Kyowa Kirin Co., Ltd.

Japan's Kirin Holdings has amassed enough equity to complete its takeover of health supplement maker Fancl, the company's new president has announced. The move will give Kirin the upper hand in a bid with an overseas fund and further boost the company's transformation into the healthcare sector. Mr Takeshi Tanaka, who took the company's top job in March this year, expressed expectations of declaring victory when the takeover bid closes on Wednesday. However, due to Mitsubishi Motors' acquisition of Kirin, financial regulators had to postpone the offer until September 11. Alpha Management has raised its stake in the French bank to about 10 per cent.   As of August 29, 2023, the market value of Fancl has climbed to 364.1 billion yen, equivalent to about $2.52 billion. Mr. Takeshi Tanaka previously held leadership positions in Kirin Pharmaceuticals, a subsidiary of Kirin Pharmaceuticals, and Blackmores Limited, an internationally renowned vitamin manufacturer, which was successfully acquired in a transaction of US $1.2 billion during the year. Mr. Takeshi Tanaka's joining fully reflects our company's determination and actions to unswervingly promote business transformation and upgrading in the face of the severe challenges of the Japanese market contraction and the change of international consumer preferences.   The Fancl brand, known worldwide for its outstanding cleansing oils and nutritional supplements, is a good fit with Kirin's Health Sciences product line, which the Kirin Group is aiming to build into a new pillar industry alongside the alcohol and pharmaceutical businesses. The Group's strategic goal is to achieve annual revenue of 500 billion yen in this business segment, a five-fold increase compared to the previous year. However, Takeshi Tanaka, a senior executive of Kirin Group, made it clear that internal growth alone would be difficult to achieve this ambitious goal, and the group may need to accelerate its development through overseas acquisitions. "Our strategic focus is on potential partners with unique technologies, products and brands," he stressed. "Under the guidance of this strategy, the North American market, with its continued growth momentum and great potential, is the focus of our attention." Since joining Kirin Group in 1984, Takeshi Tanaka has witnessed the company's successful entry into the pharmaceutical sector with its advanced fermentation technology. He acknowledged that it would take time for the brand's image to change in the minds of consumers, especially in overseas markets, from alcohol products to drugs and health food.   In light of the high level of consumer concern and concern over the contamination of Kobayashi Pharmaceutical's red koji products, which have led to dozens of tragic deaths in Japan this year, this is not the right time to launch a supplement product. The incident not only cast a shadow over the century-old company, but also led to the resignation of two senior executives, a sharp drop in the company's share price and a strong public call to strengthen the supervision of health food products marketed as good for health. Mr. Tanaka made it clear that Kirin has a sound quality control system to effectively reduce the risks associated with the production of supplements and other products with a relatively short history of development. "We have a well-developed process in place to ensure that all our products enjoy the same level of trust from consumers, regardless of the length of their development cycle," he stressed.   In the traditional beer and beverage business, Kirin continues to compete fiercely with Asahi Group Holdings and has introduced new brands, such as Suntory Holdings and Sapporo Holdings, to inject new competitive vitality into the domestic market. Market analysts have long predicted that the industry is bound to undergo a consolidation process in the future.   The possibility of an overseas acquisition came into sharper focus last week as Japanese retail giant Seven & i Holdings accepted a surprise takeover offer from Canada's Alimentation Couche-Tard. Mr Tanaka said Kirin would have to consider any serious offer it received. "We have to remember that the possibility is not zero at all. We need to demonstrate that all three of our main businesses are finding value in their own way, and we need to do that more effectively."

