Poolbeg Pharma gets FDA support for POLB 001 drug
Cancer immunotherapy-induced CRS is a severe side-effect that occurs in more than 70% of patients and which may lead to multi-organ failure and death. There are currently no approved preventative therapies for CRS.
Poolbeg focusses on the development of innovative medicines to address unmet medical needs and its programmes target large addressable markets including, cancer immunotherapy-induced Cytokine Release Syndrome (CRS) and metabolic conditions such as obesity.
The company said the FDA grants orphan status to support the development of medicines for rare disorders affecting less than 200,000 people in the US.
It provides Poolbeg with clinical development and commercialisation benefits including the potential for a seven-year period of US market exclusivity after regulatory approval of POLB 001, potential waiver exemption of Prescription Drug User Fee Act application fees and the potential for tax credits for qualifying clinical trials.
Poolbeg expects the first patient to be dosed in its proposed Phase 2a in the second half of this year, with interim analysis expected in the first half of 2026 and Phase 2a topline data in the second half of 2026.
Jeremy Skillington, the chief executive of Poolbeg Pharma, said that POLB 001 is potentially a breakthrough, orally delivered, preventative therapy for cancer immunotherapy-induced CRS which could significantly impact patients' lives.
"We were delighted to receive Orphan Drug Designation from the FDA, which is a significant development for Poolbeg and for POLB 001, one that we believe will enhance the commercial appeal for prospective partners and help bring POLB 001 to the market faster," he said.
"If approved, we believe POLB 001 has the potential to improve quality of life for patients, reduce pressure on healthcare systems, and expand access to cancer immunotherapies," the CEO added.
Hashtags
Try Our AI Features
Explore what Daily8 AI can do for you:
Comments
No comments yet...
Related Articles
Irish Independent
4 days ago
- Irish Independent
Disability advocate Ann Kennedy defies illness to stage first art exhibit in 17 years
Ann Kennedy, who was born in 1952, has fond memories from when she last exhibited in Bray, in 2008, when she recalled eating pizza on Albert Walk, with friends and family, before returning home only to lapse into a terribly difficult period. As she said herself: 'I ceased all creative work for 10 full years'. That creative slump was brought on by her illness, which she so candidly described in her memoir, Unprotected, which details the difficulties she has faced in life due to being born with Rubella. Congenital rubella syndrome (CRS) is an illness in infants that results from maternal infection with rubella virus during pregnancy. A child can be born with severe hearing loss and/or visual impairment, congenital cataracts, heart defects, and gradually develop a myriad of other significant challenges. Ann shares this same birth experience with her twin sister, Margaret. In 2017, she told the Irish Independent: 'I woke at seven this morning, and I was crying. I'm depressed and feel utterly hopeless. That often happens. Sometimes we think of ending it all; we are so terribly weary of fighting.' Those heartbreaking words were written when she and her sister, both then 64, admitted they were struggling to get the professional help and support they needed. As children growing up in Killiney, they had to battle with a hearing impairment, and by their early 20s, had followed their own paths. Ann was doing animation in London, collaborating on projects such as the Watership Down movie and promotions for Pink Floyd, while Margaret trained as a nurse at Great Ormond Street Hospital. Ann returned to Ireland and became an accomplished artist and award-winning writer and illustrator of children's books but when the sisters were in their 40s, they encountered health issues that had a serious impact on their ability to function. Over the next few years, they were both tested and it was discovered that some of their health problems included Parkinson's disease and muscle myopathy (muscle wasting disease), which requires them to use wheelchairs most of the time. They moved to Greystones but left in 2015, after what Ann described as 'dreadful years' in an open letter, which ended with the parting words: 'I never wish to see the place again and hope I can erase the memory from my mind swiftly once I do leave.' Thankfully, her memories of Bray are more florid as she makes a welcome return with her new exhibition, 'All in the Brain'. The exhibition of works slowly began to take form years after that visit in 2008 and the ensuing slump, when 'one day,' she said, 'being brave, I visited an art shop'. ADVERTISEMENT Learn more "I began again,' she said of the new exhibition. 'It was now all in the brain and the brain would have to perform in some fashion. I knew this was going to be different and punctuated with hesitancy, despondency and confusion. Grave self-doubt enveloped me. I worked from a perspective of loss. What I produced were experimentations, learning to capture my inadequacy and hopefully some adequacy. I gave myself permission to fail in the hope I could achieve.' While she admits she 'caved many times and withered', her strength and perseverance has brought the new body of work which will be on display at the gallery until Sunday, August 3. 'I propose to show a body of creative painting that defy my personal reason. I propose to offer hope to the community of sick, older people and those with disability that if you want to paint, then paint. The work will be eclectic as personal strength is returning and styles keep changing. I begin making decisions again, abandon some and accept others. It's all in the learning,' she said. 'A decade of inaction through ill health and mystery requires opening my personal Pandora's Box. Gold may not be in fact gold, but nothing can change a person who creates through love and abandon. That is my gold, silver and bronze. Even if not in equal proportions.' The opening reception for All in the Brain is on Sunday, July 27, from 3-5pm.
