May 17, 2023

The need-to-know this morning

PTC Therapeutics said its experimental treatment for phenylketonuria, a rare, inherited metabolic disease, achieved the primary goal of a Phase 3 clinical trial. The drug, called sepiapterin, showed an average 63% reduction in levels of Phe, a protein that accumulates to toxic levels in patients with PKU, compared to "minimal reductions" in patients treated with a placebo.
Yesterday, the Federal Trade Commission issued a press release to announce the lawsuit seeking to block the merger of Amgen and Horizon Therapeutics. This morning, the actual FTC complaint was made public. You can read it here.
Gilead Sciences named former Roche executive Cindy Perettie as the new head of its Kite cancer cell therapy division. Perettie is the former CEO of cancer diagnostics company Foundation Medicine, which was acquired by Roche in 2018. Perettie takes over leadership of Gilead's CAR-T business from Christi Shaw, who left the company two months ago.

Regulatory

The FTC wasn't kidding

After hours of speculation and conflicting reports, the FTC filed suit to block Amgen's nearly $30 billion acquisition of Horizon Therapeutics yesterday, following through on its earlier promise to crack down on potentially anti-competitive pharmaceutical mergers.

As STAT's Andrew Joseph reports, the FTC's concern is not overlap between the two companies' products but rather the possibility that Amgen would "bundle" its medications — that is, offer a discount to payers if they covered a multitude of its drugs — in order to block competition to Horizon's best-selling medicines.

In a statement, Amgen said that idea is "entirely speculative and does not reflect the real-world competitive dynamics behind providing rare-disease medicines to patients." Amgen is confident it can defeat the FTC's challenge and close its acquisition of Horizon, but the company pushed back its expected resolution to December.

Read more.

M&A

Now what?

Analysts widely expect Amgen to succeed, eventually, with its Horizon merger. But the intervening bad press and regulatory scrutiny could be damaging.

As Baird analyst Brian Skorney wrote in a note to investors, Amgen can make a convincing case that the aforementioned specter of bundling is too broad to be enforceable; by the FTC logic, if the company can't buy Horizon, it can't buy anything. But that could be "a very pyrrhic victory," according to Skorney. Amgen is already facing a lawsuit from Regeneron Pharmaceuticals claiming the company is using bundling tactics to thwart competition, and the added attention of the FTC could constrain the business.

Zooming out, while Amgen's legal trouble might give pause to major drugmakers considering large acquisitions, it could be a boon to smaller biotech firms, according to Josh Schimmer of Evercore ISI. "It's not like pharma is going to stop relying on biotech companies to help drive sustainable growth," Schimmer wrote to investors. "It's just going to force them to look earlier in development, which is where most of the biotech industry currently exists."

Read more.

In the lab

The plot to train gene therapy on cancer

Interferon, a powerful protein in the immune system's arsenal, has a checkered history as a medicine. It can be a potent treatment for cancer and infectious disease, but only at high doses that bring bracing side effects, rendering it all but unusable. Now, thanks to the technology that makes gene therapy possible, scientists believe they can shuttle the inflammatory molecule straight to the site of tumors, killing cancer while sparing the healthy cells nearby.

As STAT's Jason Mast reports, that's the idea behind Siren Biotechnology, a new startup working to adapt the harmless human viruses used to deliver gene therapy into delivery vehicles for interferon and other immune-stimulating molecules. Those viruses home in on cancer cells, which then recruit surrounding immune cells to attack them.

"This is already the way your body fights cancer," said Nicole Paulk, a former University of California San-Francisco investigator who shut down her lab to start the company. "We're basically just supercharging this and harnessing it and putting it into gene therapy."

Read more.

Biotech

Billy Dunn makes biotech landfall

Billy Dunn, the influential regulator who presided over the polarizing FDA approval of the Alzheimer's disease treatment Aduhelm, has taken his first industry job since abruptly leaving the agency earlier this year.

Dunn is joining the board of Prothena, a biotech company developing treatments for neurological diseases including Alzheimer's, the firm said yesterday. Prothena pays its directors an annual $60,000 cash retainer plus additional fees for sitting on committees, according to its most recent proxy statement. Dunn will also receive 30,000 option awards, worth about $2 million at Prothena's current share price.

Back in February, Dunn retired from the agency to "explore other opportunities," according to an internal FDA email. He became the center of a damaging FDA scandal in 2021 when the agency overruled its advisers to approve Aduhelm, the Biogen-developed Alzheimer's treatment. Two years before, Dunn had an off-the-books meeting with Biogen's head scientist, setting in motion a close collaboration between the company and the agency that led to multiple federal investigations and a stinging rebuke from an influential congressional committee.