AzurRx BioPharma is working with CROs Rho and Linical for its Phase II and Phase IIb MS1819 trials, respectively, in cystic fibrosis patients with exocrine pancreatic insufficiency (EPI), said CEO James Sapirstein.
The CROs were chosen to manage the trials based on their respective experience in conducting cystic fibrosis studies and other rare disease programmes, explained Sapirstein. MS1819 is a recombinant lipase replacement therapy designed to be a synthetic alternative to porcine pancreatic enzyme replacement therapy (PERT). PERT is a treatment option in cystic fibrosis patients with EPI, which is the inability to properly digest food due to not enough digestive enzymes from the pancreas.
The 30-patient Phase II OPTION2 study (NCT04375878) which has sites in the US is on track to facilitate topline data in 1Q, Sapirstein added. This trial is a randomised study comparing oral MS1819 to PERT. It has coprimary endpoints investigating safety and efficacy measured through the coefficient of fat absorption at six weeks. AzurRx is collaborating with Durham, North Carolina-headquartered Rho for AzurRx’s US operations.
Meanwhile, the Europe-based Phase IIb study (NCT04302662) is on track to complete its 24-patient accrual target in 2Q but may be truncated at 20 patients if a positive signal is seen, said Sapirstein. The open-label Phase IIb trial involves all patients receiving escalating doses of a spray dried MS1819 on top of porcine pancreatic extract. It has coprimary endpoints looking into safety and coefficient of fat absorption in a 15-day timeframe.
Once OPTION2 data is released, AzurRx plans to engage a strategic licensing or development partner to conduct Phase III investigations, Sapirstein said. AzurRx has been engaging with potential partners since 2019 and has already signed several NDAs with some prospective partners having been in a data room, he said. The goal is to finalise a partner by YE21, he added. The company plans to start a Phase III trial in 1Q22, as per a 7 January presentation. AzurRx’s market cap is $31.2m.
Niclosamide development in COVID-19 in the works
In a 04 January media release, AzurRx announced a licensing deal with Delray Beach, Florida-based First Wave Bio for an oral formulation of the anthelmintic drug niclosamide for COVID-19 or immune checkpoint inhibitor-induced colitis. Following this deal, AzurRx plans to start a Phase II COVID-19 study and a Phase Ib/IIa immune checkpoint inhibitor-induced colitis trial in 1H21. The company has lined up CROs for both studies, with Sapirstein declining to comment further.
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The idea behind exploring niclosamide in COVID-19 stems from research indicating the SARS-CoV-2 virus is found in different reservoirs in the body, including the gastrointestinal (GI) tract, Sapirstein explained. In these circumstances, niclosamide could clear the virus and therefore alleviate GI symptoms in COVID-19 patients, he added. The drug is available in oral and enema formulations. An oral delivery means the drug can be used in the outpatient setting, while the enema formulation can be studied for the smaller group of COVID-19 patients who are intubated, he said.
While the trial design for the COVID-19 study still needs to be finalised following discussions with the FDA, Sapirstein mentioned the potential for efficacy measures to likely focus on GI-related measurements and SARS-CoV-2 viral load. Moreover, a large trial of 500–600 patients is unlikely, but a smaller one with a focus on those COVID-19 patients with GI symptoms, he added.
With regards to niclosamide in immune checkpoint inhibitor-induced colitis, there is a need for a nonsteroidal treatment option, as per the company presentation. The Phase Ib/IIa study will be conducted at oncology centres and focus on patients with Grade 1 or Grade 2 diarrhea who do not require hospitalisation, with the goal of reducing the incidence and weight of stools, said Sapirstein.
Manasi Vaidya is an Associate Editor for Clinical Trials Arena parent company GlobalData’s investigative journalism team. A version of this article originally appeared on the Insights module of GlobalData’s Pharmaceutical Intelligence Center. To access more articles like this, visit GlobalData.