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Saturday, December 1, 2018

Catalyst Updates Positive Interim Data from Phase 2/3 Hemophilia Study


Results demonstrate efficacy of subcutaneous prophylaxis with MarzAA, Catalyst’s high potency engineered FVIIa
Data presented at 60th American Society of Hematology Annual Meeting & Exposition
Catalyst Biosciences, Inc. (NASDAQ: CBIO), a clinical-stage biopharmaceutical company focused on developing novel medicines to address hematology indications, today announced additional positive interim data from its Phase 2/3 study of subcutaneous prophylactic Factor VIIa (FVIIa) variant marzeptacog alfa (activated) (MarzAA), currently being developed for the treatment of hemophilia A or B with inhibitors. The data were presented in a poster at the 60th American Society of Hematology (ASH) Annual Meeting & Exposition on December 1, 2018 in San Diego.
“These results support the efficacy of MarzAA as a subcutaneous prophylactic treatment option for individuals with hemophilia A or B with inhibitors,” said Nassim Usman, Ph.D., chief executive officer of Catalyst. “We observed that bleeding in individuals with high annualized bleed rates is significantly reduced or eliminated during MarzAA treatment. With these new data from two additional individuals who have successfully completed the trial, we are optimistic about the potential for MarzAA treatment to achieve extremely low annualized spontaneous bleed rates with individualized, subcutaneous daily dosing.”
Dr. Howard Levy, chief medical officer of Catalyst, presented the updated results, including the new data from two additional subjects who have completed the Phase 2/3 MarzAA trial. The first subject, who had an annualized bleed rate (ABR) of 15.2, had no bleeds during 50 days of treatment with 30 µg/kg MarzAA. The second subject, who had an ABR of 22.2, experienced a bleed on Day 4 that did not require treatment. The subject continued on the 30 µg/kg dose level, as the bleed occurred within the first five days of dosing when FVIIa levels are still increasing to therapeutic levels and completed the trial with no additional bleeds during the treatment period.

Novartis: longer-term analyses from Kymriah trials showed durable responses


Novartis International AG / Novartis announces longer-term analyses from pivotal Kymriah® trials that showed durable responses are maintained in patients with advanced blood cancers . Processed and transmitted by West Corporation.
The issuer is solely responsible for the content of this announcement.
* In the updated analysis from ELIANA, Kymriah demonstrated an 82% remission rate within 3 months in pediatric patients with r/r ALL; relapse-free survival was 62% at 24 months, with median duration of remission still not reached[1]
* The longer follow-up from the JULIET study in patients with r/r DLBCL reported 64% relapse-free probability and a 43% probability of overall survival at 18 months, with median duration of response still not reached[2]
* The safety profiles observed in both longer-term analyses remained consistent with previously reported results, with no emergence of new safety signals
* ASH presentations demonstrate the Novartis commitment to understanding the long-term potential of Kymriah in transforming the treatment of ALL and DLBCL

bluebird Updates β-Thalassemia, Sickle Cell Studies at ASH


All patients with transfusion-dependent β-thalassemia and a non-β00 genotype who achieved transfusion independence continue to maintain it for up to 3.5 years in Phase 1/2 Northstar (HGB-204) study
HbAT87Q levels were stable and vaso-occlusive events were reduced in most Group A and B patients with sickle cell disease in Phase 1/2 HGB-206 study following treatment with LentiGlobin
bluebird bio, Inc. (Nasdaq: BLUE) announced new long-term data from the completed Phase 1/2 Northstar (HGB-204) study of investigational LentiGlobin™ gene therapy in patients with transfusion-dependent β-thalassemia (TDT) and from the ongoing Phase 1/2 HGB-206 study of LentiGlobin in patients with sickle cell disease (SCD) today at the 60th Annual Meeting of the American Society of Hematology (ASH).
“The breadth of our LentiGlobin data at ASH across multiple clinical trials reflects the commitment of patients, families and healthcare providers to investigate the transformative therapeutic potential of gene therapy for the beta-hemoglobinopathies,” said David Davidson M.D., chief medical officer, bluebird bio. “LentiGlobin gene therapy is designed to address the underlying genetic cause of beta-thalassemia and sickle cell disease. The longer-term data emerging from our clinical trials show that most treated patients are producing sufficient amounts of engineered HbAT87Q to achieve and maintain a therapeutic benefit.”

