Adicet Bio Inc., of Menlo Park, Calif., said its merger with Boston-based Restorbio Inc. has been completed, with the combined company operating under the name Adicet Bio Inc. Its shares will trade on Nasdaq under the ticker ACET. The company will focus on advancing a pipeline of off-the-shelf gamma delta T cells, engineered with chimeric antigen receptors and T-cell receptor-like antibodies to enhance selective tumor targeting, facilitate innate and adaptive antitumor immune response, and improve persistence for durable activity in patients.
Alimera Sciences Inc., of Atlanta, said Alimera Sciences Europe Ltd., its Ireland-based European subsidiary, has signed a distribution agreement with Slovenia-based Medis Pharmaceutical Co. d.o.o. (Medis) for the exclusive sales and distribution of Iluvien (fluocinolone acetonide intravitreal implant) in the Czech Republic and Austria. Iluvien is a sustained-release intravitreal injection approved in those countries for both the treatment of vision impairment associated with chronic diabetic macular edema considered insufficiently responsive to available therapies and prevention of relapse in recurrent non-infectious uveitis affecting the posterior segment.
Axovant Gene Therapies Ltd., of New York, said it signed a partnership with Viralgen, a contract development and manufacturing organization, to support all aspects of manufacturing for its AAV programs, including large-scale manufacturing, fill-finish and quality control in a GMP-certified environment custom-designed to bring therapies to market. Under the terms of the partnership, Axovant will have access to manufacturing resources for AXO-AAV-GM1 for GM1 gangliosidosis and AXO-AAV-GM2 for GM2 gangliosidosis with sufficient capacity to support ongoing development and eventual commercialization.
Bayer AG, of Leverkusen, Germany, entered a global license agreement with Systems Oncology LLC, of Scottsdale, Ariz., to develop and commercialize Erso, a compound in preclinical development for treating metastatic estrogen receptor-positive breast cancer. Systems Oncology will receive $25 million up front and is eligible to receive payments from Bayer on achieving certain development and commercialization milestones totaling $345 million as well as royalties on future global net sales.
Biontech SE, of Mainz, Germany, said it will receive a grant of up to €375 million (US$444.3 million) from the German Federal Ministry of Education and Research to support accelerated development of a SARS-CoV-2 vaccine. Biontech said it will use the milestone-based funding to support its mRNA vaccine program, BNT-162, which is being co-developed with New York-based Pfizer Inc. and Shanghai-based Fosun Pharma Co. Ltd. The project is designed to expand vaccine development and manufacturing capabilities in Germany as well as increase the number of participants in late-stage clinical trials.
Bostongene Corp., of Waltham, Mass., will collaborate with Brigham and Women’s Hospital, of Boston, to analyze whole transcriptome sequencing datasets generated from tumor and blood samples from patients with recurrent glioblastoma and who are treated with an oncolytic virus as part of a phase I trial. Bostongene correlation analysis aims to identify novel biomarkers of therapy response.
C3.ai, of Redwood City, Calif., said it is creating a judging panel to prioritize data science projects designed to help understand and mitigate the spread of COVID-19, improve response capabilities and help policymakers make decisions. Submitted projects may address applying machine learning and other AI methods to mitigate spread of the virus and address genome-specific COVID-19 protocols, including precision medicine of host responses. Registration deadline is Oct. 25 with a Nov. 18 submission deadline.
Cellinta Ltd., of London, said it launched by completing an undisclosed seed financing led by SV Health Investors and Cancer Research UK so it can target cancer stem cells with highly selective gene therapy. Cellinta was founded in 2019 and recently entered a collaborative research and option to license agreement with the University of Edinburgh. Soraya Bekkali is CEO and director of the board.
Combigene AB, of Lund, Sweden, and Cell and Gene Therapy Catapult, of London, said they will collaborate to develop a tailor-made package of analytical assays to ensure production of the AAV1-based gene therapy CG-01 meets regulatory requirements. CG-01 is designed to treat drug-resistant focal epilepsy. Catapult developed seven unique assays to characterize various viral vector components and their performance.
Corino Therapeutics Inc., of New York, reported data at the XVII International Symposium on Amyloidosis confirming that CRX-1008 is a potent stabilizer of TTR in both plasma and cerebrospinal fluid (CSF) using established ex vivo assays. CRX-1008 normalized TTR concentration in plasma with an overall mean increase in plasma TTR tetramer concentration of 55%. Significant concentrations of drug penetrated into the CSF decreasing monomeric TTR concentration by a mean of 48% as measured under semi-denaturing conditions. CRX-1008 is in development for hereditary leptomeningeal transthyretin amyloidosis.
