$FULC Breakout alert setup in Fulcrum Therapeutics
Entry level $15.96 = Target price $20.63
Alert set for breakout above $15.61
Average analysts price target $12 | Buy rating
Earnings call transcript
We believe our approach to the treatment of genetically defined diseases using a small molecule approach may offer significant advantages over other treatment modalities since small molecules can achieve broad biodistribution throughout the body, have an increased likelihood of tolerability, have well-defined manufacturing and quality control requirements, and can offer ease and patient access.
Fulcrum’s product engine has enabled the progress of the lead programs in our pipeline. Our most advanced candidate is losmapimod, a selective p38 alpha/beta mitogen-activated protein kinase, MAPK inhibitor. Losmapimod is being evaluated in ongoing Phase 2 trials for the treatment of FSHD. And we began enrolling in these trials in August. FSHD is a serious and progressive disease characterized by muscular degeneration in which skeletal muscle is replaced by fat. There are no currently approved treatments for FSHD and no other known industry sponsored programs in clinical development. Patients with FSHD experience significant physical impairment and disability over time, which can severely impact their day-to-day function and quality of life. The root cause of FSHD is the aberrant expression of the DUX4 gene. Using our product engine, we made the novel discovery that P38 inhibitors including losmapimod, reduce the aberrant expression of the DUX4 gene.
In February of this year, we obtained the exclusive worldwide license to losmapimod from GlaxoSmithKline, which allows Fulcrum to utilize the significant safety database that GSK generated with losmapimod during their previous studies in other indications.
In October, we presented Phase 1 data from the losmapimod trial in FSHD patients at the International Annual Congress of the World Muscle Society. Losmapimod was well tolerated in FSHD patients with no serious adverse events reported with doses up to 15 mg taken orally, twice daily for 14 days.
Importantly, we also showed that losmapimod achieved dose dependent concentrations in muscle and that are twice daily 15 mg dose resulted in sustained and robust target inhibition in blood. These levels of drug and targeted inhibition have been shown in preclinical studies to reduce DUX4 as well as DUX4 driven gene expression and restore muscle function. The data from the Phase 1 study supports the selection of 15 mg BID of losmapimod as the appropriate dose for our ongoing Phase 2 clinical trials.
ReDUX4 is a Phase 2, randomized, double-blind, placebo controlled study to evaluate the efficacy and safety of losmapimod in FSHD patients over 24 weeks. We are in the process of enrolling 66 patients randomized one-to-one in the trial with a plan to open label extension to follow. The primary endpoint of the ReDUX4 study is DUX4 activity in effected skeletal muscle at 16 weeks as measured by quantitative RT-PCR in a panel of DUX4 regulated gene transcripts. Secondary endpoints include the continued evaluation of safety and tolerability, PK and blood, losmapimod concentration in skeletal muscle biopsies and targeted engagement in blood and in skeletal muscle. This study encompasses up to 20 sites across five countries including the U.S., Canada and three countries in Europe.
COMPANY PROFILE
Fulcrum Therapeutics, Inc. is a clinical-stage biopharmaceutical company. It develops new medicines and focuses on unlocking gene control mechanisms to develop small molecule therapies. Its product candidate includes Losmapimod and FTX-HbF. The company was founded by Michael R. Green, Danny Reinberg, Rudolf Jaenisch, Jeannie T. Lee, and Bradley E. Bernstein on August 18, 2015 and is headquartered in Cambridge, MA.
Alert set for breakout above $15.61
Average analysts price target $12 | Buy rating
Earnings call transcript
We believe our approach to the treatment of genetically defined diseases using a small molecule approach may offer significant advantages over other treatment modalities since small molecules can achieve broad biodistribution throughout the body, have an increased likelihood of tolerability, have well-defined manufacturing and quality control requirements, and can offer ease and patient access.
Fulcrum’s product engine has enabled the progress of the lead programs in our pipeline. Our most advanced candidate is losmapimod, a selective p38 alpha/beta mitogen-activated protein kinase, MAPK inhibitor. Losmapimod is being evaluated in ongoing Phase 2 trials for the treatment of FSHD. And we began enrolling in these trials in August. FSHD is a serious and progressive disease characterized by muscular degeneration in which skeletal muscle is replaced by fat. There are no currently approved treatments for FSHD and no other known industry sponsored programs in clinical development. Patients with FSHD experience significant physical impairment and disability over time, which can severely impact their day-to-day function and quality of life. The root cause of FSHD is the aberrant expression of the DUX4 gene. Using our product engine, we made the novel discovery that P38 inhibitors including losmapimod, reduce the aberrant expression of the DUX4 gene.
In February of this year, we obtained the exclusive worldwide license to losmapimod from GlaxoSmithKline, which allows Fulcrum to utilize the significant safety database that GSK generated with losmapimod during their previous studies in other indications.
In October, we presented Phase 1 data from the losmapimod trial in FSHD patients at the International Annual Congress of the World Muscle Society. Losmapimod was well tolerated in FSHD patients with no serious adverse events reported with doses up to 15 mg taken orally, twice daily for 14 days.
Importantly, we also showed that losmapimod achieved dose dependent concentrations in muscle and that are twice daily 15 mg dose resulted in sustained and robust target inhibition in blood. These levels of drug and targeted inhibition have been shown in preclinical studies to reduce DUX4 as well as DUX4 driven gene expression and restore muscle function. The data from the Phase 1 study supports the selection of 15 mg BID of losmapimod as the appropriate dose for our ongoing Phase 2 clinical trials.
ReDUX4 is a Phase 2, randomized, double-blind, placebo controlled study to evaluate the efficacy and safety of losmapimod in FSHD patients over 24 weeks. We are in the process of enrolling 66 patients randomized one-to-one in the trial with a plan to open label extension to follow. The primary endpoint of the ReDUX4 study is DUX4 activity in effected skeletal muscle at 16 weeks as measured by quantitative RT-PCR in a panel of DUX4 regulated gene transcripts. Secondary endpoints include the continued evaluation of safety and tolerability, PK and blood, losmapimod concentration in skeletal muscle biopsies and targeted engagement in blood and in skeletal muscle. This study encompasses up to 20 sites across five countries including the U.S., Canada and three countries in Europe.
COMPANY PROFILE
Fulcrum Therapeutics, Inc. is a clinical-stage biopharmaceutical company. It develops new medicines and focuses on unlocking gene control mechanisms to develop small molecule therapies. Its product candidate includes Losmapimod and FTX-HbF. The company was founded by Michael R. Green, Danny Reinberg, Rudolf Jaenisch, Jeannie T. Lee, and Bradley E. Bernstein on August 18, 2015 and is headquartered in Cambridge, MA.