3 decades on dialysis: NC kidney patient beats the odds to reach rare milestone

Despite his age, Lloyd Cameron could be a poster child.

Cameron, 57, celebrated his 30th year being on dialysis Tuesday in Wilson.

The lifelong farm worker from Elm City was surrounded by nurses, doctors and friends as the rare anniversary was recognized at DaVita of Wilson.

“I love them all,” Cameron said of his supporters. “They have been so nice and kind to me. I guess that’s why I do so good. They keep me straight in line.”

Cameron has come in to the facility for dialysis three times a week since he was 27.

“I’m happy for being here for 30 years. It’s a big blessing …” Cameron said. “I hope to be here for 30 more.”

Lisa Lewis, a regional coordinator for DaVita, said Cameron’s milestone was unusual.

“Normally, they last maybe five to seven years,” Lewis said. “That is the average lifespan of a dialysis patient, so I would say 30 years is five times that. It’s pretty important. It;s a pretty big thing.”

Wanda Hinnant of Goldsboro, who worked at DaVita for 38 years, remembers treating Cameron when he first came in three decades ago.

“I think he’s a wonderful person,” Hinnant said. “He has taken care of himself, followed the rules, and that’s why he’s still here.”

Dana Barberio, registered nurse and facility administrator, explained what dialysis does.

“Basically, it’s when their kidneys stop working and they have to come in here to dialysis. We basically filter their blood and remove the fluid and toxins in their blood and return clean blood to them, and we basically do what their kidneys would normally do.”

The cleansing sessions take from two to four and a half hours.

The patients get very familiar with the nurses and doctors.

“They are really our family,” Barberio said. “They really do become part of our family, and we care deeply about every one of our patients.”

Barberio said Cameron’s accomplishment is “absolutely amazing.”

“It is a miracle. We very rarely get to celebrate this type of milestone. This is a very rare occasion,” Barberio said. “It is just wonderful to know that a patient has taken care of himself and is able this good and is still able to be with us and dialyze with us after 30 years. It’s wonderful.”

Dr. Anwar Al-Haidary, attending nephrologist and the director for the dialysis unit, said in the past, dialysis got a bad reputation.

“Medicine has advanced and dialysis has advanced to the stage now that people can live a normal life, and they can live up to 30 or 40 years,” Al-Haidary said.

Sometimes people think dialysis is a bad idea, but that shouldn’t be the case anymore, Al Haidary said.

“As you can see, this is a good example,” he said. “People who take their medicines regularly, who follow up with dialysis, can live a normal, long life.”

https://bit.ly/2INZ5Su

Bristol-Myers to webcast ASCO highlights June 4

Bristol-Myers Squibb Company (NYSE:BMY) will hold an investor event on Monday, June 4, 2018 at 8:30 p.m. EDT (7:30 p.m. CDT) to discuss data presented at the American Society of Clinical Oncology (ASCO) in Chicago.

Company executives will provide an overview of data presented from the company’s oncology portfolio, and address questions from investors and analysts.

Investors and the general public are invited to listen to a live webcast of the event at investor.bms.com. Materials related to the event will be available at the same website prior to the event. A replay of the event will be available and can be accessed at investor.bms.com.

https://bit.ly/2ktXS4h

China rare disease list could promote R&D, regulatory filings

Five Chinese government agencies, including the State Drug Administration and the National Health Commission, published a national list of 121 qualified rare diseases to support diagnosis and treatment of such conditions in a move that could spur R&D and regulatory submissions for rare disease therapies.

This is the first time China has recognized rare diseases on a national level, James Xuetold BioCentury. Xue is founder, chairman and CEO of Canbridge Life Sciences Ltd.(Beijing, China) and previously served as first China general manager of rare diseases pioneer Genzyme Corp., which is now part of Sanofi (Euronext:SAN; NYSE:SNY).

He said a publicly available list will help promote R&D, improve review and approval times, and could make it easier for companies to gain reimbursement for their drugs in China.

While China has historically shown a lack of focus on rare diseases, the list is one signal that the area is increasingly becoming a priority for the country.

