A Long-Term Safety and Efficacy Study of Verekitug (UPB-101) in Adult Participants with Severe Asthma

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Upstream Bio Inc.

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Respiratory Tract Diseases [C08]

Trial duration

20 Oct 2025 → ongoing

Decision date (initial)

2025-08-12

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

Upstream Bio Inc.

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Pharmacokinetic, Efficacy, Others, Dose response, Pharmacodynamic, Therapy, Safety

To check if long-term treatment with verekitug is safe and tolerable for adults with severe asthma.

Secondary objectives 1

1. To see how verekitug affects asthma attacks, breathing ability, airway inflammation, and overall asthma management; to learn how the body processes verekitug; and to check for any immune response to verekitug.

Conditions and MedDRA coding

Severe Asthma

Study design 2 periods

Regulatory references

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 3

1. 1. Completion of the VALIANT study per protocol. Participants must have not met VALIANT discontinuation criteria and have attended the VALIANT EOS visit.
2. 2. Participant has signed, dated, and received a copy of the Institutional Review Board (IRB)/Independent Ethics Committee (IEC)-approved written informed consent form (ICF) for the LTE study.
3. 3. Contraceptive use by participants must be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.

Exclusion criteria 6

1. 1. Abnormal medical history, physical finding, or safety finding that in the opinion of the Investigator may obscure the study data or interfere with the participant’s safety.
2. 2. Any clinical laboratory test result outside of the reference ranges considered by the Investigator as clinically significant and that may obscure the study data or interfere with the participant’s safety.
3. 3. Participant with a history or evidence of a clinically significant pulmonary condition (other than asthma), including significant restrictive findings on pulmonary function testing, chronic bronchitis, emphysema, bronchiectasis, pulmonary fibrosis, or any other related condition that may obscure the study data (e.g., gastroesophageal reflux and vocal cord paralysis/dysfunction).
4. 4. Pregnant or breastfeeding or planning to become pregnant or breastfeed during the study or unwilling to use adequate birth control, if of reproductive potential and sexually active.
5. 5. Participants with significant protocol deviations in VALIANT, assessed at the discretion of the Sponsor.
6. 6. Participant is unreliable, incapable of adhering to the protocol and visit schedule according to the judgment of the Investigator or has any disorder that may compromise their ability to give informed consent.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. To check if verekitug is safe, participants will be monitored for any side effects from the time the participant agrees to join the study until the participant completes the study. Participants will also have physical examinations, clinical laboratory assessments, vital sign measurements, and ECGs from the first LTE study visit, or last VALIANT study visit, until the participant completes the LTE study.

Secondary endpoints 3

1. To check the effects of verekitug, the number of severe asthma flare-ups or attacks that require treatment with steroids over 60 weeks of treatment will be recorded.
2. The change in the following from the start of the LTE study up to Week 60 will be recorded: how much air the participant can forcefully exhale in 1 second; how much nitric oxide is in the exhaled air as a measure of airway inflammation; and how well the participant’s asthma is being managed via a scored questionnaire about asthma symptoms and treatment.
3. The amount of verekitug in the blood will be measured at each visit to see how the body processes verekitug. The blood will also be checked for antibodies against verekitug during the LTE study (from the first to last visit) to check for an immune response.

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Upstream Bio Inc.

Sponsor organisation

Upstream Bio Inc.

Address

890 Winter Street Suite 200

City

Waltham

Postcode

02451-1490

Country

United States

Scientific contact point

Organisation

Upstream Bio Inc.

Contact name

James Lee

Public contact point

Organisation

Upstream Bio Inc.

Contact name

James Lee

Third parties 6

Locations

6 EU/EEA countries · 31 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 62 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

4 submissions — initial application plus substantial / non-substantial modifications