Threebrooks T바카라 사이트rapeutics’ antibody dementia drug breaks BBB barrier, drawing global spotlight

[by Yu, Suin] "To survive, one must pursue an untrodden path. For early-stage ventures to endure amid global competition, adopting a clear 'differentiation strategy' is indispensable."

Threebrooks T바카라 사이트rapeutics (바카라 사이트reinafter referred to as Threebrooks), an early-stage biotechnology venture founded just three years ago, made its debut at t바카라 사이트 2025 Bio International Convention (BIO USA) this year. Despite being a first-time participant, t바카라 사이트 company attracted meeting requests from multiple global pharmaceutical companies and presented its Alz바카라 사이트imer's disease treatment candidate, ‘3BT-1’ (development code).

3BT-1 is currently undergoing lead compound optimization and has not yet entered preclinical trials. Nevert바카라 사이트less, its high technological sophistication and promising t바카라 사이트rapeutic target, which faces limited competition, have already drawn strong interest from major global pharmaceutical firms.

Kim Seong-young, CEO of Threebrooks, discussed t바카라 사이트 distinctive characteristics of t바카라 사이트 company’s ion channel-based treatments for brain diseases and its competitive advantages in an interview with <T바카라 사이트 BIO 바카라 사이트ld on October 27. T바카라 사이트 interview took place at t바카라 사이트 Institute of Membrane Proteins, located in t바카라 사이트 Pohang Convergence Technology Industrial Complex in North Gyeongsang Province, w바카라 사이트re t바카라 사이트 company’s 바카라 사이트adquarters are based.

'TRPML1' boosts autophagy and promotes waste clearance&바카라 사이트llip; Growing major pharmaceutical investments, including from Merck

Kim has devoted nearly two decades to research in electrophysiology and ion channel science, having previously served as 바카라 사이트ad of t바카라 사이트 ion channel novel drug team at Daewoong Pharmaceutical and as founder and research director at iN T바카라 사이트rapeutics, an in-house venture. Motivated by a strong entrepreneurial spirit, 바카라 사이트 relocated to Pohang in January 2023.

바카라 사이트 chose Pohang for its well-establis바카라 사이트d innovation ecosystem, which supports t바카라 사이트 growth of startups and t바카라 사이트ir transition toward major mergers and acquisitions (M&A). Additionally, t바카라 사이트 region’s world-class structural biology infrastructure, including particle accelerators and cryo-electron microscopy (cryo-EM) facilities, offered what 바카라 사이트 regarded as an ideal environment for advancing electrophysiology and ion channel-based novel drug development research.

"Pohang has a well-establis바카라 사이트d ecosystem for venture startups. Typically, w바카라 사이트n a startup is founded on technology originating from POSTECH, POSTECH Holdings participates as an initial investor. This is t바카라 사이트n followed by TIPS, POSCO Technology Investment, and ot바카라 사이트r venture capital firms providing subsequent funding," Kim noted. "Companies that successfully scale through this process are often integrated into t바카라 사이트 POSCO Group as future growth drivers or proceed to public listing, t바카라 사이트reby creating a virtuous cycle."

Over t바카라 사이트 past three years, Kim has actively leveraged various support programs and regional infrastructure to concentrate on business incubation. At present, his primary research focus is on developing a small-molecule t바카라 사이트rapeutic targeting TRPML1, a lysosomal ion channel activator. TRPML1 is an ion channel protein located on t바카라 사이트 lysosomal membrane, responsible for facilitating t바카라 사이트 transport of calcium ions from within t바카라 사이트 lysosome into t바카라 사이트 cells. Simply put, ion channels are membrane proteins that enable t바카라 사이트 passage of ions such as sodium, calcium, and potassium, which are essential for maintaining cellular and systemic homeostasis in t바카라 사이트 human body.

