Sarepta Therapeutics Opens its Research and Manufacturing Center at Andover

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Sarepta Therapeutics, a U.S. commercial-stage biopharmaceutical company focused on the discovery and development of unique RNA-targeted therapeutics for the treatment of rare neuromuscular diseases, has announced the grand opening of its Research and Manufacturing Center in Andover, Massachusetts.

The 60,000 square foot state-of-the-art facility significantly enhances Sarepta’s research and manufacturing capabilities as it expands its global commercial footprint and rapidly advances its Duchenne muscular dystrophy (DMD) pipeline, which comprises a robust exon skipping platform, and next generation approaches such as gene therapy and utrophin upregulation programs. The current focus of the manufacturing facility will be to advance Sarepta’s development pipeline and research programs. By the end of the year, Sarepta could have up to seven investigational DMD treatments in the clinic.

Ultimately, Sarepta’s goal is to help as many individuals with DMD as possible. To support this objective, Sarepta plans to expand its headcount in Andover by 100 percent over the next 12 to 18 months, adding to the approximately 50 employees currently based in Andover.

“Since first moving to Massachusetts in 2013, Sarepta has benefited enormously from the Commonwealth’s talented and highly-educated workforce,” said Edward Kaye of Sarepta. “This expansion underscores our ongoing commitment to patients with Duchenne and investment in the vibrant Massachusetts economy. We are grateful to Governor Baker, the Massachusetts Life Sciences Center, MassBio, the Town of Andover, and other state and local officials who support our mission to serve the greater DMD community.”

System pharmacology modelers throw light on drug discovery in Alzheimer’s

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InSysBio scientific group led by Tatiana Karelina developed a quantitative system pharmacology model of Alzheimer’s disease. First part published in CPT PSP shows how to design initial phases of clinical trials of new drugs and to interpret the data obtained.

Alzheimer’s is a chronic neurodegenerative disease which leads to the senile cognitive impairment and memory loss. Every third person older than 70 years suffers from it. Such changes are caused by functional disorders and subsequent death of neurons. However triggers of processes resulting in brain cell death are still remain unknown. That’s why there is no effective therapy for Alzheimer’s disease.

At the moment, the most common hypothesis is a theory of the toxic effect of the beta-amyloid protein, which accumulates in the brain with age, aggregating into insoluble amyloid plaques. The presence of these plaques in the brain is the main marker of Alzheimer’s disease (unfortunately, often found postmortem). Soluble (not aggregated into plaques) forms of the protein are considered to be toxic too.

All modern therapies act in one of the three ways: they can block production of soluble beta-amyloid, destruct protein before transformation into insoluble form, or to stimulate the plaque degradation. “Clinical trials for Alzheimer’s therapies have got one significant feature – their short duration. They last for no more than 5 years, whereas the disease can progress for decades. And early Phase I-II tests last for only few weeks. With such experiment design one can affect only on the processes of distribution and degradation of the soluble beta-amyloid forms. Therefore we developed this part of our model to analyze and predict the dynamics of the new generation of drugs, for instance the inhibitors of amyloid production”, says Tatiana Karelina, the head of the neurodegenerative disease modeling group, InSysBio LLC.

The first difficulty encountered by drug developers is the interpretation of the results obtained in animal tests. In general, most studies of the distribution of amyloid are carried out on mice: scientists inject a labeled protein into the mouse brain and observe the distribution of the radioactive label. Alternatively, the dynamics of amyloid in the presence of drugs is studied. Based on the data obtained, researchers can calculate the “therapeutic window” for the medication – a range of doses from the minimum effective to the maximum non-toxic. Then doses for human or monkey are calculated by using mass or volume scaling (for the body, the parameters change as many times as its mass or volume is greater than the mass or volume of the mouse).

The project team collected the data from the literature and derived a system of equations that fully described the existing results. Firstly the model was calibrated (i.e. the missing parameters were estimated) for the mouse, and then for the human and monkey. It turned out that one cannot use the scaling method to transfer results from rodents to primates (as it’s often done). The deduced mathematical equations have shown that not only the rate of production of beta amyloid (as activity of corresponding genes) differs, but moreover the blood-brain barrier is different in rodents and higher primates. At the same time, there was no significant difference between the human and the monkey, and the standard scaling can be used to translate predictions between them.

The next big question in Alzheimer’s clinical trials is how to understand if the drug affects specific target on the short term. It is impossible to observe the processes that occur in the human brain directly. Usually a cerebrospinal fluid probe is taken for analysis of the change in the concentration of beta-amyloid. Actually, these data strongly differ from the values of amyloid concentration in the brain, since the cerebrospinal fluid is strongly influenced by the processes taking place in the blood plasma, and amyloid demonstrates another dynamicsy.

