Q3 2023 Blueprint Medicines Corp Earnings Call
We have a compelling medicine in NEVA kit, we are building a market and we're just seeing tremendous results from that.
As you well know you know when you think about science and innovation you plant seeds and they they kind of come along over time right and so what I think we've been as we've been leveraging our expertise and kept biology, we've been leveraging the relationships. We are building as we brought them forward starting five years ago in the clinic and now we have this really exciting portfolio.
Oh of programs that are focused on the allergy immunology space and that has been the work over multiple years here at blueprint medicines, following the science and thinking about where we can add tremendous value to patients as.
As well as leverage our expertise and infrastructure. What we know is having a portfolio of programs requires that leverage and for us to be able to operate efficiently and we need to see that and I think you'll also see that in our Opex results as Mike said, you know over the last six quarters, we've been flat to down in Opex in a moment when we are launching a big new med.
And and in bringing multiple programs through early clinical development and so I think that's a tremendous testament to the team and how we think about priorities and I think where we sit today is we're starting to harvest that data out of those phase one studies and we're going to make and hold very high bars onto where where do we think we're going to build to it.
First to drive the longest are the most compelling long term value for for our patients and our physicians and our shareholders and we look forward to talking about that and really 'twenty 'twenty four but we were really pleased to see this portfolio come together that kristie would really just outlined in mass cell driven disorders.
We will continue to make sure we leverage our scientific knowledge, our clinical expertise and now our commercial infrastructure and relationships as we drive what is going to be really exciting growth for the company for it.
Our next question comes from Mark from with Cowen Your line is open.
Mark.
My question, just curious have you already.
<unk>.
Sure.
Great.
Hum.
Okay.
Oh, sorry.
Susan.
Right.
Okay.
Yeah. Thank you so much for that question and you know what I'll just make one comment before handing to Philina, we've really been seen an impact in ISR diagnosis since we have been in the field.
And really developing a vacate in N I S. M. If you'll remember back in 2019, we put out that about 11000 patients in the U S had been diagnosed with without them and now we're well above 17 or 18000 at this point and so I think that's the results of both the development of advocate over the last number of years and now also the commercial.
They sit and where we're going to continue to see no patients being diagnosed with with this disease. We actually think the opportunity is probably bigger than anyone had expected.
Do you want to take some of that.
Yeah. Thanks, Alex for the question. We're so proud of our results in the third quarter and we're just getting started I think Kate address some of our efforts to increase diagnosis rates over the past several years, frankly, then and we've seen that continue to increase.
I think the most important point is that the already diagnosed prevalent patients represent a blockbuster opportunity and so our efforts are primarily focused on converting those already diagnosed not well controlled patients I think our results this quarter and the breadth of very positive leading indicators.
Make us very confident that there's significant headroom to grow within this patient population alone.
Increasing the diagnosis rate further increases be upside above this opportunity and we're more than confident we will get there.
Right.
Our next question comes from Yang with Jefferies. Your line is open.
Thank you great quarter congrats.
You know you added more than 200 patients in turned a corner compared to second quarter. So is it fair to say that you are adding about 100, a new page ISR patients per month now and are you seeing growth from there. Thank you.
No it really doesn't do anything.
Yeah. Thanks for the question you're in and you know we're very pleased to have added over 200 patients on therapy in the prior quarter and to your question I would say we are very confident in continued strong and steady growth.
Every time, we we see an opportunity we see multiple opportunities behind that is we had a chance to get out in the field and I personally have had a chance to engage hundreds of customers alongside our field teams and so we see significant headroom to be able to grow.
It's important to also remember that this is a rare disease launch where we are building a market one patient at a time and that's certainly what our teams continue to be focused on.
So you mentioned you know.
On the second quarter call.
In June you added about 35, ISR patients and that trend was a continuing through July so he's simpson like uptick.
Kagan Ah patients.
In in August and September must have been pretty significant.
Significant.
Yes. This is Kate.
When we were talking about the Q2 results one of the thing because it was such a stub quarter. We you know we had just gotten approve we only had a few weeks of launch under our belt.
We did is provide try to help provide a little bit of framework any kind of qualitative discussion.
A discussion around what we were seeing in July because of that we won't really comment on once we're not we're in a steady and at a place where we're seeing full quarters of launch where I can really comment on on kind of quarter. The next quarter's launch we do look forward to providing guidance on the Q4 call here, which is going to be in short order.
Will be inclusive of I S. M for all of 2024, and so I think it's cleaner side. We can expect that continued kind of strong and steady growth. We we're really pleased about where we are and all of the signs we're seeing in the marketplace lead to you'll point to.
