ELA-026

SOUTH SAN FRANCISCO, Calif.--( BUSINESS WIRE )-- Electra Therapeutics, Inc., a clinical stage biotechnology company developing antibody therapies against novel targets for immunological diseases and cancer, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for ELA026 for the treatment of secondary hemophagocytic lymphohistiocytosis (sHLH). ELA026, a first-in-class antibody therapy targeting signal regulatory proteins (SIRP), is a novel approach to treat secondary HLH (sHLH), a rare, life-threatening disease for which there is no approved treatment. “Receiving Fast Track designation for ELA026 reinforces the urgent need for effective treatment options for patients suffering from sHLH,” said Kathy Dong, PharmD, CEO of Electra Therapeutics. “This designation gives us the opportunity to accelerate the development of ELA026, a therapy that we believe has the potential to transform the treatment landscape for sHLH patients. We are committed to advancing this drug candidate with urgency and to work closely with the FDA to bring this innovative therapy to patients as soon as possible.” The FDA grants investigational medicines Fast Track designation to facilitate the development and expedite the review of medicines that demonstrate the potential to treat serious conditions and fill an unmet medical need. Programs with Fast Track designation can benefit from early and more frequent interactions with the FDA to discuss the product candidate's development plan in addition to a rolling submission of the marketing application. Product candidates with Fast Track designation may also be eligible for priority review and accelerated approval. About Secondary Hemophagocytic Lymphohistiocytosis Secondary hemophagocytic lymphohistiocytosis (sHLH) is a rare, life-threatening hyperinflammatory disease for which there is no approved treatment. It can be triggered by cancer, infection, autoimmune disease, or immunotherapy. sHLH is associated with a severe inflammatory response for which patients require immediate intervention. Without effective treatment, patients may experience multiple organ failure and death. sHLH has a high mortality rate during the first months of diagnosis, with malignancy-associated HLH (mHLH) patients having the poorest outcomes. About Electra Therapeutics Electra Therapeutics is a clinical stage biotechnology company developing therapies against novel targets for immunological diseases and cancer. The company’s lead product candidate, ELA026, is a first-in-class monoclonal antibody that targets SIRP on the cell surface of myeloid cells and T lymphocytes, and selectively depletes pathological immune cells. ELA026 is in clinical development for secondary hemophagocytic lymphohistiocytosis (sHLH), a rare, life-threatening hyperinflammatory condition for which there is no approved treatment, as well as additional disease indications. For more information, please visit www.electra-therapeutics.com and follow us on LinkedIn .

ELA026 demonstrated a high overall response rate (ORR) across a range of sHLH patients and a favorable safety profile in this life-threatening hyperinflammatory condition In patients with the poorest prognosis, those with malignancy-associated HLH, early treatment with ELA026 resulted in 100% ORR and improved survival SOUTH SAN FRANCISCO, CA, USA I June 16, 2024 I Electra Therapeutics, Inc. , a clinical stage biotechnology company developing antibody therapies against novel targets for immunological diseases and cancer, announced the presentation of clinical data for ELA026 in secondary hemophagocytic lymphohistiocytosis (sHLH), a life-threatening hyperinflammatory condition. The results were presented today as one of six abstracts selected for oral presentation in a late-breaking session at the European Hematology Association (EHA) Congress in Madrid, Spain. Results were reported for sHLH patients in the ongoing Phase 1b clinical study, which has enrolled a majority of patients with the deadliest sHLH subtype, malignancy-associated HLH (mHLH). In treatment‑naive mHLH patients, ELA026 achieved a 100% overall response rate (ORR) by week 4 and improved survival at two months compared to natural history studies. ELA026 also demonstrated a high response rate across a range of sHLH patients and a favorable safety profile in this patient population. ELA026 is a first-in-class monoclonal antibody that targets signal regulatory protein (SIRP)-α/β1/γ on the cell surface of myeloid cells and T lymphocytes, the pathological immune cells that induce hyperinflammation in sHLH. The Phase 1b study is an ongoing open-label, multi-dose, single-arm, multicenter study designed to evaluate the safety and efficacy of ELA026, assess biomarkers, and identify a dose for Phase 2/3 testing (ClinicalTrials.gov identifier: NCT05416307 ). “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,” said Swaminathan P. Iyer, MD, Professor in the Department of Lymphoma/Myeloma at The University of Texas MD Anderson Cancer Center. “Notably, the analysis reveals improved survival was achieved with ELA026 in treatment-naïve mHLH patients, suggesting benefit from early treatment intervention for patients with this rapidly progressing disease.” Study Results Presented at EHA 2024 Congress The oral presentation at EHA2024, entitled “ELA026 Targeting of SIRP(+) Immune Cells Results in a High Response Rate and Improved 2-Month Survival of Treatment-naïve Malignancy-associated Hemophagocytic Lymphohistiocytosis in a Phase 1 Study,” was presented by the lead author, Abhishek Maiti, MD, Assistant Professor in the Department of Leukemia at The University of Texas MD Anderson Cancer Center. The data describe analysis of sHLH patients in three cohorts of the ongoing Phase 1b clinical study, including the following highlights: “We are delighted that the results of the clinical study of ELA026 in sHLH were recognized and selected as a late-breaking presentation at EHA. This interim data is extremely encouraging, particularly with the high response rates and improved survival at two months achieved in treatment-naïve mHLH patients, and suggests ELA026’s promise as a first-line treatment. Survival at two months is a clinically meaningful benefit for this patient population and demonstrates the ability of ELA026 to rapidly extinguish the hyperinflammation in sHLH, enabling mHLH patients to pursue curative therapies for their underlying cancer,” said Kim‑Hien Dao, DO, PhD, Chief Medical Officer at Electra. “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.” About Secondary Hemophagocytic Lymphohistiocytosis (sHLH) Secondary hemophagocytic lymphohistiocytosis (sHLH) is a rare, life-threatening inflammatory disease for which there is no approved treatment. It can be triggered by cancer (malignancy-associated HLH, or mHLH), infection, autoimmune disease, or immunotherapy. sHLH is associated with a systemic inflammatory response for which patients require immediate intervention. Without treatment, patients may experience multiple organ failure and death. sHLH has a high mortality rate during the first months of diagnosis, with mHLH patients having the poorest outcomes. About Electra Therapeutics Electra Therapeutics is a clinical stage biotechnology company developing therapies against novel targets for immunological diseases and cancer. The company’s lead product candidate, ELA026, is a first-in-class monoclonal antibody that targets SIRP on the cell surface of myeloid cells and T lymphocytes, and precisely depletes pathological immune cells. ELA026 is in clinical development for secondary hemophagocytic lymphohistiocytosis (sHLH), a rare, life-threatening hyperinflammatory condition for which there is no approved treatment, as well as additional disease indications. For more information, please visit www.electra-therapeutics.com and follow us on LinkedIn . 1 Löfstedt A, et al. Blood. 2024 Jan 18;143(3):233-242. SOURCE: Electra Therapeutics