On April 22, 2021 Vertex Pharmaceuticals Incorporated (Nasdaq:VRTX) and Obsidian Therapeutics reported that the two companies have entered into a strategic research collaboration and licensing agreement focused on the discovery of novel therapies that regulate gene editing for the treatment of serious diseases (Press release, Obsidian Therapeutics, APR 22, 2021, View Source [SID1234578363]). The collaboration leverages Obsidian’s cytoDRiVE platform technology to discover gene-editing medicines whose therapeutic activity can be precisely controlled using small molecules and Vertex’s established scientific and clinical capabilities in small molecule, cell and genetic therapies to more rapidly bring these approaches to patients.

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"This collaboration with Obsidian builds upon and will expand Vertex’s leadership in small molecule and genetic therapies, and we’re excited to partner with the team at Obsidian to explore the capabilities of their technology," said Vertex’s David Altshuler, Executive Vice President, Global Research and Chief Scientific Officer. "The ability to tune gene-editing activity to a specific level is an important innovation that has the potential to address several serious diseases."

"At Obsidian, we’re using our cytoDRiVE technology to pioneer a new generation of engineered cell and gene therapies for patients with serious diseases," said Paul Wotton, Chief Executive Officer of Obsidian Therapeutics. "Partnering with Vertex, an established leader at the forefront of genetic therapies, will further advance our technology into the gene therapy field and accelerate development of controllable gene editing therapies for patients."

About the Collaboration

Under the terms of the agreement, Obsidian will use its cytoDRiVE technology to develop novel regulated gene editing therapy candidates for multiple serious diseases. Obsidian grants Vertex the exclusive option to license worldwide rights to candidates discovered and developed under the collaboration. Following Vertex’s exercise of its options, Vertex will be responsible for further preclinical and clinical development and commercialization.

Vertex will pay Obsidian up to $75 million in upfront payments and research milestones that may be paid during the research term, including an equity investment in Obsidian. Obsidian is eligible to receive up to $1.3 billion in potential payments based upon the successful achievement of specified research, development, regulatory, and commercial milestones across up to five potential programs. In addition, Vertex will pay tiered royalties on future net sales on any products that may result from this collaboration. Specific diseases that are the subject of this collaboration are not disclosed.

On April 22, 2021 Illumina, Inc. (NASDAQ: ILMN) and Kartos Therapeutics, Inc. reported a new partnership to co-develop a TP53 companion diagnostic (CDx) based on the content of Illumina’s comprehensive genomic profiling assay, TruSight Oncology 500 (TSO 500) (Press release, Illumina, APR 22, 2021, View Source [SID1234578379]). This companion diagnostic for multiple hematologic indications will be the first to use TSO 500 with peripheral whole blood as a diagnostic sample type.

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"With this partnership, Illumina will expand the TruSight Oncology offerings into hematologic malignancies," said Phil Febbo, MD, Chief Medical Officer of Illumina. "By leveraging our technology and harnessing the expertise at Kartos, we continue to advance Illumina’s commitment to develop standardized, globally distributable tools for precision oncology."

The initial focus of the collaboration will be the co-development of multiple CDx claims in blood cancers for Kartos’ KRT-232, a potent and selective oral MDM2 inhibitor that activates p53 to drive tumor cell death in TP53 wild-type cancers.

"Kartos is dedicated to developing novel, targeted therapeutics that meaningfully improve the lives of patients with cancer," said Jesse McGreivy, MD, Chief Executive Officer and Chief Medical Officer at Kartos. "This partnership will allow us to capitalize on TSO 500 as we explore the expanded use of KRT-232, which offers a unique mechanism to restore the function of p53, one of the most critical tumor suppressor proteins, resulting in apoptosis of malignant cells across a variety of hematologic and solid tumor types."

The collaboration with Kartos builds on a solid history and varied portfolio of Illumina’s oncology partnerships with industry leaders, with the united goal of advancing cancer diagnostics and precision medicine.

