On May 18, 2023 Processa Pharmaceuticals, Inc. (Nasdaq: PCSA) ("Processa" or the "Company") reported that it has received guidance from the U.S. Food and Drug Administration ("FDA") regarding the Company’s next trial for Next Generation Chemotherapy-Capecitabine ("NGC-Cap") (Press release, Processa Pharmaceuticals, MAY 18, 2023, View Source [SID1234631854]). The trial for NGC-Cap, the combination of PCS6422 and capecitabine, will be a Phase 2 safety-efficacy trial in colorectal cancer patients following the principles of FDA’s Project Optimus Oncology Initiative, the recent FDA recommendation on how oncology drugs are to be developed going forward.

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David Young, Pharm.D., Ph.D., Processa’s President and CEO, commented, "Our communications with the FDA have been extremely productive. One of the most important advantages of NGC-Cap and all our NGC drugs is that they have been designed to decrease the side effects associated with the treatment while increasing the exposure of cancer cells to proven cancer-killing molecules. These changes are expected to increase the number of patients who will benefit from each NGC drug given fewer side effects as well as have a significant impact on a patient’s response.

"By combining our NGCs and the Processa Regulatory Science Approach with FDA’s Project Optimus, Processa can efficiently provide better therapeutic options to cancer patients while increasing the likelihood of successful marketing approval. In addition, prior to approval, we expect to show that there are significant advantages to treating patients with our NGC drugs over both existing chemotherapy and oncology drugs directed toward new targets or new mechanisms. We look forward to continued collaboration with FDA and to providing continued updates to our shareholders," concluded Dr. Young.

The Phase 2 trial will be designed to determine the dose-and exposure-response relationships for both anti-tumor activity and safety/tolerability by evaluating different dosage regimens. The final dosage regimens to be used in the Phase 2 trial will be defined following the determination of the maximum tolerated dose from the Company’s ongoing Phase 1b trial and in collaboration with the FDA. To expedite the enrollment of the first patient, Processa has begun the upfront study preparation tasks, including protocol preparation for submission to the existing IND and clinical enrollment planning.

On May 18, 2023 PDS Biotechnology Corporation (Nasdaq: PDSB), a clinical-stage immunotherapy company developing a growing pipeline of targeted immunotherapies for cancer and infectious disease, reported the completion of enrollment in the immune checkpoint inhibitor naïve group (ICI naïve) of its VERSATILE-002 Phase 2 trial for the treatment of recurrent or metastatic human papillomavirus (HPV)16-positive head and neck cancer (Press release, PDS Biotechnology, MAY 18, 2023, View Source [SID1234631853]).

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VERSATILE-002 (NCT04260126) is a Phase 2, open-label, multicenter trial of the efficacy and safety of PDS0101 administered in combination with Merck’s anti-PD-1 therapy, KEYTRUDA (pembrolizumab), in adults with HPV16-positive, unresectable recurrent or metastatic head and neck squamous cell carcinoma (HNSCC). VERSATILE-002 is investigating two patient populations with HPV16-positive head and neck cancer patients whose cancer has returned or spread. The first group has not been previously treated with an ICI, also known as the ICI naïve cohort, and is PD-L1 positive. The second group of patients has failed treatments, including ICI therapy (ICI refractory). In December 2022, PDS Biotech announced the completion of enrollment in the first stage of the ICI refractory group.

PDS Biotech recently announced that updated data from the ICI naïve group will be the subject of a poster presentation at the 2023 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting. The abstract was also selected as one of the featured posters to be reviewed by an expert panel in the Head and Neck Cancer discussion session. The data will build upon previously announced preliminary efficacy data reported at ASCO (Free ASCO Whitepaper) 2022 from 17 ICI naïve VERSATILE-002 patients, which demonstrated an objective response rate of 41% (confirmed and unconfirmed responses), clinical benefit rate of 77%, and an overall survival rate of 87% at nine months.

