Mauna Kea Technologies Announces Ground-Breaking Positive Clinical Results in Lung Cancer Obtained with Cellvizio

On September 16, 2024 Mauna Kea Technologies (Euronext Growth: ALMKT), inventor of Cellvizio, the multidisciplinary probe and needle-based confocal laser endomicroscopy (p/nCLE) platform, reported the publication in the peer-reviewed Journal of Thoracic Disease of new ground-breaking clinical results demonstrating the efficacy of its needle-based Confocal Laser Endomicroscopy (nCLE) technology for the characterization of peripheral lung cancer and preparation of surgical resection (Press release, Mauna Kea Technologies, SEP 16, 2024, View Source [SID1234646690]).

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This study, conducted by the team of Professor Stéphane Renaud at the University Hospital of Nancy, evaluated the use of Cellvizio’s nCLE platform in combination with Electromagnetic Navigation Bronchoscopy (ENB) to characterize suspicious pulmonary nodules and prepare them for surgical resection when needed. The results highlight the potential of nCLE to rapidly and accurately identify malignant lesions while minimizing the risks associated with traditional diagnostic methods including ionizing radiation.

The study included 30 patients with suspicious pulmonary nodules with a median size of 16 mm. The findings revealed a sensitivity of 96.43% and a specificity of 100% in characterizing malignant lesions, based on previously published nCLE image classification. The median time of contact with the suspicious lesions was just 5 minutes with no major complications reported, demonstrating the technology’s efficiency in clinical practice. Additionally, the use of nCLE also improved the quality and quantity of tissue sampling, thus enabling molecular and genomics analyses, which have become essential tools for the choice and planning of treatments.

Professor Stéphane Renaud, Thoracic Surgeon at University Hospital Nancy and principal investigator of the study, stated: "We are extremely pleased with the results of this study, which brings to light the high diagnostic value of needle-based endomicroscopy in combination with Electromagnetic Navigation Bronchoscopy and its ability to improve our treatment planning and minimize patient risk. With a much-enhanced ability to target our biopsies, we have also been able to obtain essential tissue material for molecular and genomics analyses, which are crucial for guiding immunotherapy strategies. This combination of technologies will certainly play a key role in the fight against lung cancer in the future."

"We are thrilled by these outstanding clinical results, which reinforce the value of our needle-based endomicroscopy platform in advancing the field of lung cancer diagnostics and treatment," commented Sacha Loiseau, Ph.D., Chairman and CEO of Mauna Kea Technologies. "This study confirms that Cellvizio can play a pivotal role not only in enabling real-time, accurate, and minimally invasive diagnosis, but also in helping physicians improve their treatment planning and, in turn, patient outcomes."

ESMO 2024 Late-Breaking Data: Phase III ARANOTE Trial Shows NUBEQA® (darolutamide) Significantly Reduced Risk of Radiological Progression or Death in Metastatic Hormone-Sensitive Prostate Cancer

On September 16, 2024 Bayer reported results from the investigational pivotal Phase III ARANOTE trial demonstrated that NUBEQA (darolutamide) plus androgen deprivation therapy (ADT) showed a statistically significant and clinically meaningful improvement in radiological progression-free survival (rPFS) compared to placebo plus ADT in patients with metastatic hormone-sensitive prostate cancer (mHSPC) (Press release, Bayer, SEP 16, 2024, View Source [SID1234646689]). The results were presented today as a late-breaking oral presentation at the 2024 European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress in Barcelona, Spain and published simultaneously in The Journal of Clinical Oncology.

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The results were consistent with the established safety profile of NUBEQA, with no new safety signals observed. Rates of serious adverse events were similar between the treatment arms (23.6% for NUBEQA plus ADT compared to 23.5% for placebo plus ADT), while discontinuation due to treatment-emergent adverse events (TEAEs) was 6.1% in patients treated with NUBEQA plus ADT compared to 9% in patients receiving placebo plus ADT.1

NUBEQA is indicated in the U.S. for the treatment of adult patients with mHSPC in combination with docetaxel and for the treatment of adult patients with non-metastatic castration-resistant prostate cancer (nmCRPC).2

The randomized, double-blind, placebo-controlled Phase III ARANOTE trial was designed to assess the efficacy and safety of NUBEQA plus ADT in patients with mHSPC. A total of 669 patients were randomized 2:1 to receive either 600 mg of NUBEQA (N=446) or placebo (N=223) twice daily in addition to ADT.1

