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spk05: Thank you for standing by and welcome to the Crystal Biotech Third Quarter 2023 earnings conference call. As a reminder, today's conference call is being recorded. I will now like to hand the conference over to your host, Meg Dodge, head of investor relations and corporate communications. Please begin.
spk12: Good morning and thank you all for joining today's call. Earlier, we released our financial results for the third quarter of 2023. The press release is available on our website at crystalbio.com. Our earnings 8K was filed earlier today. And additionally, we filed our 10Q with the SEC. Joining me on the call are Krish Krishnan, chairman and chief executive officer, Suma Krishnan, president of research and development and Kate Romano, chief accounting officer. I'd like to note that during this call, we'll be making a number of forward looking statements about our future business plans, strategies, financial performances and projections, product candidate development plans, including statements about Vajavac. These forward looking statements involve risks and uncertainties, any of which are beyond Crystal's control. Actual results could materially differ from these forward looking statements as any and such risks can materially and adversely affect the business, results of operations and trading price of Crystal's common stock. For a detailed description of applicable risks and uncertainties, we encourage you to view our SEC filings. The company does not undertake any obligation to publicly update its forward looking statements, including any financial projections provided today based on subsequent events or circumstances. With that, I'd like to now turn the call over to Krish. Krish.
spk07: Thank you, Meg. Welcome to Crystal Biotech's first ever earnings call. It's a bit of a coincidence that we're having our first earnings call when we have positive earnings, but I want to be clear that this quarter's positive EPS is simply driven by the sale of the priority review voucher in Q3. More importantly, we're having this call on the heels of EB awareness week. We thank the entire dystrophic epidermalosis and bolosa community who worked tirelessly to improve outcomes for patients with the genetic disease. As we recognize the challenges that the patients and their families face every single day, our goal remains focused on helping as many deaf patients as possible. We know that this disease extends far beyond the skin and we're committed to continuing to work to treat this disease comprehensively. We will discuss our early initial efforts in treating deaf patients with lesions in the eye and those with squamous cell carcinoma of the skin later in this call. With that, I'd like to say that after the first full quarter into the Vizovec launch, I'm extremely pleased and proud by the commercial progress we're making. We continue to see strong patient demand into Q4 and as we announced, we finished Q3 with 284 stock forms since Vizovec was approved. Our estimate right now is about an 85% conversion from stock forms to patients on drug, but we'll continue to update the stat in subsequent quarters as more and more stock forms continue to convert. That said, even at 85%, we're at a 20% penetration of the identified base of patients following one full quarter post-launch. Of the 284 stock forms received, 20% were from patients suffering from the dominant form of TEB. As I have mentioned before, patients' scripts from dominant deaf patients continue to expand our base of identified patients. One third or 33% of the stock forms were from patients 10 years of age or younger to as young as six months old. We track that because it gives us a better sense of an overall estimate of the induction phase as we believe that patients younger than 10 years of age could potentially have a longer induction phase than adults, meaning that they will potentially consume more vials annually for a longer period of time than adults did. So of the 284 stock forms, 20% were from dominant deaf patients, 33% from patients 10 years or younger, and presently estimate an 85% conversion related to patients on drug, which could potentially be higher as we move into subsequent quarters. What is interesting and maybe a bit surprising is that only 46%, less than half of patients' stock forms, were from centers of excellence with a balance of scripts written by the community physician. So it has not been a bolus from the center of excellence, but rather a steady flow of stock forms. And this is because of the fact that it is attributable to, one, that some KOLs wanting a patient to physically visit at their centers prior to initiating them on Visevec. Now that takes a certain amount of time because a lot of patients do not live right next to the center of excellence, and scheduling can often be a challenge given the busy schedule of these KOLs. Some other KOLs, generally those who did not have prior experience with Visevec in a