On September 17, 2025 Microbiotica, a clinical-stage biopharma company developing a pipeline of oral precision microbiome medicines called live biotherapeutic products (LBPs), reported that patient recruitment is complete in its advanced melanoma (MELODY-1) trial (Press release, Microbiotica, SEP 17, 2025, View Source [SID1234656033]).

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This international trial has recruited 41 patients at clinical centres in the UK, France, Italy, and Spain. Initial results are expected in the first half of 2026.

MELODY-1 is a Phase 1b study to evaluate the safety and tolerability of MB097 given in combination with pembrolizumab in patients with melanoma who demonstrate primary resistance to anti-PD-1 therapy. It is a first-in-human, randomised open-label clinical trial with all patients receiving MB097 and pembrolizumab for up to six months. Half of the participants also receive vancomycin before starting the co-therapy to determine whether it helps the bacterial strains in MB097 embed and grow in the gut more efficiently. Participants benefiting from the treatment at the end of the initial six-month period may continue to receive pembrolizumab for up to an additional 18 months (approximately 24 months total).

Melanoma is a life-threatening skin cancer that can spread to other parts of the body in its advanced stages. PD-1 inhibitor immunotherapies have revolutionised cancer treatment and are now commonly used to treat melanoma. However, new treatment options are still needed to extend the benefit to patients for whom immunotherapies do not work (treatment-resistant patients). This can be up to 50% of all advanced melanoma patients. There is a growing body of evidence demonstrating that the make-up of the gut microbiome can significantly influence a patient’s ability to respond to immunotherapy.

MB097 is a once daily, orally administered LBP consisting of a defined consortium of nine strains of commensal bacteria designed to enhance the efficacy of immune checkpoint inhibitors (ICIs). The MELODY-1 study is designed to investigate the safety, tolerability, and initial signals of efficacy of MB097 in advanced (metastatic) melanoma, in combination with KEYTRUDA (pembrolizumab), MSD’s (Merck & Co., Inc., Rahway, NJ, USA) anti-PD-1 therapy, in patients with cutaneous melanoma who have failed to respond to immunotherapies. MSD has supplied KEYTRUDA (study identifiers NCT06540391; MSD KEYNOTE-E75; 023-507377-17) to Microbiotica.

The bacterial strains in MB097 were identified by analysing the microbiome of patients in multiple studies of ICIs in melanoma, including the MELRESIST study carried out with the company’s collaborators at Cambridge University Hospitals, UK. Collectively, the MB097 bacterial consortium provides microbiome signalling that appears to be needed for ICI response. Pre-clinical studies demonstrate that MB097 activates core pathways of the immune system including Cytotoxic T Lymphocytes and Natural Killer cells to enable them to kill tumour cells. Research to understand the mechanism of action of the nine bacterial strains has indicated that in addition to this immune-activating effect, the bacteria in MB097 produce metabolites that act directly at the site of the tumour.

Dr Robert Tansley, Microbiotica’s Chief Medical Officer, said, "It is another significant milestone that this clinical study is fully recruited. In cancer patients, the bacteria in MB097 appear to be associated with better response rates to immune checkpoint inhibitor therapies, such as anti-PD-1 drugs. MB097, with its precisely selected microbes based on data from responsive patients, in combination with ICIs, could therefore activate a therapeutic benefit for non-responding patients with advanced melanoma. Moreover, as the MB097 bacteria are found in healthy subjects as well as in patients who responded to ICIs, we anticipate a favourable safety profile. We thank the investigators and patients for participating in the study and look forward to the results."

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.

Schedule your 30 min Free 1stOncology Demo!
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

                  Schedule Your 30 min Free Demo!