Fresh preclinical data from Cue Biopharma Inc., of Cambridge, Mass., showed tumor penetration and antigen-specific T-cell engagement with its Immuno-STAT-based protein scaffolds. The positron emission tomography approach demonstrated the scaffolds’ ability to selectively engage tissue-resident T cells, including intratumoral T cells of defined specificity. The platform is designed to harness the body’s intrinsic immune system without the need for ex vivo manipulation.
Cullinan Oncology LLC, of Cambridge, Mass., and Tübingen, Germany, the German Cancer Research Center and the Eberhard Karls University of Tübingen, Faculty of Medicine at the University of Tübingen, Germany, have formed Cullinan Florentine, a company focused on developing a novel FLT3 x CD3 bispecific antibody for treating patients with acute myeloid leukemia (AML). The antibody was developed in Tübingen within the German Cancer Consortium, whose core is the German Cancer Research Center in Heidelberg, Germany. Cullinan Florentine has acquired an exclusive license to develop CLN-049 from the University of Tübingen and the consortium. FLT3 is a commercially validated target in AML, yet unlike small-molecule inhibitors targeting FLT3, a T-cell engaging antibody like CLN-049, which binds to the extracellular domain of FLT3, is agnostic to mutations in the intracellular signaling domain, opening up a broader patient population and avoiding resistance mechanisms, the company said.
Emergex Vaccines Holding Ltd., of Doylestown, Pa., said it determined a class I MHC expression library, or ligandome, for SARS-CoV-2-infected cells, in collaboration with George Mason University and the National Center for Biodefense and Infectious Diseases. During the collaboration, researchers at George Mason grew SARS-CoV-2, the virus that causes COVID-19, in human cells expressing ACE-2 representing six HLA supertypes. The MHC class I peptide expression library for cell surface expressed class I molecules and the precursors for internal class I bound peptides feeding the surface pool were determined using Emergex’s immunoproteomics 2-D liquid chromatography mass-spectrometry platform. The library of approximately 30,000 class I bound viral-derived peptides contains the first detailed empirical data for class I epitopes that are presented by a SARS-CoV-2 infected cell, the company said, defining the T-cell repertoire necessary for CD8+ cytotoxic T cells to perform their kill-and-clear function of an infected cell.
ERS Genomics Ltd., of Dublin, said it signed an agreement with Applied Stemcell Inc., of Milpitas, Calif., covering commercialization of CRISPR gene editing services and reagents. Financial terms were not disclosed.
Exelixis Inc., of Alameda, Calif., and Iconic Therapeutics Inc., of South San Francisco, reported preclinical data supporting continued development of ICON-2, an antibody-drug conjugate (ADC) comprising an anti-tissue factor (TF) antibody and a linker-payload from Zymeworks Inc., of Vancouver, British Columbia, targeting treatment of solid tumors. Key findings from the studies showed that ICON-2 binds to TF on human and nonhuman primate (NHP) cells with high affinity but does not affect coagulation, measured by FXa conversion and thrombin generation assays, and did not induce neutropenia in NHPs. In a mouse xenograft model of human pancreatic tumor cells, ICON-2 was shown to be more potent than an ADC containing monomethyl auristatin E (MMAE) conjugated to the same anti-TF antibody, and it was active in patient-derived xenograft models derived from multiple tumor types. In a NHP study, ICON-2 showed superior tolerability and exposure compared with an MMAE ADC using the same anti-TF antibody. The data are being presented in a poster at the World ADC Digital Conference.
Humanigen Inc., of Burlingame, Calif., said it formed a strategic collaboration with Lonza Ltd., of Basel, Switzerland, to expand the manufacturing capacity for lenzilumab, its GM-CSF ligand inhibitor in phase III trials to treat COVID-19, in advance of potential emergency use authorization in 2020 and commercialization. Production of lenzilumab will begin at Lonza's manufacturing facilities in Hayward, Calif., with technology transfer slated to begin in the third quarter of 2020.
Innovation Pharmaceuticals Inc., of Wakefield, Mass., said it received additional data from a U.S. regional biocontainment laboratory (RBL) indicating that, in human lung epithelial cell line experiments, brilacidin in combination with remdesivir (Veklury, Gilead Sciences Inc.) showed statistically significant and synergistic inhibition of SARS-CoV-2 vs. remdesivir alone. Overall viral load was reduced by 99.85% in one combination experiment, with remaining virus dropping to near undetectable levels. In other tests at the RBL, the dual PDE3/4 inhibitor was shown to inhibit SARS-CoV-2 in a human intestinal epithelial cell line and in primary fibroblast cells obtained from human donors. The company said the RBL is nearing completion of the asset’s in vitro testing against SARS-CoV-2 and plans to submit findings for peer-reviewed publication.
