Investigators at Stanford University Medical Center have treated the first patient in a pivotal phase III study of Abeona Therapeutics Inc.'s EB-101, an autologous cell therapy for recessive dystrophic epidermolysis bullosa (RDEB). The trial, delayed by an FDA clinical hold placed in September 2019, has now resumed, with the majority of its 15 expected participants pre-screened. Preparations to open a second trial site on the East Coast remain underway.
Company shares (NASDAQ:ABEO) rose 40.7% to close at $2.04 on March 17, though remain substantially depressed from a 52-week high of $8.41.
Abeona CEO João Siffert acknowledged that impacts from the COVID-19 pandemic are certain to disrupt the company's work, which depends on a tightly choreographed process, involving biopsies, cross-country tissue transport and gene correction. But with the first successful transplant of the company's cell therapy successfully completed, he said he’s now hoping to see the first trial participant's wounds heal as durably as those of patients from the company's phase I/II trial.
“Successful wound healing following EB-101 treatment,” he said during a Tuesday morning earnings call, could provide a “meaningful reduction in time and expense spent on wound care and lower reliance on opioids and other analgesics, potentially improving patients' quality of life.”
The primary endpoint of the randomized study, called Viital, is wound healing. Investigators will compare treated with untreated wound sites on the same patient. Secondary endpoints include the assessments of pain, as well as other patient-reported outcomes.
The company, and likely the RDEB community, are clearly ready for the trial to progress following an unexpected clinical hold that arrived before it had even begun last year. At the time, the agency asked Abeona's team to replicate quality tests following transport of EB-101 from its manufacturing suites in Cleveland to clinical sites like the one at Palo Alto, Calif.-based Stanford. Having already arrived at a point where he felt his team's process was "locked and loaded," the experience frustrated Siffert.
Even ahead of the clinical hold, Abeona had already made significant investments in developing optimized manufacturing, transportation and packaging for EB-101, which comprises sheets of skin cells modified via transduction with a retroviral vector containing the COL7A1 gene. Patients with RDEB have a defect in COL7A1, resulting in the inability to produce type VII collagen, which plays an important role in anchoring the dermal and epidermal layers of the skin.
Now, with the hold lifted in December 2019, the company's team has resumed its work, preparing trial participants’ apportionment of gene-corrected cells, each of which consists of six sheets of tissue, about 40 square centimeters per sheet. The sheets are then packaged individually, stapled to gauze to provide scaffolding and structure and packed for hand delivery.
At present, there are no approved treatments available for RDEB in the U.S. or Europe, though Abeona faces competition from both Castle Creek Biosciences Inc. and Krystal Biotech Inc., each of which are working on gene therapies of their own.
Meanwhile, Abeona is also making strides with its mucopolysaccharidosis (MPS) III gene therapy programs, with interim data from its ongoing phase I/II Transpher A study having demonstrated that its candidate ABO-102 preserved neurocognitive skills up to two years after treatment in MPS IIIA patients treated early in life. It has also reported dose-dependent reductions in disease-specific biomarkers for patients with MPS IIIB, finding "a clear biologic effect" from treatment with another candidate, ABO-101, in the phase I/II Transpher B study, which is now enrolling a third dose cohort.
Following closure of a $103.5 million underwritten public offering, the company reported March 16 that it can fund its operations "well into 2021."