Krystal Biotech Q4 2023 Earnings Call Transcript

Quartr
Provided by Quartr
February 26, 2024

There are 14 speakers on the call.

Operator

Thank you for standing by, and welcome to the Crystal Biotech Fourth Quarter and Full Year 2023 Earnings Conference Call. At this time, all participants are in a listen only mode. After the speakers' presentations, there will be a question and answer session. As a reminder, today's conference is being recorded. I would now like to hand the conference over to your host, Meg Dodge, Head of Investor Relations and Corporate Communications.

Operator

Please begin.

Speaker 1

Good morning, and thank you all for joining today's call. Earlier today, we released our financial results for the Q4 and full year of 2023. The press release is available on our Web site at www.crystalbio.com. Our earnings 8 ks was filed earlier today, and additionally, we filed our 10 ks with the SEC. Joining me will be Krishnan, Chairman and Chief Executive Officer Suma Krishnan, President of Research and Development and Kate Romano, Chief Accounting Officer.

Speaker 1

I'd like to note during this webcast, we will be making a number of forward looking statements about our future business plan, strategies, financial performances and projections, product candidate development plans, including statements about Vyjuvek. These forward looking statements involve risks and uncertainties, any of which are beyond Cristal's control. Actual results could materially differ from these forward looking statements as any and such risk can materially adversely affect the business, results of operations and trading price of Cristal's common stock. For a detailed description of applicable risks and uncertainties, we encourage you to review 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.

Speaker 1

With that, let me now turn the call over to Krish.

Speaker 2

Meg, thank you. Good morning and thanks for joining the Cristal's earnings call. 2023 was a spectacular year for Cristal and an inflection point for our company. With the approval and launch of Ijuvac for the treatment of dystrophic epidermolysis bullosa or DEB, we've now brought to market the 1st and only treatment that addresses the genetic cause of this debilitating disease. It has been immensely rewarding to hear from patients, families and the entire DEP community on the positive impact Wijeweck has had on their lives.

Speaker 2

It's also exciting to have secured this first approval for an entirely new treatment paradigm. We believe that a redosable topical off the shelf gene therapy that can be dosed at home by HCP holds significant potential not only for dead patients, but for many others suffering from other rare and serious diseases. We've only just begun tapping into the power of the HSV-one based gene therapy platform. As I will share in a moment, our U. S.

Speaker 2

Launch of IVYZUVAC is in a great place, tracking closely to some of the most successful rare disease launches from recent years with over $50,000,000 in net product revenue from VIZUVEC in the last 6 months since approval. We've seen rapid uptake across the U. S. Patient compliance is high. Access is almost behind us.

Speaker 2

And all these are starting to reflect in our net revenue. We're also making significant progress globally and look forward to growing the number of patients benefiting from VYZUVAC in the days, months and years to come. Our success in 2023 allowed us to deliver another year of significant value creation for our shareholders and perhaps more importantly, set us down a path for continued growth in the years to come. Our vision for Cristal has always been to build a fully integrated commercially established biotechnology company, developing and commercializing a portfolio of high value genetic medicines and generating long term shareholder returns. Thanks to a breakthrough year in 2023 and growing Visovac revenue, we are positioned better than ever to deliver on that vision.

Speaker 2

We expect to have at least 5 clinical trials this year. We have alignment with the FDA on a development path for VVAC, formulated as eye drops to help treat eye lesions in deaf patients and could potentially start another clinical trial in the second half of twenty twenty four. With strong financial resources and FDA approved redosable gene therapy platform and 2 commercial scale GMP manufacturing facilities were uniquely positioned to develop and bring to market a highly differentiated gene therapies for many rare and serious diseases. We look forward to making continued progress in 2024. As you have no doubt seen by now, our commercial launch is proceeding exceptionally well.

Speaker 2

We reported net Visorix revenue of $42,100,000 for the Q4 $50,100,000 for 2023. Gross margins were 93% for the quarter, in line with our previous guidance of margins north of 90% at launch and expected to improve to over 95% after a few years. Gross to net adjustments in the Q4 were 14%. Our long term guidance on GTN is unchanged and we expect it will settle into the mid to high teens range, reflecting a roughly even split of debt patients on commercial and government plants. With respect to future revenue, I'd like to highlight that earlier this year, a permanent J code was assigned to IZUVAC.

Speaker 2

The J code is an important milestone for finalizing coverage by Medicaid as it is required for many mandatory Medicaid states. Although this is a long term tailwind and sets us up for sustained broad access in the U. S, I will note that the revenue in the month of January was temporarily impacted as commercial and Medicaid coverage plans, including those that were previously covering VYZUVAC under a temporary J code were updated to the now issued permanent J code. You can also expect to see an impact this year as we accrue for patients on contracted commercial plans, potentially hitting the price cap of 900,000 dollars gross per patient per calendar year in 2024. Before sharing details on the U.

Speaker 2

S. Launch, I'd like to highlight a revision to our outlook on the total market opportunity that exists for VIZURVAC. Now that we have the benefit of 6 months launch data in the U. S. And a clear line of sight on registrations and patient numbers in Europe and Japan and alignment with the FDA on the development plan to get BVec formulated as eye drops to treat eye lesions in DEP patients, we're confident in the total global market opportunity for VIZUVAC to get over $1,000,000,000 Based on the trajectory of our launch and the feedback we're receiving from the field, our conviction in the total U.

Speaker 2

S. Market opportunity for BiZUVAC is only growing. We have spoken before about a total debt population of 3,000 in the U. S, of which 1200 are identified. That 1200 figure initially based on independent third party claims datasets from multiple sources has been further strengthened by our latest data from the field, which includes interactions with over 1500 unique HCPs as well as real time claims alerts that they use to optimize field developments.

