PTC Therapeutics at Barclays Conference: Strategic Insights on Rare Disease Focus

On Tuesday, 11 March 2025, PTC Therapeutics (NASDAQ: PTCT) presented at the Barclays 27th Annual Global Healthcare Conference, offering insights into its strategic direction. The company highlighted both the successes of 2024 and the challenges ahead. Key discussions centered around the Huntington’s disease program and the anticipated launch of Cepatirn for PKU, alongside financial guidance for 2025.

Key Takeaways

• PTC Therapeutics reported $807 million in total revenue for 2024, a significant achievement.
• The Huntington’s disease program is progressing, with a Phase II study readout expected in Q2.
• Cepatirn’s launch for PKU is anticipated in Q4, with revenue impact expected in subsequent years.
• Financial guidance for 2025 is set between $600 million and $800 million.
• The company’s cash position exceeds $2 billion, supporting growth and strategic initiatives.

Financial Results

• Total Revenue (2024): PTC Therapeutics reported $807 million, marking a successful year.
• Cash Position: Over $2 billion pro forma, ensuring flexibility for future growth and development.
• Revenue Guidance (2025): Projected between $600 million and $800 million, driven by existing products like Translarna and Emflaza.
• Cepatirn Contribution: Expected to begin impacting revenue in Q4 2025, with more significant contributions in the following years.

Operational Updates

• Huntington’s Disease Program:

- Phase II PIVOT HD study data expected in Q2, focusing on target engagement and clinical benefits.

- Collaboration with Novartis includes a 40% profit share in the U.S.

• Friedreich’s Ataxia Program:

- FDA priority review accepted, with a PDUFA date set for August.

- Potential ADCOM discussion anticipated during the review process.

• Cepatirn (PKU):

- Launch strategy targets therapy-naive patients and premium pricing compared to existing therapies.

- Clinical data shows high efficacy, with 84% of patients reaching target guidelines.

Future Outlook

• Huntington’s Disease Program:

- Discussions with Novartis on a potential Phase III or confirmatory study are ongoing.

- Strategies to target earlier-stage patients with lower doses are being explored.

• Friedreich’s Ataxia Program:

- Awaiting further FDA communication regarding potential ADCOM and label discussions.

• Cepatirn (PKU):

- Preparing for a Q4 launch, aiming for substantial market penetration and revenue growth in future years.

Q&A Highlights

• Huntington’s Disease Program:

- FDA alignment on using Huntington protein lowering as a surrogate endpoint for accelerated approval.

• Friedreich’s Ataxia Program:

- The company is prepared for an ADCOM, depending on FDA decisions.

• Cepatirn (PKU):

- Confident in achieving premium pricing and targeting a large patient population.

PTC Therapeutics remains optimistic about its pipeline and commercial execution. For a detailed account, readers can refer to the full transcript below.

Full transcript - Barclays 27th Annual Global Healthcare Conference:

Gina Wen, SMICARE biotech analyst, Barclays: Sorry. Are we already live? Okay, perfect. Thank you. Good afternoon, everyone.

My name is Gina Wen. I’m a SMICARE biotech analyst at the Barclays. It is welcome to our twenty seventh Annual Global Healthcare Conference. It is my great pleasure to introduce our next presenting company, PBC Therapeutics. On the stage with me, we have Matthew Klein, Chief Executive Officer.

We also have Pierre Grevier, Chief Financial Officer. So, maybe before I dive into the specific questions, I know we have tons of questions with them, tons of programs we can discuss, maybe very brief overview about PTCG for those less familiar with the company.

Matthew Klein, Chief Executive Officer, PBC Therapeutics: Absolutely. Thank you very much, Gina. It’s great to be here. PTC is a global biotech company. We discover, develop and commercialize innovative therapies for diseases of high unmet need.

