Category Archives: Drug Development

Amgen CEO says Aimovig is “one of most successful launches”

Wax Selection – Leaders in Pharma, Biotech & MedTech Recruitment

As biosimilars start to eat into sales of its blockbuster biologic drugs, Amgen needs new products to deliver growth – and it says migraine therapy Aimovig is off to a great start.

Chief executive Bob Bradway told investors on the company’s third-quarter results call that Aimovig (erenumab) is “shaping up to be one of the industry’s most successful recent launches” with sales reaching $22m in the quarter, ahead of analyst expectations.

“People suffering from migraine and their physicians have been waiting for years for an effective new therapy and they’ve reacted very well to Aimovig,” he said. The company estimates that more than 100,000 patients have taken the Novartis-partnered CGRP inhibitor since its launch in May as a once-monthly, self-injected preventive treatment for migraines.

The strong take-up has come despite approvals for rival CGRP inhibitors from Teva and Lilly, and Amgen says its first-mover advantage is backed up by a patient-friendly delivery device, as well as a willingness by healthcare payers to cover the product thanks to a price that encourages access.

The company is also making much of the fact that Aimovig is the only drug that targets the CGRP receptor rather than the CGRP protein itself, although for now there’s no strong evidence to suggest that confers a clinical advantage.

There’s no doubt that the rapid roll-out comes from pent-up demand for the drug among people with migraine who still suffer headaches despite other therapies. However Amgen’s commercial head Murdo Gordon said he believes “we’re scratching the surface of helping chronic migraine sufferers [and] we think that we’ll see continued demand growth.”

Overall, Amgen posted a 2% rise in revenues to $5.78bn in the quarter, despite pressure on older drugs like while blood cell booster Neulasta (pegfilgrastim) which showed the first signs of weakness from biosimilar competition after Mylan/Biocon launched their Fulphila rival in July. TNF inhibitor Enbrel (etanercept) also fell 5% in the face of competition from newer drugs in indications such as psoriasis.

There was also disappointing news once again for cholesterol drug Repatha (evolocumab) which rose 35% to $120m but missed expectations. Last week, Amgen reduced its price by 60% to $5,850 in a bid to kickstart sales growth and overcome payer resistance to the drug.

“This should substantially lower their out-of-pocket costs and lower the abandonment rate which is as high as 75%,” said Gordon, who said the cut was necessary to compete and would build much-needed volumes at the expense of near-term sales.

The Repatha discount comes as the US administration is ramping up the pressure on the biopharma industry on drug pricing, most recently proposing a reference pricing model to bring process closer into line with other affluent nations.

Commenting on the overall environment and how it looks likely to develop in 2019, Bradway said that the US has benefitted enormously from a market which allows patients to access new therapies “more quickly and more comprehensively than in other countries.”

“We think what’s required is more innovation, not less, and the system that enables people to get access to that innovation swiftly,” he suggested.

SOURCE: www.pmlive.com/pharma_news

Roche and SQZ expand cell therapy deal to more than $1.3bn

Wax Selection – Leaders in Pharma, Biotech & MedTech Recruitment

US biotech SQZ Biotechnologies has expanded a collaboration with Roche to develop cancer therapies based on antigen presenting cells, which may be able to target solid tumours and which are cheaper to make than the recently approved CAR-T drugs.

The deal announced late yesterday expands a collaboration that began in 2015 to a deal worth more than $1.375 billion if all development targets are met.

SQZ’s technology is based around antigen presenting cells (APCs), which occur naturally in the body and present antigen on their surface to stimulate a T-cell response against that particular antigen.

SQZ is able to genetically engineer APCs with tumour antigens, that when injected into the body can produce powerful responses from CD8 T-cells – also known as killer T-cells.

The hope is that these killer T-cells will produce a strong response against any tumour target of interest, across a wide range of cancers.

Roche already has a cancer immunotherapy on the market – its Tecentriq (atezolizumab) is already approved in certain kinds of bladder and lung cancer.

But unlike Novartis and Gilead it does not have cell therapy approved yet and is looking to develop cell therapies that could be used in solid tumours, instead of blood cancer like its rivals’ CAR-T therapies.

