On April 16, 2021 IDEAYA Biosciences, Inc. (NASDAQ: IDYA), a synthetic lethality-focused precision medicine oncology company committed to the discovery and development of targeted therapeutics, reported clinical data from the ongoing Phase 1/2 trial evaluating darovasertib (IDE196) monotherapy and binimetinib combination therapy in patients with solid tumors, including Metastatic Uveal Melanoma (MUM) and Skin Melanoma (ClinicalTrials.gov Identifier: NCT03947385) (Press release, Ideaya Biosciences, APR 16, 2021, View Source [SID1234578132]).

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"The darovasertib single-agent one-year survival data in MUM is encouraging and compares favorably to historical survival rates in this indication, where a therapy has yet to be approved," said Meredith McKean, MD, MPH, Associate Director, Melanoma and Skin Cancer Research, Sarah Cannon Research Institute at Tennessee Oncology, Nashville, TN. "The early partial responses observed in the darovasertib and binimetinib combination in MUM are exciting where historical response rates have been from zero to low to mid-single-digit percent, and we look forward to seeing the data set mature," said Richard Carvajal, MD, Co-Leader, Precision Oncology and Systems Biology Program, Director of Experimental Therapeutics and Director of the Melanoma Service, Columbia University Irving Medical Center.

Darovasertib Monotherapy Clinical Efficacy in Solid Tumors
There have been 81 darovasertib monotherapy BID MUM and 7 Skin Melanoma patients enrolled across the IDEAYA and Novartis Phase 1/2 clinical trials at the time of data and analyses cutoff on April 13th, 2021, with an aggregate of 88 patients evaluable for safety and 81 evaluable for efficacy based on RECIST 1.1. Reported data is preliminary and based on an unlocked database. Evaluation and follow-up of the monotherapy arm of the clinical trial continues.

Darovasertib Monotherapy Preliminary Results Summary

57% 1-Year Overall Survival (OS) Rate was observed in predominantly second line, third line and heavily pre-treated (out to 7 and 8 lines of prior treatment) Metastatic Uveal Melanoma (MUM) patients with 95% CI (44%, 69%); Historical 1-Year OS Rate in similar MUM populations has been reported at 37% (Source: Rantala 2019, Immunocore March 2021 presentation, Synthetic Control Arm, 2+ L)
Median OS of 13.2 months was observed in predominanantly second line, third line and heavily pre-treated (out to 7 and 8 lines of prior treatment) MUM patients with 95% CI (10.7 months, Not Reached); Historical median OS in similar MUM populations has been reported at approximately 7 months (Source: Rantala 2019, Immunocore March 2021, Synthetic Control Arm, 2+ L)
61% (n=46) of MUM patients out of 75 evaluable had tumor reduction per RECIST 1.1. evaluation, including 15 patients (20%) with >30% target lesion reduction and one confirmed complete response. In the Skin Melanoma cohort, 80% (n=4) of evaluable patients (n=5) had tumor reduction per RECIST 1.1. evaluation, including one confirmed partial response
Darovasertib Monotherapy Clinical Safety
Overall safety profile of darovasertib monotherapy is consistent with prior reports (Ref. 2019 AACR (Free AACR Whitepaper)) and includes primarily common low grade but manageable GI toxicities and hypotension.

Preliminary Darovasertib and Binimetinib Combination Clinical Efficacy in MUM
The combination of darovasertib plus binimetinib is being evaluated pursuant to a clinical trial collaboration and drug supply agreement with Pfizer, which the companies have amended to support a target enrollment of approximately 40 patients in the darovasertib and binimetinib clinical combination arm in MUM. At the time of the data and analyses cutoff on April 13th, 2021, twenty four MUM patients have enrolled in the darovasertib and binimetinib combination study, including 8 patients dosed in the Phase 1/2 dose expansion cohort of the combination study. Reported data is preliminary and based on an unlocked database. Enrollment in the darovasertib and binimetinib combination arm of the clinical trial is ongoing.

