Data presented at the AACR Virtual Annual Meeting II – June 22-24

Company Product Description Indication Status
Allogene Therapeutics Inc., of South San Francisco DLL3-targeted AlloCAR T Genetically modified T cells expressing CARs targeting Delta-like ligand 3 Small-cell lung cancer Preclinical data showed lead DLL3 candidates had robust efficacy in both subcutaneous and systemic models of SCLC; while DLL3 RNA has normal tissue expression in the brain, pituitary and testis, toxicity studies using subcutaneous and intracranial tumor models showed no tissue damage in the brain or pituitary
Allogene Therapeutics Inc., of South San Francisco Inducible TurboCAR Technology allowing cytokine activation signaling to be selectively controlled by a small-molecule on-switch  Cancer Research indicated iTurboCARs can be tailored for diverse, programmable and combinatorial signaling outputs, and may have potential to provide benefits of cytokine signaling while minimizing safety risks associated with concomitant cytokine therapy or constitutive cytokine secretion
Beyondspring Inc., of New York Plinabulin Immune and stem cell modulator Tumors Preclinical data showed drug improves tumor control by enhancing dendritic cell maturation and CD8 T-cell infiltration in combination with immunoradiotherapy; findings include a 100% complete response of the triple immuno-oncology combination of plinabulin, anti-PD-1 and radiation in a PD-1 antibody non-responsive model
Cue Biopharma Inc., of Cambridge, Mass. CUE-012/A02  Immuno-STAT biologics designed to target and expand WT1-specific T cells Cancer Preclinical studies demonstrated selectivity and potency of drug to bind, activate and expand WT1-specific T cells
Jaguar Health Inc., of San Francisco Mytesi (crofelemer) Non-opiate chloride ion channel modulator antidiarrheal drug Diarrhea associated with breast cancer drug Nerlynx Preclinical data of study conducted in collaboration with Puma Biotechnology Inc. showed 7 of 8 dogs treated with neratinib/crofelemer QID were responders (p=0.02) and 6 of 8 treated with neratinib/crofelemer BID were responders (p=0.03) vs. 3 of 8 dogs in control group for 28-day dosing period
Medigene AG, of Martinsried, Germany PD1-41BB switch receptor PD1-41BB switch receptor Solid tumors Preclinical data showed drug significantly improves the functional activity of T-cell receptor-modified T cells, especially against solid tumor cells
Mersana Therapeutics Inc., of Cambridge, Mass. XMT-1592 Dolasynthen antibody-drug conjugate targeting NaPi2b Ovarian cancer and lung adenocarcinoma Preclinical data showed drug improved in vivo activity, pharmacokinetics and clinical pathology relative to its stochastic counterpart; data also showed that XMT-1592 induced sustained tumor regressions in an NSCLC adenocarcinoma patient-derived xenograft
Mersana Therapeutics Inc., of Cambridge, Mass. STING-agonist antibody-drug conjugates STING-agonist antibody-drug conjugates Tumors Preclinical data showed target-dependent antitumor immune responses in vitro and in vivo as a single well-tolerated dose for multiple targets in multiple preclinical models; potent ADC-mediated tumor regression led to durable immunological memory in an immune competent model
Molecular Templates Inc., of Austin, Texas MT-511 HER2-targeted engineered toxin body Metastatic cholangiocarcinoma, metastatic breast cancer, metastatic gastroesophageal junction carcinoma Data from ongoing phase I trial showed no dose-limiting toxicities to date, including no cardiotoxicity; ongoing subject from cohort 2 with metastatic breast cancer has stable disease and remains on treatment, now in cycle 5; 1 subject in cohort 3 with metastatic breast cancer has had a follow-up CT scan at the end of cycle 2 and has stable disease; 6 subjects have discontinued for disease progression and 2 are too early to evaluate
Molecular Templates Inc., of Austin, Texas MT-6402 PD-L1-targeted, antigen seeding engineered toxin body Tumors Preclinical data showed potent in vitro activity against a variety of PD-L1+ tumor cells along with tumor growth delay and survival benefits in NSCLC PDX in vivo model; nonhuman primate (NHP) data showed MT-6402-mediated PD-L1+ immune cell clearance can elicit highly potent monotherapy immune activation in a way not seen previously in NHP models with checkpoint inhibitors 
