Global Roundup: ProQR Licenses Axiomer RNA Editing Platform to Eli Lilly


Netherlands-based ProQR N.V. partnered with pharma giant Eli Lilly for use of the company’s proprietary Axiomer RNA editing platform to progress new drug targets toward clinical development and commercialization.

The platform technology enables the editing of single nucleotides in RNA in a highly targeted and specific manner. Andrew C. Adams, Eli Lilly’s vice president for New Therapeutic Modalities said the company intends to use the technology to unlock novel treatments to improve the lives of patients across a spectrum of diseases.

ProQR’s unique Axiomer platform technology enables the editing of single nucleotides in RNA in a highly targeted and specific manner. The technology is based on editing oligonucleotides, or EONs, which are designed to recruit endogenous Adenosine Deaminases Acting on RNA (ADAR) enzymes to a selected target adenosine in a disease-associated RNA. It is thought the technology has the potential to reverse more than 20,000 different G to A mutations known to cause disease.

The companies will collaborate to develop up to five targets. Under the terms of the agreement, ProQR will receive $50 million consisting of an upfront payment of $20 million, as well as an equity investment in its ordinary shares of $30 million. ProQR is also eligible to receive up to approximately $1.25 billion in milestones.

Elsewhere around the globe:

AM-Pharma BV – Netherlands-based AM-Pharma licensed ilofotase alfa, AM-Pharma’s proprietary recombinant human alkaline phosphatase, to Japan’s Kyowa Kirin Co., Ltd. Ilofotase alfa is currently being evaluated in the global pivotal REVIVAL Phase III clinical study as a potential first disease-altering treatment for sepsis-associated acute kidney injury (SA-AKI). In July, AM-Pharma announced the enrollment of the first patient in Japan as part of the ongoing REVIVAL trial. Under the terms of the agreement, AM-Pharma will receive €20 million in an upfront payment, and €30 million related to milestones prior to regulatory submission.

Gedea Biotech – Sweden’s Gedea was granted a patent from the U.S. Patent and Trademark Office for the treatment of fungal infections by the company’s compound glucono-δ-lactone. The granted patent confers protection for treatment of any fungal infection by glucono-δ-lactone.

Owlstone Medical– Based in the U.K., Owlstone raised $58 million in a Series D financing round. The company has raised a total of $150 million since its 2016 founding. The round was led by returning investor Horizons Ventures, which now takes a seat on Owlstone Medical’s Board, and is joined by other existing and high-profile new investors from Asia, the Middle East and the United States.

Babraham Bioscience – U.K.-based Babraham Bioscience Technologies, which manages and develops the Babraham Research Campus, has rebranded as Babraham Research Campus Ltd. The name change reflects the activity of the organization and is also in response to the refreshed and ambitious vision for the campus, which was announced in May this year, the company said in its announcement.

MEDIAN Technologies – France-based Median announced it achieved “outstanding performance” based on a large-scale lung cancer screening patient cohort using its iBiopsy CADx. The screening was conducted to characterize malignant vs benign lung nodules based on a deep learning algorithm on low-dose chest computed tomography. The screening showed a sensitivity of 95.2% for a specificity of 95.7%.

OSE Immunotherapeutics – OSE secured patent protection through 2035 for Tedopi, a combination of neoepitopes, protecting its administration schedule for inducing early T-lymphocyte memory response. Tedopi is used in the treatment of non-small cell lung cancer (NSCLC) in HLA-A2 positive patients.

Hutchmed – China’s Hutchmed announced it will present new analyses on ongoing studies of amdizalisib, a PI3Kδ inhibit, savolitinib, known as Orpathys in China, and fruquintinib, sold as Elunate in China, at the upcoming 2021 European Society for Medical Oncology meeting.

In addition to the presentations, Hutchmed also announced that it has initiated a Phase III study with Orpathys, a highly selective MET tyrosine kinase inhibitor combined with AstraZeneca’s epidermal growth factor receptor Tagrisso as a potential first-line treatment in certain non-small cell lung cancer (“NSCLC”) patients whose tumors harbor EGFR mutation and overexpress MET. The primary endpoint of the study is median progression free survival (PFS) as assessed by investigators. Other endpoints include median PFS, median overall survival (OS), objective response rate (ORR), duration of response (DoR), disease control rate (DCR), time to response (TTR), and safety. 

EnGeneIC Limited – Australia’s EnGeneIC developed a globally unique nanocellular technology that in pre-clinical animal studies has been shown to stimulate a broad and powerful anti-COVID-19 immune response. Additionally, the company said the technology neutralizes the known mutant COVID-19 viruses of concern, particularly the Delta variant. The company initiated a Phase I study in Melbourne, which will test for safety, as well as a plethora of anti-COVID-19 immune responses.

Nabriva Therapeutics – Ireland-based Nabriva announced its developmental partner Sumitomo Pharmaceuticals has received approval to market oral and intravenous formulations of Xenleta (lefamulin) for the treatment of community-acquired pneumonia (CAP) in adults in Taiwan. The two companies entered into an agreement in May for development and commercialization in the Greater China Region.

Xenikos B.V. – Netherlands-based Xenikos closed a €40 million in convertible debt consisting of two equal tranches of €20 million. The financing was led by Veloxis Pharmaceuticals, with participation from existing investors, Medicxi, RA Capital Management, Oost NL and Sanquinnovate. Xenikos will use the proceeds of the financing to initiate a registrational Phase III clinical trial in the U.S. and EU, designed to evaluate the efficacy and safety of T-Guard for the treatment of steroid-refractory acute graft-versus-host disease (SR-aGVHD) in patients following allogeneic stem cell transplantation versus ruxolitinib. T-Guard is designed to reset the body’s immune system safely and swiftly in life-threatening T cell-mediated conditions, including transplant-related rejection, acute solid-organ rejection and severe autoimmune disease.

Exscientia – AI-driven Exscientia, based in the U.K., announced a four-year collaboration with the Bill & Melinda Gates Foundation to develop small molecule therapeutics that tackle COVID-19 and also looks ahead to prepare for future pandemics. The collaboration will initially focus on developing broad-spectrum coronavirus agents, including accelerating Exscientia’s lead program, which targets the main protease (Mpro) of SARS-CoV-2. Subsequently, the collaboration will expand to develop therapeutics for influenza and Paramyxoviridae (the Nipah virus).

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