Q4 2023 Amylyx Pharmaceuticals Inc Earnings Call
Good morning, My name is Jenny and I will be a conference operator today.
At this time.
I'd like to welcome everyone to the analytics Pharmaceuticals fourth quarter and full year 2023 earnings conference call.
All participants will be any listen only mode.
After todays presentation there.
There will be an opportunity to ask questions.
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Please be advised that this call is being recorded at the company's request.
I would now like to turn the call over to Lindsey Alley.
Investor Relations and communications. Please proceed.
Good morning, and thank you for joining us today to discuss our fourth quarter and full year 2023 earnings.
With me on the call are Josh Cohen, and just thankfully our co Ceos, Jim Frias, our Chief Financial Officer, and Dr. Camille Bedrosian, Chief Medical Officer.
Before we begin I would like to remind everyone that any statements, we make or information presented on this call that are not historical facts are forward looking statements that are made based on our current beliefs plans and expectations and are made pursuant to the safe Harbor provisions of the private securities.
<unk> Litigation Reform Act of 1995.
These statements include but are not limited to our expectations with respect to our library O also known as Al Rio is a R. M. At zero there are three five.
Statements regarding our current and planned clinical trials and regulatory developments and the expected timing thereof.
Our business and marketing strategy and outlook.
Expectations regarding results of the global Phase III Phoenix trial of Amex. There is there are three five for the treatment of a L. S. Any impact of those results uptake Iva liberal are already or is that.
The potential to expand global approvals for Amex Theres, there are three five and a L S and other neurodegenerative diseases.
And our expected financial performance.
Actual events and results could differ materially from those expressed or implied by any forward looking statements as a result of various risks uncertainties and other factors, including those set forth in our most recent filings with the SEC and any other future filings that we may make with the SEC.
You're cautioned not to place any undue reliance on these forward looking statements and analytics disclaims any obligation to update such statements unless required by law now I will turn the call over to Justin.
Thank you Lindsey and good morning.
<unk> 23 was a transformational year for analytics. It was the first full year that our commercial product for lithium also known as <unk> in Canada was available for people living with ALS.
The first full year of launch generated $388 million in net product revenue, including $108 4 million in the fourth quarter.
We also made great progress on our global expansion built a pipeline of potential medicines for neurodegenerative disease and transformed into a profitable company.
We continue to have a steady number of new scripts being written in the quarter and our discontinuation rates and net patients unrelenting remained stable from the third quarter.
Prescribing remains concentrated with roughly 80 prescribers, mostly at major AOS centers still representing approximately half of all delivery of prescription.
We believe that we have significant room to grow with both the AOS specialist at these centers and the general neurologists.
We are proud that one year into our launch thousands of people with ALS in the U S and Canada are being treated with our therapy.
We know there are tens of thousands for people to help.
To reach them and achieve our goal of having at least 10000 people on delivery in the U S. At any point in time, we are focused on three key initiatives.
First expanding the use of delivery out with physicians that specialize in AOS, whereas the group care for about half of all people living with a L. S.
Second increasing awareness of <unk>, among general neurologists and other physicians, who care for the other half of all people living with ALS and.
And third educating on the importance of sustained treatment, what's the Olivia.
Bold with practical strategies to manage side effects.
During Q4, we began rolling out these initiatives, we expect the impact of these efforts will build throughout 2024.
On that point, we recently on boarded Dan Monahan as the general manager and head of U S commercial.
Dan is an accomplished innovated leader with more than 20 years of experience across marketing sales and market access covering numerous disease states, including his most recent position as VP of CNS marketing and portfolio strategy at Osaka.
We are excited to have Dan meeting our U S commercial organization and believe his leadership approach and experience will help us with the goal of bringing the benefits of our delivery out to many more people.
Of course, an important aspect of our launch will be our phase III Phoenix data.
We are targeting top line data for Phoenix during the second quarter of this year or before.
We are thrilled to have Dr. Camille Bedrosian, our new Chief Medical officer join Us on the call today to discuss Phoenix in more detail.
Camille brings nearly 30 years of leadership and developing successful medicines for people living with rare diseases and facilitating global access to them.
Her experience will be invaluable as we advance our mission.
