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spk06: Greetings and welcome to the Addi Bioscience Incorporated first quarter 2022 earnings conference call. At this time, all participants are in a listen-only mode. A brief question and answer session will follow the formal presentation. If anyone should require operator assistance during the conference, please press star zero on your telephone keypad. As a reminder, this conference is being recorded. It is now my pleasure to introduce your host, Andrew Kwan, VP of Business Development and Corporate Strategy. Thank you. You may begin.
spk03: I would like to welcome everyone to the first Addi Bioscience Quarterly Results Call for Q1 2022. With us on the call today from Addi Bioscience are Neil Desai, founder, president, and CEO, who will give a quick overview of the quarter, Brendan Delaney, Chief Operating Officer, who will provide early commercial uptake commentary and metrics, Dr. Loretta M. Eitri, Chief Medical Officer, who will walk us through clinical development priorities and forthcoming updates, and Scott Giacobbello, Chief Financial Officer, who will provide an overview of our financials this quarter. Before we get started, I would like to remind everyone that statements we make on this conference call will include forward-looking statements. Actual events or results could differ materially from those expressed or implied by any forward-looking statements as a result of various risks, uncertainties, and other factors, including those set forth in the risk factors section of our annual and quarterly filing with the Security and Exchange Commission, and can be found at www. or on our website at www.addibio.com. In addition, any forward-looking statements made on this call represent our views only as of today, May 12, 2022, and should not be relied upon as representing our views as of any subsequent date. We specifically disclaim any obligation to update or revise any forward-looking statements. With that, let me turn the call over to Neil.
spk04: Thank you, Andrew. Good morning, everyone, and thank you for joining. The first quarter of 2022 was transformative for Addi Bioscience. We successfully transitioned into a fully integrated biopharmaceutical company, commercializing and launching our first product, Phyaro, an albumin-bound mTOR inhibitor, also known as Napsirolimus, for advanced malignant pachoma on February 22nd of this year. We delivered on our promise to begin enrolling patients in our new registration-directed tumor agnostic trial, known as Precision 1, in March. In the first quarter, we have continued our company building and recruitment of high-quality talent in all areas of the organization. And our cash position remains strong at approximately $130 million and is expected to continue funding our commercial and R&D operations into 2024. Our key priorities in 2022 are to maximize the clinical and commercial potential of FIRO to further strengthen our ability to create long-term value. FIRO is now the first and only FDA-approved treatment specifically indicated for advanced malignant picoma patients, and the first and only NTOR inhibitor approved for this indication. Even though advanced malignant pachoma is an ultra-rare sarcoma with around 100 to 300 new patients per year in the U.S., the launch parameters for FIARO in advanced malignant pachoma are encouraging. While we are still early in our launch, it is clear that FIARO has been well-received by patients and their treating physicians alike. In six weeks since launch to the end of the first quarter, we achieved 2.3 million in net sales of FIARO. We attribute this encouraging performance in part to both pent-up demand and transitioning of expanded access program and MPEG clinical trial patients to commercial product, and Brendan will discuss this further. In general, we are very pleased with the high level of awareness and interest among physicians, favorable payer sentiment, and strong patient demand. Early in the first quarter, we received recommendation by the NCCM guidelines highlighting FIRO as the preferred treatment for PECOMA. And very recently, we have secured a permanent J code for FIRO, which will be effective July 1, 2022, and should make prescribing FIRO even easier for physicians. I would like to thank Brendan and his entire commercial team for executing such a great start. We believe this quick adoption of FIRO is clear evidence that we have a highly differentiated product in a difficult-to-treat cancer with a dire unmet need. And Brendan will have a lot more to say on this topic in a few moments. On the R&D front, we are very pleased with our progress on Precision-1, our registration-directed tumor agnostic trial for MAP serolimus in TSC-1 or TSC-2 alterations in solid tumors. This trial has the potential to significantly broaden the future application of MAP serolimus across many different tumor types. We enrolled our first patient in March and are steadily working to open high-quality clinical sites. We are further augmenting