Belgium’s UCB raises 2019 forecasts after net sales beat in fourth quarter

Belgian drugmaker UCB raised its revenue and earnings per share guidance for 2019 on Friday, helped by better-than-expected net sales due to strong demand for its Cimzia and Vimpat drugs in the fourth quarter.

Full-year revenue is expected now at about 4.9 billion euros (4.2 billion pounds), above the previously expected range of 4.6–4.7 billion euros, and UCB targets now EPS higher than 4.80 euro, it said in a statement.

The company, which now also sees recurring core profit at least at the upper end of its range of 27-29%, said it funded additional marketing activities to optimize the growth opportunity.

https://www.marketscreener.com/UCB-5963/news/UCB-Belgium-s-UCB-raises-2019-forecasts-after-net-sales-beat-in-fourth-quarter-29819126/

Cal., Others Develop Schemes to Enter Generic Market and Drive Down Prices

Generic drugs and their biologic cousins, biosimilars, are viewed as a way to drive down the costs of drugs and provide savings to consumers and healthcare providers. The State of California is considering a measure to sell its own brand of generic drugs in an effort to get in on those cost-savings.

California’s Gov. Gavin Newsom is expected to reveal the scheme today in his new state budget. Newson’s office has released the broad outlines of the plan, which would have the state contracting with one or more generic drug companies to manufacture specific prescriptions under the state’s label. They would then be sold to Californians at a lower cost.

“It’s an interesting idea without there being any specifics,” Craig Garthwaite, director of the healthcare program at Northwestern University’s Kellogg School of Management, told the Los Angeles Times. “The question is: What is the goal? Is it to decrease aggregate spending on drugs or fix market failures?”

The California plan is the first state-run generic program to date, although there are a number of other state-related plans as well as federal programs and healthcare organizations looking at ways of getting into the generic market.

At the federal level, much of the most recent efforts have been on allowing consumers to buy drugs legally from Canada. In addition to the announcement of a notice of proposed rulemaking (NPRM), the administration announced the availability of a new draft guidance for the biopharma industry that laid out procedures they can follow to facilitate importation of prescription drugs, including biologics that are FDA approved, manufactured outside the U.S., and authorized for sale in any foreign country, and were originally intended to be marketed in that country.

In October 2019, the FDA announced that its efforts to increase drug competition via generic drugs had resulted in a record number of generic drug approvals this year. For fiscal year 2019, the agency reported 1,171 generic drug approvals, 935 of them full approvals and 236 tentative approvals. This is up from last year’s all-time record of 971 generic drug approvals.

That announcement came only a day after it was reported that Teva Pharmaceuticals was discontinuing manufacture of its generic version of vincristine, a chemotherapy drug vital to the treatment of childhood cancers, including leukemias, lymphomas and brain tumors. In this case, Pfizer manufactures the only branded version of the chemotherapy agent, which has physicians worried this will result in shortages.

The Center for Biosimilars noted, “Such care interruptions have become more common, due to shortages of older, off-patent drugs and insufficient generic competition. Besides creating patient care issues, the shortages also limit potential cost savings.”

Perhaps the most interesting and successful effort has been Civica Rx, which was launched in January 2018 by five healthcare systems as a not-for-profit generic drug company. The companies were the U.S. Department of Veterans Affairs (VA), Intermountain Healthcare, Ascension, SSM Health and Trinity Health. Since then, more than 1,000 hospitals in 46 states joined Civica Rx.

Civica Rx was to initially focus on establishing price transparency and stable supplies of 14 generic drugs often used in hospitals. The idea was to depend on long-term contracts member organizations sign stating they will buy a fixed percentage of their drug volume from Civica Rx. One focus was on drugs that had a 50% or greater price increase between 2014 and 2016 and essential drugs that were on national shortage lists.

In May 2019, Civica Rx signed its first produce supply agreement with Copenhagen, Denmark-based Xellia Pharmaceuticals. Xellia will manufacture essential antibiotics, including Vancomycin and Daptomycin, for Civica’s members.

In July, Civica Rx announced a five-year deal with London, UK-based Hikma Pharmaceuticals to manufacture and supply Civica’s members with 14 sterile injectable drugs as a private label distributor. Hikma will also use its Abbreviated New Drug Applications (ANDAs) and Civica’s labeling and National Drug Code (NDC).

