New study results reinforce TAGRISSO® (osimertinib) as the backbone therapy for EGFR-mutated lung cancer across stages and settings

On March 25, 2025 New study results reported at the European Lung Cancer Congress (ELCC) 2025, March 26 to 29, demonstrate the role of AstraZeneca’s TAGRISSO (osimertinib), as monotherapy and as the backbone for novel combinations, across stages and settings of epidermal growth factor receptor-mutated (EGFRm) non-small cell lung cancer (NSCLC) (Press release, AstraZeneca, MAR 25, 2025, View Source [SID1234651434]). Highlights include:

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LAURA Phase III trial of TAGRISSO in unresectable, Stage III EGFRm NSCLC after chemoradiotherapy (CRT) (LBA4).
SAVANNAH Phase II trial of TAGRISSO plus savolitinib in advanced EGFRm NSCLC with high levels of MET overexpression and/or amplification following disease progression on 1st-line TAGRISSO (#2O).
ORCHARD Phase II platform trial of TAGRISSO plus datopotamab deruxtecan-dlnk in advanced EGFRm NSCLC following disease progression on 1st-line TAGRISSO (#1O).
FLAURA2 Phase III trial of TAGRISSO plus chemotherapy as 1st-line treatment for advanced EGFRm NSCLC (#53P).
Myung-Ju Ahn, MD, PhD, Professor of Hemato-Oncology at the Department of Medicine, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, South Korea, said: "A critical goal in treating every patient with lung cancer is to not only extend a patient’s life but also maintain quality of life while on treatment. The continued overall survival trend seen here at ELCC in the unresectable Stage III setting and the promising data for combinations that can address progression in the advanced setting, together reinforce osimertinib as an effective, safe and convenient treatment for patients with EGFR-mutated lung cancer across stages and lines of treatment."

Susan Galbraith, Executive Vice President, Oncology Hematology R&D, AstraZeneca, said: "Having now treated more than one million patients around the world, TAGRISSO has repeatedly transformed expectations for patients with EGFR-mutated lung cancer by not only extending survival but also showing it is possible to maintain quality of life during cancer treatment. The breadth of data at ELCC reinforce TAGRISSO as the backbone therapy for patients with this disease and show that adding savolitinib or datopotamab deruxtecan-dlnk at the time of disease progression can help prolong patients’ responses to treatment."

Professor Virginia Harrison, EGFR Positive UK, said: "For people undergoing treatment for lung cancer, maintaining quality of life is so important, but it can be challenging. The development of simple and effective treatment regimens that minimize negative side effects can make a huge difference. We are excited to see this progress for patients with EGFR-mutated lung cancer, where there is still significant unmet need."

TAGRISSO continued to demonstrate encouraging overall survival (OS) trend in unresectable, Stage III setting in LAURA trial
Updated results from the LAURA Phase III trial showed an improved trend towards OS benefit with TAGRISSO compared to placebo in patients with unresectable, Stage III EGFRm NSCLC (hazard ratio [HR] 0.67; 95% confidence interval [CI] 0.40, 1.14, maturity 31%). Median OS was 58.8 months (95% CI 54.1, not calculable [NC]) in patients treated with TAGRISSO versus 54.1 months with placebo (95% CI 42.1, NC), despite 78% of patients on placebo receiving subsequent treatment with TAGRISSO upon progression. The trial will continue to assess OS as a key secondary endpoint at the final analysis.

TAGRISSO previously demonstrated a statistically significant and highly clinically meaningful improvement in progression-free survival (PFS). These results were published in The New England Journal of Medicine and formed the basis for regulatory approvals around the world including in the US, EU and China.

Safety results and discontinuation rates due to adverse events (AEs) were as expected and no new safety concerns were identified.

TAGRISSO plus savolitinib showed durable response rates in lung cancer patients with high levels of MET overexpression and/or amplification whose disease progressed on 1st-line TAGRISSO in SAVANNAH trial
Results from the SAVANNAH Phase II trial showed TAGRISSO plus savolitinib (300 mg twice-daily [BID]) demonstrated a clinically meaningful and durable objective response rate (ORR) in patients with EGFRm NSCLC with high levels of MET overexpression and/or amplification whose disease progressed on treatment with 1st-line TAGRISSO. Among patients screened for enrollment in SAVANNAH, an estimated 62% had tumors with MET overexpression and/or amplification, and approximately 34% met the defined high MET level cut-off.

TAGRISSO plus savolitinib demonstrated a confirmed ORR of 56% (95% CI 45-67%), with a median duration of response (DoR) of 7.1 months (95% CI 5.6-9.6). Median PFS (mPFS) was 7.4 months (95% CI 5.5-7.6).

Safety results and discontinuation rates due to AEs were consistent with the established profiles of each medicine and no new safety concerns were reported. In all patients treated with TAGRISSO plus savolitinib (300 mg BID), Grade 3 or higher AEs occurred in 57% of patients.

Savolitinib is an oral, potent and highly selective MET tyrosine kinase inhibitor (TKI) being jointly developed and commercialized by AstraZeneca and HUTCHMED. In 2023, TAGRISSO plus savolitinib received Fast Track designation from the Food and Drug Administration (FDA) in this setting.

TAGRISSO plus datopotamab deruxtecan-dlnk showed encouraging response rates in patients whose disease progressed on 1st-line TAGRISSO in ORCHARD trial
First results from the TAGRISSO plus datopotamab deruxtecan-dlnk module of the ORCHARD Phase II platform trial showed the combination demonstrated promising efficacy and manageable safety in patients with advanced EGFRm NSCLC whose disease progressed on treatment with TAGRISSO.

The module enrolled an all-comer population of patients with EGFRm NSCLC and evaluated two doses of datopotamab deruxtecan-dlnk (4 or 6 mg/kg) which, in combination with TAGRISSO, showed similar ORRs of 43% (80% CI 31-55%) and 36% (80% CI 25-49%), respectively. PFS and DoR results favored the 6 mg/kg dose with a mPFS of 11.7 months (95% CI 8.3, NC) and 64% of patients continuing to respond at 9 months versus a mPFS of 9.5 months (95% CI 7.2-9.8) and 15% of patients continuing to respond at 9 months on the 4 mg/kg dose.

The safety profile of TAGRISSO plus datopotamab deruxtecan-dlnk was consistent with the known safety profiles of each medicine and no new safety concerns were identified at either dose level. Grade 3 or higher treatment-related AEs occurred in 34% and 56% of patients receiving the 4 mg/kg or 6 mg/kg doses, respectively.

Datopotamab deruxtecan-dlnk is a specifically engineered TROP2-directed antibody drug conjugate (ADC) discovered by Daiichi Sankyo and being jointly developed and commercialized by AstraZeneca and Daiichi Sankyo. The Companies are evaluating datopotamab deruxtecan-dlnk alone and with TAGRISSO as treatment for patients with advanced or metastatic EGFRm NSCLC in the TROPION-Lung14 and TROPION-Lung15 Phase III trials.

New results for TAGRISSO plus chemotherapy in the 1st-line setting reinforce strong PFS benefit in FLAURA2 Phase III trial
An exploratory post-hoc analysis of the FLAURA2 Phase III trial assessed PFS by length of exposure to pemetrexed maintenance treatment in patients with locally advanced (Stage IIIB-IIIC) or metastatic (Stage IV) EGFRm NSCLC. Patients were treated with TAGRISSO with the addition of chemotherapy (pemetrexed plus cisplatin) followed by maintenance treatment with TAGRISSO and chemotherapy (pemetrexed) or TAGRISSO alone. Results showed an mPFS of more than two years regardless of length of pemetrexed maintenance exposure, with a trend associating longer PFS with longer pemetrexed treatment.

The safety profile of TAGRISSO plus chemotherapy was consistent with the established profiles of the individual medicines. Grade 3 or higher chemotherapy-related AEs were reported in 16% of patients who received maintenance treatment for 3 to less than 9 months, and 10% for patients who received maintenance for 9 or more months. Chemotherapy discontinuation rates due to AEs were 18% and 10%, respectively.

TAGRISSO plus chemotherapy previously demonstrated a statistically significant and clinically meaningful improvement in PFS. These results were published in The New England Journal of Medicine and formed the basis for regulatory approvals around the world including in the US, EU, Japan and China. Previously presented OS data from the second interim analysis showed a favorable trend with the TAGRISSO plus chemotherapy arm (HR 0.75; 95% CI 0.57-0.97, maturity 41%), with consistent results across all prespecified subgroups. The trial will continue to assess OS as a key secondary endpoint.

IMPORTANT SAFETY INFORMATION

There are no contraindications for TAGRISSO
TAGRISSO can cause severe and fatal interstitial lung disease (ILD)/pneumonitis. ILD/pneumonitis occurred in 4% of the 1813 patients treated with TAGRISSO monotherapy who had not received recent definitive chemoradiation therapy; 0.4% of cases were fatal
ILD/Pneumonitis with TAGRISSO in combination with Pemetrexed and Platinum-based Chemotherapy:
In the FLAURA2 study, ILD/pneumonitis occurred in 3.3% of the 276 patients who received TAGRISSO in combination with pemetrexed and platinum-based chemotherapy; 0.4% of cases were fatal
ILD/Pneumonitis Following Definitive Platinum-based Chemoradiation Therapy (CRT):

