Fulcrum Therapeutics (ticker: NASDAQ:FULC) has entered into a significant collaboration with Sanofi (NASDAQ:SNY) for the development and commercialization of losmapimod, aimed at treating facioscapulohumeral muscular dystrophy (FSHD) outside the United States. This partnership includes an $80 million upfront payment to Fulcrum, with the possibility of an additional $975 million based on regulatory and sales milestones, as well as tiered royalties.
Fulcrum retains its focus on the US market, planning a commercial launch in 2026. The company also updated on pociredir, its treatment for sickle cell disease, and reported a net loss of $26.9 million for Q1 2024 despite decreased G&A expenses. Fulcrum's cash reserves are projected to fund operations into 2027.
Key Takeaways
- Fulcrum Therapeutics announced a collaboration with Sanofi for losmapimod's commercialization outside the US.
- Fulcrum to receive $80 million upfront, with potential for up to $975 million more, plus royalties.
- Phase 3 trial of losmapimod expected to report in Q4 2024; US commercial launch targeted for 2026.
- Fulcrum to reinitiate Phase 1b study of pociredir for sickle cell disease.
- As of March 31, 2024, Fulcrum reported $213.3 million in cash reserves.
- No collaboration revenue in Q1 2024; net loss of $26.9 million reported.
- Fulcrum is hiring a chief commercial officer and additional staff for market access.
Company Outlook
- Fulcrum's cash reserves, bolstered by the Sanofi payment, are expected to support operations until 2027.
- The company is preparing for a New Drug Application (NDA) filing and commercial launch in the US.
Bearish Highlights
- The company reported a net loss of $26.9 million in the first quarter of 2024.
- There was no collaboration revenue during Q1 2024, compared to $0.3 million in the same period of 2023.
Bullish Highlights
- Fulcrum's collaboration with Sanofi is a strong endorsement of losmapimod's potential.
- The company's engagement with European regulators has been positive, with agreement on trial design and endpoints.
Misses
- The decrease in collaboration revenue year-over-year indicates a potential area of underperformance.
Q&A Highlights
- CEO Alex Sapir emphasized the importance of hiring a chief commercial officer experienced in rare disease treatments.
- Sapir praised Sanofi's neuromuscular franchise and regulatory expertise as beneficial for global commercialization efforts.
- Fulcrum is working closely with the FDA and European regulators on clinical trials and endpoints for losmapimod.
- The deal with Sanofi has been in the works for over a year, with Sanofi privy to confidential program materials.
In conclusion, Fulcrum Therapeutics is strategically aligning with Sanofi to maximize the potential of losmapimod globally while gearing up for a US launch. Despite a reported net loss, the company's financial position appears stable, with sufficient funding to carry through near-term operational plans. As Fulcrum prepares for pivotal trial results and navigates the regulatory landscape, its partnership with Sanofi and proactive staffing for commercialization signal a forward-looking approach to bringing its therapies to market.
InvestingPro Insights
Fulcrum Therapeutics (FULC) is navigating a dynamic phase, bolstered by its strategic alliance with Sanofi. The company's financial health and operational strategies are key factors for investors. Here are some insights based on InvestingPro data and tips:
- Fulcrum's market capitalization stands at $464.29 million, reflecting investor valuation of the company in the current market.
- The company's price-to-earnings (P/E) ratio is -4.69, indicating that investors are valuing the company despite its lack of profitability in the last twelve months.
- With a revenue decline of -55.77% over the last twelve months as of Q4 2023, Fulcrum faces challenges in generating sales growth, which is critical for its long-term success.
InvestingPro Tips reveal that Fulcrum holds more cash than debt on its balance sheet, which is a positive sign for financial stability. However, the company is quickly burning through cash, which could be a concern if not managed efficiently. Analysts have revised their earnings downwards for the upcoming period, possibly due to the anticipated sales decline in the current year. Moreover, Fulcrum's stock price movements have been quite volatile, which could be a risk factor for investors seeking stability.
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Full transcript - Fulcrum Therapeutics Inc (FULC) Q1 2024:
Operator: Good morning and welcome to Fulcrum Therapeutics' First 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 made 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, clinical development, timelines, and financial projections. While these forward-looking statements represent Fulcrum'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; Dr. Iain Fraser, Senior Vice President of Early Development; and Paul Bruno, Senior Vice President of Business and Corporate Development. After providing updates on our key programs, there will be a brief Q&A in which Alex, Alan, Iain, and Paul will be available to answer your questions. With that, it's my pleasure to turn the call over to Alex.
