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spk00: Good morning, everyone, and welcome to AB Owner's fourth quarter and full year 2023 earnings conference call. At this time, all participants have been placed in a listen-only mode, and the floor will be open for questions after the presentation. If anyone should require operator assistance during the conference, please press star zero on your phone keypad. Please note this conference is being recorded. I will now turn the conference over to your host, Greg Gin, VP of Investor Relations. Greg, over to you.
spk04: Thank you, Jenny. And good morning, everyone. I would like to welcome and thank everyone for joining us on our fourth quarter and full year 2023 conference call. The press release announcing the full year 2023 results is available on our website at www.abostherapeutics.com. I would like to note that remarks made during today's call may contain projections and court-looking statements. Forward-looking statements are made pursuant to the safe harbor provisions of the federal securities law. These forward-looking statements are based on current expectations and are subject to change, and actual results may differ materially from those expressed or implied in the forward-looking statements. Various factors that could cause actual results to differ include, but are not limited to, those identified under the risk factor section in our Form 10-K and periodic reports filed with the SEC. These documents are available on our website at www.ablenttherapists.com. On the call today with prepared remarks are Dr. Vish Sasadri, Chief Executive Officer, Dr. Mata Vizantavada, Chief Commercial Officer and Head of Business Development, and Joe Giordano, Chief Financial Officer. Also joining us for the Q&A session will be Dr. Brian Keveny, Chief Technical Officer. And with that, I will now turn the call over to Vish Sasadri to lead us off. Vish? Thank you, Greg.
spk05: Hello, everybody, and thank you for joining us this morning. I'm pleased to highlight several important milestone achievements for Aviona since our last quarterly call. We continue to make significant progress towards U.S. commercialization. Our top priority is to secure FDA approval and launch Pradimogene Xamycarasso, or PZ cell, our investigational autologous Col7a1 gene-corrected epidermal sheet for recessive dystrophic epidermolysis bullosa, or RDEB. As a reminder, The FDA accepted and granted priority review in November for our PZ cell BLA and assigned a PDUFA date of May 25th, 2024. The BLA process is ongoing and we have several positive status updates to share with you today. The FDA has now completed a bioresearch monitoring BIMO inspection of Abiona, the BLA mid-cycle meeting, and a two-week pre-license inspection, or PLI, of our manufacturing sites. The BIMO inspection was conducted on-site at our headquarters in Cleveland, Ohio, from Jan 22nd through Jan 24th, 2024, and reviewed the conduct and practices that pertain to the clinical studies of PT cells and its data integrity. The FDA inspector did not issue any observations or form 483s during that inspection. The BLA mid-cycle review meeting took place on Jan 25th, 2024, following the BIMO inspection. At the meeting, the FDA indicated that there were no major safety issues and reaffirmed that they are not planning to hold an advisory committee meeting to discuss the BLA. In addition, the FDA advised that risk evaluation and mitigation strategies, or REMS, are not anticipated for PZ-Cell, although BLA review is still ongoing with a proof of target action date of May 25, 2024. In February, the FDA conducted a two-week PLI, or pre-license inspection, of Fabiona's manufacturing facility, which concluded on March 1st. During the inspection, which was conducted by five FDA inspectors, the FDA reviewed the facilities, systems, and processes at the Cleveland site. This comprehensive evaluation included observing the manufacturing process for PV cell, as well as the performance of various in-process and loss-relief assays. Upon completion of the inspection, the FDA inspector issued a Form 483 with observations related to process controls and recommended steps for resolution in a collaborative manner. On March 15, 2024, we submitted a response to the FDA outlining steps either already implemented or ongoing toward resolution based on the FDA's guidance. In addition, the agency has completed its site inspection of the two clinical study sites, Stanford University School of Medicine and University of Massachusetts Medical School, which treated subjects in the pivotal Phase III vital study supporting the PV cell BLA with no form 483 observations noted. We're looking forward to continuing to work with the FDA through the remainder of the BLA review process. Having reached these significant regulatory milestones, we continue to advance our preparations for the planned commercial launch of PV cell in the U.S. following FDA approval. and now turn the call over to our Chief Commercial Officer, Dr. Madhav Vasanthavada, to provide an update on our commercialization readiness activities. Madhav. Thanks, Vaish. If approved, PZ-Cell is expected to deliver unique value for patients with R-DEP. It would be the only therapy which, in clinical trials, address large body surface areas, including toughest-to-treat wounds, and demonstrated wound healing and pain reduction with years of durability after a single treatment. In terms of commercial opportunity and readiness, I want to highlight three important takeaways, all of which are very encouraging. First, dermatologists have indicated a high willingness to refer patients for PZ cell treatment. Following