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Travere Therapeutics(TVTX) - 2024 Q4 - Annual Report
TVTXTravere Therapeutics(TVTX)2025-02-21 01:36
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Drug Approvals and Efficacy - FILSPARI® (sparsentan) received full FDA approval on September 5, 2024, for slowing kidney function decline in adults with primary IgAN, following accelerated approval in February 2023 [493]. - In the PROTECT Study, FILSPARI demonstrated a significant reduction in kidney function decline with a mean eGFR slope of -3.0 mL/min/1.73 m²/year compared to -4.2 mL/min/1.73 m²/year for irbesartan, resulting in a treatment effect of 1.2 mL/min/1.73 m²/year (p=0.0168) [495]. - The FDA-approved label for FILSPARI includes two-year efficacy data showing a 3.8 mL/min/1.73 m² difference in mean change from baseline in eGFR between FILSPARI and irbesartan [495]. - FILSPARI has been granted seven years of Orphan Drug Exclusivity in the U.S. for both the reduction of proteinuria and slowing kidney function decline in adults with primary IgAN [496]. - The estimated prevalence of IgAN in the U.S. is up to 150,000 patients, with more than 70,000 potentially addressable under FILSPARI's full approval indication [498]. - In April 2024, the European Commission granted conditional marketing authorization for FILSPARI for adults with primary IgAN, with a regulatory milestone payment of 17.5millionexpecteduponfullapproval[501].−ThePhase3DUPLEXStudyforsparsentaninFSGSshowedthat42.017.5 million expected upon full approval [501]. - The Phase 3 DUPLEX Study for sparsentan in FSGS showed that 42.0% of patients achieved FSGS partial remission of proteinuria compared to 26.0% for irbesartan (p=0.0094) [507]. - The FDA indicated that the two-year results from the DUPLEX Study alone were insufficient for an sNDA submission, prompting a collaborative effort to define alternative proteinuria-based endpoints for FSGS [508]. - The company plans to submit an sNDA for traditional approval of FILSPARI for FSGS by the end of Q1 2025, based on existing data from the Phase 3 DUPLEX and Phase 2 DUET studies [508]. Financial Performance - Total revenue for the year ended December 31, 2024, was 233.2 million, an increase of 87.9millioncomparedto87.9 million compared to 145.2 million in 2023, primarily driven by a 103.0millionincreaseinFILSPARIsales[549].−FILSPARIsalesreached103.0 million increase in FILSPARI sales [549]. - FILSPARI sales reached 132.2 million in 2024, compared to 29.2millionin2023,reflectingafullyearofsalesfollowingitslaunchinFebruary2023[550].−Totalnetproductsalesincreasedby29.2 million in 2023, reflecting a full year of sales following its launch in February 2023 [550]. - Total net product sales increased by 99.2 million to 226.7millionin2024,whilelicenseandcollaborationrevenuedecreasedby226.7 million in 2024, while license and collaboration revenue decreased by 11.2 million to 6.5million[549].−Operatingexpensestotaled6.5 million [549]. - Operating expenses totaled 557.0 million in 2024, an increase of 23.6millionfrom23.6 million from 533.4 million in 2023, driven by restructuring costs and in-process research and development [552]. - The company recognized a non-recurring charge of 65.2millioninin−processresearchanddevelopmentexpenseinMarch2024relatedtopegtibatinase[562].−ThelossfromdiscontinuedoperationsfortheyearendedDecember31,2024,was65.2 million in in-process research and development expense in March 2024 related to pegtibatinase [562]. - The loss from discontinued operations for the year ended December 31, 2024, was 0.9 million, a significant decrease from a gain of 264.9millionin2023duetothesaleofthebileacidbusiness[567].−Cashusedinoperatingactivitiesfromcontinuingoperationsdecreasedto264.9 million in 2023 due to the sale of the bile acid business [567]. - Cash used in operating activities from continuing operations decreased to 230.0 million in 2024 from 325.4millionin2023,attributedtoa325.4 million in 2023, attributed to a 99.2 million increase in total net product sales [602]. - Cash provided by investing activities was 99.3millionin2024,downfrom99.3 million in 2024, down from 151.6 million in 2023, due to a decrease in net purchases of marketable debt securities [603]. - Cash provided by financing activities was 139.4millionin2024,comparedto139.4 million in 2024, compared to 220.1 million in 2023, influenced by a public offering that generated 134.7millioninnetproceeds[604].CorporateActionsandWorkforceManagement−Thecompanyimplementeda20134.7 million in net proceeds [604]. Corporate Actions and Workforce Management - The company implemented a 20% workforce reduction in December 2023, expected to result in annualized savings of approximately 25.0 million starting in 2024 [525]. - The company implemented a 20% workforce reduction in December 2023 to align resources for the FILSPARI launch and ongoing clinical studies [569]. - As of December 31, 2024, total non-recurring charges incurred due to restructuring amounted to 13.8million[525].−ThecompanycurrentlyhasfourPhase3clinicaltrialsinprocess,withongoingnon−clinicalsupporttrialsthatmayimpactfutureexpenses[558].StrategicPartnershipsandLicensing−AnexclusivelicensingagreementwithRenalysPharmawasestablishedtobringsparsentantopatientsinJapanandotherAsiancountries,withanopenlabelregistrationstudyinitiatedin2024[502].−ThecompanyhasalicenseagreementwithCSLViforthatcouldyieldupto13.8 million [525]. - The company currently has four Phase 3 clinical trials in process, with ongoing non-clinical support trials that may impact future expenses [558]. Strategic Partnerships and Licensing - An exclusive licensing agreement with Renalys Pharma was established to bring sparsentan to patients in Japan and other Asian countries, with an open label registration study initiated in 2024 [502]. - The company has a license agreement with CSL Vifor that could yield up to 845.0 million in milestone payments and royalties for the commercialization of sparsentan [576]. - The company received an upfront cash payment of 210.0millionfromthesaleofitsbileacidproductportfoliotoMirumPharmaceuticals[522].−Thecompanyiseligibletoreceiveupto210.0 million from the sale of its bile acid product portfolio to Mirum Pharmaceuticals [522]. - The company is eligible to receive up to 235.0 million in milestone payments based on specified annual net sales of the bile acid products sold to Mirum [523]. - The company received an upfront cash payment of 210.0millionfromthesaleofitsbileacidbusinessinAugust2023,withpotentialadditionalpaymentsofupto210.0 million from the sale of its bile acid business in August 2023, with potential additional payments of up to 235.0 million based on sales milestones [575]. - The company has a contingent payment obligation of up to 427.0millionrelatedtotheacquisitionofOrphanTechnologiesLimited,basedondevelopmentandregulatorymilestones[590].ResearchandDevelopment−Pegtibatinasedemonstrateda55.1427.0 million related to the acquisition of Orphan Technologies Limited, based on development and regulatory milestones [590]. Research and Development - Pegtibatinase demonstrated a 55.1% mean relative reduction in total homocysteine (tHcy) from baseline in the highest dose cohort of 1.5 mg/kg during the Phase 1/2 COMPOSE Study [512]. - In the sixth cohort of the Phase 1/2 COMPOSE Study, treatment with 2.5 mg/kg of pegtibatinase resulted in a 67.1% mean relative reduction in tHcy from baseline [514]. - The pivotal Phase 3 HARMONY Study was initiated in December 2023 to evaluate the efficacy and safety of pegtibatinase for classical HCU [515]. - A voluntary pause of enrollment in the Phase 3 HARMONY Study was announced in September 2024 to address manufacturing scale-up improvements, with plans to restart enrollment in 2026 [516]. Market and Economic Conditions - The company is exposed to interest rate risk, with a potential 1.7 million impact on investments from a 100 basis point change in interest rates [605]. - Inflation has primarily impacted the company through increased labor costs, but it has not affected the current outlook or business objectives [608].