On May 16, 2022 Adamis Pharmaceuticals Corporation (NASDAQ: ADMP), a biopharmaceutical company developing and commercializing specialty products for allergy, opioid overdose, respiratory and inflammatory disease, reported financial results for the quarter ending March 31, 2022 (Press release, Adamis Pharmaceuticals, MAY 16, 2022, View Source [SID1234614699]).

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Product and Pipeline Updates and Other Corporate Developments

ZIMHI

·According to the Centers for Disease Control and Prevention (CDC), drug overdoses resulted in over 108,000 deaths in the U.S. over the most recent 12 months of data. Two thirds of these involved fentanyl.

·Our U.S. commercial partner, US WorldMeds, commercially launched our high dose naloxone product, ZIMHI, at the end of March.

·We are encouraged by the early acceptance of ZIMHI in the market.

SYMJEPI

·In March, Adamis announced a voluntary recall of certain lots of SYMJEPI.

·Manufacturing of SYMJEPI has been on hold pending the results of an investigation to determine the root cause. The Company believes the investigation is nearing completion, that a root cause relating to a particular batch of syringe needles has been identified, and that corrective and preventive actions have been and will be taken.

·We anticipate a resolution and resumption of manufacturing after those issues are satisfactorily addressed, which we believe will occur during the second quarter.

TEMPOL

·The Company’s Phase 2/3 clinical trial of Tempol as a treatment for COVID-19 is continuing.

·In March, the Data Safety Monitoring Board (DSMB) overseeing the Tempol trial met to evaluate the clinical and safety data from the first planned interim analysis and, following their evaluation, recommended the study continue without modification.

·The DSMB plans to meet again, anticipated to be at the end of May or in June, to review interim data analysis for the first 124 patients.

·In addition to the work in COVID, the Company continues to explore additional indications for the use of Tempol including, but not limited to, asthma and long COVID.

Financial Results

Revenues for the quarters ending March 31, 2022 and 2021 were approximately $1.2 million and $1.4 million, respectively. Revenues for the quarter ended March 31, 2022 consisted mainly of approximately $1.1 million of sales of ZIMHI to our commercial partner US WorldMeds in anticipation of the commercial launch of ZIMHI announced at the end of March. Due to the SYMJEPI manufacturing hold and the voluntary recall of certain lots, no revenues relating to SYMJEPI were reported for the first quarter of 2022.

Selling, general and administrative expenses for the quarters ending March 31, 2022 and 2021 were approximately $3.4 million and $3.5 million, respectively. SG&A expenses in the first quarter of 2022 reflected a decrease in legal and compensation expenses, offset by an increase in accounting and finance related expenses.

Research and development expenses were approximately $4.2 million and $2.2 million for the first quarter of 2022 and 2021, respectively. The increase was primarily due to expenses relating to the ongoing clinical trial for our Tempol product candidate.

Net loss from discontinued operations for the three months ended March 31, 2022 and 2021 was approximately $165,000 and approximately $1.5 million, respectively. The decrease in loss was primarily attributable to the winding down and cessation of US Compounding’s operations.

Cash and cash equivalents at March 31, 2022, totaled approximately $17.8 million. For this year, we expect to receive additional proceeds resulting from amounts payable to us pursuant to our sale of certain USC assets to Fagron and from the disposition of the remaining USC assets which includes the land, the building, the machinery and the equipment.

Conference Call

Adamis will host a conference call and live webcast today, May 16, 2022, at 2 p.m. PT (5 p.m. ET) to discuss its financial and operating results for the first quarter 2022, as well as provide an update on business developments and activities.

A live audio webcast of the conference call will also be available via this link. If you are unable to participate in the live call, a replay will be available shortly after the live event. To listen to the replay please visit the events page of the Adamis investor relations section of the company website at View Source

On May 16, 2022 Faron Pharmaceuticals Ltd. (AIM: FARN, First North: FARON), a clinical stage biopharmaceutical company focused on building the future of immunotherapy by harnessing the power of the immune system to tackle cancer and inflammation, reported that both the U.S. Food and Drug Administration (FDA) and Finnish Medicines Agency (FIMEA) have cleared Faron’s Investigational New Drug (IND) application to begin the Company sponsored BEXMAB study (Press release, Faron Pharmaceuticals, MAY 16, 2022, View Source [SID1234614687]). BEXMAB is a novel Phase I/II study to assess safety, tolerability and preliminary efficacy of bexmarilimab, Faron’s wholly-owned investigational precision cancer immunotherapy, in combination with standard of care (SoC) therapy in patients with relapsed acute myeloid leukemia (AML), myelodysplastic syndrome (MDS), or chronic myelomonocytic leukemia (CML). This marks the first time bexmarilimab will be assessed as part of a clinical study in hematologic malignancies.

