Q3 2023 Cytokinetics Inc Earnings Call
Okay.
Okay.
Good afternoon, and welcome ladies and gentlemen, societal kinetics third quarter 2023 conference call.
At this time I would like to inform you that this call is being recorded and that 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 question per participant.
I would now turn the call over to Diane Weiser Cytogenetics 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, I will begin with an overview of the quarter and recent developments that email like E. V. P of R&D will provide updates related to etsy, Camden focus just acquire HCM and forest HCM Stuart Kupfer S V P M.
Chief Medical Officer will provide additional updates rafi Camden relating to Acacia HCM and Maple HCM and I'll also discuss our early stage pipeline inclusive of CK 586, and CK 136.
Andrew Kal, our EVP and Chief commercial officer will discuss commercial readiness activities for I'll see Camden, 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 strategies.
And finally, Robert Brown will provide closing comments and review upcoming expected key milestones.
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 those projected in these forward looking statements additional information concerning factors.
It 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 third quarter 2023 financial results filed on form 8-K that was furnished to the FCC 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.
I am pleased with the progress we made in the third quarter, particularly focus to Alfie campton, our top priority with emphasis on the execution of Sukhoi HCM and advancement of its broad development program.
Thanks to the diligence and dedication of our teams we remain on track to share top line results from Sequoia HCM in late December well. This timeline is admittedly tight we're confident due to the superb organisation and oversight.
HCM by our clinical operations and statistical teams.
Like all of you on the call we're eager to see these topline results.
Our society will elaborate we have strong conviction that the topline results from Sequoia H D. M will meet our high expectations for both safety and efficacy based on the patient population enrolled in Sukhoi H C M as well as the unique attributes of Alfie Kimpton.
<unk> H C M meets our expectations on both efficacy and safety, we expected may contribute to expansion of the cardiac myosin inhibitor or CMI category on both accounts.
Simultaneously during the quarter, we continued to build our specialty cardiology franchise by advancing the ongoing clinical trials in the development program for <unk> Kimpton as well as earlier stage clinical research with enrollment underway in both Maple HCM and Acacia HCM.
We foresee near term future with multiple catalysts for Camden.
And rounding out our specialty cardiology franchise, our CK five eight fix a second cardiac myosin inhibitor and CK 136, a cardiac troponin activator on which Stuart will provide an update.
Our cash position at the end of the quarter represents over 18 months of cash runway based on our 2023 net cash burn guidance.
Importantly, as is our practice for both financial health and prudent planning, we're gating spending through the readout of top line results of Sukhoi HCM and were judiciously, focusing our current spending on activities related to our top priorities.
Then with focus to RFE, kimpton, and a critical eye towards cost efficiencies.
Today, you'll hear about the continued progress we made during the quarter and what you expect through the end of the year.
As we approach 2024, and as you heard a few weeks ago at our Investor and Analyst day.
<unk> kinetics remains laser focused to delivering on the promise of Alfie kimpton as well as two advancing our earlier stage pipeline as we build our specialty cardiology franchise for the potential benefit of both patients and shareholders.
And with that I'll turn the call over to 30.
Thanks Robert.
The third quarter, we made significant headway across the development program for <unk> Canton.
Currently we were pleased to present the baseline characteristics of patients enrolled in <unk> HCM, the pivotal phase III clinical trial of that be captain at the HCM Society scientific sessions.
The baseline characteristics met our objective for the intended trial population, having enrolled a global diverse and real world population with a substantial deficit in exercise capacity and significant symptom burden despite existing standard of care.
Patients enrolled in Sukhoi HCM had an average peak oxygen uptake our peak via two of $18 five milliliters per kilogram per minute at baseline or 56, 9% of that predicted for their age and sex, which is an objective indicator of the extent of their reduced exercise capacity.
<unk>.
Nearly a quarter of the patients were <unk> functional class III.
An average case, you see Q score of $74 seven further reflective of a highly symptomatic patient population despite background treatment with guideline directed medical therapies.
Background medical therapy included beta blockers calcium channel blockers, and disopyramide with patients permitted to receive combination background medical therapy.
Beta blockers, which are known to blunt maximum exercise capacity due to their effect to slow heart rate for used and 61% of the patients.
We are very pleased to see that our objectives are achieved and that we enrolled the population that we intended.
As we approach the readout of Sukhoi HCM later this year, we're confident in the completeness of the data and that we're outperforming the design assumptions for the trial.
The extent of missing data and the standard deviation of the primary endpoint are all within our assumptions, which should augment the power of the trial to assess the change in the primary endpoint.
I'm also pleased to report that nearly all patients have completed dosing and sites have not reported any patients with lv ejection fraction less than 40%.
We're eagerly anticipating sharing their topline results in late December.
Our convictions are high that the results of core HCM will meet our expectations. These convictions or further reinforced by the long term efficacy and safety of Athey Campton that we're observing in forest HCM. The open label extension clinical trial.
At our recent Investor and Analyst day, we shared new longer term data with data available in some patients for greater than two years.
More than 200 patients have been enrolled enforced HCM to date and data from 143 patients with obstructive HCM where available for this analysis.
The new data from Forest HCM showed that no patient had a treatment related lv ejection fraction less than 50% during the treatment period during the titration period.
And approximately two thirds are receiving 15 milligrams or 20 milligrams of <unk> Camden.
