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.
About Avacta
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.
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BUSINESS INSIGHTS, PHARMA TECH
EyePoint Pharmaceuticals | February 28, 2023
On February 27, 2023, EyePoint Pharmaceuticals, Inc., a leading pharmaceutical firm committed to the development and commercialization of therapeutics to help improve the lives of patients with severe eye disorders and Rallybio Corporation, a clinical-stage biotechnology company dedicated to identifying and accelerating the development of life-transforming therapeutics for patients with severe and rare diseases, announced a research collaboration to explore the sustained delivery of Rallybio's complement component 5 (C5) inhibitor for the treatment of geographic atrophy, an advanced form of age-related macular degeneration that can cause irreversible vision loss. The research will use EyePoint's proprietary Durasert® technology, which is designed for sustained intraocular drug delivery.
Approximately one million individuals in the United States suffer from geographic atrophy, which is characterized by atrophic lesions in the central region of the macula. Geographic atrophy can cause irreversible vision loss and can lead to legal blindness. Currently, geographic atrophy treatments are limited in choice and duration of action, leaving a significant unmet need for safe and effective treatment options.
Under the terms of the research partnership, EyePoint and Rallybio will assess and explore the viability of utilizing Rallybio's C5 inhibitor in EyePoint's Durasert technology, with the intention to increase the collaboration upon mutual agreement following the evaluation.
EyePoint Pharmaceuticals' M.D., President and Chief Operating Officer Jay Duker commented, "We are excited to begin this research collaboration to explore the combination of Rallybio's C5 inhibitor with our bioerodible Durasert sustained release drug delivery technology to develop a potential long-acting treatment for geographic atrophy," He further emphasized, "Geographic atrophy associated with dry macular degeneration is a devastating eye disease, and the inhibition of complement is a proven treatment pathway. We hope to leverage our Durasert technology in this collaboration to create a potential best-in-class, long-acting intravitreal insert, which we believe could provide a more desirable option for patients given that the existing approved therapy is injected every one to two months."
(Source – Business Wire)
About EyePoint Pharmaceuticals
EyePoint Pharmaceuticals is a leading pharmaceutical company that develops and commercializes therapeutic products to help patients suffering from serious eye disorders. Headquartered in Watertown, Massachusetts, it is committed to using its innovative drug delivery platform to improve patients' quality of life. The company's pipeline utilizes its proprietary Durasert technology, which enables sustained intraocular drug delivery. Its current pipeline includes EYP-1901, a potential six-month intravitreal anti-VEGF treatment aimed at treating wet age-related macular degeneration. Durasert technology has been successfully used across four FDA-approved products, including YUTIQ®, which treats chronic non-infectious uveitis affecting the posterior segment of the eye.
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RESEARCH, PHARMA TECH
Asklepios BioPharmaceutical, Inc. (AskBio) | March 01, 2023
Asklepios BioPharmaceutical, Inc. (AskBio), a subsidiary of Bayer AG, recently announced that the European Commission (EC) has granted orphan drug designation to AB-1003 (also known as LION-101) for the treatment of limb-girdle muscular dystrophy (LGMD). AB-1003 is an investigational recombinant adeno-associated virus (AAV) based gene therapy that is being developed as a one-time intravenous infusion for patients with LGMD type 2I/R9.
The disease affects 4.5 people per million worldwide, with more than 5,000 people affected in the EU and the US. The EC decision was received through AskBio's EU-based subsidiary BrainVectis, following a positive opinion from the European Medicines Agency's (EMA) Committee for Orphan Medicinal Products (COMP)
AB-1003 is currently being investigated in a Phase 1/2 multicenter study in the US to evaluate its tolerability, safety, and efficacy in adult subjects with genotypically confirmed LGMD2I/R9.
The EC for medicinal products grants orphan drug designation developed to treat a life-threatening disease that affects no more than five people in 10,000 in the EU, provided there is no other adequate treatment option or the medicine can bring significant value to those affected by a specific condition. This designation will provide special incentives in the EU, including eligibility for protocol assistance and possible reductions or exemptions in certain regulatory fees. Additionally, if the medicine is approved for marketing, this designation will provide ten years of marketing exclusivity.
Limb-girdle muscular dystrophy (LGMD) is a group of diseases that cause progressive weakening and wasting of the muscles in the arms and legs. The severity, features of LGMD and age of onset vary among the many subtypes of the condition and are often inconsistent, even within the same family. Limb-Girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9) is a form of LGMD caused by mutations in the FKRP gene. Signs and symptoms of LGMD2I/R9 often appear in late childhood and may include difficulty walking and running. The symptoms intensify with time, eventually leading to considerable disability, and afflicted persons typically require a wheelchair for movement roughly 23-26 years after beginning. Unfortunately, there is presently no treatment that slows the advancement of the disease, and management is based on the indications and symptoms that each individual exhibits.
About Asklepios BioPharmaceutical, Inc. (AskBio)
AskBio is a leading, fully integrated gene therapy company dedicated to developing innovative gene therapeutics and life-saving medicines. With operations worldwide, its pipeline includes gene therapy candidates for various diseases, including the central nervous system, neuromuscular, metabolic, and cardiovascular diseases. AskBio's global headquarters are located in RTP, NC, with European headquarters in Edinburgh. In addition, the company operates AAV research centers in Philadelphia, PA; Columbus, OH; and Paris, and modern clinical and commercial facilities in San Sebastian, Spain.
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