New Cardinal Health cancer research study published in JAMA

On March 2, 2021 Cardinal Health Specialty Solutions reported that it is making important strides in cancer research using real-world evidence (RWE) (Press release, Cardinal Health, MAR 2, 2021, View Source [SID1234575923]). Research developed by Cardinal Health, published today in the Journal of the American Medical Association, suggests that RWE may be able to be applied more broadly in cancer research and regulatory filings, and could lead to accelerated clinical trials in cancer research . Real-world evidence is generated during routine clinical practice and outside the context of randomized controlled trials.

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"The potential to use RWE to complement and supplement traditional data from randomized controlled trials is exciting because it could significantly accelerate the speed at which clinical trials are conducted, and as a result, innovative medicines could be approved and reach patients faster," said Bruce Feinberg, DO, Chief Medical Officer at Cardinal Health Specialty Solutions and lead author of the study.

Although randomized controlled trials remain the standard for assessing the safety and efficacy of new cancer therapies, there is increasing recognition that this research method is costly, time consuming and does not accurately represent the general population. These factors – combined with the growth of targeted therapies and the rapidly expanding field of drug development – have researchers and policy makers like the Federal Drug Administration (FDA) exploring how real-world evidence can be used to accelerate the drug development process.

The Cardinal Health study included 956 patients who were receiving first-line treatment for thyroid cancer, breast cancer or melanoma between 2014 and 2017. The research used a novel, real-world application of the Response Evaluation Criteria in Solid Tumors (RECIST) methodology to assess how the patients’ tumors responded to the therapy.

"The study findings show that applying RECIST methodology to real-world data may allow for accurate comparisons between real-world data and RCT data in solid tumors. We believe these findings may open the door for broader use of RWE across many areas of cancer research," said Ajeet Gajra, MD, Sr. Medical Director at Cardinal Health and one of the co-authors of the study.

MannKind Corporation to Participate in H.C. Wainwright Global Life Sciences Conference

On March 2, 2021 MannKind Corporation (Nasdaq: MNKD), a company focused on the development and commercialization of inhaled therapeutic products for patients with endocrine and orphan lung diseases, reported that its Chief Executive Officer, Michael Castagna, PharmD, will present at the H.C. Wainwright Global Life Sciences Conference which will be available on demand starting on March 9, 2021 at 7:00 am (ET) (Press release, Mannkind, MAR 2, 2021, View Source [SID1234575939]). Interested parties can access a link to the on demand webcast of the presentation from the Events & Presentations section of the Company’s website at View Source The webcast replay will remain available for 14 days following the live presentation.

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Verastem Oncology to Participate in the H.C. Wainwright Global Life Sciences Conference

On March 2, 2021 Verastem, Inc. (Nasdaq:VSTM) (also known as Verastem Oncology), a biopharmaceutical company committed to advancing new medicines for patients battling cancer, reported that the Company will participate in the virtual H.C. Wainwright Global Life Sciences Conference (Press release, Verastem, MAR 2, 2021, View Source [SID1234575955]). The fireside chat will be made available for on-demand listening beginning Tuesday, March 9, 2021 at 7:00 a.m. EST.

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The webcast of the fireside chat will be available on the "Media" section of the Company’s website at www.verastem.com under "Events & Presentations".

Epizyme Outlines Clinical Progress, TAZVERIK Development Strategy and Pipeline Priorities During Company’s Strategic Vision Call

On March 2, 2021 Epizyme, Inc. (Nasdaq: EPZM), a fully integrated, commercial-stage biopharmaceutical company developing and delivering novel epigenetic therapies, reported outlining the Company’s strategic vision and focus on oncology treatment with the Company’s epigenetic pipeline (Press release, Epizyme, MAR 2, 2021, View Source [SID1234575971]). A link to the presentation and slideshow can be found here.

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"The recent accelerated approvals of TAZVERIK in Epithelioid Sarcoma and Relapsed / Refractory Follicular Lymphoma are, of course, the critical foundation on which we will continue to expand Epizyme’s mission of rewriting treatments for cancer through novel epigenetic medicines," said Robert Bazemore, President and Chief Executive Officer of Epizyme. "We believe TAZVERIK has the potential to become a backbone of treatment across lines of ES and FL therapy. Beyond that, we are focused on developing the pipeline-in-a-drug potential we see in TAZVERIK, while expanding our portfolio and bringing novel oncology therapeutics into clinical development. We aspire to change the standard of care for patients and physicians by developing targeted medicines with fundamentally new mechanisms of action directed at specific causes of cancers."