"Each diagnosis of metastatic hormone-sensitive prostate cancer is unique, shaped by factors such as age, comorbidities and patient preferences. Each patient therefore requires a tailored treatment approach that thoughtfully addresses these key considerations," said Fred Saad, Professor and Chairman of Surgery and Director of Genitourinary Oncology at the University of Montreal Hospital Center (CHUM), and Principal Investigator of the ARANOTE trial. "With the positive results from ARANOTE, in addition to the ARASENS data, darolutamide has now demonstrated efficacy and safety data both with and without chemotherapy in mHSPC."

"The positive outcomes from the ARANOTE trial provide physicians with additional data that could broaden the use of NUBEQA as a treatment option for more patients with metastatic hormone-sensitive prostate cancer, which accounts for approximately 10% of prostate cancer diagnoses in the United States," said Neal Shore, M.D., FACS, Medical Director, Carolina Urologic Research Center. "These data demonstrate the potential of this therapy to provide significant benefits to patients with mHSPC, regardless of chemotherapy use."

"The ARANOTE trial was designed to investigate NUBEQA plus ADT compared to placebo plus ADT to provide an additional treatment option for patients with metastatic hormone-sensitive prostate cancer," said Christian Rommel, Ph.D., Head of Research and Development at Bayer’s Pharmaceuticals Division. "Supported by our robust clinical development program, our goal is to expand the option of NUBEQA to as many patients as possible."

Bayer plans to submit the data from the ARANOTE trial to the U.S. Food and Drug Administration (FDA) to support the expanded use of NUBEQA in patients with mHSPC.

Detailed Results from ARANOTE1

Results of the rPFS analysis were consistent across prespecified subgroups, including 40% risk reduction (HR 0.60, 95% CI: 0.44-0.80) in patients with high-volume mHSPC and 70% risk reduction (HR 0.30, 95% CI: 0.15-0.60) in patients with low-volume disease. An analysis of immature overall survival data (OS), which measures the time from treatment until death from any cause, showed an HR of 0.81 (95% CI 0.59-1.12) versus placebo plus ADT. The ARANOTE data also suggested clinical benefits across all other secondary endpoints, including delaying the time to castration-resistant prostate cancer (CRPC) (HR 0.40; 95% CI, 0.32-0.51), time to PSA progression (HR 0.31; 95% CI 0.23-0.41), time to pain progression (HR 0.72; 95% CI 0.54-0.96), and time to initiation of subsequent systemic therapy (HR 0.40; 95% CI 0.29-0.56), compared to placebo plus ADT, though not assessed for statistical significance.

Incidence rates for adverse events Grade 3 or higher were similar between the two groups (35.5% and 35.7%, respectively). The incidence of fatigue was lower with NUBEQA plus ADT than with placebo plus ADT (5.6% and 8.1%, respectively).

About the ARANOTE Trial1

The primary endpoint of the ARANOTE trial is rPFS, measured as time from randomization to date of first documented radiological disease progression or death due to any cause, whichever occurs first. Secondary endpoints include overall survival (time to death from any cause), time to first castration-resistant event, time to initiation of subsequent anti-cancer therapy, time to prostate-specific antigen (PSA) progression, PSA undetectable rates, time to pain progression, and safety assessments.

About the ARASENS Trial3

The ARASENS trial (NCT02799602) is the only randomized Phase III, multi-center, double-blind, placebo-controlled trial prospectively designed to compare the use of a second-generation androgen receptor inhibitor (ARi) (NUBEQA) plus androgen deprivation therapy (ADT) and the chemotherapy docetaxel to ADT plus docetaxel in patients with metastatic hormone-sensitive prostate cancer (mHSPC). A total of 1,306 newly diagnosed patients were randomized in a 1:1 ratio to receive 600 mg of NUBEQA twice a day or matching placebo plus ADT and docetaxel.

The primary endpoint of the trial was overall survival (OS). Secondary endpoints included time to castration-resistant prostate cancer (CRPC), time to pain progression, time to first symptomatic skeletal event (SSE), and time to initiation of subsequent anticancer therapy, all measured at 12-week intervals, as well as adverse events (AEs) as a measure of safety and tolerability.

