Anavex Life Sciences Corp., of New York, said it is responding to regulatory, institutional and government guidance and policies related to COVID-19. It remains committed to its clinical development plans and is working closely with all stakeholders to try to mitigate the effect of COVID-19 on the company’s ongoing clinical trials for Rett syndrome, Parkinson’s disease dementia and Alzheimer’s disease. The company reports that clinical sites continue to operate and see patients where possible, The sites continue to screen and enroll patients into the active trials and the respective open-label extensions for the two phase II Rett syndrome trials and the phase IIb/III Alzheimer’s disease trial, while the Parkinson’s disease dementia phase II trial has completed enrollment and top-line results are expected from this study by mid-2020.
Bionomics Ltd., of Adelaide, Australia, said a paper describing the pharmacology of BNC-375, a positive allosteric modulator of the alpha 7 nicotinic acetylcholine receptor, was published online in the Journal of Pharmacology and Experimental Therapeutics. It describes the preclinical pharmacology of the compound targeting cognitive dysfunction associated with Alzheimer’s disease and other central nervous system conditions. The paper presents data from a comprehensive range of preclinical studies performed with BNC-375 to explore the potential of the molecule to improve cognitive impairment.
Brii Biosciences Ltd., of Durham, N.C., and Beijing, along with Tsinghua University and the 3rd People’s Hospital of Shenzhen agreed to collaborate to discover, develop, manufacture and commercialize fully human neutralizing monoclonal antibodies to address the COVID-19 pandemic. Tsinghua and 3rd People’s Hospital published results in BioRxiv identifying multiple neutralizing monoclonal antibodies with therapeutic potential against SARS-CoV-2 that have been characterized from patients in China who have recovered from COVID-19. With Brii Bio, they intend to advance multiple candidates for prophylactic and therapeutic intervention.
Cytomx Therapeutics Inc., of South San Francisco, said it hit a clinical milestone in its CX-2029 program, triggering a $40 million payment from Abbvie Inc., of North Chicago. In April 2016, the two companies entered a co-development and licensing agreement for the Probody drug conjugate against CD71. CD71, also called the transferrin receptor 1, is a cell surface protein needed for iron uptake in dividing cells and is highly expressed in several solid and hematologic cancers. Cytomx is responsible for clinical development up to initial clinical proof of concept and Abbvie will lead late-stage clinical development and global commercial activities. Cytomx could receive a profit share in the U.S. and tiered double-digit royalties on net product sales outside of the U.S. Cytomx retains an option to co-promote in the U.S.
Dyne Therapeutics Inc., of Waltham, Mass., received an equity investment from CureDuchenne Ventures to help develop therapeutics to treat Duchenne muscular dystrophy. Dyne’s platform is designed to help deliver exon-skipping therapeutics to skeletal, cardiac and smooth muscle to improve efficacy and reduce dosing frequency. Its therapeutic molecule consists of an antibody linked to an exon-skipping oligonucleotide. The antibody binds to receptors that are highly expressed on the surface of muscle cells. The amount of the funding was not disclosed.
Evotec SE, of Hamburg, Germany, will use its platform in a collaboration with Seoul, South Korea’s Ildong Pharmaceutical Co. Ltd. to develop Ildong’s products. The two companies agreed to jointly develop three to six projects, the first being IDG-16177, a glucose-lowering drug candidate being evaluated to treat type 2 diabetes.
Genfit SA, of Lille, France, said all its phase I trials – including pharmacokinetic, food effect and bioequivalence studies – have been put on hold. It has also put its phase II combination study and phase III study in primary biliary cholangitis on hold. The company said it will continue the extension phase of its RESOLVE-it trial using virtual clinic visits, local lab assessment, home delivery of the study drug and the halting of screening new patients. The company’s most advanced drug candidate, elafibranor, is being evaluated in the RESOLVE-IT study as a NASH treatment.
