Fulcrum Therapeutics Q2 2024 Earnings Call Transcript

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Provided by Quartr
July 31, 2024

There are 10 speakers on the call.

Operator

Good morning, and welcome to Fulcrum Therapeutics Second Quarter 2024 Financial Results and Business Update Conference Call. Currently, all participants are in a listen only mode. This call is being webcast live and can be accessed on the Investors section of Fulcrum's website at www.fulcrumtx.com and is being recorded. Please be reminded that remarks during this call may contain forward looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. These may include statements about the company's future expectations and plans, expectations, timelines and financial projections.

Operator

While these forward looking statements represent Full Time's views as of today, this should not be relied upon as representing the company's views in the future. Fulcrum may update these statements in the future, but it's not taking on an obligation to do so. Please refer to Fulcrum's most recent filings with the Securities and Exchange Commission for a discussion of certain risks and uncertainties associated with the company's business. Leading the call today will be Alex Sapir, CEO and President of Fulcrum. Joining Alex on the call are Alan Musso, Chief Financial Officer Doctor.

Operator

Pat Horne, Chief Medical Officer

Speaker 1

and Doctor.

Operator

Lynn Fazer, Senior Vice President of Development. After providing updates on our key programs, there will be a brief Q and A in which Alex, Allen, Pat and Lee will be available to answer your questions. With that, it's my pleasure to turn the call over to Alex.

Speaker 2

That's great. Thank you, Lisa, and good morning, everyone, and thanks to all of you for joining us for our Q2 conference call. We've organized today's call to provide you with updates on recent progress and upcoming milestones for our 2 clinical stage assets, losmapimod and posiridir. And after a brief introduction, we'll segue into our pipeline. I'll then ask Alan to review the financials.

Speaker 2

And finally, we'll end by taking your questions. I am happy to report that we are on track to report top line data for the Phase 3 REACH trial of losmapimod by the end of October, compared to our previous guidance of the Q4. As we advance toward this important inflection point, we continue to build out the team and add talent as we prepare for the potential NDA filing and the U. S. Commercial launch of losmapimod.

Speaker 2

In parallel, we are working with Sanofi in preparation for regulatory filings and the launch of lasmapimod outside of the United States. As a reminder, in May of this year, we announced our collaboration and license agreement with Sanofi for the development and commercialization of losmapimod for facios scapulohumeral muscular dystrophy or FSHD for short for all territories outside of the U. S. We believe we selected the best possible partner for lasmapimod as this collaboration combines Fulcrum's expertise in FSHD with Sanofi's deep regulatory, development and commercial capabilities in neuromuscular markets around the world. Together, we look forward to delivering on our shared commitment to address the high unmet need of patients in the FSHD community.

Speaker 2

Let me spend a bit more time on losmapimod, which as many of you know is an oral small molecule selective p38 alpha beta MAP kinase inhibitor that inhibits DUX4 expression and many of its downstream transcripts and thus prevents muscle cell death in patients with FSHD. An estimated 30,000 patients in the U. S. Have FSHD, which is characterized by a slow but relentless loss of muscle function year after year, resulting in significant impairment of upper extremity muscle function and mobility. And while there is a degree of heterogeneity in the onset and disease progression of FSHD, this relentless loss of muscle function means that approximately 20% of patients become wheelchair bound.

Speaker 2

I It's important to remind everyone that there are currently no approved therapies for FSHD and no drugs used off label to help these patients. Now let's turn our attention to our Phase 3 registrational trial called REACH, which I spoke about earlier. As a reminder, REACH is a 48 week Phase 3 trial intended to be registration enabling both here in the U. S. And in ex U.

Speaker 2

S. Geographies. In September of last year, we completed enrollment in REACH with a total of 2 60 patients. As presented at the 31st Annual FSHD Society International Research Congress in June of this year, baseline characteristics of the REACH study population are similar to those of our Phase 2 REDUX-four study population. Building on the encouraging clinical benefit and favorable tolerability observed in our REDUX-four trial, the primary endpoint for the REACH study is the change from baseline in the relative surface area or RSA, which is a quantitative assessment of reachable workspace and has been shown to correlate with disease severity and progression.

