president and CEO Electra Therapeutics,
Kathy Dong, PharmD, is the president and CEO of Electra Therapeutics, a clinical stage biotechnology company developing antibody therapies against novel targets for immunological diseases and cancer. The company’s lead drug candidate, ELA026, is a first-in-class monoclonal antibody that represents the new generation of drugs for immunology and inflammation—now referred to as I&I drugs—that selectively target disease-causing pathways of the immune system with precision. Discovered by Electra, ELA026 targets signal regulatory proteins (SIRP) on the 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 disease for which there is no approved treatment.
Dong recently spoke with Damian Doherty, editor-in-chief of Inside Precision Medicine, to discuss this rare inflammatory disease, the encouraging clinical trial results for ELA026, and her dedication to developing first-in-class drugs that address complex and difficult-to-treat diseases.
Q: What motivated you to tackle such a complex, challenging disease as sHLH?
Dong: We were driven by the strong scientific rationale and the high unmet medical need. Our team doesn’t shy away from tackling hard challenges and making bold decisions to drive an innovative drug forward to benefit patients.
At Electra, we applied a pioneering approach to target SIRP on certain immune cells as a novel approach for treating immunological diseases and cancer. When our first SIRP-targeted drug candidate, ELA026, emerged and showed the ability to selectively deplete the principle immune cells that drive the pathogenesis of sHLH, we selected it as the lead indication. Despite sHLH being a complex condition with no approved treatment and no established path for drug development, we were motivated by the strong scientific basis and significant unmet need for an effective therapy.
I’ve always had a passion for working on first-in-class treatments that can have a meaningful impact on diseases, particularly those that are challenging to tackle or may be overlooked. The experience at Electra builds on my earlier career. I spent nine years at Gilead Sciences, where I was involved in the launch of transformative treatments for hepatitis B and C, including Viread, SOVALDI, and HARVONI. These drugs changed the standard of care for millions of patients worldwide and enabled us to focus on enhancing diagnosis and linkage to care so that as many patients as possible can achieve clinical benefits. At True North Therapeutics, I was part of a stellar team that pioneered drug development targeting classical complement biology, and discovered a portfolio of antibodies that has now progressed to an approved first-in-class drug for a rare hematological condition as well as clinical proof-of-concept in other diseases.
Like many who work in drug development, I find it so rewarding to work as part of a team, bringing together a shared passion and complementary skills to achieve groundbreaking results.
Q: Can you please describe secondary hemophagocytic lymphohistiocytosis?
Dong: Secondary hemophagocytic lymphohistiocytosis (sHLH) is a rare and life-threatening hyperinflammatory condition. It occurs when the body’s immune system becomes excessively activated and is unable to regulate its response, leading to severe inflammation. The pathophysiology of sHLH is initially triggered by a strong antigenic stimulus such as cancer, infection, or autoimmune disease. Awareness of sHLH has been on the rise over the past two decades and particularly during the last few years, driven by COVID-19 and immunotherapy being possible triggers for sHLH. This has resulted in significant increases in the clinical detection of sHLH.
Clinically, patients with sHLH present with symptoms such as persistent fever, low blood cell counts, enlargement of the spleen and liver, and issues with blood clotting. These symptoms are signs of an overwhelming immune reaction, often referred to as a cytokine storm, which can rapidly lead to multiple organ failure and death if not effectively treated.
Secondary HLH has a high mortality rate, particularly within the first few months after diagnosis. Cancer patients with malignancy-associated HLH, or mHLH, tend to have the poorest outcomes.
Q: What types of treatment are currently available for sHLH?
Dong: Unfortunately, there is no currently approved treatment for sHLH, and existing therapies that are used off-label, including chemotherapy and anti-cytokine therapies, have shown limited effectiveness, underscoring the urgent need for new, safer, and more effective treatments to improve survival for these patients. The severity of this condition, along with the lack of effective therapies, provided a strong rationale for developing a new treatment option to improve outcomes for sHLH patients.
