Novartis strikes deal with Chinese firm to make Kymriah gene cancer med

Novartis has enlisted Chinese manufacturer Cellular Biomedicine (CBMG) to make its $475,000 (362,663 pounds) gene-modifying cancer treatment Kymriah as the Swiss drugmaker intends to win approval for the therapy in the world’s most populous country.

Local regulations require that Novartis manufactures Kymriah in China if it wants to treat the country’s patients with the drug.

The Chinese company’s shares rose more than 16 percent in pre-market trading after the deal was announced on Thursday. Novartis shares closed 0.9 percent higher.

Basel-based Novartis is paying $40 million to buy 9 percent of CBMG’s shares, the Chinese company said. Novartis also gets rights to develop and sell products using CBMG technology in the deal.

The deal fits Novartis’ push to expand its global manufacturing footprint for Kymriah, a one-time, personalised CAR-T therapy in which doctors remove disease fighting T-cells from individual patients to be modified to attack cancer before being re-infused into patients.

Novartis recently announced production pacts in France, Germany and Switzerland, where it is building its own Kymriah factory. Novartis would lead distribution, commercialisation and approval efforts within China, it said, but declined to give the status of Kymriah’s progress with Chinese drug regulators.

“For proprietary reasons, we cannot disclose our strategy in China,” a spokeswoman said in an e-mail. “However, our collaboration with CBMG is a step towards our efforts to bring Kymriah to patients in China.”

The treatment, aimed at patients who have failed to respond to other drugs, has already been approved in Europe and the United States to treat gravely ill children with acute lymphoblastic leukemia (ALL), as well as adults with diffuse large B-cell lymphoma (DLBCL).

Novartis is initially rolling out the treatment in Europe just for young patients with ALL, after encountering problems with the quality of batches for DLBCL patients. The company has said the treatment for DLBCL patients requires additional work to meet commercial specifications.

A similar CAR-T therapy, Gilead Sciences Yescarta, has also been approved for DLBCL patients in the United States and Europe. Last year, Yescarta’s maker began manufacturing Yescarta in China, part of its bid for eventual approval there.

https://www.marketscreener.com/CELLULAR-BIOMEDICINE-GROU-16860801/news/Novartis-strikes-deal-with-Chinese-firm-to-make-Kymriah-27332205/

China’s GenScript Hit on Report Critical of Unit’s Gene-Therapy Tests

GenScript Biotech Corp.’s stock tumbled nearly 27% on Thursday following a research report alleging safety violations by the Chinese company’s subsidiary that has partnered with Johnson & Johnson.

The report alleged that Nanjing Legend Biotechnology Co. bypassed standard safety procedures while testing an experimental gene therapy on Chinese patients. It accused the parent company, GenScript, of cherry picking results disclosed to investors. GenScript, which suspended trading in Hong Kong after its shares slipped to 11.86 Hong Kong dollars, and Legend deny any wrongdoing.

Little is known about Flaming Research, the compilers of the report. The group’s website describes it as a “a team of seasoned equity investors who are sickened by the inability of Hong Kong regulators” to crack down on corporate fraud and who “will be exposing fraudulent companies to protect the interest of minority shareholders.”

The website doesn’t list a corporate address or a telephone number. Flaming couldn’t be reached for comment.

Still, the accusations made in its 14-page report spooked investors.

In December, impressed with early results from patient tests in China, J&J’s drug unit paid $350 million for the global rights to co-develop and market Legend’s experimental therapy. The companies are testing it on patients in the U.S. A J&J spokesman said the company is looking into the report.

Legend’s progress is closely watched because it is the first Chinese company to get approval to test a gene therapy in the U.S. It is also one of the few companies testing a CAR-T candidate.

CAR-T therapy involves extracting patients’ disease-fighting white blood cells, genetically modifying them to more vigorously attack cancer, then re-injecting them into their bodies.

The first CAR-T therapy, Novartis AG’s Kymriah to treat aggressive forms of leukemia, was approved last year. The treatment costs hundreds of thousands of dollars for its potential to cure patients. Legend’s targets multiple myeloma.

Among the safety issues that Flaming alleges is an instance where a patient’s relative transported cells hundreds of miles from Xi’an, where they were collected at a hospital participating in Legend’s research, to Nanjing, where the company is based and conducts the cell modifications.

Legend Chief Executive Frank Fan said doctors extracted and sealed the sample, suggesting there was no risk of contamination, and that the relative volunteered to take it because at the time Legend lacked a reliable way to transport samples. Samples are tested for quality before being modified, he said.

