On May 8, 2017 Rasna Therapeutics, Inc. (OTCQX: RASP), a clinical stage biotechnology company focused on the development of disease-modifying drugs for hematological malignancies, reported an update from further studies towards the development of formulated RASP-101, a novel modality for treatment of NPM1-mutated acute myeloid leukemia (AML) (Press release, Rasna Therapeutics, MAY 8, 2017, View Source [SID1234518920]). Schedule your 30 min Free 1stOncology Demo! NPM1-mutated AML is a specific genetic variation of leukemia that accounts for approximately one third cases of AML in adults. A phase II clinical trial which was initiated in 2014 and completed in two and a half years, involved patients with refractory or relapsed NPM1-mutated AML treated with cycles of dactinomycin at a dose of 15µg per kilogram per day for 5 consecutive days (EudraCT number 2014-000693-18). As earlier published, intravenous treatment of refractory or relapsed AML patients with dactinomycin (12.5µg or 15µg per day) for 5 consecutive days had produced hematological complete response in specific patients (Falini et al., N Eng J Med. 373: 12, 2015).
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"We are pleased to report an update to these initial promising findings, which are now corroborated by the follow up of a phase II clinical study which achieved complete response in 40% of patients. Although further studies are warranted to understand the mechanism of action of dactinomycin in NPM1-mutated AML, we are delighted to move forward with development of a formulated RASP-101 for treatment of AML patients," said Dr. Brunangelo Falini, a member of the scientific advisory board of Rasna Therapeutics, Inc.
"We believe that our approach is a breakthrough and a first-in-class treatment for AML patients. We are determined to rapidly develop formulation of RASP-101 to enable multi-center clinical studies in NPM1-mutated AML. Patient stratification, based on specific AML gene mutations, is part of Rasna’s strategy to improve clinical outcome for this unmet clinical need," commented Alessandro Padova, Chairman of Rasna.
Year: 2017
Kite Reports First Quarter Financial Results
On May 8, 2017 Kite Pharma, Inc. (NASDAQ:KITE), a cell therapy company, reported first quarter 2017 financial results and provided a corporate update for the period ended March 31, 2017 (Press release, Kite Pharma, MAY 8, 2017, View Source [SID1234518912]). Schedule your 30 min Free 1stOncology Demo! "Kite is intensely focused on bringing axicabtagene ciloleucel to market in 2017. Our preparation for the potential commercialization of the first CAR-T therapy in aggressive non-Hodgkin lymphoma began two years ago. With the team and infrastructure we now have in place, we are confident in our readiness to deliver upon potential approval in the U.S. and expect to file for approval in Europe in the third quarter of this year," said Arie Belldegrun, M.D., FACS, Chairman, President, and Chief Executive Officer of Kite. "We are also keeping an eye toward future growth with additional indications across the KTE-C19 program and development of earlier stage product candidates, including KITE-585, which we believe has the potential to become the next significant opportunity for Kite."
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First Quarter 2017 Financial Results
Revenues were $9.8 million for the first quarter of 2017.
Research and development expenses were $65.9 million for the first quarter of 2017, which includes $12.7 million of non-cash stock-based compensation expense.
General and administrative expenses were $35.8 million for the first quarter of 2017, which includes $11.4 million of non-cash stock-based compensation expense.
Net loss was $90.4 million, or $1.74 per share, for the first quarter of 2017.
Non-GAAP net loss for the first quarter of 2017 was $66.3 million, or $1.28 per share, excluding non-cash stock-based compensation expense of $24.1 million.
As of March 31, 2017, Kite had $804.0 million in cash, cash equivalents, and marketable securities. A public offering of common stock generated approximately $409.7 million in gross proceeds to Kite. In addition, Kite received a $50 million upfront payment related to its strategic collaboration with Daiichi Sankyo.
2017 Financial Guidance
Kite continues to expect the full year 2017 net cash burn to be between $325 million and $340 million. This guidance assumes GAAP operating expenses to be between $490 million and $515 million, which includes approximately $135 million in non-cash stock based compensation expense. Kite expects 2017 total operating expenses to consist of approximately 60 percent Research and Development and approximately 40 percent General and Administrative.