Hikma has a portfolio of more than 100 injectable drugs. It is the third largest supplier of generic injectables to the U.S.

Other states have made alliances with pharmaceutical companies to eradicate hepatitis C infections. These include AbbVie and the State of Washington, and Louisiana and Gilead Sciences subsidiary Asegua Therapeutics. However, partly because the bidding for these contracts remain confidential, critics are questioning the secrecy and whether these deals actually are financially beneficial.

The Louisiana deal, in what is being called a “Netflix model,” the (WSHCA) inked a contract with AbbVie with a goal of eliminating hepatitis C virus (HCV) in the state by 2030. AbbVie won the contract, but the price and details of the deals are confidential under the Washington state Public Records Act, as well as federal rules that hide drug prices related to them being company trade secrets.

Critics note that without transparency, it’s impossible to know whether this is a good public policy or just a great deal for drug companies. USA Today wrote that these deals could “primarily benefit manufacturers hoping to lock down payments of perhaps $10,000 per patient for drugs from Medicaid. The same drugs from the same manufacturers can cost less than $100 per course of treatment in other parts of the world.”

In terms of the California proposal, Gov. Newsom stated, “The cost of healthcare is just too damn high, and California is fighting back.”

Trish Riley, executive director of the National Academy for State Health Policy, told the Los Angeles Times, “Every state has been frustrated with high drug prices. They want to balance their budgets and these drug price spikes make it difficult. This could have a tremendous impact.”

Riley points out that although no other state has tried to cut out the supply chain for generic drugs, Civica Rx is a smaller-scale model California might emulate. “It’s an example of what aggressive purchasing can accomplish.”

Although details are still pending, that hasn’t stopped critics.

“Frankly, I think it’s a ludicrous proposal that demonstrates a profound misunderstanding of generic drug economics,” Adam Fein, chief executive of the Drug Channels Institute, told the Los Angeles Times. “It’s like saying you want to go to Post [Consumer Brands] for your Fruity Pebbles and open a supermarket to buy them. It doesn’t make sense.”

https://www.biospace.com/article/california-considering-entering-the-generic-drug-market/

FDA to Pull Nearly 250 ANDAs, Continues to Revamp Office of New Drugs

With a new year underway, the U.S.Food and Drug Administration (FDA) is doing some house-cleaning of sorts. The regulatory agency said it intends to pull nearly 250 Abbreviated New Drug Applications (ANDAs) following the failure of manufacturers to submit annual reports.

First reported by Regulatory Focus, the publication of the Regulatory Affairs Professional Society (RAPS), the FDA is pulling 249 ANDAs from the generic drugmakers. According to the report, the FDA said the manufacturers will be able to request a hearing to appeal the agency’s decision to pull the ANDAs. The manufacturing companies will have to prove the regulatory agency with a strong reason for why those ANDAs should not be pulled.

“A request for a hearing may not rest upon mere allegations or denials but must present specific facts showing there is a genuine and substantial issue of fact that requires a hearing,” the FDA said, according to the RAPS report. The FDA added that it could deny the hearing if the request from the manufacturer is incomplete or not supported by those specific facts required.

Most companies affected by the action are smaller manufacturers, such as Alra Laboratories and American Therapeutics, which have 28 and 26 proposed ANDAs set to be withdrawn, respectively. There are some larger names that can be found in the list, including Pfizer, which has two ANDAs, Valeant Pharmaceuticals, which has two, Abbott, which has two, and Fresenius USA and Sandoz, which both have on ANDA that would be withdrawn.

In addition to the pulling of the ANDAs, the FDA is also seeking input on ways to improve its Office of New Drugs. According to RAPS, companies like Pfizer, Regeneron and Amgen are weighing in on the reorganization, which was first announced in the fall. The report said the drugmakers provided input on the use of more case studies in clinical trial design, as well as the concern over inconsistent applications of the guidelines.

In its submitted comments, Pfizer said it has seen a “wide variation in actual expectations across divisions” in applying guidance to safety database. Pfizer said it has also seen inconsistencies regarding the “‘waiving’ collection/submission of non-serious adverse events for drugs that have a well-characterized safety profile,” RAPS said.

In its comments to the FDA, Regeneron remained focused on the broader use of genomic data, according to the report. The Tarrytown, N.Y.-based company said the FDA should consider using genomic information “as a component of the efficacy and safety evaluation of new drugs, and fundamentally, as a valued source of substantial evidence in regulatory decision-making,” RAPS reported.

Sanofi asked the FDA to include more analyses of data that underlies its regulatory decision-making. That way, companies can be better informed regarding their own strategies. Amgen asked for the broader sharing of “accumulated FDA experience” through the publication of FAQs or with the addition of a Q&A section on its website regarding guidance, RAPS said.

Finally, professors of health policy and economics are urging Congress to provide the FDA with greater resources to combat what they see as procedural abuses of the Orphan Drug Act. Rebecca Haffajee of the University of Michigan and Harvard Medical School’s Richard Frank wrote in the New England Journal of Medicine of the need to curb abuses of the act, such as the filing of “sham citizen petitions” that will extend the market exclusivity of drugs for rare diseases. The academic duo specifically pointed to Reckitt Benckiser’s Suboxone, a treatment for opioid addiction. The article points how Reckitt, which demerged from Indivior, the owner of the drug, abused the orphan drug status to earn billions of extra dollars through preventing other companies from marketing challengers in the U.S. Indivior has been indicted by the U.S. Department of Justice for its marketing practices.

To prevent additional abuse of the Orphan Drug Act, the duo said Congress should prohibit the “’grandfathering’ of orphan drugs and require the FDA to base qualifying economic-recoupment determinations for orphan drugs on unbiased sales projections and to revoke designations if revenues exceed projected sales,” RAPS reported. Additionally, Congress should alter the filing practices for citizen petitions, Haffajee and Frank wrote.

https://www.biospace.com/article/fda-to-pull-nearly-250-andas-continues-to-revamp-ond/

HCA acquires Valify

HCA Healthcare (NYSE:HCA) has acquired healthcare accounts payable analytics provider Valify for an undisclosed sum.

The Frisco, TX-based firm has developed a web-based platform that provides healthcare systems with in-depth analyses across a range of service categories.

https://seekingalpha.com/news/3531090-hca-acquires-valify

Class of antibiotics could be potential treatment for frontotemporal dementia

Researchers at the University of Kentucky’s College of Medicine have found that a class of antibiotics called aminoglycosides could be a promising treatment for frontotemporal dementia.

Results of their proof of concept study, which was a collaborative effort between UK’s Department of Molecular and Cellular Biochemistry and the University of California San Francisco’s Department of Pathology, were recently published in the journal, Human Molecular Genetics.

Frontotemporal dementia is the second-most common dementia after Alzheimer’s disease and the most common type of early onset dementia. It typically begins between ages 40 and 65 and affects the frontal and temporal lobes of the brain, which leads to behavior changes, difficulty speaking and writing, and memory deterioration.

A subgroup of patients with frontotemporal dementia have a specific genetic mutation that prevents brain cells from making a protein called progranulin. Although progranulin is not widely understood, its absence is linked to the disease.

A group led by Haining Zhu, a professor in UK’s Department of Molecular and Cellular Biochemistry, discovered that after aminoglycoside antibiotics were added to neuronal cells with this mutation, the cells started making the full-length progranulin protein by skipping the mutation.

These patients’ brain cells have a mutation that prevents progranulin from being made. The team found that by adding a small antibiotic molecule to the cells, they could ‘trick’ the cellular machinery into making it.”

Matthew Gentry, co-author of the study and the Antonio S. Turco Endowed Professor in the Department of Molecular and Cellular Biochemistry

The researchers found two specific aminoglycoside antibiotics – Gentamicin and G418 – were both effective in fixing the mutation and making the functional progranulin protein. After adding Gentamicin or G418 molecules to the affected cells, the progranulin protein level was recovered up to about 50 to 60%.

These results could be promising to drug development. Currently, there are no effective therapies for any type of dementia.