Molecular Templates Inc., of Austin, Texas CTLA4 ETB 1 CTLA4-targeted engineered toxin bodies designed to deplete regulatory T cells Tumors Preclinical data from co-culture models showed CTLA4 ETBs relieved Treg suppression of T-effector proliferation; experiments in mice showed that CTLA4 ETB 1 displayed short serum half-life and is well-tolerated in vivo
Molecular Templates Inc., of Austin, Texas SLAMF7-targeting ETB Engineered toxin bodies targeting SLAMF7 (CS1) Multiple myeloma SLAMF7 ETBs shown to be active alone and in presence of elotuzumab and can combine with standard-of-care chemotherapy and bortezomib in a positive manner in vitro
Neoleukin Therapeutics Inc., of Seattle NL-201 IL-2/IL-5 agonist Cancer Preclinical data showed ability of NL-201 to stimulate and expand CD8+ and NK cells at very low doses with minimal impact on immunosuppressive regulatory T cells; treatment in animal models was well-tolerated and induced durable, antitumor immunity; minimal immunogenicity reported following 5 weekly doses in nonhuman primates
Nimbus Therapeutics Inc., of Cambridge, Mass. NMBS-1 Small-molecule HPK1 inhibitor Tumors Preclinical data showed high selectivity against T cell-specific kinases and kinases in MAP4K family and activity in in vitro and in vivo models; drug enhanced IL-2 production from stimulated human T cells, alleviated PGE2-mediated immunosuppression of T-cell activation and enhanced IL-6 production, proliferation and IgG secretion from B cells; in mouse syngeneic tumor model, oral administration resulted in significant tumor growth inhibition, both as monotherapy and in combination with anti-CTLA4
Nkarta Inc., of South San Francisco CAR NK cells to disrupt TGFbR2 and CISH Targets genes related to down-regulation of cytokine signaling and immune cell activation Tumors Preclinical data showed TGFbR2 knockout CAR NK cells were resistant to TGFb-mediated inhibition of tumor cell killing; CISH knockout CAR NK cells had improved proliferation, survival, cytokine production and tumor cell killing; findings showed gene editing can be combined with engineering to enhance NK cell function and resist the inhibitory action of the tumor microenvironment
Nkarta Inc., of South San Francisco CAR-NK and CAR T cells Combination of CAR natural killer cells and CAR T cells Tumors Research showed enhanced antitumor cytotoxicity and persistence, which lowered accumulation of cytokines associated with cytokine release syndrome, promoted NK cell expansion while reducing antigen-dependent proliferation of T cells, and prevented tumor relapse in an animal model for at least 4 months
Oric Pharmaceuticals Inc., of South San Francisco ORIC-101 Glucocorticoid receptor antagonist Tumors Preclinical data showed transcriptional signature of GR activity identified in panel of 32 cell lines across triple-negative breast cancer, non-small-cell lung cancer and pancreatic ductal adenocarcinoma, which translated from preclinical models to human tumors; drug overcame GR-mediated resistance to chemotherapeutic agents in both in vitro and in vivo efficacy studies in multiple solid tumor types; ORIC-101 reversed GR-activated pathways involved in drug resistance, and reversed in vivo markers of epithelial-to-mesenchymal transition, anti-apoptosis and hypoxia
Oric Pharmaceuticals Inc., of South San Francisco ORIC-533 CD73 inhibitor Tumors Preclinical data demonstrated suppression of adenosine production in vitro across multiple cell types and rescued activation of CD8+ T cells exposed to AMP with greater potency than competitor compounds; drug was shown to result in sustained inhibition of adenosine production after drug washout, consistent with its slow off-rate, and differentiating from other CD73 inhibitors; daily oral delivery of CD73 inhibitors significantly inhibited tumor growth, with corresponding in vivo reduction of adenosine levels in tumors, and immune modulation consistent with decreased immunosuppression
Phio Pharmaceuticals Corp., of Marlborough, Mass. PH-804 TIGIT-targeting Intasyl self-delivering RNAi compound Colorectal carcinoma Preclinical data showed tumor growth inhibition was determined for both PH-804 and an anti-TIGIT antibody in colorectal carcinoma tumor-bearing mice; compound was efficiently delivered intratumorally to immune cells, resulting in a dose-dependent inhibition of tumor growth, reaching statistical significance levels for PH-804 and anti-TIGIT antibody treatment arms; analysis of tumor microenvironment in PH-804-treated mice showed a dose-dependent increase in cytotoxic effector T cells, and a dose-dependent activation of such T cells as shown by the expression of activation makers such as CD25 and CD69