We’re coming off a tremendous 2024 with a number of important accomplishments, including continued successful commercial execution with total revenue of $8.00 $7,000,000 We submitted four approval applications to the FDA, all of which were accepted for review, setting us up for the potential for four commercial launches in The U. S. Within twelve months. And we solidified our balance sheet now with over $2,000,000,000 pro form a, giving us sufficient cash to get to cash flow breakeven and beyond as well as allow us to continue to grow the company, succeed in our launches and conduct strategic business development to complement our R and D and commercial portfolio. So exciting 2024, setting us up for a number of value creating catalysts in 2025.

Gina Wen, SMICARE biotech analyst, Barclays: Thank you. I wanted to make a comment. The Huntington program so far, I think we haven’t seen any one can surpass that deal term. It’s so favorable to PDCT. I really congratulations on that deal.

Matthew Klein, Chief Executive Officer, PBC Therapeutics: Yes. And

Gina Wen, SMICARE biotech analyst, Barclays: then I know this is one of very important pipeline asset and that’s why maybe Novartis are willing to pay so much money for the programs. So, with the midyear update and wanted to maybe give a little bit layout exactly I know you’ve mentioned in the past about 150 patients, maybe lay out exactly where you will be presenting the data and what should we expect?

Matthew Klein, Chief Executive Officer, PBC Therapeutics: Yes, absolutely. And first, thank you. It was a tremendous deal that I think speaks to the tremendous potential and value of the program already. And not only did we have the significant upfront, but we’re well positioned to enjoy a significant amount of the upside with the 40% profit share in The U. S.

So really a win win for us in many ways. The data readout coming in Q2 is going to be the twelve month readout from the Phase II PIVOT HD study and will include about 100 to 110 additional subjects who cross the twelve month time point, bringing the total number of subjects who are at twelve months to about 140, as you said, 140 to 150. This study was designed to inform pharmacodynamic activity, so evidence of target engagement in terms of lowering of Huntington protein in the blood and the CSF, as well as demonstrate safety and tolerability of the drug and additional markers, including clinical scales that can help confirm that not only is the drug doing what it’s supposed to do, getting where it’s supposed to go in the brain, but also starting to show early signs of clinical benefit that in the long term could represent significant efficacy. So we’ll be sharing at the twelve months an update on Huntington protein lowering in the blood, Huntington protein lowering in the CSF, safety and tolerability, data from clinical scores such as the CU HDRS total motor score and others as well as other biomarkers of disease including NFL.

Gina Wen, SMICARE biotech analyst, Barclays: Thank you. So, I did a quick calculation. We and you didn’t mention in the past like it will be evenly split between stage two and stage three and each will be three arms. So when we calculate roughly each arm is about 25 patients.

Matthew Klein, Chief Executive Officer, PBC Therapeutics: Close enough. Okay. Yes, that’s exactly right. So there’s stage two patients and stage three randomized evenly between placebo five milligrams and ten milligrams. In ten

Gina Wen, SMICARE biotech analyst, Barclays: milligram, yes. So now when we look at the last update, I know each arm is about temptation and you did show those response and the certainly in the blood role lowering and then also the magnitude like a 43% in the CSS and then you do see the longer follow-up, you do see the further improvement in terms of protein lowering? Yes. Yes. And then should we and then of course you do you also did share a lot of the functional measurement, right?

Some has a more clear trend, some a little bit noisy because given small number of patients. So now we have say more than doubling of each arm of the patient like should we expect the similar trend largely persist with this more comprehensive data update?

Matthew Klein, Chief Executive Officer, PBC Therapeutics: Yes, we can certainly we certainly would expect to continue to see similar magnitude of Huntington protein lowering. We expect that to be consistent. And we do expect to see the continued trends of clinical benefit as well. Now it may look a little different for the stage two and stage three patients because as the disease progresses, the clinical scales that are most relevant, the clinical scales on which we can record an effect on disease progression are known to be a little bit different between stage two and stage three. So we will, of course, look for that early evidence of clinical effect with the understanding that it might look a little different for the stage three patients than the stage two patients.

