On May 11, 2016 CytRx Corporation (NASDAQ: CYTR), a biopharmaceutical research and development company specializing in oncology, reported financial results for the three months ended March 31, 2016, and provided an overview of recent accomplishments and upcoming milestones for its research and development programs (Press release, CytRx, MAY 11, 2016, View Source;p=RssLanding&cat=news&id=2167453 [SID:1234512256]).

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"So far, 2016 has been a very productive year for CytRx," said Steven A. Kriegsman, Chairman and CEO of CytRx. "On the clinical front, we reached the 191 progression events in our global, pivotal Phase 3 trial with aldoxorubicin in second-line soft tissue sarcoma to trigger the data verification and analysis. We look forward to announcing top-line results at the end of June 2016. We announced that we will present updates on three aldoxorubicin clinical trials at the ASCO (Free ASCO Whitepaper) Annual Meeting in June 2016. Additionally, our team recently presented data at the American Association of Cancer Research Annual Meeting on our LADRTM technology platform and on DK049, the first drug to emerge from that platform."

Mr. Kriegsman continued: "On the corporate side, we added commercial oncology expertise with the addition of Ms. Olivia Ware to our executive management team. We also raised an additional $25 million in non-dilutive capital to fund clinical and pre-commercial activities for aldoxorubicin."

First Quarter 2016 and Recent Highlights

Bolstered the Management Team. In January 2016, CytRx hired Ms. Olivia Ware as Chief Commercial Officer. Ms. Ware brings more than 20 years of biotechnology and pharmaceutical experience, including thirteen years at Genentech where she was responsible for key aspects of the launches of the oncology drugs Rituxan, Herceptin and Avastin. While at Genentech, Ms. Ware was a Senior Director of Oncology and was responsible for the initial commercial launch of Avastin in the U.S., which reached $1 billion in sales during its first year on the market. Prior to this, she was head of Herceptin marketing and held a number of other positions in the commercial organization. Later, Ms. Ware was a Senior Director in the Product Portfolio Management Group, managing approximately 20 senior leaders responsible for building and leading the cross-functional drug development teams that developed and implemented strategic plans and guided the drug development processes for all oncology products at Genentech.

Strengthened the Balance Sheet. In February 2016, CytRx entered into a long-term loan for up to $40 million with Hercules Technology Growth Capital. The initial tranche of $25 million was received, and CytRx can access an additional $15 million, subject to certain research and development milestones.

Reached the Target Number of Events in the Global Pivotal Phase 3 Trial. In April 2016, CytRx achieved the target number of progression events in the aldoxorubicin global, pivotal Phase 3 trial in patients with second-line soft tissue sarcomas. Our contract research organization started the collection and verification of the trial data from all 433 patients enrolled at 79 sites around the globe. CytRx expects to report top-line results following the analysis of the data in June 2016.

Presented Data on LADRTM Technology Platform and DK049. In April 2016, CytRx presented two posters at the American Association of Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting detailing both our Linker Activated Drug Release (LADRTM) Platform and DK049, an albumin-binding derivative of the widely used chemotherapy agent gemcitabine combined with our LADRTM technology. DK049 was nominated for clinical development in 2015.

Upcoming Milestones

Present updated aldoxorubicin clinical trial results at the ASCO (Free ASCO Whitepaper) Annual Meeting in June 2016 from the ongoing Phase 2 clinical trial in patients with unresectable glioblastoma, the Phase 2 clinical trial in Kaposi’s sarcoma and from the Phase 1b combination trial with gemcitabine in patients with advanced cancers
Announce top-line results from the global pivotal Phase 3 clinical trial with aldoxorubicin as a treatment for patients with second-line soft tissue sarcomas
Present aldoxorubicin pivotal Phase 3 clinical trial results at a major medical meeting
Complete enrollment in the global Phase 2b clinical trial evaluating aldoxorubicin in patients with second-line small cell lung cancers
First Quarter 2016 Financial Results

CytRx reported cash, cash equivalents and short term investments of $68.2 million as of March 31, 2016.

Net loss for the quarter ended March 31, 2016 was $12.6 million, or $0.19 per share, compared with a net loss of $17.5 million, or $0.31 per share, for the quarter ended March 31, 2015. During the first quarter of 2016, CytRx recognized a non-cash loss on warrant derivative liability of $0.2 million, compared to a non-cash loss of $1.9 million for the three-month period ended March 31, 2015. The Company did not recognize revenues for the first quarters of 2016 or 2015.

