MEI Pharma Reports Fiscal Year 2015 Results

On September 2, 2015 MEI Pharma, Inc. (Nasdaq: MEIP), an oncology company focused on the clinical development of novel therapies for cancer, reported results for its fiscal year ended June 30, 2015 (Press release, MEI Pharma, SEP 2, 2015, View Source [SID:1234507384]).

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"Despite a challenging end to the fiscal year, I am quite pleased with how the Company is positioned entering fiscal year 2016," said Daniel P. Gold, Ph.D., President and Chief Executive Officer of MEI Pharma. "While we were obviously disappointed with the top-line data from our randomized study of Pracinostat in front-line myelodysplastic syndrome (MDS), our subsequent findings from the study, combined with the continued maturation of data from our ongoing study in acute myeloid leukemia (AML) and discussions with our clinical advisors, suggest that Pracinostat, in combination with hypomethylating agents (HMA) or other drug candidates, may still play a meaningful role in the treatment of patients with advanced hematologic diseases.

"Meanwhile," continued Dr. Gold, "recent data surrounding the two other drug candidates currently in our pipeline, ME-344 and PWT143, only increase our enthusiasm regarding the potential of these assets and help to inform the next clinical studies anticipated to commence during the first half of calendar year 2016. In the meantime, abstracts relating to all three of our drug candidates have been submitted for presentation at the upcoming American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December 2015. We are poised for an exciting year ahead and I look forward to providing updates on our progress."

Fiscal Year 2015 Company Highlights

Top-line data from randomized Phase II study of Pracinostat in front-line MDS. In March 2015, the Company reported that the addition of Pracinostat to azacitidine (marketed as Vidaza) did not increase the overall CR rate, the study’s primary endpoint, compared to azacitidine alone in this population of intermediate-2 or high-risk patients with previously untreated MDS. Fatigue, gastrointestinal toxicities and myelosuppression occurred more frequently in the combination group and resulted in a higher rate of drug discontinuations compared to azacitidine alone. Data from event-driven endpoints, including overall survival, are still immature. Exploratory follow-up data suggest that patients receiving Pracinostat plus azacitidine for more than four cycles may derive benefit. These data have been submitted for presentation at the ASH (Free ASH Whitepaper) Annual Meeting in December 2015.

Positive data from open-label Phase II study of Pracinostat in AML. The combination of Pracinostat and azacitidine continues to produce a high rate of durable responses in this population of elderly patients with newly diagnosed AML. To date, the primary endpoint of complete response (CR) plus complete response with incomplete blood count recovery (CRi) plus morphologic leukemia-free state (MLFS) has been observed in 27 out of 50 patients (54%), of which 21 (42%) have achieved a CR. Most responses occured within the first two cycles and many continued to improve with ongoing therapy. Median overall survival has not yet been reached in the study; 31 patients (62%) continue to be followed (range: 9-19 months). The combination of Pracinostat and azacitidine was generally well tolerated in this study. The most common treatment-emergent adverse events include febrile neutropenia, thrombocytopenia, nausea and fatigue. Updated response and overall survival data have been submitted for presentation at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December 2015.

Clinical milestone in Phase II study of Pracinostat in refractory MDS. The objective of this study was to determine if the addition of Pracinostat to a HMA can improve clinical responses in MDS patients who progressed while on their HMA alone. Of the first 28 patients who received Pracinostat in combination with azacitidine or decitabine (marketed as Dacogen), one achieved a partial response and two achieved marrow complete responses, exceeding the pre-specified clinical improvement rate for the study. The Company completed enrollment with 39 patients in this group and will continue to follow these patients for response and survival. A second group, patients with stable disease following initial HMA therapy, was closed due to insufficient enrollment. Pracinostat plus azacitidine or decitabine was generally well tolerated in the study. The most common treatment-emergent adverse events included anemia, fatigue and gastrointestinal disorders.

