Prothena Reports Initiation of NEOD001 Global Phase 3; Keryx Offers Phase 2 Trial Results for Auryxia Print E-mail
By William Kent   
Tuesday, 02 December 2014 19:49
Below is a look at some of the headlines for companies that made news in the healthcare sector on December 2, 2014.

Prothena Corporation plc (NASDAQ: PRTA)
, a late-stage clinical biotechnology company focused on the discovery, development and commercialization of novel antibodies for the potential treatment of diseases that involve amyloid or cell adhesion, today announced the initiation of the VITAL Amyloidosis Study, an international, multi-center, registrational Phase 3 clinical trial, based on positive results from an ongoing Phase 1/2 clinical study of NEOD001 in patients with AL amyloidosis and persistent organ dysfunction.

"The results of our ongoing Phase 1/2 clinical study confirm and strengthen data presented in April 2014 at the XIV International Symposium on Amyloidosis and I'm pleased to report that as of September 30, 2014, we believe that the primary and secondary endpoints of the trial have been met. Importantly, we believe the robust 50.0% cardiac and 42.9% renal best response rates in patients treated with NEOD001 compare favorably with historical data that would have predicted 26.5% cardiac and approximately 24% renal response rates in patients treated solely with off-label standard of care," said Gene Kinney, PhD, Chief Scientific Officer and Head of Research and Development at Prothena. "We look forward to further evaluation of NEOD001 in the expansion portion of our Phase 1/2 study that is currently enrolling additional patients with AL amyloidosis and selected persistent organ dysfunction for which we expect to present initial results in 2015, and annually thereafter. Today, following favorable conversations with the US and EU regulatory bodies, we are excited to announce initiation of the VITAL study, our registrational Phase 3 trial in newly-diagnosed, treatment-naive patients with AL amyloidosis."

Cardiac and Renal Biomarker Responses in Phase 1/2 Study -- Seven of 14 cardiac-evaluable patients (50.0%) treated with NEOD001 demonstrated a cardiac response, defined as more than 30.0% and 300 pg/mL decrease in levels of NT-proBNP (a validated cardiac biomarker associated with mortality). Cardiac responders, on average, showed more NT-proBNP decline with added monthly NEOD001 infusions. The 50.0% cardiac response rate compares favorably with the expected results of a 26.5% cardiac response rate from historical data in patients treated solely with off-label standard of care (Comenzo, et al., Leukemia. 2012;26:2317-2325). As noted in numerous peer-reviewed publications, increasing levels of NT-proBNP predicts higher mortality rates in patients with AL amyloidosis. Conversely, decreasing levels of NT-proBNP predicts lower mortality rates.

In a best response analysis of renal-evaluable patients treated with NEOD001, six of 14 renal-evaluable patients (42.9%) demonstrated a response, defined as a 30.0% decrease in proteinuria in the absence of estimated glomerular filtration rate (eGFR) worsening. The 42.9% renal response rate compares favorably with the expected results of an approximately 24% renal response rate from historical data in patients treated solely with off-label standard of care (Palladini, et al., Blood. 2014 124: 2325-2332). Increased levels of proteinuria and decreased eGFR predicts faster progression to dialysis where decreased levels of proteinuria and increased eGFR predicts delayed time to dialysis.

"NEOD001 potentially holds significant promise for patients with AL amyloidosis as we now see clinically meaningful decreases in both cardiac and renal biomarkers with monthly NEOD001 infusions," said Raymond L. Comenzo, MD, Professor of Medicine and Pathology at Tufts University School of Medicine. "NEOD001 appears to be the first agent that works directly to address the buildup of light-chain (AL) amyloid in organs. The results presented today strongly support moving into a Phase 3 trial, which I believe is designed to test whether or not NEOD001 provides meaningful clinical benefit for patients with AL amyloidosis."

Mechanism of Action -- The clinical results demonstrated to date expand on more than a decade of amyloid research. NEOD001 elicits a rapid response initially, and a deepening response with additional monthly infusions. The results are consistent with the mechanism of action showing that NEOD001 functions in two ways: neutralization of circulating soluble amyloid and clearance of deposited insoluble amyloid within affected organs. The Phase 1/2 data supports that NEOD001 acts as a disease-modifying agent in AL amyloidosis which is distinct from current off-label standard of care therapies that attempt to solely reduce production of immunoglobulin light chain and are associated with adverse events.

