Q4 2022 VectivBio Holding AG Earnings Call
Speaker 1: Welcome to the VCTIS-BIO's fiscal year 2022 results call. At this time, all participants are in a listen-only mode. After the speaker's presentation, there will be a question-and-answer session. To ask a question during your session, you will need to press star 1-1 on your telephone.
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Speaker 1: To withdraw your question, please press star 11 again.
Speaker 1: Please be advised that today's conference is being recorded.
Speaker 1: I would now like to hand the conference over to your speaker today, Patrick Malloy, Senior Vice President Investor Relations. Please go ahead. Patrick Malloy, Senior Vice President Investor Relations.
Speaker 2: Thank you and good morning everyone. Welcome to today's call. During which we will provide an update on the company and review our financial results for the full year and in December 31st, 2022. Earlier this morning, we should press release, summarizing our financial results and progress across the company, which is available on our website.
Speaker 2: I'd like to remind everyone that during today's discussion, we will make statements about our future expectations, plans, and prospects that constitute forward-looking statements for the purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995.
Speaker 2: Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including the risk factors discussed in our FCC filings, and we are not under any obligation to update these forward-looking statements.
Speaker 2: Joining me on today's call are Lucas Santorelli, our Chief Executive Officer, Claudia De Algusta, our Chief Financial Officer, along with Chief Medical Officer, Mark Wajra, and Kevin Harris, our Chief Commercial Officer.
Speaker 2: Following the prepared remarks, we'll open up the call to a question and answer session.
Speaker 2: Now, I'd like to turn the call over to Luca Santorelli.
Speaker 3: Thank you, Pat, and thanks to everyone for joining us today.
Speaker 3: For those hearing our story for the first time, Vectibio is a growing, late-stage biopharmaceutical company that is focused on developing potentially light-transforming therapies for people living with serious rare diseases.
Speaker 3: We are a team of individuals with deep scientific expertise, driven by a relentless passion to make a meaningful difference in the lives of people living with rare diseases, by developing treatments that are both transformational and tailored to the needs of individual patients.
Speaker 3: In 2022, we have made tremendous progress in developing a lead molecule, aprobutide, a next generation GLP2 analog that bears the promise of unlocking the full potential of the GLP2 class, thanks to its unique pharmacology and also our patient tailored development strategy.
Speaker 3: We believe that apaglutide could transform the lives of people suffering from a broad range of severe rare gastrointestinal diseases, including short bowel syndrome and intestinal failure, or SBS-IF, and acute graft-versus-source disease, or AGDHD.
Speaker 3: In 2022, we focused on execution both towards our R&D and corporate goals.
Speaker 3: And we also position ourselves for our next stage of growth.
Speaker 3: We were able to achieve several key strategic and program milestones despite the challenges that 22 created in the biotech environment.
Speaker 3: In Q1 2022, we signed a Japan licensing deal with the Zikaze Pharmaceutical designed to develop and commercialize a propitiate in Japan.
Speaker 3: In Q2, we announced the dosing of the first two patients in TARGASE, our proof-of-concept study in acute GVHD, currently recruiting across US and Europe .
Speaker 3: In Q3, we announced promising interim six-month data from the Stash nutrition study, a first-of-its-kind study in patients with colony continuity anatomy, or CSC, which represents an underserved majority of SPS-IF patients.
Speaker 3: In November , we announced the completion of enrollment for the colony continuity stratum of the Phase III Stash Pivotal Study. And finally, we strengthened our cash position by raising a total of $284 million to be above non-dilutive inequity transactions.
Speaker 3: The current cash position provides VECTI bio with a financial runway into 2025, which is over 12 months after the phase 3 results.
Speaker 3: We believe that 2023 will be a transformation year for rectiBIO with clinical readouts from Stas nutrition, Stargaze and the Phase III stars all over the next several months.
Speaker 3: 2023 will be a transformation a year for rectiBio with clinical readouts from Stas Nutrition, Stargaze, and the Phase 3 stars all over the next several months. More specifically,
Speaker 3: In early May, six months data from Stas Nutrition will be presented at an upcoming scientific conference.
Speaker 3: This study aims to demonstrate that apaglutide can improve intestinal absorption and reduce the need of parenteral support in SBSIF patients with colonic continuity.
Speaker 3: an anatomical subtype of SPS where there is limited data supporting the use of GATX.
