On October 16, 2020 Moffitt Cancer Ctr reported that Personalized treatment options for patients with lung cancer have come a long way in the past two decades (Press release, Moffitt Cancer Ctr, OCT 16, 2020, https://moffitt.org/newsroom/press-release-archive/2020/moffitt-researchers-develop-tool-to-better-predict-treatment-course-for-lung-cancer-patients [SID1234568606]).. For patients with non-small cell lung cancer, the most common subtype of lung cancer and the leading cause of cancer-related death worldwide, two major treatment strategies have emerged: tyrosine kinase inhibitors and immune checkpoint inhibitors. However, choosing the right therapy for a non-small cell lung cancer patient isn’t always an easy decision, as biomarkers can change during therapy rendering that treatment ineffective. Moffitt Cancer Center researchers are developing a noninvasive, accurate method to analyze a patient’s tumor mutations and biomarkers to determine the best course of treatment.

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In a new article published in Nature Communications, the research team demonstrates how a deep learning model using positron emission tomography/computerized tomography radiomics can identify which non-small cell lung cancer patients may be sensitive to tyrosine kinase inhibitor treatment and those who would benefit from immune checkpoint inhibitor therapy. The model uses PET/CT imaging with the radiotracer 18F-Fluorodeoxyglucose, a type of sugar molecule. Imaging with 18F-FDG PET/CT can pinpoint sites of abnormal glucose metabolism and help accurately characterize tumors.

"This type of imaging, 18F-FDG PET/CT, is widely used in determining the staging of patients with non-small cell lung cancer. The glucose radiotracer used is also known to be affected by EGFR activation and inflammation," said Matthew Schabath, Ph.D., associate member of the Cancer Epidemiology Department. "EGFR, or epidermal growth factor receptor, is a common mutation found in non-small cell lung cancer patients. EGFR mutation status can be a predictor for treatment, as patients with an active EGFR mutation have better response to tyrosine kinase inhibitor treatment."

For the study, the Moffitt team developed an 18F-FDG PET/CT-based deep learning model using retrospective data from non-small cell lung cancer patients at two institutions in China: Shanghai Pulmonary Hospital and Fourth Hospital of Hebei Medical University. The model classifies EGFR mutation status by generating an EGFR deep learning score for each patient. Once created, the researchers further validated the model using patient data from two additional institutions: Fourth Hospital of Harbin Medical University and Moffitt Cancer Center.

"Prior studies have utilized radiomics as a noninvasive approach to predict EGFR mutation," said Wei Mu, Ph.D., study first author and postdoctoral fellow in the Cancer Physiology Department. "However, compared to other studies, our analysis yielded among the highest accuracy to predict EGFR and had many advantages, including training, validating and testing the deep learning score with multiple cohorts from four institutions, which increased its generalizability."

"We found that the EGFR deep learning score was positively associated with longer progression free survival in patients treated with tyrosine kinase inhibitors, and negatively associated with durable clinical benefit and longer progression free survival in patients being treated with immune checkpoint inhibitor immunotherapy," said Robert Gillies, Ph.D., chair of the Cancer Physiology Department. "We would like to perform further studies but believe this model could serve as a clinical decision support tool for different treatments."

This work was supported by U.S. Public Health Service research grants (U01 CA143062, R01 CA190105), the National Natural Science Foundation of China (81971645, 81627901 and 81471724), the Tou-Yan Innovation Team Program of the Heilongjiang Province (2019-15), Natural Science Foundation of Heilongjiang Province (JQ2020H002), National Basic Research Program of China (2015CB931800) and the Key Laboratory of Molecular Imaging Foundation.

On October 16, 2020 Sanofi reported that Sarclisa (isatuximab) has been recommended in combination with pomalidomide and dexamethasone (pom-dex) for adult with relapsed/refractory multiple myeloma who have received three prior lines of treatment, and at least two prior therapies including lenalidomide and a proteasome inhibitor (Press release, Sanofi, OCT 16, 2020, View Source [SID1234568600]).

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The recommendation was based on data from the ICARIA-MM trial, which demonstrated that Sarclisa plus pom-dex showed a reduction in risk of disease progression or death in adults by 40%.

The treatment potentially extended progression-free survival to 11.5 months, compared to 6.5 months when treated with pom-dex alone. Overall, Sarclisa showed an additional 5.0 months without disease progression.

