NEW YORK, NY / ACCESSWIRE / September 8, 2020 / Kadmon Holdings, Inc. (NYSE:KDMN) today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to belumosudil (KD025), the Company's Rho-associated coiled-coil kinase 2 (ROCK2) inhibitor, for the treatment of systemic sclerosis (SSc). SSc is a chronic immune disorder characterized by fibrosis of the skin and internal organs, affecting approximately 75,000 to 100,000 people in the United States.
"The dual mechanism of belumosudil, which has been shown in preclinical studies to address both the immune and fibrotic components of systemic sclerosis, represents a promising new therapeutic approach for this devastating disease for which new treatments are urgently needed," said Harlan W. Waksal, M.D., President and CEO of Kadmon. "This orphan drug designation adds momentum to our clinical development of belumosudil, which has demonstrated therapeutic potential in several immune disorders."
Belumosudil is being studied in an ongoing double-blind, placebo-controlled Phase 2 clinical trial in adults with SSc. The study is enrolling 60 patients to receive belumosudil 200 mg QD, belumosudil 200 mg BID or placebo for 24 weeks. In addition, the Company plans to initiate a small (12-15 patient), open-label Phase 2 clinical trial of belumosudil in patients with diffuse cutaneous systemic sclerosis in Q1 2021.
Belumosudil is also being developed for the treatment of chronic graft-versus-host disease (cGVHD), a complication following hematopoietic cell transplantation that leads to inflammation and fibrosis in multiple tissues. Kadmon expects to submit a New Drug Application (NDA) for belumosudil in cGVHD in the fourth quarter of 2020.
The FDA grants orphan designation to promote the development of promising products for rare conditions. It qualifies a company for certain financial benefits, including seven years of market exclusivity following marketing approval, tax credits for clinical research costs, eligibility for Orphan Product grants and the waiver of certain administrative fees.
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. The FDA has granted Breakthrough Therapy Designation for belumosudil for the treatment of patients with cGVHD who have received at least two prior 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. The FDA has accepted belumosudil for the treatment of cGVHD for review under its 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.
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; (iv) the timing or likelihood of regulatory filings and approvals, especially in light of the COVID-19 pandemic; (v) our ability to expand our sales and marketing capabilities; (vi) our ability to expand our sales and marketing capabilities; (vii) the commercialization, pricing and reimbursement of our product candidates, if approved; (viii) the implementation of our business model, strategic plans for our business, product candidates and technology; (ix) the scope of protection we are able to establish and maintain for intellectual property rights covering our product candidates and technology; (x) our ability to operate our business without infringing the intellectual property rights and proprietary technology of third parties; (xi) costs associated with defending intellectual property infringement, product liability and other claims; (xii) regulatory developments in the United States, Europe, and other jurisdictions; (xiii) estimates of our expenses, future revenues, capital requirements and our needs for additional financing; (xiv) the potential benefits of strategic collaboration agreements and our ability to enter into strategic arrangements; (xv) our ability to maintain and establish collaborations; (xvi) the rate and degree of market acceptance of our product candidates, if approved; (xvii) developments relating to our competitors and our industry, including competing therapies; (xviii) our ability to effectively manage our anticipated growth; (xix) our ability to attract and retain qualified employees and key personnel; (xx) our expected use of cash and cash equivalents and other sources of liquidity; (xxi) the potential benefits of any of our product candidates being granted orphan drug designation; (xxii) the future trading price of the shares of our common stock and impact of securities analysts' reports on these prices; (xxiii) our ability to apply unused federal and state net operating loss carryforwards against future taxable income and/or (xxiv) 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. 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
SOURCE: Kadmon Holdings, Inc.