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Agios Reports Fourth Quarter and Full Year 2017 Financial Results

On February 14, 2018 Agios Pharmaceuticals, Inc. (NASDAQ:AGIO), a leader in the field of cellular metabolism to treat cancer and rare genetic diseases, today reported business highlights and financial results for the fourth quarter and year ended December 31, 2017 (Press release, Agios Pharmaceuticals, FEB 14, 2018, View Source [SID1234523961]). In addition, Agios highlighted select 2018 corporate milestones and data presentations for its clinical development programs.

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"2017 was an extraordinary year for Agios with the U.S. approval of IDHIFA, our first internally discovered and developed drug, the NDA submission for our wholly owned medicine ivosidenib and our sixth IND submission since the company’s inception," said David Schenkein, M.D., chief executive officer at Agios. "It was a data-rich year where we set the stage for building long-term value across our cancer and rare disease portfolios. Execution in 2018 will be equally critical as we ready our organization for the potential approval and launch of ivosidenib, initiate our pivotal program for AG-348 in pyruvate kinase deficiency and advance our robust discovery portfolio."

KEY UPCOMING MILESTONES

The company plans to achieve the following key milestones in 2018:

Cancer:

Potential approval and commercialization of ivosidenib in the United States for relapsed/refractory (R/R) acute myeloid leukemia (AML) with an isocitrate dehydrogenase-1 (IDH1) mutation in the third quarter of 2018.
Potential submission of a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for ivosidenib for IDH1m R/R AML in the fourth quarter of 2018.
Support, in conjunction with Celgene, the initiation of an intergroup sponsored, global, registration-enabling Phase 3 trial combining ivosidenib or enasidenib with standard induction and consolidation chemotherapy in frontline AML patients with an IDH1 or IDH2 mutation in the fourth quarter of 2018.
Initiate a perioperative ‘window’ trial with ivosidenib and AG-881 in low-grade glioma in the first quarter of 2018 to further investigate their effects on brain tumor tissue.
Initiate a Phase 1 dose-escalation trial for AG-270, a first-in-class methionine adenosyltransferase 2a (MAT2A) inhibitor, in patients with methylthioadenosine phosphorylase (MTAP)-deleted tumors in the first quarter of 2018.
Rare Genetic Diseases:

Initiate two global pivotal trials for AG-348 in pyruvate kinase (PK) deficiency in the first half of 2018:
ACTIVATE-T: A single arm trial of approximately 20 regularly transfused patients is expected to initiate in the first quarter of 2018.
ACTIVATE: A placebo-controlled trial of approximately 80 patients who do not receive regular transfusions is expected to initiate in the second quarter of 2018.
Initiate a global registry, known as PEAK, for adult and pediatric patients with PK deficiency in the first quarter of 2018.
Initiate a Phase 2 proof of concept trial of AG-348 in thalassemia in the fourth quarter of 2018.
Research:

Submit an investigational new drug (IND) application for our newest development candidate, an inhibitor of the metabolic enzyme dihydroorotate dehydrogenase (DHODH) for the treatment of hematologic malignancies in the fourth quarter of 2018.
ANTICIPATED KEY 2018 DATA PRESENTATIONS

Updated data from the expansion phase of the ongoing Phase 1 study of ivosidenib in IDH1m R/R AML has been submitted to the 2018 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting.
Updated data from the ongoing Phase 1/2 combination trial of enasidenib or ivosidenib with VIDAZA in patients with newly diagnosed AML with an IDH2 or IDH1 mutation ineligible for intensive chemotherapy has been submitted to ASCO (Free ASCO Whitepaper).
First clinical data from the Phase 1 study of AG-881 in advanced IDHm positive solid tumors, including glioma, has been submitted to ASCO (Free ASCO Whitepaper).
Updated data from the ongoing Phase 1 combination trial of enasidenib or ivosidenib with standard-of-care intensive chemotherapy in patients with newly diagnosed AML with an IDH2 or IDH1 mutation to be submitted to the 2018 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition.
FOURTH QUARTER 2017 HIGHLIGHTS & RECENT PROGRESS