So when we share the data, we expect to be showing not only the patients together, but also if necessary breaking it out by stage two and stage three. So we can clearly understand if there is a different pattern of benefit.

Gina Wen, SMICARE biotech analyst, Barclays: So I know you would not be able to comment on the actual data, but in terms of pattern difference, what kind of a functional measurement you think it could be different between stage two and stage three?

Matthew Klein, Chief Executive Officer, PBC Therapeutics: Yes. So I think it’s well known that one of the endpoints that’s particularly sensitive to change at stage two is the total motor scale. And not surprisingly, when we look at the data from last June’s readout, which included all Stage two patients, we saw a very clear dose dependent pattern of clinical effect on TMS. So that’s one where we may see a similar effect on Stage three, we may not. Something like the CU HDRS, which has different subscales relevant to both stage two and three, maybe a very good measure for looking at the whole population together.

Because again, given the structure of the CUH DRS having many parts to it, it’s able to assay clinical effect across a broader spectrum of disease severity.

Gina Wen, SMICARE biotech analyst, Barclays: So what about the other functional measurements? Any major differences between the two classes?

Matthew Klein, Chief Executive Officer, PBC Therapeutics: There shouldn’t be. I would say with the exception of well, total functional capacity is interesting because total functional capacity is changes on the TFC signifies the transition from stage two to stage three. The definition of a stage two patient with Huntington’s disease is a TFC of 13, which is normal. And someone has said to cross into stage three when that you start seeing a lowering to 12 or 11. So we’ll be looking at the TFC, though I think it’s also important for stage two patients because we can be looking at a transition from a 13 to a 12 as being informative progression as well.

So that’s a to be determined one.

Gina Wen, SMICARE biotech analyst, Barclays: Okay. Very good. And then you likely wish you either separate or group?

Matthew Klein, Chief Executive Officer, PBC Therapeutics: Whichever is most informative. And I realize I didn’t answer your earlier question about where we’re going to be sharing the data. It’ll be similar as we always do, which should be a press release and a call. It’s not going to be at any meeting.

Gina Wen, SMICARE biotech analyst, Barclays: Okay, good. And do you see all these data real time?

Matthew Klein, Chief Executive Officer, PBC Therapeutics: No. In fact, we remain blinded. We were blinded for the first data readout or blinded in treatment assignment and we don’t do any interim looks at the data. I don’t believe in doing blinded data reviews because when you look at data that are blinded, they always seem to look the way you want them to look. That’s why you do blinded studies.

So we just focus on execute we focus on executing the study to the highest quality possible. We lock the database, do the data cleaning and then we’ll analyze the data and understand them.

Gina Wen, SMICARE biotech analyst, Barclays: Okay. Does any clinical team in your company see the data, blinded data?

Matthew Klein, Chief Executive Officer, PBC Therapeutics: No, no, we do the same. It’s the same way through.

Gina Wen, SMICARE biotech analyst, Barclays: Okay. So the same the whole company?

Matthew Klein, Chief Executive Officer, PBC Therapeutics: Yes. We don’t do that right. We do not use any data. Yes.

Gina Wen, SMICARE biotech analyst, Barclays: Okay, good. And then unblinding and then do the analysis, like how long would that take?

Matthew Klein, Chief Executive Officer, PBC Therapeutics: It depends. There’s a lot of analysis that has to be done because remember we have a lot of biomarker data to

Gina Wen, SMICARE biotech analyst, Barclays: analyze. Right.

Matthew Klein, Chief Executive Officer, PBC Therapeutics: We tend to run we try to run the biomarker data in as, let’s just say infrequently as possible. So they’re given subject, what you want to do is run their baseline three months, nine months, twelve altogether because that decreases the variability in the assay. So there’s going to be a lot of biomarker analyses that are going to have to be done that just simply takes time. And so that will dictate I can’t give an exact estimate, but that will dictate where we are in terms of being able to read out the trial. I can comment that we have completed last patient, last visit for the twelve month time point.

