On September 25, 2026 AstraZeneca reported supplemental Biologics License Application (sBLA) for Imfinzi (durvalumab) in combination with enfortumab vedotin (EV) has been accepted and granted Priority Review in the US for the treatment of patients with muscle-invasive bladder cancer (MIBC) who are ineligible for or have declined cisplatin-based chemotherapy.

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The Food and Drug Administration (FDA) grants Priority Review to applications for medicines that, if approved, would offer significant improvements over available treatment options by demonstrating safety or efficacy improvements, preventing serious conditions or enhancing patient compliance.1 The Prescription Drug User Fee Act (PDUFA) date, the FDA action date for its regulatory decision, is anticipated during the fourth quarter of 2026.

Approximately one in four patients with bladder cancer has muscle-invasive disease, where the tumour invades the muscle wall of the bladder, without distant metastases.2,3 As many as 50% of patients are ineligible for cisplatin-based chemotherapy due to impaired renal function or comorbidities.4,5 The standard treatment for these patients has historically been radical cystectomy alone but, despite undergoing this major surgery, patients experience high rates of recurrence and have a poor prognosis.4-6

Susan Galbraith, Executive Vice President, Oncology Haematology R&D, AstraZeneca, said: "This Priority Review reinforces the potential of Imfinzi to become the immunotherapy backbone treatment for muscle-invasive bladder cancer, where patients face high rates of recurrence despite bladder removal surgery. If approved, this would be the first perioperative regimen with enfortumab vedotin given only before surgery; a potentially new standard of care offering practice-changing efficacy and tolerability in this curative-intent setting."

The sBLA is based on results from the VOLGA Phase III trial, which will be presented at a forthcoming medical meeting. In a planned interim analysis, perioperative treatment with Imfinzi in combination with neoadjuvant EV demonstrated statistically significant and clinically meaningful improvements in event-free survival (EFS) and overall survival (OS) versus radical cystectomy (surgery to remove the bladder) with or without approved adjuvant treatment.

The safety and tolerability of Imfinzi plus EV was consistent with the known safety profiles of the individual medicines, with no new safety signals identified.

Regulatory applications are currently under review in the EU, Japan and several other countries based on the results of the VOLGA trial.

Imfinzi is approved in over 50 countries for patients with cisplatin-eligible MIBC, based on the NIAGARA Phase III trial. Imfinzi in combination with Bacillus Calmette-Guérin (BCG) induction and maintenance therapy is approved in the US, Japan and other countries for patients with BCG-naïve, high-risk non-muscle-invasive bladder cancer, based on the POTOMAC Phase III trial. In July 2026, positive high-level results from the NILE Phase III trial showed Imfinzi plus chemotherapy demonstrated a statistically significant and clinically meaningful improvement in OS versus chemotherapy as 1st-line treatment for patients with PD-L1 high unresectable, locally advanced or metastatic urothelial cancer.

Notes

Bladder cancer
Bladder cancer is the 8th most common cancer in the world, with more than 635,000 cases diagnosed each year.7 The most common type is urothelial carcinoma, which begins in the urothelial cells of the urinary tract.8

In 2026, around 15,000 patients will be treated for MIBC in the United States.9 In 2025, the NIAGARA Phase III trial established a new standard of care by adding perioperative Imfinzi to neoadjuvant cisplatin-based chemotherapy and radical cystectomy.10 However, up to half of patients are not eligible to receive cisplatin, and approximately 50% of MIBC patients who undergo bladder removal surgery experience disease recurrence.4,6 New treatment options that prevent both progression before surgery and recurrence after surgery are critically needed in this curative-intent setting.

VOLGA
VOLGA is a Phase III, randomised, open-label, multi-centre global trial evaluating perioperative Imfinzi with or without Imjudo in combination with neoadjuvant EV as treatment for patients with MIBC undergoing radical cystectomy who are not eligible for or have declined cisplatin compared to radical cystectomy with or without approved adjuvant therapy. In the trial, 695 patients were randomised 1:1:1 to Arm 1 (three cycles of Imfinzi and EV, plus two cycles of Imjudo prior to surgery, followed by nine cycles of Imfinzi plus one cycle of Imjudo as adjuvant therapy), Arm 2 (three cycles of Imfinzi and EV prior to surgery, followed by nine cycles of Imfinzi adjuvant monotherapy) and Arm 3, the comparator arm.

The trial was conducted in 182 centres across 25 countries in Europe, North America, South America and Asia. Its dual primary endpoints are EFS, defined as the time from randomisation to first recurrence post-radical cystectomy, first progression in patients who did not undergo radical cystectomy, failure to undergo radical cystectomy in patients with residual disease or death due to any cause, for both experimental arms versus the comparator arm. Secondary endpoints include OS (Arm 1 vs. Arm 3 and Arm 2 vs. Arm 3), pathologic complete response, disease-free survival and pathologic downstaging across both experimental arms.

