On November 20, 2023 Cofactor Genomics, the company bridging the precision medicine gap, reported successful validation of the OncoPrism immunotherapy predictive diagnostic assay, enabling physicians to use the test to help guide treatment decisions for patients with metastatic or recurrent squamous cell carcinoma of the head and neck (HNSCC) (Press release, Cofactor Genomics, NOV 20, 2023, View Source [SID1234637868]). The successful readout is announced on the heels of the yearly national PREDAPT Summit, where the participating institutions from across the country gathered and reviewed the study results. The assay was validated in independent HNSCC patient cohorts receiving anti-PD-1 inhibitors pembrolizumab and nivolumab both as a single agent and in combination with chemotherapy. This milestone clears the assay for nationwide clinical use in an interventional setting where doctors can now incorporate OncoPrism to help guide patient treatment in a landscape of multiple therapies, each benefiting a subset of the patient population.

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"This is a proud day for all those working tirelessly to bring precision medicine to immunotherapy and improve the outcome and quality of care for patients. We have learned not only from the study’s validation results but from physicians across the nation who have had significant input on the study and the OncoPrism assay these last 3 years," stated Jarret Glasscock, Cofactor’s CEO.

This validation announcement follows a publication earlier this year in the Journal of Cancer Research and Clinical Oncology, where Cofactor along with more than 20 participating institutions reported on OncoPrism’s capability to predict HNSCC patients’ response to anti-PD-1 immune checkpoint inhibitors. Cofactor’s broader work, on the technology’s ability to predict response to immune checkpoint inhibitors (ICI) across multiple cancers, was recognized by the Nature Publishing Group as the second most downloaded oncology paper of the year.

While the validation of OncoPrism in HNSCC patients was the focus of this week’s announcement, Cofactor’s national PREDAPT clinical trial is aimed at evaluating OncoPrism in 11 cancers including lung, head and neck, colorectal, breast, cervical, liver, kidney, esophageal and bladder. US healthcare systems supporting and participating in the PREDAPT trial collectively provide care to approximately 20% of the total US patient population.

On November 20, 2023 VBI Vaccines Inc. (Nasdaq: VBIV) (VBI), a biopharmaceutical company driven by immunology in the pursuit of powerful prevention and treatment of disease, reported that additional biomarker data from the Phase 1/2a study of VBI-1901, the Company’s cancer vaccine immunotherapeutic candidate, in recurrent glioblastoma (GBM) patients, were highlighted in a poster presentation at the 28th Annual Meeting and Education Day of the Society for Neuro-Oncology (SNO), held on Friday, November 17, 2023 (Press release, VBI Vaccines, NOV 20, 2023, View Source [SID1234637864]).

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New proof-of-mechanism data demonstrated that vaccination with VBI-1901 was associated with T-cell activation capable of trafficking across the blood-brain barrier to the tumor microenvironment, an area which is known to be highly immunosuppressive and difficult to infiltrate. The additional peripheral biomarker data assessed levels of C4G – a protein fragment produced when cytotoxic T-cells, or killer T-cells, migrate into and throughout the tumor microenvironment – high baseline levels of which have been associated with longer overall survival in studies conducted in metastatic melanoma patients [Prakash et al., 2014].1 After vaccination with VBI-1901 in the Phase 1/2a study in recurrent GBM patients, the data showed there were higher blood/plasma levels of C4G in patients who achieved partial tumor responses than in patients with stable or progressive disease. Additionally, higher levels of C4G after vaccination with VBI-1901 have been associated with longer progression-free survival.

"We are very pleased to share these significant mechanistic findings at SNO," said David E. Anderson, Ph.D., VBI’s Chief Scientific Officer. "The GBM tumor microenvironment is notoriously immunosuppressive and difficult to penetrate, which is why most treatment needs to be administered intratumorally, and why there are currently so few effective treatment options for patients. We believe that these data continue to enrich the potential for VBI-1901 to have meaningful impact in recurrent and primary GBM, and we look forward to data from the next phases of development in each setting."

Data Highlights from Phase 1/2a Study Data in Recurrent GBM Patients- VBI-1901 10µg + GM-CSF Study Arms

(n=16)

44% disease control rate achieved (n=7/16) – disease control rate is defined as stable disease (SD) + partial tumor response (PR) + complete tumor response (CR)
2 PRs were observed – 1 patient was on treatment for more than 28 months (2.33 years), surviving at least 40 months (3.33 years) as of August 1, 2023, with a maximum tumor reduction of 93% relative to baseline
5 additional patients demonstrated SD for a sustained period of time
All patients with a tumor response (PR or SD) (n=7/16) reached a minimum survival of 12 months
Median overall survival (mOS) was 12.9 months, comparing favorably to 8-month mOS for monotherapy standard-of-care2
Ongoing Phase 2b Study Design in Recurrent GBM Patients

