Abstract/Full Text (subscription or payment may be required)

Editorial (subscription or payment may be required)

Compared with insulin glargine, initial injectable therapy with a combination of insulin degludec and liraglutide aids achievement of blood glucose goals for a longer period of time in patients with uncontrolled type 2 diabetes on oral antidiabetic drugs, according to a study published online June 9 in The Lancet Diabetes & Endocrinology to coincide with the annual meeting of the American Diabetes Association, held from June 7 to 11 in San Francisco.

Abstract/Full Text (subscription or payment may be required)
Editorial (subscription or payment may be required)

Zygel

Zynerba Pharmaceuticals Receives Fast Track Designation for Zygel for the Treatment of Behavioral Symptoms Associated with Fragile X Syndrome (FXS)

Devon, PA, May 6, 2019 — Zynerba Pharmaceuticals, Inc. (NASDAQ:ZYNE), the leader in innovative pharmaceutically-produced transdermal cannabinoid therapies for rare and near-rare neuropsychiatric disorders, today announced that the U.S. Food and Drug Administration (FDA) has
granted Fast Track Designation for the Company’s lead development candidate Zygel™ (ZYN002 CBD gel) for treatment of behavioral symptoms associated with Fragile X Syndrome (FXS). FDA’s Fast Track program is designed to facilitate the development of drugs intended to treat serious conditions and fill unmet medical needs, and can lead to expedited review by FDA in order to get new important drugs to the patient earlier.
“The FDA’s decision to grant Fast Track Designation for Zygel underscores the significance and severity of the unmet medical need that exists for patients living with Fragile X Syndrome and their caregivers,” said Armando Anido, Chairman and Chief Executive Officer of Zynerba. “We believe that Zygel has the potential to be the first treatment indicated to directly address the core behavioral symptoms of this syndrome, and we look forward to working closely with the FDA to obtain approval to market Zygel as soon as possible.”
Zynerba is conducting a pivotal trial to assess the efficacy and safety of Zygel as treatment for the behavioral symptoms of FXS in pediatric and adolescent patients (three through 17 years of age). Zygel is a pharmaceutically-manufactured CBD formulated as a patent-protected permeation-enhanced clear gel, designed to provide controlled transdermal drug delivery into the bloodstream.
About Fast Track Designation

Fast Track Designation is intended to facilitate development and expedite review of drugs to treat serious or life-threatening conditions so that a product can reach the market expeditiously. A drug that is intended to treat a serious or life-threatening condition that demonstrates the potential to address an unmet medical need may qualify for Fast Track designation. Features of this designation include opportunities for frequent interactions with the review team. These include meetings with the FDA to discuss items such as study design, extent of safety data required to support approval, dose-response concerns, accelerated approval, the structure and content of an NDA, and other critical issues. In addition, such a product could be eligible for priority review if supported by clinical data at the time of NDA.
About Fragile X Syndrome (FXS)

Fragile X syndrome is a rare genetic developmental disability that is the leading known cause of both inherited intellectual disability and autism spectrum disorder, affecting 1 in 3,600 to 4,000 males and 1 in 4,000 to 6,000 females. It is the most common inherited intellectual disability in males and a significant cause of intellectual disability in females. FXS is caused by a mutation in the Fragile X Mental Retardation gene (FMR1) located on the X chromosome and leads to dysregulation of the endocannabinoid pathway including the reduction in endogenous cannabinoids (2-AG and anandamide). The disorder negatively affects synaptic function, plasticity and neuronal connections, and results in a spectrum of intellectual disabilities and behavioral symptoms, such as social avoidance and irritability. In the US, there are about 71,000 patients suffering with FXS.
About Zygel

Zygel (CBD gel) is the first and only pharmaceutically-manufactured CBD formulated as a patent-protected permeation-enhanced clear gel, designed to provide controlled drug delivery into the bloodstream transdermally (i.e. through the skin). Recent studies suggest that FXS and other neuropsychiatric conditions may be associated with a disruption in the endocannabinoid (EC) system. Clinical and anecdotal data suggest that CBD may modulate the EC system and improve certain core social and behavioral symptoms, including social avoidance (prefers isolation from others, prefers solitary activities, avoids new social activities), irritability (aggressive to others, tantrums/outbursts, and stubbornness), and social unresponsiveness/lethargy (lack of attention/interaction, inactive/lack of movement and can resist physical contact).
Enrollment is ongoing in the multi-national, randomized, double blind placebo controlled Clinical study of Cannabidiol (CBD) in Children and Adolescents with Fragile X (CONNECT-FX), a pivotal clinical trial of ZYN002 in FXS (https://www.connectfxtrial.com/); topline data from CONNECT-FX are expected in the second half of 2019. Additionally, Zynerba expects topline data from its Phase 2 Open Label Study to Assess the Safety and Efficacy of ZYN002 Administered as a TransdermalGel to Children and Adolescents with Developmental and Epileptic Encephalopathy (BELIEVE 1) clinical trial in the third quarter of 2019. Zynerba has also initiated a Phase 2 study of Zygel in Autism Spectrum Disorder, with data expected in the first half of 2020.
About Zynerba Pharmaceuticals, Inc.

