Eli Lilly research focuses on patient needs and partnerships

Participatory Partnering

Emily Kramer-Golinkoff, who was  trained as a bioethicist from the University of Pennsylvania, is “the engine behind a multi-level approach to changing the game for the advancement of treatments for patients with certain protein production mutations of Cystic Fibrosis (CF) called nonsense mutations – all while living with the condition 24/7,” according to an article by Joe Kim in Lilly Trial Guide. Kramer-Golinkoff provides funding for many phases of scientific research, advocates for patients with Cystic Fibrosis, participates in clinical research, influences the design of research, connects disparate scientific fields and scientists and inspires action through her public speaking, Kim said.

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Study merges clinical research investigators data from three government databases

Complete Picture

The three primary U.S. government sources of data about clinical research investigators -- National Library of Medicine: ClinicalTrials.gov, FDA: Bioresearch Monitoring Information System (BMIS)and CMS: Open Payments (Sunshine Act) -- provide incomplete and inconsistent data not linked to data in the other databases. While the FDA is aware of the problems, researchers have used the databases to understand investigator demographics and activities.

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FDA steps up in regulating safety concerns

Data-Driven Progress

The U.S. Food and Drug Administration (FDA) has established a new objective for itself: “to become the international leader in medical device safety through better use of big data,” according to an article by Conor Hale in FiercePharma. The agency has made a commitment to quick device safety responses “using real-world data and active surveillance,” Hale explained.

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Psychedelic mushroom component could be used medicinally

Far Out!

Depending on who is administering it and how it is being used, psilocybin, the active compound in hallucinogenic mushrooms, could be reclassified from a drug with no known medical benefit to a drug with the properties of prescription sleeping pills. In a review to assess the safety and abuse of medically administered psilocybin, researchers at Johns Hopkins University made a case for doing just that, possibly opening up possibilities for the psychedelic drug to be used to treat depression and anxiety and help people stop smoking.

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Analyst says blockchain technology could help clinical trials

Blockchain Bonanza

Caron Dhillon, a health care analyst at Results Healthcare, believes that the use of blockchain technology could solve many of the challenges of the clinical trials process, according to an article in Outsourcing Pharma by Melissa Fassbender.

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New company targets CNS disorders

Brain Biopharma

US pharma giant Pfizer and private equity firm Bain Capital have joined forces to create  Cerevel Therapeutics, a new privately held biopharmaceutical company. The new company aims to develop drugs to treat central nervous system (CNS) disorders. In addition to securing $350 million from funds affiliated with Bain Capital Private Equity and Bain Capital Life Sciences, there will be further capital available if needed in the future.

New York-based Pfizer, which will hold a 25 percent stake in Cerevel Therapeutics, is providing a portfolio of pre-commercial neuroscience assets, including three clinical-stage compounds and various pre-clinical compounds to target multiple CNS disorders such as Parkinson’s, Alzheimer’s, epilepsy, schizophrenia and addiction. The new company is designed to provide treatment options in a therapeutic area with urgent unmet needs. Two Pfizer executives – Morris Birnbaum, chief scientific officer of internal medicine, and Doug Giordano, senior vice president of worldwide business development – are part of Cerevel’s board of directors.

According to an article by Alaric Dearment in MedCity News, “The current pipeline, consisting of drugs created by Pfizer, includes three clinical-stage candidates – a D1 partial agonist for Parkinson’s, a GABA 2/3 agonist for epilepsy and a muscarinic M4 positive modulator – and several preclinical ones. Modulation of M4 msucarinic receptors has been investigated as a potential mechanism of action for use in schizophrenia. The preclinical candidates include a dopamine D3 antagonist and a kappa opiate antagonist in candidate-stage development, and an M4 agonist, a PDE4b inhibitor and an LRRK2 inhibitor in lead development.”