First Quarter 2017 and Recent Highlights
Axicabtagene Ciloleucel/KTE-C19 Progress
Presented positive topline results from the primary analysis of the ZUMA-1 study of axicabtagene ciloleucel in patients with aggressive non-Hodgkin lymphoma (NHL) at the 2017 American Association of Cancer Research annual meeting. Data included the 99 percent success rate in the manufacturing of clinical product patient dose from a single apheresis for the multi-center ZUMA-1 clinical trial.
Completed the submission of a Biologics License Application (BLA) to the FDA for axicabtagene ciloleucel in patients with aggressive NHL.
Initiated ZUMA-5 study of axicabtagene ciloleucel in patients with follicular NHL.
Initiated ZUMA-9 study to provide patients access to axicabtagene ciloleucel during the regulatory review period.
Strategic Collaborations
Entered a strategic collaboration with Daiichi Sankyo to develop and commercialize axicabtagene ciloleucel in Japan.
Established a joint venture with Fosun Pharma to develop and commercialize T-cell therapies, including axicabtagene ciloleucel, in China.
Pipeline Expansion
Cleared an investigational new drug (IND) application for KITE-718, a T cell receptor (TCR) cell therapy candidate that targets MAGE-A3/A6 antigens expressed on solid tumors.
Commercial Preparation
Started recruitment and training of cell therapy account managers to support customer service and logistical coordination.
Additional 2017 Clinical Milestones
KTE-C19 and axicabtagene ciloleucel
Submit marketing authorization application (MAA) to the European Medicines Authority (EMA) for axicabtagene ciloleucel in aggressive NHL in the third quarter of 2017.
Availability of preliminary 12-month follow-up data from ZUMA-1 study of axicabtagene ciloleucel in patients with aggressive NHL.
Availability of preliminary follow-up Phase 1 data from ZUMA-3 and ZUMA-4 studies of pediatric and adult acute lymphoblastic leukemia, respectively.
Advance ZUMA-3 and ZUMA-4 studies into Phase 2.
Availability of preliminary data from ZUMA-6 combination study of axicabtagene ciloleucel and atezolizumab PD-L1 checkpoint inhibitor in aggressive NHL.
Cell Therapy Pipeline
Initiate Phase 1 study of KITE-718 in patients with solid tumors in the second quarter of 2017.
File IND for KITE-585, a CAR T cell therapy candidate that targets BCMA for the treatment of multiple myeloma, in the third quarter of 2017.
Inovio & Regeneron Enter Immuno-Oncology Clinical Study Agreement for Glioblastoma Combination Therapy
On May 8, 2017 Inovio Pharmaceuticals, Inc. (NASDAQ:INO) and Regeneron Pharmaceuticals, Inc. (NASDAQ:REGN) reported a clinical study agreement for a phase 1b/2a immuno-oncology trial (Press release, Inovio, MAY 8, 2017, View Source;Regeneron-Enter-Immuno-Oncology-Clinical-Study-Agreement-for-Glioblastoma-Combination-Therapy/default.aspx [SID1234518911]). The study will be conducted by Inovio in patients with newly diagnosed glioblastoma multiforme (GBM) and will evaluate Regeneron’s PD-1 inhibitor, REGN2810, in combination with Inovio’s INO-5401 T cell activating immunotherapy encoding multiple antigens and INO-9012, an immune activator encoding IL-12.
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The open-label trial, which is expected to begin later this year, is designed to evaluate the safety and efficacy of the combination therapy in approximately 50 patients. The study will be conducted at 30 U.S. sites and the primary endpoints are safety and tolerability. The study will also evaluate immunological impact, progression-free survival and overall survival.
GBM is a devastating disease for both patients and caregivers. It is the most aggressive brain cancer and its prognosis is extremely poor, despite a limited number of new therapies approved over the last ten years. The median overall survival for patients receiving standard of care therapy is approximately 15 months and the average five-year survival rate is less than three percent.
"Regeneron’s approach to oncology includes evaluating the combination of innovative therapies that act on diverse pathways and targets," said Israel Lowy, MD, PhD, Vice President of Translation Sciences and Oncology, Regeneron. "Using our PD-1 inhibitor as a therapeutic backbone alongside Inovio’s T cell-generating therapies offers a new path for exploration and heightens the potential to develop new, desperately-needed treatment options for patients."
