On April 25, 2022 OncoC4, Inc., a clinical stage biopharma company developing novel immunotherapies for cancer, reported that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to ONC-392, the Company’s next-gen anti-CTLA-4 monoclonal antibody (mAb) that preserves the CTLA-4 immune checkpoint function, as a single agent for the treatment of patients with metastatic non-small cell lung carcinoma (NSCLC) who have had disease progression on prior anti-PD-(L)1 therapy (Press release, BioNTech, APR 25, 2022, View Source [SID1234655987]).

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"We are grateful for the Fast Track designation for ONC-392, which underscores the unmet need for new treatment options for patients with PD(L)-1-resistant NSCLC," said Yang Liu, PhD, co-founder, CEO and Chief Scientific Officer of OncoC4. "We look forward to more frequent interactions with the FDA to accelerate our clinical development path as we seek to bring this promising drug candidate to patients as expeditiously as possible. Our Phase 1b dose expansion study is ongoing for multiple indications, including PD(L)1-resistant NSCLC, with pivotal studies being planned in the near term."

Fast Track designation is intended to facilitate the development and expedite the review of new therapeutics for the treatment of serious or life-threatening conditions where there is an unmet medical need. The ONC-392 program may be eligible for more frequent meetings with the FDA to discuss the drug’s development plan ensuring collection of appropriate data needed to support approval, and more frequent written communications with the FDA regarding topics such as the design of the proposed clinical trials. Fast Track programs also have eligibility for rolling review as well as eligibility for Accelerated Approval and Priority Review if relevant criteria are met.

ONC-392 is currently in Phase 1 clinical testing to evaluate safety, pharmacokinetics, and efficacy as monotherapy and in combination with anti-PD-1 standard of care in advanced solid tumors and NSCLC (PRESERVE-001; NCT04140526). In recently completed Parts A and B of the study, OncoC4 observed promising clinical activity of ONC-392 in PD(L)1-resistant cancer, including NSCLC.

About NSCLC: Lung cancer, of which 84% are NSCLC, accounts for 25% of all cancer deaths, more than any other cancer, in the United States. Immunotherapy targeting PD(L)1 has been transformative for NSCLC in terms of lives saved. Unfortunately, most lung cancer remains resistant to PD(L)1-targeting therapy, and PD(L)1-resistant NSCLC is one of the biggest challenges for cancer immunotherapy and a huge unmet medical need. CTLA-4 is another clinically validated target although no CTLA-4-targeting drug has been approved as monotherapy for lung cancer.

About ONC-392: Based on decades of research led by OncoC4 co-founders, Drs. Yang Liu and Pan Zheng, OncoC4 is developing ONC-392, a nextgen anti-CTLA-4 mAb that most effectively and selectively eliminates regulatory T cells, a major culprit of immune evasion, in the tumor microenvironment. Unlike traditional anti-CTLA-4 antibodies, ONC-392 does not cause lysosomal degradation of CTLA-4 and thus preserves the immune tolerance checkpoint function of CTLA-4 in the rest of the body. As a result, ONC-392 has a much-improved therapeutic index compared to other CTLA-4-targeting drugs.

About the PRESERVE-001 Trial (NCT04140526): The trial is conducted to assess the safety, pharmacokinetics, and efficacy of ONC-392 as a single agent in advanced solid tumors and in combination with anti-PD(L)1 standard of care in Non- Small Cell Lung Cancer and other solid tumors. The study consists of three linked parts: Part A is a dose-finding rapid titration study of ONC-392 as a single agent in patients with advanced solid tumors of various histologies to define the recommended Phase II dose for ONC-392 monotherapy (RP2D-M); Part B is dose-finding for combination with standard dose of pembrolizumab (Part B) to define the Recommended Phase II dose for ONC-392 combination therapy (RP2D-C); and Part C comprises expansion cohorts of ONC-392 in monotherapy and in combination therapy with pembrolizumab to determine safety and initial efficacy.

