On November 15, 2017 Abeona Therapeutics Inc. (NASDAQ:ABEO), a leading clinical-stage biopharmaceutical company focused on developing novel gene therapies for life-threatening rare diseases, reported financial results for the third quarter and recent business highlights (Press release, Abeona Therapeutics, NOV 15, 2017, View Source;p=RssLanding&cat=news&id=2317120 [SID1234522085]). The Company will provide investors an update on recent and ongoing business activities and an overview of its 3Q17 financials on Monday, November 20th, at 10:00 am (Eastern). Interested parties are invited to participate in the call by dialing 877-269-7756 (toll free domestic) or 201-689-7817 (international).

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"The third quarter was marked with achievements across multiple clinical programs, including initiating enrollments at our global clinical sites for ABO-102 for MPS IIIA and reporting additional data that underscored the durability and clinical benefit of the gene therapy. Our Epidermolysis Bullosa program achieved FDA Breakthrough Therapy designation, completed its Phase 1/2 clinical trial and continues to advance as we finalize the clinical protocol before initiating the pivotal Phase 3 trial next year. We were pleased to have recently initiated screening in our MPS IIIB program and look forward to commencing enrollments shortly," stated Timothy J. Miller, Ph.D., President and CEO. "In addition, work in optimizing our AIM vector platform demonstrated exciting progress, including enhanced tissue tropisms compared to naturally occurring AAV capsids."

3rd Quarter Summary Financial Results:

Cash position: Cash and cash equivalents as of September 30, 2017 were $56.5 million, compared to $58.3 million as of June 30, 2017. Net cash used in operating activities in the nine months ended September 30, 2017 was $17.6 million as compared to $9.6 million in the same period in 2016. Cash and cash equivalents includes approximately $5 million from exercised warrants in the third quarter. Subsequent to the end of the third quarter, the Company closed a public offering of common stock with gross proceeds of $92 million. Total cash as of October 31, 2017 was $142.6 million.
Revenues: Revenues were $219 thousand for the third quarter of 2017, compared to $184 thousand in the third quarter of 2016. Revenues consisted of a combination of royalties from marketed products, primarily MuGard, and recognition of deferred revenues related to upfront payments from early license agreements.
Loss per share: Loss per share was $0.13 for the third quarter of 2017, compared to a loss per share of $0.08 in the comparable period in 2016.
Abeona Recent Highlights:

November 9, 2017: Enrolled First Subject at Spain Clinical Site in Ongoing Phase 1/2 Clinical Trial in MPS IIIA
October 19, 2017: Announced Closing of $92 Million Underwritten Public Offering and Full Exercise of Underwriters’ Option to Purchase Additional Shares
October 16, 2017: Announced a Grant of up to $13.85 Million from Leading Sanfilippo Syndrome Foundations for Clinical Development of MPS III Gene Therapies
October 11, 2017: Hosted inaugural R&D day and announced enrollment of First Two Patients in Global Expansion of Phase 1/2 Clinical Trial in MPS IIIA
October 6, 2017: Announced Top-Line One Year Data from ABO-102 MPS IIIA Trial at ARM’s Cell & Gene Meeting on the Mesa
Gene therapy demonstrated durable and significant reduction of underlying disease pathology across multiple clinical measures in Cohort 1 (n=3) compared to a natural history control group (n=8-12)
Systemic biopotency demonstrated time- and dose-dependent reductions of disease causing Heparan Sulfate in the Cerebrospinal fluid (CSF) and liver volumes
Preservation of deep brain architecture observed after intravenous administration
Stabilization of neurocognitive assessment scores at one year post-injection
October 4, 2017: Announced Dedication of Commercial Gene Therapy Manufacturing Facility in Cleveland, Ohio
September 28, 2017: Announced Collaboration with Brammer Bio for Commercial Translation of ABO-102
August 29, 2017: Received FDA Breakthrough Therapy Designation for EB-101 Autologous Cell Therapy in Epidermolysis Bullosa
July 25, 2017: Announced Appointment of Juan Ruiz, M.D., Ph.D. as Chief Medical Officer
July 18, 2017: Received Guidance from FDA to Commence Pivotal Phase 3 for EB-101 Gene Therapy for Patients with Epidermolysis Bullosa

"We have made great progress in the quarter towards becoming a key player in the development of novel breakthrough gene and cell therapies for rare genetic diseases," stated Steven H. Rouhandeh, Executive Chairman. "The recent investment from high-quality investors and leading foundations is another achievement that demonstrates our internal capabilities and commitment to the advancement of our robust pipeline and next generation vector platform, including MPS III gene therapy products. We look forward to further strengthening our efforts with key hires, advancing clinical capabilities, and commercial expansion in the coming quarters."