RTX-240 / Rubius Therap - LARVOL DELTA

RTX-240 Monotherapy and in Combination With Pembrolizumab (clinicaltrials.gov) - P1/2 | N=69 | Terminated | Sponsor: Rubius Therapeutics | No DLTs, related deaths or SAEs were reported. RTX-240 cleared circulation rapidly.

Combination therapy • Enrollment change • Monotherapy • Trial completion date • Trial primary completion date • Trial termination • Acute Myelogenous Leukemia • Gastrointestinal Cancer • Lung Cancer • Non Small Cell Lung Cancer • Oncology • Renal Cell Carcinoma • Solid Tumor • IL15 • PD-1 • PD-L1

Dr. Spira on the Examination of RTX-240 in a Phase 1 Trial with Solid Tumors (OncLive) - "Alexander I. Spira, MD, PhD, FACP, discusses the examination of RTX-240 in a phase 1 trial with solid tumors....Moreover, it is an off-the-shelf therapy and doesn't need to be personalized, Spira concludes."

Video

Phase 1 Trial of RTX-240, allogeneic red blood cells engineered to express 4-1BBL and trans-presented IL-15, in patients (Pts) with advanced solid tumors (AACR 2022) - "RTX-240 is well tolerated at all tested doses, schedules and routes. The drug leads to activation, expansion and trafficking of two target cells (memory CD8+ T cells and NK cells) into the TME, exhibits a clear dose response effect with NK cells and demonstrates preliminary evidence of anti-tumor activity. Dose escalation continues and a PD-1 inhibitor combination arm is also enrolling.1"

Clinical • IO biomarker • P1 data • Anal Carcinoma • Eye Cancer • Gastrointestinal Cancer • Lung Cancer • Melanoma • Non Small Cell Lung Cancer • Oncology • Renal Cell Carcinoma • Solid Tumor • Uveal Melanoma • CD8 • IFNG • IL15

RTX-240 Monotherapy and in Combination With Pembrolizumab (clinicaltrials.gov) - P1/2 | N=166 | Active, not recruiting | Sponsor: Rubius Therapeutics | Recruiting ➔ Active, not recruiting

Combination therapy • Enrollment closed • Monotherapy • Acute Myelogenous Leukemia • Gastrointestinal Cancer • Lung Cancer • Non Small Cell Lung Cancer • Oncology • Renal Cell Carcinoma • Solid Tumor • IL15 • PD-1 • PD-L1

Rubius Therapeutics Reports First Quarter 2022 Financial Results and Provides Business Update (GlobeNewswire) - "In the first quarter of 2022, Rubius invested $38.3 million in research and development (R&D) related to its novel RED PLATFORM® and towards expanding and advancing its product pipeline, compared to $27.7 million in the first quarter of 2021. This year-over-year increase was principally due to a $5.3 million increase in costs related to our lead cancer programs, RTX-240, RTX-321 and RTX-224, primarily from clinical research organization (CRO) and internal manufacturing costs incurred in connection with all three programs."

Commercial • Acute Myelogenous Leukemia • Breast Cancer • Cervical Cancer • Gastrointestinal Cancer • Genito-urinary Cancer • Gynecologic Cancers • Head and Neck Cancer • Hematological Malignancies • Leukemia • Lung Cancer • Non Small Cell Lung Cancer • Oncology • Solid Tumor • Squamous Cell Carcinoma of Head and Neck • Triple Negative Breast Cancer • Urothelial Cancer

RTX-240: allogeneic RBCs drive expansion & trafficking of CD8+ T-cells & NK cells into the TME (YouTube) - "Omid Hamid, MD...discusses findings from a Phase I study evaluating RTX-240, an allogeneic cellular therapy genetically engineered to express high copy numbers of trimeric 4-1BBL and IL-15/IL-15Rα fusion proteins on the red blood cell (RBC) surface membrane in order to activate and expand CD8+ memory T-cells and NK cells...."

