On July 8, 2025 Syntara Limited (ASX: SNT), a clinical-stage drug development company, reported that the World Health Organization (WHO) has formally granted the International Non-Proprietary Name (INN) of amsulostat to its advanced clinical development asset SNT-5505 (Press release, Syntara, JUL 8, 2025, View Source [SID1234654266]).

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Commonly known as a generic name, an INN is a globally recognised, unique name for a pharmaceutical substance or active ingredient.

Amsulostat is an innovative oral therapy currently in clinical development for myelofibrosis (MF), a debilitating bone marrow disorder characterised by fibrosis that severely impacts blood cell production.

Recent interim Phase 2 clinical trial results, which demonstrated promising efficacy and an excellent safety profile in combination with ruxolitinib, underscore the potential of amsulostat as a differentiated treatment option for patients with MF who exhibit suboptimal responses to existing therapies.

The designation of an INN represents an important milestone in the drug development process, providing global recognition of amsulostat’s unique chemical identity and facilitating clearer communication among healthcare professionals and regulatory bodies worldwide.

Syntara CEO Gary Phillips said: "After very recently being awarded Fast Track Designation by the FDA, the granting of amsulostat as the INN for SNT-5505 is another important step forward, reflecting the drug’s unique mechanism of action and clinical promise. We remain focused on advancing amsulostat through clinical development to address significant unmet medical needs in myelofibrosis and other fibrotic diseases."

On July 7, 2025 NANOBIOTIX (Euronext: NANO –– NASDAQ: NBTX – the ‘‘Company’’), a late-stage clinical biotechnology company pioneering physics-based approaches to expand treatment possibilities for patients with cancer, reported two important developments that aim to reinforce the global positioning of potential first-in-class radioenhancer JNJ-1900 (NBTXR3), which is licensed by Janssen Pharmaceutica NV, a Johnson & Johnson company (Press release, Nanobiotix, JUL 7, 2025, View Source [SID1234654360]).

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Health authorities in major European countries have agreed to formally reclassify JNJ-1900 (NBTXR3) from a medical device to a drug, completing a process initiated by Johnson & Johnson to harmonize the product candidate’s regulatory status with classifications already in place in the United States and other major markets. The reclassification follows updated insights into the product candidate’s mechanism of action.

In parallel, Nanobiotix has filed a new composition of matter patent for JNJ-1900 (NBTXR3) that aims to reinforce the intellectual property foundation supporting the product candidate.

"These updates reflect our continued commitment to the JNJ-1900 (NBTXR3) program," said Laurent Levy, co-founder and chairman of the executive board at Nanobiotix. "We are pleased with the progress achieved with health authorities and proud to continue advancing this potential first-in-class product candidate toward patients in need."

JNJ-1900 (NBTXR3) is currently the subject of a comprehensive global clinical development program across multiple tumor types and therapeutic combinations, including a pivotal Phase 3 trial in head and neck cancer.

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 July 7, 2025 Natera, Inc. (NASDAQ: NTRA), a global leader in cell-free DNA and precision medicine, reported data from a colorectal cancer (CRC) study of its tissue-free Latitude MRD assay, which were presented at the 2025 European Society for Medical Oncology GI Congress (ESMO GI) (Press release, Natera, JUL 7, 2025, View Source [SID1234654272]).

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The clinical performance of the assay was analyzed in a cohort of ~200 patients (>1,300 plasma samples) with resectable stage I-IV colorectal cancer. Samples were derived from the GALAXY arm of the CIRCULATE-Japan study, one of the largest and most comprehensive prospective studies of MRD testing in resectable CRC. Key results include:

