Amylyx Pharmaceuticals Inc (AMLX) 2022 Q3 法說會逐字稿

Good afternoon. My name is Chuck, and I will be your conference operator for today. At this time, I would like to welcome everyone to the Amylyx Pharmaceuticals Third Quarter 2022 Earnings Conference Call.

(Operator Instructions)

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

Good afternoon, and thank you for joining us today to discuss our third quarter 2022 earnings. With me on the call are Joshua Cohen and Justin Klee, our co-CEOs; Margaret Olinger, 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 based on our current beliefs plans and expectations and are made pertinent to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995.

Examples of forward-looking statements made during this call include our expectations for the commercialization of and the timing of receipt of revenue from RELYVRIO and ALBRIOZA. Statements regarding regulatory developments, the anticipated timing of coverage and policy decisions by insurance plans related to RELYVRIO and whether those decisions will be favorable.

Expectations regarding access to and coverage our expectations with respect to recruitment and completion of our ongoing PHOENIX trial and the anticipated ramp in our SG&A expenses. Actual events and results differ materially from those expressed or implied by any forward-looking statement 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 Amylyx disclaims any obligation to update such statements. Now I will turn the call over to Justin.

Thank you, Lindsey, and thank you, everyone, for joining the call. Our mission is to end this suffering caused by neurodegenerative diseases. And while much work remains, the past few months have marked incredible progress, including the FDA approval and commercial launch of RELYVRIO for adults living with ALS in the United States.

This marks our second commercial launch following the approval of ALBRIOZA with conditions in Canada. We know the ALS community is counting on us, and we are working every day to help ensure that every eligible person who can benefit from RELYVRIO and ALBRIOZA can gain access as quickly and efficiently as possible. Given we are only a few weeks into the launch in the U.S., it is too early to discuss specific expectations, but we are encouraged by the initial engagement with both physicians and with people living with ALS.

Margaret will share some further insights shortly. Outside of North America, we are actively pursuing regulatory approval of AMX0035 for the treatment of ALS in the European Union. Our marketing authorization application remains under review by the Committee for Medicinal Products for Human Use, or CHMP, of the EMA.

We are now in possession of the (inaudible) day 150 joint assessment report, which is provided to applicants as a reference as the CHMP collects comments to responses made to the Day 120 list of questions. While we have resolved several major objections from the Day 120 list of questions, some still remain, and we will work to continue to try to address them as part of the MAA review process. The next step in the process is the Day 180 list of outstanding issues which we will respond to accordingly. The CHMP also intends to hold a scientific advisory group meeting as part of the regulatory review process. The EMA process is still on track, and we expect the decision in the first half of next year. With that, I'll turn the call over to Margaret to share some insights into the early days of our launch in the United States and to provide an update on our Canadian launch.

Great. Thanks, Justin. In the days and weeks following the FDA's approval of RELYVRIO on September 29, we immediately started receiving enrollments and prescriptions through the Amylyx care team and have heard very positive feedback from physicians in the ALS community about the level of support we are providing.

Importantly, on October 24, the first shipment of RELYVRIO from one of our specialty pharmacies was sent to a person living with ALS earlier than we had originally anticipated. In regard to interest in RELYVRIO, we are seeing a solid initial bolus, including an encouraging number of product enrollment forms and prescriptions coming into the Amylyx care team.

This initial excitement has also been widespread across the country and not limited to one geography or a group of physicians. The field teams have engaged with clinicians throughout the country and the feedback from those serving the community has been positive.

We are initially focused on the leading 186 ALS centers, and the teams are already actively engaged with all of these centers since approval. We are also dedicating time to smaller local centers and neurology practices to help ensure that every person living with ALS has access to and information on RELYVRIO.

While we are pleased to see this level of initial excitement, and have both the people and the product needed to fulfill it. We cannot yet comment on how quickly this demand will translate into revenue. Given it can take 30 to 45 days for a person with ALS to get on treatment to the payer's medical exception process, which is standard for any new launch, especially in orphan diseases.

As the result, we continue to expect the gradual commercial ramp-up as outlined on our approval call in September. As it relates to working with insurers, our conversations have been productive. We had robust discussions with the majority of major payers and pharmacy benefit managers.

And in general, they have been supportive of RELYVRIO's clinical profile and have understood the urgency to provide access to people living with ALS. We are working hard to educate and encourage the broadest coverage possible which we believe is appropriate given the data supporting the RELYVRIO and the label. Although it is early days, we are receiving generally good feedback at this point. we anticipate payers will begin to publish policy decisions in the first half of next year.

