[Edaily Reporter KIM SAE-MI ] The major events in the biotech industry in July can be summarized as the decision on whether to grant marketing approval for HLB INC.(028300) ’s new liver cancer drug, Riboceranib, and the release of top-line results from the Phase 3 clinical trial of Kolon TissueGene, Inc.(950160)’s cell-gene therapy for osteoarthritis, TG-C.

The Day of “Riboceranib’s” Destiny… What Are the Chances of FDA Approval Amid the CMC Inspection Gap?

The biggest focus of the biotech industry in July is whether HLB PHARMACEUTICAL’s riboceranib and Hengrui Pharmaceuticals’ immune checkpoint inhibitor camrelizumab—in a combination therapy for first-line treatment of liver cancer—will be able to clear the U.S. Food and Drug Administration (FDA) hurdle.

HLB INC. is awaiting a decision on July 23 regarding the approval of its new liver cancer drug, Riboceranib, in combination with Kamrelizumab. Riboceranib is a targeted anticancer drug developed by HLB INC.’s U.S. subsidiary, Eleva Therapeutics, and the company is seeking approval for its use in combination with Kamrelizumab, produced by China’s Hengrui Pharmaceuticals. This combination therapy has previously received two Complete Response Letters (CRLs) from the FDA.

The biggest point of contention had been when the manufacturing and quality control (CMC) inspection of Hengrui Pharmaceuticals would take place, but the possibility of a document review in lieu of an on-site inspection has recently emerged. With the approval of Riboceranib just over a month away, investor anxiety was mounting as the CMC inspection of Hengrui Pharmaceuticals had not yet been conducted. Against this backdrop, HLB INC. suddenly drew the market’s attention on the 16th by mentioning the possibility of a document review in lieu of an on-site inspection.

HLB INC. officials stated, “The U.S. Food and Drug Administration (FDA) operates a system called RLI (Reports in Lieu of Inspection), under which it reviews documents and data submitted by the manufacturer to substitute for an on-site inspection,” adding, “The fact that there have been numerous recent cases of this is a positive development.”

The biotech industry, however, views the likelihood of the FDA applying RLI to this case as low. An FDA regulatory expert noted, “Considering that the FDA has repeatedly rejected applications due to issues with the production facilities of China’s Hengrui Pharmaceuticals, it seems unlikely that they would now replace an on-site inspection with a document submission.”

HLB INC.’s approval timeline for Riboceranib is also linked to the schedule for HLB Group’s ( HLB PHARMACEUTICAL(047920)) rights offering. Previously, on May 13, HLB PHARMACEUTICAL resolved to conduct a 120 billion won rights offering, followed by a public offering of unsubscribed shares. However, when the Financial Supervisory Service requested the submission of an amended filing on the 27th of the same month, the effectiveness of the securities registration statement was suspended, delaying the capital increase schedule by about one month. Since the approval of Riboceranib is a key factor that could directly impact investment sentiment across the entire HLB INC., the outcome of the approval process is expected to determine the subscription sentiment and ultimate success of HLB PHARMACEUTICAL’s capital increase.

Kolon TissueGene, Inc. to Release ‘TG-C’ Top-Line Results… Will It Wash Away the Nightmare of ‘Inbosa’s Approval Revocation’

? A series of events related to stem cell therapies are scheduled for July in the biotech industry. Among these, Kolon TissueGene, Inc.’s bid for redemption is drawing particular attention from the biotech sector. The top-line data from Kolon TissueGene, Inc.’s global Phase 3 clinical trial for TG-C (formerly Invossa), a cell- and gene-based therapy for osteoarthritis, set to be released in July, is also a matter of intense interest in the biotech industry. As expectations for the success of the TG-C Phase 3 trial grow, Kolon TissueGene, Inc.’s stock price had surged 126.9% over the past year as of the 19th.