The addition of Sarclisa to pom-dex was also found to be well-tolerated, with no increase in treatment discontinuation or incidence of fatal events compared to that which was observed in the pom-dex group.

In the UK, approximately 5,700 people are diagnosed with multiple myeloma each year and around 3,000 multiple myeloma related death each year. Patients often experience frequent relapses and the cancer can become resistant to a number of available treatments.

"The limited therapies available for patients at later stages of the disease mean that increasing options at this point in the treatment pathway is crucial," said Laura Kerby, chief executive officer of Myeloma UK.

"Having access to a treatment that can provide valuable extra months without disease progression offers hope that people will have more time with their loved one," she added.

On October 16, 2020 Portage Biotech Inc. (CSE: PBT.U, OTC Markets: PTGEF) ("Portage" or the "Company") reported that the Company has filed its interim financial statements and related management’s discussion and analysis for the three-month period ending June 30, 2020 and related delivery requirements (the "Q1 Filings").

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Previously on August 28, 2020, Portage announced that pursuant to blanket relief granted by the Canadian Securities Administrators, it would not file the Q1 Filings by the filing deadline of August 31, 2020.

The Company relied on an exemption set out in Ontario Instrument 51-505: Temporary Exemption from Certain Corporate Finance Requirements and similar extension periods provided for by the Canadian Securities Administrators in the other provinces and territories of Canada which provides blanket relief of a 45-day extension provided for periodic filings normally required to be made by issuers during the period from June 2, 2020 to August 31, 2020.

This announcement serves as confirmation that Portage has filed the Annual Filings before the extension deadline required by the Canadian Securities Administrators. The Company is now current on its continuous disclosure.

Also, further to the Company’s news release of October 13, 2020 regarding issuance of common shares and warrants in settlement of certain SalvaRx loan note obligations, the Company has issued 397,604 (corrected from 375,014) common shares at a deemed price of US$6.64 per common share and 49,701 (corrected from 72,291) common share purchase warrants exercisable at a price of $6.64 per common share for a period of 2 years. This correction has been issued as a result of a late notification received by the Company from an arm’s length loan note holder.

On October 16, 2020 Kazia Therapeutics Limited (ASX: KZA;NASDAQ: KZIA), an Australian oncology-focused biotechnology company, reported that it has executed a definitive agreement with the Global Coalition for Adaptive Research (GCAR) to commence Kazia’s participation in the GBM AGILE pivotal study in glioblastoma (Press release, Kazia Therapeutics, OCT 16, 2020, View Source [SID1234568592]). The study will open a new arm with Kazia’s investigational new drug, paxalisib (formerly GDC-0084), and will now move into an operational phase with recruitment of patients to the paxalisib arm expected to begin in Q1 CY2021.

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Key Points

GBM AGILE (NCT03970447) is intended to serve as the pivotal study for registration of paxalisib in key markets
Dr Ingo Mellinghoff (Memorial Sloan Kettering Cancer Center) and Dr Eudocia Q Lee (Dana-Farber Cancer Institute) have been named as Principal Investigators for the paxalisib arm; Dr Timothy Cloughesy (UCLA) is the Principal Investigator for the overall study
Kazia will pay an initial fee of US$ 5 million to GCAR, with further milestone payments payable throughout the course of the study
The duration of paxalisib’s enrollment period in GBM AGILE is expected to total approximately 30 – 36 months, plus follow-up, but will depend on emerging study data, recruitment rates, and other variables
Kazia CEO, Dr James Garner, commented, "we have spent the last nine months or so working closely with the GCAR team to plan paxalisib’s entry into GBM AGILE, and we are very gratified to now be moving into the operational phase of the study. GBM AGILE is truly a ground-breaking clinical trial, driven by some of the world’s leading experts in the field, and we are proud to be a part of it. We expect GBM AGILE to provide definitive clinical evidence for the approval of paxalisib by regulatory agencies in key markets. This is a faster, more cost effective, and higher quality study than any company of our size could mount independently, and we are confident that it will provide the best possible opportunity for paxalisib to demonstrate its potential in this very challenging disease."

Dr Meredith Buxton, Chief Executive Officer at GCAR added, "We are pleased to welcome paxalisib into GBM AGILE. Our mission is to help drive the development of new therapies for glioblastoma, by creating an efficient model for testing and confirming new potentially beneficial treatments for patients with GBM. We look forward to continuing to work closely with the Kazia team to bring paxalisib into the study and support its evaluation."

