On September 8, 2025 PhotonPharma, a biotechnology company developing next-generation cancer immunotherapies, reported the issuance of U.S. Patent No. 12,280,039 B2 titled "Cancer Vaccine Compositions and Methods of Use Thereof (Press release, PhotonPharma, SEP 8, 2025, View Source [SID1234655845])." The patent, issued by the United States Patent and Trademark Office on April 22, 2025, covers the Company’s proprietary method for producing cancer immunotherapy products using photochemically-inactivated cancer cells that maintain immunogenic properties.

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Novel Approach to Cancer Immunotherapies

The patented technology, Innocell, represents a significant potential advancement in cancer therapies. Riboflavin (vitamin B2) acts as a photosensitizer that when combined with UV light, inactivates cancer cells while preserving critical tumor antigens. This innovative approach addresses a fundamental challenge in cancer immunotherapy: how to effectively present tumor antigens to the immune system to stimulate immune response specific to the patient’s own tumor.

Key advantages of technology include:

Product Profile: Cancer cells are rendered completely incapable of replication while maintaining their antigenic properties. Cancer cells with enhanced antigen presentation are produced and can be used to stimulate a broad immune response to the patient’s tumor.

Preserved Immunogenicity: Critical surface markers and tumor-associated antigens remain intact for immune recognition. Cell metabolism is preserved. These altered cells can stimulate an immune response that has been demonstrated to target tumor cell destruction in pre-clinical studies.

Scalable Manufacturing: The photochemical inactivation process is rapid, reproducible, and suitable for clinical-scale production.

Broad Applicability: The technology can be applied across multiple cancer types and stages.
Compelling Preclinical Data

The patent includes extensive preclinical validation demonstrating the technology’s potential:

Tumor Growth Inhibition: Significant reduction in tumor growth rates in multiple animal models

Enhanced Survival: Extended survival times compared to control groups in efficacy studies

Immune System Activation: Robust T-cell responses and immunological memory formation
"This patent issuance validates our innovative approach to cancer immunotherapy and strengthens our intellectual property portfolio as we advance toward clinical trials," said Alan Rudolph, Ph.D., PhotonPharma Inc. board member. "Our technology has the potential to transform recurrent ovarian cancer treatments, offering patients a safer and more effective option."

According to the National Cancer Institute (View Source), ovarian cancer will impact 20,890 women in the US with 12,730 deaths in 2025 alone. The average five-year relative survival rate is 51.6% (2015-2021).

Strategic Value and Market Opportunity

The global Cancer Immunotherapy Market Size is projected to reach between $231-277 billion (CAGR 13.6% – 11.9%) by 2031 driven by increasing cancer incidence and growing adoption of immunotherapy approaches (Statista 2025, BioSpace 2022). This patent positions PhotonPharma at the forefront of next-generation cancer immunotherapy development.

Development Timeline

PhotonPharma is initiating Phase I trials, targeting recurrent epithelial ovarian cancer patients.

On September 8, 2025 OncoC4, Inc., a late-stage biopharmaceutical company developing novel medicines for cancer reported that it will have a poster presentation at the upcoming World Conference on Lung Cancer (WCLC) 2025 hosted by the International Association for the Study of Lung Cancer (IASLC), taking place September 6-9, 2025 in Barcelona, Spain (Press release, OncoC4, SEP 8, 2025, View Source [SID1234655992]). The presentation will feature the pivotal trial Stage II design for PRESERVE-003 (NCT05671510), a two-stage, randomized, open-label, active-controlled Phase 3 Study evaluating gotistobart monotherapy compared to docetaxel in squamous non-small cell lung cancer (sqNSCLC) after progression on a PD-(L)1 inhibitor.

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Squamous NSCLC is among the deadliest cancers with very limited treatment options following traditional chemotherapy and immunotherapy (IO) with immune checkpoint inhibitors1. OncoC4 and its strategic partner BioNTech have embarked on this indication based on encouraging Phase 1/2 results and the match between the vulnerability of the sqNSCLC and the mechanism of action of gotistobart (ONC-392/BNT316). The Stage I of the 2-stage PRESERVE-003 study was designed for dose confirmation based on safety, efficacy and exposure-response analyses. The data supported selecting the high dose for Stage II, which consists of 1:1 randomization between gotistobart and docetaxel in patients with squamous NSCLC. Enrollment of Stage II (potentially registrational) continues to progress well with nearly 160 active sites worldwide.

Gotistobart is a next generation, acid pH-sensitive anti-CTLA-4 monoclonal antibody candidate that avoids antibody-induced lysosomal target degradation for better therapeutic index. Together with modifications in Fc, gotistobart induces more potent and selective depletion of regulatory T cells in the tumor microenvironment.

