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Wednesday, June 1, 2022

Sage gets a postpartum reprieve

 Sage Therapeutics has finally had some good news: its Gaba-A modulator zuranolone produced a convincing win in Skylark, its pivotal postpartum depression study. The question is how much this will impress investors, who have their eye on the bigger use of major depressive disorder – and here there are still doubts about zuranolone’s approvability and commercial prospects. Sage and its partner Biogen have started a rolling submission in MDD, and plan a separate PPD filing early next year. One question is cost: Stifel analysts have speculated that Sage will price the drug for the larger MDD indication, although the company is staying tight-lipped for now. Sage estimates that around 500,000 women in the US have PPD, but only 28% of these are currently diagnosed; it hopes that an oral therapy will drive up this number. Sage already has a PPD drug in Zulresso, but that is given via a 60-hour infusion. The main downside in Skylark was a higher incidence of side effects like somnolence versus the lower-dose Robin trial, for arguably no additional clinical benefit. Sage reckons 50mg will be the go-to dose, with the option to reduce if required. The company’s stock climbed 4% this morning.

Flying high or wings clipped? Zuranolone's post-partum depression data
 Skylark (NCT04442503), 50mgRobin (NCT02978326), 30mg
HAMD-17 score change at…Placebo-adjusted differencep valuePlacebo-adjusted differencep value
 - day 3-3.40.0008-2.70.0255
 - day 15*-4.00.0007-4.20.0029
 - day 28-2.90.0203Not givenNot given
 - day 45-3.50.0067-4.10.0027
Somnolence26.5%N/A12.8%N/A
*Primary endpoint. Source: company releases & clinicaltrials.gov.

https://www.evaluate.com/vantage/articles/news/trial-results-snippets/sage-gets-postpartum-reprieve

Helius Launches MS Neurostimulation Patient Therapy Access Program

 Helius Medical Technologies, Inc. (Nasdaq:HSDT) (“Helius” or the “Company”), a neurotech company focused on neurological wellness, today announced the launch of its Patient Therapy Access Program (“PTAP”), which will provide qualifying patients access to on-label Portable Neuromodulation Stimulator (“PoNS”) Therapy at a significantly reduced price. PoNS is indicated in the U.S. for use as a short-term treatment of gait deficit in adults with mild-to-moderate symptoms from multiple sclerosis (“MS”) and is available by prescription only.

https://finance.yahoo.com/news/helius-medical-technologies-inc-announces-110500795.html

Tonix Gets U.S. Patent for Smallpox, Monkeypox Vaccine and Recombinant Pox Virus Tech

 Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP), a clinical-stage biopharmaceutical company, announced today that the United States Patent and Trademark Office (USPTO) issued U.S. Patent No. 11,345,896 to the Company on May 31, 2022. This patent, entitled "Synthetic Chimeric Poxviruses," includes claims covering synthetic horsepox virus, the basis for the Company’s TNX-8011 vaccine in development to protect against monkeypox and smallpox and for the Company’s Recombinant Pox Virus (RPV) platform to protect against other pathogens, including SARS-CoV-2. This patent is expected to provide Tonix with U.S. market exclusivity until 2037, excluding any possible patent term extensions or patent term adjustments.

https://finance.yahoo.com/news/tonix-pharmaceuticals-announces-issuance-u-110000909.html

Mexico totally bans sales of e-cigarettes

 Mexican President Andrés Manuel López Obrador signed a decree Tuesday outlawing the sale of e-cigarettes, continuing the government’s anti-vaping policy.

Mexico had already prohibited imports of the devices since at least October. And even before that, consumer protection and other laws had been used to discourage sales.

Despite Tuesday’s decree, many Mexicans import or buy vaping cartridges or fluid under the table or online domestically.

Assistant Health Secretary Hugo López Gatell lashed out at industry claims that vaping is safer than smoking, calling it “a big lie.”

The government’s own figures estimate that at least 5 million Mexicans have tried vaping at least once.

https://apnews.com/article/politics-health-caribbean-mexico-0379fcb161611f58588a6cc8de59c7fd

6 Things People Living with Alzheimer's and Other Dementia Want You to Know

 This June, during Alzheimer's & Brain Awareness Month, the Alzheimer's Association is revealing insights from people living with early-stage dementia and what they wish others knew about living with Alzheimer's and other dementia.

