FDA grants orphan drug status to CERo’s leukemia treatment

Published 17/06/2025, 13:22
FDA grants orphan drug status to CERo’s leukemia treatment

SOUTH SAN FRANCISCO - CERo Therapeutics Holdings, Inc. (NASDAQ:CERO), whose stock is currently trading near its 52-week low at $6.87, announced Tuesday that the U.S. Food and Drug Administration has granted Orphan Drug Designation to its lead candidate CER-1236 for the treatment of acute myeloid leukemia (AML). According to InvestingPro data, the company’s shares have seen a significant decline over the past year, despite maintaining more cash than debt on its balance sheet.

CER-1236, currently in Phase 1 clinical trials, is designed to engineer a patient’s own T cells to deploy phagocytic mechanisms that enable the cells to "eat" cancer targets alongside traditional T cell destroying mechanisms.

The ongoing first-in-human, multi-center Phase 1/1b study is evaluating the safety and preliminary efficacy of CER-1236 in patients with various forms of AML, including relapsed/refractory cases, patients in remission with measurable residual disease, and newly diagnosed patients with specific mutations.

"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," said Chris Ehrlich, CERo CEO, according to the company’s press release. While InvestingPro analysis indicates the company’s current financial health score as weak, analyst price targets range from $60 to $220, suggesting significant potential upside if the treatment proves successful.

The FDA’s Orphan Drug program aims to advance treatments for conditions affecting fewer than 200,000 U.S. patients annually. The designation provides CERo with incentives including FDA assistance in clinical trial design, access to grants, exemption from application fees, and potential seven-year marketing exclusivity.

CERo’s technology platform creates what it calls Chimeric Engulfment Receptor T cells (CER-T), which the company claims may potentially treat both blood cancers and solid tumors.

The announcement was made in a company press release issued Tuesday. For investors interested in detailed analysis of biotech companies like CERo, InvestingPro offers comprehensive financial metrics, including 14 additional ProTips and advanced valuation tools to assess investment potential in the sector.

In other recent news, CERo Therapeutics Holdings, Inc. announced a one-for-twenty reverse stock split, which will reduce the company’s outstanding common shares from approximately 10.3 million to about 516,092 shares. The company also issued additional Series D Preferred Stock, raising $750,000, and could potentially raise up to $2.25 million more. This funding is intended to support CERo’s clinical programs, including trials for both liquid and solid tumors. CERo has secured two U.S. patents and one European patent application, strengthening its proprietary position in engineered T cell therapeutics. The company initiated a Phase 1 clinical trial for its lead product, CER-1236, targeting acute myeloid leukemia, with the first patient now undergoing treatment. Maxim Group initiated coverage on CERo Therapeutics with a Buy rating and a price target of $3.00, citing the potential of CERo’s CER-T technology. The analyst highlighted that CER-1236’s dual action in leveraging the immune system could offer a more controlled targeting mechanism. This development follows a successful equity financing round that raised $8 million, expected to sustain operations through the release of initial AML data in the third quarter of 2025.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.

Latest comments

Risk Disclosure: Trading in financial instruments and/or cryptocurrencies involves high risks including the risk of losing some, or all, of your investment amount, and may not be suitable for all investors. Prices of cryptocurrencies are extremely volatile and may be affected by external factors such as financial, regulatory or political events. Trading on margin increases the financial risks.
Before deciding to trade in financial instrument or cryptocurrencies you should be fully informed of the risks and costs associated with trading the financial markets, carefully consider your investment objectives, level of experience, and risk appetite, and seek professional advice where needed.
Fusion Media would like to remind you that the data contained in this website is not necessarily real-time nor accurate. The data and prices on the website are not necessarily provided by any market or exchange, but may be provided by market makers, and so prices may not be accurate and may differ from the actual price at any given market, meaning prices are indicative and not appropriate for trading purposes. Fusion Media and any provider of the data contained in this website will not accept liability for any loss or damage as a result of your trading, or your reliance on the information contained within this website.
It is prohibited to use, store, reproduce, display, modify, transmit or distribute the data contained in this website without the explicit prior written permission of Fusion Media and/or the data provider. All intellectual property rights are reserved by the providers and/or the exchange providing the data contained in this website.
Fusion Media may be compensated by the advertisers that appear on the website, based on your interaction with the advertisements or advertisers
© 2007-2025 - Fusion Media Limited. All Rights Reserved.