Rare disease

Huntington's researcher treads (lightly) into UniQure-FDA fray

From my colleague Andrew Joseph: When the biotech UniQure last year said that its experimental gene therapy for Huntington’s disease had slowed the progression of the untreatable condition by 75%, Ed Wild, a longtime Huntington's clinician at University College London and one of the trial's investigators, described the outcomes as “the result we’ve been waiting for.”

A lot, of course, has changed since then.

To recap: The results seemed to tee up accelerated approval at the FDA. But then the FDA, which UniQure said had previously agreed on the company’s trial design and reliance on historical data as a comparator group, told the company that it could no longer apply for approval with its data. FDA officials have since suggested they don’t think the therapy works; the agency and company remain at odds over what another trial should look like and whether there was ever an agreement over the accelerated approval application plan in the first place.

Speaking at the Advanced Therapies conference in London today, Wild was diplomatic in discussing what had ensued since the top-line results were announced.

“A new and interesting approach from the agency would be applied,” was how he described the FDA changing its view on whether the earlier study could merit a review and its demand for a new trial with a sham surgery as a control arm.

And on where things may shake out with the FDA and the therapy, called AMT-130: “The agency is in a state of change, and it remains to be seen where they will settle.” (That could have been a reference to the fact that Vinay Prasad, who came into the FDA as the agency’s top gene therapy regulator with the Trump administration and is seen as a harsh scrutinizer of gene therapies, is leaving the agency, a move that was announced soon after the most recent flap over the Huntington’s gene therapy.)

Wild noted that the U.S. had an election since UniQure announced the top-line results of its study last year, with the Trump administration shaking up FDA leadership. Asked how researchers were interpreting the shifting FDA goalposts and what that could mean for designing future trials, Wild said: “We should take nothing for granted. Elections matter, that’s certainly very true.”

Pharma

J&J wins approval for first-of-its kind psoriasis pill

Johnson & Johnson won U.S. approval for a daily psoriasis pill that rivals the benefits of injectable medicines and could reshape one of the most competitive fields in the pharma industry.

The drug, Icotyde, is approved for patients over the age of 12 with moderate to severe plaque psoriasis. It's designed to mimic the effects of top-selling injectable treatments, including AbbVie’s Skyrizi and J&J’s own Tremfya.

Patients with psoriasis typically cycle through topical drugs before getting prescribed an injectable therapy, and many opt out of treatment for reasons of convenience or cost. J&J thinks Icotyde has the potential to expand the market, and it expects the drug to bring in more than $5 billion in peak annual sales.

Read more from STAT's Damian Garde.

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VC firm Dimension seeks $700 million for new fund

Venture capital firm Dimension is starting to raise its third fund, hoping to amass around $700 million to invest in companies that combine artificial intelligence with science, my colleague Allison DeAngelis has learned.

Dimension was founded by a trio of young VCs who previously worked at Lux Capital and Obvious Ventures. One of its most high-profile investments has been in Chai Discovery, a company working on AI drug discovery that now commands a $1.3 billion valuation.

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