On March 21, 2022 Compugen Ltd. (Nasdaq: CGEN), ("Compugen," the "Company"), a clinical-stage cancer immunotherapy company and a pioneer in computational target discovery, reported that it will give a presentation today on exploring the immune-tumor microenvironment (TME) using high resolution single-cell spatial transcriptomics at the Keystone Symposium: Cancer Immunotherapy: Decoding the Cancer Immunity Interactome, March 20-24 at Whistler, British Columbia, Canada (Press release, Compugen, MAR 21, 2022, View Source [SID1234610502]).

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"Compugen’s predictive computational platform is the cornerstone of our drug discovery and development capabilities. The biology of the TME is complex, and an in-depth understanding is required to develop novel cancer immunotherapies," said Anat Cohen-Dayag, Ph.D., President and CEO of Compugen. "At the Keystone Symposium, we will share how we are successfully employing high resolution single cell spatial mapping of immune cells to decipher this complexity. Leveraging our long-term expertise in computational immuno-oncology biology we have used a cutting-edge technology to provide an unprecedented view into the composition and spatial localization of individual cells in the TME. Initial findings further suggest the presence of the DNAM-1 pathway including PVRIG, an immune checkpoint discovered by Compugen, at the sites of T cell priming, including the tertiary lymphoid structures. This is exciting as it confirms what we have seen previously and further supports the rationale to block PVRIG to address immunotherapy resistance in both inflamed and less inflamed tumors. Our ability to study cancer at the spatially resolved single-cell level is expanding our understanding of the complex interactions in the TME and opens the door to new therapeutic approaches."

Compugen’s cloud-based computational platform integrates proprietary omics data, such as proteomics and spatial single-cell transcriptomics with public domain genomics and clinical metadata towards a machine learning based discovery of novel immuno-oncology specific targets, biomarkers, and mechanism of action. The computational-driven hypotheses are then rapidly tested and validated by an internal wet-lab experimental group. The validated information is integrated back into the discovery cycle, providing an additional layer of proprietary data which is being utilized to further optimize the computational predictive models. This tight in-house integration of computational prediction with experimental validation is one of Compugen’s strengths and has proved to be essential in its immuno-oncology discoveries, clinical-stage programs, and pipeline progression.

Presentation & poster details
Meeting title: Cancer Immunotherapy: Decoding the Cancer Immunity Interactome
Session title: Imaging Cancer Immunity
Presentation & poster title: Exploring the immune-tumor microenvironment using high resolution single-cell spatial transcriptomics
Lead author: Roy Granit
Oral presentation date: Monday, March 21, 2022
Poster date: Tuesday, March 22, 2022
Poster number: Poster #2041

The presentation is available on the publication section of Compugen’s website www.cgen.com.

On March 21, 2022 Diffusion Pharmaceuticals Inc. (NASDAQ: DFFN) ("Diffusion" or the "Company"), an innovative biopharmaceutical company developing novel therapies that enhance the body’s ability to deliver oxygen to areas where it is needed most, reported financial results for 2021 and provided a business update (Press release, Diffusion Pharmaceuticals, MAR 21, 2022, View Source [SID1234610467]).

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Corporate Strategy and Events

Business and financial highlights during 2021 and 2022 year-to-date include:

Declared Lead Development Program: The Company announced its intent to develop its novel, oxygen enhancing therapeutic, Trans Sodium Crocetinate ("TSC"), as an adjunct to standard of care therapy for hypoxic solid tumors in November 2021 ("Hypoxic Solid Tumor Program").

Strengthened Balance Sheet: In February 2021, the Company raised approximately $34.5 million in gross proceeds through an up-sized, follow-on public offering of its common stock with proceeds to be used to fund research and development efforts for the TSC oxygenation trials ("Oxygenation Trials") and corporate expenses. As of December 31, 2021, the Company reported a total of $37.3 million in cash and cash equivalents. As of March 18, 2022, the Company expects cash resources to fund operations through 2023.

Board Updates: Jane Hollingsworth appointed as new Board Chair in June 2021. Diana Lanchoney, M.D., and Eric Francois also elected to the Company’s board of directors in June 2021.