On April 25, 2022 AI Therapeutics, Inc., an AI driven clinical-stage biopharmaceutical company developing novel therapeutics for rare diseases, reported the acquisition of EntreChem, S.L.’s investigational drug candidate EC-8042 (redesignated AIT-102), a novel targeted cancer therapy in development for rare pediatric and other cancers (Press release, EntreChem, APR 25, 2022, View Source [SID1234648560]).

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Despite recent advances in the understanding of tumor biology, treatment for many pediatric cancers remains limited to surgery, radiation and chemotherapy which often have poor outcomes and are associated with significant side effects. AIT-102 specifically targets the mutations responsible for the formation and progression of two notable pediatric tumors, rhabdoid tumors and Ewing sarcoma, and holds the potential to treat a broader family of tumors that share a common mechanism of mutated or dysregulated SWI/SNF activity.

"As a physician treating children with cancer on a daily basis, I am acutely aware of the need for more effective and less toxic therapies," said Patrick Grohar, M.D., Ph.D., Director of Translational Research with the Center of Childhood Cancer Research at Children’s Hospital of Philadelphia, a leader in the study and treatment of pediatric tumors. "For many tumor types, the biological culprit is well understood, but has proven difficult to target with pharmaceuticals. I have dedicated the last 15 years of my professional career to understanding the mechanism of tumor biology in children and finding new, targeted, therapies to treat these tumors. I am particularly impressed with the strong preclinical data from our studies of AIT-102 in rhabdoid tumors and Ewing sarcoma, and I believe that AIT-102 has the potential to transform the treatment of these cancers, as well as many others."

"AIT-102 is a powerful addition to AI Therapeutics’ rare disease treatment pipeline," said Brigette Roberts, M.D., Chief Executive Officer of AI Therapeutics. "Cancer is the leading cause of death by disease in children in the US today. With AIT-102, we hope to change these statistics. Beyond pediatrics, AIT-102 has the potential to treat up to 20% of cancers with altered SWI/SNF activity, either through mutation or dysregulation, including epithelioid sarcoma, synovial sarcoma, lung adenocarcinoma, colorectal cancer, and others. We are thrilled to have this opportunity to bring forward a promising new agent to help fight these tumors."

Financial terms of the transaction were not disclosed.

On 25 April 2022 Epitopea, a transatlantic cancer immunotherapeutics company and global leader in exploiting a new class of untapped tumour-specific antigens (TSAs), reported a $13.6M (£10.3M) seed investment from a transatlantic syndicate of top-tier life sciences investors, including Advent Life Sciences, CTI Life Sciences, Cambridge Innovation Capital (CIC) and Fonds de solidarité FTQ (Press release, Epitopea, APR 25, 2022, View Source [SID1234632198]). The seed round was also supported by Novateur Ventures and the Harrington Discovery Institute/University Health Holdings. The funding will leverage Epitopea’s ground-breaking CryptigenTM approach to create transformational immunotherapies that target broad cancer patient populations in both solid and haematological cancers.

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The company’s proprietary technology provides an innovative approach to identifying shared, aberrantly expressed tumour specific antigens, known as CryptigensTM, that it has exclusively licensed from the Université de Montréal (UdeM). The licensing deal was catalysed by IRICoR, a Canadian Centre of Excellence in Commercialization and Research, which supported and incubated the development of the technology. CryptigenTM TSAs are uniquely and broadly presented across cancer types, providing tumour-specific targets for immunotherapies that are predicted to kill malignant cells while sparing non-cancerous cells, potentially delivering significant therapeutic benefit across broad patient populations with minimal side effects.

The seed round funding will be used to build the company’s executive team, advance further research on this new class of antigens, and catalyse their translation into novel cancer immunotherapeutics, including therapeutic vaccines, cell therapies, and TCR-based biologics.

Dr Jon Moore, CEO, Epitopea and Operating Partner at Advent Life Sciences, said

"The outstanding work of Epitopea’s co-founders, Université de Montréal scientists Drs. Claude Perreault, and Pierre Thibault, has opened a tremendous and potentially transformative opportunity for future cancer patients. With this significant seed financing by a syndicate of world-class investors from Canada and the UK, we can start translating these discoveries into novel therapeutics. We will be guided by science, deploying the best modalities available to help cancer patients achieve durable benefits."