Under the collaboration, SQZ may receive up to $125 million in upfront payment and near-term milestones.

SQZ could earn up to $250 million in clinical, regulatory and sales milestones per product that emerges from the collaboration.

The Massachusetts-based biotech may receive development milestone payments of over $1 billion. Within the collaboration, SQZ and Roche could share commercial rights for certain approved products.

Howard Bernstein, chief scientific officer of SQZ, said: “This collaboration allows for a SQZ APC product engine that could potentially generate products with more potent immunologic responses through a simplified, more efficient manufacturing process.”

Chimeric antigen receptor T-cell (CAR-T) drugs are potentially curative in the patients that respond – but are very expensive to make as they involve harvesting a patient’s T-cells, genetically modifying them in a lab, and then re-injecting them to fight cancer.

SOURCE: www.pharmaphorum.com/news

FDA gives go-ahead for CRISPR-based sickle cell disease trial

Wax Selection – Leaders in Pharma, Biotech & MedTech Recruitment

Development of a stem cell therapy for sickle cell disease from Vertex and development partner CRISPR therapeutics can go ahead, after the FDA lifted a hold on a review.

The companies had applied to begin an early stage trial of CTX001, a gene therapy derived from a patient’s own stem cells, for beta-thalassemia and sickle cell disease.

Although it’s a long way from the market, the drug could be one of the first to use the revolutionary CRISPR/Cas9 gene editing technology to correct a genetic defect causing a disease.

After an application in April for a phase 1/2 trial in the UNS for adults with sickle cell disease, the FDA had further questions that needed to be resolved.

Without revealing further details, the companies said the trial had been put on hold until they could provide the information the FDA required.

The FDA has now lifted the clinical hold and allowed development to go ahead, although the companies gave no further information about the information required by the regulator.

CRISPR and Vertex have obtained approval for clinical trial applications for several countries outside the US for beta-thalassemia and SCD.

They said they are on track to begin a phase 1/2 study in SCD by the end of 2018 and are enrolling patients transfusion dependent beta-thalassemia in a phase 1/2 trial in Europe.

CTX001 uses the CRISPR gene editing technique to make a patient’s haematopoietic stem cells produce high levels of foetal haemoglobin (HbF) in red blood cells.

HbF is a form of the oxygen carrying molecule haemoglobin naturally present at birth, which is replaced by the adult form of haemoglobin.

The elevation of HbF by CTX001 could alleviate transfusion requirements for beta-thalassemia patients and painful and debilitating sickle crises for sickle cell patients.

CRISPR and Vertex began a strategic research collaboration in 2015 to discover and develop gene editing treatments using the CRISPR/Cas9 technology to correct defects in genes known to cause or contribute to certain diseases.

Vertex has exclusive rights to license up to six new CRISPR/Cas9-based treatments that emerge from the collaboration, and CTX001 represents the first treatment to emerge from the joint research program.

For CTX001, CRISPR and Vertex will equally share all research and development costs and profits worldwide.

Novartis yesterday unveiled data showing its crizanlizumab reduced occurrence of the painful and potentially fatal vaso-occlusive crises that occur when blood cells become stacked in patients with SCD, blocking arteries and cutting the oxygen supply to vital organs.

SOURCE: www.pharmaphorum.com/news/fda

Novo Nordisk to launch connected insulin pens in early 2019

Wax Selection – Leaders in Pharma, Biotech & MedTech Recruitment

Danish healthcare firm Novo Nordisk has unveiled plans to introduce new durable, connected insulin pens in early 2019.

The new NovoPen 6 and NovoPen Echo Plus connected insulin pens will replace the NovoPen 5 and NovoPen Echo insulin pens.

Novo Nordisk will commence the launch of new connected insulin pens in the first quarter of 2019. They are expected to be available in more than 50 countries.

The company expects to deliver connectivity for its disposable, pre-filled injection pens later in 2019.

Novo Nordisk commercial strategy and corporate affairs executive vice president said: “Our non-exclusive partnership strategy allows us to integrate with the various digital platforms that people are already using to help manage their diabetes.

