She has created toxin - smothering antibodies that have been shown to be safe in humans by Caprion Pharmaceuticals, paving the way
for efficacy trials.
Not exact matches
In particular, the complaint alleges that throughout the Class Period, defendants made materially false and / or misleading statements and / or failed to disclose that (1) the
trials for GED - 0301 suffered from fatal design defects, such that GED - 0301 had failed to demonstrate meaningful clinical
efficacy; (2) the growth of Otezla sales had dramatically slowed during Celgene's third fiscal quarter of 2017; and (3) the clinical and nonclinical pharmacology data in Celgene's new drug application («NDA»)
for Ozanimod were insufficient to permit a complete review by the FDA, which resulted in the FDA issuing a refusal to file letter to Celgene regarding the NDA.
These risks and uncertainties include, among others: the unfavorable outcome of litigation, including so - called «Paragraph IV» litigation and other patent litigation, related to any of our products or products using our proprietary technologies, which may lead to competition from generic drug manufacturers; data from clinical
trials may be interpreted by the FDA in different ways than we interpret it; the FDA may not agree with our regulatory approval strategies or components of our filings
for our products, including our clinical
trial designs, conduct and methodologies and,
for ALKS 5461, evidence of
efficacy and adequacy of bridging to buprenorphine; clinical development activities may not be completed on time or at all; the results of our clinical development activities may not be positive, or predictive of real - world results or of results in subsequent clinical
trials; regulatory submissions may not occur or be submitted in a timely manner; the company and its licensees may not be able to continue to successfully commercialize their products; there may be a reduction in payment rate or reimbursement
for the company's products or an increase in the company's financial obligations to governmental payers; the FDA or regulatory authorities outside the U.S. may make adverse decisions regarding the company's products; the company's products may prove difficult to manufacture, be precluded from commercialization by the proprietary rights of third parties, or have unintended side effects, adverse reactions or incidents of misuse; and those risks and uncertainties described under the heading «Risk Factors» in the company's most recent Annual Report on Form 10 - K and in subsequent filings made by the company with the U.S. Securities and Exchange Commission («SEC»), which are available on the SEC's website at www.sec.gov.
Actual results and the timing of events could differ materially from those anticipated in the forward - looking statements due to these risks and uncertainties as well as other factors, which include, without limitation: the uncertain timing of, and risks relating to, the executive search process; risks related to the potential failure of eptinezumab to demonstrate safety and
efficacy in clinical testing; Alder's ability to conduct clinical
trials and studies of eptinezumab sufficient to achieve a positive completion; the availability of data at the expected times; the clinical, therapeutic and commercial value of eptinezumab; risks and uncertainties related to regulatory application, review and approval processes and Alder's compliance with applicable legal and regulatory requirements; risks and uncertainties relating to the manufacture of eptinezumab; Alder's ability to obtain and protect intellectual property rights, and operate without infringing on the intellectual property rights of others; the uncertain timing and level of expenses associated with Alder's development and commercialization activities; the sufficiency of Alder's capital and other resources; market competition; changes in economic and business conditions; and other factors discussed under the caption «Risk Factors» in Alder's Annual Report on Form 10 - K
for the fiscal year ended December 31, 2017, which was filed with the Securities and Exchange Commission (SEC) on February 26, 2018, and is available on the SEC's website at www.sec.gov.
