FDA Grants Orphan Drug Status to Sotigalimab for Patients With Soft Tissue Sarcoma


An orphan drug designation has been granted to the CD40 agonistic monoclonal antibody, sotigalimab, for the treatment of patients with soft tissue sarcoma.

An orphan drug designation has been granted to the CD40 agonistic monoclonal antibody, sotigalimab (APX005M), for the treatment of patients with soft tissue sarcoma, according to a press release issued by Apexigen, Inc.1

“We are very pleased to receive orphan drug designation for sotigalimab, an important step toward addressing the medical need that exists for patients with sarcoma,” said Xiaodong Yang, MD, PhD, president and chief executive officer, Apexigen, in the press release.

A phase 2 clinical trial is underway to investigate the safety and efficacy of sotigalimab given in combination with the current standard-of-care therapy, doxorubicin, to patients with advanced soft tissue sarcoma. Investigators of this study hypothesize that adding immunotherapy will improve outcomes for these patients.

Approximately 27 patients will be enrolled in the study and evaluated for the primary end point of objective response rate calculated by the percentage of patients with partial or complete responses to therapy, according to imaging results. The secondary end points include the recommended dose for sotigalimab and doxorubicin, the adverse events associated with the combination, and progression-free survival. The exploratory end points being evaluated in the trial include changes in immune cells infiltrated in baseline and on-study biopsies, and expression of CD40 in baseline and on-study biopsies.

Patients are actively being recruited for the study at Columbia University Medical Center in New York, where Matthew Ingham, MD, is the study lead. Patients are considered eligible for the study given they have histologically confirmed advanced soft tissue sarcoma, locally advanced and unresectable or metastatic disease, measurable disease per RECIST v1.1, and ECOG performance status of 0 or 1, and adequate laboratory and echocardiogram tests at the time of baseline screening.

The treatment strategy of sotigalimab in combination with chemotherapy has been explored previously in patients with metastatic pancreatic adenocarcinoma in a phase 1b study.Thirty patients in the study were dosed with sotigalimab plus gemcitabine and nab-paclitaxel with or without nivolumab (Opdivo). Clinical activity was signaled in the study with 58% of patients having a response to the combination. Investigators concluded from the phase 1b findings that sotigalimab plus chemotherapy could replace standard of care chemotherapy in this patient population.2 Investigators of the sarcoma study are looking for a similar result.2

“There are currently limited treatment options available for patients with soft tissue sarcoma, so we are excited by the emerging data from our ongoing phase 2 study in this indication, coupled with data across our broad clinical development program, that suggest sotigalimab may provide a superior clinical benefit. We will continue to advance our development plans to overcome outstanding challenges in oncology and work with the FDA to bring sotigalimab to patients as efficiently as possible, “Yang added, in the release.


1. Apexigen receives orphan drug designation from the FDA for sotigalimab (APX005M) for the treatment of soft tissue sarcoma. News release. Apexigen, Inc. August 5, 2021. Accessed August 5, 2021. https://www.apexigen.com/news/08-05-21/

2. O’Hara MH, O’Reilly EM, Varadhachary G, et al. CD40 agonistic monoclonal antibody APX005M (sotigalimab) and chemotherapy, with or without nivolumab, for the treatment of metastatic pancreatic adenocarcinoma: an open-label, multicentre, phase 1b study. Lancet Oncol. 2021;22(1):118-131. doi: 10.1016/S1470-2045(20)30532-5

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