On September 16, 2026 Avacta Therapeutics (AIM: AVCT), a life sciences company developing innovative, targeted oncology drugs, reported that it has achieved clinical proof of mechanism for AVA6103, its next-generation controlled-release pre|CISION peptide-drug conjugate (PDC) platform in the ongoing phase 1 FOCUS-01 trial.

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Preliminary data from the Phase 1a trial of AVA6103 in patients with select solid tumors demonstrate proof of mechanism with two key findings:

AVA6103 pre|CISION controlled-release exatecan demonstrates a clean safety profile through the first three dose levels, including a payload dose level 50% higher than the maximum tolerated dose (MTD) of conventional exatecan, and
The comparison of the preclinical modeled pharmacokinetic (PK) data and clinical trial PK data demonstrates an exceptional alignment through the first 3 dose levels with controlled release of exatecan evident in patients for days after dosing
The alignment of these critical datasets greatly increases our confidence that the safety profile, tumor selectivity and antitumor efficacy observed in the preclinical studies of AVA6103 will translate into the clinic.

The first head-to-head comparison of AVA6103 and Enhertu, a marketed antibody drug conjugate (ADC) that targets HER2, demonstrates that AVA6103 shows better antitumor activity vs. Enhertu, with deep and durable responses delivered by our dose dense regimen that has been applied in the FOCUS-01 trial.

Christina Coughlin, CEO of Avacta, commented:

"We are thrilled to report the proof of mechanism data with our first Next-Generation pre|CISION molecule in the clinic, which continues to underscore the potential of our platform to make a significant difference to cancer patients. AVA6103 moved from candidate status to Investigational New Drug application in less than a year, and has now reached an initial clinical readout with excellent safety and PK data, showing it is performing exactly as expected from the data generated in our preclinical studies.

"Our head-to-head comparison with the marketed ADC Enhertu shows better activity in a HER2+ preclinical model even at low FAP levels, with the dose-dense regimen demonstrating advantages of AVA6103 over traditional dosing of ADCs.

"We continue our discussions with potential partners on the Next Gen assets and these data greatly increase our partnering position across the Next Gen platform. The FOCUS-01 trial continues to enroll patients into two parallel arms and we look forward to providing further updates as the trial progresses.

"With FAP expression in ~90% of solid tumors, these clinical findings provide a gateway for Avacta to link multiple payloads and access previously unaddressable markets. This is a major inflection point for our Next Gen pre|CISION platform, providing the opportunity for pipeline expansion to benefit patients and shareholders alike"

Clinical Proof of Mechanism of AVA6103 in the FOCUS-01 Trial

Trial design and progress:

The FOCUS-01 Phase 1 clinical trial of AVA6103 is enrolling patients with locally advanced or metastatic disease with one of six indications, being: colorectal cancer, pancreatic ductal adenocarcinoma, gastric/gastroesophageal junction cancers, cervical cancer or small cell lung cancer.

The first three dose levels have completed enrollment of patients (n=19) in two parallel arms with doses administered every two weeks (Q2W) or every three weeks (Q3W) and assessed for safety and tolerability, PK / pharmacodynamics (PD) and preliminary efficacy. The first three dose levels in the AVA6103 trial were 1.5 mg/m2, 3 mg/m2 and 4.5 mg/m2 to enable the comparison to both the Phase 1 trial of Enhertu and conventional exatecan:
The first three dose levels are similar to the absolute payload dose levels in the published Enhertu Phase 1 trial (Doi, et al. 2017);
Dose level 2 of AVA6103 is similar to the MTD of conventional exatecan (Rowinsky, et al. 2000); and
Dose level 3 represents an approximate 50% increase over this MTD of conventional exatecan.
Safety, tolerability and PK data in the first three dose levels are presented. Screening is ongoing for enrollment of patients into both the Q2W and Q3W arms at dose level 4.