"The unmet need for effective therapies in GBM remains extremely high. Certain immune checkpoint inhibitors have shown efficacy in certain cancers, but evidence increasingly suggests that the benefit of checkpoint inhibitors can be enhanced when used in combination with therapies that generate T cells," said David Reardon, MD, Clinical Director of the Center for Neuro-Oncology at Dana-Farber Cancer Institute and Professor of Medicine at Harvard Medical School. "Inovio has an innovative immunotherapy platform which has shown the ability to generate antigen-specific T cells in disease areas including cancer. We look forward to exploring the potential of combining a T cell generating immunotherapy encoding multiple antigens with REGN2810, a PD-1 checkpoint inhibitor."
Dr. J. Joseph Kim, Inovio’s President and Chief Executive Officer, said, "I am a strong believer in this combination regimen approach in immuno-oncology: use Inovio immunotherapies to generate killer T cells to turn ‘cold’ tumors into ‘hot’ tumors, then block T cell suppression via checkpoint inhibition. This step with INO-5401 is very important for us in 2017, as we believe INO-5401 has the potential to be a powerful cancer immunotherapeutic in combination with promising checkpoint inhibitors such as Regeneron’s REGN2810, and we look forward to investigating its potential for GBM and multiple other challenging cancers."
Under the terms of the agreement, the trial will be solely conducted and funded by Inovio, based upon a mutually agreed upon study design, and Regeneron will supply REGN2810. Inovio and Regeneron will jointly conduct immunological analyses in support of the study. Regeneron, in collaboration with Sanofi, is developing REGN2810 both alone and in combination with other therapies for the treatment of various cancers.
About Glioblastoma
Glioblastoma, also known as glioblastoma multiforme (GBM), is the most common and aggressive type of brain cancer. GBM is usually found in the area of the brain which controls some of the most advanced processes, such as speech and emotions. GBM treatment is often limited by the tumor location and ability of a patient to tolerate surgery. Consequently, it is a particularly difficult cancer to treat. Worldwide there are an estimated 240,000 cases of brain and nervous system tumors per year; GBM is the most common and most lethal of these tumors.
About INO-5401
INO-5401 includes Inovio’s SynCon antigens for WT1, hTERT and PSMA and has the potential to be a powerful cancer immunotherapy in combination with checkpoint inhibitors. The National Cancer Institute previously highlighted WT1, hTERT and PSMA among a list of attractive cancer antigens, designating them as high priorities for cancer immunotherapy development. WT1 was at the top of the list. The hTERT antigen relates to 85 percent of cancers, and WT1 and PSMA antigens are also widely prevalent in many cancers.
Bellicum Pharmaceuticals Reports First Quarter 2017 Financial Results
On May 8, 2017 Bellicum Pharmaceuticals, Inc. (Nasdaq:BLCM), a leader in developing novel, controllable cellular immunotherapies for cancers and orphan inherited blood disorders, reported financial results for the first quarter ended March 31, 2017 and provided an update on recent developments (Press release, Bellicum Pharmaceuticals, MAY 8, 2017, View Source [SID1234518908]). Schedule your 30 min Free 1stOncology Demo! "We had a productive first quarter across our pipeline," said Rick Fair, Bellicum’s President & Chief Executive Officer. "We continued to make progress on the registration trial for BPX-501, and presented updated clinical data highlighting its potential to transform patients’ lives. We are actively recruiting initial clinical trials with our controllable CAR T and TCR product candidates, and presented preclinical data on exciting new enhancements to our pioneering technology platform. This progress underscores our commitment to developing novel cell therapies in areas of dire need."
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PROGRAM HIGHLIGHTS AND CURRENT UPDATES
BPX-501
Adjunct T-cell therapy, administered after allogeneic hematopoietic stem cell transplantation, to support faster immune recovery, improved infection control, and reduced mortality and Graft versus Host Disease (GvHD)
Registration Studies Advancing in the European Union
Bellicum continues to enroll its registration trial in the E.U. with BPX-501 and rimiducid in pediatric patients with orphan inherited blood disorders or hematologic cancers receiving a haploidentical transplant, and is preparing to initiate a separate observational trial in a comparative sample of patients receiving a matched unrelated donor, or MUD, transplant to support regulatory submission.
