Q2 2023 Agios Pharmaceuticals Inc Earnings Call
[music].
Good morning, and welcome to the AGL second quarter 2023 conference call. At this time all participants are in a listen only mode there'll be a question and answer session. At the end. Please be advised that this call is being recorded I just request I would now like to turn the call over to Chris Taylor Vice.
President Investor Relations and corporate communications.
Thank you operator, good morning, everyone and welcome to <unk> second quarter 2023 Conference call you can access slides for today's call by going to the investors section of our website at <unk> Dot com.
On today's call I am joined by our Chief Executive Officer, Brian Goff, Dr. Sarah Hewins, Chief Medical Officer, and head of research and development.
Set of melanoma, our chief commercial officer, and Cecilia Jones, Chief Financial Officer.
Before we get started I would like to remind everyone that some of the statements. We make on this call will include forward looking statements actual events and results could differ materially from those expressed or implied by any forward looking statements. As a result of risks uncertainties and other factors, including those set forth in our most recent Phi.
Fillings with the SEC and any other future filings that we may make with the SEC.
And with that I'll turn the call over to Brian .
Thanks, Chris Good morning, everyone and thank you for joining us.
<unk> is focused on delivering transformative therapies for patients living with rare diseases and in particular, where the pioneering leader in PK activation focused on hematologic diseases in the second quarter, we made significant progress advancing our industry, leading pipeline of PK activators targeting humans.
<unk> diseases and share a common underlying pathophysiology.
With each step forward each data readout the probability of success for the platform has strengthened and we are excited to share updates with you today as.
As we articulated at the beginning of this year, we're prioritizing potential business development opportunities based on five key criteria rare.
Rare disease focus transformative for patients and identified regulatory pathway.
Potential to Derisk early and a clear path to value creation.
Earlier. This morning, we were very pleased to announce a licensing agreement without an island pharmaceuticals, the leading RNA Therapeutics company that is highly aligned with these five criteria.
Under this agreement <unk> will acquire the rights to develop and commercialize novel preclinical ESI RNA for the potential treatment of polycythemia Vera or PV.
PV is a rare and potentially fatal hematologic disease that affects approximately 100000 patients in the U S.
And for which phlebotomy is the standard of care or.
Our goal is to address the high unmet need in PV by delivering a convenient disease modifying treatment option that reduces or eliminates the need for full bottoming.
This agreement is therefore aligned not only with our business development strategy, but also our core scientific expertise and clinical and commercial capabilities in rare hematology.
We look forward to initiating IND, enabling studies later this year Sara will provide more detail on the Si RNA development candidate in just a few minutes.
Also this quarter, we announced positive results from the phase II portion of the operationally seamless phase III three rides up study of <unk> in sickle cell disease.
The study met the primary endpoint of hemoglobin response for patients in both mid pit that treatment arms and in recent weeks. Our team has continued to analyze the results and has selected the 100 milligram dose for the phase III portion of the study we.
We are now focused on phase III execution and are quite eager to enroll the first patients later this year.
Broadly these results add to the growing body of consistent and compelling data that we have continued to generate with our PK activators highlighting the potential of this differentiated mechanism of action to transform patient function quality of life and long term outcomes across multiple disease areas.
He is in.
In fact with more than eight years of clinical experience and the largest dataset for any PK activator pirate kind has demonstrated consistent results across three distinct diseases.
In this context, we were also pleased to announce this quarter that we've completed enrollment in both phase III studies submitted pipette in thalassemia as well as the phase <unk> study of our novel PK Activator AG 946 in lower risk Mds.
This progress reflects our operational excellence in clinical development.
And investigators' enthusiasm for the potential of PK activation in these indications.
Just on this progress we continue to expect two readouts from the energized and energize T Phase III studies in Dallas EMEA next year.
And we pulled forward the expected timing of the topline results for the Phase Iia study in lower risk Mds to the end of this year.