However, China still has a way to go towards encouraging development of rare disease treatments. For instance, Xue said there is no definition or criteria for what constitutes a rare disease in the country and no national reimbursement policy for Orphan drugs.

Xue said current policies are geared more towards rare disease drugs already approved in western markets. There is “no policy on newly developed drugs in China that encourages companies to take risks and includes exclusivities like pricing reimbursement, or IP,” he said.

Of the 121 diseases on the list, which was released on May 22, 110 already have a drug approved by U.S.FDA, Xue said. The list may encourage sponsors of those drugs to pursue approval in China. It also could facilitate a shorter review timeline and allow them to reference the list to signal an unmet medical need in China while seeking reimbursement, according to Xue.

Additionally, Xue said any company that wants to submit an IND to study a rare disease treatment in China is likely to have receptive discussions with regulators, if the targeted disease is on the list.

Furthermore, he believes the list will be a useful guidance for initiating price reimbursement discussions. He expects guidance will be released to set terms for coverage for rare disease therapies “relative to what other countries price drugs.”

Over the past year, the State Drug Administration (formerly China FDA) has published a series of regulations intended to bring many of the agency’s policies closer in line with western standards.

Draft regulations released in May 2017 set 10 years of exclusivity for innovative drugs for rare or pediatric diseases. In December, SDA issued draft guidelines on a conditional approval pathway for life-threatening rare diseases and guidance to implement a priority review pathway that includes rare diseases. Guidance to encourage development of generics also includes drugs for rare diseases (see BioCentury Extra, Dec. 29, 2017).

Joining SDA and the National Health Commission in compiling the list were the Ministry of Science and Technology (MOST), the Ministry of Industry and Information Technology(MIIT) and the State Administration of Traditional Chinese Medicine of China.

https://bit.ly/2slEoCi

Kite Eyes More Worldwide Facilities, Links To National Cancer Institute

Kite, a Gilead Company (Nasdaq: GILD), today announced it has leased a new facility in the Netherlands to engineer cell therapies in Europe. The 117,000 square-foot site in Hoofddorp (SEGRO Park Amsterdam Airport) will enable Kite to efficiently manufacture and deliver its cell therapies to people living with cancer in Europe and will provide more than 300 new jobs when fully operational in 2020.

The facility will engineer and produce innovative cell therapies, including axicabtagene ciloleucel, a Chimeric Antigen Receptor T cell (CAR T) therapy that is currently under review by the European Medicines Agency and which is approved in the United States as Yescarta^®.

“We are pleased to be leading a new frontier of cancer innovation that is bringing hope for people living with cancer,” commented John F. Milligan, PhD, Gilead’s President and Chief Executive Officer. “This new European manufacturing facility will enable personalized cell therapies to be manufactured in closer geographic proximity to the patients who will receive them, potentially shortening the turnaround time for people who urgently need care.”

In addition to the Netherlands facility, Kite has recently purchased a new building in Santa Monica from Astellas Pharma Inc. that will be used for cell therapy research, development and the expansion of clinical manufacturing capabilities, and has leased a 26,000 square-foot facility in Gaithersburg, Maryland. The Maryland site will support the work of a new Cooperative Research and Development Agreement (CRADA) with the National Cancer Institute (NCI) to develop adoptive cell therapies targeting patient-specific tumor neoantigens. Neoantigens are mutations found on the surface of cancer cells that are unique to each person and tumor, offering the potential for more targeted antitumor activity.

“We are proud to be at the forefront of advancing cell therapy, which we believe has the potential to transform cancer treatment,” said Alessandro Riva, MD, Gilead’s Executive Vice President, Oncology Therapeutics & Head, Cell Therapy. “The addition of these three new facilities and the expanded CRADA with our research collaborators at the NCI will help us bring cell therapies to more people with cancer around the world.”

https://bit.ly/2xk5L5s

Rural NC Has Growing Pains as Novo Nordisk Builds Manufacturing Plant

In late March, Novo Nordisk broke ground on a new manufacturing facility in rural Clayton, North Carolina. It’s not unusual for small communities to both welcome the jobs and business, while expressing concern over the changes to the community, and Clayton is no different.

