Q4 2023 Amylyx Pharmaceuticals Inc Earnings Call

Participants

Neena Bitritto Garg; Analyst; Deutsche Bank

Corinne Jenkins; Analyst; Goldman Sachs Group, Inc.

Michael Cherny; Analyst; Evercore ISI Institutional Equities

Graig Suvannavejh; Analyst; Mizuho Securities USA LLC

Andrew Fein; Analyst; H.C. Wainwright & Co., LLC

Presentation

Operator

Good morning. My name is Jenny, and I will be your conference operator today. At this time, I would like to welcome everyone to the Analex Pharmaceuticals Fourth Quarter and Full Year 2023 earnings conference call. All participants will be in a listen only mode.
After today's presentation, there will be an opportunity to ask questions. (Operator Instructions) 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 Allen, Head, 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 Justin Klee our co-CEOs, James Frates, our Chief Financial Officer, and Dr. Camille Bedrosian, our 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. It remains based on our current beliefs, plans and expectations and are made pursuant to the Safe Harbor provisions of the Private Securities Litigation Reform Act of 1995.
Statements include, but are not limited to our expectations with respect to our delivery, also known as Albireo's or NAMAC 0035 statements regarding our current and planned clinical trials and regulatory developments and the expected timing thereof business and marketing strategy and outlook patient regarding results of the global Phase three clinical trial of AMX0035 for the treatment of ALS and the impact of those results on uptake of delivery are Albireo's that the potential to expand global approvals for Amex 0035 and ALS 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 are cautioned not to place any undue reliance on these forward-looking statements.
And AMRIX 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. 2023 was a transformational year for Analex. It was the first full year that our commercial product for Livio also known as LBOs in Canada, was available for people living with ALS.
The first full year of launch generated $380.8 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 continued to have a steady number of new scripts being written in the quarter and our discontinuation rates and net patients on reveal remained stable from the third quarter. Prescribing remains concentrated with roughly 80 prescribers, mostly at major ALS centers. Phil representing approximately half of all delivery of prescriptions.
We believe that we have significant room to grow with both the ALS specialists at the centers and the general neurologists. We are proud that one year into our launch. Thousands of people with ALS in the US 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 10,000 people on delivery in the US at any point in time, we are focused on three key initiatives. First expanding the use of delivery with physicians that specialize in ALS, who as a group care for about half of all people living with ALS.
Second, increasing awareness of Relovair, among general neurologists and other physicians who care for the other half of all people living with ALS. And third, educating on the importance of sustained treatment with Reveal, coupled with practical strategies to manage side effects.
During Q4, we began rolling out these initiatives, and we expect the impact of these efforts will build throughout 2024. On that point, we recently onboarded Dan Monahan as the General Manager and Head of US Commercial. Dan is an accomplished innovative 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 leading 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 three Phoenix data.
We are targeting top line data from 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 in 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 US launch, we remain committed to bringing the benefits of delivery out to the more than 200,000 people living with ALS worldwide. While the vast majority of people taking delivery of our in the US and Canada, we have leveraged early access pathways in Europe and Israel in 2023, helping people gain access and generating revenue.
We are making progress in Japan and it 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 ALS from the disease for which symptom management is the standard of care to a disease that is treatable with Livial is an important tool in that transformation as it is the first and only therapy in ALS that has been shown to slow disease progression and help maintain functional independence in 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 many more thousands of people living with the ALS. in the U.S., Canada and globally that can 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, Jeff, and good morning. Has mentioned 2023 was a transformational year for analytics. We delivered a strong launch for people living with ALS, we've become a profitable business. In addition, we're developing a significant pipeline in 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 at $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, including Revenue 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 $380.8 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 at 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, representing roughly 9% and 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 receive delivery of for free in Q4 as they did in Q3 through either our interim Asyst program or patient assistance program. Going forward, we expect free drug to remain in the range of 10% to 15% of total US 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 three trial and TST, which was initiated in December of 2023, the open label extension of Phoenix 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 study and continue to build out our preclinical portfolio selling general 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 rollout additional growth initiatives for delivery in the United States and prepare for additional growth internationally should the PHOENIX study be supportive. Our work this year led to a strong bottom line with $49.3 million in net income for the full year.