Safety data

Patients in the FOCUS-01 trial were treated with escalating doses (1.5 mg/m2 to 4.5 mg/m2) and assessed for safety and tolerability. These data were compared with published data for Enhertu (releasing a highly similar payload, deruxtecan) and with conventional exatecan administered in the standard regimen (once daily for five days).

The safety profile of AVA6103-derived exatecan is highly favorable at the first three dose levels when compared with equivalent dosing of topoisomerase I inhibitors (Topo Ii) (conventional exatecan and Enhertu-derived deruxtecan), with minimal toxicity reported with AVA6103.
Notably, AVA6103 produced little toxicity at the equivalent and escalated doses of the MTD of conventional exatecan in heavily pretreated patients (Rowinsky et al. 2000) and at similar payload doses in the first three cohorts in the Phase 1 of Enhertu (Doi et al. 2017). Relevant comparison data include:
Neutropenia: 0% with AVA6103, 22% with similar doses of Enhertu, and 64% at the MTD of conventional exatecan (equivalent to the 3 mg/m2 dose of AVA6103).
Thrombocytopenia: 5% (1/19) with AVA6103, 11% with Enhertu and 43% with conventional exatecan;
Anemia: 16% (3/19) with AVA6103, 11% with similar doses of Enhertu and 43% with conventional exatecan; and
Nausea and vomiting: 5% (1/19) with AVA6103, 44% with Enhertu and 67% with exatecan (reported across all dose levels in this trial).
Pharmacokinetic data

Patients were assessed for plasma PK properties across the first three dose levels of AVA6103 and compared to the corresponding dose levels of Enhertu.

As seen previously in preclinical models, the first three dose levels in the trial demonstrate rapid reduction in the plasma level of the PDC (AVA6103), with prolonged low-level release of both products of the cleavage reaction, the pre|CISION peptide and released exatecan.
Given the very short half-life of the released peptide (2-3 hours) that was demonstrated in the AVA6000 program, and the detection of low levels of the released peptide from AVA6103 in the plasma up to 48 hours after dosing indicates that the PDC is being retained in the tumor in a ‘drug reservoir’ that is slowly being cleaved to release the peptide and exatecan, as this mechanism was designed to do.
The remarkable consistency of these results with predictive PK modeling based on preclinical studies greatly increase our confidence that AVA6103 is performing as intended, and that the robust preclinical data will translate to the clinic and support ongoing dose-escalation and efficacy evaluation in the Phase 1 trial.
Preclinical efficacy comparison with Enhertu

The comparative efficacy of Enhertu and AVA6103 was tested in a head-to-head format using a gastric cancer patient-derived xenograft model that is HER2+ and FAP+ (by IHC).
When tumors had grown to 100-200 mm3 in size, animals were randomized to receive either AVA6103 or Enhertu at the preclinical dose with evidence of activity (Enhertu x 1 or AVA6103 QWx3 dose dense regimen).
Enhertu treatment resulted in a slowed growth compared to vehicle control, with two animals demonstrating small tumor reductions and 1/6 with progression as best response. In comparison, AVA6103 treatment resulted in deep and prolonged partial responses in 6/6 animals treated.
Outlook

The design of the FOCUS-1 trial of AVA6013 and preclinical updates will be presented in Trials in Progress presentations at both the American Association of Cancer Research (AACR) (Free AACR Whitepaper) Conference on Pancreatic Cancer, being held on September 25-28, 2026, and the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress, being held on October 23-27, 2026.
First efficacy data from the FOCUS-01 trial with AVA6103 is anticipated to be presented in H1 2027 – including data from clinical tumor biopsies which are anticipated to confirm AVA6103 is being retained in a ‘drug reservoir’ in the tumor, based on the preliminary Phase 1 data.
The selection of the payloads and data to support clinical candidate selection for the Dual Payload Next Gen Program (AVA6207) will be presented in Q4 2026.
Clinical data from the First Gen faridoxorubicin (AVA6000) program will also be presented in Q4 2026 at the ESMO (Free ESMO Whitepaper) Congress.
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 16, 2026, View Source [SID1234670876])

On September 16, 2026 Daiichi Sankyo reported that Enhertu (trastuzumab deruxtecan) and Datroway (datopotamab deruxtecan) have been approved in Japan for two new indications in the first-line setting of patients with metastatic breast cancer.