Q: Tell me about ELA026 and recent clinical trial results.
Dong: We believe ELA026 has the potential to transform the treatment landscape for sHLH patients. During this year, promising results from our initial clinical study in sHLH were selected for oral presentation at the most prominent medical meetings in our field, including the European Hematology Association and the American Society of Hematology.
ELA026 is a monoclonal antibody designed to target immune cells that contribute to the cytokine storm in sHLH. It binds to specific receptors (SIRPα, SIRPβ1, and SIRPγ) on myeloid and T cells, and promotes the removal of these pathologically activated cells through cell killing mechanisms. This targeted approach allows for a rapid onset of action, which is crucial in treating the acute nature of sHLH.
At these medical forums, it has been gratifying to share our early data with the clinical and research community. In patients with mHLH who have the poorest prognosis, frontline treatment with ELA026 achieved a 100% response rate by week four, compared to a historical response rate of approximately 40–50% with standard therapies. Additionally, 100% of these patients were discharged from the hospital and about 90% achieved 60-day survival. This improvement in early survival is clinically significant, given the high mortality rate observed in the early stages of sHLH.
In addition, ELA026 has shown effects on key biomarkers of inflammation, such as CRP, ferritin, and sCD25, while demonstrating a favorable safety profile for this patient population. These findings suggest that ELA026 could provide rapid suppression of hyperinflammation and deliver meaningful clinical benefits, representing a critical advancement in treatment options for this severely ill patient population. It is particularly striking that ELA026 achieved such positive initial results in the most challenging segment of patients with mHLH.
Q: How have you been able to build the case for your first-in-class drug candidate?
Dong: Advancing any new drug idea entails both the scientific promise and the practical pathway to market—and for a first-in-class drug candidate, even more rigor and resolve are required. Confidence in a novel drug opportunity is bolstered by showcasing early-stage clinical validation, offering tangible evidence that supports the drug’s potential. Equally important is outlining a clear strategy for addressing unmet needs and how the new drug will fit into the existing market landscape or change it.
I believe we are successfully building the case for ELA026, with a strong scientific basis and compelling clinical proof-of-concept serving as the foundation. Through our clinical program, we are demonstrating meaningful benefits, including improved survival, as a frontline treatment in the patient population with the highest unmet need. I believe these elements will naturally lead to a transformative impact for patients and a compelling value proposition that will result in a successful drug candidate.
Like many drug hunters, we continue to strengthen our case every day as we interrogate scientific queries and act with urgency to achieve progress toward bringing a first-in-class drug to help patients address their disease in a new way.
Q: Looking to the future, what are you most excited about?
Dong: There is tremendous momentum at Electra, and the entire team is excited about the progress of ELA026 in sHLH as well as the potential of our pipeline. The early clinical data for ELA026 in sHLH is highly encouraging and underscores the potential to offer meaningful benefits to patients. As we continue to advance ELA026 through the development process, we are optimistic about its role in transforming outcomes for sHLH patients and potentially becoming an example of a first-in-class treatment in the rapidly emerging landscape of next-generation I&I drugs. Furthermore, validation of the novel SIRP-targeted biology and the unique approach that we set out to explore five years ago enables the expansion of our pipeline into numerous other opportunities.
Damian Doherty has been in media and publishing for nearly 30 years, beginning in the early nineties at News Corporation. Damian has managed, edited, and launched life science titles in drug discovery and precision medicine. He was features editor of Drug Discovery World for fourteen years and founded, established, and edited the Journal of Precision Medicine in 2014. In parallel, Damian founded and organized the Precision Medicine Leaders’ Summit, a global, immersive 3-day senior leadership conference that still runs today. He edited AIMed magazine in 2019 before launching Photo51Media, a platform for illuminating untold, compelling stories in precision healthcare. Damian joined Mary Ann Liebert in 2021 to help steer the new rebrand and relaunch of Clinical OMICS to Inside Precision Medicine.
