UniQure will submit Huntington's therapy for approval

The FDA has reversed its opposition to a closely watched experimental treatment for Huntington’s disease, clearing a path for its maker, the biotech company UniQure, to file for U.S. approval, the company said morning.

UniQure will submit a marketing application in the third quarter seeking accelerated approval for the treatment, called AMT-130, STAT's Adam Feuerstein reports. The company said the decision comes after a recent meeting with FDA officials during which the agency agreed that a three-year analysis of an early-stage study that showed a benefit for patients with Huntington’s was “acceptable” to support a marketing application, the company said.

AMT-130, if approved, would be the first genetic treatment for Huntington’s.

biotech

Why one VC doesn't want to restrict China access

In recent months, some legislators have been pushing to add biotech to the list of industries covered by the COINS Act, which allows the U.S. government to restrict certain investments abroad. Many biotech VCs have spoken out against this move.

One of them is Julie Grant, a partner at venture capital firm Canaan.

“Small, lean companies are the ones that create the miracle medicines of the future. … Forcing them to do things slower and more expensively in the United States does not save us. It actually makes it harder,” she told my colleague Allison DeAngelis. She also had more to say about a downside of striking an M&A deal, and about the ways in which SpaceX’s and Anthropic’s IPOs could affect biotech investing.

infectious disease

FDA appears open to Moderna's flu vaccine ahead of adcomm

From STAT's Helen Branswell: Moderna's mRNA flu vaccine may have an easier pathway to market than appeared to be the case several months ago.

The FDA's expert panel on vaccines, VRBPAC, meets tomorrow to pore over the vaccine's data and advise whether it thinks the benefits of its use outweigh any risks for people aged 50 through 64, and people 65 and older. The company is seeking traditional approval for the younger group, and accelerated approval for those 65 and older using immunogenicity data that suggest it is at least as effective as the high-dose and adjuvanted flu vaccines that are preferentially recommended for people in the older age group. The company has promised to do a Phase 4 study to establish efficacy in that latter group.

Documents prepared for the meeting suggest the FDA has concluded the vaccine met pre-specified criteria for success in the studies conducted. There were no safety concerns, though it was noted that reactogenicity was higher with the Moderna vaccine than the standard-dose flu vaccine used as a comparator.

Earlier this year, Vinay Prasad, then the head of the FDA's vaccines and biologics division, issued a stunning "refusal-to-file" letter — over the advice of FDA career staff — stating that the data Moderna had submitted was insufficient to warrant consideration of the vaccine. After a public outcry, the FDA relented and agreed to consider the application, setting an August decision date.

M&A

Lilly snaps up pain-focused biotech

Eli Lilly is buying 4E Therapeutics, a biotech developing non-opioid pain drugs, continuing its string of acquisitions.

With the cash from its blockbuster GLP-1 franchise, Lilly has been investing heavily in therapeutic areas outside of obesity. The new deal adds to its existing pipeline of pain therapies. The terms of the deal were not disclosed.

There's been increased interest in the field as Vertex last year got approval for its non-opioid pain drug, Journavx, to be used in acute pain.

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