Excellent clinical sensitivity to recurrence: The Latitude assay detected recurrence with 58% sensitivity in the post-surgical MRD window and 81% sensitivity in the surveillance setting, with median diagnostic lead time of 4.6 months.
High clinical specificity: In the surveillance setting, patient-level specificity was 92%, and sample-level specificity was 97%.
Highly prognostic of recurrence risk: MRD positivity was significantly associated with inferior outcomes during both the MRD (HR: 10, p<0.001) and surveillance (HR: 18, p<0.001) windows.
Highly predictive of adjuvant chemotherapy (ACT) benefit: Among high-risk stage II and stage III patients, those who were MRD-positive after surgery derived significant benefit from ACT (p<0.001), while no significant treatment benefit was observed in MRD-negative patients (p=0.54).
"This study demonstrates strong clinical potential for Natera’s tissue-free MRD assay," said Yoshiaki Nakamura, M.D., Ph.D., principal investigator of the study. "We clearly observed the prognostic and predictive value of the assay, seeing strong correlation of recurrence risk and adjuvant treatment outcomes."

"These results reinforce the data previously presented at ASCO (Free ASCO Whitepaper) GI, where our tissue-free Latitude MRD assay demonstrated high overall concordance to Signatera as well as strong standalone clinical performance," said Alexey Aleshin, M.D., corporate chief medical officer and general manager of oncology at Natera. "When offered alongside Signatera, our tissue-free assay gives patients and providers the latitude to get a reliable MRD assessment even when tissue is unavailable."

On July 7, 2025 PanTher Therapeutics ("PanTher" or the "Company"), a clinical-stage company redefining cancer treatment with therapeutics administered continuously and exclusively at the tumor site, reported that the first patient has been treated with PTM-101 in a Phase 1b clinical trial in pancreatic ductal adenocarcinoma (PDAC) (Press release, PanTher Therapeutics, JUL 7, 2025, View Source [SID1234654271]). PTM-101 is the most advanced product candidate within PanTher’s portfolio of innovative formulations for continuous, high-dose, localized drug administration directly to the site of the tumor.

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"The start of this second clinical trial of PTM-101 is an exciting milestone in our journey to transcend the dosing limitations of today’s cancer treatments by reimagining how chemotherapy is delivered," said Laura Indolfi, Ph.D., Chief Executive Officer and Co-founder of PanTher Therapeutics. "Powerful cancer drugs exist but their toxicity lowers maximum dose and limits dosing frequency — leaving too many opportunities for cancers to continue spreading while patients grapple with debilitating side effects. Our investigational formulations are designed to circumvent the toxic effects of systemic chemotherapy while retaining a much higher dose of the drug exclusively at the tumor, with the goal of shrinking difficult-to-treat tumors and extending patients’ lives."

PTM-101 is a polymeric thin film formulation of paclitaxel, a well-established chemotherapy drug, designed to deliver a sustained (~6 weeks) high dose to the tumor site with little to no systemic exposure. A previous first-in-human Phase 1 study (ACTRN12621000881831) of PTM-101 at the 100 mg dose level, combined with standard of care chemotherapy in borderline resectable and locally advanced PDAC, reported promising tumor shrinkage and a favorable safety profile. No paclitaxel was detected systemically at any time. The study additionally demonstrated PTM-101’s ability to fit into current PDAC treatment protocols and deliver potential therapeutic benefit early in the clinical paradigm, beginning weeks before intravenous chemotherapy.

The ongoing dose escalation and expansion Phase 1b study (NCT06673017) is assessing safety, tolerability, and anti-tumor activity of PTM-101 at two higher dose levels when combined with standard of care neoadjuvant chemotherapy (FOLFIRINOX) in patients with borderline resectable or locally advanced PDAC. The first patient was dosed at Virginia Mason Medical Center in Seattle, Washington, under Drs. Vince Picozzi and William "Scott" Helton. In addition to Virginia Mason, trial enrollment is presently ongoing at Northwell Health Zuckerberg Cancer Center in Lake Success, New York; Hoag Memorial Hospital Presbyterian in Newport Beach, California; and the Barbara Ann Karmanos Cancer Institute in Detroit, Michigan. This non-randomized, open-label study plans to enroll approximately 30 treatment-naïve patients across multiple clinical sites in the U.S.

