Valorem Life Sciences

Tusk Therapeutics to be acquired by Roche

Posted on September 28th, 2018

Tusk Therapeutics Ltd (“Tusk”) was today acquired by Roche. Tusk has developed an antibody with a novel mode of action aimed at depleting regulatory T-cells (Tregs). Tregs suppress immune responses, including those against cancer cells. Preclinical data has shown that depleting Tregs from the tumor microenvironment can enhance and/or restore anti-tumor immunity. Tusk’s antibody has been designed to deplete these harmful Tregs, while not interfering with other immune cells acting against the tumor. Tusk’s program is expected to start clinical trials in cancer patients towards the end of 2019.

Tusk was founded in 2014 by Droia Oncology Ventures, Tusk’s majority shareholder. Under the terms of the agreement, Tusk’s shareholders will receive an upfront cash payment of Euro 70 million, plus additional contingent payments of up to Euro 585 million based on achievements of certain predetermined milestones.

Luc Dochez, Chief Executive Officer of Tusk Therapeutics, said: “We are delighted that Roche will further develop this novel antibody and drive the development ahead. The remaining portfolio of our immune-oncology targets will be further developed by Black Belt Therapeutics, a newly formed company spun out of Tusk Therapeutics.”

Source: GlobeNewsWire

PAH patients in England denied access to Actelion’s Uptravi on NHS

Posted on July 10th, 2018

Actelion’s Uptravi will not be funded on the NHS for patients with Pulmonary Arterial Hypertension (PAH), after the drug failed to make it into NHS England’s latest round of specialised commissioning approvals.

The drugmaker has slammed the decision, which it argues will delay access to Uptravi (selexipag) as a combination treatment for PAH in adult patients with one of the more severe forms of the disease who are insufficiently controlled on oral treatment.

“This ruling will create a disparity in access to selexipag throughout the UK, with patients in Scotland and Wales already having access,” it stressed.

The Scottish Medicines Consortium backed NHS funding for the drug back in May, but restricted its use to combination therapy in a sub-population of patients with PAH specifically those in WHO FC III who are insufficiently controlled with an ERA and a PDE-5 inhibitor and who would be considered for treatment with inhaled iloprost.

This was followed by a green light from the All Wales Medicines Strategy Group (AWMSG) in June, which recommended the drug as part of a triple combination therapy for the treatment of the condition, in adults with one of the more severe forms of the disease who are insufficiently controlled on oral treatment with two other classes of PAH medicines.

PAH is a progressive disease with high rates of morbidity and mortality affecting around 6,000-7,000 people in the UK. It is characterised by abnormally high blood pressure in the arteries between the heart and lungs, causing symptoms such as such as breathlessness, fatigue, weakness and angina.

While options are available to address these symptoms there is currently no treatment that slows progression or cures the disease, and fewer than 40 percent of patients live beyond five years of diagnosis.

Uptravi’s European approval in May 2016 came on the back of data from the Phase III GRIPHON study showing that the drug, a selective IP prostacyclin receptor agonist, delayed the time to a first morbidity or mortality event compared to placebo.

In the trial, exposure to the drug was up to 4.2 years with a median duration of exposure of 1.4 years; the risk of this primary composite end-point was reduced by 40 percent with Uptravi compared to placebo (a relative risk reduction of 16 percent).

“Selexipag is a step-change in the treatment of these patients as it is the first oral medicine targeting the important prostacyclin pathway, which offers hope for those who have had insufficient benefit from existing oral treatments. It is therefore extremely disappointing that PH clinicians will not be able to offer this treatment to patients,” said Dr John Wort, clinical lead for pulmonary hypertension at the Royal Brompton & Harefield NHS Trust.

Also expressing disappointment at NHS England’s decision, Dr Iain Armstrong, chair, Pulmonary Hypertension Association UK called for an “urgent review” on decisions regarding new therapies available for PH currently blocked or deferred by national funders.

“As a PH community we don’t ask for much; we just want the same equality and access to treatment as other disease areas like cancer. We are extremely disappointed by this decision and as a dynamic patient organisation we will continue to fight for equality and equity of access to service provision in the UK.”

Source: Pharma Times

Rare disease therapy Crysvita to be barred from NHS

Posted on June 20th, 2018

Children and young people with X-linked hypophosphataemia (XLH) are unlikely to get routine access to Kyowa Kirin’s Crysvita on the NHS.

In draft guidelines, the National Institute for Health and Care Excellence has rejected NHS funding for the drug, after concluding that the most likely cost-effectiveness estimates are much higher than would normally be considered value for money for highly specialised technologies.

There are around 250 children and young people in England with XLH, an inherited genetic disorder that causes low levels of phosphate in the blood. This leads to soft, weak bones, and children with the condition usually have bowed or bent legs, short stature, bone pain and delayed walking, and may also have dental problems and hearing loss.

Current treatment mainly consists of vitamin D supplementation and oral phosphate, and is aimed at improving growth, lessening the severity of symptoms and preventing skeletal abnormalities.

Crysvita (burosumab) is an anti-FGF23 fully human monoclonal antibody, and the first treatment to target the underlying pathophysiology of XLH.

An injection given every two weeks from the age of one until the skeleton stops growing, aims to increase reabsorption of phosphate from the kidney and, through vitamin D production, improve intestinal absorption of calcium and phosphate.

However, while evidence suggests the drug is more clinically effective than conventional therapy and provides clinical benefits in children aged between one and 12 years, the data is “limited and uncertain”, NICE said, and also highlighted a lack of data in young people aged between 13 and 17 years.

Also, while the committee recognised that there may be some lifelong benefits from Crysvita because it can prevent irreversible bone damage, the long-term consequences of disease progression – which the drug may not affect – are uncertain, the Institute said.

Final guidance is expected in October.

Source: Pharma Times