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FibroGen Reports Second Quarter 2017 Financial Results

On August 7, 2017 FibroGen, Inc. (NASDAQ:FGEN), a science-based biopharmaceutical company, reported financial results for the second quarter of 2017 and announced positive topline results of the company’s Phase 2 randomized, double-blind, placebo-controlled study and two combination sub-studies of pamrevlumab in idiopathic pulmonary fibrosis (IPF) (Press release, FibroGen, AUG 7, 2017, View Source [SID1234520068]). Pamrevlumab is a proprietary anti-connective tissue growth factor (CTGF) antibody being evaluated in fibrotic disease and cancer.

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"We are very encouraged by the topline IPF Phase 2 clinical study results that we announced today, in which pamrevlumab-treated patients had a significantly lower rate of decline in lung function, as compared to the placebo-treated patients. In addition, pamrevlumab continued to be well tolerated as a monotherapy in this IPF study, and was well tolerated in combination with pirfenidone and nintedanib," said Thomas B. Neff, FibroGen’s Chief Executive Officer. "We believe that the promising outcomes of these studies enable us to advance pamrevlumab into Phase 3 clinical development."

Recent Developments
Pamrevlumab for Idiopathic Pulmonary Fibrosis (IPF)

Reported positive topline Phase 2 clinical results from a double-blind, placebo-controlled study, and two double-blind, active-controlled combination sub-studies
Pamrevlumab for Pancreatic Cancer

Orphan Drug Designation status was granted by the U.S. Food and Drug Administration (FDA)
Phase 2 clinical results are expected year-end 2017/first quarter 2018
Roxadustat for Anemia in Chronic Kidney Disease (CKD)

On track to submit the new drug application (NDA) to the FDA in 2018
The independent data safety monitoring board, which reviews the U.S. and European Phase 3 programs quarterly, recommended in August 2017 that all trials continue without modification to current protocols
U.S. Roxadustat for Anemia in Myelodysplastic Syndromes (MDS)

Phase 3 clinical trial is anticipated to start in the third quarter of 2017
China Roxadustat for Anemia in CKD

On track to submit the NDA for anemia associated with CKD in dialysis-dependent and non-dialysis patients to the China Food and Drug Administration in the third quarter of 2017
China Roxadustat for Anemia in MDS

Phase 2/3 clinical study is on schedule to initiate in the fourth quarter of 2017
Corporate and Financial Highlights

Net loss per basic and diluted share for the quarter ended June 30, 2017 was $0.48, as compared to a net income per diluted share of $0.35 a year ago
At June 30, 2017, FibroGen had $414.7 million of cash, restricted time deposits, cash equivalents, investments, and receivables
Closed an equity financing in April 2017 that generated $115.1 million in net proceeds

About Pamrevlumab
Pamrevlumab is a proprietary therapeutic antibody developed by FibroGen to inhibit the activity of connective tissue growth factor (CTGF), a common factor in chronic fibrotic and proliferative disorders characterized by persistent and excessive scarring that can lead to organ dysfunction and failure. FibroGen is currently conducting clinical studies of pamrevlumab in idiopathic pulmonary fibrosis (IPF), pancreatic cancer, and Duchenne muscular dystrophy (DMD). In desmoplastic or fibrotic cancers, such as pancreatic cancer, CTGF promotes abnormal proliferation of stromal cells and tumor cells. For information about pamrevlumab studies currently recruiting patients, please visit www.clinicaltrials.gov.

About Roxadustat
Roxadustat is a first-in-class, orally administered small molecule currently in global Phase 3 clinical development as a therapy for anemia associated with chronic kidney disease (CKD). Roxadustat is a hypoxia-inducible factor prolyl hydroxylase inhibitor (HIF-PHI) that promotes erythropoiesis through increasing endogenous erythropoietin, improving iron regulation, and reducing hepcidin. Administration of roxadustat has been shown to induce coordinated erythropoiesis – increasing red blood cell count while maintaining plasma erythropoietin levels within or near normal physiologic range in multiple subpopulations of CKD patients – including in the presence of inflammation and without a need for supplemental intravenous iron.

