On April 21, 2022 Moleculin Biotech, Inc., (Nasdaq: MBRX) ("Moleculin" or the "Company"), a clinical stage pharmaceutical company with a broad portfolio of drug candidates targeting highly resistant tumors and viruses, reported that the US Food and Drug Administration (FDA) is allowing the Company’s Investigational New Drug (IND) application to study WP1066 for the treatment of recurrent malignant glioma (Press release, Moleculin, APR 21, 2022, View Source [SID1234612710]). With this IND now cleared, Moleculin plans to evaluate strategic partnerships and collaborations to conduct a Phase 1 open label, single arm, dose escalation study of the safety, pharmacokinetics and efficacy of oral WP1066 in adult patients with recurrent malignant glioma.

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WP1066 is the Company’s flagship Immune/Transcription Modulator designed to stimulate the immune response to tumors by inhibiting the errant activity of Regulatory T-Cells (TRegs) while also inhibiting key oncogenic transcription factors, including p-STAT3 (phosphorylated signal transducer and activator of transcription 3), c-Myc (a cellular signal transducer named after a homologous avian virus called Myelocytomatosis) and HIF-1α (hypoxia inducible factor 1α). These transcription factors are widely sought targets that are believed to contribute to an increase in cell survival and proliferation, and the angiogenesis (coopting vasculature for blood supply), invasion, metastasis and inflammation associated with tumors. They may also play a role in the inability of immune checkpoint inhibitors to affect more resistant tumors.

"WP1066 has demonstrated significant anti-tumor activity in a wide range of tumor cell lines and increased survival in a number of animal models to-date. Additionally, the preliminary results demonstrated in the ongoing trial of WP1066 for pediatric brain tumors bolster our confidence and this IND clearance provides further momentum for its continued research and development," commented Walter Klemp, Chairman and CEO of Moleculin. "We expect the clearance of this IND to further support the ongoing pediatric studies being conducted by the team at Emory University, and we are evaluating the potential for additional externally funded investigator-initiated studies."

WP1066 is currently being evaluated in collaboration with Emory University for the treatment of pediatric brain tumors, including Diffuse Interstitial Pontine Glioma (DIPG). The Emory trial for pediatric brain tumors has now treated three subjects in the first two cohorts of the Phase 1 dose escalation portion of physician-sponsored clinical trial. In that trial, one of the subjects in the first cohort with DIPG showed an apparent response to the treatment with both clinical improvement and radiologic reduction of tumor size. The Company cautions that this is preliminary data, and no conclusions should be drawn from this single event. One subject has been treated in the third cohort at the dose level of 8mg/kg. Two more subjects will be treated at this dose level. Emory University has amended its protocol to allow dosing at 16 mg/kg after these two additional subjects have been dosed, and the third cohort dosing has been deemed safe.

The Company has received Orphan Drug Designation for WP1066 for the treatment of brain tumors, as well as Rare Pediatric Disease designation for three other pediatric indications. Additionally, WP1066 + radiation is being evaluated, pre-clinically, in the treatment of Glioblastoma Multiforme (GBM).

Glioma is a common type of tumor originating in the brain. About 33% of all brain tumors are gliomas, which originate in the glial cells that surround and support neurons in the brain, including astrocytes, oligodendrocytes and ependymal cells. GBM is the most aggressive malignant primary brain tumor and remains as an incurable tumor with a median survival of only 15 months[1]. It is the most common malignant primary brain tumor making up 54% of all gliomas and 16% of all primary brain tumors,[2] and despite advancements, survival rates for patients with GBM have shown no notable improvement in population statistics in the last three decades.[3] The average annual age-adjusted incidence rate of GBM is 3.19 per 100,000 persons in the United States.[4]

On April 21, 2022 MaaT Pharma (EURONEXT: MAAT – the "Company"), a French clinical-stage biotech and a pioneer in the development of microbiome-based ecosystem therapies dedicated to improving survival outcomes for patients with cancer reported on March 28th, 2022, the inclusion of the first patient in a pivotal Phase 3 clinical trial, potentially the last step of clinical development before marketing authorization, evaluating MaaT013 for the treatment of acute gastrointestinal Graft-versus-Host Disease (aGvHD), a serious complication following stem cell transplantation (Press release, MaaT Pharma, APR 21, 2022, View Source [SID1234612709]). Every year, more than 10,000 people are diagnosed in Europe and in the United States of America[1], and the prognosis for the patients with the most advanced form is poor with a mortality rate of up to 80%[2] in the first year. This new milestone for MaaT Pharma, the first Euronext-listed company developing microbiome-based therapies, builds on the success story of its founding partnership with INRAE initiated in 2014, in particular via the scientific support agreement[3] between MaaT Pharma, INRAE and Dr. Joël Doré, INRAE Research Director, Scientific Director of Métagénopolis, author of nearly 500 publications, and one of the world’s most cited authors in the microbiome sphere today.

