On November 3, 2022 OSE Immunotherapeutics SA (ISIN: FR0012127173; Mnemo: OSE) reported that its leukemia research program on OSE-127 has been selected for an oral presentation at the American Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting (New Orleans, Louisiana; December 10 – 13, 2022) (Press release, OSE Immunotherapeutics, NOV 3, 2022, View Source [SID1234646952]). This upcoming presentation has received the merit-based "Abstract Achievement Award" from the peerreview committee.

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Nicolas Poirier, Chief Executive Officer of OSE Immunotherapeutics, comments: "We are honored by the recognition and the interest in the quality of our research in Leukemia from the prestigious American Society of Hematology (ASH) (Free ASH Whitepaper). This award is the result of our fruitful collaboration with the University of Kiel evaluating, in patient-derived xenograft models, the therapeutic potential of OSE-127 in targeting and blocking the high and dysregulated IL-7 receptor-expression observed in more than 80% of B- or T-Acute Lymphoblastic Leukemia (ALL) patients".

Pr. Denis Schewe (Head of the Pediatrics Department, Otto-von-Guericke-University, Magdeburg and formerly from the University Medical Center Schleswig-Holstein of Kiel) and Dr. Lennart Lenk (Department of Pediatrics I, Christian-Albrechts University Kiel and University Medical Center Schleswig-Holstein, Kiel), leading the research program in collaboration with OSE Immunotherapeutics, say: "Acute lymphoblastic leukemia is a very aggressive tumor arising from B or T cells precursors (B- and T-ALL, respectively). Relapse remains a clinical challenge in B-ALL in highrisk patients and treatment options for T-ALL remain very limited. Novel targeted immunotherapy approaches are urgently needed to prevent relapse and to treat refractory diseases in ALL patients".

The global intellectual property for OSE-127 has been further strengthened, based on a unique and innovative dual anti-leukemic mechanism of action. This antibody both blocks oncogenic interleukin-7 fuel pathway and simultaneously triggers macrophage-driven phagocytosis of leukemic cells. Additional patent applications were filed in 2021 and 2022 covering the use of anti-IL-7 receptor antagonist antibodies with macrophage-redirected phagocytic activity for the targeted treatment of IL-7 receptor-positive cancers.

Furthermore, OSE-127 is currently being developed in clinical stage in partnership with Servier. Two clinical studies are ongoing in inflammatory diseases: a phase 2a study conducted in primary Sjögren’s syndrome by Servier and a phase 2 study conducted in ulcerative colitis by OSE Immunotherapeutics.

About Acute Lymphoblastic Leukemia (ALL)
Acute lymphoblastic leukemia (ALL) is a heterogeneous group of lymphoid disorders resulting from clonal proliferation of immature lymphocytes of B-cell (85%) or T-cell (15%) lineages(1) in the blood, bone marrow, and other lymphoid organs.

Although it is one of the most common cancers in children, accounting for approximately 25% of all
childhood cancer diagnoses among children under 15 years of age (2), adults can also develop ALL.
About 40% cases of ALL diagnosed are in adults and among them about 50% present refractory disease
or undergo relapse under current conventional therapies(2)

On November 3, 2022 Auransa Inc., a clinical stage drug development company using propriety AI drug discovery platform to identify novel drug candidates for oncology, CNS and inflammatory diseases, reported that the U.S. Food and Drug Administration (FDA) has accepted its Investigational New Drug (IND) application for AU409, a novel, orally active agent showing anticancer activity in preclinical studies of hepatocellular carcinoma (HCC) (Press release, Auransa, NOV 3, 2022, View Source [SID1234635671]). Initial clinical studies will focus on patients with advanced primary liver cancers and patients with advanced solid tumor with liver predominant metastatic disease.

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"The FDA’s acceptance of the IND for AU409 is an important milestone for Auransa’s AI drug discovery platform. We look forward to initiating the Phase 1 study, our first-in-human trial of a compound derived from Auransa’s proprietary SMarTR Engine." said Pek Lum, Ph.D., founder and chief executive officer of Auransa.

"AU409’s anti-cancer activity in preclinical models is through a mechanism of action distinct from currently available, FDA approved tyrosine kinase inhibitors used for hepatocellular carcinoma making this an important new opportunity for clinical testing." said Andrew Protter, Ph.D. Auransa’s Chief Scientific Officer.