Roxadustat is currently advancing through Phase 3 clinical trials worldwide, supported by extensive Phase 2 clinical data demonstrating correction and maintenance of hemoglobin levels in multiple subpopulations of CKD anemia patients. To date, roxadustat has been evaluated in Phase 1 and Phase 2 studies involving more than 1,400 subjects. Globally, a total of 15 studies are currently underway involving a total of more than 11,000 patients. Of these, 15 are Phase 3 pivotal studies comprising 10,400 patients, and are currently being conducted to support independent regulatory approvals of roxadustat in both non-dialysis and dialysis CKD patients in the U.S., Europe, Japan, and China. Later this year, roxadustat will also enter a Phase 3 clinical trial in the U.S., and a Phase 2/3 trial in China, for the treatment of anemia in myelodysplastic syndromes (MDS). For information about roxadustat studies currently recruiting patients, please visit www.clinicaltrials.gov.

Celyad announces new agreements with Celdara Medical and Dartmouth College

On August 7, 2017 Celyad (Euronext Brussels and Paris, and NASDAQ:CYAD), a pioneer in the discovery and development of CAR-T cell therapies, and its fully-owned subsidiary OnCyte LLC, reported revised terms to their agreements with Celdara Medical LLC and Dartmouth College (Filing, 6-K, Celyad, AUG 7, 2017, View Source [SID1234520066]).

Following encouraging initial results of the THINK trial, which have led to increased confidence in the long-term potential of Celyad’s CAR-T assets, Celyad has amended its existing agreements with Celdara Medical, LLC and Dartmouth College. Under the amended agreements Celyad will receive an increased share of future revenues generated by these assets, including revenues from its sublicensees. In return, Celyad will pay Celdara Medical LLC and Dartmouth College an upfront payment of $12.5 million (€10.6 million) and $12.5 million worth of Celyad shares at a share price of €32.35 corresponding to a 14% premium versus last trading day.

Christian Homsy, CEO of Celyad, commented: "With our increased confidence in the clinical opportunity of our lead product candidate CYAD-01 and the significant potential value creation opportunities of our allogeneic IP patents, we have decided to shift some of the value of the original deal upfront, in order to increase our share of potential future revenues from sublicenses. We believe these revised agreements provide incremental value to Celyad’s shareholders while the increased ownership of Celdara Medical in Celyad signals a clear and long-term commitment to our development of promising CAR-T cell therapies."

Celyad obtained access to its CAR-T NKR cell drug product candidates and related technology, including technology licensed from Dartmouth College, in January 2015, through its acquisition of OnCyte, LLC from Celdara Medical, LLC, a privately-held U.S. biotechnology company. This portfolio included three autologous CAR-T cell therapy products and an allogeneic T cell platform. Since the acquisition, Celyad has focused on further developing the portfolio and is currently in pre-clinical or clinical phase for a number of product candidates.

Christian Homsy, CEO of Celyad, further commented: "The upfront payment related to our deal with Novartis covers partially the cash portion of our renewed agreements with Celdara Medical LLC and Dartmouth College. Therefore, our ability to finance all of our operations remain unaltered. With our current cash position, we continue to have the means to fund our operating expenses and capital expenditure requirements through H1 2019."

Through its activities and expertise, Celyad is gaining international recognition for the discovery and development of CAR-T cell therapies and has established partnerships with industrial and academic world-class players such as Novartis (Switzerland), ONO Pharmaceutical (Japan), Moffitt Cancer Center (United States) and Institut Curie (France).

Kite Announces Initiation of Axicabtagene Ciloleucel CAR-T Clinical Program in the European Union

On August 7, 2017 Kite Pharma, Inc., (Nasdaq:KITE), a leading cell therapy company, reported that patients in the European Union (EU) are now being treated with its lead investigational candidate, axicabtagene ciloleucel, in the safety expansion cohort of ZUMA-1 (ClinicalTrials.gov, NCT: 02348216) (Press release, Kite Pharma, AUG 7, 2017, View Source [SID1234520063]). Kite is currently enrolling adult patients with relapsed/refractory diffuse large B-cell lymphoma (DLBCL), primary mediastinal B-cell lymphoma (PMBCL), and transformed follicular lymphoma (TFL) at multiple EU medical centers. Kite filed a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for axicabtagene ciloleucel in July 2017, the first CAR-T application in Europe.