TG-C is regarded as a symbol of KOLON CORPORATION’s biotech business. Although it received marketing approval from the Ministry of Food and Drug Safety in 2017 as the world’s first gene therapy for osteoarthritis, it suffered the setback of having its domestic marketing approval revoked in 2019. Although the U.S. Phase 3 clinical trial was subsequently suspended, the tide turned when the trial resumed in April 2021.

#KOLON CORPORATION has devoted more than 20 years to the development of TG-C. From 2021 through this year, the group has provided Kolon TissueGene, Inc. with 256 billion won in funding alone through third-party private placements over the past five years. In addition, some observers note that the group’s commitment to the project was reaffirmed when Lee Kyu-ho, Vice Chairman of KOLON CORPORATION and the eldest son of Honorary Chairman Lee Woong-yeol, joined the board of directors of Kolon TissueGene, Inc. in late March.

The primary endpoints that will determine the success or failure of the TG-C global Phase 3 clinical trial are the change in the WOMAC Pain Score and the change in the Visual Analog Scale (VAS) score. Its potential as a disease-modifying osteoarthritis drug (DMOAD) can be gauged by changes in the medial joint space width (mJSW) of the knee and whether total knee arthroplasty (TKA) is delayed. However, DMOAD-related indicators are not decisive factors in determining regulatory approval.

KANGSTEM BIOTECH CO., LTD.(217730)Topline results from the Phase 2a clinical trial of Oscar, a treatment for knee osteoarthritis, are also scheduled to be announced in late July, providing an opportunity to gauge the development progress of domestic osteoarthritis treatments. Oscar is a new drug candidate for knee osteoarthritis that combines allogeneic stem cell components with a cartilage acellular matrix (CAM), and is being developed with the goal of becoming a disease-modifying osteoarthritis drug (DMOAD).

Previously, on the 10th, KANGSTEM BIOTECH CO., LTD. held an investor briefing in Yeouido, Seoul, where it expressed confidence that it would demonstrate efficacy through the Phase 2a topline data. Given that most domestic mesenchymal stem cell (MSC)-based new drugs have failed to prove efficacy, all eyes are on whether Oscar can turn the tide.

S.Biomedics Co., Ltd.(304360)The company plans to present detailed topline data from the 24-month follow-up of the Phase 1/2a clinical trial for TED-A9, a new drug candidate for Parkinson’s disease, at the International Society for Stem Cell Research (ISSCR) conference to be held in Montreal, Canada, from July 8 to 11.

These 24-month data are significant as they provide key evidence to confirm whether the improvement in motor function following cell transplantation is sustained over the long term and whether safety issues do not accumulate. Based on these data, S.Biomedics Co., Ltd. plans to submit an IND application for a Phase 3 clinical trial to the FDA this year.

'RYBREVANT SC' Assigned U.S. J-Code… Curocell Inc. to Reapply for Reimbursement Review In the U.S., the J-code

will be applied to RYBREVANT FASPRO

,

the subcutaneous (SC) formulation of RYBREVANT

,

starting July 1. RYBREVANT FASPRO is used in combination with Yuhan(000100)’s Lazertinib (U.S. name: Razcluz) as a first-line treatment for EGFR-mutated non-small cell lung cancer. As it becomes easier for U.S. hospitals and clinics to bill for RYBREVANT SC, barriers to prescribing the combination therapy are expected to decrease. Consequently, Yuhan Corporation’s royalty revenue is also expected to increase.

Curocell Inc.(372320)Curocell Inc.’s chimeric antigen receptor (CAR-T) therapy, Limkato, is scheduled for a re-review by the Health Insurance Review and Assessment Service’s Drug Reimbursement Evaluation Committee on July 8. If it passes the re-review, a decision on national health insurance coverage is expected within the third quarter, and the drug is projected to become available for prescription following the announcement of the drug price in October. Curocell Inc. plans to propose a lower price than that of Kymriah, the existing CAR-T therapy.