Imfinzi
Imfinzi (durvalumab) is a human monoclonal antibody that binds to the PD-L1 protein and blocks the interaction of PD-L1 with the PD-1 and CD80 proteins, countering the tumour’s immune-evading tactics and releasing the inhibition of immune responses.

In addition to its bladder cancer indications, Imfinzi is the global standard of care based on OS in the curative-intent setting of unresectable, Stage III non-small cell lung cancer (NSCLC) in patients whose disease has not progressed after chemoradiotherapy (CRT). Additionally, Imfinzi is approved as a perioperative treatment in combination with neoadjuvant chemotherapy in resectable NSCLC, and in combination with a short course of Imjudo (tremelimumab) and chemotherapy for the treatment of metastatic NSCLC. Imfinzi is also approved for limited-stage small cell lung cancer (SCLC) in patients whose disease has not progressed following concurrent platinum-based CRT; and in combination with chemotherapy (etoposide and either carboplatin or cisplatin) for the treatment of extensive-stage SCLC.

Imfinzi is also approved in combination with chemotherapy in locally advanced or metastatic biliary tract cancer and in combination with Imjudo in unresectable hepatocellular carcinoma (HCC). It is also approved as a monotherapy in unresectable HCC in Japan, China and the EU, and in resectable, early-stage and locally advanced gastric and gastroesophageal junction cancers in the US, EU and Japan. Additionally, in April 2026, Imfinzi in combination with Imjudo, lenvatinib and transarterial chemoembolisation (TACE) demonstrated a statistically significant and clinically meaningful improvement in the primary endpoint of PFS versus TACE alone for patients with unresectable HCC eligible for embolisation in the EMERALD-3 Phase III trial.

Imfinzi in combination with chemotherapy followed by Imfinzi monotherapy is approved as a 1st-line treatment for primary advanced or recurrent endometrial cancer (mismatch repair deficient disease only in US, EU and China). Imfinzi in combination with chemotherapy followed by Lynparza (olaparib) and Imfinzi is approved for patients with mismatch repair proficient advanced or recurrent endometrial cancer in EU and Japan.

Since the first approval in May 2017, more than 470,000 patients have been treated with Imfinzi. As part of a broad development programme, Imfinzi is being tested as a single treatment and in combinations with other anti-cancer treatments for patients with NSCLC, bladder cancer, breast cancer, ovarian cancer and several gastrointestinal cancers.

(Press release, AstraZeneca, SEP 25, 2026, View Source [SID1234671064])

On September 24, 2026 Primmune Therapeutics, a biotech company harnessing the power of the innate and adaptive immune system, reported the enrollment of the second patient in its Phase 2 INFLECTION-003 clinical study triggering the release of the second tranche of financing. The financing advances the development of Polvitolimod HCl (also known as PRTX007) as a curative therapy in the neoadjuvant and adjuvant settings for stage III melanoma.

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"Polvitolimod HCl is a novel oral TLR7/IRF7 agonist that induces robust systemic interferon responses and has demonstrated a favorable safety and tolerability profile in two Phase 1 studies" said Andrew Sharabi, MD, PhD, Chief Medical Officer of Primmune Therapeutics. "We have designed the Phase 2 INFLECTION-003 study to evaluate whether the combination of polvitolimod HCl and pembrolizumab before surgery will increase major pathologic response rates in patients with resectable stage III melanoma. We believe that priming the immune system with polvitolimod will enhance the clinical activity of pembrolizumab without a significant increase in toxicity, which would be a meaningful step forward for patients with this diagnosis."

INFLECTION-003 is a Phase 2, multi-center, single-arm study evaluating the safety, tolerability, and activity of neoadjuvant polvitolimod HCl in combination with pembrolizumab in participants with resectable stage III melanoma. Participants will receive oral polvitolimod HCl, a toll-like receptor 7 (TLR7) agonist prodrug, administered in combination with intravenous pembrolizumab for 9 weeks prior to surgical resection. The primary objective is to determine the major pathologic response (MPR) rate following neoadjuvant therapy. Secondary objectives include evaluation of safety, pathologic complete response (PCR) rate, event-free survival, overall survival, pharmacokinetics, and immune-related biomarkers. For more information, please visit ClinicalTrials.gov (NCT07565285).

"Polvitolimod HCl is the only oral TLR7/IRF7 specific agonist being developed for patients in the neoadjuvant solid tumor setting that we are aware of," said Charlie McDermott, Chief Executive Officer and Director of Primmune Therapeutics. "We believe there is a significant opportunity for safer and better tolerated immunotherapy combinations in early-stage solid tumor disease given the drug-related morbidity observed in clinical studies evaluating other immunotherapy combinations, such as nivolumab plus ipilimumab or pembrolizumab plus high-dose interferon."

