April 3, 2023

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Biotech Correspondent

Today, a dive deep into how an experimental gene therapy for epidermolysis bullosa might change lives, we delve into the biotech stock-moving events expected for the second quarter, and more.

Gene therapy
Experimental therapy offers hope for epidermolysis bullosa

A life with epidermolysis is one of constant pain. Merely bumping into something can excoriate the skin; the genetic disease causes the skin to be so exquisitely delicate that it's likened to a butterfly wing, STAT's Andrew Joseph writes. But a gene therapy developed by Krystal Biotech called B-VEC could soon be available to patients with this rare condition. The FDA will decide in the next month or so whether or not to approve it; analysts expect that it'll be green-lighted.

For some patients who have participated in the positive B-VEC clinical trials, wounds are healing faster than ever before.

"I used to have wounds on my back the size of a piece of paper," one patient in Krystal's trial told STAT. "Like 70% of my back was completely raw. Now it's 100% healed. I can sleep on my back. I don't have to worry about so much pain and blood in the shower. It's more than life-changing. It's easier to do everything."

drug pricing

Why wholesale drug prices kinda came down

When accounting for inflation, wholesale drug prices actually dropped 3% in the last quarter, a new study shows. That said, brand-name drugmakers hiked wholesale prices 5% in the fourth quarter, up from 4.3% a year earlier. And the net prices that health plans paid for medicines, when counting for inflation, dropped 8.5% — the largest quarterly decline seen by SSR Health, a research firm that conducted the analysis.

Some categories of drugs had higher net prices year-over-year, however — including oncology, atopic dermatitis, multiple sclerosis, and HIV. SSR analysts expect the growth in wholesale drug prices to parallel inflation.

Small study shows Lilly drug clears amyloid plaques

A small study showed that Eli Lilly's experimental Alzheimer's medicine, remternetug, clears amyloid plaques — in a "rapid and robust" kind of way. But it also triggered a side effect seen in other amyloid-clearing drugs like the now-approved Leqembi: brain swelling and hemorrhaging. The symptoms are part of a class of side effects called amyloid-related imaging abnormality, or ARIA. The Phase 1 study was conducted in patients with mild cognitive impairment or mild-to-moderate dementia from Alzheimer's disease.

"One participant experienced a serious adverse event due to ARIA, with symptoms resolving after discontinuation of the study drug and the administration of oral steroids," a Lilly spokesperson told FierceBiotech.

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