Q3 2023 Geron Corp Earnings Call
[music].
Okay.
Good morning, My name is Audrey and I will be your conference operator today.
At this time I would like to welcome everyone to the Geron Corporation third quarter 2023 earnings conference call.
Today's conference is being recorded all.
All lines have been placed on mute to prevent any background noise. After the speakers' remarks, there will be a question and answer session. If you would like to ask a question. During this time simply press the star key followed by the number one on your telephone keypad.
If you would like to withdraw your question Press Star one again.
At this time I would like to turn the conference over to Erin Feingold VP of Investor Relations and corporate Communications. Please go ahead.
Okay.
Good morning, everyone. Welcome to the Geron Corporation third quarter 2023 earnings Conference call I Am Erin Finegold Jones, Vice President of Investor Relations and corporate communications.
I'm joined today by several members of <unk> management team, Dr. John Scarlett, Chairman and Chief Executive Officer, Michelle Robertson Executive Vice President and Chief Financial Officer Doctor Base Dollar Executive Vice President and Chief Medical Officer, and Neil Kapoor Executive Vice President of corporate strategy.
And Chief commercial officer, and Dr. Andrew <unk> Executive Vice President and Chief operating Officer before we begin. Please note that during the course of this presentation and question and answer session. We will be making forward looking statements regarding future events performance plans expectations and other projections, including.
Those relating to the therapeutic potential and potential regulatory approval, then that helps that anticipated clinical and commercial events unrelated timeline, the sufficiency of Jared on financial resources and other statements that are not historical fact.
Actual events or results could differ materially therefore, I refer you to the discussion under the heading risk factors in <unk> quarterly report on Form 10-Q for the quarter ended September 32023, which identifies important factors that could cause actual results to differ materially from those contained in the forward looking Steve.
[noise] Geron undertakes no duty or obligation to update our forward looking statements.
That I will turn the call, Alberta Chit chat.
Thanks, Aaron Good morning, everyone. Thanks for joining us today.
This quarter, we continued to make important progress and build momentum along our planned path to develop and commercialize <unk> our first in class former actually Peter.
This path, we believe represents a great opportunity for both near and longer term value creation.
2023 has been a signal year for us the <unk> NDA for the treatment of transfusion dependent anemia in patients with lower risk Mds, we submitted and subsequently accepted for FDA review in August the FDA assigned it could do for action date of June 16th of 2024.
This was followed by validation of the MAA for the same indication in September of this year now that our MAA is under review we expect the earliest potential approval could occur in late 2024 with a European launch potentially in 2025, we are continuing to evaluate our strategic options.
<unk> self commercialization or partnering and expect to be able to provide an update later in 2024.
If approved we believe the <unk> commercial opportunity in this indication is both differentiated and compelling.
For three reasons.
First <unk> has been shown to be highly effective in several key patient subgroups, where today's available treatments did not satisfactorily address the needs of patients with this disease.
These include Rs negative patients patients with high transfusion burdens and patients with high serum Epo levels.
These key clinical attributes of <unk> had been further reinforced by the Ash abstracts published this morning.
New analyses using data from the phase III emerge trial in transfusion dependent lower risk Mds continue to show a significant durability and breath of transfusion independence across subgroups, including patients whose needs are not being met by current treatments.
Second there is a very large market opportunity for Intelsat and lower risk Mds patients with transfusion dependent anemia, which we estimate represents a total addressable market or Tam of approximately $3 $5 billion by 2033 in the U S.
And the EU for as well as the U K.
And third this is a market that seem low competitive intensity and relatively little innovation in the last decade.
Despite the recently approved <unk> label expansion.
Just last month, the national comprehensive cancer network or <unk> published a revised version of the Mds guidelines.
Which still point to limited treatment options available the hematologist as they manage their transfusion dependent lower risk Mds patients.
Moving beyond lower risk Mds, another key component and Gerrans path to value creation is an expected interim analysis emerge which is a phase III study in jakafi.
Lapsed in refractory MF. That's currently anticipated in the first half of 2025.
Impact MF is the first and the only phase III trial with overall survival as a primary endpoint.
If the expected interim analysis in 2025 for the final analysis expected in 2026, if positive these data could be transformational for patients with relapsed refractory MF, which also represents an underserved very substantial market opportunity with an estimated $3 $5 billion Tam by <unk>.
