On September 24, 2020 VBI Vaccines Inc. (Nasdaq: VBIV) (VBI), a commercial-stage biopharmaceutical company developing next-generation infectious disease and immuno-oncology vaccines, reported upcoming presentations highlighting data from multiple clinical programs – including Sci-B-Vac, the company’s 3-antigen hepatitis B vaccine; VBI-2900, the company’s coronavirus vaccine program; and VBI-1901, the company’s cancer vaccine immunotherapeutic – at the World Vaccine Congress Washington 2020 and ID Week 2020 (Press release, VBI Vaccines, SEP 24, 2020, View Source [SID1234565567]).

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Presentation Details

World Vaccine Congress Washington 2020: September 28 – October 1, 2020

COVID-19 Panel Discussion: What existing treatments and vaccine development platforms show promise to control the rate of infection and spread?

Session: Emerging and Infectious

Participant: David E. Anderson, Ph.D., VBI’s Chief Scientific Officer

Date: Monday, September 28, 2020

Time: 3:50 – 5:20 PM ET

Presentation: Sci-B-Vac: Results on Ph3 Hep B vaccine

Session: Emerging and Infectious

Presenter: Francisco Diaz-Mitoma, M.D., VBI’s Chief Medical Officer

Date: Tuesday, September 29, 2020

Time: 4:35 – 5:05 PM ET

Presentation: Targeting CMV for the development of effective GBM immunotherapy

Session: Cancer & Immunotherapy

Presenter: David E. Anderson, Ph.D., VBI’s Chief Scientific Officer

Date: Wednesday, September 30, 2020

Time: 1:40 – 2:10 PM ET

World Vaccine Congress Event Website: View Source

ID Week 2020: October 21-25, 2020

Poster #: 8

Title: Higher hepatitis B antibody titers induced in all adults vaccinated with a tri-antigenic hepatitis B (HBV) vaccine, compared to a mono-antigenic HBV vaccine: results from two pivotal phase 3 double-blind, randomized studies (PROTECT and CONSTANT)

Poster Session: Adult Vaccines

Presenter: Joanne Langley, M.D., Professor of Pediatrics and Community Health and Epidemiology, CIHR-GSK Chair in Pediatric Vaccinology, Dalhousie University, and Head of the Division of Infectious Disease, IWK Health Centre, and principal investigator of the PROTECT study

Date: Available October 21-25, 2020

Title: Rapid onset of seroprotection rates in young adults immunized with a tri-antigenic hepatitis B virus (HBV) vaccine compared to a mono-antigenic HBV vaccine

Poster Session: Hepatitis

Presenter: Timo Vesikari M.D., Ph.D., Professor Emeritus and Director of the Nordic Vaccine Research Network in Finland, and principal investigator of the PROTECT and CONSTANT Phase 3 clinical studies

Date: Available October 21-25, 2020

ID Week 2020 Event Website: View Source

About Sci-B-Vac

Sci-B-Vac is a licensed, third-generation hepatitis B vaccine that has demonstrated safety and efficacy in over 750,000 patients. Sci-B-Vac is the only 3-antigen hepatitis B vaccine, comprised of the S, pre-S1, and pre-S2 surface antigens of the hepatitis B virus, and is approved for use and commercially-available in Israel. In December 2017, VBI initiated patient dosing in a global Phase 3 clinical program that consisted of two concurrent pivotal studies: PROTECT, a safety and immunogenicity study, and CONSTANT, a lot-to-lot consistency study. Data from both the PROTECT study and the CONSTANT study, which were announced in June 2019 and January 2020, respectively, will comprise the basis for the regulatory submissions in the U.S., Europe, and Canada, expected to begin in the fourth quarter 2020.

To learn more about Sci-B-Vac, visit: View Source

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 is exceptionally lethal.