During the maintenance phase of course, HCM there've been no instances of Lv, plus was 40%, which would require treatment interruption and only three instances of LTE plus 50% that simply required a dose down titration.
Of the 579 monitoring Echocardiograms completed during the maintenance phase of treatment 99, 5% of them did not result in a dose titration.
Titration.
We also observed in these reported data from Forest HCM.
Mean resting in valsalva, Lv Ot gradients remained reduced and below the diagnostic threshold for obstructive HCM after treatment for more than two years.
Patients also experienced sustained reductions in cardiac biomarkers and improved symptoms.
Approximately half of patients who are NY at Shea functional class, one are asymptomatic and 80% of patients improved by one or more functional class.
Furthermore, 90% of the SRT eligible patients at baseline.
No longer SRT eligible at the time of this analysis.
<unk> has been generally well tolerated with no treatment related serious adverse events as assessed by investigators.
Additionally, approximately 30% of patients have reduced doses of background therapy or discontinued it entirely at the discretion of the treating physician and or by request from the patient.
This supports the rationale for Maple HCM with Stuart Stuart will discuss next.
These new results from forest HCM are quite compelling and we look forward to continuing to gather more longer term data for Abbvie campton in this open label extension.
Shifting briefly back to Sequoia HCM following the top line readout of the results in late December we expect to hold a meeting with FDA in the first quarter of next year to discuss the topline results with the goal of potentially submitting a new drug application for Abbvie captain in the second half of 2020.
For <unk>.
During that initial meeting to review the topline results and we'd expect to begin a dialogue about how does the safety and efficacy of Abbvie campton observed in Redwood HCM, Sequoia HCM and forest HCM will influence the design of a Rems program.
We look forward to these interactions and we will provide further updates next year.
Now I'll hand, it over to Stuart to elaborate on additional progress we've made for <unk> and provide an update on our earlier stage clinical pipeline.
Thanks Patty.
During the third quarter, we started Acacia HCM pivotal phase III clinical trial of Abbvie Camden in patients with symptomatic non obstructive HCM.
Anthony Azzam among investigators as high as many of the investigators participating in Acacia HCM have prior experience with Abbvie Camden from Redwood HCM Forest, HCM and Sequoia HCM.
Additionally, as you may know patients with non obstructive HCM have limited treatment options.
Ended up care medications, including beta blockers and calcium channel blockers are not very effective and.
And therefore patients with non obstructive HCM, often struggle with a high symptom burden.
Our hope for this clinical trial is.
To gather evidence about the potential of athey Camden for this important segment of the HCM population, which lacks treatment options.
In the third quarter. We also continued patient enrollment in Maple HCM, the phase III clinical trial evaluating the potential superiority of attic Camden as monotherapy compared to the top of <unk> as monotherapy in patients with obstructive HCM.
We're pleased to report that enrollment is progressing according to plan.
If positive naval HCM should support the possibility for Abbvie Camden to be considered for first line therapy in obstructive HCM.
Shifting to our earlier stage pipeline as Robert mentioned, we also made progress advancing both CK 586, and CK 136 during the quarter.
We've now completed the single dose cohorts of healthy participants in the phase one study of CK 586.
A cardiac myosin inhibitor in.
In development for the potential treatment of a subgroup of patients with heart failure with preserved ejection fraction or have passed.
With hyper contractility.
We've now concluded our analysis of the single dose cohorts, which are supportive of proceeding to the multiple dose portion of the study.
Which we expect to start in this fourth quarter.
As the phase one study progresses, we also recently unveiled new preclinical data for CK 586.
At our Investor and analyst day, showing improved diastolic function and reduced cardiac fibrosis in an animal model of headsets.
These data along with the results observed with Abbvie Camden in non obstructive HCM, which has features similar to that of path with hyper contractility.
Of course, the potential benefit of CK 586 in this patient population.
Shifting to CK 136, our cardiac troponin activator, we completed the single ascending dose cohorts in the phase one study in healthy participants.
And we're now analyzing these data to inform potentially proceeding to the multiple dose cohorts of the phase one study.
With that I'll turn the call over to Andrew.
Thanks, Doug.
We outlined at our Investor and Analyst day, our approach to commercial readiness in 2023 centered around understanding the patient journey and the holistic experience of living with HCM.
As such we have been focused on gaining a deep understanding of the HCM market with the intention of designing an optimal physician and patient experience.
During the quarter, we continue to conduct market research to assess potential patient profiles and available market segments in obstructive HCM.
Which if confirmed symptomatic patient population in need of disease modifying treatment with a potential next in class.
By anchoring our commercial strategy to this patient centric approach.
We're preparing to address a high unmet customer need with the objective of positively impacting patients and shareholders. The fact, the captain is approved.
At the same time, we're learning through our market research that physicians indicated a strong interest in the potential target product profile for IP Katherine.
Shoving that there is room to capture share among newly treated HCM patients.
And look to expand the total market.
Furthermore, we expect <unk> penetration of eligible for obstructive HCM patient to be less than 20% at the time of expected launch of actually Catherine meaning that over 80% of the obstructive HCM eligible patient population will remain untreated with the CMO.
We are therefore, focusing our commercial readiness strategies to those enabling expanded use of <unk> building on the expected Mexican class profile Effie Camden, arriving from our clinical trials progress.