“If there is such a big structural model calibrated on the big amount of data one can easily match the results of cerebrospinal fluid sample analysis with the real processes in the patient brain. This will greatly accelerate the development of new drugs and improve the accuracy of the therapy selection”, explains Tatiana Karelina.

Scientists report that their model allows to predict how these new drugs must be administered. Total daily dose can be diminished but should be split for several parts during the day, providing optimal brain efficacy. InSysBio team is confident that the systems-pharmacological modeling can greatly improve the development of drugs from Alzheimer’s disease and are already negotiating the introduction of technology with their partners in the pharmaceutical industry.

Almac Group Announces Publication of Prostate Cancer Metastatic Assay Validation

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Almac Group’s Diagnostics business unit has announced the Journal of European Urology has published results relating to its Prostate Cancer Metastatic Assay. The publication demonstrates the assay can be used to analyse primary prostate cancer FFPE samples to identify a molecular subgroup with a high risk of developing distant metastases. The assay therefore has the potential to guide the choice of therapy for patients presenting with primary prostate cancer.

Professor Richard Kennedy, MD, PhD, VP and Medical Director, Almac Diagnostics and McClay Professor in Medical Oncology, Queen’s University Belfast commented “An unbiased discovery approach was used to identify a molecular subtype of primary prostate cancer that demonstrated metastatic biology. This approach has created a very robust assay with excellent performance, independent of clinical factors such as Gleason and CAPRA. We believe it will play a significant role in aiding clinicians to select the most appropriate therapy regimen for their patients.”

The study was conducted in conjunction with The Movember / Prostate Cancer UK funded Prostate Cancer Centre of Excellence at Queen’s University of Belfast and Manchester University along with Cardiff University, University College Dublin, Oslo University and the University of Surrey. Independent assay validation was performed using 322 radical prostatectomy samples with Metastatic Assay positive patients having increased risk of biochemical recurrence (Multivariable HR 1.62; p= 0.0092) and metastatic recurrence (Multivariable HR=3.20; p=0.0001). A combined model with CAPRA-S identified patients at increased risk of biochemical and metastatic recurrence superior to either model alone (HR=2.67; p<0.0001 and HR=7.53; p<0.0001) respectively. “The publication of this manuscript in a journal of this calibre represents a significant milestone in the assay’s development and with two additional manuscripts being prepared for submission, further milestones are expected to be reached later this year. The first is another clinical validation but this time using biopsy FFPE tissue and the second is analytical validation to demonstrate the assay can be transferred to several commercial platforms.” said Professor Paul Harkin, President and Managing Director, Almac Diagnostics. He went on to say “Almac has been at the forefront of RNA based genomic innovation for over 10 years and this assay represents just one of our innovative pipeline of predictive and prognostic tests”.

Metsä Board Launches Its Unified Offering of Products and Services

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The Better with Less initiative highlights the company’s aims to improve consumer experience and make packaging even more sustainable

Metsä Board has launched its clarified product and service portfolio on May 4th at Interpack in Düsseldorf. The company presents its offering of premium paperboards and related services, with focus on contributing to even more sustainable, safe and efficient packaging, now found under one Metsä Board family.

The Better with Less initiative showcases Metsä Board’s aim to create, together with its customers, considered and innovative, renewable contemporary packaging solutions fitting brands and demands of the future world.

“Consumers expect better experiences with less environmental impact. Brand owners are looking at how to optimise packaging to be more sustainable, safer and lighter in weight. We are known for our pioneering expertise in high-quality, lightweight paperboards and are committed to improving further. Now with our unified product portfolio and targeted services we can provide even stronger support to our customers to jointly improve sustainability and efficiency of packaging throughout the value chain,” says Mika Joukio, CEO of Metsä Board.

As part of the launch Metsä Board renews the product names of its premium quality lightweight paperboards to bring the offering to customers in a clearer way that makes product selection even easier. The three service areas – Packaging Analysis & Design, Availability Services and Technical Expertise and R&D – complement the products and provide collaboration initiatives for joint development with customers to develop better and lighter packaging.

While the company’s offering and product names will be harmonised, the products and their specifications will remain unchanged. All Metsä Board mills hold PEFC™ and FSC® Chain-of-Custody certificates, as well as the highest environmental and manufacturing quality standards. Metsä Board’s paperboards use fresh forest fibres from sustainably managed northern European forests.

The “BB Dose”: a Unither innovation that guarantees more safety with the infant

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Unither Pharmaceuticals is the world leader in the production of sterile single-unit dosage forms using the Blow-Fill-Seal (BFS) technology.

Since 1993, Unither Pharmaceuticals has been conducting various studies to better meet laboratories needs and thus facilitate patient medication intake. The new system brought by Unither, called “nose project”, does not damage the baby’s nose.

