RESEARCH, BUSINESS INSIGHTS
Medidata Solutions | March 13, 2023
Medidata, a leading provider of innovative software solutions for clinical trials that streamline the clinical trial process, reduce time and cost, and accelerate the development of new treatments, recently announced that Allucent, a contract research organization (CRO) that brings innovation to biopharma firms, is expanding the global use of Medidata's Clinical Cloud to enhance its clinical trial operations offering.
This agreement extends Allucent's current usage of Medidata's technology, which includes eCOA, Rave EDC, RTSM, and Rave Imaging, supporting crucial development areas such as rare and orphan indications, oncology and cell and gene therapies. As part of the deal, Allucent's team achieved 100% Rave EDC certification, reinforcing their dedication to quality and providing clients with industry-leading technology.
Allucent is a sponsor of Medidata NEXT London and will give a presentation on data analytics for early-phase oncology trials. The presentation will cover how working with Medidata has helped them apply visualization techniques and strategies to extract the total value of information for all stakeholders.
Medidata is a Dassault Systèmes company poised to drive the digital transformation of life sciences in the age of personalized medicine with its 3DEXPERIENCE platform. This platform is the first end-to-end scientific and business solution, from research to commercialization.
Medidata's head of Global Sales, Janet Butler, commented, "Allucent is leveraging the power of the Medidata Clinical Cloud to solidify their position at the forefront of clinical technology." She further emphasized, "Their work with novel therapies requires agile, patient-centric solutions. We are proud to build on our eight-year collaboration to advance the digital transformation of life sciences and deliver clinical innovations to help reach their goals."
(Source – Business Wire)
About Medidata Solutions
Medidata Solutions is a New York-based global provider of cloud-based software solutions for clinical trials. Its platform, the Medidata Clinical Cloud, enables pharmaceutical, biotechnology, medical device companies, and academic research organizations to manage all aspects of clinical trials, including study design, data capture, management, and analysis. Medidata's platform can capture real-time data from multiple sources, including electronic health records and wearable devices, to monitor patients closely, identify potential safety issues earlier, and make informed decisions about trial design and treatment protocols. The platform also features advanced analytics tools like machine learning and artificial intelligence algorithms to help researchers analyze and interpret their data. In 2019, Dassault Systèmes, a global leader in 3D design software and simulation solutions, acquired Medidata, further enhancing its platform.
BioMarin Pharmaceutical Inc. | March 08, 2023
On March 7, 2023, BioMarin Pharmaceutical Inc., a global biotech company focused on improving lives via genetic discovery, announced that the US FDA has accepted its supplemental New Drug Application (sNDA) for VOXZOGO® (vosoritide) for injection to expand the treatment of achondroplasia in children under the age of 5.
Achondroplasia is a common form of disproportionate short stature. The FDA has set an action date of October 21, 2023, for the sNDA. The sNDA is supported by data from a Phase 2 double-blind, randomized, placebo-controlled clinical trial, which showed similar safety and efficacy profiles in children under five years of age compared to those aged five years and older.
The European Medicines Agency (EMA) also validated BioMarin's application for VOXZOGO in January to treat children under the age of 2. If approved, VOXZOGO could be prescribed for over 1,000 additional children from birth for achondroplasia.
VOXZOGO is the first EMA and FDA-approved treatment for achondroplasia with open bone growth plates. The treatment uses a new class of therapy, C-type natriuretic peptide (CNP) analog, which promotes bone growth by acting as a positive regulator of the signaling pathway downstream of the fibroblast growth factor receptor 3 gene (FGFR3).
The drug is approved in the EU, Brazil, and Australia for children aged two years and above with epiphyses (bone growth plates) and in Japan for children from birth who have achondroplasia with open growth plates. BioMarin has enrolled 250 children with achondroplasia in seven clinical studies in eight countries to evaluate the safety and efficacy of VOXZOGO.
About BioMarin Pharmaceutical Inc.
BioMarin is a leading biotechnology firm established in 1997 with a mission to transform people's lives by using genetic discovery. The company creates targeted therapies that address the underlying cause of genetic disorders to improve the quality of life for individuals with rare genetic diseases. It has developed eight first or best-in-class treatments and has a range of product candidates to address various genetic disorders using the same science-based approach. As BioMarin continues to create new and innovative solutions, it has the potential to impact the lives of even more people. The company is committed to prioritizing patients and their needs in its approach to drug development.
BUSINESS INSIGHTS, PHARMA TECH
Avacta | January 23, 2023
Avacta Group plc, a leading life sciences company focused on developing innovative, targeted oncology medicines and powerful diagnostics, recently announced that AVA6000 has maintained a very favorable safety profile in the fourth dose cohort of the ALS-6000-101 dose escalation phase 1 clinical trial. Furthermore, analysis of tumor samples acquired from six patients across multiple cohorts shows that doxorubicin is being released within the tumor tissue, supporting the pre|CISIONTM technology's tumor-targeting capability.
Patients in cohort 4 continue to tolerate AVA6000 well, with a significant decrease in the occurrence and severity of the commonly associated toxicities with the administration of traditional doxorubicin treatment. Nausea, vomiting, Alopecia, mucositis, myelosuppression, and cardiotoxicity are all among the typical toxicities. Notably, the typical drug-related cardiotoxicity of doxorubicin was not found even at the highest dosage levels in group 4, equivalent to more than double the standard dose of doxorubicin.
In order to confirm the release of the active chemotherapy, doxorubicin, in tumor tissue, several tumor biopsies acquired from patients in different cohorts have been analyzed. This analysis demonstrates that AVA6000 targets the release of doxorubicin to tumor tissue at therapeutic levels that are significantly higher than those detected in the bloodstream at the same time point.
To date, AVA6000 has been administered to 19 patients with advanced and/or metastatic solid tumors who were enrolled across four groups. Based on the very favorable safety profile of AVA6000 in the study thus far, the Safety Data Monitoring Committee (SDMC) has recommended that the study be extended to higher dose cohorts in order to identify a maximum tolerated dose (MTD) required to inform dosing levels for the phase 1b and future studies. The Company anticipates completing these additional cohorts within the first half of 2023.
Avacta is a healthcare group that promotes human health and well-being by creating new cancer treatments and powerful in vitro diagnostics. Its mission is to create the future of medicine by producing safe and effective medications as well as high-performance diagnostics using its unique Affimer® and pre|CISIONᵀᴹ platforms. By merging these two platforms, the Company is constructing a pipeline of innovative cancer therapeutics to fulfil its goal of developing successful treatments for all cancer patients, including those who do not react to current immunotherapies.