FDA Gives Go-Ahead for ROCK2 Inhibitor Study

On Tuesday, Kadmon Holdings Inc stated that the US Food and Drug Administration (FDA) granted a receipt for finalized guidance on a March 2018 Type C meeting for the company’s progression with the pivotal Phase 2 study of its ROCK2 inhibitor KD025.

The drug intends to treat patients with chronic graft-versus-host disease (GVHD), a disease where donor bone marrow or stem cells attack the recipient.

GVHD can affect between 30% and 70% of transplanted patients, and it can be fatal. KD025 (SLx-2119) is an oral small molecule inhibitor.² The open-label study plans to include patients with chronic GVHD who have had a minimum of 2 previous, different systematic therapies. Participants will be administered either 200 mg of KD025 twice or four times daily. The study will analyze the drug’s effect on the overall response rate.

Kadmon CEO, Harlan Waksal, stated his excitement over the drug’s progression. “We are pleased with the FDA's guidance, which provides us with a clear regulatory path forward to support a submission for potential approval.”¹

Based on the guidance from the FDA, the will be composed so that either of the 2 dosing regiments evaluated can support registration. Eligible participants can either have failed on Imbruvica (ibrutinib) or be on steroids and immunomodulating agents additionally.

The study will include 3 three experimental study arms: the KD025 200 mg QD arm will be administered 2 100 mg capsules (200mg) of KD025 once daily; the KD025 200 mg BID arm will be administered 2 100 mg capsules (200 mg) of KD025 twice daily; and the KD025 400 mg QD arm will be administered 4 100 mg capuseles (400 mg) of KD025 once daily.³

Jefferies equity analyst, Biren Amin, positively commented on the study’s design. “[The trial design] bodes well for accelerated timeline to KD025 approval in cGVHD.” Amin also cited the study’s similarity to the Imbruvica Phase 2 trial, which secured the drug's approval as a second-line therapy in steroid-refractory chronic GVHD patients.

According to a preliminary analysis of the 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.

KD025 also currently carries an Orphan Drug Designation aside from being in mid-stage testing for idiopathic pulmonary fibrosis and moderate-to-severe psoriasis.

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References:

1. Kadmon gets FDA go-ahead for Rock2 inhibitor study. Biopharmadive.com https://www.biopharmadive.com/news/kadmon-gets-fda-go-ahead-for-rock2-inhibitor-study/521086/. Accessed April 12, 2018.
2. KD025 in Chronic Graft-Versus-Host Disease. Kadmon.com http://kadmon.com/research-development/pipeline/. Accessed April 12, 2018.
3. A Study to evaluate the Safety, Tolerability, and Activity of KD025 in Subjects With Chronic graft Versus Host Disease. Clinicaltrials.gov. https://clinicaltrials.gov/ct2/show/record/NCT02841995?term=KD025&cond=GVHD&rank=1. Accessed April 12, 2018.