PRGN-3006 / Precigen - LARVOL DELTA

Precigen Reports Third Quarter 2024 Financial Results and Business Updates (PRNewswire) - "Key Program Highlights:...(i) PRGN-2009 AdenoVerse Gene Therapy in HPV-associated cancers: PRGN-2009 Phase 2 clinical trials under a cooperative research and development agreement (CRADA) with the National Cancer Institute (NCI) in recurrent/metastatic cervical cancer and in newly diagnosed HPV-associated oropharyngeal cancer are ongoing...; (ii) PRGN-3006 UltraCAR-T in AML and MDS: The Company has completed enrollment of the Phase 1b trial for PRGN-3006 in acute myeloid leukemia (AML)...and is preparing for an end of Phase 1b meeting with the FDA to discuss next steps."

FDA event • Trial status • Acute Myelogenous Leukemia • Cervical Cancer • Myelodysplastic Syndrome • Oropharyngeal Cancer

Precigen Strategically Prioritizes Portfolio to Focus on First Potential Gene Therapy Launch (PRNewswire) - "PRGN-2009 AdenoVerse Gene Therapy Clinical Trials: The Company plans to continue PRGN-2009 Phase 2 trials under a cooperative research and development agreement (CRADA) with the National Cancer Institute (NCI) in recurrent/metastatic cervical cancer and in newly diagnosed HPV-associated oropharyngeal cancer. PRGN-2009 clinical trial enrollment at non-NCI clinical sites will be paused. UltraCAR-T Clinical Programs: The Company has completed enrollment of the Phase 1b trial for PRGN-3006 in acute myeloid leukemia (AML), which received Fast Track designation from the US Food and Drug Administration (FDA), and is preparing for an end of Phase 1b meeting with the FDA to discuss next steps. The Company will pause all other UltraCAR-T clinical programs, including PRGN-3005 and PRGN-3007."

Discontinued • Pipeline update • Cervical Cancer • Gynecologic Cancers • Head and Neck Cancer • Oncology • Oropharyngeal Cancer • Solid Tumor • Squamous Cell Carcinoma of Head and Neck

Precigen Reports Second Quarter and First Half 2024 Financial Results and Business Updates (PRNewswire) - "Key Program Highlights:...(i) PRGN-2009 AdenoVerse Gene Therapy Clinical Trials: The Company plans to continue PRGN-2009 Phase 2 clinical trials under a cooperative research and development agreement (CRADA) with the...NCI in recurrent/metastatic cervical cancer and in newly diagnosed HPV-associated oropharyngeal cancer. PRGN-2009 cervical cancer clinical trial enrollment at non-NCI clinical sites will be paused; (ii) UltraCAR-T Clinical Programs: The Company has completed enrollment of the Phase 1b trial for PRGN-3006 in acute myeloid leukemia (AML)...and is preparing for an end of Phase 1b meeting with the FDA to discuss next steps. The Company will pause the PRGN-3005 and PRGN-3007 clinical trials. The Company will minimize UltraCAR-T spend and focus on strategic partnerships to further advance UltraCAR-T programs."

FDA event • Trial status • Acute Myelogenous Leukemia • Cervical Cancer • Fallopian Tube Cancer • Oropharyngeal Cancer • Ovarian Cancer • Peritoneal Cancer

PRGN-3006 Adoptive Cellular Therapy for CD33-Positive Relapsed or Refractory AML, MRD Positive AML or Higher Risk MDS (clinicaltrials.gov) - P1 | N=88 | Active, not recruiting | Sponsor: Precigen, Inc | Recruiting ➔ Active, not recruiting

Enrollment closed • Minimal residual disease • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology

Precigen Reports Full Year 2023 Financial Results and Business Updates - "PRGN-3006 in AML/MDS: An interim Phase 1b dose expansion data presentation is anticipated in the second half of 2024; PRGN-3007 in Advanced ROR1+ Hematological and Solid Tumors: A preliminary Phase 1 dose escalation data presentation is anticipated by the end of 2024."

