Nasdaq GlobeNewswire

Abeona Therapeutics Reports Fourth Quarter 2017 Financial Results and Business Highlights

Del

Investor Conference Call on Tuesday, March 27th at 10:00 am ET

NEW YORK and CLEVELAND, March 16, 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 financial results for the fourth quarter. The Company will host a call to update investors on recent clinical developments and year-end financial results on Tuesday, March 27th at 10:00 am (Eastern). Interested parties are invited to participate in the call by dialing 877-407-9210 (toll free domestic) or 201-689-8049 (International).

"The past year was marked by several defining events in the company's history, having advanced our two lead clinical programs, EB-101 in Epidermolysis Bullosa and ABO-102 in MPS IIIA, and initiated our third clinical program, ABO-101 in MPS IIIB.  The strong safety and biopotency data observed in our three active clinical trials and the strategic initiative of building an in-house commercial GMP manufacturing facility further strengthens our position in developing gene and cell therapies to treat these devastating and rare pediatric diseases," stated Timothy J. Miller, Ph.D., President and CEO.  

4th Quarter and Year-end Summary Financial Results:

  • Cash position: Cash, cash equivalents and marketable securities as of December 31, 2017 were $137.8 million, compared to $56.5 million as of September 30, 2017.  Net cash used in operating activities in the twelve months ended December 31, 2017 was $22.9 million as compared to $13.0 million in the same period in 2016, an increase of $9.9 million.
     
  • Offering: During the fourth quarter, on October 19, 2017, Abeona closed an underwritten public offering of 5,750,000 shares of common stock, at a public offering price of $16.00 per share. The gross proceeds to the Company were $92 million, before deducting the underwriting discounts and commissions and estimated offering expenses payable by the Company.
     
  • Revenues: Revenues were $215 thousand for the fourth quarter of 2017, compared to $256 thousand in the fourth quarter of 2016. Revenues for twelve months ended December 31, 2017 were $837 thousand, compared to $889 thousand in the same period in 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 $0.19 for the fourth quarter of 2017, compared to a loss per share of $0.18 in the comparable period in 2016. Loss per share was $0.66 for the twelve months ended December 31, 2017, compared to a loss per share of $0.64 in the same period in 2016.

Abeona Recent Highlights:

  • March 15, 2018: Abeona received FDA Rare Pediatric Disease Designation for ABO-202 gene therapy program in CLN1 disease
  • February 12, 2018: Received FDA Orphan Drug Designation for ABO-202 program in CLN1 disease
  • February 8, 2018: Reported top-line data from Phase 1/2 gene therapy trial in MPS IIIA  
    -  ABO-102 results presented at WORLDSymposium for Lysosomal Diseases showed significant time- and dose-dependent reduction of underlying disease pathology, including decreased CSF and urine GAGs (HS fragments) and diminished liver volumes 
    -  Evidence of cognitive benefit at six months post treatment in Cohort 2 and at one year in Cohort 1  
    -  Company receives FDA allowance to lower enrollment age to six months  
  • February 7, 2018: Reported on initial safety and biopotency signals in MPS IIIB gene therapy clinical trial  
    -  ABO-101 is well tolerated and demonstrates early biopotency signals with significant disease-specific heparan sulfate (HS) reductions in cerebral spinal fluid, urine, and plasma and greater than 300-fold increase in NAGLU enzyme activity observed in first subject at 30 days post injection  
  • January 29, 2018: Received FDA Regenerative Medicine Advanced Therapy designation for EB-101 in Epidermolysis Bullosa
  • December 20, 2017: Enrolled first patient in ABO-101 Phase 1/2 clinical trial for MPS IIIB
  • November 9, 2017: Enrolled first subject in Spain in ongoing Phase 1/2 clinical trial in MPS IIIA
  • October 16, 2017: Announced $13.85M grant from leading Sanfilippo syndrome foundations for clinical development of MPS III gene therapies
  • October 11, 2017: Enrolled first two patients in the Cohort 3 expansion of the 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
  • October 4, 2017: Abeona broke ground on GMP commercial manufacturing facility for cell and gene therapies

"2017 was transformative for Abeona, with significant progress made towards our goal of building a strong leadership position in rare disease gene therapy development, and manufacturing technology and capability," stated Steven H. Rouhandeh, Executive Chairman. "The momentum will continue in 2018, with the continued enrollment in the high-dose cohort in our ABO-102 Phase 1/2 clinical trial in Sanfilippo syndrome Type A (MPS IIIA), alongside the clinical and regulatory progress in moving our EB program to a pivotal Phase 3 study and the additional work on our own proprietary AIM(TM) vector platform."