Speaker 3: Alongside the progress of the Stash Musician Study, the enrollment completion in our Phase 3 program of apaglutide in SBSIS is anticipated by the end of this quarter.
Speaker 3: At this point, we have closed screening of new patients, as those currently under assessment will enable us to reach the enrollment target based on our current screen failure rate.
Speaker 3: We plan to issue a press release following the randomization of the last patient.
Speaker 3: Moving on to acute GVSD, I'm happy to announce that we have enrolled all patients necessary to perform the pre-planned interim analysis of our Stargaze Phase II program by the end of Q2.
Speaker 3: This analysis will help us inform the development and filing strategy in acute GVHD, where aproglutide has the potential to become the first non-immune suppressive treatment to address GI damage, which is one of the most critical complications of this disease leading to mortality.
Speaker 3: A positive stargaze outcome would also establish the rationale to explore the GI healing and regenerative properties of apaglutide in other inflammatory GI conditions, where immunological therapy is currently the main therapeutic approach.
Speaker 3: Now, I would like to turn the call over to our Chief Medical Officer, Dr. Omar Quajia, who will share additional details on our pipeline progress.
Speaker 3: Omar, over to you.
Speaker 4: Thanks, Luca, and good morning, everyone. I will provide more detail on some of the major studies of acroglutide that we'll read out this year.
Speaker 4: At Digestive Disorders Week in Chicago on May 9th, we'll present important six-month data on all nine patients enrolled in our Stiles Nutrition Study.
Speaker 4: SARS nutrition is the first ever dedicated study in patients with SBSIS with colon incontinuity, or CIP.
Speaker 4: These are patients where the remnants small intestine remains connected to a functional colon and represent the majority of SBS-IS patients.
Speaker 4: The study participants were treated with the same dose used in our phase three study, and to whom the same CIC specific parental support leaning algorithm was applied.
Speaker 4: Many participants were treated with the same dose used in our phase three study and to whom the same CIC specific parental support weaning algorithm was applied. The study has two components.
Speaker 4: The main component is an assessment of PS reduction at 6 months after the first metabolic balance period.
Speaker 4: We released intrinsic on site patients in October 2022, which demonstrated the very robust effect of a 50% reduction in PS volume at six months.
Speaker 4: We will present data from all patients at this time point.
Speaker 4: The study also has an exploratory component, which is a metabolic balance analysis to quantitatively assess acroglutide effects on intestinal absorption at an early time point of four weeks and a late time point of 48 weeks compared to baseline. We will report data from four week time points at DDW.
Speaker 4: where we aim to show data that supports the early positive effects of apoglutamide on measures of absorption.
Speaker 4: Mid-year, we will have data from the first interim analysis of patients in our Stargate Faith II Proof of Concept Study of Acroglucide in Patients with Steroid Refractory Acute GAI. Grass TMFO.
Speaker 4: This disorder is a consequence of attack by donor T cells on the GI tract.
Speaker 4: mortality at six months and represents a very high unmet medical need.
Speaker 4: We're recruiting up to 34 patients who are treated with a high or low dose level of Afroglutide on top of corticosteroids and ruxilizumab.
Speaker 4: The interim analysis will include safety, tolerability, response rate, and durability of response in the first 17 patients in the study treated for at least 56 days.
Speaker 4: All patients to support this interim analysis have been randomized.
Speaker 4: Positive data from the Stargate can support a pistol program in acute PBHD.
Speaker 4: In addition, we see this data in an immune disease of the GI tract as proof of principle that may unlock the potential for Afroglutide in other immune and inflammatory GI conditions, such as ulcerative colitis or Cre storytelling.
Speaker 4: Finally, by Q4, we will have data from our Phase III STAR study in patients with SBSIS.
Speaker 4: Positive data will form the basis of our application for approval of apaglutide for use in patients with SDS who are dependent on parental support.
Speaker 4: STARS is the largest clinical study ever conducted in SBSIS patients.
Speaker 4: and is recruiting 144 patients with prospectus stratification to include 50% CIT and 50% STERNA.
Speaker 4: Study participants are randomized to a single 5 milligram dose of paproglycerides.
Speaker 4: 2.5 milligrams for patients with a body weight below 50 kilos, also placebo in a two to one ratio.
Speaker 4: In the placebo control phase, seven patients are treated for 24 weeks, while CIT patients, who are thought to accrue benefits after six months, are treated for 48 weeks.
Speaker 4: The primary endpoint is parental support volume reduction at 24 weeks in the whole study population.
