Abeona Therapeutics Inc. Common Stock Morgan Stanley 24th Annual Global Healthcare Conference

I have some updates I want to talk to you about. We'll chat later about Okay other stuff.

Yeah. Yep. Great. Thank you all for coming to the annual Morgan Stanley Global Healthcare Conference.

My name is Rock Byun. I am the Head of West Coast Healthcare Investment Banking for Morgan Stanley. I have the pleasure of hosting the Abeona Therapeutics management team today. We'll go through a few questions, but recognizing that this is the first time Abeona's at our conference, would love to maybe start with a round of introductions.

Thank you so much for having us, Rock. I'm Vish Seshadri. I'm the President and CEO of Abeona, and it's a pleasure to attend the Morgan Stanley Conference. This is a very important year for us, the first full year of launch since we got our first approval, so very excited to be here.

Thank you, Rock. Madhav Vasanthavada, I'm the Chief Commercial Officer at Abeona.

Great. So maybe just to start with some high-level questions about Abeona, just so that the audience here and those listening in can get a better sense and hear from you. Abeona has made the transition from being a development stage biotech now to a commercial stage company, and congratulations. What has been the most different or most surprising about operating a company on the other side of approval?

Yeah, thank you for that question. In fact, just to level set, our approval happened last year, and we really launched this product in December when we treated the first patient with ZEVASKYN, which is an autologous cell sheet-based gene therapy for patients with recessive dystrophic EB, a connective tissue genetic disorder. If you look at the launch itself, a lot of the transition that you see from being an R&D company to a commercial stage is what we would expect in the natural course. Madhav and myself, we have a lot of launch experiences in our past, including cell therapies such as these. I think what you would normally expect is we've grown the company from being an 80, 90 people R&D stage company two, three years ago to now we're about 250 people with GMP manufacturing and supply.

As you can imagine, that in itself is a little bit of a cultural shift because our day-to-day is focused on launch performance and such indicators moving from a data generation kind of remit. That comes with a little bit of a cultural shift. What has been the constant with our teams and our culture is the focus on the patient. Now that we have an approval, we feel that responsibility, making sure that the benefit from this drug is going to be. Every time there's a patient at our site or in a conference, all our employees watch with the same spellbound attention as we always do, and that's really some of the most motivating events for us. That hasn't changed. I think what is different when you talk about an ultra-rare disease, plus an autologous cell therapy with an 84-hour shelf life, with a qualified treatment center requirement of a new team, that multidisciplinary team that you need, which did not preexist.

I think that brings with that some unique features of launch that you may not have seen very much with precedents.

I think that's really what's unique, and there's a lot of learnings and we're very excited to talk about it. Given where we are with the third quarter really from launch, I think we're laying some important elements of foundation right now, and things are looking good. We're very happy where we are, and that is important.

Great. Can you just tell us a little bit around the indication and the current treatment paradigm and the patient journey?

The indication really is this is a cell sheet-based gene therapy. This is a connective tissue genetic disorder where patients are missing a functional gene for collagen VII. Collagen VII is that glue that basically keeps skin intact. The skin is two layers, and the two layers are tethered in normal skin, and you lose that integrity in patients that have the deficiency of collagen VII. So what happens over their lifetime is they have blistering and wounding of skin, so the integrity of the skin is lost, and that leads to a lot of complications. They have large wounds that become chronic over a lifetime, infections, sepsis. Nothing is normal in their lives, and it is there 24/7. This is not episodic. They have to live with these wounds all through their lives, and three, four times in a week, they have to undergo wound dressing changes that take six, seven hours, and they have to put bleach in the bathtub.

There is so much procedural burden for patients and their families caring for these patients. Over their lifetime, they develop complications such as squamous cell carcinoma or sepsis. For these reasons, you do not see these patients often live beyond their 30s and 40s. So that is the kind of morbidity and mortality that the disease inflicts- on these patients.