Pfizer said that putting the set of neuroscience assets in a company with dedicated focus and expertise in CNS was the optimal next step after halting its own research in CNS in January, according to Pharmaceutical Business Review. After stopping that research, Pfizer announced in June that it had bolstered the funding pool of its venture capital arm by $600 million, one-quarter of which was devoted to neuroscience. As Pfizer senior vice president, internal medicine chief scientific officer Morris Birnbaum explained, “We are excited that Cerevel will continue to develop the Pfizer compounds, contributing to the broad scientific understanding of both Alzheimer’s and Parkinson’s and fulfilling responsibility to patients with these devastating diseases and their families.”

As Bain Capital Life Sciences managing director Adam Koppel stated, “We are dedicated to developing this promising portfolio of assets thoughtfully and will provide the right resources to enable Cerevel to build an experienced leadership team that is committed to developing life changing therapies for patients who are struggling with CNS diseases. The broad portfolio we are investing behind consists of several clinical candidates with unique chemistry to enable specific receptor targeting, which has the potential to drive a differentiated clinical profile.”

New cloud-based Parexel technology provides better patient interactions

Meaningful Data

Leveraging its relationship with Microsoft to assist with drug development, Parexel has introduced the world to several new patient-centric solutions that utilize its Perceptive Cloud platform. The patient-centric solutions for drug development feature mobile and wearable tech to make access to data for better patient interactions during clinical research faster and better, the company explained.

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Milestone procures $80M to fund at-home phase 3 trial of tachycardia rescue nasal spray

Bringing It Home

Etripamil, a “short-acting calcium channel blocker being developed for paroxysmal supraventricular tachycardia, or PSVT, where the heart can beat over 200 times per minute,” is certainly exciting investors, according to an article by Conor Hale in Fierce Biotech. Milestone Pharmaceuticals of Montreal, Canada, has raised $80 million to fund its lead program, an at-home, phase 3 clinical trial of its nasal spray for rapidly reducing super high heart rates.

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Digital health startups need to analyze stakeholders

Cracking the Code

While some forms of high technology succeed using a “move fast and break things” approach, digital health startups have to go deeper into the requirements of the industry to “truly transform healthcare,” according to an article by Paul Yock, a cardiologist, health technology innovator, professor of medicine and bioengineering and founder of the Byers Center for Biodesign at Stanford University. Dr. Yock, writing in Fast Company (www.fastcompany.com/90251795/why-do-digital-health-startups-keep-failing, said that money and buzz are not enough to be successful in healthcare, “a much more complex and regulated industry.”

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Allogene begins Nasdaq trading at $18 per share

Living Drug

Allogene Therapeutics, a clinical stage biotech company specializing in allogeneic CAR-T (AlloCAR T) treatments for cancer, started trading on the Nasdaq Stock Exchange at $18 per share. The company, which launched in April 2018 with a $300 million Series A financing, is expecting to raise $288 million by selling about 16 million shares, reported Mark Terry in BioSpace.

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Veeva announces single app to manage clinical trial data

Cloud Tool

Veeva Systems Inc. -- a leader in cloud-based software for the global life sciences industry -- is offering a new cloud-based application that combines several of its tools. It includes coding, EDC, data cleaning and reporting. Enabling companies to manage clinical trials from build through execution, the new app is designed to achieve a complete and concurrent view of all clinical data within a trial, according to the company.

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FDA suggests heavy fines for not posting clinical trial results online

Transparency 101

The U.S. Food & Drug Administration (FDA) has issued a draft guidance statement proposing heavy fines for pharmaceutical companies and clinical research organizations (CROs) that neglect to post clinical trial results online. According to the statement, the FDA wants to fine organizations that fail to register clinical trials or to report results on to the government database ClinicalTrials.gov as much as $10,000 per day.

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Data Cubed uses gaming technology to facilitate human interaction in clinical trials: Making a Game out of It

Basing its approach on popular computer games, Data Cubed has created a platform that could “increase patient engagement in clinical research,” according to an article by Melissa Fassbender in OutsourcingPharma.com. In 2016 when Data Cubed (D3) was founded, the company took advantage of several trends -- structured big data, mobile technologies, patient-centric care, and social networks, the article explained.