These risks and uncertainties include: Gilead's ability to achieve its anticipated full year 2018 financial results; Gilead's ability to sustain growth in revenues
for its antiviral and other programs; the risk that private and public payers may be reluctant to provide, or continue to provide, coverage or reimbursement
for new products, including Vosevi, Yescarta, Epclusa, Harvoni, Genvoya, Odefsey, Descovy, Biktarvy and Vemlidy ®; austerity measures in European countries that may increase the amount of discount required on Gilead's products; an increase in discounts, chargebacks and rebates due to ongoing contracts and future negotiations with commercial and government payers; a larger than anticipated shift in payer mix to more highly discounted payer segments and geographic regions and decreases in treatment duration; availability of funding
for state AIDS Drug Assistance Programs (ADAPs); continued fluctuations in ADAP purchases driven by federal and state grant cycles which may not mirror patient demand and may cause fluctuations in Gilead's earnings; market share and price erosion caused by the introduction of generic versions of Viread and Truvada, an uncertain global macroeconomic environment; and potential amendments to the Affordable Care Act or other government action that could have the effect of lowering prices or reducing the number of insured patients; the possibility of unfavorable results from clinical
trials involving investigational compounds; Gilead's ability to initiate clinical
trials in its currently anticipated timeframes; the levels of inventory held by wholesalers and retailers which may cause fluctuations in Gilead's earnings; Kite's ability to develop and commercialize cell therapies utilizing the zinc finger nuclease technology platform and realize the benefits of the Sangamo partnership; Gilead's ability to submit new drug applications
for new product candidates in the timelines currently anticipated; Gilead's ability to receive regulatory approvals in a timely manner or at all,
for new and current products, including Biktarvy; Gilead's ability to successfully commercialize its products, including Biktarvy; the risk that physicians and patients may not see advantages of these products over other therapies and may therefore be reluctant to prescribe the products; Gilead's ability to successfully develop its hematology / oncology and inflammation / respiratory programs; safety and
efficacy data from clinical studies may not warrant further development of Gilead's product candidates, including GS - 9620 and Yescarta in combination with Pfizer's utomilumab; Gilead's ability to pay dividends or complete its share repurchase program due to changes in its stock price, corporate or other market conditions; fluctuations in the foreign exchange rate of the U.S. dollar that may cause an unfavorable foreign currency exchange impact on Gilead's future revenues and pre-tax earnings; and other risks identified from time to time in Gilead's reports filed with the U.S. Securities and Exchange Commission (the SEC).
«The positive
efficacy results observed in the pivotal phase 3 clinical
trials indicate that sarecycline can be an effective treatment option
for patients with moderate to severe acne,» said Allergan R&D chief David Nicholson in a statement.
If large - scale
trials can replicate safety and
efficacy results, the drug could be approved
for legal use by 2021.
We do this by conducting clinical
trials in which we collect safety and
efficacy data about our experimental drugs with the goal of submitting those data to regulatory authorities, like the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA), so that these experimental drugs can be approved
for use by patients.
Most biotechs would have shuttered a clinical program that showed absolutely no
efficacy whatsoever in a late - stage
trial, but Novavax has instead doubled down on RSV by going forth with its phase 3
trial of the RSV F vaccine
for infants via maternal immunization, and begun planning
for another
trial in older adults that's reportedly on track to kick off in 2018.
The DAME
trial was a multi-site, randomised controlled
trial of antenatal expression of colostrum in late pregnancy
for women with diabetes in pregnancy to explore the safety and
efficacy for mother, foetus and infant.
Safety and
efficacy of antenatal milk expressing
for women with diabetes in pregnancy: protocol
for a randomised controlled
trial.
The
efficacy and safety of the probiotic bacterium Lactobacillus reuteri DSM 17938
for infantile colic: A meta - analysis of randomized controlled
trials.
Although professional lactation support can improve the duration of overall breast feeding, its effect in improving exclusive breast feeding is unclear.11 18 22 Thus far, studies that report improvement of rates of exclusive breastfeeding have involved mainly community based peer counselling strategies.23 24 25 Even then, a randomised
trial in the UK recently cast doubt on the
efficacy of this approach.26 There are current recommendations from NICE
for the UK - wide implementation of the baby friendly initiative.4 5 6 The 2006 NICE costing report on routine postnatal care of women and their babies estimates that efforts to improve rates of breast feeding will result in substantial cost savings
for the NHS.6
The
efficacy of these treatments
for other anxiety disorders in children and adolescents has been supported by multiple randomized controlled
trials (e.g., Walkup et al., 2008).
«A future clinical
trial for NMN will tell us if it has any
efficacy in humans.»
«This phase III
trial will be noteworthy
for being the first prostate cancer
trial to assess a biomarker, namely AR - V7 in circulating tumour cells, as a predictor of response at the same time as testing the
efficacy of the drug,» Prof Taplin will conclude.
Ultimately, AA147 — or some version of it — would need to undergo clinical
trials for safety and
efficacy before it could potentially be used to treat patients.
Although gene therapy research has made great strides in recent years, it has yet to be widely deployed, and no CRISPR - edited genes have yet been tested
for safety or
efficacy in human clinical
trials.
The CO-STAR (Hepatitis C Patients on Opioid Substitution Therapy Antiviral Response)
trial sought to evaluate the
efficacy and safety of elbasvir - grazoprevir
for injection drug users.
Clinical
trials that specifically test ADAR1 - targeted therapeutics
for their safety and
efficacy against multiple myeloma are still necessary before this approach could become available to patients.
«The study results elucidate the molecular mechanisms underlying disease progression in multiple sclerosis models, providing a basis
for future clinical
trials to determine safety and
efficacy of these chemical agents in humans with demyelinating disorders,» says Patrizia Casaccia, MD, PhD, Professor of Neuroscience, Genetics and Genomic Sciences at Mount Sinai and senior author of the study.
«Randomized
trials have confirmed the value of radiation dose escalation
for prostate tumors, and the potential benefits of larger radiation doses in fewer fractions, are expected to increase the therapeutic
efficacy for men with prostate cancer,» said Anders Widmark, MD, a professor of radiation sciences at Umeå University in Umeå, Sweden and lead author of the study.
Dr. Rassool says that a clinical
trial is planned to test whether low doses of a DNMT inhibitor, decitabine, and an investigational PARP inhibitor, talazoparib, can be safely combined and whether this therapy shows
efficacy for AML patients, especially those who can not receive intensive chemotherapy, whose leukemia is resistant to treatment, or who have experienced a relapse after treatment.
Respondents in this year's survey pointed to five main causes of the field's less than favorable reputation: drug and product recalls such as the withdrawal of Avandia; safety issues such as the discovery of problems with raw material from China used in medical products; scandals, including evidence that pharmaceutical companies have failed to release data from
trials whose results cast doubts on their drugs» safety and
efficacy; lawsuits brought against companies that failed to warn patients of problems with their products; and ethical issues such as kickbacks
for physicians promoting specific medications.
Schlom, of the National Cancer Institute, says the next challenge
for researchers is to find ways to combine different immunotherapy drugs into single treatments and measure their
efficacy in clinical
trials.
Following the success of this preliminary safety and feasibility study, more patients are being recruited
for a larger clinical
trial of the procedure to test the
efficacy and durability of the procedure.
The company hopes that early next year it will initiate clinical
trials on product safety, which will pave the way
for human
efficacy testing that could be completed by 2011.
The determination of each
efficacy level was also based on the rigor and quantity of published studies on the drug class: to be in Level A,
for example, a class of drugs must have been supported by at least two «Class I» studies — well - designed, double - blind, randomized, placebo - controlled clinical
trials.
With proof of
efficacy now published in monkeys and rabbits, preparations are being made
for the first clinical
trial in humans.
The documentation ranges across the whole spectrum of drug development: Investigators» brochures provide information on all that is currently known about the medicine and so need periodic updating; accurate and concise protocols are required to ensure that
trials are performed effectively; clinical
trial reports (generally from phase II and III studies) present the information gathered from the
trials; higher level documents provide summaries of
efficacy and safety data from clinical
trial programmes; expert reports provide critical interpretation of the results; and response documents clarify any points that are not clear to the regulatory agencies or provide additional analyses or supporting data
for any items of concern.
Human safety
trials for a vaccine to jump - start immunity could begin later this year; larger
efficacy trials may be a year and a half away.
Downing and the team evaluated the strength of clinical
trial evidence supporting FDA approval decisions
for new drugs by characterizing key features of
efficacy trials, such as
trial size, design duration, and end points.
Conventional drug discovery remains a hit - and - miss affair and Hunter believes the 50 percent failure rates seen
for experimental compounds in mid - and late - stage clinical
trials due to lack of
efficacy is unsustainable, forcing a shift to AI.
The data obtained from this study provide a basis
for more rapid, cost - effective clinical
trials to evaluate new influenza drugs or to determine the
efficacy of candidate vaccines
for both seasonal and pandemic influenza.
The rule provides a framework
for potential licensure of medical products when definitive
efficacy studies that would involve exposing healthy human volunteers are unethical and field
trials after accidental human exposure are not feasible.
«Given the effectiveness of inosine in promoting cortical plasticity, axonal sprouting, and dendritic branching, the present evidence of
efficacy after cortical injury in a non-human primate, combined with a long history of safe use, indicates a need
for clinical
trials with inosine after cortical injury and spinal cord injury,» noted Dr. Moore.
Yale enrolled the largest number of participants at any one site (84 of 790)
for these double - blind, placebo - controlled
trials that investigated the
efficacy of testosterone gel
for multiple outcomes, including sexual function, physical function, and vitality.
A clinical
trial investigating a treatment
for blindness is under way this winter to evaluate the safety and
efficacy of replacing diseased eye cells with stem cells.
Vaccine
efficacy was set at an average of 60 % (actual numbers depended on the different virus subtypes and on whether a person had been exposed due to a prior natural infection) based on recent phase III
trial results
for the Sanofi - Pasteur vaccine that had shown moderate effectiveness.
«Right now, one of the biggest areas of failure is in phase II
trials, when you get to those places where you're looking
for efficacy and you suddenly discover that the compound that worked great in your animal model doesn't do much in humans,» he says.
Further research will be needed to adapt the technique
for humans, but the approach could offer improved safety and
efficacy control
for human clinical
trials, which are now underway in Canada.
This compound has been recently designated
for phase III clinical
trials to test its
efficacy for treating epilepsy and providing relief of neuropathic pain.
The Phase 2/3
trials will be sponsored by Vtesse, a new biotech company that was formed specifically to evaluate the safety and
efficacy of cyclodextrin
for the treatment of NPC and ultimately obtain FDA labeling if the drug is effective.
A pooled analysis of data from two randomised
trials comparing vitamin E versus placebo, and the placebo group from another
trial comparing vitamin E use versus non-use, demonstrates that the
efficacy of vitamin E is comparable to other treatments
for NASH, including pioglitazone, metformin and obeticholic acid.
Two new phase III clinical
trials investigating the
efficacy and safety of bitopertin, a glycine uptake inhibitor considered to be a promising new add - on therapy
for treating negative symptoms in schizophrenia, failed to show a benefit of the drug over placebo.
If these preclinical studies are successful, the researchers plan to further develop their CAR T cell therapy and test its safety and
efficacy for different types of metastatic cancer in upcoming clinical
trials.
A total of 347 patients (155 treated with vitamin E, 192 not treated with vitamin E) were included in the analysis which compared data from three clinical
trials that investigated the
efficacy and safety of vitamin E as a treatment
for NASH: the PIVENS, TONIC and FLINT
trials.
The standard pharmaceutical development path
for products that target pathogens moves slowly from studying safety, dosing, and biological responses in hundreds of people to an expensive
efficacy trial with thousands of participants at high risk of becoming naturally infected.
At the meeting, GlaxoSmithKline (GSK) of Rixensart, Belgium, which has the vaccine furthest in development, spelled out how it might scale up production in parallel with the safety and
efficacy trials now under way so that the product could be ready
for wider distribution by April if warranted.
«The first year results from the CoreValve US Pivotal
Trial support the safety and
efficacy of this therapy in patients unsuitable
for surgical aortic valve replacement,» said lead investigator Steven Yakubov, MD..