Daily Snap - 1. September 2025

Author

Joachim Eeckhout
September 01, 2025

Good morning! Argentine biotech company Kheiron Biotech has created the world’s first genetically edited horses using CRISPR, cloning five foals from a prize-winning polo horse. By reducing the expression of the myostatin gene, the foals were engineered for greater speed and power. Although Argentina’s polo scene has embraced cloning, the introduction of gene-edited horses has sparked controversy. The Argentine Polo Association has banned them from competition, and breeders have raised concerns about tradition and market disruption, prompting Kheiron to pause commercialization while awaiting broader acceptance. In a twist of irony, the foals were cloned from a mare named Polo Pureza (Polo Purity in English), but with science rewriting their DNA, ‘purity’ might need a new definition.

Enjoy today’s read!

—Joachim E.

SNIPPETS

What’s happening in biotech today?

🏃‍♂️RA retreat: Johnson & Johnson has discontinued the development of Imaavy (nipocalimab) as a combination therapy with anti-TNFα agents for rheumatoid arthritis (RA) after a phase 2a trial showed no added benefit over anti-TNFα therapy alone in patients with refractory RA. The company did not release full data from the 12-week Daisy study but cited the lack of efficacy as the reason for ending this specific clinical pursuit. Despite this setback, J&J remains optimistic about Imaavy’s broader potential, particularly in treating generalized myasthenia gravis and other immunological conditions, positioning the drug for over $5 billion in peak sales.

🧬TYK2 takeover: Fosun Pharma has signed its third inflammatory disease partnership in three weeks, acquiring Greater China rights to Accro Bioscience’s TYK2/JAK1 inhibitor, AC-201, for an upfront payment of 60 million yuan ($8.3 million), with up to 96 million yuan ($13.4 million) in potential milestones. AC-201, an oral small molecule for immune-mediated inflammatory diseases, completed a phase 2 trial in moderate-to-severe plaque psoriasis, meeting its primary endpoint with no serious safety concerns. The deal supports Fosun’s strategic expansion in immunology and inflammation, which now forms half of its core focus alongside oncology. Accro retains global rights outside Greater China.

🚀Hypertension moonshot: Despite missing the primary endpoint in its phase 2 KARDIA-3 trial, Alnylam Pharmaceuticals, together with Roche, is moving ahead with a phase 3 program for zilebesiran, its RNAi therapeutic for uncontrolled hypertension. The ZENITH trial, set to begin by the end of 2025, will enroll roughly 11,000 high-risk patients already on multiple antihypertensives to test whether zilebesiran reduces cardiovascular death, nonfatal myocardial infarction, stroke, and heart failure compared to placebo. While phase 2 results fell short, they informed trial design and showed potential for sustained blood pressure lowering. Safety data across treatment groups indicated manageable adverse event rates.

🫁Oxygen boost: Cytokinetics presented full Phase III MAPLE-HCM trial results showing that its investigational drug aficamten significantly outperformed the beta blocker metoprolol in treating obstructive hypertrophic cardiomyopathy (oHCM), challenging long-standing treatment norms. The study, involving 175 patients, demonstrated superior outcomes for aficamten in exercise capacity, functional class, quality of life, and cardiac biomarkers, with a notable 2.3 mL/kg/min advantage in peak oxygen uptake. Safety profiles were comparable, though aficamten showed fewer treatment-related dose adjustments. These findings could position the drug as a strong competitor to BMS’s Camzyos.

🧪PBC win: Zydus Therapeutics has reported positive results from a phase 2b/3 trial of its PPAR agonist saroglitazar in patients with primary biliary cholangitis (PBC), achieving a 48.5% biochemical response rate after 52 weeks of treatment, thus meeting the trial’s primary endpoint. This positions the company to seek FDA approval in early 2026, entering a competitive market alongside Gilead’s Livdelzi (62% response) and Ipsen’s Iqirvo (51%), both approved in 2024. While cross-trial comparisons are limited due to missing placebo and symptom data, Zydus aims to demonstrate potential benefits in safety and tolerability.

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