|Arrowhead Issues Phase 1 Data on ARC-520 at HepDART '13; FDA to Review Results of Acura's Study AP-ADF-301|
|By Mary Davila|
|Monday, 09 December 2013 19:54|
Arrowhead Research Corporation (NASDAQ: ARWR), a biopharmaceutical company developing targeted RNAi therapeutics, announced COO and Head of R&D, Bruce Given, M.D., presented data on the Phase 1 clinical study of ARC-520, the company's clinical candidate for the treatment of chronic hepatitis B infection, at the HepDART 2013 conference being held on The Big Island, Hawaii. New data including pharmacokinetics (PK) and adverse event (AE) attribution presented today in a poster and in an oral presentation tomorrow, support the previous findings that ARC-520 appears to be generally safe and well-tolerated at all six dose levels studied.
The Phase 1 study was designed to characterize the safety profile of ARC-520 across a range of doses and evaluate pharmacokinetics. It is a single-center, randomized, double-blind, placebo-controlled, single dose-escalation, first-in-human study of ARC-520 administered intravenously to healthy adult volunteers. 36 subjects have been enrolled in 6 groups randomized at a ratio of 2:1 to receive ARC-520 or placebo: Placebo (n=12), ARC-520 0.01 mg/kg (n=4), 0.1 mg/kg (n=4), 0.3 mg/kg (n=4), 0.6 mg/kg (n=4), 1.2 mg/kg (n=4), and 2.0 mg/kg (n=4). The placebo group included 7 male and 5 female subjects with average of 28.1 +/- 9.6 years. The treatment group included 12 male and 12 female subjects with average age of 26.9 +/- 6.7 years. Subjects were admitted to the unit overnight pre-dose and vital signs, telemetry, ECGs, safety labs, PK, and adverse events were monitored for 24 hours post-dose. Return visits occurred for repeat safety evaluations and recording of adverse events at 48 hrs, 72 hours, day 7, day 14 and day 28 post dosing.
Preliminary results from the phase 1 clinical study of ARC-520 indicate that to date there have been no serious AEs, no dose limiting toxicities, no discontinuations, and a modest occurrence rate of AEs with no dose related increase in frequency or severity, with the possible exception of mild lightheadedness which occurred in two subjects in the 2 mg/kg dose group. There were no general differences observed or findings rated clinically significant on vital signs, ECGs, physical examinations, or clinical laboratories in the ARC-520 groups relative to placebo. Adverse event frequency and severity did not differ between placebo and ARC-520, with 75% of both treated and placebo subjects reporting mild or moderate AEs. There was a low occurrence rate of abnormal laboratory tests, with no observed relationship to timing or dose. PK results appear to indicate that C (equivalent to C[Max]) and AUC[0-∞] increase linearly with dose (r^2=0.984).
The Phase 1 study has thus demonstrated that a single intravenous administration of ARC-520 appears to be safe and well tolerated up to and including a dose of 2 mg/kg, the highest dose tested.
Acura Pharmaceuticals (NASDAQ: ACUR) announced that it met on December 5th with the U.S. Food and Drug Administration (FDA) to review the results of Acura's Study AP-ADF-301 (Study 301), a phase II clinical study in 40 recreational drug abusers assessing the abuse liability of snorting a crushed hydrocodone bitartrate with acetaminophen tablet formulated with Acura's abuse deterrent AVERSION technology (AVERSION H&A). The primary purpose of the meeting was to discuss if the FDA will consider whether the results of Study 301 are acceptable for submission in a New Drug Application (NDA). The meeting was designated a pre-NDA meeting.
The FDA agreed to further review the study results and supporting data within the context of the meeting discussions and provide a definitive response as to the suitability of Study 301 to be considered in an NDA review, including as part of product labeling. The Company and FDA also discussed, among other things, certain inactive ingredients contained in Acura's AVERSION technology, and the Company agreed to capture specific adverse events and safety information associated with these ingredients within its remaining planned development program. At this time, the Company does not expect the need to run additional clinical studies to provide the additional information requested by the FDA, beyond the pharmacokinetic studies planned but currently on hold.
A revised projected timeline for submission of the NDA for AVERSION H&A will be determined following receipt of the FDA's response.
Accelrys, Inc. (NASDAQ: ACCL), a leading provider of scientific innovation lifecycle management solutions, today announced that it has acquired Ireland-based QUMAS, a leading global provider of Cloud-based and on-premises enterprise compliance software supporting regulatory and quality operations in life sciences and other highly regulated industries.
Aurinia Pharmaceuticals Inc. (TSX-V:AUP) today announced the resignation of Dr. Robert Foster from its Board of Directors. Dr. Foster remains Chief Scientific Officer of the Company.
Chelsea Therapeutics International, Ltd. (Nasdaq:CHTP) today announced that the first patient has been dosed in Study 401, a multi-center, multi-national, randomized, parallel-group, placebo-controlled, double-blind study of investigational drug NORTHERA™ (droxidopa), an orally active synthetic precursor of norepinephrine, being studied for the treatment of symptomatic neurogenic orthostatic hypotension (nOH) in patients with primary autonomic failure (Parkinson's disease, multiple system atrophy and pure autonomic failure), dopamine beta hydroxylase deficiency and non-diabetic autonomic neuropathy.