About Polvitolimod HCl (PRTX007)
Polvitolimod HCl is a novel orally administered, systemically acting, toll-like receptor 7 (TLR7) agonist designed to functionally tune immune signaling toward an IRF7-driven poly-interferon response and away from the NF-B-mediated pro-inflammatory cytokine signaling that has limited the utility of systemically acting TLR7, TLR7/8, and TLR8 agonists. Polvitolimod HCl has been administered to 118 healthy human volunteers in two Phase 1 clinical studies (Study PRTX007-001 and Study PRTX007-002). In these studies, PRTX007 drove the desired systemic IRF7 poly-IFN response without undesired NF-B driven pro-inflammatory response. PRTX007 was generally well-tolerated in both Phase 1 studies with no serious adverse events reported.

(Press release, Primmune Therapeutics, SEP 24, 2026, View Source [SID1234673179])

On September 24, 2026, BioMarin Pharmaceutical Inc. (BioMarin) reported to have entered into a Settlement and License Agreement (the Agreement) with Ascendis Pharma A/S (Ascendis).

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The terms of the Agreement are consistent with those contained in the binding term sheet previously entered into by the parties, which was reported by BioMarin on a Current Report on Form 8-K filed with the Securities and Exchange Commission on August 31, 2026 (the Term Sheet). The Agreement supersedes and replaces the Term Sheet and is deemed effective as of August 28, 2026 (the Effective Date).

In accordance with the Agreement and consistent with the previously reported terms and conditions of the Term Sheet, BioMarin has granted to Ascendis and its affiliates a non-exclusive, worldwide, transferable, royalty-bearing license to certain patents (the BioMarin Patent Rights) to research, develop, manufacture, use, sell, and commercialize any product, method, or service that includes the C-type natriuretic peptide known as "TransCon CNP," "navepegritide," or "Yuviwel" (collectively, the Licensed Products) for all current and potential indications, including achondroplasia and hypochondroplasia, and in combination with other medicines. In consideration for the rights and licenses granted to Ascendis, Ascendis will pay to BioMarin a royalty equal to 20% of annual net sales of Licensed Products in the United States and 18% of annual net sales of Licensed Products in the European Union, Brazil and South Korea, in each case, from the date of the first commercial sale in each territory (applied retroactively, as applicable) until May 2030.

Pursuant to the Agreement, the parties will also dismiss with prejudice all pending litigation between them relating to the BioMarin Patent Rights.

The Agreement also contains a mutual regulatory non-interference clause, a covenant by Ascendis to not challenge BioMarin’s Patent Rights, a release by BioMarin of claims against Ascendis for infringement of BioMarin’s Patent Rights prior to the Effective Date and a covenant not to sue Ascendis for infringement of BioMarin’s Patent Rights, and certain other terms that are customary for transactions of this nature.

(Filing, BioMarin, SEP 24, 2026, View Source [SID1234671180])

On September 24, 2026 InnoCare Pharma (HKEX: 09969; SSE: 688428), a leading biopharmaceutical company focusing on the treatment of cancer and autoimmune diseases, reported that the Company has entered into a strategic research collaboration and license agreement with Eli Lilly and Company ("Lilly") to develop new medicines.

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InnoCare will leverage its proprietary drug discovery platform and extensive research experience to discover and advance compounds against up to five targets to address critical unmet medical needs.

"We are excited to leverage our R&D platform to collaborate with a global pharmaceutical leader like Lilly," said Dr. Jasmine Cui, the Co-founder, Chairwoman and CEO of InnoCare. "We are dedicated to expanding our partnership and innovation footprint."

Under the terms of the agreement, InnoCare will be eligible to receive up to $100 million in upfront and near-term payments, and up to approximately $3.25 billion in development and commercial milestone payments. In addition, InnoCare will be eligible to receive single-digit tiered royalties based on annual net product sales.

(Press release, InnoCare Pharma, SEP 24, 2026, View Source [SID1234671076])

On September 24, 2026 Biodesix, Inc. (Nasdaq: BDSX), a leading diagnostics solutions company, reported strategic progress in two collaborative programs for tumor-informed and tumor-naïve Molecular Residual Disease (MRD) solutions intended to support cancer centers and clinical laboratories. Both solutions detect circulating tumor DNA (ctDNA), the tumor DNA that a cancer sheds into the blood, to assess whether disease remains or is returning after treatment. The tumor-informed solution is the result of a multi-year collaboration with Bio-Rad Laboratories. The tumor-naïve solution is a newly launched program with Thermo Fisher Scientific that extends a collaboration for companion diagnostic validation and other development work. Both approaches will be validated by Biodesix in collaboration with key investigators at Memorial Sloan Kettering Cancer Center (MSK) and are designed to run on partner platforms already installed in laboratories around the world.

