FDA Grants Rare Pediatric Disease Designation to Elraglusib for Ewing Sarcoma

The FDA granted rare pediatric disease designation to elraglusib (Actuate Therapeutics) for the treatment of Ewing sarcoma (EWS). Elraglusib is a novel glycogen synthase kinase-3 beta inhibitor.¹

“Early clinical data from our ongoing phase 1/2 trial show promising anti-tumor activity with objective tumor responses, including 2 ongoing durable complete responses in the first 6 patients treated with relapsed/refractory EWS, reinforcing our confidence in elraglusib’s potential impact in this challenging disease setting,” Daniel Schmitt, president and CEO of Actuate, said in a news release.¹

Bone Cancer, Ewing Sarcoma, Oncology, Pediatric Patients | Image Credit: © Richman Photo | stock.adobe.com

According to the Mayo Clinic, EWS is a cancerous growth that begins in the bone and soft tissue around the bone and is most frequently present in children and young adults. Symptoms can include a lump in the affected area, bone pain, bone fracture, fever, weight loss, and tiredness. There have been advancements in treatment, but patients may be impacted by latent effects from strong treatment.²

The ongoing trial, Actuate-1902 (NCT04239092), is open-label and multicenter to evaluate the safety and efficacy of the drug in pediatric patients who have relapsed/refractory malignancies. Investigators reported that 8 patients with relapsed/refractory EWS have been treated with the study drug combined with topotecan/cyclophosphamide.¹

The study has a cohort of more than 90 patients with advanced refractory malignancies. The phase 1 study evaluated the safety and efficacy of the drug in pediatric patients and phase 2 established the dose for further pediatric patient studies. The primary outcome of the study was the number of individuals with treatment-related adverse events from 3 to 12 months, according to the clinical trial information.³

Patients included were under the age of 22 years, had either measurable or evaluable disease, had a disease state for which there is no known curative therapy or therapy proven to prolong survival with an acceptable quality of life, had recovered from the acute clinically significant toxic effects of prior anti-cancer therapy, received at least 1 first line treatment regimen for the malignancy, and had adequate organ and marrow function on the first day of study treatment.³

In August 2023, the FDA granted orphan drug designation to elraglusib for the treatment of individuals with pancreatic cancer. The drug was evaluated as a monotherapy and in combination with cytotoxic agents for patients with refractory cancers, with 1 arm focusing on elraglusib plus gemcitabine (Gemzar; Eli Lilly) and nab-paclitaxel (Abraxane; Bristol Myers Squibb) for patients with advanced pancreatic cancer.⁴

In September 2024, the FDA granted orphan drug designation to elraglusib for the treatment of patients with soft tissue sarcoma based on a phase 2, open-label trial that evaluated the drug with gemcitabine and docetaxel in patients aged 10 years and older with unresectable or metastatic soft tissue or bone sarcomas.⁵

REFERENCES

1. Actuate Announces FDA Rare Pediatric Disease Designation Granted to Elraglusib for Treatment of Ewing Sarcoma. News release. Actuate Therapeutics. November 12, 2024. Accessed November 12, 2024. https://www.globenewswire.com/news-release/2024/11/12/2979076/0/en/Actuate-Announces-FDA-Rare-Pediatric-Disease-Designation-Granted-to-Elraglusib-for-Treatment-of-Ewing-Sarcoma.html

2. Mayo Clinic. Ewing sarcoma. December 29, 2023. Accessed November 12, 2024. https://www.mayoclinic.org/diseases-conditions/ewing-sarcoma/symptoms-causes/syc-20351071

3. 9-ING-41 in Pediatric Patients with Refractory Malignancies. ClinicalTrials.gov identification: NCT04239092. Updated November 8, 2024. Accessed November 12, 2024. https://clinicaltrials.gov/study/NCT04239092

4. Rosa K. FDA Grants Orphan Drug Designation to Elraglusib for Pancreatic Cancer. OncLive. August 1, 2023. Accessed November 12, 2024. https://www.onclive.com/view/fda-grants-orphan-drug-designation-to-elraglusib-for-pancreatic-cancer

5. Sava J. FDA Grants Elraglusib Orphan Drug Designation in Soft Tissue Sarcoma. Targeted Oncology. September 12, 2024. Accessed November 12, 2024. https://www.targetedonc.com/view/fda-grants-elraglusib-orphan-drug-designation-in-soft-tissue-sarcoma