Coronado Biosciences, Inc. (Nasdaq:CNDO), a biopharmaceutical company focused on the development of novel immunotherapy agents for the treatment of autoimmune diseases and cancer, today announced that it has submitted an Investigational New Drug (IND) application to the U.S. Food and Drug Administration to begin a Phase 2 clinical study of Trichuris suis ova (TSO) for the treatment of moderate to severe chronic plaque psoriasis.
Curis, Inc. (Nasdaq:CRIS), an oncology-focused company developing novel, targeted drug candidates for the treatment of human cancers, today announced preliminary first-in-human results from its ongoing Phase 1 trial of CUDC-907.
Cyclacel Pharmaceuticals, Inc. (Nasdaq:CYCC) (Nasdaq:CYCCP), today announced new, primary endpoint data from an ongoing, open-label, multicenter, randomized Phase 2 trial of oral sapacitabine capsules, the Company's lead product candidate, in older patients with myelodysplastic syndromes (MDS) after treatment failure of front-line hypomethylating agents, such as azacitidine and/or decitabine.
Enzymotec Ltd. (Nasdaq:ENZY), a developer, manufacturer and marketer of innovative bio-functional lipid ingredients, announced today the acceptance for future publication of its clinical study results evaluating the safety and effectiveness of Vayacog® usage in non-demented elderly.
Hemispherx Biopharma, Inc. (NYSE MKT:HEB) today announced that its Phase 1/2 clinical trial at the University of Alabama Vaccine Research Clinic is now being expanded based on an analysis of safety results to date conducted by both the Food and Drug Administration ("FDA") and a Data Monitoring Committee, the latter group comprised of a team of independent clinical, basic research, and statistical professionals.
Immunomedics, Inc. (Nasdaq:IMMU), a biopharmaceutical company primarily focused on the development of monoclonal antibody-based products for the targeted treatment of cancer, autoimmune and other serious diseases, today announced that veltuzumab, administered subcutaneously as a single agent, produced an overall objective response rate of 49% in 47 evaluable patients with relapsed immune thrombocytopenia (ITP), including 15 patients (32%) who reported a complete response.
Kraig Biocraft Laboratories, Inc. (OTCQB:KBLB), the leading developer of advanced spider silk based fibers, announced today that the Company delivered the first shipment of its Monster Silk(TM) recombinant spider silk fiber to Warwick Mills, Inc.
MacroGenics, Inc. (Nasdaq:MGNX), a clinical-stage biopharmaceutical company focused on discovering and developing innovative monoclonal antibody-based therapeutics for the treatment of cancer and autoimmune diseases, today announced the presentation of pre-clinical data for its proprietary DART® molecule, MGD006, at the 55th Annual Meeting of the American Society of Hematology (ASH), taking place in New Orleans.
Myriad Genetics, Inc. (Nasdaq:MYGN) today announced that Janssen Research & Development, LLC, will use Myriad's BRACAnalysis test in connection with its Phase 3 clinical trial of Yondelis® (trabectedin) in the treatment of advanced-relapsed epithelial ovarian, primary peritoneal or fallopian tube cancers (NCT01846611).
Nuvilex, Inc. (OTCQB:NVLX), an international biotechnology company providing cell and gene therapy solutions for the treatment of diseases, announced today it recently held three days of meetings to solidify the objectives and plans of Nuvilex for future research and development.
Onconova Therapeutics, Inc. (Nasdaq:ONTX), a clinical-stage biopharmaceutical company focused on discovering and developing novel products to treat cancer, today announced two presentations relating to clinical trials of its most advanced product candidate, rigosertib, at the 55th American Society of Hematology (ASH) Annual Meeting in New Orleans, Louisiana, December 7-10, 2013.
Pharmacyclics, Inc. (NASDAQ: PCYC) today announced that The Lancet Oncology published results of a study evaluating ibrutinib in previously untreated elderly patients with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL).
Portola Pharmaceuticals (Nasdaq:PTLA) today announced additional results of a Phase 2 proof-of-concept study that showed andexanet alfa's ability to immediately reverse the anticoagulation activity of XARELTO® (rivaroxaban) through the administration of a short intravenous bolus.
QLT Inc. (Nasdaq:QLTI) (TSX:QLT) today announced that the first patient has been dosed in the Company's Phase IIa proof-of-concept trial of QLT091001 in adult subjects with Impaired Dark Adaptation (IDA), a condition that results in decreased ability to recover visual sensitivity in the dark after exposure to bright lights.
Synergetics USA, Inc. (NASDAQ: SURG), a medical device company that designs, manufactures, and markets innovative surgical devices for ophthalmic and neurosurgical applications, today reported growth in sales and reported gross profit, operating margin, income from continuing operations and net income for the first quarter ended October 31, 2013.
TG Therapeutics, Inc. (Nasdaq:TGTX), an innovative, clinical-stage biopharmaceutical company today announced clinical results from an ongoing first-in-human Phase I single agent study of TGR-1202, the Company's oral PI3K-Delta inhibitor being developed for the treatment of select hematologic malignancies.
Vascular Solutions, Inc. (Nasdaq:VASC) announced today that the U.S. District Court for the District of Minnesota has granted Vascular Solutions' request for a preliminary injunction against Boston Scientific's Guidezilla guide extension catheter.