<unk> thousand 33.
Together, our lead indications represent a potential tam of $7 billion in 2033.
And beyond that we continue to investigate the potential of Intelsat and other hematologic malignancy indications and combination trials that could build additional value for the company.
Given that opportunity we've been preparing at an enterprise level for transitioning to a commercial company for several years.
This has enabled us to efficiently hire and onboard our commercial and medical affairs leadership teams scale, our internal systems and operations and build the competencies needed to succeed as a commercial company.
Neil will see further just the commercial opportunity later during this call.
We also have a strong cash position of approximately $382 million at the end of the quarter, which based on our current plans and expected available resources.
We expect will enable us to fund a potential successful launch in transfusion dependent lower risk Mds in the U S.
And fund our planned operations through the end of the third quarter of 2025.
Finally, we have outstanding individuals to lead our organization through this transformation from a development stage to a commercial company. These individuals now include Michelle Robertson, who we recently appointed CFO. Following the retirement of our long time CFO Olivia Bloom.
We're thrilled to have Michelle onboard at this very important moment in our history.
Michelle brings with her over 30 years of financial and commercial operations experience. Most recently as CFO at a tough medicine and before that as CFO of momentum before its acquisition by J&J. She.
She also spent over 13 years and the financing commercial operations groups at Genzyme.
Her deep command, our financial operations her experience with managing the financial and organizational needs of a biotech company preparing to potentially launch its first commercial product <unk>.
And her prior experience with investors analysts investors and bankers as well as their hands on experiences with commercial launches in the past, we will all be extremely valuable to our organization going forward.
With that I'll turn the call over to Fay for our regulatory and clinical update.
Thanks, Chip and good morning to everyone on the call as chip mentioned to be accomplished critical regulatory milestones this quarter with the acceptance of our regulatory filings in the U S and EU for review of at Mattel Stat for the treatment of transfusion dependent anemia in patients with lower risk Mds, who have failed to respond.
<unk> response to or are ineligible for Esa use.
The FDA assigned the Paducah action date of June 16, 2024.
The FDA also informed us that they are planning to hold an advisory Committee meeting we have no further details at this time about a potential date or agenda for this meeting.
Best practice in our industry, we are working with a consultancy group who has expertise in advisory committees to complement our deep in house regulatory experience and we expect to be highly prepared.
We believe we have an important medicine today, Mattel stat, and we look forward to the opportunity to discuss it with experts.
As chip also mentioned we are pleased that the ash abstract published this morning continued to demonstrate what we believe are the unique and differentiated qualities of Intelsat and lower risk Mds scene in the emerge phase III study.
Including the breath of effect across Mds subgroups, and the unprecedented durability of transfusion independence.
I will now provide a brief overview of the abstracts.
Please note that we issued a press release this morning at nine a M. Eastern time, which describes the abstracts in more detail and that all abstracts are available on the ash website.
The first abstract was accepted for an oral presentation and characterize as Ti responses, and then Intelsat and placebo treated patients using various risk classification systems.
This subgroup analysis demonstrated that <unk> consistently had higher Ti response rates and placebo across different risk subgroups irrespective of risk classification system.
Using the most recent and precise.
SaaS and classification system, which takes into account the prognostic effect of molecular mutations.
Over 13% of Etfs, low or intermediate one risk patients enrolled in the merge phase III were upstaged have moderate high high or very high risk Mds.
Notably, despite having mds that would be classified as higher risk under Ips and these patients benefited from metals that treatment compared to placebo.
For example, as you can see in the last few rows of the table on the slide.
Among the M Ips as high very high group, the eight week RBC Ti rate was 40% from Intelsat and zero for placebo.
This shows that Ti within metals that treatment is achievable regardless of risk classification.
The next abstract was accepted for poster presentation and examines the efficacy of <unk> versus placebo.
Underlying mutations associated with Mds.
Results show consistent Ti responses in <unk> treated patients across different molecularly defined subgroups.
Regardless of the presence of mutations associated with poor prognosis, including ASX, all one or T. P 53, or the number of mutations.
For example, the eight week Ti rate for patients, who had three or more mutations a group with known poor outcomes with 56% with him Intelsat and 14.