We also will strive to design, a seamless and patient friendly <unk> experience through our comprehensive patient support program to help address the emotional financial and educational needs of a patient throughout their journey.
With our patient experience in mind, we recently held administered theater program at the HSA annual scientific meeting, where we facilitated a discussion surrounding the impact of obstructive HCM.
It may have an in patient on patient quality of life medical health and wellbeing as well as how cardiologists can better understand and address these concerns.
This approach of elevating the holistic and human centric view of HCM, it's resonated with HGTV.
And hope we hope it may help foster better physician patient conversations and raise more awareness of the myriad impacts of HCM.
Through the end of the year, we plan to continue.
Our market research and go to market planning proxy campaign.
In 2020 for our experienced team will shift focus to the design and build of our commercialization strategies as informed by market research the results of Sequoia ATM and our deepening market insights with that I'll turn the call over to Robert Wong.
Thanks, Andrew we ended the third quarter with $554 7 million in cash and investments during the quarter, we received a $50 million milestone payment from royalty pharma a pause in the start of Acacia HCM, which is treated as a liability on our balance sheet in accordance with GAAP.
In addition, recently we received a $2 5 million milestone payment from Cheesing received upon the start of Acacia HCM.
Our third quarter 2023, R&D expenses increased to $82 5 million from $62 7 million in the third quarter of 2022, primarily due to spending on our cardiac myosin inhibition programs.
Our third quarter 2023, G&A expenses were $40 1 million down from $48 2 million in Q3, 2022, due primarily to lower outside service spending now I'll hand, it over to chip to review, our financial outlook and corporate development strategy.
Thanks Robert.
As Robert mentioned, we ended the quarter was approximately $555 million on the balance sheet.
Which represents over 18 months of cash runway based on our 2023 net cash burn guidance.
In the second half of the year, we have reduced our spending and are critically assessing our go forward spending priorities to help facilitate the best return on our financial commitments.
Our main priority remains at Kempton.
And as we approach 2024.
We are being prudent in our spending around preparations for the potential commercial launch of epic Kempton.
Towards that am we.
We are getting expenses ahead of our sharing the topline results of Sequoia HCM.
Once we have the results and provided they are positive we will be able to refined our assumptions further in <unk> spending.
As usual, we plan to provide guidance to our 'twenty 'twenty four spending during the Q4 earnings call.
As for access to capital I'll remind you.
That through our transaction was the royalty pharma, we remain eligible for two additional loan tranches under our development funding agreement.
Including $75 million upon or potentially sharing positive results from Sequoia HCM.
And $100 million upon acceptance of an NDA submission for <unk> in the U S.
As it relates to corporate development over the year, we have engaged in a rigorous process.
We met with multiple potential partners.
We have noted a high degree of interest in <unk>.
Both independently and in some cases in concert with potential to include only Camden Mccarville.
In regions outside of North America.
This process has affirmed the value operation proposition to our go to Europe strategies.
With that said, we do not expect to enter into a partnering deal prior to announcing the topline results from Sequoia ACM.
And we'll continue to evaluate how we access and deploy capital as we will learn more about epic Hampton from Sequoia HCM.
Now I will turn the call back to Robert Blum.
Thank you Ching.
As we proceed to close out 2023, we're approaching an important inflection point for our company.
Without the kimpton, representing the leading edge of our specialty cardiology franchise. The topline results from Sequoia HCM will potentially accelerate our momentum as we planned transitions to become a fully integrated biopharmaceutical company in 2024 in.
In 2023, much was put into motion that is now carrying us closer to our goal of bringing forward new medicines for patients with diseases of impaired muscle function.
While our priority remains at the Kimpton and our earlier stage pipeline during the quarter, we submitted a formal dispute resolution request to the Fda's office of new drugs regarding the <unk> or complete response letter for <unk>.
Our objective is to appeal fda's conclusion that substantial evidence of effectiveness has not been established to support approval of <unk>.
As I've said before we do not currently have plans to conduct another clinical trial of <unk> and we may not be able to address the deficiencies noted in the krill. However, we still believe in the science underlying or <unk> and the demonstrated evidence to potentially benefit patients with advanced or worsening of <unk>.
<unk> failure.
If our appeal to FDA proved successful we will then consider potential next steps for <unk>.
And importantly, as through the lens of a company for which our top priority is Alfie kimpton.
We also continued to pursue potential approval for <unk> outside of the U S.
Regarding Europe, we submitted our responses to the day 120 questions to the EMA and now await their feedback.
As it relates to China, our partners using submitted a request for voluntary withdrawal of the NDA for <unk> to the center for drug evaluation of the National Medical products administration of the People's Republic of China.
Subject to potential resubmission upon receipt of favorable feedback from EMA or FDA with regard to potential drug approval for <unk> in the EU or U S respectively.
Shifting now to business development as Ching said and to be clear, we do not expect to partner out campton, leading into the readout of Sukhoi HCM. Our focus remains on that which is under our control and which is the most meaningful meaningful for our company and for patients and shareholders.
Which is advancing kimpton.
However, it is reinforcing to our corporate strategies to have engaged with potential partners, who have expressed a high level of interest in nasty kimpton and ultimately validate our own planning. We believe that we are doing right by all of our stakeholders to objectively and critically evaluate our own.
Plants.
As a result with the readout of results from Sequoia HCM will be better prepared to execute on our strategies.
