Veru Reports Record Fiscal 2021 Full-Year Financial Results

On December 2, 2021 Veru Inc. (NASDAQ: VERU), an oncology biopharmaceutical company with a focus on developing novel medicines for the management of breast and prostate cancer, reported that fiscal 2021 full-year net revenues increased 44% to $61 million and gross profit increased 56% to $48 million, achieving new historical highs (Press release, Veru, DEC 2, 2021, View Source [SID1234596401]).

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Fourth-Quarter Financial Highlights: Fiscal 2021 vs Fiscal 2020

Net revenues increased 33% to $15.6 million from $11.7 million
FC2 prescription net revenues climbed 55% to $13.6 million from $8.7 million
Gross profit rose 29% to $12.3 million from $9.6 million
Gross margin was 79% of net revenues compared with 81% of net revenues
Operating loss was $1.9 million versus $11.3 million. Operating loss in the fiscal 2020 period included a $14.1 million non-cash impairment charge.
Net loss was $4.3 million, or $0.05 per share, compared with $11.8 million, which included a non-cash impairment charge of $14.1 million related to intangible assets or $0.17 per share.
Full-Year Financial Highlights: Fiscal 2021 vs Fiscal 2020

Net revenues increased 44% to $61.3 million from $42.6 million, a record high when compared to any prior fiscal year
FC2 prescription net revenues climbed 71% to $46.5 million from $27.1 million
Gross profit rose 56% to $47.9 million from $30.8 million
Gross margin increased to 78% of net revenues from 72% of net revenues
Operating income was $13.0 million, which included an $18.4 million gain on the December 2020 sale of the PREBOOST business, compared with operating loss of $14.7 million, which included the $14.1 million non-cash impairment charge.
Net income, which included the gain on the sale of the PREBOOST business, was $7.4 million and diluted EPS was $0.09 compared with net loss of $19.0 million and diluted loss per share of $0.28, which included the non-cash impairment charge.
Balance Sheet Information

Cash and cash equivalents were $122.4 million as of September 30, 2021 versus $13.6 million as of September 30, 2020
Net accounts receivable were $8.8 million as of September 30, 2021 versus $5.2 million as of September 30, 2020
"Once again we’ve reported new historical highs for full fiscal year net revenues and gross profit based on the robust growth of our US FC2 prescription business," said Mitchell Steiner, M.D., Chairman, President and Chief Executive Officer of Veru Inc. "On a sobering note, US and global COVID-19 hospitalizations and deaths are on the rise again. The identification of the omicron variant and the possibility that the current vaccines and antibody drugs may not be as effective against this variant means that drugs to treat severe COVID-19 are desperately needed. The mechanism of drug action of sabizabulin is that it disrupts microtubule intracellular transport of the coronavirus, a process that will still be required by new variants or strains of COVID-19, including omicron, to cause infection. While there have been recent developments evaluating molnupiravir and PAXLOVID (PF-07321332; ritonavir) for the treatment of unhospitalized patients with mild to moderate COVID-19 at a relatively lower risk of dying, sabizabulin is being developed for hospitalized patients with a high risk of death. In our positive Phase 2 clinical study in hospitalized COVID-19 patients at risk for acute respiratory distress syndrome, sabizabulin treatment resulted in an 82% relative reduction in death compared to placebo. If our Phase 2 clinical results are replicated to any significant degree in our global Phase 3 clinical study, we believe sabizabulin would fill a significant current unmet medical need for hospitalized patients. In our global Phase 3 clinical study of sabizabulin we are enrolling 300 hospitalized patients with moderate to severe COVID-19 who are at high risk for Acute Respiratory Distress Syndrome. We expect to have clinical results in the first half of calendar 2022.

