On November 19, 2020 Invitae (NYSE: NVTA), a leading medical genetics company, reported that study findings that show nine percent of patients with pancreatic cancer had genetic changes in DNA damage repair (DDR) genes that would make them eligible for PARP inhibitor therapy or clinical treatment trials (Press release, Invitae, NOV 19, 2020, View Source [SID1234571423]). Despite professional guidelines that recommend testing for all pancreatic cancer patients, it remains underutilized in routine care. The study was presented at the National Society of Genetic Counselors 39th Annual Conference.

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Invitae’s (NVTA) mission is to bring comprehensive genetic information into mainstream medical practice to improve the quality of healthcare for billions of people. www.invitae.com (PRNewsFoto/Invitae Corporation)

"New therapeutics have recently become available to treat pancreatic cancer for patients with certain changes in genes such as BRCA1 and BRCA2. Yet despite the availability of these treatments and professional guidelines recommending testing, utilization is still lagging," said Robert Nussbaum, M.D., chief medical officer of Invitae and study author. "Pathogenic variants in these genes are associated with an increased risk of other cancers as well, such as breast, ovarian and prostate cancer, which means that a failure to test patients with pancreatic cancer impacts not only their treatment, but also the health of their families."

Importantly, the study of over 2,000 patients found that 15% of patients with actionable genetic changes reported no family history of cancer, which underscores the limitations of using testing criteria based on reported family history. National Comprehensive Cancer Network (NCCN) guidelines recommend genetic counseling and germline genetic testing for everyone diagnosed with pancreatic cancer as well as their first degree relatives — approximately 3.5 million individuals in the United States.

In addition to evaluating the clinical relevance of genetic testing results, the study offered sponsored, no-charge testing to patients to evaluate the role of cost as a barrier to testing. Researchers found a small but significant increase (2%) in testing among African-American patients compared to typical rates among patients using health insurance, suggesting reducing cost may increase access to testing among this population.

The research was presented at the virtual annual meeting of the National Society of Genetic Counselors. The full research presentation from Invitae included:

Oral platform presentations:

Increasing access for patients with pancreatic cancer to germline genetic testing: Clinical impacts across disease stage and ethnicity. Presented by Ed Esplin, MD, PhD, FACMG, FACP
Evaluating variant reclassification in reproductive carrier screening. Presented by Julia Wilkinson, MS, LCGC
Poster presentations:

Utility of adding phenotypic criteria refinement to ACMG guidelines. Presented by Lauren Frank, MS, CGC
Comprehensive germline multigene panels change clinical care and inform treatment strategies for breast cancer patients. Presented by Sarah Nielsen, MS, LCGC
Carrier screening for X-linked conditions is common practice. Presented by Dana Neitzel, MS, CGC
In addition to its scientific presence, Invitae will again partner with NSGC to present the Heart of Genetic Counseling award, which honors excellence in genetic counseling and patient care as recognized by patients. Nominations include stories from patients that highlight both the clinical and personal impact a genetic counselor had on their lives and the lives of their families. This year’s award will be presented during a virtual ceremony on Thursday, November 17th. The finalists include:

Rachelle Manookian, MS, LCGC, City of Hope in Duarte, CA, was nominated by a man who had been searching for answers about the origin of his cancer for more than 10 years. He hoped that any identified mutations would lead to a newly developed drug or clinical trial that could attack his cancer with precision. Manookian left no stone unturned and remained in touch until she found a clinical trial. Thanks to Manookian’s persistence, he finally got answers and learned that his sons did not carry the same increased genetic risk of prostate cancer.
Donna McDonald-McGinn, MS, LCGC, Children’s Hospital of Philadelphia in Philadelphia, was nominated by the mother of a son with a rare genetic condition called 22q11.2 deletion syndrome. McDonald-McGinn has been his care coordinator and navigator for 21 years, explaining and managing the care that he has needed through his life. Always acting as his advocate, McDonald-McGinn has been there for each of his 21 surgeries and is a staunch advocate for those with 22q11.2 deletion syndrome.
Gretchen MacCarrick, MS, CGC, Johns Hopkins Medicine in Baltimore, was nominated by a mother whose daughter has Loeys-Dietz syndrome (LDS), which was discovered in 2005. For nearly 12 years, MacCarrick has been by their side guiding them. She explains what tests are needed, what the results mean and navigates their daughter’s care during their regular visits to Baltimore, including a major heart surgery. Importantly, MacCarrick has even developed a strong relationship directly with the patient who is now 16 and getting more engaged in her care.