Per cent with placebo.
These results complement the data I just described for the Ips S. M classification can we further believe support telomerase inhibition as a mechanism that can address the many different clones and causes of lower risk Mds.
The next abstract was also accepted for poster presentation and focuses on the patients who achieved.
<unk>, one year Ti within the Telus that treatment.
Unprecedented clinical outcome in lower risk Mds, which Ricky recapitulate, what we saw in emerge phase two.
Among the nearly 20% of it in the Tulsa treated patients who achieved a one year Ti. The median duration of Ti was over two years and the median increase in hemoglobin with over five grams per deciliter.
Of the 18, Intelsat treated one year Ti responders with mutation data available nearly three quarters achieved greater than 50% reduction in variant allele frequency and.
And importantly, nearly half experienced reduction of Mds associated mutations.
Detectable variant allele frequency.
We believe these cases of long term uninterrupted ti accompanied by robust increases in hemoglobin and the elimination of MBS associated mutations suggests the potential within mattel's back to have disease modifying activity.
The next abstract accepted for poster presentation summarizes results of Geron sponsored study in collaboration with the Moffitt Cancer Center.
As population level analysis was based on a large U S health care insurance claims database that included over 5000 patients with lower risk Mds.
This real World data analysis show durable transfusion independence. After second line treatment is associated with improved survival.
The median overall survival from the start of second line therapy was $23 four months overall and $37 nine months versus nine three months, among 16 week Ti responders versus non responders.
This difference in OS with clinically and statistically significant with a P value lessons airplanes 001.
For transfusion dependent patients achievement of Ti with second line treatment is associated with improved Alas supporting the clinical benefit of Ti and underscoring the importance of Ti as a clinical trial primary endpoint.
We look forward to all of these presentations and discussions in San Diego. This December.
Turning now to our phase III trial with Intelsat in relapsed refractory MF.
Enrollment is progressing and we anticipate completing 50% enrollment by the end of this year.
We continue to expect an interim analysis in the first half of 2025, which will occur when approximately 35% of the planned enrolled patients have died.
Our final analysis is expected in the first half of 2026 when over 70% of the planned enrolled patients have died.
We look forward to continuing to keep you updated on this important study.
In addition to our phase III programs. We are also evaluating <unk> in a phase one study as combination therapy with <unk> in patients with frontline myelofibrosis.
The study was informed by data from preclinical studies, describing that the sequential treatments or <unk>, followed by <unk> that has a selective inhibitory effect on malignant myelofibrosis stem cells.
Sparing normal hematopoietic stem cells.
Our main goal for improve en masse is to determine the safety profile of the combination regimen of <unk> inhibitor and the top of that.
In addition, we plan to explore any potential activity in the frontline MF disease setting.
Last month, the improve MF safety evaluation team or sat which is comprised of study investigators evaluated patient data from the first cohort of patients no dose limiting.
<unk> toxicities were identified and made a unanimous decision to escalate to the second dose cohort.
We are very pleased with this progress and look forward to providing future updates.
With that I'll turn the call over to Neil for a commercial update aneel.
Thank you Phil and good morning, everyone.
We have made significant progress regarding our promotional efforts and this quarter.
We remain on track for launch readiness in the U S and <unk> CT and I'm looking forward to the opportunity to bring this important new option to patients as they fight their disease.
I'll start by highlighting the recent updates to the NCC and treatment guidelines.
And yes.
This follows the reason.
Naval expansion from the Cytosorb in the frontline space.
As you may be aware, the NCC guidelines, along with publication of results from randomized trials remain among the most important factors that influence clinically.
Their boxes and significantly important prescribing behavior.
This next slide with this simplified schematic effecting the revised <unk> guidelines.
On slide four the largest segment of the frontline Rs negative patients.
<unk> remained up therefore, Keith and corporations.
There also continues to be limited treatment options for patients with <unk>.
And that means greater than 500.
And participation in clinical trials is encouraged for those patients.
Given these revised guidelines.
Expect the front line lower risk Mds space will evolve towards the use of both Esa analyst that affect.
It is important to remember that the majority of the patients with symptomatic anemia.
Can you talk to David Esa is in the frontline setting and most of them fail treatment in approximately two years.
