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Patients Eligible for Opioid Rescue Medication to Double Under HHS Rule
April 5, 2016
The number of patients to whom physicians could prescribe opioid rescue medications would double under an HHS proposed rule.
Currently physicians only can prescribe buprenorphine and the combination of buprenorphine and naloxone to 100 patients. If they were to prescribe to more than 100 patients, they would need to register as an opioid treatment program. The proposed rule would expand that number of patients to 200.
The proposed rule also includes $11 million for states to purchase and distribute naloxone.
Bid ask 17.40pm
ACAD 12 to 2. FOR
Crap I am going to have to mow the grass again! If this would ever go to 20 I could pay someone to mow my grass. I wonder how many more times I will have to mow the grass? Over under 50.
MNOV fast track news. Small OS.
Ash FYI I am no longer a premium subscriber therefore I can no longer post on Bob's board and no PM. I do have premium over on IV. I just got tired of all the pop up adds.
Msg 47319 of 47322 at 3/20/2016 11:23:04 PM by
Rob Cos
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Med Tech Ltr-XON – FDA Okays Field Test of Anti-Zika Mosquito in Florida Keys-XON is a BUY under 42 with a TARGET PRICE of 60
(see also previously posted ZIOP Med Tech update pasted under the XON commments here):
XON – FDA Okays Field Test of Anti-Zika Mosquito in Florida Keys
The FDA’s preliminary finding stated that XON’s genetically-engineered mosquito being used in the fight against Zika will not have a significant impact on the environment. The FDA findings agree with the draft environmental assessment submitted by Oxitec, the UK unit of XON that developed the mosquito. Males of the self-limiting strain of the Aedes aegypti mosquito are modified so their offspring die before being able to reproduce. Field data from Brazil has shown an 82% reduction in the mosquito population versus at best 50% that can be achieved with heavy pesticide applications.
Zika has been linked to a spike in microcephaly, a rare birth defect, as well as Guillain-Barre syndrome, which can cause paralysis. The main mosquito that transmits Zika virus — and also dengue, chikungunya and yellow fever — is Aedes aegypti, a particularly wily and adaptable foe. It prefers urban areas and bites mainly people, making it very efficient at spreading disease. It bites in the day, so bed nets, a common way to protect people against the night-biting malaria mosquitoes, have little effect. It breeds in small containers of water, such as flower pots, cans and tires that collect rainwater.
Eighteen organizations are working on developing a vaccine for the Zika virus, but the most advanced product is still months from starting human clinical trials, and the release of a vaccine is still two to three years away at best. Health experts are also grappling with measures that could be taken to limit exposure to the mosquitoes that spread the disease. Spraying insecticides to kill mosquitoes has had no evident effect on halting the spread of dengue fever, raising concerns that it may be equally ineffectual in checking the spread of the Zika virus. Given the added benefit of not just stopping Zika but a slew of other diseases at the source, in our view, it remains just a matter of when, not if, for XON to receive substantial contracts to implement their ingenious solution, genetically engineered males that produce offspring that die before being able to reproduce. Effectively ending one generation after another of disease spreading mosquitos before they can spread disease.
XON is a BUY under 42 with a TARGET PRICE of 60
Med Tech Letter-ZIOP unlocks the potential for rapid and inexpensive personalized T-cell receptor (TCR) gene therapy. BUY ZIOP under $12 for $18 target....
ZIOP – Sleeping Beauty Published
ZioPharm has announced the publication of an article describing the use of its truly novel Sleeping Beauty non-viral gene transfer technology, that modifies T cells for the targeting of neoantigens present within solid tumors.This approach unlocks the potential for rapid and inexpensive personalized T-cell receptor (TCR) gene therapy aimed at the unique mutations within a patient’s cancer cells.
The article, titled “Stable, non-viral expression of mutated tumor neoantigen-specific T-cell receptors using the Sleeping Beauty transposon/transposase system,” was published in the journal Molecular Therapy (5 March 2016, doi:10.1038/mt.2016.51). “The DNA plasmids from the Sleeping Beautyplatform provide a customizable solution to personalized TCR gene therapy, which is needed to open the door to safely target solid tumors,” said Laurence Cooper, M.D., Ph.D., Chief Executive Officer of ZioPharm and anauthor of the paper. “There is growing consensus that a non-viral approach to gene therapy will be essential to targeting individual neoantigens. In Sleeping Beauty, we have the only clinically validated non-viral gene transfer platform. This compels us to bring this important technology to patients.”
Neoantigens unique to each patient’s tumor can be recognized by autologous T cells through their T-cell receptors, but the low frequency and/or terminal differentiation of mutation-specific T cells may limit their utility as adoptive T-cell therapies.
The publication describes how the Sleeping Beauty platform can be used to transfer and stably express TCR genes into T cells while retaining their proliferative potential. The Sleeping Beauty gene transfer occurs without the need for cell division thus facilitating the genetic modification of minimally-differentiated T cells. The authors note that the Sleeping Beautyplatform“can facilitate the use of personalized T cell therapy targeting unique neoantigens.”
The Sleeping Beauty transposon-transposase system is one more piece to the puzzle of providing off the shelf personalized T-cell therapies for not just blood cancers but also solid tumors. The technology was exclusively licensed by ZIOP/XON through the University of Texas MD Anderson Cancer Center where Dr. Cooper used to work before following his technology to ZIOP. The Company has an advantage in targeting neoantigens, which will figure importantly in therapies for solid tumors. These antigens are patient- and tumor specific proteins that are not found in normal cells andSleeping Beauty facilitates the creation of T cell receptors against such aberrant proteins. Adding the best gene therapy on/off switch, RheoSwitch, with Sleeping Beauty and it is easier to see why we believe Dr. Cooper and ZIOP are leading the development of the next generation of personalized cancer medicines.
ZIOP is a BUY under 12 with a TARGET PRICE of 18
TBIO news out.
SAN DIEGO, March 18, 2016 (GLOBE NEWSWIRE) -- Otonomy, Inc. (OTIC), a biopharmaceutical company focused on the development and commercialization of innovative therapeutics for diseases and disorders of the ear, today announced the publication of the positive results of the Phase 3 clinical trials of OTIPRIO™ (ciprofloxacin otic suspension) in JAMA Otolaryngology – Head & Neck Surgery. The trial results are from two identically designed, prospective, double-blind, sham-controlled, multicenter Phase 3 randomized clinical trials of OTIPRIO in a total of 532 pediatric patients undergoing tympanostomy tube placement (TTP).
“These trials demonstrated the safety and efficacy of a single administration of OTIPRIO in pediatric patients for the treatment of bilateral middle ear effusion at the time of ear tube placement,” said Eric A. Mair, M.D., of Charlotte, Eye, Ear, Nose, & Throat Associates. “Administration of a single dose of OTIPRIO by the ENT during the procedure provides treatment with assured compliance.”
“I would like to sincerely thank the patients, their families and the physicians who participated in the OTIPRIO clinical trials,” said David A. Weber, Ph.D., president and CEO of Otonomy. “We are extremely pleased that JAMA Otolaryngology – Head & Neck Surgery has chosen to publish these data in their journal. The commercial launch of OTIPRIO is now underway in the U.S. and the publication of these results in a well-respected, peer reviewed journal provides broad access for physicians to the Phase 3 clinical data.”
The two Phase 3 trials enrolled a total of 532 patients at approximately 50 centers in the U.S. and Canada. Pediatric patients with bilateral middle ear effusion on the day of surgery were randomized to receive TTP alone or TTP with a single intraoperative administration of OTIPRIO. The primary efficacy endpoint was treatment failure defined as the presence of otorrhea, use of otic or systemic antibiotics, loss to follow-up or missed visits through Day 15. The cumulative proportion of treatment failures was 24.6% in Trial 1 and 21.3% in Trial 2 in the OTIPRIO treatment groups, compared to 44.8% in Trial 1 and 45.5% in Trial 2 in the TTP alone groups, with p<0.001 for each trial. The most frequently reported adverse events in both trials were nasopharyngitis, irritability, and rhinorrhea. No drug-related serious adverse events were seen. No evidence of increased tube occlusion and no negative effect on results of audiometry, tympanometry, or otoscopy were noted with OTIPRIO administration.