Turning to our commercial business, we're encouraged to see that the consistent and compelling efficacy.
Observed in the clinical trial experience as continued to translate to persistency on therapy, among adults living with PK deficiency in the real world.
As we continue to maximize the opportunity in the current launch in PK deficiency.
We're building the capabilities needed to fully realize the potential of anticipated future launches in thalassemia sickle cell disease and lower risk Mds.
Scott will provide a detailed update on our commercial performance in just a few minutes.
As Youll hear from Cecilia, we ended the second quarter with a cash position of nearly $950 million on the balance sheet.
One brief reminder, as part of the divestiture of our oncology business to Serbia in 2021, we retain the rights to a potential $200 million milestone upon FDA approval or aside nib.
And royalties on potential U S net sales.
We were encouraged by the results of <unk> Phase III trial, and we look forward to tracking next steps.
We're expecting a number of additional milestones by the end of the year, including enrolling more than half of the patients in the phase III activate kits and activate <unk> T studies of <unk> in pediatric PK deficiency.
Filing the IND for our <unk> stabilizer for the treatment of PKU and the newly added milestone the data readout from the Phase Iia study of AG 946 in lower risk Mds.
We're very enthusiastic about the clinical development momentum, we're building and look forward to anticipated readouts from the phase III studies submitted tape out in thalassemia in 2024, and Readouts from the phase III studies of <unk> in sickle cell disease in pediatric PK deficiency in 2025.
With that I'll now turn the call over to Sarah.
Thanks, Brian .
Logan response were accompanied by improvements in markers of hemolysis, an urgent voices as well as the medical reductions in the end of life, Rachel <unk> being crisis before it gets 12 week study.
Specifically patients in the placebo arm experienced an annualized rate of <unk> pain prices of 1.71 compare 2.83 in the 50 milligram arm and 0.51 in the 100 milligram treatment arm.
The safety profile for myself without observed in this study was generally consistent with previously report the data for me to talk about in other studies of sickle cell disease, and utter hemolytic anemia, and there were no adverse events, leading to discontinuation in any study arm.
And finally of the 79 stations enrolled in the study 73 continued into phase two open label extension period.
These data further underscore the potential for me to talk about to address the high unmet need of patients with sickle cell disease by delivering a novel oral treatment options at both improves anemia and reduce it <unk>.
We are excited to present, a full analysis of the face to data and half submits as an abstract for ash later this year.
Based on continuous analysis of the compelling data served at both doses in recent weeks, we have selected the 100 milligram dose for phase three.
As a reminder, the phase III portion of rise up will include a 52 week placebo controlled period in which 198 patients will be randomized to two wants to eat or <unk> or a placebo twice daily.
The primary influence our hemoglobin response, an annualized rate of sickle cell being practice and we look forward to enrolling the first patient in the phase three portion of the riots study in the fourth quarter of this year.
Turning to our broader development pipeline, we have been very pleased with the pace of enrollment Crawford programs in the first half of the year we.
We have completed enrollment in bulk phase III studies of Dr Pepper, and <unk>, including energize, which enrolled patients who are not regularly transfused with a primary endpoint of hemoglobin response, and energize T, which enrolled patients who are regularly transfused with a primary end point of transfusion reduction response.
Together. These studies will deliver data relevant to the entire thalassemia population and does allow us to evaluate potential mixed up if I were to become the first oral therapy to improve hemolytic anemia, and ineffective erythropoiesis across all thalassemia subtypes.
We look forward to the <unk> of the <unk> study in the first half of next year and the read out of energy Iced tea in the second half of next year.
We were also pleased to complete enrollment in the face to a study of 80 946 in lower risk M. D. S disorder. Several months ahead of schedule.
80, 946 is a novelty get activator that has the potential to strengthen our P. K activate a franchise.
We now expect offline results from the phase two a study of 80 946 am lower risk M. D. S. By the end of this year, making this our next clinical data read out.
