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Agios Reports Fourth Quarter and Full Year 2016 Financial Results and Highlights Key 2017 Milestones

On February 16, 2017 Agios Pharmaceuticals, Inc. (NASDAQ:AGIO), a leader in the field of cellular metabolism to treat cancer and rare genetic diseases, reported business highlights and financial results for the fourth quarter and year ended December 31, 2016 (Press release, Agios Pharmaceuticals, FEB 16, 2017, View Source [SID1234517729]). In addition, Agios highlighted select corporate milestones and data presentations for its preclinical and clinical development programs.

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"Our 2016 accomplishments, including the enasidenib NDA submission with our collaboration partner Celgene and clear proof-of-concept data for our PK deficiency program, demonstrate our ability to transform our scientific discoveries into important precision medicines," said David Schenkein, M.D., chief executive officer at Agios. "In 2017, we are focused on making the transition to a commercial stage company by delivering our lead cancer programs to patients, bringing our first rare genetic disease program into pivotal development and advancing our next research program, focused on MTAP-deleted cancers, into the clinic."

KEY UPCOMING MILESTONES

The company expects to achieve the following key milestones:

IDH Mutant Inhibitors in Hematologic Malignancies

Potential approval of enasidenib in the United States for IDH2m positive relapsed/refractory (R/R) acute myeloid leukemia (AML) in collaboration with Celgene by the end of 2017.
Submit a new drug application (NDA) to the U.S. FDA for ivosidenib (AG-120) for IDH1m positive R/R AML by the end of 2017.
Initiate a global, registration-enabling Phase 3 study (AGILE) combining ivosidenib (AG-120) and VIDAZA in newly diagnosed AML patients with an IDH1 mutation ineligible for intensive chemotherapy in the first half of 2017.
IDH Mutant Inhibitors in Solid Tumors

Complete the dose-escalation phase of the ongoing Phase 1 study of AG-881 in IDHm positive glioma in the first half of 2017.
Rare Genetic Diseases

Finalize design for a global pivotal trial of AG-348 in pyruvate kinase (PK) deficiency in the third quarter of 2017.
Initiate a global pivotal trial of AG-348 in PK deficiency in the first half of 2018.
Cancer Metabolism Research:

Submit an Investigational New Drug (IND) application for the development candidate targeting methylthioadenosine phosphorylase (MTAP)-deleted tumors by the end of 2017. MTAP is a metabolic enzyme that is deleted in approximately 15 percent of all cancers.
ANTICIPATED 2017 DATA PRESENTATIONS

First data from the expansion phase of the ongoing Phase 1 study of ivosidenib (AG-120) in R/R AML in the second half of 2017
First data from the ongoing Phase 1b combination study of enasidenib or ivosidenib (AG-120) with standard-of-care intensive chemotherapy in newly diagnosed AML in the second half of 2017
First data from the cholangiocarcinoma expansion cohort of the ongoing Phase 1 study of ivosidenib (AG-120) in advanced IDH1m positive solid tumors in the first half of 2017
Updated data from the glioma expansion of the ongoing Phase 1 study of ivosidenib (AG-120) in advanced IDH1m positive solid tumors in the second half of 2017
Updated data from AG-348 Phase 2 DRIVE PK study in PK deficiency in both the first and second half of 2017
Updated preclinical data for the program targeting MTAP-deleted tumors at the Keystone Tumor Metabolism Meeting taking place March 5-9, 2017 in Whistler, British Columbia
FOURTH QUARTER 2016 HIGHLIGHTS

Supported Celgene’s submission of an NDA for enasidenib in IDH2m positive R/R AML.
Initiated a global, registration-enabling randomized Phase 3 study (ClarIDHy) for ivosidenib (AG-120) in IDH1m positive advanced cholangiocarcinoma. The FDA also granted ivosidenib Fast Track Designation for the treatment of patients with previously treated, unresectable or metastatic cholangiocarcinoma with an IDH1 mutation.
Completed the dose-escalation phase of the Phase 1 study of AG-881 in IDHm positive hematologic malignancies. The study is now closed for enrollment.
Selected a development candidate targeting MTAP-deleted tumors to enter IND-enabling studies.
FULL YEAR 2016 FINANCIAL RESULTS

Cash, cash equivalents and marketable securities as of December 31, 2016 were $573.6 million, compared to $375.9 million as of December 31, 2015. This increase was driven by cash received under our collaboration agreements with Celgene totaling $258.2 million, which includes a $200 million upfront payment from the May 2016 collaboration agreement, $25 million related to initiation of the enasidenib Phase 3 IDHENTIFY study and $33.2 million of program funding, net proceeds of $162.1 million received from the company’s September 2016 public offering, and $7.9 million from stock award activities. These items were offset by a decrease in cash related to expenditures to fund operating activities and purchases of fixed assets of $230.6 million during the year ended December 31, 2016.