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

Precigen Highlights Pipeline Updates to be Presented at the 42nd Annual J.P. Morgan Healthcare Conference (PRNewswire) - P1 | N=88 | NCT03927261 | Sponsor: Precigen, Inc | "Phase 1 dose escalation data showed that PRGN-3006 was well-tolerated with no dose-limiting toxicities (DLTs) and a 27% objective response rate (ORR) in heavily pre-treated r/r AML patients infused following lymphodepletion; An interim Phase 1b dose expansion data presentation is anticipated in the second half of 2024."

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

PRGN-3006 Adoptive Cellular Therapy for CD33-Positive Relapsed or Refractory AML, MRD Positive AML or Higher Risk MDS (clinicaltrials.gov) - P1 | N=88 | Recruiting | Sponsor: Precigen, Inc | Trial completion date: Jan 2024 ➔ Aug 2025 | Trial primary completion date: Jan 2023 ➔ Aug 2024

Minimal residual disease • Trial completion date • Trial primary completion date • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology • CD4 • CD8

Precigen Reports First Quarter 2023 Financial Results and Business Updates (PRNewswire) - "PRGN 2009 AdenoVerse™ Immunotherapy in HPV-associated Cancers...Enrollment was completed in the Phase 2 monotherapy arm with 20 evaluable patients in newly diagnosed oropharyngeal squamous cell carcinoma (OPSCC) patients. An interim clinical data presentation from the Phase 2 monotherapy arm is expected in the second half of 2023. PRGN-3006 UltraCAR-T^® in AML...A Phase 1b clinical data presentation is expected in 2024."

Enrollment closed • P1 data • P2 data • Acute Myelogenous Leukemia • Head and Neck Cancer • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology • Oropharyngeal Cancer • Solid Tumor • Squamous Cell Carcinoma • Squamous Cell Carcinoma of Head and Neck

Precigen Regains Exclusive Rights to Proven CAR-T Targets, CD19 and BCMA, to Enable Unencumbered Development and Commercialization of UltraCAR-T (PRNewswire) - "Precigen...announced the amendment of its exclusive license agreement with Alaunos Therapeutics, Inc. (Alaunos). With this amendment, Precigen has the unique ability to utilize the clinically validated UltraCAR-T platform for unencumbered development and commercialization of two proven CAR-T targets, CD19 and B-cell maturation antigen (BCMA)."

Licensing / partnership • Acute Myelogenous Leukemia • Fallopian Tube Cancer • Gynecologic Cancers • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology • Ovarian Cancer • Peritoneal Cancer • Solid Tumor

Preclinical Characterization of Prgn-3006 Ultracar-T™ for the Treatment of AML and MDS: Non-Viral, Multigenic Autologous CAR-T Cells Administered One Day after Gene Transfer (ASH 2019) - P1; "MOLM-13 tumor cells were engrafted into NSG mice on Day 0 and PRGN-3006 were administered on Day 6 (arrow). Data shown is tumor burden (expressed as total flux in photons/sec) as the mean±SEM for each group of mice shown, with n=6-7 mice mice/group at the start of the study."

