Amylyx Pharmaceuticals Q2 2023 Earnings Call Transcript

Quartr
Provided by Quartr
August 10, 2023

There are 12 speakers on the call.

Operator

Afternoon. My name is Tom, and I will be your conference operator today. At this time, I would like to welcome everyone to the Amelix Pharmaceuticals Second Quarter 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

Please be advised that this call is being recorded at the company's request. I would now like to turn the conference over to Lindsey Allen, Head of Investor Relations and Communications. Please go ahead.

Speaker 1

Good afternoon and thank you for joining us today to discuss our Q2 2023 earnings. With me on the call are Josh Cohen and Justin Klee, our Co CEOs Marker Ohlinger, our Chief Commercial Officer and Jim Frates, our Chief Financial 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 Litigation Reform Act of 1995. These statements include, but are not limited to, Our expectations with respect to RELIBRIO and ALBRIOZA statements regarding our clinical trials and regulatory developments and Expected timing thereof, our business strategy and outlook 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.

Speaker 1

You are cautioned not to place any undue reliance on these forward looking statements and Amelix disclaims any obligation to update such statements unless required by law. Now, I will turn the call over to Justin.

Speaker 2

Thank you, Lindsay, and good afternoon. In the Q2, we made significant progress in bringing Relivrio and Albriosa to people with ALS in the U. S. And Canada respectively and advancing our mission of one day ending the suffering caused by ALS and other neurodegenerative diseases. We are incredibly proud of our entire team who has continued to deliver on the goals that we outlined following the full FDA approval of Relivrio last September.

Speaker 2

These goals included raising awareness and educating people living with ALS and physicians about Relivrio, Working with insurers to provide access to our approved treatment, helping people who have been prescribed ILIBRIO through our Amelix Care Team patient support program and deepening our understanding of the ALS market. Let me walk you through our progress. Our commercial organization is off to a strong start And raising awareness about Relivrio as evidenced by the strong and steady demand we saw in the second quarter. As of June 30, 2023, there were roughly 3,800 people on Relivrio in the U. S, up from roughly 3,000 people on Relivrio as of March 31, 2023 and just over 1300 at the end of 2022.

Speaker 2

As for an insurance coverage, adoption has been rapid and a vast majority of policies have been broad and supportive. Insurers covering nearly all people living with ALS have published formal policies and are covering Relivrio. And people living with ALS that have been prescribed Relivrio are now able to start therapy more quickly. The average time between a Description being written and Relivrio being shipped was about 25 days in the second quarter, down from around 30 days in the Q1 of this year and a little over 45 days when we shared this metric on

Speaker 3

our Q4 earnings call.

Speaker 2

Turning to Canada, We reached our 1 year anniversary of the Health Canada approval with conditions in June. As we reflect on our progress, We are pleased with what we have achieved on behalf of the Canadian ALS community. In the Q4 of last year, we announced that all major private insurers in Canada And we are pleased to share today that by the end of August, we expect Albriosa coverage to be in place for the vast majority of publicly insured lives in Canada. The early success of our commercial launches has enabled To both invest in bringing Relivrio to more people living with ALS globally and advance our pipeline opportunities that support our mission. We continue to expect a final opinion on our MAA from CHMP in the fall and prepare to execute a successful launch in the EU if approved.

Speaker 2

We are excited to initiate our new Phase 3 ORION clinical trial of AMX35 in progressive supranuclear palsy later this year. And of course, we continue to work diligently to complete the Pfenex trial with top line results anticipated in mid-twenty 24. In summary, we are proud of our progress so far in 2023 and excited about what we can achieve as an organization. We have a clear mission and we are executing on our goals. I will now turn the call over to Margaret to provide an update on our commercial performance.