Puretech Health plc, of Boston LYT-200 Fully human monoclonal antibody targeting galectin-9 Cancer Preclinical data showed efficacy in reducing tumor growth and reactivating human effector T cells in preclinical, patient-derived tumor culture models
Rgenix Inc., of New York RGX-104 LXR agonist Advanced solid tumors Dose-escalation stage of phase I trial as monotherapy or in combination with either Opdivo (nivolumab, Bristol Myers Squibb Co.), Yervoy (ipilimumab, BMS) or docetaxel in heavily pretreated patients with refractory or relapsed solid tumors, including patients who had progression on prior checkpoint inhibitors (CPIs), showed objective clinical activity in all treatment arms, with partial responses (PRs) achieved in patients with non-small-cell lung cancer, small-cell lung cancer, melanoma, squamous cell carcinoma of the head and neck and endometrial cancer; in combination arms, 28.6% objective response rate observed in all evaluable patients who previously progressed on CPI, with 4 of 14 achieving PRs
Rgenix Inc., of New York RGX-019 MerTK-targeting monoclonal antibody Cancer Preclinical data showed drug inhibited human cancer cell viability and induced immune-stimulatory cytokine expression in M2 macrophages, consistent with robust inhibition of MerTK signaling; treatment in vivo led to near complete elimination of MerTK from the surface of tumor cells, which was associated with antitumor efficacy in mouse xenograft models; favorable safety profile shown in 28-day toxicology study in monkeys
Rubryc Therapeutics Inc., of San Carlos, Calif. RTX-003 CD25-targeted monoclonal antibody Cancer Preclinical data showed antibodies bound specifically to CD25+ cells, preserved IL-2 signaling via pSTAT5 assays and elicited potent ADCC activity using human effector cells, and had in vivo efficacy superior to benchmark antibodies
Silverback Therapeutics Inc., of Seattle  SBT-6050 HER2-directed TLR8 ImmunoTAC myeloid cell agonist Multiple tumors In preclinical studies, SBT-6050 activates human myeloid cells in the presence of HER2-expressing tumor cells, resulting in proinflammatory cytokine and chemokine production, inflammasome activation and indirect activation of cytolytic activity
Sotio AS, of Prague, Czech Republic SO-C101 IL-15 superagonist Relapsed/refractory advanced/metastatic solid tumors. Reduced and prevented tumor growth in mouse models 
Spectrum Pharmaceuticals Inc., of Henderson, Nev. Rolontis (eflapegrastim) G-CSF therapy Neutropenia Duration of neutropenia in a rat model of chemotherapy-induced neutropenia was observed to be significantly shorter with Rolontis vs. pegfilgrastim (Neulasta, Amgen Inc.), regardless of the timing of administration: concomitantly or on the same day post-chemotherapy at 2, 5 or 24 hours post-chemo
Sutro Biopharma Inc., of South San Francisco STRO-002 Antibody-drug conjugate targeting folate receptor alpha Advanced epithelial ovarian cancer Preclinically, treatment induced hallmarks of immunogenic cell death, killing tumor cells while activating immune cells, including monocytes
TG Therapeutics Inc., of New York TG-1701 Anti-PD-L1 monoclonal antibody; BTK inhibitor Mantle cell lymphoma Showed greater selectivity for BTK than, and similar activity to, ibrutinib (Imbruvica, Pharmacyclics LLC/Johnson & Johnson) in preclinical models
Theratechnologies Inc., of Montreal TH1-902, TH-1904 Peptide-drug conjugates Multiple tumor types Sortilin 1, the drugs' target, is highly expressed in cancer cells involved in the vasculogenic mimicry (VM) process in ovarian cancer and triple-negative breast cancer; CD133, a gene associated with cancer stem cells, is also highly expressed during VM formation; TH-1902 (peptide-docetaxel conjugate) and TH-1904 (peptide-doxorubicin conjugate) strongly inhibit VM at very low doses preclinically
Tolero Pharmaceuticals Inc., of Salt Lake City Alvocidib and TP-1287, an oral prodrug of alvocidib CDK9 inhibitor  Acute myeloid leukemia Presented a biomarker detection method to assess CDK9 inhibition through reduction of myeloid cell leukemia-1 expression and of phosphorylation of RNA polymerase II 
