Cytokinetics Incorporated Q1 2023 Earnings Call
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Okay.
Good afternoon, and welcome ladies and gentlemen decided kinetics first quarter 2023 conference call at this time I would like to inform you that this call is being recorded and then all participants are in a listen only mode at the company's request, we will open the call for questions and answers. After the presentation, we will allow for one.
One question per participant I will now turn the call over to Diane Weiser vital, Connecticut, Senior Vice President of corporate Communications and Investor Relations. Please go ahead.
Good afternoon, and thanks for joining us on the call today, Robert Blum, President and Chief Executive Officer will begin with an overview of the quarter and recent development that he might like E. V. P of R&D will provide updates related to Etsy campaign and other drug candidates comprising our early stage pipeline Stuart Kupfer S. P.
P N Chief Medical Officer will provide further updates on the development program for Epic Hampton, Andrew tell us EVP and Chief commercial officer will speak further about commercial preparation activities for Etsy, Camden and the market opportunity Robert Wong VP, and Chief Accounting Officer will provide a financial overview of the past quarter.
And Ching jaw, SVP, and Chief Financial Officer will discuss our financial outlook and corporate development strategy.
Robert Brown will provide closing comments and review expected key milestones for 2023. Please note that portions of the following discussion, including our responses to questions contain statements that relate to future events and performance rather than historical facts and constitute forward looking statements our actual results might differ materially from that.
Its projected in these forward looking statements additional information concerning factors that could cause our actual results to differ materially from those in these forward looking statements is contained in our SEC filings, including our current report regarding our first quarter 2023 financial results filed on form 8-K that was furnished to the SEC today.
We undertake no obligation to update any forward looking statements. After this call and now I will turn the call over to Robert.
Thank you Diane and thanks for joining us on the call today.
While we began the first quarter with a clinical stage program directed to the potential treatment of ALS. We ended the quarter acknowledging that we can no longer commit to that opportunity in.
In the best interest of the science and the shareholders, we must own that reality and we dedicate to that which ultimately holds the greatest promise for patients.
Towards that end I want to emphasize our confidence in and priority commitment to our specialty cardiovascular pipeline of novel cardiac muscle directed drug candidates.
Despite the disappointment related to ALS in the first quarter of 2023 significant progress was made across our cardiovascular pipeline with particular focus to Africa Hampton and its broad development program at <unk>.
And Stuart will elaborate screening and Socorro HCM, our pivotal phase III clinical trial in patients with obstructive HCM is expected to close tomorrow, enabling enrollment to conclude in a few weeks and the readout of results in the fourth quarter of this year as planned we also recently reported.
Data from cohort four of Redwood HCM as well as 48 week data from forest HCM, both providing compelling evidence for our next in class cardiac myosin inhibitor.
Finally, we made progress towards expanding development for Alfie Camden with a plan to soon start Maple HCM. The active comparator phase III trial versus metoprolol and continued preparations for our planned phase III clinical trial of Abbvie Camden in non obstructive HCM.
As you know during the quarter. We also received a complete response letter from the FDA for <unk>, we're continuing to engage with FDA and are planning to meet with FDA this quarter to discuss their feedback and to better understand what may be our options going forward. We expect to provide an update when we have something more.
Meaningful to report.
In the meantime, we're pressing forward towards potential international approvals for <unk> in Europe , we're engaging EMA and its review of the MAA and are preparing to respond to day 120 questions that in China. The center for drug evaluation of the National Medical products administration.
He is reviewing the NDA submission for <unk> <unk>.
As I previously mentioned during the first quarter, we also announced encourage AOS to phase III clinical trial of real disruptive met criteria for futility and the second planned interim analysis.
Our dedication to the ALS community over the last decade has been a hallmark of our commitment to novel muscle directed medicines.
And we are disappointed that will not be able to move forward a drug candidate for patients with this grievous disease given.
Given the data from the interim analysis, we proceeded to conclude study conduct encourage AOS and we're completing closeout activities in both this trial as well as the open label extension study during the second quarter, we plan to present full results from courage AOS at a medical meeting later this year.
The trial was designed and conducted with scientific integrity and in the interest of people living with ALS and we hope it may serve as a blueprint for how to conduct rigorous patient centric clinical research.
While we're certainly very disappointed in this outcome for courage ILS era.
<unk> is an opportunity to realize certain savings in 2023, which we believe can extend our cash runway and enable us to reallocate resources to further increase our focus on a few Camden.
Previously over 60% of our R&D budget was already devoted to RV Camden, but with savings. We now expect in 2023, and even higher proportion of our total spending will now be dedicated to RV Camden Ching will elaborate.
<unk> further on our expected 2023 spending in a moment.
So while the first quarter brought some unexpected setbacks, we remain as committed as ever to our mission to bring forward new medicines for patients with diseases of high unmet need are late stage as well as our early stage pipeline of potential cardiovascular medicines provide a solid path forward for <unk>.
Defined by our strong balance sheet to support continued execution in the interest of all of our stakeholders.
And with that I'll turn the call over to Saudi.
Thanks, Robert and the first quarter, we made substantial progress across a broad development program for <unk> in campton.
In March we presented data from cohort four of Redwood HCM at ACC.
Which showed that treatment with <unk> <unk> and resulted in significant improvements in heart failure symptoms and cardiac biomarkers in patients with non obstructive HCM.
Cohort four was a dose finding exercise, allowing for two dose titrations, but the goal of increasing dose with echocardiographic guidance and evaluating the effects on symptoms and biomarkers.
