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Google has quietly launched a project with one of the nation’s largest nonprofit health systems in which it has gained access to millions of patient records, including names and birth dates, to help deliver more targeted medical treatment.

The initiative, dubbed “Project Nightingale,” gives the tech giant the ability to analyze personal health information compiled by Ascension, a Catholic hospital system that operates in 21 states. It appears to be unusual in that it grants such a large amount of patient data to a third-party technology company without the knowledge of doctors or patients.

Ascension released a statement Monday after details of the partnership were disclosed by the Wall Street Journal. The hospital system said its work with Google (GOOGL) is “HIPAA compliant and underpinned by a robust data security and protection effort and adherence to Ascension’s strict requirements for data handling.”

In recent years, hospitals nationwide have begun entering into contracts with large technology companies to store patient information in their clouds, where it can be analyzed using machine learning tools to help providers gain insight into patients’ health needs and zero in on the most effective treatments for specific conditions. Earlier this year, Mayo Clinic inked a sweeping 10-year partnership with Google to store its data in the company’s cloud and use its tools to analyze clinical information, although it emphasized any patient data shared with Google would be de-identified.

Deciding whether and how to disclose those arrangements to patients, doctors, and the broader public can be tricky for hospitals, because of concerns about their ability to safeguard personal health information. Many hospital information specialists have cited the enhanced security capabilities of large technology companies, along with their artificial intelligence expertise, to justify such arrangements. 

Ascension said its work with Google involves transferring data to the company’s cloud, deploying its G Suite product for collaboration between caregivers and the use of machine learning tools to generate insights that may improve the quality of care, provider satisfaction, and patient safety.

Citing anonymous sources, the Wall Street Journal reported that as many as 150 Google employees already have access to data on tens of millions of patients, and that some Ascension employees have raised questions about the manner in which patient information is being collected and shared.  

The president of Google Cloud, Tariq Shaukat, said in a statement: “By working in partnership with leading healthcare systems like Ascension, we hope to transform the delivery of healthcare through the power of the cloud, data analytics, machine learning, and modern productivity tools — ultimately improving outcomes, reducing costs, and saving lives.”

CAMBRIDGE, Mass. and RESEARCH TRIANGLE PARK, N.C., Nov. 14, 2019 (GLOBE NEWSWIRE) — Sarepta Therapeutics, Inc.,  (NASDAQ: SRPT), the leader in precision genetic medicine for rare diseases, and StrideBio, Inc., a leading developer of novel adeno-associated viral (AAV) based gene therapies, today announced the signing of a collaboration and license agreement to develop in vivo AAV-based therapies for up to eight central nervous system (CNS) and neuromuscular targets. Pursuant to the agreement, Sarepta is granted an exclusive license on selected targets to leverage StrideBio’s novel, structure-driven capsid technology, intended to enhance specific tropism to tissues of interest and evade neutralizing antibodies. The parties also plan to focus on strategies intended to address re-dosing challenges in patients who have received AAV-delivered gene therapy. StrideBio will conduct all investigational new drug (IND) enabling research, development and manufacturing for the first four CNS targets, which are MECP2 (Rett syndrome), SCN1A (Dravet syndrome), UBE3A (Angelman syndrome), and NPC1 (Niemann-Pick). Additionally, Sarepta will have an exclusive option to four additional targets based on StrideBio’s capsid technology.

StrideBio possesses an innovative and proprietary platform that is enabled by a deep knowledge of AAV structure and a unique approach to engineering capsids with novel functions, and was co-founded by Mavis Agbandje-McKenna, Ph.D., and Aravind Asokan, Ph.D., leading scientists in the field of AAV biology and gene therapy. StrideBio has created a growing portfolio of novel AAV capsids evolved in non-human primates that show reduced seroprevalence and potential for improved tropism to targeted tissues.

“With our partnership with StrideBio, Sarepta continues to build on its leadership position in gene therapies to treat rare diseases. We are excited to work with StrideBio and access its innovative AAV platform for next-generation capsids,” said Doug Ingram, Sarepta’s President and Chief Executive Officer. “Our partnership with StrideBio expands our research portfolio by up to eight new targets and, through our strategic partnering approach that has our collaborator lead all IND-enabling research and development, ensures that we gain access to new technology and targets while not distracting Sarepta from its near-term priorities.”   

