Recursion Enters Into Global Licensing Agreement with Takeda to Develop TAK-733 in Hereditary Cancer Syndrome

On May 14, 2020 Recursion, a digital biology company industrializing drug discovery, reported it has entered into a global licensing agreement with Takeda Pharmaceutical Company Limited (Takeda) to gain rights to TAK-733, a clinical-stage MEK inhibitor, and develop it for the treatment of a hereditary cancer syndrome and related areas of oncology (Press release, Recursion Pharmaceuticals, MAY 14, 2020, View Source [SID1234558074]).

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"TAK-733 is a great example of the power of our approach to decode challenging and important areas of biology. By applying machine learning to images of cells, we capture cellular changes accompanying hundreds of unique biological perturbations, and even loss of just a single gene," said Chris Gibson, Ph.D., co-founder and CEO, Recursion. "Using our platform, we uncovered targeted areas of oncology where TAK-733 could be effective. And because our dataset is fully relatable, we then cross-referenced TAK-733 against hundreds of disease models we’ve developed already or will develop in the coming years."

TAK-733 was identified as a potential treatment for a hereditary tumor syndrome using Recursion’s approach to creating cellular models of diseases where genes are inactive. Using its automated drug discovery platform, Recursion discovered the potential of TAK-733 by testing more than 200 potential molecules from Takeda’s library against the most effective potential treatment for cancers carrying particular mutations.

"We’re making immense progress in oncology by understanding the genetic drivers of different tumor types and developing targeted therapeutics," said Ron Alfa, M.D., Ph.D., Senior Vice President, Translational Discovery. "Almost all available drugs today target a particular type of mutation that result in activated proteins — oncogenes. However, most tumors also harbor mutations that eliminate or deactivate proteins, tumor suppressors, and only a few of these pathways have been understood well enough to develop therapeutics. Recursion’s platform opens the door to discovering targeted therapeutics that are effective when these tumor suppressors are inactive."

Under the terms of the agreement, Recursion obtains exclusive worldwide rights to develop and commercialize TAK-733. For more information on Recursion, please visit www.recursionpharma.com.

Bayer and ArcherDX Announce Global Collaboration to Develop Next-Generation Sequencing (NGS)-based Companion Diagnostic (CDx) for VITRAKVI® (larotrectinib)

On May 14, 2020 Bayer and ArcherDX, Inc. reported a global collaboration for the development and commercialization of a next-generation sequencing (NGS)-based companion diagnostic (CDx) for Vitrakvi (larotrectinib) (Press release, Bayer, MAY 14, 2020, View Source [SID1234558073]). The primary objective of the collaboration is to broaden patient access to comprehensive genomic testing inclusive of neurotrophic receptor tyrosine kinase 1 (NTRK1), NTRK2 and NTRK3 gene fusions and to help improve identification of appropriate treatment options for patients with TRK fusion cancer which can lead to meaningful treatment options.

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Vitrakvi is indicated for the treatment of adult and pediatric patients with solid tumors that have a neurotrophic tropomyosin receptor kinase (NTRK) gene fusion without a known acquired resistance mutation, are either metastatic or where surgical resection will likely result in severe morbidity, and have no satisfactory alternative treatments or that have progressed following treatment. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.1

Genomic cancer testing helps detect changes in a tumor’s DNA, called genomic profiling, that can determine how the cancer behaves and why it grows or spreads.2 Almost 50 percent of patients who undergo genomic cancer testing may have actionable genomic alterations, meaning the alterations can be matched to treatments that have been approved or are in clinical trials.3,4 Genomic cancer testing has already impacted patient cancer care in both common and rare malignancies. This has allowed for the identification of personalized treatment options.5 However, studies show that only a small percentage of cancer patients undergo such testing.5 It is therefore critical that testing becomes part of routine clinical practice to help guide treatment choice.

"In order to help more patients to benefit from Vitrakvi, broader access to high quality testing via next-generation sequencing is of key importance," said Robert LaCaze, Member of the Executive Committee of Bayer’s Pharmaceuticals Division and Head of the Oncology Strategic Business Unit at Bayer. "The collaboration with ArcherDX perfectly complements Bayer’s CDx strategy and fits our ambition to provide the right treatment to the right patients with cancer."

