Fulcrum Therapeutics, Inc.

Good morning, and welcome to Fulcrum Therapeutics' third quarter 2020 conference call. Currently, all participants are in the listen-only mode. There will be a question-answer session at the end of this call. I would now like to turn the call over to Christy Warrick, Director of Investor Relations and Corporate Communications at Fulcrum. Please proceed.

Thank you, Sonia. Good morning, and thank you for joining us. Earlier this morning, we issued a press release outlining our recent progress and financial results for the third quarter of 2020. If you don't have a copy of the release, you can find it in the investor relations section of our website at fulcrumtx.com. 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 our future expectations and plans, clinical development timelines, and financial projections. While these forward-looking statements represent our views as of today, they should not be relied upon as representing our views in the future. We may update these statements in the future, but we are not taking on an obligation to do so. Please refer to our most recent filings with the Securities and Exchange Commission for discussion of certain risks and uncertainties associated with our business. With me on today's call are Robert Gould, President and Chief Executive Officer, Owen Wallace, Chief Scientific Officer, and Brian Stewart, Chief Operating Officer. Let me quickly run through this morning's agenda. Robert will begin the call with an overview of our recent progress. Owen will provide an update on our sickle cell program and product engine, and Brian will cover our financials. With that, it's my pleasure to turn the call over to Robert.

Robert? Thanks, Christy, and good morning, everyone. Thank you for joining us today. This quarter, we've seen tremendous progress throughout the company as we strive to improve the lives of patients which genetically define rare diseases in areas of high unmet medical need. In research and development, we made important progress with our program evaluating losmaphimod, a selective P38 MAP kinase inhibitor, and fascioscapulohumeral muscular dystrophy, or FSHD. We're now in clinical development with FTX6058 and screening healthy volunteers in our Phase I sickle cell trial. We initiated LOSFIT, our phase three trial with LOSMAPIMOD for the treatment of COVID-19. Our discovery product engine continues to grow. In July, we executed a collaboration with Myocardia to identify therapeutics that address the genetic drivers of cardiomyopathies. Our progress in FSHD and sickle cell disease has been highlighted in multiple posters accepted at various scientific meetings, and we made key appointments to our management teams. Before turning the call over to Ellen to discuss the progress in our program to elevate fetal hemoglobin for the potential treatment of sickle cell disease and beta thalassemia, I'd like to review our program with Asmaqamod for the treatment of FSHD. FSHD is a rare, progressive, and disabling disease characterized by severe muscular degeneration that occurs as skeletal muscle is replaced by fat. Unlike other diseases that can be characterized by the lack of a gene, FSHD is characterized by the aberrant expression of the gene, Dux4, the root cause of the disease. Dux4 expression results in apoptosis, muscle death, and deterioration. This is the second most common muscular dystrophy with no current treatment options. LasMapMod has previously demonstrated safety and tolerability in approximately 3,500 subjects across multiple indications, and is the only potential treatment in clinical development. This quarter, we presented multiple posters at the International Congress of the World Muscle Society. These posters highlighted our integrated approach to evaluating FSHD patients. We have also made tremendous progress with our clinical strategy, utilizing molecular biomarkers, imaging, functional readouts, and patient-reported outcomes. These studies have enabled the design of clinical trials to evaluate potential benefits of losmaphimod in FSHD patients. The encouraging data reported from our interim analysis suggests losmaphimod may be reducing Dux4-driven gene expression and that muscles with the highest Dux4-driven gene expression in pretreatment biopsies show large reductions following treatment with losmaphimod compared to placebos. We remain on track to announce full data in the second quarter of 2021. Data will include the primary endpoint, reduction from baseline of Dux4-driven gene expression, in all patients. Additionally, we'll report a pre-specified sensitivity analysis, assessing biopsies with the highest pretreatment level of Dux4-driven gene expression, and key secondary and exploratory endpoints. We believe we'll be able to provide the most comprehensive assessment of any therapeutic in FSHD with a totality of data in the second quarter next year, and we will not report top line results separately. We also look forward to the continued analysis of the ongoing single center open label study, which includes change from baseline in skeletal muscle, MRI biomarker, and several clinical outcome assessments in subjects who have been treated up to a year. For FTX6058, we recently presented comparative pharmacology data and additional preclinical data supporting the future development in sickle cell disease and beta thalassemia. FTX6058 is a highly potent small molecule inhibitor of EED designed to induce the expression of fetal hemoglobin to treat the root cause of sickle cell disease. Owen will discuss this potentially promising and differentiated therapeutic option for patients in more detail. And we'll share more on December the 15th when we host a key opinion leader symposium with sickle cell experts. Turning now to our COVID program. As a reminder, LOSSVID is an international multi-center phase three trial with LossMapMod for hospitalized subjects with COVID-19. The trial is designed to assess the safety and efficacy of a 15 milligram twice per day oral dose of losmacmon compared to placebo for 14 days on top of standard of care in approximately 400 subjects who are at risk of progression to critical illness based on older age and elevated systemic inflammation. The primary endpoint is the proportion of patients who progress to death or respiratory failure by day 28. While we saw some delays in opening trial sites in August and September, we continue to make progress in patient enrollment and anticipate providing an update on the LossFit trial in the first quarter of 2021. As part of our progress, we've also expanded our management team. I'm pleased to announce today that Kurt Oldmans will join Fulcrum at the end of the month as our general counsel. Kurt brings a wealth of experience from his prior roles at DaVita, Array, Novo Nordisk, and Eli Lilly, covering a variety of legal roles, including regulatory and intellectual property. We've also appointed Dr. Alan Ziekiewicz to serve as a clinical advisor following the departure of Diego Kadavid, as announced in our release. Alan has served on our board since 2016 and has made important contributions to advancing our pipeline of clinical programs. Previously, Alan was founder and CEO of Abide Therapeutics, and prior to Abide, He spent a number of years at Merck where he served as the Senior Vice President in clinical research. We'll be launching a search for a Chief Medical Officer, but in the meantime, look forward to benefiting from Alan's expertise as part of our executive team. I'm also pleased to announce that Kim Hazen has been promoted to Senior Vice President, Human Resources. Kim has been an invaluable member of the leadership team since she joined Fulcrum in 2017. Lastly, I'd like to thank everyone at Fulcrum who continues to be phenomenal through these challenging times. We've made a tremendous amount of progress, all while keeping our patient communities at the forefront of what we do. With that, I'll turn it over to Owen.