About NUBEQA (darolutamide)2

NUBEQA (darolutamide) is an androgen receptor inhibitor (ARi) with a distinct chemical structure that competitively inhibits androgen binding, AR nuclear translocation, and AR-mediated transcription.

In addition to the ARANOTE trial, darolutamide is being evaluated in a robust clinical development program, which includes studies across various stages of prostate cancer, including in the ARASTEP Phase III trial evaluating darolutamide plus ADT versus ADT alone in HSPC patients with high-risk biochemical recurrence (BCR), no evidence of metastatic disease by conventional imaging, and a positive PSMA PET/CT at baseline, as well as in the Australian and New Zealand Urogenital and Prostate Cancer Trials Group (ANZUP) led international Phase III co-operative group DASL-HiCaP (ANZUP1801) trial evaluating NUBEQA as an adjuvant treatment for localized prostate cancer with very high risk of recurrence. Information about these trials can be found at www.clinicaltrials.gov.

NUBEQA is developed jointly by Bayer and Orion Corporation, a globally operating Finnish pharmaceutical company.

INDICATIONS

NUBEQA (darolutamide) is an androgen receptor inhibitor indicated for the treatment of adult patients with:

Non-metastatic castration-resistant prostate cancer (nmCRPC)
Metastatic hormone-sensitive prostate cancer (mHSPC) in combination with docetaxel
IMPORTANT SAFETY INFORMATION

Warnings & Precautions

Ischemic Heart Disease – In a study of patients with nmCRPC (ARAMIS), ischemic heart disease occurred in 3.2% of patients receiving NUBEQA versus 2.5% receiving placebo, including Grade 3-4 events in 1.7% vs. 0.4%, respectively. Ischemic events led to death in 0.3% of patients receiving NUBEQA vs. 0.2% receiving placebo. In a study of patients with mHSPC (ARASENS), ischemic heart disease occurred in 3.2% of patients receiving NUBEQA with docetaxel vs. 2% receiving placebo with docetaxel, including Grade 3-4 events in 1.3% vs. 1.1%, respectively. Ischemic events led to death in 0.3% of patients receiving NUBEQA with docetaxel vs. 0% receiving placebo with docetaxel. Monitor for signs and symptoms of ischemic heart disease. Optimize management of cardiovascular risk factors, such as hypertension, diabetes, or dyslipidemia. Discontinue NUBEQA for Grade 3-4 ischemic heart disease.

Seizure – In ARAMIS, Grade 1-2 seizure occurred in 0.2% of patients receiving NUBEQA vs. 0.2% receiving placebo. Seizure occurred 261 and 456 days after initiation of NUBEQA. In ARASENS, seizure occurred in 0.6% of patients receiving NUBEQA with docetaxel, including one Grade 3 event, vs. 0.2% receiving placebo with docetaxel. Seizure occurred 38 to 340 days after initiation of NUBEQA. It is unknown whether antiepileptic medications will prevent seizures with NUBEQA. Advise patients of the risk of developing a seizure while receiving NUBEQA and of engaging in any activity where sudden loss of consciousness could cause harm to themselves or others. Consider discontinuation of NUBEQA in patients who develop a seizure during treatment.

Embryo-Fetal Toxicity – Safety and efficacy of NUBEQA have not been established in females. NUBEQA can cause fetal harm and loss of pregnancy. Advise males with female partners of reproductive potential to use effective contraception during treatment with NUBEQA and for 1 week after the last dose.

Adverse Reactions

In ARAMIS, serious adverse reactions occurred in 25% of patients receiving NUBEQA vs. 20% of patients receiving placebo. Serious adverse reactions in ≥1% of patients who received NUBEQA included urinary retention, pneumonia, and hematuria. Fatal adverse reactions occurred in 3.9% of patients receiving NUBEQA vs. 3.2% of patients receiving placebo. Fatal adverse reactions in patients who received NUBEQA included death (0.4%), cardiac failure (0.3%), cardiac arrest (0.2%), general physical health deterioration (0.2%), and pulmonary embolism (0.2%). The most common adverse reactions (>2% with a ≥2% increase over placebo), including laboratory test abnormalities, were increased AST, decreased neutrophil count, fatigue, increased bilirubin, pain in extremity and rash. Clinically relevant adverse reactions occurring in ≥2% of patients treated with NUBEQA included ischemic heart disease and heart failure.