Preparation Ongoing for U.S. Registration Trials
Bellicum continues to prepare for pivotal trials of BPX-501 in the U.S. in pediatric patients with orphan inherited blood disorders and blood cancers and in adults with high- and intermediate-risk AML receiving haploidentical transplant.
Data Update Highlights Promise of BPX-501 Clinical Program
At the Bone Marrow Transplant (BMT) Tandem Meeting in February, Bellicum reported data from the BP-004 trial which showed a low incidence of transplant-related mortality, rapid immune recovery, a low rate of GvHD that was manageable with standard treatments or rimiducid, and no serious adverse events associated with the use of BPX-501 or rimiducid.
BPX-601
Novel GoCAR-T product candidate designed with the proprietary iMC activation switch to improve efficacy
Phase 1 BPX-601 Clinical Trial Underway
Bellicum is evaluating its first GoCAR-T product candidate in patients with nonresectable pancreatic cancer who test positive for prostate stem cell antigen (PSCA).
BPX-701
High affinity T-cell receptor (TCR) product candidate designed with the CaspaCIDe safety switch
Phase 1 BPX-701 Clinical Trial Enrolling
The trial is recruiting HLA-A2 positive patients with refractory or relapsed acute myeloid lymphoma (AML) and myelodysplastic syndromes (MDS) who test positive for preferentially-expressed antigen in melanoma (PRAME).
PRECLINICAL RESEARCH
In April, Bellicum reported positive preclinical data at AACR (Free AACR Whitepaper) on the first-ever dual-switch technology incorporated into CAR T and TCR constructs, an approach offering the possibility of both activating cells to enhance efficacy and eliminating them to manage toxicity in a single product.
FIRST QUARTER 2017 FINANCIAL RESULTS
Cash Position and Guidance: During the first quarter of 2017, Bellicum completed an underwritten public offering of common stock that provided approximately $64.6 million of net proceeds. Bellicum also borrowed the final $10.0 million tranche under its loan agreement with Hercules Capital. As of March 31, 2017, the Company had cash, restricted cash and investments totaling $164.6 million, compared to $113.4 million at December 31, 2016. Based on current operating plans, Bellicum expects to end 2017 with approximately $85 to $95 million in cash and investments, and that current cash resources will be sufficient to meet operating requirements through the end of 2018.
Net Loss: Bellicum reported a net loss of $22.0 million for the first quarter of 2017, compared to a net loss of $15.1 million for the first quarter of 2016. The results included non-cash, share-based compensation charges of $3.4 million and $3.1 million for the first quarter of 2017 and 2016, respectively.
R&D Expenses: Research and development expenses were $15.3 million and $10.9 million for the three months ended March 31, 2017 and 2016, respectively. The higher 2017 costs were due primarily to an additional $2.9 million of clinical development expenses for BPX-501 reflecting increased clinical trial activities and manufacturing costs due to increased enrollment in clinical trials, and an additional $1.5 million of expenses for increased personnel, overhead charges and manufacturing facility start-up costs.
G&A Expenses: General and administrative expenses were $5.9 million for the three months ended March 31, 2017, and $4.3 million for the three months ended March 31, 2016. The increase in G&A expenses of $1.6 million in 2017, was due primarily to higher personnel costs as a result of hiring additional employees and to severance costs.
GLYCOMIMETICS REPORTS PROGRAM UPDATES AND FIRST QUARTER 2017 RESULTS
On May 8, 2017 GlycoMimetics, Inc. (NASDAQ: GLYC) reported progress on its clinical development programs and its financial results for the first quarter ended March 31, 2017 (Filing, Q1, GlycoMimetics, 2017, MAY 8, 2017, View Source [SID1234518898]). Schedule your 30 min Free 1stOncology Demo! "In the first quarter of 2017, GlycoMimetics delivered on its plan to provide updates on both data and the status of its ongoing pipeline programs. The acceptance of our clinical and preclinical abstracts for presentation at key oncology congresses, including AACR (Free AACR Whitepaper) and more recently, ASCO (Free ASCO Whitepaper), reinforces our enthusiasm for the GMI-1271 and GMI-1359 programs, and demonstrates to the scientific and physician communities the relevance of our work to address unmet medical needs in a novel way. As we look forward to the remainder of 2017, our plan is to build on this progress with further data updates: at the Annual ASCO (Free ASCO Whitepaper) meeting in Chicago followed by the European Hematology Association (EHA) (Free EHA Whitepaper) meeting, both before the end of the second quarter in June. Additionally, we can report that the Phase 3 program to evaluate rivipansel for vaso-occlusive sickle cell crisis, according to our partner Pfizer, remains on track for completion in the second half of 2018, " said Rachel King, GlycoMimetics’ Chief Executive Officer.
