Elucida Oncology Announces Positive Early Phase 1 Safety Data Along with Preclinical Data for the Treatment of Brain Metastases for ELU001, an Anti-Folate Receptor Alpha C’Dot Drug Conjugate

• Clinical data presented at the AACR meeting supports a differentiated safety profile for ELU001 in patients with solid tumors
• No evidence of interstitial lung disease, peripheral neuropathy, liver, kidney, cardiac or ocular adverse events
• Efficacy data from dose escalation and expansion studies anticipated in 2023
• New preclinical data demonstrates potential ability of ELU001 to also treat brain metastases

MONMOUTH JUNCTION, N.J., April 20, 2023 (GLOBE NEWSWIRE) -- Elucida Oncology, a clinical-stage biotechnology company developing the next frontier in targeted cancer therapy, presented positive initial safety data from Phase 1 dose escalation trial of ELU001 (NCT05001282) in a poster titled “ELU-FRα-1: A Study to Evaluate ELU001, a C’Dot Drug Conjugate, in Patients with Solid Tumors that Overexpress Folate Receptor Alpha (FRα)” (abstract #CT255) at the American Association for Cancer Research (AACR) 2023 Annual Meeting. In addition, the company presented new preclinical data in a poster titled “Preclinical development of ELU001 - a folate receptor alpha (FRα)-targeted C’Dot drug conjugate (CDC) for the treatment of brain metastases” (abstract #837).

“We are excited to present, for the first time, safety data from our ongoing Phase 1 clinical trial of ELU001, an FRα targeting C’Dot drug conjugate with an exatecan payload for the treatment of refractory solid tumors. The data highlights the differentiated safety profile, with avoidance of many of the normal tissue toxicities associated with antibody drug conjugates targeting FRα as well as those using a topoisomerase 1 payload” stated Geno Germano, President and CEO of Elucida Oncology. “Our ultra-small CDCs are further differentiated from antibody drug conjugates by their ability to penetrate deep into solid tumors, delivery of high concentrations of drug payload, and binding to cancer cells with high avidity. In addition, the avoidance of uptake into healthy tissue and efficient renal elimination reduces the potential for off target toxicities.”

“Data on two different dosing schedules demonstrates an ELU001 safety profile with side-effects confined to predictable, manageable, and reversible hematologic and gastrointestinal adverse events consistent with its exatecan payload. Importantly, there has been no evidence of interstitial lung disease, peripheral neuropathy, or ocular, liver, renal or cardiac toxicities observed to date. We remain on track to complete the dose escalation study by mid-2023 and initiate expansion studies in endometrial and ovarian cancers. We look forward to presenting efficacy data from our dose escalation and expansion studies in 2023,” said Dr. Eliel Bayever, Chief Medical Officer of Elucida Oncology.

ELU001, currently in a Phase 1/2 trial in patients with solid tumors overexpressing folate receptor alpha (FRα), contains ~20 molecules of the topoisomerase-1 inhibitor exatecan-linked via a proteolytic cleavable linker and ~13 folic acid molecules to target FRα-overexpressing cancers. FRα is overexpressed on a variety of tumors including ovarian, endometrial, colorectal, triple negative breast and non-small cell lung, but is minimally expressed on normal tissues making it an attractive tumor-associated antigen for targeted drug delivery.

Preclinical data on brain metastases presented at AACR

New preclinical data with ELU001 highlights its unique ability to penetrate the disrupted blood brain barrier for the treatment of brain metastases, potentially addressing a critically ill patient population. “We were excited to present compelling preclinical data demonstrating our lead agent, ELU001, is capable of selectively localizing in, and treating, lung tumors established in the brains of mice while avoiding the healthy regions of the brain," said Gregory Adams, Ph.D., Chief Scientific Officer of Elucida Oncology.

“ELU001 mediated a significant reduction in the size of early and late tumors in the brains in these animals and often led to the recovery of body weight loss that occurred due to the growth of these tumors. These exciting results suggest that ELU001 may fill a significant unmet need for many patients with metastatic brain cancers,” concluded Dr. Adams.

About Elucida Oncology

Elucida Oncology, Inc., is a clinical-stage biotechnology company pioneering the next frontier in targeted cancer therapy with its first-in-class, ultra-small nanoparticle C’Dot drug conjugate (CDC) platform. CDCs are designed to penetrate deeper into tumors and deliver a significantly higher payload compared to antibody drug conjugates (ADCs). This combined with greater avidity for the target antigen, longer retention in tumors with minimal systemic exposure due to rapid renal clearance confers unique Target or Clear^® properties. In preclinical studies, this has resulted in enhanced efficacy irrespective of antigen expression levels with reduced off-target toxicity, thereby potentially addressing the limitations of ADCs and other novel drug carriers. For more information, please visit www.elucidaoncology.com.

Released April 20, 2023