Umoja Biopharma Presents Data on its iPSC-Based Cell Therapy Platform at the American Association for Cancer Research (AACR) Annual Meeting 2022

On April 8, 2022 Umoja Biopharma, Inc., an oncology company leveraging its proprietary integrated technologies to create next-generation off-the-shelf in vivo(VivoVec) and induced pluripotent stem cell (iPSC) based immunotherapies for the treatment of solid tumors and hematologic malignancies, reported the presentation of data supporting the use of a synthetic cytokine receptor, the Rapamycin Activated Cytokine Receptor (RACR), to derive synthetic innate lymphoid cells, RACR-induced cytotoxic innate lymphoid cells (RACR-iCILs) (Press release, Umoja Biopharma, APR 8, 2022, View Source [SID1234611702]). The data will be presented in a poster presentation at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2022, held from April 8-13, 2022.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"Some of the current limitations of cell therapies are the need for lymphodepletion, and repetitive lymphodepletion at that, in addition to the challenge of in vivo expansion," said Andy Scharenberg, M.D., co-founder and Chief Executive Officer of Umoja. "Our induced pluripotent stem cell platform seeks to address many of the limitations holding back cell therapy as we know it. By using a synthetic cytokine receptor that can mimic the downstream signaling of the common gamma chain cytokines essential to lymphoid cell differentiation, we are able to create a renewable starting material for scalable manufacturing of synthetic allogeneic chimeric antigen receptor cell products."

On Sunday, April 10th, Samantha O’Hara, Ph.D., Principal Scientist at Umoja Biopharma, will present a poster (Abstract 547) titled "Generation of synthetic cytokine receptor-induced cytotoxic innate lymphocytes (iCILs) from iPSCs as off-the-shelf cancer therapeutics." The data highlights Umoja’s iPSC-based cell therapy platform in which stem cells are engineered ex vivo to express Umoja’s RACR system and a universal adapter-specific TagCAR. The RACR system is intended to be utilized during the manufacturing process where it has the potential to deliver controlled and consistent proliferation and differentiation signals, resulting in the production of a highly pure, induced cytotoxic innate lymphoid cell product. In addition, the RACR system could enable enhanced in vivo engraftment and persistence in the absence of lymphodepleting chemotherapy. The universal adapter-specific TagCAR is designed to enable simultaneous targeting of tumor, tumor stroma, and immunosuppressive cells in the presence of a cocktail of bispecific small molecule adapters, called TumorTags.

Nkarta Presents New Preclinical Data from Engineered NK Cell Platform at AACR Annual Meeting 2022

On April 8, 2022 Nkarta, Inc. (Nasdaq: NKTX), a biopharmaceutical company developing engineered natural killer (NK) cell therapies to treat cancer, reported the presentation of four preclinical data abstracts focused on its natural killer cell platform and pipeline at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2022 (Press release, Nkarta, APR 8, 2022, View Source [SID1234611701]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"The data we presented at this year’s AACR (Free AACR Whitepaper) meeting highlight the breadth and diversity of our scientific efforts, as we continue to expand our ability to deliver off-the-shelf cell therapies with the potential to disrupt the cancer treatment landscape," said James Trager, PhD, Chief Scientific Officer of Nkarta. "Our research activities are designed to extend the capabilities of NK cells, to lay the groundwork for further pipeline programs – including our pioneering NK+T cell program – and to implement cutting edge translational methods to support our ongoing clinical programs. Our findings reported at AACR (Free AACR Whitepaper) further support exploration of multiply edited CD70 CAR NK cells for clinical application, one focus of our ongoing collaboration with our partners at CRISPR Therapeutics."

Details of the preclinical poster presentations at AACR (Free AACR Whitepaper) follow. Posters are available for download on the Nkarta website (View Source) and on the AACR (Free AACR Whitepaper) e-poster website (View Source).

