Arcellx Presents Pre-Clinical Data for ACLX-002, a Novel CD123-targeted Universal CAR-T Cell Therapy for Relapsed or Refractory Acute Myeloid Leukemia at the American Association for Cancer Research Annual Meeting

On April 8, 2022 Arcellx, Inc. (NASDAQ: ACLX), a biotechnology company reimagining cell therapy through the development of innovative immunotherapies for patients with cancer and other incurable diseases, reported the presentation of preclinical data utilizing its novel ARC-SparX platform for ACLX-002, a CD123-targeted universal CAR-T cell therapy for relapsed or refractory Acute Myelogenous Leukemia (AML) and high-risk myelodysplastic syndrome (MDS) (Press release, Arcellx, APR 8, 2022, View Source [SID1234611789]). The data show that ACLX-002 completely regressed disseminated MOLM14 and MV4-11 tumors in a schedule and dose-dependent manner without the aid of alloreactivity and performs similarly to a traditional CD123-targeting D-domain based CAR. Additionally, ACLX-002 completely regresses multiple cell line and patient-derived AML xenografts.

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"There are limited treatment options for AML and MDS patients as heterogeneity is known to be a major challenge for targeted therapeutics in this patient population," said Rami Elghandour, Arcellx’s chairman and chief executive officer. "Our ARC-SparX platform is designed to allow for controllability and adaptability, to potentially reduce toxicities that are often associated with serious dose-limiting adverse events and to overcome tumor heterogeneity. These ACLX-002 preclinical data provide the scientific rationale to move forward with initiating a Phase 1 clinical trial and our ambition to deliver a meaningful treatment option for these patients. We look forward to starting this study in the second half of this year."

The data are being presented at the American Association of Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting taking place April 8-13, 2022. Presentation details are as follows:

The poster can be accessed through the AACR (Free AACR Whitepaper) Annual Meeting program or the company’s corporate website www.arcellx.com/pipeline-focus-area/#scientific-publications

About the ARC-SparX Platform Technology
The ARC-SparX platform is designed to allow for controllability and adaptability, to potentially reduce toxicities that are often associated with serious dose-limiting adverse events and to overcome tumor heterogeneity. It is a modular therapy which utilizes a universal ARC-T cell combined with an off the shelf SparX protein to separate the tumor-recognition and tumor-killing functions. SparX (soluble protein antigen-receptor X-linkers) proteins utilize our D-Domain technology engineered to recognize antigens on the surface of diseased cells and flags those cells for detection by the ARC-T (Antigen Receptor Complex-T) cells. ARC-T cells express a D-Domain-based CAR engineered to specifically recognize a unique TAG in the SparX protein. ARC-T cells are dosed separately and only activated to kill the target cell when they encounter a SparX protein bound to the target antigen thus are controlled through SparX dose modulation. Arcellx has developed a collection of SparX proteins that bind different antigens on the surface of diseased cells. Multiple SparX proteins with different antigen specificity can be administered to potentially address antigen heterogeneity or antigen escape that contribute to relapsed and refractory disease.

Omega Therapeutics Presents Preclinical Data on OTX-2002, First-in-Class Epigenomic Controller, as Potential Treatment for Hepatocellular Carcinoma at the AACR Annual Meeting 2022

On April 8, 2022 Omega Therapeutics (NASDAQ: OMGA) (Omega), a development-stage biotechnology company pioneering the first systematic approach to use mRNA therapeutics as a new class of programmable epigenetic medicines by leveraging its OMEGA Epigenomic Programming platform, reported that it will present preclinical data highlighting the potential of its lead Omega Epigenomic Controller, OTX-2002, to regulate overexpression of the c-Myc (MYC) oncogene in models of hepatocellular carcinoma (HCC) in a poster presentation at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2022, taking place in New Orleans, Louisiana, April 8-13, 2022 (Press release, Omega Therapeutics, APR 8, 2022, View Source [SID1234611786]).