As we approach the end of 2023, we reflect on a year marked with both ups and downs admittedly, we have faced certain setbacks, which tested our company's grit and resilience, but we've also made great progress today, we're uniquely positioned for success with a <unk>.
Strong specialty cardiology franchise led by our own broad late stage development program for RP Campton complemented by earlier stage drug candidates that have arisen from our industry, leading research and leadership in muscle biology, and the mechanics of contractility.
That plus our relationships with key stakeholders and our access to capital sets us apart as does our passion our dedication to our mission and are focused on doing right by patients.
I look forward to 2024 with optimism and eager anticipation for what's yet to come.
Lastly, and before I recap, our upcoming milestones I'd like to also mention that leading into the results and the readout of Sequoia topline in late December we plan to enter our quiet period, starting on Monday December four.
Now our upcoming milestones for <unk>, we expect to share top line results from Sequoia HCM in late December and continuing enrollment of patients in Maple HCM and the case for HCM and continued to advance our go to market strategies for Camden.
For <unk>, we expect to continue to pursue potential approval for <unk> in Europe.
And for CK 136, we expect to analyze data from the single ascending dose cohorts of the phase one study to inform potentially proceeding to the multiple ascending dose cohorts in that phase one study and finally for CK 586, we expect to proceed to the multiple ascending dose cohorts.
In that phase one study in this quarter Q4 2023.
Operator with that we can now open up the call to questions. Please.
Thank you as.
As a reminder 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.
In the interest of time, we kindly ask that you. Please limit yourself to one question at this time, please standby, while we compile the Q&A roster.
And our first question will come from Joe <unk> from H C. Wainwright Your line is open.
Good afternoon, Joe.
Good afternoon, thanks for taking the question.
So curious on Fridays comments with regard to.
The patient populations in Sequoia and specifically.
Any more color you can provide with regard to the background therapy reductions and more importantly, how these.
Therapy reductions might improve the quality of life and the safety of these patients.
And then secondly, just quickly you know.
Anything meaningful to discuss with regard to the cost impacts of the.
The formal dispute with the FDA and EMA filings from a captive thanks a lot.
Sure OLED fatty answer your first question and I'll take the second.
Thanks, Joe I think.
With regard to withdraw a background therapy.
The protocol.
As we reported back at ACC allows investigators to.
And we're slowly withdraw beta blockers are other background therapy, such as disopyramide or in calcium channel blockers based on patient tolerance.
And then at the same time on.
There are also allowed to up titrate assay canton as necessary.
To compensate.
And so we see.
I can't give you exact percentages, but we see substantial about two thirds I think.
Withdraw from all background therapy.
I think in most cases, they have been able to discontinue disopyramide.
And also.
And also with beta blockers as well quite successful in terms of withdrawing from those.
So I think from a patient point of view it simplifies medical therapy.
It gets them off of some of the troublesome.
Adverse events that they experienced with those drugs.
And.
It simplifies their medical regiment.
And to your second question regarding potential costs associated with our submitting the formal dispute resolution, they're really quite minimal these are costs that relate to.
Internal activities and together with Council no New studies no new.
Now, let's see nothing that would represent a meaningful investment of spending.
Thank you guys.
Thank you.
Thank you.
And our next question will come from Dane Leone from RJ S. Your line is open.
Okay.
I forgot and Robert and team.
Yeah, we've been talking a lot lately. So it really only one question for me that that keeps coming up with investors is.
Given the late December nature.
The top line and Sequoia can you just provide any detail of how you think that the team will handle disclosure of the top line data.
I think a lot of people point back to some of the early Redwood disclosures that your team made.
Around description.
A description of some.
Some of the key primary endpoints and may be some.
Detail around left ventricular ejection fraction adverse events, but anything you can tell us.
Our team plans to handle I think could be really helpful for setting expectations. Thank you.
Sure I'll do my best.
Recognizing that we don't have the data so it's hard to.
Be as clear as you might want us to be but our goal is to disclose all of that which should hopefully convey.
Our.
The ability to have achieved a next in class potentially best in class profile for <unk> and how much of that will be quantitative with P values primary and secondary endpoints safety and adverse effects.
Ultimately going to depend on that which we can.
Consider alongside of hopefully the American College of Cardiology, where we would hope to be presenting these data.
Albeit not for several more months afterwards.
So our goal will be to be communicative as much as we can but knowing we don't want to jeopardize any presentation at a proper scientific forum.
So we recognize what is <unk>.
Expected if we're going to have a next in class potentially best in class profile and those are the things that are top of mind for us as we will think about that which we can disclose obviously, we've got a disclosed that which is deemed material to shareholders.
Excellent. Thank you.
Hugh.
Thank you.
Our next question will come from Salim Sayed from Mizuho. Your line is open.
Good afternoon Liam.
Hey, good afternoon, Robert how are you doing.
I guess a question for me I'll, just follow up on that on Sequoia.
Wanted to maybe hear from you or fat.
Just so that we are all clear on.
How how we should compare maverick Hampton to to avid kimpton.
Pete.
And then on one <unk> change on on peak video too and I think there was about a 6%.
Im injection.
Injection fraction sub 50% just curious if you could just framework for US the book end low end and high end on peak for you to like is it a one point change in the $1 eight where it would be comparable or where do you actually start to feel like you can differentiate on efficacy and the same question just on safety how much different do you need to be.
