On May 15, 2025 Kazia Therapeutics Limited (NASDAQ: KZIA) ("Kazia" or the "Company"), an oncology-focused drug development company, reported a summary of its recent progress across its business and also provided a business update (Press release, Kazia Therapeutics, MAY 15, 2025, View Source [SID1234653171]).

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"During the first quarter of 2025, we made significant progress both with respect to our clinical programs and corporate strategy," said John Friend, M.D., Chief Executive Officer of Kazia Therapeutics. "We recently advanced paxalisib into multiple new clinical trials, reached the last patient follow-up in a Phase 1 trial of EVT801 for the treatment of solid tumours, and raised $3 million in new capital, including $1 million in non-dilutive funding."

"Looking ahead, we anticipate a busy year for Kazia, as both paxalisib and EVT801 continue to advance across multiple clinical trials based on strong investigator interest. We are excited about the potential of our advanced-stage pipeline candidates to address significant areas of unmet need within oncology, and we look forward to providing additional updates on our progress throughout 2025."

Pipeline – paxalisib

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On February 20, 2025, the Company announced a research grant awarded from The Michael J. Fox Foundation for Parkinson’s Research (MJFF) to fund research between The Hebrew University of Jerusalem (Hebrew University) and Kazia to explore the therapeutic potential of paxalisib as a treatment for Parkinson’s disease (PD). The grant will fund collaborative preclinical studies at Professor Ronit Sharon’s lab (Hebrew University) aimed at establishing an operational link between a specific pathway in the pathophysiology of PD and paxalisib. The research will assess the impact of paxalisib on mouse survival, motor and non-motor performances, as well as specific biochemical, pathological and molecular disease biomarkers that will be determined in brains of treated mice. Data from this research is expected to provide valuable insights into its potential activity for the treatment of PD.

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In February 2025, Kazia executed an agreement to evaluate paxalisib in the next-Generation aGile Genomically Guided Glioma platform (5G) study. The 5G study is an academic trial conducted by the Drug Development Unit – Investigator Initiated Team and sponsored by the Institute of Cancer Research, London and fully funded by Cancer Research UK and the Australian charity, Minderoo Foundation. Every patient in this study will have their genome sequenced, enabling researchers to target their treatment with greater precision. Patients with PI3k/mTOR related mutations will be enrolled to receive paxalisib once a day.

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On January 30, 2025, the Company announced the regulatory approval and launch of a clinical trial evaluating the combination of paxalisib and immunotherapy in patients with advanced breast cancer. This novel treatment combination offers what is believed to be a unique approach to targeting this highly aggressive and treatment-resistant type of breast cancer. The ABC-Pax (Advanced Breast Cancer – Paxalisib) study is the first known trial conducted to assess the safety and efficacy of paxalisib in combination with KEYTRUDA (pembrolizumab) or LYNPARZA (olaparib) in women with triple negative breast cancer. ABC-Pax is a multi-center, open-label phase 1b study that will enroll 24 patients who will receive the combination therapy for up to 12 months. Currently the study is open and actively screening patients at The Royal Brisbane and Women’s Hospital, Gold Coast University Hospital and Sunshine Coast University Hospital in Queensland, Australia with plans to open up to 4 additional sites in Australia.

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On December 31, 2024, the Company provided a regulatory update on paxalisib for the treatment of glioblastoma (GBM) following its Type C clinical meeting with the United States Food and Drug Administration (FDA). The FDA’s current position is that data on overall survival (OS) would generally not be appropriate for accelerated approval but could be considered to support a traditional/standard approval. The FDA further commented that the secondary endpoint OS data from the GBM-AGILE study are supportive and informative for designing and executing a pivotal registrational study in pursuit of a standard approval. Importantly, the Company aligned with the FDA on key aspects of the design of a proposed registrational/pivotal phase 3 study, including patient population, primary endpoint, and the comparator arm to be used.

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The study will be a randomized, controlled study of paxalisib versus standard of care (SOC) in patients with newly diagnosed unmethylated glioblastoma to determine Overall Survival as well as other parameters of clinical efficacy, safety and tolerability. Approximately 366 patients will be enrolled over 14 months into the study with a 1:1 treatment ratio between the paxalisib and SOC (temozolomide) arm. We anticipate engaging roughly 50 clinical sites across the globe (North America, UK, Europe, Asia-Pacific) for this study. The Kazia team is exploring various bids from Contract Research Organizations (CROs) in parallel to discussions with strategic partners and cooperative groups to participate or fully fund the trial.