Speaker 4: with common and anatomy specific alpha-controlled key secondary endpoints assessed for 24 weeks and 48 weeks.
Speaker 4: In addition to important clinically meaningful secondary endpoints, such as days off parental support and enteral autonomy, the study also includes an extensive suite of patient reported outcomes.
Speaker 4: to further establish the clinical benefits of acroglutide. All patients from STARS are eligible to roll over into the STARS EXTEND four-year open-label extension study.
Speaker 4: Recruitment has remained well on track.
Speaker 4: We completed recruitment in the CICR for the 48-week treatment period in October 2022.
Speaker 4: We are close to completing recruitment into the stoma arm, which requires a 24-week treatment period.
Speaker 4: As Luca mentioned, we have closed screening of new patients, as those currently in screening will enable us to reach our enrollment target.
Speaker 4: Study retention has been extremely good and we look forward to our top-line results in Q4.
Speaker 4: We have made strong progress in the clinical development of acroglutide in SBS as well as the QGVHD and are actively exploring additional indications.
Speaker 4: We look forward to providing updates from the key readouts in 2023. I'll turn the call over to our Chief Commercial Officer, Kevin Harris.
Speaker 2: Thanks Omar. In 2022, we've made great progress advancing our launch strategy and plans for a pragmatize. And I've been laying a strong foundation for commercialization over the past 3 years.
Speaker 2: We have been focused on 4 key areas. The 1st area is market development. We have a good understanding of the unmet needs and lessons learned from 1st generation GLC 2 treatment and the challenges of living with chronic support. Which will provide an important foundation for our market development messaging and plans. The 2nd key area is market development messaging.
Speaker 2: is building our product strategy and branding for a Praglitide, where we have completed key long lead time activities in advance of Phase III results.
Speaker 2: The third key area is market access. We have had significant engagement with US and European payers, which has informed our value strategy and our market access and data generation plans.
Speaker 2: The fourth key area is building our patient-centric infrastructure to support the successful launch of a Preglotide. We have completed the design work for a high-touch distribution and patient services model to enable a positive experience with a Preglotide from day one. We have also designed our compliance roadmap and IT infrastructure plans for long.
Speaker 2: patients in the advocacy community.
Speaker 2: As we look ahead, 2023 is the year of launch readiness as we continue to finalize and execute our plans in advance to phase 3 results.
Speaker 2: We are already off to a strong start this year. We recently completed a large and robust US insurance claims project.
Speaker 2: We are already off to a strong start this year. We recently completed a large and robust U.S. Insurance Claims Project with input from experts.
Speaker 2: This project had 2 goals. The 1st goal was to further refine US projected prevalence of the population. And improve our knowledge of patient demographics and treatment characteristics.
Speaker 2: We now estimate the number of adult patients living with SPSIS in the US to be 9,000 and growing.
Speaker 2: These are patients who have had surgical resections and are on chronic and continuous parental support. For at least 6 months, we also estimate an additional 3 to 4000 pediatric patients living with in the US.
Speaker 2: The second goal of the project was to generate more accurate and actionable targeting information for our commercial deployment strategy.
Speaker 2: We now have a very good understanding of who is treating FBSIS patients and where they are being treated and have established a prioritized targeting list at the position and the count level.
Speaker 2: Account level targeting will be our primary go to market approach as we seek to support the multidisciplinary team that takes care of the patient.
Speaker 2: We will use this information as the basis for our field-forced size and structure work, which will begin later this year. We continue to be on track for all pre-launch planning activities and look forward to continuing to update you on the progress of our plans.
Speaker 2: With that, I will now turn the call over to our CFO Claudia Degusta to discuss our 2022 financial results. Claudia?
Speaker 5: Thank you, Kevin. I will briefly comment now on the key financials for the year 2022. We recognize revenue from contracts with customers of 27.3 million dollars over the year and the December 31st, 2022, related to the partnering agreement with AKP.
Speaker 5: Resurption development expenses were $74 million for the year and the December 31, 2022, as compared to $50.2 million for the year and the December 31, 2021.
Speaker 5: The increase of $23.8 million year-over-year was primarily due to an increase of clinical manufacturing expenses of $16.1 million related to the progress made on our Phase III STARS study.
Speaker 5: The base 2 stars nutrition study of apagodine in SPSIS patients and the stargaze proven concept study in acute grafts versus host disease and the dual-camber syringe activities. An increase of 0.9 million dollar of entry expenses, primarily caused by the increase in the paid all expenses of 2.9 million dollar of entry expenses.