From our perspective these updated guidelines reflect a lack of effective new treatment options.
In particular for the artist negative risk Mds patients coupon secured 75% of the market and for those who stayed on repo levels greater than 500.
This is a need we believe <unk> can follow fully address and given the lack of effective treatment options, we expect and it does start to be adopted for these patients for the peak window lower risk Mds by the prescriber community.
We believe the Lotus MBS market is right for the new <unk>.
Hey, Dave and durable treatment that can be used broadly across MBA subtypes.
We expect this large attractive market opportunity and it does start across three main patient segments across both the U S. EU food and UK highlighted on the right hand side of this slide <unk>.
These segments include <unk>.
Client patients who are Esa ineligible.
Given the high baseline Epo levels.
Hey, Phil Rs positive patients.
There are limited treatment options, including the side effect in Hma's.
Lastly, there is also a very large segment of approximately 24000, Esa fail Rs negative patients who tend to have worse prognosis than Addis positive patients and then there is a significant need for durable treatment.
Together these three market segments represent a significant commercial opportunity for <unk>.
With a total addressable market of $1 $3 5 billion.
<unk> 33 in the U S and these key European markets.
This slide highlights the key attributes of <unk>, starting the phase III trial that resonates most strongly with community and academic Hematologists.
Our unprecedented ironwood phase III data that highlights totality of clinical benefit.
Steve Mento transfusion independence, regardless, levada subtypes and meaningful durability of response, along with the piano data that highlighted improvement in fatigue when treated with <unk>.
Highly with physicians and service critical Differentiators and form our value proposition.
We continue to work towards bringing all of these elements along with the real world analysis of the importance of achieving transfusion independence.
Bears and prescribers through appropriate channels to highlight the Msos Scott value proposition.
This next slide highlights key aspects of our market research with Hematologist from both the U S and key European markets.
First <unk> start first in class mechanism of action is seen as a key driver for future market adoption.
Together with the durability of response physicians view the opportunity for a new and novel mechanism is highly competitive.
Okay.
Hey, Mike I'll start is projected to be part of the standard of care across both Rs negative Rs positive patient subgroups.
Physicians expressed a clear preference to use them. It does start first quarter their frontline artist negative Esa ineligible patients.
Across all of those fiber prior treated patients.
And the ESA relapsed refractory patient segment physicians note that metal star demonstrates significant improvements in long term response.
That is the 24 week transfusion independence over available option, especially in the Rs negative patients.
Fourth for high transfusion burden patients relapsed and refractory lower risk Mds patients physicians believe Amit I'll start maybe a compelling option or with currently approved therapies.
This next slide highlights the results from the survey, we conducted post <unk> frontline combining data use.
This wasn't done with 50, plus practicing hematologist and the U S across both community and academic settings to understand how they would use and it does start if available as compared to other available therapies.
On the left hand side of this slide you can see that the responses indicate strong enthusiasm for them. It does start to be integrated across the three different patient segments and the notice Mds treatment landscape upon potential approval.
The graph on the right side shows responses further segmented for Odyssey positive <unk> negative patients within these subgroups.
Sponsors as you can see here indicate that they'll start is not only expected to be broadly adopted for the treatment of nordisk MBS, but that it is especially likely to become a new standard of care for second line Rs negative patients.
As a reminder, the oddest negative patient segment is approximately three times larger than the RF positive patient segment and treatment options for the oddest negative patients remain suboptimal.
Holistically, we believe that the qualitative market research on earlier.
And quantitative survey highlighted here are highly aligned with the updated end CCN documents. These.
These results emphasize the significant unmet treatment needs across the lower risk Mds patient population.
Therefore, we strongly believe that <unk> if approved can play a meaningful role in the treatment paradigm of Nordisk MBS moving forward.
Okay.
We believe that we are well positioned to execute against this tremendous potential commercial opportunity.
I have described in detail in the past the robust internal planning to prepare and it'll start the market in general as an organization. So that we can deliver a seamless customer experience to all stakeholders, while ensuring broad reimbursement for <unk>.
Okay.
With regards to the U S launch planning with Alpha <unk> date of June 16th 20 granted port in hand, we expect to be ready to launch upon potential FDA approval.
Significant progress on multiple fronts as highlighted on this slide.