As a reminder, unlike in obstructive HCM, where the goal is to titrate patients to a minimally effective dose necessary to eliminate the <unk> gradient.
In non obstructive HCM.
Our goal is to titrate to the maximum dose based on ejection fraction.
And cohort four by week, 685% of patients achieved the highest dose of 15 milligrams of Abbvie Campton importantly, despite uptight trading patient to higher doses there were no drug discontinuation due to adverse events as.
As we previously reported.
Three patients had LDF drops below 50 at the week 10 visit.
However, the ELV abnormalize in all three of these patients after the two week washout period. The data from this dose finding cohort will inform the phase III dosing scheme.
In terms of safety <unk> was well tolerated.
There was one death due to sudden cardiac death.
The unrelated to treatment with Apple Hampton.
This patient had a history of sudden cardiac death prior to participation in Redwood HCM.
Two days prior to the event the patient's LDF as normal symptoms and Biomarkers improved and the plasma concentration of <unk> <unk> was within inspect the expected range.
We and the investigators believe that depth was unrelated to App you Captain and therefore, we remain confident about the safety and Tolerability of <unk> <unk>.
Yes.
Building on the presentation at ACC later this month, we're pleased to be presenting further data from cohort four and the late breaking clinical trial session at the European Society of Cardiology heart failure 2023.
Taking place in Prague.
These results will include data from additional patients who are not included in the results presented at ACC due to timing as well as data related to the Kansas City Cardiomyopathy questionnaire and other additional findings.
To provide some perspective on the goal of cardiac myosin inhibition in non obstructive HCM.
And it's important to note that although these patients lack of significant lv ot gradient their disease burden as measured by Nyj class and Casey Q as well as biomarkers, including anti program.
As just as severe as those patients with obstructive HCM.
The magnitude of improvement in these markers of disease burden that we've observed with Abbvie campaign, where substantial nearly equaling what we observed in obstructive HCM and provide a strong indication of treatment benefit.
We're pleased with the initial data from cohort four and we view them, both as supportive of advancing to phase III and highly encouraging of the potential effect of <unk> Camden in this important segment of the HCM population.
Additionally, at ACC, we presented 48 week data from Forest HCM. The open label extension study showing that treatment with Abbvie Catherine was associated with significant and sustained reductions in the average resting in valsalva Lv Ot gradient significant improvements in N Y J class.
And significant improvements in NT Pro BNP.
At 48 weeks, 88% of patients experienced an improvement of at least one NOI shape functional class, including many patients who are no longer symptomatic.
And those patients that reached 48 weeks, none remained an NOI class III compared to 47% of <unk>.
Patients who are class III at baseline.
Reducing symptoms can have a profoundly positive impact on patient lives and.
And so these data are quite notable.
Treatment with Abbvie campton appeared to be safe and well tolerated with no instances of treatment interruption or discontinuation attributed taffy Camden or.
We're encouraged that with longer term treatment with Abbvie campton patients are experiencing a sustained treatment benefit.
Through these data.
<unk> not only a clearer picture of the potential benefit at Abbvie Kempton may have for patients.
But also how epic campton appears to show potential to optimize both patient and physician experience.
During the quarter, we continued conduct of Sequoia HCM, the pivotal phase III clinical trial of <unk> in patients with obstructive HCM as.
As Robert mentioned, we expect to complete patient screening tomorrow and randomized the last patient soon thereafter words.
And we are tremendously grateful to all of our investigative sites around the world for working with us to achieve this milestone.
<unk> the DMC reviewed safety efficacy and Biomarkers for Sukhoi HCM and recommended that the trial continue as planned.
Enrollment nearly complete we're pleased with the patient population that we enrolled and believe we're achieving our goal of focusing on patients who stand to benefit from epic Hampton.
We met for important targets for enrollment that give us confidence in the results we hope to see.
First the proportion of patients who are taking beta blockers as background therapy, which limit heart rate from increasing and can blunt exercise performance met our expectations.
Second we enrolled patients with clearly impaired exercise capacity as evidenced by an average peak via two at baseline well below normal.
Third the proportion of patients using bicycle our treadmill to perform their exercise test met our expectations and finally enrollment in <unk> HCM took place globally enrolling our balanced and diverse patient population in the U S Europe , Israel and China.
We believe that the way we have designed and conducted Sukhoi HCM and the patient population we have enrolled.
We will contribute to a clear read on the potential efficacy and safety of <unk>.
Before I hand, it over to Stuart I also want to touch briefly on our earlier stage pipeline.
First in the prior quarter, we completed three ascending dose cohorts in the phase one study of CK 136.
Our cardiac troponin activator in healthy volunteers and expect data in the second half of the year.
Additionally, following our IND submission for a second cardiac myosin inhibitor CK 586, we received FDA clearance to initiate its phase one study.
586 represents a key new clinical program for the expansion of our pipeline and we intend to develop it for the potential treatment of heart failure with preserved ejection fraction.
Another important addition to our growing specialty cardiology business.
With that I'll turn the call over to Stuart to speak more on the expanding development program for <unk> in campton, including major Maple HCM and our planned phase III clinical trial in non obstructive HCM.
Thanks Patty.
In the first quarter, we made progress towards the beginning of <unk>, which we expect to open for patient enrollment in this quarter.
As a reminder, maple HCA is the second phase III clinical trial in patients with obstructive HCM during which we'll be assessing assay campton as monotherapy compared to my top of the law.
Primary endpoint is change in peak <unk> assessed by cardiopulmonary exercise testing from baseline to week 24.