Pipeline – EVT801

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In November 2024, the last patient completed follow-up in a Phase 1 study (NCT05114668) evaluating EVT801 for the treatment of patients with histologically-confirmed advanced or metastatic solid tumours, unresponsive to standard treatment, or for whom no standard treatment is available or appropriate. We anticipate receiving the final data in 2Q2025 and presenting data later this year at an international medical congress.

Corporate

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On May 12, 2025, the Company received a notification (the Notification) from the Listing Qualifications Staff of the Nasdaq Stock Market LLC (Nasdaq) notifying the Company that that from March 28, 2025 to May 9, 2025, the Company’s Market Value of Listed Securities (MVLS) was below the minimum of $35 million. The Notification has no immediate impact on the Company’s operations or listing and Kazia’s American Depositary Shares (ADSs) will continue to trade on the Nasdaq Capital Market under the ticker "KZIA". In accordance with Nasdaq Listing Rule 5810(c)(3)(C), the Company has 180 calendar days to regain compliance with the MVLS Requirement. The Notification states that, to regain compliance with the MVLS Requirement, the Company’s MVLS must close at $35 million or more for a minimum of ten consecutive business days during the compliance period ending on November 10, 2025. Kazia has no intention of delisting and is currently exploring options to regain compliance, which include raising additional capital and possible merger and acquisition-related strategies. While the Company is exercising diligent efforts to maintain the listing of its ADS on Nasdaq, there can be no assurance that the Company will be able to regain or maintain compliance with the applicable continued listing standards set forth in the Nasdaq Listing Rules.

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Effective April 17, 2025, Kazia changed the ratio of its ADSs to Ordinary Shares from one ADS representing one hundred Ordinary Shares to one ADS representing five hundred Ordinary Shares. This adjustment, equivalent to a one-for-five reverse ADS split, was necessary to maintain compliance with Nasdaq’s minimum bid price requirement.

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On March 31, 2025, the Company announced the sale of all intellectual property and trademarks rights to Cantrixil for USD $1 million. In March 2021, Vivesto licensed the exclusive global development and commercialization rights for Cantrixil from Kazia Therapeutics. Having decided not to pursue the development of Cantrixil in ovarian cancer, as originally anticipated under the license, Vivesto is currently exploring Cantrixil preclinically for the treatment of hematological cancers.

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On February 26, 2025, CEO Dr John Friend bought 8,000 ADSs (split adjusted). The ADSs were bought at a split-adjusted price of $4.2465 per ADS for a total transaction of $33,972.

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On January 14, 2025, the Company announced the closing of a registered direct offering with existing fundamental healthcare investor, Alumni Capital LP. The gross proceeds to the Company from the offering were approximately $2.0 million, before deducting the placement agent’s fees and other offering expenses payable by the Company.

On May 15, 2025, Xuanzhu Biopharm reported the latest announcement released by the National Medical Products Administration attracted widespread attention in the oncology community and the pharmaceutical market (Press release, Xuanzhu Biopharmaceutical, MAY 15, 2025, View Source [SID1234653189]). Bireociclib tablets (trade name: Xuanyuening), a Class 1 anti-tumor new drug independently developed by Xuanzhu Biopharmaceutical was officially approved for marketing. This innovative drug is indicated for two important conditions in HR+/HER2- breast cancer. It not only injects new vitality into the domestic breast cancer treatment field but also brings new hope to a large number of breast cancer patients.

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Bireociclib tablets are applicable to adult patients with advanced or metastatic breast cancer who are positive for hormone receptor (HR) and negative for human epidermal growth factor receptor 2 (HER2). On the one hand, it can be combined with fulvestrant for patients with disease progression after previous endocrine therapy. On the other hand, it is the only CDK4/6 inhibitor approved in China as monotherapy for the patients with disease progression after receiving two or more endocrine therapies and one chemotherapy in the metastatic stage. According to authoritative data, approximately 420,000 new breast cancer cases are diagnosed in China each year, among which nearly 100,000 are advanced breast cancer patients. The launch of Bireociclib tablets undoubtedly provides a new and effective treatment option for this large patient group.

Globally, breast cancer is the most common malignant tumor among women, with approximately 70% being the hormone receptor-positive subtype. Although the advent of CDK4/6 inhibitors has significantly extended patients’ survival, current treatments still face many challenges. The efficacy of existing drugs is limited in premenopausal patients; patients with visceral crisis have a poor prognosis; some patients have difficulty tolerating the adverse reactions of the drugs; and the problem of primary drug resistance is also prominent. Bireociclib tablets, with their innovative multi-target mechanism of action (acting on CDK2, 4, 6, and 9), can precisely block the tumor cell proliferation pathway while significantly reducing the incidence of hematological toxicity. This not only achieves a strong and continuous inhibition of tumors but also greatly improves patients’

On May 15, 2025 Akari Therapeutics, Plc (Nasdaq: AKTX), a biotechnology company developing novel Antibody Drug Conjugates (ADCs) with immuno-oncology payloads for the treatment of cancer, reported its financial results for the first quarter ended March 31, 2025 and provided a corporate update (Press release, Akari Therapeutics, MAY 15, 2025, View Source [SID1234653207]).