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Enhertu in combination with pertuzumab was approved for the treatment of adult patients with HER2 positive unresectable or recurrent breast cancer. Datroway was approved for the treatment of adult patients with hormone receptor (HR) negative and HER2 negative unresectable or recurrent breast cancer, a subtype commonly referred to as triple negative breast cancer (TNBC).

Enhertu and Datroway are specifically engineered DXd antibody drug conjugates (ADC) discovered by Daiichi Sankyo (TSE: 4568) and being developed and commercialized by Daiichi Sankyo in Japan.

Breast cancer is the most common cancer among women in Japan. 1 Both HER2 positive and TNBC are aggressive breast cancer subtypes, with only about 30% and 15% of patients, respectively, living five years following diagnosis.2,3 For patients with these aggressive subtypes, treatments that significantly delay disease progression in the first-line metastatic setting are needed to improve long-term outcomes.

"These two approvals represent significant advances for the treatment of metastatic breast cancer. Enhertu in combination with pertuzumab is the first new treatment regimen in more than a decade for patients with metastatic HER2 positive disease and Datroway is the only TROP2 directed medicine to demonstrate an overall survival benefit for patients with metastatic triple negative breast cancer in the first-line setting," said Yuki Abe, PhD, Senior Executive Officer, Head of R&D Division in Japan and Head of Research, Daiichi Sankyo. "With these new approvals of Enhertu and Datroway, there are now two Daiichi Sankyo medicines available in Japan that can be used as first-line treatments across the most aggressive subtypes of metastatic breast cancer."

Data Supporting Enhertu Approval

The approval of Enhertu by Japan’s Ministry of Health, Labour and Welfare (MHLW) is based on results from the DESTINY-Breast09 phase 3 trial. In the trial, Enhertu in combination with pertuzumab reduced the risk of disease progression or death by 44% versus taxane, trastuzumab and pertuzumab (THP) (hazard ratio [HR]=0.56; 95% confidence interval [CI]: 0.44-0.71; p<0.00001) in patients (n=383) with HER2 positive metastatic breast cancer who had not received prior chemotherapy or HER2 targeted therapy or had received neoadjuvant or adjuvant HER2 targeted therapy more than six months before the diagnosis of advanced or metastatic disease. Median progression-free survival (PFS) was 40.7 months (95% CI: 36.5-not estimable [NE]) with Enhertu in combination with pertuzumab compared to 26.9 months (95% CI: 21.8-NE) with THP as assessed by blinded independent central review (BICR). These data were presented at the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting and subsequently published in The New England Journal of Medicine.

In DESTINY-Breast09, the safety profile of Enhertu was consistent with previous clinical trials with no new safety concerns identified. Adverse reactions occurred in 373 patients (97.9%) treated with Enhertu (5.4 mg/kg) in combination with pertuzumab including 39 Japanese patients. The most common adverse reactions were nausea (71.1%), diarrhea (55.9%), alopecia (46.2%), vomiting (42.0%) and anemia (34.9%). Interstitial lung disease (ILD) occurred in 33.3% of Japanese patients treated with Enhertu in combination with pertuzumab as determined by an independent ILD adjudication committee.

Data Supporting Datroway Approval

The approval of Datroway by Japan’s MHLW is based on results from the TROPION-Breast02 phase 3 trial, which included patients with metastatic TNBC who experienced early relapse following prior treatment and were not candidates for PD-1/PD-L1 inhibitor therapy. In this trial, Datroway demonstrated a statistically significant and clinically meaningful 5.0 month improvement in median overall survival (OS) versus investigator’s choice of chemotherapy (HR=0.79; 95% CI: 0.64-0.98; p=0.029). Median OS was 23.7 months for patients treated with Datroway versus 18.7 months for those treated with chemotherapy. Datroway reduced the risk of disease progression or death by 43% compared to chemotherapy (HR=0.57; 95% CI: 0.47-0.69; p<0.0001) as assessed by BICR. Median PFS was 10.8 months for patients treated with Datroway versus 5.6 months for those treated with chemotherapy. These data were presented at the 2025 European Society for Medical Oncology Congress and subsequently published in Annals of Oncology.