"We are pleased with the progress we achieved in 2021. We executed our corporate, clinical, and regulatory strategies, accelerating development of our lead product candidate, TSC. The capital we raised in the first half of 2021 allowed us to design and execute critical clinical trials to assess TSC’s therapeutic potential in treating hypoxia-related conditions using three unique short-term experimental clinical models of oxygenation," said Robert Cobuzzi, Jr., Ph.D., President and Chief Executive Officer of Diffusion.

Dr. Cobuzzi continued, "We believe the hard work demonstrated by our team in 2021 has laid a solid foundation for Diffusion’s success in 2022. The initiation of our Altitude and ILD-DLCO trials in the last quarter of 2021 represented a significant inflection point on the route to achieving the goal of demonstrating TSC’s potential to enhance the body’s ability to deliver oxygen to areas where it is needed most and potentially provide a novel treatment option for a variety of conditions complicated by hypoxia. Moreover, in late 2021 we announced our intent to design and execute a clinical program to support the use of TSC as an adjunct treatment of hypoxic solid tumors as the next step in our efforts to realize TSC’s potential. We believe targeting hypoxic solid tumors is appropriate for TSC given the significant unmet medical need, the compelling preclinical and clinical data accumulated to date, and the current, intravenous formulation of TSC. The team has been working closely with our advisors to design a Phase 2 study that we believe, if successful, will help patients and enhance shareholder value."

Clinical Trial Execution & Strategy

TCOM Trial: In March 2021, the Company completed enrollment and dosing of all participants in its Phase 1 trial of TSC utilizing a transcutaneous oxygen monitoring ("TCOM") device to evaluate the pharmacodynamic effects of TSC on peripheral tissue oxygenation in healthy, normal volunteers. As reported in June 2021, the topline results of the study showed TSC was safe and tolerable at all doses tested with no serious adverse events or dose-limiting toxicities. Furthermore, the Company announced that a positive trend in peripheral tissue oxygenation was observed among participants treated with TSC when compared to placebo-treated participants. This trend persisted through the one-hour, post-dosing measurement period. The Company believes the data from the TCOM Trial validate the thesis that TSC enhances oxygenation without causing hyperoxygenation. The data from the TCOM Trial have guided the design of the two additional Oxygenation Trials, as well as the design of the Hypoxic Solid Tumor Program.

Altitude Trial: In November 2021, Diffusion announced the dosing of the first participants in the Altitude Trial which was designed to evaluate the effects of TSC in normal healthy volunteers subjected to incremental levels of physical exertion while exposed to hypoxic and hypobaric conditions, or "simulated altitude." Due to enrollment delays experienced during early 2022 related to the omicron variant wave of the COVID-19 pandemic, the Company now anticipates completing dosing in the second quarter of 2022 and topline results are expected to be reported within one to two months of study completion.

ILD-DLCO Trial: Subsequent to receiving Investigational New Drug application clearance from the U.S. Food and Drug Administration ("FDA") in the third quarter of 2021, the Company announced the dosing of the first patient in the Phase 2 ILD-DLCO Trial in December 2021. The ILD-DLCO Trial was designed to evaluate the effects of TSC on carbon monoxide uptake through the lungs ("DLCO") and into the bloodstream of interstitial lung disease ("ILD") patients. DLCO measurement is used in the study as a surrogate measure of oxygen transfer efficiency, or uptake, from the alveoli of the lungs through the plasma, and onto hemoglobin within red blood cells. The Company intends to evaluate the effect of TSC on lung function as measured by DLCO, as well as distance covered in a standard six-minute walk test. Due to enrollment delays experienced during early 2022 related to the omicron variant wave of the COVID-19 pandemic, the effect of the omicron variant on patients with pulmonary diseases such as ILD, and staffing issues at the facilities where the ILD-DLCO Trial is being conducted, the Company now anticipates dosing to be completed by the middle of 2022, with topline results reported within two months of study completion.

