Drug Discovery

Ivy Brain Tumor Center and its partner Sonalasense have announced positive initial results in a first-in-human Phase 0/1 clinical trial involving recurrent glioblastoma patients. Sonalasense has developed a noninvasive sonodynamic therapy (SDT) that pairs low-intensity ultrasound with chemotherapeutic agents known as sonosensitizers. The therapy is noninvasive. Data from the trial indicate that SDT swiftly causes…

The Vaccines and Related Biological Products Advisory Committee (VRBPAC) unanimously supported emergency use authorization to administer the Pfizer-BioNTech Comirnaty vaccine as a booster to those 65 years of age and older, and individuals at high risk of developing severe COVID-19. The panel, however, resoundingly rejected a plan to administer the vaccine as a booster to…

FDA has expanded the emergency use authorization for Lilly’s (NYSE:LLY) bamlanivimab and etesevimab combo to include post-exposure prophylaxis. Earlier in the year, federal authorities had counseled against the use of the  monoclonal antibody cocktail in several states given the rise of SARS-CoV-2 variants in the U.S., including Gamma (P.1) and Beta (B.1.351). Meanwhile, demand for…

Johnson & Johnson’s Janssen Pharmaceutical Companies today announced a positive CHMP opinion on its Byannli schizophrenia treatment. The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) issued the positive opinion. The committee recommended using long-acting atypical antipsychotic Byannli (6-monthly paliperidone palmitate; PP6M) therapy for the maintenance treatment of schizophrenia. The recommendation…

Minorities tend to participate in clinical trials at far lower rates than their real-world demographics and prevalence of the disease, according to the American Society of Clinical Oncology. In addition, minorities often have worse outcomes for certain cancers than the broader public. Just one example: the mortality rate for Black women with breast cancer is 40% higher than…

Privately-held biotech QurAlis plans to begin clinical development for the first-in-class molecule QRL-201 for amyotrophic lateral sclerosis (ALS) in the second half of 2022. QRL-201 is now the subject of IND-enabling studies. The drug candidate targets the restoration of STATHMIN-2 (STMN2) expression in ALS. STMN2 is a protein encoded by the STMN2 gene that is involved in…

This September is Pulmonary Fibrosis Awareness Month, which the Pulmonary Fibrosis Foundation (PFF) and allies have launched to educate the public about the disease family. Involving scarring of the lungs, pulmonary fibrosis gradually robs the breath from patients it affects.  Some 250,000 Americans live with idiopathic pulmonary fibrosis, according to PFF.   At present, two drugs…

Sanofi (NSDQ:SNY) announced today that it has completed the purchase of its mRNA partner Translate Bio. The previously announced deal, according to Sanofi, accelerates the French pharma giant’s efforts to develop transformative vaccines and therapies using mRNA technology. The deal comes during a year in which mRNA-based Moderna (NSDQ:MRNA) and Pfizer-BioNTech (NYSE:PFE/NSDQ:BNTX) COVID-19 vaccines are…

The rapid pace of development of COVID-19 vaccines and antibodies has redefined expectations for clinical trials. As SARS-CoV-2 variants such as Delta continue to fuel the epidemic, the industry must continue to ensure testing and clinical trials stay ahead of mutations. To get a clearer sense of what the ramifications of this accelerated development will…

Direct-to-patient clinical trial company Medable (Palo Alto, California) has recently hired MaryAnne Rizk as its chief strategy officer. Rizk was IQVIA’s former senior vice president, digital R&D strategy.  Rizk was also recently named a PharmaVoice 100 leader. Rizk is also a graduate advisor and an adjunct professor for the school of engineering at the Stevens Institute of…

AB Science today touted results from a study of masitinib in combination with docetaxel in treating prostate cancer. Paris-based AB Science presented the data at the 2021 American Urological Association (AUA) annual meeting, according to a news release. Masitinib in combination with docetaxel as a first-line treatment of metastatic castrate refractory prostate cancer (mCRPC) eligible…

AstraZeneca (LON: AZN) announced that Imfinzi (durvalumab) together with tremelimumab and platinum-based chemotherapy improved overall survival by 23% and progression-free survival by 28% compared to chemotherapy alone in patients with Stage IV (metastatic) non-small cell lung cancer (NSCLC). The data came from a Phase 3 trial known as POSEIDON. Imfinzi and tremelimumab are immune checkpoint…

