Clinical Catch-Up: Jan 20-24

Clinical research

The week after the JP Morgan Healthcare Conference tends to be slower than the week of, and last week was no outlier. Still, there were clinical trial updates. Here’s a look.

Verrica Pharmaceuticals presented positive data from a post-hoc pooled analysis of its Phase III CAMP trials of VP-102 for molluscum contagiosum. The data showed the percentage of patients with complete clearance was statistically higher in the VP-102 group than vehicle across all body regions. VP-102 (cantharidin 0.7% topical solution) is a potential first-in-class topical treatment of molluscum contagiosum and common warts.

Hutchison China MediTech (Chi-Med) announced that the independent Data Monitoring Committee of the Phase III trial of surufatinib in advanced neuroendocrine tumors – pancreatic (SANET-P) completed a pre-planned interim analysis. The IDMC recommend the company halt the trial early because the pre-defined primary endpoint of progression free survival (PFS) had already been met. The company now plans to set up a pre-NDA meeting with the China National Medical Products Administration to discuss preparing the NDA. Surufatinib is a novel, oral angio-immune kinase inhibitor that selectively inhibits TKA associated with VEGFR and FGFR and CSF-1R.

Anokian announced the FDA had accepted its IND for KAN-101 for celiac disease. The company plans to initiate its multi-center Phase I trial in the first quarter of this year. KAN-101 is an immune modulator developed for autoimmune diseases.

Rafael Pharmaceuticals is collaborating with Michigan Medicine to begin a Phase Ib/II trial of devimistat (CPI-613) in combination with gemcitabine and cisplatin for biliary tract cancer. The trial will study gemcitabine and cisplatin with or without devimistat as first-line treatment for locally advanced, unresectable or metastatic biliary tract cancer patients who have not had previous treatment. Devimistat is a first-in-class compound that targets enzymes involved in cancer cell energy metabolism located in the cancer cells’ mitochondria. It targets the mitochondrial tricaroxylic acid (TCA) cycle.

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CytoDyn announced additional promising data from its clinical trials of leronlimab for metastatic triple-negative breast cancer (mTNBC) and metastatic breast cancer (MBC). New data from the first patient in the mTNBC Phase Ib/II trial continued to show no detectable levels of circulating tumor cells or putative metastatic cells in the peripheral blood after 15 weeks of treatment with leronlimab with carboplatin. The second patient, who was enrolled through an emergency IND with stage 4 HER2+ MBC that had metastasized to the liver, lung and brain, demonstrated a 50% shrinkage of the primary tumor and no new metastasis in the brain after receiving leronlimab as a monotherapy. Leronlimab is a humanized IgG4 mAb that blocks CCR5.

Neurotrope completed analysis of the data from its recently reported Phase II trial of bryostatin-1 in Alzheimer’s patients. They indicated there was a significant imbalance of 4.8 points in the baseline Severe Impairment Battery scores between the Bryostatin-1 group and placebo group. Statistical experts advised the company that in a such a small study, the imbalance could prevent a definitive analysis of the drug’s treatment versus placebo at the primary Week 13 point and secondary endpoints. So they conducted a post-hoc analysis using paired data for individual patients, with each patient as his or her own control. This found a statistically significant improvement over baseline, 4.8 points, in the mean SIB at week 13 in patients receiving Brystatin-1. There was, however, also a statistically significant increase in the placebo group.

Vaxart announced results from its H1 influenza oral tablet vaccine challenge. The trial demonstrated a single dose with the Vaxart oral tablet H1 influenza vaccine was well tolerated and gave statistically significant protection against H1 influenza infection in the human challenge model. Efficacy data trended favorably compared to Fluzone, which was the active comparator in the study.

LipoMedix announced Phase I data of Promitil (PL-MLP) in metastatic colorectal cancer. The study looked at 53 patients with advanced, treatment-refractory CRC receiving Promitil as a single agent or in combination with capecitabine and/or bevacizumab. The drug was well tolerated with a good safety profile. The therapy showed a substantial rate of disease stabilization. Promitil allows efficient and selective delivery of mitomycin-c lipidic prodrug (MLP) with rapid activation to a mitomycin-c metabolite.

BlackThorn Therapeutics dosed the first patient in its Phase II trial of BTRX-33514440 in major depressive disorder. The drug is a selective antagonist for the kappa opioid receptor (KOR). The trial is a double-blind, placebo-controlled, randomized proof-of-concept trial.

Leap Therapeutics presented updated clinical data from its recently wrapped Phase I/II trial of DKN-01 in advanced or recurrent esophagogastric cancer (EGC). Primarily, there was updated response and survival data in the checkpoint inhibitor naïve gastric/gastroesophageal junction adenocarcinoma subgroup of patients receiving DKN-01 plus Merck’s Keytruda (pembrolizumab). There was a strong correlation between response and survival in patients whose cancer expressed high levels of DKK1, the target of DKN-01.

Biohaven Pharma indicated the FDA gave it the go-ahead for its ALS trial of verdiperstat. The trial will begin with its collaborators at the Sean M. Healey & AMG Center for Amyotrophic Lateral Sclerosis (ALS) at Massachusetts General Hospital. Biohaven has already begun a separate trial of the drug in multiple system atrophy. Verdiperstat is a first-in-class, oral, brain-penetrant, irreversible inhibitor of the MPO enzyme.

Genentech, a Roche company, announced positive topline results from its pivotal Part 2 of the FIREFISH study looking at risdiplam in infants aged 1-7 months with Type 1 SMA. The primary endpoint of Part 2 of the FIREFISH trial was the proportion of infants sitting without support for at least five seconds at 12 months of treatment. It was assessed via the Gross Motor Scale of the Bayley Scales of Infant and Toddler Development – Third Edition (BSID-III). Risdiplam is a survival motor neuron-2 (SMN-2) splicing modifier. It is engineered to increase and sustain SMN protein levels throughout the central nervous system and peripheral tissues.

The company also announced that its Phase III IMvigor010 trial of Tecentriq as a post-surgery monotherapy didn’t hit its primary endpoint in muscle-invasive urothelial cancer (MIUC). The primary endpoint was disease-free survival (DFS). The safety signals were consistent with the known safety profile

Ipsen paused patient dosing in its Phase III trial of palovarotene in fibrodysplasia ossificans progressive (FOP), as well as an ongoing Phase II extension study. The decision to pause dosing was based on data of a futility analysis reviewed by the Independent Data Monitoring Committee (IDMC). It suggested that the Phase III trial was unlikely to meet its primary efficacy endpoint, change in new HO volume compared to Natural History Study. Palovarotene is a RARgamma agonist.

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