One of the studies conducted by UC/Consultants focuses on the use of physiological serum on infants. We found that mothers perceive the “physiological serum” as a neutral water, with a neutral pH, which does not necessarily contain antiseptic; Or a liquid such as blood. As for its use, the mothers always operate with a defined ritual, which shows their involvement around the product. Their expectations are very strong: the mothers are paying attention to every detail. The fear of hurting the baby with the (badly cut) tip and the risk of injury inside the nose remain present when using physiological serum.

Recently, Unither Pharmaceuticals partnered with Biolane to developed a single-unit dose featuring a more rounded tip that fits perfectly with babys’ nostrils. The “BB dose” guarantees a greater safety of the gesture of the mother with the infant. And avoids to skin him during the use of the physiological serum.

In addition to that, BFS technology enables the manufacture of preservative-free single-unit doses. Preservatives are recognized as potentially harmful to the sensitive mucosae of the eyes, nose, and lungs, so this is an enormous benefit to sensitive patients such as infants. Another advantage of unit-doses is that they are portable and easy to use individually, excellent properties for today’s active lifestyles.

Catalent Provides Commercial Manufacturing Of Lexicon Pharmaceuticals’ Orphan Drug XERMELO™ Following FDA Approval

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Catalent Pharma Solutions recently welcomed Lexicon Pharmaceuticals, Inc.’s announcement that its pioneering orphan drug, XERMELO™, has been approved by the U.S. Food and Drug Administration (FDA). Catalent has been working in partnership with Lexicon since 2007 to develop the drug formulation, and will be manufacturing XERMELO for commercial supply.

XERMELO (telotristat ethyl) 250 mg is a first-in-class orally administered therapy for the treatment of carcinoid syndrome diarrhea in combination with somatostatin analog (SSA) therapy in adults inadequately controlled by SSA therapy.

“We are pleased that the skill and expertise that our scientists have dedicated to this project for ten years has resulted in this pioneering treatment reach the market,” commented Matthew Mollan, Catalent’s General Manager at its Kansas City, Missouri site. “Catalent first partnered with Lexicon when the drug was in Phase I development, and continued to support its manufacture through to supplying launch products.”

“We are proud to have discovered this ground-breaking orphan drug, and our successful collaboration with Catalent means we are now able to make it available for the thousands of patients currently suffering from this condition,” added Lonnel Coats, Lexicon’s president and chief executive officer.

Carcinoid syndrome is a rare condition that occurs in patients living with metastatic neuroendocrine tumors (mNETs) and is characterized by frequent and debilitating diarrhea that often prevents patients from leading active, predictable lives, as well as by facial flushing, abdominal pain, fatigue and, over time, heart valve damage.

Catalent undertook development and manufacture of the drug at its 450,000-square-foot Kansas City facility, where the company provides a range of fully integrated support services, from formulation development and analytical testing, to clinical and commercial scale manufacturing and packaging of various oral dose forms.

Aptar Pharma’s Electronic Lockout Device Approved by EMA

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Aptar Pharma, a world leader in innovative drug delivery systems, is pleased to announce the approval by the European Medicines Agency (EMA) of the first integrated electronic nasal lockout device (e-Lockout) following a multi-year development with Takeda Pharmaceuticals International AG. Aptar Pharma agreed to supply Takeda with its e-Lockout device for a multidose nasal spray version of Instanyl®. The EMA has granted marketing authorization for this multidose nasal spray treatment under the name Instanyl DoseGuardTM.

This represents a major milestone for Aptar Pharma, with the e-Lockout device being the first and only fully integrated electronic nasal drug delivery device to be approved by a U.S. or European regulatory authority.

Already available in unidose and multidose nasal spray versions, Takeda will launch Instanyl® DoseGuard in Europe in several multidose strengths, all using Aptar Pharma’s patented electronic lockout system, which marks another product innovation in the management of breakthrough pain.

Advanced e-Device Technology Ensures Safe Compliance

Instanyl® is a fast-acting nasal opioid approved for relieving breakthrough pain in adult cancer patients already treated with opioids for their usual pain. Breakthrough pain is an additional sudden pain that occurs despite having taken one’s usual pain relieving medicines.
Aptar Phama’s e-Lockout device uses advanced electronic technology to help patient compliance in the treatment of chronic disease. Aptar Pharma’s e-Lockout device is intended to ensure safe patient compliance by limiting the number of doses available during a 24 hour period.

The system’s built-in lock-out mechanism prevents the device from being used for a period of time after a pre-defined number of spray actuations. The electronic display shows the number of priming strokes, the number of doses left in the device and whether the nasal spray is locked or ready for use. The e-Lockout also features a child-resistant cap.

Long-term Strategic Partnership with Takeda

The multi-year supply agreement reinforces a long-standing partnership between Takeda and Aptar Pharma, who currently supplies Takeda with unidose and multidose nasal spray devices for Instanyl® in Europe.

