HLB has secured its first drug approved by the U.S. Food and Drug Administration after 19 years of drug development efforts. The approval of Lytgobi (ingredient: lirafugratinib), a treatment for bile duct cancer, follows years of global clinical trial and regulatory experience accumulated since the company secured global rights to rivoceranib in 2007.

Lytgobi, developed by HLB’s U.S. subsidiary Elevar Therapeutics, is an oral targeted cancer therapy for patients with bile duct cancer harboring FGFR2 fusions or rearrangements. In global Phase 1 and 2 trials, the drug recorded an objective response rate (ORR) of 45.7% and a median duration of response (mDoR) of 11.8 months.

The FDA approval is significant in that HLB has converted its accumulated global drug development experience into an actual regulatory approval. In particular, U.S. clinical trials and regulatory processes are continuing for follow-up pipeline programs, drawing attention to what results HLB’s drug development strategy may produce following the Lytgobi approval.

First Drug Development Milestone in 19 Years, Expansion Beyond Cancer Drugs

According to industry sources Sept. 26, HLB’s drug development efforts began in 2007, when LSKB, the predecessor to Elevar, secured global rights outside China to rivoceranib from Advenchen Research.

HLB, which started with a single drug candidate at the time, gained drug development experience by directly pursuing global clinical trials for rivoceranib. It later expanded its development strategy beyond rivoceranib monotherapy by combining the drug with camrelizumab, an immunotherapy developed by China’s Jiangsu Hengrui Pharmaceuticals.

The combination of rivoceranib and camrelizumab was developed as a first-line treatment for liver cancer. By combining anticancer drugs with different mechanisms of action to enhance treatment effects, the company expanded its development approach beyond a single-candidate model.

HLB later expanded its drug development portfolio into precision medicine targeting FGFR2 genetic alterations by acquiring lirafugratinib. Beyond bile duct cancer, the company is also conducting tumor-agnostic trials targeting various solid tumors with FGFR2 fusions or rearrangements.

Its drug development approaches have also broadened. HLB Therapeutics (115450) is conducting a global Phase 3 trial of RGN-259, a recombinant protein-based treatment for ophthalmic diseases. HLB Innovation (024850)’s U.S. subsidiary Verismo Therapeutics is developing treatments for blood cancers and solid tumors based on its next-generation CAR-T platform, KIR-CAR.

As the group expands from small-molecule targeted cancer drugs into protein therapeutics and cell therapies, the scope of its drug development pipeline is also growing.

Next Catalysts: Liver Cancer and Corneal Disease, With Multiple Clinical Results Ahead

Among the follow-up pipeline programs, the combination of rivoceranib and camrelizumab is drawing the most attention. HLB is preparing to resubmit the treatment for first-line liver cancer to the FDA.

The resubmission process is expected to move forward once the final outcome of the cGMP inspection of the manufacturing facility for the finished rivoceranib product is issued as VAI, or voluntary action indicated.

If both Lytgobi and the liver cancer treatment enter the U.S. market, they could potentially be linked during commercialization. Both drugs target cancers in the hepatobiliary and pancreatic area, allowing them to potentially share networks of medical institutions and physicians. The U.S. commercial organization and experience established during the launch of Lytgobi could also potentially be used for follow-up products.

HLB Therapeutics’ RGN-259 is also approaching a clinical data announcement. RGN-259, being developed as a treatment for neurotrophic keratitis (NK), has completed final visits for all patients in its global Phase 3 trial, and data analysis is currently underway. The company plans to announce topline results in November.

Clinical progress is also continuing in the CAR-T field. Interim results from a Phase 1 trial of Verismo Therapeutics’ solid tumor CAR-T therapy SynKIR-110 were presented during a plenary session at the American Association for Cancer Research (AACR) annual meeting. Tumor reductions and partial responses were observed in some patients.

SynKIR-310, a CAR-T therapy for blood cancers, is also scheduled to have interim Phase 1 results presented at a global medical conference in the second half of this year.

The FDA approval of Lytgobi marks the first regulatory approval in HLB’s 19-year drug development effort. Further progress in the resubmission of its liver cancer treatment, Phase 3 results for RGN-259 and CAR-T clinical trials will provide key indicators of HLB’s drug development capabilities.

HLB has secured its first drug approved by the U.S. Food and Drug Administration after 19 …

After 19-Year Push, HLB Wins First FDA Drug Approval With More Cancer Programs Ahead