Serendex Pharmaceuticals A/S : receives positive scientific advice from EMA

Serendex Pharmaceuticals A/S has received positive scientific advice from the European Medicines Agency (EMA) on the pre-clinical and clinical development of granulocyte macrophage colony stimulating factor (GM-CSF), molgramostim, for the treatment of autoimmune pulmonary alveolar proteinosis (aPAP).

The scientific advice from EMA fully supports the current development plan for aPAP containing one pivotal Phase II/III trial with an estimated start in H2 2015 and an estimated Marketing Authorization in H2 2018.

Molgramostim is a designated orphan medicinal product in aPAP and the EMA scientific advice fully recognises the high unmet medical need in this patient population, endorsing the aggressive development plan.

Specifically, the scientific advice from EMA endorses Serendex pre-clinical programme and consider it sufficient for the planned clinical trial and for a MAA (Marketing Authorisation Application). In addition, EMA has approved the concept of one pivotal trial in aPAP and has also endorsed the suggested study design.

Based on the scientific advice from EMA, Serendex will continue the toxicology program for GM-CSF in accordance with the guidance, and progress into a Phase I clinical trial for GM-CSF in-line with expectations. This is set to form the basis for Serendex phase II/III trial for GM-CSF in aPAP in Europe.

For further information, please contact:
Kim Nielsen, CEO Mobile [phone] E-mail: [e-mail]

Background information on GM-CSF and aPAP patients
PAP is a rare disease characterized by the accumulation of surfactant within alveolar macrophages and alveoli, which impairs pulmonary gas transfer. Clinical severity ranges from an asymptomatic clinical presentation to respiratory failure and death. More than 90% of reported cases are aPAP, which is associated with high levels of autoantibodies against GM-CSF in alveoli and blood that neutralize the normal biologic action of GM-CSF in the lungs. Autoantibodies to GM-CSF are also found in healthy subjects but the levels are far lower than seen in aPAP patients indicating that these autoantibodies may be the direct cause of GM-CSF dysfunction in aPAP. In patients with aPAP, autoantibodies to GM-CSF appear to be the direct cause of GM-CSF dysfunction and local supplementation with rhGM-CSF through Molgradex is expected to have positive effects in these patients.ctations will be included in the updated budget presented to the Board by end of February 2015.

About Serendex Pharmaceuticals A/S

We are listed at Oslo Axess and develop drugs to treat respiratory conditions such as PAP (Pulmonary Alveolar Proteinosis), BE (Bronchiectasis), CF (Cystic Fibrosis related lung infections) and DAH (Diffuse Alveolar Hemorrhage). The conditions have an acute or chronic and a high medical need, we have therefore obtained Orphan Drug Designation (ODD) for many of our indications in both Europe and USA.

Common for all our drugs is that they are inhaled or induced to the lungs of the patient. This improves the risk/efficacy ratio of the drug and treatment.

The development strategy is a reposition strategy, e.g. finding new indications and administration routes for existing drugs. Repositioning reduces the risk and the cost of the development process thus reducing the time to market.

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