Initiation of Phase II study of Pracinostat in myelofibrosis. The goal of this study is to learn if Pracinostat, when given in combination with ruxolitinib (marketed as Jakafi and Jakavi), can help to control myelofibrosis, a rare disease of the bone marrow. The study, sponsored by the M.D. Anderson Cancer Center, began enrollment earlier this year and is expected to enroll 25 patients.
Cohort expansion in Phase Ib study of mitochondrial inhibitor ME-344. The Company’s study of ME-344 plus topotecan (marketed as Hycamtin) advanced to the cohort-expansion stage after confirming the maximum tolerated dose of the combination in 14 patients. The expansion stage enrolled patients into two cohorts, ovarian cancer and small cell lung cancer, at nine sites in the U.S. and U.K. The combination of ME-344 and topotecan has been generally well tolerated in the study. The most frequent side effects are fatigue and gastrointestinal disturbances. The Phase Ib study enrolled a total of 13 small cell lung cancer patients and 28 ovarian cancer patients. The Company will continue to follow these patients for response and survival.

New findings show enhanced activity of ME-344 when combined with TKI. The Company recently announced results from several pre-clinical studies demonstrating mitochondria-specific effects of ME-344 in cancer cells, including substantially enhanced anti-tumor activity when combined with a vascular endothelial growth factor receptor (VEGFr) tyrosine-kinase inhibitor (TKI) to inhibit both mitochondrial and glycolytic metabolism. These findings will help to inform the next clinical study of ME-344 in combination with a VEGFr TKI.

Encouraging preliminary data from first-in-human study of PWT143. The Company initiated a first-in-human clinical study of PWT143, a highly selective oral inhibitor of phosphatidylinositide 3-kinase (PI3K) delta, in healthy subjects in June 2015. Preliminary data show measurable plasma levels of PWT143 as well as significant on target activity observed at the 10 mg starting dose level. In addition, the pharmacokinetic results suggest the potential for once-daily dosing. These data have been submitted for presentation at the ASH (Free ASH Whitepaper) Annual Meeting in December 2015. The Company expects to initiate a Phase I study of PWT143 in patients with hematologic cancers during the first half of calendar year 2016.

Pharmaceutical industry veteran Kevan Clemens elected to Board of Directors. Dr. Clemens has a long and distinguished career in the pharmaceutical industry, highlighted by his role as head of Global Oncology at Hoffmann-La Roche.

Strengthened balance sheet with $46 million public offering. The net proceeds from the offering will enable the Company to continue to execute on its clinical development programs.

Fiscal Year 2015 Financial Highlights

As of June 30, 2015, MEI Pharma had $63.8 million in cash, cash equivalents and short-term investments, with no outstanding debt. The Company believes its cash, cash equivalents and short-term investments will be sufficient to fund operations through at least calendar year 2016.

Net cash used in operations was $28.1 million for the year ended June 30, 2015, compared to $19.5 million for 2014. Net cash used in operations was $6.7 million for the quarter ended June 30, 2015.

Research and development (R&D) expenses were $23.8 million for the year ended June 30, 2015, compared to $19.3 million for 2014. The increase was primarily due to costs associated with Phase II clinical trials for Pracinostat, as well as costs associated with a Phase I clinical trial for ME-344 and pre-clinical costs related to PWT143. R&D expenses for the year ended June 30, 2015 included share-based compensation of $1.0 million.

General and administrative expenses were $8.9 million for the year ended June 30, 2015, compared to $7.9 million for 2014. The increase primarily relates to higher levels of salaries and benefits.

Net loss was $32.7 million, or $1.16 per share, for the fiscal year ended June 30, 2015, compared to $27.1 million, or $1.35 per share for 2014.

8-K – Current report

On September 2, 2015 MEI Pharma, Inc. (Nasdaq: MEIP), an oncology company focused on the clinical development of novel therapies for cancer,reported results for its fiscal year ended June 30, 2015 (Filing, 8-K, MEI Pharma, SEP 2, 2015, View Source [SID:1234507382]).

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"Despite a challenging end to the fiscal year, I am quite pleased with how the Company is positioned entering fiscal year 2016," said Daniel P. Gold, Ph.D., President and Chief Executive Officer of MEI Pharma. "While we were obviously disappointed with the top-line data from our randomized study of Pracinostat in front-line myelodysplastic syndrome (MDS), our subsequent findings from the study, combined with the continued maturation of data from our ongoing study in acute myeloid leukemia (AML) and discussions with our clinical advisors, suggest that Pracinostat, in combination with hypomethylating agents (HMA) or other drug candidates, may still play a meaningful role in the treatment of patients with advanced hematologic diseases.

