Drug Discovery and Development Archives - Page 28 of 158

QRL-201, a novel therapy targeting STMN2 expression, enters phase 1 ALS trial

Cambridge, Massachusetts-based QurAlis has dosed the first patient with the biotherapeutic QRL-201 in a Phase 1 clinical trial (ANQUR) in Canada. QRL-201 is a novel therapy targeting the restoration of STATHMIN-2 (STMN2) expression in amyotrophic lateral sclerosis (ALS) patients. QurAlis believes ANQUR the first study to evaluate such a treatment.

Stathmin-2 (STMN2), also known as superior cervical ganglia neural specific 10 (SCG10), belongs to the stathmin protein family involved in the regulation and dynamics of microtubules. Predominantly expressed in neurons, STMN2 serves key functions in neuronal development, axonal growth and regeneration.

Recent research has revealed the significance of STMN2 in neurodegenerative diseases, especially ALS and frontotemporal dementia (FTD). Researchers have linked the pathogenesis of those conditions to the loss of STMN2 function.

“In mice, STMN2 deletion causes axonal degeneration and loss of muscle innervation, w…

BioTheryX’s Prodegy platform takes center stage in cancer research partnership with Incyte

BioTheryX, a privately-held company, and Incyte (Nasdaq:INCY) have formed a research collaboration and license agreement. Their focus is on discovering and developing molecular glue targeted protein degraders for oncology targets.

Several other privately-held companies also focus on protein degradation. Examples include Kymera Therapeutics (Cambridge, Massachusetts), Arvinas (New Haven, Connecticut), Nurix Therapeutics (San Francisco), Cullgen (San Diego) and Vividion Therapeutics (La Jolla, California).

BioTheryX’s Prodegy platform serves as the partnership’s centerpiece. It aims to identify and develop molecular glue degraders for multiple historically undruggable oncology targets. “Our platform enables flexible degrader approaches, allowing us to tailor the molecular approach to a given target,” said Philippe Drouet, CEO of Biotheryx, over email.

Conventional small molecule inhibitors often insufficiently drug many targets considered druggable. …

Understanding cellular heterogeneity and its implications for disease diagnosis and treatment

[Anusorn/Adobe Stock]

Cellular heterogeneity enables cells in the body to perform an array of functions, but it also plays a role in diseases like cancer as well. In oncology, this fact influences molecular therapy targets, tumor evolution as well as drug sensitivity and resistance. Cellular heterogeneity is also a consideration in areas such as immunotherapy, neuroscience, stem cell research and infectious diseases. By probing cellular heterogeneity in diseased tissues, drug developers can help uncover potential molecular targets for treatment, highlight varying drug sensitivities and unravel the mechanisms responsible for treatment resistance.

To learn more about the importance of cellular heterogeneity, we recently conducted an email interview with two executives at Bio-Rad Laboratories, Mina Zeinali, single cell field application specialist and Joby Chesnick, senior segment manager, single cell technologies …

FDA authorization of vilobelimab signals new opportunities for drug developers in inflammatory diseases

The FDA has granted InflaRx (Nasdaq:IFRX) emergency use authorization (EUA) for the monoclonal antibody Gohibic (vilobelimab) to treat critically-ill COVID-19 patients. The company’s shares were up yesterday almost 84% to $3.77. Today, its shares jumped an additional 62% to $6.10.

The EUA represents a significant advance for the Jena, Germany–based company, which on March 31, 2022, encountered a setback related to the antibody. At that time, the company announced that vilobelimab failed a topline readout for critically ill intubated patients suffering from COVID-induced pneumonia in the phase 2/3 PANAMO study. Later, however, InflaRx reported in a predefined analysis without site-stratification that vilobelimab significantly decreased all-cause mortality at 28 days. While the initial protocol did not require site-stratification, the company amended the protocol based on regulators’ recommendations.

Regulatory nod for vilobelimab could spark new developments …

The psychedelic neuroplasticity and safety conundrum: Reaping the rewards of neuroplasticity with caution

[nobeastsofierce/Adobe Stock]

Evidence continues to build that serotonergic psychedelics such as psilocybin, DMT, and LSD promote neuroplasticity. Cortical atrophy is involved in the development of a broad swathe of neuropsychiatric conditions ranging from depression to substance use disorder. But it remains unclear how psychedelics may potentially treat mood disorders and other conditions. Despite their therapeutic potential, these compounds continue to remain unpopular in some quarters and could be contraindicated for many individuals even if they ultimately win regulatory approval.

Drug developers, such as Boston-based Delix Therapeutics and Onsero Therapeutics, aim to develop compounds that trigger neuroplasticity in what they deem to be a safer and more accessible therapeutic option than classic psychedelics.

Meanwhile, players such as Compass Pathways (Nasdaq:CMPS) and Small Pharma (CVE:DMT), are wo…

The ‘Iron man suit’ for QA Managers: Quality management software taps power of ChatGPT

Gone are the days when Microsoft’s animated paperclip, Clippy, popped up on your screen with helpful — and sometimes annoying — tips and tricks for your Word document. Now, the Israeli eQMS compliance provider Dot Compliance has launched what it hails as an industry-first electronic Quality Management System (eQMS) for life sciences with a prominent generative AI-enhanced digital assistant that could take intelligent assistance to new heights. “It’s not just ChatGPT. It’s many other algorithms that are evolving,” said Doron Sitbon, founder and CEO of the company.

