Plasma pharmacodynamic biomarkers may be a reliable tool for biosimilarity assessment without having to rely on clinical trials, which are costly and time consuming.
For Verve Therapeutics Inc., the good news was the first human proof-of-concept data for a single-course in vivo base-editing treatment, presented at the American Heart Association Scientific Sessions over the weekend, showed treatment with VERVE-101 led to promising dose-dependent reductions in low-density lipoprotein cholesterol in patients with heterozygous familial hypercholesterolemia. The bad news was to be found in the safety data for the 10 patients treated to date, which included a myocardial infarction in one patient deemed potentially related to treatment.
The largest genetic analysis of abdominal aortic aneurysm (AAA) carried out to date has identified almost 100 new risk variants linked to the disorder. The study also highlighted a possible therapeutic target for this pathology that, at the moment, has no treatment. AAA affects 4% of people over 65 years of age in the U.S. and causes 41,000 deaths per year. The incidence is three to four times higher in men than in women.
Heterozygous familial hypercholesterolemia (HeFH) is caused by mutations in the LDL receptor gene, resulting in unusually high levels of low-density lipoprotein (LDL-C) in serum. Researchers from Epigenic Therapeutics Co. Ltd. presented the discovery of an epigenetic modulation therapeutic, EPI-001, for HeFH.
Heterozygous familial hypercholesterolemia (HeFH) patients treated with lerodalcibep achieved a 58.6% reduction in LDL-cholesterol at week 24 and a 65% reduction at the mean of weeks 22 and 24 in the phase III trial Liberate-HeFH. The developer, Cincinnati-based Lib Therapeutics Inc., was founded in 2015 when it licensed the technology from Bristol Myers Squibb Co. but is just now emerging from stealth.
Merck & Co. Inc. is pledging major resources on its prospect in the proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitor space with a phase III program that together will enroll about 17,000 subjects and test oral peptide MK-0616’s effect on tackling low-density lipoprotein cholesterol.
Obesity is tied to airway hyperresponsiveness and lung fibrosis, which may lead to patients with asthma and obesity poorly responding to asthma therapy. Blocking proprotein convertase subtilisin/kexin type 9 (PCSK9) is known to reduce serum cholesterol levels and to exert anti-inflammatory effects.
Verve Therapeutics Inc.’s heart disease candidate, VERVE-101, is the latest gene editing-based therapy to hit a snag at the FDA, which issued a clinical hold, delaying the start of phase I testing in the U.S. News of the hold, which followed preclinical presentations over the weekend at the American Heart Association 2022 meeting, sent shares of Verve (NASDAQ:VERV) falling 30.5% to close Nov. 7 at $21.75.
Verve Therapeutics Inc. has packed a lot into the past few weeks. The latest is a four-year research deal with Vertex Pharmaceuticals Inc. to find and develop an in vivo gene editing program for an undisclosed liver disease. Vertex will pick up the tab for program costs as Verve does the preclinical R&D. Verve is getting an up-front $60 million from Vertex, along with a $35 million equity investment.
Astrazeneca plc and Ionis Pharmaceuticals Inc. have unveiled some eye-catching figures from their cholesterol drug, AZD-8233, in a phase IIb trial, taking aim at the PCSK9 inhibitor market where Novartis AG, Sanofi SA/Regeneron Inc. and Amgen Inc. are vying for supremacy.
