4D Molecular Therapeutics Inc. (4DMT), of Emeryville, Calif., said it concluded an agreement with the Cystic Fibrosis Foundation (CF Foundation) to develop precision gene medicines for cystic fibrosis (CF). The foundation will support the completion of IND-enabling R&D activities and the planned phase I/II study of 4D-710, the company’s wholly owned CFTR gene stimulator for the aerosol treatment of CF lung disease, with $14 million in committed funding, including a $10 million equity investment in 4DMT’s recently completed series C financing and additional funding contingent upon achievement of a preclinical milestone, all part of the Foundation’s $500 million Path to a Cure initiative. 4DMT, which will match the Foundation’s funding, retained global development and commercialization rights to 4D-710 and other potential medicines.
Amarin Corp. plc, of Dublin, said it reached an agreement with Apotex Inc., of Toronto, to resolve patent litigation that would have resulted from the abbreviated new drug application filed by Apotex with the FDA seeking approval of a generic form of Vascepa (icosapent ethyl) based on Amarin’s Marine study. Although Amarin is currently appealing to the U.S. Court of Appeals for the Federal Circuit regarding a March 2020 patent invalidity ruling by the U.S. District Court for the District of Nevada that favored generic drugmakers Hikma Pharmaceuticals USA Inc., a unit of Hikma Pharmaceuticals plc, of London, and Dr. Reddy’s Laboratories, of Hyderabad, India, Apotex was not a party to that litigation. As part of the settlement agreement, Apotex agreed that it may not sell a generic version of Vascepa in the U.S. until Aug. 9, 2029 – the same date named in a 2018 settlement agreement with Teva Pharmaceuticals USA Inc. – unless certain circumstances occur, including an unsuccessful appeal or en banc review of the Hikma/Dr. Reddy’s decision. The agreement also resolved future potential litigation with Apotex related to Vascepa’s cardiovascular risk reduction indication based on the Reduce-It study. Amarin did not commit to supplying Apotex with icosapent ethyl at any time. Additional terms were not disclosed.
Armata Pharmaceuticals Inc., of Marina del Rey, Calif., said it received a $15 million award for a three-year program from the U.S. Department of Defense through the Medical Technology Enterprise Consortium, with funding from the Defense Health Agency and Joint Warfighter Medical Research Program. Armata will apply funds from the award toward a phase Ib/II dose-escalation study of its phage-based candidate, AP-SA02, to treat complicated Staphylococcus aureus bacteremia infections. Primary objectives include assessments of safety and tolerability of AP-SA02 as an adjunct to best available antibiotic therapy and determination of appropriate doses for future efficacy trials. Due to the impact of the COVID-19 pandemic, Armata does not expect the trial to initiate prior to mid-2021, although a trial of AP-PA02 targeting Pseudomonas aeruginosa is set to begin enrolling in 2020.
Berg LLC, of Framingham, Mass., said it plans to tap into the Summit supercomputer located at the Department of Energy's Oak Ridge National Laboratory (ORNL) to conduct drug discovery research in the fight against COVID-19. The partnership will extend ORNL's ongoing drug development research on potential small-molecule drug compounds that might warrant further study against the SARS-CoV-2 coronavirus with Berg's human-derived disease model for rapid identification of targets. The company will be able to leverage its Interrogative Biology platform to assist in conducting research that identifies high priority targets where drug treatment could be introduced to treat COVID-19 infections.
Cellics Therapeutics Inc., of San Diego, said results of a study evaluating the potential benefits of macrophage and pulmonary epithelial nanosponges, derived from human cells, in neutralizing SARS-CoV-2 infectivity have been published in Nano Letters. Nanosponges, derived from macrophages and human pulmonary epithelial cells, were incubated with SARS-CoV-2-infected cells to assess if they are able to neutralize live SARS-CoV-2 virus, and they demonstrated significant neutralization of SARS-CoV-2 infectivity after two hours of incubation in a concentration-dependent manner. The company is currently developing macrophage nanosponges for the treatment of sepsis.
