Glaucoma is a progressive optic neuropathy with medical therapies historically focusing on topical administration of drops. Thus, large barriers to effective treatment exist, including poor medication adherence and intolerance to local adverse effects. Emerging therapies are targeted toward circumventing some of these challenges and are now focused on newer topical formulations, sustained-release implants, and more recently gene therapy.

Immune thrombocytopenia (ITP) is an autoimmune disorder leading to low platelet counts and increased bleeding risk. The management of ITP, particularly in chronic or refractory cases, presents ongoing challenges, necessitating the development of novel therapeutic approaches that target its complex immunopathophysiology.

The treatment armamentarium for multiple myeloma (MM) has evolved substantially over the past 20 years. Standard-of-care regimens for newly diagnosed MM and early relapsed/refractory disease (RRMM) include quadruplets and triplets comprising CD38 monoclonal antibodies, proteasome inhibitors, and/or immunomodulatory drugs, plus dexamethasone. Targeted agents and immune-based therapies are being used increasingly early in the treatment algorithm. There is an ongoing need for novel treatment options to improve outcomes with existing therapies and in subsequent lines of treatment.

Bullous pemphigoid (BP) is the most prevalent autoimmune blistering disorder. It is associated with high mortality partially due to side-effects of immunosuppressive drugs. Development of new therapeutics for BP has turned out to be exceedingly challenging, among others, due to frailty of the patients and heterogeneity of the disease.

Myelin oligodendrocyte glycoprotein antibody-associated disease (MOGAD) is an emerging autoimmune demyelinating disorder distinct from multiple sclerosis and AQP4-IgG-positive neuromyelitis optica. Despite increasing recognition, no therapies are currently approved for MOGAD, and treatment remains empirical, with significant variability in clinical response and access to care.

Although long known for their psychoactive effects, psychedelic drugs have only recently been investigated for medicinal use. Psilocybin has attracted the greatest interest with studies suggesting that it may be a useful agent in psychiatry and in palliative care.

Over the last decade, immune checkpoint inhibitors (ICIs) like PD-1/PD-L1 or CTLA-4, which reinvigorate T cells for tumor control have become standard-of-care treatment options. In response to the increasingly recognized mechanisms of resistance to T cell activation in immunologically cold tumors, immuno-oncology drug development has started to shift beyond T cell approaches. These include tumor-associated macrophages (TAMs), a major pro-tumor immune cell population in the tumor microenvironment known to silence immune responses.

In the era of precision medicine, a molecularly targeted therapeutical approach is still missing in the field of neuroendocrine neoplasms (NENs) other than for radioligand therapy (RLT). So far sunitinib is the only tyrosine kinase inhibitor (TKI) available in clinical practice for NENs in Western countries, limited to advanced, progressing pancreatic neuroendocrine tumors (NETs). A few TKIs worldwide reached an advanced stage of clinical investigation with promising results.

The symptomatic treatment of Parkinson Disease (PD) relies on levodopa. With disease progression, the response to levodopa becomes variable, leading to fluctuations in benefit on PD symptoms. These motor fluctuations are challenging to manage and negatively impact quality of life in PD.

Severe asthma is a chronic airway disease characterized by many pathomechanisms known as endotypes. Biological therapies targeting severe asthma endotypes have significantly improved the treatment of this disease, thus remarkably bettering patient quality of life.

Sarcomas are a rare and diverse group of mesenchymal-origin solid tumors, constituting only 1% of adult malignancies and classified into soft tissue and bone sarcomas. For localized diseases, surgery and radiotherapy remain the cornerstone treatments. However, systemic options for advanced stages are limited, with an overall survival of approximately 20 months. There is an urgent need to refine clinical trial designs and evaluate novel therapies to improve patient outcomes.

Preclinical and clinical pharmacologic evidence indicates that orexin systems are relevant to sleep-wake cycle regulation and dimensions of reward and cognition, providing the basis for hypothesizing that they may be effective as therapeutics in mental disorders. Due to the limited efficacy and tolerability profiles of existing treatments for Major Depressive Disorder (MDD), investigational compounds in novel treatment classes are needed; seltorexant, an orexin receptor antagonist, is a potential new treatment currently under investigation.