Enanta Pharmaceuticals Inc 2024年度报告

Annual Report2024 January 2025 To Our Shareholders, At Enanta, we are dedicated to our mission of discoveringand developing groundbreaking small molecule drugs fordiseases with significant unmet need. We are proud to report that 2024 was markedby progress toward accomplishing many ofour goals. We made strides in advancing ourclinical-stagecandidatesforrespiratorysyncytial virus (RSV) as well as leveraging ourprovenexpertise in medicinal chemistry toexpand our discovery efforts in immunology.Both our pipeline and R&D efforts underscoreour commitment to discovering and developingnovel, best-in-class medicines to transform thelives of patients with curative therapies. once-daily single agents or in combination forspecific high-risk populations. Looking forward,we expect to report results from RSVHR, ourongoing Phase 2b study of zelicapavir in adultswith RSV infection who are at high risk ofcomplications, in the third quarter of 2025. In 2024, we expanded our pipeline into anadjacentarea,immunology,where we areconcentrating on diseases with high unmetmedical need, validated targets and a clearclinical development path. We made notableprogressas we focused on designing anddeveloping highly potent and selective, oralsmallmolecule inhibitors for the treatmentofinflammatory diseases.Specifically,inNovember,we announced the selection ofEPS-1421, a novel, potent and selective oralinhibitorof KIT,designed to treat chronicspontaneousurticaria and other mast cell-drivenindications by depleting mast cells,thereby addressing a primary driver of thesediseases. We plan to conduct scale-up activitiesand IND enabling studies for EPS-1421 in 2025. Ourdevelopment programs in virology arefocused on RSV, an area of significant unmetneed with no therapies currently available.We have two clinical-stage candidates for thetreatment of RSV — zelicapavir (EDP-938), anN-protein inhibitor, and EDP-323, an L-proteininhibitor. In December, we announced positivedata from a first-in-pediatrics Phase 2 studyevaluating zelicapavir in both hospitalized andnon-hospitalized children aged 28 days to 36months with RSV. Prior to that, in September,we reported positive data from a Phase 2achallenge study of EDP-323. We believe theresults for EDP-323 are among the strongesteverreported in an RSV challenge study,raising the high bar set by zelicapavir, anddemonstrating the potential for our leadingclinical candidates in RSV to be developed as Wealso introduced a second immunologyprogram aimed at developing an oral treatmentof type 2 immune-driven diseases. Specifically,we are focused on discovering oral STAT6inhibitorsfor atopic dermatitis,and other indications, by blocking the IL-4/IL-13 signalingpathway, thereby addressing a primary driverof these diseases. Enanta’s prototype inhibitorsin development demonstrate potent activityandhigh selectivity for STAT6 over otherSTATs in both biochemical and cellular assays.We continue to evaluate multiple compoundsinpreclinical studies and expect to selecta development candidate in the second halfof 2025. programs in a capital efficient manner and willalso evaluate partnering opportunities. We lookforward to reporting on the next milestonesin 2025, including announcing data from ourRSVHR trial, further progressing our KIT andSTAT6 programs, and continuing to expand ourimmunology portfolio. Inclosing,I would like to recognize thepatientsand caregivers involved in ourongoing and completed studies. I would alsolike to acknowledge our employees for theirdedication and hard work in the past year.And again, I’d especially like to thank you, ourshareholders. We owe you our sincere gratitudefor the opportunity to execute our missionof developing transformative medicines andappreciate your support to do so. Operationally and financially, we ended 2024in a solid position. As of September 30, 2024,Enantahad$248 million in cash,cashequivalentsand marketable securities,inaddition to a pending net operating loss refundof approximately $30 million from the InternalRevenue Service. This cash position is enhancedby the ongoing portion of royalty revenuefrom AbbVie’s sales of MAVYRET®/MAVIRET®,whichremains a leading treatment forhepatitisC virus.In 2023,we monetized54.5% of our royalties, and as such we stillreceive45.5%of all royalties.Throughprudentcash management,combined withour ongoing royalties, we are able to supportourprograms into fiscal year 2027.Thisprovides us with a strong foundation as weexecuteour clinical and corporate goals,working to deliver improved value to all ourstakeholders — patients, caregivers, physicians,and shareholders. Sincerely, Jay R. Luly, Ph.D.President and Chief Executive Officer As we turn the corner on 2024 and look aheadto 2025, I believe we are in a strong positionon multiple fronts — financially, scientifically,clinically and operationally. We enter the newyear with a continued focus on advancing our UNITED STATESSECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 Fo