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"Despite its essential role in a broad range of cancers, MYC has remained undruggable to date," said Thomas McCauley, Ph.D., Chief Scientific Officer of Omega Therapeutics. "However, we believe that targeting the MYC gene pre-transcriptionally within its Insulated Genomic Domain (IGD), and epigenetically tuning it using our epigenomic controller, could overcome the challenges that have limited previous technologies including small molecules, antisense oligos and siRNA. We believe that these data strongly support OTX-2002’s ability to tune and restore MYC expression to a normal range and demonstrate the broader potential of our Epigenomic Programming platform to tackle previously intractable diseases. We are excited to continue advancing OTX-2002 into clinical trials and look forward to filing an Investigational New Drug application in the first half of this year."

Key findings

A single dose of OTX-2002 induced durable changes in the epigenetic profile of the MYC gene
OTX-2002 reduced MYC mRNA expression and protein levels over approximately 2 weeks in vitro
Downregulation of MYC in multiple HCC cell lines resulted in significant loss in viability of MYC-addicted cancer cells while sparing normal cells
In murine xenograft HCC models, OTX-2002 significantly reduced tumor growth and was well-tolerated
Cumulatively, these data support the filing of an Investigational New Drug application with the U.S. Food and Drug Administration for the clinical development of OTX-2002 in the first half of 2022.

The poster can be viewed on the Omega website at View Source

About OTX-2002

OTX-2002 is a first-in-class Omega Epigenomic Controller in development for the treatment of hepatocellular carcinoma (HCC). OTX-2002 is designed to modulate levels of c-MYC (MYC) expression by utilizing targeted mRNA-expressed proteins to mediate epigenetic regulation while potentially overcoming MYC autoregulation. The MYC oncogene is associated with aggressive disease in up to ~70% of patients with HCC. Omega is currently evaluating OTX-2002 in Investigational New Drug (IND)-enabling studies.

KAZIA THERAPEUTICS ANNOUNCES PRECLINICAL DATA PRESENTED AT AACR CONFERENCE BY JOHNS HOPKINS UNIVERSITY SHOWING ACTIVITY OF PAXALISIB IN PAEDIATRIC BRAIN TUMOURS

On April 8, 2022 Kazia Therapeutics Limited (NASDAQ: KZIA; ASX: KZA), an oncology-focused drug development company, reported new preclinical data demonstrating the activity of paxalisib in two forms of childhood brain cancer with very high unmet medical need (Press release, Kazia Therapeutics, APR 8, 2022, View Source [SID1234611784]).

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This data is the subject of three abstracts presented at the Annual Meeting of the American Association for Cancer Research (AACR) (Free AACR Whitepaper), held in New Orleans, LA, from April 8 – 13, 2022.

Two abstracts by scientists working in the laboratory of Assistant Professor Jeffrey Rubens at Johns Hopkins University in Baltimore, MD, describe the use of paxalisib as a backbone therapy in a childhood brain cancer known as atypical teratoid / rhabdoid tumours (AT/RT). This is the first time that data exploring paxalisib in this form of brain cancer has been presented, and it opens an important new potential indication for the drug.

The third abstract, from a different team of scientists at Johns Hopkins University, led by Associate Professor Eric Raabe and Dr Katherine Barnett, showed evidence of strong synergy between paxalisib and another class of cancer therapies in a model of diffuse intrinsic pontine glioma (DIPG). Paxalisib has previously shown evidence of activity in this disease, both as monotherapy and in combination with several types of cancer therapy, and the new data further validates its potential in this very challenging disease.