clinical study or in the open label extension study, are choosing a gated approach, prioritizing their most severe patients on Visevec first, seeing how it goes, and then writing prescriptions for moderate patients. That said, we've encountered no reluctance from any KOL with respect to wanting to put their patients on Visevec. It's simply a timing issue. And our medical affairs team is working closely with these KOLs to educate them on the importance of getting the patients started on Visevec as soon as possible. With respect to reimbursement, 45% of the patient stock forms that have been received as of the end of Q3 were from patients with a commercial insurance plan. Most of these patients, over 80% of them, are already eligible for commercial reimbursement. As we mentioned in the press release, the company has received positive coverage determinations from all major commercial national health plans and several regional health plans. So 45% commercial insurance, of the remaining 55% on government insurance, 74% are presently eligible for reimbursement. And we expect the remaining to be eligible once the permanent J-code becomes available in Jan 2024. So overall, access is in a really good place. And we expect almost all patients to be eligible for some form of reimbursement early in 2024, at which point we intend to transition to reporting number of patients on drugs as opposed to patient stock forms as patients on drugs will become a much better predictor of net revenues than stock forms. So we talked about color on the stock forms and on access, now about conversion to net revenues. Crystal's guiding principle in the Visevac launch is centered around the patient experience and we work tirelessly to ensure that each patient's path with respect to getting on Visevac and staying on it is smooth, timely and hassle-free. We're partnering with patients and families who were previously traveling to a center of excellence for palliative treatment and transitioning them to a home health visit by an HCP to apply the medication at a convenient time in their weekly schedule. Think about that. Definitely more steps involved than simply having a physician write a script and getting it filled at the local pharmacy. In addition, we're also working with payers who almost all agree that the home setting is best for the patient. So patient experience is foremost on our mind and with respect to the Visevac launch, we work to ensure that we do not make a patient wait for long, that we don't accept the stock form unless we have a clear line of sight into getting our patients access to Visevac within a reasonable time. Our goal in 2024 is to convert stock forms to -on-drug in about two to three weeks. We're not there yet, but we expect we'll have most of access in place by the end of the year and we're learning from some of the experiences on the initial set of patients so we feel really confident in achieving that objective. It's really difficult to go below two to three weeks because it takes many families a week or two to get comfortable with the nurse and schedule a home health visit. So this launch has been all about home dosing. Over 88% of the patient stock forms received by the end of September were for patients who want to be dosed at home. And we expect that trend to continue and potentially go higher. This has definitely helped adherence to drugs, which has been excellent to rate and currently tracks around 96%. We shall continue to update this statistic in upcoming quarters. So following approval in May, it took us a few weeks to get the drug in the channel, negotiate reimbursement, and schedule home health visits. So our first paid patient did not get on Visevac until early August. So the net revenue number of 8.6 million is approximately two full revenue months in Q3 during a period where both commercial and government policies and reimbursement continued to be negotiated an issue. The point being while patient stock forms is attributable to a full quarter, net product revenue is only attributable to two out of the three months in the quarter. So to summarize, we believe we have a strong launch in our hands. We see really good demand from both recessive and dominant patients. Access coverage has been relatively smooth. And home health visits are pointing to a high patient compliance. We expect this momentum to continue going forward. Beyond the U.S. commercial launch, we're also looking to expand the number of patients treated with Visevac, and we are working towards the named patient program in EU as we await our marketing authorization approval in the second half of 2024 and launch thereafter. Sumo will speak to the strength of our pipeline shortly, but with close to 600 million on our balance sheet, a strong launch and a very productive pipeline, we're well positioned strategically and financially to support the global launch of Visevac and advance our clinical programs. I shall turn the call over to Summa to provide color on the clinical programs.
