Asterias: Positive Outcome from Data of OPC1 Spinal Cord Injury Study

Asterias Biotherapeutics, Inc. (NYSE American: AST), a biotechnology company dedicated to developing cell-based therapeutics to treat neurological conditions associated with demyelination and cellular immunotherapies to treat cancer, today announced a positive outcome from an independent Data Review Panels review of the data generated by patients enrolled in the Companys ongoing Phase 1/2a SCiStar study designed to evaluate the safety and efficacy of OPC1 in the treatment of severe cervical spinal cord injury. Based on a review of the data, the Panel recommended moving forward with the continued clinical development of OPC1. The next step in the development of OPC1 is a meeting with the Food and Drug Administration (FDA) later this year to discuss proposed next steps for the OPC1 clinical development program, including the trial design for a randomized controlled Phase 2 trial.

We believe the the positive feedback we received from the Panel will strengthen our meeting with the Food and Drug Administration (FDA) later this year, commented Ed Wirth, Chief Medical Officer of Asterias. Assuming successful execution of the FDA meeting and obtainment of additional CIRM funding or alternative financing, we expect to enroll the first patient in the Phase 2 randomized controlled trial in the first half of 2020.

Asterias worked with the California Institute for Regenerative Medicine (CIRM) to complete the review of the data under CIRMs Clinical Advisory Panel process. The Panel comprised of outside medical and scientific experts that include James Guest, M.D., Ph.D., Professor of Clinical Neurological Surgery at the University of Miami, John Steeves, Ph.D., Co-Chair of the Spinal Cord Outcomes Partnership Endeavor (SCOPE) and Professor of International Collaboration on Repair Discoveries (ICORD) at the University of British Columbia, and Ann Parr, MD, PhD, Neurosurgeon and Director of Spinal Neurosurgery at the University of Minnesota. The Panel reviewed the most recent safety, engraftment and efficacy data from the SCiStar study including analysis of the data after removing a small subset of subjects that are likely to be excluded from next trial.

From our review of the data, the combination of the safety of the cells and administration procedure, the level of motor recovery, and the compelling evidence of the engraftment of OPC1 cells is an unprecedented step forward for the program and spinal cord injury community, commented Drs. Guest and Steeves.

The SCiStar trial is an open-label, single-arm trial testing three sequential escalating doses of OPC1 administered at up to 20 million OPC1 cells in 25 subjects with subacute motor complete (AIS-A or AIS-B) cervical (C-4 to C-7) spinal cord injuries. Asterias has completed enrollment and dosing in all five of its planned SCiStar study cohorts. The Company intends to report 12-month results for the entire SCiStar study in the first quarter of 2019. As previously announced, the Company has scheduled a Type B meeting with the FDA in accordance with the Regenerative Medicine Advanced Therapy (RMAT) designation under the 21st Century Cures Act.

https://www.marketscreener.com/ASTERIAS-BIOTHERAPEUTICS-18177640/news/Asterias-Biotherapeutics-Announces-Positive-Outcome-from-Data-Review-from-OPC1-Spinal-Cord-Injury-27524460/

Demand for new hips to boost Smith & Nephew profit margin

Britain’s Smith & Nephew expects its trading profit margin to be higher this year than last on a better performance for its hips franchise, boosting its shares.

Shares in the artificial hips and knees maker jumped 5.2 percent to 1,340 pence in early trading on Thursday after the positive update.

“Underlying growth was solid, and there was a welcome return to good growth in the hips business,” Bernstein analysts said.

The company forecast that for 2018 its underlying revenue would be in the lower half of the 2 percent to 3 percent range, but said that 2018’s trading profit margin would benefit from improved cost controls and a favourable legal settlement.

To help secure future growth, Smith & Nephew also said it would rearrange its commercial framework, appointing presidents of three franchises to help add new sales.

Smith & Nephew posted third-quarter revenue of $1.17 billion (£906 million), up 3 percent on the same period last year on an underlying basis, boosted by strong demand in the United States and emerging markets.

Hip reconstruction results buoyed the third quarter, said analysts at Investec.

