|Celator Announces Enrollment is Complete in CPX-351; Revance Therapeutics to Initiate Two Key Trials for RT001|
|By David Fowler|
|Wednesday, 03 June 2015 19:35|
Below is a look at some of the headlines for companies that made news in the healthcare sector on June 3, 2015.
Celator Pharmaceuticals, Inc. (NASDAQ: CPXX), announced 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).
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."
Revance Therapeutics, Inc. (NASDAQ: RVNC), announced plans to move forward with two key clinical studies for its investigational drug product candidate RT001, a topical gel formulation of botulinum toxin type A.
"We recently conducted an open-label study for the treatment of crow's feet lines, which enrolled an aggregate of 69 subjects across multiple sites. In the study, we evaluated the 28-day efficacy of a single topical application of the RT001 drug products tested. The subject response observed in the open-label study, taken together with our analysis of prior studies and early data from newly developed clinical methods, has led us to proceed with our RT001 U.S. Phase 3 clinical trial for the treatment of crow's feet lines. Based upon what we have learned, we believe we have an opportunity to achieve clinical and statistical significance in a Phase 3 clinical study," said Dan Browne, President and Chief Executive Officer at Revance. "We are making promising advances in the development of clinical methods, such as electromyography (EMG), intended to demonstrate the ability of RT001 to effectively cross the skin and achieve an associated paralytic effect directly on the target muscle," continued Browne.
In the second half of 2015, Revance plans to initiate a U.S. Phase 3 pivotal study to evaluate the safety and efficacy of a single topical application of RT001 on adults with moderate to severe crow's feet lines. The company also plans to begin a U.S. Phase 2 study to evaluate the safety and efficacy of a single application of topical RT001 on adults with moderate to severe axillary hyperhidrosis, or excessive sweating. Revance plans to provide trial design details and timelines for these studies when each commences later this year.
"We are pleased to be moving ahead with the development of RT001, which has the potential to transform today's botulinum toxin market. By eliminating the need for needles in aesthetic and therapeutic treatments, we believe our topical RT001 could significantly expand the estimated $3 billion neurotoxin market by removing many of the barriers that currently keep patients from adopting its use, including pain and bruising," concluded Browne.
RT001 and RT002 Drug Product Candidates -- Revance is currently developing two botulinum toxin type A investigational drug product candidates. RT001 is a topical formulation, which has the potential to be the first commercially available non-injectable dose form of botulinum toxin type A. Revance is studying topical RT001 for aesthetic indications, such as crow's feet lines (wrinkles around the eyes) and therapeutic indications such as hyperhidrosis (excessive sweating). RT002 is a novel, injectable formulation of botulinum toxin type A designed to be more targeted and longer lasting than currently available injectable botulinum toxin products. Revance is studying injectable RT002 for aesthetic indications, such as glabellar (frown) lines and therapeutic uses, such as muscle movement disorders (cervical dystonia and upper limb spasticity). Both products would have the potential to expand into additional aesthetic and therapeutic indications in the future.
Alliqua BioMedical, Inc. (Nasdaq:ALQA), a provider of advanced wound care products, today announced the appointment of Nino Pionati to the position of Chief Strategy and Marketing Officer.
Amicus Therapeutics (Nasdaq:FOLD), a biopharmaceutical company at the forefront of therapies for rare and orphan diseases, has submitted a marketing authorization application (MAA) to request full approval of the oral small molecule pharmacological chaperone Galafold (migalastat HCl) for Fabry patients who have amenable genetic mutations.
Arno Therapeutics, Inc. (OTCQB:ARNI), a clinical stage biopharmaceutical company primarily focused on the development of therapeutics for the treatment of cancer and other life threatening diseases, today announced it has entered into a Cooperative Research and Development Agreement (CRADA) Material Transfer Agreement with the US Army Medical Research Institute of Infectious Diseases (USAMRIID) to evaluate the anti-viral activity of AR-12 and various analogues against Ebola and other viruses of biodefense interest.
Bacterin International Holdings, Inc. (OTCQX:BONE), a leader in the development of revolutionary biomaterials, today announced the release of ArthroFuse, a patent pending allograft specifically designed for the treatment of hammertoe deformities.