In addition to our focus on our U S launch, we remain committed to bringing the benefits of delivery out to more than 200000 people living with ALS worldwide.
While the vast majority of people taking delivery or are in the U S and Canada, we have leverage early access pathways in Europe, and Israel in 2023, helping people gain access and generating revenue.
We are making progress in Japan and has met with the pharmaceuticals and medical devices agency to discuss a potential regulatory path forward.
We also continue to engage with key stakeholders around the world to identify other potential opportunities for pathways to access.
Our long term goal is to transform AOS from a disease for which symptom management as the standard of care to a disease that is treatable.
Delivery is an important tool in that transformation as it is the first and only therapy in AOS that has been shown to slows disease progression and help maintain functional independence and the same clinical trial and extend overall survival and a longer term post hoc analysis.
We have made significant progress in our first full year of launch.
We look forward to continuing our work this year to reach the many more thousands of people living with it a lot in the U S, Canada and globally that could benefit from treatment.
I will now turn the call over to Jim to discuss our financial results for the fourth quarter and full year 2023, Thanks, Jonathan and good morning.
Mentioned 2023 was a transformational year for <unk>, we delivered a strong launch for people living with ALS.
Become a profitable business and.
In addition, we are developing a significant pipeline of neurodegenerative disease and preparing for long term growth.
Now, let me turn to the financial results for the quarter.
Net product revenues were $108 4 million for the fourth quarter compared to net product revenue of $102 7 million for the third quarter of 2023 and.
$21 9 million for the fourth quarter of 2022.
The increase from Q3 to Q4 was primarily driven by an increase in net product revenues in international markets.
<unk> revenue is generated in Canada and through early access pathways in Europe.
In the U S, which represents the vast majority of our revenue our net product revenue in Q4 were slightly ahead of our revenues in Q3.
For the full year net product revenues were $388 million.
Gross to net adjustments were approximately 14% in the quarter and 12% for the full year in line with our expected gross to net range.
They were higher in Q4 as a greater proportion of our revenue came from outside the United States.
Going forward, we continue to expect gross to net adjustments to be in the range of 12% to 15% until more significant revenues begin to accrue from outside the United States.
Channel inventory levels at quarter end were as expected with approximately two weeks of inventory in the channel and specialty pharmacies similar to what we've seen in previous quarters.
Cost of sales were $9 4 million for the quarter and $25 4 million for the full year.
Presenting roughly nine 7% of net product revenues for the quarter and year, respectively due to slightly higher write offs in the quarter.
We continue to expect Cogs to be in the range of 5% to 10% of sales.
In terms of free goods roughly the same number of people in the United States received delivery of for free in Q4 as it did in Q3 through either our interim access program or patient assistance program.
Going forward, we expect free drug to remain in the range of 10% to 15% of total U S prescriptions.
Research and development expenses were $44 9 million for the quarter and $128 2 million for the full year.
The increase in the fourth quarter was primarily driven by increases in personnel expenses, our global phase III trial in PST, which was initiated in December of 2023.
The open label extension of Phoenix and.
And spend related to the advancement of other programs in our portfolio.
We expect quarterly R&D expenses to grow modestly quarter by quarter as we enroll patients in our Orion studies and continue to build out our preclinical portfolio.
Selling general and administrative expenses or SG&A were $52 2 million for the quarter and $188 4 million for the full year.
The increase was mainly driven by increases in personnel and marketing expenses.
Again, we expect SG&A to grow modestly as we roll out additional growth initiatives for delivery or in the United States and prepare for additional growth internationally should the Phoenix study would be supportive.
Our work this year led to a strong bottom line with $49 3 million and net income for the full year.
This represents our first full year profitability and is a testament to the demand that we see both near and long term.
For an efficacious treatment of ALS.
Supported by our operating profits we ended the year with a strong balance sheet, we had $371 $4 million in cash and short term investments and zero debt.
Finally, before I turn the call over to Camille I'd like to take a moment to reflect on our first full year of launch.
We're proud that <unk> has already been prescribed the thousands of people with AOS in the United States, and Canada, and our therapy is having a transformative effect on the way people with AOS retreated.
We believe we have significant growth opportunities ahead of us as we work to bring our treatment to many more thousands of people.
