Pharmaxis poprawia perspektywy leczenia mukowiscydozy

Pharmaxis poprawia perspektywy leczenia mukowiscydozy
17 grudnia 07:34 2015 Print This Article

Pharmaxis enhances outlook for cystic fibrosis treatmentsnap_002

Wednesday, December 16, 2015 by Proactive Investors

Pharmaxis has received positive trial results for a cystic fibrosis treatment

Pharmaxis has received positive trial results for a cystic fibrosis treatment

Pharmaceutical research company Pharmaxis (ASX:PXS) has reinforced the commercial prospectivity of its cystic fibrosis treatment, snap_001Bronchitol, with positive results from a recent paediatric trial.

The trial conducted across 39 centres in Canada and Europe was designed in consultation with the European Medicines Agency (EMEA) as a condition of the marketing authorisation granted for Bronchitol for treating adult cystic fibrosis patients in Europe.

Based on the encouraging results with patients aged 6 to 17,Pharmaxis will consider an application to extend the European Union marketing authorisation to include children and adolescents.

To meet the condition in full, Pharmaxis will submit a detailed study report to the EMEA in 2016.


The first scientific presentation of the results will be made at the European Cystic Fibrosis Society meeting in Switzerland in June 2016.

The trial confirmed that Bronchitol was efficacious in young patients, regardless of concomitant dornase alfa use.

The drug works by reducing the amount of mucus build-up in the lungs of patients suffering from chronic respiratory conditions.

It is a precision spray-dried form of mannitol, delivered to the lungs by a specially designed, portable inhaler.

The product is approved for marketing for patients aged over 6 years in Australia and for patients aged 18 years and over throughout the European Union.  snap_001

Marketing authorisation approvals are expected in Russia and Brazil in 2016.


Trial details

The latest Bronchitol trial involved 92 children and adolescents receiving either 400mg of the drug or a placebo twice a day for eight weeks on top of standard of care before a washout period of eight weeks followed by a further eight week treatment period on the alternate treatment.

During the Bronchitol treatment period patients had a statistically significant improvement in lung function compared to placebo.

Patients on Bronchitol also experienced about 25% fewer lung infections and exacerbations of cystic fibrosis, which is supportive of the improvements seen in earlier studies despite the short duration of this study.

In the trial subjects, Bronchitol was well-tolerated overall and had a favourable safety profile.

There was no difference in the rate of adverse events or serious adverse events between the treatment groups.


Broader portfolio

Progress with Bronchitol builds on a broader momentum for Pharmaxis, notably including a landmark sales deal earlier this year for anti-inflammatory drug PXS-4728A with Boehringer Ingelheim, one of the world’s 20 leading pharmaceutical companies.

PXS-4728A is targeted at treating diseases such as the liver related disease non-alcoholic steatohepatitis, which is commonly found in people who are overweight or obese.

Trials for the drug so far have indicated that low doses are efficacious in long-lasting inhibition of an enzyme which reduces inflammation and oxidative stress. It is rare to be able to demonstrate effective target engagement in a phase 1 study, so the fact that the long-lasting enzyme inhibition seen in the phase 1a study was reinforced when given once a day for 14 days adds to confidence in PXS-4728A.

Pharmaxis has also recorded significant financial traction in recent months, with the company’s cash holdings increasing 102% year on year at the end of September to A$50.3 million as revenues climbed 44.4% to $2.1 million.

This stronger footing coincided with new research collaboration with U.K. biotech company Synairgen (LSE: SNG) which has aimed to develop a Pharmaxis drug candidate for the treatment of the fatal lung disease idiopathic pulmonary fibrosis.

By sharing the risk and cost of the preclinical and clinical development on a drug for this disease, Pharmaxis is able to increase the overall number of amine oxidase chemistry based programs it is pursuing and increase its opportunities for success.

Analysis

The latest clinical trial for Bronchitol is encouraging since the positive results were seen in a group of patients with a range of genetic subtypes, helping to reinforce the view that Bronchitol has a clear place in the treatment of cystic fibrosis.

The placebo effect seen in this study is minimal and has therefore provided important and reassuring additional evidence on the benefit of Bronchitol in the paediatric and adolescent population.

It has also highlighted that the results of the earlier phase 3 studies – where a control effect was seen in younger patients – may have been understated.snap_001

Positive progress for Bronchitol in Europe also reflects well on Pharmaxis’ plans to tap the U.S.  cystic fibrosis market, which is the biggest in the world.

Pharmaxis is already advancing trials in the U.S. for Bronchitol with its partner Chiesi, which is responsible for regulatory filing and commercialisation drives in the region.

A Bronchitol launch in the U.S. is expected in 2017.

Pharmaxis benefits from a strong cash position of A$50.3 million as of the end of September, with significant value milestones from existing partner deals within reach.

Potential price catalysts for the company in 2016 include completion of trail work with Chiesi in the U.S. and completion of the pre-clinical program with Synairgen.

Progress related to Pharmaxis’ drug trialling successes and new partnerships this year (including the agreement with Boehringer Ingelheim) has had a dramatic effect on the company’s share price, with a 172% increase in the value of the stock since February.

This growth has been characterised by a growing institutional presence to more than 45% of the share register.

Proactive Investors Australia is the market leader in producing news, articles and research reports on ASX emerging companies with distribution in Australia, UK, North America and Hong Kong / China.

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