Horizon Discovery Group plc, of Waterbeach, U.K., and Budapest’s Pharmahungary Group reported positive early stage results for their micro-RNA therapeutic for treating ischemic heart diseases. The micro-RNA (an miRNA-125b-mimic compound) was identified as suitable for further development based on favorable pharmacokinetic results as well as pharmacodynamic data at the molecular level.
Immunoprecise Antibodies Ltd., of Victoria, British Columbia, said its collaborator, Evqlv Inc., of New York, submitted its first panel of antibody sequences composed of DNA sequences encoding SARS-CoV-2. Immunoprecise said it will review the candidates, then select about 1,200 for in vitro validation.
Index Pharmaceuticals Holding AB, of Stockholm, received a SEK2 million (US$200,000) grant from innovation agency Vinnova for preclinical development of compounds for treating inflammation. Index’s platform uses oligonucleotides that function as immunomodulatory agents by binding to Toll-like receptor 9. The company’s lead compound is cobitolimod, which met the primary endpoint of its phase IIb study for treating moderate to severe ulcerative colitis.
Junshi Biosciences, of Shanghai, said its proposed A-share listing has been approved by the listing committee of the Shanghai Stock Exchange Star Market. The listing is subject to registration approval by the China Securities Regulatory Commission. Junshi said it has 21 candidates in its R&D pipeline for treating cancer, metabolic diseases, autoimmune diseases and neurologic diseases.
Kleo Pharmaceuticals Inc., of New Haven, Conn., said it will collaborate with South Korea’s Green Cross Labcell to develop COVID-19 treatments. Kleo’s platform is designed to develop synthetic bifunctional molecules to redirect antibodies and keep them from binding on the virus to human cells. Green Cross focuses on NK cell and stem cell therapeutics.
Myovant Sciences GmbH, of Basel, Switzerland, and Gedeon Richter plc, of Budapest, Hungary, signed an exclusive license agreement for Gedeon to commercialize the relugolix combination tablet (relugolix 40 mg, estradiol 1 mg and norethindrone acetate 0.5 mg) for uterine fibroids and endometriosis in Europe, the Commonwealth of Independent States including Russia, Latin America, Australia and New Zealand. Myovant will receive an up-front payment of $40 million and is eligible to receive up to $40 million in regulatory milestones and $107.5 million in sales-related milestones, and tiered royalties on net sales following regulatory approval. Myovant retains all rights to the relugolix combination tablet in the U.S., as well as rights to relugolix in other therapeutic areas outside of women’s health. Myovant will continue to lead the global development of the tablet. Gedeon will be responsible for local clinical development, manufacturing and all commercialization for its territories. Myovant has also granted Gedeon an option to collaborate on the tablet for future indications in women’s health other than fertility.
Neubase Therapeutics Inc., of Pittsburgh, disclosed positive preclinical data from its pharmacokinetics studies in nonhuman primates and in vitro pharmacodynamics data in patient-derived cell lines. Neubase said it believes the data validate the key advantages of its peptide-nucleic acid antisense oligonucleotide platform and support the company’s decision to advance the development of its Huntington’s disease and myotonic dystrophy type 1 programs, as well as the potential expansion of its therapeutic pipeline into other indications. Quantitative whole-body autoradiography was performed on the primates to gain the data, Neubase said.
Newron Pharmaceuticals SpA, of Milan, Italy, said, due to the COVID-19 pandemic, it anticipates a delay in the start of its planned studies with evenamide in schizophrenia. The company said it cannot predict the length of the delay but will continue to work to prepare the studies for initiation as soon as it is safe to do so. Evenamide, a drug that targets the voltage-gated sodium channel, so far has demonstrated promising results as an add-on therapy in a phase II schizophrenia trial.
Nuvo Pharmaceuticals Inc., of Mississauga, Ontario, said its wholly owned subsidiary, Nuvo Pharmaceuticals DAC, has received notice from Takeda Pharmaceutical Co. Ltd., of Tokyo, that Japan's Ministry of Health, Labor and Welfare has approved Cabpirin combination tablets, pairing the potassium-competitive acid blocker vonoprazan fumarate and low-dose aspirin, which will be co-promoted in Japan by Takeda and its co-promotion partner Otsuka Pharmaceutical Co. Ltd., of Tokyo. Takeda holds a nonexclusive license to Nuvo Ireland's Japanese patent, which covers the Cabpirin formulation. The approval triggers a $2 million milestone payment from Takeda to Nuvo Ireland, which expects receiving the payment no later than May 29, 2020.
Orgenesis Inc., of Germantown, Md., said it has developed a manufacturing process for Bioxomes through its collaboration and licensing agreement with Excellabio Ltd., of New York. Exosomes, or extracellular vesicles, are small vesicles that transfer DNA, RNA and proteins to other cells, thereby altering the function of the targeted cells. They are believed to provide the same therapeutic benefit of cells without the risks and difficulties of administering entire cells to patients. Orgenesis and Excellabio have developed Bioxomes, which are synthetic exosomes. Until now, exosome production has been based on conventional ultracentrifugation or ultrafiltration, both of which are complex and costly techniques, the companies said.
Oxford Biotherapeutics Ltd., of Oxford, U.K., said partner Boehringer Ingelheim GmbH, of Ingelheim, Germany, has selected a second asset to advance into formal IND-enabling studies. The asset discovery was enabled by Oxford’s OGAP target discovery platform. It is one of several assets that have emerged from OGAP, including some in preclinical and clinical development by Oxford. Boehringer’s selection triggered a milestone payment to Oxford.
Phio Pharmaceuticals Corp., of Marlborough, Mass., said data from various animal studies validated the potential of Phio's Intasyl technology as a cancer immunotherapy platform for developing novel compounds. The preclinical findings, which will be presented in further detail at upcoming scientific conferences, demonstrate that the Intasyl-enabled compounds can effectively be used to reduce immunosuppression in the tumor microenvironment. Phio scientists conducted several animal studies with a mouse version of PH-762 (mPH-762) and with PH-894 in a validated mouse model of cancer (murine hepatocellular carcinoma model with Hepa1-6 cells). The studies show that a local administration of mPH-762 or PH-894 through intratumoral injection resulted in potent antitumor effects. The treated animals showed a complete and statistically significant (p<0.0001) inhibition of tumor growth, whereas placebo-treated animals displayed exponential tumor growth, Phio said.
Predictive Oncology Inc., of Minneapolis, disclosed continued progress by its Helomics subsidiary toward the goal of developing an artificial intelligence (AI)-driven predictive model of ovarian cancer that will predict drug response and outcome. Predictive models embody the knowledge from those profiles to create a “computational expert” or a “virtual patient” that can be queried, the company said. Such models can be used to quickly select patients for clinical trials and assess potential new drugs or biomarkers computationally before initiating expensive laboratory experiments, Predictive noted.
Primmune Therapeutics Inc., of San Diego, said it has selected PRTX-007 as its lead clinical development candidate for SARS-CoV-2 and for oncology indications. PRTX-007 is an oral small molecule that specifically activates Toll-like receptor 7, which drives the innate immune system's primary response to viral infections, the company said. It was derived from Primmune's extensive discovery program and has demonstrated sustained dose-dependent innate immune induction in vivo without the production of inflammatory cytokines, such as interleukin-6 or tumor necrosis factor-alpha.
SAB Biotherapeutics Inc., of Sioux Falls, S.D., was awarded a contract from the U.S. Department of Defense to develop and test a rapid response antibody program, valued at up to $27 million. The three-stage, multiyear contract calls for the development of a state-of-the-art, pharmaceutical platform technology capable of rapidly and reliably producing antibody-based medical countermeasures for biological threats. The goal of the program is to accelerate the delivery of potent, human, polyclonal antibody therapeutics to address known and novel emerging biodefense (viral, bacterial or toxin) threats.
