Amgen-Astellas BLINCYTO OKd In Japan For Acute Lymphoblastic Leukemia

First Approved Oncology Treatment From Amgen Astellas Joint Venture
BLINCYTO is the First-and-Only Approved CD19-Directed CD3 Bispecific T Cell Engager (BiTE®) Immunotherapy

Amgen (NASDAQ:AMGN) today announced that the Japanese Ministry of Health, Labour and Welfare has granted marketing approval for BLINCYTO^® (blinatumomab) for the treatment of relapsed or refractory B-cell acute lymphoblastic leukemia (ALL). BLINCYTO was developed in Japan by Amgen Astellas BioPharma K.K. (AABP), a joint venture between Amgen and Astellas Pharma Inc., a pharmaceutical company headquartered in Tokyo.

“As proof-of-concept for our bispecific T cell engager technology, BLINCYTO has laid the groundwork for Amgen to deliver on our passion of addressing cancer by exploring numerous biologic pathways and therapeutic modalities,” said David M. Reese, M.D., executive vice president of Research and Development at Amgen. “This innovation is a good example of how we provide new options to patients with serious illnesses like cancer. In bringing BLINCYTO to Japanese patients, we reinforce our commitment to deliver novel cancer therapies on behalf of patients worldwide.”

BLINCYTO is the first-and-only bispecific T cell engager (BiTE^®) immunotherapy construct approved globally. It is also the first approved immunotherapy from Amgen’s BiTE^® platform, an innovative approach that helps the body’s immune system target cancer cells.

“Today’s approval of BLINCYTO marks a significant milestone that reinforces our commitment to addressing unmet medical needs of patients in Japan,” said Steve Sugino, president and representative director, AABP. “As our first oncology treatment approved in the region, we are proud to provide a much-needed innovative treatment option for adults and children with relapsed or refractory B-cell ALL, one of the most aggressive B-cell malignancies.”

Hitoshi Kiyoi, M.D., Ph.D., professor of internal medicine, Hematology and Oncology, Nagoya University Graduate School of Medicine said, “The standard therapy for relapsed or refractory B-cell ALL has not been established in Japan and therefore different chemotherapy regimens have been selected, depending on the condition and background of each patient. BLINCYTO is a much-needed and important new treatment option for patients with relapsed or refractory B-cell ALL, as demonstrated by the efficacy and survival benefit seen in the TOWER study.”

The approval is based on data from multiple global studies, including the Phase 3 TOWER study and Japan Phase 1b/2 Horai study. In the TOWER study, BLINCYTO demonstrated a superior improvement in median overall survival (OS) versus standard of care (SOC) chemotherapy. Median OS was 7.7 months (95 percent CI: 5.6, 9.6) for BLINCYTO versus 4.0 months (95 percent CI: 2.9, 5.3) for SOC (HR for death=0.71; p=0.012). Safety results among subjects who received BLINCYTO were comparable to those seen in the previous Phase 2 studies of BLINCYTO in adult patients with Philadelphia chromosome-negative (Ph-) relapsed or refractory B-cell precursor ALL. In the TOWER study, major adverse reactions were pyrexia (39.0 percent), decrease in white blood cell count (14.6 percent), cytokine release syndrome (13.5 percent), febrile neutropenia (10.9 percent), headache (10.1 percent), elevated liver enzyme (10.1 percent) and decrease in platelet count (10.1 percent). In the Phase 1b/2 Horai study, BLINCYTO was administered to 35 Japanese adult and pediatric patients with relapsed or refractory B-cell precursor ALL. The safety results from the Horai study were comparable to those seen in the global studies, including TOWER. In the Horai study, major adverse reactions in adult patients were cytokine release syndrome (46.2 percent), pyrexia (46.2 percent), decrease in white blood cell count (38.5 percent) and decrease in platelet count (34.6 percent), and major adverse reactions in pediatric patients were elevated liver enzyme (66.7 percent), pyrexia (66.7 percent), cytokine release syndrome (55.6 percent) and abdominal pain (44.4 percent).

