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ALPN-101

Phase 2

Systemic Lupus Erythematosus | Small molecule | Immunology |Vertex Pharmaceuticals Incorporated|Last Updated: Aug 15, 2025

Success Probability

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLED
Total Trials1
Total Enrollment76

FDA Designations

No designations recorded

Clinical trial landscape

ALPN-101 · 2 trials · 2 indications

Phase 2 1Phase 1 1
NCT04835441ALPN-101 (Acazicolcept) in Systemic Lupus ErythematosusSystemic Lupus Erythematosus
COMPLETED76 Analytics
PHASE2COMPLETED
ALPN-101 (Acazicolcept) in Systemic Lupus Erythematosus
Systemic Lupus ErythematosusUnlock trial analytics

Study Endpoints

Primary Endpoints

Safety and Tolerability as Assessed by Number of Participants With Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Day 1 up to Safety follow-up (up to 28 weeks)
Percentage of Participants Achieving a Systemic Lupus Erythematosus (SLE) Responder Index (SRI)-4
At Day 169

The SRI-4 is a composite index of SLE disease improvement that consists of scores derived from the SLE Disease Activity Index 2000 (SLEDAI-2K), the British Isles Lupus Assessment Group (BILAG) 2004 Index, and the Physician's Global Assessment (PGA). Participants classified as responder if they met all of the following criteria: 1) ≥ 4-point reduction in the SLEDAI-2K total score; 2) no new severe disease activity (BILAG A organ score) or more than 1 new moderate organ score (BILAG B) compared with baseline; and 3) No worsening from baseline in participants' lupus disease activity (i.e., increase of ≥0.3 0 on a 3-point scale) in PGA. The SLEDAI-2K total score falls between 0 and 105, with higher scores representing increased disease activity. SLEDAI-2K: assesses improvement in disease activity (range: 0 to 105; higher score = higher severity). BILAG: assesses disease extent, severity (range: A\[severe\] to E\[no disease\]). PGA: assesses worsening in participant's general health.

Percentage of Participants Achieving a British Isles Lupus Assessment Group-based Composite Lupus Assessment (BICLA) Response
At Day 169

The BICLA is a responder index developed to measure response to therapy, and it includes scores from the BILAG, SLEDAI-2K, and Physician's Global Assessment (PGA). BICLA response is defined as: 1) at least 1 gradation of improvement in baseline BILAG 2004 scores in all body systems with moderate disease activity at entry (eg, all B \[mild disease\] scores falling to C \[Stable and mild\], or D \[no activity\]); 2) no new BILAG A or more than 1 new BILAG B scores; 3) no worsening of total SLEDAI-2K score from baseline; 4) ≤ 10% deterioration in PGA score. The PGA is measured on a 0 to 100 mm scale with score 0 indicates No Disease Activity and score 100 indicates the most Severe Disease Activity.

Safety, as assessed by adverse events according to CTCAE v5.0
From study Day 1 (dosing of ALPN-101 or placebo) until the End of Study visit (approx. 4-8 weeks)

The rate of adverse events will be compared between the 2 treatment groups.

Secondary Endpoints

Annualized Flare Rate by British Isles Lupus Assessment Group (BILAG)-2004 Flare Index
From Baseline to Day 169
Time-to-first Flare by BILAG-2004 Flare Index
From Baseline to Day 169
Percentage of Participants Achieving a Lupus Low Disease Activity State (LLDAS)
At Day 169
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Study Design & Arms

AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
ALPN-101 (Acazicolcept)EXPERIMENTALParticipants received a weight-based dose of 3 milligrams/kilogram (mg/kg) ALPN-101 once every 2 weeks (Q2W) up to 24 weeks.
PlaceboPLACEBO_COMPARATORParticipants received placebo matched to ALPN-101 up to 24 weeks.
Single Ascending Dose ALPN-101ACTIVE_COMPARATOR -
Single Dose PlaceboPLACEBO_COMPARATOR -
Multiple Ascending Dose ALPN-101ACTIVE_COMPARATOR -
Multiple Dose PlaceboPLACEBO_COMPARATOR -

Interventions

NameTypeDescription
ALPN-101DRUGIntravenous infusion via an infusion pump.
PlaceboDRUGIntravenous infusion via an infusion pump.
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Eligibility Criteria

Age Range18 Years to 70 Years
SexALL
Healthy VolunteersNo
Study Sites46

Key Inclusion Criteria Summary * SLE onset ≥ 6 months prior to Screening * Positive ANA and/or elevated anti-dsDNA and/or elevated anti-Smith antibody test * Active lupus at Screening and Baseline, as defined per-protocol and confirmed by the study's medical monitor, including a SLEDAI score at Scr...

Countries:United StatesFranceHungaryPolandPuerto RicoSpainTaiwanAustralia
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Frequently asked questions about ALPN-101

What is ALPN-101?

ALPN-101, also known as acazicolcept, is an investigational small molecule being developed by Vertex Pharmaceuticals Incorporated (VRTX). It has been studied in healthy volunteers and in patients with systemic lupus erythematosus (SLE). The drug is currently in clinical development and is not approved by the FDA.

What is ALPN-101 used for in systemic lupus erythematosus?

ALPN-101 (acazicolcept) is being studied for the treatment of systemic lupus erythematosus (SLE). A Phase 2 clinical trial evaluated its safety and efficacy in adult patients with SLE across multiple countries, including the United States, France, Hungary, Poland, Puerto Rico, Spain, and Taiwan.

Who makes ALPN-101?

ALPN-101 is being developed by Vertex Pharmaceuticals Incorporated, a biopharmaceutical company traded on the NASDAQ under the ticker symbol VRTX. The drug is also known by the name acazicolcept.

What phase is ALPN-101 in?

ALPN-101 has completed a Phase 1 trial in healthy volunteers and a Phase 2 trial in patients with systemic lupus erythematosus. Both trials are listed as completed, and the drug remains investigational, meaning it has not received FDA approval.

What clinical trials is ALPN-101 in?

ALPN-101 has been studied in two completed clinical trials. NCT03748836 was a Phase 1 study in 96 healthy volunteers in Australia, and NCT04835441 was a Phase 2 study in 76 patients with systemic lupus erythematosus across multiple countries.

Is ALPN-101 the same as acazicolcept?

Yes, ALPN-101 is also known as acazicolcept. The Phase 2 trial in systemic lupus erythematosus used the name acazicolcept in its title, confirming that both names refer to the same investigational drug.