What is the literature supporting the use of Lenacapavir?

Comment by InpharmD Researcher

Recent guidelines currently recommend use of long-acting ibalizumab and/or lenacapavir as an addition to an optimized background regimen (OBR) for heavily treatment-experienced adults with multidrug-resistant HIV-1 with limited anticipated activity from oral antiretroviral agents alone. This recommendation is primarily supported by the phase 3 CAPELLA trial, in which patients who received lenacapavir had a greater reduction from baseline in viral load than those who received placebo in patients with multidrug-resistant HIV-1 when added to OBR. The PURPOSE trial series will aim to evaluate subcutaneous lenacapavir as a single agent for pre-exposure prophylaxis in high-risk populations, with preliminary data from PURPOSE 1 finding superiority of lenacapavir over Truvada. Additionally, a phase 1b trial supports the tolerability and potential efficacy of lenacapavir in combination with broadly neutralizing antibodies (teropavimab and zinlirvimab) in virologically suppressed adults with HIV-1 infection. Oral lenacapavir is also being studied in combination with other oral antiretrovirals for the treatment of naive patients, but studies are still ongoing.

Background

A recent guideline endorsed by various human immunodeficiency virus (HIV) consensus groups and the American College of Clinical Pharmacy recommends use of long-acting (LA) ibalizumab and/or lenacapavir as an addition to optimized background regimen (OBR) for heavily treatment-experienced adults with multidrug-resistant HIV-1 with limited anticipated activity from oral antiretroviral (ART) agents alone. In general, ibalizumab or lenacapavir for HIV-1 treatment are indicated for heavily treatment-experienced adults with multidrug-resistant HIV-1 failing their current ART regimen. Evidence to support the use of lenacapavir in highly treatment-experienced people with multidrug-resistant HIV-1 is supported by the Phase 3 CAPELLA trial (see Table 1). Of note, lenacapavir has low systemic clearance, resulting in a half-life longer than 1 week for the oral product, and greater than 2 months for the subcutaneous injection due to slow-release kinetics. Oral lenacapavir is being studied in combination with other oral antiretrovirals for the treatment of naive patients, while injectable lenacapavir is being studied as a single agent administered subcutaneously every 6 months for pre-exposure prophylaxis (PrEP). [1]

Clinicians should reserve lenacapavir for individuals in whom a suppressive ART regimen cannot be constructed based on available alternatives, have access to the medication, and are able to receive subcutaneous administration every 6 months. More research is necessary to determine the optimal timing and frequency of efficacy monitoring for people with HIV-1 receiving LA ART treatment with lenacapavir. Additionally, resistance testing, if available, in cases of self-discontinued LA lenacapavir, or among individuals lost to follow-up, is recommended to evaluate susceptibility to the background ART regimen. If lenacapavir requires discontinuation due to adverse events, it may remain detectable in the systemic circulation for a prolonged period of up to nine months after discontinuation. To minimize risk of developing viral resistance, an alternative antiretroviral should replace lenacapavir in the fully suppressive regimen no later than 28 weeks after the final injection of lenacapavir. [1]

The PURPOSE trials are an ongoing series of trials that aim to evaluate the safety and efficacy of twice-yearly long-acting subcutaneous lenacapavir compared to emtricitabine/tenofovir alafenamide (Descovy®) or emtricitabine/tenofovir disoproxil fumarate (Truvada®) for HIV PrEP in a variety of populations at risk for HIV infection. In the PURPOSE 1 trial, study participants were randomized in a 2:2:1 ratio to lenacapavir, Descovy, or Truvada, respectively. According to a press release by Gilead Sciences, Inc., the manufacturer of lenacapavir, an interim analysis of the PURPOSE 1 trial found lenacapavir twice yearly to demonstrate 100% efficacy for PrEP in cisgender women. PURPOSE 1 also met its key efficacy endpoints of superiority over once-daily oral Truvada and background HIV incidence. There were 0 incidence cases of HIV infection among 2,134 women in the lenacapavir group compared to 16 incidence cases among 1,068 women in the Truvada group (p<0.001). For this reason, the independent data monitoring committee recommended that Gilead stop the blinded phase of the trial and offer open-label lenacapavir to all participants. Gilead expects results in late 2024/early 2025 from the program’s PURPOSE 2 trial, which is assessing twice-yearly lenacapavir for PrEP among cisgender men who have sex with men, transgender men, transgender women, and gender non-binary individuals who have sex with partners assigned male at birth. [2], [3], [4], [5], [6], [7]

