BioSpace Global Roundup, July 11
uniQure – Netherlands-based UniQuire provided updated clinical data from an ongoing Phase IIb study of AMT-061, the company’s investigational AAV5-based gene therapy for the treatment of patients with severe and moderately severe hemophilia B. The 36 weeks of follow-up data show that all three patients have sustained increases in FIX levels after the one-time administration of AMT-061, with two of the three patients maintaining FIX activity in the normal range. Mean FIX activity for the three patients at 36 weeks after administration was 45% of normal, with the first patient achieving FIX activity of 54% of normal, the second patient achieving FIX activity of 30% of normal and the third patient achieving FIX activity of 51% of normal. The second and third patients had previously screen-failed and were excluded from another gene therapy study due to pre-existing neutralizing antibodies to a different AAV vector. Reported FIX activity was measured using an activated partial thromboplastin time (aPTT) assay performed at a central laboratory. No patient in the study has experienced a material loss of FIX activity, reported any bleeding events or required any infusions of FIX replacement therapy for bleed, the company said.
Horizon Discovery – U.K.-based Horizon Discovery today announced that Celyad, a clinical-stage biopharmaceutical company focused on the development of CAR-T cell-based therapies, received FDA Acceptance of Investigational New Drug Application for the autologous NKG2D based CAR-T cell therapy CYAD-02 that deploys Horizon’s optimized SMARTvector shRNA technology. The Phase I trial will be the first CAR-T cell therapy to employ the SMARTvector platform. Horizon will receive an undisclosed milestone payment for the successful IND filing. Celyad has been investigating the use of shRNAs to support the clinical development of its CAR-T cell platform. The FDA approved IND application involves CYAD-02, a next-generation CAR-T cell therapy in which shRNA is employed to suppress two genes. Celyad has pre-clinical data indicating that this improves in vivo engraftment and efficacy of CYAD-02.
Prestige BioPharma – Singapore-based Prestige BioPharma and Pharmapark LLC entered into an exclusive agreement for the commercialization of Prestige BioPharma´s Trastuzumab biosimilar in the Russian Federation. Prestige´s Trastuzumab is a mAb biosimilar to Roche’s Herceptin, which is used to treat patients with HER2-overexpressing breast cancer, HER2-overexpressing metastatic gastric or gastroesophageal junction adenocarcinoma. The product has recently been accepted for review at the European Medicines Agency. Terms of the deal between Prestige and Pharmapark were not disclosed.
In addition to its partnership with Pharmapark, Prestige also announced positive results from a Phase I clinical trial evaluating the pharmacokinetics, safety and immunogenicity of biosimilar candidate HD204 to Avastin (bevacizumab). Overall, the study demonstrated that HD204 is bioequivalent to both US-licensed Avastin and EU-licensed Avastin after a single IV infusion at 1mg/kg.
PhoreMost – Based in Cambridge, England, PhoreMost, which is focused on drugging disease targets considered undruggable entered into a collaboration with the Centre for Chemical Biology and Therapeutics (CCBT), Bangalore, India. The aim of the collaboration is the structural visualization of novel druggable sites across multiple targets, to rapidly advance new therapies. PhoreMost’s next-generation phenotypic screening platform SITESEEKER probes the entire proteome in a live cell environment for novel druggable targets. PhoreMost has built a pipeline of novel targets and early drug discovery programs using this platform, and the new collaboration will draw on CCBT’s structural and chemical biology expertise to progress selected targets within PhoreMost’s discovery portfolio.
Sol-Gel Technologies – Israel-based Sol-Gel shared positive results from its Phase III program evaluating Epsolay microencapsulated benzoyl peroxide cream, 5%, made with the Company’s proprietary microencapsulation technology, for the treatment of papulopustular rosacea. In two 12-week clinical studies, Epsolay demonstrated statistically significant improvement in both co-primary endpoints of the number of patients achieving “clear” or “almost clear” in the Investigator Global Assessment and absolute reduction from baseline in inflammatory lesion count. Epsolay is the first in a pipeline of dermatologic product candidates in development using Sol-Gel’s proprietary microencapsulation technology.