BLINCYTO is now approved in 57 countries, including the United States (U.S.), all member countries in the European Union (EU) and the European Economic Area, Canada and Australia.

https://www.biospace.com/article/releases/blincyto-blinatumomab-approved-in-japan-for-the-treatment-of-relapsed-or-refractory-b-cell-acute-lymphoblastic-leukemia/

Pharma Firms React to Employers, Plans Excluding New Therapies Based on Cost

The high cost of prescription medications has been a common subject of discussion in the news as of late. Many studies have been done regarding this, including one that shows that, on average, the US pays more than 10 other wealthy nations combined for medication.

There have been a lot of ideas proposed to help combat these costs, but few can agree on what plan would work best. Recently, however, a few companies have decided to enact their own plans that they believe will help bring down the cost of new therapies.

Insurance companies’ New Programs

CVS Caremark has launched a new program that will allow its clients to exclude any medication that launches at a price greater than $100,000 per quality-adjusted life-year (QALY), which is a benchmark created by the Institute for Clinical and Economic Review (ICER). This is intended to force pharmaceutical companies to drop the price of their medications by denying patients from purchasing them if they cost more than the threshold CVS has set.

But CVS isn’t the only one attempting to implement new policies to try and bring drug prices down. New York state’s Medicaid program has decided to exclude a new cystic fibrosis medication, citing that it costs too much for how effective it is. The drug in question, Orkambi, costs $272,000 a year but hasn’t shown to be very effective at helping patients.

Other companies like Express Scripts are following suit as well, using their own exclusionary plans that they hope will force pharma companies to give them better prices.

Whether or not these new programs will have the desired effect remains to be seen and with all of these health plans enacting these new programs, pharmaceutical companies have begun asserting their positions on the topic.

Reactions from Pharma Companies

Not long after CVS and the state of New York made their announcements for their new programs, Dr. Robert W. Dubois, the chief science officer and executive vice president of the National Pharmaceutical Council, spoke out against them.

Much of Dr. Dubois’s argument against these kinds of programs hinges on the idea that patients won’t be able to access the medication that they require. He also believes that this plan focuses too much on cost-effectiveness when deciding the value of a new therapy.

According to Dubois, to fully determine the correct value of a medication, many other considerations need to be taken into account. A few of the examples he gave include the medication’s ability to treat illnesses that were previously insufficiently treated, the possibility of a cure for the illness and the hope that it creates, and the ease of the regimen compared to other treatments.

Dubois also criticizes CVS’s implementation of the plan, noting that ICER’s framework has a variable economic threshold while CVS will be using a single threshold set at $100,000. He believes that such a dichotomous decision is a mistake and will end up severely limiting the kinds of new therapies patients have access to as, under CVS’s plan, a medication that is even slightly above their set threshold will be denied.

After Dr. Dubois made his statement against CVS and New York State’s new plans, CVS wrote their own article to fire back at him and the pharmaceutical companies he speaks for. They start off by pointing out that Dubois represents various pharmaceutical companies and, thus, his words should be seen in that light. They follow that up by defending their usage of ICER’s benchmark and how they’ve implemented their new plan.

If one is to take Dr. Dubois’s words as representative of the pharmaceutical industry as a whole, then it would seem as though pharma companies are very much against these new plans that companies like CVS are implementing.

However, not all pharma companies are completely against adjusting the cost of medications according to ICER’s benchmarks. Both Sanofi and Regeneron have announced that they will make their cholesterol medication more affordable to be in line with ICER’s analysis. But it remains to be seen if they will follow this plan for more medications later on.

In Conclusion

It would appear that pharmaceutical companies are still divided on the best method to go about lowering the cost of medications for patients. Insurance companies and other healthcare programs are also uncertain about the best way to force pharma companies to lower their prices, with each one attempting to enact their own plans to solve this issue.