enrollment via partnerships with NGS providers and community oncology networks. With these patient recruitment strategies in place, we feel confident about our intention to present preliminary data from Precision 1 in the first half of 2023 and complete the study in 2024. In addition to the PRECISION-1 trial, our team is also evaluating options for new single-agent or combination treatments with Napsirolimus with the goal to kick off new clinical studies in 2023. Loretta and her clinical team have done a terrific job in getting the PRECISION-1 trial up and running. She will give additional color on this topic shortly. In addition, in the first quarter, we have continued our company expansion and recruitment of high-quality talent in all areas of the organization. We have recently established a new office in Morristown, New Jersey, a national hub in the Northeast for oncology expertise and industry talent. And so we are now officially bi-coastal. In summary, Addi is firing on all cylinders and working to maximize the value of FIRO for our shareholders. while helping the many patients with unmet need to gain access to this important and novel drug that efficiently targets the mTOR pathway. Our goal, indeed, is to establish Addi as the leading company in precision oncology. Brendan will now discuss our launch and commercial progress in greater detail.
spk10: Brendan? Thank you, Neil. It is an exciting time at Addi Bioscience. and I'm thrilled to share results today from Addi's first partial quarter as a commercial stage company, as well as early insights regarding the launch of Faiaro in Pacoma. It is important to consider that Faiaro became commercially available on February 22nd, so the results that will be shared today reflect 28 selling days over a six-week time period. We are in the early days of the launch, so we will be discussing commercial trends on the call today. Even though early, we are very encouraged by the initial uptake trends of Fyaro. Underlying our success are the patients battling for coma who truly needed access to this important treatment option. We are humbled to help these people as it is they that represent the most meaningful success story of this launch. Since making product available in the market on February 22nd of this year, our launch has been driven by three strategic imperatives. First, we need to quickly establish FiARO as a standard of care in the frontline PECOMA setting. In order to accomplish that goal, our ability to accelerate brand awareness is paramount to success. It is too early to share meaningful brand awareness metrics. However, the breadth of ordering institutions and the early FiARO uptake across treatment centers leads us to believe that our brand awareness metrics are tracking on target. Second, our teams have been focused on managing expectations for treatment and educating healthcare providers to ensure a positive first clinical experience. Based on feedback from first-time FIARO prescribers, when volunteered by these early adopters, they have expressed a positive overall experience when using the drug. Based on early experience, it appears that these new users can effectively initiate treatment and manage patients through multiple cycles of therapy. And third, we need to establish Addi as a leading company in precision oncology. Throughout the early launch, we have continued to engage sarcoma key opinion leaders and have increased our visibility within the sarcoma patient advocacy community. And throughout the ongoing launch, we will continue to strengthen our partnerships and differentiate Addi Bioscience as a trusted industry leader in the PECOMA space. In six weeks since launch, we achieved $2.3 million in net sales despite the small and ultra rare nature of PECOMA. We attribute this encouraging performance to several factors. First, a group of PECOMA patients treated in the expanded access program and the AMPECT trial were successfully transitioned to commercial product within the first few weeks of March. Additionally, during the intervening period between our approval on November 22nd and the launch on February 22nd, pent-up patient demand had been building in anticipation of commercial product availability. Lastly, as specialty distributors were reacting to early patient volume, we observed some launch inventory build that we expect to dissipate as we reach more steady-state patient demand. Based on early feedback, the reaction to the FIARO clinical profile has been very positive. In addition to the reported overall response rate, the duration of response data and the disease control rate from the AMPAC registrational trial, as reported in the labeling, are seen as highly differentiating. Although early market share estimates can be unreliable, Recent market research conducted with oncologists for Addy in the U.S. revealed that intent to prescribe Fyaro in the first-line advanced malignant Pacoma treatment setting exceeded 70%. Momentum for Fyaro was further supported by the rapid update of the NCCN guidelines early in the first quarter to reflect Fyaro as the only preferred treatment option for Pacoma. As of March 31st, There were 35 accounts that ordered FIARO. Because PECOMA is a rare form of sarcoma, we expected to have a large representation of academic cancer centers as early adopting accounts, and that has proven to be the case, with approximately 75% of early orders coming from the academic treatment setting. More specifically, we have also had a strategic focus on the top sarcoma centers of excellence in the United States. which represent approximately 60 academic medical centers. I'm happy to report that through the first six weeks of launch, our sales team has detailed 85% of the high potential prescribers within these accounts. Recognition of both the unmet need and advanced malignant pachoma and the strong FIARO clinical profile has also been reflected in the sense of urgency with which payers have been adopting formal coverage policies for the product. Having formal coverage policies in place with large payers removes barriers for treating physicians and paves the way for broad patient access. As we have been monitoring the time to pay or review metrics for FIARO during the first six weeks of launch, we see that they are exceeding the performance of other oncology product launches. I'm happy to report that as of March 31st, payers covering 70% of commercial lives in the United States market have reviewed and adopted a formal FIARO coverage policy. I'm also pleased to report that the quality of that coverage is equally impressive, with the vast majority of policies implementing a prior authorization to prescribing information position without major restrictions. Very recently, we announced that CMS also approved our application for a new permanent J-code for FIARO, which will become effective July 1st. This is welcome news as a permanent J-code streamlines reimbursement across all sites of care. All of this rapid payer progress, combined with a strong suite of patient support resources available through our patient program, AddiAssist, has resulted in strong patient access for Fyaro since FDA approval. In summary, we are excited about the progress we've made since the launch of Fyaro, and we are happy that the coma patients now have access to this novel mTOR inhibitor. Before turning it over to Loretta, I would like to extend another sincere thank you to all my Addi Bioscience colleagues for making this early launch a tremendous success. Now Loretta will give you an update on our clinical program.
spk01: Thank you, Brendan. I'm proud to report important progress on several funds in the last quarter. First, we have significantly strengthened our R&D group, hiring high-quality talent and launching a fully operational clinical development division with all functions nearly completely staffed. This has enabled us to rapidly set up trial sites and facilitate patient recruitment while providing appropriate oversight of clinical trial conduct for the ongoing Precision 1 tumor agnostic trial. Second, we have enrolled our first patients in this trial at the Dana-Farber Cancer Institute and MD Anderson Cancer Center and have made great progress towards our goal of initiating the study in at least 20 major cancer centers in the United States by the end of this year. Next slide, please. In order to ensure strong enrollment in the Precision 1 trial, we have partnered with the leading next-generation sequencing providers, including Foundation Medicine and Tempus. We are working closely with these and other NGS providers who are helping to identify patients with TSC1 or TSC2 alterations, and also utilizing their physician networks to recommend participation of appropriate patients in the PRECISION-1 trial. Next slide, please. Importantly, we have also formed a partnership with U.S. Oncology to leverage its entire physician network of more than 1,400 oncologists. That includes more than 500 cancer treatment locations and more than 1.2 million patients participating in clinical trials annually. This partnership will help us target community centers as well as additional academic centers in our enrollment efforts. Using USO's STAR program, we can rapidly activate individual sites once a patient is identified who qualifies for the study. Taken together, this series of focused activities should augment enrollment into the Precision 1 trial by targeting both academic and community-based oncology practices and by rapidly identifying appropriate patients with TSC1 or TSC2 alterations who may benefit from trial participation and expediting their enrollment into this study. We believe that these efforts will drive full patient enrollment within the next 24 months. Next slide, please. As a reminder, Here is an outline of the trial design for the PRECISION-1 study. The PRECISION-1 trial is a multicenter, open-label, tumor-agnostic pivotal study of nab serolimus. The trial will evaluate approximately 120 adult and adolescent patients with solid tumors harboring pathogenic inactivating alterations in TFC1 or TFC2 genes. The trial will have two independent arms of 60 patients each to separately evaluate patients with either TSC1 or TSC2 alterations. Adi has received fast track designation to evaluate NAB serolimus in this indication from the FDA. The first patient was treated in March 2022. Last month, data on the incidence of TFC1 and TFC2 alterations were presented at the annual meeting of the American Academy of Cancer Research Conference. This work was supported by a grant from Adi and both confirmed and expanded the initial estimates that approximately 12,000 patients in the United States carry these gene alterations. The frequency demonstrated for this genetic alteration renders TFC1 or TFC2 one of the largest categories within targeted oncology, occurring in 2% to 3% of all solid tumor patients. These findings give us additional confidence regarding our ability to enroll a sufficient number of patients to complete the Precision 1 trial in a timely manner. The reception from the oncology physician community has been encouraging. It is unusual to have a newly approved agent that has already shown clinical proof of principle in an enriched group of patients within a specific tumor type. In this case, advanced malignant pachoma. This is especially compelling given the well-defined safety and pharmacology profiles of nab serolimus that are both known and in common practice. Although the analysis was exploratory, Impressive results were obtained in patients who expressed TSC1 or TSC2 alterations, which were seen in 56% of patients with advanced malignant pachoma, that is 14 of 25 biomarker-evaluable patients. Indeed, as reported in the Journal of Clinical Oncology, a high percentage of patients with a known inactivating TSC1 or TSC2 alteration were that is 9 of 14, or 64%, in the AMPACT registrational study of patients with advanced malignant glaucoma achieved a partial response or a complete response with impressive durability. We continue to carefully evaluate other potential opportunities for either single-agent or combination therapies with nabsorolimus to expand use in other patient populations. We believe there are very interesting opportunities across different indications, and we have been inspired by the many suggestions we have been receiving from the community. I will now turn it over to Scott for a financial update.
spk11: Thanks, Loretta, and good morning. I'll now summarize financial results for the quarter ended March 31st, 2022. A more detailed discussion of results will be provided in our 10Q to be filed later today. We ended the quarter with cash and cash equivalents of $129.8 million. Based on our current plans, we expect cash and cash equivalents to fund operations into 2024. Total revenue for the quarter was 2.3 million, consisting entirely of net product sales of Faiaro following the February 22nd launch. As Brendan mentioned, this number reflects AMPAC trial and EAP patients who transitioned to commercial product during the quarter, pent-up patient demand related to the timing between FIARO approval and launch, and initial inventory bill at the specialty distributors. Cost of product sales amounted to $0.2 million for the quarter, consisting primarily of royalties on FIARO sales. Cost of sales is favorably impacted by FIARO inventory costs incurred prior to FDA approval, which were expensed as research and development in the period incurred. Research and development expenses for the quarter were $6.8 million compared to $3.6 million in the prior year quarter. This increase is due primarily to costs related to the initiation of the Precision I trial and the build out of the R&D organization. Selling general and administrative expenses increased to $9.1 million in the quarter from $0.6 million in the prior year quarter. This substantial increase is mainly the result of expenses related to the build-out of our commercial operations and infrastructure and increased marketing expenses to prepare for the commercial launch of FIARO. The resulting net loss for the quarter was $13.9 million compared to $5.5 million for the prior year quarter. Thank you, and I'll now hand the call back to Neal.
spk04: Thank you, Scott. In summary, we are very pleased with the early acceptance of FIRO by the physician and patient communities, and we remain encouraged by the trend seen so far. We are intently focused on enrollment in Precision 1, and look forward to maximizing the application of NAPS serolimus to treat other cancer types. Before we open the call for Q&A, I'd like to take a moment and thank the entire team here at Addi Bioscience for their impressive focus, hard work, and execution over this past quarter. Looking forward, I'm confident we are well-positioned to continue to execute successfully, both on our FIARO commercial launch, building on the momentum of our first partial quarter that we reported today, and on the R&D side, our continued execution of our Precision 1 tumor-agnostic trial. With that, operator, I'd like to open the call for questions.