"This important milestone underscores our commitment to providing a potentially curative therapy to patients with serious blood cancers worldwide," said David Chang, M.D., Ph.D., Executive Vice President of Research and Development and Chief Medical Officer of Kite. "Our CAR-T expertise established in the United States will be key as we expand our global footprint this year."

The first patient treated in the safety expansion cohort was at the Academic Medical Center (AMC) in Amsterdam by Professor Dr. Marie José Kersten. Additional patients are expected to be treated in multiple clinical sites across Europe in 2017.

"We are encouraged by the promising results observed in ZUMA-1 in the United States and are excited to be one of the first medical centers to bring this novel treatment modality to the EU," said Professor Dr. Marie José Kersten, Principal Investigator and Head of the Department of Hematology of the AMC in Amsterdam. "As a lymphoma specialist, I am gratified that we can now offer this potentially transformative therapy to patients with refractory, aggressive NHL who previously had no other therapeutic options."

Kite has been granted access to Priority Medicines (PRIME) regulatory support in the EU for treatment of refractory DLBCL. Access to the PRIME initiative is granted by the EMA to support the development and accelerate the review of new therapies to treat patients with a high unmet need.

About axicabtagene ciloleucel

Kite’s lead product candidate, axicabtagene ciloleucel, is an investigational therapy in which a patient’s T cells are engineered to express a chimeric antigen receptor (CAR) to target the antigen CD19, a protein expressed on the cell surface of B-cell lymphomas and leukemias, and redirect the T cells to kill cancer cells. Axicabtagene ciloleucel is currently under review by the U.S. Food and Drug Administration (FDA) for aggressive non-Hodgkin lymphoma and was granted Breakthrough Therapy Designation status for diffuse large B-cell lymphoma (DLBCL), transformed follicular lymphoma (TFL), and primary mediastinal B-cell lymphoma (PMBCL).

BioCryst Reports Second Quarter 2017 Financial Results

On August 7, 2017 BioCryst Pharmaceuticals, Inc. (NASDAQ:BCRX) reported financial results for the second quarter ended June 30, 2017 (Press release, BioCryst Pharmaceuticalsa, AUG 7, 2017, View Source [SID1234520059]).

"We are excited by the positive results previously reported in Parts 1 and 2 of the APeX-1 clinical trial that indicate we have an active oral drug, and look forward to completing the trial and reporting complete trial results in the third quarter of this year," said Jon P. Stonehouse, President & Chief Executive Officer. "These trial results should give us additional information to determine what doses we propose to regulatory authorities later this year for the Phase 3 program, with the goal of starting a pivotal trial early next year."

Second Quarter Financial Results

For the three months ended June 30, 2017, revenues decreased to $3.1 million from $4.8 million in the second quarter of 2016. The decrease in revenue was primarily due to a decrease in collaboration revenue under U.S. Government development contracts.

Research and Development (R&D) expenses for the second quarter of 2017 increased to $15.8 million from $14.2 million in the second quarter of 2016, primarily due to increased spending on the Company’s hereditary angioedema (HAE) portfolio.

General and administrative (G&A) expenses for the second quarter of 2017 of $2.8 million were in line with $2.7 million of G&A expense in the second quarter of 2016.

Interest expense was $2.1 million in the second quarter of 2017 as compared to $1.4 million in the second quarter of 2016, an increase related primarily to the September 2016 closing of a $23 million senior credit facility. Also, a $400,000 mark-to-market loss on the Company’s foreign currency hedge was recognized in the second quarter of 2017, as compared to a $3.7 million mark-to-market loss in the second quarter of 2016. These losses result from periodic changes in the U.S. dollar/Japanese yen exchange rate. During the second quarters of 2017 and 2016, we also realized currency gains of $921,000 and $811,000, respectively, from the exercise of a U.S. Dollar/Japanese yen currency option within our foreign currency hedge.