“We firmly believe that this will help more people realise the full benefit of our innovative medicines and begin to ease the mental burden of diabetes treatment for those individuals.”

In parallel, the company has also entered into new partnership agreements with major diabetes technology firms such as Dexcom, Glooko and Roche to enable future integration of its connected pens with various digital health solutions.

As part of the deal with Roche, the partnership will incorporate insulin dosage information from Novo Nordisk’s connected pen technology into Roche’s open ecosystem, enabling to communicate with its digital diabetes management solutions such as mySugr.

The firm will also incorporate data from connected pen devices with its partners’ diabetes management solutions such as continuous glucose monitoring (CGM) systems and blood glucose meters (BGM).

Novo Nordisk will incorporate insulin dosing data with Dexcom CGM data in the coming years.

Roche diabetes care global head Marcel Gmuender said: “We believe in the tremendous benefits integrated digital diabetes management solutions can bring to people with diabetes, caregivers and healthcare systems as part of an open ecosystem and are excited to partner with Novo Nordisk to further drive innovation in this area.”

With marketing activities in more than 170 countries, Novo Nordisk is engaged in providing treatment solutions for obesity, haemophilia, growth disorders and other serious chronic diseases.

SOURCE: www.compelo.com/medical-devices/news

Study using DFMO shows positive results for children with high risk neuroblastoma

Wax Selection – Leaders in Pharma, Biotech & MedTech Recruitment

A paper published September 27 in Scientific Reports shows the positive results of a phase II clinical trial using the oral medication DFMO to prevent relapse in children with High Risk Neuroblastoma (HRNB).

Neuroblastoma is a form of cancer that develops from immature nerve cells found in several areas of the body. It occurs most often in infants and young children, usually under the age of five. The disease remains a challenge in pediatric oncology and current treatments include therapies that have significant long-term side effects for patients.

HRNB accounts for 15 percent of all childhood cancer deaths, in part, due to the fact that nearly half of all patients who reach remission will relapse.

“These results are promising and have changed the outlook for our patients with high risk neuroblastoma,” said Giselle Sholler, MD, director of pediatric oncology research at Spectrum Health Helen DeVos Children’s Hospital and principal investigator of the study.

“By using DFMO for two years after finishing conventional therapy, we’ve seen an overall two-year survival rate for these children of 97 percent. This is a large increase in survival,” Sholler added. “Previously it was believed that children with refractory and relapsed neuroblastoma were considered incurable. This study shows more than 50 percent of patients remaining in remission up to four years.”

Beat Childhood Cancer’s trial studied the use of difluoromethylornithine (DFMO) as a single agent for enrolled patients at 20 children’s hospitals from June 2012 to February 2016. The children received two years of oral DFMO twice daily and were evaluated for outcomes of event free survival (EFS*) and overall survival (OS). The study used targeted oral therapy of an ODC inhibitor (DFMO), as a maintenance therapy to prevent relapse in HRNB patients after standard therapy. DFMO works by targeting specific cancer stem cell pathways and “turning off” the cells, thereby preventing the cancer from growing back.

There were two arms in this study, the first designed for patients who had completed standard therapy, and the second for children who were able to achieve remission after having previously relapsed. Both of these patient populations are at very high risk of relapsing after completing treatment and therefore can be very good candidates for using a maintenance therapy with the goal of preventing relapse.

With a median follow up of 3.5 years, the first arm of the study had 100 eligible patients. The results show that two-year EFS was 84 percent and two-year OS was 97 percent.

With a median follow up of 3.7 years, the study enrolled 39 previously relapsed patients and the results reported in the journal showed that two year EFS was 54 percent and two-year OS was 84 percent for these children who had previously relapsed.

“While these EFS and OS figures at two years are remarkable, the really exciting part of these results is that EFS and OS are stable out to four years,” said Patrick Lacey of Beat NB Cancer Foundation, one of the childhood cancer parent-led foundations that funded this clinical trial. “Not only did this oral drug lead to a prolonged and stable remission for the children in this study, but the drug was extremely safe and well tolerated in this patient population.”