Speaker 5: in 2021.
Speaker 5: General and administrative expenses worth $33.9 million for the year and the December 31, 2022.
Speaker 5: Compare to $36.5 million for the year and December 31, 2021.
Speaker 5: The decrease of $2.6 million year over year was mainly attributable to a decrease in employee expenses of $5.4 million from having caused by a decrease of the non-cash share-based compensation of $6.8 million.
Speaker 5: partially upset with an increase in the paid expenses of $1.4 million, driven by an increase in employee account.
Speaker 5: and an increase in professional services expenses of $2.1 million, mainly due to corporate activities.
Speaker 5: Cash and cash equivalent were $221.4 million as of December 31, 2022, compared to $102.7 million as of December 31, 2021. Based upon our current operating plan, we estimated out
Speaker 6: Love you.
Speaker 3: We covered a lot of ground during today's call and to close I'd like to underscore the following three points.
Speaker 3: today's call and to close I'd like to underscore the following three points. first
Speaker 3: 2023 promises to be a transformational year for Vectibio.
Speaker 3: As we will read out important data sets, including the six month nutrition data in just a couple of weeks.
Speaker 3: followed by the interim analysis of the phase two Stargate study, and then, perhaps most importantly, top line results for our phase three star study.
Speaker 3: Second, we are executing a comprehensive launch preparation plan with the aim of achieving a blockbuster launch of apaglutide in SBS intestinal failure.
Speaker 3: And finally, we are operating from a position of financial strength with a cash runway that takes us more than 12 months past the top line readout of the Phase III STAR Study.
Speaker 3: And with that, we would like to open the call for questions. Operator?
Speaker 1: As a reminder, to ask a question, please press star 11 on your telephone and wait for your name to be announced.
Speaker 1: To withdraw your question, please press star 11 again. Please stand by while we compile the Q&A roster.
Speaker 1: The first question comes from Alice in Brazil with Kuiper Sandler. Your line is now open.
Speaker 7: Hey, good morning guys and thanks for taking the questions. So just first, the starting instruction update at DDW, could you just frame for us the scope of the data that's going to be available next month and just what measures body weight?
Speaker 7: wet-weight absorption or other metrics in addition to parenteral support reduction are going to be most important to illustrating the benefits of APRA in the CIC population. And then I do have a follow-up question after that.
Speaker 4: Hey, Ali, thanks for your question. Thanks for jumping on today. How about we have Omar handle that first question. Yeah, thanks Ali. So DDW as well as the PS volume reduction will also show the energy content of the PS, so the number of the mass calories in the PS and how that changed over the six month period.
Speaker 7: And then also what happens to the patient's body weight over that timeframe. Great. And then just on the Stargaze readout this quarter, can you just kind of frame for us what you need to see to feel like you've established proof of concept in GVHD.
Speaker 7: And also, to do like, give established proof of principle and inflammatory GI diseases more broadly. And I just hope that you could also describe your thinking on the opportunity or the role of APRA in larger indications like Crohn's and UC and just how the star gaze interim data kind of informs on your strategy there.
Speaker 4: Great, we'll have Luca take that question.
Speaker 3: Thanks Ali. So in terms of what we will report in that analysis, we likely will report the effects of the drug on overall response rate, both at one month and two months and durability of response.
Speaker 3: which obviously have a precedent as endpoints from previous trials, for testing, for example, a racks of liquefied in this indication. And the exact bar is being set by the data that was generated in the past, both.
Speaker 3: from those combined datasets.
Speaker 3: You know, the importance of those other sets is such because we all of those have a son of the care of Roxolidinit and of course steroids. And we are able to extract all the patients that had GI symptoms among which represents about 80% of it.
Speaker 3: the patient's in a steroid refractory GBSD. So it's a very appropriate data set. In terms of where the bar is, we'll communicate that as soon as we have that those data sets finalized.
Speaker 3: In terms of new indications, well, first, I would say I would like to start by saying that.
Speaker 3: Clearly, the novelty of this approach is that we're combining regeneration of the intestine and healing of the intestine with immune suppression. So, immune suppression in this patient's exclusion to dampen the alloy immune response from the engrossed tissue.
Speaker 3: and is achieved by steroids and other immune suppression such as JAK inhibitors.