Speaker 2

RSA is a measure of upper extremity range of motion and muscle function that specifically evaluates shoulder and arm mobility using 3 d motion sensor technology and is indicative of the ability to perform activities of daily living. Based on collaborative interactions with the clinical outcomes assessment or COA group at FDA, we are further assessing the extent to which a specific change in the RSA score is meaningful to patients. I'm pleased to report that we are on track to complete the activities agreed upon with the FDA at the time of reporting the REACH top line data. Additional key secondary endpoints in REACH include muscle fat infiltration or MFI, which is a marker of disease pathology measured by whole body MRI, shoulder dynamometry and patient global impression of change or PEGIC for short. We believe that the implementation of self reported quality of life measures and healthcare utilization questionnaires will help inform our payer strategy as we begin preparing for commercial launch to deliver the first FSHD treatment for patients.

Speaker 2

We are now progressing towards the completion of the 48 week treatment phase of the trial for all enrolled patients. As of June 30, 2024, of the 234 out of the 260 who completed the 48 week treatment phase, 232 of those or 232 of those patients or 98% chose to enroll in Part B, the open label extension of the study in which all patients received drug. This very high percentage of patients opting to move into the open label phase is similar to what was observed in our Phase 2 clinical trial and we believe is indicative of the high unmet need for patients with FSHD. Again, we are on track to report top line data by the end of October, which will bring us one step closer to delivering the first ever FDA approved therapy for FSHD patients. Now let's talk a little bit about Posiradir, which is our oral HBF inducer for the potential treatment of patients with sickle cell disease.

Speaker 2

The elevation of HBF or fetal hemoglobin is a clinically validated therapeutic rationale for sickle cell disease, a lifelong inherited blood disorder that severely impairs quality of life for approximately 100,000 people in the U. S. And approximately 4,400,000 people worldwide, making sickle cell disease one of the most prevalent non malignant hematologic diseases. Historically, the standard treatment for sickle cell disease has included blood transfusion, pain medications and hydroxyurea that focus only on symptom relief. While exciting scientific progress has enabled the advancement and more recently the approval of gene editing therapeutic approaches, we believe there remains a high unmet need for an oral therapeutic option that is broadly protective of sickle cell disease symptomatology.

Speaker 2

As a 1st in class oral small molecule HBF inducer, we believe procirudir has the potential to address this unmet need. Turning now to our Phase 1b clinical trial, the PIONEER trial, we continue to make progress. Given that many of the sites we are newly activating our academic sites here in the U. S. As well as outside the U.

Speaker 2

S, both of which have long site activation lead times, together with our narrower inclusionexclusion criteria, it is taking longer than initially expected. We expect to have study data to share with everyone in 2025. As a reminder, Cohort 3 of the Phase 1b trial will evaluate prociridir at the 12 milligram once daily dose with a dosing duration of 3 months followed by cohort 4 at the 20 milligram once daily dose also for 3 months. Both cohorts are expected to enroll approximately 10 patients each. We look forward to building on the encouraging clinical data obtained prior to the clinical hold, which demonstrated that POCERODIER increased total fetal hemoglobin of a magnitude that could translate into a meaningful improvement in disease severity.

Speaker 2

These interim results of the Phase 1b trial involving 16 patients were recently reported in June at the EHA conference in Madrid and were very well received by the medical community. We believe that posiridir as an oral HBF inducer has the potential to provide a differentiated therapeutic option for people living with sickle cell disease and addressing the significant unmet need in the sickle cell disease community remains a key priority for us. So with that update on the business, let me now turn it over to our Chief Financial Officer, Alan Musso to run through the financials. Alan, over to you.