recent market research with 64 dermatologists across both the Centers of Excellence, or COEs, and community setting, we have gained compelling quantitative and qualitative insights. These insights have validated a key assumption of our launch strategy, namely that death patients, in particular severe death patients, are co-managed between COEs and community derms. There's a high concentration of severe death patients in EB centers, and a high level of interest from community dermatologists to refer their severe patients to these EB centers of excellence for a treatment like PZ-Cell. The physicians we surveyed each treat, on average, seven to 10 RDEP patients on an ongoing basis. To hear about the strong enthusiasm from community DERMs and their willingness to refer patients for PZ-Cell, despite the logistical coordination required for surgical application, is very encouraging. The reason for such enthusiasm from both COEs and community practitioners is the clinical study data demonstrating PZ cells' ability to heal large wound areas and offer potential durability and significant pain reduction following a single application. They also noted that some patients currently travel three to four hours to a center of excellence for special procedures and would be willing to do so for a therapy like PZ Cell because their wounds could turn chronic over time and are at high risk of secondary infections and squamous cell carcinomas. We also hear from surveyed physicians as well as prominent physicians in the EB space about the need for using complementary treatment modalities for deaf patients. In addition to physicians, we surveyed patients and caregivers who stated their interest in PZ-Cell because of its ability to cover multiple wounds at once and they wanted to know specifics of the logistical aspects of getting PZ cell therapy. In phase three clinical vital trials, we had the ability to treat with six credit card-sized sheets that were applied either in a quilt-like manner for one large wound area or were discreetly applied to individual wounds as needed. In the commercial setting, we'll be able to supply up to 12 credit card-sized skin sheets, and this is viewed as a big deal by physicians as well as by patients whom we surveyed. The second takeaway is the continued engagement at the Centers of Excellence where we plan to make PZ Cell available. As stated in prior calls, our focus strategy is to partner with five to seven high-volume EV centers in the launch year and onboard them so that patients and caregivers get the best possible experience. We have a clear action plan and continue to gain buy-in from our target centers. Many of the identified centers treat both pediatric and adult EB patients and are geographically dispersed across the U.S., which would make help easy cell treatment accessible for patients. EB physicians at the target centers are in fact championing the onboarding process, including identifying a multidisciplinary team of surgeons, anesthesiologists, and coordinators. They're also helping us to navigate their institutional legal processes and working with their cell and gene therapy departments, which all helps expedite the process. We have signed CDAs with target centers and have initiated discussions on a master service agreement, which is often the most time-consuming process. Overall, we expect the site onboarding process to be like that of other autologous cell therapies, And speaking from personal experience, this typically takes around six to nine months of process. The initial target sites are at varying stages of onboarding, and we believe that they will be fully trained, buttoned up on the logistics, and ready to treat with PZ cells around three months after approval. In addition to the currently targeted sites, we are seeing interest from additional COEs to bring on PZ cells. We just wrapped up some productive meetings with physicians at the American Academy of Dermatology annual meeting in San Diego, and this direct physician feedback gives us reason to believe that the underlying medical need and the opportunity with PZ-Cell is strong. The third and final takeaway is the early encouraging dialogue we've had with payers to enable access due to the high unmet need and the transformational value proposition of PZ-Cell. We have initiated pre-approval information exchange with various commercial and managed Medicaid stakeholders to educate them on Rdap disease and PZ cells differentiated and meaningful clinical profile. We'll be speaking with many of the top payers in the coming weeks and months. From a pricing perspective, we believe PZ cells value is different from that of currently approved products in the EP space because PZ cells can cover much larger areas of a patient's body, multiple wounds at once, and does not rely on the need for a chronic application. We are currently working through our pricing models and expect pricing to be commensurate with the value of approved gene therapies that offer years of durable benefit following the single application. Lastly, we have engaged with the Centers of Medicare and Medicaid Services, CMS, to request a PZ Cell-specific procedure code that, if granted, would support efficient billing, access, and reimbursement of PZ Cell. In addition to the stated takeaways I would just like to note also that we are making rapid progress in building a highly experienced and nimble commercial team, and we are working with a strong network of collaborators to build capabilities for a successful launch and beyond. With that, I'll now hand the call over to our Chief Financial Officer, Joe Vizzano, to discuss our financial results.