Collaboration revenue was $69.9 million for the year ended December 31, 2016, compared to $59.1 million for the prior year.

Research and development (R&D) expenses were $220.2 million, including $25.4 million of stock-based compensation expense, for the year ended December 31, 2016, compared to $141.8 million, including $17.4 million in stock-based compensation expense, for the year ended December 31, 2015. The increase in R&D expenses was primarily due to increased costs to support advancement of the company’s lead investigational medicines toward later-stage development. Celgene is responsible for all development costs for enasidenib and certain development costs for AG-881 and reimburses the company for development costs incurred for these investigational medicines.

General and administrative (G&A) expenses were $50.7 million, including $16.7 million of stock-based compensation expense, for the year ended December 31, 2016, compared to $36.0 million, including $14.5 million of stock-based compensation expense, for the year ended December 31, 2015. The increase in G&A expense was largely due to increased headcount and other professional expenses to support growing operations.

Net loss for the year ended December 31, 2016 was $198.5 million, compared to a net loss of $117.7 million for the year ended December 31, 2015.

CASH GUIDANCE

Based on its current operating plans, the company expects that its existing cash, cash equivalents and marketable securities as of December 31, 2016, together with anticipated interest income, and anticipated payments from Celgene under our collaboration agreements, 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 2018.

District Court Grants MorphoSys’s Request to Add Second Patent in Lawsuit

On February 15, 2017 MorphoSys AG (FSE: MOR; Prime Standard Segment, TecDAX; OTC: MPSYY) reported that it has added a second patent with US Patent Number 9,200,061 to its lawsuit against Janssen Biotech, and Genmab, A/S (Press release, MorphoSys, FEB 15, 2017, https://www.morphosys.com/media-investors/media-center/district-court-grants-morphosyss-request-to-add-second-patent-in [SID1234556345]). This patent claims methods of treating hematologic cancer associated with the undesired presence of CD38-positive cells by administering antibodies that bind to a specific region of the target molecule, CD38. In a hearing that took place on February 6, 2017 the District Court granted MorphoSys’s request to add the 9,200,061 patent to the case.

On April 4, 2016 MorphoSys filed a lawsuit in the United States (U.S.) District Court of Delaware against Janssen Biotech, and Genmab, A/S for patent infringement of U.S. Patent Number 8,263,746.

By its complaint, MorphoSys seeks redress for infringement by Janssen’s and Genmab’s daratumumab, a CD38-directed monoclonal antibody indicated for the treatment of certain patients with multiple myeloma. Janssen Biotech obtained FDA approval on daratumumab and markets the product as Darzalex(R) in the U.S. and other countries. MorphoSys continues to develop MOR202, its own investigational human antibody to CD38, for the treatment of cancer, including multiple myeloma.

Cytori Completes Acquisition of Azaya Therapeutics Assets, Initiates Nanomedicine Program

On February 15, 2017 Cytori Therapeutics, Inc. (NASDAQ:CYTX) reported it has completed its acquisition of assets of privately held Azaya Therapeutics, Inc., a leader in the research, development and manufacturing of nanoparticle therapeutics (the "Acquisition"). The Acquisition provides Cytori with a proprietary liposomal nanoparticle technology platform that is intended to complement Cytori’s leadership position in regenerative medicine and expand its pipeline with two promising nanoparticle oncology drugs.

"This acquisition is a very important step in the development of Cytori," said Dr. Marc Hedrick, President and Chief Executive Officer of Cytori. "The Acquisition provides Cytori both a near term opportunity to seek regulatory approval in Europe for the acquired ATI-0918 drug candidate, a generic form of nanoparticle encapsulated doxorubicin, with the goal of launching it in Europe as early as 2019, and bolsters Cytori’s early and intermediate stage pipeline with new drug candidates in regenerative medicine and oncology".

Cytori plans to develop nanoparticle-based therapeutics under the name Cytori Nanomedicine in the areas of oncology and regenerative medicine. In addition to ATI-0918, the generic nanoparticle encapsulated doxorubicin, Cytori has added the clinical stage ATI-1123 drug candidate, a protein-stabilized nanoparticle formulation of docetaxel and a preclinical stage regenerative medicine drug candidate for scleroderma.

