On August 9, 2019 Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader in gene and cell therapy, reported second quarter 2019 financial results and business updates, which will be discussed on a conference call scheduled for Monday, August 12 at 10:00 a.m. ET (Press release, Abeona Therapeutics, AUG 9, 2019, View Source [SID1234538540]). Interested parties are invited to participate in the call by dialing 844-369-8770 (toll-free domestic) or 862-298-0840 (international) or via webcast at View Source

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"The second quarter was highlighted by progress made in both of our MPS III programs," said João Siffert, M.D., Chief Executive Officer. "Data from our Transpher A study showed that children with MPS IIIA who were treated early with ABO-102 preserved neurocognitive development within the normative range 12-18 months post treatment. Our MPS IIIB program has also progressed, with enrollment of additional patients in cohort 2 of the Transpher B study. Our team remains highly focused on our lead programs, including the start of our VIITAL Phase 3 clinical trial in recessive dystrophic epidermolysis bullosa, continued enrollment in the MPS III programs, and preparations to start the clinical trial in CLN1 disease."

Second Quarter Financial Results:

Cash, cash equivalents and marketable securities as of June 30, 2019, were $62.5 million compared to $68.3 million as of March 31, 2019. The decrease in cash was driven primarily by the net cash used in operating activities of $15.2 million.

Research and development expenses for the second quarter ended June 30, 2019 were $16.3 million compared to $7.9 million in the same period of 2018. The increase in R&D expense was primarily attributable to increased in-house manufacturing activities and related headcount costs.

General and administrative expenses for the second quarter ended June 30, 2019 were $5.6 million compared to $4.6 million in the same period of 2018. The increase in G&A expenses was primarily due to increased headcount and related facility costs.

Net loss was $0.49 per share for the second quarter of 2019 compared to $0.26 per share in the same period of 2018.

Second Quarter and Recent Highlights:

-July 25, 2019: Announced positive interim data from the Phase 1/2 AAV9 gene therapy clinical trial in MPS IIIA showing preservation of neurocognitive function for the three youngest patients treated with ABO-102, as well as robust and sustained improvements in biomarkers of the disease. No product-related serious adverse events were reported to date.
-June 26, 2019: Appointed Dr. Victor Paulus as Senior Vice President of Regulatory Affairs and Jodie Gillon as Vice President of Patient Advocacy and Clinical Affairs
-June 18, 2019: Received FDA Fast Track Designation for ABO-202 AAV9 gene therapy in CLN1 disease
-May 21, 2019: Announced FDA clearance of Investigational New Drug application for ABO-202 AAV9 gene therapy in CLN1 disease
-May 14, 2019: Announced treatment of first patient in second cohort of Phase 1/2 clinical trial for ABO-101 AAV9 gene therapy in MPS IIIB
-May 1, 2019: Reported preclinical data demonstrating broad therapeutic potential of AIM gene therapy in retinal diseases at Association for Research in Vision and Ophthalmology Annual Meeting
-April 30, 2019: Reported preclinical data demonstrating therapeutic potential of ABO-401 for treatment of cystic fibrosis at American Society of Gene and Cell Therapy annual meeting
-April 4, 2019: Received FDA Fast Track Designation for ABO-101 AAV9 gene therapy for MPS IIIB

Steven H. Rouhandeh, Abeona’s Executive Chairman, said, "Abeona has continued the development of its breakthrough gene and cell therapies for rare genetic diseases through 2019 with important regulatory and clinical achievements secured. We look forward to progressing our MPS programs, and to starting of our Phase 3 VIITAL trial in EB before year end."