Speaker 2

We expect to close the gap between 1203,000 in the years to come. We're already finding new patients, patients not part of the IDentify pool in the 1st few quarters of launch and we expect this to continue if not accelerate as we increase our focus on patient finding in later quarters. Once we reach a meaningful threshold of 1500 patients, we'll update our identified patient population number. Also, Christine Wilson has now joined Cristal as the Head of U. S.

Speaker 2

Sales and Marketing reporting to me. Christine brings decades of commercial experience to Cristal, including a strong record of leading U. S. Rare disease sales teams at Treveor, Reata and Shire. Most importantly, Christine has direct experience finding and activating rare disease patients in the community and will play a critical role for us as we continue to penetrate in and grow our identified patient pool in the months years to come.

Speaker 2

Outside of the United States, we benefit from patient registries, which give us a high degree of confidence in the global market opportunity. In France, Germany, Italy, Spain and the U. S. Alone, we believe there are over 2,200 DEP patients that could benefit from BIJUVAC and there are 100 more in Japan. We look forward to seeing continued growth in 2024, not only in the U.

Speaker 2

S, but also globally as we make progress towards EU and Japanese approvals. As Sumo will share, we're on track to launch in both regions in 2025. Now I'd like to share a few highlights on the U. S. Launch.

Speaker 2

With respect to patient start forms, we're pleased to report that we have now received start forms representing 35% of the initial 1200 identified patient pool. We're happy to see that we're both expanding and deepening the prescriber base as we progress in our launch. We now have over 195 unique prescribers of Vizovac, up from the 136 unique prescribers we reported in the 3rd quarter. We're encouraged to see the broadening of VisoVec prescriber base as awareness of our product grows through both our direct promotion efforts and organically through patient groups and the medical community. We're also seeing increasing depth of prescribing as the number of physicians that had written 2 or more prescriptions for death patients increased from 37 in the Q3 to 60 as of mid February.

Speaker 2

We believe this increase is a reflection of the growing familiarity among prescribers with VYZUVAC and we expect this positive trend to continue as patient outcomes on VYZUVAC become more widely known. With respect to the split of PFS between centers of excellence in the community, trends are in line with what we saw earlier in the Q3. 47% of patient start forms received as of mid February were from centers of excellence and the balance of the scripts were written by community physicians. We continue to see a steady flow of start forms from centers of excellence at a cadence correlated to EB clinic days. The split between DDEB and rDDEB start forms is also largely in line with what we saw in the Q3 with a slight uptick in the proportion of prescriptions received from DDEB patients.

Speaker 2

Importantly, we are starting to see significant conversion of patients converted to paid drug with 228 patient reimbursement approvals secured to date. Most approvals are for 6 months or more, so a few have been up for reauthorization so far, but all have been either approved or still in process. In line with our high conversion rates, Access is in a great place. We have received positive coverage policies or decisions for 97% of lives covered by commercial plans and 88% of lives covered by Medicaid. With the permanent J code now assigned, the trajectory for access and reimbursement approvals is only looking better.

Speaker 2

At home administration demand continues to be high, now 2 full quarters into launch with 98% of the drug starts occurring in a home setting. And accordingly, we also continue to see strong patient compliance to once weekly treatment while on drug holding at 96% in the 4th quarter. We believe that compliance is both a reflection of the significant clinical benefits that can be realized with the VYZUVEC therapy as well as our commitment to the patient experience and ensuring a smooth process to getting on drug. At this point in our launch, I think it's also worthwhile to reiterate and update on some of the key dynamics that shape the trajectory of the VYJAVAK update. Although there is a temptation to think that for a severe rare disease such as DEB, all patients would get prescriptions the day after approval, there are practical considerations that need to be understood.

Speaker 2

First, it's important to recall that COE visits occur at a steady pace around EB clinics, with only a subset of that EB patients seen at each visit due to the time spent on each patient and the multi specialty care they receive in the COE setting. In addition, we are also seeing some KOLs less familiar with VYZUVEC taking a stepwise approach starting with the more severe RDEB patients and state gating, particularly in the early stages of access where reimbursement was still being set up. 2nd, it's important to understand that deaf patients are not all clustered around centers of excellence. Prior to 2023, only palliative treatment options were available, whether offered by a COE or a community physician. As a result, there was often little reason for a patient to visit, let alone return to a COE unless it was conveniently located.

Speaker 2

Certainly, there are some patients that are actively managed at COEs, but an equal number or more exists in the community and many patients have relied on community physicians for many years prior to 2023. This also has implications for the launch. It means that we have to make local doctors aware of BIJUVEC, educated on DEB, gene therapy and sometimes both. Recall as well that prior to BIJUVAX approval, care of EB wounds did not require genetic confirmation. So even establishing this practice is an evolution in patient management.

Speaker 2

This is all addressable through physician education programs, but it does take a certain amount of time and contributes to a more gradual ramp in patient starts. Finally, there are logistical aspects of integrating at home dosing of Bijovex into existing wound care and bandaging routines. As the 1st and only FDA approved re dosable gene therapy, providing access to and coordinating weekly home treatment of Bijuvaq is not as simple as dispensing pills at a local pharmacy for patient pickup or even one and done gene therapies administered at a large specialty center. Once the initial scheduling and setup is complete, which again takes a certain amount of time, what we're seeing is strong adherence given that BIJOVIC is designed to be integrated into existing wound care routines and demonstrated efficacy. Together, these real world dynamics influence the shape of our launch curve, which we expect to be characterized by a steady stream of prescriptions and patient conversions to drug.

Speaker 2

Recall that our own estimates for the United States are informed by multiple third party estimates and our own real time and boots on the ground insights and further supported by the prevalence in Europe by robust patient registries and genetic testing indicate their prevalence rates well in excess of our U. S. Identified patient pool. So trying to correlate the shape of the launch curve to prevalence is simply erroneous. Our conviction in the total opportunity and our ability to capture it have only been strengthened after these first few quarters of launch.