Speaker 3

Thanks, Alex. I'll now go over our results for the Q2 ended June 30, 2024. Let me start with our cash position. As of June 30, 2024, cash, cash equivalents and marketable securities were $273,800,000 as compared to $236,200,000 as of December 31, 2023. The increase in our cash position is due to the $80,000,000 upfront payment received from Sanofi in the Q2 of 2024, partially offset by cash used to fund our operating activities in the first half of the year.

Speaker 3

Collaboration revenue was $80,000,000 for the Q2 of 2024 compared to $900,000 for the Q2 of 2023. The increase of $79,100,000 was primarily due to recognition of the $80,000,000 upfront license payment received from Sanofi during the Q2 of 2024. Research and development expenses were $17,300,000 for the Q2 of 2024 compared to $17,800,000 for the Q2 of 2023. The decrease of $500,000 was primarily due to the luspamod global development cost sharing reimbursement from Sanofi, partially offset by increased costs related to the advancement of the REACH trial. The general and administrative expenses were $10,200,000 for the Q2 of 2024 compared to $10,300,000 for the Q2 of 2023 and the the $100,000 decrease was primarily due to lower employee compensation costs.

Speaker 3

For the Q2 of 2024, we had net income of $55,400,000 compared to a net loss of $23,800,000 for the Q2 of 2023. For the foreseeable future, excluding the potential for future milestone payments under our Sanofi collaboration, we expect to be in a loss position, including for the year ended December 31, 2024. And finally, turning to our cash runway guidance. Based on our current operating plans, we continue to expect that our existing cash, cash equivalents and marketable securities will be sufficient to fund our operating requirements into 2027. And with that, let me turn the call back over to Alex.

Speaker 2

Great. Thanks so much, Alan. So with that overview of the business and the financials, Lisa, let's go ahead and open it up for questions.

Operator

Thank And our first question will come from Corinne Johnson of Goldman Sachs. Your line is open.

Speaker 4

Maybe first, with respect to the work that you're doing to validate regional workspace for the agency, has the agency specified like specific metrics that are most important or the magnitude of changes like the change or the benefit they'd like to see or are they just asking for, kind of like general proof that reachable workspace is potentially beneficial? And then are there multiple analyses that they requested and do they all sort of have to go in the same direction? And then, my other question would just be in terms of the cash runway guidance you just provided in '27, what specific, like clinical milestones and then commercial activity?

Operator

Thanks.

Speaker 2

Yes, it's great. Thanks, Corinne. And thanks for the question. And if there are any more follow-up questions, maybe just speak up a little bit. You were a little bit difficult to hear.

Speaker 2

I think in terms of your questions around Reachable Workspace and some of the work we're doing to prove out the clinical meaningfulness of that work. I'll turn that one over to Ian and then I'll ask Alan to provide an update on your question around the financials. So Ian?

Speaker 1

Yes, sure. Thanks, Corinne. So since the Reachable Workspace has not previously been used for any approvals, there are no pre established criteria. And so there has not been a numerical criterion put forward by the agencies for this. And the work we are doing is in order to evaluate that across a range of different approaches leading towards an understanding of the meaningful score difference, which is the FDA term for a change within a given patient that is considered meaningful.

Speaker 1

So as I say, there is no pre specified numerical value for that and the results coming out of the work that we are doing will inform that.

Speaker 2

And then Alan, maybe toward the second question that Karim had.

Speaker 3

Yes, sure Karim. Good morning. Yes, the cash runway guidance that we're giving that does anticipate a commercialization of losmabumab. It also funds the completion of the ongoing PASERDIO trial as well as the follow-up trial that we anticipate and funds preclinical work that we have ongoing for which we expect that we'll have new products entering into the clinic from that work. So it's sort of all in what we anticipate with the current portfolio of the continued advancement.

Speaker 4

Thanks. Sorry for the audio quality.

Speaker 2

No worries. Thanks, Corinne. Lisa, next question?

Operator

Thank you. And our next question will be coming from the line of Kristin Kreuzov of Cantor Fitzgerald. Your line is open.