One ongoing phase 2 trial will aim to evaluate the combination of lenacapavir with broadly neutralizing antibodies (teropavimab and zinlirvimab) in virologically suppressed adults with HIV-1 infection. This trial comes after a randomized, blinded, phase 1b proof-of-concept study in which adults with a plasma HIV-1 RNA concentration <50 copies/mcL who had at least 18 months on oral ART and CD4 counts of ≥500 cells/mcL stopped their oral ART and were randomized to receive one dose of 927 mg subcutaneous lenacapavir plus an oral loading dose, 30 mg/kg intravenous teropavimab, and 10 mg/kg or 30 mg/kg intravenous zinlirvimab on day 1. No serious adverse events occurred; however, two grade 3 adverse events occurred, including lenacapavir injection-site erythema and injection-site cellulitis, both of which resolved. The most common adverse events were symptoms of injection-site reactions, reported in 17 (85%) of 20 participants who received subcutaneous lenacapavir. One participant in the zinlirvimab 10 mg/kg experienced viral rebound, but overall, HIV-1 suppression for at least 26 weeks was deemed to be feasible and generally well tolerated with this regimen at either zinlirvimab dose in selected people with HIV-1. The phase 2 trial to follow this phase 1b study is scheduled to be completed in December 2029. Another phase 2 trial will aim to evaluate the efficacy of oral weekly islatravir in combination with lenacapavir in virologically suppressed people with HIV; however, the trial is also not scheduled to be complete until November 2027. [8], [9], [10], [11]

An ongoing phase 2/3 multi-center, randomized, open-label, active-controlled study is assessing the safety and efficacy of bictegravir/lenacapavir versus stable baseline regimens (SBR) for those with HIV-1. In the phase 2 period, patients will be randomized to receive bictegravir/lenacapavir 75 mg/25 mg daily, bictegravir/lenacapavir 75 mg/50 mg daily, or a stable baseline regimen for at least 24 weeks. If receiving lenacapavir, patients will receive 600 mg daily loading dose for 2 days. In all groups and after 24 weeks, patients will have the option of enrolling in the extension period, where their regimen can be optimized to receive bictegravir/lenacapavir 75 mg/50 mg fixed dose combination (FDC) tablets. In the phase 3 period, patients will be randomized to either continue SBR or switch to SBR bictegravir/lenacapavir 75 mg/50 mg FDC for at least 48 weeks. An extension enrollment period is offered in this study as well. [12]

This phase 2/3 study led to an ongoing phase 3, randomized, double-blinded, parallel trial which aims to evaluate the effectiveness of switching to bictegravir/lenacapavir FDC versus current therapy bictegravir/emtricitabine/tenofovir alafenamide FDC in virologically suppressed people with HIV-1. Patients will be randomized in parallel in one of two treatment groups during the blinded phase for at least 48 weeks. Group 1 patients currently on bictegravir/emtricitabine/tenofovir alafenamide FDC will switch to bictegravir/lenacapavir 75 mg/50 mg FDC and placebo to match bictegravir/emtricitabine/tenofovir alafenamide FDC. Group 2 patients will continue with bictegravir/emtricitabine/tenofovir alafenamide FDC and start placebo to match bictegravir/lenacapavir. Participants in both treatment groups will be given the option to continue bictegravir/lenacapavir FDC treatment during the open-label phase. The primary outcome of both studies is the proportion of patients with HIV-1 RNA ≥50 copies/mL at week 24 and week 48, respectively. Anticipated study completion of the phase 2/3 and phase 3 study is by November 2028 and December 2029, respectively. [12], [13]

References:

[1] Sherman EM, Agwu AL, Ambrosioni J, et al. Consensus recommendations for use of long-acting antiretroviral medications in the treatment and prevention of HIV-1: Endorsed by the American Academy of HIV Medicine, American College of Clinical Pharmacy, Canadian HIV and Viral Hepatitis Pharmacists Network, European AIDS Clinical Society, and Society of Infectious Diseases Pharmacists. Pharmacotherapy. 2024;44(7):494-538. doi:10.1002/phar.2922
[2] ClinicalTrials.gov. Pre-Exposure Prophylaxis Study of Lenacapavir and Emtricitabine/​Tenofovir Alafenamide in Adolescent Girls and Young Women at Risk of HIV Infection (PURPOSE 1). Updated February 28, 2024. Accessed July 23, 2024. https://clinicaltrials.gov/study/NCT04994509
[3] ClinicalTrials.gov. Study of Lenacapavir for HIV Pre-Exposure Prophylaxis in People Who Are at Risk for HIV Infection (PURPOSE 2). Updated July 12, 2024. Accessed July 23, 2024. https://clinicaltrials.gov/study/NCT04925752
[4] ClinicalTrials.gov. Study of Lenacapavir and Emtricitabine/​Tenofovir Disoproxil Fumarate (F/​TDF) in Prevention of HIV in Cisgender Women in the United States (HPTN 102) (PURPOSE 3). Updated July 23, 2024. Accessed July 23, 2024. https://clinicaltrials.gov/study/NCT06101329
[5] ClinicalTrials.gov. Study of Lenacapavir and Emtricitabine/​Tenofovir Disoproxil Fumarate (F/​TDF) for Prevention of HIV in People Who Inject Drugs (HPTN 103) (PURPOSE 4). Updated July 3, 2024. Accessed July 23, 2024. https://clinicaltrials.gov/study/NCT06101342
[6] ClinicalTrials.gov. Study of Lenacapavir Taken Twice a Year for HIV Pre-Exposure Prophylaxis (PrEP) (PURPOSE 5). Updated July 22, 2024. Accessed July 23, 2024. https://clinicaltrials.gov/study/NCT06513312
[7] Gilead Sciences, Inc. Press Release – Gilead’s Twice-Yearly Lenacapavir Demonstrated 100% Efficacy and Superiority to Daily Truvada® for HIV Prevention. Published June 20, 2024. Accessed July 23, 2024. https://www.gilead.com/news-and-press/press-room/press-releases/2024/6/gileads-twiceyearly-lenacapavir-demonstrated-100-efficacy-and-superiority-to-daily-truvada-for-hiv-prevention#:~:text=Gilead%20expects%20results%20in%20late,assigned%20male%20at%20birth%20in
[8] ClinicalTrials.gov. Study to Evaluate the Safety and Efficacy of Teropavimab and Zinlirvimab in Combination With Lenacapavir (GS-6207) in Virologically Suppressed Adults With HIV-1 Infection. Updated October 27, 2023. Accessed July 23, 2024. https://clinicaltrials.gov/study/NCT04811040
[9] Eron JJ, Little SJ, Crofoot G, et al. Safety of teropavimab and zinlirvimab with lenacapavir once every 6 months for HIV treatment: a phase 1b, randomised, proof-of-concept study. Lancet HIV. 2024;11(3):e146-e155. doi:10.1016/S2352-3018(23)00293-X
[10] ClinicalTrials.gov. A Study of GS-5423 and GS-2872 in Combination With Capsid Inhibitor Lenacapavir in Virologically Suppressed Adults With HIV-1 Infection. Updated July 15, 2024. Accessed July 23, 2024. https://clinicaltrials.gov/study/NCT05729568
[11] ClinicalTrials.gov. Study Evaluating the Safety and Efficacy of Islatravir in Combination With Lenacapavir in Virologically Suppressed People With HIV. Updated January 19, 2024. Accessed July 23, 2024. https://clinicaltrials.gov/study/NCT05052996
[12] ClinicalTrials.gov. Study to Compare Bictegravir/​Lenacapavir Versus Current Therapy in People With HIV-1 Who Are Successfully Treated With a Complicated Regimen (ARTISTRY-1). Updated July 11, 2024. Accessed July 23, 2024. https://clinicaltrials.gov/study/NCT05502341
[13] ClinicalTrials.gov. Study to Compare Bictegravir/​Lenacapavir Versus Current Therapy in People With HIV-1 Who Are Successfully Treated With Biktarvy (ARTISTRY-2). Updated June 21, 2024. Accessed July 23, 2024. https://clinicaltrials.gov/study/NCT06333808

Literature Review

A search of the published medical literature revealed 3 studies investigating the researchable question:

What is the literature supporting the use of Lenacapavir?

Level of evidence

A - Multiple high-quality studies with consistent results  Read more→



Please see Tables 1-3 for your response.


 

Capsid Inhibition with Lenacapavir in Multidrug-Resistant HIV-1 Infection (CAPELLA)

Design

International, randomized, placebo-controlled cohort study

N= 72

Objective

To assess the efficacy and safety of lenacapavir in patients with multidrug-resistant human immunodeficiency virus type 1 (HIV-1) infection

Study Groups

Cohort 1 (n= 36)

Lenacapavir (n= 24)

Placebo (n= 12)

Cohort 2 (n= 36)

Inclusion Criteria

Age ≥12 years, HIV-1 RNA ≥400 copies/mL at screening, currently receiving a stable failing antiretroviral (ARV) regimen >8 weeks before screening, documented resistance ≥2 ARV medications from each of ≥3 of the 4 main classes, and have ≤2 fully active ARV medications remaining from the 4 main classes 