Mavic Hampton in order to feel like you can actually differentiate.
And how that speak turnarounds et cetera. Thank you.
Sure Cilium. So you won't be surprised for me to again emphasize that we're not going to be making comparative statements with Maverick Hampton.
The Sequoia HCM study is a different study.
And then was the study explorer HCM that led to the approval of Mavic Kimpton, all I can speak to in all Faddy will speak to is that which relates to <unk>.
Coffee kimpton and what our expectations are ultimately labeling and positioning.
Physicians see those two potential medicines will define what will be our hope of expanding the category and best in class. So with that long caveat, maybe I'll ask Saudi to try his best to speak to what our expectations are around coffee Camden in Sequoia.
Yes.
I think what we will see and hope to see is that.
The changes are going to be meaningful a magnitude.
Such that.
A question isn't really one of.
Whether there's a slight difference between one or the other.
Both drugs are going to be well I should say mavic Camden, we know is quite effective.
And patience we hope.
Hope to see happy campton be equally or even better effective I don't think you can judge those types of things just based on comps.
Comparisons of a single number.
Really look at the totality of the data across all the endpoints you have to couple it with the safety profile.
Yes.
Again as we might.
We might see in the study and so there is not a simple answer to your question.
As I know you are hoping to provide.
Okay, No worries I just wanted to ask the question, but I appreciate the answer thank you.
Thank you.
Thank you.
Our next question will come from Jason Symanski from Bofa. Your line is open.
Hello, Jason.
Good afternoon. This is Kevin Zhang from Bank of America Securities on for Jason with Nancy Congrats.
<unk> on the progress and thanks for taking our question I'd like to ask a follow up on Es. If I may <unk> recently reported data from Floris HCM <unk> three patients had an LVL production below 50%. What do you think you need to show regarding LVL production into <unk> to make regulators comfortable permitting fewer acres.
As part of the potential rent.
Have to fall below explorer, 6% or following the death and others.
Regulators to be more cautious on the class as a whole at least initially.
Sure and thank you for the question I have read some of the research that <unk>.
A published relating to this matter and maybe I could clarify how we think about it.
We're looking at data from Sequoia HCM.
With already having.
Discussed with FDA in the design of <unk> HCM.
A proto.
Protocol, that's permitting of down titration, when <unk> falls below 50, as opposed to dose interruption or dose termination.
The consequences of down titration being well within the norm of Cardiology group practices in this area.
We approached the data when it will be available from Sukhoi HCM with those same optics.
Meaning I don't think it's really a question of it's 3% or 6% or any number within that range, but how does that really affect patients and physicians and how likely would FDA feel about what would be.
The consequence, if <unk> does fall.
We're feeling emboldened by the fact that <unk> has.
Our half life, that's enabling of a relatively rapid.
Return to normalcy with EMEA up such that if you have does drop it may be inconsequential and in that way a simple down titration would be acceptable by physicians without any concerns and worry we believe that could be very important for ultimate.
Lee the profile of Alfie Camden.
So I wouldn't dwell on the number even though as Saudi reported we've had.
Quite a low number of Es excursions and 99, 5% of echo's have not resulted in any adjustment to dose.
But for the fact that.
These down titrations might occur occasionally I would think that that will go a long way to be providing hopefully if the data and sukhoi a bear out like we've seen in forest pre.
Profile that will be deemed <unk>.
Next in class.
Anything you want to add to that.
I just might add that cardiologists are really quite adaptive managing.
Drugs that have to be monitored and if.
You think about for instance, the heart failure therapies like mineralocorticoid receptor antagonist.
These drugs have long been known to cause rises in potassium even life threatening increases in potassium and if you read the label of a player known for instance in 1% to 4% patient set that received these drugs.
And Cardoso, no stranger to drug said.
Need to be monitored so I don't think its.
Ultimately as one becomes familiar with the class that.
It's not surprising.
That one begins to see monitoring become.
Sure.
Changing over time and that it becomes just part of.
Standard practice, so I think this is <unk>.
Robert said, when we've seen to date with Abbvie campton, coupled with familiarity with the class would hopefully lead to.
Something thats, both the reasonable for patients and physicians to execute and maintain patient safety.
I appreciate the color. Thank you.
Thank you.
Thank you.
Our next question comes from Serge Belanger from Needham Your line is open.
Good afternoon Serge.
Good afternoon Robert.
Follow up on a prior question regarding the.
Data disclosure that we're expecting in late December.
The FCC has historically been pretty restrictive.
What can be disclosed ahead of.
The late breaking clinical trial presentations at their meeting soon.
Some cases.
Thank you.
<unk> heard nothing more than whether it met the primary endpoint so.
Just curious if youre confident that you will be allowed to provide.
More data than that.
On both the efficacy and safety side of the trial.
Thank you again.
Not having the data its difficult to be as clear as you might.
Ultimately want but yes, I'm confident I think we can communicate what we need to communicate to be that.
Satisfying both our objectives.
To be disclosing that which is deemed material for the benefit of shareholders and ultimately also enabling of a proper presentation at the ACC when it comes.
We've been down this path before others have too and I think this is something that can be accomplished.
Thanks.
Thank you.
And our next question will come from Aswany Verma from UBS. Your line is open.
Hi, good afternoon guys.