In ARASENS, serious adverse reactions occurred in 45% of patients receiving NUBEQA with docetaxel vs. 42% of patients receiving placebo with docetaxel. Serious adverse reactions in ≥2% of patients who received NUBEQA with docetaxel included febrile neutropenia (6%), decreased neutrophil count (2.8%), musculoskeletal pain (2.6%), and pneumonia (2.6%). Fatal adverse reactions occurred in 4% of patients receiving NUBEQA with docetaxel vs. 4% of patients receiving placebo with docetaxel. Fatal adverse reactions in patients who received NUBEQA included COVID-19/COVID-19 pneumonia (0.8%), myocardial infarction (0.3%), and sudden death (0.3%). The most common adverse reactions (≥10% with a ≥2% increase over placebo with docetaxel) were constipation, rash, decreased appetite, hemorrhage, increased weight, and hypertension. The most common laboratory test abnormalities (≥30%) were anemia, hyperglycemia, decreased lymphocyte count, decreased neutrophil count, increased AST, increased ALT, and hypocalcemia. Clinically relevant adverse reactions in <10% of patients who received NUBEQA with docetaxel included fractures, ischemic heart disease, seizures, and drug-induced liver injury.

Drug Interactions

Effect of Other Drugs on NUBEQA – Combined P-gp and strong or moderate CYP3A4 inducers decrease NUBEQA exposure, which may decrease NUBEQA activity. Avoid concomitant use.

Combined P-gp and strong CYP3A4 inhibitors increase NUBEQA exposure, which may increase the risk of NUBEQA adverse reactions. Monitor more frequently and modify NUBEQA dose as needed.

Effects of NUBEQA on Other Drugs – NUBEQA inhibits breast cancer resistance protein (BCRP) transporter. Concomitant use increases exposure (AUC) and maximal concentration of BCRP substrates, which may increase the risk of BCRP substrate-related toxicities. Avoid concomitant use where possible. If used together, monitor more frequently for adverse reactions, and consider dose reduction of the BCRP substrate.

NUBEQA inhibits OATP1B1 and OATP1B3 transporters. Concomitant use may increase plasma concentrations of OATP1B1 or OATP1B3 substrates. Monitor more frequently for adverse reactions and consider dose reduction of these substrates.

Review the Prescribing Information of drugs that are BCRP, OATP1B1, and OATP1B3 substrates when used concomitantly with NUBEQA.

For important risk and use information about NUBEQA, please see the full Prescribing Information.

About Metastatic Hormone-Sensitive Prostate Cancer

Prostate cancer is the second most common cancer in men and the fifth most common cause of cancer death in men worldwide.4 In 2020, an estimated 1.4 million men were diagnosed with prostate cancer, including almost 300,000 cases in the U.S., and about 375,000 died from the disease worldwide.5,6

At the time of diagnosis, most men have localized prostate cancer, meaning their cancer is confined to the prostate gland and can be treated with curative surgery or radiotherapy. Upon relapse when the disease will metastasize or spread, androgen deprivation therapy (ADT) is the cornerstone of treatment for this hormone-sensitive disease. Approximately 10% of men will already present with mHSPC when first diagnosed.7,8,9 Men with metastatic hormone-sensitive prostate cancer (mHSPC) will start their treatment with hormone therapy, such as ADT, androgen receptor inhibitor (ARi) plus ADT, or a combination of the chemotherapy docetaxel and ADT. Despite this treatment, most men with mHSPC will eventually progress to castration-resistant prostate cancer (CRPC), a condition with limited survival.

Natera Announces Three New Signatera Publications; Includes Groundbreaking Overall Survival Data Published in Nature Medicine and also Released at ESMO

On September 16, 2024 Natera, Inc. (NASDAQ: NTRA), a global leader in cell-free DNA and genetic testing, reported the simultaneous publication of three peer-reviewed papers, crossing a milestone of more than 85 peer-reviewed publications on Signatera (Press release, Natera, SEP 16, 2024, View Source [SID1234646688]).
This includes:

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Groundbreaking colorectal cancer (CRC) data from the GALAXY arm of the ongoing CIRCULATE-Japan trial, published today in Nature Medicine, and also available in Poster Presentation #553P at the 2024 Congress of the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) in Barcelona, Spain.
Additional CRC data from GALAXY, published today in Annals of Oncology, and also available in Poster Presentation #558P at ESMO (Free ESMO Whitepaper).
A new paper from the BELLINI trial, published today in Nature Medicine, investigating the feasibility and potential efficacy of immune checkpoint inhibitors (ICI) without concurrent chemotherapy in triple negative breast cancer (TNBC).
CRC Data from GALAXY (Nature Medicine & ESMO (Free ESMO Whitepaper) Poster)

In this study, 2,240 patients with stage II– IV CRC were monitored using Signatera after curative-intent surgery with a median follow-up of 23 months. This data provides the first evidence of Signatera-based molecular residual disease (MRD) detection to predict overall survival (OS) and highlights Signatera’s ability to predict adjuvant chemotherapy (ACT) benefit. Full details on the data are available here, as announced on Sept. 14, 2024.

CRC Data from GALAXY (Annals of Oncology & ESMO (Free ESMO Whitepaper) Poster)

This study retrospectively analyzed 190 patients enrolled in GALAXY who underwent surgical resection for colorectal liver metastases and underscores Signatera’s ability to risk-stratify CRC patients. Of the patients who were Signatera-positive within 2-10 weeks after surgical resection, 24-month disease free survival (DFS) was superior for those who received ACT compared to patients on observation, whereas no statistically significant benefit of ACT was observed among Signatera-negative patients.

Breast Cancer Data from Bellini Trial (Nature Medicine)

This study reports initial results from the BELLINI trial, a phase 2 study that enrolled 43 patients with stage I-III triple-negative breast cancer (TNBC). Patients in the study underwent short-term (2-3 cycles) of nivolumab, alone or in combination with ipilimumab, prior to standard-of-care neoadjuvant chemotherapy or surgery. Circulating tumor DNA (ctDNA) analysis with Signatera was performed before treatment and after 4 or 6 weeks of treatment. All clinical responders including patients who achieved pathologic complete response demonstrated at least a 50% decrease in ctDNA levels, or were ctDNA-negative at baseline.

"We are extremely proud to announce the publication of these datasets in top-tier medical journals, underscoring the breadth of clinical evidence we continue to build on the utility of Signatera," said Alexey Aleshin, MD, MBA, general manager of oncology and chief medical officer of Natera. "The first overall survival readout in colorectal cancer from GALAXY marks a landmark moment that may fundamentally change how resectable colorectal cancer is managed for the hundreds of thousands of patients at risk of recurrence each year. We are especially grateful to the patients who participated in these trials, as well as our collaborators and study investigators for their dedication to improving outcomes for patients."

About Signatera

Signatera is a personalized, tumor-informed, molecular residual disease test for patients previously diagnosed with cancer. Custom-built for each individual, Signatera uses circulating tumor DNA to detect and quantify cancer left in the body, identify recurrence earlier than standard of care tools, and help optimize treatment decisions. The test is available for clinical and research use and is covered by Medicare for patients with colorectal cancer, breast cancer, ovarian cancer and muscle invasive bladder cancer, as well as for immunotherapy monitoring of any solid tumor. Signatera has been clinically validated across multiple cancer types and indications, with published evidence in more than 85 peer-reviewed papers.

Data from a Phase 1 Trial of HighField Biopharmaceuticals’ HF1K16 Show the New Immuno-Oncology Drug is Safe and Signals Efficacy in Treating Refractory Metastatic Cancers

On September 16, 2024 HighField Biopharmaceuticals, a clinical stage company using lipid-based therapeutics to treat cancer, diabetes and other diseases, reported its CEO and Scientific Founder Yuhong Xu, Ph.D, presented positive safety and efficacy findings from an ongoing Phase 1 clinical trial of HF1K16 (K16) in refractory metastatic cancer patients at the European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress in Barcelona, Spain, September 13 – 17, 2024 (Press release, HighField Biopharmaceuticals, SEP 16, 2024, View Source [SID1234646687]).

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K16 is an ATRA-encapsulated immune modulating liposome combining a lipid bilayer structure and all-trans retinoic acid (ATRA). It targets immature immune cells, called myeloid-derived suppressor cells (MDSCs), inducing them to become mature active immune cells, such as dendritic cells, which summon T cells to attack cancer.