As a brief reminder, the primary objective of this study is to establish proof of concept for 80, 946, and participants with lower risk M. D. S by measuring the following primary endpoints hemoglobin response defined as an increase of 1.5 grams per deciliter or more from baseline in the average hemoglobin concentration from <unk>.
Eight three weeks 16, and transfusion independence to find that transfusions 348 or more consecutive weeks during the study for participants with low transfusion Burton only.
In parallel we continue to advance the phase III activate Gibson activate gets these studies of my stuff is up in pediatric decayed efficiency and progress towards the filing of the I M. D for our small molecule ph stabilizer to directly address the underlying cause and you'll get an <unk>.
Finally, I Brian mentioned, we are pleased with the agreement that we announced this morning to get a <unk> a pioneer in RNA I therapeutics with a best in class platform.
Oh, They said Tamia Viera is a rare hematologic disease that is well aligned with our internal expertise.
T V. The cats drive by excessive production of Red blood cells, which leads to increased blood volume and viscosity and can result in from both the cardiovascular events and <unk>.
The S R and a development candidates <unk> six a key driver of Red blood cell production.
None of them for six increases hip sudden and reduces red blood cell production Wheeler.
We look forward to initiating IMD, enabling studies later this year and leveraging our deep expertise in rare hematology S. Progress this program to work the clinic.
But that I will not during the call over to spit up.
Thank you Sarah.
A lot of commercial organization remains focused on maximizing deer Park do anything in the Koran launch in big a deficiency by executing on our strategy across all phases of the patient journey.
The capabilities, we're building today on disease awareness and education access and initiation and adherence and persistency will also serve as a foundation to fully realize the potential of M. P. C baby's future launches into lithemia sickle cell disease and low risk.
M D S.
I want a market research data continue to indicate that nearly 100% of our target healthcare providers are likely to recommend final kind. So they are adult patients with became efficiency.
But getting nature.
Drivers of these recommendations include <unk> in T mobile will be level reduction in transfusion frequency and a positive impact on long term disease complications.
So that's what I understand the three human experience on final kind, we recently come duct taped interviews with a sample of patients or their caregivers.
Most patients reported positive experiences, including improvement in hemoglobin N in energy level reduction in fatigue and decrees <unk>.
Important he these feedback from both patients inclination is consistent with the strong proceed currency of threes menus, we observed in the real world after that initial Bea reauthorization.
Moreover, this continuation remain low and the reauthorization have not been a barrier.
In the second quarter of 2023, we generated $6.7 million in that violent crimes revenue at once you're pretending craze over Q1 ear.
<unk> of 147 patients have now completed a prescription enrollment form or B E S, including 20 in the second quarter of 2023, a 16% increase versus the first quarter of 2023.
We've had translated into net 99 patients on therapy, and 11% increase over last quarter.
Based on some therapy continues to stem from a growing and the reverse provider base of 130 physician.
Represents a bronco demographic and disease money, but based on the range that it's gonna assistant with a doubt b J D. The efficiency population.
Given the older around nature of these disease, we continued to expect slow and steady uptake.
In the early stages, we remain focused on in Densifying provide they're likely to three adult patients with big inefficiency.
Our efforts center on utilizing data analytics to improve physician targeting as we believe there are meaningful number of potential prescribers, who may have a valid spaghetti patients under management.
We also continued to improve efficiency and inbox educating treating physicians and in stealing a sense of urgency regarding the benefits of diagnosis and appropriate treatment.
By doing so well how to maximize the potential of the current launch and lay the foundation for potential launches in meaningfully a larger patient populations.
The first of this potential launches is in 2025 into Lithemia, where approximately 60% of the lithemia patients in the U S do not have a <unk> treatment option.
This next slide you just read the breakdown of the Lithemia subtypes in the U S, including Alpha and beta Lithemia and transfusion independent and non transfusion depend on <unk>.
Importantly meet the fever has the potential to become the first oral therapy Dream Poodle hemolytic anemia and in in fact, <unk>. It's Ah called the full range of the Leucemia patients.
