Hearing Loss, Sensorineural

BARCELONA, Spain I April 1, 2025 I ESTEVE has signed a license and supply agreement with Eton Pharmaceuticals with global rights for Increlex®, except for USA. This biologic product, the first biologic in ESTEVE’s portfolio, is used for the long-term treatment of growth failure in patients aged 2 to 18 years due to a condition known as ‘severe primary insulin-like-growth-factor-1 deficiency’ (SPIGFD). Patients with this condition have low levels of the hormone insulin-like growth factor-1 or IGF-1, which is required for normal growth. 1 “As a rare disorder, patients suffering SPIGFD, often face difficulties associated with receiving a  late correct diagnosis, with consequent delayed treatment initiation with limited access to appropriate therapy. This has a considerable impact on the physical health and quality of life of these patients. 2 The disease awareness and the multidisciplinary  approach are critical to improve those people’s lives. This standard of care medicine is perfectly aligned with our strategy,focused on highly specialized treatments for high unmet medical and patient needs”, confirms José María Giménez Arnau, Chief Scientific & Medical Officer of ESTEVE. Increlex® was designated an ‘orphan medicine’ (a medicine used in rare diseases) in 2006. 1 It is approved by the U.S. Food and Drug Administration (FDA) 3 and European Medicines Agency (EMA) 1 and authorized in 40 territories. References About ESTEVE ESTEVE ( www.esteve.com ) is a global pharmaceutical company with a clear purpose: to improve people’s lives. Founded in 1929 and headquartered in Barcelona, ESTEVE has a strong international presence with pharmaceutical affiliates in Spain, Portugal, Italy, Germany, France, the UK, and the USA. ESTEVE is focused on delivering highly specialized treatments that address significant unmet medical needs in several therapeutic areas. In addition, to our innovative pharma business, we offer comprehensive Contract Manufacturing services (CMO), specializing in the production of Active Pharmaceutical Ingredients (APIs) through world-class facilities in Spain, Mexico, and China. ESTEVE’s strong commitment to its core values—people matter, transparency, and accountability—remains at the heart of everything it does. SOURCE: Esteve

There’s still money to be raised by gene therapy startups with good clinical data. On Wednesday, North Carolina biotech Atsena Therapeutics said it had raised a $150 million Series C to try and be the first to treat a rare form of ocular disease that leads to blindness. Bain Capital Life Sciences led the financing. Another new Atsena investor, Wellington Management, also took part as did all existing backers, including Lightstone Ventures, Sofinnova Investments and Abingworth, among others. “There is a place here for programs and companies that do have compelling clinical data in diseases with high unmet clinical needs,” Atsena CEO Patrick Ritschel said in an interview. The gene therapy field has been held back by struggles with commercialization, pipeline cuts and funding pullbacks. Only a few ocular gene therapy companies have been able to secure nine-figure financings since 2022, including Beacon , Ray and Frontera . Atsena last disclosed a $24.5 million Series B in October 2023. Atsena is working on Part B of a Phase 1/2 study of ATSN-201, a gene therapy that it hopes will treat a genetic ocular disease called X-linked retinoschisis, or XLRS. The condition is usually found in childhood and impacts about 30,000 men in the US and European Union, the biotech said. “In a majority of the nine patients treated, we see anatomic resolution of disease and we see functional improvement in the majority. So it’s not often you can get that kind of convincing signal on a small dataset ,” Leonard Feiner, a principal at Bain Capital Life Sciences, said in an interview. The disease causes an abnormal split in the retinal layers and can’t be fixed by glasses, eventually leading to blindness. “The interesting thing about this disease, XLRS, is that in addition to having functionally decreased vision, these patients also have an anatomic problem, which is a lack of adhesion between the layers of the retina,” Feiner said. The therapy is a subretinal injection and uses an adeno-associated virus. The new funding is slated to carry Atsena all the way through a biologics license application at the FDA “and beyond,” Ritschel said. The startup will meet with the FDA later this year to clarify if the Phase 1/2 could support an accelerated approval, or if the biotech would need to run an additional study. The future of cell and gene therapy decisions at the FDA is now in limbo as top leadership, most notably Peter Marks, has been forced out or resigned in recent days . “We’ve seen others in this space in [the past] 12 months or so obtain accelerated approval alignment with the FDA in rare disease, genetic therapy environments, so we would be hopeful for that as well,” Ritschel said. It’s not the first company to try and treat the disease. Biogen had a $1 billion-plus pact with Applied Genetic Technologies Corporation, but their gene therapy failed a Phase 1/2 study in 2018 . Atsena is also working on ATSN-101 for Leber congenital amaurosis type 1, or LCA1. The biotech began searching for a partner for the former Sanofi gene therapy in 2023 and shortly after presented one-year data from a Phase 1/2 trial. It secured a licensing agreement with Kyoto-based Nippon Shinyaku last fall. Ritschel said the pivotal study has yet to begin and declined to disclose any expected timing. The Series C will also support a preclinical pipeline of gene therapies for inherited retinal diseases like MYO7A-associated Usher syndrome, or USH1B, the company said. Atsena employs “just over 20 people today,” Ritschel said. Prior to joining the company in 2020, he was involved in other gene therapy upstarts as co-founder of StrideBio and an angel investor in Bamboo Therapeutics. Ginkgo bought AAV capsid assets from StrideBio in 2023 and Pfizer acquired Bamboo for up to $645 million in 2016.