We're focused on delivering for people living with ALS, expanding the use of delivery or in other neurodegenerative diseases and expanding our research in the new mechanisms, even as we deliver on our bottom line.
We're in a unique and exciting position as we enter 2024.
I'll now turn the call over to Camille.
Thanks, Tim and I too wish you a good morning.
I am pleased to have this opportunity to speak with all of them today.
I am just a few months ago at an important inflection point for the company as we near anticipated top line data from the Phoenix trial.
Hey, Alex team has made great strides and redefining how people with ALS are treated.
And in developing innovative therapeutic candidates.
Neuro degenerative diseases more broadly.
Prior to analysts I was the chief Medical officer at Ultra Janet <unk>.
Alexia.
Thank you.
And now I'm excited to be a part of this mission driven ammo team as they work to change the lives of people living with ALS and other neuro degenerative diseases.
Many doctors consider Alex one of the worst diagnoses possible to get a person.
<unk> is a disease of being locked in our bodies are let leslie losing its ability to function independently, while also being cognizant of what is going on.
Everyday people living with ALS wake up in the morning, and discover they will never again independently button their shirt.
Putting their contact lenses.
First speak with their families.
They lose the ability to eat to how their loved one and talk.
Knowing that the next losses, just around the corner people living with ALS and their families living in a perpetual state of uncertainty at a loss.
We have sounded a trajectory of ALS can be meaningfully changed but let me address the first and only Alice therapy to help slow disease progression.
Maintain functional independence and extend overall survival in the same trial called principal.
With Centaur, we completed a randomized placebo controlled study.
It's pre specified primary endpoint.
This result led to regulatory approvals in the U S and Canada.
Our locations in the New England Journal of Medicine.
Karen I'll, let neurology neurosurgery in psychiatry and several other peer reviewed journals.
The Phoenix trial will provide additional efficacy and safety data in a larger population of people living with ALS and build on the robust and positive results observed in the sense of our trial.
Phoenix enrolled 664 people living with ALS and will evaluate function over 48 weeks using the same primary endpoint essentially.
The ALS functional rating scale revised for ALS FRS or.
Which is the gold standard measure of disability progression and al.
Baseline characteristics. So the population enrolled in Phoenix has substantial overlap with central also presented and counts until I tunnels from three.
The Phoenix population as I said, one of central on the baseline ALS FRS our score.
And within one month of Centaur baseline constant symptom onset.
Phoenix has the potential to be the largest phase III study in ALS to demonstrate a treatment benefit which would make a living out of the first and only therapy NLS to generate positive data in two different trials.
This would be an achievement never before seen in alloy and we are enthusiastic about what's important data from Phoenix might mean for people living with this disease.
Okay.
Moving to our R&D pipeline, we are advancing several important programs and have a number of expected milestones coming up in 2024.
We believe targeting neuronal death with delivery I'll also unless amex 35 may have applicability across other neuro degenerative diseases.
Our multinational phase III trial of Amex, 35, and progressive Supranuclear palsy, or PSP was initiated in December 2023, and.
And we anticipate top line results in 2025 or 2026.
We shared the scientific rationale on an F 35 for the treatment of PSP and detail and a webinar posted on our website in July 2023.
Turning now to our phase III Helios trial of Amex 35 at Wolfram syndrome.
The trial is now fully enrolled and we expect to report results in the second half of this year.
Wolfram syndrome also rare genetic disease that leads to a multi system failure, resulting in blindness deafness.
Diabetes.
Tax your neuro degeneration, and typically desk by early adulthood.
There are no approved therapies for <unk> syndrome.
Our R&D team has been collaborating with Washington University for many years to conduct research, which led to our decision to conduct a phase II trial.
We're looking forward to what the data from Helios with me for people with Wolfram syndrome, a community where the unmet need is incredibly high.
We are also advancing amex <unk> four our anti sense oligonucleotide candidate targeting <unk> two to IMD, enabling studies and expect to enter the clinic by year end.
<unk> two is a critical factor of axonal degeneration, which is implicated in the pathogenesis of ALS.
Camille.
