On May 6, 2021 Kadmon Holdings, Inc. (NASDAQ:KDMN) reported financial and operational results for the first quarter of 2021 (Press release, Kadmon, MAY 6, 2021, View Source [SID1234579440]).

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"We continue to ramp up commercial launch preparation activities for belumosudil in anticipation of the PDUFA goal date of August 30, 2021. Labeling discussions with the FDA are progressing and we believe that belumosudil, if approved, will serve a critical, unmet need for patients with cGVHD," said Harlan W. Waksal, M.D., President and CEO of Kadmon. "In addition, we are exploring the therapeutic potential of belumosudil in systemic sclerosis, a disease with similar manifestations to cGVHD. We continue to enroll patients in two separate Phase 2 studies in this indication and expect to present initial data from the open-label study by year-end 2021. In parallel, we are advancing the ongoing Phase 1 clinical trial of KD033, our anti-PD-L1/IL-15 fusion protein. We look forward to sharing initial safety data from this trial at ASCO (Free ASCO Whitepaper) in June 2021 and additional clinical data in the fourth quarter of 2021."

2021 Anticipated Key Clinical Milestones:

Belumosudil in chronic graft-versus-host disease (cGVHD)

Continue commercial launch readiness activities in anticipation of the Prescription Drug User Fee Act (PDUFA) goal date of August 30, 2021
Belumosudil in diffuse subcutaneous systemic sclerosis (dcSSc)

Continue enrollment in the open-label Phase 2 clinical trial of belumosudil in patients with dcSSc (KD025-215); the Company plans to present initial data from the trial by year-end 2021
Continue enrollment in ongoing placebo-controlled Phase 2 clinical trial in dcSSc (KD025-209)
KD033

Present initial safety data from the ongoing Phase 1 clinical trial of KD033, the Company’s anti-PD-L1/IL-15 fusion protein, in patients with metastatic or locally advanced solid tumors, at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, to be held June 4-8, 2021; additional clinical data from the trial are expected to be available in Q4 2021
Enrollment in the first two dose cohorts of the KD033 Phase 1 trial (KD033-101) was successfully completed; enrollment is ongoing in the next dose level (cohort 3)
Financial Results

First Quarter 2021 Results

Loss from operations for the three months ended March 31, 2021 was $27.4 million compared to $16.1 million for the same period in 2020, which included $6.0 million in one-time license revenues related to the Meiji strategic partnership.

The $5.6 million increase in operating expenses for the three months ended March 31, 2021 as compared to 2020 was primarily related to belumosudil commercial launch readiness activities, as well as research and development costs for KD033 and our preclinical product candidates.

Liquidity and Capital Resources

At March 31, 2021, the Company’s cash, cash equivalents and marketable debt securities totaled $295.9 million, compared to $123.9 million at December 31, 2020.

About Belumosudil

Belumosudil (KD025) is a selective oral inhibitor of Rho-associated coiled-coil kinase 2 (ROCK2), a signaling pathway that modulates inflammatory response and pro-fibrotic processes. The FDA granted Priority Review for the New Drug Application (NDA) for belumosudil for the treatment of cGVHD and has assigned a PDUFA goal date of August 30, 2021. The NDA is being reviewed under the FDA’s Real-Time Oncology Review (RTOR) and Project Orbis pilot programs. The FDA has granted Breakthrough Therapy Designation to belumosudil for the treatment of patients with cGVHD after failure of two or more lines of systemic therapy. The FDA has also granted Orphan Drug Designation to belumosudil for the treatment of cGVHD and for the treatment of systemic sclerosis.