Speaker 5

Phase, we've been getting a lot of questions just because another company also had data on this. And to your point, given it really is a new endpoint and hasn't been previously used for approval, wondering now that we have 2 data sets supporting the endpoint, if this helps in your opinion derisk this endpoint for this disease?

Speaker 2

Yes, that's great. Hi, Kristen, and thanks for the question. I think to answer that, I'll turn it over again to Ian.

Speaker 1

Yes. Thanks, Kristen. I think it is encouraging for us to see that others are using this particular endpoint in FSHD. We certainly had hints from the agency that that's the case that they're hearing about this as we engage with them around our discussions around reachable workspace. And I think it also reinforces the point that we've made previously that we haven't been suggested an alternative primary endpoint in FSHD.

Speaker 1

So I think we're overall encouraged by those reports that have been coming out.

Speaker 2

Yes. And the only the thing I would add, Kristen, this is Alex, is and you had mentioned some other data that had recently been published or reported out. I assume you're referring to the AVIDITY data. I think what the AVIDITY data does is it certainly validates not only the DUX4 pathway, but more specifically to your question, reachable workspace as the clinical endpoint, we believe that many of the registrational trials that will come after us will be required as part of their primary endpoint.

Speaker 5

Okay. Appreciate that. And then assuming that the trial is successful, when you plan to meet with the FDA, can you just remind us first what the safety data set is including from the previous company that had studies in other indications? And then second, how much open label extension data you're going to have from Phase 2 at that point to add on to the pool of evidence? Thanks so much again.

Speaker 2

Yes. Thanks, Kristen. Maybe I'll take the first question and I'll turn the second question over to Ian. Yes, I think that is really one of the truly unique things about losumabimab. I don't know if this is unprecedented, but we will be filing our new drug application with a patient safety database that includes over 3,600 patients.

Speaker 2

So I think for a rare disease such as FSHD with a prevalence of 30,000 to have 3,600 patients in the patient safety database is quite remarkable. And I think one of the encouraging signs is the drug does have a fairly sort of unremarkable AE profile and we've demonstrated that our REDUX-four study and expect to replicate that in our REACH study. And then Alan sorry, Ian, maybe to Kristen's second question.

Speaker 1

Yes. So we'll have data from the REDUX for Phase 2 study. Remember that enrolled 80 patients and there's been a high rate of retention in the open label expansion of that study, now up to exceeding 3 years of exposure in some of those patients, the ones that were enrolled at the very earliest in that study. We have about 11 patients in a separate open label study in Europe that also has a prolonged duration of treatment. And then in the REACH study itself, there are 2 60 patients.

Speaker 1

And as Alex mentioned in the original remarks, a very high proportion of those are electing to roll over into the open label extension. And so they will continue to experience exposure to lasmapimod as we move towards filing. So there is not only a large safety database from other indications that preceded the work that we had done, but also a large and growing safety database for this relatively rare disease.

Speaker 5

Thank you.

Speaker 2

That's great. Thanks, Kristen. Lisa, next question?

Operator

Thank you. And our next question will be coming from the line of Joseph Schwartz of Leerink Partners.

Speaker 6

Hi, thanks for the updates. Sorry. So I was wondering a couple of things on Posiridir first and then lasmapimod. Can you talk a little bit more about why it's taking longer to proceed with the Cohort 3 group of patients for prociridir? Is this at the IRB level?

Speaker 6

Is it enrollment? Can you give us any clearer progress update and maybe talk about what initiatives you have to accelerate things? And then I have a question on Losmabumab.

Speaker 2

Sure. That's great, Joe. Thanks for the question. This is Alex. I'll take the first one and then depending on what the second question is, we'll figure out who we can to triage that one too.

Speaker 2

Yes, so as I mentioned, and maybe I'll give a little bit more color. The 7 or 8 sites that were involved in the PIONEER trial prior to the initiation of the clinical hold, which happened, I think, in February of 2023. Most of those sites tended to treat younger patients that didn't have the disease severity that was matching our new inclusionexclusion criteria that we reached agreement on with the agency in order to get off clinical hold. So many of those existing sites would have been terrific to reactivate. It would have taken several months to get the new protocol through the IRB.