"These strong financial results have also enabled us to continue to advance our deep late clinical stage drug pipeline portfolio. We are heavily committed to developing our drug candidate assets in breast and prostate cancer. In our breast cancer program, we are addressing 3rd line treatment of ER+ metastatic breast cancer through two separate studies with patient populations depending on the AR expression in the breast cancer tissue. Targeting patients whose AR expression in breast cancer is ≥ 40% AR, we are enrolling the Phase 3 ARTEST clinical study to evaluate enobosarm monotherapy. Targeting patients whose AR breast cancer expression is <40%, we plan to conduct a Phase 2b clinical study of sabizabulin monotherapy. We are also moving enobosarm earlier in the treatment sequence to the 2nd line treatment of AR+ER+ metastatic breast cancer by targeting patients with AR breast cancer expression ≥ 40% in the Phase 3 ENABLAR-2 study. The Phase 3 ENABLAR-2 study will evaluate the efficacy and safety of enobosarm and CDK 4/6 inhibitor combination. Finally, for AR+ metastatic triple negative breast cancer, we plan to conduct a single arm Phase 2 clinical study evaluating the combination of enobosarm and sabizabulin treatment in patients who have progressed after receiving at least 2 systemic chemotherapies. Because of the importance of determining the patient’s AR status and based on the recommendation of FDA, we will develop a companion diagnostic AR test. We are pleased to be partnering with Roche/Ventana Diagnostics, a global oncology diagnostics company, who will develop and, if approved, commercialize a companion diagnostic AR test.

"In our prostate cancer program, we are enrolling our Phase 3 VERACITY clinical study of sabizabulin for metastatic castration and androgen receptor targeting agent resistant prostate cancer, but prior to IV chemotherapy. We are also enrolling our Phase 2 dose-finding clinical study of VERU-100, a novel long-acting GnRH antagonist 3-month depot injection formulation for androgen deprivation therapy for hormone sensitive advanced prostate cancer. Once completed, we will start the Phase 3 registration study.

"Finally, our Sexual Health division, led by robust FC2 sales, and if approved this month, the addition of ENTADFI sales expected in the first half of calendar year 2022, we will continue to have substantial resources to invest in our premium oncology drug pipeline line which is dedicated to addressing significant unmet medical needs for two of the most prevalent cancers, prostate and breast cancer. I am pleased and excited with Veru’s transformation into a premium oncology biopharmaceutical company seeking large market opportunities. The Company remains duty-bound during this persistent global pandemic to pursue this COVID-19 indication even though it is not the primary focus of the Company."

Pharmaceutical Pipeline Highlights:

Breast Cancer Program

Enobosarm, a Novel Oral Selective Androgen Receptor Targeted Agonist, for the 3rd Line Treatment of Androgen Receptor Positive (AR+), Estrogen Receptor Positive (ER+) and Human Epidermal Growth Factor Receptor 2 Negative (HER2-) Metastatic Breast Cancer with AR ≥ 40% – Phase 3 ARTEST Clinical Study- Enrolling.

Enobosarm is the first new class of targeted endocrine therapy in advanced breast cancer in decades. Enobosarm is an oral, new chemical entity, selective androgen receptor agonist that targets the androgen receptor, a tumor suppressor, in AR+ER+HER2- metastatic breast cancer without the unwanted masculinizing side effects. Enobosarm has extensive nonclinical and clinical experience having been evaluated in 25 separate clinical studies in over 2,000 patients including three Phase 2 clinical studies in advanced breast cancer involving more than 250 patients. In the two Phase 2 clinical studies conducted in women with AR+ER+HER2- metastatic breast cancer, enobosarm demonstrated significant antitumor efficacy in heavily pretreated cohorts that failed estrogen receptor targeting agents, chemotherapy, and/or CDK 4/6 inhibitors and was well tolerated with a favorable safety profile. We are enrolling the Phase 3 multicenter, international, open label, and randomized (1:1) ARTEST registration clinical trial design to evaluate the efficacy and safety of enobosarm monotherapy versus physician’s choice of either exemestane everolimus or a SERM as the active comparator for the treatment of AR+ ER+ HER2- metastatic breast cancer in approximately 210 patients with AR nuclei staining ≥40% in their breast cancer tissue who had tumor progression on a nonsteroidal aromatase inhibitor, fulvestrant, and a CDK4/6 inhibitor.

Sabizabulin, Novel Oral Cytoskeleton Disruptor Agent, Monotherapy for the 3rd Line Treatment of AR+ER+HER2- Metastatic Breast Cancer with AR< 40% – Phase 2b Clinical Study.