"Completing enrollment in the ICI naïve arm is an important milestone in the VERSATILE-002 Phase 2 trial and the ongoing development of PDS0101 in combination with KEYTRUDA as a potential treatment for recurrent and/or metastatic HPV16-positive head and neck cancer," said Dr. Lauren V. Wood, Chief Medical Officer of PDS Biotech. "HPV-driven HNSCC is a growing problem, and there is a large unmet medical need to develop an HPV-targeted immunotherapy. Preliminary data reported at ASCO (Free ASCO Whitepaper) 2022 and highlighted at our October 2022 Head and Neck Cancer KOL Roundtable suggest that PDS0101 in combination with KEYTRUDA may lead to improved outcomes in ICI naïve, recurrent or metastatic HNSCC patients. We now look forward to reporting updated data from the VERSATILE-002 trial at ASCO (Free ASCO Whitepaper) 2023 as the next step towards a planned global confirmatory randomized, controlled trial investigating the combination of PDS0101 and KEYTRUDA in this same patient population."

90% of HPV-associated head and neck cancers in the U.S. are reported to be caused by HPV16, as reported in a study published in the Journal of Clinical Medicine (J Clin Med 2018 Sep;7(9):241). The U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to the combination of PDS0101 and KEYTRUDA for the treatment of HPV16-positive HNSCC.

About PDS0101

PDS0101, PDS Biotech’s lead candidate, is a novel investigational human papillomavirus (HPV)-targeted immunotherapy that stimulates a potent targeted T cell attack against HPV-positive cancers. PDS0101 is given by subcutaneous injection alone or in combination with other immunotherapies and cancer treatments. In a Phase 1 study of PDS0101 in monotherapy, the treatment demonstrated the ability to generate multifunctional HPV16-targeted CD8 and CD4 T cells with minimal toxicity. Interim data suggests PDS0101 generates clinically effective immune responses, and the combination of PDS0101 with other treatments can demonstrate significant disease control by reducing or shrinking tumors, delaying disease progression and/or prolonging survival. The combination of PDS0101 with other treatments does not appear to compound the toxicity of other agents.

About VERSATILE-002

VERSATILE-002 is a single-arm Phase 2 trial evaluating the safety and efficacy of PDS0101, an HPV16-targeted investigational T cell-activating immunotherapy that leverages PDS Biotech’s proprietary Versamune technology, in combination with Merck’s anti-PD-1 therapy, KEYTRUDA (pembrolizumab). The combination is being evaluated in immune checkpoint inhibitor (ICI)-naïve and ICI-refractory patients with recurrent/metastatic HPV16-positive head and neck squamous cell carcinoma (HNSCC) and was granted Fast Track designation by the Food and Drug Administration in June 2022.

Preliminary efficacy and safety data were presented at the 2022 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting for ICI naïve patients (PR link). Preliminary data from the first 19 patients demonstrated that 77% of the patients with available imaging (17 of 19) had either disease stabilization or tumor shrinkage. Additionally, the overall survival rate of these patients at 9 months was 87%.

On May 18, 2023 Orna Therapeutics, a biotechnology company pioneering a new investigational class of engineered circular RNA (oRNA) therapies, presented data on the progress of lead program ORN-101, a development-stage in situ CAR program, at the Protein Engineering Summit (PEGS) and at the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) Annual Meeting in Los Angeles (Press release, OrnaTherapeutics, MAY 18, 2023, View Source [SID1234631852]).

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The presentations include a session and talk at PEGS, as well as a poster presentation at ASGCT (Free ASGCT Whitepaper), which may be viewed on the Orna website here.

"Using the Orna FoRCE platform, we’ve been able to develop ORN-101, a potent anti-cancer circular RNA therapy delivered via LNP," said Robert Mabry, PhD, Chief Scientific Officer at Orna. "We have achieved 20-fold lower dosing in animal models compared to earlier versions of the product, allowing us the flexibility to administer multiple doses of our off-the-shelf immunotherapy – something that could allow us to treat more patients quickly and easily."

Synthetic Circular RNA as a New Therapeutic Modality

Elevated and durable protein expression – two features where mRNA falls short – are maximized with circular RNA in part due to Orna’s discovery of novel internal ribosome entry site (IRES) elements necessary for translation. Compared to previously known IRES elements, Orna’s library of IRES elements can be used to drive higher protein expression in desired cell type targets. oRNA, because of its shape, is more resistant to quick degradation in the body, is easier to manufacture and formulate in the lab, and is immunoquiescent. These desirable features further allow oRNA therapeutics to be developed for applications beyond infectious disease, including oncology and genetic disorders.