Alex Sapir: That's great. Thanks, Shannon. And thanks to all of you for joining us on the call this morning. Beyond another solid quarter of progress across our two clinical stage assets, losmapimod and pociredir, we are super excited to announce our collaboration and license agreement with Sanofi for the development and commercialization of losmapimod. So what I'd like to start off by doing this morning is just provide an overview of the structure and strategic importance of this collaboration with Sanofi. In brief, Sanofi will obtain exclusive commercialization rights for losmapimod outside of the US and that will allow Fulcrum to focus our efforts on planning for a strong US commercial launch in 2026. This collaboration allows both parties to do what we each do best. It combines Fulcrum’s expertise in FSHD with Sanofi's exceptional regulatory development and commercial capabilities across approximately 100 countries outside the US. Importantly, having a partner like Sanofi will allow us to reach patients in ex-US markets quicker than we could do on our own and mitigates the commercial execution risk that is typically inherent for any biotech company undertaking the commercial launch of its first product on its own. We believe we have selected the best possible partner for losmapimod, and we look forward to delivering on our shared commitment to addressing the unmet need of patients with FSHD. We're also very pleased with the deal terms, which allows us to retain substantial ex-US value in both the near term and over the product life cycle. In short, we will receive $80 million upfront which enables us to fund the launch of losmapimod in the US with non-dilutive capital. We're also eligible to receive up to an additional $975 million in specific regulatory and sales milestones, along with the tiered royalties starting in the low teens of annual net sales of losmapimod outside of the US. The companies will also share equally in the global development cost for losmapimod going forward. Now before providing an update on our two clinical assets, pociredir and losmapimod, I would also like to take the opportunity to welcome our newly appointed Chief Medical Officer, Dr. Patrick Horn, an accomplished industry veteran who has successfully guided multiple therapies in the rare disease space through late clinical development, regulatory approval, and commercial launch. This is an ideal time for Pat's appointment, and with the complementary expertise of Dr. Ian Fraser, we are well positioned as we advance toward near term inflection points. Pat does send his apologies as he's unable to attend today's meeting on account of a family emergency. So let's go a bit deeper and start with our most advanced program, losmapimod, which, as most of you know, is an oral small-molecule selective p38 alpha/beta MAP kinase inhibitor that inhibits DUX4 expression and thus prevents downstream muscle death in patients with FSHD. As a quick reminder, FSHD is a rare form of muscular dystrophy with an estimated US prevalent patient population of 30,000. FSHD 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, many patients are unable to perform daily life activities that you and I take for granted, and thus are unable to live independently. Ultimately, 20% of FSHD patients become wheelchair-bound. There are currently no approved treatment options for these patients, which underscores the significant unmet need for this debilitating disease. Our Phase 2 study, the ReDUX4 trial, was recently published last month in Lancet Neurology and demonstrated improvements in functional outcomes as measured by reachable workspace, structural outcomes as measured by muscle fat infiltration, and patient-reported outcomes for patients treated with losmapimod, all as compared to placebo. Building on the encouraging clinical benefit and favorable tolerability observed in our Phase 2 trial, we completed enrollment in our global Phase 3 trial for losmapimod in September of last year with a total of 260 patients. And as of the end of April of this year, 146 of the 260 patients had completed the 48-week treatment period, and 144 of these patients elected to continue into the open-label extension of the study in a fully blinded fashion. 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 indicative of the high unmet clinical need for patients with FSHD. We are on track to report top line in the fourth quarter of this year, which will bring us one step closer to delivering the first ever FDA-approved therapy for patients with FSHD. Now, as a quick reminder, our Phase 3 trial, the REACH trial, it is a well-powered 48-week trial intended to be registration enabling both in the US and in ex-US geographies. The primary endpoint for REACH is the change for baseline in the relative surface area or RSA, which is a quantitative assessment of reachable workspace. RSA is a measure of upper extremity range of motion and muscle function that specifically evaluates shoulder and arm mobility using 3D motion sensor technology and has been shown to correlate with abilities to perform certain activities of daily