Speaker 2

Before closing on the U. S. Launch, I'd like to underscore that thanks to the efforts of our team and our focus on the patient experience with changing the treatment paradigm for dDeP patients. Years ago, we set out to build the necessary infrastructure to support a strong commercial launch, including our patient support program, Crystal Connect, our no cost genetic testing program, decode DEB and a network of local pharmacies to prepare and deliver VYZUREK to a patient's home along with the trained nurse to apply the treatment each week. As I just shared, this at home administration infrastructure has been in high demand since launch, supporting strong compliance to the therapy.

Speaker 2

As I also reported, we're broadening and deepening the prescriber base. All KOLs within centers of excellence across the U. S. Are now prescribed by JoVac. And outside of the centers of excellence, our field team has had tremendous success educating regional, adult and pediatric dermatologists and community physicians as evidenced by the continued influx of community prescriptions.

Speaker 2

Sales cycles tend to be a bit longer in the community, but for 2024, with more evidence of a dispersed patient community, we expect to continue to generate demand from large EB centers, regional derm specialists and community physicians. And finally, our marketing team has invested and will begin deploying several social media campaigns on different channels to generate awareness to patients and families directly who we know similar to other rare diseases, having varying levels of engagement with the healthcare system as they've learned to self manage their DEP wounds, but deserve to learn about VYZUVAC and the benefit it can provide to them. Combined with our partnership with advocacy groups, we're committed to reaching all patients and families to share the powerful wound healing that VIZOVAC can provide them. Altogether, these successes point to a strong long term growth strategy for VYZUVEC in the U. S.

Speaker 2

And in the interim, it's important to highlight that by any metric, the launch of VYJAVEK has been among the top tier of rare disease launches in recent years. Despite all the nuances that I outlined, in only 2 full quarters since approval, we recorded $50,700,000 in revenue. And now as of mid February, we've secured reimbursement approvals for 228 patients and have positive coverage policies in place for over 93% of commercial and Medicaid patients. We are excited about our success to date and more excited so by the years to come as we work to change the treatment paradigm for every patient with DEB. Finally, before I turn the call over to Suma, I'd like to touch on the power of our platform and our milestone rich clinical pipeline.

Speaker 2

The Crystal story is more than just device effect. HSV-one is unlike any other vector system currently in development with a capacity for large genetic cargo, broad tissue tropism, redosability and no integration risk. HSV-one is also amenable to formulation to target multiple tissues such as the skin, the lung and the eye, providing us with a large number of opportunities to develop 1st and best in class genetic medicines for rare and serious diseases. Our team is hard at work leveraging these unique platform attributes and advancing our programs to and through the clinic. These include a new eye drop formulation of BVEC to treat ocular complications of DEB, which has already been evaluated in single patient under compassionate use, 2 clinical stage lung programs targeting cystic fibrosis and alpha-one antitrypsin deficiency, an emerging oncology program that leverages our experience in skin and lung drug delivery as well as clinical stage candidates for rare skin disease and aesthetic skin conditions.

Speaker 2

With 4 active clinical programs and more to come later this year, we look forward to the data readouts, which will validate the breadth of our HSV-one based gene delivery platform. I shall now turn the call over to Suma to provide a clinical and a regulatory update and provide more color on the clinical programs.

Speaker 3

Thank you, Krish. 2023 was a milestone year for Cristal and our development team securing the first ever FDA approval for a redosable gene therapy. It has been immensely gratifying to see a growing number of U. S. Patients benefit from VYZUVAC as our commercial launch progresses.

Speaker 3

However, our work is not yet done. Our goal has always been to enable global access for patients suffering from death. And I'm happy today to share we are on track for commercial launches in both Europe and Japan by 2025. For Europe, we announced in October that we filed for a marketing authorization with the European Medical Agency. Subsequently, in November, our filing was validated by the agency, putting us on track for a decision by the European authorities in the second half of twenty twenty four.

Speaker 3

In Japan, we recently announced in December that we had received orphan drug designation by Japan's Pharmaceuticals and Medical Device Agency, a designation which confers specific benefit for orphan drug development, including priority review of applications, extended registration validity and reduced development costs. With our open label bridging study in Japanese patients fully enrolled, we are on track for a Japanese new drug application filing in 2024 and a decision by Japanese authorities in 2025. We are also making important progress on our broader clinical pipeline

Speaker 4

where

Speaker 3

we have 4 programs in the clinic and more coming in the first half of this year. As said previously, we are committed to treating death comprehensively. Ocular complications are thought to affect over half of patients with recessive death. And to date, only supportive care and surgical interventions are available. Earlier this month, the New England Journal of Medicine published the clinical results for the first patient treated with topical BVAC to the eye.

Speaker 3

The improvement seen in this patient was dramatic, including full healing of the corneal epithelium and improvement of visual equity from hand motion to 2025 by 8 months. Given these outcomes, we started discussion with the FDA earlier this year regarding a potential development program to bring an ophthalmic DVAC formulation to market. And earlier this month, we reached alignment with the FDA on a single arm open label study to enable approval of BVAC eye drops for the treatment of lesions in the eye of dap patients. We are now planning to initiate this study in the second half of twenty twenty four. On KB-four zero seven, our redosable inhaled gene therapy for the treatment of cystic fibrosis, we completed Cohort 1 of the CORO-one study with no severe or serious adverse events and initiated dosing in Cohort 2.

Speaker 3

CORAL-one is a Phase 1 opal level dose escalation study in patients with cystic fibrosis. The primary objective of this study is to evaluate safety and tolerability of KB-four zero seven, but bronchoscopies are planned for Cohort 3 to allow evaluation of airway epithelial cell transduction and expression of CFTR transcript and protein. Cohort 3 also includes minimum enrollment thresholds for patients that are not eligible for modulators, an important patient population for which no effective disease modifying therapies exist. The pace of enrollment in CORAL-one is improving as clinicians grow more familiar with ENIEL KB-four zero seven. The potential for patients to roll over into multiple cohorts is also expected to help us progress more rapidly through Cohort 2 and 3.

