On June 6, 2022 Bluestar Genomics, Inc., an early cancer detection company leading the development and commercialization of next-generation liquid biopsy tests initially focused on non-invasive detection of high-mortality cancers in high-risk patient populations through a standard blood draw, reported it has received Clinical Laboratory Improvement Amendments (CLIA) certification from the U.S. Department of Health and Human Services’ Centers for Medicare and Medicaid Services (Press release, Bluestar Genomics, JUN 6, 2022, View Source [SID1234615663]).

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The certification confirms that Bluestar Genomics’ laboratory meets the federal regulations for clinical diagnostic testing, ensuring high quality and safety for patient testing. As part of the certification process, Bluestar Genomics recently completed analytical validation evaluating the accuracy and reliability of the company’s early detection test for pancreatic cancer. Top-line results showed significantly stronger performance than previously reported particularly in early stage (I/II) detection. Full data from the study will be published in a peer reviewed journal in the coming months.

"Securing CLIA certification is an important step for Bluestar Genomics. It underscores our pancreatic cancer test’s competitive performance, enables us to start clinical testing in our lab and allows us to move forward with larger clinical validation studies," said David Mullarkey, chief executive officer. "This certification, combined with our growing body of scientific evidence, positions us to become the first cancer detection company to offer clinical epigenomic testing for early pancreatic cancer detection."

Currently, pancreatic cancer ranks as the third most deadly cancer with a combined five-year survival rate of only 5% to 10%. Research has shown that early detection in pancreatic cancer can increase patient survival nearly ten-fold.

On June 6, 2022 Enterome, a clinical stage biopharmaceutical company developing first-in-class immunomodulatory drugs based on its bacterial Mimicry drug discovery platform, reported that proof-of-concept immune response data and first clinical data from its Phase 1/2 clinical trial of EO2401 in combination with an immune checkpoint inhibitor (nivolumab, Opdivo) in patients with non-resectable adrenocortical carcinomas (ACC), treated with at least one line (but not more than two prior lines of systemic therapy), or without prior systemic therapy for advanced/metastatic disease (SPENCER trial, EOADR1-19, NCT04116658) (Press release, Enterome, JUN 6, 2022, View Source [SID1234615679]). The data1 were presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, on June 4, 2022 in Chicago and virtually.

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EO2401 is Enterome’s first-in-class off-the-shelf OncoMimics cancer immunotherapy. It combines three OncoMimics peptides that closely mimic IL13Ra2, BIRC5 and FOXM1, all of which are known driver antigens present on aggressive solid tumors. In addition, EO2401 contains a CD4 helper peptide UCP2. Enterome selected these OncoMimics peptides using its Mimicry platform, which applies best-in-class biocomputational tools and bioassays to identify novel therapeutics from its proprietary database of 20+ million bioactive gut microbiome peptides and proteins.

Key highlights from the EO2401 poster presentation covering the Phase 1/2 SPENCER trial were:

Proof-of-concept immune response data

Immune monitoring of Cohort 2a demonstrates the ability of microbiome-derived peptides to induce a strong Tc1- skewed CD8+ T cell response with strong cross-reactivity against human selected tumor associated antigens.

The level of the specific CD8+ T cell immune responses against the microbiome-derived peptides comprising EO2401 were also found to correlate with clinical outcome (objective responses and progression-free survival).

Promising clinical outcome in sub-group of patients

The combination of EO2401 plus nivolumab was evaluated:

in patients with previously treated ACC (Cohort 2a, n=26) and
in patients who received no prior systemic therapy for advanced/metastatic disease (Cohort 2b, n=7)
Cohort 2a – group of patients showing benefit in objective tumor responses and time to progression

Analysis of Cohort 2a identified patients with clearly different efficacy outcomes, with one group showing benefit in objective tumor responses and time to progression, and another group with short progression-free survival (PFS) and short survival.

To refine the population for expansion in a randomized Phase 2 trial, different laboratory and clinical parameters were investigated as possible selection criteria.

No correlation was found between clinical outcome and tumor mutational burden, MSI-status, PD-L1 expression, serum cytokines/chemokines, or hormonal levels. However, the study found that patients in Cohort 2a who fulfilled the criteria of having received prior mitotane treatment, ECOG ≤ 1, ACC primary diagnosis > 9 months, max lesion size ≤ 125 mm, ≤ 3 organs involved, reduced lymphocytes ≤ grade 1 (n=14), demonstrated a clinical benefit vs patients not fulfilling these criteria (n=12), as follows:

Objective response rates (ORR) – 28.6% (95% CI 8.4; 58.1) vs 0% (95% CI 0.0; 26.5)
Disease control rates (DCR) – 64.3% (95% CI 35.1; 87.8) vs 8.3% (95% CI 0.2; 38.5)
Median progression-fee survival (PFS) – 3.9 months (95% CI 1.9; 9.2) vs 1.6 months (95% CI 0.5; 1.9)
Median survival – 13.0 months (95% CI 11.3; not evaluated) vs 2.1 months (95% CI 1.5; 7.4)
Cohort 2b – further follow up to be conducted

The clinical responses to EO2401 plus nivolumab observed in patients in Cohort 2b showed no benefit over the results from all patients in Cohort 2a, and further follow-up will be conducted.

