Tirvalimogene Teraplasmid

Phase 3 clinical trial in China meets primary endpoint On track to in China in 2024 SEOUL, South Korea--(BUSINESS WIRE)-- Genexine Co. Ltd. (KOSDAQ:095700, CEO Neil Warma), a publicly traded, clinical-staged Korean biopharmaceutical company committed to the discovery and development of novel biologics for the treatment of unmet medical needs, today announced positive topline results from the multi-center, randomized, open-label, active-controlled pivotal phase 3 study (CTJ101PGHD301) being conducted in China to evaluate the efficacy and safety of eftansomatropin alfa (Genexine’s proprietary long-acting growth hormone, also known as GX-H9) in children with growth hormone deficiency. The study being conducted by Genexine’s partner in China, I-Mab, met its primary endpoint of annualized height velocity (AHV) at week 52 and demonstrated that eftansomatropin alfa was non-inferior to Norditropin®. Eftansomatropin alfa was given by weekly injection vs. Norditropin® given by daily injection. The mean AHV was 10.76 (cm/year) for eftansomatropin alfa vs. 10.28 (cm/year) for Norditropin®, with a difference of 0.47 [95% CI -0.06,1.00] and non-inferiority p-value <0.0001. Eftansomatropin alfa was well tolerated and no drug discontinuation was reported due to treatment related adverse events. The safety pro eftansomatropin alfa was comparable to Norditropin®. The Company is planning to BLA submission in China in 2024. “We are pleased with the results from this registration trial as this demonstrates clear safety and efficacy in this potential best-in-class drug to treat children with growth hormone deficiency,” said Neil Warma, Genexine President and CEO. “This is a significant milestone for Genexine as we demonstrate our ability to bring our novel products to market and the value of our proprietary long-acting hyFc® platform. We are excited to be preparing to submit the BLA in 2024 and advancing this product to market and to patients in China. We remain committed to our efforts to commercialize our leading products in various key markets.” About Eftansomatropin alfa (long-acting recombinant human growth hormone) GX-H9 has been developed utilizing Genexine’s hyFc® platform as a treatment for growth hormone deficiency (GHD). GX-H9 is currently being evaluated in a phase 3 registration trial in pediatric GHD in China with a BLA submission expected in 2024. GX-H9 is a potential best-in-class product that is differentiated by its extended half-life resulting in significantly fewer injections (weekly versus daily) and its improved safety profile. Patient compliance has been dramatically improved, which is critical for the pediatric population. GX-H9 is being co-developed with Handok under a strategic partnership and with I-Mab for development in greater China. I-Mab has partnered with Jumpcan for product launch and commercialization of eftansomatropin alfa in China. About Genexine Genexine, Inc. is a publicly traded, clinical-stage biotechnology company focused on developing and commercializing immunotherapeutics and next-generation long-acting biologics. Its primary technology platforms are Therapeutic DNA vaccine technology and hyFc® fusion technology. The Company has multiple products in clinical development including several undergoing Phase 3 registration trials. The Company's proprietary pipeline includes GX-188E (tirvalimogene teraplasmid) for head and neck cancer and cervical cancer, GX-I7 (efineptakin alfa) for multiple cancers, GX-H9 (eftansomatropin alfa) for Pediatric Growth Hormone Deficiency and GX-E4 for CKD-induced anemia, among others. Genexine has established multiple partnerships with global companies in order to expedite product development and commercialization and create significant value. Genexine is listed on the Korean exchange (KOSDAQ: 095700) and is headquartered in Seoul, Korea. Genexine is committed to the well-being and care of patients worldwide. Forward Looking Statements This press release contains forward-looking statements regarding the business of Genexine, Inc. ("Genexine"). Any statement describing Genexine's goals, expectations, financial or other projections, intentions or beliefs, development plans and the commercial potential of Genexine's drug development pipeline, including without limitation GX-I7 (efineptakin alfa), GX-188E, GX-H9 (eftansomatropin alfa), GX- E4 is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to risks and uncertainties, particularly those challenges inherent in the process of discovering, developing and commercialization of new drug products that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such drugs. Genexine's forward-looking statements also involve assumptions that, if they never materialize or prove correct, could cause its results to differ materially from those expressed or implied by such forward-looking statements. Although Genexine's forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Genexine. As a result, you are cautioned not to rely on these forward-looking statements. These and other risks concerning Genexine's programs are described in additional detail in Genexine's annual reports on the DART (Data Analysis, Retrieval and Transfer System) internet site ( ) of the Korean Financial Services Commission. Genexine assumes no obligation to update the forward-looking statements or to update the reasons why actual results could differ from those projected in the forward-looking statements, except as required by law.

