Actinium Pharmaceuticals Inc., of New York, said it has been awarded a Small Business Technology Transfer grant from the U.S. NIH to support a clinical collaboration with Memorial Sloan Kettering Cancer Center (MSK), which will study Iomab-ACT, the company's CD45-targeting antibody radio-conjugate, for targeted conditioning to achieve lymphodepletion prior to administration of a CD19-targeted CAR T-cell therapy developed at MSK. The CD19 CAR T has been previously studied by MSK in a phase II trial with chemotherapy conditioning in patients with relapsed or refractory B-cell acute lymphoblastic leukemia or diffuse large B-cell lymphoma. MSK will lead the study to utilize targeted radiopharmaceutical ARC-based lymphodepletion to replace chemotherapy-based conditioning prior to CAR T-cell therapy. It will assess the feasibility of using Iomab-ACT targeted lymphodepletion prior to MSK's 19-28z CAR T and assess safety and efficacy outcomes relative to results with MSK's CAR T 19-28z in patients who had received chemotherapy-based lymphodepletion prior to CAR T administration.
Biomx Inc., of Ness Ziona, Israel, presented preclinical data from its phage panel for the treatment of inflammatory bowel disease at IDWeek 2020. The presentation detailed a phage therapy panel, comprising phage directed against Klebsiella pneumoniae (KP), showing a broad target host range and the potential to address carbapenem-resistant and extended spectrum beta-lactamase-producing bacteria. Those results demonstrate the feasibility of identifying phage with potent in vitro activity against antibiotic resistant KP isolates. In vivo studies, testing phage efficacy in animal models of infections, are currently ongoing.
Bridgebio Pharma Inc., of Palo Alto, Calif., and the University of Colorado Anschutz Medical Campus will collaborate to research therapies for genetically driven diseases. Bridgebio will support early discovery research already underway in the school’s labs. Bridgebio, in addition to gene therapy, is developing treatments for cancer.
Cannabics Pharmaceuticals Inc., of Tel Aviv, Israel, and Bethesda, Md., said it signed an agreement with a clinical quality and validation processes consultancy to oversee preparation and submission of a pre-IND meeting request to the FDA on cannabinoid-based antitumor candidate RCC-33 for treating colorectal cancer. The request is expected to be submitted in the first quarter of 2021. The company said it plans to conduct a proof-of-concept study using in vivo animal models.
Cidara Therapeutics Inc., of San Diego, reported preclinical data from its Cloudbreak antiviral program at 2020 IDWeek, supporting development of candidate CD-377 for universal influenza protection for all people, regardless of immune status. Results showed a single dose of CD-377 was protective against lethal challenge with a panel of seasonal and pandemic influenza subtypes in mice, demonstrating its broad-spectrum activity. The candidate also demonstrated superior dose-dependent viral load reduction in the lung compared to approved influenza treatment oseltamivir in lethal influenza mouse and ferret models. Additional data in mice showed CD-377 administered 28 days prior to infection completely protected against several strains of influenza, and treatment efficacy was observed with a single dose of CD377 72 hours-post infection.
Curelab Oncology Inc., of Boston, said it commenced operations in Saudi Arabia, Kuwait, the UAE, Qatar, Bahrain and Oman. Curelab's partners in the region will test the efficacy of the company's DNA-based products against cancers and non-cancerous diseases involving chronic inflammation. Curelab’s lead product, Elenagen, is a DNA encoding gene called p62/SQSTM1.
Curis Inc., of Lexington, Mass., said CA-4948, a small-molecule inhibitor of IRAK4, demonstrated potent IRAK4 inhibition with favorable selectivity over other kinases, cellular activity, pharmacokinetics, efficacy and safety. It also showed tumor regression in a diffuse large B-cell lymphoma xenograft model without any overt toxicities. CA-4948 is in a phase I dose-escalating trial in patients with non-Hodgkin lymphomas, including those with myeloid differentiation primary response 88 alterations. CA-4948 is also being investigated in a phase I trial for treating acute myeloid leukemia and myelodysplastic syndromes.
