On September 11, 2025 Ferring Pharmaceuticals Co., Ltd. reported that the PMDA has accepted the NDA for nadofaragene firadenovec for review, following submission on August 27th, 2025 (Press release, Ferring, SEP 11, 2025, View Source [SID1234655932]). This non-replicating gene therapy, administered intravesically, offers patients with NMIBC a bladder-sparing treatment option. Nadofaragene firadenovec’s quarterly dosing eliminates the burden of frequent treatments, while delivering a non-chemotherapy mechanism of action through interferon gene therapy. The NDA acceptance further highlights Ferring’s ongoing commitment to establish the new standard of care for high-risk BCG- unresponsive NMIBC.

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Professor Keiji Inoue, M.D., Ph.D., Department of Urology, Kochi Medical School, stated: "Nadofaragene firadenovec represents an option for those who failed NMIBC treatment. As the first choice after BCG failure, this bladder sparing gene therapy offers patients a non-chemotherapy option that transforms their own bladder cells into interferon-producing factories. The 75% complete response rate achieved with convenient quarterly dosing provides hope for patients who previously faced limited treatment options."

The NDA for nadofaragene firadenovec is based on results from a Phase 3 trial conducted in Japan.1 The primary outcome of a complete response (CR) rate of 75% at 3 months, following a single dose in 20 high-risk Japanese patients with carcinoma in situ (CIS), with or without concomitant high-grade Ta or T1 papillary lesions, was presented at the 112th Annual Meeting of the Japanese Urological Association (JUA, April 17-19, 2025, Fukuoka). All treatment-related adverse events were limited to Grade 1 (84.2%) or Grade 2 (15.8%), with zero Grade 3, 4, or 5 adverse events reported – confirming the therapy’s favourable safety and tolerability profile.1

These results are consistent with independent US real-world data presented by the Mayo Clinic, which demonstrated 79% complete response rate.2 The results from the Mayo Clinic were achieved following a single quarterly dosing, representing clinical and convenience advantages over existing therapies, without requiring re-induction protocols.2 Patients who achieved complete response continued quarterly maintenance dosing.

Joern Jakobsen, M.D., Ph.D., Vice President and Head of Global Research and Medical for Uro-Oncology and Urology, Ferring Pharmaceuticals stated "Traditionally, when BCG therapy proved insufficient, patients had no choice but to undergo highly invasive treatments such as radical cystectomy or risk cancer progression. Nadofaragene firadenovec is expected to offer a new bladder-sparing treatment option. At Ferring, we are committed to addressing the unmet needs in bladder cancer treatment by providing urologists with critical insights that enable effective, personalized, and groundbreaking therapies."

"Our ambition is to establish nadofaragene firadenovec as the new standard of care and the backbone therapy for NMIBC treatment," said Bipin Dalmia, Global Head, Uro-Oncology & Urology Franchise. "High-risk NMIBC patients who no longer respond to BCG have endured decades of little progress and currently face bladder removal as their primary option. This PMDA acceptance validates our strategic commitment to bring this treatment to Japanese patients."

About nadofaragene firadenovec
Nadofaragene firadenovec represents the first and only FDA-approved intravesical non-replicating gene therapy for adult patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumours.

As the first choice therapy following BCG failure, this non-chemotherapy approach utilises a non-replicating adenovirus vector-based therapy containing the interferon alfa-2b gene. Administered locally as convenient monotherapy by catheter directly into the bladder once every three months only, the mechanism transforms bladder wall cells into interferon microfactories, creating high and transient local expression of interferon alfa-2b protein – amplifying the body’s natural cancer-fighting capabilities through gene therapy rather than traditional chemotherapy approaches.

This therapeutic approach has been investigated through comprehensive clinical evaluation including 157 patients in the US with high-risk BCG-unresponsive NMIBC who demonstrated inadequate BCG response (full inclusion criteria published on clinicaltrials.gov: NCT02773849). 5-year follow-up data confirmed 80% overall survival rate and 49% cystectomy-free survival rate in adult patients with high-risk BCG-unresponsive NMIBC with CIS with or without papillary tumours (±Ta/T1), and in patients with high-grade Ta/T1 without CIS. Long-term safety profile maintained with most treatment emergent AEs remaining transient Grade 1 or 2 (66% of all patients studied), and 4% experiencing Grade 3 AEs3.