In situ CAR Therapy Using oRNA

Over the past four years, Orna has worked to develop a therapeutic class capable of delivering a chimeric antigen receptor (CAR) to immune cells within a patient, eliminating the need for lymphodepletion typically required for engineered cell therapies, while providing off-the-shelf redosability in an autologous setting. ORN-101 combines oRNA and a proprietary LNP, and these latest data show ORN-101’s tumor eradication ability after 2-3 doses in a mouse model, effective at 10-20-fold lower doses than Orna has previously reported. ORN-101 features high expression of the CAR driven by an optimized IRES element, selected by Orna’s FoRCE platform to yield durable protein expression.

About ORN-101:

ORN-101, Orna’s lead program, is a development-stage in situ CAR therapy designed to modify a patient’s immune cells inside their body. Comprising an oRNA molecule packaged inside a proprietary lipid nanoparticle (LNP) formulation, this easily redosable format could avoid patient lymphodepletion and allow for reliable dose control, overcoming barriers of existing autologous ex vivo CAR-T therapies without sacrificing efficacy. Preclinical data demonstrates tumor suppression and eradication in animal models, suggesting the possibility that oRNA-LNP based cancer therapies could disrupt traditional CAR-T cellular therapies.

On May 18, 2023 Biodexa Pharmaceuticals PLC (NASDAQ: BDRX) ("Biodexa" or the "Company"), a clinical stage biopharmaceutical company developing a pipeline of products aimed at primary and metastatic cancers of the brain is pleased to announce that it has signed non-binding letter of intent to potentially acquire Varian Biopharmaceuticals, Inc. ("VarianBio"), a private US precision oncology company developing novel therapeutics for the treatment of cancer (Press release, Midatech Pharma, MAY 18, 2023, View Source [SID1234631849]).

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Background to the proposed acquisition

In recent communications, the Company has referenced a shift in strategy from a drug delivery company to a therapeutics company. Currently, the Company’s only clinical asset is MTX110 which is being developed for three intractable rare and orphan brain cancers. The Company’s intention is to add to, and diversify, its development pipeline with a continued focus on rare and orphan products and/or oncology therapeutics and VarianBio represents the first of such opportunity.

On May 18, 2023 Micronoma reported that it is serving as the industry partner on a grant the NYU Grossman School of Medicine received from the National Cancer Institute to identify microbial and host genomic signatures that can be used with other methodologies to predict non-small cell lung cancer (NSCLC) in its early stages as well as the chance of post-surgical recurrence (Press release, Micronoma, MAY 18, 2023, https://www.micronoma.com/micronoma-teams-up-with-nyu-school-of-medicine-on-lung-cancer-research-grant-this-latest-collaboration-seeks-synergistic-methodologies-that-will-enable-the-earliest-best-predictions-of-lung-cancer/ [SID1234631848]).

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"Micronoma is thrilled to partner again with NYU researchers to advance the cause of using microbial biomarkers to diagnose cancer as early as possible as well as establish potential recurrence markers," said Micronoma CEO and co-founder Sandrine Miller-Montgomery. "Lung cancer remains the leading cancer killer and the development of non-invasive methods to identify it is key to improving patient outcomes."

"This work will build on our groundbreaking work as the first biotech company offering early cancer detection using OncobiotaLUNG, a microbiome-driven liquid biopsy platform," she added.

Micronoma and NYU Langone Health are currently collaborating to use blood samples to derive multi-omics, including circulating tumor microbial DNA associated with early lung cancer diagnosis, which the company should make commercially available under CLIA regulations later this year.

With this grant, NYU and Micronoma intend to continue identifying the leading microbial and host biomarkers that can be used to predict early-stage lung cancer diagnosis and prognosis and use cutting-edge bioinformatic mechanisms and potentially targeted molecular tools to pursue developing early-stage testing and prognosis.

"We are extremely excited to bring a novel use of microbial genomic signatures as a potential biomarker for early-stage lung cancer to the Early Detection Research Network (EDRN) multicenter consortium," said Leopoldo N. Segal, M.D., Associate Professor of Medicine. "This work has been made possible through the collaborative efforts between NYU Langone Health and Micronoma. This is a promising area of research that we expect to be important in our efforts to diagnose early-stage lung cancer and risk stratify them."