Presented jointly with CRISPR Therapeutics:

Title: CBLB, CISH and CD70 multiplexed gene knockout with CRISPR/Cas9 enhances cytotoxicity of CD70-CAR NK cells and provides greater resistance to TGF-β for cancer immunotherapy
Session Category:Immunology
Session Title:Preclinical Immunotherapy
Abstract Number:5512

This study illustrates gene editing approaches that enhance the ability of NK cells to target CD70, an antigen highly expressed in a variety of malignant settings, including renal cell carcinoma (RCC) and adenocarcinoma. Editing targets included cytokine inducible SH2-containing protein (CISH) and the E3 ubiquitin ligase CBLB, both negative regulators of NK cell function. Preclinical results showed that a combined editing and engineering strategy to armor primary NK cells via co-expression of the CD70 CAR and a membrane bound form of IL-15 (mbIL-15), together with a triple knockout of CISH, CBLB and CD70 genes using the CRISPR/Cas9 system enhanced anti-tumor activity against renal cell carcinoma (RCC) solid tumor cell lines and provide greater resistance to TGF-β mediated inhibition. These data support the further exploration of CD70/CISH/CBLB triple gene knockout CD70 CAR NK cells for clinical application.

Nkarta presentations:

Title: Surveying surface antigen expression in multiple myeloma preclinical models
Session Category: Tumor Biology
Session Title: Nonclinical Models of Cancer
Abstract Number: 6004

Multiple myeloma (MM) is a progressive hematological cancer with a 5-year survival rate of 53%. Novel therapeutic strategies are being developed to target specific MM surface antigens. Yet, changes in antigen expression through MM progression are poorly understood in the clinic and have not been well characterized in preclinical models. Data presented in this study highlighted antigen expression differences in MM cells when analyzed in mouse tissue compared to in vitro culture. Like the widely variable expression observed between patients, BCMA and CD138 were differentially expressed in the mouse bone marrow between MM models. Commonly targeted antigens in MM also vary kinetically in vivo and can be measured and tracked using flow cytometry. The present findings also support the use of specific cell lines when assessing BCMA, CD38 and CD138-specific immunotherapies or combinatorial approaches to MM treatment.

Title: Development of multiomics approaches to evaluate NKG2D ligand dynamics and anti-tumor immune responses during CAR-NK treatment
Session Category: Clinical Research Excluding Trials
Session Title:Immuno-oncology
Abstract Number:5187

NKX101 is an investigational NK cell therapy engineered to overexpress a chimeric receptor consisting of NKG2D ectodomain, costimulatory signaling motifs, and a membrane-bound form of IL-15. NKX101 is currently under clinical evaluation for treatment of relapsed/refractory acute myeloid leukemia (AML) and higher risk myelodysplastic syndrome (MDS). To better understand patterns of response to NKX101, we describe the development of several key translational methods, including (i) a single-cell (sc) RNAseq approach to assess gene expression pattern changes in NKX101 and patient cells, (ii) a multiplex IHC panel to monitor NKG2D-ligand expression by cancer cells, and (iii) an ELISA method to detect shed NKG2D-ligand in serum. Employing multiplex IHC and digital image analysis, we found that membrane bound NKG2D-ligands are upregulated in AML and HCC compared to age-matched normal tissue controls. Lastly, using in-house developed ELISAs, we determined that shed NKG2D-ligands can be successfully detected in serum isolated from patients with MDS. Taken together, these assays provide methods for evaluation of NKG2D-ligand dynamics as well as the detection and phenotypic analysis of CAR-NK and immune cell populations in clinical samples.