Exclusion Criteria

Active infection (other than HIV-1 infection), opportunistic infection requiring acute treatment within 30 days before screening, estimated glomerular filtration rate ≤60 mL/minute, active or previous (<3 months before screening) treatment with immunosuppressants, systemic steroids, or chemotherapeutic agents

Methods

Patients with stable viremia (<0.5 log10 copies/mL HIV-1 RNA decline) and HIV-1 RNA ≥400 copies/mL were assigned to Cohort 1 and randomized (2:1) to receive oral lenacapavir (600 mg on days 1 and 2 and 300 mg on day 8) or matching placebo in addition to current failing ARV therapy during the functional monotherapy period of 14 days; this part of the study period was double-blinded. The maintenance period started on day 15, with the oral lenacapavir group transitioning to subcutaneous (SC) lenacapavir (927 mg as two 1.5 mL abdominal injections) every 6 months with optimized background regimen (OBR). The placebo group switched to oral lenacapavir (600 mg on days 15 and 16 and 300 mg on day 22) and OBR, then SC lenacapavir every 6 months with OBR.

Cohort 2 included patients with reduced viremia (≥0.5 log10 copies/mL HIV-1 RNA decline), HIV-1 RNA <400 copies/mL, or qualified for enrollment following Cohort 1 cutoff. Patients received open-label oral lenacapavir (600 mg on day 2 and 300 mg on day 8) plus OBR followed by SC lenacapavir once every 6 months plus OBR. 

Virologic resistance analysis was conducted in patients meeting virologic failure criteria: HIV-1 RNA viral load ≥50 copies/mL and decrease of <1 log10 copy/mL at week 4 after oral lenacapavir initiation, confirmed rebound viral load ≥50 copies/mL after previous measure of <50 copies/mL, confirmed increase from nadir value of >1 log10 copy/mL, or viral load ≥50 copies/mL at week 26 or time of trial discontinuation. 

Duration

Enrollment: November 2019 to January 2021

Intervention: 54 weeks

Follow-up: 30, 60, and 90-day visits following treatment discontinuation

Outcome Measures

Primary: percent of patients in Cohort 1 with plasma HIV-1 RNA reduction from baseline of ≥0.5 log10 copies/mL to day 15 (end of functional monotherapy period)

Secondary: At week 26 following SC lenacapavir initiation, percent of patients in Cohort 1 and Cohort 2 with HIV-1 RNA viral load <50 copies/mL, percent of patients with HIV-1 RNA viral load <200 copies/mL, change in viral load, and change in CD4+ count

Baseline Characteristics

 

Cohort 1:

Lenacapavir (n= 24)

Cohort 1:

Placebo (n= 12)

Cohort 2:

Lenacapavir (n= 36)

Age, years (IQR)

55 (24 to 71) 54 (27 to 59) 49 (23 to 78)

Female

7 (29%) 3 (25%)  8 (22%) 

Race

Black

White

Asian

 

10 (42%)

12 (50%)

2 (8%) 

 

6 (55%)

4 (36%)

1 (9%) 

 

11 (31%)

13 (36%)

12 (33%)

HIV-1 viral load, log10 copies/mL

3.97 ± 0.92 4.87 ± 0.39  4.06 ± 1.16 

HIV-1 RNA >100,000 copies/mL

1 (4%) 6 (50%) 7 (19%)

CD4+ count, cells/mm3

<50

50 to <200

200 to <500

≥500

199 ± 166

3 (12%)

13 (54%)

7 (29%)

1 (4%)

85 ± 63

4 (33%)

7 (58%)

1 (8%)

0

258 ± 273

9 (25%)

10 (28%)

12 (33%)

5 (14%)

Resistance to ≥2 drugs in major class

NRTI

NNRTI

PI

INSTI

All 4 major classes

 

23 (96%)

22 (92%)

20 (83%)

20 (83%)

14 (58%) 

 

12 (100%)

12 (100%)

8 (67%)

7 (58%)

3 (25%) 

 

36 (100%)

36 (100%)

30 (83%)

23 (64%)

16 (44%) 

Resistance to entry inhibitor, no./total no.

