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Aptamer Sciences Inc. Files IND Application for Innovative Liver Cancer Treatment AST-201: A Promising Approach to Address Under Medical Needs

2024-03-26

临床1期临床申请上市批准

SEONGNAM, South Korea, March 25, 2024 /PRNewswire/ -- Aptamer Sciences Inc. (KOSDAQ, 291650) announced the filing of an Investigational New Drug (IND) application for the phase 1 clinical trial of AST-201 with the Korean Ministry of Food and Drug Safety on March 12th, 2024.

AST-201 structure & mechanism of action

AST-201, the company's pioneering candidate in its pipeline, is an ApDCTM (Aptamer-Drug Conjugate) designed to target GPC3 protein, which exhibits high expression levels in specific cancer cells, notably liver cancer. It comprises gemcitabine as the payload and an aptamer that selectively binds to GPC3 protein. Gemcitabine, a proven systemic anticancer agent, is widely utilized in treating various cancers, including ovarian, breast, non-small cell lung, and pancreatic cancer. The choice of gemcitabine as the payload was deliberate, owing to its moderate toxicity profile, particularly suitable for liver targeting, given the organ's susceptibility to drug toxicity. Additionally, gemcitabine's nature as a DNA analogue facilitates its synthesis within the aptamer DNA sequence. The company's strategic focus on liver cancer addresses significant unmet medical needs, as evidenced by AST-201's superior efficacy over sorafenib in animal models and its synergistic effect when co-administered with anti-PD-1 therapy.

AST-201ancer poses a significant health challenge, particularly in East Asia, with a dismal survival rate. While combiGPC3 proteinpies such as atezolizumab and bevacizumab have emergcancerstandard treatmeliver cancernced liver cancgemcitabinefindings published in Nature Medicine suggest their limitGPC3 proteininGemcitabinever cancer populations with high GPC3 expression levels, highlighting substancancersmet medical needs. In this context, AST-201 offers a promising therapeutic approach by gemcitabiney targeting GPC3.gemcitabine liver cancer AST-201 sorafenib PD-1

Liver cancerphase 1 trial seeks to evaluate the safety, pharmacokinetic profile, and preliminary efficacy of AST-201 in patients with GPC3-posiatezolizumabd solbevacizumabNotably, Dr. Hong Jae Chon from CHA Bundaadvanced liver cancerprominent medical oncologist in South Korea, will serve as the coordinating investigator.liver cancerill be conducted acrossGPC3r hospitals, including CHA Bundang Medical Center, Samsung Medical Center, SeverancAST-201tal, and the National Cancer Center in South Korea.GPC3

Dr. Chon remarked, "GPC3 has garnered considerable attention in liver cancer therapeutic development among biAST-201ceutical companiesGPC3-positive advanced solid tumors GPC3-positive optimism that "AST-201 may emerge as a novel treatment CHA Bundang Medical Centertients."CHA Bundang Medical Center Severance Hospital National Cancer Center Cancer

Aptamer Sciences IncGPC3ms to showcase the clinical benefits of liver cancerthe technological value of its ApDCTM platform through this trial. Moreover, the companAST-201ipates that this milestone will propel its rliver cancerdevelopment efforts, enhancing the competitiveness of its subsequent hemato-oncology pipeline products, including AST-202 targeting CD25, ApRC (Aptamer Radioligand Conjugate), ApIS (Aptamer Immune Stimulator Conjugate), and so on.

Aptamer Sciences Inc.of Aptamer Sciences Inc., emphasized, "SincAST-201econd aptamer drug Izervay approved by FDA last year, aptamer technology has garnered significant attention in the pharmaceutical market," highlighting, "The AST-201 clinical trial will showcase the technological capabilities of the ApDCTM platform and expedite ongoingAST-202logy transfCD25iscussion with multiple partners. This milestone underscores Aptamer Sciences Inc.'s commitment to advancing innovative therapies and addressing critical unmet needs in oncology."

Fig 1. AST-201 structureAptamer Sciences Inc.of GPC3 aptamer, the payload of gemcitabine iIzervaygated as the FDAt of sequence.)AST-201 Aptamer Sciences Inc.

Fig 2. AST-201 mechanism of actionGPC3 aptamer GPC3 gemcitabine

1. AST-AST-201gets and binds to the GPC3 protein located on the surface of cancer cells.

2. AST-201g undergoes internalizatioGPC3 proteinancer cell by endocytosis.cancer

3. After the lysosomal degradation, gemcitabinecancerhosphate (dFdCMP), the active form of gemcitabine, is released within the cell leading to programmed cell death of the tumor cell (apoptosis).

About Aptamer Sciences, Inc.gemcitabine tumor

AptameAptamer Sciences, Inc.d on KOSDAQ in South Korea, is a global leading company in aptamer field. The company has established an integrated aptamer technology platform, called ApDCTM (Aptamer Drug Conjugate) and focused on developing oncology pipelines.

Aptamer Sciences Inc. please visit the website, www.aptsci.com/en or contact [email protected].

SOURCE Aptamer Sciences Inc.