-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/

  -Integration of existing AI drug development platforms ‘Jewelry’ and ‘Clover’ with expanded AI capabilities-Consolidation of biological and chemical informatics big data and AI models-Transition to a public cloud environment within the year to significantly accelerate candidate discovery-Strengthening AI-driven innovative drug development and open innovation with external research institutions August 2024 JW Pharmaceutical is accelerating the development of innovative drugs through its advanced AI platform. On the 27th, JW Pharmaceutical announced the official launch of its AI-based integrated drug discovery and development platform, ‘JWave’.JWave (JW AI-powered Versatile drug Exploration) is JW Pharmaceutical’s proprietary AI-powered drug development platform. It integrates the company’s existing big data-driven drug exploration systems, Jewelry and Clover, while significantly expanding the application scope of its AI models. Designed for seamless operation throughout the entire drug discovery cycle, JWave allows JW’s New Drug Research Center and C&C New Drug Research Institute to utilize AI technology within a web portal environment. From the rapid identification of active compounds targeting disease-causing proteins to lead optimization and drug candidate discovery, JWave helps reduce costs and shorten research timelines. The platform harnesses JW’s extensive biological and chemical informatics big data, including over 500 cell lines, organoids, genomic information from various disease animal models, and approximately 40,000 synthetic compounds. It also incorporates more than 20 proprietary AI models to predict and optimize indications, target protein structures, drug design, and ADMET (absorption, distribution, metabolism, excretion, and toxicity). Notably, JW Pharmaceutical plans to transition JWave from its internal servers to a public cloud environment by the end of this year. This shift is expected to drastically reduce the time required for large-scale genomic data analysis and AI model training, bringing about revolutionary changes to the development timelines of JW’s internal drug discovery projects. JW Pharmaceutical also intends to leverage JWave to collaborate actively with external biotech companies and research institutions in the pursuit of innovative drug development. Recently, JW joined the ‘K-MELODY’ project, a national initiative led by the Ministry of Health and Welfare and the Ministry of Science and ICT. This collaborative effort aims to develop an ADMET federated learning AI model through partnerships with domestic industry, academia, research, and medical sectors. Chan-hee Park, Chief Technology Officer (CTO) at JW Pharmaceutical, stated, “JW Pharmaceutical has been at the forefront of building proprietary data science platforms, such as Jewelry and Clover, which have enabled the discovery of over 10 First-in-Class innovative drug candidates. With JWave, we aim to accelerate the development of novel First-in-Class drugs targeting Wnt, Stat, and other innovative mechanisms in oncology, immunology, and regenerative medicine. Additionally, leveraging JWave’s AI capabilities will allow us to advance Best-in-Class and Fast Follower strategies, enabling swift market entry for targeted therapeutics.”

 Leveraging Zebrafish with 80–90% genetic similarity to humans to expand new drug pipelinesCollaboration with Zebit to identify metabolic disease drug candidates based on JW's compoundsCost reduction and accelerated drug development through alternative animal testing July 2024 JW Pharmaceutical is accelerating the expansion of its drug development pipeline by leveraging zebrafish as an innovative alternative model.On the 10th, JW Pharmaceutical announced that it has signed a joint research agreement with Zebit, a preclinical testing institution specializing in zebrafish models, to develop new drugs for metabolic diseases Through this agreement, JW Pharmaceutical will utilize Zebit’s zebrafish models to advance research into drug candidates for metabolic diseases such as obesity and diabetes. Zebit will provide customized zebrafish models for metabolic diseases using gene-editing technologies, along with its advanced drug screening platform. The two companies aim to identify innovative drug candidates with novel mechanisms of action by the end of this year. JW Pharmaceutical will provide early-stage active compounds derived from its AI-based R&D platform, Jewelry, which Zebit will screen to evaluate their therapeutic potential and safety.The partnership follows the formalization of their collaboration through an MOU signed last year. Since then, JW Pharmaceutical has been conducting indication expansion studies on its own drug candidates using Zebit’s zebrafish models and has been developing follow-up strategies accordingly.Zebrafish, a tropical freshwater fish, have gained attention as a non-clinical translational research model that can substitute for mammalian experiments due to their over 80% genetic similarity to humans. With high reproductive rates and efficient screening capabilities, zebrafish are particularly useful in the early stages of drug development, enabling rapid validation of drug targets, efficacy, and safety. Furthermore, zebrafish possess metabolic organs similar to humans, such as the pancreas, muscle, liver, and adipose tissue, making them an optimal model for metabolic research. Leading pharmaceutical companies in the United States and Europe are already utilizing zebrafish models extensively during the drug discovery process. JW Pharmaceutical has also led the way domestically by establishing a specialized zebrafish facility, the “Zebrafish Room”, at its headquarters in Gwacheon last year. This facility is being actively utilized for mechanism-of-action studies to identify biological and chemical processes of new drug candidates. Ki-baek Lee, CEO of Zebit, stated, “Zebrafish-based metabolic disease drug screening is a state-of-the-art platform that meets global standards. Our collaboration with JW Pharmaceutical demonstrates open innovation at its finest, combining the strengths of pharmaceutical companies and biotech firms to create powerful synergies.” Chan-hee Park, Chief Technology Officer (CTO) of JW Pharmaceutical, added, “This partnership with Zebit is expected to enhance our ability to develop metabolic disease treatments with novel mechanisms. Drug research using non-animal models such as zebrafish, AI, and organoids offers more efficient and reliable outcomes compared to conventional methods.” Meanwhile, JW Pharmaceutical is actively pursuing an open innovation strategy to expand its drug candidate modalities (therapeutic approaches). By integrating technologies from domestic and global biotech firms, JW aims to broaden its drug development pipeline and enhance R&D efficiency through collaborations with partners possessing advanced R&D platforms, including zebrafish, AI, and organoids. 

