Authored by Karl Simpson
Last week also bought news from another company with a product in the DMD area, Santhera Pharmaceuticals. The company released news that the Phase III DELOS (DuchEnne Muscular Dystrophy Long-term IdebenOne Study) study of orally administered Catena® in patients with Duchenne successfully passed a planned futility and safety analysis.
Patients with Duchenne not only suffer skeletal muscular weakening reducing ambulation, but they often suffer increasing respiratory and cardiac complications. This can lead to considerable weakness in the respiratory muscles and problems clearing airways, developing into restrictive pulmonary syndrome and respiratory insufficiency. It is this respiratory condition which Santhera wishes to test its Catena® drug against in an expanded Phase III study. In the Phase IIa DELPHI study, a doubleblind randomized placebo-controlled clinical trial was conducted to investigate the tolerability and efficacy of idebenone therapy in children with DMD. Twenty-one DMD patients (aged 8–16 years) were randomly assigned to daily treatment with 450 mg idebenone (Catena®) (n = 13) or placebo (n = 8) for 12 months. Idebenone was safe and well tolerated in all patients and had significant treatment effect on the respiratory peak expiratory flow of patients. The Phase IIa study was conducted in patients between ages 8-16 years, whereas the planned Phase III study will be conducted in patients recruited from the US and Europe between 10 and 18 years.
Many of the drugs tested in Duchenne are tested against the primary endpoint of the 6MWT (6 minute walk test), however, Santhera have agreed with both the FDA and the European Medicines Agency (EMA) to respiratory endpoints. With their intended indication to alleviate respiratory complications in the mid-late stages of the disease, Santhera have agreed with the agencies that the primary endpoint will be the measurement of the Peak Expiratory Flows (PEF). Santhera will be testing the drug in Duchenne patients of any mutational etiology, ambulatory or non-ambulatory. The study will sequentially enrol two cohorts of patients, 60 corticosteroid non-using and 200 corticosteroid using patients, allowing separate testing of efficacy and analysis in the final primary endpoint.
Santhera’s small molecule drug, Catena® is an orally available medication and therefore easily administered, which is a distinct advantage in providing treatment for this disease. Because the drug is not dependent on any particular genetic mutation in Duchenne patients, it is potentially a product which could provide a treatment option for all patients living with Duchenne. Santhera is already partnered with Takeda for Catena® in Europe, however, in discussions with Liftstream, Santhera Pharmaceuticals’ CEO, Dr. Thomas Meier told Liftstream “the time has come to intensify efforts to find a suitable partner in the U.S who could co-develop the drug and ultimately commercialise it, although Takeda might fulfil this U.S. partner role too’. Dr. Meier will lead the partnering effort with support from Santhera’s advisors Piper Jaffrey.
With many different advancing clinical programmes in the Duchenne area, Santhera brings forward another programme into Phase III clinical trials for this rare disease. Clearly Santhera believes it can optimise the success of this programme by working in partnership with another organisation, utilising their expertise and knowledge.
To download a free copy of the special report: The Pathway to New Therapies – Duchenne, please visit: https://www.liftstream.com/novel-therapies-in-dmd.html