Poniard Pharmaceuticals, Inc. is a biopharmaceutical company focused on the development and commercialization of innovative oncology products.

The Company is currently focused on developing picoplatin, a new and differentiated platinum-based chemotherapeutic agent that is in clinical development for multiple cancer indications, treatment combinations and by two routes of administration.

Poniard is headquartered in San Francisco, CA and has an office in Seattle, Washington. Poniard is traded on the NASDAQ Global Market under the symbol PARD.

Picoplatin Overview

Picoplatin is a new and differentiated platinum-based chemotherapeutic agent that is in clinical development for multiple cancer indications, treatment combinations and by two routes of administration. Study data to date suggest 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. Approximately 1,100 patients have received picoplatin. Results obtained to date suggest that hematologic events are common but manageable. Kidney toxicity (nephrotoxicity) and nerve toxicity (neurotoxicity) are less frequent and less severe than is commonly observed with other platinum chemotherapy drugs. Picoplatin has demonstrated anti-tumor activity in a variety of solid tumors including lung, ovarian, colorectal and prostate cancers.

Picoplatin Value Proposition

• Multiple development paths to market
  • Patients previously treated with a platinum agent
  • Platinums not approved or accepted as standard of care
  • First-line head-to-head vs. another platinum
• Poised for registration strategies in multiple indications
  • Leveraging clinical insights from ~1,100 patients data
  • Targeting variety of tumor indications and settings
    • Lung Cancers (Small Cell Lung and Non Small Cell Lung Cancers)
    • Colorectal Cancer
    • Prostate Cancer
    • Ovarian Cancer
• Preferred platinum agent in combination with partner’s product pipeline
  • Potential synergy with partner’s products
  • Portfolio opportunity for multiple indications to mitigate development risks
• Fast-to-market potential with existing data
  • Address near term pipeline gap
  • Near term revenue potential

Colorectal Cancer (CRC)

Colorectal Cancer (CRC) is the third most common cancer in both American men and women, and the third leading cause of cancer death in the U.S. In 2009, an estimated 146,970 new cases of colon and rectal cancer were expected to be diagnosed in the U.S., with an estimated 49,920 deaths.¹ A FOLFOX-containing regimen is the current standard of care but is associated with a high incidence of peripheral sensory neuropathy. Discontinuation of oxaliplatin from FOLFOX is recommended by the National Comprehensive Cancer Network (NCCN) Practice Guidelines in Oncology (v.2.2010) after three months of therapy or sooner for unacceptable neurotoxicity, with other drugs maintained until the time of tumor progression.

During the American Society of Clinical Oncology 2010 Gastrointestinal Cancers Symposium, Poniard presented final results from a randomized, controlled Phase 2 trial evaluating picoplatin as a neuropathy-sparing alternative to oxaliplatin for the first-line treatment of metastatic CRC. The 101 patient study met its primary objective, as picoplatin in combination with 5-fluorouracil and leucovorin in the FOLPI regimen, produced a statistically significant reduction in neurotoxicity, p <0.004, compared to oxaliplatin given in combination with 5-fluorouracil and leucovorin, in the FOLFOX regimen. Neuropathy was 2.5 times more frequent for FOLFOX- treated patients compared to FOLPI-treated patients, and no Grade 3/4 neuropathy occurred in the FOLPI treated patients. Results suggest that FOLPI had anti-tumor activity comparable to FOLFOX, as measured by disease control, PFS and overall survival. Hematologic toxicity was the most frequent adverse event in the FOLPI regimen, but was manageable.

Poniard, in collaboration with its advisory board and the FDA, will leverage insights from its Phase 2 trial to develop a registration strategy for picoplatin in colorectal cancer.

Prostate Cancer

More than 2 million American men are currently living with prostate cancer, and it is the most common cancer, other than skin cancers, among men in the U.S. In 2009, an estimated 192,280 new cases of prostate cancer are expected to be diagnosed. Prostate cancer is the second leading cause of cancer death in American men, behind only lung cancer, with an estimated 27,360 deaths in 2009.¹ All patients with metastatic prostate cancer eventually become refractory to hormone treatment. To date, a docetaxel-containing regimen is the only treatment regimen proven to prolong the lives of patients with castration-resistant prostate cancer (CRPC).

Poniard presented final data from the Company’s Phase 2 clinical trial of picoplatin in 32 men with metastatic CRPC during the American Society of Clinical Oncology 2010 Genitourinary Cancers Symposium. These data indicated that picoplatin, when added to the recommended first-line therapy of docetaxel and prednisone for CRPC, is active as demonstrated by overall survival (21.4 months), progression-free survival (PFS) (7.4 months), and prostate specific antigen (PSA) response rate (78 percent). In contrast, data from published literature demonstrate an overall survival benefit of 18.9 months and a PSA response of 45 percent for patients who received recommended doses of docetaxel and prednisone alone. The Phase 2 trial evaluated the efficacy and safety of intravenous picoplatin (120 mg/meter squared) administered every three weeks in combination with full doses of docetaxel (75 mg/meter squared) with twice daily prednisone (5 mg) as a first-line treatment in chemotherapy naive patients with metastatic CRPC. These results also showed that picoplatin can be safely administered with full doses of docetaxel and prednisone, without neurotoxicity being observed in these patients.