The net loss for the second quarter of 2017 was $16.9 million, or $0.21 per share, compared to a net loss of $16.3 million, or $0.22 per share, for the second quarter 2016.

Cash, cash equivalents and investments totaled $95.6 million at June 30, 2017, and reflect an increase from $65.1 million at December 31, 2016. Net operating cash use for the second quarter of 2017 was $12.2 million, and the first six months of 2017 was $21.0 million, which excludes the impact of $47.8 million of net proceeds from our March 2017 public offering.

Year to Date Financial Results

For the six months ended June 30, 2017, revenues increased to $12.5 million from $9.6 million in the first half of 2016. The increase in revenue was primarily due to a $4.3 million increase in royalty revenue from Shionogi & Co. Ltd., Green Cross Corporation and Seqirus, and a $2.0 million milestone payment from Seqirus associated with the Canadian regulatory approval of RAPIVAB. The increase in royalty revenue was largely the result of continued Japanese Government stockpiling of RAPIACTA. Future government stockpiling orders are difficult to predict, as they are subject to the relevant appropriation and stockpiling processes. These revenue increases were partially offset by a decrease in collaboration revenue under U.S. Government development contracts.

R&D expenses decreased to $32.5 million from $34.7 million in the first half of 2016, primarily due to lower development costs for the HAE portfolio of product candidates and, to a lesser extent, a decrease in galidesivir expenses under U.S. Government development contracts.

G&A expenses for the first half of 2017 of $5.9 million were in line with $5.9 million of G&A expense in the first half of 2016.

Interest expense was $4.2 million in the first half of 2017 as compared to $2.9 million in the first half of 2016, an increase related primarily to the September 2016 closing of a $23 million senior credit facility. A $1.9 million mark-to-market loss on the Company’s foreign currency hedge was recognized in the first half of 2017, as compared to a $6.4 million mark-to-market loss in the first half of 2016. These losses result from periodic changes in the U.S. dollar/Japanese yen exchange rate. During 2017 and 2016, we also realized currency gains of $921,000 and $811,000, respectively, from the exercise of a U.S. Dollar/Japanese yen currency option within our foreign currency hedge.

The net loss for the first half of 2017 was $31.1 million, or $0.40 per share, compared to a net loss of $39.1 million, or $0.53 per share, for the first half 2016.

Clinical Development Update & Outlook

On May 25, BioCryst announced positive results from a second interim analysis of its Phase 2 APeX-1 clinical trial in HAE. This second interim analysis of pooled data from Parts 1 and 2 evaluated doses of BCX7353 125 mg (n=7), 250 mg (n=6) and 350 mg (n=18) QD versus placebo (n=20) for 28 days. The pre-specified per-protocol (PP) interim analysis included data on a total of 44 subjects with confirmed Type 1 or Type 2 HAE completing 28 days of treatment. The percentage reductions by treatment group in the mean rate of independently-adjudicated angioedema attacks for the pre-defined effective dosing period (weeks 2 through 4) in BCX7353 treated subjects were: 125 mg QD, 73% (p=0.002); 250 mg QD, 37% (p=0.128) and 350 mg QD, 58% (p=0.001) compared to placebo. In the intent-to-treat (ITT) population, corresponding reductions by treatment group were: 125 mg QD, 73% (p=0.004); 250 mg QD, 44% (p=0.090) and 350 mg QD, 45% (p=0.014) compared to placebo.

Oral BCX7353 once-daily for 28 days was generally safe and well tolerated in subjects with HAE. There were no serious AEs and no severe AEs. Three subjects in the BCX7353 350 mg treatment arm discontinued study drug before day 28. The most common treatment-emergent adverse events were the common cold and diarrhea. The gastrointestinal AEs previously observed in the 350 mg arm were not seen at the 125 mg dose. Additionally, no significant laboratory abnormalities were observed in the two lower dose groups.

On August 2, BioCryst announced the dosing of the first subject into ZENITH-1, a clinical trial studying up to three dosage strengths of a liquid formulation of BCX7353 given as a single oral dose for the acute treatment of angioedema attacks in patients with HAE.

