On November 23, 2015 Hanmi Pharmaceutical Co., Ltd. and ZAI Lab Limited reported that they have executed a collaboration and license agreement under which ZAI Lab will acquire exclusive rights in China (including Hong Kong and Macau) to develop, manufacture and commercialize HM61713, a novel, third-generation EGFR targeted therapy for the treatment of EGFR mutation positive lung cancer.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

HM61713 is a novel, third-generation, irreversible EGFR mutant-selective tyrosine kinase inhibitor (TKI) developed to specifically target tumors with T790M mutations. At the ASCO (Free ASCO Whitepaper) Annual Meeting 2015, interim results of the Phase I/II clinical trial were presented and showed strong efficacy signals, combined with a favorable safety profile. In July this year, Hanmi and Boehringer Ingelheim entered into an exclusive license and collaboration agreement for the development and global commercialization rights, except South Korea, China and Hong Kong, of HM61713 (BI1482694).

"We are delighted to partner with Hanmi, a leader in novel drug R&D in Asia and globally," said Samantha Du, Chairman and CEO of ZAI Lab, "NSCLC is a huge unmet need in China with the world’s largest patient population. Our collaboration with Hanmi is aimed at bringing this late stage and potentially revolutionary drug quickly to the Chinese patients. We also look forward to building and continuing our strategic collaboration with Hanmi."

The agreement with ZAI Lab reflects the fact that the population of NSCLC patient in China which HM61713 targets, takes more than a half of total NSCLC patient in the world. According to the Global Data, 46% of the total NSCLC patient in the world this year is in China and by 2020, the Chinese patient population would rapidly increase up to 62% of the total.

Dr. Gwan Sun Lee, CEO of Hanmi said, "Through our collaboration with ZAI Lab with well-built, experienced R&D capability, the possibility to develop HM61713 as a first-in-class drug in China is now open. We look forward to providing innovative treatment options to Chinese patients suffering from lung cancer."

On November 23, 2015 Genmab A/S (OMX: GEN) reported that the Phase III study of single agent ofatumumab compared to single agent rituximab in patients with follicular non-Hodgkin’s lymphoma (NHL) that has relapsed at least 6 months after completion of treatment with a rituximab-containing regimen will be stopped early (Press release, Genmab, NOV 23, 2015, View Source [SID:1234508333]). The decision to stop the study was made after a planned interim analysis performed by an Independent Data Monitoring Committee (IDMC) showed that it was unlikely that ofatumumab would show superiority if the trial was to be completed as planned.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"The outcome of the interim analysis in this study is disappointing as we had hoped to see superiority of ofatumumab. The data from the study will now be prepared so that it can be presented at a future scientific conference," said Jan van de Winkel, Ph.D., Chief Executive Officer of Genmab.

Today’s news does not impact any other ongoing studies with ofatumumab.

About the study
This Phase III study aimed to randomize up to 516 patients to receive ofatumumab (1000 mg) or rituximab (375 mg/m2) by intravenous infusion for four weekly doses. Patients who had stable or responsive disease then received single infusions of ofatumumab or rituximab every two months for four additional doses for a total of eight doses over nine months. The primary endpoint of the study was progression free survival.

About Ofatumumab (Arzerra)
Ofatumumab is a human monoclonal antibody that is designed to target the CD20 molecule found on the surface of chronic lymphocytic leukemia (CLL) cells and normal B lymphocytes.

In the United States, Arzerra is approved for use in combination with chlorambucil for the treatment of previously untreated patients with CLL for whom fludarabine-based therapy is considered inappropriate. In the European Union, Arzerra is approved for use in combination with chlorambucil or bendamustine for the treatment of patients with CLL who have not received prior therapy and who are not eligible for fludarabine-based therapy. In more than 50 countries worldwide, Arzerra is also indicated as monotherapy for the treatment of patients with CLL who are refractory after prior treatment with fludarabine and alemtuzumab.
Arzerra is not approved anywhere in the world as treatment for relapsed follicular NHL.

Please see full Prescribing Information, including Boxed WARNING for Arzerra (ofatumumab).

