On March 1, 2017 Intrexon Corporation (NYSE: XON), a leader in the engineering and industrialization of biology to improve the quality of life and health of the planet, reported its fourth quarter and full year financial results for 2016 (Press release, Intrexon, MAR 1, 2017, View Source [SID1234517933]).

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Business Highlights and Recent Developments:

Oxitec, a wholly-owned subsidiary of Intrexon, opened its large scale mosquito production facility in Brazil with the capacity to produce 60 million Friendly Aedes per week. Given ongoing discussions in Brazil management expects the factory’s egg capacity will be committed within 2017;
Oxitec expanded its Friendly Aedes project in Piracicaba, Brazil with the initiation of releases of self-limiting Aedes aegypti mosquitoes in ten additional neighborhoods in the city’s center covering an additional 60,000 people;
The Cayman Islands Mosquito Research and Control Unit commenced operational roll-out of Oxitec’s Friendly technology in West Bay, Grand Cayman, in July 2016 as the first phase of an anticipated island-wide deployment, and recent results indicate the program is on track;
Okanagan Specialty Fruits (OSF) achieved the first commercial harvest of its non-browning Arctic Golden apple variety and plans commercial launch of fresh sliced apples in select markets across North America in the fall of 2017;
Together with collaborator ZIOPHARM Oncology, Inc. (NASDAQ: ZIOP) announced signing of a Cooperative Research and Development Agreement (CRADA) with the National Cancer Institute (NCI) for the development of adoptive cell transfer-based immunotherapies using autologous peripheral blood lymphocytes genetically modified using the Sleeping Beauty system to express T-cell receptors for the treatment of solid tumors in patients with advanced cancers;
Collaborator ZIOPHARM announced end-of-phase 2 meeting with the FDA for Ad-RTS-hIL-12 + veledimex in recurrent glioblastoma and expects to announce the outcome of this meeting in the first quarter with the goal of initiating a pivotal clinical trial in 2017;
Collaborator ZIOPHARM announced improved production times in its ongoing Phase I trial of 2nd generation Sleeping Beauty CD19+ CAR-T cells and progress toward its "Point-of-Care" approach. One patient with multiple-relapsed acute lymphoblastic leukemia achieved a complete response and a patient with triple-hit non-Hodgkin lymphoma was treated with T cells manufactured in 2 weeks;
Collaborator ZIOPHARM presented promising pre-clinical data at the 58th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting that a single, low-dose of 3rd gen Sleeping Beauty CAR+ T cells co-expressing a CD19-specific CAR and membrane-bound IL15 produced in <2 days resulted in sustained in vivo persistence, potent anti-tumor effects and superior leukemia-free survival;
Collaborator Fibrocell Science, Inc. (NASDAQ: FCSC) announced dosing of first patient in Phase I portion of Phase I/II clinical trial of FCX-007 gene therapy for treatment of recessive dystrophic epidermolysis bullosa (RDEB);
Established joint ventures in the Health and Food sectors: Intrexon T1D Partners, LLC to develop ActoBiotics based antigen-specific immunotherapy to treat type 1 diabetes in humans, and EnviroFlight, a joint venture with Darling Ingredients, Inc. (NYSE: DAR), to employ black soldier fly in the development of sustainable high quality nutrients for the aquaculture and livestock industries;
Entered into Exclusive Channel Collaborations with Genten Therapeutics, Inc., CRS Bio, Inc., Relieve Genetics, Inc., Exotech Bio, Inc., and AD Skincare, Inc., startups backed by the Harvest Intrexon Enterprise Fund. The collaborations are focused on biologically based delivery of therapeutic molecules to target human health conditions including celiac disease, chronic rhinosinusitis, neuropathic pain, cancer, and aging facial skin, respectively;
Intrexon’s collaboration with a leading global agricultural company utilizing ActoBiotics technology advanced to its next phase of development for biological crop protection solutions following initial studies validating the efficacy of dsRNA for insect control applications;
Exemplar Genetics, a wholly-owned subsidiary of Intrexon, was awarded a subcontract with Leidos Biomedical Research, Inc., prime contractor for the Frederick National Laboratory for Cancer Research, sponsored by the National Cancer Institute, to support the NIH’s National Center for Advancing Translational Sciences in creating genetically engineered miniswine models of sickle cell disease that could potentially lead to new treatments for the disorder;
Two of Intrexon’s subsidiaries achieved additional regulatory approvals: OSF’s non-browning Arctic Fuji apple was granted deregulated status by the U.S. Department of Agriculture’s Animal and Plant Health Inspection Service and Health Canada approved AquaBounty Technologies, Inc. (NASDAQ: AQB, AIM: ABTU) AquAdvantage Salmon for commercial sale in Canada;
Oxitec announced that the Board of the Florida Keys Mosquito Control District voted to approve the investigational agreement for use of self-limiting Friendly mosquitoes in an effectiveness trial following an approval vote by residents in Monroe County;
Oxitec announced plans to move forward with open field trials of its self-limiting Mediterranean fruit fly (medfly) in Australia after a series of successful studies across multiple countries demonstrated the self-limiting medfly’s ability to mate with wild medfly and subsequently suppress the pest population;
Oxitec and Gangabishan Bhikulal Investment and Trading Limited announced initiation of outdoor caged trials in India to demonstrate the efficacy of Oxitec’s Friendly mosquitoes in suppressing the local population of Aedes aegypti, the primary vector for many dangerous viruses including dengue, Zika, and chikungunya. Recently published work estimates dengue alone infects almost 5.8 million people in India annually and the total financial cost of dengue exceeds $1 billion per year;
Four of Intrexon collaborators’ gene therapy programs attained development achievements with the U.S. Food and Drug Administration: Fast Track designation to Fibrocell for FCX-007 for the treatment of RDEB, Fast Track designation to Oragenics, Inc. (NYSE MKT: OGEN) for ActoBiotics AG013 for the treatment of oral mucositis, Orphan Drug designation to Agilis Biotherapeutics’ AGIL-FA for the treatment of Friedreich’s ataxia, and Orphan Drug designation to Fibrocell for FCX-013 for the treatment of linear scleroderma;
AquaBounty completed the listing of its common shares on the NASDAQ Stock Market and completed an equity subscription from Intrexon. In conjunction with the listing on NASDAQ, Intrexon distributed a special stock dividend of shares of AquaBounty common stock it owned to its shareholders while maintaining majority ownership of AquaBounty’s outstanding common stock; and
Entered into a definitive agreement to acquire GenVec, Inc. (NASDAQ: GNVC), a clinical-stage company and pioneer in the development of AdenoVerse gene delivery technology, with the goal of integrating and expanding upon GenVec’s expertise in adenoviral (AdV) vectors and cGMP drug product manufacturing to enhance Intrexon’s broad gene transfer capabilities that encompass multiple viral and non-viral platforms and develop a next generation AdV platform with significantly higher payload capacity compared to current systems.