“We are very excited to initiate this multi-target collaboration with Sarepta, an established leader in the development and commercialization of genetic medicines,” stated Sapan Shah, Ph.D., Chief Executive Officer, StrideBio. “This partnership will provide significant resources and expertise to enable StrideBio’s continued rapid expansion of our research and manufacturing platform, as well as accelerate the development of AAV gene therapies for multiple rare disease targets. We are looking forward to working together with Sarepta to bring novel treatments to patients as quickly as possible.”

Terms of Agreement

Under the terms of the agreement, StrideBio will be responsible for AAV capsid development, non-clinical development and manufacturing of preclinical candidates to be selected for advancement into clinical studies. The parties will also share early clinical development activities for selected targets, with Sarepta responsible for late stage development and commercialization. A total of four initial targets are specified under the collaboration. StrideBio will receive a $48 million upfront payment in the form of cash and Sarepta stock, in addition to significant future development, regulatory and commercial milestones for the four programs. StrideBio will also receive royalties on worldwide net sales of any commercial products developed through the collaboration. Sarepta has also obtained an exclusive option to expand the collaboration to include up to an additional four targets with an upfront payment of up to $42.5 million along with future downstream milestone payments, while StrideBio has an option to obtain co-development and co-commercial rights to one of the collaboration targets. In addition, Sarepta has made a commitment to invest in StrideBio’s next financing round. Further financial terms were not disclosed.

About Sarepta Therapeutics

Sarepta is at the forefront of precision genetic medicine, having built an impressive and competitive position in Duchenne muscular dystrophy (DMD) and more recently in gene therapies for Limb-girdle muscular dystrophy diseases (LGMD), MPS IIIA and other CNS-related disorders, totaling over 20 therapies in various stages of development. The Company’s programs and research focus span several therapeutic modalities, including RNA, gene therapy and gene editing. Sarepta is fueled by an audacious but important mission: to profoundly improve and extend the lives of patients with rare genetic-based diseases. For more information, please visit www.sarepta.com.

About StrideBio, Inc.

StrideBio, Inc is a gene therapy company focused on creating and developing novel adeno-associated viral (AAV) therapies for rare diseases.  Our STRucture Inspired DEsign approach holds the potential to generate unique AAV capsids with improved characteristics including potency, tissue tropism, and ability to evade pre-existing antibodies.  This powerful new approach has broad application, enabling gene addition, gene silencing and gene editing modalities for a wide range of diseases, including rare genetic diseases. StrideBio is headquartered in Research Triangle Park, NC.  Current investors include Hatteras Venture Partners, Takeda Ventures, UCB Ventures and Alexandria Real Estate Equities, Inc.  For more information, please visit www.stridebio.com.

Forward-Looking Statements

This press release contains “forward-looking statements.” Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as “believes,” “anticipates,” “plans,” “expects,” “will,” “intends,” “potential,” “possible” and similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements regarding the potential benefits of the collaboration between Sarepta and StrideBio, including enhancing specific tropism to tissues of interest, evading neutralizing antibodies, granting Sarepta access to new technology and targets, providing significant resources and expertise to enable StrideBio’s continued rapid expansion of its research and manufacturing platform, and accelerating the development of AAV gene therapies for multiple rare disease targets; the potential of StrideBio’s portfolio to improve tropism to targeted tissues; the parties’ plan to focus on strategies intended to address re-dosing challenges in patients who have received AAV-delivered gene therapy; the terms of the agreement between Sarepta and StrideBio, including expected and future payments, future development, regulatory and commercial milestones, future royalties, options and Sarepta’s commitment to invest in StrideBio’s next financing round; and Sarepta’s mission to profoundly improve and extend the lives of patients with rare genetic-based diseases.

