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Dr. Thomas Bock, CEO However, precision medicines have an average response rate of less than 50-percent, and only 15 percent of cancer patients carry an actionable biomarker and target for developing new precision medicines.
This is where Notable, a clinical stage therapeutics company, is changing the equation for cancer patients. The company’s tech-bio platform—Predictive Precision Medicine Platform (PPMP)— predicts the efficacy of a given therapy for patients without the need for biomarkers and ensures patients can receive the best treatment for them individually.
“By bio-simulating a patient’s cancer treatment on our platform, we identify patients predicted to respond to the drug in the real world,” says Dr. Thomas Bock, CEO of Notable.
The PPMP co-processes treatments with a patient’s blood or bone marrow sample under proprietary conditions, and measures the response of both cancer and normal cells over several days. Notable’s automated high-throughput workcells collect many layers of imaging and other data, integrating hundreds of thousands of data points from single cells, from which Notable’s digital algorithms, developed over years through virtuous learning cycles, establish whether the patient will clinically respond or not. Three consecutive clinical validation studies across AML and MDS, in collaboration with Stanford, MD Anderson Cancer Center, and Texas Children’s Hospital, have demonstrated an 83-100 percent precision in accurately predicting clinical responders, which would translate into an 83-100 percent response rate in medical practice or dug development applications of the platform.
Notable partners with biotech, pharmaceutical, and academic institutions in the oncology space. As a drug development tool, its platform helps identify clinical responders before treatment and conduct trials that selectively enroll predicted responders.
As a leader in developing the new class of predictive precision medicines for cancer, Notable’s high-throughput automated lab is GAP-accredited and continuously growing with every patient sample through machine learning. The company fosters partnerships with more than a dozen pharmaceutical companies in all R&D stages—from preclinical lead identification to late clinical development - to predict the efficacy of candidate drugs. For instance, Notable tested drugs for two pharmaceutical partners and identified the efficacies across patient populations, to help focus on the most promising drugs and populations, saving time and costs. In 2021, Notable started to in-license and co-develop two clinical stage hematology drugs, initiating a growing pipeline of novel precision medicines that are developed selectively in predicted clinical responders – precision medicines.
Currently focused on redefining the treatment for hematological cancer, Notable plans to expand and address the most pressing needs of patients across hematology and oncology, advancing predictive precision medicine together with a fast-growing number of academic and industry partners.
Company
Notable Labs
Management
Dr. Thomas Bock, CEO
Description
Notable is transforming the historical standards of care by ensuring dramatic impact for patients with its Predictive Precision Medicine Platform. They are revolutionizing how patients seek and receive treatment, and how new cancer medicines are developed, envisioning that each patient will receive the precise treatment that is most effective for them personally.
“We are very pleased to share the result of our innovative sample handling study with the cancer community at AACR 2024. The ability to derive a high-quality assessment of the response to treatment based on the results of cryopreserved samples is an important innovation. It has been previously thought that frozen samples could not be effectively thawed and used for cell viability assays. Based on the data presented at AACR, Notable now has the potential to develop partnerships to assess new treatment regimens, including combination therapies, using Notable’s PPMP platform and archived, cryopreserved samples thawed according to our protocols and proprietary media compositions,” said Thomas Bock, M.D., Chief Executive Officer of Notable. “We hope this opens new opportunities to apply our PPMP to a broad range of archived samples and therapeutics to unlock value and new treatment options for patients with cancer.”
Joseph Wagner, Ph.D., Chief Scientific Officer of Notable, added, “As we are ready to initiate the Phase 2 program with volasertib in Q2 2024, we are dedicated to maintaining a focus on innovation for our PPMP. The careful evaluation of sample handling protocols, presented at AACR 2024, makes our PPMP more comprehensive and enhances the opportunity to study and apply it more broadly. As we continue to execute, we look forward to further demonstrating the potential of PPMP and partner with biopharmaceutical companies and innovators to provide potential new treatment options for patients with cancer.”
Summary of Data and Findings for Notable’s Sample Handling Study:
Overview: The study was designed to evaluate the potential use of cryopreserved samples as an alternative to fresh samples for predicting treatment response in acute myeloid leukemia (AML) using Notable’s PPMP.
• Cryopreserved samples are known for exhibiting low sample viability and cell recovery, rendering their practical utilization challenging
• Notable adapted an existing methodology to process cryopreserved patient-derived leukemia cells to investigate their use as an alternative to fresh samples in the PPMP assay
Method: First, a method was established using freshly isolated white blood cells (WBCs) from AML patients (<2 days from sample collection). A fraction of these cells was cryopreserved. Both fresh and cryopreserved cells (after thawing and culturing) were assessed on the PPMP and results compared. Second, the established method was applied to cryopreserved samples (from a biobank) collected from AML patients prior to their treatment with chemotherapy (cytarabine plus idarubicin). Results obtained on the PPMP were compared to the clinical responses experienced by the patients after treatment with cytarabine and idarubicin.
Results: For the method development study, ex vivo responses to 3 different compounds (venetoclax, 5-azacytidine and decitabine) were assessed after culturing of the cryopreserved cells for 24- and 48-hour post thaw using two different media (“A” and “B”). There was a strong correlation (R2 from 0.77 to 0.92) for venetoclax and 5-azacytidine between the results obtained with fresh vs. frozen samples. For the biobank samples treated with cytarabine and idarubicin, culture media A and B show a similar dose-response behavior. Importantly, the PPMP correctly predicted clinical responders (complete remission) and non-responders (refractory).
While Notable will continue to evaluate and validate these sample handling methodologies, these data open the door to the use of cryopreserved samples from retrospective cohorts.