spk01: Thanks, Manav. I would like to remind everyone that you can find additional details on our financial results for the year ended December 31st, 2023, in our most recent Form 10-K, which is available on our website. Starting with the financial resources on our balance sheet, we had cash, cash equivalents, restricted cash, and short-term investments of $52.6 million as of December 31st, 2023, as compared to $52.5 million as of December 31st, 2022. Net cash used in operating activities was $37 million for the full year of 2023, compared to $43.5 million in the full year of 2022. Based on our current operating plan and assumptions, our existing cash, resources, and access of up to $50 million via our credit facility, we estimate we have sufficient financial resources to fund the operations through PZCell launch, if approved and into the first quarter of 2025. Our cash runway assumptions do not account for any potential revenues from commercial sales of PZ-Cell or proceeds from the sale of a priority review voucher, or PRV, if awarded by the FDA. I'll remind you that PZ-Cell has been granted rare pediatric disease designation by the FDA, so upon its potential approval, we believe that we are eligible to receive a PRV. License and other revenues for the year ended December 31st, 2023 were $3.5 million as compared to $1.4 million for the same period of 2022. The revenues in both years primarily represent clinical milestone payments under a license agreement with patient gene therapy for an investigational AAV-based gene therapy for Rett syndrome. Research and development expenses were $31.1 million for the year ended December 31, 2023, compared to $29 million for the same period, December 31, 2022. Our spend on general and administrative activities was $19 million for the year ended December 31, 2023, compared to $17.3 million for the year ended December 31, 2022. Net loss attributable to common shareholders was $54.2 million for the year ended December 31st, 2023, or $2.53 loss per common share as compared to a net loss attributable to common shareholders of $43.5 million or $5.53 loss per common share for the year of 2022. With that, operator, can you please open the Q&A session?
spk00: No problem. We will now be opening the floor for questions. If you would like to ask a question, please press star 1 on your phone keypad now. A confirmation tone will indicate that your line is in the queue. You may press star 2 if you would like to remove your question from the queue. For anyone using speaker equipment, it may be necessary to pick up your handset before you press the keys. Please hold a moment whilst we poll for questions. Thank you. Your first question is coming from Maury Rakoff of Jefferies. Maury, your line is live.
spk03: Hi, good morning. Congrats on the progress and thanks for taking my questions. I was going to ask about the Form 483, if you can elaborate on what specific process controls were referenced and do you anticipate hearing back from FDA on this Form 483 or what are expectations for next steps?
spk05: Morning, Maury. Thank you for the question, and thanks for joining us. We're not getting into the technical details of the observations on the 483, but I just wanted to reiterate that this is not something that was unexpected. In fact, 483s from inspections for complex products like ours is more the rule rather than the exception, so if you look at the past records. So we don't observe this as an atypical result, so that's first number one. The process controls that we talked about, I won't get into the technical nature of it, but they are sometimes even interrelated, what's noted in the observations. And this is something that we had a very collaborative interaction with the agency. Clearly, they laid out what's required for resolution, and some of that we've already implemented. And as I mentioned, some of that is ongoing in our plan, and we have already submitted our response to the 483 on March 15th. So as per the FDA's guidance, there's a 45-day window after the completion of the 483 when we should expect to hear, to get a formal note from the FDA in the form EIR. So that's regarding the 483. And you had a second question, Maury, what to expect. I think there are other milestones that the VLA review is still ongoing. So the process of receiving the IRs and responding to them, that is still ongoing. But importantly, our PDUFA date is May 25th. So that's what we're targeting for.
spk03: Got it. And you mentioned the 45-day window. Could you hear back from FDA sooner than that? And maybe just talk about, is there any risk to a delay to the PDUFA?
spk05: Sure. In terms of, we don't want to speculate when exactly we will get the EIR. As per their guidance on SMD 145, it is 45, you know, we should hear within 45 days of completion. So we don't want to speculate how quickly that can happen. In terms of your other part of the question, could there be an extension of the PRUFA, we don't have any current reason to believe that is the case because in our Latest communications, our PDUFA date as provided to us by the FDA is May 25th. So if there is any change to that, obviously we will, you know, update all of you.
