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.

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 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.

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.

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 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 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]).

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 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 BeyondSpring Inc. (NASDAQ: BYSI) reported publication of a human clinical study in Med (Cell Press) demonstrating that Plinabulin, when combined with radiation and a checkpoint inhibitor, induces dendritic cell (DC) maturation and elicits tumor responses in patients across multiple cancer types who had failed prior ICI therapy (Press release, BeyondSpring Pharmaceuticals, JUL 7, 2025, View Source;utm_medium=rss&utm_campaign=beyondspring-publishes-human-clinical-study-in-med-cell-press-showing-plinabulin-driven-dendritic-cell-maturation-and-tumor-response-after-prior-checkpoint-inhibitor-failure [SID1234654257]). The study also identified a potential biomarker—baseline GEF-H1 immune signature—that may enable patient pre-selection and clinical response prediction.

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!

"These results offer early but important signals that Plinabulin’s dendritic cell maturation mechanism could play a pivotal role in reversing ICI-acquired resistance," said Dr. Steven Lin, M.D., Ph.D., corresponding author and Professor of Radiation Oncology at The University of Texas MD Anderson Cancer Center. "The ability of Plinabulin to activate the immune system in this setting is both scientifically intriguing and clinically promising—particularly given the durability of responses in some heavily pretreated patients."

Dr. Lin added, "It is especially noteworthy that Plinabulin combination demonstrated the best responses in non-small cell lung cancer, head and neck squamous cell carcinoma, and Hodgkin lymphoma."

"This study builds upon the seminal work of Nobel Laureate Dr. Ralph Steinman and Dr. Ira Mellman, who helped define the essential role of dendritic cells in immune activation," said Lan Huang, Ph.D., Co-Founder, Chairman, and CEO of BeyondSpring. "Plinabulin’s ability to drive dendritic cell maturation and induce immune responsiveness offers a potential breakthrough strategy for patients who are refractory or relapsed on checkpoint inhibitors. We are committed to advancing Plinabulin’s development in partnership with pioneering cancer research institutions like MD Anderson."

Triple I/O Combination Study Highlights

This investigator-initiated, Phase 1 translational trial (NCT04902040) evaluated a triple immunotherapy approach combining Plinabulin, radiation (RT), and anti-PD-1 checkpoint inhibitors in patients with eight cancer types who are refractory or relapsed on prior ICI therapy. RT was administered only during the first cycle. The primary endpoint was tumor response in non-irradiated lesions.

Clinical Results
Nineteen patients received the combination regimen—14 on pembrolizumab and 5 on nivolumab. Tumor responses were evaluable in 13 ICI-relapsed patients across eight tumor types. Objective response rate (ORR) was 23%, and disease control rate (DCR) was 54%. Clinically meaningful benefits (PR, partial response; SD, stable disease) were observed in NSCLC (2/2), HNSCC (2/3), and Hodgkin lymphoma (2/2). Both Hodgkin lymphoma patients had durable responses exceeding 19 months despite 12–16 prior lines of therapy.
Mechanism Confirmation
Plinabulin triggered DC maturation post-RT via GEF-H1 signaling. Flow cytometry of whole blood revealed increased expression of DC maturation markers (CCR7, CD80, CD83) and a shift in monocyte subpopulations from classical to proinflammatory phenotype in responders.
Biomarker Insight
Single-cell RNA sequencing differentiated responders from non-responders and identified baseline GEF-H1 immune gene expression as a potential predictive biomarker for Plinabulin response.
About the Med Publication

Lin S.H., Subbiah V., Cohen E.N. et al. "Plinabulin following radiation enhances dendritic cell maturation and checkpoint inhibitor retreatment of relapsed/refractory cancers." Med. Published June 27, 2025. (View Source(25)00179-5)

About the Plinabulin Basket Study

This open-label, single-arm Phase 1 basket study (NCT04902040) at MD Anderson Cancer Center investigates safety and efficacy of Plinabulin plus radiation and PD-1 inhibitor in patients refractory or relapsed after prior immunotherapy. The primary endpoint is investigator-assessed ORR (RECIST 1.1) in non-irradiated lesions; secondary endpoints include DCR.