Enfuvirtide

Fostemsavir

Ibalizumab

Maraviroc

 

2/23 (9%)

5/23 (22%)

8/23 (35%)

19/24 (79%)

 

3/10 (30%)

5/10 (50%)

3/10 (30%)

8/11 (73%)

 

0

7/21 (33%)

6/25 (24%)

14/26 (54%)

Composition of OBT

NRTI

INSTI

PI

NNRTI

Ibalizumab

Maraviroc

Fostemsavir

Enfuvirtide

 

23 (96%)

16 (67%)

12 (50%)

6 (25%)

9 (38%)

2 (8%)

3 (12%)

1 (4%)

 

9 (75%)

9 (75%)

9 (75%)

4 (33%)

3 (25%)

4 (33%)

0

2 (17%)

 

29 (81%)

22 (61%)

24 (67%)

14 (39%)

5 (14%)

4 (11%)

5 (14%)

2 (6%)

Number of fully active agents in OBR

0

1

≥2

 

4 (17%)

7 (29%)

13 (54%)

 

2 (17%)

7 (58%)

3 (25%)

 

6 (17%)

13 (36%)

17 (47%)

IQR, interquartile range; NRTI, nucleoside reverse-transcriptase inhibitor; NNRTI, non-nucleoside reverse transcriptase inhibitor; PI, protease inhibitors; INSTI, integrase strand-transfer inhibitors 

Results

Endpoint

Cohort 1: 

Lenacapavir (n= 24)

Cohort 1:

Placebo (n= 12)

p-value or LSMD (95% CI)

Functional Monotherapy Period end at day 15

HIV-1 RNA reduction from baseline of ≥0.5 log10 copies/mL

Change in viral load, log10 copies/mL

 

21 (88%)

-2.1 ± 0.15

 

2 (17%) 

0.07 ± 0.22

 

<0.001

-2.17 (-2.74 to -1.59)

 

Cohort 1 (n= 36)

Cohort 2 (n= 36)

 

Maintenance Period at Week 26

HIV-1 RNA viral load <50 copies/mL 

HIV-1 RNA viral load <200 copies/mL 

Change in viral load, log10 copies/mL

Change in CD4+ count, cells/mm

 

29 (81%)

32 (89)

-2.58 ± 1.04

+75

 

30 (83%) 

31 (86%)

-2.49 ± 1.34

+104

 

Virologic resistance was seen in 19 patients overall, with 8 patients developing lenacapavir-associated capsid substitutions during the maintenance period of the study. Resuppresion of HIV-1 RNA levels occurred in 4 of these 8 patients during lenacapavir treatment, with 2 patients with persistent viremia, 1 died at week 10 unrelated to study intervention and 1 discontinued treatment at the investigator's discretion at week 4. Of the remaining 11 patients without capsid mutations, 7 patients exhibited suppression of HIV-1 RNA levels while on study treatment. 

Abbreviations: CI, confidence interval; LSMd, least-squares mean difference

Adverse Events

Common Adverse Events: nausea (13% in oral lenacapavir group vs. 0 in placebo group)†; injection site reactions (63%), nausea (12%), constipation (11%), and diarrhea (11%)‡

Serious Adverse Events: No serious adverse events were reported to be related to study drug.

Percentage that Discontinued due to Adverse Events: One patient discontinued treatment due to injection site nodule 10 weeks following week 52 injection.

†Analyzed in cohort 1 during the functional monotherapy period

‡Adverse event analysis included both cohorts 1 and 2, with all 72 patients receiving oral lenacapavir and ≥1 dose of SC lenacapavir.

Study Author Conclusions

In patients with multidrug-resistant HIV-1 infection, those who received lenacapavir had a greater reduction from baseline in viral load than those who received placebo.

InpharmD Researcher Critique

This study had a small sample size and limited follow-up. However, this study shows that lenacapavir is efficacious in the reduction of HIV-1 RNA viral loads and improvement in CD4+ counts in patients with multi-drug resistant HIV-1 when used in combination with optimized background therapy.

References:

Segal-Maurer S, DeJesus E, Stellbrink HJ, et al. Capsid Inhibition with Lenacapavir in Multidrug-Resistant HIV-1 Infection. N Engl J Med. 2022;386(19):1793-1803. doi:10.1056/NEJMoa2115542

 

Lenacapavir Administered Every 26 Weeks or Daily in Combination with Oral Daily Antiretroviral Therapy for Initial Treatment of HIV: A Randomised, Open-label, Active-controlled, Phase 2 Trial

Design

Randomized, multi-center, open-label, active comparator phase 2 trial

N= 183

Objective

To explore the efficacy of lenacapavir in various combination regimens as initial and maintenance therapy for human immunodeficiency virus (HIV)

Study Groups

Group 1 (n= 52)

Group 2 (n= 53)

Group 3 (n= 52)

Group 4 (n= 25)

Inclusion Criteria

Age ≥ 18 years old, antiretroviral (ARV) naive with no use of any ARV < 1 month of screening, use of pre-exposure prophylaxis or post-exposure prophylaxis, HIV-1 ribonucleic acid (RNA) ≥ 200 copies/mL at screening, cluster determinant 4+ (CD4+) cell count ≥ 200 cells/μL at screening

Exclusion Criteria

Current Hepatitis B Virus or Hepatitis C virus infection

Methods

Patients were randomly assigned (2:2:2:1) to four different groups of antiretroviral therapy regimens. 

