On February 03, 2022 Dyve Biosciences, Inc., a clinical-stage biotech company applying its proprietary transdermal delivery technology to a broad pipeline of clinical assets, reported a joint, two-year research collaboration with Moffitt Cancer Center (Press release, Dyve Biosciences, FEB 3, 2022, View Source [SID1234629085]). The collaboration will study Dyve’s systemic buffering agent, DYV800, utilizing its proprietary and broadly applicable transdermal delivery technology, in various cancer laboratory models developed by Robert Gillies, Ph.D., Chair, Department of Cancer Physiology, at Moffitt. If successful, the laboratory results will support the advancement of DYV800 into focused human trials for specific oncology indications.

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Dr. Gillies’ published pre-clinical research has shown that pH-adjusting agents can successfully modify the acidic microenvironment within tumors that allow cancer cells to evade treatment. In 2015, Dr. Gillies and his colleagues published research demonstrating that systemic buffering increased the efficacy of checkpoint inhibitors in animal models of cancer. The research concluded that many solid tumors are known to create an acidic microenvironment that protects it from the body’s normal defenses and decreases the effect of anticancer therapy.1

Dyve’s buffering agent is expected to decrease the acidic microenvironment in tumors, thereby better allowing the body’s own immunological defense mechanisms to better penetrate the tumor, and hence, increasing the effectiveness of current anti-cancer agents. Dyve’s investigational candidate, DYV702, which leverages a similar buffering mechanism of action as DYV800, recently demonstrated a strong dose-dependent response in a Phase 2 program in patients with acute gout pain flares and was shown to be well-tolerated.

Ryan Beal, M.D., Chief Executive Officer of Dyve, stated, "This collaboration is a fortuitous convergence of Dr. Gillies’ long-standing, world-renowned research on the tumor microenvironment and the successful development of transdermal buffering agents at Dyve. We are excited about this opportunistic collaboration because it signals our entry into the cancer area with a novel mechanistic class of therapeutic agents that we expect to advance into the clinic to treat specific cancers. Our experience with DYV702 validates our novel scientific platform for transdermal delivery and underscores our ability to deliver clinically meaningful levels of buffering capacity into the human body."

Dr. Gillies commented, "Our prior attempts to orally deliver our published therapy to patients failed due to poor taste and poor compliance. The prospect of transdermal delivery is truly exciting and may be a game changer."

Dr. Shari Pilon-Thomas, also of Moffitt Cancer Center and a colleague of Dr. Gillies, added, "We have been hoping that we can study in the laboratory an investigational agent like DYV800 since the results will provide validation of our hypotheses in cancer patients, and we are thrilled to be collaborating with our new partners at Dyve Biosciences."

On February 3, 2022 Simbec-Orion reported that Lupuzor is an in-development treatment for Lupus, a chronic inflammatory disease thought to affect around 5 million people worldwide (Press release, Simbec-Orion, FEB 3, 2022, View Source [SID1234607666]). Having recently undergone FDA approval, Lupuzor is commencing into phase 3 clinical trials.

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Simbec-Orion are proud to have been selected by ImmuPharma as a partner to conduct the international phase 3 trial of Lupuzor involving pharmacokinetic study. Phase 3 research has been granted ethic committee approval, and volunteer selection and screening is set to be conducted in December 2021.

The Lupuzor phase 3 pharmacokinetic trial aims to deliver data by the end of 2022’s first quarter, with dosing of volunteers getting underway in January 2022. This builds upon the first pivotal phase 3 Lupuzor trial completed in 2018, which assessed the drug through open label extension study.

Progress from phase 1 and 2 Lupuzor trials has been promising. ImmuPharma have taken the opportunity to improve the drug’s product characterisation and analytical method validations. As a result, research has made developments with a new proprietary synthesis of P140, resulting in greater IP protection and lower production costs.

Find out more about our clinical development services.

Lupuzor: Key Findings So Far
ImmuPharma have made significant advancements in developing a new treatment for Lupus. This drug, Lupuzor has shown potential as a treatment for Lupus during clinical research and has undergone significant testing across clinical trial phases.

Throughout clinical research stages, Lupuzor has shown strong efficacy in treating Lupus and has also demonstrated a strong safety profile. This provides promising results in the development of a new Lupus treatment, as the current standard treatment presents several side effects.

Phase 3 LupuzorTrials
As part of phase 3 Lupuzor research, Simbec-Orion partnered with ImmuPharma to complete the initial phase 3 trial. As a CRO with expertise in Lupus research, our team ran the first Lupuzor phase 3 trial, which enrolled 62 patients to complete an open label extension study.

This initial Lupuzor phase 3 trial confirmed a successful safety profile for the drug, reporting no serious side effects or reactions. These insights enabled our trial management team to commence with an optimal phase 3 trial design together with ImmuPharma.

