Kadmon (KDMN) Receives Final Meeting Minutes from FDA Following Pre-NDA Meeting for KD025 for Treatment of cGVHD, on Track for NDA Submission in Q4

Kadmon Holdings, Inc. (NYSE: KDMN) today announced that it has received the final meeting minutes from the U.S. Food and Drug Administration (FDA) following its recent New Drug Application (NDA) pre-submission meeting for KD025 for the treatment of patients with chronic graft-versus-host disease (cGVHD). The purpose of the meeting was to discuss the content and timing of the Company's planned NDA submission.

Based on the feedback from the FDA, the Company believes the proposed data package will be sufficient to support a submission of KD025 for the treatment of patients with cGVHD; acceptance of the final NDA will be subject to the FDA's review of the complete filing.

The Company is on track to submit the NDA with data from the primary analysis of ROCKstar (KD025-213), its ongoing pivotal trial of KD025 in cGVHD, with a cutoff date six months after completion of enrollment. As previously reported, Kadmon announced enrollment completion in ROCKstar in August 2019. As requested by the FDA, the Company plans to submit one-year follow-up data from the study within 60 days of the initial submission, in the fourth quarter of 2020.

As previously announced, Kadmon remains on track to announce top-line results from the primary analysis of ROCKstar in the second quarter of 2020.

"We are very pleased with the outcome of our pre-NDA meeting and remain encouraged by our ongoing discussions with the FDA, which we believe support our efforts to bring KD025 to cGHVD patients in need as quickly as possible," said Harlan W. Waksal, M.D., President and CEO of Kadmon. "We look forward to continuing to work closely with the agency as we prepare to submit our NDA later this year."

Dr. Waksal continued, "We are fortunate that prior to the COVID-19 outbreak in the United States, we completed the last patient visit for the primary analysis of ROCKstar, and therefore do not anticipate significant delays in collecting and finalizing the data. We remain on track to submit our NDA in the fourth quarter of 2020 and believe our overall submission timeline for KD025 will not be meaningfully impacted by COVID-19."

As previously reported, Kadmon met the primary endpoint of Overall Response Rate (ORR) at the interim analysis of ROCKstar, conducted as scheduled two months after completion of enrollment. KD025 showed statistically significant and clinically meaningful ORRs of 64% with KD025 200 mg once daily (95% Confidence Interval (CI): 51%, 75%; p<0.0001) and 67% with KD025 200 mg twice daily (95% CI: 54%, 78%; p<0.0001). Response rates were consistent across key subgroups, including in patients with severe cGVHD (n=87; 61%), patients previously treated with ibrutinib (n=45; 62%), patients previously treated with ruxolitinib (n=37; 62%) and patients who had no response to their last line of treatment (n=74; 68%). KD025 has been well tolerated, and no apparent increased risk of infection has been observed.

In October 2018, KD025 was granted Breakthrough Therapy designation by the FDA for the treatment of patients with cGVHD who have received at least two prior lines of systemic therapy. The FDA has also granted Orphan Drug Designation for KD025 for the treatment of cGVHD.

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