ELA026 Receives Orphan Drug Designation to Treat Hemophagocytic Lymphohistiocytosis

The FDA granted orphan drug designation for ELA026 (Electra Therapeutics), a first in class antibody therapy targeting signal regulatory proteins (SIRP), for the treatment of hemophagocytic lymphohistiocytosis (HLH).¹

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HLH is a rare disease that causes the immune system to attack the body instead of bacteria or a virus, which commonly occurs in infants and young children, but can also impact adults. HLC is a generic condition that cannot be prevented and if left untreated can cause life-threatening outcomes.²

The condition is presented in 2 types—primary HCL, which occurs through genetic mutation and secondary HLH, which occurs when an individual has an underlying medical condition that impacts immune system function.² The study authors noted that secondary hemophagocytic lymphohistiocytosis (sHLH) is often triggered by cancer, infection, autoimmune disease, or immunotherapy. The life-threatening hyperinflammatory disease does not have any approved treatments, which could lead to multiple organ failure and death if not treated. Researchers noted that sHLH has a high mortality rate during the first months of diagnosis.¹

“The orphan drug designation is an important milestone in ELA026’s clinical development in sHLH,” Kim‑Hien Dao, DO, PhD, chief medical officer of Electra Therapeutics, said in a news release. “This designation underscores the high unmet medical need in sHLH and supports our continued efforts to evaluate the effectiveness of ELA026 in depleting the pathogenic immune cells responsible for the cytokine storm and addressing the high early mortality observed in this disease.”¹

As a first-in-class monoclonal antibody, ELA026 targets SIRP on the cell surface of myeloid cells and T lymphocytes, which are the pathological immune cells that induce hyperinflammation in sHLH.³

The company recently announced findings from the ongoing open-label, multi-dose, single-arm, multicenter phase 1b study that assessed the safety and efficacy of ELA026 among individuals with sHLH. The study results displayed 100% overall response rate by week 4 and improved survival at 2 months among individuals with treatment-naïve malignancy-associated HLH (mHLH). Additionally, ELA026 exhibited high response rate and favorable safety profile among individuals with sHLH.³

“These data show very promising results for ELA026 as a potential treatment for sHLH, which is a challenging disease that is devastating for patients and has no approved treatment options,” Swaminathan P Iyer, MD, professor in the department of lymphoma/myeloma at The University of Texas MD Anderson Cancer Center, said in a news release.³

According to the release, the phase 1b trial has been expanded to include 20 individuals in cohort 3 and will continue to review ELA026 as frontline therapy for individuals with differing sHLH subtypes.³

“We are delighted that the results of the clinical study of ELA026 in sHLH were recognized and selected as a late-breaking presentation at [European Hematology Association]. This interim data is extremely encouraging, particularly with the high response rates and improved survival at 2 months achieved in treatment-naïve mHLH patients and suggests ELA026’s promise as a first-line treatment,” said Dao in a news release. “We look forward to continuing enrollment in this study and advancing the clinical program to further assess the safety and efficacy of ELA026 in sHLH patients who currently have no approved therapies.”³

REFERENCES

1. Electra Therapeutics Receives FDA Orphan Drug Designation for ELA026 for the Treatment of Hemophagocytic Lymphohistiocytosis (HLH). Electra Therapeutics. News release. October 30, 2024. Accessed November 4, 2024. https://www.businesswire.com/news/home/20241030945860/en

2. Hemophagocytic Lymphohistiocytosis. Cleveland Clinic. October 13, 2022. Accessed November 4, 2024. https://my.clevelandclinic.org/health/diseases/24292-hemophagocytic-lymphohistiocytosis

3. Electra Therapeutics Presents Late-Breaking Clinical Data at EHA2024 from Ongoing Phase 1b Study of ELA026 in Secondary Hemophagocytic Lymphohistiocytosis (sHLH). BioSpace. News release. June 16, 2024. Accessed November 4, 2024. https://www.biospace.com/electra-therapeutics-presents-late-breaking-clinical-data-at-eha2024-from-ongoing-phase-1b-study-of-ela026-in-secondary-hemophagocytic-lymphohistiocytosis-shlh