On September 29, 2022 Gamida Cell Ltd. (Nasdaq: GMDA), the leader in the development of NAM-enabled cell therapies for patients with hematologic and solid cancers and other serious diseases, reported the presentation of new long term follow-up data and health-related quality of life scores of patients treated with omidubicel at the Tenth Annual Meeting of the Society of Hematologic Oncology (SOHO), being held in Houston, Texas (Press release, Gamida Cell, SEP 29, 2022, View Source [SID1234621567]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"These data reinforce our commitment to advance transformational cell therapy research and underscore the potential of our NAM technology platform. Our lead stem cell therapy candidate, omidubicel, addresses the unmet need for patients with hematologic malignancies, demonstrated by the robust and growing body of encouraging clinical evidence, including the long-term follow up data and quality of life improvement," said Ronit Simantov, M.D., Chief Medical Officer of Gamida Cell. "As we approach the PDUFA date of January 30, 2023, and upon potential FDA approval, we are prepared to execute our plan that ensures access to those patients who can benefit from omidubicel as quickly as possible."

The long-term, durable clinical benefit of omidubicel was observed at three years across a patient population that typically has a poor prognosis. A study titled, "Multicenter Long-Term Follow Up of Allogeneic Hematopoietic Stem Cell Transplantation with Omidubicel: A Pooled Analysis of Five Prospective Clinical Trials," highlighted long-term follow-up of 105 patients transplanted with omidubicel between 2006-2020 (median follow-up of 22 months). The data demonstrated an overall survival and disease-free survival of 63% (95% CI, 53%-73%) and 56% (95% CI, 47%-67%) at three years, respectively, as well as durable long-term hematopoiesis and immune competence. Learn More

Overall well-being health-related quality of life scores for patients treated with omidubicel demonstrated clinical benefit compared to standard of care. A study titled, "Health-Related Quality of Life Following Allogeneic Hematopoietic Stem Cell Transplantation with Omidubicel Versus Standard Umbilical Cord Blood" featured an analysis of 108 patients that completed validated health-related quality of life (HRQL) surveys on screening and days 42, 100, 180, and 365 post-transplant. Measures of physical and functional well-being and other HRQL scores were more favorable with omidubicel. These data suggest clinically meaningful and sustained improvements in physical, functional, and overall well-being compared to umbilical cord blood transplantation. Learn More

About NAM Technology

Our NAM-enabling technology is designed to enhance the number and functionality of targeted cells, enabling us to pursue a curative approach that moves beyond what is possible with existing therapies. Leveraging the unique properties of NAM (nicotinamide), we can expand and metabolically modulate multiple cell types — including stem cells and natural killer cells — with appropriate growth factors to maintain the cells’ active phenotype and enhance potency. Additionally, our NAM technology improves the metabolic fitness of cells, allowing for continued activity throughout the expansion process.

About Omidubicel

Omidubicel is an advanced cell therapy candidate developed as a potential life-saving allogeneic hematopoietic stem cell (bone marrow) transplant for patients with blood cancers. Omidubicel demonstrated a statistically significant reduction in time to neutrophil engraftment in comparison to standard umbilical cord blood in an international, multi-center, randomized Phase 3 study (NCT0273029) in patients with hematologic malignancies undergoing allogeneic bone marrow transplant. The Phase 3 study also showed reduced time to platelet engraftment, reduced infections and fewer days of hospitalization. One-year post-transplant data showed sustained clinical benefits with omidubicel as demonstrated by significant reduction in infectious complications as well as reduced non-relapse mortality and no significant increase in relapse rates nor increases in graft-versus-host-disease (GvHD) rates. Omidubicel is the first stem cell transplant donor source to receive Breakthrough Therapy Designation from the FDA and has also received Orphan Drug Designation in the US and EU.

Omidubicel is an investigational stem cell therapy candidate, and its safety and efficacy have not been established by the FDA or any other health authority. For more information about omidubicel, please visit View Source

On September 29, 2022 Meliora Therapeutics, a biotech company pioneering a machine learning-driven platform to decipher oncology drugs’ true mechanisms-of-action, reported the close of an $11M seed financing round led by HOF Capital and ZhenFund, with participation from Obvious Ventures, Village Global, BrightEdge (the impact investment arm of the American Cancer Society), Pebblebed (fund of Keith Adams, former Chief Architect at Slack), Axial, Olive Capital, and prominent individual investors Michael Polansky (co-founder of Parker Institute for Cancer Immunotherapy and ArsenalBio), Wes Sterman (founding investor for Gilead), and others (Press release, Meliora Therapeutics, SEP 29, 2022, View Source [SID1234621566]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Founded in September 2021, Meliora’s computational platform enables researchers to develop a precise understanding of a drug’s true mechanism of action (MOA) based on data and not guesswork. The company aims to improve the current success rate of oncology drugs in development by building the industry’s first atlas of drug mechanisms and their impact on cancer biology through the use of machine learning. The founding team includes David Li, CEO & Co-Founder, Jason Sheltzer, Ph.D., Scientific Co-Founder and Joan Smith, Technical Co-Founder.

