May 16, 2023

The need-to-know this morning

The Federal Trade Commission is preparing to file a lawsuit to block Amgen's $27.8 billion acquisition of Horizon Therapeutics, according to media reports last night, citing unnamed sources familiar with the matter. The FTC has not announced a suit (yet), and Amgen said last night that it was not aware of any decision made by the agency. Horizon shares fell 17% on the reports, Amgen shares were unchanged.
The possible FTC intervention signals heightened antitrust scrutiny of biopharma mergers at a time when stock prices across the sector are rising, in part, because of increased M&A activity. Nearly $70 billion in deals have been announced since the start of the year. There is little competitive overlap in the approved medicines sold by Amgen and Horizon, but analysts are speculating that a more aggressive FTC under Chair Lina Khan could be objecting to the potential overlap in their respective research pipelines of drugs for autoimmune diseases; or using a "bad actor" argument against Amgen, accusing it of bundling products to block competition.
Filing a lawsuit and winning one are not the same, but an FTC action could delay the closing of the Amgen-Horizon deal, which was set for next week. It may also raise additional antitrust concerns about Pfizer's intention to acquire Seattle Genetics for $43 billion. More broadly, the sector's M&A mini-bubble could deflate.

biotech

How a DNA curiosity led to a novel idea for treating cancer

It has been more than five decades since scientists first spotted the tiny rings of DNA that float through tumor cells but almost never in healthy cells. Now, that curious genetic material, called extrachromosomal DNA, is the target of an ambitious — and well-funded — biotech project.

As STAT's Jonathan Wosen reports, San Diego's Boundless Bio is running a clinical trial to test whether targeting extrachromosomal DNA can control the growth and spread of deadly tumors. The company closed a $100 million funding round to bankroll the study and support its work on earlier stage drug candidates.

Boundless is enrolling patients with a variety of tumor types, so long as their cancer expresses certain genetic mutations. The company expects to have data on the safety of its drug, called BBI-355, by the end of this year and early signs of its efficacy in the first half of 2024.

Read more.

Chart of the day

The Sarepta effect

D3 vis exported to PNG (56)

The XBI biotech index has risen about 4% since last week, approaching its 2023 heights thanks in part to Sarepta Therapeutics' surging stock price.

Sarepta rose about 30% yesterday in reaction to a Friday FDA meeting in which agency advisers voted narrowly in favor of granting accelerated approval to the company's gene therapy for Duchenne muscular dystrophy.

The news was a boon to other companies working on potentially curative medicines for rare diseases. Intellia Therapeutics, CRISPR Therapeutics, and Beam Therapeutics also spiked yesterday, reflecting investors' optimism that the FDA will extend the same leniency in the future.

Deals

Gilead is going back to the well

Back in 2019, Gilead Sciences paid $5 billion to Galapagos NV with the goal of building a pipeline of medicines for inflammatory disease. Three years and three late-stage failures later, that deal hasn't quite panned out.

But Gilead isn't giving up. In a new agreement with Arcus Biosciences, disclosed yesterday, Gilead is paying $35 million to work with the company on up to four anti-inflammatory targets. Arcus is due up to $420 million in milestone payments. The deal builds on Gilead's existing agreement with Arcus covering cancer treatments.

Gilead CEO Daniel O'Day has said Galapagos' many setbacks haven't dampened the company's ambitions to expand beyond its banner antiviral business and growing oncology division into medicines for inflammatory disease.

Research

Gene therapies for the rarest of rare diseases

An ambitious Foundation for the National Institutes of Health effort to develop gene therapies for the world's rarest diseases said today it would to put treatments for eight diseases into clinical trials by the middle of 2024.

Some of these serious diseases, including severely degenerative conditions such as spastic paraplegia 50 and multiple sulfatase deficiency — along with types of genetic blindness — may be unfamiliar even to many rare disease researchers. That's part of the point. The $80 million program, known as the Bespoke Gene Therapy Consortium, is designed to tackle a few of the many diseases that are theoretically treatable with modern technologies but are so rare no company is willing to invest the required resources. In the process, the NIH and its partners hope to build standardized processes to continually develop such ultra-rare therapies going forward.

The conditions were chosen for the level of industry (dis)interest, how feasible they would be to treat with validated technology and how quickly one could generate efficacy data, among other factors, said Courtney Silverthorn, associate VP of science partnerships at FNIH. The goal is to have early results within 12 to 18 months of dosing.

What happens to each program afterwards, though, remains unclear. Sometimes, with ultra-rare diseases, a gene therapy proves effective but goes nowhere because no company will foot the bill for regulatory filings and manufacturing. For now, Silverthron said, the NIH is focused on everything through Phase 1 trials. After that, it will assist anyone hoping to advance the treatment further and keep the studies open for additional patients, but only if they can separately secure the cost of manufacturing.

Neuroscience

Another twist in 'Alzheimer's resilience'

For the second time in one extended family, researchers have identified a person who was genetically predisposed to develop early Alzheimer's disease but somehow remained cognitively sound for several more decades.

As STAT's Andrew Joseph reports, the finding came out of a long-running study of a large extended Colombian family with a rare genetic mutation that virtually guarantees early-onset Alzheimer's. As in the first case of apparent Alzheimer's resilience, scientists zeroed in on a genetic variant that seemed to be protective, overcoming the impact of the mutation that would have otherwise set this man on the course for dementia in his 40s.

But this time it was a different gene, suggesting there might be multiple pathways that stave off cognitive decline despite overwhelming predisposition. Scientists were already at work on potential Alzheimer's therapies based on the first discovery, and the emergence of a second protective mutation gives them another target to pursue.

Read more.

More around STAT