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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MEDTECH, INDUSTRIAL IMPACT
Janux | December 29, 2022
Janux Therapeutics, Inc. is a clinical-stage biopharmaceutical company that uses its Tumor Activated T Cell Engager (TRACTr) and Tumor Activated Immunomodulator (TRACIr) platforms to develop a diverse pipeline of novel immunotherapies. On December 27, 2022, the company announced that it had submitted an investigational new drug (IND) application for JANX008 to U.S. Food and Drug Administration, an EGFR-TRACTr in (SCCHN).
The company is pleased to announce its second IND. It considers this a big step forward for its TRACTr platform, which has also helped many next-generation immunotherapies move forward.
JANX008 is made in a particular way to avoid cytokine release syndrome and the known harmful effects of EGFR in on-target healthy tissue. As a result, the company thinks it could be easier to treat EGFR-expressing cancers in many ways.
JANX008 is a double-masked TRACTr that conceals both the EGFR-binding and T-cell-specific binding domains (CD3). JANX008 exhibited minor cytokine release syndrome and healthy tissue toxicity in preclinical studies. Besides, it demonstrated superior safety and pharmacokinetic (PK) qualities compared to unmasked TCEs. These data and JANX008's strong manufacturability qualities justify its continued development as a potential therapy for solid tumors.
About theJanux
Janux Therapeutics is a clinical-stage business developing immunotherapies that target cancers without affecting healthy tissue. The company uses platform masking to generate innovative therapeutic candidates that address T-cell engager toxicity and efficacy limitations. It develops next-generation cancer therapeutics using TRACTR platform. Janux Therapeutics focuses on developing medically-validated T cell engagers (TCEs). Its pipeline includes PSMA, EGFR, and TOP2 efforts. All of its programs are IND-enabling or discovery.
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INDUSTRIAL IMPACT
Resilience | February 20, 2023
National Resilience, Inc. (Resilience), a manufacturing company with a technology-focused mission to improve access to complex medications, recently announced a 10-year biomanufacturing partnership with a top pharmaceutical company for its portfolio of services in process and analytical development, drug substance manufacturing, and drug product manufacturing. The extensive agreement covers prospective biologic, vaccine, nucleic acid, cell, and gene therapy manufacturing.
Resilience’s recently acquired facility in Cincinnati, Ohio, is its worldwide center of excellence to produce commercial medicinal products, and will deliver the first product as stipulated under the agreement. The development of complicated pharmaceuticals, such as cell and gene therapies, has not kept up with the rate of scientific advancements that are supporting them. Resilience brings together exceptional minds in science and engineering to bridge this gap with access to state-of-the-art technology.
Rahul Singhvi, Sc.D., Chief Executive Officer of Resilience, said, “The partnership announced today is a major milestone for Resilience.” He further added, “We are grateful to have this opportunity to work with our partner to deliver important, life-saving medicines to patients. We understand the trust placed with us to manufacture the medicine with the highest quality, regulatory compliance, and reliability. We are committed to maintaining a responsible manufacturing operation that exceeds the expectations of our partners and enables us to make a meaningful impact on global healthcare.”
(Source – Business Wire)
With the addition of this alliance to its expanding commercial portfolio, the company anticipates generating record revenues in 2023.
About Resilience
Resilience is a technology-focused biomanufacturing startup. The company intends to increase access to sophisticated medications. The firm is establishing a long-term network of cutting-edge, all-inclusive manufacturing solutions to guarantee that the therapies of today and the future may be produced efficiently, securely, and at scale. Resilience aims to continuously develop the science of biopharmaceutical manufacture and development, so their partners concentrate on the discoveries that enhance patients' lives and safeguard biopharmaceutical supply chains from future disruptions.
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