On May 10, 2019 Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader in gene and cell therapy, reported first quarter 2019 financial results and business highlights, which will be discussed on a conference call scheduled for Tuesday, May 14 at 10:00 a.m. ET (Press release, Abeona Therapeutics, MAY 10, 2019, View Source [SID1234536157]). 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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"We’ve had a great start to 2019, with continued progress across our pipeline, including the completion of CMC work in advance of initiating our Phase 3 VITAL clinical trial evaluating EB-101, our gene-corrected cell therapy for patients with recessive dystrophic epidermolysis bullosa," said João Siffert, M.D., Chief Executive Officer. "The study, which will utilize clinical material produced at our Cleveland manufacturing facility, is on track to start mid-2019 following completion of ongoing FDA review."

"We have continued to advance our lysosomal storage disease programs. Our increased efforts around patient recruitment are starting to bear fruit as we observe an uptick in patient screening for both MPS programs. In addition, we have submitted the IND for our CLN1 program." added Dr. Siffert.

First Quarter Financial Results:

Cash, cash equivalents and marketable securities as of March 31, 2019 were $68.3 million, compared to $85.0 million as of December 31, 2018. The decrease in cash of $16.7 million was driven primarily by the net cash used for operating activities of $15.1 million.

Research and development expenses for the first quarter ended March 31, 2019 were $11.7 million, compared to $8.2 million for first quarter 2018. The increase in research and development expense was primarily attributable to increased R&D headcount, related facility costs and internal manufacturing costs.

General and administrative expenses for the first quarter ended March 31, 2019 were $5.7 million, compared to $2.9 million for the first quarter 2018. The increase in general and administrative expenses was primarily attributable to increased headcount and related facility costs.

Net loss was $0.39 per share for the first quarter of 2019, compared to $0.22 per share in the same period of 2018.

First Quarter and Recent Highlights:

·May 8, 2019: Abeona Therapeutics Announces Presentation of EB-101 Data at Society for Investigative Dermatology Annual Meeting
oFollow up data showed that three years after treatment with investigational product, EB-101, a majority of RDEB patients had durable wound healing and improvements in pain and itching. There were no serious treatment-related adverse events three years post-treatment and no replication competent virus present at any time point.
·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
oIntravitreal administration of the Company’s novel AIM AAV204 capsid in non-human primates led to robust transgene expression in the inner and outer retina. These preclinical data support the potential use of intravitreal administration to deliver gene therapy in an out-patient setting for a wide range of inherited and acquired retinal diseases.
·April 30, 2019: Reported New Preclinical Data Demonstrating Therapeutic Potential of ABO-401 for Treatment of Cystic Fibrosis at American Society of Gene and Cell Therapy Annual Meeting
oABO-401, the Company’s novel gene therapy for cystic fibrosis (CF) efficiently delivered a highly-expressed, functional copy of human mini-CFTR (hCFTR) to the lung of CF mice and restored CFTR function in human CF patient nasal and bronchial epithelial cells.
·April 4, 2019: Received FDA Fast Track Designation for ABO-101 for Treatment of Sanfilippo Syndrome Type B (MPS IIIB)
·February 11, 2019: Appointed João Siffert, M.D. Chief Executive Officer
·January 31, 2019: Presented New Supportive Data for Novel Gene Therapies at WORLDSymposium
oPlatform and poster presentations highlighted study results on biodistribution and tissue tropism of the next-generation AIM AAV vector platform in Pompe and Fabry diseases, as well as data from programs in MPS IIIA and CLN3 disease.
·January 8, 2019: Strengthened Financial Leadership with New Executive Appointments: Christine Silverstein as Chief Financial Officer and Edward Carr as Chief Accounting Officer

"We expect 2019 to be a transformative year for Abeona, as we prepare to execute on a number of milestones in the clinic, in our mission to develop gene and cell therapies that could transform the treatment of serious diseases," said Steven H. Rouhandeh, Chairman of the Board and Executive Chairman. "We are encouraged to continue advancing the proprietary technology of our AIM vector platform, especially following recent promising data presented across multiple indications, for which we continue to evaluate avenues of development internally, and with potential partners."