The U.K. regulator tasked with advising the country's health service on what drugs to use said Friday that it will recommend AstraZeneca PLC's (AZN.LN) asthma medication Fasenra, reversing an earlier negative decision.
The National Institute for Health and Care Excellence said the judgment comes after it struck a deal over pricing with the Anglo-Swedish pharmaceutical company. Fasenra has a list price of 1,955 pounds ($2,475) per dose, but NICE said it was able to negotiate this down, though the final price remains confidential.
Fasenra is a monthly shot for the treatment of severe eosinophilic asthma, a type of asthma in which the body produces an excessive amount of white blood cells called eosinophils. It is Astra's first respiratory drug in a class of medicines called biologics, which are derived from living organisms.
The drugmaker has previously told Dow Jones that it hopes Fasenra can eventually replace the use of steroids, which have extensive side-effects, as a treatment.
Biologic medicines can transform the lives of patients suffering from severe eosinophilic asthma, which can otherwise hold them back from doing daily tasks, said Meindert Boysen, director of NICE's Centre for Health Technology Evaluation.
"This recommendation of a further biological option demonstrates how a competitive pharmaceuticals market combined with NICE's appraisal process provides the NHS and patients with value-for-money and choice," Mr. Boysen said.
https://www.marketscreener.com/ASTRAZENECA-4000930/news/AstraZeneca-U-K-s-NICE-Approves-Astra-s-Asthma-Medication-After-Striking-Pricing-Deal-27820678/
Friday, January 4, 2019
Bayer: U.S. judge limits evidence in trial over Roundup cancer claims
A federal judge overseeing lawsuits alleging Bayer glyphosate-based weed killer causes cancer has issued a ruling that could severely restrict evidence that the plaintiffs consider crucial to their cases.
U.S. District Judge Vince Chhabria in San Francisco in an order on Thursday granted Bayer unit Monsanto’s request to split an upcoming trial into two phases. The order initially bars lawyers for plaintiff Edwin Hardeman from introducing evidence that the company allegedly attempted to influence regulators and manipulate public opinion.
Thursday’s order applies to Hardeman’s case, which is scheduled to go to trial on Feb. 25, and two other so-called bellwether trials which will help determine the range of damages and define settlement options for the rest of the 620 Roundup cases before Chhabria.
But Hardeman’s lawyers contended that such evidence, including internal Monsanto documents, showed the company’s misconduct and were critical to California state court jury’s August 2018 decision to award $289 million in a similar case. The verdict sent Bayer shares tumbling though the award was later reduced to $78 million (61.7 million pounds) and is under appeal.
Under Chhabria’s order, evidence of Monsanto’s alleged misconduct would be allowed only if glyphosate was found to have caused Hardeman’s cancer and the trial proceeded to a second phase to determine Bayer’s liability.
Bayer denies allegations that glyphosate causes cancer, saying decades of independent studies have shown the world’s most widely used weed killer to be safe for human use.
But the company faces more than 9,300 U.S. lawsuits over Roundup’s safety in state and federal courts across the country.
Bayer in a statement welcomed Chhabria’s decision.
“The court’s decision to keep the focus of the trial on the extensive science relevant to human health is encouraging,” the company said.
Aimee Wagstaff, one of Hardeman’s lawyers, in a statement said she was confident the jurors will find Roundup caused the man’s cancer and proceed to the second phase.
Hardeman’s attorneys had opposed proposals to split up the trial on the grounds that their scientific evidence allegedly showing glyphosate causes cancer was inextricably linked to Monsanto’s alleged wrongful conduct.
Bayer has also asked that some of the plaintiffs’ evidence on causation, specifically a finding by the World Health Organisation’s cancer unit that glyphosate is “probably carcinogenic,” be excluded in the first phase because it has no basis in science.
Chhabria, who has previously expressed scepticism of that finding, on Thursday said he would soon decide to which degree he would allow it to be introduced at trial.
The assessment is central to the plaintiffs’ claims, as other regulatory agencies, including the U.S. Environmental Protection Agency, have determined glyphosate likely does not cause cancer.
Zealand Pharma completes investment in strategic partner Beta Bionics
- Zealand Pharma invests USD 3.5 million in the final close of strategic partner Beta Bionics’ series B financing round
- Beta Bionics is the developer of the iLet™ bionic pancreas – an autonomous dual hormone artificial pancreas, using Zealand’s dasiglucagon for automated glucose regulation in type 1 diabetes
- Dasiglucagon is a potential first-in-class glucagon analogue for use together with insulin in dual-hormone infusion therapy with potential to transform diabetes management
Zealand Pharma A/S (“Zealand”) (NASDAQ: ZEAL), a Copenhagen-based biotechnology company focused on the discovery and development of innovative peptide-based medicines, announced the completion of a USD 5 million strategic equity investment in Beta Bionics, a medical technology company developing the iLet™ bionic pancreas system. The iLet is a pocket-sized, dual-chamber, autonomous, glycemic control system that mimics a biological pancreas by calculating and dosing insulin and/or glucagon as needed based on data from the diabetic patient’s continuous glucose monitor. The iLet will utilize Zealand’s proprietary first-in-class stable glucagon analogue, dasiglucagon, in combination with insulin. This dual hormone therapy has the potential to significantly improve diabetes management for people who today are on insulin therapy.