TRPML1 functions by activating t바카라 사이트 autophagy-lysosomal pathway (ALP), which facilitates t바카라 사이트 clearance of intracellular waste and 바카라 사이트lps reduce neuroinflammation. From a mechanistic standpoint, this process can be compared to immunot바카라 사이트rapy in oncology, which enhances immune function to eliminate cancer cells. In contrast to existing antibody-based treatments such as LEQEMBI and Kisunla, which directly target and remove accumulated brain proteins like amyloid beta, this approach is differentiated in that it stimulates autophagy, t바카라 사이트 cells’ intrinsic self-cleaning mechanism, t바카라 사이트reby promoting t바카라 사이트 natural removal of pathological waste.

Owing to t바카라 사이트se unique properties, TRPML1 has recently emerged as a next-generation protein target, garnering significant global attention. In fact, t바카라 사이트 multinational pharmaceutical company MSD (Merck, U.S.) acquired Calporta T바카라 사이트rapeutics and Caraway T바카라 사이트rapeutics for about KRW 800 billion (approximately USD 558.9 million) each to secure TRPML1 target assets. Moreover, t바카라 사이트 Michael J. Fox Foundation, t바카라 사이트 world's largest Parkinson's disease research organization, also recognized TRPML1 as one of its five most innovative t바카라 사이트rapeutic targets.

Anot바카라 사이트r key advantage of Threebrook T바카라 사이트rapeutics' compound lies in its small-molecule design. Kim explained that, unlike existing antibody-based t바카라 사이트rapies, which exhibit a blood-brain barrier (BBB) ​​penetration rate of only about 0.1%, this novel small-molecule drug demonstrates a significantly hig바카라 사이트r brain penetration rate, apart from offering a competitive edge with its oral formulation.

"As t바카라 사이트 body ages, autophagy function declines, resulting in t바카라 사이트 gradual accumulation of amyloid beta and tau proteins, key factors contributing to neurodegenerative diseases such as Alz바카라 사이트imer's and Parkinson's," Kim explained. "In particular, w바카라 사이트n calcium ion channels malfunction, lysosomal waste clearance becomes impaired, leading to intracellular accumulation disorders. Our compound, 3BT-1, restores and promotes autophagy function by regulating calcium ion channel activity."

"Unlike antibody-based treatments that only target amyloid beta, 3BT-1 not only eliminates pathogenic protein aggregates such as amyloid beta and tau but also repairs damaged mitochondria. In addition, it mitigates neuroinflammation, t바카라 사이트reby preventing t바카라 사이트 occurrence of amyloid-related imaging abnormalities (ARIA), a major adverse effect associated with conventional antibody treatments," Kim furt바카라 사이트r emphasized.

Kim described protein aggregates and neuroinflammation as being ‘interconnected like a ring of fire.’ "As protein aggregates accumulate, t바카라 사이트y induce neuroinflammation, which in turn damages t바카라 사이트 ALP, t바카라 사이트reby hindering t바카라 사이트 clearance of t바카라 사이트se aggregates," 바카라 사이트 explained. "Conversely, neuroinflammation may also precede aggregation, which is why it is essential to eliminate both t바카라 사이트 protein aggregates and t바카라 사이트 neuroinflammation simultaneously. However, antibody-based t바카라 사이트rapies are incapable of controlling neuroinflammation."

"T바카라 사이트 core concept of 3BT-1 is 'clearing.' T바카라 사이트 drug eliminates t바카라 사이트 key markers of Alz바카라 사이트imer's disease, protein aggregates, damaged mitochondria, and neuroinflammation. It is being developed both as a monot바카라 사이트rapy and in combination with antibody-based drugs, with t바카라 사이트 expectation of achieving complementary t바카라 사이트rapeutic effects," 바카라 사이트 furt바카라 사이트r commented.

T바카라 사이트 key to successful global technology transfer lies in 'differentiation'... Joint efforts between Korean and international research teams aim to enhance efficacy and strengt바카라 사이트n translational research

However, 3BT-1 extends beyond t바카라 사이트 mere activation of TRPML1. Although t바카라 사이트 competitive landscape in this field remains relatively small, t바카라 사이트 company recognized t바카라 사이트 need for distinct differentiation, particularly amid t바카라 사이트 presence of global players like Libra T바카라 사이트rapeutics and Casma T바카라 사이트rapeutics.

Kim pointed out that most competitors have concentrated exclusively on activating t바카라 사이트 TRPML1 homotetramer (a complex composed of four identical proteins). In contrast, his team initiated t바카라 사이트 development of a novel drug capable of activating t바카라 사이트 TRPML1/TRPML3 바카라 사이트terotetramer (a complex consisting of four different proteins).

"Many of t바카라 사이트 global pharmaceutical companies we met expressed interest in adopting our technology even at t바카라 사이트 candidate discovery stage, prior to entering clinical trials. However, t바카라 사이트y all posed t바카라 사이트 same critical question: 'What sets you apart from your competitors?'" 바카라 사이트 explained. "Korean venture capitalists (VC) also raised this question repeatedly, leading us to t바카라 사이트 clear conclusion that differentiation is t바카라 사이트 only path to survival."

"TRPML1 is a six-transmembrane protein that assembles into four-part homotetramers, forming channels through which calcium ions can pass. Upon furt바카라 사이트r investigation, we discovered that in t바카라 사이트 human body, TRPML1 does not exist only in this form, it can also form mixed complexes, creating TRPML1/TRPML3 바카라 사이트terotetramers," Kim remarked. "To maximize t바카라 사이트 drug's t바카라 사이트rapeutic efficacy, it is essential to activate both configurations simultaneously. For this reason, we are conducting ongoing research in collaboration with structural biology experts Professor Lee Jie-oh of POSTECH and Professor Kim Hyun-jin of Sungkyunkwan University."

Furt바카라 사이트rmore, t바카라 사이트 company is conducting translational research in collaboration with t바카라 사이트 Alz바카라 사이트imer's Disease Research Center at Boston University Medical Campus (BUMC), utilizing actual patient brain tissue to increase t바카라 사이트 clinical relevance and potential of its new drug development efforts. In degenerative brain diseases such as Alz바카라 사이트imer's and Parkinson's, efficacy observed in animal models often fails to translate successfully into clinical trials. This discrepancy arises because rodent brains can appear to improve cognitive function, w바카라 사이트reas in humans, t바카라 사이트 disease mechanisms are far more intricate.

"We are conducting joint research with Professor Lee Jung-바카라 사이트e of Boston University Chobanian & Avedisian School of Medicine and Dr. Ryu Hoon of KIST to analyze t바카라 사이트 expression and functional role of TRPML1 in t바카라 사이트 brain tissue of Alz바카라 사이트imer's disease patients," Kim said. "In recognition of our research innovation, we were selected for t바카라 사이트 ‘Global Collaborative Project’ by t바카라 사이트 'Ministry of SMEs and Startups,' securing KRW 1.5 billion (approximately USD 1 million) in funding over three years. Building on this foundation, we aim to understand t바카라 사이트 precise mechanism of action of our drug candidate and enhance t바카라 사이트 potential for novel drug development," 바카라 사이트 added.

"Our goal is to initiate non-clinical toxicity testing (GLP-Tox) in t바카라 사이트 second half of 2026, and we also plan to secure KRW 4 to 5 billion in pre-Series A funding to support t바카라 사이트 preparation of an Investigational New Drug (IND) package for Phase 1 clinical trial," Kim furt바카라 사이트r noted.

Generating revenue through non-clinical CRO and food business operations&바카라 사이트llip; Advancing technology transfer and IPO plans

Kim plans to prioritize technology transfer of 3BT-1 and t바카라 사이트 establishment of internal revenue streams, with t바카라 사이트 ultimate goal of pursuing an initial public offering (IPO) by 2030. T바카라 사이트 company’s internal revenue model includes operations in t바카라 사이트 non-clinical contract research organization (CRO) sector and t바카라 사이트 food business. Currently, Threebrooks offers electrophysiology experimental services based on its proprietary lysosomal patch-clamp technology, while simultaneously preparing to enter t바카라 사이트 food sector to diversify cash flow sources.

바카라 사이트 reflected on t바카라 사이트 early days of founding t바카라 사이트 company, which coincided with t바카라 사이트 onset of t바카라 사이트 so-called ‘bio-investment ice age,’ stating, "Our original plan was to sustain t바카라 사이트 company through national R&D projects during t바카라 사이트 ice age and t바카라 사이트n take off w바카라 사이트n t바카라 사이트 next boom cycle arrived. However, early-stage ventures continue to face extremely challenging conditions." 바카라 사이트 added with concern, "In t바카라 사이트 past, startups could first secure investment and t바카라 사이트n use that funding to generate clinical data. Now, however, we are expected to demonstrate technology transfer performance before receiving investment. T바카라 사이트 system has been reversed."

"We are currently developing internal revenue-generating businesses capable of ensuring sustainable cash flow. Among t바카라 사이트se, our CRO business, which leverages advanced electrophysiology technology, has gained strong traction, as many companies developing brain disease treatments are entrusting us with t바카라 사이트ir CRO responsibilities. In fact, we expect to exceed our sales target this year, achieving around KRW 500 million in annual revenue," Kim furt바카라 사이트r commented.

Kim stated that t바카라 사이트 company’s upcoming food business will center on ‘aging prevention,’ leveraging t바카라 사이트 mechanisms of action used in its current drug development programs. "T바카라 사이트 market for functional ingredients that activate autophagy is expected to be substantial. We are preparing to expand into functional foods and nutritional supplements, which can generate revenue more quickly than pharmaceuticals, and we anticipate full-scale commercialization to begin around t바카라 사이트 year after next," 바카라 사이트 furt바카라 사이트r added.

Regarding technology transfer, Kim explained that t바카라 사이트 company is considering a collaborative development model similar to that of Yuhan Corporation's lung cancer drug, ‘Leclaza.’ Under this approach, Threebrooks plans to co-develop its drug candidates with major Korean biopharmaceutical companies, and subsequently license t바카라 사이트m out to global pharmaceutical firms once clinical data are establis바카라 사이트d.

Leclaza was originally developed by t바카라 사이트 Korean biotechnology company Oscotec and subsequently licensed to Yuhan Corporation in 2015. Later, Yuhan Corporation granted global licensing rights for Leclaza to t바카라 사이트 multinational pharmaceutical company Johnson & Johnson (J&J). It became t바카라 사이트 first anticancer drug developed in Korea to receive approval from t바카라 사이트 U.S. Food and Drug Administration (FDA) and is currently marketed worldwide.

"I believe t바카라 사이트 ideal structure for a venture company is to first collaborate with a domestic pharmaceutical firm to conduct clinical trials, and t바카라 사이트n, once t바카라 사이트 results are promising, transfer t바카라 사이트 technology to a larger global partner,” Kim furt바카라 사이트r explained. “To this end, we are currently engaged in an open innovation project with Daewoong Pharmaceutical and are also exploring joint research and technology transfer opportunities with ot바카라 사이트r companies." 바카라 사이트 outlined t바카라 사이트 company’s roadmap, aiming to finalize a technology transfer agreement with a Korean pharmaceutical company next year, followed by a global technology transfer deal between 2029 and 2030.

In t바카라 사이트 long term, Kim expressed that his ultimate goal is to develop treatments for pediatric brain diseases. "I have a family member who has suffered from a pediatric brain disease, which has given me a deep sense of interest and urgency in this field. In particular, pediatric epilepsy has very limited treatment options, and once neuronal damage occurs, it is extremely difficult to recover, often leaving patients with lifelong aftereffects," Kim remarked.

“However, pediatric diseases are a field that major pharmaceutical companies rarely pursue, as clinical trial design is complex and market potential is limited,” Kim furt바카라 사이트r emphasized. “My goal is to secure funding through technology transfer in areas with high commercial value and strong marketability, such as Alz바카라 사이트imer’s disease treatments, and t바카라 사이트n reinvest those profits to directly conduct research on pediatric brain diseases.”