Kadmon drops detailed look at cGVHD data; analysts see 'underappreciated potential'

FDA Building 2
Kadmon said it's planning an FDA pre-NDA meeting next month. (FDA)

Kadmon has posted positive and more detailed data out of its so-called ROCKstar pivotal trial of KD025 in chronic graft-versus-host disease (cGVHD).

The data, presented over the weekend at the 2020 Transplantation & Cellular Therapy Meetings, give a deeper look into the initial, interim results originally posted last November.

Then, the biotech said KD025 had met the primary endpoint of overall response rate (ORR) at the study's planned interim analysis and showed statistically significant and clinically meaningful ORRs of 64% with KD025 200 mg once a day and 67% with KD025 200 mg twice a day.

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Now, it says, in the expanded KD025-213 data set, ORRs were “consistent with the previously reported interim analysis across key subgroups, including in patients with four or more organs affected by cGVHD (n=69; 64%), patients who had prior treatment with ibrutinib (n=45; 62%) and patients who had prior treatment with ruxolitinib (n=37; 62%).”

Three patients also hit a complete response. Secondary endpoints won’t be released until later in the year.

Chronic GVHD is an immune condition that occurs after a patient gets a transplant, but the immune cells from the donor (i.e., the graft or graft cells) attack the recipient patient host's tissues. It’s more common after an allogeneic bone marrow transplant (stem cell transplant) and can be life-threatening.

“We are extremely pleased with the interim outcomes of this pivotal trial of KD025 in cGVHD, which track closely our findings from our earlier Phase 2 study. KD025 achieved robust response rates across all subgroups of this difficult-to-treat patient population, who had a median of four prior lines of therapy, and 73% of whom had no response to their last line of treatment,” said Harlan Waksal, M.D., president and CEO of Kadmon.

"We plan to meet with the FDA for a pre-NDA meeting in March 2020 and to announce topline results from the primary analysis of this trial in Q2 2020."

Analysts at Cantor Fitzgerald Strong noted that “safety [was] continuing to show no increased risk of infection.”

A rolling submission is in the cards for mid-year, but the key question, according to Cantor, is “when the clock would start on the review (e.g., after the 6-month cut is submitted or the 12-month).”

Either way, the firm sees the drug as an “underappreciated potential in chronic GVHD, for which we forecast a 2021 US launch and $500M in peak sales.”

They conclude that KD025’s “strong tolerability and safety profile will drive a meaningful treatment duration and a greater-than-appreciated commercial opportunity in cGVHD.”

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