In both groups 1 and 2, participants received lenacapavir (927 mg) administered subcutaneously (SC) every 26 weeks following a 2-week oral loading period (600 mg on days 1 and 2, followed by 300 mg on day 8). Additionally, participants were given oral daily doses of emtricitabine (200 mg) and tenofovir alafenamide (25 mg) for 28 weeks.

After this initial period, group 1 continued with SC lenacapavir (927 mg) and oral daily tenofovir alafenamide (25 mg), while group 2 received SC lenacapavir (927 mg) in combination with oral daily bictegravir (75 mg).

Group 3 was administered oral daily lenacapavir (600 mg on days 1 and 2, followed by 50 mg daily) along with emtricitabine (200 mg) and tenofovir alafenamide (25 mg). Meanwhile, group 4 received oral daily bictegravir (50 mg), emtricitabine (200 mg), and tenofovir alafenamide (25 mg).

Duration

Enrollment: November 22, 2019 to August 27, 2020

Intervention: ≥ 80 weeks

Follow-up: 38 to 199 days

Outcome Measures

Primary: Percentage of participants with virological suppression (HIV-1 RNA <50 copies per mL) at week 54

Secondary: Percentage of participants with HIV-1 RNA <50 copies/mL at weeks 28 and 38

Baseline Characteristics

 

Group 1 (n= 52)

Group 2 (n= 53)

Group 3 (n= 52)

Group 4 (n= 25)

Age, years (IQR)

31 (26 to 40) 28 (24 to 33) 28 (24 to 36) 29 (26 to 33)

Female

5 (10%) 1 (2%) 6 (12%) 0

Race

American Indian or Alaska Native

Asian

Black

Native Hawaiian or Pacific Islander

White

Other

Latinx ethnicity

 

1 (2%)

1 (2%)

24 (46%)

1 (2%)

23 (44%)

2 (4%)

25 (48%)

 

0

0

24 (45%)

1 (2%)

28 (53%)

0

21 (40%)

 

0

1 (2%)

31 (60%)

0

19 (37%)

1 (2%)

24 (46%)

 

0

0

16 (64%)

0

8 (32%)

1 (4%)

12 (48%)

Cisgender

50 (96%) 50 (94%) 45 (87%) 24 (96%)

Weight, kg (IQR)

78 (64 to 97) 77 (67 to 90) 77 (68 to 94) 80 (72 to 99)
Body mass index, kg/m2 (IQR)  26 (21 to 30) 25 (22 to 29) 26 (22 to 30) 27 (23 to 31)

HIV-1 RNA

Log10 copies/mL (IQR)

>100 000 copies/mL

 

4.3 (3.8 to 4.6)

5 (10%)

 

4.3 (4.0 to 4.7)

9 (17%)

 

4.5 (3.8 to 4.8)

9 (17%)

 

4.4 (4.1 to 4.8)

4 (16%)

CD4+ count, cells/μL (IQR) 404 (320 to 599) 450 (332 to 599) 409 (301 to 600) 482 (393 to 527)

Distribution

<50 cells/μL

≥50 to <200 cells/μL

≥200 to <350 cells/μL

≥350 to <500 cells/μL

≥500 cells/μL

 

0

0

17 (33%)

17 (33%)

18 (35%)

 

0

1 (2%)

15 (28%)

15 (28%)

22 42%)

 

0

3 (6%)

16 (31%)

15 (29%)

18 (35%)

 

0

4 (2%)

52 (29%)

58 (32%)

68 (37%)

IQR, interquartile range

Results

Endpoint

Group 1 (n= 52)

Group 2 (n= 53)

Group 3 (n= 52)

Group 4 (n= 25)

HIV-1 RNA <50 copies/mL at week 54

47 (90%) 45 (85%) 44 (85%) 23 (92%)

HIV-1 RNA <50 copies/mL

Week 28

Week 38

 

49 (94%)

47 (90%)

 

49 (93%)

47 (89%)

 

49 (94%)

46 (89%)

 

25 (100%)

24 (96%)

CD4+ cell count at week 54 (IQR)

219 (102 to 318) 219 (102 to 318) 219 (102 to 318) 177 (30 to 290)

There were no significant differences between groups 1-3 and group 4 for the primary outcome.

