Accessibility Statement Skip Navigation
  • Resources
  • Blog
  • Journalists
  • +44 (0)20 7454 5110
  • Client Login
  • Send a Release
Return to PR Newswire homepage
  • News
  • Products
  • Contact
When typing in this field, a list of search results will appear and be automatically updated as you type.

Searching for your content...

No results found. Please change your search terms and try again.
  • News in Focus
      • Browse News Releases

      • All Public Company News
      • All Multimedia News
      • View All News Releases

      • Regulatory News

      • D/A/CH Regulatory News
      • UK Regulatory News
      • View All Regulatory News

  • Business & Money
      • Auto & Transportation

      • Aerospace & Defense
      • Air Freight
      • Airlines & Aviation
      • Automotive
      • Maritime & Shipbuilding
      • Railroads & Intermodal Transportation
      • Supply Chain/Logistics
      • Transportation, Trucking & Railroad
      • Travel
      • Trucking & Road Transportation
      • View All Auto & Transportation

      • Business Technology

      • Blockchain
      • Broadcast Tech
      • Computer & Electronics
      • Computer Hardware
      • Computer Software
      • Data Analytics
      • Electronic Commerce
      • Electronic Components
      • Electronic Design Automation
      • Financial Technology
      • High Tech Security
      • Internet Technology
      • Nanotechnology
      • Networks
      • Peripherals
      • Semiconductors
      • View All Business Technology

      • Entertain­ment & Media

      • Advertising
      • Art
      • Books
      • Entertainment
      • Film & Motion Picture
      • Magazines
      • Music
      • Publishing & Information Services
      • Radio & Podcast
      • Television
      • View All Entertain­ment & Media

      • Financial Services & Investing

      • Accounting News & Issues
      • Acquisitions, Mergers & Takeovers
      • Banking & Financial Services
      • Bankruptcy
      • Bond & Stock Ratings
      • Conference Call Announcements
      • Contracts
      • Cryptocurrency
      • Dividends
      • Earnings
      • Earnings Forecasts & Projections
      • Financing Agreements
      • Insurance
      • Investments Opinions
      • Joint Ventures
      • Mutual Funds
      • Private Placement
      • Real Estate
      • Restructuring & Recapitalisation
      • Sales Reports
      • Shareholder Activism
      • Shareholder Meetings
      • Stock Offering
      • Stock Split
      • Venture Capital
      • View All Financial Services & Investing

      • General Business

      • Awards
      • Commercial Real Estate
      • Corporate Expansion
      • Earnings
      • Environmental, Social and Governance (ESG)
      • Human Resource & Workforce Management
      • Licensing
      • New Products & Services
      • Obituaries
      • Outsourcing Businesses
      • Overseas Real Estate (non-US)
      • Personnel Announcements
      • Real Estate Transactions
      • Residential Real Estate
      • Small Business Services
      • Socially Responsible Investing
      • Surveys, Polls & Research
      • Trade Show News
      • View All General Business

  • Science & Tech
      • Consumer Technology

      • Artificial Intelligence
      • Blockchain
      • Cloud Computing/Internet of Things
      • Computer Electronics
      • Computer Hardware
      • Computer Software
      • Consumer Electronics
      • Cryptocurrency
      • Data Analytics
      • Electronic Commerce
      • Electronic Gaming
      • Financial Technology
      • Mobile Entertainment
      • Multimedia & Internet
      • Peripherals
      • Social Media
      • STEM (Science, Tech, Engineering, Math)
      • Supply Chain/Logistics
      • Wireless Communications
      • View All Consumer Technology

      • Energy & Natural Resources

      • Alternative Energies
      • Chemical
      • Electrical Utilities
      • Gas
      • General Manufacturing
      • Mining
      • Mining & Metals
      • Oil & Energy
      • Oil & Gas Discoveries
      • Utilities
      • Water Utilities
      • View All Energy & Natural Resources