As we eagerly await the Phoenix topline results, we are developing an updated clinician engagement and marketing program and believe supportive data would help in all aspects of our launch and of course, our plan in the event of supportive data will be to Reengage global regulatory agencies.
Including in Europe in support of our goal to help more people access the benefits of our Libya as quickly as possible.
And to preempt a question that may be on many of your minds Theres no early look into the Phoenix data and we do not yet know the outcome of Phoenix today.
In addition, we are continually striving to transform and improve the way AOS is treated in to this and we're working to accelerate the time it takes to diagnose a L. S.
Current data suggests that diagnosis takes about a year from symptom onset and is not a straightforward path.
Many people with ALS or misdiagnosed initially and see several specialists before they are accurately diagnosed with ALS valuable time that could be spent on a therapy that slows neuronal death.
To accomplish this we are developing a novel biomarker panel to assist in diagnosing AOS.
And we intend to provide results from initial experimentation this year.
Lastly, in addition to Camille and Dan we're thrilled that Linda Arsenals recently joined US as our new Chief Human Resources Officer. She brings over 30 years of leadership experience, including most recently from Synovia, where she was the chief Human Resources Officer, we are excited to welcome her.
Our team.
In closing we are proud of the achievements we made in 2023.
Had a strong start to our launch of <unk>.
And I've reached many thousands of people living with AOS and 2024, we are planning for Phoenix topline data anticipated during the second quarter or before which we believe is supportive will be instrumental to our growth trajectory.
We are a profitable company, which enables us to self fund, our R&D pipeline and evaluate delivery and other neurodegenerative diseases.
We have a substantial opportunity ahead of us both with our commercial launch in the U S and globally unless our pipeline.
We are intently building, a differentiated long term franchise focused on developing and delivering innovative and impactful treatments for people living with neurodegenerative diseases around the world.
Now we'd be happy to take your questions. Operator, please open the call up to Q&A.
We will now begin the question and answer session.
To ask a question. Please press star one on your telephone keypad. So would you like your question. Please press star two.
Please limit your questions to one with one follow up.
If you have additional questions you may rejoin the queue.
At this time, we will pause momentarily to assemble our roster.
Okay.
Your first question is from Geoff Meacham from Bank of America. Please ask your question.
Great. This is Charlie.
Jess Thanks for taking the question.
I guess for this year you know Covid. This part did a redesign can you just talk about what's the potential gross.
Jackson for for this coming year, and secondly can you also tell us about what's the the net.
As for the fourth quarter compared to help quiet quarter.
Thank you.
Yes, maybe I'll start on the part D benefit redesign and take a pass to Justin on the net patients as well.
And the part D benefit redesign kind of happens in two steps and Theres. Some degree of the redesign that happened. This year and then it goes into full implementation at the start of 2025. The big change. This year is that the co pay during the catastrophic phase of part D.
It's no longer in effect for patients. So I think we view that generally as <unk>.
It's positive for patients but of course, you already had robust.
Graeme to provide financial assistance or free drug when necessary. So we don't think it's going to be a major impact on the business and I'll pass adjustment that patients in our prepared remarks, we said net patients on therapy were stable to Q3, meaning it was approximately the same level of solar reported third quarter.
Okay.
Okay.
Thank you. Your next question is from Neena <unk> Garg from Deutsche Bank, Canada. Please ask your question.
Hey, guys. Thanks for taking my question I was just wondering if you could talk a little bit more about what the gross to net dynamics that impacted the numbers specifically in the U S. I need to take our gross price increase me off I'm. Just wondering if that was offset and then if you could maybe explain a little bit more about the magnitude.
Here are the ex U S sales and how that compares to prior quarters and how we should think about that contribution going forward. Thanks.
Sure Hey, Thanks Nina.
And I didn't realize you were part of Deutsche Bank, Canada, but that's good.
Me neither.
Yeah.
So yes, there really I mean, I think the gross to net you see sort of a typical fluctuations and as I pointed out in the remarks, most of the changed sort of upward trend in Q4 really came from actually the European and Canadian revenue that we saw growing.
So you know those have higher gross to nets than they did we do in the United States and so the United States, We did take a.
A modest price increase it was less than the inflation rate actually.
And really didn't impact gross to nets.
At all so those remain stable and we're not doing any contracting with insurers that hasnt changed.
