Tapinarof is a potential first-in-class, once-daily therapeutic aryl hydrocarbon receptor modulating agent (TAMA) topical cream being developed for the treatment of plaque psoriasis and atopic dermatitis. In August 2020, Dermavant announced positive results from PSOARING 1 and PSOARING 2, two identical, multi-center, randomized, vehicle-controlled, double-blind, parallel studies to evaluate the efficacy and safety of tapinarof cream, 1% in adult patients with plaque psoriasis.

In both PSOARING 1 (N=510) and PSOARING 2 (N=515), tapinarof cream demonstrated highly statistically significant improvement in Physician Global Assessment (PGA) score of clear (0) or almost clear (1) with a minimum 2-grade improvement compared to vehicle from baseline at Week 12 (both P<0.0001), thus meeting the primary endpoint of each trial. In addition, all secondary endpoints were achieved in both trials, including PASI75. To date, over 2,200 subjects have enrolled in 17 clinical trials of tapinarof.

ARU-1801 is an investigational gene therapy for sickle cell disease and β-thalassemia. ARU-1801 utilizes proprietary technology intended to increase functioning red blood cells by inserting a modified fetal hemoglobin gene into autologous stem cells through a lentiviral vector. Studies have indicated that sickle cell patients with elevated levels of fetal hemoglobin have fewer vaso-occlusive crises and hospitalizations.

Cerdulatinib is a topical dual janus kinase (JAK) and spleen tyrosine kinase (Syk) inhibitor. Dermavant is developing cerdulatinib as a topical therapy for a variety of dermatologic conditions. Dermavant believes that the profile of cerdulatinib is ideal for development in skin diseases where a growing body of evidence suggests that both JAK and Syk are important drivers of disease manifestation.

Tapinarof is a potential first-in-class, once-daily therapeutic aryl hydrocarbon receptor modulating agent (TAMA) topical cream being developed for the treatment of plaque psoriasis and atopic dermatitis. In August 2020, Dermavant announced positive results from PSOARING 1 and PSOARING 2, two identical, multi-center, randomized, vehicle-controlled, double-blind, parallel studies to evaluate the efficacy and safety of tapinarof cream, 1% in adult patients with plaque psoriasis.

In both PSOARING 1 (N=510) and PSOARING 2 (N=515), tapinarof cream demonstrated highly statistically significant improvement in Physician Global Assessment (PGA) score of clear (0) or almost clear (1) with a minimum 2-grade improvement compared to vehicle from baseline at Week 12 (both P<0.0001), thus meeting the primary endpoint of each trial. In addition, all secondary endpoints were achieved in both trials, including PASI75. To date, over 2,200 subjects have enrolled in 17 clinical trials of tapinarof.

Warm autoimmune hemolytic anemia (WAIHA) is a rare hematologic disease in which autoantibodies mediate hemolysis, or the destruction of red blood cells. WAIHA approximately affects 42,000 people in the United States and 66,000 people in Europe. The clinical presentation is variable and most commonly includes non-specific symptoms of anemia such as fatigue, weakness, skin paleness, and shortness of breath. Symptoms typically develop chronically over several weeks to months, but rapid progression over a span of days has also been observed.

Immunovant is developing IMVT-1401, a novel, fully human monoclonal antibody that selectively binds to and inhibits the neonatal fragment crystallizable receptor (FcRn), for the treatment of immunoglobulin G (IgG) mediated diseases, such as WAIHA. IMVT-1401 is being developed as a subcutaneous injection, providing a convenient and minimally invasive treatment option for patients.

Myasthenia gravis (MG) is a chronic autoimmune neuromuscular disease characterized by weakness and fatigue of voluntary muscles. Symptoms typically emerge in the eyes (e.g., drooping eyes, double-vision, blurred vision) and progress into the face, throat, and limbs. MG is a rare disease that affects approximately 65,000 people in the United States and 104,000 people in Europe, with a greater prevalence among younger women and older men.

The chronic nature of MG requires patients to cope with fluctuating symptoms and multiple treatment regimens for management. Persistent muscle weakness associated with MG often negatively interferes with patients’ ability to engage in daily and physical activities, including demands of work, family, and social functions.

