Advanced Accelerator Applications SA, of Saint-Genis-Pouilly, France, said it will build a 50,000-square-foot targeted radioligand therapy manufacturing plant in Indianapolis. The cancer therapy combines a precision targeting compound with a radioactive particle that binds to markers expressed by tumors, inhibiting tumor growth and replication. Advanced accelerator is part of Basel, Switzerland-based Novartis AG.
Audentes Therapeutics Inc., of San Francisco, an Astellas company, disclosed in a June 23 statement posted to the Joshua Frase Foundation website that a second patient in the phase I/II Aspiro trial testing gene therapy AT-132 in X-linked myotubular myopathy died due to sepsis, according to preliminary findings. As was the case of the first patient death, reported earlier, the patient was one of three older patients who received AT-132 at the dose of 3x1014 vg/kg in whom investigators recently observed new serious adverse events of hepatobiliary disease. Although the investigations around both deaths are ongoing, preliminary reports indicate that the clinical course was similar in the two patients who died, the company said. The study has been on FDA clinical hold. A BLA submission is no longer anticipated for mid-2020. Earlier this year, Astellas Pharma Inc., of Tokyo, bought Audentes in a deal worth about $3 billion.
Basilea Pharmaceutica Ltd., of Basel, Switzerland, said it will move its corporate headquarters, including office space and laboratories, to Allschwil, Switzerland, in 2022. Basilea develops therapies for oncology and infectious diseases.
Bioasis Technologies Group, of Guilford, Conn., and Chiesi Global Rare Diseases, of Boston, part of the Chiesi Group, entered a worldwide, exclusive licensing agreement for the xB3 BBB platform technology in treating rare diseases with a focus on four undisclosed lysosomal storage disorders. Chiesi will pay Bioasis an up-front fee of $3 million. Bioasis could receive additional development, regulatory and commercial milestone payments of up to $138 million, as well as additional royalties on net sales of licensed products, pending market approval. Chiesi will be responsible for all costs associated with research, development and commercialization of the four undisclosed programs.
Burning Rock Biotech Ltd., of Guangzhou, China, and Cstone Pharmaceuticals Co. Ltd., of Suzhou, China, will collaborate to develop companion diagnostics for pralsetinib in China. Pralsetinib is an investigational treatment developed by Cstone’s partner, Blueprint Medicines Corp., of Cambridge, Mass. The companion diagnostics will be used to detect RET-altered non-small-cell lung cancer, thyroid cancer and other solid tumors. No financial terms were disclosed.
Camino Pharma LLC, of San Diego, said it received a $920,000 Small Business Innovation Research grant from the National Institute of Mental Health to fund a collaboration with the University of California San Diego and Sanford Burnham Prebys Medical Discovery Institute to validate negative allosteric modulators of metabotropic glutamate receptor 2 and 3 for treating major depressive disorder and treatment-resistant depression.
CNS Pharmaceuticals Inc., of Houston, said it engaged Berry Consultants, a clinical statistical consulting group, for advice ahead of the phase II trial of its lead drug candidate, berubicin, for treating glioblastoma multiforme. Berry Consultants specializes in the Bayesian approach to medical statistics. The study is expected to begin by year-end.
New data from Genespire Srl, of Milan, Italy, and the San Raffaele Telethon Institute for Gene Therapy show increased homology-directed recombination efficiency in hematopoietic stem cells (HSCs) by forcing cell-cycle progression and transiently up-regulating components of the homology-directed recombination machinery. The findings are validated by clonal tracking of the edited HSCs in experimental transplantation models, which shows improved polyclonal engraftment by long-term repopulating HSCs, the company said. People with genetic diseases affecting the hematopoietic lineage may benefit from corrective targeted gene therapy in HSCs. Those cells are self-renewing and can differentiate into all the cell types of the hematopoietic lineage, providing the potential for a one-time therapy, the company said.
Hoth Therapeutics Inc., of New York, entered a sponsored research agreement with Virginia Commonwealth University for a COVID-19 treatment. The collaboration is designed to develop and optimize peptide biomimetics to achieve high binding rates of peptide to the RBD of SARS-CoV-2 spike protein at the nanomolar level with demonstrated negligible toxicity through combined computational and experimental efforts. The optimized, in silico peptides will be synthesized and experimentally characterized through structure determination, binding to the spike protein and finally cell challenge assays with SARS-CoV-2. Hoth has exclusive license to the peptide COVID-19 therapeutic.
Shareholders of Arya Sciences Acquisition Corp. and Immatics Biotechnologies GmbH, of Munich and Houston, approved a business combination agreement. Shares of the combined company, Immatics NV, will be traded under the symbol IMTX. Arya is a special purpose acquisition company and Immatics develops T-cell redirecting cancer immunotherapies.
Marker Therapeutics Inc., of Houston, said it executed a lease agreement to establish a 48,500-square-foot cGMP manufacturing facility to allow scalable production according to FDA guidelines. The facility is expected to be completed by year-end 2020 and operational in 2021.
Meissa Vaccines Inc., of South San Francisco, said it initiated preclinical studies and manufacturing and completed a pre-IND meeting with the FDA to address development of MV-014-210, a live attenuated vaccine candidate to induce immunity and protect against SARS-CoV-2. The candidate was derived by modifying the company’s respiratory syncytial virus (RSV) candidate, MV-012-968, and replacing the RSV glycoproteins with a functioning SARS-CoV-2 Spike protein. Clinical trials are expected to begin in early 2021.
Optinose Inc., of Yardley, Pa., said it initiated development of OPN-019, which combines the company’s nasal exhalation delivery system technology with an antiseptic shown in vitro to kill the virus that causes COVID-19. Because components of the drug-device combination candidate are commercially available in the U.S., the company expects to progress rapidly to a meeting with the FDA to discuss an IND and then to initiate clinical trials.
Vertex Pharmaceuticals Inc., of Boston, said it expanded its reimbursement agreement with NHS England for cystic fibrosis medicines to include Kaftrio (ivacaftor/tezacaftor/elexacaftor), in a combination regimen with Kalydeco (ivacaftor) 150 mg, ahead of the medicine’s anticipated approval by the European Commission.
Ziphius Therapeutics, of Ghent, Belgium, said it obtained promising preclinical results for ZIP-1642, an mRNA-based vaccine against the novel coronavirus, and is preparing to move the candidate into the clinic by year-end 2020. The Ziphius vaccine consists of a combination of mRNA molecules that encode SARS-CoV-2 antigens, such as a prefusion stabilized form of the Spike protein. Additional details were not disclosed.