Recently, ZIOPHARM Oncology, Inc. (ZIOP) received a boost from the US Food and Drug Administration (FDA) when the US agency granted orphan drug status to its candidate darinaparsin (Zinapar). The candidate is being developed to treat patients suffering from peripheral T-cell lymphoma (PTCL). PTCL refers to a biologically diverse group of blood cancers. The incidence of PTCL is more prevalent in men (over 60) than women.
The orphan drug status is granted by the US regulatory agency to drugs developed for treating a rare disease, which affects less than 200,000 people in the US. Furthermore, the designation brings with it seven years of marketing exclusivity in the US following approval. The status also makes the candidate eligible for an accelerated review process and speedy approval wherever appropriate. Additionally, this status also provides several other benefits such as, exemption from user fees, tax credits for research expenses and grant funding for research of rare diseases.
The company, which has recently received the Japanese patent for the candidate, intends to initiate an early-stage study shortly to evaluate the candidate (as a combination therapy) in patients suffering from PTCL. The candidate will be studied in conjunction with Cyclophosphamide, Doxorubicin, Vincristine and Prednisone- the standard of care – to find out the tolerability of the combination. Ziopharm intends to move the combination into a late-stage study by the end of 2011 if it fares well in the early-stage study.
We believe that the successful development and commercialization of the candidate as a front-line therapy for PTCL would boost the top line significantly at Ziopharm. Darinaparsin would address a market with a huge unmet need as currently there are no FDA approved front-line therapies for treating PTCL.
We remind investors that darinaparsin has been proved to be effective in a mid-stage study as a monotherapy in some lymphoma patients who did not respond to other drugs. Currently, Allos Therapeutics’ (ALTH) Folotyn is the only FDA approved drug for treating patients with relapsed or refractory PTCL.
Currently, we have a Neutral stance on Ziopharm in the long-term, which is supported by our Zacks # 3 Rank (short-term ‘Hold’ recommendation) on the stock.
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