On November 19, 2020 VBI Vaccines Inc. (Nasdaq: VBIV) ("VBI") a commercial-stage biopharmaceutical company developing next-generation infectious disease and immuno-oncology vaccines, reported Phase 2a (Part B) data from its ongoing Phase 1/2a study of VBI-1901, the company’s cancer vaccine immunotherapeutic candidate designed to target cytomegalovirus (CMV) as a foreign viral antigen in recurrent glioblastoma (GBM) (Press release, VBI Vaccines, NOV 19, 2020, View Source [SID1234571439]). The data were presented in an e-poster at the Society for Neuro-Oncology (SNO) 2020 Annual Meeting, November 19-21, 2020.

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Data from Phase 2a (Part B) of the ongoing study showed:

2 partial responses (PRs) and 2 stable disease (SD) observed in the VBI-1901 + GM-CSF vaccinated group, resulting in a disease control rate of 40% (n=4/10)
A 56% disease control rate achieved in the group vaccinated with VBI-1901 + AS01, with 5 stable disease observations (n=5/9) – tumor response data for the 10th patient enrolled is pending
Presumed pseudoprogression was observed in both vaccinated groups– defined as immune infiltration into the tumor which appears initially as tumor growth, but later subsides resulting in tumor growth stabilization and/or shrinkage
Andrew B. Lassman, M.D., Chief of Neuro-oncology at Columbia University Irving Medical Center and Associate Director for Clinical Trials at the Herbert Irving Comprehensive Cancer Center, and principal investigator of the study presented the e-poster, commenting, "Tumor response data is one of the most objective measures of efficacy in open label studies, especially in this difficult-to-treat patient population. Few treatment options are available to recurrent glioblastoma patients, and the tumor response data seen to-date in this ongoing study are encouraging. Any treatment that could demonstrate clinical benefit would be incredibly meaningful."

Emmanuel Hanon, Senior Vice President, Head of R&D Vaccines at GSK, commented, "The early data seen to-date in this ongoing study are encouraging, underscoring the potential benefit of adjuvants in combination with VBI-1901 in the clinical setting. Previous research in the context of other vaccines has shown AS01’s ability to boost T cell-mediated immunity. This is the first time GSK’s adjuvant system is assessed in oncology and we are looking forward to getting more data about the potential of therapeutic vaccination to treat such an aggressive and recurring disease."

David E. Anderson, Ph.D., VBI’s Chief Scientific Officer, commented, "This ongoing study continues to demonstrate the potential of VBI-1901, with both the GM-CSF adjuvant and GSK’s AS01 adjuvant system, to be an effective cancer vaccine immunotherapeutic. The tumor responses seen to-date across both study arms, including 2 partial responses and 7 stable disease, are meaningful, especially as an outcome of a monotherapy. This data supports the continued development of the program, both as a monotherapy as well as part of a combination regimen."

Based on the available data, VBI is exploring a randomized, controlled clinical study, including a potential registration study, for the next phase of development, which could begin in 2021, pending approval from regulatory bodies.

A webcast of Dr. Anderson discussing these data with Jeff Baxter, VBI’s President and CEO, can be found here: View Source

A copy of the e-poster is available on the "Events/Presentations" page in the "Investors" section of the VBI Vaccines website.

About the Phase 1/2a Study Design

VBI’s two-part Phase 1/2a study is a multi-center, open-label, dose-escalation study of VBI-1901 in up to 38 patients with recurrent GBM:

Phase 1 (Part A)
Dose-escalation phase that defined the safety, tolerability, and optimal dose level of VBI-1901 adjuvanted with granulocyte-macrophage colony-stimulating factor (GM-CSF) in recurrent GBM patients with any number of prior recurrences.
This phase enrolled 18 recurrent GBM patients across three dose cohorts of VBI-1901: 0.4 µg, 2.0 µg, and 10.0 µg.
Enrollment completed in December 2018.
Phase 2a (Part B)
Subsequent extension of the optimal dose level, 10.0 µg, as defined in the Part A dose escalation phase.
This phase is a two-arm study, enrolling 10 first-recurrent GBM patients in each vaccinated group, assessing 10.0 µg of VBI-1901 in combination with either GM-CSF or GSK’s proprietary AS01 adjuvant system as immunomodulatory adjuvants.
Enrollment of the 10 patients in each adjuvant group is complete.
VBI-1901 is administered intradermally when adjuvanted with GM-CSF and intramuscularly when adjuvanted with GSK’s AS01 adjuvant system. Patients in both phases of the study receive the vaccine immunotherapeutic every four weeks until tumor progression.

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 November 19, 2020 In this worldwide exclusive agreement, Carina Biotech reported that it will create Chimeric Antigen Receptor T cells (CAR-T cells) and other adoptive cell therapies to the leucine-rich repeat-containing G-protein coupled receptor 5 (LGR5) using the first-in-class humanised antibody BNC101 developed by Bionomics Limited (Press release, Carina Biotech, NOV 19, 2020, View Source [SID1234571346]).

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LGR5 is a molecular marker that is highly expressed on cancer stem cells within solid tumours including colorectal, breast, pancreatic, ovarian, lung, liver and gastric cancers.