Principal Investigators

Dr Ingo Mellinghoff and Dr Eudocia Q Lee will serve as Principal Investigators for the paxalisib arm. Dr Timothy Cloughesy is the Principal Investigator for the overall study.

Dr Mellinghoff is the Chair of the Department of Neurology at Memorial Sloan Kettering Cancer Center in New York, NY. He is a highly experienced neuro-oncologist with an extensive track record of published research in brain tumours, and is a Professor at the Gerstner Sloan Kettering Graduate School of Biomedical Sciences and the Graduate School of Medical Sciences at Weill Cornell University. His laboratory focuses on the study of biochemical pathways that regulate the growth of brain cancer, and he has participated in numerous clinical trials for glioblastoma and other forms of brain cancer.

Dr Mellinghoff commented, "we have seen little progress in the treatment of glioblastoma for over two decades, and the need for new therapies is urgent. We have seen encouraging signals from the paxalisib program thus far, and my colleagues and I look forward to exploring its potential in the GBM AGILE pivotal study."

Dr Lee is a neuro-oncologist at Dana-Farber Cancer Institute in Boston, MA, Director of Clinical Research at the Center for Neuro-Oncology at Dana-Farber, and an Assistant Professor of neurology at Harvard Medical School. She is a widely published clinical researcher, with a primary research interest in tumours of the brain and spinal cord, and their neurologic complications. Dr Lee has been an investigator in previous clinical trials of paxalisib in glioblastoma and has first-hand clinical experience with the drug.

Dr Lee added, "GBM AGILE has been designed to provide a definitive assessment of the efficacy of new drugs for glioblastoma. Paxalisib has already been evaluated in two clinical trials in this disease, and GBM AGILE will now greatly enrich our understanding of how best to use it for the benefit of patients."

GBM AGILE

GBM AGILE (Glioblastoma Adaptive Global Innovative Learning Environment) is an international platform study that has been established specifically to facilitate the approval of new medicines for glioblastoma.

The scientific leadership of GBM AGILE comprises many of the leading experts in glioblastoma, and they have worked in collaboration with the US FDA on its development. It is sponsored by the Global Coalition for Adaptive Research (GCAR), a US-based 501(c)(3) non-profit organisation. At present, the study is underway in 30 sites in the United States and Canada, with plans to launch in Europe and China during CY2021. One drug candidate is currently participating, and paxalisib will be the second candidate to join the study.

GBM AGILE is an adaptive study, so the number of patients recruited, and their allocation within the study, will be continuously adjusted in the light of emerging results. It is expected that between 50 and 200 patients will receive paxalisib, depending on the safety and efficacy of the drug. The data from these patients will be compared against data from an estimated several hundred patients in a shared control arm, allowing for considerable operational efficiency.

The paxalisib arm of GBM AGILE will recruit newly diagnosed patients with unmethylated MGMT promotor status, which is the same population that has been investigated in Kazia’s ongoing phase II study. In addition, GBM AGILE will recruit recurrent patients to the paxalisib arm. The drug may ultimately be considered efficacious in either or both of these patient groups, and Kazia will frame any future application for regulatory approval on the basis of this data.

Dr Mellinghoff added, "we see interesting signals of activity in the phase I study of paxalisib in recurrent glioma patients, and so my colleagues and I consider it important to evaluate the drug also in this later-stage group, where the unmet medical need is very substantial. Including both newly diagnosed and recurrent patients in GBM AGILE enables us to observe how paxalisib performs across the spectrum of the disease, and provides us with a significant amount of additional data as we move towards registration."

The primary endpoint of GBM AGILE is overall survival (OS), which is considered the gold standard endpoint for the assessment of new cancer therapies.

Indicative Costs and Timelines

Kazia will initially pay a fee of US$ 5 million to GCAR in consideration for paxalisib joining GBM AGILE. Additional payments will be due throughout the duration of the study, dependent on the attainment of key milestones. The full financial terms of the agreement between Kazia and GCAR are considered commercially confidential. In addition, the total cost of the study will depend on the number of patients ultimately recruited and other operational variables.

Kazia and GCAR expect that necessary regulatory filings and submissions to institutional review boards will be actioned during 4Q CY2020. First patient in to the paxalisib arm is currently anticipated to occur early in CY2021.

The duration of paxalisib’s participation in GBM AGILE is unpredictable due to the adaptive nature of the study. As an indicative base case estimate, Kazia expects at this stage that paxalisib will enrol patients for between 30 – 36 months, plus follow-up. However, this figure could change, either in an upward or downward direction, depending on emerging data from the study as well as operational matters such as recruitment rates.