Safety, Tolerability, Pharmacokinetics and Immunogenicity -- Data from the Phase 1/2 study continued to demonstrate that chronic monthly infusions of NEOD001 are safe and well-tolerated in patients with AL amyloidosis and persistent organ dysfunction. A database analysis as of September 30, 2014 showed a total of 27 patients in seven dosing cohorts received 209 infusions, with each patient treated on average for approximately eight months. No hypersensitivity reactions or drug-related serious adverse events were reported and no anti-NEOD001 antibodies were detected. NEOD001 demonstrated excellent pharmacokinetic properties, supporting a dose level of 24 mg/kg on a 28 day cycle. The most frequently reported adverse events (more than 10% of subjects) were fatigue, cough, dyspnea, diarrhea, upper respiratory infection, anemia, headache, hyponatremia, nausea and edema. All adverse events were mild to moderate and no dose limiting toxicities have been observed. As of September 30, 2014, 19 patients continue on therapy (eight patients discontinued). No patient discontinued due to drug-related adverse events. Following selection of 24 mg/kg as the Phase 3 recommended dose, in consultation with their treating physician, 13 out of 14 eligible patients continuing in the dose escalation portion of the Phase 1/2 study have chosen to escalate to 24 mg/kg.

Expansion Portion of Phase 1/2 Study -- Prothena is now enrolling up to an additional 25 patients, with AL amyloidosis and selected persistent organ dysfunction, in an open-label expansion portion of the Phase 1/2 study. The company plans to enroll 10 patients with cardiac dysfunction, 10 patients with renal dysfunction and five patients with peripheral neuropathy, all of whom will receive 24 mg/kg intravenously every 28 days. The expansion phase will continue to evaluate safety, tolerability, pharmacokinetics and immunogenicity of NEOD001 as well as the specific clinical activity against cardiac, renal and neuropathy biomarkers. The company expects to present results from the NEOD001 expansion portion of the Phase 1/2 study at least once annually at appropriate medical conferences, beginning in 2015.

VITAL Phase 3 Registrational Trial Design -- The multi-center, randomized, double-blind, placebo-controlled Phase 3 study continues Prothena's commitment to provide disease-modifying therapeutic alternatives for patients suffering from AL amyloidosis. The trial is designed to support global regulatory approvals and to enroll approximately 230 newly-diagnosed, treatment-naive patients with cardiac dysfunction. Patients will be randomized on a 1:1 basis to receive 24 mg/kg of NEOD001 or placebo via infusion every 28 days, with both arms receiving concurrent standard of care therapy.

The composite primary endpoint is event-based, with all-cause mortality or cardiac hospitalizations as qualifying events. Secondary endpoints of the study include evaluation of the cardiac biomarker NT-proBNP, renal biomarker proteinuria, six-minute walk test, and multiple quality of life evaluations including SF-36 and the Kansas City Cardiomyopathy Questionnaire. Prothena designed the study with 90% power to detect as little as 30% change in the event rate between the treatment and placebo groups with a two-sided alpha of 0.05. The trial allows for an interim analysis to assess the primary endpoint for efficacy and futility.

"Based on the encouraging results from our ongoing NEOD001 Phase 1/2 clinical study, which confirms safety, tolerability and pharmacokinetic properties, and noteworthy responses in patients with organ dysfunction, we are pleased to initiate the VITAL study, our global Phase 3 trial for newly-diagnosed, treatment-naive patients with AL amyloidosis," said Dale Schenk, PhD, President and Chief Executive Officer of Prothena. "We believe the responses in multiple organs of NEOD001-treated patients compare favorably to historic data in AL amyloidosis, and support and inform the initiation of the VITAL study, our global Phase 3 registrational trial."

"This is an important event for the AL amyloidosis community, who is in desperate need of new safe and well tolerated therapeutic alternatives for this deadly, progressive disease. It is also a significant corporate milestone for Prothena, as it marks the first Phase 3 program to evaluate a disease-modifying agent targeting toxic amyloid in AL amyloidosis," continued Dr. Schenk.

Conference Call Details -- Prothena management will discuss these new Phase 1/2 study results with NEOD001 for the treatment of AL amyloidosis in addition to the Phase 3 study design for NEOD001, in a live audio webcast and conference call today, Tuesday, December 2, 2014 at 4:30 p.m. ET. The webcast and slide presentation will be made available on the company's website at under the Investors tab in the Events and Presentations section. Following the live audio webcast, a replay of the webcast will be available on the Company's website for 90 days.


Keryx Biopharmaceuticals, Inc. (Nasdaq:KERX)
, announced the publication of Auryxia phase 2 clinical trial results in non-dialysis dependent chronic kidney disease (NDD-CKD) patients in the American Journal of Kidney Diseases (AJKD). Based on the results of this study, in September 2014, the Company initiated a Phase 3 pivotal program of Auryxia as a treatment for iron-deficiency anemia in patients with Stages 3-5 NDD-CKD.