Interview • Video

RTX-240 Monotherapy and in Combination With Pembrolizumab (clinicaltrials.gov) - P1/2 | N=166 | Recruiting | Sponsor: Rubius Therapeutics | Trial completion date: Jun 2023 ➔ Dec 2023 | Trial primary completion date: Jan 2023 ➔ Jul 2023

Combination therapy • Monotherapy • Trial completion date • Trial primary completion date • Acute Myelogenous Leukemia • Gastrointestinal Cancer • Lung Cancer • Non Small Cell Lung Cancer • Oncology • Renal Cell Carcinoma • Solid Tumor • IL15 • PD-1 • PD-L1

Rubius Therapeutics Reports Updated Clinical Data at AACR from the Ongoing Monotherapy Phase 1 Arm of the Phase 1/2 Clinical Trial of RTX-240 in Advanced Solid Tumors Demonstrating Single-Agent Activity and Favorable Tolerability (GlobeNewswire) - "Upcoming Anticipated Milestones...Report initial Phase 1 clinical results for RTX-321 for the treatment of HPV 16-positive cancers during the second half of 2022; Report initial Phase 1 clinical results for RTX-240 in combination with pembrolizumab in advanced solid tumors and data from the additional NSCLC and RCC patients in the second half of 2022...and Report initial Phase 1 clinical results for RTX-224 for the treatment of advanced solid tumors during the first quarter of 2023."

P1 data • Genito-urinary Cancer • Kidney Cancer • Lung Cancer • Non Small Cell Lung Cancer • Oncology • Renal Cell Carcinoma • Solid Tumor

Rubius Therapeutics Reports Updated Clinical Data at AACR from the Ongoing Monotherapy Phase 1 Arm of the Phase 1/2 Clinical Trial of RTX-240 in Advanced Solid Tumors Demonstrating Single-Agent Activity and Favorable Tolerability (GlobeNewswire) - P1/2 | N=234 | NCT04372706 | Sponsor: Rubius Therapeutics | "Updated Data from the Phase 1 Trial of RTX-240 in Patients with Advanced Solid Tumors...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 non-small cell lung cancer (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 An 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."

P1 data • Anal Carcinoma • Gastrointestinal Cancer • Genito-urinary Cancer • Kidney Cancer • Lung Cancer • Melanoma • Non Small Cell Lung Cancer • Oncology • Renal Cell Carcinoma • Skin Cancer • Solid Tumor • Uveal Melanoma

Rubius Therapeutics Reports Updated Clinical Data at AACR from the Ongoing Monotherapy Phase 1 Arm of the Phase 1/2 Clinical Trial of RTX-240 in Advanced Solid Tumors Demonstrating Single-Agent Activity and Favorable Tolerability (GlobeNewswire) - P1/2 | N=234 | NCT04372706 | Sponsor: Rubius Therapeutics | "Final Phase 1 Clinical Results in Relapsed/Refractory AML...No DLTs were observed and there were 3 treatment-related Grade 3/4 adverse events. There were no investigator-reported immune-related AEs. Five patients had SD greater than 3 months, and 1 patient had a significant blast count reduction (53% to 6%)"

P1 data • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology

Rubius Therapeutics to Host Investor Webcast to Discuss Updated Clinical Data at AACR from the Ongoing Monotherapy Phase 1 Arm of the Phase 1/2 Clinical Trial of RTX-240 in Patients with Advanced Solid Tumors on April 8, 2022, at 1:15 PM ET (GlobeNewswire) - "Rubius Therapeutics...announced the details of its webcast to discuss updated clinical data from the ongoing monotherapy Phase 1 arm of the Phase 1/2 clinical trial of RTX-240 in patients with advanced solid tumors and relapsed/refractory acute myeloid leukemia on April 8, 2022, at 1:15 p.m. ET."