Now turning to our launch in Canada. The interest in ALBRIOZA are across the ALS community continues to be wide and national. The number of prescribers continues to build month-over-month with interest spread across the country. As it relates to access, as announced on our U.S. approval call in September.

We are in negotiations with the 4 largest private Canadian insurers, which represent approximately 80% of the private insurance population. To date, we have already closed one negotiation and expect the others to follow suit.

On the public side, we are currently in negotiations with the provinces and territories about the scope and breadth of coverage they will provide. Our team knows that people living with ALS and their families have no time to wait, and we continue to be committed to reaching our goal of broad and equitable reimbursement criteria.

We continue to strongly believe that RELYVRIO and ALBRIOZA have the potential to make a significant impact on the treatment of ALS. Our commercial and medical affairs teams are in place to make this important treatment as accessible as (inaudible).

Each quarter, you will hear more from us as we progress through the launch and more experience and insights or gain. To close, I want to add that at every step, our focus is on providing access and support to every appropriate person living with ALS who can benefit from our product, and we will continue to partner closely with the ALS community. With that, I'll turn the call over to Jim to review our financials for the quarter.

Thanks, Margaret. I'll now summarize our results from the third quarter, where we were particularly focused on launching in Canada, the reconvened Advisory Committee, and our September 29 PDUFA date. For the quarter ended September 30, 2022, net product revenues were $0.3 million and related to the sales of ALBRIOZA in Canada.

Although we've seen strong interest in Canada thus far, we do not expect it to translate to meaningful revenue in the near term, given that the reimbursement process takes 6 to 12 months, and there are only a small number of people covered by private insurers at this time.

Cost of sales were $0.2 million and represented the costs associated with our revenue in Canada this quarter. This is elevated as a percentage of revenue since the majority of people who received ALBRIOZA received it for free, and a dynamic that will continue into the fourth quarter.

Research and development expenses were $24.9 million for the quarter compared to $12. 9 million for the same period in 2021. The increase was mainly driven by higher product manufacturing and development expenses to support the global Phase III PHOENIX trial and launch preparation activities for commercialization.

It was also driven by additional personnel-related expenses due to new headcount to support research and development efforts. Selling and general and administrative expenses, or SG&A, were $29.9 million for the quarter compared to $10. 4 million for the same period in 2021.

The increase was primarily due to personnel-related costs, which included hiring additional headcount to support commercialization preparation initiatives and other expenses for launch readiness activities and operations. We expect some growth in SG&A expense going forward as we incur additional sales and marketing expenses as part of our U. S. launch.

Net loss for the quarter was $53.9 million compared to a net loss of $23.1 million for the same period in 2021. As for cash, we're in a strong position. Cash and short-term investments were $162.6 million at September 30 compared to $206.7 million at June 30, 2022.

The third quarter balance does not reflect the net proceeds from the recent follow-on stock offering we closed on October 11 of approximately $230.8 million in net proceeds. On a pro forma basis, we had roughly $390 million of cash and short-term investments at the start of Q4.

We're well positioned with sufficient capital and resources necessary to fund our ongoing commercial launches in the U.S. and Canada and our launch in Europe in 2023 and if AMX0035 is approved. This is an exciting time for all of us at Amylyx as we deliver RELYVRIO to the patients in the U.S. for the first time. With that, I'll turn the call over to Josh for some closing remarks.

Thanks, Jim. I wanted to provide a brief status update on our Phase III PHOENIX trial. PHOENIX is progressing well, currently enrolling in Europe and on schedule. We continue to expect to recruit approximately 600 adults with ALS into the PHOENIX trial with top line data planned for mid- to late 2024.

We are on track with PHOENIX and continue to look forward to the results from this trial. To close, I want to take a moment to thank our team here at Amylyx. We are excited about the strong initial in interest that we are seeing only a couple of weeks into launch. Our goal is that every eligible person who can benefit from RELYVRIO will access as quickly and efficiently as possible. and our team is entirely dedicated to this. We look forward to keeping you all updated on our progress.

Here at Amylyx we are motivated by the community, and every day looking for even greater opportunities to make a difference in the lives of people living with ALS. Thank you for joining us and for your support. We welcome questions. Operator, please open the call to Q& A.

(Operator Instructions)

And the first question will come from Corinne Jenkins with Goldman Sachs.

Great. Sorry, my headset seems a little funky. Can you just maybe provide any detail on the kinds of questions that are outstanding from the Day 120 list of questions you got from the EMA? And then how confident are you that you'll be able to address those questions with your existing set of data?

