Takeda Announces Approval of HYQVIA® 10% S.C. (Subcutaneous) Injection Set in Japan for Patients with Agammaglobulinemia or Hypogammaglobulinemia




Takeda Announces Approval of HYQVIA® 10% S.C. (Subcutaneous) Injection Set in Japan for Patients with Agammaglobulinemia or Hypogammaglobulinemia

• HYQVIA [Immune Globulin Infusion 10% (Human) with Recombinant Human Hyaluronidase] is the First and Only Facilitated Subcutaneous Immunoglobulin (fSCIG) Approved in Japan for Agammaglobulinemia and Hypogammaglobulinemia
• Administration of Recombinant Human Hyaluronidase Prior to Immunoglobulin Facilitates Subcutaneous Infusion of Larger Volumes, Potentially Reducing Frequency and Giving Patients More Flexibility
• Approval Expands Takeda’s Portfolio of Differentiated Immunoglobulin Therapies and Reflects the Company’s Commitment to Bring High-Quality Plasma-Derived Therapies to Patients in Japan

OSAKA, Japan & CAMBRIDGE, Mass.–(BUSINESS WIRE)–Takeda (TSE:4502/NYSE:TAK) today announced that the Japanese Ministry of Health, Labour and Welfare has approved the use of HYQVIA^® [Immune Globulin Infusion 10% (Human) with Recombinant Human Hyaluronidase] in patients with agammaglobulinemia or hypogammaglobulinemia¹, disorders characterized by very low or absent levels of antibodies and an increased risk of serious recurring infection caused by primary immunodeficiency (PID) or secondary immunodeficiency (SID)². The approval marks availability of the first and only facilitated subcutaneous immunoglobulin (fSCIG) therapy as a treatment option for appropriate patients in Japan.

HYQVIA is the first plasma-derived therapy for subcutaneous injection in Japan that consists of a combination of one vial of Immunoglobulin 10% and one vial of Recombinant Human Hyaluronidase PH20 (rHuPH20). The administration of rHuPH20 increases the dispersion and absorption of immunoglobulin (IG) in the subcutaneous tissue, allowing larger volumes to be infused in the infusion site. This allows for less frequent dosing compared to other subcutaneous IG products, while avoiding the need for venous access. The ability to infuse a larger infusion volume is expected to increase administration flexibility for patients with agammaglobulinemia or hypogammaglobulinemia by decreasing the dosing frequency to once every 3 or 4 weeks, as compared to weekly or bi-weekly with conventional SCIG treatments.

The approval is based on data from two pivotal Phase 3, open-label, non-controlled studies evaluating the efficacy, safety, tolerability and pharmacokinetics in Japanese subjects with PID (NCT05150340, NCT05513586). In these studies, the efficacy and safety profile of HYQVIA in 16 patients aged 2 years or older in Japan were evaluated based on the results of the clinical trials. The Geo Mean of IgG trough level at the last 3 visits was 9.494g/L and was maintained at level comparable to treatment with intravenous or subcutaneous immunoglobulin (Geo Mean of IgG trough level 9.624g/L). The major adverse reactions were pyrexia 5 patients (31.3%) and infusion site erythema, injection site erythema, infusion site swelling, infusion site pain, and headache (12.5%)¹. Data from two Phase 3 clinical trials conducted in patients with PID in North America (NCT00814320, NCT01175213) was also included in the submission.

“We are delighted that HYQVIA, approved in more than 40 countries worldwide, has now been approved in Japan,” said Naoyoshi Hirota, Regional Head of Research & Development for Takeda’s Plasma-Derived Therapies Business Unit in Japan. “The subcutaneous IG therapies currently available in Japan for patients with agammaglobulinemia or hypogammaglobulinemia require infusion once every week or every 2 weeks. We are proud to offer Japanese patients the first and only facilitated subcutaneous treatment option that offers a reduced dosing frequency of every 3 or 4 weeks.”

“There is a high unmet need for plasma-derived therapies (PDTs) in patients in Japan, which is anticipated to increase as education and timely diagnosis rates continue to improve,” said Kristina Allikmets, head of research & development for Takeda’s Plasma-Derived Therapies Business Unit. “The approval of HYQVIA, the first and only facilitated SCIG treatment, is further evidence of Takeda’s commitment to add to the standard of care for patients in Japan. We look forward to continuing to bring new therapeutic options that support and enhance the experience of patients in our home country throughout the next decade.”

With this approval, Takeda is now able to offer a range of SCIG therapies to patients based on their individual administrative needs, reflecting the company’s commitment to offer patients in Japan a broader choice of treatment options. It also follows the announcement of a significant investment to build a new manufacturing facility for plasma-derived therapies (PDTs) in Osaka, Japan. HYQVIA is also currently under review in Japan for additional indications.

About Agammaglobulinemia or Hypogammaglobulinemia

Agammaglobulinemia is an inherited disorder caused by a gene defect that blocks the growth of normal, mature immune cells called B lymphocytes³. Hypogammaglobulinemia is a condition in which patients have low levels of antibodies due either to inherited genetic conditions (PID) or secondary effects (SID) – like chemotherapy, certain comorbid disorders, or immunosuppressants². Individuals with any form of antibody deficiency frequently experience recurring and/or severe infections, and immunoglobulin replacement therapy can increase the level of antibodies in the body.

About Primary Immunodeficiency and Secondary Immunodeficiency

Primary immunodeficiency describes a heterogeneous group of more than 480 rare genetic diseases wherein part of the immune system is missing or not functioning properly⁴. Secondary immunodeficiency is defined as an acquired impairment of the immune response resulting from an underlying condition or factors extrinsic to the immune system. SID may occur as a consequence of malnutrition, metabolic disorders, use of immunosuppressive medications, chronic infections, malignancies, or severe trauma⁵. Due to their impaired immune system, patients with PID and SID may be more susceptible to infection, and it may take longer to recover from it. In patients with antibody deficiency and increased susceptibility and/or persistent infections, substitution with functional antibodies (immunoglobulin replacement therapy) is the standard of care to support the immune system’s functioning⁵.

About HYQVIA^®

HYQVIA is the combination product for subcutaneous injection containing one each vial of Subcutaneous Immunoglobulin 10% (SCIG10%) and Recombinant Human Hyaluronidase PH20 (rHuPH20). As of May 2024, HYQVIA has been approved in more than 40 countries worldwide.

HYQVIA Product Overview in Japan

About Takeda

Takeda is focused on creating better health for people and a brighter future for the world. We aim to discover and deliver life-transforming treatments in our core therapeutic and business areas, including gastrointestinal and inflammation, rare diseases, plasma-derived therapies, oncology, neuroscience and vaccines. Together with our partners, we aim to improve the patient experience and advance a new frontier of treatment options through our dynamic and diverse pipeline. As a leading values-based, R&D-driven biopharmaceutical company headquartered in Japan, we are guided by our commitment to patients, our people and the planet. Our employees in approximately 80 countries and regions are driven by our purpose and are grounded in the values that have defined us for more than two centuries. For more information, visit www.takeda.com.

Important Notice

For the purposes of this notice, “press release” means this document, any oral presentation, any question-and-answer session and any written or oral material discussed or distributed by Takeda Pharmaceutical Company Limited (“Takeda”) regarding this release. This press release (including any oral briefing and any question-and-answer in connection with it) is not intended to, and does not constitute, represent or form part of any offer, invitation or solicitation of any offer to purchase, otherwise acquire, subscribe for, exchange, sell or otherwise dispose of, any securities or the solicitation of any vote or approval in any jurisdiction. No shares or other securities are being offered to the public by means of this press release. No offering of securities shall be made in the United States except pursuant to registration under the U.S. Securities Act of 1933, as amended, or an exemption therefrom. This press release is being given (together with any further information which may be provided to the recipient) on the condition that it is for use by the recipient for information purposes only (and not for the evaluation of any investment, acquisition, disposal or any other transaction). Any failure to comply with these restrictions may constitute a violation of applicable securities laws.

The companies in which Takeda directly and indirectly owns investments are separate entities. In this press release, “Takeda” is sometimes used for convenience where references are made to Takeda and its subsidiaries in general. Likewise, the words “we”, “us” and “our” are also used to refer to subsidiaries in general or to those who work for them. These expressions are also used where no useful purpose is served by identifying the particular company or companies.