This represents our first full year of 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 really Rio has already been prescribed to thousands of people with ALS in the United States and Canada and our therapies having a transformative effect on the way people with ALS are treated, we believe we have significant growth opportunities ahead of us as we work to bring our treatment to many more thousands of people were focused on delivering for people living with ALS, expanding the use of Rio and other neurodegenerative diseases and expanding our research into 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, Jim, and I want to wish you a good morning. I am pleased to have this opportunity to speak with all of them today. I joined analysts just a few months ago at an important inflection point for the Company. As we near anticipated top line data from the PHOENIX trial, the Atlas team has made great strides in redefining how people with ALS are treated and in developing innovative therapeutic candidates for the treatment of neuro degenerative diseases.
More broadly prior to analytics, I was the Chief Medical Officer at Ulta Agennix Alexei on an area. And now I'm excited to be a part of this mission driven analyst team as we work to change the lives of people living with ALS and other neurodegenerative diseases. Many doctors consider ALS. one of the worst diagnoses possible to give a person.
Als is a disease that being lost in a body relentlessly losing its ability to function independently while also being conscious of what is going on every day. People living with ALS wake up in the morning and discovered they will never again independently button their shirt, putting their contact lenses, brush their teeth first speak with their families.
They lose the ability to eat to how their loved one and to walk knowing that the next losses just around the corner, people living with ALS and their families living in a perpetual state of uncertainty and law, we have shown the trajectory of ALS can be meaningfully changed Bolivia is the first and only ALS therapy to help slow disease progression, maintain functional independence and extend overall survival in the same trial called Central with Central, we completed a randomized placebo-controlled study that met its prespecified primary endpoint. This result led to regulatory approvals in the US and Canada publications in the New England Journal of Medicine, the Journal of Neurology, neurosurgery and psychiatry and several other peer-reviewed journals.
The PHOENIX trial will provide additional efficacy and safety data and a larger population of people living with ALS and build on the robust and positive results observed in the central trial, PHOENIX enrolled 664 people living with ALS, and we'll evaluate function over 48 weeks using the same primary endpoint as central the ALS Functional Rating Scale revolves for ALSFRSR, which is the gold standard measure of disability progression in ALS baseline characteristics.
So the population enrolled in Phoenix has substantial overlap with central as was presented at ankles and July 2023. The Phoenix population is within one point of central on the baseline ALSFRSR. score and within one month a center of baseline time since symptom onset.
Phoenix has the potential to be the largest Phase three study in ALS to demonstrate a treatment benefit, which would make delivery of the first and only therapy in ALS to generate positive data in two different trials. This would be an achievement, never-before-seen analysis, and we are enthusiastic about what's important data from Phoenix. It might mean for people living with this disease.
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 reveal also known as AMX35 may have applicability across other neurodegenerative diseases are multinational Phase 3 ORION trial of AMX. 35 and progressive super nuclear policy or PSP was initiated in December 2023, and we anticipate top line results in 2025 or 2026. We shared the scientific rationale on AMX35 for the treatment of PSP. in detail, and a webinar are posted on our website in July 2023.
Turning now to our Phase 2 Healios trial of AMX35 and welcome syndrome trial is now fully enrolled, and we expect to report results in the second half of this year were from syndrome is an ultra-rare genetic disease that leads to multisystem failure resulting in blindness, deafness, diabetes, ataxia, neurodegeneration and typically death by early adulthood. There are no approved therapies for welcome syndrome.
Our R & D team has been collaborating with Washington University for many years to conduct research, which led to our decision to conduct the Phase two trial. We are looking forward to up the data from Healios would mean for people with Wilson syndrome.
Our community where the unmet need is incredibly high. We are also advancing MX are well-known for our antisense oligonucleotide candidate, targeting helping to through IND-enabling studies and expect to enter the clinic by year end campaign to as a critical factor of external degeneration, which is implicated in the pathogenesis of ALS. Again, I am very excited to be part of the analytics and look forward to meeting many of you.
I will now turn over the call to Josh.

