On June 16, 2022 Umoja Biopharma, Inc., an immuno-oncology company pioneering off-the-shelf, integrated therapeutics that reprogram immune cells in vivo to treat patients with solid and hematologic malignancies, reported that it will have a poster presentation at the 2022 International Society for Stem Cell Research (ISSCR) Annual Meeting, to be held June 15-18, 2022 in San Francisco, California (Press release, Umoja Biopharma, JUN 16, 2022, View Source [SID1234616046]).

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On Wednesday, June 15th, Principal Scientist & iPSC Team Lead, Teisha Rowland, Ph.D., will give a poster presentation titled, "A Synthetic Cytokine Receptor Platform for Producing Cytotoxic Innate Lymphocytes as Off-the-Shelf Cancer Therapeutics." The presentation will discuss Umoja’s engineered induced pluripotent stem cell (iPSC) platform, that incorporates the synthetic cytokine receptor system rapamycin-activated cytokine receptor (RACR) platform. Umoja’s engineered iPSCs that are modified to express RACR, called RACR-induced cytotoxic innate lymphoid (iCIL) cells, drive differentiation and expansion of the cells while eliminating the need for expensive cytokines and other raw materials. The RACR platform has the potential to enable cytokine-free manufacturing and engraftment of the engineered cells in the patient without the need for toxic lymphodepletion.

"Despite the advances chimeric antigen receptor T cell therapies have provided to the oncology space, we continue to battle significant challenges that these therapies cannot address, like limited expansion capacity and scalability, manufacturing complexity, variability among patients, and the need for toxic chemotherapy administration to combat patients’ anti-allograft response," said Andy Scharenberg, M.D., co-founder and Chief Executive Officer of Umoja. "We are developing an engineered iPSC platform, including the RACR platform, to address these challenges by enabling a scalable, virtually unlimited, and simplified manufacturing of engineered, cancer-fighting cytotoxic innate lymphocytes."

On June 16, 2022 PerkinElmer Inc., a global leader committed to innovating for a healthier world, reported that it is collaborating with Novartis, a leading global medicines company, to expand newborn screening for sickle cell disease (SCD) in sub-Saharan Africa (Press release, PerkinElmer, JUN 16, 2022, View Source [SID1234616045]). PerkinElmer together with the Novartis Africa Sickle Cell Disease program aims to expand advocacy efforts to educate patients, caregivers and communities about the importance of newborn screening and early intervention with hydroxyurea (HU) and other SCD treatments.

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Newborn screening for SCD and comprehensive disease management in high-income countries like the United States has reduced mortality in children under five years old by 94%[1]. Yet, in sub-Saharan Africa which bears the highest burden of the disease, no national newborn screening program exists. This collaboration aims to galvanize governments to provide universal newborn screening as part of national health programs in support of early diagnosis and comprehensive interventions such as prophylactic penicillin, a pneumococcal vaccination and therapies like HU. With effective screening and management of SCD, governments could save many precious lives.

The Novartis Africa Sickle Cell Disease program was launched in Ghana in 2019 as an end-to-end effort that encompasses SCD screening, diagnosis, treatment, education, research and advocacy. Today this unique program has been expanded to Uganda, Tanzania, Kenya and Zambia, with plans to reach a total of 10 countries.

Among its varied contributions to the program, PerkinElmer will work toward strengthening existing SCD programs by providing training, consultations, support and related services to health care professionals and lab technicians across participating countries in sub-Saharan Africa. The Company will also help establish new lab facilities to build capacity for SCD screening in participating countries.

"As a global leader in newborn screening solutions we are pleased to be working with Novartis to assist people across sub-Saharan Africa affected by this condition," said Petra Furu, general manager, reproductive health at PerkinElmer. "By building awareness around the importance of newborn screening, our hope is that more babies receive an early diagnosis that leads to earlier treatment – ensuring they have the best possible chance of a healthy start to life."