Forward-Looking Statements

This press release and any materials distributed in connection with this press release may contain forward-looking statements, beliefs or opinions regarding Takeda’s future business, future position and results of operations, including estimates, forecasts, targets and plans for Takeda. Without limitation, forward-looking statements often include words such as “targets”, “plans”, “believes”, “hopes”, “continues”, “expects”, “aims”, “intends”, “ensures”, “will”, “may”, “should”, “would”, “could”, “anticipates”, “estimates”, “projects” or similar expressions or the negative thereof. These forward-looking statements are based on assumptions about many important factors, including the following, which could cause actual results to differ materially from those expressed or implied by the forward-looking statements: the economic circumstances surrounding Takeda’s global business, including general economic conditions in Japan and the United States; competitive pressures and developments; changes to applicable laws and regulations, including global health care reforms; challenges inherent in new product development, including uncertainty of clinical success and decisions of regulatory authorities and the timing thereof; uncertainty of commercial success for new and existing products; manufacturing difficulties or delays; fluctuations in interest and currency exchange rates; claims or concerns regarding the safety or efficacy of marketed products or product candidates; the impact of health crises, like the novel coronavirus pandemic, on Takeda and its customers and suppliers, including foreign governments in countries in which Takeda operates, or on other facets of its business; the timing and impact of post-merger integration efforts with acquired companies; the ability to divest assets that are not core to Takeda’s operations and the timing of any such divestment(s); and other factors identified in Takeda’s most recent Annual Report on Form 20-F and Takeda’s other reports filed with the U.S. Securities and Exchange Commission, available on Takeda’s website at: https://www.takeda.com/investors/sec-filings/ or at www.sec.gov. Takeda does not undertake to update any of the forward-looking statements contained in this press release or any other forward-looking statements it may make, except as required by law or stock exchange rule. Past performance is not an indicator of future results and the results or statements of Takeda in this press release may not be indicative of, and are not an estimate, forecast, guarantee or projection of Takeda’s future results.

Medical Information

This press release contains information about products that may not be available in all countries, or may be available under different trademarks, for different indications, in different dosages, or in different strengths. Nothing contained herein should be considered a solicitation, promotion or advertisement for any prescription drugs including the ones under development.

______________________________
REFERENCES

¹ HYQVIA 10% Subcutaneous Injection Set Package Insert in Japan.

² Pimenta et al. Hypogammaglobulinemia: a diagnosis that must not be overlooked. BJMBR. 2019;52(10): e8926.

³ National Organization for Rare Disorders https://rarediseases.org/rare-diseases/agammaglobulinemia/
⁴ Tangye et al. Human Inborn Errors of Immunity: 2022 Update on the Classification from the International Union of Immunological Societies Expert Committee. J Clin Immunol. 2022;42(7):1473-1507.

⁵ Tuano KS, Seth N, Chinen J. Secondary immunodeficiencies: An overview. Ann Allergy Asthma Immunol. 2021;127(6):617-626.

Contacts

Media:

International Media
Kristine Kelly

Kristine.Kelly@takeda.com
+41 79 268 68 21

Japanese Media
Shigeyuki Matsui

Shigeyuki.Matsui@takeda.com
+81 (80) 3594-2460

Exicure, Inc. Partners with GPCR Therapeutics to Fuel New Growth in Biotech




Exicure, Inc. Partners with GPCR Therapeutics to Fuel New Growth in Biotech

CHICAGO–(BUSINESS WIRE)–Exicure, Inc. (Nasdaq: XCUR, “Exicure”, “the Company”), has announced the signing of a Memorandum of Understanding (MOU) with GPCR Therapeutics, Inc. (“GPCR Therapeutics”) on December 24, 2024, aimed at the acquisition of GPCR USA, a subsidiary of GPCR Therapeutics, and the technology transfer and collaborative research on GPCR Therapeutics’ ongoing drug development pipelines. Through this acquisition, Exicure plans to secure key technical personnel by purchasing all shares of GPCR USA held by GPCR Therapeutics. Following this, Exicure intends to receive technology transfer for GPCR Therapeutics’ CXCR4 inhibitor, which is currently in Phase 2 clinical trials with the FDA, along with its related patents and intellectual property (IP). By acquiring excellent research personnel and clinical pipelines, Exicure aims to advance as a clinical-stage biotech company. GPCR Therapeutics plans to successfully finalize ongoing clinical trials involving stem cell mobilizers (SCM) targeting multiple myeloma patients and prepare for clinical studies related to acute myeloid leukemia (AML). The market size for the ongoing Phase 2 trials is estimated to be around $1 billion to $2 billion annually.

Moreover, Exicure plans to engage in collaborative research and development in various forms for GPCR Therapeutics’ ongoing research in immuno-oncology, fibrosis treatments, and obesity therapies. GPCR Therapeutics, the partner with Exicure in this MOU, is a South Korean drug development company specializing in GPCR (G Protein-Coupled Receptor, “GPCR”), which represents over one-third of all drug targets. GPCR Therapeutics has secured target patents around prominent GPCRs such as CXCR4 and possesses multiple pipelines addressing blood cancers and solid tumors, genetic disorders, idiopathic pulmonary fibrosis, and obesity, including ongoing Phase 2 trials in the U.S. for multiple myeloma. Dr. Pina Cardarelli, who previously served as Vice President at Bristol-Myers Squibb, where she led the development of the first-ever immuno-oncology drugs, Yervoy (ipilimumab) and Opdivo (nivolumab), has served as the Chief Scientific Officer (CSO) of GPCR Therapeutics since 2019.

In 2021, GPCR Therapeutics established a subsidiary in the U.S. to align its R&D with global pharmaceutical standards and demands. During the American Society of Hematology (ASH) conference held from December 7-10, 2024, GPCR Therapeutics presented three exhibits detailing: (1) interim results from ongoing Phase 2 trials, (2) data on enhancing treatment efficacy for acute myeloid leukemia (AML), and (3) preclinical data on boosting T cell responses to offer various therapies, including in vivo CAR-T. Additionally, on December 24, 2024, Exicure completed the $8.7 million capital investment. Combined with the $2 million capital investment executed on December 9, 2024, the $1.3 million raised in November, and an expected additional $4 million, the Company plans to secure a total of $14 million in capital investments. The funds will primarily be used for the acquisition of GPCR USA, clinical trial costs, and its ongoing operations.

About GPCR Therapeutics, Inc.

GPCR Therapeutics, Inc., headquartered in Seoul, South Korea, is a clinical-stage international biopharmaceutical company with an innovative approach to developing therapeutics built on its proprietary GPCR data. The company’s lead small molecule asset, GPC-100/Burixafor, targets CXCR4, one of the most prevalent chemokine GPCRs overexpressed in various cancers. GPCR Therapeutics is currently conducting a Phase 2 clinical trial in the U.S. to assess the efficacy of the combination of GPC-100 and propranolol in patients with multiple myeloma. The company is also actively collaborating with domestic and international biotechnology firms. By targeting the unique pharmacology of GPCR pairs, GPCR Therapeutics aims to develop life-changing treatments for cancer and other diseases. Notably, the interaction between CXCR4 and the beta-2 adrenergic receptor (B2AR) presents an alternative signaling pathway that is synergistically dependent on the activation of both CXCR4 and B2AR.

About Exicure, Inc.

Exicure, Inc. has historically been an early-stage biotechnology company focused on developing nucleic acid therapies targeting ribonucleic acid against validated targets. Following its recent restructuring and suspension of clinical and development activities, the Company is exploring strategic alternatives to maximize stockholder value, both with respect to its historical biotechnology assets and more broadly. For further information, see www.exicuretx.com.

Forward-Looking Statements

This press release includes “forward-looking statements” within the meaning of the safe harbor provisions of the United States Private Securities Litigation Reform Act of 1995. There can be no assurance regarding our ability to comply with the Panel’s decision and the applicable listing criteria by the deadline or thereafter. Forward-looking statements involve known and unknown risks, uncertainties and other factors that may cause actual outcomes to differ materially from the outcomes expressed or implied by this report. Such risks include, among others, the possibility we will not be able to cure existing listing deficiencies, the possibility of additional deficiencies, the risk that the Company may not adequately comply with the terms of the Panel’s decision, and the risk that Nasdaq will ultimately delist the Company’s common stock. All such factors are difficult to predict and may be beyond the Company’s control. The Company undertakes no obligation and does not intend to update or revise any forward-looking statements contained herein, except as required by law or regulation. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date of this report.

Contacts

Josh Miller

847-673-1700

media@exicuretx.com

OS Therapies Partners with B2i Digital to Engage with Biotech Investors Online




OS Therapies Partners with B2i Digital to Engage with Biotech Investors Online

• Digital Marketing Campaign to Highlight Clinical-Stage Cancer Innovation OST-HER2 that activates cellular immunity against HER2+ cancer
• OS Therapies Joins the B2i Digital Featured Companies Program

NEW YORK–(BUSINESS WIRE)–OS Therapies, Inc. (NYSE-A: OSTX), a clinical-stage biotechnology company advancing immunotherapies and targeted drug conjugates for cancer treatment, has partnered with B2i Digital to enhance investor outreach. B2i Digital will implement data-driven strategies to raise awareness about OS Therapies’ pioneering research and upcoming milestones, including its potentially pivotal Phase 2b clinical trial for OST-HER2 in the rare pediatric orphan disease recurrent, resected metastatic osteosarcoma.

