On August 18, 2025 Genprex, Inc. ("Genprex" or the "Company") (NASDAQ: GNPX), a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes, reported that the U.S. Patent and Trademark Office has issued a Notice of Allowance for a patent to Genprex that covers the use of the Company’s lead drug candidate, Reqorsa Gene Therapy, in combination with PD-L1 antibodies, such as Tecentriq (Press release, Genprex, AUG 18, 2025, View Source [SID1234655351]). Similarly, the European Patent Office has issued a Notice of Allowance to Genprex for a patent covering the use of REQORSA in combination with PD-1 antibodies. Both patents will expire in 2037 at the earliest.

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"We are very proud to continue building a fortress of intellectual property protection around our oncology program for REQORSA, and these newly granted patents strengthen and expand our intellectual property estate for REQORSA in combination with immune checkpoint inhibitors applicable to our Acclaim-3 clinical trial," said Thomas Gallagher, Esq., Senior Vice President of Intellectual Property and Licensing at Genprex.

Genprex has been granted patents for the use of REQORSA in combination with PD-L1 antibodies in Korea. Genprex is pursuing additional patent applications in Europe, Canada, Brazil, China and Israel. Should these applications grant, they would be applicable to Genprex’s Acclaim-3 clinical trial.

Genprex has been granted patents for the use of REQORSA in combination with PD-1 antibodies in the U.S., Japan, Mexico, Russia, Australia, Chile, China, Korea and Singapore.

PD-L1 and PD-1 antibodies are a type of targeted immunotherapy and a part of a group of checkpoint inhibitor anti-cancer drugs that block the activity of PD-L1 and PD-1 immune checkpoint proteins present on the surface of cells.The Acclaim-3 study is a Phase 1/2 clinical trial that uses a combination of REQORSA and Genentech’s Tecentriq as maintenance therapy for patients with extensive stage small cell lung cancer (ES-SCLC) who are candidates for maintenance therapy after receiving Tecentriq and chemotherapy as initial standard treatment. The Acclaim-3 clinical trial has received U.S. Food and Drug Administration (FDA) Fast Track Designation for this patient population, and Acclaim-3 has received FDA Orphan Drug Designation.

On August `8, 2025 FibroGen, Inc. (NASDAQ: FGEN) reported that the China State Administration for Market Regulation approved the sale of FibroGen International (Hong Kong) Ltd. to AstraZeneca Treasury Limited, pursuant to the Share Purchase Agreement, dated February 20, 2025 (Press release, FibroGen, AUG 18, 2025, View Source [SID1234655344]).

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The closing of the transaction remains subject to other contractual closing conditions and deliverables and remains on track to close in the third quarter of 2025.

On August 18, 2025 SystImmune Inc. (SystImmune), a clinical-stage biotechnology company, and Bristol Myers Squibb reported that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) to izalontamab brengitecan (iza-bren) for the treatment of locally advanced or metastatic non-small cell lung cancer (NSCLC) with epidermal growth factor (EGFR) exon 19 deletions or exon 21 L858R substitution mutations whose disease has progressed on or after treatment with an EGFR tyrosine kinase inhibitor (TKI) and platinum-based chemotherapy (Press release, Bristol-Myers Squibb, AUG 18, 2025, View Source;FDA-for-Patients-with-Previously-Treated-Advanced-EGFR-Mutated-Non-Small-Cell-Lung-Cancer/default.aspx [SID1234655343]).

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Iza-bren is a potential first-in-class bispecific antibody-drug conjugate (ADC) which targets both epidermal growth factor receptor and human epidermal growth factor receptor 3 (EGFRxHER3) with a topoisomerase 1 inhibitor payload. Iza-bren is being developed by Biokin in China and jointly developed by SystImmune and Bristol Myers Squibb under a collaboration and exclusive license agreement in territories outside of China.

The granting of BTD underscores the strength of these data and highlights the potential of iza-bren to address the significant clinical unmet need patients face after EGFR TKI and platinum-based chemotherapy treatment. While EGFR TKIs have shown clinical efficacy in the frontline setting, most patients eventually see their cancer progress after about 18 months. Subsequent treatment options often contain platinum-based chemotherapy, which are of limited efficacy and come with significant toxicities. Breakthrough Therapy Designation from the US FDA is intended to expedite the development and review of drugs that may demonstrate significant benefit over current standards of care.

The FDA’s decision was based on efficacy and safety data from three ongoing clinical trials: BL-B01D1-101 and BL-B01D1-203, conducted in China by Sichuan Biokin Pharmaceutical Co., Ltd., and the global BL-B01D1-LUNG-101 study conducted by SystImmune across the United States, Europe and Japan. Across these trials, iza-bren demonstrated evidence to suggest improved efficacy with a manageable safety profile in patients with EGFR-mutant NSCLC who had progressed after third-generation EGFR TKIs and platinum-based chemotherapy.