In TROPION-Breast02, the safety profile of Datroway was consistent with previous clinical trials with no new safety concerns identified. Adverse reactions occurred in 296 patients (92.8%) treated with Datroway (6 mg/kg) including 17 Japanese patients. The most common adverse reactions included stomatitis (57.1%), nausea (44.5%), alopecia (40.8%), dry eye (23.8%) and constipation (22.6%). ILD was not observed in the 17 Japanese patients treated with Datroway.

Both Enhertu and Datroway are approved in Japan with a Warning in their prescribing information for ILD. ILD occurred in 11.7% of patients treated with Enhertu and 3.1% of patients treated with Datroway across multiple clinical trials. As cases of ILD, including fatal cases, have occurred in Enhertu and Datroway treated patients, both medicines are to be used in close collaboration with a respiratory disease expert. Patients should be closely observed during therapy by monitoring for early signs or symptoms of ILD (such as dyspnea, cough or fever) and performing periodical percutaneous oxygen saturation (SpO2) tests, chest X-ray scans and chest CT scans. If abnormalities are observed, discontinue administration of Enhertu or Datroway and take appropriate measures, such as corticosteroid administration. Prior to initiation of Enhertu or Datroway therapy, a chest CT scan should be performed and medical history taken to confirm the absence of any comorbidity or history of ILD with the patient and carefully consider the eligibility of the patient for Enhertu or Datroway therapy.

About DESTINY-Breast09

DESTINY-Breast09 is a global, multicenter, randomized, open-label, phase 3 trial evaluating the efficacy and safety of Enhertu (5.4 mg/kg) either alone or in combination with pertuzumab versus standard of care THP as firstline treatment in patients with HER2 positive metastatic breast cancer.

Patients were randomized 1:1:1 to receive either Enhertu monotherapy with a pertuzumab matching placebo; Enhertu in combination with pertuzumab; or THP. Randomization was stratified by prior treatment (de novo metastatic disease versus progression from early-stage disease), HR status and PIK3CA mutation status.

The primary endpoint of DESTINY-Breast09 is PFS as assessed by BICR in the Enhertu monotherapy and Enhertu combination arms. Secondary endpoints include investigator-assessed PFS, OS, overall response rate (ORR), duration of response (DoR), pharmacokinetics and safety. The investigational arm assessing Enhertu monotherapy versus THP remains blinded to patients and investigators and will continue to the final PFS analysis. DESTINY-Breast09 enrolled 1,157 patients across multiple sites in Africa, Asia, Europe, North America and South America. For more information about the trial, visit ClinicalTrials.gov

About TROPION-Breast02

TROPION-Breast02 is a global, multicenter, randomized, open-label phase 3 trial evaluating the efficacy and safety of Datroway versus investigator’s choice of chemotherapy (paclitaxel, nab-paclitaxel, capecitabine, carboplatin or eribulin) in patients with previously untreated locally recurrent inoperable or metastatic TNBC for whom immunotherapy was not an option. This included patients whose tumors did not express PD-L1 as well as patients with PD-L1 expressing tumors who could not receive immunotherapy due to prior exposure in early-stage disease, comorbidities or immunotherapy not being accessible in their geography. Enrollment included patients with de novo or recurrent disease, regardless of disease-free interval, and those with poor prognostic factors such as stable brain metastases.

The dual primary endpoints of TROPION-Breast02 are OS and PFS as assessed by BICR. Secondary endpoints include PFS as assessed by investigator, ORR, DoR, disease control rate, pharmacokinetics and safety.

TROPION-Breast02 enrolled 644 patients at sites in Africa, Asia, Europe, North America and South America. For more information visit ClinicalTrials.gov.

