On August 9, 2022 Rubius Therapeutics, Inc. (Nasdaq: RUBY), a clinical-stage biopharmaceutical company that is biologically engineering red blood cells to create an entirely new class of cellular medicines called Red Cell Therapeutics for the treatment of cancer and autoimmune diseases, reported second quarter 2022 financial results and provided a business update (Press release, Rubius Therapeutics, AUG 9, 2022, View Source [SID1234617941]).
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"We continue to advance enrollment in the NSCLC and RCC expansion cohorts of our Phase 1 clinical trial of RTX-240 in combination with pembrolizumab, with initial clinical results expected in the second half of 2022, including data in advanced solid tumors and data from the initial patients with NSCLC and RCC enrolled in the study. Enrollment also progressed in the Phase 1/2 clinical trial of RTX-224 in select advanced solid tumors, with initial results expected by year-end or in the first quarter of 2023," said Pablo J. Cagnoni, M.D., president and chief executive officer. "Also, during the quarter, we presented new preclinical data, as part of our type 1 diabetes program, that demonstrated prevention of diabetes and bystander suppression in the stringent non-obese diabetes preclinical model. We plan to select a clinical candidate for our type 1 diabetes program later this year."
Recent Highlights
Oncology
RTX-240 + Pembrolizumab in Advanced Solid Tumors
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 (4-1BBL) 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 CD8+ memory T cells to generate an anti-tumor response.
Advanced enrollment in the expansion cohorts of the Phase 1 combination arm of RTX-240 plus pembrolizumab in patients with advanced solid tumors
Enrolling up to 20 patients who have experienced disease progression with 1-2 prior treatment regimens in the metastatic setting in preparation for a future Phase 2 clinical trial of RTX-240 in combination with pembrolizumab in an earlier line of therapy
Initial clinical results in advanced solid tumors and data from the initial enrolled NSCLC and RCC patients expected in the second half of 2022
RTX-224
RTX-224 is an allogeneic, off-the-shelf cellular therapy product candidate that is engineered to express hundreds of thousands of copies of 4-1BBL and IL-12 on the cell surface. In contrast to RTX-240, RTX-224 is designed as a broad immune agonist of both adaptive and innate responses, activating CD8+ and CD4+ T cells, promoting antigen presentation and activating and expanding NK cells. It is expected to produce a broad and potent anti-tumor T cell response, an innate immune response and have anti-tumor activity in those tumor types with known sensitivity to T cell killing, including tumor types with high mutational burden, PD-L1 expression and prior activity of checkpoint inhibitors.
Continued dose escalation in the Phase 1/2 clinical trial of RTX-224 in select relapsed/refractory or locally advanced solid tumors, including non-small cell lung cancer, cutaneous melanoma, head and neck squamous cell carcinoma, urothelial (bladder) carcinoma and triple-negative breast cancer
Initial clinical results are expected by year-end or during the first quarter of 2023
Autoimmunity
Type 1 Diabetes
RCT product candidates are engineered to express specific autoantigens that are the target of immune responses which cause autoimmune disease. This approach takes advantage of the natural tolerogenic properties of red blood cells. RCTs are designed to suppress, modulate or eliminate disease-causing cells. Type 1 diabetes is a T-cell driven autoimmune disease with well-defined antigens, making it an ideal disease indication for this antigen-specific tolerance approach.
In June, the Company presented new preclinical data for its type 1 diabetes program at the FOCIS 2022 Annual Meeting in San Francisco:
Demonstrated tolerance induction and bystander suppression in stringent type 1 diabetes preclinical models
From ongoing experiment, showed new efficacy data in the NOD preclinical model
By increasing to 3 doses administered and optimizing the dosing schedule, bystander suppression was achieved at 25 weeks by delivering only two antigens, indicating disease prevention caused by many autoantigens
Established efficacy in the BDC2.5 adoptive transfer model with data supporting that repeated dosing extends duration of disease protection, reverses established inflammation, which is important for the treatment of existing autoimmunity, and induces two types of regulatory T cells, resulting in protection against re-challenge
These findings are potentially translatable beyond type 1 diabetes to multiple autoimmune diseases, including multiple sclerosis and celiac disease
Leadership Update
In July 2022, Rubius announced that Susanne Schaffert, Ph.D., joined its board of directors. Dr. Schaffert most recently served as President of Novartis Oncology and brings more than 25 years of experience across clinical development, marketing and sales, finance and commercialization in the global pharmaceutical and biotechnology industries, with a focus on oncology, immuno-oncology and cell therapy.