In 2017, Zealand made an initial USD 1.5 million equity investment in Beta Bionics with an option to invest a further USD 3.5 million pending the achievement of specific development milestones. Zealand has now exercised this option based on the significant regulatory and clinical progress seen throughout 2018. This strategic equity investment, as part of the Series B financing, further strengthens the collaboration between Zealand Pharma and Beta Bionics in moving forward with the Phase 3 program for iLet in 2019.
Prior clinical studies have demonstrated that patients with type 1 diabetes using the autonomous dual hormone system spend significantly more time in the normal glucose range, had significantly fewer dangerous hypoglycemic events (low blood sugar), and reduced need for correcting the low blood glucose with excessive food intake (source: data on file).
“We are truly impressed with the clinical data generated so far with the iLet™ and excited by the progress in our collaboration with Beta Bionics. We look forward to starting the pivotal phase 3 development program in the second half of 2019,” said Adam Steensberg, Chief Medical and Development Officer at Zealand. “Zealand’s proprietary stable glucagon analogue, dasiglucagon, has great potential to dramatically improve Type 1 diabetes patient care and outcomes when used in cutting edge dual-hormone artificial pancreas pumps like the iLet.”
Zealand Pharma has demonstrated in multiple clinical trials that dasiglucagon is effective in preventing and correcting insulin-induced low plasma glucose events (hypoglycemia). With its unique stability profile in liquid formulations, dasiglucagon is strongly suited for use in dual-hormone artificial pancreas pump systems as already demonstrated with Beta Bionics’ iLet.
“Our enduring commitment at Beta Bionics is to bring the dual-hormone iLet to market to improve the lives of as many people with type 1 diabetes as possible,” said Ed Damiano, President and Chief Executive Officer at Beta Bionics. “We are very excited by the impressive results achieved with dasiglucagon in pre-clinical studies and clinical trials. We look forward to beginning our phase 3 pivotal trial program with the talented team at Zealand Pharma.”
Vectura hopes new respiratory device will breathe life into business
Vectura Group is pinning its future on a relatively new respiratory device aimed at treating several ailments beyond asthma, as the British drugmaker looks to revive its business after the costly acquisition of rival SkyePharma in 2016.
Vectura, which has partnerships with Bayer, Novartis and GSK, said on Thursday its 2018 adjusted core earnings would exceed analysts’ forecasts, citing rising sales of inhalers and improved margins.
Vectura’s shares, which fell more than 40 percent in 2018, closed 13 percent higher at 80 pence, giving the company a valuation of around 532 million pounds.
The company also said research and development expenses for 2019 would remain unchanged from its forecast of 45-55 million pounds, with 2018 investments at, or around, the bottom of the 55 million to 65 million pounds range.
Most of that cash is being spent on its “nebulised platform”, which is expected to treat common respiratory diseases such as asthma as well as other ailments, Chief Financial Officer Paul Fry told Reuters on Thursday.
Fry said the company’s new nebulised devices, which convert liquid medicine into mist that is inhaled by patients, would also focus on paediatric asthma, cardio pulmonary vascular disease and cystic fibrosis.
“We are looking to explore that platform in a number of different therapy areas like cystic fibrosis … we believe there is an important market opportunity and something that perhaps not many people are exploiting.”
Fry said Vectura was seeking a partner for its paediatric asthma treatment, but did not give details on any potential deal.
Vectura stopped developing a treatment for severe uncontrolled asthma in November after trials showed it failed to have a significant impact on the condition.
The company said then it would take an impairment charge related to the failure that would hit its 2018 pretax results by 40 million pounds.
While the company posted a 96.2 million pounds operating loss in 2017, due to an impairment charge related to previous acquisitions, it reported adjusted core earnings of 25.8 million pounds.
Fry said 2018 margins benefited as a larger proportion of sales of its biggest product Flutiform came from Japan and other high-margin regions.
Margins were also helped by fewer batches of products being written down and lower production costs due to changes in suppliers.
Fry added that Vectura’s deal with Hikma Pharmaceuticals for generic versions of GSK’s Ellipta portfolio also boosted revenue in 2018.
