2009.05.29 – FDA, Clinical Trial Updates- JNJ, INCY, MEDX, CYTX, AGN, PPHM



FDA, Clinical Trial Updates: JNJ, INCY, MEDX, CYTX, AGN, PPHM


By: Mike Havrilla   

May 29, 2009 8:13 AM

Sectors: Medical 



Below is a summary of updates to the BioMedReports.com database of over 200 entries included in the FDA and Clinical Trial Calendars. The FDA Calendar includes companies with pending new drug, biological agent, or medical device new product decisions at the FDA sorted by their PDUFA decision deadline dates while the Clinical Trial Calendar encompasses pending clinical trial results (with a focus on late-stage, Phase 3 trials), pending new submissions to the FDA (e.g. NDA, BLA, 510k, PMA, sNDA, sBLA filings), and pending re-submissions to the FDA for complete response rulings by the agency which require more information before an approval can be granted. 


On 5/28/09, Incyte Corp. (NASDAQ: INCY) announced that based on recent input from the FDA regarding the Company’s request for a Special Protocol Assessment (SPA) for INCB18424 for patients with primary myelofibrosis (PMF), post-polycythemia vera myelofibrosis (PPV-MF) and post-essential thrombocythemia myelofibrosis (PET-MF), it is clear that the most appropriate single primary endpoint for Incyte’s U.S. Phase 3 trial is the proportion of treated patients achieving a 35% reduction in spleen volume as compared to patients receiving placebo.


The Company provided guidance that included a modest delay in starting the Phase 3 study, and INCY expects to complete the trial in a time frame that will allow for filing a New Drug Application in late 2010 or early 2011 assuming positive results are achieved (which is still in-line with previous guidance). Incyte expects to begin enrollment of COMFORT-I (COntrolled MyeloFibrosis Study with ORal JAK Inhibitor Treatment) in August 2009. The Phase 3 European trial, COMFORT-II, is scheduled to begin enrollment in June 2009.


Johnson & Johnson (NYSE: JNJ) and Bayer (PINK: BAYRY): Xarelto (Rivaroxaban) NDA for treatment of deep vein thrombosis (DVT) + pulmonary embolism (PE). On 3/19/09, FDA Advisory Panel of outside advisers voted 15 to 2 in favor of Xarelto, but cited lingering concerns about possible liver damage and bleeding. On 5/28/09, the FDA issued a complete response letter (CRL). The FDA has not requested that any new clinical or non-clinical studies be conducted to evaluate the drug, according to the companies.


“Together with our development partner (JNJ), we are evaluating the letter and we will promptly address the FDA’s questions,” Bayer said, declining to specify what type of information was requested. The drug is already approved in Europe to prevent blood clots after major orthopedic surgery, and the two companies are also seeking clearance for the potential blockbuster in the United States, its largest market.


Xarelto was backed by an FDA advisory committee in March 2009 for use in knee and hip replacement patients despite concerns over possible side-effects and worries about long-term use.

JNJ and Medarex (NASDAQ: MEDX): On 5/26/09, JNJ announced that the FDA extended by three months the review timeline for the Biologic License Application (BLA) for Stelara (ustekinumab) to provide time for a full review of an amendment to the pending application. The application seeks approval to market Stelara for the treatment of adult patients with moderate to severe plaque psoriasis who are candidates for phototherapy or systemic therapy. The BLA amendment was submitted by JNJ within three months of the original PDUFA decision date and relates to testing results to establish the product’s shelf life.


The FDA has requested no additional clinical trials for Stelara. Medarex is set to receive UltiMAb Royalties on sales of Stelara, which was already approved in the EU in January 2009 and received a postive recommendation from a FDA Advisory panel in June 2008. In December 2008, JNJ received a Complete Response letter for Stelara from the FDA requesting additional information, including a proposal for a Risk Evaluation and Mitigation Strategy (REMS), to which the Company responded to in full in January 2009.


Peregrine Pharma (NASDAQ: PPHM): On 5/28/09, PPHM provided an update on progress in the Company’s clinical program for Cotara, a targeted monoclonal antibody-based therapy being tested in a Phase 2 trial as a potential new treatment for recurrent glioblastoma multiforme (GBM), a deadly form of brain cancer. The company also reported that patient enrollment in the final cohort of a second Cotara GBM trial, a dose confirmation and dosimetry study, is nearing completion and that interim data from this trial has been accepted for an oral presentation at the Society of Nuclear Medicine Annual Meeting to be held June 13-18, 2009. Cotara Presentation Details – Abstract Title: Dosimetry of phase I interstitial 131I-chTNT-1/B MAb (Cotara) for the treatment of recurrent glioma – Tuesday, June 16: 12:30 PM – 2:00 PM – Room 701B.


Cytori Therapeutics (NASDAQ: CYTX): On 5/27/09, CYTX announced that it completed enrollment in the first study to investigate adipose derived stem and regenerative cells in chronic heart disease. The trial, which has been named the PRECISE study, was carried out at leading cardiology centers in Europe. It specifically enrolled patients suffering from an advanced form of chronic heart disease, known as chronic myocardial ischemia, for which there is no generally accepted treatment.


The trial enrolled 27 patients and was designed as a double-blind, randomized, placebo controlled, dose escalation study. The primary objectives of the study were to assess safety and feasibility of Cytori’s Celution System as part of a novel procedure for chronic heart disease. The Company believes it will be able to fully assess these primary objectives with the data obtained from 27 patients (the protocol allowed for up to 36 patients). Further, the independent data safety and monitoring board had not identified any safety concerns relating to the Celution output or the procedure. Six month results are expected in the first half of 2010.


On 5/28/09, CYTX announced publication of the first preclinical study to demonstrate adipose-derived stem and regenerative cells (ADRCs) significantly improved cardiac function after a heart attack. The APOLLO study is the first clinical trial to investigate uncultured adipose-derived stem and regenerative cells in heart attacks in human patients. Enrollment was recently completed in APOLLO, a double blind, placebo controlled, safety and feasibility trial. Data are expected to be reported in early 2010. The APOLLO study is sponsored by Cytori and has incorporated the Company’s Celution System to automate the processing of the patients’ cells at the point of care.


My 17-page stock profile report for CYTX from mid-March 2009 is available to view or download for free at the BioMedReports.com stock research page.


Allergan (NYSE: AGN): On 5/26/09, AGN announced that it receieved a complete response letter from the FDA regarding the Company’s Supplemental Biologics License Application (sBLA) for Botox (Botulinum Toxin Type A) to treat upper limb spasticity in post-stroke adults. Allergan submitted its sBLA for this indication during 3Q08. The FDA has not requested additional pre-approval clinical studies.


However, the FDA has identified items that must be completed before the sBLA can be considered for approval, including the following: (1) Risk Evaluation and Mitigation Strategy (REMS) and Safety Update; (2) Source Data Documentation related to a pivotal clinical study conducted in 1999; and (3) product labeling revisions, including proposed revised labeling that would broaden the indication of use to include upper limb spasticity regardless of underlying cause.


Also, noting the number of pediatric patients such as those with juvenile cerebral palsy who suffer from upper limb spasticity, the FDA requested that Allergan conduct a post-approval study for BOTOX® to treat upper limb spasticity in pediatric patients 2-17 years of age. AGN estimates that the re-verification of source data documentation (issue #2 above) can be completed with an analysis submitted to the FDA in about 60-90 days.


Disclosure: Long CYTX.