Pharmaceutical Industry Roundup: Key Developments and Deals

Madrigal Expands into GLP-1 Space with CSPC Deal

Madrigal Pharmaceuticals has made a significant move into the GLP-1 market, securing global rights to an oral GLP-1 drug from Hong Kong-based CSPC Pharmaceutical Group. The deal involves an upfront payment of $120 million, with potential additional payments of up to $2 billion tied to development, regulatory, and commercial milestones.

The acquired drug, SYH2086, is currently in preclinical development. Madrigal plans to combine it with their MASH (metabolic dysfunction-associated steatohepatitis) drug Rezdiffra in a once-daily pill. This combination aims to leverage the weight loss benefits of GLP-1 drugs alongside Rezdiffra's antifibrotic and fat-reducing properties, potentially offering enhanced disease control for MASH patients.

FDA Approvals Bolster Treatment Options for Rare Diseases

The FDA has granted several important approvals, expanding treatment options for patients with rare diseases:

1. Apellis Pharmaceuticals received approval for broader use of Empaveli in treating C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis in patients 12 years and older. Clinical trials demonstrated Empaveli's ability to reduce urine protein levels and stabilize kidney function significantly more than placebo.

2. PTC Therapeutics' drug Sephience gained approval for the treatment of phenylketonuria (PKU) in patients one month of age and older. The approval covers all disease subtypes, providing a new option for this rare genetic disorder.

These approvals follow Novartis' earlier FDA clearance for Fabhalta, indicated for adult C3G patients.

Strategic Partnerships and Milestone Payments

The pharmaceutical industry continues to see significant deal-making activity:

• Viridian Therapeutics has partnered with Kissei Pharmaceutical, granting Japanese rights to two antibody drugs for thyroid eye disease. The deal includes a $70 million upfront payment and up to $315 million in milestone payments. Viridian's lead candidate, veligrotug, has shown positive Phase 3 results and aims to compete with Amgen's Tepezza by offering a more convenient dosing regimen.

• Arrowhead Pharmaceuticals received a $100 million milestone payment from Sarepta Therapeutics after meeting patient enrollment targets in a Phase 1/2 trial for their myotonic muscular dystrophy treatment. This payment comes as a relief following recent uncertainty surrounding Sarepta's gene therapy Elevidys.

These developments highlight the ongoing collaborative nature of pharmaceutical research and development, as companies leverage partnerships to advance promising therapies and expand their market reach.