Lung Ambition Alliance – With a goal of eliminating lung cancer as a form of death, the International Association for the Study of Lung Cancer (IASLC), Guardant Health, the Global Lung Cancer Coalition (GLCC) and AstraZeneca came together to form the Lung Ambition Alliance. The first goal of the Alliance will be to double five-year survival for patients with lung cancer by 2025. The founding partners have identified three priorities: increasing screening and early diagnosis, delivering innovative medicine and improving the quality of care for people with lung cancer. The alliance will sponsor an international survey, led by Ipsos MORI, to identify local barriers that need priority focus. Results, expected in the fall of 2019, will be used to further refine and shape the priorities of the Lung Ambition Alliance.
AG Mednet – France-based AG Mednet and Boston-based Median Technologies renewed their partnership to use AG Mednet's global infrastructure for the collection, QC and delivery of medical images and ancillary data in support current and future clinical trials and programs. AG Mednet works with global CROs, providing technology solutions for clinical trial adjudication and imaging. Through the partnership, AG Mednet is delivering support to Median for its growing global portfolio of trials including its large programs in China.
Check-Cap – Israel-based Check-Cap announced positive final results from its recently completed post-CE approval study evaluating the clinical performance and safety of the C-Scan system, the only preparation-free capsule-based screening method for the prevention of colorectal cancer through the detection of precancerous polyps. The primary efficacy endpoint of the study was sensitivity, the ability to correctly identify patients with polyps, and specificity, the ability to correctly identify patients with lack of polyps of the C-Scan system compared to FIT in detecting subjects with polyps that are greater than 10 mm. The results demonstrate that C-Scan achieved a sensitivity of 76% in patients, while FIT achieved a sensitivity of 29%. C-Scan achieved a specificity of 82% in all patients, while FIT achieved a specificity of 96% in all patients. Check-Cap is currently conducting a pilot study in the U.S.
ArcherDX – Based in Israel, ArcherDX and Ayala Pharmaceuticals announced a strategic collaboration to develop companion diagnostic (CDx) tests targeting cancers bearing Notch activating mutations and fusions.
AblaCare – Medtech company AblaCare, based in Paris, raised €10 million in Series A financing from Sofinnova Partners. Proceeds from the financing will be used to advance the company’s clinical and regulatory programs in Europe and the United States. AblaCare’s treatment, a minimally invasive procedure called “ovarian rebalancing,” uses ablation to restore ovulation in women with infertility related to polycystic ovary syndrome (PCOS). This new technology transforms an older, invasive surgical method, called “ovarian drilling,” into a minimally invasive and much simpler office-based procedure.
Osivax – French company Osivax secured €8 million in a Series A financing round to fund further progress in the clinical development of its lead flu vaccine candidate and to support strategic corporate growth. The financing was led by Noshaq, previously known as Meusinvest, a Belgian investment firm, and joined by Anaxago, an innovative investment platform. The use of the funding will be to achieve clinical proof-of-concept for Osivax’s lead vaccine candidate, OVX836. The vaccine comprises a protein engineered to include the natural NP from an A-strain virus and the company’s proprietary oligoDOM technology, an auto-assembling structure with a positively-charged tail that creates a large protein nanoparticle with multiple copies of the antigen. In conjunction with the Series A closing, Osivax is opening a new subsidiary in Liege, Belgium, in the heart of the Walloon region, which is a center for innovation in the field of vaccines, and home to the BioWin health cluster.
Sernova Corp. – An interim review of a Phase I/II trial conducted by Canada-based Sernova showed initial safety, as well as key efficacy measures, including glucose-stimulated C-peptide, insulin production and additional clinically significant measures of glucose control in the first study patient with type-1 diabetes and severe hypoglycemia unawareness. The trial is examining Sernova's Cell Pouch transplanted with islet cells. The Cell Pouch was well-incorporated with vascularized tissue, which enabled successful transplant of the purified islets, the company said. Also, there was an 87.5% reduction in hypoglycemic events from baseline collected over a two-week monitoring period. No incidences of adverse events were determined to be related to the Cell Pouch implant, the company added.