This will be Carina’s latest CAR-T cell candidate in a growing pipeline, underpinned by its proprietary platform that produces "supercharged" CAR-T cells. CAR-T cell therapy is a revolutionary new treatment option for cancer that harnesses the power of the immune system. CAR-T cells are genetically modified immune cells (T cells) that are targeted at certain molecular markers found on cancer cells.

Carina Biotech’s CEO Dr Deborah Rathjen said, "Cancer stem cells, or CSCs, seem to have some kind of resistance to anti-cancer therapies. Anti-cancer drugs can kill proliferating cancer cells but CSCs survive – hence some cancers will respond to initial treatment but inevitably the cancer returns".

"We are excited to have done this deal with Bionomics given the wealth of clinical data available on BNC101. By creating a CAR-T cell that targets CSCs, we will be able to get to the core of some cancers, to kill those cells that enable cancers to keep bouncing back. We hope that LGR5-targeting CAR-T cells will go a long way to achieving our vision – a future that defeats cancer."

This is the third commercial deal for Carina in three months, following the sale of its first proprietary CAR-T cell to UK company Biosceptre, and a deal with Sydney immuno-oncology company Glytherix.

"Our serial-killing CAR-T cells do what so far has been very difficult to do – travel to the site of the cancer and disrupt the immunosuppressive tumour microenvironment. This allows for our CAR-T cells to get up close to cancer cells for potent and repeated killing action," says Dr Rathjen.

Carina’s platform also enables efficient CAR-T manufacture – reducing manufacturing time, improving yield and quality with CAR-T delivery efficiency of more than 90%.

Under the worldwide, exclusive License Agreement, Carina will fund all research and development activities. Bionomics is eligible to receive up to A$118 million in clinical and development milestones plus royalty payments if Carina fully develops and markets the new therapy. In the event that Carina sub-licenses the CAR-T treatment, Bionomics is eligible to receive sub-licensing revenues.

On November 19, 2020 Neurocrine Biosciences, Inc. (Nasdaq: NBIX) reported that the Company has entered into separate, privately negotiated transactions (the "Agreements") with certain holders of its existing 2.25% Convertible Senior Notes due 2024 (the "2024 Notes") to repurchase approximately $83 million aggregate principal amount of the 2024 Notes for an aggregate repurchase price of an amount of cash estimated to be the sum of (i) approximately $110 million based on the Company’s November 18, 2020 closing stock price of $86.91 per share, (ii) an amount based in part on the daily volume-weighted average prices per share of the Company’s common stock during a five-trading day pricing period following execution of the Agreements and (iii) accrued and unpaid interest (Press release, Neurocrine Biosciences, NOV 19, 2020, View Source [SID1234571401]). The 2024 Notes repurchases are expected to close on December 2, 2020, subject to customary closing conditions. Such repurchases of the 2024 Notes could affect the market price of the Company’s common stock.
This press release does not constitute an offer to sell or a solicitation of an offer to buy these securities, nor shall there be any offer or sale of these securities in any state or jurisdiction in which the offer, solicitation, or sale would be unlawful prior to the registration or qualification thereof under the securities laws of any such state or jurisdiction.

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On November 19, 2020 Oncopeptides reported that (Press release, Oncopeptides, NOV 19, 2020, View Source [SID1234571424])

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Financial overview July 1 – September 30, 2020
Net sales amounted to SEK 0.0 M (0.0)
Loss for the period was SEK 383.4 M (loss: 189.8)
Loss per share, before and after dilution, was SEK 5.71 (loss: 3.53)
On September 30 cash and cash equivalents amounted to SEK 1,251.6 M (1,122.3)
Significant events during the period July 1 – September 30, 2020
The FDA granted priority review of melflufen for patients with triple-class refractory multiple myeloma and set the PDUFA date to February 28, 2021
Patient enrolment in the pivotal phase 3 OCEAN study was completed including 495 patients
Patient enrolment for the phase 1/2 study in AL-amyloidosis began, this is the first study with melflufen in an indication outside multiple myeloma
The phase 2 PORT study evaluating alternative administration of melflufen and dexamethasone in multiple myeloma started
Oncopeptides further coordinated the global and US organizational structure and appointed Mohamed Ladha as General Manager of the US Business Unit
Significant events after the reporting period
Oncopeptides announced that the company intends to submit a conditional marketing authorization application for melflufen in the EU
Oncopeptides entered into a €40 M loan agreement with the European Investment Bank (EIB)
An IND application was submitted to the FDA to initiate clinical studies with OPD5, Oncopeptides’ second drug candidate

This information is information that Oncopeptides is obliged to make public pursuant to the EU Market Abuse Regulation and the Securities Markets Act. The information was submitted for publication, through the agency of the contact persons set out above, at 08:00 CET on November 19, 2020.