We have been highly successful in attracting DP experienced talent regeneron with significant operation in the U S launch experiences.
Our non field facing commercial organization is now fully deployed and deeply engaged in bringing <unk> to the U S market.
We plan to hire.
In facing teams, including our sales force in quarter, one 'twenty for any port to ensure proper time for Onboarding and training.
We have already identified many talented and experienced individuals who are eager to join the team and are very much looking forward to welcoming this critical function, but yet on next year.
We are pleased with our progress to date and look forward to sharing updates in the future.
I will now turn the call over to Michele product financial update Michelle.
Thanks, Aneel and good morning, everyone.
As this is my first earnings call at Jan I wanted to start by saying, how very excited I am to be part of this amazing team and how privileged ICL to lead the finance function through this pivotal time for the company.
With regards to Q3 financials for detailed results. Please refer to the press release, we issued this morning, which is available on our website I will now review some highlights from the quarter.
At the end of Q3, our cash cash equivalents in marketable securities were $381 9 million. This balance includes approximately $28 3 million in proceeds from warrant exercises in the third quarter. There are approximately $2 5 million.
Standing and the potential proceeds from these warrant is $3 2 million.
R&D expenses for the three and nine months ended September 30 were $29 4 million to $92 1 million, respectively, compared to $24 6 million and $67 3 million for the same periods in 2022 expenses have increased year over year, primarily related to supporting our clinical trials in <unk>.
Phase III and impact EMS.
Both personnel and consulting costs increased port regulatory submissions and increased investment in manufacturing as we prepare for the potential commercialization in transfusion dependent lower risk Mds.
G&A expenses were $18 4 million and $47 7 million for the three and nine months ended 932023 compared to $15 6 million and $29 8 million for the same periods in 2022.
The increase in G&A expense is primarily attributed to head count and external expenses to support the commercial readiness activities.
At the end of September 30th the company had head count of 137 employees, which we project will grow to approximately 160 by the end of 2023.
Our projected full year 2023, GAAP operating expenses are expected to be between 200 and $210 million moving forward. We plan to provide only GAAP guidance for consistency with our SEC financial.
Based on our current operating plan and expectations regarding the timing of potential approval of our <unk> NDA that is currently under FDA review and subsequent potential U S. Commercial launch we believe that our current cash runway together with projected revenues from U S sales of Intelsat proceeds from the exercise.
Outstanding warrants and funding under our loan facility will be sufficient to support our operations through the end of the third quarter of 2025.
I will now turn the call back over to chip.
Thanks, very much Michelle.
Yeah.
With our strong emerge phase III data and with our regulatory submissions based on these data currently under review by both the FDA and EMA.
We believe there is a robust market opportunity in front of us in transfusion dependent lower risk Mds. In addition, we remain excited by our second phase III readout, and Jack I relapsed and refractory MF for which we expect an interim analysis in the first half of 2025 and a final analysis in the first half of 2026.
These programs represent important opportunities for the patients we serve and for our shareholders whose interests we represent.
We look forward to keeping you updated on our progress and we'll now open the lines for your questions operator.
Thank you at this time I would like to remind everyone in order to ask a question Press Star then the number one on your telephone keypad.
We will go first to Stephen Willey of Stifel.
Hey, good morning, guys.
Can you guys hear me okay.
Yes, we can.
Okay.
Yes. This is Tony on for Steve. Thank you for taking my question.
We have plenty of questions on our end Firstly can you guys gave us an additional color on the dose level, one basically what the dose level for those level was in the <unk> program F. <unk> MF study and maybe more color on the dose escalation scheme, where they get those.
Thing was higher or about the same dose level as low risk Mds and whether maybe like those being scheduled that would be helpful. And second can you guys talk a little bit about whether FDA granting of a broad label towards those parts of that in the first line patients.
<unk> to our perception of risk benefit of emitter, Scott and lastly are you guys planning on naturally is closing or announcing when the enrollment hit 50% target impact MF study that would be it. Thank you.
Great. Thank you very much this is chip.
No.
I'm going to invite Fay to make any comments that you can about the improve MF dosing and how that might relate to some of our other dosing.
For example, lower risk Mds or.
Hum.
<unk> single agent use.
