Drug Discovery and Development Archives - Page 8 of 158

Microsoft and 1910 Genetics are discovering health data’s hidden gems: Here’s how

In episode 2 of AI Meets Life Sci, DeviceTalks Managing Editor Kayleen Brown, and Pharma Editor Brian Buntz explore how Big Tech is developing artificial intelligence (AI) infrastructure in a variety of healthcare organizations.

Elena Bonfiglioli, global business leader for Health and Life Sciences at Microsoft, weighs in on the subject, also sharing that she found her passion for technology while programming welfare policy simulations and being continually intrigued by finding the insightful hidden gems held within population data. She explains that changing the DNA of an organization and shifting mindsets are imperative for future businesses to enable the responsible use of AI in healthcare.

Jen Nwankwo, founder and CEO of 1910 Genetics and a Microsoft collaborator, holds an impressive pedigree in biochemistry and biophysics, eventually earning her PhD in pharmacology. She emphasizes that her goals with AI and machine learning (ML) are to solve longstanding p…

AbbVie bets on quality of life approach to gain share in migraine treatment market

In a crowded migraine treatment landscape, AbbVie is aiming to differentiate itself by redefining migraine treatment beyond just counting headache days and other traditional clinical endpoints. At the European Headache Congress (EHC), the company touted its patient-centered approach in evaluating quality of life, daily functioning, rapid relief, and balanced benefit-risk profiles, while aiming to continue addressing unmet needs in the disease area.

“Everyone always talks about patient centricity, but really showing that with our treatments is what matters and what we highlighted with atogepant,” said Michael J. Seminerio, global therapeutic lead at AbbVie. “We actually improved function, quality of life and disability across all trials.”

The ELEVATE, PROGRESS, and ADVANCE atogepant trials

This approach was a guiding principle in the ELEVATE, PROGRESS, and ADVANCE studies, all featured at EHC. The trio of studies concentrated on atogepan…

LSD shows promise as standalone anxiety treatment in trial

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Phase 2b data from MindMed’s LSD-based therapy adds to the mounting evidence that psychedelic-assisted therapies could treat mental health conditions like anxiety, depression and PTSD.

In particular, the study found statistically significant dose-dependent improvements in the Hamilton Anxiety rating scale (HAM-A) at the 100 and 200 μg dose levels in the four-week study. Similarly, recent phase 2 Cybin data found 79% remission for depression after two doses of its psilocybin-based therapy. And MAPS filed for MDMA approval in PTSD based on phase 3 results.

But MindMed’s study was unique in its evaluation of MM-120 as a standalone single-dose oral medication without psychotherapy. The compound, an LSD tartrate formulation, showed clinically and statistically significant reductions in anxiety symptoms over four weeks when given as a single monotherapy dose in patients with generalized an…

Unlocking generative AI requires reshaping culture, operations, and talent dynamics

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In drug discovery and development, generative AI and natural-language processing (NLP) tools promise more than incremental productivity gains. For companies that can integrate such tools strategically into their workflows, the tools open the door to a fundamental rethinking of operational processes. For instance, generative AI tools can accelerate drafting of research articles, novel target identification, and the creation of SOPs for recipe and formulation. NLP, conversely, can mine unstructured scientific data and complex research papers. Because roughly 80% of healthcare data is unstructured, NLP promises to unlock previously inaccessible insights, transforming raw data into actionable knowledge.

But deploying such tools at scale requires a mix of strategic thinking, curiosity and new approaches to cultivating talent. As we enter 2024, life science organizations must rethink their approach to ad…

Delix’s psychedelic-inspired DLX-001 poses no safety or hallucinogenic risks in early testing

Biotech startup Delix Therapeutics has unveiled promising interim phase 1 safety data for its lead neuroplasticity drug candidate DLX-001 at a leading neuroscience conference, marking the first clinical validation of its “psychoplastogen” pipeline. In essence, the company is developing psychedelic-inspired therapies that can prompt neuroplasticity without the hallucinogenic, psychotomimetic, or dissociative effects typical with traditional psychedelic compounds.

According to Delix’s chief medical officer Dr. Aaron Koenig, DLX-001 is safe and well-tolerated. With neuroplasticity emerging as a key factor in rapid-acting antidepressant mechanisms, the company is optimistic about this early human proof-of-concept, which could pave the way for larger efficacy studies and offer an alternative approach in the field where psychedelic medicines have shown limitations, particularly in terms of side effects and potential cardiac liability.

The interim data fro…

MDMA’s potential shift from party drug to PTSD therapy could hinge on strict safety measures

MDMA, the stimulant mood-lifting drug commonly known as ecstasy, could soon transition from party staple to FDA-approved medication — but likely with tight control measures to address its abuse potential and safety risks.

On Tuesday, the nonprofit Multidisciplinary Association for Psychedelic Studies (MAPS) filed an application seeking FDA approval of MDMA-assisted therapy for post-traumatic stress disorder, based on positive phase 3 results demonstrating significant PTSD reductions for most trial participants. In the studies, a significant number of those receiving MDMA-assisted therapy no longer qualified for a PTSD diagnosis after three treatment sessions, with a large majority seeing clinically meaningful improvements.

