Rubius Therapeutics Reports First Quarter 2022 Financial Results and Provides Business Update
Presented Updated Clinical Data from Monotherapy Phase 1 Arm of RTX-240 Clinical Trial in Advanced Solid Tumors, Showing Clinical Responses with Favorable Tolerability in PD-(L)1 Refractory Disease
Expanded Ongoing Phase 1 Arm of RTX-240 + Pembrolizumab to Focus on Non-Small Cell
Dosing Patients in Phase 1 Clinical Trial of RTX-224 in Select Solid Tumors; Initial Results Expected 4Q’22 or 1Q’23
Successfully Scaled Manufacturing to 200L Bioreactors
Company Provides Update on Phase 1 Clinical Trial of RTX-321 in Advanced Human Papilloma Virus (HPV) 16-Positive Cancers
“With the updated results showing single-agent activity and encouraging tolerability of monotherapy RTX-240 in patients whose disease progressed on PD-(L)1 inhibitors, we believe we have the opportunity to develop RTX-240 as a combination therapy with immune checkpoint inhibitors in earlier lines of therapy, where the greatest need exists for patients with NSCLC and RCC,” said
Recent Highlights
Broad Immune Stimulation
RTX-240
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.
Monotherapy RTX-240 in Advanced Solid Tumors
- Reported updated clinical data from the monotherapy Phase 1 arm of RTX-240 in relapsed/refractory or locally advanced solid tumors at the
American Association for Cancer Research Annual Meeting inApril 2022 - Results included updated safety (n=34) and efficacy (n=27) data from 9 completed dose cohorts at the time of the
March 4, 2022 , data cutoff - There were three partial responses (PR) in NSCLC, anal cancer and uveal melanoma patients:
- an unconfirmed PR (uPR) with 41% decrease of all target lesions and a notable decrease of an external protruding chest wall mass in a patient with NSCLC whose disease had progressed on prior anti-PD-L1 therapy;
- a confirmed PR with a 54% reduction in the target lesions in a patient with metastatic anal cancer whose disease had progressed on anti-PD-L1 therapy; and
- a uPR with 100% decrease of the target hepatic lesion and resolution of multiple non-target hepatic lesions in a patient with metastatic uveal melanoma whose disease had progressed on anti-PD-1 therapy
- an unconfirmed PR (uPR) with 41% decrease of all target lesions and a notable decrease of an external protruding chest wall mass in a patient with NSCLC whose disease had progressed on prior anti-PD-L1 therapy;
- The uPR in NSCLC and 5 cases of stable disease (SD) were observed across the 3e10 cohorts, including 3 SDs in patients with metastatic NSCLC and 2 with RCC supporting the Company’s decision to expand the Phase 1 arm of RTX-240 plus pembrolizumab to NSCLC and RCC patients
- One patient each with NSCLC and RCC remained on monotherapy treatment with SD greater than 6 months as of the cutoff date
- All of these patients had experienced disease progression on prior anti-PD-(L)1 therapy
- One patient each with NSCLC and RCC remained on monotherapy treatment with SD greater than 6 months as of the cutoff date
- RTX-240 was shown to have been generally well tolerated with no treatment-related or investigator-identified immune-related Grade 3/4 adverse events (AE’s) and no dose-limiting toxicities.
- Based on the totality of clinical, tolerability and pharmacodynamic data, a recommended monotherapy Phase 2 dose of 5e10 cells administered every 3 weeks was selected
- This dose is being further explored in the combination expansion cohort of NSCLC and RCC patients
- Enrollment continues in the monotherapy arm of the trial at the recommended Phase 2 dose of 5e10 cells administered every 3 weeks
RTX-240 + Pembrolizumab in Advanced Solid Tumors
- Advanced enrollment in the Phase 1 combination arm of RTX-240 plus pembrolizumab in patients with advanced solid tumors
- Expanded ongoing Phase 1 arm to enroll up to 20 patients each with NSCLC and RCC who are less heavily pretreated in preparation for a future Phase 2 clinical trial of RTX-240 in combination with pembrolizumab in an earlier line of therapy
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.
- Continuing dose escalation in the Phase 1/2 clinical trial of RTX-224 in selected relapsed/refractory or locally advanced solid tumors that include 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
Antigen-Specific Immune Stimulation
RTX-321 Artificial Antigen-Presenting Cell (aAPC)
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 and is designed to mimic human T cell-APC interactions.
Three dose cohorts were completed (n=9) with one patient with anal squamous cell carcinoma with SD ongoing at 16 weeks at the highest dose cohort to date of 1e10 administered every three weeks. Prior to enrollment, the patient experienced disease progression on anti-PD-1 therapy. RTX-321 was generally well tolerated with no DLTs or Grade 3/4 treatment-related AE’s. Consistent with the combined mechanism of action of IL-12 and 4-1BBL, increases in activated CD4+ T cells, activated CD8+ T cells and activated NK cells were observed at the higher dose levels.
Manufacturing
- Scaled manufacturing to 200L bioreactors in support of a potential future pivotal trial for RTX-240 and potential commercialization
- This scaleup represents 4 times more cells than what was produced using the 50L bioreactor
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 within the next 12 months and 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.
First Quarter 2022 Financial Results
Net loss for the first quarter of 2022 was
In the first quarter of 2022, Rubius invested
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 |
||||||||||
| 2022 | 2021 | |||||||||
| Revenue | $ | $ | ||||||||
| Operating expenses: | ||||||||||
| Research and development | 38,299 | 27,677 | ||||||||
| General and administrative | 12,563 | 13,240 | ||||||||
| Total operating expenses | 50,862 | 40,917 | ||||||||
| Loss from operations | (50,862 | ) | (40,917 | ) | ||||||
| Other income (expense), net | (1,550 | ) | (1,413 | ) | ||||||
| Net loss | $ | (52,412 | ) | $ | (42,330 | ) | ||||
| Net loss per share, basic and diluted | $ | (0.58 | ) | $ | (0.51 | ) | ||||
| Weighted average common shares outstanding, basic and diluted: | 90,149,049 | 82,314,577 | ||||||||
Condensed Consolidated Balance Sheet Data
(in thousands)
(unaudited)
| 2022 | 2021 | ||||
| Cash, cash equivalents and investments | $ | 176,517 | $ | 225,848 | |
| Total assets | 267,123 | 318,021 | |||
| Total liabilities | 132,474 | 139,239 | |||
| Total stockholders’ equity | 134,649 | 178,782 | |||
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-224, RTX-240 and RTX-321, beliefs about the opportunities for and advantages of our drug candidates, our interpretations of data, including as to the efficacy of our product candidates, expectations regarding the therapeutic potential and safety profile of our pipeline candidates, beliefs about patients’ needs, expectations for our cash position, as well as beliefs about our manufacturing plans and accomplishments. 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, our ability to execute on our plans and expectations, our analyses of clinical and preclinical data and other risks identified in our filings with the
Contacts:
Investors
lori.murray@rubiustx.com
Media
marissa.hanify@rubiustx.com
Source: Rubius Therapeutics