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Avacta Group plc: The FOCUS-01 Clinical Trial Design and Preclinical Data Further Demonstrate the Potential of AVA6103 at AACR Conference on Pancreatic Cancer

On September 25, 2026 Avacta Therapeutics (AIM: AVCT, the "Company", "Avacta"), a life sciences company developing innovative, targeted oncology drugs, reported the presentation of the FOCUS-01 trial design and preclinical data highlighting the potential of AVA6103 (FAP-Exd) in pancreatic ductal adenocarcinoma (PDAC). FOCUS-01 is the Phase 1 trial of AVA6103, the Company’s first Next-Generation pre|CISION peptide-drug conjugate (PDC) and is being presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) ("AACR") Conference on Pancreatic Cancer in San Diego, California, held between 25-28 September 2026.

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The presentation highlights the design of the FOCUS-01 Phase 1 study of AVA6103 and updated preclinical data supporting the assessment of AVA6103 in patients with PDAC. AVA6103 is designed to significantly increase the therapeutic window of the highly potent payload, exatecan, and the trial implements a dose-dense arm, dosing every two weeks (Q2W), to enhance the delivery of payload to the tumor over traditional dosing of ADCs at every three weeks (Q3W). Preclinical studies using the dose-dense regimen in patient-derived xenograft (PDX) mouse models of PDAC demonstrate that AVA6103 achieved tumor-targeted delivery of exatecan with highly durable complete and partial responses in multiple models observed for weeks following cessation of dosing.

Additionally, researchers observed high FAP expression in PDAC with close proximity of FAP-expressing cancer-associated fibroblasts (CAFs) to both blood vessels and tumor cells, shown via multiplex immunofluorescence (mIF). These findings support the mechanism of action of the Next Gen pre|CISION medicines: efficient PDC delivery to the tumor, cleavage and release of payload with tumor cell uptake of released payload.

Christina Coughlin, CEO of Avacta, commented:

"The preclinical data presented at AACR (Free AACR Whitepaper) underscore the significant potential of AVA6103 in addressing the challenges of pancreatic cancer, a disease with limited treatment options and poor prognosis. The robust preclinical efficacy, combined with the high FAP expression in PDAC, further strengthens our confidence in AVA6103 as a promising therapeutic candidate to improve treatment options for patients.

"This adds to the momentum of AVA6103 and highlights the potential of our unique pre|CISION technology to bring hope to patients battling PDAC and other aggressive cancers. The first clinical data from the FOCUS-01 trial, providing clinical evidence consistent with tumor-specific delivery of exatecan by AVA6103, have further reinforced our confidence in this program and we are now moving towards an initial efficacy readout from the study in H1 2027."

FOCUS-01: Trial in Progress

The FOCUS-01 Phase 1a trial is a first in-human, multicenter dose escalation trial of AVA6103 and is enrolling patients with select tumor types predicted to be sensitive to the payload exatecan, including PDAC. Patients with PDAC are enrolled in the Q2W dosing arm, a dose-intense regimen designed to match standard of care in the indication. The ability to deliver dose intensity and flexible administration is unique to pre|CISION Next-Gen PDCs because:

In contrast to antibodies, the pre|CISION peptide does not accumulate, enabling more frequent dosing when appropriate; and

The pre|CISION Next-Gen mechanism retains and cleaves the conjugate inside the tumor, significantly limiting systemic exposure to the payload and enabling the interval between doses to be shortened, allowing delivery of higher dose intensity compared to ADCs.
Preliminary safety and pharmacokinetic (PK) data from the first three dose levels of the FOCUS-01 trial, reported earlier this month, demonstrated proof of mechanism in patients treated in the first three cohorts in the trial. AVA6103 demonstrated a favorable safety profile and was well tolerated through the first three dose levels up to a payload dose approximately 50% higher than the maximum tolerated dose (MTD) of conventional exatecan.

The pharmacokinetic data of the PDC, released peptide and released exatecan patients were remarkably consistent with predictive PK modelling based on preclinical data, greatly increasing confidence that the safety profile, tumor selectivity and antitumor efficacy observed in the preclinical studies of AVA6103 will translate into the clinic.

