Shares of Esperion Therapeutics Inc. (NASDAQ:ESPR) sank 22%, or $1.43, to trade midday at $4.92 on full results from the phase III outcomes trial called Cholesterol Lowering via Bempedoic Acid, an ACL-Inhibiting Regimen, known by the rough acronym CLEAR. The drug, branded Nexletol and approved by the U.S. FDA in February 2020, significantly reduced major adverse cardiovascular (CV) events – MACE-4 and MACE-3 – by 13% (p=0.004) and 15% (p=0.006), respectively. CLEAR data made public March 4 also show that Nexletol significantly reduced the risk of myocardial infarction by 23% (p=0.002) and coronary revascularization by 19% (p=0.001). The study enrolled almost 14,000 patients with or at risk for CV who were unable to maximize or tolerate a statin.

Bridgebio stock surges on phase II achondroplasia data 

Positive results from Bridgebio Pharma Inc.’s phase II study of infigratinib in children with achondroplasia, a genetic disease that inhibits bone length and leads to short stature, prompted the company stock to surge. Participants receiving the highest dosage, which was the fifth cohort getting 0.25 mg/kg daily, saw a 3.03-centimeter, about 1.19-inch, increase in their height annually, which produced a “p” value of 0.0022.The results for the oral, small-molecule FGFR3 inhibitor prompted Bridgebio to enroll children for a pivotal phase III study of the treatment. Bridgebio’s stock (NASDAQ:BBIO) had risen a sharp 55% at midday to about $17 per share.

AAAS 2023: Understanding Long COVID’s neurological effects in Recover program

The U.S. Recover program, set up in July 2022 to identify the causes of Long COVID, find biomarkers of disease and discover new therapeutic targets, is now preparing to move to its next phase and begin testing potential treatments in a multi-arm, randomized, placebo-controlled trial. But with 200 different symptoms, and limited understanding of relevant system-level pathological targets, there are significant hurdles to be overcome.

Developers advancing CD47 prospects as discoveries continue to roll out

Findings published in Nature showing that dual blockage of PD-L1 and CD47 can boost the therapeutic effects of oxaliplatin chemotherapy as well as the FOLFOX regimen – in a CT-26 mice tumor model, anyway – served to highlight CD47, a target that has spurred added efforts of late. CD47 is “experiencing a resurgence of interest, development and success,” Wainwright analyst Joseph Pantginis noted in a Feb. 13 report. A major force in the space is Gilead Sciences Inc., but others are coming on strong, too.

Newco news: ‘Biology no one else will have seen;’ startup Relation seeks new genetic targets

Newco Relation Therapeutics Ltd. is showing its colors after raising $25 million in a seed round to work on integrating single cell transcriptomics, functional genomics and machine learning – and cut through previously undecipherable combinatorial space – to find and validate drug targets in the non-coding genome. Last week saw the opening of the company’s “lab in a loop” facility, where outputs of wet biology – for example, gene expression profiles of bone cells extracted from femoral heads removed during hip replacement surgery – will be fed directly into Relation’s machine learning engine.

Also in the news

Achieve, Adagene, Albireo, Applied Molecular Transport, Aptorum, Aqilion, Assertio, Ayala, Bellerophon, Bristol Myers Squibb, Cardiff, Carisma, Catalent, Congruence, Corbus, Diamedica, Edgewise, Exelixis, Eyebiotech, Harrow, Hepion, Indivior, Ipsen, Kinimmune, Krystal, Mediwound, Neurocrine, Ocugen, Opiant, Orchard, Praxis, Ractigen, Radiopharm, Sesen, Terns, Thea, Travere, Unicycive