OBR Radar

The FDA will make a final decision on whether to give Avastin full approval as a first-line treatment for advanced breast cancer patients by Sept. 17. On July 20, an FDA advisory panel, in a 12 to 1 vote, recommended that Avastin's provisional approval as a breast cancer treatment in combination with chemotherapy be revoked on the basis of two follow-up studies that failed to live up to the clinically meaningful results demonstrated in the original study. The Phase 3 E2100 study supporting Avastin's accelerated approval in Feb. '08 showed that adding the drug to paclitaxel added 5.5 months to median progression-free survival. Women in the two later studies also experienced more severe side effects.

Eisai announced on June 1 that its new treatment for advanced breast cancer will get a priority U.S. regulatory review. The FDA will decide whether to approve eribulin by Sept. 30.

Celgene announces on June 30 that it will acquire Abraxis BioScience for $2.9B in cash and stock to expand the company's blood cancer drug business. The deal will include Abraxis's breast cancer drug Abraxane, also being tested in lung and pancreatic cancer, and will expand Celgene's commercial operations into the solid tumor market. Celgene's announces on Aug. 4 that the deal should close in the third or fourth quarter of 2010.

Results from a second large, Phase 3 study announced on July 1 showed that first-line treatment with Avastin plus chemotherapy, followed by the continued use of Avastin alone, increased the time women with previously untreated ovarian cancer lived without the disease worsening (progression-free survival or PFS, the primary endpoint), compared to chemotherapy alone. Genentech didn't say how much longer the women treated with Avastin actually lived; data from the ICON 7 study is expected to be presented at an upcoming medical meeting. Genentech announced at ASCO on June 7 that a similar Phase 3 trial in ovarian cancer, GOG 0218, had also increased PFS for women with advanced ovarian cancer.

Top-line data from a pivotal Phase 2 trial in relapsed and refractory Hodgkin lymphoma being conducted under a Special Protocol Assessment (SPA) is expected. Seattle Genetics plans to submit an NDA in the first half of 2011 to the FDA for the indication.

Interim top-line data from a Phase 2 trial in relapsed and refractory systemic anaplastic large cell lymphoma (ALCL) is anticipated.

Final results of the RADIANT-3 late stage study of Afinitor in advanced pancreatic neuroendocrine tumours (NET) is expected at the 35th ESMO Congress in Milan, Italy in mid-October 2010. Novartis announced on July 1 that everolimus met the study's primary endpoint and significantly extended median progression-free survival from 4.6 to 11.0 months vs. placebo; everolimus also reduced the risk of cancer progression in patients by 65%. Novartis plans worldwide regulatory filings for the indication this year.

Amgen will present detailed results at the 35th ESMO Congress from its Phase 3 SPECTRUM trial, testing Vectibix in combination with chemotherapy as a first-line treatment in patients with squamous cell head and neck cancer versus chemotherapy alone. On August 11, Amgen announced what it called "disappointing" top-line results from the study, which missed its primary endpoint and did not show a statistically significant difference in overall survival for patients when the drug was added to chemotherapy compared to chemotherapy alone.

The sNDA for Sprycel as a treatment for newly diagnosed chronic myeloid leukemia patients in chronic phase (CML-CP) has been granted a priority review by the FDA with a projected action date of October 28, the companies announce on July 8. The filing is based on the results of the pivotal Phase 3 DASISION trial, in which Sprycel demonstrated a superior rate of confirmed complete cytogenic response (CCyR) compared to the standard first-line treatment, Gleevac (77% for Sprycel patients vs. 66% for Gleevac patients by 12 months).

Exact expects to announce data from three validation studies for its genetic screening test for colon cancer on Oct. 29 at the American Association of Cancer Research meeting in Philadelphia. This will be the first validation data measuring the sensitivity of the company's colon cancer test, designed to detect small genetic changes in stool. The results could put the company's test ahead of currently used fecal blood tests in detecting the early warning signs of colon cancer.

An FDA General and Plastic Surgery Devices Panel will meet to review MelaFind, a skin cancer screening device which aids in the detection of early melanoma. The advisory panel was originally scheduled to meet on August 26, but the FDA said it needed more time and moved the date to an as yet-unspecified date in November. MelaFind's Pre-Market Approval (PMA) application was filed with the FDA in June 2009 and is currently under review.

Amgen announces on July 16 that the FDA has granted a priority review to denosumab's BLA for the reduction of skeletal related events in advanced cancer patients. The application's basis are three pivotal Phase 3 head-to-head trials testing denosumab vs. Zometa®. The PDUFA action date for an FDA decision on approval is November 18, 2010.

Researchers expect to present new data on bosutinib as a first-line agent in chronic myelogenous leukemia (CML) in December at the ASH annual meeting. At ASCO, the investigational agent demonstrated clinical efficacy as a 2nd and 3rd line agent in two separate Phase 3 studies of chronic-phase CML patients who had failed previous therapy with either imatinib (Gleevec) or both first-line imatinib and second-line dasatinib (Sprycel).

The anticipated PDUFA date is Dec. 23 for the FDA's response in approving the empowered antibody T-DM1, a next-generation version of Herceptin, in advanced breast cancer, on the basis of trial results announced in December '09 at SABCS. In that study, T-DM1 partially or completely shrank tumors in about one-third of 110 extremely sick patients who had exhausted other treatment options. While safety and efficacy data from the trial were convincing, since there was no control group or placebo group in the study there is a chance that the FDA might ask for additional trial data from more rigorous clinical studies.

The FDA is slated to make an approval decision on ipilimumab as a treatment for patients with previously treated advanced melanoma. The investigational drug received a priority review from U.S. regulators in August 2010.

