March 2022 • PharmaTimes Magazine • 6

// MEDICINE NEWS //


Parkinson’s UK and Domainex join forces to develop therapies

Parkinson’s UK and Domainex have announced a collaboration focused on developing small molecule therapies targeting neuroinflammation, which could slow the progression of Parkinson’s disease.

Domainex – a leading integrated medicines research services partner – will provide fully integrated drug discovery services including assay biology, medicinal and computational chemistry.

The partnership is anticipated to be undertaken over a two-and-a-half year period and Parkinson’s UK will invest up to £3m in the project via its drug development arm – the Parkinson’s Virtual Biotech.

Inflammation is the biological response of body tissues to harmful stimuli, such as pathogens, damaged cells or irritants. It is essential for defending the body from infections, injuries and toxins. In the case of Parkinson’s, however, there is excessive chronic inflammation in the brain, which is believed to play a role in damaging brain cells.

As part of the collaboration, Domainex will conduct integrated drug discovery of molecules which target a protein found on the surface of microglia, the main immune cells in the brain that become overactive in Parkinson’s.

“We are thrilled to have been selected by Parkinson’s UK to work on this promising project which has the potential to improve the lives of 145,000 people living with Parkinson’s in the UK,” said Tom Mander, CEO of Domainex.


Addison’s disease phase 2 study begins

Diurnal has announced that the first patient has been dosed in its phase 2 European clinical trial of modified-release hydrocortisone.

It is treating people with adrenal insufficiency (AI), also known as Addison’s disease, while the trial also represents a significant marketing opportunity for the company across Europe and throughout the UK.

The CHAMPAIN phase 2 study aims to evaluate the efficacy, safety and tolerability of modified-release hydrocortisone versus Plenadren in AI. It is anticipated that it will take six months to reach completion.

Modified-release hydrocortisone is a preparation of hydrocortisone that has been specifically designed for patients with diseases of cortisol deficiency – such as AI – and additionally for congenital adrenal hyperplasia (CAH). It is approved for the latter disease in Europe and the UK under the commercial name Efmody.

AI is a long-term endocrine disorder, which affects approximately 298,000 patients in Europe and the UK. It is caused by inadequate production of steroid hormones in the cortex of the adrenal glands. AI can result in severe fatigue and – if left untreated – adrenal crisis may be life-threatening.

Martin Whitaker, CEO of Diurnal, commented: “We believe modified-release hydrocortisone has the potential to replicate the physiological overnight rise of cortisol in these patients and we look forward to the data read-out from the CHAMPAIN study in H2 2022.”


Ontozry available for patients in Scotland with drug-resistant epilepsy

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Around 55,000 people in Scotland are currently living with epilepsy. Significant treatment needs remain for those suffering from the treatment-resistant disease.

New hope, however, has been offered to adults in Scotland with uncontrolled focal epilepsy, as the Scottish Medicines Consortium (SMC) has accepted Ontozry (cenobamate) for adults with drug-resistant epilepsy.

Angelini Pharma’s cenobamate is an oral anti-seizure medicine (ASM), now available for use within NHS Scotland for eligible adults as a second-line adjunctive ASM, after the failure of the first adjunctive ASM.

The decision follows the NICE recommendation for cenobamate. In the UK, only 52% of people with epilepsy are seizure-free and those with poorly controlled seizures are more likely to experience comorbidities, social stigmatisation and poor quality of life.

“I welcome the decision on the use of cenobamate for some patients with resistant epilepsy,” commented John Paul Leach, Professor of Clinical Neurology at the University of Glasgow School of Medicine. “It marks an important step forward in epilepsy care in Scotland, giving physicians a new treatment option to help the one-third of patients who have epilepsies resistant to current anti-seizure medications.”