Abeona Therapeutics Announces Positive Phase 1 Study Results for EB-101 Gene Therapy Clinical Trial

- November 3rd, 2016

Abeona Therapeutics, a clinical-stage biopharmaceutical company focused on delivering gene therapies for life-threatening rare diseases, announced positive clinical trial results from the EB-101 Phase I gene therapy clinical trial.

Abeona Therapeutics Inc. (NASDAQ: ABEO):

  • EB-101 Phase 1 clinical trial therapy demonstrated clinical efficacy of 67% healed wounds at 6 month post-treatment, lasting through 12 months, including collagen biomarker expression
  • EB-101 was well tolerated in patients with recessive dystrophic epidermolysis bullosa (RDEB)
  • Ongoing Phase 2 clinical trial enrolling at Stanford University

Abeona Therapeutics Inc. (NASDAQ: ABEO) a clinical-stage biopharmaceutical company focused on delivering gene therapies for life-threatening rare diseases, announced that positive clinical trial results from the EB-101 Phase I gene therapy clinical trial were published as “Safety and wound outcomes following genetically corrected autologous epidermal grafts in patients with recessive dystrophic epidermolysis bullosa” in the Journal of the American Medical Association (JAMA): http://jamanetwork.com/journals/jama/article-abstract/2576610. Abeona recently announced commencing enrollment in the Phase 2 portion of the clinical study (NCT01263379).

Typically, wounds in patients with RDEB, also known as “butterfly skin” syndrome, can remain unhealed for months to years due to the inability of the skin to stay attached to the underlying dermis and can cover a large percentage of the body. Results from the clinical study demonstrated that treatment with EB-101 restored Type VII collagen expression at the dermal-epidermal junction at the graft sites in 90% of the biopsy samples at 3 months post-treatment, in 66% at 6 months post-treatment, and in 42% samples at 12 months post-treatment. Importantly, correct type VII collagen localization was observed at anchoring fibrils. Wounds that demonstrated type VII collagen at graft sites displayed 87% healing at 3 months, 67% at 6 months, 50% at 12 months compared with baseline wound sites.

“Phase 1 data indicate that EB-101 COL7A1 ex-vivo gene transfer has a favorable safety profile and capable of cutaneous type C7 delivery, highlighting the potential of durable cell-based RDEB therapy in humans in devastating non-healing chronic wounds associated with high levels of morbidity and mortality,” noted Steven H. Rouhandeh, Executive Chairman. “We are looking forward to completing Phase 2 enrollment and exploring approaches to make this potential breakthrough treatment available to RDEB patients.”

The Phase 1 clinical trial with gene-corrected skin grafts has shown promising wound healing and safety in patients with RDEB. Investigators at Stanford University are enrolling adolescent and adult patients for the Phase 2 EB-101 trial to determine the safety and efficacy of COL7A1 gene-corrected grafts on wound healing.

“The clinical data demonstrate that EB-101 gene therapy corrected the underlying genetic deficit in RDEB patient wounds for months to over a year, and the wounds closed — which is remarkable for a disease where the patient’s skin can blister and erode every day,” said Timothy J. Miller, Ph.D., President and CEO of Abeona Therapeutics. “We are very pleased that JAMA recognized the efforts of Drs. Peter Marinkovich, Jean Tang and the team at Stanford University for a decade of work and publish the clinical study results.”

About Epidermolysis Bullosa (EB): EB is a group of devastating, life-threatening genetic skin disorders impacting children that is characterized by skin blisters and erosions all over the body. The most severe form, recessive dystrophic epidermolysis bullosa (RDEB), is characterized by chronic skin blistering, open and painful wounds, joint contractures, esophageal strictures, pseudosyndactyly, corneal abrasions and a shortened life span. Patients with RDEB lack functional type VII collagen (C7) owing to mutations in the gene COL7A1 that encodes for C7 and is the main component of anchoring fibrils that attach the dermis to the epidermis. EB patients suffer through intense pain throughout their lives, with no effective treatments available to reduce the severity of their symptoms. Along with the life-threatening infectious complications associated with this disorder, many individuals often develop an aggressive form of squamous cell carcinoma (SCC).

About Abeona: Abeona Therapeutics Inc. is a clinical stage biopharmaceutical company developing gene and plasma-based therapies for life-threatening rare genetic diseases. Abeona’s lead programs are ABO-102 (AAV-SGSH) and ABO-101 (AAV-NAGLU), adeno-associated virus (AAV) based gene therapies for Sanfilippo syndromes (MPS IIIA and IIIB, respectively). Abeona is also developing EB-101 (gene-corrected skin grafts) for recessive dystrophic epidermolysis bullosa (RDEB), EB-201 for epidermolysis bullosa (EB), ABO-201 (AAV-CLN3) gene therapy for juvenile Batten disease (JNCL), ABO-202 (AAV-CLN1) gene therapy for treatment of infantile Batten disease (INCL), and 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 has a plasma-based protein therapy pipeline, including SDF Alpha™ (alpha-1 protease inhibitor) for inherited COPD, using its proprietary SDF™ (Salt Diafiltration) ethanol-free process. For more information, visit www.abeonatherapeutics.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, 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. These 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 develop our products and technologies; the ability to achieve or obtain necessary regulatory approvals; the impact of changes in the financial markets and global economic conditions; and other risks as may be detailed from time to time in the Company’s Annual Reports on Form 10-K 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.

