CERo Therapeutics Holdings, Inc. Announces FDA Orphan Drug Designation Granted to CER-1236 for the Treatment of Acute Myeloid Leukemia (AML)

Rhea-AI Summary

CERo Therapeutics (NASDAQ: CERO) has received FDA Orphan Drug Designation (ODD) for CER-1236, its lead drug candidate targeting acute myeloid leukemia (AML). CER-1236 is an innovative T cell therapy that engineers patients' own immune cells to deploy phagocytic mechanisms alongside traditional T cell destroying mechanisms. The drug is currently in a Phase 1/1b clinical trial evaluating safety and preliminary efficacy in various AML patient groups. The study includes dose escalation and expansion phases, measuring outcomes such as adverse events, toxicities, and response rates. The ODD status grants CERo significant benefits, including FDA trial design assistance, grant eligibility, fee exemptions, and 7 years of marketing exclusivity upon approval.

Positive

• FDA Orphan Drug Designation granted for lead candidate CER-1236, providing 7 years of marketing exclusivity and other benefits
• Innovative therapy approach combining phagocytic mechanisms with T cell therapeutics shows potential differentiation in AML treatment
• Phase 1/1b clinical trial already underway with comprehensive evaluation of safety and efficacy endpoints
• Company eligible for FDA assistance in trial design and exemption from drug approval application fees

Negative

• Drug still in early Phase 1 stage with no efficacy data available yet
• Safety and tolerability profile yet to be established
• Potential competition from other AML treatments in development

Insights

Oncology Clinical Development Expert

FDA's Orphan Drug Designation for CER-1236 provides regulatory benefits and market exclusivity, bolstering CERo's AML treatment development pipeline.

The FDA has granted Orphan Drug Designation (ODD) to CERo Therapeutics' lead candidate CER-1236 for acute myeloid leukemia (AML) treatment, providing significant regulatory advantages. This designation is particularly valuable as it offers seven years of market exclusivity upon approval, exemption from FDA application fees, and special assistance with clinical trial design.

CER-1236 represents an innovative approach in the challenging AML treatment landscape. The therapy engineers patients' own T cells to deploy phagocytic mechanisms (literally "cell-eating") alongside traditional T cell killing methods. This dual-mechanism approach could potentially address limitations of existing therapies in a disease with historically poor outcomes.

The ongoing Phase 1/1b trial is investigating CER-1236 across multiple AML patient populations with significant unmet needs: relapsed/refractory patients, those in remission with measurable residual disease, and newly diagnosed patients with TP53 mutations (which typically indicate poor prognosis). The trial design appropriately focuses on both safety (adverse events, dose-limiting toxicities) and preliminary efficacy markers including overall response rate and complete response.

While this ODD status represents regulatory recognition of the therapy's potential, investors should note that CER-1236 remains in early clinical development. The Phase 1 status indicates several years of development likely remain before potential commercialization, though the orphan designation may accelerate this timeline somewhat.

SOUTH SAN FRANSCISCO, Calif., June 17, 2025 (GLOBE NEWSWIRE) -- CERo Therapeutics Holdings, Inc., (Nasdaq: CERO) (“CERo” or the “Company”) an innovative immunotherapy company seeking to advance the next generation of  engineered T cell therapeutics that deploy phagocytic mechanisms, announces that the U.S. Food and Drug Administration (FDA) has granted CERo’s Orphan Drug Designation (ODD) for the company's lead drug candidate CER-1236, for the treatment of acute myeloid leukemia (AML). CER-1236 is an innovative therapy that engineers a cancer patient's own T cell therapeutics that deploy phagocytic (i.e., target-cell eating) mechanisms alongside the array of built-in target cell destroying mechanisms used by T cells.

CER-1236 is currently in Phase 1 clinical trials for AML. The first-in-human, multi-center, open label, Phase 1/1b study is designed to evaluate the safety and preliminary efficacy of CER-1236 in patients with acute myeloid leukemia that is either relapsed/refractory, or in remission with measurable residual disease, or newly diagnosed patients with TP53 mutated MDS/AML or AML. The two-part study has begun with dose escalation to determine highest tolerated dose and recommended dose for Phase 2, followed by an expansion phase to evaluate safety and efficacy.  Primary outcome measures include incidence of adverse events (AEs) and serious adverse events (SAEs), incidence of dose limited toxicities and estimation of overall response rate (ORR), complete response (CR), composite complete response (cCR), and measurable residual disease (MRD).  Secondary outcome measures include pharmacokinetics (PK).

Chris Ehrlich, CERo CEO, commented, “Orphan Drug Designation underscores the importance of developing new treatments for AML, and the potential for CER-1236 to provide a new and differentiated approach toward treatment.  We believe that we are at the forefront of innovation in immuno-oncology and are grateful for the recognition from FDA.  We look forward to providing updates on our trial in the near term.”

