-Participating in the 'World Orphan Drug Congress USA 2026' in Boston from June 9 to 11-Unveiling research achievements of 'DDC-02' targeting rare genetic pediatric brain diseases, including Pitt-Hopkins syndrome-Discovered via the proprietary AI platform 'JWave,' anticipated to expand global partnership opportunities May 2026 JW Pharmaceutical announced on the 14th that it will participate in the 'World Orphan Drug Congress USA 2026,' held in Boston, USA, from June 9 to 11, to present the preclinical research results of 'DDC-02' (tentative development code), a novel drug candidate for rare pediatric brain diseases.DDC-02 is a First-in-Class oral small-molecule compound targeting rare genetic pediatric brain disorders, including Pitt-Hopkins syndrome. Focusing on the degradation of Wnt signaling typically observed in these conditions, JW Pharmaceutical has been meticulously researching a small-molecule therapeutic capable of normalizing this deficient signaling pathway. The newly unveiled DDC-02 was discovered utilizing 'JWave,' JW Pharmaceutical's proprietary, integrated AI-based drug discovery platform. Notably, by leveraging the platform's advanced genomic analysis capabilities, the company significantly enhanced research efficiency and precision, successfully elucidating the molecular understanding of the disease and the drug's mechanism of action (MoA). DDC-02 is currently undergoing comprehensive safety evaluations. Through its upcoming presentation at the congress, JW Pharmaceutical plans to showcase the candidate's robust research milestones and developmental potential to the global market, while actively exploring diverse collaborative opportunities. “This presentation is a meaningful opportunity to demonstrate the global potential of JW's innovative drug candidate in the field of rare pediatric brain diseases,” an official from JW Pharmaceutical stated. “Through in-depth interactions with leading experts in the rare disease sector, we will expand various collaboration opportunities, including technological partnerships, and accelerate the development of novel drugs that can significantly contribute to improving patients' quality of life.” ■ About the World Orphan Drug Congress USA The World Orphan Drug Congress USA is a premier global event dedicated to the advancement of therapeutics for rare diseases. It serves as a vital networking and knowledge-sharing platform where key stakeholders across the orphan drug ecosystem—including major pharmaceutical and biotechnology companies, government and regulatory agencies, patient advocacy groups, and investors—convene to discuss the latest industry trends and innovative case studies. 

 -Multinational Phase 3 trials conducted across five Asian countries, including South Korea-Evaluates serum uric acid reduction efficacy and safety… Clinical Study Report (CSR) expected by the end of the year April 2026 JW Pharmaceutical announced on the 27th that it has successfully completed the last patient dosing (LPD) in the multinational Phase 3 clinical trial of its gout treatment candidate, 'Epaminurad' (development code: URC102). Epaminurad is a uricosuric agent featuring a mechanism that selectively inhibits hURAT1 (human uric acid transporter-1). It is an oral novel drug candidate currently under development for hyperuricemia and gout, conditions characterized by abnormally high levels of uric acid in the bloodstream. Since receiving Investigational New Drug (IND) approval for the multinational Phase 3 trial from the Ministry of Food and Drug Safety (MFDS) in 2022, JW Pharmaceutical has been rigorously conducting clinical trials on gout patients across five Asian countries: South Korea, Taiwan, Thailand, Malaysia, and Singapore. The Phase 3 trial was intricately designed to evaluate Epaminurad's efficacy in reducing serum uric acid levels and its safety profile compared to the existing standard treatment, febuxostat. The final patient dosing was successfully concluded in Malaysia on the 23rd. Gout is a metabolic disorder that arises when uric acid generated in the body is not smoothly excreted and accumulates. When uric acid in the blood or synovial fluid remains as crystals and deposits in cartilage, periarticular tissues, or subcutaneous tissues, it triggers severe inflammation and pain. There is a particularly pressing medical need for therapeutic options that promote uric acid excretion for patients with 'underexcretion-type' gout. However, the prescription of existing treatments has been heavily restricted due to severe safety concerns regarding kidney and liver functions. JW Pharmaceutical is developing Epaminurad to effectively address these critical unmet medical needs. In the previously conducted Phase 2 clinical trials, the drug successfully met all primary and secondary efficacy endpoints while robustly confirming its safety and tolerability. Throughout the Phase 3 trial, the Data Safety Monitoring Board (DSMB), an independent committee that reviews drug safety, consistently recommended continuing the clinical trial as originally planned across multiple review meetings. Notably, JW Pharmaceutical recently registered a method-of-use (dosage and administration) patent for Epaminurad with the United States Patent and Trademark Office (USPTO). This pivotal registration significantly extended the drug's exclusivity period in the U.S. market from the original 2029 to 2038. This dosage and administration patent is now registered in a total of 18 countries, including South Korea, the U.S., Canada, and Australia, with patent examinations actively underway in 11 additional regions such as Europe, Japan, and China. Following the completion of the last patient dosing, JW Pharmaceutical plans to conduct comprehensive follow-up observations and data compilation, aiming to release the definitive Clinical Study Report (CSR) by the end of this year. "With the completion of the last patient dosing in the Phase 3 trial of Epaminurad, we will concentrate our full capabilities on follow-up observations, meticulous data compilation, and detailed analysis, targeting the release of the CSR by the end of the year," a JW Pharmaceutical official stated. "We remain deeply committed to developing Epaminurad into a groundbreaking therapeutic option capable of resolving the significant unmet medical needs in the field of gout treatment." ■ About Gout and Market SizeGout is a disease experiencing a steady increase in patient numbers, largely propelled by the westernization of dietary habits and corresponding lifestyle changes. According to the Healthcare Big Data Hub provided by the Health Insurance Review & Assessment Service, the number of gout patients in South Korea climbed sharply from 468,083 in 2020 to 553,254 in 2024. In tandem with the surging patient population, the global gout treatment market is expanding rapidly. According to a report by the global market research firm Grand View Research, the global gout treatment market, valued at approximately $2.8 billion in 2024, is projected to grow to an estimated $4.1 billion by 2030. 

-Secures exclusive domestic rights for the development and commercialization of the diabetes and obesity drug candidate, 'Bofanglutide'-Enhances dosing convenience over existing once-weekly treatments; demonstrates exceptional weight loss efficacy in clinical trials-Secures a next-generation growth engine in the metabolic disease sector by expanding its portfolio of innovative original drugs April 2026 JW Pharmaceutical announced on the 9th that it has entered into an exclusive domestic in-licensing agreement with Beijing-based Gan & Lee Pharmaceuticals for 'Bofanglutide' (development code: GZR18), a novel GLP-1 receptor agonist candidate. The contract was officially signed on the 8th. Under the terms of this agreement, JW Pharmaceutical has secured the exclusive rights to develop, register, market, and commercialize Bofanglutide in South Korea. Gan & Lee Pharmaceuticals will provide the necessary regulatory data for Investigational New Drug (IND) approvals and New Drug Applications (NDA) in Korea, maintaining close collaboration on related regulatory affairs. JW Pharmaceutical will pay Gan & Lee an upfront payment of $5 million and up to $76.1 million in development and commercial milestone payments, bringing the total potential deal value to $81.1 million. The milestones cover the development, approval, and sales across four distinct indications: Type 2 diabetes, obesity, obstructive sleep apnea (OSA), and metabolic dysfunction-associated steatohepatitis (MASH). Royalties on net sales will be paid separately based on tiered commercial performance. Bofanglutide is a synthetic peptide novel drug currently being developed as a bi-weekly subcutaneous (SC) GLP-1 receptor agonist. The drug acts on GLP-1 receptors in the pancreas to stimulate insulin secretion and regulate blood glucose levels. Concurrently, it delays gastric emptying, prolonging the feeling of satiety. Through these mechanisms, it suppresses appetite and induces weight loss, targeting indications such as diabetes, obesity, OSA, and MASH. Currently in Phase 3 clinical trials in China, Bofanglutide has already demonstrated superior convenience and efficacy compared to existing treatments. In the Phase 2b clinical trial for the obesity indication, patients achieved an average weight loss of 17.29% over 30 weeks with only bi-weekly administration. Compared to clinical data of currently available once-weekly products, Bofanglutide has shown highly potent weight loss and blood glucose-lowering effects within a relatively short administration period, highlighting its strong potential as a next-generation obesity treatment. Furthermore, Bofanglutide has received IND clearance from the U.S. Food and Drug Administration (FDA) for a Phase 2 trial targeting chronic weight management. An active Phase 2 study is currently underway in the U.S., directly comparing Bofanglutide with a placebo and tirzepatide in overweight or obese patients. JW Pharmaceutical anticipates that Bofanglutide will gain a significant competitive edge through its 'dosing convenience' in the current GLP-1 market, which is predominantly driven by once-weekly treatments. The company's strategy is to proactively address the rapidly expanding metabolic disease therapeutic market by swiftly introducing a highly advanced clinical-stage candidate. To date, alongside its proprietary innovative drug R&D, JW Pharmaceutical has successfully introduced and established promising overseas drugs in the domestic market, including the dyslipidemia treatment 'Livalo', the rheumatoid arthritis treatment 'Actemra', and the hemophilia treatment 'Hemlibra'. This latest agreement is an extension of this proven 'in-licensing' strategy and is expected to further solidify the company's robust portfolio of original prescription drugs in the metabolic disease sector. JW Pharmaceutical plans to simultaneously initiate domestic Phase 3 clinical trials for Bofanglutide in both obesity and Type 2 diabetes indications in the second half of this year. “Through this agreement, we are expanding our portfolio in the rapidly growing metabolic disease sector, centered on diabetes and obesity,” said Shin Young-sup, CEO of JW Pharmaceutical. “Leveraging JW's proven development and regulatory capabilities, we will successfully drive the domestic commercialization of Bofanglutide to provide patients with an innovative treatment option.” Dr. Li Zhi, Chief Business Officer (CBO) of Gan & Lee Pharmaceuticals, stated, “We are delighted to establish the foundation for the development and commercialization of Bofanglutide in the Korean market through our collaboration with JW Pharmaceutical. We will work closely together to ensure we can deliver a novel metabolic disease treatment option to Korean patients as swiftly as possible.” Meanwhile, according to Grand View Research, the Asia-Pacific GLP-1RA (GLP-1 receptor agonist) market is projected to be one of the fastest-growing globally, with an estimated compound annual growth rate (CAGR) of 14%, expanding from $5.47 billion in 2025 to $16.94 billion by 2033. ■ About Gan & Lee PharmaceuticalsFounded in 1998 and headquartered in Beijing, China, Gan & Lee Pharmaceuticals is a leading biopharmaceutical company with robust research, development, manufacturing, and commercialization capabilities in the field of insulin analogs and metabolic disease therapeutics. The company developed China's first domestic insulin analog and currently boasts a comprehensive portfolio of insulin products and injection devices. Recently, Gan & Lee has been expanding its innovative drug pipeline into metabolic diseases such as obesity and diabetes, spearheaded by its GLP-1 receptor agonist candidate, Bofanglutide. [Photo Caption]On the 8th, JW Pharmaceutical signed an exclusive domestic in-licensing agreement with China's Gan & Lee Pharmaceuticals for the novel GLP-1 receptor agonist candidate, 'Bofanglutide' (development code: GZR18). Lee Kyung-ha, Chairman of JW Group (right), and Wei Chen, Chairman of Gan & Lee Pharmaceuticals, pose for a commemorative photo after signing the agreement. 

 -A GFRA1 receptor agonist and First-in-Class topical novel drug candidate-Phase 1 IND approved by the MFDS; safety and tolerability to be evaluated in healthy adults at Seoul National University Hospital February 2026 JW Pharmaceutical announced on the 13th that it has received Investigational New Drug (IND) approval from the Ministry of Food and Drug Safety (MFDS) for Phase 1 clinical trials of its hair loss treatment candidate, 'JW0061.' Following this approval, JW Pharmaceutical will initiate a Phase 1 study involving 104 healthy Korean and Caucasian adults at Seoul National University Hospital. The clinical trial aims to evaluate the drug's safety and tolerability when applied topically directly to the scalp. It will also assess the pharmacokinetic (PK) profile of the drug, monitoring its absorption and metabolic processes in the body. JW0061 is a First-in-Class drug candidate designed to induce hair growth by binding directly to the GFRA1 receptor on hair stem cells. Unlike conventional treatments that rely heavily on androgen suppression or vasodilation, JW0061 employs a novel mechanism that physiologically activates innate hair growth pathways. It is currently being developed as a topical formulation suitable for both men and women. The compound has consistently proven its competitive edge through outstanding preclinical data presented at multiple international scientific conferences. In human skin organoid models, JW0061 demonstrated a 7.2-fold increase in hair follicle neogenesis compared to standard treatments. Furthermore, in animal models, it accelerated the hair growth rate by up to 39%, firmly establishing its potential as a global breakthrough drug. JW Pharmaceutical has completed the registration of substance patents for JW0061 in several countries, including South Korea, Japan, China, Australia, and Brazil. Last month, the company further solidified its intellectual property portfolio by securing a U.S. substance patent, ensuring exclusive rights to its proprietary technology in the U.S. market until 2039. Based on the robust data to be obtained from this Phase 1 trial, JW Pharmaceutical plans to concretize its subsequent clinical development plans and systematically evaluate potential indications and broader development strategies. “This IND approval holds immense significance, marking JW0061's successful transition from preclinical research into the clinical stage,” a JW Pharmaceutical official stated. “We will seamlessly execute our clinical development to successfully establish JW0061 as a globally recognized, innovative hair loss treatment.” Meanwhile, JW0061 was previously selected as a supported project by the Korea Drug Development Fund (KDDF) to conduct its non-clinical research phase.

 -Secures exclusivity until 2039… Demonstrating the competitiveness of its proprietary technology-A GFRA1 receptor agonist, positioned as a First-in-Class topical novel drug candidate-Phase 1 IND application submitted, accelerating full-scale clinical development January 2026 JW Pharmaceutical announced on the 19th the successful registration of a U.S. patent for 'JW0061,' a novel hair loss treatment candidate targeting the GFRA1 receptor. The newly registered patent is a substance patent covering the novel heterocyclic derivatives of JW0061, along with their salts and isomers. It protects the technology utilized for the treatment and prevention of various hair loss conditions, including androgenetic alopecia and alopecia areata. With the patent valid until May 2039, JW Pharmaceutical has secured long-term exclusive rights in the U.S. market. Including this U.S. patent, JW Pharmaceutical has now completed the registration of substance patents for JW0061 in a total of nine countries—including South Korea, Japan, China, Australia, and Brazil—with patent examinations currently underway in regions such as Europe and Canada.Developed as a topical formulation applied directly to the scalp, JW0061 is a First-in-Class drug candidate operating as a GFRA1 agonist. The compound binds directly to the GFRA1 receptor expressed on hair stem cells, activating downstream signaling pathways to promote hair follicle neogenesis and accelerate hair growth. A key characteristic of JW0061 is its design, which is based on a mechanism that physiologically activates the body's innate hair growth pathways. This represents a novel mechanism of action that differentiates it from conventional treatments relying on androgen suppression or vasodilation, making it an innovative therapeutic option applicable to both male and female patients. JW Pharmaceutical has consistently presented preclinical results for JW0061 at major international conferences, including the Society for Investigative Dermatology (SID). In studies utilizing human skin organoids and animal models of androgenetic alopecia, the company confirmed the candidate's superiority over existing standard treatments in terms of hair growth rate and follicle generation capacity. Notably, in the human skin organoid assay, JW0061 demonstrated up to a 7.2-fold increase in hair follicle generation compared to standard therapies. Furthermore, animal models showed up to a 39% improvement in efficacy, firmly establishing its competitive edge as a breakthrough drug candidate. Building upon these strong preclinical achievements, JW Pharmaceutical recently submitted an Investigational New Drug (IND) application for Phase 1 clinical trials of JW0061 to the Ministry of Food and Drug Safety (MFDS). “JW0061 is a next-generation novel drug candidate for hair loss targeting the GFRA1 receptor. This U.S. patent registration is highly significant as it represents official recognition of our proprietary technology's competitiveness in the world's largest market,” a JW Pharmaceutical official stated. “We will seamlessly advance our clinical development to establish JW0061 as a global innovative hair loss treatment.” Meanwhile, JW0061 was previously selected as a supported project by the Korea Drug Development Fund (KDDF) to conduct its non-clinical research phase.

 -First-in-Class drug candidate discovered utilizing the proprietary ‘JWave’ platform-Accelerating research to enter preclinical trials with 24 months of funding December 2025 JW Pharmaceutical announced on the 8th that its research on a novel metabolic disease therapeutic candidate, discovered via its AI-based drug discovery platform ‘JWave,’ has been selected for the ‘2nd Korea Drug Development Fund (KDDF, Director Park Young-min) Project of 2025: Building a New Drug R&D Ecosystem (Candidate Material).’ By leveraging JWave, JW Pharmaceutical applied advanced structure-based modeling and reinforcement learning algorithms to rapidly optimize hit compounds and secure a lead compound featuring a novel mechanism of action. This compound is currently undergoing rigorous optimization at the candidate stage. Backed by this KDDF grant, the company will receive research funding over the next 24 months. JW Pharmaceutical plans to systematically conduct structural optimization of the lead compound, elucidate its mechanism of action, and perform preliminary toxicity testing to successfully advance the candidate into preclinical trials. “The newly secured lead compound is an oral First-in-Class candidate equipped with a completely novel mechanism of action, fundamentally distinct from existing metabolic disease treatments. We anticipate it will provide a groundbreaking new treatment option for patients,” stated an official from JW Pharmaceutical. “By continually strengthening our AI-driven drug discovery capabilities, we will concentrate our efforts on creating innovative therapeutics in disease areas burdened with high unmet medical needs.” ■ About JWave JW Pharmaceutical is maximizing its drug development efficiency through its integrated AI-based R&D platform, ‘JWave.’ Constructed by merging the company's legacy big data-driven drug discovery systems, ‘Jewelry’ and ‘Clover,’ JWave is meticulously designed for deployment across the entire drug discovery lifecycle—from target exploration to lead optimization. The platform operates roughly 20 distinct AI models, fueled by a vast repository of biological and chemical data. This includes genomic information from over 500 cell lines, organoids, and disease animal models, alongside data on approximately 40,000 synthetic compounds. JW Pharmaceutical is currently expanding JWave into a cloud-based environment to significantly accelerate large-scale genomic analysis and AI training. Furthermore, the platform is being actively utilized in collaborative research initiatives spanning industry, academia, research institutes, and hospitals. ■ About the KDDF The Korea Drug Development Fund (KDDF) is an interagency national R&D initiative launched to bolster the global competitiveness of the domestic pharmaceutical and biotechnology sectors. Over a 10-year span beginning in 2021, the fund comprehensively supports the entire life cycle of drug development. Its primary objectives include fortifying the domestic R&D ecosystem, driving global commercialization success, and generating substantial public health benefits.

 -Exclusivity period extended to 2038… accelerating the development of a global Best-in-Class gout drug-Multinational Phase 3 clinical trials on track… DSMB recommends 'trial continuation' in all four consecutive reviews December 2025 JW Pharmaceutical announced on the 3rd that it has acquired a method-of-use (dosage and administration) patent for its gout treatment, Epaminurad (development code: URC102), from the United States Patent and Trademark Office (USPTO). This patent registration, alongside the substance patent for Epaminurad, is evaluated as a core intellectual property (IP) asset that can effectively block market entry by latecomers. Consequently, the drug's exclusivity period in the U.S. market has been significantly extended from the original 2029 to 2038. The dosage and administration patent for Epaminurad is now registered in 18 countries, including South Korea, the U.S., Canada, and Australia, with examinations currently underway in 11 additional countries such as Europe, Japan, and China. This recent U.S. patent registration is expected to exert a positive influence on the review processes in other nations. Developed as an oral medication, Epaminurad is a uricosuric agent that selectively inhibits hURAT1 (human uric acid transporter-1). It is a highly efficacious novel drug candidate for hyperuricemia and gout, conditions characterized by abnormally high uric acid levels in the blood. Amid surging market demand for effective and safe novel therapeutics—driven by the growing gout patient population and safety concerns surrounding existing treatments—JW Pharmaceutical aims to develop Epaminurad as a 'Best-in-Class' drug designed to satisfy these critical unmet medical needs. Epaminurad is currently undergoing Phase 3 clinical trials across five Asian countries, including South Korea. Previously, in Phase 2 clinical trials, the drug successfully met both primary and secondary efficacy endpoints while demonstrating excellent safety and tolerability profiles. The Data Safety Monitoring Board (DSMB), an independent committee reviewing drug safety during the Phase 3 trials, has recommended continuing the study as planned in all four meetings held to date. These consecutive recommendations from the DSMB are highly significant, reflecting a robustly positive assessment of Epaminurad's safety and data reliability. The Phase 3 trial is scheduled to complete its last patient dosing by April 2026, with the Clinical Study Report (CSR) anticipated by the end of that year. Concurrently, JW Pharmaceutical is pursuing global out-licensing partnerships targeting advanced markets such as the U.S. and Europe. In 2019, the company successfully out-licensed the developmental and commercial rights for China, Hong Kong, and Macau to Simcere Pharmaceutical Group. "This U.S. method-of-use patent registration is a pivotal achievement that strengthens Epaminurad's intellectual property (IP) foundation on a global scale," said an official from JW Pharmaceutical. "With the exclusivity period extended to 2038, we expect its global commercialization value and strategic competitiveness to increase exponentially." ■ About Gout TreatmentsGout is generally classified into two types. The first is 'underexcretion' gout, where uric acid is not efficiently excreted, unlike in healthy individuals. The second is 'overproduction' gout, where the body produces more uric acid than normal. While medications that reduce uric acid production are currently on the market, uricosuric agents for underexcretion gout are not actively prescribed despite the large patient population due to severe safety concerns regarding the kidneys and liver. Thus, the medical need for a genuinely 'safe' gout treatment is greater than ever. In Phase 2 clinical trials involving gout patients, Epaminurad met all primary and secondary efficacy endpoints, definitively confirming its excellent safety and tolerability. ■ About Gout and Market SizeGout, known for causing excruciating pain at even the "slightest breeze," is a representative intractable disease. Driven by the westernization of dietary habits and a sharp rise in incidence among younger demographics, the number of patients in South Korea has surged rapidly from 468,083 in 2020 to 553,254 as of 2024 (Source: Healthcare Big Data Hub, Health Insurance Review & Assessment Service).Gout is a metabolic disorder that occurs when uric acid generated in the body accumulates instead of being excreted through urine. Uric acid in the blood or synovial fluid forms crystals that deposit in cartilage, periarticular tissues, and subcutaneous tissues, causing severe inflammation and pain. Frequent meat consumption, heavy drinking, and overeating are common risk factors, and recently, the disease has been striking across diverse age groups without specificity. Like hypertension and diabetes, once it develops, a complete cure is difficult, making it a chronic condition requiring continuous management.In parallel with the growing patient population, the gout treatment market is expanding significantly. According to a report by global market research firm Grand View Research, the global gout treatment market, valued at approximately 4 trillion KRW ($2.8 billion) as of 2024, is projected to reach around 6 trillion KRW ($4.1 billion) by 2030.

