Investors found in an 8-K filing by Intellia Therapeutics Inc. the news of one case of liver-enzyme elevation in the ongoing phase III Magnitude study with nexiguran ziclumeran (nex-z, NTLA-2001), and in reaction pushed shares of the firm (NASDAQ:NTLA) down to close May 29 at $7.45, a loss of $2.21, or 23%, after the stock traded as low as $6.90 during the day.
Developing a therapy for an ultra-rare condition has its challenges, including finding enough patients for clinical enrollment and convincing regulatory authorities that limited data prove the candidate is safe and effective. For that reason, Stealth Biotherapeutics Inc. has faced numerous roadblocks getting its mitochondria-targeting elamipretide across the finish line for Barth syndrome, a condition that affects about 230 to 250 males worldwide, including fewer than 150 in the U.S.
Atherosclerotic cardiovascular disease (ASCVD) remains a leading cause of mortality worldwide despite advances in managing classic risk factors and therapies lowering LDL-cholesterol. Inhibiting angiopoietin-like protein 4 (ANGPTL4), a key regulator of triglyceride metabolism, is emerging as a potential strategy to treat atherogenic dyslipidemia and lower ASCVD risk.
Boston Scientific Corp. reported the latest setback in its transcatheter aortic valve replacement (TAVR) aspirations with the news that it is officially discontinuing worldwide sales of its Acurate neo2 and Acurate Prime systems
Shockwave Medical Inc. is confident that its intravascular lithotripsy (IVL) system can continue to dominate the market for calcium modification even as other technologies emerge, chief medical officer Nick West told BioWorld.
EBR Systems Inc. raised AU$55.9 million (US$36 million) to commercialize its Wise cardiac resynchronization therapy system following U.S. FDA approval in April of the wireless, endocardial pacing system to stimulate the heart’s left ventricle.
Rocket Pharmaceuticals Inc. CEO Gaurav Shah said his firm is investigating how its gene therapy for Danon disease may have created an “unexpected and paradoxical” effect that led to problems for a phase II patient who ultimately died.
Frontera Therapeutics Inc. has developed FT-017, an adenovirus-associated vector(AAV)-based gene therapy that carries a human MYBPC3 optimized codon, for the treatment of hypertrophic cardiomyopathy (HCM).
Edwards Lifesciences Corp. released new economic and clinical data on severe aortic stenosis (AS) demonstrating intervening in a patient with the disease before symptoms develop can reduce costs to the health care system by $36,000. Data also shows earlier intervention can also lead to fewer follow-ups and hospitalizations for heart failure, after treatment.
RSS
