On November 9, 2020 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 third quarter 2020 financial results and provided an overview of operational progress (Press release, Rubius Therapeutics, NOV 9, 2020, View Source [SID1234570326]).
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"Rubius Therapeutics continues to demonstrate strong execution across our pipeline of Red Cell Therapeutics for the treatment of cancer. Our IND filing for RTX-321 included, for the first time, frozen drug substance as part of the manufacturing process, resulting in a truly off-the-shelf cellular therapy with a potential shelf life of up to several years," said Pablo J. Cagnoni, M.D., president and chief executive officer of Rubius Therapeutics. "As we continue to escalate the dose in the RTX-240 solid tumor trial, we are assessing the safety profile and biological effects of RTX-240 on innate and adaptive immune responses. We believe that by demonstrating that RTX-240 is working as intended to induce anti-tumor innate and adaptive immunity, we can unlock the potential of the RED PLATFORM across our entire pipeline of cancer and autoimmune programs."
Third Quarter and Recent Highlights
RTX-240 Phase 1/2 Clinical Program for Advanced Solid Tumors or Relapsed/Refractory Acute Myeloid Leukemia (AML)
Completed dosing of the fourth cohort in the Phase 1/2 solid tumor clinical trial
Assessing the safety profile and biological effects of RTX-240 on innate and adaptive immune responses by measuring biomarkers associated with activation and proliferation of NK and T cells in both peripheral blood and paired tumor biopsy samples across dose levels
Dosed the first patient with relapsed or refractory AML in second Phase 1 arm of ongoing RTX-240 clinical trial
Demonstrating robust momentum in manufacturing and producing clinical supply of RTX-240 for both arms in the Phase 1/2 clinical trial of RTX-240
RTX-321 Artificial Antigen-Presenting Cell (aAPC) Development Program for Human Papillomavirus (HPV) 16-Positive Cancers
Filed an IND with U.S. FDA for RTX-321 for the treatment of HPV 16-positive cancers
For the first time, the IND filing includes frozen drug substance as part of the manufacturing process, resulting in a truly off-the-shelf cellular therapy with a potential shelf life of up to several years
Conducting manufacturing in support of planned GMP clinical supply needs for the RTX-321 clinical trial
Leadership Team
Strengthened the leadership team by appointing Jose "Pepe" Carmona as chief financial officer
Pepe brings to Rubius extensive experience in global finance and operations, as well as a track record of financing clinical-stage and commercial biotech companies
Preclinical Data Presentations
This morning, Rubius presented preclinical data from its lead clinical oncology candidate, RTX-240, at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)’s Annual Meeting, demonstrating:
RTX-240 increased CD8 T cell and NK cell expansion and activation in vitro when compared to a combination of 4-1BB agonist antibody plus recombinant IL-15, which was directly correlated with the percentage of 4-1BBL and IL-15TP expressed on the cell surface
RTX-240 expanded CD56dim NK cells, a cell population with high cytotoxicity
RTX-240 promoted NK cell-killing of a myeloid leukemia cell line, K562, which was accompanied by increased NK cell degranulation and activation
A murine surrogate for RTX-240, mRBC-240, promoted significant expansion of CD8 T cells and NK cells in vivo in a murine model of colorectal cancer (CT26)
mRBC-240 demonstrated potent antitumor activity in a B16F10 melanoma model that was directly correlated with the expansion of terminally differentiated NK cells in the tumors
Presented preclinical data at the Federation of Clinical Immunology Societies Annual Meeting and the American Association of Cancer Research Tumor Immunology and Immunotherapy Conference, from its lead aAPC program, RTX-321, for the treatment of HPV 16-positive tumors demonstrating:
RTX-321 and its mouse surrogates demonstrated a dual mechanism of action in vivo
Functions as an aAPC to boost antigen-specific CD8+ T cell responses
Promotes antigenindependent stimulation of both innate and adaptive immune responses
Mouse surrogates of RTX-321 promote tumor control, memory formation and epitope spreading in tumor models in vivo
Treatment with the RTX-321 mouse surrogate results in minimal, reversible effects in vivo, likely due to biodistribution to the vasculature and spleen
RTX-321 functions as an aAPC to boost HPV 16 antigen-specific T cells in vitro
RTX-321 promotes HPV 16-independent adaptive and innate immune responses in vitro
Third Quarter Financial Results
Net loss for the third quarter of 2020 was $40.9 million or $0.51 per common share, compared to $47.0 million or $0.59 per common share in the third quarter of 2019.
In the third quarter of 2020, Rubius invested $28.2 million in research and development (R&D) related to its novel RED PLATFORM and towards expanding and advancing its product pipeline, as compared to $33.5 million in the third quarter of 2019. The year-over-year decrease was driven by a $7.5 million reduction in costs following the deprioritization of our rare disease pipeline in March 2020. The decrease in rare disease program expenses was offset by $9.6 million in incremental spend to advance our cancer programs, including costs associated with our Phase 1/2 clinical trial for RTX-240 for the treatment of solid tumors as well as costs associated with preclinical and IND-enabling activities for RTX-321. In addition, R&D expenses not allocated to programs decreased by $7.4 million driven primarily by reductions in non-program labor, laboratory supplies, research materials and contract research as pre-GMP manufacturing and discovery activities shifted to support the development of clinical candidates.
G&A expenses were $12.0 million during the third quarter of 2020, as compared to $15.0 million for the third quarter of 2019. The lower costs were principally driven by a reduction in stock-based compensation expense.
Nine Month Financial Results
Net loss for the first nine months of 2020 was $127.2 million or $1.58 per common share, compared to $119.0 million or $1.52 per common share in the first nine months of 2019.
In the nine months ended September 30, 2020, Rubius invested $90.5 million in R&D related to its novel RED PLATFORM and towards expanding and advancing its product pipeline, as compared to $81.9 million in the first nine months of 2019. The year-over-year increase was driven by $28.3 million of incremental costs to advance our cancer programs, including costs incurred for our Phase 1/2 clinical trial for RTX-240 for the treatment of solid tumors as well as costs of preclinical and IND-enabling activities for RTX-321. The increase in cancer program costs was offset by a $12.8 million decrease in expenses related to our rare disease pipeline following the deprioritization of these programs in March 2020. Additionally, costs not allocated to programs decreased by $6.9 million driven primarily by a shift in manufacturing activities towards the technical development and production of clinical supply for our oncology programs and the shift in discovery activities from the development of clinical candidates towards work to support IND enabling activities.
G&A expenses were $36.2 million during the first nine months of 2020, as compared to $42.3 million for the same period in 2019. The lower costs were principally driven by a reduction in stock-based compensation expense.
Cash Position
As of September 30, 2020, cash, cash equivalents and investments were $207.9 million as compared to $283.3 million as of December 31, 2019, providing Rubius with a cash runway into 2022. During 2020, the Company used $97.1 million of cash to fund operations and $4.5 million to fund capital expenditures, consisting mostly of payments for assets purchased in 2019. In addition, during 2020 the Company drew down the third and final tranche of $25.0 million pursuant to its $75.0 million loan agreement with Solar Capital.