Aeglea BioTherapeutics presented new Phase 1/2 data for pegzilarginase in patients with Arginase 1 Deficiency at the 2019 Annual Meeting of the Society for Inherited Metabolic Disorders in Bellevue, Washington. The new Phase 1/2 data continues to demonstrate that pegzilarginase is highly effective in sustainably lowering plasma arginine, which is the primary endpoint in Aeglea’s single, global pivotal Phase 3 PEACE trial. In addition, the new data shows that the marked improvement in plasma arginine control is accompanied by clinically meaningful responses in mobility and adaptive behavior, which are secondary endpoints in the PEACE trial. The treatment was generally well tolerated. Hypersensitivity reactions were infrequent, manageable with standard measures, and did not lead to treatment discontinuation.
Sunday, April 7, 2019
How High Housing Costs Impact Health Care
A new national report commissioned by Enterprise Community Partners Inc. as part of its Health Begins with Home initiative found that more than half of renters surveyed delayed health care because they couldn’t afford it.
The survey revealed renters who are paying a high percentage of their income for housing—particularly those paying more than 50 percent of their monthly income—routinely make difficult decisions between paying for rent and health care. Of that group, 83 percent said they prioritize paying rent before anything else, compared with 1 percent that prioritized healthcare costs. Nearly half (45 percent) said they have not followed a treatment plan from health care professionals because they couldn’t afford it, compared with 34 percent of all renter respondents. And nearly one-third (31 percent) of severely rent-burdened respondents said they delayed a routine checkup because they couldn’t afford it, compared to 23 percent of all renter respondents.
“No one should have to choose between paying rent and paying for health care,” Laurel Blatchford, Enterprise president, said in a prepared statement. “And yet, thousands of people make that difficult trade-off every day. That’s wrong. By working closely with health care organizations, we’re creating ways for renters to afford the health care they need.”
The survey, the first to examine renters’ ability to afford health care and medical professionals’ perception of those challenges, also found that every one of the 500 medical professionals surveyed reported at least some of their patients have expressed concerns about affordable housing. Thirty-one percent of those professionals reported at least a quarter of their patients said they had concerns about affordable housing. That number increased to 42 percent among those professionals who treat a large number of lower-income patients. Forty-four percent stated they believe a lack of accessible health care hinders the health of lower-income communities.
“This survey is the first to carefully document how these challenges affect both renters and medical professionals, and will help both the health and the housing sectors collaborate to save lives. This interdependence between health and housing must remain at the forefront of our collective health equity efforts,” Brian Rahmer, vice president, Health and Housing, at Enterprise, said in prepared remarks.
PARTNERSHIPS AND COLLABORATION ARE KEY
The Columbia, Md.-based national affordable housing non-profit is working with a broad group of partners to promote health as a top priority in the development and preservation of affordable housing and is deploying $250 million over five years to spur more collaboration through the Health Begins at Home initiative launched in January. One of the groups Enterprise is working with is Kaiser Permanente, which had announced a joint Enterprise-managed $85 million Housing for Health fund in the San Francisco Bay Area and a $100 million national loan fund.
Rahmer told Multi-Housing News the survey results, while not surprising to those who work in the affordable housing and public health sectors, “give us some additional points to stand on as we bring on new partners in different regions across the country.”
He said the survey findings would help Enterprise and others “shine more light on that connection” between housing and health outcomes.
“The accumulation of data and information continues to grow about people’s health outcomes and the trajectory of those outcomes, particularly in the United States,” Rahmer said in an interview, shortly after the survey was released Wednesday morning.
The survey also showed that respondents had lower satisfaction rates with housing-related factors that impact their health, including adequate access to outdoor spaces (47 percent), lack of exposure to indoor toxins (48 percent) and air quality (38 percent).
MENTAL HEALTH IMPACTS
Perhaps just as important, Rahmer noted information gleaned from the report also revealed how the economic burdens of high housing costs impact renters’ mental health too, causing ongoing stress.
“This chronic stress has a wearing effect, a weathering effect,” he said. “That is not good for anybody.”
Inadequate medical care and family stress also affects children’s health and well being, starting with their readiness for kindergarten and elementary school and continuing to have a dramatic impact throughout their lives, commented Rahmer.
Providing quality, stable and safe affordable housing is key to creating better outcomes, he stated.
