New preclinical data from AC Immune SA, of Lausanne, Switzerland, show its TAR DNA-binding protein 43 (TDP-43) PET tracers were characterized using a radio-binding assay, identifying several distinct chemical series of compounds that bind to recombinant and brain-derived TDP-43 aggregates. Selected compounds also demonstrate direct target engagement on patient-derived brain tissue as assessed by a positive signal in a proprietary high-resolution autoradiography assay that co-localized with pathological TDP-43, the company said. AC Immune’s therapeutic and diagnostic programs target pathological forms of alpha-synuclein and TDP-43.
Affinivax Inc., of Cambridge, Mass., said it received an award from CARB-X to develop a vaccine targeting Staphylococcus aureus bacterial infections. The award substantially funds all development activities for the program through an initial clinical study to assess safety and preliminary efficacy. The award commits funding of up to $22 million by achieving manufacturing, preclinical and clinical development milestones.
Allarity Therapeutics A/S, of Hørsholm, Denmark, said its preclinical study of dovitinib, a pan-tyrosine kinase inhibitor, yielded positive data as an antitumor activity in osteosarcoma. Dovitinib alone and combined with an anti-PD-1 strategy in a mouse model showed treatment with dovitinib compared to control treatment increased survival time by 50%. Antitumor growth activity was also seen for dovitinib as a single agent. The study found no significant antitumor activity in mice treated with the single-agent anti-PD-1 antibody at the dosage and dosing schedule.
Preclinical data showed compounds from Arkuda Therapeutics Inc., of Watertown, Mass., increased activity of a key lysosomal enzyme deficient in Parkinson’s disease patients with GBA1 mutations. The new data demonstrated that an Arkuda small molecule increased the enzymatic activity of beta-glucocerebrosidase, which plays a key role in normal lysosome function and is essential for the health of cells in the CNS, the company said.
Asceneuron SA, of Lausanne, Switzerland, said it received a $2.2 million award from the Alzheimer's Drug Discovery Foundation for a phase I study of its O-GlcNAcase inhibitor, ASN-51. The trial will recruit healthy volunteers and Alzheimer's disease patients in Europe and Australia starting in the second quarter of 2021. Interim data are expected in the third quarter of 2021. O-GlcNAcase is a drug target in CNS drug development, the company said, since deficient glycosylation patterns of intracellular proteins have been associated with diseases of aging and neuronal dysfunction.
Cambridge Allergy Ltd., of Cambridge, U.K., said it was awarded a £1.1 million (US$1.53 million) through the Innovate UK Biomedical Catalyst Award program administered by Innovate UK. The money will be used to accelerate CA-002, its oral peanut allergy immunotherapy, for phase III trials beginning in the first quarter of 2021.
Fresh data from Capricor Therapeutics Inc., of Los Angeles, demonstrated that manipulation of cell culture conditions with GSK3 inhibitors led to up-regulation of beta-catenin and down-regulation of CD90, leading to a more consistent and potent cell line. Activation of canonical Wnt signaling, the company added, correlated with increased enrichment of therapeutically relevant miRs, including miR-22 and miR-146a, both implicated in cell potency. Capricor's lead candidate, CAP-1002, is an allogeneic cell therapy in clinical development for treating Duchenne muscular dystrophy and the cytokine storm associated with COVID-19.
Centogene NV, of Cambridge, Mass., Rostock, Germany, and Berlin, said it has recruited and genetically tested 10,000 participants in its Parkinson’s disease study at more than 120 sites around the world. In 2018, Centogene struck a strategic collaboration with Denali Therapeutics Inc. for the targeted global identification and recruitment of Parkinson’s disease patients with mutations in the LRRK2 gene. Mutations in LRRK2 are a genetic cause of Parkinson’s disease. Patients enrolled in ROPAD with an LRRK2 mutation may be eligible for participation in future therapeutic clinical studies, the company said.
