Medical safety board approves dose escalation for second cohort of Abeona’s Sanfilippo trial

Posted: 6 October 2016 | | 1 comment

Following review of the safety data, the DSMB authorised that the clinical trial proceed with enrolment and dose escalation for the second cohort…

The data safety monitoring board (DSMB), a group of independent medical monitors, have reviewed the initial safety data from the low dose cohort (n=3) in the Phase 1/2 clinical trial of Abeona Therapeutics’ ABO-102 (AAV-SGSH), enrolling at Nationwide Children’s Hospital in Columbus, Ohio.


Following review of the safety data, the DSMB authorised that the clinical trial proceed with enrolment and dose escalation for the second cohort. The high-dose cohort will enrol up to six additional patients dozsed at 1.0 X 1013 vg/kg, which is twice the amount of ABO-102 received by patients in the low-dose cohort.

“These early results support Abeona’s unique approach to treating patients with Sanfilippo syndrome, where there are both profound CNS and whole body manifestations of the disease,” stated Timothy Miller PhD President and CEO of Abeona Therapeutics. “We look forward to reporting on future progress and potential for ABO-102 as we begin to enrol patients at the high dose and open additional clinical sites internationally.”

Abeona’s ABO-102 program has been granted both orphan product designation and rare paediatric disease designation in the US and plans to open two additional clinical sites, one in Spain and one in Australia, to test ABO-102. A Phase 1/2 clinical study of ABO-102 in Spain was recently approved by the Agencia Espanola de Medicamentos y Productos Sanitarios, and the Company is preparing to conduct this clinical study at Cruces University Hospital in Bilbao, Spain.

Sanfilippo Syndrome Type A

Sanfilippo Syndrome Type A, or MPS IIIA, is a rare lysosomal storage disease caused by genetic mutations that result in a deficiency of SGSH enzyme activity, leading to abnormal accumulation of certain sugars (specifically, the glycosaminoglycan (GAG) heparan sulfate) in the central nervous system (CNS) and systemic tissues and organs. The accumulation of heparan sulfate results in neurocognitive decline, speech loss, loss of mobility, and premature death.


ABO-102 is an adeno-associated viral (AAV)-based gene therapy for patients with MPS IIIA (Sanfilippo syndrome), that is delivered as a one-time intravenous injection.

ABO-102 delivers a functioning, corrective copy of the SGSH gene to cells of the central nervous system (CNS) and other organs with the goal of correcting the underlying deficits caused by the inborn genetic errors that are the cause the disease. ABO-102 has been well tolerated through 30 day post-injection in subjects injected with the low-dose (n=3).


Encouraging signs of early biopotency have been observed in urinary and CSF GAG (glycosaminoglycan, specifically, heparan sulfate) measurements, as well as potential disease-modifying effects in the liver and spleen of the initial subjects enrolled and treated in the trial.

The clinical study is supported by neurocognitive evaluations, biochemical assessments and MRI data generated in a 25-subject MPS III Natural History Study, also conducted at Nationwide Children’s Hospital, where patients continued through one-year of follow up assessments.

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One response to “Medical safety board approves dose escalation for second cohort of Abeona’s Sanfilippo trial”

  1. Richard Bazinet says:

    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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