When Enterprise introduced its Health Begins with Home Initiative in January, executives said it would focus on four key areas: conducting research; awarding grants to nonprofits to fund housing and community health programs and increase partnerships between housing and healthcare organizations; providing technical assistance and connecting capital from healthcare organizations, institutional investors and social impact funds to develop and preserve affordable, healthy and well-designed homes.
Rahmer told MHN Enterprise will be putting that $250 million to work with current partners but will also be working on creating new partnerships to invest in capital programs, health equity funds and loan funds.
Allergan’s failed depression drug shows promise in opioid withdrawal
Last month, Allergan said that rapastinel—the centerpiece of its $560 million acquisition of Naurex—had failed in three pivotal trials in major depressive disorder. But not all is lost. Scientists at Duke University and Villanova University may have found a use for the drug in treating opioid withdrawal.
People who become dependent on opioids can experience excruciating withdrawal symptoms, such as anxiety, vomiting, diarrhea and insomnia. These symptoms often prevent them from seeking help, or they cause relapse in patients who do seek out treatment for addiction.
Rapastinel might help manage those symptoms, according to a new study by Julia Ferrante at Villanova University and Cynthia Kuhn at Duke University. The experimental drug substantially reversed acute withdraw signs in rats in three days, the researchers said in a release. The study was presented at the American Society for Pharmacology and Experimental Therapeutics annual meeting in Orlando.
There are FDA-approved drugs to treat opioid use disorder. Buprenorphine and methadone are commonly used to help eliminate the opioid cravings. But they are also opioids that can be abused, they can cause side effects, and patients sometimes need to use them over the long term to avoid relapse.
Two other drugs, naloxone and naltrexone, are not opioids. But patients need to detox before they can use naltrexone, and naloxone is more appropriate for use in emergency settings to reverse opioid overdoses.
Rapastinel, in comparison, binds to the same NMDA receptor that the “party drug” ketamine uses, but at a different site. In their study, Ferrante and Kuhn gave rat models of opioid dependence either rapastinel, ketamine or a saline solution. On the third day, rats given rapastinel showed significantly fewer signs of withdrawal than the other animals did, the researchers reported.
Opioid overdose has become a public health crisis in the U.S., with an estimated 130 people dying each day from opioid-related drug overdoses, according to the U.S. Department of Health and Human Services. While health regulators are pursuing tactics for cutting down on opioid use, scientists are stepping up their research efforts, looking for new ways to fight addiction.
Last May, the FDA greenlighted US WorldMeds’ Lucemyra as the first approved drug specifically for opioid withdrawal, even though the drug had been used for more than two decades in the U.S.
Scientists at the Walter Reed Army Institute of Research previously found a heroin vaccine could block the euphoric effects of opioids in the brain by blocking heroin from crossing the blood-brain barrier.
Although rapastinel’s use in depression turned out to be a dead end, clinical trials have shown it is well-tolerated in people. Before rapastinel can be moved into clinical trials in opioid withdrawal, more studies are needed to investigate its molecular effects and to determine whether it can reduce the possibility of relapse, according to the researchers.
Biotech week ahead, April 8
Biotech stocks ended higher for the second straight week, with the presentations at the American Association of Cancer Research annual meeting taking the spotlight. Additionally, there were clinical trial readouts from companies that carried a fourth-quarter timeframe.
ADMA Biologics Inc ADMA 2.12% shares rallied sharply after the FDA nod for its immunodeficiency therapy. But Evoke Pharma Inc EVOK 0.98%‘s NDA for diabetic gastroparesis drug Gimoti received a thumbs-down from the regulatory agency, sending its shares down about 50% Tuesday.
Looking ahead to the unfolding week, the following are some key catalytic events biotech investors will be focusing on.
Conferences
- Society for Inherited Metabolic Disorders 2019 annual meeting: April 6-9 in Bellevue, Washington.
- H.C. Wainwright Global Life Sciences Conference: April 8-9 in London.
- 13th International Conference on Endocrinology, Diabetes and Metabolism: April 8-9 in Wellington, New Zealand.
- 12th World Conference on Human Genomics and Genomic Medicine: April 8-9, in Abu Dhabi, UAE.
- 18th Annual Needham Healthcare Conference: April 9-10 in New York City.
- 3rd International Conference on Influenza and Emerging Infectious Diseases: April 10-11 in Toronto, Canada.
- 2019 Congress of the Schizophrenia International Research Society: April 10-14 in Orlando, Florida.
- The European Association for the Study of the Liver, or EASL, International Liver Congress 2019: April 10-14 in Vienna, Austria.