“While many children have been able to attain remission with the current, albeit harsh, upfront therapies, these remissions are not historically durable,” Dr. Sholler added. “The current five-year survival curves have not changed significantly in the past two decades despite recent increases in two-year survival as a result of intensified therapies and new multimodal therapies.”

Principal Investigator at MUSC, Jaqueline Kraveka, MD, states survival for children with high-risk neuroblastoma remains a challenge. “These results are groundbreaking and very exciting for oncologists and their patient families. I am thrilled to have our confirmatory study open at so many sites across the USA and Canada, enabling children to receive this treatment close to home.”

SOURCE: www.news-medical.net/news/20181001

JM to offer a new process to synthesise CBD

Wax Selection – Leaders in Pharma, Biotech & MedTech Recruitment

Johnson Matthey to offer a new process to synthesise ultra-pure cannabidiol, expanding on existing cannabinoid offerings.

Johnson Matthey, a global leader in science that enables a cleaner and healthier world has announced an expansion to its API and controlled substances portfolio by establishing a new synthetic method for ultra-pure cannabidiol, a medicinal component of the cannabis plant.

This synthesis will help Johnson Matthey (JM) support companies in developing novel treatments and medicines to help patients across a range of disease areas.

Cannabidiol (CBD) is one of the many chemical compounds in the cannabis plant and is known to possess medicinal and therapeutic properties. Unlike tetrahydrocannabinol (THC), another molecule in the cannabis plant, CBD does not cause intoxication or euphoria, two unwanted side-effects for medicines.

In light of CBD’s medicinal applications and the absence of psychoactive side-effects, demand for GMP-grade CBD from pharmaceutical companies has increased extensively in recent years.

It is now being investigated as a potential therapeutic treatment for various illnesses and diseases, including multiple cancers, seizures, dermatological conditions and anxiety.

JM has established more than 15 years’ experience in developing and commercialising a portfolio of ultra-pure synthetic cannabinoids and other controlled substances. By adding cannabidiol into this portfolio and filing a US drug master file (DMF) with a validated synthetic process, JM is excited to support companies looking to explore CBD’s medicinal applications further.

With manufacturing sites based in the US, JM is able to apply its knowledge of synthetic chemistry and purification techniques to CBD synthesis. In particular, JM’s solid form chemistry expertise has enabled the development of a free flowing crystalline powder, which is able to be particle size adjusted for a variety of formulation requirements.

Furthermore, JM has established a full suite of references of standards and impurities to facilitate CBD product development, which helps to ensure molecules are synthesised to an ultra-pure standard.

As well as synthetic chemistry expertise in controlled substances, JM also offers API manufacturing capabilities for botanical extraction and purification of cannabinoids. Based on the growing popularity of medicinal cannabinoids, JM is actively investigating the development of other cannabinoid compounds.

“As a leader in API development, we are delighted to add the high-value synthesis of cannabidiol to our expanding portfolio of Pharma solutions,” said Paul Evans, VP Generic Products and Solutions at JM.

“This will enable companies to easily explore the medicinal properties of cannabinoids, and combined with our development and manufacturing capabilities, deliver novel treatments and medicines to patients.”

SOURCE: www.manufacturingchemist.com/news

Teva wins FDA OK for crucial migraine drug

Wax Selection – Leaders in Pharma, Biotech & MedTech Recruitment

Vital to Teva’s turnaround, Ajovy will challenge Novartis/Amgen’s Aimovig.

Teva has secured US approval for migraine drug Ajovy, a key part of new chief execeutive Kåre Schultz’s turnaround plan for the company.

The FDA approved the CGRP inhibitor for the prevention of migraine in adults with either monthly or quarterly dosing, based on two phase 3 studies showing that it could reduce the number of migraine days over a 12 -week period compared to placebo.

Ajovy (fremanezumab) is the second CGRP inhibitor to be approved for marketing after Novartis and Amgen’s Aimovig (erenumab), which got the green light in May and is dosed monthly, and ahead of late-stage rivals from Eli Lilly (galcanezumab) and Alder Biopharma (eptinezumab).