Secondary endpoints include change in Hydro IHA class Casey CQ anti pro BNP and measures of structural remodeling.
Our goal with this trial is to evaluate the potential superiority of <unk> to the standard of care therapy at beta blockers.
Beta blockers are used as first line therapy for the majority of patients with HCM because historically there have been few alternatives.
While they have lockers can improve gradients and lead to mild symptom improvement a substantial portion of patients did not achieve desired symptom reduction with beta blockers.
And their associated with many undesirable side effects.
Additionally, beta blockers do not improve exercise capacity and when used in combination with a cardiac myosin inhibitor may attenuate its beneficial effects.
Without the Camden is shown to be superior to many hopper law. It may simplify the approach to treating HCM by enabling the use of Abbvie canton as first line monotherapy.
We're encouraged by data previously presented from forest HCN, demonstrating successful withdrawal from background therapy in patients treated with <unk> Hey, Robert.
Okay.
And our clinical cost Amit here.
And our clinical study sites are enthusiastic about participating in this trial.
We look forward to starting enrollment in Maple HCM.
Additionally, as <unk> mentioned the positive data from cohort four of Redwood HCM presented at ACC, we are encouraging for our plans to further expand the development program for ASIC Hampton by.
By starting a phase III clinical trial in non obstructive HCM.
We've recently had productive interactions with FDA to ready for this trial.
We're finalizing our planning and preparations and expect this third phase III trial of <unk> to begin in the second half of this year.
We look forward to sharing more information relating to the planned trial design later this year with that I'll turn the call over to Andrea.
Thanks, Stuart in quarter, one we continued activities and preparations for the potential approval and commercialization of Africa Hampton, including market research to further expand our understanding and better characterize the market and the opportunity while the overall prevalence of HCM in the U S is estimated to be roughly seven.
100000 to $1 million, we estimate the diagnosed patient population inclusive of both obstructive and non obstructive HCM to be approximately 290000.
Therefore, the undiagnosed population the sizable up to 700000 patients. We believe currently undiagnosed patients represent a very significant growth opportunity for category growth.
Through our market research, we've learned that the vast majority of diagnosed patients are currently being treated with beta blockers calcium channel blockers or both.
And we believe approximately two thirds of those patients are symptomatic and may be eligible for a cardiac myosin activator like Abbvie Camden if approved.
At the same time, we also expect the diagnosed prevalence of ATM to increase over time at a mid single digit growth rate driven by evidence generation commercialized treatment options escalating patient and physician interest expanding use of <unk> outside of the centers of excellence and the overall appreciation of CMI to treat.
<unk> of HCM.
Additionally, during the quarter, we continue to engage further with Payors, who expressed interest in both ATM CMI. It's generally an ASIC Hampton specifically some payers have limited experience with HCM due to a lack of approved therapies until recently, but they are interested in the disease, especially in patients with <unk> more highly symptomatic.
We see this as an opportunity for education and up leveling of their knowledge of HCM overtime.
Our development program for <unk> <unk> has been designed to provide ample evidence for <unk>. If approved may optimize both patient and physician experience through quick and sustained improvement of symptoms straightforward dose titration minimal drug drug interaction and avoiding dose interruption through maple HCM. We're also attempting to show.
That actually campaign is superior to the standard of care beta blocker to potentially further enhance patient experienced by avoiding beta blockers altogether and enabling simple mono therapy <unk>.
Finally, as we explained earlier this year the majority of the commercial team has been shifted to support Effie campaign launch readiness activities should it be commercialized and we plan to continue to assess our resourcing to account for the shift in focus from OMA captive mccarville readiness to Abbvie campaign readiness.
We are fortunate to have a talented and dedicated team with deep experience in cardiovascular disease markets propelling us forward to become a global specialty cardiology company, leading with Abbvie captive and with that I'll turn it over to Robert law.
Thanks, Andrew we ended the first quarter with approximately $704 million in cash and investments.
Our revenue in Q1, 2023 came primarily from a milestone payment we recognize from <unk> Sheng for the expected phase III trial of <unk> in patients with HCM to occur later this year as well as earned revenue from Astellas in the quarter.
Our first quarter 2023, R&D expenses increased to $79 4 million from $45 9 million in the first quarter of 2022, primarily due to increased spending for our clinical development activities related to our cardiac myosin inhibitor programs encourage AOS.
Our first quarter 2023, G&A expenses were $49 7 million up from $33 1 million in Q1, 2022, due primarily to higher personnel related costs, including stock based compensation and pre commercial launch readiness expenses and now Ching will review our financial outlook in <unk>.
Development strategies.
Thanks, Robert we ended the quarter was approximately $704 million cash on the balance sheet, which represents two years of cash runway.
To be clear, we're not adjusting our 2023 guidance today.
However, we do expect to reduce our overall spending in 2023 by more than 10% primarily through a reduction in planned outsource services and head count growth, thereby resulting in over $50 million in projected savings relative to previously forecasted spending for <unk>.
'twenty three.
By employing these measures were able to extend our existing cash runway this year as well as in 2024 during which time, we have previously anticipated incurring costs for manufacturing commercial supply commercial readiness preparations and medical Education Act.
<unk> four router center.
We expect to provide new financial guidance with our Q2 earnings call.
To further add to our balance sheet. This year I'll remind you that we also expect to receive a $50 million nonrefundable milestone payment from royalty pharma upon the start of the pivotal phase III clinical trial of <unk> in patients with non obstructive HCM.
Which is expected to begin in the second half of the year.
Yes.
