Fulcrum Therapeutics, based in Cambridge, Massachusetts, closed on an $80 million Series B financing.
Fulcrum Therapeutics, based in Cambridge, Massachusetts, closed on an $80 million Series B financing. The round was led by Foresite Capital. Fidelity, 6 Dimensions Capital, Casdin Capital, Sanofi Ventures, Section 32, NS Investments, and entities affiliated with Leerink Partners and undisclosed institutional investors also participated.
The company’s president and chief executive officer, Robert Gould, indicates that the company will likely launch an initial public offering (IPO) in 2019. In the meantime, it plans to use the funds raised to begin clinical trials for its lead program in facioscapulohumeral muscular dystrophy (FSHD) and its pipeline of treatments for rare, genetically-based neuromuscular, central nervous system and hematologic disorders.
FSHD is a genetic muscle disease where the muscles of the face, shoulder blades and upper arms are the most affected. Symptoms usually start before the age of 20 with weakness and atrophy of the muscles around the eyes, mouth, shoulders, upper arms and lower legs. As the disease progresses, it can spread to abdominal muscles and hip muscles. It is typically associated with a mutation in the DNA on chromosome 4.
The FSH Society estimates 870,000 people are affected by the disease worldwide. FSHD typically has a slow progression and isn’t fatal, but it does often leave patients in wheelchairs or on respirators. Fulcrum’s lead compound attempts to prevent the expression of the DUX4 protein, which is linked to the disease.
“We are delighted to have the support of this exceptional group of investors as we continue to work towards our vision of bringing new futures to patients and families affected by debilitating genetic diseases,” Gould said in a statement. “This funding will provide crucial support as we move rapidly towards the clinic with our lead drug candidate in FSHD and further advance our pipeline of small molecule therapies.”
Gould was at one time an executive at Merck, and previously took oncology company Epizyme public in 2013, raising approximately $80 million at $15 per share. He left Epizyme in 2015 and became chief executive of Fulcrum in 2016.
Fulcrum’s focus is on developing drugs that affect the molecular switches that turn genes on or off.
In addition to the financing, Jim Tananbaum of Foresite Capital will join Fulcrum’s board of directors. “Fulcrum is leading the way with their small molecule, precision medicine approach to treating severe genetic diseases,” Tananbaum said in a statement. “We believe the convergence of biology and data science will be the key to unlocking novel treatments for patients and families that have run out of options. We look forward to supporting the Fulcrum team as it works to advance its rare disease-focused pipeline.”
Ben Fidler, writing for Xconomy, says, ‘Its scientific aspirations aside, however, Fulcrum is a product, of sorts, of the FDA’s increasing willingness to incorporate patient perspectives throughout the drug development process. Fulcrum reached out to patient groups to figure out which diseases to go after, even before the company was officially formed in 2016. That led it to FSHD. That approach is atypical; more often, drug developers connect with patient groups later on, Michael Tranfaglia, medical director, chief scientific officer, and co-founder of the FRAXA Research Foundation, told Xconomy in 2017.”
Fulcrum launched in 2016 with a $55 million Series A from Third Rock Ventures. Other pipeline compounds focus on sickle cell disease, Duchenne muscular dystrophy (DMD) and Fragile X syndrome.