Darovasertib and Binimetinib Combination Therapy Preliminary Data Summary

2 partial responses observed out of nine MUM patients with at least 2 post-baseline scans (22%) by RECIST 1.1 guidelines, including 1 confirmed partial response and 1 unconfirmed partial response (-40.5%) awaiting a confirmatory scan
79% of evaluable MUM patients with at least 1 post-baseline scan show tumor reduction; follow-up for overall response is still immature
Combination doses for Phase 1/2 dose expansion have been selected based on anticipation of activity and overall tolerability in a larger treatment cohort
Treatment-related adverse events observed in the darovasertib and binimetinib combination arm in MUM primarily include: nausea, vomiting, diarrhea, rash, edema, AST/ALT increase and CK increase (>10%); and hypotension (<10%)
IDEAYA’s clinical development strategy in MUM is focused on darovasertib combinations, including with binimetinib, a MEK inhibitor, and in a separate clinical study with crizotinib, a cMET inhibitor, each pursuant to the clinical trial collaboration and drug supply agreement with Pfizer.

Darovasertib Investor Day Webcast
IDEAYA will host an investor webcast with a presentation at 8:00 a.m. ET today. A link to the webcast and a copy of the presentation is posted on the Investor Relations Events section of the Company’s website at View Source The update may also be accessed by dialing 1-866-248-8441 (domestic) or 1-720-452-9102 (international) five minutes prior to the start of the call and providing the passcode 2793795. An archived replay of the webcast will be accessible for 90 days following the event.

Meredith McKean, MD, MPH, Associate Director, Melanoma and Skin Cancer Research, Sarah Cannon Research Institute at Tennessee Oncology, Nashville, TN, and Richard Carvajal, MD, Co-Leader, Precision Oncology and Systems Biology Program, Director of Experimental Therapeutics and Director of the Melanoma Service, Columbia University Irving Medical Center, will participate in the webcast.

On April 16, 2021 Peptron, Inc. ("Peptron", KOSDAQ: 087010), a South Korea-based biotech company, and Qilu Pharmaceutical ("Qilu"), a leading vertically integrated pharmaceutical company focused on discovering, developing, manufacturing and commercializing innovative medicines reported that the companies have entered into an exclusive licensing agreement for the manufacturing, development and commercialization of Peptron’s PAb001-ADC, the antibody-drug conjugate (the "ADC") product containing the anti-MUC1 monoclonal antibody PAb001 as effective of March 26, 2021 (Press release, Qilu Pharmaceutical, APR 16, 2021, View Source [SID1234578131]).

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Under the terms of the agreement, Peptron grants Qilu an exclusive global right and license to develop, manufacture, sell, and commercialize PAb001-ADC for the treatment of cancers.

PAb001-ADC is a pre-clinical ADC entering into Investigational New Drug ("IND") enabling studies. It targets MUC1, a high-potential ADC target for multiple solid and hematological malignancies. MUC1 is overexpressed in many cancers and is recognized as a promising molecular target for therapeutic development for various types of cancers.

"We are excited to collaborate with Qilu to achieve the global opportunity of this potentially differentiated PAb001-ADC developed by us," said Ho-il Choi, CEO of Peptron. "Qilu is one of the leading pharmaceutical companies with global expertise and capacity in innovative R&D, manufacturing and marketing in China."

"We are very pleased to establish the partnership with Peptron to obtain global rights to the asset with highly differentiated attributes," said Dr. Binhui (Ben) Ni, Chief Investment Officer and Chief Business Officer, Corporate Vice President of Qilu Pharmaceutical, "We look forward to working closely with Peptron and The National Medical Products Administration (NMPA) to bring it to the patients."

On April 16, 2021 AIkido Pharma Inc. (Nasdaq: AIKI) ("AIkido" or the "Company") reported that a recent study announced positive early results from a PSMA 617-Lu177 study conducted by a leading global pharmaceutical company (Press release, AIkido Pharma, APR 16, 2021, View Source [SID1234578130]). The report provides strong indications for analogous success in similar technology the Company is helping to develop.