"PTM-101 is a novel, innovative approach to treating the primary pancreatic tumor," said Vince Picozzi, M.D., a medical oncologist and a principal investigator of the Phase 1b clinical trial. "Doing so successfully is the first step towards curative therapy."

Studies have shown that only about 1% of systemically delivered chemotherapy reaches the tumor, with the remaining 99% of drug producing toxic effects on off-target tissues — including neutropenia, hair loss, nausea and vomiting, and peripheral neuropathy. Additionally, pancreatic cancer is notoriously poorly vascularized, making it extremely difficult for systemic chemotherapy to reach therapeutic levels at the tumor site.

"Targeted cancer treatment is an area of immense interest for oncology," said Scott Helton, M.D., a pancreatic surgeon in Seattle, Washington. "The ability of PTM-101 to integrate into our current PDAC care pathway is promising, offering the possibility of transforming a diagnostic step into the start of therapy, weeks before the patient can begin intravenous chemotherapy."

PanTher is additionally developing polymeric drug formulations for the treatment of a range of other solid tumor types.

About PTM-101

PanTher’s most advanced product candidate, PTM-101, is an absorbable thin film formulation of paclitaxel for non-metastatic pancreatic cancer. PTM-101 is designed to deliver continuous, long-term, high-dose chemotherapy to the tumor with little to no systemic exposure. The product, laparoscopically implanted at the tumor site, easily integrates with common minimally-invasive procedures used in staging pancreatic cancer. PTM-101 is currently being evaluated in a Phase 1b clinical trial (NCT06673017) with support from the Cancer Prevention & Research Institute of Texas (CPRIT) DP220066.

On July 7, 2025 FUJIFILM Pharmaceuticals U.S.A., Inc., drug development center and a leading provider of Contract Development and Manufacturing Organization (CDMO) services for drug delivery system (DDS) technologies, reported that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Fujifilm’s FF-10832 — an investigational liposomal formulation of gemcitabine — for the treatment of biliary tract cancer (BTC) (Press release, Fujifilm, JUL 7, 2025, View Source [SID1234654270]). Phase 1 study (NCT03440450) results presented at ASCO (Free ASCO Whitepaper) 2025 suggest FF-10832 is well tolerated and has anti-tumor activity in patients with advanced BTC. FF-10832 is currently being evaluated in phase 2a studies (NCT05318573) as monotherapy or in combination with pembrolizumab for the treatment of solid tumors in the U.S.

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The FDA grants orphan drug designation to support the development of therapies for rare diseases that affect fewer than 200,000 people in the U.S.1 Small patient populations can limit preclinical research and slow clinical trials, stalling progress in therapeutic development for rare diseases. The designation supports research and clinical development by providing 7-years marketing exclusivity and other financial incentives.

There are approximately 16,000 new cases of BTC in the U.S per year.2 Most (≥70%) patients present with unresectable or metastatic disease at diagnosis, and current treatments of surgery, chemotherapy, and radiation show limited efficacy in advanced stages. High recurrence (50–70%) and low 5-year survival rates (4–13%) highlight the urgent need for new and effective treatments.

Gemcitabine has been a key component of BTC treatment since its approval in the 1990s, with all major first line regimens currently containing gemcitabine.3 FF-10832’s novel liposomal formulation of gemcitabine for intravenous administration is designed to enhance anti-tumor activity by prolonging plasma half-life and improving targeted delivery to tumors.

"BTCs are rare but aggressive malignancies associated with a poor prognosis and limited treatment options," said Susumu Shimoyama, president, FUJIFILM Pharmaceuticals U.S.A., Inc. "Receiving orphan drug designation highlights the significant unmet medical need that still remains and supports development of FF-10832 for patients with BTC who have few satisfactory options."

FF-10832 is manufactured by FUJIFILM Toyama Chemical, providing seamless CDMO end-to-end services for full integration from formulation development to GMP manufacturing. Fujifilm’s investigational drug candidates FF-10502 and FF-10850 have also been granted orphan drug designations for cholangiocarcinoma and Merkel cell carcinoma, respectively.