“This is a particularly strong performance in hips, suggesting the company is taking share,” they said in a note.

https://www.marketscreener.com/SMITH-NEPHEW-9590181/news/Smith-Nephew-Demand-for-new-hips-to-boost-Smith-Nephew-profit-margin-27524415/

AbbVie Gets EU OK of VENCLYXTO + Rituximab for Lymphocytic Leukemia

AbbVie (NYSE: ABBV), a research-based global biopharmaceutical company, announced today that the European Commission (EC) has approved the type-II variation application for VENCLYXTO^® (venetoclax) in combination with rituximab for the treatment of patients with relapsed/refractory chronic lymphocytic leukemia (R/R CLL) who have received at least one prior therapy. This approval allows more patients to receive VENCLYXTO in the second-line setting and gives healthcare providers the ability to prescribe this medicine to a broader population of patients with R/R CLL than the previously approved indication for VENCLYXTO as monotherapy in the European Union (EU). The approval is valid in all 28 member states of the EU, as well as Iceland, Liechtenstein and Norway.

https://www.biospace.com/article/releases/abbvie-receives-european-commission-approval-of-venclyxto-venetoclax-plus-rituximab-for-the-treatment-of-patients-with-chronic-lymphocytic-leukemia-who-have-received-at-least-one-prior-therapy/

Health Of Babies In The U.S. Continues To Worsen: March Of Dimes Report Card

As health systems struggle to care for all moms, U.S. preterm birth rate increased again in 2017 despite progress in some states   

For the third year in a row, more U.S. babies were born too soon with serious risks to their health, according to the 2018 Premature Birth Report Card from March of Dimes, the nation’s leading maternal and infant health nonprofit. Premature birth and its complications are the largest contributor to death in the first year of life in the United States, and the leading cause of death of children under age 5 worldwide.

The overall U.S. preterm birth rate rose to 9.93 percent of births in 2017 from 9.85 percent in 2016, according to data from the National Center for Health Statistics (NCHS).  While there is no single cause of preterm birth, research shows that chronic inequities and unequal access to quality health care do have a negative impact on these rates. These factors contribute to the reality that women of color are up to 50 percent more likely to deliver prematurely and their children can face a 130 percent higher infant death rate compared to white women. Promising interventions can help reverse these trends, and better access to health care is essential. A recent March of Dimes report revealed the unequal access to maternity care across the US, particularly in communities with higher poverty rates.

The continued rise in its preterm birth rate earned the U.S. a “C” grade on the March of Dimes Premature Birth Report Card, which grades all 50 states, DC and Puerto Rico on their preterm birth rate. This year, 30 states had a worse rate compared to last year and 10 of those states received a worse grade. The Report Card shows the racial, ethnic and geographic disparities in preterm birth within each state. (Visit March of Dimes to download broadcast-quality videos, high-resolution pictures, documents and other links about preterm birth.)

“We must all come together to take concrete, commonsense steps to reverse this alarming trend,” says Stacey D. Stewart, president of March of Dimes. “Our country’s most important resource is human potential. That begins with ensuring every baby has the healthiest possible start in life, regardless of racial and ethnic background or their family’s income. By expanding proven programs and innovative solutions we can shift our health care system to improve treatment and preventive care for moms and lower the preterm birth rate. Birth equity is our goal; it can be reached.”

The 2018 Report did show bright spots of progress in several states where a range of organizations, including the March of Dimes, have been able to reverse the trend and lower the preterm birth rate.

• Rhode Island: Increased leadership and collaboration among Rhode Island’s Department of Health, March of Dimes, and health care systems in the state led to a full percentage point drop in the premature birth rate and Rhode Island moving from a C to a B grade.
• Knox County, TN: Wider use of locally developed and tailored programs, such as group prenatal care, helped Knox County lower its preterm birth rate from 12.5% in 2007 to 9.8% in 2016, an improvement of more than 20% over the past decade.
• Raleigh, NC: Adoption of the North Carolina Pregnancy Medical Home model, which coordinates care for pregnant women, helped the city tackle early elective deliveries and smoking, forcing Raleigh’s preterm birth rate down from 9.9% in 2015 to 9.3% in 2016. Late preterm births also decreased from 6.9% to 6.1%.

While many of the underlying causes of preterm birth remain unknown, March of Dimes leads the fight for the health of all moms and babies by:

• Working to ensure women have access to preventive and supportive care before, during and after pregnancy.
• Delivering programs to improve the care that moms and babies receive. Group prenatal care can reduce health disparities, and March of Dimes is expanding its group prenatal care program, called Supportive Pregnancy Care,
• Empowering families and communities with the knowledge and tools to have healthier pregnancies.
• Supporting moms through every stage of the pregnancy journey, even when everything doesn’t go according to plan. The Share Your Story online community unites and supports families during their most vulnerable time. As families navigate the newborn intensive care unit (NICU), March of Dimes offers information and comfort with its NICU Family Support® program and its My NICU Baby™ App.
• Advancing research at our six Prematurity Research Centers. Experts study the causes of premature birth, knowing that the answers are going to involve a combination of interventions to prevent and solve this urgent health crisis.
• Advocating for policies to protect moms and babies and give them the best possible start.
• Amplifying the voices of all women and their families. We mobilize communities across the country to work toward achieving birth equity.

The March of Dimes Premature Birth Report Card is based on final 2017 natality data from NCHS. Compared to 2016, preterm birth rates in 2017 worsened in 30 states, stayed the same in 6 states and improved in 16 states.

• 1 state –Vermont– earned an “A” on the 2018 Premature Birth Report Card;
• 15 states received a “B”;
• 16 states got a “C”;
• 14 states, Puerto Rico and the District of Columbia got a “D”;
• 4 states (Alabama, Louisiana, Mississippi, West Virginia) received an “F.”

Among the 100 largest U.S. cities, based on the number of births in 2016, Irvine, California once again had the best (lowest) rate of preterm birth at 5.5 percent, and Detroit, Michigan now has the worst (highest) preterm birth rate at 14.5 percent.

https://www.biospace.com/article/releases/health-of-babies-in-the-u-s-continues-to-worsen-march-of-dimes-report-card-shows/

Children’s Hospital of Philadelphia Opens Manufacturing Unit for Disease Therapies

CHOP and CEO Council for Growth Host Grand Opening, Signaling Philadelphia’s Emergence as Nation’s Biomedical Hub    

For over a century after the Civil War, Philadelphia laid claim to being the “Workshop of the World,” producing trains, tools, textiles and a multitude of other manufactured goods. An event today at Children’s Hospital of Philadelphia (CHOP) highlights the city’s present-day role in manufacturing 21st century products: tools for precision medicine.

CHOP partnered with the CEO Council for Growth of the Chamber of Commerce for Greater Philadelphia to host the grand opening of the hospital’s new Clinical Manufacturing Facility, which will produce clinical-grade biotechnology tools, known as vectors, to deliver cell and gene therapy for difficult-to-treat diseases. Today’s opening is part of a broader regional effort to showcase Philadelphia’s position as a hub of scientific and medical innovation, attract world-class talent, generate jobs and drive the regional economy.

“Philadelphia is a city of breakthroughs,” said Madeline Bell, President and CEO of Children’s Hospital of Philadelphia, during the event. She added, “In this facility, we make the tools—the vectors—that scientists use to deliver cell and gene therapies, bringing dramatic precision medicine treatments to patients.”

An earlier version of the hospital’s manufacturing facility developed vectors crucial to last year’s medical milestones—the first-ever gene therapies approved by the U.S. Food and Drug Administration. Collaborating with Penn Medicine scientists, CHOP experts produced the first vectors for CAR T-cell therapy, used against leukemias, and Luxturna, used to treat a form of inherited blindness.

“The Greater Philadelphia region is already a global leader in the field of precision medicine,” said Rob Wonderling, President of the CEO Council for Growth and President and CEO of the Chamber of Commerce for Greater Philadelphia. He noted, “With the opening of this facility we demonstrate our ongoing commitment to the future of this very exciting field of medicine. This investment, in particular, will enhance the capacity to bring more cell and gene therapies to patients, a goal we’re extremely pleased to support.”

To share a parent’s perspective on what these innovations could mean for children and families, Devaki Jean spoke briefly. She is the mother of a child with sickle cell anemia, one of the diseases being targeted with new therapies under development at Children’s Hospital.

Today’s event also featured a panel of research leaders deeply involved in the Clinical Manufacturing Facility. Moderating the discussion was Bryan Wolf, MD, PhD, Chief Scientific Officer at CHOP. Joining him on the panel were Beverly Davidson, PhD, CHOP’s Chief Scientific Strategy Officer and Director of the Raymond G. Perelman Center for Cellular and Molecular Therapeutics, which manages the facility, and Stefano Rivella, PhD, a CHOP hematology researcher with expertise in sickle cell disease.