Thanks, Chuck the dosing for the improve MF study is mostly based on this is a safety study. So it starts at a lower dose and then dose escalate to our highest dose that we used in Ms and dosing scheme is.
Publicly available on <unk> Dot Gov.
And I think in within the corporate deck.
Appendix the first dose level.
$4 seven milligrams per kilogram of Intelsat.
And the next.
60 milligram per kilogram.
Importantly, again.
Study is really to assess the safety of the combination therapy in <unk>.
Frontline setting.
Great.
The second question related.
Did you have any follow up questions.
Or shall we move on to the FDA.
Oh, sorry, the broad label commentary.
Okay.
Move on.
So the second question I'm going to invite Aneel to address I believe the question was did the assignment of a broad label to.
The lease pattern frontline use.
Use does that read in any way on.
From our perception of risk benefit for them. It helps that I believe I got that right.
Yes.
Sure.
Thank you for the question.
The frontline indication in the frontline population is very.
Very different from the population that's being staggered.
For them it does start to emerge.
Oh.
I suppose DSA relapsed refractory patients who are transfusion dependent.
So thats a very different population from the frontline commands, which looked at the front.
Yes, and I'll use patient population that are treated.
So as of now.
<unk> mentioned my feeling would be that it does not have a risk benefit or impact to our trial, but I wouldn't buy it.
Failure to provide her clinical judgment it is not.
Agree aneel.
The patient population studied in the command.
Has minimal if any overlap with any patient population within in March so I impact on on risk benefit.
Okay.
Legible or nonexistent in my opinion.
And then the third question was.
Do we plan on disclosing when 50% enrollment as yet.
Impact MF. The answer is yes, we do that has historically been our our practice.
All of the clinical studies, we've run for the last many years I'm not sure exactly the form that that will take kind of depends on timing around other other activities et cetera, but we do plan to make that publicly available.
Okay, maybe we could go to the next question.
Yeah.
Well move next to Corinne Jenkins at Goldman Sachs.
Good morning. This is Greg on for Karen So one question from us.
Previously announced the initiation of your EAP and I guess now having some time under your belt can you give us some color on the types of patients that have enrolled in that and maybe some of the feedback that you've received so far on the use of an intelsat through it.
Okay.
That's a great question I think the question of.
The question was what what kind of feedback or what are we seeing with the types of patients who are being proposed for the EAP, maybe say you could talk a little bit about that.
Okay.
Sure. Thanks for this question.
Just a reminder, that the EAP is similar.
Similar to a clinical trial and that the enrollment criteria and.
Site initiation criteria similar to that that we use.
The emerge phase III study however different.
From a clinical trial and that we cannot accrue for it.
It is.
Based on investigator request.
No.
The trial is still in progress and ongoing.
Assessing.
Enrollment in the types of patients and we are not providing further details at this time.
Got it that's helpful and one more from us.
So how are you guys all preparing for the Advisory Committee meeting with the FDA.
And what sorts of topics do you anticipate it will be in focus at the event.
Yes.
Ill, let fay take that and our supplement as needed, but say why don't you take the first crack at that.
Sure. Thanks chip.
Regarding the <unk>.
Contract and we've been at.
It's common in industry to prepare.
Foreign O'donoghue ahead of time, so we even before the announcement or the communications from the FDA, we have been working with.
A accredited by the very well known vendor.
Specializes in Odessa preparation in order to.
Anticipate or game plan any types of questions or issues that may be of relevance.
Yeah.
Overall as it most.
It will be come down to a clinical quest.
Question of risk versus benefit and other than that we don't have any further details.
Yes. Thanks.
I'd like to make a couple of other points about.
About <unk> just to remind people we were told that the ftes.
Planning and overreact, but we don't know if that will actually occurred there is historical.
There is historical.
Information about other products in which <unk> plans, they've actually been scheduled than they've been canceled last minute. So I think we'll just have to wait and see on that.
We often get a question.
Craig about when did the most kodak's occur.
I think in general our research shows that they occur four to six weeks before the producer date, something like that and.
I think the other comment is that we have a variety of consultants, so as well as the consultant that.
Okay.
Scribing, who is a very specialized.
Oh deck, an advisory committee a consultant we have other.
Broad.
Regulatory.
Consultants. So I think we feel like we will be very prepared through this process for whatever may come in as.