Transforming quality assurance with AI-driven quality management software

Rather than Clippy, Sitbon uses another metaphor to illustrate the company’s ChatGPT-enhanced “Dottie” capabilities for its QMS Xpress quality management software. “I think of it as an Iron Man suit for a [quality assurance] manager,” Sitbon quipped. The Dottie digital assistan…

The new National Cancer Plan launches with goal of cutting cancer death rate by half in 25 years

[Image courtesy of National Institutes of Health]

Today, the U.S. Department of Health and Human Services (HHS) debuted the National Cancer Plan with an ambitious albeit vague quest to “end cancer as we know it.”

Specifically, the initiative will serves as a guideline to support President Biden’s Cancer Moonshot objectives, which aims to reduce the cancer mortality rate by 50% over a 25-year period.

NCI will lead the National Cancer Plan and Cancer Moonshot program

HHS will put the National Institutes of Health’s (NIH) National Cancer Institute (NCI) in charge of the program, which supports the Cancer Moonshot initiative from President and First Lady Biden.

The President’s Cancer Moonshot has galvanized the goal of advancing the fight against cancer,” said Dr. Monica M. Bertagnolli, National Cancer Institute director, in a press release.

Despite Biden’s focus on ramping up oncology res…

An overview of the RSV vaccine candidate landscape in early 2023

[RSV image from NIAID]

Respiratory syncytial virus (RSV) remains a prominent global health concern. Each year, the virus is to blame for 2.1 million outpatient visits for children under 5 years old, according to CDC. Other vulnerable populations, including older adults and immunocompromised individuals, are also at risk.

Nevertheless, RSV has long been a research focus. Scientists first identified RSV in 1956. A year later, the pediatrician and virologist Robert M. Chanock linked RSV to respiratory infections in children. Attempts to develop a vaccine for the virus soon followed.

Despite numerous attempts beginning in the 1960s, an FDA-approved RSV vaccine has remained elusive until recently. Out front is GSK’s (NYSE:GSK) RSV vaccine for adults aged 60 and older, which has won the backing of an FDA advisory panel. Close behind is Pfizer’s (NYSE:PFE) single-dose vaccine for the same a…

XOMA acquires Ixinity’s commercial payment and milestone rights in $9.6M

Biotech royalty aggregator XOMA (Nasdaq:XOMA) has acquired the commercial payment and part of the milestone rights to Ixinity (trenonacog alfa), a hemophilia B therapy originally from Aptevo Therapeutics (Nasdaq:APVO). The aggregator expects the move to bolster its royalty and milestone portfolio, potentially providing consistent cash flow and ramping up its royalty aggregation strategy.

The acquired asset, Ixinity, is now marketed by Medexus Pharmaceuticals (TSE:MDP) to control and prevent bleeding episodes and postoperative management in people with hemophilia B.

Aptevo sold Ixinity rights to Medexus Pharmaceuticals in 2020. The FDA first approved Ixinity in 2015.

A win-win deal

Brad Sitko

Brad Sitko, chief investment officer of XOMA, explained the company’s mission to Drug Discovery & Development, stating, “Our focus is to provide non-dilutive capital to biotech companies, so they …

Study suggests mGlyR could be a promising antidepressant target

A model illustrates glycine molecules (teal) interacting with GPR158 brain cell receptors, influencing the nervous system. [Martemyanov lab, The Wertheim UF Scripps Institute]

A study published in Science has revealed important information about a brain receptor known as mGlyR, which could be a potential antidepressant target. An abbreviation of “metabotropic glycine receptor,” mGlyR is highly active in the brain’s medial prefrontal cortex (mPFC). Abnormal activity in the brain region is associated with depression.

Formerly known as GPR158, mGlyR is a specific brain receptor type that responds to certain chemical signals. In addition to depression, researchers have linked mGlyr to cancer development and cognition. GPR158 directly interacts with a chemical known as glycine, which plays a role in various brain functions.

Both glycine and aurine affect mGlyR. Researchers believe these chemicals …

Pharmacovigilance in personalized medicine: Adapting to a new era of drug development

[Image courtesy of LeoWolfert via iStock]

One of the ways that the COVID-19 pandemic had a profound impact on the pharma industry was how it highlighted the importance of pharmacovigilance. As personalized medicine gained traction, drug developers had to evolve their strategies to ensure safety and efficacy. In their report, “The Future of Pharmacovigilance,” Dr. Sabine Jeck-Thole and Ben Enejo, partners at Arthur D. Little, discussed the challenges and opportunities in this new landscape.

Embracing the shift in perception of adverse events and side effects

“When we heard about adverse events and side effects, it was not bad news,” Jeck-Thole said in an interview. “It indicated that we understood our drug or vaccine much better than without any safety information.” She emphasized that this shift in perception was essential for the industry as it moved towards pharmacovigilance in personal…

Big Pharma, it’s time to talk: Investors demand transparency on patent thickets

[PIRO4D/Pixabay]

Pharmaceutical companies are running into pushback for their “patent thicket” tactics, designed to maintain exclusive rights and delay cheaper generics. The Financial Times reported that an ethical investor coalition is pushing for greater transparency and urging major drug makers to disclose their patent strategies.

Unraveling the patent thicket mystery

Patent thickets are a common intellectual property tactic in pharma. Companies file numerous patents beyond the primary one for a specific compound. Critics say this cunning approach stalls the introduction of generics even after the 20-year exclusivity of primary patents expires. Consequently, drug prices remain high, and affordable medications remain out of reach for many.

Drugs like Humira (adalimumab) and Keytruda (pembrolizumab), for example, have experienced extended periods without biosimilar or generic competition, lea…