DIOS-203

Germany-based Cevec Pharmaceuticals GmbH signed a licensing agreement with Biogen for the use of its proprietary ELEVECTA Technology for the manufacturing of adeno-associated virus (AAV) vectors for gene therapy applications. Few details of the deal were disclosed in the announcement. The deal will provide Biogen the rights to use the technology across their portfolio of gene therapy products. Cevec will be eligible for milestone payments, including clinical and commercial milestones. Nicole Faust, chief executive officer of Cevec, touted the deal and said it strengthens the company’s relationship to Biogen. “Over the last several years we have seen a strong need across Pharma and Biotech companies to establish scalable and robust manufacturing technologies for AAV vectors. This agreement represents the next major step on our ELEVECTA journey. Biogen has been a great collaboration partner and we look forward to continuing to support them with our innovative technologies,” Faust said in a statement. Elsewhere across the globe: Theratechnologies – In a full-year fiscal report, Montreal-based Theratechnologies Inc. provided an update on its ongoing NASH program. This week, the company said the U.S. Food and Drug Administration gave it the green light to initiate a Phase III study of tesamorelin for the treatment of adults with Nonalcoholic Steatohepatitis (NASH) with liver fibrosis. However, before the Phase III program may begin, the FDA requested a meeting with Theratechnologies to discuss questions and comments received on certain aspects of the proposed trial design, to ensure alignment with the agency’s expectations with NASH trials. The proposed Phase III trial design will enroll participants with liver-biopsy confirmed NASH and stage 2 or 3 fibrosis. A second liver biopsy will be performed after 18 months. These data will form the basis for filing a supplemental Biologics License Application with the FDA to seek accelerated approval. Alcon – Switzerland-based Alcon launched AcrySof IQ VivityTM IOL (Vivity), the first and only non-diffractive extended depth of focus intraocular lens (IOL) in the U.S. Vivity is a first-of-its-kind, non-diffractive extended depth of focus IOL with Alcon’s proprietary non-diffractive X-WAVE technology, which stretches and shifts light without splitting it. This presbyopia-mitigating lens is available to patients undergoing cataract surgery. Cataracts are the most common cause of vision loss globally. Vivity received FDA approval in February 2020. Field Trip Health – Based in Toronto, Field Trip Health has been selected as a trial location for a MAPS-sponsored study on the safety, feasibility and preliminary outcomes of MDMA-assisted therapy to treat eating disorders, including Anorexia Nervosa, a mental health condition and eating disorder characterized by low weight, food restriction, fear of gaining weight and a strong desire to be thin. Subject to the site inspection, Field Trip’s Toronto location will be used as the trial site for the Health Canada-approved Anorexia Nervosa arm of the Eating Disorder Study. ProQR – Netherlands-based ProQR Therapeutics N.V. completed patient enrollment in the Phase II/III ILLUMINATE study of sepofarsen for the treatment of Leber Congenital Amaurosis 10 (LCA10) due to the p.Cys998X mutation in the CEP290 gene. Top-line results are expected in the first half of 2022. This trial is intended to support application for marketing approval of sepofarsen for patients with LCA10 due to the p.Cys998X mutation in the CEP290 gene. Sepofarsen has been granted orphan drug designation in the United States and the European Union and received fast-track designation and rare pediatric disease designation from the FDA as well as access to the PRIME scheme by the EMA. SIRION Biotech – Germany-based Sirion Biotech GmbH forged a deal with Cellectis that granted to that company non-exclusive right under its proprietary lentiviral transduction enhancer LentiBOOST. This product complements Cellectis’ portfolio of technologies involved in manufacturing allogeneic CAR T-cells. Under terms of the deal, Sirion is entitled to undisclosed upfront and milestone payments and is eligible to receive royalties on future product net sales plus license fees tied to commercial success. LentiBOOST is designed to improve lentiviral transduction of difficult cell types like hematopoietic stem cells, and in addition generates stable integration of large constructs generally needed to generate CAR-T cells. AM-Pharma – Based in the Netherlands, AM-Pharma B.V. announced the first patients with COVID-19 infection and sepsis-associated acute kidney injury (SA-AKI) have been enrolled in the company’s Phase III REVIVAL study. The study will assess an exploratory cohort to assess the safety, tolerability and clinical benefit of recombinant alkaline phosphatase. Patients with severe COVID-19 infection often present with acute severe inflammation and organ failure. Recent studies conducted in the U.S. demonstrated that up to 90% of the COVID-19 patients that received mechanical ventilation also suffered from AKI and that the development of AKI in these patients is associated with poor prognosis. RhoVac AB – The first patient in RhoVac’s U.K.