"I am really excited about Epizyme’s comprehensive and innovative clinical development plan both for tazemetostat and our pipeline, for which we anticipate a steady stream of data," said Dr. Shefali Agarwal, Epizyme’s, EVP, Chief Medical and Development Officer. "Our aim is to demonstrate the benefits of tazemetostat to patients in earlier lines of treatment for ES and FL by exploring various combinations with standard of care therapies. We also plan to further explore tazemetostat as both monotherapy and in combinations across multiple new hematologic and solid tumor cancers. Our experience and platform have led to an incredibly rich pipeline of both clinical and discovery programs targeting several classes of epigenetic modulators and we expect that many these programs will be entering clinical trials in the next several years."

Highlights from the Next EPIsode call included:

Preliminary Data from Confirmatory Phase 1b/3 Study EZH-302 in Follicular Lymphoma

The combination of TAZVERIK + R2 (Lenalidomide and Rituximab) is being evaluated in the Phase 1b safety run-in portion of the EZH-302 trial, which enrolled a total of 13 patients. Among the 13 patients evaluated in this standard dose escalation design, no Dose Limiting Toxicities (DLTs) were observed during the first cycle of treatment up to the highest dose of 800mg of TAZVERIK twice daily. As of mid-February, initial data from the trial also showed:

All but one of the 13 patients enrolled remain on therapy, and, to date, seven of the patients were also considered evaluable for efficacy based on the availability of tumor scans from investigators.
All seven evaluable patients demonstrated a response to treatment with TAZVERIK+R2 with three complete responses (CR) and four partial responses (PR).
The safety profile of TAZVERIK + R2 observed in these patients to date is consistent with that previously described with the respective drugs; no patients have discontinued due to an adverse event.
Based on these early safety and activity findings, Epizyme is preparing to commence the Phase 3 randomization portion of the trial. The Company plans to present further data from the Phase 1b portion of the study at a medical meeting in 2021.

Preliminary Data from Phase 1b/2 Study EZH-1101 In Prostate Cancer

The combination of tazemetostat with standard of care treatments, enzalutamide or abiraterone, is being evaluated in the Phase 1b safety run-in portion of the EZH-1101 trial, which enrolled a total of 21 men with metastatic prostate cancer. Among the 21 patients enrolled in a standard dose escalation design, no DLTs were observed at any dose of tazemetostat up to a maximum dose of 1600mg twice daily. As of mid-February, initial data from the trial also showed:

Seven out of 21 patients had a PSA response of ≥50%; one additional patient had a PSA response of ≥35%.
Six of the PSA50 responses were in the tazemetostat + enzalutamide cohort and one was in the tazemetostat + abiraterone/prednisone cohort.
The Company also observed a 47% disease control rate to-date and presented an example of radiographic response in a patient achieving a partial response (PR) in the trial.
The Company highlighted that all responses were in ARV7 negative patients using the EPIC platform, considered more rigorous in detecting ARV7 status. Only one ARV7 positive patient was enrolled in the safety run-in portion of the trial.
Based on these early safety and activity findings, Epizyme recently initiated enrollment in the Phase 2 efficacy portion of the trial. The Company plans to present further data from the Phase 1b portion of the study at a medical meeting in 2021.

Initiation of Basket Trials in Additional Heme and Solid Tumors Planned in Second Half 2021

Epizyme will initiate two signal finding basket studies to evaluate tazemetostat safety and efficacy across multiple new types of heme and solid tumors. With this approach, the Company will study multiple combinations with standard of care therapies and novel mechanisms of action to expand the potential of TAZVERIK to patients and the physicians who treat them. Epizyme announced on today’s call that it plans to initiate both basket studies in the second half of 2021.

Advancing Epizyme’s Novel SETD2 Inhibitor Program into the Clinic in 2021

SETD2 is a histone methyltransferase, similar to EZH2, which plays multiple important roles in oncogenesis. Based on the promise of SETD2 inhibition in multiple settings, including high risk t(4;14) multiple myeloma and in other B-cell malignancies such as large-cell lymphoma, as well as in combination with existing and emerging therapies including tazemetostat, Epizyme is planning to submit an IND (Investigational New Drug) application with the FDA in mid-2021.

"We are extremely excited about the potential for our SETD2 program and breadth of our growing discovery pipeline. Our ambition is to deliver at least five of these programs to the clinic in the next five years," said Jeffery Kutok, M.D., Ph.D, Epizyme’s Chief Scientific Officer. "We look forward to continuing to update you on the considerable number of promising compounds against important oncology targets in our pipeline as they move toward clinical development, furthering Epizyme’s mission of delivering medicines that can make meaningful differences in the lives of patients with cancer."

On the call today, the company reiterated its financial strength, with cash expected to be sufficient to fund these initiatives into 2023. Additionally, Epizyme plans to continue to evaluate collaborations with companies with an established commercial presence outside the United States, as well as potential clinical trial collaborations involving drugs where a tazemetostat combination makes sense. The scientific advice process with the European Medicines Evaluation Agency (EMEA) for tazemetostat in follicular lymphoma has begun and Epizyme will provide more details from these interactions and the path ahead later this year.

