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Kazia Therapeutics Reports Early Efficacy Data from First Triple-Negative Breast Cancer Patient Receiving Paxalisib Combination Regimen achieving >50% Reduction in Circulating Tumor Cells in Phase 1b Trial

On July 9, 2025 Kazia Therapeutics (NASDAQ: KZIA) reported preliminary results from the first patient in its Phase 1b trial evaluating a combination regimen of Paxalisib, pembrolizumab (Keytruda), and standard chemotherapy after completing Cycle 1 (21 days) of dosing (Press release, Kazia Therapeutics, JUL 9, 2025, View Source [SID1234654307]). The patient, a 61-year-old woman with metastatic triple-negative breast cancer localized to the left upper lobe of the lung, has shown highly encouraging preliminary results at 21 days, with a >50% reduction in circulating tumor cells (CTCs) and a notable decrease in CTC clusters.

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The early data in this first patient closely mirror the mechanistic preclinical findings published in Molecular Cancer Therapeutics (View Source), which highlight that Paxalisib, when combined with immunotherapy, significantly disrupted both single CTCs and multicellular clusters in preclinical models.

Key Highlights

– Patient Profile: 61-year-old female, metastatic triple-negative breast cancer (lung metastasis).

– Investigational Regimen: Paxalisib, pembrolizumab, and chemotherapy.

– Results at Day 21 (End-of-Cycle 1):

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>50% reduction in total CTC count.

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Comparable reduction in CTC clusters—these aggregates are associated with heightened metastatic potential.

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Reduction in the mesenchymal phenotype of the remaining CTCs; this phenotype is one of the hallmarks of aggressive metastatic seeding cancer cells.

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First-in-human data support potential for potent CTC mobilization suppression by this combination.

Clinical Significance of Patient Data

CTC clusters have long been recognized as critical mediators of metastasis and markers of poor prognosis. They are known to resist apoptosis, evade immune detection, and seed new tumor sites with exceptional efficiency. Notably, standard chemotherapy has been shown in some studies to transiently increase CTC and cluster counts within the first cycle, with levels sometimes doubling before normalizing after cycle two. In contrast, immunotherapy alone has demonstrated variable impact, often showing delayed or modest effects on CTCs, likely due to immune-mediated mechanisms over weeks to months.

In this case, the combination regimen of Paxalisib and immunotherapy achieved a rapid reduction in both CTC numbers and clusters as well as a reduction in the mesenchymal phenotype—an outcome not typically seen with chemotherapy or immunotherapy alone after only 21 days of treatment. This early clinical data reflects mechanistic synergy consistent with the preclinical data described in the MCT manuscript.

Dr. John Friend, MD, Chief Executive Officer of Kazia Therapeutics, said "It is very exciting to see our extensive preclinical research translate into such positive early data in this first patient receiving a combination of Paxalisib and immunotherapy. The degree of reduction in tumor cell dissemination markers in just 21 days gives us strong reason for optimism as we continue this clinical trial."

Dr. Friend continued "CTC clusters are emerging as key drivers of metastatic spread—they’re 20–100X more efficient at seeding than single CTCs—and the sharp decline we’re seeing is truly encouraging. We believe this combination may offer a meaningful early intervention against systemic disease progression."

Next Steps

– Explore potential relationship between CTC kinetics and radiographic responses

– Enrollment continues in the Phase Ib study, expanding cohort size to assess safety, tolerability, and pharmacodynamics

– Planned comprehensive analysis of immune microenvironment and CTC kinetics across all patients through serial monitoring

– Longer-term follow-up will include imaging, progression-free survival, and assessment of correlation with molecular biomarkers

For investor and media, please contact Alex Star, Managing Director LifeSci Advisors LLC, [email protected], +1-201-786-8795.

Immuneering Granted U.S. Composition of Matter Patent for Highly Differentiated Cancer Drug Candidate Atebimetinib

On July 9, 2025 Immuneering (Nasdaq: IMRX), a clinical-stage oncology company outpacing cancer to help patients outlive their disease, reported that the United States Patent and Trademark Office (USPTO) granted the company a composition of matter patent for atebimetinib (IMM-1-104), an oral once-daily deep cyclic inhibitor of MEK (Press release, Immuneering, JUL 9, 2025, View Source [SID1234654306]). MEK is a key component of the signaling pathway that drives the majority of cancers, including pancreatic cancer.

