Kazia Therapeutics Limited (NASDAQ: KZIA; ASX: KZA), an oncology-focused drug development company, is pleased to announce the presentation of promising new data from an ongoing phase I clinical trial of paxalisib in combination with radiotherapy for the treatment of brain metastases, sponsored by Memorial Sloan Kettering Cancer Center inNew York, NY.
Interim data from the first stage of the study reports that all 9 evaluable patients experienced complete or partial response, representing an overall response rate (ORR) of 100%. For comparison, a typical ORR associated with whole brain radiotherapy alone can commonly range from 20-45% in published studies, of which some representative examples are indicated below.
The data has been accepted for an oral presentation at the upcoming 2022 Annual Conference on CNS Clinical Trials and Brain Metastases, jointly organised by the Society for Neuro-Oncology (SNO) and the American Society for Clinical Oncology (ASCO), and held in Toronto, Canada from 12-13 August 2022.
The U.S. Food and Drug Administration declined to approve expanded use of Acadia Pharmaceuticals Inc's antipsychotic drug for treating psychosis related to Alzheimer's disease, the company said on Thursday.
The health regulator concluded there were limitations in the interpretability of Acadia's antipsychotic drug study and an additional study would be required, according to the company.
The U.S. regulator's decision comes roughly a month after its panel of outside advisors voted 9-3 that available data does not support conclusion that the drug, pimavanserin, was effective for treatment of hallucinations and delusions in Alzheimer's disease.
"We are disappointed with this outcome. The treatment of Alzheimer's disease psychosis continues to be an area of high unmet need, for which there is no approved therapy," Acadia Chief Executive Officer Steve Davis said.
This was Acadia's second attempt to secure approval for expanded use of the drug. In April 2021, the FDA told Acadia its clinical trial data was not sufficient to support approval of pimavanserin in patients with dementia-related psychosis.
The company resubmitted its application in February with a focus on Alzheimer's patients.
The drug, sold under the brand name Nuplazid, is already approved for a similar indication in patients with Parkinson's disease and brought in sales of $484 million last year.
Pre-NDA (New Drug Application) Meeting with the U.S. Food and Drug Administration (FDA) Scheduled for the Third Quarter of 2022 to Discuss NDA Submission of Reproxalap for the Treatment of Dry Eye Disease
Pre-NDA Meeting with the FDA Planned for the Second Half of 2022 to Discuss NDA Submission of ADX-2191 for the Treatment of Primary Vitreoretinal Lymphoma
Top-line Results from Phase 3 GUARD Trial of ADX-2191 in Proliferative Vitreoretinopathy Expected in the Second Half of 2022
Top-line Results from Phase 2 Clinical Trial of Oral RASP Modulator ADX-629 in Acute Alcoholic Hepatitis Expected in the Second Half of 2022
Cash, Cash Equivalents, and Marketable Securities of $196.7 Million as of June 30, 2022
Management to Host Conference Call at 8:00 a.m. ET Today
Aldeyra will host a conference call at 8:00 a.m. ET today to discuss recent corporate highlights and financial results for the quarter ended June 30, 2022. The dial-in numbers are (844) 200‑6205 for domestic callers and (929) 526‑1599 for international callers. The access code is 908644. Please dial in at least 10 minutes prior to the start time.
A live webcast of the conference call can be accessed via the Investors & Media page of the Aldeyra website at https://ir.aldeyra.com. After the live webcast, the event will remain archived on the website for 90 days.
Announced analyses from Phase I study of etavopivat in sickle cell disease indicated reduction of reported pain-related adverse events, supporting potential to reduce vaso-occlusive crises
Recently entered into an exclusive license agreement with Rigel Pharmaceuticals, Inc. for olutasidenib, a mutant IDH1 inhibitor for the potential treatment of relapsed or refractory acute myeloid leukemia
Highlighted progress across portfolio at inaugural Research and Development (R&D) Day
Appointed new members of Executive Team to prepare for growth in late-stage clinical development and commercialization
Cash balance of $395.9 million positions company with runway through the third quarter of 2024
A fifth child is presumed to have monkeypox in the United States since the country’s first two pediatric cases were reported in July.
California health officials announced the latest presumptive pediatric monkeypox case in a Long Beach child on Tuesday, four days after Indiana health officials confirmed that two children in that state also tested positive.
Last month, another California toddler tested positive for monkeypox as well as an infant resident of a foreign country who was passing through Washington D.C., bringing the total U.S. pediatric cases to five.
The Long Beach Department of Health and Human Services (DHHS) is carrying out a contact tracing investigation and told The Epoch Times it had no further information about those efforts since Tuesday.