The study's Co-Chairmen were Geoffrey Block, MD, Director of Clinical Research at Denver Nephrology; Glenn Chertow, MD, Professor of Medicine and Chief, Division of Nephrology at Stanford University School of Medicine; and Steven Fishbane, MD, Division Chief, Kidney Disease and Hypertension at North Shore University Hospital/Long Island Jewish Medical Center.

Dr. Block commented, "Iron deficiency anemia is very common in chronic kidney disease stages 3 to 5, with more than 1.5 million Americans estimated to be impacted by this condition. Currently, there are no good treatment options for patients as conventional oral iron formulations are poorly absorbed and not well tolerated. The results of this study support further evaluation of Auryxia for the treatment of iron deficiency anemia in this patient population."

Also Tuesday:

ADMA Biologics, Inc. (Nasdaq:ADMA),
a late-stage biopharmaceutical company that develops, manufactures, and intends to market specialty plasma-based biologics for the treatment and prevention of certain infectious diseases, today announced it is scheduled to present at the Oppenheimer 25th Annual Healthcare Conference at 10:20 AM ET on December 11, 2014 at the Crowne Plaza Hotel in New York City.

Adamis Pharmaceuticals Corporation (Nasdaq:ADMP)
today announced that the Company is scheduled to present a company overview at two upcoming conferences.

Apricus Biosciences, Inc. (Nasdaq:APRI)
, a biopharmaceutical company advancing innovative medicines in urology and rheumatology, today announced the appointment of Brian T. Dorsey as the Company's Chief Development Officer, a newly established position.

Bio Blast Pharma Ltd. (Nasdaq:ORPN)
, a clinical-stage biotechnology company committed to developing clinically meaningful therapies for patients with rare and ultra-rare genetic diseases, announced today that Dr. Dalia Megiddo, Chief Executive Officer, will present a corporate overview and business update at the Oppenheimer & Co., Inc.'s 25th Annual Healthcare Conference, being held at the Crowne Plaza Hotel in New York City on December 10-11, 2014.

Calithera Biosciences, Inc. (Nasdaq:CALA)
, a clinical-stage pharmaceutical company focused on discovering and developing novel small molecule drugs directed against tumor metabolism and tumor immunology targets for the treatment of cancer, announced today the appointment of H. Ward Wolff to the company's Board of Directors, where he will chair the company's Audit Committee.

Capricor Therapeutics, Inc. (OTCBB:CAPR)
, a biotechnology company focused on developing novel therapeutics for the treatment of cardiovascular diseases, announced today that CEO and President Linda Marbán will deliver an overview of Capricor Therapeutics, Inc., to Piper Jaffray's clients during a fireside chat session in the Rutherford Room of the New York Palace Hotel, December 2, 2014, at 9:00 a.m. EST.

Cara Therapeutics, Inc. (Nasdaq:CARA)
, a biopharmaceutical company focused on developing and commercializing new chemical entities designed to alleviate pain and pruritus by selectively targeting kappa opioid receptors, today announced the successful completion of a Phase 1a / 1b clinical trial of an oral tablet formulation of its peripherally-selective kappa opioid agonist, CR845, for the treatment of acute and chronic pain.

Cara Therapeutics, Inc. (Nasdaq:CARA)
, a biopharmaceutical company focused on developing and commercializing new chemical entities designed to alleviate pain and pruritus by selectively targeting kappa opioid receptors, today announced the appointment of Joseph Stauffer, D.O., as Chief Medical Officer, effective immediately.

Celsus Therapeutics (Nasdaq:CLTX)
, an emerging growth, development-stage biopharmaceutical company, announces that Gur Roshwalb, MD, chief executive officer, is scheduled to present at the Oppenheimer 25th Annual Healthcare Conference at Crowne Plaza Hotel in New York City.

Cortex Pharmaceuticals, Inc. (CORX)
, a leader in developing drugs for respiratory disorders, particularly drug-induced respiratory depression as well as obstructive, central and mixed sleep apneas, announces that it will be participating in and presenting at the 7th Annual Biotech Showcase 2015™ being held at the Parc 55 Wyndham Hotel in San Francisco, California from January 12 – 15, 2015.

Ekso Bionics Holdings, Inc. (OTCQB:EKSO)
, a robotic exoskeleton company, today announced that Ekso Bionics recently met with FDA to discuss a change to the regulatory status of the Ekso robotic exoskeleton and other similar medical devices.

Galena Biopharma, Inc. (Nasdaq:GALE)
, a biopharmaceutical company developing and commercializing innovative, targeted oncology treatments that address major medical needs across the full spectrum of cancer care, today announced that it has received a Notice of Allowance from the United States Patent and Trademark Office (USPTO) for a patent application for GALE-401 composition of matter.

