BioSpace Global Roundup, Jan. 16

Companies from across the globe provide updates to their business and pipeline.

Horizon Discovery – U.K.-based Horizon Discovery Group plc signed a collaboration with Mammoth Biosciences for that company’s novel CRISPR platform. The companies plan to deliver a new generation of genetically engineered CHO cells for the production of biotherapeutics such as therapeutic antibodies. Under the terms of the agreement, the companies will collaborate to optimize certain CRISPR tools to rapidly develop proprietary CHO cell lines. In these cell lines, selected genes are ‘knocked out’ to improve performance parameters such as increasing productivity or reducing naturally-occurring contaminants to facilitate purification.

NBE Therapeutics – Based in Switzerland, NBE Therapeutics AG closed a $22 million Series C financing round. The funding was led by existing shareholders Boehringer Ingelheim Venture Fund and the PPF Group and with participation from all private shareholders. The funds will enable NBE to advance the development of the lead asset NBE-002 through early clinical trials and to further strengthen the development team, management and board of the company. The first-in-human study with NBE-002 is expected to begin by mid-2020, focusing on triple-negative breast cancer and lung cancer, as well as including other solid cancer indications and lymphomas.

HalioDx -- Immuno-oncology diagnostic company HalioDx, which is based in France, announced that the Clinical Coding & Schedule Development (CCSD) Working Group has recognized the “utility and robustness” of its Immunoscore assay by granting it a diagnostic CCSD code following a review of clinical evidence. The assay is used to help post-surgical chemotherapy decisions for stage II and III colon cancer patients. Immunoscore is a risk-assessment tool in localized colon cancer that provides independent and superior prognostic value than the usual tumor risk parameters, and should be used as an adjunct to the TNM classification, the company said.

Hutchison China MediTech -- England’s Hutchison China MediTech Limited, better known as Chi-Med, initiated a Phase II study in China of surufatinib in combination with Tuoyi (toripalimab) in patients with advanced solid tumors. This follows the recent completion of the Phase I dose finding study and successful establishment of the Phase II combination dosing regimen for surufatinib and Tuoyi, the company said. This China Phase II clinical study is part of a global collaboration with Shanghai Junshi Biosciences Co. Ltd. to evaluate surufatinib, Chi-Med’s oral angio-immuno kinase inhibitor, in combination with Tuoyi, Junshi’s anti-programmed cell death protein 1 (“PD-1”) monoclonal antibody which was approved by China’s National Medical Products Administration in 2018. The study is designed to test the potential synergistic anti-tumor effects of the combination’s ability to simultaneously target multiple cell types and signaling pathways in the tumor microenvironment. It will explore multiple solid tumor patient populations. In December 2019, surufatinib was granted Priority Review status by the Center for Drug Evaluation of China’s NMPA.

ERS Genomics – Ireland-based ERS Genomics signed a license agreement with Daiichi Sankyo that provides the Japanese company with access to CRISPR/Cas9 genome editing technology intellectual property for use in its internal research and development. Under the terms of the license, Daiichi Sankyo is able to use CRISPR technology to support its R&D initiatives to address areas of unmet medical need.

apceth Biopharma – Germany’s apceth Biopharma GmbH, a subsidiary of Hitachi Chemical Co., Ltd., began the commercial manufacture of bluebird bio’s Zynteglo in Germany. Zynteglo has been conditionally approved in Europe as a gene therapy for patients 12 years and older with transfusion-dependent β-thalassemia (TDT) who do not have a β00 genotype, for whom hematopoietic stem cell (HSC) transplantation is appropriate but a human leukocyte antigen (HLA)-matched related HSC donor is not available.

Horizon Therapeutics – Horizon announced topline results from its MIRROR open-label study in which methotrexate with Krystexxa (pegloticase injection) provided an increased durability of response for people living with chronic gout refractory to conventional therapies – also known as uncontrolled gout. Trial data showed that 79% of the trial patients achieved a complete response, defined as the proportion of serum uric acid (sUA) responders at six months. Detailed results from the study will be presented at a future medical meeting.