Triple combination using Genexine’s DNA vaccine GX-188E, its long-acting Interleukin-7 GX-I7 and Keytruda showed a major pathological response rate of 63.6% in a phase 2 trial, satisfying primary evaluation criteria A promising new therapeutic option for HPV-positive HNSCC patients as a neoadjuvant therapy SEOUL, South Korea--(BUSINESS WIRE)-- Genexine (KQ 095700, CEO Neil Warma), a publicly traded, clinical-staged Korean biopharmaceutical company committed to the discovery and development of novel biologics for the treatment of unmet medical needs, announced the publication of an abstract of its phase 2 clinical trial on triple combination neoadjuvant therapy for HNSCC (head and neck squamous cell carcinoma) in the ASCO (American Society of Clinical Oncology). The study evaluated the DNA vaccine GX-188E (tirvalimogene teraplasmid) and the lymphopenia-correcting immune-oncology drug GX-I7 (efineptakin alfa) in combination with immune checkpoint inhibitor Keytruda® (pembrolizumab). The phase 2 investigator-initiated clinical trial (IIT) being conducted in South Korea led by Professor Hye-Ryun Kim, Division of Medical Oncology of Yonsei University Severance Hospital, and jointly conducted with a research team of Professor Yoon Woo Koh, Department of Otolaryngology, enrolled a total of 11 patients with HPV-positive cervical cancer who were scheduled for surgery. The patients received Keytruda 200mg on day 1 and 22, Genexine’s GX-188E DNA vaccine 2mg on day 1, 8, 22 and GX-I7 on day 8 to amplify the number of T cells before surgery. The primary endpoint evaluated the major pathological response (MPR) and other evaluation criteria included safety, recurrence rate and survival rate. All 11 patients who participated in the trial underwent surgery as planned after neoadjuvant therapy with no increase in surgical delay or surgical complications. Seven patients (63.6%) showed a major pathological response (MPR), and four patients (36.3%) achieved a pathological complete response (pCR), indicating satisfactory primary evaluation variables. Furthermore, comparative analysis of the tissue before and after combination therapy revealed an increase in follicular helper T cells (CD4+) and reactivation of killer T cells (CD8+) in the tumor microenvironment. Using AI-based analysis technology, the triple combination therapy was found to increase the density of tumor-infiltrating lymphocytes (TIL) and completely transform tumors classified as immune-desert or immune-excluded types into inflamed tumors. Professor Hye-Ryun Kim, who led the clinical trial, said, “Through this clinical study conducted for human papillomavirus (HPV)-positive HNSCC patients, the efficacy and safety of the triple combination therapy appear to be confirmed, and the therapy could become an effective new treatment strategy for HPV positive HNSCC patients in the future." "We are pleased by these early data in this important trial as this is the first time this combination therapy has been used,” said Neil Warma, President and CEO of Genexine. “Head and neck cancer remains such a serious unmet need and we believe there is potential to expand the label for GX-188E beyond cervical cancer to include HNSCC." HNSCC cancer is one of the malignant tumors that occur on the patient's facial area and is commonly caused by factors such as smoking and high-risk HPV infection. While various treatment methods such as surgery, radiation therapy and chemotherapy have been used in the past, the size of the surgical area can significantly impact the patient's quality of life. Therefore, immunotherapy has been gaining attention as a treatment option along with the development of immune checkpoint inhibitors. The results of this study will be presented in a poster session at the ASCO 2023 Annual Meeting, which will be held in Chicago from June 2 to 6, 2023. Details of the poster presentation are as follows: Abstract Title Neoadjuvant pembrolizumab, GX-188E, and GX-I7 in patients with human papillomavirus-16- and/or 18-positive head and neck squamous cell carcinoma: Single-arm, phase 2 trial with single cell transcriptomic analysis and artificial intelligence-powered spatial analysis. Session Title Head and Neck Cancer Abstract Number 6075 Date and Time June 5, 2023, 1:15 - 4:15 PM About Genexine Genexine, Inc. is a publicly traded, clinical-stage biotechnology company focused on developing and commercializing immunotherapeutics and next-generation long-acting biologics. Its primary technology platforms are Therapeutic DNA vaccine technology and hyFcR fusion technology. The Company has multiple products in clinical development including several undergoing Phase 3 registration trials. The Company's proprietary pipeline includes GX-188 (tirvalimogene teraplasmid) for cervical cancer, GX-I7 (efineptakin alfa) for multiple cancers, GX-H9 (eftansomatropin alfa) for Pediatric Growth Hormone Deficiency and GX-E4 for CKD-induced anemia, among others. Genexine has established multiple partnerships with global companies in order to expedite product development and commercialization and create significant value. Genexine is listed on the Korean exchange (KOSDAQ: 095700) and is headquartered in Seoul, Korea. Genexine is committed to the well-being and care of patients worldwide. Forward Looking Statements This press release contains forward-looking statements regarding the business of Genexine, Inc. ("Genexine"). Any statement describing Genexine's goals, expectations, financial or other projections, intentions or beliefs, development plans and the commercial potential of Genexine's drug development pipeline, including without limitation GX-I7 (efineptakin alfa), GX-188E, GX-H9 (eftansomatropin alfa), GX- E4 is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to risks and uncertainties, particularly those challenges inherent in the process of discovering, developing and commercialization of new drug products that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such drugs. Genexine's forward-looking statements also involve assumptions that, if they never materialize or prove correct, could cause its results to differ materially from those expressed or implied by such forward-looking statements. Although Genexine's forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Genexine. As a result, you are cautioned not to rely on these forward-looking statements. These and other risks concerning Genexine's programs are described in additional detail in Genexine's annual reports on DART (Data Analysis, Retrieval and Transfer System) internet site ( ) of the Korean Financial Services Commission. Genexine assumes no obligation to update the forward-looking statements, or to update the reasons why actual results could differ from those projected in the forward-looking statements, except as required by law. View source version on businesswire.com: Contacts For further information, please contact: Mr. Jongsoo Lee Investor Relations, Genexine, Inc. jongsoo.lee@genexine.com Source: Genexine, Inc. View this news release online at: