Scrip is part of Informa PLC

This site is operated by a business or businesses owned by Informa PLC and all copyright resides with them. Informa PLC’s registered office is 5 Howick Place, London SW1P 1WG. Registered in England and Wales. Number 8860726.

This copy is for your personal, non-commercial use. For high-quality copies or electronic reprints for distribution to colleagues or customers, please call +44 (0) 20 3377 3183

Printed By

UsernamePublicRestriction

AstraZeneca's Ultomiris Disappoints In ALS, Setting Back Rare Disease Ambitions

Phase III CHAMPION-ALS Study Discontinued

Executive Summary

AstraZeneca is discontinuing a Phase III trial of the long-acting complement inhibitor it gained via its Alexion acquisition in amyotrophic lateral sclerosis because of lack of efficacy in an interim analysis. Interest is now turning to antisense and cell therapy candidates from other companies that are in late-stage development for the condition.

You may also be interested in...



AstraZeneca Goes All In On AL Amyloidosis With Caelum Acquisition

AstraZeneca’s recently-acquired Alexion unit is paying $150m upfront to buy the remainder of Caelum Biosciences and its late-stage therapy for light chain  amyloidosis, a rare disease in which misfolded amyloid proteins build up in organs throughout the body, including the heart and kidneys.

AZ's Rare Disease Unit Reports Positive Phase III Results For Copper-Binding ALXN1840

Alexion/AstraZeneca has announced positive top-line results from the Phase III study of ALXN1840 in patients with the rare genetic disorder, Wilson disease, including in patients already on standard-of-care therapies. The compound is ahead of two candidate gene therapies from other companies at early-stage clinical development.

Argenx's Ambitious Plans For Efgartigimod On Track

The company's first-in-class FcRn antagonist, which has an FDA action date in December, is now being reviewed by the EMA and although the regulator rejected a request for an accelerated assessment of efgartigimod for generalized myasthenia gravis, commercial teams are being built up in Europe.

Related Content

Topics

Related Companies

UsernamePublicRestriction

Register

SC144934

Ask The Analyst

Ask the Analyst is free for subscribers.  Submit your question and one of our analysts will be in touch.

Thank you for submitting your question. We will respond to you within 2 business days. my@email.address.

All fields are required.

Please make sure all fields are completed.

Please make sure you have filled out all fields

Please make sure you have filled out all fields

Please enter a valid e-mail address

Please enter a valid Phone Number

Ask your question to our analysts

Cancel