THE WHY NOW BULLETS
CTIX 1) Prurisol Trial launch is close enough to bite you
2)Kevetrin Phase I results follow on the heels of Prurisol Launch
NWBO 1)Phase III for DVAX-L nearing completion
2)P3 results and NDA filing early 2014
ARWR 1)Phase 1 dosing has begun in HepB trial
2)December results could lead to Big Ph deal
ISCO 1)Recent selloff creates buying opp of a lifetime
2)Mgmt committed to solid timeline for PD trial
3)cash NOW on hand may prevent further dilution d
Reading materials for those needing fundamentals
On CTIX
Cellceutix Corporation (OTCBB: CTIX) (the "Company"), a clinical stage biopharmaceutical company focused on discovering small molecule drugs to treat unmet medical conditions, including drug-resistant cancers and autoimmune diseases, announces today that the pharmaceutical tablets for the Company's clinical trial of Prurisol as a new drug candidate for the treatment of psoriasis have been formulated by Dr. Reddy's Laboratories and are currently undergoing stability testing. The stability testing is scheduled to be completed in about 45 days.
"We have been advised that stability testing began approximately two weeks ago and that the Prurisol tablets will be shipped to the clinical site in Europe before the end of the 60-day stability research in preparation for the commencement of the clinical trial," commented Leo Ehrlich, Chief Executive Officer at Cellceutix. "We are enthusiastic about reaching another milestone for our company with the start of second clinical trial. We are very optimistic about the potential for Prurisol to reproduce the laboratory results showing the drug to effectively eliminate all signs of psoriasis."
Cellceutix also wishes to inform shareholders that it has recently conducted discussions with the University of Bologna regarding Kevetrin, the Company's novel anti-cancer drug currently in clinical trials at Dana-Farber Cancer Center and Beth Israel Deaconess Medical Center, for the planned clinical trial as a new drug candidate for Acute Myelogenous Leukemia, or AML. All regulatory submissions have been made and the hospital is awaiting Kevetrin reaching its Maximum Tolerated Dose in the trials at Dana-Farber before commencing their trial.
"We are taking a different approach to realize some of the benefits of conducting the clinical trials of both Prurisol and Kevetrin in Europe," said Dr. Krishna Menon, Chief Scientific Officer at Cellceutix. "It is a similar strategy that companies like Clovis Oncology employ to follow European protocol to target a broader, yet more specific, patient population. Through a promising pipeline and efficient development model, Clovis has built an impressive valuation. In our current trials at Dana-Farber, the protocol requires that we only treat Stage IV cancer patients, which presents its own set of unique challenges that can slow research. The good news is we are likely more than half way there in terms of the trial, and we are still increasing dosage. We are very pleased with the results to date especially considering the lower doses to date. With reference to the planned University of Bologna trial, by utilizing protocol where a 'measurable stage' of the disease is not required for the patient, i.e., we are not limiting the trial to Stage IV patients, this should allow the trial to go much faster, and hopefully get that perfect 'poster
On NWBO
BETHESDA, Md., May 16, 2013 /PRNewswire/ -- Northwest Biotherapeutics ( NWBO ) (NW Bio), a biotechnology company developing DCVax ® -L personalized immune therapies for solid tumor cancers, today announced that its Phase III clinical trial with DCVax ® -L for brain cancer has been initiated at King's College Hospital in the UK.
This is one of the first late-stage clinical trials in Europe with active immune therapies, and its opening is the culmination of years of planning, development and regulatory and institutional approvals. King's College Hospital, a major center for neuro-oncology, is leading the way for the many other European sites. Three other sites in the UK are also preparing to open, and nearly 20 sites in Germany are in various stages of preparation. In addition, medical centers in other European countries have requested to be added to the trial.
This Phase III trial is for newly diagnosed Glioblastoma multiforme (GBM), the most common and most lethal form of brain cancer. The trial is already well under way in the US, with 46 active sites at present, and is expected to enroll an aggregate total of 312 patients in the US and Europe. Notably, there are few competing brain cancer trials in Europe, although Europe has a population larger than the US.
DCVax-L mobilizes a cancer patient's whole immune system to attack the full set of biomarker targets on the tumor. Current standard of care for GBM brain cancer includes surgical removal of the tumor, followed by six weeks of daily radiation to the brain and daily chemotherapy, followed by monthly chemotherapy. With all of these treatments, GBM tumors typically recur within about 6.9 months, and the patients typically live for about 14.6 months. In prior Phase I/II clinical trials, in patients treated with DCVax-L the tumors typically did not recur for 2 years, and the patients typically lived for 3 years. A substantial percentage of patents lived even longer, and to date two patients have exceeded 10 years. In addition, DCVax-L is non-toxic and is anticipated to cost less than other recent cancer drugs.
Dr. Keyoumars Ashkan, Consultant Neurosurgeon [specialist] and Lead for Neuro-Oncology at King's College Hospital said: "We are pleased to be leading the way in bringing these novel immune therapies to patients in the UK. Brain cancers are some of the most lethal cancers, and there is a great need for new and better treatments."
"The positive data from the clinical trials in the US were very encouraging in delaying disease progression and extending survival times, without significant toxic side effects. We are hopeful that similar results will be seen in the large, randomized clinical trial which we have now launched in the UK."
"This is an important landmark, as we begin patient recruitment in our pioneering Phase III trial of DCVax-L for brain cancer in Europe, the largest medical market in the world after the US," commented Linda Powers, CEO of NW Bio. "This is one of the first late-stage clinical trials with active immune therapies in Europe, and is bringing patients a much needed new treatment option. We are excited to be launching this trial with King's College Hospital, one of Europe's premier opinion-leader institutions."
