On January 17, 2023 Xenetic Biosciences, Inc. (NASDAQ:XBIO) ("Xenetic" or the "Company"), a biopharmaceutical company focused on advancing innovative immune-oncology technologies addressing hard to treat oncology indications, reported that the Canadian Intellectual Property Office (CIPO) has issued a notice of allowance for Patent Application No. 3,001,543 titled, "Method to Improve Safety and Efficacy of Anti-Cancer Therapy (Press release, Xenetic Biosciences, JAN 17, 2023, https://ir.xeneticbio.com/news/detail/132/xenetic-biosciences-inc-announces-notice-of-allowance-for-canadian-patent-covering-use-of-dnase-enzyme-for-preventing-or-ameliorating-toxicity-associated-with-chemotherapy [SID1234626273])." A patent from the recently allowed application is expected to be issued in the coming months.

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"We continue to be excited about the potential of the DNase platform technology and are committed to advancing its development as a top priority. As we continue to execute on our progress towards the clinic, strengthening the intellectual property portfolio around DNase is a focus for us. We are pleased to add this Canadian patent to our existing worldwide IP estate, which includes the U.S. as well as the other patent applications we have filed around the world," commented Jeffrey Eisenberg, Chief Executive Officer of Xenetic Biosciences.

The allowed patent covers claims including use of a therapeutically effective amount of a DNase enzyme for preventing or ameliorating a toxicity associated with a cytostatic and/or cytotoxic chemotherapy in a subject suffering from a cancer and received or deemed to receive said chemotherapy, wherein said amount of the DNase enzyme is effective to prevent or ameliorate at least one side effect of said chemotherapy.

The Company’s interventional DNase based oncology platform is aimed at improving outcomes of existing treatments, including immunotherapies. The Company’s exclusive license to CLS Therapeutics’ intellectual property for uses of DNases in cancer include systemic co-administration of DNases along with standard therapies, including chemotherapy, radiation and checkpoint inhibitors, or along with conventional chimeric antigen receptor (CAR) T therapies. Xenetic is focused on advancing its systemic DNase program into the clinic as an adjunctive therapy for pancreatic carcinoma and other locally advanced or metastatic solid tumors.

On January 17, 2023 Biomea Fusion, Inc. ("Biomea") (Nasdaq: BMEA), a biopharmaceutical company focused on the discovery and development of covalent small molecules to treat patients with genetically defined cancers and metabolic diseases, reported that the first patient has been dosed in COVALENT-102, the company’s Phase I/Ib trial of BMF-219, an oral, selective, covalent menin inhibitor in patients with KRAS-mutated unresectable, locally advanced, or metastatic NSCLC, CRC, and PDAC (Press release, Biomea Fusion, JAN 17, 2023, View Source [SID1234626272]).

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"We are eager to explore the potential of BMF-219 as a pan-KRAS inhibitor in patients with three of the most prominent KRAS-mutant solid tumor types, including those with tumors that have failed to respond to investigational and approved mutation-specific KRAS inhibitors," said Steve Morris, M.D., Biomea’s Chief Medical Officer. "As a covalent menin inhibitor, we believe BMF-219 has critical advantages over late stage, mutation-specific inhibitors of KRAS including independence on the KRAS activation state, reduced likelihood for acquisition of resistance mutations, and its potential to address multiple activating KRAS mutations."

KRAS is the most frequently mutated isoform amongst RAS oncogenes in human solid tumors, with high prevalence in NSCLC, CRC, and PDAC. KRAS G12C, KRAS G12D and KRAS G12V are among the most common KRAS mutations. However, there are numerous other known activating KRAS mutations. With only two approved therapies both targeting only KRAS G12C for locally advanced or metastatic NSCLC, KRAS-driven tumors continue to represent a significant unmet medical need.

