Thu.Jan 19, 2023

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3 biotech executives on the year ahead: deals, drug pricing and the down market

Bio Pharma Dive

“What we’re experiencing now is the aftershock of the party that went on for the last three years,” said Ovid CEO Jeremy Levin in a BioPharma Dive panel that included former Sage head Jeff Jonas and biotech entrepreneur Greg Verdine.

Marketing 334
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US FDA grants Fast Track status for Avidity’s AOC 1020 to treat FSHD

Pharmaceutical Technology

The US Food and Drug Administration (FDA) has granted Fast Track designation for Avidity Biosciences’ AOC 1020 to treat facioscapulohumeral muscular dystrophy (FSHD). AOC 1020 has been designed for the treatment of the underlying cause of FSHD, which is caused by the abnormal expression of a gene known as double homeobox 4 or DUX4. This DUX4 protein abnormal expression leads to modifications in gene expression in muscle cells which are associated with progressive muscle function loss in FSHD pat

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Editas, in next restructuring step, to sell cell therapy work to Shoreline

Bio Pharma Dive

Shoreline will acquire a preclinical NK cell therapy Editas has been developing, as well as a license to use the CRISPR biotech’s gene editing technology.

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Sun Pharma to buy Concert Pharmaceuticals for $576m

Pharmaceutical Technology

Sun Pharmaceutical Industries has signed a definitive agreement to buy all outstanding shares of Concert Pharmaceuticals, in a deal valued at $576m. Under the deal terms, the company will buy all shares of Concert common stock through a tender offer for $8.00 per share in cash upfront payment. Additionally, the stockholders of Concert will receive a contingent value right (CVR), based on the net sales milestones of deuruxolitinib, entitling them to up to $3.50 per share of common stock in cash.

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The New Age of Decentralized Clinical Trials

White paper that delves into the complex topic of Decentralized Clinical Trials and how to master them within the confines of FDA Regulations

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Sun Pharma to buy Concert, pick up experimental hair loss drug

Bio Pharma Dive

Sun intends to submit the drug, called deuruxolitinib, for U.S. approval in the first half of this year, following in the footsteps of similar therapies from Pfizer and partners Eli Lilly and Incyte.

Drugs 141
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D&C Rules need revamp as there is no clause of compensation for ADRs: regulatory experts

AuroBlog - Aurous Healthcare Clinical Trials blog

Taking into account the four main components of regulator, industry, trade and patient/consumer in a medicine value supply chain, the Drugs and Cosmetics (D&C) Rules, 1945 and D&C Act, 1940 needs an urgent revamp as there is no clause of compensation for ADRs in patients, according to drug regulatory experts.

Cosmetics 150

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This Biomarker Can Foreshadow Early Alzheimer’s Years Before Symptoms

AuroBlog - Aurous Healthcare Clinical Trials blog

A simple blood test could one day diagnose a rare inherited form of Alzheimer’s disease, decades before symptoms of memory loss even appear. Researchers in Europe and the United Kingdom have now shown that cases of early-onset Alzheimer’s are associated with increasing levels of a biomarker in blood plasma.

Research 130
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Cell therapies in AML inch closer to market while facing challenges

Pharmaceutical Technology

Acute myeloid leukemia (AML) is part of a market of blood malignancies that commercial cell therapies have not managed to penetrate yet. While allogeneic hematopoietic stem cell transplants (HSCT) provide long-term benefit to a subset of AML patients, other cell therapy modalities such as natural killer (NK) cells or chimeric antigen receptor (CAR)-T cells have failed to show conclusive benefit in late-stage clinical trials.

Marketing 130
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Roche says Tecentriq immunotherapy reduces risk of liver cancer recurrence

Bio Pharma Dive

While the company did not reveal specific study data, the results could help Tecentriq become the first drug approved for adjuvant liver cancer treatment.

Drugs 141
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A new study links social media use to changes in teen brains. Is that a bad thing?

STAT News

Today’s teens and tweens have never known a world without social media. There are still a lot of open questions about how sites like TikTok and Instagram may shape their development — and stories focusing on the potential negative impacts of social media tend to dominate the news. But a recent study published in JAMA Pediatrics is the latest in a growing body of research that suggests the relationship that young people have with social media is too complicated to be categorized as

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Roles and Responsibilities of Specialized Clinical Supply Experts

When selecting a clinical supply provider, consideration often focuses upon the manufacturing, packaging, storage and distribution capabilities available that will, at face-value, be sufficient to meet the needs of the sponsor and their trial. However, there are human-based and knowledge-driven factors that are often overlooked that go beyond these basic physical capabilities and are integral to the development and delivery of high performing clinical supply chains.