Multi-center, randomized, controlled, open-label study in up to 60 patients with first recurrent GBM

Patients will be randomized in a 1:1 ratio across two study arms:
Intradermal VBI-1901 + GM-CSF: 10 µg dose every 4 weeks until clinical disease progression
Active comparator: monotherapy standard-of-care – either intravenous carmustine or oral lomustine, every 6 weeks until disease progression or intolerable toxicity
Endpoints include:
Safety and tolerability
Overall survival (OS) – median and overall
Tumor response rate (TRR)
Progression-free survival (PFS)
Immunologic responses
Reduction in corticosteroid use relative to baseline
Change in quality of life compared to baseline
The U.S. Food and Drug Administration (FDA) has considered demonstration of a statistically significant improvement in overall survival relative to a randomized control arm to be clinically significant and has recognized this as criteria to support the approval of new oncology drugs.3

For more information about the Phase 2b study, visit clinicaltrials.gov and reference trial identifier: NCT03382977.

About GBM and VBI-1901

Scientific literature suggests CMV infection is prevalent in multiple solid tumors, including glioblastoma (GBM). GBM is among the most common and aggressive malignant primary brain tumors in humans. In the U.S. alone, 14,000 new cases are diagnosed each year. The current standard of care for treating GBM is surgical resection, followed by radiation and chemotherapy. Even with aggressive treatment, GBM progresses rapidly and has a high mortality.

VBI-1901 is a novel cancer vaccine immunotherapeutic candidate developed using VBI’s enveloped virus-like particle (eVLP) technology to target two highly immunogenic cytomegalovirus (CMV) antigens, gB and pp65. The FDA has granted VBI-1901 Fast Track Designation and Orphan Drug Designation for the treatment of recurrent glioblastoma. These designations are intended to provide certain benefits to drug developers, including more frequent meetings with the FDA, and Accelerated Approval and Priority Review, if relevant criteria are met, among other benefits.

On November 20, 2023 Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultrarare genetic diseases, reported that management, will participating in two upcoming investor conferences (Press release, Ultragenyx Pharmaceutical, NOV 20, 2023, View Source [SID1234637862]).

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6th Annual Evercore ISI HealthCONx (Miami, FL)

Tuesday, November 28, 2023, management will host 1×1 meetings.

Piper Sandler 35th Annual Healthcare Conference (New York City, NY)

Wednesday, November 29, 2023, at 10:00am, Emil Kakkis, M.D., Ph.D., CEO and President, will participate in a fireside chat and host 1×1 meetings.
The live and archived webcast of the fireside chat will be accessible from the company’s website at View Source

On November 20, 2023 Tvardi Therapeutics, Inc. ("Tvardi"), a privately held, clinical-stage biopharmaceutical company focused on the development of STAT3 inhibitors, reported that the management team will present at the 35th Annual Piper Sandler Healthcare Conference on Tuesday, November 28, 2023, and will participate in one-on-one meetings with investors throughout the day, in New York, NY (Press release, Tvardi Therapeutics, NOV 20, 2023, View Source [SID1234637861]).

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Presentation Details:
Date: Tuesday, November 28, 2023
Time: 10:10 a.m.-10:30 a.m. EST
Location: Lotte New York Palace, 455 Madison Ave, New York, NY 10022

On November 20, 2023 TRACON Pharmaceuticals, Inc. (Nasdaq: TCON), a clinical stage biopharmaceutical company utilizing a cost-efficient, CRO-independent product development platform (PDP) to advance its pipeline of novel targeted cancer therapeutics and to partner with other life science companies, reported that it has licensed its proprietary PDP of CRO-independent clinical research to a clinical stage biotech company for a $3.0 million upfront payment (Press release, Tracon Pharmaceuticals, NOV 20, 2023, View Source [SID1234637860]).

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"We are excited to announce the first license of our PDP to a company that recognizes the value of internalizing its clinical operations to reap the benefits of CRO-independent clinical trial implementation that we enjoy at TRACON," said Charles Theuer, M.D., Ph.D., TRACON’s Chief Executive Officer. "We are now in a position to widely license our PDP to allow clinical stage biotechnology and pharmaceutical companies to transform their clinical operations with the expectation of potentially dramatic cost reductions and shorter clinical trial timelines."

Under the terms of the Agreement, TRACON granted a non-exclusive and non-transferable license of its PDP to the clinical stage biotech company for the design, conduct and administration of clinical trials and related research and development activities, including activities relating to regulatory filings, submissions and approvals. A licensee can integrate TRACON’s configuration documentation with a widely-used software package, enabling validation and qualification of the software package, in conjunction with TRACON’s standard operation procedure documents, policies, work instructions, and clinical operation templates.