Abeona Therapeutics Details Pathway for Advancing Lead Clinical Programs and Unveils New Cystic Fibrosis Program Born from Next Generation AIM™ Vector Platform at 2018 R&D Day

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NEW YORK and CLEVELAND, Dec. 06, 2018 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO), a leading clinical-stage biopharmaceutical company focused on developing novel cell and gene therapies for life-threatening rare genetic diseases, today announced key pipeline updates during the Company’s 2018 R&D Day.

“The important clinical and preclinical updates we shared today further establish Abeona’s pathway to bring long-term value to our shareholders and hope to our patients,” said João Siffert, M.D., interim Chief Executive Officer, Chief Medical Officer and Head of R&D. “We are very pleased to share next steps for our lead programs and to unveil the potential of the novel AIM AAV vector platform that could be a catalyst for the next generation of gene therapy.”

The Company expects to initiate a pivotal clinical trial evaluating the potential of EB-101 for the treatment of RDEB in the middle of 2019. The VITAL Study will be a multicenter, randomized, Phase III clinical trial assessing 10-15 patients treated with EB-101 compared to intra-patient untreated wounds. The primary outcome measure of the study will be the proportion of treated wounds with >50% healing at three months, with secondary endpoints of investigator global assessment of wounds and changes in pain and itch from baseline.

The Company also reported that it has established GMP manufacturing capability for EB-101 at its gene therapy manufacturing facility in Cleveland. The facility, known as the Elisa Linton Center for Rare Disease Therapies, can produce clinical product, and has scalable capacity to support the potential commercial launch of EB-101.

“We believe that we are strongly positioned to initiate a pivotal trial evaluating EB-101 by mid-2019 thanks to the important CMC work undertaken by colleagues at our gene therapy manufacturing facility in Cleveland, which also addressed guidance received through frequent regulatory interactions afforded by the Regenerative Medicine Advanced Therapy and other designations we hold. We believe this work is critical for our path towards BLA filing,” added Dr. Siffert.

The Company plans to amend its ongoing Phase I/II trial evaluating ABO-102 for MPS IIIA to enroll patients at earlier stages of disease. ABO-102 has been well tolerated to date with no serious drug-related adverse events. The study has also demonstrated a substantial, dose-related improvement in biomarkers, including reductions in cerebrospinal fluid heparan sulfate levels and liver volume in patients treated with ABO-102. Investigators also observed encouraging neurocognitive signals in younger, higher functioning patients enrolled in the higher dose of Cohort 3. Patients unable to participate in the modified Phase I/II study may be eligible to enroll in other studies within our MPS IIIA program.

“The encouraging data generated to date and our interactions with the FDA and EMA have informed the advancement of our Phase I/II trial, which will seek to enroll patients likely to receive the most benefit from treatment,” added Dr. Siffert.

“Our AIM vector platform enables the potential for gene therapy for patients living with cystic fibrosis, regardless of mutation, which could change the landscape of treatment and alter the course of this progressive, genetic disease,” said Timothy J. Miller, Ph.D., co-Founder, President, and Chief Scientific Officer. “We are very encouraged by the preclinical data presented today demonstrating delivery and correction of the underlying genetic deficit in CF patient cells. Furthermore, we are very excited to show the capability of the AIM vectors for delivering genes to the eye and are excited about their potential as the next generation of gene therapy across tissue types.”

The Company presented new preclinical data today that will inform the submission of an investigational new drug application (IND) for ABO-202 in the first quarter of 2019. Findings from a combination pre-clinical, dose-escalation study suggest that ABO-202 may have a favorable safety profile, with no significant toxicology findings. Other IND-enabling studies also demonstrated normalized survival, improvement of motor function and cognition in affected mice treated with ABO-202, and that combination dosing of intravenous and intrathecal administrations may enhance the therapeutic potential of ABO-202.

Abeona has received numerous regulatory designations from the FDA and EMA for its pipeline candidates and is the only company with RMAT designation for two investigational therapies (EB-101 and ABO-102).

More news and information about Abeona Therapeutics Inc.

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Globe Newswire: 17:30 GMT Thursday 6th December 2018

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