We intend to conduct a Phase 2b clinical study of sabizabulin for the treatment of AR+ ER+ HER2- metastatic breast cancer in patients with an AR nuclei staining <40%. The Phase 2b clinical study will be an open label, multicenter, and randomized (1:1) study evaluating the efficacy and safety of sabizabulin 32mg monotherapy versus active comparator (exemestane ± everolimus or a SERM, physician’s choice) for the treatment of ER+ HER2- metastatic breast cancer in approximately 200 patients with AR nuclei staining <40% in their breast cancer tissue who had tumor progression on a nonsteroidal aromatase inhibitor, fulvestrant, and a CDK4/6 inhibitor. The Phase 2b study is expected to commence in calendar Q1 2022.

Enobosarm and Abemaciclib, CDK 4/6 Inhibitor, Combination Therapy for the 2nd Line Treatment of AR+ER+HER2- Metastatic Breast Cancer with AR ≥ 40% – Phase 3 ENABLAR-2 Clinical Study.

CDK 4/6 inhibitor and estrogen blocking agent combination has become first line therapy for patients with ER+HER2- advanced breast cancer. Unfortunately, almost all patients will develop resistance to CDK 4/6 inhibitors, and will eventually, have breast cancer progression. Based on positive Phase 2 clinical data and the preclinical data supporting the use of enobosarm in combination with a CDK 4/6 inhibitor in patients that are CDK 4/6 inhibitor and estrogen blocking agent resistant, we plan to conduct a Phase 3 multicenter, open label, randomized (1:1), active control clinical study, named ENABLAR-2 to evaluate the efficacy and safety of enobosarm plus abemaciclib combination therapy versus an alternative estrogen blocking agent (fulvestrant or an aromatase inhibitor) in subjects with AR+ ER+ HER2- metastatic breast cancer who have failed first line palbociclib (a CDK 4/6 inhibitor) plus an estrogen blocking agent (non-steroidal aromatase inhibitor or fulvestrant) and have an AR nuclei staining ≥ 40% in their breast cancer tissue. We plan to enroll approximately 186 subjects in this Phase 3 clinical study which is expected to commence during the first quarter of calendar year 2022.

Sabizabulin and Enobosarm Combination Therapy for AR+ Metastatic Triple Negative Breast Cancer Patients who have Progressed After Receiving at Least Two Systemic Chemotherapies – Phase 2 Clinical Study.

Sabizabulin is an oral, first-in-class, new chemical entity that targets and inhibits microtubules to disrupt the cytoskeleton. Sabizabulin is not a substrate for P-glycoprotein drug resistance protein. Over expression of P-glycoprotein is a common mechanism that results in taxane and chemotherapy resistance in metastatic triple negative breast cancer. Preclinical studies in human triple negative breast cancer grown in animal models demonstrate that sabizabulin significantly inhibits cancer proliferation, migration, metastases, and invasion of triple negative breast cancer tumors that have become resistant to paclitaxel (taxane). Furthermore, an enobosarm + pembrolizumab combination Phase 2 study in 18 heavily pretreated women with AR+ metastatic triple negative breast cancer demonstrated that enobosarm was well tolerated and resulted in promising preliminary efficacy of 25% clinical benefit rate (CR+PR+SD) at 16 weeks and objective tumor responses (1 CR and 1 PR). Thus, the combination of two oral agents, sabizabulin + enobosarm, may provide a new treatment option for women who have AR+ metastatic triple negative breast cancer. We intend to conduct a single arm, sabizabulin plus enobosarm combination therapy Phase 2 clinical study in approximately 111 women in calendar Q1 2022.

Companion Diagnostic AR Test
We have identified that patients who have greater than 40% androgen receptor nuclei staining in their breast cancer tissue are most likely to respond to enobosarm. Based on the recommendation of FDA to have a companion diagnostic test to determine the patient’s AR status, we are partnering with Roche/Ventana Diagnostics, a global oncology diagnostics company, who will develop and, if approved, commercialize a companion diagnostic AR test. The companion diagnostic test will be developed in parallel with the Phase 3 ARTEST clinical study.

Prostate Cancer Program

Sabizabulin, a Novel Oral Androgen Receptor Transport Disruptor, for the Treatment of Metastatic Castration and Androgen Receptor Targeting Agent Resistant Prostate Cancer – Phase 3 VERACITY Clinical Study – Enrolling.