In the real world setting, between November 2023 and October 2024, 45 patients across three Mayo Clinic locations in the US were treated with nadofaragene firadenovec for BCG-unresponsive NMIBC. Out of 45 treated patients, 29 were included in the analysis.2 After a median follow-up of 8.2 months: 72% of patients showed a complete response or were free from high-grade recurrence at 3 months. 62% maintained this response at 6 months. 94% avoided bladder removal surgery (cystectomy). 100% were still alive at 6 months.

About Non-Muscle Invasive Bladder Cancer (NMIBC)
NMIBC affects the superficial bladder layer without deeper invasion or metastatic spread. Bladder cancer represents a major clinical challenge as the 13th most commonly diagnosed cancer in Japan and ninth globally,4 with 75% presenting as NMIBC in 2022.4 While intravesical BCG remains first-line standard care for high-risk NMIBC, over 50% of patients experience disease recurrence and progression within one year, with many developing BCG-unresponsive disease requiring radical intervention.5

Current treatment options remain limited for BCG-unresponsive patients, with JUA guidelines recommending only radical cystectomy or clinical trial participation6. Nadofaragene firadenovec offers the first choice non-chemotherapy gene therapy option for these patients, providing an alternative to immediate radical surgery.

On September 11, 2025 Eli Lilly and Company (NYSE: LLY) reported it will participate in the Bernstein’s 2nd Annual Healthcare Forum on September 25, 2025. Lucas Montarce, executive vice president and chief financial officer, will take part in a fireside chat at 8 a.m., Eastern time (Press release, Eli Lilly, SEP 11, 2025, View Source [SID1234655931]).

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A live audio webcast will be available on the "Webcasts & Presentations" section of Lilly’s investor website at View Source A replay of the presentation will be available on this same website for approximately 90 days.

On September 10, 2025 Mednovo Group Co., Ltd. ("Mednovo") reported that the first patient has been successfully dosed in its Phase III clinical trial of Lutetium [177Lu] Oxodotreotide Injection, a Class 3 targeted radiotherapeutic drug (Press release, Mednovo, SEP 10, 2025, View Source [SID1234655935]).

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As the exclusive clinical supply and manufacturing partner, C-Ray Therapeutics delivered end-to-end support for this milestone, including technology transfer, clinical batch production, quality release, and logistics. Leveraging China’s first fully automated radiopharmaceutical production line built to global cGMP standards, C-Ray ensured the trial began on schedule. The company’s philosophy—high quality, cost efficiency, and first-time-right production—underpinned its execution.

Lutetium [177Lu] Oxodotreotide Injection targets somatostatin receptors and is primarily indicated for the treatment of gastroenteropancreatic neuroendocrine tumors (GEP-NETs). Entering Phase III represents a pivotal step for the program, requiring production processes and quality controls that meet near-commercial standards. C-Ray’s cGMP manufacturing capabilities and globally compliant quality system provided a solid foundation for reliable clinical supply.

Since the project was accepted by China’s Center for Drug Evaluation (CDE), C-Ray has worked closely with Mednovo’s clinical schedule, efficiently completing raw material release, technology transfer, production, testing, and nationwide logistics to ensure timely first-patient dosing.

Looking ahead, C-Ray will continue supporting Mednovo’s multi-center trial with its patented, internationally compliant cold-chain packaging for radiopharmaceuticals and its integrated land-air logistics network.

A Mednovo representative commented that C-Ray Therapeutics is a vital partner for their Lutetium [177Lu] Oxodotreotide Injection program. Their professional expertise, efficiency, and cGMP-compliant automated production line ensured the stable, high-quality supply of clinical materials for this Phase III trial. They look forward to advancing this program together.

To date, C-Ray has successfully supported more than 50 CRDMO (Contract Research, Development, and Manufacturing Organization) projects across the full lifecycle—from early-stage R&D to clinical and commercial supply. With a 28,000 m² cGMP-compliant R&D and manufacturing base and a global radionuclide supply chain, C-Ray continues to deliver high-quality, efficient, and fully compliant solutions for pharmaceutical partners worldwide.