Good afternoon, thanks for taking our questions bottler here on behalf of Ashwin <unk> from UBS.
Just two quick ones here, we understand that you have a low beta blocker using your study from the baseline characteristics and that can help tease out the effect size wrath of Camden buttons beta blocker usage is pretty high in the real world with off account and effect size in real world potentially be lower than what Neil is showing the Sequoia study.
And then just a second quick one we wanted to get your views on what is the relationship between <unk> and reduction in peak Rio two we have some studies like one from Kansas, Iowa that show a correlation but there are other studies from beta blockers that show that there is no correlation so when do you see causality or the correlation between these two.
Endpoints when it comes to offer campaign.
Sure. So I'll just start and then ask Patty to speak to your questions.
We designed and conducted the studies to core HCM that we believe is very representative of.
The population that we hope ultimately Camden will be available to treat.
So it's not an artificial construct it's not a manufactured population. It is in fact real world and in that way, we went to great.
<unk>.
Pains to ensure that this was a study that was enabling of physicians ultimately upon the availability of data to apply these results to their own practices and with that I'll turn it to 30.
Yes, let me remind you that.
I said, 61% for the majority of patients are taking beta blockers, it's not out of line with what <unk> seen in the real world. Additionally.
Look at the use of calcium channel blockers, the allowance for combination therapy of calcium channel blockers, and beta blockers or beta blockers and Disopyramide I.
I would argue that core HCM provides.
A very relevant real world experience.
In terms of ERP captains effectiveness, when we have those data.
So I think that question.
Is really answered by the baseline characteristics that show that there is a high.
High degree of background medical therapy that is employed.
The.
I think the second point as we pointed out is that.
Background therapy may not be optimal for these patients ultimately some of these background therapies may be.
Holding them back and beta blockers being an example.
One of the reasons you see less good correlations between Lv Ot gradient reduction and peak via two is because beta blockers.
<unk> that correlation.
In the absence of being able to increase your heart rate during exercise.
Even as <unk> gotten rid of the Caribbean you can increase your exercise performance because you can increase here heart rate and so it's.
It's more complicated unfortunately than a clean scientific experiment, where you change one variable and.
Measure another variable.
But roughly there is a good correlation reducing the gradient has always been the target of <unk>.
Medical and surgical therapy.
And obviously there is precedent in the class that reducing the gradient improves peak there too.
Great. Thanks for taking my question.
Thank you.
Thank you.
And our next question will come from Jason Butler from JMP Securities. Your line is open.
Hi, Thanks for taking my question Hi, Robert.
Just wondering if you could comment on from a commercial planning perspective, how feedback from FDA, assuming positive data from from Sequoia.
Without the <unk> 'twenty for how that feedback on a rems program will impact your commercial planning and scenario.
Sure.
The rems could be similar to <unk> 10, or less burdensome the mavic Hampson thanks.
So I just want make sure I captured your question properly.
Could you repeat the first part.
Yes.
When you get feedback from FDA on a potential of what our Rems program may look like.
That would impact your commercial planning and scenarios, where the Rams was similar to that of the khamsin or less burdensome than <unk>.
Okay now I understand thank you I'm going to ask Andrew to speak to some of this but please understand that.
Absent the data.
Right now these are abstract scenarios that we've contemplated in market research and once we have their data we will be able to do more refined market research and ultimately as we may propose a rems. This is <unk>.
Something that is.
Going to be achieved through interactions.
The sponsor with FDA.
So all of this is subject to still a great deal of.
Uncertainty until we have the data.
To which it's paced, but we do have certain expectations as we have seen redwood data as we've seen forest data.
And as we have.
Understood what's behind the Rems for the other.
Cardiac myosin inhibitor, we have a view to what might ultimately be enabling of a lesser rems.
But that's still quite speculative as you can imagine at this point in time.
Andrew can you speak to how you've approached this from a market research standpoint.
Sure. So I guess theres two important things to understand wanted we probably won't have that much clarity in terms of exactly what a rems program is going to look like early in the review process.
That we proposed to the FDA, but relative to our patient support services independent of what the revenue will look like there is there are certain elements of patient support we're going to offer. So we know we're going to offer things like patient assistance and co pay for.
We'll look at Echo reimbursement will help with benefits verification.
Education on disease state. So there are certain things that we know are at minimum and that we're going to do there is other things that based on the complexity of the rems.
That will do as well.
Our assumption going into planning is that we're going to assume a complex range with all elements and it's a lot easier for us to simplify our back down from that once the Rems program that comes into view.
It does come into view, it's a negotiation as youre negotiating label near the end and we would not have enough time to react. So hopefully that gives you a sense of how we're thinking about it.
Yeah, and just to add one more comment if you read the summary basis of approval.
There's a lot that one can learn about what the FDA was seeking to accomplish.
In putting in place the Rems program that does exist today.
And we will obviously be.
<unk> based on that prior knowledge to know what we need to glean from the evidence from forest and Sequoia in order to hopefully enable something that is different.
But ultimately that's something that comes.
Down the road.
Following the acceptance and the review of a new drug application.
Okay. Thank you.
Thank you.
Thank you.
And our next question will come from shrink Crippa Devore Conder from <unk> Securities. Your line is open.
Good afternoon, good afternoon, Robert and the team. Thank you so much for taking my question.
And really looking forward to the data and about a couple of months hopefully less than that.