About Metastatic Breast Cancer

Breast cancer is the most common cancer in women and the leading cause of cancer-related deaths among women worldwide. 4 Approximately 2.4 million breast cancer cases were diagnosed in 2024, with more than 690,000 deaths globally.4 In Japan, breast cancer is the most common cancer in women, with approximately 94,000 cases diagnosed and 16,800 deaths in 2024.

There are three main breast cancer subtypes: HR positive, HER2 positive and TNBC.2,5,6 Approximately 15% to 20% of breast cancer cases are considered HER2 positive where the disease is driven by overexpression or amplification of HER2, and 15% are considered to be triple negative where the tumor tests negative for estrogen receptors, progesterone receptors or overexpression of HER2. 7,8 Both HER2 positive and TNBC are aggressive breast cancer subtypes, with only about 30% and 15% of patients, respectively, living five years following diagnosis.

While HER2 targeted therapies have improved outcomes for patients with HER2 positive metastatic breast cancer, prognosis remains poor with most patients experiencing disease progression within two years of first-line treatment with THP, which has been the standard of care for more than a decade.

For patients with metastatic TNBC with PD-L1 expressing tumors, the addition of immunotherapy to chemotherapy has improved outcomes in the first-line setting.13,14 However, for approximately 70% of patients with metastatic TNBC who are not candidates for immunotherapy, chemotherapy was the standard first-line treatment.

(Press release, Daiichi Sankyo, SEP 16, 2026, View Source [SID1234670875])

On September 16, 2026 Chugai Pharmaceutical Co., Ltd. (TOKYO: 4519) reported that it has obtained approval from the Ministry of Health, Labour and Welfare (MHLW) on June 17, 2026 for FoundationOneCDx Cancer Genomic Profile to be used as a companion diagnostic for Ojemda Tablets 100 mg and Ojemda Dry Syrup 300 mg (generic name: tovorafenib), an anti-cancer agent/BRAF inhibitor, which IPSEN Co., Ltd. obtained a regulatory approval in Japan, for BRAF V600 alterations and BRAF fusion gene-positive glioma.

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This approval enables the detection of BRAF V600 alterations and BRAF fusion genes using the FoundationOne CDx Cancer Genomic Profile to guide the decision to use tovorafenib for BRAF V600 alterations and BRAF fusion gene-positive glioma. The efficacy and safety of tovorafenib for relapsed or progressive low-grade glioma with BRAF V600 alterations or BRAF fusion gene-positive were evaluated in an overseas Phase II clinical study (FIREFLY-1 study). IPSEN Co., Ltd. obtained a regulatory approval from the MHLW on September 16, 2026.

FoundationOne CDx Cancer Genomic Profile has continuously expanded its companion diagnostic capabilities, enabling a single comprehensive genomic profiling test to support treatment decisions for multiple medicines. With the addition of a new companion diagnostic for glioma, the test now has companion diagnostic capabilities covering 9 cancer types including solid tumors. This is expected to further enhance its clinical utility.

As a leading company in oncology, Chugai is committed to realizing more advanced personalized healthcare in the oncology field and contributing to patients through the wider adoption of comprehensive genomic profiling.

Approval information The underlined and bolded part has been newly added.