Sabizabulin is a novel oral new chemical entity that targets microtubules in the cytoskeleton to disrupt androgen receptor transport. In June, the Company initiated the open label, randomized (2:1), multicenter Phase 3 VERACITY clinical study evaluating sabizabulin 32mg versus an alternative androgen receptor targeting agent for the treatment of chemotherapy naïve men with metastatic castration resistant prostate cancer who have failed at least one androgen receptor targeting agent. The primary endpoint is radiographic progression free survival. The Phase 3 VERACITY clinical study is expected to enroll approximately 245 patients from 45 clinical centers.

VERU-100, a Novel Proprietary Long-Acting Gonadotropin-Releasing Hormone (GnRH) Antagonist Peptide 3-Month Subcutaneous Depot Formulation, for Androgen Deprivation Therapy of Advanced Prostate Cancer – Phase 2 Clinical Study – Enrolling.

VERU-100 formulation is designed to address the current limitations of commercially available androgen deprivation therapy. Androgen deprivation therapy is currently the mainstay of advanced prostate cancer treatment and is used as a foundation of treatment throughout the course of the disease even as other endocrine, chemotherapy, or radiation treatments are added or stopped. Specifically, VERU-100 is a chronic, long-acting GnRH antagonist peptide administered as a small volume, three-month depot subcutaneous injection without a loading dose. VERU-100 immediately suppresses testosterone with no testosterone surge upon initial or repeated administration, a problem that occurs with currently approved luteinizing hormone-releasing hormone agonists used for androgen deprivation therapy. There are no GnRH antagonist depot injectable formulations commercially approved beyond a one-month injection. In June, the Company initiated the Phase 2 dose finding clinical study of VERU-100 androgen deprivation therapy for hormone sensitive advanced prostate cancer. The Phase 2 VERU-100 clinical study is expected to enroll approximately 35 patients. A Phase 3 registration clinical study has been agreed upon with FDA and will enroll approximately 100 men. The Phase 3 clinical study is anticipated to begin in 1H calendar 2022.

COVID-19 Program

Sabizabulin for the Treatment of Hospitalized COVID-19 Patients at High Risk for Acute Respiratory Distress Syndrome (ARDS) Phase 3 Clinical Study- Enrolling.

Sabizabulin has both broad anti-viral and anti-inflammatory activities which may serve a two-pronged approach to the treatment of COVID-19 virus infection and the subsequent debilitating inflammatory effects that lead to ARDS and death. In May, we initiated the Phase 3 clinical study which is a double-blind, multicenter, multinational, randomized (2:1), placebo-controlled study evaluating daily oral doses of 9mg sabizabulin for up to 21 days versus placebo in 300 hospitalized COVID-19 patients (200 subjects will be treated with sabizabulin and 100 subjects will receive standard of care) who are at high risk for ARDS. The primary efficacy endpoint will be proportion of patients that die on study up to Day 60. Secondary endpoints will include the proportion of patients without respiratory failure, days in ICU, WHO Ordinal Scale for Clinical Improvement change from baseline, days on mechanical ventilations, days in the hospital, and viral load. The study is being conducted in the US, Brazil, Argentina, Mexico, Colombia and Bulgaria.

Sexual Health Division

ENTADFI (Tadalafil 5mg and Finasteride 5mg Capsule) for the Treatment of Benign Prostatic Hyperplasia (BPH) – PDUFA Date December 2021.

ENTADFI (tadalafil 5mg and finasteride 5mg combination capsule) was developed to treat urinary tract symptoms caused by BPH without adverse sexual side effects. The co-administration of tadalafil and finasteride has been shown to be more effective for the treatment of BPH than finasteride alone without causing erectile dysfunction. The PDUFA date is scheduled for December 2021. If approved, ENTADFI is expected to be marketed and distributed by our own "direct to patient" telemedicine and telepharmacy platform. We have also partnered with GoodRx, America’s digital resource for healthcare, to reach their almost 20 million monthly visitors, which include both consumers and healthcare providers, and offer a unique cash price to ensure our treatment is more affordable and accessible. We will augment our marketing and sales efforts by seeking partners in the US and ex-US. We expect to begin commercialization in the first half of calendar year 2022.