P1 data • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • Solid Tumor

Rubius Therapeutics to Present Updated Results from Single-Agent Phase 1 Clinical Trial of RTX-240 at the American Association of Cancer Research Annual Meeting (GlobeNewswire) - "Rubius Therapeutics, Inc...announced that the Company will present updated results from its single-agent Phase 1 clinical trial of RTX-240 in advanced solid tumors in a poster presentation at the American Association for Cancer Research (AACR) Annual Meeting being held from April 8-13, 2022 at the Ernest N. Morial Convention Center in New Orleans, Louisiana. The Company also plans to host a webcast to discuss the results from the single-agent Phase 1 trial of RTX-240 in solid tumors and AML on April 8, 2022, at 1:15 p.m. ET, following the release of the AACR abstracts....'We also intend to share our expected next steps for Phase 2 clinical development.'"

P1 data • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • Solid Tumor

Rubius Therapeutics Reports Fourth Quarter and Full Year 2021 Financial Results and Provides Business Update (GlobeNewswire) - "Increased cells produced per batch by four times in 50L bioreactors from 2020 to 2021, enabling uninterrupted clinical supply for three Phase 1 arms of the RTX-240 clinical trial and Phase 1 RTX-321 trial; Introduced frozen drug substance for RTX-321 and RTX-224, potentially enabling inventory storage of greater than two years...In the fourth quarter of 2021, Rubius invested $39.8 million in research and development...This year-over-year increase was principally due to a $11.0 million increase in costs incurred for the Company’s lead cancer programs, RTX-240 and RTX-321, primarily from clinical research organization (CRO) and internal manufacturing costs incurred in connection with the three arms of its Phase 1/2 clinical trial of RTX-240, for its Phase 1 clinical trial of RTX-321 for the treatment of HPV16-positive cancers and for start-up costs related to its Phase 1 clinical trial of RTX-224."

Commercial • Acute Myelogenous Leukemia • Anal Carcinoma • Bladder Cancer • Breast Cancer • Cervical Cancer • Cutaneous Melanoma • Gastrointestinal Cancer • Genito-urinary Cancer • Gynecologic Cancers • Head and Neck Cancer • Hematological Malignancies • Leukemia • Lung Cancer • Melanoma • Oncology • Solid Tumor • Squamous Cell Carcinoma of Head and Neck • Triple Negative Breast Cancer • Urothelial Cancer

Rubius Therapeutics Recaps 2021 Achievements and Outlines 2022 Objectives Across the RED PLATFORM at the 40th Annual J.P. Morgan Healthcare Conference (GlobeNewswire) - "Anticipated 2022 Catalysts and Operational Objectives...Present additional clinical results from the Phase 1 arm of the RTX-240 Phase 1/2 clinical trial in advanced solid tumors and the Phase 1 arm in relapsed/refractory acute myeloid leukemia (AML) during the first quarter of 2022; Initiate RTX-240 Phase 2 expansion cohorts in select solid tumor types during the first quarter of 2022; Report initial clinical results from the Phase 1 clinical trial of RTX-321 in patients with advanced HPV 16-positive cancers during the second half of 2022; Present initial clinical data from the Phase 1 arm of the RTX-240 clinical trial in combination with pembrolizumab in patients with advanced solid tumors during the second half of 2022"

P1 data • Trial status • Acute Myelogenous Leukemia • Cervical Cancer • Gynecologic Cancers • Hematological Malignancies • Leukemia • Oncology • Solid Tumor

Rubius Therapeutics to Highlight the Power of its RED PLATFORM, Significant Advances Across Red Cell Therapeutic Oncology Pipeline and New Type 1 Diabetes Program at Platform and Pipeline Day (GlobeNewswire) - “RTX-240: Additional clinical results are expected from this trial and the Phase 1 arm in relapsed/refractory AML during the first quarter of 2022. The Company plans to initiate single-agent RTX-240 Phase 2 expansion cohorts in select solid tumor types during the first quarter of 2022….RTX-224: Planning to initiate the Phase 1 clinical trial of RTX-224 in patients with certain advanced solid tumors during the first quarter of 2022….RTX-321: Continuing enrollment in Phase 1 clinical trial of RTX-321 in patients with advanced HPV 16-positive cancers. Planning to report initial clinical results during the first quarter of 2022”

New P1 trial • New P2 trial • P1 data • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • Solid Tumor

Rubius Therapeutics Reports Third Quarter 2021 Financial Results and Provides Business Update (GlobeNewswire) - "Near-Term Catalysts and Operational Objectives: (i) 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; (ii) 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 (iii) Report initial Phase 1 clinical data from RTX-321 for the treatment of HPV 16-positive cancers by the first quarter of 2022."