Arzerra is marketed under a collaboration agreement between Genmab and Novartis.

On November 20, 2015 GenSpera Inc. (OTCQB: GNSZ), a biotech company developing a novel prodrug therapeutic for the treatment of cancer, reported results from the Phase II study investigating the use of mipsagargin (G-202) for the treatment of glioblastoma multiforme (Filing, 8-K, GenSpera, NOV 23, 2015, View Source [SID:1234508334]). The results will be presented today in a poster entitled, "Phase II study of mipsagargin (G-202), a PSMA-activated prodrug targeting the tumor endothelial cells, in adult patients with recurrent or progressive glioblastoma," at the Society for Neuro-Oncology (SNO) Annual Scientific Meeting in San Antonio, Texas. The poster is available on the company website.

"We are seeing disease stabilization in a subset of patients and, importantly, clinical benefit appears to be correlated with PSMA expression, which offers enrichment strategies to enroll only potential responders in future clinical trials," said David Piccioni, M.D, Ph.D., the study’s Principal Investigator. "These results demonstrate mipsagargin’s potential to treat a patient population with few therapy options."

In addition to Dr. Piccioni, the two-stage, single-arm, open-label study (NCT02067156) is led by neuro-oncologist Santosh Kesari, M.D., Ph.D., and is being conducted at the UC San Diego Moores Cancer Center in La Jolla, Calif. The Phase II results indicate that mipsagargin appears to confer clinical benefit in a subset of patients and is well tolerated by glioblastoma patients. The results are as follows:

• Three of 11 efficacy evaluable patients demonstrated at least stable disease at the first disease assessment (2 stable disease, 1 partial response), one of which has met the primary endpoint of six-month progression-free survival.
• No dose-limiting toxicities have occurred. Preliminary evidence suggests that mipsagargin is well tolerated and may induce disease stabilization or treatment response.
• PSMA (Prostate-Specific Membrane Antigen) staining of tumor tissues shows variability of expression but all three responders have >2+ staining. Biomarker evaluation is ongoing.

"We are very encouraged with the positive results from this interim Phase II trial that demonstrate the tolerability and indications of effectiveness of mipsagargin in advanced brain cancer patients," said Craig Dionne, Ph.D., chief executive officer at GenSpera. "Mipsagargin is a first-in-class agent with a novel mechanism of action that is unlike any other drug being tested in patients with advanced brain cancer."

Potential Advantages of Mipsagargin in Glioblastoma Patients

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Glioblastoma is the most common and most aggressive malignant primary brain tumor in humans. There are approximately 10,000 new cases of malignant glioblastoma diagnosed each year in the United States and, despite optimal treatment, the median survival for these patients is only 12 to 15 months. Treatment commonly consists of surgery followed by radiation and the drug temozolomide. A few drugs have been approved in patients that have recurrent tumors, but none have been shown to promote long-term tumor stabilization or survival. Glioblastomas are particularly resistant to conventional chemotherapy drugs as most cannot cross the blood-brain barrier. This disadvantage of conventional chemotherapy does not apply to mipsagargin because mipsagargin directly attacks the PSMA-expressing cells of the tumor-associated blood vessels that comprise the blood-brain barrier.

On November 23, 2015 CTI BioPharma Corp. (CTI BioPharma) (NASDAQ and MTA: CTIC) reported findings from an investigator-sponsored preclinical study showing the potential synergistic effect of combining pacritinib, an investigational oral kinase inhibitor with specificity for JAK2, FLT3, IRAK1 and CSF1R, with an epidermal growth factor receptor (EGFR) inhibitor in decreasing brain tumor initiating cells (BTIC) viability (Press release, CTI BioPharma, NOV 22, 2015, View Source;p=RssLanding&cat=news&id=2114521 [SID:1234508311]). BTICs have cancer stem cell characteristics and are believed to be responsible for disease initiation and recurrence. Additionally, high rates of mutations in BTICs may lead to the rapid emergence of resistance in GBM, the most common and aggressive primary brain cancer. Given that upregulation of the JAK/STAT pathway may be a common mechanism of resistance to EGFR antagonists, the current study demonstrates the potential for combination therapies with pacritinib to be a promising therapeutic avenue in GBM. The early phase findings also showed prolonged survival in an intracerebral BTIC xenograft preclinical model of human GBM when delivered in combination with temozolomide.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