EpiCept is seeking FDA approval for Ceplene, administered concomitantly with low-dose interleukin-2 (IL-2), for the remission maintenance and prevention of relapse in patients with Acute Myeloid Leukemia (AML) in first complete remission. Phase 3 data showed that patients with AML in complete remission who received up to 18 months of treatment with Ceplene/IL-2 experienced a significantly longer period of leukemia-free survival (LFS) compared to the standard-of-care, which is no treatment. The company submitted an NDA on June 29 and requested priority review. If granted, priority review should result in an FDA decision date in late December 2010.

Results are due from a Phase 3 study of the drug in first-line, or newly treated melanoma patients comparing ipilimumab plus the chemotherapy DTIC against DTIC plus a placebo. Overall survival is the study's primary endpoint. The company presented positive results at ASCO from a Phase 3 study of the drug in previously treated melanoma patients and said it would seek FDA approval based on the data despite questions about the strength of the results.

Results are expected in Q4 '10 from the Phase 3 '147 study in men with hormone refractory prostate cancer which compares denosumab to placebo in prolonging bone-metastasis-free survival, the study's primary endpoint. U.S. regulators have assigned a PDUFA action date of November 18, 2010, for Amgen's BLA for denosumab as a treatment for bone metastases in advanced cancer patients.

The Phase 3 study of zibotentan targets the same patient population as Dendreon's Provenge--men with prostate cancer who no longer respond to hormone treatment but who have minimal bone pain and have not yet started chemotherapy. The trial compares treatment with zibotentan against best supportive care with a primary endpoint of overall survival. If results from the Phase 3 are positive and zibotentan gains FDA approval, doctors will have another treatment choice in addition to Provenge for this group of prostate cancer patients.

Results from a Phase 2 study in patients with non-small cell lung cancer are due by year's end.

Merck announced in mid-June that the FDA had extended its review of the company's application to broaden use of its cervical-cancer vaccine to include older women. The company began seeking regulatory approval more than two years ago to expand Gardasil's use in women up to age 45, but US regulators said they wanted to see longer-term efficacy data, after four years of follow-up. Merck submitted that data in late 2009, but hasn't yet publicly disclosed the results. Merck expects a response from the FDA by the end of the year.

Data from the pivotal trial of the orally administered PHP inhibitor forodesine in cutaneous T-cell lymphoma (CTCL) is expected in 2H10. The single arm study comprises patients with CTCL who have failed three or more previous therapies including bexarotene. The primary outcome measure of the trial is objective response rate. Forodesine is also being tested in a Phase 2 trial for CLL, with results expected in 2H10, and is in a Phase 1 trial for ALL.

Enrollment in the pivotal Phase 3 SUCCEED trial of oral ridaforolimus in patients with metastatic sarcomas was completed in Dec. '09. An independent Data Monitoring Committee (DMC) completed the second interim efficacy analysis and recommended to continue to its final analysis, expected in 2H 2010. Ridaforolimus has been designated both as a fast-track and orphan drug product by the FDA.

An interim analysis from the pivotal Phase 3 VELOUR trial of aflibercept plus FOLFIRI in 2nd line colorectal cancer is expected in 2H10, with full results expected in 2H11. VELOUR may provide the first randomized evidence of the effect of VEGF blockade post-bevacizumab. The primary endpoint of the trial is overall survival.

The last patient was to have completed treatment in Yaupon's pivotal Phase 2 trial of Clearazide for the treatment of early-stage cutaneous T-cell lymphoma (CTCL) by June 2010. The private company announced previously that if study results were positive, it would file an NDA "shortly thereafter" for the indication. Clearazide is a topically-delivered cytotoxic agent granted orphan drug designation for treating cutaneous T-cell lymphoma - mycosis fungoides (CTCL-MF). If approved by the FDA, it would be the first new therapy available for the treatment of early-stage CTCL in almost a decade.

Sanofi's PARP1 inhibitor, iniparib, which was acquired when the company bought BiPar, is currently in a Phase 3 trial for metastatic triple-negative breast cancer with data expected by first-quarter 2011. The drug has fast-track status from the FDA. In updated results from the Phase 2 study presented at SABCS in 2009, patients with the particularly tough-to-treat cancer who received iniparib in combination with carboplatin and gemcitabine lived a median 12.2 months, some 4.5 months longer than those in a control group.

Dendreon's shares tumbled 23% on July 1 in after-hours trading as the Centers for Medicare and Medicaid Services (CMS) said it had begun evaluating Provenge to make a National Coverage Determination (NCD) on the prostate cancer immunotherapy. Medicare administrators will take a full year to review the costly treatment to decide whether to cover it. The CMS plans on holding a meeting before the end of 2010 to review Provenge's clinical data, followed by a proposed NCD being issued on March 30, 2011. A final ruling on coverage is expected in June 2011. Investors were taken by surprise since the CMS almost never issues NCDs for cancer drugs.

Top-line data are currently expected to be reported in a Phase 2b trial of lintuzumab plus low-dose chemotherapy for patients 60 years and older with acute myeloid leukemia (AML) to evaluate whether the combination extends overall survival. Early stage data presented at ASH 2007 showed that some patients had either partial or complete tumor shrinkage, and some were reported to live longer. The only other drug for AML that hit the same target as SGN-33 was Pfizer's Mylotarg, which was pulled off the market in June.

BMY announces that the FDA has accepted, for filing and review, the Biologics License Application (BLA) for ipilimumab for the treatment of adult patients with advanced melanoma who have been previously treated. The company's application receives a priority review with a projected FDA action date of December 25, 2010. The filing is based on results from the primary analysis of the pivotal MDX010-020 Phase 3 trial, presented at ASCO in June 2010, which was the first randomized trial to show an improvement in survival for patients with advanced melanoma.