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"We are pleased that our IND application was cleared to proceed, and we can further explore the strong scientific rationale for combining bexmarilimab and azacitidine." said Marie-Louise Fjällskog, M.D., Ph.D., Chief Medical Officer of Faron. "Research has shown a clear survival benefit among certain blood cancer patients with low Clever-1 expression, a receptor known to be expressed on immunosuppressive macrophages in the tumor microenvironment. By adding bexmarilimab to standard of care we expect to downregulate Clever-1 expression, thereby increasing antigen presentation and allowing the immune system to better identify and kill cancer cells."

The primary objective of the BEXMAB study is to determine the safety and tolerability of bexmarilimab in combination with SoC treatment and to identify the recommended Phase 2 dose. Secondary objectives include characterizing the pharmacokinetic profile of bexmarilimab in combination with SoC treatment (azacitidine) and to assess the immunogenicity of bexmarilimab. Based on initial safety data, there is potential for Phase II expansion and to include a first line triplet therapy of bexmarilimab, azacitidine and venetoclax in newly diagnosed AML patients who are not able to tolerate chemotherapy. Patient recruitment is expected to begin in the coming weeks.

"We know from pre-clinical research, some of which was presented recently at EHA (Free EHA Whitepaper), that certain blood cancer cells, especially with myeloid background, carry significant amounts of cell surface Clever-1," said Dr. Markku Jalkanen, Chief Executive Officer of Faron. "This corresponds with the presence of considerable amounts of soluble Clever-1, which limits T cell activation leading to a possible systemic loss of immune capacity. Directly targeting Clever-1 could ignite the immune system, limit the replication capacity of cancer cells, and allow current chemotherapy treatments to be more effective."

In addition to the BEXMAB study focused on hematologic malignancies, Faron is also investigating bexmarilimab in solid tumors. The ongoing Phase I/II MATINS clinical trial is assessing bexmarilimab as a potential monotherapy in late-stage, heavily pre-treated patients across multiple tumor types. Additionally, the Company expects to initiate a trial assessing the safety and tolerability of bexmarilimab in combination with an approved anti-PD-1 molecule in multiple solid tumors later this year.

About Bexmarilimab

Bexmarilimab is Faron’s wholly-owned, investigative precision immunotherapy with the potential to provide permanent immune stimulation for difficult-to-treat cancers through targeting myeloid cell function. A novel anti-Clever-1 humanised antibody, bexmarilimab targets Clever-1 positive (Common Lymphatic Endothelial and Vascular Endothelial Receptor 1) tumour associated macrophages (TAMs) in the tumour microenvironment, converting these highly immunosuppressive M2 macrophages to immune stimulating M1 macrophages. In mouse models, bexmarilimab has successfully blocked or silenced Clever-1, activating antigen presentation and promoting interferon gamma secretion by leukocytes. Additional pre-clinical studies have proven that Clever-1, encoded by the Stabilin-1 or STAB-1 gene, is a major source of T cell exhaustion and involved in cancer growth and spread. Observations from clinical studies to date indicate that Clever-1 has the capacity to control T cell activation directly, suggesting that the inactivation of Clever-1 as an immune suppressive molecule could be more broadly applicable and more important than previously thought. As an immuno-oncology therapy, bexmarilimab has potential as a single-agent therapy or in combination with other standard treatments including immune checkpoint molecules. Beyond immuno-oncology, it offers potential in infectious diseases, vaccine development and more.

On May 16, 2022 Orna Therapeutics, a biotechnology company pioneering a new class of fully engineered circular RNA (oRNA) therapies, reported that data from its lead isCAR program that validates the potential of the company’s novel oRNA technology and LNP delivery platform (Press release, Orna Therapeutics, MAY 16, 2022, View Source [SID1234614686]). Based on advances in the expression of oRNA as well as its delivery to immune cells, Orna has demonstrated tumor suppression and eradication in an animal model pointing to the possibility that oRNA-LNP based cancer therapies could eventually overtake cell therapies .

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Additional data to be presented demonstrate the utility of Orna’s proprietary FoRCE screening platform which has enabled the company to discover and characterize a major new resource for protein expression based on internal ribosome entry sites (IRESs). Orna will also present data showing the development of novel immunotropic lipid nanoparticles (LNPs), and the potential of oRNA in genetic muscle disease and vaccines. View full presentations from ASGCT (Free ASGCT Whitepaper) on our website here.