Hypoxic Solid Tumor Treatment: In November 2021, based on the available preclinical and clinical data and the significant unmet medical need, including the TCOM and COVID-19 Trial results, the Company announced its intent to focus its near-term efforts on evaluating TSC as an adjunct to standard of care in the treatment of hypoxic solid tumors as a first indication. Solid tumors, which comprise approximately 90% of all adult cancers, are often affected by hypoxia, leading to treatment resistance, metastasis, and worse prognoses. Following, among other things, analyses and discussions with the Company’s oncology-focused SAB members and other external advisors over the last several months, Diffusion believes they have the necessary data to support the design and initiation of an appropriate Phase 2 trial as the first study in the Hypoxic Solid Tumor Program. The Company intends to file the study protocol with the FDA and expects to commence the trial in the second half of 2022, subject to FDA feedback and the availability of clinical drug supply.

2021 Financial Results

Diffusion had cash and cash equivalents of $37.3 million as of December 31, 2021, compared with $18.5 million as of December 31, 2020. Net cash used in operating activities during 2021 was $14.5 million, compared with $13.6 million used during 2020.

Research and development expenses were $8.5 million for 2021, compared with $9.4 million for 2020. The decrease was primarily attributable to lower project spending due to the completion and/or wind-down of the Company’s clinical studies evaluating TSC in COVID-19, glioblastoma multiforme brain cancer, and stroke.

General and administrative expenses were $7.4 million for 2021, compared with $6.4 million for 2020. The increase was primarily driven by increased headcount resulting in higher compensation expense and other costs associated with the hiring of new employees, as well as an increase in expense related to consulting services.

In 2021, the Company also recognized a non-recurring $8.6 million non-cash impairment charge related to the write down of its DFN-529 IPR&D asset.

On March 21, 2022 Aura Biosciences Inc. (NASDAQ: AURA), a clinical-stage biotechnology company developing a novel class of virus-like drug conjugate (VDC) therapies for multiple oncology indications, reported that the European Commission has granted Orphan Drug Designation to AU-011, its first VDC product candidate, for the treatment of uveal melanoma (Press release, Aura Biosciences, MAR 21, 2022, View Source [SID1234610486]). The designation of uveal melanoma includes choroidal melanoma as well as melanomas of the iris and the ciliary body. Choroidal melanoma represents approximately 90% of uveal melanomas.

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"There are currently no approved drug therapies for the treatment of early-stage choroidal melanoma, and receiving Orphan Drug Designation from EMA underscores the unmet need that AU-011 could fill for patients with this life-threatening disease," said Mark De Rosch, Ph.D., Chief Operating Officer and Head of Regulatory Affairs. "We have alignment with U.S. and European agencies on our pivotal program and are on track to initiate this program before the end of 2022."

The European Commission grants Orphan Drug Designation for medicinal products intended to treat life-threatening or chronically debilitating conditions that affect fewer than five in 10,000 people in the European Union (EU) and when no satisfactory method of diagnosis, prevention, or treatment of the condition can be authorized. The designation provides certain benefits and incentives in the EU, including protocol assistance, fee reductions, and ten years of market exclusivity once the medicine is on the market.

AU-011 was previously granted Orphan Drug Designation for the treatment of uveal melanoma by the U.S. Food and Drug Administration.

About AU-011

AU-011 is a first-in-class virus-like drug conjugate (VDC) therapy in clinical development for the first line treatment of choroidal melanoma. The virus-like component of the VDC selectively binds unique heparin sulphate proteoglycans (HSPGs), which are modified and overexpressed on the tumor cell surface of malignant cells in the choroid and AU-011 delivers a potent cytotoxic drug that is activated with infrared light. Upon activation with an ophthalmic laser, the cytotoxic drug rapidly and specifically disrupts the cell membrane of malignant cells with a pro-immunogenic cell death that can activate the immune system generating long term anti-tumor immunity. The unique specificity of tumor binding by the VDC enables the preservation of key eye structures, which may allow for the potential of preserving patients’ vision and reducing other long-term complications of radiation treatment. AU-011 can be delivered using equipment commonly found in an ophthalmologist’s office and does not require a surgical procedure, pointing to a potentially less invasive, more convenient therapy for patients and physicians. AU-011 for the treatment of choroidal melanoma is currently in Phase 2 clinical development and the company plans to expand the clinical program into choroidal metastasis.

