On June 30, 2022 Kazia Therapeutics Limited (NASDAQ: KZIA; ASX: KZA), an oncology-focused drug development company, reported an update on recent progress with its two pipeline assets, paxalisib and EVT801, and on recent corporate financing activity (Press release, Kazia Therapeutics, JUN 30, 2022, View Source [SID1234616396]).

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Key Points

EVT801 phase I study protocol has cleared third dose level and is recruiting well.

GBM AGILE pivotal study has opened in France, the fourth country to commence recruitment to the paxalisib arm.

Phase I study of paxalisib in combination with radiotherapy for treatment of brain metastases at Memorial Sloan Kettering Cancer Center has been accepted for presentation at an upcoming academic conference in Q3 CY2022.

ATM financing facility has realized gross proceeds of US$ 2,956,036 for the period ending June 2022, at an average price of $6.08 (approximately AU$ 0.88 per share).

Kazia CEO, Dr James Garner, commented, "Despite a challenging first half equity market for biotech companies, Kazia has continued to make good progress. The GBM AGILE pivotal study is progressing well, and appears on track for data in 2H CY2023, as anticipated. We have been pleased in the first half to see new data presented and milestones delivered from several projects, and we expect that pace to continue and increase during the second half."

EVT801 Phase I Study

The phase I study of EVT801 in patients with advanced cancer continues to recruit well. The drug has recently completed the third of a potential eight dose levels and is anticipated to open recruitment to the fourth dose cohort in the near future. To date, the drug appears generally well-tolerated. Depending on how many dose cohorts are required to establish a maximum tolerated dose (MTD), interim data from the study is anticipated in 2H CY2022 or 1H CY2023.

GBM AGILE

More than forty sites are currently open to the paxalisib arm of GBM AGILE. The first site to commence recruitment to the paxalisib arm in France opened in June 2022, making France the fourth country to join the study, alongside the United States, Canada, and Switzerland.

In January 2022, the Global Coalition for Adaptive Research (GCAR), the sponsor of GBM AGILE, stated that over a thousand patients had been screened to the study, with enrolment rates approximating 0.75 to 1.00 patients per site per month, which is around four times higher than would generally be expected in a clinical study of glioblastoma.

Kazia continues to work closely with GCAR and Simcere Pharmaceutical to open the study in China. Public health measures in some Chinese cities relating to the ongoing COVID pandemic have had a modest impact on operational activities, but it is currently anticipated that the study will open in China during 3Q CY2022.

Paxalisib in Brain Metastases

In June 2022, the company announced that a multi-drug, genomically-guided study in brain metastases run by the Alliance for Clinical Trials in Oncology had seen the paxalisib arm graduate to an expansion cohort in patients with breast cancer brain metastases, having seen positive efficacy signals in the initial exploratory cohort. Paxalisib continues to recruit to the exploratory cohort in two other patient subgroups.

Interim data from a phase I study of paxalisib in combination with radiotherapy for patients with brain metastases run by Memorial Sloan Kettering Cancer Center has been accepted for an oral presentation at an upcoming international conference in Q3 CY2022. Kazia looks forward to sharing data from this presentation as soon as it is available.

Financing

In May 2022, Kazia established a NASDAQ-based at-the-market financing facility with Oppenheimer and Company. For the period ending June 30, 2022, the company has issued 486,281 American Depository Shares (ADSs) under this facility, at an average price of $6.08, for total gross proceeds of US$ 2,956,036 (approximately AU$ 4,256,691). Of note, these shares have been issued at no discount, with no warrant coverage, and with transaction fees approximately half of those associated with traditional financing.

On June 30, 2022 Astellas Pharma Inc. (TSE: 4503, President and CEO: Kenji Yasukawa, Ph.D., "Astellas" ), Nitto Denko Corporation (TSE: 6988, President: Hideo Takasaki, "Nitto"), and M. Heart Co., Ltd. (President and CEO: Yoshimi Mizunuma, "M. Heart") reported that the three companies have entered into an agreement for a sales pilot of disposable Holter ECG device "EG HolterTM" for the Japan market (Press release, Astellas, JUN 30, 2022, View Source [SID1234616392]). Based on this agreement, Astellas has established an e-commerce site for healthcare professionals and will start the sales pilot of the device today.