Title:Immune masking strategies to extend the pharmacokinetics of allogeneic cell therapies
Session Category: Immunology
Session Title:Preclinical Immunotherapy
Abstract Number:5511

Development of immune evasion strategies are underway to improve the pharmacokinetics of allogeneic cell therapies by engineering them to avoid host vs. graft disease, where allogeneic NK and T cells are rapidly targeted by the patient’s own immune system. Hypoimmune strategies are particularly important for the development of allogeneic products containing mixed NK and T cell populations to minimize product cell fratricide. A conventional method for preventing host T rejection of allogenic T cells, is to combine knockout (KO) β-2 microglobulin (β2M) to diminish expression of MHC class I proteins with overexpression of nonclassical MHC class I protein, HLA-E, to evade host NK cell rejection. This study evaluated the effectiveness of HLA-E and other molecules in β2M deficient T cells for inhibiting NK cell cytotoxicity. Concurrently, a high throughput platform was developed to screen NK inhibitory peptides and synthetic ligands to identify novel immune masking strategies for extending allogeneic cell therapy persistence for broad patient populations.

The study showed that the benefit of HLA-E expression in suppressing NK cell cytotoxicity is highly correlated with the expression of NKG2A on the host NK cells. Viral peptides were potent suppressors of NK cell activity, and less dependent on donor NKG2A expression. These data support further exploration of different immune masking strategies in order to extend the pharmacokinetics of allogeneic cell therapies.

Seagen to Highlight Preclinical Data for Enfortumab Vedotin and Two Novel Antibody-Drug Conjugates at AACR Annual Meeting

On April 8, 2022 Seagen Inc. (Nasdaq: SGEN) reported data from intravesical instillation of enfortumab vedotin (EV) in a non-muscle invasive bladder cancer (NMIBC) preclinical model in addition to preclinical data from SGN-ALPV and SGN-B7H4V, two of its novel antibody-drug conjugates (ADCs) that utilize the company’s proprietary vedotin drug linker technology (Press release, Seagen, APR 8, 2022, View Source [SID1234611700]). These data will be presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting, taking place in New Orleans, April 8-13, 2022. Seagen and Astellas Pharma Inc. are co-developing enfortumab vedotin under a 50:50 worldwide development and commercialization collaboration.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"We are encouraged by the preclinical intravesical EV data showing limited systemic exposure and antitumor activity with an encouraging safety profile. These data served as the basis for initiating the ongoing phase 1 trial in patients with NMIBC," said Scott Peterson, Ph.D., Senior Vice President of Research at Seagen. "Additionally, preclinical data presented from SGN-ALPV suggest it may have applications across several tumor types and adds to our broad pipeline of novel ADCs."

Highlights of Seagen Programs Presented at AACR (Free AACR Whitepaper)

Enfortumab Vedotin

Enfortumab vedotin demonstrated antitumor activity in preclinical models of non-muscle invasive bladder cancer (NMIBC). Enfortumab vedotin is directed to Nectin-4, which is highly prevalent in NMIBC. Studies with human bladder cancer cells expressing Nectin-4 showed sustained activity under conditions mimicking intravesical dosing and achieved tumor growth inhibition of 97% when administered in an orthotopic xenograft animal model of NMIBC. Intravesical administration of enfortumab vedotin was well tolerated in a good laboratory practice (GLP) study with minimal local and no systemic toxicities at doses up to six-fold the intravenous maximum tolerated dose.

The antitumor activity and safety profile seen in these preclinical studies support further investigation of intravesical enfortumab vedotin in this patient population, which is now enrolling in a phase 1 trial, EV-104.

SGN-ALPV

SGN-ALPV is a novel ADC designed to target two proteins, ALPP and ALPPL2, to maximize drug delivery. ALPP and ALPPL2 are aberrantly co-expressed in a broad set of solid tumors, including ovarian, endometrial and germ cell cancers. In preclinical studies, SGN-ALPV exhibited robust antitumor activity in cell line and patient-derived xenograft cancer models with both homogenous and heterogeneous expression of placental alkaline phosphatases ALPP and ALPPL2, consistent with robust monomethyl auristatin E (MMAE)-directed cytotoxicity and bystander activity of vedotin ADCs.