living. In our Phase 2 study, losmapimod demonstrated a 10% net change in the RSA score relative to placebo at 48 weeks. Based on our collaborative interactions with FDA, and in particular with the Clinical Outcomes Assessment group, or COA, at FDA, we are currently assessing the extent to which a specific change in the RSA score is meaningful to patients. Additionally, key secondary endpoints include muscle fat infiltration, or MFI, which is an important marker of disease pathology measured by whole body MRI, shoulder dynamometry, as well as self-reported quality of life measures and healthcare utilization questionnaires that will help inform our thinking on our payer strategy as we begin for our commercial launch here in the US. Taken together, we continue to take important steps forward to position Fulcrum for commercial readiness. Now, turning to pociredir, our oral HbF inducer for the potential treatment of patients with sickle cell disease, or SCD for short. The elevation of fetal hemoglobin, or HbF, is a validated therapeutic rationale for SCD, a lifelong inherited blood disorder that severely impairs quality of life for approximately 100,000 people in the US and approximately 4.4 million people worldwide. This makes sickle cell disease one of the most prevalent non-malignant hematologic diseases. Now, historically, the standard of treatment for SCD has involved blood transfusions, pain medications, and hydroxyurea, but all of these treatments only focus on symptom relief. And 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 a safe and accessible therapeutic option that are broadly protective of SCD symptomatology. As a first-in-class oral small molecule HbF inducer, we believe pociredir has the potential to address this unmet need. So in August of 2023, the FDA lifted the clinical hold on the Phase 1b study we call the PIONEER study. It's important to note there were no changes in the protocol-defined dose escalation scheme or the three-month treatment duration. We continue to work expeditiously to reinitiate the PIONEER study, and we have activated several new sites and are building key relationships with leading physicians in the SCD community. Cohort 3 of the phase 1b trial will evaluate pociredir at the 12 milligram once-daily dose with a dosing duration of three months, followed by Cohort 4 at the 20 milligram once-daily dose also for three months. Both cohorts are expected to enroll approximately 10 patients. We look forward to building on the encouraging clinical data obtained prior to the clinical hold, which demonstrated that pociredir increased total HbF of a magnitude that could translate into a meaningful improvement in disease severity. More specifically, after only 42 days of treatment, we observed up to a 10 percentage point increase in HbF from baseline or total HbF of approximately 25%. We believe that pociredir as an oral HbF inducer has the potential to provide a differentiated therapeutic option for patients living with sickle cell disease, addressing the significant unmet need in sickle cell in the sickle cell community remains a key priority for us and we are excited to build on this momentum in the years ahead. And so with that update on the business, let me now turn it over to our Chief Financial Officer, Alan Musso, to run through our financials. Alan, over to you.
Alan Musso: Thanks, Alex. I'll now go over our financial results for the first quarter ended March 31, 2024. As of March 31, 2024, cash, cash equivalents and marketable securities were $213.3 million, as compared to $236.2 million as of December 31, 2023. The decrease in our cash position of $22.9 million is due to net cash used in operating activities. As of March 31, 2024, on a pro forma basis, considering the $80 million milestone due under the collaboration and license agreement with Sanofi, our cash, cash equivalents and marketable securities were $293.3 million. We had no collaboration revenue in the first quarter of 2024 compared to $0.3 million for the first quarter of 2023. The decrease was attributable to the completion of our research services during the fourth quarter of 2023 under our myocardia collaboration agreement. Research and development expenses were $19.8 million for the first quarter of 2024, as compared to $16.7 million in the first quarter of 2023. The increase of $3.1 million was primarily due to increased costs related to the advancement of the REACH clinical trial. General and administrative expenses were $10.1 million for the first quarter of 2024 as compared to $11.5 million for the first quarter of 2023. The decrease of $1.4 million was primarily due to decreased employee compensation costs. And our net loss was $26.9 million for the first quarter of 2024, as compared to $24.8 million for the first quarter of 2023. Finally, based on our current operating plans, we now expect that our cash, cash equivalents and marketable securities, together with the $80 million upfront payment to be received from Sanofi, will be sufficient to fund operating requirements into 2027. With that, let me turn it back over to you, Alex.
