Regulatory Milestones May Hasten Novel Immunotherapy for AML

The US FDA has granted fast track designation to CER-1236, an investigational cellular immunotherapy, for the treatment of acute myeloid leukemia (AML).¹

This designation is in addition to the existing orphan drug designation for the compound and is expected to accelerate its development and review process.

The FDA's fast track program is designed to expedite the development of drugs that treat serious or life-threatening conditions and address an unmet medical need. This designation offers several benefits to the drug developer, including increased opportunities for FDA interactions, eligibility for priority review, and the ability to submit a biologics license applicaton (BLA) on a rolling basis.²

The FDA's orphan drug designation, previously granted to CER-1236, is reserved for drugs and biologics intended to treat rare diseases or conditions, defined as those affecting fewer than 200,000 people in the United States.³ This designation provides various incentives, including tax credits for clinical research costs, user fee waivers, and a period of market exclusivity for a successful product. The combination of fast track and orphan drug designations underscores the severity of AML and the potential for CER-1236 to address a critical therapeutic gap.

“These FDA designations are important additional validation with regard to the urgency of the condition as well as the potential that the agency sees in the existing data as submitted. Further, these designations help to decrease the potential time to market and provide additional benefits across the FDA process that are expected to prove both medically and financially valuable. We continue to believe that CER-1236 represents a truly novel approach to cancer immunotherapy and are gratified by this regulatory milestone,” said CERo Therapeutics CEO Chris Ehrlich in a press release.¹

CER-1236 is a novel type of engineered T-cell therapy, referred to as a chimeric engulfment receptor T cell (CER T). Unlike traditional chimeric antigen receptor (CAR) T-cell therapies that primarily rely on T-cell cytotoxicity, CER T cells are engineered to integrate both innate and adaptive immune mechanisms. This approach is designed to redirect patient-derived T cells to eliminate cancer cells by activating phagocytic pathways, a process by which cells "engulf" and destroy foreign particles or pathogens.

The investigational drug is currently being evaluated in a first-in-human, multi-center, open-label, phase 1/1b study (NCT06834282) in patients with various presentations of AML. This includes patients with relapsed/refractory disease, those in remission with measurable residual disease (MRD), and newly diagnosed patients with TP53-mutated myelodysplastic syndrome (MDS) or AML. The study is structured in 2 parts: a dose-escalation phase to determine the maximum tolerated dose and a subsequent expansion phase to further evaluate safety and preliminary efficacy.⁴

Key outcome measures for the trial include the incidence of adverse events and serious adverse events, dose-limiting toxicities, and several efficacy end points such as overall response rate, complete response, composite complete response, and MRD. Pharmacokinetic analysis is also a secondary outcome. The goal is to establish the safety profile and an initial understanding of the therapeutic potential of CER-1236 in this patient population, which has a significant unmet need.

This article was generated with assistance from Google Gemini. It was edited and reviewed by Targeted Oncology staff. If you have any questions about the use of AI, please contact us.

REFERENCES:

1. CERo Therapeutics Receives FDA Fast Track Designation for CER-1236 in Acute Myeloid Leukemia (AML). News release. CERo Therapeutics. September 5, 2025. Accessed September 5, 2025. https://tinyurl.com/3mpjsvkj

2. Fast Track. US FDA. Updated August 13, 2024. Accessed September 5, 2025. https://tinyurl.com/y748ywj9

3. Designating an Orphan Product: Drugs and Biological Products. US FDA. Updated August 12, 2024. Accessed September 5, 2025. https://tinyurl.com/5ckfaxtv

4. CER-1236 in Patients With Acute Myeloid Leukemia (AML) (CertainT-1). ClinicalTrials.gov. Updated June 29, 2025. Accessed September 5, 2025. https://clinicaltrials.gov/study/NCT06834282