I have a question about maple.
Trial <unk>.
Talk about enrolling patients that are naive to therapy as well as those who were on background therapy, but.
From it can you remind me if there's any restriction in Toronto.
How long these patients could have been on therapy symptomatic and on the standard of care and how that fits into how the trial was powered.
Very good questions I will turn to fatty please.
Well I'll ask Stewart to answer since you discussed maple in his section but in general.
Patients could have been on beta blockers for any length of time.
As long as they can be withdrawn they're considered in certain classes and depending on the length of time I'll ask Stuart to elaborate please.
And thank you for the question.
Concept here was to evaluate.
You can't then.
<unk>.
First line use.
More as an optional monotherapy for patients who.
Who have been treated with <unk>.
Standard of care for.
Let's say a number of years and.
So.
We sort of subgroup these patients into.
I wonder if that's either nave to beta blocker therapy or no longer receive innovative locker therapy.
Or on beta blockers for a short period of time.
Versus those who have been treated beta blockers or standard of care for more than a year and that's sort of the breakdown.
And the strategy for evaluating.
At Camden monotherapy, either as first line.
Versus again a.
Possibly superior option.
Versus standard of care.
Is that the Camden mom.
Monotherapy or patients who had been on standard of care for an extended period of time.
So.
The study's design of course too.
Evaluate potential for superiority.
That they can't do monotherapy versus beta blocker monotherapy.
In either of those subgroups of patients.
Got it thank you.
Thank you.
Thank you.
Our next question will come from Jeff Hung from Morgan Stanley. Your line is open.
Hello, Joe Hi, Gabor Hi, Good afternoon. This is Katherine on for Josh. Thank you so much for taking our question.
You mentioned previously that physicians have indicated their interest and excitement about the Camden. We wanted to ask in either your research to date or any feedback that you've received what aspects of Abbvie can't build profile is the physician community finding most compelling.
So Andrew May I ask you to respond to that please.
Our market research, we've put an aspirational profile in front of physicians.
Things that they mentioned.
Cited about our.
Preservation of <unk> function.
Which is the safety element change in New York Heart class.
Our improvement going from a three to a two or three to one or two to one as an example, changing KCG Q and limited DDI that really don't need to be monitored relative to dosing. So those are the things that I've mentioned that are most would drive their preference for Camden.
Okay. Thank you so much.
Thank you.
Thank you.
Our next question will come from Yasmin Rahimi from Piper Sandler Your line is open.
Good afternoon guys.
Hi, Robert Thank.
Thank you so much for all your.
Comments.
I guess with data expected late December and train quite period, I guess December for like could you maybe highlight to US what is your definition of late December.
Then secondly, just maybe comment on how has been the data cleaned up and you guys have been.
Collecting and or is it just like that.
Do you start like cleaning up the data.
Thanks get locked down in early December.
Thank you I appreciate it.
You know taxable to me.
Sure. So I hope this can be helpful. But please understand as fatty shared with you while nearly all of the patients have completed.
Their maintenance phase not all of them have and there is still some additional visits that must occur from which data collection still must follow in order to be enabling of database lock.
So while we're in the very late innings of this project.
It's not yet done so we can't be as precise as you might like in asking the question about what do we mean by late December.
What we mean is that it's going to be in December.
Yes.
Whether thats.
Towards if we thought it was early December we would've said so late December probably means it's as you can imagine weeks into the month and that's ultimately going to be defined by how quickly can we get.
To database lock and the enabling of analyses.
It's not uncommon where <unk>.
Study like this it would typically take four to six or more weeks to go from database lock two tables listings and figures from which one could distill topline results were.
To try to do that faster and as fast as we can subject to.
What are still data to be collected to this point in time, we feel very good about.
Where we are with data collection, enabling of database lock and analysis.
I hope that's helpful to your question.
Thanks Robert.
Thank you.
Our next question will come from Charles Duncan from Cantor Your line is open.
Hey, Charles.
Robert Congrats on the progress by you and the team.
Thanks for taking our questions I had a couple of quick ones.
Great questions asked on Sequoia, but I did want to ask about the baseline data with regard to certain geographies and the need for action I guess Im wondering when do you think about all the patients that are from China versus Europe, or Israel are there any call it.
Phenotypic or.
Call. It behavioral differences that you think may impact call. It the read through from China versus Israel or Europe in terms of exercise.
Thanks.
Good question.
I would say ive been myself.
Engaging with our colleagues too.
I understand what could be.
Accomplished four are making this a truly international Registrational study and.
What can we learn from prior studies and what do we know so well.
I'll ask <unk> to speak specifically to your question.
Yeah, Hi, Charles.
I think.
With regards to exercise performance.
You are an athlete.
If you look at.
Our Olympic level athletes, you see they come from all over the world and performances.
Can be seen in all of those populations at an extremely high level.
Similarly, I think in studies like this.
We have the opportunity to see improvements in exercise performance across the board.
I am pleased to really buy the.
The quality of the data that we've received from all of those regions.
Have experience in Galactic for instance in terms of response Soma Campton mccarville.
And all of those regions and the response to AUM Accountemps was quite strong, particularly in China in fact.
So I don't have any concerns at the moment with regards to regional variability, obviously, we will see when the data emerge.
Charles just to add if your question is in any way tied to is there additional risk for the fact that we're enrolling in some of those regions.