In the LAURA study, following definitive platinum-based CRT, ILD/pneumonitis including radiation pneumonitis, occurred in 80 of the 143 patients (56%) who received TAGRISSO monotherapy and 28 of the 73 patients (38%) who received placebo. There was one fatal case (0.7%), 3.5% Grade 3, 34% Grade 2, and 18% Grade 1 adverse reactions of ILD/pneumonitis in TAGRISSO-treated patients. For TAGRISSO-treated patients, ILD/pneumonitis led to permanent discontinuation of TAGRISSO in 7% of patients and dosage interruptions of TAGRISSO in 35% of patients. Among the 46 patients who were rechallenged with TAGRISSO, 11% had recurrence of ILD/pneumonitis. In the 80 TAGRISSO-treated patients, ILD/pneumonitis resolved in 40%, resolved with sequelae in 1.3%, were resolving in 16%, did not resolve in 41%, and resulted in death in 1.3%
For patients receiving TAGRISSO who have not received recent definitive platinum-based CRT, withhold TAGRISSO and promptly investigate for ILD in patients who present with worsening of respiratory symptoms which may be indicative of ILD (e.g., dyspnea, cough, and fever). Permanently discontinue TAGRISSO if ILD/pneumonitis is confirmed. For patients who have received recent definitive platinum-based CRT with Grade 1 ILD/pneumonitis, continue TAGRISSO or interrupt and restart, as appropriate. Permanently discontinue TAGRISSO in patients diagnosed with Grade ≥2 ILD/pneumonitis

TAGRISSO can cause heart rate-corrected QT (QTc) interval prolongation. Of the 1813 TAGRISSO monotherapy-treated patients in clinical trials, 1.1% were found to have a QTc >500 msec, and 4.3% of patients had an increase from baseline QTc >60 msec. Of the 276 patients treated with TAGRISSO in combination with pemetrexed and platinum-based chemotherapy in the FLAURA2 study, 1.8% were found to have a QTc >500 msec, and 10.5% of patients had an increase from baseline QTc >60 msec. No QTc-related arrhythmias were reported. Clinical trials of TAGRISSO did not enroll patients with baseline QTc of >470 msec. Conduct periodic monitoring with ECGs and electrolytes in patients with congenital long QTc syndrome, congestive heart failure, electrolyte abnormalities, or those who are taking medications known to prolong the QTc interval. Permanently discontinue TAGRISSO in patients who develop QTc interval prolongation with signs/symptoms of life-threatening arrhythmia
TAGRISSO can cause cardiomyopathy, including cardiac failure, chronic cardiac failure, congestive heart failure, pulmonary edema or decreased ejection fraction. Cardiomyopathy occurred in 3.8% of the 1813 TAGRISSO-treated patients; 0.1% of cardiomyopathy cases were fatal. In the FLAURA2 study, cardiomyopathy occurred in 9% of the 276 patients who received TAGRISSO in combination with pemetrexed and platinum-based chemotherapy; 1.1% of cardiomyopathy cases were fatal. A decline in left ventricular ejection fraction (LVEF) ≥10% from baseline and to <50% LVEF occurred in 4.2% of 1557 patients who had baseline and at least one follow-up LVEF assessment. In the ADAURA study, 1.5% (5/325) of TAGRISSO-treated patients experienced LVEF decreases ≥10% from baseline and a drop to <50%. In the LAURA study, following platinum-based CRT, 3% (4/135) of TAGRISSO-treated patients and no placebo-treated patients experienced LVEF decreases ≥10% and a drop to <50%. In the FLAURA2 study, 8% (21/262) of patients treated with TAGRISSO in combination with pemetrexed and platinum-based chemotherapy, who had baseline and at least one follow-up LVEF assessment, experienced LVEF decreases ≥10% and a drop to <50%. For patients receiving TAGRISSO monotherapy, conduct cardiac monitoring in patients with cardiac risk factors, including assessment of LVEF at baseline and during treatment. For patients receiving TAGRISSO in combination with pemetrexed and platinum-based chemotherapy, conduct cardiac monitoring in all patients, including assessment of LVEF at baseline and during treatment. Assess LVEF in patients who develop relevant cardiac signs or symptoms during treatment. For symptomatic congestive heart failure, permanently discontinue TAGRISSO
Keratitis was reported in 0.6% of 1813 patients treated with TAGRISSO monotherapy in clinical trials. Promptly refer patients with signs and symptoms suggestive of keratitis (such as eye inflammation, lacrimation, light sensitivity, blurred vision, eye pain and/or red eye) to an ophthalmologist
Postmarketing cases consistent with erythema multiforme major (EMM), Stevens-Johnson syndrome (SJS) and toxic epidermal necrolysis (TEN) have been reported in patients receiving TAGRISSO. Withhold TAGRISSO if EMM, SJS, or TEN is suspected and permanently discontinue if confirmed
Postmarketing cases of cutaneous vasculitis including leukocytoclastic vasculitis, urticarial vasculitis, and IgA vasculitis have been reported in patients receiving TAGRISSO. Withhold TAGRISSO if cutaneous vasculitis is suspected, evaluate for systemic involvement, and consider dermatology consultation. If no other etiology can be identified, consider permanent discontinuation of TAGRISSO based on severity
Aplastic anemia has been reported in TAGRISSO-treated patients in clinical trials (0.06% of 1813) and postmarketing. Some cases had a fatal outcome. Inform patients of the signs and symptoms of aplastic anemia including but not limited to, new or persistent fevers, bruising, bleeding, and pallor. If aplastic anemia is suspected, withhold TAGRISSO and obtain a hematology consultation. If aplastic anemia is confirmed, permanently discontinue TAGRISSO. Perform complete blood count with differential before starting TAGRISSO, periodically throughout treatment, and more frequently if indicated
Verify pregnancy status of females of reproductive potential prior to initiating TAGRISSO. Advise pregnant women of the potential risk to a fetus. Advise females of reproductive potential to use effective contraception during treatment with TAGRISSO and for 6 weeks after the last dose. Advise males with female partners of reproductive potential to use effective contraception for 4 months after the last dose
Because of the potential for serious adverse reactions in breastfed infants from TAGRISSO, women should not breastfeed during treatment with TAGRISSO and for 2 weeks after the last dose
Most common (≥20%) adverse reactions, including laboratory abnormalities, were:
TAGRISSO monotherapy: leukopenia, lymphopenia, thrombocytopenia, anemia, diarrhea, rash, musculoskeletal pain, neutropenia, nail toxicity, dry skin, stomatitis, and fatigue
TAGRISSO monotherapy following platinum-based chemoradiation therapy: lymphopenia, leukopenia, ILD/pneumonitis, thrombocytopenia, neutropenia, rash, diarrhea, nail toxicity, musculoskeletal pain, cough and COVID-19
TAGRISSO in combination with pemetrexed and platinum-based chemotherapy: leukopenia, thrombocytopenia, neutropenia, lymphopenia, rash, diarrhea, stomatitis, nail toxicity, dry skin, and increased blood creatinine
INDICATIONS