"Barriers currently exist that inhibit the broad adoption of genomic testing to inform treatment decisions. A kit-based CDx that is tumor agnostic and detects all NTRK gene fusions will enable high-quality genomic testing to be deployed in regional and community settings, where 85 percent of cancer patients receive care.6 For these patients, our technology provides RNA-based profiling for the detection of actionable fusions across known and novel NTRK fusion partners," said Jason Myers, Chief Executive Officer and co-founder, ArcherDX. "We are pleased to collaborate with Bayer to address their medical need and accelerate access to therapy."

NGS-based CDx tests aim to unlock molecular information from each patient’s tumor genome to guide treatment decisions for cancer therapies. ArcherDX is developing and pursuing regulatory clearances for an in vitro diagnostic (IVD), a comprehensive NGS-based therapy selection product that utilizes Archer’s proprietary Anchored Multiplex PCR (AMP) technology to measure clinically relevant genomic mutations for tumor profiling and CDx from both tissue and blood.

ArcherDX and Bayer are developing a kit-based CDx to detect NTRK gene fusions, including NTRK1, NTRK2 and NTRK3 for Vitrakvi and plan to seek approval in different markets, including the European Union (EU), U.S. and Japan. The CDx version of this panel will allow local laboratories to provide referring physicians with potentially actionable genomic information, so that appropriate patients can be matched to specific therapeutic options based on their genomic findings.

The Pharmaceuticals Business Development & Licensing team of Bayer facilitated this collaboration. Financial terms of the agreement were not disclosed.

About Vitrakvi (larotrectinib)

Vitrakvi (larotrectinib) is a first-in-class TRK inhibitor for TRK fusion cancer across solid tumors. Vitrakvi is indicated for the treatment of adult and pediatric patients with solid tumors that have a neurotrophic tropomyosin receptor kinase (NTRK) gene fusion without a known acquired resistance mutation, are either metastatic or where surgical resection will likely result in severe morbidity, and have no satisfactory alternative treatments or that have progressed following treatment. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.1

Vitrakvi is approved in several markets, including the U.S., EU, Canada and Brazil.

TRK fusion cancer is rare overall, affecting no more than a few thousand patients across the U.S. and Europe. It affects both children and adults and occurs in varying frequencies across various solid tumor types. TRK fusion cancer occurs when an NTRK gene fuses with another unrelated gene, producing an altered TRK protein. These TRK fusion proteins act as oncogenic drivers that fuel the spread and growth of the patients’ cancer, regardless of where it originates in the body.

Important Safety Information for VITRAKVI (larotrectinib)

Neurotoxicity: Among the 176 patients who received VITRAKVI, neurologic adverse reactions of any grade occurred in 53% of patients, including Grade 3 and Grade 4 neurologic adverse reactions in 6% and 0.6% of patients, respectively. The majority (65%) of neurologic adverse reactions occurred within the first three months of treatment (range 1 day to 2.2 years). Grade 3 neurologic adverse reactions included delirium (2%), dysarthria (1%), dizziness (1%), gait disturbance (1%), and paresthesia (1%). Grade 4 encephalopathy (0.6%) occurred in a single patient. Neurologic adverse reactions leading to dose modification included dizziness (3%), gait disturbance (1%), delirium (1%), memory impairment (1%), and tremor (1%).1

Advise patients and caretakers of these risks with VITRAKVI. Advise patients not to drive or operate hazardous machinery if they are experiencing neurologic adverse reactions. Withhold or permanently discontinue VITRAKVI based on the severity. If withheld, modify the VITRAKVI dose when resumed.1

Hepatotoxicity: Among the 176 patients who received VITRAKVI, increased transaminases of any grade occurred in 45%, including Grade 3 increased AST or ALT in 6% of patients. One patient (0.6%) experienced Grade 4 increased ALT. The median time to onset of increased AST was 2 months (range: 1 month to 2.6 years). The median time to onset of increased ALT was 2 months (range: 1 month to 1.1 years). Increased AST and ALT leading to dose modifications occurred in 4% and 6% of patients, respectively. Increased AST or ALT led to permanent discontinuation in 2% of patients.1

Monitor liver tests, including ALT and AST, every 2 weeks during the first month of treatment, then monthly thereafter, and as clinically indicated. Withhold or permanently discontinue VITRAKVI based on the severity. If withheld, modify the VITRAKVI dosage when resumed.1