“OS Therapies is an exceptional addition to our platform,” said David Shapiro, CEO of B2i Digital. “Their innovative cancer treatments, such as OST-HER2 for recurrent, resected metastatic osteosarcoma, hold significant potential to address unmet medical needs in oncology. We look forward to helping them share their progress and vision with the investment community.”

B2i Digital will highlight OS Therapies’ lead candidate, OST-HER2, an off-the-shelf immunotherapy designed to activate the immune system to target HER2-positive cancer cells. The Phase 2b trial for OST-HER2 in recurrent, resected metastatic osteosarcoma has completed enrollment and all patient visits, with the next clinical data update expected to be announced during the week of the JP Morgan Healthcare Conference 2025. Additionally, OS Therapies is advancing its tunable Antibody-Drug Conjugate (ADC) platform, which offers tailored cancer treatments with the potential improved efficacy, and reduced side effects.

“Our collaboration with B2i Digital aligns with our mission to bring life-changing treatments to patients and increase awareness of the unique therapeutic potential of our pipeline,” said Paul Romness, MHP, CEO of OS Therapies. “We are eager to engage with investors as we prepare for key milestones that could reshape how challenging cancers, especially metastatic disease, are addressed.”

About B2i Digital, Inc.

B2i Digital, Inc. leverages the latest digital marketing technologies and investor conferences to share a company’s story with retail investors, institutional investors, and research analysts. B2i Digital creates robust profiles for companies on its platform, b2idigital.com, and launches targeted digital marketing campaigns to support their online and offline investor engagement efforts. B2i Digital was founded in 2021 by David Shapiro, previously the Chief Marketing Officer and an investment banker at Maxim Group, LLC. David was also one of the founders of Maxim’s investor awareness platform, M-Vest.com.

About OS Therapies

OS Therapies is a clinical stage oncology company focused on the identification, development, and commercialization of treatments for Osteosarcoma (OS) and other solid tumors. OST-HER2, the Company’s lead asset, is an immunotherapy leveraging the immune-stimulatory effects of Listeria bacteria to initiate a strong immune response targeting the HER2 protein. The Company has completed enrollment for a 41-patient Phase 2b clinical trial of OST-HER2 in resected, recurrent osteosarcoma, with results expected in the fourth quarter of 2024. OST-HER2 has completed a Phase 1 clinical study primarily in breast cancer patients, in addition to showing preclinical efficacy data in various models of breast cancer. OST-HER2 has been conditionally approved by the U.S. Department of Agriculture for the treatment of canines with osteosarcoma. In addition, OS Therapies is advancing its next-generation Antibody Drug Conjugate (ADC) platform, known as tunable ADC (tADC), which features tunable, tailored antibody-linker-payload candidates. This platform leverages the Company’s proprietary silicone linker technology, enabling the delivery of multiple payloads per linker. For more information, please visit www.ostherapies.com.

Forward-Looking Statements

Statements in this press release about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute forward-looking statements within the meaning of the federal securities laws. These forward-looking statements and terms such as “anticipate,” “expect,” “intend,” “may,” “will,” “should” or other comparable terms involve risks and uncertainties because they relate to events and depend on circumstances that will occur in the future. Those statements include statements regarding the intent, belief or current expectations of OS Therapies and members of its management, as well as the assumptions on which such statements are based. Prospective investors are cautioned that any such forward-looking statements are not guarantees of future performance and involve risks and uncertainties, including those described under the section entitled “Risk Factors” of our Registration Statement on Form S-1 declared effective by the Securities and Exchange Commission (the “SEC”) on July 31, 2024, as well as any of our periodic reports filed with the SEC, and that actual results may differ materially from those indicated by such forward-looking statements. Any forward-looking statements contained in this press release speak only as of the date hereof, and, except as required by the federal securities laws, OS Therapies specifically disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise.

Contacts

B2i Digital Contact Information:
David Shapiro

Chief Executive Officer

B2i Digital, Inc.

https://b2idigital.com
212.579.4844 Office

david@b2idigital.com
https://x.com/b2idigital
https://www.instagram.com/b2i_digital
https://www.facebook.com/b2idigital
https://www.linkedin.com/company/b2i-digital

OS Therapies Contact Information:
Jack Doll

410-297-7793

Irpr@ostherapies.com
https://x.com/OSTherapies
https://www.instagram.com/ostherapies/
https://www.facebook.com/OSTherapies/
https://www.linkedin.com/company/os-therapies/

Axcelead DDP Enters into Drug Discovery Service Agreement with Astellas for Targeted Protein Degraders




Axcelead DDP Enters into Drug Discovery Service Agreement with Astellas for Targeted Protein Degraders

TOKYO–(BUSINESS WIRE)–#Astellas–Axcelead Drug Discovery Partners, Inc. (HQ: Fujisawa, Kanagawa, Japan; “Axcelead DDP”) announced today that it has entered into a drug discovery service agreement with Astellas Pharma, Inc. (HQ: Chuo-Ku, Tokyo, Japan; “Astellas”) to explore new candidate compounds for targeted protein degraders.

Axcelead DDP focuses on targeted protein degradation as the next generation of small-molecule drug discovery and provides an integrated service solution called DegLead^TM Platform to solve various challenges in targeted protein degrader discovery, including synthetic technologies for rapid generation of targeted protein degraders, compound libraries, diverse high-throughput screening systems and profiling assay systems.

Under the agreement, Axcelead DDP will support Astellas’ targeted protein degrader discovery utilizing DegLead^TM Platform and contribute to the generation of breakthrough new drug candidate compounds.

“We are pleased to conclude this agreement with Astellas based on their positive evaluation of our core technology – the integrated DegLead^TM Platform solution,” said Kengo Okada, PhD., Representative Director and CEO of Axcelead DDP. “This agreement is evidence that our drug discovery solution for targeted protein degrader is a promising service that provides proprietary value to our clients. We continue to support our clients’ drug discovery by continuously evolving our pharmaceutical company-derived drug discovery platform by incorporating cutting-edge technologies.”

About Axcelead DDP

Axcelead DDP is Japan’s first drug discovery solution provider established in July 2017 by inheriting the drug discovery platform from Takeda Pharmaceutical. Screening, medicinal chemistry, pharmacology/biology, DMPK, and safety research functions are integrated into one center with a state-of-the-art research base and original compound library. Together with the most advanced AI capability integration to Axcelead DDP’s service, Axcelead DDP creates high quality drug candidates rapidly. By leveraging these strengths, Axcelead DDP provides solutions to various challenges faced in the process from exploratory research to clinical in drug discovery. For more information, please visit https://axcelead-us.com/

Contacts

Media Contact
Axcelead, Inc.

contact@axcelead-hd.com

Meiji Seika Pharma: Announcement of the Establishment of Local Subsidiary “Taiwan Meiji Pharma Co., Ltd.” in Taiwan




Meiji Seika Pharma: Announcement of the Establishment of Local Subsidiary “Taiwan Meiji Pharma Co., Ltd.” in Taiwan

TOKYO–(BUSINESS WIRE)–Meiji Seika Pharma Co., Ltd. (Headquarters: Chuo-ku, Tokyo, President: Daikichiro Kobayashi) announced the establishment of a local subsidiary, “Taiwan Meiji Pharma Co., Ltd.” (Headquarters: Taipei City, Taiwan, Chairman: Kenshi Murase), on December 18^th. This subsidiary will focus on pharmaceutical sales and other related activities in Taiwan and is scheduled to commence operations in February 2025.

1. Purpose and Background of Establishment

Meiji Seika Pharma aims to become “a leading company in Asia in the field of infectious diseases” as part of the “Meiji Group 2026 Vision” targeting the year 2026. By establishing Taiwan Meiji Pharma Co., Ltd. in Taiwan, the fifth largest pharmaceutical market in Asia, we aim to build a business foundation and achieve further growth by supplying products. Taiwan Meiji Pharma Co., Ltd. plans to launch “REZUROCK” (product name in Japan, generic name: belumosudil mesylate), a selective ROCK2 inhibitor for the treatment of chronic graft-versus-host disease (Chronic GVHD), which is currently under application for regulatory approval with the Taiwan Food and Drug Administration (TFDA). Chronic GVHD is a disease that occurs following hematopoietic stem cell transplantation performed for the treatment of leukemia.

We will also consider the continuous introduction of other products such as antibiotics and vaccines.

2. Company Overview

(1) Name: Taiwan Meiji Pharma Co., Ltd.