Yes. Great question, Graham. So as you probably know, we generally don't provide granular details as we go through regulatory process on questions we're receiving and everything like that. What I can say is we have a lot of confidence in the data and the studies that we've run. We've already been through 2 regulatory approvals and processes.

But of course, every regulator has to review our package, which is only one study to support approval and come to that decision. I'd add, too, that we have an excellent team behind this. People may be aware of Tammy Sanelli, our Global Head of Regulatory, who has brought us through our first initial 2 approvals.

And we also have an excellent head of regulatory in Europe named Dan, who's doing a fantastic job there as well. So we're working through it. I think it's much too early to know what the final decision will be. But of course, we have a lot of confidence in our data and our ability to present the data.

That's helpful. And then with respect to the ongoing launch in the U.S., how should we think about the cadence for that patient assistance program? I think you mentioned patients today are all getting free drug, but how quickly do we expect that to normalize?

So I did not mention that patients today are getting free drug through the Amylyx care team in the U.S. We've just started to see our initial now that we have product and channel, we're starting to see a solid initial bolus of demand from physicians across the country. And right now, we're not giving any data as to whether or not those are EAP patients versus clinical [diag] patients. But I will say that there's very few IAP patients or PAP patients going on right now.

The next question will come from Umer Raffat with Evercore ISI.

This is Mike DiFiore in for Umer. Congrats on all the progress. A couple for me. One, when will you stop providing sales guidance and/or patient number updates will that occur sooner rather than later. And with respect to patients who are in the Q2 receive RELYVRIO, how many of them would you say are currently enrolled in commercial insurance versus enrolled in Medicare and Medicaid? And does this split reflect the broader U.S. ALS market in general? And I have 1 follow-up.

Yes. Mike, it's Jim. Thanks for the questions. I'll start with that, and maybe Margaret, you can address the insurance coverage that we see so far. But -- it's really -- we're 17 days from having drug in channel.

So it's hard to give guidance at this stage, right, in a process that's going to take 30 to 45 days to get through -- on average to get through all the insurance coverage and insurance verifications. So I expect we'll know a lot more when we talk again after Q4.

And the thing that we want to make sure of is any trends that we see and we start projecting are trends that we understand, right? So we're going to have to get a little bit of experience under our belt before we start to give projections. But as you said, I think as you've heard from each of us on the call today, we're very happy where we sit today. with the response.

Yes. And maybe just to follow up on your second question. So just as a reminder, in the U.S., our payer mix is roughly 50% commercial and 50% Medicare. It's too early to know exactly which patients in the funnel are commercial versus Medicare, but I would accept that, that national trend to actually be the breakdown of what we see coming into the Amylyx (inaudible) team.

Great. That's very helpful. And last question, in terms of the total addressable market in the U. S. Do you foresee the roughly 7,000 patients that are currently taking (inaudible) every month as the total desal market? Or could Deliver expand i t by penetrating into more of the untreated prevalence pool?

Yes. So there's 29,000 prevalent patients in the U.S. We certainly feel like that's going to be the the number that we try to target and penetrate, we definitely think that will be added on top of relive or standard of care as we were in the CENTAUR trial. And physicians really do believe in combination therapy, and I'm getting every one of these patients -- it's all of our mission to give them every shot on goal for treatment.

And we've heard from physicians that certainly, their intent is to add RELYVRIO of on top of whatever product they're on. And ultimately, our strategy is to become foundational treatment and have all the products added on to RELYVRIO.

The next question will come from Marc Goodman with SVB.

Yes. I have 2 questions. First, how long do you expect patients to stay on drug in your model? Like, how long are you modeling that? And how long is it for some of these other products that are there? And second question is just Radicava. Obviously, a new launch with the oral.

Just curious if you're seeing anything out there? Is there any way that the market's changed? Is that product doing well? Is there any impact on you with respect to your launch and the payers perfectly willing to pay for both? I'm just kind of curious what you're hearing.

Good question. So as it relates to time on therapy, we haven't given specific guidance as to time on therapy for our product. But I can say we've done some research on pass products, probably mostly on riluzole, where we see in the ballpark of the year. on average or a median. That being said, one of our hopes with having a really robust education and patient support function is that we'll be able to educate about the benefits of staying on therapy as well.

So that will certainly be one of our goals. And as it relates to the oral (inaudible) launch, I think we're very confident in the data we have, the relationships we've built and the education we've done in the market. So I think we have a lot of confidence in where we sit, as Jim said. But I don't know if, Margaret, you want to add any other insights on that?

