BioSpace Global Roundup, Oct. 1

GENFIT– France’s GENFIT and LabCorp agreed to a five-year exclusive licensing agreement for GENFIT’s NIS4 technology to help identify patients with at-risk non-alcoholic steatohepatitis (NASH). As part of the agreement, LabCorp will develop and commercialize a blood-based molecular diagnostic test powered by NIS4 technology throughout the U.S. and Canada enabling widespread access to healthcare providers. The two companies initially partnered in 2019 when LabCorp began offering NIS4 technology to customers for use in clinical studies through Covance, its drug development business. NASH remains a highly underdiagnosed disease due to its asymptomatic nature and the limitations of existing diagnostic approaches. Liver biopsy, a highly invasive procedure, is the current clinical standard to formally diagnose NASH and to determine the stage of fibrosis.

Fundacion MEDINA – Spain’s Fundacion MEDINA and its partners, the Drugs for Neglected Diseases Initiative and Institut Pasteur Korea received a €995,000 grant from “la Caixa” Health Research 2020 program to identify novel natural products as potential effective treatments for leishmaniasis and Chagas disease. Leishmaniasis and Chagas disease are neglected tropical diseases that cause thousands of deaths worldwide each year. The joint project addresses this challenge by exploring the chemical diversity of MEDINA’s vast microbial natural product libraries, using the whole-cell phenotypic screening assays of Institut Pasteur Korea. In addition, a new image-based “parasite painting” assay with cutting-edge cell-imaging technology will be developed at Institut Pasteur Korea to identify new modes of action of novel compounds and to select the most promising compounds for further development.

Sapreme – Netherlands-based Sapreme announced positive preclinical data on its proprietary endosomal escape platform in two presentations at the 16th Annual Meeting of the Oligonucleotide Therapeutics Society. Sapreme is developing macromolecule delivery methods based on compounds that release therapeutic cargo from the endo-lysosome, improving access to intracellular targets and enhancing the therapeutic window for these therapeutics. Current macromolecular biologics rely on receptor-mediated endocytic uptake into the endosome and inefficient passive release from these vesicles into the cell to achieve therapeutic efficacy. The company’s presentations demonstrate that Sapreme’s SPT001 compound improves intracellular release of targeted antisense oligonucleotides (ASOs) and thereby also their therapeutic efficacy. In addition to ASOs, SPT001 has also been demonstrated to enhance delivery of other targeted payloads such as antibody-conjugated toxins.

Orchard Therapeutics -- Orchard Therapeutics receives EMA PRIME Designation for OTL-203 for the Treatment of mucopolysaccharidosis type I (MPS-I). OTL-203 is an investigational ex vivo autologous hematopoietic stem cell (HSC) gene therapy. Additional interim data was recently presented from the ongoing proof-of-concept clinical trial evaluating the safety and efficacy of OTL-203 in the severe Hurler subtype of MPS-I. Eight patients have been treated in the study, which completed enrollment in December 2019. As of July 2020, all patients had been followed for a minimum of six months, with the longest follow-up extending out to 24 months. Orchard expects to release full proof-of-concept results at one year and initiate a registrational study for OTL-203 in 2021.

Celyad Oncology – Belgium-based Celyad entered into a clinical trial collaboration with Merck to pair its investigational non-gene edited allogeneic CAR T candidate, CYAD-101, with Keytruda in refractory metastatic colorectal cancer (mCRC) patients with microsatellite stable (MSS) / mismatch-repair proficient (pMMR) disease.

Immunovia – Following positive results from a verification study, Sweden’s Immunovia will now conduct verification studies, the last step towards the launch of IMMray PanCan-d. The verification study analysis shows results in line with the previous commercial test model study (CTMS), which paves the way for the final blinded validation study.

Kurma Partners – France-based Kurma Partners, a European venture capital company focused on the life sciences, closed its Kurma Diagnostics 2 (KDx2) fund at €50 million. This second fund will be devoted to molecular and digital diagnostic technologies. This first closing is higher than the €35 million raised by its predecessor, Kurma Diagnostics, in 2015.

Joonghun Pharmaceuticals – South Korea-based Joonghun Pharmaceutical acquired European CE mark for six of its Hyaluronic Acid Dermal Fillers from the Lorient line of cross-linked hyaluronic acid dermal fillers. Lorient is intended to be injected into the subcutaneous layer of the skin to temporarily improve facial wrinkles in adults.