"As we consider the next five years, we expect to be the preferred partner of choice for accessing innovation in epigenetics. We have established a deep biological understanding of how best to evaluate novel, best in class epigenetic targets and, importantly, ensuring the right translational insights are captured early," said Matthew Ros, EVP, Chief Strategy and Business Officer. "We plan to leverage clinical and research collaborations to expand our ability to interrogate multiple investigative therapies with partners with like-minded scientific interests and expertise."

Next EPIsode Presentation and Slideshow

A replay of today’s webcast and the full slideshow presentation can be accessed and downloaded by visiting the Investor section of the Company’s website at www.epizyme.com.

Myriad Genetics Joins Forces with Intermountain Precision Genomics for a Comprehensive Offering of Germline and Somatic Tumor Testing Services

On March 2, 2021 Myriad Genetics, Inc. (NASDAQ: MYGN), a leader in genetic testing and precision medicine, reported a new strategic precision oncology collaboration with Intermountain Precision Genomics, a service of Intermountain Healthcare (Press release, Myriad Genetics, MAR 2, 2021, View Source [SID1234575994]). This collaboration creates a comprehensive germline genetic testing and somatic tumor offering that combines Myriad’s advanced hereditary cancer and companion diagnostic tests with Intermountain Precision Genomics’ world-class laboratory services, utilizing the TheraMap test—powered by the TruSight Oncology 500 (TSO 500) next-generation sequencing test from Illumina, Inc. (NASDAQ: ILMN).

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TSO 500 is rapidly becoming a standard in oncology. Through the offering, oncologists will obtain a complete genetic analysis in one, easy-to-interpret report, enabling them to leverage the benefits of comprehensive genomic profiling.

"As cancer treatment continues to evolve, most oncologists seek both germline and somatic tumor testing results to best guide treatment decisions. Advancing precision oncology requires strong collaboration between forward-looking organizations across the healthcare ecosystem. This alliance with Intermountain Precision Genomics is an example of our drive to improve health outcomes for patients with cancer and expand innovative treatment options for the healthcare providers who care for them," said Paul J. Diaz, president and CEO, Myriad Genetics. "We are successfully merging the power of companion diagnostics, next-generation tumor sequencing, and world-class testing services to help raise the global standard of care in precision oncology."

"As a physician, I’m excited to make this comprehensive offering available to more patients," said Lincoln Nadauld, MD, PhD, oncologist and vice president and chief of precision health and academics at Intermountain Healthcare. "This partnership allows us to make a tremendous impact by providing the best that precision cancer care has to offer."

Strategic Partnership with Intermountain Precision Genomics
Intermountain Precision Genomics is a nationally recognized program focused on building partnerships to accelerate the field even further. The new, comprehensive offering – set to be available in the second half of 2021 – will utilize germline genetic testing with the Myriad myRisk Hereditary Cancer test, and somatic tumor profiling with Myriad myChoice CDx. Intermountain Precision Genomics will perform tumor profiling and tissue analysis using its TheraMap test that is powered by Illumina’s TSO 500 assay.

Myriad myChoice CDx is widely supported by private insurance payers. Both myChoice CDx and TheraMap are covered by Medicare.

About TheraMap
TheraMap: Solid Tumor is a hybrid capture DNA- and RNA-based test that detects SNV, INDELs, Copy Number Variants (CNV), and fusions in solid tumors. TheraMap is validated for all solid tumor types and includes sequencing over 500 genes identified as relevant to cancer treatment, relevant gene fusion events, including NTRK fusions, as well as the important microsatellite instability (MSI) and tumor mutational burden (TMB) biomarkers. TheraMap is powered by Illumina’s TSO 500 assay.

About Myriad myRisk Hereditary Cancer
The Myriad myRisk Hereditary Cancer test uses an extensive number of sophisticated technologies and proprietary algorithms to evaluate 35 clinically significant genes associated with eight hereditary cancer sites including: breast, colon, ovarian, endometrial, pancreatic, prostate and gastric cancers and melanoma. For more information, visit View Source

About myChoice CDx
Myriad myChoice is the most comprehensive HRD test, enabling physicians to identify patients with tumors that have lost the ability to repair double-stranded DNA breaks, resulting in increased susceptibility to DNA-damaging drugs such as platinum drugs or PARP inhibitors. The myChoice test includes tumor sequencing of the BRCA1 and BRCA2 genes and a composite of three proprietary technologies (loss of heterozygosity, telomeric allelic imbalance, and large-scale state transitions). For more information, visit View Source