First-line pancreatic cancer patients treated with atebimetinib plus chemotherapy had a remarkable 94% probability of surviving 6 months, in data from Immuneering’s ongoing Phase 2a study announced in June, with few serious side effects observed. In prior studies of the most common global standard of care chemotherapy in first-line pancreatic cancer patients, the probability of surviving 6 months was only 67%.

U.S. Patent No. 12,351,566, titled: "MEK Inhibitors and Therapeutic Uses Thereof", includes claims to atebimetinib’s composition of matter. The patent’s term, which includes a patent term adjustment, is currently expected to expire in August 2042. The patent may also be eligible for patent term extension to recover a portion of the time required to fulfill regulatory approval requirements.

"Our priorities are to make medicines that keep working, so cancer patients keep living, and to make medicines that have fewer side effects, so cancer patients can feel like themselves and live normal lives. We have already observed exceptional durability and a markedly favorable tolerability profile in first-line pancreatic cancer patients treated with atebimetinib+mGnP, and this is just the beginning of the important impact that we believe atebimetinib and our entire pipeline of deep cyclic inhibitors will have on the treatment of cancer," said Ben Zeskind, Ph.D., Co-founder and Chief Executive Officer of Immuneering.

"We believe the granting of our composition of matter patent validates the novelty of our approach and secures key intellectual property around our lead product candidate, as part of a broader intellectual property strategy," Zeskind continued. "We expect that the long patent runway we are forging for atebimetinib will support our efforts to maximize its full therapeutic potential, starting with first-line pancreatic cancer and extending to many different cancer types and combinations."

Atebimetinib previously received FDA Fast Track designations for the treatment of first- and second-line pancreatic ductal adenocarcinoma (PDAC), as well as for patients with unresectable or metastatic NRAS-mutant melanoma who have progressed on or are intolerant to PD-1/PD-L1 based immune checkpoint inhibitors. The FDA also previously granted atebimetinib orphan drug designation for the treatment of pancreatic cancer. Immuneering has also announced plans to study atebimetinib in combination with other therapeutics – in a variety of additional cancers.

Anixa Biosciences Announces Issuance of Additional U.S. Patent for Ovarian Cancer Vaccine Technology

On July 9, 2025 Anixa Biosciences, Inc. ("Anixa" or the "Company") (NASDAQ: ANIX), a biotechnology company focused on the treatment and prevention of cancer, reported that the United States Patent and Trademark Office (USPTO) will issue U.S. Patent Number 12,357,593 on July 15, 2025 covering key aspects of its ovarian cancer vaccine technology (Press release, Anixa Biosciences, JUL 9, 2025, View Source [SID1234654304]). The patent includes broad claims related to methods of eliciting an immune response targeting anti-Müllerian hormone receptor, type II (AMHR2), a promising target for ovarian cancer prevention and treatment.

Anixa’s ovarian cancer vaccine, being developed in a collaboration between Cleveland Clinic and the National Cancer Institute, represents a novel approach to preventing and treating ovarian cancer, particularly among high-risk populations such as those carrying BRCA mutations or with a family history of the disease.

The patent includes methods of administering an immunogenic composition comprising a nucleic acid encoding the AMHR2 polypeptide, specifically the extracellular domain of human AMHR2, to elicit an AMHR2-specific immune response. This patent was issued to Cleveland Clinic and Anixa exclusively holds the world-wide rights to the patent.

Dr. Amit Kumar, Chairman and CEO of Anixa Biosciences, commented, "The issuance of this patent by the USPTO continues to strengthen broad protection for the various components and delivery mechanisms of our vaccine technology. This patent supports the continued advancement of our program."

Actithera Raises $75.5M in Oversubscribed Series A Financing to Redefine Precision Radioligand Therapy

On July 9, 2025 Actithera, a radiopharmaceutical biotech company translating medicinal chemistry insights into next-generation radioligand therapies (RLTs), reported the close of an oversubscribed $75.5 million Series A financing round (Press release, Actithera, JUL 9, 2025, View Source [SID1234654293]). The financing will support the advancement of Actithera’s lead FAP asset into clinical development in multiple indications, while also enabling the continued development of its proprietary RLT discovery platform and preclinical pipeline.