Even the relative prevalence in competitive defenses across on the female subtypes, we look forward to implementing a big for for a bus commercials strategy that leverage capabilities gained from the current launch.
Together.
A full range of color scheme. Your patience is comprised of approximately 18 to 23000 patients in the U S N E five and.
In a meaningful addressable market in additional geography's, such as the Gulf Council countries or G. C C where in the prevalence is approximately 70000 patients.
The prevalence of sickle cell disease is also concentrated in the U S. N G C C a region.
[noise] overlap into like geography provides an opportunity for us to leverage our commercial efforts Intel asemia, so accelerates and support the future launch <unk> disease.
<unk> I don't know during the call over to Cecilia.
Thanks to better our second quarter of 2023 financial results can be found in the press release, we should this morning and more details will be included in our 10-Q, which would be five later today.
I'd like to take a moment to provide some context and highlight a few points.
Second quarter of 2023, net pirate fan revenue was $6.7 million, an increase of $1.1 million compared to Q1 2023.
A fire can I get the first therapy for this ultra rare a Dutch P. K D patient population, we continue to gather data and insights on the launch trajectory and would therefore not be providing guidance at this time, but we continue to expect a slow and steady trajectory to peek.
Consistent with other <unk> launches broke janette is expected to be in the 10% to 20% range on an annual basis.
Cost of sales for the quarter was $1.1 million.
Moving to expenses on the balance sheet.
Hi, Randy expenses was $68.9 million for the second quarter, a decrease of $5.6 million compared to the second quarter of 2022.
This decrease was primarily driven by a decreasing workforce related expenses as a result of reduced headcount related to the evolution of a research organization in 2022.
SG&A expenses were $34 million for the second quarter, an increase of $2.1 million compared to the second quarter of 2022 that was primarily driven by an increase in stock based compensation expense.
As a reminder to silver royalty has ceased given the faith of our rights to 5% royalty. Some U S. Net sales are available to safeguard in October 2022.
And that's part of it is the best picture of our quality business to Serbia, we retain rights to a potential 200 million dollar milestone upon F. D. A approval of horrified and 15% royalties unpretentious U S <unk>.
We ended the quarter with cash cash equivalents and marketable securities of approximately $947 million.
We expect that this patent together with anticipated product revenue interest income and the potential for a sudden milestone will enable the company to fund our operating expenses and capital expenditures to several vacuum, creating might've stones and at least into 2026.
This guidance does not include cash inflows from potential royalties from <unk> commercializing, we tip, even outside of the U S for the one almost partnerships or other potential strategic business a financial agreement.
We are excited about a potential <unk> lawn chair and continued to a big fan of 200 to 225 and Magenta R X P. K T in the U S and $1 billion for worldwide revenues for P. K T and set a female combined.
We remained focused on creating shareholder value, including by proactively managing a cough space and deployment disciplined gotcha location approach as we prepare to support the potential additional dollar tip of Palestine and continue to make strategic investments to advance and grow our pipeline all of which in fact, our timing too.
Good evening.
Following the significant momentum created the execution of our development plan and as we approach additional upcoming potentially value, creating much depends on the horizon I am confident that are strong balance sheet will enable us to execute from a position of strength to continue to look for ways to create shareholder value.
I will now turn the call back over to Brian for his closing remarks.
Thanks for <unk>. This was a tremendous quarter at all Jews, we announced positive phase two data and sickle cell disease completed enrollment in three clinical studies.
In licensed a compelling external program that has the potential to transform the course of a rare hematologic disease with profound unmet need.
And we continue to strengthen our commercial capabilities to support future launches.
The data we generally we continue to generate a crossword industry, leading pipeline of P. K aren't debaters remain consistent and compelling and we continue to make meaningful progress towards our vision <unk>, which is to develop an established hematology franchise with approval spending three hemolytic anemia is.