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"We remain laser focused on becoming a key player in the ADC space and advancing our novel ADC platform built around immuno-oncology payloads and our lead asset AKTX-101, an ADC targeting Trop2 with our immuno-oncology payload, PH1. We continue to develop and execute a clear path forward for our ADC pipeline and support these efforts with ongoing activities and building the team that we believe positions us for success in the near and long term," commented Abizer Gaslightwala, President and Chief Executive Officer of Akari. "In particular, we were pleased to recently welcome Mark Kubik, a seasoned leader in the Antibody Drug Conjugate space, as Head of Business Development, Oncology and believe his expertise will be invaluable as we continue to advance our novel ADC platform technology."

Leveraging its innovative payload platform, the Company is advancing a pipeline of potentially first-in-class, best-in-class ADC candidates across a wide range of cancer tumor targets. These initial candidates have shown significant tumor-killing activity in preclinical models with the ability to robustly activate the immune system to drive durable, and sustained outcomes.

Upcoming Expected Value-Driving Milestones

Novel ADC’s With Immuno-Oncology Payloads

Anticipate presenting preclinical data showing that a proof-of-concept ADC with PH1 payload exhibits robust immuno-oncology activity, at a scientific conference in second half of 2025.
Complete additional preclinical studies for novel PH1 payload exploring activity in prostate cancer cell lines.
Explore preclinical activity for AKTX-101 in different solid tumor indications including lung, as single agent and in combination with other approved agents.
Continue to focus on operational excellence and efficient capital allocation to advance novel payload ADC platform.
Ongoing efforts to seek strategic partners for research collaborations on PH1 immuno-oncology payload across customized tumor targets. Continued discussions with partners on advancing AKTX-101 ADC (Trop2/PH1 payload) through additional IND-enabling activities.
Non-Core Asset Out Licensing

Continue efforts to out-license non-core assets across inflammation, ophthalmology, and rare diseases as a source of non-dilutive capital to invest into ADC platform.
Summary of Financial Results for First Quarter 2025

The net loss from operations for the three months ended March 31, 2025 was approximately $3.7 million compared to $5.6 million for the same period in 2024.

The Company reported research and development expenses of $0.8 million for the three months ended March 31, 2025 compared to approximately $2.3 million for the same period in 2024. The decrease was primarily due to our decision to suspend our HSCT-TMA clinical stage program with nomacopan in May 2024.

General and administrative expenses were approximately $2.7 million for the three months ended March 31, 2025 compared to approximately $3.7 million for the same period in 2024. The decrease was primarily due to (i) decreases in legal and professional fees (primarily related to the Merger) and (ii) a decrease in directors’ and officers’ insurance.

As of March 31, 2025, the Company had cash of approximately $2.6 million. The net proceeds from the Company’s March 2025 offering, after deducting placement agent fees and other offering expenses, were approximately $6.0 million, of which $4.0 million was received in April 2025.

On May 15, 2025 Immutep Limited (ASX: IMM; NASDAQ: IMMP) ("Immutep" or "the Company"), a late-stage immunotherapy company targeting cancer and autoimmune diseases, reported that a 60.8% response rate and 90.2% disease control rate, according to RECIST1.1, has been achieved in the investigator-initiated INSIGHT-003 trial as of the data-cut off date of 06 May 2025 (Press release, Immutep, MAY 15, 2025, View Source [SID1234653089]). INSIGHT-003 is evaluating eftilagimod alpha (efti) in combination with the anti-PD-1 therapy, KEYTRUDA (pembrolizumab) and doublet chemotherapy as first-line treatment for patients with advanced or metastatic non-squamous non-small cell lung cancer (1L NSCLC).

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Marc Voigt, CEO of Immutep, stated, "Our level of confidence in efti driving a new standard of care for patients with non-small cell lung cancer via our pivotal TACTI-004 trial continues to rise with the strength of the data from INSIGHT-003 and TACTI-002. Across two trials we have now efficacy data from 165 patients with 1L NSCLC who have been treated with efti and KEYTRUDA, either with or without chemotherapy. In multi-national settings, efti has generated consistent and remarkable improvements in response rates. In particular, the interim ORR data in patients with PD-L1 expression below 50% in the ongoing INSIGHT-003 trial, who represent over two-thirds of the 1L NSCLC patient population, is very encouraging."