These forward-looking statements involve risks and uncertainties, many of which are beyond Sarepta’s control. Known risk factors include, among others: the expected benefits and opportunities related to the collaboration between Sarepta and StrideBio may not be realized or may take longer to realize than expected due to challenges and uncertainties inherent in product research and development; in particular, the collaboration may not result in any viable treatments suitable for commercialization due to a variety of reasons, including any inability of the parties to perform their commitments and obligations under the agreement, the results of research may not be consistent with past results or may not be positive or may otherwise fail to meet regulatory approval requirements for the safety and efficacy of product candidates, possible limitations of company financial and other resources, manufacturing limitations that may not be anticipated or resolved for in a timely manner, and regulatory, court or agency decisions, such as decisions by the United States Patent and Trademark Office with respect to patents that cover Sarepta’s product candidates; and those risks identified under the heading “Risk Factors” in Sarepta’s most recent Annual Report on Form 10-K for the year ended December 31, 2018 and most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by the Company which you are encouraged to review.

Any of the foregoing risks could materially and adversely affect the Company’s business, results of operations and the trading price of Sarepta’s common stock. For a detailed description of risks and uncertainties Sarepta faces, you are encouraged to review Sarepta’s 2018 Annual Report on Form 10-K and most recent Quarterly Report on Form 10-Q filed with the SEC as well as other SEC filings made by Sarepta. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release. Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof.

The European Commission approved the world’s first Ebola vaccine. The vaccine is manufactured by Merck and has a trade name of Ervebo.

“The European Commission’s marketing authorization of Ervebo is the result of an unprecedented collaboration for which the entire world should be proud,” said Kenneth C. Frazier, Merck’s chairman and chief executive officer. “It is a historic milestone and a testament to the power of science, innovation and public-private partnership.”

Frazier added, “After recognizing the need and urgency for an Ebola Zaire vaccine, many came together across sectors to answer the global call for outbreak preparedness. We at Merck are honored to play a part in Ebola outbreak response efforts and we remain committed to our partners and the people we serve. We also look forward to continuing to work with the FDA and the African countries on their regulatory reviews over the coming months and with the World Health Organization on vaccine prequalification, which will help broaden access to this important vaccine for those who need it most.”

Ebola virus causes a hemorrhagic illness that begins with common symptoms of fever, headache, muscle pain and fatigue, but can move to severe internal bleeding and death. It spreads via direct contact with bodily fluids of someone who has the diseases or died from it. The average fatality rate is 50%.

Ervebo is currently being used under a “compassionate use” program in the eastern Democratic Republic of Congo (DRC), where there has been the second-largest Ebola outbreak on record. Since August 2018, more than 3,000 people in the country have tested positive for Ebola and more than 2,000 people have died of it. In the last year, more than 250,000 people have been dosed with the Merck vaccine in DRC.

Another Ebola vaccine being developed by Johnson & Johnson, is being administered to 50,000 people in Goma, a city with two million people on the Rwandan border of eastern Congo. The J&J vaccine requires two injections eight weeks apart, while the Merck vaccine only requires a single shot.

“The introduction of a second vaccine is not meant to replace [Merck’s] vaccine, but to complement it and hopefully provide us with an additional tool in the fight against future Ebola outbreaks,” said John Johnson, who is leading the project for Médicins Sans Frontières (MSF, or Doctors Without Borders), reported Physicians Weekly.

The Merck vaccine is being distributed in what is called “ring vaccination.” This strategy focuses on determining who is most likely to infect or have been infected and vaccinating the most susceptible in “rings” in order to isolate the virus and slow and prevent its transmission.

The U.S. Food and Drug Administration (FDA) is currently reviewing Merck’s application for the vaccine, with a target action date in the first quarter of 2020. Forbes notes that, “Ervebo won’t be a blockbuster money maker for Merck. Vaccines typically are far from the most profitable products for pharmaceutical companies, and the Ebola virus is far from common in higher income countries.”

As a result, the World Health Organization and other world health organizations will have to work on ways to support this and other similar vaccines.

Despite some of the economic obstacles to vaccines, they provide a significant return on investment. A 2016 Johns Hopkins University study found that for every dollar invested in vaccination in the 94 lowest-income countries in the world, $16 were expected to be saved in healthcare costs, lost wages and lost productivity. Taking into consideration healthier and longer lives and the long-term burden of disability, the net return increases to $44 per $1 invested.

At this point, Merck’s vaccine is the only one tested during an outbreak, where it has shown high effectiveness in preventing infection. It was first patented in 2003 and has been used in DRC and in 2015 during an Ebola outbreak in Guinea.