And an expanded portfolio fuelled by business development and advancement over internal pipeline there, there's a line with our core expertise in rare disease.
There's always will continue to strive to be responsible stewards over balance sheet and evaluate meaningful opportunities for value creation.
Finally, I'd like to thank all of our employees for their hard work and dedication to our mission of transforming the lives of patients living with rare diseases, and all of our partners, including the physicians patients caregivers and participants in our clinical development programs.
With that we'll open the call for questions.
So it's a question. Please <unk> have your telephone and wait for it to be announced to withdraw your question. Please post or one word again please <unk>.
One moment for first question.
My first question is whose line of Greg Harrison from Bank of America. Your line is open.
Hey, good morning, Thanks for taking my question. Thank.
Congrats on all the updates.
Start off with.
Curious what factors.
Lead you to the decision to license the T V treatment from my loan and is this a model for future B D. In terms of the development stage and having the potential where you can add your own expertise and value and then would you expect.
But to be broadly applicable across P V patients or or would it be more focused on the specific patient population.
Sure well good morning, Gregory no. Thanks, a lot for the question yet it's been a great quarter and I'm happy to provide some comments around the deal that we announced this morning. This is.
Probably just like give me a bureau is a large market. We believe it is ripe for disruption in gross.
And as I noted in my comments.
If you just think about the standard of care it's lobotomy.
To us that's just a great opportunity to do what we do best which is reinvent the way that these rare diseases are treated and bring potential disease modifying therapies to these patients. So we're really excited about this particular temper six acid from Illinois.
<unk> really well with our discipline BD criteria.
Builds on our core capabilities, and that's across or scientific expertise or development capabilities or commercial capabilities and if you think about the the BB criteria that we've talked about so many times it checks all the boxes. So this is a rare disease.
It is transformational potential for patients we see an opportunity for early Derisking, which we've talked about a lot as a sweet spot for us. We believe there's a clear regulatory pathways and ultimately this will be value, creating is is our strong belief.
Because it comes from the <unk> World clash or on a I platform. We also have strong conviction in the probability of success from the data generated today.
So we're excited about this I guess to your question of where we go from here for clinical development I think they don't.
Sure if she wants to make comments, but it's a little too early for us to define the target product profile, specifically, we have many options, we'll be looking at the efficacy dimensions speed of action for the product itself for patients safety profile and of course convenience and all of those were in.
Place, but given the state of development, we're just gonna pause and bring your acid in really look at it deeply for where we can put our development expertise in motion and then will provide updates accordingly, and then the last thing I'll just say to your point about is this a theme of the types of bvb deals.
<unk> routine optionality for a range of different beady deals. This one in particular really as I mentioned checked all the boxes and so we were eager to bring it in but we'll we'll routine or discipline BD criteria going forward.
Is there anything you wanted to add.
Only to say that you know.
Very excited about this we are of course once we move forward with the program and have more details on our clinical development program, we will always be shooting to deliver value for patients and making meaningful change with the development program and you can expect execution on the program as well and we were excited to do that.
Thanks, Greg Greg that's helpful. Thanks.
One moment for next question.
Our next question will come from the line, Okay. Gregory <unk> from our received capital mortgage your line is open.
Great Good morning, Brian and team of congrats on the progress and thanks for taking my questions.
Maybe just a couple of quick ones for me just uhm.
Following up on me and my friends are my own that that might be helpful to hear your thoughts and Sarah just just on the preclinical data today certainly.
Others exploring manipulation of temper six and just curious how you think of this as it can potentially differentiator or sets up well when you as the survey the available data today, and then and then secondly, great to see the the dose selection for the for the phase three of <unk>. Just curious just the the rationale of you looked at the body of data.
Over the last several weeks since since the top line and and made this step for the hundred Meg. Thanks, so much and congrats again.
Thanks, a lot Greg I'm gonna send this over to Sarah then for both questions.