LNC Therapeutics – French biotech LNC Therapeutics is rebranding itself as YSOPIA Bioscience. Since 2018, the company has been focusing on the development of a portfolio of innovative drugs derived from the microbiome using the unique properties of single strain bacteria. YSOPIA Bioscience targets more specifically a group of keystone bacteria essential to human health: christensenella. The Company is also extending its field of application to several other chronic indications with a strong need for breakthrough therapeutic innovations. Following the recent approval by the FDA of its clinical trial application in obesity and associated metabolic diseases, this name change marks YSOPIA Bioscience’s development strategy and its transition to a clinical-stage biotechnology company.

MedinCell – France’s MedinCell dosed its first patient in a study to validate the safety of continuous administration of Ivermectin in oral form in COVID-19 patients. The objective of the mdc-TTG program is to protect from Covid-19 with a subcutaneous injection of a 1-month active treatment of Ivermectin, a molecule already widely used in other indications. Since the beginning of the pandemic, clues about its potential efficacy against Covid-19 have piled up. This prevention strategy called “pre-exposure prophylaxis” (PrEP) is similar to the one already used against HIV. People are protected for the duration of treatment. It has demonstrated its efficacy but also the need for long-acting injectable treatments, the only ones to guarantee the continuity of protection.

Avacta Group -- Avacta Group announced its BAMs assay to detect COVID-19 is now in use as a research kit, courtesy of partner company Adeptrix. The bead-assisted mass spectrometry (BAMS) assay uses the Affimer reagents specific to the SARS-CoV-2 virus to capture the virus spike protein from the sample for rapid detection by mass spectrometry. Up to one thousand samples per day can be analyzed by a single technician using BAMS.

Biocartis Group – Belgium’s Biocartis received a €1.2 million grant from VLAIO, the Flanders organization for Innovation & Entrepreneurship, for the development of the highly innovative GeneFusion Assay on its easy, rapid molecular and fully automated diagnostics platform Idylla. The Idylla GeneFusion Assay will include a highly multiplexed panel of established and emerging biomarkers, and will be the first FFPE RNA-based assay on the Idylla platform. The Idylla GeneFusion Assay is expected to bring results in approximately three hours, with less than two minutes hands-on time. The VLAIO grant is intended to support the development of the GeneFusion Assay on the Idylla platform, and to support related research studies on different sample and tumor tissue types, including on lung cancer tissue.

Relief Therapeutics – Switzerland’s Relief Therapeutics and NeuroRx, Inc. established supply chain agreements and ordered sufficient drug substance (RLF-100) to prepare to treat 1 million patients with COVID-19, should the pandemic continue. RLF-100 is FDA-approved phase IIb/III clinical trials for the treatment of critical COVID-19 in the US. A readout by the study’s Data Monitoring Committee is expected within the next month. European trials with RLF-100 are in preparation and are scheduled to start in Q1 2021.

Iterum Therapeutics – Iterum Therapeutics, which is based in Dublin and focused on developing next generation oral and IV antibiotics to treat infections caused by multi-drug resistant pathogens in both community and hospital settings, announced that, based on discussions at a pre-NDA meeting with the FDA plans to proceed with an NDA submission for sulopenem etzadroxil/probenecid, a bilayer tablet, for the treatment of uncomplicated urinary tract infections (uUTI) in patients with a quinolone-resistant pathogen.

IntraBio Inc. – U.K.-based IntraBio announced positive data from its multinational clinical trial of IB1001 for the treatment of Niemann-Pick disease Type C (NPC). IB1001 demonstrated a statistically significant and clinically meaningful improvement in symptoms, functioning, and quality of life in both primary and topline secondary endpoints for both pediatric and adult patients with NPC. The trial met its primary endpoint, the Clinical Impression of Change in Severity (CI-CS), which was assessed by blinded, centralized raters. The trial also met topline secondary endpoints, including the Scale for the Assessment and Rating of Ataxia (SARA) and the Investigators’ Clinical Global Impression of Change (CGI-C) assessment. IB1001 was observed to be safe and well-tolerated, with no drug-related serious adverse events.

Ultimovacs – Norway’s Ultimovacs announced positive topline results from the first cohort of 20 patients in its ongoing US-based Phase I clinical trial evaluating the company’s lead candidate, UV1, in combination with Keytruda as a first line treatment in patients with metastatic malignant melanoma. The results confirm achievement of the primary endpoints of safety and tolerability and indicate initial signs of clinical response. As per the cut-off date of Sept. 30, every patient in the first cohort reached at least 12-months of follow-up post treatment with UV1 and Keytruda. At the one-year landmark, the overall survival rate was 85%. Median Progression-Free Survival was not reached at 12 months, indicating that more than half of the participating patients did not demonstrate disease progression.