FDA Accepts New Drug Application and Grants Priority Review for Ivosidenib in Relapsed or Refractory AML with an IDH1 Mutation

On February 15, 2018 Agios Pharmaceuticals, Inc. (NASDAQ:AGIO), a leader in the field of cellular metabolism to treat cancer and rare genetic diseases, reported that the U.S. Food and Drug Administration (FDA) has accepted the company’s New Drug Application (NDA) for ivosidenib (AG-120) for the treatment of patients with relapsed or refractory (R/R) acute myeloid leukemia (AML) with an isocitrate dehydrogenase 1 (IDH1) mutation (Press release, Agios Pharmaceuticals, FEB 15, 2018, View Source [SID1234523997]). The NDA was granted Priority Review and has been given a Prescription Drug User Fee Act (PDUFA) action date of August 21, 2018. The FDA’s Priority Review status accelerates the review time from 10 months to a goal of six months from the day of filing acceptance and is given to drugs that may offer major advances in treatment or may provide a treatment where no adequate therapy exists. Agios completed the NDA submission in late December 2017.

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!

"After decades of little change, treatment of AML has begun to shift dramatically as result of new therapies, and IDHm inhibitors will play an important role in how we treat this terrible disease," said David Schenkein, M.D., chief executive officer of Agios. "Today marks an important milestone in our efforts to rapidly advance what could be the first targeted treatment for R/R AML patients with an IDH1 mutation. We appreciate the FDA’s collaboration during the application process, and we look forward to continuing our productive dialogue."

Ivosidenib is a first-in-class, oral, targeted inhibitor of mutant IDH1. The NDA submission is based on results from AG120-C-001, a Phase 1 dose-escalation and expansion study of ivosidenib in patients with advanced hematologic malignancies and an IDH1 mutation. Data from the R/R AML patients in this study were presented at the 2017 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting.

Additionally, Abbott has submitted a Premarket Approval (PMA) application for the FDA review of an IDH1 assay on the Abbott m2000 RealTime System, an automated sample preparation and batch analyzer system for nucleic acid amplification and detection. In 2014, Abbott and Agios entered into an exclusive agreement under which Abbott is responsible for development and commercialization of a RealTime PCR assay for detection of the IDH1 mutation in bone marrow and blood. The Abbott assay will serve as a companion diagnostic for ivosidenib.

IDH1 mutations occur in about 6 to 10 percent of AML patients. Recent publications have highlighted the advances in the understanding of the genetics underlying AML and the need for routine mutational analysis at diagnosis and relapse.

Ivosidenib is an investigational drug that has not been approved for any use in any country.

About Acute Myelogenous Leukemia (AML)
AML, a cancer of blood and bone marrow characterized by rapid disease progression, is the most common acute leukemia affecting adults. Undifferentiated blast cells proliferate in the bone marrow rather than mature into normal blood cells. AML incidence significantly increases with age, and the median age at diagnosis is 68. The vast majority of patients do not respond to chemotherapy and progress to relapsed/refractory AML. The five-year survival rate for AML is approximately 20 to 25 percent. IDH1 mutations are present in about 6 to 10 percent of AML cases.

Eagle Pharmaceuticals, Inc. to Discuss Fourth Quarter and Year End 2017 Financial Results on February 26, 2018

On February 15, 2018 Eagle Pharmaceuticals, Inc. ("Eagle" or "the Company") (Nasdaq: EGRX) reported that the Company will release its 2017 fourth quarter and full year financial results on Monday, February 26, 2018, before the market opens (Press release, Eagle Pharmaceuticals, FEB 15, 2018, View Source [SID1234524002]).

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!

Scott Tarriff, Chief Executive Officer, and Pete Meyers, Chief Financial Officer, will host a conference call to discuss the results as follows:


Date Monday, February 26, 2018
Time 8:30 a.m. EST
Toll free (U.S.) 866-518-6930
International 203-518-9797
Webcast (live and replay)
www.eagleus.com, under the "Investor Relations" section

A replay of the conference call will be available for one week after the call’s completion by dialing 800-677-7320 (US) or 402-220-0666 (International) and entering conference call ID EGRXQ417. The webcast will be archived for 30 days at the aforementioned URL.

