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Orphan Drug Company Ziopharm Oncology’s Pipeline

December 11, 2012

Ziopharm Oncology is an US pharmaceutical company with its focus on developing new cancer therapies. There are 5 drugs in the company’s robust drug pipeline.

One of these drugs, is Palifosfamide (IV). Palifosfamide in combination with Doxorubicin is currently in a Phase 3 trial, Picasso III, for Soft Tissue Sarcoma (STS). A 2nd Phase 3 trial, MATISSE, is evaluating Palifosfamide in combination with Carboplatin and Etoposide (PaCE) for Small Cell Lung Cancer (SCLC).

Upcoming catalysts for Palifosfamide are :

  1. Phase III PICASSO trial data for STS is expected in the 1st Quarter 2013
  2. Phase III MATISSE trial for SCLC is currently enrolling patients.

Palifosfamide Facts :

  1. Chemotherapy drug that is part of a group known as alkylating agents, which stops tumor growth by “binding to cancer cell DNA and interfering with its function”
  2. Receives FDA Orphan Drug Designation (ODD) May 5, 2008, for treatment of STS
  3. Receives European Medicines Agency (EMA) orphan designation on December 3, 2008, for STS
  4. Composition of matter exclusivity through 2029
  5. Ziopharm is working on an oral capsule form.

Soft Tissue Sarcoma (STS) Facts :

  1. In the EU, effects < 2.5/10,000 people: effects < 125,000 people
  2. In the US, 12,000 new cases are diagnosed each year; < 1% of all cancers
  3. Death rate is high because STS tumors are usually not found until the development of metastasis – statistical median survival of 8 – 12 months.

A second drug, Darinaparsin, is in ongoing clinical studies that is to end in June 2013. Darinaparsin is being developed in partnership with Solasia Pharma, for the treatment of Peripheral T-Cell Lymphoma.

Darinaparsin Facts :

  1. Novel mitochondrial-and hedgehog-targeted agent (organic arsenic)
  2. Receives FDA ODD September 13, 2010, for treatment of Peripheral T-Cell Lymphoma
  3. Receives European Medicines Agency (EMA) orphan designation on April 15, 2011, for Peripheral T-Cell Lymphoma.

Peripheral T-Cell Lymphoma Facts :

  1. In the EU, effects < 0.7/10,000 people; effects < 35,000 people.

Looking at the other 3 drugs in Ziopharm’s Pipeline in combination with Palifosfamide and Darinaparsin, concludes that the Pipeline is strong for the future.


Sarcoma Foundation of America December 10, 2012 article titled, “Ziopharm: Key Oncology Catalyst Approaching”

The Leukemia & Lymphoma Society(LSS)

Ziopharm August 2, 2012 News Release titled, “Ziopharm Oncology Granted New US Patent for Palifosfamide”

Copyright © 2012-2013, Orphan Druganaut Blog.  All rights reserved.

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