On January 20, 2023 Universal Diagnostics (Universal DX), a bioinformatics and multi-omics company on a mission to transform cancer into a curable disease, reported the results of an international, observational cohort study which evaluated the effectiveness of utilizing a combination of cell-free DNA (cfDNA) methylation, fragmentation and machine learning to detect early-stage colorectal cancer (CRC) (Press release, Universal Diagnostics, JAN 20, 2023, View Source [SID1234626435]).

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This is the latest in a series of findings from Universal DX; today’s news demonstrates the use of methylation and fragmentation characteristics of cancer-related cfDNA regions, combined with a machine-learning algorithm is highly accurate for early-stage (I-II) CRCs (92% sensitivity at 94% specificity). These results were achieved on a prospectively collected patient sample set from four different populations: the U.S., Spain, Germany, and Ukraine.

Universal DX has previously shown that non-invasive blood testing can be used to detect CRC and pre-cancerous advanced adenomas (AA) through both analysis of cell-free circulating tumor DNA (ctDNA) methylation, fragmentation and microbiome patterns with single targeted sequencing analysis and combining it with advanced computational biology and machine learning algorithms. In 2022, the company extended early-stage colorectal cancer detection to prognostics and stratification; the ability to do so could lead to better outcomes and improved survival rates.

"This study further validates and reinforces the work we are doing to develop tests that detect cancer in its earliest stages," said Christian Hense, COO at Universal DX. "With a completely new sample set, we have again demonstrated highly-accurate early-stage CRC detection, further verifying the robustness of our technology and use of biomarkers to find traces of cancer in a person’s blood. At Universal DX, we believe early detection is one of the most powerful tools for improving survival rates, and are encouraged to see these promising results once again."

Study results:

Prediction model that utilized a panel of methylation and fragmentation scores originating from cfDNA biomarkers that belong to relevant cancer development and progression-related pathways correctly classified 92% (87/95) of CRC patients.
Sensitivity per cancer stage ranged from 91% (21/23) for stage I, 92% (23/25) for stage II, 91% (30/33) for stage III and 93% (13/14) for stage IV.
Fragmentation signals contributed most to early-stage cancers (I-II), while methylation signals were more significant for late stage (III-IV) detection.
Specificity of the model was 94% (199/204), with 97% (28/29) NAA (non-advanced adenoma), 93% (116/125) BEN (benign colonoscopy findings of diverticulosis/diverticulitis, hemmorhoids, hyperplastic/inflammatory polyps) and 94% (47/50) cNEG (colonoscopy negative) patients correctly identified.
Lesion location, gender, age and country of origin were not significantly correlated to prediction outcome.
Universal DX leverages proprietary, state-of-the-art computational biology tools combined with a targeted next generation sequencing assay platform that allows for simultaneous detection of methylation, fragmentation and microbiome signals for highly-sensitive cancer signal scoring of cell-free DNA regions linked to cancer of interest.

Hense continued: "Colorectal cancer is the third most common cancer diagnosed in the U.S.; in 2023, The American Cancer Society estimates there will be more than 150,000 new diagnoses of colon and rectal cancer. This demonstrates the need to not only prioritize screening, but find tools that help us detect cancer in its earliest forms, when there are more treatment options available and survival rates are higher."

Universal DX will present its findings live at the ASCO (Free ASCO Whitepaper) Gastrointestinal Cancers Symposium in San Francisco, CA on January 21st as poster #201 titled "Use of methylation and fragmentation signals in the detection of early-stage colorectal cancer."

In addition to today’s results, the company will be presenting findings from additional studies in the upcoming months.

On January 20, 2023 Genexine (KOSDAQ: 095700), a publicly traded, clinical-staged Korean biopharmaceutical company committed to the discovery and development of novel biologics for the treatment of unmet medical needs, reported it received Fast Track Designation (FTD) from the Korean Ministry of Food and Drug Safety (MFDS) for GX-188E (tirvalimogene teraplasmid), its first-in-class proprietary therapeutic DNA vaccine (Press release, Genexine, JAN 20, 2023, View Source [SID1234626434]).

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Following an evaluation of the full set of Phase 2 data from the recently completed clinical trial in advanced cervical cancer, Korea’s Health Authority (MFDS) concluded that GX-188E met the criteria for fast-track designation. Under MFDS regulations, FTD is given to a drug that is intended to treat a serious condition and the nonclinical or clinical data demonstrate the potential to address an unmet medical need. Having such a designation can mean that a drug can move more quickly through the development and regulatory process in an expedited manner.