The round was co-led by founding investor M Ventures and new lead investors Hadean Ventures, Sofinnova Partners, and 4BIO Capital, with additional participation from Bioqube Ventures, Innovestor’s Life Science Fund, Investinor, Surveyor Capital (a Citadel company), and second founding investor, Arkin Bio Ventures II.

The Company’s discovery platform combines rational drug design with radiochemistry to create novel small molecule radioligands that overcome current limitations in radiopharmaceutical development. Its three-pillar platform includes first-in-class covalent targeting strategies, designed to optimize tumor residence time, while ensuring rapid systemic clearance – improving precision, safety, and efficacy. Two additional proprietary approaches further support compound differentiation and improve tumor residence time and selectivity. This platform was validated through Actithera’s work on FAP, a high-value theranostic target known for being difficult to drug with molecules that maintain prolonged tumor residency. These efforts have resulted in a FAP-directed RLT development candidate with best-in-class potential due to its optimal pharmacokinetic profile and tumor specificity.

Dr. Andreas Goutopoulos, founder and CEO, brings over 25 years of pharmaceutical and biotech industry experience, including a track record of more than a dozen development candidates. His background includes over a decade of discovery leadership at EMD Serono, where he led medicinal chemistry. In his role as Entrepreneur-in-Residence (EIR) at M Ventures, he led the scientific efforts of and supported a number of oncology small molecule biotechs. At Actithera, he is pioneering a chemistry-driven, precision approach to RLTs by integrating novel covalent-targeting chemistries, rational drug design principles and an isotope-agnostic philosophy.

"We set out to bring structure-based and kinetics-driven thinking from small molecule drug design into the world of radiopharmaceuticals," said Dr. Andreas Goutopoulos. "This oversubscribed Series A, backed by a truly global and experienced investor syndicate, is strong validation of our approach. We engineer our radioconjugates for extended retention within tumors, making them ideally suited for longer-lived radionuclides and ultimately delivering more convenient dosing schedules and enhanced efficacy and safety for patients."

Karl Naegler, incoming Board member and Partner at Sofinnova Partners, noted: "Actithera is applying Big Pharma discipline to an emerging field with enormous potential. Its radioligand therapies represent a meaningful shift in oncology, with the opportunity to redefine the therapeutic index. We’re excited to support that vision."

Roger Franklin, incoming Board member and Partner at Hadean Ventures, added: "Actithera stands out as one of the most thoughtfully constructed radiopharma platforms we’ve seen, combining smart molecular design with a deep understanding of tumor biology and clinical need. The team’s work to align pharmacokinetics with therapeutic effect could transform how patients experience and benefit from radioligand therapies."

Therese Liechtenstein, incoming Board member and Investment Director at 4BIO Capital, added: "We are honored to support Actithera, whose molecules address key challenges in the nascent radioligand therapies space; a large therapeutic window through high tumor retention and low systemic exposure, applied to a lead program that has significant pan-tumor therapeutic potential."

Hakan Goker, current Chairman of Actithera, and Managing Director at M Ventures, said: "We are excited to see Actithera evolve from the one-person ideation we seeded with Andreas and Arkin to the transatlantic company it has become today. The innovative chemistry platform built and the first-in-class approach on FAP have the potential for a large impact in the RLT field and a significant benefit for patients. We welcome the new investor group and Board members to the company aligned with this bold vision of building the next generation of RLTs."

As part of the Series A financing, Roger Franklin, Partner at Hadean Ventures, Karl Naegler, Partner at Sofinnova Partners, Therese Liechtenstein, Investment Director at 4BIO Capital, and Debbie Dumont, Managing Partner at Bioqube Ventures will join the Actithera Board of Directors, including Noga Yerushalmi, Investment Director at M Ventures, who is currently on the Board.

