Portola Gets FDA Orphan Drug Tag for Peripheral T-Cell Lymphoma Treatment

Portola Pharmaceuticals (Nasdaq: PTLA) today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to cerdulatinib, an investigational, oral Syk/JAK inhibitor for the treatment of peripheral T-cell lymphoma (PTCL).

“We are pleased that the FDA has granted cerdulatinib Orphan Drug Designation, as it recognizes its potential to provide a significant clinical benefit to a group of patients with limited treatment options,” said John Curnutte, M.D., Ph.D., Portola’s interim co-president and head of research and development. “We look forward to presenting additional data from the Phase 2a trial at a scientific congress early next year and to continuing discussions with the FDA regarding next steps for the development of cerdulatinib, including the potential for an accelerated approval pathway.”

The FDA’s Office of Orphan Products Development grants orphan status to support development of medicines for the treatment of rare diseases that affect fewer than 200,000 people in the United States. Orphan Drug Designation may provide certain benefits, including a seven-year period of market exclusivity if the drug is approved, tax credits for qualified clinical trials and an exemption from FDA market application fees.

https://www.marketscreener.com/PORTOLA-PHARMACEUTICALS-I-13255910/news/Portola-Pharmaceuticals-Receives-FDA-Orphan-Drug-Designation-for-Cerdulatinib-an-Oral-Syk-JAK-Inhib-27316889/

UK’s BMI Healthcare reported close to £2 billion restructuring deal

Britain’s largest private healthcare group, BMI Healthcare, is nearing a 2 billion pounds restructuring deal that could cut millions of pounds from its annual rent bill, a person familiar with the matter said on Tuesday.

An announcement under which BMI’s rent bill will be cut by about 60 million pounds is likely to be made imminently, the source told Reuters.

Funds including Centerbridge and Och-Ziff Capital Management will inject about 50 million pounds of equity into the business, and maturity of about 2 billion of debt on the balance sheet of BMI and its property arm General Healthcare Group will be extended by six years, the source said.

Sky News had earlier reported about the deal on Tuesday.

Last year, South African private healthcare operator Netcare had made an all-share offer to buy out minority BMI Healthcare shareholders saying it was making the move because trading conditions “remained difficult” across the private healthcare market.

In March, Netcare drew a line under its ambitions in Britain saying it would exit the market due to difficult trading conditions.

Och-Ziff Capital Management and Centerbridge were not immediately available for comment.

BMI Healthcare operates 59 hospitals and clinics throughout the United Kingdom and had about 276,000 inpatient visits per year, according to its website.

https://www.marketscreener.com/NETCARE-LIMITED-1413397/news/Netcare-BMI-Healthcare-close-to-2-billion-restructuring-deal-source-27317048/

Global Settlement Resolves DOJ Probe Involving Community Health

Community Health Systems, Inc. CYH, -2.29% (“the Company”) announced today that it has reached a global resolution and settlement agreements ending the U.S. Department of Justice investigation into conduct by Health Management Associates, Inc. (“HMA”) and its affiliated entities reflected in qui tam lawsuits that were initiated and pending, and known to the Company, before HMA was acquired by merger in January 2014.

The settlement concludes the government’s investigations into whether HMA and its affiliated hospitals billed Medicare, Medicaid and TRICARE for certain inpatient admissions following emergency room visits between January 2008 and December 2012 that should have been billed as outpatient or observation cases. The settlement also resolves allegations of Stark and Anti-Kickback Act violations at certain HMA affiliated hospitals.

In reaching the settlement, the government noted that all conduct reviewed in its investigation pre-dated the HMA acquisition, and that following the acquisition, Community Health Systems engaged in remedial measures, including removing the HMA Board of Directors and senior executives and integrating the HMA affiliated hospitals into Community Health System’s existing compliance program.

The settlement includes a Non-Prosecution Agreement with HMA in which the government agreed not to bring criminal charges against HMA, as long as HMA and the Company abide by the provisions of the global settlement, and also includes a guilty plea to one count of conspiracy to commit healthcare fraud by Carlisle HMA, LLC (“Carlisle HMA”), the HMA affiliated entity that formerly operated Carlisle Regional Medical Center.

