Appili Therapeutics Inc., of Halifax, Nova Scotia, said interim preclinical data presented at the Chemical and Biological Defense Science and Technology Conference in Cincinnati showed that administration of its vaccine candidate, ATI-1701, resulted in complete (100%) protection 90 days after vaccination from a lethal exposure to the pathogen Francisca tularensis. That time period is the longest vaccine protection period tested to date in that model, the company said. Researchers will conduct an additional evaluation of vaccine efficacy up to 365 days in the same model in the first half of 2020. The U.S. Defense Threat Reduction Agency, an arm of the U.S. Department of Defense, is funding the trial.

CNS Pharmaceuticals Inc., of Houston, completed an agreement with Houston Pharmaceuticals Inc. (HPI), also of Houston, to obtain the rights to a worldwide, exclusive royalty-bearing license for the chemical compound commonly known as berubicin. The deal with HPI grants CNS the exclusive right to develop certain patented chemical compounds for use in the treatment of cancer globally. In its agreement with HPI, CNS acquired multiple U.S. patents for berubicin. CNS intends to apply for orphan drug status for berubicin with the FDA for the treatment of malignant glioma.

Codiak Biosciences Inc., of Cambridge, Mass., said the first preclinical data for its Engex platform program, exoASO, presented at the American Association for Cancer Research’s Special Conference on Tumor Immunology and Immunotherapy in Boston, demonstrate the potential of engineered exosomes incorporating an antisense oligonucleotide (ASO) to selectively reprogram tumor-associated macrophages and generate potent antitumor activity. The data show the utility of exoASO in selectively targeting classically undruggable transcription factors and successfully reprogramming immunosuppressive macrophages, the company said.

Cytodyn Inc., of Vancouver, Wash., said leronlimab inhibited a human colon carcinoma cell line, SW480 cells, metastases to liver and lung in a well-accepted mouse model. The human colon carcinoma cells were implanted in the colon walls of immunodeficient mice and treated with a control antibody or leronlimab. After four weeks, the mice were sacrificed and the lungs and livers removed for analysis of invasion of the colon cancer cells.  Leronlimab produced statistically significant inhibition of metastases to lung and liver in that mouse model. Leronlimab is a humanized IgG4 monoclonal antibody that blocks CCR5.

Excision Biotherapeutics Inc., of Philadelphia, presented data at the International Symposium on Neurovirology in Oakland, Calif., showing that gene editing strategies are promising tools for eradicating viral genomes and possible cures for herpes simplex virus (HSV) and John Cunningham (JC) virus-induced progressive multifocal leukoencephalopathy (PML). There are no current effective therapies for the latter indication. Patients with HSV have limited treatment options, too. Researchers working with collaborators at the Lewis Katz School of Medicine at Temple University demonstrated the ability to remove JC virus and HSV from cell lines and animals using CRISPR.

Positive preclinical data from Genprex Inc., of Austin, Texas, and the University of Texas MD Anderson Cancer Center show that TUSC2 immunogene therapy conferred sensitivity to checkpoint blockade for some resistant metastatic human cancers, including the KRAS and LKB1 mutations, in mice with human immune cells with lung metastases. When TUSC2 was combined with anti-PD-1 therapy, Keytruda (pembrolizumab, Merck & Co. Inc.), in humanized mice with KRAS and LKB1 lung metastases, there was significantly increased antitumor activity than when compared to either agent alone. That combination and model also demonstrated TUSC2-related natural killer (NK) cell activation. A significantly higher percentage of CD56+ NK and CD56+CD59+ active NK cells were found in the mice that received TUSC2 alone and in those that received the combination of TUSC2 and pembrolizumab than in those that received pembrolizumab alone. Shares of Genprex (NASDAQ:GNPX) gained 20 cents, or 49%, to close Tuesday at 62 cents.

Idorsia Ltd., of Allschwil, Switzerland, and Antares Pharma Inc., of Ewing Township, N.J., plan to develop a drug-device product combining Idorsia's P2Y12 receptor antagonist, selatogrel, with the Antares Quickshot auto-injector. Idorsia is preparing for a phase III study to investigate the efficacy and safety of selatogrel following subcutaneous self-administration for treating a suspected acute myocardial infarction (AMI) in adult patients at risk of recurrent AMI. Selatogrel is a single-dose, disposable drug-device product, consisting of a self-administration auto-injector containing a drug prefilled syringe. The product will be tested through studies ahead of the phase III. 

Kadimastem Ltd., of Ness Ziona, Israel, reported reaching the objectives for its proof-of-concept study of Isletrx, a purified functional human pancreatic islet cells product for treating insulin-dependent diabetes. Results showed efficacy by prolonged normalized blood sugar levels in treated immunocompetent diabetic mice throughout the three-month preclinical study. No disease or treatment-related complications were observed and all treated animals remained healthy throughout the study. In comparison, a control group of nontreated diabetic mice presented severe hyperglycemia, leading to the death of the nontreated mice. Isletrx protected the islet cells from host immune system response, without the need for immunosuppressive drug treatment. Based on the results, Kadimastem plans to advance the development program to the clinic. 

New preclinical data on treating pulmonary arterial hypertension with PBI-4050 from Liminal Biosciences Inc., of Rockville, Md., showed the G protein-coupled receptor reduced angioproliferative artery smooth muscle cell proliferation and microvascular endothelial cell endoplasmic reticulum stress. PBI-4050, Liminal’s lead drug candidate, is an agonist of GPR40 and GPR120, and an antagonist of GPR84. The company is considering a phase III study of PBI-4050 for treating Alstrom syndrome.  