spk09: Thank you, Krish. This is an exciting time for us in that with the approval of Visevac, our commercial team is leading the way for a successful U.S. launch. Our objective is to provide access to Visevac globally and someday treat this debilitating disease comprehensively. To that extent, we filed a marketing authorization with the European Medical Agency in October, and we anticipate an approval in the EU in the second half of 2024. Additionally, following acceptance of the open label extension study of Visevac by Japan's Pharmaceuticals and Medical Device Agency in July 2023, we initiated the extension study and dosed five patients. Following completion of the open label extension study in Japan, we intend to file a Japanese new drug application for Visevac for Deb in the first half of 2024. We are presently expecting launch in the EU and in Japan in 2025. With respect to treating this disease comprehensively, you have all seen the remarkable benefit in treating V.Vec patients with lesions in the eye, and we are in early stages of working with the FDA to develop an approach for studying Visevac for this indication. We estimate approximately 750 patients with this manifestation in the EUF. In addition, we are hoping to enroll V.Vec patients with spawned cell carcinoma of the skin in our phase one oncology study. While Visevac could potentially slow down the onset of SEC in the skin in the long term, having a near term benefit with KB707 will go a long way towards treating this disease comprehensively. Moving on to a rich clinical pipeline using our HSV1 platform, on KB407, we completed cohort one of the cohort one study with no severe or serious adverse events and plan on initiating cohort two in the upcoming weeks. We anticipate announcing data from this study in 2024. In addition, we continue to work closely with the TDN network of the CF Foundation to provide us access to the broader network, which will enable us to complete the study a lot faster than the pace we are at right now. On KB408, for the treatment of alpha one antitrypsin deficiency, which is formulated for inhale delivery to the respiratory cells of the lungs via nebulization, the phase one clinical trial is a phase one open label single dose escalation study in adult patients with AATD with a PIZZ genotype. Three planned dose levels of KB408 will be evaluated with three patients in each cohort to evaluate the safety, tolerability, and expression of the protein in lung cells and the serum. In September, we announced that FDA cleared our IND for KB408 and the agency granted KB408 orphan drug designation. We expect to dose the first patient in the clinic in a phase one clinical trial in the first quarter of 2024. Our oncology program, KB707, has made advancements this past quarter after the FDA cleared our IND and granted us a fast track application for KB707 in July for the treatment of locally advanced or metastatic solid tumor malignancies. As a reminder, KB707 is a modified HSV1 vector designed to deliver genes encoding both IL-12 and IL-2 to the tumor microenvironment and promote systemic immune mediated tumor clearance. We have two formulations of KB707 in development, a liquid formulation for intratumoral injection and an inhaled formulation for lung delivery. The intratumoral KB707 phase one OPAL1 study is an open label multi-center monotherapy dose escalation and expansion study enrolling patients with locally advanced or metastatic solid tumors who relapsed or are refractory to standard of care with at least one measurable and injectable accessible tumor. The primary objective of the study is to evaluate safety and tolerability of KB707. Advocacy will also be assessed by multiple measures, including overall response rate, progression-free survival and overall survival and the immune effects of KB707 monotherapy will be assessed in tumor, tissue, lymph nodes and blood. In October, the first patient was dosed in the phase one study to evaluate intratumoral KB707 in patients with locally advanced or metastatic solid tumor malignancy. We are on track to file an amendment to the existing KB707 IND in the fourth quarter of 2023 to allow us to evaluate inhaled KB707 in a clinical trial to treat tumors in a patient's lungs. We expect to dose the first patient with inhaled KB707 in the first half of 2024. Data was presented at the Society for Immunotherapy of Cancer in October. On localized delivery of comminuteroin IL-12 and IL-2 for the treatment of cutaneous malignancies, we presented preclinical data showing that intratumoral injection enhances tumor regression and survival in B16F10 murine melanoma compared to control or single vector treatment. Additional data was presented showing that when administered intratracheally, enhances tumor regression and survival in K7M2 murine, osteosarcoma lung metastasis model compared to control or single vector treatment. Regarding our dermatology program, KB405, for the treatment of TGM1, ARCI, and KB104 to treat patients with Nettleton syndrome, we continue to move forward with both programs. We are on track to commerce the phase two cohort of the KB105-02 trial for the treatment of TGM1-ARCI in 2024. For KB104, we plan to file an IND to initiate a clinical trial of KB104 to treat patients with Nettleton syndrome in late 2024. As stated in our press release, other pipeline programs continue to advance. I'd like to finish by saying we are presently working to advance four ongoing clinical programs and anticipate presenting clinical data across these programs in 2024, besides advancing our preclinical and clinical efforts in the skin and in ophthalmology. With that, I would like to turn the call to