Fourth Quarter Financial Highlights:

Total revenues of $46.0 million, an increase of 11% over the fourth quarter of 2015;
Net loss of $44.1 million attributable to Intrexon, or $(0.37) per basic share, including non-cash charges of $38.9 million;
Adjusted EBITDA of $(5.8) million, or $(0.05) per basic share;
The net change in deferred revenue related to upfront and milestone payments, which represents the cash and stock received from collaborators less the amount of revenue recognized during the period, was a decrease of $11.3 million compared to a net increase of $22.3 million in the fourth quarter of 2015;
Cash consideration received for reimbursement of research and development services covered 54% of cash operating expenses (exclusive of operating expenses of consolidated subsidiaries);
Total consideration received for technology access fees, reimbursement of research and development services and products and services revenues covered 61% of consolidated cash operating expenses; and
Cash, cash equivalents, and short-term and long-term investments totaled $243.2 million, the value of investment in preferred stock totaled $129.5 million, and the value of equity securities totaled $23.5 million at December 31, 2016.

Full Year Financial Highlights:

Total revenues of $190.9 million, an increase of 10% over the full year ended December 31, 2015;
Net loss of $186.6 million attributable to Intrexon, or $(1.58) per basic share, including non-cash charges of $159.0 million;
Adjusted EBITDA of $(26.6) million, or $(0.23) per basic share;
The net increase in deferred revenue related to upfront and milestone payments was $116.5 million compared to $74.1 million in the full year ended December 31, 2015;
Cash consideration received for reimbursement of research and development services covered 57% of cash operating expenses (exclusive of operating expenses of consolidated subsidiaries); and
Total consideration received for technology access fees, reimbursement of research and development services and products and services revenues covered 129% of consolidated cash operating expenses.