(2) Location: 14F, No.51 Section2 Keelung Rd. Xinyi District, Taipei city, 110502, Taiwan (R.O.C)

(3) Representative (Chairman): Kenshi Murase

(4) Business Description: Sales of pharmaceutical medicines, etc.

(5) Capital: 30 million New Taiwan Dollars

(6) Date of Establishment: December 18^th, 2024

(7) Shareholder Composition: Wholly owned subsidiary with 100% investment by Meiji Seika Pharma Co., Ltd.

Through Taiwan Meiji Pharma Co., Ltd., Meiji Seika Pharma will stably supply products needed by patients and contribute to the health of the people in Taiwan.

Contacts

For inquiries regarding this matter:

Meiji Seika Pharma Co., Ltd.

Kentarou Misaku (Taiwan Representative Office)

Phone: +88 6 911 081 507

kentarou.misaku@meiji.com

Ellipses Launches Pioneering Clinical Trial Programme in Middle East




Ellipses Launches Pioneering Clinical Trial Programme in Middle East

LONDON–(BUSINESS WIRE)–Ellipses Pharma Limited (“Ellipses”), a global drug development company focused on accelerating the development of cancer treatments through an innovative drug development model, announced today it has inaugurated the first large-scale Phase 1/2 oncology clinical study of its kind in the United Arab Emirates, marking a significant milestone in the region’s healthcare landscape.

Following approval by the UAE regulators and the Ministry and Department of Health, this study will be conducted initially at two major healthcare centres in Abu Dhabi – the Cleveland Clinic and Tawam Hospital. The inclusion of a third study centre is pending. Patient recruitment has commenced.

The trial will focus on Ellipses’ next generation selective RET inhibitor (SRI), EP0031/A400, a potential treatment of RET-altered tumours, most prevalent in non-small cell lung cancer and thyroid cancer.

Professor Sir Christopher Evans, OBE, Executive Chairman, Ellipses, said: “Our mission to find outstanding potential cancer treatments and develop them at pace has been made possible in a very large part by the unwavering support we have had from our investors in Abu Dhabi who embraced the Ellipses model and vision from its early stages.

“This tremendous commitment has led us to the point where our achievements can be brought to bear for the potential benefit of patients in UAE. In many ways, we can now bring hope and an innovative therapy to people in the UAE and also enable healthcare professionals to collaborate and share research. This trial is the first of many trials we hope to conduct in the region.”

Sir Chris is one of several global life sciences and healthcare experts on the Advisory Board of M42, the pioneering Abu Dhabi based tech enabled company, which is supporting the Ellipses initiative, and serves on the board of directors of the Emirates Drug Establishment.

The U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation to EP0031/A400 in November 2023 and in March of this year granted it Fast Track Designation, which facilitates the expedited development and review of new drugs intended to treat serious or life-threatening conditions and demonstrate the potential to address unmet medical needs.

Professor Tobias Arkenau, Global Head of Drug Development and Chief Medical Officer, Ellipses commented: “The opening of the EP0031/A400 trial in the UAE is a very exciting initiative. Through trials carried out to date, our belief has been reinforced that EP0031/A400 has the potential to be a transformational next generation RET inhibitor – in particular for patients with RET-positive NSCLC and thyroid cancers, which remain a progressive and fatal condition for hundreds of thousands of patients worldwide.”

About EP0031/A400

EP0031/A400 is being developed jointly by Ellipses in a global clinical phase 1/2 trial under the name EP0031 (NCT05443126) and Kelun Biotech (6990.HK) in China under the name A400 (NCT05265091). Modular Phase 1 studies have been conducted in the US, Europe and China and registrational Phase 2 trials in China and US, Europe and UAE are ongoing.

In March 2021, Kelun-Biotech granted Ellipses an exclusive license for EP0031/A400 in certain territories including the US and Europe, with Kelun-Biotech retaining certain rights in Greater China and certain Asian countries. An Investigational New Drug Application (IND) application for EP0031/A400 was approved by China’s National Medicinal Products Administration in June 2021 and a registrational Phase 2 trial is ongoing in China. In June 2022, the U.S. Food and Drug Administration approved the EP0031/A400 Phase 1/2 study IND application in the US.

About RET altered malignancies

Activating RET mutations and rearrangements have been identified as actionable drivers of oncogenesis in numerous tumour types and are most prevalent in non-small cell lung and thyroid cancer. It is estimated that RET mutations and rearrangements may be responsible for ~2% of all solid tumours. After the successful development of first generation SRIs and an increasing understanding of escape mechanisms to these agents, there is an unmet need to develop new treatments that can address acquired resistance, including the development of next-generation SRIs.

About Ellipses Pharma Limited

Ellipses is a global drug development company based in London, focused on accelerating the development of cancer treatments through an innovative drug development model that combines unbiased vetting to de-risk initial asset selection with an uninterrupted funding flow to minimise the time it takes to advance lead products through clinical trials and reach patients.

www.ellipses.life

About Kelun-Biotech

Kelun-Biotech (6990.HK) is a holding subsidiary of Kelun Pharmaceutical (002422.SZ), which focuses on the R&D, manufacturing, commercialization and global collaboration of innovative biological drugs and small molecule drugs. The company focuses on major disease areas such as solid tumors, autoimmune, inflammatory, and metabolic diseases, and in establishing a globalized drug development and industrialization platform to address the unmet medical needs in China and the rest of world. The Company is committed to becoming a leading global enterprise in the field of innovative drugs. At present, the Company has more than 30 ongoing key innovative drug projects, of which 1 project has been approved for marketing, 3 projects are in the NDA stage, and more than 10 projects are in the clinical stage. The company has established one of the world’s leading proprietary ADC platforms, OptiDC™, and has 1 ADC project approved for marketing, 1 ADC project in NDA stage, and multiple ADC or novel ADC projects in clinical or preclinical research stage. For more information, please visit https://kelun-biotech.com/.

Contacts

media@ellipses.life

CORRECTING and REPLACING Exicure, Inc. Announces Shareholders Approve the $8.7 Million Equity Financing and Reports Executive Management and Board Changes




CORRECTING and REPLACING Exicure, Inc. Announces Shareholders Approve the $8.7 Million Equity Financing and Reports Executive Management and Board Changes

CHICAGO–(BUSINESS WIRE)–Please replace the release dated December 20, 2024 with the following corrected version due to multiple revisions.

The updated release reads:

EXICURE, INC. ANNOUNCES SHAREHOLDERS APPROVE THE $8.7 MILLION EQUITY FINANCING AND REPORTS EXECUTIVE MANAGEMENT AND BOARD CHANGES

Exicure, Inc. (Nasdaq: XCUR, “the Company”, “Exicure”), today announced that in connection with the change of control transaction approved by the stockholders at the Special Meeting of the Stockholders on December 17, 2024 (the “Change of Control”), the stockholders of the Company approved a second investment of $8.7 million from HiTron Systems Inc. (“HiTron”), a publicly listed company (KOSPI) in South Korea. This investment is expected to close within a few days depending on regulatory approvals. Also, as of December 19, 2024, the Board of Directors (the “Board”) of the Company changed the authorized number of directors comprising the Board to nine directors and appointed four new directors to the Board and Andy Yoo as Chief Executive Officer of the Company.

The total investment from HiTron will be $10 million after the initial investment of $1.3 million closed in November. The share issuance price for HiTron is $3 per share per the common stock purchase agreement that was signed in early November, and once the second investments closes, HiTron will become Exicure’s largest shareholder, owning over 50% of the Company. On December 17, 2024, HiTron completed the acquisition of Exicure’s management rights through the appointment of board members.

As disclosed in the 8-K filed on December 9, 2024, the Company Exicure secured $2 million through a share issuance from institutional investors. The Company plans to raise approximately $2.4 million in the near term through further capital increases. The total amount raised is expected to be around $15 million USD.

With these additional investments, the Company is positioned to focus on developing its core pipeline and pursuing strategic transactions. The Company is in discussions with a US therapeutics company and expects to provide an additional update in January.

As previously disclosed, on June 20, 2024, the Company was notified by The Nasdaq Stock Market LLC (“Nasdaq”) that the Company no longer satisfied the minimum $2.5 million stockholders’ equity requirement for continued listing on The Nasdaq Capital Market (the “Equity Requirement”). On December 17, 2024, the Company presented its plan to regain compliance with the Equity Requirement to a Nasdaq Hearings Panel (the “Panel”). With these recent investments, the Company believes that the funds raised will allow the Company to meet the requirements for Nasdaq continued listing and awaits a decision from the Panel.

About Exicure, Inc.

Exicure, Inc. has historically been an early-stage biotechnology company focused on developing nucleic acid therapies targeting ribonucleic acid against validated targets. Following its recent restructuring and suspension of clinical and development activities, the Company is exploring strategic alternatives to maximize stockholder value, both with respect to its historical biotechnology assets and more broadly. For further information, see https://investors.exicuretx.com/overview/default.aspx.