And finally on the corporate development side, so further support our balance sheet and our path forward. This year, we are continuing to seek a potential partner in Europe for <unk> as well as in Japan for both <unk> and Abbvie Kimpton.
We also believe that we may need to open the aperture and potential European partnering of Abbvie Camden as part of our broader cardiovascular portfolio in light of recent events.
That said.
We continue to prepare for U S commercialization independently.
And as we progress our corporate development for the balance of this year. We will continue to make decisions that are in the best interest of patients and our shareholders and with that I'll turn the call back over to Robert bump.
Thank you Ching today, our company looks different than how we expect it to look as we started 2023 however.
However, we're confident in where we stand and in the road ahead of US we've overcome challenges before during our 25 year history, and we believe that with our foundation of a strong research and development pipeline rooted in muscle biology, we now have an opportunity to build the industry's leading specialty cardiology business.
As we look to the future we are optimistic and we have strong conviction in campton and also our other novel mechanism drug candidates advancing in our cardiovascular pipeline.
Also last quarter, we were proud to release our inaugural corporate responsibility report. This report reflects our commitment and continued integration of corporate responsibility throughout our organization as well as decision, making processes and our ongoing commitment to provide transparency and accountability on matters related.
To corporate responsibility, we intend to release, an annual update to this report on our progress and goals building on the foundation and as we continue to focus on how we can better serve patients the communities around us as well as the environment.
As gene mentioned, we're an advantaged position with the cash that we have and we're managing it carefully.
We're prudently managing our spending our growth in our allocation of resources, we have a promising development program with our cardiac myosin inhibitors, <unk> and CK 586, and in addition, we're equipped with an early stage and research pipeline that adds further to our portfolio of muscle directed therapies.
Our intention is to build value for our company through our science and that has not changed.
And we will continue to balance the needs of both patients and shareholders as we advance forward.
Now I'll recap our upcoming milestones for <unk>, we expect to continue to pursue potential international approvals for Omi captive mccarville, including in Europe , and in Japan, I'm, sorry in China.
Rafi Canton, we expect to present additional data from cohort four of Redwood HCM at heart failure 2023 on May 20 in Prague, and we expect to complete patient enrollment in Sequoia HCM in Q2 2023 with results expected in Q4 2000.
'twenty three we expect to begin Maple HCM, the second phase III clinical trial of <unk> as monotherapy in patients with obstructive HCM in Q2, 2023, and we expect to begin a phase III clinical trial of <unk> and non obstructive HCM in the second half of <unk>.
23, as well, we expect to advance our U S go to market strategy. During 2023 for CK 136, we expect single ascending dose data from the phase one study in the second half of 2023 for CK 586, we expect to advance that compound into.
First in human Phase one study in this quarter Q2, 2023, and finally for <unk>, we expect to conclude clinical trial conduct and complete the majority of closeout activities for courage AOS in Q2, 2023, and we expect to share results in the second half of the year.
And operator with that we can now open up the call please to questions.
To ask a question. Please press star one one on your telephone and wait for your name to be announced to withdraw. Your question. Please press star one again as a reminder, please limit to one question. Please.
Please standby will be compile the Q&A roster.
And our first question comes from <unk> Dunn with <unk>.
<unk> condo the truest your line is open.
Thank you so much good afternoon. My question. Good afternoon. Thanks for taking my question.
For epic Hampden and it looks like you're on track for enrollment completion in the second quarter.
Yeah.
Just was wondering how confident are you about the timelines for fourth quarter is there any chance that it could spill over into early.
2024, and then as you see can launch and whatever you are hearing from them from the ground.
Has that changed what do you think you need to do to prepare for commercialization of <unk>. Thank you so much.
So two questions really.
The first one is around enrollment and we do expect to be completing enrollment as you heard from 30 such.
Such that randomization and enrollment are both concluding in the month of May and therefore, we are confident to see results.
In the fourth quarter of this year.
With regard to a potential commercial launch of <unk> Camden, we're monitoring the uptake and cardiac myosin inhibitors as a class.
Carefully and we like what we see we believe that.
The.
The market is evolving very much as we predicted and we're not expecting that that should change the way, we think about the commercial uptake.
Maybe I'll just turn to Andrew to see if there's anything he wants to add.
I agree Robert.
BMS recently reported their first quarter earnings.
I think showed good momentum.
And we're hearing very positive things for.
The category overall from clinicians so it doesn't change the way we feel about it all I think if anything we're even more bullish about it than maybe we were before.
Okay.
Thank you so much.
Sure.
One moment for our next question.
Our next question comes from.
Salim Sayed with Mizuho Your line is open.
Absolutely right.
Hey, Robert Thanks for the question. So just one for me.
On the enrollment Sequoia and I, just want to make sure I understand this correctly.
I'd love to just get confirmation from <unk> or some color on when was the last time you looked at the <unk>.
Blinded aggregate of standard deviation for peak via two in Sequoia and can you confirm that we are indeed.
At $3 five on the standard deviation there is known and that there's no risk of trial increase at this point. Thank you.
Just to answer that there is no.
Risk of increasing.
<unk> at this point.
As I've said, we're going to be done enrolling patients tomorrow.
And we will finish randomization shortly and standard deviation I can't tell you exactly the last time I saw probably in the last week, but.
It's certainly within the expected parameters.
Super helpful. Thanks, So much guys.
Thank you Celine.
One moment for our next question.
Our next question comes from Tess Romero with J P. Morgan Your line is open.
Good afternoon guys.
Hi, good afternoon, everyone. Thanks, so much for taking our question.
One from us on non obstructive HCM.