In Phase 3 results that Amgen called "disappointing," Vectibix failed to meet the primary endpoint, overall survival, of a Phase 3 study testing the drug as a first-line treatment in combination with chemotherapy in squamous cell head and neck cancer. The median survival period for those taking Vectibix was 11.1 months in the trial, vs. nine months for chemotherapy alone, a difference not deemed statistically significant. Shares fell 3.8% in afternoon trading. The drugmaker plans to report detailed results from the study at the upcoming 35th ESMO meeting in October.

Allos announces top-line results from a Phase 2b study testing Folotyn versus Roche/OSI's Tarceva in advanced non-small cell lung patients who had received one or two prior systemic treatments. Patients receiving Folotyn had a 16% reduction in the risk of death compared to Tarceva in the overall patient population. Positive trends in overall survival were observed favoring Foltyn in all other patient subgroups except patients with squamous cell carcinoma and patients previously treated with Lilly's Alimta. Folotyn won accelerated approval from the FDA late last year as the first and only FDA-approved treatment for peripheral T-cell lymphoma (PTCL).

Onyx announces positive top-line results from a pivotal Phase 2b 003-A1 study of single-agent carfilzomib in severely ill multiple myeloma patients whose cancer had worsened after an average of five prior treatment regimens. The study found that 24% of patients, one out of four, responded to carfilzomib for a median duration of 7.4 months. The company's shares jumped 18%; Onyx said it planned to file for the indication based on the study's results and seek an accelerated six-month review from the FDA before the end of 2010. Full details of the trial, including side effects, weren't released, although Onyx said that the drug had been well-tolerated by patients. Onyx added the proteasome inhibitor, which is also being tested in earlier-stage myeloma, to its pipeline when it acquired Proteolix in 2009.

Roche's shares fell to their lowest level in trading in more than a year the day after an FDA advisory panel voted 12 to 1 to recommend that the agency revoke their approval of Avastin for the treatment of advanced breast cancer. The panel unanimously said that two follow-up trials, AVADO and RIBBON 1, taken together, failed to demonstrate enough clinically meaningful benefit for patients to warrant using it given the drugs' risks. The FDA will make a final decision on whether to revoke Avastin's provisional approval for the breast cancer indication, granted in Feb. '08, by Sept. 17.

A pivotal Phase 3 trial of Afinitor in patients with advanced pancreatic neuroendocrine tumors (NET) met its primary goal and significantly extended progression-free survival, more than doubling the time without tumor growth, for patients compared with placebo. Everolimus extended median progression-free survival from 4.6 to 11.0 months vs. placebo and reduced the risk of cancer progression by 65% for patients in the RADIANT-3 study. The data validated earlier studies. A final analysis of the trial is expected to be presented at the 2010 ESMO annual meeting in Milan, Italy in mid-October. Worldwide regulatory filings are planned for 2010.

Following a priority review, the FDA approves Tasigna for newly diagnosed adults patients with Ph+ CML in chronic phase. The approval makes Tasigna the first new therapeutic option approved as a first-line treatment for CML since Glivec. The US approval was based on the results from the ENTESTnd Phase 3 pivotal trial presented at ASCO; in that head-to-head study, 44% of patients taking the newer oral therapy Tasigna had a major molecular response compared with 22% for Glivec after 12 months. The response rate is an indicator usually associated with higher rates of long-term survival.

The FDA approves Jevtana in combination with prednisone for patients with metastatic hormone-refractory prostate cancer (mHRPC) previously treated with a docetaxel-containing treatment regimen. In the Phase 3 TROPIC study, the chemotherapy drug reduced the risk of death by 28% overall in men receiving it plus a steroid compared with men taking another chemotherapy drug plus a steroid. The company said that Jevtana is the first and only therapy to provide a significant survival benefit in the second-line treatment of one of the most difficult-to-treat populations. The ruling came more than three months ahead of the FDA's decision deadline of Sept. 30. Jevtana, administered intravenously, became available in the U.S. on July 19.

Curis's shares plunged 46% when its hedgehog pathway inhibitor failed to meet a primary goal based on topline results from a Phase 2 trial. GDC-0449 failed to extend the time of disease progression or death as a first-line treatment in metastatic colorectal cancer patients. The drug was tested in combination with Avastin and chemotherapy compared to the current standard of care. Curis said it has hopes for the drug in other cancer indications. Results from a Phase 2 study in advanced ovarian cancer are due in 2H '10, along with results from a pivotal Phase 2 trial in basal cell carcinoma in 2011. At ASCO, early-stage trial data was positive for the drug in treating pediatric brain tumors.

Final results from a Phase 2 study of perifosine in previously treated patients with advanced colorectal cancer confirmed a statistically significant improvement in both time to tumor progression (TTP) and overall survival (OS) for patients in the perifosine plus capecitabine arm (P-CAP) vs. those in the placebo plus capecitabine (CAP) arm. The Phase 3 X-PECT trial of perifosine in patients with advanced refractory colorectal cancer is currently enrolling patients.

Data from the TREAT 1 Phase 3 trial presented at ASCO demonstrated that toremifene 80 mg treatment had a more pronounced fracture reduction and better safety profile in men on androgen deprivation therapy for prostate cancer who were younger than age 80. For patients less than age 80 at enrollment, toremifene 80 mg treatment vs. placebo demonstrated a 79.5% reduction in the incidence of new vertebral fractures. GTx expects to initiate TREAT 2, the second Phase 3 trial by year end 2010.

Final clinical data from a Phase 2 study of single agent vosaroxin in women with platinum-resistant ovarian cancer was presented during an oral session at ASCO. Data from the trial demonstrated encouraging, durable anti-tumor activity across all three dose cohorts, with the majority of patients achieving stable disease or an objective response. Sunesis said the promising data warrant further study in ovarian cancer in both the later stage, salvage setting, and in earlier lines of therapy.