Many Americans struggle with what to say and do when a family member, friend, co-worker or neighbor is diagnosed with Alzheimer's disease or another dementia. The shock of someone revealing a dementia diagnosis can leave many at a loss for how to engage. Efforts to be supportive can be dampened by concerns of saying or doing the wrong thing. Worse, not knowing what to say or do, some individuals distance themselves from diagnosed individuals, further deepening the sadness, stigma and isolation people living with Alzheimer's and dementia can experience in the wake of a diagnosis.

The Alzheimer's Association recently asked those living with early-stage Alzheimer's and other dementia what they want others to know about living with disease. Here are six things they shared:

My Alzheimer's diagnosis does not define me. Although an Alzheimer's diagnosis is life changing, many living with the disease say their diagnosis does not change who they are. "I love the same people and doing the same things I did before my diagnosis," said Dale Rivard, 64, East Grand Forks, Minn. "I understand Alzheimer's is a progressive disease and I may not be able to do all the things I once did, but I want to continue doing the things I enjoy for as long as I can."

If you want to know how I am doing, just ask me. The sudden change in how others communicate with someone recently diagnosed with Alzheimer's or another dementia is a frustrating experience for many living with the disease. "It's upsetting to have others ask my wife how I am doing when I am sitting right there or nearby," said Jerry Smith, 78, Middleton, Wis. "I want to be open and honest about my diagnosis. Talking around me only makes me feel more isolated and alone."

Yes, younger people can have dementia. While the vast majority of Americans affected by Alzheimer's and other dementia are age 65 and older, the disease can affect younger individuals. While disease-related symptoms are similar, the challenges associated with an earlier diagnosis can be different. "I was diagnosed with dementia at age 53," said Deborah Jobe, 55, St. Louis, Mo. "I was at the peak of my career and had to step away from a job I loved. Suddenly, the plans I had for retirement with my husband looked very different. Most people just assume that Alzheimer's and dementia is only a diagnosis for old people, but I tell people if you have concerns about your cognition, get it checked regardless of your age."

Please don't debate my diagnosis or tell me I don't look like I have Alzheimer's. While family members and friends may be well-intended in attempting to dismiss an Alzheimer's diagnosis, many living with the disease say such responses can be offensive. "It's hard enough to tell someone you have Alzheimer's, let alone have to defend it," said Laurie Waters, 57, Clover, S.C. "It drives me crazy when someone tells me I am too young or that I don't look like I have Alzheimer's. People living with Alzheimer's all look different. You may not see my illness, but I live it every day."

Understand sometimes my words and actions are not me, it's my disease. As Alzheimer's disease and other dementia progresses, individuals can experience a wide range of disease-related behaviors, including anxiety, aggression and confusion. "I want people to understand that even though I may look myself, my disease sometimes causes me to not act myself," said Clint Kershaw, 61, North Turo, Mass. "So, if I ask for help doing something I once did easily or respond to a question in an unexpected way, be patient with me. I have good days and bad days, and on the bad days, I just need a little more help."

An Alzheimer's diagnosis does not mean my life is over. Earlier detection and diagnosis of Alzheimer's disease and other dementia is enabling diagnosed individuals more time to plan their futures and prioritize doing the things most important to them. "My diagnosis has been devastating, but my life is not over," said Nia Mostacero, 47, Meridian, Idaho. "I am still living a fulfilling life. I've put together my bucket list and intend to keep making memories for as long as I can."

During Alzheimer's & Brain Awareness Month in June, the Alzheimer's Association encourages everyone to learn more about disease-related challenges facing those living with Alzheimer's and other dementia. Educating yourself and others about the disease is one of the best ways to reduce stigma and misperceptions. The Alzheimer's Association offers guidance for navigating every stage of the disease. The Association's Live Well series provides tips to help early-stage individuals live their best lives. For other disease-related information and resources, visit alz.org.

Fight the Darkness of Alzheimer's on the Day with the Most Light, The Longest Day – June 21
During June, the Alzheimer's Association is also inviting people across the world to participate in The Longest Day® on June 21. Held annually on the summer solstice, The Longest Day invites participants to fight the darkness of Alzheimer's through a fundraising activity of their choice.

