On July 21, 2021 Kazia Therapeutics Limited (NASDAQ: KZIA; ASX: KZA), a leading drug development company in the field of brain cancer, reported that Glioblastoma (GBM) Awareness Day and calls for a greater focus on research from industry, academia, and government in order to better treat the disease (Press release, Kazia Therapeutics, JUL 21, 2021, View Source [SID1234585040]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

GBM is the most common and most aggressive form of primary brain cancer, and it is expected that approximately 13,000 Americans will receive a GBM diagnosis in 2021. Unlike many other cancers there has been little progress made in the treatment of this disease and the standard of care has remained largely unchanged since the turn of the century.

Current treatment for GBM includes surgery, radiotherapy and chemotherapy with temozolomide. However, around two-thirds of patients receive no meaningful benefit from temozolomide. The median survival from diagnosis remains approximately 16 months and has improved little in the last twenty years.

Kazia CEO, Dr James Garner, said, "Enormous progress has been made in the treatment of so many cancers, but GBM has thus far remained intractable, with outcomes for patients relatively unchanged in two decades. However, the future for this disease looks much more promising, with new insights into the biology of GBM and a renewed focus from academia and industry combining to explore new therapeutic frontiers. We remain wholly confident that this disease can be beaten, and we call on all stakeholders – clinicians, researchers, government, industry, and patient advocates – to redouble their efforts on improving the prognosis for patients with glioblastoma."

About GBM Awareness Day

Glioblastoma Awareness Day takes place on July 21, 2021, with a specific focus on:

Increasing public awareness of glioblastoma
Honoring the individuals who have lost their lives to glioblastoma, a devastating disease, or are currently living with it
Supporting efforts to develop better treatments for glioblastoma that will improve the long term prognosis and quality of life of individuals diagnosed with the disease
Expressing support for the individuals who are battling brain tumors, as well as the families, friends, and caregivers of those individuals
Urging a collaborative approach to brain tumor research, which is a promising means of advancing the understanding and treatment of glioblastoma
Encouraging continued investment in glioblastoma research and treatments, including through the Glioblastoma Therapeutics Network and other existing brain tumor research resources
About Kazia’s Paxalisib

Kazia Therapeutics is at the forefront of potential new therapies for glioblastoma with the drug paxalisib, a brain-penetrant inhibitor of the PI3K / Akt / mTOR pathway. Licensed from Genentech, Inc, in October 2016, paxalisib is currently completing a phase II study which has shown promising indications of clinical efficacy in interim analyses. Paxalisib has been granted orphan designation and Fast Track designation by FDA.

GBM AGILE

The GBM AGILE clinical trial, sponsored by the Global Coalition for Adaptive Research, a 501(c)(3) non-profit organization, commenced recruitment in June 2019. GBM AGILE aims to accelerate the development of new therapies for GBM via an adaptive study design that can explore multiple potential therapies in parallel. To date, three investigational drugs have joined the study: Bayer’s regorafenib, Kintara Therapeutics’ VAL-083, and Kazia Therapeutics’ paxalisib.

To date, GBM AGILE has screened over 650 patients. This progress is expected to accelerate as new sites in new territories come on stream.

For further information on GBM Awareness Day please visit: View Source

On July 21, 2021 Vanda Pharmaceuticals Inc. (Vanda) (Nasdaq: VNDA) reported it will release results for the second quarter 2021 on Wednesday, July 28, 2021, after the market closes (Press release, Vanda Pharmaceuticals, JUL 21, 2021, View Source [SID1234585038]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Vanda will host a conference call at 4:30 PM ET on Wednesday, July 28, 2021, during which management will discuss the second quarter 2021 financial results and other corporate activities. To participate in the conference call, please dial 1-866-688-9426 (domestic) or 1-409-216-0816 (international) and use passcode 5298904.

The conference call will be broadcast simultaneously and archived on Vanda’s website, www.vandapharma.com. Investors should go to the website at least 15 minutes early to register, download, and install any necessary audio software.

A replay of the call will be available on Wednesday, July 28, 2021, beginning at 7:30 PM ET and will be accessible until Wednesday, August 4, 2021, at 7:30 PM ET. The replay call-in number is 1-855-859-2056 for domestic callers and 1-404-537-3406 for international callers. The passcode number is 5298904.