Speaker 3

In addition, we continue to work closely with the therapeutic development network of cystic fibrosis foundation to provide us access to the broader site network, which should enable us to accelerate enrollment. On KB-four zero eight, our redosable inhale therapy for alpha-one antitrypsin deficiency, we just recently dosed our 1st patient in our SERPENTINE I study. SERPENTINE I is a Phase I open label single dose escalation study in adult patients with AATD to allow assessment of safety, tolerability and alpha-one antitrypsin levels and key pharmacodynamic biomarkers. With strong support from the alpha-one research community, we are optimistic that this study will enroll quickly in 2024. We also made several advancements in our oncology program, KB707, last quarter and earlier this year.

Speaker 3

Recall that KB707 is a modified HSC1 vector designed to deliver genes encoding both human IL-twelve and IL-two to the tumor microenvironment and promote systemic immune mediated tumor clearance. We have 2 formulations of KD-seven zero seven in development, a liquid formulation for inter tumor injection and an inhaled formulation for lung delivery. In October, the first patient was dosed in Phase 1 OPAL-one study to evaluate intratumoral KB707 monotherapy in patients with locally advanced on metastatic solid tumor malignancies. Additionally, an amendment to the existing KD-seven zero seven IND was cleared earlier this January, allowing us to evaluate inhaled KB707 in a clinical trial to treat tumors in patients' lungs. We will initiate the study, which we'll be calling KINITE-one in the first half of twenty twenty four.

Speaker 3

It will be an open label, multi center dose escalation and expansion study to evaluate inhaled KB707 monotherapy in patients with advanced solid tumor malignancies affecting the lung. And finally, we are looking forward to sharing a data update on our lead aesthetic program, KB301, later this year. KB301 is being evaluated in the PERL-one multi cohort Phase 1 study. We previously announced data from cohorts 12, which demonstrated that KB301 encoded with payload COL3A1 was expressed locally after intradermal injection and that injections to the upper and lower cheek and above the knees were generally well tolerated and associated with clinically meaningful and durable improvement in subject satisfaction. We are now running cohort 34 of PERL-one in parallel to evaluate KD-three zero one for 2 potential target indications: improvement of lateral canthal lines at rest and improvement of dynamic wrinkles of the declati.

Speaker 3

Both were identified as potential indications based on initial data from Cohort 1 and Cohort 2 and the high demand for effective aesthetic options in these regions. We expect to announce data in first half of twenty twenty four. We are also progressing on our broader pipeline in dermatology. We expect to commence the Phase 2 cohort of this KV105-two O2 JADE 1 trial for the treatment of CGM1 ARCI later this year, subject to alignment with FDA on study endpoints. Additional preclinical candidates are also in development, and we expect to share updates in the quarters to come.

Speaker 3

Our HSV platform has potential to yield a large number of highly differentiated redosable gene therapies. We look forward to making continued progress in 2024 and sharing data updates on our clinical pipeline later this year. With that, I would like to turn the call to Kate.

Speaker 4

Thank you, Suma. We concluded 2023 with $358,300,000 of cash on hand and $594,100,000 of cash and investments, which positions us well for our future growth, the expansion of VIJUVEC outside of the U. S. And our pipeline development plans. In our Q2 since the launch, we recorded $42,100,000 in net product revenues from VIZIVEC in the 4th quarter and $50,700,000 for the year ended 2023.

Speaker 4

As Vyseveq was approved by the FDA in May of 2023, there were no comparative period revenues. Cost of goods sold was $2,900,000 for the quarter or about 7% of net product revenues and $3,100,000 for the year or about 6% of net product revenues. Cost of goods sold increased on a sequential quarter basis as a portion of the initial costs associated with the manufacturing of VIJOVAC were expensed as research and development costs earlier in the year prior to approval. Research and development expenses for the quarter were $11,400,000 inclusive of stock based compensation of $2,400,000 compared to $10,700,000 for the prior year's quarter, inclusive of $2,400,000 of stock based compensation. Research and development expenses for the year were $46,400,000 inclusive of stock based compensation of $10,100,000 compared to $42,500,000 in the prior year, inclusive of $7,900,000 of stock based compensation.

Speaker 4

Higher research and development expenses in 2023 were due to increases in payroll and facility related costs, primarily driven by an increase in personnel to support our overall growth, especially as we ramp up our KB707 program, partially offset by decreases in R and D manufacturing costs as following our FDA approval, our costs related to the manufacturing of Vyjuvec are now recorded as part of our cost of inventory. Selling, general and administrative expenses for the quarter were $24,800,000 inclusive of stock based compensation of $7,500,000 compared to $24,000,000 for the prior year's quarter, inclusive of stock based compensation of 7,200,000 dollars Selling, general and administrative expenses for the year were $98,400,000 inclusive of stock based compensation of $29,900,000 compared to $77,700,000 in the prior year, inclusive of stock based compensation of $25,300,000 Higher selling, general and administrative expenses in 2023 were largely driven by an increase in personnel, increased selling expenses related to the launch of VIAVIQ, increased IT infrastructure and software costs as well as increases in travel, sponsorship and professional fees. These increases were partially offset by a decrease in commercial preparation expenses, a decrease in medical affairs costs and a decrease in business development costs.