Continued strong and steady growth.
Our next question comes from Brian <unk> with Stifel.
Line is open.
Thank you and congrats on a great quarter maybe.
Maybe first just a quick spin on unions question can you comment on how many patients were added specifically in September.
More broadly as you plan to provide guidance in February what elements of your thinking or assumptions behind your forecast will be most important as you watch the commercial metrics evolve over the next three months. Thank you.
Yeah, I'll just take the first piece because I know you did I would know while we while we will not break out kind of patient numbers by month, we'll continue to report them in a quarterly basis, and then maybe Selena do you want to talk about how you're thinking but the most important drivers for revenue growth in 2024, yeah. Thanks, Brad for the question I mean, some of the key leading indicators.
We've emphasized on this call that we see is most important for signaling continued growth in <unk>.
Include the number of patients on therapy, which we see as a strong predictor of continued revenue given the potential of these patients to remain on therapy for chronic durations. In addition to the new patient starts that we expect to continue to drive by increasing both the breadth and the depth of prescribing across our prescriber base.
Our next question comes from Ren Benjamin with JMP Securities. Your line is open.
Hey, good morning, everyone and thanks very much for taking the questions.
On a great quarter.
Maybe can you talk a little bit about the penetration reaching into alagoas versus tmall.
Kind of.
<unk>.
Solutions.
Oh.
215 ships that were added.
I think you've been asked this multiple times I'm going to try it before.
How many of the ISI am versus.
Okay.
Would we expect a similar.
Think about it in Europe.
Ultimate approval in Europe should we be thinking about it.
Trajectory.
Thank you so much for the question Ron I Weep, you broke up a little bit there at the end, but I think it's it's really a question around.
The patients we added and how can we think about the breakdown between different parts of Assam advanced versus I S. M.
And you know as we've mentioned before that's very hard to tease out because of the common diagnosis code, but I'll certainly let something that continue to provide detailed there I also think you asked a little bit about European approval and then how do you think about the breadth and depth of prescribing. So maybe if you could take it would take those.
Yeah. Thanks for the question so of the 200 patients I mean, I think the bottom line is the inflection point that we've seen in this past quarter is primarily driven by I S. M. We can see that by the vast majority of patients who started on the 25 milligram dose your question on the.
Prescriber base, we are really pleased with the mix of prescriber base that we were seeing we saw that big step up in the number of new prescribers that and importantly that occurred among Asia. The allergists, who are important because they tend to see a greater concentration of ISR patients.
And secondly, agnostic of specialty we're seeing that penetration into new prescribers, who are just seeing really the deepest concept, but the biggest concentration of SM patients who have the biggest potential to deepen over time. So in sum, we see a really healthy mix in terms of our prescriber base as well as headroom to.
Continue to grow on that breadth and depth.
And then just to comment on the EU EU approval, because you're going to talk about where we are with US sure and so we are looking forward to approval in Europe that review is is on track and I think around your question was kind of higher level of regarding the opportunity and how we see it unfolding in the U S versus Europe, and you know what.
I would say is I think what the U S experience has shown us at this point is that these patients are there the unmet need is significant the receptivity to the profile has been incredibly positive.
And you know Eva Cat is is really transforming patient lives and there is we expect to see very similar trends as we launch globally and ethylene are sad the dynamics that we see in the U S alone based on the market as it sits today get advocate to a blockbuster.
A status and so you know we see opportunity to continue to expand that through geographic launches by growing the market in the U S et cetera, but the bottom line is this opportunity is unfolding exactly as we had expected and we are well on our way to making <unk> a blockbuster in S. M.
Our next question comes from solving Richter with Goldman Sachs. Your line is open.
Good morning, Thanks for taking my question, maybe pivoting here to ash as we look to the 263 data could you help us understand exactly what will be presented and your view here and what would be a competitive profile in the context of advocate, but also competitors. Thank you.
So thank you sell being.
Christy really laid out is <unk> is positioned to help us maximize the long term performance of our <unk> franchise is <unk> is really meeting the broad medical needs across the spectrum of us and so the solution for those patients are truly in the market today, but you know I don't know how they're going to have a great space in our portfolio as we think.
About building that franchise for 10 years and beyond and so back or do you want to talk about what we'll be showing at ash, yes sure. Thanks, Just a reminder that we.
We're here to make a difference in patients' lives for years to come by improving their symptoms and ISO.
Eva has set a tremendously high bar.