Thanks, Camille. As we eagerly await the Phoenix top line results, we are developing 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 Libya as quickly as possible.
And to preempt a question that may be on many of your minds, there's 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 ALS is treated and to this end, we are working to accelerate the time it takes to diagnose a loss. Current data suggest that diagnosis takes about a year from symptom onset and is not a straightforward path.
Many people with ALS are misdiagnosed initially and see several specialists before they're 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 ALS early, and we intend to provide results from initial experimentation this year.
Lastly, in addition to Camille And Dan, we are thrilled that lender Arsenault recently joined us as our new Chief Human Resources Officer. She brings over 30 years of leadership and experience, including most recently from Sunovion where she was the Chief Human Resources Officer, and we are excited to welcome her to our team.
In closing, we are proud of the achievements we made in 2023 we had a strong start to our launch of Olivia and have reached many thousands of people living with ALS in 2024. We are planning for Phoenix top line data anticipated during the second quarter or before which we believe if 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 malaria and other neurodegenerative diseases.
We have a substantial opportunity ahead of us, both with our commercial launch in the U.S. and globally and with our pipeline, we are intently building a differentiated long-term franchise focused on developing and deliver 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.

Question and Answer Session

Operator

We will now begin the question and answer session. (Operator Instructions)
Geoff Meacham, Bank of America.

Great. This is Charlie on for Jeff. Thanks for taking the question. I guess for this year, you know, given this Part D redesign, can you just talk about what's the potential of our growth projection for this coming year? And secondly, can you also tell us what's the net net patient adds for the fourth quarter compared to our prior quarter? Thank you.

Yes, maybe I'll start on the Part D benefit redesign and a capacity adjustment on the new patients as well on the Part D benefit redesign kind of happens in two steps on there's 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 is no longer in effect for patients. So I think we view that generally as on as positive for patients. But of course, we already had robust programs 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. I'll pass Justin and that patients.

Yes, in our prepared remarks, you said net patients on therapy were stable to Q3, meaning it was approximately the same level as fully reported third quarter.

Operator

Neena Bitritto Garg, Deutsche Bank Canada.

Neena Bitritto Garg

Hey, guys, thanks for taking my question. I was just wondering if you could talk a little bit more about some of the gross to net dynamics that impacted the numbers specifically in the US, I need to take a gross price increase in the US.
So just wondering if that was offset and then if you could maybe explain a little bit more about the magnitude of the ex-U sales and how that compares to prior quarters and how we should think about that contribution moving forward?

Sure. Hey, thanks, Neena. And I didn't realize you were part of Deutsche Bank Canada (multiple speakers) but that's good news for us. And so yes, there really was I mean, I think the gross-to-net you see zero sorry, typical fluctuations and as I pointed out in the remarks, most of the change sort of the upper trend in Q4 really came from actually the European and Canadian revenue that we saw growing and so, you know, those have higher gross in US than they do in the United States.
And so United States, we did take 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 hasn't changed. And we think our access is actually really excellent in the US.
So related to the European and Canadian sales on, you know, we announced our early access program in France in October. There are other managed access programs across Europe, some free some paid. That's a that's a far cry from obviously a full approval and the access that we want. But of course, post the successful Phoenix as Josh mentioned, we'll be back and looking for broader global approvals as quickly as is appropriate, should the database supportive.
But, you know, we did point out that the revenue in the United States, it was quite stable. It did grow slightly quarter over quarter, but the majority of our growth came from those 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.
Als is a disease that unfortunately strikes people equally in all regions of the world. And there's a major opportunity, as I think all of you know, in rare disease 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.

Neena Bitritto Garg

And thank you so much.

You're welcome.

Operator

Corinne Jenkins, from Goldman Sachs.

Corinne Jenkins

Thanks and good morning. Maybe from 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 you're 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 hydrogen? Thank you for the question. So but we are we've been reporting on our six months it persistence rate and it's held steady and so in the US, we're still seeing approximately 60% of people still on delivery oh 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 our learnings from Canada and applying those to the U.S. And I think what we've found is that 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 comes down to education. If people know that this is they're being prescribed this treatment because it'll slow down their ALS and will keep them alive longer, then they're much more likely to stay on treatment, and they're also very practical strategies for mitigating any potential side effects.
Again, we're trying to transform ALS from a disease that has historically focused mostly on symptom management. So one that focuses or 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 practical ways that we can do that.
And so I think in terms of when that will translate to revenue, but we I think made these recognitions recently, we've 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.