Forward-Looking Statements

This press release includes “forward-looking statements” within the meaning of the safe harbor provisions of the United States Private Securities Litigation Reform Act of 1995. There can be no assurance regarding our ability to comply with the Panel’s decision and the applicable listing criteria by the deadline or thereafter. Forward-looking statements involve known and unknown risks, uncertainties and other factors that may cause actual outcomes to differ materially from the outcomes expressed or implied by this report. Such risks include, among others, the possibility we will not be able to cure existing listing deficiencies, the possibility of additional deficiencies, the risk that the Company may not adequately comply with the terms of the Panel’s decision, and the risk that Nasdaq will ultimately delist the Company’s common stock. All such factors are difficult to predict and may be beyond the Company’s control. The Company undertakes no obligation and does not intend to update or revise any forward-looking statements contained herein, except as required by law or regulation. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date of this report.

Contacts

Media Contact:
Josh Miller

847-673-1700

media@exicuretx.com

PureTech’s Deupirfenidone (LYT-100) Slowed Lung Function Decline in People with Idiopathic Pulmonary Fibrosis (IPF) as Measured by Forced Vital Capacity (FVC), Achieving the Primary and Key Secondary Endpoints in the ELEVATE IPF Phase 2b Trial




PureTech’s Deupirfenidone (LYT-100) Slowed Lung Function Decline in People with Idiopathic Pulmonary Fibrosis (IPF) as Measured by Forced Vital Capacity (FVC), Achieving the Primary and Key Secondary Endpoints in the ELEVATE IPF Phase 2b Trial

Dose-ranging trial evaluated deupirfenidone 550 mg three times a day (TID) (approximately equivalent exposure to pirfenidone 801 mg TID¹) and deupirfenidone 825 mg TID and successfully demonstrated dose-dependent response

Decline in lung function seen with deupirfenidone 825 mg TID as a monotherapy was -21.5 mL; natural lung function decline expected in healthy adults >60 years is ~-15 to ~-25 mL²; decline in lung function with pirfenidone 801 mg TID was -51.6 mL

Deupirfenidone 825 mg TID showed strong, consistent and durable efficacy with a treatment effect versus placebo of 80.9% with favorable tolerability, while pirfenidone 801 mg TID had a 54.1% treatment effect versus placebo

Deupirfenidone was generally well-tolerated with a favorable adverse event profile at both doses studied

Data support continued development of deupirfenidone and highlight its potential to serve as a new standard-of-care treatment for IPF

Company to host a webcast and conference call today at 9:00am EST / 2:00pm GMT

BOSTON–(BUSINESS WIRE)–PureTech Health plc (Nasdaq: PRTC, LSE: PRTC) (“PureTech” or the “Company”), a clinical-stage biotherapeutics company dedicated to changing the lives of patients with devastating diseases, today announced positive results from ELEVATE IPF, a Phase 2b randomized, double-blind, active- and placebo-controlled, dose-ranging trial evaluating deupirfenidone (LYT-100) at two dose levels three times a day (TID) over 26 weeks in patients with idiopathic pulmonary fibrosis (IPF).

“The ELEVATE IPF trial broke new ground in Phase 2 trial design in IPF; this was the first time that a new therapy (deupirfenidone) has been evaluated alongside one of the two existing standard-of-care treatments (pirfenidone),” said Toby Maher, M.D., Ph.D., Professor of Medicine and Director of Interstitial Lung Disease at Keck School of Medicine, University of Southern California, Los Angeles, and lead investigator in the ELEVATE IPF trial. “Deupirfenidone 825 mg TID reduced lung function decline to near-physiologic levels over 26 weeks and had an effect size, compared with placebo, that was approximately 50% greater than that seen with pirfenidone. Deupirfenidone has the potential to offer patients a highly effective and tolerable treatment option. These are extremely exciting results from a Phase 2b trial, and I am very enthusiastic about the continued development of deupirfenidone.”

Participants in the trial were randomized 1:1:1:1 to receive deupirfenidone 550 mg, deupirfenidone 825 mg, pirfenidone 801 mg (the FDA-approved dose), or placebo TID for 26 weeks, and had the option to enroll in an ongoing, open-label extension study. The two doses of deupirfenidone were chosen based on PureTech’s Phase 1 data, which showed that a 550 mg TID dose of deupirfenidone provided approximately equivalent drug exposure to pirfenidone, 801 mg TID.¹

The trial achieved its primary endpoint based on the prespecified Bayesian analysis, with a 98.5% posterior probability. This means there is a 98.5% probability that the pooled deupirfenidone arms were superior to placebo in slowing the rate of lung function decline in people with IPF, as measured by forced vital capacity (FVC) at 26 weeks. The trial also successfully demonstrated a dose-dependent response.

The rate of FVC decline³ at week 26 with:

• deupirfenidone 825 mg TID compared to placebo was statistically significant (-21.5 mL vs. -112.5 mL, respectively; p=0.02)⁴ and represents a robust treatment effect of 80.9% as a monotherapy; for context, the level of six-month natural decline in lung function as measured by FVC expected in healthy adults over 60 years old is approximately -15.0 mL to -25.0 mL. ²
• pirfenidone 801 mg TID showed a treatment effect of 54.1% compared to placebo (-51.6 mL vs. -112.5 mL, respectively), which is consistent with previously reported pirfenidone clinical trial data.⁵

The trial also achieved its key secondary endpoint based on a prespecified Bayesian analysis, with a posterior probability of 99.6%. This means that there is a 99.6% probability that the pooled deupirfenidone arms were superior to placebo in slowing the rate of lung function decline in people with IPF, as measured by the forced vital capacity percent predicted (FVCpp) from baseline to week 26. While FVCpp and FVC (the primary endpoint) are both measures of lung function, FVCpp accounts for key patient characteristics (age, sex, height, race) and therefore normalizes the results at the patient level. Deupirfenidone 825 mg TID also demonstrated a benefit on this endpoint compared to placebo that was statistically significant (-0.43 vs. -3.43, respectively; p=0.01),^3,4 reinforcing the robustness of the treatment’s impact.

“Our goal in developing deupirfenidone is to offer better outcomes to people living with IPF. The adoption and adherence of currently approved antifibrotics has been limited by a tradeoff between efficacy and tolerability, specifically related to gastrointestinal adverse events. This prevents many patients from initiating treatment or maintaining optimal therapeutic doses and, in turn, achieving the best possible outcomes,” said Eric Elenko, Ph.D., President and Co-founder of PureTech. “I could not be more pleased that deupirfenidone showed a favorable tolerability profile at both doses evaluated and – most importantly – has demonstrated the potential to offer patients enhanced efficacy at the higher dose.”

Both doses of deupirfenidone were generally well-tolerated in the trial. The overall number of patients experiencing any gastrointestinal (GI)-related adverse events (AEs) was similar across the deupirfenidone 825 mg TID and pirfenidone 801 mg TID arms. Deupirfenidone 825 mg TID demonstrated a favorable tolerability profile compared to pirfenidone 801 mg TID, with a lower percentage of patients reporting key GI AEs that occurred in ≥5% of participants in at least one arm: nausea (20.3% vs. 27.0%), dyspepsia (14.1% vs. 22.2%), diarrhea (7.8% vs. 11.1%), constipation (4.7% vs. 6.3%) and vomiting (1.6% vs. 3.2%). The only key GI AE increase observed was abdominal pain (14.1% vs. 7.9%). There were five deaths in the pirfenidone arm, two deaths in the placebo arm and one death in each of the deupirfenidone arms. None of the deaths was deemed to be treatment related.

Overall, 187 out of 257 patients completed the trial: 43 out of 63 patients in the pirfenidone 801 mg TID arm; 42 out of 65 patients in the deupirfenidone 550 mg TID arm; 50 out of 64 patients in the deupirfenidone 825 mg TID arm; and 52 of 65 patients in the placebo arm.

Of those who completed the trial, 170 patients (more than 90%) opted to enroll in an ongoing open-label extension (OLE) evaluating the two doses of deupirfenidone. To date, preliminary data support a durable treatment effect and a consistent tolerability profile with deupirfenidone 825 mg. Across the randomized trial and OLE, the longest treatment duration with deupirfenidone 825 mg TID is 79 weeks and with deupirfenidone 550 mg TID is 81 weeks.

“These data are remarkable, particularly for a monotherapy, and – if supported by a Phase 3 trial – would represent a step change in the treatment of IPF,” said Bharatt Chowrira, Ph.D., J.D., CEO of PureTech. “At PureTech, our approach is centered on identifying simple and elegant solutions to big problems that underlie tremendous patient need, and we are proud that our R&D engine has generated another potentially transformative treatment. We are committed to the rapid advancement of deupirfenidone, with the goal of delivering a new standard of care to patients while generating value for our shareholders. On behalf of the entire PureTech team, I extend my sincere gratitude to the people living with IPF, their caregivers, the clinical trial investigators and advocacy groups as well as our talented team for supporting this mission.”

