The UK’s Competition and Markets Authority (CMA) is set to review AstraZeneca’s (AZ) planned takeover of rare diseases specialist Alexion.

The competitions regulator will be inviting comments from interested parties to aid in its assessment of the merger – a routine process for ‘major’ deals.

In December 2020, AZ announced its intention to acquire US-based Alexion for the high sum of $39bn.

The acquisition was one of the largest deals announced last year and is also AZ’s largest corporate acquisition ever, according to Reuters.

While the CMA has announced its intention to scrutinise the deal, the US Federal Trade Commission has already cleared the proposed acquisition. Competition clearances have also been completed in Canada, Russia, Brazil and other countries.

The CMA’s initial analysis will form ‘phase 1’ of the review and is likely to be completed by 21 July, although the regulator will post its decisions following this, depending on whether the deal is cleared or if it progresses to an additional ‘phase 2’ review.

“The commencement of the UK CMA’s formal review is another important step towards the closing of the proposed acquisition, which we continue to expect will be in the third quarter of 2021,” said an AZ representative.

If the deal is cleared, AZ will immediately gain access to assets within Alexion’s commercial portfolio, which are estimated to be worth approximately $6bn.

Biotech company Annovis Bio has revealed positive results for its lead investigational therapeutic ANVS401 in Alzheimer’s disease (AD) and Parkinson’s disease (PD).

The results come from a small phase 2a study evaluating ANVS401 in 14 Alzheimer’s patients and 14 Parkinson’s patients.

The results show that patients treated with ANVS401 for 25 days achieved statistically significant cognitive improvement, measured by the Alzheimer’s Disease Assessment Scale-Cognitive Subscale 11 (ADAS-Cog11).

Specifically, patients in the ANVS401-treated group saw their ADAS-Cog11 scores improve by 4.4 points from baseline until 25 days – an improvement of 30%.
The ANVS401 treatment group also showed an improvement of 3.3 points (22%) compared to placebo at 25 days.

Biogen’s aducanumab, in comparison, showed a 1.4 point improvement in ADAS-Cog13 over 18 months, while a phase 2 study of Cassava’s simufilam demonstrated a 1.6 point improvement on ADAS-Cog11 after six months.

ANVS401 is designed to inhibit the translation of multiple neurotoxic proteins – high levels of neurotoxic proteins result in impaired axonal transport, which plays a key role in the communication between and within nerve cells.

Through this inhibition, ANVS401 can improve axonal transport, reduce inflammation, protect nerve cell death and improve cognition and function, Annovis said in a statement.
Annovis is continuing to analyse the cerebral spine fluid and plasma samples of the first 28 patients from the phase 2a study, with the aim of measuring the reversal of the toxic cascade.

bluebird bio’s cerebral adrenoleukodystrophy (CALD) gene therapy Skysona has moved closer towards EU approval after gaining a positive opinion from the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP).

The CHMP has recommended marketing authorisation for Skysona (elivaldogene autotemcel, Lenti-D) for the early treatment of CALD in patients under 18 years old with an ABCD1 genetic mutation, and who do not have a matched sibling haematopoietic stem cell donor.

bluebird bio’s Skysona is a potential one-time gene therapy designed to add functional copies of the ABCD1 gene into a patient’s haematopoietic stem cells.

In the phase 2/3 Starbeam study evaluating Skysona, 90% of CALD patients met the month 24 major functional disability-free survival endpoint as of the last data cut-off date.

Major functional disabilities are the six severe disabilities commonly attributed to CALD that have the most severe effect on a patient’s ability to function independently.

In addition, 26 out of 28 evaluable patients maintained a neurologic function score (NFS) less than or equal to one until month 24, with 24 of those patients having no change in their NFS.

The CHMP’s positive opinion is now due to be reviewed by the European Commission, with a final decision for Skysona expected in mid-2021.

The UK’s National Institute for Health and Care Excellence (NICE) has recommended Alexion’s long-acting C5 complement inhibitor Ultomiris for NHS use in patients with rare blood disorders.

Final guidance has recommended Ultomiris (ravulizumab) as a treatment for paroxysmal nocturnal haemoglobinuria (PNH), while draft guidance published last month has accepted the therapy for use in atypical haemolytic uraemic syndrome.

NICE has approved Ultomiris for the treatment of aHUS and PNH patients with a body weight of 10kg or more who are complement inhibitor treatment-naive or have received Alexion’s Soliris (eculizumab) for at least three months and have evidence of response to this therapy.

Clinical trial evidence has shown that treatment with Ultomiris in people with PNH is at least as clinically effective as Soliris and it is administered less frequently – every eight weeks as opposed to every two weeks.

Ultomiris has also been found to cause fewer episodes of breakthrough haemolysis – a symptom of PNH – thereby reducing the number of hospital admissions and need for blood transfusions.

Meindert Boysen, deputy chief executive and director of the Centre for Health Technology Evaluation at NICE, commented: “We are pleased to be able to recommend [Ultomiris] for people who have paroxysmal nocturnal haemoglobinuria or atypical haemolytic uraemic syndrome. We are hopeful that the increased time between doses with this new treatment will lead to a better quality of life for these individuals and their loved ones,” he added.