Majority of Patients have PD-L1 TPS <50%
Notably, ~92% of all evaluable patients (N=51) in the INSIGHT-003 study have PD-L1 TPS <50%. This includes 49% of patients with PD-L1 Tumour Proportion Score (TPS) of 1-49% and 43% of patients with PD-L1 TPS <1%, as shown in the table below.

Strong Response Rates Across All PD-L1 Expression Levels
Data from all evaluable patients demonstrates significant improvement of Overall Response Rate (ORR) according to RECIST 1.1 across all levels of PD-L1 expression compared to historical control from a registrational trial of anti-PD-1 and doublet chemotherapy in non-squamous 1L NSCLC1:

75.0% ORR versus 62.1% ORR in patients with high PD-L1 expression (TPS >50%)
64.0% ORR versus 49.2% ORR in patients with low PD-L1 expression (TPS 1-49%)
54.5% ORR versus 32.3% ORR in patients with negative PD-L1 expression (TPS <1%)

The 60.8% response rate regardless of PD-L1 expression (TPS 0-100%) represents a substantial improvement compared to historical control of 48.0%.1 The relative outperformance is particularly strong given the registrational trial has four times as many patients with high PD-L1 expression (~32% of patients versus ~8% in INSIGHT-003), who have the highest response rates.

Importantly, in patients with TPS <50% (N=47), who have a high unmet need and represent over two-thirds of the 1L NSCLC patient populaton, the triple combination with efti achieved a 59.6% response rate as compared to historical control of 40.8%.1

INSIGHT-003 Overall Response Rate & Disease Control Rate, according to RECIST1.1
PD-L1 Expression Levels TPS 0-100%
(N=51) TPS <1%
(N=22) TPS 1-49%
(N=25) TPS <50%
(N=47) TPS ≥50%
(N=4)
ORR % 60.8 54.5 64.0 59.6 75.0
DCR % 90.2 86.4 92.0 89.4 100

Safety
Safety continues to be favourable for the triple combination in 1L NSCLC with no new safety signals.

Next Steps
Additional data updates from this trial are expected to be presented at a medical conference in 2025 and beyond.

About INSIGHT-003
INSIGHT-003 is an investigator-initiated study conducted by the Frankfurt Institute of Clinical Cancer Research IKF and several other German centres. It is being run as the third arm (Stratum C) of the ongoing Phase I INSIGHT trial with Prof. Dr. Salah-Eddin Al-Batran as lead investigator. The study is evaluating a triple combination therapy in front-line non-small cell lung cancer patients consisting of efti administered subcutaneously in conjunction with an existing approved standard-of-care combination of anti-PD-1 therapy (pembrolizumab) and chemotherapy (carboplatin and pemetrexed) delivered intravenously. The trial will assess the safety, tolerability, and initial efficacy of the combination.

About Eftilagimod Alfa (efti)
Efti is Immutep’s proprietary soluble LAG-3 protein and MHC Class II agonist that stimulates both innate and adaptive immunity for the treatment of cancer. As a first-in-class antigen presenting cell (APC) activator, efti binds to MHC (major histocompatibility complex) Class II molecules on APC leading to activation and proliferation of CD8+ cytotoxic T cells, CD4+ helper T cells, dendritic cells, NK cells, and monocytes. It also upregulates the expression of key biological molecules like IFN-ƴ and CXCL10 that further boost the immune system’s ability to fight cancer.

Efti is under evaluation for a variety of solid tumours including non-small cell lung cancer (NSCLC), head and neck squamous cell carcinoma (HNSCC), and metastatic breast cancer. Its favourable safety profile enables various combinations, including with anti-PD-[L]1 immunotherapy and/or chemotherapy. Efti has received Fast Track designation in first line HNSCC and in first line NSCLC from the United States Food and Drug Administration (FDA).

On May 15, 2025 CytoDyn Inc. (OTCQB: CYDY) ("CytoDyn" or the "Company"), a biotechnology company developing leronlimab, a CCR5 antagonist with the potential for multiple therapeutic indications, reported the Company’s poster presented at the European Society for Medical Oncology’s ("ESMO") Breast Cancer meeting on May 14-17, 2025, in Munich, Germany (Press release, CytoDyn, MAY 15, 2025, View Source [SID1234653150]).

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A copy of the poster is available at the following link: ESMO (Free ESMO Whitepaper) Poster May 2025

More information about the data CytoDyn presented at ESMO (Free ESMO Whitepaper) suggesting the novel mechanism of action of leronlimab for the treatment of solid tumors can be found in the Company’s press release issued on May 13, 2025.