 -Lead compound selectively and directly inhibits XBP1s-Supported by research funding for two years, advancing to an oral first-in-class anticancer drug candidate-Expected to show antitumor effects in patients resistant to standard hormone therapy-Discovered through AI-based R&D platform CLOVER, continuously generating new drug candidates in oncology and immunological diseases June 2024 JW Pharmaceutical (CEO Shin Young-seop) announced on the 25th that its subsidiary, C&C Research Laboratories (CEO Park Chan-hee), has been selected for the "2024 1st National New Drug Development Project for Building a New Drug R&D Ecosystem" to develop a treatment for hormone-resistant and non-responsive prostate cancer. The National New Drug Development Project is an interagency national R&D initiative launched to strengthen the global competitiveness of the domestic pharmaceutical and biotechnology industries. The National New Drug Development Project Team (led by Director Park Young-min, hereafter referred to as 'the team') aims to support all phases of new drug development from 2021 to 2031, enhancing the domestic new drug R&D ecosystem, creating globally practical outcomes, and generating public health benefits. Over the next two years, C&C Research Laboratories will receive research funding from the team to optimize a lead compound that directly inhibits XBP1s and develop an oral first-in-class anticancer drug candidate. XBP1s is a protein that is highly expressed in various solid tumors, playing a crucial role in cancer proliferation. Specifically, XBP1s increases the activity of genes related to cancer cell survival, diminishing the efficacy of standard treatments and enabling cancer cells to evade immune responses. The lead compound selected for this project was discovered through the institute's proprietary data science platform, CLOVER, which combines chemical and biological informatics big data with AI and deep learning technologies. This lead compound demonstrated excellent anticancer effects by directly binding to and inhibiting the XBP1s protein in cell experiments, and showed tumor suppression effects. It also exhibited superior antitumor efficacy in drug response measurements in prostate cancer animal models compared to control groups. C&C Research Laboratories plans to conduct structural optimization research on the XBP1s-targeted anticancer lead compound and derive a preclinical new drug candidate by 2025. A representative from JW Pharmaceutical stated, "Prostate cancer, the second most common cancer in men, has a high demand for innovative drugs with new mechanisms of action due to resistance and non-responsiveness to first-line standard hormone therapies. We aim to develop the lead compound targeting XBP1s into an innovative anticancer drug that can overcome the limitations of existing treatments not only for prostate cancer but also for various solid tumors." C&C Research Laboratories, established in 1992 as the first Korea-Japan joint venture bioventure by JW Pharmaceutical and Chugai Pharmaceutical, a member of the Roche Group, became a 100% subsidiary of JW Pharmaceutical in 2020. Since 2010, C&C Research Laboratories has been discovering innovative drug candidates through its R&D platform, CLOVER, which integrates AI and deep learning technologies with chemical and biological informatics big data. It currently holds a pipeline of over 10 new drug candidates for oncology and immunological diseases, including gout treatment 'Epaminurad (URC102)', atopic dermatitis treatment 'Ezzofrant (JW1601)', and STAT3-targeted anticancer drug 'JW2286', all of which have been licensed to JW Pharmaceutical. The institute is also focusing on developing new drugs targeting STAT proteins that regulate cell growth, mutation, and proliferation, alongside its XBP1s-targeted research. C&C Research Laboratories' new drug pipeline has been continuously recognized as a national project by the team. In 2021, the institute was selected for the lead compound optimization research for a STAT3-targeted atopic dermatitis treatment, in 2022 for the preclinical research of the STAT3-targeted anticancer drug JW2286, and in 2023 for the lead compound optimization research for a STAT5/STAT3 dual-targeted acute myeloid leukemia treatment. 

About Prostate Cancer and the Market

Prostate cancer is a malignant tumor that develops in the prostate gland cells and is the second most frequently diagnosed cancer in men worldwide. Its incidence increases with age, and while early-stage disease progresses slowly with a five-year survival rate of 97.1%, the survival rate for metastatic prostate cancer drops sharply to 34.1%. Prostate cancer is often asymptomatic in its early stages, making detection difficult, but as it progresses, symptoms such as urinary abnormalities, testicular pain, and erectile dysfunction may appear. The primary treatment, hormone therapy, is initially effective but most patients develop resistance within 18-24 months, leading to recurrence. This creates a high unmet medical need for new therapies. Globally, while the incidence of prostate cancer is decreasing or stabilizing, the number of diagnosed patients is expected to increase due to population aging. According to "Datamonitor Healthcare" in 2023, approximately 597,440 people were diagnosed with prostate cancer worldwide in 2022, and this number is projected to rise to about 681,600 by 2032. The global prostate cancer treatment market is expected to grow from $10.667 billion in 2022 to over $19.998 billion by 2032, reflecting a compound annual growth rate (CAGR) of 6.5%. 