“This is a vaccine with huge potential,” said Seth Berkley, chief executive officer of Gavi, the Vaccine Alliance based in Geneva, Switzerland. “It has already been used to protect more than 250,000 people in the DRC and could well make major Ebola outbreaks a thing of the past.”

While CROs are gung-ho on some new technologies to manage trials, they still rely heavily on paper-based systems, a new survey shows.

Electronic data capture (EDC), electronic trial master files (eTMF) and clinical trial management systems (CTMS) are increasingly in use, but half of CROs responding to the survey said they still use paper to manage information exchange with sites.

Because most sites still store their trial data in regulatory binders rather than electronic systems, CROs’ ability to share information with their sites electronically is limited, the survey says. Most respondents, 96 percent, said they experience significant challenges with exchanging information with sites, according to Veeva’s 2019 Unified Clinical Operations Survey Report.

Seventy-one percent of respondents had issues with tracking and reporting paper documents, 59 percent with misfiled or missing documents and 48 percent with manual document exchange.

Attempting to overcome these hurdles, 93 percent of respondents said they use a standalone EDC system, 77 percent use an eTMF system, 71 percent a randomization and trial supply management (RTSM) system, and 70 percent a clinical trial management system (CTMS).

The number of CRO respondents using an eTMF has tripled since 2014, with 60 percent of CROs now using a purpose-built eTMF application. Over the past five years, eTMF has steadily replaced CTMS, according to the survey.

EDC, RTSM, and CTMS systems in use by CROs are all up from 2017, with EDC, which is the most heavily used, showing a 7 percentage point increase.

Since 2017, RTSM saw a 19 percentage point increase due to the complexity of protocol design, according to the survey, and CTMS use increased 12 percentage points as CROs aimed to reduce costs and improve study quality, the survey said.

Although new types of software can be used for many different parts of trials, they are particularly effective in study startup.

Study startup is actually slower today than it was a decade ago, and CROs are working hard to develop apps and purpose-built software to expedite the time that it takes to bring a drug to market. According to the survey, 86 percent of trials experience a study startup delay.

Only about half of CROs surveyed use CTMS, eTMF, or internally developed applications for study startup. For example, 28 percent of CROs use custom study startup applications, more than twice as many as sponsors.

Tools used to manage study startup processes are still dominated by Excel, at 78 percent. A close second to manage study startup was CTMS at 51 percent, followed by using eTMF applications at 44 percent.

To improve study startup, 60 percent of CROs say it would be best to reduce the use of spreadsheets and manual processes.

By Colin Stoecker

MORRISVILLE, N.C. and NEW YORK, Nov. 12, 2019 (GLOBE NEWSWIRE) — Syneos Health^®(Nasdaq:SYNH), the only fully integrated biopharmaceutical solutions organization, and AiCure, an AI and advanced data analytics company, today announced a novel strategic partnership to drive faster, smarter trials to optimize patient engagement. This collaboration amplifies a pre-existing relationship and responds to increasing customer demand for digital solutions that change behaviors and improve patients’ lives.

The collaboration leverages AiCure’s proprietary intelligent software and AI-driven insights, and Syneos Health’s behavioral insights-driven product development model, to capture, analyze, and predict patient behaviors influencing clinical trial adherence.

“The AiCure platform aligns with our unique product development model that harnesses behavioral insights to unlock real world experiences,” said Alistair Macdonald, Chief Executive Officer, Syneos Health. “By deploying the AiCure platform to our investigative sites, we create insights-enriched patient communities to advance clinical research. This collaboration reinforces our Dynamic Assembly philosophy where we join forces with the latest and most agile data and technology partners to address our customers’ needs.”

“Syneos Health’s first mover participation in our AiCure Partnerships for Excellence (AiPEX™) program demonstrates the Company’s dedication to the implementation of innovative technologies in clinical research. This full-service, collaborative delivery of AI to improve engagement, medication exposure, and achieve 360° access to clinical trial subjects, sites, and study insights represents our shared pursuit of therapeutic excellence,” said Adam Hanina, Chief Executive Officer of AiCure. “Syneos Health’s scale as a leading global CRO, and the largest Contract Commercial Organization (CCO), will enable valuable customer engagements and expands our reach with biopharmaceutical companies of all sizes.”

