HLB Group’s drug development pipeline is poised for a series of milestones in the latter half of this year, with key clinical and regulatory events on the horizon. The company anticipates several critical developments, including a U.S. Food and Drug Administration (FDA) decision on a bile duct cancer treatment in September, a resubmission for liver cancer drug approval, results from Phase 3 trials for an ophthalmic treatment, and the release of clinical data for a next-generation CAR-T therapy by year-end.
Industry sources reported on Thursday that HLB’s FGFR2-targeted cancer drug, Lirapegrafinib, is set to be the first in the HLB Group’s pipeline to yield results this fall. Developed by U.S. subsidiary Elevar Therapeutics, lirafugratinib is under FDA review for the treatment of bile duct cancer with FGFR2 gene fusion or rearrangement. The FDA’s decision is expected by September 25, the Prescription Drug User Fee Act (PDUFA) target date.
Lirafugratinib has garnered significant attention, receiving FDA orphan drug designation in 2022 and breakthrough therapy status in 2023. Its New Drug Application (NDA) has been granted priority review. Elevate recently conducted a late-cycle meeting with the FDA to discuss post-marketing requirements, reporting no new issues that could impact the approval process.
Liver Cancer Combination Therapy Gears Up for FDA Resubmission
Another key development slated for the latter half of the year is HLB’s resubmission of its liver cancer combination therapy, Rivoceranib plus Camrelizumab, to the FDA. Following a Complete Response Letter (CRL) from the FDA in July, Elevate is working with partners to prepare for resubmission.
The company clarified that the CRL primarily addressed Good Manufacturing Practices (GMP) issues at the drug substance manufacturing facility, rather than concerns about clinical efficacy or safety. With the FDA inspection of the facility concluding with a Voluntary Action Indicated (VAI) status, the company aims to resolve these uncertainties and secure final approval upon resubmission.
Ophthalmic Treatment Awaits Global Phase 3 Results
November will see HLB Therapeutics unveil global Phase 3 results for RGN-259, its treatment for neurotrophic keratitis (NK). The company’s U.S. subsidiary, Regentry, has completed final patient visits for the SEER-2 trial, conducted across 50 medical centers in the U.S. and Europe. Data compilation and statistical analysis are currently underway.
RGN-259, an eye drop solution containing the naturally occurring protein Thymosin Beta4, is slated for topline result announcement in November. Positive efficacy and safety outcomes could significantly boost the treatment’s potential for technology transfer and commercialization.
Verismo Set to Unveil Blood Cancer CAR-T Therapy Data
By year-end, HLB Innovation expects to release clinical data for SynKIR-310, a blood cancer CAR-T therapy developed by its U.S. subsidiary, Verismo Therapeutics. Verismo is conducting a Phase 1 trial for SynKIR-310, which targets CD19, and plans to present interim results at an upcoming global conference.
SynKIR-310 employs a novel KIR-CAR platform, distinguishing it from existing CAR-T therapies. This innovative approach leverages the KIR receptors of natural killer (NK) cells and the DAP12 signaling system to enhance T cell anticancer activity and persistence.
An HLB Group spokesperson emphasized the significance of the coming months, stating that the latter half of this year marks a crucial period for validating the ongoing drug development efforts. It anticipates that the progress across the pipeline – from cancer drug approvals and resubmissions to Phase 3 results in ophthalmology and CAR-T clinical data – will substantially bolster the group’s standing in the competitive landscape of drug development.