On September 22, 2020 Kazia Therapeutics Limited (ASX: KZA; NASDAQ: KZIA), an Australian oncology-focused biotechnology company, reported that it has entered into a collaboration with Dana-Farber Cancer Institute (DFCI) in the United States, to investigate the use of Kazia’s investigational new drug, paxalisib (formerly GDC-0084), in primary central nervous system (CNS) lymphoma, a potential new indication for the drug (Press release, Kazia Therapeutics, SEP 22, 2020, View Source [SID1234565464]).

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Key Points

Lymphoma is a cancer of white blood cells. It occurs in the lymphatic system and can spread almost anywhere in the body; primary CNS lymphoma (PCNSL) occurs exclusively in the brain and central nervous system

The PI3K inhibitor class is well validated in lymphoma outside the brain; three of the four FDA-approved PI3K inhibitors are treatments for forms of lymphoma, but they are assumed ineffective for PCNSL since they cannot cross the blood-brain barrier

DFCI will initiate an open-label phase II clinical trial of paxalisib in PCNSL

The study is expected to recruit up to 25 patients, taking up to 2 years to complete

Kazia will provide support including study drug and a financial grant

This study will be the sixth ongoing clinical trial of paxalisib in brain cancer

Dana-Farber Cancer Institute (DFCI) is a world-leading cancer treatment and research centre, based in Boston, Massachusetts. It is a principal teaching affiliate of Harvard Medical School and has been designated a Comprehensive Cancer Center by the US National Cancer Institute. DFCI participates in as many as 600 clinical trials at any given time and has been an important contributor to the development of many important new cancer therapies.

Kazia CEO, Dr James Garner, commented, "this is an exciting new opportunity for the paxalisib program. We are delighted to support the team at Dana-Farber to explore the potential for paxalisib to benefit patients with PCNSL. Dana-Farber is one of the world’s leading centres of excellence in this disease, so we are immensely fortunate to be working with them. We are pleased also to see a new and important target added to the broader paxalisib clinical program, and we look forward to seeing the project commence."

Kazia’s financial support to the study will use a portion of the funds contributed by shareholders in the Share Purchase Plan (SPP) conducted in May 2020.

Primary CNS Lymphoma

Lymphoma is a haematological malignancy (blood cancer) that originates from lymphocytes, a type of white blood cell involved in the immune system. PCNSL is a specific form of the disease that originates in the brain and central nervous system.

Three of the four PI3K inhibitors approved by the US Food and Drug Administration (FDA) are treatments for various forms of lymphoma, provide a strong validation for PI3K as a target in this disease. Paxalisib is the only PI3K inhibitor in mainstream development with the ability to penetrate the blood-brain barrier, and as such has a unique rationale for development in PCNSL.

PCNSL accounts for approximately 4% of brain tumours, and the incidence is increasing with time. Patients are typically in their 60s or older, and the disease is slightly more common in men.1 The mainstays of treatment comprise chemotherapy and radiotherapy, but recurrence is common and only approximately 30% of patients remain alive five years after diagnosis.2 Many of the drugs used to treat lymphoma elsewhere in the body are ineffective in PCNSL due to their inability to cross the blood-brain barrier.

Clinical Trial Design

Dana Farber Cancer Institute will launch a single-arm phase II clinical trial in patients with relapsed or refractory PCNSL, who are resistant to existing treatments. The primary endpoint will be to assess efficacy via overall response rate (ORR), which measures the ability of paxalisib to shrink tumours. Safety and other efficacy endpoints will also be captured. The study will also examine tissue and cerebrospinal fluid samples to identify potential predictors of response.

The principal investigator for the study is Dr Lakshmi Nayak, Director of the CNS Lymphoma Center at Dana-Farber Cancer Institute. Dr Nayak is an Assistant Professor of Neurology at Harvard Medical School and a board-certified neuro-oncologist. Her research interests focus on metastatic brain cancer, glioblastoma, and PCNSL, and she is extensively published in the field of brain cancer. She has been an investigator for multiple clinical trials of experimental drugs in this disease area.