Key Points

AT/RT is a rare brain cancer that predominantly affects infants and young children. There are no FDA approved drugs for AT/RT and existing therapeutic options are very limited. Fewer than one in five patients survive more than two years from diagnosis.
Data from Professor Rubens’ laboratory shows that the PI3K pathway is commonly activated in AT/RT, and that treatment with paxalisib alone is active in preclinical models of the disease. Moreover, combination with either RG2822, an HDAC inhibitor, or TAK580, a MAPK inhibitor, appears to substantially extend survival when compared to monotherapy treatment.
DIPG is a rare brain cancer that is typically seen in young children and adolescents. There are no FDA approved drugs, and average life expectancy from diagnosis typically averages around ten months.
Previous data from several teams of researchers, and particularly from Professor Matt Dun’s team at the Hunter Medical Research Institute, has shown that paxalisib is highly active in DIPG and combines synergistically with several cancer drugs.
Data from Drs Raabe and Barnett and colleagues identifies an additional novel treatment combination, with the HDAC inhibitor RG2833, which exhibits evidence of strong synergy in a preclinical model of DIPG.
Kazia CEO, Dr James Garner, added, "This is very promising data, and we are grateful to the team at Johns Hopkins for this important and encouraging research. Paxalisib is already the subject of an ongoing phase II clinical trial in DIPG and diffuse midline gliomas (NCT05009992) and this new data suggests potential wider applications for the drug in childhood brain cancers. We look forward to working with the Johns Hopkins team, and with other partners and advisors, to explore these opportunities further."

Summary of Abstracts

SESSION OPO.CL02.01 – Pediatric Cancer: Clinical Investigations

April 8, 2022 – 12:00pm-1:00pm

Abstract 5225 – The PI3K inhibitor Paxalisib combines with the novel HDAC1/3 inhibitor RG2833 to improve survival in mice bearing orthotopic xenografts of atypical teratoid/rhabdoid tumors
Tyler Findlay, Kristen Malebranche, Charles Eberhart, Eric Raabe, Jeffrey Rubens.
Johns Hopkins School of Medicine, Baltimore, MD

Abstract 5226 – The PI3K inhibitor Paxalisib combines synergistically with the pan-Raf inhibitor TAK580 (DAY 101) to extend survival in orthotopic xenograft models of atypical teratoid/rhabdoid tumors
Kristen Malebranche, Tyler Findlay, Charles Eberhart, Eric Raabe, Jeffrey Rubens.
Johns Hopkins School of Medicine, Baltimore, MD

SESSION PO.ET04.01 – Identification of Molecular Targets

April 13, 2022 – 9:30am-12:30pm

Abstract 3987 / 9 – Brain penetrant HDAC and PI3K/mTOR inhibitors synergize to induce DIPG cell death
Katherine Barnett, Hyuk Jean Kwon, Orlandi Novak, Charles Eberhart, Eric Raabe.
Johns Hopkins Hospital, Baltimore, MD

Bantam Pharmaceutical and Collaborators to Present New Data in Support of its lead drug BTM-3566 at AACR Annual Meeting 2022

On April 8, 2022 Bantam Pharmaceutical, a drug discovery and development company targeting selective modulation of mitochondrial dynamics in cancer, reported that it will present new data on its lead drug candidate BTM-3566 at AACR (Free AACR Whitepaper) 2022 taking place the 8th-13th of April in New Orleans (Press release, Bantam Pharmaceutical, APR 8, 2022, View Source [SID1234611783]). The data demonstrate that BTM-3566 co-opts a specific mitochondrial quality control pathway to induce apoptosis in cell lines, xenografts and patient-derived xenograft (PDX) models of Diffuse Large B-cell Lymphoma (DLBCL).

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The poster presentation on Wednesday, April 13th highlights the robust in vivo activity of BTM-3566, including complete and durable regressions in 6 out of 9 PDX models with a 100% response rate across a range of difficult to treat DLBCL tumors. Equally important, BTM-3566 acts through a novel mechanism of action with activity across multiple genomic and phenotypic subtypes of DLBCL, suggesting this represents an impactful treatment option for patients progressing on existing standard-of-care agents. Emerging data characterizing the responses and dependency on expression of a mitochondrial protein will also be presented.

The abstract is available now on the AACR (Free AACR Whitepaper) website.

Michael Stocum, CEO of Bantam Pharmaceutical, commented: "We are excited to be presenting this new data at AACR (Free AACR Whitepaper). Together with our academic collaborators, we continue enhancing our understanding of the novel mechanism of BTM-3566 and its capacity to induce apoptosis in cancer cells. We expect to file an Investigational New Drug (IND) application for BTM-3566 this quarter (2Q 2022) and are prepared to initiate clinical trials later this year. Our IND and pre-clinical data are generating significant enthusiasm with oncologists as we advance toward the clinic."