"At Orna, we’ve created the world’s leading circular RNA company and are building a platform and pipeline with the potential to change the way we treat disease," said Tom Barnes, PhD, Orna’s Chief Executive Officer and oral presenter at ASGCT (Free ASGCT Whitepaper). "Presented for the first time, these data suggest our oRNA technology and LNP delivery favorably combine to maximize our reach into multiple therapeutic areas – validating our expanded pipeline beyond cancer to include muscle genetic diseases and vaccines."

isCAR: Revolutionizing CAR-T Cell Therapy with the Possibility of Eradicating Tumors
Our lead program is an in situ CAR therapy that combines oRNA and custom engineered LNPs to create modified immune cells within the patient. This easily redosable format would not require patient lymphodepletion and would allow for reliable dose control, overcoming barriers of ex vivo CAR-T therapies. Data being presented demonstrate that oRNA-LNP can eradicate cancer cells in an animal model. Additional iterative data in rodents and non-human primates gives Orna confidence that this may successfully translate into humans.

"We are excited about the preclinical results in our lead isCAR program as we clearly see the opportunity to overcome significant hurdles in current ex vivo approaches, suggesting that oRNA-LNP based cancer therapies may eventually overtake cell therapies," said Robert Mabry, PhD and Chief Scientific Officer at Orna. "We believe that data from iterative animal studies can support our plans to deliver in situ CAR-T therapies to the clinic."

FoRCE: Formulated oRNA Cell-based Evaluation Platform
Orna is also presenting new data from our proprietary FoRCE screening platform, which captures the entire oRNA production, formulation, and evaluation process in an arrayed and automated format. In a first application, Orna has screened and characterized thousands of IRES elements in multiple primary human cell types. IRES identification and development is critical for optimizing oRNA function via tunable protein expression. Orna has discovered many novel IRES elements that drive oRNA expression to levels well above those of standard IRES elements, including some that show differential activity across cell types. These results open a new technological toolkit for driving protein expression from circular RNA.

Breadth of Platform
Orna has extended its oRNA-LNP technology into several other indications including Duchenne Muscular Dystrophy and vaccines and believes there are many additional opportunities this technology can bring to existing therapeutics.

Duchenne Muscular Dystrophy (DMD):
Orna will present data highlighting the ease of working with very large oRNAs. Data demonstrate, for the first time, non-viral delivery of a large, full-length, dystrophin-encoding RNA in human cells, as well as in vivo delivery of smaller length versions in mouse models. These data are an encouraging first step on the path to delivering full-length gene therapy to patients with DMD.

Vaccines:
Orna is investigating the suitability of oRNA combined with intramuscularly administered immunotropic LNPs for vaccine applications, including for COVID-19. The oRNA half-life observed in muscle and immune cells, combined with the intramuscular administration of immunotropic lipids, suggests that oRNA-LNP technology may be beneficially applied to vaccine development.

Conference presentation details are shared below.

Oral Presentations:
In situ CAR Therapy Using oRNA Lipid Nanoparticles Regresses Tumors in Mice
Presenter: Tom Barnes, Ph.D., CEO
Date/Time/Location: Monday, May 16, 2022 from 9:10 – 9:45 a.m. ET in Room 207
Session: Scientific Symposium: Function and Therapeutics Applications of Circular RNAs (circRNAs)

Discovery of Translation Initiation Elements Enabled by a Parallel Arrayed Screen of Full-length Viral UTRs in Synthetic Circular RNA
Presenter: Alexander Wesselhoeft, Ph.D., Director, Molecular Biology
Date/Time/Location: Monday, May 16, 2022 from 11:30 – 11:45 a.m. ET in Salon H
Session: Oral Abstract Session: Oligonucleotide Therapeutics

Poster Presentations:
Improved Immune Cell Expression with Circular RNA (oRNA) in vivo
Presenter: Kevin Kauffman, Ph.D., Principal Scientist
Date/Time/Location: Monday, May 16, 2022 at 5:30 p.m. ET in Hall D
Session: Poster Session: Oligonucleotide Therapeutics I

Systemic Delivery of Circular RNA Encoding Partial Dystrophins and Expression in Skeletal Muscle
Presenter: Tatiana Fontelonga, Ph.D., Scientist
Date/Time/Location: Tuesday, May 17, 2022 at 5:30 p.m. ET in Hall D
Session: Poster Session: Oligonucleotide Therapeutics II

On May 16, 2022 PACT Pharma, Inc., a clinical-stage company developing transformational personalized neoantigen-specific T cell receptor (neoTCR) T cell therapies for the eradication of solid tumors, reported that new data on PACT^NV, the company’s non-viral precision gene editing technology, were highlighted in a poster presentation at the American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 25th Annual Meeting (Press release, PACT Pharma, MAY 16, 2022, View Source;cell-therapy-asgct-25th-annual-meeting-301547277.html [SID1234614685]). Presented findings demonstrated the potential of the PACT^NV technology to enhance the functionality and applicability of T cell-based therapeutics, as well as other cellular therapies, through targeted gene editing. Specifically, researchers showed the ability of the versatile platform to knock-out, knock-down, knock-in and precisely regulate target genes in a single step to achieve desired activity for its neoTCR T cells. The ASGCT (Free ASGCT Whitepaper) conference is being held May 16-19, 2022, in Washington, D.C.