The one thing that they all seem to agree on, however, is that the costs of new medications are currently too high for most patients and something needs to be done about it.

https://www.biospace.com/article/ip3o-pharma-companies-react-to-employers-and-health-plans-excluding-new-therapies-based-on-cost/

Tesaro Touts Maintenance Therapy Zejula During Ovarian Cancer Awareness Month

In 2015 Anne was diagnosed with stage III ovarian cancer. The diagnosis was made after she wound up in the emergency room complaining of severe back pain.

The diagnosis came as a complete shock and Anne immediately began a treatment regimen that included surgery. Months later she relapsed and was forced to continue treatment. Typically, when a woman has a recurrence of ovarian cancer, it is considered incurable. But, last year, Anne was told she was a good candidate for a newly-approved maintenance therapy that could halt or delay progression of the disease – Tesaro’s Zejula.

Formerly known as Niraparib, Zejula is an oral, once-daily PARP inhibitor used as maintenance treatment for ovarian cancer. It was the first PARP inhibitor to be approved by the U.S. Food and Drug Administration (FDA) that does not require BRCA mutation or other biomarker testing.

Since being placed on Zejula, Anne said she has something of a new lease on life. Her illness is under control and she’s been able to return to work – something she did not think was going to be possible.

Anne, along with Dr. Robert Holloway, the medical director of the Gynecologic Oncology Program at Florida Hospital, was part of an ovarian cancer awareness media blitz Tesaro conducted to highlight National Ovarian Cancer Awareness month.

Speaking to BioSpace, Holloway said there are a number of new treatment options for women with ovarian cancer that are allowing them to live longer following initial diagnosis. Holloway specifically pointed to Tesaro’s Zejula and said the once-per-day therapy is helping women maintain remission and preventing recurrence of the disease. Until Zejula, Holloway said, many patients had to go through a “wait and see” approach to their treatment. About 85 percent of patients went through a recurrence or relapse of disease, which required more chemotherapy or other treatments, Holloway said. When Zejula was approved last year though, ovarian cancer patients were able to benefit from the maintenance therapy and keep their cancers at bay.

“This is something that really benefits patients in the long run,” Holloway said.

Ovarian cancer is the fifth leading cause of cancer-related deaths in the United States. It is estimated that this year there will be more than 22,000 women in the U.S. diagnosed with the disease and about 14,000 disease-related deaths. The vast majority of diagnoses, about 80 percent, are found at an advanced stage.

Since the approval of Zejula last year, there has been significant movement in the treatment of ovarian cancer. The following are a few highlights from select company pipelines.

In June, Genentech received the green light from the U.S. Food and Drug Administration for Avastin (bevacizumab) as a treatment for stage III or stage IV ovarian cancer. The FDA approved Avastin in combination with chemotherapy followed by Avastin alone for the treatment of those cancer patients who have undergone initial surgical resection. According to the trial data that led to the regulatory approval, women who received Avastin in combination with chemotherapy, and continued use of Avastin alone, had a median progression-free survival of 18.2 months compared to 12 months in women who received chemotherapy alone.

Also in June, AstraZeneca and Merck revealed positive Phase III results from a trial testing Lynparza (olaparib) in BRCA-mutated (BRCAm) advanced ovarian cancer. Lynparza, also a PARP inhibitor, is being studied as a first-line maintenance therapy. The data showed patients treated with Lynparza demonstrated statistically significant and clinically meaningful improvement in progression-free survival compared to those receiving placebo. Lynparza is also being tested in another Phase III trial in combination with bevacizumab as first-line maintenance therapy for women with newly diagnosed advanced ovarian cancer, regardless of BRCA status. Data from that trial is expected in 2019.