P1 data • Trial status • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • Solid Tumor

[VIRTUAL] A phase 1 trial of RTX-240, an allogeneic engineered red blood cell with cell -surface expression of 4-1BBL and trans-presented IL-15, in patients with advanced solid tumors (AACR 2021) - P1/2 | "RTX-240 is tolerable and leads to activation, expansion and trafficking of memory CD8+ T cells and NK cells, with preliminary evidence of anti-tumor activity. Exploration of the dose and schedule are ongoing in this study (NCT04372706).1"

Clinical • IO biomarker • P1 data • Anal Carcinoma • Colorectal Cancer • Gastrointestinal Cancer • Melanoma • Oncology • Solid Tumor • CD8 • IL15

Rubius Therapeutics Reports Second Quarter 2021 Financial Results and Provides Business Update (GlobeNewswire) - “Single-Agent RTX-240 Phase 1/2 Clinical Trial for Advanced Solid Tumors…The Company currently plans to present comprehensive results from this study at the end of 2021 or during the first quarter of 2022…Phase 1 Arm in Ongoing Phase 1/2 RTX-240 Clinical Trial in Relapsed/Refractory (R/R) Acute Myeloid Leukemia…Dosing has been completed in the first four dose cohorts and enrollment in an additional dose cohort is expected to begin in the third quarter of 2021.”

P1/2 data • Trial status • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • Solid Tumor

Rubius Therapeutics Announces Publication of RTX-240 Preclinical Data in Cancer Immunology, Immunotherapy (GlobeNewswire) - "Rubius Therapeutics...announced the publication of preclinical data in the peer-reviewed journal Cancer Immunology, Immunotherapy, for its lead clinical oncology program, RTX-240, for the treatment of adults with advanced solid tumors and relapsed/refractory acute myeloid leukemia....'These preclinical data supported advancing RTX-240 into the clinic, where the initial clinical results have shown that RTX-240 has the ability to activate and expand a patient’s own immune system to fight cancer, leading to clinical responses in certain patients with advanced solid tumors,' said Christina Coughlin, M.D., Ph.D., chief medical officer of Rubius Therapeutics."

Preclinical • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • Solid Tumor

Anti-tumor effects of RTX-240: an engineered red blood cell expressing 4-1BB ligand and interleukin-15. (PubMed, Cancer Immunol Immunother) - "RTX-240 promotes T cell and NK cell activity in preclinical models and shows efficacy and an improved safety profile. Based on these data, RTX-240 is now being evaluated in a clinical trial."

Journal • Hepatology • Immune Modulation • Inflammation • Liver Failure • Melanoma • Oncology • Solid Tumor • CD8 • IL15

Rubius Therapeutics Announces First Patient Dosed with RTX-240 in Combination with KEYTRUDA (pembrolizumab) in Ongoing Phase 1/2 Clinical Trial for the Treatment of Advanced Solid Tumors (GlobeNewswire) - "Rubius Therapeutics...announced that the first patient has been dosed in a new Phase 1 arm of the ongoing Phase 1/2 clinical trial of RTX-240 in combination with KEYTRUDA® (pembrolizumab)1 for the treatment of patients with relapsed/refractory or locally 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."

Trial status • Oncology • Solid Tumor

Rubius Therapeutics Reports First Quarter 2021 Financial Results and Provides Business Update (GlobeNewswire) - "Anticipated 2021 Catalysts and Operational Objectives: Present additional clinical data from the RTX-240 solid tumor Phase 1 clinical trial; Select specific solid tumor types that will be pursued in the Phase 2 expansion cohort of RTX-240; Report initial clinical data from the Phase 1 arm of the RTX-240 clinical trial in relapsed/refractory AML; Initiate the Phase 1 clinical trial of RTX-240 in combination with anti-PD-1 therapy in advanced solid tumors in the second half of 2021; Report initial Phase 1 clinical data from RTX-321 for the treatment of HPV 16-positive cancers by the first quarter of 2022; Submit an Investigational New Drug Application for RTX-224 by year-end."