Speaker 4

Our net income for the quarter was $8,700,000 which represented $0.31 per basic share and $0.30 per diluted share. Net income for the year ended 2023 was $10,900,000 which represented $0.40 per basic share and $0.39 per diluted share compared to a $140,000,000 net loss in 2022, which equated to a loss of $5.49 per share, both basic and diluted. We would also like to provide some perspective on our forecast for operating costs in 20 24 given the objectives we have for the year. We expect between $150,000,000 $175,000,000 in combined non GAAP R and D and SG and A costs this year. This projection excludes stock based compensation.

Speaker 4

This expected increase over 2023 is driven primarily by increased SG and A costs associated with our continued U. S. Launch and planned expansion for VizaVec outside of the U. S. From a research and development perspective, we expect a pickup in our clinical and regulatory costs associated with our oncology and respiratory programs as well as for costs associated with initiating a clinical trial for the BVAC formulation to treat lesions in the eye of DEB patients.

Speaker 4

With that being said, and given our projected revenue trajectory in 2024 and our current strong balance sheet, we are well capitalized and positioned for these growth drivers, and we look forward to reporting our progress over the next several quarters. And now I will turn the call back over to Krish.

Speaker 2

Before we open the call up to Q and A, I'd like to underscore our excitement for 2024 as we have successfully transitioned Cristal to a fully integrated commercial biotech with a deep clinical pipeline. With a great launch underway and growing revenue to contribute to our already strong balance sheet, we believe we are well positioned to create value for our shareholders and deliver our mission for patients now and in the future. Thanks for listening. And I'd like to now open the call for Q and A.

Operator

Thank you. At this time, we'll be conducting a question and answer session. First question today is coming from Alex Stranahan from Bank of America. Alex, your line is live.

Speaker 5

Hey, guys. Thanks for taking our questions and congrats on the strong close to 0.3. Two questions from us. I guess what has been roughly the average conversion time between new start forms and initiating therapy in 4Q? And where do you see this tracking over the next few quarters?

Speaker 5

Or is this really not the metric we should be looking at anymore? And second, could you maybe walk us through the anticipated J code impact to uptake looking at the next couple of quarters, especially in the Medicare segment? Thanks.

Speaker 2

Thanks, Alek. Hey, on your first question, at the end of Q3, we were tracking like at a 2 month pace between start forms to patient on drug. We were making progress and presently we're about 30 days from a start form to patient on drug. Our goal, at least by the second half of this year, is to shrink it down by a few weeks, maybe 2 to 3 weeks. A lot of it has to do with single case agreements and with access getting behind us, we expect the trend to only decrease and get to a steady state of 2 to 3 weeks in the second half of this year.

Speaker 2

With respect to the J Code, the reason I mentioned that comment, look, J Code is a great tailwind for the company long term. I just was simply pointing out that in January, as our specialty pharmacy was transitioning people from into the official J code from a prior J code, you should expect a few weeks of a revenue hit to the story in the interim. But outside of that, we don't particularly see the takeout having any impact going forward, especially impact in the positive direction, not having any kind of impact in the other direction.

Speaker 5

Thank you.

Operator

Thank you. The next question is coming from Yigal Nochomovitz from Citi. Yigal, your line is live.

Speaker 6

Hi, Christian, team. Thank you so much for taking the question. Could you just help us clarify on the numbers? The 228 reimbursement approvals and the 35% of the 1200, the 420, is the 228 a subset of the 420? Are they distinct populations?

Speaker 6

Just to clarify, please.

Speaker 2

No, no. I mean, let me answer it this way. The 1, the 35%, the 4 20, that's clearly the start forms that we've been mentioning in the past. The 228 talks about out of the start forms we've gotten, the exact number of reimbursement we have gotten from we like that metric because it's a much closer indicator of net revenue in the long term. And so yes, obviously, it is a subset of all the start fronts we've gotten sort of answer that very directly.

Speaker 2

But it's a different metric in the sense that it's a more important metric as revenue becomes a much more important part of the story going forward.

Speaker 6

Okay, thanks. And then you referenced that I think you said 60 of the 195 were prescribing 2 or more. I was just trying to do a little math on that. It suggests that for those higher prescribers, it's somewhere around 5 patients per prescriber. Is that the correct thinking or is it

Speaker 2

a different? Thanks. I think I mean it's a difficult question to answer immediately, but I think you're right. I think you're right. Like all we were trying to show is that It shows they are expanding to a broader base of patients.

Speaker 2

There were a couple of COEs, I've mentioned this before, who were positioning that let me get my ARTIF patients on drug before I get my dominant patients on drug and see how they're doing. All in all, the reason for that metric is to show increasing confidence of a prescriber towards continuing to prescribe BIJUVA.

Speaker 6

Okay. And then just one quick one on the eye drop, certainly very interesting early data. Can you just discuss the strategy there longer term? Is that going to be provided as an adjunct to the topical for those that have the eye complications? Or would it be an additionally priced drug separate from the cream?

Speaker 2

I think it's a bit too early to be very definitive on the matter, but our objective is to have a separately priced product like we do right now on that single patient. We ship that patient the next bio. And so our thinking is to have a separate NDC number, a separate pricing and send it along with the topical gel.

Speaker 6

Okay. Thank you very much.

Operator

Thank you. The next question is coming from Debbie.

Speaker 3

Yes. I just want to clarify, sorry, on the eye. As I said, we have pre IND guidance from the agency. So this is going to be a new IND, and it's going to be a BLA. So a brand new label with the new NDC code, as Krish mentioned.

Speaker 3

Just want to clarify.

Speaker 6

Thank you.

Operator

Thank you. The next question is coming from Debjit Chattopadhyay from Guggenheim. Debjit, your line is live.

Speaker 7

Hey, good morning team. This is Robert on for Debjit. Can you provide any color on the number of dominant patients on therapy as of December 31 Feb 24? And also the number of recessive patients on therapy as of December 31? Then I have a follow-up on the ocular complications.