"This collaboration with PerkinElmer reaffirms our commitment to reimagining care for sickle cell disease patients, by accelerating access to national newborn screening and hydroxyurea through public-private partnerships with local governments and other organizations," said Racey Muchilwa, Country President and the Head of Novartis sub-Saharan Africa. "We will also be educating patients, caregivers and communities on the importance of newborn screening, early intervention and treatments while elevating the capacity for healthcare professionals to address the high unmet need of SCD."

In an initiative announced in March 2021, PerkinElmer works alongside the American Society of Hematology (ASH) (Free ASH Whitepaper) and its Consortium on Newborn Screening in Africa (CONSA) to provide resources for SCD screening programs in countries throughout sub-Saharan Africa, including Ghana, Kenya, Nigeria, Uganda, Zambia, Liberia and Tanzania. Novartis announced its partnership with ASH (Free ASH Whitepaper) in early June, which will advance ASH (Free ASH Whitepaper)’s CONSA objectives to demonstrate the benefits of newborn screening and early interventions for children with SCD and create a sustainable infrastructure for SCD newborn screening.

On June 16, 2022 Panbela Therapeutics, Inc. (Nasdaq: PBLA), a clinical stage company developing disruptive therapeutics for the treatment of patients with cancer, reported the closing of its acquisition of Cancer Prevention Pharmaceuticals, Inc. (CPP), a private clinical stage company developing therapeutics to reduce the risk and recurrence of cancer and rare diseases, for a combination of stock and future milestone payments (Press release, Panbela Therapeutics, JUN 16, 2022, View Source [SID1234616044]).

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The combined entity will focus on maximizing its extensive pipeline addressing an estimated aggregate $5 billion market opportunity for the areas of initial focus: familial adenomatous polyposis (FAP), first-line metastatic pancreatic cancer, colorectal cancer prevention and ovarian cancer.

"Closing this transaction achieves our goal of creating a diversified pipeline with an ability to hit multiple targets, four clinical stage assets, two of which are late-stage registration assets, thereby increasing the potential of the combined company," said Jennifer K. Simpson, PhD, MSN, CRNP, President & Chief Executive Officer of Panbela. "Together, our capabilities will have an even greater chance to help more patients. The addition of CPP is an excellent fit that we feel produces substantial shareholder value."

The closing of the merger follows the satisfaction of all customary closing conditions, including the unanimous approval by the boards of directors of each company and the stockholders of CPP.

About our Pipeline

The pipeline consists of assets currently in clinical trials with an initial focus on familial adenomatous polyposis (FAP), first-line metastatic pancreatic cancer, neoadjuvant pancreatic cancer, colorectal cancer prevention and ovarian cancer. The combined development programs have a steady cadence of catalysts with programs ranging from pre-clinical to registration studies.

SBP-101

SBP-101 is a proprietary polyamine analogue designed to induce polyamine metabolic inhibition (PMI) by exploiting an observed high affinity of the compound for pancreatic ductal adenocarcinoma and other tumors. The molecule has shown signals of tumor growth inhibition in clinical studies of US and Australian metastatic pancreatic cancer patients, demonstrating a median overall survival (OS) of 14.6 months which is final, and an objective response rate (ORR) of 48%, both exceeding what is seen typically with the standard of care of gemcitabine + nab-paclitaxel suggesting potential complementary activity with the existing FDA-approved standard chemotherapy regimen. In data evaluated from clinical studies to date, SBP-101 has not shown exacerbation of bone marrow suppression and peripheral neuropathy, which can be chemotherapy-related adverse events. Serious visual adverse events have been evaluated and patients with a history of retinopathy or at risk of retinal detachment will be excluded from future SBP-101 studies. The safety data and PMI profile observed in the current Panbela sponsored clinical trial provides support for continued evaluation of SBP-101 in a randomized clinical trial. For more information, please visit View Source .

