Clinical Catch-Up: September 23-27

Research

It was a moderately busy week for clinical trial news. Here’s a look.

Ipsen announced positive data from the ENGAGE Phase IIIb/IV clinical trial of Dysport (abobotulinumtoxinA) to treat upper and lower limb spasticity in adults with a Guided Self-rehabilitation Contract (GSC). The trial evaluated Dysport in patients with spastic hemiparesis in both upper and lower limbs in combination with GSC, which is a personalized diary-based rehabilitation program. The primary efficacy endpoint was percentage of patients classified as responders at the sixth week after the second injection. The study demonstrated 72.1% of patients classified as responders, hitting the predefined CXA improvement threshold in the PTT limb of equal to or greater than 35 degrees in upper limbs or greater than or equal to five degrees in the lower limbs.

Mustang Bio announced that City of Hope had treated the first patient in a Phase I clinical trial of MB-105, a prostate stem cell antigen (PSCA) chimeric antigen receptor (CAR)) T technology for prostate cancer. MB-105 came out of the research of Saul Priceman and Stephen Forman of City of Hope. The trial plans to enroll up to 33 patients. Its primary endpoints are to determine the side effects and appropriate dose of PSCA CAR-T cells in patients with pSCA-positive castration-resistant prostate cancer (CRPC) that has metastasized to other parts of the body.

Entera Bio presented positive results from its Phase II PK/PD trial of oral parathyroid hormone (PTH) drug. The data suggested that it delivered different frequencies, and with different doses, could deliver customized therapy effectively. Trial is designed to evaluate the pharmacokinetics and pharmacodynamics of two doses and three regimens of the Oral hPTH (1-34) and included subcutaneous Natpara 100 micrograms once a day. It was studied in 16 patients with hypoparathyroidism.

Avadel Pharma announced the FDA had agreed to the company’s proposal to decrease the sample size for its ongoing Phase III clinical trial of FT218 for narcolepsy. No changes were made to the fundamental design of the trial, but will now enroll 205 patients, down from the previous goal of 264 patients. FT218 has received Orphan Drug Designation from the FDA for the treatment of narcolepsy.

Avadel also released pharmacokinetic (PK) data for FT218 from four Phase I clinical trials. FT218 is a once-nightly formulation of Micropump controlled-release sodium oxybate. Various dosages are being investigated for the treatment fo excessive daytime sleepiness and cataplexy in patients with narcolepsy. The overall data from the trials showed the drug formulation has a desirable profile for once-nightly dosing. The company expects to complete enrollment of its Phase III trial of the drug by the end of this year with topline data in the second quarter of 2020.

Eloxx Pharmaceuticals announced the Cystic Fibrosis Therapeutics Development Network sanctioned its Phase II trial protocol in the U.S. for ELX-02 in cystic fibrosis. The CF Foundation is funding the program. The Phase II trial program will be two open-label, dose-escalation studies of ELX-02 in CF patients who have at least one G542X allele. It will enroll up to eight patients.

Neurana Pharmaceuticals announced positive topline results from its Phase II trial of tolperisone, a non-opioid, for acute back muscle spasm without drowsiness or sleepiness. The trial showed overall efficacy of the drug with the 600 mg dose with sleepiness similar to placebo. The drug was generally well tolerated. Tolperisone is widely prescribed outside the U.S. for acute muscle spasms and spasticity.

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GenSight Biologics reported the first group of data from Week 96 of its RESCUE Phase III clinical trial. The trial is studying a single intravitreal injection of GS010 in 39 patients who have lost vision because of 11778-ND4 Leber Hereditary Optic Neuropathy (LHON). In RESCUE, data suggests continued efficacy of the drug two years after injection, with BCVA holding clinically meaningful improvement. Patients receiving the drug had their eyesight initially get worse to a point defined as the nadir, or worst point, where it then started to recover.