spk06: Kate. Thank you, Summa. With our focus in the Vyjevec launch being centered around the patient's journey and resulting initial strong patient adherence on drug in the first few months of launch, we recorded $8.6 million in net product revenues, which began in August of 2023 through the end of the third quarter. As Vyjevec was approved by the FDA in May of 2023, there were no comparative period revenues. Cost of goods sold was $223,000 for the quarter as compared to zero for the previous year's third quarter due to initial sales of Vyjevec after FDA approval was obtained on May 19th, 2023. Prior to receiving FDA approval, costs associated with the manufacturing of Vyjevec were expensed as research and development expense, and as such, a portion of the cost of inventory sold during the period had been previously expensed prior to FDA approval. We expect that cost of goods sold will continue to be lower as we sell off the remaining inventory that had portions of its costs that were previously expensed as R&D prior to approval. Research and development expenses for the quarter were $10.6 million inclusive of $2.3 million of stock-based compensation compared to $11.5 million inclusive of $2.2 million of stock-based compensation for the quarter ended September 30th, 2022. This overall decrease of $887,000 was primarily due to costs related to the manufacturing of Vyjevec being recorded to inventory following our FDA approval that were previously expensed to research and development expense. Selling general and administrative expenses for the quarter were $23.7 million inclusive of $6 million of stock-based compensation compared to $19.9 million inclusive of $6.9 million of stock-based compensation for the quarter ended September 30th, 2022. This overall increase of $3.8 million was largely due to costs incurred related to launching Vyjevec such as salaries, travel, technology and other professional fees and was offset by lower marketing costs due to the timing of developing marketing materials. This quarter, we also recorded a gain from the sale of our rare pediatric disease priority review voucher which was awarded to the company in connection with the FDA's approval of Vyjevec of $100 million. I want to emphasize that this gain was a one-time item recorded in other income and was the primary driver of net income and positive EPS this quarter. Finally, we closed the quarter well-capitalized with $598.6 million in cash, cash equivalents and investments on hand as of September 30th. And we believe this cash on hand is sufficient to fund all of our planned activities for the next several quarters. And with that, I will turn the call back over to Krish.
spk07: Thanks, Kate. While Crystal has always been known for execution on the developmental front, the third quarter demonstrated our ability to execute equally well on the commercial front. We're now a fully integrated company with a commercially validated platform that allows us the privilege of developing medicines to serve patients with debilitating diseases. With a strong launch of productive pipeline and $600 million or so on the balance sheet, we are in a strong place financially to execute on our objectives. Finally, as the largest shareholders in the company, management is aligned and well positioned to continue to deliver increasing value to all our shareholders now and over the years ahead. Thanks for listening. And I'd like to now open the call for Q&A.
spk05: Absolutely. We will now begin the question and answer session. If you would like to ask a question, please press star followed by one on your telephone keypad. If for any reason you would like to remove that question, please press star followed by two. Again, to ask a question, press star one. If you're screaming today's call, please dial in and enter the number star one. As a reminder, if you're using a speakerphone, please remember to pick up your hands up before asking your question. We will pause here briefly as questions are registered. The first question comes from a line of Robert Finke with Guggenheim Partners. Your line is now open.
spk02: Hey, good morning, team. This is Robert on for digit. Thanks for taking our question and congrats on the strong launch. One question from us today, do you anticipate any slowdown in the fourth quarter compared to third quarter as far as patient start form rate? And if so, what does this imply about peak demands and problems? Thank you. Chris, excuse me, but there's nothing coming from your line. You might be on mute.
spk08: Oh, I'm sorry? I'm sorry. Oh, thanks, Robert. I was coming
spk07: on and on for a minute. So as of now, Robert, the pace continues to be as it was at the end of three Q. What is difficult to predict is how the holidays are, if at all, are gonna have an impact on the pace of start forms. So my best answer is as of now, we continue at the same pace and it remains to be seen how the next two months go by. And with respect to peak demand, look, when we made the call at the time of approval, it was simply based on a base of 1,200 to 1,500 identified patients paying about $500,000 annually. That's how we came up with 750 million. As we find more dominant patients, as we start getting approved in Europe and Japan, as the base of identified patient grows, we'll continue to update that number.
spk02: Great, thank you. And one follow-up from us. The start form numbers flowed from approximately 20 per week in the second quarter to 13.5 per week in the third quarter. What's your best explanation for why this has occurred? Next question.
