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On November 30, 2020, the Company issued a press release announcing that the U.S. Food and Drug Administration (FDA) has accepted for Priority Review the Company’s New Drug Application (NDA) submission for belumosudil (KD025), the Company's Rho-associated coiled-coil kinase 2 (ROCK2) inhibitor, for the treatment of patients with chronic graft-versus-host disease (cGVHD).
The full text of the press release is attached to this Current Report on Form 8-K as Exhibit 99.1 and is incorporated herein by reference.
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Kadmon Holdings, Inc.
Date: November 30, 2020
Harlan W. Waksal
President and Chief Executive Officer
Kadmon Announces FDA Acceptance of NDA for Belumosudil in Patients with Chronic Graft-Versus-Host Disease
– FDA Grants Priority Review and Sets PDUFA Action Date of May 30, 2021 –
– Application Being Evaluated Under FDA’s Real-Time Oncology Review (RTOR) and Project Orbis Pilot Programs –
NEW YORK, November 30, 2020 – Kadmon Holdings, Inc. (NASDAQ: KDMN) today announced that the U.S. Food and Drug Administration (FDA) has accepted for Priority Review the Company’s New Drug Application (NDA) for belumosudil (KD025), the Company's Rho-associated coiled-coil kinase 2 (ROCK2) inhibitor, for the treatment of patients with chronic graft-versus-host disease (cGVHD). The FDA also granted Priority Review and set a Prescription Drug User Fee Act (PDUFA) date of May 30, 2021 for the completion of its review of the NDA.
“The FDA’s acceptance of our NDA for belumosudil represents an important milestone for Kadmon and further highlights the efforts of the Agency to bring meaningful new therapies to cGVHD patients as quickly as possible,” said Harlan W. Waksal, M.D., President and CEO of Kadmon. “We look forward to the opportunity to bring belumosudil to market as we continue preparations for a launch, if approved.”
Kadmon submitted the NDA for belumosudil on September 30, 2020 under the FDA’s Real-Time Oncology Review (RTOR) pilot program. This pilot program aims to explore a more efficient review process to ensure that safe and effective treatments are available to patients as early as possible, while maintaining and improving review quality.
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 partners.
The FDA had previously granted Breakthrough Therapy Designation (BTD) to belumosudil for the treatment of patients with cGVHD after failure of two or more lines of systemic therapy. The FDA had also granted Orphan Drug Designation to belumosudil for the treatment of cGVHD.
The NDA submission is supported by positive data from ROCKstar (KD025-213), the Company's pivotal clinical trial evaluating belumosudil in patients with cGVHD who have received two or more prior lines of systemic therapy. On November 25, 2020, the Company submitted 12-month follow-up data from the ROCKstar study to the FDA.
ROCKstar (KD025-213) is an ongoing open-label trial of belumosudil in patients with cGVHD who have received at least two prior lines of systemic therapy. Patients were randomized to receive belumosudil 200 mg once daily or 200 mg twice daily, enrolling 66 patients per arm. The primary endpoint of the study is Overall Response Rate (ORR). The ORR endpoint was met at the interim analysis, conducted two months after completion of enrollment. At the study's primary analysis, conducted six months after completion of enrollment, belumosudil achieved ORRs of 73% and 74% in the respective arms. Belumosudil has been well tolerated and adverse events have been consistent with those expected in the patient population.
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. Kadmon has submitted an NDA to the U.S. FDA 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.
cGVHD is a common and often fatal complication following hematopoietic stem cell transplantation. In cGVHD, transplanted immune cells (graft) attack the patient's cells (host), leading to inflammation and fibrosis in multiple tissues, including skin, mouth, eye, joints, liver, lung, esophagus and gastrointestinal tract. Approximately 14,000 patients in the United States are currently living with cGVHD.
Kadmon is a clinical-stage biopharmaceutical company that discovers, develops and delivers transformative therapies for unmet medical needs. Our clinical pipeline includes treatments for immune and fibrotic diseases as well as immuno-oncology therapies.
Forward Looking Statements
This press release contains forward-looking statements. Such statements may be preceded by the words “may,” “will,” “should,” “expects,” “plans,” “anticipates,” “could,” “intends,” “targets,” “projects,” “contemplates,” “believes,” “estimates,” “predicts,” “potential” or “continue” or the negative of these terms or other similar expressions. Forward-looking statements involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements. We believe that these factors include, but are not limited to, (i) the initiation, timing, progress and results of our preclinical studies and clinical trials, and our research and development programs; (ii) our ability to advance product candidates into, and successfully complete, clinical trials; (iii) the impact of the COVID-19 pandemic on our business, workforce, patients, collaborators and suppliers, including delays in anticipated timelines and milestones of our clinical trials and on various government agencies who we interact with and/or are governed by; (iv) our reliance on the success of our product candidates; (v) the timing or likelihood of regulatory filings and approvals, especially in light of the COVID-19 pandemic; (vi) our ability to expand our sales and marketing capabilities; (vii) our ability to expand our sales and marketing capabilities; (viii) the commercialization, pricing and reimbursement of our product candidates, if approved; (ix) the implementation of our business model, strategic plans for our business, product candidates and technology; (x) the scope of protection we are able to establish and maintain for intellectual property rights covering our product candidates and technology; (xi) our ability to operate our business without infringing the intellectual property rights and proprietary technology of third parties; (xii) costs associated with defending intellectual property infringement, product liability and other claims; (xiii) regulatory developments in the United States, Europe, and other jurisdictions; (xiv) estimates of our expenses, future revenues, capital requirements and our needs for additional financing; (xv) the potential benefits of strategic collaboration agreements and our ability to enter into strategic arrangements; (xvi) our ability to maintain and establish collaborations; (xvii) the rate and degree of market acceptance of our product candidates, if approved; (xviii) developments relating to our competitors and our industry, including competing therapies; (xix) our ability to effectively manage our anticipated growth; (xx) our ability to attract and retain qualified employees and key personnel; (xxi) our expected use of cash and cash equivalents and other sources of liquidity; (xxii) the potential benefits of any of our product candidates being granted orphan drug designation; (xxiii) the future trading price of the shares of our common stock and impact of securities analysts’ reports on these prices; (xxiv) our ability to apply unused federal and state net operating loss carryforwards against future taxable income and/or (xxv) other risks and uncertainties. More detailed information about the Company and the risk factors that may affect the realization of forward-looking statements is set forth in the Company’s filings with the U.S. Securities and Exchange Commission (the “SEC”), including Kadmon’s Annual Report on Form 10-K for the fiscal year ended December 31, 2019 and subsequent filings with the SEC. Investors and security holders are urged to read these documents free of charge on the SEC’s website at www.sec.gov. The Company assumes no obligation to publicly update or revise its forward-looking statements as a result of new information, future events or otherwise.
Ellen Cavaleri, Investor Relations