Speaker 2

All the contracting had been done. But because what we were hearing from those PIs is that they did not have those patients. Essentially, we had to activate all new sites, both in the U. S. As well as in countries outside of the U.

Speaker 2

S, specifically in Africa. Our goal is still to activate about 15 to call it 17, 18 sites in the U. S. And a handful of sites outside of the U. S.

Speaker 2

So we should have 20 sites, which should be more than adequate to enroll these 20 patients in these next two cohorts. So I think it's really just it's this long lead time of activating these sites, many of which are academic sites. And as many of you know, it can take up to 9 months from contracting to IRB approval, all of the back and forth at the sites to get these sites activated again at these major academic institutions. In terms of what we're doing to try to speed that up, I think we're trying to do I think we're doing a couple of things. Number 1, when a contract provision comes in, we're typically returning that around in about 24 to 48 hours.

Speaker 2

So the people in our legal group knows that Pioneer is a top priority and anything that comes in from Pioneer gets put to the top of the pile. Unfortunately, we're sort of at the whim of the large institutional bureaucracy and unfortunately things just take time. But I will say that of the sites that we have activated and we have activated a number of sites, they continue to be very encouraged of the transformational potential that pociridir could bring to their patient population. There is a relatively small number of patients somewhere between 7.5% 10% of that 100,000 that I mentioned that do meet our inclusion, exclusion criteria, so call it about 10,000 patients. But we believe that's more than an adequate number to be able to recruit these 20 patients.

Speaker 2

And as I mentioned, we will expect to have data that we can share with everybody sometime in 2025. And then maybe turning to your lasmapimod question.

Speaker 6

Yes, thanks. That was helpful color on Vasyrdir. So as far as lasmapimod goes, I was wondering if you could talk about the output that will emerge from the work to define the minimally clinically relevant difference on the RWS. Is this just one single number? Is it a range which might provide more context?

Speaker 6

And how do you feel about the ability of losmapimod to provide a benefit which exceeds whatever you define as the MCD?

Speaker 2

Sure. Thanks, Joe. I think to answer that, let me turn that one over to Ian as well.

Speaker 1

Yes. Thanks, Joe. And just to clarify, I think the number that in the FDA terminology is the meaningful score difference. And I think the important thing to emphasize about that is that that's not a mean score for the overall population that needs to be exceeded, but that's the score for within patient change. So that's understanding what a change in the reachable workspace score is for an individual patient.

Speaker 1

And so what that's most likely to be reflected in looking at the clinical trial data is proportions of patients individual patients that exceed that meaningful score difference. So I think that's just conceptually a way of thinking about it. That's an important piece of it. And that's really the focus of this. There might well be a range of these depending on the outputs of that work and we'll report that at the time of the top line data.

Speaker 3

Very helpful. Thank you.

Speaker 2

Thanks, Joe. Leak, next question?

Operator

And our next question will be coming from the line of Daegung of Stifel. Your line is open.

Speaker 7

Hey, great. Good morning. Thanks for taking our questions. I'll stick my two questions with the lasmapimod side of things. Specifically on powering, so maybe a question for Ian.

Speaker 7

So the first question is you previously talked about powering for reach as having benefited from the over enrollment. I think it was 96% to show 10% placebo adjusted RSA. Just to clarify, was this just for the FSHD Type 1 or inclusive of Type 2? And now that you do have the Type 2 baseline characteristics disclosed like what would that powering be if any different from the original one? And then second question on powering as well.

Speaker 7

Just curious, have you guys done additional sensitivity analysis around sort of the evolving RESOLVE natural history data and what might that mean for the powering of REACH? Thanks so much.

Speaker 2

That's great. Thanks, Dae Gon, and thanks for the question. Yes, Ian, you want to take that one?