PureTech is committed to continuing development of deupirfenidone and intends to discuss the Phase 2b results with regulatory authorities to align on the appropriate path forward. Additional data from this trial will be presented at a future forum.

Webcast and Conference Call Details

Members of the PureTech management team will host a conference call at 9:00am EST / 2:00pm GMT today, December 16, 2024, to discuss these results. A live webcast and presentation slides will be available on the investors section of PureTech’s website under the Events and Presentations tab. To join by phone, please dial:

United Kingdom (Local): +44 20 3936 2999

United Kingdom (Toll-Free): +44 800 358 1035

United States (Local): +1 646 787 9445

United States (Toll-Free): +1 855 9796 654

International: +44 20 3936 2999

Access Code: 860033

For those unable to listen to the call live, a replay will be available on the PureTech website.

About the ELEVATE IPF Trial

The Phase 2b ELEVATE IPF trial was a randomized, double-blind, active- and placebo-controlled, dose-ranging trial designed to evaluate the efficacy, tolerability, safety and dosing regimen of deupirfenidone (LYT-100) in patients with IPF compared to placebo. 257 participants were randomized in a ratio of 1:1:1:1 to receive either 550 mg of deupirfenidone, 825 mg of deupirfenidone, 801 mg pirfenidone or placebo three times a day (TID) for 26 weeks. Participants who completed the trial had the option to enroll in an open-label extension, which is ongoing.

The primary endpoint of the trial was the rate of decline in Forced Vital Capacity (FVC) for the combined deupirfenidone arms versus placebo over the 26-week treatment period. FVC is a measure of the maximum amount of air (in mL) that an individual can forcibly exhale after fully inhaling. It is a standard measurement in clinical trials for IPF and is used to assess disease progression as well as to predict mortality.

A prespecified Bayesian analysis was utilized to assess the primary endpoint and provided a posterior probability, which is the probability of a positive treatment difference for deupirfenidone compared to placebo. This also allowed for augmentation of the placebo arm with placebo data from historical IPF trials. This approach enabled a more patient-centric clinical trial design by minimizing the number of trial participants exposed to placebo—a key consideration since IPF is progressive and fatal—while delivering a robust, placebo-controlled dataset.

About Deupirfenidone

Deupirfenidone is a deuterated form of pirfenidone, which is one of the two standard-of-care treatments approved to treat IPF, in addition to nintedanib. Deuteration is intended to make deupirfenidone break down more slowly in the body than pirfenidone.

About Idiopathic Pulmonary Fibrosis (IPF)

IPF is a rare, progressive and fatal lung disease with a median survival of 2-5 years.⁶ Pirfenidone is one of only two drugs approved to treat IPF, and for those patients able to tolerate treatment, it has been shown to improve survival by approximately 2.5 years compared to supportive care alone.⁶ However, tolerability issues with both of the standard-of-care medications result in patients discontinuing treatment or reducing their dose. This contributes to nearly three out of every four people with IPF in the US not receiving treatment with these otherwise efficacious medicines.⁷ There are over 232,000 people in the US and the EU5 countries (Italy, Spain, France, Germany and the United Kingdom) living with IPF.⁸

About PureTech Health

PureTech is a clinical-stage biotherapeutics company dedicated to giving life to new classes of medicine to change the lives of patients with devastating diseases. The Company has created a broad and deep pipeline through its experienced research and development team and its extensive network of scientists, clinicians and industry leaders that is being advanced both internally and through its Founded Entities. PureTech’s R&D engine has resulted in the development of 29 therapeutics and therapeutic candidates, including three that have been approved by the U.S. Food and Drug Administration. A number of these programs are being advanced by PureTech or its Founded Entities in various indications and stages of clinical development, including registration-enabling studies. All of the underlying programs and platforms that resulted in this pipeline of therapeutic candidates were initially identified or discovered and then advanced by the PureTech team through key validation points.

For more information, visit www.puretechhealth.com or connect with us on X (formerly Twitter) @puretechh.

Cautionary Note Regarding Forward-Looking Statements

This press release contains statements that are or may be forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation those related to the LYT-100 development program and development plans, and its potential benefits to patients, plans for discussions with regulatory authorities, the further development of the program, future presentation of additional data from the trial and our future prospects, developments and strategies. The forward-looking statements are based on current expectations and are subject to known and unknown risks, uncertainties and other important factors that could cause actual results, performance and achievements to differ materially from current expectations, including, but not limited to, those risks, uncertainties and other important factors described under the caption “Risk Factors” in our Annual Report on Form 20-F for the year ended December 31, 2023, filed with the SEC and in our other regulatory filings. These forward-looking statements are based on assumptions regarding the present and future business strategies of the Company and the environment in which it will operate in the future. Each forward-looking statement speaks only as at the date of this press release. Except as required by law and regulatory requirements, we disclaim any obligation to update or revise these forward-looking statements, whether as a result of new information, future events or otherwise.

The information contained within this announcement is deemed by the Company to constitute inside information as stipulated under the Market Abuse Regulations (EU) No. 596/2014 which forms part of UK domestic law by virtue of the European Union (Withdrawal) Act 2018 (‘MAR’). Upon the publication of this announcement via a Regulatory Information Service (‘RIS’), this inside information is now considered to be in the public domain.

<sup>_______________________

1</sup> Maher, T., Chen, M., Korth, C., Elenko, E., Harnett, M., Garg, V., Graham, C., Fares, W., Krop, J. (2023). Deupirfenidone (LYT-100) dose-selection rationale for a Phase 2b idiopathic pulmonary fibrosis study — ELEVATE IPF. Poster presented at the CHEST Annual Meeting, Honolulu, HI.

² FVC decline at 6 months was estimated assuming linear decline over time. Valenzuela, C., Bonella, F., Moor, C., Weimann, G., Miede, C., Stowasser, S., Maher, T. (2024). Decline in forced vital capacity (FVC) in subjects with idiopathic pulmonary fibrosis (IPF) and progressive pulmonary fibrosis (PPF) compared with healthy references. Poster presented at the European Respiratory Society International Congress, Vienna, Austria; and Luoto, J., Pihlsgård, M., Wollmer, P., & Elmståhl, S. (2019). Relative and absolute lung function change in a general population aged 60-102 years. The European Respiratory Journal, 53(3), 1701812. https://doi.org/10.1183/13993003.01812-2017
³ Efficacy analyses used a random coefficient regression model with absolute FVC or FVCpp including baseline as response variable and week, treatment and interaction between week and treatment as fixed effect. The analyses were performed based on the predefined Full Analysis Set.

⁴ All p values are two-sided and have not been corrected for multiplicity.

⁵ Roche. (2014). Esbriet^® (pirfenidone) prescribing information. Retrieved from https://www.accessdata.fda.gov/drugsatfda_docs/label/2014/022535s000lbl.pdf
⁶ Fisher M, Nathan SD, Hill C, et al. Predicting Life Expectancy for Pirfenidone in Idiopathic Pulmonary Fibrosis. J Manag Care Spec Pharm. 2017;23(3-b Suppl):S17-S24. doi:10.18553/jmcp.2017.23.3-b.s17

⁷ Dempsey, T. M., Payne, S., Sangaralingham, L., Yao, X., Shah, N. D., & Limper, A. H. (2021). Adoption of the Antifibrotic Medications Pirfenidone and Nintedanib for Patients with Idiopathic Pulmonary Fibrosis. Annals of the American Thoracic Society, 18(7), 1121–1128. https://doi.org/10.1513/AnnalsATS.202007-901OC
⁸ GlobalData Epidemiology and Market Size Search, EU5=United Kingdom, France, Germany, Italy and Spain

THIS ANNOUNCEMENT CONTAINS INSIDE INFORMATION FOR THE PURPOSES OF ARTICLE 7 OF THE UK VERSION OF THE MARKET ABUSE REGULATION (EU 596/ 2014) AS IT FORMS PART OF UK LAW BY VIRTUE OF THE EUROPEAN UNION (WITHDRAWAL) ACT 2018, AS AMENDED

Contacts

PureTech
Public Relations

publicrelations@puretechhealth.com
Investor Relations

IR@puretechhealth.com

UK/EU Media
Ben Atwell, Rob Winder

+44 (0) 20 3727 1000

puretech@fticonsulting.com

US Media
Justin Chen

+1-609-578-7230

jchen@tenbridgecommunications.com

ViroCell Biologics Expands U.S. Business Development Team With Two New Appointments




ViroCell Biologics Expands U.S. Business Development Team With Two New Appointments

Meredith Brown and Elaine Rihn bring strong commercial expertise in cell and gene therapy to roles focused on driving ViroCell’s continued growth

LONDON & NEW YORK–(BUSINESS WIRE)–ViroCell Biologics (“ViroCell” or the “Company”), a cell and gene therapy (“CGT”) contract development and manufacturing organization (“CDMO”) specializing in GMP viral vector manufacturing for clinical trials, announces the expansion of the Company’s U.S. business development team with the appointments of Meredith Brown, as Senior Director, and Elaine Rihn as Director of Business Development. Both join from Lonza Inc., a global CDMO, where their roles focused on generating and expanding commercial contracts with clients in CGT manufacturing. Meredith and Elaine will focus on growing ViroCell’s U.S. market share and report to Traci Kyes, ViroCell’s Vice President of Business Development.

