Liss' Lowdown #34

Preclinical Breakthrough of the Month:

New Melanoma Therapy Prevents Brain Metastasis in Preclinical Studies

A team led by Dr Sheri Holmen at Huntsman Cancer Institute has developed a promising combination therapy that could reshape the treatment of advanced melanoma. The therapy, tested in preclinical models, successfully prevented melanoma from spreading to the brain - a major cause of mortality in late-stage patients.

The key lies in targeting focal adhesion kinase (FAK), a driver of brain metastasis. When combined with inhibitors of RAF and MEK, the treatment significantly improved survival and halted brain colonisation by cancer cells.

Backed by the National Cancer Institute (NCI) and developed with Verastem Oncology , the oral therapy pairs defactinib (FAK inhibitor) with avutometinib (RAF/MEK inhibitor) - offering a potentially more accessible alternative to infusion-based immunotherapies.

Clinical trials are now underway at the University of Utah and University of Iowa , bringing new hope to patients facing one of the deadliest forms of melanoma.

Clinical Breakthrough of the Month:

Transcend Therapeutics hits key milestone in PTSD trial

Transcend Therapeutics has reported positive Phase II results for TSND-201, a distinctive form of methylone, a stimulant similar to MDMA but without hallucinogenic effects due to its lack of impact on the serotonin 2A receptor. Instead, it primarily targets monoamine transporters. The study, which included 65 patients with severe PTSD, successfully met its primary endpoint, showing a significant reduction in symptoms compared to the placebo group.

After four weekly doses, patients treated with TSND-201 saw a 9.64-point drop in PTSD symptoms (CAPS-5 scale) by day 64. Over half (57.1%) achieved a 50%+ improvement, and nearly a third entered remission – both far higher than in the placebo group.

The therapy was well tolerated, with only mild, short-term side effects. Transcend now plans to move into Phase III trials, positioning TSND-201 as a potential new option in a field still dominated by talk therapy and antidepressants.

Partnership of the month:

Clario Acquires NeuroRx to Deepen Neuroscience Imaging Expertise

Clario has made its second neuroscience-focused acquisition in a week, acquiring NeuroRx , a specialist in medical imaging for neurological diseases. The deal brings Clario a seasoned team and cutting-edge imaging capabilities that have contributed to 12 FDA-approved multiple sclerosis treatments since 2011 and supported over 300 clinical trials.

NeuroRx founder and CEO, Douglas Arnold , will join Clario as a senior scientific advisor, reinforcing Clario’s position at the forefront of neurological drug development. This follows Clario’s recent acquisition of WCG ’s eCOA division, further cementing its commitment to advancing neuroscience clinical trials.

With a legacy of over 30 years in imaging across oncology, cardiovascular, ophthalmology, and general medicine, Clario is now expanding its reach deeper into the complex world of neurodegenerative and CNS disorders.