On June 3, 2015 4SC AG (Frankfurt, Prime Standard: VSC), a discovery and development company of targeted small molecule drugs for cancer and autoimmune diseases, reported that it published clinical data on safety, pharmacokinetics and efficacy for the 4SC-205 cancer compound at the annual meeting of the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) in Chicago (Press release, 4SC, JUN 3, 2015, View Source [SID:1234506550]). The data has been obtained from the clinical Phase I AEGIS trial examining 4SC-205 in various dosing schemes in 59 patients with advanced solid tumours. 4SC-205 inhibits specifically the human kinesin spindle protein Eg5 (Kif 11), which has been shown to play a crucial role in mitosis (cell division) and, therefore, in tumour growth. To 4SC’s knowledge, 4SC-205 is the only orally available Eg5 inhibitor in clinical development worldwide. The ASCO (Free ASCO Whitepaper) poster can be downloaded from the 4SC website at View Source

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Since the Eg5 target molecule is present in the cell only during mitosis, and since mitoses in humans – in contrast to preclinical models – are very rare events of limited duration (approx. 30 minutes per mitosis), it is necessary to ensure continuous exposure of 4SC-205. This means that potentially efficacious and tolerable levels of the compound must be permanently available in patients in order to become effective immediately as soon as mitosis occurs.

Conducted at two study centres in Germany, the first-in-man, open-label AEGIS dose escalation and dose-schedule finding study investigated the oral administration of 4SC-205 in patients with advanced solid tumours. Initially, the compound was tested in a conventional, intermittent dosing scheme (consisting of higher single doses and longer breaks between treatments). Given its oral availability and its mechanism of action as a potential inhibitor of mitosis (cell division), 4SC-205 was subsequently examined in a daily (continuous) dosing scheme consisting of smaller single daily doses without breaks between treatments. The objective of the study was to evaluate safety, tolerability and pharmacokinetics, and to determine a recommended Phase II dose. The trial ended when treatment of the last patient was completed in the first quarter of 2015.

The data in detail: Good profiles of safety and tolerability, linear pharmacokinetic parameters, 20 mg daily dose of 4SC-205 recommended as Phase II dose

– A comprehensive safety and tolerability profile of 4SC-205 was established. Specifically, no peripheral neuropathies were observed. This side effect is typically observed in connection with conventional chemotherapeutic agents such as taxanes. This means that the approach of a targeted, anti-mitotic therapy, during which these side effects should not occur, was confirmed.

– The main side effects were neutropenia in the intermittent dosing scheme. At a daily dose of 20 mg the neutropenia development was well manageable.

– The oral compound demonstrated very good linear pharmacokinetic parameters, which is an ideal prerequisite for daily dosage.

– A pharmacodynamic biomarker is regulated in a dose-dependent manner, even at a low daily dose.

– The daily dose of 20 mg is recommended as the dose for further Phase II development of the compound. This dose was safe and well-tolerated by patients and furthermore demonstrated initial signs of clinical efficacy.

– An additional, positive result of the daily dose of 20 mg of 4SC-205 was the median time on treatment of 162 days, thus being four times longer than the time on treatment seen in the intermittent treatment regime (42 days). Moreover, 67% of patients in the 20 mg daily cohort showed disease stabilisation for more than 100 days.

Enno Spillner, CEO of 4SC AG, commented: "We are pleased with the successful completion of the AEGIS trial. 4SC-205 very specifically inhibits an interesting therapeutic target in anti-cancer treatment, the Eg5 protein, which plays an important role in cell mitosis and tumour growth. Since 4SC-205 is orally available, we were able to evaluate this compound as the first of its kind in a clinically promising continuous dosing scheme. In this process we identified a safe and potentially effective dose for possible future Phase II trials. We are currently reviewing scenarios of further clinical development and will intensify discussions with clinical experts and potential academic and industry partners."