About Coronaviruses

Coronaviruses are a large family of enveloped viruses that usually cause respiratory illness of varying severities, including the common cold and pneumonia. Only seven coronaviruses are known to cause disease in humans, four of which most frequently cause symptoms of the common cold. Three of the seven coronaviruses, however, have more serious outcomes in people: (1) SARS-CoV-2, a novel coronavirus identified as the cause of coronavirus disease 2019 (COVID-19); (2) MERS-CoV, identified in 2012 as the cause of Middle East respiratory syndrome (MERS); and (3) SARS-CoV, identified in 2002 as the cause of an outbreak of severe acute respiratory syndrome (SARS).1,2

On September 24, 2020 Nucleix, a liquid biopsy company revolutionizing cancer treatment by detecting the disease earlier, reported a published independent review of Bladder EpiCheck studies which showed high diagnostic accuracy and high performance for surveillance of disease recurrence in patients with non-muscle invasive bladder cancer (NMIBC) (Press release, Nucleix, SEP 24, 2020, View Source [SID1234565586]). The publication, titled "The Bladder EpiCheck Test as a Non-Invasive Tool Based on the Identification of DNA Methylation in Bladder Cancer Cells in the Urine: A Review of Published Evidence," was published in the International Journal of Molecular Sciences.

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The review analyzed data from five prospective, blinded, single-arm cohort studies of Bladder EpiCheck for NMIBC detection. The review focused on data from populations of patients in surveillance for histologically proven NMIBC. Notably, Bladder EpiCheck showed a high sensitivity and negative predictive value (NPV) in several studies and a higher sensitivity and NPV than cytology in all studies. Authors concluded Bladder EpiCheck had the greatest diagnostic power in patients with high-grade NMIBC, and it could be used as a monitoring tool to extend time between cystoscopies.

"Patients with NMIBC are at high risk for recurrence, making surveillance critical to identify recurrence, progression, or localization in different areas of the urinary tract. However, quarterly cystoscopies are invasive and painful for patients, which places a high burden on them and on healthcare systems," said Aharona Shuali, M.D., vice president of medical at Nucleix. "We are pleased that this review of Bladder EpiCheck demonstrates how the test’s high sensitivity and NPV in detection of high-grade disease makes it a meaningful option to safely reduce frequency of cystoscopies."

The review noted that some positive cases of Bladder EpiCheck with negative cystoscopy and positive/negative cytology results, seem to present DNA methylation changes. These ‘false-positive’ cases of Bladder EpiCheck could identify a genetic high-risk population that may relapse in the near future and deserves close clinical attention. Bladder EpiCheck negative cases could be based on a more stable methylation pattern, which authors propose could represent a population of high-risk bladder cancer patients with an inferior risk of progression.

"We need a test with high accuracy for NMIBC, especially for high-grade NMIBC, to help avoid unnecessary cystoscopies," said Mariangela Mancini, M.D., consultant urological surgeon at the University of Padua in Padua, Italy, and lead author of the study. "The high performance demonstrated by the current evidence, combined with Bladder EpiCheck’s non-invasive nature, could make it an invaluable tool in the future for follow-up of bladder cancer patients. Furthermore, having a safe alternative for follow-up that does not require patients to come to the hospital, could be even more valuable during the COVID-19 pandemic."

About Bladder EpiCheck

Bladder EpiCheck provides patients and clinicians with a simple, objective urine test to detect recurrence of bladder tumors. The test analyzes subtle disease-specific changes in DNA methylation markers, allowing for the detection of 92% of the high-risk (non Ta-LG) cancers. Bladder EpiCheck demonstrated negative predictive value (NPV) of 99% for high-risk cancer, meaning that when receiving a negative Bladder EpiCheck result, there is 99% chance that no high-risk cancer is present1. Bladder EpiCheck is intended for use as a noninvasive method for monitoring of tumor recurrence in conjunction with cystoscopy in patients previously diagnosed with bladder cancer. Bladder EpiCheck is CE-marked and available in Europe. The test is not available for sale in the United States.

On September 24, 2020 Oncoheroes Biosciences, a biotech focused on advancing new therapies for childhood cancer, reported that the United States Food and Drug Administration (FDA) has granted the designation of rare pediatric disease to volasertib, an investigational treatment for rhabdomyosarcoma (Press release, Oncoheroes Biosciences, SEP 24, 2020, View Source [SID1234568287]).