      • Environ­ment

      • Conservation & Recycling
      • Environmental Issues
      • Environmental Policy
      • Environmental Products & Services
      • Green Technology
      • Natural Disasters
      • View All Environ­ment

      • Heavy Industry & Manufacturing

      • Aerospace & Defence
      • Agriculture
      • Chemical
      • Construction & Building
      • General Manufacturing
      • HVAC (Heating, Ventilation & Air-Conditioning)
      • Machinery
      • Machine Tools, Metalworking & Metallurgy
      • Mining
      • Mining & Metals
      • Paper, Forest Products & Containers
      • Precious Metals
      • Textiles
      • Tobacco
      • View All Heavy Industry & Manufacturing

      • Telecomm­unications

      • Carriers & Services
      • Mobile Entertainment
      • Networks
      • Peripherals
      • Telecommunications Equipment
      • Telecommunications Industry
      • VoIP (Voice over Internet Protocol)
      • Wireless Communications
      • View All Telecomm­unications

  • Lifestyle & Health
      • Consumer Products & Retail

      • Animals & Pets
      • Beers, Wines & Spirits
      • Beverages
      • Bridal Services
      • Cannabis
      • Cosmetics & Personal Care
      • Fashion
      • Food & Beverages
      • Furniture & Furnishings
      • Home Improvement
      • Household, Consumer & Cosmetics
      • Household Products
      • Jewellery
      • Non-Alcoholic Beverages
      • Office Products
      • Organic Food
      • Product Recalls
      • Restaurants
      • Retail
      • Supermarkets
      • Toys
      • View All Consumer Products & Retail

      • Entertain­ment & Media

      • Advertising
      • Art
      • Books
      • Entertainment
      • Film & Motion Picture
      • Magazines
      • Music
      • Publishing & Information Services
      • Radio & Podcast
      • Television
      • View All Entertain­ment & Media

      • Health

      • Biometrics
      • Biotechnology
      • Clinical Trials & Medical Discoveries
      • Dentistry
      • FDA Approval
      • Fitness/Wellness
      • Health Care & Hospitals
      • Health Insurance
      • Infection Control
      • International Medical Approval
      • Medical Equipment
      • Medical Pharmaceuticals
      • Mental Health
      • Pharmaceuticals
      • Supplementary Medicine
      • View All Health

      • Sports

      • General Sports
      • Outdoors, Camping & Hiking
      • Sporting Events
      • Sports Equipment & Accessories
      • View All Sports

      • Travel

      • Amusement Parks & Tourist Attractions
      • Gambling & Casinos
      • Hotels & Resorts
      • Leisure & Tourism
      • Outdoors, Camping & Hiking
      • Passenger Aviation
      • Travel Industry
      • View All Travel

  • Policy & Public Interest
      • Policy & Public Interest

      • Animal Welfare
      • Corporate Social Responsibility
      • Economic News, Trends & Analysis
      • Education
      • Environmental
      • European Government
      • Labour & Union
      • Natural Disasters
      • Not For Profit
      • Public Safety
      • View All Policy & Public Interest

  • People & Culture
      • People & Culture

      • Aboriginal, First Nations & Native American
      • African American
      • Asian American
      • Children
      • Diversity, Equity & Inclusion
      • Hispanic
      • Lesbian, Gay & Bisexual
      • Men's Interest
      • People with Disabilities
      • Religion
      • Senior Citizens
      • Veterans
      • Women
      • View All People & Culture

  • Overview
  • Distribution
  • Paid Placement
  • Multichannel Amplification
  • Disclosure Services
  • SocialBoost
  • Rooms
    • MediaRoom
    • ESG Rooms
  • AI Tools
  • General Enquiries
  • Media Enquiries
  • Partnerships
  • Hamburger menu
  • Cision PR Newswire UK provides press release distribution, targeting, monitoring, and marketing services
  • Send a Release
    • Phone

