Arrakis Therapeutics Inc., of Waltham, Mass., said data demonstrating the capabilities of its Pearl-seq technology for the systematic identification of binding sites in RNA molecules, as an advanced research method to facilitate the discovery of RNA-targeted small-molecule (rSM) medicines, were published online in ACS Chemical Biology. The study demonstrates the ability of the technology platform to identify ligand binding sites within a model aptamer RNA as well as to identify potential off-target binding interactions. These data, combined with conventional RNA structure probing methods, can be used to generate and refine two- and three-dimensional structures of RNA-small molecule complexes, thereby facilitating the rational design of rSM therapeutics, the company said.
Biocryst Pharmaceuticals Inc., of Research Triangle Park, N.C., said the National Institute of Allergy and Infectious Diseases (NIAID), part of the U.S. NIH, has awarded it a new $44 million contract, and has added approximately $3 million to an existing contract, to support the development and completion of parts 1 and 2 of an ongoing clinical trial of galidesivir in Brazil; to conduct a phase II trial of galidesivir in non-hospitalized COVID-19 patients at high risk for developing severe disease and complications of COVID-19; to conduct a clinical pharmacology trial of galidesivir to determine appropriate dosing in patients with renal impairment; and to increase the supply of galidesivir, an investigational broad-spectrum antiviral drug that was safe and well-tolerated in previous phase I trials in healthy subjects.
Clinuvel Group, of Melbourne, Australia, said it opened an R&D center to be operated by subsidiary Vallaurix Pte Ltd. in Singapore. The labs and offices will expand the company’s capacities in molecular profiling, peptide chemistry, and polymer and formulation sciences, as well as the capability to conduct ex vivo experiments and bioassays, and studies on fresh biological and tissue cultures. Vallaurix is developing drugs from the family of melanocortins.
The Commonwealth of Pennsylvania announced grants totaling $10 million to 23 entities to develop effective COVID-19 vaccines, treatments and therapies. The award to Thomas Jefferson University, which received the required matching funds from partner Bharat Biotech International Ltd., of Hyderabad, India, and The Philadelphia Foundation, was granted for the development of Coravax, a Jefferson-developed COVID-19 vaccine. The grant funding will accelerate Coravax formulation optimization, a final step needed for development and implementation of the vaccine for human trials. Bharat Biotech is currently manufacturing the vaccine for a human phase I safety and immunogenicity study, expected to begin within the next several months.
Elektrofi Inc., of Boston, and Ballerup, Denmark’s Leo Pharma A/S agreed to develop subcutaneously administered antibodies. Elektrofi will license its technology to Leo and co-develop formulations of investigational Leo antibodies. The license is exclusive for use against a specific target in dermatology indications. Elektrofi will receive up-front payments for access to its technology. Milestone payments pend achieving prespecified clinical, regulatory and commercial milestones. Other terms were not disclosed. Also, Leo said it divested a portfolio of four non-core medicines to Cheplapharm GmbH, of Greifswald, Germany, for €300 million ($US357 million). One-Alpha, Locoid, Pimafucin and Zineryt are the divested products, which are included within bone disorders/nephrology, dermatology and gynecology areas.
New preclinical data from Glycomimetics Inc., of Rockville, Md., suggest an E-selectin-targeting strategy with uproleselan may help patients with acute myeloid leukemia (AML) overcome resistance to venetoclax and hypomethylating agent-based therapy. An analysis of combination therapy demonstrated statistically significant prolongation of survival in a patient-derived xenograft model. Uproleselan, in a phase III development program in AML, received breakthrough therapy designation for treating adult AML patients with relapsed or refractory disease. Uproleselan is designed to block E-selectin (an adhesion molecule on cells in the bone marrow) from binding with blood cancer cells as a targeted approach to disrupting mechanisms of leukemic cell resistance within the bone marrow microenvironment.
Immutep Ltd., of Sydney, said the Australian Research Council awarded the company and Monash University a AU$671,427 (US$496,057) grant to support research into lymphocyte activation gene-3 (LAG-3) for another three years. The collaboration, which began in 2017, is for investigating the structure of LAG-3 and how it binds to its main ligand, MHC class II. The new funding will allow further investigation into the way LAG-3 controls T-cell function for treating cancer, autoimmune diseases or infectious diseases.
Kalos Therapeutics Inc., of Phoenix, said its peptides inhibited up to 42% of CoV-2 spike peptides from its binding domain in the ACE receptor, demonstrating a peptide-peptide interaction producing inhibition. A similar approach would also be useful in immunocompromised patients where the addition of the Kalos drug to an existing I.V. would provide, touchless, non-immunostimulant therapy for patients exposed to or diagnosed with SARS CoV-2 virus, the company said. Kalos said the combination approach couldn’t be used in treating COVID-19 with drugs like remdesivir and that it may work best as a monotherapy in immunocompromised patients. All Kalos peptides used in this project were eight amino acids in length, unmodified and contained all-natural amino acids.
Ligand Pharmaceuticals Inc., of San Diego, said its wholly owned subsidiary, Pelican Acquisition Sub Inc., is commencing a tender offer to buy all outstanding shares of San Diego-based Pfenex Inc.’s common stock at $12 each in cash, plus one nontransferable contractual contingent value right per share representing the right to receive a contingent payment of $2 in cash if a certain specified milestone is achieved. The transaction is expected to close in the fourth quarter of 2020.
Lineage Cell Therapeutics Inc., of Carlsbad, Calif., said it received $24.6 million in cash from Juvenescence Ltd., a British Virgin Islands-based holding company, representing principal and interest due under a convertible promissory note issued in August 2018 as partial payment for the sale by Lineage to Juvenescence of 14.4 million shares of common stock of Agex Therapeutics Inc., of Alameda, Calif.
