FDA has published draft guidance to clarify its perspective on including children in clinical trials. The agency notes that it wrote the draft guidance to help industry, sponsors and institutional review boards (IRBs) protect children in clinical studies testing drugs, biological products and medical devices. The draft guidance is titled “Ethical Considerations for Clinical Investigations of Medical…

On the heels of launching a Phase 2b study of LSD in generalized anxiety disorder, MindMed (Nasdaq: MNMD) has dosed the first subject in a Phase 1 study evaluating MDMA-like substances in healthy volunteers.  MindMed is developing a synthetic enantiomer of MDMA as a potential treatment for core symptoms of autism spectrum disorder (ASD). Early…

Dr. Susan Galbraith, executive vice president of oncology R&D at AstraZeneca, says her entry into the pharmaceutical industry was serendipitous. “I always wanted to be a doctor, and during medical school, I was fascinated by the science behind how cancer happens and what we can do to better treat it,” she said. After following this…

First synthesized in 1962, the dissociative anesthetic ketamine is rapidly growing in popularity for its off-label use in treating depression and mood disorders. In recent years, hundreds of clinics across the U.S. have begun offering ketamine for depression, anxiety and other mood disorders.   The surge in interest has also attracted startups such as Mindbloom…

One of the largest industry conferences, DPharm, was recently held on September 13 to 14 in Boston. Focusing on innovations to modernize clinical research, this year’s event featured speakers ranging from the popular author Malcolm Gladwell to former FDA Commissioner Dr. Scott Gottlieb and BIO CEO Dr. Michelle McMurry-Heath. Here, we provide reflections from several…

Eli Lilly and Company’s (NYSE:LLY) Phase 3 monotherapy ADvocate trials in atopic dermatitis (AD) found that the investigational interleukin 13 inhibitor lebrikizumab supported durable skin clearance and reduced itching in a subset of patients with atopic dermatitis. The Indianapolis–based company defined responders as those with at least 75% improvement in the Eczema Area and Severity…

Unified regulatory standards and pharmacovigilance harmonization are critical to enabling global pharmaceutical firms to operate more safely and efficiently. By normalizing the reporting of clinical data, stakeholders can minimize risks, improve market conditions, support early access to products, promote competition and efficiency, and reduce the unnecessary duplication of clinical testing. Multiple regulatory agencies, including the…

FDA has granted Pfizer’s (NYSE:PFE) investigational Group B Streptococcus (GBS) vaccine candidate, GBS6 or PF-06760805, Breakthrough Therapy Designation. The designation covers active immunization of mothers during pregnancy to confer protection against invasive GBS disease in newborns and young infants. GBS disease can result in lifelong neurologic impairments or death, according to a 2019 article in…

The healthcare industry traditionally has used site-based clinical trials to assess the efficacy and safety of drugs and medical devices. Data collected from patients participating in these site-based studies would be analyzed to evaluate the suitability of a medical product for commercial release. However, the exponential increase in patient data recorded by digital devices in…

Rocket Pharmaceuticals (Nasdaq:RCKT) is a late-stage clinical biotech focusing on developing gene therapies for rare pediatric diseases with high unmet need. In 2016, that focus drew in Kinnari Patel, president and COO, and Jonathan Schwartz, chief medical officer of the company. “My passion has been making rare disease drug development more efficient and having more therapies…

The World Health Organization (WHO) has awarded prequalification to GSK’s (LSE/NYSE:GSK) Mosquirix. The move marks the first time that a malaria vaccine has won prequalification. The WHO decision regarding the Mosquirix malaria vaccine is a prerequisite for United Nations (UN) agencies like UNICEF to procure the vaccine in regions with moderate to high P. falciparum malaria transmission. Malaria…

The risk of cancer deaths has fallen steadily in recent years. Before the pandemic, the risk of death from cancer fell roughly 2% annually from 2015 to 2019. In the 1990s, the risk of cancer death fell approximately 1% each year, according to the American Cancer Society. In addition to improved screening and diagnosis, cancer…

Cancer diagnoses and screenings dramatically dropped during the peak of the COVID-19 pandemic. Recent data from tens of thousands of patients suggest that the U.S. is likely to see at least two years of increased cancer diagnosis rates and poorer outcomes, according to Jeff Elton, CEO of ConcertAI. EHR data reflect a pronounced drop in pan…