Shares of Ziopharm climbed on Fri., May 21, a day after data from a mid-stage study of palifosfamide showed the effectiveness of the company's experimental therapy. The Phase 2 PICASSO study compared palifosfamide, or Zymafos, plus doxorubicin vs. doxorubicin alone in patients with soft-tissue sarcoma. The combination of palifosfamide plus doxorubicin almost doubled progression-free survival over the standard chemotherapy drug alone (7.8 months versus 4.4 months). A pivotal Phase 3 study of the drug in soft tissue sarcoma is anticipated to begin later in 2010.

In one of Amgen's highlighted studies at ASCO, AMG 386, combined with paclitaxel, showed promising antitumor activity and extended progression-free survival in a Phase 2 trial of women with recurrent ovarian cancer. Median progression-free survival (PFS) was 7.2 months for the 10 mg/kg arm of the trial vs. 5.7 months for the 3 mg/kg arm and 4.6 months in the placebo group. Amgen plans to move the AMG 386 into a Phase 3 trial for recurrent ovarian cancer.

Pfizer presented data from a Phase 2 study of bosutinib in patients with Philadelphia chromosome positive chronic phase CML showing that patients who failed to benefit from or were unable to tolerate Gleevac (imatinib) and other tyrosine kinases responded to the experimental therapy, a dual Src and Bcr-Abl kinase inhibitor.

Ahead of an ASCO oral presentation on June 7, Novartis issues a June 4 release announcing 18-month follow-up data from the Tasigna vs. Glivec trial in newly diagnosed Ph+ CML showing that Tasigna significantly surpasses Glivec in slowing disease progression in adult patients. In the Phase 3 ENESTnd study, the first head-to-head comparison of the two oral therapies, Tasigna produced deeper molecular responses and significantly reduced progression to advanced disease, resulting in fewer deaths due to CML. Tasigna is under priority review by the FDA for the indication.

Data from the global Phase 3 EMBRACE study released on June 6 showed that Eisai's chemotherapy drug eribulin significantly improved median overall survival vs. Treatment of Physician's Choice (TPC) in patients with locally recurrent or metastatic breast cancer who had previously received at least two chemotherapeutic regimens. Women who received eribulin survived a median of 2.5 months longer than patients who received TPC (overall survival of 13.12 months versus 10.65 months). The FDA has granted Eisai's application filed for approval of eribulin priority review with a decision expected by Sept. 30.

In Phase 3 study results ipilimumab extended survival in patients with late-stage melanoma who had failed two previous therapies and kept about a quarter of them alive for two years--almost twice the proportion associated with standard treatments. The clinical trial is the first randomized study to find an improvement in survival for advanced melanoma, which has few treatment options. Bristol-Myers' said it would seek FDA approval for the immunotherapy this year.

Data from two separate Phase 3 studies (CALBG 100104 and IFM 2005-02) of Revlimid, presented during an oral session at ASCO, showed that when taken as maintenance therapy following stem-cell transplantation, the multiple myeloma drug reduced the risk of disease progression more than 50 percent in patients and kept the disease in check longer than for those patients who took a placebo.

About 48% of women with platinum-resistant/refractory ovarian cancer saw sustained benefits from treatment with NKTR-102 in a small Phase 2 study reported at ASCO. Twenty-three percent of patients on a dose schedule of once every three weeks saw substantial shrinkage in their tumors, and 38% saw a marked reduction in the ovarian cancer biomarker CA-125.

In Phase 3 (GOG 0218) trial data presented during the ASCO Plenary Session, Avastin met its primary endpoint of progression-free survival (PFS) and slowed tumor growth by nearly 4 months when used for a long period of time in previously untreated women with advanced ovarian cancer. Roche said the results highlight the importance of continuing with Avastin after combining Avastin with chemotherapy in this setting.

In a Phase 1/2 study of crizotinib, Pfizer's oral lung cancer drug reduced tumor size significantly in 57% of NSCLC patients with an ALK gene aberration and stopped the progression of the disease in 87% of those in the study. The gene flaw occurs in only about 5% of lung cancer patients.

In a Phase 3 trial (Myeloma IX) of newly diagnosed multiple myeloma patients adding Zometa to first-line chemotherapy vs. oral clodronate added to chemotherapy significantly improved overall survival, demonstrating a 5.5 month survival improvement. Zometa also reduced the relative risk of skeletal-related events, or SREs, associated with multiple myeloma 24% more than clodronate.

A Phase 3 study of patients carrying the normal KRAS gene with early-stage colon cancer found that there was no survival advantage when they were given Erbitux plus chemotherapy, compared with those given chemotherapy alone. The study results came as a surprise to researchers since Erbitux has been found to improve survival in the same group of KRAS-bearing patients with advanced colon cancer.

Delcath's PHP drug delivery system helped melanoma patients whose cancer had spread to their liver live more than three times as long as patients treated with best available care. The major side effect seen in the Phase 3 trial was bone marrow suppression. Positive study results in progression-free survival (PFS), previously released on April 21, saw Delcath's stock soar 24%.

A Phase 2 trial of ARQ 197 plus Tarceva in previously treated EGFR inhibitor-negative patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) improved overall survival by 24% compared to Tarceva and a placebo, but the results were not statistically significant. ARQ197 was most effective in tumors that were non-squamous, had mutations in a gene called KRAS, or did not have genetic mutations of the epidermal growth factor receptor.

Pharmacyclics announced interim data from a small Phase 1 trial evaluating PCI-32765 as a monotherapy for patients with relapsed aggressive non-Hodgkin's lymphoma (NHL). The latest results showed about 49% of patients, or 17 out of 35 evaluated in the trial, responding to the drug.