Athenex Meets Enrollment Target for Oraxol Phase III Clinical Trial in Metastatic Breast Cancer

On February 15, 2018 Athenex (Nasdaq:ATNX), a global biopharmaceutical company dedicated to the discovery, development and commercialization of novel therapies for the treatment of cancer and related conditions, reported that the enrollment of patients is on target for the Company to be able to conduct a second interim analysis in the Oraxol KX-ORAX-001 Phase III clinical trial in the third quarter of 2018 (Press release, Athenex, FEB 15, 2018, View Source;p=RssLanding&cat=news&id=2332730 [SID1234523998]).

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!

Oraxol, an innovative development in the treatment of cancer, is a novel oral formulation of paclitaxel, a very effective and commonly used chemotherapy treatment for many cancers, combined with HM30181A (a novel orally non-absorbable gastrointestinal tract P-glycoprotein pump inhibitor). The Oraxol KX-ORAX-001 Phase III clinical trial is an international randomized controlled clinical trial comparing Oraxol monotherapy against intravenous (IV) paclitaxel monotherapy in patients with metastatic breast cancer, with target sample size of 360 patients. The study is designed to show superiority of clinical efficacy of Oraxol over IV paclitaxel based on independently confirmed response rates.

In accordance with the protocol the Company conducted a first interim analysis when 90 patients completed 18 weeks of treatment in October 2017. Based on that analysis, the independent Drug Safety Monitoring Board (DSMB) unanimously recommended continuation of the study. The DSMB was impressed by the conduct of the study in achieving a good overall response rate. The DSMB noticed that neuropathy was rare with Oraxol treatment. Neuropathy is a dose limiting toxicity of IV paclitaxel. The DSMB encouraged the rapid patient recruitment toward the scheduled second interim analysis at 180 patients.

Athenex has already enrolled more than 180 patients and therefore expects the second interim analysis in the third quarter of 2018.

Dr. Rudolf Kwan, Chief Medical Officer, commented, "In January 2018, we had positive feedback from the FDA that if this study meets the primary endpoint with an acceptable benefit/risk profile, it could be adequate as a single comparative trial to support registration of Oraxol for a metastatic breast cancer indication in the United States. We are looking forward to seeing results from the second interim analysis of this clinical trial."

Dr. Johnson Lau, Athenex’s Chief Executive Officer and Chairman of the Board, stated, "We are very proud of the excellent planning and execution of our clinical team. To be able to deliver both the enrollment for the Oraxol Phase III study on target time and the enrollment of two Phase III studies for KX-01 Ointment for the treatment of actinic keratosis ahead of schedule reflect the hard work, quality and the commitment of our clinical operation team. We are delighted that we are going to again deliver an interim analysis of the topline Oraxol Phase III study results on schedule as planned. Athenex is also conducting clinical studies on Oraxol in other geographic areas as well as for the expansion of clinical indications. We are also proud that Oraxol has recently obtained the Promising Innovative Medicine (PIM) designation by the United Kingdom Health Authority MHRA and also the allowance of two IND (HM30181A and oral paclitaxel capsules) by the Chinese FDA."

Genocea Reports Fourth Quarter and Full-Year 2017 Financial Results

On February 15, 2018 Genocea Biosciences, Inc. (NASDAQ:GNCA), a biopharmaceutical company developing neoantigen cancer vaccines, today reported financial results for the fourth quarter and full year ended December 31, 2017 and recent corporate developments (Press release, Genocea Biosciences, FEB 15, 2018, View Source [SID1234524004]).

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!

"We’ve had a productive start to 2018 and are encouraged by both the progress with our lead neoantigen cancer vaccine, GEN-009, and the broader application of our ATLAS technology to cancer," said Chip Clark, president and chief executive officer of Genocea. "Notably, we believe that our $55 million financing suggests a strong endorsement of our technology, our corporate strategy, and our team. We continue to advance GEN-009, bringing us very close to filing our first cancer vaccine IND. We are also continuing to explore partnership opportunities for our ATLAS platform, a technology that we believe uniquely differentiates us from our peers as the only platform to identify true T cell antigens, which we anticipate will result in more effective cancer vaccines."