Nkarta Therapeutics Inc., of South San Francisco, said a preclinical study presented at the American Association for Cancer Research, Tumor Immunology and Immunotherapy meeting in Boston described the evaluation of a series of engineered NK cells targeting CD19, a tumor marker found in a variety of B-cell malignancies. NK cells were expanded using Nkarta’s stimulatory cell line, then transduced with a γ-retrovirus encoding different CD19 CARs and membrane-bound IL-15, Nkarta’s approach to increase NK cell persistence. In a series of in vitro and in vivo experiments, Nkarta scientists identified a CAR construct containing an OX40 co-stimulatory domain as optimal for further development. The research was used to inform the selection of NKX-019, Nkarta’s clinical candidate, with an IND expected in the third quarter of 2020.

Oligomerix Inc., of New York, said preclinical data published in the Journal of Alzheimer’s Disease demonstrated that an oral small-molecule drug inhibits the formation of neurotoxic tau oligomers in an animal model of tau aggregation most relevant to Alzheimer’s. The study showed that the compound blocked tau self-association, which is the earliest step in the toxic tau aggregation cascade, and inhibited the downstream events that lead to tau fibril formation. The tau oligomer inhibitor was administered in feed at three different doses, and was well-tolerated by the treated mice, with no adverse events or behavioral abnormalities observed. Oligomerix is conducting IND-enabling studies and aims to be in the clinic in 2021.

Omeros Corp., of Seattle, reported new findings on GPR174, its cancer immunotherapy target, demonstrating that GPR174-deficiency enhances antitumor immune responses in animals. The studies were conducted in mouse models of melanoma and of colon carcinoma, each of which was modified to partially deplete regulatory T cells. GPR174 deficiency in those mice resulted in significantly reduced tumor growth and improved survival of the animals (p=0.006 in melanoma; p=0.03 in colon cancer) vs. normal mice. Findings were presented at the American Association for Cancer Research Conference on Tumor Immunology and Immunotherapy meeting in Boston.

Oncoceutics  Inc., of Philadelphia, said data published in Nature Communications show selective antagonism of the G protein-coupled receptor (GPCR) dopamine receptor D2 (DRD2) by Oncoceutics’ lead candidate, imipridone (ONC-201). The drug was found to specifically bind and antagonize DRD2, and its functionally redundant family member DRD3, without affecting other dopamine receptors, other GPCRs, nuclear hormone receptors, kinases or other drug targets of FDA-approved cancer therapies.

Palleon Pharmaceuticals Inc., of Waltham, Mass., reported preclinical data for lead candidate EAGLE-Her2, showing that selectively removing the terminal sialic acids of sialoglycans within the tumor microenvironment effectively inhibits the Siglec-Sialoglycan immune checkpoint and reinvigorates both the innate and adaptive responses to cancer. The research was presented at the Annual Protein & Antibody Engineering Summit meeting in Lisbon, Portugal.

Personalis Inc., of Menlo Park, Calif., said it inked a collaboration with Merck KGaA, of Darmstadt, Germany, to investigate biomarkers of response and mechanisms of resistance to cancer therapies. Merck will utilize Personalis’ newest cancer immunogenomics platform, Immunoid Next, for clinical biomarker identification and development. Financial terms were not disclosed.

Redx Pharma plc, of Alderley Park, U.K., said Innovate UK awarded Biomedical Catalyst funding to Redx and Medicines Discovery Catapult to fund development and validation of a panel of translational biomarkers to assess therapeutics in idiopathic pulmonary fibrosis. The collaboration will enable preclinical assessment and inform the clinical development strategy for Redx's Porcupine (RXC-006) and ROCK2 inhibitors for fibrotic diseases. The total grant awarded to fund the project is £515,595 (US$665,850).

Regenerx Biopharmaceuticals Inc., of Rockville, Md., said data published in the Journal of Investigative Surgery demonstrated the protective effect of thymosin beta 4 (Tβ4) in an ischemic acute kidney injury model in laboratory rats. Researchers said administration of Tβ4 to animals with induced kidney injury significantly reduced ischemic acute kidney injury and, in metabolic studies, demonstrated that Tβ4 appears to work by reduction of inflammation, inhibition of the extracellular remodeling process, and suppression of the apoptosis cascade via modulation of renal redox status. 

Therapix Biosciences Ltd., of Tel Aviv, Israel, said it entered a memorandum of understanding with Heavenly Rx Ltd., a consumer hemp CBD company, in which the two agreed to pursue a business combination. The parties agreed to enter an initial share exchange, in which Therapix will issue up to 20% of its outstanding American depositary shares to acquire, from certain shareholders, an interest in Heavenly Rx, a move intended to represent an initial step toward the proposed business combination. Therapix is developing cannabinoid-based therapies, including THX-110, a drug based on tetrahydrocannabinol for treating Tourette syndrome, obstructive sleep apnea and pain.

Zelluna Immunotherapy A/S, of Oslo, Norway, and Glycostem Therapeutics BV, of Oss, the Netherlands, said they entered a development, license and supply agreement that will focus on the development and manufacture of allogeneic TCR guided NK cell therapies (TCR-NKs) for the treatment of patients with cancer. Zelluna will lead development and commercialization of TCR-NK products with its TCRs and TCR development competence, while Glycostem will contribute by manufacturing clinical-grade umbilical cord-derived NK cells and providing NK-cell expertise, product process development, and clinical and commercial supply. Financial terms were not disclosed.

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