Out of all the areas of medicine, clinical trials in oncology have the absolute lowest success rate—3%. One reason for the high failure rate is that many cancer drugs in development have an incorrect characterization of their mechanism of action, according to a paper published by Meliora co-founder Jason Sheltzer, Ph.D., in Science Translational Medicine.

"Modern cancer drug development is often not mechanism driven, leading to clinical development failures down the line. Meliora is using a novel molecular fingerprint matching method to determine the true mechanism of action of precision cancer therapies," said David Li, CEO & Co-Founder, Meliora Therapeutics. "By using machine learning to connect MOA with specific therapeutic molecules, we will increase the overall probability of success. We’re thrilled to have the support of this top-tier investor syndicate, and the funds will be used to expand our team and accelerate development of our proprietary computational platform."

"We believe AI will bring revolutionary changes to the drug discovery industry, and are impressed by Meliora’s approach to develop a mechanism fingerprint atlas. We are glad to have the opportunity to partner with the amazing team at Meliora," said Anna Fang, CEO and founding partner at ZhenFund.

"Meliora’s computational platform allows companies to identify the true mechanism of action in small molecule compounds, and uncover mischaracterized compounds in the process," said Jason Sheltzer, Ph.D., Co-Founder, Meliora Therapeutics. "By expanding the therapeutic window of these molecules with known safety profiles, the Meliora team has built a flywheel of precision medicine oncology assets with a higher potential for clinical success by understanding how the drug really works."

"We are excited about Meliora’s machine-learning-based approach to mechanism-driven drug discovery. We believe that their novel approach will be a game-changer in the oncology drug development space," said Fady Yacoub, Co-Founder, Managing Partner at HOF Capital.

"With better knowledge of a drug’s unique mechanism, scientists can improve drug discovery and increase the success rate of precision therapies in oncology. Additionally companies may also find new vulnerabilities in cancer cells to target or new ways to identify patients who are most likely to respond to a particular targeted therapy. We believe Meliora’s novel approach will be highly impactful in the oncology drug development space," said Michael Polansky, co-founder of Parker Institute for Cancer Immunotherapy and ArsenalBio.

Alice Pomponio, Managing Director of BrightEdge, the impact investment arm of the American Cancer Society (ACS) said, "We celebrate the achievements of our ACS grantees and now, through BrightEdge, we are deepening our innovation acceleration capabilities that enable scientific founders like Dr. Jason Sheltzer to translate their research into emerging biotech companies that accelerate ACS’s mission to defeat cancer and advance health equity."

The seed funding will be used to expand Meliora’s computational platform, which includes building a proprietary data moat and selecting mischaracterized scaffolds as strong starting points for its lead precision therapeutic programs. The company has already built a computational prototype which identifies the correct mechanism of action (MOA) for small molecule drugs and generated in-vitro wet lab data for dozens of compounds computationally identified as having an alternate mechanism of action, with many of these compounds generating strong potency against the in silico predicted target. The funding will also be used to expand the team, operations, and build industry partnerships.

On September 29, 2022, MAIA Biotechnology, Inc., (NYSE American: MAIA) ("MAIA", the "Company"), a targeted therapy, immuno-oncology company focused on developing potential first-in-class oncology drugs, reported that it will present the results of a study of the anticancer agent 6-thio-dG (THIO) in hepatocellular carcinoma (HCC) in vitro and in vivo models at the EORTC-NCI-AACR (Free EORTC-NCI-AACR Whitepaper) (ENA) Symposium on Molecular Targets and Cancer Therapeutics. The symposium is taking place Oct. 26-28, 2022, in Barcelona, Spain (Press release, MAIA Biotechnology, SEP 29, 2022, View Source [SID1234621565]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The data to be presented outline high anticancer activity of THIO in HCC cancer cells. Notably, sequential administration of THIO followed by cemiplimab (cemi) demonstrated enhanced antitumor efficacy, including complete responses, in a syngeneic immunocompetent HCC mouse model, in comparison with either single agent used alone. Moreover, the treated tumor-free mice demonstrated a complete rejection of the same tumor type cells upon re-challenge: anticancer immune memory was confirmed.