It is a biologic product used to treat children and adolescents from 2 to 18 years-old who suffer from severe primary insulin-like growth factor 1 deficiency1 The acquisition of the treatment for this rare disease is an example of ESTEVE's commitment focused on growth opportunities in high unmet medical and patient needs. BARCELONA, Spain, April 1, 2025 /PRNewswire/ -- ESTEVE has signed a license and supply agreement with Eton Pharmaceuticals with global rights for Increlex®, except for USA. This biologic product, the first biologic in ESTEVE's portfolio, is used for the long-term treatment of growth failure in patients aged 2 to 18 years due to a condition known as 'severe primary insulin-like-growth-factor-1 deficiency' (SPIGFD). Patients with this condition have low levels of the hormone insulin-like growth factor-1 or IGF-1, which is required for normal growth.1 "As a rare disorder, patients suffering SPIGFD, often face difficulties associated with receiving a late correct diagnosis, with consequent delayed treatment initiation with limited access to appropriate therapy. This has a considerable impact on the physical health and quality of life of these patients.2 The disease awareness and the multidisciplinary approach are critical to improve those people's lives. This standard of care medicine is perfectly aligned with our strategy,focused on highly specialized treatments for high unmet medical and patient needs", confirms José María Giménez Arnau, Chief Scientific & Medical Officer of ESTEVE. Increlex® was designated an 'orphan medicine' (a medicine used in rare diseases) in 2006.1 It is approved by the U.S. Food and Drug Administration (FDA)3 and European Medicines Agency (EMA)1 and authorized in 40 territories. References Increlex | European Medicines Agency (EMA) Challenges in the care of individuals with severe primary insulin-like growth factor-I deficiency (SPIGFD): an international, multi-stakeholder perspective - PubMed Drug Approval Package: Increlex (Mecasermin [rDNA origin]) NDA #021839 About ESTEVE ESTEVE () is a global pharmaceutical company with a clear purpose: to improve people's lives. Founded in 1929 and headquartered in Barcelona, ESTEVE has a strong international presence with pharmaceutical affiliates in Spain, Portugal, Italy, Germany, France, the UK, and the USA.   ESTEVE is focused on delivering highly specialized treatments that address significant unmet medical needs in several therapeutic areas. In addition, to our innovative pharma business, we offer comprehensive Contract Manufacturing services (CMO), specializing in the production of Active Pharmaceutical Ingredients (APIs) through world-class facilities in Spain, Mexico, and China.  ESTEVE's strong commitment to its core values—people matter, transparency, and accountability—remains at the heart of everything it does.  For more information: Irene Simón, Head of Global External Communications & ESG, ESTEVE Tel.+34 934 466 000 – [email protected] Gemma Mestre, External & Digital Communications Manager, ESTEVE Tel.+34 934 466 000 – [email protected] Logo - SOURCE ESTEVE WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED