1cellbio Inc., of Cambridge, Mass., said it established a partnership agreement with the Open Medicine Institute, an independent precision medicine clinical and research organization. The institute's researchers will apply and validate 1cellbio's Indrop system as part of a clinical research project to investigate the potential for single-cell RNA-sequencing technology in precision medicine applications. The collaboration will initially focus on generating predictive data for patients with an undiagnosed immune dysfunction condition. Researchers will attempt to identify putative biomarkers and demonstrate how an integrated 'omics approach can improve sensitivity, accuracy and specificity of complex immune illness diagnosis, and potentially guide therapeutic decision-making. The study will also use machine learning and additional big data analysis techniques to identify clinically relevant patterns of gene expression in single cells, as well as relationships between disparate-omic data types and single-cell expression patterns.
The Medicines Patent Pool, of Geneva, said it entered a royalty-free license agreement with North Chicago-based Abbvie Inc. for glecaprevir/pibrentasvir (G/P), a World Health Organization-recommended treatment for people living with chronic hepatitis C. The license will enable quality-assured manufacturers to develop and sell generic medicines containing G/P in 99 low- and middle-income countries and territories at affordable prices, enabling access to and treatment scale-up with the most effective pan-genotypic regimens.
Arena Pharmaceuticals Inc., of San Diego, presented preclinical data for its investigational compound, APD-418, a first-in-class calcium-independent myofilament derepressor, beta3 adrenergic receptor (AdrR) selective antagonist, at the American Heart Association Scientific Sessions 2018 in Chicago. The compound is being developed to improve cardiac contractility with minimal effect on heart rate and blood pressure for decompensated heart failure. Inhibition of beta3 AdrR-mediated myofilament repression may provide a unique cardiomyocyte-specific target to enhance cardiac contractile performance while avoiding adverse events associated with current inotrope therapies.
Astrazeneca plc,of Cambridge, U.K., said it agreed to sell the U.S. rights to Synagis (palivizumab), used for the prevention of serious lower respiratory tract infection (LRTI) caused by respiratory syncytial virus (RSV), to Stockholm-based Swedish Orphan Biovitrum AB (Sobi), which will now commercialize the product in the U.S. As part of the transaction, approximately 130 Astrazeneca employees will transfer to Sobi. Sobi will also have the right to participate in Astrazeneca's share of U.S. profits and losses related to a potential new medicine, MEDI-8897. Astrazeneca will continue to develop MEDI-8897, a single-dose extended half-life anti-RSV FmAb being developed for the prevention of LRTI caused by RSV in all infants entering their first RSV season and children with chronic lung disease or congenital heart disease entering their first and second RSV seasons, in collaboration with Sanofi Pasteur, the vaccines division of Paris-based Sanofi SA. Under the agreement, Astrazeneca will receive an up-front payment of $1.5 billion, consisting of $1 billion in cash and $500 million in ordinary shares of Sobi upon completion. It will also receive up to $470 million in sales-related payments for Synagis; a $175 million milestone following the submission of the biologics license application for MEDI-8897; potential net payments of approximately $110 million on achievement of other MEDI-8897 profit and development-related milestones; and a total of $60 million in non-contingent payments for MEDI-8897 during 2019-2021.
Catabasis Pharmaceuticals Inc., of Cambridge, Mass., said it is collaborating with Pradeep Mammen, founder and medical director of the Neuromuscular Cardiomyopathy Clinic at the University of Texas Southwestern Medical Center, to explore the potential of edasalonexent, an oral NF-kB inhibitor, to improve cardiac function in Duchenne and Becker muscular dystrophies. Young boys with Duchenne muscular dystrophy typically have an elevated heart rate that exceeds the normal resting rate for their age, which is the first cardiac manifestation in boys with the condition. The one-year collaboration will utilize the muscular dystrophy mdx mouse model with reduced utrophin as those animals display an early and prominent cardiomyopathy. The study will evaluate both functional and echocardiographic assessments of the heart as well as histological, biochemical and molecular assessments to study the potential benefits of edasalonexent treatment. Results are expected in the second half of next year.
Cocrystal Pharma Inc., of Bothell, Wash., said preclinical data presented at the ISIRV-AVG Conference, Advances in Respiratory Virus Therapeutics, in Washington, showed CC-42344, the company's broad-spectrum influenza A strain PB2 inhibitor, binds to a highly conserved PB2 site of influenza polymerase complex and exhibits a novel mechanism of action. The company is currently in IND-enabling studies.
CTD Holdings Inc., of Alachua, Fla., retained Torreya Capital LLC as its strategic advisor to support business development. No further details were disclosed.
Cybrexa Therapeutics Inc., of New Haven, Conn., presented preclinical data on its alphalex peptide conjugated to Talzenna (talazoparib, Pfizer Inc.) at the EORTC-NCI-AACR Molecular Targets and Cancer Therapeutics Symposium in Dublin. The conjugated drug had a pH-dependent delivery into tumor cells in vitro as well as sustained and selective in vivo tumor localization. The drug prevented bone marrow toxicity when combined with chemotherapy. In model systems, the drug killed xenografts that were either homologous recombination deficient (HRD) or non-HRD, including patient-derived xenografts.
Diamedica Therapeutics Inc., of Minneapolis, applied to list its common shares on Nasdaq. In conjunction with the planned listing, the company is doing a 20-for-1 reverse split of its shares, which is expected on or about Nov. 15.
Dnatrix Inc., of Houston, is collaborating with Valo Therapeutics Ltd., of Helsinki, to develop an oncolytic vaccine for cancer therapy using Dnatrix's adenovirus platform and Valo's peptide-coated conditionally replicating adenovirus vaccination technology. Terms of the collaboration weren't disclosed.
Enable Injections Inc., of Cincinnati, has entered strategic partnerships with both Apellis Pharmaceuticals Inc., of Crestwood, Ky., and UCB SA, of Brussels, to use Enable's Enfuse technology to develop wearable devices for subcutaneous delivery of high-volume therapeutics. Terms of the deals weren't disclosed.
Evox Therapeutics Ltd., of Oxford, U.K., secured £655,000 (US$849,000) in funding from Duchenne UK to test the ability of its exosome-based platform to deliver either full-length dystrophin or shorter variants of the gene in preclinical models of Duchenne muscular dystrophy.
Exuma Biotechnology, of Hong Kong, an affiliate of F1 Oncology Inc., of West Palm Beach, Fla., has formed Shanghai Exuma Biotechnology Co. Ltd., to acquire all rights to CAR T-cell therapy and manufacturing assets in Shanghai and Shenzhen for the development and commercialization of cellular therapies in greater China.
Gammadelta Therapeutics Ltd., of London, published data on gamma delta T-cell receptors in Nature Immunology. The receptors combine innate immunity with adaptive immunity using different regions of the receptor to differentiate healthy tissues from malignant cells.
Immunorestoration Inc., of Doylestown, Pa., completed the transfer of an IND for BB-IND-11043, an integrated dendritic cell-based immune system, from the investigator to the company. The phase I and phase II studies will continue under funding from Immunorestoration.
Invagen Pharmaceuticals Inc., of Hauppauge, N.Y., a subsidiary of Mumbai, India-based Cipla Ltd., is acquiring Avenue Therapeutics Inc., of New York, in a two-stage process. In the first stage, Invagen will acquire a 33.3 percent stake of Avenue for $35 million, or $6 per share. In the second stage, Invagen will acquire the remaining shares in a reverse triangular merger with Avenue remaining as the surviving entity, for up to $180 million in the aggregate, or approximately $13.92 per share. The second stage has conditions with respect to the approval of I.V. Tramadol, which is in phase III development.
Kempharm Inc., of Celebration, Fla., added KP-879 to its pipeline. The company plans to file an IND for KP-879, which uses serdexmethylphenidate, a prodrug of d-methylphenidate, as early as the end of 2018 to develop the drug as a treatment for stimulant use disorder.
Nash Pharmaceuticals Inc., of Vancouver, British Columbia, reported preclinical data for NP-178, a repurposed, oral neurological drug, which it's developing for ulcerative colitis. The drug improved body weight, stool consistency, colon length and weight ratios and occult positivity, comparable to 5-aminosalicylic acid. The data will be presented at an upcoming medical conference.
Syros Pharmaceuticals Inc., of Cambridge, Mass., reported preclinical data for its cyclin-dependent kinase 7 (CKD7) inhibitor, SY-1365, showing what it said was synergistic antitumor activity in combination with carboplatin in models of ovarian cancer. The results provide a mechanistic rationale for the ongoing investigation of the combination in a phase I trial, the company said. The company showcased additional preclinical data on its oral CDK7 inhibitors, demonstrating 200- to 1,200-fold greater selectivity for CDK7 over other members of the CDK family and antiproliferative activity in triple-negative breast cancer and ovarian cancer cell lines. The data helped support the selection of the company's next development candidate, SY-5609, which is advancing toward IND-enabling studies. The data were presented at the EORTC-NCI-AACR Molecular Targets and Cancer Therapeutics Symposium in Dublin.
Tempest Therapeutics Inc., of San Francisco, said significant reductions in tumor growth and stimulation of durable antitumor immunity seen in a preclinical test of its small-molecule inhibitor of peroxisome proliferator activated receptor alpha TPST-1120 support moving it into clinical studies in patients with advanced solid tumors in early 2019. It presented the data in a poster at the Society for Immunotherapy of Cancer annual meeting in Washington.
Vernalis Research, a Cambridge, U.K.-based subsidiary of Ligand Pharmaceuticals Inc., said it earned multiple milestone payments under its drug discovery collaboration with Tokyo-based Daiichi Sankyo Co. Ltd. The research, which began in December 2017, is using Vernalis' fragment and structure-based drug discovery platform for work on two undisclosed oncology targets. A final success payment has been earned for one target and the research collaboration continues for the second target, Vernalis said. The values of the payments were not disclosed.
Wugen Inc., of St. Louis, negotiated a license agreement with Washington University in St. Louis for intellectual property and technology related to CAR T therapies. The agreement gives the company exclusive rights to technology to be used in the development of CAR T approaches for blood cancer therapies.