Details of ASCO (Free ASCO Whitepaper) poster presentation

View Source
Abstract No. 2528
Poster Title: Overcoming the proliferation rate paradox: Clinical evaluation of a continuous dosing scheme of the novel oral Eg5 inhibitor 4SC-205.
Time/Location: 30 May 2015, 8:00-11:30am, CDT, McCormick Place: S Hall A, Board #244
Poster Session: Developmental Therapeutics – Clinical Pharmacology and Experimental Therapeutics
Sub-Category: Cell Cycle and Checkpoints
Authors: Klaus B. Mross, Dirk Scharr, Heike Richly, Sebastian Bauer, Babett Krauss, Rolf Krauss, Bernhard Hauns, Tanja Prenzel, Hella Kohlhof, Roland Baumgartner, Max E. Scheulen;
Klinik für Tumorbiologie, Freiburg, Germany; Department of Medical Oncology, West German Cancer Center, University Hospital Essen, Germany; University Clinic Essen, Germany; 4SC AG, Planegg-Martinsried, Germany; Innere Universitaetsklinik und Poliklinik, Essen, Germany

About 4SC-205

4SC-205 is a specific small molecule inhibitor of the human kinesin spindle protein Eg5 which is of crucial importance for cell division (mitosis). Eg5 interacts with microtubules, a component of the cellular mitosis machinery, and mediates the segregation of the two spindle poles resulting in the correct distribution of the chromosomes to the daughter cells. Inhibition of Eg5 leads to cell cycle arrest in mitosis und subsequent programmed cell death (apoptosis). Mitosis is the fundamental process leading to cell division and tissue growth. The mitotic spindle apparatus has been for decades a primary target for the development of anti-mitotic agents such as the taxanes and vinca alkaloids which are broadly used in cancer therapies as single chemotherapeutic agents or in combination. In preclinical tests 4SC-205 has proven to be a particularly effective inhibitor of tumour cell proliferation of various cancer origins, both in vitro and in vivo.

On June 3, 2015 Celgene International Sàrl, a wholly-owned subsidiary of Celgene Corporation (NASDAQ:CELG), reported that data from 11 abstracts (two oral presentations, six poster presentations and three published in The Abstract Book) evaluating Celgene investigational and marketed products will be presented at the European League Against Rheumatism (EULAR) Annual Congress in Rome, Italy, June 10 – 13, 2015 (Press release, Celgene, JUN 3, 2015, View Source [SID:1234512521]). The data will include the latest research findings on Otezla (apremilast), the Company’s oral, selective inhibitor of phosphodiesterase 4 (PDE4), in psoriatic arthritis and plaque psoriasis, as well as CC-220, an investigational immunomodulatory compound for systemic lupus erythematosus (lupus).

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Among the data presented will be long-term (104-week) results from Celgene’s PALACE program, including pooled results of three phase III trials (PALACE 1, 2 and 3) assessing the effects of OTEZLA on two distinct manifestations of psoriatic arthritis – enthesitis (inflammation at sites where tendons or ligaments insert into bone) and dactylitis (inflammation of an entire digit). Additional analyses of PALACE trials will evaluate long-term safety and efficacy of OTEZLA in patients with active psoriatic arthritis, as well as the impact of OTEZLA on work productivity and physical function in these patients.

Data will also be presented on the effect of CC-220 on blood cell levels of Ikaros and Aiolos – transcription factors that, when mutated, are associated with an increased risk of systemic lupus erythematosus. The presentation will include phase I data on the impact of CC-220 on the immune response in healthy volunteers. Additional preclinical studies on CC-220 in lupus will be presented.

"We are excited about the presentation of these new long-term data of OTEZLA in psoriatic arthritis at EULAR. Additionally, data presented on our investigational compound CC-220 provide one example of the depth of Celgene’s clinical trial programs in other serious inflammatory diseases with high unmet medical need, including lupus," said Scott Smith, President, Celgene Inflammation & Immunology. "We remain committed to further developing our new and existing therapies to provide innovative treatment options for patients living with painful, debilitating chronic immune conditions."

Celgene will also host a variety of educational programs during the Congress on the unmet needs of patients with psoriatic arthritis, including a symposium for healthcare professionals as well as programs for patient/professional advocacy organizations and media.