Speaker 2

No. I think for now and I don't want to dig through to figure the split out. I think for now, as we mentioned in the call, 25% of the total storefronts were the dominant type. We did not, just to be clear, see any big change in the trend of the dominant versus successive at the moment.

Speaker 7

Okay. Thank you. And then on ocular, do you currently have 10 or more patients with complications identified who could quickly be enrolled? And how long is follow-up planned for the study?

Speaker 3

I can answer this question. Yes. In fact, we have a lot of patients and the reason we are accelerating is because there is request for even off label. So we want to avoid that. Yes, we do have enough patients who have been who have a good natural history of their disease.

Speaker 3

And as you can see, it's a single arm open label study. So it should be pretty straightforward.

Speaker 2

Great. Thank you.

Operator

Thank you. The next question is coming from Dae Gon from Stifel. Dae Gon, your line is live.

Speaker 8

Great. Thanks for taking our questions and let me add my congrats on the quarter as well. Krish, just going back to the roughly 136 start forms that you reported as of February. I was just kind of curious if you could talk about the cadence of patients. I know it's kind of a combination of Q4 and a little bit of 2024.

Speaker 8

And you've added a lot of conviction around sort of the year end, I mean, the opportunity this year. So if you could kind of comment on that cadence. And then going back to the more than $1,000,000,000 opportunity you were talking about, I guess a little early, but if you can maybe frame for us what exactly are you thinking, because in prior, I guess, VITRAVEC approval, you've given some conservative estimates that you had to revise. So what should we be thinking in terms of BVAC ophthalmic solution there? Thanks so much.

Speaker 2

Great. Hey, on the cadence of patients, just to correct you, I think 35% of 1200, I want to be clear that's the number of star forms. I heard a number 136 that I wasn't sure about. But that said, look, definitely the holiday period were a bit slow. Slow as in appointments getting pushed out by a week or 2, EB clinics being rescheduled.

Speaker 2

And that was a singular cadence that we observed, which is why we kind of pushed out the start from reporting to as of February. So that's the cadence on that. On the opportunity, look, there was some confusion when we first reported. When we first reported the market opportunity, all we had was an approval in the U. S.

Speaker 2

And it felt a bit conservative. The feedback I got was super conservative. Now we're starting to see feedback from the MA. We're starting to see feedback from the Japanese authorities. Our conviction in the drug getting approved in Europe and Japan is definitely a lot higher than it was 6 months ago.

Speaker 2

Then we're also starting to align with the agency on the ophthalmic indication in the eye. And now that it's a single label study and as Suma mentioned, there are people with natural history, we feel at the moment pretty good about 50% of the RDEB patients or 25% of the total DTEB patients, however you want to look at it, also adding to the market opportunity. And so we thought we'd take the moment to alleviate any confusion from prior and be very definitive on the market opportunity of BBAC going forward.

Speaker 8

I guess just to clarify on that point, are you able to give some percentage breakdown of how much would that be from BVAC as it currently is as VIGAVAC or BVAC formulation?

Speaker 2

You mean the eye formulation? Look, without the eye formulation, we believe it's north of $1,000,000,000 The eye formulation, and this is a very premature estimate from my end, probably adds another $250,000,000 to $300,000,000 to the opportunity itself.

Speaker 8

Excellent. Thank you very much.

Operator

Thank you. The next question is coming from Joe Pantginis from H. C. Wainwright. Joe, your line is live.

Speaker 9

Hi, everybody. Good morning. Thanks for taking the question. Of course, congrats as well, but also thank you, Krish, because of the real clarity and visibility that you're providing around the launch. So we all appreciate that.

Speaker 9

First, I guess when you come to the actual patients, do you have any data that you can share with us about the ranges of the treatment area sizes, number 1. And then number 2, I know there's great compliance so far as you've alluded to. Anything you could share with us as to the reasons for patient stops? Thanks a lot.

Speaker 2

It's a great question, Joe. In terms of ranges and area sizes, let me say, look, as the patient population grows and as the visits are weekly, we're not particularly tracking every single patient and every single one. But that said, through feedback, through Crystal Connect, we do know that there are a good number of patients treating large wound sizes, good number of patients choosing to start with the small ones moving into the large ones. We see no difference in the way the drug works between recurrent and chronic. And all in all, we believe that VYZUV has been great to treat wounds of all sizes and wounds of all cadence as in recurring versus chronic, etcetera.

Speaker 2

The only point that is that we are working on, not immediately, but in the medium term or so, is to see if we can increase the dosage per vial per week. That would be beneficial, but that's not an immediate solution. People seem very happy with the drug. People seem very happy with the home dosing. I will say one of the best things we got on the label was home dosing.

Speaker 2

Compliance is high because of this. We see patients unwilling to even go to a local physician to get it done on a weekly basis. And so home dosing has really helped a lot with the launch. In terms of compliance, look, when we look back at our clinical studies, I mean like just before the launch or the 1st few months of launch, compliance was very high. We expect compliance to continue to be high in 2024.

Speaker 2

But as wounds start to heal, one has to think about maybe a handful of patients slowing down, either taking a pause and restarting or going from 4 to maybe 3 to 2 vials a month, but it's not immediate. I think our original assumption of having a patient on vials for 12 months or longer as of today continues to track properly.

Speaker 9

Great. Thank you. Really appreciate it.

Operator

Thank you. The next question is coming from Andrea Tan from Goldman Sachs.

Speaker 10

Chris, just could you speak a little bit more about the evolution of the competitive landscape as you see it? And how you envision the treatment paradigm shifting if Abeona's graft is approved?

Speaker 2

Great. Hey. On the competitive landscape, Cristal was started in 2016 when we realized that the competitive landscape was not a very commercially viable approach to treating DEB. I mean, we obviously appreciate anyone and all working in this debilitating disease. But our view right now and this could be different since BiJoVac has the ability to treat larger wounds, we doubt if patients would go through the inconvenience of having to graft over and over and again in the same location and takes a second bit of time.