The next stage of phase 3 Lupuzor research will involve a pharmacokinetic study, helping further evaluate the drug’s safety profile and efficacy.

Why Lupus Research is Important
Affecting an estimated 5 million people worldwide, Lupus is a chronic inflammatory, autoimmune disease that can be life threatening. Lupus can impact various parts of the body, including skin, kidneys, the brain, heart and lungs.

Diagnosis can be challenging, since Lupus can appear in several different forms and although awareness is steadily increasing, it does not have the greatest level of recognition.

As well as challenges in diagnosis, the current standard treatment for Lupus can often present adverse side effects. As well, the standard treatment has limited efficacy. As a result, there is an unmet need for an effective Lupus treatment with stronger efficacy and fewer side effects.

Partner With Simbec-Orion
Experts in clinical trial management, we’re here to support you with your clinical development. Get in touch with our expert team to find out more.

On February 3, 2022 Syndax Pharmaceuticals, Inc. ("Syndax," the "Company" or "we") (Nasdaq: SNDX), a clinical-stage biopharmaceutical company developing an innovative pipeline of cancer therapies, reported that Michael A. Metzger, President and Chief Operating Officer, will transition to the role of Chief Executive Officer, effective today (Press release, Syndax, FEB 3, 2022, View Source [SID1234607684]). Briggs W. Morrison, M.D., Chief Executive Officer, will transition to the role of President, Head of Research and Development (R&D) . Mr. Metzger and Dr. Morrison will remain on the Company’s Board of Directors. Both Mr. Metzger and Dr. Morrison joined Syndax together in 2015 with the goal of building a world-class oncology company.

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"Briggs and I have worked very closely in partnership over the last seven years to expand Syndax’s portfolio of assets through business development transactions while simultaneously building the organization, developing the pipeline, and executing on key capital raising initiatives," said Mr. Metzger. "This transition will better align our roles and priorities with our individual areas of expertise. Going forward, I will be focused on executing on the strategic vision for the Company, including establishing the operational groundwork to expand Syndax into a commercial entity and growing our pipeline through transactions. Briggs will continue to lead the R&D organization, with a key focus on bringing two assets to approval and helping to bring new assets into the organization. I look forward to our continued partnership as we work to deliver innovative therapies to areas of high unmet need in oncology and build long-term value for all of our stakeholders."

"With topline data expected early next year for both of our ongoing SNDX-5613 and axatilimab registration programs, we are keenly focused on preparations for multiple potential New Drug Application filings in 2023," said Dr. Morrison. "I look forward to focusing my efforts, together with the talented team at Syndax, on developing these exciting product candidates into approved therapies and to advancing them into new indications in the clinic, including the first-line and maintenance settings for SNDX-5613 in acute leukemia, and in idiopathic pulmonary fibrosis for axatilimab. We expect this transition of roles to be seamless, and I look forward to continuing to partner with Michael as we work to advance our mission of realizing a future in which people with cancer live longer and better than ever before."

Prior to joining Syndax, Mr. Metzger served as President, Chief Executive Officer and a Director of Regado Biosciences through its merger with Tobira Therapeutics (acquired by Allergan). He has also served in senior leadership and business development roles at Mersana Therapeutics, Forest Laboratories (acquired by Allergan), and in various other industry and investment roles. He received a B.A. from George Washington University and an M.B.A. in finance from the New York University Stern School of Business.

Prior to Syndax, Dr. Morrison served in several senior leadership roles in the industry, including as Executive Vice President, Global Medicines Development and Chief Medical Officer at AstraZeneca, Head, Medical Affairs, Safety and Regulatory Affairs at Pfizer, and Vice President, Clinical Sciences, Oncology, at Merck & Co. He received a B.S. in biology from Georgetown University and an M.D. from the University of Connecticut.

On February 2, 2022 Medigene AG ( View Source) (Medigene, FSE: MDG1, Prime Standard), a clinical-stage immuno-oncology company focusing on the development of T-cell-based cancer therapies, reported that promising preliminary efficacy and immune monitoring data from the Phase I part of the Phase I/II clinical trial of Medigene’s T cell receptor-modified T cell (TCR-T) therapy MDG1011 in patients with advanced-stage blood cancers (ClinicalTrials.gov Identifier: NCT03503968 ( View Source)) (Press release, MediGene, FEB 3, 2022, View Source [SID1234607703]). Data demonstrating safety and tolerability, with no cases of dose-limiting toxicity (DLT) associated with MDG1011, were published in December 2021.

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MDG1011 is a TCR-T immunotherapy directed against the tumor antigen PRAME (PReferentially expressed Antigen in MElanoma). Patients with relapsed or refractory acute myeloid leukemia (AML), myelodysplastic syndrome (MDS) or multiple myeloma (MM) were included in the open-label, dose-escalation Phase I part of the study that was conducted at nine clinical centers in Germany. Following standard pre-conditioning, patients received MDG1011 as a single intravenous infusion at specified dose levels of 0.5, 1 or 5 million TCR-transduced T cells per kg body weight. The primary study objectives were to evaluate safety, tolerability, and feasibility to manufacture autologous MDG1011 TCR-T cells for heavily pretreated patients. In addition, preliminary signs of clinical efficacy and immune monitoring data were investigated.