    • +44 (0)20 7454 5110 from 8 AM - 5:30 PM GMT

    • ALL CONTACT INFO
    • Contact Us

      +44 (0)20 7454 5110
      from 8 AM - 5:30 PM GMT

  • Client Login
  • Send a Release
  • Resources
  • Blog
  • Journalists
  • News in Focus
    • Browse News Releases
    • Regulatory News
  • Business & Money
    • Auto & Transportation
    • Business Technology
    • Entertain­ment & Media
    • Financial Services & Investing
    • General Business
  • Science & Tech
    • Consumer Technology
    • Energy & Natural Resources
    • Environ­ment
    • Heavy Industry & Manufacturing
    • Telecomm­unications
  • Lifestyle & Health
    • Consumer Products & Retail
    • Entertain­ment & Media
    • Health
    • Sports
    • Travel
  • Policy & Public Interest
    • Policy & Public Interest
  • People & Culture
    • People & Culture
  • Client Login
  • Send a Release
  • Resources
  • Blog
  • Journalists
  • Overview
  • Distribution
  • Paid Placement
  • Multichannel Amplification
  • Disclosure Services
  • Cision Communications Cloud®
  • AI Tools
  • Client Login
  • Send a Release
  • Resources
  • Blog
  • Journalists
  • General Enquiries
  • Media Enquiries
  • Partnerships
  • Client Login
  • Send a Release
  • Resources
  • Blog
  • Journalists

Daiichi Sankyo and Plexxikon Announce Study Published in NEJM Demonstrates PLX3397 Induced Prolonged Tumor Regression in Tenosynovial Giant Cell Tumor (TGCT) in Most Patients


News provided by

Daiichi Sankyo Europe , Plexxikon Inc.

30 Jul, 2015, 07:00 GMT

Share this article

Share toX

Share this article

Share toX

- Fifty-two percent of patients achieved a partial response and 30 percent of patients had stable disease following treatment with PLX3397, an investigational CSF-1R inhibitor

- A rare tumor of the joint or tendon sheath, TGCT can lead to significant pain, stiffness, loss of mobility and joint destruction; currently no systemic therapy is approved for TGCT

- Pivotal phase 3 ENLIVEN study currently enrolling patients with locally advanced TGCT to further evaluate efficacy and safety of PLX3397

MUNICH and BERKELEY, California, July 30, 2015 /PRNewswire/ -- Daiichi Sankyo Europe and Plexxikon Inc., a member of the Daiichi Sankyo Group, announced today that The New England Journal of Medicine (NEJM) published clinical trial results demonstrating that the investigational drug, PLX3397, an oral targeted CSF-1R inhibitor, induced prolonged tumor regressions in most patients with tenosynovial giant cell tumor (TGCT), a rare, locally aggressive neoplasm of the joint or tendon sheath.

Following phase 1 dose-escalation, which evaluated the safety and pharmacokinetics of PLX3397 in solid tumors, 23 patients with advanced TGCT were dosed in a single-arm, multi-center, extension cohort to evaluate the safety and efficacy of PLX3397 at 1,000 mg/day. In an intention-to-treat analysis of response by RECIST 1.1 criteria, 12 out of 23 patients achieved a partial response with PLX3397 for an overall response rate of 52 percent [95 percent CI: 32 - 73 percent], and an additional seven patients had stable disease, providing a disease control rate of 83 percent [95 percent CI: 67 - 98 percent]. Responses to PLX3397 usually occurred within four months of treatment and the median duration of response exceeded eight months. Median progression-free survival has not yet been reached in this study.

"TGCT can be a very difficult disease to manage, with treatment options largely limited to surgery to remove as much of the tumor as possible. Despite the best surgical intervention, recurrence of diffuse TGCT is high and the disease may advance to the point where surgery is no longer an option," said William D. Tap, MD, lead author of the study and Chief of the Sarcoma Medical Oncology Service at Memorial Sloan Kettering Cancer Center. "These preliminary results demonstrate that by targeting CSF-1R, PLX3397 may inhibit tumor growth in some patients with TGCT, potentially offering those patients an alternative non-surgical treatment option."

