Rubius Therapeutics Reports Third Quarter 2021 Financial Results and Provides Business Update
Clinical Results Expected by Year-End or First Quarter 2022 in Phase 1 Trials of Single-Agent RTX-240 in Advanced Solid Tumors and Relapsed/Refractory Acute Myeloid Leukemia (AML)
Laurence “Larry” Turka, M.D., Promoted to Head of Research & Translational Medicine
“With the clearance of our latest IND application for RTX-224, we will have three Red Cell Therapeutic™ product candidates for the potential treatment of cancer in the clinic, demonstrating continued strong execution across preclinical and clinical development as well as manufacturing,” said
“Larry’s impressive record of scientific achievement in immunology and ability to foster an environment of innovation has been instrumental in integrating our preclinical and clinical development programs. As Rubius enters a period of potentially significant clinical data generation, we look forward to leveraging Larry’s expertise in translational medicine and building our clinical development strategy,” said
Along with this leadership change, chief medical officer
Third Quarter 2021 and Recent Highlights
Clinical Development in Oncology
- Enrollment continues across Rubius’ portfolio of Red Cell Therapeutics for the treatment of cancer:
- The Phase 1 arms of single-agent RTX-240 in advanced solid tumors and relapsed/refractory AML.
- The Company plans to present comprehensive results by the end of 2021 or during the first quarter of 2022.
- The Phase 1 arm of the ongoing Phase 1/2 RTX-240 clinical trial in combination with KEYTRUDA® (pembrolizumab) for advanced solid tumors.
- To be eligible for the trial, patients must have disease that is relapsed or refractory to an anti-PD-1 or PD-L1 therapy. With its mechanism of action as a broad immune agonist, RTX-240 may have synergy with immune checkpoint inhibition and potentially overcome resistance to PD-1 inhibition.
- The Phase 1 clinical trial of RTX-321 in patients with advanced HPV 16-positive cancers, including cervical, head & neck cancers, and anal cancer.
- The company plans to present initial clinical results during the first quarter of 2022.
- The Phase 1 arms of single-agent RTX-240 in advanced solid tumors and relapsed/refractory AML.
U.S. FDA cleared the IND application for RTX-224, Rubius’ third oncology product candidate.- The company expects to dose its first patient during the first quarter of 2022.
- The company expects to dose its first patient during the first quarter of 2022.
- The Company plans to present preclinical data for RTX-224 at the upcoming
Society for Immunotherapy of Cancer’s (SITC) 36th Annual Meeting onNovember 12 th at8:00 a.m. ET .- The data indicates that RTX-224 activates immune cells in the spleen and blood, leading to their trafficking into the tumor microenvironment to deliver an antitumor effect in preclinical models.
- In
July 2021 , the manuscript entitled “Anti-Tumor Effects of RTX-240: an Engineered Red Blood Cell Expressing 4-1BB Ligand and Interleukin-15” was published in the peer-reviewed journal Cancer Immunology, Immunotherapy, highlighting preclinical findings for RTX-240, which demonstrated that RTX-240 activates and expands CD8+ T cells and NK cells in vitro and in vivo generating potent anti-tumor activity in both a colorectal and melanoma model.
Near-Term Catalysts and Operational Objectives
- Present additional clinical data from the RTX-240 solid tumor Phase 1 clinical trial at the end of 2021 or during the first quarter of 2022;
- Select specific solid tumor types that will be pursued in the Phase 2 expansion cohorts of RTX-240;
- Report initial clinical data from the Phase 1 arm of the RTX-240 clinical trial in relapsed/refractory AML at the end of 2021 or during the first quarter of 2022; and
- Report initial Phase 1 clinical data from RTX-321 for the treatment of HPV 16-positive cancers by the first quarter of 2022.
- Initiate Phase 1 clinical trial for RTX-224 in advanced solid tumors during the first quarter of 2022.
