The Japan Times - OBI-902 Has Been Granted by US FDA for Orphan Drug Designation for the Treatment of Cholangiocarcinoma

TAIPEI, TW / ACCESS Newswire / November 17, 2025 / OBI Pharma, a clinical-stage oncology company (4174.TWO) received notification from the US FDA stating that the request for Orphan Drug Designation of OBI-902 TROP2 ADC for the treatment of Cholangiocarcinoma has been granted. OBI-902 is the first OBI-developed ADC that incorporates our proprietary site-specific glycan-conjugated ADC enabling technology.

Cholangiocarcinoma is a rare and lethal malignancy with fewer than 50,000 patients in the United States and a 5-year survival rate ranging from 2% and 23% depending on disease stage, histological subtype, and localization ¹ . At present, there are no FDA approved ADC therapies for cholangiocarcinoma.

To encourage the industry to develop new treatment options for rare diseases, the US FDA grants Orphan Drug Designation to experimental therapies that have the potential to treat these diseases. In the United States, a rare disease is defined as any condition that affects fewer than 200,000 patients. After granting Orphan Drug Designation, the US FDA qualifies companies or drug developers incentives such as tax credits for clinical trials, exemption from user fees, and marketing exclusivity².

In August 2025, OBI launched a phase I/II clinical trial in the United States and Taiwan, recruiting patients with advanced solid tumors. The objectives of the trial are to study the safety, pharmacokinetics, and preliminary efficacy profile of OBI-902 in these patient populations.

Heidi Wang, Ph.D, OBI Pharma's Chief Executive Officer noted, "Based on our preclinical data, OBI-902 has several important advantages over other TROP2 ADCs either approved or in development; including high stability in blood circulation, excellent bystander effect that extends the killing to neighboring cancer cells lacking TROP2 expression, potential ability to overcome drug resistance, and outstanding activity in animal and organoid models of cancer. Importantly, this marks the first time an ADC that incorporates OBI's proprietary GlycOBI^® ADC technology is being evaluated in patients, including those diagnosed with cholangiocarcinoma. We look forward to investigating this potential best-in-class TROP2 ADC in the clinic."

About OBI-902

OBI-902 is a TROP2-targeted antibody-drug conjugate (ADC) that carries a potent topoisomerase I inhibitor payload to kill tumor cells and with a drug-antibody ratio (DAR) of 4. TROP2 is highly expressed in a variety of solid tumors such as breast, lung, biliary, bile duct (cholangiocarcinoma), ovarian, gastric, and many other cancer types, rendering it an ideal target for cancer therapy.

OBI-902 is a novel site-specific glycan-conjugated ADC using OBI's proprietary GlycOBI platform, which provides improved stability and enhanced hydrophilicity. OBI-902 demonstrated remarkable antitumor efficacy, improved pharmacokinetic characteristics, and a favorable safety profile in various animal models. The IND of OBI-902 was cleared by the US FDA on April 30, 2025.

Since December 2021, OBI has been granted by Biosion, Inc. (www.biosion.com) an exclusive, worldwide (except in China) license to a TROP2 targeting antibody amino acid sequence. Biosion holds exclusive rights to that antibody sequence in China. OBI holds worldwide commercial rights to OBI-902, except for the rights pertaining to the antibody in China.

About GlycOBI^®

OBI has developed a unique clinical stage, glycan-based site-specific ADC technology (GlycOBI^®), which is in a 'Plug and Play' format and compatible with any antibodies, linkers, and payloads in drug-antibody ratio (DAR) up to 16. Utilizing OBI's proprietary dual-function enzyme (EndoSymeOBI^®) and linker technology (HYPrOBI^®), homogenous ADCs are manufactured with an efficient and scalable process under GMP conditions. The conjugation process of GlycOBI^® avoids disrupting the antibody structure and ensures the ADC has similar biophysical characteristics to the native antibody. Furthermore, OBI's linker technology has improved conjugation efficiency of the payload, reduced aggregation propensity, which provides advantages on manufacturing ADC products. GlycOBI^® conjugated ADCs have overcome the limitations of traditional ADCs and achieved better antitumor activity and stability in various in vivo animal studies. GlycOBI^®, EndoSymeOBI^®, and HYPrOBI^® are part of the armamentarium of OBI's Obrion™ ADC Enabling Technologies that also include ThiOBI^® and GlycOBI DUO™. OBI-902 is the first ADC utilizing OBI's Obrion™ ADC enabling technology for evaluation of safety and efficacy in Cancer, currently under Phase I/II clinical trial in the US and Taiwan.

About OBI Pharma

OBI is a clinical stage global oncology company that is headquartered in Taiwan and established in 2002. Its mission, together with its wholly owned subsidiary OBI Pharma USA, Inc., is to develop novel therapeutic agents for patients with high unmet medical needs.

OBI's primary focus is the development of novel ADCs, including the first-generation cysteine-based TROP2 ADC, OBI-992. Using the company's proprietary ADC enabling technology, GlycOBI^®, powered by EndoSymeOBI^® and HYPrOBI^®; OBI has created its next-generation novel ADC pipeline, including monospecific: OBI-902 (TROP2), OBI-904 (Nectin-4), bispecific single payload (HER2 x TROP2), and bispecific, dual payload (cMET x HER3) ADCs. To broaden the applicability of linker technology, HYPrOBI^®, OBI further developed a novel ThiOBI^® platform to enable irreversible cysteine-based conjugation. Additionally, OBI's pipeline includes the first-in-class AKR1C3-targeted small-molecule prodrug OBI-3424, which selectively releases a potent DNA-alkylating antitumor agent in the presence of the aldo-keto reductase 1C3 (AKR1C3) enzyme that is highly expressed in tumors. Additional information can be found at www.obipharma.com.

GlycOBI^®, EndoSymeOBI^®, ThiOBI^® and HYPrOBI^®are registered trademarks of OBI Pharma. Obrion™ and GlycOBI DUO™ are trademarks under registration.

¹National Institute of Health for Rare Diseases. Sept. 2025

https://rarediseases.info.nih.gov/diseases/9304/cholangiocarcinoma

²US FDA website. Designating an Orphan Product: Drugs and Biological Products Sept.25 https://www.fda.gov/industry/medical-products-rare-diseases-and-conditions/designating-orphan-product-drugs-and-biological-products

Forward-Looking Statements

Statements included in this press release that are not a description of historical facts are forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements include, but are not limited to, statements about future clinical trials, results and the timing of such trials and results. Such risk factors are identified and discussed from time to time in OBI Pharma's reports and presentations, including OBI Pharma's filings with the Taiwan Securities and Futures Bureau.

COMPANY CONTACT:
Kevin Poulos, Chief Business Officer
OBI Pharma USA, Inc.
+1 (619) 537 7698, ext. 102
[email protected]

SOURCE: OBI Pharma USA, Inc.

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T.Sasaki--JT