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

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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

Tags: Phase 1 Oncology Head and Neck Cancer Fast Track FDA

FDA grants Fast Track designation for RM-1929 to treat recurrent Head and Neck cancer patients that have failed two lines of therapy

Aspyrian plans to initiate pivotal clinical trials for RM-1929 in the first quarter of 2018

Aspyrian has recently initiated Phase 1 clinical trials for RM-1929 in Japan

SAN DIEGO, Jan. 16, 2018 /PRNewswire/ -- 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.

For more information on the clinical trial, please follow this link to the study record on ClinicalTrials.gov: Study of RM-1929 and Photoimmunotherapy in Patients With Recurrent Head and Neck Cancer

About Aspyrian Therapeutics Inc.Aspyrian Therapeutics, Inc., is a privately funded clinical stage biotechnology company developing a new class of precision targeted oncologic drugs for the treatment of solid tumors.

Aspyrian's mission is "Conquering Cancer. For Life". Aspyrian's vision and strategy is aligned with this long-term mission, by building a leading, patient-focused corporation that integrates research, development, and commercial operations to provide the most value to our patients, society, employees, partners, and investors.

Aspyrian has secured the exclusive license of the Photoimmunotherapy platform from the National Cancer Institute (NCI) in the USA, and has licensed the dye IRDye 700DX® from LI-COR (Lincoln, Nebraska, USA) for development of Photoimmunotherapy products. The Company is currently working with a number of monoclonal antibodies with the potential to treat various types of cancers, including head and neck, esophageal, lung, brain, pancreatic, colorectal, breast, glioblastoma, and ovarian.

Contacts:

For more information about this press release or for business or partnering questions please contact Abhijit Bhatia at: [email protected]

Aspyrian Therapeutics Inc. Forward Looking StatementsThis 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 Aspyrian Therapeutics Inc. 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 Aspyrian Therapeutics' products such as RM-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 RM-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 similar expressions, and are based on our current beliefs and expectations. 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 RM-1929 which may be impacted by, among other things, problems with the manufacturing process for RM-1929, the occurrence of adverse safety events, failure to demonstrate therapeutic benefit, and the other risks and uncertainties. We undertake no obligation to publicly update any forward-looking statement, whether because of new information, future developments or otherwise.

SOURCE Aspyrian Therapeutics, Inc.

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