Drugs / PBF-999
Trials 3
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1 | NCT03786484 | Oct 2017 → Jun 2022 | cancer | Palobiofarma SL | Completed | No outcome recorded |
| Phase 1 | NCT02907294 | Mar → Apr 2016 | Huntington disease | Palobiofarma SL | Terminated | No outcome recorded |
| Phase 1 | NCT02208934 | Sep 2014 → Oct 2015 | Huntington disease | Sant Pau Research Institute | Completed | No outcome recorded |
News releases announcing trial results or a regulatory action · 4
| Date | Issuer | Release |
|---|---|---|
| 2025-10-29 | Palo Biofarma, S.L | Regulatory Palobiofarma announce several important milestones dealing with the clinical development of PBF-999 for Prader-Willi Syndrome palobiofarma.com ↗
The FDA has granted an IND for the program |
| 2024-03-04 | Palo Biofarma, S.L | Regulatory PBF-999, the first in class treatment in development for the Prader-Willi Syndrome has received “Orphan Drug Designation” by the European Medicines Agency (EMA) and the US Food and Drug Administration (FDA). palobiofarma.com ↗
Palobiofarma S.L. is pleased to announce that the FDA and the EMA have granted Orphan Drug Designation to our novel therapeutic candidate, PBF-999, for the treatment of Prader-Willi Syndrome (PWS). |
| 2023-11-06 | Palo Biofarma, S.L | Regulatory Palobiofarma S.L. Receives Rare Pediatric Disease Designation from the U.S. FDA for PBF-999 for the Treatment of Prader-Willi Syndrome palobiofarma.com ↗
Palobiofarma S.L. announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) Designation for the company’s phosphodiesterase 10 (PDE-10) inhibitor, PBF-999, for the treatment of patients with Prader-Willi Syndrome (PWS). |
| 2018-10-01 | Palo Biofarma, S.L | Results Palobiofarma completed the dose escalation part of the Phase I study with PBF-999 in cancer patients palobiofarma.com ↗
The product has demonstrated to be safe and well tolerated at all the doses treated, with stabilization of the disease seen in several patients. |
All press releases naming this drug 7 releases
Evidence & citations 6 cited values
Every value below carries the sentence it was read from. 4 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | PBF-999 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT03786484 ↗ |
| Action | Inhibit | “Phosphodiesterase 10 inhibitor (PDE10i)” NCT03786484 ↗2“Adenosine A2a receptor antagonist” CHEMBL3942498 ↗ “Phosphodiesterase 10A inhibitor” CHEMBL3942498 ↗ |
| Modality | Small molecule | — CHEMBL3942498 ↗ |
| Route | Oral | “Single Ascending Oral Doses of PBF-999” NCT02208934 ↗ |
| Target | ADORA2A | “Adenosine A2a receptor antagonist” CHEMBL3942498 ↗ |
| Target | PDE10A | “Phosphodiesterase 10A inhibitor” CHEMBL3942498 ↗ |