A Phase 3 study found that Sprycel is superior to the standard first-line drug, Gleevec, in patients newly diagnosed with chronic myeloid leukemia (CML). Complete cytogenic response--or the disappearance of the CML cells, or tumor--occurred in 77% of Sprycel patients after 12 months compared with 66% for Gleevec, a result considered statistically significant. Sprycel also topped Gleevec in major molecular response rate--46% with Sprycel vs. 28% with Gleevec. The 519-patient study looked at response of the cancer after 12 months, a potential predictor of what long-term patient survival could be with Sprycel, now approved only as a second-line treatment for CML.

Data from the Phase 3 PRIMA study found that the risk of cancer returning in lymphoma patients was cut by half when Rituxan was used as a maintenance treatment for two years. PRIMA enrolled patients with previously untreated advanced follicular lymphoma who responded to initial treatment with Rituxan plus chemotherapy. The study suggests that lymphoma might be treated like a chronic disease with patients maintaining remission through chronic therapy. Roche and Biogen have filed in the US and Europe to extend the drug's current label to include such "maintenance" treatment for patients with advanced follicular lymphoma.

Afinitor helped shrink benign brain tumors in patients with the rare genetic disease, tuberous sclerosis. The small 28-patient study showed a significant reduction in the size of tumors in 75% of patients after six months of treatment with the cancer drug.

Preliminary results from a Phase 1b/2 trial (TDM4373g) combining T-DM1 and Roche's pertuzumab in women with advanced HER2-positive breast cancer showed that the two experimental drugs shrank tumors in nine of 23 women whose cancer had worsened after a median of eight prior treatments with other drugs including Herceptin and Tykerb. Updated safety and preliminary efficacy results on the drug combination from additional patients was reported to be promising. Roche plans to file for accelerated FDA approval of TDM-1, based on positive Phase 2 data, in 2H '10.

Denosumab delayed fractures and complications longer than Novartis AG's Zometa in men whose prostate cancer had spread to their bones, based on Phase 3 study results presented at ASCO. The data showed that men taking denosumab went an average of 20.7 months before their initial bone complication, vs. 17.1 months for men on Zometa. Denosumab, which will be marketed as Prolia, won FDA approval on June 1 as an osteoporosis treatment for postmenopausal women. Amgen is also seeking approval from U.S. regulators to use the drug to prevent skeletal problems in cancer patients.

GTx's shares sank 35% after Phase 3 topline results were reported for its experimental prostate cancer drug in men with a high grade, premalignant lesion of the prostate. Incidence of prostate cancer was lower in men receiving toremifene 20 mg compared to placebo but not "statistically significantly different". The company might conduct a bigger trial after reviewing the data. GTx said it would focus on a 2nd final-stage trial of toremifene's 80 mg dose to prevent bone loss in prostate cancer patients that GTx is conducting to satisfy the FDA.

Dendreon's stock shot up 27%, closing at $50.18 on the Nasdaq, as Provenge's historical FDA approval on April 29 made news. It was the biggest gain in a year, and the announcement also boosted the stocks of companies developing similar products. The first cancer immunotherapy product ever approved in the U.S was greenlighted on the basis of data from the Phase 3 IMPACT trial which showed that Provenge extended the lives of men with castration-resistant prostate cancer by an average 4.1 months. The new therapy is indicated for the treatment of asymptomatic or minimally symptomatic metastatic, castrate-resistant (hormone-refractory) prostate cancer (CRPC). The FDA refused to approve Provenge in 2007 despite an outside advisory committee's unanimous vote in favor of the treatment.

Delcath's stock soared 24% as Phase 3 data was released on its PHP system in patients with melanoma whose cancer has metastasized to the liver, and the late-stage trial met its main goal. The study compared the experimental technology, used with the chemotherapy drug melphalan, to best alternative care. The best alternative care arm showed progression-free survival of only around 2 months compared with the PHP arm, which showed an expected progression-free survival of 7 months.

The FDA approves Tarceva's use as a first-line maintenance treatment in advanced non-small cell lung cancer (NSCLC) after chemotherapy fails. The once-daily pill had been previously approved in 2004 for patients with NSCLC whose cancer worsens after chemotherapy, and for pancreatric cancer. On Dec. 16, FDA advisors had voted 12-1 against expanding the drug's label because they argued that only a “modest improvement" of one-month prolonged survival was shown over the three-month survival benefit when Tarceva was taken as a maintenance treatment.

ChemGenex announces receipt of a Complete Response Letter from the FDA regarding its NDA for Omapro as a chronic myeloid leukemia (CML) treatment for adults who have failed prior therapy with imatinib and have the Bcr-Abl T315I mutation. The company says that regulators haven't requested a new study or asked for increased patient enrollment into Omapro's pivotal study. ChemGenex indicates that the main issues underlying the FDA's non-approval are those raised at the March 22 ODAC meeting and the company will work with the agency to address those matters. ChemGenex said at an April 9th FDA meeting it discussed a path forward to develop a diagnostic test for the T315I mutation that would meet the agency's requirements, another issue that impeded Omapro's approval.

Ahead of an April 23 PDUFA action date, the FDA issues a Complete Response Letter to CTI regarding its NDA for Pixuvri™, formally rejecting its approval for relapsed or refractory aggressive NHL. Earlier on March 22, an FDA advisory panel had unanimously recommended against the drug's approval. CTI says in a statement that the agency's decision is the result of previous concerns raised at the March ODAC meeting and that the FDA has recommended an additional trial to demonstrate pixantrone's safety and effectiveness. The company plans to pursue an expanded access program for pixantrone while it conducts the additional study in NHL. Shares closed on April 8th at 63 cents.

GenVec's stock fell about 75% on the news that the company was discontinuing its pivotal Phase 3 trial of TNFerade in advanced pancreatic cancer based on data from a second interim analysis. Patients treated with TNFerade combined with radiation and chemotherapy had only an 8% reduction in the risk of death compared to treatment with radiation and chemotherapy alone. The first interim analysis in Nov. 2008 demonstrated a survival benefit of 25% for patients treated with the therapy plus standard of care compared to treatment with standard of care alone.