Sanofi's flu vaccine manufacturing hiccup in China, DualityBio's proposed Hong Kong IPO and UCB's $680 million sale of some Chinese assets made our news this week. Sanofi has suspended distribution of its two flu vaccines in China out of concern of waning potency. BioNTech's antibody-drug conjugate partner, DualityBio, has filed for a Hong Kong IPO. UCB will offload some Chinese assets for $680 million. And more.1. Sanofi halts flu vaccine distribution in China over fear of declining potencySanofi has temporarily stopped selling its two flu vaccines, Vaxigrip and VaxigripTetra, in China after a routine quality follow-up test flagged a decline in the vaccines’ potency in doses that were not yet distributed. The company attributed the problem to “unexpected circumstances” at the company’s production line in Shenzhen, China, which supplies doses only for the Chinese market.2. Duality plans Hong Kong IPO to fund trials of BioNTech-partnered ADCs and beyondChina’s Duality Biotherapeutics has filed for a Hong Kong IPO. The antibody-drug conjugate specialist set up shop in 2019 and has attracted deals with BioNTech, BeiGene and Adcendo that could be worth more than $4 billion combined. Duality named BioNTech-partnered ADCs targeting HER2 and B7-H3 as its “core products,” with the HER2 program nearing a potential application for accelerated approval in 2025.3. UCB sells off production plant and a clutch of older meds in China for $680MAnother foreign company is trimming its business in China. UCB is selling off its mature neurology and allergy brands in mainland China, plus a manufacturing site in the Chinese city of Zhuhai, to investment shops CBC Group and Mubadala for $680 million. The Belgian company will instead explore the launch of novel medicines in immunology, neurology and rare diseases in China, UCB CEO Jean-Christophe Tellier said.4. FDA weighs limiting PD-1 drugs in stomach cancer, calls on experts to discuss Merck, BMS and BeiGene medsThe FDA will hold an advisory committee meeting to discuss whether approvals for PD-1 inhibitors in advanced gastric and gastroesophageal junction adenocarcinoma and esophageal squamous cell carcinoma should be restricted based on tumors’ PD-L1 expression. BeiGene’s two open applications for Tevimbra, plus existing approvals for Merck’s Keytruda and Bristol Myers Squibb’s Opdivo, will be examined closely.5. As sales climb, Jazz's Epidiolex falls short in Japanese study in treatment-resistant epilepsiesA phase 3 Japanese study of Jazz Pharma’s cannabinoid drug Epidiolex has failed on the primary endpoint measuring seizure frequency in certain patients with treatment-resistant epilepsies. Still, the company pointed to numeric improvements across primary and secondary endpoints, as well as the drug’s overall global data, saying it plans to speak with Japanese authorities about a potential filing.6. Ipsen sues CSPC Pharma to block rival to cancer drug Onivyde (Bloomberg Law)Ipsen has filed a patent infringement lawsuit against China’s CSPC Pharma. The lawsuit centers on Ipsen’s Onivyde, a liposome formulation of the chemotherapy irinotecan, and CSPC’s irinotecan liposome candidate, which was filed with the FDA in late 2023 for previously treated pancreatic cancer. Ipsen alleges that CSPC steps on its new patent, which was issued Aug. 13, according to the complaint (PDF).Other News of Note:7. Bora's expansion tear continues as CDMO takes up stake in Tanvex, gains manufacturing capabilities8. With new focus, Kyowa Kirin pulls decades-old breast cancer drug Fareston from US market9. Bharat Biotech to launch oral cholera vaccine developed in partnership with Hilleman Labs (release)10. Tech company Fujitsu partners with Paradigm Health to boost clinical trials in Japan11. YolTech sells China rights to cholesterol gene editing therapy to Salubris for $29M12. API maker Global Calcium stung with Form 483 filing after FDA inspection of Indian plant