About TruSight Oncology 500

TSO 500 is a Research Use Only comprehensive pan-cancer assay designed to identify 523 known and emerging tumor biomarkers. TSO 500 utilizes both DNA and RNA from tumor samples to identify key variants critical for cancer development and progression, such as small DNA variants, fusions, and splice variants. Based on the content of TSO 500, Illumina will be adding an in vitro diagnostic (IVD) test to the TruSight Oncology product family. This comprehensive tumor profiling assay will have similar chemistry and analytics to TSO 500.

On April 22, 2021 NRG Oncology reported that The first National Institutes of Health (NIH) National Cancer Institute (NCI)-funded clinical study examining stereotactic body radiotherapy (SBRT) in the treatment of oligometastatic breast, prostate, and non-small cell lung (NSCLC) cancers displayed evidence that SBRT can be safely used to treat patients who have multiple metastases (Press release, NRG Oncology, APR 22, 2021, View Source [SID1234578397]). These results were recently published in JAMA Oncology.

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The results of the Phase I NRG-BR001 trial, conducted by the NCI National Clinical Trials Network group NRG Oncology, indicate that SBRT treatment in standard doses was safe for 35 evaluable patients with a median of 3 metastases. There were no dose-limiting toxicities and over 50% of trial participants were alive at 2 years following treatment.

"Prior to this trial, little to no evidence was available to support that SBRT is a safe and tolerable treatment option for patients who have multiple metastases. Researchers have hypothesized that SBRT could improve survival outcomes for this patient population; however, it was imperative we determine the safety of this procedure, appropriate dose and scheduling, and how to coordinate across multiple centers the quality assurance of the procedures prior to testing its efficacy," stated Steven J. Chmura, MD, PhD, of the Department of Radiation and Cellular Oncology at the University of Chicago Comprehensive Cancer Center and the lead author of the NRG-BR001 manuscript. "To ensure safety, this trial used an extensive radiation QA process to test the accuracy of treating moving tumors and was the first NRG trial to require the use of 3D image guidance during treatment for soft tissue tumors."

NRG-BR001 enrolled up to 6 evaluable patients for each of the following 7 selected anatomic locations: bone/osseous (BO), spinal/paraspinal (SP), peripheral lung (PL), central lung (CL), abdominal/pelvic (AP), mediastinal/cervical lymph node (MC), and liver (L).As a single patient could contribute to more than one location, the safety question was able to be answered with 35 evaluable patients from the 42 enrolled trial participants. Patients were required to have 3-4 metastases or 2 metastases in close proximity to each other. SBRT starting dose was 50 GY over 5 fractions for the CL and MC groups, 45 GY over 3 fractions for the PL, AP, and L groups and 30 Gy over 3 fractions for the BO and SP group. Additional patients would be accrued as needed at defined de-escalated doses if any of the starting doses were not deemed to be safe.

The 35 evaluable patients had breast (n=12), NSCLC (n=10), and prostate (n=13) cancers. No dose de-escalations were needed. There were 8 instances of grade 3 adverse events. There were no treatment-related deaths.

SBRT for multiple metastases is now utilized in multiple ongoing Phase II and III NCI-sponsored trials. Follow-up research should be done in long surviving oligometastatic patients.

"These are important data from the multicenter study, confirming that complicated stereotactic body radiotherapy to multiple sites is safe and feasible. We eagerly await the results of ongoing, larger randomized trials to demonstrate how effective this is when compared to drug therapy alone for metastatic cancer," stated Mitchell Machtay, MD, the Associate Dean for Clinical Cancer Research at the Penn State College of Medicine and the interim Group Chair for NRG Oncology.

NRG Oncology BR001 was supported by grants UG1CA189867 (NRG Oncology NCORP), U10CA180868 (NRG Oncology Operations), U10CA180822 (NRG Oncology SDMC), U24CA180803 (IROC) from the National Cancer Institute (NCI).