Alex Sapir: That's great. Thanks so much, Alan. So before opening up for questions, Shannon, I just wanted to remind everybody of the key events for this quarter. We selected the ideal partner in Sanofi to commercialize losmapimod outside the US. We extended our cash runway into 2027. We enriched our leadership team and we activated several new sites for our Phase 1b PIONEER trial of pociredir. We remain on track to report top line data for the Phase 3 Reach trial in the fourth quarter of 2024 and continue to prepare for the potential NDA filing and commercial launch of losmapimod in the US. We look forward to building on this momentum in the months and years ahead. And with that Shannon, let's go ahead and open it up for questions.
Operator: Thank you. [Operator Instructions] Our first question comes from the line of Edward Tenthoff with Piper Sandler. Your line is now open.
Edward Tenthoff: Great. Thank you very much, and congratulations on the partnership and all the progress in the quarter.
Alex Sapir: Thanks, Ed.
Edward Tenthoff: So, quick question. When it comes to actually manufacturing drug and things like that for the partnership, can you walk us through how that works? And then just anything else we should think about in terms of final things that need to happen ahead of the Phase 3 data. Obviously final patient visits and all of that stuff, but just anything else that you guys are focused on as we drive towards the data in the fourth quarter for losmapimod? Thank you.
Alex Sapir: Yeah, that's great, Ed. Thanks so much. And joining us on the call is Paul Bruno, and Paul is our Head of Corporate Strategy and Business Development, really the individual at Fulcrum who really led the deal efforts with Sanofi. So maybe, Paul, I'll turn that over to you to address the manufacturing question and then some of the more general questions Ed had.
Paul Bruno: Yeah, thanks, Ed. So right now the plan is for us to continue supplying for both clinical and commercial globally. There is the opportunity and flexibility for Sanofi to take over manufacturing and supply in the future.
Edward Tenthoff: Great. Thank you. And then just in terms of final work towards the Phase 3 readout, anything we need to be focused on? Thanks.
Paul Bruno: Yeah. Nothing offhand, but maybe I'll turn it over to Iain.
Iain Fraser: This is Ian, Ed. No, that's all pretty much on track and unaffected by this. We are proceeding with that. We know who the last patients in the study are, where they are, and obviously we're working with those sites to make sure there are no delays there. But that continues unaffected at this point.
Edward Tenthoff: Well, tough to imagine a better partner. Congratulations, guys.
Alex Sapir: Yeah, thanks so much, Ed.
Operator: Thank you. Our next question comes from the line of Gregory Renza with RBC Capital Markets. Your line is now open.
Gregory Renza: Great. Good morning, Alex and team. Congrats on the progress in the deal and thanks for taking the question.
Alex Sapir: Thanks, Greg.
Gregory Renza: And maybe just a couple from us. Just first question, when we look across the competitive landscape for FSHD development, especially when it comes to trial design and endpoints, maybe beyond DUX expression, we do know that Avidity plans to report data on the changes to gene expression downstream to DUX4. So I'm just curious, what can you tell us about losmapimod’s ability to impact that expression downstream of DUX4, and maybe how can we think about that and most probably by being positioned competitively in light of this? And then I have a follow-up.
Alex Sapir: Yeah, great question, Greg. And let me start and then I'll turn it over to Iain for some more detail. And yes, Avidity has talked a lot about the fact that AOC 1020 directly inhibits DUX4. I think that the clinical impact of that still remains to be seen. They have talked a lot about being able to measure genes downstream to DUX4 because of the challenges that we've spoken about and they've spoken about, about the stochastic nature of DUX4 and how difficult it is to measure. Now we do know that losmapimod works upstream from DUX4, but we also know that losmapimod can reduce DUX4 gene expression in preclinical models. And we also know that losmapimod has been able to reduce gene expression that are downstream to DUX4 in preclinical models as well. And maybe, I guess Iain, just to elaborate on that a little bit, maybe I'll turn it over to you to maybe talk in a bit more detail about some of the genes that we have been able to reduce downstream to DUX4.