On July 21, 2021 Ascletis Pharma Inc. (HKEX code: 1672) reported that China National Medical Products Administration (NMPA) has approved the Phase III clinical trial application of ASC40 combined with Bevacizumab for treatment of patients with recurrent glioblastoma (rGBM) (Press release, Ascletis, JUL 21, 2021, View Source [SID1234585037]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The Phase III registrational study is a randomized, double-blind, placebo-controlled, multi-center clinical trial in China to evaluate progression-free survival (PFS), overall survival (OS) and safety of patients with rGBM. Approximately 180 patients will be 1:1 randomized to Cohort 1 (oral ASC40 tablet QD + Bevacizumab) and Cohort 2 (matching placebo tablet QD + Bevacizumab).

On May 25, 2021, the Company announced that the clinical trial application for rGBM was accepted for review by China NMPA (Details referring to press release: View Source).

Based on published data, in China, glioblastoma (GBM) represents 46.1% of gliomas and has an incidence rate of approximately 2.85 to 4.56 per 100,000 population per year, suggesting approximately 40,000 to 64,000 new cases of GBM per year. More than 90% GBM patients will relapse after surgery, radiation and chemotherapies. In the United States, GBM represents 56.6% of gliomas and has an incidence rate of approximately 3.21 per 100,000 population per year.

Bevacizumab is the only drug which has been approved for rGMB indication in China as of September, 2020. The data of BELOB Trial indicated that median PFS was three months for patients with rGBM after Bevacizumab treatment.

Lipid metabolism has been reported to play a critical role in various cancers. Fatty acid synthase (FASN) is one of the most important proteins which regulate lipid metabolism. Many solid and hematopoietic tumors overexpress FASN, including rGBM, non-small cell lung, breast, ovarian, prostate, colon, pancreatic cancers, and non-Hodgkin lymphoma.

ASC40 (known as TVB-2640 outside China) is a potent, selective and safe oral small molecule inhibitor of FASN. The data from the Phase II trial have shown that the overall response rate (ORR) for ASC40 (TVB-2640) plus Bevacizumab was 65% including a complete response (CR) of 20% and a partial response (PR) of 45%. Furthermore, the Phase II data indicate that the progression-free survival at six months (PFS6) observed for ASC40 (TVB-2640) plus Bevacizumab was 47%, representing a statistically significant improvement in PFS6 over the historical Bevacizumab monotherapy PFS6 of 16% (BELOB Trial) (P=0.01). ASC40 (TVB-2640) in combination with Bevacizumab was safe and well tolerated in such patient population (ClinicalTrials.gov Identifier: NCT03032484).

"I am thrilled to be the principal investigator leading the ASC40 Phase III trial for rGBM," said Dr. Wenbin Li, Vice Chairman and Secretary General of Glioma Committee of Chinese Cancer Association, Director of the Comprehensive Tumor Treatment Center, Beijing Tiantan Hospital, Capital Medical University, "Based on strong Phase II data, I hope and expect that targeting tumor lipid metabolism with ASC40 will offer a promising therapeutic approach to treat rGBM which is one of the most devastating cancers."

"ASC40 Phase III clinical trial approval by NMPA is a significant milestone for our oncology pipeline since the Company announced, in March this year, an investment escalation in R&D of cancer lipid metabolism and oral checkpoint inhibitors." said Dr. Jinzi J. Wu, Founder, Chairman and CEO of Ascletis. "With the strong momentum from the ASC40 Phase III approval, our talented R&D team is accelerating the clinical development programs of ASC40 for other oncology indications as well as oral PD-L1 small molecule inhibitor programs."

A Phase I clinical trial was completed on 136 patients with advanced tumor from the United States and United Kingdom. The patients were treated with ASC40 (TVB-2640) alone and with a taxane. The data from this Phase I trial have demonstrated FASN target engagement, good safety, pharmacokinetics as well as promising responses of ASC40 (TVB-2640) in patients with advanced solid tumors, particularly in lung cancer with KRAS mutations, ovarian cancer, and breast cancer (ClinicalTrials.gov Identifier: NCT02223247).