Flynpovi

Flynpovi is a combination of CPP-1X (eflornithine) and sulindac with a dual mechanism inhibiting polyamine synthesis and increase polyamine export and catabolism. In a Phase 3 clinical trial in patients with sporadic large bowel polyps, the combination prevented > 90% subsequent pre-cancerous sporadic adenomas versus placebo. Focusing on FAP patients with lower gastrointestinal tract anatomy in the recent Phase 3 trial comparing Flynpovi to single agent eflornithine and single agent sulindac, FAP patients with lower GI anatomy (patients with an intact colon, retained rectum or surgical pouch), Flynpovi showed statistically significant benefit compared to both single agents (p≤0.02) in delaying surgical events in the lower GI for up to four years. The safety profile for Flynpovi did not significantly differ from the single agents and supports the continued evaluation of Flynpovi for FAP.

CPP-1X

CPP-1X (eflornithine) is being developed as a single agent tablet or high dose power sachet for several indications including prevention of gastric cancer, treatment of neuroblastoma and recent onset Type 1 diabetes. Preclinical studies as well as Phase 1 or Phase 2 investigator-initiated trials suggest that CPP-1X treatment is well tolerated and has potential activity.

On June 16, 2022 Invectys, Inc. (Invectys), The University of Texas MD Anderson Cancer Center and the Cell Therapy Manufacturing Center (CTMC), a joint venture between MD Anderson and National Resilience, Inc., reported a strategic collaboration to jointly develop a reliable, compliant and scalable process for human leukocyte antigen (HLA)-G targeted chimeric antigen receptor (CAR) T cell therapy for solid tumors (Press release, Invectys, JUN 16, 2022, View Source [SID1234616043]).

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The collaboration will build upon the HLA-G platform pioneered by Invectys to advance novel CAR T cell therapies through preclinical development with CTMC into early-phase clinical studies at MD Anderson. The collaboration brings Invectys’ technology together with the cell therapy development and manufacturing expertise of CTMC and the clinical trials expertise of MD Anderson.

Uniting the complementary capabilities of Resilience and MD Anderson, CTMC was launched to accelerate the development and manufacturing of innovative cell therapies for patients with cancer.

"This agreement is truly about joining the strengths of each collaborator for the benefit of cancer patients," said Praveen Tyle, Ph.D., President and Chief Executive Officer of Invectys. "Invectys is a cancer immunotherapy company developing novel approaches to target HLA-G. With our combined expertise and shared goals, we can act quickly to advance impactful new cell therapies."

The HLA-G molecule is a powerful modulator of the human immune system that is normally found during pregnancy, when it acts to protect the fetus from rejection by the mother’s immune system. However, HLA-G is aberrantly expressed in cancer, making it an attractive tumor-specific antigen because the HLA-G tumor cells suppress a patient’s own innate immune responses. Invectys’ technology is designed to target and remove tumor cells that express HLA-G, thus reducing these immunosuppressive effects and thereby reactivating the patient’s own immune system.

"Immunotherapies have revolutionized the treatment landscape for cancer, but currently approved treatments are able to overcome immune suppression only in limited groups of patients," said Aung Naing, M.D., professor of Investigational Cancer Therapeutics at MD Anderson. "This novel HLA-G technology can revitalize immune cells by identifying and killing solid tumor cancer cells, thereby offering the potential to improve treatment outcomes for a wider group of cancer patients."

Together with researchers at Invectys, the CTMC team will work to develop a clinical-grade HLA-G targeted CAR T cell therapy for solid tumors that can be produced at scale. The collaboration will facilitate therapeutic development toward a Phase I clinical trial to be co-led by Naing and Samer Srour, M.D., assistant professor of Stem Cell Transplantation and Cellular Therapy at MD Anderson.

"CTMC was established to accelerate patient impact by addressing the hurdles associated with the development and manufacturing of cell therapies," said Jason Bock, Ph.D., Chief Executive Officer of CTMC. "We are excited to work with the Invectys team and their unique technology to enable the anti-HLA-G CAR T cell therapy to reach its full potential, hopefully bringing an effective new treatment option to patients in need."

