Iptacopan

Atropos Health is collaborating with Novartis to develop and evaluate multiple AI models aimed at identifying individuals who are unaware they have a rare disease, with the goal of shortening the time it takes to diagnose such cases. The partnership is initially focused on paroxysmal nocturnal haemoglobinuria (PNH), a condition in which a person's immune system attacks their red blood cells. The California-based startup has already built an initial algorithm as part of the collaboration. Trained on longitudinal patient records and real-world data to flag possible PNH cases, the tool — available for integration into health systems — is designed to move patients more quickly from symptom reporting to diagnosis and treatment. Rodney Gillespie, head of oncology at Novartis US, said the project reflects the company’s focus on earlier diagnosis and timely care for PNH. The pharma markets Fabhalta (iptacopan), which in late 2023 became the first oral monotherapy approved for PNH in the US (see – Spotlight On: Novartis' Fabhalta set to turn up heat on AstraZeneca's PNH portfolio). Other AI models built under the partnership will be trained on real-world data from the Atropos Evidence Network, supported by the company’s GENEVA OS. The system sits inside a hospital’s own cloud environment, where it turns medical records into searchable timelines of a patient’s history. Clinicians can query these timelines to look for patterns in symptoms, lab results or treatments, with the system pulling deidentified data from across multiple sources without moving sensitive information. Atropos says this setup allows health systems to generate evidence in minutes instead of days, making it easier to spot patients who may benefit from earlier testing. For PNH, that speed matters. The condition affects 10 to 20 people per million worldwide, yet patients often wait a year, and in some cases more than five, to be diagnosed. Symptoms vary and involve multiple organ systems, a key reason why diagnosis is so often delayed until an individual is in their 30s or 40s. Earlier identification could reduce the risk of complications such as blood clots and organ damage. "Building AI models tested and trained on high-quality real-world data is truly the next frontier in precision medicine," said Atropos CEO Brigham Hyde, adding that the accuracy of the models could help reduce guesswork for clinicians, giving patients the chance to be tested sooner and potentially improving their experience of care.The collaboration extends Atropos’ work applying its technology beyond research and into everyday medicine. In January, the company inked a deal with Merck & Co. to use GENEVA OS for drug development by generating real-world evidence and replicating studies. Atropos has also partnered with Scipher Medicine and other organisations as part of a wider push to test whether real-world data platforms can speed up both research and clinical care.

Today, a brief rundown of news involving Madrigal Pharmaceuticals and Viridian Therapeutics, as well as updates from Apellis Pharmaceuticals, PTC Therapeutics and Arrowhead Pharmaceuticals that you may have missed.Seeking to expand treatment of the liver disease MASH, Madrigal Pharmaceuticals is paying $120 million to Hong Kong-based drugmaker CSPC Pharmaceutical Group for global rights to an oral GLP-1 drug in preclinical development. The deal offers CSPC potentially $2 billion in additional payments if certain development, regulatory and commercial milestones are achieved. Madrigal executives said they plan on combining the CSPC drug, called SYH2086, with the companys MASH drug Rezdiffra in a once-daily pill. They envision treatment could combine the benefits of weight loss from a GLP-1 pill with the antifibrotic and fat reducing qualities of Rezdiffra to give people with MASH additional disease control. Jonathan GardnerKissei Pharmaceutical is paying $70 million upfront and offering up to $315 million in milestone payments to Viridian Therapeutics for Japanese rights to two antibody drugs for thyroid eye disease. Viridian has already announced positive Phase 3 study data for the most advanced drug in the deal, called veligrotug. The second, called VRDN-003, is in ongoing Phase 3 testing. The company hopes to compete with Amgens marketed drug for thyroid eye disease, Tepezza, by offering a more convenient dosing regimen. Jonathan GardnerThe Food and Drug Administrationon Monday approved broader use of Apellis Pharmaceuticals rare disease drug Empaveli, clearing it for the kidney diseases C3 glomerulopathy or primary immune complex membranoproliferative glomerulonephritis in people 12 years of age or older. In testing, treatment with Empaveli reduced urine protein levels by significantly more than placebo and stabilized kidney function. Earlier this year, Novartis won FDA approval of its drug Fabhalta for use in adults with C3G. Ned PagliaruloPeople with the rare disease phenylketonuria, or PKU, have a new treatment option after the FDA on Monday approved the drug Sephiencefrom PTC Therapeutics. In a statement, the company highlighted what it described as a broad label that includes all disease subtypes in people who are 1 month of age or older. News of the approval sent shares in PTC higher by nearly 10% this week. The company is also awaiting agency decisions on drugs for<!--td {border: 1px solid #cccccc;}br {mso-data-placement:same-cell;}-->Friedrich's ataxia and Duchenne muscular dystrophy. Ned PagliaruloArrowhead Pharmaceuticals said Monday that it had earned a $100 million milestone payment from Sarepta Therapeutics under their research alliance, after meeting a patient enrollment target in a Phase 1/2 trial of its treatment for myotonic muscular dystrophy. Such fees are part of the normal course of business in biotech, but payment by Sarepta had suddenly become in doubt after the companys standoff with the FDA over whether it could continue selling its gene therapy Elevidys. The Duchenne treatment is vital to Sareptas financial future, so the prospect of it being removed from market sparked concerns Sarepta might reach a point when it would no longer be able to meet its obligations. That risk appears to have receded for now, after the FDA relented Tuesday. Arrowhead expects to receive the $100 million within 60 days. Ned Pagliarulo '