Compugen Ltd., of Holon, Israel, said a review article published in Cancer Discovery provides an overview of the DNAM-1 axis, including axis checkpoint molecules DNAM-1, TIGIT, PVRIG and CD96, along with their ligands, PVR and PVRL2. Compugen has an ongoing phase I/II study testing the triple simultaneous blockade of PVRIG, TIGIT and PD-1.
Durect Corp., of Cupertino, Calif., said a peer-reviewed research paper published in The Journal of Lipid Research describes the binding sites and proposed mechanism of action of its lead candidate, DUR-928, an endogenous sulfated oxysterol and epigenetic regulator. Data showed the drug binds to and inhibits the activity of DNA methyltransferases (DNMTs), DNMT-1, 3a and 3b, epigenetic regulating enzymes that add methyl groups to DNA. As such, by inhibiting DNMT activity, DUR-928 inhibits DNA methylation, thereby regulating the expression of genes that modulate crucial cellular activities, including those associated with cell death, stress response and lipid biosynthesis.
Eli Lilly and Co., of Indianapolis, and Biolojic Design Ltd., of Tel Aviv, Israel, said they entered a research collaboration and license agreement to use Biolojic’s AI-based multibody platform to discover and develop an antibody-based therapy to treat diabetes. Under the terms, Lilly will pay research fees associated with the collaboration. Biolojic also is eligible to receive up to a total of $121 million, consisting of potential development and commercialization milestones and a promissory note that may be convertible into Biolojic equity at a future date. Additionally, Biolojic is eligible to receive tiered royalties in the low- to mid-single digits on product sales should Lilly successfully commercialize a therapy from the collaboration.
Exevir Bio BV, of Ghent, Belgium, reported preprint communication on BioRxiv from research by VIB scientists on the XVR-011 antibody, demonstrating potent viral neutralizing activity, protection against infection by SARS-COV-2 and therapeutic efficacy in in vivo mouse and hamster models and minimized development of alveolar damage.
Genome and Co., of Seoul, South Korea, said it signed a second clinical trial collaboration and supply agreement with Merck KGaA, of Darmstadt, Germany, and Pfizer Inc., of New York, with the aim of developing an immuno-oncology microbiome therapeutic. The objective of the phase IIa trial, designated Study 201, is to investigate the efficacy and safety of the combination of GEN-001 and PD-L1 inhibitor avelumab for gastric and gastroesophageal junction adenocarcinomas that continue to be difficult to treat. The trial will be conducted simultaneously at six or more hospital and medical center sites in the Republic of Korea. The latest agreement comes a year after the companies first agreed to test the combination of avelumab and GEN-001. Under the terms, Genome and Co. will be the sponsor of the studies, and Merck and Pfizer will supply avelumab for both studies. Both parties will have access to the clinical data.
Greenlight Biosciences Inc., of Medford, Mass., and TFF Pharmaceuticals Inc., of Austin, Texas, said they partnered for feasibility studies aimed at opening broader global vaccine distribution through production of a shelf-stable powder form of a mRNA COVID-19 vaccine that would be easily reconstituted prior to injection and not require the extreme cold chain of current RNA vaccines. The agreement will use TFF’s dry powder technology with Greenlight’s COVID-19 mRNA vaccine candidate. Financial terms were not disclosed.
Insilico Medicine Ltd., of Hong Kong, an AI drug discovery company, said UCB SA, of Brussels, will use Insilico’s Chemistry42 in its internal drug discovery pipeline. The technology is designed to provide the ability to design novel hit compounds that satisfy multiple predefined parameters rapidly and streamline lead optimization. Terms were not disclosed.
Medolife Rx Inc., of Burbank, Calif., a majority owned subsidiary of Quanta Inc., reported preclinical results on lead candidate Escozine, a formulation consisting of small-molecule peptides derived from Rhopalurus princeps scorpions amplified by the company’s polarization technology, showing that, at maximum dose levels, the product is non-toxic and safe. Escozine is targeting COVID-19, and the company has already completed a human trial in the Dominican Republic.