China didn’t have rules for gene-therapy experiments until a few months ago.

Flaming also slammed the company for administering its therapy through regular syringes instead of the intravenous infusions Novartis uses. Dr. Fan said the doses were small enough to be administered through regular syringes and that doing so is safe.

Flaming further said that Legend’s early results may be fraudulent because, on follow-up visits, patients were evaluated with blood tests instead of a more accurate bone-marrow biopsy. Dr. Fan said biopsies were done, but not on every visit. The procedure, he said, “is painful and stressful and cannot, and does not, need to be done each time.”

Flaming accused GenScript of cherry picking what it disclosed to investors, and when, in an attempt to keep its stock price high. In one instance, it said the company delayed publicly announcing a trial death until after posting promising early results. GenScript said it would respond with a statement later Thursday.

Even after Thursday’s drop, its stock was three times what it was in June last year, when Legend unveiled its experimental therapy at a cancer conference in the U.S.

https://www.marketscreener.com/GENSCRIPT-BIOTECH-CORP-25502710/news/Genscript-Biotech-Stock-of-China-s-GenScript-Plummets-on-Report-Critical-of-Unit-s-Gene-Therapy-Te-27329783/

Theravance financial flexibility, assets underappreciated, says Piper Jaffray

Piper Jaffray analyst Tyler Van Buren says recent meetings with Theravance Biopharma management support his Overweight rating on the shares. The Trelegy royalty stream could potentially be monetized in the near-term as a source of non-dilutive financing, which would alleviate concerns regarding the need for cash, a significant portion of the current bear thesis, Van Buren tells investors in a research note. Further, he notes that management remains confident in a Yupelri approval on November 13. The analyst continues to recommend that investors own Theravance shares as he believes both its financial flexibility and current assets are underappreciated.
https://thefly.com/landingPageNews.php?id=2796195

Medicaid spent $581B in 2016; how it’s projected to grow over the next decade

Medicaid provided healthcare coverage to 72.2 million people in fiscal year 2016, an increase of 3.1% over 2015.

That increase corresponded with a 4.9% increase in spending over 2015, amounting to a total of $580.9 billion in spending for 2016, according to a new CMS report (PDF). The agency projected that over the following 10 years, expenditures would grow at an average annual rate of 5.7%, reaching over $1 trillion by 2026.

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In a statement, CMS Administrator Seema Verma said the projected growth in spending was “simply unsustainable” and that the government should be looking for ways to slow that growth.

However, Verma did note that due to reductions in projected long-term care spending, the average annual cost per enrollee will grow at a slower rate than previously expected.

Administrator Seema Verma

@SeemaCMS

Based on the projection of admin costs & slower projected growth in long-term care spending for @MedicaidGov, the avg. annual per enrollee cost growth is projected to be lower over the next 10 years. https://go.cms.gov/2pnXI0M 

5:03 PM – Sep 20, 2018

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• See Administrator Seema Verma’s other Tweets

“Compared to the prior report, total projected Medicaid expenditures for benefits and administrative costs are expected to be $104.1 billion less from 2016 through 2025, or 1.4 percent lower, reflecting slower growth in benefit expenditures (particularly for long-term care services),” the report said. “Annual per enrollee costs are projected to grow by 4.2 percent, or at a 0.1-percent lower rate, over the same period.”

The federal government has a good reason to be concerned about the growth of Medicaid spending. According to CMS projections, most of the added expenditures over the next decade will be paid by the federal government.

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That’s because many of the new Medicaid enrollees came in during the Affordable Care Act’s Medicaid expansion, an expansion that was heavily funded by the federal government. Some Republican health plans have proposed simply reducing the federal share of Medicaid expenditures, which would mean states would have to cover more of the expense growth themselves.

That would not, however, change the overall number of enrollees in the program. And according to CMS, most of the adults added by the expansion have already been accounted for.

“An estimated 12.2 million expansion adult enrollees were covered in 2017, based on enrollment counts included in 2017 financial data reported by the States to CMS,” the agency’s actuaries wrote. “By 2026, the expansion adult population is projected to grow to 13.3 million. These estimates are based on the assumption that 55 percent of potential expansion enrollees reside in States with expanded eligibility in 2017 and after.”

