On September 17, 2025 NANOBIOTIX (Euronext: NANO –– NASDAQ: NBTX – the ‘‘Company’’), a late-clinical stage biotechnology company pioneering nanotherapeutic approaches to expand treatment possibilities for patients with cancer and other major diseases, reported new results focused on patients with primary cutaneous melanoma from the ongoing Phase 1 Study 1100 evaluating JNJ-1900 (NBTXR3) in combination with immune checkpoint inhibitors (pembrolizumab or nivolumab) for patients with advanced cancers (Press release, Nanobiotix, SEP 17, 2025, View Source [SID1234656034]). These findings were presented at the 2025 ImmunoRad conference in Paris, France on September 17.

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Patients with anti-PD-1 resistant primary cutaneous melanoma in the study had advanced disease that progressed despite multiple prior lines of therapy, including anti–PD-1, ipilimumab, T-VEC, TIL, and radiotherapy ("RT"), among others. All patients received a one-time intratumoral injection of JNJ-1900 (NBTXR3) followed by RT and sequential anti–PD-1 therapy. As of the August 21, 2025, data cutoff, 21 patients had been injected with JNJ-1900 and 19 were evaluable for tumor response (2 patients were not evaluable due to lack of post-treatment imaging).

"The patients in this analysis represent one of the more difficult clinical challenges we face as many have exhausted standard therapies, including checkpoint inhibitors," said Study 1100 Coordinating Investigator Colette Shen, MD, PhD, Assistant Professor of Radiation Oncology, University of North Carolina Lineberger Comprehensive Cancer Center. "While these data are preliminary, the responses we’re seeing provide a strong signal that this treatment approach could potentially offer a new possibility for patients who need more options."

Safety and Feasibility

All 21 patients with primary cutaneous melanoma had shown prior resistance to anti-PD-1 and treatment with RT-activated JNJ-1900 (NBTXR3) followed by anti-PD-1 showed a favorable safety profile:

Injection feasibility was confirmed at the recommended Phase 2 dose (33% GTV)
In total, 16 patients experienced grade 1, grade 2, or grade 3+ TEAEs related to the overall therapeutic regimen (RT, anti-PD-1, JNJ-1900 (NBTXR3), and injection procedure)
Of which 5 patients experienced grade 1, grade 2, or grade 3+ treatment-emergent adverse events (TEAEs) related to JNJ-1900 (NBTXR3) and/or the injection procedure
Of these patients, 1 patient experienced grade 3+ TEAEs (hypotension and pleuritic pain)
Early Signs of Efficacy

JNJ-1900 (NBTXR3) demonstrated preliminary signals of efficacy in 19 patients who were evaluable for tumor response:

A best observed objective response rate ("ORR") in all lesions of 47.4% (9/19) per RECIST 1.1, including 4 complete responses and 5 partial responses
A best observed disease control rate ("DCR") in all lesions of 78.9% (15/19) per RECIST 1.1
In JNJ-1900 injected & irradiated tumors, a DCR of 100% (19/19) was observed
A median Overall Survival (mOS) of 14.6 months [95% CI: 10.7 months; 16.7 months] in all patients treated (n=21)
Notably, a relationship was observed between the depth of local response and systemic tumor regression, suggesting a possible priming or re-activation of immune response.

"We are encouraged by these new findings and the potential signals of activity in this difficult-to-treat population," said Louis Kayitalire, MD, Chief Medical Officer of Nanobiotix. "Notably, the relationship we observed between the depth of local response and systemic tumor regression further supports our hypothesis regarding the potentially broad applicability of JNJ-1900 (NBTXR3) for patients with cancer. We look forward to further clinical evaluation of JNJ-1900 (NBTXR3) to better understand its capacity to drive both local and systemic responses in primary cutaneous melanoma."

Nanobiotix Conference Call

Nanobiotix will host a conference call and webcast featuring Nanobiotix chief executive officer, Laurent Levy, to discuss the new data on Thursday, September 18th, 2025, at 8:00 AM EDT / 2:00 PM CET.

Details for the call are as follows:

Webcast link: View Source

Audio-only dial-in link: View Source

Participants can use the audio-only link above to register and obtain dial-in instructions to listen to the presentation via phone and ask questions during the Q&A session, or participants can use the webcast link to register and listen and watch the slide presentation online; the replay version will be available under the same webcast link shortly after the presentation and will be archived on the Company’s website at www.nanobiotix.com. It is recommended to join 10 minutes prior to the event start. Participants are invited to email their questions in advance to [email protected].