ON ARWR
PASADENA, Calif.--(BUSINESS WIRE)--
Arrowhead Research Corporation ( ARWR ), a biopharmaceutical company developing targeted RNAi therapeutics, today announced that it has initiated dosing in a Phase 1 clinical trial of ARC-520, the Company’s candidate for the treatment of chronic hepatitis B virus infection. Trial initiation followed successful completion of the Clinical Trial Notification (CTN) regulatory process in Australia. The objectives of the study are to characterize the safety profile of ARC-520, determine maximum tolerated dose, and evaluate pharmacokinetics in healthy volunteers. ARC-520 is the first candidate to use Arrowhead’s proprietary Dynamic Polyconjugate (DPC) delivery platform and includes two distinct siRNA sequences that have pan-genotypic coverage for 99.6% HBV GenBank sequences.
The Phase 1 trial 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 and is being conducted in Melbourne, Australia. Each dose cohort includes 6 subjects randomized at ratio of 1:2 (placebo:active) to receive a single intravenous injection of either placebo or ARC-520. Arrowhead expects to complete this Phase 1 trial in the fourth quarter of 2013 and begin a Phase 2a trial in chronic HBV patients in 2014.
“This Phase 1 study will establish a safety profile for ARC-520 as well as provide the first human data for our DPC delivery platform. This is an important step forward as we seek to advance ARC-520 into HBV patients and build additional RNAi therapeutics based on what we believe is the most potent delivery system in the industry,” said Dr. Christopher Anzalone, President and Chief Executive Officer.
Hepatitis B is the world’s most common serious liver infection. It is estimated that 350 million people worldwide are chronically infected with HBV, representing approximately 1 in 20 people on the planet. No currently available treatment methods can reliably achieve meaningful cure rates. ARC-520 is designed to reduce the production of new viral particles and viral proteins. Many experts believe that reducing key viral proteins can revive patients’ adaptive immune response and potentially lead to a functional cure of chronic HBV infection with a finite treatment regimen. Arrowhead previously presented data generated in rodent models and in a chimpanzee chronically infected with HBV, showing that ARC-520 induces rapid, deep, and durable knockdown of both circulating HBV DNA and key viral proteins, including hepatitis B s-antigen, e-antigen, and the core protein that forms the capsid.
About ARC-520
Approximately 350 million people worldwide are chronically infected with the hepatitis B virus. Chronic HBV infection can lead to cirrhosis of the liver and is responsible for 80% of primary liver cancers globally. Arrowhead’s RNAi-based candidate ARC-520 is designed to treat chronic HBV infection by reducing the expression and release of new viral particles and key viral proteins. The goal is to achieve a functional cure, which is an immune clearant state characterized by hepatitis B s-antigen negative serum with or without sero-conversion. The siRNAs in ARC-520 intervene at the point of DNA transcription, upstream of where nucleotide and nucleoside analogues act. In transient and transgenic mouse models of HBV infection, a single co-injection of Arrowhead’s DPC delivery vehicle with cholesterol-conjugated siRNA targeting HBV sequences resulted in multi-log knockdown of HBV RNA, proteins and viral DNA with long duration of effect. In a chimpanzee chronically infected with HBV and high viremia and antigenemia, ARC-520 induced rapid reductions of 90-95% in HBV DNA, e-antigen, and s-antigen. Arrowhead is conducting a Phase 1 single-ascending dose study in normal volunteers, which the company expects to follow with a Phase 2a study in chronic HBV patients.
On ISCO
CARLSBAD, CA--(Marketwired - May 30, 2013) - International Stem Cell Corporation ( OTCQB : ISCO ) ( www.internationalstemcell.com ) a California-based biotechnology company developing novel stem cell based therapies, announced today the initiation of its IND-enabling pharmacology and toxicology non-human primate studies, under the direction of Yale School of Medicine Professor D. Eugene Redmond Jr. MD, the internationally recognized neurosurgeon and a leader in the use of stem cells for the treatment of Parkinson's disease.
The studies will use non-human primates with moderate to severe Parkinson's disease symptoms to assess the safety and functional efficacy of ISCO's proprietary stem cell-derived neuronal cells. The endpoints of these multi-dose studies include determining cell fate, biodistribution and primate behavioral evaluations using a standardized rating scale to assess potential extrapyramidal side effects associated with the cell engraftment. The first, interim results are expected in Q4 of 2013 with the final results available in Q2 of 2014.
Leading the studies will be Prof. Redmond, a recognized expert in the use of transplanted neural cells to treat parkinsonism in primates and the development of effective replacement strategies using stem cell derived neurons. In his career of over 25 years, Prof. Redmond has made many significant contributions in the field of cellular repair in the nervous system for Parkinson's disease. His accomplishments include establishing one of the first preclinical models for Parkinson's disease in primates, the first successful transplantation of fetal tissue into the brain of a primate and one of the first clinical studies of fetal tissue in Parkinson's patients.
"Building on the results from the successful primate and rodent studies we reported earlier this year, we expect these formal studies to be the final step allowing ISCO to file an IND for Parkinson's disease," said Dr. Ruslan Semechkin, Vice President of Research and Development. "As one of the leaders in this field, it's tremendously exciting for ISCO to have such an experienced and influential clinical scientist as Prof. Redmond to direct this research. His clinical experience will be invaluable as we prepare both our IND submission to the FDA and the subsequent phase I clinical trial," Dr. Semechkin continues.
