Bluebird Bio Inc., of Cambridge, Mass., offered updates that included a revised diagnosis for the previously reported case of myelodysplastic syndrome (MDS) in its phase I/II study of Lentiglobin for sickle cell disease (SCD) (bb1111). The case of MDS reported in February has been further assessed following the review of results from additional tests. The treating investigator has concluded this is not a case of MDS and has revised the diagnosis to transfusion-dependent anemia. Bluebird has reported the update to regulatory agencies and study investigators. The company continues to work with the treating investigator to determine the potential cause of this patient’s anemia.
Can-Fite Biopharma Ltd., of Petach Tikva, Israel, said that in preparation for anticipated potential marketing registration filings for its drug candidates piclidenoson and namodenoson in the U.S. and Europe, the company has initiated a series of preclinical studies required by regulators. As part of filings in the U.S. and Europe, the company is required to submit certain preclinical data be submitted along with the pivotal phase III data.
Cannabics Pharmaceuticals Inc., of Tel Aviv, Israel, launched a new research program using psylocibin and psilocin. The company was able to obtain a license from the Israeli Ministry of Health to conduct scientific research using the compounds at its in-house laboratory facilities.
D&D Pharmatech Inc., of Geonggi-Do, South Korea, signed a sponsored research agreement with Yale University relating to optimization of two novel approaches that allow immune system cells and drug molecules to bypass the blood-brain barrier for the treatment of brain cancer and other disorders. The targets of the approaches include brain cancer and other central nervous system diseases.
Elektrofi Inc., of Boston, signed a collaboration and license agreement with Argenx BV, of Breda, the Netherlands, to explore new subcutaneous formulations for therapeutic products directed at the human neonatal Fc receptor, including efgartigimod, and up to one additional target. The Elektrofi-enabled formulations are aimed to promote additional optionality for patients through at-home and self-administration capabilities. Argenx will make an up-front payment and future milestones payments across both targets pending achievement of predefined development, regulatory and commercial milestones. Elektrofi will also receive a mid-single-digit royalty on sales of commercialized products.
Gain Therapeutics Inc., of Bethesda, Md., signed a multitarget collaboration agreement with Zentalis Pharmaceuticals Inc., of New York, to discover product candidates for the treatment of cancer. Gain will use its SEE-Tx computational platform technology to identify new sites on target proteins for potential use in oncology. SEE-Tx applies a computational algorithm and supercomputer processing to the published 3D structure of proteins to discover new binding sites with the ability to modulate protein function. The intended output is newly discovered targets or target protein interactions that can then be drugged for therapeutic benefit to intervene on protein misfolding.
Hoth Therapeutics Inc., of New York, said its cancer therapeutic exhibited positive results in humanized mast cell neoplasm models, representative in vitro and in vivo models for aggressive, mast cell-derived cancers such as mast cell leukemia and mast cell sarcoma. The cancer therapeutic, which is in development, uses mRNA frame shifting that induces apoptosis of neoplastic mast cells. The findings show it rapidly induces apoptosis and cell death in neoplastic mast cells, Hoth said. Systemic administration of the therapeutic in an in vivo humanized mast cell neoplasm model showed a significant reduction in tumors and neoplastic mast cell infiltration from the therapeutic group compared to the vehicle control group, the company added.
Lamassu Pharma LLC, of Durham, N.C., said it is developing an acute pancreatitis treatment, RABI-767, that advances the understanding of fatty acids’ role in COVID-19. RABI-767 is a small-molecule lipase inhibitor licensed from the Mayo Foundation for medical education and research. Researchers aim to mitigate the toxicity and organ failure associated with the disease that causes lengthy hospitalizations and death, the company said. Lamassu has completed preclinical studies on RABI-767 and the results indicate the compound decreases the breakdown or release of unsaturated and saturated fats during acute pancreatitis attacks.
Lineage Cell Therapeutics Inc., of Carlsbad, Calif., and Immunomic Therapeutics Inc., of Rockville, Md., will collaborate to generate a candidate derived from Lineage’s VAC allogeneic cancer immunotherapy platform and targeting a proprietary Tumor Associated Antigen (TAA) construct provided by Immunomic for treating glioblastoma multiforme (GBM). Lineage and Immunomic will collaborate in the manufacturing and clinical development of a candidate. Following full development and delivery of cGMP VAC product material, Immunomic will assume full and independent clinical and commercial responsibility and further advancement of the program. Lineage will be entitled to up-front payments totaling $2 million anticipated in the first year and up to $67 million in development and commercial milestones across multiple indications and territories. Lineage also will be eligible to receive royalties up to 10% on net sales of future products.
Oncotelic Therapeutics Inc., of Agoura Hills, Calif., made a presentation at the American Association for Cancer Research annual meeting, titled “Combination therapy of anti-sense oligonucleotide targeting TGF-β2 (TASO) and IL-2 (Proleukin) has anti-cancer effect in solid cancer.” The combination treatment of trabedersen and low dose Proleukin (aldesleukin, Clinigen Group plc) decreased cancer cell viability in an in vitro experiment in solid cancer cell lines. Melanoma and triple-negative breast cancer tumor growth was delayed in a humanized NSG mouse model by trabedersen and low-dose IL-2 combination therapy, and tumor growth delay was statistically significant to trabedersen alone or IL-2-alone group.
Resverlogix Corp., of Calgary, Alberta, said new research revealed the interaction between SARS-CoV-2 protein E with BET proteins, suggesting that apabetalone, RVX-208, a small-molecule bromodomain and extra-terminal inhibitor, has the potential to combat COVID-19 through a dual mechanism. Apabetalone treatment prevents SARS-CoV-2 from infecting human cells, the company said, and then reduces inflammation and cytokine storm response that can result in organ damage and long-term negative impacts. BET inhibition is an epigenetic mechanism that can regulate disease-causing genes. Selective inhibition of apabetalone on BD2 produces a specific set of biological effects, the company added, with potentially important benefits for patients with high-risk cardiovascular disease, diabetes mellitus, chronic kidney disease, end-stage renal disease treated with hemodialysis, neurodegenerative disease, Fabry disease, peripheral artery disease and other orphan diseases.
New evidence from Seqirus Inc., of Summit, N.J., indicated its adjuvanted, trivalent influenza vaccine was more effective in reducing influenza-related medical encounters compared with standard egg-based quadrivalent influenza vaccine and high-dose trivalent influenza vaccine among adults 65 and older during the 2017/18 and 2018/19 U.S. influenza seasons. This Seqirus-sponsored and conducted retrospective cohort analysis compared relative vaccine effectiveness of an adjuvanted egg-based, trivalent influenza vaccine, egg-based quadrivalent influenza vaccines and high-dose trivalent influenza vaccine. Seqirus is part of CSL Ltd.
Sparingvision SAS, of Paris, said it entered a definitive agreement to acquire Gamut Therapeutics SA, also of Paris. Gamut uses a gene-independent approach to treat later stages of rod-cone dystrophies such as retinitis pigmentosa. The acquisition will be paid mostly in new Sparingvision shares and is expected to close in the second quarter of 2021. Financial terms were not disclosed. Gamut’s lead product, now called SPVN-20, is a mutation-agnostic gene therapy for restoring function of dormant cone cells in the retina.