Immunovant is developing IMVT-1401, a novel, fully human monoclonal antibody that selectively binds to and inhibits the neonatal fragment crystallizable receptor (FcRn), for the treatment of immunoglobulin (IgG) mediated diseases, such as MG. IMVT-1401 is being developed as a subcutaneous injection, providing a convenient and minimally invasive treatment option for patients.

Thyroid eye disease (TED), also known as Graves’ ophthalmopathy, is an autoimmune disorder that affects the muscles and other tissues around the eyes. TED approximately affects 33,000 people in the United States and 52,000 people in Europe. Moderate-to-severe TED is characterized by swelling and redness of the eyelids, proptosis (protrusion of the eyeball), double vision, and, in severe cases, corneal ulceration and decreased visual acuity.

Immunovant is developing IMVT-1401, a novel, fully human monoclonal antibody that selectively binds to and inhibits the neonatal fragment crystallizable receptor (FcRn), for the treatment of immunoglobulin (IgG) mediated diseases, such as TED. IMVT-1401 is being developed as a subcutaneous injection, providing a convenient and minimally invasive treatment option for patients.

AXO-Lenti-PD is an investigational gene therapy for Parkinson’s disease that delivers three genes via a single lentiviral vector to encode a set of critical enzymes required for dopamine synthesis, with the goal of reducing variability and restoring steady levels of dopamine in the brain. The gene therapy aims to provide patient benefit for years following a single administration.

AXO-AAV-GM1 delivers a functional copy of the GLB1 gene via an adeno-associated viral (AAV) vector, AAV9, which is effective in crossing the blood-brain barrier and transducing neurons, with the goal of restoring β-gal enzyme activity for the treatment of GM1 gangliosidosis. The gene therapy is delivered intravenously, which has the potential to broadly transduce the central nervous system and treat peripheral manifestations of the disease.

In preclinical studies, AXO-AAV-GM1 was shown to improve β-gal enzyme activity, reduce GM1 ganglioside accumulation, improve neuromuscular function, and extend survival. Magnetic resonance imaging (MRI) of felines with GM1 gangliosidosis treated with AXO-AAV-GM1 showed normal brain architecture through at least two years of age.

AXO-AAV-GM2 delivers functional copies of the HEXA and HEXB genes via two, co-administered AAVrh8 vectors delivered directly to the central nervous system with the goal of restoring Hex A enzyme activity to address both Tay-Sachs and Sandhoff diseases. The preclinical data for AXO-AAV-GM2 in murine models showed dose-dependent increases in Hex A enzyme activity, reductions of GM2 gangliosides in the brain and prolonged survival rates.

Initial data with AXO-AAV-GM2 suggest stabilization of disease course, attainment of normal developmental milestones, and improvement in myelination on brain MRI.

DMVT-504 is a combination of the muscarinic antagonist oxybutynin with the muscarinic agonist pilocarpine. DMVT-504 uses proprietary technology to control the release and dosing of pilocarpine with the goal of reducing the frequency and severity of dry mouth and potentially other side effects associated with oxybutynin.

Cerdulatinib is a topical dual janus kinase (JAK) and spleen tyrosine kinase (Syk) inhibitor. Dermavant is developing cerdulatinib as a topical therapy for a variety of dermatologic conditions. Dermavant believes that the profile of cerdulatinib is ideal for development in skin diseases where a growing body of evidence suggests that both JAK and Syk are important drivers of disease manifestation.

CVT-TCR-01 is a T cell receptor therapy being developed for the treatment of solid tumors. T cell receptor therapies utilize a patient’s own immune system to target tumor cells. In preparation for the therapy, a patient’s T cells are modified to express tumor-specific receptors that are then able to activate and mount an antitumor response. Through ex-vivo modification of a patient’s T cells, T cell receptor therapies can overcome natural T cell tolerance toward cancer cells and tumor-induced immunosuppression. Unlike CAR-T therapy, TCR-T therapy is designed to recognize intracellular antigens in addition to extracellular antigens, which may increase the number of tumors that can be successfully targeted.