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At the end of 2014, following a project development period as an entrepreneur-in-residence with Seventure Partners, Hervé Affagard, currently CEO, founded MaaT Pharma alongside Dr. Joël Doré, now MaaT Pharma’s Scientific Advisor. As early as 2015, MaaT Pharma and INRAE co-filed three patents, for which the Company now holds exclusive exploitation rights. These patents are also at the root of the development of the Company’s native products, MaaT013 and MaaT033, both currently in clinical trials.

"Microbiome science has been at the heart of my research for more than 30 years. INRAE has established itself first as a pioneer, then as an international center of excellence in the field. In 2021, for example, INRAE was the 5th largest academic player in the world in terms of microbiome-related patents. In my opinion, the rapid growth of MaaT Pharma, which has entered a Phase 3 clinical trial, perfectly illustrates the efficient and successful transition from pioneering science to innovative therapeutic development for the benefit of patients. Through my research, I hope to contribute to the fast emergence of a new medicinal modality, that takes into account the symbiotic relationship between human health and the microbiome," said Dr. Doré.

Following the advent of metagenomics[4], the research produced at INRAE on the gut microbiome has led to unprecedented results disrupting our understanding of the digestive ecosystem, and opening novel avenues for improving the health of millions of patients worldwide suffering from cancers, metabolic diseases, or inflammatory diseases to name just a few of the potential indications.

MaaT Pharma has developed a portfolio of three products since its creation including two currently in clinical phases. The Company has initiated five Phase 1 and Phase 2 clinical trials, consolidated its patent portfolio (including 13 international patent families), and raised a total of €83 million. The Company currently employs 45 people and MaaT013 has been administered to more than 100 people with aGvHD in a Phase 2 clinical trial and in an ongoing compassionate access program[5] in France.

Hervé Affagard, CEO and co-founder of MaaT Pharma commented, "Since our foundation, we have been looking to develop the microbiome ecosystem in France and have forged partnerships with medical and scientific centers of excellence as well as with renowned industrial players to develop the Company. In 2016, we launched our first clinical trial just 18 months after the company’s creation, and we have since strengthened our position as a pioneer in microbiome-based therapies in oncology. Our translational expertise allows us to rapidly transform science into groundbreaking and safe drug candidates, with the support of our scientific partners, to improve survival outcome for cancer patients."

The collaboration between these two pioneers continues to move forward with the formation of industry structures and the growth of national expertise in the microbiome space in Europe with the Alliance Promotion Microbiote (APM), of which both organizations are among the co-founders. APM is an association under the French law of 1901 and includes 25 private and public players (companies, clusters, research institutes, investment funds), of which Hervé Affagard was elected president in January 2022. APM aims to contribute to making France, a pioneering nation in microbiome research and engineering, into a European leader to meet the current and future challenges of this sector recognized in 2021 as a priority in the "Health Innovation Plan 2030 ".

[1] Source: Global Data GVHD Epidemiology Report, Jan 2020.
[2] Source: Essai REACH1
[3] Article L 531-8 and 9 of the French Research Code allowing the participation of research personnel in the creation of companies or in the activities of a company and the promotion of their work
[4] metagenomics: a method used to study the microbiome – Qin, J., Li, R., Raes, J. et al. A human gut microbial gene catalogue established by metagenomic sequencing. Nature 464, 59–65 (2010)
[5] Compassionate use programs allow the use of investigational drugs before they are authorized for marketing for patients with no treatment-options.

On April 21, 2022 LUMICKS, a leading life science tools company that develops instruments for dynamic single-molecule and cell avidity analysis, reported that it has completed a new installation of its revolutionary z-Movi Cell Avidity Analyzer in the lab of Prof. Dirk Busch, Professor of Medical Microbiology, Immunology and Hygiene at the School of Medicine, Technical University of Munich (TUM) (Press release, LUMICKS, APR 21, 2022, View Source;utm_medium=rss&utm_campaign=new-customer-installation-of-lumicks-zmovi-cell-avidity-analyzer [SID1234612708]). The installation demonstrates the expanding interest in measuring cell avidity as a key biomarker in understanding immune oncology.

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The z-Movi instrument measures the avidity between immune cells and their targets, enabling researchers to identify the most potent immunotherapeutic effector cells. This new technology provides predictive, reproducible, and fast results at a single-cell resolution. LUMICKS’ cell avidity solutions use acoustics to measure forces and interactions between cells, with the goal of shortening the drug development cycle for adoptive cell therapies and other immunotherapies and reducing failure rates in clinical trials. First introduced in 2020, the z-Movi has found wide appeal in academic and industrial laboratories around the world, with a rapid uptake in sales in 2021.

Prof. Busch and, Dr. Elvira D’Ippolito, a senior post-doctoral researcher at the Busch Lab, noted that "A broad applicability of immunotherapy with T-cell receptor (TCR)-engineered T cells requires the fast identification of highly functional TCRs for diverse antigens. By measuring cellular avidity, we expect to select candidate TCRs for clinical application with unprecedented speed and precision."

