolysaccharidosis is 1 in 70000 newborns. The disease is inherited through an autosomal recessive pattern and can be diagnosed through a urine test and measurement of enzyme activity through blood and skin samples. In addition, genetic screening can provide information as to which gene is malfunctioning.

The Current treatment options for this disease are aimed at alleviating symptoms and are multidisciplinary in nature. However there are companies that are engaged in extensive research and development activities, and trying to create novel therapeutics for this disease. Gene Therapy, Enzyme replacement therapy, stem cell and substrate reduction therapy are some of the emerging fields, which are offering hope to both the patients and caregivers.

A clinical-stage Biopharmaceutical Company, Abeona Therapeutics is developing an AAV-based gene therapy named ABO-102, which has received the orphan drug designation, rare pediatric disease and RMAT and Breakthrough therapy designation from the FDA. ABO-102 uses an AAV9 vector platform to deliver a functional copy of the defective SGSH gene to cells in the central nervous system. It is designed to be one-time gene therapy that will help the body generate its own enzyme to break down glycosaminoglycans. The Company is currently conducting a Phase I/ 2 trials for high-functioning patients, with plans to conduct another trial for patients with advanced form of the disease as well.

The Company is also working on creating therapeutics for Epidermolysis Bullosa (EB) and Batten disease. EB-101, its lead candidate for the treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB) is currently enrolling patients in a crucial Phase III clinical trial named “VIITAL”.

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ABO-002 is designed to use an AAV9 vector platform, to deliver a functional copy of the PPT1 gene, the defective CLN1 gene, using a combination of intravenous and intrathecal administrations. This can promote proper metabolism in lysosomes by activating the PPT1 enzyme. Results from preclinical trials have demonstrated the safety and tolerability of the drug, along with improvement in survival rates, motor function and cognitive abilities in mice. The drug has been awarded an Orphan Drug Designation and a rare pediatric disease designation in the U.S, in addition to an Orphan Drug Designation in the E.U.

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The Company has a state-of the-art cell and gene manufacturing and production facility to address its needs. Its pipeline also consists of candidates for the treatment of Cystic Fibrosis (ABO-401) and Retinal Diseases (ABO-50X). Furthermore it is working on an undisclosed target named the AIM Vectors, touted to be a next generation AAV product. The AIM vector platform are next-generation AAV capsids that are non-replicating and can selectively target delivery of genetic material to the CNS, lungs, eye, muscle, liver and other tissues as per the Company. The Company’s AIM Capsid library has more than 100 capsids with tissue tropisms, having the potential to target numerous organs and routes of delivery.

Risk Assessment

  • The success of any cell and gene therapy is dependent on having a robust manufacturing capability, which allows stringent control on supply, costs and quality. The Company not only has a fully-operational, cGMP-compliant in-house manufacturing facility but has also tied up with The Elisa Linton Center for Rare Disease Therapies for clinical and commercial production of its adeno-associated virus (AAV)-based gene therapies.
  • The Company is building a diverse pipeline focused on using a range of delivery platforms and technologies such as autologous, gene-corrected cell therapy, one-time gene therapies using the AAV9 vector and a preclinical pipeline based on its AIM™ AAV platform. Some of the areas that Abeona is working on are cystic fibrosis, retinal disorders, RDEB, Infantile and Juvenile Batten Disease, which have a large unmet medical need and present an excellent opportunity for the Company.
  • Clinical Trials are fraught with risk and uncertainty. The Company’s pivotal phase III VIITAL trial in patients with recessive dystrophic epidermolysis bullosa was put on hold by the FDA for want of more information on its transport stability to clinical trial sites. The hold was later removed in December 2019, which allowed the Company to go ahead with the trial. Any such event could have a significant impact on the other candidates in the existing pipeline and its resources in the future as well.
  • Partnerships are critical for the success of clinical-stage companies as they not only provide funding but also assist with regulatory and scientific knowhow, Abeona does not have any strategic partnerships or licensing agreements yet, preferring to develop the pipeline independently. The Company however claims to entertain strategic alternatives consistent with standard industry practices.
  • In December 2019, the Company raised approximately $103.5 million, through a public offering, the proceeds from which are expected to provide cash runaway into 2021, and fund the clinical development of its programs and impending clinical milestones. In addition Abeona had cash, cash equivalents and marketable securities of $47.9 million as of September 30, 2019 as per its Q3, 2019 financial results.
  • Disclosure: I/we have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours.

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