US Senator seeks “unannounced” FDA inspections; Nektar reports manufacturing issues with lead project

US Senator seeks “unannounced” FDA inspections; Nektar reports manufacturing issues with lead project

By PharmaCompass

2019-08-15Impressions: 2570

US Senator seeks “unannounced” FDA inspections; Nektar reports manufacturing issues with lead project

This week in Phispers, we bring you news about Nektar Therapeutics which disclosed declining efficacy of its lead project due to manufacturing problems.

In Congo, two experimental drugs showed survival rates of nearly 90 percent in patients suffering from the deadly Ebola fever.

US Senator Chuck Grassley has sought unannounced FDA inspections of drug manufacturing facilities overseas.

Bayer announced it would fully acquire biotechnology firm BlueRock, while CDMO Cambrex was bought out by private equity firm Permira Funds.

Amgen won a key Enbrel patent case against Sandoz’s biosimilar.

Two AstraZeneca cancer drugs — Tagrisso and Lynparza — posted promising results for new indications.

And Canada announced final regulations to reduce price of patented drugs — the biggest reform to its drug price regime since 1987.


US Senator seeks “unannounced” FDA inspections of plants overseas

In a letter sent to Alex Azar, secretary, Department of Health and Human Services (HHS), and Norman Sharpless, acting commissioner, US Food and Drug Administration (FDA), Chuck Grassley, chairman of the US Senate Committee on Finance, highlighted that the committee has an obligation to ensure that FDA upholds its responsibility to protect public health by properly overseeing the nation’s drug supply and ensuring that the drugs Americans use are safe and effective.

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Referencing the valsartan drug safety issues that made headlines in the past year and an interview with ex-FDA investigator Motammed Masoud who “had tried to sound the alarm on what he flagged as potential systemic problems at two facilities in China and India that produce the API in generic valsartan and other blood pressure medications”, the letter also highlights that the “FDA only inspected one in five registered human drug manufacturing facilities abroad last year.”

As the Trump administration is pushing for drug imports from overseas in an effort to lower drug prices in the US, the letter states “the FDA does not track in its databases whether a foreign inspection was subject to an announced or unannounced visit.” Further, Grassley said he has learned that the FDA generally does not perform unannounced visits of drug manufacturing facilities in foreign countries but does perform unannounced visits at facilities based in the US.

The letter goes on to mention that in 2013 the FDA created a pilot program in India that eliminated advanced notice and instead used short notice or unannounced visits. In 2015, the pilot program was shut down without explanation. It is unclear why the Obama administration shut the pilot program.

However, because of its reported successes, Grassley said he would strongly encourage the administration’s demonstration projects to include unannounced inspections in foreign manufacturing facilities to determine “if they meet the required API and drug quality and safety standards to include sufficient record-keeping, testing, and protections against counterfeiting”.

Meanwhile, following data accuracy issues with Novartis’ recently approved gene therapy Zolgensma, Grassley has asked Novartis to provide details on data manipulation related to its US$ 2 million gene therapy.

Grassley wants Novartis to submit additional details, including the date when it came to know that it issued manipulated data to the FDA as well as the number of employees whose services were terminated due to this issue. Novartis said it has received the Senator’s letter and was reviewing the request.


After Novartis, Nektar reports manufacturing issues with lead project

Last week, we had carried an article on the data accuracy issues with Novartis’ recently approved gene therapy Zolgensma. This week too, compliance continues to dominate news headlines.

Shares of Nektar Therapeutics plummeted after the company disclosed declining efficacy of its lead project — NKTR-214 (bempegaldesleukin). During an analyst call, Nektar Therapeutics said new assays had revealed two of its earliest production batches differed from others. And these two batches correlated with the study subjects who yielded relatively low response rates to NKTR-214.

The study subjects had received “suboptimal” product batches. The “single suboptimal batch of in-process intermediate” led to two lots of 22 being ineffectively produced.

In February 2018, Bristol-Myers Squibb and Nektar Therapeutics had announced a global development and commercialization collaboration for Nektar’s NKTR-214. Bristol-Myers Squibb had inked a US$ 1.85 billion partnership with Nektar to evaluate the full-potential of NKTR-214 given along with BMS’ flagship immune-oncology treatment Opdivo (nivolumab) across numerous tumors.

