On January 11, 2021 Rubius Therapeutics, Inc. (Nasdaq:RUBY), a clinical-stage biopharmaceutical company that is genetically engineering red blood cells to create an entirely new class of cellular medicines called Red Cell Therapeutics, reported an operational update and announced its 2021 objectives. Pablo J. Cagnoni, M.D., chief executive officer, will present these updates and review 2020 achievements on Wednesday, January 13, 2021, at 8:20 a.m. EST at the virtual 39th Annual J.P. Morgan Healthcare Conference (Press release, Rubius Therapeutics, JAN 11, 2021, View Source [SID1234573796]).

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"Rubius Therapeutics made significant progress in advancing our oncology pipeline and in-house manufacturing capabilities in 2020," said Pablo J. Cagnoni, M.D., president and chief executive officer. "To date, in the initial data from the Phase 1 clinical trial of RTX-240 in advanced solid tumors, we have observed activation and expansion of both target cell populations, NK and T cells, indicating the ability of RTX-240 to stimulate both innate and adaptive immune responses. We plan to present additional clinical results in early 2021, and we plan to submit for presentation at a scientific conference."

By showing that RTX-240 is activating and expanding NK and T cells, Rubius Therapeutics believes that the full data set from the Phase 1 clinical trial will unlock the potential of the RED PLATFORM across the entire pipeline of Red Cell Therapeutics for the treatment of cancer.

Clinical Program Updates

RTX-240 Phase 1/2 Clinical Trial for the Treatment of Advanced Solid Tumors and Relapsed/Refactory Acute Myeloid Leukemia (AML)

RTX-240 is an allogeneic, off-the-shelf cellular therapy product candidate that is engineered to simultaneously present hundreds of thousands of copies of the costimulatory molecule 4-1BB ligand and IL-15TP (trans-presentation of IL-15 on IL-15Rα) in their native forms. RTX-240 is designed to broadly stimulate the immune system by activating and expanding both NK and memory T cells to generate a potent anti-tumor response.

To date, Rubius has completed dosing of 5 cohorts (n=14) in the Phase 1/2 RTX-240 solid tumor clinical trial. Trial enrollment continues in additional cohorts.
Key takeaways from initial data to date show:
○ No treatment-related Grade 3 or Grade 4 adverse events and no dose limiting toxicities observed (n=14)
○ All patients showed activation of NK or T cells or both cell types (n=14)
○ In the majority of patients (n=8), all of the following were observed across dose levels:

◾ Activation of NK cells, activation of T cells, expansion of NK cells and expansion of T cells

As more patients are enrolled and data mature, the Company expects to disclose additional clinical results, including
○ additional safety and tolerability data;
○ biomarkers associated with the activation and expansion of NK and T cells in peripheral blood
○ immune cell trafficking into tumors assessed by optional tumor biopsies from participating patients; and
○ potential responses as measured by objective response rate

Enrollment continues in the Phase 1 clinical trial of RTX-240 in relapsed/refractory AML
RTX-321 Artificial Antigen-Presenting Cell (aAPC) Development Program for Human Papillomavirus (HPV) 16-Positive Cancers

RTX-321 is an allogeneic, off-the-shelf aAPC therapy product candidate that is engineered to induce a tumor-specific immune response by expanding antigen-specific T cells. RTX-321 expresses hundreds of thousands of copies of an HPV peptide antigen bound to major histocompatibility complex class I proteins, the costimulatory molecule 4-1BBL and the cytokine IL-12 on the cell surface to mimic human T cell-APC interactions.

The Investigational New Drug (IND) application has been cleared and patients are being screened in the Phase 1 clinical trial of RTX-321 in advanced HPV 16+ cancers, including cervical cancer, head and neck cancer and anal cancer
Manufacturing Update and Recent Achievements

Recognizing the importance of controlling manufacturing to produce consistent and reproducible product at greater scale, Rubius acquired, renovated and operationalized a manufacturing facility in Smithfield, RI. The site has achieved the following milestones:

Provided consistent cGMP supply for the two Phase 1 arms in the ongoing RTX-240 clinical trial in advanced solid tumors and relapsed/refractory AML
○ Increased productivity in manufacturing of cGMP supply of RTX-240 in 50L bioreactors
○ Increased liquid in-vial shelf life from 28 to 52 days for RTX-240
○ Continuously met red blood cell identity (CD233+, mean corpuscular hemoglobin, purity, enucleation cell population) and target product profile criteria (protein expression, cell viability) for clinical supply lots

Completed engineering runs and now producing cGMP supply for the Phase 1 clinical trial of RTX-321 for advanced HPV 16-positive cancers
○ Introduced frozen drug substance for the first time as part of the IND application for RTX-321, resulting in a truly off-the-shelf cellular therapy with a potential shelf life of up to several years
○ Following liquid reformulation, RTX-321 drug product has an in-vial shelf life of 52 days

Significant potential to expand manufacturing capabilities based on future supply needs and for potential commercial production
Preclinical Data 2020 Summary

The Company presented preclinical oncology data for RTX-240 and RTX-321 at the following conferences:

Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting;
Federation of Clinical Immunology Societies (FOCIS) Virtual Annual Meeting;
American Association for Cancer Research (AACR) (Free AACR Whitepaper) Tumor Immunology Conference; and
American Society of Gene & Cell Therapy 23rd Annual Meeting
Anticipated 2021 Catalysts and Operational Objectives

Present additional clinical data for the Phase 1 RTX-240 solid tumor trial in early 2021;
Continue to enroll patients with AML in the second Phase 1 arm of the RTX-240 clinical trial;
Dose the first patient in the RTX-321 clinical trial in HPV-positive tumors;
Continue to produce cGMP material for the RTX-240 and RTX-321 clinical trials; and
Present an integrated clinical program for RTX-240, including plans for expansion cohorts, and oncology pipeline
Listen to the Webcast

A live audio webcast of Dr. Cagnoni’s presentation will be available on January 13, 2021 at 8:20 a.m. EST within the Investors & Media section of the Rubius Therapeutics website. An archived replay will be accessible for 30 days following the event.