Iain Fraser: Yeah, absolutely. Yeah, all the work that was done to identify losmapimod as a potentially useful agent in FSHD was done in vitro in cells derived from patients with FSHD. The myoblasts from those patients differentiated in vitro into myotubes. In that process, DUX4 typically is upregulated. We've been able to show across a range of cells derived from different patients, both FSHD1 and FSHD2, that there very clearly is a significant reduction in DUX4 itself, which you can measure in-vitro somewhat more readily than you can in vivo. And that along with those concentration-dependent reductions in DUX4 across those myotube cultures, you see a concomitant decrease in a number of the downstream genes that we know are impacted by DUX4, that are regulated by DUX4, and show a concentration-dependent decrease in all of those, as well as decreases in markers of apoptosis in the muscles and ultimately decreases in muscle cell death. So that mechanism is fully fleshed out in cultures, and as Alex alluded to, the challenge in the clinic is demonstrating that in the biopsies from these patients. And I think one of the challenges in addition to the stochastic nature of DUX4 expression is the variability in the tissue content of the biopsies. In the clinic, these are not normal muscles, so there's a bunch of fat, fibrous tissue, inflammatory tissue, extracellular matrix components and so on that lead to the variability in documenting that. But the overall pathway in vitro in those myotube cultures is very clear, concentration dependent reduction in DUX4 and similarly reduction in the downstream gene impacted by DUX4.
Gregory Renza: That's great, really appreciate that. And then just on the deal this morning, I certainly heard your mention of the benefit of Sanofi reaching multiple countries and you’re building now to focus stateside and on the US. Just wanted to ask, just to receive a little more color and maybe some of those additional inputs that you and the team had in consideration of these deal terms reflecting the full value of losmapimod. But could you just talk through some of the deal terms and what inputs that you use to feel comfortable with the value calculus here? Thanks so much and congrats again.
Alex Sapir: Yeah, thanks, Greg. And I'll start, and then Paul if I missed anything, please jump in. I think first and foremost, we've been very clear that it has always been our goal to try to find a partner outside of the US. And I think the reason for that is, finding a partner outside the US allows us to do a couple of things, Greg, right? It allows us to get losmapimod to patients around the world faster than we could do on our own simply because of the scale of the commercial infrastructure that Sanofi has. And then with the upfront payment and the cost sharing, we obviously can take that capital, that non-dilutive capital, and really apply our effort and more importantly, our focus on ensuring that we can have the best possible launch here in the US, which obviously we have been obviously gearing up for. I think one of the reasons you'll see that the deal terms were quite competitive and significantly above comps for products that have yet to flip over the Phase 3 card is I think simply because of the competitive nature of this. There was a number of parties that were interested, not surprising. FSHD is a large market in the US, about 30,000 patients. Some estimate about a million patients outside of the US, and there are currently no treatment options. And Fulcrum has what we believe to be probably about a three-year head start before the next closest competitor comes into play. So I think for all of those reasons, we could not be happier with the partner that we've selected. It allows us to get the drug faster to patients that we could do on our own outside of the US, but it also allows us as a small biotech company to focus on what still continues to this day to be the most profitable and lucrative market in the world, that being the US market.
Gregory Renza: Fantastic. Thank you.
Alex Sapir: Thanks, Greg.
Operator: Thank you. Our next question comes from the line of Corinne Johnson with Goldman Sachs. Your line is now open.
Corinne Johnson: Thanks and good morning. You mentioned in that answer to the last question that you plan to use this upfront capital in supporting the launch. I guess what are your top priority action items that you think will be necessary to set up a strong launch out the gate? And then I'd be curious if you could talk to what you view as then if you potentially bringing to the table as a potential partner or as a partner now for ex-US commercialization beyond just kind of the financial terms? Thanks.
Alex Sapir: Sure, sure. No, absolutely. Thanks, Corinne, for asking the question. Yeah, I think one of our key priorities on the commercial side is to bring on board a very, very strong chief commercial officer that has experience of launching rare diseases in the pharmacy benefit portion of the payer space. And we're having a number of conversations with a number of very, very talented chief commercial officers that meet that criteria. So that's obviously going to be our first important hire. And then we'll be doing a couple of additional hires this year, particularly around market access. And we more than likely will end up recruiting somebody who is exclusively focused on ensuring that genetic testing does not become an impediment to access at launch. So I think those are some of our key priorities right now for the US. I think what Sanofi brings, and we talked a little bit about this during our prepared remarks, I think beyond the financials, they have a very, very strong neuromuscular franchise with their programs such as Fabrazyme and Cerezyme, Myozyme. I mean, that's a $3 billion portfolio globally. So they've got a very, very strong commercial footprint. But beyond that, they obviously have very strong regulatory as well as clinical expertise in the neuromuscular space as well. So having that expertise as we're negotiating with the various regulatory agencies around the world, as well as with the various pricing and reimbursement agencies in various countries around the world, I think that that experience that Sanofi brings to the table will be invaluable.
