On March 2, 2017 BeiGene, Ltd. (NASDAQ:BGNE), a clinical-stage biopharmaceutical company developing molecularly-targeted and immuno-oncology drugs for the treatment of cancer, reported the dosing of the first patient in a pivotal clinical trial of BGB-3111, an investigational Bruton’s Tyrosine Kinase (BTK) inhibitor, in Chinese patients with relapsed or refractory mantle cell lymphoma (MCL) (Press release, BeiGene, MAR 2, 2017, View Source [SID1234517970]). BGB-3111 is also currently being evaluated in a global Phase III study in comparison with ibrutinib for the treatment of patients with Waldenström’s Macroglobulinemia. Schedule your 30 min Free 1stOncology Demo! "We are pleased to announce the beginning of the first pivotal clinical trial of BGB-3111 in China. BGB-3111 has been under clinical investigation since August 2014, and over 300 patients with various B-cell malignancies have been treated with BGB-3111 in Australia, New Zealand, the United States, Korea, and China," commented John V. Oyler, Founder, Chief Executive Officer, and Chairman.
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"The initiation of the first pivotal trial in China with one of our portfolio compounds is an important step towards our goal of advancing innovative cancer treatments for patients in China," commented Lai Wang, Ph.D., Head of China Development.
The Phase II single-arm, open-label, multi-center study is designed to investigate the efficacy and safety of BGB-3111 in patients with relapsed or refractory MCL. The study’s primary endpoint is the objective response rate, defined as achievement of either a partial response or complete response at any time on study drug. Secondary endpoints include progression free survival, duration of response, time to response, safety, and tolerability. Professor Jun Zhu of the Beijing Cancer Hospital is the lead principal investigator of the trial.
About BGB-3111
BGB-3111 is a potent and highly selective investigational small molecule inhibitor of BTK. BGB-3111 has demonstrated higher selectivity against BTK than ibrutinib (the only BTK inhibitor currently approved by the U.S. Food and Drug Administration and the European Medicines Agency) based on biochemical assays, higher exposure than ibrutinib based on their respective Phase I experience, and sustained 24-hour BTK occupancy in both the blood and the lymph node.
Karyopharm Announces Results from Interim Analysis of Phase 2 SOPRA Study Evaluating Selinexor in Relapsed/Refractory Acute Myeloid Leukemia
On March 2, 2017Karyopharm Therapeutics Inc. (Nasdaq:KPTI), a clinical-stage pharmaceutical company, reported the results of a planned interim analysis of the Phase 2 SOPRA study evaluating single agent selinexor in relapsed/refractory acute myeloid leukemia (AML) (Press release, Karyopharm, MAR 2, 2017, View Source [SID1234517968]).
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The Company determined in concert with the study’s independent Data Safety Monitoring Board (DSMB) that SOPRA will not reach statistical significance for overall survival (OS), the study’s primary endpoint. However, since selinexor-treated patients that achieved a complete response (CR) showed a substantial OS benefit as compared with the physician’s choice (PC) arm, Karyopharm and the DSMB agreed that patients would be permitted to continue on the selinexor arm or the PC arm, as applicable, following discussion between the patient and their treating physician. The Company plans to continue clinical development of selinexor in AML through investigator sponsored trials in multiple combination regimens, including with chemotherapy, given encouraging data to date across these settings.
SOPRA is a Phase 2 randomized study of patients 60 years of age or older with relapsed or refractory AML who were ineligible for intensive chemotherapy and/or transplantation. Patients were randomized to either receive single-agent oral selinexor 60mg twice weekly or PC. PC included best supportive care (BSC) alone, or BSC plus either azacitidine (Vidaza), decitabine (Dacogen), or low dose cytosine arabinoside (LD-AraC). Based on unaudited site data, SOPRA enrolled 176 patients (median of two prior regimens) in the U.S., Canada, Europe and Israel. Among patients on the selinexor arm, 13% demonstrated a CR with or without full hematologic recovery (CRi) compared to 3% of patients on the PC control arm. Some patients remained on selinexor for over one year, but this did not result in a statistically superior OS compared to the PC arm. The DSMB found no new clinically significant adverse events in the patients receiving selinexor. Importantly, rates of sepsis and febrile neutropenia (FN) were lower on the selinexor arm (sepsis 4.9%, FN 14.7%) compared to the PC arm (sepsis 6.1%, FN 36.4%). As expected, the most common selinexor-related adverse events were nausea, anorexia, fatigue, vomiting, and thrombocytopenia. Patients who have benefited from selinexor treatment on the SOPRA study have the option to continue therapy.