TAGRISSO is indicated as adjuvant therapy after tumor resection in adult patients with non-small cell lung cancer (NSCLC) whose tumors have epidermal growth factor receptor (EGFR) exon 19 deletions or exon 21 L858R mutations, as detected by an FDA-approved test
TAGRISSO is indicated for the treatment of adult patients with locally advanced, unresectable (stage III) NSCLC whose disease has not progressed during or following concurrent or sequential platinum-based chemoradiation therapy and whose tumors have epidermal growth factor receptor (EGFR) exon 19 deletions or exon 21 L858R mutations, as detected by an FDA-approved test
TAGRISSO is indicated for the first-line treatment of adult patients with metastatic non-small cell lung cancer (NSCLC) whose tumors have epidermal growth factor receptor (EGFR) exon 19 deletions or exon 21 L858R mutations, as detected by an FDA-approved test
TAGRISSO is indicated in combination with pemetrexed and platinum-based chemotherapy, for the first-line treatment of adult patients with locally advanced or metastatic NSCLC whose tumors have epidermal growth factor receptor (EGFR) exon 19 deletions or exon 21 L858R mutations, as detected by an FDA-approved test
TAGRISSO is indicated for the treatment of adult patients with metastatic epidermal growth factor receptor (EGFR) T790M mutation-positive NSCLC, as detected by an FDA-approved test, whose disease has progressed on or after EGFR tyrosine kinase inhibitor (TKI) therapy
Notes
NSCLC
Lung cancer is the leading cause of cancer death among men and women, accounting for about one-fifth of all cancer deaths.1 Lung cancer is broadly split into small cell lung cancer or NSCLC, the latter accounting for about 80% of cases.2 Approximately 10 to 15% of patients with NSCLC in the US and Europe and 30 to 40% of patients in Asia have an EGFR mutation.3-5

While EGFR-TKIs have significantly improved outcomes in the 1st-line setting, mechanisms of resistance and disease progression are extremely common, and a significant unmet need exists in later-line settings for effective and well-tolerated treatment options.6-9

LAURA
LAURA is a randomized, double-blind, placebo-controlled, multi-center, global Phase III trial in patients with unresectable, Stage III EGFRm NSCLC whose disease has not progressed following definitive platinum-based CRT. Patients were treated with TAGRISSO 80 mg once-daily (QD) oral tablets until disease progression, unacceptable toxicity or other discontinuation criteria were met. Upon progression, patients in the placebo arm were offered treatment with TAGRISSO.

The trial enrolled 216 patients in more than 145 centers across more than 15 countries, including in the US, Europe, South America and Asia.

SAVANNAH
SAVANNAH is an ongoing randomized, global Phase II trial studying the efficacy of savolitinib added to TAGRISSO in patients with EGFRm, locally advanced or metastatic NSCLC with MET overexpression and/or amplification whose disease progressed following treatment with TAGRISSO. Based on the original single-arm trial design, patients were treated with savolitinib 300 or 600 mg QD or 300 mg BID, in combination with oral TAGRISSO 80 mg QD. In 2022, a comparison of savolitinib 300 mg BID and TAGRISSO 80 mg QD to savolitinib 300 mg BID and placebo was added to the trial to evaluate contribution of components.

The trial has enrolled 369 patients to date in more than 80 centers globally, including in the US, Canada, Europe, South America and Asia. The primary endpoint is ORR and key secondary endpoints include PFS and DoR.