In April West Lafayette, Indiana-based On Target Laboratories dosed the first patient in the Phase III trial of OTL38 in detecting ovarian cancer. As BioSpace previously reported, OTL38 is a ligand that targets folate receptors that appear in various types of cancers. OTL38 is injected into patients and includes an infrared dye that allows surgeons to visually identify cancer cells that can guide them in removing the cancer tissue.

Massachusetts-based ImmunoGen in March announced positive results from its FORWARD IItrial that showed the combination of mirvetuximab soravtansine and Merck’s Keytruda yielded positive results in platinum-resistant epithelial ovarian cancer. Then in June, the FDA granted Fast Track designation to Immunogen’s mirvetuximab soravtansine for patients with medium to high folate receptor alpha (FRα)-positive platinum-resistant ovarian cancer who received at least one, but no more than three prior systemic treatment regimens. On its own, mirvetuximab soravtansine is being evaluated in a Phase III trial as a stand-alone treatment.

While Holloway noted there has been a significant improvement in treatment options for ovarian cancer patients, he said little headway has been made in the diagnosis of the disease. There has been some new understanding that there is a genetic component to ovarian cancer, which means women who have a family member diagnosed with the disease should proactively work with their doctors to watch for signs and symptoms of the disease.

“We’re hopeful for a blood test or other kind of test, but that’s not been brought forth yet,” Holloway said.

https://www.biospace.com/article/cp8r-tesaro-highlights-benefits-of-maintenance-therapy-zejula-during-ovarian-cancer-awareness-month/

Piper sees early winners from annual list of new codes from CMS

The Centers for Medicare & Medicaid Services published its annual list of new codes ahead of the final rate determination including 99 codes, Piper Jaffray analyst William Quirk tells investors in a research note. He says Genomic Health (GHDX), LabCorp (LH), Myriad Genetics (MYGN), Natera (NTRA) and Quest Diagnostics (DGX) look like early winners in the 2019 Preliminary Clinical Laboratory Fee Schedule Fee Schedule determinations, with an incrementally positive competitive move for CareDx (CDNA). The analyst notes that Genomic Health received increased reimbursement for a couple tests and CareDx competitor TAI Diagnostics ends up with a likely longer pathway to reimbursement.
https://thefly.com/landingPageNews.php?id=2794795

Underinvestment in medtech threatens sector growth – EY

In its 2018 report on medical technology, Pulse of the industry, EY states that short-term growth is prioritised over long-term growth, with R&D suffering as a result.

This is despite aggregate revenue for the industry hitting new heights in 2017. Growth was only 4%, contrasting sharply with the 15% average annual growth rate achieved between 2000 and 2007.

In fact, medtech companies returned $16.4 billion to investors in buybacks and dividends last year, compared to $15.9 billion invested in R&D activities.

Pamela Spence, EY global life sciences industry leader, said, “Medtechs must invest in new data and customer-centric capabilities to build stronger ties with consumers or risk being ousted by technology companies and other entrants from outside the sector.

“To succeed in the digital future, medtechs will be judged not only on the safety and efficacy of their devices and tests, but on their ability to capture and deploy insights from these products to inform care delivery, with a growing emphasis on coordinated care.”

Technology companies – as opposed to medtech firms – are well placed to capitalise on the industry’s shifting balance of power, the report states. This is because patients are increasingly taking on an active role in their healthcare by wearing sensors, through using apps and via other digital interfaces.

One example is Apple’s watch, which incorporates an electrical heart rate sensor that can take an electrocardiogram (ECG) using an app that has been granted a ‘de novo’ classification by the US Food and Drug Administration (FDA).

Another point highlighted is the importance of data. The latter, states EY, will replace devices as the key value driver in medtech, with new market entrants benefiting over medtech companies if they fail to accelerate their digital agendas.

Jim Welch, life sciences advisory partner, Ernst & Young LLP, said medtechs have a ‘unique’ opportunity to capitalise on the progress made in the digital arena, particularly as they have strong connections with healthcare stakeholders.