About Abeona: Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing cell and gene therapies for life-threatening rare genetic diseases. Abeona's lead programs include EB-101 (gene-corrected skin grafts) for recessive dystrophic epidermolysis bullosa (RDEB), ABO-102 (AAV-SGSH), an adeno-associated virus (AAV) based gene therapy for Sanfilippo syndrome type A (MPS IIIA) and ABO-101 (AAV-NAGLU), an adeno-associated virus (AAV) based gene therapy for Sanfilippo syndrome type B (MPS IIIB).  Abeona is also developing ABO-201 (AAV-CLN3) gene therapy for CLN3 disease, ABO-202 (AAV-CLN1) for treatment of  CLN1 disease, EB-201 for epidermolysis bullosa (EB), ABO-301 (AAV-FANCC) for Fanconi anemia (FA) disorder and ABO-302 using a novel CRISPR/Cas9-based gene editing approach to gene therapy for rare blood diseases. In addition, Abeona is developing a proprietary vector platform, AIM(TM), for next generation product candidates.  For more information, visit www.abeonatherapeutics.com.

Investor Contact:
Christine Silverstein
SVP, Investor Relations & Finance
Abeona Therapeutics Inc.
+1 (646) 813-4707
csilverstein@abeonatherapeutics.com

Media Contact:
Lynn Granito
Berry & Company Public Relations
+1 (212) 253-8881
lgranito@berrypr.com

This press release contains certain statements that are forward-looking within the meaning of Section 27a of the Securities Act of 1933, as amended, and that involve risks and uncertainties. These statements include, without limitation, statements about our ability to develop our products and technologies; our plans for continued development and internationalization of our clinical programs; that patients will continue to be identified, enrolled, treated and monitored in the EB-101 clinical trial, and that studies will continue to indicate that EB-101 is well-tolerated and may offer significant improvements in wound healing and we plan to initiate a pivotal Phase III trial early next year; we have recently initiated enrollment in our MPS IIIB program; our expectation that we will continue to advance our gene therapy for MPS IIIA patients.  Such statements are subject to numerous risks and uncertainties, including but not limited to continued interest in our rare disease portfolio, our ability to enroll patients in clinical trials, the impact of competition, the ability to secure licenses for any technology that may be necessary to commercialize our products, the ability to achieve or obtain necessary regulatory approvals, the impact of changes in the financial markets and global economic conditions; our belief that initial signals of biopotency and clinical activity, which suggest that ABO-102 successfully reached target tissues throughout the body, including the central nervous system and the increased reductions in CNS GAG support our approach for intravenous delivery for subjects with Sanfilippo syndromes, risks associated with data analysis and reporting, and other risks as may be detailed from time to time in the Company's Annual Reports on Form 10-K and quarterly reports on Form 10-Q and other reports filed by the Company with the Securities and Exchange Commission. The Company undertakes no obligations to make any revisions to the forward-looking statements contained in this release or to update them to reflect events or circumstances occurring after the date of this release, whether as a result of new information, future developments or otherwise.




This announcement is distributed by Nasdaq Corporate Solutions on behalf of Nasdaq Corporate Solutions clients.
The issuer of this announcement warrants that they are solely responsible for the content, accuracy and originality of the information contained therein.
Source: Abeona Therapeutics Inc via Globenewswire

Om Nasdaq GlobeNewswire

Nasdaq GlobeNewswire
Nasdaq GlobeNewswire
One Liberty Plaza - 165 Broadway
NY 10006 New York

+1 212 401 8700http://www.nasdaqomx.com

NASDAQ (NASDAQ: NDAQ) is a leading provider of trading, exchange technology, information and public company services across six continents.

Følg saker fra Nasdaq GlobeNewswire

Registrer deg med din epostadresse under for å få de nyeste sakene fra Nasdaq GlobeNewswire på epost fortløpende. Du kan melde deg av når som helst.

Siste saker fra Nasdaq GlobeNewswire

Abeona Therapeutics Receives Orphan Drug Designation in the European Union for ABO-202 Gene Therapy Program in Batten Disease20.4.2018 14:15Pressemelding

NEW YORK and CLEVELAND, April 20, 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, announced today that the European Medicines Agency (EMA) Committee for Orphan Medicinal Products has granted Orphan Drug Designation (EMA/OD/013/18) for Abeona's gene therapy program ABO-202 for the treatment of subjects with neuronal ceroid lipofuscinosis, also known as Batten Disease, a fatal lysosomal storage disease that primarily affects the nervous system in children. ABO-202 is an adeno-associated virus developed to introduce a functional copy of the CLN1 gene into cells in order to restore the enzyme activity that is needed to break down certain lipopigment proteins that are deficient in patients with CLN1 disease. "This designation is an important step in our CLN1 program as it encourages us to proceed towards clinical development," st

Taconic Biosciences Licenses Easi-CRISPR Technology from the University of Nebraska20.4.2018 14:00Pressemelding

RENSSELAER, N.Y., April 20, 2018 (GLOBE NEWSWIRE) -- Taconic Biosciences, a global leader in providing genetically engineered mouse model and service solutions, has been granted a license to the Easi-CRISPR technology from the University of Nebraska. Easi-CRISPR was developed in the laboratories of Drs. Masato Ohtsuka (Tokai University in Japan), and Channabasavaiah Gurumurthy (University of Nebraska). The terms of the agreement include a world-wide, non-exclusive license to the technology, allowing Taconic rights to generate and sell rodent models generated via Easi-CRISPR. When compared to traditional CRISPR, Easi-CRISPR represents a significant advantage, allowing researchers to perform whole genetic insertions versus being limited to point mutations and constitutive knockouts. "Easi-CRISPR is the next progression of the revolutionary CRISPR/Cas technology. Combining the speed of CRISPR with larger genetic insertions allows complex projects typically requiring homologous recombinati

PayByPhone appoints Andreas Gruber as new President and CEO20.4.2018 00:19Pressemelding

VANCOUVER, British Columbia, April 19, 2018 (GLOBE NEWSWIRE) -- PayByPhone, the global leader in mobile parking payments, today announced that it will appoint Andreas Gruber as president and Chief Executive Officer effective June 1st, 2018. Francis Dupuis, the former President and CEO, will take over a position in Germany with parent company Volkswagen Financial Services AG. To provide additional impetus to the PayByPhone board, two new board positions have been created and as a result Maggie Clay (CFO) and Catherine Scott (Legal Counsel) will be appointed effective June 1st, 2018. During his time at PayByPhone, Francis led the business through significant growth and organizational change and leaves the company well positioned for continued success. "We would like to thank Francis for his strong leadership and look forward to building on his experience within Volkswagen Financial Services," Gerd Künne, Vice-President of mobility unit at Volkswagen Financial Services, said. Francis is e

Progress and Potential for Achieving Cleaner Air on a Global Basis20.4.2018 00:09Pressemelding

Adoption of New Generation Diesel Technology Delivers Rapid Clean Air Benefits New understanding of global air quality highlights the need for technologies like new-generation diesel engines, which are capable of accelerating emissions reductions WASHINGTON, April 19, 2018 (GLOBE NEWSWIRE) -- Recent reports highlighting air quality conditions in the United States (State of the Air) and globally (State of Global Air 2018) celebrate the progress made in developed and developing countries, but also emphasize significant disparities in progress toward reducing levels of key pollutants such as particulate matter and ozone. "Achieving continued clean air progress while also sustaining economic growth is possible. Cities and countries must move to proven cleaner engines and fuels," said Allen Schaeffer, executive director of the Diesel Technology Forum. "We can and should bring the tremendous benefits of new technology diesel engines to communities and countries large and small around the wor

SD-WAN for Office 365: Aryaka Helps eLearning company, City & Guilds Group, Improve Office 365 Application Response Time19.4.2018 18:54Pressemelding

Aryaka's Global SD-WAN reduces time for global users to open SharePoint files from 10 to 0.05 seconds; Outlook performance improves 9x worldwide LONDON, April 19, 2018 (GLOBE NEWSWIRE) -- Aryaka®, the leading global SD-WAN provider, announced today its SD-WAN technology has enabled City & Guilds Group, a worldwide leader in workplace skills, eLearning, and executive coaching, to achieve faster Office 365 application response times leading to an increase in productivity and better collaboration between users in Europe and Asia-Pacific. Headquartered in London, the City & Guilds Group works with education providers, governments and major corporations to help shape and support skills development for employers in over 100 countries. Team members of the company are similarly scattered around the world and primarily collaborate using Microsoft Office 365 applications, but many remote users were struggling to access the group's instance of Office 365 hosted in Dublin. For example, it could ta

Canacol Energy Ltd. Announces Private Offering of Senior Notes19.4.2018 16:23Pressemelding

CALGARY, Alberta, April 19, 2018 (GLOBE NEWSWIRE) -- Canacol Energy Ltd. ("Canacol" or the "Corporation") (TSX:CNE) (OTCQX:CNNEF) (BVC:CNEC) is pleased to announce that it intends, subject to market and other conditions, to offer senior unsecured notes (the "Notes") in a private placement to qualified institutional buyers in the United States pursuant to Rule 144A under the Securities Act of 1933, as amended (the "Securities Act"), to non-U.S. persons in transactions outside the United States pursuant to Regulation S under the Securities Act and pursuant to certain prospectus exemptions in Canada (the "Offering"). The Notes will be fully and unconditionally guaranteed by certain subsidiaries of Canacol. The interest rate, redemption prices and other terms of the Notes are to be determined upon pricing of the Offering. Canacol intends to use the net proceeds from the Offering: (i) to repay the outstanding amounts borrowed under its credit facility; (ii) to pay fees and expenses of the O

I vårt presserom finner du alle våre siste saker, kontaktpersoner, bilder, dokumenter og annen relevant informasjon om oss.

Besøk vårt presserom