Tonix Pharmaceuticals Holdings Corp., of New York TNX-1700 Stabilized recombinant version of trefoil factor 2 Colorectal cancer Anti-PD-1 monotherapy was unable to evoke antitumor immunity in a model of colorectal cancer, but the drug augmented the efficacy of anti-PD-1 therapy
Transgene SA, of Strasbourg, France TG-4050 Patient-specific neoantigen cancer vaccine Non-small-cell lung cancer Data showed the prediction algorithm used to customize TG-4050 for each patient is accurate at identifying immunogenic cancer mutations even among a large set of candidate tumor mutations
Transgene SA, of Strasbourg, France BT-001 Oncolytic Vaccinia virus armed with Treg-depletion-optimized recombinant human anti-CTLA4 antibody and GM-CSF to target tumor microenvironment Multiple tumor types Cure rates exceeding 70% were seen in multiple mouse models
Transgene SA, of Strasbourg, France TG-6010 Invir.IO-based oncolytic virus encoding human cytidine deaminase Multiple tumor types Induced DNA damage; showed potent antitumor effects preclinically
Turning Point Therapeutics Inc., of San Diego Repotrectinib Small (low-molecular-weight), macrocyclic tyrosine kinase inhibitor of ROS1, TRK and ALK Multiple tumor types Shown to increase the effectiveness of KRAS-G12C and MEK inhibitors in cancer models
Turning Point Therapeutics Inc., of San Diego TPX-0131 Next-generation ALK inhibitor Multiple tumor types In cell-proliferation assays, exhibited greater potency against wild-type ALK as compared to proxy molecules for approved front-line ALK inhibitors
Twoxar Pharmaceuticals Inc., of Mountain View, Calif. TXR-311 Cytotoxic agent Hepatocellular carcinoma Demonstrated greater activity and selectivity in killing tumor cells than standard of care, sorafenib (Nexavar, Bayer AG), and significantly inhibited growth in 2 patient-derived xenograft tumor models; showed in vivo efficacy comparable to sorafenib with good tolerability
Tyme Technologies Inc., of New York SM-88 (racemetyrosine) Tyrosine derivative Multiple tumor types Preclinically demonstrated statistically significant tumor reduction in drug-treated arms vs. controls
Tyme Technologies Inc., of New York TYME-18 Surfactant delivery agent with a specific sulfonic acid component Multiple tumor types A minor reduction in tumor cell mass was observed in response to the surfactant component alone, with a statistically significant response observed in the group treated with TYME-18 (surfactant component + sulfonic acid component) (p=0.001); preclinical data showed that 91.6% (11/12) of mice treated with TYME-18 were tumor-free by study end compared with tumor growth of about 16 times mean growth in the control group
VBI Vaccines Inc., of Cambridge, Mass. VBI-1901 Cancer vaccine immunotherapeutic Recurrent glioblastoma Emerging data from the ongoing phase I/IIa study suggest that patients with a normal baseline CD4+/CD8+ T-cell ratio may be more likely to experience delayed progression or tumor reduction, reflected as a tumor response; 5 of 6 tumor responses seen to date, including a recently confirmed partial response, defined as a tumor reduction of more than 50% per the Response Assessment in Neuro-Oncology criteria, suggest the biomarker may predict patients most likely to respond to, and derive clinical benefit from, treatment with the drug
VBL Therapeutics Ltd., of Tel Aviv, Israel MOSPD2 bispecific antibody candidates T-cell activators Metastatic cervical and breast cancers Significantly extended the lives of animals tested
Verastem Inc., of Boston Defactinib FAK inhibitor Metastatic uveal melanoma Preclinical data support proof-of-concept for combining VS-6766, the company's RAF/MEK inhibitor, with defactinib
Xencor Inc., of Monrovia, Calif. Xmab-30819 Xmab 2+1 ENPP3 x CD3 bispecific antibody  Renal cell carcinoma Demonstrated safety and efficacy in in vivo preclinical studies
Xencor Inc., of Monrovia, Calif. Xmab candidate Affinity tuned Xmab 2+1 anti-mesothelin x anti-CD3 bispecific antibody Ovarian cancer Induced selective T cell-directed cell cytotoxicity of human ovarian cancer cells
Xencor Inc., of Monrovia, Calif. Xmab candidate Affinity tuned Xmab 2+1 PSMA x CD3 bispecific antibodies Prostate cancer Demonstrated selective activity in prostate cancer models

Notes

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