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Key Points
Rare Pediatric Disease Designation qualifies Oncoheroes to receive fast track review, and a priority review voucher (PRV) at the time of marketing approval of volasertib.
PRV holders can benefit from an expedited six-month review of a new drug application for any disease by the FDA.
PRVs are transferable to other sponsor companies and historically have had a selling price range of USD 67 to 350 million.
The US FDA actively supports companies that develop drugs for rare diseases, defined as diseases affecting less than 200,000 Americans. One of the relevant programs created by the agency is for rare diseases primarily affecting individuals younger than 18 years old, called the Rare Pediatric Disease Designation (RPDD) which comes with the related priority review voucher opportunity

"We are delighted that volasertib has been awarded with RPDD by the FDA. This recognition acknowledges the unmet medical need for better treatments for children and adolescents with rhabdomyosarcoma. We hope this big news will allow Oncoheroes to speed up the drug development process of volasertib," stated Ricardo Garcia, Oncoheroes’ Founder and CEO.

Upon drug approval, the RPDD may provide substantial financial incentives by making companies eligible for a Priority Review Voucher (PRV) that is fully transferable. The PRV grants accelerated FDA review of a drug candidate, for any indication, reducing the review period to 6 months and potentially gaining early market access. To date, 12 out of 25 PRVs received for pediatric indications have been sold for a cumulative sale price of USD 1.6 billion.

"Current rhabdomyosarcoma treatments are based on decades-old therapies and generally lack efficacy against the most aggressive subtypes of the disease, for which the 5-year survival rate is currently 20-30%. We are excited about upcoming clinical studies and we hope that volasertib could be a game-changer for rhabdomyosarcoma patients," explained Cesare Spadoni, PhD, Oncoheroes’ Founder and COO.

Around 500 new patients each year in the US are diagnosed with rhabdomyosarcoma, an aggressive and highly malignant form of cancer (soft tissue sarcoma) that develops from skeletal muscle cells that have failed to fully differentiate. There is a clear unmet medical need for the treatment of the most aggressive forms of this disease.

Volasertib is an inhibitor of Polo-like-kinase 1 (PLK1), an enzyme known to be involved in disease progression in a number of cancers. The compound was originally discovered and developed by Boehringer Ingelheim for the treatment of Acute Myeloid Leukemia until the company decided to discontinue the compound for strategic reasons. Meanwhile, independent academic groups generated strong data in support of further development of volasertib for rhabdomyosarcoma and, possibly, a few other pediatric cancer indications. In 2019, Oncoheroes in-licensed volasertib from Boehringer Ingelheim to continue the clinical development of this drug candidate for the benefit of younger cancer patients.

Preclinical research in Rhabdomyosarcoma
A number of publications highlight the potential of volasertib in rhabdomyosarcoma. High PLK1 expression has been associated with poor prognosis in a number of cancers, including rhabdomyosarcoma. It was shown that the drug may have a specific anti-cancer effect in this disease, which is driven by the PAX3-FOXO1 fusion protein in a large subset of patients. PAX3-FOXO1 is a challenging drug target. However, it was shown that PLK1 inhibition by volasertib reduces the stability of this fusion protein leading to its degradation and cancer growth inhibition in PDX models. Interestingly, in vivo data also point to a strong synergy between volasertib and vincristine, a drug already in the standard treatment protocol for rhabdomyosarcoma. Most of these data were generated by European laboratories that are part of the Innovative Therapies for Children with Cancer (ITCC) consortium. Oncoheroes is planning to collaborate with ITCC for the clinical development of volasertib.

On September 24, 2020 Nuformix plc (LSE:NFX) ("Nuformix" or "the Group"), a pharmaceutical development company focused on unlocking the therapeutic potential and value of known drugs to develop novel medicines to provide enhanced benefit, reported it has signed an exclusive option agreement with Oxilio Ltd ("Oxilio") (Press release, Nuformix, SEP 24, 2020, View Source [SID1234621605]).