Yeah. Thank you so around the preclinical data. So we are you know we'd have demonstrated in vivo proof of concept in <unk> in non human primates with a safety.
Profile that was very favorable and very good knock down the target observed. So we feel very confident about that and as I mentioned earlier were very eager to.
To get going on the IMD, enabling studies and and the C. D. P S.
And and provide more detail S on that as we progress with that I think for US. Yes. There are others that are also looking at the start of it to Brian . This is a very big market. There is going to be differentiation around efficacy safety mode of administration and off the frequency of my administration. So we believe that our C. D T.
We'll be able to deliver on what the market needs and again I'm very excited to get going on this for your second question around the phase III dose selections for sickle cell disease.
Obviously, we are very excited we're very excited about the date of the phase two of them are very fortunate to have both of those.
She'll benefit risk profile that was favorable we have pre specified criteria in the protocol and the team have time to you know look at all of the data. So we we followed that framework and selected the 100 milligrams. We have not disclosed further details on the data you know we mentioned that we have submitted the abstract to ash.
There are now a waiting the outcome of that and then we'll we're hopeful to be able to provide all that detail at that conference and Greg maybe I'll just tag onto and say I always love to take the opportunity to speak with pride about Sarah and her entire R&D team. This is just a great example of the.
The excellence operationally that we have on the efficiency of the design of the rise up phase two three trial and the fact that we were prepared for both doses. We were thrilled to see that both doses were affected but now that we have the 100 milligram dose. The fact that we can proceed in the fourth quarter with the first patient dose in the <unk>.
While I think is again, just a mark of excellence and real point of pride for the organization.
Absolutely that thanks, Bryan and thank you Sarah.
You bet. Thanks, a lot.
One moment for next question.
Our next question is <unk> W. <unk> Kelly <unk>.
That's open.
Hi, Thanks for taking my question and congrats on the corner I'm from work just to hold on we're just curious what do you define or how do you define success M. E. N D. S trial I know that there are other several <unk> do you.
Those competitive or do you see the blood the opportunities maybe to build combinations with itchy and then I have a follow up.
Yep. Thanks did you.
I will just start by saying as is the case with all the D. C area is that we're focused on we have a very different mechanism of action with part of a big Chinese activation and a bit of course.
The question for low risk and yes. This is with our other PK activator E. G 946, and I know Sarah will be eager to talk about what we're all waiting in terms of the fees to a data.
So yeah. Thanks for the questions I think for the Mds program, one I want to highlight here again that the team has done an amazing job on enrollment of that phase two eight program I think it also highlights the <unk> of the investigators afford this mechanism of action.
For Mds patients. We are we have not declared our framework four mdf's to define success, but it is a face to a so we are indeed looking for signal of efficacy and favorable safety and then our setup. If that's if that's their than we are set up to move fast into a face too.
<unk>, which we will do a proper dose finding it is indeed, a market that is evolving and it's <unk> I think it speaks to that this is also a market that is right for a change and we do believe that with this very differentiated mechanism of action, which is is not being currently studied by are are not a fast fast by us.
That'd be really are set up for success, there as well and then I think what cannot be underestimated is that ours is also immoral therapy, which provides the convenience of used for a patient population that is typically an older patient populations.
That that's really helpful. And then just a quick question on the sickle cell program could you remind us on if you've just close the <unk> for the phase three trial and then have you discuss 100 milligram dose with the F. D. A or is that just mostly based on your internal discussion.
Analysis of the data.
So for the sickle cell disease program to 100 milligram dose for right now deceased internal framework that we have applied in all of the data that we have we are very excited about interface to meeting with the F. D. A we always look forward to our.
Constructive dialogue that we have together and you know it's it's always.
It's always great because I do feel like we are all one thing the same thing right across all of our programs. We are really is shooting to deliver.
Medications that have a favorable benefit to risk and can provide change to patients. So we're always I'm very pleased with the constructive dialogue that we have.
In regards to the pouring assumption. So obviously did phase III much bigger sample size than the than the fees too we for.