Research and development (R&D) expenses were $8.2 million for the first quarter of 2016, and included development expenses of $5.8 million for the aldoxorubicin program. R&D expenses were $12.6 million for the first quarter of 2015.

General and administrative (G&A) expenses were $4.0 million for the first quarter of 2016, compared to $3.1 million for the first quarter of 2015.

About Soft Tissue Sarcoma
Soft tissue sarcoma is a cancer occurring in muscle, fat, blood vessels, tendons, fibrous tissues and connective tissue, and can arise anywhere in the body at any age. According to the American Cancer Society, there are approximately 50 types of soft tissue sarcomas. In 2013 more than 11,400 new cases were diagnosed in the U.S. and approximately 4,400 Americans died from this disease. In addition, approximately 40,000 new cases and 13,000 deaths in the U.S. and Europe are part of a growing underserved market.

About Small Cell Lung Cancer
An estimated 1.6 million new cases of lung cancer are diagnosed worldwide each year. In the Western world, approximately 13-15% of cases are SCLC, a deadly form of lung cancer associated with tobacco use. The five year survival rate is less than 7%, in part because an estimated 70% of patients have extensive disease at diagnosis. According to the National Cancer Institute, more than 30,000 new cases will be diagnosed in the USA in 2014. The estimated 2014 SCLC incidences for Europe and Asia are over 58,000 and 136,000, respectively.

About Glioblastoma Multiforme
Glioblastoma is the most common and most malignant primary brain tumor in adults and afflicts more than 12,000 new patients in the U.S. annually. The median survival after diagnosis is approximately 14 months, despite patients subsequently receiving surgical resection, radiotherapy and chemotherapy. Limited efficacy of chemotherapeutic agents has been attributed to several contributing factors including insufficient drug delivery to the tumor site through the blood:brain barrier.

About Aldoxorubicin
The widely used chemotherapeutic agent doxorubicin is delivered systemically and is highly toxic, which limits its dose to a level below its maximum therapeutic benefit. Doxorubicin also is associated with many side effects, especially the potential for damage to heart muscle at cumulative doses greater than 450 mg/m2. Aldoxorubicin combines doxorubicin with a novel single-molecule linker that binds directly and specifically to circulating albumin, the most plentiful protein in the bloodstream. Protein-hungry tumors concentrate albumin, thus increasing the delivery of the linker molecule with the attached doxorubicin to tumor sites. In the acidic environment of the tumor, but not the neutral environment of healthy tissues, doxorubicin is released. This allows for greater doses (3 ½ to 4 times) of doxorubicin to be administered while reducing its toxic side effects. In studies thus far there has been no evidence of clinically significant effects of aldoxorubicin on heart muscle, even at cumulative doses of drug well in excess of 2,000 mg/m2.

About DK049
DK049 is a novel LADRTM-enhanced derivative of the commonly used chemotherapeutic agent gemcitabine. Like aldoxorubicin, DK049 binds to circulating albumin for transport to the tumor. It incorporates a novel dual-releasing linker that is cleaved in an acidic environment and requires a specific enzyme for prolonged release of the active drug at the tumor site. DK049 appears to overcome the major deactivation and resistance mechanisms that limit the effectiveness of gemcitabine. Preclinical studies in animal models of non-small cell lung, pancreatic and ovarian cancers showed superior anti-tumor activity compared to gemcitabine. CytRx recently presented preclinical data on DK049 at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting in April 2016.

On May 11, 2016 Heat Biologics, Inc. ("Heat") (Nasdaq:HTBX), an immuno-oncology company developing novel therapies that activate a patient’s immune system against cancer, reported its financial results for the first quarter ended March 31, 2016 (Press release, Heat Biologics, MAY 11, 2016, View Source [SID:1234512295]).

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"We are focused on achieving near-term milestones to rebuild shareholder value. Our primary goal is to advance HS-410 for the treatment of non-muscle invasive bladder cancer with one-year disease-free survival, immune response, safety and tolerability data expected in the fourth quarter of this year," said Jeff Wolf, Heat’s Founder and CEO. "In addition, we are looking forward to presenting new data at the upcoming ASCO (Free ASCO Whitepaper) Annual Meeting in June around our Phase 1b trial evaluating HS-110 in combination with an anti-PD-1 checkpoint inhibitor for the treatment of non-small cell lung cancer."

