Q4 2022 Rigel Pharmaceuticals Inc Earnings Call
Greetings and welcome to Rigel Pharmaceuticals Financial conference call for the fourth quarter and year end 2022.
This time all participants are in a listen only mode. A question and answer session will follow the formal presentation.
If anyone should require operator assistance during the conference. Please press star zero on your telephone keypad as a reminder, this conference is being recorded.
It is now my pleasure to introduce your first speaker right theory, Rogers Executive Vice President General Counsel and corporate Secretary. Thank you you may begin.
Hello, and welcome to our fourth quarter and year end 2022 financial results and business update conference call.
The financial press release for the fourth quarter and year end 2022 was issued a short.
Short while ago and can be viewed along with the slides for this presentation and the news and events section of our Investor Relations site on <unk> Dot com.
As a reminder, during today's call we may make forward looking statements regarding our financial outlook.
Our plans and timing for regulatory and product development. These statements are subject to risks and uncertainties that may cause actual results to differ from those forecasted.
A description of these risks can be found in our most recent annual report annual report on Form 10-K for the year ended December 31, 2022 on file with the SEC any forward looking statements are made only as of today's date and we undertake no obligation.
These forward looking statements to reflect subsequent events or circumstances.
At this time I would like to turn the call over to our President and Chief Executive Officer, Raul Rodriguez Roe.
Thank you rich and welcome to the team and thank you everyone for joining today.
Also with me today are Dave Santos, our Chief Commercial officer, Dr. Wolfgang <unk>, our Chief Medical Officer, and Dean shortening our Chief Financial Officer.
So beginning on slide five.
We are thrilled with the progress we've made in growing our hematology oncology business in 2022.
We look forward to continuing this momentum in 2023.
We are committed to driving sales growth for our first approved product Saba Liza Nike P.
In the fourth quarter, we achieved our highest quarterly net product sales to date.
Alongside our international partners, we are focusing on making sure top of leases available for adult patients with chronic ITT across the globe.
Our partner T. SEC recent recently took an important step towards achieving this goal with Japan's P. M D. A approval of tower lease.
We congratulate them on this achievement.
In December 2020 to our commercial business was transformed with the approval and launch of our second product red lithium for the treatment of relapsed or refractory acute myeloid leukemia.
Video is an important treatment option.
For patients with relapsed or refractory AML and we're keenly focused on the execution on the commercial launch.
With two approved products.
<unk> sales team and a highly experienced marketing and operations team. We are in a strong position to drive sales for both Teva lease ambriz Lydia.
Beyond our commercial focus we are committed to growing our business through internal development programs partnered programs and then licensed opportunities.
Our Iraq, one for our <unk> inhibitor, our two week nine continues to progress in a phase one study in patients with lower risk Mds and we are pleased to report that we've completed enrollment in the first cohort of the study.
Additionally, we continue to evaluate post imatinib and I'll, let you go.
For follow on opportunities beyond their currently approved indications.
Alongside our partner Eli Lilly, we are advancing our buy side to a K one inhibitor and we look forward to initiation of a phase Iia study in rheumatoid arthritis as part of the first in the first half of 2023.
Our colleagues at NIH are also continuing to evaluate Boston and COVID-19, and the active four phase III clinical trial.
Now moving on to slide six.
I wanted to spend some time talking about the importance of achievements with Gardner as we expanded our hematology oncology portfolio with rest medium.
In just a few short months, we went from announcing a license agreement with forma therapeutics to the FDA approval and the launch of Ritz Lydia.
Our ability to navigate this quick turnaround is a testament to our business development.
<unk> and medical affairs teams as well as wireless commitment to patients and this is the E Mail company.
But announcing the deal in August our team begin to working diligently to prepare for the commercialization numbers like and I am proud to say that despite the early approval in December we were ready to execute on our critical priorities for lunch.
Shortly after the approval, we have the opportunity to engage and educate the medical community at the 2022 Ash meeting we were able to share a new longer term data from the Registrational phase II study of risk Lydia in relapsed or refractory AML, highlighting its durable efficacy and well characterized safety profile.
Also in December but as linear was made available in the U S by prescription and since then has been added to the NCC guidelines for AML and highlighted in blood advances with the publication of the phase II Registrational study.
While pleased with the success we've had so far we are focused on continuing execution of the <unk> commercial launch.
So with that I'll turn the call over to Dave for an overview of the quarter Dave.
You Raul.
Now I'd like to take a few minutes to discuss our progress with rents linear in the first three months of launch and then transition to our continued growth in top lease during a record Q4.
On slide eight you will see our FDA approved indication, which is for adult patients with relapsed or refractory acute myeloid leukemia with the susceptible IV H one mutation as detected by an FDA approved test.
Moving to slide nine the American cancer Society estimates that more than 20000 patients will be diagnosed with AML in 2023, and unfortunately about 11300 patients will die from the disease. This year.
In terms of the eligible relapsed or refractory IV H, one positive AML patients. Our research showed that up to 60% of patients progressing to years and for unfit patients most are refractory or relapsed within two years.
With 6% to 9% of patients on each one positive that gives us a near term opportunity to impact the lives and around 1000, New youth 98, one patients in the relapsed or refractory AML setting each year.
On slide 10, we believe Reds Linea <unk> has the potential to address many key patient and HCP need in the relapsed refractory AML space.
It is a promising new treatment targeting mutant 90, H one that has shown impressive durable responses in patients who have failed previous therapies.
