Redwood City

Researchers are evaluating autologous Chimeric Antigen Receptor T cells CART cells in people with active pemphigus vulgaris PV, a condition causing painful blisters on skin and mucous membranes. This phase 12 open-label study includes two parts the main study focused on DSG3-CAART therapy for mucosal PV patients not well controlled by standard treatments, and a sub-study assessing CABA-201 therapy in mucosal-dominant and mucocutaneous PV patients. The study aims to find safe dosing and to understand if these therapies can control disease activity. The main study now closed to enrollment tested different doses and infusion schedules of DSG3-CAART, sometimes combined with other treatments. The open sub-study involves a single infusion of CABA-201 with varying preconditioning regimens using cyclophosphamide and fludarabine. Treatments are given intravenously, and the sub-study is currently enrolling participants. Participants will be monitored for adverse events up to 3 months for DSG3-CAART and up to 156 weeks for CABA-201. Researchers will measure safety, immune cell levels, antibody changes, disease activity scores, and remission status over up to 36 months. The study includes regular clinical assessments, laboratory tests, and ongoing safety follow-up to evaluate treatment effects and tolerability.

Researchers are evaluating the efficacy and safety of HBS-301 in adults aged 18 years and older who have idiopathic hypersomnia IH, a condition marked by excessive daytime sleepiness EDS. This Phase 3, multicenter, randomized, double-blind, placebo-controlled study aims to better understand how HBS-301 affects symptoms of IH including sleep inertia, fatigue, and cognitive complaints. Participants will be assigned to receive either HBS-301 tablets or matching placebo tablets once daily in the morning upon waking during an 8-week double-blind treatment period. Following this, there is an optional one-year open-label extension where all participants may receive HBS-301. The study begins with a screeningbaseline period lasting up to 28 days and concludes with 30 days of safety follow-up after treatment. During the trial, participants will undergo various assessments including the Epworth Sleepiness Scale to measure daytime sleepiness, the Idiopathic Hypersomnia Severity Scale, Sleep Inertia Questionnaire, and other patient-reported outcome measures. Researchers will monitor changes in fatigue, cognitive function, quality of life, work productivity, and side effects throughout the study and extension period. Total participation may last up to about 16 months including safety follow-up.

Researchers are conducting a Phase 3, multicenter, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of HBS-301 in adults aged 18 years and older with narcolepsy. The study focuses on treating excessive daytime sleepiness EDS, cataplexy, sleepinesswakefulness, and fatigue in participants diagnosed with narcolepsy. Approximately 258 participants will be included in this trial. Participants will be randomly assigned to receive either HBS-301 tablets or matching placebo tablets once daily in the morning upon waking. The study includes a ScreeningBaseline period lasting up to 28 days, followed by an 8-week Double-blind Treatment period. After this, participants may choose to enter a 1-year Open-label Extension period where they will receive HBS-301. Finally, a 30-day safety follow-up will monitor participants after treatment ends. Throughout the study, participants will undergo various assessments including evaluations of daytime sleepiness using the Epworth Sleepiness Scale, cataplexy frequency, wakefulness through the Maintenance of Wakefulness Test, fatigue levels, cognitive complaints, quality of life, and work productivity. Safety will be monitored by tracking adverse events and pharmacokinetic measurements. The total study participation may last up to approximately 16 months including all phases.

Researchers are evaluating the safety and preliminary effectiveness of CTX112, a CD19-directed CAR T cell immunotherapy, in adults with difficult-to-treat autoimmune diseases such as systemic lupus erythematosus SLE, systemic sclerosis SSc, and idiopathic inflammatory myopathy IIM. This Phase 1, open-label, multicenter study involves genetically modified allogeneic T cells from healthy donors using CRISPR-Cas9 gene editing technology. Participants receive CTX112 through an intravenous infusion following lymphodepleting chemotherapy. The study may include up to 80 subjects and focuses on escalating doses of the therapy to assess safety. The treatment period is followed by long-term monitoring to evaluate the drugs effects over time. During the study, participants undergo scheduled visits, laboratory tests, and evaluations to monitor safety and treatment response. Researchers will assess pharmacodynamics, pharmacokinetics, and preliminary efficacy for up to 60 months after infusion. The primary outcome is safety within the first 28 days post-infusion, with extended follow-up to understand longer-term effects.

Researchers are evaluating efruxifermin EFX in a phase 3, randomized, double-blind, placebo-controlled study involving adults with compensated cirrhosis caused by NASH Nonalcoholic Steatohepatitis or MASH Metabolic Dysfunction-Associated Steatohepatitis. This study aims to assess the safety and effectiveness of EFX in preventing significant clinical events such as disease progression and liver decompensation over a period of up to 5 years. Participants are randomly assigned to receive either efruxifermin 50 mg or a placebo, both given by subcutaneous injection. The study includes two cohorts one with biopsy-proven compensated cirrhosis and specific metabolic scores, and another with biopsy-proven or non-invasive diagnosis of compensated cirrhosis. The study treatment and monitoring extend up to 5 years, with evaluations at 96 weeks and long-term follow-up to track liver fibrosis, markers of liver injury, insulin sensitivity, glycemic control, body weight, and safety outcomes. During the trial, participants undergo regular assessments including laboratory tests, ECGs, ultrasounds, and vital sign monitoring. Researchers will measure changes in liver fibrosis, steatohepatitis resolution, and metabolic markers throughout the study. Safety and tolerability are closely tracked by documenting adverse events and exposure duration. The study duration allows for long-term observation of treatment effects and disease progression, with participant involvement lasting up to 5 years.

Researchers are evaluating efruxifermin EFX in adults with non-cirrhotic nonalcoholic steatohepatitis NASH or metabolic dysfunction-associated steatohepatitis MASH who have liver fibrosis stage 2 or 3. This Phase 3, multi-center, randomized, double-blind, placebo-controlled study aims to assess the safety and efficacy of EFX compared with placebo. The trial includes about 1,650 participants divided into two cohorts based on liver biopsy characteristics and fibrosis stage. Participants will be randomly assigned to one of three groups EFX 28 mg, EFX 50 mg, or placebo, each given as a weekly subcutaneous injection. Cohort 1 will be evaluated over 52 weeks for histologic efficacy endpoints, while Cohort 2 will have assessments over 96 weeks. After these periods, participants may continue long-term treatment and clinical follow-up for up to approximately 240 weeks total. A follow-up visit will occur about 30 days after the last dose. During the study, participants will undergo liver biopsies, blood tests, and non-invasive assessments such as FibroScan and Enhanced Liver Fibrosis ELF score to monitor liver health and fibrosis. Researchers will track liver-related clinical outcomes, including liver events and survival, as well as safety and tolerability of the treatment. Participants who stop the study drug may still continue with scheduled assessments to support long-term safety and efficacy evaluations.

Researchers are evaluating DR-01, a non-fucosylated human immunoglobulin G1 monoclonal antibody, in adult patients with large granular lymphocytic leukemia or various cytotoxic lymphomas. This first-in-human, multicenter Phase 12 study aims to assess the safety, tolerability, pharmacokinetics, pharmacodynamics, and anti-tumor activity of DR-01 in these patient populations. The study is sponsored by Dren Bio and involves sequential dose escalation and expansion cohorts to find the optimal treatment regimen. Participants will receive DR-01 at different starting doses ranging from 0.3 mgkg to 10 mgkg, following one of three dosing schedules during the first month bi-weekly dosing, dosing on days 1, 8, 15, and 29, or dosing on days 1-5, 15, and 29. After the first month, dosing continues monthly with doses adjusted up to 10 mgkg for up to 25 cycles. Dose escalation and de-escalation cohorts are included, and optimized dosing regimens are evaluated separately for leukemia and lymphoma subjects in expansion phases. Throughout the study, participants will be monitored for adverse events, dose-limiting toxicities, and overall response rates using disease-specific criteria. Safety and pharmacological assessments will occur up to 25 months, with special focus on the first 28 days for dose-limiting toxicities and up to 6 months for dose optimization. Participants will undergo regular evaluations including clinical assessments and tissue biopsies when applicable. The study aims to determine the best dosage while closely monitoring safety and treatment effects over two years.

Idiopathic Hypersomnia IH is a condition where adults feel extremely sleepy during the day, especially in the morning, even after sleeping a lot at night. This condition can cause difficulty waking up, trouble focusing, and symptoms like dizziness. Researchers are studying TAK-360, a drug that acts like orexin, a brain chemical that helps keep people awake. The study aims to understand how safe and tolerable TAK-360 is for adults with IH and to find the right dose to help with wakefulness. Participants in the study will be randomly assigned to receive either TAK-360 tablets or placebo tablets that look the same but contain no active medicine. Both treatments will be taken orally for 4 weeks. The study uses a double-blind design, meaning neither participants nor researchers know who receives the drug or placebo, to fairly evaluate the effects of TAK-360. During the study, participants will be monitored for safety and side effects up to 8 weeks. Researchers will measure changes in daytime sleepiness using the Epworth Sleepiness Scale and symptoms using the Idiopathic Hypersomnia Severity Scale at the start and after 4 weeks. The trial will also track any treatment-emergent adverse events. The total participation time may include screening, treatment, and follow-up assessments over several weeks.

Narcolepsy Type 1 NT1 is a sleep disorder characterized by excessive daytime sleepiness and sudden muscle weakness while conscious, known as cataplexy. This trial evaluates the safety and tolerability of TAK-360 in adults with NT1. Participants must stop their current NT1 medications before starting the study treatment. They may be randomly assigned to receive either TAK-360 or a placebo, which looks identical but contains no active medicine. The trial is sponsored by Takeda and uses a randomized, double-blind design. Participants will take TAK-360 or matching placebo tablets orally. The study includes multiple parts where participants receive either TAK-360 or placebo in a parallel design. After the treatment period lasting up to about 28 weeks, participants will be monitored for an additional two weeks. Following study treatment, participants can resume their usual NT1 medications. Throughout the trial, participants will visit the clinic multiple times for evaluations. Researchers will assess treatment-emergent adverse events, sleep latency using the Maintenance of Wakefulness Test, sleepiness levels with the Epworth Sleepiness Scale, and weekly cataplexy rates. Safety and tolerability will be closely monitored, and participants usual care will continue after the study ends.

Narcolepsy Type 2 NT2 is a lifelong condition causing excessive daytime sleepiness, difficulty staying awake, and problems with concentration and memory, impacting daily life activities. Researchers are evaluating TAK-360, a drug that acts like orexin, a brain chemical that promotes wakefulness, to understand its safety, tolerability, and ability to help adults with NT2 stay awake. This is a Phase 2, randomized, double-blind, placebo-controlled study to find the right dosage of TAK-360. Participants will be randomly assigned to receive either TAK-360 tablets or matching placebo tablets orally for 4 weeks. TAK-360 is being studied to assess its effects compared to placebo. The study uses a parallel design and includes a 4-week treatment period where participants take the assigned tablets daily. During the study, participants will be monitored for treatment-emergent adverse events for up to 15 weeks. Researchers will assess wakefulness using tools like the Epworth Sleepiness Scale and the Maintenance of Wakefulness Test at baseline and after 4 weeks of treatment. Safety, tolerability, and efficacy data will be collected throughout the study, which runs until November 2026.