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Anti-resistance investments, a Q2 scorecard, & news out of ACC

    

 

The Readout

It's Meghana today, and will be Meghana all week. Got some news out of ACC, as well as details on the first investments out of a venture fund focused entirely on combatting antibiotic resistance. 

Drug resistance venture fund invests for the first time

A venture fund focused on generating medications meant to combat antibiotic resistance has made its first investments. The AMR Action Fund, which has an initial $1 billion in backing, will funnel some of that cash into Venatorx Pharmaceuticals and Adaptive Phage Therapeutics — two privately held biotechs with different approaches to fighting resistance.

Venatorx is developing a novel intravenous drug that’s being tested in UTIs; it plans to seek regulatory approval later this year. It’s also developing a medicine for hospital-acquired bacterial pneumonia. Adaptive Phage, meanwhile, is developing a library of phages — that is, naturally occurring viruses that infect and kill bacteria. It’s working on matching phages with infections that affect joints in prosthetics and in the bones and lungs.

Read more.

The Q2 biotech scorecard

It’s a new quarter, so we’ve got a handy scorecard to keep track of stock-moving biotech events.

On the roster: Alnylam Pharmaceuticals will have a new readout for its approved RNA therapeutic, Onpattro, to expand its use among patients with transthyretin amyloidosis cardiomyopathy. Another interesting one: Fulcrum Therapeutics is developing a pill called FTX-6058 to treat patients with sickle cell disease. It works by increasing the production of oxygen-carrying fetal hemoglobin — the same tactic used by several gene therapy companies. Results from a Phase 1 trial are coming out this quarter.

Twenty-one such events are coming up in the next few months.

Read more.

Cardiomyopathy drug reduces need for invasive intervention

A Cleveland Clinic-led trial showed that an experimental drug called mavacamten can be used as an alternative to heart surgery or alcohol septal ablation in patients with severe cardiomyopathy. These therapies are used to reduce the thickening of the septum, which is the wall that separates the right and left sides of the heart.

The trial enrolled 112 patients with symptomatic hypertrophic cardiomyopathy, all of whom needed either surgery or ablation. They were given either a high or low dose of mavacamten, which was given orally, or placebo. The drug works by stopping unnecessary contractions of the heart muscle, making it more efficient. After 16 weeks, 43 of the 56 patients given placebo still needed surgery; meanwhile, only 10 patients out of 56 given mavacamten needed invasive intervention.

“These results could give what can be a very sick patient population a non-invasive therapy alternative,” the study’s lead author said in a statement. The results were released during a late-breaking session during the annual meeting of the American College of Cardiology.

Silence Therapeutics siRNA drug (so far) works as expected

A siRNA drug from Silence Therapeutics seems to be reasonably safe, according to a Phase 1 trial whose results were just published in JAMA and described during a late-breaking session at the annual meeting of the ACC. The drug, called SLN360, lowered the plasma concentrations of lipoprotein(a) in participants, up to 98% of whom had elevated levels and no known cardiovascular disease.

The trial effectively shows that Silence Therapeutics’ RNA-based approach to cardiovascular disease can work. Silence Therapeutics is a “gene silencing” company — whose platform works to temporary block a specific gene’s impact.

More reads

  • How long covid is accelerating a revolution in medical research. (Washington Post)
  • Moving xenotransplantation research into human trials will require adjusting our expectations, researchers say. (STAT)
  • Mark Cuban’s company is building a ‘parallel supply chain’ for prescription drugs. (STAT)
  • Should race be included in allocation algorithms for Covid-19 therapies? (STAT)

Thanks for reading! Until tomorrow,

@megkesh
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Monday, April 4, 2022

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