Kiadis Pharma NV, of Amsterdam, the Netherlands, said it received $9.5 million from the Advanced Regenerative Manufacturing Institute’s (ARMI) BiofabUSA program, in partnership with the U.S. Department of Defense, to fund its natural killer cell K-NK-ID101 program. The funding will support research on the asset’s activity and mechanism of action to treat COVID-19, a company-sponsored phase I/IIa trial to evaluate K-NK-ID101 in COVID-19 and GMP manufacturing scale-up. Additionally, Kiadis will collaborate with ARMI and BiofabUSA to establish large-scale manufacturing capacity for K-NK-ID101 in the U.S. to support potential industrialization of the therapy.
Mabion SA, of Konstantynów Łódzki, Poland, said it inked a memorandum of understanding with Vaxine Pty Ltd., of South Australia, outlining agreements to collaborate on the development, production and commercialization in the EU of Covax-19, Vaxine's phase I vaccine candidate against SARS-CoV-2.
Melior Pharmaceuticals Inc., of Exton, Pa., said it was awarded a grant from the Commonwealth of Pennsylvania’s COVID-19 Vaccines, Treatments and Therapies Program to support its phase II study of tolimidone. The study plans to recruit 428 individuals recently diagnosed and showing early symptoms of COVID-19 but not hospitalized. Tolimidone, an insulin sensitizer and lyn tyrosine kinase stimulator, will be assessed for its effectiveness in mitigating the number of participants who develop respiratory distress and the severity of those cases.
Mindset Pharma Inc., of Vancouver, British Columbia, said it completed its merger with North Sur Resources Inc. through a share exchange agreement to create Mindset Pharma Inc., with the former Mindset shareholders owning approximately 62.3% of the issued and outstanding common shares in the combined company. Mindset intends to pursue development of pharmacologically optimized psychedelic medicines.
Miragen Therapeutics Inc., of Boulder, Colo., said it restructured its leadership as it conducts a review of its R&D pipeline. The company plans to prioritize development of lead candidate MRG-229, a collagen gene inhibitor targeting idiopathic pulmonary fibrosis, which is set to move into IND-enabling activities. Miragen also plans to conclude its phase II Solar trial of legacy cancer compound cobomarsen, an miR-155 inhibitor, in individuals with cutaneous T-cell lymphoma and to report by year-end 2020 top-line data collected to date from 37 participants. Miragen reported in 2019 that delays in the onboarding process for Solar would delay data until 2021. The company also said it is conducting a comprehensive review of strategic alternatives and engaged Ladenburg Thalmann & Co. Inc. as financial advisor to assist in the process.
Momenta Pharmaceuticals Inc., of Cambridge, Mass., said the waiting period expired under the Hart-Scott-Rodino Antitrust Act in connection with the $6.5 billion all-cash tender offer for its acquisition by a wholly owned subsidiary of Johnson & Johnson, of New Brunswick, N.J., enabling the transaction to proceed. The deal is expected to close in early October 2020.
Mylan NV, of Hertfordshire, U.K., and Pfizer Inc., of New York, said they received final approval from the European Commission (EC) for their proposed combination of Mylan with Pfizer’s Upjohn business after the EC approved buyers of Mylan’s proposed divestiture of certain products in Europe – a condition of the transaction – and Mylan inked agreements with those buyers on terms the EC accepted. Mylan and Pfizer said they also received approval from the Australian Competition and Consumer Commission and the New Zealand Commerce Commission related to the business combination. In separate news, Mylan said the Technical Board of Appeal of the European Patent Office held that Yeda Research and Development Co. Ltd.’s European Patent No. 2 949 335, related to the Copaxone (glatiramer acetate) 40 mg/mL three times weekly product formulation by Teva Pharmaceutical Industries Inc., of Tel Aviv, Israel, was invalid and revoked across Europe. Mylan said the ruling will allow it to return its therapeutically equivalent version of Copaxone to the EU market to treat relapsing forms of multiple sclerosis.
Novavax Inc., of Gaithersburg, Md., said it amended an agreement with Serum Institute of India Private Ltd., (SIIPL), of Maharashtra, allowing SIIPL also to manufacture the antigen component of NVX‑CoV2373, the COVID‑19 vaccine candidate from Novavax. The agreement enables Novavax to increase manufacturing capacity of the COVID-19 Spike glycoprotein modulator to more than 2 billion doses annually once planned capacity is brought on-line by mid-2021. The antigen component of NVX-CoV2373 is being manufactured at Novavax CZ in Bohumil, Czech Republic, along with partnered manufacturing sites while Novavax’ Matrix-M adjuvant is being manufactured at Novavax AB in Uppsala, Sweden, and partnered manufacturing sites.