"Meanwhile," continued Dr. Gold, "recent data surrounding the two other drug candidates currently in our pipeline, ME-344 and PWT143, only increase our enthusiasm regarding the potential of these assets and help to inform the next clinical studies anticipated to commence during the first half of calendar year 2016. In the meantime, abstracts relating to all three of our drug candidates have been submitted for presentation at the upcoming American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December 2015. We are poised for an exciting year ahead and I look forward to providing updates on our progress."
Fiscal Year 2015 Company Highlights

• Top-line data from randomized Phase II study of Pracinostat in front-line MDS. In March 2015, the Company reported that the addition of Pracinostat to azacitidine (marketed as Vidaza) did not increase the overall CR rate, the study’s primary endpoint, compared to azacitidine alone in this population of intermediate-2 or high-risk patients with previously untreated MDS. Fatigue, gastrointestinal toxicities and myelosuppression occurred more frequently in the combination group and resulted in a higher rate of drug discontinuations compared to azacitidine alone. Data from event-driven endpoints, including overall survival, are still immature. Exploratory follow-up data suggest that patients receiving Pracinostat plus azacitidine for more than four cycles may derive benefit. These data have been submitted for presentation at the ASH (Free ASH Whitepaper) Annual Meeting in December 2015.

• Positive data from open-label Phase II study of Pracinostat in AML. The combination of Pracinostat and azacitidine continues to produce a high rate of durable responses in this population of elderly patients with newly diagnosed AML. To date, the primary endpoint of complete response (CR) plus complete response with incomplete blood count recovery (CRi) plus morphologic leukemia-free state (MLFS) has been observed in 27 out of 50 patients (54%), of which 21 (42%) have achieved a CR. Most responses occurred within the first two cycles and many continued to improve with ongoing therapy. Median overall survival has not yet been reached in the study; 31 patients (62%) continue to be followed (range: 9-19 months). The combination of Pracinostat and azacitidine was generally well tolerated in this study. The most common treatment-emergent adverse events include febrile neutropenia, thrombocytopenia, nausea and fatigue. Updated response and overall survival data have been submitted for presentation at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December 2015.

• Clinical milestone in Phase II study of Pracinostat in refractory MDS. The objective of this study was to determine if the addition of Pracinostat to a HMA can improve clinical responses in MDS patients who progressed while on their HMA alone. Of the first 28 patients who received Pracinostat in combination with azacitidine or decitabine (marketed as Dacogen), one achieved a partial response and two achieved marrow complete responses, exceeding the pre-specified clinical improvement rate for the study. The Company completed enrollment with 39 patients in this group and will continue to follow these patients for response and survival. A second group, patients with stable disease following initial HMA therapy, was closed due to insufficient enrollment. Pracinostat plus azacitidine or decitabine was generally well tolerated in the study. The most common treatment-emergent adverse events included anemia, fatigue and gastrointestinal disorders.

• Initiation of Phase II study of Pracinostat in myelofibrosis. The goal of this study is to learn if Pracinostat, when given in combination with ruxolitinib (marketed as Jakafi and Jakavi), can help to control myelofibrosis, a rare disease of the bone marrow. The study, sponsored by the M.D. Anderson Cancer Center, began enrollment earlier this year and is expected to enroll 25 patients.

• Cohort expansion in Phase Ib study of mitochondrial inhibitor ME-344. The Company’s study of ME-344 plus topotecan (marketed as Hycamtin) advanced to the cohort-expansion stage after confirming the maximum tolerated dose of the combination in 14 patients. The expansion stage enrolled patients into two cohorts, ovarian cancer and small cell lung cancer, at nine sites in the U.S. and U.K. The combination of ME-344 and topotecan has been generally well tolerated in the study. The most frequent side effects are fatigue and gastrointestinal disturbances. The Phase Ib study enrolled a total of 13 small cell lung cancer patients and 28 ovarian cancer patients. The Company will continue to follow these patients for response and survival.

• New findings show enhanced activity of ME-344 when combined with TKI. The Company recently announced results from several pre-clinical studies demonstrating mitochondria-specific effects of ME-344 in cancer cells, including substantially enhanced anti-tumor activity when combined with a vascular endothelial growth factor receptor (VEGFr) tyrosine-kinase inhibitor (TKI) to inhibit both mitochondrial and glycolytic metabolism. These findings will help to inform the next clinical study of ME-344 in combination with a VEGFr TKI.