LONDON and BOSTON, Aug. 28, 2024 (GLOBE NEWSWIRE) -- Orchard Therapeutics, recently acquired by Kyowa Kirin with the goal of accelerating the delivery of new gene therapies to patients around the globe, today announced nine presentations from across its late-stage neurometabolic hematopoietic stem cell (HSC) gene therapy portfolio will be featured at the Society for the Study of Inborn Errors of Metabolism (SSIEM) 2024 Annual Symposium taking place September 3-6 in Porto, Portugal. Featured data include several accepted abstracts and an oral presentation detailing neurological, skeletal, and other clinical outcomes from a proof-of-concept (PoC) study of investigational OTL-203 in the Hurler subtype of mucopolysaccharidosis type I (MPS-IH). In addition, an encore presentation highlighting the safety and efficacy of atidarsagene autotemcel, formerly OTL-200, which is marketed as Libmeldy® in Europe and was recently approved as Lenmeldy™ in the United States for the treatment of early-onset metachromatic leukodystrophy (MLD). The company will also host a sponsored symposium on Thursday, September 5, from 12:30 to 1:30 p.m., local time titled “New horizons for metachromatic leukodystrophy with the advent of newborn screening,” featuring expert speakers with deep experience across the diagnostic journey and continuum of care who will discuss the evidence and efforts to support widespread newborn screening for this devastating neurometabolic disease. Details of the oral presentations are as follows (all times in WEST): Title: Newborn Screening (NBS) for Metachromatic Leukodystrophy: A Prospective StudyDate/Time: Wednesday, September 4 at 9:30 a.m.Presenter: Lucia Laugwitz Title: Interim analysis on neurological outcomes in Hurler syndrome patients treated with autologous ex-vivo haematopoietic stem cell gene therapyDate/Time: Thursday, September 5 at 11:30 a.m.Presenter: Giulia Consiglieri Details of the poster presentations are as follows (all times in WEST): Title: Atidarsagene autotemcel (autologous hematopoietic stem cell gene therapy) preserves cognition, language, and speech and slows brain demyelination and atrophy in early-onset metachromatic leukodystrophyDate/Time: Wednesday, September 4 and Thursday, September 5 from 12:15 to 2:15 p.m.Presenter: Francesca Fumagalli#PO-190 Title: Lentiviral hematopoietic stem cell gene therapy for late juvenile metachromatic leukodystrophyDate/Time: Wednesday, September 4 and Thursday, September 5 from 12:15 to 2:15 p.m.Presenter: Francesca Fumagalli#PO-198 Title: Quantitative Sulfatides as diagnostic tool and disease biomarker for Metachromatic LeukodystrophyDate/Time: Wednesday, September 4 and Thursday, September 5 from 12:15 to 2:15 p.m.Presenter: Francesca Fumagalli#PO-232 Title: Facilitating successful cross-border treatment of patients with rare disease via the S2 pathway for ATMPsDate/Time: Wednesday, September 4 and Thursday, September 5 from 12:15 to 2:15 p.m.Presenter: Cecilia Marinova#PO-199 Title: Non-neurological, non-skeletal outcomes after autologous hematopoietic stem cell gene therapy in Hurler patients: retrospective comparison with allogeneic hematopoietic stem cell transplantationDate/Time: Wednesday, September 4 and Thursday, September 5 from 12:15 to 2:15 p.m.Presenter: Giulia Consiglieri#PO-191 Title: Hematopoietic Stem Cell Gene Therapy for Hurler Syndrome: interim skeletal outcome and skeletal cross-correction mechanismsDate/Time: Wednesday, September 4 and Thursday, September 5 from 12:15 to 2:15 p.m.Presenter: Giulia Consiglieri#PO-189 Title: Design of a randomized phase 3 clinical trial (HURCULES) evaluating the safety and efficacy of OTL-203 in patients with MPS-IH versus standard of care with allogeneic hematopoietic stem cell transplantationDate/Time: Wednesday, September 4 from 6:15 to 8:15 p.m. (poster walk)Presenter: Giulia Consiglieri#EP-042 About Lenmeldy / LibmeldyLenmeldy™ (atidarsagene autotemcel), formerly known as OTL-200, is the only approved therapy in the U.S. for the treatment of children with pre-symptomatic late infantile (PSLI), pre-symptomatic early juvenile (PSEJ) or early-symptomatic early juvenile (ESEJ) metachromatic leukodystrophy (MLD). For additional details about Lenmeldy, please refer to the full Prescribing Information. In Europe, Lenmeldy is known as Libmeldy®, where it has been approved by the European Commission (EC), UK Medicines and Healthcare products Regulatory Agency (MHRA), and Swiss Agency for Therapeutic Products (Swissmedic). For more information about Libmeldy, please see the Summary of Product Characteristics (SmPC) available on the EMA website. The program was originated by and developed in partnership with the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy. INDICATION LENMELDY™ (atidarsagene autotemcel) is