The European Commission has granted Orphan drug status for Alterity Therapeutics’ (ASX:ATH) lead molecule, PBT434, for the treatment of Multiple System Atrophy (MSA).

That gives Alterity 10 years of market exclusivity in the European Union for PBT434 in the treatment of MSA, as well as other benefits including assistance in developing clinical protocols, reduced fees and access to EU-funded research grants.

MSA is a rare neurodegenerative disorder caused by the degeneration of nerve cells in the brain and characterised by tremors, slow movement, muscle rigidity, and postural instability (collectively known as parkinsonism) and ataxia.

Orphan drug status is given to treatments for rare diseases, so called because due to their limited market few pharmaceutical companies pursue research into such products.

“This is an important milestone for Alterity as we advance PBT434 toward patient studies and it follows Orphan Drug designation from the US FDA for treating MSA,” Dr David Stamler, the company’s chief medical officer and senior VP for clinical development, said.

“It reinforces the dire need for treatment options for this particularly debilitating disease and supports our efforts in preparing for our phase two clinical trial.”

Last year the company announced phase one clinical trial results where PBT434 was found to be safe and well tolerated in adult and older adult patients.

It has also been found to inhibit the build up of alpha-synuclein protein in the brain, preserve neurons and improve motor function in pre-clinical models of Parkinson’s and MSA.


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GI Dynamics (ASX:GID) says a study from the University of Freiburg in Germany indicates its EndoBarrier implant improves cardiovascular risk biomarkers and predicted four-year risks of major cardiovascular events. Endobarrier is an intestinal sleeve that causes food to be absorbed lower down the intestinal tract. This helps aid weight loss and reduction of blood sugar levels in patients with type 2 diabetes.

Volpara (ASX:VHT) cofounder Sir Mike Brady is retiring from the board. The UK-based scientist has been on the board for a decade. Cofounder Ralph Highnam remains as CEO of the company, which is one of the top biotechs on analyst and broker lists for 2019 and 2020.

It’s tax refund season. Orthocell (ASX:OCC) just got $2.9m which it plans to spend on getting regulatory approvals for its bone and soft tissue regeneration tech CellGro, and the development of its tendon and cartilage cell regeneration tech Ortho-ACI. PharmAust (ASX:PAA) got $713,000, to spend on more clinical trials for dog and human cancer treatments.

PainChek (ASX:PCK) has got a patent in Japan for its smart phone pain assessment and monitoring software.

Anatara Lifesciences (ASX:ANR) had $4.3m in cash at the end of December. It says it will start a human clinical trial this year for its GaRP dietary supplement for chronic bowel conditions, and expects to sign a licensing deal for its non-antibiotic animal product for gut health, Detach, by the end of June. It’s also looking to buy an already-on sale gut health product.