The global settlement includes a total payment of $262 million, which is expected to be paid in October 2018. As part of the accounting for the HMA acquisition by merger, the Company established a liability related to the legal claims underlying the contingent value right (“CVR”) that was issued to HMA shareholders as part of the merger consideration. This liability represented the Company’s best estimate of fair value of the potential future payments associated with the legal matters assumed in the HMA merger. This estimate has accordingly been updated over time as additional facts and circumstances have become known. Based on the settlement terms and calculation of any payment as defined in the CVR agreement, the Company anticipates that no distribution will be owed to CVR holders.

Under the terms of the global settlement, the Company’s existing corporate integrity agreement (CIA) has been amended and extended. The extension begins immediately and effectively adds two years to the existing CIA, with the amended CIA now running through 2021.

Commenting on the resolution of the government’s investigation into HMA, Wayne Smith, chairman and chief executive officer of Community Health Systems, Inc., said, “Since acquiring HMA in 2014, it has been our goal to resolve the government’s investigation into all of these allegations which occurred prior to the acquisition and which were already under investigation at the time of the transaction. We are pleased to have reached the settlement agreements so we can move forward now without the burden or distraction of ongoing litigation. As an organization, we are committed to doing our very best to always comply with the law in what is a very complex regulatory environment and to operate our business with integrity, ethical practices and high standards of conduct.”

https://www.marketwatch.com/press-release/global-settlement-resolves-us-department-of-justice-investigation-into-health-management-associates-inc-and-related-affiliates-2018-09-25

AnaptysBio’s lead IL-33 antibody clears midstage test in asthma

Next-generation antibody company AnaptysBio has positive data for its lead pipeline drug etokimab in a third indication, adding asthma to its earlier wins in peanut allergy and atopic dermatitis.

The phase 2a trial in severe eosinophilic asthma gives the San Diego company another proof-of-concept study for the interleukin-33 (IL-33) inhibitor, setting up a third phase 2b program that it sayswill start early next year. Shares in the company were down marginally after the announcement.

The study was small—involving just 25 patients who received a single intravenous dose of etokimab (ANB020)—but according to the company showed that the anti-IL-33 drug had a rapid impact on lung function that lasted for around two months.

Using the forced exhalation in one second (FEV1) measure, the trial showed an 8% improvement for the antibody compared to placebo the second day after dosing, and this rose to 11% by day 64, although the difference fell back to 4% and 6% at timepoints in between.

Blood biomarkers supported the proposed mechanism of the drug, with eosinophil levels down 31% at day two and 46% at day 64 versus control, though once again with dips in the interim. All the patients were also taking inhaled steroids and beta agonist bronchodilators.

The trial’s principal investigator—University of Oxford respiratory specialist Professor Ian Pavord—said the data shows “the potential for IL-33 inhibition in treating severe eosinophilic asthma” but will need to be investigated further in additional studies.

Earlier this year, AnaptysBio reported proof-of-concept data with etokimab in peanut allergy, once again with some questions about study in terms of its size and the San Diego biotech’s decision to exclude a couple of patients from the analysis, which improved the results.

In 2017, it reported data in atopic dermatitis which CEO Hamza Suria said were “a solid foundation for the continued development of [the drug] across a number of atopic diseases”—and helped the company complete a $195 million public offering to help fund its phase 2 programs, which will use a new subcutaneous formulation of the drug.

The company has two phase 2b trials on the go—ATLAS in atopic dermatitis and ECLIPSE in chronic rhinosinusitis with nasal polyps—that are due to read out in the second half of 2019, said Suria on a conference call.

With three separate studies now showing an impact on inflammatory diseases, the biotech is becoming increasingly confident that IL-33 is a target worth pursuing. Etokimab is pitching at increasingly competitive indications with atopic dermatitis and severe asthma however, as both diseases have new biologic therapies in the rollout phase.

“We are thrilled to have demonstrated proof-of-concept in this single dose Phase 2a trial and look forward to advancing the development of etokimab for patients suffering from eosinophilic asthma,” commented Suria, who says the biotech is out in front in the anti-IL-33 category.