Safety

The combination of EO2401, administered sub-cutaneously with the adjuvant Montanide ISA 51 VG, with nivolumab was well tolerated. The safety profile was consistent with the profile of nivolumab monotherapy, except the addition of local administration site reactions (occurring in 21% of patients).

Dr. Eric Baudin, Associate Professor and Head of the Endocrine Oncology Unit at the Institut Gustave Roussy (Villejuif, France) commented, "There has been no significant innovation in the treatment of advanced adrenal tumors and patients with this rare group of diseases are desperately in need of new effective therapies especially at relapse after first-line therapy. If confirmed, EO2401 in combination with nivolumab, represents the most promising therapy of the last three decades in this ultra rare cancer. I am pleased to be participating in this trial and to move forward into a randomized Phase 2 trial in this underserved patient population."

Dr. Jan Fagerberg, Chief Medical Officer of Enterome, said, "The data presented at ASCO (Free ASCO Whitepaper) for EO2401 in combination with nivolumab are very encouraging, with strong immune responses correlating to clinical outcome in patients having been previously treated for adrenocortical carcinoma. We are also pleased to have been able to define a set of clinical selection criteria that will enable us to refine a patient population for expansion in a Phase 2 randomized trial. We are on track to start this new trial in the coming months and look forward to the results in due course."

EO2401 in recurrent glioblastoma

At ASCO (Free ASCO Whitepaper), and separately announced today, Enterome presented the first clinical data from its Phase 1/2 clinical trials of EO2401 in combination with nivolumab +/- bevacizumab for the treatment of patients with recurrent glioblastoma (the ROSALIE trial, EOGMB1-18). View press release here.

About SPENCER

The SPENCER trial (EOADR1-19, NCT04187404) is a multicenter, open-label, Phase 1/2 study assessing the safety, tolerability, immunogenicity and preliminary efficacy of EO2401 in combination with the immune checkpoint inhibitor nivolumab in patients with adrenal tumors (adrenocortical carcinoma [ACC] and metastatic pheochromocytoma/paraganglioma [MPP]). The trial is expected to enrol at least 100 patients at clinical sites in Europe and the US.

References

Baudin, E. et al. EO2401, a novel microbiome-derived therapeutic vaccine for patients with adrenocortical carcinoma (ACC): Preliminary results of the SPENCER study. J Clin Oncol 40, 2022 (suppl 16; abstr 4596) doi: 10.1200/JCO.2022.40.16_suppl.4596

On June 6, 2022 VBI Vaccines Inc. (Nasdaq: VBIV) (VBI), a biopharmaceutical company driven by immunology in the pursuit of powerful prevention and treatment of disease, reported that new tumor response and overall survival (OS) data from the ongoing Phase 2a study of VBI-1901, the Company’s cancer vaccine immunotherapeutic candidate, in recurrent glioblastoma (GBM) was presented at the 2022 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting on June 5 (Press release, VBI Vaccines, JUN 6, 2022, View Source [SID1234615614]). The expanded Phase 2a data were selected for presentation in both a poster session and a poster discussion session.

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"We continue to be motivated by the data seen in this Phase 2a study of VBI-1901 as we endeavor to provide new treatment options to patients with very few available to them," said David E. Anderson, Ph.D., VBI’s Chief Scientific Officer. "Considering the high mortality rate among GBM patients, particularly in the recurrent setting, median overall survival of approximately 13-15 months seen in our two study arms suggests an additional survival benefit of nearly six months in comparison with historical control data in the recurrent population after treatment with a monotherapy.1 Moreover, the correlation of tumor responses and clinical response benefit observed in tandem is very encouraging. We remain in close discussion with our study investigators and scientific advisors as we move toward the next stages of development in both the recurrent and frontline GBM settings, and look forward to advancing this development program as diligently as possible."

Key results from the ASCO (Free ASCO Whitepaper) poster presentation:

Data were collected as of May 9, 2022

VBI-1901 + GM-CSF Study Arm – High Dose Part A + Part B (n=16)

Two (2) partial tumor responses and five (5) stable disease seen across Part A and Part B
18-month overall survival (OS) of 25% (n=4/16)
Median OS (mOS) reached at 12.9 months, comparing favorably to 8-month mOS for standard-of-care1
One patient remains on protocol beyond two years, with a 93% tumor reduction relative to initiation of treatment at the beginning of the study – this reduction has been sustained for over 6 months
VBI-1901 + GSK’s AS01B Adjuvant System2 (n=10)

Five (5) stable disease observed
18-month overall survival (OS) of 40% (n=4/10)
Achieved mOS of 14.6 months, comparing favorably to 8-month mOS for standard-of-care1
With few effective treatment options available for recurrent GBM patients, historical control data have demonstrated OS to be ~60% at 6-months and ~30% at 12-months after treatment with a monotherapy.1

This dataset continues to build upon data first shared at ASCO (Free ASCO Whitepaper) 2021, which resulted in Fast Track Designation granted by the U.S. Food and Drug Administration (FDA) in June 2021, and then updated at the World Vaccine & Immunotherapy Congress in December 2021.

