BUSINESS INSIGHTS, PHARMA TECH
KSQ Therapeutics | January 30, 2023
KSQ Therapeutics, a leading clinical-stage biotechnology company developing therapies to treat cancer and autoimmune diseases, recently announced that Ono Pharmaceutical (ONO) has acquired multiple research-stage DNA damage response (DDR) programs. The programs were identified using KSQ's CRISPRomics platform technology. All of the programs are new and have the potential to be first-in-class therapies.
ONO's Discovery & Research Executive Director/ Senior Executive Officer Toichi Takino commented, "ONO appreciates the ability of KSQ's CRISPRomics platform technology to discover novel therapeutic targets with high selectivity for cancer cells." He added, "Through this acquisition agreement, we expect that the programs acquired from KSQ will lead to the creation of innovative medicines that will help treat cancer patients."
(Source – Business Wire)
KSQ's Chief Executive Officer Qasim Rizvi commented, "This acquisition of multiple research-stage oncology programs by ONO is further validation of the power of our platform to identify novel oncology targets and develop potential first-in-class programs." He added, "This is an important transaction for KSQ, enabling us to focus on our other programs. We look forward to watching ONO's progress as they advance these programs toward clinical studies."
(Source – Business Wire)
As per the terms of the agreement, ONO will pay KSQ a double-digit million upfront payment, prospective near-term milestone payments with potential long-term value in the triple-digit millions, and net sales-based royalties.
About KSQ Therapeutics
Founded in 2015, KSQ Therapeutics is a clinical-stage firm using its CRISPRomics® discovery platform to systematically decode the genome and identify optimal gene targets for oncology and autoimmune disease. It utilizes the platform for innovative drug development across diverse therapeutic domains and is actively creating a proprietary pipeline of tumor- and immune-focused drug candidates. Headquartered in Cambridge, Massachusetts, KSQ was formed by functional genomics thought leaders and CRISPR screening technology pioneers.
RESEARCH, PHARMA TECH
Ionis Pharmaceuticals, Inc. | March 10, 2023
Ionis Pharmaceuticals, a leading RNA-targeted therapy firm, recently announced that the USFDA has accepted for review a new drug application (NDA) for eplontersen. Eplontersen is an investigational antisense medicine designed to reduce the production of transthyretin (TTR) protein and treat hereditary and non-hereditary forms of ATTR amyloidosis (ATTR).
The NDA has been given a Prescription Drug User Fee Act (PDUFA) action date of December 22, 2023. The FDA has declared that no review concerns were detected and made no new data requests. The FDA also stated that it does not intend to conduct a meeting of the advisory committee to discuss the application.
ATTR amyloidosis is a systemic disease caused by the accumulation of TTR protein in organs, leading to progressive organ damage and eventually death. Patients with hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN) experience ongoing debilitating nerve damage throughout their bodies, resulting in the progressive loss of motor function.
The NDA for eplontersen is based on results from the global Phase 3 NEURO-TTRansform study showcased at the International Symposium on Amyloidosis (ISA). In the study, eplontersen showcased a statistically significant and clinically meaningful change relative to the baseline for its co-primary and critical secondary endpoints compared to the external placebo group. In addition, Eplontersen also demonstrated a favorable safety and tolerability profile.
Ionis and AstraZeneca announced a strategic partnership to develop and commercialize eplontersen in December 2021. Eplontersen is being developed and commercialized jointly by both businesses in the United States and will be created and commercialized by AstraZeneca throughout the rest of the globe, except Latin America.
The FDA granted eplontersen Orphan Drug Designation in the United States in January 2022. Eplontersen is also being studied in the worldwide Phase 3 CARDIO-TTRansform trial for transthyretin amyloid cardiomyopathy (ATTR-CM), a systemic, progressive, and deadly disorder leading to progressive heart failure and death within 3 to 5 years after disease start.
About Ionis Pharmaceuticals, Inc.
Ionis Pharmaceuticals, Inc. is a leading biopharmaceutical company headquartered in Carlsbad, California. Its unique antisense technology platform allows them to develop RNA-targeted therapies that modify the production of disease-causing proteins, leading to the development of treatments for rare genetic disorders, cardiovascular diseases, and neurodegenerative diseases. The company's most significant achievement is developing and commercializing Spinraza, the first approved treatment for spinal muscular atrophy. Ionis Pharmaceuticals has also developed drugs for other rare diseases, including transthyretin amyloidosis, Huntington's disease, and myotonic dystrophy type 1.
BUSINESS INSIGHTS, PHARMACY MARKET
VERO Biotech | February 03, 2023
On February 2, 2023, VERO Biotech Inc., a commercial-stage biotechnology company focused on neonatal intensive care and the acute care hospital community, announced that FDA has approved its innovative second-generation GENOSYL® Inhaled Nitric Oxide (iNO) delivery system for use in the operating room with rebreathing anesthesia. The second generation GENOSYL® DS is now the first and only iNO delivery device approved for use in both rebreathing and non-rebreathing anesthesia procedures, enhancing patient care, saving money for the hospital, and minimizing waste anesthetic gas pollution.
GENOSYL® DS is the first tankless inhaled nitric oxide delivery system. Nitric oxide, when inhaled, dilates the pulmonary blood vessels and can improve oxygenation in neonates suffering from hypoxic respiratory failure and pulmonary hypertension. GENOSYL® DS, unlike tank-based systems, generates and delivers iNO at the bedside via a small disposable cassette. This helps eliminate the need for hospitals to manage large, cumbersome tanks while also aiding in the simplification of clinical workflow.
The following are the expected advantages of the second generation GENOSYL® DS
Ability to employ rebreathing anesthesia: Rebreathing allows reduced gas flows, resulting in less usage of expensive anesthetic drugs, thereby reducing hospital costs; enhanced patient comfort (by preserving patient body temperature and moisture).
Smoother care process: seamless iNO delivery from the ICU to surgery and post-operative care leads to process and workflow enhancements for the healthcare organization, thus lowering the hospital's overall cost.
Set and forget: It allows anesthesiologists to continue using rebreathing anesthesia, thereby preventing potentially dangerous, time-consuming and cumbersome workarounds.
Reduced ecological consequences of anesthesia delivery: less anesthetic is released into the environment.
It is important to note that FDA has only approved the second generation GENOSYL® DS for use with rebreathing anesthesia in the surgical suite. The third generation GENOSYL® DS, which was recently approved, has not been tested with rebreathing anesthesia. VERO Biotech is currently conducting similar validations and expects to have data available in the first quarter of 2023.
About VERO Biotech
Headquartered in Atlanta, GA, VERO Biotech is an emerging biotechnology firm that focuses on designing, developing, and commercializing next-generation inhaled nitric oxide (NO) delivery systems to tackle unmet medical needs in patients with cardiopulmonary conditions. The company's mission is to improve patients' lives by pioneering innovative technologies for inhaled NO delivery in acute care hospitals and beyond, wherever nitric oxide treatment is required. By focusing on the science, development, and commercialization of its innovations, it aims to exceed customer expectations regarding safety, efficacy, and growth.