an autologous hematopoietic stem cell-based gene therapy indicated for the treatment of children with pre-symptomatic late infantile (PSLI), pre-symptomatic early juvenile (PSEJ), or early symptomatic early juvenile (ESEJ) metachromatic leukodystrophy (MLD). IMPORTANT SAFETY INFORMATION WARNINGS AND PRECAUTIONS Thrombosis and Thromboembolic Events:Treatment with LENMELDY may increase the risk of thrombosis and thromboembolic events. A child with PSEJ MLD died after experiencing a left hemisphere cerebral infarction secondary to a thrombotic event in a large blood vessel approximately 1 year after treatment with LENMELDY. Evaluate the risk factors for thrombosis prior to and after LENMELDY infusion according to best clinical practice. Encephalitis:Treatment with LENMELDY may increase the risk of encephalitis. A child with ESEJ developed a serious event of encephalitis after treatment with LENMELDY. The etiology of this event is unclear but attribution to LENMELDY cannot be ruled out. Treatment with LENMELDY may trigger a relapsing-remitting pattern of disease progression. No other events related to encephalitis have been reported during the clinical development of LENMELDY. Monitor children for signs or symptoms of encephalitis after LENMELDY treatment. Serious Infection:In the period between start of conditioning and within 1 year after LENMELDY treatment, severe Grade 3 infections occurred in 39% of all children (21% bacterial, 5% viral, 5% bacterial and viral or bacterial and fungal, and 8% unspecified). Grade 3 febrile neutropenia developed within 1 month after LENMELDY infusion in 82% of children. In the event of febrile neutropenia, monitor for signs and symptoms of infection and manage with broad-spectrum antibiotics, fluids, and other supportive care as medically indicated. Monitor children for signs and symptoms of infection after myeloablative conditioning and LENMELDY infusion and treat appropriately. Administer prophylactic antimicrobials according to best clinical practice. Veno-Occlusive Disease:Three children (8%) treated in clinical trials of LENMELDY developed veno-occlusive disease (VOD) with one Grade 4 SAE and two Grade 3 AEs. None of these three events met Hy’s Law criteria. Monitor children for signs and symptoms of VOD including liver function tests in all children during the first month after LENMELDY infusion. Consider prophylaxis for VOD with anti-thrombotic agents based on risk factors for VOD and best clinical practice. Delayed Platelet Engraftment (DPE):DPE has been observed with LENMELDY treatment. Bleeding risk is increased prior to platelet engraftment and may continue after engraftment in children with prolonged thrombocytopenia. In clinical trials of LENMELDY, 4 (10%) children had delayed platelet engraftment after day 60 (range day 67-109), with 3 children requiring platelet transfusions until engraftment occurred. Patients should be informed of the risk of bleeding until platelet recovery has been achieved. Monitor patients for thrombocytopenia and bleeding until platelet engraftment and recovery are achieved. Neutrophil Engraftment Failure: There is a potential risk of neutrophil engraftment failure after treatment with LENMELDY. Monitor neutrophil counts until engraftment has been achieved. If neutrophil engraftment failure occurs in a child treated with LENMELDY, provide rescue treatment with the unmanipulated back-up collection of CD34+ cells. Insertional Oncogenesis: There is a potential risk of LVV-mediated insertional oncogenesis after treatment with LENMELDY. Children treated with LENMELDY may develop hematologic malignancies and should be monitored life-long. Monitor for hematologic malignancies with a complete blood count (with differential) annually and integration site analysis as warranted for at least 15 years after treatment with LENMELDY. In the event that a malignancy occurs, contact Orchard Therapeutics at 1-888-878-0185 for reporting and to obtain instructions on collection of samples for testing. Hypersensitivity Reactions: The dimethyl sulfoxide (DMSO) in LENMELDY may cause hypersensitivity reactions, including anaphylaxis which is potentially life-threatening and requires immediate intervention. Hypersensitivity including anaphylaxis can occur in children with and without prior exposure to DSMO. Monitor for hypersensitivity reactions