About OTIPRIO
OTIPRIO (ciprofloxacin otic suspension) 6% is a fluoroquinolone antibacterial indicated for the treatment of pediatric patients with bilateral otitis media with effusion undergoing tympanostomy tube placement. OTIPRIO is administered by a physician as a single 0.1 mL (6 mg) intratympanic administration into each affected ear, following suctioning of the middle ear effusion. The thermosensitive suspension exists as a liquid at or below room temperature and gels when warmed. In two Phase 3 trials, a single intraoperative administration of OTIPRIO demonstrated a statistically significant reduction in the cumulative proportion of study treatment failures compared to tubes alone (p-value <0.001).
Important Safety Information for OTIPRIO
Contraindications: OTIPRIO is contraindicated in patients with a history of hypersensitivity to ciprofloxacin, to other quinolones, or to any of the components of OTIPRIO.
Warnings and Precautions - Potential for Microbial Overgrowth: OTIPRIO may result in overgrowth of nonsusceptible bacteria and fungi. If such infections occur, institute alternative therapy.
Adverse Reactions: Adverse reactions (incidence at least 3%) that occurred in two Phase 3 trials with OTIPRIO vs sham were: nasopharyngitis (5% vs 4%), irritability (5% vs 3%), and rhinorrhea (3% vs 2%).
Use in Specific Populations - Pediatric Use: The safety and effectiveness of OTIPRIO in infants below six months of age have not been established.
Full prescribing information can be found at www.OTIPRIO.com.
CorMedix Inc. (NYSEMKT:CRMD) had its price target increased by stock analysts at Rodman & Renshaw from $6.50 to $7.50 in a research report issued to clients and investors on Thursday, ARN reports. The firm presently has a “buy” rating on the specialty pharmaceutical company’s stock. Rodman & Renshaw’s price target points to a potential upside of 227.51% from the stock’s current price.
Shares of CorMedix (NYSEMKT:CRMD) traded down 8.30% during mid-day trading on Thursday, reaching $2.10. 90,044 shares of the company’s stock were exchanged. CorMedix has a one year low of $1.15 and a one year high of $10.40. The company has a 50 day moving average price of $1.96 and a 200-day moving average price of $2.18. The stock’s market cap is $74.06 million.
CRMD has been the subject of several other reports. FBR & Co. initiated coverage on shares of CorMedix in a report on Thursday, March 3rd. They issued an “outperform” rating and a $7.00 price target for the company. Zacks Investment Research downgraded shares of CorMedix from a “hold” rating to a “sell” rating in a report on Tuesday. One investment analyst has rated the stock with a sell rating, one has assigned a hold rating and three have given a buy rating to the company. The company currently has an average rating of “Hold” and a consensus price target of $5.63.
CorMedix Inc (NYSEMKT:CRMD) is a pharmaceutical company that in-licenses, develops and commercializes prophylactic and therapeutic products for the treatment of infectious diseases in cardiac, renal and oncology patients. The Company has in-licensed all of the products in its pipeline.
That is PON. All US studies show complete. At least one European study is still ongoing.
Watch IDRA.
Orbimed is a large holder. CAMBRIDGE, Mass., March 17, 2016 (GLOBE NEWSWIRE) -- Dimension Therapeutics, Inc. (DMTX), a biopharmaceutical company advancing novel, liver-directed treatments for diverse rare diseases based on an adeno-associated virus (AAV) gene delivery technology, today announced that the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) has issued a positive opinion recommending DTX301 for designation as an orphan medicinal product for the treatment of Ornithine Transcarbamylase (OTC) Deficiency. Designed to address the underlying genetic defect, DTX301 delivers stable expression and activity of OTC. DTX301 has been shown in preclinical studies to normalize levels of urinary orotic acid, a marker of ammonia metabolism, which if elevated, can cause serious neurological deficiencies in patients. Dimension has completed candidate selection in its DTX301 program and plans to submit an investigational new drug (IND) application with the FDA in the second half of 2016.
“The COMP positive opinion for DX301 in the EU, as well as recent orphan drug designation in the US, underscores the compelling need for new treatments for patients living with OTC deficiency. Current medical options are not curative and unfortunately do not eliminate the risk of metabolic crises from OTC deficiency. The DTX301 approach is designed to address these challenges and offer a new option with improved outcomes for patients," said Annalisa Jenkins, MBBS, MRCP, Chief Executive Officer of Dimension. “We are making significant progress on growing and advancing our unique pipeline of programs addressing the inherited metabolic diseases associated with the liver. The planned submission of the IND for DTX301 later this year will be another important milestone for our company and our goal to transform the care of patients living with these devastating diseases.”
The COMP issues an opinion on the granting of orphan drug designation, after which the opinion is submitted to the European Commission (EC) for endorsement of the opinion. Orphan drug designation by the EC provides regulatory and financial incentives for companies to develop and market therapies that treat a life-threatening or chronically debilitating condition affecting no more than five in 10,000 persons in the European Union (EU), and where no satisfactory treatment is available. In addition to a 10-year period of marketing exclusivity in the EU after product approval, orphan drug designation provides incentives for companies seeking protocol assistance from the EMA during the product development phase, and direct access to the centralized authorization procedure.
About DTX301
Dimension is developing its AAV gene therapy product DTX301 for the treatment of individuals with OTC deficiency. DTX301 is designed to deliver Ornithine Transcarbamylase gene expression in a durable fashion, preventing the complications associated with OTC deficiency. Preclinical studies completed to date indicate DTX301 has the potential to be a well-tolerated, effective therapy for OTC deficiency.
Background on OTC Deficiency
OTC deficiency, the most common urea cycle disorder, is caused by a genetic defect in a liver enzyme responsible for detoxification of ammonia. Individuals with OTC deficiency can build-up excessive levels of ammonia in their blood, potentially resulting in neurological deficits and other toxicities. It is estimated that more than 10,000 patients are affected by OTC deficiency worldwide, of which approximately 80% are classified as late-onset, Dimension’s target population. The greatest percentage of patients, including males and females, experience late-onset disease, representing a clinical spectrum of disease severity. Neonatal onset disease occurs in males, presents as severe disease, and can be fatal at an early age. Approved therapies, which must be taken multiple times a day for the patient's entire life, do not eliminate the risk of future metabolic crises. Currently, the only curative approach is liver transplantation.
Maybe no word on PONEZUMAB is good?
UPDATED: Axovant routed after Pfizer flags failure of rival Alzheimer's drug
February 2, 2016 | By John Carroll
Pfizer ($PFE) has quietly shuttered a Phase II Alzheimer's drug study, noting the termination in a pipeline update released today with its annual numbers for 2015. And shares of Axovant ($AXON) tumbled 25% on the news as it faces fresh questions about the implications of the failure on a similar drug it has pushed into a Phase III trial.
According to clinicaltrials.gov, Pfizer--which has invested heavily in Alzheimer's R&D over the years--opted to end the study of PF-05212377 on Oct. 23 based on futility, or a conclusion that the drug would fail the key endpoint laid out for it. The move puts the spotlight on the drug's mechanism of action, raising fresh questions about the likely efficacy of other drugs that try to safeguard cognitive abilities by targeting the serotonin 6 receptor (5-HT6).
"We decided to terminate the Phase II study (B2081011) of our investigational program PF-05212377 following an External Data Monitoring Committee recommendation after a planned unblinded review of the interim efficacy data," noted a spokesperson for Pfizer in a response to a query from FierceBiotech. "We're currently evaluating next steps for this program, including plans for publishing the results of this trial."
GBIM from IV.Msg 97 of 97 at 3/16/2016 10:03:12 AM by
wbart21
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In response to msg 96 by buygene view thread
Re: GlobeImmune Announces Updates and Financial Results for Full Year 2015
$9.9mm is about $1mm more than i thought. One less scientist,so only 3 employees w/ CEO taking 50% of salary= minimal burn going forward and most importantly, GILD has agreed to release 24week primary endpoint data on GS-4774 in Q2 ! Can't believe GILD would make a go no-go decision in Q2 rather than awaiting the full 48 week data set.
Obviously the million dollar question is.... is the move up of 24 week data to Q2 an attempt by gbim(GILD obviously had to agree)to search for some evidence of effect @ 24 weeks to drive a better strategic transaction and save the company or because this is an OLS do gild/gbim already have some good 24 data points in hand. As JT pointed out on twtr, EASL is a mere 4 weeks away. Stock is an easy hold here,irrespective , as they have almost $10mm cash, 3 employees, still retain Cantor and if 4774 fails, they still have CELG MTC P2 readout later this year and the Chordoma trial w/ CELG/NCR- so 2 more shots on goal ;)
AbstractSend to:
J Neuropathol Exp Neurol. 2015 Dec 15. pii: nlv001. [Epub ahead of print]
Characterization of Early Pathological Tau Conformations and Phosphorylation in Chronic Traumatic Encephalopathy.
Kanaan NM1, Cox K2, Alvarez VE2, Stein TD2, Poncil S2, McKee AC2.
Author information
Abstract
Chronic traumatic encephalopathy (CTE) is a neurodegenerative tauopathy that develops after repetitive head injury. Several lines of evidence in other tauopathies suggest that tau oligomer formation induces neurotoxicity and that tau oligomer-mediated neurotoxicity involves induction of axonal dysfunction through exposure of an N-terminal motif in tau, the phosphatase-activating domain (PAD). Additionally, phosphorylation at serine 422 in tau occurs early and correlates with cognitive decline in patients with Alzheimer disease (AD). We performed immunohistochemistry and immunofluorescence on fixed brain sections and biochemical analysis of fresh brain extracts to characterize the presence of PAD-exposed tau (TNT1 antibody), tau oligomers (TOC1 antibody), tau phosphorylated at S422 (pS422 antibody), and tau truncated at D421 (TauC3 antibody) in the brains of 9-11 cases with CTE and cases of nondemented aged controls and AD (Braak VI) (n?=?6, each). All 3 early tau markers (ie, TNT1, TOC1, and pS422) were present in CTE and displayed extensive colocalization in perivascular tau lesions that are considered diagnostic for CTE. Notably, the TauC3 epitope, which is abundant in AD, was relatively sparse in CTE. Together, these results provide the first description of PAD exposure, TOC1 reactive oligomers, phosphorylation of S422, and TauC3 truncation in the tau pathology of CTE.
© 2015 American Association of Neuropathologists, Inc. All rights reserved.
KEYWORDS:
Alzheimer disease; Chronic traumatic encephalopathy; Dementia; Oligomers; Phosphatase-activating domain; Tauopathy; Traumatic brain injury.
PMID: 26671985 [PubMed - as supplied by publisher]
Any of you day traders may out on this. Could have got over 100%.
GlobeImmune Announces Updates and Financial Results for Full Year 2015
March 16, 2016 06:30 ET
GlobeImmune Announces Updates and Financial Results for Full Year 2015
LOUISVILLE, CO--(Marketwired - March 16, 2016) - GlobeImmune, Inc. (NASDAQ: GBIM) today provided an update on the Company's business and clinical programs and announced financial results for the full year ended December 31, 2015.
The Company continues to seek potential strategic transactions and, after a thorough process, has not identified a potential transaction to date. Cantor Fitzgerald & Co. has been retained by the Company to assist in reviewing ways to maximize stockholder value. There is not a defined timeline for the strategic review process and the review may not result in any specific action or transaction.
The Company has three ongoing clinical trials being conducted by the Company's corporate collaborators, Gilead Sciences, Inc. and Celgene Corporation. GS-US-330-1401, the GS-4774 Phase 2 clinical trial in patients with chronic HBV infection who are currently not receiving treatment, is fully enrolled and the 24-week results are projected to be available in the second quarter of 2016. The results from a GI-6207 Phase 2 trial in subjects with medullary thyroid cancer are projected to be available in the second half of 2016. A Phase 2 clinical trial designed to investigate the safety and efficacy of evaluating GI-6301 in combination with radiation therapy in patients with chordoma is still enrolling patients.
Financial Results - Fiscal Year 2015
For the full year ended December 31, 2015, GlobeImmune reported a net loss of $2.8 million compared to $16.3 million in 2014. The decrease in net loss for 2015 was due to $1.8 million of revenue from the license of the GI-6200 program to Celgene, lower research and development expenses due to lower compensation expense, as well as no interest expense, early retirement expense associated with convertible notes and no fair-value adjustments of warrants as these costs terminated upon the closing of the Company's initial public offering in July 2014. GlobeImmune reported a loss applicable to common stockholders of $2.8 million, or $0.48 per share, for the year ended December 31, 2015 compared to loss applicable to common stockholders of $23.4 million, or $8.04 per share, in 2014.
Research and development for proprietary programs expense for the year ended December 31, 2015 was $1.8 million compared to $2.2 million for the year ended December 31, 2014, a decrease of $0.4 million. The decrease was primarily due to a reduction in expenses related to the GI-4000 program and a decrease in salary expense due to layoffs during the third quarter. Costs of manufacturing services for 2015 were $0.3 million compared to $1.5 million in 2014. The decrease was primarily due to a decrease in expenses relating to manufacturing services for Gilead for the Phase 2 HBV trial. Costs of collaboration license and services for the year ended December 31, 2015 was $2.4 million compared to $3.5 million for the year ended December 31, 2014, a decrease of $1.1 million. The decrease was primarily due to a decrease in expenses related to the Phase 1 clinical trial for GS-4774 and reduction in salary expense due to layoffs during the third quarter. General and administrative expense for the year ended December 31, 2015 was $4.6 million compared to $4.3 million for the year ended December 31, 2014, an increase of $0.3 million. The increase was related to expenses associated with being a public company for a full year ended December 31, 2015 offset by lower travel expenses and gain on sale of property and equipment.
At December 31, 2015, GlobeImmune had cash and equivalents of $9.9 million. The Company's major financial obligation is the remaining balance on its lease. Net of the total payments due over the remaining 36 months of the lease obligation, the Company believes it has sufficient cash to operate the company through the end of 2016 as it continues to evaluate strategic alternatives.
10q is out. Looks pretty good for the small OS.
Who has been buying the last few days?
NEW HAVEN, Conn., March 16, 2016 (GLOBE NEWSWIRE) -- BioBlast Pharma Ltd. (ORPN), a clinical-stage, orphan disease-focused biotechnology company, announced positive results from its HOPEMD Phase 2 six-month open-label clinical study in patients with oculopharyngeal muscular dystrophy (OPMD), a rare progressive muscle-wasting disease characterized by swallowing difficulties (dysphagia), leading to the risk of aspiration of food into the lungs, weight loss, and generalized progressive muscle weakness. These results will be presented today by Prof. Zohar Argov M.D., Senior Medical Advisor to BioBlast, during the plenary session at Myology 2016, a leading muscle conference in Lyon, France, and, in April, 2016, during two general sessions at the American Academy of Neurology in Vancouver, Canada. See information in this release regarding these scientific symposia.
The primary objective of the HOPEMD Phase 2 open-label study was to assess the safety and tolerability of trehalose 90mg/mL IV solution. Although not powered for efficacy, secondary endpoints were included to explore if trehalose 90mg/mL IV solution could improve or prevent worsening of OPMD disease markers. The study enrolled 25 patients with clinical dysphagia and muscle weakness at two centers in Canada and Israel. All patients have now completed 24 weeks of weekly treatment with trehalose 90mg/mL IV solution and these are the data presented today.
Trehalose 90mg/mL IV solution was observed to be safe and well-tolerated with no drug-related serious adverse events. Improvements versus baseline were observed in multiple secondary efficacy endpoints related to dysphagia and muscle strength and function as further discussed below.
“The final study results are consistent with the interim data previously reported and continue to look promising,” stated Bernard Brais, MD, M.Phil., PhD, FRCP(C), Professor, Departments of Neurology and Neurosurgery and Human Genetics, Faculty of Medicine, McGill University, Co-director Rare Neurological Diseases Group at the Montreal Neurological Institute, and Principal Investigator in the study. “With no pharmacotherapy available to treat OPMD, trehalose 90mg/mL IV solution may be a step forward in caring for these patients. We look forward to confirming these results in the planned Phase 2b double blind placebo controlled study.”
HOPEMD Phase 2 Open-Label Clinical Study - Results
Safety and Tolerability – Trehalose 90mg/mL IV solution was observed to be safe and well tolerated with no drug-related serious adverse events reported. There were no significant changes in lab safety data including chemistry, hematology, and ECG tests. There was one death due to aspiration pneumonia that was not considered drug-related but instead, related to the underlying disease. No patients chose to discontinue the study for reasons related to safety or side effects.
Dysphagia Tests (swallowing difficulties) – The dysphagia endpoints were the timed cold water drinking test (80mL) for all sites, the nectar (80mL) and honey (80mL) timed drinking tests at the Canadian site and the Penetration Aspiration Score as measured by video fluoroscopy (VFS-PAS), a radiographic technique to determine the severity of swallowing difficulties and risk of aspiration. A patient reported swallowing quality of life questionnaire (SWAL-QOL) specifically developed for patients suffering from swallowing problems was employed to assess the degree to which patients felt that their swallowing capability improved with treatment.
There was a mean reduction in time to complete the cold water drinking test of 31.8% versus baseline (n=23). In the nectar and honey timed drinking tests, time to complete was reduced by 43.8% and 46.6% respectively (n=11). Out of the 11 patients in Canada whose scores were evaluated in the per protocol analysis of the VFS-PAS, six patients improved (54.5%), two patients showed stabilization (18.2%), and three patients deteriorated (27.2%). As previously reported, due to deviations from protocol and deficient radiological procedures, the VFS-PAS tests from the Israel cohort were excluded from the final analysis. With respect to the SWAL-QOL questionnaire, there was a 12.7% (n=24) improvement versus baseline with the mean total symptom severity score increasing from 43.2 to 48.7.
Muscle Strength Tests – As measured quantitatively by a digital hand-held dynamometer, there was a mean increase in lower body muscle strength compared to baseline in knee extension of 15.0% (n=22) and foot dorsiflexion of 22.4% (n=22). Hip flexion did not materially change (1.3% deterioration, n=21). For the upper extremity strength tests, arm (bicep) flexion increased on average 17.9% (n=22), and shoulder abduction by 11.4% (n=22).
Muscle Function Tests – The 30 second arm-lift test showed a 16.0% increase in the number of completed tasks (n=20) at 24 weeks of treatment versus baseline while the 30 second sit-to-stand test showed a 16.6% increase (n=21). The standard 4-stair climbing test did not materially change (1.5% deterioration, n=21).
“We are encouraged by the safety profile observed and the early efficacy signals noted in this relatively small study of OPMD patients. With this knowledge, we can proceed to develop our double blind placebo controlled study to confirm these results and progress our clinical program to registration,” stated Warren Wasiewski M.D., Chief Medical Officer of BioBlast.
“These positive safety and efficacy signals give us confidence to continue the development of trehalose 90mg/mL IV solution in OPMD,” said Colin Foster, President and Chief Executive Officer of BioBlast. “We look forward to the possibility of significantly helping patients afflicted with this disease.”
HOPEMD Phase 2 Open-Label Clinical Study - Design
The HOPEMD study was designed as an initial proof-of-concept open-label clinical study in 74 patients for 24 weeks, following which it was intended that all patients would be randomized into a treatment arm or non-treatment control group, and followed for an additional 12 months in a separate continuation study. The study was conducted at two centers - Montreal Neurological Institute at McGill University in Montreal, Canada, and Hadassah-Hebrew University Medical Center in Jerusalem, Israel. The primary objective was to assess the safety and tolerability of trehalose 90mg/mL IV solution in patients suffering from OPMD. Although not powered for efficacy, secondary endpoints were included to explore if trehalose 90mg/mL IV solution could improve or prevent worsening of OPMD disease markers, notably those related to dysphagia (difficulty in swallowing) and upper and lower muscle weakness. As previously reported, based on the interim efficacy signals seen in the first 25 patients enrolled in Canada (11 patients) and Israel (14 patients), recruitment was terminated in mid-2015. Now that all patients have received at least 24 weeks of treatment, BioBlast intends to discontinue the ongoing continuation study and offer patients access to the drug in an expanded access program.
Scientific Symposia Information
Final results of the HOPEMD Phase 2 open-label study will be presented at two upcoming scientific neurology conferences:
1. 5th International Congress of Myology 2016, March 14-18, 2016 in Lyon, France
Plenary Session – Pharmacotherapy, March 16, 2016 ~ 2:30pm-4:00pm
Presenter: Prof. Zohar Argov M.D. (Jerusalem, Israel)
Title: Intravenous Trehalose for Treatment of Dysphagia and Muscle Function in Oculopharyngeal Muscular Dystrophy (OPMD): Final Results of 24 Week Open-Label Phase 2 Trial
2. American Academy of Neurology 2016 Annual Meeting, April 15-21 in Vancouver, Canada
Presentations:
• Integrated Neuroscience Session – Advances in Acquired and Genetic Muscle Diseases, April 17, 2016 ~ 5:00pm
• Experimental Therapies in Neuromuscular Diseases, April 19, 2016 ~ 7:15am
Presenter: Prof. Zohar Argov M.D. (Jerusalem, Israel)
Title: Intravenous Trehalose for Treatment of Dysphagia and Muscle Function in Oculopharyngeal Muscular Dystrophy (OPMD): Final Results of 24 Week Open-Label Phase 2 Trial
About Trehalose 90mg/mL IV Solution
Trehalose 90mg/mL IV solution is a chemical chaperone that protects against pathological processes in cells. It has been shown to reduce pathological aggregation of proteins within cells in several diseases associated with abnormal cellular-protein aggregation as well as acting as an autophagy enhancer. Trehalose 90mg/mL IV solution has been documented as demonstrating significant promise in preclinical animal models of OPMD and other PolyA/PolyQ diseases.
In OPMD, trehalose 90mg/mL IV solution is being developed to prevent the aggregation of the pathological protein (PABPN1) in muscle cells, the hallmark of the disease, by stabilizing the protein, reducing the formation of protein aggregations, and promoting their clearance from cells through autophagy, thus preventing muscle cell death.
About Oculopharyngeal Muscular Dystrophy (OPMD)
OPMD is an inherited myopathy characterized by dysphagia (difficulty in swallowing), eyelid drooping (ptosis), the loss of muscle strength, and weakness in multiple muscles of the body. Symptoms generally appear in mid-life and get worse over time. As the dysphagia becomes more severe, patients become malnourished, lose significant weight, and may suffer from repeated incidents of aspiration pneumonia. Aspiration pneumonia and severe emaciation may result in death. The disease is caused by a genetic mutation responsible for the creation of a mutant protein (PABPN1) with an expanded polyalanine domain that aggregates within patient muscle cells. There is currently no approved pharmacologic treatment for OPMD.
REDWOOD CITY, Calif., March 16, 2016 (GLOBE NEWSWIRE) -- Capnia, Inc. (CAPN), a diversified healthcare company that develops and commercializes innovative diagnostics, devices and therapeutics addressing unmet medical needs, today announced its entry into an exclusive international five-year distribution agreement with Shanghai CiiC Science and Technology Development Company (Shanghai CiiC) for the sale of its CoSense End-Tidal Carbon Monoxide Monitor and Precision Sampling Sets in China.
Under the terms of the agreement, Shanghai CiiC will have the exclusive right for sales, marketing, distribution and field service activities for CoSense in China. Shanghai CiiC, at its expense, will secure and maintain all registration and approvals for CoSense in China. CoSense sales are expected to commence by the end of 2016, subject to Shanghai CiiC securing all necessary regulatory approvals.
“This agreement with Shanghai CiiC allows us to pursue a rapid path to the China market, potentially one of the largest opportunities in the world for CoSense with 16 million births every year. This long-term partnership will provide synergies with respect to marketing authorization, sales, marketing and distribution, which will add significant value for CoSense,” said Anish Bhatnagar, MD, Chief Executive Officer of Capnia. “In China, Shanghai CiiC is known for technological innovation in both healthcare and medical devices and they are well equipped to bring CoSense to the market in this important global territory.”
Wenan Zhu, General Manager for Shanghai CiiC stated, “We are extremely excited to pursue this arrangement with Capnia. Shanghai CiiC is well positioned to bring CoSense to market in China, the world’s largest healthcare market. Our joint effort will facilitate the approval and commercial launch in China, thereby providing physicians with a better tool to detect hemolysis and manage newborns with jaundice. Hemolytic disease is prevalent in our country, and we anticipate that CoSense will become a key technology for aiding in the early detection of hemolysis.”
Shanghai CiiC specializes in the import and export of the latest technological applications, including healthcare and medical devices and they are currently commercializing other novel devices in the field of neonatology. They are the largest and most profitable subsidiary of CiiC, which is one of the state-owned backbone enterprises under the direct management of the Chinese Central Government.
Xtant Medical Holdings, Inc.
12 hours ago
GlobeNewswire
See footnote about the use of pro forma financial information
Fourth Quarter 2015 Highlights:
Consolidated total revenue increased 14.1% to a record $22.3 million compared to pro forma fourth quarter 2014 revenue of $19.5 million
Consolidated gross profit increased 14.9% to $14.9 million compared to pro forma fourth quarter 2014 gross profit of $13.0 million
Consolidated gross margins improved to 67.0%, compared to 66.5% reported in the fourth quarter of 2014
Consolidated net income for the quarter improved to $11.6 million compared to a pro forma net loss of $5.0 million in the fourth quarter of 2014
Consolidated earnings per share improved to $0.97 compared to pro forma loss per share of $0.35 in the fourth quarter of 2014
Full-Year 2015 Highlights:
Pro forma total revenue increased 11.4% to $86.5 million compared to pro forma 2014 revenue of $77.7 million
Pro forma gross profit increased 10.5% to $56.6 million compared to pro forma 2014 gross profit of $51.2 million
Pro forma gross margins for the year were 65.4%, which were slightly lower than the previous year
Pro forma net loss for the year improved to $5.8 million compared to pro forma net loss of $15.6 million in 2014
BELGRADE, Mont., March 15, 2016 (GLOBE NEWSWIRE) -- Xtant™ Medical Holdings, Inc. (XTNT), a leader in the development of regenerative medicine products and medical devices, today reported its financial results for the period ended December 31st, 2015. On a pro forma basis, the Company reported annual revenues of approximately $86.5 million and a net loss for the year of approximately $5.8 million.
Revenue
Consolidated fourth quarter 2015 revenue was approximately $22.3 million, an increase of 14.1% compared to pro forma revenue of approximately $19.5 million for the same period during 2014.
Pro forma revenue for the full year 2015 was approximately $86.5 million, compared to pro forma 2014 revenue of approximately $77.7 million, representing an increase of 11.4% over the prior year. Excluding revenue associated with an Original Equipment Manufacturer (OEM) customer, pro forma full year 2015 revenue increased 14.8% to $81.4 million.
Stated in 000's
FY '14 FY '15
Pro Forma Revenue $ 77,651 $ 86,518
Growth 11.4 %
Pro Forma OEM Revenue $ 6,751 $ 5,093
Pro Forma Revenue Excluding OEM $ 70,900 $ 81,425
Growth 14.8 %
Gross Profit
Consolidated gross profit for the fourth quarter of 2015 was $14.9 million or 67.0% of revenues, compared to pro forma gross profit of $13.0 million or 66.5% of revenues for the fourth quarter of 2014.
For the year, pro forma gross profit was approximately $56.6 million, compared with pro forma 2014 gross profit of $51.2 million. Pro forma gross margin for the year was 65.4%, slightly lower than 2014 pro forma gross margin of 66.0%
Sales and Marketing Expenses
Consolidated fourth quarter 2015 sales and marketing expenses increased to $10.6 million, as compared to pro forma sales and marketing expenses of $9.3 million during the same period in 2014. For the quarter, sales and marketing as a percentage of revenues increased slightly to 47.6%, compared to 47.4% in the fourth quarter of 2014, on a pro forma basis.
Pro forma 2015 sales and marketing expenses increased to $39.3 million, as compared to pro forma 2014 sales and marketing expenses of $33.3 million. As a percentage of revenues, sales and marketing expenses increased to 45.5% compared to 42.9% reported for the full year 2014. The increase was mainly due to the Company's planned and strategic expansion of its salesforce, combined with its acquisition of X-spine during the third quarter of 2015.
General and Administrative Expenses
In the fourth quarter, consolidated general and administrative expenses increased to $4.2 million as compared to pro forma general and administrative expenses of $3.3 million reported for the same period last year. As a percentage of revenues, general and administrative expenses were 18.9% during the period as compared to 16.7% for the same period during 2014. The increase in general and administrative expenses was primarily attributable to increased headcount for the X-spine subsidiary.
Pro forma 2015 general and administrative expenses increased to $16.6 million as compared with $13.9 million reported for the same pro forma period last year. As a percentage of revenues, general and administrative expenses were 19.2% as compared to 17.9% for 2014.
Net Income / Loss
The fourth quarter 2015 consolidated net income was $11.6 million, compared to the pro forma year-ago period net loss of $5.0 million. The increase was primarily due to a one-time recording of a deferred tax benefit of $17.5 million. Consolidated earnings per share improved to $0.97 compared to pro forma loss per share of $0.35 in the fourth quarter of 2014.
For the full-year 2015, the Company had a pro forma net loss of $5.8 million compared to a pro forma net loss of $15.6 million for 2014. Pro forma net loss per share for the full-year 2015 was $0.65 a share compared to pro forma net loss per share of $1.11 for 2014.
EBITDA
The Company defines earnings before interest, taxes, depreciation and amortization ("EBITDA") as net income/loss from operations before depreciation, amortization, impairment charges, non-recurring expenses and non-cash stock-based compensation. Consolidated EBITDA for the fourth quarter of 2015 was a loss of $350,000 compared to a pro forma gain of $403,000 for the same period during 2014. The unfavorable change in EBITDA was due to higher sales and marketing and general and administrative expenses.
Full year 2015 pro forma EBITDA was a loss of $33,000 compared to a pro forma gain of $3.9 million in the prior year.
Financial Liquidity
Cash on hand as of December 31, 2015, was $6.4 million, as compared to $4.5 million as of December 31, 2014. Net working capital as of December 31, 2015 increased $13.3 million to $23.9 million, as compared to $10.6 million as of December 31, 2014.
Outlook for Full Year 2016
The Company also provided full year 2016 revenue guidance based on the following:
Stated in 000's 2014 2015 2016 Guidance
Pro Forma Revenue $ 77,651 $ 86,518 $94,000 - $99,000
Growth 11.4 % 8.7% - 14.4%
Pro Forma Revenue $ 6,751 $ 5,093 $250 - $500
Pro Forma Revenue Excluding OEM $ 70,900 $ 81,425 $93,750 - $98,500
Growth 14.8 % 15.1% - 21.0%
EBITDA $ 3,854 $ (33 ) $4,300 - $6,300
Conference Call to be Held March 16, 2016
An accompanying conference call will be hosted by Dan Goldberger, Chief Executive Officer, and John Gandolfo, Chief Financial Officer, to discuss the results. The call will be held at 10:00 AM ET, on March 16, 2016. Please refer to the information below for conference call dial-in information and webcast registration.
Conference Date: March 16, 2016, 10:00 AM ET
Conference Dial-in: 877-269-7756
International Dial-in: 201-689-7817
Conference Call Name: Xtant Medical's Fourth Quarter 2015 Results Call
Webcast Registration: Click Here
Following the live call, a replay will be available on the Company's website, www.xtantmedical.com, under "Investor Info."
Use of Pro Forma Financial Information
On July 31, 2015, Bacterin International Holdings, Inc. acquired all of the issued and outstanding stock of X-Spine Systems, Inc. and the combined company was renamed Xtant Medical Holdings, Inc. Except for the financial results for the three months ended December 31, 2015, the results presented are on a pro forma basis as if the two companies were combined for the periods shown. Certain pro forma adjustments have been made to reflect the impact of the purchase transaction, primarily consisting of amortization of intangible assets with determinable lives and interest expense on long-term debt. In addition, certain historical expenses, such as warrant expense and interest expense associated with debt that was immediately repaid, were eliminated from these pro-forma results. The pro forma information does not necessarily reflect the actual results of operations had the acquisition been consummated at the beginning of the fiscal reporting period indicated nor is it indicative of future operating results. The pro forma information does not include any adjustment for potential revenue enhancements, cost synergies or other operating efficiencies that could result from the acquisition.
Additional information regarding the business combination and its impact on the Company’s financial position will be set forth in the Company’s Form 10-K for the fiscal year ended December 31, 2015, which will be filed with the Securities and Exchange Commission on or about March 30, 2016 and will include the Company’s audited consolidated financial statements as of and for the years ended December 31, 2015 and December 31, 2014.
About Xtant™ Medical Holdings, Inc.
Xtant Medical Holdings, Inc. (XTNT) develops, manufactures and markets class-leading regenerative medicine products and medical devices for domestic and international markets. Xtant products serve the specialized needs of orthopedic and neurological surgeons, including orthobiologics for the promotion of bone healing, implants and instrumentation for the treatment of spinal disease, tissue grafts for the treatment of orthopedic disorders, and biologics to promote healing following cranial, and foot and ankle surgeries. With core competencies in both biologic and non-biologic surgical technologies, Xtant can leverage its resources to successfully compete in global neurological and orthopedic surgery markets. For further information, please visit www.xtantmedical.com.
Important Cautions Regarding Forward-looking Statements
CRMD 8k presentation slides.
http://ih.advfn.com/p.php?pid=nmona&article=70766979&symbol=CRMD
Ponezumab study in Europe still ongoing. What do you make of the sub study?
https://www.clinicaltrialsregister.eu/ctr-search/trial/2013-001557-27/GB
Here is a name from the past. On the AVXL board.
basparks79 • 3 minutes 8 seconds ago Flag
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Expect to see Media Coverage on Anavex soon...
Reply from the media PoC to my email regarding anticipated media coverage...
"BioCentury will be covering shortly. Stay tuned! More to come!"
Jules Abraham
Principal
JQA Partners
LifeSci Capital Initiates Coverage of MediciNova
Accesswire 1 hour ago
Targeting Neurological, Fibrotic, and Respiratory Diseases with Unmet Medical Needs; Report Available here: www.lifescicapital.com/equity-research/medicinova/
NEW YORK, NY / ACCESSWIRE / March 14, 2016 / LifeSci Capital, LLC, a research-driven investment bank with deep domain expertise in the life sciences sector, today announced that it has initiated coverage of MediciNova (MNOV), a clinical-stage biopharmaceutical company developing treatments for neurological and fibrotic diseases with unmet medical needs. The Company is developing lead candidate, MN-166 (ibudilast), for primary and secondary progressive multiple sclerosis (MS), amyotrophic lateral sclerosis (ALS) and drug dependence, as well as MN-001 for non-alcoholic steatohepatitis (NASH) and idiopathic pulmonary fibrosis (IPF).
MN-166 has been approved in Japan for asthma and post-stroke dizziness since 1989, and there is a safety database containing several million patients. MN-166 has been shown to dampen the activation of microglia, which are involved in the pathogenesis of MS, as well as ALS and other neurodegenerative diseases. A Phase IIb study of MN-166 in primary and secondary progressive MS is ongoing and is fully enrolled. The study is being funded by the National Institutes of Health (NIH) and is being conducted through its NeuroNext program. NIH will conduct an interim analysis in the second half of 2016 and final results are expected in 2017.
MediciNova has also demonstrated that MN-001 produces both anti-inflammatory and anti-fibrotic effects and may be beneficial in the treatment of fibrotic diseases, such as NASH and IPF. Both of these indications represent multi-billion market opportunities. MN-001 was previously studied in a positive Phase II study in patients with asthma, which showed a favorable safety and tolerability profile. MediciNova recently launched Phase II studies for MN-001 in NASH and IPF.
In a 47 page Initiation Report LifeSci Capital describes the unmet medical needs that MediciNova is targeting, as well as the Company's clinical development plan and regulatory strategy.
Dr. Isaacson's full Initiation Report, including important disclosures, is available to download at no cost at the LifeSci Capital website, www.lifescicapital.com/equity-research/. In addition to this Initiation Report, LifeSci Capital intends to provide ongoing coverage and event-based research updates on MediciNova as developments occur.
The LifeSci Capital research team is led by Dr. Jerry Isaacson, an industry veteran with broad experience in biotechnology, having worked in both public and private biotech companies in areas ranging from medicinal chemistry and analytical chemistry to patents and investor/public relations. Dr. Isaacson holds a Bachelor of Arts degree in Chemistry from Harvard University and received his Ph.D. in Organic Chemistry from the University of California in San Diego.
About LifeSci Capital:
LifeSci Capital (Member: FINRA/SIPC) is a research-driven investment bank with deep domain expertise in the life sciences. Our service model as a boutique investment bank is unique in that we exclusively serve emerging life science companies that discover, develop, and commercialize innovative products. We view our clients as our partners, and we work closely with them to establish and execute their capital markets strategies. Our broadly-distributed equity research product is differentiated and provides a deep understanding of our clients' businesses and the opportunities they are addressing. To learn more about LifeSci Capital, visit the company's website, www.lifescicapital.com.
Analyst Contact:
Jerry Isaacson, Ph.D.
Phone: (646) 597-6991
Email: jisaacson@lifescicapital.com
SOURCE: LifeSci Capital
Ritter Pharmaceuticals Initiates Phase 2B/3 Clinical Trial of RP-G28 in Lactose Intolerance
Marketwired Ritter Pharmaceuticals, Inc. 26 minutes ago
REDWOOD CITY, Calif., March 14, 2016 (GLOBE NEWSWIRE) -- Capnia, Inc. (CAPN), a diversified healthcare company that develops and commercializes innovative diagnostics, devices and therapeutics addressing unmet medical needs, today announced that the Company has obtained re-certification of the CE Mark for Serenz, its nasal, non-inhaled CO2 product for the treatment of symptoms related to allergic rhinitis (AR or nasal allergies). CE Mark certification is a conformance mark granted by the European Commission for the sale of certain medical devices without restriction across the 28 member nations of the European Union (EU). With the CE Mark, the Company plans to move forward with pilot sales of Serenz to pharmacies in the EU during the second quarter of 2016. The Company intends to provide limited supplies of Serenz to a number of pharmacies with the intent of gathering commercial feedback on the product in preparation of a possible full launch of Serenz.
“CE Mark certification is an essential regulatory milestone as we advance toward commercialization of Serenz in Europe, and we look forward to initiating pilot sales to EU pharmacies during the second quarter of 2016,” said Anish Bhatnagar, MD, Chief Executive Officer of Capnia. “AR is typically an episodic disorder with intermittent symptoms. However, there is no treatment currently available that provides truly rapid relief of all nasal symptoms. We believe that Serenz has an ideal profile for an as-needed therapeutic for AR and may provide advantages over regularly dosed, slow to act currently marketed products.”
A CE Mark certification was previously granted to the Company in December 2011 for the marketing of Serenz in the EU.
About Serenz
Serenz is a hand-held device that is designed to provide rapid relief from the symptoms related to AR. In clinical trials to date, Serenz has shown relief of symptoms related to allergies within 30 minutes and a mild side effect profile. The Serenz technology is based upon the observation that non-inhaled CO2 delivered at a low-flow rate into the nasal cavity alleviates the symptoms related to allergies. Capnia’s nasal, non-inhaled CO2 is currently in Phase 2 development in the U.S.
Anavex Presents New Data on Preclinical Development of ANAVEX 3-71
Results Demonstrate Significant Reversal in Cognitive Deficit, Inflammation and Alzheimer’s-like Amyloid Pathology
GlobeNewswire Anavex Life Sciences Corp. 5 minutes ago
NEW YORK, March 14, 2016 (GLOBE NEWSWIRE) -- Anavex Life Sciences Corp. (“Anavex” or the “Company”) (AVXL), a clinical-stage biopharmaceutical company developing differentiated therapeutics for the treatment of neurodegenerative diseases including Alzheimer’s disease, other central nervous system (CNS) diseases, pain and various types of cancer, today announced positive preclinical data on sigma-1 agonist ANAVEX 3-71 (formerly AF710B). The results show a statistically significant reduction in the rate of cognitive deficit, amyloid beta pathology and inflammation with the administration of ANAVEX 3-71.
The poster, titled “AF710B, a concomitant activator of M1 muscarinic and sigma-1 receptors: possible disease-modifying properties in McGill-R-Thy1-APP rats,” was presented at the 14th International Symposium on Advances in Alzheimer’s Therapy (AAT) from March 9-12, 2016. The study examined the responses of ANAVEX 3-71 in aged transgenic McGill-R-Thy1-APP rats and wild-type rats dosed with 10 micrograms/kg/day ANAVEX 3-71. Both types of rats (12-14 months of age) were given either oral ANAVEX 3-71 or a placebo control for 4.5 months and then subjected to a washout period for five weeks. Following the washout, a number of cognitive tests were performed with the rats, now 18-20 months of age.
Despite the treatment interruption, the effects of administration of ANAVEX 3-71 resulted in a statistically significant improvement in cognitive function assessed by Novel Object Recognition and Morris Water Maze. In the Novel Object Recognition task, ANAVEX 3-71 completely reversed the deficit observed in transgenic rats (p<0.001), which performed similarly to wild-type rats. In the Morris Water Maze task, treated transgenic rats also fully recovered to perform at a level comparable to that of wild-type rats (p<0.001). A statistically significant reduction (p<0.05) in two measures of amyloid-beta pathology commonly associated with Alzheimer’s disease was also observed in comparison to control. Additionally, ANAVEX 3-71 demonstrated anti-inflammatory properties, compared to control (p<0.05), which may also have relevance in Alzheimer’s disease.
“The data from this study further validates the belief that ANAVEX 3-71 may have significant utility in the treatment of Alzheimer’s disease. This study examined potential long-term effect of the compound following a five-week hiatus from dosing, which suggests the potential for disease modification – something we have not often seen,” said Professor Claudio Cuello, Professor at the Department of Pharmacology and Therapeutics, McGill University, Montreal, Canada and main author of the study. “This preclinical study provides a variety of improvements in disease progression, including anti-inflammatory properties in aged rats, a test subject that more closely reflects the disease model in Alzheimer’s patients and which we hope to see repeated in human subjects.”
“This body of data of ANAVEX 3-71, in addition to our clinical candidate, ANAVEX 2-73 suggests that our approach of targeting the complex pathology of Alzheimer’s disease further upstream may have the potential to address the areas of unmet need for individuals living with this disease,” said Christopher U. Missling, PhD, President and Chief Executive Officer of Anavex.
OPNT which is LLTP and MNOV may benefit from this?
HHS awards $94 million to health centers to help treat the prescription opioid abuse and heroin epidemic in America
Today, Health and Human Services (HHS) Secretary Sylvia M. Burwell announced $94 million in Affordable Care Act funding to 271 health centers in 45 states, the District of Columbia, and Puerto Rico to improve and expand the delivery of substance abuse services in health centers, with a specific focus on treatment of opioid use disorders in underserved populations.
“The opioid epidemic is one of the most pressing public health issues in the United States today,” said Secretary Burwell. “Expanding access to medication-assisted treatment and integrating these services in health centers bolsters nationwide efforts to curb opioid misuse and abuse, supports approximately 124,000 new patients accessing substance use treatment for recovery and helps save lives.”
The abuse of and addiction to opioids, such as heroin and prescription pain medication, is a serious and increasing public health problem. Approximately 4.5 million people in the United States were non-medical prescription pain reliever users in 2013, and an estimated 289,000 were current heroin users. HHS also estimates the number of unintentional overdose deaths from prescription pain medications has nearly quadrupled from 1999 to 2013, and deaths related to heroin increased 39 percent between 2012 and 2013.
Administered by the HHS Health Resources and Services Administration (HRSA), these awards to health centers across the country will increase the number of patients screened for substance use disorders and connected to treatment, increase the number of patients with access to MAT for opioid use and other substance use disorder treatment, and provide training and educational resources to help health professionals make informed prescribing decisions. This $94 million investment is expected to help awardees hire approximately 800 providers to treat nearly 124,000 new patients.
“Health centers treat some of the most at-risk patients in the country,” said HRSA Acting Administrator Jim Macrae. “These awards position health centers to be at the forefront of the fight against opioid abuse in underserved communities.”
Research demonstrates that a whole-patient approach to treatment through a combination of medication and counseling and behavioral therapies is most successful in treating opioid use disorders. In 2014, over 1.3 million people received behavioral health services at health centers, This represents a 75 percent increase since 2008 and was made possible with support from the Affordable Care Act (ACA) and the Recovery Act. Today’s funding builds upon and leverages these previous investments by providing support to health centers to improve and expand the delivery of MAT substance abuse services in an integrated primary care/behavioral health model with a specific focus on treatment of opioid use disorders in underserved populations.
Today, over 1,300 health centers operate approximately 9,000 service delivery sites in every U.S. state, D.C., Puerto Rico, the Virgin Islands and the Pacific Basin; these health centers employ more than 170,000 staff who provide care for nearly 23 million patients. In 2014, health centers provided behavioral health services to more than 1.3 million patients, including those in need of substance abuse services.
“HRSA's innovative investment in the delivery of medication-assisted treatment for substance use disorders affirms the importance of behavioral health to overall health,” said Kana Enomoto, Acting Administrator of the Substance Abuse and Mental Health Services Administration (SAMHSA).
Addressing the opioid crisis is a top priority for the Administration and the Department. The Department is focused on three key areas: improving opioid prescribing practices, increasing the use of naloxone, and increasing access to MAT. In addition, the President has made addressing the prescription opioid abuse and heroin epidemic a top priority and issued a Presidential Memorandum last year on improving access to medication-assisted treatment (MAT) for opioid use disorders. Today’s awards are an example of HHS taking every available step to expand access to MAT. Building on these efforts, the President’s Budget includes a $1.1 billion initiative to help ensure that all individuals with opioid use disorders who want treatment are able to access it.
To view a list of the award winners, visit: http://bphc.hrsa.gov/programopportunities/fundingopportunities/substanceabuse/fy16awards.html
For more information on the Department’s key areas of focus to address the opioids crisis, visit: http://www.hhs.gov/news/press/2015pres/03/20150326a.html
To learn more about HRSA’s Health Center Program, visit http://bphc.hrsa.gov/about/index.html
To find a health center in your area, visit http://findahealthcenter.hrsa.gov/
###
Reading boards they have a deal with CELG. They also own a lot of shares.
GBIM up 100%
Anyone know why GBIM is up 100%?
XON.
OXFORD, England, March 11, 2016 /PRNewswire/ --
The US Food and Drug Administration's Center for Veterinary Medicine (FDA-CVM) today released in the Federal Register a preliminary finding of no significant impact (FONSI) on Oxitec's self-limiting OX513A Aedes aegypti mosquito for an investigational trial in the Florida Keys. The finding agrees with the draft environmental assessment (EA) submitted by Oxitec, Ltd., that concludes a field trial of the Company's genetically engineered (GE) OX513A mosquitoes in Key Haven, Florida, will not result in a significant impact on the environment. This follows an FDA-led evaluation of potential impacts on health and the environment of the proposed trial.
(Logo: http://photos.prnewswire.com/prnh/20150630/227348 )
Oxitec's Chief Executive Officer Hadyn Parry said, "We are pleased that the FDA-led team has released this preliminary FONSI. The Aedes aegypti mosquito represents a significant threat to human health, and in many countries has been spreading Zika, dengue and chikungunya viruses. This mosquito is non-native to the US and difficult to control, with the best available methods only able to reduce the population by up to 50%, which is simply not enough. We look forward to this proposed trial and the potential to protect people from Aedes aegypti and the diseases it spreads."
The purpose of the proposed trial is to determine the efficacy of Oxitec's self-limiting mosquitoes for the control of the local population of Aedes aegypti in Key Haven, Monroe County, Florida.
Oxitec's self-limiting mosquitoes have been genetically engineered so that their offspring die before reaching adulthood. Male Oxitec mosquitoes, which do not bite or spread disease, are released to mate with wild female Aedes aegypti so that their offspring die, reducing the population. Efficacy trials in Brazil, Panama, and the Cayman Islands have tested this approach, and in these trials the population of Aedes aegypti was reduced by more than 90% - an exceptional level of control compared to conventional methods, such as insecticides.