Event Details
Interested parties may access the call by dialing 1-800-341-1602 from the U.S. or 1-412-902-6706 from outside the U.S. and asking to be joined into the Veru Inc. call. The call will also be available through a live, listen-only audio broadcast via the Internet at www.verupharma.com. Listeners are encouraged to visit the website at least 10 minutes prior to the start of the scheduled presentation to register, download and install any necessary software. A playback of the call will be archived and accessible on the same website for at least three months. A telephonic replay of the conference call will be available, beginning the same day at approximately 12 p.m. (noon) ET by dialing 1-877-344-7529 for U.S. callers, or 1-412-317-0088 from outside the U.S., passcode 10161217, for one week.

InnoCare Announces Inclusion of Orelabrutinib in China National Reimbursement Drug List

On December 2, 2021 InnoCare Pharma (HKEX: 09969), a commercial-stage biotech company, reported that its BTK inhibitor orelabrutinib has been included in the updated National Reimbursement Drug List (NRDL) by the China National Healthcare Security Administration (NHSA) (Press release, InnoCare Pharma, DEC 2, 2021, View Source [SID1234596419]). Orelabrutinib is an oral, once-daily BTK inhibitor with national Category 1 designation. It is included in the NDRL as the treatment of patients with r/r Chronic Lymphocytic Leukemia (CLL) /Small Lymphocytic Lymphoma (SLL), and the treatment of patients with r/r Mantle Cell Lymphoma (MCL).

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"Lymphoma is one of the malignant tumors with the fastest growth of incidence rate and one of the top ten malignant tumors with the highest mortality rate in China. The inclusion of orelabrutinib in the NRDL could significantly improve access of this innovative drug in China, which can benefit more patients," said Dr. Jasmine Cui, the co-founder, chairwoman and CEO of InnoCare.

"We are grateful that orelabrutinib has been included in the NRDL. We will continue to fulfil our mission — ‘Science drives innovation for the benefit of patients’, and accelerate exploring more indications for orelabrutinib," said Dr. Cui.

Orelabrutinib has excellent target selectivity with near 100% BTK occupancy. Orelabrutinib’s once-daily oral administration is convenient for continued treatment.

About Orelabrutinib
Orelabrutinib is a small molecule Bruton’s tyrosine kinase inhibitor (BTKi) developed for the treatment of cancer and autoimmune diseases.

Orelabrutinib had received approval on Dec. 25, 2020 from the China National Medical Products Administration (NMPA) in two indications: the treatment of patients with r/r Chronic Lymphocytic Leukemia (CLL) /Small Lymphocytic Lymphoma (SLL), and the treatment of patients with r/r Mantle Cell Lymphoma (MCL). In addition, multi-center, multi-indication clinical trials are underway in the US and China for orelabrutinib as monotherapy or in combination therapies for the treatment of Marginal Zone Lymphoma (MZL), Central Nervous System Lymphoma (CNSL), Waldenstrom’s Macroglobulinemia (WM), Diffuse large B-cell lymphoma (DLBCL), etc.

Orelabrutinib was granted as Breakthrough Therapy Designation for the treatment of r/r MCL by U.S. Food and Drug Administration (FDA).

Attributed to its excellent selectivity and clinical safety profiles, orelabrutinib is also evaluated in the global phase II studies for the treatment of Multiple Sclerosis (MS), phase II clinical trials for the treatment of Systemic Lupus Erythematosus (SLE) and Primary Immune Thrombocytopenia (ITP) in China.

Quest Diagnostics Now Offers Biocept’s Liquid Biopsy Test for Lung Cancer, Expanding its Menu of Advanced Cancer Diagnostics

On December 1, 2021 Physicians in the United States can now order the liquid biopsy-based Target Selector NGS Lung Panel test directly from Quest Diagnostics (NYSE: DGX), the nation’s leading provider of diagnostic information services (Press release, Quest Diagnostics, DEC 1, 2021, View Source,-Expanding-its-Menu-of-Advanced-Cancer-Diagnostics [SID1234596323]). Developed by Biocept, Inc. (Nasdaq: BIOC), a leading provider of molecular diagnostic assays, products and services, the lab-developed liquid biopsy test aids genomic profiling in patients with advanced non-small cell lung cancer (NSCLC), helping physicians identify potential targeted therapies and monitor the effectiveness of treatment .

