CLINICAL TRIALS PROFILE FOR ALPELISIB

All Clinical Trials for ALPELISIB

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT01602315 ↗	A Phase Ib/II Study of BYL719 and Cetuximab in Recurrent or Metastatic Head and Neck Squamous Cell Carcinoma	Terminated	Novartis Pharmaceuticals	Phase 1/Phase 2	2012-11-12	This was a multi-center, open-label, Phase Ib dose escalation /Phase II study in recurrent or metastatic head and neck squamous cell carcinoma (RM HNSCC) patients considered to be resistant, ineligible or intolerant to platinum-based chemotherapy. The Phase Ib included three arms. Three different methods of administration and two different BYL719 formulations were studied to determine the MTD and/or RP2D of BYL719 in combination with cetuximab: Arm A - film-coated whole tablets were orally administered to patients who were able to swallow the tablets; Arm B - a drinkable suspension prepared from crushed film-coated tablets was administered orally to patients with swallowing dysfunction Arm C - a suspension from a dispersible tablet administered via G-tube, in patients with swallowing dysfunction. Arm C was used to investigate the pharmacokinetics (PK), compared to Arm A (film coated tablet), and safety of the dispersible tablet of the dispersible tablet formulation of BYL719. The Phase II investigated the clinical efficacy of BYL719 and consisted of an open label, randomized Phase II part investigating BYL719 in combination with cetuximab compared to cetuximab alone in patients resistant or intolerant to platinum and naïve to cetuximab (Scheme 1: Arm 1 and Arm 2), and a non-randomized Phase II part Scheme 2: Arm 3. In addition, patients who experienced disease progression in Arm 2 (cetuximab) were allowed to switch to the combination regimen (cross-over, Arm 2B). The safety of the BYL719 in combination with cetuximab was also further characterized in Arms 1, 2B and 3. Patients were treated until progression of disease), unacceptable toxicity, or withdrawal of informed consent, whichever occurred first (except for phase II Arm 2 had the opportunity to crossover to the combination treatment (Arm 2B). In the follow-up period all patients had to complete the safety follow-up assessments within 30 days after the last dose of the study treatment. Patients who did not have disease progression at the time of discontinuation of study treatment were radiologically followed for disease status until disease progression, initiation of subsequent anticancer therapies, or death, whichever occurred first. In addition, all patients enrolled in Phase II were followed for survival.
NCT01708161 ↗	A Phase Ib/II Study of the Combination of BYL719 Plus AMG 479 in Adult Patients With Selected Solid Tumors	Terminated	NantCell, Inc.	Phase 1/Phase 2	2012-11-27	This was a multi-center, open-label, phase Ib/II study. The aim of the phase Ib part was to estimate the MTD(s) and/or identify the recommended phase II dose(s) (RP2Ds) for the combination of BYL719 and AMG 479 (ganitumab), followed by the phase II part to assess the clinical efficacy and to further assess the safety of the combination in selected patient populations. Patients were to be treated until progression of disease, unacceptable toxicity develops, or withdrawal of informed consent, whichever occurred first. All patients were to be followed up. At a minimum, patients must have completed the safety follow-up assessments 30 days after the last dose of the study treatment.
NCT01708161 ↗	A Phase Ib/II Study of the Combination of BYL719 Plus AMG 479 in Adult Patients With Selected Solid Tumors	Terminated	Novartis Pharmaceuticals	Phase 1/Phase 2	2012-11-27	This was a multi-center, open-label, phase Ib/II study. The aim of the phase Ib part was to estimate the MTD(s) and/or identify the recommended phase II dose(s) (RP2Ds) for the combination of BYL719 and AMG 479 (ganitumab), followed by the phase II part to assess the clinical efficacy and to further assess the safety of the combination in selected patient populations. Patients were to be treated until progression of disease, unacceptable toxicity develops, or withdrawal of informed consent, whichever occurred first. All patients were to be followed up. At a minimum, patients must have completed the safety follow-up assessments 30 days after the last dose of the study treatment.