Speaker 2

And if you look at the convenience aspect, it gets much Vazurex obviously is much more convenient and amenable. So we're not presently watching or too anxious about an autologous approach to treating the disease at the moment. We feel that the convenience aspect of IZURAC is much more that is much more convenient. Now on the other competitive environment, which is does not fundamentally address the cause of the disease, we're less concerned about that because at the end of the day, we believe that most patients and most physicians want an approach that fundamentally treats the nature of the disease.

Speaker 3

And I'm just going to add another point to Abi on our therapy. It's keep in mind, it's 50% wound closure. I mean, we had a high bar of 100% wound closure. So we are seeing, even with large wounds, pretty good closure. So I think again, as Krish mentioned, easy to apply topical.

Speaker 3

We don't have to biopsy these patients. These patients cannot stand any sort of pain or inflict on hospitalization. They can't afford that. So I think from that perspective, because of our ease of application and good efficacy, again, we are not concerned.

Speaker 10

Great. And then, Timna, maybe one question here on the pipeline as you think about the upcoming data reads that you have across the different programs. Just wondering if you could help frame expectations into each one of those. And what would trigger either a go or no go decision to bring those forward? Thanks so much.

Speaker 3

I mean, again, 407, we are excited. We finished Cohort 1. Cohort 2 is I mean, the acceleration of enrolling those patients is ongoing right now. So we are excited to move that. And quickly into Cohort 3, we think we really believe we have already sites that are established who can do bronchoscopy, and we have now patients identified.

Speaker 3

So we believe in 407, we should, by end of the year, be able to dose those patients and have some positive bronchoscopy data. But also, we are working on some preclinical models, which we feel we have some promise in getting confidence to get to establish a functionality for TDN to collaborate with us. So we feel pretty positive on 407. 408, again, as we mentioned, we dosed our first patient. This patient was not on augmentation therapy.

Speaker 3

So again, with the biomarkers that we can we are measuring because we're going to see blood levels of A180. And again, it's easy to measure both neutrophil elastase, binding of neutrophil elastase in sputum and see the plasma levels. We feel that these biomarkers will help us identify or get a faster read on 408. And we are very excited actually about 408. I think with the division, the OTP division open to biomarker approach for approval, we think with 408, we hold a good chance of getting in front of them once we have positive data to discuss biomarker approach for approval.

Speaker 3

So again, 408 is an exciting program for us. 707 is also exciting. I mean, we have, as Krish mentioned, we haven't provided too many specifics, but the program with the inter tumoral injection is moving forward. So hopefully, we'll have some outcome and data read end of the year. And of course, our lung program also, again, we are excited.

Speaker 3

So hopefully, end of the year, it could be exciting data read on all our programs.

Operator

Thank you. The next question is from Tim Lugo from William Blair.

Speaker 11

Congratulations on the quarter and also all the clarity. Can you give us a sense of product revenue ramping through 2024? It sounded like there was some seasonality around scheduling in Q4 and we're also 2 thirds of the way through Q1. Do you have a sense of it's going to be kind of incremental increases at least in the near term or if there might be some lumpiness quarter to quarter?

Speaker 2

It's Tim. We're not providing any guidance to it with respect to revenue. Understood. Yes, a nice start. Look, there's not much of a seasonality.

Speaker 2

I was just pointing out that Jayco transition is a one time event. It's not a seasonal event. And with that, it's a powerful tailwind behind us. The second, as I mentioned, I mentioned about a handful of patients hitting the cap at the end of the year. And what we're trying to do is to kind of accrue for it as we move through the year to avoid any kind of lumpiness in Q4 one way or the other.

Speaker 2

So that said, I think as more and more patients come on drug, you can start figuring out what the revenue potential of IGORA is going to be this year. But no comment from us.

Speaker 11

Understood. Before maybe a question on your platform, you obviously have some unique capabilities with the platform and re dosing. Are there any pipeline opportunities? You've built out a number of pipeline assets. But are there any that you looked at that might have just been too expensive to run the clinicals on that you think a partner would be appropriate for?

Speaker 11

I'm getting a sense of just how much capacity there is within the manufacturing and kind of development side.

Speaker 2

Yes. Look, our position on this is stayed the same for large indications. I'll talk about alpha-one antitrypsin, large indication. Some areas in oncology, large indication. Anytime in CF, we're primarily going after the null mutation.

Speaker 2

We view it much more as a rare disease than as a really large indication. I think we are always open to partnering on large indications. The company wants to stay focused on its ability to develop, manufacture and commercialize rare diseases and definitely would like to seek partner on larger indications. But we have enough capacity manufacturing wise to support any partnership, small or large, with the product either in the development stage or the commercial stage.

Speaker 11

Understood. Thank you and congratulations.

Speaker 6

Thanks.

Operator

Thank you. The next question is coming from Gavin Clark Gardner from Evercore ISI. Gavin, your line is live.

Speaker 12

Hey, thanks for taking the questions. Just had a couple of clarifications and then questions. On the 228 reimbursement approvals, I wanted to be clear, was this also through mid February or was that at the end of December?

Speaker 2

It said on the deck as of February, if I'm not mistaken, and we hold to that. Like let's not we're not trying not to get too cute between mid versus early versus late, but it was as of February.

Speaker 12

Okay. That's helpful. And just trying to quantify the holiday impact a little bit. Were the start forms coming in, in January, February, were those at a higher rate than later November or December?

Speaker 2

Yes. It was a shift, right? And so that kind of affected the rate, shifting from, say, middle of December on to January, etcetera.

Speaker 12

Okay. And have there been any differences in the conversion rate or

Speaker 7

the conversion

Speaker 12

time between the Medicaid and commercial patients?