spk06: Thank you. We will now conduct a question and answer session. If you would like to ask a question, please press star one on your telephone keypad. A confirmation tone will indicate your line is in the question queue. You may press star two if you would like to remove your question from the queue. For participants using speaker equipment, it may be necessary to pick up your handset before pressing the star keys. One moment while we poll for our first question. Our first question comes from Boris Peeker with Cowan. Please proceed.
spk09: Good morning, and congratulations on the progress. Can you hear me, guys? Yeah, Boris, I can hear you.
spk12: Fantastic. So my first question is, you mentioned that part of the revenue was inventory build. Can you comment approximately how much? Do you have an estimate or an estimate of end-user demand instead?
spk04: So, hi, this is Neil. Brendan, would you like to answer that question, please?
spk10: Yeah, I can answer that. Thanks, Boris. You know, Boris, it's hard to comment and quantify it. What I would say is that, you know, usually, obviously, it's expected in an oncology launch that you'd have some built up inventory. I think here, you know, you're probably a couple of weeks on hand above what we would expect. And I think that's a little bit of, you know, specialty distributors reacting to higher than anticipated volume early on, right? So I think that's part of it. It's hard to quantify, though, because there's multiple levels of inventory that we don't see. For example, a lot of the ordering, as you heard in the prepared remarks, was done by academic institutions, many of which are carrying some inventory as well in addition to the specialty distributors. So it just introduces another, you know, level of complexity in trying to quantify it. I would say we're pretty excited about, you know, what we're seeing and, you know, extrapolating as underlying patient demand. But I will say also the pent-up demand in the situation where we launched in or received FDA approval in November and ultimately launched in February. I think patients were building at that time as well, and that certainly also contributed to, I think, an exciting start here.
spk12: Great. And just how long do you anticipate these patients to remain on treatment?
spk10: Again, hard to say, Morris. I think it's encouraging what we see as far as reorder patterns, right? But we don't have patient-level data to track individual patients. But I think when we see you know, that accounts that ordered are reordering. It's certainly encouraging, but that's a, you know, a loose kind of metric for duration at this point, which is going to be very difficult, not, not only in this early part, but for the next, you know, months ahead to, to really determine. But I think it's early, but we're encouraged by what we see. That's the best.
spk12: My last question for precision one. You mentioned the first interim is going to be in the first half of next year. Can you comment on what we should be expecting in terms of the number of patients or any other expectations for that interim update?
spk04: Yeah, hi, Boris. This is Neil. I can take that one. So as we've said before, this trial has just begun enrolling, and so the visibility into the enrollment rate, et cetera, will, of course, come but that'll come later in the year as many more centers get online. So in terms of the exact sort of nature of the update that we would provide in the first half, it's hard to predict exactly the number of patients, but that said, we expect a meaningful number of patients so that investors and analysts like yourself can look at the data and feel confident about the future. of the drug. In terms of the other types of analysis or information that we would provide, we would probably give some color on the different tumor types that have been enrolled in the study and give some information about the outcomes in these patients at that time. But again, it's a bit early right now to predict exactly the nature of the outcome. We should also keep in mind that this is a registration trial, and so there are some limitations thereof. And secondly, and importantly, we're in the unusual situation that we have an approved drug that is being marketed for advanced PECOMA, but we have to be very careful of off-label type promotion and how any data we put out there can be construed. So with those limitations, the goal, of course, is to give investors a look at what we're seeing in the trial early on.
spk12: Great. Thank you very much for taking my questions, and congratulations on the progress.
spk06: Our next question comes from Arul Demir with Lattenberg-Dalman. Please proceed.