John W. Hadden II, CEO at ViroCell, commented: “We are delighted to appoint two highly talented and experienced professionals to further accelerate ViroCell’s strong commercial traction in the vector market. Team ViroCell extends a warm welcome to Meredith and Elaine, who have proven track records of delivering commercial results in cell and gene therapy manufacturing, to bring ViroCell’s unique viral vector design, de-risking, and high titer GMP offering to more innovators across the U.S.”

Meredith Brown

Meredith Brown has a wealth of expertise in business development, executive leadership, and venture capital strategy in the biotech industry. Most recently, Meredith was Director of Business Development and Account Management at Lonza, Inc. She facilitated the company’s commercial activities by negotiating contracts for CGT process development and GMP manufacturing across all clinical stages. Before joining Lonza in 2019, Meredith was founder and CEO of Gravitas Biomanufacturing, established to provide specialized manufacturing to early-stage CGT companies requiring clinical grade products. Meredith had also launched another start-up company to provide vectors for cell therapy, gene therapy and DNA vaccines.

Elaine Rihn

Elaine brings over 15 years of CGT commercial development and customer relationship experience to ViroCell, as the Company seeks to capture greater U.S. market share. Elaine was most recently Director of Business Development, Cell and Gene Technology at Lonza Inc., where she focused on technology capabilities in gene, viral and cell therapies, collaborating with potential customers to offer clinical phase service solutions. Prior to this, Elaine worked at Gamida Cell Ltd. as a Director of Commercial Readiness and Supply Chain Operations in the U.S. portfolio from 2020. Before this, Elaine spent 11 years as Senior Program Manager and Global Lead in Cell and Gene Therapy at Lonza Walkersville Inc.

Notes to editor:

ViroCell

ViroCell Biologics is an innovation-driven Contract Development and Manufacturing Organization (“CDMO”) focused exclusively on the design, derisking, and GMP manufacture of viral vectors for clinical trials. Built around one of the most prolific academic viral vector manufacturing teams, ViroCell was created to address the global demand for precisely engineered viral vectors. The team leverages its deep track record to help clients to de-risk and accelerate novel cell and gene therapies into and through clinical development, with a mission of being the partner of choice for corporate and academic innovators. Focused initially on manufacturing lentivirus and gamma-retrovirus vectors, ViroCell enables clients to start clinical trials on a scalable platform, delivering value by reducing costs, time and regulatory risk.

www.virocell.com

Contacts

For more ViroCell information, please contact:

ViroCell
John W. Hadden II, CEO

info@virocell.com

For ViroCell media enquiries, please contact:

FTI Consulting
Simon Conway / Victoria Foster Mitchell / Tim Stamper

ViroCell@fticonsulting.com
+44 (0)20 3727 1000

Celltrion Receives Positive CHMP Opinion for Three Biosimilars in the European Union




Celltrion Receives Positive CHMP Opinion for Three Biosimilars in the European Union

• The Committee for Medicinal Products for Human Use (CHMP) adopts positive opinions for Celltrion’s three biosimilar candidates – Eydenzelt^® (aflibercept), Stoboclo^® and Osenvelt^® (denosumab), and Avtozma^® (tocilizumab)
• Celltrion is committed to expanding access to biologic treatments in skeletal-related disorders, ophthalmology, and immunology

INCHEON, South Korea–(BUSINESS WIRE)–Celltrion today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted positive opinions and recommended marketing authorisations for three biosimilar candidates: Eydenzelt^® (CT-P42, aflibercept), Stoboclo^® and Osenvelt^® (CT-P41, denosumab), and Avtozma^® (CT-P47, tocilizumab). This significant milestone reflects the company’s leadership in biosimilar innovation and commitment to expanding access to biologic treatments across Europe.

Eydenzelt (40 mg/mL solution for injection in vial and pre-filled syringe), a biosimilar to Eylea^® (aflibercept), is recommended for approval to treat multiple retinal disorders, including neovascular (wet) age-related macular degeneration (AMD), macular edema following retinal vein occlusion (RVO, branch RVO or central RVO), diabetic macular edema (DME) and myopic choroidal neovascularisation (myopic CNV). In a Phase III study of Eydenzelt, the efficacy, safety, pharmacokinetics and immunogenicity of Eydenzelt was compared to Eylea in patients with diabetic macular edema (DME), demonstrating therapeutic equivalence to Eylea by meeting the predefined equivalence criteria.¹ If marketing authorisation is granted by the European Commission (EC), Eydenzelt would be one of the first-wave aflibercept biosimilars in Europe.

Stoboclo (60 mg solution for injection in pre-filled syringe) and Osenvelt (120 mg solution for injection in vial) have been recommended for approval for all indications of the reference products Prolia^® and Xgeva^®, respectively. The positive CHMP opinion was based on the totality of evidence including results from a Phase III clinical trial in postmenopausal osteoporosis (PMO), and the results showed that CT-P41 had equivalent efficacy and pharmacodynamics (PD) to reference denosumab, with similar pharmacokinetics (PK) and comparable safety and immunogenicity profiles.²

Avtozma, a biosimilar referencing RoActemra^® (tocilizumab), has been recommended for all indications of its reference product, including moderate to severely active rheumatoid arthritis (RA), active systemic juvenile idiopathic arthritis (sJIA), juvenile idiopathic polyarthritis (pJIA) and giant cell arteritis (GCA). The positive CHMP opinion for Avtozma was supported by a comprehensive data package and totality of evidence demonstrating Avtozma’s biosimilarity to RoActemra, with no clinically meaningful differences in efficacy, PK equivalence, safety or immunogenicity.^3,4

“With CHMP approvals for Eydenzelt, Avtozma, and Prolia/Xgeva biosimilars, Celltrion solidifies its leadership in the European biosimilar market, offering one of the most extensive antibody biosimilar portfolios. Our vertically integrated model ensures supply chain stability while addressing the specific challenges faced by European healthcare professionals and patients. These approvals underscore our commitment to supporting European healthcare systems by improving access to high-quality, affordable treatments,” said Taehun Ha, Vice President and Head of Europe. “Our focus remains on empowering clinicians with the tools and solutions they need, as we aim to transition from a pioneer to a frontier leader in European healthcare.”

The CHMP’s recommendations will now be referred to the EC, which will decide whether to grant a marketing authorisation for Eydenzelt, Stoboclo and Osenvelt, and Avtozma. If marketing authorisations are granted, the three biosimilars will be made available across EU member states, furthering Celltrion’s commitment to delivering innovative and accessible healthcare solutions.

About CT-P41 Phase III Clinical Trial

The Phase III study randomised 479 patients to receive 60 mg of CT-P41 or reference product every six months (Weeks 0 and 26; treatment period [TP] I). Before dosing at Week 52, patients initially assigned to reference product in TP I were re-randomised in a 1:1 ratio to continue with the reference product or switch to CT-P41 in TP. All patients initially randomly assigned to CT-P41 in TP I continued their treatment with CT-P41 in TP II. In TP II, 422 patients were randomised in Week 52 (CT-P41 maintenance group: 221, reference product maintenance group: 100, switched to CT-P41 group: 101). The primary efficacy endpoint was met in terms of percent change from baseline in bone mineral density (BMD) for lumbar spine (L1-L4) by dual-energy X-ray absorptiometry (DXA) at Week 52, and the primary pharmacodynamics (PD) endpoint was met in terms of area under the effect curve (AUEC) of serum carboxy-terminal cross-linking telopeptide of type I collagen (s-CTX) over the initial six months. Results of the study showed that CT-P41 had equivalent efficacy and PD to reference denosumab, with similar PK and comparable safety and immunogenicity profiles.²

