President and CEO of Abeona Explains the Excitement Surrounding ABO-102

Mathew Shanley

Last week, Abeona Therapeutics announced that the first patient in Spain was enrolled in its Phase 1/2 clinical trial for ABO-102 (AAV-SGSH) for the treatment of Sanfilippo syndrome Type A (mucopolysaccharidosis type IIIA [MPS IIIA]). In October, the company announced the enrollment and administration of the therapy in a patient at Adelaide Women’s and Children’s Hospital in Adelaide, Australia.

Rare Disease Report sat down with Timothy Miller, Ph.D., President and CEO of Abeona, to discuss the excitement surrounding the new therapeutic option for this rare disease, why it’s so important to inform the public about global study sites, and what 2018 holds for the company and these studies.

RDR: What have you seen specifically throughout your work with Sanfilippo syndrome, as it pertains to the patient population and also how ABO-102 can help?

Miller: The kids that are born with Sanfilippo syndrome have a problem making the enzyme that is responsible for chewing up the sugar of heparin sulfate; that’s the key biomarker of disease pathology. What we’ve been able to show is between a 70-90% reduction in target organs going through the high dose, or even some of the lower doses, through 1 year worth of follow-up. That has demonstrated to us that the therapy is durable – We’ve seen time- and dose-dependent effects. We’re looking at multiple disease targets, while knowing that the key marker of the disease is heparin sulfate and when you reduce that, all of the effects that it can have over time. All of our clinical data, to date, tracks – along with our preclinical data, which has been published – that when you remove that underlying disease pathology, the heparin sulfate, that you’re able to see changes in organ structure. These kids present with 100% enlarged livers and spleens, and a year out, we’re able to reduce that liver volume by about 85%. That then starts to translate into, “Okay, well what else do you see?” We see changes in systemic effects leading to reductions in the urinary heparin sulfate and sugars. Then, because we’ve been able to remove that underlying disease pathology, we’re able to change the organ structure and improvements can be seen in more important areas, like the brain. In the end, time is neurons, and now that we’ve looked at some 5 and 6 year olds, it’s important for us to ask, “How can we continue to push this so that these kids have a more normative development?”

RDR: What are your thoughts on the dosing regimens for ABO-102?

Miller: So, our current cohorts are 1x, 2x and 6x dose escalations. Right now, we’ve got 3 patients who are enrolled in cohort 1; 3 patients who are enrolled in cohort 2; and 3 patients enrolled in cohort 3. We’re continuing, however, to expand the cohort 3 dose. When you look at AAV gene therapies for a lot of these lysosomal storage diseases, or any of the autosomal recessive disorders, the mantra is “Treat early, and treat with as much is safely possible.” We’re basically continuing based on that clinical hypothesis.

RDR: Why has Abeona dedicated so much effort to letting people know about the trials taking place around the world? Do you think there’s a difference in patient populations in different countries?

Miller: No, not at all. Really, it goes back to the way that our company was envisioned when we formed it; we have this very global approach to rare disease. We had gotten involved and funded, originally, by over a dozen rare disease foundations, some of which are in Europe. Of course, they wanted us to bring our trials to Europe. In looking at the United States and Europe and a number of other places, including Australia, the way that we wanted to pursue this was to have multiple clinical sites for the enrolling trials. Now that we’ve kind of fulfilled that promise, the benefit to the patient, and also the benefit to the company, is that it expands the trial and its scope, and it hopefully moves us closer to the goal line for a potential approval.

RDR: Why is it so important to you to keep people informed of where you’re conducting the trials?

Miller: Well, it’s really important for the communities (that we relay the information of our global trials). You can find so many parent-led organizations that work within an umbrella structure and this is fulfilling our promise to those parent groups and foundations. We are coming. We are trying to find new ways to treat your kids. When there’s nothing else out there, this is the lifeline that a lot of them have. It’s important for us to keep an open channel of communication to many of these families so that they know where we are.

RDR: What do the remainder of the year and the start of 2018 hold in store for Abeona and ABO-102?

Miller: Right now, we’ve got three clinical sites up and enrolling, and each of them is trying to vie for patients. I don’t know if we’ll expand to any more sites with this current trial, but we do have additional rare disease trials coming up.


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