Drug Discovery and Development Archives - Page 52 of 158

Why technology is critical for pharmacovigilance harmonization

[Image courtesy of LeoWolfert via iStock]

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 International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) and the Pan American Network for Drug Regulatory Harmonization, are looking for ways to achieve this. Through the use of technology, stakeholders can deploy tools such as artificial intelligence (AI), machine learning (ML), and natural language processing (NLP) to improve safety monitoring and shift the industry from reactive to proacti…

LSD-assisted therapy resulted in durable anxiety improvement in Phase 2 study

[Image courtesy of PubChem]

A randomized placebo-controlled Phase 2 study found that lysergic acid diethylamide (LSD)–assisted therapy yielded significant reductions in anxiety and depression symptoms for up to 16 weeks.

The data from the study was recently published in the peer-reviewed Biological Psychiatry.

Researchers at the University Hospital Basel (UHB) conducted the investigator-initiated study, which involved 42 patients.

While there was a substantial amount of research on LSD-assisted therapy in the 1950s and 1960s, psilocybin has emerged as a more popular compound for psychedelic-based therapy in recent years.

In the data published in Biological Psychiatry, researchers used Spielberger’s State-Trait Anxiety Inventory–Global (STAI-G) score and the HAM-D-21 (Hamilton Depression Rating Scale) to quantify anxiety and depression, respectively. Investigators also used the Beck Depressio…

Two new SARS-CoV-2 antibodies could neutralize COVID-19 strains with high efficacy

This scanning electron microscope image shows SARS-CoV-2 in yellow. [Image courtesy of NIAID RML]

Researchers at Tel Aviv University have identified two monoclonal antibodies that are broadly effective against SARS-CoV-2 variants.

The researchers investigated the activity of nine anti-SARS-CoV-2 monoclonal antibodies (mAbs) against several variants of concern, including alpha, beta, gamma, delta and omicron.

The scientists at the Tel Aviv University’s Sackler Faculty of Medicine suspect that the antibodies may reduce the future need for regular boosters.

The research was published in Communications Biology.

Natalia Freund and doctoral students Michael Mor and Ruofan Lee at Tel Aviv University led the research in collaboration with Ben Croker of the University of California at San Diego. Other research partners included Ye Xiang, a professor at Tsinghua University in Beijing and professors Me…

The 10 fastest growing pharma companies of 2021

Photo by Chris Liverani on Unsplash

By now, it should come as no surprise that developers of COVID-19 vaccines had healthy profits in 2021. Nevertheless, it remains difficult to fathom that BioNTech (Nasdaq:BNTX) saw annual revenue growth of 3,834.4% last year. Unsurprisingly, BioNTech topped the list of fastest growing pharma companies of the year. The company, which jointly developed one of the most popular COVID-19 vaccines with Pfizer (NYSE:PFE), had a five-year compound annual growth rate (CAGR) of 319%. In 2021, Moderna had annual revenue growth 2,199.1% last year, thanks to its role developing a popular COVID-19 vaccines.

Outside of vaccines, COVID-19 products such as anti-SARS-CoV-2 monoclonal antibodies also played a role in making Regeneron one of the fastest growing pharma companies. The company had 89.1% revenue growth last year compared to Pfizer’s 2021 growth rate of 95.2%.

The mar…

How real-world evidence can augment clinical trial data

Photo by Joshua Sortino on Unsplash

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 recent years has introduced to trial sponsors and researchers the possibility that real-world data (RWD) and real-world evidence (RWE) may be used to augment data from a clinical trial.

While RWD and RWE often are conflated, they are not the same. As the FDA explains, “Real-world data are the data relating to patient health status and/or the delivery of healthcare routinely collected from a variety of sources.” RWD can come from multiple sources, including electronic health record…

Rocket Pharmaceuticals focuses on the root cause of rare pediatric diseases

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 available per patient,” Patel said. “That’s always been my goal since I started my student rotation at the Office of Orphan Product Development at FDA in 2004.”

While safety concerns have recently surfaced in a limited number of gene therapy clinical trials, the field’s promise to develop curative therapies for rare diseases remains captivating.