Clearside Biomedical Inc., of Alpharetta, Ga., which is developing treatments that restore and preserve vision for people with serious back-of-the-eye diseases, said it is making multiple presentations on its pipeline and SCS microinjector targeting the suprachoroidal space at the ARVO 2020 meeting. The data will include CLS-AX, a suspension of axitinib delivered via the SCS microinjector, which demonstrated sustained, well-tolerated and targeted delivery to the back of the eye in preclinical studies. In addition, CLS-AX showed inhibition of neovascularization and leakage, as well as durability. With its intrinsic highly potent pan-VEGF inhibition through receptor blockade, CLS-AX has the potential to be an effective therapy for neovascular age-related macular degeneration, the company explained. In addition, preclinical delivery of a complement inhibitor showed the agent was well-tolerated, sustained high drug levels, and merits further studies in the development of long-acting small-molecule complement inhibitors for dry age-related macular degeneration.
Cyclo Therapeutics Inc., of Gainesville, Fla., which is developing cyclodextrin-based products for the treatment of Niemann-Pick disease type C (NPC) and Alzheimer’s disease, said the final report from Kerwin Research Center on “Expanded access to Trappsol Cyclo for an individual patient with late onset Alzheimer’s disease” has been received by the FDA. The company collaborated with the center in the conduct of the expanded access program over the course of 18 months to evaluate Trappsol Cyclo, its formulation of hydroxypropyl beta cyclodextrin, as a potential disease-modifying treatment in a single patient with dementia of the Alzheimer’s type. The drug was administered once a month by intravenous infusion. The report states that the drug showed a favorable safety profile as well as encouraging observations of benefit.
Innovation Pharmaceuticals Inc., of Wakefield, Mass., said data from ongoing laboratory testing, being conducted at a U.S. Regional Biocontainment Laboratory (RBL), showed brilacidin exhibited a statistically significant (p<0.0001) and potent inhibitory effect on SARS-CoV-2 in a human lung epithelial cell line –reducing viral load by 95% and by 97%, compared to control – at two therapeutic concentrations tested. Based on a CC50 value, the concentration of drug at which 50% of cells maintain viability, brilacidin was also shown to be noncytotoxic in the lung cell line. Testing results observed to date formed the basis for a federal grant application that was submitted last week by the RBL, in collaboration with the company, proposing to evaluate brilacidin’s potential as a pan-coronavirus therapeutic, with possible extension into other viruses. The company is in the process of manufacturing the product for intravenous dosing and will be seeking FDA guidance for a planned COVID-19 clinical study.
ISA Pharmaceuticals BV, of Leiden, the Netherlands, said it plans to start a potentially pivotal trial for the combination of ISA-101b and Libtayo (cemiplimab, Regeneron Pharmaceuticals Inc./Sanofi SA) in oropharyngeal cancer, under its strategic immuno-oncology collaboration with Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y., which has increased its equity stake in the company. ISA-101b, an immunotherapy targeting human papillomavirus type 16 (HPV16), is in a phase II trial for first- and second-line HPV16-positive head and neck cancer in combination with Libtayo, a PD-1 inhibitor. A second phase II trial investigating the same combination in cervical cancer is also planned.
Kamada Ltd., of Rehovot, Israel, said it completed manufacturing of the first batch of its plasma-derived IgG product for COVID-19 utilizing its IgG platform technology, and additional production is ongoing. The initial vials are available for compassionate use in Israel. In addition, the company’s proposed clinical protocol for a phase I/II trial was submitted to the Israeli Ministry of Health, and the company expects to initiate the study during the third quarter of the year. Also, with the support of Kedrion Biopharma Inc., of Castelvecchio Pascoli, Italy, it intends to conduct a pre-IND meeting with the FDA in the third quarter in order to obtain the agency’s acceptance of a proposed U.S. clinical development program.
Stockholders at Lantheus Holdings Inc., of North Billerica, Mass., approved issuing common stock related to the company’s proposed merger with Progenics Pharmaceuticals Inc., of Tarrytown, N.Y. The merger is expected to close on or about June 19. Lantheus and Progenics entered an amended and restated agreement and merger plan Feb. 20, with Progenics to become a wholly owned Lantheus subsidiary.