Importantly, ALPP and ALPPL2 exhibit a highly restricted normal tissue expression, which may enable a favorable safety profile. Differential expression of ALPP and ALPPL2 in the tumor versus normal tissue, antibody specificity, antitumor activity and tolerability of SGN-ALPV provided a strong rationale for the initiation of the first-in-human phase 1 clinical study currently enrolling patients.

Details of Seagen Presentations at AACR (Free AACR Whitepaper) Annual Meeting 2022:

Abstract Title

Abstract #

Presentation

Presenter

Enfortumab vedotin, a Nectin-4 directed ADC, demonstrates compelling tolerability and anti-tumor activity with intravesical instillation in preclinical models of non-muscle invasive bladder cancer

#1140

Poster session: PO.ET05.02-Preclinical and Clinical Pharmacology, Section 25

April 11

9:00 a.m. – 12:30 p.m. ET

C. Carosino

SGN-B7H4V shows immunomodulatory activity through induction of immunogenic cell death

#1281

Poster session:

PO.CL06.04-Immune Mechanisms Invoked by Other Therapies, Section 32

April 11
9:00 a.m. – 12:30 p.m. ET

E. Gray

SGN-ALPV a novel, investigational vedotin ADC demonstrates highly effective targeting of oncofetal phosphatases ALPP and ALPPL2 in preclinical models

#1766

Poster Session: PO.ET01.04- Antibody-Drug Conjugates, Section 21

April 11

1:30 p.m. – 5:00 p.m. ET

S. Anderson

About Enfortumab Vedotin

Enfortumab vedotin-ejfv (PADCEV) is an antibody-drug conjugate (ADC) that is directed against Nectin-4, a protein located on the surface of cells and highly expressed in bladder cancer.i,ii Nonclinical data suggest the anticancer activity of enfortumab vedotin is due to its binding to Nectin-4 expressing cells followed by the internalization and release of the anti-tumor agent monomethyl auristatin E (MMAE) into the cell, which result in the cell not reproducing (cell cycle arrest) and in programmed cell death (apoptosis).

About SGN-ALPV

SGN-ALPV is a novel, investigational vedotin antibody drug conjugate (ADC) designed to bind and internalize the homodimers (ALPP or ALPPL2) or heterodimers (ALPP/ALPPL2-ADC) complex from the surface of tumor cells and release the microtubule-disrupting agent MMAE, inducing cell cycle arrest and apoptosis. SGN-ALPV has demonstrated highly effective targeting of alkaline phosphatases ALPP and ALPPL2 in preclinical models and will be initially evaluated in ovarian and endometrial cancer, non-small cell lung cancer (NSCLC), gastric cancer, cervical cancer, testicular germ cell tumors and ovarian germ cell tumors where ALPP and ALPPL2 are highly prevalent.

About SGN-B7H4V

SGN-B7H4V is a novel, investigational vedotin ADC directed to the T cell checkpoint ligand, B7-H4. B7-H4 expression is limited on normal tissue and overexpressed on a variety of solid malignancies, including breast, ovarian and endometrial tumors. SGN-B7H4V is designed to bind and internalize the B7-H4/ADC complex from the surface of the tumor cells and release the therapeutic payload MMAE, inducing MMAE-mediated direct cytotoxicity, bystander killing and immunogenic cell death, as well as antibody-dependent cellular cytotoxicity (ADCC) and phagocytosis (ADCP). SGN-B7H4V demonstrates strong activity in xenograft models, including models with heterogenous B7-H4 expression.

Coherus and Junshi Biosciences Present Results of Phase 3 Study of Toripalimab in First Line Treatment of Recurrent or Metastatic Nasopharyngeal Carcinoma at 2022 AACR Annual Meeting

On April 8, 2022 Shanghai Junshi Biosciences Co., Ltd ("Junshi Biosciences", HKEX: 1877; SSE: 688180) and Coherus BioSciences, Inc. ("Coherus", Nasdaq: CHRS) reported the results of the prespecified final progression-free survival ("PFS") analysis and the interim overall survival ("OS") analysis of the JUPITER-02 study (NCT03581786), a pivotal Phase 3 trial in first-line treatment of recurrent or metastatic nasopharyngeal carcinoma ("NPC") (Press release, Coherus Biosciences, APR 8, 2022, View Source [SID1234611698]). The JUPITER-02 results are summarized in a poster presentation at the annual meeting of the American Association for Cancer Research (AACR) (Free AACR Whitepaper) ("AACR").

