PICOPLATIN
Picoplatin Overview

Next generation of platinum-based chemotherapy

Our lead product candidate is Picoplatin, a small molecule, next-generation platinum-based chemotherapy drug previously developed by Poniard and Genzyme (now Sanofi). 
In approximately 20 previous clinical trials, Picoplatin has demonstrated not only comparable efficacy but the ability to overcome platinum resistance and
significantly reduced levels of certain of the side effects associated with platinum chemotherapy in solid tumors. Study data to date suggests that Picoplatin has an
improved safety profile relative to existing platinum-based cancer therapies and can be safely administered in combination with multiple approved oncology products.
Platinum drugs, a backbone of cancer treatment

Platinum-based drugs are prescribed for a significant portion of newly diagnosed cancer patients, generating several billion in annual sales. 
A majority of patients with colorectal, lung, ovarian, head and neck, and bladder cancers receive platinum drugs as a part of their treatment. 
In many cases, these treatments succeed in reducing tumors. Cisplatin, discovered in 19692, became the most widely used chemotherapeutic drug in the US. 
The mechanism of action of platinum drugs and the reasons which cause drug-resistance in cancer patients are considered to be well understood
Limitations of Existing Platinum Therapy

Current platinum therapies suffer from two major shortcomings. First, platinum-based chemotherapy often causes serious side effects.
Second, and even worse, recipients often do not respond to these treatments, resulting in the loss of critical time for alternative therapies.
Therefore, while platinum drugs are widely viewed as effective in the treatment of cancer, improvements are needed.
We believe that our strategy to integrate a new platinum molecule (Picoplatin) with improved properties into pre-existing combination treatment regimens has the potential to improve the success rates of such therapies, especially because we expect that our technology will allow us to identify prospectively patients that will be more likely to respond positively to the treatment.
We believe these factors make Picoplatin a potentially attractive compound for other pharmaceutical companies to partner with us for the commercialization of Picoplatin upon or prior to the completion of our anticipated clinical trials and/or FDA approval, particularly considering
the platinum-based drug market which has been genericized due to the lack of recent developments and innovation.
For example, as the chart below indicates, nearly 50% of patients with colorectal cancer treated by FOLFOX, the standard of care which includes
Oxaliplatin, did not respond to this treatment
Nearly 50% of patients with colorectal cancer treated by FOLFOX, the standard of care which includes Oxaliplatin, did not respond to this treatment.
Median progression-free survival in the first-line treatment for responders is 12 months (range 8.9-23.2) and 2 months for non-responders (range 1.5-4.1).
Standard practice requires up to 6 months to reach response/non-response conclusion, and by then, 10-20% of non-responders could die and all have cancer progressing.
We believe that separating responders from non-responders and treating them differently has the potential to significantly improve the outcome of cancer therapy.
References

1. Johnstone TC, Park GY, Lippard SJ. Understanding and improving platinum anticancer
    drugs-phenanthriplatin. Anticancer Res. 2014 Jan;34(1):471-6.
2. Rosenberg B, VanCamp L, Trosko JE, Mansour VH. Platinum compounds:
    a new classof potent  antitumour agents. Nature. 1969 Apr 26;222(5191):385-6.
3. Dasari S, Tchounwou PB. Cisplatin in cancer therapy: molecular mechanisms ofaction.
    Eur J Pharmacol. 2014 Oct 5;740:364-78.
4. Ruggiero A, Trombatore G, Triarico S, Arena R, Ferrara P, Scalzone M, Pierri F & Riccardi
    R 2013 Platinum compounds in children with cancer:
    toxicity and clinical management. Anti-Cancer Drugs 24 1007-1019.
5. Tang CH, Parham C, Shocron E, McMahon G, Patel N. Picoplatin overcomes resistance to cell
    toxicity in small-cell lung cancer cells previously treated with cisplatin and carboplatin.
    Cancer Chemother Pharmacol. 2011 Jun;67(6):1389-400.