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This is Astellas’ first initiative in Rx+ program to promote a product through an e-commerce site. After verifying the business model through the sales pilot, Astellas will move to full-scale marketing of the product.

EG Holter is a holter electrocardiograph designed and developed by Nitto. Because it is a disposable device, it is hygienic and does not require maintenance. It is 6 mm thick,11 g in weight, has no cords, and is water resistance (IPX4). It is easy to attach and remove.

Data obtained by EG Holter will be analyzed by "MYHOLTER II", which is software for the holter analyzer jointly developed by Astellas and M. Heart. MYHOLTER II is a program that analyzes holter ECG data with a proprietary analytical algorithm using artificial intelligence (AI). M. Heart has obtained cerification for EG Holter and MYHOLTER II as medical device (Class II).

Astellas, Nitto and M. Heart have come together to create a simple, convenient, patient-centered solution for patients and medical institutions.

Medical costs for heart diseases and other cardiovascular diseases in Japan exceed 6 trillion yen per year―the highest of any disease category1. Of particular concern is cardiogenic cerebral embolism, which occurs when a thrombus that formed in the heart blocks an artery in the brain or neck. This has a high mortality rate (20%) and often results in severe sequelae, such as a bedridden state (40%)2. Atrial fibrillation is said to be the cause of cardiogenic cerebral embolism in 3 out of 4 cases2. Early detection of atrial fibrillation is an important social issue.

By providing a total solution that involves combining convenient ECG testing using the EG Holter and the data analysis using MYHOLTER II, Astellas, Nitto, and M. Heart propose to enable early detection and appropriate treatment of atrial fibrillation, a condition estimated to affect approximately 700,000 patients in Japan2, and contribute to the prolongation of healthy life expectancy.

On June 29, 2022, ImmuneOnco Biopharmaceuticals (Shanghai) Inc. (hereinafter referred to as "ImmuneOnco" and the company) announced that SIRPaFc recombinant protein targeting human CD47 ，IMM01， combined with azacitidine (AZA) in the treatment of relapsed and refractory acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS) Phase Ib/II clinical study (clinical research project number: IMM01-02), completed the first patient enrollment for first dosing (Press release, ImmuneOnco Biopharma, JUN 29, 2022, View Source [SID1234655663]). The infusion process went smoothly. The subject is a 25-year-old female AML patient with disease progression after prior chemotherapy.

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The IMM01-02 clinical trial project was launched simultaneously in more than 20 hospitals across the country to evaluate the safety and efficacy of IMM01 in combination with AZA for AML and MDS indications.

Dr. Tian Wenzhi , Founder, Chairman and CEO of ImmuneOnco, feels confident in clinical trial research of IMM01.

"I am very pleased to know that our IMM01 combined with azacitidine for relapsed and refractory AML and MDS indications has completed the first patient enrollment and dosing. Due to the unique design of the molecular structure, IMM01 does not bind to erythrocytes and prevent it from causing RBC clogs and severe anemia. what is more, due to the glycosylation of Fc modification, the immunogenicity of the drug is greatly reduced, so that the PK and the bioavailability of the drug is significantly improved. So far, based on preliminary data, 1 complete remission (CR), 2 partial remissions (PR), and 8 stable disease (SD) occurred in different dose groups in open-labeled IMM01 mono-drug therapy trials."

"We will work closely with clinical experts and subjects to accelerate the clinical trial research of IMM01 and step the pace faster to launch IMM01 market available to benefit relapsed and refractory cancer."

On June 29, 2022 ReCode Therapeutics, a genetic medicines company using superior delivery to power the next wave of messenger RNA (mRNA) and gene correction therapeutics, reported the close of a Series B extension financing round, co-led by new investors, Leaps by Bayer, the impact investment unit of Bayer AG, AyurMaya, an affiliate of Matrix Capital Management, and with participation from Amgen Ventures (Press release, ReCode Therapeutics, JUN 29, 2022, View Source;utm_medium=rss&utm_campaign=recode-therapeutics-to-expand-next-generation-delivery-platform-and-diversify-genetic-medicines-pipeline-with-oversubscribed-series-b-financing-totaling-200m [SID1234619533]). With the additional $120M in new financing, the company has secured a total of $200M in Series B funding. In connection with the financing, Alan Colowick, M.D., M.P.H., managing director of Matrix, and Rakhshita Dhar, senior director of Venture investments Health at Leaps by Bayer, will join ReCode’s Board of Directors.