Pfizer (NYSE:PFE) and Morgan State University have unveiled a novel program known as the Doctor of Public Health (DrPH) Fellowship in Vaccines Medical Development. A central aim of the program is to foster diversity in vaccine development.  In particular, the two-year DrPH program aims to increase the number of minority scholar-practitioners involved in vaccine development. In addition,…

Genentech has opened a novel clinical supply center at its birthplace in South San Francisco. The planning for the new $250 million center began in 2019 when Genentech, a member of the Roche Group, began earnest efforts to imagine what the future of biomanufacturing looks like. “We were looking at where we are going in…

Moderna (Nasdaq:MRNA) announced today that it is suing Pfizer (NYSE:PFE) and its COVID-19 vaccine partner BioNTech (Nasdaq:BNTX) over patent infringement in U.S. and German courts. Cambridge, Massachusetts–based Moderna said it is in the process of filing the complaints in U.S. District Court in Massachusetts and the Regional Court of Düsseldorf in Germany. The suits could…

Pfizer (NYSE:PFE) and BioNTech (Nasdaq:BNTX) have filed paperwork with the FDA requesting Emergency Use Authorization (EUA) for an updated COVID-19 vaccine booster. Specifically, the request relates to an omicron BA.4/BA.5-adapted bivalent COVID-19 vaccine. In late June, FDA recommended including an omicron BA.4/5 component for COVID-19 vaccine boosters. Last week, the UK approved Moderna’s bivalent COVID-19 vaccine…

COVID-19 is an emerging focus for EpicentRx, a private biotech focused on multiple immune-mediated disorders. The La Jolla, California–based company is developing a next-gen COVID vaccine for SARS-CoV-2 based on nucleocapsid rather than spike protein.  The recombinant replication-competent adenoviral vaccine that expresses the SARS-CoV-2 nucleocapsid protein could be more resistant to COVID-19 variants.  The real-world…

It’s clear that drug discovery and development costs have rapidly increased in recent decades. That said, estimates vary widely. For example, a 2020 article in JAMA noted that estimates range from $314 million to $2.8 billion. A high drug discovery failure rate exacerbates the problem. Part of the challenge of making such calculations is the…

Not long ago, diversity in clinical research seemed like an obscure topic. Ben Enejo, a partner at Arthur D. Little, recalls mentioning the theme in the past and getting blank stares. But the pandemic and the murder of George Floyd helped highlight the importance of diversity on multiple fronts, including in clinical research. As a result, there are…

Decades after compounds like LSD and psilocybin were made illegal , psychedelic medicine appears poised to go mainstream in the coming years, potentially treating conditions ranging from anxiety to PTSD. In the interim, however, hurdles remain. The American Psychiatry Association (APA) recently released a conservative position statement on psychedelics and empathogens such as MDMA. In…

In 2016, the FAIR Guiding Principles for scientific data management and stewardship were published, laying out guideposts for scholarly data producers to make their data discoverable and usable in the future. The FAIR principles seek to ensure that data is Findable, Accessible, Interoperable, and Reusable. At the time of their publication, they articulated and centralized many points…

New York City-based Kallyope is one of the few biotechs focused solely on the gut-brain axis.  The privately-held company has built a cross-disciplinary team that weaves together advanced technologies in sequencing, bioinformatics, neural imaging, human genetics, and cellular and molecular biology to interrogate the hormonal and neural signaling pathways that comprise the gut-brain axis.  The…

Axcella Therapeutics (Nasdaq:AXLA) has announced positive topline results from a Phase 2a randomized, double-blind, placebo-controlled study of the metabolism-modulator AXA1125 in patients with fatigue from long COVID. The biotech’s Phase 2a study was among the first drug studies to focus on long COVID. In February, FDA granted a Fast Track Designation to AXA1125 to treat…

It’s easy to feel like clinical trials are complex. They are experiments involving human beings — so things change. But, despite feeling like complexity is everywhere, there is little clarity on what constitutes a complex trial and what sponsors need to do to prepare these trials for success. Both the FDA and EMA, for example,…