Evonetix Ltd. – U.K.-based Evonetix partnered with imec to increase production of Evonetix’s proprietary microelectromechanical systems (MEMS)-based silicon chips, enabling the platform to be manufactured at a commercial scale. The novel silicon chip is a key component of Evonetix’s desktop DNA platform which, once fully developed, will facilitate and enable the rapidly growing field of synthetic biology, the company said. Under the terms of the collaboration, imec will work with Evonetix to scale up manufacturing of the MEMS technology on 8-inch silicon wafers, enabling Evonetix to supply customers in volume.

BioMed X – Germany’s BioMed X launched a new schizophrenia research program with Boehringer Ingelheim. The aim of the new research project is to investigate myelination deficits of the adolescent brain in connection with the development of schizophrenia. The program will specifically investigate the myelination deficits, which have been observed in the brains of schizophrenia patients. The innovative approach includes the development of in vitro and in vivo platforms to study oligodendrocyte development and myelination. The goal of the project is to identify novel extrinsic (neuronal) and intrinsic (oligodendrocytic) factors affecting myelination as potential new targets for the prevention of schizophrenia in high-risk individuals.

Mereo BioPharma – London-based Mereo BioPharma Group plc announced a license agreement with Oncologie to develop navicixizumab, an anti-DLL4/VEGF bispecific antibody. It is currently being evaluated in an ongoing Phase Ib study in combination with paclitaxel in patients with advanced heavily pretreated ovarian cancer. Navicixizumab is one of two product candidates Mereo acquired through its 2019 merger with OncoMed Pharmaceuticals, Inc. In October 2019, the U.S. Food and Drug Administration granted Fast Track designation to navicixizumab. Under the terms of the License Agreement, Oncologie will receive an exclusive worldwide license to develop and commercialize navicixizumab. Mereo will receive an upfront payment of $4 million with an additional payment of $2 million conditional on a CMC (Chemistry, Manufacturing and Controls) milestone. Oncologie will be responsible for all future research, development and commercialization of navicixizumab. Additionally, Mereo will be eligible to receive up to $300 million in future clinical, regulatory and commercial milestones.

Single Technologies – Sweden-based Single Technologies completed a heavily oversubscribed SEK 15 million convertible bond issue. The funds will be used to accelerate development of the company’s high throughput DNA sequencing platform Theta. Single Technologies is looking to deliver a paradigm shift in biomolecular analysis with Theta by removing the current limitations of imaging and microfluidics and ensuring true economy of scale in the generation of DNA sequencing data, the company said.

Sigrid Therapeutics – Sweden’s Sigrid Therapeutics published a preclinical study that showed the enzyme-blocking effects of mesoporous silica particles in mice benefits gut health. In the study reported in Nanomedicine, mice were fed high fat, high calorific diets, to induce weight gain, mixed with specially engineered MSPs. The results showed that MSPs reduced food efficiency by 33% leading to a lower weight gain, and a positive effect on the metabolic profile, as well as significant lower levels of adipose tissue formation and leptin, together with lower levels of circulating insulin.

ProBioGen AG – Germany’s ProBioGen and Lava Therapeutics B.V. from The Netherlands entered into a cell line development and manufacturing agreement. The company’s will focus on Lava’s novel bispecific antibody lead candidate, a γδ T-cell engaging bispecific antibody format.

Dermavant Sciences – Roivant subsidiary Dermavant, a dermatology-focused company, entered into a collaboration with Japan Tobacco Inc. for rights to develop, register and market tapinarof in Japan for the treatment of dermatological diseases and conditions, including psoriasis and atopic dermatitis.
Tapinarof is a potential first-in-class, once-daily topical therapeutic aryl hydrocarbon receptor modulating agent. The asset demonstrated positive results for both indications in mid-stage trials. If the late-stage study is good, Dermavant expects to seek regulatory approval in the United States in 2021.