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"At AyurMaya, we help build companies that have the potential to transform the therapeutic landscape for patients. ReCode has an impressive combination of groundbreaking science, a highly accomplished leadership team and investor base, and the opportunity to have an immense impact on accelerating precision genetic medicines through delivery to disease-relevant cells," said Alan Colowick, M.D., M.P.H, Matrix Capital Management. "I am delighted to join ReCode’s Board during this important growth trajectory for the company and am eager to work with the leadership team to expand ReCode’s next generation genetic medicines delivery platform and diversify its pipeline."

The initial Series B financing announced in October 2021 was co-led by Pfizer Ventures and EcoR1 Capital and included Sanofi Ventures, Orbimed, Vida Ventures, MPM Capital, Colt Ventures, Hunt Technology Ventures L.P., Osage University Partners, Tekla Capital Management LLC, Superstring Capital, and NS Investment. Proceeds from the financing will be used to fund the diversification of ReCode’s pipeline into central nervous system (CNS), liver, and oncology indications, while continuing to advance ReCode’s lead mRNA programs for primary ciliary dyskinesia (PCD) and cystic fibrosis (CF) into the clinic. The funds also will be used to advance development of ReCode’s platform to deliver a wider range of genetic medicine cargoes, including additional gene correction modalities and small interfering RNA (siRNA) therapies, to a wider range of target cell types in a predictable and programmable fashion.

"The successful administration of billions of doses of mRNA COVID-19 vaccines, and continued progress with novel RNA and gene correction therapeutics, have catapulted us into a new era of possibility for genetic medicines. We are harnessing that potential with our novel selective organ targeting (SORT) lipid nanoparticle (LNP) delivery platform which is engineered with unique properties to enable delivery of genetic therapeutics directly to the organs and cells most impacted by disease, offering improved efficacy and potency. This potentially game-changing precision medicine technology creates vast possibilities for genetic medicines which have been limited by current technologies," said Shehnaaz Suliman, M.D., MBA, M.Phil., chief executive officer and board member, ReCode Therapeutics. "With the infusion of new capital and the addition of AyurMaya, an affiliate of Matrix Capital Management, and Leaps by Bayer to our high-caliber board, we are confident that our team will successfully drive ReCode’s mission to develop new precision genetic medicines enabled by superior delivery."

The capability to precisely target specific organs and cell types is important for maximizing the efficacy of genetic medicines and limiting potential adverse effects. Additionally, as evidenced with the mRNA COVID vaccines and novel RNA and gene correction therapeutics, LNP packaging enables redosing. For people with genetic diseases such as CF that require potentially lifelong therapy, this means LNP delivered therapeutics can be administered repeatedly over time, potentially without significant immunogenicity.

"The ability to directly target specific organs and cells beyond the liver remains a key challenge for genetic medicines," said Juergen Eckhardt, head of Leaps by Bayer. "At Leaps by Bayer, we strongly focus on finding breakthrough solutions that have the potential to make a meaningful difference for people with rare genetic diseases, and we believe ReCode is a unique company in our industry whose innovations have the potential to fundamentally shift an entire area of genetic medicine."

In May 2022, ReCode presented preclinical data from its two lead programs in PCD and CF at the American Thoracic Society (ATS) 2022 International Conference. These data demonstrated that selective organ targeting (SORT) LNP-formulated therapeutic candidates for PCD and CF can be precisely delivered directly to disease-relevant cells without significant exposure to other tissue, effectively releasing the encapsulated genetic cargo, and expressing the correct proteins at relevant levels. The company plans to advance to investigational new drug (IND) filings for PCD and CF in late 2022 and mid-2023, respectively.