On October 16, 2020 Photocure ASA (OSE:PHO) reported a highlight from the BLADDR 2020 congress, a poster presentation on new findings from the Nordic Flexible BLC registry, an ongoing prospective multicenter study (Press release, PhotoCure, OCT 16, 2020, View Source [SID1234568591]). It demonstrates that flexible Blue Light Cystoscopy helped resolve a substantial amount of cases by complete removal on-site or direct referral to intravesical treatment, providing increased efficacy to manage non-muscle-invasive bladder cancer (NMIBC) in the office setting.

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The BLADDR congress, held virtually this year, is solely focused on bladder cancer and attracts the interest of experts from all over the world.

"It is encouraging to see how data on the blue light cystoscopy procedure continues to provide new insight into the efficiency of how to manage NMIBC in the office setting. The BLC procedure is a tool that helps physicians solve such cases outside of the OR. Using blue light cystoscopy with Hexvix, they trust their detection capabilities in the office and can treat the lesions immediately, perform confirmatory biopsies or initiate intravesical therapy. Such options are less costly than OR procedures, thus helping to keep costs down for the healthcare system and sparing the patient additional surgical procedures in the OR. This is a clear win-win situation for both patient and healthcare system", says Dan Schneider, President and CEO of Photocure.

Methodology and data from the study:

The Nordic Flexible BLC registry is an ongoing prospective multicenter study initiated to observe the clinical use and explore possible benefits of blue light cystoscopy in the surveillance setting. From five participating sites, 354 patients in follow-up of NMIBC have been included in the study. Data from 462 blue light procedures were included.

In follow-up of NMIBC, in a predominant intermediate and high-risk patient population, this analysis shows how blue light cystoscopy in a surveillance setting helped resolve a substantial amount of cases by complete removal of lesions on-site, or direct referral to intravesical instillation. In 88% of cases where suspicious lesions were seen, no further diagnostic or surgical treatment procedures (TURBT) were needed.

The authors conclude that blue light cystoscopy in the surveillance setting provides increased efficiency to manage NMIBC.

Here is a link to the poster.

Note to editors:

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About Bladder Cancer

Bladder cancer ranks as the sixth most common cancer worldwide with 1 650 000 prevalent cases (5-year prevalence rate), 550 000 new cases and almost 200 000 deaths annually in 2018.1

Approx. 75% of all bladder cancer cases occur in men.1 It has a high recurrence rate with an average of 61% in year one and 78% over five years.2 Bladder cancer has the highest lifetime treatment costs per patient of all cancers.3

Bladder cancer is a costly, potentially progressive disease for which patients have to undergo multiple cystoscopies due to the high risk of recurrence. There is an urgent need to improve both the diagnosis and the management of bladder cancer for the benefit of patients and healthcare systems alike.

Bladder cancer is classified into two types, non-muscle invasive bladder cancer (NMIBC) and muscle-invasive bladder cancer (MIBC), depending on the depth of invasion in the bladder wall. NMIBC remains in the inner layer of cells lining the bladder. These cancers are the most common (75%) of all BC cases and include the subtypes Ta, carcinoma in situ (CIS) and T1 lesions. In MIBC the cancer has grown into deeper layers of the bladder wall. These cancers, including subtypes T2, T3 and T4, are more likely to spread and are harder to treat.4

1 Globocan. Incidence/mortality by population. Available at: View Source
2 Babjuk M, et al. Eur Urol. 2019; 76(5): 639-657
3 Sievert KD et al. World J Urol 2009;27:295-300
4 Bladder Cancer. American Cancer Society. View Source

About Hexvix/Cysview (hexaminolevulinate HCl)

Hexvix/Cysview is a drug that preferentially accumulates in cancer cells in the bladder making them glow bright pink during Blue Light Cystoscopy (BLCTM). BLC with Hexvix /Cysview improves the detection of tumors and leads to more complete resection, fewer residual tumors and better management decisions.

Cysview is the tradename in the U.S. and Canada, Hexvix is the tradename in all other markets. Photocure is commercializing Cysview /Hexvix directly in the U.S. and the Nordic region and has strategic partnerships for the commercialization of Hexvix/Cysview in Europe, Canada, Australia and New Zealand. Please refer to https://bit.ly/2wzqSQQ for further information on our commercial partners.