16 February 2018

Roche to take over Flatiron Health for $1.9bn 

Roche has entered a definitive agreement to acquire all shares of Flatiron Health, a US-based healthcare technology and services firm, for a total consideration of $1.9bn.

The deal expands Roche’s current 12.6% equity stake in the firm and is expected to aid in the development and delivery of new innovative medicines for cancer patients.

Flatiron Health carries expertise in oncology-specific, electronic health record software and generation of real-world evidence for cancer research.

The firm worked together with multiple community oncology practices and academic medical centres in the country to develop a range of software products that help in rolling out the real-world evidence at the point of care.

Roche Pharmaceuticals CEO Daniel O’Day said: “This is an important step in our personalised healthcare strategy for Roche, as we believe that regulatory-grade real-world evidence is a key ingredient to accelerate the development of, and access to, new cancer treatments.

“As a leading technology company in oncology, Flatiron Health is best positioned to provide the technology and data analytics infrastructure needed not only for Roche, but for oncology research and development efforts across the entire industry.”

Roche plans to preserve Flatiron’s existing business model, partnerships network, overall objectives, sales and marketing, provider-facing and life science business activities.

Flatiron expects to continue offering its services to both the existing and future partners.

Flatiron Health co-founder and CEO Nat Turner said: “This important milestone will allow us to increase our investments in our provider-facing technology and services platform, as well as our evidence-generation platform, which will remain available to the entire healthcare industry.”

Planned to conclude in the first half of this year, the deal is subject to customary closing conditions.

15 February 2018

CureVac to develop mRNA vaccines for influenza and malaria

The Bill & Melinda Gates Foundation has awarded two new grants to CureVac, a German biopharmaceutical firm, to support the development of mRNA-based vaccines for the prevention of influenza and malaria infections.

Set to utilise CureVac’s RNActive prophylactic vaccine technology, the new programmes will aim for a universal flu vaccine that will shield from all influenza A strains over the long term.

This vaccine is expected to replace the seasonal vaccine, removing the need for a new flu vaccine every year.

In addition, a new mRNA vaccine will be developed to provide protection from Plasmodium falciparum malaria parasite, which is believed to pose the greatest threat.

CureVac co-founder and CEO Ingmar Hoerr said: “Successfully exploring the potential of the mRNA platform for development of a universal flu vaccine would be a consequential achievement, benefitting much of the world’s population.

“Our mRNA platform enables the cost-effective and fast manufacturing of vaccines to prevent these and other serious diseases, potentially making a difference in the lives of millions of people around the world.”

The new funding is part of an agreement signed between the firms in 2015, during which the Bill & Melinda Gates Foundation provided a $52m equity investment to advance the development of CureVac’s mRNA technology platform and support the construction of an industrial-scale production facility.

It is intended to aid development of new prophylactic vaccines for infectious diseases that disproportionately affect people in economically backward countries.

The terms of the ongoing partnership will see the availability of all Gates Foundation-funded products at an affordable price in such countries by CureVac, which can market them in developed countries on its own and/or through licensees.

14 February 2018

‘Killer cells’ could be a cheaper alternative to CAR-T therapy

Researchers at the MD Anderson Cancer Center at the University of Texas are conducting trials on natural killer cells (NK cells) that could offer an alternative to costly Chimeric Antigen Receptor T-cells (CAR-T cells) used in cancer treatment.

NK cells are a type of white blood cell that kill virally-infected cells and control early signs of cancer. They can be harvested from cord blood – blood from the placenta and umbilical cord that is rich in blood stem cells.

Early data from a trial involving participants with chronic lymphocytic leukaemia showed around two-thirds of participants administered with NK cell therapy experienced a complete response after 30 days, a similar level of efficacy to CAR-T cells.

Researchers have been able to manufacture 40 million NK cells targeting CD19, a biomarker found in certain blood cancer cells. Producing a similar amount of CAR-T cells would be around 100 times more expensive.

In August last year, the first gene therapy was approved by the US Food and Drugs Administration. It has been used to successfully treat blood cancers such as childhood leukaemia and some lymphomas.

The therapy requires drawing blood from patients, separating out T cells and genetically modifying them. The engineered cells further multiply in the patient’s body and identify and kill cancer cells. Although CAR-T therapy has proved extremely effective, it is costly to produce and administer, with a single infusion costing $475,000 when the therapy was first approved.

As NK cells can be harvested from readily-available cord blood supplies and then stored, they could offer a cheaper option.

However, with ‘off-the-shelf’ therapy, there is a risk of graft-versus-host syndrome in which the donated blood stem cells view the recipient’s body as foreign and attack it.

Professor of stem cell transplantation and cellular therapy at the MD Anderson Cancer Center Katy Rezvani said: “Natural killer cells are the immune system’s most potent killers, but they are short-lived and cancers manage to evade a patient’s own NK cells to progress.