"SOPRA is a robust, well-conducted trial and the response rates achieved with single-agent selinexor in this heavily pretreated older population have been encouraging," said Hagop Kantarjian, MD, Chair of the Department of Leukemia, The University of Texas MD Anderson Cancer Center. "Importantly, the safety profile was as expected and the recommended Phase 2 dose was generally well-tolerated. Unfortunately, as is common in AML, the higher response rates observed with single-agent selinexor versus physician’s choice did not translate into extended survival in the overall population of these frail and heavily pretreated patients."
"After performing an in-depth analysis, we and the DSMB agree that, despite the higher complete response rates observed with selinexor, the phase 2 SOPRA study evaluating single-agent selinexor in relapsed or refractory AML has not reached statistical significance for overall survival, the primary endpoint of the study," said Michael G. Kauffman, MD, PhD, Chief Executive Officer of Karyopharm. "While we are disappointed with the overall outcome, we are pleased that 60mg of single-agent selinexor dosed twice per week was well-tolerated and carried no increased risk of sepsis or febrile neutropenia. At Karyopharm, our primary focus remains the advancement of selinexor in relapsed or refractory multiple myeloma, where we believe we have a clear path to regulatory approval."
Dr. Kauffman continued, "Beyond myeloma, we see diffuse large B-cell lymphoma (DLBCL) and liposarcoma as high unmet need indications where selinexor has a meaningful opportunity for clinical success and where we are expecting key data readouts during 2017. We look forward to reporting top-line data from our randomized Phase 2b SADAL study evaluating single-agent selinexor in patients with relapsed or refractory DLBCL in early 2017 and top-line data from the Phase 2 portion of the randomized Phase 2/3 SEAL study evaluating single-agent selinexor in patients with advanced liposarcoma in mid-2017."
Sharon Shacham, PhD, MBA, President and Chief Scientific Officer of Karyopharm, commented, "We continue to believe selinexor has potential in AML, most likely in combination with other agents in front line and later settings. We continue to explore the use of selinexor in combination with novel and standard agents through investigator-sponsored AML studies. Clinical data recently reported at the 2016 American Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting demonstrated that selinexor in combination with certain standard therapies, including intensive chemotherapy as well as hypomethylating agents, demonstrated encouraging activity in AML in adults, both as an initial therapy and in the relapsed setting. The benefit of selinexor in combination with intensive chemotherapy will be assessed in randomized investigator sponsored trials that we expect will begin in 2017. Furthermore, selinexor in combination with intensive chemotherapy has shown very promising responses in pediatric patients with heavily pretreated AML."
"We are deeply grateful for the support and commitment of the AML investigators and the patients and families who have taken part in or contributed to the SOPRA study," Dr. Shacham concluded.
More About the Phase 2 SOPRA Study
The Phase 2 SOPRA (Selinexor in Older Patients with Relapsed/Refractory AML) study is a randomized trial evaluating single-agent selinexor (KPT-330), Karyopharm’s lead, novel, oral Selective Inhibitor of Nuclear Export / SINE compound, versus physician’s choice in patients 60 years of age or older with relapsed or refractory AML who were ineligible for intensive chemotherapy and/or transplantation. In the SOPRA study, 176 patients with AML whose disease had relapsed after, or was refractory to, first line therapy were randomized 2:1 to receive either oral selinexor (60 mg twice per week) or one of four physician’s choice (PC) therapies. Physician’s choice included best supportive care (BSC) alone, or BSC plus either azacytidine (Vidaza), decitabine (Dacogen), or low dose cytosine arabinoside (LD-AraC). The primary endpoint of the SOPRA study was overall survival (OS), with a target of a 75% improvement in OS from 3.0 months in the PC arm to 5.2 months in the selinexor arm. SOPRA was conducted at approximately 94 sites worldwide, including sites in the U.S., Canada, Europe and Israel.