About XBP1s

XBP1s (X-Box-binding Protein 1 splicing form) is a protein activated by endoplasmic reticulum (ER) stress and androgen receptor (AR) signaling, known to play a critical role in the proliferation of various solid tumors. High ER stress and increased expression of XBP1s are common in patients resistant to standard treatments, making it a major cause of drug resistance. XBP1s increases the activity of genes related to cancer cell survival, rendering standard treatments ineffective and further weakening the efficacy of drugs by inhibiting immune cells' anticancer responses, allowing cancer cells to evade immune attacks. Currently, there are no clinically developed XBP1s inhibitors as anticancer drugs worldwide. Therefore, JW Pharmaceutical expects that C&C Research Laboratories' XBP1s inhibitor will be developed as a new targeted therapy providing both anticancer effects and immune modulation for patients resistant to standard treatments.

 -STAT3 Inhibitor JW2286 Developed as a First-in-Class Global Innovative Drug-Expected to Address Unmet Medical Needs in Various Solid Tumors-Progression to Clinical Phase with In-House R&D Platform Targeting Wnt and STAT June 2024 JW Pharmaceutical announced on the 19th that it has received approval from the Ministry of Food and Drug Safety for the clinical phase 1 IND for its STAT3 inhibitor 'JW2286', which is being developed as a treatment for solid tumors. JW2286 is a novel mechanism-based first-in-class candidate that selectively inhibits STAT3. It is being developed as an oral drug, targeting triple-negative breast cancer, gastric cancer, and colorectal cancer. This clinical trial aims to evaluate the safety, tolerability, and pharmacokinetic properties of JW2286 in approximately 70 healthy Korean and Caucasian adults at Seoul National University Hospital. STAT3 is a protein (transcription factor) that promotes the expression of various genes within cells. Abnormal activation of STAT3 is deeply involved in the growth, proliferation, metastasis, and drug resistance of cancer cells, and it is also known to cause inflammatory diseases and autoimmune disorders such as atopic dermatitis. According to preclinical pharmacological evaluation results presented by JW Pharmaceutical at the 2021 American Association for Cancer Research (AACR) Annual Meeting, JW2286 demonstrated higher efficacy and safety compared to standard therapies in several solid tumors with high STAT3 activity as a biomarker, showing particularly strong efficacy in triple-negative breast cancer. Triple-negative breast cancer is a type of breast cancer that is not affected by female hormones or epidermal growth factor (HER2), with very high unmet medical needs. A JW Pharmaceutical representative stated, "The IND approval is a significant milestone in the development of JW2286, recognizing its potential to address unmet medical needs in oncology and immunological disorders. We will continue to develop JW2286 as the first innovative drug that selectively inhibits STAT3." JW2286 was selected as a supported project in the '2022 2nd National New Drug Development Project'. With funding from the National New Drug Development Project, JW Pharmaceutical completed the GLP (Good Laboratory Practice) toxicity evaluation and production of the clinical drug for JW2286. 

About STAT and JW Research

The JAK-STAT pathway is a representative intracellular signal transduction system involved in cell development, differentiation, growth, survival, and various immune responses. STAT proteins consist of seven different types (STAT1~4, STAT5A, STAT5B, STAT6) with distinct functions, activated by the upstream protein JAK. While the JAK-STAT pathway is typically controlled within a normal range, abnormal activation (overexpression or hyperactivation) of the proteins can lead to various diseases. Pharmaceutical companies, including global big pharma, are focusing on developing treatments for immune and tumor diseases by targeting JAK to inhibit the JAK-STAT signaling pathway. However, to date, no new drugs have been developed globally that directly inhibit STAT. JW Pharmaceutical is developing specific small molecule inhibitors that directly bind to and inhibit the activity of STAT3, STAT5, STAT6, aiming to achieve powerful effects and overcome the side effect issues of JAK inhibitors. JW Pharmaceutical's subsidiary, C&C New Drug Research Institute, is focused on discovering new drugs targeting STAT proteins, currently securing a pipeline of over 10 new drug candidates for tumor and immune disorders. C&C's new drug pipeline has been continuously recognized as a national project. In addition to the preclinical research on the STAT3-targeted anticancer drug JW2286 (2022), the optimization research for the lead compound of the STAT3-targeted atopic dermatitis treatment (2021), and the optimization research for the lead compound of the STAT5/STAT3 dual-targeted acute myeloid leukemia treatment (2023) were each selected as national projects. 