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

In the final PFS analysis, results from JUPITER-02 demonstrated that toripalimab in combination with chemotherapy provided a statistically significant improvement in PFS assessed by the blinded independent review committee ("BIRC") compared to chemotherapy plus placebo, with an improvement in median PFS of 13.2 months (21.4 versus 8.2 months). Furthermore, the addition of toripalimab to chemotherapy provided significant improvements in the secondary endpoints of PFS assessed by the investigator, objective response rate ("ORR") and duration of response ("DoR"), while maintaining a safety profile consistent with that in previously reported toripalimab clinical trials. Although the median OS ("mOS") was not yet mature in either arm, the interim OS analysis showed a trend favoring the toripalimab arm and will be formally tested in a prespecified final analysis.

"First-line treatment options for advanced NPC remain limited for this difficult-to-treat tumor, resulting in poor outcomes for patients due to therapeutic resistance to chemotherapy, which is the current standard of care," said Professor Ruihua Xu, the poster’s corresponding author from Sun Yat-sen University Cancer Center (SYSUCC). "The JUPITER-02 results validate the potential advancement that toripalimab in combination with chemotherapy would represent as a new standard-of-care first-line therapy for patients with advanced NPC."

Rosh Dias, MD, MRCP, Coherus’ Chief Medical Officer, added, "Innovative immuno-oncology approaches including anti-PD-1 monoclonal antibody treatments represent a promising new option for advanced nasopharyngeal carcinoma, for which there are currently no approved immuno-oncology treatments in the United States. The significant improvement demonstrated in JUPITER-02 with the combination of toripalimab and chemotherapy across key clinically meaningful endpoints compared to chemotherapy alone supports its use as a potential new standard of care treatment option for advanced NPC."

"We are excited that the updated results from JUPITER-02 confirm that the addition of toripalimab to chemotherapy significantly extends the median PFS of patients with advanced NPC by more than a year," said Dr. Patricia Keegan, Chief Medical Officer of Junshi Biosciences. "We believe that toripalimab can revolutionize the treatment of advanced NPC and are working closely with the FDA and our partner, Coherus, to provide the first approved therapy for patients with this rare disease in the U.S."

The United States Food and Drug Administration ("FDA") granted breakthrough therapy designation for toripalimab in combination with gemcitabine and cisplatin as first-line treatment for patients with advanced recurrent or metastatic NPC and for toripalimab monotherapy for second-line or later treatment of recurrent or metastatic NPC after platinum-containing chemotherapy. A biologics license application ("BLA") for these indications is under priority review by the FDA. Coherus and Junshi Biosciences are working closely with the FDA to complete the review process and schedule any required inspections in China.

About JUPITER-02 Study Results

JUPITER-02, conducted in mainland China, Taiwan and Singapore, is the largest Phase 3 clinical study to date to evaluate a checkpoint inhibitor plus chemotherapy for the first-line treatment of recurrent or metastatic NPC. Two hundred eighty-nine patients with advanced NPC, who had received no prior chemotherapy for recurrent or metastatic disease, were randomized 1:1 to receive toripalimab 240 mg or placebo in combination with gemcitabine 1000 mg/m2 (d1, 8) and cisplatin 80 mg/m2 (d1), Q3W followed by toripalimab or placebo monotherapy until disease progression, intolerable toxicity or completion of two years of treatment. PFS and response were assessed by the BIRC and by the investigator per RECIST v1.1. There was one prespecified interim analysis of PFS at 130 (65%) PFS events and a final analysis at 200 PFS events.