Celgene, Acceleron on Results of Phase 3 Beta-Thalassemia Trial at ASH


Pivotal phase 3 data demonstrated treatment with investigational luspatercept resulted in significant reduction of transfusion burden compared to placebo
Regulatory submissions planned in the United States and Europe in the first half of 2019
Celgene Corporation (NASDAQ: CELG) and Acceleron Pharma Inc. (NASDAQ: XLRN) today announced results from a pivotal, phase 3 trial (BELIEVE) evaluating the safety and efficacy of luspatercept for the treatment of adults with beta-thalassemia-associated anemia who require regular red blood cell (RBC) transfusions. The data were presented by Maria Domenica Cappellini, M.D. in an oral session of the 60th American Society of Hematology (ASH) Annual Meeting and Exposition in San Diego, CA (Abstract #163).
“Currently, the standard of care to help patients with beta-thalassemia manage their anemia is regular, lifelong red blood cell transfusions, which over time can result in iron overload and life-threatening co-morbidities,” said Professor Cappellini, M.D., Professor of Medicine, University of Milan – Fondazione IRCCS. “These findings from the BELIEVE study are exciting because they suggest that luspatercept may help patients reduce their dependence on red blood cell transfusions.”
BELIEVE met the primary endpoint of erythroid response, defined as a ≥33% reduction in RBC transfusion burden (with a reduction of ≥ 2 units of RBC) during weeks 13–24 compared to the baseline 12-week interval prior to randomization. The study also included secondary endpoints that evaluated the impact of treatment on RBC transfusion burden. Mean change in transfusion burden from baseline to weeks 13-24 (luspatercept vs. placebo) was -1.35 RBC units.

Indiana city owes $3M in overdue bills to Cigna in employee health coverage switch


City administrators this week are switching employees to a new health care insurance company as they pay down $4.6 million in overdue payments to their former insurance company, CIGNA.
Gary Mayor Karen Freeman-Wilson said the the city paid CIGNA$1.6 million in overdue payments this week and will pay the remaining $3 million balance to CIGNA by the end of the year.
The city is switching from CIGNA to AIM Medical Trust, resulting in nearly $800,000 in savings.
“This was solely the city’s decision to go with a more competitively priced plan, while providing a similar level of coverage for employees,” she said. “There will be added costs for co-pays for medication. However, there are also added savings for hearing aids, office visits and certain diseases.”
The switch to a new health care company is part of the administration’s financial recovery plan aimed at tackling the city’s $17 million budget deficit.
The mayor has said the plan is critical to addressing longstanding debt in the city and getting Gary on stable, financial footing.
Through a combination of aggressive permit and licensing fee collections, department consolidation, revenue generators and economic development, Freeman-Wilson has said she hopes city government can match spending levels with revenue in due time.

FDA picks eight medical device firms to help battle opioid crisis


Eight medical device makers, including a startup that uses virtual reality to treat chronic pain, topped an innovation contest aimed at addressing the opioid crisis, the U.S. Food and Drug Administration said on Friday.
Silicon Valley-based startup CognifiSense, which is developing the virtual reality therapy, and iPill Dispenser, which uses a biometrically controlled mobile app that aims to cut overconsumption by dispensing pills based on prescriptions, were among the winners of the FDA’s contest.
The health regulator will work directly with the companies to expedite the development and review of their devices in a manner similar to the agency’s Breakthrough Devices Program, which fast-tracks the review of certain products.
Over 72,000 Americans died from drug overdose last year, including illicit drugs and prescription opioids, with President Donald Trump declaring the opioid addiction crisis a public health emergency.
The FDA received over 250 applications for the innovation challenge, which seeks to prioritize the approval of novel medical devices including digital health technologies such as mobile medical apps.
Under Commissioner Scott Gottlieb, the FDA has raised the bar for approval of opioid-based painkillers by restricting the distribution and use of these drugs. The agency has also rolled out initiatives to encourage developers of alternative therapies.
“We believe the greatest opportunities for medical devices to help prevent opioid use disorder are devices that could help identify people likely to become addicted, devices that manage pain as an alternative to opioids or reduce the need for opioid medications,” the FDA said in a statement here.

