On March 4, 2021 Kadmon Holdings, Inc. (NASDAQ:KDMN) reported financial and operational results for the fourth quarter and full year ended December 31, 2020 (Press release, Kadmon, MAR 4, 2021, View Source [SID1234576084]).

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"With the acceptance of Kadmon’s new drug application for belumosudil in cGVHD in hand, we are preparing for our potential commercial launch in light of our May 30th PDUFA date," said Harlan W. Waksal, M.D., President and CEO of Kadmon. "We are leveraging our existing commercial infrastructure and have thus nearly completed the scale-up required to launch belumosudil, if approved. We continue to seek to understand the needs of cGVHD patients in key transplant centers throughout the United States and look forward to working closely with FDA to bring this much-needed therapy to cGVHD patients in the coming months."

Dr. Waksal added, "We remain enthusiastic about belumosudil’s potential in systemic sclerosis; our ongoing placebo-controlled Phase 2 trial continues to attract patients in spite of the broader COVID backdrop and our plans to initiate our open-label trial in this indication are squarely on track. Additionally, we recently completed enrollment in the first two cohorts of our Phase 1 clinical trial of KD033, our anti-PD-L1/IL-15 fusion protein, in patients with metastatic or locally advanced solid tumors. We look forward to sharing initial data from both of these programs in late 2021. By completing a transformative convertible bond financing in February 2021 that was led by fundamental, healthcare investors, we have over $300 million in capital and are well positioned to achieve our strategic goals."

2021 Anticipated Key Clinical Milestones:

Belumosudil

Continue ongoing dialogue with the U.S. Food and Drug Administration (FDA) as they review the New Drug Application (NDA) of belumosudil, the Company’s (ROCK2) inhibitor, for the treatment of patients with chronic graft-versus-host disease (cGVHD).
The FDA set a Prescription Drug User Fee Act (PDUFA) date for belumosudil of May 30, 2021. The NDA is being reviewed under the FDA’s Real-Time Oncology Review (RTOR) pilot program, which aims to explore a more efficient review process to ensure safe and effective treatments are available to patients as early as possible.
The review of the belumosudil NDA is also being conducted under Project Orbis, an initiative of the FDA Oncology Center of Excellence. Project Orbis provides a framework for concurrent submission and review of oncology drugs among participating international countries.
Continue belumosudil commercial launch readiness activities in anticipation of potential FDA approval in 1H 2021. The Company has the majority of its planned field sales and medical science liaison teams. The Company believes these focused teams can adequately address the concentrated cGVHD market, where approximately 80% of patients are treated at 70 U.S. transplant centers.
Initiate small (12-15 patient), open-label Phase 2 clinical trial of belumosudil in patients with dcSSc (Study KD025-215) in 2021. The Company plans to present initial data from this study by year-end 2021.
Continue enrollment in ongoing placebo-controlled Phase 2 clinical trial in dcSSc (KD025-209); the Company continues to work with sites and trial coordinators to facilitate patient enrollment amid the COVID-19 pandemic
KD033

Enrollment in the first two dose cohorts was successfully completed in the ongoing dose-escalation Phase 1 clinical trial of KD033, Kadmon’s anti-PD-L1/IL-15 fusion protein, in patients with metastatic or locally advanced solid tumors; enrollment is ongoing in the next dose level (cohort 3).
The Company plans to present initial data from KD033-101 in 2021.
KD045

Continue investigational new drug (IND)-enabling activities for KD045, Kadmon’s next-generation ROCK inhibitor for the treatment of fibrotic diseases.
Financial Results

Fourth Quarter 2020 Results

Loss from operations for the three months ended December 31, 2020 was $31.2 million compared to $18.3 million for the same period in 2019.

The increase in loss from operations for the three months ended December 31, 2020 as compared to 2019 was primarily attributable to increased expenses related to clinical development and regulatory costs for belumosudil, as well as costs related to preparation for the potential launch of belumosudil. Additionally, the fourth quarter of 2019 included $4.0 million in license revenues related to the BioNova strategic partnership.

Full Year 2020 Results

Loss from operations for the year ended December 31, 2020 was $102.3 million, compared to $89.1 million for the same period in 2019.

The increase in loss from operations for the year ended December 31, 2020 as compared to 2019 was primarily related to clinical development and regulatory costs for belumosudil, as well as costs related to preparation for the potential launch of belumosudil. This increase in expenses was partially offset by an increase in license revenues of $2.0 million related to the Meiji strategic partnership.

Liquidity and Capital Resources

At December 31, 2020, the Company’s cash, cash equivalents and marketable debt securities totaled $123.9 million, compared to $139.6 million at December 31, 2019. On February 16, 2021, the Company issued $240.0 million aggregate principal amount of 3.625% convertible senior notes due 2027 (the "Notes"), in a private offering conducted in accordance with Rule 144A under the Securities Act of 1933, which included the full exercise of the $40 million over-allotment option. The initial conversion price of the Notes is equivalent to $6.96 per share, a premium of approximately 30% over the $5.35 per share closing price of Kadmon’s common stock on February 10, 2021. The Company entered into capped call transactions to raise the conversion price to $10.70 per share, a premium of 100% over the $5.35 per share closing price of Kadmon’s common stock on February 10, 2021. The Company received net proceeds from the offering of approximately $200.0 million, after deducting the initial purchasers’ discount and the cost of the capped call transactions.

In addition, as of December 31, 2020, the Company held approximately 0.7 million ordinary shares of MeiraGTx Holdings plc (MeiraGTx), a clinical-stage gene therapy company, as compared to approximately 2.1 million ordinary shares held as of December 31, 2019. During fiscal year ended December 31, 2020, the Company entered into multiple transactions pursuant to which it sold approximately 1.4 million ordinary shares of MeiraGTx for total net proceeds of approximately $19.8 million.

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. In November 2020, the U.S. Food and Drug Administration (FDA) accepted the NDA for belumosudil for the treatment of patients with cGVHD. The FDA granted Priority Review for the NDA for belumosudil and assigned a Prescription Drug User Fee Act (PDUFA) target action date of May 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 as well as Orphan Drug Designation to belumosudil for the treatment of cGVHD.