So we're looking forward to that.
<unk> presentation. There later this month on cohort four as we recall <unk> is one of the key endpoints you're monitoring for health status can you quickly oriented briefly to you what magnitude of change you are looking for over 12 weeks of treatment.
<unk>.
Is there a change that would be considered clinically meaningful there.
And kind of what we should be expecting.
With respect to approximate baseline score thanks, so much.
So obviously for the fact that this is an open label study you recognize that we've seen these data so while we don't want to front run the data ahead of the presentation as a late breaker.
Later this month I will ask <unk> to try his best to answer your question without being too.
Forward, leaving.
Yes, I don't really want to give quantitative.
Expectations, given I know, what the quantitative numbers are but.
I should say that the baseline data indicate.
A severely symptomatic population.
In terms of the baseline <unk> score and in.
In general.
Expectations of increase of five points of core is what's key.
Considered clinically meaningful.
And so I think those are that's how I would put that in context as you see the data later later this month.
And any other kind of analyses that we should be specifically looking out four beyond PC CPU that we haven't already seen to date.
Yes, we will.
Actually be showing data.
In relation to.
The assessment of angina, which is something that.
Hasnt really been assessed in.
And in a trial like this before.
So I think we'll see some of the first data in terms of managing a score in HCM.
We will be looking at some of the echo parameters.
And Youll see.
<unk>.
More of the KC CQ data than just what the Delta was so youll see that.
The.
Responders that are have smaller or larger responses in proportion of Stifel.
Okay.
Thanks.
Thank you.
One moment for our next question.
Our next question comes from Charles Duncan with Cantor Fitzgerald. Your line is open.
Hi, Charles.
Hey, Robert.
<unk> congrats.
Nearing full enrollment of Sequoia and thanks for taking my questions I suspect there'll be a lot of questions. So I'll ask one.
On <unk> and the SaaS program Im wondering would that be an out licensing candidate and ex U S. What are your plans for it.
So right now for around basin.
Sure.
So right now in light of the fact that we're still conducting.
Ah study closeout activities.
We really can't know for certain how the final data are going to shape up but based on the interim analysis, we've seen enough data to suggest that it wouldn't be proper to continued conduct of that study in ALS.
We've seen signals of activity of <unk> in other indications in phase II.
But in light of this phase III.
Result.
We really don't have any current plans for real deceptive, but we'll assess those again once we see the final study data and Thats, both as it relates to U S and ex U S. We do have other.
Activity is associated with fast skeletal muscle and we're assessing how they may contribute to our pipeline growth and advancement going forward.
Obviously, we also have other programs in research that read on skeletal muscle and neuromuscular.
Indications and similarly, we'll be assessing all of those together.
In light of this recent developments so probably can't answer your question well enough to your satisfaction right now, but it is something that's top of mind for us.
I guess, if I just ask Ken can I assume that you are very much focused on becoming.
Cardio or beat being becoming known as premier cardio innovator and not leveraging the platform more broadly.
Okay.
I wouldn't conclude that as generally as you stated it but certainly our clinical pipeline is focused to specialty cardiology and thats what were going to be focused on with regard to a majority of our investment spending we still have research programs that are directed to other muscle types and other indications, but those are not.
Things that typically our shareholders are privy to.
In light of the fact that they are earlier stage and therefore, we keep them still confidential as it relates to what shareholders are mostly focused to yes, I think you can.
Conclude that our focus is on specialty cardiovascular medicines of which we have for such drug candidates now in our pipeline.
Yes, very good.
Thanks for taking the question.
Thank you Charles.
Our next question.
Our next question comes from Carter Gould with Barclays. Your line is open.
Hello Carter.
Hey, guys you have got everyone on the line for Carter. Thanks for taking our questions. We Scott wanted to have a Camden how would you set expectations for how quickly <unk> is planning to turn around an NDA on the back of the Sequoia data have you already started that process ahead of the data and then a follow up housekeeping question should we expect a press release announcing the completion of.
Enrollment in Sequoia when that does happen.
So to answer your first question, yes, we've already started that process around which were.
Looking at timelines, how we might bring them in what we can be doing ahead of the.
The results of Sequoia and how we can move as quickly as possible.
To submit not just in the U S. But also internationally.
For a potential approval of <unk>. So that's very much a priority and already in recent weeks, we've had many such meetings on that very matter.
Your second question related to whether or not we're going to be announcing completion of enrollment and the answer is no.
With regard to Sequoia, what we've indicated on this call is what we intend to have stand as our communication, which is we expect to complete screening tomorrow, and we're going to be completing enrollment and randomization here.
In this month of May.
Thank you.
Thank you.
One moment for our next question.
Our next question comes from Yasmin Rahimi with Piper Sandler Your line is open.
Hi, good afternoon guys.
Hello, Robert Thank you so much for always thoughtful remarks.
Team earlier this week, we saw data from.
The explorer study conducted in China.
And when we benchmark data versus the explorer study it appeared that the treatment response with greater than.
Maybe if you could just comment on is there anything different than that.
Asian population or is it just the baseline demographics that could have led to differences and then more importantly get the question leads into what percentage of the population will be in China or could that also be impacted and maybe.
Boost the results further I appreciate any color you could give us in that regard.
Jim backward yes.
Sure good questions and good pick up certainly we noted that.
Communication regarding the data in China and.
I'll ask <unk> to comment and I'm thinking he'll probably also defer to Stuart on some of that too. So between the two of them hopefully will get to your questions will enough.
Yes, I think I think the first thing.
And of note is that net.
