Select Fourth Quarter 2016 and Recent Corporate Highlights
Select Business and Corporate Highlights
Building on the pioneering discovery of RNAi by RXi founder and Nobel Laureate, Dr. Craig Mello, scientists at RXi have harnessed the naturally occurring RNAi process which has the ability to "silence" or down-regulate the expression of a specific gene that may be overexpressed in a disease condition. RXi developed a robust RNAi therapeutic platform, including self-delivering RNA (sd-rxRNA®) compounds, that have the ability to highly selectively block the expression of any target in the genome, thus providing applicability to many therapeutic areas.
Immuno-Oncology
In March 2015, MirImmune Inc., a privately-held company focused on the development of next generation immunotherapies for the treatment of cancer, entered into an exclusive license agreement for use of RXi's sd-rxRNA technology in developing innovative cell-based cancer immunotherapies. MirImmune's progress in cell therapy using RXi's technology formed a strong foundation for therapeutic development in the immuno-oncology space. As a result, RXi entered into an agreement to acquire MirImmune which was completed earlier this year. The Company's goal, through internal research and external partnerships, is to develop more effective treatments resulting in better quality of life and extended survival for patients.
The Company has initiated a program developing cell-based immunotherapies to treat cancer based on its proprietary sd-rxRNA therapeutic compounds. To date, the unique applicability of sd-rxRNA for immune checkpoint modulation in cellular immuno-oncology therapies has been demonstrated, including:
Selection of lead sd-rxRNA compounds against six different extracellular and intracellular immune check points
Demonstrated silencing of all tested checkpoint targets in vitro, singly and in combinations
Efficient and long-lasting silencing of immune checkpoints in vivo
Applicability of sd-rxRNA transfection in cell therapy to solid tumors
Filing of intellectual property that covers the use of RNAi compounds for use in cell therapy
To support this ongoing initiative, Alexey Eliseev, PhD has been appointed as RXi's Chief Business Officer. Dr. Eliseev is a highly accomplished leader with over 20 years of experience in academia, biotechnology industry and venture capital and most recently was the founder and CEO of MirImmune Inc. In addition, RXi has appointed two leading oncology experts to its Scientific Advisory Board. RXi's new SAB members are Dr. Rolf Kiessling, Professor in Experimental Oncology at Karolinska Institutet and Senior Chief Physician of Radiumhemmet at Karolinska Hospital as well as medical oncology expert Dr. James D. Griffin, Chairman, Department of Medical Oncology, Dana-Farber Cancer Institute. Dr. Griffin also serves as Professor, Medicine, Harvard Medical School and Director, Medical Oncology, Brigham and Women's Hospital.
Dermatology
The Company's ongoing Phase 2 clinical trial, RXI-109-1402, is being conducted to evaluate its first clinical candidate RXI-109, an sd-rxRNA compound targeting connective tissue growth factor (CTGF) to reduce scar formation in the skin following scar revision surgery. This study is now fully enrolled and the Company will provide full read-out, for Cohorts 3 and 4, H2-2017.
Samcyprone™, the Company's second clinical candidate, is a topical immunotherapy currently being evaluated in a Phase 2a clinical trial. RXI-SCP-1502 is a multi-center, multi-dose trial conducted in subjects with at least one cutaneous, plantar or periungual wart. The Company expects to share early read-outs H2 2017.
Consumer Health Program
RXi's consumer health compound RXI-231 targets tyrosinase, a key player in the production of melanin. A formulation has been developed at RXi that allows delivery of the compound into the epidermis. We are in the process of finalizing the first two protocols for testing in volunteers. To support these initial studies, RXI-231 was manufactured and is being formulated for topical use.
Ophthalmology
As in dermal scarring, CTGF is known to play a role in retinal scarring. Reduction of CTGF in the eye by RXI-109 treatment may reduce the formation of retinal fibrosis that often accompanies late stage AMD and contributes to permanent vision loss. Enrollment in the first two cohorts in the Company's Phase 1/2 trial, RXI-109-1501, is complete. RXI-109 has been well-tolerated in the eye to date; enrollment into the third cohort at the next higher dose level is ongoing.