AELIX Therapeutics Reports Key Findings from the AELIX-002 Study
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AELIX Therapeutics Reports Key Findings from the AELIX-002 Study

Pharma Tech Outlook | Wednesday, March 17, 2021

Among early-treated HIV patients, the study tested the reliability, tolerability, immunogenicity, and effectiveness of AELIX's HTI T-cell therapeutic HIV vaccine.

FREMONT, CA: AELIX Therapeutics S.L. (AELIX), a clinical-stage biotechnology corporation focused on discovering and advancing HIV immunotherapies, reported promising topline findings from the AELIX-002 study. Among early-treated HIV patients, the study tested the reliability, tolerability, immunogenicity, and effectiveness of AELIX's HTI T-cell therapeutic HIV vaccine. The trial's findings were discussed today at the "abstract link" of the 2021 Conference on Retroviruses and Opportunistic Infections (CROI).

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The primary and secondary endpoints for stability, tolerability, and immunogenicity were all met in the sample. The trial also looked at how effective HTI vaccines were at preventing, delaying, or including viral rebound compared to a placebo group. Participants were given an Analytical Treatment Interruption (ATI) of their Antiretroviral Therapy (ART) for up to 24 weeks for this study. Plasma viral load was tracked on a weekly basis during this period. The results indicate that the treated category has a larger percentage of participants who are not on ART. This signal is highest in participants who do not have a desirable genetic history—for example, those who do not have unique HLA class I alleles related to spontaneous HIV control. Eight (40 percent) of the vaccinated patients could stay off ART therapy for more than 22 weeks, compared to only one (8 percent) in the placebo group.

Strong data supporting a breakthrough development in the field

"These encouraging efficacy data demonstrate that the HTI vaccine was able to modulate an individual's HIV-specific immune response, in a way that can contribute to a better HIV control in the absence of ongoing antiretroviral therapy," said Dr. Beatriz Mothe, Principal Investigator of the study at Fundaci Lluita contra la Sida i les Malalties Infeccioses (FLS) and associate researcher at the IrsiCaixa AIDS Research Institute, located at the Hospital Germans Trias i Pujol, in Badalona, Spain.

"We wish to express our deep gratitude to all the participants for their altruism and commitment to this long clinical trial, as well as to our colleagues at FLS, IrsiCaixa and BCN-Checkpoint, especially for their courage in finalizing the study under the stressful conditions of the COVID-19 pandemic," said Dr. Jos Molt, sub-investigator at FLS. "Their collective effort has been rewarded with this important milestone towards improving health care for people living with HIV."

The AELIX-002 study was a randomized, single-center, double-blind, and placebo-controlled trial to evaluate the safety, tolerability, immunogenicity, and antiviral effect of AELIXs therapeutic HIV vaccine - after discontinuation of ART in 45 early-treated people living with HIV (PLWH). The vaccine used in the study included a DNA vector (DNA.HTI - D), a Modified Vaccinia Ankara virus vector (MVA.HTI - M), and an adenoviral vaccine vector (ChAdOx1.HTI - C). Participants were randomized (2:1) to receive heterologous prime-boost vaccination regimens consisting of DDDMM followed by CCM or matched placebo (P), followed by a 24-week ATI.

Key results of the AELIX-002 Phase I/IIa trial include:

• A total of 45 participants received DDDMM (n=30) or PPPPP (n=15). Of the 45 participants, 41 further completed the CCM (n=26) or PPP (n=15) regimen and entered the ATI

• Immunizations were well tolerated, with neither unexpected nor related serious adverse events (SAEs), and were immunogenic in 97% of vaccine recipients (defined by a >2-fold increase in HTI-specific T cell responses compared to baseline)

• For participants without beneficial HLA class I alleles (32 of the 41); 8 of the 20 vaccinees (40%) versus 1 (8%) of the 12 placebo recipients were able to remain off ART for 22 weeks (ˆ† 32%, 80% CI [7.6; 55.7]); with pVL <2,000 copies/mL being observed in 5 vaccines and one placebo recipient, respectively

• The magnitude of HTI-specific responses at the time of ATI start correlated with time off ART in vaccinees (Rho 0.65, p<0.01)

"The HTI vaccine is aimed at refocusing the immune response to especially vulnerable sites in HIV. The vaccine contains antigenic regions of HIV such as the ones that are more commonly targeted by individuals who naturally control the virus," said Dr. Christian Brander, Chief Scientific Officer, AELIX. "Maintenance of viral remission without antiretroviral therapy represents the next frontier in HIV infection treatment."

"The AELIX-002 data are super exciting; this is the first time we have seen this impact on viral replication. I think the study has convincingly shown that the HTI vaccines can generate immune control; it is clear that they should be considered as a backbone for future HIV cure eradication trials," said Prof Sharon Lewin, director of the Peter Doherty Institute for Infection and Immunity (Melbourne, Australia) and professor of medicine at the University of Melbourne.

Results Support AELIXs Plans for Larger Combination Trials and Financing Round

"With these promising data, AELIXs first-in-class HTI lead candidate could be a game-changer in HIV cure strategies," said Dr. Jos Luis Cabero, CEO, AELIX. "Developing a cure for HIV is a global priority. An intervention that can immunologically control the virus would be of great interest to individuals living with HIV and to the HIV research community. We have shown for the first time that a T-cell vaccine can help control HIV. This supports our vision to position HTI as the backbone for future cure strategies, where it can be combined with other agents. In this sense, AELIX is a front-runner in the race to achieve a functional cure, or long-term viral suppression in the absence of ART, for people living with HIV. We are looking forward to moving ahead with the next financing round and working on larger combination trials."

AELIX and Gilead Sciences collaborated on the AELIX-002 trial as part of a clinical collaboration agreement. The current AELIX-003 clinical trial investigating HTI in conjunction with Gilead's investigational TLR7 agonist vesatolimod (NCT04364035) in people living with HIV on ART is evaluating HTI-based vaccines as a part of combination cure regimens.

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