In August 2022, positive interim ORR results from the SAVANNAH trial were presented at the International Association for the Study of Lung Cancer 2022 World Conference on Lung Cancer (WCLC).

The global SAFFRON Phase III trial will further assess the TAGRISSO plus savolitinib combination versus platinum-based doublet chemotherapy in patients with EGFRm, MET-overexpressed and/or amplified, locally advanced or metastatic NSCLC following treatment with TAGRISSO. Patients are being prospectively selected using the high MET level cut-off identified in SAVANNAH.

ORCHARD
ORCHARD (Osimertinib Resistance CoHorts, Addressing 1L Relapse Drivers) is an open-label, multi-center Phase II platform trial evaluating numerous TAGRISSO-based combinations as a treatment for patients with advanced EGFRm NSCLC whose disease has progressed on 1st-line TAGRISSO. The trial has 10 modules examining the efficacy, safety and tolerability of these targeted and non-targeted combination options, including TAGRISSO 80 mg QD plus datopotamab deruxtecan-dlnk given intravenously at 4 or 6 mg/kg on day one of every three-week cycle.

The trial has enrolled 247 patients to date in more than 40 centers globally, including in the US, Europe and Asia. The primary endpoint is ORR. Key secondary endpoints include PFS and DoR.

FLAURA2
FLAURA2 is a randomized, open-label, multi-center, global Phase III trial in the 1st-line treatment of patients with locally advanced (Stage IIIB-IIIC) or metastatic (Stage IV) EGFRm NSCLC. Patients were treated with TAGRISSO 80 mg QD oral tablets with the addition of chemotherapy (pemetrexed (500 mg/m2) plus cisplatin (75 mg/m2) or carboplatin (AUC5) every three weeks for four cycles, followed by TAGRISSO with pemetrexed maintenance every three weeks.

The trial enrolled 557 patients in more than 150 centers across more than 20 countries, including in the US, Europe, South America and Asia. The primary endpoint is PFS. The trial is ongoing and will continue to assess the secondary endpoint of OS.

TAGRISSO
TAGRISSO (osimertinib) is a third-generation, irreversible EGFR-TKI with proven clinical activity in NSCLC, including against central nervous system metastases. TAGRISSO (40 mg and 80 mg QD oral tablets) has been used to treat patients across its indications worldwide and AstraZeneca continues to explore TAGRISSO as a treatment for patients across multiple stages of EGFRm NSCLC.

There is an extensive body of evidence supporting the use of TAGRISSO in EGFRm NSCLC. TAGRISSO is the only targeted therapy to improve patient outcomes in early-stage disease in the NeoADAURA and ADAURA Phase III trials, locally advanced stages in the LAURA Phase III trial and late-stage disease in the FLAURA and FLAURA2 Phase III trials.

As part of AstraZeneca’s ongoing commitment to treating patients as early as possible in lung cancer, TAGRISSO is also being investigated in the early-stage adjuvant resectable setting in the ADAURA2 Phase III trial.

Savolitinib
Savolitinib is an oral, potent, and highly selective MET TKI that has demonstrated clinical activity in advanced solid tumors. MET is a tyrosine kinase receptor that has an essential role in normal cell development. Savolitinib blocks atypical activation of the MET receptor tyrosine kinase pathway that occurs because of mutations (such as exon 14 skipping alterations or other point mutations), gene amplification or protein overexpression. MET overexpression and/or amplification can lead to tumor growth and the metastatic progression of cancer cells, and is a known mechanism of acquired resistance to EGFR TKIs. The prevalence of MET depends on the sample type, detection method and assay cut-off used.

Savolitinib is approved in China for the treatment of adult patients with locally advanced or metastatic NSCLC with MET exon 14 skipping alterations.

It is currently under clinical development for multiple tumor types, including lung, kidney and gastric cancers as a single treatment and in combination with other medicines.

Datopotamab deruxtecan-dlnk
Datopotamab deruxtecan-dlnk (datopotamab deruxtecan in rest of world) is a TROP2-directed ADC. Designed using Daiichi Sankyo’s proprietary DXd ADC Technology, datopotamab deruxtecan-dlnk is one of six DXd ADCs in the oncology pipeline of Daiichi Sankyo, and one of the most advanced programs in AstraZeneca’s ADC scientific platform. Datopotamab deruxtecan-dlnk is comprised of a humanized anti-TROP2 IgG1 monoclonal antibody, developed in collaboration with Sapporo Medical University, attached to a number of topoisomerase I inhibitor payloads (an exatecan derivative, DXd) via tetrapeptide-based cleavable linkers.

Datopotamab deruxtecan-dlnk (6 mg/kg) is approved in the US, Japan and Russia for the treatment of adult patients with unresectable or metastatic HR-positive, HER2-negative (IHC 0, IHC 1+ or IHC 2+/ISH-) breast cancer who have received prior endocrine-based therapy and chemotherapy for unresectable or metastatic disease based on the results from the TROPION-Breast01 Phase III trial.