spk07: Look, maybe the first six weeks were, there was a little bit of bolus from the open label extension study. As I mentioned in the call, we're also a bit careful of rushing to put somebody on a start form if we don't have a clear line of sight into when the nurse is gonna come home or they're gonna get reimbursed. The worst thing we can do is to have a patient submit a start form and have them wait a significant amount of time before converting. And so to some extent, we managed the inbound queue a little bit. That should start to open up. As I mentioned with access, we pretty much have majority of the commercial plans in place, a significant amount of the Medicaid plans in place, especially if I'm starting in October. And so some of it is the OLE, some of it a bit is our own doing to maximize patient experience.
spk02: Great, thank you.
spk05: Thank you. The next question comes on a line of Alex Shradahan with Bank of America, Maryland. Your line is now open.
spk04: Hey guys, thanks for taking our questions and congrats from me as well on the early launch progress. Just a couple of questions from us. Maybe first, could you walk us through the process a bit further of getting new patient start forms and then converting those patients onto therapy? And what was the barrier for maybe the 15% or so that didn't convert? And I think you just touched on this in the last question, but you think it's reasonable to assume that now that insurance is probably coming online that the rate of conversion between start form and initialization on therapy could accelerate?
spk07: We believe so. That 85% is an estimate, right? We don't have enough to make a very substantive prediction on that number. 85% is just based on some patients maybe wanting not to get going for a while, some patients do not have insurance, some patients choosing to wait to see how other patients are doing, so it's a pure estimate. We definitely expect that number to go up. I alluded to that in the call. But in terms of process, look, we like to get a quality and audited and audited start form to begin with because that helps from a timing perspective of converting them to patient on drug. Because if you don't have a fully filled start form with the physician report and the genetic sequencing, a lot of information on prior history of the patient, it takes longer, at least in the beginning, it took longer with insurance to get them reimbursed. And to avoid that, we were trying to get a clear line of sight before we totally accepted a start form internally because patients get very anxious once they submit a start form to get access to the drug as soon as possible. We tried to bridge that gap with some free, the free vial program while waiting for the insurance. And that varies depending on if you're commercial or Medicaid. And once we get a start form, it's essentially about a couple of things happen in parallel. One's about getting them on reimbursement. And the other path is to work with our specialty pharmacy to get the nurse scheduled to come home at a time that's convenient to them. And that usually takes a week or two. So that's essentially the process. But once the patient is on drug and the reimbursement is in place, the adherence rate has been really high because the nurse is essentially basically going home to fit with the patient's schedule as opposed to put any burden on the patient having to travel week after week to some site. And as I mentioned, a significant percentage of us, of our patients are on home dosing at the moment.
spk04: Okay, thanks. And one more question. When you look to approval and launch in the EU and Japan, Chris, how will you be approaching these markets? And if you do seek to partner, would you still be making VyjuVec yourselves presumably? And how would this work logistically?
spk07: Yeah, I think we're sticking to what we've been saying that our intent is to launch in EU-5 and Japan. We already have a team in the EU. We're starting to think about building out a small team in Japan in anticipation of a launch in 2025. In terms of supply of the drug, it'll all be supplied from Enchorus, which has the capacity for a global supply of VyjuVec at the right point in time, maybe supplemented with Astra, which is now up and running. So essentially we do not anticipate setting up any manufacturing facility in either Europe or Japan.
spk04: Okay, great. And thanks. Congrats again on the progress.
spk08: Thanks, Alan.
spk05: Thank you. The next question, press on the line Aritu Barrow with Cowan. Your line is now open.
spk11: Good morning, guys. Thanks for taking the question and thanks for hosting the call this time. Krish, I wanted to sort of get my arms around the sort of intent to prescribe. So you mentioned that the 284 START forms, if I'm interpreting your answers correctly, the 284 START forms that you reported this morning, these are high quality accepted START forms. And there are additional START forms that either you have not accepted or that you're sort of pushing back and I guess waiting to tally as part of your report. Can you give us an idea, qualitative or quantitative, about the number of outstanding like half filled START forms or inadequate START forms just to give us an idea of intent to prescribe? And then I'm also wondering about the non-center of excellence prescribing doctors. Is there a profile of these doctors that is emerging? And then I have another follow-up, thanks.