So now we’re in that data cleaning timeframe now.

Gina Wen, SMICARE biotech analyst, Barclays: Okay. Because we are almost at the end of 1Q, so you only have

Matthew Klein, Chief Executive Officer, PBC Therapeutics: three more months. Yes. Right.

Gina Wen, SMICARE biotech analyst, Barclays: So Okay, good. Maybe the neurofilament as a biomarker, how important that is?

Matthew Klein, Chief Executive Officer, PBC Therapeutics: So I think there’s two ways to think about neurofilament and Huntington’s disease. It’s become incredibly important as a safety marker. A number of the other HD therapies have shown NFL spikes either early on or over the course of treatment, which of course is not good because that signifies neuronal injury. And we have not seen any treatment related NFL spikes with PTC-five 18, which is a testament to its safety and tolerability. Now, on the other hand is the potential of NFL as a biomarker of efficacy.

It’s understood that in neurodegenerative disorders, neuro inflammatory disorders, neurofilament light chain will increase over time. So if you can slow that increase, that’s thought to be a marker of efficacy. Now in Huntington’s disease, it’s much more of a longer term biomarker. The reason for that is one, it’s a more indolent disease, slow moving disease versus something like ALS where you have rapid progression and therefore market changes in NFL over a short period of time. And over the shorter period of time, such as twelve months, it’s understood that there could be intra patient sort of variability.

And in fact, if you look at the spaghetti plots that we’ve shared previously on the NFL levels, you can see within an individual patient, there’s not that there’s spikes, but there’s slight ups and downs, which in the short term makes it hard to detect a reliable signal of efficacy. We’ll nonetheless look at NFL and are open to the possibility that we could see an early effect there. But I think in Huntington’s disease, things like NFL and brain volume, it’s really hard to get a meaningful effect in a short period of time because things are just moving so slowly.

Gina Wen, SMICARE biotech analyst, Barclays: With the data update, will these two also be presented?

Matthew Klein, Chief Executive Officer, PBC Therapeutics: Yes. Okay. The data update will look very much like what we presented in June in terms of the type of endpoints.

Gina Wen, SMICARE biotech analyst, Barclays: And the FDA communication, like what kind of data you think that will satisfy their comments on, I forgot the exact Associations. Yes, associations between the Huntington protein lowering and the function.

Matthew Klein, Chief Executive Officer, PBC Therapeutics: So we met with the FDA in December and the purpose of that meeting was to align with the agency on the potential for Huntington protein lowering to serve as a surrogate endpoint that could support accelerated approval. Our argument was based on the fact that Huntington’s disease is a monogenic disorder caused by a toxic mutant protein. And there is substantial literature showing that if you can lower Huntington protein 20% to 50%, you can impact disease progression. And so with that in mind, we wanted to ensure that the agency was aligned with our thinking and they said they were aligned scientifically, the rationale for Huntington protein to be a surrogate endpoint. But not surprisingly asked with the large data readout coming, can we show in that readout some associations between changes in Huntington protein and clinical changes.

Why? Because the definition of a surrogate endpoint is something that is likely to predict clinical benefit. And so they asked to see in this data set, can we see some things while it’s early, can we still see some suggestions that over the longer of treatment time, this lowering of Huntington protein can translate to clinical benefit. They were not prescriptive in terms of what they wanted to see, which I think is tells us that we need to look at the data and dose dependent changes on the clinical scares tells you that if you can lower someone’s Huntington protein more, you are likely to have a greater clinical effect. To me that is an association between Huntington lowering clinical effect.

So I think that’s one example of the type of data we could present to the agency.