Investor Contact:
Christine Berni-Silverstein
Vice President, Investor Relations
Abeona Therapeutics Inc.
+1 (212)-786-6212
csilverstein@abeonatherapeutics.com

Media Contact:
Andre’a Lucca
Vice President, Communications & Operations
Abeona Therapeutics Inc.
+1 (212)-786-6208
alucca@abeonatherapeutics.com

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4 responses to “Abeona Therapeutics Announces Positive Phase 1 Study Results for EB-101 Gene Therapy Clinical Trial

  1. Stock promoters responsible for numerous biotech wipeouts run this “company,” while ABEO temporarily trades near the highest market capitalization in its 20-year history.ABEO’s auditor Whitley Penn cited by PCAOB repeatedly for “audit deficiencies”. This is the same tiny auditor who oversaw the alleged UDF “Ponzi scheme” exposed by Kyle Bass.Insiders have collected a shocking ~$35m in compensation, which exceeds 50% of ABEO’s R&D spending since 1996, while losing over $325m of shareholder cash, and yet accomplishing apparently nothing.ABEO insiders have accumulated multi-decade track records of shareholder wreckage including several prior companies that went to essentially zero.ABEO is set to decline 92% as its science fails and the stock promotion comes unwound, just like other Steven Rouhandeh biotech stocks.ABEO appears to be a bottomless pit of self-enrichment for the long-time stock promoters who run this “company” ABEO’s science is completely unviable, calling into question the very reason for the company’s existence. ABEO’s 20 Year History: Crummy Reverse Merger Penny Stock with Ties to Insiders Convicted of Fraud. Abeona is essentially a failed rollup of Blech and Rouhandeh investments that each collapsed. Abeona is the end product of a more than 20-year history of failure and shareholder value destruction that has ties directly to a convicted felon who served jail time for biotech securities fraud.
    An in depth review of Rouhandeh’s past involvements revealed an alarming pattern of repeated shareholder value destruction. Abeona has been nothing short of remarkable as what appears to be a bottomless pit of cash burn and value destruction. Abeona has lost over $325 million since 2006 and the cash burn has accelerated in recent years Millions of dollars have been shuffled into the hands of ABEO insiders and their affiliates Why is Abeona paying its largest shareholder millions of dollars for investor relations? We looked back and could not find a single important scientific discovery in the entire history of Abeona Estimated ABEO insider compensation has exceeded $34 million. ABEO stock is tantamount to simply giving your money to insiders: You get the dilution, they keep the cash. ABEO’s Auditor Currently Entangled in “Ponzi-Like Scheme” Allegations. ABEO’s auditor issued 8 “unqualified going concern” opinions from 2006 to present.

  2. Stock promoters responsible for numerous biotech wipeouts run this “company,” while ABEO temporarily trades near the highest market capitalization in its 20-year history.ABEO’s auditor Whitley Penn cited by PCAOB repeatedly for “audit deficiencies”. This is the same tiny auditor who oversaw the alleged UDF “Ponzi scheme” exposed by Kyle Bass.Insiders have collected a shocking ~$35m in compensation, which exceeds 50% of ABEO’s R&D spending since 1996, while losing over $325m of shareholder cash, and yet accomplishing apparently nothing.ABEO insiders have accumulated multi-decade track records of shareholder wreckage including several prior companies that went to essentially zero.ABEO is set to decline 92% as its science fails and the stock promotion comes unwound, just like other Steven Rouhandeh biotech stocks.ABEO appears to be a bottomless pit of self-enrichment for the long-time stock promoters who run this “company” ABEO’s science is completely unviable, calling into question the very reason for the company’s existence. ABEO’s 20 Year History: Crummy Reverse Merger Penny Stock with Ties to Insiders Convicted of Fraud. Abeona is essentially a failed rollup of Blech and Rouhandeh investments that each collapsed. Abeona is the end product of a more than 20-year history of failure and shareholder value destruction that has ties directly to a convicted felon who served jail time for biotech securities fraud.
    An in depth review of Rouhandeh’s past involvements revealed an alarming pattern of repeated shareholder value destruction. Abeona has been nothing short of remarkable as what appears to be a bottomless pit of cash burn and value destruction. Abeona has lost over $325 million since 2006 and the cash burn has accelerated in recent years Millions of dollars have been shuffled into the hands of ABEO insiders and their affiliates Why is Abeona paying its largest shareholder millions of dollars for investor relations? We looked back and could not find a single important scientific discovery in the entire history of Abeona Estimated ABEO insider compensation has exceeded $34 million. ABEO stock is tantamount to simply giving your money to insiders: You get the dilution, they keep the cash. ABEO’s Auditor Currently Entangled in “Ponzi-Like Scheme” Allegations. ABEO’s auditor issued 8 “unqualified going concern” opinions from 2006 to present.

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