Completed an underwritten public offering in January of 8,152,986 shares of common stock at the offering price of $67.00 per share, resulting in proceeds, net of underwriting discounts and commissions, of approximately $516.2 million.
Submitted a new drug application (NDA) to the U.S. Food and Drug Administration (FDA) for ivosidenib for the treatment of patients with R/R AML with an IDH1 mutation.
Received FDA clearance of an IND application for AG-270, a MAT2A inhibitor, for the treatment of MTAP-deleted tumors.
Presented new and updated data from the IDH and PKR programs at the 2017 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition (ASH) (Free ASH Whitepaper):
First data from the expansion phase of the ongoing Phase 1 trial of ivosidenib in IDH1m R/R AML and advanced hematologic malignancies
First data from the ongoing Phase 1 combination trial of ivosidenib or enasidenib with standard-of-care intensive chemotherapy in patients with newly diagnosed AML with an IDH1 or IDH2 mutation
First data from the ongoing Phase 1/2 combination trial of ivosidenib or enasidenib with VIDAZA in patients with newly diagnosed AML with an IDH1 or IDH2 mutation ineligible for intensive chemotherapy
Updated data from the AG-348 Phase 2 DRIVE PK study in PK deficiency
Appointed Jacqualyn "Jackie" Fouse, Ph.D., former president and chief financial officer of Celgene, to Agios’ board of directors.
Presented updated data from the glioma expansion cohort of the ongoing Phase 1 trial of ivosidenib in advanced IDH1m positive solid tumors at the 2017 Society for NeuroOncology Annual Meeting.
FULL YEAR 2017 FINANCIAL RESULTS

Cash, cash equivalents and marketable securities as of December 31, 2017 were $567.8 million, compared to $573.6 million as of December 31, 2016. The decrease in cash was driven by expenditures to fund operations of $306.8 million during the year ended December 31, 2017. These expenditures were offset by an increase in cash driven by net proceeds of $270.2 million from the April follow on offering, $17.0 million of cost reimbursements under our collaboration agreements with Celgene and $14.2 million received from employee stock transactions.

Revenue for the year ended December 31, 2017 was $43.0 million, which includes $41.1 million of collaboration revenue and $1.9 million of royalty revenue from net sales of IDHIFA. Revenue for the year ended December 31, 2016 was $69.9 million, which included a $25.0 million milestone payment related to the initiation of the Phase 3 IDHENTIFY trial with IDHIFA under the 2010 Agreement.

Research and development (R&D) expenses were $292.7 million, including $30.8 million of stock-based compensation expense, for the year ended December 31, 2017, compared to $220.2 million, including $25.4 million in stock-based compensation expense, for the year ended December 31, 2016. The increase in R&D expense was primarily attributable to the ivosidenib program, including manufacturing and regulatory activities to prepare the NDA submission, start-up costs for the Phase 3 AGILE clinical trial, and on-going site activation and patient enrollment of the Phase 3 ClarIDHy clinical trial. R&D expense also increased compared to the prior year due to IND enabling activities for AG-270.

General and administrative (G&A) expenses were $71.1 million, including $17.0 million of stock-based compensation expense, for the year ended December 31, 2017, compared to $50.7 million, including $16.7 million of stock-based compensation expense, for the year ended December 31, 2016. The increase in G&A expense was primarily attributable to an increase of $21.1 million to support our growing commercial organization for the launch of IDHIFA and the potential launch of ivosidenib in 2018.

Net loss for the year ended December 31, 2017 was $314.7 million, compared to a net loss of $198.5 million for the year ended December 31, 2016.

CASH GUIDANCE

In January, Agios completed an underwritten public offering of 8,152,986 shares of common stock, which includes the full exercise of the underwriters’ option to purchase an additional 1,063,433 shares, at the offering price of $67.00 per share, resulting in proceeds, net of underwriting discounts and commissions, of approximately $516.2 million.

The company expects that its cash, cash equivalents and marketable securities as of December 31, 2017, together with the net proceeds from the recent financing, anticipated product and royalty revenue, anticipated interest income, and anticipated expense reimbursements, but excluding any additional program-specific milestone payments, will enable the company to fund its anticipated operating expenses and capital expenditure requirements through at least the end of 2020.

CONFERENCE CALL INFORMATION

Agios will host a conference call and live webcast with slides today at 8:00 a.m. ET to discuss fourth quarter and full year 2017 financial results and recent business activities. To participate in the conference call, please dial 1-877-377-7098 (domestic) or 1-631-291-4547 (international) and referring to conference ID 3198522. The live webcast can be accessed under "Events & Presentations" in the Investors section of the company’s website at www.agios.com. The archived webcast will be available on the company’s website beginning approximately two hours after the event.

GT BIOPHARMA ANNOUNCES DR. JEFFREY MILLER, RENOWNED NK CELL CANCER SPECIALIST, WILL PRESENT AT CELL THERAPY SYMPOSIA

On February 13, 2018 GT Biopharma Inc. (OTCQB: GTBP) (Euronext Paris: GTBP.PA) reported that Dr. Jeffrey Miller, Deputy Director of the Masonic Cancer Center, University of Minnesota will be presenting at the Keystone Symposia: Emerging Cellular Therapies: T Cells and Beyond; ‘Novel Ways to Activate and Target NK Cells to Treat Cancer’ (Press release, GT Biopharma , FEB 13, 2018, View Source [SID1234539534]). This presentation will highlight GT Biopharma’s TriKE and TetraKE platforms.