“Genotypic studies have validated the key role played by IL-33 in asthma, and we believe etokimab’s upstream mechanism has the potential for a broad therapeutic benefit across multiple atopic disorders.”

https://www.fiercebiotech.com/biotech/anaptysbio-s-lead-il-33-antibody-clears-mid-stage-test-asthma

Tariffs now touch nearly every U.S. medical device sold in China

The trade war between the U.S. and China has grown in both size and scope—with Chinese retaliatory tariffs now reaching nearly every U.S. medical device exported to the country—while uncertainty over the years ahead has companies looking to prepare against larger impacts.

At midnight EST on Monday, Sept. 24, or noon in Beijing, the Trump administration imposed 10% tariffs on $200 billion worth of imports. At the same time, China returned with 5% to 10% tariffs on $60 billion of U.S. goods, including $3.5 billion in medtech-related trade.

Added to tariffs levied this past summer, the latest round totals nearly all U.S. medtech exports to China in 2017, or about $4.75 billion, according to AdvaMed, the industry’s trade association.

“Depending on where you are in the industry, the tariff situation and related activities will impact you differently,” AdvaMed CEO Scott Whitaker told reporters during a press event at the trade group’s annual conference. For example, companies in the imaging space may feel a more direct impact because of reliance on component parts, he said.

In addition, because of the disparity in the total dollar amounts between the two countries, China also threatened to impose extra, unspecified, non-tariff barriers with more qualitative effects—such as slow-walked regulatory approvals or possible investigations into various companies’ operations within the country.

Meanwhile, the latest $200 billion in tariffs on imports from China are slated to increase from 10% to 25% on Jan. 1, after the holiday shopping season. President Donald Trump has also called for an additional $267 billion in tariffs on top of that, totaling essentially all of China’s exports to the U.S., depending on the country’s actions going forward.

“As the number as gotten bigger, and as the back and forth between China and the U.S. has grown, we’re starting to see it impact us a little bit more,” Whitaker said. “When you get to $200 billion, and when you get to $267 billion, then you’re really starting to affect all aspects of the economy.”

AdvaMed is currently working to assess the tariffs’ impact by company and product category, and is aiming to release a report in the coming months, Whitaker said. China’s retaliatory tariffs first hit the industry directly in late August, with a matching $16 billion riposte against U.S. exports that included $1.25 billion against medtech products.

At the association’s conference in Philadelphia, Claire Reade, former assistant U.S. trade representative for China affairs, detailed companies’ options for navigating the new U.S. tariffs, such as applying for exclusionary waivers for specific products.

Companies would have to prove no comparable product exists outside of China, that Chinese supply has been critical to the company’s efforts over the past several years and that losing that access would cause severe harm.

In addition, the product has to be physically distinct from other products under the same tariff category—trademarks or logos are not enough, said Reade, who also served as the Office of the U.S. Trade Representative’s chief counsel for China trade enforcement. She is now a senior counsel at the law firm Arnold & Porter.

Aside from that, three options include absorbing the tariffs, such as by building up the company’s stock of products; adjusting procurement to other locations, including non-Chinese plants from a Chinese supplier or from other parts of the world; or by changing the company’s manufacturing footprint through shifts to contract manufacturing or setting up new plants, said Rene Buck, CEO of Buck Consultants International.

But what should companies not do? “There are a bunch of Chinese companies today on the internet that will offer to bring your products to Malaysia, label them as ‘Made in Malaysia’ and then send them over,” Buck warned of the illegal bypass. “You could imagine cases where that might be appealing, but probably we both agree it’s not what you should do, right?”

Reade added: “Criminal customs fraud. Prison. Major fines. Okay? Okay.”

https://www.fiercebiotech.com/medtech/trade-war-tariffs-now-touch-nearly-every-u-s-medical-device-sold-china

Thousands of deaths from infected NHS blood probed

A public inquiry has opened to look into how infected NHS blood killed more than 2,900 people since the 1970s.

The Infected Blood Inquiry, headed up by Sir Brian Langstaff, a former high court judge, was announced in July last year following years of campaigning by those affected, their families, and organisations.