Effrx Pharmaceuticals SA, of Freienbach, Switzerland, said it entered an exclusive license agreement with Diurnal Group plc, of London, which is developing therapies for chronic endocrine diseases, for the registration and commercialization of Alkindi for pediatric adrenocortical insufficiency (AI) in Switzerland. Effrx has exclusive rights to register and commercialize Alkindi in Switzerland. Alkindi is an oral, immediate-release pediatric formulation of hydrocortisone granules in capsules for treating pediatric AI and congenital adrenal hyperplasia.
Epsilogen Ltd., of London, said it was awarded a £1 million (US$1.32 million) grant from Innovate UK. Epsilogen will collaborate with King's College London and use the grant to further develop the company's cancer immunotherapeutic, EPS-201, an IgE-based therapeutic targeting HER2-positive solid tumors.
GT Biopharma Inc., of Beverly Hills, Calif., said a report in Cancers showed that a B7-H3-targeted trispecific natural killer (NK) engager, or Trike, studied in vitro and in vivo, suggested the potential to enhance NK cell immunotherapy in solid tumor settings, supporting further development of the company’s technology. The research was conducted at the University of Minnesota and at Massachusetts General Hospital/Harvard Medical School.
Hemoshear Therapeutics LLC, of Charlottesville, Va., said it entered a research collaboration and service agreement with Takeda Pharmaceutical Co. Ltd., of Osaka, Japan. Under the terms, Hemoshear will receive an up-front payment and funding to develop a new human tissue-based model of a rare liver disease using the company's Reveal-Tx Platform, which combines physiological and computational models of disease to identify novel treatment approaches and select drug candidates in a human-relevant disease context. Hemoshear has an ongoing three-year collaboration with Takeda to discover and develop therapeutics for liver diseases, including nonalcoholic steatohepatitis.
Intelgenx Corp., of Saint Laurent, Quebec, inked an amended and restated license agreement granting Tetra Bio-Pharma Inc., of Ottawa, Ontario, additional exclusive global rights, including manufacturing rights, to its Adversa mucoadhesive delivery technology. Tetra plans to use the technology to develop a THC-based mucoadhesive film containing its PPP-002 (dronabinol) candidate to treat chemotherapy-induced nausea and vomiting. Tetra agreed to make three milestone payments, with 45% to be paid on Nov. 15, 2020, 45% on March 1, 2021, and a final payment of 10% upon successful technology transfer. Amounts were not disclosed. In addition, Intelgenx is eligible for a low-single-digit royalty on net sales.
Kintara Therapeutics Inc., of San Diego, said it executed an agreement with the Global Coalition for Adaptive Research (GCAR) to include its cancer therapy candidate, VAL-083 (dianhydrogalactitol), in GCAR's Glioblastoma Adaptive Global Innovative Learning Environment (GBM AGILE) study. The global effort is enrolling people with newly diagnosed and recurrent GBM using an FDA-approved master protocol to test multiple drugs simultaneously and over time against a common control arm using an adaptive design with a stage 1 (phase II) learning and adapting phase and a stage 2 (phase III) expansion and confirmation phase. Kintara intends to use results from the VAL-083 arm to file for FDA approval. Under the terms of their agreement, Kintara will supply GCAR with VAL-083 and provide funding to support the VAL-083 arm of the registrational study. GCAR will manage operational aspects of the study, including site activation and patient enrollment.
Moleculin Biotech Inc., of Houston, said results from an independent laboratory validated internal animal studies showing the ability of its anthracycline derivative, annamycin, to target lung localized tumors. Additionally, the experiments compared free annamycin (F-anna) and liposomal annamycin (L-anna) with standard-of-care doxorubicin (dox). Preliminary results showed that F-anna accumulates in the lungs of rats at a nine-fold higher concentration than dox, suggesting that differences in the molecular structures of annamycin and dox are responsible for annamycin's comparatively high accumulation in lungs and potential to target tumor cells there. The L-anna formulation further increased accumulation in the lungs, with a concentration more than 30-fold higher than dox.