Title: BTM-3566 co-opts mitochondrial quality control pathways to induce apoptosis, and complete tumor regression in DLBCL cell lines, xenografts and PDX models

About BTM-3566

BTM-3566 is an orally-available novel small molecule compound with broad anti-cancer activity in hematologic and solid tumors, initially focused on Diffuse Large B-cell Lymphomas (DLBCL). BTM-3566’s anti-cancer mechanism of action is unique and differentiated from other therapeutics, disrupting mitochondrial function in tumor cells to induce apoptosis (cell death). An IND application for BTM-3566 in B-cell malignancies is being completed for submission in Q2 2022.

Nuvalent Presents New Data Demonstrating Expanded Preclinical Activity with ROS1-Selective Inhibitor NVL-520 and ALK-Selective Inhibitor NVL-655 at AACR Annual Meeting 2022

On April 8, 2022 Nuvalent, Inc., (Nasdaq: NUVL) a clinical-stage biopharmaceutical company creating precisely targeted therapies for patients with cancer, reported new data to support broad clinical exploration of its parallel lead programs NVL-520 – a ROS1-selective inhibitor – and NVL-655 – an ALK-selective inhibitor (Press release, Nuvalent, APR 8, 2022, View Source [SID1234611782]). NVL-520 and NVL-655 are central nervous system (CNS)-penetrant kinase inhibitors designed to specifically solve for the dual challenges of kinase resistance and selectivity commonly observed with currently available inhibitors.

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The data are available via two posters presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2022, which runs from April 8 through April 13. The posters will also be available on the Nuvalent website at www.nuvalent.com/news/.

"We are pleased to share new data today resulting from our continued collaborations with leading investigators in ROS1 and ALK research, which we believe further demonstrate the potential for our highly selective inhibitors to be differentiated within the dynamic treatment landscapes for non-small cell lung cancer (NSCLC) and beyond," said James Porter, Ph.D., Chief Executive Officer of Nuvalent. "These preclinical data support the inclusion of various fusion partners and resistance mutations in our ARROS-1 and ALKOVE-1 clinical trials for ROS1- and ALK-positive NSCLC, respectively, as well as the inclusion of exploratory cohorts for other advanced solid tumors outside of NSCLC."

Monika Davare, Ph.D. is an Associate Professor of Pediatrics, Division of Hematology and Oncology at Oregon Health & Science University School of Medicine and leading expert in ROS1 oncoprotein biology. Professor Davare’s research is directed towards overcoming therapeutic bottlenecks in oncology, including those that arise from the lack of validated translational research models for genomic subsets of cancer.

"Translational models of ROS1-driven cancers have centered on NSCLC, where the clinical impact of fusion partners is not yet well characterized and new treatment-resistant variants continue to emerge. Hypothesizing that broad coverage of fusion partners and resistance mutations is a beneficial feature for next-generation ROS1 inhibitors, we conducted an extensive comparative analysis of NVL-520 versus currently approved as well as investigational ROS1 inhibitors," said Professor Davare. "In contrast to comparator compounds, NVL-520 exhibited consistently high potency (IC50 < 10 nM) across all models tested, and in particular displayed potencies against the recurrently problematic G2032R solvent front mutation that were ≥ 1-order of magnitude higher than all comparative agents tested."

"While models of ROS1-driven cancers outside of NSCLC are sparse, we further present data that NVL-520 induces regression in a human cell-line derived model of glioblastoma driven by a ROS1 fusion," continued Professor Davare. "This suggests potential for clinical utility outside of NSCLC and highlights the importance of developing additional research models to help accelerate the development of new therapies for genomically-driven cancers."

Luc Friboulet, Ph.D. is an investigator at Gustave Roussy focused on investigating molecular mechanisms of tumor adaptation to kinase inhibitors in solid tumors, with particular expertise in understanding resistance to ALK kinase inhibitors.