Throughout the month and culminating on June 21, Longest Day participants will bake, bike, hike, golf, knit, play bridge and participate in other favorite activities to raise funds and awareness for the care, support and research efforts of the Alzheimer's Association. For more information and to register: visit alz.org/thelongestday.

https://www.biospace.com/article/releases/six-things-people-living-with-alzheimer-s-disease-and-other-dementia-want-you-to-know/

Sanofi: FDA Grants Efanesoctocog Alfa Breakthrough Therapy Tag For Hemophilia A

 French drug major Sanofi (SNYNF, SNY) announced Wednesday that the U.S. Food and Drug Administration has granted Breakthrough Therapy designation to efanesoctocog alfa (BIVV001) for the treatment of people with hemophilia A.

Efanesoctocog alfa is the first factor VIII therapy to be awarded Breakthrough Therapy designation by the FDA.

Hemophilia A is a rare and life-threatening bleeding disorder.

Efanesoctocog alfa is a novel and investigational factor VIII therapy designed to provide normal to near-normal factor activity levels for the majority of the week in a once-weekly prophylactic treatment regimen.

The FDA decision is based on data from the pivotal XTEND-1 Phase 3 study. Topline results from the trial demonstrate efanesoctocog alfa met the primary endpoint, showing a clinically meaningful prevention of bleeds in people with severe hemophilia A over a 52-week period.

Breakthrough Therapy designation is designed to expedite the development and review of drugs in the US that target serious or life-threatening conditions.

Sanofi and Swedish biopharmaceutical company Sobi collaborate on the development and commercialization of efanesoctocog alfa, which is currently under clinical investigation. Its safety and efficacy have not been evaluated by any regulatory authority.

Sanofi has final development and commercialization rights in North America and all other regions in the world excluding the Sobi territory, essentially Europe, North Africa, Russia and most Middle Eastern markets.

John Reed, Global Head of Research and Development at Sanofi, said, "The Breakthrough Therapy designation highlights efanesoctocog alfa's potential to transform treatment for people with hemophilia A by providing higher protection for longer duration. This potential new class of factor VIII therapy represents how we are boldlyadvancing science to address unmet needs for the hemophilia community."

The company expects to share data from the XTEND-1 Phase 3 study at an upcoming medical meeting, and those data will serve as the basis for submission to FDA mid-year 2022.

The FDA granted efanesoctocog alfa Orphan Drug designation in August 2017 and Fast Track designation in February 2021.

The European Commission also granted efanesoctocog alfa Orphan Drug designation in June 2019. Regulatory submission in the EU will follow availability of data from the ongoing XTEND-Kids pediatric study, expected in 2023.

https://www.nasdaq.com/articles/sanofi%3A-fda-grants-efanesoctocog-alfa-breakthrough-therapy-designation-for-hemophilia-a

Letting vets down: VA backslides on wait times, employee fraud

 The agency charged with caring for America's veterans has been backsliding in performance after making improvements from a scandal a decade ago, as patient wait times grow and numerous of its employees are caught swindling taxpayers or fudging statistics.

The sheer breadth of problems inside the Veterans Affairs Department were laid bare this month in a lengthy semiannual report by its inspector general that identified nearly $4 billion in losses and problems in just six months.

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The issues ranged from a failure by the VA to recover more than $200 million in reimbursements from private medical providers to audacious criminal plots by employees and contractors ranging from stealing furnishings donated to homeless veterans to massive kickbacks. Investigations resulted in 104 arrests and more than 500 administrative actions in the first half of 2022.

Just before Memorial Day, for instance, Alisa Catoggio of Boca Raton, Fla., a former pharmacy technician and high-level executive assistant, pled guilty to taking part in a multimillion-dollar kickback scheme that went on for years undetected.

Catoggio admitted she participated in a scheme that paid approximately $40 million in kickbacks to patient recruiters in exchange for their referring prescriptions issued to a compounding pharmacy in Florida.

While inspections of individual medical centers showed some improvements in patient care, the inspector general still uncovered examples of egregious and deadly care of patients.

