Clinical-stage biopharmaceutical company Fulcrum Therapeutics Inc. (FULC:NASDAQ), which is focused on developing medicines to treat patients with genetically defined rare diseases, today announced operating and financial results for its second quarter of 2021 ended June 30, 2021.
Fulcrum Therapeutics President and CEO Bryan Stuart commented, "This quarter was marked by meaningful progress, particularly as we furthered our efforts with our lead candidates for FSHD and sickle cell disease…We made groundbreaking progress with losmapimod and reported clinical data from ReDUX4 demonstrating slowed disease progression and improved function in FSHD. We are also pleased with the dose proportional target engagement data, fetal hemoglobin (HBG) mRNA induction and F-reticulocyte increases that we observed in our ongoing Phase 1 trial in healthy adult volunteers with FTX-6058, demonstrating proof of mechanism and proof of biology. These results, along with the safety, tolerability and pharmacokinetics observed to date, reinforce our belief in FTX-6058's potential to be a best-in-class treatment for select hemoglobinopathies."'
"Our team continues to optimize the potential of FulcrumSeek™, our proprietary product engine. Driven by our focus on the patient and our ability to rapidly identify novel, high quality targets that modulate the root cause of genetically defined rare diseases, we are on track to submit two investigational new drug applications (INDs) by the end of the first quarter of 2023. With these important advances and cash runway that takes us into the first quarter of 2023, we believe we are well positioned to continue to build on our momentum to bring important therapies to patients with genetically defined rare diseases," Stuart added.
The company discussed several recent business developments and noted that today it reported highly positive interim results from its current single- and multiple-ascending dose (SAD and MAD) Phase 1 trial that is evaluating FTX-6058 in healthy adult volunteers.
The company described FTX-6058 as an "investigational, potent and selective small molecule inhibitor of EED designed to induce expression of fetal hemoglobin (HbF) with the potential to treat hemoglobinopathies, such as sickle cell disease and beta-thalassemia."
The firm said that FTX-6058 was well tolerated in the study and that in Q4/21 it plans to commence enrollment in a Phase 1b clinical trial in sickle cell patients. Fulcrum added that it intends to a submit an IND to the U.S Food & Drug Administration (FDA) by the end of 2021 for us in non-sickle cell disease hemoglobinopathies.
The company also commented on results from its Phase 2b ReDUX4, trial of losmapimod. The firm described losmapimod as a "selective p38α/β mitogen activated protein kinase (MAPK) inhibitor, demonstrated slowed disease progression and improved function in facioscapulohumeral muscular dystrophy (FSHD)." Fulcrum advised that though the primary endpoint was not met in the study, losmapimod was generally well tolerated and stated the company plans to meet with FDA and other key health authorities in H2/21 to advance losmapimod for approval in FSHD.
Fulcrum reported that in Q2/21 collaboration revenue was $4.4 million, compared to $2.0 million in Q2/20. The company advised the revenue increase was primarily due to its partnerships and license agreements with MyoKardia and Acceleron.
The firm stated that as of June 30, 2021, it held $125.6 million in cash and liquid assets on its balance sheet, versus the $112.9 million it held as of December 31, 2020. The company stated that is operations and fully funded into Q1/23.
The company indicated that it incurred research and development (R&D) expenses of $17.4 million in Q2/21, compared to $12.8 million in Q2/20 and that the $4.6 million increase was associated mostly with supporting its ongoing and scheduled clinical trials.
Fulcrum advised that it posted a net loss of $19.6 million in Q2/21, compared to a net loss of $15.7 million in Q2/20.
Fulcrum mentioned that "FSHD is characterized by progressive skeletal muscle loss that initially causes weakness in muscles in the face, shoulders, arms and trunk, and progresses to weakness throughout the lower body." Ultimately, most patients end up needing to use a wheelchair for daily mobility due to progressive skeletal muscle deterioration. The disease is one of the more common types of muscular dystrophy and it is thought to effect between 16,000 to 38,000 people in the U.S.
The firm advised that "sickle cell disease (SCD) is a genetic disorder of the red blood cells caused by a mutation in the HBB gene." The company stated that individuals afflicted with SCD usually experience a variety of severe symptoms such as anemia, heart disease, infections, kidney failure, liver disease, pain, pulmonary hypertension, and stroke which greatly reduces life expectancy.
In a separate news release today, Fulcrum Therapeutics announced "positive interim results from the ongoing single- and multiple-ascending dose (SAD and MAD) Phase 1 trial with FTX-6058 in healthy adult volunteers."
The company described FTX-6058 as "investigational, potent, and selective small molecule inhibitor of EED designed to increase the expression of fetal hemoglobin (HbF) with the potential to treat hemoglobinopathies, such as sickle cell disease and beta-thalassemia."
Fulcrum noted that the results obtained from the MAD portion of the study demonstrated "a dose proportional induction in HBG mRNA and accompanying increases in HbF-containing reticulocytes (F-reticulocytes) and added that at 10mg, the highest dose studied to date, the mean changes were 4.5-fold and 4.2-fold, respectively."
CEO Stuart remarked, "We demonstrated consistent 2-3-fold induction of HBG mRNA and HbF protein both in vitro and in vivo…These clinical results reported today not only underscore the consistency observed preclinically, but also demonstrate the first evidence that FTX-6058 can achieve or exceed these preclinical thresholds predicted to provide meaningful clinical benefits to individuals with SCD."
Fulcrum Therapeutics is a clinical-stage biopharmaceutical company based in Cambridge, Mass. The firm focuses its efforts on developing treatments for patients with genetically defined rare diseases. The company's stated that "its proprietary product engine, FulcrumSeek, identifies drug targets which can modulate gene expression to treat the known root cause of gene mis-expression." At present, the company is conducting a Phase 2 study of losmapimod for treating facioscapulohumeral muscular dystrophy and is advancing FTX-6058 in a Phase 1 trial to address sickle cell disease and beta-thalassemia.
Fulcrum Therapeutics started off the day with a market cap of around $272.2 million with approximately 32.68 million shares outstanding and a short interest of about 2.3 %. FULC shares opened almost 60% higher today at $13.21 (+$4.88, +58.58%) over yesterday's $8.33 closing price and then rose to a new 52-week high of $22.09. The stock has traded today between $12.07 and $22.09 per share and closed at $18.77 (+$10.44, +125.33%).
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