In this part of the disease.
Another reminder, is that <unk> are all equally potent against <unk> 16 V and for any new agents coming in it's necessary to demonstrate broad symptom impact that doesn't come at the price of a worst safety profile.
And as a reminder, 25 milligrams of Eva This was evident with no grade three adverse events in part one and an amazing safety profile that we saw in pioneer.
And as Keith said, a point of the <unk> program is to expand and enhance our leadership in SM and really address all parts of the disease.
Ash will show data showing that <unk> is active and safe and in patients with indolent systemic mastocytosis and.
Raising candidate for development in this space.
Our next question comes from Michael Schmidt with.
Guggenheim Your line is open.
Hey, guys. Thanks for taking my question and congrats on that great of a kit.
Sales this quarter.
Perhaps a slight follow up on the prior question at an estimate.
Sure.
What are your latest thoughts on next development steps after ash, how should we think about the opportunity for this drug beyond what <unk> already addressing.
And then secondly also on the pipeline wondering how we should think about the syndication program at this point I know you're very excited about it and I was just curious.
Understand.
With the study there.
Yeah, Michael Thank you for those questions Christy if you take the question on S M and L. A that's good and then back on when you weighing on syndicated sure. So thanks, Michael for the question the role that <unk>.
Plays in our portfolio as I said is really extending and expanding our franchise over the long term Ava kit is very much the cornerstone.
And the bar for any new therapy is <unk> and we're very acutely aware that that bar as I said get higher everyday that we're in the market. We are excited about our potential to develop a <unk> based on the deep understanding that we have of this disease.
Now we can bring it forward in clinical development to develop a data set that is bespoke.
And what really speak to the role and the efficacy and safety of that that this therapy can play an S. M again over the long term. So ash, we really see as you know a first step in demonstrating that the molecule is safe. It's active how we bring it forward will unfold frankly in months and years coming out of.
Ash based on the knowledge that we have and we think that we are and frankly, a unique and unparalleled position to be able to develop a next generation therapy an S. M based on the proprietary knowledge that we have.
I think the CDK do sure.
So as you remember at <unk>, we presented data showing the unique tolerability profile of <unk> compared to others in the field.
Very little impact on hematology.
And any other side effect profile really that would be limited combination with pharmacyclics. So the main focus is in breast cancer and that's that's what really drives the value for any CDK to program we.
We have we believe that we've closed the gap with Pfizer and the feedback we're getting from investigators is that the <unk> is really leading the pack right now with great Tolerability data and also we've shown the efficacy starting to emerge we've shown a response in breast cancer and we continue to enroll patients.
In breast cancer, and other indications where still in dose escalation.
We have begun the combination as we indicated and we look forward to sharing more data next year.
Our next question comes from Derek called Sheila with Wells Fargo. Your line is open.
Hey, good morning, and congrats on the progress thanks for taking our questions.
So it was very interesting commentary on the 2024 Opex I just wanted to understand do you see continued rationalization and prioritization beyond 2024, meaning it is out your spend.
Become a lower and can you get more aggressive on that 100 million as you indicated.
For 2024.
Yes, thanks for the question.
We will be providing more view on how we're thinking about operating expenses in a kind of a more official weigh on our Q4 call.
Really I think what Mike was trying to talk about is just the fact that like we look at consensus out there that the numbers are really well off where we're our plan is.
And I think you'll see.
Where we stand when we see the the ramping revenues at <unk>, and we think not to about 'twenty 'twenty four but this path to $1 billion market opportunity in north of that as we continue to launch we see a really compelling profile for the company might be able to provide some more color yeah I think that's it.
Keith's right like the commentary we provided was specific for 2024 guidance because we think that's just well off consensus sorry, if that's what you're trying to break consensus if we think that's well off base.
We're obviously not providing specific guidance on 2024, Opex right now, but fundamentally it's going to come down versus 2023, and I think importantly is our total cash burn is going to come down significantly in 2024.
These are the guidelines that were pointing to right now we will in early 2024 and spend more time talking about our capital allocation strategy and I think that will give more color as to what Kate was saying how do we think about the longer term investments that we'll be making into portfolio. How do we think about partnerships and I think it's really exciting, but we feel we're in a very strong place financially in 2024 and beyond.
Yeah.
We now turn to deal with.
Your line is open.
Hi, Good morning, let me add my congratulations to the great launch our ISN.
Perhaps if you could add any color around where the patients are and maybe concentration of patients.
Within physician offices, I think you mentioned briefly that you.
You're starting to see prescribers that have the potential for meaningful depth of prescribing.