Enrollment is ongoing in both arms of the study with patients treated at dose level 4, which represents an absolute dose of payload that is more than double the MTD of conventional exatecan and approaches the equivalent topoisomerase I inhibitor payload dose of the approved dose of Enhertu in breast cancer.

Details of the poster presentation

Title: A Phase I Trial of FAP-Exd (AVA6103), a Fibroblast Activation Protein (FAP)-enabled pre|CISION Peptide-drug Conjugate Delivering Sustained Tumor Microenvironment (TME) Release of Exatecan in Patients with FAP-positive Solid Tumors

First Author: Alex Spira, MD

Session: New Frontiers in Biology and Therapeutic Development

Date: September 26, 2026

Enhertu (trastuzumab deruxtecan; T-DXd) is a protease cleavable-linker ADC, approved for both breast cancer and gastric cancer indications (an AstraZeneca/Daiichi Sankyo product). Enhertu is a registered trademark of Daiichi Sankyo Company, Limited and AstraZeneca.

(Press release, Avacta Life Sciences, SEP 25, 2026, View Source [SID1234671065])

Perioperative Imfinzi plus neoadjuvant enfortumab vedotin granted Priority Review in the US for patients with muscle-invasive bladder cancer

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.

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])

InnoCare Announces Strategic Research Collaboration and License Agreement with Lilly to Develop New Medicines

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.

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])

Biodesix Advances Two Complementary Approaches to Molecular Residual Disease (MRD) Monitoring

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.

"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])

Ligand and AvenCell Therapeutics Enter Financing Agreement to Advance Pipeline of Next-Generation CAR-T Therapies for Up to $47 Million

On September 24, 2026 Ligand Pharmaceuticals Incorporated (Nasdaq: LGND) reported that it has entered into a financing agreement with AvenCell Therapeutics, Inc., a clinical-stage cell therapy company developing controllable, allogeneic CAR-T therapies for patients with cancer, for up to $47 million. The investment will help advance AvenCell’s pipeline, including AVC-201 for the treatment of relapsed/refractory acute myeloid leukemia (AML) and AVC-203 for the treatment of B-cell malignancies.

Under the terms of the agreement, Ligand has committed up to $41 million in exchange for a mid single-digit to low double-digit royalty on worldwide annual net sales of all current and future AvenCell pipeline assets, including AVC-201 and AVC-203, with the applicable rate determined based on the total amount ultimately funded. The capital will be funded in four tranches: with the first payable at closing, and the remaining three tranches payable upon achievement of certain predetermined clinical milestones and other specified financing conditions. Ligand has also committed up to $6 million in concomitant Series C financing, details of which will be announced separately.

"AvenCell has built a differentiated cell therapy platform that brings together CRISPR-engineered allogeneic CAR-T technology with a unique switchable CAR approach designed to provide greater control over CAR-T activity," said Todd Davis, CEO of Ligand. "We believe the combination of these technologies, together with the encouraging clinical data generated to date with AVC-201, highlights the potential of AvenCell’s platform across a broad range of diseases. We look forward to working closely with the AvenCell team as it advances its pipeline of next-generation cell therapies."

AvenCell was founded in 2021 combining switchable CAR-T technology developed by GEMoaB GmbH (now AvenCell Europe GmbH) with Intellia’s CRISPR/Cas9-based Allogeneic Engineering Technology to develop next-generation cell therapies designed to overcome key limitations of existing CAR-T treatments. AvenCell’s proprietary platform is designed to enable readily available, "off-the-shelf" cell therapies with greater control over CAR-T activity and the potential for broad application across hematologic malignancies and autoimmune diseases.

The company’s lead program, AVC-201, is an anti-CD123 CAR-T currently in a Ph1b expansion trial for the treatment of relapsed or refractory AML. AvenCell is also advancing AVC-203, a Phase 1a program for B-cell malignancies.

"We are excited to have the support and expertise of the Ligand team as we look to advance our pipeline programs through the clinic," said Andrew Schiermeier, President & CEO of AvenCell. "This investment provides us critical resources to support the continued development of these potentially important new treatment options for patients impacted by these difficult-to-treat cancers."

Hogan Lovells Cadwalader served as legal advisor to Ligand.

(Press release, Ligand, SEP 24, 2026, View Source [SID1234671074])