ImmunoForge Receives IND Approval for Phase 2 Clinical Trial of 'Pemziviptadil' for DMD Cardiomyopathy Treatment from FDA, and KF1601, a chronic myeloid leukemia treatment, phase 1 clinical trial IND approved by MFDS

SEOUL, South Korea, Jan. 14, 2025 /PRNewswire/ -- ImmunoForge announced that it has received approval for the Phase 2 clinical trial IND for 'Pemziviptadil (development code name PF1804),' a treatment for DMD (Duchenne Muscular Dystrophy) cardiomyopathy, from the U.S. Food and Drug Administration (FDA).

Pemziviptadil is a once-weekly DMD cardiomyopathy drug developed based on ImmunForge's long-acting drug platform, the ELP Platform (Elastin Like Polypeptide Platform). Pemziviptadil is a vasoactive intestinal peptide (VIP) that selectively acts on the vasoactive intestinal peptide receptor 2 (VPAC2) to increase both cardiac contraction and relaxation, thereby improving cardiac function.

Vasoactive intestinal peptide has been difficult to develop as a therapeutic agent because its half-life in the body is less than 1 minute. However, ImmunoForge has developed a first-in-class new drug with an increased half-life of 60 hours by combining it with the ELP platform, a long-acting drug platform, and selectively acting on vasoactive intestinal peptide receptor 2 (VPAC2) while avoiding gastrointestinal side effects such as diarrhea of vasoactive intestinal peptide receptor 1 (VPAC1). The effect of suppressing cardiac dysfunction was confirmed in a non-clinical MDX mouse model conducted in the United States, and following this approval for phase 2 clinical trials, phase 2 clinical trials are scheduled to be conducted in the United States and Korea.

The most common cause of death for DMD (Duchenne Muscular Dystrophy) patients was respiratory failure, but due to improvements in respiratory care and other treatments, cardiomyopathy is emerging as the main cause of death for these patients rather than respiratory failure. Deficiency of cardiac dystrophin protein impairs calcium channel function, which plays an important role in muscle contraction, leading to an increase in intracellular calcium, which in turn increases protein-degrading enzymes and causes protein degradation. This leads to muscle fiber and cell necrosis, and most patients develop cardiomyopathy in their 30s. It is estimated that there are currently 500,000 DMD patients worldwide, and since more than 80% of DMD patients die from this cardiomyopathy, if proper treatments are developed in the future, the DMD cardiomyopathy treatment market is expected to reach tens of trillions. Current treatments, such as beta-blockers and calcium channel blockers, can reduce heart rate and myocardial contractility, but there is no treatment for long-term and fundamental myocardial improvement.

ImmunoForge's co-CEOs Sung-Min Ahn and Kiho Chang said, "Based on this US FDA approval for phase 2 clinical trials, we expect to confirm the therapeutic effect of Pemziviptadil in phase 2 clinical trials and thereby secure an unrivaled position in the DMD cardiomyopathy treatment market, where there is currently no appropriate treatment." He added, "As many overseas companies are currently showing interest in Pemziviptadil, we will actively pursue collaborative models such as joint development and technology transfer."

Pemziviptadil is a new drug that is expected to be effective not only for DMD cardiomyopathy but also for other heart diseases such as heart failure (HF) and pulmonary arterial hypertension (PAH) through nonclinical studies and various phase 1 clinical trials and has received orphan drug designation (ODD) for pulmonary arterial hypertension in addition to DMD cardiomyopathy from the US FDA. Meanwhile, Pemziviptadil was selected for a strategic technology-type international joint technology development project by the Korea Institute for Advancement of Technology in 2023 and is receiving a total of KRW 3 billion in support until 2026.

Around the same time, ImmunoForge also announced that it has received approval for the phase 1 clinical trial IND for 'KF1601 (development code name)', a treatment for Chronic Myelogenous Leukemia (CML), from the Korea Ministry of Food and Drug Safety.

KF1601 is a novel synthetic oral TKI targeted therapy that inhibits BCR::ABL1. It effectively inhibits the T315I mutation during the nonclinical development process and exhibits excellent safety and tolerability. It is expected to be a treatment that can be used more universally than existing CML treatments, and it is expected to contribute to managing chronic myeloid leukemia as a chronic disease that can overcome resistance by replacing existing treatments.

Chronic myeloid leukemia is a blood cancer that is caused by the excessive proliferation of myeloid cells and inhibition of apoptosis of BCR::ABL1 tyrosine kinase, which is expressed as a result of the Philadelphia chromosome (Philadelphia chromosome (Ph) abnormality caused by the fusion between the Abelson (Abl) tyrosine kinase gene on chromosome 9 and the Breakpoint Cluster Region (Bcr) gene on chromosome 22, a chimeric oncogene. CML accounts for approximately 30% of the total adult leukemia incidence, and it presents differently depending on age, gender, and region, and is reported to occur in approximately 0.4 to 1.75 cases per 100,000 people per year. Especially, ImmunoForge's KF1601 has the effect of simultaneously inhibiting FLT3 in the Blast Phase patient group, and this effect was announced at the Molecular Cancer Society last year, so it is expected to be a differentiating point for KF1601 in CML drugs.

ImmunoForge's co-CEOs Sung-min Ahn and Kiho Chang said, "Based on this Phase 1 IND approval, we plan to confirm the safety of KF1601 in the Phase 1 clinical trial and develop it targeting the global market including the US and Europe in addition to Korea." They added, "Based on the development results, we plan to participate in academic conferences and global partnering events and actively pursue technology transfers with global pharmaceutical companies." ImmunoForge is currently in discussions with several domestic and foreign companies.

Meanwhile, ImmunoForge is currently in the process of Series C funding and plans to apply for a technology assessment by the end of this year and pursue a KOSDAQ listing next year.