What Is Amlitelimab?
Amlitelimab is an investigational drug currently being studied in clinical trials. It is administered as an injection solution, given subcutaneously (under the skin). While the specific mechanism of action is not detailed in the provided trial descriptions, the drug is being investigated for its potential to treat various inflammatory and autoimmune conditions.
To date, Amlitelimab has been studied in 17 clinical trials, with a total enrollment of 8,998 participants. The first trial began on November 23, 2021, and the latest started on November 13, 2024. Most of these studies are sponsored by Sanofi, with additional sponsorship from Kymab Limited and the Scleroderma Research Foundation, Inc.
Uses and Conditions Under Study
Amlitelimab is being investigated for a range of conditions, primarily focusing on inflammatory and autoimmune disorders. These studies aim to evaluate the drug's safety and effectiveness across different patient populations.
- Skin Conditions: The most significant area of study is for skin-related inflammatory conditions. Amlitelimab is being evaluated in 9 trials for Dermatitis Atopic, commonly known as eczema, which is a chronic inflammatory skin condition causing dry, itchy patches. It is also being studied in 1 trial for general Eczema and in 1 trial for Hidradenitis, a chronic inflammatory skin disease characterized by painful lumps.
- Respiratory Conditions: Two trials are investigating Amlitelimab for Asthma, a chronic respiratory condition that causes airways to narrow and swell. Additionally, 1 trial is exploring its use in Interstitial Lung Disease Due to Systemic Disease, a group of disorders that cause progressive scarring of lung tissue.
- Gastrointestinal Autoimmune Conditions: Amlitelimab is being studied in 1 trial for Celiac Disease and 1 trial for Coeliac Disease (an alternative spelling), both of which are autoimmune disorders where consuming gluten leads to damage in the small intestine.
- Other Autoimmune Conditions: The drug is also under investigation for Alopecia Areata (1 trial), an autoimmune condition causing patchy hair loss, and Scleroderma (1 trial), a group of rare diseases that involve the hardening and tightening of the skin and connective tissues.
- Healthy Volunteers: One trial involves Healthy Volunteers to assess the drug's pharmacokinetics (how the body affects the drug) and safety profile in individuals without specific conditions.
Dosing
Amlitelimab is administered as an injection solution via subcutaneous (SC) injection. Clinical trials have explored various dosages and regimens to determine the most effective and safest treatment options.
Specific doses and formulations studied include:
- Single doses of Amlitelimab at different levels, often referred to as "dose 1," "dose 2," or "dose group 1."
- Specific strengths such as 62.5 mg, 125 mg, and 250 mg.
- Regimens that include a loading dose, such as Amlitelimab 62.5 mg With 125 mg Loading Dose, Amlitelimab 125 mg With 250 mg Loading Dose, and Amlitelimab 250 mg With 500 mg Loading Dose.
- Earlier studies also investigated KY1005, an investigational compound likely related to Amlitelimab, at doses of 62.5 mg, 125 mg, and 250 mg, including a 250mg (500mg Loading Dose) KY1005 and a 250mg (No Loading Dose) KY1005.
- Some studies have also examined Amlitelimab in combination with other substances, such as CYP substrates or gluten-free products (GFP), to understand potential drug interactions or efficacy in specific dietary contexts.
Side Effects
In clinical trials, the overall rate of at least one treatment-emergent adverse event (TEAE) was similar across different doses of Amlitelimab (ranging from 66.2% to 67.9% of participants) compared to 60.3% of participants on placebo. The most commonly reported side effects in patients taking Amlitelimab included:
- Nasopharyngitis (common cold symptoms): 10.5% of patients taking Amlitelimab experienced this, compared to 10.4% on placebo.
- Bronchitis: 9.7% of patients taking Amlitelimab experienced this, compared to 6.3% on placebo.
- Headache: 6.8% of patients taking Amlitelimab experienced this, compared to 4.1% on placebo.
- Acute Sinusitis: 4.8% of patients taking Amlitelimab experienced this, compared to 1.6% on placebo.
- Viral Upper Respiratory Tract Infection: 4.5% of patients taking Amlitelimab experienced this, compared to 2.3% on placebo.
Certain conditions, such as asthma and atopic dermatitis, were reported as adverse events but occurred less frequently in patients treated with Amlitelimab compared to placebo. For example, 33.9% of patients on Amlitelimab reported asthma, while 41.7% on placebo did. Similarly, 18.1% of patients on Amlitelimab reported atopic dermatitis, compared to 40.3% on placebo.
Clinical Trial Results
Atopic Dermatitis (The STREAM-AD Study - NCT05131477)
In a study evaluating Amlitelimab for moderate-to-severe atopic dermatitis, patients experienced significant improvements in skin symptoms and quality of life. In Part 1 of the study, patients receiving Amlitelimab 250 mg with a 500 mg loading dose showed an average reduction of up to 68.01% in their Eczema Area and Severity Index (EASI) score, compared to a maximum reduction of 33.72% in the placebo group. A higher percentage of patients on Amlitelimab achieved substantial skin clearance:
- EASI 75 (at least 75% reduction in EASI score): Up to 54.5% of patients on Amlitelimab 250 mg (with loading dose) achieved this, compared to 17.7% on placebo.
- IGA 0/1 (clear or almost clear skin with at least a 2-point improvement): Up to 45.5% of patients on Amlitelimab 250 mg (with loading dose) achieved this, compared to 11.4% on placebo.
- Pruritus NRS (itch severity) reduction of at least 4 points: Up to 31.2% of patients on Amlitelimab 250 mg (with loading dose) experienced this improvement, compared to 7.6% on placebo.
In Part 2, which included re-randomized patients, those who continued on Amlitelimab showed sustained and further improved responses. For example, patients re-randomized to Amlitelimab 250 mg with a loading dose achieved an average EASI score reduction of up to 93.56% from baseline, while those continuing on placebo from Part 1 had an average reduction of up to 78.51%.
Moderate-to-Severe Asthma (Dose Ranging Study - NCT05421598)
Amlitelimab was also studied in adult patients with moderate-to-severe asthma. Over 48 weeks, patients treated with Amlitelimab 125 mg with a 250 mg loading dose experienced a lower annualized rate of severe asthma exacerbations (0.312 exacerbations per patient-year) compared to 0.598 exacerbations per patient-year in the placebo group. Key improvements in asthma control and lung function were also observed:
- Asthma Control Questionnaire-5 (ACQ-5) Score: At Week 48, patients on Amlitelimab 250 mg (with loading dose) showed an average improvement of -1.25 points (lower scores indicate better control) compared to -0.83 points for placebo.
- Forced Expiratory Volume in One Second (FEV1): At Week 48, patients on Amlitelimab 125 mg (with loading dose) had an average increase of 0.19 liters in pre-bronchodilator FEV1, compared to 0.09 liters for placebo.
- St. George's Respiratory Questionnaire (SGRQ) Total Score: At Week 48, 66.1% of patients on Amlitelimab 250 mg (with loading dose) achieved at least a 4-point decrease in their SGRQ score (indicating improved quality of life), compared to 48.8% on placebo.
Currently Recruiting Trials
Amlitelimab is currently being investigated in several clinical trials, offering opportunities for patients to contribute to medical research. These studies aim to evaluate the drug's effectiveness and safety across various conditions.
One ongoing Phase 2 study, NCT06195072, is titled "Platform Clinical Study for Conquering Scleroderma." Sponsored by the Scleroderma Research Foundation, Inc., this trial evaluates investigational products, including Amlitelimab and BI 1015550 (Nerandomilast), against a placebo. Researchers are observing changes in lung capacity over 52 weeks in participants with Interstitial Lung Disease Secondary to Systemic Sclerosis. The study seeks to enroll 400 individuals.
Another trial, NCT06241118, is a Phase 3 study investigating Amlitelimab for moderate-to-severe Atopic Dermatitis. Sponsored by Sanofi, this multinational, randomized, double-blind, placebo-controlled study will assess the efficacy and safety of two different subcutaneous Amlitelimab doses. It is designed for participants aged 12 years and older who have not responded adequately to prior biologic therapy or oral JAK inhibitors, while continuing background topical corticosteroids. The target enrollment for this study is 636 participants.
A long-term extension study, NCT05492578, is also underway, focusing on the safety and efficacy of Amlitelimab in individuals who previously participated in Amlitelimab clinical trials for moderate to severe Atopic Dermatitis. This open-label Phase 2/Phase 3 study, sponsored by Sanofi, aims to enroll 1,663 participants, offering continued treatment with two different Amlitelimab dose levels.
Where to Participate
Amlitelimab clinical trials are accessible across a broad geographic area, with study sites in 39 states, encompassing 98 cities and a total of 144 locations. This widespread presence aims to make participation convenient for a diverse group of patients.
Top participating cities include:
- Philadelphia, Pennsylvania (7 sites)
- Los Angeles, California (7 sites)
- Miami, Florida (7 sites)
- New York, New York (6 sites)
- Birmingham, Alabama (4 sites)
- Chicago, Illinois (4 sites)
- Dallas, Texas (4 sites)
- Scottsdale, Arizona (3 sites)
- Tampa, Florida (3 sites)
- Portland, Oregon (3 sites)
General eligibility criteria for some Amlitelimab trials include individuals aged 12 to 18 years, with participation open to all genders. Healthy volunteers are not typically sought for these studies, as they focus on specific medical conditions.
Development Timeline
The journey of Amlitelimab in clinical development began on November 23, 2021, with its first clinical trial. Since then, the program has grown significantly, encompassing a total of 17 trials and involving nearly 9,000 participants.
Sanofi has been the primary driver of Amlitelimab's development, sponsoring 15 of these trials, with additional support from Kymab Limited and the Scleroderma Research Foundation, Inc.
Initially, Amlitelimab was explored for conditions such as IBS-C and hyperphosphatemia. Over time, its potential has led to an expansion into a wider range of indications. The development pipeline now includes studies for Celiac Disease, Eczema, Hidradenitis, Interstitial Lung Disease Due to Systemic Disease, Alopecia Areata, Scleroderma, and Atopic Dermatitis.
The drug's progression through clinical phases reflects this expansion, with two Phase 1 trials, nine Phase 2 trials, five Phase 3 trials, and one Phase 2/Phase 3 study currently recorded, demonstrating a robust and evolving research program.