"We are grateful to the Korean Health Authority for their careful evaluation and recognition that GX-188E has the potential to be a key life-saving drug for the treatment of advanced cervical cancer," said Neil Warma, Genexine’s President and CEO. "We are committed to the cancer patients in which this therapy could be effective and appreciate that FTD could help to possibly speed our time to market to deliver the drug to patients more rapidly. We are in the process of designing the optimal Phase 3 study with GX-188E and expect to initiate that study this year."

Genexine recently reported Phase 2 trial data which evaluated the efficacy and safety of the combination of GX-188E and KEYTRUDA (pembrolizumab), MSD’s (Merck & Co., Inc., Rahway, NJ., USA) anti-PD-1 therapy, in a total of 65 patients (safety population) with HPV 16- and/or HPV 18- positive recurrent or metastatic advanced cervical cancer. The final efficacy analysis evaluated in 60 patients (efficacy evaluable population) showed an Objective Response Rate (ORR) of 35% (21 of 60 patients) indicating that of the 60 patients with advanced cervical cancer, 21 patients saw either over 30% reduction in tumor size or complete remission.

On January 20, 2023 BriSTAR Immunotech, a clinical-stage cell therapy company, reported it will showcase its T-cell receptors (TCR) and antigen receptor (STAR)-T cell therapy technology platform at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) special conference on acute myeloid leukemia and myelodysplastic syndrome, taking place January 23-25, 2023, in Austin, Texas (Press release, Bristar Immunotech, JAN 20, 2023, View Source [SID1234626425]).

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"The data shows that our LILRB4 STAR-T cell therapy exhibits potent anti-tumor activity against AML in preclinical models," said Dr. James Pan, CEO at BriSTAR Immunotech. "These findings support our strategy to maximize the potential of our unique STAR-T technology platform."

Presentation Details:

Abstract title: Development of LILRB4 biparatopic synthetic T-cell receptor and antigen receptor (STAR)-T cells for the treatment of acute myeloid leukemia (AML)
Presenter: Dr. James Pan
Date / Time: Tuesday, January 24, 2023 7:15 – 9:30 p.m. CST
The complete abstract will be available here. An electronic copy of the poster is available upon request EMAIL: [email protected].

About LILRB4 STAR-T

BriSTAR Immunotech has developed LILRB4 STAR-T cell therapy for the treatment of relapsed/refractory acute myeloid leukemia (R/R AML). This novel cell therapy drug candidate was developed using BriSTAR’s STAR-T platform to introduce the biparatopic STAR gene targeting LILRB4 into autologous T cells with a lentiviral gene transduction approach. This next generation T cell therapy uses two novel nano-antibodies that bind to different epitopes and fused to the alpha and beta chains of the STAR structure, respectively. This gives LILRB4 START-T better antigen engagement and increased cytotoxicity against this highly aggressive AML. In December 2022, the LILRB4 STAR-T program received Orphan Drug Designation from the U.S. FDA.

About Acute Myeloid Leukemia (AML)

Acute myeloid leukemia (AML) is a cancer of the blood and bone marrow. AML occurs when a bone marrow cell suddenly develops genetic abnormalities. It is highly heterogeneous with limited therapeutic options and poor prognosis. AML is the most common acute leukemia in adults, with an incidence of over 20 000 cases per year in the United States alone, and China ranked among the top three in the world in terms of the number of AML cases and deaths, with 13,200 and 7,100 cases, respectively. For patients with relapse/refractory AML, there is no treatment that significantly extends the survival period of 5-9 months.

About STAR-T Platform

BriSTAR Immunotech’s synthetic TCR and antigen receptor (STAR)-T cell therapy technology platform is highly effective at building a product pipeline for both hematological and solid tumors. STAR-T has the characteristics of natural T cells, such as sensitive target engagement and strong tumor infiltration and offers a natural framework for engineering dual-targeting T cell products.

In September 2022, an investigational new drug (IND) application for CD19/CD20 STAR-T cell injection (research code: HXYT-001) was granted by Center for Drug Evaluation (CDE), National Medical Products Administration (NMPA), for the treatment of recurrent/refractory non-Hodgkin lymphoma (B-NHL). In addition, the STAR-T exploratory clinical studies have been initiated for several types of solid tumors.