Mybiotics Pharma Ltd., of Rehovot, Israel, said it was awarded €2 million (US$2.3 million) by the EU as part of Horizon 2020 to support development of a technology enabling efficient delivery of personalized probiotics to prevent gut-related diseases and recover damaged gut microbiota.
Neoimmunetech Inc., of Rockville, Md., said it agreed to a second trial collaboration with F. Hoffmann-La Roche, a unit of Roche Holding AG, of Basel, Switzerland, to evaluate the combination of NT-I7 (efineptakin alfa), its long-acting IL-7 agonist, and the PD-L1 inhibitor Tecentriq (atezolizumab, Roche). The master agreement will focus initially on a phase II trial of the combination in previously untreated, PD-L1-expressing, locally advanced or metastatic non-small-cell lung cancer.
Neumentum Inc., of Morristown, N.J., said it agreed to an exclusive development and commercialization license for NTM-001 in China with Nuance Biotech Co. Ltd., of Shanghai. The alcohol-free formulation of ketorolac in a pre-mixed bag for continuous 24-hour intravenous infusion is designed to treat moderately severe acute pain requiring analgesia at the opioid level, usually in a postoperative setting. Neumentum expects to receive an up-front payment of $3 million and is eligible for development, regulatory and sales milestone payments of up to $50 million as well as low-double-digit percentage royalty payments on net sales. Neumentum also granted Nuance a nonexclusive option to develop and commercialize NTM-001 in certain Asian Pacific countries and territories outside China.
Noveome Biotherapeutics Inc., of Pittsburgh, said preclinical results regarding its amnion-derived multipotent progenitor (AMP) cells, published in Neurotherapeutics, showed neuroprotective properties when delivered systemically in mice with experimental autoimmune encephalomyelitis-induced experimental optic neuritis and myelitis. The AMP cells were administered in a single dose of 2 x 106 cells/200µl vehicle on day nine post-immunization or in three doses of 1 x 106 cells/200µl vehicle on days nine, 12 and 15 post-immunization. Controls received 200 µL vehicle on the same schedule. Intravenous and intraperitoneal administration of AMP cells reduced ascending paralysis and attenuated visual dysfunction in the mice. Histological analysis revealed statistically significant decrease in spinal cord demyelination and a trend toward decreased inflammation scores. In the optic nerves, AMP cell treatments showed statistically significant decrease in inflammation and reduced demyelination. The studies were conducted at the University of Pennsylvania’s Perelman School of Medicine.
PCI Biotech Holding ASA, of Oslo, Norway, said Astrazeneca plc, of Cambridge, U.K., declined to establish a definitive agreement for PCI’s Fimanac technology following a preclinical collaboration between the companies initiated in 2015 to assess the technology’s potential to enhance delivery of nucleic acid compounds. PCI said Astrazeneca’s decision was based primarily on a strategic evaluation of its development pipeline. The companies will seek to publish the preclinical findings. On Oct. 21, PCI’s shares, trading as PCIB on the Oslo Stock Exchange, fell 51.5%, losing NOK23.80 (US$2.59) to close at NOK22.45 (US$2.44).
Poseida Therapeutics Inc., of San Diego, and Tscan Therapeutics Inc., of Waltham, Mass., said they formed a research collaboration and license agreement to explore development of T-cell receptor (TCR)-T-cell therapies to treat COVID-19. The alliance will combine Poseida’s allogeneic T-cell platform and Tscan's immunodominant epitopes and TCR sequences for the development and commercialization of allogeneic TCR-T-cell therapies. The agreement gives Poseida access to Tscan's data and intellectual property related to TCR sequences and targets of potential value to treat COVID-19. Tscan will provide TCR expertise and additional guidance. Financial terms were not disclosed.
