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.
NCT01822613 ↗	Study of Safety & Efficacy of the Combination of LJM716 & BYL719 in Patients With Previously Treated Esophageal Squamous Cell Carcinoma (ESCC)	Completed	Novartis Pharmaceuticals	Phase 1	2013-07-26	To study the safety and efficacy of the combination of LJM716 and BYL719 against currently available treatments of physician's choice in previously treated esophageal squamous cell carcinoma patients.
NCT01923168 ↗	Study of Letrozole With or Without BYL719 or Buparlisib, for the Neoadjuvant Treatment of Postmenopausal Women	Completed	Novartis Pharmaceuticals	Phase 2	2014-03-11	The purpose of the study was to determine whether treatment with a PI3K inhibitor plus letrozole led to an increase in pathologic clinical response and Objective Response Rate compared to treatment with placebo plus letrozole in patients with Breast cancer.

Clinical Trial Conditions for Alpelisib

Condition Name

Condition Name for Alpelisib
Intervention	Trials
Breast Cancer	20
Metastatic Breast Cancer	10
Advanced Breast Cancer	8
HER2-negative Breast Cancer	7

Condition MeSH

Condition MeSH for Alpelisib
Intervention	Trials
Breast Neoplasms	44
Neoplasms	8
Carcinoma	8
Carcinoma, Squamous Cell	6

Clinical Trial Locations for Alpelisib

Trials by Country

Trials by Country for Alpelisib
Location	Trials
United States	194
Italy	79
Spain	44
China	36
Japan	26

Trials by US State

Trials by US State for Alpelisib
Location	Trials
California	15
Texas	14
New York	14
Massachusetts	11
Missouri	10

Clinical Trial Progress for Alpelisib

Clinical Trial Phase

Clinical Trial Phase for Alpelisib
Clinical Trial Phase	Trials
Phase 4	1
Phase 3	10
Phase 2/Phase 3	1

Clinical Trial Status

Clinical Trial Status for Alpelisib
Clinical Trial Phase	Trials
Recruiting	29
Not yet recruiting	22
Completed	5

Clinical Trial Sponsors for Alpelisib

Sponsor Name

Sponsor Name for Alpelisib
Sponsor	Trials
Novartis Pharmaceuticals	30
Novartis	9
National Cancer Institute (NCI)	3

Sponsor Type

Sponsor Type for Alpelisib
Sponsor	Trials
Industry	68
Other	40
NIH	3

Alpelisib: Clinical Trials, Market Analysis, and Projections

Introduction to Alpelisib

Alpelisib, also known by its brand names Piqray, Pivikto, and Trezilent, is a phosphatidylinositol 3-kinase (PI3K) inhibitor developed by Novartis. It is used primarily in the treatment of various cancers, most notably advanced breast cancer, by targeting the PI3K alpha pathway, which is often mutated in cancer cells[3].

Clinical Trials Update

Current Studies and Objectives

Alpelisib is currently under several clinical trials to expand its therapeutic applications and assess its efficacy and safety in different patient populations.

Lymphatic Malformations

A Phase II/III multi-center study (NCT05948943) is ongoing to evaluate the efficacy, safety, and pharmacokinetics of alpelisib in pediatric and adult patients with PIK3CA-mutated lymphatic malformations. This study is divided into two stages: Stage 1 involves a 24-week open-label core phase to select the optimal dose for Stage 2, which will be a 24-week randomized, double-blind, placebo-controlled confirmatory phase[1].

Advanced Breast Cancer

A Phase III study (NCT05038735) is designed to assess the efficacy and safety of alpelisib in combination with fulvestrant for men and postmenopausal women with hormone receptor-positive, HER2-negative advanced breast cancer who have a PIK3CA mutation. This study aims to complement previous data from the SOLAR-1 trial and will randomize approximately 234 participants to either alpelisib plus fulvestrant or a placebo plus fulvestrant[4].

Other Indications

Alpelisib is also under development for various other indications, including metastatic breast cancer, endometrial cancer, ovarian cancer, and PIK3CA-related overgrowth spectrum disorder (PROS). These studies are at different phases, with some in Phase II, evaluating the drug's potential in these additional cancer types[3].

