Novaliq GmbH

Earlier this year, Avidity started a pivotal Phase III HARBOR trial evaluating del-desiran inpatients with DM1. Image credit: JHVEPhoto /Shutterstock. The US Food and Drug Administration (FDA) has lifted the partial clinical hold on Avidity Biosciences ’ investigational drug, delpacibart etedesiran (del-desiran). In 2022, the US regulatory agency placed a partial hold after a report of a serious adverse event in a newly enrolled patient in the Phase I/II MARINA trial (NCT05027269), which evaluated del-desiran in patients myotonic dystrophy type 1 (DM1). The company did not disclose the details of the adverse event and the US FDA eased the partial clinical hold the following year, allowing the company to recruit new patients. The clinical hold has not slowed down Avidity’s efforts to advance the clinical development of del-desiran in DM1. Earlier this year, Avidity started a pivotal placebo-controlled Phase III HARBOR trial (NCT06411288). The study is expected to enrol 150 participants with DM1 who will be assessed over 54 weeks. The trial’s primary endpoint is the improvement of myotonia over 30 weeks, measured using video Hand Opening Time (vHOT), the average time to extend the right third and first digits. The study’s secondary endpoints also include the functional assessment of myotonia across 30 weeks, including hand grip strength, quantitative muscle testing composite score and Myotonic Dystrophy Type 1 activity and participation scale c. Del-desiran is an antibody oligonucleotide conjugate (AOC) made up of a monoclonal antibody that binds to the transferrin receptor 1 (TfR1) conjugated with a small interfering RNA (siRNA) targeting myotonic dystrophy protein kinase (DMPK) mRNA, responsible for producing DMPF protein. If approved, GlobalData expects del-desiran to pull in over $1.2bn in sales by 2030. See Also: BMS receives FDA approval for Opdivo to treat NSCLC Novaliq and Laboratoires Théa announce EC approval for dry eye disease therapy GlobalData is the parent company of Pharmaceutical Technology . mRNA vaccines were instrumental during the Covid-19 pandemic. Since then, there has been increased interest in mRNA therapies beyond the vaccines. GlobalData forecasts the mRNA non-vaccine therapeutics field to reach $2bn by 2028. DM1 is a genetic disorder caused by a triplet-repeat in the DMPK gene. It primarily affects skeletal and cardiac muscle causing muscle weakness and wasting leading to respiratory problems, and cardiac abnormalities. There are no approved treatments for DM1 and management is mostly aimed at symptomatic treatment. Another therapy in development for treating DM1 is AMO Pharma’s AMO-02 (tideglusib). The therapy is an irreversible inhibitor of glycogen synthase kinase 3 (GSK-3), it has the potential to disrupt the RNA repeats that cause congenital DM1. In May, AMO announced plans to start a Phase III trial for AMO-02 in DM1 patients.

Vevizye’s effectiveness in treating DED was confirmed by two placebo-controlled trials. Credit: fizkes via Shutterstock. Novaliq and Laboratoires Théa have announced approval from the European Commission (EC) for Vevizye (ciclosporin 0.1% eye drops solution) for patients with dry eye disease (DED) in Europe. The partnership between the two companies has also been closed, with Théa acquiring the rights to commercialise Vevizye in Europe and selected Middle East and North African countries. Vevizye is the only water-free ciclosporin 0.1% eye drop solution that received approval in the EU to treat moderate to severe DED in adults who have not responded to tear substitute treatments. Novaliq CEO Christian Roesky said: “Théa’s focus on innovation, its leading commercial capabilities and the strong track record in Europe makes them the perfect partner for Novaliq.” DED affects an estimated 15 million diagnosed patients in the five largest European countries and presents a significant treatment challenge. See Also: Potential for peptides to replace antibodies in radiopharma, says WuXi AppTec exec BPGbio’s mitochondrial disorder drug gets paediatric disease status The efficacy of the product for treating DED was established through two randomised, double-masked, multicentre, vehicle-controlled studies, ESSENCE-1 and ESSENCE-2. The primary endpoint in both trials was the change from baseline in total corneal fluorescein staining (tCFS) score at Day 29. Results showed a statistically significant reduction in tCFS, with up to 71.6% of patients experiencing clinical improvement within four weeks. Statistically significant effects were also observed in all other key secondary ocular surface sign endpoints. In the US, the Food and Drug Administration approved the product as VEVYE in May 2023. Vevizye received approval in all 27 EU member states. In August 2023, the European Medicines Agency accepted Novaliq’s marketing authorisation application f or CyclASol, another cyclosporine ophthalmic solution at 0.1%, as a treatment for DED.

The dark matter of the genome is the majority of the human genome, making up around 98% of it. Image credit: Shutterstock/ Tartila. Enara Bio has raised $32.5m in a Series B financing round to advance its platform shining a light on cancer-specific antigens from previously uncharted genomic dark matter. The round was led by Pfizer ’s and Merck KGaA’s respective investment arms, who joined as new backers. The Francis Crick Institute also joined the round as a new investor, alongside original backers including RA Capital, Samsara BioCapital and SV Health Investors, as per a 3 October press release. UK-based Enara said the funds will be used to develop its pipeline of T cell receptor (TCR)-based therapies that target regions of the genome considered ‘dark’ or non-coding. As far as scientists know, these areas of the genome play no role normally in protein-coding. These areas are known as genomic dark matter and although usually silenced in healthy cells, they are often expressed in tumours following alterations to cellular processes. Enara states that cancer cells can therefore present novel peptide antigens on their surface, coded by these dark areas. The biotech attributes the name ‘Dark Antigens’ to these novel therapeutic targets it discovered. Enara’s EDAPT platform is what the company uses to discover and validate Dark Antigen targets for potential cancer immunotherapies. The platform combines bioinformatics databases with mass spectrometry of tumour and healthy tissues alongside RNA in situ hybridisation. See Also: ImmunoPrecise and Biotheus partner on bispecific antibodies to treat cancer Novaliq and Laboratoires Théa announce EC approval for dry eye disease therapy Whilst Enara has not shared details about its pipeline yet, the company said its “internal focus is developing TCR-directed immunotherapies against Dark Antigen targets that are highly prevalent and homogenously expressed across solid tumours.” Pfizer ventures partner Rana Al-Hallaq, who joins Enara’s board of directors following the financing, said: “Dark Antigens may unlock the potential of immunotherapy for many additional patients with solid tumours, and we are pleased to support Enara’s development of this cutting-edge platform to discover and validate these previously untapped targets.” Before Pfizer and Merck KGaA came on board, Enara had already attracted the interest of another big pharma. Boehringer Ingelheim licensed multiple Dark Antigens from the biotech to develop off-the-shelf immunotherapies for non-small cell lung cancer (NSCLC) in January this year as part of an ongoing collaboration originally started in 2021 . Boehringer did not disclose the upfront payment three years ago, but milestone and royalty payments could mean Enara is in line to receive $1.1bn from the partnership with the German drugmaker. Genomic dark matter has become a promising area for cancer detection, monitoring, and treatment. Earlier this year, researchers at Johns Hopkins University in the US used artificial intelligence (AI) to analyse these non-coding areas of genetic code found in tumours and the bloodstream. Enara is not the only biotech in the dark genome space – Rome Therapeutics has developed a candidate that targets the ‘jumping gene’ LINE-1 for the treatment of autoimmune conditions. The company raised $72m in a Series B round in September 2023, with backing from Johnson & Johnson (J&J) Innovation and Bristol Myers Squibb (BMS).