"Over the course of 2016, while nevertheless achieving its overall financial goals, significantly advancing a great many of its partnered programs, and meaningfully extending its technology platforms, the Company faced political and regulatory headwinds in our marketable products portfolio that we had not fully appreciated at this time last year, causing us to underachieve commercially as compared with our expectations," commented Randal J. Kirk, Chairman and Chief Executive Officer of Intrexon.

"Operationally, however, we executed exceptionally well. We essentially balanced cost recovery, deal money, and products and services revenues to our cash operating expenses, thus maintaining our desired capital efficiency, strengthened our leadership team, moved many of our developmental programs forward in the lab, the field or the clinic, and our more than 600 scientists extended our leadership in the engineering of biology by providing several new technology platforms that should enable many novel, valuable and differentiated products. Several of these already have drawn partnering interest, and others should add significant incremental value to some of the Company’s existing partnerships."

"In addition, we are evaluating structural alternatives concerning our business in healthcare as we appreciate that, as compared with the rest of our business, healthcare is unique as an industry, having a discrete shareholder base, methods of measurement and a vastly greater industrial maturity."

Mr. Kirk concluded, "We therefore view our prospects for 2017 with the highest expectations for performance. We more than ever are confident that Intrexon can lead the greatest industrial vector in history."

Fourth Quarter 2016 Financial Results Compared to Prior Year Period

Total revenues increased $4.5 million, or 11%, over the quarter ended December 31, 2015. Collaboration and licensing revenues increased $6.6 million from the quarter ended December 31, 2015 due to (i) the recognition of deferred revenue for upfront payments received from collaborations signed by the Company in 2016, including the consideration received in June 2016 from ZIOPHARM to amend the collaborations between us; and (ii) increased research and development services for these collaborations and for the expansion of programs or the addition of new programs with previously existing collaborators. Product revenues decreased $1.5 million, or 17%, and gross margin decreased from the quarter ended December 31, 2015. The decrease in product revenues and gross margin primarily relates to a decrease in the quantities of pregnant cows, livestock previously used in production and live calves sold due to lower customer demand for these products and also due to a decline in average sales price of livestock previously used in production. Service revenues and gross margins were consistent quarter over quarter.

Research and development expenses increased $2.8 million, or 11%, due primarily to increases in (i) salaries, benefits and other personnel costs for research and development employees hired to support new or expanded collaborations, (ii) lab supplies and consulting expenses incurred as our collaborator programs progress towards the clinical phase, and (iii) amortization of intangible assets which commenced upon regulatory approvals received by our subsidiaries. While selling, general and administrative (SG&A) expenses were generally flat quarter over quarter, legal and professional expenses increased $4.7 million due to (i) expenses incurred to support domestic and international government affairs for regulatory and other approvals necessary to commercialize the Company’s products and services; and (ii) increased legal fees to defend ongoing litigation. Salaries, benefits and other personnel costs for SG&A employees decreased $6.0 million primarily due to a decrease in performance-based cash incentives for the Company’s executive officers in 2016, partially offset by the costs of increased headcount, including the hiring of two new executive officers and additional business development professionals.

Total other income (expense), net, decreased $12.4 million, or 336%, from the quarter ended December 31, 2015. This decrease was attributable to the $16.0 million unrealized and realized losses recognized on the Company’s equity securities portfolio, partially offset by dividend income from the Company’s investment in preferred stock.

Full Year 2016 Financial Results Compared to Prior Year Period

Total revenues increased $17.3 million, or 10%, over the year ended December 31, 2015. Collaboration and licensing revenues increased $22.1 million over the year ended December 31, 2015 due to (i) the recognition of deferred revenue for upfront payments received from collaborations signed by the Company in 2016, including the consideration received in June 2016 from ZIOPHARM to amend the collaborations between us; and (ii) increased research and development services for these collaborations and for the expansion of programs or the addition of new programs with previously existing collaborators. This increase is partially offset by the recognition in 2015 of previously deferred revenue related to collaboration agreements for which the Company satisfied all of its obligations or which were terminated during 2015. Product revenues decreased $4.9 million, or 12%, and gross margin decreased from the year ended December 31, 2015. The decrease in product revenues and gross margin primarily relates to a decrease in the quantities of pregnant cows, livestock previously used in production and live calves sold due to lower customer demand for these products and also due to a decline in average sales price of livestock previously used in production. Service revenues and gross margin on services were consistent year over year.

