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.

On November 3, 2022 Helix BioPharma Corp. (TSX: "HBP") ("Helix" or the "Company"), a clinical-stage biopharmaceutical company developing unique therapies in the field of immuno-oncology, based on its proprietary technological platform DOS47, reported that it has closed a private placement financing for net proceeds of CAD $4,629,019.86 from the issuance of 25,716,777 common shares at a price of $0.18 per common share (Press release, Helix BioPharma, NOV 3, 2022, View Source [SID1234624188]).

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The purchase of common shares by directors/ insiders is considered a "related party transaction" within the meaning of Multilateral Instrument 61-101 – Protection of Minority Security Holders in Special Transactions ("MI 61-101"). The Company relied on exemptions from the formal valuation and minority approval requirements in sections 5.5(a) and 5.7(1)(a) of MI 61-101 in respect of the insiders purchase of common shares. The Company did not file a material change report in respect of the related party transaction less than 21 days prior to the closing of the private placement, which the Company deems reasonable in the circumstances so as to be able to avail itself of the proceeds of the private placement in an expeditious manner.

"We would like to thank all investors for their strong support and confidence in Helix. We look forward to continued efforts toward this exciting program" said Mr. Gabor, CEO of Helix.

The common shares issued pursuant to the Private Placement are subject to a statutory hold period of four months and one day ending on March 4, 2023, in accordance with applicable securities law. In connection with the closing, the Company will pay a cash fee of 10% of gross proceeds raised to an eligible finder.

The Company intends to use the net proceeds of the private placement for working capital and advancing the Company’s L-DOS47 drug development program.

The securities offered have not been registered under the United States Securities Act of 1933, as amended, and may not be offered or sold in the United States or to, or for the account or benefit of, U.S. persons absent registration or an applicable exemption from registration requirements. This news release does not constitute an offer for sale of securities in the United States.