CLINICAL TRIALS PROFILE FOR GRANIX

Biosimilar Clinical Trials for GRANIX

This table shows clinical trials for biosimilars. See the next table for all clinical trials

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT02098109 ↗	Non-inferiority Study of XM02 Filgrastim (Granix) and Filgrastim (Neupogen) in Combination With Plerixafor for Autologous Stem Cell Mobilization in Patients With Multiple Myeloma or Non-Hodgkin Lymphoma	Completed	Washington University School of Medicine	Phase 2	2014-08-20	This study will compare the results of stem cell mobilization using drugs called filgrastim (Neupogen) and plerixafor with the results of stem cell mobilization using drugs called XM02 filgrastim (Granix) and plerixafor.

All Clinical Trials for GRANIX

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT02343666 ↗	HIV-Resistant Gene Modified Stem Cells and Chemotherapy in Treating Patients With Lymphoma With HIV Infection	Withdrawn	National Cancer Institute (NCI)	Phase 1	2016-08-15	This pilot phase I trial studies the side effects and best dose of human immunodeficiency virus (HIV)-resistant gene modified stem cells in treating HIV-positive patients who are undergoing first-line treatment for Hodgkin or Non-Hodgkin Lymphoma. Stem cells are collected from the patient and HIV-resistance genes are placed into the stem cells. The stem cells are then re-infused into the patient. These genetically modified stem cells may help the body make cells that are resistant to HIV infection.
NCT02343666 ↗	HIV-Resistant Gene Modified Stem Cells and Chemotherapy in Treating Patients With Lymphoma With HIV Infection	Withdrawn	National Heart, Lung, and Blood Institute (NHLBI)	Phase 1	2016-08-15	This pilot phase I trial studies the side effects and best dose of human immunodeficiency virus (HIV)-resistant gene modified stem cells in treating HIV-positive patients who are undergoing first-line treatment for Hodgkin or Non-Hodgkin Lymphoma. Stem cells are collected from the patient and HIV-resistance genes are placed into the stem cells. The stem cells are then re-infused into the patient. These genetically modified stem cells may help the body make cells that are resistant to HIV infection.
NCT02112045 ↗	A Study of Granix to Disrupt the Bone Marrow Microenvironment in Patients With Multiple Myeloma Undergoing Autologous Transplantation	Terminated	Washington University School of Medicine	Phase 2	2015-01-20	This randomized phase II trial compares how well adding XMO2 Filgrastim (Granix) to melphalan before a stem cell transplant works in treating patients with multiple myeloma. Chemotherapy drugs, such as melphalan, are given to prepare the bone marrow for the stem cell transplant. Giving colony-stimulating factors, such as XMO2 Filgrastim (Granix), may help multiple myeloma cells move from the patient's bone marrow to the blood where they may be more sensitive to treatment with melphalan. It is not yet known whether adding XMO2 Filgrastim (Granix) to melphalan before a stem cell transplant will work better than melphalan alone in treating multiple myeloma
NCT02282514 ↗	Stem Cell Transplantation for Stiff Person Syndrome (SPS)	Terminated	Northwestern University	Phase 1/Phase 2	2014-10-01	Non-myeloablative regimens (as the investigators use herein) are designed to maximally suppress the immune system without destruction of the bone marrow stem cell compartment. When using a non-myeloablative regimen recovery occurs without infusion of stem cells and the stem cells are autologous. While not necessary for recovery, stem cell infusion may shorten the interval of neutropenia and attendant complications. Thus in reality there is no transplant only an autologous supportive blood product. Based on our encouraging results of non-myeloablative hematopoietic stem cell transplantation, for patients with multiple sclerosis and chronic inflammatory demyelinating polyneuropathy, the investigators will investigate the role of non-myeloablative hematopoietic stem cell transplantation for patients with SPS who require assistance to ambulate.
NCT02098109 ↗	Non-inferiority Study of XM02 Filgrastim (Granix) and Filgrastim (Neupogen) in Combination With Plerixafor for Autologous Stem Cell Mobilization in Patients With Multiple Myeloma or Non-Hodgkin Lymphoma	Completed	Washington University School of Medicine	Phase 2	2014-08-20	This study will compare the results of stem cell mobilization using drugs called filgrastim (Neupogen) and plerixafor with the results of stem cell mobilization using drugs called XM02 filgrastim (Granix) and plerixafor.
NCT01445821 ↗	Autologous Stem Cell Systemic Sclerosis Immune Suppression Trial	Terminated	Northwestern University	Phase 3	2011-09-15	ASSIST I was the first randomized trial in patients with scleroderma to not just slow disease progression but rather actually reverse it. It is the first treatment to have ever demonstrated reversal of lung disease in scleroderma with improvement in FVC, total lung capacity (TLC), high-resolution computed tomography (HRCT), and QOL. We now, therefore, purpose to compare the ASSIST I conditioning regimen of cyclophosphamide and rATG to a less intense regimen of rATG/cyclophosphamide/Fludarabine. In the new regimen the cyclophosphamide dose is decreased to 120mg/kg (60mg/kg/day x 2) compared to 200mg/kg (50mg/kg/day) in the standard regimen. The lower dose of cyclophosphamide will be less cardiotoxic. This study will determine if the less cardiotoxic regimen will be safer than the standard regimen and as effective as the standard regimen.
NCT00787722 ↗	Hematopoietic Stem Cell Transplant in Devic's Disease	Completed	Northwestern University	Phase 1/Phase 2	2009-10-10	This study is designed to examine whether treating Devic's disease patients with high dose cyclophosphamide together with rabbit antithymocyte globulin (rATG)/rituximab (drugs which reduce the function of the immune system), followed by return of previously collected patient's stem cells will result in improvement in Devic's disease. Stem cells are undeveloped cells that have the capacity to grow into mature blood cells, which normally circulate in the blood stream. The purpose of the intense chemotherapy is to destroy the cells in patient's immune system, which may be causing his/her disease. The purpose of the stem cell infusion is to produce a normal immune system that will no longer attack patient's body. The purpose of study is to examine the safety and efficacy of this treatment. The drugs used in this study treatment are drugs for commonly used for immune suppression.