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With a physician’s order, individuals are now able to provide a blood specimen for testing at one of Quest’s 2,200 conveniently located patient service centers across the United States. Collected specimens will be forwarded to Biocept’s CLIA-certified, CAP-accredited laboratory in San Diego for testing.

The Target Selector NGS Lung Panel contributes to better patient outcomes by providing physicians with direction on therapeutic options for those battling NSCLC. Healthcare providers may perform genomic profiling of tissue to aid their selection and response monitoring of gene-targeted NSCLC therapies. However, patients are not always able to have a tissue biopsy, which can take weeks to produce a result and may be limited by the quality and quantity of tissue that can be collected. Liquid biopsies identify genetic material from tumor cells that are shed and circulate in the blood stream, complementing insights from tissue biopsies and reducing dependence on tissue.

The addition of this blood-based panel to Quest’s advanced diagnostic testing menu enables Quest to provide actionable insights in an easier and less invasive manner, supplementing its services in tissue biopsy.

"By providing Biocept’s Target Selector NGS Lung Panel test under our advanced diagnostics portfolio, Quest will enable more physicians and patients to receive insights necessary to provide the appropriate treatment for NSCLC," said Kristie Dolan, General Manager, Oncology Franchise, Quest Diagnostics. "Our relationship with Biocept is the latest example of how Quest’s scale and expertise helps innovators reach larger markets and serve patients most in need."

"Our collaboration with Quest Diagnostics reflects our shared commitment to excellence in oncology diagnostics and allows us to serve more patients nationwide and expand our customer base, with the goal of improving care for those with lung cancer," said Michael Nall, President and CEO, Biocept. "The Target Selector NGS Lung Panel provides critical information to aid in the creation of more personalized treatment plans for patients with NSCLC and help oncologists get the answers they need to provide the best care possible."

Biocept’s multi-gene, tumor-specific NGS Lung Panel allows physicians and researchers to use a simple blood sample to analyze actionable biomarkers associated with specific solid tumor types. It combines Biocept’s liquid biopsy biomarker testing expertise with next generation sequencing and decision support resources. The biomarkers included in the Target Selector NGS Lung Panel are those that physicians frequently rely upon when making treatment decisions for their patients diagnosed with NSCLC. The assay is targeted and actionable—the majority of the biomarkers investigated are based on National Comprehensive Cancer Network guidelines and/or FDA-approved therapies.

Quest Diagnostics is the world’s leading provider of cancer diagnostics, providing physicians and health systems a single source solution via the most comprehensive capabilities in anatomic pathology and molecular diagnostics. Quest’s innovative test menu includes various options for screening, diagnosis, treatment and monitoring recurrence. The company provides a range of pathology services through its AmeriPath and Dermpath specialty businesses.

Surface Oncology Announces FDA Clearance of IND Application for GSK4381562, a Novel Antibody Targeting PVRIG

On December 1, 2021 Surface Oncology (Nasdaq: SURF), a clinical-stage immuno-oncology company developing next-generation immunotherapies that target the tumor microenvironment, reported that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug Application (IND) for GSK4381562 (formerly SRF813) to proceed into a first-in-human clinical trial (Press release, Surface Oncology, DEC 1, 2021, View Source [SID1234596341]). GSK4381562 is a fully human IgG1 antibody targeting PVRIG (also known as CD112R), an inhibitory protein expressed on natural killer cells (NK cells) and T cells.

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In December 2020, Surface announced it had entered into an agreement in which GSK exclusively licensed worldwide development and commercial rights to GSK4381562. Surface will receive a $30 million milestone payment in conjunction with the initiation of the first Phase 1 study for GSK4381562 and is eligible to receive an additional $700 million in future milestone payments, as well as be eligible to receive tiered royalties on global net sales.

"GSK4381562 is a differentiated anti-PVRIG antibody and represents a new therapeutic approach designed to enhance NK and T cell activation in the tumor microenvironment," said Rob Ross, M.D., chief executive officer. "We are pleased to have reached this milestone with our partners at GSK and look forward to continued advancement of this promising program in the clinic."