Corinne Jenkins

Thank you.

Operator

Michael Cherny, Evercore ISI.

Michael Cherny

Hey, guys, thanks so much for taking my question, too for me on number one, how is the campaign of taking learnings 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 when you may lift the restriction on prescription reporting data, when might we expect that to happen?

Thank you. Yes, thank you. And I'll certainly take the first one. So I agree. So it's early, but I think the learnings from Canada, the initiatives now that we're advancing in the US, we had very positive feedbacks. I think, again, if you tried to think about what does it mean to transform a disease that historically is focused on symptom management because that's largely the tools that have been available. I think that in short is education.
So for example, I'm not saying that well, this is not a cure, but it may slow down your ALS and keep you alive longer. That's that's a big deal. And that change is how one even talks about the disease and by focusing on mitigation of any potential side effects. Again, in the past, if one has not had to it managed side effects, then those things can be uncertain.
But again, it just comes down to education. And I think that and the doctors and nurses and therapists who care for people they last. I mean, first of all, they're just amazing people. And second, I think they're very, very excited at the opportunity to see this sort of transformation.
But I think it's important that we and not just we, but all of our partners provide the 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 previous question, I think that the impact long term on revenue will we'll see continue to build throughout the year.

Yes, Mike, and maybe I'll take the question on the access to the prescription data from, I think from our read of the situation, it's not atypical to come to not have that data available in a rare disease indication. We don't have any plans to change it now. And I think 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.

Michael Cherny

Thank you.

Operator

Graig Suvannavejh, Mizuho.

Graig Suvannavejh

Thank you. Good morning, Phil. Two questions. If I could one any insights you could give into how things are progressing early in 2024, given like the changes that you're trying to make from a commercial perspective and whether there's any uptick from versus the fourth quarter trends? And then secondly, I'm curious about this novel ALS biomarker panel that you're developing. Also there. Could you provide some color on which exact what types of biomarkers might be included in that panel would be appreciated. Thanks.

Yes. Thank you, Greg. So I would say, as I was saying in the previous remarks, I think with the initiatives we've seen, I think a great reception from the community and Navy break things out as I did in the opening remarks, again into sort of two broad categories, I think versus physicians who specialize in ALS.
And I think there the educational opportunities, we've seen some people who have fundamentally changed their medical practice. 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's about half of people day less 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 of ALS is what they learned in medical school mean it might be 20, 25 years old. So they're they're very unaware of not just our treatment, but even the general advances that the field has made so there, I think awareness is really key.
And I would highlight as well that I think Phoenix has a really great opportunity in both of those segments on both to increase confidence and for the specialists in the potential benefits of delivery of patients as well as the opportunity to increase awareness for generally in the medical community.
And on the on the diagnostics side? I'll definitely pass the, Josh.

Sure. So when we thought about the ALS diagnostic, I think the first thing to note is that when you think about the path of physiology of ALS. You have the neurons that go from the bringing into the spinal cord and then a second set of neurons that go from the spinal cord to the muscle that degenerate in massive quantity over the course of just two or more years.
So it stands to reason that at least something something must be released or measured or measurable when that process occurs. So we've collaborated with several experts sooner in biomarkers and clinically in the ALS. space.
And what we can tell from from looking is there hasn't really been a serious effort thus far in the field to trying to develop a diagnostic in this space through many markers that seem to be up or down common ALS that nobody's taking those together towards the kind of serious effort towards making diagnostics. So we're quite excited about that program. I think there's there's a lot of low hanging fruit there, but we'll be reporting data on that later this year.

Graig Suvannavejh

Thank you.

Operator

Marc Goodman, Leerink Partners. Please ask your question.

Hi. This is my view on the line for Mark are patients who are starting unreal, if you will, of your home delivery 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 as engagement with the general neurologists there and patients? And then high? Or just curious on your experience there so far.