Recent Developments & First Quarter 2016 Corporate Highlights

In May, Heat had an abstract accepted for poster presentation at the ASCO (Free ASCO Whitepaper) Annual Meeting being held on June 3-7, 2016 in Chicago, IL. The poster is entitled "Broadening response rates to PD-1 therapy in advanced lung adenocarcinoma: Viagenpumatucel-L (HS-110) in combination with nivolumab in the ongoing DURGA trial" (Abstract #TPS9102). Abstracts will be made available on the ASCO (Free ASCO Whitepaper) website at www.asco.org in line with the conference’s embargo policy.

In April, Heat presented three posters at the AACR (Free AACR Whitepaper) Annual Meeting. In the poster entitled "Phase I/II Study of Patients with Non-Muscle Invasive Bladder Cancer (NMIBC) Treated with Vesigenurtacel-L (HS-410) with or without BCG," Heat reported that no additional recurrences had been reported to-date, with all patients now at least 18 months out from enrollment. In another poster, Heat reported initial preclinical results from its collaboration with OncoSec Medical Incorporated in which researchers concluded that combining Heat’s ComPACT vaccine with OncoSec’s intratumoral DNA electroporation delivery platform stimulated an expansion of neoantigen-specific CD8+ T cells, leading to a regression in both treated and untreated cancer lesions in two mouse studies. In the third poster, Heat reported positive preclinical data on its next generation ComPACT platform technology, which combines a T cell priming vaccine and a T cell co-stimulator in a single product.

In April, Heat implemented cost-saving measures and a focused corporate strategy to achieve data readout, with its current cash on-hand, anticipated in the fourth quarter of 2016 for its lead Phase 2 program evaluating HS-410 for the treatment of NMIBC. These cost-saving measures included a workforce reduction of approximately 22 percent of the company’s headcount.

In April, Heat appointed John Prendergast, Ph.D., to its Board of Directors.

In March, Heat closed a public offering of approximately $6.8 million in gross proceeds, which will primarily be used to complete its Phase 2 clinical trial evaluating HS-410 for the treatment of NMIBC. Remaining funds will be used to advance the current eight patients enrolled in our Phase 1b trial evaluating HS-110 in combination with nivolumab for the treatment of non-small cell lung cancer (NSCLC) through the reporting of topline data, as well as for licensing or acquisition of assets complementary to our existing programs and working capital and general corporate purposes.

In March, Heat presented additional preclinical data from its ComPACT platform technology at the Keystone Symposia on Cancer Vaccines. Data presented demonstrated that ComPACT secreting OX40L generated the most potent immune response among other ComPACT co-stimulator variations including TL1A, 4-1BBL and ICOSL, as well as compared to systemic delivery of OX40 agonist antibody and vaccine alone.

In February, Heat announced that it will no longer enroll new patients in its Phase 2 monotherapy trial arm evaluating HS-410 alone for the treatment of NMIBC following the resolution of the standard of care BCG shortage and discussions with the U.S. FDA. Heat anticipates reporting topline 6-month data from the 16 enrolled patients in the fourth quarter of 2016, contemporaneous with reporting data from Heat’s BCG combination cohorts.

In February, Heat announced that the U.S. FDA lifted the partial clinical hold on its HS-410 Phase 2 clinical trial and patient enrollment was resumed after less than one week; clinical timelines were materially unchanged. The partial clinical hold came after Heat concluded that the cell line on which HS-410 is based had been previously misidentified and immediately notified FDA of this conclusion. The FDA placed Heat’s HS-410 Phase 2 clinical trial on partial clinical hold while they reviewed updated documentation. The partial clinical hold did not relate to concerns regarding the safety or efficacy of HS-410.

In January, Heat reported three-month interim data from the unblinded, monotherapy arm in its Phase 2 trial evaluating HS-410 for the treatment of NMIBC. Images of the bladder taken from several patients treated with HS-410 alone showed changes that resemble T cell-rich structures that Heat has observed in biopsy samples, indicating that systemic administration with HS-410 leads to a localized immune response within the bladder that cannot be attributed to standard of care.