About CT-P42 Phase III Clinical Trial

In a randomised, double-masked, parallel-group, multi-centre Phase III study of Eydenzelt^® (CT-P42), the efficacy, safety, pharmacokinetics, usability and immunogenicity of Eydenzelt was compared to Eylea^® (aflibercept) in patients with diabetic macular edema (DME). The primary endpoint was the change from baseline in best corrected visual acuity (BCVA) measured at Week 8, comparing Eydenzelt and Eylea. Results of the study showed that Eydenzelt met the predefined equivalence criteria, and secondary endpoints of efficacy, safety, and immunogenicity also showed trends similar to Eylea.^1,5

About CT-P47 Phase III Clinical Trial

This was a Phase III, randomised, active-controlled, double-blind trial to compare the efficacy and safety of Avtozma^® (CT-P47) and RoAtemra^® (tocilizumab) in patients with moderate to severely active rheumatoid arthritis (RA). The Phase III study randomised 479 patients to receive 8mg/kg of CT-P47 or reference tocilizumab every 4 weeks (Week 0 and 24; treatment period [TP] I). Before dosing at Week 24, patients initially assigned to tocilizumab in TP I were re-randomised in a 1:1 ratio to continue with tocilizumab or switch to CT-P47 in TP II up to Week 52. In TP II, 444 patients were randomised in Week 24 (CT-P47 maintenance group: 225, tocilizumab maintenance group: 109, switched to CT-P47 group: 110). The primary endpoint was mean change from baseline in Disease Activity score in 28 joints (DAS28; erythrocyte sedimentation rate [ESR]) at week 12. Efficacy equivalence was determined if confidence intervals (CIs) for the treatment difference was within the predefined equivalence margin: (95% CI: -0.6, +0.6 (analysis of covariance [ANCOVA]) at week 12). Therapeutic equivalence of CT-P47 and reference tocilizumab in treating RA was demonstrated and supported by comparable and sustained efficacy results up to Week 52. CT-P47 was also well tolerated with a safety profile comparable to reference tocilizumab, and no notable safety issue was identified following the single transition from reference tocilizumab to CT-P47 compared with maintenance groups up to Week 52. ^3,4

About Stoboclo^® (CT-P41, biosimilar candidate of denosumab)

Stoboclo^® (denosumab), a receptor activator of NF-κb ligand (RANKL) inhibitor, is a treatment developed as a biosimilar to reference product Prolia^® (denosumab). In Europe, Stoboclo has been recommended for approval to treat osteoporosis in postmenopausal women and in men at increased risk of fractures, bone loss associated with hormone ablation in men with prostate cancer at increased risk of fractures, and bone loss associated with long-term systemic glucocorticoid therapy in adult patients at increased risk of fracture.

About Osenvelt^® (CT-P41, biosimilar candidate of denosumab)

Osenvelt^® (denosumab) is a receptor activator of NF-κb ligand (RANKL) inhibitor developed as a biosimilar referencing Xgeva^® (denosumab). Osenvelt has been recommended for approval in Europe to prevent skeletal-related events in adults with advanced malignancies involving bone, and to treat adults and skeletally mature adolescents with giant cell tumour of bone that is unresectable or where surgical resection is likely to result in severe morbidity.

About Eydenzelt^® (CT-P42, biosimilar candidate of aflibercept)

Eydenzelt^® (aflibercept) is a vascular endothelial growth factor (VEGF) inhibitor referencing Eylea^®. Based on comprehensive data from a Phase III clinical trial confirming therapeutic equivalence to Eylea¹, Eydenzelt was filed for regulatory approval with the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) in June and November 2023, respectively.

About Avtozma^® (CT-P47, biosimilar candidate of tocilizumab)

CT-P47, containing the active ingredient tocilizumab, is a recombinant humanised monoclonal antibody that acts as an interleukin 6 (IL-6) receptor antagonist. Based on data from the global Phase III clinical trial, designed to evaluate the efficacy, pharmacokinetics (PK), safety, and immunogenicity of CT-P47 compared to the reference product RoActemra^®4, CT-P47 was filed for regulatory approval with the U.S. FDA and EMA in January and February 2024, respectively.

About Celltrion

Celltrion is a leading biopharmaceutical company based in Incheon, South Korea that specialises in researching, developing, manufacturing, marketing and sales of innovative therapeutics that improve people’s lives worldwide. The company’s solutions include world-class monoclonal antibody biosimilars such as Remsima^®, Truxima^® and Herzuma^®, providing broader patient access globally. Celltrion has also received U.S. FDA and EC approval for Vegzelma^® and Yuflyma^®, FDA approval for Zymfentra^®, and EC approval for Remsima^® SC, Omlyclo^®, SteQeyma^®. To learn more, please visit www.celltrion.com/en-us.

FORWARD-LOOKING STATEMENT

Certain information set forth in this press release contains statements related to our future business, and financial performance and future events or developments involving Celltrion Inc. and its subsidiaries that may constitute forward-looking statements, under pertinent securities laws.

These statements may be identified by words such as “prepares,” “hopes to,” “upcoming,” ”plans to,” “aims to,” “to be launched,” “is preparing,” “once gained,” “could,” “with the aim of,” “may,” “once identified,” “will,” “working towards,” “is due,” “become available,” “has potential to,” the negative of these words or such other variations thereon or comparable terminology.

In addition, our representatives may make oral forward-looking statements. Such statements are based on the current expectations and certain assumptions of Celltrion Inc. and its subsidiaries’ management, of which many are beyond its control.

Forward-looking statements are provided to allow potential investors the opportunity to understand management’s beliefs and opinions in respect of the future so that they may use such beliefs and opinions as one factor in evaluating an investment. These statements are not guarantees of future performance and undue reliance should not be placed on them.

Such forward-looking statements necessarily involve known and unknown risks and uncertainties, which may cause actual performance and financial results in future periods to differ materially from any projections of future performance or results expressed or implied by such forward-looking statements.

Celltrion Inc. and its subsidiaries undertake no obligation to update forward-looking statements if circumstances or management’s estimates or opinions should change except as required by applicable securities laws.

Trademark

Stoboclo^® and Osenvelt^® are registered trademarks of Celltrion Inc. 

Prolia^® and Xgeva^® are registered trademarks of Amgen Inc.

Eydenzelt^® is a registered trademark of Celltrion Inc. 

Eylea^® is a registered trademark of Bayer AG.

Avtozma^® is a registered trademark of Celltrion, Inc., used under license. 

RoActemra^® is a registered trademark of Chugai Pharmaceutical Co., Ltd.

References

¹ Sebastian Wolf et al., Long-term efficacy and Safety of CT-P42 compared to Reference Aflibercept in Diabetic Macular Edema: 52-Week Results from the Phase 3 CT-P42 3.1. [EURETINA 2024, Abstract #CA24-2257-8397]. Available at: https://abstracts.euretina.org/2024/ca24-2257-8397/r/recxUD7DqYfFfjC7s [Last accessed December 2024]. 

² Reginster JY et al. Efficacy and safety of candidate biosimilar CT-P41 versus reference denosumab: a double-blind, randomized, active-controlled, Phase 3 trial in postmenopausal women with osteoporosis. Osteoporos Int. 2024 Nov;35(11):1919-1930. doi: 10.1007/s00198-024-07161-x. Epub 2024 Jul 23. PMID: 39042292; PMCID: PMC11499533. Available at: https://pubmed.ncbi.nlm.nih.gov/39042292/ [Last accessed December 2024]. 

³ Smolen JS et al., Efficacy and safety of CT-P47 versus reference tocilizumab: 32-week results of a randomised, active-controlled, double-blind, phase III study in patients with rheumatoid arthritis, including 8 weeks of switching data from reference tocilizumab to CT-P47. RMD Open. 2024;10(4), e004514. Available at: https://rmdopen.bmj.com/content/10/4/e004514.abstract [Last accessed December 2024]. 

⁴ Gerd Burmester et al., Similar Efficacy, PK, Safety, and Immunogenicity of Tocilizumab Biosimilar (CT-P47) and Reference Tocilizumab in Patients with Moderate-to-Severe Active Rheumatoid Arthritis: Week 52 Results from the Phase III Single Transition Study. Poster Presentation (abstract no. 0502). Presented at ACR 2024. Available at: https://acrabstracts.org/abstract/similar-efficacy-pk-safety-and-immunogenicity-of-tocilizumab-biosimilar-ct-p47-and-reference-tocilizumab-in-patients-with-moderate-to-severe-active-rheumatoid-arthritis-week-52-results-from-the/ [Last accessed December 2024]. 

⁵ Sebastian Wolf et al., Biosimilar Candidate CT-P42 in Diabetic Macular Edema: 24-Week Results from a Randomized, Active-Controlled, Phase III Study. Available at: https://www.sciencedirect.com/science/article/pii/S2468653024003063 [Last accessed December 2024].

Contacts

Donna Gandhi

dgandhi@hanovercomms.com
+44 (0) 203 817 6591