The manufacturing plant will make active pharmaceutical ingredients (API) for several of Novo Nordisk’s current and future GLP-1 and insulin medicines for diabetes patients. The plan is expected to be fully operational in 2020.

The facility will have 833,000 square feet with a footprint of 417,639 square feet. It is expected to create almost 700 new jobs, bringing the total to 1,700, which is exceptional when you consider that in the 2010 census, Clayton had a population of 16,116, currently around 20,000.

The jobs will have an average salary of $68,000, which is $22,000 more than the current average salary and $15,000 more than the median household income in Johnston County. The company also projects an employment effect during the construction period of up to 2,500 people.

The new site is adjacent to Novo Nordisks’ 457,000 square feet Clayton facility, which has expanded several times since 1996. Novo Nordisk is investing $2 billion into production facilities in Clayton; Malov, Denmark; and Kalundborg, Denmark, with most of it, $1.8 billion, going into the Clayton site.

Still, it’s a bit of a change for the community. The town’s mayor, Jody McLeod, told STAT,“We’ve gone from the farm to the pharm. I used to work in the tobacco fields during the summers. Then those tobacco fields became subdivisions. Then the industry came in.”

It’s not as if the area is unfamiliar with biopharma. It’s basically a suburb of Raleigh, and not far from Durham and Research Triangle Park. This is an area rife with biopharma companies, including Argos Therapeutics, BioAgilytix Labs, bioMerieux,FUJIFILM Diosynth Biotechnologies, and others.

Roger Hill, a minister at the Powhatan Original Free Will, the 130-year-old Baptist Church in the area, tells STAT,“They’ve been good neighbors. I wonder in the back of my mind, with Novo continuing to expand, how long will it be until they’ll consider buying the property our church is on?”

Local officials pledged more than $100 million in corporate subsidies to Novo Nordisk.

The U.S. diabetes market has been tough, with insurers pushing back on pricing for drugs. It’s also a hotly contested market, with Eli Lilly’s Trulicity, an injectable GLP-1, Novo Nordisk’s Victoza, also an injectable GLP-1 battling for market share. Sanofi is also a major player in the diabetes market. In February 2018, Novo Nordisk announced results of its Phase III clinical trial of GLP-1 oral semaglutide. The drug met its primary statistical endpoint, showing significant and superior improvements in HbA1c for all three doses compared to placebo. The 14 mg dose also showed significant and superior weight loss compared to placebo.

The company has nine additional ongoing PIONEER trials of the drug this year and expects to make regulatory submissions in 2019.

The North Carolina plant is in part of what has been dubbed the “diabetes belt,” which ranges from Louisiana up to West Virginia and over to North Carolina. This is where there is a higher rate of diabetes in the U.S.

Before the plants were set in Clayton, Novo Nordisk’s APIs were manufactured in Denmark. The ones in the U.S., such as in Clayton or other areas outside of Denmark, manufactured medical devices or assemble packaging. At this point, although details of what will be manufactured at the Clayton site are vague, it looks like it will make insulin injectables and GLP-1, the hormone first derived from Gila monster venom, that stimulates insulin production. A Novo Nordisk spokesperson said it would “ensure diabetes production capacity in the U.S. for the decade ahead.”

For the most part, local officials are happy about Novo Nordisk’s growth and continuing presence. But it’s not without its downside. It hired a full-time city inspector to deal solely with Novo Nordisk permits. I will also need to manage a $32 million wastewater facility that Novo Nordisk has agreed to build at its own expense.

But it’s a lot of money and jobs coming into the community, and that tends to win over most skeptics. And one of the town’s small business owners, David Townsend, who runs Nancy Joe’s Homemade, a bakery, said sales have grown 300 percent since the company broke ground–a lot of dough.

https://bit.ly/2sl0NQ7

LabConnect Expands Advanced Lab Operations Center 11,000+ Sq Ft in TN

LabConnect, LLC, a leading global provider of central laboratory and support services for biopharmaceutical, medical device and contract research organizations, has completed phase one of an 11,000+ square foot facility expansion in Johnson City, Tennessee. The new space will significantly increase the capacity for peripheral blood mononuclear cell (PBMC) processing and allow LabConnect to better serve its clients.