First Quarter 2016 Financial Highlights

Research and development expenses decreased to approximately $500,000 in the first quarter of 2016 compared to approximately $504,000 in the first quarter of 2015, a decrease of approximately $4,000. The decrease is attributable to reductions in patent, license and other professional fees offset by compensation costs associated with new hires, as well as supplies and facilities as we bring more capabilities in-house.

Clinical and regulatory expenses increased to approximately $3.2 million in the first quarter of 2016 compared to approximately $2.2 million in the first quarter of 2015, an increase of approximately $1.0 million. The increase is attributable to clinical trial execution expenses, personnel costs and expenses related to the production of our clinical trial material.

General and administrative expenses decreased to approximately $1.0 million in the first quarter of 2016 compared to approximately $1.3 million in the first quarter of 2015, a decrease of approximately $0.3 million. The decrease is attributable to non-cash stock compensation expense for non-employees associated with the company’s reduced shared price, as well as reduced professional services and third party expenses.

Net loss for the first quarter of 2016 was $4.7 million compared to a net loss of $4.0 million for the first quarter of 2015.

Cash, cash equivalents and short-term investments totaled approximately $11.8 million at March 31, 2016 compared to $11.6 million at December 31, 2015. This includes the $6.1 million in net proceeds raised during our March 2016 public offering.

On May 11, 2016 Kura Oncology, Inc., (NASDAQ:KURA) a clinical stage biopharmaceutical company, reported financial results for the first quarter ended March 31, 2016 (Press release, Kura Oncology, MAY 11, 2016, View Source;p=RssLanding&cat=news&id=2167626 [SID:1234512296]).

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"Kura has made important progress in advancing its pipeline of precision oncology drug candidates over the past quarter," said Troy Wilson, Ph.D., J.D., President and Chief Executive Officer of Kura Oncology. "We continue to enroll patients in our on-going Phase 2 clinical trials of tipifarnib in patients with HRAS mutant solid tumors and in relapsed or refractory peripheral T-cell lymphoma, and earlier in May 2016, we initiated our Phase 2 trial of tipifarnib in lower risk myelodysplastic syndromes (MDS)."

Dr. Wilson added: "In the second half or 2016, we are also planning to initiate a Phase 2 clinical trial with tipifarnib in patients with chronic myelomonocytic leukemia (CMML), a population for which prognosis is very poor, with a three-year survival rate estimated at less than 30 percent. Objective responses, including complete responses, have been previously observed with tipifarnib in CMML. Moreover, as part of the study, we plan to test a biomarker hypothesis, which may allow us to identify those patients most likely to experience durable responses. We believe our ongoing Phase 2 clinical trials and our additional planned trial in CMML provide us with multiple potential opportunities to position tipifarnib for registration-enabling Phase 3 studies in late 2017 or early 2018."

Upcoming Clinical and Preclinical Milestones for Kura Oncology Programs

IND submission for ERK inhibitor, KO-947, is anticipated in the second quarter of 2016 followed by initiation of a Phase 1 clinical trial, which is anticipated in the second half of 2016
Receipt of topline data from the Phase 2 clinical trial for tipifarnib in HRAS mutant solid tumors is anticipated in the second half of 2016
Initiation of a Phase 2 clinical trial for tipifarnib in patients with CMML is anticipated in the second half of 2016
Nomination of a development candidate for the menin-MLL program is anticipated in the second half of 2016
Financial Results for the First Quarter 2016

Cash, cash equivalents and short-term investments totaled $78.5 million as of March 31, 2016, compared with $85.7 million as of December 31, 2015. Management expects that existing cash, cash equivalents and short-term investments will be sufficient to fund current operations into 2018.
Research and development expenses for the first quarter of 2016 were $4.6 million, compared to $3.6 million for the first quarter of 2015.
General and administrative expenses for the first quarter of 2016 were $2.4 million, compared to $1.1 million for the first quarter of 2015.
Net loss for the first quarter of 2016 was $6.6 million, or $0.36 per share, compared to a net loss of $4.5 million, or $1.41 per share, for the first quarter of 2015.
Subsequent to the first quarter of 2016, Kura Oncology put in place a $20.0 million long-term debt financing agreement, of which $7.5 million has been drawn down. Use of proceeds is for development programs and general corporate purposes.