The VA hospital in Augusta, Ga., for instance, failed to detect the declining condition of a veteran after surgery, including malnutrition and alcohol withdrawal, before he died recently 

"Prior to the patient's surgery, primary care staff failed to provide sufficient care coordination and treatment," the report said. "A provider failed to address the patient's abnormal chest images and poor nutrition, and failed to communicate test results to the patient as required.

"A primary care nurse failed to respond to the patient's secure message request for assistance two days before surgery. Additionally, a barium swallow test was not scheduled. The surgical team completed a preoperative assessment but failed to detect the patient's overall poor health. During the patient's hospital stay after surgery, medical-surgical nurses did not consistently assess alcohol withdrawal symptoms or administer medications as required."

Members of Congress also are increasingly worried that patient wait times — the issue at the heart of the Obama-era scandal that started in Phoenix in 2014— are creeping back up, particularly for new patients.

website created by the VA to monitor patient service shows new patient wait times of 48 days at the Atlanta VA clinic, 88 days, at the Covington, Ga., facility, 25 to 50 days in the Phoenix area, 47 days at Scottsdale, Ariz., 66 days in Victorville, Calif., and 70 days in Valley Stream, N.Y.

To make matters even worse, some VA facilities are fudging wait times by failing to use the required calculations, an inspector general report revealed last month.

The Veterans Health Administration "has sometimes presented wait times with different methodologies, using inconsistent start dates that affect the overall calculations without clearly and accurately presenting that information to the public," the IG reported.

VA Secretary Denis McDonough has acknowledged the problems with honestly calculating wait times and lowering them for the benefit of veterans. 

"We have to do a better job with that," he told Congress  late last month. "We're working really hard on it because I am frustrated with it myself."

Peter O'Rourke, a former acting VA secretary under President Trump, said the slippage in wait times has "been a really sad thing to watch," after years of progress.

He said bureaucrats feel more emboldened under President Biden to revert back to old ways after years of pressure to innovate and meet performance standards.

"We're just seeing over and over again decisions being made, programs being reversed and really just the same kind of disappointing trajectory," he told the John Solomon Reports podcast.

The repeated failures inside the VA institutions have some candidates in the fall election running on plans to further privatize veterans' health care, allowing patients to skip clunky federal bureaucracies when they want.

"The VA is just really a disaster of a bureaucracy," said Joe Kent, a retired Green Beret deployed 12 times to war zones who is now running for a House seat in Washington state. 

"I think that the VA is almost beyond repair," he added. "We need to put choice in veterans' hands. We have a massive country — it's impossible for veterans to get the services and the help they need at just a few centralized, [VA-run] hospitals.

"We need to actually have veterans go get the healthcare they need on the economy, whatever is closest and the most convenient for them."

The agency also struggles with accounting. The inspector general found the VA did not recover more than $217 million in costs that were recoverable from private health providers who treated veterans for non-military related ailments, a massive sum that could have gone to additional care.

The VA "did not establish an effective process to ensure staff billed veterans' private health insurers as required," the watchdog reported. "An estimated 54 percent of billable community care claims paid between April 20, 2017, and October 31, 2020, were not submitted before filing deadlines expired."

But of all the issues flagged by the department's internal watchdog, none has ignited more anger than VA employees engaged in fraud and wrongdoing.

Just last week a former VA employee in Phoenix was sentenced for stealing property, mostly home furnishings, that had been donated by Walmart to the VA for use by homeless and destitute veterans.

Other convictions in the last few months include:

  • A former VA travel clerk pled guilty to a scheme that embezzled more than $487,000 in VA travel reimbursement funds, which he helped administer as part of his official duties.
  • The VA pharmacy chief at the Erie, Pa., medical center was sentenced to two years of probation for unlawfully obtained multiple dosage units of hydrocodone and oxycodone from 2017 to 2020 from pill bottles awaiting delivery to VA patients. 
  • The former chief of cardiology at the Palo Alto, Calif., VA hospital pled guilty to sexual battery for repeatedly subjecting a subordinate to unwanted and unwelcome sexual contact, forcing the victim to resign her job.
  • A total of 17 doctors and two healthcare executives involved in a kickback scheme for VA patients have agreed on settlements and paying $2.7 million in penalties.