"The FDA’s granting of Breakthrough Therapy Designation underscores the potential of iza-bren to meaningfully improve clinical outcomes for patients with previously treated epidermal growth factor receptor mutation NSCLC," said Dr. Jonathan Cheng, Chief Medical Officer of SystImmune. "The data we have generated to date suggest that iza-bren could address a critical unmet need in patient care, and we look forward to working closely with the FDA to conduct the relevant clinical studies and seek regulatory approval."

About EGFRmt NSCLC

Non-small cell lung cancer (NSCLC) accounts for approximately 80% of all lung cancer cases, which remains the leading cause of cancer-related death worldwide. Among patients with NSCLC, 10% to 15% in Western populations and up to 50% in Asian populations harbor activating EGFR mutations. These tumors, most commonly of non-squamous histology, initially respond to EGFR TKIs such as osimertinib. However, resistance is nearly universal, often occurring after about 18 months, and treatment options beyond TKIs and platinum-based chemotherapy provide limited clinical benefit with significant toxicities, highlighting the critical need for new, effective therapies.

About iza-bren

SystImmune, in collaboration with BMS outside of China, is developing iza-bren (BL-B01D1), a bispecific antibody-drug conjugate (ADC) that targets both EGFR and HER3 targets that are highly expressed in various epithelial cancers and are known to be associated with cancer cell proliferation and survival. Iza-bren’s dual mechanism of action blocks EGFR and HER3 signals to cancer cells, reducing proliferation and survival signals. In addition, upon antibody mediated internalization, iza-bren’s therapeutic payload is released causing genotoxic stress that leads to cancer cell death.

On August 18, 2025 Aptose Biosciences Inc. ("Aptose" or the "Company") (OTC: APTOF, TSX: APS), a clinical-stage precision oncology company developing the tuspetinib (TUS)-based triple drug frontline therapy to treat patients with newly diagnosed AML, reported a data update from the Phase 1/2 TUSCANY trial in newly diagnosed AML (Press release, Aptose Biosciences, AUG 18, 2025, View Source [SID1234655342]). The trial was initiated in December 2024, and the growing body of positive data includes the recently completed third cohort of 120 mg TUS in the TUS+VEN+AZA triplet therapy. Data to date from ten (10) patients across all three cohorts, 40 mg, 80 mg or 120 mg TUS dose in TUS+VEN+AZA, support the use of TUS with standard of care treatment across all AML populations, including those carrying mutations that are the most difficult to treat and those with mutated and unmutated (wildtype) FLT3 genes.

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The TUS+VEN+AZA triplet is being developed as a safe and well-tolerated mutation agnostic frontline therapy to treat large, mutationally diverse populations of newly diagnosed AML patients who are ineligible to receive induction chemotherapy. At the 120 mg TUS dose level in combination with VEN+AZA, as with the prior reported 40 mg and 80 mg TUS dose cohorts, no significant safety concerns or dose limiting toxicities (DLTs) have been observed in the TUSCANY trial, including no prolonged myelosuppression in Cycle 1 of subjects in remission, no reports of drug-related QTc prolongation or differentiation syndrome (DS), and no treatment-related deaths. Nine out of ten dosed patients remain on study across all dose cohorts and enrollment is being advanced to the 160 mg TUS dose level following the Cohort Safety Review Committee (CSRC) meeting.

"We already have data from three different TUS dose levels in the TUSCANY trial, and the data continue to strengthen at higher doses of TUS and over time. We are building a strong case for TUS+VEN+AZA as a triplet frontline therapy of choice to address a broad AML population, including subgroups with the most adverse of mutations," said William G. Rice, Ph.D., Chairman, President and Chief Executive Officer of Aptose.

Data highlights:

Comparison of CR/CRh1 Response rates2-5:

VEN+AZA2 TUS+VEN+AZA
All subjects 65% 90% (9/10)
NPM1-mutant 67% 100% (2/2)
FLT3-ITD 61% 100% (2/2)
TP53-mutant 52% 100% (2/2)

Comparison of MRD-negativity6 rates among All Subjects and among CR/CRh Responders3:

VEN+AZA2,3 TUS+VEN+AZA
Among All Subjects 23.4% 70% (7/10)
Among CR/CRh Responders 40.9% 78% (7/9)

Comparison of MRD-negativity rates among more difficult-to-treat Patient Subpopulations defined as Lower Benefit (TP53-mutated) and Intermediate Benefit (FLT3-ITD or RAS-mutated) relative to VEN+AZA5:

VEN+AZA3,5 TUS+VEN+AZA
Intermediate Benefit 27.9% 100% (3/3)
Lower Benefit 14.5% 100% (2/2)

