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Agios Pharmaceuticals(AGIO) - 2023 Q3 - Quarterly Report
AGIOAgios Pharmaceuticals(AGIO)2023-11-01 16:00

Financial Performance - The company reported a net loss of 256.1millionfortheninemonthsendedSeptember30,2023,comparedtoanetlossof256.1 million for the nine months ended September 30, 2023, compared to a net loss of 268.3 million for the same period in 2022[91]. - As of September 30, 2023, the company had an accumulated deficit of 726.7million[91].−NetlossforthethreemonthsendedSeptember30,2023,was726.7 million[91]. - Net loss for the three months ended September 30, 2023, was 91.3 million, compared to a net loss of 81.7millionforthesameperiodin2022,drivenbya81.7 million for the same period in 2022, driven by a 17.5 million up-front payment associated with the agreement with Alnylam[118]. - Total revenue for the nine months ended September 30, 2023, was 19.7million,up19.7 million, up 9.8 million from the same period in 2022, primarily due to increased product revenue from PYRUKYND®[109]. - PYRUKYND® product revenue for Q3 2023 was 7.4million,anincreaseof7.4 million, an increase of 3.9 million compared to Q3 2022, attributed to its FDA approval in February 2022[108]. - Net cash used in operating activities was 223.6millionfortheninemonthsendedSeptember30,2023,comparedto223.6 million for the nine months ended September 30, 2023, compared to 243.3 million for the same period in 2022[124]. - Cash, cash equivalents, and marketable securities balance was 872.4millionatSeptember30,2023,downfrom872.4 million at September 30, 2023, down from 1.1 billion at December 31, 2022[137]. Research and Development - The lead product candidate, PYRUKYND®, received FDA approval on February 17, 2022, for the treatment of hemolytic anemia in adults with PK deficiency[93]. - The company expects to incur significant expenses and net losses as it continues to advance clinical development activities for PYRUKYND® and AG-946[91]. - The company continues to invest in late-stage research programs, including a PAH stabilizer for the treatment of phenylketonuria[83]. - AG-946, a novel PK activator, is in a phase 1 trial with topline results expected by the end of 2023[103]. - The company expects to report topline data from the ENERGIZE trial in the first half of 2024, which evaluates PYRUKYND® for non-transfusion-dependent thalassemia[101]. - The ENERGIZE-T trial has completed enrollment, with topline data expected in the second half of 2024, focusing on transfusion-dependent thalassemia patients[101]. - Total research and development expenses increased by 8.4millionfortheninemonthsendedSeptember30,2023,primarilyduetoan8.4 million for the nine months ended September 30, 2023, primarily due to an 18.4 million increase in direct expenses, partially offset by a 9.9milliondecreaseinindirectexpenses[114].−ResearchanddevelopmentexpensesforQ32023were9.9 million decrease in indirect expenses[114]. - Research and development expenses for Q3 2023 were 81.8 million, an increase of 16.9millioncomparedtoQ32022,drivenbyhigherdirectexpensesrelatedtoPYRUKYND®andin−processresearchanddevelopment[113].CommercializationandSales−Thecompanyanticipatessignificantcommercializationexpensesrelatedtoproductsales,marketing,manufacturing,anddistributionasitcontinuestodevelopandcommercializePYRUKYND®[130].−ThecompanyhasinitiatedaglobalmanagedaccessprogramforPYRUKYND®intheEUandGreatBritain,providingfreeaccesstoeligiblepatients[100].−Thecompanyanticipatesgeneratingfuturerevenuefromproductsales,royalties,andpotentialcollaborationsorlicensingagreements[94].−Thecompanyrecognizedincomeof16.9 million compared to Q3 2022, driven by higher direct expenses related to PYRUKYND® and in-process research and development[113]. Commercialization and Sales - The company anticipates significant commercialization expenses related to product sales, marketing, manufacturing, and distribution as it continues to develop and commercialize PYRUKYND®[130]. - The company has initiated a global managed access program for PYRUKYND® in the EU and Great Britain, providing free access to eligible patients[100]. - The company anticipates generating future revenue from product sales, royalties, and potential collaborations or licensing agreements[94]. - The company recognized income of 127.9 million from the sale of rights to future contingent payments related to its oncology business[86]. - The sale of the oncology business to Servier included a payment of approximately 1.8billionincashatclosing,withadditionalcontingentpaymentsbasedonfutureapprovals[84].AgreementsandMilestones−Thecompanymadeanupfrontpaymentof1.8 billion in cash at closing, with additional contingent payments based on future approvals[84]. Agreements and Milestones - The company made an upfront payment of 17.5 million to Alnylam Pharmaceuticals for the development and commercialization rights of a novel siRNA targeting TMPRSS6[88]. - The company is responsible for up to 130.0millioninpotentialdevelopmentandregulatorymilestonesrelatedtothelicensedproductfromAlnylam[88].−AlicenseagreementwithAlnylamforthedevelopmentofproductstargetingtheTMPRSS6genewasestablishedinJuly2023,withanINDfilingexpectedbytheendof2023[104].−ThecompanyenteredintoalicenseagreementwithAlnylam,whichmayrequireupto130.0 million in potential development and regulatory milestones related to the licensed product from Alnylam[88]. - A license agreement with Alnylam for the development of products targeting the TMPRSS6 gene was established in July 2023, with an IND filing expected by the end of 2023[104]. - The company entered into a license agreement with Alnylam, which may require up to 130.0 million in potential development and regulatory milestones[136]. Cash Flow and Investments - Cash provided by investing activities was 144.1millionfortheninemonthsendedSeptember30,2023,primarilyduetohigherproceedsfrommaturitiesandsalesofmarketablesecurities[126].−Thecompanyexpectstofunditsoperatingexpensesandcapitalexpendituresthroughexistingcash,anticipatedproductrevenue,andpotentialmilestonepaymentsthroughatleast2026[131].−Interestincomeincreasedsignificantlyduetorisinginterestrates,withnetinterestincomeof144.1 million for the nine months ended September 30, 2023, primarily due to higher proceeds from maturities and sales of marketable securities[126]. - The company expects to fund its operating expenses and capital expenditures through existing cash, anticipated product revenue, and potential milestone payments through at least 2026[131]. - Interest income increased significantly due to rising interest rates, with net interest income of 24.7 million for the nine months ended September 30, 2023, compared to 6.3millionin2022[115].−ThecompanysolditsrightstofuturecontingentpaymentsassociatedwithroyaltiesonU.S.netsalesofTIBSOVO®for6.3 million in 2022[115]. - The company sold its rights to future contingent payments associated with royalties on U.S. net sales of TIBSOVO® for 131.8 million in October 2022[121]. Market Risks - The company is exposed to market risk related to changes in foreign currency exchange rates due to contracts with CROs in Asia and Europe[138]. - As of September 30, 2023, and December 31, 2022, liabilities denominated in foreign currencies were immaterial[138].