-based Phase Ib prostate cancer study BraVac has been enrolled. The trial will assess whether or not the drug candidate RV001 can prevent or limit the development of advanced prostate cancer after curative intent treatment. Ultimovacs ASA – Norway-based Ultimovacs announced its participation in the Phase II DOVACC collaboration study with the Nordic Society of Gynaecological Oncology – Clinical Trial Unit (NSGO-CTU), the European Network of Gynaecological Oncological Trial Groups (ENGOT) and AstraZeneca. The Phase II clinical trial will evaluate Ultimovacs’ proprietary universal cancer vaccine, UV1, in combination with AstraZeneca’s durvalumab and olaparib in patients with relapsed ovarian cancer. UV1 is a peptide-based vaccine inducing a specific T cell response against the universal cancer antigen telomerase. Mogrify – U.K.-based Mogrify Limited expanded its research collaboration with MRC Laboratory of Molecular Biology. The project aims to develop novel protein expression systems by leveraging recent advances in direct cell reprogramming to help improve the production of proteins which are not produced sufficiently well in existing expression systems. The Mogrify technology will be applied to predict combinations of transcription factors to induce trans-differentiation from one cell type to another. The resulting target cell types could provide researchers with improved access to important proteins found in human cell types that are difficult to obtain and allow for more efficient protein production. Cybin – Toronto-based Cybin, a psychadelics company, entered into an agreement with HI, LLC dba Kernel to leverage its innovative technology, Kernel Flow (“Flow”), for its upcoming sponsored clinical work. Cybin expects the quantitative measurements enabled by Flow may improve the development, delivery and scaling of its psychedelic therapeutics. Valneva SE – Specialty vaccine company Valneva, which is based in France is in advanced discussions with the European Commission for the supply of up to 60 million doses of its COVID-19 vaccine, VLA2001. VLA2001 is currently the only inactivated vaccine candidate in clinical trials against COVID-19 in Europe. VLA2001 entered Phase I/II clinical studies in December and Valneva expects to report initial safety and immunogenicity data in April 2021. DiosCURE Therapeutics – Germany’s DiosCURE Therapeutics SE published a report in Science describing its core technology of multivalent single-chain antibodies with a unique molecular mode-of-action to inactivate SARS-CoV-2 virions. The company’s lead candidates, DIOS-202 and DIOS-203, are synergistic combinations of single-domain antibodies derived from camelid heavy chain-only antibodies. These next-generation immunotherapies against SARS-CoV-2 were designed based on detailed structural information of the antibodies’ interaction with its viral target protein and result from functional and evolutionary experiments. Liminial BioSciences – Canada’s Liminial announced its wholly-owned subsidiary Prometic Plasma Resources, has received FDA approval for its plasma collection center located in Amherst, NY. The center commenced operations and initiated source plasma collection in early 2020, and is now FDA licensed to collect and introduce into interstate commerce human source plasma for further manufacturing in the U.S. The approval follows the FDA’s pre-licensure inspection in September 2020. Oxgene – U.K.-based Oxgene introduced its self-labelling integral membrane (SLIM) protein discovery system. The novel method allows for the identification of antibodies against membrane proteins in their native configuration in mammalian cells, which could catalyze the discovery of new candidates for antibody-based therapies. Domainex Ltd – Domainex, an integrated drug discovery services company based in Switzerland, invested in the WAVEdelta instrument from Creoptix AG. WAVEdelta uses Grating-Coupled Interferometry to measure target binding on sensor chips, generating binding affinity and on/off rate kinetics in a high throughput manner. LSP – European life sciences investor LSP, based in Amsterdam, launched the LSP Dementia Fund, dedicated to fight neurodegenerative diseases. The LSP Dementia Fund focuses on all stages of dementia drug and medtech development and as such is the first of its kind in the world, the company said. The focus will be on finding novel treatments to battle the different neurodegenerative diseases, such as Alzheimer's disease, that cause dementia, as well as generating a strong financial return for investors. Aker BioMarine – Norway’s Aker BioMarine entered into an agreement with serial biotech entrepreneur Michael Davidson and his wholly owned company Medical Food Solutions Research, to develop pharmaceutical therapies for brain and eye health based on Aker BioMarine’s product LYSOVETA, LPC bound EPA/DHA. A new company will be established to develop the therapies and Aker BioMarine will hold a 50% stake in the company. Aker BioMarine will supply the company with all its LPC product needs. The company will seek to raise approximately $30 million from external investors to fund early clinical trials to obtain proof of concept for the therapies. Follicum – Sweden’s Follicum announced the European Patent Office approved the company’s patent EP 3 618 845 B1, which refers to the company’s novel peptides that are being developed to treat diabetes. The approval provides protection for the company's peptides and their use to treat diabetes in Europe until 2038.

