Five Clinical Trial Readouts to Watch in 2025

As the fields of cell therapy and gene therapy continue to advance, more and more clinical trials for such products are currently ongoing within oncology, neurology, rare diseases, and other disease areas. The coming year promises to be as pivotal a period as any, with a number of biotech companies having already announced expected data readouts for ongoing studies in a diverse range of indications. These studies hold the potential to not only provide direct evidence of the efficacy and safety of the individual products themselves, but also to enhance our understanding of the broader modalities and the complex diseases themselves.

Typically, when a trial is launched and the first patient is dosed, the study sponsors will provide an estimate of when initial clinical data is expected to be reported. Similarly, when a major update is provided regarding product for which some clinical data has already been announced, the sponsors will note when the next readout, with longer-term follow-up, is to be expected. As such, we've compiled a list of some of our recent coverage of such announcements for trials with readouts expected in 2025, with links back to the full articles containing additional details regarding the investigational candidates in question.

Click the read more buttons for more details and information about each update.

Longer-Term Data Anticipated from Intellia Therapeutics’ Ongoing Phase 1/2 Study for Hereditary Angioedema Gene Editing Therapy NTLA-2002

January 23, 2025 - The first patient has been dosed in Intellia Therapeutics’ pivotal phase 3 HAELO clinical trial (NCT06634420) evaluating NTLA-2002, an investigational CRISPR/Cas9-based gene-editing therapy that is delivered systemically as a single-dose, for the treatment of hereditary angioedema (HAE).

HAELO takes the form of a double-blind, placebo-controlled study, and will randomly assign participants in a 2:1 ratio to receive either a 50mg dose of NTLA-2002 or placebo. The trial is expected to enroll 60 patients in total, and includes adults with both type 1 and type 2 HAE. Participants who are assigned to the placebo group may cross over to receive the gene-editing therapy at 28 weeks posttreatment. The number of HAE attacks and the number of patients who are attack-free from weeks 5 through 28 posttreatment will serve as key end points for the study. HAELO is currently recruiting patients at sites in Arizona, California, Colorado, and Ohio in the United States and at a site in Ontario, Canada. Intellia anticipates that enrollment in the study will be finished in the second half of this year.

NTLA-2002 is also currently being evaluated in an ongoing phase 1/2 clinical trial (NCT05120830) for adults with HAE, which launched on December 10, 2021. NTLA-2002 is a CRISPR/Cas9-based, in vivo genome editing therapy intended to inactivate the kallikrein B1 (KLKB1) gene to reduce plasma kallikrein protein activity and prevent HAE attacks.

“We are pleased to have initiated dosing in the HAELO phase 3 study as we are in our final lap of clinical development for NTLA-2002,” John Leonard, MD, the president and chief executive officer of Intellia, said in a statement. “With the promising data we’ve presented thus far, we believe patients could achieve independence from both HAE attacks and medications required to treat this disease. We look forward to presenting longer-term data from the ongoing phase 1/2 study later this year highlighting the durability of effect of NTLA-2002.”

First Patient With Multiple Myeloma Dosed in EsoBiotec's Trial for In Vivo CAR-T ESO-T01

January 9, 2025 - The first patient has been dosed in an early phase 1 investigator-initiated clinical trial (NCT06691685) taking place in China for EsoBiotec's ESO-T01, an investigational BCMA-directed in vivo chimeric antigen receptor T-cell (CAR-T) therapy being evaluated for the treatment of relapsed/refractory multiple myeloma (MM).

According to EsoBiotec, this is the first time a patient has been treated with an in vivo BCMA-directed CAR-T therapy in a clinical trial. ESO-T01 is intended to reprogram patients’ own T-cells within their body via the use of EsoBiotec’s ENaBL, a third-generation immune-shielded cell specific lentiviral vector platform, and their collaborator Pregene Biopharma’s BCMA CAR-T transgene. EsoBiotec states that their approach may be able to provide the treatment at a cost 1 order of magnitude lower than the cost of ex vivo CAR-T therapy.