Great. Great question. So I think over time, naturally, I think we'll get people who were closer to diagnosis. I think at launch, 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 changes how you think about your education and persistence efforts as well. And then on the on the second question is Spirit on the experience side of the centers. So I think our prescribing remain fairly concentrated sales. So roughly 80 and 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 on some of our initiatives that we've just started kicking off and we're very excited to have Dan. It lead as well, are 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 broaden and throughout the year. But I think it's a really nice place to start from where we have people who have fundamentally changed their medical practice. These are the ALS specialists who see and most people with ALS. So I think it's a really nice foundation to have and then we can broaden out from there.

Thank you.

Operator

Joel Beatty, Baird.

Hi, this is Dan 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 Tu-Ka ALS trial results are announced?

Yes. So I think first, it's really important to note that these are different products and different trials. And so our drug delivery is a combination of sodium phenylbutyrate and versatile and in proprietary co-formulation and so it is not the same product as Dan Tucker.
And additionally, these are very different trials on Phoenix and center. We're trials designed with all the experts in the field on using some of the most state of the art ways of running these trials in a controlled and rigorous way. And I'd say thus far on the Type two trial that's been run is quite different.
And design is a smaller smaller trial on primarily with kind of an academic on design tilt that was also run during the peak of COVID upside. I think probably the broad strokes is we don't really think they're comparable and that there will be a read-through between the two arms.
So we wouldn't draw those on comparisons on as that data comes.

And I would just reiterate to Delivery Hero is the only FDA approved medication that it was shown to slow ALS progression on the ALS functional 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 among the ALS specialists, we've seen such excitement. That's why FDA took the medication on to.

Operator

Andrew Fein, H.C. Wainwright.

Andrew Fein

Yes, good morning. A footfall, Euro congratulates. Congratulations on reaching the milestone of completing the 1st year of the loans. It has increased. You're not seeing the story evolve from the infection and being the last maybe I have two questions.
The first one is related to Phoenix, the Phoenix and the inclusion criteria mentioned definite and clinically provable ALS patients. So my my question is, how do one defined clinically probable ALS patient? What was the rational behind including that, that group of patients? And how does it change the way the data is analyzed?

Yes. So maybe first on probably I'm trying not to get too in the weeds. But first on in terms of the definitions of the LS. score, our criteria of ALS defines many different potential groups, including suspected possible possible or probable ad-supported, probable and definite on and did the difference between those is essentially how many regions of the body are convincingly affected by ALS with possible being one probable lab support to being one plus different lab tests, probable clinically probable being two and definitely being three or four.
The body is divided into four regions on the scale of what's been shown over time is that even people with possible LS. on this criteria, how they last about 98% of them have ALS. And so it's not like this that it's not actually a scale of certainty. It's much more a scale of on a diffusion basically how how far across the body on ALS has progressed when we did statistical modeling on going for the probable and definite on patients does give you a more consistent and fast progression group, which 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 there within a point on the baseline ALSFRS-R and within a month on the baseline time since symptom onset, which are two of the most important measures when we're thinking about our cohort, it's likely to progress and end to perform it.

Andrew Fein

Thank you. And the second question is related to the rare disease. Yesterday, there was this public discussion on the biomarkers in the rare disease space, where Peter Marks, I think was very positive about using biomarkers for accelerated approval pathway now being in the company where you are looking at rare diseases like ALS, Wolfram syndrome.
And you were there was discussions on developing a biomarker panels for ALS. What is is there a strategy to kind of develop a very definitive biomarkers like surrogate biomarkers that that might help you get to that accelerated approval pathway for other rare diseases in the pipeline such as Wilson. Thank you.

This is Camille. I appreciate the question. We actually are very heartened by Dr. Mark's comments around the possibility of using biomarkers for accelerated approval and rare disease. It will make a tremendous impact and giving the opportunity to treat individuals sooner study, the disease process and come and impact these and particularly neurodegenerative diseases where there is often a small window of opportunity.
And therefore, we 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 from syndrome in similar ways as we consider with our initial trial ways of learning about the clinical biology as well as potential biomarkers there. So stay tuned.

Operator

Thank you. There are no further questions at this time. I will now turn the call back to Mr. Klee for the closing remarks.

Thank you, operator, and thank you all for joining us on our call today and for your support We hope you have a great day.

Operator

Thank you. Ladies and gentlemen, the conference has now ended. Thank you all for joining. You may all disconnect.