SAN DIEGO /PRNewswire/ -- Rakuten Aspyrian, a biotechnology company developing precision-targeted cancer therapies based on its proprietary Photoimmunotherapy platform, today announced that enrollment is underway in its global, pivotal Phase 3 clinical trial evaluating lead product candidate ASP-1929 in patients with recurrent local regional head and neck squamous cell carcinomas (HNSCC). Rakuten Aspyrian expects to enroll 275 patients at 75 sites in the U.S., Europe and Asia.
"We look forward to rigorously evaluating ASP-1929 in this pivotal trial to determine if our Photoimmunotherapy results in improved survival and quality of life for patients with few treatment options," said Mickey Mikitani, chief executive officer of Rakuten Aspyrian. "We believe that treatment with ASP-1929 and Photoimmunotherapy can lead to the selective destruction of head and neck cancer cells and provide an effective therapy to manage the disease."
The multi-center, randomized, open-label study called LUZERA-301 will test the effectiveness, safety and tolerability of ASP-1929 in patients with late-stage, recurrent, local regional HNSCC who have previously failed at least two lines of therapy and are not eligible for surgery or radiation. Participants will be randomized to receive experimental therapy or investigator's choice of systemic therapy (2:1). In the experimental arm of the study, patients will receive up to eight cycles of intravenous doses of ASP-1929 each followed by fixed amounts of red light applied at the tumor site with sufficient energy to activate the drug and induce cancer cell necrosis. In the control arm, investigator's choice may include methotrexate, docetaxel or cetuximab.
The dual-primary endpoints of the study are progression-free survival and overall survival, and multiple secondary endpoints include objective response rate, complete response rate and validated quality of life measurements. The study includes a pre-specified interim analysis for efficacy and if the results are significant, Rakuten Aspyrian may submit them to the U.S. Food and Drug Administration for consideration for early approval.
"Leading oncology research institutes in the U.S., Japan and Europe are participating in this first-ever randomized Phase 3 trial of ASP-1929 Photoimmunotherapy for third-line treatment of recurrent locoregional head and neck cancer, including those that participated in our previous Phase 1 and 2 clinical trials," said Merrill Biel, M.D., Ph.D., F.A.C.S., chief medical officer of Rakuten Aspyrian. "Interim results from previous studies of our first-in-class therapy support the potential for ASP-1929 to treat this deadly disease while preserving normal healthy tissues in the head and neck area that are so critical to maintaining a patient's quality of life. We very much look forward to the potential realization of this novel therapy to treat a disease of medical need."
About Head and Neck Cancer
Head and neck cancer is the sixth most commonly diagnosed cancer and the eighth most common cause of cancer death worldwide.1 Squamous cell carcinoma accounts for greater than 90% of all head and neck cancer. More than 650,000 new cases are diagnosed globally each year.2 Five-year survival rates among patients with stage III and IV HNSCC have only marginally improved over the past 30 years.3
The treatment outcomes for patients with recurrent HNSCC who have failed two lines of therapy remain poor, with essentially no curative options and very low response rates. Due to the poor prognosis of the patients, there is a need for new therapeutic modalities that would provide recurrent refractory HNSCC patients with treatment options that are targeted, minimally invasive, have limited systemic side effects and provide for improved tumor response and control and quality of life.
About ASP-1929 Photoimmunotherapy
ASP-1929, a conjugate of cetuximab and IRDye 700DX®, targets epidermal growth factor receptor (EGFR), a cancer antigen expressed in multiple types of solid tumors, including head and neck squamous cell carcinomas, esophagus, lung, colon, pancreas and other cancers. This first-in-class therapy targets cancer cells, after which the compound is locally activated with red light using a proprietary investigational laser and fiber optics. The local activation of the tumor-selective conjugate targets the tumor but not surrounding normal tissues and structures. ASP-1929 received Fast Track designation by the U. S. Food and Drug Administration for the treatment of HNSCC.
Interim results of a Phase 1/2 trial in patients with HNSCC showed a clinically meaningful improvement in the objective response rate, and potential improvements in progression free survival and overall survival when compared to historical data for the standard of care treatments currently available to this patient population. Top line results of the ASP-1929 Phase 1/2 trial are expected in the first quarter of 2019.
ASP-1929 is an investigational compound that is not approved for any use in any country.
Forward Looking Statements
This press release contains forward-looking statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995 that involve risks, uncertainties and assumptions that could cause Rakuten Aspyrian actual results, plans and experience to differ materially from anticipated results and expectations expressed in these forward-looking statements. These "forward-looking" statements include statements relating to, among other things, the commercialization efforts and other regulatory or marketing approval efforts pertaining to Rakuten Aspyrian's products such as ASP-1929. Such approvals or success may not be obtained or achieved on a timely basis or at all. Forward-looking statements include statements relating to the potential benefits, safety and efficacy of ASP-1929, and the status of current regulatory filings. These statements may be identified by words such as "anticipates," "believes," "hopes," "estimates," "looks," "expects," "intends," "potential," "may," "suggest, "plan," "strategy," "should," "will" and all other similar expressions, and are based on our current beliefs. In addition, this press release includes qualifying terms such as "significant," "remarkable," "extraordinary," etc. that describe opinions on clinical data. Ongoing clinical studies involve risks and uncertainties that could cause actual results to differ materially from those reflected in such statements, including uncertainty of success in regulatory approval or commercialization of ASP-1929 which may be impacted by, among other things, problems with the manufacturing process for ASP-1929, the occurrence of adverse safety events, failure to demonstrate therapeutic benefit, and the other risks and uncertainties, both reasonable and unreasonable. We undertake no obligation to publicly update this or any other forward-looking statement, whether because of new information, future developments or events, changes in assumptions, changes in the factors affecting forward-looking statements or otherwise except to the extent required by applicable laws. If Rakuten Aspyrian updates one or more forward-looking statement(s), no inference should be drawn that we will make additional updates with respect to that, those or other forward-looking statements.