As a covalent menin inhibitor, BMF-219 has manifested a differentiated profile over the commercially approved KRAS-targeted inhibitors LUMAKRAS (sotorasib) and KRAZATI (adagrasib) in multiple pre-clinical studies. As previously reported by Biomea, KRAS-mutant NSCLC, CRC, and PDAC cell lines and ex vivo patient specimens were highly sensitive to BMF-219 in preclinical models. The higher levels of activity of BMF-219 were observed among various KRAS-mutant solid tumor cell lines, but not KRAS wild type, suggesting that BMF-219 broadly inhibited mutant KRAS in these tumor models. A targeted pan-KRAS inhibitor has the potential to treat a large number of NSCLC, CRC, and PDAC patients.

About COVALENT–102
COVALENT-102 is an open-label, multi-cohort, multicenter, Phase I/Ib dose escalation and expansion study evaluating the safety, tolerability, and optimal biologic dose of BMF-219 administered orally to adult patients with KRAS-mutated unresectable, locally advanced or metastatic NSCLC, CRC, and PDAC. Additional information about the Phase I/Ib clinical trial of BMF-219 can be found at ClinicalTrials.gov using the identifier NCT05631574.

About Non-Small Cell Lung Cancer (NSCLC)
NSCLC is the most common form of lung cancer, representing approximately 82% of all lung cancer cases or approximately 200,000 cases in the U.S. each year (Source: NCI SEER Data). The five-year survival rate of NSCLC is approximately 25%. While lung cancer is the third most common form of cancer in the U.S. based on incidence, it contributes to the highest number of annual cancer deaths in the U.S. KRAS is the most frequently mutated oncogene in NSCLC, occurring in approximately 30% of patients. There remains a great unmet need for targeted therapies to address all KRAS driver mutations and avoid known mechanisms of resistance.

About Colorectal Cancer (CRC)
CRC is the fourth most common form of cancer and the second leading cause of cancer death in the U.S., representing approximately 150,000 cases in the U.S. each year (Source: NCI SEER Data). These cancers start in the rectum or the colon and can be diagnosed/identified early, even potentially as noncancerous polyps. The five-year survival rate of CRC is approximately 65%. Among other mutations, KRAS mutations occur in approximately 40% of patients with CRC. These mutations can not only help predict the absence of response to anti-EGFR therapy, but also result in poorer overall survival. Therefore, there remains a significant unmet need for personalized therapies for patients with KRAS-mutant colorectal cancer.

About Pancreatic Cancer (PDAC)
Pancreatic cancer is a relatively rare form of cancer in the U.S., representing approximately 60,000 cases in the U.S. each year (Source: NCI SEER Data). Pancreatic cancer is an aggressive cancer with a very low five-year survival rate of approximately 11%, indicating that there is a large unmet need. 80% of patients are diagnosed at an advanced stage, contributing to the low survival rate. KRAS mutations are found in nearly all pancreatic cancer patients and are considered as a driver of the malignant process in most of those patients.

On January 17, 2023 Idera Pharmaceuticals, Inc. ("Idera") (Nasdaq: IDRA), a clinical-stage biopharmaceutical company committed to transforming the care of people living with rare pulmonary and rheumatic diseases, reported that the Company has changed its name and symbol to Aceragen, Inc. ("Aceragen," the "Company," "we," "us," or "our") and "ACGN". Additionally, the Company’s stockholders approved the conversion into common stock of the Series Z preferred shares resulting from the previously announced merger with Aceragen and authorized a reverse split of common stock (Press release, Idera Pharmaceuticals, JAN 17, 2023, View Source [SID1234626271]). The Company’s Board of Directors has approved the reverse stock split at a ratio of 1-for-17 shares. As a result of these changes, which will be effective upon the market open on Wednesday, January 18, 2023, the Company will be in compliance with all applicable Nasdaq listing standards. Nasdaq has issued an approval letter confirming Aceragen’s listing.