About Selinexor
Selinexor (KPT-330) is a first-in-class, oral Selective Inhibitor of Nuclear Export / SINE compound. Selinexor functions by binding with and inhibiting the nuclear export protein XPO1 (also called CRM1), leading to the accumulation of tumor suppressor proteins in the cell nucleus. This reinitiates and amplifies their tumor suppressor function and is believed to lead to the selective induction of apoptosis in cancer cells, while largely sparing normal cells. To date, over 1,900 patients have been treated with selinexor and it is currently being evaluated in several mid- and later-phase clinical trials across multiple cancer indications, including in multiple myeloma in combination with low-dose dexamethasone (STORM) and backbone therapies (STOMP), and in diffuse large B-cell lymphoma (SADAL), and liposarcoma (SEAL), among others. Karyopharm plans to initiate a pivotal randomized Phase 3 study of selinexor in combination with bortezomib (Velcade) and low-dose dexamethasone (BOSTON) in patients with multiple myeloma in early 2017. Additional Phase 1, Phase 2 and Phase 3 studies are ongoing or currently planned, including multiple studies in combination with one or more approved therapies in a variety of tumor types to further inform the Company’s clinical development priorities for selinexor. The latest clinical trial information for selinexor is available at www.clinicaltrials.gov.
Aduro Biotech Announces Upcoming Data Presentations at the Keystone Symposia on Cancer Immunology and Immunotherapy Conference
On March 2, 2017 Aduro Biotech, Inc. (Nasdaq:ADRO), a biopharmaceutical company with three distinct immunotherapy technologies, reported data presentations relating to its technology platforms to be given at the Keystone Symposia on Cancer Immunology and Immunotherapy Conference: Taking a Place in Mainstream taking place in Whistler, B.C., Canada, March 19 through 23, 2017 (Press release, Aduro Biotech, MAR 2, 2017, View Source [SID1234517967]). Schedule your 30 min Free 1stOncology Demo!
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Poster 1061: Administration of the synthetic STING agonist ADU-S100 leads to durable anti-tumor immunity via promoting T-cell responses
Poster Session: 1
Date/Time: Monday, March 20, 7:30 p.m. to 10:00 p.m. PT
Poster 2038: Efficacy of pLADD, a personalized immunotherapy strategy targeting tumor-specific neoantigens using live attenuated Listeria monocytogenes
Poster Session: 2
Date/Time: Tuesday, March 21, 2017, 7:30 p.m. to 10:00 p.m. PT
Foundation Medicine Receives Medicare Payment in Non-Small Cell Lung Cancer under a Local Coverage Determination for FoundationOne®, the Company’s Comprehensive Genomic Profiling Assay
On March 2, 2017 Foundation Medicine, Inc. (NASDAQ:FMI) reported that it has received payment from Palmetto GBA, the Company’s Medicare Administrative Contractor (MAC) in North Carolina, for its FoundationOne comprehensive genomic profiling assay when used in the clinical course of care for individuals in the United States with Stage IIIB/IV non-small cell lung cancer (NSCLC) who meet the eligibility requirements under Palmetto GBA’s Local Coverage Determination L36143 (LCD) (Press release, Foundation Medicine, MAR 2, 2017, View Source [SID1234517962]). The LCD was most recently updated on December 22, 2016. Foundation Medicine began submitting an initial set of claims to Palmetto GBA in January 2017 for FoundationOne, and received its first payments for claims under this LCD on March 1, 2017. Schedule your 30 min Free 1stOncology Demo! "Coverage and payment for FoundationOne under Palmetto GBA’s LCD is a positive step toward advancing access to precision medicines for individuals living with non-small cell lung cancer," said Troy Cox, chief executive officer for Foundation Medicine. "We look forward to continuing to work with Palmetto GBA as we gain additional payment experience under this LCD for non-small cell lung cancer. We will continue to work with FDA and CMS as they review our universal companion diagnostic test through the Parallel Review process with the goal of being the first pan-cancer, universal companion diagnostic test to receive FDA approval and a National Coverage Determination from CMS."
