Date: 2014-11-03
Type of information: Production agreement
Compound: Mydicar®
Company: Lonza (Switzerland) Celladon (USA - CA)
Therapeutic area: Cardiovascular diseases
Type agreement: manufacturing
production
Action mechanism: Mydicar® is a genetically targeted enzyme replacement therapy (ERT) intended to restore levels of SERCA2a, a regulator of calcium cycling in the heart and cardiac contractility. SERCA2a levels decline in all forms of late-stage Heart Failure resulting in deficient heart function. With Mydicar®, the SERCA2a gene is delivered using recombinant adeno-associated virus (AAV) as the vector. AAV is a naturally occurring virus not associated with any disease in humans. Mydicar® is delivered in a single dose directly to the heart during a routine outpatient cardiac catheterization procedure, similar to an angiogram.
Disease: advanced heart failure
Details: * On October 2, 2012, Lonza, a global leader in the field of viral therapy manufacturing, and Celladon Corporation, a biopharmaceutical company focused on the discovery and development of innovative treatments for cardiovascular diseases, have entered into a process transfer and GMP manufacturing agreement. Under the terms of the agreement, Lonza will manufacture cGMP-grade Mydicar® (AAV1/SERCA2a) for use in future clinical trials. Mydicar® is currently under clinical investigation in a 200 patient Phase 2b clinical trial in NYHA Class III/IV heart failure patients. In the event Celladon intends to commence commercial scale manufacture of Mydicar®, Celladon and Lonza have agreed to negotiate a commercial scale supply agreement.
Financial terms:
Latest news: * On January 5, 2015,Celladon Corporation, a clinical-stage biotechnology company with industry-leading expertise in the development of cardiovascular gene therapy, announced that it has conducted initial scale up of its viral manufacturing process for Mydicar® (AAV1/SERCA2a) to commercial scale. The scale up of primary production and downstream processing development to 2,000 liters was undertaken together with Lonza, in its facility in Houston, Texas. * On November 3, 2014, Lonza and Celladon announced that they have entered into an agreement providing for the future commercial production of Mydicar® (AAV1/SERCA2a), Celladon’s enzyme replacement therapy for advanced heart failure that is currently in Phase 2b clinical development. This agreement follows a successful multi-year clinical manufacturing relationship and provides for initiation of pre-construction activities and the reservation of Lonza resources giving Celladon an opportunity to trigger construction of the dedicated facility and secure a long term commercial supply arrangement. The establishment of this facility construction and commercial supply agreement provides Celladon with a strategic path to commercial supply, including plans for a dedicated cGMP production train within a new, state-of-the-art viral therapy facility. In the near term, Lonza will complete a detailed engineering design for the facility to be located in Portsmouth, NH (USA). The facility will be separate from Lonza’s existing clinical and commercial mammalian operations facility also located in Portsmouth. In exchange for a reservation fee, Celladon has the option to trigger construction of the facility with a multi-year commitment to Lonza for the supply of Mydicar®. Upon the trigger, Lonza will purchase from Celladon shares of Celladon common stock valued at $10 million. Upon completion of the new manufacturing facility, Lonza is to transfer Celladon’s 2000 liter commercial-scale process from Lonza’s facility in Houston, TX (USA) to the new facility in Portsmouth. Process validation is expected to be completed in the new Portsmouth facility. Celladon has completed enrollment of a 250 patient Phase 2b clinical trial evaluating the efficacy of Mydicar® in reducing the frequency of, or delaying heart failure-related hospitalizations. This randomized, double-blind, placebo-controlled, multinational trial is evaluating a single intracoronary infusion of Mydicar® versus placebo added to a maximal, optimized heart failure regimen in patients with New York Heart Association class III or IV symptoms of chronic heart failure due to systolic dysfunction. The Company has received Breakthrough Therapy designation from the FDA for this Mydicar® program and expects to report results from the Phase 2b clinical trial in April 2015. In addition, Celladon has identified a number of potential first-in-class compounds addressing novel targets in diabetes and neurodegenerative diseases with its small molecule platform of SERCA2b modulators.