Commencement of recruitment to the study is expected in early CY2021, but is subject to receiving necessary approvals from FDA and from institutional review boards. The study will be listed on clinicaltrials.gov closer to the commencement of recruitment.

On September 22, 2020 Synaffix B.V., a biotechnology company enabling antibody-drug conjugates (ADCs) with best-in-class therapeutic index, reported that it has won the "Best ADC Platform Technology" category at the 2020 World ADC Awards ceremony (Press release, Synaffix, SEP 22, 2020, View Source [SID1234565463]).

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Synaffix’ ADC platform consists of GlycoConnect, HydraSpace and toxSYN which comprise site-specific technology and payloads to enable best-in-class ADCs.

Synaffix was presented the award during the 2020 World ADC Digital event. The finalists were shortlisted through a voting pool of over 1,000 individuals, with a panel of distinguished, independent industry experts from across the ADC field, assessing each finalist to decide the winners.

The judging panel highlighted the following three cornerstone features of the Synaffix ADC platform:

Consistent delivery of highly competitive ADC product candidates
Strong commercial and scientific validation (over $420m in out-licensing deals,at least six ADCs in development with two in clinical trials)
Compatible and easy to use with any antibody

On September 22, 2020 AQILION AB reported that TAK1 as the target of the internal drug discovery program Alnitak and will present data from the discovery phase at the international partnering event BIO-Europe 2020 Digital in October (Press release, Aqilion, SEP 22, 2020, View Source [SID1234565462]). Alnitak was initiated as an internal program last year with focus on chronic inflammation and autoimmune diseases in addition to genetically driven cancers.

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The internally generated Alnitak program is aimed at discovering and developing small molecule inhibitors of TAK1. The target TAK1 has been shown to act as a master regulator of inflammatory signaling. The Aqilion team, consisting of experienced former AstraZeneca R&D management team members and scientists, has identified TAK1 as a highly promising drug target. A TAK1 inhibiting drug has the potential to treat inflammatory and autoimmune diseases in addition to certain genetically driven cancers.

Among the key inflammatory processes is the formation of a multi-protein complex called inflammasome. The inflammasomes are part of the innate immune system and they play a vital role that help to recruit immune cells to sites of infection and inflammation. Dysfunctional inflammasomes are involved in harmful inflammations, which can become chronic in many diseases. NLRP3 is the most studied inflammasome in the scientific litterature to date. Recent publications have shown that TAK1 functions as a central mediator of NLRP3 activation in human cells.

Aqilion believes that new research and knowledge regarding human immunology and availability of novel research models, re-evaluates and positions TAK1 as an ideal drug target.

"Using structure-based drug discovery methods in collaboration with leading research laboratories, the project has already developed proprietary best in class inhibitors with excellent druglike properties. This is Aqilion’s first internal project to deliver results since we initiated the implementation of our new strategy with a focus on chronic inflammatory and immunological driven diseases. I am proud of our team and collaborators and very pleased to acknowledge that we have reached an important milestone in the development of Aqilion as a biotech company," says Sarah Fredriksson, CEO of AQILION AB.

On September 21, 2020 Biocure Technology Corp. ("CURE" or the "Company") (CSE:CURE; OTCQB: BICTF) BiocurePharm, Korea ("BPK"), a subsidiary of Biocure Technology Inc. ("CURE") reported that BPK has entered into a non-binding MOU with S&B Biopharm ("SBB") located in Sofia, Bulgaria (Press release, Biocure Technology, SEP 21, 2020, View Source [SID1234628816]). This agreement will allow BPK to locally manufacture CD-19 CAR T in Bulgaria and sell to the Eastern European market, including Poland, Turkey and Bulgaria. Expansion into the European market could assist with bringing ALL patients more affordable solutions of CAR T Therapy.

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As part of the agreement, SBB will be required to raise funds the equivalent of USD12Million to cover the clinical trial costs along with the GMP manufacturing facility. Upon the execution of the definitive agreement SBB will have 6 months to allocate the funds and begin the clinical trial. At the completion of the agreement, BPK will be required to transfer its manufacturing technology of CD19 CAR T to the JV for the designated markets, to be defined in the definitive agreement. The manufacturing technology of Lenti virus shall be transferred by a separate agreement afterwards. The initial structure of the JV will be 51:49 between BPK and SBB.

Dr. Sang Mok Lee, CEO and President of Biocure and BPK, states "This MOU is the very first step for the Company to advance it’s entrance into the European market with its CAR T technology. We are about to start a clinical trial in Korea and are confident that if we have positive outcomes from the Korean clinical trial, this should enable the European process to be faster and more efficient. We strongly believe that our business model could enable European ALL patients with an affordable CAR T Therapy to save more lives. We continue to work hard to make this happen as soon as possible."

On September 21, 2021 Oncology Venture A/S ("OV") reported that it will change its company name to Allarity Therapeutics ("Allarity" or the "Company") and will significantly restructure its Board of Directors to align with the company’s current and long-term strategy (Press release, Allarity Therapeutics, SEP 21, 2020, View Source [SID1234586756]). Both the proposed name change and appointment of new Board members are subject to approval of shareholders at the upcoming Extraordinary General Meeting (EGM) planned to be convened on 7 October 2020.

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Following the proposed restructuring, Allarity’s Board of Directors will comprise five members, including existing Directors Mr. Steve Carchedi (CEO), Mr. Duncan Moore (Chairman), and Dr. Magnus Persson, and new members in both the U.S. and Scandinavia, including Mr. Søren Gade (a current member of European Parliament and former Minister of Defense in Denmark) and Ms. Gail Maderis (current CEO of Antiva Biosciences, Inc., Board member of Valitor, Inc., and former CEO of Five Prime Therapeutics, Inc.). New Board members will receive warrants in the Company as part of their compensation package.

The changes to the Company name and composition of its Board of Directors are part of a planned strategic shift towards commercialization for the U.S. oncology and financial markets, while the company maintains its roots and laboratory headquarters in Denmark, which continue to be directly overseen by Dr. Steen Knudsen, Chief Scientific Officer and Founder, and Mr. Thomas Jensen, Senior Vice President of Information Technologies and Founder. The Hørsholm, Denmark facility will serve as the Research & Development global headquarters for the company’s DRP companion diagnostic platform. As part of this strategic shift, current Chief Financial Officer (CFO) Mr. Henrik Moltke will be departing the Company to pursue other opportunities. Mr. James G. Cullem, currently Senior Vice President of Corporate Development, will serve as interim CFO, while the Company conducts an immediate search for a U.S.-based, CFO with Nasdaq experience.

Steve Carchedi, CEO of the Company, noted "Our new company name, Allarity Therapeutics, better reflects our focus on developing promising new cancer therapeutics, together with DRP companion diagnostics, to match cancer patients with the best therapeutic options for their particular cancers, thereby realizing the promise of personalized medicine. We are also very enthusiastic about the planned restructuring of our Board of Directors to strengthen the strategic oversight of the Company and support our Executive Team in achieving our goals. I am thrilled to have Gail Maderis and Søren Gade join our Board, as both will provide valuable connections for us related to international cancer drug development while further expanding the geographic footprint of our Board to both sides of the Atlantic."

Mr. Carchedi further commented, "I also want to thank Henrik for his leadership over the past year. He has been instrumental in adjusting our cost base, cleaning up our balance sheet, and successfully changing our financing strategy towards low-cost equity financing. Henrik’s departure is bittersweet for the Company, but will allow us to find a new CFO who is an ideal match for the many tasks ahead on our continued journey towards bringing healthcare therapies to market for the benefit of patients."

Duncan Moore, Board Chairman of the Company, commented "We are grateful for the efforts and contributions of our prior Board members, Steen Knudsen, Carani Sanjeevi, and Frank Knudsen, as well as CFO Henrik Moltke, and we look forward to working alongside our new Board members, Gail Maderis and Søren Gade, to advance the Company’s mission to improve cancer patient care by realizing truly personalized medicine."