spk03: Got it. Okay. And maybe last question. Just wondering if you've started label discussions or have had any preliminary feedback as it relates to the label and including the debt population in the label as well.
spk05: Yeah, thanks for that question. We have not had extensive discussions about the label language. As you can anticipate, this is something that happens in the April timeframe. We're still more than two months away from the PDUFA date. So as and when those conversations ensue, we will update you as well.
spk03: Understood. Okay, thanks for taking my questions.
spk00: Thank you very much. Just a reminder, if you have any remaining questions, please press star 1 on your phone keypad now. Your next question is coming from Kristen Kluska of Council Fitzgerald. Kristen, your line is live.
spk02: Hi, good morning, everybody. Thanks for taking the questions. I have a few here. The first is, can you clarify the 45-day window? Is that from March 1st or March 15th? And then you noted that the specific comments were something that weren't atypical. So can you just maybe speak to your level of confidence in the response? I mean, you were able to kind of submit this within two weeks. So I guess that backs up that it was maybe something that was on your mind or radar and that you had been preparing for this regardless.
spk05: Thank you, Kristen. Yes, so our understanding is that the 45-day window is triggered from the March 1st date, which was when the inspection concluded. And yes, these are things that we have ourselves. In fact, even before the inspection took place, we've started to implement some steps because we were aware of all of those points that were discussed. So as you mentioned, the very fact that these are all in process is how we were able to respond even within that 15-day window. So, yeah, we believe whatever steps we're taking is exactly consistent with what guidance the FDA themselves provided as well.
spk02: Thank you. And then just on the cost side, there's a recent JAMA article highlighting that standard of care can cost the average patient somewhere between 10 to 20 million dollars in a lifetime so now that people are doing a lot more analysis on this curious how you think something like this could factor into your therapy if approved and you know while i respect you haven't announced the price yet can imagine it would be lower than than what was cited there
spk05: Yes, hey, Kristen. It's Madhav here. Absolutely, that's your point on the JAMA paper, Raymakers et al. is put on. And in fact, in that paper, they have used a $300,000 estimate for the cost of the two-week when that was done. And of course, now we know that that is not the case. So we are evaluating what are therefore our pricing or value proposition should look like. And in fact, we are getting feedback from payers because the earlier market research on pricing we had done was before Vajovec was launched. And so we still are in the process, and as you can appreciate, we are not able to actually pinpoint the price range, primarily also because some patients with BZ cell could get more than one treatment, and hence how do we value what the pricing model should look like. But we'll stay tuned. We will look to have that as soon as we are able to have some confidence and as we get closer to PDUFA.
spk02: Okay, thank you for that. And then last question for me is just on the inspection side. Other than this outstanding Form 483, are there any other large check marks or things you're waiting for from the CMC manufacturing side of things just ahead of the PDUFA date? Thanks again.
spk05: Thank you, Kristen. Yeah, as I mentioned, the review is still ongoing, and the formal inspection step, the 483, we've answered to the 483. So the EIR will be the final result of that. We don't have anything else from the 483 per se. The review aspect is different. That's a different train that is running in parallel. So that is still ongoing, and we are answering the questions that are coming through these information requests. Any updates that we learn, we will relay that.
spk02: Thank you.
spk05: Thanks, Kristen.
spk00: Thank you very much. If you have any remaining questions, you can press star 1 on your phone keypad now. Okay, we don't appear to have any further questions in the queue. I am now going to hand back over to Vish for any closing comments.
spk05: Thank you. In closing, we're very pleased with the meaningful progress we've made over the last 12 months. Based on our strong clinical data and enthusiasm from the medical community, we believe that PZ Cell could be an important potential treatment option for patients. With our depth, we have a strong foundation to support the launch of PZ Cell when approved. Based on our clinical data, manufacturing capabilities, commercial readiness efforts, an extensive and diverse commercial and launch experience with pathologist cell therapies. Thank you, everyone, for joining us for today's quarterly call. With that, we'll talk to you again soon.
spk00: Thank you very much. This does conclude today's conference. You may disconnect your phone lines at this time and have a wonderful day. Thank you for your participation.
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