spk05: Good morning. Congratulations on the progress, and thanks for taking my questions. I have two questions on the commercial side then. we'll go into the precision one. On the commercial side, could you please comment on the number of prescribers and also payers coverage considering Medicare versus the commercial payers aspect?
spk09: Yeah, how are you? Oh, sorry. Go ahead, Brendan.
spk10: Hey, yeah, sorry. Yeah, so on the, we don't have visibility on the prescribers. As you know, with an IV therapy, Once the vials are shipped from the distributors to the institutions, we lose visibility in many levels, including the patient level. So we don't have patient level or prescriber level type of information. The best we have is institution level, which we reported there that in the first six weeks, 35 institutions have ordered. And then your second question on the payer was the mix between commercial and Medicare. Okay.
spk05: That's correct. Yes, Brendan.
spk10: Yeah, so we don't have a specific mix of what it is on the snapshot. The anticipated payer mix is around 50% to 60% commercial payer, and maybe in the 30% range on Medicare. Keep in mind that that's driven by the nature of PECOMA, where it's more prevalent in females who are under the age of 65, so the payer mix makes sense. But as far as commenting on The mix, you know, as it stood in the six weeks, it's very difficult to do. I would say that we've been very encouraged by the response we've had from payers, as I mentioned in the prepared remarks, with how quickly they're putting coverage policies in place, but also their policies without restrictions. And that's very encouraging. So on the payer front, it looks very positive as well.
spk05: Thanks for that information, Brandon. My other question is on the Precision one. How many sites are currently open, and how many additional sites are we expecting to open up this year?
spk04: Yeah, thanks, Ahu. This is Neil. Loretta, would you like to answer that question, please?
spk01: Sure. Currently, we have six sites open here in the United States, and you can Take a look and see who they are on the clintrials.gov website. We update that periodically. Our goal is to open at least 20 major cancer centers here in the United States by the end of the year. And of course, that will be augmented by our partnership with U.S. Oncology, et cetera. So that is our goal, and we're on target, and we have every intention of achieving that.
spk05: Thank you. If I may, I have one last question. At the AACR presentation, the combinational strategies were disclosed as IO, BRAF, and FGFR3 inhibitor combinations. I'm just curious if you could elaborate more on that, specifically FGFR3, since They are more specific to urethral cancers. Just curious on your comments.
spk04: Yes, so this is Neil. I can take that one. Again, in the ACR presentation, the key information that we presented was the incidence of TSC1 and TSC2 relevant alterations, you know, in terms of number of patients on an annual basis in the U.S., which is about 12,000. It was also interesting to note that we looked at other type mutations that can co-occur. Overall, they were a small percent of the total TSC1, TSC2 population, but the suggestion there is that given some co-mutations in the future as we analyze other strategies for combination therapies, we will be looking at strategies like that. So, for example, which combination drugs can we use along with our drug? We haven't finalized that yet, and that process of evaluation is ongoing, as there's many types of very interesting combinations that we can get into. But that look and that presentation of data certainly helps us in finding populations that may be highly relevant in the future.
spk05: Very helpful. Thank you very much. I appreciate you taking the time to answer my questions.
spk00: Thank you.
spk06: Our next question comes from Joe Cantazzaro with Piper Sandler. Please proceed.
spk02: Hey, guys. Thanks for taking my questions and congrats on the progress here. Maybe one first on the FIARO launch. Wondering if maybe you could help us think about the rest of the year commercially and speak to whether there are still any EAP patients left that need to make the transition as well as how we should think about the pace of new patients starts moving forward and following sort of this pent-up demand that you experience. Thanks.
spk04: Yeah, thanks, Joe. Brendan, would you like to take that, please?