Anticipated 2022 Catalysts and Operational Objectives
To evaluate the full potential of RTX-240, Rubius’ other oncology programs and the RED PLATFORM, Rubius plans to execute several critical milestones in the near term and, based on its current cash and cash equivalents, including borrowings under its credit facility, has sufficient cash runway into the second half of 2023:
Report initial Phase 1 clinical results for RTX-240 in combination with pembrolizumab in advanced solid tumors and data from the initial enrolled NSCLC and RCC patients in the second half of 2022;
Select a clinical candidate for the first autoimmune program in type 1 diabetes during the second half of 2022; and
Report initial Phase 1 clinical results for RTX-224 for the treatment of advanced solid tumors by year-end or during the first quarter of 2023.
Second Quarter Financial Results
Net loss for the second quarter of 2022 was $44.2 million or $0.49 per common share, compared to $50.2 million or $0.56 per common share in the second quarter of 2021.
In the second quarter of 2022, Rubius invested $33.0 million in research and development (R&D) related to its novel RED PLATFORM and towards expanding and advancing its product pipeline, as compared to $36.1 million in the second quarter of 2021. The year-over-year decrease was principally due to a decrease in direct costs of $2.9 million related to the deprioritization of RTX-321 and RTX-240 AML and monotherapy studies. We expect these costs to continue to decrease in future periods. This decrease was partially offset by an increase in clinical costs related to RTX-224. Platform development, early-stage research and unallocated expenses decreased by $0.1 million primarily due to a decrease of $0.8 million in stock-based compensation expense related to a reduction in the market price of our common stock, resulting in a lower valuation of stock options granted in 2022, and a decrease of $0.3 million in facility-related and other expenses due to lower spend on non-capitalized software costs in the current year. These decreases were partially offset by an increase of $0.4 million in contract research and development related to drug discovery activities and platform development. Additionally, personnel-related costs increased by $0.5 million to support our prioritization of clinical programs.
G&A expenses were $9.9 million during the second quarter of 2022, as compared to $13.9 million for the second quarter of 2021. The lower costs were primarily due to a decrease in stock-based compensation expense of $3.7 million, which was driven by stock option awards that fully vested during the second half of 2021 and first half of 2022, as well as a reduction in the market price of our common stock, resulting in a lower valuation of stock options granted in 2022.
Six Month Financial Results
Net loss for the first six months of 2022 was $96.7 million or $1.07 per common share, compared to $92.5 million or $1.08 per common share in the first six months of 2021.
In the six months ended June 30, 2022, Rubius invested $71.3 million in R&D related to its novel RED PLATFORM and towards expanding and advancing its product pipeline, as compared to $63.7 million in the first six months of 2021. The year-over-year increase was driven primarily by an increase in direct costs of $2.1 million related to an increase in preclinical and clinical costs associated with RTX-224 and RTX-240, principally due to clinical research organization, or CRO, costs and internal manufacturing costs. This increase was partially offset by a decrease in clinical costs related to RTX-321 due to start-up activities in the prior period and current period deprioritization of clinical development. We expect these costs to decrease in future periods. Platform development, early-stage research and unallocated expenses increased by $5.4 million principally due an increase of $2.9 million in personnel-related costs related to headcount increases through the second half of 2021 to support operations. Additionally, increases of $1.7 million in contract research and development and $1.0 million in laboratory supplies and research materials were related to drug discovery activities and platform development. These increases were partially offset by a decrease of $0.4 million in facility-related and other expenses due to lower spend on non-capitalized software costs and a reduction in building operating costs in the current year.
G&A expenses were $22.5 million during the first six months of 2022, as compared to $27.1 million for the same period in 2021. The lower costs were due to a decrease in stock-based compensation expense of $5.0 million, which was driven by stock option awards that fully vested during the second half of 2021 and first half of 2022, as well as a reduction in the market price of our common stock, resulting in a lower valuation of stock options granted in 2022. The decrease was partially offset by an increase in personnel-related expenses of $0.3 million driven by additions to headcount in our general and administrative function.
Cash Position
As of June 30, 2022, cash, cash equivalents and investments were $140.7 million (including $75 million in borrowings under its credit facility), compared to $225.8 million as of December 31, 2021. During the first six months of 2022, the Company used $81.4 million of cash to fund operations, $78.4 million to purchase investments and $4.2 million to fund capital expenditures, consisting mostly of renovation costs incurred at our manufacturing facility.