CAR T-Cell Therapy • IO Biomarker • Preclinical • IFNG • IL15

[VIRTUAL] A Phase 1/1b Safety Study of Prgn-3006 Ultracar-T™ in Patients with Relapsed or Refractory CD33-Positive Acute Myeloid Leukemia and Higher Risk Myelodysplastic Syndrome (ASH 2020) - P1 | "Superior efficacy of UltraCAR-T cells was demonstrated in an aggressive murine xenograft model of AML where a single administration of PRGN-3006, only one day after gene transfer, showed significantly higher expansion and persistence; effectively eliminated tumor burden; and significantly improved overall survival compared to traditional CD33 CAR-T cells lacking mbIL15 expression (Blood (2019) 134(S1): 2660)...Key inclusion criteria include an absolute lymphocyte count ≥ 0.2k/µL, KPS > 60%, absence of other active malignancy within 1 year of study entry, daily corticosteroid dose < 10mg of prednisone daily, adequate organ function and a backup allogeneic donor should bone marrow aplasia occur. Hydroxyurea is allowed for cytoreduction with cessation 3 days prior to apheresis/infusion but can be reinitiated post-infusion. To test the hypothesis that expression of mbIL15 on PRGN-3006 cells is sufficient to promote CAR-T cell expansion and persistence, study..."

Clinical • IO biomarker • P1 data • Acute Myelogenous Leukemia • Aplastic Anemia • Chronic Myelomonocytic Leukemia • Gene Therapies • Graft versus Host Disease • Hematological Malignancies • Immunology • Leukemia • Myelodysplastic Syndrome • Oncology • Transplantation • CD33 • IL15

Phase 1/1b Safety Study of Prgn-3006 Ultracar-T in Patients with Relapsed or Refractory CD33-Positive Acute Myeloid Leukemia and Higher Risk Myelodysplastic Syndromes (ASH 2022) - P1 | "Pts received PRGN-3006 infusion without (Cohort 1, C1) or with lymphodepletion (fludarabine 30mg/m2 and cyclophosphamide 500mg/m2 days -5 to -3; Cohort 2, C2)...Only 1 pt (DL1, C1) had transient grade 3 CRS that resolved in < 24 hours with tocilizumab and dexamethasone... Administration of PRGN-3006 UltraCAR-T cells targeting CD33, without or with lymphodepletion, have been well tolerated with low grade CRS. PRGN-3006 UltraCAR-T expressing mbIL15 demonstrated a dose-dependent robust expansion and durable persistence in blood and bone marrow with or without lymphodepletion. Encouraging objective responses have been observed in AML patients treated with lymphodepletion."

Clinical • P1 data • Acute Myelogenous Leukemia • Aplastic Anemia • Chronic Myelomonocytic Leukemia • Gene Therapies • Hematological Malignancies • Inflammation • Leukemia • Myelodysplastic Syndrome • Oncology • Transplantation • CD33 • IL15

Precigen Reports Fourth Quarter and Full Year 2022 Financial Results and Business Updates (PRNewswire) - "PRGN-3006 UltraCAR-T® in AML...The Company received FDA clearance to incorporate repeat dosing in the Phase 1b expansion trial. A Phase 1b clinical trial data presentation is expected in 2024....PRGN-3005 UltraCAR-T® in Ovarian Cancer: The Company completed enrollment in the Phase 1 dose escalation cohorts of the intraperitoneal (IP) and intravenous (IV) arms without lymphodepletion as well as in the lymphodepletion cohort in the IV arm. A Phase 1 dose escalation data presentation is expected in the first half of 2023. The Company initiated a Phase 1b dose expansion trial of PRGN-3005. The Company received FDA clearance to incorporate repeat dosing in the trial. A Phase 1b clinical trial data presentation is expected in 2024."

P1 data • Acute Myelogenous Leukemia • Gynecologic Cancers • Hematological Malignancies • Oncology • Ovarian Cancer

Precigen Provides Pipeline and Corporate Updates at the 41st Annual J.P. Morgan Healthcare Conference (PRNewswire) - "PRGN-3006 UltraCAR-T® in Acute Myeloid Leukemia (AML)...Program Status: Enrollment was completed in the Phase 1 dose escalation cohorts of the study....The Phase 1b dose expansion study of PRGN-3006 UltraCAR-T was expanded to Mayo Clinic in Rochester, Minnesota, enhancing the decentralized manufacturing model. The first patient was successfully dosed at the expansion site with PRGN-3006 UltraCAR-T. Site activation activities are in progress at several additional major cancer centers across the US as part of the multicenter expansion of the study....Phase 1b clinical trial data presentation is expected in 2024."

