BUSINESS INSIGHTS, PHARMA TECH
Northstar and Inhibrx | January 09, 2023
NorthStar Medical Radioisotopes, LLC, announced a collaboration with Inhibrx, Inc., to develop and produce new radiopharmaceuticals for cancer treatment.
Inhibrx will develop a new generation of targeted alpha treatments utilizing its improved single-domain antibodies (sdAbs). With a long history of sdAb innovation, it has created a library of sdAbs targeting tumor-associated antigens. Its high binding affinity and specificity for tumor cells or antigen-expressing cells within the tumor microenvironment make sdAbs an ideal biological targeting agent for radioisotope delivery. In addition, alpha-emitting radioisotopes have a small range of activity and high energy transfer, allowing for the targeted destruction of cancer cells with minimum damage to adjacent healthy tissue.
NorthStar is expanding its position as an industry leader in the rapidly-growing field of therapeutic radioisotopes, and it is now prepared to become the first commercial-scale manufacturer of non-carrier added (n.c.a.) actinium-225 (Ac-225) and copper-67 (Cu-67).
Under the terms of this agreement, NorthStar will provide the supply of Ac-225 and access to its comprehensive radiopharmaceutical contract development and manufacturing organization (CDMO) services to assist the development of a predetermined number of Inhibrx's novel biologic medicines. NorthStar will also prepare Inhibrx patient dosages for clinical studies and, upon approval, may manufacture and supply radionuclides for its commercial use.
About NorthStar Medical Radioisotopes, LLC
NorthStar Medical Radioisotopes is a commercial-stage nuclear medicine company with a mission to provide patients with global access to game-changing radiopharmaceuticals. The company uses ecologically preferable technologies to create, manufacture, and distribute diagnostic imaging and therapeutic radiopharmaceuticals. It is the only commercial U.S. producer of molybdenum-99, an essential diagnostic imaging radioisotope (Mo-99). The FDA-approved RadioGenix® System (technetium Tc 99m generator) uses US-manufactured, non-uranium-based Mo-99 as the source material to generate technetium-99m (Tc-99m), which is used to assess heart disease and cancer.
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, 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.