And we think our access is actually really excellent.
In the U S. So related to the European and Canadian sales.
You know we announced our early access program in France in October there are other manage access programs across Europe, some free some paid.
That's a far cry from a obviously a full approval and the access that we want but of course post a successful Phoenix as Josh mentioned, we will be back and looking for a broader global approvals as quickly as is appropriate should the database supportive.
You know, we did point out that the revenue in the United States.
It was quite stable it did grow slightly.
<unk> over quarter, but the majority of our growth came from those are those programs ex U S and I think it does point to a global opportunity for us as we move forward right. We retain rights around the world for this product AOS as a disease and unfortunately strikes people equally.
All regions of the World and there is a major opportunity as I think all of you know in rare disease.
<unk>.
Treatments for the global contribution to be quite significant over time, obviously, we'll have to wait and see how Phoenix goes, but I think it portends well for the future.
Awesome. Thank you so much.
Youre welcome.
Thank you. Your next question is from Corinne Jenkins from Goldman Sachs. Please ask your question.
Yeah, Thanks, and good morning.
Maybe for me can you just provide an update on the duration of therapy, you're seeing in the U S and talk to us about some of the specific strategies youre deploying to improve that duration of therapy.
And then maybe as a follow on like when do you expect those to potentially translate into real benefit as measured by revenue.
Yes, Hi, Greg Yeah. Thank you for the question. So you know our we've been reporting on our six months.
Persistence rate.
And it's held steady.
So in the U S. We're still seeing approximately 60% of people.
Phil on the Liberty O.
Six months after initiating treatment.
And in Canada that number is roughly 80% at six months.
So I think the first thing is that we're taking all the learnings from Canada and applying those to the U S and I.
What we've found is that a word.
When you look doctor by Doctor, some doctors have much much higher persistence rates than others and I think what we've learned from Canada as well as looking at those individual doctors is that it it comes down to education. If people know that this is they are being prescribed this treatment because it'll slow down.
Theyre AOS, but I'll keep them alive longer than they're much more likely to stay on treatment there.
They're also very practical strategies for mitigating any potential side effects again, we're trying to transform a loss from a disease that has historically focused mostly in system management to one that focuses are we're trying to focus on interventions and so part of that is as things come up how do we manage them and they're very.
Arcticles ways that that we can do that and so I think in terms of when that will translate to revenue.
I think made these rec.
Recognition recently, we put many initiatives in place we now have Dan Monahan, leading our U S commercial efforts as well. So we expect all of these things to build throughout the year.
Thank you.
Thank you.
Our next question is from Michael <unk> from Evercore ISI. Please ask your question.
Hi, guys. Thanks, so much for taking my question.
Two for me number one how is the campaign.
Taking learning from Canada, and applying it to the U S prescribers, what's the reception been like so far I realize it's pretty early.
And my follow up is any thoughts or any color you could add on on when.
You may lift the restriction on prescription reporting data when might we expect that to happen. Thank you.
Yeah. That's it thank you and I'll certainly take the first one.
No.
I agree so so it's early but I think.
Earnings from Canada, the initiatives now that we're advancing in the U S. We've had very positive feedback.
Again, if you try to think about what does it mean to transform a disease that historically has focused on system manage it because that's largely the tools that have been available.
I think that in short.
It's education.
So for example.
Saying that well.
This is not a cure, but it may slow down your AOS and keep you alive longer that's that's a big deal and that changes how uneven talks about the disease.
Focusing on mitigation of any potential side effects again in the past if one has not had to manage side effects. Then you know those things can be uncertain, but again it just comes down to education and I think that the.
The doctors and nurses and therapists, who care for people with AOS I mean first of all they're just amazing people and second I think there are very very excited at the opportunity to see this sort of transformation, but I think it's important that we in and not just we but all of our partners provide there.
Right education on these things as well so I would say that I think.
The reception has been excellent as I was saying in the in the previous question I think that the impact long term on revenue, we'll we'll see continued to build throughout the year.
Yeah, Mike maybe I'll take the question on the access to the prescription data.
I think from our read of the situation, it's not atypical to.
To not have that data available in a rare disease indication, we don't have any plans to change it now.
