Abeona Therapeutics (ABEO) Granted U.S. FDA Fast Track Designation for ABO-102 in Sanfilippo Syndrome Type A

October 25, 2016 7:47 AM EDT
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Abeona Therapeutics Inc. (Nasdaq: ABEO) announced that the U.S. Food and Drug Administration (FDA) granted Fast Track designation for ABO-102, a single intravenous injection of AAV gene therapy for subjects with MPS IIIA (Sanfilippo syndrome type A), a rare autosomal recessive disease affecting every cell and organ in the body causing neurocognitive decline, speech loss, loss of mobility, and premature death in children.

"Fast Track designation underscores the importance that the FDA places on developing new treatments for life-threatening disorders, such as MPS IIIA, and reinforces our mandate of accelerating the development of ABO-102 to market," stated Timothy J. Miller, Ph.D., President and CEO of Abeona Therapeutics. "We look forward to providing additional updates for our ongoing Phase 1/2 clinical trial."

Fast Track designation is a process designed to facilitate the development and expedite the review of drugs to treat serious conditions that address an unmet medical need. Advantages of Fast Track designation include opportunities for more frequent interactions with the FDA during all aspects of development, and eligibility for priority review and accelerated approval. This designation is in addition to ABO-102 being granted Orphan Drug designation by the FDA and the European Medicines Agency (EMA), as well as having already received the Rare Pediatric Disease Designation from the FDA.

"The Fast Track designation comes with an increase in interaction and feedback from the FDA during the development process of a drug and signifies that the FDA may be able to expedite the review and approval of the ABO-102 gene therapy product which, in preclinical and initial clinical work, has shown encouraging signals of biopotency," stated Steven H. Rouhandeh, Executive Chairman. "This designation also demonstrates to the children and families afflicted with MPS IIIA, the FDA's recognition of the severity and importance of addressing this rare orphan disease."

The ongoing Phase 1/2 study is designed to evaluate safety and preliminary indications of efficacy of ABO-102 in subjects suffering from MPS IIIA. Abeona recently announced initial 30 day post-injection data from subjects that received the low dose:

  • ABO-102 was well-tolerated in subjects injected with the low dose of 5E12 vp/kg ABO-102 with no treatment related adverse events or serious adverse events (SAEs). Following favorable review of the safety data by the independent Data Safety Monitoring Board (DSMB), enrollment in the high dose cohort has commenced.
  • In the natural history study evaluating MPS III subjects, it was shown that urine and cerebral spinal fluid (CSF) GAG (heparan sulfate or "HS") are significantly elevated in the subject population as a symptom of disease pathology.
  • All subjects in the low-dose cohort experienced reductions from baseline in both urinary HS and CSF. At 30 days post-injection, urinary HS reduction was 57.6% +/- 8.2%. Reduction in CSF HS was 25.6% +/- 0.8%, suggesting that ABO-102 crossed the blood brain barrier after intravenous administration.
  • The natural history study in 25 subjects with MPS III (Truxal et. al., 2016, Mol. Genet. Metab.) demonstrated that subjects had increased liver and spleen volumes averaging 116% and 88%, respectively, at baseline that did not change over a year of follow up.
  • All three subjects demonstrated significant reductions in liver volume (17.7% +/- 1.9%), and spleen volume (17.6% +/- 7.1%) from baseline, as measured by MRI at 30 days post-injection.

Per the design of the clinical trial, subjects in the low-dose cohort received a single, intravenous injection of ABO-102 to deliver the AAV viral vector systematically throughout the body to introduce a corrective copy of the gene that underlies the cause of the MPS IIIA disease. Subjects were evaluated at multiple time points over the initial 30 days post-injection for safety assessments and initial signals of biopotency, which suggest that ABO-102 successfully reached target tissues throughout the body, including the central nervous system, to reduce GAG content that underlies the lysosomal storage pathology central to MPS IIIA (Sanfilippo syndrome type A).

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