MoonLake has a pattern of obscuring clinical data

MoonLake Immunotherapeutics yesterday released what it described as “positive” results from a late-stage study of its autoimmune drug candidate. But the inadequate data in the press release make it hard to really judge the treatment, STAT's Adam Feuerstein writes.

It's not the first time the biotech has avoided providing detailed data, Adam points out, and drugmakers generally don't withhold results when they are strong.

gene therapy

New biotech takes aim at common cause of deafness

A new company, Skylark Bio, launched this morning and said it's dosed its first trial participant with a gene therapy aimed at restoring hearing.

Earlier this year, Regeneron received the first U.S. approval of a gene therapy for deafness. While that treatment targets the OTOF gene, Skylark is aiming for what scientists see as the holy grail — the GJB2 gene. Mutations in this gene are regarded as the most common culprits for inherited hearing loss.

The GJB2 gene is, however, a much harder scientific target.

Read more from STAT's O. Rose Broderick.

research

Can institutional review boards be fixed?

Institutional review boards, the committees that review the ethics and safety of clinical trials, are supposed to focus solely on the welfare of study participants. But over the past few decades, researchers have turned to commercial IRBs that appear to prioritize speed over patient safety.

One ethicist says it is a “a customer service model of the IRB, where they view the investigator as the customer.”

But one non-profit is trying to change the system, aiming to provide what it thinks will be independent and ethical research oversight.

oncology

A radiopharma therapy gets a surprise rejection

The FDA yesterday rejected a radiopharmaceutical therapy developed by ITM Isotope Technologies Munich SE.

The company said it was for manufacturing issues and not for any clinical data problems, but the move still halts the commercialization of the treatment, which was expected to rival a Novartis product.

Radiopharmaceuticals are an increasingly popular type of treatment designed to shoot radioactive isotopes directly at tumor cells.

Read more from STAT's Allison DeAngelis.

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