About AU409
AU409 is a novel small molecule with oral active in models of hepatocellular carcinoma. In preclinical studies, AU409 has been shown to modulate transcription of certain genes thereby altering the gene expression profile of liver cancer cells. The mechanism of action of AU409 is distinct from that of current drugs approved for HCC including the tyrosine kinase inhibitors (TKIs) such as sorafenib or regorafenib. Non-clinical safety, toxicology and genetic toxicology studies support the first in human clinical studies being proposed.

About Hepatocellular Carcinoma
Despite major improvements in the treatment of primary liver cancers including hepatocellular carcinoma, patients with advanced disease continue to have limited median overall survival due to primary or secondary resistance to existing therapies. While chronic hepatitis B and C infections continue to be important risk factors for liver cancer, the rising prevalence of obesity, non-alcoholic steatohepatitis (NASH) and alcohol consumption are becoming the dominant risk factors for liver cancer in the United States as well as the rest of the world. Liver cancer has recently been estimated to be the third most common cancer related death worldwide.

On November 3, 2022 Targovax reported its financial report of third quarter 2022 (Presentation, Targovax, NOV 3, 2022, View Source [SID1234624679]).

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On November 3, 2022 Cellectis reported Business Update and Reports Financial Results for Third Quarter and First Nine Months 2022 (Press release, Cellectis, NOV 3, 2022, View Source [SID1234624610]).

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On November 3, 2022 Pacylex reported that data from the ongoing Phase 1 dose escalation clinical trial of PCLX-001, an N-myristoyltransferase (NMT) inhibitor, as well as the scientific rationale for its use in acute myeloid leukemia (AML) patients, reported that it will be presented at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition from 10-13 December 2022 (Press release, Greenfire, NOV 3, 2022, View Source [SID1234624502]). The ongoing study is in Non-Hodgkin’s Lymphoma and solid tumor patients and the company will present the non-clinical evidence that AML patients may benefit as well.

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In addition to the presentation, the abstract will be published online in the November supplemental issue of Blood.

Seventeen Non-Hodgkin Lymphoma and solid tumor patients have been accrued through 5 dose levels of oral, once-per-day, PCLX-001. No dose limiting toxicities have been identified. Pharmacokinetic analysis shows rapid oral absorption, an elimination half-life of ~ 10 hours, no plasma accumulation, and drug exposure increasing linearly with dose. The PK is consistent with once-per-day oral administration. Last month the U.S. FDA granted Orphan Drug Designation to PCLX-001 for "treatment of patients with acute myeloid leukemia." This month Pacylex received clearance from the U.S. Food and Drug Administration (FDA) of an Investigational New Drug (IND) application for PCLX-001 for the treatment of AML.

"We are very pleased by the clinical progress which has demonstrated that an NMT inhibitor can be dosed safely to levels proportional to those that achieved efficacy in vitro and in animal cancer models," said John Mackey, CMO of Pacylex.

The poster will also present results from in vitro and in vivo studies that indicate PCLX-001 is at least as effective against AML cancer cells, including leukemic stem cells (LSCs), the sub-population believed to be responsible for AML recurrence, as it is against lymphoma cells. "In two different patient derived xenograft models, the LSC subpopulation were even more sensitive to PCLX-001 than AML circulating blast cells, which were still very sensitive," said Luc Berthiaume, CSO of Pacylex. "This provides further support for our plans to initiate a clinical in AML patients in the coming months."

The ongoing clinical PCLX-001 trial in NHL and solid tumor patients is registered at ClinicalTrials.gov Identifier: NCT04836195.

PCLX-001
PCLX-001 (aka DDD86481) is a first-in-class, small molecule NMT inhibitor originally developed by the University of Dundee Drug Discovery Unit as part of a program to treat African sleeping sickness, funded by Welcome Trust. Pacylex is developing PCLX-001 in the form of a once-a-day pill initially to treat leukemia and lymphoma. PCLX-001 has also been shown to inhibit the growth of lung and breast cancer tumors in animal models. In leukemia, lymphoma and breast cancer patients, the levels of NMT2 is correlated with survival, suggesting an important biological role in these cancers.