About AI and Data Science Platform-Based Innovative Drug Development Targeting Wnt and STAT

JW Pharmaceutical is continuously generating new drug candidates in oncology, immunological diseases, and regenerative medicine by leveraging its in-house data science platforms 'JWELRY' and 'CLOVER', targeting Wnt and STAT. Recently, JW has been enhancing and differentiating its R&D platform by integrating AI (artificial intelligence) and deep learning technology with chemical and biological informatics big data and actively pursuing open innovation with external AIDD (AI-based Discovery) platforms. JW's new drug research center is utilizing 'JWELRY (JW Excellent LibraRY)', specialized in the Wnt signaling pathway, as a core technology. JWELRY is based on a library of tens of thousands of compounds involved in the Wnt pathway and drug evaluation and mechanism research technologies that distinguish inhibition and activation of the Wnt pathway. CLOVER (C&C Research Laboratories Omics serVER) is the R&D platform of C&C New Drug Research Institute, a research subsidiary of JW Pharmaceutical, actively engaged in developing new drugs targeting STAT proteins. JW is accelerating the development of its new drug pipeline targeting Wnt or STAT, with plans to initiate clinical trials for the Wnt-targeted hair loss treatment 'JW0061' and the STAT3-targeted anticancer drug 'JW2286' in 2024. Additionally, JW is continuously striving to enhance translational research to reduce the gap between preclinical and clinical stages and increase the likelihood of clinical success by integrating R&D platforms such as AI, organoids, and zebrafish (open innovation).

 -First Efficacy Evaluation on Human Skin Organoids and Androgenic Alopecia Animal Model Revealed at the Society of Investigative Dermatology-Demonstrated Excellent Hair Follicle Proliferation and Hair Growth Compared to Standard Treatments-Innovative First-in-Class Drug Activating Wnt, First to Elucidate Hair Growth Mechanism-Developed as a Global New Drug that Can Complement and Replace Existing Hair Loss Treatments, Suitable for Both Men and Women' May 2024 JW Pharmaceutical announced on the 20th that it had presented the superiority of Wnt-targeted hair loss treatment ‘JW0061’ in hair follicle formation and hair growth compared to existing treatments at the Society of Investigative Dermatology, held from May 15 to 18 in Dallas, Texas. At this conference, JW Pharmaceutical revealed for the first time the efficacy of JW0061 evaluated on human skin organoids (organ-like structures) and an androgenic alopecia animal model. The study using skin organoids differentiated from human induced pluripotent stem cells was designed to predict whether JW0061 generates hair follicles on the actual human scalp. Organoids can replace animal testing and are a next-generation drug development technology that can assess drug efficacy without direct human trials. When JW0061 and the standard treatment were applied to skin organoids, the number of hair follicles in the JW0061-treated organoids significantly increased compared to the standard treatment. Specifically, JW0061-treated organoids had 7.2 times and 4.0 times more hair follicles on the 5th and 10th days of drug application, respectively, than the standard treatment. Notably, JW Pharmaceutical’s skin organoid research presentation was the only result in the poster session at this conference, drawing significant attention from participants. In addition, the hair growth effect was confirmed in an androgenic alopecia animal model induced by the male hormone testosterone. The androgenic alopecia animal model was divided into four groups: low-dose JW0061, high-dose JW0061, standard treatment (SoC), and placebo. Both low-dose and high-dose JW0061 groups showed faster hair growth promotion compared to the standard treatment. On the 17th day of administration, low-dose and high-dose JW0061 showed 18% and 39% efficacy improvement, respectively, compared to the standard treatment, demonstrating dose-dependent responses. The efficacy presented at this conference resulted from JW0061 directly binding to the GFRA1 protein in dermal papilla cells and activating the Wnt signaling pathway. JW Pharmaceutical previously gained attention by presenting JW0061 as the first low-molecular-weight drug to elucidate the hair growth mechanism at the ‘Wnt 2022’ conference. JW0061 is an innovative first-in-class drug candidate that promotes hair follicle proliferation and hair regeneration by activating the Wnt signaling pathway. This substance was discovered through JW Pharmaceutical’s AI-based data science platform, 'JWELRY.' The Wnt signaling pathway plays a crucial role in skin development and hair follicle formation during embryogenesis and body growth, promoting hair follicle differentiation of skin stem cells. It is particularly involved in the proliferation of dermal papilla cells, located at the base of hair follicles, regulating hair growth and maintenance. Based on the various preclinical results, including those presented at this conference, and the toxicity evaluations completed according to GLP (Good Laboratory Practice) at overseas institutions, JW Pharmaceutical plans to initiate Phase 1 clinical trials within this year. A representative from JW Pharmaceutical stated, “We expect JW0061 to emerge as a promising treatment option that can complement or replace existing hair loss treatments in the global market through this conference presentation. We will develop JW0061 into a global innovative hair loss treatment that is safe and effective for both male and female hair loss patients.” Meanwhile, JW0061 was selected as a project supported by the National New Drug Development Institute last year and received funding for preclinical research. 