“Our cord-blood derived NK cells, genetically equipped with a receptor that focuses them on B-cell malignancies and with interleukin-15 to help them persist longer – potentially for months instead of two or three weeks – are designed to address these challenges.”

13 February 2018

Purdue Pharma will no longer promote opioids to doctors

The largest US opioid manufacturer, Purdue Pharma, has announced that it is ending the promotion of opioids to physicians.

Purdue Pharma will no longer send sales representatives to doctors’ offices to market the prescription of opioids. The company has also announced that it will be reducing its sales force from more than 400 to 200, with its remaining marketing staff focusing on the promotion of non-opioid drugs.

“We have restructured and significantly reduced our commercial operation and will no longer be promoting opioids to prescribers,” Purdue said in a statement.

This follows multiple lawsuits that have been filed against opioid manufacturers for their role in the opioid crisis, with at least 14 US states currently suing Purdue.

Among other drugs, Purdue manufactures oxycodone – a semi-synthetic opioid – under the brand name OxyContin. OxyContin is prescribed to help relieve severe ongoing pain, and is marketed as providing 12-hour pain relief for patients. The company made over $35bn in sales of OxyContin, which is its bestselling drug, from 1996 to 2017.

A 2016 investigation by the Los Angeles Times found that the intended effect of the drug wore off in less than 12 hours, causing patients to experience withdrawal symptoms including an intense craving for the drug that could lead to addiction.

The company has faced criticism for a marketing campaign, launched in 1996, that exaggerated the drug’s benefits and incorrectly claimed that the rate of addiction among pain patients who are treated by doctors was ‘much less than 1%’. The overprescription of the drug and the aggressive marketing campaign used to promote it are widely blamed for triggering a prescription opioid crisis in the US.

Purdue was fined $634.5m in 2007 for misleading the public about the drug’s risk of addiction after an investigation by the US Drug Enforcement Administration concluded that that Purdue’s marketing was ‘aggressive, excessive and inappropriate’.

Of the 42,000 overdose deaths in the US in 2016, 40% were caused by prescription opioids. Although many are now calling for other manufacturers to follow suit and end the promotion of opioids, for some there is still a way to go to fully address the scale of the crisis.

Kevin O’Grady, who heads the Midwest Recovery Centers in Kansas City, told USA Today: “This recognition by Purdue is a step in the right direction. However, it only represents a small fraction of the problem. The focus still seems to be trying to stop this epidemic by increasing legal consequences, rather than treating it as an illness.”

12 February 2018

FDA releases generics guidances to encourage competition

The US Food and Drug Administration (FDA) has released 57 new and revised product-specific guidances for generic drugmakers, following criticism from the US Government Accountability Office (GAO).

Product-specific guidances are issued by regulatory bodies to ensure that generics are pharmaceutically equivalent to their reference listed drug and that the two drugs have the same safety and efficacy. These reports are used in the development of generic versions of brand name drugs.

Generic drugs must have the same dosage, intended use, effects, side effects, route of administration, risks, safety and strength as the original drug.

The most recent posting contains 57 product-specific guidances – 35 new and 22 revised. Fifteen of the new draft guidances and four of the revised guidances are for complex drug products, including multiple products that do not yet have generic competition.

This follows a report by the GAO that accused the FDA of failing to issue product-specific guidance documents and recommended that the agency announce its plans to issue and revise guidance for complex drugs.

The newest product-specific guidances include guidances for Pfizer’s eczema drug Eucrisa and Clovis Oncology’s PARP therapy Rubraca. They may facilitate the development of cheaper generic alternatives to these complex drugs.

FDA commissioner Scott Gottlieb said: “Since becoming commissioner I’ve made it my goal to improve patient access to affordable drugs. No patient should be priced out of critical medicine. It’s a public health concern if patients can’t access the drugs they need. It’s an issue we’re addressing using the full scope of our authorities.

“As part of this commitment, we’re advancing many policies aimed at making it easier to bring safe, effective, high-quality generic competition to the market, especially for a category of branded medicines known as ‘complex drugs.’ These drugs are often harder to ‘genericise,’ or copy, under our traditional approaches. In too many cases, that has discouraged competition. It’s meant branded drugs have enjoyed monopolies long after their patents and exclusivities have lapsed.”

In a statement, the agency said that it would continue to routinely post and revise product-specific guidances.

In June last year, the FDA launched a Drug Competition Action Plan to improve patient access through the development of generic drugs in pharmaceutical categories that lacked competition. This particularly focused on ‘complex’ drugs, which are drugs that involve a complex formulation or complex active ingredient, thus making it harder to develop a generic equivalent. Increased transparency in the publication of product-specific guidances may encourage further competition and greater public access to cheaper generic drugs.

12 February 2018

Boehringer to focus on schizophrenia therapies development

Boehringer Ingelheim has announced plans to shift its focus onto drug development for the treatment of schizophrenia after its investigational compound BI 409306 failed to meet efficacy endpoints in Phase II clinical trials for Alzheimer’s disease (AD).