Poniard, in collaboration with its advisory board and the FDA, will leverage insights from its Phase 2 trial to develop a registration strategy for picoplatin in prostate cancer.

Ovarian Cancer

Ovarian cancer accounts for about 3 percent of cancers among women and ranks second among gynecologic cancers. More than 21,000 American women were diagnosed with ovarian cancer in 2009, and approximately 15,000 died from the disease. Women are usually diagnosed with advanced stage disease and the five year overall survival rate for women with ovarian cancer is only 46 percent.¹

The current standard of care for the first-line treatment of ovarian cancer is surgery and a platinum-containing chemotherapy regimen. Although more than 50 percent of patients have a complete response to first-line therapy, 70-90 percent of patients who have advanced disease will require second-line treatment.

Picoplatin was evaluated in two separate clinical trials in women afflicted with ovarian cancer who have become refractory or resistant to a first-line platinum-containing treatment regimen. The first trial, conducted in 94 women, was a Phase 2 single agent clinical trial where patients achieved a 41 percent objective response rate and 8 patients achieved complete responses. In the second trial, picoplatin was safely combined with pegylated liposomal doxorubicin in 16 patients (including 5 ovarian and 1 peritoneal cancer patient). Patients achieved a 31 percent overall response rate (3 patients with ovarian cancer had partial responses, and the patient with peritoneal cancer achieved a complete response).

Poniard, in collaboration with its advisory board and the FDA, will leverage insights from these Phase 2 trials to develop a registration strategy for picoplatin in ovarian cancer.

Small Cell Lung Cancer (SCLC)

SCLC is the most aggressive form of lung cancer and tends to be widespread by the time of diagnosis. According to the American Cancer Society, SCLC accounts for about 10 to 15 percent of all lung cancers. An estimated 32,000 new cases of SCLC were expected to be diagnosed in the United States in 2009, and 52,000 SCLC patients were projected to be treated in the United States in 2008.1 The prognosis for patients with SCLC that have progressed despite chemotherapy is exceedingly poor. Effective second-line treatment for SCLC is a major unmet need. There is no standard chemotherapy for SCLC patients that do not respond to initial platinum-based treatment.

In June 2010, the Company reported full results from the Phase 3 SPEAR (Study of Picoplatin Efficacy After Relapse) trial, which enrolled 401 patients and evaluated intravenous picoplatin in patients who were refractory to, or who progressed within six months following initial treatment with a platinum-based therapy.

• The data analysis showed a statistically significant difference in favor of the picoplatin arm for progression free survival (PFS) in the intent-to-treat population, with a PFS of 9.0 weeks compared to 6.6 weeks in the best supportive care (BSC) alone arm (p-value=0.0281; Hazard Ratio=0.783).
• There was a statistically significant difference in favor of the picoplatin arm for time to progression in the intent-to-treat population, with a median time to progression of 11.3 weeks versus 6.7 weeks in the BSC alone arm (p-value=0.0002; Hazard Ratio=0.610).
• Overall survival in the intent-to-treat population, the primary endpoint of the study, which was based on 320 evaluable events, showed a median overall survival of 20.6 weeks in the picoplatin arm compared to 19.7 weeks in the BSC alone arm (p-value=0.0895, Hazard Ratio= 0.817). As previously reported, the primary endpoint of the study was not met, potentially due to an imbalance in the use of post-study chemotherapy between the picoplatin and BSC alone arms: 27.6 percent of patients in the picoplatin arm received post-study chemotherapy, compared to 40.6 percent in the BSC alone arm (p-value, 0.012).
• Among 273 patients that did not receive post-study chemotherapy, the picoplatin arm (n=194) demonstrated a statistically significant improvement in overall survival compared to the BSC alone arm (n=79). In this population, the picoplatin arm demonstrated a median survival of 18.3 weeks compared to 14.4 weeks in the BSC arm (p-value, 0.0345, Hazard Ratio=0.730).
• Among 294 patients who were refractory or relapsed within 45 days of first-line platinum-based therapy, the picoplatin arm (n=202) demonstrated a statistically significant improvement in overall survival as compared to the BSC alone arm (n=92). In this patient population, the picoplatin arm demonstrated a median overall survival of 21.3 weeks compared to 18.4 weeks in the BSC arm (p-value=0.0173, Hazard Ratio=0.717).
• In the SPEAR trial, picoplatin was well-tolerated and demonstrated a safety profile consistent with prior clinical experience. The most common adverse events were hematological, and included thrombocytopenia, anemia and neutropenia. These events were well managed without clinical sequelea. Adverse events associated with bleeding were mild and did not include any Grade 3/4 events. The incidence of neurotoxicity, a common adverse event among platinum agents, was low, with 0.8 percent of patients reporting grade 3 neuropathy and no incidences of grade 4 neuropathy.

Based on the clinical activity picoplatin demonstrated in the SPEAR trial, Poniard, in collaboration with the FDA and its clinical advisory board, is developing a regulatory strategy for picoplatin in small cell lung cancer.