Explored China trial didn't use <unk> and <unk>.
And their trial. So I don't think you can really speak to larger results per se.
Since that time.
The primary assessment of function wasn't included in that trial.
Certainly had sizable changes ingredients.
<unk> and <unk> and NOI J class.
Okay.
We are quite substantial but I'd be cautious about trying to compare.
Magnitudes of responses between trials that were enrolled at different times in different geographies.
Different populations.
Do you want to have anything to add to.
I agree with that and it's always a challenge to compare across studies.
Generally speaking, but the results certainly look encouraging continue to build.
The evidence base for supportive correct myosin inhibitors worldwide.
With respect to Sequoia.
We are collaborating with our colleagues you are seeing in enrolling patients in China.
<unk>.
Patients from China.
Definitely contributed to enrolment of Sequoia so.
We'll have a very diverse group of patients where.
When we have the final results to reflect upon and.
<unk> support.
Supportive regulatory strategy.
We're not disclosing the specific number or the percentage of patients enrolled in China other than to say that we believe it should be sufficient to support registration in China.
Thank you so much.
Jonathan Thank you.
One moment for our next question.
Our next question comes from a cause to worry with Jefferies. Your line is open hey.
Hey, guys. Thanks.
Hey.
So look in your view.
Why would partnering at the Camden in Europe being the best interest of our shareholders versus an outright sale of the company, especially before Sequoia has read out I was kind of surprised to hear that on the call today and what could really swing that decision one way or the other and then any thoughts on releasing baseline characteristics or the Sequoia population midyear.
If the readout for Q4, thank you.
So I'll answer the first and last Friday to answer the second and the point of that comment that you made was not to say that we are committed to partnering Effie kimpton.
Outside of the United States other than we think it's in the interest of shareholders that we assess what could be.
Possible.
And to your question about whether we should go down that path versus selling the company, obviously thats not something that we can address on the call like this.
For the fact that those are matters that.
Don't warrant.
A discussion.
In light of what could be our corporate development strategy. Our corporate development strategy is very much to maximize shareholders for those matters that are under our direct control.
And this is one of those things that we can control as it relates to the best way to.
Enable us to access capital and do what's best for science patients and shareholders. So we think it's incumbent upon us to assess what would be possible in the opportunity to this point there have been lots of inquiries around <unk> and we've been relatively.
Close minded to considering them, but in light of recent developments et cetera kinetics, we think it's in our interest and net of shareholders to at least be opening the aperture to what could be those possibilities and thats, what we intend to do.
The next question you asked was related to baseline characteristics in Sequoia and maybe I'll ask <unk> to comment on what might be our plans there.
Yes, thanks caution or the question I've been asked this before were looking.
See whether perhaps we can pull together.
Final baseline data and find the venue for presenting them they won't come.
Far in advance of probably the final data.
From Sequoia or rather the topline data from Sequoia.
But.
We'll update.
Folks once we have an idea of what we are.
Planning to do there for sure.
Thanks very much.
One moment for our next question.
Our next question comes from Jeff Hung with Morgan Stanley . Your line is open.
No Jeff.
Hi, Thanks for taking my question.
It depends on the Sequoia data, but how do you think about the likely potential scenarios for rins on the attic Camden label like what would you think you need to show to have run their less onerous than those for <unk> or to not even apparatus.
Yes.
Well, obviously, that's a difficult question to answer until such time as we see data from Sequoia and also continue to have good data from the open label extension, but it is our hope that we will have.
From forest HCM as well as Sequoia data that would be enabling of us to make a case to FDA for what might be.
A lesser rems or no rems and in that regard.
Really difficult to speculate until we have the actual data but.
With the fact that you asked the question, maybe I'll see if Saudi or Stewart of anything to add in terms of what they're going to be looking for that might determined ultimately our regulatory strategy that way.
Well I think we'll certainly be looking at how dosing was implemented how tolerated.
Okay.
No adverse events related to the need to monitor patients.
As we pointed out in forest HCM.
We summarize.
Summarize the 48 week data earlier.
We haven't had drug discontinuation due to low ejection fraction.
Our treatment interruptions.
And.
We hope that those events.
Are limited to the extent that.
The monitoring of them can be.
Yes.
Appropriately gauge if you will so that may be there is monitoring during the titration phase.
And then some mineral thereafter twice a year or something like that but I think it will depend on the data and it's difficult to speculate at this time.
Okay. Thank you.
One moment for our next question.
Our next question comes from Jason <unk> with Bank of America. Your line is open.
Hi, Jason Hello, Thank you so much for taking our questions and congrats on the great progress.
Going to the earlier stage pipeline now.
586, entering the clinic can you provide some color over your developmental our development strategy and half path.
Do you still see this as an opportunity for <unk> as well and maybe Conversely, where does HCM fit and plans for 586.
Excellent very good questions.
I'll ask <unk> to respond what we're doing in <unk> is become a central part of our development strategy 586.
<unk> is a compound that.
Has.
Routes in the same science and biology that gave rise to <unk> kimpton, but it comes from a very different chemical class with a different mechanism and as such we think it may be better suited for our development and half Perth in Saudi can speak about the therapeutic hypothesis and how we see that rolling forward.
Yes, hi, so CK 586.
As we.
We have developed it has a lot of the same properties of <unk> campaign and that and the shallow exposure response relationship. We think it's relatively clear of drug drug interactions.
Thank the dosing should be predictable PK should be appropriate for something that needs to be titrated and reversible.
And.
What we have strategically decided to do is to really separate the development of HCM from half to half by using App campton.