Next Steps for VBI-1901

Based on the data seen to date in the Phase 1/2a study in recurrent GBM patients, VBI expects to assess VBI-1901 in randomized, controlled clinical studies in both primary and recurrent GBM patients in the next phase of development:

Q3 2022: Expected initiation of next phase of development in recurrent GBM setting, aiming to expand the number of patients in the ongoing Phase 1/2a study and adding a control arm, with the potential for accelerated approval based on tumor response rates and improvement in overall survival
Q4 2022: Evaluation of VBI-1901 in the primary GBM setting expected to initiate as part of the Individualized Screening Trial of Innovative Glioblastoma Therapy (INSIGhT), a Phase 2 adaptive platform trial
To learn more about VBI’s ongoing Phase 1/2a study and the INSIGhT trial, visit clinicaltrials.gov (Respective Identifiers: NCT03382977 and NCT02977780).

About Fast Track Designation

The Fast Track program facilitates the expedited development and review of new drugs or biologics that are intended to: 1) treat serious or life-threatening conditions, and 2) demonstrate the potential to address unmet medical needs. A therapeutic that receives Fast Track Designation is eligible for some or all of the following: 1) more frequent meetings with FDA to discuss the development plan and data needed to support approval, 2) more frequent written communication from FDA relating to the design of the proposed clinical trials and use of biomarkers, 3) Accelerated Approval and Priority Review, if relevant criteria are met, and 4) Rolling Review, which means the company can submit completed sections of its Biologic License Application (BLA) or New Drug Application (NDA) for review by FDA, instead of waiting until all sections of the application are completed.

Fast Track Designation was granted to VBI-1901, adjuvanted with granulocyte macrophage colony-stimulating factor (GM-CSF), for the treatment of first-recurrent GBM.

About VBI-1901 and GBM

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. 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, 12,000 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.

On June 6, 2022 PTC Therapeutics, Inc. (NASDAQ: PTCT) reported that management will present a company overview at the William Blair 42nd Annual Growth Stock Conference on Thursday, June 9 at 2:20 p.m. ET (Press release, PTC Therapeutics, JUN 6, 2022, View Source [SID1234615631])

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The presentation will be webcast live on the Events and Presentations page under the Investor section of PTC Therapeutics’ website at View Source and will be archived for 30 days following the presentation. It is recommended that users connect to PTC’s website several minutes prior to the start of the webcast to ensure a timely connection.

On June 6, 2022 Eureka Therapeutics, Inc., a clinical-stage biotechnology company developing novel T cell therapies to treat solid tumors, reported the publication of a preclinical study in blood entitled "A TCR Mimic CAR T Cell Specific for NDC80 Is Broadly Reactive With Solid Tumors and Hematological Malignancies" (Press release, Eureka Therapeutics, JUN 6, 2022, View Source [SID1234615648]). The study was led by Dr. Cheng Liu, President & Chief Executive Officer of Eureka and Dr. Martin Klatt and Dr. David Scheinberg of Memorial Sloan Kettering Cancer Center (MSK).

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Chimeric antigen receptor (CAR) T cells represent a novel class of FDA-approved drugs with high efficacy against refractory B cell-derived malignancies. However, target identification and selection for CAR T cell therapy remains challenging as most cell surface proteins are not cancer-specific and therefore often not adaptable for CAR T cell therapy. In contrast, many intracellular proteins are highly tumor-specific and targetable after being presented on the cell surface by human leukocyte antigen (HLA) complexes in the form of peptide-MHC complexes. T cell receptor mimic (TCRm) antibodies can recognize peptide-MHC complexes similarly to TCRs, but with the versatility and applicability of an antibody.

In collaboration with MSK, Eureka scientists used its E-ALPHA platform to identify TCRm antibodies that target the NDC80-derived complex. NDC80 is an intracellular protein over-expressed in multiple tumor types such as ALL, AML, lymphoma, melanoma, mesothelioma, pancreatic and thyroid cancer. In the study, anti-NDC80 TCRm-redirected T cells demonstrated high specificity in recognizing and killing multiple cancer cell lines. Moreover, no toxicities to healthy leukocytes and hematopoietic stem cells were observed.

"TCR mimic antibodies can unlock the potential of T-cell therapies by targeting intracellular proteins that are presently not druggable by conventional antibodies or conventional CAR-T cells that target cell surface proteins," said Dr. Cheng Liu, Founder and CEO, Eureka Therapeutics. "We are excited by the study and look forward to seeing the expanded use of TCR mimic antibodies in other cancer types."