UroGen Taps Oncology Veteran Elizabeth Barrett as Its New CEO

Novartis and Pfizer veteran Elizabeth Barrett will take over as the new president and chief executive officer of UroGen. Barrett, who most recently helmed Novartis Oncology, will take over as Ron Bentsur steps down from his role as CEO.

Barrett will make her first official appearance as CEO of UroGen at the J.P Morgan Healthcare Conference in San Francisco next week. Barrett has more than 30 years of experience in the field of oncology. During that time, she has gained considerable expertise in pharmaceutical development and commercialization of oncology products. That experience will be critical as UroGen looks to make the leap into becoming a leading commercial-stage entity in the urological setting.

Barrett takes over as the company moves forward with a goal of developing the first drug approved for low-grade upper tract urothelial cancer. In December, New York-based UroGen initiated a rolling submission to the U.S. Food and Drug Administration for a New Drug Application for UGN-101, (mitomycin gel). The company hopes to see approval of the asset this year. The company’s pipeline is built on its RTGel technology platform, a sustained release, hydrogel-based formulation.

Arie Belldegrun, the chairman of the board of directors for UroGen and the former Kite Pharma executive, touted Barrett as an executive who has proven to be “an exceptional leader with a well-established track record in oncology.” Belldegrun said she has the “vision and experience” to lead UroGen as the company moves forward with the potential commercialization of UGN-101.

Barrett joins UroGen from her brief tenure as head of Novartis Oncology. Prior to that, she served as Global President of Oncology at Pfizer Inc.

In a posting on her LinkedIn account, Barrett said she chose to leave big pharma for a smaller company for several reasons. Over the course of her career, she has seen a number of significant innovations in oncology, but she said uro-oncology has been a field that “innovation has left behind.”

“Therapeutic options to manage urologic cancers are limited, and for many patients, the only treatments are surgical, involving removal of the kidney, bladder and/or ureter. UroGen is the first to take important steps to treat these patients differently,” she wrote.

Barrett pointed to the RTGel platform as a potential solution to the unmet needs in uro-oncology. She said the “simple elegance” of the platform has the “potential to revolutionize how we treat urologic cancers and beyond.” In her post, she said she wants to be part of that revolution.

Barrett added that she has had an opportunity to be entrepreneurial in her various roles throughout her career, but with UroGen, she can take those lessons and apply them to a smaller company that is on the “cusp of transformation.”

“I cannot think of a more exciting time to join UroGen and work with its outstanding team as we begin to revolutionize uro-oncology and beyond,” she said in a statement.

As Bentsur steps away from his role with the company, he said he believes Barrett will have the experience to build on the clinical successes he oversaw and guide the company through commercialization.

https://www.biospace.com/article/urogen-taps-oncology-veteran-elizabeth-barrett-as-its-new-ceo/

Tiburio Launches With $31M, 2 Ipsen Rare Disease Compounds

New biotech Tiburio has surged onto the scene with $31 million in financing and two novel Phase II-ready assets licensed from Ipsen Pharma. The new company, spun out of orphan drug accelerator Cydan, is focused on rare endocrine diseases.

Abraham N. Ceesay has left his role as chief operating officer of scPharmaceuticals to take over as the new chief executive officer of Tiburio, which will be based in Cambridge, Mass. In an interview with BioSpace, Ceesay said he was drawn to the Tiburio because of its focus on developing therapies for unmet needs in neuroendocrine diseases.

“This was an opportunity to build a special company… that truly has a clinical stage pipeline,” Ceesay said.

The assets Tiburio secured from Ipsen are TBR-760, which is being developed for the treatment of an unmet need in non-functioning pituitary adenomas (NFPAs) and TBR-065 for treatment for rare endocrine diseases that the company has not disclosed. Both compounds are dopamine-somatostatin chimeric molecules, which inhibit NFPA cell proliferation and have the potential to shrink or halt tumor growth.

Ceesay noted that TBR-065, the second compound the company licensed, has a potentially wider scope than TBR-760. He said the company sees the compound for an unmet need in treating NFPAs, but has a “little more work to do to determine what the most appropriate target indication is for that.” Ceesay plans to initiate the first trial for TBR-760 in the second half of 2019. As the company moves forward with the two compounds, Tiburio will have two former Ipsen executives, Heather Halem and Michael Culler, the former head of endocrinology research, to help guide them through development.