Dr. Miller will be presenting to leaders in the field of cell therapies at the conference on Feb. 14th from 5:00 -7:00 pm MT. He will be discussing GT Biopharma’s unique single-chain, tri-specific NK cell engager (TriKE) and tetra-specific NK cell engager (TetraKE) platforms targeting hematologic malignancies, sarcomas and carcinomas (solid tumors). The presentation will address both the TriKE and TetraKE constructs, as well as our second-generation anti-CD16-IL-15-anti-CD33 TriKE (OXS-C3550); another first-of-its-kind, single-chain, tri-specific NK cell engager (TriKE).

NK cell cancer-killing activity is expected to be increased by bringing the NK cells in close proximity to the cancer cells. This may be achieved by ‘engagers’ that bind to CD16 on the surface of NK cells and bind specific proteins (such as CD33) on the surface of cancer cells, thus forming an immune synapse between the NK cell and the cancer cell. Our lead TriKE, anti-CD16-IL-15-anti-CD33 (OXS-3550) is expected to be in the clinic in the second half of 2018. The TriKE constructs utilize the inclusion of interleukin-15 (IL-15), a peptide that leads to proliferation and activation of the NK cells. This further increases NK cancer-cell killing capabilities and improves their function in the tumor microenvironment (Vallera et al,2016).

Unlike traditional CAR-T platforms, TriKEs are potentially a cost effective cell therapy and not relegated to treating liquid tumors only. GT Biopharma believes that TriKEs are an antibody platform that can be tailored to treat any form of cancer, liquid or solid tumors.

Dr. Jeffrey Miller said, "I am pleased to present additional information regarding these immune-oncology platforms. As a researcher, I continue to believe that both have the potential to generate candidates with the ability to have a significant impact on the treatment of cancer and other diseases."

GT Biopharma Chief Medical officer (CMO) Dr. Raymond Urbanski said, "The TriKE and TetraKE concepts and constructs potentially have significant advantages over current and other development-stage therapies. Dr. Miller is a luminary in NK cell biology and its applications, and we continue to be excited by the potential opportunity related to this technology."

Celyad to Host Key Opinion Leader Meeting on CAR-T Therapy for the Treatment of Blood Cancers

On February 13, 2018 Celyad (Euronext Brussels and Paris, and NASDAQ: CYAD), a clinical-stage biopharmaceutical company, reported that it will host a Key Opinion Leader luncheon on the topic of CYAD-01 (CAR-T NKG2D) cell therapy for the treatment of blood cancers in New York City on Tuesday, February 20 (Press release, Celyad, FEB 13, 2018, View Source [SID1234532519]).
The meeting will feature a presentation by Key Opinion Leader Marco Davila, MD, PhD (Moffitt Cancer Center), who will discuss the evolving treatment landscape of CAR-T therapies in development, as well as the unmet medical need for treating patients with blood cancers, including Acute Myeloid Leukemia (AML). Dr. Davila will be available to answer questions at the conclusion of the event.

Celyad’s management team will provide details about the clinical strategy of its lead candidate, CYAD-01, an autologous CAR-T NKG2D cell therapy in clinical development for patients with hematological and solid cancers.

Marco Davila, MD, PhD is an Associate Member of the Blood and Marrow Transplantation Department at the Moffitt Cancer Center and an Associate Professor of Oncologic Sciences at the University of South Florida. His research is dedicated to developing gene-engineered cell therapies that target cancer cells in animal models of cancer. The goal of this research is to identify optimal cell therapies that can then be evaluated in cancer patients. He has also been the Principal Investigator on several clinical trials using genetically engineered T-cells targeted against malignant B cells. Dr. Davila’s laboratory at the Moffitt Cancer Center is interested in developing further chimeric antigen receptors (CARs) that can target hematologic and solid tumor malignancies. His work has been published in many peer-reviewed medical journals.

This event is intended for institutional investors, sell-side analysts, investment bankers, and business development professionals only. Please RSVP in advance if you plan to attend, as space is limited. For those who are unable to attend in person, a live webcast and replay will be accessible here.

10-K – Annual report [Section 13 and 15(d), not S-K Item 405]

Neurocrine Biosciences has filed a 10-K – Annual report [Section 13 and 15(d), not S-K Item 405] with the U.S. Securities and Exchange Commission .