Embryo-Fetal Toxicity: VITRAKVI can cause fetal harm when administered to a pregnant woman. Larotrectinib resulted in malformations in rats and rabbits at maternal exposures that were approximately 11- and 0.7-times, respectively, those observed at the clinical dose of 100 mg twice daily.1

Advise women of the potential risk to a fetus. Advise females of reproductive potential to use an effective method of contraception during treatment and for 1 week after the final dose of VITRAKVI.1

Most Common Adverse Reactions (≥20%): The most common adverse reactions (≥20%) were: increased ALT (45%), increased AST (45%), anemia (42%), fatigue (37%), nausea (29%), dizziness (28%), cough (26%), vomiting (26%), constipation (23%), and diarrhea (22%).1

Drug Interactions: Avoid coadministration of VITRAKVI with strong CYP3A4 inhibitors (including grapefruit or grapefruit juice), strong CYP3A4 inducers (including St. John’s wort), or sensitive CYP3A4 substrates. If coadministration of strong CYP3A4 inhibitors or inducers cannot be avoided, modify the VITRAKVI dose as recommended. If coadministration of sensitive CYP3A4 substrates cannot be avoided, monitor patients for increased adverse reactions of these drugs.1

Lactation: Advise women not to breastfeed during treatment with VITRAKVI and for 1 week after the final dose.1

Please see the full Prescribing Information for VITRAKVI (larotrectinib).

About Oncology at Bayer

Bayer is committed to delivering science for a better life by advancing a portfolio of innovative treatments. The oncology franchise at Bayer includes six marketed products and several other assets in various stages of clinical development. Together, these products reflect the company’s approach to research, which prioritizes targets and pathways with the potential to impact the way that cancer is treated.

Foundation Medicine and Collaborators to Share New Data During the ASCO20 Virtual Scientific Program Showcasing the Importance of Comprehensive Genomic Profiling (CGP) in Informing Advanced Cancer Clinical Decision-Making

On May 14, 2020 Foundation Medicine, Inc. reported that new data supporting the clinical use of its portfolio of tissue and liquid comprehensive genomic profiling (CGP) tests will be presented at the 2020 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Virtual Scientific Program, which will be held from May 29 to May 31 (Press release, Foundation Medicine, MAY 14, 2020, View Source [SID1234558072]). The company and its collaborators will present a total of 22 studies, including two clinical symposia presentations and one poster discussion. The data highlight insights into the utility of liquid biopsy CGP testing to help inform treatment selection across a variety of advanced cancers and the predictive role of tumor mutational burden (TMB) as a genomic signature to optimize precision medicine approaches.

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Highlights of these presentations include:

new data demonstrating the use of liquid biopsy CGP to identify predictive and prognostic biomarkers to improve clinical outcomes for patients with advanced cancer, including metastatic breast cancer, non-small lung cancer (NSCLC) and prostate cancer;
additional evidence of the role of complex biomarkers and genomic signatures such as TMB may have in patient response to targeted therapies and immunotherapies; and
the clinical value of liquid biopsy as a tool for oncologists in various treatment settings, including the ability to identify resistance mechanisms across primary and metastatic tumors and enable precision medicine.
"At this meeting, we’ll share new data demonstrating the use of clinically relevant insights from our tissue and liquid comprehensive genomic profiling tests to inform treatment decision-making and improve patient outcomes through methods such as identifying resistance mechanisms," said Brian Alexander, M.D., M.P.H., chief medical officer at Foundation Medicine. "We’re at an important juncture in personalized medicine where pan-tumor complex signatures such as TMB and MSI are growing in their clinical importance, and our data show the unique ability of CGP to investigate these nuanced biomarkers of response and advance precision treatment approaches."

Clinical Validity of Liquid Biopsy Comprehensive Genomic Profiling

In a study conducted in collaboration with the Dana Farber Cancer Institute, researchers characterizing NSCLC cases found MET exon 14 skipping alterations present in 2.3 percent of tumor DNA samples, representing six major subtypes. The analysis of paired tumor and liquid biopsy samples identified potential acquired resistance mechanisms that may be critical to predicting response to MET inhibitors and identifying matched targeted therapy options, which have recently shown clinical efficacy for this subset of patients, demonstrating the importance of testing NSCLC patients for these alterations.

