Search This Blog

Monday, December 10, 2018

Achillion: Interim Trial Data and Strategic Update Planned for Dec 17


Achillion Pharmaceuticals, Inc. (Nasdaq: ACHN), a clinical-stage biopharmaceutical company focused on advancing its orally administered factor D complement inhibitors into late-stage development and commercialization, announced today that the company will host a live webcast and conference call on December 17th at 4:30PM EST.
Joseph Truitt, President and Chief Executive Officer and Dr. Stephen Zelenkofske, Executive Vice President and Chief Medical Officer will present Achillion’s 2019 development plans along with interim data for the Company’s Phase 2 clinical trials in PNH and C3G as well as initial Phase 1 data on Achillion’s next generation factor D inhibitors.
The live audio and subsequent archived webcasts of the Company’s presentations will be accessible from the Company’s investor relations website: http://ir.achillion.com.
The audio recording will be archived for 30 days following the live presentation. Please connect to Achillion’s website several minutes prior to the start of the presentation to ensure adequate time for any software downloads that may be necessary.
Alternatively, the webcast and audio can be accessed directly as follows:
US Toll-Free Dial-In Number: (866) 205-4820
International Dial-In Number: (419) 386-0004
Conference ID# 5987613
Posted by at No comments:

GW: SC docs, insurers turn to first FDA-approved cannabidiol med for epilepsy


South Carolina physicians are prescribing a medication containing a marijuana byproduct for the first time, following approval this summer from the Food and Drug Administration.
Epidiolex, manufactured by GW Pharmaceuticals, was approved by the FDA in late June, specifically to treat two rare forms of epilepsy. The medicine became available for prescription in early November.
The medication contains CBD. It is THC, not CBD, that is the component of marijuana that can get the user high. Epidiolex is approved for anyone 2 years old or older with two specific epilepsy syndromes.
In late September, the Drug Enforcement Administration announced they would move Epidiolex into Schedule 5 of the Controlled Substances Act, which is the least restrictive option. Marijuana is still in Schedule 1.
Leadership at the pharmaceutical company told investors in August the drug costs about $32,500 per year, Business Insider reported.
GW Pharmaceuticals has said that cost is on par with other epilepsy drugs, and it has a patient support program to help make the product more affordable.
The CBD-based medication is approved to treat Lennox-Gastaut syndrome and Dravet syndrome, both serious forms of epilepsy. Lennox-Gastaut, in particular, is resistant to many other medications, according to the National Organization for Rare Disorders. Both appear in early childhood.
“Because these patients have historically not responded well to available seizure medications, there has been a dire need for new therapies that aim to reduce the frequency and impact of seizures,” Justin Gover, GW Pharmaceuticals’ CEO, said in a statement.
Dr. Emma Grace Carter, a pediatric neurologist at the Medical University of South Carolina, said she has begun prescribing Epidiolex to her patients. She explained the approval of the drug is good news for patients with the two syndromes, who often try many medications in attempts to address their epilepsy.
“Some of them are interested in it because they feel it’s a more natural medication,” Carter said. “It’s another option to look into.”
She said Epidiolex is not going to be the first option specialists turn to. Rather, they will prescribe it when other medications have failed.
Carter said her team has only been prescribing the medication for a short time, so it is hard to say yet how effective it will be among her patients.
“We are hopeful about it, but we don’t have a lot of the clinical data ourselves,” she said.
BlueCross BlueShield of South Carolina, the largest health insurance company in the state does not yet cover it.
Dr. Matthew Bartels, chief medical officer for BlueCross BlueShield of South Carolina, said in a statement last week that Epidiolex is still going through the insurer’s review process for newly approved medicines.
“This approach allows us to evaluate the drug’s effectiveness and safety in a population beyond the clinical trial participants,” he said. “In the meantime, there are other anti-epileptic drugs available and covered, and we have processes to ensure that our members receive appropriately prescribed medications.”
Colleen Mullis, spokeswoman for the S.C. Department of Health and Human Services, said the drug will be covered by the state’s Medicaid program, so long as it meets the FDA requirements.
Medicaid covers mostly children in South Carolina.
In South Carolina, 53,400 people and 7,100 minors have some form of epilepsy, according to the Centers for Disease Control and Prevention .
Posted by at No comments:

Can-Fite Agrees to Explore Med’s Anti-NASH Effect With Mt. Sinai Icahn School


Can-Fite to Support Research Directed by Scott Friedman, M.D. on Namodenoson in Human Liver Cells
Can-Fite BioPharma Ltd. (NYSE American: CANF) (TASE:CFBI), a biotechnology company advancing a pipeline of proprietary small molecule drugs that address cancer, liver and inflammatory diseases, announced a collaborative research agreement with the Icahn School of Medicine at Mount Sinai in New York City. The agreement will support research directed by Scott Friedman, M.D., Dean for Therapeutic Discovery and Chief of the Division of Liver Diseases at the Icahn School of Medicine. The research is aimed at transcriptomic and molecular analyses to further explore the mechanisms of action of Namodenoson (CF102) in human hepatic stellate cells in order to clarify its effect on fibrogenesis that occurs in non-alcoholic steatohepatitis (NASH).
“We are privileged to work with Dr. Friedman, a Key Opinion Leader in the arena of nonalcoholic fatty liver disease (NAFLD) and NASH, to further study and advance our understanding of the molecular mechanism of action of Namodenoson,” said Pnina Fishman, Ph.D., Founder and Chief Executive Officer of Can-Fite. “This work aims to provide a more solid foundation for our ongoing efforts to understand the potential of Namodenoson in these therapeutic areas.”
In the transcriptomic bioinformatics analysis, Dr. Friedman will research differentially expressed genes and modulated molecular pathways in the transcriptome datasets in association with the experimental perturbations. Integrative (cross-species) analysis with clinical datasets for assessment of association with clinical disease phenotypes and outcomes will be performed. An emphasis will be given to the analysis of the PI3K and the Wnt/β-catenin pathways, which are up-regulated in NAFLD/NASH livers and found to be improved upon treatment with Namodenoson.
Recent preclinical studies with Namodenoson in collaboration with Rifaat Safadi, M.D., the Head of the Liver Unit, Gastroenterology and Liver Diseases, Division of Medicine at Hadassah Medical Center in Israel, showed an improvement in three cardinal NASH parameters including steatosis, inflammation and fibrosis.
Can-Fite is currently enrolling patients in a Phase II study of Namodenoson in NAFLD/NASH. Based on the recent preclinical data, the primary endpoint of the Phase II study is Percent Change From Baseline (PCFB) in serum alanine aminotransferase (ALT) levels at Week 12 for each dose of CF102 compared to placebo, with the major secondary endpoint being percentage of liver fat as measured by magnetic resonance imaging-proton density fat fraction (MRI-PDFF).
Posted by at No comments:

Recro: New Data Demonstrating IV Meloxicam’s Effect on Platelet Function


Recro Pharma, Inc. (NASDAQ:REPH), a revenue generating specialty pharmaceutical company focused on therapeutics for the hospital and other acute care settings, today announced a poster presentation highlighting intravenous (IV) meloxicam’s effect on platelet function at the 72nd PostGraduate Assembly in Anesthesiology (PGA72), sponsored by the New York State Society of Anesthesiologists (NYSSA), taking place December 7-11, 2018, in New York City.
The poster, titled “An Ex-vivo Assessment of the Effects of Meloxicam IV on Platelet Function,” which was presented by lead author Jonathan S. Jahr, M.D., David Geffen School of Medicine at UCLA, describes results from an ex vivo study assessing the effects of various concentrations of IV meloxicam (5mcg/mL, 10mcg/mL, 15mcg/mL and 20mcg/mL) on platelet function compared to ketorolac (2.5mcg/mL and 5mcg/mL) and an untreated control.  The 5mcg/mL concentration of IV meloxicam in this study corresponds to the maximum plasma levels following a 30mg dose in the clinical setting.  Similarly, the 5mcg/mL concentration of ketorolac in this study corresponds to a 30mg and the 2.5mcg/mL dose corresponds to the 15mg dose in the clinical setting.  In this study, blood samples provided by healthy volunteers (n=8) were analyzed to determine closure time (CT) following the addition of IV meloxicam and ketorolac at the previously mentioned concentrations to the blood plasma samples.  The researchers conducted the analyses using a platelet function analyzer (PFA-100), a standard of care for platelet function testing, and two different reagents, collagen with epinephrine (CEPI) and collagen with adenosine diphosphate (CADP).
In the CEPI reagent analysis, a significant treatment effect was observed for changes in CT (p=0.0441).  No significant difference was observed in CT for the IV meloxicam treated samples versus the untreated control at any of the evaluated concentrations (p≥0.5400).  When compared to untreated control, the ketorolac treated sample CT values were significantly prolonged in both the 2.5mcg/mL and the 5mcg/mL concentrations (p≤0.0257).  All IV meloxicam concentration levels had a significantly shorter CT compared to the 2.5mcg/mL ketorolac concentration (p<0.005).  All IV meloxicam concentration levels had numerically shorter CTs compared to the 5mcg/mL ketorolac concentration, though only the 10mcg/mL IV meloxicam concentration reached statistical significance.  In the CADP reagent analysis, neither IV meloxicam nor ketorolac demonstrated a significant change in CT versus untreated control (p≥0.0907).
“A concern around the use of non-steroidal anti-inflammatory drugs (NSAIDs) in the peri- or post-operative setting is the potential for platelet dysfunction and risk of bleeding related events,” commented Dr. Jahr.  “Our thesis here was that IV meloxicam’s higher affinity for COX-2 inhibition, versus COX-1, would translate into a lower risk for platelet dysfunction.  The data presented this year at PGA72 show that IV meloxicam achieved roughly the same closure times (CTs) as untreated controls and numerically shorter CTs than ketorolac.  In contrast, significant CT prolongations were observed in ketorolac samples compared with untreated control.  These results suggest that IV meloxicam has the potential to provide a meaningful clinical benefit over ketorolac with respect to a decreased risk of platelet dysfunction.”
“NSAIDs are an integral part of the analgesic toolkit and are the physician’s first-line defense to prevent or treat pain,” said Stewart McCallum, M.D., F.A.C.S., Chief Medical Officer of Recro Pharma and co-author of the poster.  “These compelling data continue to build upon the growing body of data supporting IV meloxicam’s safety and reinforce the positive safety findings elucidated by our comprehensive IV meloxicam development program.  The IV meloxicam New Drug Application is currently under review by the U.S. Food and Drug Administration and we are currently awaiting our PDUFA goal date of March 24, 2019.”
A downloadable copy of the poster can be accessed by visiting the “Investors” section of the Recro Pharma website and clicking “Presentations.”
Posted by at No comments:

Axsome: Positive Interim Analysis of Phase 2/3 Alzheimer’s Agitation Med


IDMC recommends continuation of trial evaluating AXS-05 versus placebo in Alzheimer’s disease agitation
IDMC recommends no further enrollment to single-agent bupropion arm
Axsome Therapeutics, Inc. (NASDAQ: AXSM), a clinical-stage biopharmaceutical company developing novel therapies for the management of central nervous system (CNS) disorders, today announced a positive outcome of the interim analysis for the ADVANCE-1 Phase 2/3 trial of AXS-05 in agitation associated with Alzheimer’s disease (AD). An independent data monitoring committee (IDMC) conducted the unblinded, pre-specified interim analysis for futility. Based on the results of the analysis, the IDMC recommended continuation of the AXS-05 treatment arm. Additionally, the IDMC recommended no further randomization of subjects to the bupropion treatment arm of the study. The IDMC did not indicate that there were any safety concerns in the study. Axsome intends to follow the IDMC’s recommendations.
AXS-05 is a novel, oral, investigational medicine consisting of dextromethorphan and bupropion. Patients in the ADVANCE-1 trial were being randomized in a 1:1:1 ratio to treatment with AXS-05, placebo, or bupropion. The primary endpoint of the study is the change in the Cohen Mansfield Agitation Inventory (CMAI) for AXS-05 as compared to placebo. The single-agent bupropion arm of the study was included to compare the efficacy of AXS-05 to the bupropion component, with the goal of demonstrating the superiority of AXS-05, as required by the U.S. Food and Drug Administration’s (FDA’s) combination product rule. In accordance with the IDMC’s recommendation, the study will now continue enrollment in a 1:1 ratio to only the AXS-05 and placebo arms. The ADVANCE-1 interim futility analysis was performed on the first approximately 30% of the target number of subjects.
“The positive recommendation by the IDMC to continue the ADVANCE-1 trial is an important milestone for the Alzheimer’s disease agitation program and we look forward to the continued development of AXS-05 for this indication,” said Herriot Tabuteau, MD, Chief Executive Officer of Axsome. “We are pleased that further enrollment to the bupropion single-agent arm is no longer deemed necessary. Inclusion of that arm was driven by the FDA’s guidelines to examine contribution of the individual components of product candidates like AXS-05 that contain two active agents. We anticipate that implementation of the IDMC’s recommendations may provide greater flexibility to our operating plans and timelines which we will evaluate in the coming weeks. We will continue to be rigorous with our oversight of the conduct of the study.”
Posted by at No comments:

Cytokinetics New Data Presented at the International Symposium on ALS/MND


Cytokinetics, Incorporated (Nasdaq: CYTK) today announced that new data were presented at the 29th International Symposium on ALS/MND in Glasgow, Scotland, UK, including patient baseline characteristics and demographics from FORTITUDE-ALS, the Phase 2 clinical trial of reldesemtiv in patients with ALS. In addition, posters presented at the conference contained data from an analysis of the correlation between slow vital capacity (SVC) measured at home and in the clinic in FORTITUDE-ALS, additional analyses from VITALITY-ALS, the Phase 3 clinical trial of tirasemtiv in patients with ALS, new results from IMPACT ALS, a patient and caregiver survey conducted by The ALS Association and funded in part by Cytokinetics, and results from analyses conducted by Origent Data Sciences on the validation of machine learning models to predict ALS disease progression using data from VITALITY-ALS.
FORTITUDE-ALS, a Phase 2 clinical trial of reldesemtiv designed to assess the change from baseline in percent predicted SVC and other measures of skeletal muscle function after 12 weeks of treatment with reldesemtiv in patients with ALS, completed enrollment in November 2018. The trial enrolled 458 patients with ALS from centers in the U.S., Canada, Europe and Australia.
Baseline characteristics of patients enrolled in FORTITUDE-ALS are similar to those of other recent large clinical trials in ALS, including BENEFIT-ALS and VITALITY-ALS. Patients enrolled in FORTITUDE-ALS were on average 58.7 years of age, 60.7 percent male, 8.4 months from diagnosis, 22.9 months from their first symptom and had an average percent predicted SVC of 86.8 percent. Regarding other therapies for ALS, the majority of patients were taking only Rilutek® (riluzole) (259, 56.6%), while 19 (4.1%) were taking only Radicava® (edaravone), 94 (20.5%) were taking both riluzole and edaravone, and 86 (18.8%) were taking neither. Demographics and baseline characteristics were similar among patients who received riluzole alone, edaravonealone, received both or received neither.
“We are encouraged to see that the baseline characteristics from FORTITUDE-ALS are consistent with previous ALS trials, and are pleased to be able to share them with the ALS community,” Jeremy Shefner, M.D., Ph.D., Lead Investigator of FORTITUDE-ALS, Professor and Chair of Neurology at Barrow Neurological Institute, and Professor and Executive Chair of Neurology at the University of Arizona, Phoenix. “With enrollment now complete in this trial, we look forward to learning how reldesemtiv may impact slow vital capacity and other measures of skeletal muscle function to potentially complement current therapies for patients bravely battling ALS.”
Posted by at No comments:

Achaogen: FDA OKs Thermo Scientific QMS Plazomicin Immunoassay


Achaogen, Inc. (NASDAQ: AKAO), a biopharmaceutical company discovering, developing and commercializing innovative antibacterial agents to address multi-drug resistant (MDR) gram-negative infections, today announced that the Center for Devices and Radiological Health (CDRH) of the U.S. Food and Drug Administration (FDA) has provided clearance for the Thermo Scientific QMS Plazomicin Immunoassay that provides a quantitative measure of plazomicin in human plasma using automated clinical chemistry analyzers.  Results of the assay will be used to assist in the management of patients receiving plazomicin therapy.
Plazomicin, marketed as ZEMDRI™ in the United States, was approved by the U.S. Food and Drug Administration for the treatment of adults with complicated urinary tract infections, including pyelonephritis, due to certain Enterobacteriaceae.
“The QMS Plazomicin Immunoassay will enable therapeutic drug management (TDM) which is critical to support the use of ZEMDRI in a subset of the more than one million annual cases of multi-drug resistant or recurrent complicated urinary tract infections in the United States,” said Janet Dorling, Achaogen’s Chief Commercial Officer.  “This is an important milestone and we are excited about the commercialization of the QMS Plazomicin immunoassay.”
Thermo Fisher Scientific and Achaogen jointly developed the QMS Plazomicin immunoassay.  Both companies entered into a collaboration to develop an assay that would help healthcare professionals make important dosing determinations regarding the use of ZEMDRI for appropriate patients.
Posted by at No comments:
Subscribe to: Posts (Atom)