Speaker 3: And then you combine with those standards of care, you combine the GLP2 mechanism, which adds gut regrowth as well as increased barrier function, healing, improvement and absorption, all of those features that are known to be part of the GLP2 mechanism.
Speaker 3: But the importance of this proof of context is that this can be extended to additional immunological conditions in testing. We think I would ease the next place to look for opportunity when it comes to upper glutide. And of course, they are the notion and the concept would be similar. We would be combining our drug.
Speaker 3: with existing immunosuppression therapy, for example, anti-GNFs or anti integrins that are currently the standard of care for biologists in this indication or at least second line in this indication. The helped usGU well as.
Speaker 7: Great. Maybe just one last one. I think we talked about developing a pre-celled surrender, dual team research. Could you just remind us what the status of that? And if you have a sense of what kind of data you need to generate to...
Speaker 3: to get a prefilled trench for APRA approved. Thanks. Yeah, so we're developing with the timelines that is compatible with launching in 2025 with such a device, drug device combination. So our main case is to launch
Speaker 3: a base case is to launch with the dual chamber per field syringe. We're going through a series of planned programs in the development of the syringe, including human factor studies, including ROTB-A studies and or by availability studies.
Speaker 3: and validation studies that are purely on the CMC side and all of that is work is progressing in parallel with the conduct of the Phase 3. We have completed the GNT manufacturing that will be required for the...
Speaker 3: a relative bioavailability study, which is due to start imminently. So we're basically on time for a launch with the dual chambers range.
Speaker 7: Excellent. Thank you.
Speaker 1: Please stand by for our next question. The next question comes from Tazeen Ahmed with Bank of America. Your line is now open.
Speaker 8: Hi, guys. Thanks so much for taking my questions. I have a couple. So maybe just conceptually, Luca, can you explain to us? We've been getting a lot of in-bounds with interest specifically the CIC population that you've enrolled into the STAR study. Obviously, the competitive landscape is wide open for CIC, but I think it would help if you could frame for us.
Speaker 3: One is that one is molecule specific, and the other reason is trial design specific. So when it comes to the molecule, we believe our molecule has one of the greatest features, pharmacologically speaking, both in terms of half-life and pharmacology like affinity to the receptor that make it.
Speaker 3: more potent as shown in published clinical head to head studies. And then when it comes to design, I think this point is actually perhaps just as important if not more important.
Speaker 3: CIC patients are physiologically different from stomach patients. They display a response to drug in a way that is distinct from stomach patients.
Speaker 3: The old methodology for detecting response and adjusting parental support in clinical trials was perfected for some patients, but not for CIC patients.
Speaker 3: And we have evolved that methodology as shown in our nutritional trial. Well, that methodology was deployed for the first time to basically be increase the sensitivity of detecting the response in CIC patients. So to eliminate this Sammy what I've Social data into Vancouver channel. I just did a Britain tab over the holidays last night and this is the strut the Pew antidepressants because Sports Commission and Wisht familiarity from Lucy
Speaker 3: Essentially, we are enhancing both the chance of seeing a response because of the molecular nature of apatootide as well as for the methodology used in the trial.
Speaker 3: And of course, to be more specific in CSE patients, the focus in terms of tracking their response is not on changes to their metabolism, changes to their body weight, their stool, consistency of frequency, and less so on their fluid balance of changes in other words, the uruses and absorption of fluids.
Speaker 3: which have more critical for summer. Those are the key different differentiation aspects of the trial design. And lastly, I would add to that to the fact that being able to inform the use of the drug in a bespoke fashion, in a patient tailor fashion in the two subtytes, we'll also increase the adherence to treatment and ease of use and compliance.
Speaker 3: and persistency which are critical objectives for our drive when one sees on the market. So, you know, to help make the use easier and more appropriate with patients, something that has been left on the table we think from the process experience of the pioneering experience of GADES.
Speaker 8: Okay, that's super helpful. Is there anything that we should be looking for at that top line for the STARS results as it relates to CIC? You talked about some of the observations that might be more important for those patients, but as it relates to those endpoints, are there certain cutoffs that you think are necessary in order for this to be? There are some recreations that I can talk about at the end of the link here but that
considered clinically meaningful data in that subgroup? Well, in the CIC subgroup, we put a lot of emphasis, first of all, there's a couple of different aspects that are relevant. The...
meaningful data in that subgroup? Well, in the CIC subgroup, we put a lot of emphasis. First of all, there's a couple of different aspects that are relevant. The timeline of the response...
as it focus more on metabolic changes and less on fluid changes, it's a bit longer. So the...
more metabolic changes and less fluid changes is a bit longer. So the
the expectation to have a very quick response should be tempered for this patient. That's why we ran our phase three trial and our nutrition trial for 48 weeks to double the duration of what we've done for stoma and what has been done in the past.