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.Agreement covers a licence for the development and exploitation of NXP001 in oncology
· Up-front payment for an exclusive option period of 6 months, within which the global licensing agreement can be triggered
· Should Oxilio exercise the option, Nuformix will licence its patent estate and know-how on NXP001 in return for a significant upfront payment and additional development milestones and a royalty on net sales, capped at £2 million per annum

Under the terms of the agreement Nuformix will also provide a fixed amount of consultancy services to Oxilio during the option period. Oxilio will develop and seek to exploit NXP001 globally for the treatment of cancer and early clinical trials will determine which cancer types respond best to treatment. Cancer is the second leading cause of death globally with around 10 million deaths per annum, in spite of extensive studies to find new treatment regimens and more effective drugs. However, the cost of cancer treatment is increasing, in large part due to the expense of taking drugs through clinical trials where historically there is a very low rate of success. Therefore, there is an urgent need to develop safe, effective, and readily available anticancer agents.

Oxilio is focused on developing a therapeutic with the potential to treat patients with various cancer types representing a global therapeutics market estimated to be more than $130 billion (BCC Publishing, Jan 2019). Oxilio is focused on alleviating the current dilemma of a shortage of drug candidates for finding new cancer therapies, by adopting a drug repurposing strategy (identifying new uses for approved or investigational drugs that are outside the scope of the original medical indication). The major advantage of this approach is that the pharmacokinetics, pharmacodynamics and toxicity profiles of these drugs are already reasonably well established.

Thus, drug repurposing may hold the potential to result in a less risky development route with substantially lower associated development costs. The collaboration with Nuformix allows Oxilio to focus on developing rapidly a unique formulation and dosage form with NXP001. This would potentially overcome the key hurdle in drug repurposing, patent consideration-induced market exclusivity, as well as providing an accelerated route to the clinic.

Dr Chris Blackwell, Executive Chairman, said: "This agreement allows Nuformix the opportunity to benefit from the upside of a significant global market opportunity whilst realising short-term revenues. Furthermore, it demonstrates our commitment to explore and leverage value creating opportunities for all our pipeline assets. We look forward to working with Oxilio on this collaboration."

Dr Simon Yaxley, Co-Founder and Director of Oxilio said: "This collaboration with Nuformix allows Oxilio to continue the development of NXP001 as a potential new treatment in the battle against cancer. We are excited by the opportunity of accelerating our science towards our first clinical trials through this collaboration and we look forward to working with the Nuformix team to realise the opportunity".

About NXP001-Oncology
NXP001 has been developed by Nuformix, to date, with the aim of improving its use in the cancer critical care setting, particularly with regard to chemotherapy-induced nausea and vomiting (CINV). Business development activities continue with this focus alongside the option agreement for Oxilio to explore its potential in treating cancers.

About Cocrystals Pharmaceutical cocrystals are materials composed of two or more different molecules, usually an active pharmaceutical ingredient together with a pharmaceutically acceptable "coformer" molecule. Cocrystals can be engineered to enhance the bioavailability, pharmacokinetics, stability and manufacturing of drug products.

On September 24, 2020 Alligator Bioscience (Nasdaq Stockholm: ATORX) reported that the company launches a new concept for patient specific immunotherapy, called Neo-X-Prime (Press release, Alligator Bioscience, SEP 24, 2020, View Source [SID1234565569]). Alligator presents the concept at the scientific congress 11th World Bispecific Summit held September 22-24, 2020.

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The new concept, based on Alligator’s bispecific format RUBY, build on bispecific antibodies that physically connect tumor debris from the patient’s cancer cells with the relevant immune cells, resulting in strong and tumor selective immune activation. This is thought to increase both the accuracy of the immune attack, and the overall anti-tumor effect. The issue with today’s cancer vaccines is that it is not obvious what structure the immune system should be directed towards, and the vaccine must be produced specifically for each single patient. In Alligator’s concept, the bispecific antibody could solve all this.

"The new concept may be viewed as a patient specific therapeutic vaccination with the aim of curing cancer. The early data are extremely promising and show that Neo-X-Prime antibodies have the potential to induce anti-tumor effects superior to any current treatment option", said Per Norlén, CEO at Alligator Bioscience.
Today, September 24 at 5:20 pm CEST (1:20 pm EDT) Dr Peter Ellmark, VP Discovery at Alligator, will give a presentation with the title "Priming Neoantigen Specific T-Cells with a Novel Bispecific Antibody". For further information, see bispecific.com.

The information was submitted for publication, through the agency of the contact person set out above, at 4:45 p.m. CEST on September 24, 2020.