Market Analysis

Market Size and Growth

The alpelisib market is projected to grow significantly over the next decade. As of 2024, the market size is estimated to be in the millions of USD, with a compound annual growth rate (CAGR) expected to drive the market to higher values by 2034. The exact CAGR and market size projections vary slightly across reports, but all indicate a strong growth trajectory[2][5].

Regional Market Performance

• North America: Currently dominates the alpelisib market with a significant share, driven by advanced healthcare infrastructure and high healthcare expenditures[2][5].
• Europe: Market expansion in Europe is facilitated by substantial government programs that cover cancer treatments. Countries like Germany, France, and the UK are key contributors to this growth[2].
• Asia Pacific: This region is expected to be the fastest-growing segment during the forecast period, driven by an extensive patient pool, increasing disposable income, and better healthcare facilities. China and India are particularly significant due to rising cancer incidence rates and improved access to advanced therapies[2].

Distribution Channels

The distribution of alpelisib is expected to occur through various channels, including drug stores, mail order pharmacies, and online platforms. Each channel is anticipated to contribute to the overall market revenue, with online pharmacies potentially seeing significant growth due to convenience and accessibility[2].

Key Market Players

Novartis AG is the primary company driving the alpelisib market, given its role in developing and marketing the drug. Other pharmaceutical companies may also be involved, but Novartis remains the dominant player[2].

Projections and Future Outlook

Market Forecast

The alpelisib market is forecasted to reach substantial revenue by 2034, driven by its expanding therapeutic applications and growing demand in various regions. The market is expected to benefit from personalized medicine and precision treatment approaches, particularly in cancer therapy[2][5].

Regulatory and Clinical Milestones

The success of alpelisib in clinical trials, especially in Phase II and Phase III studies, will be crucial for its future market performance. GlobalData's report indicates that Phase II drugs for metastatic breast cancer have a 32% phase transition success rate, which suggests that alpelisib's progression to Phase III and eventual approval will be closely watched[3].

Competitive Landscape

The competitive landscape for PI3K inhibitors is evolving, with several other drugs in development. However, alpelisib's specific targeting of the PI3K alpha pathway and its established use in advanced breast cancer give it a strong position in the market. Continued innovation and expansion into other cancer types will be key to maintaining its market share[2][3].

Key Takeaways

• Clinical Trials: Alpelisib is undergoing several clinical trials, including studies for lymphatic malformations and advanced breast cancer.
• Market Growth: The alpelisib market is projected to grow significantly, driven by its therapeutic applications and regional demand.
• Regional Performance: North America currently dominates, but the Asia Pacific region is expected to be the fastest-growing segment.
• Distribution Channels: Drug stores, mail order pharmacies, and online platforms will contribute to market revenue.
• Key Players: Novartis AG is the primary company driving the market.

FAQs

What is alpelisib used for?

Alpelisib is primarily used in the treatment of advanced breast cancer, particularly in patients with hormone receptor-positive, HER2-negative, and PIK3CA-mutated tumors. It is also being developed for other cancer types and conditions like lymphatic malformations[3].

What is the current stage of alpelisib clinical trials?

Alpelisib is in various stages of clinical trials, including Phase II and Phase III studies for different indications such as lymphatic malformations and advanced breast cancer[1][4].

Which regions are expected to drive the growth of the alpelisib market?

North America currently dominates the market, but the Asia Pacific region is expected to be the fastest-growing segment due to an extensive patient pool and improving healthcare facilities[2].

Who are the key players in the alpelisib market?

Novartis AG is the primary company driving the alpelisib market, given its role in developing and marketing the drug[2].

What is the projected market size of alpelisib by 2034?

The exact market size projection varies, but it is expected to reach substantial revenue by 2034, driven by its expanding therapeutic applications and growing demand in various regions[2][5].

Sources

1. Novartis Clinical Trials: "Alpelisib in Pediatric and Adult Patients With Lymphatic Malformations" - Novartis.
2. We Market Research: "Alpelisib Market Research Report 2024-2034" - We Market Research.
3. Pharmaceutical Technology: "Alpelisib by Novartis for Metastatic Breast Cancer: Likelihood of Approval" - Pharmaceutical Technology.
4. Novartis Clinical Trials: "Study to Assess the Efficacy and Safety of Alpelisib Plus Fulvestrant" - Novartis.
5. Cognitive Market Research: "Global Alpelisib Market Report 2024 Edition" - Cognitive Market Research.