Research and development expenses decreased $35.3 million, or 24%, due primarily to the inclusion in 2015 of a $59.6 million payment in common stock for an exclusive license to certain technologies owned by the University of Texas MD Anderson Cancer Center. This decrease was partially offset by increases in (i) salaries, benefits and other personnel costs for research and development employees, (ii) lab supplies and consulting expenses, and (iii) depreciation and amortization. Salaries, benefits and other personnel costs increased $7.3 million due to (i) an increase in research and development headcount to support new and expanded collaborations and (ii) a full year of costs for research and development employees assumed in 2015 acquisitions. Lab supplies and consulting expenses increased $10.6 million as a result of (i) the progression into the preclinical phase with certain collaborators; (ii) the increased level of research and development services provided to collaborators; and (iii) a full year of compensation costs incurred for employees assumed in 2015 acquisitions. Depreciation and amortization increased $5.7 million primarily as a result of (i) the inclusion of a full year of depreciation and amortization on property, equipment and intangible assets assumed in 2015 acquisitions and (ii) a full year of amortization of AquaBounty’s intangible assets which commenced upon regulatory approval in November 2015. SG&A expenses increased $33.3 million, or 31%, over the year ended December 31, 2015. Salaries, benefits and other personnel costs for SG&A employees increased $3.2 million due to (i) increased headcount, including the hiring of two new executive officers and additional business development professionals; (ii) a full year of non-cash compensation paid to the Company’s CEO pursuant to the compensation agreement into which the Company entered in November 2015; and (iii) a full year of salaries, benefits and other personnel costs for employees assumed in 2015 acquisitions. These increases were partially offset by a decrease in performance-based cash incentives for our executive officers in 2016. Legal and professional expenses increased $18.6 million primarily due to (i) a full year of non-cash consulting expenses pursuant to the Company’s services agreement with Third Security into which the Company entered in November 2015; (ii) expenses incurred to support domestic and international government affairs for regulatory and other approvals necessary to commercialize the Company’s products and services; (iii) increased legal fees to defend ongoing litigation; and (iv) incremental costs incurred to support the 2015 acquisitions and other business development activities. In 2016, the Company also recorded $4.3 million in litigation expenses arising from the entrance of a court order in Trans Ova’s trial with XY, LLC.

Total other income (expense), net, decreased $116.7 million, or 170%, from the year ended December 31, 2015. This decrease was attributable to the $81.4 million realized gain recognized upon the special stock dividend of all of Intrexon’s shares of ZIOPHARM to the Company’s shareholders in June 2015 and the decrease in fair value of the Company’s equity securities portfolio. These decreases were partially offset by preferred stock dividend income received from ZIOPHARM.

Conference Call and Webcast

The Company will host a conference call today Wednesday, March 1st, at 5:30 PM ET to discuss the fourth quarter and full year 2016 financial results and provide a general business update. The conference call may be accessed by dialing 1-888-317-6003 (Domestic US), 1-866-284-3684 (Canada), and 1-412-317-6061 (International) and providing the number 2431343 to join the Intrexon Corporation Call. Participants may also access the live webcast through Intrexon’s website in the Investors section at View Source

On March 1, 2017 Geron Corporation (Nasdaq:GERN) reported financial results for the fourth quarter and year ended December 31, 2016 and recent events (Press release, Geron, MAR 1, 2017, View Source [SID1234517930]).

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Fourth Quarter and Year-End 2016 Results

For each of the fourth quarter of 2016 and 2015, the company reported a net loss of $8.5 million, or $(0.05) per share. For 2016, the company reported a net loss of $29.5 million, or $(0.19) per share, compared to net income of $46,000, or $0.00 per share, for 2015. The company ended 2016 with $129.1 million in cash and investments.