Chi-Med Completes Enrollment of 527 Patients in Pivotal Phase III FALUCA Trial with Fruquintinib in Lung Cancer

On February 13, 2018 Hutchison China MediTech Limited ("Chi-Med") (AIM/Nasdaq: HCM) reported it has completed patient enrollment of FALUCA, its Phase III pivotal trial of fruquintinib in advanced, third-line, non-small cell lung cancer ("NSCLC") patients in China (Press release, Hutchison China MediTech, FEB 13, 2018, View Source [SID1234523945]). Fruquintinib is a highly selective and potent oral inhibitor of vascular endothelial growth factor receptors ("VEGFR") 1, 2 and 3, that has met its primary endpoint in several Phase II and III clinical trials in China for the treatment of lung, colorectal and gastric cancers. Top-line FALUCA data is expected to be reported in late 2018 when the overall survival ("OS") data is mature and, subject to a positive outcome, would be followed by a second New Drug Application ("NDA") submission thereafter. Fruquintinib’s first NDA, for the treatment of colorectal cancer, was submitted to the China Food and Drug Administration ("CFDA") in June 2017.

About Fruquintinib

Fruquintinib (HMPL-013) is a highly selective small molecule drug candidate that has been shown to inhibit VEGFR 24 hours a day via an oral dose, with lower off-target toxicities compared to other targeted therapies. Its tolerability, along with its clean drug-drug interaction profile demonstrated to date, may enable rational combination with other cancer therapies such as in our ongoing clinical trials of fruquintinib in combination with chemotherapy and targeted therapy. VEGFR plays a pivotal role in tumor-related angiogenesis.

About FALUCA

FALUCA is a randomized, double-blind, placebo-controlled, multi-center, Phase III registration study of fruquintinib targeted at treating patients with advanced non-squamous NSCLC, who have failed two lines of systemic chemotherapy. Patients were randomised at a 2:1 ratio to receive either 5mg of fruquintinib orally once per day, on a three-weeks-on / one-week-off cycle, plus best supportive care ("BSC"); or placebo plus BSC. Randomization was stratified by EGFR gene status and history of treatment by VEGF inhibitors. The primary endpoint is OS, with secondary endpoints including progression free survival ("PFS"), objective response rate (ORR), disease control rate (DCR) and duration of response (DoR). Additional details about this study can be found at clinicaltrials.gov, using identifier NCT02691299.

It was initiated following a similar Phase II clinical trial in 91 third-line NSCLC patients that succeeded in meeting its primary efficacy endpoint of PFS, with no unexpected safety issues. Results were highlighted in an oral presentation at the 17th World Conference on Lung Cancer on December 6, 2016 (clinicaltrials.gov identifier NCT02590965)

Other Fruquintinib Development Programs

Lung cancer in China: Along with FALUCA, fruquintinib is concurrently being studied in a Phase II study in combination with Iressa (gefitinib) in first-line setting for patients with advanced or metastatic NSCLC (clinicaltrials.gov identifier NCT02976116). Preliminary results were highlighted in an oral presentation at the 18th World Conference on Lung Cancer on October 16, 2017.

Colorectal cancer in China: The CFDA acknowledged acceptance of the NDA for fruquintinib for the treatment of patients with advanced colorectal cancer ("CRC") in June 2017. Fruquintinib was subsequently awarded priority review status in view of its significant clinical value, according to a CFDA announcement in September 2017. The NDA is supported by data from the successful FRESCO study, a Phase III pivotal registration trial of fruquintinib in 416 patients with CRC in China, which was highlighted in an oral presentation at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting held on June 5, 2017 (clinicaltrials.gov identifier NCT02314819). The FRESCO study followed an initial Phase I trial in 40 solid tumor patients, a Phase Ib study in 62 CRC patients, and a Phase II clinical trial in 71 CRC patients.

Gastric cancer in China: In October 2017, Chi-Med initiated a pivotal Phase III clinical trial of fruquintinib in combination with Taxol (paclitaxel), known as the FRUTIGA study, for the treatment of over 500 patients with advanced gastric or gastroesophageal junction (GEJ) adenocarcinoma who have progressed after first-line standard chemotherapy (clinicaltrials.gov identifier NCT03223376). The FRUTIGA study followed a Phase I/II clinical trial in 34 patients that demonstrated that combination therapy of fruquintinib and Taxol in such patients was generally well-tolerated with promising tumor response (clinicaltrials.gov identifier NCT02415023).

In China, fruquintinib is jointly developed with Eli Lilly and Company.

United States bridging trial: In December 2017, Chi-Med initiated a multi-center, open-label, Phase I clinical study to evaluate the safety, tolerability and pharmacokinetics of fruquintinib in U.S. patients with advanced solid tumors. Additional details about this study may be found at clinicaltrials.gov, using identifier NCT03251378.