Company Remains on Track to Report Interim Analysis by End of 2019
NEW YORK, NY / ACCESSWIRE / August 13, 2019 / Kadmon Holdings, Inc. (NYSE:KDMN) today announced that KD025-213, an open-label registration trial of KD025 for the treatment of chronic graft-versus-host disease (cGVHD), is fully enrolled. The study is being conducted at more than 30 clinical sites throughout the United States. The Company expects to disclose outcomes from a planned interim analysis before year end. Data from this analysis are intended to serve as the basis for a pre-New Drug Application (NDA) meeting with the U.S. Food and Drug Administration (FDA) regarding KD025 in cGVHD.
"The rapid enrollment in this cGVHD trial highlights the importance of evaluating new therapeutic candidates for this complex disease. We look forward to sharing the outcome of our interim analysis by year-end and filing a pre-NDA meeting request with the FDA," said Harlan W. Waksal M.D., President and CEO of Kadmon. "We are grateful to the clinical investigators and their teams, who were instrumental in enrolling the trial in a timely manner, and to the study participants and their families. We believe that KD025 holds significant promise as a potential new treatment option for patients with this devastating disease."
KD025-213 is an open-label registration trial of KD025 in adults and adolescents with cGVHD who have received at least two prior lines of systemic therapy. Patients were randomized to receive KD025 200 mg QD or 200 mg BID, enrolling 63 patients per arm. The primary endpoint is the Overall Response Rate (ORR), defined as the percentage of patients who achieve a complete or partial response at any time point during the study, per the 2014 National Institutes of Health (NIH) overall response criteria. Based on FDA guidance, an ORR of ≥30% is considered clinically significant. The KD025-213 study protocol is based on FDA guidance received in a Type C meeting and was designed in consultation with leading cGVHD experts. The FDA has granted Breakthrough Therapy Designation to KD025 for the treatment of cGVHD following at least 2 prior systemic therapies. The FDA has also granted Orphan Drug Designation to KD025 for the treatment of cGVHD.
KD025 is a selective oral inhibitor of Rho-associated coiled-coil kinase 2 (ROCK2), a signaling pathway that modulates inflammatory response. In addition to cGVHD, KD025 is being studied in an ongoing Phase 2 clinical trial in adults with diffuse cutaneous systemic sclerosis (KD025-209).
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 GI tract. Approximately 14,000 patients in the United States are currently living with cGVHD, and approximately 5,000 new patients are diagnosed with cGVHD per year.
Kadmon is a biopharmaceutical company developing innovative products for significant unmet medical needs. Our product pipeline is focused on inflammatory and fibrotic diseases as well as immuno-oncology.
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) our reliance on the success of our product candidates; (iv) the timing or likelihood of regulatory filings and approvals; (v) our ability to expand our sales and marketing capabilities; (vi) the commercialization of our product candidates, if approved; (vii) the 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 or obtain additional grant funding; (xvi) the rate and degree of market acceptance of our product candidates; (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 ability to achieve cost savings and other benefits from our efforts to streamline our operations and to not harm our business with such efforts; (xxi) the use of proceeds from our recent public offerings; (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; and/or (xxiv) other risks and uncertainties. More detailed information about Kadmon 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 the Company's Quarterly Report on Form 10-Q filed with the SEC on August 5, 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.
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