Revenues for the fourth quarter of 2016 were $94,000 compared to $220,000 for the comparable 2015 period. Revenues for 2016 were $6.2 million compared to $36.4 million for 2015. Revenues for 2016 included the full recognition of an upfront payment of $5.0 million from Janssen Pharmaceuticals, Inc. under a license agreement for certain rights to the company’s specialized oligonucleotide backbone chemistry and novel amidates. Revenues for 2015 included the full recognition of the $35.0 million upfront payment from Janssen Biotech, Inc. (Janssen) as collaboration revenue upon the company’s transfer of the imetelstat license rights and completion of technology transfer-related activities outlined under the imetelstat collaboration agreement with Janssen. The upfront cash payment was received in December 2014 and recorded as deferred revenue at that time.

Total operating expenses for each of the fourth quarter of 2016 and 2015 were $8.9 million. Total operating expenses for 2016 were $36.8 million compared to $36.9 million for 2015. Operating expenses for 2015 included restructuring charges of $1.3 million in connection with the company’s organizational resizing announced in March 2015.

Research and development expenses for the fourth quarter of 2016 were $4.1 million compared to $4.0 million for the comparable 2015 period. Research and development expenses for 2016 were $18.0 million compared to $17.8 million for 2015. The increase in research and development expenses in 2016 compared to 2015 primarily reflected the net result of higher costs for the company’s proportionate share of clinical development expenses under the imetelstat collaboration with Janssen, partially offset by reduced personnel-related costs resulting from the March 2015 organizational resizing and lower costs for the manufacturing of imetelstat drug product.

General and administrative expenses for the fourth quarter of 2016 were $4.8 million compared to $4.9 million for the comparable 2015 period. General and administrative expenses for 2016 were $18.8 million compared to $17.8 million for 2015. The increase in general and administrative expenses in 2016 compared to 2015 primarily reflected the net result of higher non-cash stock-based compensation expense and an increased allocation of facilities and other overhead costs to general and administrative activities, partially offset by lower consulting and legal costs.

Interest and other income for the fourth quarter of 2016 was $321,000 compared to $196,000 for the comparable 2015 period. Interest and other income for 2016 was $1.2 million compared to $677,000 for 2015. The increase in interest and other income for 2016 compared to 2015 primarily reflected higher yields on the company’s marketable securities portfolio.

"In 2016, the imetelstat program progressed with the ongoing clinical trials in patients with myelofibrosis and myelodysplastic syndromes being conducted by Janssen, as well as through a range of preclinical and translational studies in collaboration with academic scientists around the world," said John A. Scarlett, M.D., Geron’s President and Chief Executive Officer. "2017 will be another important year for imetelstat. We expect Janssen to conduct the second internal reviews of data from IMbark and IMerge to inform Janssen’s decisions regarding further development plans for the drug, including prospects around dosing in IMbark and opening the Phase 3 part of IMerge. We expect Janssen’s decision-making to occur in the second quarter of 2017."

2016 EVENTS SUMMARY

Imetelstat Clinical Development by Janssen

In January, the first patient was dosed in IMerge, a Phase 2/3 clinical trial to evaluate imetelstat in transfusion dependent patients with IPSS low or intermediate-1 risk myelodysplastic syndromes (MDS) who have relapsed after or are refractory to prior treatment with an erythropoiesis stimulating agent (ESA). IMerge is the second clinical trial to be initiated and conducted by Janssen under the terms of the exclusive worldwide imetelstat license and collaboration agreement between Geron and Janssen. The first, IMbark, was designed to evaluate two dose levels of imetelstat in patients with intermediate-2 or high risk myelofibrosis (MF) who have relapsed after or are refractory to prior treatment with a JAK inhibitor.

In September, Janssen completed planned internal reviews of data from IMbark and IMerge. As a result of these data reviews, both trials are continuing in order to obtain additional and more mature data. While patients remaining in the treatment phase of IMbark and IMerge continue to be dosed with imetelstat, new patient enrollment in both trials is suspended until completion of the second internal data reviews.
Publications and Presentations

In March, Blood Cancer Journal published clinical safety and efficacy data on imetelstat from patients with a form of MDS known as refractory anemia with ringed sideroblasts (MDS-RARS) enrolled as part of the Mayo Clinic Pilot Study. The data included nine patients enrolled in the study cohort, classified as having either IPSS intermediate-1 or intermediate-2 risk disease. Three of the eight (37.5%) patients who were dependent on red blood cell transfusions at study entry became transfusion independent for at least eight weeks. The median duration of transfusion independence was 28 weeks (range: nine weeks to 37 weeks).