"The mechanism of action of gotistobart has the potential for clinical development of the drug beyond lung cancers for other indications with unmet medical needs, either as monotherapy or in combination with other therapeutic modalities," said Yang Liu, PhD, Co-Founder, Chief Executive Officer (CEO), and Chief Scientific Officer (CSO) of OncoC4.

WCLC Poster Presentation Details

Title: PRESERVE-003: A Phase 3 Study of Gotistobart Versus Docetaxel in Metastatic NSCLC
After Progression on PD-(L)1 Inhibitors (NCT05671510)

Abstract Number: 1363

Session: P3.18 – Ongoing Clinical Trials

Date: Tuesday, September 9, 2025

Time: 10:00 AM CEST

Presenter: Dr. Tianhong Li, Professor, Department of Internal Medicine, Division of Hematology and Oncology at UC Davis Comprehensive Cancer Center, CA

About PRESERVE-003

PRESERVE-003 is a randomized, open label, active controlled, multi-center Phase 3 trial (ClinicalTrials.gov NCT 05671510) sponsored by OncoC4 and consists of two stages. Stage 1, the dose-confirmation stage, assessed the efficacy and safety of two gotistobart dosing regimens (3 mg/kg Q3W and 6 mg/kg Q3W with 2 loading doses of 10 mg/kg Q3W) in comparison to docetaxel 75 mg/m2 Q3W in patients with squamous and non-squamous non-small cell lung cancer (NSCLC). Stage 2 assesses the safety and efficacy of gotistobart at 6 mg/kg Q3W with 2 loading doses of 10 mg/kg versus docetaxel (75 mg/m2 Q3W) on squamous cell NSCLC. The primary endpoint is Overall Survival.

About gotistobart (BNT316/ONC392)

Gotistobart is licensed to BioNTech for commercialization and jointly developed clinically by OncoC4 and BioNTech for oncology indications. There are several ongoing clinical trials in different tumor types, investigating gotistobart either as monotherapy or in combination with other therapeutic agents.

About squamous non-small cell lung cancer (NSCLC)

Squamous cell non-small cell lung cancer makes up 25 – 30% of all lung cancers, and is the most common lung cancer found in smokers2. In the US, first-line treatment for metastatic squamous NSCLC commonly involves a combination of chemotherapy and immunotherapy. However, treatment choices diminish when patients progress on prior IO options.

On September 8, 2025 Moleculin Biotech, Inc., (Nasdaq: MBRX) ("Moleculin" or the "Company"), a late-stage pharmaceutical company with a broad portfolio of drug candidates targeting hard-to-treat cancers and viral infections, reported that it has enrolled the first two subjects, and treated one, in the European Union (EU) in its pivotal Phase 2B/3, multi-center, randomized, double-blind, placebo-controlled, adaptive design study of Annamycin in combination with cytarabine (also known as "Ara-C" and for which the combination of Annamycin and Ara-C is referred to as "AnnAraC") for the treatment of adult patients with acute myeloid leukemia (AML) who are refractory to or relapsed (R/R) after induction therapy (R/R AML) (Press release, Moleculin, SEP 8, 2025, View Source [SID1234655831]). This Phase 3 "MIRACLE" trial (derived from Moleculin R/R AML AnnAraC Clinical Evaluation) is a global approval trial, including sites in the US, the EU, and other parts of Europe.

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"We now have active sites recruiting in the US, Spain, Ukraine, Georgia, and Romania with more sites expected to come online by the end of September. For our first site in Spain to have opened up with two subjects enrolled, we believe, indicates the unmet need in treating second line R/R AML," said Walter Klemp, Chairman and CEO of Moleculin. "All of this, importantly, supports our goal to recruit the first 45 subjects for Part A before the end of 2025 for which efficacy and safety will be unblinded."

The MIRACLE study is a Phase 2B/3 clinical trial whereby data from the 2B portion will be combined with the Phase 3 portion for purposes of measuring its primary efficacy endpoint. MIRACLE is subject to appropriate future filings with and potential additional feedback from the FDA and their foreign equivalents, utilizes an adaptive design whereby the first 75 to 90 subjects will be randomized (1:1:1) in Part A of the trial to receive high dose cytarabine (HiDAC) combined with either placebo, 190 mg/m2 of Annamycin, or 230 mg/m2 of Annamycin, which Annamycin doses were specifically recommended by the FDA in the Company’s end of Phase 1B/2 meeting.

