On June 17, 2022 VBI Vaccines and Hepion Pharmaceuticals reported that it received Orphan Drug Designations from the U.S. Food and Drug Administration Friday for their experimental cancer drugs (Press release, Hepion Pharmaceuticals, JUN 17, 2022, View Source [SID1234616086]).

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Orphan Drug Designations are granted for drugs that show promise for treatment, prevention or diagnosis of orphan diseases, which in the U.S. is defined as affecting fewer than 200,000 people. It comes with certain incentives, including tax credits for qualified clinical trials, user fees exemption and potential seven years of market exclusivity after approval.

Based in Cambridge, MA, VBI received its designation for a bivalent cytomegalovirus gB/pp65 enveloped virus-like particles (VLPs) designed to treat glioblastoma, a type of brain cancer. The company focuses on developing VLPs, leveraging a proprietary enveloped VLP (eVLP) platform technology. These are so-called cancer vaccines that act by mimicking viruses with the intention of stimulating the human immune system.

Earlier this month, the company presented new tumor response and overall survival (OS) data from the ongoing Phase IIa trial of VBI-1901 in recurrent glioblastoma. It was included in a poster presentation at the 2022 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting.

David E. Anderson, Ph.D., chief scientific officer of VBI, said at the time, "We continue to be motivated by the data seen in this Phase IIa study of VBI-1901 as we endeavor to provide new treatment options to patients with very few available to them. Considering the high mortality rate among GBM patients, particularly in the recurrent setting, median overall survival of approximately 13-15 months seen in our two study arms suggests an additional survival benefit of nearly six months in comparison with historical control data in the recurrent population after treatment with a monotherapy."

He added, "Moreover, the correlation of tumor responses and clinical response benefit observed in tandem is very encouraging. We remain in close discussion with our study investigators and scientific advisors as we move toward the next stages of development in both the recurrent and frontline GBM settings, and look forward to advancing this development program as diligently as possible."

Hepion received the designation for rencofilstat for hepatocellular carcinoma. Rencofilstat is the company’s lead oral drug candidate for nonalcoholic steatohepatitis (NASH) and viral hepatitis-induced liver disease.

The drug binds to Cyclophilin A, which blocks it from binding to specific inflammatory cell receptors. This decreases the infiltration of the inflammatory cells into the tissue and lessens harmful inflammatory responses that can cause damage to the liver.

It also blocks the actions of Cycophilin B, which regulates collagen production in stellate cells, causing fibrosis (scarring) in the liver. And it binds to Cyclophilin D, preventing or reversing the formation of pores in the mitochondrial membrane that causes the mitochondria to rupture. This allows the mitochondria to resume normal energy production and helps liver cells survive.

In January, Hepion, headquartered in Edison, NJ, announced the results of a nonclinical research study of rencofilstat that decreased liver tumor growth and extended mouse survival when combined with an anti-PD-1 antibody, or immune checkpoint inhibitor. The effects were seen in fatty livers, which may be associated with less ant-PD-1 efficacy in hepatocellular carcinoma (HCC). This suggests that the drug may improve the treatment potential of anti-PD-1 treatment in human liver cancer. Examples of anti-PD-1 drugs include Merck’s Keytruda (pembrolizumab) and Bristol Myers Squibb’s Opdivo (nivolumab).

At the time, Daren Ure, Ph.D., Hepion’s chief scientific officer, noted, "Anti-PD-1 therapies that stimulate immune cell attack on cancer cells are approved and effective in many types of cancer, but they have had limited success in HCC clinical trials. The lack of clinical outcomes success may be partly due to the occurrence of HCC in individuals that also have non-alcoholic fatty liver disease (NAFLD) or the more advanced form of the disease, NASH."

He added, "NAFLD and NASH are both highly prevalent across the globe, and recent reports indicate that a fatty liver environment blocks the efficacy of anti-PD-1 drugs, and perhaps other checkpoint inhibitors. In addition, as successful treatments for viral hepatitis have been implemented, NASH has been quickly becoming the leading cause of HCC."

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 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 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 OSE Immunotherapeutics reported that the Company is one of the first biotech startup companies selected by Microsoft France to be a privileged partner of its support program for French Biotech & Deeptech Startups (Press release, OSE Immunotherapeutics, JUN 16, 2022, View Source [SID1234646963]). This program was launched during the 6th edition of "Viva Technology", the most important Startup and Tech event organized in Europe being held in Paris from June 15-18, 2022.

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Microsoft France’s « Biotech Scaler Program » aims to support companies at the crossroads of research and technology to accelerate products’ discovery and development. This program provides the selected startups with technical support in order to develop a technical framework of architecture securing scalability and startups’ data security. It also proposes a business support from Microsoft France and its partners through an ecosystem approach connecting startups, the research community, investment funds and the various health players.

Nicolas Poirier, Chief Scientific Officer of OSE Immunotherapeutics, comments: "We are very happy to have been selected by Microsoft to join their Biotech & Deeptech support program and we warmly thank the organizers. This collaboration confirms that OSE perfectly fits the target of the most cuttingedge biotechs by pairing its scientific skills and expertise in artificial intelligence and algorithmic approach to develop immunotherapies based on breakthrough innovations and to meet unmet medical needs in immunooncology and immuno-inflammation."

Since 2019, OSE Immunotherapeutics has been collaborating with MAbSilico, a DeepTech-Bio company specializing in artificial intelligence (AI) algorithms and machine learning to accelerate the research and characterization of therapeutic antibodies.

Since 2021, OSE has benefitted from privileged access to the MAbFactory platform which centralizes MAbSilico’s innovative solutions based on AI. MAbFactory is deployed in the Microsoft Azure cloud with the access to an automatic and autonomous use to support the development of new antibodies and therapeutic proteins. The expertise of OSE’s R&D teams combined with MAbSilico’s AI platform is being used to successfully guide the discovery, the characterization and the optimization of new molecules, reducing both the time of development and the risk of failure in the preclinical testing.

The collaboration between the biotech OSE Immunotherapeutics, the Tech-Bio MAbSilico and the technology provider Microsoft reflects the interest of the Microsoft’s biotech program and will enable to strengthen and to go further in the deployment of a research based on AI, at the crossroads of this fruitful partnership between a Biotech and a Deeptech.