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PIK3CA-Related Overgrowth Spectrum (PROS)

6
Pipeline Programs
5
Companies
8
Clinical Trials
3 recruiting
0
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
0
0
6
0
0
0
Early DiscoveryClinical DevelopmentMarket

Drug Modality Breakdown

Small Molecule
5100%
+ 4 programs with unclassified modality

Competitive Landscape

5 companies ranked by most advanced pipeline stage

Sandoz
SandozAustria - Kundl
5 programs
3
AlpelisibPhase 2Small Molecule1 trial
AlpelisibPhase 2Small Molecule1 trial
AlpelisibPhase 2Small Molecule1 trial
alpelisibN/ASmall Molecule1 trial
alpelisibN/ASmall Molecule1 trial
Active Trials
NCT04285723Completed57Est. Apr 2021
NCT04085653Available
NCT06997588Recruiting104Est. Sep 2030
+2 more trials
Relay Therapeutics
Relay TherapeuticsMA - Cambridge
1 program
1
RLY-2608Phase 21 trial
Active Trials
NCT06789913Recruiting277Est. Oct 2031
Human BioSciences
Human BioSciencesWV - Martinsburg
1 program
1
SirolimusPhase 21 trial
Active Trials
NCT02428296Completed39Est. Feb 2018
Genome & Company
Genome & CompanyKorea - Suwon
1 program
1
SirolimusPhase 2
HaiHe Biopharma
HaiHe BiopharmaChina - Shanghai
1 program
CYH33PHASE_1_21 trial
Active Trials
NCT06975618Recruiting141Est. Dec 2029

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
2029
2030
2031
SandozAlpelisib
Relay TherapeuticsRLY-2608
SandozAlpelisib
SandozAlpelisib
Human BioSciencesSirolimus
HaiHe BiopharmaCYH33
Sandozalpelisib

Clinical Trials (8)

Total enrollment: 865 patients across 8 trials

NCT06997588SandozAlpelisib

EPIK-P4: A Phase II Single-arm Study to Assess the Efficacy, Safety and Pharmacokinetics of Alpelisib (BYL719) in Pediatric and Adult Patients With PIK3CA-related Overgrowth Spectrum (PROS)

Start: Oct 2025Est. completion: Sep 2030104 patients
Phase 2Recruiting
NCT06789913Relay TherapeuticsRLY-2608

A Phase 2 Study of Mutant-selective PI3Kα Inhibitor, RLY-2608, in Adults and Children With PIK3CA Related Overgrowth Spectrum and Malformations Driven by PIK3CA Mutation

Start: Jun 2025Est. completion: Oct 2031277 patients
Phase 2Recruiting
NCT04980833SandozAlpelisib

Study Assessing Long-term Safety and Efficacy of Alpelisib in Patients With PIK3CA-Related Overgrowth Spectrum (PROS) Who Previously Participated in Study CBYL719F12002 (EPIK-P1)

Start: Jan 2022Est. completion: Aug 202741 patients
Phase 2Active Not Recruiting
NCT04589650SandozAlpelisib

Study Assessing the Efficacy, Safety and PK of Alpelisib (BYL719) in Pediatric and Adult Patients With PIK3CA-related Overgrowth Spectrum

Start: Apr 2021Est. completion: Jan 2031206 patients
Phase 2Active Not Recruiting
NCT02428296Human BioSciencesSirolimus

Study of Sirolimus Therapy for Segmental Overgrowth Caused by Somatic PI3K Activation

Start: Apr 2015Est. completion: Feb 201839 patients
Phase 2Completed
NCT06975618HaiHe BiopharmaCYH33

Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Preliminary Efficacy of CYH33 in Patients With PIK3CA-related Overgrowth Spectrum (PROS) and PIK3CA-related Vascular Malformations (PRVM)

Start: Aug 2023Est. completion: Dec 2029141 patients
Phase 1/2Recruiting
NCT04085653Sandozalpelisib

Managed Access Programs for BYL719, Alpelisib

N/AAvailable
NCT04285723Sandozalpelisib

Retrospective Chart Review Study of Patients With PIK3CA-Related Overgrowth Spectrum Who Have Received Alpelisib

Start: Jun 2020Est. completion: Apr 202157 patients
N/ACompleted

Phase Legend

Preclinical— Lab & animal studies
Phase 1— Safety & dosing
Phase 2— Efficacy testing
Phase 3— Large-scale trials
On Market— Approved & available

Key Insights

3 actively recruiting trials targeting 865 patients
Small Molecule is the dominant modality (100% of programs)
5 companies competing in this space

The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.