spk08: Yeah,
spk07: Sav, the second one first. Predominantly, P. derms is a profile of the community physician, but that varies. We could be a dermatologist. Some of the patients go to blood transfusion centers, probably getting tired of going to dermatologists and managing palliatively at home in the past. But in terms of the START forms itself, we like to accept a very high quality START form. Sometimes we make exceptions when there's an urgent need or a request by the physician, but by and large, the START forms are highly audited. In terms of providing any kind of guidance on how many is in the queue or that we're trying to convert, I think it would be a bit premature and not right for me to talk about that. But I will say that we expect, now with access in place, we expect the pace to be just as good, if not better, going forward.
spk11: Got it. And then I just wanted to ask a follow-up on the persistence rate. How are you defining that 96%? Is it patients who are reimbursed and who either skip a week or are you finding, are you tallying patients who skip like two weeks and don't intend to resell? I'm just wondering how that 96% is defined. Thank you.
spk07: It's basically, I mean, to simplify, look, it's, if you can see my bile a week, sometimes once in a while somebody misses a visit for some personal reason or schedule, but most of the patients on drug to date are at four vials a week, whether they're recessive or dominant. So when I say compliance is really high, it means worshiping four vials a week to a patient at the moment.
spk11: Got it. Thank you very much.
spk07: Four vials a month. I'm sorry. I meant to say a month.
spk11: Yep.
spk05: Thank you. The next question comes from a line of Carly Kanzler with Citigroup. Your line is now open.
spk13: Great morning. Thank you for taking my questions. Two questions for me. First, on the reimbursement side, just wondering if there have been any surprises with respect to the policies insurance companies are putting in place, particularly as it relates to the prior off process, just anything unexpected there. And then the second question is if you can just remind us what proportion of patients, of the identified patients are tied to centers of excellence in the US. Thank you.
spk08: On access, Carly, thanks for the question, Carly. On
spk07: access, things have been relatively smooth. There's nothing unexpected. We have a good system of offering contracts to payers if they are accepting, then they get eligible for the price gap. So both on the commercial side and on the government side, access has been at a good pace, like we're pleased. We expect to get the J-code finalized, officially published in January, which is, I think, on time based on when we got approval. So overall, pretty pleased, nothing unexpected. In terms of patients at the center of excellence, that's a great question, it's a tough one, because a lot of patients who once saw a physician at a center of excellence, a lot of them have actually stopped and gone back to the local community in the absence of an approved drug. And so trying, what we like, the way we think about it is the number of active patients presently at a center of excellence. And so, which is why, if you look at the star forms, if you think about like only 46% came from the center of excellence, we believe there is demand left at the centers of excellence, limited by the two factors I mentioned, one, physicians wanting to literally have a patient visit prior to getting them on Visevac, which happens in various diseases quite a bit, and it's a tough one to overcome, because they'd like to see the patient talk to them about it, at least some of them. And the second is something we're trying to educate around, which is when physicians decide, hey, I'm gonna put a handful of my patients, see how Visevac works, before opening the gate to the remainder of the patients. And that we disagree a bit with, and we're using our medical affairs to work very, I mean, very closely with these KOLs to convey the urgency of getting the patient on drug.
spk13: Okay, that's very helpful, thank you.
spk05: Thank you. The next question comes from a line of Dei Gong Ha. With Stifle, your line is now open.
spk01: Hey, good morning, guys, thanks for taking the question. Thanks for the first call, and congrats on the progress, Krish, maybe one more on the Visevac before switching over to KB407. In terms of the third quarter, I guess, can you talk about sort of the cadence of start forms that kind of came in? I know you don't wanna talk about sort of the queue, but what can you qualitatively say about that? And bearing in mind the holiday season is upon us, is that cadence at all kind of representative, you think, or at least 20%, 30% haircut? How should we think about that from a modeling perspective? And then second question on KB407, just wondering if you can narrow that guidance in 24, is it closer to ECFS or NACFC? And just looking at the competitors' recent data, it seems like expression of the protein may not necessarily be representative of clinical profiles. So can you maybe remind us what's the differentiation that you think you're going for? Thanks so much.