The other point is we expect the effects not to stop a six months with a crew beyond six months. So basically we're likely able to demonstrate that there is still benefit for patients to be on treatment beyond six months in a nine months to 12 months timeframe.
And then we can record maybe a larger effect size beyond the six months point for this group. And again, that's why we kept the stars.
phase 3 trial blinded for these patients all the way to 48 weeks.
In terms of what outcomes are more relevant for these patients, of course, these are patients that are more likely to be able to shave days off because they don't dehydrate as readily as stomach patients. Dehydration is a key enemy of being able to come off days of infusion because one can dehydrate very quickly.
within a 24 hour period. And secondly, so again, days off is a crucial endpoint that we have at the top of our hierarchy. And then another point is obviously enteral autonomy or complete win off from parental support, which we think is more achievable for CIC.
Okay, perfect. Thanks, Luca.
Please stand by for our next question.
The next question comes from Thomas Smith, SBB Securities. Your line is now open.
Hey, guys. Good morning. Thanks for taking the questions and congrats on all the progress. A couple of questions on our end. I guess first, a follow-up question on the STARS PIVITAL trial and some of the assumptions you're making for the CIC patients. We know in the GATEX program there was a 24% reduction in PF volume in the placebo arm for CIC, but we're not
Thanks for the question. So basically we're.
We have changed not just the winning algorithm, which should increase the sensitivity of detection of responses to the CSE, but we have also changed significantly the optimization stability mechanisms that are designed to reduce the possible responses in this population.
Essentially, what was observed in the past was on average a 20, 20, 20, 22% placebo response, which was attributable to patients doing the, you know, in the course of the trial, requiring less parental support irrespective of the of the GFP to treatment.
because of a better optimization of their nutrition, oral nutrition, or parental support nutrition. So we have invested significant amount of effort and time in making this patient very stable and optimized before the status of the drop.
So again, so these two factors, the placebo response reduction and the winning algorithm which we tested already in the nutrition trial are predicted to give us a more significant signal in the CSE subgroup. Now, in terms of what we have powered towards for the CSE, we...
so it's just relatively, you know, small delta that we are aiming for here. What we have seen in the in the nutrition trial would indicate that the delta could be as big as 30%.
But, of course, we had to assume that in the placebo control trial, we may not get that large delta between placebo and an active.
simply because it's a more typically when you run trials on a large number of sites and large number of countries the signal detection diminishes so we we we think that the
nutrition which has a reduction of about 50% if you assume 20% placebo is about close to 30% Delta there. I think that will be sort of the best case scenario. Got it, that's super helpful. Great and then just one on the
competitive landscape in SPS. Can you just update us on your latest expectations for competing GLP2 programs and generic GATX and how you see the market shaping up over the next three to five years?
Yeah, I'll start and then we'll have Kevin continue. In terms of the expectations from other sponsors, we're obviously not common about our programs. We're confident we have a very competitive package here.
We are the only company with a weekly drug. We are the only company with an ability to discriminate between different anatomical subtypes, which we think is very crucial for this population.
And we are the only company that has a significant amount of patient reported outcomes that are bespoke that were designed specifically for this population. And so there's plenty. And if you combine that with what we know about our molecule versus the others, we're very confident we have.
a very high chance of differentiation in terms of the, in terms of the generic landscape, but I think we now are further advanced into the generalization of Gadigal, a lab cabin comment on that. So, good.
because he has done a lot of work in this space, Kevin. Yeah, thanks, Luca. As you may know, maybe aware of, there could have been a possibility for a generic of Gathex to launch.
in the second half of March of this year. And we haven't seen one emerge at this point. We think there's a lot of reasons why both the SBSIF market and the profile of GATX in particular has limited attractiveness for generics.
There's some challenges one has to overcome in transitioning from the recombinant manufacturing approach for GATX today to synthetic manufacturing, which would be required under the and the guidelines and that clearly would require pretty significant CMC investment. It does take some effort to identify patients and to keep them on treatment as we've shown.