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Forbes contributing writer takes aim at Harvard professor who pans pharma

Disclosure Dilemma

Leading cancer researcher Dr. Jose Baselga, the former chief medical officer of Memorial Sloan Kettering Cancer Center, did not disclose income he received from biopharmaceutical companies in articles that he published in major medical journals and, as a result, resigned from his position. This prompted a scathing response from Dr. Marcia Angell, a senior lecturer at Harvard Medical School and the first woman to serve as editor-in-chief of The New England Journal of Medicine.

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Uninsured People and Clinical Trials

Both ethical and practical issues raise concerns

 How does an uninsured person’s participation in a clinical trial affect the outcome? Does the lack of affordability of healthcare give rise to untenable situations in choosing participants for clinical trials?

 These were some of the issues addressed by George Marzouka, MD, who wrote an article called “Ethical Concerns Arise in Clinical Trial Enrollment as Number of Uninsured and Underinsured Americans Skyrockets” in Medical Bag recently.

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Innovation in Pharma: Is there a problem, and can it be fixed?

In an article in Forbes, healthcare contributing writer Standish Fleming dissected the theories of blogger Kelvin Stott in his recent posts, “Pharma’s broken business model, An industry on the brink of terminal decline.” Stott thinks technology can solve pharma’s problems, while Fleming thinks “but the solution to the innovation crisis, if there is one, lies not in technology but a new business model.”

 First of all, Fleming points to the decline in productivity in pharma, which could reach a no-profit status by 2020. According to Fleming, Stott “diagnoses the problem as a failure of technology and so looks for a science-based solution to the innovation crisis. What he finds provides no way out of the dilemma.”

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Can virtual clinical trials be as good as the real thing?

New Reality

 While the drug testing is real, the recruitment, the screening and the venue are changing the way clinical trials are conducted, reported an article by Barbara Mandark in Undark and reprinted by Fast Company.

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Attacking Alzheimer’s: Human-on-a-chip model could lead to better treatments

Alzheimer’s is a “devastating memory-wasting disease that has continually seen failure after failure in the clinic,” according to an article by Ben Adams in Fierce Biotech. With no cure yet in sight, a medical technology company in Florida has taken a new approach to simulating a human with the condition.

Hesperos, an Orlando-based company that to characterize an individual’s biology with human-on-a-chip microfluidic systems, has garnered National Institutes of Health (NIH) funding for Alzheimer’s “human-on-a-chip” trials. The company received a phase 1 grant from the National Institute on Aging in order to “realistically mimic the biology of Alzheimer’s disease (AD), and the effects of potential new therapies under realistic human physiological conditions,” NIH said.

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Theranos Dissolving: Health startup will pay off creditors from remaining cash and cease to exist

It looks like the end is near for a beleaguered Silicon Valley company. The move comes after a failed attempt by the company to sell itself, during which it reached out to more than 80 potential buyers through Jefferies Group.

Theranos, a blood-testing company embroiled in federal fraud charges against its founder and its COO, is formally dissolving, according to a story in The Wall Street Journal. The company sent an e-mail to shareholders explaining its dissolution and plan to pay out its remaining cash to creditors. The e mail from Chief Executive David Taylor also explained, “Unfortunately, none of those leads to buyers has materialized into a transaction. We are now out of time.” The letter was published by the WSJ.

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Three AI companies take on metabolism and diseases related to aging

AI and Aging

Developing medicines to combat aging-related diseases will be the focus od three organizations in a joint venture, according to an article by Conor Hale in Fierce Biotech. Hale recently reported that Insilico, Juvenescence and the Buck Institute were forming an artificial intelligence (AI)-based venture to expedite drug discovery as it applies to metabolism and aging-related diseases. It seems that the bottom line is only part of the story.

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