clinical trial Archives - Medical Design Sourcing

NKGen Biotech’s SNK01 ‘natural killer’ cell therapy for Alzheimer’s gets FDA nod for clinical trial

Santa Ana, California–based NKGen Biotech has received FDA clearance to begin a phase 1/2a trial for their “natural killer” cell therapy, SNK01, designed to treat Alzheimer’s Disease. The autologous, non-genetically modified natural killer (NK) cell therapy involves extracting a patient’s own cells through a blood draw, and then processing them to enhance their natural abilities in a laboratory setting before reinfusing them into the patient’s body.

NKGEN Biotech modifies NK cells to improve their cytotoxicity and their activating receptor expression, which helps them to identify and engage with target cells. This process imparts the NK cells with the ability to target and combat the specific cells associated with Alzheimer’s.

Early data are promising

Data from a phase 1, 3 + 3 dose-escalation trial found SNK01 to be well-tolerated. The drug candidate appeared to cross the blood-brain barrier to help reduce proteins and neuroinflam…

Leqembi could mark new era in Alzheimer’s treatment progress: An overview of the evolving drug development scene

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Today, the FDA granted traditional approved to lecanemab (branded as Leqembi), a monoclonal antibody from Eisai and Biogen for adult patients with Alzheimer’s disease. The agency made the decision on the basis of a confirmatory trial that showed its clinical benefit.

The drug, which reduces the formation of amyloid plaques in the brain, is the first of its kind to transition from an accelerated approval to a traditional approval for Alzheimer’s treatment.

Leqembi’s annual launch price is $26,500.

Following approval, lecanemab stands to potentially gain Medicare coverage with qualifications.

The approval of Leqembi is a positive step forward and will spur further advances and investments in Alzheimer’s research, according to Dr. Howard Fillit, co-founder and chief science officer of the Alzheimer’s Drug Discovery Foundation (ADDF).

“We final…

Delix Therapeutics launches phase 1 study for novel neuroplasticity-promoting therapeutic

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Boston-based Delix Therapeutics has won regulatory approval to commence a study of the non-hallucinogenic psychoplastogen DLX-001. Psychoplastogens are a class of drugs that have the potential to promote neuroplasticity, which refers to the brain’s ability to adapt and form new connections between neurons. That capability could enable improvements in conditions such as depression, anxiety and addiction.

DLX-001 offers potential for a range of conditions, according to Retsina Meyer, head of corporate strategy at Delix. “The aperture for indications for psychoplastogens is broad and is even broader for our non-hallucinogenic compounds of this class,” Meyer said. DLX-001 holds promise for conditions where cortical dendritic/synaptic atrophy plays a role in the pathology. Such atrophy is involved in conditions ranging from major depressive disorder (MDD), schizophrenia, Parkinson’s disease…

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…

Walgreens announces clinical trial offerings

The drug store giant Walgreens (Nasdaq:WBA) has launched a clinical trial business aimed at optimizing patient experience, recruitment and clinical trial diversity.

Deerfield, Illinois–headquartered Walgreens will offer both in-person and virtual care clinical trial options.

In a press release, Walgreens underscored FDA’s push to improve racial and ethnic diversity in clinical studies. In 2020, three-fourths of clinical trial volunteers were white, and only 11% were Hispanic, while fewer than 10% were Black and Asian, according to FDA.

Walgreens intends to use pharmacy and patient-authorized clinical data to match diverse patient populations to clinical trials in an array of disease areas using race, gender, socioeconomic status and location data.

Walgreens also promises to help drug developers improve patient recruitment and enrollment in clinical trials. The company operates almost 9,000 stores. More than half of them are based in socially vulne…

Axogen announces positive topline data from Phase 3 nerve graft study

Focused on developing peripheral nerve regeneration and repair products, Axogen (Nasdaq:AXGN) announced that the Phase 3 RECON study met its primary endpoint in comparing its Avance nerve graft to conduits in digital nerve injuries.

The primary endpoint was the return of sensory function based on static two-point discrimination. This test measures the ability to detect that two close objects touching the skin are distinct points.

“The study data confirmed that as gap lengths increased, Avance returned superior levels of sensation, and this was achieved at earlier time points than those observed for conduits,” said co-lead Investigator Dr. Jonathan Isaacs, professor and chair, division of hand surgery at Virginia Commonwealth University.

The Avance nerve graft also had a good safety profile in the study.

Axogen plans to submit a Biologics License Application (BLA) for the product in the second half of 2023.

The COVID-19 effect: Research platforms require new capabilities

Photo by Chris Liverani on Unsplash

Like a Category 5 hurricane, COVID-19 has uprooted traditional clinical trial methods to clear space for emerging models and practices to grow and flourish. Three related and complementary trends will have a profound and lasting impact.

The shift to virtual/decentralized clinical trials. The popularity of decentralized clinical trials (DCTs) skyrocketed following the nationwide lockdown last March. With safety concerns a top priority, the FDA encouraged researchers to consider alternatives for patient assessments and data collection, including virtual visits. Since elements of the DCT model (full or hybrid) enable patient participation from their homes and can be integrated into any clinical trial, DCTs are fast becoming the new normal. The increasing utility and acceptance of real-world data. Real-world data (RWD) refers to data relating to patient health status and/or the …

Is J&J’s COVID-19 vaccine linked to tinnitus?

In Johnson & Johnson’s (NYSE:JNJ) Phase 3 trial for the Ad26.COV2.S COVID-19 vaccine, six vaccine recipients developed tinnitus or ringing in the ears.

In five of those individuals, tinnitus had either resolved or was resolving. The condition was unresolved in the other trial volunteer.

No placebo recipients developed the condition.

J&J concluded that the tinnitus reports were likely unrelated to the vaccine. The principal investigator in the trial that two of the events were related and the remainder unrelated.

A 21-year-old volunteer involved in an earlier clinical trial for the J&J vaccine developed tinnitus and sudden hearing loss 34 days after receiving the vaccine. The patient recovered 69 days after receiving the injection. Johnson & Johnson also determined that the event was unrelated to the vaccine.

In all, some 43,783 volunteers participated in the Phase 3 trial. A total of 19,630 participants received the Ad26.COV…