Nascent Biotech Inc., of San Diego, said pritumumab will be advanced to laboratory-based animal studies following in vitro studies. The compound is being investigated for potential use with COVID-19 and related viral Infections.
NTC Srl, of Milan, Italy, said it granted exclusive license and distribution rights in Israel to one of its research and development products, a fixed combination in eyedrops, to Taro Pharmaceutical Industries Ltd., of Hawthorne, N.Y. The product combines a quinolone antibiotic with an anti-inflammatory steroid in eyedrops for post-cataract surgery. NTC said it completed Leader7, an international, clinical study designed to compare a one-week course of antibiotic prophylaxis to a two-week course with a reference comparator, in the prevention and treatment of inflammation and the prevention of infection associated with cataract surgery in adults.
PDS Biotechnology Corp., of Florham Park, N.J., and Farmacore Biotechnology Ltd., of Brazil, will collaborate on a COVID-19 vaccine, accelerating development of Versamune-CoV-2FC into phase I testing in Brazil, with initial financial support provided by the Brazilian Ministry of Science, Technology, Innovation and Communication. Versamune-CoV-2FC combines a Farmacore-developed recombinant SARS-CoV-2 protein with PDS’ platform nanotechnology. The program expands the R&D collaboration between PDS and Farmacore to develop a tuberculosis vaccine. Farmacore retains commercialization rights in Latin America while revenues from Latin America sales will be shared. PDS retains the right of first refusal for commercialization outside of Latin America.
Sirnaomics Inc., of Gaithersburg, Md., and Precision Nanosystems Inc., of Vancouver, British Columbia, are partnering to develop and manufacture a polypeptide nanoparticle-based RNAi therapy. The collaboration includes execution of a platform license and supply agreement, plus productions of Sirnaomics’ candidates STP-705 and STP-707 for ongoing IND-enabling safety, toxicity and clinical studies. STP-705, Sirnaomic’s lead candidate, is an siRNA therapeutic with a dual-targeted inhibitory property and a polypeptide nanoparticle-enhanced delivery to knock down TGF-β1 and COX-2 gene expression.
Staidson Biopharma Inc., of Beijing, and European CRO Pivotal said they are collaborating to assess Staidson’s BDB-001, a monoclonal anti-C5a antibody, in a Europe-based clinical trial in progressive severe COVID-19. A growing body of evidence indicates that SARS-CoV infection can overactivate the complement cascade, thus affecting other organs and producing disease exacerbation. It has been shown that inhibiting the strongest anaphylatoxin C5a can alleviate MERS-CoV-mediated lung tissue damage in infected preclinical models, the company said. The study is expected to enroll 60 patients and will measure the efficacy of BDB-001 vs. placebo and best supportive care in COVID-19 hospitalized patients who are suffering from severe pneumonia and acute respiratory distress syndrome.
Transcelerate Biopharma Inc., of Philadelphia, said it expanded access to COVID-19-related patient-level clinical data-sharing via its platform to non-member companies. Transcelerate will enlarge the type of data shared to include data from both the investigational product and control arms of COVID-19 clinical trials. The cloud-based sharing platform allows for deidentified, anonymized preclinical and clinical data types to be requested and voluntarily shared.
Ubiquigent Ltd., of Dundee, Scotland, is continuing its collaboration with New York-based Bristol Myers Squibb Co. (BMS) that allows BMS to use Ubiquigent’s deubiquitylase (DUB) enzyme inhibitor drug discovery platform. The central role of ubiquitylation in diverse cellular processes, including protein degradation, makes the enzymes responsible for ubiquitylation and de-ubiquitylation attractive drug targets to address a range of pathologies. Since the clinical approval of proteasome inhibitors, there has been growing interest in modulating the various components of the ubiquitylation machinery, such as DUBs, or exploiting the ubiquitin pathway via proteolysis-targeting chimeras and molecular glues to target the undruggable genome, Ubiquigent said.