The following abstracts will be presented at EULAR as an exchange of scientific and clinical information (all times, CEST):

OTEZLA (apremilast) Abstracts at a Glance

Oral Presentation 3594; Friday, June 12, 10:30 AM – 12:00 PM
Apremilast, an Oral Phosphodiesterase 4 Inhibitor, is Associated with Long-Term (104-Week) Improvements in Enthesitis and Dactylitis in Patients with Psoriatic Arthritis: Pooled Results from Three Phase III Randomized, Controlled Trials; Dafna Gladman, MD
Location: Hall 3, 10:45 AM – 10:55 AM

Poster Number 1113; Poster Tour Presentation Thursday, June 11, 12:05 PM – 1:45 PM
Apremilast, an Oral Phosphodiesterase 4 Inhibitor: Improvements in Nail and Scalp Psoriasis and Psoriasis Area and Severity Index in Patients with Moderate to Severe Plaque Psoriasis (ESTEEM 1 and 2); Kim Papp, MD
Poster Tour Presentation location and time: Hall 5, 12:05 PM

Poster Number 2907; Poster Display Thursday, June 11, 8:00 AM – 5:30 PM
Long-Term (104-Week) Efficacy and Safety Profile of Apremilast, an Oral Phosphodiesterase 4 Inhibitor, in Patients with Psoriatic Arthritis: Results from a Phase III, Randomised, Controlled Trial and Open-Label Extension (PALACE 1); Arthur Kavanaugh, MD
Poster Tour Presentation location and time: Hall 5, 12:00 PM – 1:45 PM

Poster Number 2889; Poster Tour Presentation Thursday, June 11, 12:05 PM – 1:45 PM
Disease Activity and Safety During Long-Term (104-Week) Treatment with Apremilast, an Oral Phosphodiesterase 4 Inhibitor, in Patients with Psoriatic Arthritis: Results from a Phase III, Randomized, Controlled Trial and Open-Label Extension (PALACE 3); Christopher Edwards, MD
Poster Tour Presentation location and time: Hall 5, 12:05 PM

Poster Number 2986; Poster Tour Presentation Saturday, June 13, 10:20 AM – 12:00 PM
Long-Term (104-Week) Efficacy and Safety of Apremilast Monotherapy in DMARD-Naïve Patients with Psoriatic Arthritis: A Phase III, Randomized, Controlled Trial and Open-Label Extension (PALACE 4); Alvin Wells, MD
Poster Tour Presentation location and time: Hall 5, 11:20 AM

Poster Number 3582; Poster Display Thursday, June 11, 8:00 AM – 5:30 PM
Long-Term (104-Week) Safety Profile of Apremilast, an Oral Phosphodiesterase 4 Inhibitor, in Patients with Psoriatic Arthritis: Pooled Safety Analysis of Three Phase III, Randomized, Controlled Trials; Philip Mease, MD
Poster Tour Presentation location and time: Hall 5, 12:00 PM – 1:45 PM

Poster Number 3590; Poster Display Saturday, June 13, 8:15 AM – 2:00 PM
Long-Term Work Productivity Improvement Associated with Apremilast, an Oral Phosphodiesterase 4 Inhibitor, in Patients with Psoriatic Arthritis: Pooled Analysis of Three Phase III Studies; Frank Zhang, MD
Poster Tour Presentation location and time: Hall 5, 10:15 AM – 12:00 PM

Publication Number 4114
Long-Term Impact of Apremilast on Physical Function in Patients with Psoriatic Arthritis Using the HAQ-DI Assessment; Frank Zhang, MD

CC-220 Abstracts at a Glance
Oral Presentation 3498; Thursday, June 11, 10:30 AM – 12:00 PM
The CRL4CRBN E3 Ubiquitin Ligase Modulator CC-220 Induces Degradation of the Transcription Factors Aiolos and Ikaros: Immunomodulation in Healthy Volunteers and Relevance to Systemic Lupus Erythematosus; Peter Schafer, Ph.D.
Oral Presentation location and time: Hall 8 – Room 8A, 11:35 AM (preliminary)

Publication Number 3187
B-Cell Proliferation and Plasmablast Generation from Naïve and Memory B Cells are Differentially Regulated by Baff, Il-21, and Cd40l and Inhibited by the Systemic Lupus Erythematosus Drug Candidate CC-220; Yumi Nakayama, MD

Publication Number 3487
Effects of CC-220, a CRL4CRBN E3 Ubiquitin Ligase Modulator, on Immune Responses; Ying Ye, Ph.D.