 -AI-based small molecule drug research recognized as a national R&D project-Direct STAT6 inhibition mechanism… expected to yield novel treatments for Type 2 inflammatory immune diseases-Backed by 16 months of research funding, aims to advance as an oral First-in-Class drug candidate June 2025 C&C Research Laboratories announced on the 30th that its research program developing a STAT6-targeted treatment for eosinophilic esophagitis (EoE) has been selected as a project for the ‘1st Korea Drug Development Fund (KDDF) of 2025.’ The KDDF is an interagency national R&D initiative launched to bolster the global competitiveness of the domestic pharmaceutical and biotechnology industries. Over a 10-year period beginning in 2021, the fund supports the entire life cycle of drug development with the goals of strengthening the domestic R&D ecosystem, driving global commercialization, and generating public health benefits. Through this project, C&C Research Laboratories will receive research funding for 16 months to optimize a lead compound that directly inhibits the STAT6 protein. The company plans to accelerate its research with the objective of entering preclinical stages to develop this compound into an oral, small-molecule therapeutic. Eosinophilic esophagitis (EoE) is a chronic inflammatory and rare disease caused by an excessive Type 2 (Th2) immune response in the esophagus, and currently presents limited treatment options. In particular, due to issues with recurrence, refractory conditions, and low patient adherence to steroids and biologics, there is a significant unmet medical need for targeted therapies featuring novel mechanisms of action. STAT6 acts as a key regulator of the Type 2 inflammatory response triggered by IL-4/IL-13 stimulation. It is a major factor controlling the expression of eotaxin, a protein that induces eosinophil infiltration into the esophagus. C&C Research Laboratories has successfully identified a lead compound that selectively and directly inhibits the STAT6 protein. The company has also confirmed the reduction of Th2 gene expression and its anti-inflammatory efficacy through in vitro and in vivo studies. This research centers on a lead compound discovered via 'JWave,' JW's proprietary, integrated AI-based drug R&D platform. Operating in a web-based environment, JWave is utilized throughout the entire drug research cycle, from hit discovery to lead optimization. Supported by this new funding, C&C Research Laboratories intends to secure foundational data—including structure optimization, pharmacokinetics (PK), and toxicity profiling—necessary to propel the candidate into preclinical trials. Furthermore, the company will explore the potential to expand the drug's indications to other Th2-driven inflammatory immune diseases, such as atopic dermatitis and asthma. “STAT6 is a crucial regulator of the Type 2 inflammatory response, and we anticipate this lead compound will emerge as a groundbreaking treatment option for various immune disorders, including rare diseases,” an official from C&C Research Laboratories stated. “We remain committed to advancing innovative drug research leveraging our AI-based platform to lead the therapeutic market in areas of high unmet need.” About C&C Research Laboratories Established in 1992 as Korea's first Korea-Japan joint bio-venture by JW Pharmaceutical and Chugai Pharmaceutical (a member of the Roche Group), C&C Research Laboratories became a wholly-owned subsidiary of JW Pharmaceutical in 2020. Since initiating its independent research projects in 2006, the institute has been discovering First-in-Class drug candidates through an advanced R&D platform integrating AI and deep learning technologies. It currently boasts a robust portfolio of eight novel drug pipelines targeting oncology and immunological diseases.

• First-in-Class oral dual inhibitor targeting STAT5/STAT3• Discovered through JW’s AI-based integrated R&D platform, JWave• Demonstrated potential to overcome resistance to FLT3 and BCL-2 inhibitors in AML models April 29, 2025 C&C Research Laboratories announced that it presented preclinical results for its investigational therapy CNC-01, a dual STAT5/STAT3 inhibitor, at the American Association for Cancer Research Annual Meeting 2025 (AACR 2025) on April 28 in Chicago, USA. AACR is the world's largest academic conference dedicated to cancer research. At this year’s meeting, C&C Research Laboratories unveiled the mechanism of action and preclinical efficacy data of CNC-01 for the first time, while also exploring potential global licensing opportunities. CNC-01 is an oral, small-molecule compound that selectively and directly inhibits both STAT5 and STAT3 proteins. It represents a First-in-Class therapeutic approach designed to address resistance mechanisms seen with existing acute myeloid leukemia (AML) treatments. According to the preclinical data presented at AACR 2025, CNC-01 demonstrated superior tumor suppression compared to current FLT3 inhibitors in resistant models, including FLT3-TKD (F691L) mutations and bone marrow microenvironment-mimicking models. Moreover, CNC-01 exhibited robust antitumor efficacy as a monotherapy in animal models unresponsive to BCL-2 inhibitors. Combination treatment with BCL-2 inhibitors further enhanced therapeutic outcomes. Notably, both complete remission (CR) and partial remission (PR) responses were observed in monotherapy and combination settings. In survival analyses, CNC-01 monotherapy significantly prolonged survival