Citation
Chmura S, Winter KA, Robinson C, Pisansky TM, Borges V, Al-Hallaq H, Matuszak M, Park SS, Yi S, Hasan Y, Bazan J, Wong P, Yoon HA, Horton J, Gan G, Milano M, Sigurdson ER, Moughan J, Salama JK, White J. The Safety of Stereotactic Body Radiotherapy (SBRT) for the Treatment of Multiple Metastases: Findings from the NRG Oncology NRG-BR001. JAMA Oncol. doi:10.1001/jamaoncol.2021.0687. [Epub ahead of print].

On April 22, 2021 Certara, Inc. (Nasdaq: CERT), a global leader in biosimulation, reported that it will release preliminary financial results for the first quarter of 2021 after the market close on Thursday, May 6, 2021 (Press release, Certara, APR 22, 2021, View Source [SID1234578348]). Company management will host a conference call to discuss financial results at 5:00 p.m. ET.

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Investors interested in listening to the conference call may do so by dialing (833) 360-0946 for domestic callers or (914) 987-7661 for international callers, followed by Conference ID: 9802129. A live and archived webcast of the event will be available on the "Investors" section of the Certara website at View Source

On April 22, 2021 Biodesix, Inc. (Nasdaq: BDSX), a leading data-driven diagnostic solutions company with a focus in lung disease, reported a webinar to discuss biomarkers and risk assessment in lung nodule management (Press release, Biodesix, APR 22, 2021, View Source [SID1234578364]). The virtual session, hosted by the American College of Chest Physicians (CHEST) and led by Susan Garwood, MD, National Physician Director of Pulmonary Disease at HCA Healthcare, is titled, "Using biomarker-based risk assessment to inform lung nodule referral decisions" and scheduled for 2:30 p.m. CT on Monday, April 26, 2021. Participants are encouraged to register using the following Zoom registration link: View Source

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Due to more frequent chest imaging for general diagnostic purposes, lung nodules are increasingly detected incidentally at various points of care throughout the healthcare system. In this webinar, Dr. Garwood will discuss identification of malignant lung nodules in a distributed population and how risk assessment using blood-based biomarker testing can help standardize referral patterns by stratifying patients who can be safely monitored with computerized tomography (CT) surveillance and those that should be referred to a specialist for diagnostic intervention.

"As an advanced bronchoscopist, I don’t want to miss an opportunity to diagnose lung cancer at an early stage where there is a substantial survival advantage," said Dr. Garwood.

"As an advanced bronchoscopist, I don’t want to miss an opportunity to diagnose lung cancer at an early stage where there is a substantial survival advantage," said Dr. Garwood. "Most lung nodules are benign, so it can be challenging to determine which patients should be evaluated for a diagnostic procedure. In order to increase referral volume, I must show responsible handling of nodules to my referral sources to have an opportunity to find lung cancer early. Education on risk assessment is crucial because it allows providers to confidently monitor low-risk patients with serial surveillance CT, while prioritizing indeterminate and high-risk patients for referral to advanced diagnostic centers."

"As an advanced bronchoscopist, I don’t want to miss an opportunity to diagnose lung cancer at an early stage where there is a substantial survival advantage," said Dr. Garwood. "Most lung nodules are benign, so it can be challenging to determine which patients should be evaluated for a diagnostic procedure. In order to increase referral volume, I must show responsible handling of nodules to my referral sources to have an opportunity to find lung cancer early. Education on risk assessment is crucial because it allows providers to confidently monitor low-risk patients with serial surveillance CT, while prioritizing indeterminate and high-risk patients for referral to advanced diagnostic centers."

Dr. Garwood is a pulmonologist practicing in Nashville, TN and is affiliated with multiple HCA facilities including Tri-Star Centennial Medical Center where she practices in a multi-specialty practice with thoracic surgery and interventional pulmonary. She completed her pulmonary training at the Medical University of South Carolina.

CHEST is a global leader in the prevention, diagnosis and treatment of chest diseases and serves as an essential resource for its 19,000+ members from around the world who provide patient care in pulmonary, critical care and sleep medicine. The journal CHEST features the best in peer-reviewed, cutting-edge original research in the multidisciplinary specialties of chest medicine, including CHEST’s highly regarded clinical practice guidelines and consensus statements.