[Characterization of 1,387 NSCLCs with MET exon 14 (METex14) skipping alterations (SA) and potential acquired resistance (AR) mechanisms. Abstract #9511.]

In a retrospective analysis, researchers compared more than 325,000 tumor tissue samples to over 28,000 patient-matched plasma samples to understand the clinical validity of liquid biopsy CGP in detecting kinase fusions. Kinase gene fusions identified by tissue-based CGP were also detected by liquid biopsy CGP in temporally-matched plasma samples, with high positive percent agreement between tissue and liquid biopsies. The analysis of liquid biopsy samples also identified acquired alterations consistent with known mechanisms of resistance.

[Pan-tumor analyses of kinase fusions detected in circulating tumor DNA (ctDNA) and concordance with paired tissue. Abstract #3517. Poster #247.]

A clinically advanced prostate cancer study used CGP on pre-treatment primary tumor biopsy, post-treatment metastatic biopsy and liquid biopsy to uncover differences in genomic alterations and potential impact on treatment selection. The study demonstrated how systemic treatment resulted in genomic changes in metastases as the tumor evolves to survive. Given the variance between metastatic sites, liquid biopsy may capture a broader range of therapy opportunities for patients.

[Contrasting genomic profiles in post-systemic treatment metastatic sites (MET), pretreatment primary tumors (PT), and liquid biopsies (LB) of clinically advanced prostate cancer (PC). Abstract #5534. Poster #115.]

Complex Biomarkers as Predictor of Immunotherapy Response

In an analysis of the Phase III IMvigor130 study, researchers explored the potential predictive role of TMB, PD-L1 expression and T-effector gene expression biomarkers in patients with metastatic urothelial cancer. The results reinforce the potential predictive nature of biomarkers associated with response or resistance to atezolizumab monotherapy or atezolizumab in combination with platinum-based chemotherapy.

[Tumor, immune, and stromal characteristics associated with clinical outcomes with atezolizumab (atezo) + platinum-based chemotherapy (PBC) or atezo monotherapy (mono) versus PBC in metastatic urothelial cancer (mUC) from the phase III IMvigor130 study. Abstract #5011.]

The following is a list of select abstracts that will be presented at the meeting. To access all abstracts being presented by Foundation Medicine and its collaborators, please visit: meetinglibrary.asco.org.

Abstract #

Title

Collaborator

Clinical Science Symposia

9511

Characterization of 1,387 NSCLCs with MET exon 14 (METex14) skipping alterations (SA) and potential acquired resistance (AR) mechanisms

Dana Farber Cancer Institute

5011

Tumor, immune, and stromal characteristics associated with clinical outcomes with atezolizumab (atezo) + platinum-based chemotherapy (PBC) or atezo monotherapy (mono) versus PBC in metastatic urothelial cancer (mUC) from the phase III IMvigor130 study

Poster Discussion

3517

Pan-tumor analyses of kinase fusions detected in circulating tumor DNA (ctDNA) and concordance with paired tissue

Poster Presentations

1050

Patient-matched tissue and liquid biopsies identify shared and acquired genomic alterations in breast cancer

5534

Contrasting genomic profiles in post-systemic treatment metastatic sites (MET), pretreatment primary tumors (PT), and liquid biopsies (LB) of clinically advanced prostate cancer (PC)

TPS2087

A multi-stakeholder platform to prospectively link longitudinal, real-world clinico-genomic, imaging and outcomes data for patients with metastatic lung cancer

Flatiron Health

3060

Characteristics and outcomes of real-world (RW) patients (pts) with microsatellite instability-high (MSI-H) solid tumors treated with pembrolizumab monotherapy (P) after FDA approval

Flatiron Health

9591

Real-world (RW) outcomes for non-small cell lung cancer (NSCLC) patients (pts) with EGFR exon 19 deletions (x19del) stratified by deletion size

Flatiron Health

3622

Increased tumor purity and improved biomarker detection using precision needle punch enrichment of specimen paraffin blocks: method validation and implementation in a prospective clinical trial

3620

Pan-cancer analysis of FGFR1-3 genomic alterations to reveal a complex molecular landscape

5527

Association of BRCA alteration (alt) type with real-world (RW) outcomes to PARP inhibitors (PARPi) in patients (pts) with metastatic castrate resistant prostate cancer (mCRPC)

Exicure to Present at the American Society of Clinical Oncology 2020

On May 14, 2020 Exicure, Inc. (NASDAQ: XCUR), the pioneer in gene regulatory and immunotherapeutic drugs utilizing spherical nucleic acid (SNA) constructs, reported that it will present a poster at the 2020 Annual Meeting of the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper), taking place virtually from May 29-31 (Press release, Exicure, MAY 14, 2020, View Source [SID1234558071]).