As more clinicians review the demographics of a relapsed refractory patient population in our pivotal cohort.
And put that together with a complete response rate of 32%.
Our CRA train of 35% and especially the medium duration CRC or age of longer than two years.
They see the value of an ancient like brands linear in their treatment armamentarium.
Combining that efficacy with a well characterized safety profile without the requirement for cardiac monitoring it becomes even more compelling to adopt press linear in their music I D. H, one relapsed refractory AML patients.
Overall, we continue to see exciting potential to become a market leading treatment in mute 90, H, one relapsed or refractory AML and are looking forward to continuing to execute a launch plan.
Moving to slide 11, our commercial and medical affairs teams have done a great job executing our launch plans to quickly raise awareness among leukemia treaters ensure quick product availability and access for patients.
The accelerated launch momentum through the publication of critically important third party references and continue educating our field team to hone our is linear messaging.
Immediately upon approval on December 1st our website was up and within the first few days of launch we had several marketing and patient access materials available to customers online and through our field teams. It.
It was perfect timing.
Our team was at the 64th annual American Society of Hematology meeting a week later on December nine with our booth prominently announcing approval and team members interacting with important customers who were in attendance.
Then on December 22nd just over a week after ash.
Our team worked closely together have product available to stock our distribution network for year end.
And finally, we were extremely pleased that over the holidays on December 30th our distribution network shipped out our first two bottles to an account for an AML patient.
It was a meaningful way for us and 2022.
On Slide 12, you will see that we continued to make strong progress on the medical affairs front as we moved into the new year.
On January 13, we were delighted that the N P. C N clinical practice guidelines for AML were updated to include the latest didn't it as a recommended targeted therapy for relapsed refractory disease with an IV H one mutation.
This quick unanimous decision by this recognized panel of experts is critically important for formulary and prescribing decisions by payers GPS and other key accounts and prescribers.
We will continue to highlight this important recommendation in our messaging.
Also in January we were pleased to confirm coverage through our first paid claims for rez linear patients.
We continue to work with key payer and GPO accounts as well as individual prescribers to ensure that they have everything they need to minimize any reimbursement hurdles and maximize patient access to Wrestlemania.
And last month, our launch momentum continues with the February 1st online publication in blood advances of the data from our pivotal phase II cohort entitled alluded to NIM induces durable complete remissions in patients with relapsed or refractory <unk> mutated AML.
The conclusion states that.
Sydney monotherapy induce durable remissions and transfusion independence, among patients with relapsed or refractory mute 90, H, one AML with a well characterized and manageable safety profile.
The observed efficacy is clinically meaningful and represents a therapeutic advance in this molecularly defined patient population with a poor prognosis and limited treatment options.
This publication, which will soon appear in print in a well known and respected peer reviewed journal is another highly credible third party reference Ferenc linear that will be important for years to come.
And finally, just a few weeks ago, our entire commercial and medical affairs team met in person for our annual meeting we had a highly productive meeting and continued to build on the teams foundational knowledge established upon approval our team left that meaning more prepared than ever to continue spending.
Awareness of breast plenty, yet and accelerate uptake as we move into our next phase of launch.
I look forward to providing updates as we continue our raise linear launch journey.
Now on to growing sales of tower lease in IPP.
I have a few brief comments on our <unk> progress in Q4.
On Slide 14, you will see our FDA approved indication, which is for adult patients with chronic immune thrombocytopenia or C. I T E.
An insufficient response to a previous treatment.
Moving to slide 15, I am thrilled to announce that we had our best Holly's quarter ever in Q4 shipping 'twenty 196 bottles to patients and clinics, representing 23% growth over Q4 of last year.
This robust growth was again driven by more new part patient starting Tolleys I'm happy to report that we had more new patient starts in 2022 than any other year since launch.
For Q4, we achieved net sales of $21.9 million, representing a 25% increase or $4 $3 million more than the same quarter last year.
We are extremely pleased with how we ended 2022 and the solid year over year growth. We are seeing an I T. T cells. We will continue to focus on targeted clinicians to identify appropriate patients that can benefit from top of ways to grow our new patient starts beyond the record levels we saw in.
2022.
And finally on slide 16, I wanted to update you on how we are continuing to expand access for patients globally with top lease in December he say our partner in Japan announced P. M. D. A approval for <unk> for the treatment of chronic eye and T. P.
Rigel earned $20 million in a regulatory milestone with the approval we remain committed to continuing to impact C. I T P and patients around the globe with continued expansion of probably used as a commercial footprint through our partners.
Thanks for your attention and I will now turn the call over to Wolfgang to provide a brief update on our development progress.
King.
Thank you, Dave I will briefly summarize all other programs slide 18.
Let me start with lower risk Mds, our phase one study evaluating already 289 rifles, Iraq, one for dual inhibitor in lower risk Mds has completed enrolling the first cohort and is now dose escalated to enrolled patients due to the next cohort.
So far the drug was well tolerated and no adverse events of concern have been seen.
As you recall this study uses a three by three dose escalation design.
Established a maximally tolerated dose before expanding a dose level two patients to obtain additional safety data as well as preliminary efficacy information.
We believe our Detroit Miami May represent a promising new approach to treating patients with lower risk Mds.
Look forward to investigating are between nine for that in this study.
On Slide 19, you can see the overviews of our ongoing programs.
As Robert mentioned, we are primarily focused on growing our heme all the business.
With our internal development programs.
I just took you through the progress is oriented <unk> 90, lower risk Mds on the previous slide.