Gina Wen, SMICARE biotech analyst, Barclays: So do you think that I totally agree with you. The minimal one you have to show those response in terms of Huntington protein lowering and ideally both in the blood and the CSF. Yes. So do you think the agency also will be looking for the correlation? I know they use the word association, but were they also wanted to see the dose response in terms of a functional measurement?

And then which are the I’m pretty sure you wish you all the functional measurement and which one you think are most importantly likely what they care about, like say in the future if you do the confirmatory study that could be the primary endpoint?

Matthew Klein, Chief Executive Officer, PBC Therapeutics: Yes. I think it’s hard to know what they prefer to see. I do think the dose dependent changes in the clinical endpoints would be important. Some of this will also be driven by what the data look like. If the biomarker data are clustered around the median, clustered around the mean, then those dependent changes are going to tell you a lot on the clinical measures.

If we see more variety and more of a spread on the biomarker data, that would then allow you to look at, say, a subject that had really much higher than average lowering, did they have much better scores. So that’s going to be a little bit of the nature of the data are going to help inform how best to show those associations. But I think we’ll look at a number of the different scales. And again, I think what was so compelling about the rate out in June is that on two of the more meaningful endpoints, TMS and CUH DRS, we saw that dose dependent effect.

Gina Wen, SMICARE biotech analyst, Barclays: So for the 2Q update, let’s see, three scenarios, maybe even one in three, some gray area. So one is a very clear association, like what would you define as a clear data that you think FDA will be happy or have an accelerated approval path there? Yes.

Matthew Klein, Chief Executive Officer, PBC Therapeutics: So let me just I’ll answer the second, but let me just give some context. I think where we are with the program for the long term. Very importantly, we’ve already established safety and tolerability. We’ve shown that the drug is working the way it’s supposed to work and going to where it’s supposed to go. So we have de risked this program for Phase three.

And I think the only scenario where that would be in doubt is if there was a strong negative safety signal, which we don’t expect to see given the fact that there’s a DSMB monitoring the study on an ongoing basis and we have some idea there and everything seems to point to the fact that the drug continues to be safe and well tolerated. So that’s really, really good. So now what we’re really talking about is the upside for an accelerated approval and what that would look like. And as you said, I think the upside would be seeing some of these dose dependent associations like we saw in June. I think the upside would be seeing what we a data that looks a lot like the one from last June.

Gina Wen, SMICARE biotech analyst, Barclays: I see. So you think last year data that should be sufficient?

Matthew Klein, Chief Executive Officer, PBC Therapeutics: I think that would give us a very strong basis of argument to the FDA to support XRAR.

Gina Wen, SMICARE biotech analyst, Barclays: So when you talk to the FDA in December and did they say if you can repeat this data, you have the path forward?

Matthew Klein, Chief Executive Officer, PBC Therapeutics: Yes. Things haven’t changed at the FDA so much that they would ever say that. But what they try to because they in general don’t like to tell you if this and that because that boxes you in. And quite frankly, it’s good for us that they don’t because that gives us the leeway to look at the data and be able to have a narrative that’s compelling of how this shows an association that would support ultimate competency. Okay.

And is it

Gina Wen, SMICARE biotech analyst, Barclays: fair to say ten milligram will be the going forward dose if we saw similar datasets.

Matthew Klein, Chief Executive Officer, PBC Therapeutics: That’s something discussion now about the PTC and Novartis teams are discussing that now as we think of the efficacy trial because as I mentioned, we’re all of the belief that we’re moving forward with that efficacy trial. Great, if that’s going to be a confirmatory study in the context of accelerated approval. If we’re not yet at the point that we can file for accelerated approval, that will be designed as the Phase III trial. So all that’s moving forward now and the thinking is we may move forward with both five and ten. The other thing we’re thinking a lot about is not only the Stage two and Stage three patients, but the very, very large number, probably three times the number of Stage two and Stage three patients that are earlier that may have no clinical symptoms at all that would absolutely benefit from a drug like PTC-five eighteen that can slow the onset of symptom presentation.