- 12th World Congress on Dementia and Alzheimer Rehabilitation: April 11-12 in Stockholm, Sweden.
- 29th European Congress of Clinical Microbiology & Infectious Diseases: April 13-16 in Amsterdam, Netherlands.
PDUFA Dates
The FDA is set to rule on Merck & Co., Inc. MRK 0.83%‘s sBLA for Keytruda as a monotherapy for the first-line treatment of locally advanced or metastatic non-small cell lung cancer in patients whose tumors express PD-L1 without EGFR or ALK genomic tumor aberrations. The FDA decision is due Thursday, April 11.
Clinical Trial Readouts
Alkermes Plc ALKS 3.51% is due to release new Phase 3 data for ALKS 3831, which is being evaluated in a study dubbed ENLIGHTEN-2 for schizophrenia, at the 2019 Congress of the Schizophrenia International Research Society on Thursday, April 11.
The EASL International Liver Congress 2019 Presentations
Thursday, April 11
- Eiger Biopharmaceuticals Inc EIGR 1.03%: Late-breaker presentation of already released Phase 2 data for pegylated interferon lambda (hepatitis delta virus).
- Assembly Biosciences Inc ASMB 4.74%: Poster presentation of Phase 1a data for ABI-H2158 (hepatitis B virus).
- Intercept Pharmaceuticals Inc ICPT 1.12%): Full Phase 3 data for Ocaliva from the REGENERATE study (non-alcoholic steatohepatitis).
- Viking Therapeutics Inc VKTX 2.91% and Ligand Pharmaceuticals Inc. LGND 1.15%: additional Phase 2 data for VK2809 (hypercholesterolemia and non-alcoholic fatty liver disease, or NAFLD).
Friday, April 12
- Spring Bank Pharmaceuticals Inc SBPH 0.42%: full Phase 2 data for inarigivir and vemlidy (hepatitis B virus).
- Albireo Pharma Inc ALBO 0.23%: Phase 2 data for A4250 (liver disease).
- Altimmune Inc ALT 2.84%: Phase 1 data for HepTCell (hepatitis B).
Saturday, April 13
- Assembly Biosciences: Interim Phase 2a data for AB-H0731 (hepatitis B virus).
- Conatus Pharmaceuticals Inc CNAT 3.85%: Late-breaker presentation of Phase 2b data for emricasan (NASH cirrhosis).
29th European Congress of Clinical Microbiology & Infectious Diseases Presentations
Saturday, April 13
J&J wins trial over Cal. man’s talc-cancer claim
Johnson & Johnson won the latest trial over claims its iconic baby powder can cause cancer, as a jury in Long Beach, California, rejected a lawsuit brought by a 65-year-old retired teacher who claimed the product was contaminated with asbestos.
The J&J victory Friday comes the week after the company settled three cases alleging baby powder caused a rare asbestos-connected cancer. Two, in Oklahoma and California, were settled during trial. The other, in New York, was settled less than two weeks before trial was set to begin.
Plaintiff Robert Blinkinsop was diagnosed in July 2017 with mesothelioma, a rare cancer of the lining of the lung generally caused by asbestos exposure. Blinkinsop used Johnson’s baby powder daily for personal hygiene from 1977 to 1994 and on his children from 1992 to 1996, his attorney, Mark Bratt, said at trial. He also used the company’s Shower to Shower talc product.
J&J is facing more than 13,000 lawsuits linking baby powder and another talc product, Shower to Shower, to ovarian cancer or mesothelioma. More than two dozen trials have been scheduled in U.S. courts in 2019.
“Today’s jury unanimously ruled that Johnson’s Baby Powder does not contain asbestos and was not the cause of the plaintiff’s disease,’’ Kim Montagnino, company spokeswoman, said in an emailed statement. “This conclusion is aligned to the decades of clinical evidence and scientific studies by medical experts around the world that support the safety of Johnson’s Baby Powder.’’
Blinkinsop’s attorney said he was shocked and disappointed by the verdict.
“Given the hundreds of positive historical tests from J&J showing asbestos in its talc containing baby powder, as well as J&J’s ongoing decision to use deceptive testing techniques that allow for false negative results — we have no doubt asbestos was, and currently still is in J&Js talc baby powder products,” Bratt said in a statement. “Asbestos in baby powder is a huge public health concern that is far bigger than just this case.”
The company’s record at trial has been mixed so far. The company lost a $29 million jury award in a California mesothelioma case March 13 and won a defense verdict in a New Jersey trial March 27, the same day it settled the three cases.