Irish Times
6 days ago
- Irish Times
Hvivo revenue falls as pharma market uncertainty hits
Revenue at Irish clinical trials company Hvivo fell in the first six months of the year as the uncertainty in the pharma market, particularly in the US, hit contracts. But the company remained on track to reach £47 million in revenue for the year, it said. In a trading update for the six months ended June 30th 2025, the company said revenue for the period was £24.2 million, down from £35.6 million a year earlier. Hvivo, which offers contract research to pharmaceutical companies, said in May that a significant contract for a human challenge trial had been cancelled, while another had been postponed. A smaller study had also been cancelled, the company said, blaming the uncertainty in the pharmaceutical sector and depressed financing for the biotech market. READ MORE Its weighted contracted order book was £40 million at the end of the first half, down from £71 million in the prior year. However, the company said it had a strong sales pipeline, with opportunities that were not yet contracted and the aggregate value of customer proposals submitted in the first half of the year already surpassing the total in 2024. Its acquisitions of CRS and Cryostore, which were completed for £10.5 million net of cash acquired, are also diversifying its offerings. The acquisitions contributed £5.5 million to group revenue in the first half. The group said it is in advance discussions for a number of major human challenge trial projectsand is also pursuing significant opportunities for its hLAB and clinical services operations. 'Some persistent macro and sector-specific headwinds remain however, including a subdued biotech funding environment partly connected to uncertainties in the US, which have impacted the broader CRO industry, resulting in delayed contract conversion and a number of cancellations and postponements,' Hvivo said in a statement. It said the issues affecting the sector are likely to the 'transitory rather than fundamental', and predicted its business would return to growth as the market normalises, and biotech funding improves. 'The utility of our services remains strong, as demonstrated by the recent success of our client's Phase II candidate. Our diversification strategy is already delivering results, and we expect continued momentum across all revenue streams,' said chief executive Dr Yamin 'Mo' Khan. 'While macroeconomic and sector-specific headwinds are still affecting contract conversions, we remain confident in the long-term growth trajectory of our services and the overall prospects for HVIVO. I'm encouraged by the strength of our sales pipeline, with several major opportunities that could enhance the growth of our services. We believe that we are well positioned to deliver growth in FY26, and we look forward to keeping shareholders updated on our progress.'
The Journal
18-07-2025
- The Journal
Vaping giant Juul wins key regulatory battle in US
WELL-KNOWN VAPE brand Juul has been approved for sale in the US three years after being temporarily banned by US health authorities during the Biden administration in 2022. The US Food and Drug Administration (FDA) had originally banned the vapes as Juul did not prove its products would be 'appropriate for the protection of the public health', although this measure was suspended after two weeks to conduct a scientific review. On Thursday, Juul Labs said that the FDA had officially authorised it to market its vaping system and refill capsules. The decision means Juul can continue to sell products that have been on the US market. The well-known e-cigarette brand was available on the Irish market for only two years after being launched in 2019. Juul Labs exited several European markets at the end of 2020. Its products were authorised for sale in the US as the benefits for smoking cessation were 'sufficient to outweigh the risks of the product,' including to anyone not using tobacco products. Advertisement Juul markets its products as providing a public health benefit because they can shift smokers away from cigarettes closely linked to deadly illnesses. But the company has been criticised for its marketing practices, agreeing to pay $438.5m (€375.93m) in a 2022 settlement with 34 US states to resolve accusations of marketing to underage smokers. Juul won the new marketing granting orders (MGO) after submitting more than 110 scientific studies to the agency, it said in a statement. 'Following rigorous evaluation of the data, FDA decided that an MGO for the Juul System was 'appropriate for the protection of public health' – the standard required by statute for authorisation,' Juul said. However, a spokesperson for the US Department of Health and Human Services said its decision 'does not mean these tobacco products are safe, nor are they 'FDA approved''. The Department also indicated it will continue to monitor Juul's compliance with youth marketing restrictions.
