Scrip: industry news and insights
18 Sep 2020
A composite endpoint conceals Actemra’s lack of mortality benefit – while life-saving dexamethasone gets EMA endorsement.
Below is Scrip’s regular roundup, including information from Strategic Transactions, of recent technology transfer agreements and collaborations between companies and academic or other research institutions.
T-cell immunotherapy firm AlloVir Inc. reported on 23 March that it is expanding its research collaboration with Baylor College of Medicine to discover and develop off-the-shelf, allogeneic, virus-specific TCR therapies to treat SARS-CoV-2, the coronavirus strain that causes COVID-19.
The Cambridge, MA-based company, then known as ViraCyte, licensed T-cell receptor technology from Baylor University in 2017 to advance its Viralym-M off-the-shelf therapy into clinical development against multiple pathogens.[See Deal] Now in Phase II, Viralym-M treats up to five different infections simultaneously – adenovirus, BK virus, cytomegalovirus, Epstein-Barr virus and human herpesvirus-6.
In addition to SARS-CoV-2, the expanded collaboration also might address other coronavirus strains including SARS-CoV, MERS-CoV and the endemic CoVs that commonly afflict immunocompromised patients. AlloVir aims to develop a therapy for CoVs that can be used as a stand-alone treatment or incorporated into its multi-respiratory virus therapy, ALVR106, which is designed to address other devastating and life-threatening community-acquired respiratory viruses.
In February, Viralym-M obtained priority medicines (PRIME) status from the European Medicines Agency, after the candidate got a regenerative medicine advanced therapy (RMAT) designation from the US Food and Drug Administration in 2019. (Also see "AlloVir Gains PRIME Status For Anti-Virus T-Cell Therapy" - Scrip, 13 Feb, 2020.)
In another ongoing research collaboration being expanded into coronavirus work,Soligenix Inc. said on 23 March that an ongoing partnership with University of Hawaii researchers into heat-stable subunit filovirus vaccines for Ebola and Marburg viruses will investigate the technology’s potential application to coronaviruses, including SARS-CoV-2.
The partners claim that their platform technology could yield vaccines that are broadly applicable, including patients often excluded from common viral vector vaccine approaches such as children, the elderly and immunocompromised patients. The platform comprises a viral surface glycoprotein that mediates entry and fusion of the virus with host cells, an adjuvant to enhance cell-mediated and humoral immunity, and a formulation that avoids the need for cold-chain storage and shipping.
The research has produced formulation conditions that enable heat stabilization of each antigen for vaccine, separately or in combination, for at least 12 weeks at temperatures up to 40 Celsius/104 Fahrenheit. The vaccines are manufactured using a proprietary insect-cell expression system that offers protein-specific affinity purification, Soligenix said.
Virginia-based Cel-Sci Corp. and the University of Georgia Research Foundation’s Center for Vaccines and Immunology said on 23 March that they’ll work together to test the biotech’s Ligand Antigen Epitope Presentation System (LEAPS) peptide technology to develop a treatment for patients at greatest risk of dying due to lung tissue damage resulting from COVID-19.
The research will investigate technology that previously demonstrated an ability to reduce morbidity and mortality in mice infected with the H1N1 influenza virus in a study funded by the National Institute of Allergy and Infectious Diseases.
Cel-Sci said that prior research might indicate that the LEAPS technology could yield a coronavirus immunotherapy that would reduce or arrest progression of the SARS-CoV-2 virus and prevent the tissue damage resulting from virally induced inflammation in the lungs.
Under a research and licensing agreement unveiled on 18 March, Asklepios BioPharmaceutical Inc. and the University of North Carolina will combine their technologies to develop and commercialize a gene therapy for Angelman syndrome. No financial terms were disclosed.
Asklepios said the partnership will bring together groundbreaking research into the disease at UNC with its adeno-associated virus technology. Currently an unmet medical need with an estimated 500,000 patients worldwide, Angelman is a neurogenetic disorder caused by loss of function by the UBE3A gene.