And so as we start thinking about a strategy to go early and earlier, we may want to also have a lower dose available to understand what the effect could be in earlier stage patients. So right now, I think both are very much in play. Of course, as you alluded to, the data readout will help confirm which of those doses we bring forward, but it will also help our thinking in terms of which we’re going to be looking at over the longer term to access that very large number of patients who clearly would benefit from something that could slow disease onset.

Gina Wen, SMICARE biotech analyst, Barclays: So wouldn’t that be likely would benefit from accelerated approval because functional data would take us forever, right?

Matthew Klein, Chief Executive Officer, PBC Therapeutics: There’s no question that the strategy there will likely be a biomarker bridging strategy, where if you think about it and you could show that a biomarker like Huntington lowering is associated with long term benefit, then the extension early would really be about showing you’re similarly lowering Huntington protein, which of course is something that we would be confident we’d be able to do.

Gina Wen, SMICARE biotech analyst, Barclays: Okay, good. Okay. Now, switching gears, you still have tons of other pipeline assets. We have a few more minutes. I wanted to ask about the ventricle in the fractures ataxia.

Yes. So, I think I was informed that you guys will not have the 74 letter. So that’s already FDA communicate with you guys already.

Matthew Klein, Chief Executive Officer, PBC Therapeutics: Well, we as you know, we the vatipinone for free ataxia program, we received acceptance of the filing with priority review and that was notified in the day 60 letter. So they weren’t obligated to issue a day seventy four letter since they’ve already confirmed that the filing was accepted with priority review. So the next update we would have would be during the review cycle.

Gina Wen, SMICARE biotech analyst, Barclays: Okay. So the mid cycle review basically. And then I did in my rough calculation, that should be April timeframe. That’s all right.

Matthew Klein, Chief Executive Officer, PBC Therapeutics: Yes. We usually don’t guide to mid cycle because it’s the first there’s the internal FDA mid cycle and then it may be a couple of weeks before we know about it. But let’s just say I trust your math is probably you could calculate on six months what the middle would look like. So Right.

Gina Wen, SMICARE biotech analyst, Barclays: Okay, good. And then, so at what point when you don’t hear from FDA, hey, you will not have they never talk about Adacom that you know that okay, you will not have Adacom?

Matthew Klein, Chief Executive Officer, PBC Therapeutics: Yes. Typically, so in the day sixty letter, what they communicated to us is they hadn’t yet reached a decision about an ADCOM. And what that suggests is they want to get a little bit further into their review before they make a decision about an ADCOM or not. I think from our standpoint, we look forward to whatever they decide. I think clearly if they had decided that an ad com would be beneficial, we would certainly be in a very strong position to be able to have the appropriate people support the strong scientific rationale around efficacy.

We know that this is a very vocal and thoughtful patient community, very educated patient community, who we know will be at the ready to help come and explain the unmet need, the unmet need for pediatric patients with FA and also share the experiences that the FA patients have had with this drug. So if the FDA wants it, we’ll be ready. If they don’t, we’ll be ready to continue to work with them and work towards an approval.

Gina Wen, SMICARE biotech analyst, Barclays: So do you think that you have a better chance with EDACONE or without EDACONE?

Matthew Klein, Chief Executive Officer, PBC Therapeutics: I don’t think it matters. I think we have a strong chance in both. I was merely saying that if we had an EDCOM, we would certainly take advantage of it to continue to articulate the important value of this therapy, both in terms of efficacy, safety and meeting a very significant unmet need for pediatric patients with pediatric ataxia.

Gina Wen, SMICARE biotech analyst, Barclays: Maybe based on your prior experience, at what point you will start to discuss with the FDA about the label?

Matthew Klein, Chief Executive Officer, PBC Therapeutics: So I think we’d be a little bit further into the review. I think that sort of gets closer to late cycle time. Again, this is an accelerated this is an expedited our prior review. So it’s relatively short from the acceptance of February to August, which is the PDUFA date. So I think we’d be getting closer to this summer timeframe before we’d start discussing label.