Nantkwest Inc., of El Segundo, Calif., said its stockholders approved proposals related to the pending merger with Immunitybio Inc., of Culver City, Calif. The merger was expected to close March 9, with the combined company’s shares beginning to trade on Nasdaq on March 10 under the ticker IBRX.
Neurorx Inc., of Radnor, Pa., and TFF Pharmaceuticals Inc., of Austin, Texas, said they entered a feasibility and material transfer agreement, under which Neurorx will deliver Zyesami (aviptadil, synthetic VIP) materials to TFF for feasibility formulation work and testing. The goal is to formulate and identify an optimal, long-term-stable formulation of Zyesami in a dry powder form that has superior aerosol properties for delivery directly to the lungs. Zyesami is a synthetic form of vasoactive intestinal peptide, which is known to protect the alveolar type II cell that is targeted by the SARS-CoV-2 virus. Terms of the agreement were not disclosed.
Oragenics Inc., of Tampa, Fla., said it entered a material transfer agreement with Biodextris Inc., of Laval, Quebec, for the use of three intranasal mucosal adjuvants in the company’s Terra CoV-2 vaccine against COVID-19. BDX-100, BDX-300 and BDX-301 are proteosome-based adjuvants comprising proteins and lipopolysaccharides with improved attributes, including enhanced immune response, manufacturing efficiency and the benefits of intranasal vaccine administration. The initial agreement calls for the three intranasal adjuvants to be used in combination with the Oragenics’ antigen vaccine candidate as part of the preclinical immunological evaluation of Terra CoV-2, for the prevention COVID-19. The deal allows for the future collaboration regarding the intranasal delivery of vaccine during clinical development with the opportunity to enter a commercial agreement upon regulatory approval of the intranasal vaccine.
Orionis Biosciences BV, of Ghent, Belgium, and the University of Texas MD Anderson Cancer Center’s Therapeutics Discovery division said they launched Project Helios, a research collaboration designed to use genome-scale mapping of drug-target interactions. The project will focus initially on developing therapies for unmet needs in oncology, with the possibility of expanding to additional therapeutic areas in the future.
Promatix Inc., of London, was launched by Alsa Ventures to develop therapeutics for cancer using its multi-omic database, Txpro, to mine for novel, and validate existing, drug targets. The company will combine its platform with third-party public and private databases to create a multi-omic engine to help identify targets for rapid early development. It will continue to be incubated in the near term with the Alsa Group, which is providing seed funding to establish its first suite of targeted research projects.
PTC Therapeutics Inc., of South Plainfield, N.J., and the Spinal Muscular Atrophy Foundation said they entered a new collaboration focused on regenerative medicine to further advance scientific research in spinal muscular atrophy (SMA) and other neuromuscular disorders with the goal of developing new treatments. The partnership will provide funding, managed by the SMA Foundation, to academic institutions and other collaborators to advance foundational research in the area of regenerative medicine.
Scisparc Ltd., of Tel Aviv, Israel, said it entered an agreement with The Sheba Fund for Health Services and Research to perform a preclinical study for the evaluation of the company's SCI-210 drug development program, a pharmaceutical preparation containing non-psychoactive cannabinoid cannabidiol (CBD) and palmitoylethanolamide for the treatment of status epilepticus (SE). The study is expected to test the combination vs. CBD single treatment in a mouse model of SE.
Selexis SA, of Geneva, and Boston Immune Technologies and Therapeutics Inc. (BITT), of Boston, said they entered a service agreement to develop the cell line for BITR-2101, BITT’s lead tumor necrosis factor receptor 2 antagonist antibody for the treatment of cancers and infectious diseases. Under the agreement, BITT will use Selexis’ Suretechnology, a platform comprising modular technologies designed to overcome the complex challenges of protein-expression. Terms were not disclosed.
Soligenix Inc., of Princeton, N.J., said it received preliminary approval for a tax credit from the New Jersey Economic Development Authority's New Jersey Technology Business Tax Certificate Transfer program. As a result, the company anticipates being able to transfer that credit and receive about $865,000 in net proceeds.