About JNJ-1900 (NBTXR3)

JNJ-1900 (NBTXR3) is a novel, potentially first-in-class oncology product composed of functionalized hafnium oxide nanoparticles that is administered via one-time intratumoral injection and activated by radiotherapy. Its proof-of-concept was achieved in soft tissue sarcomas through a successful randomized Phase 2/3 study in 2018. The product candidate’s mechanism of action (MoA) is designed to induce significant tumor cell death in the injected tumor when activated by radiotherapy, subsequently triggering adaptive immune response and long-term anti-cancer memory. Given the physical MoA, Nanobiotix believes that JNJ-1900 (NBTXR3) could be scalable across any solid tumor that can be treated with radiotherapy and across any therapeutic combination, particularly immune checkpoint inhibitors.

Radiotherapy-activated JNJ-1900 (NBTXR3) is being evaluated across multiple solid tumor indications as a single agent or combination therapy. The program is led by NANORAY-312—a global, randomized Phase 3 study in locally advanced head and neck squamous cell cancers. In February 2020, the United States Food and Drug Administration granted regulatory Fast Track designation for the investigation of JNJ-1900 (NBTXR3) activated by radiation therapy, with or without cetuximab, for the treatment of patients with locally advanced HNSCC who are not eligible for platinum-based chemotherapy—the same population being evaluated in the Phase 3 study.

Given the Company’s focus areas, and balanced against the scalable potential of NBTXR3, Nanobiotix has engaged in a collaboration strategy to expand development of the product candidate in parallel with its priority development pathways. Pursuant to this strategy, in 2019 Nanobiotix entered into a broad, comprehensive clinical research collaboration with The University of Texas MD Anderson Cancer Center to sponsor several Phase 1 and Phase 2 studies evaluating JNJ-1900 (NBTXR3) across tumor types and therapeutic combinations. In 2023, Nanobiotix announced a license agreement for the global co-development and commercialization of JNJ-1900 (NBTXR3) with Janssen Pharmaceutica NV, a Johnson & Johnson company.

On September 17, 2025 Propanc Biopharma, Inc. (Nasdaq: PPCB) ("Propanc" or the "Company"), a biopharmaceutical company developing novel cancer treatments for patients suffering from recurring and metastatic cancer, reported that a certificate of grant for the Company’s "proenzyme composition" patent was received from the US Patent & Trademark Office (USPTO) (Press release, Propanc, SEP 17, 2025, View Source [SID1234656035]). The patent specifically captures a future clinical dose of the Company’s lead asset, PRP. This is the fourth US patent granted in this key strategic jurisdiction. Currently, the Company’s intellectual property portfolio consists of 90 patents filed in major jurisdictions relating to the use of PRP against solid tumors.

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The proenzymes composition patent is an important part of the IP portfolio covering a possible future clinical dose of PRP, as the Company advances to a Phase 1B, First-In-Human (FIH) study in advanced cancer patients suffering from solid tumors. The patent has also been granted in other major jurisdictions such as Europe, Japan and throughout Southeast Asia. PRP is targeting the global metastatic cancer treatment market, projected to be worth US$111.2 billion by 2027, according to Emergen Research.

"We continue to grow our intellectual property portfolio in the United States, which is our most important jurisdiction," said Mr. James Nathanielsz, Propanc’s Chief Executive Officer. "Our lead asset, PRP, is an exciting method to prevent and treat metastatic cancer from solid tumors without the severe, or even serious side effects often associated with standard therapies. PRP is relatively non-toxic because it does not kill cancer cells directly but induces cell differentiation so that they become less malignant and die off naturally. This unique phenomenon is specific to PRP, which acts as an ‘EMT (epithelial to mesenechymal transition) modulator’, enforcing the cancer cells to behave more as a normalized cell so that it is no longer a threat. We are pushing towards the commencement of the Phase 1B study next year. We are also pursuing every avenue for raising sufficient capital and employing a digital asset diversification strategy to ensure optimal cash flow as we advance PRP towards important clinical milestones."

On September 17, 2025 Propanc Biopharma, Inc. (Nasdaq: PPCB) ("Propanc" or the "Company"), a biopharmaceutical company developing novel cancer treatments for patients suffering from recurring and metastatic cancer, reported that a certificate of grant for the Company’s "proenzyme composition" patent was received from the US Patent & Trademark Office (USPTO) (Press release, Propanc, SEP 17, 2025, View Source [SID1234656035]). The patent specifically captures a future clinical dose of the Company’s lead asset, PRP. This is the fourth US patent granted in this key strategic jurisdiction. Currently, the Company’s intellectual property portfolio consists of 90 patents filed in major jurisdictions relating to the use of PRP against solid tumors.

