Scientists will investigate whether cutting-edge technology such as base editing could be used to cure inherited heart muscle conditions after an international team co-led by Harvard Medical School won a research challenge. The $36 million Big Beat Challenge, run by the British Heart Foundation, is one of the largest non-commercial awards ever given and will focus on inherited heart muscle diseases known as genetic cardiomyopathies.
Although Pfizer Inc. has the only drugs approved in the U.S. to treat a rare, progressive heart disease, the U.S. Court of Appeals for the Second Circuit agreed this week with the Department of Health and Human Services, and a lower court, that Pfizer’s proposed copay assistance program for middle-income Americans covered by Medicare would violate the federal Anti-Kickback Statute – even if the company has no “corrupt” intent.
Barely a month after signing a €1 billion-plus deal with Menarini Group for cholesterol-lowering drug obicetrapib, Newamsterdam Pharma BV has struck a $235 million SPAC merger deal that will see the biotech list on Nasdaq in late 2022. The deal will finance phase III development and potential regulatory filings of the drug once dropped by Amgen Inc. as big pharma turned away from the cholesteryl ester transfer protein inhibitor drug class around five years ago.
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.
Stem cell therapy company Mesoblast Ltd. said that for patients with chronic heart failure with reduced ejection fraction, treatment with rexlemestrocel-L, its allogeneic product candidate, resulted in greater improvement in a prespecified analysis of left ventricular ejection fraction at 12 months relative to controls in the phase III DREAM-HF trial.
Several genetic studies in a range of model organisms have pointed to an important role for the B-cell lymphoma 2 (BCL2)-associated athanogene 3 (BAG3) gene in the maintenance of cardiac function.
Armed with compelling phase IIb data and with two phase III trials underway, Newamsterdam Pharma BV has sealed a European commercialization deal worth more than €1 billion (US$1.6 billion) with Menarini Group for its cholesterol lowering drug, obicetrapib.
From 20 years of research on metabolic change as a result of salt intake, Karen Duggan discovered in 2003 that a naturally occurring molecule in the human body, native vasoactive intestinal peptide (VIP), was capable of reversing fibrosis caused by hypertension and other chronic diseases such as diabetes. From that discovery, Vectus Biosystems Ltd. was founded, and the company has developed a new class of mimetic drug candidates and a drug library based on VIP.
Philadelphia’s Mineralys Therapeutics Inc. has secured a further $118 million to develop MLS-101, a potential hypertension drug in-licensed from Mitsubishi Tanabe Pharma Corp.
Luca Science Inc. has raised $30.3 million in an oversubscribed series B financing, enabling it to advance a mitochondria replacement therapy toward the clinic. The company is planning a phase Ib trial in myocardial infarction, in which 8 to 12 patients will receive injections of mitochondria immediately after having a heart attack.
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