On November 5, 2020 Kadmon Holdings, Inc. (Nasdaq: KDMN) reported financial and operational results for the third quarter of 2020 (Press release, Kadmon, NOV 5, 2020, View Source [SID1234570298]).
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"The submission of our belumosudil New Drug Application to the FDA represents a significant achievement for Kadmon and advances our efforts to make this therapy available to patients living with chronic GVHD," said Harlan W. Waksal, M.D., President and CEO of Kadmon. "We look forward to presenting 12-month safety, efficacy and durability data from our ongoing ROCKstar pivotal trial of belumosudil at the ASH (Free ASH Whitepaper) Annual Meeting in December 2020."
Dr. Waksal continued, "Beyond cGVHD, we were pleased to announce that the FDA granted Orphan Designation to belumosudil for the treatment of systemic sclerosis, currently in Phase 2 clinical development. Additionally, we continue to enroll patients with metastatic or locally advanced solid tumors in our Phase 1 clinical trial of KD033, our novel anti-PD-L1/IL-15 immuno-oncology fusion protein. We successfully completed enrollment in the first cohort of this trial; enrollment in the second cohort is ongoing. I am thrilled with the progress we are making across our clinical programs and look forward to sharing updates as we achieve new milestones."
Upcoming Milestones:
Belumosudil (KD025)
Present 12-month data from ROCKstar pivotal trial at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting on December 6, 2020; the presentation will include updated efficacy and safety data and key secondary endpoints including duration of response, Failure-Free Survival, steroid dose reductions and quality-of-life improvements
Continue ongoing dialogue with the U.S. Food and Drug Administration (FDA) as they review the New Drug Application (NDA) under their 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
Continue progressing belumosudil commercial launch readiness activities in anticipation of potential FDA approval in 2021
The Company expects to share an update on the path forward for belumosudil in Europe in 1H 2021
Continue enrollment in ongoing placebo-controlled Phase 2 clinical trial in diffuse cutaneous systemic sclerosis (KD025-209); the Company continues to work with sites and trial coordinators to facilitate patient enrollment amid the COVID-19 pandemic
Initiate small (12-15 patient), open-label Phase 2 clinical trial of belumosudil in patients with diffuse cutaneous systemic sclerosis in Q1 2021
KD033
The first 3-patient dose cohort 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 2)
KD045
Continue ongoing Investigational New Drug Application (IND)-enabling activities of KD045, Kadmon’s next-generation ROCK inhibitor for the treatment of fibrotic diseases
Financial Results
Third Quarter 2020 Results
Loss from operations for the three and nine months ended September 30, 2020 was $28.0 million and $71.0 million, respectively, compared to $23.2 million and $70.8 million for the same periods in 2019.
The increase in loss from operations for the three months ended September 30, 2020 as compared to 2019 was primarily due to an increase in development costs for belumosudil.
Liquidity and Capital Resources
At September 30, 2020, the Company’s cash, cash equivalents and marketable debt securities totaled $150.5 million, compared to $139.6 million at December 31, 2019. The increase primarily reflects $50.0 million in gross proceeds the Company accessed through its At-The-Market (ATM) facility in May 2020 along with $19.8 million in non-dilutive financing the Company accessed through the divestiture of 1.4 million ordinary shares of MeiraGTx Holdings plc during the nine months ended September 30, 2020. As of September 30, 2020, the Company held approximately 0.7 million ordinary shares of MeiraGTx Holdings plc, a clinical-stage gene therapy company.
About Belumosudil (KD025)
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 Company has submitted an NDA for belumosudil for the treatment of patients with cGVHD and the NDA is being reviewed under the FDA’s RTOR pilot program. 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. In addition, belumosudil is in Phase 2 clinical development in patients with diffuse cutaneous systemic sclerosis (SSc). The FDA has granted Orphan Drug Designation to belumosudil for the treatment of systemic sclerosis