We're really focused on making sure that we can deliver growth, particularly after Phoenix and internationally, that's going to be where our focus remains.
Great. Thank you.
You're welcome.
<unk>.
Thank you. Your next question is from Greg <unk> from Mizuho. Please ask your question.
Thank you good morning two.
Two questions if I could one.
Any insights you could give into how things are progressing our early in 2024, given like the changes are that you're trying to make from a commercial perspective and whether there's.
Any uptick.
Versus the fourth quarter trends and then secondly, I'm curious about this novel.
A L S biomarker panel that Youre developing also there could you provide some color on which exact or what types of biomarkers might be included in that panel, but would be I. Appreciate it. Thanks.
Yeah. Thank you Greg So you know.
I would say as I was saying in the previous remarks, I think that the initiatives we've seen.
Great reception from the community and it maybe break things out as I did in the opening remarks again into sort of two broad categories. I think first is physicians, who specialized in a last and I think there the educational opportunities we've seen some people who had fundamentally changed their medical <unk>.
Others have not and so I think there it's about educating on the benefits of delivery as well as tools practical tools to mitigate side effects.
And then secondly.
There is about half of people with AOS don't make it to a specialist or are not primarily cared for by a specialist and for them for many of them their knowledge and awareness at AOS is what they learned in medical school I mean, it's it's it might be 2025 years old.
So are there they're very unaware of not just our treatment than even the general the dances that the field is made so there I think you know awareness is really key and I would highlight as well that I think Phoenix is a really great opportunity in both of those segments on both the increased confidence for the specialists.
The potential benefits of Bolivia for their patients as well as the opportunity to increase awareness for generally in the medical community and on the on the diagnostic side I will definitely pass the Josh sure.
So when we thought about the AOS diagnostic I think the first thing to notice that when you think about the pathophysiology of AOS you have the neurons that go from the brain into the spinal cord and then a second set of neurons that go from the spinal cord to the muscle that degenerate and massive quantity over the course of just two or more years.
So it stands to reason that at least something something must be.
We released our measured or measurable when that process occurs. So we've collaborated with several experts and biomarkers and clinically in the AOR space and what we can tell from you know from looking is there has not really been a serious effort thus far in the field to try to develop a.
Stick in this space, there's many markers that seem to be up or down on AOS, but nobody's, taking those together towards the kind of serious effort towards making diagnostic. So we're quite excited about that program I think there's a lot of low hanging fruit there, but we'll be reporting data on that later this year.
Thank you. Your next question is from Marc Goodman from Leerink Partners. Please ask your question.
Yeah.
Hi, This is my view on the line for Mark.
Our patients who are starting unreal real live your lives.
Now earlier on in their disease or closer to diagnosis than earlier in the launch and then secondly, what's your experience been outside of centers of excellence, maybe in more community centers.
Engagement with the general neurologists, there and patience.
And high or just curious on your experience there so far.
Yeah, no great Great question. So I think over time naturally I think we'll get people who are closer to a diagnosis I think it launched we're looking at the prevalent population, whereas over time, the expectation is to get people closer and closer to diagnosis and then I think that.
<unk>, how you think about your education and persistence efforts as well.
And then on the on.
On the second question spirit experience outside of the centers, yes. So I think our prescribing remained fairly concentrated itself. So roughly 80 doctors account for about half of all prescriptions and as you might imagine those are those are the vast majority are ALS specialists. So I think you know some of our.
<unk> that we've just started kicking off and we're very excited to have Dan lead as well.
To boost general awareness in the broader medical community and so.
We'll continue to do those throughout 2024 and I think then we'll expect to see the prescriber base.
Robin.
Throughout the year, but I think it's a really nice place to start from where we have people who had fundamentally changed their medical practice. These are the AOS specialist you see.
The most people with a last so I think it's a really nice foundation to have and then we can broaden out from there.
Thank you. Your next question is from Joel Beatty from Baird. Please ask your question.
Hi, This is Ben on for Joel Beatty. Thanks, so much for taking our question.
Could you walk us through what the potential implications for revenue could be when the T. A L. S trial results are announced.
Yeah. So I think first its really important to note that these are different products in different trials.