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Anthony Hayes, CEO of AIkido Pharma, stated, "As a rising tide lifts all boats, these results indicate the strength of our prior investment in this technology field. These early reports indicate there is strong evidence that developing radioligand therapies have the potential to be an alternative cancer treatment and provide positive outcomes for patients with advanced prostate cancer. The Company’s stock price has been down recently, but we believe these positive results from the Novartis Phase III study provides an incremental proof point that reinforces our confidence in the radiopharmaceutical space, PSMA and our investment in this technology. With testing data for our technology expected in Q2 of this year, we are excited about the results and the advancement of this technology."

On April 16, 2021 Castle Biosciences, Inc. (Nasdaq: CSTL), a skin cancer diagnostics company providing personalized genomic information to improve cancer treatment decisions, reported that data on its three skin cancer gene expression profile tests at the 10th World Congress of Melanoma and 17th European Association of Dermato-Oncology (EADO) Congress (Press release, Castle Biosciences, APR 16, 2021, View Source [SID1234578128]).

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DecisionDx-Melanoma:

DecisionDx-Melanoma is Castle’s gene expression profile test that uses an individual patient’s tumor biology to predict risk of cutaneous melanoma metastasis or recurrence, as well as sentinel lymph node (SLN) positivity, independent of traditional staging factors. Castle presented data on DecisionDx-Melanoma with two virtual posters. The first is a late-breaking abstract, titled "Integration of the 31-gene expression profile test with clinicopathologic features (i31-GEP) to assess sentinel lymph node positivity risk in patients with cutaneous melanoma." More details can be found here in the Company’s news release from April 15, 2021.

A second poster entitled "Using a 31-gene expression profile test to stratify patients with sentinel lymph node negative stage I-II cutaneous melanoma according to recurrence risk: Longer-term follow-up from a prospective, multicentre study" was also presented. This poster can be found here.

Study methods and findings:

Interim data from an independent, prospective multicentre Spanish cohort was previously published.
This poster provided long-term performance data (median follow-up 3.9 years) of 86 primary melanoma tumors from patients staged I-II at diagnosis according to the American Joint Committee on Cancer (AJCC) 7th edition criteria with negative SLN biopsy (SLNB) results, which were tested with DecisionDx-Melanoma.
Significantly different risk profiles (p=0.005) with respect to disease-free survival (DFS) were maintained between groups with:
3-year Disease-Free Survival for Class 1A = 100%
3-year Disease-Free Survival for Class 1A, 1B/2A = 91%
3-year Disease-Free Survival for Class 2B = 75%
Patients with a DecisionDx-Melanoma Class 2B result had three times the odds of recurrence relative to those with a Class 1A result.
Distant metastasis was not observed in the Class 1A group, while 75% occurred in the high-risk Class 2B group.
The study authors conclude that DecisionDx-Melanoma significantly differentiates recurrence risk in SLN negative patients, which may allow for better resource allocation to patients at the highest risk of melanoma recurrence.
DecisionDx-SCC:

DecisionDx-SCC is Castle’s prognostic gene expression profile test for patients diagnosed with high-risk cutaneous squamous cell carcinoma (SCC) designed to use a patient’s tumor biology to predict individual risk of metastasis for patients with SCC and one or more risk factors. Castle presented data on DecisionDx-SCC with two virtual posters. Both posters highlight studies in which the 40-gene expression profile test demonstrated the ability to accurately classify risk for metastasis in SCC patients with one or more risk factors.

The first poster is entitled "Incorporation of a prognostic 40-gene expression profile (40-GEP) test into clinicopathological risk assessment using newly published guidelines for cutaneous squamous cell carcinoma (cSCC)." The poster can be found here.

