The Alliance for Regenerative Medicine (ARM), an international advocacy organization representing the cell and gene therapy and broader advanced therapies sector, published a report outlining the latest trends in clinical trials with advanced therapy medicinal products (ATMPs) based in Europe, and its recommendations for how to improve its competitiveness compared to other global regions. The report, developed following a global analysis of all new clinical trials with cell and gene medicinal products initiated between January 2014 to June 2019 and via an online survey with ARM therapeutic developer member organizations, provides insights on clinical trials, including the number, type of technology, phase of development or approval process, specifically within Europe and also compared to other regions in the world. Among the findings were that, though the number of ATMP therapeutic developers based in Europe is about half of that based in North America, the number of clinical trials that have been initiated in Europe during the 2014-2018 period is only about a third of that in North America. During the same time period, the number of new clinical trials during the 2014-2018 increased by 32% globally, 36% in North America, 28% in Asia and less than 2% in Europe.
Arrowhead Pharmaceuticals Inc., of Pasadena, Calif., presented preclinical data at the North American Cystic Fibrosis Conference in Nashville, Tenn., showing that ARO-EnaC, an inhaled RNAi therapeutic, can accelerate mucociliary clearance in normal sheep and also preserve airway physiology in a sheep disease model of impaired mucociliary clearance. Animal models also indicated that ARO-ENaC can reduce ENaC activity in the lung while avoiding impact in the kidney.
Beam Therapeutics Inc., of Cambridge, Mass., signed a collaboration and license agreement with a newly formed company called Prime Medicine Inc. to research and develop a gene editing technology called prime editing, recently developed by one of Beam's co-founders, David Liu, and his group at the Broad Institute of Harvard and the Massachusetts Institute of Technology. Under the terms, Beam has the exclusive right to develop prime editing technology for the creation or correction of any single-base transition mutations, as well as for the treatment of sickle cell disease, both of which Beam is already pursuing with its base editing technology. Transition mutations (e.g. A to G, C to T) are the largest single class of disease-associated genetic mutations, and are also potentially treatable with base editing. Beam plans to evaluate prime editing technology for potential use in future programs.
Brickell Biotech Inc., of Boulder, Colo., initiated an arbitration proceeding related to the license agreement with Bodor Laboratories Inc., of Miami. The arbitration seeks a declaratory judgment that the purported termination of the deal by Bodor was invalid and unenforceable. Brickell concurrently filed with the U.S. District Court for the Southern District of Florida a motion to dismiss the complaint brought against Brickell by Bodor on Oct. 24, 2019.
Cycle Pharmaceuticals Ltd., of Cambridge, U.K., signed an agreement with Catalent Inc., of Somerset, N.J., to develop new formulations targeting rare disease patients. The agreement covers four products in rare metabolic and neurological disorders and each product will utilize Catalent's Zydis (olanzapine) oral disintegrating tablet (ODT) and Zydis Ultra technologies. The Zydis Ultra platform is the company's next-generation ODT technology and allows for increased drug load and better taste masking to be incorporated into its Zydis ODT dosage form.
Hemostemix Inc., of Calgary, Alberta, conducted an assessment of the status, timeline and financial requirements for the company to complete its current ACP-01 phase II study for critical limb ischemia. Hemostemix has enrolled 46 of the 95 patients. A third party clinical research organization has estimated that for the current trial design at the current enrollment rate, it would cost an additional $11.2 million to complete the trial and obtain top-line results in the second quarter of 2022. The company continues to search for strategic alternatives for financing, licensing or partnering to complete the study.
Laurent Pharmaceuticals Inc., of Montreal, said it reached an agreement to receive additional funding of up to $3 million from the Cystic Fibrosis Foundation to further support its APPLAUD phase II study evaluating lead compound LAU-7b in adult patients with cystic fibrosis (CF). That adds to the $5 million development award already made available by the foundation. LAU-7b is designed to act on the resolution phase of the inflammation, with the potential to treat chronic pulmonary inflammation that leads to irreversible lung damage in patients with CF.