Committed to accompanying pharmaceutical companies throughout their product lifecycle management, Aptar Pharma continues to partner to provide customers with innovative and smart solutions to enable safe, convenient and compliant medication delivery.

“This approval and subsequent product launch underscores Aptar Pharma’s ability to partner with the pharma industry to bring innovative, compliant and safer devices through the regulatory authorization process,” explained Salim Haffar, President, Aptar Pharma. “This is yet another example of Aptar Pharma’s expertise and technology at the heart of a new market launch. This is a significant step in strengthening Aptar Pharma’s credentials in the electronics and connected health markets. We are pleased to be building on our trusted, long-term partnership with Takeda,” he added.

Novartis Access and government of Pakistan sign memorandum of understanding

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Ministry of National Health Services, Regulations and Coordination has signed a Memorandum of Understanding with a multinational healthcare company Novartis to help the poor gain access to treatment of chronic diseases.

Under the MoU, Ministry and Novartis will partner in the delivery of a program called Novartis Access. This program will provide access to a basket of high-quality medicines in the public sector targeting four key non communicable diseases (NCDs)–cardiovascular diseases, diabetes, respiratory illnesses, and breast cancer. These diseases kill one-fifth of Pakistanis between the ages of 30 and 70 years every year.

Products within the Novartis Access portfolio are among the world’s most frequently prescribed medicines for the targeted chronic diseases.

In pilot phase, Novartis Access medicines will be available through selected empanelled hospitals of district Islamabad under the Prime Minister’s National Health Program. The government is committed to making these NOT for SALE drugs available free of charge to patients within the program. The aim is to extend the program to all districts of Prime Minister’s National Health Program over time and work has already started in this regard.

Speaking after witnessing the signing, Saira Afzal Tarar Minister for National Health Services Regulations and Coordination, said: “I am very pleased over the signing of this Memorandum of Understanding with Novartis. Pakistan is grievously affected by the growth of non communicable diseases, and having access to high-quality treatment at low cost is a critical part of our work to lessen the impact of chronic disease in Pakistan. It is part of the Prime Minister’s ambitious plans to make Pakistan a true welfare state.”

The MoU was signed on behalf of Ministry of National Health Services, Regulations and Coordination by Director Prime Minister’s National Health Program Dr. Faisal Rifaq and CEO Novartis Mr. Shahab Rizvi.

This is the beginning of the Novartis Access partnership, and the first treatment against chronic diseases are expected to reach Pakistan soon.

Boehringer Ingelheim Inaugurates World-Class Biopharmaceutical Manufacturing Facility in China

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Boehringer Ingelheim inaugurated its commercial production site for biopharmaceuticals in Zhang Jiang Hi-tech Park of Shanghai (China). The site, with the first-phase investment of more than €70 million, is the first and only biopharmaceutical facility established by a leading multinational active biopharmaceutical manufacturer in China utilizing mammalian cell culture technology. With its global network of biopharmaceutical production sites in Biberach ( Germany), Vienna (Austria), Fremont (USA) and now Shanghai, the contract manufacturing business Boehringer Ingelheim BioXcellence™ is well positioned to fulfil strongly increasing demands of the biopharmaceutical industry for innovative products – both in China and worldwide.

“Our Shanghai facility plays an important role in our globally leading biopharmaceutical contract manufacturing business and embodies our continuous and long-term commitment to China,” said Hubertus von Baumbach, Chairman of the Board of Managing Directors at Boehringer Ingelheim. “With this investment, we expect to have a significant impact on the development of China’s biopharmaceutical industry to ultimately supply innovative medicines to patients following high quality standards.”

Boehringer Ingelheim has made this strategic move with the long-term goal to become a leader for contract development and manufacturing of monoclonal antibodies and recombinant proteins in China. Since 2014, Boehringer Ingelheim China Biopharmaceuticals has been operating its Good Manufacturing Practice (GMP) clinical material supply at 100L and 500L scales. After its inauguration, the facility will operate for clinical and commercial supply on a 2000L single-use bioreactor scale. It is designed to flexibly add additional 2000L single-use bioreactors and fill/finish capabilities to meet increasing market demand.

“As a global leader in biopharmaceutical contract manufacturing, Boehringer Ingelheim has an extraordinarily long history in the field of biotechnology, having been in the industry for over 35 years,” said Dr Luo Jiali, General Manager of Boehringer Ingelheim Biopharmaceuticals (China) Co Ltd. “We offer tailor-made contract development and manufacturing services to the biopharmaceutical industry, providing the entire production technology chain from DNA to the finished product under one roof. With our strong know-how, global network, technology, and international quality standards we can support innovative Chinese and international companies to industrialize their research results.”