I hope you can draw comfort from the fact that as.
<unk> pointed out we're quite comfortable with the integrity of the data.
The absence of missing data.
The standard deviation and those things that ultimately read on statistics. So those are things that we continually monitor and we're quite pleased with where we sit.
Yes that actually the answer is both both parts of the question both in terms of at the patient level, but at the clinical site level. Let me ask you. One last question. If you could wax wax poetic if you will Robert when you think about the vision of becoming.
High growth specialists focused cardiovascular company.
Company.
What are the analogs that you look to to really.
Be able to define success in the next couple of years. Thanks.
But I don't foresee that Theres really an analog in biopharma today, a company that is a specialty cardiovascular company.
Obviously, there are specialty companies in other therapeutic categories like in the areas of <unk>.
CNS for instance, but what we are focused towards in the build out of our pipeline and corporate development strategies are those kinds of opportunities that are directed to concentrated customer segments, where there would be both pricing and payer leverage where there's limited sale.
<unk> marketing infrastructure required to get to a high yield high return on investment strategy and we don't think the other predecessor cardiology companies in the Biopharma space had those advantages.
Order to be able to compete effectively we think they do exist in those markets and for those programs, where our science directed to cardiac muscle lends itself to a new business model in the Biopharma space and one for which we do believe there are elements that translate.
To maximizing shareholder value.
No.
Hope that answers your question.
Yes. It does he have been a good student of the history of the industry. So I appreciate you providing your perspective. Thanks.
Thank you.
Thank you.
And our next question will come from over one of release from Leerink Partners. Your line is open.
Good afternoon.
Good afternoon. Thanks, So a quick one from me I was curious if you could talk a bit more about the clinical meaningfulness of the 99, 5% of monitoring echoes that did not need dose reductions in the forest data and how does it actually compare to what physicians are seeing in clinical practice today with now the Camden.
We will address that but I hope you'll understand if we don't make a comparative statement to mavic Hampton.
But fatty could I ask you to take that.
Sure I think the reason.
We made that point in fact I know the reason we made that point in terms of the number of echos.
The number that don't require a change in management.
Is to give some flavor too.
What the burden of monitoring is.
If you do a test to monitor.
Monitor for something in.
It's only as.
A meaningful outcome in and.
Less than 5% of.
Measurements that you do.
Got to ask yourself are you appropriately deploying resources in a way that.
As efficient and are there better ways to deploy those resources that can maintain the same.
Degree of safety, but not necessarily burden the system quite is quite as heavily so.
I think coming on us to understand what the determinants are of those very infrequent events, how do we anticipate them.
And to eliminate.
What are.
99% of tests that have no clinical impact, but cost the system a lot in terms of resources time and money.
So I think that's ultimately why we made those points in our presentation.
Got it thanks.
Thank you.
Yes.
Thank you.
And our next question will come from Justin Kim from Oppenheimer <unk> Co. Your line is open.
Hello, Justin.
Robert Good evening everyone.
Sorry.
Joined a little bit late but.
Just had a quick question on the.
I guess the.
Sequoia study and inclusion of patients who are refractory to SRT.
Because of that I guess, the young woman experience.
<unk> presented a few weeks back curious like if that experience would be similar to what we saw in and explore and and any color there.
So I think I just want to make sure that.
We're using the right.
Language I don't know that refractory to SRT is the right way to put it but.
What we were pointing out with those patients who were eligible for SRT.
Pre treatment versus post treatment, but maybe thought he could speak to that.
Okay.
Yes, that's right.
These patients meet the criteria for SRT, which is that they have.
In New York Heart Association class of three or more and.
And that they have a gradient of 50 or more so.
In forest.
A couple of weeks ago.
Look again at the <unk>.
Percentage of those patients who remained eligible for SRT.
Once they started at the camp Street at Camden treatment in <unk>.
Only about 10% of them met those criteria.
After therapy.
The ones that had met him at the time of therapy. So.
So we're not necessarily <unk>.
Lucid.
Patients that are refractory refractory to SRT, they just meet the criteria for SRT.
Okay, I guess, maybe just a clarification.
I think there were patients who.
Had residual gradient following surgery.
And just wondering if.
The study includes dose patient here.
Just given that these patients whose benefit or change benefit yes, no no.
Patients that had septal reduction therapy is an exclusion for this particular trial for <unk> four.
Okay, great and maybe as a segue I mean just on.
The first presentation is there a sort of natural timing for.
In HCM cohort and when we might be able to see any.
Added long term treatment there.
And with regards to forest.
<unk>.
Yes, I mean, I think what we want to see is the <unk>.
Cohort to age a little bit.
So that we have about it.
With six months to a year's worth of follow up before we report those data.
No.
Probably see them in 2024.
Okay, well look forward to that ACC okay.
Thank you.
Thank you.
I am showing no further questions from our phone lines I'd now like to turn the conference back over to Robert Blum, President and CEO for any closing remarks.
Thank you operator, and thanks very much to everybody for joining us on this call today, we covered a lot of ground.
So I won't try to synthesize our summarized that other than to say, we do look forward to the results from Sequoia HTM and top lining them later in this quarter in late December we thank you for your continued support your interest in cytogenetics.
And operator with that we can now conclude the call.
Thank you. This concludes today's conference call. Thank you for your participation you may now disconnect everyone have a great day.
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