An FDA advisory panel voted 9-0 against recommending CTI's Pixuvri for approval as a treatment for advanced, aggressive non-Hodgkin's lymphoma patients who have failed two previous therapies. CTI's stock experienced its biggest decline in 12 years based on the ODAC meeting's outcome. Advisors, who reviewed data from the Phase 3 EXTEND study of 140 patients, said that the single incomplete trial of pixantrone was insufficient to support approval. Regulators had raised concerns earlier in FDA briefing documents in February concerning the small number of patients in the study, pixantrone's heart safety and the lack of an SPA. The FDA is expected to make an approval decision by April 23.

ChemGenex stock plunged 37% when an FDA advisory panel rejected the company's leukemia drug Omapro for approval in a 7-1 vote. Panel members said that the proposed treatment for chronic myeloid leukemia (CML) patients with a T315I genetic mutation should not be approved until the company shows a "well-characterized" genetic test that will accurately identify patients likely to benefit and that the test should be required and reviewed by the FDA before approval of the drug. The company plans to meet with the FDA on April 9 to review its strategy for having the drug approved.

U.S. health regulators refused to approve APF530 as a treatment for chemotherapy-induced nausea and vomiting (CINV), citing concerns regarding the drug's administration system, and asked for more studies. A.P. Pharma's stock plunged 64% to 75 cents in premarket trade. If approved, APF530 would have been the second drug in the market to treat delayed onset CINV, and competed with Eisai's Aloxi (palonosetron) in the U.S. APF530's commercial launch in 2010 is now unlikely.

In Phase 3 results from the TROPIC study, presented on Mar. 5 at the ASCO-GU symposium, sanofi's experimental chemotherapy drug increased survival by 30% in men with metastatic castration-resistant prostate cancer (mCRPC) whose tumours no longer responded to standard treatment and whose cancer worsened despite treatment with docetaxel-based chemotherapy. The trial showed median overall survival of 15.1 months for patients treated with cabazitaxel in combination with prednisone, a steroid, compared to 12.7 months for patients treated with a combination of the prostate cancer drug, mitoxantrone, and prednisone. Sanofi plans to seek approval for the new drug in both the U.S. and Europe later in 2010; cabazitaxel was recently fast-tracked by the FDA.

Updated results from Provenge's pivotal Phase 3 IMPACT trial, presented at the ASCO-GU Symposium on Mar. 5, demonstrated that the experimental cancer vaccine improved three-year survival of men with advanced prostate cancer by 40% compared with a placebo--patients receiving Provenge lived an average of 4.1 months longer than those on a placebo. The results confirmed earlier data showing that the drug improved survival by 38% in men whose prostate cancer had stopped responding to hormone blockers. Dendreon's stock showed some volatility just before the data came out on Mar. 4 when rumors speculated that another FDA advisory panel would meet to review the drug prior to its scheduled PFUDA date of May 1. Both the company and the FDA confirmed that no meeting was planned.

Novelos' shares sank more than 80% as the company announced that a pivotal Phase 3 trial of NOV-002 in advanced non-small cell lung cancer (NSCLC) failed to meet its primary endpoint of improvement in overall survival. The study evaluated lead product NOV-002 in combination with first-line chemotherapy versus first-line chemotherapy alone. Novelos cited inaccurate statistical modeling leading up to the testing as a cause for the trial's failure, and said it expects to present detailed trial results at a scientific conference later in 2010. The company had hoped to establish a new standard of care in lung cancer treatment with NOV-002, which is an older cancer drug.

Roche's drug was approved by the FDA for a new use—in combination with chemotherapy for people with previously untreated and previously treated CD20-positive chronic lymphocytic leukemia (CLL). The approval was based on data from two Phase 3 studies, CLL8 and REACH, in both patient populations respectively, which showed that Rituxan with chemotherapy significantly extended the time people with CLL lived without the disease worsening compared to chemo alone. In one late-stage study, first-time CLL patients who received Rituxan and chemotherapy went eight months longer before their disease worsened than those who just got chemotherapy.

Denosumab met both its primary and secondary endpoints in a pivotal Phase 3 trial testing it against Zometa in the treatment of bone metastases in advanced prostate cancer patients. Denosumab significantly delayed the time to first skeletal related event and significantly reduced first-and-subsequent skeletal related events compared to Zometa; both results were statistically significant. The study is the final of three pivotal trials in advanced cancer patients investigating denosumab's potential in bone metastases, which will form the basis for an application to be filed with regulators later in 2010.

The FDA issues a a complete response letter regarding the company's NDA for Zuplenz for the prevention of nausea and vomiting associated with chemotherapy, radiotherapy and surgery. Due to an agency-wide restriction on foreign travel in India, the FDA said it was unable to perform an inspection of sites for a bioequivalence study, and could not approve the application at the current time.

The FDA grants accelerated approval on Jan. 29 for a new combination regimen using Tykerb as a first-line, all-oral treatment for women with metastatic breast cancer. The approval broadens Tykerb's label for its use in combination with Novartis AG's Femara® (letrozole) to treat hormone-positive and HER2-positive advanced breast cancer in postmenopausal women for whom hormonal therapy is indicated. Women receiving a combination of the two oral drugs more than doubled the time they lived without the cancer progressing compared with those receiving Femara alone.

Spectrum announces that the FDA has requested additional data on Fusilev, although the company says that regulators did not ask for any additional efficacy studies. In Oct. '09, the FDA issues a Complete Response Letter regarding the company's sNDA for Fusilev's use in advanced metastatic colorectal cancer patients in combination with other treatments. The agency said that the drug did not show non-inferiority to the standard treatment leucovorin. Spectrum plans to submit the additional data to the FDA in 3Q10.