MIT Sloan professors’ model cuts liver transplant candidates’ deaths by 20%
Demand for liver transplants is much higher than organ supply, resulting in approximately 2,400 deaths every year. Also problematic is the current model used to identify and prioritize the “sickest” patients, which does not allow for equitable access to all waitlisted candidates, with a particular disadvantage to women. To address these issues, MIT Sloan School of Management Prof. Dimitris Bertsimasand Prof. Nikos Trichakis utilized machine learning to create a model that reduces mortality by 20%, averting nearly 400 deaths each year. Their model, Optimized Prediction of Mortality (OPOM), also provides a fairer and more equitable allocation to candidate groups, including women.
“There are many significant benefits to using this new model over the current system. Unlike the current system, which makes some arbitrary choices and results in bias against certain populations, OPOM’s methodology for prioritization is clear and understandable to surgeons — and it can save hundreds of additional lives every year,” says Bertsimas.
Trichakis noted, “OPOM fixes many of the current system’s problems because it was designed specifically for liver patients using real data. As a result, it can accurately prioritize patients across all populations without bias. This shows the potential of machine learning technology to help guide clinical practice and national policy on transplants.”
The researchers explain that the current model created in 2002 depends on the Model for End-Stage Liver Disease (MELD) score to rank disease severity and priority for receiving a liver transplant. As certain patient populations are at risk of death or of becoming too sick or unsuitable for transplantation based upon disease progressions that are not captured in their MELD score, the system arbitrarily grants them “exception” points. While the overall MELD score has led to a more objective ranking of candidates awaiting liver transplantation, the process of MELD exception point granting has resulted in inequitable and undesirable outcomes.
More specifically, the MELD exception points policy has disadvantaged women. “Data shows that women have historically had less access to liver transplantation and have had higher death rates on the wait list,” notes Trichakis. “This is due to the awarding of exception points to cancer patients, as more than 75% of those patients are men. Women also tend to have lower muscle mass and higher sodium levels, which lowers their MELD scores.”
Using a state-of-the-art machine learning method developed at the MIT Operations Research Center and real historical data from liver patients, the researchers sought a better way to prioritize the allocation of organs. With OPOM, they asked the question: What is the probability that a patient will either die or become unsuitable for liver transplantation within three months, given his or her individual characteristics?
They found that the OPOM allocation outperformed the MELD-based prediction method in terms of accuracy and fairness. In simulations, OPOM averted significantly more waitlist deaths and removed the bias against women. As a result, it allowed for more equitable and efficient allocation of liver transplants.
“Unlike MELD, which relies on an inexact approach of exception point assignment, OPOM allows for accurate prioritization of all candidates and removes bias for or against particular groups,” says Trichakis.
Bertsimas adds, “If we use this model to change how we measure mortality and allocate livers, the death rate will decrease by 20%, which is very significant. We’re hopeful that our findings will affect the national policy.”
Bertsimas and Trichakis are coauthors of “Development and validation of an Optimized Prediction of Mortality (OPOM) for candidates awaiting liver transplantation” with transplant surgeons Dr. Ryutaro Hirose of the University of California and Dr. Parsia A. Vagefi of the University of Southwestern Medical Center. Additional coauthors include MIT Sloan students Yuchen Wang and Jerry Kung. Their paper has appeared online in the American Journal for Transplantation.
DURECT Call to Provide Corporate Update on January 7, 2019
DURECT Corporation (Nasdaq: DRRX) invites interested parties to listen to a corporate update conference call that will be broadcast live over the internet on Monday, January 7, 2019 at 8:00 am Eastern Time (5:00 am Pacific Time).
A live audio webcast of the presentation will be available by accessing DURECT’s homepage at www.durect.com and clicking “Investor Relations.” If you are unable to participate during the live webcast, the call will be archived on DURECT’s website under Audio Archive in the “Investor Relations” section.
SCYNEXIS Provides Year-end Update and Outlines Plans for 2019
Initiated VANISH Phase 3 program of oral ibrexafungerp in acute VVC; top-line data expected in 1H 2020 with potential NDA filing in 2H 2020
Initiation of recurrent VVC Phase 3 trial planned for 1H 2019
Continued advancement of oral ibrexafungerp clinical development program in hospital-based invasive fungal infections
Received non-dilutive state incentive cash benefit of $6.7 million; approximately $51 million cash balance as of January 3, 2019, sufficient to ensure full funding of the VANISH Phase 3 VVC trials past top-line data
SCYNEXIS, Inc. (NASDAQ: SCYX), a biotechnology company delivering innovative therapies for difficult-to-treat and often life-threatening infections, today provided a year-end update and 2019 development plans for ibrexafungerp, an investigational antifungal agent and the first representative of a novel class of structurally-distinct glucan synthase inhibitors, triterpenoids.