On June 5, BioCryst announced that the U.S. Food and Drug Administration (FDA) has accepted for review the supplemental New Drug Application (sNDA) for a pediatric indication of RAPIVAB (peramivir injection), which was submitted in March 2017. The sNDA has been classified by the FDA as a priority review and has a Prescription Drug User Fee Act (PDUFA) goal date for a decision by the end of September 2017.

After discussions with the FDA, NIAID and BARDA, we have delayed the initiation of the galidesivir IV Phase 1 clinical trial. Based upon ongoing conversations, we expect the next step in galidesivir’s development will be to conduct an additional nonclinical efficacy study in a delayed treatment setting in Ebola disease before finalizing the Phase 1 clinical trial protocol design.

On May 30, BioCryst announced the appointment of Robert A. Ingram as Chairman of its Board of Directors.
Financial Outlook for 2017

Based upon development plans and our awarded government contracts, BioCryst continues to expect its 2017 net operating cash use to be in the range of $30 to $50 million, and its 2017 operating expenses to be in the range of $53 to $73 million. Our operating expense range excludes equity-based compensation expense due to the difficulty in reliably projecting this expense, as it is impacted by the volatility and price of the Company’s stock, as well as by the vesting of the Company’s outstanding performance-based stock options.

Conference Call and Webcast

BioCryst’s leadership team will host a conference call and webcast Monday, August 7, 2017 at 11:00 a.m. Eastern Time to discuss these financial results and recent corporate developments. To participate in the conference call, please dial 1-877-303-8027 (United States) or 1-760-536-5165 (International). No passcode is needed for the call. The webcast can be accessed by logging onto www.BioCryst.com. Please connect to the website at least 15 minutes prior to the start of the conference call to ensure adequate time for any software download that may be necessary.

About BCX7353

Discovered by BioCryst, BCX7353 is a novel, oral, once-daily, selective inhibitor of plasma kallikrein currently in development for the prevention and treatment of angioedema attacks in patients diagnosed with HAE. BCX7353 has been generally safe and well tolerated in the ongoing Phase 2 APeX-1 clinical trial for prophylaxis and in clinical pharmacology studies in healthy volunteers.

Data Monitoring Committee Completes Mid-Study Review of Celsion’s Phase III ThermoDoxÂ® OPTIMA Study in Primary Liver Cancer

On August 7, 2017 Celsion Corporation (NASDAQ:CLSN), an oncology drug development company, reported that the independent Data Monitoring Committee (DMC) for the Company’s 550 patient, multinational, double-blind, placebo-controlled, pivotal Phase III clinical study of ThermoDox in combination with radiofrequency ablation (RFA) for primary liver cancer (the OPTIMA study), has completed a planned interim analysis of the first 50% of patients randomized in the trial as of April 2017 for safety and efficacy and unanimously recommended that the study continue according to protocol to its final data readout based on the risk to benefit analysis by the Committee (Press release, Celsion, AUG 7, 2017, View Source [SID1234520058]). The OPTIMA study to date has accumulated data within acceptable safety parameters.