On March 21, 2022 RNAGene Inc., a South Korean biotech which specializes in the development of mRNA (messenger ribonucleic acid)-based therapeutics, reported that it has signed a Collaborative Research Agreement with PharmAbcine Inc. (KOSDAQ: 208340ks), a clinical-stage biotech company focusing on the development of next-generation antibody therapeutics (Press release, RNAGene, MAR 21, 2022, View Source [SID1234610504]).

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Under the agreement, both companies will collaborate to develop next-generation mRNA-based antibody therapeutics by utilizing the proprietary mRNA platform from RNAGENE.

Unlike the existing antibody treatments, in which antibodies are artificially produced from external cell lines, mRNA-based therapies involve administration of mRNA in patients so that patients’ own cells can produce the encoded therapeutic antibody.

"The Company entered this agreement because this partnership gives us an extraordinary opportunity to expand our development strategy and discover new possibilities in the mRNA therapeutic area," said Dr. Jin-San Yoo, CEO of PharmAbcine. "Through this collaboration, both parties will explore new potentials between the two platforms and develop a new therapeutic approach to help patients with unmet medical needs."

"One of the key strengths of mRNA therapy is that it is simple and cost-effective because there is no need to produce antibodies externally in a facility," said Dr. Woo Gil Lee, CEO of RNAGENE. "We are delighted to have PharmAbcine, one of the leading antibody-based therapeutic companies, as a partner for this collaborative research, and we are confident that our advanced mRNA technology platform will help create alternative therapeutic options in oncology field."

On March 21, 2022 Tempus, a leader in artificial intelligence and precision medicine, reported the launch of EDGE, an innovative platform that allows pathologists to access developing AI models intended to identify specimens with potentially actionable biomarkers using a single hematoxylin and eosin stain (H&E) slide (Press release, Tempus, MAR 21, 2022, View Source [SID1234610487]). With access to over 50 petabytes of de-identified multimodal data, Tempus is developing AI models with the aim to identify patients who would benefit from additional testing and may qualify for targeted therapies, including those in clinical trials, earlier in their cancer care journey.

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The model portfolio, called Tempus HistogenomicsTM, features digital pathology algorithms that are being developed to use a single, whole slide H&E image to predict the potential of biomarkers, such as microsatellite instability (MSI) or homologous recombination deficiency (HRD) status, in patients whose samples are not ordinarily sequenced.

Tempus is creating a network of pathology labs, called NAPA, across the country that can access the company’s growing portfolio of investigational AI models designed to identify patients that are more likely to have targetable biomarkers. In addition, Tempus is collaborating with some of the world’s leading pharmaceutical and biotech companies to leverage the EDGE platform and NAPA network to identify unique biomarkers and co-develop novel AI applications.

"We believe there is a unique opportunity to advance patient care by integrating digital pathology AI tools into our daily clinical practice," said Matthew Leavitt, MD, CEO of PathNet Labs, a national network of digital pathology practices. "This collaboration will carefully validate safe-use clinical applications of Tempus Histogenomics, directly comparing these algorithms with conventional ancillary testing methodologies while seeking to empower our pathologists with computational tools that can assist in identifying patients most likely to benefit from new therapies."

"EDGE is one of the many ways in which we will leverage our AI-enabled platform and library of multimodal data, including our diverse matched digital pathology and molecular datasets, to more effectively identify the right treatments for the right patients even earlier in their treatment journey," said Abdul Hamid Halabi, GM and SVP of Translational AI at Tempus. "This platform will bring actionable AI to pathologists’ fingertips and support them in identifying optimal therapeutic paths for patients, while also facilitating the discovery of new biomarkers for researchers developing new, targeted therapies."

In December 2021, Tempus announced a collaboration with the Data Science and Oncology teams at Janssen Research & Development LLC (Janssen) aimed at jointly creating algorithms intended to further patient pre-screening efforts for specific cancer indications, including biomarker-selected cohorts, for Janssen’s clinical trials. Tempus also announced a strategic collaboration with AstraZeneca last year, which intends to utilize Tempus’ AI-enabled platform and multimodal data to advance novel therapeutic programs across AstraZeneca’s diverse oncology pipeline.