[Edaily Reporter Minji Son ] CHONGKUNDANG(185750)is embarking on the development of a new drug for rare eye diseases by acquiring “KIO-301,” a new drug candidate for retinal diseases from U.S.-based Kiora Pharmaceuticals. The agreement goes beyond simply securing domestic sales rights; Jongkundang will take the lead in all aspects of the project, including clinical development, regulatory approval, production, reimbursement, and commercialization. This move is seen as an effort to expand the company’s scope from existing biosimilars for retinal diseases to innovative new drugs.

According to the pharmaceutical and biotech industry on the 1st, CHONGKUNDANG recently signed an exclusive licensing agreement with U.S.-based Kiora Pharmaceuticals, a company specializing in ophthalmic diseases, for KIO-301’s ophthalmic indications in South Korea. CHONGKUNDANG will be responsible for domestic development, regulatory approval, and commercialization.

Under the agreement, CHONGKUNDANG will pay Kiora Pharmaceuticals an upfront payment of $1 million. It will also pay milestone payments upon achieving future development, approval, and sales targets. Additionally, the company may be required to pay double-digit royalties once net sales are generated in South Korea.

KIO-301 is a small-molecule drug candidate currently being developed with retinitis pigmentosa—a rare hereditary retinal disease—as its primary indication. Retinitis pigmentosa is a condition in which photoreceptors, such as light-sensing rod and cone cells, degenerate, leading to narrowed visual fields and vision loss. CHONGKUNDANG estimates there are more than 10,000 patients with this condition in South Korea.

Reasons for Preemptive Acquisition Before Phase 2 Results

: However, KIO-301 is an early-stage candidate that has completed Phase 1 clinical trials and is currently undergoing Phase 2 trials. Its efficacy has not yet been confirmed in a controlled trial, and Phase 2 results have not been released. Nevertheless, CHONGKUNDANG decided to acquire the drug early, noting the significant unmet medical need, its mechanism of action—which is not limited to specific genetic mutations—and the potential for indication expansion.

KIO-301 is not a treatment that regenerates degenerated photoreceptors or corrects the causative gene. It employs a “molecular light switch” mechanism that enters retinal ganglion cells—which remain even after photoreceptors have been lost—and endows them with the ability to respond to light. It is designed so that when exposed to light, its molecular structure changes, regulating the ion channel activity of retinal ganglion cells and enabling visual signals to be transmitted to the brain.

Another key feature is that it does not depend on specific causative genes. Since retinitis pigmentosa is caused by various genetic mutations, the number of patients currently eligible for gene therapy is limited. KIO-301 aims to treat a broader patient population by utilizing the remaining retinal ganglion cells, regardless of the causative mutation.

Under the terms of the agreement, CHONGKUNDANG’s domestic development rights are not limited to retinitis pigmentosa. The two companies have defined the scope of application as all ophthalmic diseases, including retinitis pigmentosa. Kiora has suggested the possibility of expanding the indications to other hereditary retinal diseases, such as choroidal atrophy and Stargardt disease, based on the common pathological mechanism of photoreceptor degeneration.

If the development for retinitis pigmentosa is successful, this means CHONGKUNDANG has secured the legal basis to pursue the development of subsequent indications in Korea without the need for a separate licensing agreement.

CHONGKUNDANG previously laid the foundation for its ophthalmology business by developing “Lucenvis,” a ranibizumab biosimilar. Lucenvis is an intravitreal injection used to treat conditions such as wet age-related macular degeneration and diabetic macular edema. The company is expected to leverage the clinical and regulatory experience in retinal diseases, as well as its ophthalmology sales network—gained through existing products—for the development and commercialization of KIO-301.

CHONGKUNDANG official stated, “To secure a treatment for a condition with high unmet medical needs at an early stage, we independently verified the clinical data from ongoing trials and other relevant information before signing the contract.”

Taking the

“Steering Wheel”

for

Domestic Development… Alternative CMO Also Possible

According to the contract filed with the U.S. Securities and Exchange Commission (SEC), the rights CHONGKUNDANG has secured for “KIO-301” extend beyond typical domestic sales rights. First, CHONGKUNDANG will conduct domestic clinical development and submit a marketing authorization application to the Ministry of Food and Drug Safety at its own expense. Furthermore, data generated through development activities conducted domestically will be exclusively owned by CHONGKUNDANG and classified as the company’s new intellectual property. Conversely, Kiora Pharmaceuticals will provide CHONGKUNDANG with data from overseas Phase 2 and 3 clinical trials, as well as non-clinical and manufacturing and quality control (CMC) data, without separate royalties, and will support the use of such data in the domestic marketing authorization application.

Autonomy has also been secured regarding production. While CHONGKUNDANG may source KIO-301 from a contract manufacturing organization (CMO) designated by Kiora Pharmaceuticals, this is not mandatory. This means CHONGKUNDANG is not restricted to using Kiora Pharmaceuticals’ supply chain and may select a separate CMO. If CHONGKUNDANG wishes to source the product through a CMO of its own choosing, Kiora Pharmaceuticals must assist with the evaluation and approval of that CMO’s eligibility.

Furthermore, matters not agreed upon by the Joint Steering Committee (JSC) formed by the two companies will be subject to consultation among senior management; however, if consensus cannot be reached within a certain period, CHONGKUNDANG, in principle, holds the final decision-making authority. This is a relatively favorable structure that allows CHONGKUNDANG to take the lead in domestic development.

However, CHONGKUNDANG maintains that specific domestic clinical trial schedules, marketing authorization, and production and commercialization plans are still under review.

Global Phase 3 in the Planning Stage… Success of Phase 2 Is Key

Kiora Pharmaceuticals expects to proceed with the global Phase 3 clinical trial for KIO-301 in collaboration with Théa, a French ophthalmology-focused pharmaceutical company that holds rights outside Asia; Senju Pharmaceutical, its partner in certain Asian regions including Japan and China; and CHONGKUNDANG, Korea’s pharmaceutical company. The agreement also stipulates that, if requested by CHONGKUNDANG, Kiora Pharmaceuticals will make commercially reasonable efforts to include a South Korean trial site in the Phase 3 clinical trial after consulting with other global partners.

However, the launch of the global Phase 3 trial and South Korea’s participation have not yet been finalized. Japan’s Senju Pharmaceutical also holds an option—not formal development or commercialization rights—to enter into an exclusive license agreement for a certain period following the announcement of the results from the ongoing Phase 2 clinical trial.

The clinical evidence is also in its early stages. The Phase 1 clinical trial, published this year in the international journal *Nature Medicine*, was conducted on 12 eyes from 6 patients with end-stage retinitis pigmentosa. No serious adverse events or dose-limiting toxicities were observed, and changes in light perception and vision-related functions were observed in some patients.

However, these signs showed significant inter-patient variability and tended to be relatively pronounced immediately after administration but then weakened over time. There are limitations to this study, as it was an open-label, single-arm trial without a control group, and with only six patients, it is not possible to assess the treatment’s efficacy or the duration of its effects.

Kiora Pharmaceuticals is currently conducting a randomized, double-blind, controlled Phase 2 clinical trial in Australia involving 36 patients with end-stage retinitis pigmentosa. The company plans to complete enrollment of the final target number of patients by the first quarter of next year and announce topline results in the third quarter of the same year.

An industry official stated, “Since the Phase 2 clinical trial is the first to comprehensively evaluate the potential efficacy against a control group, it will serve as a turning point in determining whether to proceed with domestic development and a global Phase 3 trial.”