Hi Jbog. ADPKD affects 1 in 500-1000 I think and not something she has much contact with ...at least not more than the 2 or 3 patients she see's with IgAN .
Her attention is more near term such as approval of UNCY's OLC and when that will be available to her patients ...most of which are nowhere near serum pho goal and hate the current meds. PDUFA date June 25th I think.
RGLS is moving directly to a pivotal P 3 ?
VRTX is also in the space ...from Grok
Vertex Pharmaceuticals is actively developing a treatment for Autosomal Dominant Polycystic Kidney Disease (ADPKD) with their investigational drug VX-407. Here’s a detailed look at what they’re doing in this space as of March 27, 2025:
Vertex is targeting the underlying genetic cause of ADPKD, specifically in patients with certain PKD1 gene variants. ADPKD is driven primarily by mutations in PKD1 (about 80% of cases), which encodes the polycystin 1 (PC1) protein. When PC1 function is impaired, it leads to cyst proliferation, kidney enlargement, and eventual decline in kidney function. VX-407 is a first-in-class small molecule corrector designed to address this by correcting the folding of defective PC1 protein, aiming to restore its function. The goal is to halt cyst growth, reduce kidney volume, and slow progression to kidney failure—a significant shift from current treatments like tolvaptan, which only manage symptoms and slow progression without addressing the root cause.
The program has made notable progress:
In March 2024, the U.S. FDA cleared Vertex’s Investigational New Drug (IND) application for VX-407, allowing clinical trials to begin. A Phase 1 trial in healthy volunteers was initiated that month to assess safety, tolerability, and pharmacokinetics. This trial targets a subset of ADPKD patients with specific PKD1 variants, estimated at around 25,000 to 30,000 people (roughly 10% of the 250,000–300,000 ADPKD patients in the U.S. and Europe).
By late 2024, Vertex was nearing completion of this Phase 1 study. In their February 2025 financial results update, they confirmed plans to advance VX-407 into a Phase 2 proof-of-concept study in ADPKD patients in 2025. This next phase will evaluate efficacy—likely focusing on endpoints like total kidney volume (TKV) and estimated glomerular filtration rate (eGFR)—alongside safety.
Vertex’s approach reflects their broader strategy of serial innovation, building on their success in cystic fibrosis. They’re not just aiming for symptom management but seeking a transformative therapy that tackles ADPKD’s genetic basis.
This is a big deal because, despite ADPKD being the most common inherited kidney disease, no approved treatments currently target its underlying biology.
Their focus on a subset of PKD1 variants suggests a precision medicine angle, though they’ve expressed an ambition to eventually reach all 250,000+ ADPKD patients through iterative advancements, much like their cystic fibrosis playbook.
The program is still early-stage, so efficacy data isn’t yet available, and risks remain—Phase 2 will be critical to proving VX-407 can meaningfully alter disease progression. Vertex’s robust financial position ($640.9 million in cash as of late 2024) supports this effort, but ADPKD’s complexity (e.g., variable progression rates, cyst heterogeneity) could pose challenges. For now, they’re a key player to watch in the ADPKD space, alongside others like Regulus Therapeutics (with farabursen) and Otsuka (with tolvaptan as the current standard).
Kiwi
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