Covid hero Pfizer ramps up battle to save millions of sick children

Covid-19 vaccine pioneer Pfizer is ramping up its development of treatments for rare diseases that affect millions of children. 

The US pharmaceuticals giant is investing billions of dollars into research and development of therapies to treat illnesses such as spina bifida and haemophilia. 

The Mail on Sunday can reveal that Pfizer is aiming to launch gene therapies for use in the UK within two years and is piloting a system that uses artificial intelligence to flag potential rare disease in patients to doctors. 

A rare disease is defined as a condition that affects fewer than one in 2,000 people and includes childhood cancers, cystic fibrosis and Huntington’s disease. 

Pioneer: Pfizer is investing billions of dollars into research and development of therapies to treat illnesses such as spina bifida and haemophilia

They affect 3.5million people in the UK – more than the population of Birmingham, Liverpool, Nottingham and Sheffield combined. 

Some 75 per cent of rare conditions affect children and more than 30 per cent of them die before their fifth birthday. 

Pfizer’s Covid vaccine, developed with Germany’s BioNTech, was the first jab to be administered in the UK. 

The firm is working round the clock to meet demand and plans to improve production, though this will involve a temporary reduction in UK vaccine deliveries. 

But Owen Marks, the boss of Pfizer UK’s rare diseases arm, told The Mail on Sunday the $200billion company has not overlooked long-term research beyond Covid-19. 

He said: ‘There’s a compartmentalised approach… We have business units [each working on totally separate projects], which means we’re able to maintain focus across different areas and rare disease research can continue.’ 

The company is piloting tech across three UK hospitals which uses AI to scan doctors’ notes for ‘red flags’ that indicate the possibility of cardiomyopathy, a rare form of heart failure. 

The condition can often take three years to diagnose, but ‘if you use techniques like this you can reduce that dramatically, in some cases to months’, Marks said. 

Vaccine: Pfizer is working round the clock to meet demand and plans to improve production

Pfizer has invested $500million in a specialist gene therapy facility in North Carolina and has just spent $60million acquiring US specialist Homology Medicines. 

Marks said he expected to do more deals this year, adding: ‘We’ve been very active about collaborating with small biotechs. We basically look to where the best science and overlap is.’ 

Gene therapy, in which defective genes are replaced by healthy ones, has been used for three decades but progress in making the technique widely available has been slow. 

Marks said that Pfizer is running several trials, including one for boys with Duchenne muscular dystrophy, conducted remotely. 

He said: ‘We must not take our eye off the ball otherwise there will be a delay in those treatments getting to patients – we’ve got to avoid that.’ 

Pfizer hopes to launch a therapy within two years and Marks said this could be faster if the collaboration seen with BioNTech, regulators and governments worldwide on its Covid vaccine could be emulated. 

He said: ‘We’re already starting to see the urgency of the response to Covid create a degree of change which is remarkable and there is a lot that we can take from that for rare diseases.’ 

Marks’s perspective has been shaped by his 21-year-old son, who has a severe form of autism. 

The Pfizer chief has in a personal capacity signed a petition to give people with learning difficulties priority in receiving the Covid vaccine. 

Pfizer has seen its shares gain 35 per cent since the pandemic began. It has not received support from Washington to develop its vaccine, although the German state gave BioNTech €375million to accelerate the jab’s production capacity.

Alert over Biotech shares boom

Small British biotech companies may be unable to hang on to their astonishing share price gains when the Covid pandemic recedes, experts are warning. 

Analysis for The Mail on Sunday by AJ Bell found Covid tester Novacyt was the best performing testing or treatment firm over the past 12 months.

Its shares rose an eye-watering 6,890 per cent, turning £1,000 into £69,900. 

Shore Capital healthcare analyst Adam Barker said the likes of AIM-listed Novacyt, which has won Government testing contracts, would continue to do well with mass testing commonplace in the first half of this year. 

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But he said the shares could suffer if vaccines make testing less vital. He said Synairgen would hold on to its gains only if its inhaled Covid drug proved effective, adding: ‘Further gains would then come from a successful roll-out into hospitals, which isn’t always easy for smaller companies.’