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Palifosfamide: Ziopharm Oncology Plunges On Trial Results

March 27, 2013

Biotechnology stock movements are affected at various phases of the US FDA’s drug approval process. Big stock moves, either up or down, are associated with several catalysts :

– Releases of trial data

– Briefing documents prior to Advisory Committee meetings (AdComs)

– AdComs themselves

– Prescription Drug User Fee Act (PDUFA) dates.

One of the last catalysts before a final FDA decision, comes from a drug’s Phase III clinical trial data. Positive trial results can result in shares popping. There is also the reverse situation of failed clinical trial results causing shares to sink. Thus, late Phase clinical trials is one of the most critical steps of the orphan drug development life cycle. This is the critical time where demands for data on the drug’s safety and efficacy, comparisons to competitors, and other factors are important.

A recent example of failed Phase III clinical trial data and the impact on the company’s shares is Celsion Corporation’s Phase III HEAT Study. At the end of January 2013, Celsion Corporation announces that the long anticipated results of the Phase III HEAT Study data of orphan drug ThermoDox for primary liver cancer, did not meet the primary endpoint. Per Bloomberg, the stock fell 81% at the close in New York on January 31, 2013. It is the biggest single-day decline since the shares began trading in April 1994.

Ziopharm Oncology is a biopharmaceutical company with a focus on developing new cancer therapies. The company’s key drug in late-stage development is Palifosfamide. The drug has orphan drug designation for Soft Tissue Sarcoma (STS) in both the US and the EU. 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. Shares gain 18% after the company makes its Phase III PICASSO 3 trial announcement.

Ziopharm Oncology announces on March 26, 2013, that Palifosfamide, in its Phase III PICASSO 3 trial for metastatic STS as a first-line treatment, did not meet its primary endpoint of Progression-Free Survival (PFS). The clinical trial’s Independent Data Monitoring Committee (IDMC) recommends that patients continue to be followed for Overall Survival (OS), the secondary endpoint. The company does not expect to continue follow-up for OS. The clinical trial data will be published in a scientific journal.

Per a Reuters article,  Ziopharm Oncology’s CEO and Director, Jonathan Lewis, says that, “We know that based on (the) Progression-Free Survival (Rate), there is no way the drug will get approval anywhere in the world.” Palifosfamide is also being developed for the treatment of Small Cell Lung Cancer (SCLC). Ziopharm Oncology will evaluate all of its Palifosfamide programs.

As a result of the negative outcome of the clinical trial, Ziopharm Oncology makes a decision to terminate development of Palifosfamide for metastatic STS. The company decides “…. to immediately place exclusive strategic focus on its synthetic biology programs, which are being developed in partnership with Intrexon Corporation.”

Ziopharm Oncology shares drop by more than 66%.

Please Note: “AZT” by John Crawford (Photographer); released by the National Institute of Health (NIH), US Federal Gov’t [Public Domain] | Wikimedia Commons.

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

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