At the final PFS analysis (cut-off date June 8, 2021), the median follow-up time was 22.1 months for the toripalimab arm and 21.4 months for the placebo arm.

A summary of the results is as follows:

The addition of toripalimab to gemcitabine-cisplatin ("GP") chemotherapy as first-line treatment for advanced NPC patients provided superior PFS, OS, ORR and DoR than GP chemotherapy alone:
Significant improvement in PFS: mPFS 21.4 vs. 8.2 months, HR=0.52 (95% CI: 0.37, 0.73), P <0.0001.
Significant improvement in ORR: 78.8% vs. 67.1% (P = 0.0221).
Significant improvement in DoR: mDoR 18.0 vs. 6.0 months, HR=0.49, P = 0.0003.
Although mOS was not mature in either arm, a 41% reduction in risk of death was observed in the toripalimab arm over the placebo arm, HR=0.59 (95% CI: 0.37, 0.94), nominal P =0.0238.
The safety profile was consistent with that previously reported in other toripalimab clinical trials with no new safety signals identified with toripalimab added to GP.
About toripalimab

Toripalimab is an anti-PD-1 monoclonal antibody developed for its ability to block PD-1 interactions with its ligands, PD-L1 and PD-L2, and for enhanced receptor internalization (endocytosis function). Blocking PD-1 interactions with PD-L1 and PD-L2 promotes the immune system’s ability to attack and kill tumor cells.

More than thirty company-sponsored toripalimab clinical studies covering more than fifteen indications have been conducted globally by Junshi Biosciences, including in China, the United States, Southeast Asia, and European countries. Ongoing or completed pivotal clinical trials evaluating the safety and efficacy of toripalimab cover a broad range of tumor types including cancers of the lung, nasopharynx, esophagus, stomach, bladder, breast, liver, kidney and skin.

In China, toripalimab was the first domestic anti-PD-1 monoclonal antibody approved for marketing (approved in China as TUOYI). Currently, there are four approved indications for toripalimab in China:

unresectable or metastatic melanoma after failure of standard systemic therapy;
recurrent or metastatic nasopharyngeal carcinoma NPC after failure of at least two lines of prior systemic therapy;
locally advanced or metastatic urothelial carcinoma that failed platinum-containing chemotherapy or progressed within 12 months of neoadjuvant or adjuvant platinum-containing chemotherapy;
in combination with cisplatin and gemcitabine as the first-line treatment for patients with locally recurrent or metastatic NPC.
The first three indications have been included in the National Reimbursement Drug List (NRDL) (2021 Edition). Toripalimab is the only anti-PD-1 monoclonal antibody included in the NRDL for melanoma and NPC.

In addition, two supplemental New Drug Applications (NDAs) for toripalimab are currently under review by the National Medical Products Administration (NMPA) in China:

in combination with chemotherapy as the first-line treatment of patients with advanced or metastatic ESCC.
in combination with chemotherapy as the first-line treatment of patients with advanced or metastatic NSCLC without EGFR or ALK mutations.
In the United States, the FDA has granted priority review for the toripalimab BLA for the treatment of recurrent or metastatic NPC, an aggressive head and neck tumor which has no FDA-approved immuno-oncology treatment options. The FDA has assigned a Prescription Drug User Fee Act ("PDUFA") target action date for April 2022 for the toripalimab BLA. The FDA granted Breakthrough Therapy designation for toripalimab in combination with chemotherapy for the first-line treatment of recurrent or metastatic NPC in 2021 as well as for toripalimab monotherapy in the second or third-line treatment of recurrent or metastatic NPC in 2020. Additionally, the FDA has granted Fast Track designation for toripalimab for the treatment of mucosal melanoma and Orphan Drug Designation for the treatment of esophageal cancer, NPC, mucosal melanoma and soft tissue sarcoma. In 2021, Coherus in-licensed rights to develop and commercialize toripalimab in the United States and Canada. Coherus and Junshi Biosciences plan to file additional toripalimab BLAs with the FDA over the next three years for multiple other cancer types.

