CELL AND GENE THERAPY, INDUSTRIAL IMPACT
Rocket Pharmaceuticals, Inc | February 09, 2023
On February 7, 2023, Rocket Pharmaceuticals Inc., a renowned late-stage biotech firm advancing an integrated and sustainable pipeline of genetic therapies for rare disorders with high unmet needs, announced that FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation to RP-A501, the company's investigational adeno-associated virus (AAV)-based gene therapy for Danon Disease, a life-threatening genetic cardiac disease with no disease-altering therapies available. RMAT status was granted based on favorable safety and effectiveness findings from the Phase 1 RP-A501 clinical trial and will provide further added benefits of intensive FDA guidance and accelerated evaluation throughout the program's development.
RMAT designation, the same as Breakthrough Therapy designation, offers the advantages of intensive FDA guidance on efficient drug development, such as the ability for early FDA interactions to discuss intermediate or surrogate endpoints, potential priority review of the Biologics License Application (BLA), possible ways to support accelerated approval and satisfy post-approval requirements, and other possibilities to expedite development and assessment. Rocket's RP-A501 program also has Orphan Drug (US) and Rare Pediatric designations.
The Phase 2 pivotal trial is scheduled to begin in the second quarter of 2023. As disclosed previously, the company plans to conduct a single-arm, open-label trial with a biomarker-based composite endpoint and a natural history comparator.
The Phase 1 program's findings provide one of the most extensive investigational gene therapy datasets for any cardiac disease. In patients with Danon Disease treated in the Phase 1 study, RP-A501 was generally well tolerated, with indications of restored expression of the deficient LAMP2 protein and durable improvement or stability of clinical parameters.
Data show consistent and robust improvements in a variety of parameters, including protein expression, decreased autophagic vacuoles, troponin, brain natriuretic peptide (BNP), left ventricular (LV) mass and thickness, and enhanced NYHA class and Kansas City Cardiomyopathy Questionnaire (KCCQ/Quality of Life) measurements. Notably, these BNP and NYHA class improvements and stability contrast sharply with BNP rise and NYHA class worsening found in a representative sample of pediatric and adolescent natural history patients. Overall, the results indicate improvements and normalization in a variety of quantifiable parameters used by cardiologists in clinical practice to foster risk assessment and treatment decisions.
About Rocket Pharmaceuticals, Inc
Founded in 2015, Rocket Pharmaceuticals is a fully integrated clinical-stage firm developing an integrated and sustainable pipeline of investigational genetic therapeutics that have the potential to address the underlying causes of complex rare childhood diseases. Through its platform-agnostic approach, it can build the optimal therapy for each indication, possibly generating revolutionary options for patients suffering from rare genetic disorders. The company has a team of highly productive experts and is increasingly being recognized as a leading biotechnology innovator.
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CELL AND GENE THERAPY, INDUSTRIAL IMPACT
Evaxion Biotech A/S | January 23, 2023
Evaxion Biotech A/S, a leading clinical-stage biotechnology firm focused on the development of AI-driven immunotherapies, recently announced that FDA has granted fast-track designation for the Company's customized cancer therapy, EVX-01, in conjunction with KEYTRUDA®.
In December 2022, Evaxion gained FDA clearance to proceed with its phase 2b clinical test, where patients with metastatic melanoma are given EVX-01 in combination with KEYTRUDA®. In addition, Evaxion obtained fast-track designation for the vaccine candidate on January 17, 2023. The fast track is developed to expedite FDA's assessment of innovative, novel medications with the potential to meet an unfulfilled medical need.
EVX-01, a peptide-based immunotherapy for cancer, is Evaxion's most advanced clinical asset. The program generates a unique medicine for each patient based on gene analysis of their malignancies and matching with their immune system. The Company's proprietary AI platform, PIONEER, helps facilitate this process.
Per Norlén, CEO at Evaxion, commented," We are extremely pleased that our cancer vaccine candidate EVX-01 has received the FDA fast track designation, as it enables a potentially faster approval of the vaccine. This is first and foremost to the benefit of the patients. And it is a great validation of our AI platform, PIONEER, and our drug development candidate."
(Source – Globe Newswire)
The ongoing Phase 2b clinical trial is being done at sites in Europe, the United States, and Australia. It is conducted in partnership with Merck, which supplies its PD-1 inhibitor KEYTRUDA®. The trial was launched in Australia when the first patient was enrolled in September 2022.
About Evaxion Biotech A/S
Evaxion is a clinical-stage AI-immunology™ platform company using AI, engineering expertise and drug development knowledge to identify and develop novel immunotherapies for the treatment of various cancers, bacterial diseases and viral infections. It has utilized artificial intelligence to build a diverse pipeline of candidate immunotherapies that target two of the most critical unmet medical needs in the world, cancer and infectious diseases. Its three proprietary AI platforms include PIONEER, EDEN and RAVEN.
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MEDTECH, INDUSTRIAL IMPACT
QIAGEN | January 06, 2023
QIAGEN recently announced an exclusive strategic partnership with Helix, a California population genomics leader, to advance companion diagnostics for hereditary diseases.
The requirement for companion diagnostic devices and tests capable of detecting clinically relevant genetic abnormalities grows along with the accelerated development of precision medicines. These tests helps in making decisions by showing patients that are most likely to benefit from a specific therapeutic product or be at risk. Although companion diagnostics that use whole exome sequencing have primarily been used in oncology so far, they are widely believed to have a great deal of potential in hereditary disease areas like cardiovascular, metabolic, neurodegenerative, and auto-immune diseases.
According to the agreement, QIAGEN will be Helix’s exclusive marketing and contracting partner in the United States. This partnership will use the Helix® Laboratory Platform, the first whole exome sequencing platform, to get a de novo class II authorization from the U.S. Food and Drug Administration.
As a leader in precision medicine, QIAGEN has more than 30 master partnership agreements with multinational pharma and biotech firms to develop and commercialize companion diagnostic tests for their drug candidates. Its companion diagnostic offerings include next-generation sequencing (NGS), polymerase chain reaction (PCR), and digital polymerase chain reaction (dPCR), sample types ranging from liquid biopsy to tissue, and disease areas such as cancer and Parkinson's, including 11 FDA-approved PCR-based companion diagnostics.
Helix has developed an end-to-end platform that enables health systems, life sciences firms, and payers to enhance genomic research and expedite the integration of genomic data into clinical care. It has teamed with major health systems to enable population genomics programs across the United States. These programs significantly improve the identification and recruitment of patients for clinical trials in hereditary diseases such as Parkinson's and cardiovascular or inflammatory diseases such as non-alcoholic steatohepatitis (NASH).
About QIAGEN
QIAGEN is a leading provider of sample-to-insight solutions, enabling clients to get significant molecular insights from samples containing life's building elements. Its sample technologies are used to isolate and process DNA, RNA, and proteins from blood, tissue, and other materials. The company serves over 500,000 customers worldwide in molecular diagnostics (human healthcare) and life sciences (academia, pharma R&D, and industrial applications, primarily forensics).
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