FDA leaders have issued a statement regarding the agency’s plan for authorizing COVID-19 vaccines for children. The FDA plans to “follow the science” with eyes on COVID-19 vaccines for children younger than 12 as schools begin to get back in session around the country, the agency said in a news release attributed to acting Commissioner…

The National Institutes of Health (NIH) announced a plan to provide approximately $185 million over five years to a consortium researching genomic variation. Initiated and funded by NIH’s National Human Genome Research Institute (NHGRI), the Impact of Genomic Variation on Function (IGVF) consortium will receive the $185 million over five years in the form of…

A great many new and varied approaches to clinical trial management have gained ground during the COVID-19 pandemic through the help of virtualization tools, strong partnerships, and regulatory guidance. Despite the upheaval this year and last, there appears to be a silver lining. The systemic changes have enabled remarkably quick development in adapting trials to…

FDA determined it couldn’t conclude that the benefits of lenzilumab from Humanigen (NSDQ:HGEN) outweigh the risks for patients hospitalized with COVID-19.  Lenzilumab is the company’s lead product candidate.  The drug works by neutralizing granulocyte-macrophage colony-stimulating factor (GM-CSF), which is implicated in the so-called cytokine storm.  The drug is the subject of 16 clinical trials spanning…

Traditionally, one of the most challenging aspects of treating schizophrenia is medication adherence. Patients who neglect to take their medicine have an elevated risk of relapse, hospitalization and suicide. “There’s a higher likelihood of an exacerbation of the illness occurring,” said Dr. Gustavo Alva, medical director at ATP Clinical Research (Costa Mesa, California).  The problem…

While President Biden initially announced that COVID-19 boosters would be available by September 20, media reports suggested that the announcement caught FDA off guard. White House chief medical advisor Dr. Anthony Fauci recently suggested in an interview with CBS’s “Face the Nation” that the U.S. government is scaling back its booster plan for the broader…

Cary, North Carolina–based Thread has introduced a consultative service known as Thread Adopt to support change management in decentralized clinical trials. The service encompasses processes, tools, templates and educational functionality. “Thread Adopt guides and empowers customers to analyze their pipeline, with a view to match decentralized solutions and services to the specific needs of their…

The FDA’s relationship with Biogen is under scrutiny following the controversial approval granted to the company’s Aduhelm Alzheimer’s drug. According to a report from Endpoints News, the House Energy & Commerce and Oversight committee chairs issued a letter this week requesting that acting FDA Commissioner Dr. Janet Woodcock and the FDA provide very specific information by…

The FDA notified Kaleido Biosciences (NSDQ:KLDO) determined that the Lexington, Massachusetts-based company did not submit investigational new drug (IND) applications for clinical trials related to the investigational drug KB109, a microbiome modulator. The two clinical trials in question, dubbed “Protocol K031-120” and “Protocol K032-120,” are testing KB109 in adults with mild-to-moderate COVID-19. KB109 is a synthetic…

Although nonalcoholic steatohepatitis (NASH) is one of the most common types of chronic liver disease in the U.S., it is a disease that is unfamiliar to many Americans. Despite this, its prevalence is increasing, and by some estimates, 27 million Americans will be living with NASH by 2030. NASH is a progressive condition that starts…

The University of Minnesota Medical School has developed a therapeutic vaccine for opioid use disorder that is the subject of a new Phase 1 randomized placebo-controlled clinical trial. The trial, which recently begun enrollment, will be conducted at the Columbia University Irving Medical Center in New York. Sandra Comer, a professor of neurobiology at the College of Physicians and…

Direct-to-patient clinical trial company Medable (Palo Alto, California) has added MaryAnne Rizk to its executive team. Rizk formerly worked at IQVIA as a senior vice president, digital R&D strategy. She is also an adjunct professor at the Steven Institute of Technology (Hoboken, New Jersey) and a managing partner at Rizk Management Consulting. In August, Rizk…

Novartis (NYSE:NVS) has received FDA fast track designation for LNA043, a potential treatment for osteoarthritis of the knee. LNA043 is a modified version of the human angiopoietin-like 3 (ANGPTL3). The drug would be a potential first-in-class disease-modifying osteoarthritis therapy. Osteoarthritis is a common joint disorder. In 2017, more than 300 million people internationally had either hip…