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Thinx settled a lawsuit over chemicals in its period underwear. Here's what to know

NPR Health - Shots

The lawsuit says Thinx underwear, long marketed as safe and sustainable, contains harmful chemical substances known as PFAS. The company denies those allegations but will pay up to $5 million.

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Grand Rounds Ethics and Regulatory Series January 13, 2023: Ethical Considerations When Vulnerable Populations are Subjects in Pragmatic Trials (Emily A. Largent, JD, PhD, RN)

Rethinking Clinical Trials

                                           Speakers Emily A. Largent, JD, PhD, RN Emanuel & Robert Hart Assistant Professor of Medical Ethics and Health Policy Department of Medical Ethics and Health Policy University of Pennsylvania Perelman School of Medicine Slides Keywords Ethics, Vulnerable Populations Key Points Vulnerability guidelines and regulations often refer to groups such as pregnant people, children, and prisoners.

Trials 130
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Is it time for a reality check on rapid COVID tests?

NPR Health - Shots

At-home rapid tests have become a staple of COVID-19 precautions, but some experts worry that people are relying too much on these tests and that's creating a false sense of security.

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Listen: How blockbusters get made, new vaccines for RSV, and mRNA’s Q score

STAT News

Where do new drugs come from? Does science need billionaires? And do normal people care about messenger RNA? We cover all that and more this week on “The Readout LOUD,” STAT’s biotech podcast. Journalist Nathan Vardi joins us to talk about his new book delving into the race to develop the lifesaving cancer drug now called Imbruvica, involving a Scientologist CEO and secretive investor seeking redemption after the worst trade of his life.

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The New Age of Decentralized Clinical Trials

This new white paper defines and details the impact of Decentralized Clinical Trials on the Pharmaceutical industry and how the impact can be measured along with steps companies can take to ensure adoption.

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Therapy by chatbot? The promise and challenges in using AI for mental health

NPR Health - Shots

Some companies and researchers think smart computers might eventually help with provider shortages in mental health, and some consumers are already turning to chatbots to build "emotional resilience.

Research 129
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STAT+: For only the second time, a big drugmaker offers a warranty on a medicine

STAT News

In a bid to blunt competition and address rising drug costs, Sanofi is offering a warranty that will cover the cost for any hospital if a specific medicine fails to work, marking only the second time a major pharmaceutical company has taken such a step. In this instance, Sanofi designed a warranty program for its Cablivi medication, which is used to treat aTTP, a rare, life-threatening autoimmune blood disorder that is considered a medical emergency.

Medicine 111
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CBM looks to boost viral vector manufacturing capabilities in cell line deal

BioPharma Reporter

The unprecedented rise in approvals for new cell and gene therapies, and the increasing industry need for cell lines to support the production of therapeutics, has spurred US CDMO, the Center for Breakthrough Medicines (CBM), to act

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In a post-Dobbs world, pathologists who study pregnancy loss walk a thin line between medicine and the law

STAT News

When Mana Parast examines a placenta, she knows she may never find the answers she seeks. She’s hunting for clues — strips of dead tissue that signal autoimmune disease, white blood cells in the lining of the umbilical cord that point to an infection, thickening that could suggest blood wasn’t flowing freely to the fetus. She wants to be able to tell a parent what she thinks went wrong.

Medicine 111
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Accelerating Clinical Supply Through Integrated Drug Development

As the development pipeline for new drugs continues to grow, biopharmaceutical companies are re-evaluating how to best manage and balance resources across an increasing number of development projects and complex clinical trials. There are two approaches that can be used to speed a drug from development to clinic faster: timeline compression and parallel processing, but only one that considers the benefits of integrating clinical supply into the overall drug development process.

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Amvuttra recommended by NICE for amyloidosis

Pharma Times

Therapy is among the first to receive a positive draft recommendation under streamlined pilot process

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‘Hot mess’: Abortion pills at pharmacies could face legal quagmires, especially in restrictive states

STAT News

WASHINGTON — Federal regulators’ green light for pharmacists to dispense abortion pills is crashing into legal questions and simmering court battles. The Food and Drug Administration earlier this month removed a longtime restriction that only doctors could dispense mifepristone, which is approved for abortions up to 10 weeks. The move opens the door for pharmacists to supply the drugs and shores up protections for mail orders, which have become an important channel for abortion acc

Pharmacy 111
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High frequency brain wave patterns in the motor cortex can predict an upcoming movement

Medical Xpress

Nicholas G. Hatsopoulos, Ph.D., Professor of Organismal Biology and Anatomy at the University of Chicago, has long been interested in space. Specifically, the physical space occupied by the brain.

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STAT+: Will the European Union give vouchers to pharma to develop badly needed antibiotics?

STAT News

Amid rising concern over antibiotic resistance, a battle is brewing in Europe over a proposal to offer vouchers to companies as an incentive to develop new treatments. The controversial idea has already raised objections from more than a dozen members of the European Union. The basic premise is straightforward. A company that wins regulatory approval for a novel antibiotic gets a voucher that can then be used to extend for one year the exclusive rights to the results of clinical trial data for a

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How Machine Learning Drives Clinical Trial Efficiency

Clinical trial data management is increasingly challenging as studies grow in complexity. Quickly accessing and analyzing study data is vital for assessing trial progress and patient safety. In this paper, we explore real-time data access and analysis for proactive study management. We investigate using adverse event (AE) data to monitor safety and discuss a clinical analytics platform that supports collaboration and data review workflows.

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Vaxxas announces CEPI partnership to advance needle-free mRNA vaccine patches

pharmaphorum

Biotech Vaxxas has announced a partnership agreement with the 2017-established Coalition for Epidemic Preparedness Innovations (CEPI), in order to advance the development of Vaxxas’ needle-free vaccine-patch delivery technology with preclinical testing of an mRNA vaccine patch. CEPI is a partnership between public, private, philanthropic, and civil organisations to develop vaccines against future epidemics and pandemics.

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Opinion: Upcoding: one reason Medicare Advantage companies pay clinicians to make home health checkups

STAT News

At the start of 2023, an estimated 2.5 million Americans age 65 and older began using Medicare Advantage programs. Some made this choice in response to aggressive marketing campaigns. This brings the total enrollment of Medicare Advantage plans to nearly 31 million. One unexpected “benefit” of these plans is an offer by the insurance company sponsoring the plan to send a nurse or physician’s assistant, often from a startup company, to an individual’s home.

Nurses 105
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Tiny beetle causes 'Christmas Eye' agony in Australia

Medical Xpress

A rare and agonizing affliction dubbed "Christmas Eye", caused by the toxic secretions of a tiny native beetle, has re-emerged to torment residents in a remote part of southeastern Australia.

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With Gates Foundation backing, a health tech company aims to speed diagnosis of some neglected tropical diseases

STAT News

As it looks to curb the spread of infectious diseases, the Gates Foundation is lending its support to an effort to use artificial intelligence to make it easier for health providers in India and Nigeria to quickly diagnose certain conditions. The funding will support New York-based clinical decision support company VisualDx, which began as a collection of images to allow non-dermatologists to better identify skin conditions.

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The New Clinical Trial Supply Chain: Resilient, Flexible, and Patient-Centric

The global landscape of clinical trials is rapidly changing as studies become more complex. An increasing number of sponsors are seeking enhanced flexibility in their supply chains to address a variety of clinical supply challenges, including patient demand and reducing delays. Demand-led supply and direct-to-patient distribution are next-generation solutions that are helping to meet these growing needs, allowing for more streamlined processes and patient-centric studies.

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Emerging from a coma: Response to language can help assess states of consciousness

Medical Xpress

A man—for the sake of this exercise, let's call him "Paul"—has a heart attack and his brain is starved of oxygen. After several resuscitations and a week in a coma, he finally opens his eyes. However, he doesn't always appear to be present. Doctors say he is still unconscious, in an "unresponsive state"—the patient's eyes are open, but he doesn't move his hand when asked.

Doctors 98
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Opinion: Medical school rankings need a new metric: economic mobility index

STAT News

What are the attributes of a great medical school? U.S. News & World Report’s ranking system says the best American medical schools score high in areas that include the quality of the school’s curriculum (based on the opinions of deans, school administrators, and hospital residency directors); grade-point averages and standardized test scores of incoming students; student-to-faculty ratios; federal research activity; and the proportion of graduates who specialize in primary car

Research 105
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Scientists find potential cellular target for HIV therapies

Medical Xpress

Researchers at The Herbert Wertheim UF Scripps Institute for Biomedical Innovation & Technology have discovered a protein that appears to play a key role in helping HIV replicate in human immune cells, providing more clues about how cellular machinery allows the virus to create new copies of itself.

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STAT+: FDA rejects Lilly’s bid for accelerated approval for its Alzheimer’s drug

STAT News

Eli Lilly said Thursday that U.S. regulators had rejected its application seeking accelerated approval for donanemab, a treatment for people with early stage Alzheimer’s disease. Despite the setback, Lilly said that the planned readout from an ongoing, Phase 3 study of donanemab remains on track for the middle of the year, and if positive, will form the basis of an application for full approval “shortly thereafter.

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Deliver Fast, Flexible Clinical Trial Insights with Spotfire

Clinical research has entered a new era, one that requires real-time analytics and visualization to allow trial leaders to work collaboratively and to develop, at the click of a mouse, deep insights that enable proactive study management. Learn how Revvity Signals helps drug developers deliver clinical trial data insights in real-time using a fast and flexible data and analytics platform to empower data-driven decision-making.