|Prothena Receives FDA Fast Track Designation for NEOD001; FDA Confirms Lipocine's Plan for LPCN 1021|
|By William Kent|
|Monday, 15 December 2014 21:22|
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 that the U.S. Food and Drug Administration (FDA) granted Fast Track designation to NEOD001, a novel monoclonal antibody for the potential treatment of AL amyloidosis. This is the first investigational immunotherapy specifically targeting the disease-causing protein in AL amyloidosis to receive Fast Track designation.
The FDA's Fast Track Drug Development Program is a process designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need. An unmet medical need is a condition whose treatment or diagnosis is not addressed adequately by available therapy. The purpose of the Fast Track designation is to make important new drugs available to patients earlier. The Fast Track program also provides a company with the ability to submit sections of the Biologics License Applications (BLA) for review before the company submits the complete BLA. This enables the FDA to review sections of the BLA as they are received, rather than waiting until every section of the application is completed, and also allows for Priority Review, shortening the standard review of the final BLA to six months. A drug program with Fast Track designation permits the company to have early and frequent communications with the FDA in the development and review of the product candidate, potentially leading to faster drug approval.
"The amyloidosis community has long hoped for a drug or treatment that would remove the amyloid deposits from the involved organs," said Muriel Finkel, President of the Amyloidosis Support Groups Inc. "Prothena's published results, and those recently presented, as well as excitement generated from amyloidosis patients, provide us hope that NEOD001 may offer us this very solution for our AL amyloidosis patients."
"We believe this progressive disease is significantly underdiagnosed and often misdiagnosed," commented Isabelle Lousada, Board Chairman of the Amyloidosis Foundation. "Until now, there have been no therapeutics developed specifically to treat patients with AL amyloidosis. Treatment has been limited to unapproved use of chemotherapeutic agents, which do not address the underlying cause of the disease and may have significant side effects. We welcome Prothena's dedication to pursuing a safe and effective therapy, and are encouraged by the safety profile of NEOD001 and the organ response rate results seen to date."
VITAL Phase 3 Registrational Trial Design -- The international, 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. The trial allows for an interim analysis to assess the primary endpoint for efficacy and futility.
"In addition to Fast Track, we have previously received orphan drug designation in both the U.S. and EU for AL amyloidosis. Collectively, this recognition of NEOD001 underscores the critical need for a disease-modifying immunotherapy specifically designed to treat patients with AL amyloidosis," said Gene Kinney, PhD, Chief Scientific Officer and Head of Research and Development of Prothena. "We are delighted to have recently initiated our VITAL Phase 3 clinical trial for NEOD001 in patients with AL amyloidosis. We will continue to work closely with the FDA and European regulatory authorities to bring this therapy to patients in the most expeditious manner possible."
Lipocine Inc. (NASDAQ: LPCN) 5.3%, a specialty pharmaceutical company, today announced that it has received confirmation from the U.S. Food and Drug Administration ("FDA") that the design of its ongoing pivotal Study of Oral Androgen Replacement ("SOAR") Phase 3 clinical study (http://clinicaltrials.gov/show/NCT02081300) evaluating efficacy and safety of LPCN 1021 in hypogonadal men with low testosterone ("Low T") is currently acceptable for filing a New Drug Application ("NDA") for the class testosterone replacement therapy ("TRT") labeling. The FDA reiterated the primary efficacy endpoint required for approval being 75% of subjects with a Cavg for serum testosterone in the normal range with the lower bound of the two-sided 95% confidence interval being >65%. Lipocine remains confident that our efficacy results are in line with approval requirements.
The FDA did not identify any additional clinical studies that would be required for NDA filing, but did state that should any safety signal become apparent during analysis of our SOAR Phase 3 study results or during the course of their review, it is possible that additional data may be required. Based on the response received, Lipocine does not anticipate the need to conduct additional studies above those previously agreed to with the FDA for NDA filing. The FDA also acknowledged that they are still internally discussing the advice and recommendations from the FDA Advisory Committee held on September 17, 2014.
"We are pleased that the FDA confirmed that our SOAR Phase 3 clinical study design is sufficient for filing our NDA with LPCN 1021 for the class TRT labeling. We continue to target the second half of 2015 for filing the NDA upon completion of the ongoing safety extension," said Dr. Mahesh Patel, Chairman, President and CEO of Lipocine Inc. Dr. Patel further stated, "We continue to believe that LPCN 1021 represents a 'best-in-class' TRT option with the potential to both improve patient compliance and overcome inadvertent testosterone transference risk."
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Polydex Pharmaceuticals Limited (OTC/Pink:POLXF) is pleased to announce sustained profitability for the seventh straight quarter, as a result of Management's diligent focus on cost control, capital improvements, and attention to customer service.
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 that the U.S. Food and Drug Administration (FDA) granted Fast Track designation to NEOD001, a novel monoclonal antibody for the potential treatment of AL amyloidosis.
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