In the US, a Biologics License Application for datopotamab deruxtecan-dlnk is under Priority Review for the treatment of adult patients with locally advanced or metastatic EGFRm NSCLC who have received prior systemic therapies, including an EGFR-directed therapy. Datopotamab deruxtecan-dlnk was also granted Breakthrough Therapy Designation by the FDA for this patient population.

Anixa Biosciences Enters Letter of Intent with VERDI Solutions to Develop Artificial Intelligence-Guided Personalized and Off-the-Shelf Cancer Vaccines

On March 25, 2025 Anixa Biosciences, Inc. ("Anixa" or the "Company") (NASDAQ: ANIX), a biotechnology company focused on the treatment and prevention of cancer, reported that it has entered into a letter of intent with VERDI Solutions, GmbH to develop personalized and off-the-shelf peptide vaccines for cancer patients (Press release, Anixa Biosciences, MAR 25, 2025, View Source [SID1234651433]).

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VERDI Solutions, headquartered in Vienna, Austria, is pioneering personalized cancer treatment by integrating artificial intelligence (AI) and advanced cloud computing to accelerate the development and implementation of personalized cancer vaccines. By combining computerized vaccine design with real-world data collection and clinical trials, VERDI is making personalized cancer vaccines accessible to more patients worldwide. At the core of VERDI’s innovation is its cloud-computing platform, which enables the rapid development of personalized peptide vaccines tailored to individual cancer patients. Additionally, the company is developing off-the-shelf cancer vaccines, paired with companion diagnostics to identify likely responders and optimize treatment outcomes.

In some European countries, oncologists may administer personalized cancer vaccines under Individueller Heilversuch (Individual Healing Attempt), which allows physicians to prescribe individualized treatments for patients with life-threatening conditions who lack satisfactory therapeutic alternatives. Unlike clinical trials, where only a limited number of eligible patients can be enrolled, Heilversuch offers an innovative treatment option for any cancer patient, potentially providing valuable insights into the efficacy and safety of personalized vaccines in real-world settings. Moreover, Heilversuch aligns with the increasing recognition by global regulatory agencies of real-world evidence (RWE) as a critical component of drug development. VERDI is pioneering a data-driven approach to accelerating regulatory validation and commercialization of personalized cancer vaccines.

VERDI has successfully developed personalized vaccines using sequence data from the primary tumors for three patients with bone metastases, which were administered by their oncologists under Heilversuch. These patients experienced clinical benefits, including significant pain relief caused by bone metastases and improved quality of life. While these outcomes highlight the potential of VERDI’s platform to scale personalized cancer care, further clinical validation is needed.

Under the letter of intent, VERDI has granted Anixa a six-month exclusive right to negotiate a transaction for the research, development, and commercialization of VERDI’s cancer vaccines. While VERDI continues to develop personalized vaccines for individual treatment attempts in Europe, VERDI and Anixa plan to initiate clinical trials in the United States.

"We utilize advanced cloud computing to develop a set of personalized VERDI vaccines for each patient from their sequence data. Our proprietary algorithm predicts the efficacy of the vaccines to recognize and eliminate tumor cells in the individual patient. Our unique approach models the complex mechanisms of tumor-specific immune responses at an individual level, potentially enabling faster and more effective personalized vaccine development using state-of-the-art science compared to existing neoantigen-based cancer vaccines. Our initial results in three patients are highly encouraging, and we are eager to demonstrate broader clinical benefits in additional patients," stated Dr. Julianna Lisziewicz, Founder and CEO of VERDI.

Dr. Amit Kumar, Chairman and CEO of Anixa stated, "We are very pleased to be working with VERDI Solutions on this exciting technology that has already demonstrated benefit to terminally ill patients. The letter of intent with VERDI provides us with the optionality to collaborate on the further development of VERDI’s technology, while preserving our ability to structure the relationship in a way that is mutually beneficial to both parties. Additionally, partnering with VERDI aligns with our strategy of implementing a capital-efficient business model for personalized cancer care."

Nuvalent to Present New Preclinical Data on ALK-Selective Inhibitor, Neladalkib, and ROS1-Selective Inhibitor, Zidesamtinib, at AACR Annual Meeting 2025

On March 25, 2025 Nuvalent, Inc. (Nasdaq: NUVL), a clinical-stage biopharmaceutical company focused on creating precisely targeted therapies for clinically proven kinase targets in cancer, reported upcoming poster presentations further characterizing the preclinical profiles of its novel ALK-selective inhibitor, neladalkib, and novel ROS1-selective inhibitor, zidesamtinib, at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2025 from April 25-30, 2025, in Chicago (Press release, Nuvalent, MAR 25, 2025, View Source [SID1234651432]).