Third Quarter Financial Results
Net loss for the third quarter of 2021 was
In the third quarter of 2021, Rubius invested
General and administrative (G&A) expenses were flat at
Nine Month Financial Results
Net loss for the first nine months of 2021 was
In the nine months ended
G&A expenses were
Cash Position
As of
Condensed Consolidated Statement of Operations
(in thousands, except share and per share data)
(unaudited)
| For the three months ended |
For the nine months ended |
|||||||||||||||
| 2021 | 2020 | 2021 | 2020 | |||||||||||||
| Revenue | $ | $ | $ | $ | ||||||||||||
| Operating expenses: | ||||||||||||||||
| Research and development | 38,014 | 28,209 | 101,763 | 90,491 | ||||||||||||
| General and administrative | 12,035 | 11,976 | 39,126 | 36,241 | ||||||||||||
| Total operating expenses | 50,049 | 40,185 | 140,889 | 126,732 | ||||||||||||
| Loss from operations | (50,049 | ) | (40,185 | ) | (140,889 | ) | (126,732 | ) | ||||||||
| Other income (expense), net | 1,033 | (667 | ) | (637 | ) | (460 | ) | |||||||||
| Net loss | $ | (49,016 | ) | $ | (40,852 | ) | $ | (141,526 | ) | $ | (127,192 | ) | ||||
| Net loss per share, basic and diluted | $ | (0.55 | ) | $ | (0.51 | ) | $ | (1.62 | ) | $ | (1.58 | ) | ||||
| Weighted average common shares outstanding, basic and diluted: | 89,807,383 | 80,778,042 | 87,240,694 | 80,511,543 | ||||||||||||
Condensed Consolidated Balance Sheet Data
(in thousands)
(unaudited)
| 2021 | 2020 | ||||
| Cash, cash equivalents and investments | $ | 264,017 | $ | 176,287 | |
| Total assets | 361,064 | 277,794 | |||
| Total liabilities | 136,859 | 136,234 | |||
| Total stockholders’ equity | 224,205 | 141,560 | |||
About Red Cell Therapeutics
Red Cell Therapeutics™ (RCTs™) are a new class of allogeneic, off-the-shelf cellular therapeutic candidates for the treatment of cancer and autoimmune diseases. For the treatment of cancer, RCTs are expected to provide advantages over other therapies by potentially generating a broad anti-tumor response with limited side effects and a wide therapeutic window given the biodistribution of RCTs to the vasculature and spleen. Additionally, RCTs do not have the complex supply chain and administration logistics of other cell therapies, as RCTs are designed to be prepared in the pharmacy, administered in an outpatient setting and do not require lymphodepletion prior to administration.
About RTX-240
RTX-240, Rubius Therapeutics’ lead oncology program, 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 memory T cells to generate a potent anti-tumor response.
About RTX-321
RTX-321, the Company’s second oncology program, 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.
About 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.
About
Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding beliefs about Rubius’ execution across preclinical and clinical development, Rubius’ plans and expected timing to present clinical results for RTX-240 and RTX-321, beliefs that RTX-240 may have synergy with immune checkpoint inhibition and potentially overcome resistance to PD-1 inhibition, expectations regarding the timing for dosing for the RTX-224 trial, plans to present preclinical data and findings reported in peer-reviewed publications, expectations regarding the therapeutic potential and safety profile of our pipeline of Red Cell Therapeutics, including RTX-240 for the treatment of patients with relapsed/refractory or locally advanced solid tumors and its advantages over other therapies, RTX-321 for the treatment of HPV 16-positive cancers, and RTX-224 and its impact on tumor types with known sensitivity to T cell killing, our interpretations of preliminary data, including indications as to the biological effects of RTX-240 on innate and adaptive immunity, including activation and increased numbers of NK cells and T cells in the clinical trial of RTX-240, expectations for our strengthened leadership team, our expectations regarding our strategy, business plans and focus. The words “may,” “will,” “could,” “would,” “should,” “expect,” “plan,” “anticipate,” “intend,” “believe,” “estimate,” “predict,” “project,” “potential,” “continue,” “target” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management’s current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, those risks and uncertainties related to the development of our Red Cell Therapeutic product candidates and their therapeutic potential and other risks identified in our
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Source: Rubius Therapeutics