On January 20, 2023 Regen BioPharma, Inc. (OTC PINK: RGBP) and (OTC PINK: RGBPP), a biotechnology company advancing a diverse pre-clinical pipeline spanning cell therapies, RNA vaccines, RNA and DNA therapeutics and small molecule drugs reported today the filing of a provisional patent application covering the use of survivin-engineered dendritic cells and exosomes for stimulation of anti-cancer immunity (Press release, Regen BioPharma, JAN 20, 2023, View Source [SID1234626424]). The intellectual property provides additional means of stimulating specific elements of the immune system to selectively seek and destroy cancer cells without harming healthy tissue.

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Dendritic cells are the most potent immune cell capable of activating T cells. T cells are the effectors of the immune system and in the present case are the cells that are involved in killing survivin-expressing tumor cells. The cancer marker survivin appears to be present on most of the major cancers but not on non-malignant tissues.

The Company has previously designed dendritic cells for treatment of breast cancer utlizing a process called gene silencing. The current work capitalized on lessons learned in developing DCellVax[1], as well as novel findings regarding utilizing of exosomes, naturally occurring nanoparticles with ability to modulate the immune system.

"The field of immunotherapy is expanding at an unprecedented rate and this is exemplified by the astronomical rise in the use of immunotherapeutic drugs, which now are believed to possess a 100 billion global annual market[2]," said David Koos, CEO and Chairman of the Company. "Through positioning ourselves to control multiple means of inducing immunity to survivin, we are seeking to concurrently advance our science, diversify our portfolio and provide possible new revenue streams to shareholders."

On January 20, 2023 Duality Biologics ("DualityBio"), a clinical-stage biotech company focused on discovering and developing a pipeline of antibody-drug conjugates (ADCs) targeting cancers and autoimmune diseases, reported that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation (FTD) to DB-1303 for the treatment of patients with advanced, recurrent or metastatic endometrial carcinoma with HER2 overexpression who have progressed on or after standard systemic treatment (Press release, Duality Biologics, JAN 20, 2023, View Source [SID1234626423]). DB-1303 is a novel antibody-drug conjugate comprised of anti-HER2 monoclonal antibody, enzymatically cleavable peptide-linker, and a proprietary topoisomerase I inhibitor P1003. It is designed to have potent anti-tumor activity and bystander killing effect, high plasma stability, low free payload in circulation and wide therapeutic index.

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Dr. John Zhu, CEO of DualityBio, said: "The FDA’s decision to grant FTD underscores the potential for DB-1303 to address the unmet medical need and potentially serve as a new therapeutic option for patients with advanced, recurrent or metastatic endometrial carcinoma." "We are committed to advance this investigational drug to help those patients who are suffering from cancers. We will work closely with clinical investigators and health authorities to unlock the full potential of DB-1303 in patients with malignant tumors."

DualityBio is currently evaluating DB-1303 in an ongoing Phase I/IIa clinical trial for preliminary safety and efficacy in advanced/metastatic solid tumors with Her2 expression, including both HER2 positive and HER2 low patients.

About Fast Track Designation

Fast Track Designation is designed to facilitate the development and expedite the review of drugs to treat serious conditions and fulfill an unmet medical need, enabling drugs to reach patients earlier. Clinical programs with Fast Track designation may benefit from early and frequent communication with the FDA throughout the regulatory review process. These clinical programs may also be eligible to apply for Accelerated Approval and Priority Review if relevant criteria are met.

About Endometrial Carcinoma

Endometrial carcinoma (EC) is the most common cancer of the female genital tract in USA. On January 1, 2019, there were an estimated 822,388 people living with uterine corpus cancer in the U.S. (View Source), and it is estimated that 66200 new uterine corpus cancer cases will occur with 13030 deaths resulting from the disease in 2022. Around 1 in 4 women with endometrial cancer may experience a recurrence or be diagnosed with advanced disease. In endometrial cancer, approximately 60%–70% of high-grade carcinomas appear to overexpress HER2. There is no accepted standard of care and limited therapeutic options for the patients with recurrent or metastatic EC who have failed previous platinum-based systemic therapy with or without immunotherapy. DualityBio is working to develop treatments for the disease to fill this significant unmet medical need.

About DB-1303

DB-1303, a 3rd generation HER2 ADC molecule built from Duality Biologics’ Proprietary DITAC platform, exhibited potent antitumor activity in both HER2 positive and HER2 low tumor models with superior efficacy, safety and expanded therapeutic window. Both preclinical data and preliminary clinical data from DB1303 suggest the potential of DB-1303 to address unmet medical needs in various HER2 expressing cancers.