The FDA review team consisted of experts from the Center for Veterinary Medicine (CVM), the Centers for Disease Control and Prevention (CDC), and the Environmental Protection Agency (EPA). The FDA led an extensive review of evidence from trials in urban environments performed in Brazil, Panama and the Cayman Islands since 2009, and data from numerous safety studies, site inspections and independent experts.
For more information:
Oxitec Florida Keys Project FAQ: http://www.oxitec.com/health/florida-keys-project/
Florida Keys Mosquito Control District: http://keysmosquito.org/latest-gm-information/
AMSTERDAM, March 11, 2016 /PRNewswire/ -- Genomic Health, Inc. (GHDX) today announced new five-year clinical outcomes results from a large PlanB study, which was highlighted as an oral presentation at the 10th European Breast Cancer Conference (EBCC-10)1 in Amsterdam, Netherlands. The study, conducted by the West German Study Group (WSG), showed that 94 percent of early-stage breast cancer patients with Oncotype DX® Recurrence Score® results of 11 or less, who were treated with hormonal therapy alone, were disease-free five years after diagnosis.
These new PlanB study results with five-year outcomes provide information beyond the three-year outcomes published recently in the Journal of Clinical Oncology. The results add to the unprecedented amount of clinical outcomes data on Oncotype DX and are consistent with conclusions of the Trial Assigning IndividuaLized Options for Treatment (Rx), or TAILORx, recently published in The New England Journal of Medicine, and results from the Surveillance, Epidemiology, and End Results (SEER) program of the National Cancer Institute (NCI), recently presented at the San Antonio Breast Cancer Symposium.
"Our study shows the unique value of adding biologic information provided by the Oncotype DX test in order to identify low-risk breast cancer patients - among patients with 0-3 involved lymph nodes - who can safely be spared the toxicity and side effects of chemotherapy without compromising outcomes," said Prof. Nadia Harbeck, WSG scientific director and head of the breast center at University of Munich (LMU). "This is especially important for patients who would be considered as intermediate to high risk of recurrence based on traditional clinical parameters. These results confirm previous retrospective studies with Oncotype DX, as well as the prospective TAILORx trial, which already provided results for the node-negative population."
The study, conducted at 93 centers across Germany enrolled more than 3,100 patients with estrogen-receptor positive, HER2-negative, early-stage breast cancer, including those with node-positive disease (up to three nodes) who were considered candidates for chemotherapy by traditional parameters. The Oncotype DX test was used on all patients to identify those who could be spared adjuvant chemotherapy despite being considered as having high clinical risk disease by traditional parameters. Participants with Recurrence Score results of 12 or higher were randomized to different chemotherapy regimens, and patients with Recurrence Score results of 11 or less were treated with hormonal therapy alone.
In women with Recurrence Score results of 11 or less who were treated with hormonal therapy alone, five-year disease free survival (DFS) was estimated as 94 percent. Patients with Recurrence Score results of 12 to 25 who were treated with adjuvant chemotherapy also had high DFS rates of 94 percent, while in patients with Recurrence Score results above 25 who had also received chemotherapy, DFS rates were 84 percent.
"The compelling suite of new global prospective outcomes data generated in the last six months supports our prior validation work and all of the guidelines worldwide that include Oncotype DX to select patients for chemotherapy treatment, while providing physicians and patients with the highest level of evidence supporting the Recurrence Score as standard of care," said Steven Shak, M.D., chief scientific officer, Genomic Health.
Most recently, the German Association of Gynecological Oncology's (AGO's) treatment guidelines distinguished Oncotype DX as the first and only multi-gene breast cancer test with the highest 1A level of evidence, following publication of multiple large prospective outcomes studies. The AGO guidelines also reconfirmed Oncotype DX as the only multi-gene expression test validated to provide predictive information on the likelihood of chemotherapy benefit for women with early-stage, hormone-receptor positive, HER2-negative invasive breast cancer.
"Value in health care depends on results and outcomes, which is vital to the patient. Results from this study clearly show the value benefit of a personalized approach to breast cancer treatment," said Denis Horgan, executive director of the European Alliance for Personalized Medicine. "We hope to see more healthcare systems across Europe provide access to molecular diagnostics that are supported by a high level of scientific evidence and proven clinical utility."
Baker Bros have 42%.
AMSTERDAM, March 11, 2016 /PRNewswire/ -- Genomic Health, Inc. (GHDX) today announced new five-year clinical outcomes results from a large PlanB study, which was highlighted as an oral presentation at the 10th European Breast Cancer Conference (EBCC-10)1 in Amsterdam, Netherlands. The study, conducted by the West German Study Group (WSG), showed that 94 percent of early-stage breast cancer patients with Oncotype DX® Recurrence Score® results of 11 or less, who were treated with hormonal therapy alone, were disease-free five years after diagnosis.
These new PlanB study results with five-year outcomes provide information beyond the three-year outcomes published recently in the Journal of Clinical Oncology. The results add to the unprecedented amount of clinical outcomes data on Oncotype DX and are consistent with conclusions of the Trial Assigning IndividuaLized Options for Treatment (Rx), or TAILORx, recently published in The New England Journal of Medicine, and results from the Surveillance, Epidemiology, and End Results (SEER) program of the National Cancer Institute (NCI), recently presented at the San Antonio Breast Cancer Symposium.
"Our study shows the unique value of adding biologic information provided by the Oncotype DX test in order to identify low-risk breast cancer patients - among patients with 0-3 involved lymph nodes - who can safely be spared the toxicity and side effects of chemotherapy without compromising outcomes," said Prof. Nadia Harbeck, WSG scientific director and head of the breast center at University of Munich (LMU). "This is especially important for patients who would be considered as intermediate to high risk of recurrence based on traditional clinical parameters. These results confirm previous retrospective studies with Oncotype DX, as well as the prospective TAILORx trial, which already provided results for the node-negative population."
The study, conducted at 93 centers across Germany enrolled more than 3,100 patients with estrogen-receptor positive, HER2-negative, early-stage breast cancer, including those with node-positive disease (up to three nodes) who were considered candidates for chemotherapy by traditional parameters. The Oncotype DX test was used on all patients to identify those who could be spared adjuvant chemotherapy despite being considered as having high clinical risk disease by traditional parameters. Participants with Recurrence Score results of 12 or higher were randomized to different chemotherapy regimens, and patients with Recurrence Score results of 11 or less were treated with hormonal therapy alone.
In women with Recurrence Score results of 11 or less who were treated with hormonal therapy alone, five-year disease free survival (DFS) was estimated as 94 percent. Patients with Recurrence Score results of 12 to 25 who were treated with adjuvant chemotherapy also had high DFS rates of 94 percent, while in patients with Recurrence Score results above 25 who had also received chemotherapy, DFS rates were 84 percent.
"The compelling suite of new global prospective outcomes data generated in the last six months supports our prior validation work and all of the guidelines worldwide that include Oncotype DX to select patients for chemotherapy treatment, while providing physicians and patients with the highest level of evidence supporting the Recurrence Score as standard of care," said Steven Shak, M.D., chief scientific officer, Genomic Health.
Most recently, the German Association of Gynecological Oncology's (AGO's) treatment guidelines distinguished Oncotype DX as the first and only multi-gene breast cancer test with the highest 1A level of evidence, following publication of multiple large prospective outcomes studies. The AGO guidelines also reconfirmed Oncotype DX as the only multi-gene expression test validated to provide predictive information on the likelihood of chemotherapy benefit for women with early-stage, hormone-receptor positive, HER2-negative invasive breast cancer.
"Value in health care depends on results and outcomes, which is vital to the patient. Results from this study clearly show the value benefit of a personalized approach to breast cancer treatment," said Denis Horgan, executive director of the European Alliance for Personalized Medicine. "We hope to see more healthcare systems across Europe provide access to molecular diagnostics that are supported by a high level of scientific evidence and proven clinical utility."
OPTT is moving up.
Congratulations, I was thinking about buying today. Went with AVXL in early PM.