Novus Therapeutics Inc., of Irvine, Calif., said it completed the acquisition of privately held Anelixis Therapeutics Inc., of Boston, and, concurrently, inked a definitive agreement to sell nonvoting convertible preferred stock through a private placement to institutional accredited investors led by BVF Partners LP, with participation from Cormorant Asset Management, Ecor1 Capital, Logos Capital, Fidelity Management and Research Co., Adage Capital Partners LP, Woodline Partners LP, Ridgeback Capital, Janus Henderson Investors, Samsara Biocapital and additional unnamed investors. The private placement, in which investors were to be issued preferred shares priced at approximately $500 apiece, was expected to result in gross proceeds of approximately $108 million. The funds will be used to advance phase II trials of AT-1501, a humanized IgG1 anti-CD40L antibody, in renal transplantation, islet cell transplantation, autoimmune nephritis and amyotrophic lateral sclerosis. Ladenburg Thalmann & Co. Inc. served as exclusive financial advisor to Novus and SVB Leerink served as financial advisor and lead placement agent for the private placement, with Noble Life Science Partners, a division of Noble Capital Markets Inc., as co-placement agent. On Sept. 15, Novus shares (NASDAQ:NVUS) nearly tripled in value, gaining 73 cents to close at $1.11.
Obsidian Therapeutics Inc., of Cambridge, Mass., said Bristol Myers Squibb Co. (BMS), of New York, exercised its option to an exclusive global license to a cell therapy candidate based on Obsidian's Cytodrive technology for controlled expression of the immunomodulatory factor CD40L. The opt-in was related to a January 2019 cell therapy pact with Celgene Corp. weeks after its takeover bid by BMS. Under terms of the agreement, Obsidian is eligible for potential future milestone and royalty payments.
Privately held Oligomerix Inc., of New York, said it was awarded a $3.19 million Small Business Innovation Research/Small Business Technology Transfer Commercialization Readiness Pilot Program grant from the National Institute on Aging (NIA) at the U.S. NIH to advance its small-molecule tau self-association inhibitor, which targets the beginning of the tau aggregation cascade associated with Alzheimer’s disease.
Pharmajet Inc., of Golden, Colo., said its Stratis Needle-free Injection System will be used to deliver a DNA vaccine against COVID-19 (GX-19), which is being developed by an international consortium led by Genexine Co. Ltd., of Seoul, South Korea, and including the International Vaccine Institute, Binex, Gennbio, Korea Advanced Institute for Science and Technology and Pohang University of Science and Technology. The GX-19 vaccine was approved by South Korean regulators to start a phase I/IIa trial in June.
Protokinetix Inc., of Marietta, Ohio, said in vivo testing demonstrated transplantation of photoreceptor precursor cells (PPCs) pretreated with AAGP (PKX-001), a cell adhesion molecule inhibitor, resulted in statistically significant improvements in both the visual behavioral (optokinetic tracking test) and functional analysis (electroretinogram test) responses as compared with PPCs without pre-treatment. Imaging data revealed that pre-treatment of PPCs with AAGP also led to a substantial enhancement of cell survival as determined at three, 4.5 and six months after cell transplantation. At the six-month timepoint, the AAGP-treated cells acquired the ability to express retinal and synaptic proteins, confirming that AAGP has no adverse effect on precursor cells’ maturation. AAGP is in development for age-related macular degeneration.
Soligenix Inc., of Princeton, N.J., reported nonclinical data published in the Journal of Pharmaceuticals Sciences characterizing filovirus protein antigens (including for Ebola and Marburg viruses) and their thermostabilization. Work conducted by the company and outside researchers has demonstrated the feasibility of developing heat stable subunit protein vaccine formulations for filovirus vaccines. Protective efficacy has been demonstrated in nonhuman primates against infection with Ebola virus, Sudan virus and Marburg virus. Formulation conditions have been identified to enable heat stabilization of each antigen, alone or in combination, for at least 12 weeks at 40 degrees Celsius (104 degrees Fahrenheit). The most recent results demonstrate the thermostabilization of three virus glycoproteins (from Zaire ebolavirus, Sudan ebolavirus and Marburg marburgvirus), and the identification of key stability-indicating assays to further support mono-, bi- and trivalent vaccine formulations.
Takeda Pharmaceutical Co. Ltd., of Osaka, Japan, said it expanded its cell therapy manufacturing capabilities with the opening of a new 24,000-square-foot R&D cell therapy manufacturing facility at its R&D headquarters in Boston.
Tonix Pharmaceuticals Holding Corp., of New York, said the first patient was enrolled in the observational COV-LOGIC study (TNX-C001), a study of humoral (antibody) and cellular (T cell) immune responses to SARS-CoV-2 in volunteers who have recovered or remain asymptomatic after exposure to COVID-19. The research is part of an ongoing and broader collaboration between Tonix and Southern Research to develop and conduct animal testing of Tonix’s TNX-1800, which is a live replicating, attenuated virus vaccine designed to protect against COVID-19.