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

Precigen Announces Positive Phase 1 Dose Escalation Data for Autologous PRGN-3006 UltraCAR-T Manufactured Overnight for Next Day Infusion in Relapsed or Refractory Acute Myeloid Leukemia Patients (PRNewswire) - P1 | N=88 | NCT03927261 | Sponsor: PGEN Therapeutics | "Precigen...presented positive Phase 1 dose escalation data from the ongoing Phase 1/1b clinical study of PRGN-3006 UltraCAR-T®...at the 64th American Society of Hematology (ASH) Annual Meeting....The study enrolled a total of 26 patients (N=10 non-lymphodepletion; N=16 with lymphodepletion) and included 21 patients with r/r AML, 2 patient with chronic myelomonocytic leukemia (CMML), and 3 patients with MDS....In the non-lymphodepletion cohort, 3 out of 10 patients had Stable Disease (SD), per European LeukemiaNet (ELN) criteria, persisting for more than 3 months with one patient experiencing durable SD for more than 7 months with concomitant reduction in peripheral blast levels....An objective response rate (ORR) of 27% (3 out of 11) was reported for heavily pre-treated r/r AML patients with poor prognosis (median prior treatments: 4; range: 1-9) in the lymphodepletion cohort."

P1 data • Acute Myelogenous Leukemia • Chronic Myelomonocytic Leukemia • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology

Phase 1/1b Safety Study of Prgn-3006 Ultracar-T in Patients with Relapsed or Refractory CD33-Positive Acute Myeloid Leukemia and Higher Risk Myelodysplastic Syndromes (ASH 2021) - P1 | "Ps receive PRGN-3006 infusion without (Cohort 1) or with lymphodepletion (fludarabine 30mg/m 2 and cyclophosphamide 500mg/m 2 days -5 to -3; Cohort 2)...Pts were heavily pre-treated with a median of 3 prior regimens (1-7), with 93% and 80% of pts being r/r to a HMA + venetoclax or intensive chemotherapy, respectively...Cytokine release syndrome (CRS) occurred in 47% of pts (n=7; G1 in 5 pts) with only 1 transient grade 3 event (DL 1, Cohort 1) that resolved in < 24 hours with tocilizumab and dexamethasone...In the setting of mbIL15, there has been a dose-dependent robust expansion and durable persistence of PRGN-3006 with encouraging responses (50%) in patients treated following lymphodepletion. Enrollment is ongoing to DL4, and updated safety, efficacy, PK/PD and cytokine data to be presented."

Clinical • P1 data • Acute Myelogenous Leukemia • Aplastic Anemia • Bone Marrow Transplantation • Chronic Myelomonocytic Leukemia • Graft versus Host Disease • Hematological Malignancies • Immunology • Inflammation • Leukemia • Myelodysplastic Syndrome • Oncology • Transplantation • CD33 • IL15 • IL6 • TNFA

Precigen Reports Second Quarter and First Half 2022 Financial Results (PRNewswire) - "PRGN-3006 UltraCAR-T in AML: The Company received FDA clearance to incorporate repeat dosing in the Phase 1b expansion phase of the study and plans to initiate in 2022. Additional data for the Phase 1/1b study is expected at a major scientific conference in Q4 2022....PRGN-3005 UltraCAR-T in Ovarian Cancer: Patient follow up is ongoing and the Company expects Phase 1 data to be presented in the first half of 2023."