Genocea Presents Encouraging Initial Data from GEN-011 Phase 1/2a Trial at AACR 2022

On April 8, 2022 Genocea Biosciences, Inc. (NASDAQ: GNCA), a biopharmaceutical company developing next-generation neoantigen immunotherapies, reported that clinical, preclinical, and manufacturing data at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2022 beginning today in New Orleans and virtually (Press release, Genocea Biosciences, APR 8, 2022, View Source [SID1234611697]). The presentations include promising initial data from the TiTAN clinical trial for the neoantigen-targeted peripheral T cell (NPT) therapy product candidate GEN-011, results demonstrating successful production of GEN-011 using Genocea’s PLANET manufacturing process, and new preclinical data on Inhibigens, antigens of suppressive immune responses uniquely identifiable by Genocea’s ATLAS platform.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The Phase 1/2a TiTAN trial investigates the safety, tolerability, T cell persistence and proliferation, and clinical activity of GEN-011 in patients with refractory solid tumors. The study includes two dosing cohorts. Cohort A patients (n=2) received a lower intensity regimen without lymphodepletion with fractional GEN-011 doses monthly, and with post-infusion intermediate dose interleukin-2 (IL-2) (125K IU/kg daily s.c.). In Cohort B, patients (n=3) received GEN-011 as a single infusion after lymphodepletion, followed by IL-2. This Cohort includes one of three escalating lymphodepletion and IL-2 dose regimens, and patients have not yet been dosed at the highest regimen.

The early results presented at AACR (Free AACR Whitepaper) show anti-tumor activity despite the lower intensity regimens and heavily pretreated tumors. Stable disease was seen at the initial Day 57 scan in four of the five patients. While all patients had progressive disease (PD) at their Day 113 scan, three of the five experienced clear biologic changes after infusion. These included palpable improvement in peripheral nodal disease and resolution of severe neuropathy causing arm paralysis and pain in patients with refractory SCCHN. A patient with metastatic non-small cell lung cancer (NSCLC) experienced a 10% reduction in tumor diameters (approx. 30% reduction in volume), also with resolution of tumor associated cough. The potential for drug product proliferation and persistence for months is supported by translational assays, and clinical activity is associated with declines in detectable circulating tumor DNA (ctDNA) after treatment in some patients.

None of the initial patients have experienced dose-limiting toxicities, with no evidence of self-reactivity or autoimmune toxicity. Overall, the range of Grade 2 and Grade 3 treatment emergent adverse events (TEAEs) align with expected toxicity from cell therapy regimens. The poster presentation and additional context are available in the Scientific Resources section of the Genocea website.

Genocea has had a 100% success rate in manufacturing GEN-011 through its PLANET process to date. Of the 17 patient samples entering PLANET, 100% have either successfully yielded a released drug product (14) or are continuing in process (3). Significantly, as a result of continuous process improvements, the next six patients will be dosed with drug products that have a median two-fold increase in cell dose and greater neoantigen specificity and potency.

"Using peripherally derived T cells and our ATLAS bioassay to target specific neoantigens for inclusion or Inhibigen exclusion in GEN-011 is yielding promising early results in patients," said Thomas Davis, MD, Chief Medical Officer of Genocea. "This activity in low intensity regimens shows that the neoantigen targeted cells are recognizing and engaging with the tumor, which is a very encouraging sign of the potential for greater clinical activity in more intense regimens. Additionally, the continued improvements to our PLANET manufacturing process could lead to more clinically meaningful results in this patient population with high unmet need. We are grateful to our trial participants and are excited for more results to come in Q4 this year."