Developing that specifically into HCM.
Theres a lot of obviously the patient population there is very different than the pet population, whereas 586 as something that would be developed specifically into half path.
And thereby.
Sort of confuse one drug versus the other and where to use it and so forth. So.
We look forward to starting the phase one for 586.
Shortly and then from there proceeding into what would be a.
Beginning to look at this study this and have passed on.
We will give more details on that as we as the.
Graham progresses.
Certainly the work that we've done with healthy Kimpton and HCM.
So on what might be the potential for 586 didn't have tough and we see that there is a translation there that Len.
<unk> support for this mechanism and warrants its exploration.
Definitely thanks, so much for the color.
Thank you.
One moment for our next question.
Our next question comes from Jason Butler with JMP Securities. Your line is open.
Hi, Good afternoon, Jason Hi, Robert Thanks for taking the questions and congrats on the progress.
What about.
Non obstructive HCM.
Given the obvious lack of great answer can you talk to how you think biomarker monitoring to play into clinical practice share versus.
Just treating symptoms.
Is there something.
How do you think.
The practice could emerge here.
Good question, I'll turn to Saudi and Stewart to answer that.
Well I think.
Terms of how the.
The drug may be deployed in HCM.
Practically speaking I think symptoms and functional improvement and also what will.
Side and the physician.
More so than Biomarkers I mean, I think certainly we will want to look to see anti pro BNP go down as a sign of biological response, but.
We aren't going to really have a whole lot of.
Understanding for quite some time in terms of how much it needs to go down and what does that mean.
In the long run for the patient.
So I think a lot of ways. This will be guided by how patients feel and weather.
Theres any continued room for improvement in their symptoms as you escalate the drug.
Stuart.
Any other thoughts there.
I think thats completely completely right. It just provides more information to profile and improvement of symptoms and function and how that relates to these pharma dynamic markers, we expect to improve.
I mean, it is nice to have an objective biomarker.
Dosing something.
It can sometimes be hard to assess symptoms.
So the biomarker is helpful, but it probably won't drive implementation.
Great. Thank you.
Thanks, Jason.
One moment for our next question.
Our next question comes from Rohit basin with need him and co. Your line is open.
Good afternoon.
Hi, Good afternoon. This is Ryan on for Serge. Thanks for taking our questions can you just talk about your regulatory strategy for <unk> in Europe will the trials being run fulfill requirements or do you expect to run additional studies. Thanks.
Good questions.
We do believe that the conduct of Sequoia is occurring globally should be satisfactory to requirements.
Throughout Europe .
And also the rest of the world. So it's not just being driven by our U S strategy, but a global strategy.
And same with the way, we're thinking about maple in the way we're approaching an HCM. All of these are intended to be global studies that should be serving our interest globally.
Thank you.
Thank you.
One moment for our next question.
Our next question comes from Madhu Kumar with Goldman Sachs. Your line is open.
Hello, Madhu, alright, omari on commodities.
We have a couple of questions. So first what do you think you need to see from Maple HCM trial from cardiologists to position assay ahead of.
Metoprolol and the OSC MTA paradigm and then second are there any considerations to running a phase III trial for efficacy that is comparable to <unk> valor HCM trial.
So two.
Two part question one relating to.
I guess.
Effects that would delay the need for septal reduction therapy that was part b of your question.
Then part a was led.
Part a is what do you need to see from the Maple HCM trial for holiday on logistics position that they can't do it.
Metoprolol.
So I'll ask <unk> to comment on both A&P.
Yes, so I mean with regards to Maple I would say that strong data.
Showing that Abbvie campaign produces sizeable increases and exercise performance KCG Q Nyj class relative to beta blockers would certainly provide a.
Strong rationale in the guidelines for it being considered as first line therapy if sequoia.
Reads out the way that we hope it does it will show that.
<unk> added to standard of care is better than standard of care.
And what we hope to show is that App in campton by itself is better than the initial standard of care.
And thereby.
Provide the rationale for using it first because physicians really arent that excited about.
How about starting beta blockers and their patients they can be.
Difficult to tolerate.
And have side effects that.
Hum.
The patients don't really enjoy.
So these data would.
I would support that I think they are they.
Interest in that trial and excitement by the physician community is also a good reflection of the.
The importance of this particular question.
With regards to the.
Yes.
Whether we would conduct in our valor <unk> trial with with Abbvie Canton, we haven't really considered doing that at this point.
Certainly something.
And when you think about the cardiac myosin inhibitor class at summer nights to expand the field as opposed to just repeating.
Work, that's already been done.
If we were to do something.
Surgical eligible patients I think we probably ask a somewhat different question.
And then go from there but.
At this time no plans to do something that looks like.
Alright, thank you.
Thank you.
Our next question.
Our next question comes from Ash Verma with UBS. Your line is open.
Good afternoon.
Hi.
<unk>.
The bulk of the use is coming from the $2 five and finally doses and Hyatt.
Being used.
So does that indicate that presumably physicians are not able to titrate up underlining the need for safer effective drug for HCM.
And on the flip side of the argument do you think like if the other one incidence of systolic dysfunction is below what was seen in the clinical study.
And the FDA to lag that aims at the request of BMS.
So I think those.
Two part question is better meant for BMS to answer not for Psychokinetic. So I don't think it's appropriate for us to be commenting on the dosage strengths.
<unk> and what that means for patients.
Or otherwise what might be necessary for removal of the Rems program.
We certainly have our views, but I don't think thats something that we ought to be sharing publicly.
Thanks.
Thank you.