New P1 trial • P1 data • Trial status • Acute Myelogenous Leukemia • Hematological Malignancies • Oncology • Solid Tumor

Rubius Therapeutics Presents Initial Clinical Results from the Ongoing Phase 1/2 Clinical Trial of RTX-240 in Advanced Solid Tumors at the American Association of Cancer Research Virtual Annual Meeting (GlobeNewswire) - "Rubius Therapeutics, Inc...reprised initial clinical results from its ongoing Phase 1/2 study of RTX-240 in advanced solid tumors at the American Association for Cancer Research (AACR) Virtual Annual Meeting. Posters will be available online tomorrow on the AACR website and Publications page of the Rubius Therapeutics website at 8:30 a.m. EDT...'The clinical data shared to date demonstrate that RTX-240 has the ability to activate and expand a patient's own immune system to fight cancer, leading to clinical responses in certain patients with advanced solid tumors,' said Omid Hamid, M.D...'Additionally, by year end, we expect to select the specific solid tumor types that we plan to pursue in the Phase 2 expansion cohorts of RTX-240. We also intend to initiate a new Phase 1 arm to evaluate RTX-240 in combination with anti-PD-1 therapy in advanced solid tumors during the second half of 2021.'"

Media quote • New P1 trial • P1/2 data • Oncology • Solid Tumor

Rubius Therapeutics Reports Initial Clinical Data from Ongoing Phase 1/2 Trial of RTX-240 in Patients with Advanced Solid Tumors, Demonstrating Single-Agent Activity (GlobeNewswire) - P1/2, N=216; NCT04372706; Sponsor: Rubius Therapeutics; “The Company also shared tumor trafficking data from one patient with relapsed/refractory acute myeloid leukemia (AML) in the second Phase 1 arm of the study...Immune cell trafficking into the tumor microenvironment (TME) was assessed by tumor biopsies from participating patients with solid tumors (optional; n=4) and AML (standard of care; n=1). Trafficking of T and NK cells into the TME was observed in 3/5 patients (1.6 to 10-fold increases), including one patient each with metastatic mesothelioma, metastatic soft tissue sarcoma and refractory AML. Tumor infiltration was not observed in patients with ovarian cancer (n=1) and heavily pretreated melanoma (n=1)…In one patient with AML, trafficking of T and NK cells into the bone marrow was associated with increases in the cellularity of the marrow.”

P1 data • Acute Myelogenous Leukemia • Hematological Malignancies • Oncology

Rubius Therapeutics Reports Initial Clinical Data from Ongoing Phase 1/2 Trial of RTX-240 in Patients with Advanced Solid Tumors, Demonstrating Single-Agent Activity (GlobeNewswire) - “Upcoming Anticipated Milestones - In order to realize the full potential of RTX-240, the Company’s other oncology programs and the RED PLATFORM, in the next 12 months, Rubius plans to execute several critical milestones: Present additional clinical results from the RTX-240 solid tumor Phase 1 clinical trial; Select the recommended RTX-240 Phase 2 dose, schedule and specific solid tumor types that will be pursued in the Phase 2 expansion cohort; Report initial clinical results for the second Phase 1 arm of the RTX-240 clinical trial in relapsed/refractory AML; Initiate the Phase 1 clinical trial of RTX-240 in combination with anti-PD-1 therapy in advanced solid tumors in 2H’21; Report initial Phase 1 clinical results for RTX-321 for the treatment of HPV 16-positive cancers by 1Q’22; and Submit an Investigational New Drug Application for RTX-224 by year-end.”

IND • P1 data • Trial status • Acute Myelogenous Leukemia • Hematological Malignancies • Oncology • Solid Tumor