Corinne Johnson: Thank you.
Alex Sapir: Thank you, Corinne.
Operator: Thank you. Our next question comes from the line of Joseph Schwartz with Leerink Partners. Your line is now open.
Joseph Schwartz: Great, congratulations on the deal. I have a question on losmapimod and also one on pociredir. So first on losmapimod, I was just wondering if you can tell us how your work to establish the clinical meaningfulness of certain changes in RWS is going? Can you outline the process of performing that analysis and where you are in that process and when we might see some data on that front?
Alex Sapir: Yeah, that's great. Thanks so much, Joe, and thanks for your kind words earlier. Yeah, I think to answer the question, we're doing a lot of work on clinical meaningfulness. We've been working very closely with the COA division of the FDA, but maybe just get into some of the specifics, let me turn it over to Iain.
Iain Fraser: Yeah, thanks, Alex. Thanks, Joe. So, there are three main steps as part of the work that's ongoing and that we've aligned with the agency. The first is a survey that is now being completed and that is asking patients with FSHD what is most important to them in terms of activities of daily living. So that was a 100 patient survey unrelated to treatment with losmapimod. Again, that is completed. The two other components are ongoing at the moment. The first is a cross-sectional study, again, in patients with FSHD but not being treated with losmapimod and performing reachable workspace assessments on those patients and then matching up the ability of those patients to do the important activities of daily living which were identified in the initial step. And so that's by questionnairing those patients, so mapping reachable workspace there to the questionnaire of their ability to perform those activities. And that assessment, recruiting those patients into that is active and ongoing at the moment and that's going to be a total of about 50 patients. And then the last component, the third component of this is as part of the REACH study, so patients who've been treated with either losmapimod or placebo undergoing structured accident abuse from the study to get insight from them on what difficulties they had with activities of daily living as part of their participation in the study. That work is ongoing and will only complete once the REACH study itself is complete and we have all the data. So we expect all of these strands of data to be pulled together and available in around the same timeframe as we expect the top line data from the REACH study and that those will be around the same time.
Joseph Schwartz: Thanks, that's very helpful. And then can you update us on site activation in the ongoing study for pociredir? And at what point do you think you could share some data from this study, either in terms of calendar timing or how you're thinking in terms of numbers of patients you're aiming for before giving us the first look? That would be very helpful.
Alex Sapir: Yeah, absolutely. Thanks, Joe. I'm happy to take that one, yeah. So, we will be sharing information when we have completed the 10 patients in the 12 milligram cohort and all 10 of those patients have completed the three month study duration. And we intend to share that with everybody in advance of Cohort 4, which is the 20 milligram cohort. Again, our expectation there is that we would share the 20 milligram cohort data once those 10 patients have completed the full three months of dosing. We have been pleased with the site activation efforts to date. I would say that there is a strong interest from many sickle cell centers about the promise of having a once-daily oral medication that has the potential to raise levels of HbF to that seen with the cell and gene therapies. We haven't yet provided any specific guidance on when we would be able to share that information, because what we wanna make sure that we do, Joe, is, I mean, first and foremost, we want to make sure that we don't give a number that we don't feel comfortable with in terms of a particular quarter. So, what we said to folks is that we want to have a critical mass of patients in that 12-milligram cohort before we come back to everyone and let them know in what quarter we'll have the 12 and in a subsequent quarter the 20 milligram. And I think just having that critical mass of patients, it allows us to know a couple of things, right? It allows us to sort of see what that enrollment trajectory looks like, but it also allows us to see which sites have been the strong enrollers and to be able to go back to those PIs at those sites and really ask them point blank how many more patients that they have. So until we have that critical mass of patients or once we have that critical mass of patients and we can more accurately predict what that enrollment trajectory looks like, that'll be the point in time in which we'll come back to everybody with more specificity around which quarter we would share the 12 milligrams followed by the quarter in which we would share the 20 milligrams.