MAIA received an Orphan Drug Designation from the US FDA for the treatment of HCC with THIO earlier in 2022. HCC currently makes up around 90% of liver cancer cases; by 2025, the global incidence of liver cancer is expected to eclipse 1 million cases.1

Presentation details:

Presentation title: The novel, telomerase-directed, telomere-targeted, anticancer agent 6-thio-dG (THIO) demonstrates potent activity and induces antitumor immunity in hepatocellular carcinoma (HCC) models
Abstract number: 86
Session title: New Drugs
Date: Wednesday, Oct. 26 at 12:00pm CEST
Presenting author: Sergei Gryaznov, Ph.D., Chief Scientific Officer, MAIA Biotechnology
Location: Exhibition hall
Additional meeting information is available on ENA’s website.

About THIO

THIO (6-thio-dG or 6-thio-2’-deoxyguanosine) is a telomere-targeting agent currently in clinical development to evaluate its activity in non-small cell lung cancer (NSCLC), in sequential administration with cemiplimab (Libtayo), a PD-1 inhibitor developed by Regeneron. Telomeres play a fundamental role in the survival of cancer cells and their resistance to current therapies. THIO is being developed as a second or higher line of treatment for NSCLC for patients that have progressed beyond the standard-of-care regimen of existing checkpoint inhibitors.

On September 29, 2022 Ultivue, Inc. an industry leader in multiplexing tools and novel image analysis solutions for tissue biomarker studies and, the Translational Research Unit (TRU) platform of University of Bern, that develops novel workflows for tissue microarray (ngTMA) construction, reported a collaborative agreement to promote multiplexed immunofluorescence (mIF) assays and the use of next generation TMAs to unlock spatial analysis in scientific research and improve the quality of TMAs integrated in clinical and translational research (Press release, Ultivue, SEP 29, 2022, View Source [SID1234621564]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Ultivue develops unique solutions for use in mIF applications, imaging, and spatial phenomics. Its proprietary InSituPlex technology enabling improved signal to noise data is designed for fast and comprehensive exploration of biologically relevant targets, up to 12-plex, with same slide-H&E analysis in tissue samples combines the power of computational pathology & spatial biology to guide translational science in immuno-oncology.

ngTMA supports research collaborators with an interdisciplinary approach to studying tumor biology and disease states. Services include scientific consulting and TMA design, digital slide annotation, automated tissue punching and documentation for quality control. The ngTMA platform is embedded in an innovative research framework and as part of the Translational Research Unit (TRU), Institute of Pathology, University of Bern, with expertise in tissue-related technologies.

"We are thrilled to be partnering with ngTMA to jointly empower biopharma customers with tissue multiplex services. In addition, our scientific collaboration will investigate the relevance of the tumor microenvironment by characterizing clinically well-annotated cohorts using Ultivue reagents at ngTMA," said Florian Leiss, Ph.D. Vice President Product Strategy & Corporate Development at Ultivue.

"We are very excited to join forces with the Ultivue team and have their reagents in our pipeline in order to support translational research of our collaborators," Said Paulina Brönnimann, PhD, Head of Translational Research Unit.

On September 29, 2022 Propanc Biopharma, Inc. (OTC Pink: PPCB) ("Propanc" or the "Company"), a biopharmaceutical company developing novel cancer treatments for patients suffering from recurring and metastatic cancer, reported that Chief Scientific Officer and Co-Founder, Dr Julian Kenyon, MD, MB, ChB, explains why pancreatic and ovarian cancers are selected as the primary target therapeutic indications for planned PRP human studies (Press release, Propanc, SEP 29, 2022, View Source [SID1234621563]). According to Dr Kenyon, target indications were selected based on in vitro and in vivo data, as well as clinical observations from a compassionate use study investigating the effects of two proenzymes, trypsinogen and chymotrypsinogen against a range of malignant tumors. Overall, proenzymes appeared to exert significant effects against more aggressive, less differentiated tumor types, like pancreatic and ovarian tumors. Patients from the compassionate use study suffering from cancers of the GI tract, or endocrine tumors, such as pancreatic and ovarian cancers, benefited most from treatment. The world market for pancreatic and ovarian cancer drugs is projected to grow to $4.2 Billion in 2025 according to Grandview Research and $10.1 Billion by 2027 according to iHealthcareAnalyst, respectively, resulting in a combined global market of $14.3 Billion over the next 5-year period.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Extensive laboratory analysis confirmed that PRP reduced the main characteristics of cancer spread, namely angiogenesis (blood vessel formation), which is a critical step in tumor development, as well as the spreading of tumor metastases. In addition, assays revealed that the migration capacity of ovarian, pancreatic, melanoma and colon cancer cells was suppressed after incubation with PRP. Furthermore, evidence suggests the epithelial to mesenchymal transition (EMT), a biological process associated with wound healing and cell migration, which causes cancer stem cells (CSCs) to become motile and invasive, is associated with metastasis and inducing drug resistance in many cancers, such as pancreatic and ovarian cancers. Studies in pancreatic and cancer cell lines after PRP treatment demonstrated a significant reduction in EMT markers and genes and in fact, a reversal of the EMT process so that CSCs become benign and less resistant to standard treatments.

