- Kadmon Holdings Inc. said Tuesday it can now move ahead with a pivotal Phase 2 study of its ROCK2 inhibitor KD025, following receipt of finalized guidance from the Food and Drug Administration based on a March 2018 Type C meeting.
- In the planned open-label study, patients with chronic graft-versus-host disease (GVHD) who have had at least two different systemic therapies previously will receive either 200 mg of KD025 twice or four times daily. Kadmon's study will assess the drug's effect on overall response rate.
- KD025 carries an Orphan Drug Designation and is also in mid-stage testing for idiopathic pulmonary fibrosis and moderate-to-severe psoriasis.
Kadmon has been struggling with debt and gone through a major restructure in the last 18 months — making positive clinical progress all the more important to demonstrate.
The company, which is headed by the brother of the controversial ex-biotech CEO Sam Waksal, said the planned mid-stage study in GVHD would support potential registration if all goes according to plan.
Based on guidance from the FDA, Kadmon will design the study such that either of the two dosing regimens evaluated could support registration. Kadmon can enroll patients who have either failed on Imbruvica (ibrutinib) or on steroids and immunomodulating agents as well.
The trial design "bodes well for accelerated timeline to KD025 approval in cGVHD," wrote Jefferies equity analyst Biren Amin in an investor note. Amin also cites the study's similarity to the Imbruvica Phase 2 trial, which secured the drug's approval as second-line therapy in steroid-refractory chronic graft-versus-host patients.
"We are pleased with the FDA's guidance, which provides us with a clear regulatory path forward to support a submission for potential approval," Kadmon CEO Harlan Waksal said in a statement.
In a preliminary analysis of a Phase 2 trial of KD025 in steroid-dependent or steroid-refractory cGVHD and active disease, 12 of 17 patients taking 200 mg once daily responded to treatment.
Chronic graft-versus-host disease commonly occurs following hematopoietic stem cell transplantation, and leads to inflammation and fibrosis that can impact almost any organ in the body. It can affect between 30% and 70% of transplanted patients and is sometimes fatal.