News & Trends - Biotechnology

VIC biotech trials muscular dystrophy drug

Health Industry Hub | January 8, 2020 |
[Total: 1    Average: 4/5]

A Melbourne biotech company developing a treatment for a devastating genetic disorder is looking to build on the promising results of a small study at the Royal Children’s Hospital.

Antisense Therapeutics is in talks with regulators in Europe to begin a pivotal trial of its drug candidate ATL1102, a possible treatment for boys confined to a wheelchair with Duchenne muscular dystrophy.

The severe form of muscular dystrophy effects about 1 in 3,500 newborn boys, causing muscle weakness, wasting and eventually an early death.

There’s no cure but Antisense chief executive Mark Diamond is hoping ATL1102 could be a better treatment option than the high-dose steroids currently prescribed.

“We know we have to show good clinical benefits to expect approval,” he said on Monday.

“But when we get to the higher doses, we think that’s achievable.”

The company on December 17 reported that seven of nine Australian boys with Duchenne muscular dystrophy treated with a low dose of ATL1102 had either improved their grip strength or shown no change after 24 weeks.

“Disease stabilisation or indeed improvement in functional scores in non-ambulant DMD boys is almost unheard of and a very encouraging result,” Dr Thomas Voit, director of the Biomedical Research Centre in London, was quoted by the company as saying.

Antisense is hoping to begin a Phase IIb trial in Europe in the second half of the year, comparing treatment with ATL1102 to steroids over the course of a year.

With no adverse affects reported by the boys in the Royal Children’s Hospital trial, the European trial will involve a higher dose of the drug that Antisense hopes will show additional benefits.

Given the limited number of options for treating the disease, European regulators have indicated ATL1102 could be approved straight away if the results are positive, bypassing the customary additional studies.

“That’s very exciting,” Mr Diamond said, although adding that it would still be about three years before the drug could be commercialised.

ATL1102 is a weekly subcutaneous injection that works by inhibiting a cell receptor in the body’s immune system to reduce inflammation.

It’s part of an emerging medical strategy called antisense therapy that uses drugs to target a patient’s RNA and essentially silence faulty genes that cause genetic disorders.

Mr Diamond said Antisense’s drug comes from NASDAQ-listed Ionis Pharmaceuticals, a leader in antisense therapy with three drugs approved. Antisense hopes to follow in the footsteps of Boston-based Sarepta Therapeutics, which has a treatment for muscular dystrophy that works only in about 14% of cases, when patients have a particular gene mutation.

You may also like Temporary permission granted by TGA to advertise salbutamol puffers and inhalers

Register FREE to receive the latest news, innovations and insights from Health Industry Hub; the only one-stop-hub connecting Australia‚Äôs Pharma, MedTech and Biotech industry professionals and its key stakeholders.


About the author

About the author

Lorem ipsum dolor sit amet, consectetur adipiscing elit. Nam luctus finibus scelerisque. Nunc bibendum ipsum sed augue fringilla fringilla. Nullam at consectetur leo. Praesent viverra rutrum porta. Quisque vitae mi vel purus vulputate tincidunt. Class aptent taciti sociosqu ad litora torquent per conubia nostra, per inceptos himenaeos. Suspendisse et mi quis nisi rhoncus feugiat at ac tellus. Sed aliquam sodales nulla ac auctor. Sed pretium lobortis purus accumsan ullamcorper. Phasellus sodales vel odio in lobortis. Duis maximus sagittis bibendum. Interdum et malesuada fames ac ante ipsum primis in faucibus. Nunc dictum tincidunt ipsum in vestibulum. Donec ut sem consectetur, aliquam quam vitae, pharetra orci. Nam egestas non velit eu rhoncus. Duis congue neque non lacus tincidunt porta. Vestibulum ultricies pulvinar sem, molestie congue dui aliquet non.
  • Ut imperdiet leo id lorem fermentum consectetur.
  • Ut vitae orci et dui varius tincidunt.
  • Ut id magna non libero vestibulum pharetra ac faucibus nulla.
Aliquam erat volutpat. Vestibulum vitae varius diam. Nulla eget congue ante. Nunc ullamcorper sagittis augue vel dictum. Mauris finibus nibh ut pulvinar auctor. Vestibulum ut faucibus nisi. Orci varius natoque penatibus et magnis dis parturient montes, nascetur ridiculus mus. Quisque porta tortor ac justo malesuada elementum. Nulla tellus ante, cursus nec ex sit amet, suscipit bibendum arcu. Duis posuere orci dui, et mollis enim dictum at. Sed ullamcorper, sapien ut vulputate viverra, sem purus porttitor tellus, nec mattis mauris ligula sed risus. Nulla sagittis id ipsum eu mattis. Link to full profile

Social Responsibility

Social Responsibility and Community Engagement - World Cancer Day 2020

World Cancer Day 2020

Health Industry Hub | January 22, 2020 |

People across Australia will come together on 4 February to reduce the impact of cancer around the world. 2020 marks […]

More


News & Trends - Pharmaceuticals

Pharma Medical News - TGA reveals new regulatory science strategy 2020-2025

TGA reveals new regulatory science strategy 2020-2025

Health Industry Hub | January 22, 2020 |

New technologies such as gene therapies, other cell and tissue therapies, 3D-printing, and software as a medical device are transforming […]

More


Marketing & Strategy

Biopharma marketing - embracing change for growth

Biopharma marketing – embracing change for growth

Health Industry Hub | January 22, 2020 |

For years, biopharma companies have celebrated their “patient centricity”, yet only 35% of patient groups say that the industry is […]

More


News & Trends - Medical Technology

MedTech News - Medtronic to launch a game changer for patients with neurological disorders

Medtronic to launch a game changer for patients with neurological disorders

Health Industry Hub | January 22, 2020 |

Medtronic announced the CE Mark for Percept PC neurostimulator; it is the only Deep Brain Stimulation (DBS) system to be […]

More