Dr Laurence Rulleau, Partner, CTI Life Sciences, said

"CTI is delighted to co-lead the seed round of Epitopea, which is the first investment from our third fund, CTI LSF III. We are convinced that Epitopea has the potential to fundamentally change the paradigm of how cancer patients can be treated with therapeutics vaccines and other immunotherapies to significantly improve their quality of life. Epitopea will explore a variety of early and longer-term value creation strategies including partnerships with third parties as well as developing its own therapeutic modalities based on its CryptigenTM TSAs."

On April 25, 2022 BioVaxys Technology Corp. (CSE: BIOV, FRA:5LB,OTCQB:BVAXF) ("BioVaxys" or "Company"), reported that it has entered into an agreement with the Deaconess Research Institute ("DRI") to supply BioVaxys with surgically debulked tumors from Stage III/Stage IV ovarian cancer patients undergoing treatment at Deaconess Health System ("Deaconess") (Press release, BioVaxys Technology, APR 25, 2022, View Source [SID1234614613]). DRI, based in Evansville, Indiana, is the clinical studies arm of Deaconess, a premier regional provider of health care services in the United States. Access to ovarian cancer tumor cells is a critical step enabling BioVaxys to validate the manufacturing process for BVX-0918, the Company’s autologous haptenized tumor cell vaccine for late-stage ovarian cancer.

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The standard of care for late-stage ovarian cancer often involves surgically debulking of the tumor mass. The debulked tumor cells will be used to test and validate the tumor collection protocol, cryopackaging, cryopreservation, and supply chain logistics for BVX-0918 bioproduction. Following shipment to BioElpida s.a. ("BioElpida"), the Company’s bioproduction partner in Lyon, France, the tumor cells will then be used for process testing and manufacturing "dry runs" of BVX-0918, a major step leading to the completion of Good Manufacturing Process ("GMP") production, a requirement for the planned Clinical Trial Application ("CTA") with the European Medicines Agency ("EMA"). BioVaxys, together with its EU partner, ProCare Health of Barcelona, Spain, is preparing to launch a Phase I clinical study for BVX-0918 later this year.

BioElpida developed various tests and validation procedures needed to support GMP manufacturing, such as sterility testing for transport, hapten fixation, and cryopreservation solutions, as well as antibody generation, bioburden screening of the haptens, and endotoxin assays; access to debulked tumor means that BioElpida will begin the final stages of the vaccine production protocol and GMP validation. BioVaxys and BioElpida have also designed and fabricated a specialized shipping package which would cryopreserve the tumor sample while in transit from any hospital site to the BioElpida site.

BioVaxys President & Chief Operating Officer Kenneth Kovan says, "Having complied with the regulatory oversight involved in obtaining waste tumor samples, BioVaxys is now able to provide BioElpida with the materials required for finalizing the vaccine production protocol and performing process validation in the lead up to our planned CTA submission to the European regulator."

BioVaxys’ vaccine platform is based on the established immunological concept that modifying surface proteins—whether they are viral or tumor—with haptens makes them more visible to the immune system. This process of haptenization "teaches" a patient’s immune system to recognize and make target proteins more "visible" as foreign, thereby stimulating a T-cell mediated immune response. BioVaxys’ cancer vaccines are created by extracting a patient’s own (autologous) cancer cells, chemically linking with a hapten, and re-injecting them into the patient to induce an immune response to proteins which are otherwise not immunogenic. Haptenization is a well-known and well-studied immunotherapeutic approach to cancer immunotherapy and has been clinically evaluated in both regional and disseminated metastatic tumors.

A first generation single-hapten vaccine invented by BioVaxys Co-Founder and Chief Medical Officer David Berd, MD, achieved positive immunological and clinical results in Phase I and Phase II human trials in over 600 patients with different tumor types, as well as having no observed toxicity in years of clinical study. These studies were conducted under an FDA-reviewed IND. A first generation autologous, haptenized vaccine was also tested by Dr. Berd in women with advanced ovarian cancer who had ceased to respond to conventional chemotherapy. The results were encouraging: In 24 patients, the median overall survival was 25.4 months with a range of 4.5-57.4 months; 8 patients survived for more than 2 years. BioVaxys has enhanced the first-generation approach by utilizing two haptens ("bi-haptenization"), which the Company believes will yield superior results.

On April 25, 2022 Shuwen Biotech, an integrated in vitro diagnostic company with focuses on women’s health, reported that it has obtained a CE mark for its quantitative qPCR kit for measuring ERBB2 gene expression in breast cancer tissue (CercaTestTM ERBB2 Assay) (Press release, Shuwen Biotech, APR 25, 2022, View Source [SID1234613090]).

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Immunohistochemistry (IHC) analysis of HER2 protein expression and fluorescent in situ hybridization (FISH) have been used to characterize HER2 status in breast cancer to guide anti-HER2 therapies. However, studies have shown that current standard qualitative HER2 IHC assay is insufficient especially in the low range (0 and 1+) of HER2 expression. In particular, a recent study showed the use of HER2 IHC assays as a companion diagnostic test for the new drug trastuzumab deruxtecan may result in misassignment of patients for treatment.1 In view of the direct link between gene amplification, RNA transcription, and protein expression, experts have suggested measures of messenger RNA as potentially a more reproducible quantitative assay.2

CercaTestTM ERBB2 Assay is a quantitative real-time PCR assay for accurate measurement of ERBB2 gene expression in breast cancer tissue slices. Contiguous quantitative PCR cycle numbers may be generated from different FFPE tissue samples with the same IHC scores (such as HER2 0 or 1+), allowing delineation of different ERBB2 mRNA expression levels in full spectrum. The assay is run on commonly available PCR platforms, thus obviating the need for pathology readings and scorings and avoiding human subjectivity and errors.

"With CercaTestTM ERBB2 Assay and MammaTyper, we have shown that qPCR analysis of ERBB2 mRNA in our assays exhibits high inter- and intra-laboratory reproducibility and good concordance with HER2 IHC scores by experienced pathologists, even in HER2 low expression samples.3, 4 Coupled with our RNXtract Nucleic Acid Extraction Kit and RNXtract Automated Purification System, CercaTestTM ERBB2 Assay provides simple, fast, reproducible and accurate assessment of ERBB2 mRNA levels in tissue samples including HER2-low breast cancer samples. We believe our CercaTestTM ERBB2 Assay and MammaTyper potentially offer superior companion diagnostics for anti-HER2 therapies especially the newer generation of anti-Her2 ADC drugs," commented Barclay Li, Chief Scientific Officer of Shuwen Biotech. Both CercaTestTM ERBB2 Assay and MammaTyper are commercially available worldwide through Cerca Biotech GmbH, a European subsidiary of Shuwen Biotech.

1. Fernandez et al., Examination of Low ERBB2 Protein Expression in Breast Cancer Tissue,
JAMA Oncol. 2022 Apr 1; 8(4):1-4. doi: 10.1001/jamaoncol.2021.7239.
2. Allison et al., ERBB2-Low Breast Cancer-Is It a Fact or Fiction, and Do We Have the Right Assay? JAMA Oncol. 2022 Apr 1; 8(4):610-611. doi: 10.1001/jamaoncol.2021.7082.
3. Wirtz et al., Biological Subtyping of Early Breast Cancer: A Study Comparing RT-qPCR with Immunohistochemistry. Breast Cancer Res Treat. 2016 May 24; 157:437–446. Doi: 10.1007/s10549-016-3835-7
4. Varga et al., An International Reproducibility Study Validating Quantitative Determination of ERBB2, ESR1, PGR, and MKI67 mRNA in Breast Cancer Using MammaTyper, Breast Cancer Res. 2017 May 11; 19(1):55. doi: 10.1186/s13058-017-0848-z.