We're also evaluating other opportunities for both of them up and.
Moving on their regulatory success.
This includes evaluating ex U S partners to bring video to email patients around the globe.
I'd also like to remind everyone that the phase two clinical trial in.
The additional cohorts beyond the registration, though relapsed or refractory AML cohort and.
And we look forward to continued analyses and applications from this trial.
Supplementing these internal development efforts, we continually evaluate potential U S. Its essence to in license that can leverage our remote capabilities.
More opportunistically, we continue to work with Eli Lilly to advance or a five five to our rip kinase inhibitor towards a phase III study in rheumatoid arthritis and with the NIH has been continued enrollment in the exited four phase III study evaluating <unk> as a treatment for COVID-19.
19.
That concludes my summary, and I will turn the call over to Dean C.
Thank you Wolfgang I'm on slide 21 for the fourth quarter of 2022, we shipped 2417 bottles on top of already to our specialty distributors, resulting in $31 $5 billion of gross product sales 2196 of those bottles were shipped to patients at clinics.
While 221 bottles increase the levels remaining in our distribution channels and in the quarter.
Following our commercial launch of <unk> on December 22022, we shipped 64 bottles of <unk> to our specialty distributors, resulting in $1 million of gross product sales.
Two bottles of <unk>.
Were shipped to patients and clinics, while 62 bottles remained in our distribution channels at the end of the quarter.
We reported net product sales and probably some $21 $9 million in the fourth quarter of 2020% to 25% increase compared to the same period in 2021.
Reported net product sales from <unk> of $900000 in the fourth quarter of 2022 our.
Net product sales from <unk> were recorded net investment discounts charge backs rebates returns co pay assistance and other allowances $9 $7 million for the fourth quarter of 2022, a gross to net adjustment was approximately 30% and 15% gross product sales.
Respectively for toleration Lydia.
Before I move on from net product sales, let me review our expectations for the first quarter of 2023, let me start with Probably's. Despite the typical first quarter reimbursement initiatives confronting our industry such as the resetting of co pays and the Medicare Donut hole, we expect to see a small increase in bottles shipped to patients are critics.
In the first quarter of this year as compared to the fourth quarter of 2022, we expect to see continued growth in bottles shipped to patients and clinics throughout the year.
It is Libya as you know we are still only a couple of months into our launch as Dave highlighted we are encouraged by the initial interactions that our field team is having with physicians, who treat patients with AML with these interactions and as awareness of <unk> increases, we expect to see increasing penetration into this new market for rigel.
As we add new patients and as the year progresses, we look forward to providing more specific updates in the future as our launch progresses.
Mentally we currently expect our gross to net adjustment for the first quarter of 2023 to be approximately 30% fotopoulos, an approximate 15% first Lady.
Slide to the next slide in addition to net product sales our contract revenues from collaborations were $26 $5 million and our government contract revenue was $2 million in the fourth quarter of 2022.
Contract revenues from collaborations consisted of $20 million in revenue from key say related to a milestone payment earned upon Japan P. M D approval of <unk> for the treatment of chronic ITT.
$5 $7 million noncash revenue from their collaboration agreement Medicide $600000 of royalty revenue from referrals and $200000 in revenue related to our license agreements with Lilly moving.
Moving on to costs and expenses our cost of product sales was approximately $342000 for the fourth quarter of 2022, our cost of product sales for the quarter were inclusive of a 15% royalty on our risk maybe in net product sales.
Total costs and expenses were $49.2 million in the fourth quarter of 2022 versus $41 $8 million for the same period in 2020 what.
This expected increase in costs and expenses was primarily due to personnel related costs and commercial expenses, along with higher research and development costs related to the Iraq, one four inhibitor program.
As we look towards 2023, we currently expect our total costs and expenses for the full year of 2023 to be approximately a $160 million.
Finally, we ended the quarter with cash cash equivalents and short term investments of $58 $2 million with that I'd like to turn the call back over to Ralph.
Thank you Dean.
As we read on this call 2022 was a transformational year for right now.
And we believe that this positions us very well for a year of execution in 2023.
Commercial organization is focused on accelerating <unk> sales with our expanded sales force and increasing interactions.
We are continuing our commercial launch of prioritizing physician awareness and adoption as well as identify ex U S collaborators.
To supplement our commercial initiatives and business growth. We look forward to continued progress across our development and partnered programs, while evaluating new external assets to leverage our human capabilities.
We are very excited about this and about our commercial and clinical initiatives in 2023.
On a final note I'd like to take a moment to recognize that our CMO Wolfgang less stay at Rigel will be March 31.
It's getting on behalf both our team I'd like to thank you for your service and contributions to Rigel.
We are conducting a search for a new CMO with hematology oncology experience.
With that I'd like to thank you for your interest in our progress in the fourth quarter and throughout 2022.
Operator, if we could open up the call for questions.
Thank you, ladies and gentlemen at this time well be conducting a question and answer session.
I'd like to ask a question you May press star one on your telephone keypad confirmation tone will indicate your line is in the question queue.
Press Star two if you would like to remove your question from the queue.
For participants using speaker equipment, it may be necessary to pick up your handset before pressing the star key.
Our first question comes from the line of Jago knock your mortgage with Citi. Please proceed with your question.
I think this is carly on for Yigal. Thanks, So much for tea has a question we have a few on with Lydia I guess first can you talk a bit about how concentrated the prescriber bases here and obviously, it's still very early but are you able to comment on how many unique prescribers have actually written in.
Our script so far.
Thanks.