Sirona Biochem Corp., of Vancouver, British Columbia, said its French subsidiary lab, Tfchem, has initiated an antiviral program to produce potential new antiviral agents to assist in combating COVID-19 and other infectious diseases. After two months of extensive research, Tfchem scientists have determined that its platform technology can be applied to develop new antiviral compounds. Work on the program has begun with a goal to produce new therapies in three current antiviral categories: neuraminidase inhibitors, nucleoside analogues and iminosugars. Those categories are all glycosides with a carbohydrate core and therefore suitable for application of the technology, the company noted.
The La Jolla Institute for Immunology awarded $1.73 million from the Bill & Melinda Gates Foundation to create the Coronavirus Immunotherapy Consortium, designed to find antibodies effective against the coronavirus SARS-CoV-2 and accelerate the research pipeline to provide immunotherapeutics to protect people from severe manifestations of COVID-19. The institute said it plans to use Salt Lake City-based Carterra Inc.’s platform to screen hundreds of antibodies. The funding is part of the COVID-19 Therapeutics Accelerator launched in early March.
Vaxart Inc., of South San Francisco, said it had produced five COVID-19 vaccine candidates for testing in its preclinical models. Each of the COVID-19 vaccine constructs is based on a different coronavirus antigen combination, and Vaxart expects to advance the best performing vaccine to manufacturing for clinical trials. A phase I study could start in the second half of 2020.
VBI Vaccines Inc., of Cambridge, Mass., said it is collaborating with the National Research Council of Canada (NRC), Canada’s largest federal research and development organization, to develop a pan-coronavirus vaccine candidate targeting COVID-19, severe acute respiratory syndrome (SARS) and Middle East respiratory syndrome (MERS). VBI will use its enveloped virus-like particle (eVLP) platform technology and, based on past clinical experience with the eVLP platform, said it expects it will be possible to develop a multivalent eVLP vaccine candidate, co-expressing SARS-CoV-2, SARS-CoV and MERS-CoV spike proteins on the same particle. The collaboration will combine VBI’s viral vaccine expertise, eVLP technology platform, and coronavirus antigens with the NRC’s COVID-19 antigens and assay development capabilities to identify the most immunogenic vaccine candidate for further development.
Viscient Biosciences Inc., of San Diego, which focuses on using 3D bioprinting technology to displace the use of animal models in pharma research, said it – or a sister company to be funded and launched shortly using the same technology – will use 3D bioprinting to create lung tissue to support viral infectivity research and search for an effective therapy against SARS-CoV-2, the coronavirus which causes COVID-19. Using the paradigm developed for liver as well as previous work in lung tissue, 3D-bioprinted and other 3D tissue models made with lung cells, including a patient's own cells, are expected to be used as a "clinical trial in a dish," helping test potential COVID-19 therapies quickly and with highly accurate biology. Previous research has already shown that 3D human lung tissue better models viral infectivity compared to regular cell culture, Viscient said.
Windtree Therapeutics Inc., of Warrington, Pa., said it entered a binding term sheet with Lee's Pharmaceutical Holdings (HK) Ltd., of Hong Kong, in which Lee's will provide project financing for the continued development of Windtree’s lead acute pulmonary product candidate, Aerosurf, for the treatment of preterm infants with respiratory distress syndrome. The financing will fund the Aerosurf phase IIb bridge study which, if successful, is intended to transition the product into a phase III-ready clinical product development. The terms sheet calls for Lee’s to make nonrefundable payments to Windtree during the period from April 1 through Sept. 1. The parties have agreed that they will negotiate in good faith the terms of a definitive agreement prior to Sept. 30, which will also set forth additional semiannual nonrefundable payments to fund continued development subsequent to Sept. 30. Windtree has agreed to repay 125% of the amounts funded by Lee's for the development of Aerosurf out of proceeds received from all revenue amounts and payments received by the company for any sale, divestiture, license or other development and/or commercialization of the KL4 surfactant/Aerosurf patent portfolio with certain exclusions.