Adverse Events

Common Adverse Events: Lencapavir-related injection-site reactions (62% Group 1 vs. 47% Group 2), headache (17% vs. 9% vs. 14% vs. 12%), nausea (19% vs. 9% vs. 12% vs. 4%)

Serious Adverse Events: 6% vs. 6% vs. 8% vs. 0 (none related to study treatment)

Percentage that Discontinued due to Adverse Events: Three participants discontinued treatment with SC lenacapavir in Group 2 due to grade 1 injection-site reactions (induration; erythema/swelling).

Study Author Conclusions

These results show that lenacapavir is well tolerated and effective in combination with other antiretroviral agents at inducing and maintaining suppression of HIV-1. A range of options for antiretroviral therapy, including oral and injectable longer-acting regimens, could help to address the needs of the diversity of people with HIV.

InpharmD Researcher Critique

This study was limited due to its smaller sample size and limited duration. However, the lack of serious adverse events as well as lenacapavir's ability to induce and suppress HIV-1 RNA in combination with other ARV therapies support further investigation of lenacapavir as a viable option for those with HIV.

References:

Gupta SK, Berhe M, Crofoot G, et al. Lenacapavir administered every 26 weeks or daily in combination with oral daily antiretroviral therapy for initial treatment of HIV: a randomised, open-label, active-controlled, phase 2 trial. Lancet HIV. 2023;10(1):e15-e23. doi:10.1016/S2352-3018(22)00291-0

 

Efficacy and Safety of the Novel Capsid Inhibitor Lenacapavir to Treat Multidrug-resistant HIV: Week 52 Results of a Phase 2/3 Trial

Design

Ongoing international randomized, placebo-controlled phase 2/3 study

N= 72

Objective

To evaluate the efficacy and safety of lenacapavir with an optimized background regimen in adults living with multidrug-resistant human immunodeficiency virus-1 (HIV-1) up to 52 weeks

Study Groups

Cohort 1 (n= 36)

- Lenacapavir (n= 24)

- Placebo (n= 12)

Cohort 2 (n= 36)

- Lenacapavir (n= 36)

Inclusion Criteria

Age ≥12 years, received a stable but failing antiretroviral (ARV) therapy (ART) for ≥8 weeks, multidrug resistance (resistance to ≥2 agents from ≥3 of the 4 main classes of ARV medications), and no more than two fully active ARV drugs from the four main classes that could be effectively combined to form a suppressive regimen

Exclusion Criteria

Hepatitis C virus infection

Methods

Patients with stable viremia (HIV-1 RNA of ≥400 copies/mL and a decrease of less than 0.5 log10 copies/mL) were assigned to cohort 1 and randomized (2:1) to receive either oral lenacapavir (600 mg on days 1 and 2, then 300 mg on day 8) or matching placebo with continued failing ART. In the maintenance period, starting at day 15, oral lenacapavir group started subcutaneous (SQ) treatment (972 mg as 2 1.5 mL injections administered every 26 weeks) with an investigator-initiated optimized background regimen. The placebo group received oral lenacapavir (600 mg on days 15 and 16, then 300 mg on day 22) with an optimized background regimen, then switched to SQ lenacapavir. 

Cohort 2 included patients with reduced viremia (HIV-1 RNA of <400 copies/mL or a decrease of at least 0.5 log10 copies/mL) during the screening period, or enrollment after cohort 1 was filled. Patients received an optimized background regimen from day 1 in combination with open-label oral lenacapavir dosing similar to cohort 1. On day 15, they were switched to SQ lenacapavir and continued on an optimized background regimen. Those choosing to continue past 52 weeks are to continue SQ formulation every 26 weeks with an optimized background regimen. 

Duration

Start date was November 2021 and is ongoing

Outcome Measures

Primary: proportion of participants in cohort 1 who had a reduction of plasma HIV-1 RNA from baseline to day 15 of at least 0.5 log10 copies/mL

Secondary: proportions of participants in cohort 1 with plasma HIV-1 RNA of <50 copies/mL or < 200 copies/mL at week 26; HIV-1 RNA of <50 copies/mL or <200 copies/mL at week 52 in cohort 1 and for cohorts 1 and 2 combined

Baseline Characteristics

 

Cohort 1:

Lenacapavir (n=24) 

Cohort 1:

Placebo (n= 12)

Cohort 2:

Lenacapavir (n= 36)

 

Age, years

55 54 49  

Female

29% 25% 22%  

Race

White

Black

Asian

 

50%

42%

8%

 