Currently thank you so much Alaska things coming.
Yeah. So currently on the second part of your question, we're not going to talk today, because it's in Q1 as you know.
The results from Q1 that will share on our Q1 call. So we won't share specific top prescribers, but I will say that you know as we've talked about there about.
And AML prescribers that we are we're calling honest targets about half of those are.
Consistent with our top of lease targets and about half of that is R. Leukemia, treaters, who really are specialized in leukemia and those are the people that can be more difficult to access I mean, it's been a little bit more difficult for us.
You know a challenging, especially since we've only been at this now since December but we're.
We're making progress.
We are confident that we will continue to grow awareness in that group.
Okay, Great. That's helpful. And then you disclosed 64 bottles shipped during the fourth quarter I guess.
Can you just.
Talk a bit about how we should be thinking about AD inventory stocking dynamics during the first quarter and the second quarter of 'twenty three.
Hum.
I can take a stab at that and there's as dean are deemed to comment as well. So the 64 quarters, all but two of those were into the inventory into the channel.
And you know we have a better sense now a couple of much beyond that in terms of that what where that will stabilize we don't really know yet new institutions are ordering product and so it's still too early to say something definitively in terms of what a stable or even trend in.
Inventories may end up we do know that that there hasnt been usage from that inventory, but oh, the dynamics are still a little bit unknown given how early it is for us.
Common stays or yeah.
Yeah. The only thing I would say currently as you know are our distribution network is we have a and S. P that chimps directly.
Two patients and then we have a distributor who said ship to in Q2, our accounts and we have seen orders go out from our entire distribution network. So.
The distributors, who are shipping to hospitals.
Hospitals and as well.
The SP shipping to patients.
And just one detail as we launch our risk Lady is at top of East one month's supply is one bottle for as Lydia two weeks supply is one bottle. So as we as we do we need to translate those 64 bottles into 32 32 months worth of worth of treatment.
Okay got it and then just a last question is I think in the past you talked a little bit about a potential frontline AML opportunity for yeah. Can you can you talk about where you stand with that and thoughts on potential timing for a registrational study.
Yeah. Thank you Carl you know resolutely has tremendous opportunities we think that it's shown some of the best data the best data in our D. H O relapsed refractory setting and that gives us a molecule that I think has a lot of opportunity within it. So we are working diligently and understanding where we'd like to go with it.
And we're looking at various segments, both AML and Theres quite a number of segments. There that are potential opportunities as well as some things outside of AML EBIT, because theres opportunities there as well and these decisions take a fair amount of interactions with the kols potential clinical.
Investigators are.
N D M D E and so but that effort a body of effort is underway.
Maybe I can speak a little bit about the timing or maybe you can say a few things when we might be ready for sale.
Yeah, I would say.
This is definitely one of our considerations are I can't share with you as I mentioned earlier that we are analyzing other cohorts from the from the first study and.
Oh this patient population is a.
Population Israel, there's a population that has been experienced with a b in Asia in the past have shown very consistent efficacy numbers I don't want to give you the actual numbers today, because it's been fully analyzing it and then presented at appropriate scientific meetings, but I do.
We think we have options and the option that you just brought up.
One of them say world.
The only other thing to add to that in terms of timing later this year for sure we'll come back to you in terms of what we'd like to do next.
Okay perfect. Thank you so much for taking our questions.
Thank you Carla.
Our next question comes from the line of young Yang with Jefferies. Please proceed with your question.
So very much as a couple of questions to at the end of last year, you had about $15 million in cash.
And Tashi burn for this he has seemed to running.
So can you talk about how you fund operations for the hearing and beyond that's number. One question. Second question is you know where the two products on the market do you have some sense of when you would be able to reach our profitability and.
My last question is as you mentioned.
Looking in can you kind of are you evaluating other opportunities for stomach.
So can you talk about what are the other opportunities are you considering thank you.
Oh. Thank you you know, let me ask Dean to to address the first two are your questions and maybe ill address those.
Sure.
So so.
I suppose to your question, but I think together here. So so we ended we ended 2022 at $58 million of cash that you noted incrementally in January we received $20 million from TSA.
So we're comfortable with our with our current cash position in the business that we've discussed today, we with respect to the path to profitability.
We really do as a company it is.
The priority for our business to achieve.
Cash flow breakeven.
And as you've seen over that over the last year, we really have made some focused efforts on cost efficiencies and leverage as we establish the HELOC business that we've described today.
We are reducing our operating expense from a about.
About $176 million in 'twenty two the numbers, we just reported down to what we expect to be about $160 million. This year I would note that over $10 million of the operating expenses I just described our noncash stock comp and depreciation so that as we look forward with.
Respect to getting to breakeven.
We haven't given guidance on our top line. So it can't be specific on Weibo weight will get to breakeven.
As we look at our revenue grows over time, we just reported.
Fourth quarter run rate results of about $90 million 90 million plus that's fourth quarter run rate.
That's really almost exclusively Tabriz, we've got you know the week of <unk> sales and they're incrementally as we look at growth in that top line in 'twenty. Three we expect continued growth in properties, we expect to now outdoors Lady.
There is video revenues net revenues for the for the full year.
And then as we've seen over time, we continue to expect collaboration revenues and.
And those can be collaboration revenues from our existing partners like Lilly She say grip all they could also be collaboration revenues from new opportunities in a couple of examples of new opportunities would be the ex U S rights for Luna said Nabors led yet as well as potential partnerships for <unk>.
IRAK four program that Wolfgang described so.