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Details of the poster presentations are as follows:

Title: Mutagenesis screens support potential best-in-class profile for neladalkib (NVL-655), a brain-penetrant and TRK-sparing ALK inhibitor
Authors: Anupong Tangpeerachaikul*1, Henry E. Pelish1
Abstract Number: 1729
Session Category: Experimental and Molecular Therapeutics
Session Title: Kinase and Phosphatase Inhibitors 1
Session Date and Time: April 28, 2025, from 9:00 a.m.– 12:00 p.m. CT
Location: Poster Section 21
Poster Board Number: 4

Title: Crystal structure of drug-resistant ROS1 G2032R in complex with zidesamtinib, a clinical-stage ROS1 inhibitor with best-in-class potential
Authors: Joseph M. Magrino*1, Anupong Tangpeerachaikul1, Scot Mente1, Henry E. Pelish1
Abstract Number: 5616
Session Category: Experimental and Molecular Therapeutics
Session Title: Kinase and Phosphatase Inhibitors 3
Session Date and Time: April 29, 2025, from 2:00 p.m.– 5:00 p.m. CT
Location: Poster Section 20
Poster Board Number: 26

*Presenter, corresponding author; 1Nuvalent, Inc., Cambridge, MA, USA

About Zidesamtinib
Zidesamtinib is a novel brain-penetrant ROS1-selective inhibitor created with the aim to overcome limitations observed with currently available ROS1 inhibitors. Zidesamtinib is designed to remain active in tumors that have developed resistance to currently available ROS1 inhibitors, including tumors with treatment-emergent ROS1 mutations such as G2032R. In addition, zidesamtinib is designed for central nervous system (CNS) penetrance to improve treatment options for patients with brain metastases, and to avoid inhibition of the structurally related tropomyosin receptor kinase (TRK) family. Together, these characteristics have the potential to avoid TRK-related CNS adverse events seen with dual TRK/ROS1 inhibitors and to drive deep, durable responses for patients across all lines of therapy. Zidesamtinib has received breakthrough therapy designation for the treatment of patients with ROS1-positive metastatic non-small cell lung cancer (NSCLC) who have been previously treated with 2 or more ROS1 tyrosine kinase inhibitors and orphan drug designation for ROS1-positive NSCLC.

About Neladalkib
Neladalkib is a novel brain-penetrant ALK-selective inhibitor created with the aim to overcome limitations observed with currently available ALK inhibitors. Neladalkib is designed to remain active in tumors that have developed resistance to first-, second-, and third-generation ALK inhibitors, including tumors with single or compound treatment-emergent ALK mutations such as G1202R. In addition, neladalkib is designed for central nervous system (CNS) penetrance to improve treatment options for patients with brain metastases, and to avoid inhibition of the structurally related tropomyosin receptor kinase (TRK) family. Together, these characteristics have the potential to avoid TRK-related CNS adverse events seen with dual TRK/ALK inhibitors and to drive deep, durable responses for patients across all lines of therapy. Neladalkib has received breakthrough therapy designation for the treatment of patients with locally advanced or metastatic ALK-positive non-small cell lung cancer (NSCLC) who have been previously treated with 2 or more ALK tyrosine kinase inhibitors and orphan drug designation for ALK-positive NSCLC.

ADC Therapeutics Announces Abstracts Accepted for Presentation at the American Association for Cancer Research Annual Meeting 2025

On March 25, 2025 ADC Therapeutics SA (NYSE: ADCT), a commercial-stage global leader and pioneer in the field of antibody drug conjugates (ADCs), reported abstracts detailing multiple preclinical programs have been accepted for presentation at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2025, taking place April 25-30, 2025, in Chicago, Illinois (Press release, ADC Therapeutics, MAR 25, 2025, View Source [SID1234651431]).

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"We are excited to present preclinical data on our exatecan-based Claudin-6, PSMA, and ACST2-targeting antibody-drug conjugates," said Patrick van Berkel, PhD, Chief Scientific Officer of ADC Therapeutics. "These ADCs hold promise for targeted cancer treatment in a broad range of cancer types, and we are pleased to have the opportunity to share our learnings across select solid tumors where there remains unmet need."

Details of ADC Therapeutics’ oral presentation at AACR (Free AACR Whitepaper) are as follows:

Title: Preclinical investigation of ADCT-242, a novel exatecan-based antibody drug conjugate targeting Claudin-6, as single agent or in combination in ovarian and non-small lung cancer models
Abstract: 1163
Session Category: Experimental and Molecular Therapeutics
Session Title: Antibody-Based Cancer Therapeutic Agents
Date and Time: Sunday, April 27, 2025, 3:00-5:00 p.m. CT
Presenter: Chris Pickford, Head of Clinical Research, ADC Therapeutics

Details of ADC Therapeutics’ poster presentations at AACR (Free AACR Whitepaper) are as follows:

Title: Preclinical Development of ADCT-241, a Novel Exatecan-based Antibody-Drug Conjugate Targeting PSMA for the Treatment of Prostate Cancer
Abstract: 6736
Session Category: Experimental and Molecular Therapeutics
Session Title: Antibody-Based Cancer Therapeutics 4
Date and Time: Wednesday, April 30, 2025, 9:00 a.m. – 12:00 p.m. CT
Presenter: Ben Leatherdale, Senior Scientist, ADC Therapeutics