Recent Milestones & Events

November 2017: At the 32nd Annual Meeting of the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) (SITC 2017), Genocea presented data demonstrating the power and versatility of its ATLAS platform and its potential superiority to in silico methods of neoantigen identification, as well as data highlighting the discovery of unexpected and novel T cell antigens for vaccines against colorectal cancer and non-small cell lung cancer.
January 2018: Genocea announced completion of a $55 million financing, including significant investments by New Enterprise Associates (NEA) and Vivo Capital (Vivo).
January 2018: The U.S. Patent and Trademark Office issued an allowance on United States Patent 9,873,870, further strengthening the company’s intellectual property position on its ATLAS platform for the identification and characterization of neoantigens and tumor-associated antigens.
January 2018: Genocea and Oncovir, Inc. entered into a license and supply agreement for Oncovir’s Hiltonol (poly-ICLC) adjuvant, a key component of Genocea’s personalized cancer vaccine candidate, GEN-009.
Anticipated Upcoming Milestones & Events

Q1 2018: The company expects to file an Investigational New Drug (IND) application with the U.S. Food and Drug Administration for its GEN-009 personalized cancer vaccine.
March 2018: Genocea management is scheduled to present at the Cowen and Company Annual Health Care Conference in Boston (March 12-14), and at the Needham & Co. Annual Healthcare Conference in New York City (March 27-28).
Mid-2018: Genocea plans to initiate a Phase 1/2a clinical trial for GEN-009 in patients with a variety of tumor types. The first part of this trial is expected to enroll 6 patients with no evidence of disease but a high likelihood of relapse. Safety and immunogenicity data will be monitored, with preliminary results expected in the first half of 2019.
Corporate Update and Financial Guidance
In January 2018, Genocea completed a $55 million equity financing, including significant investments from NEA and Vivo. Net proceeds from the equity financing were approximately $51.7 million.

Genocea continues to explore strategic alternatives for GEN-003, its Phase 3-ready investigational immunotherapy to treat the large patient population infected with genital herpes, many of whom are dissatisfied with their current treatment options.

In January 2018, Genocea entered into an amendment to its loan agreement with Hercules that provides, at no incremental cost to Genocea, for a deferred principal payment period of 3 months commencing on February 1, 2018. During this time Genocea will continue to make monthly payments of interest. Genocea has initiated a process to restructure or refinance the debt facility to better align repayment of the debt with its new corporate strategy and anticipated clinical milestones. If this process is successful, the company expects to be able to defer certain debt principal payments, thereby reducing expected significant cash payments in 2018 and 2019 relating to the current debt facility.

Genocea expects that its existing cash and cash equivalents are sufficient to support its operating expenses and capital expenditure requirements into the second half of 2019 without assuming the expected benefit of the restructuring or refinancing of its debt facility.

Conference Call
Genocea will host a conference call and webcast today at 9:00 a.m. ET. The conference call may be accessed by dialing (844) 826-0619 for domestic participants and (315) 625-6883 for international callers and referencing the conference ID number 7396178. A live webcast of the conference call will be available online from the investor relations section of the Company’s website at View Source A webcast replay of the conference call will be available on the Genocea website beginning approximately two hours after the event and will be archived for 30 days.

Fourth Quarter 2017 Financial Results

Cash Position: As of December 31, 2017, cash and cash equivalents were $12.3 million compared to $22.0 million as of September 30, 2017.
Research and Development (R&D) Expenses: R&D expenses decreased approximately $3.9 million to $7.9 million for the quarter ended December 31, 2017 from $11.8 million for the same period ended December 31, 2016. The decrease was primarily driven by $6.5 million in reduced GEN-003 costs, as a result of the strategic shift and restructuring announced in September 2017. Decreases in GEN-003 costs were offset by a $3.9 million increase in costs incurred on the Company’s GEN-009 program as it continued to develop its supply chain and manufacturing capabilities to prepare for the planned IND filing and initiation of clinical trials for GEN-009.
General and Administrative (G&A) Expenses: G&A expenses decreased approximately $1.4 million to $2.5 million for the quarter ended December 31, 2017 from $3.9 million for the quarter ended December 31, 2016. The decrease was driven by reduced compensation, consulting and professional service costs, depreciation and facility related costs.
Net Loss: Net loss was $10.7 million for the quarter ended December 31, 2017, compared to a net loss of $16.0 million for the same period in 2016.