These findings were presented by Ms Katharine Jensen from the laboratory of Drs Artee Luchman and Samuel Weiss, at the Hotchkiss Brain Institute, University of Calgary in an oral presentation (abstract #ATPS-52) during the 20th Annual Scientific Meeting and Education Day of the Society for Neuro-Oncology (SNO) held November 19-22, 2015 in San Antonio, Texas.

"Although activating mutations in EGFR are common in GBM, physiological challenges with drug penetration, drug pharmacokinetics and rapid emergence of resistance have resulted in EGFR inhibitors showing limited effectiveness as monotherapies," said Dr. Weiss, Professor at the University of Calgary’s Cumming School of Medicine and Director of the Hotchkiss Brain Institute. "Our findings support the hypothesis that combination therapy, for example targeting both the JAK/STAT pathways inhibited by pacritinib and EGFR, may improve clinical outcomes for patients with GBM."

"The tolerability profile of pacritinib observed in the clinic to date, along with the ability to achieve meaningful intracerebral levels in preclinical models, makes it a candidate to be explored for use in combination therapy for GBM," said Jack W. Singer, M.D., Chief Scientific Officer and Global Head of Translational Medicine at CTI BioPharma. "We believe these findings further support the potential for clinical trials of pacritinib in difficult to treat, non-hematological cancers."

The combination of pacritinib with one of three different EGFR inhibitors (afatinib, lapatinib, erlotinib) each resulted in decreased BTIC viability. Additionally, the effect of combining pacritinib with afatinib was shown to be synergistic in BTIC growth inhibition. Ongoing investigations are focusing on whether combinatorial therapy can improve survival in a preclinical model.

About Pacritinib

Pacritinib is an investigational oral kinase inhibitor with specificity for JAK2, FLT3, IRAK1 and CSF1R. In August 2014, pacritinib was granted Fast Track designation by the FDA for the treatment of intermediate and high risk myelofibrosis, including but not limited to patients with disease-related thrombocytopenia, patients experiencing treatment-emergent thrombocytopenia on other JAK2 inhibitor therapy, or patients who are intolerant of, or whose symptoms are sub-optimally managed on other JAK2 inhibitor therapy. The FDA’s Fast Track process is designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need. Pacritinib does not have regulatory approval and is not commercially available.

CTI BioPharma and Baxalta Incorporated (NYSE:BXLT) are parties to a worldwide license agreement to develop and commercialize pacritinib. CTI BioPharma and Baxalta will jointly commercialize pacritinib in the U.S. while Baxalta has exclusive commercialization rights for all indications outside the U.S.

About Glioblastoma Multiforme

According to the National Cancer Institute, GBM is the most common and deadliest type of primary brain tumor in adults. GBM has an incidence of two to three per 100,000 adults per year, and accounts for 52 percent of all primary brain tumors.1 The current standard of care for patients with GBM is a surgical resection, if possible, followed by radiation given with concurrent temozolomide. The prognosis for the majority of patients with GBM is poor with median survival of approximately 14.6 months with less than 30 percent of patients surviving two years using current therapies.2

