Gilead Submits New Drug Application to U.S. Food and Drug Administration for Lenacapavir, an Investigational, Long-Acting Capsid Inhibitor for the Treatment of HIV-1 in People With Limited Therapy Options

If Approved, Lenacapavir Would be the First Capsid Inhibitor and the Only HIV-1 Treatment Option Administered Every 6 Months

FOSTER CITY, Calif.–(BUSINESS WIRE)–Gilead Sciences, Inc. (Nasdaq: GILD) announced today that the company completed submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) seeking approval of lenacapavir, an investigational, long-acting HIV-1 capsid inhibitor, for the treatment of HIV-1 infection in heavily treatment-experienced (HTE) people with multi-drug resistant (MDR) HIV-1 infection.

The submission is supported by data from the Phase 2/3 CAPELLA trial, which evaluated the safety and efficacy of lenacapavir administered subcutaneously every six months in combination with an optimized antiretroviral background regimen. Key data on lenacapavir will be presented during the 11th International AIDS Society (IAS) Conference on HIV Science in July 2021.

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, which is being studied as an every-six-month subcutaneous injection, is a potential first-in-class capsid inhibitor for the treatment of HIV-1 infection without overlapping resistance with any currently approved antiretroviral therapy (ART).

Lenacapavir is an important breakthrough innovation with the potential to be transformative for people living with multi-drug resistant HIV who have very limited treatment options,” said Merdad Parsey, MD, PhD, Chief Medical Officer, Gilead Sciences. “The filing moves us one step closer to providing an innovative treatment option that helps to address barriers to achieving viral suppression and meet the unmet needs of people living with multi-drug resistant HIV.”

Lenacapavir is being developed in combination with other antiretroviral agents for the treatment of HIV-1 infection in adults and pediatric patients weighing at least 35 kg with MDR HIV-1 infection who are currently on a failing antiretroviral treatment regimen due to resistance, intolerance or safety considerations. Lenacapavir is designed to inhibit HIV-1 replication by interfering with multiple, essential steps of the viral lifecycle, including capsid-mediated uptake of HIV-1 proviral DNA, virus assembly and release, and capsid core formation.

Gilead plans to submit marketing authorization applications for lenacapavir to the European Medicines Agency and other global agencies in the coming months.

Lenacapavir is an investigational compound and is not approved by any regulatory authority for any use and 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 multicenter study designed to evaluate the antiviral activity of Gilead’s investigational, long-acting HIV-1 capsid inhibitor lenacapavir administered every 6 months as a subcutaneous injection in HTE people with MDR 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 for 14 days, in addition to continuing their failing regimen (functional monotherapy). An additional 36 participants were enrolled in a separate treatment cohort. The primary endpoint was the proportion of participants randomly allocated to receive oral 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.

The study achieved its primary endpoint by demonstrating that a significantly higher proportion of participants randomly allocated to receive lenacapavir achieved a clinically meaningful viral load reduction of at least 0.5 log10 copies/mL from baseline compared with those receiving placebo during the 14-day functional monotherapy period (88% vs. 17%, p<0.0001). These data were previously presented at the virtual 28th Conference on Retroviruses and Opportunistic Infections (virtual CROI 2021). Those who received lenacapavir (n=24) achieved statistically significantly greater mean decrease in viral load than those who received placebo (n=12) during the functional monotherapy period (-1.93 log10 copies/mL vs. -0.29 log10 copies/mL, p<0.0001). Lenacapavir was generally well-tolerated, with no serious adverse events related to study drug observed 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.

Following the 14-day functional monotherapy period, participants who were randomly allocated to receive lenacapavir or placebo, in addition to continuing their failing regimen, started open-label lenacapavir and an optimized background regimen, while those enrolled in a separate treatment cohort received open-label lenacapavir and an optimized background regimen on Day 1. 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. The trial data for the first six-month period (Week 26) have been submitted to the FDA as part of the NDA filing, and will be presented at an upcoming conference.

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 and prevention of HIV-1 infection. 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 or at risk for 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.

The safety, efficacy and dosing of lenacapavir are being evaluated in multiple ongoing clinical studies. Data presented at AIDS 2020 from a completed Phase 1 study support further evaluation of lenacapavir administered subcutaneously every six-month for both HIV-1 treatment and prevention. During IDWeek 2020, the company announced plans to evaluate the use of lenacapavir as an injectable PrEP option administered every six months among cisgender women, men who have sex with men and persons of trans experience. The prevention trials have projected initiation dates in 2021.

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 eleven HIV medications, including the first single tablet regimen to treat HIV and the first once-daily oral antiretroviral tablet 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 within currently anticipated timelines or at all, including those involving lenacapavir; the possibility of unfavorable results from ongoing or additional clinical trials or studies, including those involving lenacapavir; Gilead’s ability to receive regulatory approvals in a timely manner or at all, including FDA, European Medicines Agency or other regulatory approval of lenacapavir, 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 March 31, 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.

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