Nanoviricides Inc., of Shelton, Conn., started dermal sensitization and ocular irritation studies that are required to support the IND application of NV-HHV-101. The company has contracted with MB Research Labs Inc., of Spinnerstown, Pa., to conduct the studies. On completion, the firm expects to file an IND with the FDA to advance the candidate into trials for shingles rash.
Orion Biotechnology Canada Ltd., of Ottawa, Ontario, said OB-002 was able to significantly reduce bone metastasis in a murine model of breast cancer. The study was conducted in collaboration with Proqinase GmbH, of Heidelberg, Germany. Mice randomized to receive OB-002 at a dose of 20 mg/kg demonstrated a significant reduction in day 20 primary tumor volume compared to placebo, and the mice randomized to receive 80 mg/kg had a significant reduction in spinal bone metastasis.
Helomics, a subsidiary of Minneapolis-based Predictive Oncology Inc., has begun sequencing tumor cases in its collaboration with UPMC Magee-Womens Hospital. The two are analyzing genomic and drug response profiles of women with ovarian cancer to build artificial intelligence-driven predictive models of therapy responses. The project is a retrospective look at about 400 ovarian cancer cases profiled for drug response by Helomics and outcome data from the hospital.
Ranbaxy Pharmaceuticals Canada Inc., of Ontario, joins the ranks of companies recalling prescription ranitidine products from the Canadian market. Health Canada is assessing the impurity N-nitrosodimethylamine (NDMA) detected in some ranitidine drugs. At Health Canada's request, companies marketing ranitidine products in Canada have stopped any further distribution until evidence is provided to demonstrate that they do not contain NDMA above acceptable levels.
Sanofi SA, of Paris, will pay $315 million to settle breach of contingent value rights (CVR) litigation involving claims regarding Sanofi's publicly traded CVRs issued in connection with its acquisition of Genzyme Corp. In the settlement, Sanofi and plaintiff UMB Bank agreed to fully and completely resolve the action without any admission of liability or wrongdoing. Sanofi acquired Genzyme in 2011 in a $20.1 billion deal. (See BioWorld, Feb. 17, 2011.)
Signpath Pharma Inc., of Salt Lake City, said it entered an agreement with Rain Therapeutics Inc., of Newark, Calif., which is licensing Signpath's CorreQT technology for use with Rain's lead compound, Tarlox (tarloxotinib), a pan-ErbB inhibitor, in development for cancer. CorreQT is a platform designed to eliminate cardiac arrhythmia (QT prolongation), a side effect of many drugs. The agreement provides for payments of up to $78 million upon achievement of development and commercial milestones, along with royalties on commercial sales of Tarlox products incorporating Signpath's technology.
Japan's Sumitomo Dainippon Pharma Co. Ltd. and Roivant Sciences Ltd., of Basel, Switzerland, signed their agreement to form a new company owned and supported by Sumitomo. The agreement includes Sumitomo receiving Roivant's ownership interests in five of its biopharmaceutical companies and access to Roivant's technology platforms. Sumitomo also takes an equity stake of more than 10% of shares outstanding in Roivant and will have options to acquire Roivant's ownership interests in up to six additional Vants by 2024. Exercise of the options by Sumitomo will trigger additional payments to Roivant. In addition, Sumitomo will enter separate strategic client relationships with Datavant and Alyvant to augment development and commercialization activities. Sumitomo paid $3 billion in the Roivant acquisition. (See BioWorld, Sept. 11, 2019.)
Tonix Pharmaceuticals Holding Corp., of New York, said it will effect a 1-for-10 reverse stock split, effective as of trading Nov. 1. The split, previously approved by the board, is intended to increase the per-share trading price to satisfy Nasdaq's $1 minimum bid requirement.