Haven't been specific on the time, it will get to breakeven, but we're certainly focused on it and pleased with the progress we've made over the year as we set up and launch into 2023.
Thanks Dean.
It's worth noting as Dean said in his comments what.
What we're seeing in tub, Alicia and so far this quarter has been very positive usually we see a downward drafts of Q1, we're seeing a small increase actually in patients bottles shipped to patients and clinics.
Just to remind you last quarter Q4 was our best quarter since launch given that we are seeing some growth on top of that Q1. Oddly Q1 is always challenging there's Q1 will be our best quarter since launch not our best quarter first quarter, our best quarter since launch so that's a very very positive.
And a testament to some of the expansion and focus from our commercial team in increasing total lease sales despite Q1 headwinds.
Oh and your third question was all other opportunities to post Imatinib, we're looking at other opportunities.
As you may see in the press release, we put a little bit of information on the opportunity in graft versus host, we havent done dural thorough and final diligence on that as the opportunity, but it is there because the data seems quite compelling. So that's one thing we might consider with foster well we have to look at the totality of where we want to invest or our political dollars in.
They're not implement their limited.
Between all those who've never been false demand there. So we're going to make those decisions and then come back to you later this year with what well we'd like to launch in terms of Registrational trials for these two drugs.
Yeah can I ask you one quick follow up how are you thinking about revitalizing the one auto immune hemolytic anemia indication.
Yeah.
We thank you for that question, we've had interactions with FCA and and what I think would be necessary and that is a de novo trial are once again and it might be a little smaller than our prior trial, but maybe not substantially less than that.
And so given that and given frankly other opportunities that we have in front of us with.
Boston Magnet, but also now with the with all the students.
Those are all opportunities that those probably would be the priority rather than warm autoimmune hemolytic anemia.
Thank you.
Thank you.
Yeah.
Our next question comes from the line of do Kim with Piper Sandler. Please proceed with your question.
Hi, Thanks for taking my question.
I was hoping to get out any initial feedback or sense of physician enthusiasm for rent Lydia and their willingness to use the drug and if you've seen any kind of pushback oh by docs I'm, having a preference for tip salvo.
For any reason any anything anecdotal.
I could give you a lot of anecdotes.
I'm actually just been out with customers as recently as yesterday, just look back this morning.
At the end of the day, Here's a couple of things are pretty you first of all when people truly understand this data and you know if you look at the blend advances publication things are in there that we haven't even begun to talk about commercially yet which.
Which is the survival data you know if you look at the survival data of the responders.
And you see where the C. R. C. R. H responders do you in terms of survival, but that has not yet reached and as a matter of fact that 18 months you have 78% of patients surviving.
Those are the kinds of pieces that as we take this publication and put it in a new piece and begin to talk to clinicians about it which we have begun doing a just very recently since it was published people become more.
Much more kind of convinced that there is something here I mean, we have been out there with a response rate, which is clearly and see our response rate, which is clearly high at 32% right but.
But they see our CRH rate is at 35% and people can look at that and say yeah. That's kind of what I get but then when you go to the duration data people have to recall and put that in context. These are relapsed refractory patients and when you when you do that and when we passionately talk.
And it can really have an impact. So yes, there are clinicians who say I don't mean to response rates with the same but you have to really dive into that duration and we think the two things that are really going to help in the blood advanced this publication or to be able to talk about survival.
Number one and then number two to be able to also address the fact that we had patients on the trial, who were on banana plaques and so those are two things that are in the publication that aren't going to just help augment our story and lastly, do you know we've just gotten in January our AVN.
C. C. N guidelines addition, and we're going to continue to do that it's a matter of fact, if you look at our web site now we have links to both the M. C. C N guidelines announcement M B, let it.
This publication, so those arent really two critical things that I think will help move, but so far I would say you know depending on the clinician.
And and how much.
Time, we get with them, we can move them.
And I will also say that among the top leukemia treaters in the country just have to remember that this is a phase two study that was done and variant view institutions in the U S. So a lot of people haven't had a chance to kind of use this product and it came on all of a sudden it is a matter of fact last night I was.
Talking about this with some conditions they weren't even aware that it was filed and then all of a sudden it was approved in December .
So this is the kind of thing we feel very firmly do that the more we raise awareness, especially among our key leukemia treaters are the more adoption working again.
Okay. That's that's great to hear.
Other question for you, but on tallies Ah Congrats on accelerating these the sales can you talk about what you're seeing is the driver.
The sales growth is it getting the product on formularies or is it your expanded sales force.
Where is the estimated market share of total leased now and have you gotten much penetration in the in the earlier lines.
So if you look at them I'll I'll take that first that last part of your question first if you look at our data and we look at it through our Ah patients that are on the product of <unk>, we actually have a pretty good percentage of our patients are on in second line.
Disease, if we look at our patients.
Through Rigel, one cure it actually makes up more than that more than a quarter of our patients right. Now. So we do believe we're making progress in earlier lines of therapy, but in terms of what.
Kind of driving new patients to the brand. It's it's all of the above I mean at the end of the day you know we have more folks out there and they are very sharp in their messaging. They know that we are looking for a new patients to start on top lease. It again, if you go to our website you see lots of.
Patient cases that we use a lot and again.
Just seeing a lot of customers over the weekend.
We're we're continuing to get interest in this drug and people are really beginning to get it. So I I really do believe it's all of the above.
No.