As we've commented in the past she commented today in her prepared remarks, I think we look forward to discussing the.
The opportunities for treatment with <unk> in this space.
Yeah.
Got it thank you for the questions.
Sure next.
We'll go next to calculate <unk> at B Riley.
Yeah, Hey, good morning, Thanks for taking the question.
Maybe first related to an earlier question.
I know we have the updated guidelines here.
I'm curious if we have a sense of.
How <unk> is being utilized in the real world based on your engagement with Kols.
Is the preference to use rub, Brazil, only four Rs positive patients and that frontline Esa naive group.
And does that utilization at least what youre seeing so far does it look consistent to the survey data that you highlighted.
Neil that's definitely for you reconcile in the real World is the.
System with what we've seen from survey data.
Alright. Thank you for the question <unk> I think.
So we did average pool based on the.
The second line space.
Nearly saw where things are but when we talk to physicians and start to dig into frontline use.
What we see clearly is both PSA is and thus far this up to be part of the treatment paradigm. When we're speaking to physicians, both academic and community I think you will see it referenced four Rs positive patients.
Heading towards despite us up but there are also important.
Nuances that needs to be kept into effect, especially Paul Evans.
<unk> thousand 200, typically say about <unk> hundred <unk> are also very much in play and then for Esa ineligible patients.
Commodities as you know converted north study those patients in particular with that study.
But they do have a broad label for physicians continue beside unmet need for batteries and population from our perspective.
I think we see board.
The degree of production depends upon.
Dissatisfaction or satisfaction dsos and in particular.
Our prescribers.
<unk> and we even see both of these drugs being used.
But it will not be a replacement order displacement issue for <unk> with.
To the best of our judgment per day as we go forward in these patients need a lot of new treatments and where does started those guys effort as well.
As part of the future treatment paradigm.
Furthermore, the return so hopefully this will answer your question.
Yeah, Yeah. That's helpful. Thank you and then chip you mentioned partnering partnering earlier I guess any more color on.
How youre thinking about partnering it could be useful to investors.
Maybe is it just for ex U S territories.
Yes.
Sure. Thanks, Yeah look of partnering is part and parcel of our business.
It's a an arrow that every company would hopefully have in their quiver and it's a conversation that.
<unk> needs to continue to develop and mature as a as you know as you move closer and closer to a.
Potential approvals and subsequent commercialization. So I think that we are have always been open to partnering what we commented on today.
With specifically around European commercialization, and I think that of course, we have the goal to bring <unk> to patients in that marketplace as efficiently and as effectively as possible.
Given that.
The MAA is now under review and we have a better feel for timing.
I think that we tried to point out that.
The earliest potential approval would be in quite late 2024.
And that means that European launch would be in potentially in 2025, So we're going to.
<unk>.
To evaluate our strategic options, which include.
Not only partnering but also self commercialization.
As we go into and progress through two.
2024, and I would hope we would be able to go.
An update we expect to give an update.
In 2024.
Specific part of the World.
Okay perfect.
And one last question.
This abstract.
That shows the differences in overall survival between.
Responders and non responders the transfer.
Transfusion independence, I guess have you done.
Any sort of early analysis for your own patients in emerge the emerge study.
And whether that.
That is consistent with what you're showing in that claims data from the attraction.
Well.
I think that claims data first and foremost I think that claims data is what I would call very mature data right.
<unk>.
It was a large number I don't remember the exact ends but it was very large number I think the purpose of that.
Of that study was to provide additional insights.
Ross.
Various platforms and across the various.
Drugs as to the.
Supporting the value of of transfusion.
Independence right and.
I think that that's been a staple of a belief amongst most practitioners.
And also for that matter academics, but I do think that.
Since we interact with claims databases all the time.
We're doing our own assessments, we thought this was really valuable and interesting and collaborated with Rami.
How much fee and I'm off it to produce.
To help them.
Analyze these data and make this presentation. So I think it stands on its own.
Honestly as a cause.
<unk>.
Overview of the value.
In this marketplace of transfusion.
Transfusion independent to achieving transfusion independence.
Thanks.
Perfect. Thanks, very much that's very helpful.
Okay, Great next.
Next we'll move to Gil Blum at Needham <unk> company.
