Aspyrian Therapeutics Inc. announces successful advances in RM-1929 clinical development in recurrent Head and Neck Cancer, including Fast Track designation granted by the FDA, initiation of clinical studies in Japan, and plans to start pivotal studies, which will incorporate the evaluation of anti-cancer immune responses, in early 2018.

 Aspyrian Therapeutics, Inc., a biotechnology company developing precision-targeted cancer therapies based on a proprietary Photoimmunotherapy (PIT) platform, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for RM-1929, a first-in-class precision targeted therapy, for treatment of patients with locoregional recurrent Head and Neck Squamous Cell Carcinoma (HNSCC) that have failed 2 lines of therapies.

"I am very excited by the progress that Aspyrian has made building on game-changing innovation," said Hiroshi "Mickey" Mikitani, Chairman of Aspyrian. "Aspyrian is aiming to conquer cancer, for life – some might see this as wildly ambitious but we are humbly committed to this as the end goal."

RM-1929 is currently being tested in an ongoing Phase 2 study in recurrent Head and Neck cancer (Study RM-1929/101, NCT02422979). Enrollment has been completed and Aspyrian expects to report top-line data in the second half of 2018.  Aspyrian also recently completed a successful end-of-phase 2 meeting with the FDA to discuss interim clinical data from this Phase 2 study.  The design of pivotal studies was also discussed, including clinical strategies to potentially support accelerated approval under Subpart E, Accelerated Approval of Biological Products for Serious or Life-Threatening Illnesses, Code of Federal Regulations, Title 21, Volume 7. Based on the FDA feedback, Aspyrian plans to initiate the first global pivotal study for RM-1929, to treat recurrent Head and Neck cancer, within the first quarter of 2018.

"We are extremely pleased that the FDA has granted Fast Track designation for RM-1929, a first-in-class precision targeted therapy, for treatment of patients with locoregional recurrent Head and Neck Squamous Cell Carcinoma (HNSCC) that have failed 2 lines of therapies. The Fast Track designation recognizes the potential of RM-1929 to address the large unmet medical need in this clinical setting," said Dr. Merrill Biel, MD, PhD, Chief Medical Officer of Aspyrian. "The interim results of the Phase 2 study showed improvement in the clinically meaningful endpoints of Overall Response Rate, Progression Free Survival and Overall Survival compared to the historical data for the standard of care treatments currently available to these patients. These data support the potential for RM-1929 to control this disease while preserving normal healthy tissues in the head and neck area that are so critical to maintaining the patient's quality of life and we look forward to initiating the pivotal clinical trials."

Pivotal studies will include evaluation of RM-1929 as a single agent and in combination with immunomodulators to treat both locoregional and metastatic head and neck cancer lesions. Studies conducted by Aspyrian have shown that treatment with RM-1929 can induce not only rapid tumor destruction, but also drive innate and adaptive anti-cancer immune responses.

"I congratulate the Aspyrian team for successfully driving the progression of RM-1929 towards pivotal studies in recurrent Head and Neck cancer, to start in early 2018. We are delighted that the FDA granted Fast Track designation to RM-1929 for the treatment of patients with recurrent Head and Neck cancer, illustrating their recognition of the large unmet medical need for this severe disease. Based on the interim Phase 2 data, we believe that RM-1929 as a single agent has the potential to offer effective treatment options for patients that have failed all existing therapies, providing the opportunity to treat with curative intent" said Miguel Garcia-Guzman, President and CEO of Aspyrian. "In addition, based on our preclinical and clinical data, we expect that RM-1929-mediated tumor destruction may serve to trigger innate and adaptive anti-cancer immune responses that have the potential to enhance the destruction of the patient's cancer. Aligned with the visionary leadership of our Chairman Hiroshi Mikitani and our commitment to develop the best possible treatment options for patients, we plan to initiate additional trials evaluating combination treatments of RM-1929 with immunomodulators in early 2018. The combination trials are designed to explore synergistic effects of RM-1929 together with activation of T-cell mediated anti-cancer responses."

In order to advance RM-1929 into clinical studies in Japan, Aspyrian successfully submitted a Clinical Trial Notification (CTN) application for RM-1929 to the PMDA and has initiated a Phase 1, single center, open label clinical study in Japanese patients with recurrent Squamous Cell Head and Neck Cancer.

In addition to RM-1929, Aspyrian is progressing preclinical development of other new proprietary immune oncology approaches that have the potential to eliminate the immunosuppressive behavior associated with tumor microenvironments, and to elicit adaptive systemic immune responses.  This approach is anticipated to destroy tumors that are resistant to other classes of immune modulators, such as anti-PD1 antibodies. Preclinical data have shown that Aspyrian's immune oncology treatment modality has the potential to achieve robust tumor destruction mediated by tumor specific CD8+ T-cells and to elicit long-term cancer‑specific immune memory resulting in robust cancer vaccination. Aspyrian intends to expedite the development of these new immune oncology approaches into clinical studies.

About RM-1929

RM-1929, a conjugate of cetuximab and IRDye 700DX®, targets epidermal growth factor receptors (EGFR), which are 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 uses an antibody conjugate to precisely target cancer cells.  After antibody binding to the tumor, the conjugated dye is locally activated with red light to elicit rapid anti-cancer responses. The dual specificity resulting from local activation of a tumor-selective conjugate has the potential to deliver locoregional tumor control to cancer patients and their doctors, with minimal damage to surrounding healthy tissues and structures.

RM-1929 is an investigational compound that is not approved for any use in any country.