Speaker 1

Yes. Hi, Dae Gon. As I think we've articulated before, the powering for reach was based on specifically the FSHD1 patient population and that's because the data that we used for the powering was from REDOX 4 and that study enrolled only FSHD Type 1s. And so that was the approach that was taken even though the primary endpoint for the REACH trial will include not only the Type 1s but also the Type 2s. And so the powering initially the powering was based on 210 FSHD Type 1 patients and that was the powering at 93% And the expectation was that we would in addition have 20 FSHD Type 2 patients, so push the total up to 230.

Speaker 1

The over enrollment pushed the total enrollment from 230 to 260 and the Type 1s went from 210 to 242. And so that's really the comparison for the powering was the 210 to 242 Type 1s moving it from 93% to 96%. As you've indicated, we have the baseline characteristics. We know there are 18 FSHD Type 2 patients, and we also know that the randomization is stratified for that. So the expectation is that there'll be 9 and 9 in each of the groups, placebo and active.

Speaker 2

Great. Thanks again for the question, Dae Gon. Lisa?

Operator

Thank you. And our next question will be coming from the line of Gregory Redfin.

Speaker 8

Yes. Hey, good morning, Alex and team. Congrats on the progress. We're looking forward to the data in October. Alex, just on lizumabimod, as you touched a bit upon payer engagement, maybe just give us a glimpse of what that engagement will and has been looking like?

Speaker 8

What do you see as the potential tailwinds from the potential data package and lasmapimod profile? And maybe what do you foresee as some of the greatest challenges when it comes to establishing the value proposition and assuming that the data cards slipped as you foresee?

Speaker 2

Yes, great question and thanks for that. Yes, I think so we've done some payer research following the results of the REDUX for study results. And what we heard from the payer community, and again, we probably went out and talked at the time to about 15 or so payers. The majority of those were U. S.-based payers.

Speaker 2

And I think what we heard across the board is, it will be difficult to restrict access to this drug when approved for patients given the fact that there are clearly nothing available and nothing used off label to help these patients with FSHD. I think the other thing that we heard from that payer research when probing a bit on what they expected the pricing to look like for the drug and we have not given any specific guidance around pricing. But what we clearly heard back from the payers is they would expect this to look very similar to other rare disease drugs that have been priced in the call it 100 of 1,000 of dollars every year per patient. I think it's also important to remind people that patients that will start on this drug, we assume will more than likely stay on this drug for very, very long periods of time. And we've been seeing that now greater than 85% of patients in the REDUX 4 study are still on drug after a 3 year period of time.

Speaker 2

So once we get the REACH trial results, Greg, we'll go ahead and do more significant payer research. But to your question around the tailwind, and we spent a lot of time thinking about this, I think that one of the biggest tailwinds we'll have with payers is the requirement for a confirmed genetic test at the time or prior to the approval of the drug. And what we know right now from the work that we've done is only about 20% to 30% of patients are actually receiving a confirmed genetic test. So if every payer we're operating under the assumption that payers will require that confirmed genetic test before approving the drug. And so we're doing a lot of work now.

Speaker 2

And there is a lot of examples out there of other rare diseases in which payers have required genetic tests. So this is not a new concept. We're essentially sort of replicating the playbook of many other rare disease products that have come before us. But we will make sure at the time of launch that a lack of a or we will make sure that genetic testing does not become an impediment to access and whether that's through a partnership that we do with the FSHD Society or whether we do it directly and provide genetic testing free of charge to patient, we're working through that right now to make sure that at the time of launch that that does not become the tailwind that you mentioned.

Speaker 8

That's helpful. Maybe related and as Alan has provided some detail on your runway, just how are you coordinating the urgency about the planning for building a commercial organization when it comes to the leadership in such a capability and slotting that with respect to the data coming?

Speaker 2

Yes, great question, Greg, and thanks for asking it. Yes, so we are we have begun building out our commercial organization. I would expect that sometime in Q3 of this year, we will announce the appointment of our Chief Commercial Officer. And one of the things that I said to this individual, while I was interviewing with them is we will make sure that we properly resource this launch. We will make sure that if it's a question of do we fund it or do not fund it, we will always on we should fund it instead of not funding because we have to make sure that this is properly resourced.