For the so there's two endpoints there for the primary endpoint the hemoglobin input to use leverage our internal data for all of the statistical.
Underpinnings send in for the viewer sees obviously, we have been looking at other programs to make following assumptions there and are looking to making meaningful change on <unk>.
Thank you.
One moment for next question.
Our next question comes from the line of solving Richter for Goldman Sachs. Your line is open.
Good morning. This is auto me down for solving just one question from us on the licensing agreement with on the island given the novel T. S. D. S. I R. A named modality for US. So it gives you confidence in the clinical development process. Thank you.
So thanks for the question. So I think it gives us confidence because we do have internal expertise that have worked on this modality, but also many other modalities. So it's not like well. This is a new compound or modality for all shows it's not a new compounds.
Four objects employees.
And so we're looking forward to putting that internal knowledge to work and I do think in the context of endpoints patient populations all of that it's strength into our wheelhouse of he mythology drug development and so we are looking forward to putting those skills to work there as well.
Thank you.
One moment for next question.
Alright next question will come from the line of Andy Barons from Lyric Your line is open.
Alright. Thanks, that's all you'll be all my questions are also on my M. B S program.
If you could give us some overview of how you see the <unk> <unk> <unk> <unk>.
<unk> is actually doing pretty well I'm having medications.
Sales over 700 million in the snow population.
You give us the pleasure.
Logical rationale for the Pizza you request an M B S.
As well as how're you hear potentially be used relative to the depot agents and lastly, I know it's early but how do you think you should you people agency magazines hypothetically a parts place in power.
Yep, So Andy Thanks, a lot for the question I'll just start by reinforcing what you just said we find it a very attractive opportunities based on the current incumbents in the progress that they're making the patient population is we had on one of our slides we.
We identify as 75 to 80000 patients across the U S are you five it is another disease that is ripe for disruption and coming in with BK activation of very different mechanism of action. It is Sarah is known to do a couple of times already on top of all that as an oral small molecule.
We think has significant potential to be differentiated but of course, we're going to be guided by the two way data you would've comment further Sarah well just on the part of the rationale why we believe a big activation may work in M. D. S. It's truly there are similarities between Mdf's in Dallas see me at that and we obviously.
Have not progressed and fellow senior quite well so that is one piece. It also the <unk>.
Glycolytic pathway is impacted in Mds patients at multiple levels, which we believe we can influence as well there is hexokinase.
They'd kindness ratio disruptions. There is also a fact that and you have to patients often have an acquired PK deficiency. So there's a multitude of reasons why we believe speak activation may provide benefit.
We have published data on this at the <unk> at Ash and hopefully more to come at Ash. So we are very excited about you know the.
The evidence that we continue to build in this disease.
Danielle just one other comment I'll add is our team goes deep across many potential therapeutic areas that were focused on and sitting with us today will be upset in Illinois, <unk> commercial officer, who actually.
Has been very involved in this space and so it's early of course, we're looking at phase II data, but beyond that said it will be able to add a lot of insight and expertise and not just the commercial profile of the product, but also as you asked about pricing down the line and how we maximize the value creation.
We feel like we're really well prepared and cannot wait for this upcoming milestone by your own.
Alright, thanks very much.
You bet. Thank you.
Mmm moment for next question.
Our next question comes from the <unk> of J P. Morgan.
[noise] Hi, Brian and team. Good morning. Thank you for taking my question I thought I'd ask a commercial lines today for <unk> patients on drug just trying to get a sense of how you think about <unk> trajectory of new patient at here it sounds like the drop off.
It's pretty low.
And do you think this run rate of 10 ourselves sequential on that patients ads like you've seen in the last couple of quarters in the right way to think about the launch going forward versus something maybe more accelerated given some of the initiatives on analytics et cetera.
Thanks, so much.
Yeah. Thanks, Thanks us so spell it is thrilled to have a question about commercial so I'm gonna, let her common hi good.