Study methods and findings:

The National Comprehensive Cancer Network (NCCN) published updated SCC guidelines in 2021, now categorizing local SCC into three groups: low risk, high risk and very high risk. This study was performed to assess the performance of DecisionDx-SCC within these new NCCN guidelines.
420 primary SCC archival tumor specimens with one or more associated risk factors and known three-year outcomes were collected, tested with DecisionDx-SCC and analyzed using Kaplan Meier and Cox multivariate regression analysis. All patients in this cohort were classified as high risk or very high risk by the new National Comprehensive Cancer Network (NCCN) framework.
Importantly:
DecisionDx-SCC provided significant stratification within both the NCCN high-risk group (p=0.0001) and the NCCN very high-risk group (p<0.0001).
Multivariate Cox regression analysis showed that both the DecisionDx-SCC Class 2A (p<0.001) and Class 2B (p<0.001) were independent and stronger predictors of metastasis when compared to NCCN groups (Class 2A hazard ratio = 2.92; Class 2B hazard ratio = 9.50; NCCN very high-risk hazard ratio = 1.99).
These data show that DecisionDx-SCC provides further stratification for the risk of metastasis within high-risk and very high-risk SCC sample groups.
The study validated that DecisionDx-SCC adds independent prognostic value within the new NCCN guidelines and could be applied as an adjunct to enhance SCC risk stratification and found that incorporating DecisionDx-SCC into SCC patient risk assessment could lead to more personalized and risk-appropriate pathways for improvement of patient management and disease related outcomes.
The second DecisionDx-SCC poster is entitled "Real-world clinical usage data demonstrates appropriate utilization of the prognostic 40-gene expression profile test for cutaneous squamous cell carcinoma with one or more risk factors." The poster can be found here .

Study methods and findings:

Summary metrics were generated on the first 1000 samples received for DecisionDx-SCC testing that met clinical testing criteria. Metrics on early clinical usage include:
69.0% of samples received DecisionDx-SCC Class 1 results, 26.0% received DecisionDx-SCC Class 2A results and 1.3% received DecisionDx-SCC Class 2B results.
Technical reliability of DecisionDx-SCC was 96.3%.
Most tested patients had three or more risk factors.
DecisionDx DiffDx-Melanoma:

DecisionDx DiffDx-Melanoma is designed to aid dermatopathologists in characterizing difficult-to-diagnose melanocytic lesions. DecisionDx DiffDx-Melanoma classifies these lesions as benign, intermediate-risk or malignant.

Two virtual posters highlighted studies in which DecisionDx DiffDx-Melanoma demonstrated the ability to refine diagnoses of difficult-to-diagnose melanocytic lesions.

The first poster is entitled "Performance of a diagnostic 35-gene expression profile test (GEP) on difficult-to-diagnose melanocytic lesions." The poster can be found here.

Study methods and findings:

DecisionDx DiffDx-Melanoma’s performance was evaluated in its target difficult-to-diagnose population, which included lesions of unknown malignant potential (UMP) and diagnostically discordant lesions.
In a cohort of 65 difficult-to-diagnose melanocytic lesions, the test provided a result in 100% of lesions, (i.e., no technical failures).
DecisionDx DiffDx-Melanoma provided an actionable result in 97% of these difficult-to-diagnose lesions, as only 2 cases received an intermediate result.
DecisionDx DiffDx-Melanoma’s previously published accuracy metrics in lesions with diagnostic concordance has been shown to alleviate uncertainty in difficult-to-diagnose lesions leading to recommendations for decreased unnecessary procedures while appropriately identifying at-risk patients.
The second DecisionDx DiffDx-Melanoma poster is entitled "Development, Validation, and Clinical Utility of the 35-Gene Expression Profile Test for Use as an Adjunctive Melanoma Diagnostic Tool." The data therein was previously published in SKIN: The Journal of Cutaneous Medicine. The poster can be found here.