Hexvix, a diagnostic imaging agent used as an adjunct to white light cystoscopy in the detection of non-muscle invasive bladder cancer, is approved pending FDA approval of the PMA for the blue light cystoscopy system that will be used with Hexvix on the US market and final agreements between Photocure and the FDA on labeling, and post-marketing commitments. Photocure expects the pending issues to be agreed upon with the FDA within 1H10.

An FDA advisory panel voted 1 to 12 against Tarceva's approval for its proposed indication as a first-line maintenance treatment in patients with non-small cell lung cancer. Patients receiving Tarceva had a median progression-free survival of 12.3 weeks compared with 11.1 weeks for those on placebo. The panel said the benefit of using Tarceva earlier in treatment than currently called for was modest considering other available treatment options. The PDUFA date for the drug's sNDA, originally January 18, 2010, was pushed back to April 18, 2010 on January 15.

On the heels of a Dec. 12 PDUFA date, Vion announces receipt of a complete response letter from the FDA regarding its NDA for Onrigin as a single agent for remission induction treatment for patients sixty years of age or older with de novo poor-risk acute myelogenous leukemia (AML). On Sept. 1, an ODAC panel had voted unanimously against approval. As anticipated, the FDA asks for an additional randomized study prior to approval to define the efficacy and safety of the drug, among other issues. Vion says it does not have sufficient capital to fund the additional study and that it will evaluate its strategic alternatives.

New long-term follow up-data from two large pivotal Phase 3 studies (N9831 and BCIRG 006) evaluating adjuvant Herceptin in HER2-positive early-stage breast cancer showed that Herceptin reduced the risk of cancer returning by about one-third in women compared to patients receiving chemotherapy alone. In both studies, at least 80% of women receiving one year of Herceptin were alive free of the disease at 5 years follow-up.

In a pivotal Phase 2 study of patients with very advanced HER2-positive breast cancer, T-DM1 shrank tumors in 33% of advanced breast cancer patients who were unresponsive to treatment with other drugs such as Herceptin and Tykerb. Patients also went an average of 7.3 months before their tumors began to grow again. T-DM1, a second-generation version of Herceptin, consists of Herceptin linked to a tumor-killing chemotherapy payload developed by ImmunoGen.

Key data announced at SABCS from the Phase 3 RIBBON-2 study of Avastin in advanced breast cancer showed that women who received the drug in combination with commonly used chemotherapies as second-line treatment had a 28% improvement in progression free survival compared to chemotherapy alone.

Interim data from a phase 1 study of Ariad's AP24534 showed that the multi-targeted kinase inhibitor was well-tolerated and had beneficial anti-tumor activity in treating patients with advanced blood cancers resistant to other therapies. The trial is studying patients who failed prior inhibitor therapy for chronic myeloid leukemia (CML). AP24534 could represent an important treatment option for CML patients who have become resistant or refractory to currently available therapies such as Gleevec, Sprycel and Tasigna.

Onyx's carfilzomib, acquired when the company bought Proteolix, significantly reduced multiple myeloma in 45% of patients in a Phase 2b study ("003") presented at ASH. Patients enrolled in the trial didn't respond to as many as three previous therapies. The drug also showed an improved side effects profile and produced good response rates in patients. Onyx hopes to gain accelerated approval for carfilzomib as a treatment for multiple myeloma in 2010, pitting it against Velcade, a current treatment for the disease.

Data from the Phase 3 MM-015 study of Revlimid in newly diagnosed patients with multiple myeloma at ASH showed that the drug reduced the risk of disease progression by 50%. The data also showed that patients who were treated with Revlimid as a follow-on, maintenance therapy, had a 75% reduction in the risk of their disease progressing compared with those who took an induction regimen alone.

In data reported at ASH, Treanda plus Rituxan stalled the spread of certain lymphomas - all forms of NHL - 20 months longer than a four-drug chemotherapy cocktail paired with Rituxan, the current standard of care. The study could support Treanda's use, in combination with Rituxan, as a first-choice therapy for patients with the slow-growing blood cancers. The drug is currently approved for patients with non-Hodgkin's lymphoma who haven’t responded well to previous therapies and those with chronic lymphocytic leukemia.

A day after Poniard said that picoplatin does not significantly improve survival for advanced lung cancer patients in its pivotal Phase 3 SPEAR trial, the company reports positive data on the drug as a treatment for colorectal cancer.

Results from more detailed preliminary data presented on Nov. 16 of the Phase 3 AGENDA trial of Genasense in patients with advanced melanoma did not show a statistically significant benefit for the co-primary endpoint of progression-free survival, nor for secondary endpoints of overall response or disease-control. The other co-primary endpoint in AGENDA, overall survival, is too early to evaluate, the company said. AGENDA passed a futility analysis, and the Independent Data Monitoring Committee recommended that the trial continue to completion. Pending funding, Genta indicated that the trial should continue until the overall survival analysis could be conducted, which the company anticipates could take place in the second half of 2010.

Almost a week ahead of its Nov. 12 PDUFA date, the FDA approves Gloucester's romidepsin, an HDAC inhibitor, for the treatment of cutaneous T-cell lymphoma (CTCL), a type of non-Hodgkin's lymphoma. On Sept. 2, an FDA advisory panel voted in favor of approving the therapy.

GTx announces on Nov. 2 that it has received a Complete Response Letter from the FDA regarding its NDA for toremifene 80 mg to reduce fractures in men with prostate cancer on androgen deprivation therapy (ADT). U.S. regulators cited clinical deficiencies and asked for more data. GTx has requested a meeting with the FDA to determine the appropriate next steps.

Schering-Plough announced on Oct. 30 that the FDA issued a Complete Response Letter in the company's bid to market PegIntron as a treatment for certain skin cancer patients who also undergo surgery. Schering did not say what the FDA's concerns were. On Oct. 5, an FDA advisory panel narrowly backed PegIntron's additional use in patients with advanced melanoma amid concerns about the drug's considerable side-effects, and also questioned if the drug actually helped people live longer or just increased the time before cancer recurred.