On June 16, 2022 INmune Bio, Inc. (NASDAQ: INMB) (the "Company"), a clinical-stage immunology company focused on developing treatments that harness the patient’s innate immune system to fight disease, reported that has been invited to present data on reversing resistance to HER2 targeted immunotherapy at the HER2 Targeted Therapy Summit in Boston (Press release, INmune Bio, JUN 16, 2022, View Source [SID1234616042]).

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Mucin 4 (MUC4), a glycoprotein on the cell surface of some epithelial cancers, is an easily measured biomarker in women with breast cancer (BC). MUC4 expression is upregulated by soluble tumor necrosis factor (sTNF) produced by the patient’s breast cancer cells. MUC4 expression predicts worse survival in women with HER2+ BC treated with trastuzumab (p≤0.04) and predicts resistance to trastuzumab and trastuzumab based antibody drug conjugates in pre-clinical models.

Dr. Roxana Schillaci of Instituto de Biología y Medicina Experimental in Buenos Aires and RJ Tesi, M.D. have been invited to give a special joint presentation at noon on June 16 entitled Resistance to Immunotherapy Caused by MUC4 Expression in HER2+ Tumors – Bench to Bedside. The presentation will present both the science driving MUC4-based resistance mechanisms and the translation of these pre-clinical studies to the clinic. A link to the presentation can be found HERE.

Dr. Schillaci, working in collaboration with INmune Bio, has shown in preclinical studies that INB03, a novel DN-TNF biologic that neutralizes soluble TNF, reverses MUC4 expression in HER2+ BC pre-clinical models to reestablish sensitivity to trastuzumab (traz) and trastuzumab ADC (TDM-1). In addition, women with MUC4+/HER2+ BC have a cold tumor with fewer tumor infiltrating lymphocytes (TILs) than women who do not express MUC4 (p=0.018). In a nude mouse model of MUC4+HER2+ BC, the combination of INB03+traz increased the number of activated NK cells (p=0.01) and anti-tumor macrophages (p=0.01) in the tumor microenvironment (TME) and showed anti-tumor macrophage function is more important than NK cell function in controlling tumor growth.

"We have identified three reversable resistance mechanisms in women with HER2+/MUC+ breast cancer – resistance to trastuzumab, resistance to tyrosine kinase inhibitors (eg: lapatinib and tucatinib) and a "cold" immunosuppressive TME. All three resistance mechanisms are driven by soluble TNF produced by the tumor and are reversed by neutralization of soluble TNF in the TME by INB03," said Dr. Schillaci.

"We estimate that resistance to immunotherapy occurs in about a third of women with high, low or ultra-low expression of HER2," added RJ Tesi M.D., CEO of INmune Bio. "As of result of the rapidly changing trastuzumab-based immunotherapy landscape, half of all women with breast cancer are at risk for trastuzmab resistance. MUC4 is a biomarker of resistance that can be determined by staining of patient’s breast cancer biopsy enabling a clinical team to prospectively adjust treatment to potentially improve outcome."

About INB03

INB03 is a DN-TNF inhibitor that neutralizes soluble TNF (sTNF) without affecting transmembrane TNF (tmTNF) or TNF receptors. Compared to currently available non-selective TNF inhibitors, INB03 preserves the immune response to cancer by decreasing immunosuppressive cells in the TME including TAM and MDSC while promoting recruitment of anti-tumor immune cells including cytolytic CD8+ lymphocytes, NK cells and anti-tumor macrophages. INB03 has completed an open label dose-escalation Phase I trial in patients with advanced cancer. In that trial, INB03 was found to be safe and well tolerated – no dose limiting toxicity was found. INB03 decreased blood biomarkers of inflammation in patients with advanced cancer. INMB is planning a Phase II trial that uses IN03 as part of combination therapy. More information about INB03 can be found by clicking here.