The protocol for the MIRACLE trial allows for the unblinding of preliminary primary efficacy data (Complete Remission or CR) and safety/tolerability of the three arms at 45 subjects, in addition to the conclusion of Part A (at 75 to 90 subjects). The first early unblinding is expected to yield 30 subjects treated with Annamycin (190mg/m2 and 230 mg/m2) in combination with HiDAC and 15 subjects treated with just HiDAC plus placebo. The Company expects to reach the first unblinding (45 subjects) in the second half of 2025, in addition to the second unblinding, which is expected in the first half of 2026. This accelerated estimated timeline is due in part to the positive response the Company received in meetings during December with potential investigators regarding recruitment for the trial.

As previously announced with regard to the EU, the clinical trial approval with EMA was granted under the condition that the Company present results of appropriate nonclinical GLP studies before initiating the Phase 3 portion (Part B) of the study. Results will be submitted as a substantial modification to the existing approved CTA.

For Part B of the trial, approximately 220 additional subjects will be randomized to receive either HiDAC plus placebo or HiDAC plus the optimum dose of Annamycin (randomized 1:1). The selection of the optimum dose will be based on the overall balance of safety, pharmacokinetics and efficacy, consistent with the FDA’s new Project Optimus initiative.

Patient dosing has commenced, and the initial data readout is on track for the second half of 2025. For more information about the MIRACLE trial, visit clinicaltrials.gov and reference identifier NCT06788756. Additionally, the clinical trial in the EU is on euclinicaltrials.eu and the reference identifier there is 2024-518359-47-00.

Annamycin, also known by its non-proprietary name of naxtarubicin, currently has Fast Track Status and Orphan Drug Designation from the FDA for the treatment of relapsed or refractory acute myeloid leukemia, in addition to Orphan Drug Designation for the treatment of soft tissue sarcoma. Furthermore, Annamycin has Orphan Drug Designation for the treatment of relapsed or refractory acute myeloid leukemia from the EMA.

On September 8, 2025 Cellworks Group Inc., a leader in Personalized Therapy Decision Support and Best-in-Class PTRS, reported results from a new study showing that the combination of tumor microenvironment (TME) composition and tertiary lymphoid structure (TLS) dynamics is a key predictor of how individual patients with non-small cell lung cancer (NSCLC) respond to immunotherapy (Press release, Cellworks, SEP 8, 2025, View Source [SID1234655846]). The study introduces a novel prediction model that integrates TME cell composition with a 34-gene TLS score. Together, these measures enabled the Cellworks Platform to accurately predict patient-level survival outcomes and reveal meaningful differences among NSCLC patients treated with checkpoint inhibitors.

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Results from the study were showcased in a poster presentation titled, "Cellular Heterogeneity and Tertiary Lymphoid Structure Dynamics Predict Overall Survival in Immune Checkpoint Therapy-Treated NSCLC Patients," as part of the IASLC 2025 World Conference on Lung Cancer (#WCLC25) hosted by the International Association for the Study of Lung Cancer held from September 6-9, 2025 in Barcelona, Spain.

"Despite the promise of immune checkpoint inhibitors (ICIs), only a subset of NSCLC patients benefit from these therapies," said Charu Aggarwal, MD, MPH, FASCO, Leslye M. Heisler Professor of Lung Cancer Excellence in the Perelman School of Medicine at the University of Pennsylvania, and co-author on the study. "This study provides new insights into how the structural organization of tertiary lymphoid structures and the immune cell composition of the tumor microenvironment jointly determine immunotherapy response. By moving beyond single biomarkers, this approach holds promise as a more comprehensive way to guide personalized treatment decisions in NSCLC."

"Our findings highlight that both cellular heterogeneity and TLS dynamics play critical roles in determining whether patients respond to checkpoint inhibitor therapy," said James Wingrove, PhD, Chief Development Officer at Cellworks and presenting author of the study. "By integrating these factors, we created a personalized model that can offer oncologists new insight into which patients are most likely to benefit from checkpoint inhibitors. This study underscores how computational modeling of the tumor microenvironment can advance personalized decision support in NSCLC."

"What makes this work exciting is the ability to connect molecular signals within the tumor microenvironment to real patient outcomes," said Michael Castro, MD, Chief Medical Officer at Cellworks. "By biosimulating how both cellular heterogeneity and TLS dynamics shape immunotherapy response, we move closer to a future where treatment selection is not just based on broad population markers, but on each patient’s unique tumor biology. This level of personalization has the potential to identify which patients benefit from combination chemotherapy and immunotherapy while also sparing other patients from receiving chemotherapy when it is unlikely to benefit."