At the American Association for Cancer Research (AACR) (Free AACR Whitepaper) annual meeting in April, non-clinical data on imetelstat were presented describing:

– Results from a study in which imetelstat treatment of acute myeloid leukemia (AML) cell lines enhanced the effects of hypomethylating agents currently used for the treatment of AML.

– Results from non-clinical studies that provide further evidence of potential on-target mechanisms of telomerase inhibition by imetelstat underlying the reduction in platelets observed in previously conducted imetelstat clinical trials.

At the American Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting in December, data related to the imetelstat program were presented describing:

– Results from a non-clinical study showing that treatment with imetelstat prolonged overall survival of AML xenografts derived from nine out of 15 individual patient samples compared to saline-treated controls.

– An analysis of treatment patterns and outcomes of patients with MF from two United States medical health insurance claims databases showing a median overall survival of seven months among patients who failed or discontinued frontline ruxolitinib.

– Telomere length dynamics from the prior Geron-sponsored proof-of-concept study in patients with essential thrombocythemia (ET) showing that in 10 out of 13 patients, telomere length in granulocytes was higher after nine months of treatment with imetelstat and the change correlated with the reduction of JAK2V617F mutational burden, suggesting that imetelstat may suppress neoplastic clones and favor recovery of normal hematopoiesis in these patients.

– A preliminary investigation suggesting that imetelstat treatment reduces the number of leukemia progenitor cells detected in bone marrow from xenograft models of chronic myeloid leukemia in blast crisis.
These publications and posters are available through the Publications page in the R&D section of Geron’s website (www.geron.com).

Intellectual Property

In July, U.S. patent 9,375,485 related to imetelstat was issued by the U.S. Patent and Trademark Office with claims covering the use of telomerase inhibitor compounds, including imetelstat, for alleviating at least one symptom of myelofibrosis or myelodysplastic syndromes, including chronic myelomonocytic leukemia, which is expected to remain in force until at least March 2033.
FUTURE EVENTS

In the first quarter of 2017, Janssen initiated the process for the second internal data reviews for both IMbark and IMerge. The company expects the outcomes from the second internal data reviews, regulatory considerations and the totality of other program information, including the evolving treatment landscapes in MF and MDS, to inform Janssen’s decisions regarding future development plans for imetelstat.

Data from the Phase 2 part of IMerge is expected to be submitted by Janssen for presentation at a medical conference in 2017.

On March 1, 2017 FibroGen, Inc. (NASDAQ:FGEN), a science-based biopharmaceutical company, reported financial results for the year ended December 31, 2016, and provided an update on the company’s recent developments (Press release, FibroGen, MAR 1, 2017, View Source [SID1234517929]).

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"We are pleased with the topline results reported from our two China Phase 3 trials, which are the first Phase 3 readouts for roxadustat, and bring us closer to completing our new drug application submission in China. With our partners AstraZeneca and Astellas, we are making steady progress in Phase 3 clinical development worldwide for roxadustat in CKD anemia, and continue to expect filing of our U.S. NDA in 2018," said Thomas B. Neff, FibroGen’s Chief Executive Officer. "This year, we are expanding development activities for roxadustat into oncology-related anemias. We are also anticipating important clinical milestones for pamrevlumab, or FG-3019. We began the year with the presentation and publication of promising clinical results in pancreatic cancer, and expect to report data from our placebo-controlled Phase 2 trial in idiopathic pulmonary fibrosis in the third quarter of this year."