Patients on GATX have significant challenges with persistency and half of them discontinue by month 12 and up to two thirds. By month 24, there's a there's REMS and registry requirements for GATX. And so these are just some of the factors we think why the market isn't particularly attractive for generics. We've only seen one paragraph for filing.
to date and that was done back in 2016. And we still have not yet seen an approval. So I think a generic remains to be seen whether one will come to market. If one does come to market, we've had significant engagement with payers and at the end of the day they view generic gat-techs without a lot of the support that a corporation would provide.
And as Luca mentioned, you know, we believe we'll be able to demonstrate very significant differentiation on efficacy and obviously on dosing being a weekly dose drug. So we don't see a generic as a strategic threat to the opportunity that we think a Praglitide has in SBSIS.
Got it. That's super helpful. Thanks, guys, for taking the questions. Please stand by for our next question.
Our next question comes from Patrick. Dolasal with Lifeside Capital. Your line is now open.
Hi, thanks for taking the question. So it's interesting thinking about the speed of CIC enrollment and now kind of waiting a little bit longer for the Stomachohort. Just curious, what's the relative proportion of CIC versus Stomach patients currently receiving GATX and how might your marketing strategy and pivotal trial design ultimately kind of shift this ratio over time?
countries and feasibility work we've done leading up to that, including market research we're conducting.
both in the US and in Europe . So we estimate the CIC population to have increased since the first launch of GALEX because of the greater appreciation of the importance of keeping at least a portion of the column intact.
to provide patients with the ability of absorbing fluids. And so we have seen an increase in proportion. We estimate that the number of CSE could be anywhere between 62.
70% we don't obviously know exactly exact number but I would say a fair estimate would be around 65% versus 45% you know 35% that you know being a being stormer.
And what was the other part of the question? I think it was how it may impact our marketing approach. Okay, so maybe we'll have… Yeah, well, okay, Kevin, you want to take the second part of the question?
Yeah, and we've got a couple data points to suggest that GATX is used at least one and a half to two times more frequently in stoma than CIC. Recognizing that CIC represents the majority of patients. And so we've always believed that these patients have been underserved.
We obviously have seen that GATTEX and a retrospective analysis didn't demonstrate any effect in that population. We also have a good understanding that that is one of the main drivers of discontinuation for GATTEX, which is lack of efficacy in particular in the CIC population. So much of our strategy beyond obviously having a...
The product with better pharmacology is really to address the unmet needs by an enemy. There's certainly significant room for growth in the stoma population because our market research indicated that still less than half of patients might be getting gas hex in that population, but really the market growth potential and the long-term potential of a priorglotide.
can be significantly driven by that 60 to 70% of CIC patients of whom only a fraction are getting GATX today. So our design of the face retrial with anatomy specific endpoints and our ability to be able to demonstrate in a prospective alpha-controlled manner.
days off in enteral autonomy in the CIC population, I think uniquely positioned us to be able to proactively promote and educate people not only on the benefits of APRA, but as Luca mentioned earlier, how best to measure APRA's effect and to appropriately wean the CIC patient off of PS. So I think there's very tight integration between our...
development strategy and our commercial and marketing strategy, I think we're well positioned for success. Got it, and in the Stargate trial for GVHD, could you just walk us through how GI involvement in particular affects outcomes? And then just curious how the propensity control arm is kind of handled. Do you guys have patient level data to kind of match disease characteristics and other...
And post-transplant, the...
the donor, P cells, which are contained in the graft, attack the host and the GI tract delivering the skin at the primary targets for that. And basically, in the gut that leads to profound inflammatory condition, which often leads to that.
because of the gut being denuded and the patients develop quite life-threatening bile salts and and off-movic diarrhea and out.
The other problem is that the gut barrier itself becomes intestinal barrier becomes impaired as well and said there's translocation of bacteria across the gut from the gut loom and into the bloodstream and the patient's will develop such this.
So the GI tractors thought to probably account for, you know, significant proportion of the mortality. So the skin and liver typically are reasonably well addressed with immunosuppression, but it's the gut that kind of remains a significant unmet need in the population.
Thank you.
Thank you.
Patrick, does that answer your question? Yeah, and then I guess the second part was just on the propensity match control.
So we do have patient level data and propensity score matching basically is used to ensure that we've got a well matched synthetic control to compare our outcomes from StarGaze.
Got it. Perfect. Thank you. I show no further questions at this time. I would now like to turn the conference back to Luca Santorelli for closing remarks. Thanks to everybody for participating.
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