Sorrento Therapeutics Inc., of San Diego, and the Icahn School of Medicine at Mount Sinai entered an exclusive license agreement for a collection of antibodies having SARS-CoV-2 neutralizing properties that were developed by Mount Sinai. The license also contemplates the two pursuing future collaborations in developing humanized monoclonal antibodies for therapeutic applications. The collaboration already has produced Covishield, a combination of two monoclonal antibodies designed to protect against disease caused by existing and emerging variants of SARS-CoV-2. Using the early pandemic variants as well as the emerging variants of concern (VOCs) that have increased in prevalence globally through the course of the pandemic, Sorrento identified candidate monoclonal antibody combinations with activity against the breadth of those VOCs, including the U.K. (B.1.1.7), South Africa (B.1.351) and Japan/Brazil (B.1.128) variants.
Surface Oncology Inc., of Cambridge, Mass., said it entered a clinical trial collaboration with Merck & Co. Inc., of Kenilworth, N.J., through a subsidiary, to evaluate the safety and efficacy of combining Surface’s SRF-388, an antibody targeting IL-27, with Keytruda (pembrolizumab) in a phase I study in solid tumors, with a focus on liver and kidney cancers.
Taysha Gene Therapies Inc., of Dallas, said it inked a multiyear collaboration with Yong-Hui Jiang, professor and chief of medical genetics at Yale University, to advance next-generation mini-gene payloads for AAV gene therapies for the treatment of neurodevelopmental disorders. Taysha will have an exclusive option on new payloads, constructs and intellectual property associated with, and arising from, the research conducted under the agreement.
Twelve Bio, of London, a new portfolio company of Arix Bioscience plc, has been formed to develop engineered Cas12a nucleases for therapeutic gene editing. Arix is the sole investor with a 49% ownership stake. Cas12a is a CRISPR nuclease with several advantages over other gene editing tools, according to Arix, including small size, high specificity and the ability to target sections of DNA not amenable to other Cas nucleases such as Cas9.
Valo Therapeutics Ltd., of Helsinki, said it completed a collaboration with Immunoscape Pte Ltd., of Singapore and San Diego, to identify immunogenic peptides for the development of a pan-coronavirus vaccine. The analysis was performed directly on blood cells from COVID-19 convalescent patients with different clinical outcomes and has allowed the identification of specific CD8+ T-cell responses relevant to SARS-CoV-2. In follow-up, Valo will use its Peptivax technology to coat a spike-expressing adenoviral vector vaccine with HLA-restricted peptides identified by Valo working with the University of Helsinki Immunovir Therapy Lab. The study indicated those peptides may be associated with a clinically beneficial T-cell immune responses to SARS-CoV-2's highly conserved genes.
Volastra Therapeutics Inc., of New York, and Dewpoint Therapeutics Inc., of Boston, said they signed a research and development partnership to discover modulators of biomolecular condensates to prevent cancer progression and metastasis. The work will initially focus on early drug discovery, with the option for future joint development and commercialization.
Yumanity Therapeutics Inc., of Boston, reported preclinical results at the International Conference on Alzheimer’s and Parkinson’s Diseases demonstrating pharmacological, physiological and behavioral preclinical proof of concept for lead program YTX-7739 in a Parkinson’s disease (PD) mouse model. Research showed the drug prevented motor function deficits in diseased mice after four months of oral dosing compared to placebo-treated mice. YTX-7739 concentrations in the brain reached a level that engaged and inhibited stearoyl-CoA desaturase (SCD) activity consistent with in vitro studies. In addition, SCD inhibition by YTX-7739, which can be measured by a quantitative biomarker, resulted in a decrease in monounsaturated fatty acids in both plasma and brain. Monounsaturated fatty acids may contribute to alpha-synuclein pathology. Mice treated with YTX-7739 also exhibited enhanced survival of dopaminergic neurons, the type of neuron that selectively dies in the brains of patients with PD.