On June 3, 2015 Boehringer Ingelheim reported that following a review by the National Institute for Health and Care Excellence (NICE) in the UK, VARGATEF (nintedanib*) in combination with docetaxel has been recommended for use within the National Health Service (NHS) in England and Wales (Press release, Boehringer Ingelheim, JUN 3, 2015, View Source [SID:1234505215]).1 This is positive news for patients with locally advanced metastatic or locally recurrent non-small cell lung cancer (NSCLC) of adenocarcinoma histology who have had limited treatment options that have been shown to extend survival after first-line chemotherapy.

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Dr Mehdi Shahidi, Medical Head, Solid Tumour Oncology, Boehringer Ingelheim commented: "We are delighted with this announcement from NICE which represents an important milestone in the effort to extend survival for patients with adenocarcinoma of the lung following initial treatment with chemotherapy. The survival advantage demonstrated by VARGATEF, in combination with docetaxel, has been shown with a predictable and generally manageable safety profile."

NICE has issued a Final Appraisal Determination (FAD) for VARGATEF and, subject to any appeal by consultees, the FAD may be used as the basis for the institute’s guidance on the use of the appraised technology in the NHS in England and Wales.1 Nintedanib, in combination with docetaxel, is the first and only triple angiokinase inhibitor available for EU patients with advanced NSCLC of adenocarcinoma histology after first-line chemotherapy.

Since the launch of VARGATEF in January 2015 it has also been:

accepted for use without restriction within NHS Scotland, as per its licensed indication2

accepted by the Swedish Dental and Pharmaceutical Benefits Agency to be subsidised as a treatment of advanced lung cancer patients with adenocarcinoma in combination with docetaxel after first-line chemotherapy3

assessed by the German IQWiG who indicated an added benefit for the treatment of advanced lung cancer patients with adenocarcinoma in combination with docetaxel after first-line chemotherapy without brain metastases4

These announcements are based on the outcomes of the LUME-Lung 1 study which demonstrated:5

nintedanib, plus docetaxel, significantly prolonged progression-free survival compared to docetaxel alone for patients with
adenocarcinoma (PFS: primary endpoint; 4.0 vs 2.8 months)

nintedanib, plus docetaxel, significantly extended overall survival to beyond one year for patients with adenocarcinoma, compared to docetaxel alone (OS: key secondary endpoint; 12.6 vs 10.3 months)

nintedanib, plus docetaxel, enabled 1 in 4 patients with adenocarcinoma to live for at least two years after first-line chemotherapy

Nintedanib in combination with docetaxel demonstrated a generally manageable side-effect profile without further compromising patients’ overall, health-related, quality of life compared to chemotherapy alone. The most common adverse events for patients taking docetaxel vs nintedanib plus docetaxel included: nausea 18% vs 24%; vomiting 9% vs 17%; diarrhoea 22% vs 42% and elevated liver enzymes 8% vs 29%.5

Adenocarcinoma is the most common type of lung cancer and the majority of patients are diagnosed in an advanced stage.6 Most patients will experience disease progression during or after first-line chemotherapy and there is a significant need for new, effective second-line treatments.5

VARGATEF was granted EU marketing authorisation in November 2014 and in combination with docetaxel is indicated for use in adult patients with locally advanced, metastatic or locally recurrent non-small cell lung cancer (NSCLC) of adenocarcinoma tumour histology, after first-line chemotherapy.

On June 3, 2015 RedHill Biopharma reported an update on its development pipeline and anticipated key milestones (Press release, RedHill Biopharma, JUN 3, 2015, View Source [SID:1234505217]).

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RHB-105 – for H. pylori bacterial infection

Top-line results from the first Phase III clinical study with RHB-105 are expected in the third week of June 2015. The Phase III study (the ERADICATE Hp study) is currently ongoing in the U.S. for the treatment of H. pylori bacterial infection, a major cause of chronic gastritis, peptic ulcer disease, gastric cancer, and mucosa associated lymphoid tissue (MALT) lymphoma

RHB-105 was designated by the FDA as a Qualified Infectious Disease Product (QIDP) under the Generating Antibiotic Incentives Now (GAIN) Act, allowing RedHill to benefit from fast-track development status for RHB-105, priority review, and, if ultimately approved by the FDA, an additional five years of marketing exclusivity, for a total of 8 years