There are additional clinical trials of ASC40 (TVB-2640) ongoing in the United States in patients with KRAS mutation non-small cell lung cancer (ClinicalTrials.gov Identifier: NCT03808558) and breast cancer (ClinicalTrials.gov Identifier

On July 21, 2021 Exscientia, an AI-driven pharmatech company with a mission to revolutionise how drugs are discovered, and GT Apeiron Therapeutics （Apeiron）, a Shanghai based company focused on novel oncology drugs, reported a strategic research and development collaboration agreement (Press release, Exscientia, JUL 21, 2021, View Source [SID1234585035]). The collaboration will leverage the patient-centric AI-first capabilities of Exscientia to accelerate the discovery of multiple small molecule therapeutic drug candidates designed to selectively treat aberrant cell cycle driven cancers and build a pipeline of CDK novel therapies. All pipeline products will be equally owned and Exscientia holds an equity stake in Apeiron.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

This expanded collaboration follows the successful design of multiple selective CDK7 compounds. The potent, highly selective, non-covalent CDK7 compounds have demonstrated consistent tumour responses in xenograft models as well as exceptional pharmacokinetics. In addition, using live primary tissue samples from ovarian cancer patients, the CDK7 inhibitors showed both enhanced tumour cell cytotoxicity as well as selectivity over immune cells in the same microenvironment.

"Based on what they have already achieved, Exscientia is clearly the leader in AI-driven drug discovery, and we have witnessed this first-hand in our collaboration so far," stated Dr. Mingxi Li, President of GT Apeiron Therapeutics. "We have been incredibly impressed by the combined power of the AI design and use of patient data to optimize and select molecules that are more likely to give positive effects in the clinic. This joint venture is a significant step in building GT Apeiron’s valuable and robust pipeline of CDK inhibitor drugs and substantially accelerates our early-stage output and progression towards being a clinical stage biotech company."

"We are driven to bring drugs to market that make a difference for patients," says Andrew Hopkins, chief executive officer of Exscientia. "Apeiron bring a focus and an expertise on the biological basis of multiple cancers and helps us to create better drugs for better outcomes in the clinic and beyond. This collaboration has already proven to be capable of delivering potential drug candidates with promising patient-relevant data, and we look forward to extending that into a portfolio of multiple clinical assets."

On July 21, 2021 Omniscient Neurotechnology ("o8t"), a pioneering brain mapping software company, reported that it has received U.S. Food and Drug Administration (FDA) 510(k) clearance for Quicktome, a digital brain mapping platform that allows neurosurgeons to visualize and understand a patient’s brain networks prior to performing life-changing brain surgery (Press release, Omniscient Neurotechnology, JUL 21, 2021, View Source [SID1234585033]). This announcement occurs on Glioblastoma Awareness Day, a day dedicated to increasing public understanding of the most common, complex, treatment-resistant, and deadliest type of brain cancer.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

In addition to FDA clearance in the United States, Quicktome has also received Health Canada approval as well as regulatory clearance by the Therapeutic Goods Administration (TGA) of Australia.

Quicktome is a neurosurgical planning software which incorporates "connectomics", the field of understanding brain connectivity, into routine neurosurgical planning. Critically, by visualizing networks that are responsible for complex functions such as language, movement, and cognition, Quicktome assists neurosurgeons in making more informed decisions and reduces surgical uncertainty.

Utilizing cloud computing for large-volume data processing, and intuitive browser-based interfaces, Quicktome aims to streamline the process for neurosurgeons. The digital platform furthermore enables multidisciplinary collaboration in hospitals, and greater insight on a patient before and during surgery.

"We are thrilled to receive regulatory clearance for Quicktome. This is the first digital mapping platform designed to incorporate brain connectome data for neurosurgeons to improve patient outcomes," said Stephen Scheeler, CEO, Omniscient Neurotechnology. "Until now, the tools that neurosurgeons have relied on have been no match for the brain’s complexity. Quicktome breaks information down into actionable insights to inform the impact each incision will have on the patient. Since its authorization, physicians are already relying on this technology to guide surgical planning. This is a significant milestone for Omniscient and, more importantly, begins a new era for neurosurgery for patients and physicians."

"I wish I had technology like this when I started practicing," said Dr. Michael Sughrue, Chief Medical Officer, Omniscient Neurotechnology, a neurosurgeon who has completed over 3,000 brain tumor removals. "When I learnt about connectomics, I realized the biggest impact I could make to the field of neurosurgery was to make Quicktome a routine source of insight for all neurosurgeons. With this ground-breaking technology, our hope is for a better quality of life for patients and families after brain surgery."

"Omniscient is the first to harness the power of brain connectomics for use in neurosurgery," said Stephane Doyen, Chief Data Officer at Omniscient Neurotechnology. "Big data is changing everything we know about the brain. Modern neuroscience has shown that we are our brain networks, which control everything from movement to speech. Understanding how the brain is connected and what these connections mean will drastically help us better deliver healthcare, starting with neurosurgery."