BriaCell Phase 2 Survival Achievement: 52% of Patients Surpass One-Year Milestone in Metastatic Breast Cancer

On July 8, 2025 BriaCell Therapeutics Corp. (Nasdaq: BCTX, BCTXW, BCTXZ) (TSX: BCT) ("BriaCell" or the "Company"), a clinical-stage biotechnology company developing novel immunotherapies to transform cancer care, reported updated survival data from its ongoing Phase 2 clinical study of Bria-IMT in patients with metastatic breast cancer (MBC) (Press release, BriaCell Therapeutics, JUL 8, 2025, View Source [SID1234654345]).

BriaCell’s most recent Phase 2 study cohort of 25 patients* achieved a 52% one-year survival rate (i.e. 52% of patients remained alive at least one year after starting on the study).
11 of these patients remain alive as of most recent contact, including one patient at 38.3 months and another at 30.3 months (see Table 1).
Survival rate exceeds the survival expectations with the current standard of care therapies in similar patient populations (see Table 2).
Notably, many patients had very advanced metastatic breast cancer, having already failed multiple prior lines of therapy including check point inhibitors (CPIs) and antibody-drug conjugates (ADCs) such as TRODELVY – (sacituzumab govitecan-hziy) and ENHERTU (fam-trastuzumab – deruxtecan-nxki).
No treatment discontinuations attributed to Bria-IMT have been reported.
Table 1: Select Long-Term Responders
Patient Months Survival Age Prior Regimens Cycles of Bria-IMT
01-009 38.3 74 5 13
07-001 30.3 55 7; including ENHERTU 8
11-018 21.6 66 8 28
11-019 20.0 63 9; including TRODELVY 6
16-003 19.4 80 5; including ENHERTU 8
"BriaCell’s Phase 2 data indicate a robust survival signal and a well-tolerated profile," stated Adam M. Brufsky, MD, PhD, FACP, Professor of Medicine at the University of Pittsburgh School of Medicine and Medical Director of the Magee-Women’s Cancer Program. "These results reinforce BriaCell’s potential to improve survival and tolerability for late-stage patients."

"Many patients with metastatic breast cancer unfortunately have disease progression despite treatment with CPIs and ADCs," added Aditya Bardia, MD, MPH, FASCO, a leading breast cancer expert. "BriaCell’s survival data in single arm Phase 2 trial highlights the potential activity of Bria-IMT in combination with CPIs and is subject to ongoing investigation in a Phase 3 randomized clinical trial in MBC."

Table 2: Comparable Analysis of One-Year Survival
Reference Breast Cancer Type Median prior lines of therapy Percent Survival at 1 year
Bria-IMT plus CPI All types:
61% HR+
33% TNBC
6% HER2+ 6 52%*
Cortes et al. 1 All types:
57% HR+
18-19% TNBC
18-20% HER2+ 4 ~38-40%
Kazmi et al. 2 All types:
51-52% HR+
25-29% TNBC
9-24% HER2+ 2 30-38%
Bardia et al.
(TPC arm) 3 TNBC 2-3 ~23%
Rugo et al
(TPC arm) 4 HR+ HER2- 2 47%
* 25 patients treated with the Phase 3 formulation since 2022
Cortes J, et al. Annals of Oncology 2018
Kazmi S, et al. Breast Cancer Res Treat. 2020
Bardia A, et al. J Clin Oncol. 2024
Rugo HS, et al. The Lancet. 2023
Abbreviations:

HR+: hormone receptor-positive
TNBC: Triple-negative breast cancer (lacks or has low levels of the estrogen receptor, progesterone receptor, and human epidermal growth factor 2 (HER2))
HER2+: Human epidermal growth factor receptor 2 positive
HR+ HER2-: hormone receptor-positive and human epidermal growth factor receptor 2 negative
TPC: Treatment of Physicians Choice

BriaCell’s Phase 2 study enrolled 54 heavily pre-treated metastatic breast cancer patients (median number of prior treatments = 6) who received the Bria-IMT regimen plus checkpoint inhibitor. Of these 54 patients, 37 were treated with the formulation currently being used in BriaCell’s ongoing pivotal Phase 3 study in metastatic breast cancer (listed on ClinicalTrials.gov as NCT06072612 ). Final median overall survival calculation for the Phase 2 study is pending for some sub populations as many patients remain alive. No Bria-IMT-related discontinuations have been reported to date.