Litigation Tourism Suffers Blow as J&J Dodges Trial in St. Louis
Several of the first trials resulted in plaintiffs’ verdicts, including one for $4.69 billion in St. Louis in 2018 to 22 women with ovarian cancer. But J&J has been able to win reversals of three of the first five jury awards.The company has also won other defense verdicts in cases alleging links between baby powder and mesothelioma beyond the New Jersey trial.
Some of the verdicts include awards against J&J’s talc supplier, Imerys America, which filed for bankruptcy protection in February.
Lawyers for Blinkinsop and his wife Karen alleged that J&J knew for decades that its talc contained asbestos yet didn’t warn or protect customers. People at J&J “made some really poor decisions in the past,’’ which led to Blinkinsop’s exposure to asbestos, Bratt said. Talc and asbestos were found in Blinkinsop’s lung tissue, he said.
Blinkinsop’s condition is “terminal,’’ he testified at trial. “It’s not curable,’’ he said. The doctors have “all said the same thing that if everything goes well, I might survive three years,’’ he told jurors. “Never any guarantee about the quality of life during that time.’’
The company’s attorney told jurors here is no asbestos in J&J talc products and its talc didn’t cause Blinkinsop’s illness. “For over 40 years we have been working with the best scientists, the best universities and the best laboratories who have all confirmed our products do not contain asbestos,’’ defense attorney Bruce Hurley said.
Blinkinsop could have been exposed to asbestos while working on construction jobs, he said.
“Mr. Blinkinsop’s mesothelioma was not cause by Johnson’s baby powder,’’ Hurley said. “Cosmetic talc does not cause mesothelioma.’’
https://www.ocregister.com/2019/04/05/jj-wins-trial-over-california-mans-talc-cancer-claim/
The J&J victory Friday comes the week after the company settled three cases alleging baby powder caused a rare asbestos-connected cancer. Two, in Oklahoma and California, were settled during trial. The other, in New York, was settled less than two weeks before trial was set to begin.
Plaintiff Robert Blinkinsop was diagnosed in July 2017 with mesothelioma, a rare cancer of the lining of the lung generally caused by asbestos exposure. Blinkinsop used Johnson’s baby powder daily for personal hygiene from 1977 to 1994 and on his children from 1992 to 1996, his attorney, Mark Bratt, said at trial. He also used the company’s Shower to Shower talc product.
J&J is facing more than 13,000 lawsuits linking baby powder and another talc product, Shower to Shower, to ovarian cancer or mesothelioma. More than two dozen trials have been scheduled in U.S. courts in 2019.
“Today’s jury unanimously ruled that Johnson’s Baby Powder does not contain asbestos and was not the cause of the plaintiff’s disease,’’ Kim Montagnino, company spokeswoman, said in an emailed statement. “This conclusion is aligned to the decades of clinical evidence and scientific studies by medical experts around the world that support the safety of Johnson’s Baby Powder.’’
Blinkinsop’s attorney said he was shocked and disappointed by the verdict.
“Given the hundreds of positive historical tests from J&J showing asbestos in its talc containing baby powder, as well as J&J’s ongoing decision to use deceptive testing techniques that allow for false negative results — we have no doubt asbestos was, and currently still is in J&Js talc baby powder products,” Bratt said in a statement. “Asbestos in baby powder is a huge public health concern that is far bigger than just this case.”
The company’s record at trial has been mixed so far. The company lost a $29 million jury award in a California mesothelioma case March 13 and won a defense verdict in a New Jersey trial March 27, the same day it settled the three cases.
Several of the first trials resulted in plaintiffs’ verdicts, including one for $4.69 billion in St. Louis in 2018 to 22 women with ovarian cancer. But J&J has been able to win reversals of three of the first five jury awards.The company has also won other defense verdicts in cases alleging links between baby powder and mesothelioma beyond the New Jersey trial.
Some of the verdicts include awards against J&J’s talc supplier, Imerys America, which filed for bankruptcy protection in February.
Lawyers for Blinkinsop and his wife Karen alleged that J&J knew for decades that its talc contained asbestos yet didn’t warn or protect customers. People at J&J “made some really poor decisions in the past,’’ which led to Blinkinsop’s exposure to asbestos, Bratt said. Talc and asbestos were found in Blinkinsop’s lung tissue, he said.
Blinkinsop’s condition is “terminal,’’ he testified at trial. “It’s not curable,’’ he said. The doctors have “all said the same thing that if everything goes well, I might survive three years,’’ he told jurors. “Never any guarantee about the quality of life during that time.’’