Presentation of the poster titled, "A Phase 1 study of the Myeloid-derived suppressor cells modulator HF1K16 in refractory and metastatic cancer patients: preliminary efficacy and safety," was on Saturday, September 14 in the Investigational Immunotherapy session.

"We are excited about the preliminary safety and efficacy data from our ongoing Phase 1 study," said Dr. Xu. "ATRA is a highly active vitamin A metabolite commonly found in the body. Using Highfield’s liposome technology, we were able to inject a highly concentrated ATRA offering more than 10 times higher than normal exposure in a patient, triggering the therapeutic mechanism. These patients have very advanced, heavily prior treated tumors."

The Phase 1 dose escalation study (NCT05388487) showed significant signals of desirable immune modulation and efficacy. The expansion cohort, currently recruiting, is focused on R/R glioma. As of Sept 1, 2024, 19 patients having Grade II, III and IV tumors have been enrolled. Among the 10 patients with Grade II and III gliomas, median overall survival (mOS) is 629 days. For the 9 patients with Grade IV or glioblastoma, the most aggressive brain tumors, mOS is 315 days.

The next step will be to determine the indication that is most beneficial either as a single agent or in combination with other standard of care regimen in a Phase 2 study.

Promising Anti-Tumor Activity and Safety of Ivonescimab in Combination Therapies in CRC, TNBC, and HNSCC Featured at ESMO 2024

On September 16, 2024 Summit Therapeutics Inc. (NASDAQ: SMMT) ("Summit," "we," or the "Company") reported that data for the novel, potential first-in-class investigational bispecific antibody, ivonescimab, was presented at the 2024 European Society for Medical Oncology Annual Meeting (ESMO 2024) in Barcelona, Spain, including two presentations and one poster featuring updated ivonescimab data in advanced triple-negative breast cancer (TNBC), recurrent / metastatic head and neck squamous cell carcinoma (HNSCC), and metastatic microsatellite-stable (MSS) colorectal cancer (CRC) (Press release, Summit Therapeutics, SEP 16, 2024, View Source [SID1234646686]). Each trial from which the data was generated was a Phase II study conducted in China sponsored by Akeso Inc. (HKEX Code: 9926.HK) with data generated and analyzed by Akeso.

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Based on the results of these Phase II data sets as well as data announced earlier in 2024, including early-stage non-small cell lung cancer and biliary tract cancer, Summit intends to explore further clinical development of ivonescimab in solid tumor settings outside of metastatic non-small cell lung cancer, the Company’s current area of focus in its Phase III clinical trials.

Metastatic MSS Colorectal Cancer

The first oral presentation was presented by Dr. Yanhong Deng, Sun Yat-Sen University. The presentation was entitled, The efficacy and safety of ivonescimab with or without ligufalimab in combination with FOLFOXIRI (chemotherapy) as first-line treatment for metastatic CRC, presenting the current data from AK112-206, included data from this single-region (China), multicenter, open-label, Phase II randomized study of patients with first-line metastatic MSS CRC (NCT05382442).

The study was designed to assess patients who were randomly assigned to receive ivonescimab plus FOLFOXIRI with or without ligufalimab (anti-CD47 monoclonal antibody). Note that ligufalimab, or AK117, is Akeso’s proprietary, investigational product that is not approved by any regulatory authority, and to which Summit does not have any license or ownership rights.

As of February 29, 2024, 22 patients received ivonescimab plus FOLFOXIRI ("Group A" with median follow-up time of 9 months); 18 patients received ivonescimab plus ligufalimab plus FOLFOXIRI ("Group B" with median follow-up time of 9.6 months).

Ivonescimab + Chemo

(Group A) (n = 22)

Ivonescimab + Ligufalimab + Chemo (Group B) (n = 18)a

Overall response rate

81.8%

(95% CI: 59.7, 94.8)

88.2%

(95% CI: 63.6, 98.5)

Disease control rate

100%

(95% CI: 84.6, 100)

100%

(95% CI: 80.5, 100)

Median PFS

NR

NR

9-month PFS rate

81.4%

(95% CI: 52.1, 93.7)

86.2%

(95% CI: 55.0, 96.4)

Serious TRAE

22.7%

11.1%

TRAEs Leading to Permanent Discontinuation

0

5.6%

a As of data cutoff, one patient in Group B had not yet had a post-baseline tumor assessment; Group B response and control rates based on n=17.