spk10: Yeah, you know what, Joe? I appreciate the question. It's early at this point, as you know, and I don't necessarily want to make any kind of predictions because there's multiple levels of complexity here, as you can probably appreciate. I think we're off to a good start. I think some of the new patients that will come over early on, we expect to have some duration on that, so that will obviously play out. for the remainder of the year, as well as new patient demand coming in. As far as the EAP patients, we don't have 100% visibility, but we feel pretty confident that the majority of those patients were transitioned successfully by the centers, and we wouldn't expect any necessarily additional, you know, from either AMPACT or EAP coming in. Same with the pent-up demand. that we're describing. I think that was, you know, patients building from November to February and they were waiting for the drug to be available in the market and got on pretty quickly. Again, those are estimates. It's a little early to comment on what it looks like for the rest of the year. As I said, we're encouraged by what we're seeing, but I don't think we're at a steady state, you know, underlying demand and able to predict what happens in the coma given the ultra rare nature of the disease. So I hope that helps.
spk02: Yeah, no, no, that's helpful. Maybe another follow-up here. I think last you guys had mentioned that you were going through an EMA scientific advice process. Just wondering sort of where you guys are there and what your current expectations are around a potential EMA filing.
spk04: Yeah, Joe, this is Neil. I can take that one. So, yes, we are in that process. And as we mentioned before, we expect to receive that scientific advice later in the second half, probably later in the fall, just based on timing and backup of the EMA. They've indicated several times that they're very backed up in terms of the process. So hard to predict exactly when, but we expect that later in the fall we would have gotten that advice so we can update following that.
spk02: Okay, got it. And then if I could maybe squeeze one in on precision one, wondering if you just have any comments on the early experience screening patients for TSC1 and 2, and maybe your early ability to confidently call if the alteration is pathogenic or not.
spk04: Yeah, I'll comment and then pass it on to Loretta. So in terms of screening, I'm not sure that we can provide any information at this point, since the process is very early. But, you know, the patients go through the standard NGS screen, and then we have a central reviewer. Loretta, would you like to add anything to that, please?
spk01: No, I'm really not clear on what the question is, but it has not been a problem. TSC-1 and TSC-2 are routinely included in virtually all NGS reports, so it's not hard to find them. The issue of whether or not they are inactivating I think may be the question you're asking. The way we handle that is that we actually have provided sort of a cheat sheet to all of our participating institutions so that it enables them to translate the NGS report on their own. So by the time we get the NGS report with the patient, we're pretty certain that they are inactivating. And it then has a less than 24-hour turnaround by our central reviewer. and we can confirm whether or not the TSC abnormality is indeed truncating or inactivating. So, you know, we're a pretty fine-tuned machine at this point. We can turn them around very quickly. So that, at this point, does not appear like it's going to be any kind of obstacle for us.
spk02: Okay, great. Yeah, and that was what I was sort of asking about, so that's helpful. Okay. Thanks again for taking my question.
spk06: Once again, to ask a question, that's star 1 on your telephone keypad. Our next question comes from Roger Song with Jefferies. Please proceed.
spk07: Okay, great. Congrats for the initial launch. A couple questions from us. So the first question is, what is the current reimbursement process before the J-code and how this J-code will facilitate the reimbursement and the Sequentially, how do you expect the growth to net will evolve over time? What is the kind of steady state kind of growth to net you will imagine?
spk04: Thank you. Hi, Roger. This is Neil. Thanks for joining. You were a little soft there, so if you don't mind just repeating the question again so we make sure we heard it clearly, a little bit louder. Thank you.
spk07: Yeah, sorry about the voice. So can you hear me now?
spk04: Yeah, I think that's better. Thank you.
spk07: Okay, great. So the question is, what is the current reimbursement process? And how will the JCODE facilitate the future reimbursement? And specifically, how do you expect the growth to now will evolve over time? What is the steady state growth to now will look like in your kind of a model?
spk04: Okay, thanks. We could hear that well. Brandon, would you like to take that, please?