Recent Developments
U.S. and Europe Roxadustat (FG-4592) Anemia in Chronic Kidney Disease (CKD)

Met initial target enrollment for three FibroGen-sponsored Phase 3 trials supporting U.S. and EU approvals and are continuing to enroll patients to meet overall enrollment goals among the partners
The independent data safety monitoring board (DSMB), which reviews the U.S. and European Phase 3 program quarterly, recommended in February that trials continue without modification to current protocols
China Roxadustat Anemia in Chronic Kidney Disease

Completed the controlled portion of our two Phase 3 trials in 2016
These studies met their primary endpoints for the treatment of anemia in non-dialysis and dialysis patients
— In non-dialysis patients, roxadustat achieved a significantly greater increase in mean Hb and a significantly greater Hb response rate, as compared to patients in the placebo arm
— In dialysis patients, roxadustat met the predefined non-inferiority criteria for the primary efficacy endpoint. In the pre-specified sequential analysis, roxadustat was also shown to be superior to 利血宝 (Li Xue Bao) epoetin alfa (Kirin EPO) in mean Hb increase
Japan Roxadustat Anemia in Chronic Kidney Disease

All six Phase 3 trials are now underway
Presented Phase 2 study results in non-dialysis-dependent patients in a clinical late-breaker session of the November 2016 American Society of Nephrology (ASN) Kidney Week
U.S. Roxadustat Oncology-Related Anemias

Phase 3 investigational new drug application was approved by the FDA for the treatment of anemia in myelodysplastic syndromes (MDS) in the fourth quarter of 2016
China Roxadustat Oncology-Related Anemias

Submitted a clinical trial application (CTA), which is under review by the China Food and Drug Administration (CFDA), for a Phase 2/3 clinical study of anemia in MDS
Pamrevlumab in Idiopathic Pulmonary Fibrosis (IPF)

Completed enrollment of the Phase 2 double-blind, placebo-controlled -067 study
Results from Study -049 were published in the European Respiratory Journal, accompanied by an editorial, and the corresponding open-label extension results were presented at the 19th International Colloquium on Lung and Airway Fibrosis (ICLAF)
— No safety issues reported with long-term treatment
— Trend towards improved or stable pulmonary function and stable fibrosis continued in second year of treatment
Pamrevlumab in Pancreatic Cancer

Presented findings at the 2017 Gastrointestinal Cancers Symposium (ASCO-GI) from an ongoing open-label, randomized Phase 1/2 study in locally advanced pancreatic cancer -069 showing improvement in survival among patients in the combination arm, as compared to chemotherapy alone
Published Phase 1/2 trial results in the Journal of Cancer Clinical Trials reporting statistically significant dose-related increase in survival and that pamrevlumab can be safely combined with chemotherapy standard-of-care in advanced pancreatic cancer
Corporate and Financial Highlights

Net loss per basic and diluted share for the year ended December 31, 2016 was $0.98, as compared to $1.42 a year ago
At December 31, 2016, FibroGen had $342.2 million of cash, restricted time deposits, cash equivalents, investments, and receivables
Outlook

Report pamrevlumab topline Phase 2 IPF data, placebo-controlled and combination treatment sub-study results, in the third quarter
Submit new drug application in China in the third quarter
Expect to complete enrollment of pamrevlumab open-label, randomized Phase 2 trial in locally advanced pancreatic cancer patients in the first half of the year, and to complete the patient treatment period by year-end
On track to submit the NDA for roxadustat in the U.S. in 2018

On March 1, 2017 Puma Biotechnology, Inc. (NASDAQ: PBYI), a biopharmaceutical company, reported publication of abstracts on neratinib for the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2017 (Press release, Puma Biotechnology, MAR 1, 2017, View Source [SID1234517928]). The AACR (Free AACR Whitepaper) Annual Meeting will be held at the Walter E. Washington Convention Center in Washington, D.C. from April 1 to April 5.

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Full abstracts of the following presentations are available online at www.aacr.org :

Apr. 4, 2017, 1:00 – 5:00 p.m. EDT – Abstract 4818 (Poster): Neratinib/fulvestrant but not fulvestrant alone maintain complete tumor responses after treatment with trastuzumab + paclitaxel of mice bearing ER+/HER2+ xenografts.
L.J. Schwarz et al, Vanderbilt University Medical Center.

April 4, 2017, 1:00 – 5:00 p.m. EDT – Abstract 4157 (Poster): Co-blockade of mTORC1, ERBB and estrogen receptor signaling pathways in endocrine resistant breast cancer: combating tumour plasticity.
R. Ribas et al, Institute of Cancer Research.

April 4, 2017, 1:00 – 5:00 p.m. EDT – Abstract 4038 (Poster): Exploring optimal targeted combination therapies with neratinib for HER2+ breast cancer.
M. Zhao et al, MD Anderson Cancer Center.

April 5, 2017, 8:00 – 12:00 p.m. EDT – Abstract 5167 (Poster): Stem-like colorectal cancer cell lines show response to the ERK1/2 inhibitor, SCH772984, alone and in combination with neratinib while the combination of MEK-162 and neratinib work to decrease tumor growth in inflammatory colorectal cancer subtypes.
R. Pal et al, NSABP.

April 5, 2017, 8:00 – 12:00 p.m. EDT – Abstract 5684 (Poster): NSABP FC-7 Correlative Study: HER2 amplification in circulating cell-free DNA (cfDNA) in metastatic colorectal cancer (mCRC) resistant to anti-EGFR therapy.
S. Rim Kim et al, NSABP.

Full abstracts of the following presentations are expected to be available online March 31, 2017, after 4:00 p.m. EDT:

April 2, 2017, 12:45 – 3:00 p.m. EDT – Abstract CT001 (Oral, Clinical Trials Plenary Session): Neratinib in HER2 or HER3 mutant solid tumors: SUMMIT, a global, multi-histology, open-label, phase 2 ‘basket’ study.
D. Hyman et al, Memorial Sloan Kettering Cancer Center.

April 2, 2017, 3:00 – 5:00 p.m. EDT – Abstract CT011 (Oral, Minisymposium): Circulating tumor DNA (ctDNA) sequencing for HER2 mutation (HER2mut) screening and response monitoring to neratinib in metastatic breast cancer (MBC).
C. Ma et al, Washington University School of Medicine.

April 2, 2017, 3:00 – 5:00 p.m. EDT – Abstract CT013 (Oral, Minisymposium): NSABP FB-10: Phase Ib dose-escalation trial evaluating trastuzumab emtansine (T-DM1) with neratinib (N) in women with metastatic HER2+ breast cancer (MBC).
J. Abraham et al, NSABP.

April 3, 2017, 10:30 a.m. – 12:45 p.m. EDT – Abstract LB103 (Oral, Major Symposium): Landscape of Somatic ERBB2 Mutations – Findings from AACR (Free AACR Whitepaper) GENIE and Comparison to Ongoing ERBB2 Mutant Basket Study.
A. Schram et al, Memorial Sloan Kettering Cancer Center.

April 4, 2017, 1:00 – 5:00 p.m. EDT – Abstract CT128 (Poster): Effects of adding budesonide or colestipol to loperamide prophylaxis on neratinib-associated diarrhea in patients (pts) with HER2+ early-stage breast cancer (eBC): the CONTROL trial.
E. Ibrahim et al, Beaver Medical Group LP.

On March 1, 2017 NewLink Genetics Corporation (NASDAQ:NLNK), a biopharmaceutical company focused on bringing novel immuno-oncology therapies to patients with cancer, reported that two abstracts on the company’s indoximod program will be presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) 2017 Annual Meeting in Washington, D.C (Press release, NewLink Genetics, MAR 1, 2017, View Source [SID1234517927]).

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Clinical Trials Plenary Session: Abstract CT117 – Interim analysis of the Phase 2 clinical trial of the IDO pathway inhibitor indoximod in combination with pembrolizumab for patients with advanced melanoma, to be presented during the Novel Immuno-Oncology Agent Clinical Trials session, Tuesday April 4, 2017 10:30 a.m. – 12:45 p.m. ET.

Poster Session: Abstract 4076 – A novel prodrug of indoximod with enhanced pharmacokinetic properties, to be presented during poster session PO.ET05.01 – Mechanistic Understanding of Novel Anticancer Therapies, April 4, 2017 1 p.m. – 5 p.m. ET .
The complete text of Clinical Plenary Session abstracts that have not been selected for the press program will be posted online at 4:30 p.m. ET on Friday, March 31. The text of clinical trials abstracts that have been selected for inclusion in the press program will not be posted online until the date and time of presentation.

"We are honored to have been selected by the AACR (Free AACR Whitepaper) review committee for the Clinical Trials Plenary Session at the upcoming AACR (Free AACR Whitepaper) meeting," said Nicholas Vahanian, M.D., President and Chief Medical Officer. "We look forward to sharing the data from both of these abstracts."