TUS+VEN+AZA – CR/CRh and MRD-negativity rates among Subjects with Adverse Mutations:

TP53, FLT3-ITD, RAS mutations: Achieved CR/CRh and MRD-negativity
100% (5/5)

"As illustrated in the data highlights, the addition of TUS to VEN+AZA appears to boost response rates and MRD-negativity while maintaining favorable safety and tolerability," said Rafael Bejar, M.D., Ph.D., Chief Medical Officer of Aptose, "and the 100% CR/CRh and 100% MRD-negativity rates among the five biallelic TP53-mutant, FLT3-ITD, and RAS-mutant AML cases are exciting to see, as this can correlate with longer overall survival. We have observed a trend towards achieving CRs more quickly at the higher dose levels, so we are keen to see the activity as we advance into the 160 mg TUS dose cohort."

Key messages:

Addition of TUS to VEN+AZA demonstrates excellent CR/CRh rates
100% CR/CRh among all subjects treated at 80 mg and 120 mg TUS dose levels
Appear to be achieving CR earlier with 120 mg TUS than with 40 mg or 80 mg
Addition of TUS to VEN+AZA demonstrates excellent MRD-negativity rates
MRD-negativity in 7 of 9 (78%) already achieved in patients who responded to therapy
Expect patient survival to be extended with continued long-term treatment
Excellent safety and well tolerated with no dose-limiting toxicities (No DLT) at completed dose levels
Broad-spectrum activity including patients with adverse TP53, RAS and FLT3-ITD mutations
No loss of MRD-negativity observed to date, including in one patient with over 7 months of follow up
MRD-negativity and remissions continue to mature over time on therapy
No relapses reported to date and no treatment related deaths
The only non-responder was a patient at the initial TUS dose level (40 mg) that did not achieve TUS exposures previously associated with response
Additional data are included in the new Aptose corporate presentation here.

TUSCANY: TUS+VEN+AZA Triplet Phase 1/2 Study

The tuspetinib-based TUS+VEN+AZA triplet therapy is being advanced in the TUSCANY Phase 1/2 trial with the goal of creating an improved frontline therapy for newly diagnosed AML patients that is active across diverse AML populations, durable, and well tolerated.

The TUSCANY triplet Phase 1/2 study, being conducted at 10 leading U.S. clinical sites by elite clinical investigators, is designed to test various doses and schedules of TUS in combination with standard dosing of AZA and VEN for patients with AML who are ineligible to receive induction chemotherapy. A convenient, once daily oral agent, TUS is being administered in 28-day cycles. Multiple U.S. sites are enrolling in the TUSCANY trial with anticipated enrollment of 18-24 patients by late 2025. Data will be released as it becomes available.

More information on the TUSCANY Phase 1/2 study can be found on www.clinicaltrials.gov (here: View Source).

On August 18, 2025 Anixa Biosciences, Inc. ("Anixa" or the "Company") (NASDAQ: ANIX), a biotechnology company focused on the treatment and prevention of cancer, reported that the second patient in the fourth dose cohort has been treated in the ongoing Phase 1 clinical trial evaluating the Company’s novel chimeric antigen receptor-T cell (CAR-T) therapy for recurrent ovarian cancer (Press release, Anixa Biosciences, AUG 18, 2025, View Source [SID1234655341]). The study is being conducted through a research partnership with Moffitt Cancer Center ("Moffitt").

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Patients in the fourth cohort are receiving a dose of three million CAR-positive cells per kilogram of body weight—representing a 30-fold increase over the initial dose level. To date, no dose-limiting toxicities (DLTs) have been observed in the fourth cohort.

Anixa’s proprietary CAR-T program targets the follicle-stimulating hormone receptor (FSHR), which preclinical research indicates is selectively expressed on ovarian cells, tumor vasculature, and certain cancer cells—but not in healthy tissue. The ongoing first-in-human clinical trial (NCT05316129) is enrolling adult women with recurrent ovarian cancer who have progressed after at least two prior lines of therapy. The Phase 1 study is designed to evaluate safety, determine the maximum tolerated dose, and monitor initial signals of clinical activity.

"Our therapy continues to demonstrate a favorable safety profile, even at significantly higher cell doses," stated Dr. Amit Kumar, Chairman and CEO of Anixa Biosciences. "While this study is primarily designed to assess safety, we remain encouraged by early indications of potential efficacy as the trial progresses."

Anixa’s CAR-T technology was invented by Jose R. Conejo-Garcia, M.D., Ph.D., Professor of Immunology in the Department of Integrative Immunobiology at the Duke University School of Medicine. The ongoing clinical trial is being conducted at Moffitt under the direction of Dr. Robert Wenham, Chair of the Gynecologic Oncology Program. Anixa holds an exclusive worldwide license to the FSHR-targeting CAR-T technology from The Wistar Institute.