Speaker 4

Thank you, Justin. In the Q2, we continued to make significant progress on our 3 key priorities. The first is our effort to drive awareness and educate about the benefits of Relivrio among people living with ALS and clinicians. This includes educating that Relibrio is the 1st and only approved drug for adults with ALS to demonstrate a Statistically significant benefit and function in a clinical trial as well as a survival benefit and a longer term post hoc analysis. As a reminder, clinical trial data published in the New England Journal of Medicine demonstrated that after 24 weeks, Participants treated with RELIBRIO scored on average 2.32 points higher on the ALS FRS scale than the placebo group.

Speaker 4

Even a one point change in the ALS FRS score can indicate a significant difference in a person's ability to function independently with activities of daily living, including eating, bathing, dressing or walking. Participants treated with RELYRIO at the start of the clinical trial, which means that they both started RELYRIO 6 months earlier And we're on it for longer than participants starting on placebo, so a greater survival benefit. During the Q2, interest in and demand for Relivio continued to build at a steady pace from both those that are newly diagnosed and people who have been living with ALS for years. As a result of our educational efforts, as of June 30, There were roughly 3,800 people on RELIBU in the United States. We are very pleased that so many people have gained access to this important new treatment so quickly.

Speaker 4

Now let me run through a few key metrics that demonstrate our progress and growth opportunities ahead of us. Prescribing remains fairly concentrated with just over 80 prescribers, mostly at major ALS centers, representing approximately half of all Relivio prescriptions at the end of the quarter. We are encouraged by the level of interest among this group and believe that we have an opportunity for growth as we bring our message to more prescribers and deepen our relationships within these key ALS centers. By the end of the second quarter, approximately 75% of the top 500 U. S.

Speaker 4

Prescribers and nearly all of the key ALS centers have prescribed RELIBRIO to at least one person since launch. We are focused on driving awareness and education and on deepening our penetration within the top prescribers and key ALS centers. In addition, we have a large untapped opportunity for growth outside of Group of top prescribers as we expand our outreach and educational efforts more broadly. Our second priority is engaging with payers to work to help ensure That every eligible person who could benefit from Relivio treatment has access as quickly and efficiently as possible. Consistent with the targets we previously outlined, by the end of the second quarter, our estimates suggest that U.

Speaker 4

S. Insurers Representing nearly all ALS covered lives had published formal coverage policies. The vast majority of these insurers provide broad access to Relivrio. This is a significant accomplishment just 3 quarters into our commercial launch. Our third priority is ensuring eligible people living with ALS have positive interactions through their treatment journey with Relivrio and ALS clinics have positive interactions with Amelix.

Speaker 4

This includes facilitating an organized clear Just to get people who have been prescribed Relivio access to therapy as quickly as possible and optimizing people's experience obtaining Relivio as best we can. Our team has done a tremendous job of facilitating the process, increasing the speed between the prescription being written and Relivrio being shipped. In the Q2, on average, this timeline was shortened to about 25 days, aided by the increase in insurance coverage. Our team continues to work expeditiously to get people living with ALS who have been prescribed Relivrio on therapy as quickly as possible. Turning to our launch in Canada.

Speaker 4

Interest in Albriosa remains high. We have made significant progress on our public insurance coverage efforts. We are thrilled that 5 Canadian provinces Ontario, Quebec, British Columbia, New Brunswick and most recently Alberta announced public reimbursement of Albriosa. By the end of August, we expect albriosa coverage to be in place for the vast majority of publicly insured lives in Canada. Our goal is to ultimately change the way people living with ALS are treated.

Speaker 4

And we believe RELIVIO is becoming a foundational therapy for ALS. As we move into the Q3, our team remains focused on driving awareness and education about Relivrio among people living with ALS and clinicians. I will now turn the call over to Jim to discuss our financial results for the Q2.

Speaker 3

Thanks, Margaret. We're encouraged by the strong interest and demand we continue to see from the ALS community in the 2nd quarter. From a financial point of view, our business remains strong. Net product revenues were $98,200,000 for the quarter compared to net product revenue of $71,400,000 for the Q1 of 2023, with the vast majority of that revenue coming from the United States. Gross to net adjustments were approximately 10% in the quarter, which is a little lower than that what we would normally anticipate due to the favorable true up of reserve estimates in the Q2 based on our actual experience in the past 9 months.