The 2015 global and U.S. market potential for H. pylori eradication therapies, at current branded prices, were recently estimated at approximately $4.83 billion and $1.45 billion, respectively, and could potentially grow with increasing awareness of the health risks associated with H. pylori infection and the benefits of its eradication1

BEKINDA (RHB-102) – for gastroenteritis and gastritis, and for chemotherapy and radiotherapy-induced nausea and vomiting (CINV and RINV, respectively)

Top-line results from the Phase III study with BEKINDA for acute gastroenteritis and gastritis (the GUARD study), currently ongoing in the U.S., are expected either in the fourth quarter of 2015 or the first quarter of 2016. The results are intended to support potential future submissions of marketing applications in both the U.S. and Europe, targeting an estimated potential worldwide market exceeding $650 million annually2

A meeting with the FDA is planned during the third quarter of 2015 to discuss the regulatory path of BEKINDA for the indications of acute gastroenteritis and gastritis, as well as a potential filing of a New Drug Application (NDA) for CINV

RedHill recently received feedback from European regulatory agencies regarding the European Marketing Authorization Application (MAA) submitted by RedHill in December 2014 for the oncology support indications of CINV and RINV. Clinical and manufacturing-related comments have been discussed with the UK Medicines and Healthcare Products Regulatory Agency (MHRA) and a six-month extension was agreed to, in principle, with the MHRA

A Phase IIa Proof of Concept study for a new undisclosed indication is planned to commence in the second half of 2015

RHB-104 – for Crohn’s disease, multiple sclerosis and other inflammatory diseases

Interim analysis of the Phase III study with RHB-104 for Crohn’s disease (the MAP US study) is expected in the second half of 2016, after half of the 270 patients expected to be enrolled in the study have completed 26 weeks of treatment. The primary endpoint is remission at week 26 of treatment

The Phase III MAP US study is currently enrolling patients in approximately 80 sites in the U.S., Canada, Israel and New Zealand, with new sites in Australia, New Zealand and Europe currently being added, for a total of up to 120 clinical sites

Clinical trial applications have been submitted in Europe, and initial comments received and responded to, for RHB-104’s second Phase III Crohn’s disease study (the MAP EU study) with potential European regulatory clearance expected in the third quarter of 2015

RedHill recently received a Notice of Allowance from the U.S. Patent and Trademark Office (USPTO) for two new patents covering RHB-104. Once granted, the patents are expected to be valid until at least 2029

The last patient has been screened in the ongoing Phase IIa Proof of Concept study with RHB-104 for the treatment of multiple sclerosis (MS) (the CEASE-MS study), with interim results expected either in the fourth quarter of 2015 or the first quarter of 2016

ABC294640 – for multiple inflammatory-GI diseases and related oncology indications

ABC294640 is a proprietary, first-in-class, orally administered, new chemical entity (NCE) sphingosine kinase-2 (SK2) inhibitor, which has successfully completed numerous pre-clinical studies and a Phase I study in cancer patients with advanced solid tumors.

ABC294640 targets multiple inflammatory, gastrointestinal and oncology indications within RedHill’s therapeutic focus.

A Phase Ib/II study of ABC294640 for refractory/relapsed diffuse large B cell lymphoma, primarily funded by the National Cancer Institute/STTR, is planned to commence either in the second or third quarter of 2015

A Phase II study in multiple myeloma is planned, subject to a pending National Cancer Institute/SBIR grant

A Phase II study to assess ABC294640 as a radio-protectant and radiation enhancer in cancer patients receiving radiotherapy is being planned by RedHill

RHB-106 – bowel cleanser pill

In February 2014, RedHill and Salix Pharmaceuticals, Inc. ("Salix") entered into a license agreement under which Salix acquired worldwide exclusive rights to RHB-106 and other purgative developments. In April 2015, Valeant Pharmaceuticals International, Inc. ("Valeant") announced the completion of its acquisition of Salix. The RHB-106 program is under review by Valeant following its acquisition of Salix

MESUPRON – for pancreatic cancer and other solid tumors

RedHill is preparing nonclinical studies to further evaluate the mechanism of action and define the patient population for MESUPRON, a Phase II orally-administered small molecule drug targeting pancreatic cancer and other solid tumors. MESUPRON is a first-in-class urokinase-type plasminogen activator (uPA) inhibitor