"The patient responses highlighted in the NEJM publication are very encouraging and underscore our rationale for initiating a global, phase 3 study of PLX3397 in TGCT," said Paul S. Lin, MD, MBA, Chief Operating Officer of Plexxikon. "Our TGCT trial offers important preliminary proof-of-concept on the efficacy of selective CSF-1R inhibition. The targeted molecular design of PLX3397 is another hallmark of our efforts to bring new targeted therapies to patients who need them."

About the Study
Patients enrolled in this phase 1 single-arm, multi-center, extension cohort study included those with a histologically confirmed diagnosis of TGCT with demonstrated tumor progression within the past year that was recurrent, inoperable or resectable but requiring extensive surgery. Following baseline assessment, the efficacy of PLX3397 was assessed radiologically every two months utilizing RECIST 1.1 criteria and tumor volume score (TVS), a novel scoring method developed specifically for TGCT.  Patients remained on treatment until disease progression or drug intolerance.

Analysis of efficacy by TVS was available for 14 patients who had a baseline and at least one post-baseline MRI. By TVS response assessment, 11 of 14 patients achieved a partial response (79 percent) and an additional three patients had stable disease, providing a disease control rate of 100 percent. As with the RECIST assessment, patients generally experienced a large decrease in tumor burden by TVS within the first four months that persisted over time. Mean TVS reduction was 61 percent [95 percent CI: 45 - 76 percent].

The most common treatment-related adverse events seen in the study included fatigue, hair color changes, nausea, dysgeusia (abnormal taste), and periorbital edema (swelling around the eyes), which rarely led to drug discontinuation. Treatment-related severe adverse events included fatigue, diarrhea, anemia, hyponatremia, elevated liver enzymes and neutropenia.

About TGCT
Tenosynovial giant cell tumor (TGCT) – a group of neoplasms including pigmented villonodular synovitis (PVNS) and giant cell tumor of the tendon sheath (GCT-TS) – is a rare, usually non-metastatic tumor that affects the synovium-lined joints, bursae, and tendon sheaths, resulting in swelling, pain, stiffness and reduced mobility in the affected joint or limb.1 It is estimated that TGCT has an annual incidence of 11 cases per million.2 Patients are commonly diagnosed in their 20s to 50s,3 and depending on the type of TGCT, women can be up to twice as likely to develop a tumor as men.4 

Primary treatment of TGCT includes surgery to remove the tumor, but in patients with a diffuse form where it can wrap around bone, tendons, ligaments and other parts of the joint, the tumor is more difficult to remove and may require multiple surgeries or joint replacements, eventually advancing to the point where surgery is no longer an option and amputation may be considered. It is estimated that the rate of recurrence in the diffuse form of the disease can be 45 percent or higher in some case series.5 New, effective, non-surgical treatment options are greatly needed for the treatment of TGCT.

About PLX3397
PLX3397 is an investigational novel, oral small molecule that potently and selectively inhibits CSF-1R (colony stimulating factor-1 receptor), which is a primary growth driver of abnormal cells in the synovium that causes TGCT. PLX3397 has not been approved by the U.S. Food and Drug Administration (FDA) or any other regulatory authority for uses under investigation.

A pivotal, phase 3 study of PLX3397 called ENLIVEN is currently enrolling patients with symptomatic TGCT for whom surgical removal of the tumor would be associated with potentially worsening functional limitation or severe morbidity. Approximately 126 patients are planned to be enrolled into the study at approximately 45 study sites in the U.S., Canada, EU, and Australia. For more information about ENLIVEN, please visit the study link at https://www.clinicaltrials.gov/ct2/show/NCT02371369.

PLX3397 has been granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) for the treatment of PVNS and GCT-TS and received Orphan Designation from the European Commission for the treatment of TGCT.

In addition to TGCT, PLX3397 is being evaluated in several other potential clinical indications including glioblastoma, melanoma and breast cancer as well as in combination with anti-PD-1 therapy for advanced melanoma and other multiple solid tumors. PLX3397 also has been selected for the I-SPY 2 TRIAL, a collaborative research effort studying the benefits of adding specific investigational drugs to standard chemotherapy prior to surgery in women with newly diagnosed, locally advanced breast cancer.

