Orphan drug designation is granted in the United States to expedite the development and review of drugs and biologics intended to treat patients with rare conditions (over 200,000 Americans). Once granted, drug developers also earn a variety of benefits, from tax credits and fee waivers to increased regulatory assistance and 7 years market exclusivity upon approval. According to Targeted Oncology, sotigalimab (APX005M), a potential treatment option for patients with soft tissue sarcoma (STS), recently earned Orphan Drug status at the beginning of August 2021.
Sotigalimab
So what exactly is sotigalimab? According to drug developer and biopharmaceutical company Apexigen, sotigalimab is:
a potent CD40 agonist [and humanized monoclonal antibody] that has been designed to reverse the systemic immune suppression that typically affects cancer patients, through the activation of several mechanisms of immune functionality. CD40 is a co-stimulatory receptor that is essential for activating both innate and adaptive immune systems.
Altogether, the treatment works to stimulate T cell activation and promote an anti-tumor microenvironment. As few treatment options exist for patients with STS, sotigalimab offers the potential to significantly benefit patients.
In the past, researchers evaluated sotigalimab in conjunction with chemotherapy for patients with metastatic pancreatic adenocarcinoma. During the study, researchers found that 58% of patients responded to this form of treatment, suggesting it could be better suited for patients than the current standard-of-care.
Now, researchers are looking to explore sotigalimab as a treatment for patients with STS. Apexigen will hold a Phase 2 clinical trial to determine the safety, efficacy, and tolerability of sotigalimab in conjunction with doxorubicin. 27 patients will enroll. During the trial, researchers seek to understand the objective response rate, recommended dosage, progression-free survival (PFS), adverse reactions, and CD40 expression.
Soft Tissue Sarcoma (STS)
Soft tissue sarcoma (STS) is a rare cancer that forms in soft tissues which provides support to other bodily structures. For example, STS may form in muscle, tendons, fat, lymph, or even blood vessels. Altogether, there are 50+ STS subtypes. Doctors are not sure what exactly causes STS. However, risk factors include chemical or radiation exposure or having a condition such as Li-Fraumeni syndrome, tuberous sclerosis, or retinoblastoma. In early stages of the cancer, patients may not notice any symptoms. However, once symptoms appear, they include:
- A noticeable lump or swollen area
- Pain (if pressing on nerves/muscles)
- Cramping (if the tumor is in the abdomen)
- Constipation (if the tumor is in the abdomen)