"ALK oncogenic activations as well as mutations conferring resistance to current ALK inhibitors have been characterized across a range of tumor types, suggesting that broad activity across diverse ALK-driven cancers is a beneficial feature for next-generation ALK inhibitors," said Dr. Friboulet. "In a comparative analysis of NVL-655 versus currently approved as well as investigational ALK inhibitors, NVL-655 exhibited strong activity across a wide range of fusion partners, activating mutations, and disease backgrounds, suggesting potential for broad clinical utility. Importantly, this expansive activity of NVL-655 against ALK does not come at the expense of its ability to avoid inhibition of TRKB, a limitation that has been observed with other investigational ALK inhibitors."

NVL-520 is currently under investigation in the Phase 1 portion of the Phase 1/2 ARROS-1 study (NCT05118789) for advanced ROS1-positive NSCLC and other solid tumors. Nuvalent recently announced the IND clearance for NVL-655 and plans to initiate the Phase 1 portion of the Phase 1/2 ALKOVE-1 study for advanced ALK-positive NSCLC and other solid tumors in the second quarter of 2022. In addition to these parallel lead programs, Nuvalent is advancing a robust discovery pipeline with plans to nominate two new development candidates in 2022 for ALK IXDN compound mutations and HER2 exon 20 insertions.

AACR Presentation Overview:

* Presenting authors

Title: NVL-520: Preclinical Activity of NVL-520 in ROS1-Driven Cancer Models with Diverse Fusion Partners and Kinase-Domain Mutations
Authors: Anupong Tangpeerachaikul*^, Clare Keddy^, Katelyn Nicholson, Monika A. Davare, and Henry E. Pelish*
^ Equal contributions
Poster Number: 14
Permanent Abstract: 3336
Session Category: Experimental and Molecular Therapeutics
Session Title: Tyrosine Kinase and Phosphatase Inhibitors
Session Date and Time: Tuesday April 12, 2022 from 1:30 – 5:00 p.m. Central Time
Location: New Orleans Convention Center, Exhibit Halls D-H, Poster Section 26

Summary of Presentation:

NVL-520 shows high activity against diverse ROS1 fusion partners tested including CD74, CEP85L, EZR, GOPC(L), GOPC(S), and SLC34A2 and induces regression in a ROS1-driven model of glioblastoma harboring GOPC(L)-ROS1.
NVL-520 shows high activity against diverse ROS1 kinase-domain mutations tested including S1986F, F2004C/V, L2026M, G2032R, D2033N, and G2101A.
NVL-520 shows a differentiated preclinical activity and selectivity profile compared to other inhibitors tested.
Preclinical activity against diverse ROS1 fusion partners and kinase domain mutations suggests broad potential clinical utility of NVL-520.
Title: NVL-655: Preclinical Activity of NVL-655 in ALK-Driven Cancer Models beyond Non-Small Cell Lung Cancer
Authors: Anupong Tangpeerachaikul*, Ludovic Bigot, Luc Friboulet, and Henry E. Pelish*
Poster Number: 15
Permanent Abstract: 3337
Session Category: Experimental and Molecular Therapeutics
Session Title: Tyrosine Kinase and Phosphatase Inhibitors
Session Date and Time: Tuesday April 12, 2022 from 1:30 – 5:00 p.m. Central Time
Location: New Orleans Convention Center, Exhibit Halls D-H, Poster Section 26

Summary of Presentation:

NVL-655 shows strong activity in diverse preclinical models of ALK-driven cancers: cholangiocarcinoma, neuroblastoma, lymphoma, and soft-tissue sarcoma.
Among all inhibitors tested, NVL-655 shows the broadest activity for diverse ALK oncoproteins including fusions, point mutations, and partial N-terminal deletions.
NVL-655 shows larger ALK-vs-TRK selectivity windows than lorlatinib and TPX-0131.
Preclinical activity against diverse ALK oncoproteins (fusions, mutations, and partial deletions) in multiple tumor types suggests broad potential clinical utility of NVL-655.