On May 6, 2016 La Jolla Pharmaceutical Company (NASDAQ: LJPC) (the Company or La Jolla), a leader in the development of innovative therapies intended to significantly improve outcomes in patients suffering from life-threatening diseases, reported first quarter 2016 financial results and highlighted recent corporate progress (Filing, Q1, La Jolla Pharmaceutical, 2016, MAY 11, 2016, View Source [SID:1234512493]).

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Recent Corporate Progress

• La Jolla’s ATHOS (Angiotensin II for the Treatment of High-Output Shock) 3 trial continued to enroll as planned through the first quarter of 2016. The ATHOS 3 trial is La Jolla’s multicenter, randomized, double-blind, placebo-controlled, Phase 3 clinical trial of LJPC-501, the Company’s proprietary formulation of angiotensin II, in catecholamine-resistant hypotension (CRH), which was initiated in 2015. The Company initiated the ATHOS 3 trial after reaching an agreement with the U.S. Food and Drug Administration (FDA) on a Special Protocol Assessment (SPA), in which the agreed-upon primary efficacy endpoint in ATHOS 3 is an increase in blood pressure. Results of ATHOS 3 are expected by the end of 2016.

• La Jolla reported interim results in January 2016 of its multicenter, open-label, dose-escalation Phase 1 clinical trial of LJPC-401, the Company’s novel formulation of hepcidin, in patients at risk for iron overload due to conditions such as hereditary hemochromatosis, beta thalassemia, sickle cell disease and myelodysplastic syndrome. These results suggested a dose-dependent reduction in serum iron following a single dose of LJPC-401. Complete results from this Phase 1 clinical trial, which was initiated in October 2015, are expected in the second half of 2016.
"The first quarter was a productive start to 2016 for La Jolla, highlighted by the continued enrollment of our ATHOS 3 trial and encouraging interim data from our Phase 1 clinical trial of LJPC-401," said George Tidmarsh, M.D., Ph.D., La Jolla’s President and Chief Executive Officer. "We look forward to continuing the advancement of each of our exciting programs during the rest of 2016, culminating with the results from the ATHOS 3 trial that are expected by the end of the year."
Results of Operations

As of March 31, 2016, La Jolla had $113.1 million in cash and cash equivalents, compared to $126.5 million as of December 31, 2015. The decrease in cash and cash equivalents was primarily due to net cash used for operating activities. Based on current operating plans and projections, La Jolla believes that its current cash and cash equivalents are sufficient to fund operations into 2018.

La Jolla’s net cash used for operating activities for the three months ended March 31, 2016 was $13.0 million, compared to net cash used for operating activities of $5.5 million for the same period in 2015. La Jolla’s net loss for the three months ended March 31, 2016 was $16.5 million, or $0.96 per share, compared to a net loss of $9.0 million, or $0.59 per share, for the same period in 2015. During the three months ended March 31, 2016, La Jolla recognized contract revenue of approximately $0.2 million, which was pursuant to a services agreement initiated in 2015 under which La Jolla provides research and development services to a related party. The net loss includes non-cash, share-based compensation expense of $3.7 million for the three months ended March 31, 2016, compared to $3.4 million for the same period in 2015.

The increases in net cash used for operating activities and net loss in 2016 as compared to 2015 were primarily due to increased development costs associated with the ATHOS 3 trial and the costs associated with the initiation of the Phase 1 clinical trial of LJPC-401 in iron overload. In addition, there were increases in personnel and related costs, which were mainly due to the hiring of additional personnel and increased facility costs to support the increased development activities.

On May 11, 2016 Onconova Therapeutics, Inc. (NASDAQ:ONTX), a Phase 3 clinical-stage biopharmaceutical company focused on discovering and developing novel products to treat cancer, reported a corporate update and reported financial results for the first quarter ended March 31, 2016 (Press release, Onconova, MAY 11, 2016, View Source [SID:1234512298]).

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"We recently reported two important advances in the development of rigosertib for the unmet medical needs of patients with myelodysplastic syndromes (MDS). Foremost is the elucidation of the novel mechanism of action of rigosertib targeting the RAS pathway, recently published in Cell, and the release of detailed clinical data from our ONTIME Phase 3 study in Lancet Oncology. These peer-reviewed papers provide important validation of the mechanistic rationale for rigosertib as a novel treatment option for patients with MDS," said Ramesh Kumar, Ph.D., President and CEO of Onconova. "We are encouraged by the continued progress in the global Phase 3 INSPIRE trial which is now enrolling patients in the U.S. and Europe."