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Company management will host a conference call on Thursday, May 18, 2017 at 8:30 a.m. Eastern time to provide a clinical data update from the abstracts for the upcoming ASCO (Free ASCO Whitepaper) conference. A question and answer session with the GlycoMimetics team will follow the company’s remarks. The call can be accessed by dialing (844) 413-7154 (U.S. and Canada) or (216) 562-0466 (international) and entering passcode 4110139. To access the live audio webcast, or the subsequent archived recording, visit the "Investors – Events & Presentations" section of the GlycoMimetics website at www.glycomimetics.com. The webcast will be recorded and available for replay on the GlycoMimetics website for 30 days following the call.
Key Operational Highlights for the First Quarter of 2017:
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The first of two patient cohorts in the Phase 2 portion of the AML trial of GMI-1271 has completed enrollment. This cohort is comprised of 25 patients 60 years of age or older with newly diagnosed AML. The study is designed to evaluate the potential of GMI-1271, GlycoMimetics’ E-selectin antagonist drug candidate, in combination with chemotherapy, as a treatment for patients with both newly diagnosed and relapsed/refractory AML. Enrollment in the study’s second cohort is expected to complete in the middle of this year. The two arms combined will enroll a total of about 90 patients.
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Pre-clinical research supporting the potential of two of its drug candidates, GMI-1271 and GMI-1359, against multiple myeloma was shared via an oral presentation at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2017. Combination therapy of carfilzomib with GMI-1271 or GMI-1359 prolonged survival of mice with multiple myeloma over treatment with carfilzomib alone.
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The company announced that it will provide an update on clinical data from its Phase 1/2 study of GMI-1271 in AML at the 2017 American Society for Clinical Oncology in Chicago. GMI-1271 is an antagonist of E-selectin, for which prior clinical data has shown an emerging and differentiated potential efficacy and safety profile.
First Quarter 2017 Financial Results:
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Cash position: As of March 31, 2017, GlycoMimetics had cash and cash equivalents of $34.6 million as compared to $40.0 million as of December 31, 2016. Subsequent to March 31, 2017, the Company has raised an additional $3.8 million in net proceeds under the at-the-market facility.
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R&D Expenses: The company’s research and development expenses increased to $5.9 million for the quarter ended March 31, 2017 as compared to $5.5 million for the first quarter of 2016. The increase was due to on-going costs associated with the clinical trials for GMI-1271 in AML and MM, partially offset by a decrease in expenses related to manufacturing and process development for GMI-1271.
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G&A Expenses: The company’s general and administrative expenses remained at $2.1 million for both the quarters ended March 31, 2017 and 2016.
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Shares Outstanding: Shares outstanding as of March 31, 2017 were 23,855,934.
About GMI-1271
GMI-1271 is designed to block E-selectin (an adhesion molecule on cells in the bone marrow) from binding with blood cancer cells as a targeted approach to disrupting well-established mechanisms of leukemic cell resistance within the bone marrow microenvironment. Preclinical research points to the drug’s potential role in moving cancerous cells out of the protective environment of the bone marrow where they hide and escape the effects of chemotherapy. In preclinical studies using animal models of AML, the results of which were presented at ASH (Free ASH Whitepaper) meetings, GMI-1271 was also associated with a reduction of chemotherapy-induced neutropenia and chemotherapy-induced mucositis.
About GMI-1359
GMI-1359 is designed to simultaneously inhibit both E-selectin and CXCR4. E-selectin and CXCR4 are both adhesion molecules that keep cancer cells in the bone marrow. Preclinical studies indicate that targeting both E-selectin and CXCR4 with a single compound could improve efficacy in the treatment of cancers that involve the bone marrow such as AML and MM.