About Hair Loss Disorders and Treatment Market

Hair loss can be caused by various factors, including genetic predisposition, autoimmune diseases, and mental stress, leading to a rapid increase in patient numbers. Currently, the main first-line treatments for hair loss are finasteride, dutasteride, and minoxidil. Finasteride and dutasteride are prescribed for male patients and can cause mechanism-specific side effects. Minoxidil is primarily prescribed for female patients, but its hair growth mechanism is unclear, and hair loss recurs soon after discontinuation. Therefore, there is a significant unmet need for a hair loss treatment with a clear mechanism of action that is safe for both men and women. According to the National Institutes of Health, hair loss affects 42% of men worldwide, with approximately 80 million people (25% of the total population) affected in the United States. The Korean Hair Society estimates the domestic hair loss population to be around 10 million (20% of the total population). Market research agencies estimate the global hair loss treatment market to be valued at 10 trillion KRW as of 2021, growing at an annual rate of 8.2%. The market is projected to reach nearly 16 trillion KRW by 2028 (Research and Markets, 2021). 

About the Wnt Signaling Pathway

The Wnt signaling pathway is present across all species, from nematodes and fruit flies to mammals, and plays a crucial role in cell proliferation, differentiation, organ development, and morphogenesis, as revealed by various studies over the past 40 years. Inhibiting the Wnt pathway suppresses the formation, proliferation, and metastasis of cancer cells within various tissues. Conversely, activating the Wnt pathway promotes tissue regeneration by stimulating stem cells and inducing cell proliferation. Despite its influence on many human diseases, there are currently no drugs targeting the Wnt pathway. 

About the Development of Innovative New Drugs Targeting Wnt and STAT Based on Data Science Platforms

Since the 2010s, JW Pharmaceutical has been building AI and deep learning-based data science platforms ‘JWELRY’ and ‘CLOVER,’ in addition to chemical and bioinformatics big data. Through these platforms, JW Pharmaceutical continues to generate new drug candidates targeting Wnt and STAT for cancer, regenerative medicine, and immune diseases. Since the early 2000s, JW Pharmaceutical has focused on developing new drugs targeting the Wnt signaling pathway. Currently, the company utilizes 'JWELRY' (JW Excellent LibraRY), a core proprietary technology specializing in the Wnt signaling pathway. JWELRY is based on a library of tens of thousands of compounds involved in the Wnt pathway, along with drug evaluation and mechanism research technologies for distinguishing inhibition and activation of the Wnt pathway. Through JWELRY, JW Pharmaceutical has identified various new drug candidates, including JW0061, and is generating new projects. In addition, JW Pharmaceutical is actively developing new drugs targeting the STAT protein through ‘CLOVER’ (C&C research Laboratories Omics serVER), established by the research subsidiary C&C New Drug Research Institute. The company focuses on discovering innovative new drug candidates targeting each subtype of the STAT protein, such as the STAT3-targeted anticancer drug JW2286, a STAT3 atopic dermatitis treatment, a STAT5-targeted anticancer drug (for hematologic cancers), and a STAT3-ADC anticancer drug (for solid tumors). Furthermore, JW Pharmaceutical is conducting joint research incorporating AI and organoid platforms from domestic and international biotech companies to explore new indications for its drug candidates and validate their development potential.

 -Discovered through the new drug development platform ‘JWELRY,’ targeting the Wnt signaling pathway for hair loss treatment-First efficacy evaluation on human skin organoid model revealed at international dermatology research conference May 2024 JW Pharmaceutical announced on the 7th that it will present the preclinical results of its Wnt-targeted hair loss treatment ‘JW0061’ through a poster presentation at the Society of Investigative Dermatology conference, which will be held from May 15 to 18 in Dallas, Texas. JW0061 is an innovative new drug (First-in-Class) candidate that promotes hair follicle proliferation and hair regeneration by activating the Wnt signaling pathway. This substance was discovered through JW Pharmaceutical’s AI-based data science platform, ‘JWELRY.’ The Wnt signaling pathway plays a crucial role in skin development and hair follicle formation during embryogenesis and body growth, promoting hair follicle differentiation of skin stem cells. It is particularly involved in the proliferation of dermal papilla cells, which are located at the base of hair follicles and regulate hair growth and maintenance. JW0061 directly binds to the GFRA1 protein in dermal papilla cells, activating the Wnt signaling pathway. JW Pharmaceutical previously garnered attention by presenting JW0061 as the first low-molecular-weight drug to elucidate the hair growth mechanism at the ‘Wnt 2022’ conference. At this year’s Society of Investigative Dermatology conference, JW Pharmaceutical will reveal efficacy evaluation results on various models, including human skin organoids. Organoids are next-generation drug development technologies that can replicate the structural and functional characteristics of patient organ tissues. They can replace animal testing and predict clinical trial results without directly conducting human trials. Based on preclinical research results, JW Pharmaceutical plans to initiate Phase 1 clinical trials within this year. The company has already completed toxicity evaluations according to GLP (Good Laboratory Practice) at overseas institutions and is producing clinical-grade drugs. A JW Pharmaceutical representative stated, “Various preclinical tests have confirmed JW0061’s excellent hair growth and hair follicle regeneration effects. Through this conference presentation, we aim to establish the potential of JW0061 as an innovative treatment option that can complement and replace existing hair loss treatments internationally.” Meanwhile, JW0061 was selected as a project supported by the National New Drug Development Institute last year and received funding for preclinical research. 