No. Again, what we've heard is that physicians want to add-on to standard of care for most of their patients. They're excited about our data and our label and what it could offer to their patients living with ALS. And it is just in terms of public information that's out there from pharma.

I think they communicated that there's about 2, 000 patients currently on oral Radicava, roughly 4 or 5 months into the launch. What we don't know is how many of those are naive patients versus switching from IV. So that's something we'll continue to assess as we proceed through the launch.

And just to be clear, I understand the physicians want combo. The payers' discussions that you're having, it's not going to be an issue with respect to combo?

It's not coming up. We're clearly we're doing our clinical trial data, which was studied on top of riluzole and oral (inaudible). And as you know, roughly over 70% of our patients were on standard of care therapies, so we were added on there.

Right now, it's not, and we're going to continue to keep an eye on that as payers start to together there a public policy. But it's clearly a big part of our education now.

The next question will come from Neena Bitritto-Garg with Citi.

Sorry about that. So I just wanted to ask about the revenue number that was reported in Canada. Can you give us any more color on whether or not that was just reflective of the number of patients that actually are on drug or whether that was mostly an inventory build?

Yes. Neena, it's Jim. I think that you can fairly say that's reflective of the patients that are on drug. But again, not all the patients that are getting the drug are getting paid for by the insurance in Canada, given the length of time that it takes the insurance to cover it up there.

But -- no, so that reflects the revenue number of -- based on the number of patients.

Yes. And I'd say, Neena, just to say as well, we continue to believe that it will take 6 to 12 months from commercial availability to get broad reimbursement in Canada. Those are just the sort of standard time lines. And maybe the only other reminder is just that we didn't have product in channel launch in Canada until about the end of July, start of August. So that's an aspect in that revenue as well.

Got it. And then can you also talk a little bit about how long it took for the patients that are receiving reimbursed drug in Canada? Was it like a similar time line to what you're guiding to in the U.S. for them to get reimbursement?

It's really too early to answer that question because the patients that are moving on to commercial there as we secure these private pay insurers, they are moving over. So -- it's really a slow ramp-up. And as we get the additional private payers on board, we certainly hope to see more of that. So it's a very different payer landscape in the U.S. and Canada. So I don't think it's really an analog that we can use for that.

Yes. It's also important to Josh pointed out the drug was not available until the end of July. The insurance contract that we signed didn't happen until the very end of the quarter as well. So it's really -- I know people want to look at the Canadian revenues and try and use that as a crystal ball, but it's very unique and very also specific to the timing of launch and the late in the quarter timing where we got actual reimbursement from a single insurer.

So a lot more work to do in Canada, and we'll see that come to fruition kind of more early into next year where the 6 to 12 months from the late July launch comes into play.

The next question will come from Ananda Gosh with H. C. Wainright.

I'm Ananda on behalf of Andrew Fein. I saw that there was the reference of the Calpain-2, and I was wondering how should we think about the future neurodegeneration focused pipeline? And more importantly, how do you prioritize the next indication?

Yes. Great questions. So I think we were happy to share a couple of weeks ago at the annual meals conference, some data on Calpain antisense oligonucleotide that we've developed in-house that data showed a potent knockdown as well as some other in vitro results. However, I think what's important is, one, this is a very early stage product that we've just kind of started to work on. And our focus as a company squarely and purely on launch at this time. So we're continuing to evaluate and think about how we'll continue to grow the pipeline. But I think that's all enabled by having an excellent launch. And so that's where we have to have our focus and our eyes right now. And I'd say similar on the indication side, we do continue to believe that RELYVRIO, AMX0035 has potential in (inaudible) neurodegenerative diseases, and we've been hard at work evaluating which diseases are the highest kind of clinical and scientific potential. But again, I think the top priority right now for us is the launch. And those are things that will be really exciting and enabled by a successful launch.

This concludes our question-and-answer session at this time. I would like to turn the conference back over to Mr. Justin Klee for any closing remarks. Please go ahead.

Great. And thank you, everyone, for joining us on the call today. I just wanted to conclude by saying, well, we're really proud of what our team has accomplished to date. I think this is a really remarkable quarter for us. Much work remains, and I think we all feel that working in the ALS community every day.

Our focus is on ensuring that every eligible person who can benefit from RELYVRIO will have access as quickly and efficiently as possible. We know people with ALS and their families have no time to wait. So thank you all for joining us, and hope you have a good night. The conference has now concluded. Thank you for attending today's presentation. You may now disconnect.