“In 2018, we achieved multiple meaningful clinical milestones, most notably reporting positive data from our Phase 2b DOVE study evaluating oral ibrexafungerp for the treatment of vulvovaginal candidiasis (VVC),” said Marco Taglietti, M.D., President and Chief Executive Officer of SCYNEXIS. “The identification of a clinically and mycologically effective, well-tolerated oral dose of ibrexafungerp was a critical step for the initiation of our global Phase 3 program. The outcome of this registration program could be transformative for the millions of women with VVC not satisfied with existing therapies and also for SCYNEXIS, as ibrexafungerp could represent the first new antifungal class approved since 2001.”
Dr. Taglietti continued: “We are laser-focused on ensuring the efficient and timely completion of our VANISH Phase 3 trials to allow an NDA filing expected in the second half of 2020. We start 2019 with approximately $51 million in cash, sufficient to ensure the full funding of the VANISH Phase 3 trials past top-line results. We will continue to operate thoughtfully to progress the development of ibrexafungerp across multiple indications and to opportunistically explore commercial partnerships and other non-dilutive forms of cash injections, like the recent $6.7 million we received from the New Jersey Technology Business Tax Certificate Transfer (NOL) Program.”
Ibrexafungerp Development Update:
- Initiated VVC Phase 3 registration program. SCYNEXIS remains on track to report top-line data in 1H 2020, with potential New Drug Application (NDA) filing in 2H 2020.
- The VANISH Phase 3 program comprises two Phase 3 trials (approximately 350 patients each) designed to evaluate the safety and efficacy of one-day oral ibrexafungerp versus placebo for the treatment of VVC. Pending successful completion of these two trials, SCYNEXIS plans to file an initial NDA for oral ibrexafungerp for the treatment of VVC in 2H 2020.
- SCYNEXIS plans to initiate a third Phase 3 trial (approximately 350 patients) evaluating oral ibrexafungerp versus placebo in recurrent VVC in 1H 2019, an indication with no product currently approved.
- The Phase 3 program builds on the positive top-line data reported from the Phase 2bDOVE study in July 2018, which showed that the one-day oral ibrexafungerp dose selected for Phase 3 clinical evaluation was well-tolerated, with strong overall clinical and mycological activity and improved sustained effect compared to fluconazole, the current standard of care for VVC.
- If approved, ibrexafungerp would provide an oral option for millions of women not currently well-served by existing VVC therapies, most notably patients failing fluconazole or relapsing after treatment, with infections caused by fluconazole-resistant Candida spp., with difficult-to-treat symptoms, with recurrent VVC (for which no product is currently approved) and of child-bearing age concerned about fluconazole’s reported embryo/fetal toxicities.
- Continued advancement of oral ibrexafungerp clinical development in hospital-based invasive fungal infections.
- Site initiation activities continue to progress for the Phase 2 trial (SCYNERGIA) designed to evaluate the safety and efficacy of oral ibrexafungerp in combination with standard-of-care voriconazole in patients with invasive pulmonary aspergillosis. An animal model of pulmonary aspergillosis demonstrated improved outcomes and survival rates, supporting the potential superiority of ibrexafungerp in combination with azole therapy versus standard of care alone in this high-mortality indication.
- The FURI study, evaluating oral ibrexafungerp for the treatment of patients with invasive fungal infections refractory or resistant to standard of care, is ongoing. A preliminary assessment by a Data Review Committee (DRC) of the first 20 completed patients was recently conducted, and SCYNEXIS anticipates reporting top-line findings by February 2019.
- The CARES study, evaluating oral ibrexafungerp for the treatment of patients with Candida auris infections, is ongoing with several patients enrolled. C. auris is an emerging life-threatening and multidrug-resistant fungal pathogen, with a mortality rate of up to 60%. CARES is the first study assessing an investigational agent against this pathogen.
- While oral ibrexafungerp is progressing as a potential valuable option to treat hospital-based invasive fungal infections, SCYNEXIS continues the development of the intravenous liposomal formulation of ibrexafungerp and will provide further updates in the future.
Corporate Update:
- SCYNEXIS is committed to identifying non-dilutive forms of cash injections. Through the Technology Business Tax Certificate Transfer (NOL) Program in the state of New Jersey, SCYNEXIS recently obtained $6.7 million of non-dilutive funds.
- Considering SCYNEXIS’s worldwide rights to ibrexafungerp and patent protection until 2035, SCYNEXIS continues to explore business development partnerships to maximize ibrexafungerp’s commercial opportunity.
- As of January 3, 2019, SCYNEXIS has cash, cash equivalents and short-term investments of approximately $51 million. SCYNEXIS expects this will be sufficient to ensure full funding of the VANISH Phase 3 VVC trials past top-line results, expected in 1H 2020.
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