Maybe you could give us some color around.
Kind of how concentrated are these patients are that would be helpful and just a follow up on it and that's good.
Can you talk about kind of.
How much of a duration of data in a number of patients.
Expect to see.
At the Ash update.
<unk>.
Yeah.
Give the color on the patients they will come back to your question I know nothing about there.
Yeah to your questions on on where the patients are one one construct that we've used.
Over the past year or so is this notion of the top 350 providers, who see the highest volume of SM patients and beyond that it it tails off and so that is a classic kind of rare disease distribution and if anything that distribution and the results that we've shown this past quarter.
Our very strong and healthy mix for not only this quarter, but continued growth.
And so our penetration in terms of early adoption includes many of those top 350 providers and then importantly, we also see that the overall opportunity tends to be more concentrated among the allergist immunologist, we're really pleased to see the step up there as well.
And then your question about <unk> and what to expect the ash them I mean, we will be selective about the data that we disclosed just given the competitive environment and as Christian was mentioning we have numerous insights based on our proprietary data that will allow us to drive innovation for the future state of Iowa.
Not where <unk> stands today and so we certainly don't want to provide too much of that information you know out into the marketplace given the competitive environment. So we will be sure to show. The data showed that <unk> was a very compelling safety and activity profile.
Okay.
Our next question comes from Michael <unk> with Morgan Stanley.
Line is open.
Good morning, and thanks for taking the question and congrats on the strong quarter as well.
Maybe just a follow up on <unk>, given the strong trends youre seeing in <unk> I'm, just curious what youre seeing in terms of disease severity and if youre starting to capture some of those mild patients already early in the launch here. Thanks.
Thank you Michael Good question, we plan to do it.
Yeah. Thanks, Mike for the question. So on the first patients that were seeing the early adoption in for Ava cat and not surprisingly are those patients who tend towards the more moderate to severe part of the ice on spectrum, who are who are not well controlled.
And we're really encouraged to see them.
And in some of our prescribers, who have had their first very positive experience in those patients. They are they are already starting to broaden into additional patient types, who might just have one.
Prevalent I S M symptom, which is having impact on their on their quality of life. So all of these signs make us very confident that we're going to see continued growth. Both in terms of the moderate to severe patients as well as broadening into those milder patients as well.
We now turn to Joel P. J <unk> with Baird. Your line is open.
Hi, This is Ben on for Joel Thanks for so much for taking the question I guess, maybe building on the previous question are there any particular aspects of the symptom scale that are driving growth.
Yeah. Thanks, Thanks for the question Ben I think what we're really seeing in the real world experience. It's not so literal like that rate providers aren't necessarily thinking about the TFS that that was important contract for approval, but the reality in clinical practice.
Is it the <unk>.
Concept of a patient who is not well controlled patients who have been on symptom directed medications and that field to to control the symptoms and so it's it's a it can be any breath of symptomology.
Across the spectrum of of I S M, but very practically the resounding theme I've heard from disengaging literally hundreds of customers at this point is with patients who are still symptomatic.
I think I think Ben that's where this the Eva Eva ability to broadly impacts symptoms caused all different symptoms that enables.
Physicians to consider this medicine for any patient who presented in front of them right. This is a very heterogeneous disease patients who may be bothered by all symptoms to a certain degree there are patients who maybe have one symptom that really limiting their ability to kind of operate in their life and what we see where they've occurred both from the pioneer study resolved, but now in the railroads.
Is that broad symptomatic.
Impact with such a well tolerated safety profile is very compelling and I think it's certainly what is helping drive the the usage of other kept broadly.
Hum.
Our next question comes from Peter Lawson with Barclays. Your line is open.
Great. Thanks for taking my questions.
The additional 250 patients where those exclusively.
Patients with <unk>.
You can say about that.
Awesome, and just revenues and patient growth.
Sure.
Great and then anything you can say around.
How the pace of growth changed over the quarter.
Patient.
October.
Thank you.
Yeah. Thanks, Peter for your question.
As I said, we won't be commenting on kind of forward looking months or quarters, but you certainly I'm pleased to.
To talk about just that that how do we think about those 215 patients and we saw that how you think about that mix between that says I'm just denies them right and so the inflection that we saw in this quarter was primarily driven by by I S. M.
And we expect that to be the primary value driver going forward for <unk> to achieve that over blockbuster opportunity.
Within advanced SM, we certainly continue to have headroom to grow there as well and we've talked about the sort of growth at a more moderate pace, but that vast degree of headroom is among I S. M.