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CytomX Announces Full-Year 2016 Financial Results
On March 2, 2017 CytomX Therapeutics, Inc. (Nasdaq:CTMX), a biopharmaceutical company developing investigational Probody therapeutics for the treatment of cancer, reported full-year 2016 financial results (Press release, CytomX Therapeutics, MAR 2, 2017, View Source [SID1234517961]). Schedule your 30 min Free 1stOncology Demo! As of December 31, 2016, CytomX had cash and cash equivalents and short-term investments of $181.9 million. Based upon its current operating plan, the Company expects its existing capital resources will be sufficient to fund operations into 2019.
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"Over the past year, CytomX has transformed from a research organization to a clinical-stage company, bringing us one step closer to realizing our vision of transforming lives with safer and more effective therapies," said Sean McCarthy, D.Phil., president and chief executive officer of CytomX Therapeutics. "With our lead program, CX-072, in the clinic and CX-2009 closely behind, together with our world-class pharmaceutical partnerships, we are advancing a broad and deep pipeline of differentiated Probody therapeutics that are focused on some of the most compelling targets for the treatment of cancer."
2016 Business Highlights and Recent Developments
PROCLAIM-CX-072 (PD-L1 Probody) Program
Enrollment is underway in the PROCLAIM clinical study of CX-072, a PD-L1-targeting Probody therapeutic for the treatment of cancer patients.
Clinical data is expected to begin to emerge in late 2017, and throughout 2018.
CX-2009 (CD166 Probody Drug Conjugate) Program
Following completion of GMP manufacturing and GLP toxicity studies in 2016, the IND filing for CX-2009 remains on track for the first half of 2017.
CX-2009 is a first-in-class Probody drug conjugate targeting the highly expressed tumor antigen, CD166.
Clinical data is expected to begin to emerge in late 2017, and throughout 2018.
Partnerships
CytomX continues to forge biopharmaceutical partnerships that retain meaningful downstream rights to extend the reach of our technology and in order to fund its wholly-owned programs.
As part of our ongoing collaboration, Bristol-Myers Squibb selected the third and fourth targets and selected a CTLA-4 clinical candidate for a total of $25 million in payments to CytomX in 2016.
In April 2016, CytomX entered into a collaboration with AbbVie to co-develop and co-commercialize Probody Drug Conjugates against CD71 and up to two additional targets to be selected by AbbVie. CytomX received an upfront payment of $30 million.
Full-Year Financial Results
Cash, cash equivalents and investments totaled $181.9 million as of December 31, 2016, compared to $186.7 million as of December 31, 2015. The decrease reflects cash used in operations, partially offset by a $30 million upfront payment received from AbbVie in connection with the development and collaboration agreements entered into in April 2016, and $25 million in milestone payments received from Bristol-Myers Squibb in connection with its third target selection in January 2016, and fourth target selection in December 2016.
Research and development expenses were $54.8 million for the year ended December 31, 2016, compared to $28.4 million for the year ended December 31, 2015. The increase was primarily attributable to $9.6 million in manufacturing costs for the Company’s CX-072, CX-2009 and CX-2029 programs, $4.5 million in laboratory and professional services and supplies, $3.1 million in personnel-related expenses due to an increase in headcount, $3.1 million in non-cash stock-based compensation due to higher stock valuation, $2.4 million to advance CX-072 into Phase 1/2 clinical development, $1.7 million in royalty payments triggered by the payments from Bristol-Myers Squibb’s third and fourth target selections, clinical candidate selection, as well as upfront payments from AbbVie, and $1.6 million in facilities-related expenses due to a move to a larger facility in October 2016.
General and administrative expenses were $19.9 million for the year ended December 31, 2016, compared to $12.6 million for the year ended December 31, 2015. The increase was predominantly due to $3.2 million in non-cash stock based compensation due to higher stock valuation, $2 million in professional service and outside service expenses, $1.8 million in personnel-related expenses due to an increase in headcount and $0.4 million in facilities-related expense due to a move to a larger facility in October 2016.