{"companies":[{"description":"Develops RNA interference medicines. This example follows vutrisiran in ATTR cardiomyopathy.","id":"alnylam","milestones":[{"asset":"Vutrisiran","id":"alnylam-helios-b-topline","kind":"readout","label":"HELIOS-B Phase 3 topline results","nextQuestion":"How did the revised analysis plan differ from the earlier plan, and what did the full results establish?","records":[{"correctionReason":null,"effectiveAt":null,"firstSeenAt":"2026-09-26T07:20:44Z","id":"alny-helios-b-20231102","ingestedAt":"2026-09-26T07:20:44Z","publishedAt":"2023-11-02","sourceTitle":"Q3 2023 results: HELIOS-B timing","sourceUrl":"https://investors.alnylam.com/press-release?id=27781","summary":"Alnylam expected the HELIOS-B Phase 3 topline results in early 2024. The company did not give an exact calendar deadline in this statement.","supersedes":null,"targetEnd":null,"targetPrecision":"unspecified","targetStart":null,"targetText":"Early 2024","type":"guidance"},{"correctionReason":null,"effectiveAt":null,"firstSeenAt":"2026-09-26T07:20:44Z","id":"alny-helios-b-20240215","ingestedAt":"2026-09-26T07:20:44Z","publishedAt":"2024-02-15","sourceTitle":"2023 year-end results: revised HELIOS-B plan","sourceUrl":"https://investors.alnylam.com/press-release?id=27941","summary":"Alnylam described changes to the statistical analysis plan, endpoint structure and study exposure, and expected topline results in late June or early July 2024. This records a changed timing statement, without assigning an invented day to either phrase.","supersedes":"alny-helios-b-20231102","targetEnd":null,"targetPrecision":"unspecified","targetStart":null,"targetText":"Late June or early July 2024","type":"update"},{"correctionReason":null,"effectiveAt":"2024-06-24","firstSeenAt":"2026-09-26T07:20:44Z","id":"alny-helios-b-20240624","ingestedAt":"2026-09-26T07:20:44Z","publishedAt":"2024-06-24","sourceTitle":"HELIOS-B topline results announcement","sourceUrl":"https://investors.alnylam.com/press-release?id=28231","summary":"Alnylam published HELIOS-B topline results and reported that the study met its primary and secondary endpoints. This is evidence that a readout was announced; it is not an independent review of every efficacy or safety claim.","supersedes":null,"targetEnd":null,"targetPrecision":"unspecified","targetStart":null,"targetText":null,"type":"outcome"}],"trialIds":["NCT04153149"]}],"name":"Alnylam Pharmaceuticals","ticker":"ALNY"},{"description":"Develops medicines for genetic and rare diseases. This example follows the US ROCTAVIAN review.","id":"biomarin","milestones":[{"asset":"Valoctocogene roxaparvovec (ROCTAVIAN)","id":"biomarin-roctavian-us-review","kind":"regulatory","label":"ROCTAVIAN US FDA review","nextQuestion":"What did the FDA review extension cover, and which patients are included in the resulting approval?","records":[{"correctionReason":null,"effectiveAt":null,"firstSeenAt":"2026-09-26T07:20:44Z","id":"bmrn-roctavian-20230227","ingestedAt":"2026-09-26T07:20:44Z","publishedAt":"2023-02-27","sourceTitle":"2022 year-end results: ROCTAVIAN review date","sourceUrl":"https://investors.biomarin.com/news/news-details/2023/BioMarin-Announces-Record-Fourth-Quarter-and-Full-Year-2022-Total-Revenues-Driven-by-Strong-Global-Demand-for-VOXZOGO-and-Steady-Growth-of-Enzyme-Business-02-27-2023/default.aspx","summary":"BioMarin reported a March 31, 2023 FDA target action date and explicitly said a three-month extension was possible if FDA considered it necessary. The date was a review target, not a promise of