Good morning, Sir is it certainly convenient to make progress on the lounge. Most importantly, we <unk>, we continued to see the strength of the final kinds profile and around the world, especially when it comes to persist and see the positive provider feedback and the parents support.
At the same time, we also known as Big a D is an ultra rare disease and as we said Tyler opening remarks, we would expect for convenience received slow studied uptake.
Who I expect to see some variability cause our all of our quarter or Bob's our efforts continue to be laser focused on intensifying the rights providers provide those who are likely to have patience with vacated efficiency and patients who are appropriate for iron kind either today from especially in the future.
And basically the way our our efforts in data and analytics continue to support our team as we continue to execute in the field very importantly, all of the capabilities of our building today surfers are very good foundation for US as we continue to look into the future and potential future indications.
<unk> for a violent crime. So it just doesn't seem as well.
Yeah, I mean, the only thing I'll just add to US is that this is a very unique kind of launch it's pretty far on the ultra rare spectrum and we're building warnings from it but I I would look at it is slow and steady growth and we always caution to just be a little bit careful over quarter over quarter, because it'll be won't be.
For quite some time, but we are very proud of the progress that instead of the team continue to make.
Okay, and if I could just squeeze in a commercial follow up there.
You know given these dynamics how do you think about providing longer term <unk> is that something you feel like you may be in a position to do <unk>.
Kind of exit 2023 and get into next year.
Sure. Thanks, Cecilia Yeah, you can take that so you know as we mentioned before and it says Ah.
<unk> Ah therapy, and we continue to plan data and insights at this point, we're not gonna give guidance there would come a point when you continue to evaluate when it's the right time to do that.
Yeah, we do a rancor point as we talked about peak sales $200 million to $225 million in the U S.
That's where we feel confident we we want to get more quarters under our belt until we give guidance on the revenue for P. K D and of course, the bigger picture for US anyway is that we're approaching.
The read out of the palace EMEA data, that's a meaningfully larger launch that has potential in 2025, and we may be in a scenario restrict to look at the collective guidance of multiple launches.
Great. Thanks, so much for taking my question sure. Thanks Us.
One moment for the next question.
Mmm I quit our next question comes from Daniel <unk> <unk> <unk>.
Hi, good morning. Thank you so much for the question I I also have a a question on the N license asset. So I know there are also antibodies targeting temporary six and development I was wondering if you could elaborate on why you think adds irna, maybe a superior modality and that's N vacation.
Ah more convenient dosing regimen or do you think there might be an advocacy advantage as well. Thank you.
Well, we've been so thanks, a lot Daniel further question. We think there are as I noted earlier potentially multiple opportunities for differentiation temper six we feel is now a well established pathway I mean, very simply the 90% knockdown of temper six as an uplift.
Effect on <unk>, which decreases iron, which ultimately lowers the red blood cell production that that thread through that mechanism, we feel through all of our diligence very confident in and as I said I think there will be opportunities for efficacy see.
50 of course will be that's one when you look at some of the current opportunities therapeutically for patients there's plenty of room for improvement and and then it could wind up also being a meaningful convenience opportunity because S. I R and a classically and particularly from Yale Niland platform.
<unk> has proven that that is a mechanism that has delivered benefit to patients with lower interval of treatment, but we're gonna look at all of that it's very early and we will start off IMD, enabling studies. This year and then we'll report out our progress accordingly.
Thanks, so much.
Sure. Thank you.
Q, we're showing no additional questions <unk> turn the call back to Brian Golf refined Oh come in.
Alright, well. Thank you all very much for participating in today's call and of course for your continued interest in <unk> as you heard. This morning, we are really energized by the tremendous progress we continue to make across our portfolio. We are confident in our potential deliver significant long term value for both patients and shareholders. So.
Thank you very much again, and we look forward to speaking with you soon.
This includes today's conference call. Thank you for participating give me no disconnect everyone have a great day.
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