The broad joint clinical development plan combining NKTR-214 with Opdivo and Opdivo plus Yervoy (ipilimumab) targeted registration-enabling trials in more than 20 indications across nine tumors.

During the analyst call, Nektar executives also announced that Bristol-Myers had cut back its commitment to the bempegaldesleukin partnership to just five or six trials aimed at regulatory approval.

Another company which hit headlines was Sarepta Therapeutics. An adverse event report was submitted to the FDA regarding an ongoing study of the company’s gene therapy for Duchenne muscular dystrophy (DMD). When the news broke, shares of the company plunged by as much as 19 percent.

A notice from the FDA showed a patient in the study reportedly developing a serious illness. However Sarepta’s own investigation indicated the report was not submitted by its employee or the study’s principal investigator. The FDA told Reuters that it was looking into the matter.


Experimental drugs show 90 percent survival rates for Ebola patients

The deadly Ebola hemorrhagic fever is not incurable anymore. Scientists are a step closer to being able to cure the deadly Ebola fever after two experimental drugs showed survival rates of nearly 90 percent in a clinical trial in the Democratic Republic of Congo (DRC).

Two experimental drugs — REGN-EB3 developed by Regeneron and a monoclonal antibody called mAb114 — will now be offered to all patients infected with the viral disease. Ebola has been spreading in eastern Congo since August 2018, with the outbreak killing at least 1,800 people.

The drugs showed “clearly better” results, according to the US National Institute of Allergy and Infectious Diseases (NIAID), in a trial of four potential treatments being conducted during the second-largest Ebola outbreak in history, now entering its second year in DRC.

The drugs improved survival rates from the disease more than the two other treatments being tested — ZMapp (manufactured by Mapp Biopharmaceutical) and Remdesivir (made by Gilead Sciences). The agency said 49 percent of the patients on ZMapp and 53 percent on Remdesivir died in the study. In comparison, 29 percent of the patients on REGN-EB3 and 34 percent on mAb114 died. ZMapp and Remdevisir will now be dropped.

Jean-Jacques Muyembe, director general of Congo’s Institut National de Recherche Biomédicale in DRC, who co-led the trial, said: “From now on, we will no longer say that Ebola is incurable…these advances will help save thousands of lives.”


Bayer to fully acquire BlueRock; CDMO Cambrex maybe bought out; Perrigo split put on hold

In M&A news, Bayer AG and BlueRock Therapeutics announced an agreement wherein Bayer will fully acquire BlueRock Therapeutics.

BlueRock is a privately held, US-headquartered biotechnology company focused on developing engineered cell therapies in the fields of neurology, cardiology and immunology.

Following a 2016 joint venture with Versant Ventures to establish BlueRock Therapeutics, Bayer will acquire the remaining stake for approximately US$ 240 million in cash. With Bayer currently holding 40.8 percent stake, the investment corresponds to a total company value of US$ 1 billion estimated for BlueRock Therapeutics. The closing of the transaction is expected during the third quarter of 2019.

Meanwhile, there are reports that Elanco, an Eli Lilly spin off, maybe interested in buying Bayer’s animal health business. A year ago, there were rumors that Bayer might be planning to exit the animal-health business. According to news reports, Elanco and Bayer could announce an agreement to merge as soon as next week.

Cambrex now a target for buyout: Contract developer and manufacturer (CDMO) Cambrex, which is known for its aggressive acquisitions, is now the target of a buyout bid. The bid is to the tune of US$ 2 billion.

The firm is being bought out by private equity firm Permira Funds. If shareholders and regulators give their nod, the transaction is expected to close in the fourth quarter.

Cambrex, which makes small-molecule APIs, has built itself into a top-tier CDMO after two major acquisitions last year.

Perrigo postpones spinoff of its prescription business: Last year, Perrigo Company Plc had announced its board has approved a plan to separate its prescription pharmaceuticals business following the company’s previously announced strategic portfolio review.

A year on, there is news that Perrigo will postpone the spinoff of its prescription generics business after a steady sales quarter and the fizzle of its consumer health business.

The plan, which Perrigo once said could lead to a sale or merger, was originally expected to go into action by the end of 2019 or early 2020, but that deadline is now indefinite, Perrigo said.

