Elucida Oncology announced today that new data have been presented from its recently completed dose escalation/safety phase of its first-in-human Phase 1/2 clinical trial evaluating ELU001, its lead C’Dot drug conjugate (CDC) clinical candidate, at the ESMO Congress 2023 being held in Madrid, Spain.
- Anti-tumor activity observed across all levels of folate receptor alpha (“FRα”) expression
- 89% of patients with cancers expressing FRα showed stable disease or a partial response following treatment with ELU001, and 21% had a treatment duration of at least nine months
- Generally well-tolerated, and exploratory dose/schedule selected for ongoing dose expansion cohorts in ovarian and endometrial cancers
- Clinical data from dose expansion cohorts expected in first half of 2024
MONMOUTH JUNCTION, N.J., Oct. 23, 2023 (GLOBE NEWSWIRE) -- Elucida Oncology, a clinical-stage biotechnology company pioneering the next frontier in targeted cancer therapy, announced today that new data have been presented from its recently completed dose escalation/safety phase of its first-in-human Phase 1/2 clinical trial evaluating ELU001, its lead C’Dot drug conjugate (CDC) clinical candidate, at the ESMO Congress 2023 being held in Madrid, Spain.
In total, 42 patients, with advanced malignancies and no meaningful life-prolonging therapy options available, were treated with ELU001 on a QW, Q2W or Q3W schedule, and 34 patients in the study completed at least one cycle of treatment. There were 19 patients with solid tumors expressing low to high FRα levels treated with ELU001 in the study, and among them 18 patients completed at least one cycle of treatment. FRα expression levels were assessed using the Ventana FOLR1 (FOLR1-2.1) assay and the PS2+ scoring system.
Anti-tumor activity was observed in patients with low (≥25% - <50%), moderate (≥50% - <75%) and high (≥75%) FRα expression levels, across multiple tumor types including ovarian, colorectal, and endometrial cancers. For the 19 evaluable patients with ≥25% FRα expression (FRα positive), the study achieved a disease control rate (DCR) of 89%, with 1 partial response (PR) and 16 patients achieving stable disease (SD). Additionally, one patient with a FRα- tumor (FRα negative or <25% tumor cells) achieved a partial response, for a total of two PRs in the study.
Four patients out of the 19 evaluable FRα positive patients (21%) achieved a duration of treatment of at least nine months suggesting tumor control and tolerability of ELU001. The longest duration of treatment was 16 months in a patient with endometrial cancer. Two patients were still on treatment as of October 6th, 2023.
Importantly, there was no evidence of some of the severe adverse events that are associated with ADCs, including peripheral neuropathy, ocular keratopathy, cardiac, lung, liver, or kidney toxicities. Drug-related hematological and gastrointestinal adverse events, known to be associated with the exatecan payload, were observed, yet were generally spontaneously reversible, short in duration or otherwise easily manageable, and did not appear to be cumulative.
Given the clinical benefit observed, we have advanced the 2.0 mg/m2 every two weeks (Q2W) as the exploratory dose of ELU001 in our on-going dose expansion study, which consists of 3 cohorts: ovarian cancer high FRα expression, ovarian cancer moderate and low FRα expression, and endometrial cancer positive (low, moderate, high) FRα expression. Notably, in the dose escalation phase of the study three patients with FRα positive endometrial and ovarian cancers were treated with ELU001 at the 2.0 mg/m2 Q2W dose/schedule that has been selected in the expansion study, one met the criteria of PR, and two had stable disease. Anemia and neutropenia were the most common adverse events observed at this dose, and were spontaneously reversible or manageable, and short in duration. Clinical data from the ongoing dose expansion study is expected in the first half of 2024.
Geno Germano, CEO of Elucida Oncology, stated, “We are very pleased to see preliminary anti-tumor activity across all levels of FRα antigen expression, especially considering that the subject population was heavily pre-treated with a median of 5 prior lines of therapy. In addition, the long duration on treatment for some patients at this stage of development is particularly encouraging.” Geno added, “The manageable and favorable safety profile, that is devoid of off-target toxicities observed with antibody drug conjugates, combined with the observed anti-tumor activity have provided early, yet encouraging evidence that our CDC platform is differentiated from ADCs and add confidence in our path forward.”
Dr. Eliel Bayever, Chief Medical Officer of Elucida Oncology, added, “These data are exciting and provide meaningful insights for the development of ELU001 and our platform as we continue to expand the application of our technology with the goal of making a difference to cancer patients. We expect to provide additional clinical data from the dose expansion part of the trial in the first half of 2024.”
About ELU001
ELU001, Elucida Oncology’s lead clinical candidate, is a C’Dot drug conjugate (CDC) that contains ~20 molecules of the topoisomerase-1 inhibitor exatecan linked via a proteolytic cleavable linker, and ~13 folic acid molecules to target cancers that express folate receptor alpha (FRα). ELU001’s multi-valent targeting, greater payload delivery, deep tumor penetration, and ability to access difficult to target tumors in the brain, are important properties distinguishing it from antibody-drug conjugates (ADC). Pre-clinical studies across multiple cancer models demonstrate the significant ability of ELU001 to target both high and lower FRα expressing tumor cells, resulting in enhanced cell killing as compared to an ADC designed to target the same FRα antigen. FRα is overexpressed on a variety of tumors yet is minimally expressed on normal tissues making it an attractive tumor-associated antigen for targeted drug delivery.
About the ELU001 Phase 1/2 Clinical Study
The open-label, multi-center clinical study has two parts: Part 1 Dose Escalation Safety Study to identify the maximum tolerated dose (MTD) and/or the recommended phase 2 dose (RP2D), and Part 2 Tumor Group Expansion Cohort(s) where specific cancer types will be evaluated for efficacy and safety at the RP2D. Part 1 was a basket study that enrolled patients with advanced cancers known to overexpress FRα, including ovarian cancer, endometrial cancer, colorectal cancer, gastric cancer, gastroesophageal junction cancer, triple negative breast cancer, non-small cell lung cancer, and cholangiocarcinoma. The most promising tumor types studied in Part 1 were ovarian and endometrial, and both are being investigated in Part 2. More information about the trial is available at www.clinicaltrials.gov: NCT05001282.
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 CDCs 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 on Elucida Oncology, Inc., please visit www.elucidaoncology.com.
| Investor and BD Contact: | Media Contact: |
| Ramzi Benamar, CFO | media@elucidaoncology.com |
| Elucida Oncology, Inc. | |
| rbenamar@elucidaoncology.com |