But, he warned, “What they don’t have is in-house capabilities to develop personalized health care offerings. To change this, medtechs need to continue to be efficient with their capital, and prioritize shedding non-core assets. They must also invest more in digital collaborations that expand their customer experience and data and analytics capabilities so that they can get even closer to patients.”

https://pharmaphorum.com/digital/underinvestment-in-medtech-threatens-sector-growth-ey/

Novartis, Bayer eye appeal as UK court OKs Avastin for macular degeneration

England’s High Court has ruled it is lawful to use Roche’s Avastin (bevacizumab) in an unlicensed use in wet age-related macular degeneration (AMD) instead of more expensive approved drugs from Novartis and Bayer.

While NHS organisations welcomed the judgement on the grounds that it will save money, the drug companies said they are considering an appeal against the decision, arguing it undermines the EU’s regulatory system for medicines.

Avastin is licensed as a cancer drug, where it works by attacking the blood vessels that feed oxygen to tumours.

But this mechanism of action means it can also work in wet AMD, which is caused by abnormal growth of blood vessels in the back of the eye, causing blurring or loss of vision in the centre of the field of vision.

Novartis’ Lucentis (ranibizumab) and Bayer’s Eylea (aflibercept) are approved in wet AMD, but Avastin’s manufacturer Roche has never applied for a licence in ophthalmology.

But an independent trial by the US National Institutes of Health in 2012 found that Avastin was as effective as Lucentis in treating wet AMD, although serious adverse events were slightly more common with the Roche drug than with Lucentis.

The trial paved the way for off-label use of Avastin in wet AMD, at a much lower price as it is administered at around 1/500th of the dose used in oncology indications.

In England, 12 clinical commissioning groups had been using Avastin in wet AMD, and Novartis and Bayer called for a judicial review in a bid to make them use more expensive licensed alternatives.

Bayer and Novartis issued tersely-worded statements, saying they were considering an appeal against the decision, where the High Court ruled that Avastin could be used lawfully as part of a policy to save costs.

Describing the decision as a “setback for public health,” Bayer added that “the ruling prioritises the cost of medication over doctors’ clinical judgement and expertise, as well as over the regulatory assessment of a medicine’s quality, efficacy and safety.

“Bevacizumab, marketed as Avastin for the treatment of cancer, is not manufactured or licensed for use in the eye. A service using this product risks increasing the number of clinic visits and injections a patient needs for proper monitoring of their conditions and for sight-saving treatment, placing more strain on NHS services and the already stretched workload of NHS staff.”

Novartis made a similar argument in its statement, saying the ruling “threatens to jeopardise a world-leading system that has protected patients for many years by ensuring medicines have been tested rigorously and carefully scrutinised for delivering value.”

Dr Sheuli Porkess, deputy chief scientific advisor to the Association of the British Pharmaceutical Industry (ABPI), said: “We will consider this judgement carefully. Patients and doctors need total clarity on who determines whether medicines work and are safe to be used.

“This extraordinary judgement potentially undermines the regulation of all medicines and by doing that, neither patients nor doctors have clarity on what information to trust.”

David Hambleton, chief executive officer of NHS South Tyneside clinical commissioning group (CCG), one of the NHS groups involved in the case, welcomed the judgement.

He told the BBC: “We’ve always said we think that it’s important that patients should have the choice of a very effective treatment for wet AMD, and it’s actually a fraction of the cost of the other alternatives.

“So I think what we do now is offer patients that choice. We believe that they will support very strongly having a cost-effective, safe treatment and saving the NHS generally a lot of money. It is a victory for common sense over commercial interests.”

https://pharmaphorum.com/news/novartis-bayer-consider-appeal-as-court-says-avastin-can-be-used-in-amd/

Loxo Oncology granted orphan status for solid tumors treatment

The FDA granted Loxo Oncology orphan status for its treatment of solid tumors with neurotrophic tyrosine receptor kinase-fusion proteins.
https://thefly.com/landingPageNews.php?id=2794783