|View printer-friendly version|
|Abeona Therapeutics Receives Guidance from FDA to Commence Pivotal Phase 3 for EB-101 Gene Therapy for Patients with Epidermolysis Bullosa|
Pivotal Phase 3 clinical trial is planned to commence in early 2018
EB-101 gene therapy for patients with Recessive Dystrophic Epidermolysis Bullosa (RDEB) has demonstrated promising efficacy and safety in the Phase 1/2 clinical trial
EB-101 has received Orphan Drug and Rare Pediatric Disease Designations in the
“The FDA guidance is an important milestone in our clinical development plan for EB-101, and we are pleased to be moving forward into a registrational Phase 3 clinical study in 2018. Abeona is committed to advancing innovative gene therapies that address the unmet needs of patients suffering with dystrophic epidermolysis bullosa, a devastating rare skin disease. We are grateful that the
Abeona’s EB-101 product is an autologous, ex-vivo gene therapy in which the COL7A1 gene is inserted into a patient’s own skin cells (keratinocytes) for the treatment of the underlying disease in Recessive Dystrophic Epidermolysis Bullosa. The EB-101 program has been granted Orphan Drug and Rare Pediatric Disease Designations from the
“EB Research Partnership (EBRP), along with
About EB-101 Phase 1/2 Clinical Trial:
Clinical data were presented at the
Wound healing, defined as >50% closure after EB-101 administration, was observed in:
-- 100% (36/36 treated wounds, n=6 subjects) at 3 months;
Collagen VII (C7) expression: C7 and morphologically normal NC2 reactive anchoring fibrils were observed in EB-101 treated wounds up to two years post-administration.
Importantly, data from a supportive natural history study of 1,436 wounds from 128 patients with RDEB, established by
About Epidermolysis Bullosa (EB) and Recessive Dystrophic Epidermolysis Bullosa (RDEB): EB is a group of devastating, life-threatening genetic skin disorders characterized by skin blisters and erosions all over the body. The most severe form, recessive dystrophic epidermolysis bullosa (RDEB), is characterized by chronic skin blistering, open and painful wounds, joint contractures, pseudosyndactyly and a shortened life span. Typically, wounds on patients with RDEB, also known as "butterfly skin" syndrome, can remain unhealed for months to years due to the inability of the skin to stay attached to the underlying dermis and can cover a large percentage of the body. Patients with RDEB lack functional type VII collagen owing to mutations in the gene COL7A1 that produces C7 collagen and is the main component of anchoring fibrils, the “velcro” that helps stabilize the skin on the basement membrane.
EB patients suffer through intense pain throughout their lives, with no effective treatments available to reduce the severity of their symptoms. Along with the life-threatening infectious complications associated with this disorder, many individuals often develop an aggressive form of squamous cell carcinoma (SCC).
About Abeona: Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene therapies for life-threatening rare genetic diseases. Abeona's lead programs include ABO-102 (AAV-SGSH), an adeno-associated virus (AAV) based gene therapy for Sanfilippo syndrome type A (MPS IIIA) and EB-101 (gene-corrected skin grafts) for recessive dystrophic epidermolysis bullosa (RDEB). Abeona is also developing ABO-101 (AAV-NAGLU) for Sanfilippo syndrome type B (MPS IIIB), ABO-201 (AAV-CLN3) gene therapy for juvenile Batten disease (JNCL), ABO-202 (AAV-CLN1) for treatment of infantile Batten disease (INCL), EB-201 for epidermolysis bullosa (EB), ABO-301 (AAV-FANCC) for Fanconi anemia (FA) disorder and ABO-302 using a novel CRISPR/Cas9-based gene editing approach to gene therapy for rare blood diseases. In addition, Abeona has a plasma-based protein therapy pipeline, including SDF Alpha™ (alpha-1 protease inhibitor) for inherited COPD, using its proprietary SDF™ (Salt Diafiltration) ethanol-free process. For more information, visit www.abeonatherapeutics.com.
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 that involve risks and uncertainties. These statements include, without limitation, our plans for continued development and internationalization of our clinical programs, that patients will continue to be identified, enrolled, treated and monitored in the EB-101 clinical trial, and that studies will continue to indicate that EB-101 is well-tolerated and may offer significant improvements in wound healing. These statements are subject to numerous risks and uncertainties, including but not limited to continued interest in our rare disease portfolio, our ability to enroll patients in clinical trials, the impact of competition; the ability to develop our products and technologies; the ability to achieve or obtain necessary regulatory approvals; the ability to secure licenses for any technology that may be necessary to commercialize our products; the impact of changes in the financial markets and global economic conditions; and other risks as may be detailed from time to time in the Company's Annual Reports on Form 10-K and other reports filed by the Company with the Securities and Exchange Commission. The Company undertakes no obligations to make any revisions to the forward-looking statements contained in this release or to update them to reflect events or circumstances occurring after the date of this release, whether as a result of new information, future developments or otherwise.