News information is not all-inclusive and updates are published once a week on Tuesdays. Diagnostics Concern has been building about the new SARS-CoV-2 strain discovered last month in the U.K., and on Friday, the U.S. Food and Drug Administration (FDA) issued an official alert stating that it could trigger false negatives in COVID-19 tests. NeuTigers, an Artificial Intelligence (AI) spinout of Princeton University, launched CovidDeep. CovidDeep is a screening tool that can determine if a person is positive, negative or asymptomatic for COVID-19 using wearable devices. In clinical trials, it could detect COVID-19 with 90%+ accuracy in about two minutes. Testing Therapies, Antivirals and Vaccines By the end of January, Johnson & Johnson is expected to deliver preliminary trial results for its one-dose COVID-19 vaccine candidate. One of the open questions about the various vaccines against COVID-19 has been how long the protection offered will last. Although the jury is still out, the data is beginning to look promising. Stephane Bancel, chief executive officer of Moderna, speaking at a virtual event by Oddo BHF, a financial service group, said, “We believe there will be protection potentially for a couple of years.” China’s Sinovac Biotech reported that its COVID-19 vaccine, CoronaVac, had a 78% efficacy rate in a Phase III clinical trial in Brazil. Pfizer and BioNTech reported that their COVID-19 vaccine appears to be effective against a key mutation in new variants of the SARS-CoV-2 virus that causes COVID-19. A study published by the U.S. Centers for Disease Control and Prevention (CDC) cautioned that the COVID-19 vaccine seems to cause severe allergic reactions at a much higher rate than other vaccines, such as the flu vaccine. NeuroRx, Relief Therapeutics and Quantum Leap Healthcare Collaborative announced that NeuroRx and QLHC have signed a Clinical Trial Participation Agreement for the inclusion of Zyesami (RLF-100 aviptadil) in the I-SPY COVID-19 trial. I-SPY COVID-19 is assessing multiple drugs for critical COVID-19 hospitalized or ICU patients. DalCor Pharmaceuticals initiated the Phase II dal-COVID trial of dalcetrapib for COVID-19. Dalcetrapib is a potential anti-viral treatment that binds to the catalytic site of 3CLMpro of SARS-CoV-2 in vitro. The trial will be in patients with confirmed mild to moderate symptomatic COVID-19. The European Commission is in advanced discussions with France’s Valneva SE to secure up to 60 million doses of its inactivated virus COVID-19 vaccine, VLA2001. VLA2001 entered Phase I/II clinical studies in December and Valneva expects to report initial safety and immunogenicity data in April 2021. If clinical development is successful, an initial approval may be granted in the second half of 2021, the company said. Germany’s DiosCURE Therapeutics published a report in Science describing the company’s core technology of multivalent single-chain antibodies with a unique molecular mode-of-action to inactivate SARS-CoV-2 virions. The company’s lead candidates, DIOS-202 and DIOS-203, are synergistic combinations of single-domain antibodies derived from camelid heavy chain-only antibodies. Organizational Actions/Announcements LabCorp received a contract from the U.S. Centers for Disease Control and Prevention (CDC) to provide genomic sequencing of SARS-CoV-2 samples. The FDA issued a statement saying, “Until vaccine manufacturers have data and science supporting a change, we continue to strongly recommend that health care providers follow the FDA-authorized dosing schedule for each COVID-19 vaccine.” Gilead Sciences’ Veklury, which is better known by its scientific name remdesivir, is the only antiviral drug currently approved to treat COVID-19. Despite some controversy over its effectiveness, it is bolstering the company’s bottom line and Gilead recently updated its 2020 guidance to reflect that. Pfizer and BioNTech indicated they plan to produce an output of their COVID-19 vaccine this year by more than 50%. The plan is to produce 2 billion doses this year. Other Industry News The advancing rollout of currently approved coronavirus disease 2019 (COVID-19) vaccines provides hope to many that existing lockdowns and social restrictions will soon be lifted, but experts suggest a “return to normal” likely won’t occur until herd immunity is achieved. Speaking to the Reuters Next conference, Dale Fisher, chairman of the World Health Organization's (WHO) Outbreak Alert and Response Network, said that achievement of herd immunity in most countries is unlikely in 2021. Evidence continues to build for long-lasting immunity to COVID-19 following disease infection, with the publication of a new study showing a durable immune response as far as eight months after the initial onset of symptoms. The latest peer-reviewed study, published last week in Science, echoes several non-peer-reviewed studies published last year. In a recent paper published in Alzheimer’s and Dementia, a team of researchers is calling for more prospective studies that will look into the impact coronavirus disease 2019 (COVID-19) has on brain functioning, given the disease has been frequently associated with neurologic symptoms such as seizures and psychosis. SARS-CoV-2, the virus that causes COVID-19, has mutated into several strains. Most of them are not particularly different than the primary strains, but two, B.1.1.7, which was first observed in the U.K., and now 501.V2, which originated in South Africa, have virologists watching closely. On Monday, the San Diego Zoo announced that two gorillas tested positive for COVID-19.