Proteintech Group Inc., of Chicago, said it acquired Chromotek GmbH, of Munich, which manufactures camelid, single-domain antibodies, or nanobodies, to extend its manufacturing platform in antibodies, proteins and immunoassays. Terms were not disclosed.
Prothena Corp. plc, of Dublin, said partner Roche Holding AG, of Basel, Switzerland, agreed to advance the synuclein alpha inhibitor prasinezumab into a phase IIb study in early Parkinson’s disease (PD) following efficacy signals consistent with disease modification seen in the phase II PASADENA study, which reported data in September. The study will extend the population enrolled in PASADENA to include people with early PD on stable levodopa therapy. Prothena expects to earn a $60 million milestone payment when the first participant is dosed. The companies plan to disclose details of the study design in the first half of 2021.
PTC Therapeutics Inc., of South Plainfield, N.J., said Chugai Pharmaceutical Co. Ltd., a member of the Roche Group, of Basel, Switzerland, filed an NDA for Evrysdi (risdiplam) for spinal muscular atrophy in Japan, triggering a $7.5 million milestone payment to PTC from Roche.
Scynexis Inc., of Jersey City, N.J., disclosed preclinical data on its broad-spectrum antifungal, ibrexafungerp, from posters scheduled for presentation at IDWeek 2020. In a set of four independent global in vitro studies testing the activity of ibrexafungerp against 445 Candida auris isolates, the ibrexafungerp MIC90 value against the isolates was 1 mg/mL; the modal and MIC50 values were 0.5 mg/mL each. Of the 445 isolates, 32 of the C. auris strains had elevated MICs to echinocandins and only one of the 32 had elevated MICs to ibrexafungerp. An additional preclinical study showed that a 30-mg/kg twice-daily dose of ibrexafungerp prevented pneumocystis pneumonia in a murine model.
Seqirus U.S. Inc., of Summit, N.J., a unit of CSL Ltd., of Melbourne, Australia, reported real-world evidence indicating that an MF59-adjuvanted trivalent seasonal influenza vaccine (aTIV) was more effective than standard, non-adjuvanted quadrivalent influenza vaccines (IIV4) to reduce influenza-related medical encounters in adults 65 and older with at least one underlying health condition during the U.S. 2017/18 and 2018/19 influenza seasons. aTIV also was found to be more effective than a high-dose trivalent influenza vaccine (TIV-HD) in seven of 11 outcomes assessed, including influenza-related medical office visits and cardio-respiratory disease-related hospitalizations or emergency room visits, in adults 65 and older during the 2018/19 U.S. influenza season. The company plans to present detailed findings at IDWeek 2020.
Teneobio Inc., of Newark, Calif., said it agreed to a research collaboration and commercial license with Arsenal Biosciences Inc. (Arsenalbio), of South San Francisco, covering the use of Teneobio’s Unidabs single-domain, human heavy chain only antibodies in CAR T-cell therapy. Arsenalbio gained commercial rights to Unidabs against therapy-guiding antigens for immune cell therapy programs in exchange for an up-front payment, potential clinical and regulatory milestone payments and royalties on global sales to Teneobio.
Union Therapeutics A/S, of Hellerup, Denmark, said it added UNI-91103 (intranasal niclosamide) for preventing COVID-19 to its pipeline. The nasal spray will be used in treatment trials combined with Union's inhalation product, UNI-91104, which continues development for treating COVID-19. UNI-91103 has been tested in 44 healthy volunteers without significant safety concerns and is ready for tests in COVID-19 patients as well as subjects at risk of contracting COVID-19, the company said.
Vir Biotechnology Inc., of San Francisco, reported preclinical data at IDWeek 2020 showing VIR-2482, its intramuscularly administered influenza A-neutralizing monoclonal antibody, has broad binding and neutralizing potential against all major strains of influenza A, including pandemic strains, from the last 100 years. Additionally, VIR-2482 administered prophylactically 24 hours prior to lethal doses of influenza significantly reduced morbidity and prevented mortality in mouse models.