European Medicines Agency Validates Gilead’s Marketing Authorization Application for Lenacapavir, an Investigational, Long-Acting Capsid Inhibitor for the Treatment of HIV-1 in People With Limited Therapy Options

FOSTER CITY, Calif.--(BUSINESS WIRE)-- Gilead Sciences, Inc., (Nasdaq: GILD) today announced that the company’s Marketing Authorization Application (MAA) for lenacapavir, an investigational, long-acting HIV-1 capsid inhibitor, has been fully validated and is now under evaluation with the European Medicines Agency (EMA). The proposed indication is for the treatment of HIV-1 infection, in combination with other antiretroviral(s), in adults with multidrug resistant HIV-1 infection who are currently on a failing antiretroviral treatment regimen due to resistance, intolerance or safety considerations.

The application will be reviewed by the EMA’s Committee for Medicinal Products for Human Use (CHMP) under the centralized licensing procedure for all 27 Member States of the European Union, as well as Norway, Iceland and Liechtenstein.

“Lenacapavir is a breakthrough innovation that has the potential to be transformative for people with multi-drug resistant HIV, who have very limited treatment options,” said Merdad Parsey, MD, PhD, Chief Medical Officer, Gilead Sciences. “The MAA validation is a step forward in our commitment to a patient-centered approach to helping all individuals living with HIV achieve and sustain viral load suppression, irrespective of their prior treatment history.”

The MAA is supported by data from the ongoing CAPELLA study, which achieved its primary endpoint by demonstrating that a significantly higher proportion of participants randomly allocated to receive oral lenacapavir in combination with an optimized background regimen (n=24) achieved a clinically meaningful viral load reduction of at least 0.5 log10 copies/mL from baseline compared with those randomly allocated to receive placebo (n=12) during the 14-day functional monotherapy period (88% vs. 17%, p<0.0001). Lenacapavir was generally well-tolerated, with no serious adverse events related to study drug and no study drug discontinuations through the 14-day period, including no discontinuations due to adverse events. The most common adverse events observed were injection site reactions. These data were previously presented at the virtual 28th Conference on Retroviruses and Opportunistic Infections (virtual CROI 2021). 26-week data from the CAPELLA study were presented at the 11th International AIDS Society (IAS) Conference on HIV Science. Additional data from the CAPELLA study will be presented at a future scientific conference.

In June 2021, Gilead submitted a New Drug Application (NDA) for lenacapavir seeking U.S. Food & Drug Administration (FDA) approval for the treatment of HIV-1 infection in heavily treatment-experienced people with multi-drug resistant HIV-1 infection in combination with other antiretrovirals. In May 2019, the FDA granted Breakthrough Therapy Designation for the development of lenacapavir for the treatment of HIV-1 infection in heavily treatment-experienced patients with multi-drug resistance in combination with other antiretroviral drugs.

Lenacapavir is an investigational compound and is not approved by any regulatory authority for any use; its safety and efficacy are not established. There is no cure for HIV or AIDS.

About CAPELLA (NCT04150068)

CAPELLA is a Phase 2/3, double-blinded, placebo-controlled global multi-center study designed to evaluate the antiviral activity of Gilead’s investigational, long-acting HIV-1 capsid inhibitor lenacapavir administered every six months as a subcutaneous injection in heavily treatment-experienced people with multi-drug resistant HIV-1 infection. CAPELLA includes men and women living with HIV-1 and is being conducted at research centers in North America, Europe and Asia.

In CAPELLA, 36 participants with multi-class HIV-1 drug resistance and a detectable viral load while on a failing regimen were randomly allocated to receive oral lenacapavir or placebo in a 2:1 ratio for 14 days, in addition to continuing their failing regimen (functional monotherapy). An additional 36 participants were enrolled in a separate treatment cohort, starting oral lenacapavir on Day 1. Both cohorts are part of the ongoing maintenance period of the study evaluating the safety and efficacy of subcutaneous lenacapavir administered every six months in combination with an optimized background regimen. The primary endpoint was the proportion of participants randomly allocated to receive lenacapavir or placebo for 14 days, in addition to continuing their failing regimen, achieving ≥0.5 log₁₀ copies/mL reduction from baseline in HIV-1 RNA at the end of the functional monotherapy period. Following 14 days of lenacapavir oral therapy, all participants started open-label lenacapavir, administered as a subcutaneous injection every six months, in combination with an optimized background regimen. This ongoing maintenance period of the study is evaluating the additional trial endpoints of safety and efficacy of subcutaneous lenacapavir administered every six months in combination with an optimized background regimen.