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The poster presentation reported research findings by PACT scientists demonstrating the ability to utilize the company’s PACT^NV technology to generate TCR T cells in a single step. In creating these novel patient-specific neoTCR T cells, PACT highlighted the following capabilities of its gene editing platform:

Insertion and expression of neoTCRs within T cells.
Simultaneous elimination of the expression of the endogenous TCRs within same T cells.
Knock-out of the transforming growth factor beta (TGF-β) receptor type 2 (TGFBR2) gene, generating a T cell that can both recognize a patient-specific tumor and is resistant to the immunosuppressive signaling by TGF-β.
shRNA driven knock-down of multiple additional genes without any associated double-stranded break, generating enhanced T cells that can both recognize a patient-specific tumor and, depending on the targeted transcript, possess resistance to immunosuppression, increased persistence, and/or improved functional avidity.
Development of T cell activation-induced promoters for conditional expression of payloads.
"Taken together, the powerful gene editing capabilities possessed by our PACT^NV platform offer the potential to expand the applicability of T cell therapeutics, while extending the potential use of the technology to other cellular therapies," said Stefanie Mandl, Ph.D., senior vice president, head of research at PACT Pharma. "We are particularly excited about the versatility to add, eliminate and/or precisely modulate multiple genes within our neoTCR T cells to create novel, personalized therapeutic candidates with the potential to address a range of solid tumors. We look forward to continuing our research and development efforts in support of our PACT^NV technology, while continuing our ongoing Phase 1 clinical trial of non-viral PACT^NV gene-edited autologous neoTCR T cells in advanced and metastatic solid tumors."

PACT is currently conducting a Phase 1 clinical trial evaluating the safety, tolerability and feasibility of adoptive cell therapy with its non-viral PACT^NV gene-edited autologous neoTCR T cells in advanced and metastatic solid tumors. This trial, in combination with extensive preclinical studies, has provided the company with unique data sets and insights derived from the core technologies that comprise its personalized adoptive T cell therapy platform for the treatment of solid tumors.

A copy of the poster presented at the ASGCT (Free ASGCT Whitepaper) conference is available on the "Events" page of the PACT Pharma website at: View Source

On May 16, 2022 Factor Bioscience Inc., a Cambridge-based biotechnology company focused on developing mRNA and cell-engineering technologies, reported its participation in the American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 25th Annual Meeting to be held in Washington, D.C. from May 16-19, 2022, at which Factor will deliver four presentations (Press release, Factor Bioscience, MAY 16, 2022, View Source;cell-therapy-asgct-25th-annual-meeting-301547566.html [SID1234614684]):

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iPSC-Derived Monocytes Generate Functional M1 and M2 Macrophages with Enhanced Cytokine Secretion and Tumor Cell-Killing Activity
iPSC-Derived Monocytes Generate Functional M1 and M2 Macrophages with Enhanced Cytokine Secretion and Tumor Cell-Killing Activity
Factor to reveal mRNA-based iPS cell-derived macrophages for solid-tumor targeting

"iPSC-Derived Monocytes Generate Functional M1 and M2 Macrophages with Enhanced Cytokine Secretion and Tumor Cell-Killing Activity." -to be presented by Ian Hay on May 18 at 5:00 pm, Engineered Cell Therapies Oral Abstract Session, Room 206.

"Cytotoxic Lymphocytes Derived from B2M-Knockout iPSCs Show Enhanced Expansion and Cytokine-Controlled Cytotoxicity In Vitro." -to be presented by Mackenzie Parmenter on May 17 from 5:30-6:30 pm, Cell Therapies II Poster Session.

"The Immunosuppressive TTAGGG Motif Improves Homology-Directed Insertion of DNA Sequences in Human Primary and Induced Pluripotent Stem (iPS) Cells." -to be presented by Abigail Blatchford on May 17 from 5:30-6:30 pm, Oligonucleotide Therapeutics II Poster Session.

"Resveratrol Treatment Increases Homology-Directed Repair in Primary Human Cells." -to be presented by Taeyun Kim on May 18 from 5:30-6:30 pm, Gene Targeting and Gene Correction III Poster Session.
Digital presentations will be made available on the ASGCT (Free ASGCT Whitepaper) website on May 16, 2022. For more information about the American Society of Genetic & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) Annual Meeting, visit View Source