From significant fundraisings and strategic partnerships to regulatory achievements and clinical advancements, 2024 marked an exciting period of forward momentum for clients of the MEDiSTRAVA Strategic Communications team. Here’s a recap of some of the key milestones achieved across our client portfolio, highlighting progress aimed at improving outcomes for patients worldwide.
Fundraisings
- Amber Therapeutics successfully raised $100M in Series A financing from leading healthcare and technology investors in the US and UK. The round was one of the largest Series A financings in Europe ever for a medical technology company and will support development of its neuromodulation therapy to treat mixed urinary incontinence.
- Enara Bio raised $32.5M in Series B financing to advance its first-in-class pipeline of TCR-based immunotherapies targeting novel Dark Antigens®.
- Theolytics raised £19M to advance its lead oncolytic immunotherapy THEO-260 into clinical trials in ovarian cancer. Theolytics was also awarded a £2M Transforming Cancer Therapeutics grant from Innovate UK, to support the Phase 1 clinical trial for THEO-260.
- Angelini Ventures joined Nouscom’s Series C financing round, boosting the total amount raised to €75.8M, and providing funds to support Nouscom as it advances its pipeline of off-the-shelf and personalised cancer immunotherapies.
- IMU Biosciences secured £11.5M of Series A funding to be deployed to advance its novel CytAtlas™ platform, foster new strategic partnerships and projects, conduct immune research, and expand its operations.
- Owlstone Medical secured $6.5M to support the development of breath-based diagnostics for infectious disease through its Breath Biopsy platform.
Deals
- Nxera Pharma and Boehringer Ingelheim entered an up to EUR 670M global collaboration and exclusive option-to-license agreement to develop Nxera’s portfolio of GPR52 agonists, designed to simultaneously address positive, negative, and cognitive symptoms of schizophrenia.
- Abingworth entered two strategic, clinical-co-development financing agreements: with Gilead Sciences, providing up to $210M to support select studies with Trodelvy® in non-small lung cancer; and with Teva to provide up to $150 million, to accelerate development of its dual-action asthma rescue inhaler (ICS-SABA/TEV-‘248) program.
- Boehringer Ingelheim licensed a number of cancer antigens discovered and validated through Enara Bio’s Dark Antigen discovery platform, to develop off-the-shelf immunotherapies for non-small cell lung cancer.
- Quell Therapeutics entered a multi-year strategic partnership with eXmoor pharma for the manufacture of multiple autologous engineered CAR-Treg cell therapies in Quell’s pipeline targeting autoimmune diseases.
Regulatory milestones
- Nxera’s oral dual orexin receptor antagonist QUVIVIQ (daridorexant) was approved and launched in Japan as a new treatment for adults with insomnia, a highly prevalent sleep disorder that is recognised as an important national health issue in Japan.
- AlveoGene was granted Rare Pediatric Disease Designation (RPDD) and Orphan Drug Designation (ODD) from the US FDA for its inhaled gene therapy for lethal neonatal surfactant protein B (SP-B) deficiency, a fatal genetic lung disease.
- Actimed Therapeutics was granted ODD for ACM-002 for the treatment of amyotrophic lateral sclerosis.
- Cytovation’s lead asset CY-101 was granted ODD for the treatment of adrenocortical carcinoma.
- EyeDNA Therapeutics received RPDD from FDA for its investigational gene therapy for patients with inherited retinal dystrophy caused by mutations of the PDE6b gene.
Clinical milestones
- Nxera achieved various clinical milestones with programs in its own pipeline and its extensive partnered pipeline
- Start of a Phase 3 clinical trial in South Korea evaluating daridorexant for the treatment of adult patients with insomnia.
- Partner Neurocrine reported positive results from a Phase 2 trial of NBI-1117568, an investigational muscarinic M4 selective agonist discovered by Nxera, in schizophrenia.
- Partner Centessa initiated a Phase 2 trial, following positive Phase 1 results, with ORX750, a selective orexin receptor 2 agonist designed by Nxera and in development to treat various sleep disorders.
- Nxera dosed the first subjects in a Phase 1 trial with NXE0033744, an investigational EP4 receptor agonist for the treatment of inflammatory bowel disease.
- Avillion and AstraZeneca announced positive results from a Phase 3 trial investigating AirSupra®, an inhaled, fixed-dose SABA/ICS combination rescue treatment in the US for patients with intermittent or mild persistent asthma.
- Nouscom completed patient enrollment into a randomized Phase 2 study evaluating NOUS-209, an off-the-shelf neoantigen Immunotherapy, in dMMR/MSI metastatic colorectal cancer. Nouscom also presented data from an ongoing Phase 1b/2 trial that continues to highlight the potential of NOUS-209 to ‘intercept’ cancer in Lynch Syndrome carriers, who face an elevated risk of developing life-threatening cancers, such as colorectal, gastric and endometrial.
- Amber Therapeutics presented positive preliminary results from its first-in-human study with Amber-UI, a fully implantable neuromodulation therapy for urinary incontinence.
- Quell Therapeutics advanced its lead CAR-Treg cell therapy programme QEL-001 for liver transplant patients into the efficacy cohort of the LIBERATE Phase 1/2 trial having successfully completed a safety cohort.
- Quell Therapeutics achieved its first significant research milestone in its Type 1 Diabetes CAR-Treg cell therapy programme with AstraZeneca, resulting in AZ exercising its exclusive option to license the potential therapy for further development and commercialisation.
- Oxford Biotherapeutics dosed its first patient with lead candidate, OBT076, in a Phase 1b trial in adenoid cystic carcinomas of the head and neck, sponsored by GORTEC.
- Theolytics initiated its Phase 1/2a first-in-human trial for THEO-260, involving patients with advanced-stage platinum-resistant ovarian cancer.
- Transgene and partner NEC presented extensive Phase 1 data, confirming strong immunogenicity of TG4050, an individualized neoantigen cancer vaccine, for the adjuvant treatment of head and neck cancers.
Other highlights
- Nxera Pharma successfully rebranded from Sosei Heptares. The new name expresses the Company’s vision to lead the next era of medicine and its mission to accelerate the development of life-changing medicines for patients in need. Nxera also won Biotech Company of the Year and Financing Deal of the Year at the Citeline Japan Awards 2024.
- An extensive communications programme for the Oxford-Harrington Rare Disease Centre (OHC) was conducted in 2024, designed to raise awareness of OHC’s mission to deliver 40 new therapies for rare diseases in the next decade, including
- Promoting International Rare Disease Day by the illumination of Magdalen College chapel at the University of Oxford.
- Instigating a social media campaign to promote the first OHC Rare Disease Symposium at Trinity College, Oxford, that brought together leaders from academia, biotech, pharma, venture capital, and patient groups to explore cutting-edge research and innovative treatments for rare diseases.
- Coordinating media activities around the appointment of former UK Prime Minister David Cameron as Chair of the OHC Advisory Council
- Announced the inaugural class of Oxford-Harrington Rare Disease Scholars to support groundbreaking research on treatments for rare and ultra-rare diseases across various medical fields.