It began today with a tribute to the many individuals who were given transfusions or treatments with blood infected with the HIV virus and hepatitis C.

The inquiry into the scandal, which was dubbed ‘the worst treatment disaster in NHS history’, will address how the blood was obtained and came to be used for people with haemophilia and other bleeding disorders.

Some patients had transfusions of the infected blood plasma whereas others were injected with blood-clotting protein Factor VIII, which haemophiliacs do not produce naturally.

Much of the human blood plasma used to make the product came from donors such as prison inmates, sex workers and drug addicts based in the US, who sold their blood. This was to meet a shortfall in the amount of blood available in the UK.

Blood products were not routinely heat-treated until the mid-1980s to kill any viruses. Screening of blood products began in 1991 and by the late 1990s, synthetic treatments for haemophilia were available, removing the infection risk.

Many sufferers remained quiet about the illnesses contracted through the infected blood products because of the historic stigma attached to HIV and hepatitis C.

The first evidence will be heard after Easter next year, with the process expected to last between two and five years. The inquiry will consider statements from hundreds of witnesses and experts.

Sir Brian Langstaff said: “The inquiry has already received over 100,000 documents and expects to acquire several times that number. There will also be many hundreds of witness statements. I am grateful for each and every contribution.

“There must, however, still be more who have knowledge, documents and their own accounts to add. I know that going over the past can be difficult, but I encourage them to come forward.”

Previous inquiries failed to address the issue adequately and the government has been accused of covering up the scandal, with former health secretary Andy Burnham reportedly saying in the House of Commons last year that there had been a “criminal cover-up on an industrial scale”.

https://pharmaphorum.com/news/public-inquiry-investigates-thousands-of-deaths-from-infected-blood-on-nhs/

Patients call for end to UK drug price row over cystic fibrosis med

Representatives of NHS England and cystic fibrosis drug company Vertex are to meet on Thursday to try and resolve a two-year impasse over access to life-changing medications.

Charities and patients called for a resolution to the ongoing pricing row, which began two years ago when NICE said Vertex’s combination therapy Orkambi (lumacaftor+ivacaftor) is too expensive for the NHS.

Orkambi is the first medicine to treat the underlying cause of cystic fibrosis in people with two copies of the F508del mutation, aged six or over, and Vertex has other drugs in its pipeline that will mean a wider group of patients will get treated.

NHS England and Vertex have been locked in an increasingly bitter argument over pricing, with the manufacturer accusing the NHS of undervaluing cystic fibrosis patients.

Vertex is trying to get the NHS to fund all its approved CF drugs, and any future medications in its pipeline in a long-term deal.

In July Vertex said it received an offer worth about £500 million over five years, and more than £1 billion over 10 years for Orkambi.

This was rejected and Vertex has made veiled threats that it will consider spending its R&D budget elsewhere because of the row.

It has also refused to engage with NICE until the cost-effectiveness body changes its assessment methods.

David Ramsden, chief executive of the Cystic Fibrosis Trust has written to Vertex’s chief executive Jeffrey Leiden, and NHS England’s chief executive Simon Stevens calling for the matter to be resolved.

Ramsden urged Leiden to do “everything in [his] power” to reach an agreement, while calling on Stevens to find a way to “value and reward” the innovative drugs from Vertex.

The UK has the second largest population of patients with CF in the world, with around 10,000 people affected.

Christina Walker with Luis

Christina Walker, from the patient group UKneedsorkambi, said the drug should be made available as soon as possible so that patients such as her son Luis can receive it.

Walker said: “It’s been a devastating summer for the campaign group while this impasse has persisted. We’ve watched our loved ones’ health decline with exacerbations, made many hospital visits and have mourned CF angels who’ve lost their final battle.

“Whether or not this situation continues unchecked is in the hands of the people from Vertex and the NHS around the table on Thursday.

“They must both compromise heavily – more than they want to – because lives are at stake and what’s the alternative? Too many people have died already. These transformational treatments can reduce the considerable suffering of this cruel condition, and patients must have them now. Time is up, and any further delay will be unforgivable.”

https://pharmaphorum.com/news/patients-call-for-cf-drug-price-agreement-ahead-of-meeting/