Teva would have been earlier to market but the FDA review for its products was delayed by three months while it sorted out some manufacturing issues. With Lilly due to hear from the FDA any day now, potentially setting up a three-way marketing battle, the Israeli drugmaker will be hoping the three-monthly dosing option will be popular with doctors and patients – particularly as there seems to be little to separate the antibodies when it comes to efficacy.

In a statement, Teva said that it had set a list price of $575 for the monthly dose and $1,725 for the quarterly dose, giving an annual cost of $6,900 before rebates and discounts – exactly the same level as Novartis and Amgen positioned Aimovig, and in line with stablished therapies like Allergan’s Botox.

It is estimated that with discounts the annual cost of the drugs could be around the $5,000 mark. However, the US-based Institute for Clinical and Economic Review (ICER) said recently that – even at that lower price – the new drugs should only be used with prior authorisation and after other preventive options such as Botox and anti-epilepsy drugs like topiramate because their long-tern safety is still unclear.

It is estimated that more than 36 million people in the US suffer from migraines, with around 40% of these would be suitable for preventive treatment, and that has led some analysts to predict buoyant sales of the CGRP inhibitors.

GlobalData said recently that it expects the market for migraine drugs to grow at more than 10% a year to reach almost $9bn by 2026, with the prevention category driven by the injectable CGRP inhibitors.

”Market share is going to come down to…variables such as frequency of administration, position to market, and market access strategy,” according to GlobalData analyst Rahael Maladwala.

“Each drug has its own set of advantages; Aimovig will be the first to market, Eli Lilly’s has significant experience in marketing drugs, and an extensive sales force, while both of the other drugs have a quarterly dosing regimen compared to monthly.”

On balance, GlobalData is putting Aimovig out in front in 2026 given its first-mover advantage generating nearly $1.4bn in sales in the seven major pharma markets (US, France, Germany, Italy, Spain, UK, and Japan).  Teva filed Ajovy in Europe earlier this year, but here it will also lag behind Aimovig as Novartis and Amgen picked up EMA approval for their drug in July.

Analysts at Jefferies have suggested Ajovy could become a $500m product in 2022, which will go some way towards counteracting the decline in sales of Teva’s blockbuster multiple sclerosis therapy Copaxone (glatiramer acetate), its rapidly ageing cash-cow product.

Since Schultz took the helm last year, Teva has been shedding staff and restructuring the business to cut costs, selling off its women’s health business for almost $2.5bn in order to pay down a very high level of debt stemming from its $39bn acquisition of Allergan’s generics business Actavis in 2015.

Ajovy is one of 23 New Drug Application (NDA) approvals Teva is targeting between fiscal 2019 and fiscal 2023 to help replace Copaxone and drive top-line growth, along with other new products such as movement disorder therapy Austedo (deutetrabenazine).

SOURCE: www.pmlive.com/pharma_news

Buy-out sets sights on glucose responsive insulin

Wax Selection – Leaders in Pharma, Biotech & MedTech Recruitment

Diabetes specialists Novo Nordisk have acquired Ziylo, a University of Bristol spin-out company.

Based at Unit DX science incubator in Bristol, Ziylo has been pioneering the use of its platform technology – synthetic glucose binding molecules – for therapeutic and diagnostic applications.

The acquisition gives Novo Nordisk full rights to Ziylo’s glucose binding molecule platform to develop glucose responsive insulins. The development of glucose responsive insulins is a key strategic area for Novo Nordisk in its effort to develop this next generation of insulin which would lead to a safer and more effective insulin therapy.

“Novo Nordisk is the ideal company to maximise the potential of the Ziylo glucose binding molecules in glucose responsive insulins and diabetes applications, and it brings hope of a truly groundbreaking treatment to diabetes patients,” said Dr Harry Destecroix, chief executive officer and co-founder of Ziylo. “Novo Nordisk is the leader in the diabetes field, with deep clinical development and regulatory expertise and an established commercial infrastructure to deliver important new therapies to patients.”

Ziylo’s glucose binding molecules are synthetic molecules that were designed by Professor Anthony Davis at the University of Bristol. These stable, synthetic molecules exhibit an unprecedented selectivity to glucose in complex environments such as blood. The combination of this technology with state-of-the-art insulin engineering pioneered by Novo Nordisk aims to develop the world’s first glucose responsive insulin and transform the treatment of diabetes.

Novo Nordisk acquires all shares in Ziylo for an upfront payment and earn-outs with contingent milestone payments. Total payments under the agreement could ultimately exceed 800 million dollars upon the achievement of certain development, regulatory and sales milestones by Novo Nordisk.

SOURCE: www.labnews.co.uk/news

Shire’s von Willebrand disease therapy Veyvondi approved in EU

Wax Selection – Leaders in Pharma, Biotech & MedTech Recruitment

The European Commission has approved Shire’s Veyvondi for treatment of the bleeding disorder von Willebrand disease (VWD).

VWD is the most common inherited bleeding disorder, affecting up to 1% of the global population, and is caused by deficiency or dysfunction in the protein known as von Willebrand factor (VWF).

The commission granted a marketing authorisation for Veyvondi (vonicog alfa, recombinant von Willebrand factor) for bleeding events and treatment or prevention of surgical bleeding in adults with VWD when desmopression treatment alone is ineffective or not indicated.

Veyvondi is the first recombinant treatment for VWD that addresses primary deficiency or dysfunction of VWF while also allowing the body to restore and maintain adequate Factor VIII plasma levels.

Approval is based on outcomes from three clinical trials of a total of 80 patients with VWD exposed to Veyvondi.

These include a phase 1 multicentre, controlled, randomised, single-blind, dose-escalation study of the safety, tolerability and pharmacokinectics  in subjects 18 to 60 years of age with severe VWD.

Also in the dossier was a phase 3 multicentre, open-label study to assess the pharmacokinetics, safety and efficacy of the Veyvondi and recombinant factor VIII and Veyvondi alone in the treatment of bleeding episodes in adult subjects with severe VWD.

There was also a phase 3, prospective, open-label, uncontrolled, non-randomised, international multicentre study to assess the haemostatic efficacy and safety of rVWF with or without recombinant factor VIII in 15 adult subjects with severe VWD undergoing major, minor, or oral elective surgical procedures.

Andreas Busch, head of R&D and chief scientific officer at Shire, said: “The approval in Europe for Veyvondi marks a key milestone in our efforts to tackle unmet medical needs for those living with von Willebrand disease.

“We are excited to take the next steps in ensuring that Veyvondi is widely available across Europe to address the individual needs of those affected by the condition and in need of factor replacement.”

SOURCE: www.pharmaphorum.com/news

New C.diff drug to be tested on patients for first time

Wax Selection – Leaders in Pharma, Biotech & MedTech Recruitment

A new drug aimed at treating potentially deadly Clostridium difficile (C. diff) infections is set to be tested on patients for the first time.

Glasgow-based life sciences firm MGB Biopharma (MGB) said it was preparing to launch a Phase II clinical trial of its anti-bacterial agent MGB-BP-3.

The trial is expected to involve 30 patients based in North America.

All have been diagnosed with C.diff-associated disease (CDAD).

C.diff infections can cause diarrhoea and fever.

They have been a major problem in hospitals around the world, with thousands of deaths in the US alone linked to the bug each year.

The bacteria are able to take over the gut when a course of antibiotics kills off the bugs that normally live there.

MGB’s announcement came after it raised £1.3m from investors for trials of the new drug, which was invented at the University of Strathclyde.

The funding round was led by Edinburgh-based Archangels, with co-funding from a range of sources, including the Scottish Investment Bank, Barwell and Melrose-based Tri Capital.

The cash supplements a £2.7m grant awarded earlier this year by Innovate UK.

MGB said its trial would “evaluate safety and tolerability, efficacy and in particular look for improvement in global (or sustained) cure rates”.

Chief executive Dr Miroslav Ravic said: “We are already witnessing renewed interest in our new anti-bacterial agent and its trial in key medical centres in North America where CDAD is particularly prevalent.

“This offers opportunities both to progress the study rapidly and to attract increased attention to the results for this important trial.”

The company said it was aiming to start the trials in areas of the US and Canada with a high incidence of CDAD early next year.

SOURCE: www.bbc.co.uk/news/