The firm intends to cease the evaluation of BI 409306 for AD and will direct its efforts on testing the drug candidate in two ongoing trials designed to prevent relapse and occurrence of a first psychotic episode in schizophrenia patients.

BI 409306 is a potent, selective inhibitor of phosphodiesterase E9 (PDE9) and is being developed to target the glutamatergic signalling pathways in the brain for improving synaptic strength and plasticity.

The malfunctioning of these signalling pathways is believed to be the pathophysiological basis for schizophrenia’s positive, negative and cognitive symptoms.

Boehringer Ingelheim CNS and immunology therapeutic area head Dr Jan Poth said: “We recognise the immense anticipation around any progress in brain research that brings us closer to finding solutions for the many millions of people living with dementia.

“However, this is what research is about: disappointments are a daily experience in science, but even these clinical trial results will add to the understanding of brain function and contribute to future progress in this area.”

The Alzheimer’s trials were part of a clinical trial programme aimed at investigating the efficacy of compounds targeting glutamatergic malfunctioning as potential new therapies for certain symptoms and traits of a mental illness.

Performed in a total of 457 subjects with cognitive impairment caused by Alzheimer’s, these trials were intended to assess the efficacy, safety and tolerability of BI 409306 over 12 weeks, along with its superiority over placebo.

In addition to the schizophrenia trials, Boehringer plans to conduct separate Phase II trials of another compound called BI 425809 for treating various central nervous system indications, including Alzheimer’s.

9 February 2018

Deliv Rx offers same-day prescription medicine delivery

Crowdsourced last-mile delivery company Deliv has launched Deliv Rx in the US, a service that will partner with pharmaceutical companies to provide same-day deliveries of prescription medicines to patients.

With same-day prescription medicine delivery already a crowded market, where companies such as Phox Health and Phil have held influence for years, Deliv Rx aims to work with, rather than disrupt, existing players.

Customers can order medicines through their pharmacy and ask for same-day delivery at the checkout; their pharmacy will then have the opportunity to deliver the medicines themselves, or outsource the delivery to Deliv Rx in exchange for a pre-negotiated fee.

“Deliv is not an e-commerce site, it’s not a marketplace, it’s not a store, and it’s not an app looking to manage prescriptions,” said company CEO Daphne Carmeli. “Deliv is purely a crowdsourced base, last-mile delivery service. Think of us as a local UPS without the planes and warehouses. We are an asset-free logistics provider.”

Deliv Rx is set to benefit from the resources and network provided by its partner companies; same-day drug delivery service Phil received $10m in funding in August last year, and partnered with telemedicine company Lemonaid Health in May. The service will also take advantage of Deliv’s existing infrastructure, which has 4,000 retailers and businesses as its customers, and serves 35 major US markets across over 1,400 cities.

The market for same-day medicine delivery is set to grow considerably. US consumers are expected to spend $610bn on medicine by 2021, up from $360.1bn in 2017, and in 2017, 80% of shoppers expected a next-day delivery option when buying online. In 2016, 4.45 billion prescriptions were issued in the US, and many of these were dispensed at one of only 67,000 pharmacies in the country, suggesting services such as Deliv Rx will become increasingly popular.

“Waiting for a prescription when you’re in a hurry is annoying, but waiting for it when you really need it is simply unacceptable,” said Carmeli. “Deliv Rx enables pharmacies to be there for their customers in the best way at the most critical time.”

9 February 2018

Takeda and Fujifilm to develop regenerative medicine therapies

Takeda Pharmaceutical has partnered with Fujifilm for the development of regenerative medicine therapies to treat heart failure.

Under the collaboration, Takeda will use cardiomyocytes that would be derived from iPSC at Fujifilm’s subsidiary Cellular Dynamics International in the US.

The alliance will see a combination of Fujifilm’s iPSC-related and engineering technologies expertise with Takeda’s iPSC technology capabilities.

The Center for iPS Cell Research and Application (CiRA) at Kyoto University will additionally support these efforts for regenerative medicine development through collaborative research and expertise in pre-clinical and clinical studies for making safe and effective drugs.

Fujifilm Regenerative Medicine division general manager Aiichiro Hiruma said: “In addition to establishing new treatment methods for patients with heart disease, Fujifilm and Takeda will contribute to the elevation of regenerative medication business to the industrial stage by applying our engineering technologies to manufacture high-quality cells safely and efficiently.”

Under the contract signed by the firms, Takeda holds an option to first negotiate a joint and global commercialisation of regenerative medicine products made using iPSC-derived cardiac cells.

Fujifilm is set to receive an upfront amount from Takeda, while both firms will investigate the safety and efficacy of the potential regenerative medicine therapies.

Takeda Regenerative Medicine Unit global head Seigo Izumo said: “Fujifilm has impressive capabilities in high-quality cell manufacturing technology, an innovative area with significant treatment potential.

“This collaboration allows us to leverage the strengths of both organisations with the goal of achieving meaningful, life-changing benefits for patients.”

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