“ESO-T01 has the potential to offer a simplified patient journey, with several benefits over current treatments for MM that are often costly and have unfavorable side effects,” EsoBiotec CEO Jean-Pierre Latere, PhD, said in a statement. “Our treatment does not require lymphodepletion, is immediately available to patients without any waiting time, and is administrated in a single intravenous dose that takes less than 10 minutes. This is why we value our collaboration with Pregene Biopharma to develop ESO-T01, as we share the common goal of making this groundbreaking treatment affordable and available to patients globally. We look forward to sharing initial clinical data in the second half of 2025.”

First Patient Dosed in RIDGE-1 Trial for Tenaya’s ARV Cardiomyopathy Gene Therapy TN-401

November 26, 2024 - The first patient has been dosed in the phase 1b RIDGE-1 clinical trial (NCT06228924) evaluating Tenaya Therapeutics’ TN-401, an investigational adeno-associated virus (AAV)-based gene therapy, for the treatment of PKP2-associated arrhythmogenic right ventricular cardiomyopathy (ARVC).

The patient’s dosing took place at the University of California, San Francisco, although the multicenter study is expected to eventually dose patients at other locations in the United States, United Kingdom, and Europe. The patient received a dose of 3x10¹³ vg/kg of TN-401, which in preclinical research was associated with “near-maximal efficacy”. Two more patients are expected to be treated at this dose level, sequentially, before potential dose escalation and/or expansion pending an independent safety review panel’s advice based on the results obtained.

The study is expected to enroll approximately 15 patients in total. To participate, patients are required to have an implantable cardioverter defibrillator and to undergo a premature ventricular contraction count during screening with results indicating that they have an elevated risk of experiencing arrhythmias. Tenaya anticipates reporting initial data from the trial next year.

First Patient Dosed in Trial for Adicet's T-Cell Therapy ADI-100 in Autoimmune Disease

November 19, 2024 - The first patient, who has lupus nephritis (LN), has been dosed in Adicet Bio’s phase 1 clinical trial (NCT06375993) evaluating ADI-100, its investigational allogeneic chimeric antigen receptor (CAR)-engineered gamma delta T-cell therapy, for the treatment of autoimmune diseases.

In addition to patients with LN, the trial will also include patients with systemic lupus erythematosus (SLE), systemic sclerosis (SSc), antineutrophil cytoplasmic autoantibody associated vasculitis (AAV), idiopathic inflammatory myopathy (IIM), and stiff person syndrome (SPS). Adicet stated that it expects to begin enrolling patients with SLE, SSc, IIM, and SPS in the first half of next year. On the other hand, enrollment of patients with AAV is expected to begin in the second half of next year. Adicet additionally noted that it plans to announce initial data from patients with LN in the first half of 2025.

Sangamo Reaches Accord With FDA on Plans for Accelerated Approval Pathway for Fabry Disease Gene Therapy ST-920

October 24, 2024 - Sangamo Therapeutics has emerged from a Type B interaction with the FDA having come into alignment with the agency on plans to pursue an accelerated approval pathway for isaralgagene civaparvove (ST-920), an investigational adeno-associated virus vector-based gene therapy product intended to treat Fabry disease.

The company noted that based on the interaction it intends to use 1 year posttreatment estimated glomerular filtration rate (eGFR) slope data from patients treated in the ongoing phase 1/2 STAAR clinical trial (NCT04046224) as an intermediate clinical end point for a planned biologics license application (BLA) submission. The company noted that it expects to submit a BLA in the second half of next year and that the use of the accelerated approval pathway has potential to bring the therapy to market about 3 years sooner than a traditional approval pathway would allow for because an additional clinical trial will not be necessary. It also stated that the complete data set to be used to support the BLA will be reported in the first half of next year.