• Encouraging preliminary data from first-in-human study of PWT143. The Company initiated a first-in-human clinical study of PWT143, a highly selective oral inhibitor of phosphatidylinositide 3-kinase (PI3K) delta, in healthy subjects in June 2015. Preliminary data show measurable plasma levels of PWT143 as well as significant on target activity observed at the 10 mg starting dose level. In addition, the pharmacokinetic results suggest the potential for once-daily dosing. These data have been submitted for presentation at the ASH (Free ASH Whitepaper) Annual Meeting in December 2015. The Company expects to initiate a Phase I study of PWT143 in patients with hematologic cancers during the first half of calendar year 2016.

• Pharmaceutical industry veteran Kevan Clemens elected to Board of Directors. Dr. Clemens has a long and distinguished career in the pharmaceutical industry, highlighted by his role as head of Global Oncology at Hoffmann-La Roche.

• Strengthened balance sheet with $46 million public offering. The net proceeds from the offering will enable the Company to continue to execute on its clinical development programs.
Fiscal Year 2015 Financial Highlights

• As of June 30, 2015, MEI Pharma had $63.8 million in cash, cash equivalents and short-term investments, with no outstanding debt. The Company believes its cash, cash equivalents and short-term investments will be sufficient to fund operations through at least calendar year 2016.

• Net cash used in operations was $28.1 million for the year ended June 30, 2015, compared to $19.5 million for 2014. Net cash used in operations was $6.7 million for the quarter ended June 30, 2015.

• Research and development (R&D) expenses were $23.8 million for the year ended June 30, 2015, compared to $19.3 million for 2014. The increase was primarily due to costs associated with Phase II clinical trials for Pracinostat, as well as costs associated with a Phase I clinical trial for ME-344 and pre-clinical costs related to PWT143. R&D expenses for the year ended June 30, 2015 included share-based compensation of $1.0 million.

• General and administrative expenses were $8.9 million for the year ended June 30, 2015, compared to $7.9 million for 2014. The increase primarily relates to higher levels of salaries and benefits.

• Net loss was $32.7 million, or $1.16 per share, for the fiscal year ended June 30, 2015, compared to $27.1 million, or $1.35 per share for 2014.

Dual Publications in the New England Journal of Medicine Highlight Transformative Potential of Imetelstat in Hematologic Myeloid Malignancies

On September 02, 2015 Geron Corporation (Nasdaq:GERN) reported the publication of two papers in The New England Journal of Medicine (NEJM) in which the company’s telomerase inhibitor, imetelstat, was shown to have disease-modifying activity thought to be associated with selective inhibition of the malignant progenitor cell clones responsible for the underlying disease in two hematologic myeloid malignancies, essential thrombocythemia (ET) and myelofibrosis (MF) (Press release, Geron, SEP 2, 2015, View Source [SID:1234507389]). The papers are available online in the September 3rd issue at www.NEJM.org.

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"The data in the ET and MF study publications in The New England Journal of Medicine provide compelling evidence that use of a telomerase inhibitor, such as imetelstat, may result in ground-breaking changes in how we approach the future treatment of hematologic myeloid malignancies," said John A. Scarlett, Geron’s President and Chief Executive Officer.

In the Phase 2 clinical study evaluating imetelstat in ET, all patients achieved a hematologic response, with the majority achieving a hematologic complete response. Rapid and substantial molecular responses observed in the study suggested therapeutic activity of imetelstat on the growth of malignant progenitor cell clones that drive ET. The safety and efficacy results reported in the NEJM publication are consistent with the preliminary data from this study previously presented in part at the annual meetings of the American Society of Hematology (ASH) (Free ASH Whitepaper) in December 2012 and December 2014, and the European Hematology Association (EHA) (Free EHA Whitepaper) annual meeting in June 2013.

"This study was a first look at what happens when you treat ET patients with a drug that has a totally novel mechanism of action," said Dr. David Snyder, City of Hope, Duarte, CA, and co-principal investigator of the ET study. "In the study, imetelstat had a clinically significant effect on disease burden in ET patients."