Additional data presented at AACR (Free AACR Whitepaper) highlights ongoing work characterizing inhibitory antigens, or Inhibigens – putatively pro-tumor antigens that are uniquely identifiable by ATLAS and present in nearly every cancer patient profiled by Genocea. With the benefit of ATLAS, Genocea excludes T cells to Inhibigens from GEN-011. A preclinical poster presented at the meeting demonstrates how detrimental these Inhibigens are to the efficacy of cancer therapeutics in mouse models of melanoma and pancreatic cancer.

Genocea is hosting an investor webcast with live Q&A at 4:30 pm ET on Friday, April 8. Dr. Melissa Johnson, Program Director of Lung Cancer Research and the Solid Tumor Immune Effector Cellular Therapy Program at the Sarah Cannon Cancer Institute, will join Genocea leadership to discuss the GEN-011 clinical results and other data being presented at AACR (Free AACR Whitepaper). The live webcast will be available on the Events & Presentations page of the Genocea website, with the recording and poster presentations in the Scientific Resources section immediately following the event.

AACR POSTER SESSION CATEGORY: Phase 1 Adult Clinical Trials
Abstract #CT153
Title: TiTAN: a phase 1 study of GEN-011, a neoantigen-targeted peripheral blood-derived T cell therapy with broad neoantigen targeting
Presenter: Maura Gillison, MD, PhD, MD Anderson Cancer Center
Date: Monday, April 11, 2022
Time: 1:30 p.m. – 5:00 p.m. CT
Clinical Results from 5 initial patients show no dose limiting toxicities and proliferation and persistence of desired effector memory T cells for at least 36 days. As expected from a non-exhausted peripherally-derived T cell product, this persistence supports the biological activity of GEN-011.

AACR POSTER SESSION CATEGORY: Inflammation, Immunity, and Cancer
Abstract #2088
Title: The PLANET manufacturing process reproducibly generates high-quality neoantigen-targeted peripheral T cells (NPTs) for adoptive T cell therapy in the TiTAN clinical trial
Presenter: Harshal Zope, PhD, Genocea Biosciences
Date: Monday, April 11, 2022
Time: 1:30 p.m. – 5:00 p.m. CT
Results of the PLANET manufacturing process show 100% success in the production of a customized drug product for patients, each including exclusively T cells covering 90% of the intended neoantigen targets. The resulting neoantigen-targeted peripheral T cells are non-exhausted, broadly reactive, and include up to 30 characterized neoantigen targets.

AACR POSTER SESSION CATEGORY: Clinical Research Excluding Trials
Abstract #2745
Title: ATLAS-identified Inhibigen-specific responses accelerate tumor growth in mouse melanoma and pancreatic cancer
Presenter: Jessica Flechtner, PhD, Genocea Biosciences
Date: Tuesday, April 12, 2022
Time: 9:00 a.m. – 12:30 p.m. CT
Inhibigens, regardless of antigen type, disrupt an otherwise protective vaccine in mouse models of melanoma. Furthermore, Inhibigens also promote tumor growth in pancreatic cancer models, demonstrating the effect is not cancer type-specific. Transplanting T cells into nude mice confirms that Inhibigen-specific T cells exert this pro-tumor effect.

About GEN-011
GEN-011 is a neoantigen-targeted peripherally derived T cell therapy candidate comprised of autologous CD4+ and CD8+ T cells that are specific for up to 30 ATLAS-identified neoantigens to limit tumor escape. NPTs have minimal bystander, non-tumor-specific cells, and are devoid of Inhibigen-specific cells which may be detrimental to clinical response.

About the GEN-011 TiTAN clinical trial
TiTAN is an open-label, multi-center Phase1/2a trial evaluating safety, tolerability, T cell persistence and proliferation and clinical efficacy of GEN-011. The TiTAN clinical trial is testing two cohorts. Cohort A patients receive a fractionated lower dose regimen of GEN-011 without lymphodepletion and an intermediate IL-2 regimen to maximize the tumor-killing potential of the infused cells. Cohort B patients receive a single high dose administration of GEN-011, along with one of three escalating regimens of lymphodepletion and IL-2.