Key Findings

Strong Predictive Value – The integrated TLS + TME model demonstrated high predictive significance for overall survival in both the training (HR=0.36, C-Index=0.768) and validation cohorts (HR=0.66, C-Index=0.628).
Clear Survival Differences – Patients predicted to have a high benefit from immune checkpoint inhibition showed a significant increase in overall survival, living a median of 30.8 months versus 12 months for patients predicted to have low benefit.
Immune Balance Matters – Survival benefit was tied to immune balance: pro-inflammatory environments enhanced TLS benefit, while suppressive immune cells (like neutrophils and M2-like macrophages) reduced or reversed it.
New Insights for Personalization – The integration of TLS dynamics with TME immune and stromal cell composition provided independent yet complementary insights, showing how structural organization and cellular balance cooperatively determine immunotherapy efficacy and can guide personalized treatment decisions.
Study Design

Cellworks developed and cross-validated an algorithm that deconvolutes bulk transcriptomic data to estimate cell proportions and cell-type–specific gene expression within the TME. This approach was enhanced with a TLS Score based on 34 genes representing cellular interplay and maturity, derived from bulk RNA-sequencing data of tumor samples. By integrating TLS dynamics with immune and stromal cell populations, the model captured complementary and independent contributions, providing collective insight into how tumor structure and cellular composition determine ICI efficacy. A Cox proportional hazards model was trained on advanced NSCLC patients treated with ICIs (n=63, SU2C-MARK cohort) and validated in an independent cohort of 66 patients from the same study. The locked model confirmed predictive performance at the individual patient level, underscoring its potential clinical utility.

The Cellworks Platform

The Cellworks Platform performs computational biosimulation of protein-protein interactions, enabling in silico modeling of tumor behavior using genomic data derived from next-generation sequencing (NGS). This allows for the evaluation of how personalized treatment strategies interact with the patient’s unique tumor network. Multi-omic data from an individual patient or cohort is used as input to the in silico Cellworks Computational Biology Model (CBM) to generate a personalized or cohort-specific disease model.

The CBM is a highly curated mechanistic network of 6,000+ human genes, 30,000+ molecular species and over 600,000 molecular interactions. This model along with associated drug models are used to biosimulate the impact of specific compounds or combinations of drugs on the patient or cohort and produce therapy response predictions, which are statistically modeled to produce a qualitative therapy response score for a specific therapy. The Cellworks CBM has been tested and applied against various clinical datasets with results provided in more than 125 presentations and publications with global collaborators.

On September 8, 2025 Immutep Limited (ASX: IMM; NASDAQ: IMMP) ("Immutep" or "the Company"), a late-stage immunotherapy company targeting cancer and autoimmune diseases, reported an abstract for the EFTISARC-NEO Phase II investigator-initiated trial has been accepted for oral presentation at the Connective Tissue Oncology Society (CTOS) 2025 Annual Meeting taking place 12-15 November 2025, in Boca Raton, Florida (Press release, Immutep, SEP 8, 2025, View Source [SID1234655800]).

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EFTISARC-NEO is the first study to evaluate eftilagimod alpha (efti) in a neoadjuvant setting (prior to surgery) administered in combination with radiotherapy plus KEYTRUDA (pembrolizumab) for patients with soft tissue sarcoma (STS).

Presentation Details
Title: Primary endpoint and translational correlates from EFTISARC-NEO: phase II trial of neoadjuvant eftilagimod alpha (efti), pembrolizumab, and radiotherapy in patients with resectable soft tissue sarcoma
Session: Immunotherapy & Cell Therapy in Sarcoma: Emerging Frontiers
Presenter: Pawel Sobczuk, M.D., Ph.D., Department of Soft Tissue/Bone Sarcoma and Melanoma, Maria Sklodowska-Curie National Research Institute of Oncology
Date: Thursday, 13 November 2025, 1:30 PM – 3:00 PM ET
Format: Oral Presentation
STS is an orphan disease with high unmet medical need and a poor prognosis for patients. The incidence of STS varies in different regions globally. In the United States, the number of new STS cases in 2025 is estimated to be ~13,520 with ~5,420 deaths, according to the American Cancer Society.1

EFTISARC-NEO is is being conducted by the Maria Skłodowska-Curie National Research Institute of Oncology in Warsaw, Poland. The study is primarily funded with an approved grant from the Polish government awarded by the Polish Medical Research Agency program. For more information on the trial visit clinicaltrials.gov (NCT06128863).

The presentation slides will be available on the Posters & Publications section of Immutep’s website after the presentation at CTOS 2025.