About Hair Loss Disorders and Treatment Market

Hair loss can be caused by various factors, including genetic predisposition, autoimmune diseases, and mental stress, leading to a rapid increase in patient numbers. Currently, the main first-line treatments for hair loss are finasteride, dutasteride, and minoxidil. Finasteride and dutasteride are prescribed for male patients and can cause mechanism-specific side effects. Minoxidil is primarily prescribed for female patients, but its hair growth mechanism is unclear, and hair loss recurs soon after discontinuation. Therefore, there is a significant unmet need for a hair loss treatment with a clear mechanism of action that is safe for both men and women. According to the National Institutes of Health, hair loss affects 42% of men worldwide, with approximately 80 million people (25% of the total population) affected in the United States. The Korean Hair Society estimates the domestic hair loss population to be around 10 million (20% of the total population). Market research agencies estimate the global hair loss treatment market to be valued at 10 trillion KRW as of 2021, growing at an annual rate of 8.2%. The market is projected to reach nearly 16 trillion KRW by 2028 (Research and Markets, 2021). 

About the Wnt Signaling Pathway

The Wnt signaling pathway is present across all species, from nematodes and fruit flies to mammals, and plays a crucial role in cell proliferation, differentiation, organ development, and morphogenesis, as revealed by various studies over the past 40 years. Inhibiting the Wnt pathway suppresses the formation, proliferation, and metastasis of cancer cells within various tissues. Conversely, activating the Wnt pathway promotes tissue regeneration by stimulating stem cells and inducing cell proliferation. Despite its influence on many human diseases, there are currently no drugs targeting the Wnt pathway. 

About the Development of Innovative New Drugs Targeting Wnt and STAT Based on Data Science Platforms

Since the 2010s, JW Pharmaceutical has been building AI and deep learning-based data science platforms ‘JWELRY’ and ‘CLOVER,’ in addition to chemical and bioinformatics big data. Through these platforms, JW Pharmaceutical continues to generate new drug candidates targeting Wnt and STAT for cancer, regenerative medicine, and immune diseases. Since the early 2000s, JW Pharmaceutical has focused on developing new drugs targeting the Wnt signaling pathway. Currently, the company utilizes 'JWELRY' (JW Excellent LibraRY), a core proprietary technology specializing in the Wnt signaling pathway. JWELRY is based on a library of tens of thousands of compounds involved in the Wnt pathway, along with drug evaluation and mechanism research technologies for distinguishing inhibition and activation of the Wnt pathway. Through JWELRY, JW Pharmaceutical has identified various new drug candidates, including JW0061, and is generating new projects. In addition, JW Pharmaceutical is actively developing new drugs targeting the STAT protein through ‘CLOVER’ (C&C research Laboratories Omics serVER), established by the research subsidiary C&C New Drug Research Institute. The company focuses on discovering innovative new drug candidates targeting each subtype of the STAT protein, such as the STAT3-targeted anticancer drug JW2286, a STAT3 atopic dermatitis treatment, a STAT5-targeted anticancer drug (for hematologic cancers), and a STAT3-ADC anticancer drug (for solid tumors).Furthermore, JW Pharmaceutical is conducting joint research incorporating AI and organoid platforms from domestic and international biotech companies to explore new indications for its drug candidates and validate their development potential.

 -DSMB's primary evaluation based on safety data from gout patients is positive-Accelerating Phase 3 trials in five Asian countries, including Korea... Evaluating uric acid reduction efficacy and safety-hURAT1 inhibition mechanism promotes uric acid excretion... Expected to be a 'Best-in-Class' global gout treatment February 2024 JW Pharmaceutical announced on the 14th that the U.S. Drug Safety Monitoring Board (DSMB) has positively assessed the primary results of the multinational Phase 3 trial of their gout treatment, Epaminurad (code name URC102), and recommended the continuation of the trial. The DSMB, composed of experts in rheumatology, pharmacology, nephrology, and statistics, reviewed the Phase 3 clinical data of Epaminurad on January 31 (local time) to discuss the trial’s continuation. The board unanimously recommended continuing the trial based on the review of safety data from currently enrolled gout patients. Following this first DSMB decision, JW Pharmaceutical plans to accelerate the Phase 3 trial of Epaminurad, which is being developed as a Best-in-Class new drug. Epaminurad, an oral medication, selectively inhibits hURAT1 (human uric acid transporter-1), promoting uric acid excretion. It is an effective candidate for treating hyperuricemia and gout, conditions characterized by abnormally high uric acid levels in the blood. The Phase 3 trial of Epaminurad involves 588 gout patients across five Asian countries, including Korea, and evaluates its efficacy (uric acid reduction) and safety compared to febuxostat. Alongside the Phase 3 clinical trials, JW Pharmaceutical is pursuing global technology licensing (License-Out) for Epaminurad. In 2019, they out-licensed the development and sales rights for China, Hong Kong, and Macau to Simcere Pharmaceutical Group. A JW Pharmaceutical representative stated, “The recommendation from the first DSMB to continue the global clinical development of Epaminurad positively reflects the drug’s competitiveness, patient safety, and data reliability. We plan to accelerate the development of Epaminurad as a global gout treatment to address significant unmet medical needs.” 