Yeah, Hi, this is Mario <unk> on for Bill. Thank you for taking our questions. So just two quick ones from our side. The first one maybe a little bit of a follow up from one of the previous questions.
I did try to quickly, but I was unable to find it could you give any color on the rocks dosing that was used and improve enough I know you mentioned in the metals that those things.
And then the other question I'm looking at slide 10 of the presentation. So the ash abstract regarding mutations.
We see a pretty clear.
Efficacy across the various mutations.
Just wondering if theres any.
<unk> rationale why.
In particular.
S. XL, one appeared to have a little bit lower effects than the others or if you think that's just small numbers.
<unk>.
Thank you for taking the questions.
Yes, Ethan thanks, very much for both really interesting and good questions I will invite.
We'll invite you to comment on the Rux dosing first and then and then risks a bit on the.
Efficacy across the different mutations and how our old friends ASX 201.
<unk> appears to be a difficult.
Patients with mutations appear to be particularly difficult to treat.
Okay.
Sure Thanks, Jeff and thanks for the question Nathan.
Guarding the.
I'll start with you.
Regarding yeah.
Yeah, I do believe that.
It appears to be a smaller magnitude of benefit and that's mostly due to a.
A low number of patients with the ethics hotline mutation that mutation in particular.
<unk> is a very poor prognosis and a quick transformation.
Two higher risk disease or am now so it is it.
Even.
A bit surprising that.
We had patients with these mutations in the lower risk population that speaks further too.
The relevance of the IP asset classification and the.
The results that we are showing at ash regarding that abstract as well so it all fits together that because.
The mechanism of the Intelsat directed at Telomerase and telomerase is.
Over expressed in malignant cells and drives.
Malignant phenotype by targeting.
The disease itself, we have activity across the broad applications, regardless of what prognosis.
They protesting for patients.
Yes.
To address your second question it was actually your first.
Your first question about what's a little nip dosing.
Some of the study to assess safety and <unk>.
Inc.
Yeah.
Assessing safety in the context of <unk>.
<unk>, yes, so patients.
Our.
Enter the study.
Russell isn't it at whatever dose.
It was more beneficial and tolerable for that and so the rest of <unk>.
Per patient.
Okay. Thank you very much.
Thank you.
Okay.
Thanks next question.
We'll go next to Joel Beatty at Baird.
Hi, good morning, and thanks for taking the question.
And considering the market research that shows a larger market share for <unk>.
Better so do you expect that upon launch there could be some.
Moving from loss better subsea two <unk> had some some switching or would use upon launch mainly being patients are either newly diagnosed or failing their previous therapy.
Thanks, again, and I think this is Neil Neil area to comment on whether there is possibility for at least the switching etcetera at launch.
Okay.
Sure Joe Thank you for the question.
I think Joel just one factor to keep in mind as I answer the question is.
There are very few treatment options in low risk Mds.
As Jeff spoke to earlier I think the words you used was competitively less intense.
In practice physicians when you have a handful of charges. So what <unk> is.
While we may expect or want it.
And I think the <unk> Corporation two.
Really be best treated with whatever at auction the physician things for them.
Longer see benefit.
So if that's the case of irritation, not seeing benefit and there is a more effective auction.
Physicians will consider switching their patients over but if the patient is responding.
I believe.
And typically clinical practices to allow patients to benefit fully.
Through their disease through that treatment choice.
And then consider at other choices for their patients where they are but so hopefully that answers that part of the question. The last remark I would make here is.
Have a completely novel mechanism of action the level of durability and all the clinical effectiveness that we have shown I think will become really important.
And armamentarium for physicians to consider it.
<unk>.
Continuing to optimize accretion kind of me, so I'll stop here and saying that any clinical remarks from your side that might be.
Okay.
Okay. Thank you.
<unk>.
Any further questions Joel or did that.
Scott.
Thank you.
Okay, Great next question.
And there are no further questions at this time I would like to turn the conference back over to Aaron <unk> for closing remarks.
Thank you so much operator.
Thank you everyone. So much for your participation today, we look forward to keeping you updated on our progress. Thanks, so much.
And this concludes today's conference call. Thank you for your participation you may now disconnect.
Yeah.
Okay.
Yeah.
Yeah.
Okay.
Yeah.
Yeah.