After this preclinical proof of concept study, the next step is to study the antibiotics’ effects on mice with the mutation that causes frontotemporal dementia, Zhu says. Another focus is to possibly develop new compounds from Gentamicin and G418 that could be safer and more effective. Although Gentamicin is an FDA-approved medication, its clinical usage is limited as it is associated with a number of adverse side effects.

“If we can get the right resources and physician to work with, we could potentially repurpose this drug. This is an early stage of the study, but it provides an important proof of concept that these aminoglycoside antibiotics or their derivatives can be a therapeutic avenue for frontotemporal dementia,” said Zhu.

Source:

University of Kentucky

Journal reference:

Kuang, L., et al. (2020) Frontotemporal dementia nonsense mutation of progranulin rescued by aminoglycosides. Human Molecular Genetics. doi.org/10.1093/hmg/ddz280.

https://www.news-medical.net/news/20200110/A-class-of-antibiotics-could-be-potential-treatment-for-frontotemporal-dementia.aspx

Physicians expect almost one-third of their jobs to be automated by 2040

Doctors say digital technology and data are driving change that will create a different world of medicine in the next couple of decades, a new report from Stanford Medicine finds.

In a survey, physicians, residents and medical students say they expect almost a third of their current duties could be automated in 20 years. And doctors are preparing for that very different healthcare future now, according to the report (PDF).

Nearly half of physicians (47%) and most medical students (73%) are seeking additional training in areas such as advanced statistics, genetic counseling, population health and coding. One-third are studying artificial intelligence, according to the national survey of more than 700 physicians, residents and medical students commissioned by Stanford Medicine to understand how changing trends will reach the doctor’s office and shape patient care.

“We found that current and future physicians are not only open to new technologies but are actively seeking training in subjects such as data science to enhance care for their patients,” said Lloyd Minor, M.D., dean of the Stanford University School of Medicine, in a statement.

“We are encouraged by these findings and the opportunity they present to improve patient outcomes. At the same time, we must be clear-eyed about the challenges that may stymie progress,” he said.

Key trends that are reshaping healthcare include a maturing digital health market, new health laws opening patient access to data and AI gaining regulatory traction for medical use.

And the jury’s still out when it comes to whether the private industry’s foray into healthcare—in the form of companies such as Amazon, Google and Apple— will solve problems.

Physicians, residents and students had mixed views about the impact these companies will have on healthcare, with 30% of students and residents and 21% of physicians still undecided. While patient outcomes are likely to improve, respondents are divided on whether physician effectiveness will improve and say physician job satisfaction will likely decrease, while healthcare costs likely increase.

Other findings include:

The value of data. The survey also showed that providers are heavy digital users and they believe patient data from wearables can be clinically valuable. Nearly half the survey respondents wear a health monitoring device, and most of them use the data to inform their personal healthcare decisions (71% of physicians, 60% of students and residents). A majority of students and residents (78%) and physicians (80%) say self-reported data from a patient’s health app would be clinically valuable in supporting their care. They also see value in data from consumer genetic testing reports.

Doctors aren’t prepared to implement innovations. However, most providers don’t believe the current generation of practitioners is ready for the data-driven future, even current medical students and residents. When asked to rate the effectiveness of their education to prepare them for these developments, only 18% of current medical students and residents surveyed said that their education was “very helpful.” And 44% of physicians surveyed said their education was either “not very helpful” or “not helpful at all.”

The report pointed to the need to modernize curriculum and training programs so current and future physicians can make the most of new technologies.

The ongoing struggle with medical practice burdens. And, no surprise, physicians and residents say they are struggling under medical practice burdens. Nearly 1 in 5 would change their career path if given the opportunity, citing poor work-life balance and administrative burdens as the top reasons to reconsider their decision.

https://www.fiercehealthcare.com/practices/20-years-physicians-expect-almost-one-third-their-job-to-be-automated-stanford-medicine

Alnylam initiates U.S. application for lumasiran for PH1

Alnylam Pharmaceuticals (NASDAQ:ALNY) initiates its rolling New Drug Application (NDA) in the U.S. for lumasiran for the treatment of primary hyperoxaluria type 1 (PH1). Specifically, it has submitted the non-clinical components. It expects to complete the filing this quarter.

https://seekingalpha.com/news/3531070-alnylam-launches-u-s-application-for-lumasiran-for-ph1