Kadmon Announces Receipt of Final Meeting Minutes from FDA Following Pre-NDA Meeting for KD025 for the Treatment of cGVHD
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Kadmon Announces Receipt of Final Meeting Minutes from FDA Following Pre-NDA Meeting for KD025 for the Treatment of cGVHD

Tuesday, April 14, 2020 7:59 AM
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- Kadmon on Track for NDA Submission for KD025 in Q4 2020 -

- Company to Announce Top-line Results from Primary Analysis in Q2 2020 -

NEW YORK, NY / ACCESSWIRE / April 14, 2020 / Kadmon Holdings, Inc. (NYSE:KDMN) today announced that it has received the final meeting minutes from the U.S. Food and Drug Administration (FDA) following its recent New Drug Application (NDA) pre-submission meeting for KD025 for the treatment of patients with chronic graft-versus-host disease (cGVHD). The purpose of the meeting was to discuss the content and timing of the Company's planned NDA submission.

Based on the feedback from the FDA, the Company believes the proposed data package will be sufficient to support a submission of KD025 for the treatment of patients with cGVHD; acceptance of the final NDA will be subject to the FDA's review of the complete filing.

The Company is on track to submit the NDA with data from the primary analysis of ROCKstar (KD025-213), its ongoing pivotal trial of KD025 in cGVHD, with a cutoff date six months after completion of enrollment. As previously reported, Kadmon announced enrollment completion in ROCKstar in August 2019. As requested by the FDA, the Company plans to submit one-year follow-up data from the study within 60 days of the initial submission, in the fourth quarter of 2020.

As previously announced, Kadmon remains on track to announce top-line results from the primary analysis of ROCKstar in the second quarter of 2020.

"We are very pleased with the outcome of our pre-NDA meeting and remain encouraged by our ongoing discussions with the FDA, which we believe support our efforts to bring KD025 to cGHVD patients in need as quickly as possible," said Harlan W. Waksal, M.D., President and CEO of Kadmon. "We look forward to continuing to work closely with the agency as we prepare to submit our NDA later this year."

Dr. Waksal continued, "We are fortunate that prior to the COVID-19 outbreak in the United States, we completed the last patient visit for the primary analysis of ROCKstar, and therefore do not anticipate significant delays in collecting and finalizing the data. We remain on track to submit our NDA in the fourth quarter of 2020 and believe our overall submission timeline for KD025 will not be meaningfully impacted by COVID-19."

As previously reported, Kadmon met the primary endpoint of Overall Response Rate (ORR) at the interim analysis of ROCKstar, conducted as scheduled two months after completion of enrollment. KD025 showed statistically significant and clinically meaningful ORRs of 64% with KD025 200 mg once daily (95% Confidence Interval (CI): 51%, 75%; p<0.0001) and 67% with KD025 200 mg twice daily (95% CI: 54%, 78%; p<0.0001). Response rates were consistent across key subgroups, including in patients with severe cGVHD (n=87; 61%), patients previously treated with ibrutinib (n=45; 62%), patients previously treated with ruxolitinib (n=37; 62%) and patients who had no response to their last line of treatment (n=74; 68%). KD025 has been well tolerated, and no apparent increased risk of infection has been observed.

In October 2018, KD025 was granted Breakthrough Therapy designation by the FDA 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 for KD025 for the treatment of cGVHD.

About ROCKstar (KD025-213)

ROCKstar is an ongoing open-label 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 KD025 200 mg BID, enrolling 66 patients per arm. Statistical significance is achieved if the lower bound of the 95% CI of ORR exceeds 30%, which was achieved in both arms of the trial at the interim analysis.

About KD025

KD025 is a selective oral inhibitor of Rho-associated coiled-coil kinase 2 (ROCK2), a signaling pathway that modulates immune response as well as fibrotic pathways. In addition to cGVHD, KD025 is being studied in an ongoing Phase 2 clinical trial in adults with diffuse cutaneous systemic sclerosis (KD025-209).

About 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, and approximately 5,000 new patients are diagnosed with cGVHD per year.

About Kadmon

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 impact of COVID-19 on our ability to conduct our business or our clinical trials, (ii) the initiation, timing, progress and results of our preclinical studies and clinical trials, and our research and development programs; (iii) our ability to advance product candidates into, and successfully complete, clinical trials; (iv) our reliance on the success of our product candidates; (v) the timing or likelihood of regulatory filings and approvals, including an NDA regarding KD025; (vi) our ability to expand our sales and marketing capabilities; (vii) the commercialization of our product candidates, if approved; (viii) the 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, China, Japan 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 or obtain additional grant funding; (xvii) the rate and degree of market acceptance of our product candidates; (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) 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 (xxiii) 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.

Contact Information

Ellen Cavaleri, Investor Relations
646.490.2989
[email protected]

SOURCE: Kadmon Holdings, Inc.

Kadmon Holdings, Inc.
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