Added Gerrit Sitters, General Manager of LUMICKS Cell Avidity Business, "This new installation demonstrates the increased recognition by leading researchers of the valuable insights to be gained by using our LUMICKS technology. We are very pleased to contribute to a better scientific understanding of how measuring cell avidity can uncover critical biomarkers that help advance immune oncology efforts worldwide. We look forward to the insights and discoveries about immune system interactions that these two labs will produce in the future."

On April 21, 2022 BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) reported that the company will report its first quarter 2022 financial results Thursday, May 5, 2022 (Press release, BioCryst Pharmaceuticals, APR 21, 2022, View Source [SID1234612707]).

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BioCryst management will host a conference call and webcast at 8:30 a.m. ET that day to discuss the financial results and provide a corporate update.

The live call may be accessed by dialing 877-303-8027 for domestic callers and 760-536-5165 for international callers and using conference ID # 9498023. A live webcast of the call and any slides will be available online at the investors section of the company website at www.biocryst.com. A telephone replay of the call will be available by dialing 855-859-2056 for domestic callers or 404-537-3406 for international callers and entering the conference ID # 9498023.

On April 21, 2022 Clarity Pharmaceuticals (ASX: CU6) ("Clarity"), a clinical-stage radiopharmaceutical company developing next-generation products to address the growing needs in oncology, reported that it has successfully treated its first participant in the diagnostic US-based 64Cu SAR-bisPSMA trial for patients with biochemical recurrence (BCR) of prostate cancer (Press release, Clarity Pharmaceuticals, APR 21, 2022, View Source [SID1234612668]).

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COBRA (Copper-64 SAR-bisPSMA in Biochemically Recurrent prostAte cancer) is a Phase I/II Positron Emission Tomography (PET) trial of participants with BCR of prostate cancer following definitive therapy (NCT05249127)1. It is a multi-centre, single arm, non-randomised, open-label trial of 64Cu-labelled SAR-bisPSMA in up to 50 participants. The primary objectives of the trial are to investigate safety and tolerability of 64Cu-SAR-bisPSMA as well as its ability to correctly detect recurrence of prostate cancer.

Clarity’s Executive Chairman, Dr Alan Taylor, commented, "We are excited to have dosed the first participant in the COBRA trial at the Urology Cancer Center and GU Research Network (GURN) in Omaha, Nebraska, which continues to actively screen and recruit patients. We are very pleased to see our collaboration with Dr Luke Nordquist at GURN grow and evolve as we fully explore the many clinical and logistical benefits of Targeted Copper Theranostics (TCT)."

Prostate cancer is a key focus of Clarity’s TCT program. Most recently, Clarity announced a collaboration with GURN on a diagnostic 64Cu SAR-bisPSMA investigator-initiated trial (IIT), X-Calibur (NCT05286840)2, sponsored by Dr Luke Nordquist. The US-based theranostic 64Cu/67Cu SAR-bisPSMA trial, SECuRE (NCT04868604)3, has been able to successfully image patients with metastatic castrate resistant prostate cancer from 1 hour to 72 hours post-injection. The diagnostic 64Cu SAR-bisPSMA trial in Australia, PROPELLER (NCT04839367)4, is well underway, and will soon reach full recruitment in untreated, confirmed prostate cancer patients (i.e. pre-radical prostatectomy). Clarity has previously received advice from the FDA that its prostate diagnostic clinical program with 64Cu SAR-bisPSMA is addressing the two relevant patient populations for registration: pre-prostatectomy/pre-definitive treatment as well as patients with suspected biochemical recurrence.

Dr Luke Nordquist, CEO and Urologic Medical Oncologist at the Urology Cancer Center and GU Research Network in Omaha, Nebraska, commented, "We are very excited to have treated the first participant in the COBRA trial and look forward to continuing recruitment at GURN as the more hands-on experience with the TCT platform we gain, the more impressed with these next-generation theranostics we are. Apart from the favourable clinical data acquired in the SECuRE trial at GURN to date, which includes the ability to image tumours between 1 and 72 hours, SAR-bisPSMA enables us to address the significant backlog of patients who cannot access sufficient quantities of PSMA imaging agents based on gallium-68 (Ga-68) or fluorine-18 (F-18) due to the logistical issues of short half-life isotopes. The properties, including the longer half-life of Cu-64, may offer improvements in imaging disease and facilitate central manufacture of the diagnostics, meaning that we can provide critical imaging on-demand and in large scale, delivering the next-generation of technologies to prostate cancer patients and ensuring a timely and accurate diagnosis."

Dr Taylor said, "Treating the first patient in the COBRA trial is an important step in our prostate cancer program and we look forward to receiving preliminary results in participants with suspected recurrence of prostate cancer. We hope that SAR-bisPSMA will enable improved prostate cancer detection, including low volume disease, which is particularly important in this patient population where early and accurate diagnosis has significant implications for the patients’ treatment outcome and prognosis. The preliminary data received from the PROPELLER and SECuRE trials to date is excellent as we have seen high uptake in tumours, and combined with centralised manufacture with on-demand delivery to any zip code in the continental US, this makes SAR-bisPSMA an ideal agent for the pursuit of our ultimate goal of improving treatment outcomes for cancer patients."