Study methods and findings:

DecisionDx DiffDx-Melanoma was developed using artificial intelligence methods trained on 200 benign nevi and 216 melanomas to select a panel of 32 discriminant and 3 control genes.
The test’s ability to differentiate accurately between benign and malignant pigmented skin lesions was characterized.
The test provides a modest intermediate-risk zone of 3.6% and a high technical success rate at 96.6%.
The analytical validity data of the DecisionDx DiffDx-Melanoma test demonstrates high precision as an indication of technical success.
Dermatopathologists utilized the DecisionDx DiffDx-Melanoma result to refine their diagnoses and their diagnostic confidence increased by 51%.
Dermatologists utilized the DecisionDx DiffDx-Melanoma result, which led to altered treatment plans including re-excisions in agreement with the DecisionDx DiffDx-Melanoma result.
About DecisionDx-Melanoma

DecisionDx-Melanoma is a gene expression profile test that uses an individual patient’s tumor biology to predict individual risk of cutaneous melanoma metastasis or recurrence, as well as sentinel lymph node positivity, independent of traditional staging factors, and has been studied in more than 5,700 patient samples. Using tissue from the primary melanoma, the test measures the expression of 31 genes. The test has been validated in four archival risk of recurrence studies of 901 patients and six prospective risk of recurrence studies including more than 1,600 patients. To predict likelihood of sentinel lymph node positivity, the Company utilizes its proprietary algorithm, i31-GEP, to produce an integrated test result. i31-GEP is an artificial intelligence-based neural network algorithm (independently validated in a cohort of 1,674 prospective, consecutively tested patients with T1-T4 cutaneous melanoma) that integrates the DecisionDx-Melanoma test result with the patient’s traditional clinicopathologic features. Impact on patient management plans for one of every two patients tested has been demonstrated in four multicenter and single-center studies including more than 560 patients. The consistent performance and accuracy demonstrated in these studies provides confidence in disease management plans that incorporate DecisionDx-Melanoma test results. Through December 31, 2020, DecisionDx-Melanoma has been ordered more than 68,920 times for use in patients with cutaneous melanoma.

More information about the test and disease can be found at www.CastleTestInfo.com.

About DecisionDx DiffDx-Melanoma

DecisionDx DiffDx-Melanoma is designed to aid dermatopathologists in characterizing difficult-to-diagnose melanocytic lesions. Of the approximately 2 million suspicious pigmented lesions biopsied annually in the U.S., Castle estimates that approximately 300,000 of those cannot be confidently classified as either benign or malignant through traditional histopathology methods. DecisionDx DiffDx-Melanoma classifies these lesions as: benign (gene expression profile suggestive of benign neoplasm); intermediate-risk (gene expression profile cannot exclude malignancy); or malignant (gene expression profile suggestive of melanoma). Interpreted in the context of other clinical, laboratory and histopathologic information, DecisionDx DiffDx-Melanoma is designed to add diagnostic clarity and confidence for dermatopathologists while helping dermatologists deliver more informed patient management plans.

More information about the test and disease can be found at www.CastleTestInfo.com.

About DecisionDx-SCC

DecisionDx-SCC is a 40-gene expression profile test that uses an individual patient’s tumor biology to predict individual risk of cutaneous squamous cell carcinoma metastasis for patients with one or more risk factors. The test result, in which patients are stratified into a Class 1, 2A or 2B risk category, predicts individual metastatic risk to inform risk-appropriate management.

Peer-reviewed publications have demonstrated that DecisionDx-SCC is an independent predictor of metastatic risk and that integrating DecisionDx-SCC with current prognostic methods can add positive predictive value to clinician decisions regarding staging and management.

On April 16, 2021 Moderna, Inc. (Nasdaq: MRNA), a biotechnology company pioneering messenger RNA (mRNA) therapeutics and vaccines, reported that it will host a live conference call and webcast at 8:00 a.m. ET on Thursday, May 6, 2021 to reports its first quarter 2021 financial results, and provide a corporate update (Press release, Moderna Therapeutics, APR 16, 2021, View Source [SID1234578127]).

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To access the live conference call, please dial 866-922-5184 (domestic) or 409-937-8950 (international) and refer to conference ID 7487119. A webcast of the call will also be available under "Events and Presentations" in the Investors section of the Moderna website at investors.modernatx.com. The archived webcast will be available on Moderna’s website approximately two hours after the conference call and will be available for one year following the call.