The pharmaceutical sector is seeing action in the M&A space, as Madrigal Pharmaceuticals and Bayer each announced moves, aiming to strengthen their respective pipelines through targeted acquisitions and licensing agreements.

Madrigal licenses PNPLA3-targeting siRNA

Madrigal Pharmaceuticals entered an exclusive global license agreement with Arrowhead Pharmaceuticals for ARO-PNPLA3, a clinical-stage small interfering RNA therapy targeting a key genetic driver of metabolic dysfunction-associated steatohepatitis.¹

The deal is worth upwards of $1 billion and includes an upfront payment of $25 million along with up to $975 million in milestone payments plus royalties on net sales.

ARO-PNPLA3 targets the PNPLA3 I148M variant, a well-established genetic contributor to MASH progression that is associated with increased liver fat, inflammation, fibrosis, cirrhosis, and hepatocellular carcinoma.¹ Approximately 30% of MASH patients with moderate to advanced fibrosis carry two copies of this variant, with the mutation especially prevalent in Hispanic populations.

The drug uses GalNAc-conjugated siRNA technology to deliver gene-silencing directly into hepatocytes, selectively reducing PNPLA3 protein production.

Phase I data published in the New England Journal of Medicine demonstrated liver fat reductions of up to 46% at 12 weeks following a single dose in homozygous carriers, with rapid onset at six weeks and effects sustained through at least 24 weeks. No clinically meaningful adverse events were observed.¹

Madrigal is planning to consult the FDA on the design of a Phase II combination trial pairing ARO-PNPLA3 with Rezdiffra, its approved MASH treatment, to explore whether targeting genetic disease drivers can complement Rezdiffra's broader therapeutic effects.

"MASH is a complex, heterogeneous disease, and we believe patients will benefit from personalized treatment strategies targeting key genetic risk factors that drive disease progression and adverse outcomes," said Bill Sibold, CEO of Madrigal. "We're particularly excited about the potential to advance research for members of the Hispanic community, who are disproportionately affected by MASH."

Bayer acquires Perfuse Therapeutics

In a separate deal, Bayer agreed to fully acquire Perfuse Therapeutics, a biopharmaceutical company developing PER-001, a small molecule endothelin receptor antagonist in Phase II development for glaucoma and diabetic retinopathy.²

The deal carries a total potential value of up to $2.45 billion, comprised of a $300 million upfront payment and additional development, regulatory, and commercial milestones. The transaction is subject to antitrust clearances and Perfuse stockholder approval.

PER-001 targets endothelin, the most potent vasoconstrictor in the human body, which is upregulated in glaucoma, diabetic retinopathy, and other retinal diseases. The drug is delivered as a bio-erodible intravitreal implant designed to provide sustained release via a single-use applicator, allowing for convenient dosing.² It is being studied for its ability to improve visual fields in glaucoma patients and to improve contrast sensitivity and reduce ischemia in diabetic retinopathy patients.

Glaucoma affects an estimated 76 to 80 million people worldwide and is the leading cause of irreversible vision loss, with projections pointing to 112 million affected by 2040.² Despite its prevalence, no approved treatments exist that independently prevent disease progression without relying on lowering intraocular pressure. Diabetic retinopathy affects approximately 146 million people globally, with 25 million living with vision-threatening disease.

"We are excited by the work of the team at Perfuse Therapeutics and encouraged by the potential of PER-001," said Juergen Eckhardt, M.D., head of business development and licensing at Bayer Pharmaceuticals. "With this acquisition, we are complementing our expertise in ophthalmology and our pipeline, reinforcing our commitment to developing urgently needed therapies for patients."

Sources

1. Bayer to acquire Perfuse Therapeutics to complement ophthalmology pipeline Bayer Global May 6, 2026 https://www.bayer.com/media/en-us/bayer-to-acquire-perfuse-therapeutics-to-complement-ophthalmology-pipeline/
2. Madrigal Adds Clinical-Stage siRNA Asset Targeting PNPLA3 to its MASH Pipeline Madrigal May 5, 2026 https://www.globenewswire.com/news-release/2026/05/05/3287637/0/en/madrigal-adds-clinical-stage-sirna-asset-targeting-pnpla3-to-its-mash-pipeline.html