BioSpace Global Roundup, March 19
Abivax – France-based Abivax had a successful 2019 as it continues to recruit patients in the United States for a clinical trial assessing its ulcerative colitis treatment, ABX464, following approval of an Investigational New Drug Application by the U.S. Food and Drug Administration. After the promising results obtained in the Phase 2a induction study, Abivax presented data from the subsequent 12-month open-label extension study. The observations confirmed the preliminary positive results on the safety and tolerability of ABX464 along with the first evidence of its excellent long-term efficacy. Furthermore, the data showed that ABX464 maintained the overexpression of miR-124 (a critical factor of immunity and inflammation modulated by ABX464) during the 12-month study period. In addition to ulcerative colitis, ABX464 is being assessed for rheumatoid arthritis, Crohn’s disease, and other inflammatory conditions. Abivax is also developing a number of antiviral drugs for use in COVID-19, as well as Respiratory Syncytial Virus (RSV), Influenza and Dengue.
Haselmeier – Germany’s Haselmeier received a Master File number from the FDA for its D-Flex product platform, a new generation of injection pen systems for subcutaneous self-administration. A device master file provides the FDA with proprietary data about a material, component or manufacturing process. It enables the company that submits the information to the FDA to comply with regulatory requirements for trading in the U.S. while protecting its intellectual property from potential partners, competitors and customers. In addition to the D-Flex injection system, Haselmeier also presented its D-Flex Connect system to the FDA. D-Flex Connect comprises the D-Flex pen, a smart cap and an innovative, future-proof software platform.
OSE Immunotherapeutics – OSE and Servier announced an amendment to a two-step global licensing option agreement signed in 2016 for rights to interleukin-7 receptor (IL-7R) antagonist OSE-127 being assessed for treatment of ulcerative colitis and in Sjögren’s syndrome. The amendment modifies the provisions regarding the option exercise modalities and associated financial conditions. Based on the positive Phase I results with OSE-127, two independent Phase II clinical studies are expected to start in 2020: in Sjögren’s syndrome, under Servier sponsorship, and in ulcerative colitis, under OSE sponsorship. Under the current amendment, the second option provides a €5 million milestone payment upon enrollment of the first patient in the Phase IIa clinical study in Sjögren’s syndrome and a €15 million milestone payment upon completion of both Phase II clinical studies and exercise of the option, in priority upon successful completion of the Phase II in Sjögren’s syndrome.
Horama – France’s HORAMA SA, a company focused on gene therapy for the treatment of rare genetic diseases in ophthalmology, announced an exclusive licensing agreement with the Leiden University Medical Center (LUMC) for global rights to a gene therapy program to treat the Inherited Retinal Dystrophy associated with pathogenic CRB1 gene mutations, a rare but devastating ophthalmic condition leading to blindness. Under the agreement, HORAMA will receive an exclusive worldwide license to certain patent rights for the drug candidate, HORA-001. In return for these rights, LUMC will receive an undisclosed upfront payment, milestone payments and royalties on net sales of products. HORAMA shall be responsible to bring the gene therapy to market with the completion of the non-clinical and clinical studies. Based on current timelines, and subject to regulatory review, HORAMA expects to initiate a Phase I/II clinical study with HORA-001 in 2023.
XPhyto – Vancouver-based XPhyto Therapeutics Corp. is moving forward with the development of its cannabidiol-based Epilepsy product. In a statement released this week, XPhyto said over the past three months, Vektor Pharma TF GmbH, the company's wholly-owned German subsidiary, has completed the initial stage of product development and established a number of critical parameters necessary for an efficient and well-defined dissolvable oral CBD dosage form. The company is now finalizing the formulation and preparing for European-based clinical studies in later this year. The company's CBD-based Epilepsy treatment program is one of several dissolvable oral drug delivery initiatives for 2020. XPhyto is also planning to announce an update on its infectious disease programs, particularly as they relate to products relevant to the COVID-19 pandemic.
IMV – Nova Scotia-based IMV Inc. is advancing the clinical development of a DPX-based vaccine candidate against COVID-19. The goal of this development program will be to establish the clinical safety and immunogenicity of a vaccine candidate, DPX-COVID-19, based on the company’s DPX delivery technology and incorporating peptides targeting novel epitopes from the coronavirus strain. The company believes that this peptide-based approach, combined with the portability of the DPX platform, offers the potential for accelerated development and rapid, large-scale production of a vaccine.
QIAGEN – QIAGEN obtained approval in Germany for its newly developed QIAstat-Dx Respiratory SARS-CoV-2 Panel test to be sold as an in vitro diagnostic for the detection of SARS-CoV-2. At the same time, the company announced that the panel has received CE marking for Europe-wide sale as an in vitro diagnostic (IVD) test to detect SARS-CoV-2. As the first syndromic testing solution cleared for use in the coronavirus epidemic, the QIAstat-Dx test kit can differentiate the SARS-CoV-2 coronavirus from 21 other serious respiratory infections in patients who may have similar symptoms in a single testing run of about an hour, the company said. The QIAstat-Dx Respiratory SARS-CoV-2 Panel is a multiplexed nucleic acid test that evaluates samples such as nasopharyngeal swabs obtained from individuals suspected of respiratory tract infections. The newly approved panel includes assays targeting two genes used to detect the pathogen behind the disease, Severe acute respiratory syndrome-related coronavirus 2 (SARS-CoV-2), according to company data.