Rapid and Sustained Response of Biochemically Uncontrolled Acromegaly to Once-daily Oral Paltusotine Treatment
Primary result · IGF-1 normalizationResult — go to the resultOdds ratio 42.81 95% CI 8.44–455.82Authors' conclusion
IGF-I normalized in significantly more patients with uncontrolled acromegaly treated with paltusotine vs placebo. Paltusotine was associated with rapid, sustained IGF-I reduction, significant symptom improvement and stable or reduced pituitary tumor size and was well tolerated.
Study context
- Population
- Adults with medically untreated, biochemically uncontrolled acromegaly at randomization (stratum 1: medication-naïve or off medications ≥4 months, IGF-1 ≥1.3 × ULN; stratum 2: controlled on SRL with washout, IGF-1 increase ≥30% to ≥1.1 × ULN)stratum 1 n = 82
- Participants
- 111 adults
- Comparison
- Once-daily oral paltusotine vs placebo for 24 weeks
- Paltusotinen = 54
- Placebon = 57reference
- Primary endpoint
- IGF-1 normalization
- Follow-up
- 24 weeks
Methodological detailsphase 3, double-blind, placebo-controlled
- Phase 3
- Double-blind
- Placebo-controlled
Primary result
As reported in the abstract
Odds ratio
42.81
95% CI 8.44–455.82
P < 0.0001
By group
Paltusotinevs Placebo
OR 42.81 (95% CI 8.44–455.82); P < 0.0001
Secondary outcomes
| Outcome | Paltusotine | Placebo | OR (95% CI) | P value | |
|---|---|---|---|---|---|
| IGF-1 < 1.3×ULN | 66.7% | 14.0% | 18.32 (5.64–79.16) | P < 0.0001 | |
| GH (5-sample mean) < 1.0 ng/mL | 57.4% | 17.5% | 7.59 (2.78–23.48) | P < 0.0001 | |
| 0.050.21520Odds ratio (log scale) |
| Outcome | Paltusotine | Placebo | P value |
|---|---|---|---|
| Decrease in IGF-1 within first 4 weeks | 92.6% | — not stated in the abstract | |
| Change in IGF-1 | mean −0.82 (SE 0.08) ×ULN | mean 0.09 (SE 0.08) ×ULN | P < 0.0001 |
| Change in Acromegaly Symptom Diary scoreMean | −2.7 (SE 1.4) | 2.8 (SE 1.4) | P = 0.004 |
— not stated in the abstract
Pituitary tumor volume
- Pituitary tumor volume stable or reduced with paltusotine
Safety
Adverse events
- Most AEs were acromegaly symptoms or mild, transitory GI effects characteristic of SRLs (eg, diarrhea, abdominal pain)
Original publication
Rapid and Sustained Response of Biochemically Uncontrolled Acromegaly to Once-daily Oral Paltusotine Treatment. J Clin Endocrinol Metab. Published 23 October 2025. doi:10.
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