1.1 million people over 10 years isn’t a ground-shaking increase. And in fact, enrollment is set to taper off by 2026 (after reaching around 82 million people) while costs continue to surge.

https://cdn.iframe.ly/api/iframe?url=https%3A%2F%2Fpublic.tableau.com%2Fprofile%2Feli.richman%23!%2Fvizhome%2FMedicaiddatabyyear%2FDashboard1%3Fpublish%3Dyes&key=5a82ccc8caa9bb33ac6f2d668fcf304b&app=1

https://www.fiercehealthcare.com/payer/medicaid-spending-was-581b-2016-here-s-how-it-s-projected-to-grow-over-next-decade

HHS urging providers to use telemedicine for medication-assisted opioid treatment

HHS is pushing providers—particularly those in rural areas—to take advantage of telemedicine to increase access to medication-assisted addiction treatment (MAT).

But providers seeking to prescribe these medications require waivers, which can hinder access, especially in remote areas where doctors with these qualifications are in short supply.

So the Department of Health and Human Services and the Drug Enforcement Administration have worked together to ease these restrictions. Doctors can now prescribe buprenorphine, one of the most commonly used drugs for MAT, through a virtual platform, when before these clinicians had to be physically present with patients to offer these prescriptions.

“Not everyone is waivered, and as you might imagine, particularly in underserved areas there’s a profound shortage” of these clinicians, Brett Giroir, M.D., assistant secretary for health and senior adviser for opioid policy, said at a press briefing on Thursday.

As long as a clinician with a DEA number, which can include advanced practice providers who aren’t doctors, is present with the patient at the time of the consult, the waivered provider on the other end of the line can prescribe buprenorphine when deemed appropriate, Giroir said.

MAT, used in conjunction with other options such as psychological services, is a crucial way to address opioid addiction. Boosting access to MAT, particularly in rural regions hit hardest by the opioid epidemic, was one of a slew of recommendations issued late last year by the White House’s opioid commission.

The MAT waiver adjustments were made earlier this year, but HHS is now issuing a full-court press on alerting providers to their telemedicine options for opioid treatment, Giroir said.

HHS is taking steps to get the word out after a Substance Abuse and Mental Health Services Administration report showed progress in efforts to address the opioid crisis, such as fewer new heroin users and a decline in the number of people misusing opioids in 2017. More people got into treatment for opioid use disorder, as well, according to the report. However, more than 11 million American adults used opioids inappropriately last year, the report estimates.

Tackling the opioid crisis is a top priority for HHS and is one of Secretary Alex Azar’s central agenda items. At the briefing, Azar also noted that the agency had released $1 billion in grants for programs to address the epidemic.

“All of these actions are part of a well-coordinated strategy that we are bringing to bear here at HHS,” Azar said. “We’re tackling a problem that we have been dealing with for decades and did not get into overnight.”

Giroir said addressing the waiver problem is a clear example of why it’s crucial that the Trump administration takes a multiagency approach to combat the opioid epidemic. Effective solutions require not just HHS but also the Department of Justice, the Department of Housing and Urban Development and the Department of State, among others, he said.

“There is almost no single part of the solution that can be done in a silo,” Giroir said. “It must be done across the agency in a synergistic way.”

https://www.fiercehealthcare.com/tech/hhs-urging-providers-to-use-telemedicine-to-enhance-access-to-medication-assisted-opioid

New kidney care firms eye making money by keeping patients out of dialysis

San Francisco-based Cricket Health only has about a dozen employees.

But the tech-enabled kidney care provider has some big ambitions in the next year or so when it comes to disrupting the world of kidney care. Namely, they intend to show that their proprietary data analytics can identify high-risk patients to allow early intervention, improve outcomes and lower the costs of the care that chronic kidney disease often requires.

In a world of perverse payment incentives, that is far easier said than done, Arvind Rajan, Cricket’s CEO, told FierceHealthcare in an interview.

“It’s a completely broken system from end to end and that’s really what we set out to tackle,” Rajan said. “We provide care where the primary focus is slowing the progression of kidney failure.”

The company recently raised a $24 million series A funding round from a who’s who of investors including Cigna Corp., as well LinkedIn CEO Jeff Weiner, Joe Montana’s Liquid 2 Ventures and Rock Health co-founder Halle Tecco. They are not yet profitable, said Rajan, but they expect to become so in the next few years by saving payers money through better managing patients, keeping them out of the hospital and out of dialysis centers as long as possible.

Over the next 24 months, the company will begin ramping up its activities, scaling its workforce to about 100 people and deploying its first commercial programs for both payers and health systems around the country, he said.

If successful, they’ll be in good company. Earlier this year, CVS Health—which is still working to close a deal to acquire insurance giant Aetna—announced its plan to enter the chronic kidney care market. Its plan is to focus on patient education and early detection of kidney disease while working on developing and deploying a home-based hemodialysis device.

They’re also part of a wave of startups gaining investor attention for their focus on disrupting the massive kidney care market in light of shifts to value-based payments across healthcare. Among these companies are groups started by former executives of Denver-based kidney care giant DaVita.

Town Hall Ventures, the venture firm co-founded by a group of health industry veterans including former CMS Acting Director Andy Slavitt, just announced it invested in kidney care startup Strive Health. The company, which is led by former DaVita executives Bob Badal and Chris Riopelle and has gained investments from New Enterprise Associates, boasts that its kidney care service line, powered by specialized analytics, can cut inpatient utilization by 50% and triple rates of home dialysis adoption.

In April, Town Hall Ventures also announced it invested in Somatus, a Vienna, Virginia-based kidney care company led by another DaVita alum: CEO Ikenna Okezie. That company also specializes in offering value-based kidney care management services to health systems and payers.

“There is a big entrenched system, particularly among the two dominant players like DaVita where they have brick-and-mortar centers, they have dialysis chairs they need to fill and they’ve spent a lot of money on that infrastructure and system,” Trevor Price, general partner at Town Hall, told FierceHealthcare.

But that no longer makes sense, Price said.

“In today’s world, you can treat patients with chronic kidney disease in different settings that are frankly much better for them and lower cost for the system,” Price said. “You can provide forms of dialysis in the home. You don’t need brick-and-mortar settings and you don’t need to have all the big real estate infrastructure costs these companies have built up trying to dominate and control this massive industry. There is the opportunity to disrupt the system. And it benefits everyone.”

Rajan estimates Cricket Health is chasing a market worth well over $100 billion. An estimate published earlier this year by KBV Research projects that the global dialysis market is growing at an annual rate of 5.3% and will reach $124 billion by 2023. “Our main theses as a company is the way to truly change the cost curve of kidney disease is fundamentally be intervening earlier with kidney disease patients because you can keep patients from dialysis longer,” he said.

Of course, DaVita officials have said it has also evolved to address the changing healthcare landscape.

Kent Thiry, DaVita’s chairman and chief executive officer, told Bloomberg earlier this week that his company is “unusually suited to drive” change in the industry. The company is selling its primary health business while investing in new businesses. “Every patient is different,” Thiry said. “What we’re proud of is we give each patient very customized attention in the midst of a very disciplined process. We are the clinical leader in America both in terms of execution and in innovation.”

The announcement of Cricket’s fundraising comes just weeks after California’s General Assembly passed a controversial bill which would cap payments for dialysis providers with connections to charities that subsidize their patients’ commercial insurance—a measure lauded by insurers and employers but opposed by dialysis providers and the American Kidney Fund.

In November, voters will also consider a statewide ballot initiative known as Proposal 8, which would cap the revenue dialysis providers could make from patients with commercial insurance.

Rajan said the legislation reflects the frustration of payers over the cost of in-center dialysis care, which can reach well north of $100,000 a year and is not the best treatment option for most patients.

“I think this reflects the tipping point of that frustration which has driven the legislation around the country,” he said. “What we’re excited about is it’s the first step in payers reassessing the fundamental understanding of how to approach kidney care. Even the conversations we’ve had with payers in the last six months compared to when we started three years ago is fundamentally different.”

https://www.fiercehealthcare.com/hospitals-health-systems/more-companies-invest-kidney-care-space

Geron to Update on Imetelstat Collaboration with Janssen

Geron Corporation (Nasdaq: GERN) today announced that it will host a conference call to provide an update on the imetelstat collaboration with Janssen Biotech, Inc. (Janssen) on Thursday, September 27th at 8:00 a.m. ET.

Participants may access the conference call live via telephone by dialing domestically +1 (877) 303-9139 or internationally +1 (760) 536-5195. The passcode is 7987354. Participants are advised to dial in at least 10 minutes prior to minimize any delay in joining the call. A live, listen-only webcast will also be available on the Company’s website at www.geron.com/investors/events. If you are unable to listen to the live call, an archived webcast will be available on the Company’s website for 30 days.

https://www.marketscreener.com/GERON-CORPORATION-9419/news/Geron-to-Provide-an-Update-on-the-Imetelstat-Collaboration-with-Janssen-on-September-27-2018-27325624/