AiCure provides a clinically validated, scalable, patient-centric technology platform which can see, hear, and understand how people respond to treatment. The platform identifies adherence patterns and variation in site and participant engagement, surfacing the opportunity to modify behaviors that increase compliance to improve the chances of overall trial success. As opposed to antiquated self-reporting measures, patients use a smartphone application to intuitively guide and confirm proper administration in real-time. AiCure’s computer vision algorithms verify the correct participant is taking the right medication and confirms ingestion.

Syneos Health will have access to AiCure’s digital biomarker assessments, evaluating facial, verbal, speech, and movement variables to objectively study known clinical endpoints. AiCure’s unique technology naturally inserts into the dosing interaction with the study subject while keeping all identifiable information secure and protected. The company, sponsors, and site staff will also have access to dashboards with real-time, actionable, and predictive data to improve decision making, facilitate timely interventions, and achieve greater clinical trial operational efficiencies.

About Syneos Health
Syneos Health (Nasdaq:SYNH) is the only fully integrated biopharmaceutical solutions organization. Our company, including a Contract Research Organization (CRO) and Contract Commercial Organization (CCO), is purpose-built to accelerate customer performance to address modern market realities. Created through the merger of two industry-leading companies – INC Research and inVentiv Health – we bring together approximately 24,000 clinical and commercial minds with the ability to support customers in more than 110 countries. Together we share insights, use the latest technologies, and apply advanced business practices to speed our customers’ delivery of important therapies to patients. To learn more about how we are shortening the distance from lab to life^® visit syneoshealth.com or subscribe to our podcast.

About AiCure
AiCure uses artificial intelligence to see, hear, and understand how people respond to treatment across clinical trials and patient care. Clinically proven to accurately measure and modify patient behavior, AiCure’s technologies keep patients engaged and optimized to treatment, as well as assess treatment effectiveness.

Founded in 2010 and funded by the National Institutes of Health (NIH) and leading institutional investors, AiCure has more than 100 patents filed and works with global clients in over 25 countries. AiCure is globally recognized and a recipient of the Scrip Award, AI 100, and Digital Health 150. For more information, please visit www.aicure.com and follow us @AiCureMed.

A third of pharma companies will be spending more than half of their marketing budget on digital channels over the next three years, according to a new report.

The Indegene Pharma Marketer 2020 report predicted a shift in spend to emerging new channels of communicating and engaging with healthcare providers.

On average, the report forecast that the number of organisations already spending more than 20% of their budget on digital marketing channels will increase by more than 70% over the next six years.

According to life sciences services firm Indegene, the budget increase pattern highlights how marketing organisations across the globe are undergoing a structural change.

While brand portals, KOL webinars and social media are frequently used channels, third party websites and online journals in US and tele-detailing in Europe/Asia received the highest rating for “white space marketing” – where there is no defined market for a given product.

Personalising content has also emerged as the most effective strategy for markets, with respondents confirming this has generated maximum returns on digital investments, addressing the challenges of reduced face-to-face meeting time for sales reps.

Besides personalisation, geographical nuances of content strategy in the US included retargeting and localisation of content; European companies focused on adapting content to device compatibility and promotion of content through infographics while in Asia, repurposing content into videographics and content promotion through infographics remained an over-arching priority.

Digital Analytics seemed to be the most popular technology choice among pharma marketers with close to 65% stating that it has already been implemented.

Campaign Automation is the next most widely implemented technology, with over 60% of respondents already using them within their organisation. The companies however scored poorly on tools deployment for Social Listening, which has a significant bearing on collecting real world data.

For a complete insight into the survey, download a copy of the full report here.

The government has given approval for setting up four medical device parks with a view to support Make in India initiative and provide world-class products at affordable price for treatment. The four parks will be set up in Andhra Pradesh, Telangana, Tamil Nadu and Kerala, sources said, adding that Uttarakhand and Gujarat have also approached the Centre for a go-ahead for such parks.

These parks will provide necessary infrastructure, where companies can easily plug and play, sources said.

This will not only cut import bill but will also help in easy access to standard testing facilities and reduce cost of production, they said.