Title: HuB14-VA-PL2202, a novel antibody-drug conjugate targeting ASCT2, a novel ADC target over-expressed in both solid and hematological cancers
Abstract: 1580
Session Category: Experimental and Molecular Therapeutics
Session Title: Antibody-Based Cancer Therapeutics 1
Date and Time: Monday, April 28, 2025, 9:00 a.m. – 12:00 p.m. CT
Presenter: Danilo Cucchi, Senior Scientist, ADC Therapeutics

Adcentrx Therapeutics to Showcase Novel STEAP1 and NaPi2b ADCs with Oral and Poster Presentations at AACR 2025

On March 25, 2025 Adcentrx Therapeutics ("Adcentrx"), a clinical-stage biotechnology company redefining Antibody-Drug Conjugate (ADC) therapies for cancer treatment and other life-threatening diseases, reported it will present new data at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2025, (April 25-30, 2025) in Chicago, IL (Press release, Adcentrx Therapeutics, MAR 25, 2025, View Source [SID1234651430]).

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The company will deliver an oral presentation on ADRX-0405, its clinical-stage STEAP1 ADC, and a poster presentation on ADRX-0134, its preclinical NaPi2b ADC. The presentations will underscore the versatility of Adcentrx’s ADC platform, showing advancements in therapeutic window expansion, bystander effect optimization, and payload delivery improvements for targeted cancer therapy. These innovations are powered by Adcentrx’s proprietary i-Conjugation technology, which integrates stable conjugation chemistry and a cleavable linker to optimize ADC properties, ensuring optimal payload delivery and therapeutic efficacy.

ADRX-0405, Adcentrx’s second clinical program and potential first-in-class STEAP1 ADC, has the opportunity to expand treatment options for metastatic castration-resistant prostate cancer (mCRPC) and other STEAP1-expressing tumors, where targeted therapy options remain limited. Featuring a novel topoisomerase inhibitor linker-payload, ADRX-0405 represents a distinct therapeutic approach for this disease with a high unmet need for more tolerable and effective therapies.

ADRX-0134 is a preclinical NaPi2b ADC incorporating AP052, the same clinically validated microtubule inhibitor payload used in Adcentrx’s lead program, ADRX-0706 (Nectin-4 ADC), currently enrolling patients in Phase 1b (NCT06036121). ADRX-0134 offers a differentiated approach for patients with lung and ovarian cancers, where few viable treatment options are available.

Details of the oral and poster presentations are as follows:

Oral Presentation
Title: Preclinical characterization of a novel STEAP1 antibody-drug conjugate ADRX-0405 for the treatment of mCRPC
Abstract Number: 1159
Session Date & Time: Sunday, April 27, 3:25 p.m. – 3:40 p.m. CST
Session Title: Antibody-Based Cancer Therapeutic Agents

Poster Presentation
Title: ADRX-0134 as a novel auristatin-based NaPi2b antibody-drug conjugate with widened therapeutic window
Abstract Number: 1563
Session Date & Time: Monday, April 28, 9:00 a.m. – 12:00 p.m. CST
Session Title: Antibody-Based Cancer Therapeutics 1

About i-Conjugation Technology
Adcentrx’s proprietary i-Conjugation technology platform is an important component in the design of the company’s ADCs. The platform utilizes protease-cleavable linkers and stable conjugation chemistry to enhance payload delivery. This advanced technology ensures a highly stable ADC with the desired linker-payload.

About ADRX-0405
ADRX-0405 is a clinical-stage next-generation ADC targeting six-transmembrane epithelial antigen of the prostate 1 (STEAP1), a cell surface protein that is upregulated in prostate cancer and certain other cancers with limited expression in normal healthy tissue. The ADC is composed of a humanized IgG1 antibody and novel topoisomerase inhibitor linker-payload conjugated at a drug-to-antibody ratio of eight (DAR 8) to maximize payload delivery to solid tumors. ADRX-0405 preclinical studies have demonstrated its favorable pharmacokinetics, safety profile, and significant efficacy across multiple animal tumor models. ADRX-0405 is currently being evaluated in a Phase 1a/b clinical trial.

For more information about the ADRX-0405 Phase 1a/b clinical trial, please refer to the Study ID NCT06710379 on ClinicalTrials.gov.

About ADRX-0134
ADRX-0134 is a state-of-the-art preclinical ADC targeting NaPi2b, a cell surface sodium-dependent phosphate transporter expressed in lung and ovarian cancers with minimal expression in normal healthy tissues. The ADC is a human IgG1 antibody conjugated at DAR8 with Adcentrx’s clinically validated AP052 tubulin inhibitor payload, substantially expanding the therapeutic window of auristatin-based ADCs beyond existing vedotin technology. ADRX-0134 preclinical studies have demonstrated strong efficacy in lung and ovarian tumor models, and the ADC’s pharmacokinetics and safety profile are also favorable.