Progress in sarcoma treatments and the companies offering hope to patients worldwide
20 Aug, 20264+ minutes
July is sarcoma and bone cancer awareness month, a period that aims to raise awareness and educate the public on a cancer type few of us have heard of.
Sarcoma is a very rare type of cancer, which means, as well as facing a lot of uncertainty, patients diagnosed with soft-tissue or bone sarcomas often feel alone in their experiences.
For context:
Sarcoma cases account for just 1% of adult cancer cases in the US
20% of all sarcomas fall into the ‘ultra rare’ category
As well as receiving delays in diagnosis, unclear care pathways and clinicians who may have never worked with similar cases, rare cancer patients ultimately lack treatment options.
Below are several sarcoma therapeutics that are currently in development, and the companies that are doing their bit to contribute to a health area so lacking in research and investment.
Storm Therapeutics
Storm Therapeutics is a UK-based biotech who are working in RNA (ribonucleic acid) modification, a novel mechanism of gene regulation. Their lead product, STC-15, is the first RNA-modifying enzyme inhibitor to enter human clinical trials. As of April 2026, the drug has been dosed to its first patient in a sarcoma study.
STC-15 works by stopping a certain kind of RNA-modifying enzyme called METTL3, which in high numbers can contribute to sarcoma progression. By blocking this enzyme, STC-15 can stop a critical process in the development of sarcoma cells, limiting its growth.
The company’s recent $54 million series C funding will support the advancement of their lead product and continue to support their pivotal breakthrough in tackling cancers caused by aberrant cell differentiation (when younger cells don’t mature as they should and end up taking on abnormal traits).
Biosplice Therapeutics
Based in San Diego, Biosplice Therapeutics is clinical-stage biotech developing small-molecule therapies. Although best known for their osteoarthritis program, the company has a clinical-stage asset called cirtuvivint that is currently undergoing phase 2 testing for soft-tissue sarcomas.
The trial began in May 2025 and is taking place across eight sites in Spain with 30 patients. As an oral tablet, the therapy is designed to work by inhibiting CLK1-4 and DYRK1-4 enzymes, preventing the production of proteins that are essential for tumor survival.
Cirtuvivint is particularly significant because, as a second-line treatment, it could provide patients with another effective option if their cancer becomes resistant to their initial therapy (which is common), or if side effects become too severe.
According to one report, if the trial is successful, it could “validate cirtuvivint’s mechanism of action in this specific cancer type, opening avenues for future research into its application in other sarcoma subtypes or related malignancies.”
Advenchen Laboratories
Advenchen Laboratories is a small, late-stage pharmaceutical company focused on small molecule cancer drug discovery programs, based in California.
Their most prominent lead product, AL3818 (catequentinib hydrochloride), is undergoing phase 3 testing for alveolar soft part sarcoma (ASPS), an ultra-rare cancer that primarily affects adolescents and young adults.
AL3818 is a receptor tyrosine kinase (RTK) inhibitor that is designed to work by blocking specific cell signals that allow tumor cells to grow and build new blood vessels. It has been dosed to 56 subjects with metastatic or advanced ASPS, with primary completion estimated at the end of 2027.
The therapy received orphan designation from the European Medicines Agency (EMA) for the treatment of soft tissue sarcomas in 2018, which is essentially a special status given to drugs that target very rare, life-threatening conditions – simplifying their route to approval.
If the study results in positive data, there is hope that AL3818 could become a new line of therapy offering improved progression-free survival for sarcoma patients.
Eli Lilly
Pharma giant, Eli Lilly, has developed an oral targeted therapy called abemaciclib (brand name Verzenio), which has been approved for the treatment of HR-positive or HER2-negative advanced breast cancer.
However, the company is currently trialing the drug in a Phase 3 trial for dedifferentiated liposarcoma, with study completion estimated at the end of this year. Liposarcoma is a rare type of soft-tissue sarcoma that forms in the body’s fatty tissue and has very limited treatment options.
The randomized double-blind study that began in 2021 has been analyzing how abemaciclib performs compared to a placebo. According to an exploratory analysis, earlier use provides the greatest benefit to patients as:
50% of patients who received the treatment as the first-line therapy had a median PFS (progression-free survival) of 16.4 months
The other 50%, who received it in a second (or later-line) setting, had a median PFS of 5.3 months
If successful, the trial could showcase the first targeted agent to improve patient outcomes in recurrent or metastatic dedifferentiated liposarcoma.
Conclusion
While these sarcoma therapies are still very much in the development stage, they reflect a growing investment in an area of oncology that has historically received limited attention.
These Phase 2/3 studies highlight our progress towards treatment expansion, so that patients with rare and ultra-rare sarcomas can have better available treatment options and, therefore, improved quality of life in the years to come.
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