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Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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The proenzymes composition patent is an important part of the IP portfolio covering a possible future clinical dose of PRP, as the Company advances to a Phase 1B, First-In-Human (FIH) study in advanced cancer patients suffering from solid tumors. The patent has also been granted in other major jurisdictions such as Europe, Japan and throughout Southeast Asia. PRP is targeting the global metastatic cancer treatment market, projected to be worth US$111.2 billion by 2027, according to Emergen Research.

"We continue to grow our intellectual property portfolio in the United States, which is our most important jurisdiction," said Mr. James Nathanielsz, Propanc’s Chief Executive Officer. "Our lead asset, PRP, is an exciting method to prevent and treat metastatic cancer from solid tumors without the severe, or even serious side effects often associated with standard therapies. PRP is relatively non-toxic because it does not kill cancer cells directly but induces cell differentiation so that they become less malignant and die off naturally. This unique phenomenon is specific to PRP, which acts as an ‘EMT (epithelial to mesenechymal transition) modulator’, enforcing the cancer cells to behave more as a normalized cell so that it is no longer a threat. We are pushing towards the commencement of the Phase 1B study next year. We are also pursuing every avenue for raising sufficient capital and employing a digital asset diversification strategy to ensure optimal cash flow as we advance PRP towards important clinical milestones."

On September 17, 2025 Rezolute, Inc. (Nasdaq: RZLT) ("Rezolute" or the "Company"), a late-stage rare disease company focused on treating hypoglycemia caused by hyperinsulinism, reported financial results and provided a business update for the fourth quarter and full fiscal year ended June 30, 2025 (Press release, Rezolute, SEP 17, 2025, View Source [SID1234656036]).

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"We have made substantial progress this year across our two indications for ersodetug in both congenital and tumor hyperinsulinism," said Nevan Charles Elam, Chief Executive Officer and Founder of Rezolute. "We believe that FDA alignment on a streamlined Phase 3 trial in tumor hyperinsulinism is further recognition of ersodetug’s broad applicability across multiple forms of hyperinsulinism and highlights both the urgent need and the transformative potential of our therapy for patients and families living with this condition. We remain on track to report topline results from the sunRIZE trial in December and look forward to progressing towards potential commercialization."

Recent Pipeline Progress and Anticipated Milestones

Congenital Hyperinsulinism (HI)

· Completed enrollment in sunRIZE, a Phase 3, multicenter, double-blind, randomized, controlled safety and efficacy registrational study of ersodetug for the treatment of congenital HI.

○ Exceeded enrollment with 62 participants enrolled, including approximately 15 percent from U.S. sites.

○ Topline results expected in December 2025.

· Presented "Preliminary Patient Demographics And Baseline Characteristics From A Phase 3 Study (sunRIZE) Of Ersodetug For Hypoglycemia Due To Congenital Hyperinsulinism: Trial In Progress" at the Annual Meeting of the Endocrine Society (ENDO 2025). The enrolled population is comparable to the Phase 2 RIZE study and include:

○ 3.4y average age: 35% <2 years old

○ 15 (average) hypoglycemia events/week

○ 19% daily percent time in hypoglycemia

○ 95% taking ≥1 SOC treatments

Tumor HI

· In August 2025, the Company achieved alignment with FDA on a significantly streamlined clinical development path for its ongoing Phase 3 study (upLIFT) of ersodetug for the treatment tumor HI.

o The truncated study will include as few as 16 participants and will be limited to the single-arm open-label portion of the upLIFT study, removing the need to conduct a double-blind randomized placebo-controlled trial.

o Enrollment is underway and topline results are expected in the second half of 2026.

Corporate Updates

· In August 2025 the Company appointed Dr. Sunil Karnawat as Chief Commercial Officer.

o Dr. Karnawat has over 25 years of experience in global commercialization of biopharmaceuticals and medical devices and will spearhead launch strategy and global market readiness for ersodetug.

o Before joining Rezolute, Dr. Karnawat served as a Vice President at Cytokinetics and Ultragenyx. At Ultragenyx, he was responsible for leading key commercial functions in launching four ultra-rare disease products, including Crysvita.

Fourth Quarter and Full Year Fiscal 2025 Financial Results

Cash, cash equivalents and investments in marketable securities were $167.9 million as of June 30, 2025, compared with $127.1 million as of June 30, 2024.

Research and development (R&D) expenses were $20.9 million for the fourth quarter of fiscal 2025, compared with $19.1 million for the same period a year ago. Full fiscal year 2025 R&D expenses were $61.5 million, compared to $55.7 million in fiscal year 2024. The increase from fiscal year 2024 to fiscal year 2025 was primarily due to (i) increased expenditures in clinical trial activities, (ii) manufacturing costs for ersodetug, and (iii) higher employee-related expenses, which included employee compensation and stock-based compensation.

General and administrative (G&A) expenses were $5.0 million for the fourth quarter of fiscal 2025, compared with $4.0 million for the same period a year ago. Full fiscal year 2025 G&A expenses were $18.4 million, compared to $14.7 million in fiscal year 2024. The increase was primarily attributable to professional fees and employee-related expenses due to increased headcount.

Net loss was $24.4 million for the fourth quarter of fiscal 2025 compared with a net loss of $23.0 million for the same period a year ago. Full year fiscal 2025 net loss was $74.4 million compared to net loss of $68.5 million for the fiscal year 2024.

About Ersodetug

Ersodetug is a fully human monoclonal antibody that binds allosterically to the insulin receptor to decrease receptor over-activation by insulin and related substances (such as IGF-2) in the setting of hyperinsulinism (HI), thereby improving hypoglycemia. Because ersodetug acts downstream from the pancreas, it has the potential to be universally effective at treating hypoglycemia due to any congenital or acquired form of HI.

On September 16, 2025 Oryzon Genomics, S.A. (ISIN Code: ES0167733015, ORY), a clinical-stage biopharmaceutical company and a European leader in epigenetics, reported that it continues to expand its patent portfolio for iadademstat and vafidemstat, Oryzon’s clinical-stage LSD1 inhibitors for oncology and central nervous system (CNS) disorders, following new Decisions to Grant from the Australian and European patent offices (Press release, Oryzon, SEP 16, 2025, View Source;utm_medium=email&utm_campaign=NdP.27+2025-09-16+IP+additional+decision+grant+ENG778 [SID1234655986]).

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The Australian Patent Office has issued a Decision to Grant for Oryzon’s patent application AU2020249493, titled "Combinations of iadademstat for cancer therapy". The allowed claims cover combinations of iadademstat with PD1 or PD-L1 inhibitors for the treatment of cancer, including small cell lung cancer (SCLC). A Decision to Grant is an official communication from a national patent office indicating that a patent application has met all requirements for issuance as a patent. Once formally granted, this Australian patent will remain in force until at least 2040, not including any potential patent term extensions. A corresponding patent has also been granted in Russia, and applications are pending in Europe, the United States, Japan, China, and other countries.

Iadademstat is currently being evaluated in combination with PD-L1 inhibitors (atezolizumab or durvalumab) in first line SCLC patients with extensive disease in a Phase I/II trial conducted and sponsored by the U.S. National Cancer Institute (NCI) under a Cooperative Research and Development Agreement (CRADA) with Oryzon. More than 30 sites accross the U.S. participate in the trial, including leading institutions such as Memorial Sloan Kettering Cancer Center, Johns Hopkins, City of Hope, Yale University and the University of Chicago, among others.

Oryzon has also received a Decision to Grant from the European Patent Office for its patent application EP24205125.8, titled "Vafidemstat for treating behavior alterations". The allowed claims cover the use of vafidemstat for the treatment of aggressiveness and social withdrawal associated with CNS diseases. Among the allowed claims, there are claims specifically aimed at the treatment of aggressiveness associated with Borderline Personality Disorder (BPD), Autism Spectrum Disorder (ASD), Alzheimer’s disease and other conditions, as well as claims directed to treating social withdrawal associated with diseases such as schizophrenia or ASD. Once formally granted, this European patent will remain in force until at least 2038, not including any potential patent term extensions. Additional patents in this family have already been granted or allowed in Europe, Australia, Canada, Hong Kong, Israel, South Korea, Malaysia, the Philippines, and Russia, with applications pending in other countries.

Vafidemstat is in advanced clinical development for the treatment of aggression in psychiatric disorders, with an upcoming Phase III trial in aggression in BPD (protocol submitted), and a Phase II trial in aggression in ASD patients under preparation. In addition, a Phase II trial is ongoing in schizophrenia, with a focus on negative symptoms. One of the most prominent negative symptoms of schizophrenia is social withdrawal.

"These new patent grants strengthen Oryzon’s global IP position by protecting key therapeutic indications under clinical development for iadademstat and vafidemstat, thereby extending the commercial life for both compounds", said Neus Virgili, Oryzon’s Chief IP Officer.