Zynerba Pharmaceuticals is the leader in pharmaceutically-produced transdermal cannabinoid therapies for rare and near-rare neuropsychiatric disorders. We are committed to improving the lives of patients and their families living with severe, chronic health conditions including Fragile X Syndrome, Autism Spectrum Disorder, 22q11.2 Deletion Syndrome, and a heterogeneous group of rare and ultra-rare epilepsies known as developmental and epileptic encephalopathies. Learn more at www.zynerba.com and follow us on Twitter at @ZynerbaPharma.
Cautionary Note on Forward-Looking Statements
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. We may, in some cases, use terms such as “predicts,” “believes,” “potential,” “proposed,” “continue,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should” or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such statements are subject to numerous important factors, risks and uncertainties that may cause actual events or results to differ materially from the Company’s current expectations. For example, there can be no guarantee that the Company will obtain approval for Zygel from the U.S. Food and Drug Administration (FDA) or foreign regulatory authorities; even if Zygel is approved, the Company may not be able to obtain the label claims that it is seeking from the FDA. Management’s expectations and, therefore, any forward-looking statements in this press release could also be affected by risks and uncertainties relating to a number of other factors, including the following: the Company’s cash and cash equivalents may not be sufficient to support its operating plan for as long as anticipated; the Company’s ability to obtain additional funding to support its clinical development programs; the results, cost and timing of the Company’s clinical development programs, including any delays to such clinical trials relating to enrollment or site initiation; clinical results for the Company’s product candidates may not be replicated or continue to occur in additional trials and may not otherwise support further development in a specified indication or at all; actions or advice of the FDA and foreign regulatory agencies may affect the design, initiation, timing, continuation and/or progress of clinical trials or result in the need for additional clinical trials; the Company’s ability to obtain and maintain regulatory approval for its product candidates, and the labeling under any such approval; the Company’s reliance on third parties to assist in conducting pre-clinical and clinical trials for its product candidates; delays, interruptions or failures in the manufacture and supply of the Company’s product candidates the Company’s ability to commercialize its product candidates; the size and growth potential of the markets for the Company’s product candidates, and the Company’s ability to service those markets; the Company’s ability to develop sales and marketing capabilities, whether alone or with potential future collaborators; the rate and degree of market acceptance of the Company’s product candidates; and the Company’s expectations regarding its ability to obtain and adequately maintain sufficient intellectual property protection for its product candidates. This list is not exhaustive and these and other risks are described in the Company’s periodic reports, including the annual report on Form 10-K, quarterly reports on Form 10-Q and current reports on Form 8-K, filed with or furnished to the Securities and Exchange Commission and available at www.sec.gov. Any forward-looking statements that the Company makes in this press release speak only as of the date of this press release. The Company assumes no obligation to update forward-looking statements whether as a result of new information, future events or otherwise, after the date of this press release.
Source: Zynerba Pharmaceuticals, Inc.

Has America's Fight Against HIV Stalled?

 The battle against new HIV infections has lost some steam in recent years, a new report shows.
After about five years of significant declines, the number of new HIV infections began to level off in 2013, at about 39,000 infections per year, the U.S. Centers for Disease Control and Prevention found.
Why the slowing? Effective HIV prevention and treatments are not reaching those who could most benefit, according to the CDC.
These shortfalls in prevention and treatment are most glaring in rural areas and in the South, and they disproportionately affect blacks and Hispanics.
The report found that HIV infections declined in some groups, but increased in others. From 2010 to 2016, annual HIV infections were stable among gay and bisexual men, who continue to account for the largest portion (about 70 percent) of new infections.
But infection trends varied by race, ethnicity and age.
Infections remained stable among all black gay and bisexual men, increased 30 percent among all Hispanic gay and bisexual men, and fell 16 percent among all white gay and bisexual men.
Infections decreased more than 30 percent among black gay and bisexual males aged 13 to 24, and remained stable among Hispanic gay and bisexual males aged 13 to 24. Meanwhile, they rose 65 percent among black and Hispanic gay and bisexual males aged 25 to 34.
Infections fell 17 percent among heterosexual men and women combined, including a 15 percent decrease among heterosexual black women.
Infections decreased 30 percent among people who inject drugs, but appear to have stabilized in more recent years, according to the report released Wednesday.
"After a decades-long struggle, the path to eliminate America's HIV epidemic is clear," said Dr. Eugene McCray, director of CDC's Division of HIV/AIDS Prevention.
"Expanding efforts across the country will close gaps, overcome threats and turn around troublesome trends," he said in an agency news release.
In his State of the Union address to the nation on Feb. 5, President Donald Trump called for support of a national plan to: diagnose HIV as early as possible; treat HIV rapidly and effectively; protect at-risk people with proven preventive measures, including a daily pill to prevent HIV infection; and respond rapidly to growing clusters of HIV infections.
The initiative is designed to accelerate use of these strategies in the 48 U.S. counties with the highest HIV burden, as well as in Washington, D.C.; San Juan, Puerto Rico; and seven states with a disproportionate rural HIV burden.
Intensified local efforts have already produced promising results, according to the CDC. Public health officials in New York City and Washington, D.C., set plans in motion to eliminate their local HIV epidemics -- and they seemed to have worked. From 2010 to 2016, new HIV infections decreased about 23 percent in New York City, while they dropped about 40 percent in Washington, D.C.
The national goal under Trump's proposal is to reduce new HIV infections by 90 percent over 10 years.
"We have an historic opportunity to improve the precision of prevention," said Dr. Jonathan Mermin, director of the CDC's National Center for HIV/AIDS, Viral Hepatitis, STD and TB Prevention.
"This infusion of resources will finally relegate America's HIV epidemic to the pages of history," Mermin added.

