HanAll Biopharma Announces Orphan Drug Designation for Batoclimab in Japan for Active Thyroid Eye Disease

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• Batoclimab receives Orphan Drug Designation in Japan, advancing treatment for active Thyroid Eye Disease (TED).
• Batoclimab, subcutaneous formulation, offers the potential for at-home administration, improving patient convenience and accessibility.
• Phase 3 study to confirm the potential of batoclimab to address unmet needs of patients with TED in process, with top-line results expected in the 2H 2025.

SEOUL, South Korea, March 17, 2025 /PRNewswire/ -- HanAll Biopharma Co., Ltd. (KRX: 009420.KS), a global biopharmaceutical company committed to discovering and developing innovative medicines for patients, announced that Batoclimab, an anti-FcRn treatment being developed for a range of autoimmune diseases, has received Orphan Drug Designation (ODD) from the Ministry of Health, Labor and Welfare (MHLW) in Japan for active Thyroid Eye Disease (TED).

The Orphan Drug Designation (ODD), granted by the MHLW, is awarded to drugs and biologics intended to treat rare diseases affecting fewer than 50,000 people in Japan, with eligibility based on criteria such as patient population size, medical needs, and the feasibility of development, and possibility of development^[1]. Currently, it is estimated that approximately 35,000 people in Japan are affected by TED.^[2] 

"We are thrilled to have received Orphan Drug Designation for Batoclimab in Japan, marking an important milestone in our efforts to bring this promising treatment to patients in need," said Sean Jeong, MD, MBA, CEO of HanAll Biopharma. "This designation highlights the potential impact Batoclimab could have on the lives of patients with TED. We remain dedicated to advancing the development of this treatment and are focused on bringing it closer to the market."

Batoclimab is a monoclonal antibody designed to target and inhibit FcRn, which plays a crucial role in recycling IgG antibodies. By selectively binding to FcRn, Batoclimab reduces the levels of harmful IgG antibodies, offering the potential to treat a variety of IgG-mediated autoimmune diseases. Being developed as a subcutaneous (SC) formulation, Batoclimab is expected to allow patients to administer the treatment at home, improving convenience and accessibility. Currently, Batoclimab is being investigated globally for conditions such as generalized myasthenia gravis (gMG), thyroid eye disease (TED), chronic inflammatory demyelinating polyneuropathy (CIDP), and Graves' disease.

HanAll, with its licensee, is conducting a Phase 3 study of Batoclimab in active TED. The study, which includes patient enrollment in Japan, aims to confirm the efficacy and safety of Batoclimab as a potential new treatment for individuals affected by TED.

Thyroid Eye Disease (TED), also known as Graves' orbitopathy, is a rare and debilitating autoimmune disorder primarily affecting individuals with hyperthyroidism or Graves' disease. TED is characterized by a range of severe symptoms, including eye bulging, pain, double vision, and, in some cases, vision loss. TED can severely limit daily activities such as reading, driving, and working. In addition, many individuals face significant social and psychological challenges, including concerns about their appearance, anxiety, low mood, and social withdrawal. Currently, treatment options for those with moderate to severe cases of TED remain limited, highlighting the urgent need for more effective therapies to improve patient outcomes and quality of life.

About HanAll Biopharma

HanAll Biopharma (KRX: 009420.KS) is a global biopharmaceutical company with presence in Korea, the USA, Japan, and Indonesia with the mission of making meaningful contributions to patients' lives by introducing innovative, impactful medicines to address severe unmet medical needs. HanAll has been operating a portfolio of pharmaceutical products in the therapeutic areas of endocrine, circulatory, and urologic diseases for over 50 years.

HanAll has also expanded its focus to immunology, oncology, neurology, and ophthalmology to discover and develop innovative medicines for patients with diseases for which there are no effective treatments. One of its lead pipeline assets, HL161 (INN: batoclimab), an anti-FcRn antibody, is being developed in Phase 3 and Phase 2 trials across the world for the treatment of autoimmune diseases including generalized myasthenia gravis (gMG), thyroid eye disease (TED), chronic inflammatory demyelinating polyneuropathy (CIDP). HL161ANS (IMVT-1402), another anti-FcRn antibody from HanAll, is being evaluated in Graves' Disease (GD) and Rheumatoid arthritis (RA). 

Another lead asset, HL036 (INN: tanfanercept), a TNF inhibitor protein, has commenced a Phase 3 VELOS-4 study in the US and is also being evaluated in China for the treatment of dry eye disease. Results from the Phase 1 study, HL192 (ATH-399A), a Nurr1 activator targeting Parkinson's Disease (PD), have been released, and the preparations to initiate a next study in patients with PD is progressing.

For further information, visit our website and connect with us on LinkedIn. For any media inquiries, please contact HanAll PR/IR ([email protected], [email protected]).

Disclaimer Statement 

The contents of this announcement include statements that are, or may be deemed to be, "forward-looking statements." These forward-looking statements can be identified by the use of forward-looking terminology, including the terms "believes," "estimates," "anticipates," "expects," "intends," "may," "will," or "should," and include statements HANALL (the company, we) makes concerning its 2025 business and financial outlook and related plans; the therapeutic potential of its product candidates; the intended results of its strategy and the company, and its collaboration partners', advancement of, and anticipated clinical development, data readouts and regulatory milestones and plans, including the timing of planned clinical trials and expected data readouts; the design of future clinical trials and the timing and outcome of regulatory filings and regulatory approvals. By their nature, forward-looking statements involve risks and uncertainties, and readers are cautioned that any such forward-looking statements are not guarantees of future performance. The company's actual results may differ materially from those predicted by the forward-looking statements. These may include various significant factors, such as our expectations regarding the inherent uncertainties associated with competitive developments, preclinical and clinical trial and product development activities, and regulatory approval requirements. In addition, performance may be affected by our reliance on collaborations with third parties, estimating the commercial potential of our product candidates, our ability to obtain and maintain protection of intellectual property of technologies and drugs, our limited operating history, and our ability to obtain additional funding for operations and to complete the development and commercialization of product candidates. A further list and description of these risks, uncertainties, and other risks can be found in Korea Stock Exchange (KRX) filings and reports, including in our most recent annual report as well as subsequent filings and reports filed by the company with the KRX. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements. These forward-looking statements speak only as of the date of publication of this document. We undertake no obligation to publicly update or revise the information in this press release, including any forward-looking statements, except as may be required by Korean law and regulations. 

SOURCE HanAll Biopharma