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Clinical Catch-Up: Updates in Solid Tumors, ALS, Migraine and More

By Vanessa Doctor, RN · Sep 22, 2022 · BioSpace

It’s been a busy week across the clinical development space, with Alaunos Therapeutics, Satsuma Pharmaceuticals, NeuroSense, Emyria and more reporting updates in solid tumors, migraine, amyotrophic lateral sclerosis (ALS) and MDMA drug discovery. 

Here are the highlights.

Sleeping Beauty Shows Optimistic Tumor Regression

Early clinical data from the first patient in Alaunos Therapeutics‘ ongoing TCR-T Library Phase I/II study showed the potential of its non-viral therapy to treat solid tumors.

The ongoing open-label, dose-escalation trial is testing the Sleeping Beauty TCR-T cell therapy on patients with non-small cell lung, endometrial, ovarian, colorectal, bile duct and pancreatic cancers. Trial participants all have a matching human leukocyte antigen (HLA) and hotspot mutation pairing in the company’s TCR-T library.

Researchers observed a tumor regression rate of 46.3% at six weeks and more than 51% at 12 weeks in one patient with NSCLC. The therapy also had a manageable safety profile and was well-tolerated.

Key details on this first patient will be shared at the CRI-ENCI-ACCR Sixth International Cancer Immunotherapy Conference in New York, Sept. 28 to Oct. 1. The trial is being conducted at The University of Texas MD Anderson Cancer Center.

Adamis Halts COVID-19 Trial 

Adamis Pharmaceuticals failed to achieve the primary endpoint in its Phase II/III clinical trial on the effects of tempol (MBM-02) in high-risk subjects diagnosed with early COVID-19 infection versus placebo.

Despite not having any safety concerns, the company is halting the trial early due to lack of efficacy after an independent Data Safety Monitoring Board recommendation.

Adamis will continue to analyze the data from the 248 participants enrolled to assess whether tempol may be used in other COVID patient populations, including those who are immunocompromised or suffering severe illness, the company shared. 

Emyria and PsychoGenics Partner on MDMA Drug Discovery

Australian clinical-stage biotech firm Emyria is partnering with AI drug discovery and research company PsychoGenics to advance its MDMA-inspired new drug discovery program in the United States.

Using its SmartCube platform, PsychoGenics will study novel compounds from Emyria’s program developed with the University of Western Australia.

The results are expected to help Emyria identify promising drug candidates for clinical trials. It already has the U.S. Drug Enforcement Agency’s approval to import the initial compounds into the country.

Satsuma to Seek Approval of Migraine Therapy 

Satsuma Pharmaceuticals shared positive efficacy, safety and tolerability results from its ongoing Phase III ASCEND trial of STS101 to treat migraine attacks over six and 12 months.

These outcomes, along with positive data from a Phase I study and upcoming data Phase III SUMMIT efficacy trial, support Satsuma’s bid for a New Drug Application with the FDA in the first quarter of 2023, the company shared.

Satsuma is seeking marketing approval for STS101Mk2, which incorporates the improved, second-generation nasal delivery device. The data is based on observations of over 1,600 participants over the course of 18 months. Details of the ASCEND study are published in

Verismo Announces Approval of Phase I Cancer Trial

The FDA has cleared Verismo Therapeutics‘ Investigational New Drug application (IND) to begin a Phase I clinical trial of SynKIR-110 in patients with mesothelin expressing ovarian cancer, cholangiocarcinoma and mesothelioma.

The STAR-101 study will be powered by the KIR-CAR platform, a dual-chain CAR T cell therapy that has the ability to maintain antitumor T-cell activity even in tough solid tumor environments. 

The platform may also be combined with other emerging technologies, including allogeneic cellular therapies, combination therapies, advanced T cell selection and in-vivo gene editing.

Verismo will start enrolling participants in the first quarter of 2023 at the Hospital of the University of Pennsylvania, its initial clinical site.

NeuroSense Toxicology Study Supports ALS Trial Design 

NeuroSense Therapeutics announced positive outcomes from its good laboratory practices toxicology study of celecoxib and ciprofloxacin (PrimeC) in ALS.

The FDA required NeuroSense to conduct a 90-day toxicology trial in rodents. PrimeC was given to the subjects orally twice a day. At the end of the study period, neither of the cohorts — celecoxib alone, ciprofloxacin alone and combination — manifested adverse toxicological effects. 

The study, which utilized Sprague-Dawley rats, is supportive of the company’s current Phase IIb PARADIGM study design, according to NeuroSense.

NeuroSense is currently enrolling patients for the human trial, with topline data expected to be released by the first half of 2023.

Alterity Kicks Off Phase II Trial in MSA

The FDA approved Alterity Therapeutics IND to evaluate its lead candidate ATH434 in patients with multiple system atrophy (MSA).

The IND approval authorizes the company to begin the Phase II clinical study in the U.S.

The trial will look into the effect of ATH434 on neuroimaging and protein biomarkers relevant to MSA pathology. Participants will use wearable sensors so researchers can track motor parameters.

Around 60 adult patients will be enrolled in the study to receive one of two dose levels of ATH434 or a placebo over a period of 12 months. Outcomes from this trial will determine whether or not the study will advance to Phase III.

ATH434 has an orphan drug designation for MSA from the FDA and the European Commission.