The FDA's Orphan Drug program is designed to advance the development of drugs that treat a condition affecting 200,000 or fewer US patients annually.  ODD status is given to medicinal products that represent a significant benefit over existing treatments and are intended for the treatment of a disease that is life-threatening or chronically debilitating. The ODD designation qualifies CERo and CER-1236 for certain incentives, which include FDA assistance in designing clinical trials, access to the FDA Orphan Drug Grants Program, exemption from the drug approval application fee and eligibility for seven years of marketing exclusivity.

About CERo Therapeutics Holdings, Inc.

CERo is an innovative immunotherapy company advancing the development of next generation engineered T cell therapeutics for the treatment of cancer. Its proprietary approach to T cell engineering, which enables it to integrate certain desirable characteristics of both innate and adaptive immunity into a single therapeutic construct, is designed to engage the body’s full immune repertoire to achieve optimized cancer therapy. This novel cellular immunotherapy platform is expected to redirect patient-derived T cells to eliminate tumors by building in engulfment pathways that employ phagocytic mechanisms to destroy cancer cells, creating what CERo refers to as Chimeric Engulfment Receptor T cells (“CER-T”). CERo believes the differentiated activity of CER-T cells will afford them greater therapeutic application than currently approved chimeric antigen receptor (“CAR-T”) cell therapy, as the use of CER-T may potentially span both hematological malignancies and solid tumors. CERo has commenced clinical trials for its lead product candidate CER-1236 for hematological malignancies.

Forward-Looking Statements

This communication contains statements that are forward-looking and as such are not historical facts. This includes, without limitation, statements regarding the financial position, business strategy and the plans and objectives of management for future operations of CERo, as well as statements regarding the Company’s plans to regain compliance with Nasdaq listing requirements and the ability for the Company’s securities to remain listed on Nasdaq.  These statements constitute projections, forecasts and forward-looking statements, and are not guarantees of performance. Such statements can be identified by the fact that they do not relate strictly to historical or current facts. When used in this communication, words such as “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “might,” “plan,” “possible,” “potential,” “predict,” “project,” “should,” “strive,” “would” and similar expressions may identify forward-looking statements, but the absence of these words does not mean that a statement is not forward-looking. When CERo discusses its strategies or plans, it is making projections, forecasts or forward-looking statements. Such statements are based on the beliefs of, as well as assumptions made by and information currently available to, CERo’s management.

Actual results could differ from those implied by the forward-looking statements in this communication. Certain risks that could cause actual results to differ are set forth in CERo’s filings with the Securities and Exchange Commission, including its most recent Annual Report on Form 10-K and subsequent Quarterly Reports on Form 10-Q, and the documents incorporated by reference therein. The risks described in CERo’s filings with the Securities and Exchange Commission are not exhaustive. New risk factors emerge from time to time, and it is not possible to predict all such risk factors, nor can CERo assess the impact of all such risk factors on its business, or the extent to which any factor or combination of factors may cause actual results to differ materially from those contained in any forward-looking statements. Forward-looking statements are not guarantees of performance. You should not put undue reliance on these statements, which speak only as of the date hereof. All forward-looking statements made by CERo or persons acting on its behalf are expressly qualified in their entirety by the foregoing cautionary statements. CERo undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law.

Contact:

Chris Ehrlich
Chief Executive Officer
chris@cero.bio

Investors:

CORE IR
investors@cero.bio

FAQ

What is the significance of FDA Orphan Drug Designation for CERO stock?

The FDA Orphan Drug Designation grants CERo Therapeutics significant benefits including 7 years of marketing exclusivity, FDA assistance in trial design, grant eligibility, and fee exemptions, potentially enhancing the company's market position.

What type of cancer treatment is CERo Therapeutics (CERO) developing?

CERo is developing CER-1236, an innovative T cell therapy that engineers patients' own immune cells to deploy phagocytic mechanisms alongside traditional T cell destroying mechanisms for treating acute myeloid leukemia (AML).

What phase of clinical trials is CER-1236 currently in?

CER-1236 is currently in a Phase 1/1b clinical trial, which includes dose escalation to determine the highest tolerated dose and an expansion phase to evaluate safety and efficacy.

What patient populations will CER-1236 target in its clinical trials?

The trial targets patients with relapsed/refractory AML, patients in remission with measurable residual disease, and newly diagnosed patients with TP53 mutated MDS/AML or AML.

What are the primary outcome measures for CER-1236's clinical trial?

Primary outcome measures include incidence of adverse events, serious adverse events, dose limited toxicities, and estimation of overall response rate, complete response, composite complete response, and measurable residual disease.