Clinical and biological data analysis

MDG1011 contained CD8+ TCR-T cells showing recognition and killing of specific standardized target cells in vitro and was successfully manufactured for 13 patients. The group of patients included ten with AML, two with MM and one with MDS. Four patients succumbed to disease before treatment could be administered, in line with the severity of the underlying condition of study patients. Thus, nine patients received MDG1011 at one of the three specified dose levels.

Patient immune monitoring included detection of PRAME-specific T cells (MDG1011 TCR-T cells) in blood to determine persistence over time and biomarker tracking of PRAME in bone marrow and/or blood (as an indicator for remaining cancer cells) by qPCR.

* One patient with AML treated at the lowest dose experienced complete remission at week 4 after treatment; however, this clinical response was not sustained, and the patient’s disease progressed 8 weeks thereafter. PRAME expression was slightly increased from baseline at week 4 whereas MDG1011 TCR-T cells were below the detection level.
* Two patients with AML, treated at the intermediate and highest dose respectively, experienced transient grade 1 or 2 cytokine release syndrome (CRS) within 3 days of drug administration, providing evidence of biological activity of MDG1011 therapy in vivo. MDG1011 TCR-T cells were still detectable in both patients at week 4.
* One patient with multilineage MDS and myeloproliferative neoplasm (MPN) treated at the highest dose, remained without apparent progression to secondary AML 3 and 6 months after MDG1011 administration and is still monitored. MDG1011 TCR-T cells were found at weeks 4, 8 and 12 and were still present at lower levels at month 6 after treatment. Bone marrow could not be evaluated in this patient. PRAME signals in blood were no longer detected at week 4 but gradually increased thereafter at 2, 3 and 6 months, remaining clearly below the baseline level. In concordance, blast counts both in blood and bone marrow remained well below baseline.
* MDG1011 TCR-T cells were detected in six of eight evaluable patients at one or more time points within four weeks after administration. TCR-T cells were also present in two patients at later time points, albeit at decreased levels.
* The biomarker PRAME was assessed in bone marrow samples from five patients four weeks after MDG1011 administration compared to baseline. PRAME decreases occurred in three AML and one MM patient while a slight increase was noted in another patient with MM. PRAME decreased in blood at week 4 for two patients treated at the highest dose but increased thereafter.

The preliminary clinical observations in two patients and occurrence of CRS in a further two of the nine heavily pretreated cancer patients are both encouraging and consistent with expectations of an adoptive T cell therapy. These observations are corroborated by the immune monitoring data.

Prof. Simone Thomas, from the Department of Internal Medicine III of the University Hospital Regensburg and Leibniz Institute for Immunotherapy, Principal Investigator of the study: "No immune effector cell-associated neurotoxicity syndrome (ICANS) or DLTs were detected, so MDG1011 appears to be safe and well-tolerated at all three doses. The aforementioned clinical observations taken together with the effect on PRAME levels and the persistence of MDG1011 strongly support the hypothesis of biological activity of MDG1011."

Prof. Dolores Schendel, Chief Executive Officer and Chief Scientific Officer at Medigene: "We are very pleased that all MDG1011 TCR-T drug products manufactured from such heavily pre-treated patients with a substantial disease burden showed strong functional activity in vitro, leading to detectable biological and/or clinical activity in four patients.

The effects of PRAME-specific TCR-T immunotherapy on cancer cells should be further investigated based on this safe and well-tolerated treatment. The use of higher cell numbers as well as the treatment of patients with less advanced disease stages could further increase the clinical benefit in the future. Based on our strategic shift to development of TCR-T immunotherapy for solid cancer, Medigene has announced previously that the Phase II part of this study will only be conducted with or by a partner; based on overall results from the Phase I part of the study.

Our current development programs in solid cancer concentrate on TCR discovery for novel tumor-specific antigens and implementation of innovative tools to enhance efficacy and improve safety of T-cell-based immunotherapies."

On February 3, 2022 Sapience Therapeutics, Inc., a biotechnology company focused on the discovery and development of peptide therapeutics to address difficult-to-treat cancers, reported that the company will present at the Guggenheim Healthcare Talks, 2022 Oncology Conference, taking place virtually February 9-February 11, 2022 (Press release, Sapience Therapeutics, FEB 3, 2022, View Source [SID1234607720]).

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Dr. Barry Kappel, CEO and President of Sapience, will present a company overview on Friday February 11th, at 11:30 am ET. Sapience management will also participate in one-on-one meetings with investors during the conference.