Speaker 3

Going forward, we anticipate gross to net discounts will be in the 12% to 15% range. Inventory levels At the quarter end, we're as expected with approximately 2 weeks of inventory in the channel at specialty pharmacies, similar to what we've seen in previous quarters. Cost of sales were $5,600,000 for the quarter, roughly 6% of net product revenues, and our expectation is that COGS will remain in the range of 6 to 10% of sales going forward. Note that we fully expensed all of our remaining royalty obligations this quarter and will not be incurring any additional royalty expenses related to sales in ALS going forward. For modeling purposes, Keep in mind that roughly 10% of the people on Relivio are receiving it for free through either our interim access program or patient assistance program.

Speaker 3

Research and development expenses were $29,000,000 for the quarter. You should expect R and D expenses to be in the $30,000,000 to $40,000,000 range per quarter for the remainder of the year. As we outlined in our Q1 call, our expectation is that R and Selling, general and administrative expenses or SG and A were $43,400,000 for the quarter, in line with the $45,000,000 per quarter run rate that we mentioned on our Q1 call. We continue to expect SG and A expenses in this range for the remainder of the year. These results led to an excellent bottom line.

Speaker 3

We generated $22,100,000 in net income, representing our Q2 in a row of profitability. Finally, we ended the quarter with cash and short term investments of $357,300,000 and 0 debt. We're pleased with our strong financial results and with the disciplined execution throughout the organization. From a capital perspective, we have the resources we need to execute on our mission. We're well positioned to grow our top line, invest in our pipeline to provide more much needed treatments for neurodegenerative diseases and deliver on our bottom line.

Speaker 3

I'll now turn the call over to Josh to discuss our R and D programs.

Speaker 5

Thanks, Jen. We have demonstrated the benefit of AMX35 in ALS and believe it could help in other neurodegenerative diseases. When we originally developed AMYX-thirty five, our goal was to target endoplasmic reticulum stress or ER stress and mitochondrial dysfunction, 2 connected central pathways that lead to neurodegeneration. As we announced on our last call, we intend to initiate a global pivotal Phase 3 study in PSP later this year called ORION. ORION is a well powered study that will enroll approximately 600 participants and it was designed and planned in collaboration with key global academic leaders, people living with PSP and advocacy groups.

Speaker 5

ESP is a rare but recognizable progressive and fatal neurodegenerative disease with clear and well known clinical hallmarks that include progressive disability with respect to eye movement, walking and balance, speech and swallowing and cognitive function. There are currently no disease modifying treatments for PSP and we are hopeful that AMX35 might be able to help. PSP is characterized as a tauopathy with genetic and pathological findings supporting a primary role for tau in this disease. Given AMX-thirty five's proposed mechanism of action that targets pathways upstream of tau aggregation and the Phase 2 placebo controlled PEGASIS clinical trial data, which demonstrated that AMX-thirty five Significantly lowered both phospho tau-one hundred and eighty one and total tau in the cerebral spinal fluid of people with Alzheimer's disease, We are excited to pursue this indication in PSP. We recently hosted a webcast with Professor Doctor.

Speaker 5

Gunther Hoglinger, a leading expert in PSP and the primary investigator for our Phase 3 ORION trial. As part of the webcast, We shared insights into the scientific rationale for studying AMX-thirty five in PSP and an overview of the Phase 3 trial design. Additionally, Doctor. Hoglinger provided his perspectives on the PSP treatment landscape, the role of tau in this disease and the potential to use AMYX-thirty 5 in people living with PSP should it be approved. The replay is available in the Events section of our IR website and we encourage you to listen to it if you didn't have a chance to join us for the live event.

Speaker 5

In addition to this exciting study in PSP, We are also pursuing a program in another rare disease, Wolfram syndrome, called HELIOS. Wolfram syndrome is a rare disease that leads to multisystem failure resulting in blindness, deafness, diabetes, ataxia, neurodegeneration and often death in early adulthood. Our R and D team conducted roughly 4 years of in vitro and in vivo Studies of AMX35 in Wolfram syndrome together with a leading researcher, Doctor. Fumihiko Jurano at Washington University. These studies had promising results, some of which were published in the Journal of Clinical Investigation Insight.