On September 10, 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 it has concluded dosing in the first cohort of its Phase 1b clinical trial of PTM-101 in pancreatic ductal adenocarcinoma (PDAC) (Press release, PanTher Therapeutics, SEP 10, 2025, View Source [SID1234655930]). The rapid completion of this middle-dose cohort reflects vigorous engagement of the clinical oncology community with this new approach to cancer treatment.

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The ongoing dose escalation and expansion study builds on findings from PanTher’s first-in-human trial that confirmed the safety of PTM-101 at 100 mg and reported promising tumor shrinkage. In the current U.S. Phase 1b trial, patients are being treated at escalating dose levels of 200 mg and 400 mg. Completion of the full cohort at the 200 mg dose level without evidence of toxicities further demonstrates PTM-101’s safety — even at a dose higher than what is typically used for IV infusion — and confirms its ability to be administered with well-established laparoscopic procedures. The Safety Committee has cleared advancement to the final anticipated cohort, and dosing at the 400 mg dose level has now begun, with the first patient treated.

"The swift execution of this first stage of our Phase 1b study underscores the momentum of our clinical strategy and the promise of PTM-101 as a new treatment paradigm for pancreatic cancer," said Laura Indolfi, Ph.D., Chief Executive Officer and Co-founder of PanTher Therapeutics. "We are encouraged by the good safety profile observed so far and excited by the strong engagement from our clinical investigators. Medical and surgical oncologists are eager for better treatment options for their patients and have found that PTM-101 fits seamlessly into their existing neoadjuvant clinical treatments, creating an opportunity to reach patients at a critical point early in their journey."

Intravenous chemotherapy is highly inefficient at permeating solid tumors and can cause debilitating side effects. PTM-101 is an investigational thin film formulation of the chemotherapy drug paclitaxel that is designed to overcome these limitations. It delivers a sustained (~6 weeks) high dose directly to the tumor site with little to no systemic exposure.

"From a surgical perspective, PTM-101 has been straightforward to place and has integrated seamlessly into minimally invasive staging laparoscopy and mediport placement. I favor a robotic-assisted approach, which allows for enhanced handling of the delicate tissue around the pancreas," said principal investigator Danielle DePeralta, M.D., surgical director of the Pancreas Multidisciplinary Program at the Northwell Health Cancer Institute, Lake Success, NY. "Importantly, patch placement is incorporated into procedures that the patient would otherwise need prior to initiating standard systemic chemotherapy. My hope is that targeted local treatment with the patch, paired with standard chemotherapy, will make more patients with isolated pancreatic tumors candidates for surgical removal with the goal of long-term survival and cure. Because of the proximity of pancreatic tumors to major blood vessels, even minimal tumor shrinkage can make a big difference."

The dose escalation and dose expansion Phase 1b study (NCT06673017) is assessing safety, tolerability, and anti-tumor activity of PTM-101 when combined with standard of care neoadjuvant chemotherapy (FOLFIRINOX) in subjects with borderline resectable or locally advanced PDAC. Dosing of patients has taken place at three clinical sites in the United States, with The University of Texas MD Anderson Cancer Center in Houston, Texas, recently joining the list of sites participating in the trial.

PTM-101 is the lead product candidate within PanTher’s pipeline of implantable medicines designed to directly address hard-to-treat solid tumors. 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 investigational 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 September 10, 2025 Aktis Oncology, Inc., a clinical-stage oncology company focused on unlocking the breakthrough potential of targeted radiopharmaceuticals for patient populations not addressed by existing platform technologies, reported that Matthew Roden, PhD, President and Chief Executive Officer, and other members of the Aktis Oncology leadership team, will participate in the following September investor conferences (Press release, Aktis Oncology, SEP 10, 2025, View Source [SID1234655929]):

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Oppenheimer 3rd Annual Targeted Radiopharmaceutical Therapies in Oncology Summit

Date:

Thursday, September 11, 2025

Location:

New York, N.Y.

Bank of America Healthcare Trailblazers Private Company Conference

Date:

Wednesday, September 17, 2025

Location:

Everett, Mass.