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 16, 2025 Pasithea Therapeutics Corp. (NASDAQ: KTTA) ("Pasithea" or the "Company"), a clinical-stage biotechnology company developing PAS-004, a next-generation macrocyclic MEK inhibitor reported activation of two South Korean clinical trial sites participating in its Phase 1/1b open label study to assess the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of PAS-004, in adult participants with neurofibromatosis type 1 (NF1) with symptomatic and inoperable, incompletely resected, or recurrent plexiform neurofibromas (Press release, Pasithea Therapeutics, SEP 16, 2025, View Source [SID1234656001]).

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The South Korea clinical trial sites, ASAN Medical Centre and Severance Hospital Yonsei University Health System, are now actively recruiting NF1 trial participants.

Professor Lee Beom-Hee of the Department of Pediatrics at Asan Medical Center said, "I am very pleased to partner with the Pasithea team to initiate testing of PAS-004 in adult patients with plexiform neurofibromas associated with NF1 at Asan Medical Center. Our institution has the largest NF1 caseload in South Korea and a long history of research leadership in this field. Our team was among the first to report the therapeutic benefits of MEK inhibition on neurocognitive decline, café-au-lait spots, and growth retardation caused by neurofibromatosis. We are eager to evaluate PAS-004, a next-generation MEK inhibitor that to date has demonstrated a distinct pharmacokinetic profile and a more convenient dosing regimen, which we believe may provide important benefits for our NF1 patients."

Dr. Tiago Reis Marques, chief executive officer of Pasithea commented, "With access to world-class facilities and an estimated 10,000 NF1 patients in South Korea, we believe our clinical sites in the country will play a pivotal role in the success of this trial. We are excited to include South Korean patients in our NF1 study and look forward to advancing meaningful treatment options for this community."

Asan Medical Center is a reference hospital and the teaching hospital of the University of Ulsan College of Medicine, located in Seoul, South Korea. With 2,432 beds for patients and a total floor area of approximately 280,000 square meters, it is the largest hospital in South Korea.

Severance Hospital is a teaching hospital located in Sinchon-dong, Seodaemun District, South Korea. It is one of the oldest and biggest university hospitals in South Korea. It has 2,437 beds and treats approximately 2,500,000 outpatients and 840,000 inpatients annually.

About the Phase 1/1b Clinical Trial in Adult NF1 Patients

The primary objective of the Phase 1/1b study (NCT06961565) is to evaluate the safety and tolerability of PAS-004 when administered for one 28-day treatment cycle in adult NF1 participants with at least one and up to two additional target plexiform neurofibromas (PNs) that are symptomatic and inoperable, incompletely resected, or recurrent. Secondary objectives are (i) to identify the recommended Part B dose ("RPBD") or Maximum Tolerated Dose (MTD) of PAS-004, (ii) to characterize the PK and PD profile of PAS-004, (iii) to evaluate the preliminary efficacy of PAS-004 on target PN volume, (iv) to evaluate the preliminary efficacy of PAS-004 on the size, appearance, and associated symptoms of cutaneous neurofibromas (CNs), and (v) to evaluate the impact of PAS-004 on quality of life ("QOL") and any physical symptoms attributed to the target PN. Experimental objectives are (i) to evaluate the impact of PAS-004 on QOL and any physical symptoms attributed to CNs, (ii) to evaluate the impact of PAS-004 on pain and function attributed to PNs, and (iii) to investigate PAS-004 effects on CN tumor cellular and molecular biology.

The trial will be conducted in two parts. In Part A (dose escalation phase), following a screening period of up to 28 days, up to 24 eligible participants will be enrolled sequentially to receive one of four planned dose levels of PAS-004 tablets (4mg, 8mg, 12mg, 18mg) in a modified 3+3 design. Part A will identify the recommended RPBD. During Part B (expansion phase), approximately 24 eligible participants will be enrolled in parallel to receive one of two planned dose levels of PAS-004 tablets. Participants will be dosed at the RPBD level and at a dose level below the RPBD for up to six continuous 28-day treatment cycles. Part B will identify the recommended phase 2 dose (RP2D).

The study is planned to be conducted at five clinical trial sites in Australia, South Korea and the U.S.