About Gout Treatments

Gout is classified into two types: the first is the 'underexcretion type,' where uric acid is not excreted effectively, unlike in healthy individuals, and the second is the 'overproduction type,' where the body produces more uric acid than normal. Currently, medications that decrease uric acid production are available, but treatments that promote uric acid excretion for underexcretion-type gout are not widely prescribed due to safety concerns for the kidneys and liver, despite the large number of patients. Additionally, allopurinol, a uric acid production inhibitor commonly prescribed as a first-line treatment, has weak effects at initial doses, and febuxostat is excluded as a first-line treatment in the US due to safety concerns. Therefore, the need for 'safe' gout treatments is greater than ever. In Phase 2 trials, Epaminurad met all primary and secondary efficacy endpoints and demonstrated excellent safety and tolerability in gout patients. 

Gout Disease and Market Size

Known for causing extreme pain even with the slightest touch, gout is a representative intractable disease. With the westernization of dietary habits, the incidence rate among younger people has significantly increased, with the number of patients in Korea rising sharply from 334,705 in 2015 to 509,699 in 2022 (according to the Health Insurance Review and Assessment Service’s Healthcare Big Data Open System). Gout is a metabolic disease that occurs when uric acid produced in the body is not excreted through urine and accumulates. Uric acid remains in the blood or joint fluid in crystal form and deposits in cartilage, tissues around joints, or subcutaneous tissues, causing inflammation and pain. Common causes include regular meat consumption, excessive drinking, and overeating. Recently, it has been occurring across various age groups without age specificity. Like hypertension or diabetes, once it develops, it is considered a chronic intractable disease that requires continuous treatment. With the increasing number of patients, the gout treatment market is also growing significantly. According to a report by Grand View Research, the global gout treatment market, currently valued at approximately 3 trillion KRW, is projected to grow to about 10 trillion KRW (8.3 billion USD) by 2025.

 -Phase 3 IND approvals completed in 5 Asian countries, including Korea... Efficacy in reducing blood uric acid and safety to be rigorously evaluated-hURAT1 inhibition mechanism promotes uric acid excretion... Expected to be a 'Best-in-Class' global gout treatment January 2024 JW Pharmaceutical announced on the 15th that it has received approval from Malaysia’s National Pharmaceutical Regulatory Agency (NPRA) for the Phase 3 clinical trial (IND) of its gout treatment, Epaminurad (code name URC102). The Phase 3 trial of Epaminurad will evaluate its efficacy (blood uric acid reduction) and safety compared to febuxostat in a total of 588 gout patients across five Asian countries, including Korea. With this approval, JW Pharmaceutical has now received all necessary multinational Phase 3 IND approvals for Epaminurad. In Korea, the IND was approved in November 2022, and patient enrollment and dosing began in March last year. In Taiwan, the IND was approved in August 2023, with the first patient registered by December. Subsequently, IND approvals were obtained in Thailand and Singapore in September. Epaminurad, developed as an oral medication, selectively inhibits hURAT1 (human uric acid transporter-1), promoting uric acid excretion and is an effective candidate for hyperuricemia and gout, conditions characterized by abnormally high uric acid levels in the blood. In the domestic Phase 2b clinical trial, completed in March 2021, JW Pharmaceutical confirmed Epaminurad's excellent tolerability and safety, meeting all primary and secondary efficacy endpoints. Alongside the Phase 3 clinical trials, JW Pharmaceutical is also pursuing global technology licensing (License-Out) for Epaminurad. In 2019, it out-licensed the development and sales rights for China, Hong Kong, and Macau to Simcere Pharmaceutical Group. A JW Pharmaceutical representative stated, “With the Malaysia IND approval, we can now proceed with Phase 3 clinical trials of Epaminurad across five Asian countries. Given the confirmed tolerability and safety in the previous Phase 2b trial, we aim to proceed with Phase 3 trials without setbacks to develop a Best-in-Class treatment for the significant unmet medical need in the global gout market.” 

About Gout Treatments

Gout is classified into two types: the first is the 'underexcretion type,' where uric acid is not excreted effectively, unlike in healthy individuals, and the second is the 'overproduction type,' where the body produces more uric acid than normal. Currently, medications that decrease uric acid production are available, but treatments that promote uric acid excretion for underexcretion-type gout are not widely prescribed due to safety concerns for the kidneys and liver, despite the large number of patients. Additionally, allopurinol, a uric acid production inhibitor commonly prescribed as a first-line treatment, has weak effects at initial doses, and febuxostat is excluded as a first-line treatment in the US due to safety concerns. Therefore, the need for 'safe' gout treatments is greater than ever. In Phase 2 trials, Epaminurad met all primary and secondary efficacy endpoints and demonstrated excellent safety and tolerability in gout patients. 