Speaker 2

And as Alan mentioned in one of the earlier questions, the runway guidance that we have provided assumes a launch that we have very properly resourced beginning in 2026. Is that fair, Alan? Okay.

Speaker 8

Got it. Thanks as always for the color.

Speaker 2

Yes. Thanks so much, Greg.

Operator

Thank you. One moment for the next question. And our next question will be coming from Matthew Begleiter of Opco. Your line is open.

Speaker 9

Hey guys, good morning. We were curious if in your discussions with the FDA they'd ever asked for more data around losmabumab mechanism of action. As you mentioned, Avedity had some biomarker data on DUX4 reduction clinically. I know pre clinically you showed that, but you hadn't been able to clinically. So I'm just kind of curious if you'd be open to rerunning archived biopsies from Redux using maybe a more sensitive assay if the FDA wanted you to or if not, if you think really at this point the FDA is only concerned with validating the RWS tool?

Speaker 9

Thanks.

Speaker 2

Yes. Thanks, Matt. Great question. And let me turn that over to Ian as well.

Speaker 1

Hey, Matt. Thanks. The issue of going back to biomarkers has not been something that's come up in any of the discussions with the agency. The focus I think is very much been on the endpoints given that it's a functional endpoint and the secondary endpoint similarly are functional or related to muscle health. So we haven't up to this point received any specific requests around that.

Speaker 1

So we don't have any specific plans to do so. The evidence that we have both in the original discovery of lasmapimod as well as in the characterization of it was done in muscle cells derived from patients with FSHD. And you can perform the characterizations of the effects on DUX4 and all the downstream transcripts pretty convincingly in that in vitro system where you don't have competition from other cell types and issues related to the biopsy and so on. And I think those data are very robust and show the mechanism of action on of lasmapimod on the reductions of DUX4 in the downstream transcripts.

Speaker 9

Okay. That makes sense. Thanks for that, Ian. Maybe just squeeze 1 on the Reach III guidance. Is this a revision or just more fine tuning from the prior guidance, which was year end?

Speaker 9

And if it's a revision, kind of what are some of the factors driving that faster cadence going from year end to now end of October? Thanks again.

Speaker 2

Yes, Matt, this is Alex. Yes, no, it's nothing more than simply fine tuning. As we're getting closer and we're seeing the last handful of patients having their last visit, we feel very confident in terms of our ability to report out the top line results by the end of October.

Speaker 3

Appreciate it. Looking forward to it.

Speaker 2

Great. Thanks so much, Matt.

Operator

Thank

Speaker 5

you.

Operator

There are no more questions in the queue. I would like to turn the call back to Alex for closing remarks. Please go ahead.

Speaker 2

That's great. Thanks so much, Lisa. And again, thanks to everybody for joining. Thanks to everyone for your interest in all the great things we're doing here at Fulcrum. And I guess before we conclude, I want to remind everyone that we continue to make progress with our Phase 1 PIONEER trial of posiridir and are on track to report top line data for the Phase 3 REACH trial by the end of October of this year.

Speaker 2

In parallel, we continue to prepare for the potential NDA filing and commercial launch of losmafimod in the U. S. With our cash runway into 2027, we believe we are well positioned to execute on our corporate objectives and look forward to building on this momentum in the months years ahead. And as I always like to do before we jump off the call, I would be remiss if I did not extend my sincere appreciation and gratitude to my fellow Fulcrum teammates, to the physicians we work with to advance our clinical studies, to our partners at the FSHD Society, and finally and most importantly to the patients and their families around the world. Without you and your commitment to become involved in clinical trials, none of what we do would be possible.

Speaker 2

Thanks again to everyone who joined the call this morning. Please stay safe and healthy. Thanks so much.

Operator

Thank you. For today's conference call, you all may disconnect.

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Fulcrum Therapeutics Q2 2024
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