Intended uses or indications

The product is used for comprehensive genomic profiling of tumor tissues in patients with solid cancers.
The product is used for detecting gene mutations and other alterations to support the assessment of drug indications listed in the table below.
Alterations Cancer type Relevant drugs
Activated EGFR alterations Non-small cell lung cancer (NSCLC) afatinib, erlotinib, gefitinib, osimertinib
EGFR exon 20 T790M alterations osimertinib
ALK fusion genes alectinib, crizotinib, ceritinib, brigatinib
ROS1 fusion genes entrectinib
MET exon 14 skipping alterations capmatinib
BRAF V600E and V600K alterations Malignant melanoma dabrafenib, trametinib, vemurafenib, encorafenib, binimetinib
BRAF V600 alterations and BRAF fusion genes Glioma tovorafenib
ERBB2 copy number alterations (HER2 gene amplification positive) Breast cancer trastuzumab
AKT1 alterations capivasertib
PIK3CA alterations
PTEN alterations
KRAS/NRAS wild-type Colorectal cancer cetuximab, panitumumab
Microsatellite instability high nivolumab
Microsatellite instability high Solid tumors pembrolizumab
Tumor mutational burden high pembrolizumab
NTRK1/2/3 fusion genes entrectinib, larotrectinib, repotrectinib
RET fusion genes selpercatinib
ALK fusion genes alectinib
BRCA1/2 alterations Ovarian cancer olaparib
BRCA1/2 alterations Prostate cancer olaparib
FGFR2 fusion genes Biliary tract cancer pemigatinib

About FoundationOne CDx Cancer Genomic Profile
Developed by Foundation Medicine Inc., FoundationOne CDx Cancer Genomic Profile is a next-generation sequencing based in vitro diagnostic device for the detection of substitutions, insertion and deletion alterations, and copy number alterations in 324 genes and select gene rearrangements, as well as genomic signatures including microsatellite instability (MSI) and tumor mutational burden (TMB) using DNA isolated from formalin-fixed, paraffin-embedded (FFPE) tumor tissue specimens. The program is available as a companion diagnostic for multiple molecular-targeted drugs approved in Japan.

(Press release, Chugai, SEP 16, 2026, View Source;category= [SID1234670874])

On September 15, 2026 Legend Biotech Corporation (NASDAQ: LEGN) ("Legend Biotech" or the "Company"), a global leader in cell therapy, reported that its Board of Directors has appointed Ingrid Zhang as Chief Executive Officer, effective September 15, 2026. Ingrid Zhang succeeds Alan Bash, who has served as Interim Chief Executive Officer since July 2026 and will resume his role as President, CARVYKTI.

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Ingrid Zhang is an accomplished global biopharmaceutical executive with deep experience leading commercial organizations, driving business growth, and advancing innovative medicines. Most recently, she served as Chief Commercial Officer for Novartis International. During her tenure at Novartis, she also led the company’s China business and innovative medicines organization. Earlier in her career, she held leadership positions at AstraZeneca, Pfizer, and McKinsey & Company, bringing a combination of global commercial expertise, operational leadership, and strategic insight to Legend Biotech.

"It is a privilege to join Legend Biotech at an important moment in the Company’s growth," said Ingrid Zhang, Chief Executive Officer of Legend Biotech. "Legend Biotech has established itself as a global leader in cell therapy through its commitment to scientific innovation, operational excellence, and transforming outcomes for patients. I look forward to working with the talented team at Legend to build on this strong foundation, advance our pipeline, expand our impact, and execute on our long-term growth strategy."

"Following a comprehensive search process, the Board concluded that Ingrid is the right leader to guide Legend Biotech through its next phase of growth," said Frank Zhang, EMBA, Ph.D., Chairman of the Board of Legend Biotech. "As a highly respected industry executive, Ingrid brings strategic vision, operational discipline, and experience leading complex global businesses. The Board is confident she is well-positioned to advance our mission of transforming patient care through cell therapy. We thank Alan Bash for his strong leadership as Interim CEO and are delighted to welcome Ingrid to Legend Biotech."

"Throughout this transition, our team has continued to execute against our strategic priorities with focus across the business," said Alan Bash, President, CARVYKTI. "The commitment and expertise of colleagues around the world have positioned Legend Biotech for its next chapter. The leadership team and I look forward to partnering closely with Ingrid as we continue executing our strategy and delivering meaningful impact for patients."

(Press release, Legend Biotech, SEP 15, 2026, View Source [SID1234670892])

On September 15, 2026 Juniper Biosciences reported two previously undisclosed radiopharmaceutical programs: JBS-002, a Ga-68 based cold kit for prostate cancer imaging, and JBS-004, a fluorine-18 labeled diagnostic imaging agent for Alzheimer’s disease. Both assets have been advanced privately through development and are disclosed today at the registrational stage, with registration batch manufacturing scheduled for the fourth quarter of 2026 in support of planned submissions to the U.S. Food and Drug Administration (FDA) in 2027.