One moment for our next question.
Our next question comes from Dane Leon with Raymond James Your line is open.
Hey, Dan.
Hi.
Congrats on the progress.
I'll keep it short because it's been a really long day.
The question I would be interested in hearing from from your team as you go into the pivotal results in readout courtyard.
How are you thinking of taking those results, which is obviously a very comp study to the predicate that supported the approval map Camden.
And one.
Position goes are resolved.
In terms of discussion with FDA and what you would want from a labeling discussion and think it is very important to have on the label.
To help differentiate the drug in its clinical utility versus Mavic Hampton and then secondly bring forward to the <unk>.
Clinical community to help them understand potential advantages of getting patients through that really grinding 12 month process that.
<unk> continues to be a headache with cams iOS.
So any any initial thoughts there obviously, we know ahead of the study results but.
I think we kind of nowhere where things are going.
So here again, it's not for us to be commenting on something in a comparative way to <unk>. What I will say is that we have developed coffee kimpton.
Much with an eye on demonstrating some of its properties for what could be an optimal cardiac myosin inhibitor.
As it relates to efficacy tolerability safety and convenience.
I'll ask Saudi to maybe respond to your questions as to what will be specifically looking for.
And maybe Andrew wants to comment as well.
How it might play ultimately into the marketplace, but our goal is to ensure that cardiac myosin inhibition is something that physicians are comfortable reaching for when they think about their patient's obstructive and non obstructive and not just those cardiologists, who might be in centers of excellence, but cardiologist.
Outside of those centers, who might also be encountering those types of patients.
Maybe <unk> you want to start.
Sure I think.
Obviously, we've.
We're hoping that the data from Sequoia will support a label that will make the drug easy for patients to use easy for physicians to use.
And maximize its safety and Tolerability.
Yeah.
Those data will be shared with FDA those discussions will be had.
We recognize that there is a lot of patient burden here in terms of dose escalation because of the need for iqos and things like that and monitoring and our goal will be to.
If you will.
Go with the data and what they suggest as necessary.
As a means of maximizing safety and minimizing patient burden.
And I think that's in the interest of patients and the FDA usually has the interest of patients in mind.
And from a commercial point of view, we certainly won't have comparative data for <unk> and we won't be comparing our focus will be on our data.
And how that leads to a label that fatty described making sure that we educate a broad range of cardiologists nod kit centers of excellence that we get market access that we support.
<unk> through patient services and that the market <unk>.
Land scape and ecosystem payers treaters and all Hcp's are very clear on Abbvie.
<unk> campaign, and the potential benefits and risk. So they can make an informed choice and that's really what we'll focus on.
Okay.
Alright. Thanks.
Thank you.
Operator.
Operator are you still with us and Kim with.
With Oppenheimer.
Thank you.
Hi, Robert and team.
Anything.
I'll just I'll just add maybe one question on April .
This study is sort of more important for answering clinical and commercial questions and not necessarily regulatory one.
Are there any specific populations, but the team is hoping to treat and see the benefit of that will be kept in may.
It may differ from Sequoia.
Well I think that is.
The purpose of <unk>.
Expanding the development program to include an HCM and maybe I'll ask Stuart to comment on how that complement the patient.
Patient population in Sequoia.
I was asking about.
I get it but he is asking about expanding the patient population right. So.
Sequoia.
Studying patients who are generally later and the natural history of the disease.
Most of them receiving background therapy.
For treatment of HCM and we're considering.
Targeted patient population that is somewhat earlier in the natural history of the disease.
So those details will be discussed soon but.
That's sort of generally this general the strategy because strategically we are interested as we mentioned in determining if epic can't it would be appropriate for first line therapy.
And potentially superior.
To the current standard of care in beta blocker treatment.
Maybe just a follow up.
Good morning.
Hey, Jess.
I was just going to ask I mean is it right to think about in April as maybe a setting where you hope.
And in addition might be best optimize because you're sort of preserving and.
That way you could show a benefit.
Milder disease patients.
I think in.
The question really is our beta blockers.
<unk>.
And if you use a cardiac myosin inhibitor or beta blockers, helping or hindering the Maxim maximization patient benefit.
And so <unk> designed to help us answer that question.
In terms of patient population maples towards settle enroll patients.
Potentially.
Somewhat.
More or less severe gradient slower earlier in their disease potentially.
We're trying to enroll patients that are not on beta blockers could imagine that patients naive to therapy might be in.
Patients that get enrolled in maple.
But think of when you think of HCM as.
Our whole disease entity and you have patients like in Sequoia that represent those that have high gradients.
And represent one portion of the pie patients with NACS, representing another portion of the pie and maple patients kind of representing something in between.
And then including patients like those in Sequoia.
Ultimately the goal is to try and generate <unk>.
Evidence in the whole pie and not just specific slices of it so that there's really a rationale to use cardiac myosin inhibitors in patients with HCM and not specific subsets of HCM.
Okay, great and thanks for taking my question.
Sir Thank you.
And I'm showing no further questions at this time I would now like to turn the conference back to Robert <unk> for closing remarks.
Thank you operator, and thanks to all of our participants on the teleconference. Today. Thank you for your continued support and interest in Cytogenetics. We're looking forward to keeping you abreast of our progress with regard, especially taffy Camden, which is slated for quite a lot of news flow this year and a commitment of around.
Our investment spending that I think is very much in the interest of our shareholders and with that we will keep you apprised of progress through the remainder of this year operator with that we can now conclude the call. Thanks very much.
This concludes today's conference call. Thank you for participating you may now disconnect.
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