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"We are excited to have completed the transformation of Aceragen via our merger with Idera and the subsequent adjustments to our stock and Nasdaq listing. We believe this transition strengthens our portfolio of late-stage clinical assets in cystic fibrosis and Farber disease and aligns with our goal of delivering important therapies for people living with rare diseases," stated John Taylor, Aceragen’s Chief Executive Officer. "During the course of this year, we anticipate the achievement of significant clinical milestones that include two Phase 2 data read-outs for ACG-701, as well as lifting of the clinical hold and advancing toward the initiation of our Phase 2/3 trial in Farber disease for ACG-801."

"With the positive result of the stockholder vote behind us, I share in John’s excitement and optimism for Aceragen and look forward to the progress that this team will make for patients in need," added Vincent J. Milano, Chair of Aceragen’s Board of Directors.

Clinical Development Overview
Aceragen has a portfolio of late-stage clinical assets in cystic fibrosis and Farber disease with clinical milestones anticipated in 2023.

ACG-701 for Acute Pulmonary Exacerbations in Cystic Fibrosis
ACG-701 is a proprietary oral, loading dose formulation of sodium fusidate being developed as a treatment for acute pulmonary exacerbations ("PEx") associated with cystic fibrosis ("CF"), a major factor driving lung function decline in people living with CF. Sodium fusidate has an established clinical efficacy and safety profile from more than 50 years of use in other countries, including as part of CF PEx treatment guidelines in the United Kingdom and Australia. Despite this, the compound has never been approved by the FDA and represents a new and potentially powerful approach in the United States to address the infection, inflammation, and enhanced mucin expression that are hallmark features of CF PEx.

A Phase 2 trial of ACG-701 in CF PEx (the REPRIEVE study), a randomized double-blinded, placebo-controlled study, was initiated in December 2022 at clinical sites in the United States in collaboration with the CF Foundation’s Therapeutic Development Network (TDN). The CF Foundation has also provided funding of $3.5 million in support of the study. If approved, ACG-701 would represent the first product in the United States indicated for the treatment of newly diagnosed CF PEx patients. Data from the REPRIEVE study is expected in 2H 2023. The FDA has granted Orphan Drug Designation, Fast Track and Qualified Infectious Disease Product (QIDP) status to ACG-701 for CF PEx.

ACG-701 for Melioidosis
Aceragen has also executed an ~$50 million development partnership with the Department of Defense’s Defense Threat Reduction Agency ("DTRA") to investigate ACG-701 as a potential medical countermeasure for melioidosis, a life-threatening infection caused by the B. pseudomallei pathogen. This program is centered around a Phase 2 trial, the TERRA study (NCT05105035), which is a randomized double-blind, placebo-controlled trial conducted in hospitalized melioidosis patients. TERRA was initiated in May 2022 and continues to actively enroll patients with an independent DMC review planned during Q1 2023 and a data read-out expected in 2H 2023.

ACG-801 for Farber Disease
ACG-801, recombinant human acid ceramidase, is an investigational biologic in development to be the first-ever enzyme replacement therapy for the treatment of Farber disease, a progressive, severe, and life-threatening lysosomal storage disorder that is caused by the monogenic deficiency of acid ceramidase. The biochemical hallmark of Farber disease is the loss of acid ceramidase enzyme activity leading to abnormal accumulation of ceramide, profound macrophage-driven inflammation and multi-organ disease affecting bone and joints, cartilage, the immune system, central nervous system, and the lungs. Complications of the disease are life threatening, with many patients dying in the first years of life. There are no ceramide-targeted medications currently available that can alter the disease natural history.

The Company expects to initiate the ADVANCE clinical study for ACG-801 in Farber disease, a randomized, double-blind, placebo-controlled, first-in-human study, in the first quarter of 2024 with data expected in the first quarter of 2025. Due to the ultra-rare nature of Farber disease, if successful, this study has the potential to support registration of the product. The FDA has granted Orphan Drug, Fast Track, and Rare Pediatric Disease designations for ACG-801. Rare pediatric disease designation permits priority review voucher eligibility, upon FDA marketing authorization.