Currently, the Drug Enforcement Administration (DEA) categorizes MDMA as a Schedule I controlled substance having no approved medical uses, although it is likely to reclassify the stimulant if it receives FDA approval.

MAPS founder Ric…

Zepbound helps people lose 25% of body weight on average 88 weeks, but weight regain is a concern

Lilly’s hot weight loss drug Zepbound (tirzepatide) may be one of the most effective drug therapies for weight loss. In an open-label 36-week study, participants lost an average of 20.9% of their body weight in 36 weeks. With an average weight of 107.3 kg (236.5 lbs), that equates to about 22.4 kg (49.4 lbs) on average.

Weight regain a risk for those going off Zepbound

The catch, however, is that participants needed to keep taking the drug to keep losing weight. Those who stopped taking it saw a rebound, regaining about half of what was lost in the 52-week period after the initial 36-week study. But those who kept taking tirzepatide continued losing weight — an additional 5.5% reduction from week 36 to week 88.

In general, significant weight loss followed be regain can be more than an inconvenience. A Journal of Clinical Endocrinology and Metabolism 16-year prospective cohort study looking at patients with type 2 diabetes found such fluctuations to be linked to…

Pfizer hemophilia drug marstacimab accepted for FDA, EU review

U.S. and European regulators have accepted applications for Pfizer’s marstacimab, an investigational treatment for hemophilia A and B. The FDA set a decision date in late 2024 while the EMA set the stage for a possible approval in early 2025.

The applications are based on positive data from the phase 3 BASIS trial, presented over the weekend at the American Society of Hematology’s annual conference. The data showed marstacimab reduced bleeding rates compared to standard treatments. The study, which investigated 116 people with hemophilia over a 12-month active treatment period, found that marstacimab significantly reduced bleeding rates compared to standard treatments, which include routine prophylaxis or on-demand (OD) treatment. Routine prophylaxis involves regular, scheduled doses of clotting factor concentrates to prevent bleeding episodes in individuals with hemophilia while OD treats bleeding e…

Government patent seizures could curb costs but could have unintended consequences

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The Biden administration has announced a new framework to enforce the government’s “march-in” rights to seize patents on drugs developed with taxpayer funding if it deems that their prices are unreasonably high.

While the government has had these rights for decades thanks to the 1980 Bayh-Dole Act, it has not used them previously. Before the Bayh-Dole Act, the federal government owned inventions resulting from federally funded research.

In making the decision to potentially seize patents for federally-funded therapies, officials argued that Americans often pay two to three times more than other countries for the same drugs, despite taxpayers investing billions in relevant research through agencies such as the NIH. Federal officials highlighted COVID-19 vaccines as a prominent example, balking at recent price hikes by Moderna and Pfizer despite public funds t…

Kristofer Mussar on VectorBuilder’s ethical imperative in gene delivery

VectorBuilder

As genetic technologies unlock new possibilities, companies like VectorBuilder aim to navigate progress responsibly, guided by a strong moral compass. “Ethics to me is the one thing that really is so, so important,” said Kristofer Mussar, managing director of VectorBuilder GmbH. “I have such a high ethical threshold I make decisions that are sometimes not the best business decisions, but they are the right ethical decisions.”

VectorBuilder offers an array of products and services, including vector design, vector cloning, plasmid DNA preparation, AAV capsid evolution, mRNA gene delivery systems, and CDMO services. Its international customer base includes thousands of labs and biotech/pharma companies.

In business practices, especially in a field such as gene delivery, ethics are tantamount. “If you have very strong ethics, that means you have trust,” he explained. “If everybo…

Unicorn gene-delivery firm VectorBuilder eyes future breakthroughs

This image from VectorBuilder showcases a comparative study of luciferase expression in various mouse organs, highlighting the targeted gene delivery capabilities of Anti-CD31 conjugated LNP-mRNA. Controls include IgG2a-conjugated FLuc LNP-mRNA and naked FLuc mRNA.

Genetic tools are growing more powerful by the day and hold immense medical promise. Kristofer Mussar, managing director of VectorBuilder GmbH who holds a Ph.D. in molecular genetics and epigenetics, noted that in the wake of the pandemic, genetic research requires increased awareness and ethical stewardship to responsibly tap into its power. While the current financial climate poses challenges, “the future is very bright in the cloning world and gene delivery,” Mussar noted. “At VectorBuilder, we’re all about innovation and having our finger on the pulse. We’re always focusing on the next thing.”

Earlier this y…

EDC fading in prominence as AI and cloud gain ground

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In a year or two, the clinical trial industry may move beyond electronic data capture (EDC), a technology that has been the cornerstone of clinical data management for decades, projects Raj Indupuri, CEO of eClinical Solutions. Given the potential of electronic medical records (EMR) to feed directly into data infrastructure, the need for EDC may be moot — or at least diminished.

More automated data-collection processes

Already, clinical trials are pulling considerable data from external sources, whether that be outside labs providing supplemental test results or real-world data generated through biomarkers, genomics sequencing, wearables, and other sources circumventing conventional data capture processes.

“There’s more and more data that we’re collecting directly from patients or other sources,” Indupuri said. “And the amount of data that we’re collecting through EDC — or …