Recent Business Highlights:

Progress in INSPIRE Pivotal Trial of Rigosertib in Higher-risk MDS (HR-MDS)

The global INSPIRE trial is now enrolling patients in the United States and Europe. As of May 3, 2016, 51 sites, including 13 in the U.S., are open and recruiting patients. This pivotal trial was initiated in 4Q2015.

Results from Onconova’s ONTIME trial were published in the top-tier, peer-reviewed journal, Lancet Oncology. The article, entitled, "Rigosertib versus best supportive care for patients with high-risk myelodysplastic syndromes after failure of hypomethylating drugs (ONTIME): a randomised, controlled, phase 3 trial," appeared in the March 8, 2016 online edition of the journal.
Rigosertib Mechanism of Action

The unique RAS-targeted mechanism of action for rigosertib was published in the prestigious peer-reviewed experimental biology journal, Cell. The paper, entitled "A small molecule RAS-mimetic disrupts RAS association with effector proteins to block signaling," appeared in the April 21, 2016 edition of the journal.
Proprietary NCE targeting ARK5 and CDK4/6

A presentation at the 2016 American Association of Cancer Research Annual meeting demonstrated the important differentiating features of ON 123300, Onconova’s novel pre-clinical ARK5 and CDK4/6 targeted agent. Results indicated that the inhibitory activity of ON 123300 on ARK5 and CDK4/6 resulted in the activation of programmed cell death in colorectal cancer cells, while treatment with an approved CDK4/6 inhibitor merely resulted in cytostasis.
Upcoming Events

Enrollment of the first patient in the Phase 3 INSPIRE trial in Japan: 2Q2016

Presentation of updated Phase 2 data from oral rigosertib combination trial in MDS: 2Q2016

End of Phase 2 meeting with FDA to discuss data from oral rigosertib combination trial: 2H2016
First Quarter 2016 Financial Results

Cash, cash equivalents, and marketable securities as of March 31, 2016 totaled $16.8 million, compared to $19.8 million as of December 31, 2015.
Total net revenue was $1.5 million for the first quarter of 2016 compared to $0.1 million for the first quarter of 2015.
Research and development expenses were $5.8 million for the first quarter of 2016 compared to $9.5 million for the first quarter of 2015.
General and administrative expenses were $3.2 million for the first quarter of 2016 compared to $3.0 million for the first quarter of 2015.

About Rigosertib

Rigosertib is a small molecule inhibitor of cellular signaling and acts as a RAS mimetic. These effects of rigosertib appear to be mediated by direct binding of the compound to the RAS-binding domain (RBD) found in many RAS effector proteins, including the Raf kinases and PI3K. The therapeutic focus for rigosertib is myelodysplastic syndromes (MDS), a group of bone marrow disorders characterized by ineffective formation of blood cells that often converts into acute myeloid leukemia (AML). Clinical trials for rigosertib are being conducted at leading institutions in the United States, Europe, and the Asia-Pacific region. Rigosertib is protected by issued patents (earliest expiry in 2026) and has been awarded Orphan Designation for MDS in the United States, Europe and Japan.

About INSPIRE

The INternational Study of Phase III IV RigosErtib, or INSPIRE, is based on guidance received from the U.S. Food and Drug Administration and European Medicines Agency and derives from the findings of the ONTIME Phase 3 trial. INSPIRE is a multi-center, randomized controlled study to assess the efficacy and safety of IV rigosertib in HR-MDS patients who had progressed on, failed to respond to, or relapsed after previous treatment with an HMA within the first nine months of initiation of HMA treatment. This time frame optimizes the opportunity to respond to treatment with an HMA prior to declaring treatment failure, as per NCCN Guidelines. The trial will enroll approximately 225 patients randomized at a 2:1 ratio into two treatment arms: IV rigosertib plus Best Supportive Care versus Physician’s Choice plus Best Supportive Care. The primary endpoint of INSPIRE is overall survival and an interim analysis is anticipated. Full details of the INSPIRE trial, such as inclusion and exclusion criteria, as well as secondary endpoints, can be found on clinicaltrials.gov (NCT02562443).