BioSpace Global Roundup, Oct. 15
AOP Orphan – AOP Orphan Pharmaceuticals took over the Viennese pharmaceutical company Amomed and the Luxembourgish health-tech company SciPharm. All jobs have been maintained and the workforce has grown from about 220 to 350. AOP anticipates new opportunities in Austria as a hub for research and business. The AOP Orphan Group manufactures 80% of its drugs in Europe and has more than 20 subsidiaries and representative offices across the whole of Europe as well as in the United Arab Emirates and Israel.
Priothera Limited – Ireland-based Priothera closed a Series A financing round of €30 million led by Fountain Healthcare Partners with participation from co-lead investor HealthCap and funds managed by Tekla Capital Management, LLC as well as EarlyBird Venture Capital. Priothera will use the funds to progress the clinical development of mocravimod, a modulator of sphingosine 1 phosphate (S1P) receptors, to enhance the curative potential of allogeneic hematopoietic stem cell transplantation (HSCT) for treating AML. Priothera expects to generate further randomized clinical data in high risk AML patients with this Series A funding round.
VBL Therapeutics – The European Patent Office granted a patent to Israel-based VBL’s proprietary investigational anti-MOSPD2 monoclonal antibodies to treat inflammatory conditions. VBL is currently advancing its lead anti-MOSPD2 candidate through IND-enabling studies. VBL has recently demonstrated ex-vivo activity of anti-MOSPD2 antibodies in patients with relapsing-remitting and progressive multiple sclerosis (MS), as well as in animal models of rheumatoid arthritis (RA), nonalcoholic steatohepatitis (NASH) and inflammatory bowel disease (IBD).
Inventiva – The U.S. Food and Drug Administration granted Breakthrough Therapy designation to France-based Inventiva’s experimental NASH treatment lanifibranor. Lanifibranor is believed to be the first drug candidate to be granted this status for the treatment of NASH since January 2015. The designation of lanifibranor as a Breakthrough Therapy for the treatment of NASH follows the publication in June 2020 of positive topline results from Inventiva’s NATIVE Phase IIb clinical trial with lanifibranor in NASH patients. In this 24-week clinical trial, lanifibranor met the primary endpoint with a statistically significant reduction of the Steatosis Activity Fibrosis score. Inventiva is expected to hold the end of NATIVE Phase IIb clinical trial meeting with the FDA and to receive regulatory feedback from the EMA during the fourth quarter of 2020 with the initiation of the Phase III clinical trial evaluating lanifibranor in NASH planned for the first half of 2021.
Abivax – France-based Abivax obtained a €15 million non-dilutive debt financing from Kreos Capital, with an additional €5 million to be decided before 2020 year-end. The loan is expected to extend Abivax’s cash runway until the second quarter of 2021, before any potential revenues from corporate partnering or any future additional funding, preferably non-dilutive.
Relief Therapeutics – Switzerland’s Relief Therapeutics announced topline results from a study of RLF-100 (aviptadil) in patients admitted to an intensive care unit (ICU) with critical COVID-19 and respiratory failure. Overall, 81% of RLF-100-treated patients survived beyond 60 days, compared to 17% of control patients. Patients treated with RLF-100 demonstrated a 9-fold increased probability of survival and recovery from respiratory failure, with a high degree of statistical significance.
Axol Bioscience – Based in the U.K., Axol Biosciences announced the expansion of its service offering, with the introduction of multi-electrode array (MEA) screening for human induced pluripotent stem cell (hiPSC)-derived cells. The new services have been developed to support pre-clinical research by optimizing hiPSC-derived cell culture, while providing high-quality electrophysiological data acquisition and analysis from physiologically relevant human cell models.
Iksuda Therapeutics – U.K.-based Iksuda executed its option to secure exclusive, worldwide rights to develop a novel class of tumor-activated prodrug payloads from the University of Goettingen. The highly potent and selective payload series represents a powerful new class within ADC development with novel protein alkylating cytotoxicity. Iksuda will drive onward development and commercialization, incorporating the tuneable payload series in its ADC pipeline and payload armory, to create best in class ADC therapeutics for nominated targets associated with hematological and solid tumors with high unmet need.
Apogenix – Germany’s Apogenix enrolled the first patient in the ASUNCTIS trial, an open-label Phase II study assessing the efficacy and safety of asunercept in patients with severe COVID-19 disease.
Prestige BioPharma – Singapore-based Prestige received a positive opinion from the European Medicines Agency Orphan Drug Commission (COMP) for its first-in-class anti-PAUF monoclonal antibody, PBP1510, for the treatment of pancreatic cancer. Prestige previously received orphan drug status for PBP1510 from the FDA as well as the Korean Ministry of Food and Drug Safety.