NFPAs are non-metastatic tumors in the pituitary gland in the brain. Their development can result in life-altering and potentially life-threatening consequences for patients. Currently there are no approved therapies to treat these tumors and patients must undergo transsphenoidal surgery and/or radiation to remove or shrink the tumor.

Ceesay said the company was drawn to the two Ipsen compounds because of the unmet need in treating NFPAs. He said the current standard of treatment is an “aggressive and invasive neurosurgery” to remove the tumors. He said the fledgling company sees the potential to bring the first approved therapy to halt pituitary adenomas without the need for the surgery or radiation treatment.

With these therapies, as well as others that can be developed or acquired, Ceesay said there a real opportunity for innovation in the neuroendocrine space. He wants to establish Tiburio as a significant player in that arena. Ceesay said the company plans to be opportunistic about future opportunities in the space, but did not provide any details about what that could be.

As Tiburio moves forward, Ceesay said he intends to draw on his past experience from his days at Genzyme and build a strong relationship with clinical and patient advocacy groups in the neuroendocrine arena.

“In all rare diseases, the community… is critical as you think about establishing a company and developing a therapy. We are building our relationships with patient organizations to garner guidance we need along the way,” Ceesay said. “I hold those lessons tight. At the end of the day, what we do is truly about the patients.”

Tiburio launches with $31 million in a Series A financing round that will fund the companies’ lead compound, TBR-760, through human proof-of-concept for the treatment and further clinical assessment of TBR-065. The Series A was supported Cydan’s syndicate of leading life sciences investors, including New Enterprise Associates, Longitude Capital, Lundbeckfond Ventures, and Alexandria Venture Investments. As part of the licensing agreement, Ipsen will have a minority stake in Tiburio, and will also be eligible to receive development and commercial milestone payments and royalties on sales.

https://www.biospace.com/article/tiburio-launches-with-31-million-and-two-compounds-from-ipsen-targeting-rare-endocrine-diseases/

After Opioid OD, ‘Most Patients Can Leave Hospital in an Hour’

People who overdose on opioids can often be saved quickly with a dose of naloxone, but it hasn’t been clear how long someone should be kept in the hospital after being revived.

Now, new research bolsters existing criteria that say most patients can be discharged from the emergency department in as little as an hour after getting naloxone.

The criteria for release specify pulse, blood pressure and breathing within normal ranges, and that patients can walk out of the hospital under their own steam.

“The landscape of opioid use disorder has changed dramatically,” said study author Dr. Brian Clemency. He is an associate professor of emergency medicine in the Jacobs School of Medicine and Biomedical Sciences at the University at Buffalo, in New York.

Naloxone used to be given intravenously by doctors, nurses and paramedics. Now, it’s available to the public (brand name Narcan) and is often given as a nasal spray, the researchers explained.

Still, how patients are released after getting naloxone is inconsistent, Clemency noted. Some are released immediately, while others are watched for six hours or more.

To figure out the best policy, Clemency and his colleagues studied patients who arrived by ambulance after receiving naloxone.

An hour after receiving naloxone, the nearly 540 patients had their vital signs evaluated. Most patients were observed for four hours before being discharged, the investigators found.

Most adverse events seen in patients whose vital signs were normal after receiving naloxone were minor and not likely to be life-threatening, the study authors said.

Based on these findings, the one-hour rule appears to be valid for patients with normal vitals after getting naloxone, according to the researchers.

“This rule is a way to predict which patients will have adverse outcomes after they overdose on opiates,” Clemency said in a university news release. “The rule is simple to follow and can be used by health care providers with varying levels of training and experience.”

The researchers said they hope their findings will lead to a standardized policy.

The report was published Dec. 28 in the journal Academic Emergency Medicine.

More information

For more on drug overdose, visit the Drug Policy Alliance.

SOURCE: University at Buffalo, news release, Dec. 28, 2018

https://consumer.healthday.com/bone-and-joint-information-4/opioids-990/after-opioid-od-most-patients-can-leave-hospital-in-an-hour-740969.html

Study Eases Concern That Common Diabetes Med May Harm Bones

 If you have type 2 diabetes and you’re taking canagliflozin to help control your blood sugar, a new study has some good news for you: The drug doesn’t appear to raise the risk of bone fractures.