Certainly access isn't hurting by the way we had more commercial starts last year and then we've had before and we believe that's a direct result of the work we've done to not only do the contracting we've talked about but also raise awareness of the access and the patients have the dry so.
No.
As Dean mentioned, we're looking at a Q1 here, where normally things slow down a bit due to the reimbursement hurdles in your whole goal is to start born to patients and get over that and that we believe were making.
Making progress toward that as we're sitting here in the first part of March So it's all of those things, though and it's.
It's it's taken some time, but I just give it to you know our sales.
They're really making a difference and our market access team is really.
<unk> worked so hard to ensure that the patient set especially in commercial patients have access to scrap.
Wonderful craft congrats on the progress and thanks for taking my questions. Thank.
Thank you Don.
Our next question comes from the line of Christian Costco with Cantor Fitzgerald. Please proceed with your question.
Hi, good afternoon. Thanks for taking our question. So we were seeing some inbound interest on the sea Gvhd program. Following the data you had earlier this year.
So the question is what factors are you considering as to whether you may look to conduct further studies in this patient population and then what do you view as the potential market opportunity.
I'll take a stab at that and that's what's going to come in as well. Thank you Christian we're looking at a whole variety of things you'd look at look at when Youre looking at a potential new indication for for your drug.
That includes the patient population the indication of the competitive landscape presently the competitive landscape anticipated by the time you complete your study and file for approval as well as fit to the current pricing and things like that so there's quite a number of things to consider when you do that and edition.
Two obviously interest from Kols and Pos.
Possible sites to do that as well as F. D. A feedback on your thoughts on it and sometimes they have their own views on how things should be studied as you know so it's a complicated decisions frankly in terms of doing the study. So we are actively evaluating that opportunity overall.
Let's get into the specifics of it anything else you'd like to add.
Yeah, maybe just since you have seen the paint booth and hopefully rent. The paper you looked at the numbers of course. The first thing. We do is we look at these numbers and compare them with other compounds that are available right now approved compounds.
I've seen the numbers look pretty good.
It's.
We're all response rates or are you.
Hi, certainly favorable to currently approved drugs. So then we really have two brokers if the agency wants a registrational trial could look like once the sample size.
The exact design and that will you know if you can pick some timelines and how quickly can we do that.
And also on costs and how much how expensive this is going to eat.
From a from a market opportunity, we do see a lot of potential.
And just could be a sizable sizable opportunity in the not too distant future as far as we can tell right now and then.
Right.
I think that's that's that says it well.
Okay.
Okay. Thank you for that appreciate it and then outside of the Eli Lilly partnership what are the key things you are looking out for in 2023 related to some of your partnered programs, including your ex U S partners.
Yeah, So as you know.
I'll, let you handle that with regards to tell the lease a good progress show the product no available or soon to be available commercially in Japan. They got the approval.
We ended the year and I have to get through through pricing, which they're working on now.
Look forward to that it is available in Europe all of them all the major countries in Europe , as well as Canada, Israel and so I think in terms of partnerships. We are making very good progress with all of these are we are having discussions with all the sooner.
It was part of the license we have global rights to all the students.
The opportunities are attractive ex U S. As well. So we are in discussions with potential partners regarding debt and also not just in terms of what the economics might be but also what their contributions clinically might be as you said theres various segments in AML that are of interest.
Possibly their contributions from regards to those rather than just financial.
We are very much of interest to us.
And then the Iraq program, we are have global rights to that program.
Having a partner in place that can also help us explore the opportunity and he mark indications.
<unk> is a tremendous interest and it's a very attractive program that we're now generating data.
Which we think will be helpful. In terms of setting a partnership in place. So we're looking forward to those new partnerships and then progress with our Lilly.
Chinese collaboration with the systemic program and are entering a phase Iia study in rheumatoid arthritis, where you think is a great indications very sizable indication one with the continued need for new treatments and we're excited about their testing arc 552, and a phase Iia Ah.
<unk> study in rheumatoid arthritis, the launching in the next handful of months.
Because we will get meaningful data and data that could serve as a cross comparator to other agents currently available and things in development, which May show this product to be a very attractive product in rheumatoid arthritis. We're excited about that and they also have a CNS program associated with that that's hopefully though.
Near future move into preclinical development admin into humans.
So very good progress with group collaboration so very good progress with currently marketed products and looking forward to putting a couple more in place.
Thank you.
Thank you Kristen.
Our next question comes from the line of Gary Nachman with BMO capital markets. Please proceed with your question.
Yeah.
Thanks, Good afternoon, a few more for me on MS. Lydia So how easy is it for physicians to use a diagnostic test Brian H one mutation in AML patients is that common practice, so no issues there in identifying these patients.
And then what is axis has been like so far for as Lydia how much are you minimizing reimbursement hurdles you mentioned that in your prepared remarks.
And then how is the sales force being allocated between with Libya and heavily maybe just a little bit more on that and what else can you do to further penetrate.
The leukemia specialists for rents Lydia I, you mentioned that there were some challenges there.
Sure. So David I'll ask you I mean, just about unless you can take those and if you Miss one I'll remind you.
Yeah, So I'll take the last one first.
Gary I.
I think first of all it's a matter of time.
Some of these institutions subsidies leukemia treaters.
It takes awhile to make appointments get in there and see them et cetera, you've got whole departments, you've got not only the empties you've got a you know.
And nurse practitioners P. A L. S that you need to see so you know.
We're working all of those angles in terms of getting into C. Leukemia treaters, but we also have a you know other field team members out there we they work together across the commercial and medical Affairs R. M. S. L are out there are partnering with our.