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The ASCO (Free ASCO Whitepaper) poster, by Dr. Mohammed Milhem of the University of Iowa and coauthors, titled "AST-008: A Novel Approach to TLR9 Agonism with PD-1 Blockade for Anti-PD-1 Refractory Merkel Cell Carcinoma (MCC) and Cutaneous Squamous Cell Carcinoma (CSCC)" will be presented virtually in the Developmental Therapeutics—Immunotherapy Poster Session under abstract number TPS3164.

The poster will present details of the phase Ib/II trial of Exicure’s drug candidate, AST-008, a toll-like receptor 9 agonist oligonucleotide being developed for the treatment of MCC and CSCC in patients progressing on immune checkpoint inhibitor monotherapy.

Key features of the phase Ib/II trial described in the poster include:

Advanced/metastatic MCC and CSCC are skin cancers with high unmet medical need;
Data presented earlier suggest that AST-008 elicits a Th1-type cytokine response and immune cell activation;
The phase II dose escalation study will use the recommended dose of AST-008, administered intratumorally, in combination with pembrolizumab or cemiplimab to treat two cohorts of patients with advanced/metastatic MCC or CSCC respectively;
The enablement of a dose expansion of AST-008 plus cemiplimab in CSCC without having performed a separate dose escalation with the latter combination;
Each expansion cohort will enroll up to 29 patients across about 15 US-based sites;
Patients enrolled into the phase II will have to have progressed on a single-agent checkpoint therapy;
The key objective of the phase II expansion is to provide an estimate of preliminary efficacy of intratumorally administered AST in the trial subjects. Other endpoints include safety, pharmacokinetic and pharmacodynamic assessments.
About AST-008

AST-008 is an SNA consisting of toll-like receptor 9 agonists designed for immuno-oncology applications. As of January 31, 2020, we have dosed 17 patients in the Phase 1b stage of the clinical trial. We have observed no treatment related serious adverse events, nor have we observed any dose-limiting toxicity among the treated subjects. The most common reported adverse event was injection site reactions. In December 2019, we received preliminary results from the Phase 1b/2 stage of the clinical trial showing potential signs of anti-tumor activity in patients with Merkel cell carcinoma. In the second quarter of 2020, we plan to initiate a Phase 2 dose expansion for intratumoral AST-008 in combination with approved checkpoint inhibitors to treat two cohorts of patients with advanced or metastatic Merkel cell carcinoma or cutaneous squamous cell carcinoma. Each cohort is expected to enroll up to 29 patients who have failed anti-PD-1/PD-L1, or programmed cell death protein 1/programmed death-ligand 1, therapy. We expect to open a total of up to 15 sites in the United States.

ALX Oncology to Present ALX148 Phase 1 Study at the 2020 American Society of Clinical Oncology (ASCO) Annual Meeting

On May 14, 2020 ALX Oncology Holdings Inc., a clinical-stage immuno-oncology company developing therapies that block the CD47 checkpoint pathway, reported that the Phase 1 study of ALX148 in patients with advanced solid tumors has been selected for presentation in the Poster Discussion Session at the Virtual 2020 ASCO (Free ASCO Whitepaper) Annual Meeting, May 29 – 31, 2020 (Press release, ALX Oncology, MAY 14, 2020, View Source [SID1234558070]).

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Virtual ASCO (Free ASCO Whitepaper) 2020 Presentation Information
Title: A Phase 1 study of ALX148, a CD47 blocker, in combination with standard anticancer antibodies and chemotherapy regimens in patients with advanced malignancy (Abstract #3056).
Poster Discussion Session: Developmental Therapeutics—Immunotherapy
Date: Friday, May 29