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."

On March 2, 2017 Innate Pharma SA (the "Company" – Euronext Paris: FR0010331421 – IPH) reported that data on monalizumab will be presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting, April 1 – 5, 2017, in Washington, D.C (Press release, Innate Pharma, MAR 1, 2017, View Source [SID1234517926]). Abstracts are available on the AACR (Free AACR Whitepaper) website. Monalizumab is Innate Pharma’s investigational first-in-class anti-NKG2A antibody partnered with AstraZeneca.

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These data support the rationale for the development of monalizumab:

Preclinical data will be presented in a mini-symposium and show NKG2A expression on tumor-infiltrating CD8+ T cells in patients with head and neck cancer as well as synergy between treatment with a HPV vaccine and NKG2A blockade in a mouse tumor model;
During a poster session, safety data from the dose-escalation part of a Phase Ib/II study evaluating monalizumab in combination with cetuximab in patients with recurrent or metastatic squamous cell carcinoma of the head and neck will be presented. In this study, monalizumab plus cetuximab were well tolerated with no additional safety concerns compared to monalizumab or cetuximab alone.
Monalizumab is currently being tested in five Phase I and I/II clinical trials in various cancer as a single agent and in combination with other therapies.



Presentation and poster details:

NKG2A checkpoint receptor expression on tumor-infiltrating CD8+ T cells restrains efficacy of immunotherapy
Abstract Number: 2999
Session Type: Mini-symposium, webcast
Session Title: Innate Immune Mechanisms in Cancer Treatment
Session Date and Time: Monday April 3, 2017 4:35 – 4:50 PM EST
Location: Washington Convention Center, Room 152, Level 1
Presented by Thorbald van Hall, Department of Clinical Oncology, Leiden University Medical Centre, The Netherlands
Safety of the first-in-class anti-NKG2A monoclonal antibody monalizumab in combination with cetuximab: a phase Ib/II study in recurrent or metastatic squamous cell carcinoma of the head and neck (R/M SCCHN)
Abstract Number: 5666/20
Session Type: Poster Session
Session Title: Innate Immunity to Generate Adaptive Immunity
Session Date and Time: Wednesday April 5, 2017 8:00 AM – 12:00 PM EST
Location: Washington Convention Center, Halls A-C, Poster Section 28
Presented by Roger B. Cohen M.D., Abramson Cancer Center, Philadelphia, USA