SOURCE: Abeona Therapeutics, Inc.

Abeona Therapeutics, Inc.

July 07, 2015 09:07 ET

Abeona Therapeutics Provides Update on SDF™ (Salt Diafiltration) Plasma Fractionation Products

NEW YORK, NY--(Marketwired - July 07, 2015) - Abeona Therapeutics, Inc. (NASDAQ: ABEO)

  • Confirms SDF™ technology platform fractions provide significantly enhanced yields of PTB-101 SDF Alpha™ (alpha1-proteinase inhibitor) for inherited COPD; and PTB-201 SDF IVIG™ (intravenous immunoglobulin) for autoimmune, infectious, and idiopathic diseases
  • Initiated additional programs to finalize SDF™ process for IVIG, a second protein product, and is analyzing additional SDF™ fractions for ultra-orphan protein products

Abeona Therapeutics, Inc. (NASDAQ: ABEO), a biopharmaceutical company focused on developing and delivering gene therapy and plasma-based products for severe and life-threatening rare diseases, today provided an update on the development of its proprietary SDF™ (salt diafiltration) process of extracting and purifying plasma proteins from human plasma. Through the first of two planned contract manufacturers, Abeona has run multiple batches of its two-step salt precipitation process and confirmed that resultant SDF fractions contain significantly enhanced levels of alpha-1 protease inhibitor and immunoglobulins (IVIG) when compared with the industry-standard Cohn process.

The Company is also analyzing the process to identify ultra-orphan proteins for which the SDF process can be further optimized, and has initiated a program to finalize the downstream purification process for SDF IVIG. Preliminary analysis of SDF starting fractions indicates the process should be beneficial for the isolation of a number of ultra-orphan proteins, including plasminogen, fibrinogen, Factor H and von Willebrand's factor, among others. Additionally the Company confirmed that it will be seeking a meeting with the FDA to discuss further development and a clinical pathway for its SDF protein products.

"We are pleased with the progress to date on the development of the SDF plasma process, and can confirm that the process is very efficient at fractioning plasma to provide high levels of PTB-101 SDF Alpha™ (alpha1-proteinase inhibitor) for inherited COPD; and PTB-201 SDF IVIG™ (intravenous immunoglobulin) for autoimmune, infectious, and idiopathic diseases," stated Jeffrey Davis, Chief Operating Officer. "While continuing to optimize downstream steps, we have initiated a program to confirm chromatography steps for our IVIG product, and continue to analyze the SDF fractions to identify ultra-orphan protein products for which our process is particularly well-suited."

"With continued expansion of clinical indications for these proteins, and the potential for new proteins to be extracted and purified, the human plasma protein market remains a high growth opportunity both here and abroad," stated David Nowotnik, Ph.D., Senior Vice President, Research and Development. "We are excited about the opportunity to continue development of our SDF process, and continue to work with our consultants and contract manufacturing partner to evaluate opportunities in this dynamic market."

About Plasma Proteins: The global market for drugs derived from human blood plasma fractionation is currently greater than US$15 billion, and is growing at a rate close to 10% annually. ABEO has developed and patented a new extraction process for plasma biologics that may fundamentally change the economics of blood plasma fractionation, and may make possible the extraction of several additional therapeutically useful plasma proteins. The Company believes that Abeona's proprietary fractionation process is expected to significantly enhance yields of key value blood proteins, including alpha-1 antitrypsin, expanding market opportunities while enhancing margins. The Company obtained rights to utilize and sub license to other pharmaceuticals firms, the recently patented improved methods for the extraction of therapeutic biologics from human plasma. The ability to extract several additional therapeutically useful and important proteins, due to the process being less destructive than historical fractionation processes, may enable the Company to seek new therapeutic applications and address ultra-orphan indications.

About Abeona: Abeona Therapeutics, Inc. is focused on developing and delivering gene therapy and plasma-based products for severe and life-threatening rare diseases. Abeona's lead program is an adeno-associated virus (AAV)-based gene therapy for Sanfilippo syndrome (MPS IIIA and IIIB) in collaboration with patient advocate groups, researchers and clinicians. Clinical trials for Sanfilippo types A and B are anticipated to begin in 2015. The Company recently licensed an AAV-based gene therapy program in juvenile Batten disease (JBD) from the University of Nebraska Medical Center; and licensed an AAV-based gene therapy program for Fanconi anemia (FA) disorder and other rare blood diseases using CRISPR/Cas9 gene editing from the University of Minnesota. In addition, the company is pursuing two additional proprietary platforms, Salt Diafiltration (SDF™) Process and Polymer Hydrogel Technology (PHT™), and is active in the development and commercialization of human plasma-derived therapeutics, including its proprietary alpha-1 protease inhibitor, SDF Alpha™. 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, development and internationalization of clinical programs, information regarding the future performance of the combined company, the outlook on medical needs, future pipeline expectations, management plans for the Company, the anticipated closing of the transaction, and general business outlook. These statements are subject to numerous risks and uncertainties, including but not limited the satisfaction of closing conditions for the transaction, the parties' ability to successfully integrate and operate the new company, and achieve expected synergies and other benefits; the impact of competition; the ability to develop products and technologies; the ability to achieve or obtain necessary regulatory approvals; 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.

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