DeMelle OncoPharma LLC announced on 4 March that it had licensed a first-in-class targeted therapy for pancreatic cancer from Florida’s Moffitt Cancer Center. The Phase II-ready compound, known as DM3001, targets cancers expressing the novel Exportin-2 target.
Moffitt researchers previously completed a Phase Ib study with the compound where it penetrated the pancreatic stromal barrier to produce regulated cancer cell death with minimal impact on nearby healthy tissue. In preclinical study, the drug showed an ability to inhibit pancreatic and colon cancer growth and also showed superior activity compared to gemcitabine.
DeMelle said it anticipates that DM3001 will be able to treat a broad range of cancers. No financial terms of the deal were disclosed.
On 25 February, Moffitt announced a collaboration with Oncologie Inc. to study treatment effects of various approaches targeting the tumor microenvironment in gastric and gastrointestinal cancers. The partners said they hope to detail their findings at a medical conference later this year.
Genprex Inc. received exclusive rights on 11 February from the University of Pittsburgh to a potentially curative gene therapy for type 1 and type 2 diabetes. Discovered and developed by Pitt researcher George Gittes, the gene therapy can reprogram beta cells in the pancreas to restore their function and thus replenish insulin levels. In murine models, the candidate restored normal blood glucose levels for an extended period of time (around four months).
Gittes believes that the therapy can potentially restore blood glucose levels for decades in humans. A Phase I trial is planned for the therapy, which would be the first diabetes gene therapy tested in humans. Should it successfully be developed, it would offer a new treatment option for diabetes patients who rely on insulin-replacement therapy.
Bridge Biotherapeutics Inc. licensed rights on 5 February to a compound for back-eye disorder from South Korea’s Konkuk University. Bridge paid $251k (KRW300m) up front and could shell out another $8.1m in development and regulatory milestones.
The licensed candidate is an early-stage inhibitor of undisclosed target proteins. The company will handle further development and gets exclusive global rights to the product. There is an unmet need for back-eye disease therapies.
Bridge Medicines and New York’s Rockefeller University announced a licensing agreement on 16 March to develop novel inhibitors of the epigenetic reader protein ENL-YEATS to treat acute forms of leukemia and possibly solid tumors.
Zelluna Immunotherapy and Karolinska Institute unveiled a multi-year research collaboration on 12 March to develop next-generation T-cell receptor-guided natural killer products for solid tumors.
ViiV Healthcare and the University of North Carolina signed a five-year renewal on 9 March of their research collaboration to cure HIV by inducing HIV cells that hide in immune cells during antiretroviral therapy so that they can be eliminated.
RespireRx Pharmaceuticals Inc. announced on 4 March that it signed a six-month option with the University of Wisconsin-Milwaukee to license intellectual property related to GABA(a) receptor allosteric modulators that may offer therapeutic potential in epilepsy and other convulsive disorders.
Denali Therapeutics Inc. revealed on 20 February that it will collaborate with the Neurodegeneration Consortium at the MD Anderson Cancer Center to investigate four therapeutic targets with a goal of developing potential small molecule therapies for Alzheimer’s disease and other neurodegenerative conditions.
Cell and gene therapy service provider Orgenesis Inc. unveiled an agreement on 18 February to provide cell and gene therapy technologies to Johns Hopkins University to assist in its development of cell-based immunotherapies.
Synspira Therapeutics said on 12 February that the Cystic Fibrosis Foundation will support its development of SNSP003, an oral, non-porcine enzyme replacement therapy for malabsorption syndromes, which are a comorbidity of CF.
Oklahoma’s Hough Ear Institute reported on 11 February that it has licensed noise-induced hearing loss candidate NHPN-1010 to Oblato Inc. at undisclosed terms. Previous partner Otologic Pharmaceutics Inc. completed a Phase I safety study in 2015 and Oblato is expected to conduct a Phase II study with the institute.
Scrip: industry news and insights
18 Sep 2020
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Explore how digital technologies are being used to address the disruption of clinical trials for drugs and medical technology caused by COVID-19. Stay current with insights from industry leaders, information on guidance and regulation, and an examination of how the challenges of this global crisis and the industry’s response to it may soon usher in a new era in worldwide healthcare.
Scrip: industry news and insights
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