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ZIOPHARM Oncology’s Orphan Drug Palifosfamide

February 20, 2013

This is the third of a series of Orphan Druganaut Blog Posts that will recap the orphan drug companies at the 15th Annual BIO CEO & Investor Conference, held February 11 – 12, 2013, in New York City. This Blog Post reviews the top oncology orphan drug candidate from ZIOPHARM Oncology. On February 12, 2013, ZIOPHARM Oncology’s CEO, Dr. Jonathan J. Lewis, gives a presentation (Webcast available).

ZIOPHARM Oncology is a biopharmaceutical company with a focus on developing new cancer therapies. There are several drugs in the company’s drug pipeline. The company’s lead compound, Palifosfamide, is in the late-stage of development as first-line treatment of metastatic Soft Tissue Sarcoma (STS). There has not been a 1st line therapy drug in 30 years for STS.  The drug is also being developed for treatment of Small Cell Lung Cancer (SCLC). Palifosfamide has orphan drug designation for STS in both the US and the EU.

Palifosfamide in combination with Doxorubicin is currently in a Phase III trial, Picasso III,  for STS. A second Phase III trial, The MATISSE Study, is evaluating Palifosfamide in combination with Carboplatin and Etoposide (PaCE) for SCLC.

On February 12, 2013, ZIOPHARM Oncology announces that the Phase III (PICASSO III) trial of Palifosfamide in first-line metastatic STS reaches the target number of Progression-Free Survival (PFS) events. PICASSO III is an international, randomized, double-blind, placebo-controlled trial whose primary endpoint is PFS. ZIOPHARM Oncology is to announce topline results from this trial during the last week of March 2013. Shares gain 18% on February 12, 2013, after the company makes its Phase III PICASSO 3 trial announcement.

According to Bloomberg, Dr. Lewis says in an interview that the company is in talks with potential partners to market Palifosfamide.  According to Bloomberg, Dr. Lewis says that a “partnership may happen before the data is presented, or more logically after they are known.”

There is a high unmet medical need for new drugs to treat STS. In April 2012, GlaxoSmithKline’s orphan drug Votrient (Pazopanib) won approval as a 2nd-line therapy for STS, followed by the EU August 2012 approval.

STS Facts :

  1. Is a cancer of the muscle, fat, and other body tissues.
  2. Approximately 9,000 US patients & 14,000 EU patients in the last year
  3. Death rate is high because STS tumors are usually not found until the development of metastasis – statistical median survival of 8 – 12 months.

Upcoming Catalysts For Palifosfamide :

  1. Phase III PICASSO trial topline results to be announced last week of March 2013
  2. Phase III MATISSE trial for SCLC is currently enrolling patients
  3. Possible NDA submission by the end of 2013 for STS.

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. Administered intravenously
  3. Receives FDA Orphan Drug Designation (ODD) in May 2008 for treatment of STS
  4. Receives European Medicines Agency (EMA) orphan designation in December 2008 for STS
  5. Composition of matter exclusivity through 2029
  6. Ziopharm Oncology is working on an oral capsule form.


Sarcoma Foundation of America February 19, 2013 article titled, “Gauging Ziopharm’s Chances for Positive Sarcoma Drug Trial Results”

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

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