Clinical-stage biotechnology company PharmAust PPAA has been granted small and medium-sized enterprise (SME) status by the European Medicines Agency (EMA). PharmAust is now eligible to receive regulatory fee incentives and additional support from the EMA, streamlining its regulatory engagement and the companys ability to advance its monepantel (MPL) treatment for motor neurone disease (MND)/amyotrophic lateral sclerosis (ALS). Managing director Dr Michael Thurn said the SME status enables eligible companies to receive a range of financial benefits, including reductions in fees for scientific advice, inspections and pre- and post-marketing authorisation applications. Productive new partnership This signals the beginning of a productive partnership with the key drug regulator in Europe and an important step towards accelerating start-up activities for our adaptive Phase 2/3 Strike study, Dr Thurn said. The incidence of MND/ALS in European countries is amongst the highest globally, ranging from 1.5 to 3.8 per 100,000 people [and yet] there is only one drug riluzole approved for the treatment of all types of MND/ALS in Europe, which only offers patients an additional 2-3 months in life expectancy. The SME status from the EMA provides continued momentum in our efforts to help patients and their families impacted by MND/ALS. Orphan drug designation The SME grant follows PharmAusts receipt of an orphan drug designation (ODD) for MPL from the US Food and Drug Administration (FDA) in May. The ODD status provides for the FDA to assist and encourage companies to develop safe and effective treatments for rare (impacting fewer than 200,000 persons in the US) diseases and disorders. Like the SME status, ODD recognition provides incentives including tax credits and grants, the waiving of some administrative fees for clinical trials and seven years of market exclusivity following drug approval. PharmAusts application was based on preclinical mechanistic data which demonstrated that MPL can induce autophagy in diseased cells, taking into consideration the pathology of the disease. Market support The investment market is also backing PharmAusts MPL plans, with the company receiving commitments to raise $10 million via an institutional placement in June. The companys directors and management participated with approximately $1m pledged. PharmAust is also seeking to raise a further $2m via a share purchase plan being offered to eligible shareholders. Study success continues Earlier in the month, the company unveiled the results of a new study that found treatment with MPL can reduce the risk of death by 91% when compared to PRO-ACT matched controls. The study, undertaken by specialists at Berry Consultants, found that MPL provided a statistically significant survival benefit compared to untreated matched controls from the pooled resource open-access ALS clinical trials (PRO-ACT) database for patients with MND and ALS.

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