Speaker 5

Several papers characterize the disease as a prototypical disease of ER stress. And as we have discussed in the past, We believe this study, which is currently enrolling participants, will provide key data to guide future studies, and we expect top line results next year. We also continue to broaden our ALS and neurodegenerative disease pipeline. We believe that in order to ultimately find a cure for ALS, it's going to take a combination approach, targeting multiple cellular pathways implicated in disease pathogenesis. We continue to progress AMX-one hundred and fourteen, our antisense oligonucleotide targeting CALPANE-two through IND enabling studies.

Speaker 5

We have presented data on this candidate at the NEAL's, MDA and TIDES conferences. We're excited to present more data on AMYX-one hundred and fourteen and other advancements in our pipeline in the future. In closing, we are proud of our team's progress and the work that we are doing on behalf of the ALS community. We are in a very strong financial position, which allows us to continue to support our launches in ALS and invest in our pipeline to find new treatment options for people living with ALS and other relentlessly progressive neurodegenerative diseases. Now we'd be happy to take your questions.

Speaker 5

Operator, please open the call up to Q and

Operator

We will now begin the question and answer session. We ask that you please limit yourself to 1 question and one follow-up. And if you have additional questions, you may rejoin the question And the first question comes from Corrine Jenkins with Goldman Sachs. Please go ahead.

Speaker 6

Good afternoon, everyone. Maybe just first, what are you seeing with respect to compliance and discontinuation rates? And I'll just go ahead with my follow-up here. Are you seeing any emerging trends with respect to the primary reasons for discontinuation? It would be helpful what are you can share there?

Speaker 4

Thanks very much for the question. Maybe just to start as a reminder, we report on net patients on therapy. So this is inclusive of any discontinuations. We are really pleased with our ability to serve the roughly 3,800 net patients on Relivrio at the end of Q2. I would say it's really too early to see any long term trends at this point in our launch.

Speaker 4

But maybe a reference point, in the Centaur trial, which again as a reminder was a 6 month trial, Approximately 70% of participants remained on drug and we're tracking close to that in terms of the commercial setting. But I would say this is clearly an area where we're going to continue to keep a very close eye on as we expect the patient mix to shift over time.

Speaker 6

And maybe I'll just add on the compliance reasons. Yes.

Speaker 5

Yes, sure. On the compliance side, on the reasons. So on the compliance side, we've seen most rare disease drugs you'll find in the 75% to 80% range in terms of drug compliance. We're in that range as well. And in terms of reasons for discontinuation, they're varied.

Speaker 5

People with ALS are going through Many different things. Of course, one of the more common ones certainly is death and disease progression, Which is expected in ALS as well.

Speaker 6

Thanks.

Operator

The next question comes from Geoff Meacham with Bank of Please go

Speaker 2

ahead. Hey guys, thanks for

Speaker 7

the question. Congrats on the quarter. I had a couple. The first is The EU, the CHMP process, I wanted to ask kind of how you guys view that process now And maybe just help us with kind of how you view the next steps here. I wasn't sure for reexamination What the protocol or the success rate could be there?

Speaker 7

And the second question is just related to capital deployment. I know you guys have done some deals, Some BD, but I want to ask maybe how that's evolved over time as you look to be sustainably profitable? Thank you very much.

Speaker 5

Sure. So on the EU and CHMP, so as we've shared previously, the EU adopted initially a negative opinion. We strongly disagree with that opinion. And I think the basis for that is we ran a randomized placebo controlled study Met our pre specified primary outcome showing a slowing in the rate of progression on the ALS after SAAR. We've also observed a difference on overall survival.

Speaker 5

And that data of course led to our approval by full U. S. FDA approval and our approval of conditions in Health Canada. So we believe we have a data package that should, in our view, support approval. But of course, ultimately, this is up to CHMP.

Speaker 5

So So we submitted for reexamination, that's roughly a 4 month process under which 2 new repertoires are assigned and review the application. We submitted that shortly after. We got the negative opinion. So you can expect that near the end of this year, We'll hear back regarding that opinion. In terms of capital deployment and potential BD, I'll talk to I'll pass to Jim.

Speaker 3

Yes. Thanks, Jeff. And clearly, the profitability this early on in our launch is very gratifying. I mean, I think that ultimately This is a direct result of the need in this patient in this area and the vast the direct of options that people living with ALS have had So far, so we're very proud to be filling some of that need. I think the other thing I would say too is we're really focused on what we have on our plate now, Right, with obviously still in the middle of the launch in the United States and Canada, working through what's going to happen in Europe and potentially hopefully looking forward to a launch next year.

Speaker 3

We've started off and are interacting with the Japanese and other places around the world too to see where we can continue to take this drug. With our PSP program and our Wolfram's program ongoing, first that we're doing, I'd say the watchword around here right now is focused. Now longer term, I think we have the opportunity with our launch and with the business that we're in to potentially do some business development over the longer term, but I think that hopefully we'll be able to grow the top line, grow our bottom line and then also invest robust pipeline, but we have a pretty high standard here for new programs. And as I said, I think right now the watchword is on focus.

Speaker 2

Thanks guys.

Speaker 3

You bet.

Operator

And the next question comes from Michael DiFiori with Evercore. Please go ahead.

Speaker 8

Hi, guys. Congrats on the quarter and thanks so much for taking my question. 2 for me. Now with the passage of more time, do you have any better sense of the size of the patient bolus at this point. And my follow-up is this, I want to push a little bit on the discontinuation rates.

Speaker 8

Among the early patients to have received commercial drug in 4Q of last year, presumably some of these patients Would have been on drug for at least 6 months now. Would you be able to provide any color as to what percent of them are still on therapy at this point? Again, just among the patients who started in 4Q.

Speaker 4

Yes. Maybe I'll just start with your question regarding the bolus. We continue to be pleased that the interest in and demand for Alivrio continues to be at a very strong pace. And I think Importantly includes a mix of both newly diagnosed patients and people who have been diagnosed and living with ALS for many years. Again, at the end of Q2, we had roughly 3,800 net patients on therapy, up from roughly 3,000 Patients in Q1 and just over 1300 in Q4.

Speaker 4

So we really believe that at this point in time, Relivrio is really starting to become a foundational therapy and ALS and meeting a really high unmet need for this patient community, which is obviously our mission and what we've been focused on for some time. As far as the growth opportunities, which is equally important to us, we see several different opportunities ahead of us. First, the prescribing remains fairly concentrated with the 80 prescribers, mostly at the major ALS centers where our focus was at the beginning of launch, representing about half of all Relivio prescriptions this quarter. We're also encouraged that the level of interest among this group and believe that we have a large As we bring our messaging to more prescribers and deepen our relationships within those key centers. And I think importantly, we're seeing those prescribers be much more prolific in their prescribing, which I think is an important part.

Speaker 4

And second, We have a really large untapped opportunity for growth outside of this group. As I mentioned, we were heavily focused on the key ALS centers at launch. We're continuing to expand our outreach and educational efforts more broadly because we believe it's critically important that everybody is aware that WILIMRIL is the 1st and only product to have both function and survival demonstrated in a clinical trial and we believe we can change the paradigm for treatment moving forward. And maybe just to answer your second question on discontinuations, again, we're only going to be reporting on net patient numbers per quarter. But indeed, I think it's important to reflect that the first cohort of patients who started on therapy at launch, Many of those who have been really fairly progressed early on.

Speaker 4

So I think we're going to see the dynamic of the patients change over time. So it's a little too early to really give any trends there.