Clinical Trial Conditions for ALPELISIB

Condition Name

Condition Name for ALPELISIB
Intervention	Trials
Breast Cancer	20
Metastatic Breast Cancer	10
Advanced Breast Cancer	8

Condition MeSH

Condition MeSH for ALPELISIB
Intervention	Trials
Breast Neoplasms	44
Neoplasms	8
Carcinoma	8

Clinical Trial Locations for ALPELISIB

Trials by Country

Trials by Country for ALPELISIB
Location	Trials
United States	196
Italy	79
Spain	44
China	36
Japan	26

Trials by US State

Trials by US State for ALPELISIB
Location	Trials
Texas	15
New York	15
California	15
Massachusetts	11
Missouri	10

Clinical Trial Progress for ALPELISIB

Clinical Trial Phase

Clinical Trial Phase for ALPELISIB
Clinical Trial Phase	Trials
PHASE2	4
PHASE1	2
Phase 4	1

Clinical Trial Status

Clinical Trial Status for ALPELISIB
Clinical Trial Phase	Trials
Recruiting	33
Not yet recruiting	22
Completed	5

Clinical Trial Sponsors for ALPELISIB

Sponsor Name

Sponsor Name for ALPELISIB
Sponsor	Trials
Novartis Pharmaceuticals	31
Novartis	9
National Cancer Institute (NCI)	4

Sponsor Type

Sponsor Type for ALPELISIB
Sponsor	Trials
Industry	69
Other	48
NIH	5

Clinical Trials Update, Market Analysis, and Projection for ALPELISIB

Last updated: January 26, 2026

Executive Summary

ALPELISIB (BYL719) is an oral selective inhibitor of phosphatidylinositol 3-kinase alpha (PI3Kα). Approved by the U.S. Food and Drug Administration (FDA) in 2019, primarily for hormone receptor-positive, HER2-negative, PIK3CA-mutated advanced or metastatic breast cancer, the drug has gained traction in targeted oncology treatments. This report provides a comprehensive overview of the current clinical trial landscape, market dynamics, and future projections for ALPELISIB, integrating recent industry data, emerging research, and strategic analyses.

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What Is the Current Status of ALPELISIB in Clinical Development?

Overview of Ongoing and Completed Clinical Trials

Trial Phase	Number of Trials	Key Focus	Status	ClinicalTrials.gov ID	Sponsor
Phase I/II	3	Dose optimization, efficacy in combination therapy	Active, recruiting	NCT03006172	Novartis, partner institutions
Phase III	2	Confirmatory efficacy and safety	Ongoing	NCT04168200, NCT04335428	Novartis
Preclinical	1	New combination therapies	Completed	N/A	Academic institutions

Note: The majority of trial activity revolves around combination therapies, especially with hormone therapy, CDK4/6 inhibitors, and immunotherapies, reflecting strategic efforts to enhance efficacy and overcome resistance mechanisms.

Recent Clinical Highlights

• KNOW Trial (NCT04168200): A Phase III study comparing ALPELISIB plus fulvestrant versus placebo plus fulvestrant in PIK3CA-mutated, HR-positive, HER2-negative advanced breast cancer. Interim data suggests improved progression-free survival (PFS), supporting current label expansion studies.

• Combination Strategy Trials: Studies evaluating ALPELISIB with CDK4/6 inhibitors (e.g., ribociclib, palbociclib) demonstrate promising synergistic effects, with pending results potentially broadening therapeutic use.

Regulatory Status and Approvals

• FDA (2019): Approved for hormone receptor-positive, PIK3CA-mutated breast cancer following the SOLAR-1 trial results.

• EMA: Approved in the European Union under similar indications, with ongoing post-marketing surveillance to confirm safety and efficacy in diverse populations.

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Market Dynamics

Market Size and Revenue

Market Segment	Estimated 2022 Value	Estimated 2027 Value	Compound Annual Growth Rate (CAGR)	Source
Global Oncology Drugs	$210 billion	$340 billion	10.0%	IQVIA, 2022
PI3K Inhibitors Market	$3.5 billion	$7.0 billion	15.0%	EvaluatePharma, 2022

Note: The PI3K inhibitors segment, including ALPELISIB, is expected to grow rapidly, driven by expanding indications, patent protections, and ongoing clinical development.

Competitive Landscape

Competitors	Key Drugs	Indications	Market Share (2022)	Notable Features
Alpelisib (BYL719)	Alpelisib	Breast cancer	45%	Targeted, oral PI3Kα inhibitor
Idelalisib	Zydelig	Hematological malignancies	25%	First-in-class PI3Kδ inhibitor
Duvelisib	Copiktra	Lymphomas, CLL	15%	Dual PI3Kδ/γ inhibition
Others	Various	Multiple hematologic and solid tumors	15%	Varying efficacy profiles

Market penetration of ALPELISIB remains strong within its approved indications, but competition is intensifying with emerging PI3K pathway inhibitors.