About Plexxikon
Plexxikon, a member of the Daiichi Sankyo Group since April 2011, is a leader in the structure-guided discovery and development of novel small molecule pharmaceuticals to treat human disease. Plexxikon is developing a portfolio of preclinical and clinical stage compounds to address significant unmet medical needs in oncology and other therapeutic areas. Plexxikon's Scaffold-Based Drug Discovery™ platform integrates multiple state-of-the-art technologies, including structural screening as a key component that provides a significant advantage over other drug discovery approaches.

About Daiichi Sankyo, Inc.
Daiichi Sankyo Group is dedicated to the creation and supply of innovative pharmaceutical products to address diversified, unmet medical needs of patients in both mature and emerging markets. With over 100 years of scientific expertise and a presence in more than 20 countries, Daiichi Sankyo and its 17,000 employees around the world draw upon a rich legacy of innovation and a robust pipeline of promising new medicines to help people. In addition to its strong portfolio of medicines for hypertension, dyslipidemia, bacterial infections, and thrombotic disorders, the Group's research and development is focused on bringing forth novel therapies in cardiovascular-metabolic diseases, pain management, and oncology, including biologics. For more information, please visit: www.daiichisankyo.com. 

About Daiichi Sankyo Europe
Daiichi Sankyo's European base is located in Munich and has affiliates in 12 European countries in addition to a global manufacturing site located in Pfaffenhofen, Germany. For more information, please visit: www.daiichi-sankyo.eu. 

Contact
Maike Zander (Europe)
Daiichi Sankyo Europe GmbH
Corporate Communications
press@daiichi-sankyo.eu
+49 (89) 7808-569

References:
1 Rao AS, et al. J Bone Joint Surg AM. 1984;66(1):76-94.
2 Myers BW, et al. Medicine (Baltimore). 1980;59(3):223-238.
3 Verspoor FGM, et al. Future Oncol. 2013;10:1515-31.
4 Ravi V, et al. Curr Opin Oncol. 2011;23:361-66.
5 Verspoor FGM, et al. Rheumatology. 2014;53:2063-70.

Related Links

http://www.plexxikon.com

Modal title

Contact PR Newswire

  • +44 (0)20 7454 5110
    from 8 AM - 5:30 PM GMT
  • General Enquiries
  • Media Enquiries
  • Partnerships

Products

  • Content Distribution
  • Multimedia Services
  • Disclosure Services
  • Cision Communications Cloud®

About

  • About PR Newswire
  • About Cision
  • Partnering Opportunities
  • Careers
  • Accessibility Statement
  • APAC
  • APAC - Simplified Chinese
  • APAC - Traditional Chinese
  • Brazil
  • Canada
  • Czech
  • Denmark
  • Finland
  • France
  • Germany
  • India
  • Indonesia
  • Israel
  • Japan
  • Korea
  • Mexico
  • Middle East
  • Middle East - Arabic
  • Netherlands
  • Norway
  • Poland
  • Portugal
  • Russia
  • Slovakia
  • Spain
  • Sweden
  • United States
  • Vietnam

My Services

  • All News Releases
  • Customer Portal
  • Resources
  • Blog
  • Journalists
  • Data Privacy

Do not sell or share my personal information:

  • Submit via Privacy@cision.com 
  • Call Privacy toll-free: 877-297-8921

Contact PR Newswire

Products

About

My Services
  • All News Releases
  • Customer Portal
  • Resources
  • Blog
  • Journalists
+44 (0)20 7454 5110
from 8 AM - 5:30 PM GMT
  • Terms of Use
  • Privacy Policy
  • Information Security Policy
  • Site Map
  • RSS
  • Cookie Settings
Copyright © 2025 PR Newswire Europe Limited. All Rights Reserved. A Cision company.