SillaJen: First to use lab-grown human tissue to find best cancer drug dose

South Korean biotechnology firm SillaJen Inc. is pioneering a new approach to determine the best dose for a cancer drug combination in clinical trials.

The company said it is using organoids – small lab-grown tissues that mimic human organ structures – and microphysiological system (MPS) technology to determine the recommended phase 2 dose, known as RP2D.

Even globally, successfully determining RP2D using MPS will position SillaJen at the forefront of innovation in clinical development, analysts said.

The company recently said it will apply for regulatory approval in Korea and the US in the second quarter to test a combination of its cancer drug candidate, BAL0891, with immunotherapy drug Tevimbra (tislelizumab).

“We will determine the best dose for phase 2 trials based on data we obtained from earlier patient studies and new findings from our organoid models,” said a SillaJen official.

A FIRST-IN-CLASS DUAL-TARGETING CANCER THERAPY

BAL0891 is a new type of cancer drug that targets two key proteins – TTK and PLK1 – that cancer cells rely on for proliferation. By blocking the two proteins, BAL0891 causes cancer cells to start dividing but then fail and die.

Preclinical studies have shown that BAL0891 effectively inhibits an array of cancer cells including those of triple-negative breast, endometrial, colorectal, urothelial, gastric and kidney cancer.

SillaJen’s BAL0891, a first-in-class dual-targeting oncology drug, was licensed by Swiss biotech company Basilea Pharmaceutica Ltd. in 2021.

PARTNERSHIP WITH QUREATOR

To assess the synergy between BAL0891 and immune checkpoint inhibitors, SillaJen partnered in June 2024 with Qureator, a San Diego-based biotech firm specializing in 3D disease modeling.

Qureator has been testing BAL0891 in combination with Keytruda, a PD-1 inhibitor used in cancer immunotherapy, in organoid models of triple-negative breast cancer (TNBC), renal cell carcinoma (RCC), gastric cancer (GC) and colorectal cancer (CRC).

In an interview with Hankyung Bio Insight, a biotech outlet of The Korea Economic Daily, Yoo Sanghee, head of research at Qureator, said a combination of BAL0891 and MPS technology demonstrated a 30% increase in efficacy in TNBC models than using BAL0891 alone.

Qureator was co-founded by Jeon Noo-li, a mechanical engineering professor at Seoul National University and a leading expert in MPS technology, and former Vertex Pharmaceuticals scientist Kyu Baek.

The company is recognized for its ability to replicate the tumor microenvironment (TME) – a protective "shield" that tumors form around themselves to resist treatment.

“Many drugs work well in animal tests but fail in human trials because the human body is different. Even organoid studies must accurately recreate real-life conditions, such as blood flow and immune system activity, to be useful,” Jeon said.

QUREATOR’S TECHNOLOGY

Qureator's technology creates a 3D human-like tumor environment on a lab chip by including blood vessels, immune cells (T cells) and support cells.

Qureator researchers said the MPS technology has played a crucial role in RP2D selection.

“The US Food and Drug Administration is also encouraging companies to use biomarkers rather than relying solely on maximum tolerated dose when determining RP2D,” Yoo said.

SillaJen plans to present additional findings at the American Association for Cancer Research (AACR) annual meeting in Chicago next month.

“Based on our research with Qureator, we will move forward with clinical trials combining BAL0891 with Tevimbra, an immunotherapy similar to Keytruda,” said a SillaJen official.

If successful, SillaJen could set a new global standard for using organoid technology to improve cancer drug development, making clinical trials faster, safer and more effective, analysts said.

Write to Woo-Sang Lee at idol@hankyung.com

In-Soo Nam edited this article.