Trial met its primary endpoint, with 64.3% of patients self-administering etripamil converting to sinus rhythm within 30 minutes compared to 31.2% on placebo (HR = 2.62, p<0.001)
─ Consistent safety and tolerability data support potential self-administration of etripamil
─ Analyses of pooled data show statistically significant reduction in medical interventions and visits to the emergency department
─ Company plans to submit an NDA to the U.S. FDA in mid-2023
─ Conference call and webcast to be held today at 8:00 a.m. Eastern Time
Milestone will host a conference call and webcast to discuss the results of the RAPID trial today, October 17, 2022 at 8:00 a.m. ET. To access the live call by phone, dial (877) 870-4263 (domestic) or (412) 317-0790 (international); the conference ID is 10171280. A live audio webcast of the event and accompanying slides may also be accessed through the "Investors" section of Milestone's website at www.milestonepharma.com. A replay of the webcast will be available for one year following the event.
esignation Validates Alvelestat’s Potential to Address a Serious Unmet Need
Company Plans R&D Update on Alvelestat in AATD-associated Lung Disease on October 31, 2022
Mereo BioPharma Group plc (NASDAQ: MREO), (“Mereo” or the “Company”), a clinical-stage biopharmaceutical company focused on rare diseases and oncology, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for its investigational oral neutrophil elastase inhibitor, alvelestat (MPH-966). Mereo intends to have an End-of-Phase 2 meeting with the FDA to discuss the design of a registration-enabling study for alvelestat as a treatment for AATD-associated lung disease, including the potential opportunity for an accelerated approval pathway, around the end of the year.
Minerva Neurosciences, Inc.(Nasdaq: NERV), a clinical-stage biopharmaceutical company focused on the development of therapies to treat central nervous system (CNS) disorders, today announced that the company has received a refusal to file letter from the U.S. Food and Drug Administration (FDA) regarding the company’s New Drug Application (NDA) for roluperidone for the treatment of negative symptoms in patients with schizophrenia. The FDA has indicated that the company can request a Type A meeting to discuss the content of the refusal to file letter.
“We are disappointed that the FDA has not accepted our NDA for roluperidone. Our goal remains to provide a new and much needed therapeutic option to help patients and their families, since there are currently no approved therapies to treat negative symptoms of schizophrenia in the United States,” said Remy Luthringer, Executive Chairman and Chief Executive Officer of Minerva. “The company intends to request a Type A meeting and looks forward to continued discussions with the FDA.”
The U.S. Food and Drug Administration has extended the review of Biogen Inc's experimental treatment for an inherited form of amyotrophic lateral sclerosis (ALS) by three months, the company said on Monday.
Biogen said it had submitted responses to the FDA's information requests, which the agency said would require additional time for review and set a new target action date of April 25, 2023.
The agency had accepted Biogen's application for the drug, tofersen, under its accelerated approval pathway in July this year, with an approval decision expected by Jan. 25.
Last year, the drug had failed to meet the main goal of a late-stage study and did not show statistically significant improvement in the functional status of patients with fast-progressing ALS.
Last month, Biogen released new analysis that showed that longer-term use and an early initiation of tofersen was effective in slowing disease progression.
ALS, also known as Lou Gehrig's disease, breaks down nerve cells in the brain and spinal cord that make muscles work, leading to progressive paralysis and death. Its cause is largely unknown.
Biogen is seeking approval of tofersen for ALS patients with mutations in a specific gene that leads to accumulation of toxic levels of a protein called SOD1.
One year ago,Gov. Hochulmet with grieving families who lost loved ones in nursing homes during the COVID-19 pandemic. I was with my husband, Sean, whose parents, Mickey and Dee, were two of more than 15,000 seniors who died from contracting the virus in their long-term-care facilities. I remember feeling optimistic that if she heard our stories and saw our grief first hand, she would help us find answers. The day of the meeting she listened to us, offered her condolences and promised to be fully transparent, helping us get to the bottom of what happened in spring 2020. She apologized to us and told reporters after our meeting that she “gives a damn.”
At the time, I believed her.
But now, more than 2 1/2 years later, we still don’t know why then-Gov. Andrew Cuomo and the state Department of Health ordered more than 9,000 COVID-positive patients into their residences without alerting our families. The administration never took responsibility for its reckless decisions and death-toll coverup. There is now strong evidence that Cuomo purposely hid those numbers to profit from a $5 million book he was writing.
Where’s the panel?
Hochul listened sympathetically to my husband, Sean, as he painfully explained what it was like losing both his parents within two weeks of each other and not being able to see or comfort them. My friends, Peter and Daniel Arbeeny, gave Hochul their father’s death certificate and asked why he wasn’t counted in the whitewash of nursing-home fatalities. I commented after our meeting that Hochul’s actions would speak louder than words, and a year later we have seen nothing to indicate she has been true to her promise. Her “blue-ribbon panel” into the “good, bad and ugly” decisions made during the pandemic has never happened.
Peter Arbeeny, a lifelong Democrat, says he often wonders: If Cuomo were a Republican, would we “still be here two years later waiting for the Democratic machine to start an investigation with subpoena power?”
Rep. Lee Zeldin, the Republican running for governor against Hochul, has promised families if he wins to “appoint a special prosecution my first day in office to investigate and prosecute any crimes regarding the order and the cover up.” That’s something that should happen regardless of who wins in November.
My friend and fellow advocate, Assemblyman Ron Kim, who helped facilitate the meeting between grieving families and Hochul last October, is still holding out hope that she will be true to her word, but argues she can’t choose consultants to do the report. It needs to be an “independent commission with full subpoena and investigative powers. This cannot be like the 2020 DOH report that went back to then-Governor Cuomo where he controlled the facts and the narrative.”
In the meantime, families like mine are trying to figure out what we can do on our own. One thing we’ve been trying to raise awareness about is the passing of Bill S74A, known as the Grieving Families Act. This legislation would “help in future lawsuits when a loved one’s life wrongfully ends and consider the emotional loss when awarding damages.” The bill would also increase the statute of limitations to file a claim, giving families the chance to have their day in court. However, that also requires action and a signature from one Gov. Kathy Hochul.
I wish I could be more optimistic, but that same woman who looked us in the eye and told us she cared and wanted to help has so far done absolutely nothing to make us believe her.
Have you taken a peek at the balance in your 401(k) retirement accounts lately? Here’s our advice: Don’t bother. It will ruin your whole day, week and month.
Here’s why: We’ve now had seven straight months of 8%+ inflation. A year ago we were assured by the White House economic wizards that these rapid price increases in everything from groceries, to rental cars, to gasoline at the pump, to health insurance were merely “transitory.” Whoops.
The most immediate sticker shock from Bidenflation, of course, has been to shrink real take-home paychecks of workers. We have calculated that over the past 20 months, this rise in consumer prices over wages means that the average family in America has lost nearly $6,000 in purchasing power. This from the Lunch Bucket Joe president who promised to help boost the incomes of the middle class. When, exactly?
But this pay-cut effect on family incomes is only part of the curse of runaway inflation. We’ve just completed an analysis of how the highest inflation rate in almost 40 years has impacted the retirement funds of ordinary Americans. Here is what we found.
Since President Biden took office, monthly savings have collapsed, falling 83%.Yuri Gripas – Pool via CNP / MEGA
Savings collapse
Not surprisingly, since President Biden took office, monthly savings have collapsed, falling 83%. (We could never understand how Biden could say with a straight face that Americans are saving more. His “transformation” of the US economy has had just the opposite effect.) Many millions of Americans who are living paycheck to paycheck just don’t have the money after paying the inflated bills to save much.
But to add insult to injury, even what has been already saved and invested by older Americans over past years and even over several decades has been erased from these accounts.
Thanks to the thief of inflation.
The increase in consumer prices over wages means that the average family in America has lost nearly $6,000 in purchasing power.NY Post
Most of the 150 million Americans with one form or another of retirement savings have invested the majority of those tens of thousands of dollars in stocks. The major stock indices are all way down since Biden came into office. Here are the returns as of Oct. 10, according to the Federal Reserve Bank of St. Louis:
Dow Jones Industrial Average: -6% NASDAQ: -18% S&P 500: -6%
These negative returns don’t even take account of inflation. Doing so adds roughly another 13% or so to these stock losses. Inflation also hurts returns from bonds — which typically account for between 20% and 40% of retirement fund investments. That is because, as we are now seeing, higher inflation means higher interest rates, which lower the value of the bonds you own.
Little guys hurt
Tie it all together and we calculate that since the start of this year, 401(k) plans have suffered $2.1 trillion in losses. The average 401(k) plan had over $135,000 at the start of this year.