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The Company is disclosing both assets now that they stand at the threshold of regulatory submission, revealing a deep late-stage diagnostic portfolio — two registrational programs addressing large, established imaging markets.

JBS-002: A Ga-68 based Kit for Precision Prostate Cancer Imaging

Prostate-specific membrane antigen (PSMA) is a transmembrane protein expressed at low levels in normal prostate tissue but dramatically overexpressed on the surface of prostate cancer cells, including in metastatic and hormone-resistant disease. JBS-002 is an investigational PET imaging agent that binds directly to PSMA-expressing lesions and renders them visible on a whole-body scan. PSMA PET imaging is now established in clinical practice for staging men with high-risk disease before definitive treatment and for localizing the site of recurrence in patients with rising PSA after surgery or radiation, findings that frequently change the intended course of therapy.

PSMA PET has become standard of care, and the U.S. market is now estimated at approximately $1.8 billion annually. Growth continues on two fronts: expanding NCCN-guideline use across the disease course, and the rapid adoption of PSMA-targeted radioligand therapy, which depends on PSMA imaging to select and monitor patients.

JBS-004: An F-18 Diagnostic Imaging Agent for Alzheimer’s Disease

JBS-004 is an investigational fluorine-18 labeled PET imaging agent designed to provide a non-invasive visual assessment of Alzheimer’s disease pathology in adult patients being evaluated for cognitive impairment. Historically, a definitive determination of Alzheimer’s pathology could be made only at autopsy, leaving clinicians to diagnose by exclusion. Molecular imaging changes that calculus by making the underlying pathology directly observable in living patients, supporting earlier diagnostic certainty, reducing misdiagnosis in a population where symptoms overlap with numerous other conditions, and identifying which patients are appropriate candidates for the new generation of disease-modifying Alzheimer’s therapies—treatments that require confirmation of pathology before they can be prescribed.

The commercial context for this imaging has changed fundamentally in recent years. The arrival of therapies converted Alzheimer’s PET from a research tool into a required gateway to treatment. Scan volumes have grown sharply against a small field of approved agents.

An Expanding Late-Stage Portfolio

With JBS-002 and JBS-004 both entering registration batch manufacturing in the fourth quarter of 2026, Juniper Biosciences has two late-stage diagnostic assets advancing in parallel toward FDA submission in 2027, alongside JBS-003, the Company’s previously disclosed Phase III program in HPV-positive oropharyngeal cancer. Three programs now sit in registrational or Phase III development across prostate cancer, Alzheimer’s disease, and head and neck cancer—a portfolio built by a team with deep operating experience in radiochemistry and complex manufacturing.

"Reaching registration batch manufacturing on two programs in the same quarter is a defining moment for a company of our size," said Alex Agnoletto, Chief Executive Officer of Juniper Biosciences. "JBS-002 and JBS-004 serve different physicians and different diseases, but they reflect the same conviction: better decisions require better information. Together with JBS-003, they give us three programs in registrational or Phase III development and a clear path to becoming a commercial-stage company in the near future"

Commercialization

Juniper Biosciences intends to commercialize JBS-002 and JBS-004 in the United States if the programs receive marketing approval from the FDA. The Company is advancing both agents through registration with the objective of transitioning from a development-stage organization to a commercial-stage one, and preparatory work to support a potential launch of each program is proceeding in parallel with the regulatory work described above.

JBS-002 and JBS-004 are investigational agents. Neither has been approved by the FDA or any other regulatory authority, and the safety and effectiveness of neither agent has been established. Any commercial launch is contingent on FDA approval, and neither the outcome nor the timing of the regulatory review process can be assured.

(Press release, Juniper Biosciences, SEP 15, 2026, View Source [SID1234670891])