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"Our approach to MRD reflects a core conviction: sensitivity matters, and so does patient access. We are building ultra-sensitive assays with global collaborators whose platforms can carry those assays to laboratories and patients around the world," said Scott Hutton, Chief Executive Officer of Biodesix. "Offering both tumor-informed and tumor-naïve MRD means the clinical question can drive the testing strategy, rather than forcing a single approach in every situation. We believe this work positions Biodesix to participate in the growth of MRD testing well beyond the patients that can be reached through a centralized laboratory model."

No single MRD approach serves every patient. Tumor-informed testing is generally the most sensitive option when tumor tissue is available, and tumor-naïve testing extends monitoring to patients whose tissue is unavailable or insufficient. Biodesix is advancing both, drawing on its proprietary Diagnostic Cortex bioinformatics platform, its experience developing, validating, and commercializing diagnostic tests in its CAP-accredited, CLIA- and NYS CLEP-certified laboratory, and its track record in companion diagnostics test validation with global partners. The two programs build on MRD data that Biodesix and its collaborators have presented at scientific meetings over the past two years. Together they represent the first time Biodesix has outlined its MRD strategy as a whole and represents an expansion of the company’s focus beyond lung diagnostics into a rapidly growing area of precision oncology.

Tumor-informed MRD

For the tumor-informed solution, Biodesix profiles a patient’s tumor tissue using its proprietary Diagnostic Cortex bioinformatics platform to identify multiple complex genomic changes unique to that individual. Bio-Rad’s Droplet Digital PCR technology is then used to track those changes in blood over the course of the patient’s care, partitioning each sample into thousands of separate reactions so that individual tumor molecules can be counted. The ultra-sensitive informatics and analysis workflows detect ctDNA below 1 part per million (ppm) of the total DNA in a blood sample.

"Detecting molecular residual disease means finding a handful of molecules in a complex background, the precise application for which Droplet Digital PCR has an advantage over other technologies. Our multi-year work with Biodesix has focused on making that sensitivity practical in routine laboratories. We look forward to supporting broad access to these solutions and the positive impact they will have on patient lives," said Steve Kulisch, Vice President of Genomics Product Management, Bio-Rad Laboratories.

Tumor-naïve MRD

The tumor-naïve solution detects methylated ctDNA in blood using rapid next-generation sequencing on the Genexus platform from Thermo Fisher Scientific. Biodesix has begun developing and validating this assay in its CAP-accredited, CLIA- and NYS CLEP-certified laboratory as part of the Thermo Fisher Center of Excellence Network. Because the method reads methylation patterns rather than mutations unique to an individual tumor, it requires no tumor tissue. This means the test can potentially help patients who otherwise would not have been eligible for an MRD test because their archived tissue is unavailable or insufficient.

"Methylation carries a rich biological signal, and rapid analytic and informatics workflows make it possible to act on that signal in a clinically relevant timeframe," said Gary Pestano, Ph.D., Chief Scientific Officer of Biodesix. "By providing both tumor-informed and tumor-naïve approaches, Biodesix can reach more of the patients who may benefit from molecular monitoring throughout their cancer journey."

Upcoming presentations

Gary Pestano, Ph.D., Chief Scientific Officer of Biodesix, will join a spotlight panel on the future of MRD at the 5th Annual Roth Healthcare Opportunities Conference.

Panel Title: The Rise of MRD Monitoring in Solid Tumors and Where it Goes Next
Date and Time: Tuesday, September 29, 2026, 2:00 PM ET
Location: The Metropolitan Club, New York, NY
Panelists:

Gary Pestano, Ph.D., Chief Scientific Officer, Biodesix, Inc. (Nasdaq: BDSX)
Amal Thommil, MRD Monitoring Specialist, DeciBio
Pedram Razavi, M.D., Ph.D., Medical Oncologist, Director of Liquid Biopsy & Cancer Genomics, MSK

Data supporting both MRD programs will be presented at the AMP 2026 Annual Meeting in November 2026 in Seattle, with additional data planned for the San Antonio Breast Cancer Symposium in December 2026. The presentations will feature continued collaborative work with MSK, Bio-Rad Laboratories, and Thermo Fisher Scientific.

Notes and Disclosures:

Memorial Sloan Kettering (MSK) has institutional financial interests related to Biodesix, Inc.

Droplet Digital PCR is a trademark of Bio-Rad Laboratories, Inc.

Genexus is a trademark of Thermo Fisher Scientific.

Nodify Lung, IQLung, and Diagnostic Cortex are registered trademarks of Biodesix, Inc.

(Press release, Biodesix, SEP 24, 2026, View Source [SID1234671075])