So our drug <unk> is a combination of studying penalty iterate and tour our style and our proprietary co formulation. So it is not the same product is Tucker.
And Additionally, this is a very different trials.
Phoenix, and Centaur, where trials designed with all of the experts in the field using.
Using some of the most state of the art ways of running these trials in a controlled and rigorous way.
I'd say thus far.
Good trial, that's been run is quite different and designed as a.
Smaller smaller trial.
Primarily with kind of an academic.
Design until that was also run you know during the peak of Covid. So I think probably the broad strokes, we don't really think there comparable and that there will be no read through between the two so we wouldn't draw those.
Comparisons.
As that data comes in and I would just reiterate to relive real is the only FDA approved medication that.
It was shown to slow AOS progression on the ALS multiple rating scale extend overall survival in the same clinical trial.
And was published in the New England Journal of Medicine. So I think that's why.
The AOS specialist to be seen such excitement that's why FDA approved medications.
Okay.
Thank you. Your next question is from Ananda goes from H C. Wainwright. Please ask your question.
Yes, hi, good morning first of all Congratulates, congratulations on reaching the milestone for completing the first year of the loan she doesn't create scene.
<unk> seen the story evolve from the inception.
And being the last maybe I have two questions. The first one is related to Phoenix.
The Phoenix I think do some criteria mentioned, some definite and clinically proven L. S. Patients. So my question is how do one defined clinically problem less patient what was the rationale behind including that that group of patients and how does it change.
The we do think it's analyzed.
Yes, so maybe first.
We will try not to get too in the weeds, but first in terms of the definition. So the ela scoria all criteria. The AOS defines many different potential groups, including suspected possible possible or probable app supported probable and definite.
And the difference between those is essentially how many regions of the body or convincingly affected by AOS was possible being one probable lab support to being one plus different lab tests probable clinically probable being two and a definite being three or four the body is divided into four regions.
On the scale.
What's been shown over time is that even people with possible AOS on this criteria.
So about 98% of them have a loss so.
So it's not like this that you know, it's not actually a scale of certainty it's much more of a scale of <unk>.
Diffusion basically how how far across the body I'm AOS has progressed when we Didnt statistical modeling.
For the probable indefinite pay.
Patients does give you a more consistent and fast progressing group, which you know which is important.
And that was one of the rationales for including them in the trial, but I think the most important thing when we compare center in Phoenix on the baseline characteristics that at baseline. The two populations look very similar.
Within a point on the based on AOS epperson and within a month on the baseline time since and demands that which are two of the most important measures. When we're thinking about our cohort is likely to progress in and to perform.
Got it thank you Oh Oh.
The second question is related to the rare disease yesterday. There was this public discussion on the Biomarkers in the rare disease space Weird.
Peter marks Oh, I think it was very positive about using biomarkers for accelerated approval pathway now being in the company, where you are looking at rare diseases like goodness Wolfram syndrome, and you know there was discussions on developing a biomarker panels for Atlas.
Is there a strategy to kind of develop.
Very definitely biomarkers like surrogate biomarkers that might help you know get to that accelerated approval pathway for other rare.
Rare diseases in the pipeline.
Such as Wolfram.
Yeah. Thank you this is camille.
The question.
We actually are very heartened by chapter Mark's comments around the possibility of using <unk>.
Biomarkers for accelerated approval in rare disease, it will make a tremendous impact.
Giving the opportunity to.
Treat individuals' sooner study the disease process and.
And impact.
Particular, neurodegenerative diseases, where there is often a small window of opportunity.
And are therefore have drugs available to those individuals sooner and we do see opportunities you heard Josh mentioned, the biomarker profile for diagnosis, which can be have an impact and we're looking at Wolfram syndrome, and similar ways as we consider with our initial.
File ways of learning about the clinical biology, as well as potential biomarkers there so stay tuned.
Okay.
Okay.
Thank you.
No further questions at this time I will now turn the call back to Mr. <unk> for any closing remarks.
Thank you operator, and thank you all for joining us on our call today and for your support and we hope you have a great day.
Thank you ladies and gentlemen, the conference has now ended.
You all for joining you may all disconnect.
Oh.
Okay.
Okay.
Yeah.
No.
Okay.
Yes.