Speaker 5

Yes. And one thing I'll remind that I think Margaret shared earlier as well. We in the center study, approximately 70% of people completed that study on study drug. What we're seeing in the real world is not so different from that. But again,

Operator

The next question comes from Mark Goodman with Leerink Partners. Please go ahead.

Speaker 9

Yes. Hi. First question is, are you willing to make any comment About what kind of trends you're seeing you saw in July? And then secondly, can you just confirm, you mentioned 10% 10 of the patients are getting free drug. Just to be clear, that 10% is in the numbers that you provide, right, in the 3,800 patients that you were talking about, and it's all included in your gross to net calculation and everything.

Speaker 9

I just want to be clear on that. Thanks.

Speaker 3

Yes. Hey, Mark. It's Jim. Yes, the 10% of the patients that are receiving free drug is included. So those 10% are included in the net patients.

Speaker 3

Those are all the patients on therapy at the end of June. And yes, free drug would be included in gross to nets. Excuse me, free drug is actually in COGS, right. You've given the drug and you're not getting any sales for it. So the free drug goes in COGS.

Speaker 3

Any patient assistance that we pay in terms of co pay assistance or things like that, that's what goes in the gross to nets, just to be clear on that. And then finally in terms of July, I think we'll comment on the July trends when we report our quarterly results for Q3. But I think our business is with now 3 quarters under our belt, we're all starting to get a chance to see what our business is like moving forward, But we won't be giving specifics on July at this stage.

Speaker 5

Okay. Thanks. You bet.

Operator

The next question comes from Greg Suvannave with Mizuho. Please go ahead.

Speaker 10

Okay. Thanks so much. Congrats on the quarter. Just two questions. One, maybe another way to ask a question that people seem to be trying to get at.

Speaker 10

Do you have any color on or can you provide any color on kind of new prescription trends versus refill Trends, any kind of metrics you can provide there and how that's evolved? And then my follow-up is I'm curious about the expansion efforts to go beyond the ALS centers and if you could perhaps put into context The portion of ALS prescribers, that you're targeting or that you hope to get that are outside of the ALS center setting. Thanks.

Speaker 2

Yes. So maybe I'll start And then have Margaret join in too. So again, we're 3 quarters into launch. We have roughly 3,008 100 net people on treatment as of the end of Q2, which we're very pleased about. I mean, that's 3,800 people at ALS were helping, but that means that there's many, many more people that we'd like to help as well.

Speaker 2

But I think we all here are constantly reminded of the mission at hand. And I think the ALS market in many ways is unique and it's because it's a large rare disease, there's a huge unmet medical need, and historically there have been a few treatment options. And so I think the way that we've thought about our business, as Margaret was sharing, is to focus on the ALS specialists and then continuing to look to broaden out. And so I think as we look at our prescription numbers, where our people have focused is where we're seeing the prescriptions as well. And then Margaret, I'll invite you to share any more details on that.

Speaker 4

Yes. So as we've indicated, They heavily focused that launch, which I think was the right strategic decision to focus on the key ALS centers where the majority of ALS patients are actually treated. However, there's also a number of ALS patients that are treated outside of those ALS centers for multiple reasons, either They can't transport, they can't get there at a reasonable time, and they typically need to go there every quarter to see the multidisciplinary care. There are a number of general community neurologists that are equally important to be educated and that's where we're expanding our focus and we are continuing to Increase our penetration and reach out to those, what we call our TRATRB targets. And we're just going to continue to work on that expansion moving forward because it's really important for us that every physician who treats an ALS patient is educated about The significant Relivio benefits that we can bring to be able to serve this patient community optimally.

Operator

And the next question comes from Amanda Gosh with H. C. Wainwright. Please go ahead.

Speaker 11

Hi, guys. Thanks for giving me time. Also congrats on the quarter. So I have two questions on the ALS program and one for the The first question is basically some of the question which keeps coming from the industrial community. And that would be, if Pfenex is a required confirmatory study for accelerated approval of Relivrio.

