Karuna's schizophrenia treatment effective in large trial

A schizophrenia treatment developed by Karuna Pharmaceuticals called KarXT reduced psychosis reported by patients in a large clinical trial, according to data released this morning. The data underscore similarly positive results in smaller studies, which means that Karuna will file a marketing application for the drug with the FDA by the middle of next year. If approved, KarXT will mark the first time in decades that schizophrenia has a new class of treatment options.

"KarXT could profoundly change the standard of care, and I think the psychiatric community is anticipating it," Karuna CEO Bill Meury told STAT.

When taken twice daily, KarXT reduced schizophrenia by 20.6 points, compared to a placebo decrease of 12.2 points, when measured by the Positive and Negative Syndrome Scale. The participants taking KarXT also showed statistically significant improvements in hallucinations and delusions, but had no effect on symptoms like emotional and social withdrawal.

autoimmune disease

Takeda's $6 billion experimental drug shows efficacy

Last month, Takeda Pharmaceutical poured $6 billion into an experimental pill for autoimmune and inflammatory disease. Now, we're beginning to see why: Mid-stage clinical data from 259 people with moderate-to-severe plaque psoriasis has just been released, and they show that the highest dose of the daily pill completely cleared itchy and painful patches of skin in a third of the patients after 12 weeks. "We're offering the potential for a functional cure," said Takeda's R&D president, Andrew Plump.

The pill, developed by Nimbus Therapeutics, helped other patients in the trial: Nearly half the patients taking the highest dose had 90 percent of their psoriasis cleared, and two-thirds had 75 percent cleared — compared to 6 percent receiving placebo. There were side effects, however, which include an increased risk of mild respiratory illnesses like Covid-19, and acne.

Takeda's is bullish on the drug, which it's testing in other autoimmune and inflammatory conditions, like lupus, Crohn's disease, and ulcerative colitis. "This has the potential to be one of the most broadly indicated mechanisms — outside of oncology — of any oral medicine that's ever been developed," Plump said. "I think in 10 years, we'll look back and say that this was a bargain."

multimedia

But what's a p-value, really?

Generally speaking, the whole point of the clinical trial is to determine whether an intervention outperforms placebo. To determine whether results are statistically significant, researchers use a measurement called a "p-value." But, uh, what is it?

For those of you who could use a concise explainer of how a p-value's derived and why it's still considered the gold standard for statistical significance, watch this video from STAT's Damian Garde.

Watch here.

options

Moderna CEO still donating stock windfall to charity

Moderna CEO Stéphane Bancel made $398 million in stock returns last year alone — but said in a new company filing that he's "committed to contributing the after-tax proceeds from the option exercise to charitable causes." Bancel's windfall is due to pre-planned stock sales, based on stock options he acquired in August 2013. They expire this coming August — so since last May, Bancel has been selling them off. They were purchased at $0.99 per share, and he's sold them for $140 per share.

Last year, Bancel wrote a blog post that explained his rationale for donating the money — which he projected to be about $355 million after taxes. "If we do not exercise this stock option, it will expire and be worthless," he wrote. "As such, our choice is clear: Exercise the option and use this moment to create positive change in the world."