Gout Disease and Market Size

Known for causing extreme pain even with the slightest touch, gout is a representative intractable disease. With the westernization of dietary habits, the incidence rate among younger people has significantly increased, with the number of patients in Korea rising sharply from 334,705 in 2015 to 509,699 in 2022 (according to the Health Insurance Review and Assessment Service’s Healthcare Big Data Open System). Gout is a metabolic disease that occurs when uric acid produced in the body is not excreted through urine and accumulates. Uric acid remains in the blood or joint fluid in crystal form and deposits in cartilage, tissues around joints, or subcutaneous tissues, causing inflammation and pain. Common causes include regular meat consumption, excessive drinking, and overeating. Recently, it has been occurring across various age groups without age specificity. Like hypertension or diabetes, once it develops, it is considered a chronic intractable disease that requires continuous treatment. With the increasing number of patients, the gout treatment market is also growing significantly. According to a report by Grand View Research, the global gout treatment market, currently valued at approximately 3 trillion KRW, is projected to grow to about 10 trillion KRW (8.3 billion USD) by 2025.

 -Optimization of Lead Compounds for Direct STAT6 Inhibition-Utilization of XtalPi’s AI Drug Development and Automated Synthesis Platforms December 2023 C&C Research Laboratories, a subsidiary of JW Pharmaceutical, announced on the 18th that it has entered into a collaborative research agreement with XtalPi, a US-based AI-driven drug development company, to develop small molecule therapeutics targeting STAT6 Through this agreement, C&C Research Laboratories will utilize XtalPi’s quantum physics-based AI drug development platform and automated robotic system to optimize lead compounds for STAT6-targeted small molecule therapeutics. STAT6 is known as a key protein that regulates the inflammatory immune response of type 2 helper T cells (Th2). These cells are activated by inflammatory cytokines such as interleukin-4 (IL-4) and interleukin-13 (IL-13), which are implicated in conditions like asthma and atopic dermatitis. Currently, biological agents targeting IL-4, IL-13, and their receptors, as well as JAK inhibitors, which target upstream proteins of STAT, are used as anti-inflammatory treatments. However, there are no successful examples of developing small molecule therapeutics targeting STAT6 to date. Founded in 2015 by quantum physics experts from MIT, XtalPi is an AI drug R&D platform company. It possesses core technologies in quantum mechanics-based AI drug development and automated synthesis platforms, aiming to reduce the time and effort required for drug development and transform the existing drug research ecosystem. Previously, C&C Research Laboratories secured lead compounds with direct binding affinity to STAT6 through collaborative research with XtalPi. The current joint research aims to optimize these lead compounds to develop a STAT6-targeted drug candidate. A representative from C&C Research Laboratories stated, "There is a high medical demand for new targeted therapies due to resistance and side effects associated with existing anti-inflammatory treatments. We aim to develop a STAT6-targeted anti-inflammatory therapeutic for patient groups with high unmet needs in existing treatments." About C&C Research Laboratories C&C Research Laboratories is the first Korea-Japan joint venture bio-venture established by JW Pharmaceutical and Chugai Pharmaceutical of the Roche Group in 1992. It became a wholly-owned subsidiary of JW Pharmaceutical in 2020. Since 2010, C&C Research Laboratories has been conducting its own research projects, leveraging its R&D platform 'CLOVER,' which integrates chemical and biological informatics big data with AI and deep learning technologies, to discover innovative first-in-class drug candidates. The institute currently has over ten new drug pipelines targeting oncology and immune diseases. Notable projects include the gout treatment 'Epaminurad (code name URC102),' and the STAT3-targeted anticancer agent 'JW2286,' both of which have been licensed to JW Pharmaceutical. Epaminurad is undergoing multinational phase 3 clinical trials, and JW2286 aims to start phase 1 clinical trials in 2024. The institute is also focused on discovering new drugs targeting STAT proteins (STAT1-6) that regulate cell growth, mutation, and proliferation. Prior to the development of STAT6-targeted therapeutics, C&C Research Laboratories has been developing STAT3-targeted atopic dermatitis treatments and dual inhibitors of STAT5/3, which were selected for national new drug development projects in 2021 and 2023, respectively. Additionally, the institute actively engages in joint research with domestic and international biotechs by integrating AI and organoid platforms to explore and validate new indications for its proprietary drug candidates. About XtalPi XtalPi is an innovative drug development company focused on AI and robotic automation synthesis. Established in 2015 by quantum mechanics experts from MIT, XtalPi leads digital transformation in the life sciences and new materials industries. The company provides various technological solutions, services, and products to life sciences and new materials companies through its cloud-based quantum physics and AI platforms, and large-scale robotic automated synthesis systems. About STAT Proteins The JAK-STAT pathway is a representative signaling system in the body involved in cell development, differentiation, growth, survival, and various immune responses. STAT proteins consist of seven types with different functions: STAT1-4, STAT5A, STAT5B, and STAT6, activated by upstream proteins JAK. While JAK-STAT pathways are normally regulated within a physiological range, abnormal activation (overexpression or hyperactivation) of these proteins can lead to various diseases. JW Pharmaceutical and C&C Research Laboratories aim to overcome the side effects of JAK inhibitors and achieve powerful effects by developing specific small molecule inhibitors that directly bind to and inhibit STAT3, STAT5, and STAT6.