Genome&Company "Beyond Microbes to New Anticancer Drugs... Achieving Results Within the Year"

Expecting Technology Export of New Pipeline Within the Year
'GENA-114' Targeting PD-1 Non-Responders
New Target ADC 'GENA-111'

Genome & Company, recognized as a specialist in microbiome therapeutics development, has announced plans to soon achieve concrete results in research and development (R&D), including a successful technology export deal for a new target anticancer drug.

Bae Jisoo, CEO of Genome & Company, is speaking at a press conference held on the 21st in Yeouido, Yeongdeungpo-gu, Seoul.
[Photo by Lee Chunhee]

On the 21st, at a press conference held in Yeouido, Yeongdeungpo-gu, Seoul, CEO Bae Jisoo of Genome & Company stated, "Our company has long been working on both new target anticancer drugs and microbiome anticancer drugs, but the market mainly recognizes us as a microbiome company." He added, "The purpose of this conference is to show results for the new target anticancer drug soon." He emphasized that "we are currently making great efforts for many partnerships and believe we can secure a meaningful deal within this year," highlighting that concrete achievements in the new target anticancer drug area will be made shortly.

Technology Export Success for New Target Anticancer Drugs Expected Within the Year

The subjects are 'GENA-104', for which a clinical Phase 1 trial application (IND) is planned this month, and 'GENA-111', an antibody-drug conjugate (ADC) co-developed with Swiss company Debiopharm.

GENA-104 is a new target anticancer drug inspired by reversing the 'programmed cell death protein (PD)-(L)1 inhibitor' class, which currently holds a dominant position in the immune checkpoint inhibitor field. The PD-(L)1 class is attracting attention as the broadest range anticancer drug, with the representative drug MSD's 'Keytruda' (active ingredient pembrolizumab) in the U.S. expected to achieve $24 billion (approximately 32 trillion KRW) in sales this year, positioning it as the top-selling drug globally.

However, even when patients with high PD-(L)1 expression levels are selected for treatment, about 80-90% do not respond, revealing limitations. In analyzing the characteristics of these non-responders, the company discovered a new protein target called CNTN4.

They found that even if PD-(L)1 expression is high, if CNTN4 is also highly expressed simultaneously, there is no response to PD-(L)1 inhibitors. In fact, among 19 patients with high expression of both proteins, 18 did not respond to PD-(L)1, whereas when CNTN4 expression was low, about half were non-responders. Furthermore, progression-free survival (PFS) and overall survival (OS), direct indicators of anticancer drug efficacy, also varied according to CNTN4 expression levels in PD-(L)1 treatment.

Chamiyeong, Head of New Drug Research at Genome & Company, is explaining the status of new drug R&D at a press briefing held on the 21st in Yeouido, Yeongdeungpo-gu, Seoul.
[Photo by Lee Chunhee]

Accordingly, Genome & Company currently holds patents for the CNTN4 target in Korea, Japan, and other countries and continues related development. Director Cha Miyoung of the New Drug Research Institute explained, "GENA-104 binds to CNTN4, enhancing T-cell activation, ultimately inducing cancer cell death, thereby exhibiting anticancer effects." She emphasized its potential, noting that it was confirmed to be expressed in over 70% of patients with bladder cancer, prostate cancer, melanoma, liver cancer, endometrial cancer, stomach cancer, pancreatic cancer, and gallbladder cancer.

Director Cha added, "We confirmed that GENA-104 shows excellent anticancer effects as a monotherapy." While combination therapy could enhance efficacy in the future, the initial focus will be on developing monotherapy to prove GENA-104's effectiveness. She also mentioned that if CNTN4 is expressed in cancer cells, the 'amyloid precursor protein (APP)', which appears to bind to it in T-cells, is also being studied. She explained, "Recently, 'GENA-119' was selected as a lead compound stage project by the Korea Drug Development Fund (KDDF)."

ADC Aiming for Internalization... Simultaneous Development with Microbiome Possible Through Platform

Regarding antibody-drug conjugates (ADC), a modality recently gaining industry attention, Genome & Company is developing CD239 as a new target, rather than the commonly developed targets such as human epidermal growth factor receptor (HER)2 and trophoblast cell surface antigen (TROP)-2. Director Cha stated, "There is so much R&D that it is called an ADC renaissance," and expressed expectations for increased demand for new ADCs utilizing novel targets, highlighting growth potential. She noted that CD239, being developed as 'GENA-111', shows an outstanding selective expression rate about 23 times higher in cancer cells compared to normal cells, with ovarian cancer as the primary indication.

Currently, the company is focusing on antibody development and pursuing a technology export model, but plans to move toward creating proprietary ADCs by internalizing proven linkers and payloads in the future. Director Cha explained, "Having ADC data can create more commercialization opportunities," and added, "We aim to develop ADCs using linkers and payloads without intellectual property (IP) issues."

The company cited its proprietary platform 'GNOCLE' as the reason simultaneous development of new target anticancer drugs and microbiome therapeutics is possible. Through this, they have established the 'GNOCLE-M' platform for microbiome and the 'GNOCLE-N' platform for new target anticancer drugs. Vice President Park Kyungmi emphasized, "We have secured considerable technological capabilities based on genomics for microbiome and new target anticancer drug technologies." CEO Bae also pointed out, "We do not separate microbiome and new target anticancer drugs into two; we are diligently working on both as one core competency," adding, "The fundamental technology comes from GNOCLE, our genomic analysis technology."

This content was produced with the assistance of AI translation services.