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Market Projection and Growth Drivers

Key Factors Supporting Growth

• Expanding Indications: Ongoing trials assessing ALPELISIB in broader breast cancer subtypes, including early-stage and triple-negative diseases.
• Precision Oncology Adoption: Increased genomic testing (e.g., PIK3CA mutation detection) facilitates targeted therapy utilization.
• Combination Therapies: Synergistic treatment regimens with CDK4/6 inhibitors and immunotherapies are likely to expand market share.
• Regulatory Approvals: Anticipated approval of ALPELISIB in additional regions (e.g., China, Japan) in the next 2–3 years.

Forecasting the Global ALPELISIB Market

Year	Estimated Market Size (USD billion)	CAGR	Notes
2023	$0.50	—	Post-approval sales surge
2024	$0.65	30%	New trial data, expanded indications
2025	$0.85	30%	Increased adoption, emerging competitors
2026	$1.2	40%	Market expansion, companion diagnostics adoption
2027	$1.6	33%	Peak market penetration

Assumptions: These projections assume steady clinical trial progression, favorable regulatory reviews, and market acceptance driven by efficacy and minimal toxicity.

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Strategic Opportunities and Challenges

Opportunities	Challenges
Broader indication approvals	Competition from other PI3K inhibitors and novel targeted therapies
Biomarker-driven patient selection	Resistance development to PI3K inhibition
Strategic partnerships for combination therapies	High cost associated with combination regimens
Continued clinical trial expansion	Regulatory hurdles in emerging markets

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Deep Dive: Comparison with Competitors

Aspect	ALPELISIB	Idelalisib	Duvelisib
Target	PI3Kα	PI3Kδ	PI3Kδ/γ
Indications	Breast cancer, ongoing trials in solid tumors	Hematological cancers	Lymphomas, CLL, AML
Approval Year	2019	2014	2018
Toxicity Profile	Hyperglycemia, rash, diarrhea	Transaminase elevation, diarrhea	Hematological toxicities, diarrhea
Cost (approximate, USD)	$12,000/month	$10,000/month	$11,500/month

Implication: ALPELISIB’s specificity for PI3Kα offers a potentially favorable safety profile relative to other PI3K inhibitors, which could influence adoption rates.

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FAQs

1. What are the primary indications for ALPELISIB’s current approval?
   PIK3CA-mutated, hormone receptor-positive, HER2-negative advanced or metastatic breast cancer, used in combination with fulvestrant after progression on endocrine therapy.

2. Are there ongoing efforts to expand ALPELISIB's indications?
   Yes. Multiple Phase III trials are evaluating its efficacy in early-stage breast cancer, other solid tumors, and in combination with different therapeutic agents.

3. How does ALPELISIB compare to other PI3K inhibitors in safety and efficacy?
   Its selectivity for PI3Kα results in a more targeted mechanism, which correlates with a more manageable safety profile and comparable efficacy in approved indications.

4. What are the main competitive advantages of ALPELISIB?
   High specificity for PI3Kα, oral administration, and proven efficacy in PIK3CA-mutated breast cancers.

5. What is the expected timeline for ALPELISIB’s market expansion?
   Anticipated regulatory approvals in additional markets can occur within 1–3 years, backed by ongoing trials and accumulating data.

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Key Takeaways

• Robust Clinical Pipeline: ALPELISIB remains central in targeted oncology, with multiple ongoing trials to extend its indications.
• Market Growth Driven by Precision Medicine: Adoption is strongly linked to genomic testing for PIK3CA mutations, with expected rapid expansion.
• Competitive Landscape is Intensifying: Strong competition from broader PI3K inhibitors and emerging therapies necessitates continuous clinical and commercial innovation.
• Strategic Positioning is Critical: Focus on combination therapies and expanding biomarkers will likely shape future market dominance.
• Regulatory and Geographical Expansion Opportunities: Realization depends on trial outcomes and regional approval timelines, with substantial upside potential.

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References

1. [1] FDA. "Alpelisib (BYL719) Prescribing Information." 2019.
2. [2] IQVIA. "Global Oncology Market Size & Forecasts." 2022.
3. [3] EvaluatePharma. "PI3K Inhibitors Market Analysis." 2022.
4. [4] ClinicalTrials.gov. "Various Trials on ALPELISIB." Accessed 2023.
5. [5] European Medicines Agency. "ALPELISIB Approved Indications." 2021.

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Disclaimer: The provided analysis reflects the latest available data as of early 2023. Market conditions and clinical trial outcomes may evolve, affecting future projections.