Enrollment status • P1 data • Acute Myelogenous Leukemia • Gynecologic Cancers • Hematological Malignancies • Oncology • Ovarian Cancer

PRGN-3006 Adoptive Cellular Therapy for Relapsed or Refractory AML or Higher Risk MDS (clinicaltrials.gov) - P1 | N=88 | Recruiting | Sponsor: PGEN Therapeutics, Inc., a subsidiary of Precigen, Inc. | N=56 ➔ 88 | Trial completion date: May 2025 ➔ Jan 2024 | Trial primary completion date: May 2022 ➔ Jan 2023

Enrollment change • Trial completion date • Trial primary completion date • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology • CD4 • CD8

Precigen Receives Fast Track Designation for PRGN-3006 UltraCAR-T in Patients with Relapsed or Refractory Acute Myeloid Leukemia (PRNewswire) - "Precigen, Inc...announced that the FDA has granted Fast Track designation for PRGN-3006 UltraCAR-T® in patients with relapsed or refractory (r/r) AML."

Fast track designation • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology

Precigen Provides Pipeline and Corporate Updates at the 40th Annual J.P. Morgan Healthcare Conference (PRNewswire) - "Precigen announced enrollment completion for Dose Level 3 of the lymphodepletion cohort....The Company plans to initiate a multicenter expansion phase of the study at Dose Level 3 with lymphodepletion in the first half of 2022. The Company plans to incorporate a repeat dosing regimen in the expansion phase. Additional Phase 1/1b data is expected in 2022."

Clinical protocol • Enrollment status • P1 data • Trial status • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology

Dr. Sallman on the Responses to PRGN-3006 in Select AML and Higher-Risk MDS (OncLive) - "David Sallman, MD...discusses responses achieved in a first-in-human dose escalation/dose expansion phase 1/1b clinical trial (NCT03927261) of an UltraCAR-T agent, PRGN-3006, in patients with acute myeloid leukemia (AML) and higher-risk myelodysplastic syndrome with hypomethylating agent failure....One patient subsequently underwent a successful allogeneic stem cell transplant (ASCT) and experienced a complete response (CR) with hematologic recovery for over 1 year, Sallman explains. Another patient with extramedullary AML achieved a partial response at day 28, Sallman adds."

Media quote • Video

Phase 1/1b safety study of Prgn-3006 Ulrtracar T in patients with r/R CD33 AML and higher Risk MDS (VJHemOnc)

Live event

Precigen Announces Positive Interim Phase 1 Data for PRGN-3006 UltraCAR-T in Relapsed or Refractory Acute Myeloid Leukemia (PRNewswire) - P1, N=56; NCT03927261; Sponsor: PGEN Therapeutics, Inc.; "The interim data for PRGN-3006 showed excellent, dose-dependent expansion and persistence of PRGN-3006 in peripheral blood and bone marrow following a single infusion, with detection of UltraCAR-T cells in blood more than 3 months post-infusion in the non-lymphodepletion and lymphodepletion cohorts,' said David A. Sallman, MD...'An ORR of 50% in patients treated at the two lowest dose levels in the lymphodepletion cohort is highly encouraging and the specifics of the responding patients suggest the potential for PRGN-3006 as a bridge to allo-HSCT, which is a very important potential treatment pathway for these patients."

Media quote • P1 data • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology

Precigen Announces Positive Interim Phase 1 Data for PRGN-3006 UltraCAR-T in Relapsed or Refractory Acute Myeloid Leukemia (PRNewswire) - P1, N=56; NCT03927261; Sponsor: PGEN Therapeutics, Inc.; "Precigen...presented positive interim data at the 63rd ASH Annual Meeting and Exposition (Abstract# 825) from the ongoing Phase 1/1b clinical study of PRGN-3006 UltraCAR-T....Objective response rate (ORR) of 50% (3 out of 6; 1 Partial Response (PR); 2 Complete Responses (CR)) for the lymphodepletion cohort at the two lowest dose levels; One responder in the lymphodepletion cohort subsequently bridged to allo-HSCT with ongoing survival greater than 1 year; Stable Disease (SD) for more than three months in 33% (3 out of 9) patients in the non-lymphodepletion cohort."

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

"Dr Sallman is excited about PRGN-3006. Can you please follow up $PGEN" (@Biotech2050)