All patients in both Group A and Group B experienced a reduction in their tumor burden compared to their baseline tumor assessment. The overall response rate and the disease control rate for the 39 patients who had a least one post-baseline tumor assessment was 84.6% and 100%, respectively. Median progression-free survival was not reached in either group at the time of this analysis.

The safety profile in this Phase II study was acceptable and manageable. No patients receiving ivonescimab plus FOLFOXIRI and one patient receiving ivonescimab plus ligufalimab plus FOLFOXIRI permanently discontinued drug treatment due to treatment-related adverse events. The most common TRAEs were anemia, proteinuria, white blood cell count decreases, and neutrophil count decreases in this Phase II data set.

Advanced Triple Negative Breast Cancer

The second oral presentation was presented by Dr. Xiaojia Wang, Zhejiang Cancer Hospital. The presentation was entitled, The safety and efficacy of ivonescimab in combination with chemotherapy as first-line (1L) treatment for triple-negative breast cancer (TNBC), presenting the current data from AK117-203, included data from this single-region (China), multicenter, open-label, Phase II study (NCT05227664).

The results presented were from the portion of the study intended to assess patients who received ivonescimab plus chemotherapy (either paclitaxel or nab-paclitaxel) with locally advanced or metastatic TNBC.

As of May 31, 2024, 30 patients received ivonescimab plus chemotherapy with median follow-up time of 10.2 months. Sixty percent of patients had previously received taxane-based chemotherapy in either the neoadjuvant or adjuvant setting in this Phase II data set.

Overall

(n = 30)a

Overall response rate

72.4%

Disease control rate

100%

Median PFS

9.3 months

(95% CI: 6.24, NE)

Serious TRAE

30%

TRAEs Leading to Permanent Discontinuation

0

PD-L1 CPS >10

(n = 6)

PD-L1 CPS <10

(n = 24)a

PD-L1 CPS <1

(n = 16)a

Overall response rate

83.3%

69.6%

86.7%

Disease control rate

100%

100%

100%

Median PFS

NR

(5.36, NE)

9.3 months

(5.55, NE)

9.3 months

(5.26, NE)

a As of data cutoff, one patient with a PD-L1 CPS expression of 0 had not yet had a post-baseline tumor assessment; Overall patients, PD-L1 CPS <10, and PD-L1 CPS <1 response and control rates based on n=29, n=23, and n=15, respectively.

All patients experienced a reduction in their tumor burden compared to their baseline tumor assessment. The overall response rate and the disease control rate for the 29 patients who had at least one post-baseline tumor assessment were 72.4% and 100%, respectively. Median progression-free survival was 9.30 months as the time of this analysis.

The safety profile in this Phase II study was acceptable and manageable. No patients receiving ivonescimab plus chemotherapy permanently discontinued drug treatment due to treatment-related adverse events. The most common TRAEs were white blood cell count decreases, ALT increases, alopecia, AST increases, and neutrophil count decreases in this Phase II data set.

Recurrent / Metastatic Head and Neck Squamous Cell Carcinoma

The third data presentation was a poster from Dr. Xiaozhong Chen, et al. The poster was entitled, Evaluation of the safety and efficacy of ivonescimab in combination with ligufalimab (anti-CD47) as first-line treatment for PD-L1 positive recurrent/metastasis HNSCC, presenting current data from a portion of AK117-201. The data is from a single-region (China), multicenter, open-label, Phase II study (NCT05229497).

The results presented were from the portion of the study intended to assess patients who received ivonescimab with or without ligufalimab (anti-CD47) with PD-L1 positive, locally advanced or metastatic recurrent / metastatic head and neck squamous cell carcinoma.

As of March 19, 2024, 10 patients received ivonescimab (median follow-up: 3.3 months) and 20 patients received ivonescimab plus ligufalimab (median follow-up 4.1 months). Four of 10 patients receiving ivonescimab had a PD-L1 CPS of 1-20; nine of 20 patients administered ivonescimab plus ligufalimab had a PD-L1 CPS of 1-20; the remaining patients in each arm had a PD-L1 CPS >20.