About GSK4381562:

GSK4381562 is a fully human, IgG1 antibody targeting PVRIG (also known as CD112R), an inhibitory protein expressed on natural killer cells (NK cells) and T cells. GSK4381562 binds to a distinct epitope on PVRIG and blocks the interaction of PVRIG with CD112, its binding partner that is overexpressed on tumor cells. Preclinically, GSK4381562 promotes the activation of both NK cells and T cells, with the potential to elicit a strong anti-tumor response and promote immunological memory.

VBI Vaccines to Present New Overall Survival Data from Phase 2a Study in Recurrent GBM at the World Vaccine & Immunotherapy Congress 2021

On December 1, 2021 VBI Vaccines Inc. (Nasdaq: VBIV) (VBI), a biopharmaceutical company driven by immunology in the pursuit of powerful prevention and treatment of disease, reported that David E. Anderson, Ph.D., VBI’s Chief Scientific Officer, will present updated 12-month and 18-month overall survival (OS) data from the Phase 2a study investigating VBI-1901, the Company’s cancer vaccine immunotherapeutic candidate targeting recurrent glioblastoma (GBM), at the World Vaccine & Immunotherapy Congress at 3:10 PM PT / 6:10 PM ET on December 1, 2021 (Press release, VBI Vaccines, DEC 1, 2021, View Source [SID1234596359]).

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Building on the data announced in June 2021, which supported the U.S. Food and Drug Administration (FDA) granting VBI-1901 Fast Track Designation for the treatment of recurrent GBM patients with first tumor recurrence, the updated data highlighted in Dr. Anderson’s presentation include:

Study arm 1 : VBI-1901 + granulocyte-macrophage colony-stimulating factor (GM-CSF)
18-month OS : 30% (n=3/10)
One patient remains on protocol past week 86, with a 93% tumor reduction relative to initiation of treatment at the beginning of the study
Two additional patients achieved OS of at least 20 months, but are no longer on protocol
Study arm 2 : VBI-1901 + GSK’s AS01 adjuvant system2
12-month OS : 70% (n=7/10)
18-month OS not yet reached
With few options 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 monotherapy1.

"Data from the Phase 1/2a study have shown encouraging efficacy signals, including durable tumor responses and improvements in overall survival compared to historical controls, with a promising tolerability profile," said Patrick Y. Wen, M.D., Director of the Center for Neuro-Oncology at Dana-Farber Cancer Institute, Professor of Neurology at Harvard Medical School, and principal investigator of the INSIGhT trial. "These results merit further investigation especially as GBM patients have very limited treatment options, signaling a critical need for innovative new therapies to be assessed in the clinic. We will continue to evaluate VBI-1901 in the INSIGhT trial, with the hope of providing primary GBM patients alternative treatment options."

"We are very excited to be sharing these results as part of the World Vaccine & Immunotherapy Congress, and are inspired, in particular, by and for the patient who has achieved a 93% tumor reduction," said Dr. Anderson. "If we are able to replicate the tumor responses and overall survival data seen to-date in the next phases of development, we believe VBI-1901 could become a meaningful addition to a treatment field with very few options. We look forward to evaluating VBI-1901 in both primary and recurrent GBM settings and strive to provide hope for patients battling either stage of this aggressive and devastating brain cancer."

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.

Recurrent GBM :
Expected Q1 2022 initiation of enrollment of expanded number of patients in ongoing study in recurrent GBM, subject to discussions with the FDA, increasing study size and adding a control arm to support the potential for application for accelerated approval based upon tumor response data and improvements in overall survival
Primary GBM :
Beginning mid-year 2022, VBI expects to evaluate VBI-1901 in new investigational treatment arms of INdividualized Screening Trial of Innovative Glioblastoma Therapy (INSIGhT), a Phase 2 adaptive platform trial
Patients will be randomized against a standard of care control arm, temozolomide in combination with radiotherapy, with a primary objective of improved overall survival in the frontline setting
To learn more about VBI’s ongoing Phase 1/2a study and the INSIGhT trial, visit clinicaltrials.gov (Respective Identifiers: NCT03382977 and NCT02977780).

World Vaccine & Immunotherapy Congress Presentation Details

Title: Development of eVLP platform for viral associated cancers
Presenter: David E. Anderson, Ph.D., VBI’s Chief Scientific Officer
Date: Wednesday, December 1, 2021
Time: 3:10 PM PT / 6:10 PM ET
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.