spk07: Yeah, look on cadence with the holidays. I wouldn't, I wasn't advocating any kind of discount. I was simply saying that the pace has been good as of now, as of this call, and with the upcoming holidays, we don't know if it will continue or will there be a pause, and it varies depending on the patient and the urgency and the family situation. So I wasn't guiding to any slowdown at all. I was just saying we don't know. On the 407, look, it's tough for us to guide when without access to the TDN network, because it does take us, as Summa mentioned, it takes us a bit longer to find these patients outside the network, convince them, get them on drugs. So unfortunately, not able to, like with the other programs, make a good prediction on when we will date that. Summa, do you wanna comment on, let me say one thing on the expression, I'll turn it over to Summa, our view on 4DMT, which I think you were asking, look, you have to remember it's a micro CFTR, right? It's a micro, it's not the full gene, it's a micro gene. And so I would disagree with the comment. I think one should expect that protein expression should vibrate to functionality. In our opinion, but Summa, do you wanna add something?
spk09: I think you've covered it pretty much on the dot, Krish. Agree, I mean, again, if you look at the histochemistry, it's not very clear where you see the expression, because it's all over, there's a lot of questions. We also spoke with some of the experts on that expression data, and they had similar concerns. So again, we don't know what the gene that Krish had is, if they're seeing this kind of expression level, is it the full CFTR protein, and how does that correlate to function? It's a question that still not answered. With regards to TDN, we are working very diligently. We have done beyond what other companies have done with regards to showing function, and we're pretty confident we'll get there. So we are close, we are looking at all different studies including NTD animal studies, we are looking, we know we can express the full length protein by rest in block, we see it, we're very confident, we just need to get the assay for function optimized. So we feel pretty good, I think. Hopefully we will get there sooner, and we can get that study more fully aggressively enrolled.
spk01: Great, thank you very much for taking the questions.
spk05: Thank you. The next question comes from a line of Tim at Lugo with William Blair, your line is now open.
spk03: Thanks for the question. Going back to VyjuVac, can you talk about the progress you've made identifying patients outside of the initial, I think 1200 or 1100 patients that you had identified during the summer? And I guess on top of that, penetrating 30% into that as of now, I think is what you mentioned. Can you talk about, I guess, really the need for a CCO if you're going to get 50% penetrated into the population within the next couple of quarters?
spk07: Tim, I didn't follow the second part of the question. What were you saying in terms of penetration? Oh,
spk03: the second, yeah, the second part is, I believe you mentioned you're about 30% penetrated, and it looks like- I said 20
spk07: % of the idea. You're trying to be
spk03: able to get to,
spk07: oh, 20%,
spk03: okay. All right, well, just an update on just the 1200 patients or 1100 patients that you've identified so far.
spk07: Yeah, yeah, what I did to get to 20%, just to be clear, we got 284 star forms, you multiply that by 85% conversion rate, divide by 1200, you're close to 21%. That's how we estimate the 20% penetration, it could be a bit higher. If the 85% is higher, then we estimate at the moment. With respect to finding patients, it's to date, after one full quarter into launch, it's been more opportunistic than deliberate, meaning we're still working off the tier one, tier twos that we identified going after, trying to get them converted. Our objective is to get more serious about finding patients past 1200 early next year as we start to drain out the base, the reservoir of identified patients.
spk03: And with that, I guess, can you update us on your thoughts around a new Chief Commercial Officer? I feel like previously you mentioned that that would be the focus of a new CCO.
spk07: Yeah, so we are in the process of looking honestly. We've gotten a few candidates at different levels in the queue at the moment, we hope to have one in place early in 2024. That's the objective, what's important to us is we find someone with a good fit in the way we operate. And thankfully, there's been a lot of interest from potential candidates so far.
spk03: Fantastic, thank you for the question.
spk05: Thank you. The next question comes from a line of Gavin Gartner with Evacor ISI, your line is now open.