Poor Asthma Control Tied to Worse School Performance

 Kids with poorly controlled asthma struggle in school, especially those who are ethnic minorities, a new study reports.
Researchers evaluated asthma and allergy status, lung function and school performance of 216 black, Hispanic (Latino) and white children in a U.S. city.
Those with a greater number of daily asthma symptoms had more absences, less school work completed and poorer quality work, according to the study published March 11 in Annals of Allergy, Asthma and Immunology, the journal of the American College of Allergy, Asthma and Immunology (ACAAI).
"We found associations between poor asthma status, poorer asthma control, lower lung function, more asthma symptoms and decline in academic performance," lead author Daphne Koinis-Mitchell said in an ACAAI news release.
"These associations were stronger in ethnic minority children, particularly Latino children," she added. Koinis-Mitchell is a research professor of pediatrics, psychiatry and human behavior at Brown University in Providence, R.I.
Poor asthma control could help identify children who might be at risk for problems at school, according to the researchers.
The same team previously found that urban Hispanic children with asthma have special sources of stress.
"Factors such as higher levels of fear of asthma, language barriers, stress related to fitting in to the culture, poorer symptom perception, greater concerns regarding medications and lower medication adherence may put Latinos at greater risk for poor academic performance," Koinis-Mitchell said.
ACAAI president Dr. Todd Mahr, an allergy specialist, noted that asthma is the most common chronic illness in childhood, accounting for 13.8 million missed school days each year in the United States.
"A very low proportion of the children in this study saw specialists, such as allergists," he said in the news release. Many of the kids also had undiagnosed hay fever, which can contribute to poorer asthma outcomes and lower academic success.
"Kids with asthma -- especially in urban areas -- need access to specialty care because anyone with asthma should be able to feel good, be active all day and sleep well at night," Mahr concluded. "No one should accept less."

"Because depression is such a common problem, finding effective and widely available ways to prevent depression at a population level is an important goal," said study co-author Ed Watkins.

He's a professor of experimental and applied clinical psychology at the University of Exeter in England.
"Diet and nutrition held promise as one means to reach large numbers of people. However, this trial convincingly demonstrates that nutritional supplements do not help to prevent depression," he said in a university news release.

Blacks, Hispanics Bear Burden of Air Pollution: Study

 Air pollution caused mainly by white Americans has the greatest impact on black and Hispanic Americans, a new study says.
"Similar to previous studies, we show that racial-ethnic minorities are exposed to more pollution on average than non-Hispanic whites," said lead author Christopher Tessum, a research scientist at the University of Washington in Seattle.
"What is new is that we find that those differences do not occur because minorities on average cause more pollution than whites -- in fact, the opposite is true," he said.
This is a case of "pollution inequity" -- the difference between the pollution caused by a racial-ethnic group and the harm experienced by that group.
For this study, University of Washington and University of Minnesota researchers focused on fine particulate matter (PM2.5) air pollution. It causes more than 100,000 deaths a year in the United States from heart attack, stroke, lung cancer and other diseases.
But exposure to this type of bad air is not equal among Americans, and not all are equally responsible for causing it, the study showed.
The researchers explained that whites consume more pollution-creating goods and services and therefore are responsible for more PM2.5 pollution than other groups. Also, blacks and Hispanics often live in locations with higher pollution concentrations than whites.
On average, whites have 17 percent less exposure to PM2.5 pollution than they create. Blacks, meanwhile, are exposed to about 56 percent more PM2.5 pollution than they create, and Hispanics to 63 percent more.
"Our work is at the intersection of many important and timely topics such as race, inequality, affluence, environmental justice and environmental regulation," Jason Hill, a bioproducts and biosystems engineering professor at the University of Minnesota, said in a university news release.
Julian Marshall, a professor of civil and environmental engineering at the University of Washington, said the approach used in this study could be extended to other pollutants, locations and groups of people.
"When it comes to determining who causes air pollution -- and who breathes that pollution -- this research is just the beginning," Marshall said.
The study was published March 11 in the journal Proceedings of the National Academy of Sciences.