Speaker 2

That's a good question because it doesn't have a time dimension. Now in the past, in like your 2023, Medicaid definitely took a bit longer because there was no J code, different states were at different points And commercial was doing pretty well. And I think right now, I wouldn't say starting now going forward, I don't see a measurable difference between the 2. We do depend and in fact, on the commercial side, if you end up with a single case agreement on a patient, it could take just as longer as Medicaid. So I mean to answer your question simply, and I was thinking as I was responding, we don't see any meaningful difference in time, especially going forward.

Speaker 2

And we might have seen some in the past.

Speaker 12

Okay, great. And last one, I just wanted to ask what your latest expectations for Vyjuvek pricing in Europe was? Thanks for taking all the questions.

Speaker 2

We don't know. We look, we are it's a differentiated drug. The efficacy profile is very strong. We're seeing really good demand on named patient sales from a handful of countries. Our conversations with EMA are going well.

Speaker 2

Our job is to make a case for we know it's going to be lower, but our job is to get to less than a 50% discount if we're fortunate. Less as in like I hope I'm not confusing people there. Something north of, say, $15,000 per bio would be great for us.

Speaker 12

That makes sense. Thank you.

Operator

Thank you. The next question is coming from Josh Schimmer from Cantor. Josh, your line is live.

Speaker 13

Great. Thanks for taking the questions. I have 3 quick ones. So 228 reimbursement approvals out of around 428 start forms, that's 55 percent conversion. For the remaining 45%, how many of those or what proportion of those do you expect to be able to capture with the new J code and over what period of time?

Speaker 13

Second question is if you can quantify any contribution from either inventory building or name patient access in Europe? And then last question, the annual cap of $900,000 gross, is that on a per patient basis or a per plan basis averaged across their population? Thank you.

Speaker 2

Look, on the first one, our internal goal is to try and get to all you talked about the gap between the start forms and the thing. We're trying to get to get all the patients in 2023 on to reimbursement by the end of Q1. That's the goal. And then so I don't instead of answering your question as 228 versus 428, our internal goal is all the patients who we know of at the end of 2023, we want them on reimbursement by the end of Q1. In terms of was there any inventory or NPP?

Speaker 2

Hardly any yet. We're just starting to get NPP. It will probably get reflected more going forward. And on the annual cap, look, it is the overall discount, I think I've mentioned this, is measured at the payer level. So it's kind of like a blended average depending on the number of patients in the plan.

Speaker 7

Thank you.

Operator

Thank you. And the next question is coming from Ritu Baral from TD Cowen. Ritu, your line is live.

Speaker 3

Hi, good morning guys. This is Anurag on for Rizu today. Congrats on the quarter. I have two questions. So as you see more reauthorizations taking place, could you just expand on how what's the process of that?

Speaker 3

And then what does the prescriber need to show for a successful reauthorization? And I have a follow-up after that.

Speaker 2

Yes. On the reauthorization happens every 6 months. Usually, you need some it's called clinical notes from a physician saying that the VYZUVEC is working. Of them certified without a patient visit and some certified wanting a patient visit and we're talking a local patient visit for the most part and that's what it entails. So depending on the patient, it could take a certain amount of time, but usually it's pretty quick.

Speaker 3

Got it. And then could you talk about the market research efforts in the EU ahead of your potential launch there and like in terms of how patients are currently managed there and now and just to confirm now you're expecting a launch there in next year versus later this year? Thank you.

Speaker 2

Look, later I want to be clear. It depends on when we get approved. And if you saw what happened to us in the U. S, we're from we're always adding 3 months to PDUFA date from this point on. It appears to be the norm than anything management says.

Speaker 2

Having said that, if the launch is scheduled for late in the year, like say in December, we probably push it out to January. In terms of launch plans, I think we're going to have a General Manager in Germany at the beginning of March to build out the team. That will be the 1st country of launch. In terms of market research, our current EU General Manager, Laurent, has already been talking to physicians and centers of excellence who have really strong registries. So we feel really good about the identified patient population.

Speaker 2

A lot of home dosing in Europe is handled by the hospital. So a lot of the logistical complications you see in the U. S. Like getting home dosing in place, finding patients are not as big issues in Europe. What is important in EU is that we get to a good place on access.

Speaker 2

If you get to a good place with respect to access, the EU launch becomes a bit simpler logistically than the U. S. Launch and kind of very similar in Japan story.

Speaker 3

Got it. Great, very helpful. Thank you.

Operator

Thank you. There were no other questions in queue at this time. This now concludes the call. At this time, thank you all participants for joining the Crystal Biotech 4th quarter and full year 2023 earnings call. You may now disconnect.

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Key Takeaways

  • Vyjuvek US launch: Generated $42.1 M in Q4 and $50.7 M for 2023 with 93% gross margins, over 195 prescribers, 35% of 1,200 identified DEB patients submitting start forms, 96% patient compliance, and 98% at-home dosing.
  • Access improvements: A permanent J-code was assigned to Vyjuvek, resulting in 97% coverage for commercial plans and 88% for Medicaid, and setting the stage for broader, sustained access in 2024.
  • Global expansion: Management projects a >$1 billion total market opportunity for Vyjuvek, with EU marketing approval expected in H2 2024 and launches in Europe and Japan targeted for 2025.
  • Robust pipeline: At least five clinical trials are planned in 2024, including an eye-drop formulation for ocular lesions in DEB, redosable inhaled gene therapies for cystic fibrosis (KB407) and alpha-1 antitrypsin deficiency (KB408), an oncology program (KB707), and an aesthetic skin candidate (KB301).
  • Solid financial position: Cash and investments totaled $594.1 M at year-end 2023, and combined R&D plus SG&A spending of $150–$175 M is forecast for 2024 to support the US launch and pipeline advancement.
AI Generated. May Contain Errors.
Earnings Conference Call
Krystal Biotech Q4 2023
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