In a second-quarter earnings call with investors last week, president and CEO Murray Kessler said Perrigo remained committed to a sale or spinoff but felt less pressure now, since the drugmaker’s prescription drugs were showing promising growth.


Amgen wins key Enbrel patent case against Sandoz’s biosimilar

Last week, a US judge upheld two patents related to Amgen Inc’s blockbuster rheumatoid arthritis drug — Enbreldenying a challenge posed by Novartis AG, which is seeking to launch its biosimilar version through its Sandoz unit.

The judge rejected arguments by Novartis’ Sandoz unit that the patents covering Enbrel’s API until 2029 should not have been granted because their concepts were already contained in previous patents.

Enbrel has been on the US market since 1998. It was developed by Immunex, which Amgen acquired in 2002.

“This is a key overhang removed,” Jefferies analyst Michael Yee said in a research note. “While an (expedited) appeal is in place and expected, we believe the worst-case fear is now gone.”

Novartis said it would appeal the ruling to the US Court of Appeals for the Federal Circuit.

“Sandoz respectfully disagrees with the Court’s ruling, which prevents us from launching an additional treatment option for patients with autoimmune and inflammatory diseases,” said Carol Lynch, president of Sandoz US and head of North America.


Two AstraZeneca cancer drugs — Tagrisso and Lynparza — post promising results

British drugmaker AstraZeneca Plc had positive news to share about two of its drugs — Tagrisso (osimertinib) and Lynparza (Olaparib). While Tagrisso is currently approved in 74 countries, including the US, Japan and the EU, for first-line epidermal growth factor receptor (EGFR)-mutated metastatic non-small cell lung cancer, Lynparza (which is being jointly developed with Merck) is already approved for advanced ovarian cancer and metastatic breast cancer.

AstraZeneca said Lynparza was successful in helping patients with metastatic prostate cancer and certain genetic mutations live longer without the disease worsening.

EGFR is a transmembrane protein, and more than 60 percent of non-small cell lung carcinomas (NSCLCs) express EGFR. A new late-stage study showed that Tagrisso had significantly helped patients with this type of lung cancer live longer without the disease worsening.

“Tagrisso was the only medicine that had shown statistically significant overall survival benefit in this type of disease setting,” AstraZeneca said.

On Lynparza, the company said the treatment met the main goal in a late-stage study of patients with metastatic castration-resistant prostate cancer and BRCA1/2 or ATM gene mutations. Patients were previously treated with hormonal anticancer therapies.

BRCA and ATM genes are responsible for producing proteins that repair damaged DNA, but can cause cancer growth if the genes mutate.

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“This will be a big extension to Lynparza’s market potential and will be seen as good news. After good phase II data this trial was expected to work,” Liberum analysts said in a note.

Prostate cancer is the second-most common cancer in men, with an estimated 1.3 million new cases diagnosed worldwide in 2018, AstraZeneca said.

The company has moved deeper into cancer therapy through wide-ranging deals, including those for immunotherapy and targeted therapy. Sales from AstraZeneca’s oncology unit had soared 57 percent to US$ 2.17 billion in the second quarter, accounting for 38 percent of total product sales, with revenue from Tagrisso nearly doubling to US$ 784 million.


Canada unveils biggest reform to drug price regime since 1987

Last week, the Canadian government announced final regulations that should save nearly US$ 10 billion for Canadians over a decade in terms of their spend on patented drugs.

Drug companies have been opposing this move and may eventually challenge the new rules in court. This is the biggest reform to Canada’s drug price regime since 1987. The rules will save money for patients, employers and insurers, including the government, at the expense of the profits of drug companies.

They could also eventually cut the earnings of drugmakers in the US, the world’s largest pharmaceutical market. The new rules were largely in line with a December 2017 draft.

“We are taking the biggest step in a generation to lower the price of drugs in Canada by moving forward with these regulations,” minister of health Ginette Petitpas Taylor said.

Petitpas Taylor said the rules would lay the foundation for a new national drug program. Prime Minister Justin Trudeau’s government is expected to announce a program to cover the cost of prescription drugs for some or all Canadians, but the program’s scope is not yet clear.

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