Jeil Pharma Surge on Orphan Drug Designation[K-Bio Pulse]

[Seok Ji-heon, Edaily Reporter] On March 18, Jeil Pharmaceutical and its subsidiary, Onconic Therapeutics, both hit the upper price limit after announcing that their investigational drug was designated as an orphan drug by the U.S. Food and Drug Administration (FDA). Novmeta Pharma, a biotech company, surged nearly 10% following news that it had recruited a key executive to finalize a major deal. Kolon TissueGene also jumped more than 14% on expectations surrounding FDA approval for its osteoarthritis gene therapy, TG-C.

◇‘Another Recognition of Technological Innovation’

According to KG Zeroin’s MP Doctor (formerly MarketPoint), Jeil Pharmaceutical closed at KRW 13,520, up KRW 3,120 (30%) from the previous trading day. Its subsidiary, Onconic Therapeutics, ended at KRW 18,040, up KRW 4,160 (29.97%). The holding company, Jeil Pharma Holdings, also climbed 14.6% to KRW 8,400.

Jeil Pharmaceutical stock trend (Source: KG Zeroin MP Doctor)

Onconic Therapeutics received an orphan drug designation (ODD) from the FDA for its next-generation synthetic lethal dual-target oncology candidate, “Nesuparib”, aimed at treating gastric and gastroesophageal junction cancer.

Nesuparib was previously granted orphan drug designation in 2021 for pancreatic cancer by both the FDA and South Korea’s Ministry of Food and Drug Safety (MFDS). This latest designation further underscores Onconic’s technological innovation and the potential of its cancer therapies.

Nesuparib inhibits two proteins, PARP (Poly ADP-ribose polymerase) and Tankyrase, both crucial for cancer cell survival, making it a next-generation targeted anticancer agent.

With the FDA’s orphan drug designation acceptance rate standing at just 17.6%, industry insiders see Nesuparib’s designation as significant both in terms of technical credibility and market potential. The high final approval rate for FDA-designated orphan drugs further supports expectations for Nesuparib’s commercialization. Notably, nearly 49% of new drugs approved by the FDA in 2022 had received orphan drug designation.

A Jeil Pharmaceutical representative commented on the stock’s rise: ‘If Nesuparib’s market value is fully recognized, it will drive corporate growth. By securing revenue through proprietary drugs and continuing new drug research, we anticipate a corporate value re-evaluation.’

Onconic is currently conducting a Phase 1b/2 clinical trial for pancreatic cancer and a Phase 2 investigator-initiated trial for endometrial cancer in combination with Keytruda. The latest orphan drug designation suggests that Onconic may soon enter clinical trials for gastric cancer as well. The company is scheduled to present its Nesuparib research findings at the American Association for Cancer Research (AACR) 2025 in April.

◇Boost to License-out and Investment Efforts

Novmeta Pharma’s stock surged 8.81% to close at KRW 5,560 on March 18. Given the 15% daily price limit in the KONEX market, this represents a significant gain. The rally was driven by a PharmEdaily pay-to-read article titled “PTC CEO Joins Novmeta Pharma with LOC in Hand for a $2 Trillion Blockbuster”, which was published on March 17 and made free on March 18 at 9:56 a.m.

stock trend (Source: KG Zeroin MP Doctor)

According to the report, Novmeta Pharma has brought in a key figure to complete a major deal. At its March 24 shareholder meeting, the company will vote on appointing Stuart Peltz, former CEO of PTC Therapeutics, as an internal director. Peltz was a founding member of PTC, which went public on NASDAQ in 2013, and served as CEO until 2023. During his tenure, he secured investment from BlackRock and helped PTC grow into a global biotech firm with 1,700 employees.

One of his most notable achievements was the commercialization of Evrysdi (risdiplam), an oral treatment for spinal muscular atrophy (SMA), which was licensed out to Roche. Peltz played a pivotal role in its development, licensing, and commercialization. Evrysdi received FDA approval in 2020 and has since become a blockbuster drug, generating CHF 1.6 billion (approximately KRW 2.6 trillion) in revenue in 2023.

Peltz will oversee Novmeta Pharma’s global business operations. Joining him is Kylie O’Keefe, PTC’s former Chief Commercial Officer (CCO), who led global commercialization strategies for six rare neurological and metabolic disease products across 50+ countries. At Novmeta Pharma, she will serve as Chief Operating Officer (COO).

The two executives reportedly approached Novmeta Pharma on their own, recognizing the potential of its key pipeline. Their involvement helped the company secure a Letter of Cooperation (LOC) from a leading Canadian institution specializing in hereditary muscle disorders.

Novmeta Pharma’s Vice President Heon-jong Lee stated, ‘With these globally recognized experts on board, confidence in our technology has grown significantly. Their expertise will be crucial in advancing and commercializing novel muscle disorder treatments.’

The company aims to focus on license-out deals and attracting investment, with plans to reattempt a KOSDAQ listing in the second half of the year.

◇Pioneering the First Osteoarthritis Disease-Modifying Drug

Kolon TissueGene jumped 14.46% to KRW 50,660 on growing anticipation for FDA approval of its osteoarthritis gene therapy, TG-C.

The stock has been on an upward trajectory, rising approximately 134% since the beginning of the year. Kolon TissueGene completed large-scale Phase 3 dosing for TG-C in 2024 and plans to file a Biologics License Application (BLA) with the FDA by 2026 following patient follow-ups.

Kolon TissueGene stock trend (Source: KG Zeroin MP Doctor)

TG-C is a cell-based gene therapy that provides up to two years of pain relief and improved joint function with a single injection. The company highlighted its superior efficacy, with a 86% response rate in Phase 2 trials, outperforming existing treatments.

Kolon TissueGene aims to obtain disease-modifying osteoarthritis drug (DMOAD) status from the FDA upon approval. Currently, no osteoarthritis drug has received DMOAD designation.

In a corporate report, Korea Investment & Securities analyst Hae-joo Wi stated: “If TG-C is approved as a disease-modifying osteoarthritis drug, sales could reach $7-8.2 billion, with an estimated operating profit of KRW 5.5 trillion.”

Commenting on the stock’s rise, a Kolon TissueGene spokesperson said: ‘Following our acquittal last November, market trust in the company has been restored. Recent U.S. media briefings have likely fueled optimism regarding TG-C’s FDA approval prospects.’

TG-C was originally launched in South Korea in 2017 but had its approval revoked in 2019 due to incorrect documentation on cell origins. The company has been involved in ongoing legal proceedings, though a 2023 criminal trial ruled in its favor.