Previously, research had suggested this might be the case.

“We were interested in doing this study because there was one randomized trial that said there was an increased risk of bone fractures and another that said there wasn’t. So, we conducted a real-world study with almost 200,000 people with type 2 diabetes,” said study author Dr. Michael Fralick.

“I hope these findings are reassuring to patients and physicians because these are blockbuster medications for type 2 diabetes. This class of medicines can improve blood sugar levels and help reduce heart disease risk,” he said. Fralick is from the division of pharmacoepidemiology and pharmacoeconomics at Brigham and Women’s Hospital in Boston, and a general internist at the University of Toronto.

Canagliflozin (Invokana, Invokamet) is one drug in a class of medications called SGLT-2 inhibitors. Other drugs in this class include dapagliflozin (Farxiga) and empagliflozin (Jardiance).

These drugs cause the kidneys to remove excess sugar from the blood and excrete it through urine, which lowers blood sugar levels, according to the U.S. Food and Drug Administration. This class of drugs has been linked to a number of complications, including kidney injury and serious genital infections.

Fralick said one way these drugs could potentially increase fracture risk is by lowering bone mineral density.

Dr. William Leslie, author of an editorial accompanying the study, suggested that dehydration may be another way these drugs might be linked to fracture risk. Leslie is a professor of medicine and radiology at the University of Manitoba in Canada.

For the new report, Fralick and his team reviewed data from two U.S. commercial health care databases. They found information on about 200,000 people with type 2 diabetes who were just starting to take one of two different type 2 diabetes medications — canagliflozin or a medication in a class of drugs called GLP-1 agonists, which includes Victoza, Trulicity and Byetta. These drugs haven’t been linked to an increased risk of fractures.

The researchers looked for fractures in the upper and lower arms, as well as the hips and pelvis.

In the end, the study team compared approximately 80,000 people on canagliflozin to about 80,000 treated with GLP-1 agonists. The patients’ average age was 55, and about 48 percent were female.

The study showed a similar risk of fractures in these low-risk, middle-aged populations.

Both Fralick and Leslie said the jury is still out for people who are at a higher risk of fractures, such as elderly people.

This study is “a relatively low-risk population. But, it begs the question, what about higher-risk populations? We need additional safety data,” Leslie said.

The U.S. Food and Drug Administration currently requires canagliflozin labels to carry a warning about the potential fracture risk, and Fralick said it may be too soon to change the labeling, particularly for people at high risk. Both experts said more research is needed.

In the meantime, if you’re concerned about taking canagliflozin, Fralick recommended having a conversation with your health care provider. But, he added, “For people without a high baseline risk, the risk of fracture is very small and the clear benefits to SGLT-2s outweigh that potential risk.”

The findings were published online Jan. 1 in the Annals of Internal Medicine.

More information

Learn more about oral diabetes medications from the American Diabetes Association.

SOURCES: Michael Fralick, M.D., S.M., division of pharmacoepidemiology and pharmacoeconomics, Brigham and Women’s Hospital, Boston, and general internist, University of Toronto, Canada; William Leslie, M.D., professor of medicine and radiology, University of Manitoba, Canada; Jan. 1, 2019, Annals of Internal Medicine, online

https://consumer.healthday.com/diabetes-information-10/diabetes-drug-news-179/study-eases-concern-that-common-diabetes-med-might-harm-bones-741044.html

Hospitals’ outpatient revenue nearing inpatient

The gap between U.S. hospitals’ outpatient and inpatient revenue continued to shrink in 2017 as more patients elect to get care in cheaper outpatient settings, and some believe a flip is inevitable in the coming years.

The American Hospital Association’s 2019 Hospital Statistics report showed hospitals’ net outpatient revenue was $472 billion and inpatient revenue totaled nearly $498 billion in 2017, the latest year for which the report covers, creating a ratio of 95%, up from 83% in 2013.

“It certainly reflects continued efforts from hospitals to make sure that people get the right care at the right time in the right setting,” said Aaron Wesolowski, the AHA’s vice president of policy research and analytics. “We’ve seen inpatient utilization drops in recent years and outpatient has increased.”

Hospital profits reached $88 billion in 2017, a 12.5% increase over the previous year and a 27% increase from 2013. Total net revenue reached $1 trillion in 2017, compared with $998 billion in 2016. Expenses in that time were $966 billion, up from $920 billion. The AHA provided Modern Healthcare with an exclusive copy of the report.

The jump in profit in 2017 was higher than in recent years. In 2016, profits increased 4%, compared with a 0.25% decline in 2015.

During that time, operating revenue increased just 4.6% in 2017, a lower rate than 2016 and 2015. During the same time, nonoperating revenue, which includes investment income, jumped 92%, slightly lower than 2016’s 103% spike. Wesolowski attributes the slowed operating revenue growth to stabilizing utilization.

“That’s certainly a heavy driver,” he said.

The data illustrate the continued slow bleed of patients out of hospitals. Admissions to the 5,262 U.S. community hospitals and their nursing home units increased by less than 1% to 34.3 million in 2017, from 34 million in 2016. Community hospitals include nonfederal, short-term general and special hospitals. Inpatient days were largely unchanged at 186.2 million during that time. Both inpatient surgeries and births declined slightly between 2016 and 2017 to 9.1 million and 3.7 million, respectively.

But the gradual decline in inpatient volumes didn’t translate into a significant outpatient boost in 2017. Outpatient surgeries were mostly flat at 19 million, as were emergency room visits at 144.8 million. Outpatient visits inched up by a modest 1.2% year-over-year, to 766 million in 2017. Those numbers generally include urgent care and ambulatory surgery center visits, except for cases when the facilities are not directly affiliated with a specific hospital, AHA spokeswoman Marie Johnson wrote in an email.

Even as outpatient volumes were relatively flat year-over-year, net outpatient revenue increased 5.7% between 2016 and 2017. Chuck Alsdurf, director of healthcare finance policy and operational initiatives at the Healthcare Financial Management Association, said it’s not surprising to see outpatient revenue grow as procedures increasing shift into outpatient settings. He said part of that is driven by the CMS’ two-midnight rule created in 2013, which directs Medicare contractors to deem a hospital admission legitimate if it spans two midnights.

“Typically when things move from inpatient to outpatient, especially on a procedural basis, the charges go down, as well as the payment,” Alsdurf said.

Eventually, Alsdurf predicts hospitals’ outpatient revenue will eclipse inpatient revenue, but that’s still several years out.

The amount hospitals reported having spent on uncompensated care was $38.4 billion in 2017, the same as in 2016.

There were 6,210 total hospitals in the U.S. in 2017, according to the AHA’s data. That’s nearly 9% more than a decade earlier, in which the U.S. had 5,708 hospitals, but slightly fewer than in 2016, in which there were 6,168. The total hospitals figure includes not-for-profit, investor-owned, state and local government-owned hospitals and veterans hospitals.

The number of rural community hospitals declined nearly 8% between 2017 and 2013, to 1,875, while the number of urban hospitals increased nearly 2% during that time to 3,387.

Among community hospitals, 3,494 were part of a system in 2017, up from 3,467 in 2016 and 3,322 in 2013. Fewer hospitals are members of group purchasing organizations in 2017 compared with 2016: 3,583 versus 3,726.

The number of full-time and part-time community hospital employees increased slightly to 4.5 million and 1.6 million, respectively, in 2017.

Adjusted expenses per inpatient stay were higher at not-for-profit hospitals in 2017 compared with their investor-owned counterparts. Not-for-profits spent $13,504 per inpatient stay, while investor-owned hospitals spent $10,273 for the same stay. That number was even higher for state and local government hospitals: $14,015.

That’s not surprising given investor-owned hospitals have a completely different operating model, Alsdurf said. They staff differently, and their scale allows them greater purchasing power and efficiency. Plus, there’s pressure from Wall Street.

“I still think the biggest reason is because they’re run to make money for the investors,” he said. “So there is a different goal.”

https://www.modernhealthcare.com/article/20190103/TRANSFORMATION02/190109960

Wave Life Sciences Duchenne Trial Selected for FDA Design Pilot

Wave Life Sciences Ltd. (WVE), a biotechnology company focused on delivering transformational therapies for patients with serious, genetically-defined diseases, today announced that the planned Phase 2/3 efficacy and safety trial for its lead Duchenne muscular dystrophy (DMD) clinical program has been selected for the U.S. Food and Drug Administration (FDA) pilot program for complex innovative trial designs (CID). The selection was based on the design of Wave’s Phase 2/3 clinical trial of suvodirsen (WVE-210201), an investigational therapy for boys with DMD who are amenable to exon 51 skipping. This marks the first time that the FDA has selected clinical protocols for its CID pilot program that was announced in August 2018.