Today, those assets have shrunk on average to about $101,000. In other words, the average 401(k) plan is down about $34,000 — more than 25% in less than one year!
(In terms of purchasing power, inflation also has brought the “real value,” in 2021 dollars, of the average 401(k) down another $5,000, from $101,000 to $96,000).
Let’s say you are doing a little better than average financially and you have a 401(k) plan with $300,000 saved up. You’re still far from “rich.” Your nest egg losses this year are likely to be above $75,000.
There goes the down payment on that retirement home or condo in Florida or Arizona. But these numbers don’t even take account of losses in other pension/retirement accounts.
Bidenflation is taking a toll on Americans who are living paycheck to paycheck.AP/Carolyn Kaster
More traditional pension funds also are getting flattened by inflation. At the beginning of this year, pensions in the US had $27.8 trillion in assets. Now, it’s under $24 trillion, a drop of about 15% that has wiped out the last two years’ worth of gains — nearly $4 trillion. Many union and government pension funds were already facing financial shortfalls to be able to pay promised benefits. The combination of high inflation and a bear market in stocks means insolvency is a real threat. Some may need bailouts or will have to sharply cut promised benefits.
A year ago, the White House insultingly tweeted out that inflation is merely “a high-class problem.” Wrong. The victims of ever higher prices at the store and the gas pump are not the millionaires, but the little guys — and, in particular, older Americans — whose paychecks and savings accounts get walloped.
It’s not exactly the same as a bank robber with a gun stealing a quarter of the money in your bank account. But at the end of the day, Bidenflation has had the exact same unhappy result.
Stephen Moore and E..J. Antoni are economists at The Heritage Foundation. Moore is a co-founder at Committee to Unleash Prosperity, where Antoni is a senior fellow.
The Bill and Melinda Gates Foundation says it will commit $1.2 billion to the effort to end polio worldwide.
The money will be used to help implement the Global Polio Eradication Initiative’s strategy through 2026. The initiative is trying to end the polio virus in Pakistan and Afghanistan, the last two endemic countries, the foundation said in a statement Sunday.
The money also will be used to stop outbreaks of new variants of the virus. The announcement was made Sunday at the World Health Summit in Berlin.
The foundation says in a statement on its website that it has contributed nearly $5 billion to the polio eradication initiative. The initiative is trying to integrate polio campaigns into broader health services, while it scales up use of the novel oral polio vaccine type 2.
The group also is working to make national health systems stronger so countries are better prepared for future health threats, the statement said.
“The last steps to eradication are by far the toughest. But our foundation remains dedicated to a polio-free future, and we’re optimistic that we will see it soon,” said foundation CEO Mark Suzman.
Pakistan has reported 20 polio cases so far this year, all in the north-western Khyber Pakhtunkhwa province.
Afghanistan, which has registered two cases this year, previously lacked access to vaccines because of violence and the Taliban banning polio teams in areas under its control. However last year, a few months after they took over Afghanistan, the Taliban agreed to allow United Nations health workers to begin a national campaign.
Pakistan has long struggled with Islamic militants targeting polio workers and the police protecting them, falsely claiming that vaccinations are a Western campaign to sterilize children. This year, it has the added challenge of unprecedented rainfall destroying road networks and health facilities, limiting vaccination drives, and displacing communities.
Despite the billions of dollars that have gone into the effort to eradicate polio since 1988 — the program costs about $1 billion every year — the World Health Organization and partners have missed repeated deadlines to wipe out the disease and have come under sustained criticism for failing to adapt to challenges. In recent years, for example, there have been more cases of polio linked to the oral vaccine used in eradication efforts than those caused by the wild virus.
Numerous experts have also questioned whether more money is what’s needed to eradicate polio, as the initiative is already one of the best funded in global public health and has rarely faced any funding gaps. Although WHO and partners have reduced the incidence of polio by more than 99%, that progress was largely made in the first 10 years. The disease remains stubbornly entrenched in war-torn regions of Afghanistan and Pakistan and there have been dozens of vaccine-triggered outbreaks in Africa and elsewhere in recent years, including the U.S. and Israel.
An independent panel formed to evaluate the eradication effort’s progress has repeatedly identified significant strategic mistakes made by countries, WHO and their donors, warning that their reluctance to change course, among other issues, may ultimately allow polio to resurge.
The eradication initiative is a public-private partnership led by a group of national governments that includesthe Gates Foundation, Rotary International, the World Health Organization and the U.S. Centers for Disease Control and Prevention.
