Abeona Therapeutics Inc at Jefferies Gene Therapy/Editing Summit (Virtual) Transcript

The following is excerpted from the question-and-answer section of the transcript.

Question: Maury Raycroft - Jefferies Group LLC - Analyst : And so we are going to have a fireside chat format. If anyone in the audience has questions, feel free to submit them online, and we will get to them as we go. But maybe to start off -- for those who are new to the story, can you please provide a one-minute overview of Abeona?

Question: Maury Raycroft - Jefferies Group LLC - Analyst : Great. So I guess maybe diving into the clinical programs, maybe if you can talk a little bit about those and some of the data you have shown so far.

Question: Maury Raycroft - Jefferies Group LLC - Analyst : Got it. That is a great overview. And maybe for EB-101 in RDEB, you're in Phase 3. Can you talk more about the study design and provide a status update on the latest number of patients dosed, and maybe even on total number of wounds treated?

Question: Maury Raycroft - Jefferies Group LLC - Analyst : Got it. So get the data at Quarter 3, and then maybe -- can you talk about plan for filing and even launching?

Question: Maury Raycroft - Jefferies Group LLC - Analyst : Got it. And Vish, we noticed that the UMass med school site posted on ct.gov on October 4, and that is recruiting. Can you see how it is going at that site, and has it treated any patients yet?

Question: Maury Raycroft - Jefferies Group LLC - Analyst : Got it. You talked a little bit about how EB-101 works, but maybe you can talk a little bit more about the process and timeline for screening and enrolling and treating patients in the clinical study. And how does this change when you go commercial? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. OCTOBER 27, 2021 / 7:30PM, ABEO.OQ - Abeona Therapeutics Inc at Jefferies Gene Therapy/Editing Summit (Virtual)

Question: Maury Raycroft - Jefferies Group LLC - Analyst : Got it. Okay. And for that UMass site, have you actually treated patients there or --?

Question: Maury Raycroft - Jefferies Group LLC - Analyst : Okay. And also wanted to ask how you think about competitors in this space. Krystal is going to have Phase 3 data expected by the end of this year. And then, generally, do you think the EB-101 approach could be used in combination with other approaches?

Question: Maury Raycroft - Jefferies Group LLC - Analyst : Got it. So for the Krystal data though, you are not saying on whether you think it is going to meet a certain bar or --.

Question: Maury Raycroft - Jefferies Group LLC - Analyst : Got it. Okay. And also wanted to ask if you can talk more about your graft-making capabilities and where you are at with being commercial ready.

Question: Maury Raycroft - Jefferies Group LLC - Analyst : Got it. And so that is going to happen -- basically, you make that transition to the new facility right before EB-101 goes commercial for the AAV?

Question: Maury Raycroft - Jefferies Group LLC - Analyst : Got it. And Vish, maybe if you can talk a little bit more about the market opportunity, put any numbers behind the chronic wounds versus the recurring wounds, and then how you are thinking about this opportunity over the first couple of years.

Question: Maury Raycroft - Jefferies Group LLC - Analyst : Got it. Okay, that's really helpful. And also wanted to ask how you are thinking about expanding the RDEB skin graft platform. Could you go into other indications outside of RDEB?

Question: Maury Raycroft - Jefferies Group LLC - Analyst : Interesting. And maybe last question, just going back to the opportunity. What are your latest thoughts on retreatment for patients?

Question: Maury Raycroft - Jefferies Group LLC - Analyst : Got it. That makes sense. And maybe shifting gears to MPS IIIA, now that you have FDA alignment on the primary endpoint, which is neurocognitive improvement at 42 months versus natural history on either Bayley Scales or Kaufman, can you talk more about the statistical analysis plan to detect and demonstrate an improvement? And when will this topline be confirmed with FDA?

Question: Maury Raycroft - Jefferies Group LLC - Analyst : Got it. So potentially, 4Q 2022 or --.

Question: Maury Raycroft - Jefferies Group LLC - Analyst : Quarter 1 2023. Got it. Okay. And what is the latest status for the IIIA study and when could we expect the next update from that one?

Question: Maury Raycroft - Jefferies Group LLC - Analyst : Got it. And Vish, maybe if you can provide a status update on the IIIB program, too, and what kind of updates we should be expecting from that one next year? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. OCTOBER 27, 2021 / 7:30PM, ABEO.OQ - Abeona Therapeutics Inc at Jefferies Gene Therapy/Editing Summit (Virtual)

Question: Maury Raycroft - Jefferies Group LLC - Analyst : Got it. Okay. We have talked in the past about ophthalmology. Maybe if you could talk a little bit more about the process in choosing the candidate that you go forward with initially. And what are timelines to potentially getting to an IND?

Question: Maury Raycroft - Jefferies Group LLC - Analyst : Got it. Okay. Well, Vish, I think we are pretty much out of time, but maybe to close out the conversation, if you want to talk about key events ahead that investors should be focused on.

Question: Maury Raycroft - Jefferies Group LLC - Analyst : Got it. Okay. Well, Vish, great speaking with you, and good seeing you for this conference. REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. OCTOBER 27, 2021 / 7:30PM, ABEO.OQ - Abeona Therapeutics Inc at Jefferies Gene Therapy/Editing Summit (Virtual)