On November 23, 2015 CTI BioPharma Corp. (CTI BioPharma) (NASDAQ and MTA: CTIC) reported the initiation of its rolling new drug application (NDA) to the U.S. Food and Drug Administration (FDA) for pacritinib, an investigational oral kinase inhibitor with specificity for JAK2, FLT3, IRAK1 and CSF1R (Press release, CTI BioPharma, NOV 22, 2015, View Source;p=RssLanding&cat=news&id=2114525 [SID:1234508312]). As part of the application, CTI BioPharma and its partner, Baxalta Incorporated (Baxalta), are seeking accelerated approval and priority review for pacritinib for the treatment of patients with intermediate and high-risk myelofibrosis with low platelet counts of less than 50,000 per microliter (<50,000/uL). If approved for the requested indication, pacritinib would be the first JAK2 inhibitor approved for the treatment of patients with myelofibrosis with platelet counts of less than 50,000/uL – a specific patient population for which there are currently no approved drugs. The rolling NDA allows completed portions of an NDA to be submitted and reviewed by the FDA on an ongoing basis. CTI BioPharma and Baxalta plan to complete the submission before the end of 2015.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Myelofibrosis (a type of myeloproliferative neoplasm) is a rare, but serious and life-threatening chronic bone marrow disorder caused by the accumulation of malignant bone marrow cells that triggers an inflammatory response and scars the bone marrow. Myelofibrosis is associated with significantly reduced quality of life and shortened survival, can affect patients of all ages (with a median affected age being 65 years) and an estimated prevalence in the United States of approximately 18,000 patients.

"We believe the initiation of the rolling NDA submission represents a major step forward toward potentially offering pacritinib as a next generation JAK2/FLT3 inhibitor to patients with this rare and chronic type of blood cancer," said James A. Bianco, M.D., President and CEO of CTI BioPharma. "We are excited to have achieved this milestone and look forward to working with the FDA during the review process, with the goal of bringing this important treatment to market – which we hope will fill an unmet need for many patients whose lives are profoundly impacted by myelofibrosis."

The submission includes data from the PERSIST-1 Phase 3 trial – as well as data from Phase 1 and 2 studies of pacritinib. Submission of an NDA after a single Phase 3 trial under accelerated approval, instead of waiting to complete two Phase 3 trials, could potentially reduce time to market by up to 14 months. In August 2014, pacritinib was granted Fast Track designation by the FDA for the treatment of intermediate and high-risk myelofibrosis including, but not limited to, patients with disease-related thrombocytopenia (low platelet counts); patients experiencing treatment emergent thrombocytopenia on another JAK2 therapy; or patients who are intolerant to or whose symptoms are not well controlled (or sub-optimally managed) on another JAK2 therapy.

About Myelofibrosis and Myeloproliferative Neoplasms
Myelofibrosis is a one of three main types of myeloproliferative neoplasms (MPN), which are a closely related group of hematological blood cancers. The three main types of MPNs are myelofibrosis, polycethemia vera, and essential thrombocythemia.1 Myelofibrosis is a serious and life-threatening chronic bone marrow disorder caused by the accumulation of malignant bone marrow cells that triggers an inflammatory response and scars the bone marrow. The replacement of bone marrow with scar tissue limits its ability to produce red blood cells, prompting the spleen and liver to take over this function. Symptoms that arise from this disease include enlargement of the spleen, anemia, extreme fatigue, and pain.

The estimated prevalence of MPNs suggest there are approximately 300,000 people living with the disease in the U.S. of which myelofibrosis accounts for approximately 18,000 patients.2 In Europe, there is a wide variation of prevalence observed across data sources. Myelofibrosis has a median age of 64 at the time of diagnosis2 and is a progressive disease with approximately 20 percent of patients eventually developing acute myeloid leukemia.3 The median survival for high-risk patients is less than one and a half years; median survival for myelofibrosis patients overall is approximately six years.4

About Pacritinib
Pacritinib is an investigational oral kinase inhibitor with specificity for JAK2, FLT3, IRAK1 and CSF1R. In August 2014, pacritinib was granted Fast Track designation by the FDA for the treatment of intermediate and high risk myelofibrosis, including but not limited to patients with disease-related thrombocytopenia, patients experiencing treatment-emergent thrombocytopenia on other JAK2 inhibitor therapy, or patients who are intolerant of, or whose symptoms are sub-optimally managed on other JAK2 inhibitor therapy. The FDA’s Fast Track process is designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need. Pacritinib does not have regulatory approval and is not commercially available.

CTI BioPharma and Baxalta (NYSE:BXLT) are parties to a worldwide license agreement to develop and commercialize pacritinib. CTI BioPharma and Baxalta will jointly commercialize pacritinib in the U.S. while Baxalta has exclusive commercialization rights for all indications outside the U.S.