Our field sales reps, who they know clinicians they are able to introduce those clinicians to the territory business manager and vice versa. So it's just you know taking us some time, but we're working all the angles to ensure that we get to that.
Getting contact with as many of these leukemia specialists as possible in terms of reimbursement Yeah. I mentioned that we've had paid claims our market access team our payer field team has been out there doing many many interactions with different payers and.
N P b EMS and presenting the clinical data and overall things have been very positive. We've received a number of you know.
Uh huh.
Indications that they are going to cover with a prior off to our label, which is exactly what we want and you know we we've had a number of patients get referred in to a range of one care, who have been we've been able to secure reimbursement.
But overall I would say that it's in a lot of work.
Our cross a lot of customers, resulting in a.
<unk> wins on that front so.
You know I really heard very little from customers about an issue with getting reimbursement for.
It's still early but getting reimbursement for reasonably at this time.
But the team has just done an exceptional job and.
I think.
You know the other thing that's going to happen here. If you know you do have the people who have touch this brand among kols.
And have seen really remarkable results and really believe in this really.
Believe it or lose it sitting there and you know you're seeing educational programs out there, where our clinicians are telling their peers about Ah, let it sit nampa risk linear.
And we're going to obviously do more of that as we move forward, we havent even establish our.
Our peer to peer education, yeah. So we're working on that right now so we've got a lot of work to do but I am not I'm very confident about our ability to raise awareness of what we do know is it. It's it's not this isn't a place this is the.
The drug I think its a winner and it's just about getting the word out there to the people.
I don't know.
Ignostic test how that believes that you said.
I think that is that's not an issue at all carry I mean, our clinicians can order it from any number they may happen in the institution and they make it through a third party I think more than that it's a it's the results are what they do with the results right into you know clearly in the kidney.
In the.
Academic setting they may be able to get results quicker.
And so it's more in the frontline as applicable in that are you know they may need to wait to treat that's that's the issue, but a lot of clinicians don't do that which is why we.
I believe we have an opportunity a lot of clinicians, particularly the community just move forward with the regimen and then that leaves the AR the relapsed refractory setting open to choosing a 98, one inhibitor thrive each went positive so you know.
I think from a testing standpoint.
You know, we'll continue to talk about it and you know I'm not saying, there's 100% that's going on out there right now, but it's high it's a high rate of testing and I think in the relapsed refractory setting which is.
We need to reinforce that loosening up as the drug of choice in midnight H, one relapsed refractory patients.
Okay, Great all that color was helpful. And then just on 289 so.
What will you be looking for in the overall phase one b data and in low risk Mds patients and when will that data be available or are we going to see anything any interim data are you going to wait for all the cohorts in <unk>.
What have you learned from cohort one moving into cohort two maybe a little bit on that and then at what point would you consider maybe licensing opportunities for this asset.
Oh I haven't less piece, maybe you handle the other okay.
It starts with the first dose cohort first of all it is.
Angel.
The second of these compounds, we look for safety so.
I told you with Rockwell is willing tolerating ever really know ease of any interest now.
Now I'll be going to the next cohort, which is now the first causes at really low dose with a PK profile, where we wouldn't really expect a big pharmacodynamic efficacy fixed it.
Two is approaching that range a little bit closer.
So that would be the next three patients into.
Remember all of these patients.
They all continue to be treated and this is an open label study so as the data evolves.
Cohort two.
628 days, the same safe to escalate to the next cohort, but in those patients also continue and I'd say you know what.
We'll get three months you can get a a rough handle for that Theres a major a signal.
It might be something cool tool might be something for three and once we look at you know some of the safety and activity endpoints like transfusion independence remission overall response rates things like that and what he meant to go check improvement we really expand.
A dose and add more patients and then you know I mean, you can see.
Thank you Joe.
How many patients how long do you want in order to discuss with our partner.
Whether there's a signal would be I would say probably.
Probably several patients for three to six months, depending on what the data looks and then we wouldn't be in a pretty good position to see if we have something in the audience.
So it's a.
And the lower dose groups, we will have some data from those clearly it might be more safety.
As we get into the three or four dose groups, that's where more likely we'll see some signs of efficacy after three to six months worth of dosing and that's really what the partners are probably looking at not only is it safe, but also is there some signs that this is having a benefit in this area.
To answer your question, it's hard to say because we don't know.
Which of these brokered a need to expand at plus three.
And and that enrollment enrollment is going well. So that's a good sign overall and then when we decide to do that expansion into higher dose what are the higher dose groups is not certain we will have to look at the data because it is a kind of a data driven design of the study.
But we and the partners want to see the same potential partners want to see exactly the same thing it's an exciting opportunity for this product. It's certainly a mechanism that is unique it's it's well adapted this mechanism to treating unemployment Tory conditions, such as low risk M. D. S. So we're excited about the prospect of this.
Okay, great. Thank you.
Thank you.
Our next question comes from the line of Joe parent tennis with H C. Wainwright. Please proceed with your question.
Hey, everybody. Good afternoon. Thanks for taking the question. So my first question a little more macro so I'd like to balance our deans guidance for cost for 23 of around $160 million. If I heard you correctly and your overall cost management and if we can infer that.
The commercial side of the business and the sales force is essentially right sized at this particular moment in time.
Oh, Yeah, I think I'm going to answer the right sizing of that and if there's any other comments on the guidance on the one system.
Yes, I think we are.
We have the.
People, we need and the resources, we need to drive those brands.