36%

50%

9%

 

36%

31%

33%

 

Hispanic or Latinx

25%

36%

14%

 

Cisgender

100% 83% 92%  

HIV-1 viral load, log10 copies/mL

3.97 4.87 4.06  

HIV-1 RNA >100,000 copies/mL

4% 50% 19%  

CD4 count, cells/mcL

<50

≥50 to <200

≥200 to <500

≥500

199

13%

54%

29%

4% 

85

33%

58%

8%

0

258

25%

28%

33%

14% 

 

Resistance to ≥2 ARVs in major class

NRTI

NNRTI

PI

INSTI

All four major classes

 

96%

92%

83%

83%

58%

 

100%

100%

67%

58%

25% 

 

100%

100%

83%

64%

44% 

 

Resistance to entry inhibitors

Enfuvirtide 

Fostemsavir

Ibalizumab

Maraviroc

 

9%

22%

35%

79% 

 

30%

50%

30%

73% 

 

0

33%

24%

54% 

 

Composition of optimised background regimen

NRTI

INSTI

PI

NNRTI

Ibalizumab

Maraviroc

Fostemsavir

Enfuvirtide 

 

96%

67%

50%

25%

38%

8%

13%

4.2% 

 

75%

75%

75%

42%

25%

33%

0

17% 

 

81%

61%

67%

39%

14%

11%

14%

6% 

 

Number of fully active agents in the optimized background regimen

0

1

≥2

 

17%

29%

54% 

 

17%

58%

25% 

 

17%

36%

47% 

 

Abbreviations: nucleoside reverse transcriptase inhibitor (NRTI), non-nucleoside reverse transcriptase inhibitor (NNRTI), protease inhibitor (PI), integrase strand transfer inhibitor (INSTI)

Results

Endpoint*

Cohort 1 (n= 36)

Cohort 2 (n= 36) 

p-Value

Baseline CD4 count, cells/mcL

<200 cells/mcL

≥200 cells/mcL

 

21/27 (78%)

9/9 (100%)

 

12/19 (63%)

14/17 (82%) 

 

0.072 

Baseline viral load

≤100,000 copies/mL

>100 000 copies/mL

 

25/29 (86%)

5/7 (71%) 

 

22/29 (76%)

4/7 (57%)

 

0.107 

INSTI resistance

Yes

No

 

22/27 (82%)

7/8 (88%)

 

17/23 (74%)

8/12 (67%)

 

0.450

Use of dolutegravir or daunavir

Both

Dolutegravir only

Darunavir only

Neither

 

10/12 (83%)

5/6 (83%)

8/9 (89%)

7/9 (78%)

 

5/12 (42%)

4/6 (67%)

11/1 (100%)

6/7 (86%)

 

N/A

Use of ibalizumab and fostemsavir

Both

Ibalizumab only

Fostemsavir only

Neither

Either

 

1/1 (100%)

9/11 (82%)

2/2 (100%)

18/22 (82%)

12/14 (86%) 

 

2/2 (100%)

2/3 (67%)

2/3 (67%)

20/28 (71%)

6/8 (75%) 

 

N/A

Adverse events†

Lenacapavir injection site reactions‡

 

64%

 

67%

 

N/A

*Week 52 virological responses and medication use

†Reported events (≥5%) included: diarrhea, nausea, COVID-19, constipation, cough, pyrexia, abdominal distension, back pain, headache, arthralgia, rash, urinary tract infection, dizziness, fatigue, herpes zoster, abdominal pain, insomnia, oral candidiasis, pneumonia, vomiting and weight increase

Reports included swelling, pain, erythema, SQ nodule, induration and pruritus, with most reactions being grade 1 or 2, and only 1 patient discontinuing therapy due to injection site reaction

Adverse Events

See results.

Study Author Conclusions

In individuals with multidrug-resistant HIV-1, SQ lenacapavir in combination with an optimised background regimen was well tolerated and led to high rates of virological suppression and little resistance up to 52 weeks. These data support the use of lenacapavir to treat people living with multidrug-resistant HIV-1 and its ongoing evaluation for a broader population of those living with HIV-1. 

InpharmD Researcher Critique

Limitations include small sample size, which can make subgroup analyses challenging. This study is ongoing with an anticipated completion date in 2027.

References:

Ogbuagu O, Segal-Maurer S, Ratanasuwan W, et al. Efficacy and safety of the novel capsid inhibitor lenacapavir to treat multidrug-resistant HIV: week 52 results of a phase 2/3 trial. Lancet HIV. 2023;10(8):e497-e505. doi:10.1016/S2352-3018(23)00113-3