The project of Andhra Pradesh Medtech Zone for creation of Common Facility Centre (CFC) for Superconducting Magnetic Coil Testing and Research was given in-principle approval recently.

The scheme proposes to provide Rs 25 crore or 70 percent of the project cost of setting up of CFCs, whichever is less, for creation of common facilities in any upcoming park.

According to estimates, the medical devices retail market in the country is of around Rs 70,000 crore. The domestic medical devices industry is very small even though India is the fourth largest market in Asia.

India is largely an importer of medical devices, with domestic industry accounting for about 2 percent of the global industry which stands at USD 250 billion, as per the estimates.

We’ve all heard the stories of deliberate data fraud in clinical trials: the investigator who created fictional subjects, the lab technician who duplicated test data, the coordinator who altered records. What makes people think they can get away with it?

In most cases, data errors are the result of ignorance of procedures or sloppiness in practices that can be corrected with improved training and oversight. But left unattended, an error-tolerant environment can open the door to more serious problems.

Two experts shared their personal horror stories with attendees at the MAGI Clinical Research Conference West last week, suggesting sites focus on catching problems when they are minor before they snowball and turn into flagrant research misconduct.

“These things start out small, but they can grow,” said Paula Brust, a quality and assurance auditor at QA Partners. “They can easily become something more significant.”

“Deal with things when they start going wrong,” agreed Sara Meeder, director of human research participant protections at the Maimonides Medical Center. “Otherwise, you will be in a terrible mess.”

The evolution of misconduct goes through three stages, Brust said. In the “innocent ignorance” stage, staff may make mistakes like backdating consent signatures or discarding source documents after transcription. The “surprising sloppiness” stage features problems like forgetting to obtain consent, estimating data and taking short cuts.

If these behaviors aren’t nipped in the bud, they become common practice and may lead some individuals to take advantage, leading to the “malicious malfeasance” stage, in which legitimately obtained data may be changed, undesirable data may be omitted, or data may be entirely fabricated.

Fraud often is not detectable without checking multiple sources for the data, particularly sources for pathology reports and endoscopy procedures, Brust said.

Brust told the story of a 45-site study for which she was project manager. The coordinator at one site was found to be manipulating and fabricating data, with the investigator signing off on it. The errors started out simply, Brust said, with monitoring visits cancelled at the last minute, or patients refusing to schedule lab tests and histology tests not being performed per the protocol.

This seeming lack of attention to detail quickly turned to misconduct when the coordinator took a biopsy sample from one subject and split it to create an additional record for a fictional subject. Ultimately, it was discovered that four of the study’s seven patients did not actually exist.

But even less elaborate misconduct can damage a trial’s integrity, such as changing entry data to qualify subjects, stretching of entrance criteria or coaching patients on their diary entries.

All the warning signs will be there if the trial coordinator or site manager knows where to look. Brust cites the following red flags:

• Missing documents;
• Incomplete case report files;
• Frequent appointment changes;
• Notes entered out of chronological order;
• Informed consent documents with similar patient signatures;
• Photocopied source documents with no original;
• Data that is too consistent from patient to patient;
• No mistakes made or corrected;
• Drug containers returned in pristine condition; and
• Perfect protocol compliance.

Brust advised monitors to be vigilant for these practices and to watch for attempts by the PI or trial staff to limit access to study charts and records.

Difficulties with principal investigators also can signal problems. If the PI has only minimal involvement in the study or a poor working relationship with site staff, Brust said, it pays to look into the reasons why and the resulting consequences.

“Observe the relationship,” Brust advised monitors, “because it could be bad, or if it’s too good that is also a sign something is wrong.”

Meeder told of one study in which a Spanish-speaking patient was enrolled without being provided with a Spanish-language informed consent form. When questioned, the coordinator said she had provided the “gist” of the document for the patient’s family. Meeder reported the problem to the PI, only to be told she was “overreacting.”

Ultimately, it is up to trial personnel to use their common sense and to be able to pick out and escalate innocent ignorance issues before they turn into flat-out fabrication.

“Be brave in clinical research,” said Meeder. “Because we protect research participants, you have to stand up and say something when things go wrong.”