You know across the commercial team from operations to market access to marketing and sales.
Yeah.
I think Joe we've talked for some time now about just the leverage and efficiency and in the whole commercial organization not just the sales team, but the whole field team. So I think youre seeing that and they are in our ability to launch new ideas, we did and all that all the efforts that role described.
And then we're seeing that leverage in that $160 million expected opex for 'twenty three.
Yeah, you know the the 160 is a good number for US we had a restructuring last year, we let go some old some of our earlier colleagues in earlier areas.
We also took some real rig or two in terms of managing our cost.
So that it allows us to do what we need to do in a more focused manner I think.
It was a good practice to do that and I think a look forward to continued growth on the top line both from products. There's also from partnerships.
And then kind of a flat to downward trend in our expense line from relative to last year. So in the not too distant future to get to that breakeven point.
Okay. That's helpful and then diving down a little further into the tablet franchise I guess first.
I didn't see it on the slides are here here the number I'm sorry, if I missed it though with regard to what the current refill rate is and especially with this new number that you put out here. It's it's definitely at least the way we've been looking at it and the way you guys have been describing it you know in early proxy hopefully for the.
Post COVID-19 pounding the pavement and you as you guys have been describing it. So first you know the the refill rate and what are the competitive headwinds that you're seeing right now potentially as you you know go push into the earlier lines as well as you know any sort of blocking and tackling.
You can talk about with Griffith and other territories as they look to expand into different geographies.
So can you comment on the refill rate and post COVID-19 conditions, Yeah, I mean, we.
We Joe we have not seen any change in our persistency, we have really focused on.
Bringing new patients to the brand and then the the team does.
Continue to work with clinicians who are prescribing in their offices to ensure that they know to you know things like you bring the dose up to 150 to make sure you get that efficacy make sure you keep them on the product.
We're doing all of that but we haven't seen a change in persistency our growth has been because more and more patients are on the brand.
And so you know, we just would like to keep it where it is and we haven't seen any sign of it changing.
So I hope that helps.
Certainly in terms of access and so yeah, and then from a from a post COVID-19.
Situation, Yeah, our team continues.
I think this is part of those.
Institutions, sometimes it can be more difficult still to get into those institutions and then the office buildings, but yeah. I think we're seeing more appointments are more opportunities to interact with clinicians and especially more live conferences I think that's been a great opportunity.
For all of our field teams to be able to interact with customers I think it all helps.
Okay. Thank you guys.
Thank you George Washington comes from the line of kill Pepitone with B Riley. Please proceed with your question.
Good afternoon. This is Andy pleasure on for Calvert and thank you for taking question.
Regarding the data investigator sponsored trial in Gvhd that gets published data on how do you perceive they get pent up the competitiveness of these results compared to our other emerging agent such as fantasy is rather rack or send Axis Act until Nab and then as a follow up I know that you're still in the city.
Marine development of it.
Of this asset is this something do you think you would consider potentially developing in house or maybe through a partnership. Thank you.
I'll take a stab at that Wolfgang and I can as well.
Yeah, I think I responded to a similar question really.
Can you compare the numbers.
Of overall response rates in the post Imatinib paper.
With approved compounds do you use it as a rock for example, synthetics.
Dean.
It looks like it's in certainly in the ballpark.
The sample size is limited so we don't want to over.
Interpret the numbers, but we feel the companionship and reducing some also some substantial investigators.
Enthusiasm around the data source.
So I would say it certainly it certainly looks competitive at this point, but it's still so early in this patient population.
Maybe wanted could you come in as well, yeah, and he and I.
But I wanted to say is that we have done some research since obviously that the.
This study.
Came out and was published and we tested this it's not a large N, but clinicians you know.
That's the thing about gvhd, even though they are approved products and even if you look at the Mtc and guidelines.
You know, what the Mtc and encourage usage clinical trial and they really well you know one of the agents has a category one evidence and they don't have one preferred over the other and so we look at this is you know if we if we do pursue an angle.
This is a development area I'm. This is a space that are you know there is no clear kind of a leader in gvhd.
And so you could again I encourage you to look at MCC and guidelines.
For hematopoietic.
Transplant and you will see that in M. D. Ph D number of agents are listed both approved and not approved.
You have to keep in mind, we are looking at this as an opportunity. We havent made a decision to proceed with it at this point or are you doing and like I said earlier, we do have a limited amount of money to spend.
And our new Registrational trials for these two agents and so we have to look at all of those opportunities in and place our bets, where we think it gives us the greatest return and near term returns specifically.
Thanks, I appreciate the additional color.
Thank you Eddie.
Fair enough further questions in the queue I'd like to hand, the call back to Mr. Rodriguez for closing remarks.
Thank you operator, thank you for your questions and your interest originally it was a tremendous year in 2022 boats challenging.
But I think a year that was our best year in terms of where we started the year and where we ended the year I think.
We really transformed the company.
With the growth until the lease are really getting substantial traction and then additional brands leading up to this highly complementary product positioning us well really for this year in 'twenty three and we're executing on that and that's our commitment to you that we will continue to grow both of these products look for.
Other opportunities within these two assets that.
That are exciting, but I think a well again transform the company and continue to look externally.
In terms of opportunities there are quite a number of things there as well that we have interest in so I think 2023 would be a great year for us. So thank you for your commitment to US we aim to deliver.
Ladies and gentlemen, this does conclude today's teleconference. Thank you for your participation you may disconnect. Your lines at this time and have a wonderful day.