The DMC is comprised of an independent group of medical and scientific experts and is responsible for reviewing and evaluating patient safety and efficacy data for the Company’s Phase III OPTIMA Study. The DMC reviews study data at regular intervals in order to ensure the safety of all patients enrolled in the trial and to monitor the quality and overall conduct of the trial including each site’s compliance with the minimum RFA heating time of 45 minutes specified in the study protocol. The Company also announced that enrollment in the OPTIMA Study is now over 60% of the 550 patients necessary to ensure that its primary end point, overall survival, can be achieved with statistical significance.
"Following independent confirmation of our hypothesis by the National Institutes of Health (NIH) in November 2016 that ThermoDox in combination with optimized RFA can be a treatment with curative intent for primary liver cancer, we are very pleased that the DMC has recommended continuation of the OPTIMA Study without modification. Based on their review of all the available study data from 275 patients enrolled as of April 2017, the DMC has concluded that ThermoDox is safe for newly diagnosed, intermediate stage patients and that the study is being conducted according to the highest of clinical research standards," said Nicholas Borys, MD, Celsion’s senior vice president and chief medical officer. "A key component of the OPTIMA Study protocol is the investigators’ adherence to the recommended RFA heating time for tumors greater than 3 cm. We are pleased to report that there has been a greater than 99% compliance rate with the study protocol."
On November 29, 2016, the Company announced results from an independent retrospective analysis conducted by the NIH on the intent-to-treat population of the 701 patient HEAT Study of ThermoDox plus optimized RFA for the treatment of primary liver cancer. The NIH analysis, which sought to evaluate the correlation between RFA burn time per tumor volume (min/ml) and clinical outcome, concluded that increased RFA "burn time" per tumor volume significantly improved overall survival (OS) in patients with solitary lesions treated with RFA + ThermoDox compared to patients treated with RFA alone. The NIH analysis included 437 patients with a single lesion from the Company’s HEAT Study, the same patient population being treated in the Company’s ongoing Phase III OPTIMA study.
The NIH findings are consistent with Celsion’s own analysis of the HEAT Study data, which demonstrated that over a 3.5 year period, there was a statistically significant survival benefit of approximately 2 years for patients treated with ThermoDox plus optimized RFA over the optimized RFA only group.
"We are very pleased that the DMC has unanimously recommended continuation of the OPTIMA study based on their review of all available clinical data, both safety and efficacy, in over 275 patients," stated Mr. Michael H. Tardugno, Celsion’s chairman, president and chief executive officer. "The DMC’s affirmative review is further evidence of ThermoDox’s potential to provide a new and important first line therapeutic option for patients with primary liver cancer."
Regulatory Strategy for ThermoDox. ThermoDox has received FDA Fast Track Designation and in the prior HEAT Study had been designated as a Priority Trial for primary liver cancer by the National Institutes of Health. ThermoDox has been granted orphan drug designation for primary liver cancer in both the U.S. and Europe. Based on prior discussions and subject to a successful trial, Celsion fully expects that the U.S. FDA and European EMA enrollment objectives will be met, and that the OPTIMA Study will also enrolled a sufficient number of patients to support registrational filings in China, South Korea, Taiwan and Vietnam, four other large and important markets for ThermoDox.
In December 2016, the Company met with the China Food and Drug Administration (CFDA) to discuss the ongoing Phase III OPTIMA program and regulatory pathway for ThermoDox in China. During the meeting, Celsion presented the final overall survival data from the Chinese patient cohort from the prior HEAT study, which demonstrated a survival benefit in patients treated with ThermoDox plus optimized RFA versus optimized RFA alone. The CFDA informed Celsion that if the ongoing Phase III OPTIMA trial is successful, the trial could serve as the basis for a direct regulatory filing in China without the need to file for prior approval in the U.S. or European Union which is currently required for foreign company application. This would allow the Company to accelerate its plans for a regulatory filing in China and, if approved, provide for a significantly earlier launch date in China than originally expected.
The OPTIMA study’s design and statistical plan incorporates two pre-planned interim efficacy analysis by the DMC (after patient enrollment is complete) with the intent of evaluating safety, efficacy and futility to determine if there is overwhelming evidence of clinical benefit or a low probability of treatment success to continue, modify or terminate the study. The results from the first interim efficacy analysis are expected to be made public following DMC review in Q1 of 2019.
About the OPTIMA Study
The Phase III OPTIMA Study is expected to enroll up to 550 patients in up to 70 clinical sites in the United States, Europe, China and Asia Pacific, and will evaluate ThermoDox in combination with optimized RFA, which will be standardized to a minimum of 45 minutes across all investigators and clinical sites for treating lesions three to seven centimeters, versus optimized RFA alone. The primary endpoint for the trial is Overall Survival, which is supported by post-hoc analysis of data from the Company’s 701 patient HEAT Study, where optimized RFA has demonstrated the potential to significantly improve survival when combined with ThermoDox. The statistical plan calls for two interim efficacy analyses by an independent Data Monitoring Committee.