NEW ORLEANS, LA--(Marketwired - May 14, 2015) -

  • Preclinical Studies Support Strong Safety Profile and Investigational New Drug (Ind) Application Filing for Treating Sanfilippo Syndrome (Mucopolysaccharidosis Iiib), a Rare, Fatal Autosomal Recessive Neurodegenerative Disorder
  • Clinical Studies Planned to Commence in 2015

PlasmaTech Biopharmaceuticals, Inc. (NASDAQ: PTBI), a biopharmaceutical company focused on gene therapy and cell therapy products for severe and life-threatening rare diseases announced today that IND enabling safety study data utilizing their adeno-associated virus (AAV9) gene therapy product was presented at the annual meeting of the American Society for Gene and Cell Therapy. The company recently announced its acquisition of Abeona Therapeutics, which has been developing AAV gene therapies for the treatment of Sanfilippo syndromes (MPS IIIA and MPS IIIB).

Sanfilippo syndrome is an inherited genetic disorder that affects children. Children born with Sanfilippo syndrome experience progressive intellectual and motor disability, speech difficulties, seizures, and premature death. As yet, there are no treatments available that can halt or reverse the symptoms of Sanfilippo syndrome.

"These preclinical studies demonstrate increased enzyme levels in target tissues, a strong safety profile, and support our planned clinical trials," stated Tim Miller, Ph.D., President & CEO of Abeona, and prospective CEO of the combined companies upon closing of the transaction.

About Sanfilippo syndrome (Mucopolysaccharidosis IIIB, MPS IIIB):

Sanfilippo syndrome is a group of 4 deadly genetic diseases resulting from the body's inability to properly break down certain sugars. In the class of lysosomal storage diseases, symptoms often appear in the first year of life, and the disease causes progressive muscular and cognitive decline in children after the age of two. Children afflicted with Sanfilippo syndrome experience progressive loss of speech, the ability to eat and walk, and rarely live past their second decade of life. There is no cure and currently no approved treatments for Sanfilippo syndrome.

MPS IIIB is a progressive neuromuscular disease with profound CNS involvement. In this condition, a build-up of a substance called glycosaminoglycans (GAGs) occurs in the lysosomes of cells, particularly in the central nervous system. This is directly related to the malfunction of the α-N-acetylglucosaminidase (NaGlu) enzyme, resulting in dramatic loss of intellectual ability, and a high premature death.

No FDA-approved treatment is currently available for this devastating disorder. Pre-clinical studies using a single intravenous injection of ABX-B (also known as rAAV9.CMV.hNAGLU), have demonstrated restoration of NaGlu activity and corrected the lysosomal storage pathology (histopathology and GAG content) throughout the CNS and in widespread somatic organs. Importantly, intravenous gene delivery of ABX-B improved cognitive and motor functions and normalized the survival of IIIB mice.

About PlasmaTech Biopharmaceuticals: PlasmaTech Biopharmaceuticals is focused on advancing cell therapy and gene therapy for rare diseases. PlasmaTech's lead program is a 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. 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™. The company has developed a robust product pipeline that includes two commercial stage products, MuGard® and ProctiGard™, with additional follow-on products in development. For more information, visit

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, those relating to: the Company's proposed acquisition of Abeona, anticipated acceleration in the 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.

Contact Information:

Company and Media Contact:
Andre'a Lucca
Director of Communications
PlasmaTech Biopharmaceuticals, Inc.