As a response to its 30 percent growth since 2017, LabConnect’s facility expansion is essential to serving increasingly complex studies and the related demand for corresponding services. Phase one expanded the company’s clinical sample kit-building and storage capabilities. Phase two of the facility upgrade will add more offices and project management space, while phase three will build out the PBMC laboratory capabilities.

“We have grown every year since our founding in 2002, and we are committed to maintaining our facilities with cutting-edge technology,” said Eric Hayashi, president and CEO of LabConnect. “It is an exciting time to be building our business, constantly improving sample testing services to meet clients’ needs and helping to save lives while supporting clinical trials.”

LabConnect’s expansion will be complete in September 2018. A detailed moving plan has been developed to ensure seamless business continuity, regulatory compliance and sample integrity.

For more information, visit labconnectllc.com.

https://bit.ly/2GWJQkv

Novartis’ AveXis to Build Gene Therapy Factory in Durham, North Carolina

AveXis, a Novartis company based in Illinois, is investing $55 million to build a new manufacturing facility in Durham, North Carolina. The facility will create 200 jobs.

AveXis will use the new plant to make its first product candidate, AVXS-101, a gene therapy to treat three types of spinal muscular atrophy (SMA). SMA Type 1, also known as Werdnig Hoffmann disease, is a severe form of SMA. It usually is seen before six months of age and includes worsening muscle weakness and hypotonia (poor muscle tone) because of the loss of lower motor neurons in the spinal cord and brain stem. There are also feeding and breathing problems. The disease is caused by mutations in the SMN1 gene. Currently the only treatment approved by the U.S. Food and Drug Administration (FDA) is Biogen’s Spinraza (nusinersen).

Durham was competing with the state of Illinois for the site. Illinois approved tax credits of almost $8 million to bring the jobs to Libertyville, Illinois. But AveXis took a $3 million incentive package in North Carolina.

“It’s notable that AveXis has chosen to expand its manufacturing operations in North Carolina,” North Carolina’s Secretary of Commerce, Tony Copeland, said in a statement. “Life science companies understand the many advantages of our state offers manufacturers, particularly the investments North Carolina has made in education and workforce training for the biotechnology industry.”

The new jobs will be in engineering, manufacturing and supply chain. They will have minimum average salaries of $103,449, compared to the average wage in Durham County of $67,537.

In addition to AVXS-101, AveXis plans to develop other therapeutics for rare neurological diseases, including Rett syndrome and a genetic type of amyotrophic lateral sclerosis caused by mutations to the SOD1 gene.

“The transformation of gene-based technologies into economic impact in North Carolina continues to be a focus of the Biotechnology Center, not only for human health but also for animal health and agriculture,” said Bill Bullock, senior vice president of statewide operations & economic development with the North Carolina Biotechnology Center,” reported WRAL TechWire.

Pfizer also recently announced it was investing $100 million and adding gene therapy capabilities to its manufacturing campus in Sanford, North Carolina. Bluebird bio, also in Durham, has done the same.

AveXis acquired rights to its gene therapy technology in 2015 from Asklepios BioPharmaceutical (AskBio), a company based in Chapel Hill, North Carolina. AskBio received an upfront payment and is eligible for milestone payments and royalties based on AveXis progress and possible commercialization.

On April 9, AveXis announced it had agreed to be acquired by Novartis for $218 per share or a total of $8.7 billion in cash. This was an 88 percent premium on its April 6, 2018 closing price. Both companies’ boards unanimously approved the merger. The close is expected in mid-2018.

“The commitment, drive and expertise of the entire AveXis team has created significant stockholder value, and we are pleased that Novartis recognizes that value in the potential of AVXS-101, our first in class manufacturing capabilities and our gene therapy pipeline, all of which serve to transform the lives of people devastated by rare and life threatening neurological diseases such as SMA, Rett syndrome and genetic ALS,” said Sean Nolan, AveXis’ president and chief executive officer, in a statement at the time. “With worldwide reach and extensive resources, Novartis should expedite our shared vision of bringing gene therapy to these patient communities across the globe as quickly and safely as possible.”

https://bit.ly/2LEKYN9