In fact, Schwartz said the opportunity to work on potentially curative therapies was part of his reason for joining Rocket. “It was a chance to work on cutting-edge science addressing the caus…

WHO decision clears path for GSK’s Mosquirix malaria vaccine

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 was associated with 409,000 deaths in 2019, according to WHO.

GSK shares fell 1.19% in afternoon trading to $31.47.

The Mosquirix malaria vaccine is also referred to as RTS and S/AS01.

Mosquirix has a long developmental history. GSK began developing the vaccine, then known as RTS,S, in 1987.

In 2001, GSK and PATH’s Malaria Vaccine Initiative entered into a public-private partnership to develop the vaccine for infants and young children in malaria-prone countries.

To win the WHO prequalification, GSK shared detailed c…

Bavarian Nordic launches Phase 3 clinical study of COVID-19 booster candidate

Bavarian Nordic (OMX:BAVA) has dosed the first subject in a Phase 3 clinical study of the COVID-19 vaccine candidate ABNCoV2.

The study will test whether ABNCoV2 is non-inferior to Comirnaty from Pfizer (NYSE:PFE) and BioNTech (Nasdaq:BNTX) in terms of neutralizing antibodies it produces against the original strain of SARS-CoV-2. Secondary endpoints in the study will assess how the vaccine stacks up against other variants of concern.

In May, Bavarian Nordic announced that a Phase 2 study found that ABNCoV2 induced a solid boost in neutralizing antibodies against the original omicron strain.

The non-adjuvanted ABNCoV2 vaccine candidate uses a virus-like particles (VLP) platform.

Copenhagen, Denmark–based Bavarian Nordic has become best known recently for its monkeypox and smallpox vaccine. Known as Jynneos in the U.S., the monkeypox vaccine is named Imvanex in Europe and Imvamune in Canada.

Bavarian Nordic’s product portfolio includes…

Why Futura Medical believes MED3000 could disrupt the market for PDE5i drugs like Viagra and Cialis

Futura Medical has announced Phase 3 data on its topical erectile dysfunction treatment MED3000, clearing the path for an FDA filing that could make the therapy an over-the-counter treatment option for erectile dysfunction.

Phosphodiesterase type 5 inhibitors (PDE5is) such as Viagra (sildenafil) and Cialis (tadalafil) and generic equivalents currently dominate the erectile dysfunction market.

Sildenafil and tadalafil are among the most widely prescribed drugs on the market.

“Whilst PDE5is are effective treatments, they have certain drawbacks,” said Ken James, executive director and head of R&D of Surrey, UK–based Futura Medical.

For one thing, PDE5i side effects can include headache, flushing, nasal congestion and back pain. In addition, FDA has repeatedly warned about the potential of synergistic hypotensive effects resulting from combining PDE5is with nitrates.

Ken James

In …

10 promising oncology drug candidates

[3-D model of adagrasib molecule from PubChem]

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 survival rates have fallen as a result of therapeutic breakthroughs.

In this feature, we round up a variety of oncology drug candidates that may prove to be promising cancer therapeutics in the coming years.

1. Adagrasib

Adagrasib from Mirati Therapeutics is a KRASG12C inhibitor. In 2021, the drug candidate won Breakthrough Therapy Designation for patients with advanced non-small cell lung cancer (NSCLC) with the KRASG12C mutation. The KRAS protein inhibitor has been the subject of 14 trials to date, including a Phase 3 study focused on …

Why the pandemic is enabling a data-driven approach in oncology

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 tumor diagnoses and multiple myeloma (MM) diagnoses.

Against that backdrop, Elton recommends that the oncology field adopt a data-driven approach to optimize patient care in the wake of the pandemic.

On one level, the dip in diagnostic rates in the early days of the pandemic was not surprising, given how common lockdowns were in early 2021. At that time, it seemed likely that the situation would play a role in driving later-stage oncology diagnosis. The problem is especially evident in the hematological malignancy population.

“The early phase of the pandemic — before vaccination — really hit peop…