during infusion and after infusion. Anti-Retroviral Use: Children should not take prophylactic HIV anti-retroviral medications for at least one month prior to mobilization, or for the expected duration of time needed for the elimination of the medications. Anti-retroviral medications may interfere with the manufacturing of LENMELDY. If a child requires antiretrovirals for HIV prophylaxis, initiation of LENMELDY treatment should be delayed until confirmation of a negative test for HIV. Interference With Serology Testing: Due to the likelihood of a false-positive test for HIV, children who have received LENMELDY should not be screened for HIV infection using a PCR-based assay. USE IN SPECIFIC POPULATIONS Females and Males of Reproductive Potential Pregnancy TestingAs a precautionary measure, a negative serum pregnancy test must be confirmed prior to the start of mobilization, and reconfirmed prior to conditioning procedures, and before administration of LENMELDY in females of childbearing potential. ContraceptionConsult the Prescribing Information of the mobilization and conditioning agents for information on the need for effective contraception. Males capable of fathering a child and females of childbearing age should use an effective method of contraception from start of mobilization through at least 6 months after administration of LENMELDY. InfertilityThere are no data on the effects of LENMELDY on fertility. Data are available on the risk of infertility with myeloablative conditioning. In clinical trials of LENMELDY, seven children (50% of females) developed ovarian failure. Advise children of the option to cryopreserve semen or ova before treatment, if appropriate. For additional safety information, please see the full Prescribing Information. About OTL-203OTL-203 is an investigational hematopoietic stem cell gene therapy being developed for the treatment of Hurler subtype of mucopolysaccharidosis type I (MPS-IH). It uses a modified virus to insert a functional copy of the human IDUA gene into a patient’s cells. A multi-center, randomized, active controlled clinical trial designed to evaluate the efficacy and safety of OTL-203 in patients with MPS-IH compared to standard of care with allogeneic hematopoietic stem cell transplant (HSCT) is currently ongoing. OTL-203 was originated by, and initially developed in partnership with, the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy. OTL-203 has received Rare Pediatric Disease (RPD) and Fast Track designations from the U.S. FDA, as well as priority medicines (PRIME) status from the EMA. About Orchard TherapeuticsOrchard Therapeutics, a Kyowa Kirin company, is a global gene therapy leader focused on ending the devastation caused by genetic and other severe diseases by discovering, developing, and commercializing new treatments that tap into the curative potential of hematopoietic stem cell (HSC) gene therapy. In this approach, a patient’s own blood stem cells are genetically modified outside of the body and then reinserted, with the goal of correcting the underlying cause of disease with a single treatment. Founded in 2015, Orchard’s roots go back to some of the first research and clinical developments involving HSC gene therapy. Our team has played a central role in the evolution of this technology from a promising scientific idea to a potentially life-transforming reality. Today, Orchard is advancing a pipeline of HSC gene therapies designed to address serious diseases where the burden is immense for patients, families and society and current treatment options are limited or do not exist. For more information, please visit www.orchard-tx.com. About Kyowa KirinKyowa Kirin aims to discover and deliver novel medicines and treatments with life-changing value. As a Japan-based Global Specialty Pharmaceutical Company, we have invested in drug discovery and biotechnology innovation for more than 70 years and are currently working to engineer the next generation of antibodies and cell and gene therapies with the potential to help patients with high unmet medical needs, such as bone & mineral, intractable hematological diseases/hemato oncology, and rare diseases. A shared commitment to our values, to sustainable growth, and to making people smile unites us across the globe. You can learn more about the business of Kyowa Kirin at www.kyowakirin.com.