We've talked about that that remains a steady contributor to our Ava kit next but again, it's something that's our primary focus to reach that that full potential of advocate.
We now turn to David Lebowitz with Citi. Your line is open.
Well. Thank you very much for taking my question. When you look at the new patient scripts across the doses could.
Could you give us a baseline a comparison of what that particular chart would've looked like before the ISR launch.
Okay. So you want to take that one sure. Thanks, David So you know we've been clear that prior to prior to I S. M approval them, you know the business and S and whats coming predominantly from a dance that we were aware of what we thought was you know a low volume of.
<unk> off label use in I S. M that remained quite frankly very consistent all the way up until approval. So you know the vast majority of advanced SM patients as I said, starting at 200 milligrams. We certainly see you know dose modification as needed and I think the important point here is that the range of doses that we have for Ava cat.
Is you know an incredible strength, we know that symptom control is the golfer I S. M. 25 milligrams is an incredible dose strength that delivers that symptom control with an incredibly positive benefit risk profile. So we're seeing you know the vast majority of ISR patients at that dose, we see advanced SM patients starting at 200.
But we have patients in that gray area in the middle and we understand that our area of S. M I think better than anyone.
We know that patients can you know evolve over the course of their disease and so the fact that we have this range of doses that really allows physicians to customize treatment for the clinical characteristics of the person sitting in front of them.
Is an incredible strength of the product profile that are you know, we're really getting very positive feedback on.
Our next question comes from Chris Raymond with Piper Sandler Your line is open.
Hey, Thanks, and congrats from us on the great progress.
Two questions, maybe just on the prescriber base. So allergists now at 20 or 25% of prescribers, where do you guys think that mix with allergists and he marks.
Funds of spaces in terms of the sort of.
The mix between the two.
And then also maybe where immunologist sort of sit in there and then on the guidance for <unk> for 2023 I think this is the second quarter you guys Havent sort of readdress that initial guidance or I guess the guidance that you last issued in May.
You seem to be on a pretty decent pace to sort of beat that number just kind of curious why not address that from here.
Yeah. So let me David let me take the guidance piece first and I'll hand over to fully now to talk talk more about the commercial dynamics. So from a guidance perspective. The guidance. We gave at the beginning this year was solely for the existing business at that time, So that was advanced SM and just.
I think we were really clear about that at that point that we were not going to include ISR, because it's a launch year and in January we are anticipating approval on the <unk> date in May but again, you just really don't know how those things are going to come together and so we gave guidance for this year is focused on the advance us and them in the <unk> business.
The guidance, we will provide next year on the Q4 call will be inclusive of <unk> as well and so therefore, you'll you'll get guidance on the full brand at that point.
Even though it's still early in the launch you know we're committed to providing that if we can.
Lodging iPhone excuse me, we're committed to providing that at that point when do you want to talk a little bit about the commercial dynamics, yes to your question about the prescriber base and again I think we're off to a very strong and healthy start in terms of the step up in new prescribers that we saw in this quarter, we expect that to continue to grow and that Theres a lot of headroom to continue to grow that both among.
The allergist immunologist. So we do expect the 25% contribution to continue to increase.
And we're just getting started right you know the growth that we've seen represents just a really small fraction of the overall providers, who are seeing ISR patients and there's a lot of headway to continue to grow the overall patient penetration.
Our next question comes from Matt Biegler with Oppenheimer. Your line is open.
Hey, guys.
Congrats for marketing as well.
Apologize if this has been asked before but do we have any early insights into the patient retention rate.
Our patients generally continuing to refill their scripts, let's say after the first month or two on treatment or.
Or is it still too early to tell.
Yeah. Thanks, Thanks for that question I mean, we again, we believe that the durability of treatment and <unk> is going to be one of those great drivers of growth between if you want to talk about what we're seeing today.
Yeah, So again reiterating <unk> point.
Certainly see potential for chronic dosing given the very promising benefit risk profile of a cat.
Still very early in the ISR launch, but we're really pleased to see the very high compliance rate of the patients who have come on and that they are refilling their therapy, we expect that to continue.
This concludes our Q&A.
And box of K 12 months.
Closing remarks.
Thank you operator, and thank you all for taking the time to join US today. We are very excited about the launch of <unk> and I S. M and believe it does represent one of the most exciting and attractive launches in rare diseases today and so we appreciate your continued support and look forward to additional.
Updates coming forward next year.
Ladies and gentlemen for today's call is now concluded. Thank you for your participation you may now disconnect your lines.
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