spk10: Good morning, congrats on the progress with the launch. So you noted that Gartner is getting to two to three weeks for the conversion cycle from PSS to paid revenue. Where did the numbers start from launch and how long do you think it'll take you to get to that two to three weeks?
spk07: Yeah, we're hoping to get to two to three weeks in 2024, hopefully in the first half, first quarter. Right now, it's pretty, I mean, it was long and it continues to come down. When we started, it was more like six, seven weeks. And a lot of it had to do with submitting for reimbursement, getting denied, resubmitting, single case forms. The reimbursement was not smooth. So that was a big aspect. And that has gotten steadily better over time. So that's one of the reasons we feel good about getting to two to three weeks. We find that families take about two to three weeks to schedule a nurse visit. There's some time in figuring out the right nurse that they're comfortable with, finding a time that works for them. That I don't think is compressible. So our goal is two to three weeks in 2024. And so every month that's gone by, we've been steadily coming down the curve of conversion and we're at a good point at the moment. And we hope to get to two to three weeks early in 2024.
spk10: Okay, that makes sense. And you noted off 45% of the PSFs are coming through for commercial patients. Are you able to disclose the commercial versus Medicaid split of patients that are on paid drug?
spk08: That are on paid drug.
spk07: Actually, I would say that for some of the patients the percentage of commercial versus Medicaid, if I were to estimate right now, is 51% commercial over 35% Medicaid.
spk00: Okay,
spk07: that's helpful. Okay, go ahead. Yeah, and starting October, we expect a lot of the mandatory states to start covering and so we expect the Medicaid number to go up a bit.
spk10: Yeah, that makes sense. Just a last quick clarification on the OLE patients. Have all of these patients converted over to drug, just confirming this was captured in the PSF number? And how much of this came through in the second quarter versus the third quarter?
spk07: Yes, they're all converted. I would say 60% in the second, 40% in the third.
spk10: All right, that's very helpful. Thanks so much and congrats again.
spk02: Thanks. So another question.
spk07: Is there any other further questions?
spk05: My apologies, Robert. Your line is now open with a follow-up question from Guggenheim Partners.
spk02: Thank you and Chris, thanks for taking our follow-up. On the comment you made about the OLE patients, expecting it to be the same or better now that reimbursement is largely in place. Does that pertain to patient start forms or conversion to reimbursement on therapy? Thank you.
spk08: I may have said
spk07: that comment in both contexts. Definitely conversion with access, I mean, that's a given, that's obvious. If we're not getting dinged and we're getting reimbursement we file, that process is smooth. Definitely the conversion should be faster and better. The comment I made on start forms was the base continues to be good at the moment. And I left it open to figure out what happens over the holidays which we have no visibility into at the moment.
spk02: Appreciate it. Once again, thank you.
spk05: Thank you. We now have another follow-up question from the line of Ritu Bauer with Kaplan. Your line is now open.
spk11: Hi guys, thanks for taking the follow-up. Chris, just going back to the rejected start forms, can you mention like what are the most frequent reasons for that, that you're not seeing? What are you encountering? Is it formal genotyping of these patients? Is it insurance coverage? Is it like purely administrative paperwork sort of stuff? What is that reason? And then I have another quick follow-up.
spk07: Mostly genotyping.
spk11: Got it. And then are you seeing any early trends in intent to implement buy-in bill from any of these centers? And are you seeing like clinics at centers of excellence sort of adding either clinic days or availability for appointments such that patients can come in more easily? Thank you.
spk08: Buy-in bill has been minimal.
spk07: I'm searching to remember if there's one. Actually, it's probably zero. What happens at these centers of excellence, like there is a, everybody, the KOLs, centers of excellence, the payers, everybody would like the patient to be dosed at home. And so the only thing dating in the case of a center of excellence is the first visit, I mean, first visit on VisuVac. I mean, so they wait for a patient to come visit them, examine the patient before putting them on VisuVac. But once they decide to put them on VisuVac, the actual process is not buy-in billed but sent back to the patient's home for home dose.
spk11: Got it. Thank you.
spk05: Thank you. That would conclude today's conference call. Thank you for your participation. You may now disconnect your lines.
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