Speaker 11

And what if it fails, Then can FDA initiate a process to pull it off from the market? So any clarification on that would be great. And then I have a follow-up question on the ALS program and

Speaker 2

Sure. And thank you for the question, Anda. And, yes, I mean, first, There is a full FDA approval. There is no condition of the PHOENIX study. In Canada, it's an NOCC, which means a notice in compliance with conditions.

Speaker 2

And again, our expectation in Europe, If there's an approval of that, it would also be a conditional marketing authorization and the condition there would be PHOENIX. So we continue to run the PHOENIX study. We'll have those results mid next year. That's a very highly powered study And we and I think the ALS community are looking forward to those results. But from an FDA perspective, it's a full FDA approval.

Speaker 11

Great. Thank you. The next question is, the last quarter, you had remarked that the patient had might It showed signs of stability. So is it still is that statement still remains valid As we think ahead in terms of the growth with the Veliprio.

Speaker 2

Well, so I I think the question you're bringing up on patient responses is a critical one in neurodegenerative disease and It's a new area for all of us and I think it's really, really exciting. But I'd say, it's still too early To really tell, now in the Centaur study, we certainly we saw some people who seem to progress very little and others who progress faster, but whether that was due to their specifics, Whether it's genetics or environmental or whether that was just due to their course of disease, those are the questions that we're still asking and we haven't been able to ask because we haven't had effective treatments. But I'd say at this time it's just too early to know, but certainly Those sorts of real world evidence type approaches is something that we're very, very excited to do more of, because I think you can start to ask Some really, really critical questions when you have an effective treatment.

Speaker 11

Got it. Thanks. And my last question is on PSP. Based on some natural history data, so we kind of saw that the PSP study in general has These sales, at least in the historical PSP trials. So what has been your thought on it and how have you kind of incorporated

Speaker 5

On a recent call that we did that's published on our website, a much more in-depth view of PSP and our upcoming clinical trial. So in our PSP trial, we are designing the study based on the PSP RS, the PSP rating scale. This is a scale that tracks primarily, I would say primarily kind of motor and functional rather than cognitive outcomes of PSP. The PSP rating scale studied in several past trials. What's been found quite consistently There's an approximate 10 point progression over approximately a year, with a pretty small LFR and a pretty tight kind of variance when you're in these studies.

Speaker 5

Dropout is not in our view and has not been in previous studies beyond what you might have in other neurodegenerative diseases. And again, as has been shown from the previous studies, This is a space actually where the power is quite good compared to most neurodegenerative diseases. So I'd say overall and again, I encourage people to listen to the webinar where we go into this in a lot more detail. But I'd say overall, we feel very strongly that we have a great trial design, we have a great scientific rationale

Operator

This will conclude our question and answer session. I'll turn the conference back over to Justin Klee for any closing remarks.

Speaker 2

Thank you, operator, and thank you all for joining us on the call today and for your support. So we hope you all have a good evening. Thanks for joining us.

Operator

The conference is now concluded. Thank you for attending

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Key Takeaways

  • In Q2, Amelix achieved 3,800 net patients on Relivrio in the U.S., up from 3,000 in Q1 and 1,300 at end-2022, with insurers covering nearly all ALS lives and average prescription-to-shipment time reduced to 25 days.
  • The Canadian launch of Albriosa marked its one-year Health Canada approval anniversary, with all major private insurers on board and expected public reimbursement in most provinces by end of August.
  • Key pipeline milestones include a CHMP reexamination of the EU MAA in the fall, initiation of the global Phase 3 ORION trial of AMX35 in progressive supranuclear palsy later this year, and top-line results for the PHOENIX ALS trial anticipated by mid-2024.
  • Financially, Q2 net product revenues rose to $98.2 million versus $71.4 million in Q1, driving a second straight quarter of $22.1 million net income, while ending the period with $357.3 million in cash and no debt.
  • Amelix continues to expand awareness beyond major ALS centers and deepen payer engagements, aiming to broaden Relivrio prescribing and establish it as a foundational therapy for ALS.
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Earnings Conference Call
Amylyx Pharmaceuticals Q2 2023
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