"The molecular responses suggest that imetelstat may have broad activity across hematologic myeloid malignancies which warrants further clinical study in other myeloproliferative neoplasms," noted Prof. Dr. med. Gabriela M. Baerlocher, Inselspital, Bern University Hospital and University of Bern, Switzerland, and co-principal investigator of the ET study.

In the Phase 2 pilot study evaluating imetelstat in MF patients, unprecedented complete and partial remissions, including reversal of bone marrow fibrosis and molecular responses, were observed. These results suggest the potential value of telomerase-targeting strategies for the treatment of MF, and identify imetelstat as an active drug in this disease. The safety and efficacy results reported in the NEJM publication are consistent with the preliminary data from this study previously presented in part at the annual meetings of the American Society of Hematology (ASH) (Free ASH Whitepaper) in December 2013 and December 2014.

"The clinical and molecular remissions seen in the study suggest selective anti-clonal activity, not previously documented in current drug treatment of MF," commented Dr. Ayalew Tefferi, Mayo Clinic, Rochester, MN, and principal investigator of the MF pilot study. "A much larger multi-center clinical study is needed to confirm these findings and to further investigate the mechanism by which imetelstat induces clinical responses in some patients."

Future Clinical Development of Imetelstat in Collaboration with Janssen

In November 2014, Geron entered into an exclusive license and collaboration agreement with Janssen Biotech, Inc. (Janssen) to develop and commercialize imetelstat worldwide for indications in oncology, including hematologic myeloid malignancies, and all other human therapeutics uses. Under the collaboration with Janssen, development of imetelstat initially will proceed under a mutually agreed clinical development plan which includes a Phase 2 study in MF and a Phase 2 study in myelodysplastic syndrome (MDS). In addition, the clinical development plan may also include additional, possible registration studies in MF and MDS, and possible exploratory Phase 2 and potential follow on Phase 3 studies in acute myeloid leukemia (AML). Certain regulatory, development, manufacturing and promotional activities are being managed through a joint governance structure, with Janssen responsible for these activities.

In July 2015, Janssen initiated IMbark, a Phase 2 clinical study to evaluate the activity of two dose levels of imetelstat in patients with DIPSS intermediate-2 or high-risk MF who have relapsed after or are refractory to a JAK inhibitor. Up to approximately 80 medical centers across North America, Europe and Asia are planned to participate in this clinical study and currently sites are open to patient enrollment. The study is designed to enroll approximately 200 patients (approximately 100 patients per dosing arm). For more information about the IMbark study, please visit View Source

A Phase 2 clinical study in MDS, to be conducted by Janssen, is expected to open for patient enrollment by the end of 2015.

About Imetelstat

Imetelstat (GRN163L; JNJ-63935937) is a potent and specific inhibitor of telomerase that is administered by intravenous infusion. This first-in-class compound, discovered by Geron, is a specially designed and modified short oligonucleotide, which targets and binds directly with high affinity to the active site of telomerase. Preliminary data suggest that imetelstat shows disease-modifying activity by affecting the malignant clones associated with hematologic myeloid malignancies. Most commonly reported adverse events in imetelstat clinical studies conducted to date include fatigue, gastrointestinal symptoms and cytopenias. Patients in these studies also experienced elevated liver enzymes, which resolved to normal or baseline in the majority of patients followed after imetelstat treatment was withdrawn. Imetelstat has not been approved for marketing by any regulatory authority.

TESARO Announces U.S. FDA Approval of VARUBI(TM) (rolapitant) for Nausea and Vomiting Associated With Cancer Chemotherapy

On September 2, 2015 TESARO, Inc. (NASDAQ:TSRO), an oncology-focused biopharmaceutical company, reported that the U.S. Food and Drug Administration (FDA) has approved VARUBI (rolapitant) in combination with other antiemetic agents in adults for the prevention of delayed nausea and vomiting associated with initial and repeat courses of emetogenic cancer chemotherapy, including, but not limited to, highly emetogenic chemotherapy (Press release, TESARO, SEP 2, 2015, View Source [SID:1234507385]).

