Clinical Trials

Semaglutide 7.2mg bests placebo for weight loss at 72 weeks, but with efficacy lower than seen with Novo Nordisk’s CagriSema or Lilly’s Zepbound.

Three parallel Phase III studies of iclepertin aiming to improve cognitive function have failed but optimism about other schizophrenia treatments is growing.

The company is moving quickly to bring its antibody oligonucleotide conjugates to patients in three separate muscular dystrophy diseases, causing unease for its rivals.

On the back of a stellar showing in Phase III, apitegromab is going to be filed in the US and Europe in the coming weeks for spinal muscular atrophy and CEO Jay Backstrom believes it will be a $2bn blockbuster.

With positive Phase IIb/III data for IGF-1r inhibitor linsitinib, privately held Sling plans to move into Phase III and compete in thyroid eye disease on convenience and safety.

The company believes its WVE-007 can improve on GLP-1 agonists by preserving muscle mass while also not interfering with the pleasure of eating. Promising mouse data are still to be confirmed in humans, however.

Pipeline Watch is a weekly snapshot of selected late-stage clinical trial events and approvals announced by pharmaceutical and biotech companies at medical and industry conferences, in financial and company presentations, and in company releases and statements.

Productive talks with the US FDA after securing Regenerative Medicine Advanced Therapy designation haves speeded up development of bemdaneprocel, which is about to go into a registrational trial to see if it can slow down or even stop progression of the neurodegenerative disease.

Throughout 2024, Chinese firms as sole sponsors initiated a total of seven global Phase III trials that including US sites. BeiGene, Genfleet and InnoCare are expected to add four similar studies this year.

With positive Phase II data for its amisulpride derivative, LB is planning a Phase III program to show safety and convenience with a lower dose that passes more easily through the blood-brain barrier.

With new Phase I/II data for its anti-IGF-1 agent lonigutamab, Acelyrin will move into Phase III in thyroid eye disease. But how competitive it will be versus Tepezza or Viridian’s Phase III candidate is uncertain.

Two treatments that target elF2B, a regulator of the integrated stress response that appears to be overactive in the progressive motor neurone disease, have missed their primary and secondary endpoints in a landmark platform trial.

Topline Phase III results in EGFR exon 19 deletion/L858R substitution mutation NSCLC indicate more than a year in overall survival improvement over Tagrisso, the current standard of care.

From gene therapies to small molecule drugs, investors seemed to have eschewed the risks of biotech company investment propositions in 2024 but last year’s disappointments provided good lessons going forward.

Taiwan-based PharmaEssentia hopes to add essential thrombocytopenia to the label for its pegylated interferon product, already approved to treat polycythemia vera.

The Swiss-headquartered, NASDAQ-listed group has posted a Phase II win for OCS-05 in acute optic neuritis, which opens up its potential in additional, and much larger, neuro-ophthalmic indications, CEO Riad Sherif tells Scrip.

While quieter than some recent years, 2024 did produce several clinical trial readouts destined to have an impact on their respective markets. Here, in roughly chronological order, Scrip takes a look at the 10 clinical stories that got readers clicking the most over the past 12 months.

Pipeline Watch is a weekly snapshot of selected late-stage clinical trial events and approvals announced by pharmaceutical and biotech companies at medical and industry conferences, in financial and company presentations, and in company releases and statements.

A flurry of big readouts are expected in the supercharged obesity/diabetes space, with Novo Nordisk looking to make up ground against its rival Eli Lilly.