Clinical Trial Conditions for GRANIX

Condition Name

Condition Name for GRANIX
Intervention	Trials
Multiple Myeloma	3
Devic's Disease	2
Plasma Cell Myeloma	1
Stage III Diffuse Large B-Cell Lymphoma	1

Condition MeSH

Condition MeSH for GRANIX
Intervention	Trials
Lymphoma	4
Neoplasms, Plasma Cell	3
Multiple Myeloma	3
Lymphoma, Non-Hodgkin	3

Clinical Trial Locations for GRANIX

Trials by Country

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−

Trials by Country for GRANIX
Location	Trials
United States	25

Trials by US State

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−

Trials by US State for GRANIX
Location	Trials
Illinois	8
California	3
Missouri	3
Pennsylvania	2
Washington	2

Clinical Trial Progress for GRANIX

Clinical Trial Phase

Clinical Trial Phase for GRANIX
Clinical Trial Phase	Trials
Phase 3	2
Phase 2/Phase 3	2
Phase 2	5

Clinical Trial Status

Clinical Trial Status for GRANIX
Clinical Trial Phase	Trials
Terminated	5
Recruiting	4
Withdrawn	3

Clinical Trial Sponsors for GRANIX

Sponsor Name

Sponsor Name for GRANIX
Sponsor	Trials
Northwestern University	7
National Cancer Institute (NCI)	3
Washington University School of Medicine	2

Sponsor Type

Sponsor Type for GRANIX
Sponsor	Trials
Other	16
NIH	4

GRANIX: Clinical Trials, Market Analysis, and Projections

Introduction

GRANIX, also known as tbo-filgrastim, is a non-glycosylated recombinant methionyl human granulocyte colony-stimulating factor (r-metHuG-CSF) approved by the FDA for reducing the duration of severe neutropenia in patients with nonmyeloid malignancies receiving myelosuppressive anticancer drugs. Here, we delve into the clinical trials, market analysis, and projections for this drug.

Clinical Trials Overview

Adult Patients

The efficacy and safety of GRANIX were evaluated in several randomized clinical trials involving adult patients with various types of cancer. These trials included patients with breast cancer, lung cancer, and non-Hodgkin’s lymphoma. Here are some key findings:

• Breast Cancer Study: In a multinational, multicenter, randomized, controlled Phase 3 study, 348 chemotherapy-naive patients with high-risk stage II, III, or IV breast cancer received either GRANIX, a placebo, or a non-US-approved filgrastim product. GRANIX significantly reduced the duration of severe neutropenia compared to the placebo group (1.1 days vs. 3.8 days)[1][3][4].

• Lung Cancer and Non-Hodgkin’s Lymphoma Studies: Similar studies were conducted for lung cancer and non-Hodgkin’s lymphoma, with GRANIX demonstrating efficacy in reducing severe neutropenia in these patient populations as well[1][4].

Pediatric Patients

The safety and effectiveness of GRANIX in pediatric patients were assessed in a single-arm clinical trial involving 50 patients with solid tumors treated for chemotherapy-induced neutropenia. The pharmacokinetics and safety profile of GRANIX in pediatric patients were found to be similar to those in adults[1][4].