RP101 – for pancreatic cancer and other solid tumors

RedHill is preparing nonclinical studies to further evaluate the mechanism of action and define the patient population for RP101, a Phase II orally-administered small molecule drug targeting pancreatic cancer and other solid tumors. RP101 is a first-in-class heat shock protein 27 (Hsp27) inhibitor

Ebola virus disease – early stage development program

As part of a previously disclosed nonclinical research collaboration with a U.S. government agency, initial nonclinical studies have been completed, and RedHill is currently planning the next stage of development

RIZAPORT (RHB-103) – for acute migraines

Regulatory feedback regarding the MAA submitted in October 2014 is expected either in the fourth quarter of 2015 or the first quarter of 2016

RedHill and its Canadian co-development partner IntelGenx Corp. continue to work with the FDA to address the remaining Chemistry, Manufacturing and Controls (CMC) matters, and to secure a compliant source of raw material. The existing source of raw material for RIZAPORT has been successfully audited in recent months by non-U.S. regulatory agencies, as well as an independent auditor on behalf of RedHill, and is currently awaiting another FDA inspection, after which, and subject to a successful audit, a new FDA PDUFA date is expected

On June 3, 2015 Celator Pharmaceuticals reported that enrollment is complete in a Phase 2 pharmacokinetic and pharmacodynamics (PK/PD) study evaluating the effects of CPX-351 (cytarabine:daunorubicin) Liposome Injection on cardiac repolarization in adult patients with acute hematologic malignancies, including acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), and myelodysplastic syndrome (MDS) (Press release, Celator Pharmaceuticals, JUN 3, 2015, View Source [SID:1234505218]).

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The open-label, single-arm, Phase 2 study is a thorough PK/PD assessment designed to: (1) measure the effects of CPX-351 on cardiac repolarization following the first induction cycle of CPX-351, and (2) correlate changes in cardiac repolarization with plasma pharmacokinetic data for cytarabine and daunorubicin and their metabolites. The study began enrolling patients in August 2014. Each patient received a first induction of CPX-351 on days 1, 3 and 5 and, if necessary, a second induction for patients with reduced leukemia/MDS burden not yet achieving a disease-free state. Responding patients were eligible for up to four consolidation courses. Analysis of treatment impact on cardiac electrophysiology, as measured by the QTc interval, and PK assessments were performed following the first induction course.

The study enrolled patients with newly diagnosed de novo and high-risk (secondary) AML, relapsed/refractory AML and relapsed ALL. Fifteen of the 26 patients enrolled are evaluable for response at this time. Six of the 15 patients responded to CPX-351 (defined as CR-complete response or CRi-complete response with incomplete hematologic recovery) including 2 of 3 patients (67%) with high-risk (secondary) AML, the study population of the ongoing Phase 3 trial. Responses were also seen in patients with de novo AML, relapsed AML, and relapsed ALL.

"We are happy to report that this study confirms the broad activity of CPX-351 in multiple populations of acute leukemia patients," said Tara Lin, M.D., Assistant Professor of Medicine at The University of Kansas Cancer Center, the lead investigator of this study, "and will report cardiac repolarization and pharmacokinetic data necessary for the registration of CPX-351 later this year."

The Phase 2 study was conducted to support the U.S. Food and Drug Administration (FDA) requirements of a New Drug Application (NDA) for CPX-351. Our Phase 3 study comparing CPX-351 to the current standard of care, known as 7+3, is being conducted in patients with high-risk (secondary) AML. The Phase 3 study completed enrollment in November 2014. Initial data from a secondary endpoint, induction response rate, is expected by the end of June 2015. The primary endpoint data, of overall survival, is expected in the first quarter of 2016.

"We continue to work expeditiously to bring CPX-351 before the FDA as a potential new treatment option for patients with acute hematologic malignancies," said Scott Jackson, Chief Executive Officer of Celator Pharmaceuticals. "Clinical pharmacology studies are required by the FDA for new drugs in development, so we are pleased to have completed enrollment in this Phase 2 study to support a NDA submission for CPX-351. We expect to report top-line results from this study by the end of the year."