Nabriva Therapeutics – In a post hoc evaluation of Ireland-based Nabriva’s Lefamulin Evaluation Against Pneumonia (LEAP) 1 and LEAP 2 trials, the company said Xenleta (lefamulin) demonstrated consistently high efficacy and similar safety and tolerability profiles across all patient groups, including adults over 65 years of age who are at higher risk of morbidity and mortality from community-acquired bacterial pneumonia, the most common cause of infectious death in U.S. adults age 65 and older. Results will be presented at the virtual CHEST Annual Meeting this weekend.
Enlivex – Israel-based Enlivex said the Israeli Ministry of Health authorized the initiation of a proposed investigator-initiated Phase II clinical trial evaluating Allocetra in severe and critical COVID-19 patients. The trial plans to evaluate safety, tolerability, cytokine profile and efficacy parameters.
RedHill Biopharma – Also based in Israel, RedHill announced collaborations with two specialist pharmaceutical manufacturers in Europe and Canada that will ramp-up manufacturing of opaganib, for the treatment of severe COVID-19 pneumonia, to support potential emergency use applications and subsequent demand. Opaganib is a first-in-class, orally-administered, sphingosine kinase-2 (SK2) selective inhibitor with demonstrated dual anti-inflammatory and antiviral activity that targets a host cell component, potentially minimizing the likelihood of viral resistance.
Neogene Therapeutics – Netherlands-based Neogene and Twist Biosciences announced a broad strategic partnership that will leverage Neogene’s proprietary expertise in targeting tumor neo-antigens, mutated proteins found in cancer cells due to cancer-associated DNA mutations, together with Twist’s DNA synthesis platform and product lines to develop personalized chimeric antigen receptor (CAR) T cell therapies and T cell receptor (TCR) therapies for patients with cancer.
Secarna Pharmaceuticals – Germany-based Secarna Pharmaceuticals GmbH signed an agreement with Denali Therapeutics to leverage Secarna’s LNAplus ASO discovery platform and Denali’s blood-brain barrier technology to develop novel therapies directed against targets that play a central role in major neurodegenerative diseases.
Follicum – Sweden’s Follicum AB received a patent from the U.S. Patent and Trademark Office that covers peptides from the company's two peptide classes and their use in the treatment of diabetes and its complications. The patent is valid through 2038.
ERS Genomics – Ireland’s ERS Genomics signed an agreement with FASMAC Co., Ltd of Japan. Under the terms of the non-exclusive agreement FASMAC acquires the right to use CRISPR/Cas9 technology and commercialize tools and reagents associated with it.
Polyphor – Swiss pharma company Polyphor received a new funding from CARB-X (Combating Antibiotic-Resistant Bacteria Biopharmaceutical Accelerator) to support the development of the “thanatin derivatives program” belonging to Polyphor’s novel Outer Membrane Protein Targeting Antibiotic (OMPTA) class of antibiotics to potentially treat life-threatening infections caused by difficult-to-treat Gram-negative bacteria. CARB-X will provide Polyphor with initial funding of up to $2.62 million to complete the hit-to-lead stage and up to USD 15.82 million if certain project milestones are met. This funding is the second major support for Polyphor’s antibiotics program and follows a previous grant in 2019.
Talking Medicines – Scotland’s Talking Medicines will join Tech Nation’s Applied AI 2.0 Growth Programme. Talking Medicine uses advanced AI to provide pharmaceutical companies with real-time data intelligence.
Dolmatics – U.K.-based Dotmatics Ltd. has expanded its product portfolio to include Blueprint, a self-service, scientific visualization and analytics application for small molecule discovery. The introduction of Blueprint broadens the existing Dotmatics suite for chemistry design workflows, adding to the company’s comprehensive selection of web-based solutions. Blueprint seamlessly integrates into the existing Dotmatics suite of search and experiment capture capabilities which support the full drug discovery cycle, from compound design to analysis.
U-Diagnostics B.V. – Netherlands-based U-Diagnostics joined forces with Centogene to provide increased COVID-19 testing to communities throughout the Netherlands. This partnership comes as the Netherlands is currently facing a shortage of testing capacities among the rapidly increasing number of infections.
Amolyt Pharma – France’s Amolyt dosed the first patient in a Phase I study of AZP-3601, which is in development for the treatment of hypoparathyroidism, a rare and potentially debilitating endocrine disorder. AZP-3601 is a parathyroid hormone (PTH) analog that targets a specific configuration of the PTH receptor, thereby inducing a prolonged increase in blood calcium levels. The Phase 1 clinical program will evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics and preliminary efficacy of AZP-3601 following single and multiple ascending doses in healthy subjects as well as in patients with hypoparathyroidism.
Ori Biotech – London-based Ori Biotech closed a $30 million Series A financing round. The funds will be used to bring Ori’s innovative manufacturing platform to the market. The Ori platform delivers scalable solutions to flexibly address the critical clinical and commercial manufacturing needs of CGT developers.