For further information, please see https://clinicaltrials.gov/ct2/show/NCT04150068.

About Lenacapavir

Lenacapavir is a potential first-in-class, long-acting HIV-1 capsid inhibitor in development for the treatment of HIV-1 infection. The safety and efficacy of lenacapavir are being evaluated in multiple ongoing clinical studies. Lenacapavir’s multi-stage mechanism of action is distinguishable from currently approved classes of antiviral agents and is designed to provide a new avenue for the development of long-acting therapy options for people living with HIV-1. While most antivirals act on just one stage of viral replication, lenacapavir is designed to inhibit HIV-1 at multiple stages of its lifecycle and has no known cross resistance to other existing drug classes.

About Gilead Sciences

Gilead Sciences, Inc. is a biopharmaceutical company that has pursued and achieved breakthroughs in medicine for more than three decades, with the goal of creating a healthier world for all people. The company is committed to advancing innovative medicines to prevent and treat life-threatening diseases, including HIV, viral hepatitis and cancer.

For more than 30 years, Gilead has been a leading innovator in the field of HIV, driving advances in treatment, prevention and cure research. Gilead researchers have developed 11 HIV medications, including the first single tablet regimen to treat HIV and the first antiretroviral for pre-exposure prophylaxis (PrEP) to reduce the risk of acquiring HIV infection. These advances in medical research have helped to transform HIV into a preventable, chronic condition for millions of people.

Gilead is committed to continued scientific innovation to provide solutions for the evolving needs of people affected by HIV around the world. Through partnerships and collaborations, the company also aims to improve education, expand access and address barriers to care, with the goal of ending the HIV epidemic for everyone, everywhere.

Gilead operates in more than 35 countries worldwide, with headquarters in Foster City, California.

Forward-Looking Statements

This press release includes forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 that are subject to risks, uncertainties and other factors, including Gilead’s ability to initiate, progress or complete clinical trials or studies involving lenacapavir within currently anticipated timelines or at all; the possibility of unfavorable results from ongoing or additional clinical trials or studies involving lenacapavir; Gilead’s ability to receive regulatory approvals in a timely manner or at all, including EMA approval of lenacapavir for the treatment of HIV-1 infection, in combination with other antiretroviral(s), in adults with multidrug resistant HIV-1 infection who are currently on a failing antiretroviral treatment regimen due to resistance, intolerance or safety considerations, or FDA approval of lenacapavir for the treatment of HIV-1 infection in heavily treatment-experienced people with multi-drug resistant HIV-1 infection, and the risk that any such approvals may be subject to significant limitations on use; the possibility that Gilead may make a strategic decision to discontinue development of lenacapavir and that, as a result, lenacapavir may never be successfully commercialized; and any assumptions underlying any of the foregoing. These and other risks, uncertainties and factors are described in detail in Gilead’s Quarterly Report on Form 10-Q for the quarter ended June 30, 2021, as filed with the U.S. Securities and Exchange Commission. These risks, uncertainties and other factors could cause actual results to differ materially from those referred to in the forward-looking statements. All statements other than statements of historical fact are statements that could be deemed forward-looking statements. The reader is cautioned that any such forward-looking statements are not guarantees of future performance and is cautioned not to place undue reliance on these forward-looking statements. All forward-looking statements are based on information currently available to Gilead, and Gilead assumes no obligation and disclaims any intent to update any such forward-looking statements.

GILEAD and the GILEAD logo are trademarks of Gilead Sciences, Inc. All other trademarks are the property of their respective owners.

For more information about Gilead, please visit the company’s website at www.gilead.com, follow Gilead on Twitter (@Gilead Sciences) or call Gilead Public Affairs at 1-800-GILEAD-5 or 1-650-574-3000.

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