“Preliminary test results indicate that the child has tested positive for orthopoxvirus,” the Long Beach DHHS said in a statement. “Additional testing will be performed at the Centers for Disease Control (CDC) to confirm monkeypox.”
The child was symptomatic and is now recovered, according to the Californian department, which said, in light of the pediatric diagnosis, that monkeypox can also be spread by “holding and feeding.”
“This is a reminder that everyone, regardless of age or sexual orientation, can get monkeypox if they come into contact with the virus,” Long Beach DHHS said.
[ZH: CDC Director Rochelle Walensky was a little more specific last week - the children were reportedly "gay community adjacent."]:
Pediatric Cases in Indiana
The new pediatric case in California comes days after the Indiana Department of Health (IDOH) reported two pediatric cases among its total of 45 new cases confirmed between June 18 and July 28.
IDOH said it won’t release additional information about the cases due to patient privacy.
“Like many other states, Indiana has seen an increase in monkeypox cases over the past month,” State Health Commissioner Dr. Kris Box said in a statement.
“Monkeypox does not easily spread through brief casual contact, but it’s important to remember that anyone can be affected if they are a close contact of a positive case.”
She encouraged residents who think they’ve been exposed or who develop symptoms to contact a healthcare provider.
A section of skin tissue, harvested from a lesion on the skin of a monkey, that had been infected with monkeypox virus, is seen at 50X magnification on day four of rash development in 1968. (CDC/Handout via Reuters)
In guidance, the CDC has said that children with exposure to people with monkeypox may be eligible for post-exposure prophylaxis (PEP) with vaccination, immune globulin, or antiviral medication.
PEP is commonly known for its use as an emergency antiviral medication to prevent HIV infection and is taken largely by gay and bisexual men. However, the first-line treatment for children is the antiviral Tecovirimat, commonly used for smallpox.
Household Spread
In the 2022 outbreak, monkeypox has primarily been spreading through the sexual activity of gay and bisexual men but it is not typically considered by health officials to be a sexually transmitted disease.
The CDC and World Health Organization (WHO) have said the virus can also spread through contaminated bedding and clothing.
With the fifth child now contracting monkeypox, presumably within a household, Long Beach DHHS has named other household items that could spread the virus, such as cups, towels, and utensils.
For this reason, the department has said that people with monkeypox should avoid contact with household members and follow the CDC’s guidance for limiting transmission in the home.
“With children, people are advised to minimize the number of caregivers and limit interaction between siblings, including sharing toys, clothing, linens and bedding. It is also important for the infected person to limit interactions with pets in the home,” Long Beach DHHS said.
The CDC has provided extensive guidance on household infection control to inform people with monkeypox around how to isolate, including not engaging in “sexual activity that involves direct physical contact.”
Much like with COVID-19, people with monkeypox should isolate away from other household members, and clean shared spaces appropriately until such as time when any “rash has fully resolved, the scabs have fallen off, and a fresh layer of intact skin has formed.”
Spread Beyond LGBT Community
The rise to five pediatric cases comes after a senior WHO official warned that monkeypox would likely spread beyond the LGBT population.
Dr. Catherine Smallwood, a senior emergency officer at the WHO, told CNBC in July that “we should not expect” monkeypox to remain only within the demographic of “men who have sex with men.”
Experts and health officials are still coming to grips with monkeypox’s evolution from an endemic disease limited mostly to Africa, where it is spread by animals, to the current vector of the 2022 outbreak, which is spread primarily via the sexual activity of gay and bisexual men, according to a major peer-reviewed study.
Dr. Anthony Fauci, the White House’s medical adviser and director of the National Institute of Allergy and Infectious Diseases, said health officials needed to come to a better scientific understanding of the current monkeypox outbreak.
He also said swift interventions and outreach to the LGBT community, which is most at risk from the virus, was “absolutely” needed to combat the virus.
Monkeypox case numbers in the United States are currently over 5,800 since the first case emerged in May. Last month, the WHO declared the monkeypox outbreak a public health “emergency.”
Florida Gov. Ron DeSantis, whose state has the fifth highest number of monkeypox cases as of Aug. 4, has said he will not follow suit.
The Republican governor accused other elected officials of driving a scare campaign.
“Do not listen to their nonsense … I’m so sick of politicians, and we saw this with COVID, trying to sew fear into the population,” he told reporters on Wednesday.
He added: “We’re not going to go back to like Fauci in the 80s where he was trying to tell families they were all going to catch AIDs watching TV together.”