By Colin Stoecker

The U.S. Food and Drug Administration (FDA) approved Celgene and Acceleron Pharma’s Reblozyl for the treatment of anemia in adult patients with beta thalassemia who require regular red blood cell (RBC) transfusions. The approval marks the first approved treatment in the United States for this condition.

The approval also marks the first for Acceleron, which sent shares up nearly 7% in afternoon trading. Reblozyl, an erythroid maturation agent, was approved under Fast Track Designation and the drug received Orphan Drug designation.

With the approval, Reblozyl (luspatercept-aamt) becomes a new therapeutic class for beta thalassemia patients. Reblozyl works by regulating late-stage red blood cell maturation to help patients reduce their RBC transfusion burden. The drug is not indicated for use as a substitute for RBC transfusions in patients who require immediate correction of anemia, Celgene stressed in its Friday afternoon announcement.

Takeda Pharmaceutical will unload a portfolio of over-the-counter and prescription pharmaceutical products to Germany-based STADA Arsneimittel AG for$660 million as the company continues to pare down debt from its $62 billion acquisition of Shire.

The portfolio of drugs sold to STADA is for products exclusively sold in Russia, Georgia and “a number of countries from within the Commonwealth of Independent States” that forms part of Takeda’s Growth & Emerging Markets Business Unit. The countries in the Commonwealth are Armenia, Azerbaijan, Belarus, Kazakhstan and Uzbekistan.

The acquisition of Shire placed about $30 billion of debt on the back of Takeda. The company has been looking to pare down that debt as it adjusts to the new size and scope of its portfolio from the Shire deal. Since the Shire acquisition, Takeda has been looking to sell off about $10 billion worth of non-core assets. In July, BioSpace reported that Takeda had sent out information packages of the assets up for sale to potential buyers, including smaller pharma companies and private equity firms. STADA was among those companies that Takeda was allegedly wooing at the time.

The portfolio to be divested to STADA includes over-the-counter vitamins and food supplements, as well as select products within the cardiovascular, diabetes, general medicine and respiratory therapeutic areas, Takeda said. The portfolio’s growth is driven by sales of products such as Cardiomagnyl, and other strong regional brands, the company added. Last year, the portfolio of products sold to STADA. Additionally, Takeda said it anticipates that approximately 500 employees supporting the divested assets will be given the opportunity to transition over to STADA when the deal is finalized. As part of the deal, Takeda and STADA will enter into manufacturing and supply agreements under which Takeda will continue to manufacture and supply the products to STADA.

“Takeda remains committed to the emerging markets, Russia and the countries included in this agreement. We will continue to increase patient access to our portfolio of highly innovative medicines across this region through our commercial activities and access to medicines program,” Ricardo Marek, president of Takeda’s Growth & Emerging Markets Business Unit, said in a statement. “As we execute on our divestiture goals, we continue to work to ensure that each transaction aligns with Takeda’s values. We are confident that STADA is well placed to provide patients with uninterrupted access to the divested products – a top priority for Takeda – and anticipate most of the employees supporting the divested assets will be given the opportunity to transition over to STADA once the divestiture is completed.”

This week’s divestiture marks Takeda’s fourth divestment transaction in the past six months, the company said. In addition to reducing debt, the divestitures allow the company to remain focused on its five key business areas of gastroenterology, rare diseases, plasma-derived therapies, oncology and neuroscience. Last month, Takeda sold off non-core assets to Acino for $200 million. The assets that made up that sale are marketed in countries across the Near and Middle East, as well as parts of Africa. Combined, the portfolio of products to be divested to STADA and Acino generated revenues of approximately $300 million last year, Takeda noted. Other divestitures include the sale of Xiidra (lifitegrast ophthalmic solution) 5% product to Novartis. That deal was for $3.4 billion up front in cash and up to an additional $1.9 billion in potential milestone payments. Also, the company sold TachoSil to Ethicon for $400 million in May.

Costa Saroukos, Takeda’s chief financial officer, said the sale of assets to STADA is the “latest step in Takeda’s effort to simplify our portfolio” and continue to invest in its key business areas.

“We are making strong progress towards executing our strategy and delivering enhanced value for patients and Takeda shareholders,” Saroukos said in a statement.