Latest news with #bullosa
Yahoo
13-05-2025
- Business
- Yahoo
Abeona secures cash runway with $155m priority review voucher sale
US-based Abeona Therapeutics has signed a deal to sell its priority review voucher for $155m, two weeks after picking up approval in the US for its first commercial product. The biotech company was eligible for the voucher upon securing a rare paediatric disease designation. A voucher was subsequently given to the biotech after winning approval from the US Food and Drug Administration (FDA) for gene therapy Zevaskyn (prademagene zamikeracel) late last month. Zevaskyn is a treatment for the rare disease recessive dystrophic epidermolysis bullosa (RDEB). Abeona did not disclose the voucher's buyer, saying only it had entered a definitive asset purchase agreement. Investors seemed happy with Abeona's decision, with the company's stock, listed on the Nasdaq exchange, peaking 16.8% higher in trading yesterday, from $5.27 a share at close on Friday (9 May) to a high of $6.16. The company's stock later settled at $5.60 a share at market close – a 6.26% increase on the previous trading day. This spike coincides with stock increases across the pharmaceutical sector on Monday after US President Donald Trump announced he would sign an executive order to bring down US drug prices. Abeona has a market cap of $273.27m. Priority review vouchers are awarded to pharmaceutical companies developing drugs for rare paediatric diseases, neglected tropical diseases, or material threat medical countermeasures – markets that companies are hesitant to enter due to weak financial performance. Biotechs can use the vouchers to slash the FDA review time of any drug application from the usual ten months to six months. This can be done in a more lucrative market, facilitating a recuperation of funds spent developing the original product. Alternatively, companies can sell their voucher for a quick cash injection – the route opted by Abeona. The company did not reveal how it plans to use the funds, with CEO Joe Vazzano commenting only that the $155m means the company has 'sufficient cash for more than two years of operating expenses without the need for capital infusion.' Vazzano added that cash projections neither account for sales of Zevaskyn, which has a planned commercial rollout in Q3 2025. It is likely, therefore, that at least part of the funds raised from the voucher sale will go towards the gene therapy's launch. Zevaskyn will be priced at $3.1m a dose and is a one-time treatment. Analysis by GlobalData's Pharma Intelligence Centre forecasts sales of $366m by 2031. GlobalData is the parent company of Pharmaceutical Technology. The figure achieved by Abeona in its voucher sale tallies with other recent deals. Zevra sold a voucher in February for $150m, also to an undisclosed party. In November 2024, PTC Therapeutics sold a voucher to an unnamed buyer for $150m. The highest fee for a voucher still stands at $350m, when AbbVie bought one from United Therapeutics in 2015. Though down from these heights, recent transactions are still a step up from a relatively stable $100m selling price average seen over the past seven years, perhaps a reflection that vouchers now hold more value in an uncertain US funding landscape. Rare disease development has hit rocky waters amid mass layoffs across US health agencies by the Trump administration. A big setback could arise from the failure to renew the priority review voucher programme for paediatric rare diseases, which expired, in part, due to shifting political priorities. This has cast a blanket of precariousness over future rare disease pipelines. "Abeona secures cash runway with $155m priority review voucher sale" was originally created and published by Pharmaceutical Technology, a GlobalData owned brand. The information on this site has been included in good faith for general informational purposes only. It is not intended to amount to advice on which you should rely, and we give no representation, warranty or guarantee, whether express or implied as to its accuracy or completeness. You must obtain professional or specialist advice before taking, or refraining from, any action on the basis of the content on our site. Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Yahoo
30-04-2025
- Health
- Yahoo
Graeme Souness looking to raise $1M as Liverpool legend embarks on toughest challenge yet
Former Liverpool captain and manager Graeme Souness is embarking on his toughest challenge to date by swimming the English Channel and back to raise money for a cause close to his heart. After walking from QPR's Loftus Road to Wembley Stadium for the British Heart Foundation, the ex-Sky Sports pundit will look to conquer another fundraising feat before the end of April. The Reds legend - who won five league titles and three European Cups in his glittering spell at Anfield - was characterized by his full-blooded displays on the field during his storied playing days. Now, he is turning his passions towards raising awareness and funds to help people suffering with the condition of epidermolysis bullosa (EB). The 71-year-old is a vice-president of DEBRA UK, a charity which was established to support those living with EB - an illness which leaves sufferers with painful blisters, open wounds, and excruciating itching. READ MORE: Alan Hansen health latest as Liverpool legend spotted at Anfield amid title celebrations READ MORE: Liverpool legend tells Arne Slot how rest of Premier League will respond to title win Back in 2023, Kop hero Souness swam the Channel for the charity and raised £1.5m (£2m) for the cause. This time around, he will be swimming double the distance he did last time, as he looks to raise £750,000 ($1m) to fund two EB clinical trials - which could lead to treatments to alleviate the pain those suffering from the condition live with on a daily basis. In his role as an ambassador for DEBRA, Souness is set to don his wetsuit and hit the water on April 30 at Dover. In a statement issued by the charity, the ex-Liverpool skipper expressed his commitment to helping individuals living with EB, such as his friend Isla Grist. He said: "I had to do something for my wee friend, Isla, and the thousands of other children and adults affected by this cruel condition. "With the money raised DEBRA UK was able to start testing drugs which could be life-changing but there are many more drugs that they need to test if we are to secure effective treatments for every form of EB, treatments that could help stop the extreme pain. "This is why we're getting back in the water once again. It's going to be tough; I'm 71 now and it's double the distance, but I know we can do this, we must do this, we must be the difference for EB." In an exclusive interview with last week, Souness spoke passionately about his dedication to the charity, his efforts to raise awareness of the condition and his hope that he can improve the lives of those living with EB. He said: "DEBRA is a charity that helps people with a genetic condition called EB, you're born with it and it's a genetic condition. "The best day of your life is the day you are born because it just gets worse as you get older. It's life-limiting and these poor kids have no respite from the pain and itch that they have every waking moment of their lives. Speaking about swimming the English Channel - and back - for DEBRA this month, he spoke excitedly about what will be his second crossing of the strait which separates Dover from Calais in France. He said: "So, two years ago we swam the channel as a relay team - which means an hour in the water and five out, then another in. "We did that and we're attempting to do it again. We're going to attempt to swim there and swim back. I've prepared for it and trained for it and I wish it was tomorrow!" READ MORE: Graeme Souness outlines what Arne Slot needs for Liverpool to build on Premier League success READ MORE: Graeme Souness opens up on Anfield memories, Everton wondergoal and Bob Paisley's thoughts on titles Opening up on his desire to help those affected by the condition through his courageous efforts, he added: "It's such a horrible thing to witness. These poor kids, there's not a lot we can do for them but the whole idea is on two fronts. "Firstly to make people aware that this condition exists and secondly raise enough money to repurpose drugs that are already on the market but can be used by scientists and researchers to make them work for our kids that suffer from this."