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VARUBI is a selective and competitive antagonist of human substance P/neurokinin 1 (NK-1) receptors, with a plasma half-life of approximately seven days. Results from three Phase 3 trials of VARUBI demonstrated a significant reduction in episodes of vomiting or use of rescue medication during the 25 to 120 hour period following administration of highly emetogenic and moderately emetogenic chemotherapy regimens. In addition, patients who received VARUBI reported experiencing less nausea that interfered with normal daily life and fewer episodes of vomiting or retching over multiple cycles of chemotherapy. A 180 milligram dose of VARUBI is to be administered approximately one to two hours prior to chemotherapy administration in combination with a 5-HT3 receptor antagonist and dexamethasone. No dosage adjustment is required for dexamethasone, a CYP3A4 substrate, when administering VARUBI.

"The approval of VARUBI, our first product, represents a significant milestone in TESARO’s evolution into an integrated biopharmaceutical company with strong development and commercialization capabilities," said Lonnie Moulder, CEO of TESARO. "Results from the Phase 3 trials of VARUBI demonstrated that patients receiving emetogenic chemotherapy agents, including platinum and cyclophosphamide-containing regimens, benefitted from the addition of VARUBI to their antiemetic regimen. Data from multiple well-controlled trials demonstrate that patients who receive only a 5-HT3 receptor antagonist and dexamethasone often continue to suffer from nausea and vomiting for several days following chemotherapy administration. Patient surveys and our primary market research also point to the high rate of CINV and its potentially debilitating effects. We look forward to expanding the awareness of CINV and working with healthcare providers to make this important medicine available to patients during the fourth quarter."

"While important strides in preventing nausea and vomiting associated with chemotherapy have been made, still up to half of patients receiving emetogenic cancer chemotherapy can experience delayed CINV," said Richard J. Gralla, M.D., Professor of Medicine at Albert Einstein College of Medicine in New York. "Because NK-1 receptors are key drivers of CINV, especially in the delayed Phase, NK-1 receptor antagonists such as VARUBI, when combined with a 5-HT3 receptor antagonist and a corticosteroid, provide enhanced protection from CINV, and do so in the delayed timeframe where the most help is needed."

The full prescribing information for VARUBI will be available at www.VarubiRx.com.

About Chemotherapy-Induced Nausea and Vomiting (CINV)

Chemotherapy-induced nausea and vomiting is a debilitating, yet often preventable, side effect of chemotherapy.

Up to 50% of patients undergoing highly or moderately emetogenic chemotherapy experience delayed CINV (25 to 120 hours post chemotherapy)—even when prescribed a 5-HT3 receptor antagonist and corticosteroid.

Blocking both 5-HT3 and NK-1 receptors has been shown to offer better control of nausea and vomiting than inhibiting 5-HT3 receptors alone. Adding a single dose of VARUBI to an antiemetic regimen, including a 5-HT3 receptor antagonist and corticosteroid, further improves prevention of CINV in the delayed Phase following chemotherapy.

About the VARUBI (Rolapitant) Clinical Program

The superior efficacy of VARUBI was established in multiple randomized, well-controlled, blinded clinical trials that enrolled more than 2,500 patients. VARUBI, when administered in combination with a 5-HT3 receptor antagonist and dexamethasone, was superior to a 5-HT3 receptor antagonist and dexamethasone in preventing CINV in patients receiving either moderately or highly emetogenic chemotherapy.

The clinical profile of VARUBI in cisplatin-based highly emetogenic chemotherapy (HEC) was confirmed in two identical Phase 3 studies: HEC1 and HEC2. Both trials met their primary endpoint of complete response (CR), and demonstrated statistical superiority of rolapitant 180 mg compared to active control (5-HT3 receptor antagonist + dexamethasone) in the delayed Phase (25-120 hours) of CINV. In HEC1, 264 patients received rolapitant 180 mg and 262 received control. The proportion of patients achieving a CR was 72.7% vs. 58.4% (p= < 0.001). In HEC2, 271 patients received rolapitant and 273 received control. The proportion of patients achieving a CR was 70.1% vs. 61.9% (p=0.043). The most common adverse reactions (≥3%) among patients receiving cisplatin-based chemotherapy were neutropenia (9% VARUBI vs. 8% control), hiccups (5% vs. 4%), and abdominal pain (3% vs. 2%).