About the SORT LNP Platform

ReCode is differentiated by its first-in-class modular genetic medicines delivery platform. The company’s selective organ targeting (SORT) lipid nanoparticle technology (LNP) platform is the foundation for its pipeline. Pioneered by co-founder Professor Daniel J. Siegwart, Ph.D., of the University of Texas, and described by Nature as one of the "Seven Technologies to Watch in 2022," ReCode’s SORT LNP platform is an innovation beyond the lipid delivery system used by the mRNA COVID vaccines and novel RNA and gene correction therapeutics.

LNPs are used to package and deliver genetic cargo such as mRNA. When delivered into the blood, first-generation LNPs primarily are taken up by the liver, which limits their utility for broad therapeutic applications. ReCode’s SORT LNPs are engineered with a biochemically distinct fifth lipid to help the body "sort" and direct the LNPs to other targeted organs such as the lung and spleen, with the ability to bypass the liver, if desired.

Beyond its highly selective targeting capability, ReCode’s SORT LNP platform is further distinguished by its versatility in both mode of administration (including IV, inhaled, subcutaneous, intramuscular, and intrathecal), and the diversity of genetic cargo that can be delivered (including mRNA, siRNA, DNA, gene correction components and mixed cargoes). Together, these qualities offer vast opportunities to address a wide range of unmet medical needs through a precision medicine approach that delivers the right medicine to the right organs and cells using the optimal mode of administration.

On June 29, 2022 Roche (SIX: RO, ROG; OTCQX: RHHBY) reported the launch of the BenchMark ULTRA PLUS system, its newest advanced tissue staining platform (Press release, Hoffmann-La Roche, JUN 29, 2022, View Source [SID1234616493]). The system enables quick and accurate test results so clinicians can make timely decisions regarding a patient’s care journey.

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"With the BenchMark ULTRA PLUS, we are pleased to offer an advanced tissue staining platform, co-developed with lab professionals," said Thomas Schinecker, CEO of Roche Diagnostics. "Waiting for a diagnosis is often one of the most stressful times for patients. This system enables pathologists to provide quick and accurate results that help inform patient treatment options."

Cancer and other abnormal cells can be characterised by biochemical markers from within the cells. By applying chemical solutions to tissue on glass slides with the BenchMark ULTRA PLUS, a healthcare professional can identify these markers to determine the presence or absence of key drivers that feed the unhealthy cells and, in many cases, the type of therapy that could be used to combat them.

The new BenchMark ULTRA PLUS system continues the evolution of the BenchMark series, which revolutionised cancer diagnostics by fully automating processes that used to be performed manually, one slide at a time.

Lab personnel will be able to manage their activities more efficiently as a result of simplified software and streamlined productivity and quality control. These enhancements can help support the quicker delivery of test results for patients who are waiting for a diagnosis.

The new system has several innovations such as new intuitive software, remote monitoring features, an integrated touchscreen for a more optimised user experience, and a more environmentally sustainable waste system and product packaging.

The BenchMark ULTRA PLUS system will be available to European Union countries in July 2022 and to the United States and in the Asia-Pacific market in September 2022, followed by a worldwide roll out in 2023.

About the BenchMark ULTRA PLUS system
The BenchMark ULTRA PLUS system (including hardware, software and assays) leverages the success of the current BenchMark series of IHC/ISH advanced staining platforms. It is built upon the proven technology of the BenchMark ULTRA system to deliver an enhanced lab experience. The BenchMark ULTRA PLUS system provides optimised workflow and flexible solutions, helping the anatomic pathology lab manage their activities efficiently and confidently, while selecting options that work best for their lab.

BenchMark ULTRA was the first advanced staining instrument to provide true, single-piece flow. BenchMark ULTRA PLUS pushes the envelope even further with its improved VENTANA System Software, which enhances quality control, inventory management and overall user experience due to its modern architecture. The BenchMark ULTRA PLUS incorporates several additional innovative features based on customer feedback, including remote monitoring with a connected device, new indicator lights, a retractable work surface, new slide drawers (that mitigate fluid ingress/egress), a new integrated touchscreen and more environmentally friendly waste segregation and degradation. Sustainable packaging uses 40% fewer raw materials and weighs less to provide a smaller carbon footprint while being transported. The system is manufactured using innovative and efficient assembly practices to ensure product quality and delivery.