On June 16, 2022 Pionyr Immunotherapeutics, Inc., a company developing first-in-class Myeloid Tuning antibody therapeutics that enhance the body’s anti-tumor immunity by altering, or "tuning," immune cells within the tumor microenvironment, reported that Chief Medical Officer Leonard Reyno, M.D., will lead a discussion on myeloid cell targeting to reprogram the tumor microenvironment, modulate innate immune activity, and enhance anti-tumor immunity (Press release, Pionyr Immunotherapeutics, JUN 16, 2022, View Source [SID1234616049]). The presentation will highlight preclinical and early clinical research on Pionyr’s first in class TREM1-targeting monoclonal antibody called PY159. The presentation titled, "Reprogramming TREM1+ Myeloid Cells to Overcome Immunotherapy Resistance in Solid Tumors," takes place at the 2nd Tumor Myeloid Directed Therapies Summit on June 16, 2022.

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"Pionyr was built on the premise that the myeloid compartment is a source of provocative, untapped anti-tumor drug targets that can be harnessed to modulate the innate immunity and our natural defenses against cancer cells," said Leonard Reyno, M.D., Chief Medical Officer at Pionyr. "With first-in-class drug candidates in clinical development targeting TREM1 and TREM2 associated myeloid cells, and a preclinical program targeting MARCO, our approach demonstrates the incredible potential to improve innate immunity, overcome the limitations of checkpoint inhibitors, and illustrate a foundational approach to transforming immuno-oncology."

As part of the presentation, Pionyr will highlight the PY159 development program. PY159 is a humanized monoclonal antibody that specifically binds triggering receptor expressed on myeloid cells 1 (TREM1). The presentation will highlight:

Preclinical research demonstrating expression of TREM1 to be higher in cancerous tissues vs normal tissues
Preclinical evidence showing TREM1 as a promising therapeutic opportunity for patients with checkpoint inhibitor (CPI)-hypo-responsive or resistant tumors as the receptor is expressed on the three myeloid immuno-suppressive cell populations
In vivo model data demonstrating administration of PY159 alone or in combination with CPIs resulted in complete regressions of tumors
First in human clinical experience administering PY159 both alone and in combination with pembrolizumab including identification of target archival tumor specimens from participating patients
Outline clinical plans for further study in patients with advanced refractory solid tumors
The Tumor Myeloid-Directed Therapies Summit is the only industry-focused meeting dedicated to unlocking novel therapeutic potential of the myeloid compartment where experts come together to identify, validate and clinically progress myeloid targets to expand the oncologist’s toolkit and realize better responses by engaging myeloid anti-tumor function. It is designed for experts working directly in myeloid-directed therapies, and those in adjacent immune-oncology fields, from cell therapy to checkpoint inhibitor development.

About Myeloid Tuning

Pionyr has developed a therapeutic platform called Myeloid Tuning, a process that rebalances the tumor microenvironment (TME) to promote anti-tumor immunity. Myeloid cells are a type of immune cell and are part of a family of cell types that play an important role in both the activation and suppression of the immune response to cancer.

One such critical type of myeloid cell, tumor-associated macrophages (TAM), are a key component of the TME. TAMs are generally categorized into two functionally contrasting subtypes called M1-like and M2-like macrophages: M1-like typically exerts anti-tumor functions, including directly mediating antibody-dependent cell-mediated cytotoxicity (ADCC) to kill tumor cells; M2-like macrophages can promote the occurrence and metastasis of tumor cells, inhibit T cell-mediated anti-tumor immune response, promote tumor angiogenesis and lead to tumor progression.

Myeloid Tuning effectively describes the mechanism of introducing agents that shift the balance of inhibitory myeloid cells – including M2-like TAMs – towards more inflammatory M1-like TAMs to promote anti-tumor immune responses in the TME and destroy solid tumors.

On June 16, 2022 Humanigen, Inc. (Nasdaq: HGEN) ("Humanigen"), a late-stage clinical biopharmaceutical company focused on preventing and treating an immune hyper-response called ‘cytokine storm,’ reported it entered into an agreement with PCI Pharma Services (PCI), a leading integrated global contract development manufacturing organization (CDMO), to provide importation, release and commercialization services in the United Kingdom (UK) for lenzilumab (Press release, Humanigen, JUN 16, 2022, View Source [SID1234616048]). Under the agreement, PCI will purchase lenzilumab for resale and distribution in the event a Conditional Marketing Authorization is received in the UK for use in patients hospitalized with COVID-19.

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"We continue our commercial preparation in the UK and in parallel are working closely with the Medicines and Healthcare products Regulatory Agency (MHRA) to address regulatory requirements for a potential Conditional Marketing Authorization. With its global reach, PCI will provide a critical function in the supply chain, by directly purchasing lenzilumab for further distribution in the UK and facilitating this key process for Humanigen," said Edward Jordan, Chief Commercial Officer. "It is anticipated that we will complete our response to MHRA soon after the top-line results from the ACTIV-5/BET-B clinical trial with lenzilumab are received."

If authorized, lenzilumab will offer an important treatment option to patients hospitalized with COVID-19. Hospitalizations from COVID-19 continue in the United Kingdom with more than 235,000 admitted year-to-date and with ~5,000 currently hospitalized.1 In addition, Humanigen believes that treatment with lenzilumab may deliver economic value to the healthcare system. Previously published research has demonstrated that treatment with lenzilumab may save the National Health Service over £10,000 per patient.2

Lenzilumab is an investigational product and is not approved or authorized in any country.

About Lenzilumab

Lenzilumab is a proprietary Humaneered first-in-class monoclonal antibody that has been proven to neutralize GM-CSF, a cytokine of critical importance in the hyperinflammatory cascade, sometimes referred to as cytokine release syndrome, or cytokine storm, associated with COVID-19 and other indications. Lenzilumab binds to and neutralizes GM-CSF, potentially improving outcomes for patients hospitalized with COVID-19. Humanigen believes that GM-CSF neutralization with lenzilumab also has the potential to reduce the hyperinflammatory cascade known as cytokine release syndrome common to chimeric antigen receptor T-cell (CAR-T) therapy and acute Graft versus Host Disease (aGvHD).

In CAR-T, lenzilumab successfully achieved the pre-specified primary endpoint at the recommended dose in a Phase 1b study with Yescarta in which the overall response rate was 100% and no patient experienced severe cytokine release syndrome or severe neurotoxicity. Based on these results, Humanigen plans to test lenzilumab in a randomized, multicenter, potentially registrational, Phase 3 study ("SHIELD") to evaluate its efficacy and safety when combined with Yescarta and Tecartus CAR-T therapies in non-Hodgkin lymphoma. Lenzilumab will also be tested to assess its ability to prevent and/or treat aGvHD in patients undergoing allogeneic hematopoietic stem cell transplantation.

A study of lenzilumab is also underway for patients with chronic myelomonocytic leukemia (CMML) exhibiting RAS pathway mutations. This study builds on evidence from a Phase 1 study, conducted by Humanigen, that showed RAS mutations are associated with hyper-proliferative features, which may be sensitive to GM-CSF neutralization.

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.