Providers welcome interstate licensing, while unions oppose it


When a Sanford Health hospital in North Dakota received an influx of patients recently, the parent organization was able to send nine critical-care nurses from a hospital in South Dakota to help.
The drive took nearly four hours. But if the states weren’t part of a compact that allows nurses to practice across state lines, the licensing process to let those nurses practice in North Dakota would have taken weeks or even months .
The compact is vital to Sanford, said Meghan Goldammer, senior vice president for nursing and clinical services.
“Healthcare doesn’t stop at state lines,” she said. “Those patients in the critical-care unit in North Dakota potentially would’ve had to travel somewhere else, taking the family away from their home and livelihood.” It could also jeopardize the patient’s health, Goldammer added.
Providers like Sanford support adding more states to the interstate licensing compacts because it would allow them to be nimbler and adapt to fluctuating demand. Getting licenses for individual states is a major drain on medical professionals’ time and resources. They also contend the current system limits providers’ ability to follow up with patients in noncompact states and treat them via telemedicine since licenses are based on where the patient is, not the physician or clinician.
Sanford has an internal group of about 50 travel nurses who can move seamlessly to the states in the Nurse Licensure Compact. Currently 29 states are in the compact, with Louisiana and Kansas joining in 2019 and legislation pending in a few other states. This is particularly helpful as many of the health system’s nurses near retirement, which is expected to widen the gap between the available supply of nurses and the growing demand. It also makes a big difference for Sanford’s rural facilities, which typically struggle to attract employees.
Sanford spends less on locum tenens, or temporary nurses, and can maintain the same level of care quality and continuity, Goldammer said.
The health system can send nurses freely to each state it operates in, except for one—Minnesota.
“It would be a huge benefit if they would join the compact, especially for our patients,” she said. “When we need to send a nurse to Minnesota, it requires a nurse to apply for a separate license, which could set us back two to three months.”
But the compacts are opposed by representatives of labor unions who say that granting interstate licenses cedes control. They argue that the compacts create a loophole that invites lower-quality medical professionals with questionable backgrounds. Unions also claim that in the states where they are active, continuing education requirements, among other mandates, are more stringent than others.
“State governments believe it is their obligation to make sure someone rendering care in their state meets the state legislature’s standards,” said William Horton, a partner at law firm Jones Walker, adding that states are eager to retain their policing ability. “Keeping control within the licensing process also means those who have licenses have some degree of protection from competitors.”
In addition to nursing, there are compacts for physicians, physical therapists and advanced practice registered nurses. The physician compact, or interstate medical licensure compact, includes 24 states while the physical therapist compact has 21 states. Three states are part of the advanced practice registered nurse compact.
The nursing compact works a bit differently than the agreement for physicians. When nurses earn their interstate license, they can immediately practice in any participating state. Physicians with their interstate license still have to apply through individual states, although they can do so in a matter of days rather than three to six months. This also gives state medical boards some control.
The National Council of State Boards of Nursing implemented the nursing compact in 2000, driven by the introduction of new technology that required a more fluid licensing process. By 2015, it had 25 members. The second iteration of the nursing compact took effect in January and 24 of the 25 original states signed on, with Rhode Island not participating this time around after union pushback.
The enhanced nursing compact includes a list of uniform licensing requirements, as well as federal and state criminal background checks to ensure that the nurse has no state or federal felony convictions or misdemeanor convictions related to nursing.

Quality concerns

In highly unionized Massachusetts, worker representatives say the nursing compact has a loophole that could compromise patient safety. Joe-Ann Fergus, director of membership at the Massachusetts Nurses Association, claimed that if a nurse gets into legal trouble with one state, that person can go to another state in the compact to avoid prosecution.
Massachusetts also has more stringent continuing-education requirements than is required by the compact, and the compact would disrupt their contract negotiations, Fergus added. “It’s a solution in search of a problem. There’s no need for it in Massachusetts.”
Maryann Alexander, chief officer of nursing regulation at the National Council of State Boards of Nursing, rebutted the concerns about patient safety. Also, she said there is no evidence that continuing education improves care.
“All those years of data told us that the compact was 100% safe,” Alexander said. “There were no incidents of increased discipline of unsafe nurses across state borders.”
The defense for state-by-state licensing is weaker because accepted clinical practices have become more national in scope, Horton said.
A lot of the tension is in underserved areas where telemedicine has the potential to make a huge difference, he said. “That’s one of the speed bumps in the expansion of telemedicine,” Horton said.
Expanding the physician compact could make a significant dent in the shortage of behavioral and mental health clinical personnel, said Ralph Henderson, president of professional services and staffing for AMN Healthcare, a staffing agency. “Multistate licensure increases access to care in underserved areas,” he said. “There are so many benefits with all of the skill shortages, it’s a little crazy that all states haven’t adopted it.”
When healthcare providers wanted to pitch in to help the relief effort in Louisiana following Hurricane Katrina, they were delayed due to confusion over liability issues. Louisiana at the time was not a nursing compact member. Compact states can respond to disasters quicker, rather than waiting for a governor’s emergency order allowing licensing flexibility, Alexander said.
“The benefits outweigh the detractions,” Henderson said. “It’s getting harder and harder to resist.”
The National Council of State Boards of Nursing will work with five states during their upcoming legislative sessions as they weigh joining the nursing compact; Minnesota is one of the states involved.
Eighty percent of nearly 21,000 nurses surveyed by the Minnesota Board of Nursing were in favor of their state joining the compact.
Yet it’s unlikely any legislation will be passed in the upcoming session, said Kate Johansen, the director of government relations for Mayo Clinic.
Newly elected Gov. Tim Walz isn’t likely to rock the boat, she said, especially since the compact is opposed by the Minnesota Nurses Association, which endorses many of the state’s public officials.
Since Mayo is a healthcare destination, nurses and doctors are limited in terms of their follow-up care of patients who live in noncompact states, Johansen said. “We deliver care in ways that are more mobile than ever,” she said. “Anything that helps streamline care is really the goal we should be pursuing. The nursing compact is tested.”
The outdated state-by-state licensing system doesn’t fit healthcare’s evolution, Alexander said.
“We have huge changes in our healthcare system related to education, online tools and accessibility, and yet we have this 100-year-old licensing system that many states, primarily because of their unions, are reluctant to change,” she said. “What was good for us 100 years ago is not good for us now.”