In evaluating submissions for the CID pilot program, the FDA considered two key criteria: the innovative features of the trial design and the therapeutic need (i.e., therapies being developed for use in disease areas where there are limited or no treatment options). Wave’s application for the CID pilot program includes a plan to leverage DMD historical control data to augment the placebo arm of the suvodirsen Phase 2/3 clinical trial, among other innovative design elements. Through this pilot program, Wave intends to reduce the number of patients required to deliver conclusive clinical efficacy results, thereby minimizing the number of patients required in the placebo treatment arm and potentially accelerating study completion. As a participant in the pilot program, the company will also have additional opportunities to meet with FDA staff to discuss the design elements of the trial, including the use of Bayesian methods to adapt the trial and allow for more efficient and productive clinical determinations. Details of Wave’s Phase 2/3 trial design will be presented at upcoming scientific meetings.

“In designing our clinical trials, we are constantly looking to maximize the probability of a definitive result, incorporate the feedback of patients and their families, and reduce the burden on those who are already bravely enduring the challenges associated with serious, genetically-defined diseases. The FDA’s recognition of our plan reflects the thoughtful and collaborative way in which we approach clinical development,” said Michael Panzara, MD, MPH, Chief Medical Officer of Wave Life Sciences. “We look forward to further discussions with the FDA in the coming months and sharing learnings from our trial design with others in the rare disease drug development community to drive greater efficiency and productivity in future clinical studies.”

Wave anticipates initiating the global, placebo-controlled Phase 2/3 efficacy and safety clinical trial of suvodirsen in DMD patients amenable to exon 51 skipping in 2019. The trial is designed to measure clinical efficacy and dystrophin expression, and Wave intends to use the results of this trial to seek regulatory approvals globally. Currently, suvodirsen is being studied in an ongoing open-label extension (OLE) study and Wave expects to deliver an interim analysis of dystrophin expression from this study in the second half of 2019.

The FDA CID pilot program is an initiative under the 21^st Century Cures Act, with an objective to modernize clinical trial design, help streamline and advance drug development and inform easier regulatory decision-making. In order to qualify for the CID pilot program, companies must intend to provide substantial evidence of efficacy through a complex, novel design that incorporates innovative trial design elements such as seamless trial designs, modeling and simulations to assess trial operating characteristics, the use of biomarker enriched populations, complex adaptive designs, Bayesian models and other benefit-risk determinations, among others. For more information, visit https://www.fda.gov/Drugs/DevelopmentApprovalProcess/DevelopmentResources/ucm617212.htm.

https://finance.yahoo.com/news/wave-life-sciences-duchenne-muscular-130000339.html

Vertex, Arbor to Collaborate on Novel Proteins for Gene-Editing Therapies

Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) and Arbor Biotechnologies (Arbor) today announced that the two companies have entered into a strategic research collaboration focused on the discovery of novel proteins including DNA endonucleases to advance the development of new gene-editing therapies for cystic fibrosis and four other diseases to be selected later. Arbor has developed a protein biodiscovery platform that includes a comprehensive set of technologies and techniques, integrating machine learning, genome sequencing, gene synthesis, and high-throughput screening that will augment Vertex’s efforts to develop gene-editing approaches for the treatment of serious diseases.

“Our strategy of developing transformative medicines for serious diseases is based on addressing causal human biology with innovative therapeutics,” said David Altshuler, M.D., Ph.D., Executive Vice President, Global Research and Chief Scientific Officer of Vertex. “Arbor’s proprietary high-throughput screening platform will enhance our ongoing efforts to develop innovative gene-editing therapies.”

“Vertex has a proven track record in discovering and developing innovative medicines and is an ideal partner for Arbor,” said David Walt, Ph.D, Arbor Co-Founder. “We believe that by using our powerful biodiscovery platform we will be able to identify complementary and next generation tools to enhance Vertex’s pipeline of transformative medicines and new gene-editing therapies.”

https://www.businesswire.com/news/home/20190103005194/en/Vertex-Arbor-Biotechnologies-Establish-Collaboration-Discover-Proteins