We would expect that we would have a full age spectrum of Friedreich ataxia patients given that the NDA includes both safety and efficacy data in children and adults.

Gina Wen, SMICARE biotech analyst, Barclays: Okay, good. We still have a few more minutes, wanted to discuss about Cepatira in PKU. So maybe launch strategy and then also maybe the thoughts on the pricing and then also the patient population, where you think it would be the low hanging fruit you can start?

Matthew Klein, Chief Executive Officer, PBC Therapeutics: Look, we’re incredibly excited about the opportunity for Cepiaterin. This is a pretty unique rare disease context. While there’s two approved therapies, the vast majority of patients are not on therapy and would like to be on a safe, tolerated and effective therapy. So when you think about a population of approximately 17,000 patients in The United States, that’s a very large number of patients who we believe we can provide ciputera into and make an important difference in their life. The data set we have continues to get stronger and stronger.

We’ve already shown very high proportion of patients having a significant response to sepia terran. We had eighty four percent of patients in the trial reaching target guidelines. We shared on the Q4 earnings call that the latest fee tolerance update shows that ninety seven percent of patients are liberalizing their diet, the fee tolerance protocol. Two thirds of those are reaching the recommended daily allowance and beyond, All very important points. We’re doing a little bit more work into some of the genotyping of patients who’ve been in the studies and we’ve already previously shown that we’re having an effect in patients who have what are known as non BH4 responsive mutations.

These are mutations that are often associated with classical PKU and these are types of mutations that a lot of the patients who are in the therapy naive bucket have. So to be able to have the efficacy data we have in these patients supports that not only can we reach patients who are on existing therapies, patients who failed existing therapies, but reach that bucket of patients as well who are therapy naive. And it’s when you start thinking about the ability to penetrate all key segments is why we believe we can achieve significant penetration in the market. Based on the data package, the discussions we’ve had with payers suggest that we can successfully price at a premium to Palynzi. And when you do that math, our guidance of $1,000,000,000 globally, but even likely $1,000,000,000 in The U.

S. Alone becomes very real. In terms of low hanging fruit and segments, it varies center to center. We know there’s a lot of the key centers in The U. S.

That already have hundreds of patients on waiting lists. This can include patients currently on BH4, because it’s known that all patients who’ve been on BH4 have a greater response to ceputaren, as well as patients who are therapy naive, who desperately want to be on a safe, tolerated and effective therapy. So there’s lots of sources of low hanging fruit that may vary center to center.

Gina Wen, SMICARE biotech analyst, Barclays: Okay. We’ll ask the last question to Pierre regarding the revenue guidance and how much assumption for the DME franchise versus Cybertery?

Pierre Grevier, Chief Financial Officer, PBC Therapeutics: Yes. So look, as we started the year, we provided a wide revenue guidance $600,000,000 to $800,000,000 There’s very little Trans Europe in that guidance. I think the delta is really in Flaza, for instance. There’s definitely PKU numbers in there. Although, we believe in the strong launch and the billion dollar opportunity, this is second half, right, will be starting.

So it’s a few months. So it’s not going to be meaningful this year. It’s really in line revenues for the most part.

Gina Wen, SMICARE biotech analyst, Barclays: Should we consider like a real revenue contribution will start 4Q for Cepatira?

Pierre Grevier, Chief Financial Officer, PBC Therapeutics: Absolutely, there’s revenue. Yes, of

Gina Wen, SMICARE biotech analyst, Barclays: course. Great. We’re running a little bit over time, but we have lots of great discussions and we look forward to 2Q data updates. Absolutely. Thank you guys.

Matthew Klein, Chief Executive Officer, PBC Therapeutics: Thank you, Jamie.

Gina Wen, SMICARE biotech analyst, Barclays: Okay. Bye bye.

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