The in vivo effects of PRP at different doses on tumor weight in implanted pancreatic and ovary tumors was evaluated. In the pancreatic tumor model, there was significant reduction in mean tumor weight in animals treated for 26 days with PRP with more than 85% tumor growth inhibition compared with the control. Furthermore, ovary tumor-bearing mice showed a significant reduction in mean tumor weight in animals treated for 21 days with two different doses of PRP, resulting in a 46 – 52% tumor growth inhibition compared with the control.

The clinical efficacy of a suppository formulation containing bovine pancreatic proenzymes trypsinogen and chymotrypsinogen was evaluated in the context of a UK Pharmaceuticals Special Scheme and the results were published in Scientific Reports. Clinical effects were studied in 46 patients with advanced metastatic cancers of different origin (prostate, breast, ovarian, pancreatic, colorectal, stomach, non-small cell lung, bowel cancer and melanoma) after treatment with a rectal formulation of both pancreatic proenzymes. No severe or serious adverse events related to the rectal administration were observed. Patients did not experience any hematological side effects as typically seen with classical chemotherapy regimens.

In order to assess the therapeutic activity, overall survival of patients under treatment was compared to the life expectancy assigned to a patient prior to treatment start. Nineteen from 46 patients (41.3%) with advanced malignant diseases, most of them suffering from metastases, had a survival time significantly longer than their expected, in fact, for the whole set of cancer types, mean survival (9.0 months) was significantly higher than mean life expectancy (5.6 months). In the case of pancreatic and ovarian cancers, 2 from 4 pancreatic cancer patients and 4 from 7 ovarian cancer patients significantly exceeded life expectancy.

As a result of the extensive studies undertaken, particularly in pancreatic cancer, the Company applied for and received Orphan Drug Designation (ODD) from the US Food and Drug Administration (USFDA) for the use of its lead product, PRP, for the treatment of pancreatic cancer. The approved indication is one of the most lethal malignancies with a median survival of 6 months and a 5-year survival rate of less than 5%. The lethal nature of this disease stems from its propensity to rapidly disseminate to the lymphatic system and distant organs, and is a major unmet medical issue. Under the Orphan Drug Act (ODA), drugs, vaccines, and diagnostic agents qualify for orphan status if they are intended to treat a disease affecting less than 200,000 American citizens. Under the ODA, orphan drug sponsors qualify for seven-year FDA-administered market Orphan Drug Exclusivity (ODE), tax credits of up to 50% of R&D costs, R&D grants, waived FDA fees, protocol assistance and may get clinical trial tax incentives.

"Over the past 15 years, our extensive research has uncovered a truly unique and exciting technology that selectively targets and eradicates cancer stem cells, whilst leaving healthy cells alone, making it less toxic compared with standard treatment approaches," said Dr Kenyon. "Furthermore, our technology appears to be effective against more aggressive, less differentiated tumor types where few treatment options exist, and prognosis is poor, especially in the case of pancreatic and ovarian cancers. I look forward to advancing PRP to human studies where we can fully assess the clinical efficacy of PRP in a controlled setting."

Propanc plans to undertake a First-In-Human study in 30 to 40 advanced cancer patients suffering from solid tumors to determine a maximum tolerated dose for PRP treatment, followed by two proof of concept studies in pancreatic and ovarian cancers, 60 patients in each study, to confirm the clinical efficacy of PRP in the selected target therapeutic indications.

PRP is a mixture of two proenzymes, trypsinogen and chymotrypsinogen from bovine pancreas administered by intravenous injection. A synergistic ratio of 1:6 inhibits growth of most tumor cells. Examples include kidney, ovarian, breast, brain, prostate, colorectal, lung, liver, uterine and skin cancers.