DeSantis said that Florida’s response to monkeypox won’t include a “fear” campaign to “rile people up and try to act like people can’t live their lives as they’ve been normally doing.”
“You see some of these states declaring states of emergency. They’re gong to abuse those emergency powers to try to restrict your freedom. I guarantee you that’s what will happen. We saw it with COVID,” he added.
Two new lawsuits allege that Meta, Facebook’s parent company, and a number of US hospitals violated medical privacy law HIPAA, according to The Verge. These lawsuits follow a report from The Markuppublished this June documenting how the Meta Pixel, an ad analytics tracking tool installed on many websites, potentially shared identifying patient data in a way that violated HIPAA. Both lawsuits were filed in the Northern District of California and argued that the use of the Meta Pixel on hospital websites allowed sensitive health information to be sent to Facebook. The lawyers for the plaintiffs are trying to get them classified as class action suits and demanding jury trials.
But let’s step back a bit and answer some key questions: What is the Meta Pixel, how does it work, and why are hospitals’ installing it on their websites? And since they are, is that likely to be a HIPAA violation?
The Meta Pixel is a free ad tracking tool from Facebook. According to research conducted by The Markup, approximately a third of the 80,000 most popular websites have the Meta Pixel installed. (Full disclosure: PopSci is one of them). This tool allows website owners to see analytics from Facebook and Instagram ads they run, and target Facebook and Instagram users who have visited their sites with ads.
The Meta Pixel is automatically triggered when someone visits a website with it installed. If they’re logged into Facebook (and not using a browser that protects against third-party tracking), it sends information about who they are and what they do on the site to Facebook. (Even if they’re not logged in, Facebook has other ways of attempting to glean information about visitors through the Meta Pixel). What information is sent to Facebook is controlled by the website operator, and this is where the HIPAA troubles start.
As part of The Markup’s Pixel Hunt investigation into Facebook ad tracking, it tested the websites of Newsweek’s top 100 US hospitals for 2022. It found the Meta Pixel installed on 33 of them, and all of them sent sensitive data to Facebook, including identifying information such as a visitor’s IP address, and when they attempted to schedule an appointment.
“On the website of University Hospitals Cleveland Medical Center, for example, clicking the ‘Schedule Online’ button on a doctor’s page prompted the Meta Pixel to send Facebook the text of the button, the doctor’s name, and the search term we used to find her: ‘pregnancy termination,’” The Markup reported.
For seven hospitals, the situation was even worse. The Meta Pixel wasn’t just installed on the public facing web pages, but also on the password-protected patient portals. For five of those websites, it documented real patient data—provided by volunteers who signed up to help the Pixel Hunt investigation using Mozilla’s ad-tracker tracking Rally plugin—being sent to Facebook. Some of that information included “the names of patients’ medications, descriptions of their allergic reactions, and details about their upcoming doctor’s appointments.”
According to The Markup, “former regulators, health data security experts, and privacy advocates” all expressed concern that the hospitals using the Meta Pixel on their patient portals may have violated HIPAA regulations. David Holtzman, a health privacy consultant who has previously served as a senior privacy adviser for the US government agency that enforces HIPAA, told The Markup that while he couldn’t say for certain, “it is quite likely a HIPAA violation.”
It’s important to note that Facebook itself is not subject to HIPAA as it is not a healthcare provider. Still, there is cause for legitimate scrutiny of how Meta handles sensitive data. Following a report inThe Wall Street Journal and a New York Department of Financial Services investigation in 2019, Meta said it was introducing a tool to automatically filter out sensitive medical data sent by websites through the Meta Pixel. However, according to previous reporting byThe Markup and leaked Facebook internal documents, it is unlikely that the tool is 100 percent effective at filtering out sensitive medical data.
Medical providers, on the other hand, are bound by HIPAA. They are not supposed to share data with third-parties without express consent from the patient in question. From The Markup’s reporting, it seems unlikely that any of the hospitals obtained that.
While the majority of hospitals documented by The Markup’s investigation removed the Meta Pixel from their patient portals after they were contacted (and some also removed it from their public websites), their past actions set the stage for these two lawsuits.
As well as Meta, one of the lawsuits names University of California San Francisco and Dignity Health patient portals as defendants. Apparently, a patient claims her medical information was sent to Facebook where she was then served targeted ads relating to her heart and knee conditions. The other suit doesn’t name any other defendants, but claims at least 664 healthcare providers have sent medical data to Meta.
We won’t know whether either case will become a class action or even proceed for a while yet, but it’s another bad story for Meta—which really can’t seem to catch a break.