The company’s attorney told jurors here is no asbestos in J&J talc products and its talc didn’t cause Blinkinsop’s illness. “For over 40 years we have been working with the best scientists, the best universities and the best laboratories who have all confirmed our products do not contain asbestos,’’ defense attorney Bruce Hurley said.
Blinkinsop could have been exposed to asbestos while working on construction jobs, he said.
“Mr. Blinkinsop’s mesothelioma was not cause by Johnson’s baby powder,’’ Hurley said. “Cosmetic talc does not cause mesothelioma.’’
https://www.ocregister.com/2019/04/05/jj-wins-trial-over-california-mans-talc-cancer-claim/
Ill. Medicaid to begin covering sex reassignment surgeries
Gov. J.B. Pritzker has ordered the state’s Medicaid program to begin covering the costs of sex reassignment surgeries for transgender people.
The Illinois Department of Healthcare and Family Services will develop administrative rules to offer coverage to people diagnosed with gender dysphoria to pay for genital and breast-related surgeries.
“Healthcare is a right, not a privilege, and I’m committed to ensuring our LGBTQ community and all Illinoisans have access to that right,” said Pritzker. “Expanding Medicaid to cover gender affirming surgeries is cost effective, helps avoid long term health consequences, and most importantly is the right thing to do. With continued attacks coming from Washington, this administration will always stand with our transgender community and their right to lead safe and healthy lives.”
The department said paying for the surgeries will help stave off negative health effects, such as depression and suicide. The HFS said 17 states and Washington, D.C. offer coverage for the surgeries without a significant cost increase.
The ACLU of Illinois hailed the decision to cover sex reassignment surgeries.
“We welcome Governor Pritzker’s announcement that Illinois will cover gender affirming surgery under its Medicaid program. This is a crucial step to respond to the health care needs of all Illinois residents, including those who are transgender,” said John Knight, LGBTQ and HIV Advocacy Director for the ACLU of Illinois. “By putting an end to this outdated exclusion on gender affirming surgery, Illinois now joins a long and growing list of states providing coverage for this critical, life-saving health care. We applaud the Governor for making this important step forward.”
The rule is likely to go into effect this summer.
Amgen: New Data on Osteoporosis Treatment Gap In Europe
Amgen (NASDAQ:AMGN) today announced new data presented from a cross-sectional study revealing a gap in the diagnosis and treatment of osteoporosis in Europe. The real-world study of osteoporosis management in primary care revealed that 75 percent of female patients aged 70 years and older who were at increased risk of fragility fractures were not treated for osteoporosis. The treatment gap was much lower in those with a recorded diagnosis of osteoporosis than in those without a recorded diagnosis. The results of the study, which was performed across eight European countries, were presented during the World Congress on Osteoporosis, Osteoarthritis and Musculoskeletal Diseases (WCO-IOF-ESCEO), in Paris from April 4-7, 2019.
“This study assessed patterns of real-world osteoporosis diagnosis and medical treatment in the European primary care setting,” said Eugene McCloskey, M.D., FRCPI, Professor of Adult Bone Diseases at The University of Sheffield and Director of the MRC Arthritis Research UK Centre for Integrated Research in Musculoskeletal Ageing. “Based on the results, future strategies need to increase awareness and facilitate the diagnosis of patients at risk in order to improve the treatment of osteoporosis and prevent fragility fractures from happening.”
The study enrolled 3,798 women aged 70 years or older after spontaneously visiting their primary care physician for any reason, not specifically related to their bone health. The primary outcome of the study was to assess the proportion of patients at increased risk of fragility fracture who were not receiving osteoporosis medication. Nearly 55 percent (n=2,077/3,798) of patients were considered to be at increased risk of fragility fracture, with 75 percent (n=1,550/2,077) of them not being medically treated for osteoporosis. Further, the study showed that among these untreated patients at risk for fracture, 85 percent (n=1,318/1,550) had no recorded diagnosis of osteoporosis.
“This real-world study further proves that an underdiagnosis of osteoporosis in Europe is a major barrier to treatment,” said David M. Reese, M.D., executive vice president of Research and Development at Amgen. “The finding that the vast majority of patients at increased risk of fracture remain untreated underscores the drastic need for better osteoporosis management and reinforces our ongoing commitment to help address this worldwide public health crisis1 and improve the care for millions of people living with this disease.”
Subscribe to:
Posts (Atom)