Full Year 2017 Financial Results

Cash Position: As of December 31, 2017, cash and cash equivalents were $12.3 million compared to cash, cash equivalents, and investments totaling $63.4 million as of December 31, 2016.

R&D Expenses: R&D expenses increased approximately $4.6 million to $39.2 million for the year ended December 31, 2017 from $34.6 million for the year ended December 31, 2016. On a program basis, GEN-009 and other immuno-oncology costs increased by $9.5 million, driven primarily by increased headcount, consulting and professional service costs, manufacturing and clinical and lab related costs in anticipation of the expected IND filing for GEN-009 in early 2018. GEN-003 costs increased $1.9 million, driven by increased external manufacturing related expenses, headcount and consulting and professional service costs in advance of the previously planned Phase 3 trials, offset by decreased clinical and lab related costs. Increased spending on these programs was offset by lower costs on other infectious disease programs previously deprioritized in 2016.

G&A Expenses: General and administrative expense decreased $2.0 million to $13.4 million for the year ended December 31, 2017 from $15.4 million for the year ended December 31, 2016. Decreases were driven by lower consulting and professional services costs, facility-related costs and depreciation.

Restructuring: As a result of the Company’s strategic shift to immuno-oncology, which was announced in September 2017, restructuring charges of $2.6 million were incurred for employee severance, employee benefits, contract terminations and asset impairment. Of these charges, $1.1 million were paid through December 31, 2017, $0.5 million were recorded as accrued expenses at December 31, 2017 and $1.0 million were non-cash charges recorded during the year ended December 31, 2017.

Net Loss: Net loss was $56.7 million for the year ended December 31, 2017, compared to a net loss of $49.6 million for the year ended December 31, 2016.

Asterias Expands Global IP Portfolio with New Patents, Including Key Additional Patent Protection for its Cancer Immunotherapy Program

On February 15, 2018 Asterias Biotherapeutics, Inc. (NYSE American:AST), a biotechnology company dedicated to developing regenerative medicine therapeutics to treat neurological conditions associated with de-myelination and cellular immunotherapies to treat cancer, reported that it has strengthened its global IP portfolio over the past 12 months with the issuance of 46 new patents, including in the United States, Europe, Japan, Canada, China, Australia, Israel and South Korea (Press release, BioTime, FEB 15, 2018, View Source;p=RssLanding&cat=news&id=2332687 [SID1234523999]). Of the 46 patents, 38 are owned or jointly owned by Asterias and eight patents are exclusively licensed to the Company.

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!

Included in the issued patents are those covering differentiation of pluripotent stem cells to hematopoietic progenitors and differentiation of pluripotent stem cells to immature and mature dendritic cells, providing key patent protection to the Company’s allogeneic (non-patient specific) cancer immunotherapy AST-VAC2 program. Other issued patents cover various aspects of culture and expansion of undifferentiated stem cells, providing support for both AST-VAC2 and the Company’s AST-OPC1 product candidate which is currently being evaluated in the Company’s Phase 1/2a clinical program for severe spinal cord injury. Additional issued patents cover differentiation of pluripotent stem cells to various other lineages, including neural cells such as dopaminergic neurons, cardiomyocytes, and hepatocytes.

"We are excited about these new patents as they provide additional protection for certain aspects of the AST-VAC2 manufacturing process and support scalable processes of culturing pluripotent stem cells," said Michael Mulroy, President and Chief Executive Officer of Asterias. "In addition to being important for our own programs, these patents and our entire patent portfolio could enhance shareholder value by providing future licensing and partnering opportunities with third parties."