Two research leaders from the Perelman School of Medicine at the University of Pennsylvaniaalso joined the panel: Carl June, MD, Director of the Parker Institute for Cancer Immunotherapy, who led the team that, along with CHOP and Novartis, pioneered CAR T-cell immunotherapy; and Jean Bennett, MD, PhD, F.M. Kirby Professor of Ophthalmology, an expert in retinal disorders who was one of the team leaders who pioneered Luxturna, the gene therapy for blindness now manufactured by Spark Therapeutics.

https://www.biospace.com/article/releases/children-s-hospital-of-philadelphia-opens-manufacturing-facility-making-tools-for-therapies-that-cure-disease/

Can a Pharma Drug Improve Autism Conditions?

According to the U.S. Centers for Disease Control and Prevention (CDC), about one in 59 babies born in the United States has been identified with autism spectrum disorder. Since 2000, the prevalence of autism in U.S. children has increased by nearly 120 percent, making it the fastest growing developmental disability in the country.

There are no treatments for autism, but that’s not stopping research into the development of therapies for core symptoms of autism spectrum disorder. This week, London-based AMO Pharma reported Phase II results from its investigational glycogen synthase kinase 3 (GSK3) beta inhibitor, AMO-02 (tideglusib), which showed patients dosed with the medication during the trial experienced an improvement in social withdrawal, repetitive behaviors, daily living skills, memory and sleep quality.

Privately-held AMO Pharma said that preclinical studies indicate that GSK3 beta is an enzyme that is overactive in key molecular pathways that are germane to neuronal functioning and neuronal plasticity in neurodevelopmental disorders. GSK3 beta plays an important role in circadian function and in modulating neuroinflammatory processes in the brain. In patients battling neuromuscular diseases, such as congenital myotonic dystrophy and even Duchenne muscular dystrophy, activity of glycogen synthase kinase 3 beta (GSK3ß) has been shown to increase.

The Phase II trial was a 1:1 double-blind study. Patients were treated with either AMO-02 or placebo over a 12 week period. Patients treated with AMO-02 consistently outperformed placebo in measures of social withdrawal and repetitive behaviors), as well as daily living skills, memory and sleep quality, AMO said in a statement. Outcome measures were based on measures including caregiver-and clinician-completed rating scales. A permutation test of efficacy results indicated that probability of false-positives was low, AMO Pharma added. The once-daily treatment for the core symptoms of ASD was found generally safe and well-tolerated, the company said.

Michael Snape, chief executive officer of AMO, said the Phase II TIDE study marks the first time that a GSK3 beta inhibitor has been the focus of a clinical trial for autism spectrum disorder.

“We believe the resulting data represent a positive step forward in validation of GSK3β as a molecular target in the treatment of symptoms associated with ASD beyond any existing published pre-clinical data,” Snape said in a statement.

Autism is known as a “spectrum” disorder because there is wide variation in the type and severity of symptoms people experience.

Joseph Horrigan, AMO’s chief medical officer said that over the past 10 years, there has been little progress in research related to autism spectrum disorder, all while the “incidence of ASD seemingly continues to rise.” The results of the Phase II TIDE trial provide a new level of hope for potential treatment to the conditions of ASD, Horrigan said.

“…treatment with AMO-02 has the potential to offer meaningful, multi-symptom benefit in ASD and merits further study as a potential treatment for ASD,” Horrigan said.

In addition to ASD, AMO is also investigating AMO-02 for the treatment for onset myotonic dystrophy type 1. Earlier this year, the company reported Phase II data that showed AMO-02 demonstrated clinical benefits in those patients, such as an improvement in cognitive function and an increased ability to perform daily tasks. Myotonic dystrophy type 1 is a rare, genetic, life-threatening neuromuscular disorder that causes impairment in muscle function, cognition and quality of life.

In July 2017 the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation for AMO-02. The designation is granted to developmental treatments for rare diseases/disorders that affect fewer than 200,000 people in the U.S.

https://www.biospace.com/article/can-a-pharma-drug-improve-autism-conditions-/

Welltower price target raised to $72 from $68 at MUFG

MUFG analyst Karin Ford raised her price target on Welltower to $72 and kept her Overweight rating after its Q3 earnings beat. The analyst points to the REIT’s “positive trends in the senior housing operating portfolio, outperformance in the newly acquired QCP portfolio, and attractive new medical office investments”. Ford adds that Welltower can accelerate its FFO growth in FY19 thanks to “significant accretion from the QCP transaction, potential upside off a cyclical bottom in the SHOP portfolio, and solid momentum on investments”.

https://thefly.com/landingPageNews.php?id=2814962