Fulcrum Therapeutics (FULC) — Stock, Pipeline & Market Cap

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FULC · Stock Price

USD 6.88+1.51 (+28.12%)

Market Cap: $461.1M

Historical price data

Market Cap: $461.1MPipeline: 10 drugs (2 Phase 3)Patents: 9Founded: 2015Employees: 100-250HQ: Cambridge, United States

Sector Ranking#214 in Small Molecule Drugs #54 in Rare & Orphan Diseases

Overview

Fulcrum Therapeutics is a clinical-stage biotech company with a mission to develop small molecule therapies that treat the root cause of genetically defined rare diseases by modulating gene expression. Its core achievement is the development of a proprietary functional genomics platform that identifies master regulator proteins to create targeted treatments. The company's strategy focuses on high-unmet-need disorders with clear genetic drivers, such as FSHD and sickle cell disease, leveraging regulatory pathways for rare diseases. Fulcrum's lead asset, losmapimod for FSHD, has completed Phase 3 trials, positioning the company for a potential near-term regulatory filing and commercialization.

Rare Genetic DiseasesNeuromuscularHematology

Technology Platform

A proprietary functional genomics platform that identifies and validates master regulator proteins to discover small molecule therapies designed to modulate the expression of disease-causing genes.

Pipeline

10

10 drugs in pipeline2 in Phase 3

Drug	Indication	Stage
Losmapimod oral tablet + Placebo oral tablet	COVID-19	Phase 3
Losmapimod + Placebo oral tablet	Facioscapulohumeral Muscular Dystrophy (FSHD)	Phase 3
Pociredir	Sickle Cell Disease	Phase 2
Losmapimod oral tablet + Placebo oral tablet	Facioscapulohumeral Muscular Dystrophy (FSHD)	Phase 2
Losmapimod	Facioscapulohumeral Muscular Dystrophy 1	Phase 2

Funding History

3

Total raised:$216M

IPO$96MUndisclosedJul 15, 2019

Series B$80MThird Rock VenturesJan 15, 2019

Series A$40MThird Rock VenturesJun 15, 2017

Opportunities

Fulcrum's lead hematology program, pociredir, targets the multi-billion dollar sickle cell disease market with a convenient oral therapy, entering a landscape ripe for effective small molecules.

Success here, or a viable regulatory path for losmapimod in FSHD, could drive significant value creation and potential partnership or acquisition interest.

Risk Factors

The company faces high clinical risk following the failure of losmapimod to meet its primary Phase 3 endpoint, with its future now heavily dependent on unproven earlier-stage assets.

It also faces intense competition in sickle cell disease and relies on capital markets for funding, with potential for dilution.

Competitive Landscape

In FSHD, Fulcrum competes with Dyne and Avidity, who are pursuing biologic modalities. In the crowded sickle cell arena, pociredir must differentiate itself from approved HbF inducers (e.g., Oxbryta) and curative but complex gene therapies from Vertex/CRISPR and bluebird bio.