compared to BCL-2 inhibitors alone. In combination therapy, more than half of the subjects remained alive at the study’s end, with the median survival not yet reached, underscoring the strong anticancer potential of CNC-01. A representative from C&C Research Laboratories commented, "AML remains a challenging disease with high unmet medical needs due to diverse genetic mutations and treatment resistance. Through its innovative dual inhibition mechanism, our STAT5/STAT3-targeted candidate has demonstrated outstanding preclinical efficacy. We aim to advance clinical development through global partnerships and licensing opportunities." CNC-01 was discovered via JW’s proprietary AI-driven integrated R&D platform, JWave, and is currently under development with funding support as part of the Korea Drug Development Fund (KDDF) initiative, selected in 2023. [Photo Caption]C&C Research Laboratories presented preclinical results of CNC-01, an investigational dual STAT5/STAT3 inhibitor for AML, at AACR 2025 in Chicago on April 28 (local time). ________________________________________ About C&C Research LaboratoriesFounded in 1992 as Korea’s first joint biotech venture between JW Pharmaceutical and Chugai Pharmaceutical (a member of Roche Group), C&C Research Laboratories became a wholly owned subsidiary of JW Pharmaceutical in 2020. Since 2006, the company has been pursuing innovative drug discovery by integrating chemical and biological big data with AI and deep learning technologies.C&C Research Laboratories currently manages a pipeline of eight novel drug candidates across oncology and immunology indications, with a strategic focus on targeting STAT (Signal Transducer and Activator of Transcription) proteins involved in cell growth, mutation, and proliferation. About Acute Myeloid Leukemia (AML) and Market OutlookAML is the most common type of leukemia in adults but remains difficult to treat due to high relapse rates and drug resistance. Given its rapid progression and potential for systemic spread, AML presents significant clinical challenges. Current standard therapies include cytarabine-based chemotherapy and venetoclax regimens, while relapsed/refractory AML cases are treated with targeted agents like gilteritinib (FLT3 inhibitor).According to DRG Reports, the global AML therapeutics market, valued at USD 1.355 billion in 2021, is projected to grow at a CAGR exceeding 10%, reaching approximately USD 3.323 billion by 2031.

 -First domestic partnership with Tempus AI, the world’s largest holder of clinical and genomic data-Utilizing patient-derived organoids and real-world data to evaluate drug candidates and develop optimized precision oncology therapies October 2024 JW Pharmaceutical announced on the 29th that it has partnered with Tempus AI, a global leader in AI-based precision medicine, to accelerate the development of anti-cancer drugs utilizing Real-World Data (RWD) and patient-derived organoids. This collaboration focuses on swiftly verifying the efficacy and safety of oncology drug candidates while enhancing research efficiency across multiple cancer indications. JW Pharmaceutical plans to leverage Tempus AI's multimodal datasets, including clinical records and pathology images, in combination with patient-derived organoid models. This approach will enable the precise evaluation of JW’s drug candidates and the validation of biomarkers that predict treatment response. Tempus AI provides diverse patient-derived organoid models that reflect the unique characteristics of tumors from actual cancer patients. These models are seamlessly integrated with Tempus’s next-generation genomic sequencing technology, xT, and its extensive clinical datasets. By comparing organoid research results with real patient data, the two companies aim to improve the predictive accuracy of clinical trial outcomes, ultimately developing optimized precision cancer therapies. Ryan Fukushima, Chief Operating Officer (COO) of Tempus AI, stated, “We are thrilled to partner with JW Pharmaceutical, a company leading next-generation oncology drug development using RWD and AI. Tempus AI has developed organoid panels tailored to specific cancer indications that reflect real patient characteristics. We are eager to see the significant results this research will achieve.” Chan-hee Park, Chief Technology Officer (CTO) of JW Pharmaceutical, added, “We are excited to establish this first-of-its-kind partnership in Korea with Tempus AI, a global leader in AI and precision medicine. This collaboration marks a significant turning point for real-world data-driven drug development in Korea and is expected to positively impact the domestic pharmaceutical industry by aligning with global trends.” About Tempus AITempus AI (Nasdaq: TEM) is a technology company advancing precision medicine through the application of artificial intelligence. It houses the world’s largest multimodal data library and provides personalized medical solutions through efficient operational systems. Tempus AI equips physicians with AI-based precision medicine tools, enabling personalized treatment, while supporting the discovery, development, and delivery of optimal therapeutics. Tempus AI’s mission is to help physicians deliver more effective treatments by leveraging its vast data resources. Tempus AI Press Release URL:https://www.tempus.com/news/tempus-announces-collaboration-with-jw-pharmaceutical-to-apply-real-world-data-and-biological-modeling-to-enhance-early-research-and-development/