Ivonescimab

(n=10)

Ivonescimab + Ligufalimab

(n = 20)

Overall response rate

30.0%

60.0%

Disease control rate

80.0%

90.0%

Median PFS

5.0 months

7.1 months

6-month PFS rate

NR

71.8%

Serious TRAE

0

5.0%

TRAEs Leading to Permanent Discontinuation

0

0

The overall response rate and the disease control rate for the 30 patients was 50.0% and 86.7%, respectively.

The safety profile in this Phase II study was acceptable and manageable. No patients receiving ivonescimab or ivonescimab plus ligufalimab in this data set permanently discontinued drug treatment due to treatment-related adverse events. The most common TRAEs in this Phase II data set were proteinuria, dermatitis acneiform (each observed in both arms), and hypothyroidism (observed only in the ivonescimab plus ligufalimab arm).

About Ivonescimab

Ivonescimab, known as SMT112 in Summit’s license territories, the United States, Canada, Europe, Japan, Latin America, including Mexico and all countries in Central America, South America, and the Caribbean, the Middle East, and Africa, and as AK112 in China and Australia, is a novel, potential first-in-class investigational bispecific antibody combining the effects of immunotherapy via a blockade of PD-1 with the anti-angiogenesis effects associated with blocking VEGF into a single molecule. Ivonescimab displays unique cooperative binding to each of its intended targets with multifold higher affinity when in the presence of both PD-1 and VEGF.

This could differentiate ivonescimab as there is potentially higher expression (presence) of both PD-1 and VEGF in tumor tissue and the tumor microenvironment (TME) as compared to normal tissue in the body. Ivonescimab’s tetravalent structure (four binding sites) enables higher avidity (accumulated strength of multiple binding interactions) in the TME with over 18-fold increased binding affinity to PD-1 in the presence of VEGF in vitro, and over 4-times increased binding affinity to VEGF in the presence of PD-1 in vitro (Zhong, et al, SITC (Free SITC Whitepaper), 2023). This tetravalent structure, the intentional novel design of the molecule, and bringing these two targets into a single bispecific antibody with cooperative binding qualities have the potential to direct ivonescimab to the tumor tissue versus healthy tissue. The intent of this design, together with a half-life of 6 to 7 days (Zhong, et al, SITC (Free SITC Whitepaper), 2023), is to improve upon previously established efficacy thresholds, in addition to side effects and safety profiles associated with these targets.

Ivonescimab was engineered by Akeso Inc. (HKEX Code: 9926.HK) and is currently engaged in multiple Phase III clinical trials. Over 1,800 patients have been treated with ivonescimab in clinical studies globally.

Summit has begun its clinical development of ivonescimab in non-small cell lung cancer (NSCLC), commencing enrollment in 2023 in two multi-regional Phase III clinical trials, HARMONi and HARMONi-3, with a plan to initiate HARMONi-7 in early 2025.

HARMONi is a Phase III clinical trial which intends to evaluate ivonescimab combined with chemotherapy compared to placebo plus chemotherapy in patients with EGFR-mutated, locally advanced or metastatic non-squamous NSCLC who have progressed after treatment with a 3rd generation EGFR TKI (e.g., osimertinib).

HARMONi-3 is a Phase III clinical trial which is designed to evaluate ivonescimab combined with chemotherapy compared to pembrolizumab combined with chemotherapy in patients with first-line metastatic squamous NSCLC.

HARMONi-7 is a planned Phase III clinical trial which is intended to evaluate ivonescimab monotherapy compared to pembrolizumab monotherapy in patients with first-line metastatic NSCLC whose tumors have high PD-L1 expression (PD-L1 TPS > 50%).

In addition, Akeso has recently had positive read-outs in two single-region (China), randomized Phase III clinical trials for ivonescimab in NSCLC, HARMONi-A and HARMONi-2.

HARMONi-A was a Phase III clinical trial which evaluated ivonescimab combined with chemotherapy compared to placebo plus chemotherapy in patients with EGFR-mutated, locally advanced or metastatic non-squamous NSCLC who have progressed after treatment with an EGFR TKI.

HARMONi-2 is a Phase III clinical trial evaluating monotherapy ivonescimab against monotherapy pembrolizumab in patients with locally advanced or metastatic NSCLC whose tumors have positive PD-L1 expression (PD-L1 TPS >1%).

Ivonescimab is an investigational therapy that is not approved by any regulatory authority in Summit’s license territories, including the United States and Europe. Ivonescimab was approved for marketing authorization in China in May 2024.