Medtronic’s OsteoCool(TM) RF Ablation System Receives Expanded Indication for Palliative Treatment of Metastatic Bone Tumors

On February 15, 2017 Medtronic plc (NYSE: MDT) reported that the U.S. Food and Drug Administration (FDA) has cleared an expanded indication for the OsteoCool(TM) RF Ablation System (Press release, Medtronic, FEB 15, 2017, View Source;p=RssLanding&cat=news&id=2246172 [SID1234517728]). Originally cleared for use in the spine, the FDA now allows the marketing of the OsteoCool System for palliative treatment of metastases in all bony anatomy – such as ribs, sacrum, extremities, and hip – in patients who have failed or are not candidates for standard therapy. The system uses targeted radiofrequency energy to ablate malignant metastatic bone tumors.

"Patients with metastatic bone cancer may be treated with conventional therapies such as opioids, chemotherapy or radiation therapy for pain palliation," said Sandeep Bagla, M.D., an interventional radiologist with the Vascular Institute of Virginia in Woodbridge, Va. "With the expanded indication for the OsteoCool System, I now have the option to ablate these patients’ painful bone tumors when conventional therapies are considered ineffective, too slow-acting or cause unacceptable side effects."1

Metastatic bone disease has been reported to occur in 60-80 percent of cancer patients, most frequently among patients with primary malignancies of the breast, prostate, liver, and lung.2 More than 80 percent of bone metastases are found in the axial skeleton, which includes the skull, spine, and ribs.3
"Our Pain Therapies business is deeply rooted in the Medtronic Mission – which calls us to alleviate pain,"said Jeff Cambra, general manager of the Pain Therapies Interventional business, which is part of the Restorative Therapies Group at Medtronic. "With this expanded indication, we put an important treatment option into the hands of physicians so that they can help more patients suffering from debilitating pain."
Medtronic acquired the OsteoCool technology and associated intellectual property from Baylis Medical on December 16, 2015 and partnered with the company to further innovate the system.
"We’re pleased to broaden our partnership with Medtronic to improve the treatment of patients suffering from painful metastases," said Kris Shah, president of Baylis Medical. "The expansion of the OsteoCool System to include the ablation of malignant lesions in bone adds to our company’s track record of offering innovative clinical solutions and further enhancing patient access and treatment options."
The OsteoCool system is the only cooled radiofrequency (RF) ablation technology that offers simultaneous, dual-probe capabilities – providing for procedural flexibility and predictable, customized treatment. The system is temperature controlled and uses internally water-cooled probes to prevent overheating of surrounding tissue during the procedure.
The device also has a CE mark for the ablation of benign bone tumors such as osteoid osteoma and palliative treatment of metastatic malignant lesions involving bone, including the vertebral body. This indication is not available in the United States.

TRILLIUM THERAPEUTICS TO PRESENT NEW DATA FROM TTI-621 PHASE 1 TRIAL

On February 15, 2017 Trillium Therapeutics Inc. (NASDAQ/TSX: TRIL), a clinical-stage immuno-oncology company developing innovative therapies for the treatment of cancer, reported that it will present new data from the Phase 1 trial of its anti-CD47 checkpoint inhibitor TTI-621 at the ASCO (Free ASCO Whitepaper)-SITC Clinical Immuno-Oncology Symposium on February 24, in Orlando, Florida (Press release, Trillium Therapeutics, FEB 15, 2017, View Source [SID1234517738]).

The presentation will highlight new pharmacokinetic and pharmacodynamic data from patients having received multiple weekly infusions of TTI-621. After 6 weeks of treatment, the terminal serum half-life of TTI-621 is significantly increased compared to the first infusion and is accompanied by an increase in circulating drug levels and target receptor occupancy. These data suggest that repeat dosing of TTI-621 overcomes the antigen sink and achieves circulating drug concentrations that are associated with biological activity in preclinical studies. The presentation will also feature an expanded pharmacodynamic biomarker analysis, with a focus on cytokines associated with macrophage-mediated phagocytic activity.

"These evolving data significantly advance our understanding of TTI-621 pharmacology," said Trillium’s Chief Scientific Officer, Dr. Bob Uger. "We believe continued weekly dosing overcomes the antigen sink while maintaining clinically acceptable platelet levels. In fact, emerging evidence suggests that the transient decrease in platelets observed immediately following TTI-621 exposure was attenuated in most patients receiving multiple infusions."

Leerink Partners Presentation

Trillium will provide a corporate update at Leerink Partners 6th Annual Global Healthcare Conference at the Lotte New York Palace Hotel at 3:30 pm ET today. A live audio webcast of this presentation will be available under the investor relations section of Trillium’s website at www.trilliumtherapeutics.com. A replay of the presentation will be available following the event.