Chronic Graft-Versus-Host-Disease (cGVHD), a common and often fatal complication following allogeneic Hematopoietic Stem Cell Transplantation (HSCT), is a multi-organ disorder in which transplanted immune cells (graft) attack the healthy cells (host), leading to inflammation and fibrosis in multiple organs. AbbVie and Johnson and Johnson’s Imbruvica, greenlighted by the FDA in 2017, is the first FDA-approved therapy for the treatment of cGVHD.
The Company we are profiling today is also developing a drug for cGVHD – Kadmon Holdings Inc. (KDMN), and the drug candidate in focus is KD025.
KD025 is an investigational oral drug that works by blocking the protein Rho-associated coiled-coil containing protein kinase 2 (ROCK-2), thereby stopping immune cells from attacking healthy cells.
KD025 is under a pivotal phase II trial in chronic graft-versus-host disease, dubbed ROCKstar. As per the interim outcomes of the study, KD025 has been well-tolerated and has already demonstrated high response rates in patients with severe and complex cGVHD after a median of five months of follow-up.
The Company held its pre-New Drug Application meeting with the FDA last month and anticipates receiving the final minutes from the pre-NDA meeting in the next few days. The outcome of the meeting is an important catalyst, which can have a significant impact on the Company’s stock price.
The topline results from the primary analysis of the ROCKstar trial are expected in the second quarter of 2020.
KD025 is also being tested in Systemic Sclerosis and a phase II trial in this indication is underway. Systemic Sclerosis, also known as scleroderma, is a chronic immune disorder characterized by fibrosis of the skin and internal organs. An estimated 75,000 to 100,000 people in the United States are affected by Systemic Sclerosis.
Next up in the pipeline is KD033, an immuno-oncology compound, which is expected to enter into clinical testing this quarter.
The Company is also continuing ongoing Investigational New Drug Application (IND)-enabling activities of KD045, its next-generation ROCK inhibitor for the treatment of fibrotic diseases.
Kadmon ended the year 2019 with cash and cash equivalents of $139.6 million.
KDMN has traded in a range of $1.63 to $5.50 in the last 1 year. The stock closed Thursday’s (Apr.9, 2020) trading at $4.32, up 9.64%.
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