Adverse Reactions

Common adverse reactions associated with GRANIX include bone pain, myalgia, headache, vomiting, cutaneous vasculitis, and thrombytopenia. Bone pain was the most frequent treatment-emergent adverse reaction, occurring in approximately 3.4% of patients treated with GRANIX[1][3][4].

Market Analysis

Market Entry and Adoption

GRANIX entered the US market as a quasi-biosimilar to Neupogen (filgrastim), another G-CSF product. The adoption rate of GRANIX and other biosimilars in the filgrastim market has been gradual compared to the rapid shift seen with generic drugs.

• Market Share: GRANIX captured a market share of around 5-10% within six months of its launch, with a price discount of approximately 11-23% compared to the branded product Neupogen. This is in contrast to generic drugs, which typically capture over 75% of the market share within six months with price discounts exceeding 40%[2].

• Provider Differences: The uptake of GRANIX and other biosimilars varies among different provider types. Office-based providers have shown a higher adoption rate compared to providers in outpatient hospitals. After three years, the share of biosimilar filgrastim claims, including GRANIX, was higher among office-based providers (54%) than outpatient hospital-based providers (35%)[5].

Competitive Landscape

The filgrastim market is competitive, with multiple biosimilars available, including Zarxio. Zarxio, another biosimilar to Neupogen, has also seen significant adoption, particularly among office-based providers. The total count of filgrastim claims has been increasing since the mid-2016 introduction of biosimilars, indicating a growing acceptance of these products in clinical practice[5].

Projections

Market Growth

The biosimilar market, including GRANIX, is expected to continue growing as more providers become aware of and comfortable with these products. The gradual increase in market share over the past few years suggests a steady adoption rate.

• European Experience: European markets have seen higher biosimilar adoption rates, with biosimilar filgrastim capturing between 45% and 87% of the market share in different countries. This trend is likely to influence the US market as well, driving further growth for GRANIX and other biosimilars[5].

Cost Savings

Biosimilars like GRANIX offer significant cost savings compared to their branded counterparts. While the price discounts may not be as deep as those seen with generic drugs, they still represent a substantial reduction in healthcare costs. This cost-effectiveness is expected to drive increased adoption and market penetration.

Regulatory Environment

The regulatory environment continues to evolve, with ongoing efforts to facilitate the approval and adoption of biosimilars. As regulatory pathways become more streamlined, it is likely that more biosimilars will enter the market, further increasing competition and driving down costs.

Key Takeaways

• Clinical Efficacy: GRANIX has demonstrated significant efficacy in reducing the duration of severe neutropenia in patients with nonmyeloid malignancies.
• Safety Profile: The safety profile of GRANIX is consistent with other G-CSF products, with bone pain being the most common adverse reaction.
• Market Adoption: The adoption rate of GRANIX and other biosimilars is gradual but increasing, with office-based providers showing higher adoption rates.
• Cost Savings: Biosimilars like GRANIX offer substantial cost savings, which is expected to drive further market growth.
• Regulatory Environment: An evolving regulatory environment is likely to facilitate the approval and adoption of more biosimilars.

FAQs

What is GRANIX used for?

GRANIX is used to reduce the duration of severe neutropenia in patients with nonmyeloid malignancies receiving myelosuppressive anticancer drugs.

How is GRANIX administered?

GRANIX is administered subcutaneously at a dose of 5 mcg/kg once daily, starting one day after chemotherapy, for at least five days and up to 14 days or until an ANC of ≥10,000 x 10^6/L after nadir is reached.

What are the common adverse reactions associated with GRANIX?

Common adverse reactions include bone pain, myalgia, headache, vomiting, cutaneous vasculitis, and thrombocytopenia.

How does the market share of GRANIX compare to other biosimilars?

GRANIX captured around 5-10% of the market share within six months of its launch, which is lower than the market share captured by generic drugs but consistent with other biosimilars in the filgrastim market.

What is the expected market growth for GRANIX and other biosimilars?

The market for GRANIX and other biosimilars is expected to grow as more providers adopt these cost-effective alternatives to branded products.

Sources

1. FDA Label: GRANIX (tbo-filgrastim) - accessdata.fda.gov
2. Analysis Group: The Biosimilar Revolution Is Just Beginning in the US - Analysis Group
3. Pharmacy Times: Granix by Teva Pharmaceuticals, Inc - Pharmacy Times
4. FDA Label: GRANIX (tbo-filgrastim) - accessdata.fda.gov (2023 update)
5. AJMC: Provider Differences in Biosimilar Uptake in the Filgrastim Market - AJMC