spk10: Yeah, Roger, how are you? I think on the J-code, it really just streamlines the process, right? So currently, a lot of payers have come on with, you know, as I mentioned, positive policies with a prior authorization to label, which is not that restrictive. I think what's A lot of this, the billing is done on the Medicare side through a temporary code, a miscellaneous J code. And that leaves a lot of prescribers, especially if you're in the community setting, uneasy about getting reimbursement for the drug. Also, sometimes things get held up, right, because it may get confused, missed, and it creates kind of burden for the prescribers and the patients as far as their time to get on therapy. So what the permanent J code does is assigns a specific code for Fyaro. So you don't have any of that, you know, perceived risk. And it's just much more streamlined because it has its, you know, specific code in place. There's obviously a process to apply for that. And that's that we've executed on very well. And we're getting the J code at the earliest possible time. But this is all normal, Roger, for launch execution and oncology with a, you know, an IV therapy. And I think we've executed on it very well. I don't think, you know, we've seen any type of as far as the current process with the miscellaneous J code, we haven't seen any obstacles or heard any pushback, you know, in the field. Now, part of that may be related to, you know, as I mentioned, the majority of our use early on, which is expected is in the academic medical centers, and they have, you know, a lot more resources to make, you know, work the process, the reimbursement process seamlessly, even in the early days of launch. And then, Neil, did you want me to comment on the gross net question as well?
spk04: Yeah, go ahead. And if Scott needs to add, he can certainly do that.
spk10: Yeah, I think we estimate now, Roger, but I think, you know, somewhere estimate within the 15 to 20% range for GTN is probably a safe estimate at this point. And hopefully, you know, as time goes on, we'll get better information and see what that looks like. But I think Right now, between 15% and 20% is probably a safe estimate. Scott, I don't know if you have anything else to add.
spk11: Yeah, no, nothing really to add other than to say it's hard to say how it will evolve. Roger, obviously dependent on how the commercial mix shakes out over time. But yeah, I agree with everything Brendan said on where ultimately we think it will probably end up.
spk08: Okay, great. Yeah, thank you for
spk07: the color, maybe just a squeeze one for precision. Yeah, I think you provided some very good color around the screening turnaround. Maybe just ask specifically, do you have an estimate timeline from the patient enrollment, fund of consent, and all the way to you can say definitively the patient get this diagnosed with GSD1 and GSD2 inactivating mutation, what is the overall timeline look like? I understand when you get an NGS report, you can get the final diagnosis within 24 hours, but just curious about the whole process timeline look like. Thank you.
spk04: Yeah, thanks, Roger. Loretta, would you like to just comment on the question? Were you clear on the question, first of all? I think the question was timeline from consent essentially to the time the patient officially gets on the study or receives the drug?
spk01: Well, by the time you get consent, the patient is really quite far along. They've been identified as having the abnormality. They have qualified for all of the inclusion, exclusion criteria. So for the most part, I would say from the time we consent to patients, it's relatively short. I would say two to three weeks before the patient actually goes on study. But, you know, these being patients who are seriously ill. You just never know what's going to happen. And so with COVID around, sometimes they will get a COVID infection and that puts it back another 10 days. So it's variable, highly variable. But in general, I would say two to three, four weeks, about in that range. I don't know if that's helpful, but that's what we're seeing so far.
spk08: Got it, yeah, it's helpful. Okay, so that's it for now. Thank you.
spk06: Thank you, Roger.
spk08: You're welcome.
spk06: Thank you. At this time, I would like to turn the call back over to Dr. Neil Desai for closing comments.
spk09: Thank you.
spk04: On behalf of the entire team at Addi, I would really like to thank the investors and analysts for joining the call today. We certainly look forward to interacting with you in the future. and then providing any necessary updates as we continue to progress and build value in the company. And thanks very much again for joining, and we can close the call now.
spk06: Thank you. This does conclude today's teleconference. You may disconnect your line at this time, and thank you for your participation. Have a great day.
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