Heat Biologics, Inc. (Nasdaq:HTBX)
, a clinical stage biopharmaceutical company focused on the development of cancer immunotherapies, today announced the expansion of its product development team.

Idera Pharmaceuticals, Inc. (Nasdaq:IDRA)
, a clinical-stage biopharmaceutical company developing nucleic acid therapeutics for patients with cancer and rare diseases, today announced new preclinical data that showed cancer immunotherapy with intratumoral injections of IMO-2055 and ipilimumab demonstrated potent and systemic anti-tumor activity in multiple preclinical cancer models.

Idera Pharmaceuticals, Inc. (Nasdaq:IDRA)
, a clinical-stage biopharmaceutical company developing novel nucleic acid therapeutics for cancer and rare diseases, today announced that Company management will present at the 26th Annual Piper Jaffray Healthcare Conference on Wednesday, December 3, 2014 at 3:30 p.m. EST in New York.

Marrone Bio Innovations, Inc. (Nasdaq:MBII)
, a leading provider of bio-based pest management and plant health products for the agriculture, turf and ornamental and water treatment markets, today announced that it has relocated its corporate headquarters to 1540 Drew Avenue, Davis, CA 95618, effective December 8, 2014.

Merge Healthcare Incorporated (Nasdaq:MRGE)
, a leading provider of clinical systems and innovations that seek to transform healthcare, today announced a strategic partnership with NextGen Healthcare, a wholly owned subsidiary of Quality Systems, Inc. (Nasdaq:QSII) and leading provider of integrated clinical, connectivity and financial solutions.

Myriad Genetics, Inc. (Nasdaq:MYGN)
today announced that clinical data from three studies with Prolaris in prostate cancer patients will be highlighted at the 2014 Society of Urologic Oncology (SUO) Annual Meeting being held tomorrow in Rockville, Md.

Nuo Therapeutics (OTCQX:NUOT)
, a pioneer in biodynamic therapies, today announced that it, along with its marketing partner BLDG Health, was presented with the 2014 Platinum MarCom Award by the Association of Marketing and Communication Professionals (AMCP) for its sales education modules designed to support the recently launched Aurix System™.

Oxford Immunotec Global PLC (Nasdaq:OXFD)
, a global, commercial-stage diagnostics company committed to improving patient care by providing advanced, innovative tests in the field of immunology, today announced the appointment of James R. Tobin to its Board of Directors.

Protea Biosciences Group, Inc. (OTCQB:PRGB)
announced today that its CEO and Chairman, Stephen Turner, will present at the 7th annual LD Micro "Main Event" Micro-Cap Growth Conference on Thursday, Dec. 4 at 1:00 p.m. PT.

QLT Inc. (Nasdaq:QLTI) (TSX:QLT)
today announced that Sukhi Jagpal, Chief Financial Officer of the Company, has notified the Company that he is leaving to pursue other opportunities.

RedHill Biopharma Ltd. (Nasdaq:RDHL) (TASE:RDHL)
, an Israeli biopharmaceutical company focused on late clinical-stage drugs for inflammatory and gastrointestinal diseases, including gastrointestinal cancers, today announced that it will provide Silver Sponsorship for the Israeli IBD (Inflammatory Bowel Disease) Society meeting to be held December 9, 2014 at the Dan Hotel in Tel-Aviv, Israel.

, a shockwave technology company using its patented technology in wound healing to treat diabetic foot ulcers, today announced that is has signed memoranda of understanding with Dasman Diabetes Institute and Kuwait Life Sciences Company (KLSC), a wholly owned subsidiary of the National Technology Enterprises Company (NTEC), to establish a strategic alliance and joint venture that will introduce SANUWAVE's dermaPACE® device as a preferred treatment for diabetic foot ulcers in the six nation Gulf Cooperation Council (GCC).

StemCells, Inc. (Nasdaq:STEM)
, a leader in the development of cell-based therapies for disorders of the central nervous system (CNS), hosted its first investor and analyst event in New York, on November 20, 2014.

Vascular Solutions, Inc. (Nasdaq:VASC)
today announced that it has completed the acquisition of its manufacturing facility, a 79,300-square-foot building located at 6464 Sycamore Court North in Maple Grove, Minnesota.

Zosano Pharma Corporation
and Eli Lilly and Company (NYSE:LLY) today announced that they have entered into an exclusive agreement to develop ZP-PTH, Zosano's proprietary formulation of parathyroid hormone 1-34 (PTH) using Zosano's microneedle patch system for the treatment of osteoporosis patients, globally.

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