Clinical Milestone Summary

ACG-701 – REPRIEVE study in CF PEx, data expected 2H 2023
ACG-701 – TERRA study in melioidosis, data expected 2H 2023
ACG-801 – ADVANCE study in Farber disease, initiation expected in Q1 2024
Projected cash available is expected to provide the Company with capital runway into Q3’2023.

On January 16, 2023 Pacylex Pharmaceuticals Inc. (Pacylex) is a clinical-stage pharmaceutical company focused on the development of a new class of targeted therapies, N-myristoyltransferase inhibitors (NMTi) for the treatment of hematologic and solid tumor cancers, reported that the results of its Phase 1 dose escalation safety and tolerability study for zelenirstat, an investigational NMT inhibitor and Pacylex’s lead product candidate, in refractory/ relapsed colorectal and other cancers, will be presented January 20, 2024, at the upcoming ASCO (Free ASCO Whitepaper) Gastrointestinal Cancers Symposium (Press release, Pacylex Pharmaceuticals, JAN 16, 2023, View Source [SID1234645053]).

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The Phase 1 dose escalation safety and tolerability study was conducted in 29 heavily pre-treated solid tumor and lymphoma patients who averaged 4 prior lines of therapy on which they relapsed or were refractory (R/R). Eight colorectal, two pancreatic, and one appendiceal cancer patients were among those included in the study. The most common treatment related adverse events identified in the trial were mild to moderate gastrointestinal side effects which were self-limiting and occurred in a minority of patients. A recommended Phase 2 dose (RP2D) for expansion studies was established. Zelenirstat prolonged progression free and overall survival in Phase 1 solid tumor patients receiving the RP2D. Prolonged Stable Disease was observed in patients with colorectal (320 + days on treatment and continuing with non-RECIST criteria reductions of approximately 50% in CEA (carcinoembryonic antigen) and tumor volumes) and appendiceal (160+ days on treatment and continuing with stable disease) cancer treated with the RP2D. The Company has initiated dosing in a Phase 2a expansion study of patients with B-cell non-Hodgkin lymphoma.

A poster describing the Company’s Phase 1 study safety results and efficacy signals, particularly in gastrointestinal cancer patients, will be presented at the ASCO (Free ASCO Whitepaper) Gastrointestinal Cancers Symposium on January 18-20, 2024, in San Francisco, CA. The Company’s CEO, Dr. Michael Weickert, will be available during the poster session and for one-on-one meetings.

"The extended benefits observed in patients with heavily pre-treated GI cancers has been an exciting clinical study finding," said Dr. John Mackey, Chief Medical Officer for Pacylex and a practicing oncologist. "Those patients have received the Phase 2 dose of zelenirstat for longer than anyone and the reductions in disease burden in the colorectal patient is very encouraging."

On January 16, 2023 AIGEN Sciences reported that it signed a research cooperation agreement with Yuhan Corporation to identify the mechanism of action of a new artificial intelligence (AI)-based anticancer drug (Press release, AIGEN Sciences, JAN 16, 2023, View Source [SID1234643564]).

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This agreement aims to identify the mechanism of action of Yuhan Corporation’s new anticancer drug candidate by applying Igen Science’s proprietary artificial intelligence platform.

Under this contract, Eisen Science predicts the mechanism of action of anticancer drug candidates being developed by Yuhan Corporation by utilizing its own artificial intelligence platform based on transcriptome data that can derive the drug’s potential target and mechanism of action. A target is derived. Yuhan Corporation plans to verify the target and mechanism of action proposed by Eisen Science and then accelerate the follow-up development of a differentiated new anticancer drug.

An official from Eisen Science said, "We are developing innovative new drug candidates through our own and joint development projects using artificial intelligence technology based on cell-level transcriptome data," adding, "Even before the research cooperation agreement with Yuhan Corporation, we were developing domestic anticancer drugs and antibody-drug conjugates. "We have signed contracts with (ADC) development companies and are conducting research to discover candidate substances," he said.