Ahead of its Oct. 31 PDUFA date, Arzerra receives accelerated approval as a treatment for chronic lymphocytic leukemia in patients who have already received other forms of chemotherapy, which are no longer effective at controlling the condition.

D-mab's approval hit a snag when the FDA bypassed the drug's PDUFA decision date of Oct. 19 requesting more information concerning both the company's osteoporosis and cancer-related BLAs. Amgen announced in an Oct. 21 earnings release that the FDA declined to approve the drug in treating bone loss from hormone therapy in breast cancer and prostate cancer patients, saying it wants more safety information coming from additional clinical trials. Despite the delay, analysts said they expect the drug to ultimately be approved.

The FDA approves Votrient, a new oral drug to treat kidney cancer, following an FDA advisory panel's unanimous endorsement in favor of the drug's approval on Oct. 5.

The FDA approves Elitek for use in adults with leukemia, lymphoma or solid tumors who are receiving anti-cancer therapy expected to cause tumor lysis syndrome (TLS), a life-threatening condition, or subsequent elevations of plasma uric acid (PUA), a side effect of cancer treatment.

Gardasil, already indicated for females ages 9 to 26 to prevent cervical cancer, wins FDA approval for preventing genital warts in males in the same age group. An FDA advisory panel voted earlier on Sept. 9 to expand the vaccine's label for use in boys and young men.

The FDA finally approves GSK's Cervarix for the prevention of cervical pre-cancers and cervical cancer associated with oncogenic human papillomavirus (HPV) types 16 and 18 for use in girls and young women (aged 10-25) after an FDA advisory panel greenlights the vaccine on Sept. 9.

Spectrum's stock fell the most in 18 months after the FDA refused to approve Fusilev for a new use in combination with other treatments for advanced metastatic colorectal cancer. U.S. regulators didn't think that the company's submission proved the drug to be as good as the currently available therapy. The company expects to meet with the FDA on their sNDA in January 2010.

Genzyme receives a Complete Response Letter regarding its sNDA for Clolar's potential use in adult patients with acute myeloid leukemia (AML) and requests a meeting with the FDA to discuss a path forward for the drug's approval. An ODAC panel issued a negative review of Clolar on Sept. 1, saying that the company should be required to conduct a larger, comparison study to prove that the drug is safe and effective in older patients.

The FDA grants accelerated approval for Folotyn for use as a single agent for the treatment of relapsed or refractory peripheral T-cell lymphoma (PTCL), making it the first and only drug approved for the indication.

J&J’s Centocor unit announces the receipt of Complete Response Letters for both its sNDA for Doxil in combination with docetaxel for advanced breast cancer and its NDA for Yondelis in combination with Doxil for relapsed ovarian cancer. An ODAC panel voted against recommending both drugs for their proposed indications on July 15 after weighing the modest treatment effects seen in patients treated with each therapy versus the drugs' considerable toxicity. The company says it will respond to the FDA as quickly as possible regarding both drugs.

Spectrum receives FDA approval to expand the use of its non-Hodgkin's lymphoma (NHL) drug Zevalin as a first-line consolidation therapy for NHL patients on Sept. 4 a little ahead of its PDUFA date.

Genentech / Roche announce that Avastin has been approved by the FDA in combination with interferon alfa-2a therapy for patients with first-line metastatic renal cell carcinoma. The approval is based on the global Phase 3 AVOREN study, which showed that previously untreated patients with mRCC who received Avastin plus interferon-alfa had a 67% increase in progression-free survival (PFS) compared to those who received interferon-alfa alone.

The companies announce U.S. product labelling revisions for Erbitux and Vectibix, their respective EGFR monoclonal antibody inhibitors. The revisions are based on retrospective analyses across multiple randomized clinical trials suggesting that anti-EGFR mAbs are not effective for the treatment of patients with metastatic colorectal cancer who test positive for K-ras mutations. The decision follows a Dec. '08 FDA ODAC meeting where the clinical utility of the K-ras gene as a predictive biomarker in patients with mCRC treated with the anti-EGFr antibodies was discussed.

The FDA approves Onsolis, an opioid pain reliever prescribed for certain cancer patients to manage "breakthrough" pain. Regulators also say they'll require a "risk evaluation and mitigation strategy" (REMS) due to Onsolis' drug class.

Alimta wins approval as the first maintenance treatment for advanced lung cancer in patients who have nonsquamous non-small cell lung cancer (NSCLC) whose disease hasn't progressed after four cycles of platinum-based first-line chemotherapy, expanding on its FDA-approved indications.

The FDA grants accelerated approval for Avastin in patients with glioblastoma with progressive disease following prior therapy.

Afinitor, taken orally, is approved as the first treatment for patients with advanced kidney cancer after failure of treatment with either Sutent® (sunitinib) or Nexavar® (sorafenib).

Gleevac expands it use and is approved in the post-surgical treatment of adult patients following complete surgical removal of Kit (CD117)-positive gastrointestinal stromal tumors (GIST).

Mozobil, a stem cell transplant drug, is approved for use in non-Hodgkin’s lymphoma and multiple myeloma patients.

GSK's oral drug is approved for short-term use in patients with chronic immune thrombocytopenic purpura (ITP).

Treanda is approved for non-Hodgkins lymphoma (NHL), its second FDA approval in '08.

Sancuso, a patch for the prevention of chemotherapy-induced nausea and vomiting (CINV), wins FDA approval.

Vidaza receives expanded FDA approval, reflecting new overall survival data in patients with higher-risk myelodysplastic syndromes (MDS).

Velcade wins FDA approval as a first-line treatment for multiple myeloma, widening its use beyond second-line therapy.