Biota Pharmaceuticals (Nasdaq:BOTA) announced today that it has completed the acquisition of Anaconda Pharma, a privately-held biotechnology company based in Paris, France.
Chembio Diagnostics, Inc. (Nasdaq:CEMI), a leader in point-of-care ("POC") diagnostic tests for infectious diseases, announced today that it has received approval for a CE Mark for its DPP® HIV 1/2 Assay.
Ekso Bionics Holdings, Inc. (OTCQB:EKSO), a robotic exoskeleton company, announced today that their participation in Rehab Week 2015, Valencia Spain, will kick off with an inaugural Ekso Clinical Science Organization (ECSO) meeting.
Flexion Therapeutics, Inc. (Nasdaq:FLXN) today announced that a manuscript describing final study results from a completed Phase 2b clinical trial of FX006, an intra-articular (IA) sustained release corticosteroid for the treatment of osteoarthritis (OA), has been published online1 in the Journal of Bone and Joint Surgery (JBJS).
Galectin Therapeutics Inc. (Nasdaq:GALT), the leading developer of therapeutics that target galectin proteins to treat fibrosis and cancer, reports that Marc Rubin, M.D. has been elected by the independent members of the board of directors as the lead independent director.
Immunomic Therapeutics, Inc., a privately-held biotechnology company developing vaccines based on its proprietary LAMP technology, today announces that the company's CEO, William Hearl, Ph.D., will be one of the featured speakers at this year's Biotechnology Industry Organization (BIO) International Convention on June 15-18 at the Pennsylvania Convention Center in Philadelphia.
Juno Therapeutics, Inc. (Nasdaq:JUNO) today announced that it will webcast its presentation at the Goldman Sachs 36th Annual Global Healthcare Conference at 8:40 a.m. PT on Tuesday, June 9, 2015.
Lafayette General Health (LGH) has extended its nearly 20-year partnership with Premier, Inc. (Nasdaq:PINC) for an additional five years, as well as expanded access to Premier performance services across all of its hospitals.
Medical Marijuana, Inc. (OTC Pink:MJNA) is proud to announce to the public and shareholders that its portfolio Company, HempMeds™, received authorization from the Denver Public Health Department that Real Scientific Hemp Oil™ (RSHO™) Blue and Gold hemp oil brands (both 3- and 10-gram oral syringe tubes) qualified for "potentially non-hazardous foods not requiring time or temperature control."
Portola Pharmaceuticals (Nasdaq:PTLA) today announced that William Lis, chief executive officer, will present at the Goldman Sachs 36th Annual Global Healthcare Conference on Wednesday, June 10, at 8:00 a.m. Pacific Time in Rancho Palos Verdes, Calif.
Premier, Inc. (Nasdaq:PINC), a leading healthcare improvement company, today announced that its President and CEO, Susan DeVore, will present at the William Blair 35th Annual Growth Stock Conference in Chicago on Wednesday, June 10, 2015, at 1:20 p.m. CDT.
RedHill Biopharma Ltd. (Nasdaq:RDHL) (TASE:RDHL), an Israeli biopharmaceutical company focused on late clinical-stage, proprietary, orally-administered, small molecule drugs for inflammatory and gastrointestinal diseases, including gastrointestinal cancers, today provided an update on its development pipeline and anticipated key milestones.
Revance Therapeutics, Inc. (Nasdaq:RVNC), a specialty biopharmaceutical company developing botulinum toxin products for use in aesthetic and therapeutic indications, today announced its plans to move forward with two key clinical studies for its investigational drug product candidate RT001, a topical gel formulation of botulinum toxin type A.
SmallCapVoice.com, Inc. (SCV) and Pazoo, Inc. (OTC Pinks:PZOO) (German WKN#: A1J3DK), announced today that a new audio interview with the Company, is now available.
VBI Vaccines (Nasdaq:VBIV) delivered a presentation, Enveloped Virus-Like Particles: Third-generation VLPs for the Development of a Broadly Neutralizing Hepatitis C Vaccine, today at the World Vaccine Congress Asia.