BioSpace's Global Biopharma Roundup: Jan. 31

World Map of Pills

It’s no secret that the global biopharma and life sciences industry is large. Sometimes the immense size of the industry can lead fascinating news tidbits to fall through the cracks, particularly those from smaller companies scattered across the globe.

This week BioSpace will launch a new roundup of news from companies across the globe.

AIT Therapeutics -- Rehovot, Israel-based AIT Therapeutics, a company focused on developing inhaled Nitric Oxide (NO) for the treatment of patients with respiratory conditions, licensed the commercial rights for its novel cylinder free nitric oxide (NO) generator and phasic-flow delivery (AirNOvent) system to Circassia Pharmaceuticals plc. Steve Lisi, chairman and chief executive officer of AIT, said they look forward to working with Circassia to bring the product to market in the United States and China. Under terms of the deal, AIT will receive $7.35 million in an upfront cash payment, $3.15 million payable upon successful completion of an FDA pre-submission meeting expected to take place in February 2019, and more than $21 million in other milestone payments.

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Biofrontera AG -- Leverkusen, Germany-based Biofrontera posted full-year unaudited earnings of €21 million for 2018. The earnings represent revenue growth of approximately 76 percent compared to the previous year period, with pure product sales increasing by around 98 percent, the company said. Preliminary unaudited product sales in the U.S. are expected to be approximately €4.9 million, compared to EUR 6.3 million in the same period in 2017. Product sales in Europe will be around €6.0 million, compared to EUR 4.3 million in the same period last year, the company said.

Horizon Discovery -- Horizon Discovery, based in Cambridge, England and New Jersey-based Rutgers University formed an exclusive partnership to develop next-generation novel gene editing technology known as base editing. The technology potentially has applications in the development of new cell therapies and will augment Horizon’s research tools and services, the U.K.-based company said. Base editing is a novel technology platform for engineering DNA or genes in cells that has the potential to correct errors or mutations in the DNA by modifying genes using an enzyme. Compared with currently available gene editing methodologies such as CRISPR/Cas9, which creates “cuts” in the gene that can lead to adverse or negative effects, this new technology allows for more accurate gene editing while reducing unintended genomic changes. The technology will have a significant impact in enabling cell therapies to be progressed through clinical development and towards commercialization.

“Base editing is potentially transformative for all gene editing technologies with the potential to help target many diseases that to date have no treatment,” Horizon CEO Terry Pizzie said in a statement.

Abivax – Abivax, a Paris-based company focused on developing treatments for inflammatory and autoimmune diseases, published new data characterizing the novel mechanism of action of its lead drug candidate, ABX464 in Nature Scientific Reports. The research concludes that ABX464’s ability to selectively upregulate anti-inflammatory miR-124 and selectively splice viral RNA, but not endogenous cellular RNA, may have applicability for treatment of both inflammatory diseases and HIV infection, the company said.

The research, conducted in the cooperative ABIVAX-CNRS laboratory directed by Prof. Jamal Tazi, demonstrated that ABX464 enhances the splicing of HIV RNA in infected peripheral blood mononuclear cell (PBMCs) from healthy individuals, and enhances the expression and splicing of a single long noncoding RNA to generate the anti-inflammatory miR-124 in patients. ABX464 is a first-in-class small molecule for oral administration that has been tested in Phase IIa clinical trials for ulcerative colitis (UC) and HIV infection. In the proof-of-concept clinical trial in UC patients, ABX464 statistically significantly improved UC signs and symptoms on both clinical and endoscopic endpoints, achieving a clear and clinically meaningful magnitude of effect in a small exploratory study. Abivax is preparing for a Phase IIb clinical trial in ulcerative colitis patients, as well as Phase IIa proof-of-concept studies for two additional inflammatory indications, Crohn’s disease and rheumatoid arthritis.

Sphere Fluidics – Based in the U.K., Sphere Fluidics closed a $2 million funding round that will support sales of its Cyto-Mine Single Cell Analysis System. Sphere Fluidics’ Cyto-Mine is an automated platform which integrates single-cell screening, sorting, dispensing, imaging and clone verification. It can process up to 40 million cells per day, and it assesses and isolates those that produce a specific biologic to help streamline workflows and improve throughput in antibody discovery and cell line development. The Company will use the investment to expand operations and sales capabilities across the U.K. and U.S. Sphere intents to open a new West Coast Sales Office in the U.S.

Metrion Biosciences – Contract research organization Metrion Biosciences Limited, based in the U.K., entered into a collaboration agreement with London-based LifeArc, a medical research charity, on an ion channel drug discovery project in the field of neuroscience. The project aims to identify novel selective small molecule modulators of a specific ion channel target, which human patient gene association studies have identified as likely to be involved in neurological pathogenesis. LifeArc will have responsibility for all new chemical syntheses with Metrion conducting ion channel screening. Metrion will provide support for LifeArc’s medicinal chemistry target optimization via its extensive suite of drug discovery assays.

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