Zealand Pharma announced positive results in its Phase III trial of dasiglucagon for severe hypoglycemia in diabetes. The drug is a potential first-in-class soluble glucagon analog being developed in the HypoPal rescue pen. The primary endpoint was time to plasma glucose recover. The primary result showed that time was 10 minutes for dasiglucagon, superior to placebo, which had a median of 30 minutes.

Salarius Pharmaceuticals announced the Safety Review Committees had given the go-ahead to its Phase I/II clinical trial of seclidemstat in Ewing sarcoma and the Phase I trial of the drug in advanced solid tumors (AST). Both are open-label dose-finding trials to determine the maximum tolerated dose and initial safety profile of the drug. Seclidemstat is an oral, once-daily reversible LSD1 inhibitor.

The Medicines Company announced positive topline data from its ORION-10 Phase III clinical trial of inclisiran for atherosclerotic cardiovascular disease (ASCVD) as well as its ORION-9 Phase III study of the drug in Heterozygous Familial Hypercholesterolemia (HeFH). The drug hit all primary and secondary endpoints, showing efficacy, tolerability and safety in both indications with no treatment-related liver or kidney abnormalities found in laboratory tests. Inclisiran is the first and only drug for cholesterol in the siRNA class. It prevents production of the PCSK9 protein in the liver.

Tyme Technologies announced the launch of the TYME-88-Panc trial to evaluate its lead cancer metabolism-based therapy, oral SM-88 (racemetyrosine) for third-line treatment of metastastic pancreatic cancer. It is a Phase III pivotal trial to evaluate SM-88 with metoxsalen, phenytoin, silorlimus (MPS) in metastatic adenocarcinoma of the pancreas in patients whose disease has progressed or recurred and have had two previous lines of systemic therapy.

Sequana Medical enrolled the first patient in the North American pivotal POSEIDON trial of the alfapump to treat recurrent and refractory ascites due to liver cirrhosis. It is a single-arm, open-label study that will include up to 50 patients to be implanted with the alfapump in about 15 centers in the U.S. and Canada. The primary effectiveness outcome is the proportion of patients with a 50% decrease in overall average frequency of paracentesis per month post-implantation compared to pre-implantation. The alfapump is a fully-implanted, programmable, wireless, CE-marked device that pumps ascites from the peritoneal cavity into the bladder, where it is eliminated with urine.

Aileron Therapeutics released an abstract on interim Phase IIa data from its ALRN-6924 in combination with Pfizer’s Ibrance (palbociclib) in patients with MDM2-amplified cancers. ALRN-6924 is a first-in-class dual MDM2/MDMX inhibitor. The abstract is present presented as a poster at the 2019 Congress of the European Society for Medical Oncology (ESMO) being held in Barcelona, Spain from September 27 – October 1.

Menlo Therapeutics completed patient enrollment in its Phase II trial of oral serlopitant for chronic pruritus (itch) of Unknown Origin (CPUO). Serlopitant is a small molecule, highly selective NK1receptor antagonist. The trial enrolled 233 patients.

PhaseBio Pharmaceuticals announced the completion of its Phase IIa clinical trial of PB2452. The drug showed immediate and sustained reversal of ticagrelor in older (ages 50-64) and elderly (ages 65-80) patients on dual antiplatelet therapy of ticagrelor and low-dose aspirin. Under FDA guidance, the company added a cohort for the reversal of supratherapeutic doses of ticagrelor in healthy younger subjects. In that group, the drug showed immediate and sustained reversal of ticagrelor and was well-tolerated. Statistically significant reversal of the antiplatelet activity of supratherapeutic blood levels of ticagrelor was hit within five minutes of the PB2452 infusion and was sustained for 24 hours.

Precision BioSciences announced the FDA had accepted its Investigational New Drug (IND) application for PBCAR20A, an off-the-shelf CAR-T therapy program. The therapy is in development for non-Hodgkin lymphoma (NHL), chronic lymphocytic leukemia (CLL), and small lymphocytic lymphoma (SLL). It expects to launch a Phase I/IIa trial in the fourth quarter of this year with initial data expected in 2020.

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