Regimen
– Radiation (Cycle 1 and optional Cycle 2): Local consolidative RT (8 Gy × 3; 12.5 Gy × 4; or 4 Gy × 5) on Day 1. Optional sequential RT in Cycle 2 at investigator discretion.
– Plinabulin: 30 mg/m² on Days 1 and 4 of Cycle 1 (3–6 hours post-RT); Day 1 of Cycle 2 onward; Additional Day 4 in Cycle 2 if RT is given in Cycle 2.
– PD-1 inhibitor: Pembrolizumab 200 mg on Day 1 every 21 days or nivolumab 240 mg on Day 1 every 14 days × 2 doses per cycle.
About Plinabulin

Plinabulin is a first-in-class dendritic cell maturation agent that binds reversibly to a unique site on tubulin, destabilizing microtubules in a controlled manner to release GEF-H1 (Chem 2019; Cell Reports 2019). Immune protein GEF-H1 activates the RhoA/ROCK signaling pathway, promoting dendritic cell maturation and anti-tumor T-cell immunity. This mechanism is distinct from traditional tubulin agents and does not interfere with tubulin stabilizers like docetaxel.

Across multiple clinical studies and approximately 800 patients, Plinabulin has shown durable anti-cancer activity and a favorable safety profile, and has significantly reduced chemotherapy-induced neutropenia, potentially enhancing docetaxel tolerability.

Prior Findings:
– In the Dublin-3 Phase 3 second and third line (2/3L) NSCLC, EGFR wild-type trial (n=559), Plinabulin + docetaxel demonstrated a significant overall survival benefit over standard-of-care docetaxel.
– In a Phase 2 study of Plinabulin + pembrolizumab + docetaxel in 2/3L NSCLC who progressed on PD-1/L1 inhibitors (n=47), median PFS was 6.8 months, and 15-month OS rate was 78%.

On July 7, 2025 Chugai Pharmaceutical Co., Ltd. (TOKYO: 4519, hereafter "Chugai"), and Gero PTE. LTD. (hereafter "Gero"), a Singapore-based biotechnology company, reported that they have entered into a joint research and license agreement to develop novel therapies for age-related diseases (Press release, Chugai, JUL 7, 2025, View Source [SID1234654258]).

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!

In this collaboration, Chugai will create novel antibody drug candidates using its proprietary antibody engineering technologies for new drug targets discovered by Gero through analysis of human datasets using their unique AI target discovery platform. Under this agreement, Gero grants Chugai exclusive worldwide rights for the creation, research, development, manufacturing, and commercialization of antibodies for the identified targets. In addition to an upfront payment, Chugai will potentially pay up to approximately 250 million USD in total if predetermined development or sales milestones are achieved. If Chugai successfully launches a product, it will also pay royalties on sales to Gero.

"We believe that open innovation with external partners, including leading global players, is extremely important for achieving global first-class drug discovery outlined in our growth strategy toward 2030, TOP I 2030. By combining Gero’s target discovery technology with Chugai’s drug discovery technologies, we will accelerate the creation of innovation," said Chugai’s President and CEO, Dr. Osamu Okuda.

"Our AI platform is built to identify therapeutic targets that drive multiple age-related diseases and potentially aging itself," said Peter Fedichev, CEO of Gero. "In this collaboration, we aim to translate those insights into therapeutics that can help restore the lost function. This partnership with Chugai is an important step toward achieving Gero’s mission: to meaningfully target the biological processes of human aging."

"We are excited to partner with Chugai, a leading pharmaceutical company, to unlock the synergy between human data-driven target discovery and cutting-edge therapeutic design technology platforms. Together, we aim to develop first-in-class therapeutics to address unmet needs of increasing number of patients suffering from age-related diseases," said Alex Kadet, CBO of Gero.