Maat Pharma SA, of Lyon, France, reported data from the compassionate use program of its lead microbiome restoration biotherapeutic, MaaT-013, an allogeneic fecal microbiota, during the European Society for Blood and Marrow Transplantation annual meeting. In 11 people who developed gastrointestinal acute graft-vs.-host-disease (aGVHD), the overall response rate was 82%, including five complete responses, two very good partial responses and two partial responses, all at 28 days after initial dosing. Maat’s ongoing phase II HERACLES study in gastrointestinal predominant, steroid-refractory aGVHD is expected to read out in the first quarter of 2021.
Mindset Pharma Inc., of Toronto, said it conducted pharmacological tests on its synthesized compounds, including an assay designed to determine functional activity at the human 5HT-2A receptor and other serotonin subtypes, and reported that those synthesized compounds are acting just as strongly on the 5HT-2A receptor as psilocin, with clear structure-affinity relationship and with certain compounds demonstrating a several-fold increase in efficacy and potency compared to psilocin.
Novavax Inc., of Gaithersburg, Md., said it reached an agreement in principle with the Canadian government to supply up to 76 million doses of NVX-CoV2373, its COVID-19 vaccine. Novavax and Canada expect to finalize an advance purchase agreement under which Novavax will supply doses of NVX-CoV2373 to Canada beginning as early as the second quarter of 2021. The purchase arrangement will be subject to licensure of the Novavax vaccine by Health Canada. NVX-CoV2373 is currently in multiple phase II trials.
PDL Biopharma Inc., of Incline Village, Nev., said it signed and closed a definitive agreement for the sale of three royalty interests for Kybella (deoxycholic acid), Zalviso (drug-device combo delivering sufentanil) and Coflex (interlaminar stabilization device) to SWK Funding LLC, a wholly owned subsidiary of SWK Holdings Corp., in exchange for $4.35 million in cash.
Qualigen Therapeutics Inc., of Carlsbad, Calif., said it signed a contract with Irisys LLC for the production of AS-1411, its lead candidate for the treatment of viral diseases, including COVID-19.
Regulus Therapeutics Inc., of La Jolla, Calif., said, pursuant to an amendment of its term loan agreement with Oxford LLC, the company is eligible for up to an additional seven months of interest-only payments in the event the company pays down $10 million in loan principal before April 30, 2021, utilizing proceeds from the sale of materials to, and potential milestones received from, Paris-based Sanofi SA. In the event the principal paydown event does not occur by April 30, 2021, Regulus will make principal and accrued interest payments, in arrears, commencing May 1, 2021, in accordance with the previously amended terms. If the principal paydown event occurs after April 30, 2021, but on or before July 31, 2021, then Regulus will recommence an extended interest-only payment period through Dec. 31, 2021. In the event the company receives the additional interest only period, principal and accrued interest payments will recommence on Jan. 1, 2022. The company also entered an amendment with Sanofi concerning the receipt of potential milestones for development of its miR-21 programs. Regulus also sold additional compound-related materials to Sanofi in exchange for $1 million. Under the terms of the amendment, and in lieu of the previous $10 million enrollment milestone, Regulus is eligible to receive an additional $4 million upon the completion of transfer and verification of the materials sold to Sanofi and an additional $5 million milestone upon achievement of the enrollment milestone. In the event the enrollment milestone occurs first, Regulus will receive the entire $9 million for both milestones. In addition, Regulus is eligible to receive $25 million upon the achievement of an additional development milestone related to Sanofi's development of miR-21 compounds.
Resverlogix Corp., of Calgary, Alberta, said it continues to rely on blanket exemptions issued by provincial securities commissions due to the COVID-19 outbreak that permit the company to extend the filing deadline of its annual financial statements for the year ended April 30, 2020, and related management’s discussion and analysis for a period of up to 45 days. Resverlogix continues to expect that it will file the annual filings on or before Sept. 11, 2020.
Seneca Therapeutics Inc., of Philadelphia, said it licensed TEM 8 intellectual property from Memorial Sloan Kettering Cancer Center (MSK). TEM 8 may enable pre-screening of solid tumors to determine if SVV-001 might be effective in that patient. Researchers demonstrated that the level of expression of TEM 8 and genes in the innate immune system could predict with a high degree of certainty what type of cancer cells would support SVV replication in and killing of the tumor cells. That work, originally published in 2017, will be utilized in upcoming SVV-001 clinical trials to pre-select patients that might best respond to SVV-mediated cancer therapy.
Sotio AS, of Prague, Czech Republic, an immuno-oncology company owned by PPF Group, said it acquired rights to Cambridge, Mass.-based Unum Therapeutics Inc.’s BOXR cell therapy platform and BOXR lead programs to develop T-cell therapies to treat solid tumors. BOXR, or Bolt-on Chimeric Receptor, technology is designed to incorporate novel transgenes to enhance T-cell function in the solid tumor microenvironment. The lead program, BOXR-1030 for the treatment of hepatocellular carcinoma and other solid tumors, is expected to initiate first clinical studies in 2021. Under the terms of the agreement, Sotio will make an up-front payment of $8.1 million for the BOXR technology and success-dependent future milestone payments up to $3.4 million.
Vor Biopharma Inc., of Cambridge, Mass., said it will evaluate the potential use of gene editing technology from Metagenomi Technologies LLC, of Emeryville, Calif., to develop engineered hematopoietic stem cell-based therapies for the treatment of blood cancers, such as acute myeloid leukemia. The collaboration is nonexclusive and applies to preclinical research only. Further terms of the agreement are not being disclosed.