A Phase 3 trial was also conducted to evaluate rolapitant 180 mg compared to active control in 1,332 patients receiving moderately emetogenic chemotherapy regimens, including anthracycline/cyclophosphamide combinations, carboplatin, irinotecan, pemetrexed, oxaliplatin, and doxorubicin. This trial met its primary endpoint of CR, and demonstrated statistical superiority of rolapitant 180 mg compared to active control (5-HT3 receptor antagonist + dexamethasone) in the delayed Phase of CINV. The proportion of patients achieving a CR was 71.3% vs 61.6% (p= < 0.001). The most common adverse reactions (≥3%) among patients receiving these chemotherapies were decreased appetite (9% VARUBI vs. 7% control), neutropenia (7% vs. 6%), dizziness (6% vs. 4%), dyspepsia (4% vs. 2%), urinary tract infection (4% vs. 3%), stomatitis (4% vs. 2%), and anemia (3% vs. 2%).

Primary data from the three Phase 3 studies have recently been published online ahead of print in Lancet Oncology, the analysis of the non-AC MEC population was presented at the 2015 annual meeting for the Multinational Association for Supportive Care in Cancer, and commentary has been provided in Nature Reviews Clinical Oncology.

VARUBI Additional Safety Information

VARUBI is contraindicated in patients receiving thioridazine, a CYP2D6 substrate with a narrow therapeutic index.

Use of VARUBI should be avoided in patients who are receiving pimozide, a CYP2D6 substrate with a narrow therapeutic index. Adverse reactions should be monitored if concomitant use of VARUBI and other CYP2D6 substrates with a narrow therapeutic index cannot be avoided. The inhibitory effect of VARUBI on CYP2D6 lasts for at least 7 days and may last longer after administration of a single dose of VARUBI.

VARUBI is available by prescription only.

About VARUBI

VARUBI is a substance P/neurokinin-1 (NK-1) receptor antagonist indicated in combination with other antiemetic agents in adults for the prevention of delayed nausea and vomiting associated with initial and repeat courses of emetogenic cancer chemotherapy, including, but not limited to, highly emetogenic chemotherapy. NK-1 receptors are highly concentrated in the brain and bind neurokinin substance P. Activation of NK-1 receptors plays a central role in nausea and vomiting induced by emetogenic stimuli, including certain cancer chemotherapies. A Positron Emission Tomography (PET) study with rolapitant in normal, healthy volunteers demonstrated that rolapitant crosses the blood brain barrier and occupies brain NK-1 receptors at high levels for up to 120 hours. VARUBI has a half-life of approximately 7 days, which may contribute to the ability of a single dose of VARUBI to cover the entire delayed CINV Phase (25-120 hours).

An intravenous formulation of rolapitant is also being developed. TESARO licensed exclusive rights for the development, manufacture, commercialization and distribution of VARUBI (rolapitant) from OPKO Health, Inc.

Loxo Oncology Announces FDA Orphan Drug Designation Granted to LOXO-101 for Treatment of Soft Tissue Sarcoma

On September 2, 2015 Loxo Oncology, Inc. (Nasdaq:LOXO), a biopharmaceutical company focused on the discovery, development, and commercialization of targeted cancer therapies, reported that the United States Food and Drug Administration (FDA) has granted the company orphan drug designation for LOXO-101 for treatment of patients with soft tissue sarcoma (Press release, Loxo Oncology, SEP 2, 2015, View Source [SID:1234507381]).

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Soft tissue sarcomas are cancers of the body’s connective or supportive tissues, such as cartilage, fat, muscle, fibrous tissue, and blood vessels. The FDA’s Office of Orphan Drug Products grants orphan drug designation to support the development of medicines for underserved patient populations, or rare disorders, that affect fewer than 200,000 people in the United States. Orphan drug designation provides to Loxo certain benefits, including market exclusivity upon regulatory approval if received, exemption of FDA application fees and tax credits for qualified clinical trials.

About LOXO-101

LOXO-101 is a potent, oral, selective inhibitor of tropomyosin receptor kinase (TRK) signaling molecules. The TRK family (TRKA, TRKB, and TRKC) has been implicated in diverse tumor types such as lung cancer, head and neck cancer, melanoma, colorectal cancer, sarcoma, and breast cancer. LOXO-101 was built specifically to inhibit TRK and is currently the only selective TRK inhibitor in clinical development. LOXO-101 is currently being evaluated in a Phase 1 dose escalation trial for patients with advanced solid tumors.