Joseph Schwartz: Very helpful. Thanks and congrats again.
Alex Sapir: Yeah. Thanks, Joe.
Operator: [Operator Instructions] Our next question comes from the line of Dae Gon Ha with Stifel. Your line is now open.
Dae Gon Ha: Hey, good morning guys. Thanks for taking our questions and congrats on the deal and progress as well. I'll start with the losmapimod question. In your prepared remarks, Alex, you talked about 146 of your patients being completed as of April 24, 144 electing to go into open label. So curious in terms of the Sanofi deal, I guess when did this deal kind of get underway and to what extent were they kind of given the access to the data room? I know it's blinded, but just kind of curious how much access they had there. And I've got a follow-up.
Alex Sapir: Sure. Absolutely, Dae Gon. And maybe to -- and thanks for your kind words as well, maybe to answer that question, I'll turn it over to Paul, who's really been the person that's been leading the charge on the -- we've been in conversations obviously for some period of time with the folks at Sanofi, but Paul, do you want to maybe just talk a little bit about kind of when these conversations got quite serious?
Paul Bruno: Yeah, I mean, the conversation has been taking place for over the past year. Again, It's been a competitive process with a number of parties. They have had broad access to all the confidential information related to the program. I will explicitly note that they have not seen any blinded REACH data, neither have we. But other than that, they've had broad access to the program materials.
Dae Gon Ha: Okay, great. Thanks for the details, Paul. Maybe the next question is when you think about the Sanofi infrastructure, especially access to ex-US regulators, I guess, can you just remind us where things stand in terms of the Phase 3 REACH design? How comfortable is the EMA as well as ex-US regulators about that particular endpoint as it pertains to FSHD approvability? Thanks so much guys.
Alex Sapir: Yeah, absolutely, Dae Gon. Thanks for the question. Maybe let me turn that one over to Iain.
Iain Fraser: Yeah, thanks, Dae Gon. So we have engaged with the European regulators. There was some initial engagement prior to the study which was done on a on a country-by-country basis rather than central process with the EMA but more recently we have engaged with the EMA somewhat late in the process, so while the trial was underway. I think they recognized the endpoints that we've established. They also recognized that our interactions with them were after the trial had already been initiated, and so opportunities to impact that were limited. But I think there's an understanding of our primary endpoint and the way that we've structured the secondary endpoints. And I think an overall agreement that that seems to be a reasonable design from that point of view.
Dae Gon Ha: Great. Thanks so much, and congrats again, guys.
Iain Fraser: That's great. Thanks, Dae Gon.
Operator: Thank you. Our next question comes from the line of Matthew Biegler with Oppenheimer. Your line is now open.
Matthew Biegler: Hey, good morning guys. I'll extend our congrats as well. I just wanted to maybe tag along to that last question about the partnering process here. And specifically, if you can comment on how receptive the partners were to the RWS endpoint, is this something that they've seen before or did it kind of require a lot of handholding with Paul and team throughout the due diligence process? Thanks.
Alex Sapir: Yeah, great question, Matt. Maybe, to answer -- probably best person to answer that would be Paul.
Paul Bruno: Yeah, thanks, Matt. No, we have been broadly educating folks on RWS, I don't think to a greater degree than you would for any other novel endpoint. I think it was also one of the major reasons that we were excited about Sanofi as a potential partner between their deep neuromuscular expertise but also their expertise with novel endpoints. I think this really was an ideal match for Fulcrum particularly for this rare disease drug development program.
Matthew Biegler: Thanks.
Alex Sapir: Great. Thanks, Matt.
Operator: Thank you. And I'm currently showing no further questions at this time. I'd like to hand the call back over to Alex for any closing remarks.
Alex Sapir: That's great. Thanks so much, Shannon, and thanks to all of you for joining again. Maybe just in closing, we, as always, remain deeply committed to treating the root cause of genetically defined rare diseases and bringing transformative therapies to patients. And before we conclude today's call, as I always like to do, I would like to extend my sincere appreciation and gratitude to my fellow Fulcrum teammates, to the physicians we work with to advance our clinical studies, and finally and most importantly to the patients and their families. Thanks, everybody who joined this morning, and please stay safe and healthy. Thanks.
Operator: This concludes today's conference call. Thank you for your participation. You may now disconnect.
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