approval.","supersedes":null,"targetEnd":"2023-03-31","targetPrecision":"exact","targetStart":"2023-03-31","targetText":"March 31, 2023; potential three-month extension disclosed","type":"guidance"},{"correctionReason":null,"effectiveAt":null,"firstSeenAt":"2026-09-26T07:20:44Z","id":"bmrn-roctavian-20230426","ingestedAt":"2026-09-26T07:20:44Z","publishedAt":"2023-04-26","sourceTitle":"Q1 2023 results: FDA extended the review","sourceUrl":"https://investors.biomarin.com/news/news-details/2023/BioMarin-Announces-Record-Breaking-First-Quarter-2023-Results-Including-15-Year-over-year-Growth-of-Total-Revenues-04-26-2023/default.aspx","summary":"BioMarin reported that FDA had extended the review in March and set June 30, 2023 as the new target. The company said the requested three-year study analysis constituted a major amendment. This source does not identify the exact day in March when the change occurred.","supersedes":"bmrn-roctavian-20230227","targetEnd":"2023-06-30","targetPrecision":"exact","targetStart":"2023-06-30","targetText":"2023-06-30","type":"update"},{"correctionReason":null,"effectiveAt":"2023-06-29","firstSeenAt":"2026-09-26T07:20:44Z","id":"bmrn-roctavian-20230629","ingestedAt":"2026-09-26T07:20:44Z","publishedAt":"2023-06-29","sourceTitle":"ROCTAVIAN US approval announcement","sourceUrl":"https://investors.biomarin.com/news/news-details/2023/U.S.-Food-and-Drug-Administration-Approves-BioMarins-ROCTAVIAN-valoctocogene-roxaparvovec-rvox-the-First-and-Only-Gene-Therapy-for-Adults-with-Severe-Hemophilia-A-06-29-2023/default.aspx","summary":"BioMarin announced FDA approval of ROCTAVIAN for eligible adults with severe hemophilia A on June 29, 2023. The announcement contains eligibility and safety information; approval should not be read as suitable treatment for every patient.","supersedes":null,"targetEnd":null,"targetPrecision":"unspecified","targetStart":null,"targetText":null,"type":"outcome"}],"trialIds":[]}],"name":"BioMarin Pharmaceutical","ticker":"BMRN"},{"description":"Develops medicines for neurological diseases. This example follows tofersen for SOD1-associated ALS.","id":"biogen","milestones":[{"asset":"Tofersen (QALSODY)","id":"biogen-tofersen-us-review","kind":"regulatory","label":"Tofersen US FDA review","nextQuestion":"What was the basis of accelerated approval, and what clinical benefit still required confirmation?","records":[{"correctionReason":null,"effectiveAt":null,"firstSeenAt":"2026-09-26T07:20:44Z","id":"biib-tofersen-20220726","ingestedAt":"2026-09-26T07:20:44Z","publishedAt":"2022-07-26","sourceTitle":"Tofersen application accepted for FDA review","sourceUrl":"https://investors.biogen.com/news-releases/news-release-details/fda-accepts-biogens-new-drug-application-and-grants-priority","summary":"Biogen reported FDA acceptance and priority review of the tofersen application, with a target action date of January 25, 2023. Acceptance for review was not approval.","supersedes":null,"targetEnd":"2023-01-25","targetPrecision":"exact","targetStart":"2023-01-25","targetText":"2023-01-25","type":"guidance"},{"correctionReason":null,"effectiveAt":"2022-10-17","firstSeenAt":"2026-09-26T07:20:44Z","id":"biib-tofersen-20221017","ingestedAt":"2026-09-26T07:20:44Z","publishedAt":"2022-10-17","sourceTitle":"Tofersen FDA review extended by three months","sourceUrl":"https://investors.biogen.com/news-releases/news-release-details/biogen-announces-fdas-3-month-extension-review-period-new-drug","summary":"Biogen announced a new FDA target of April 25, 2023. It said responses to FDA information requests were considered a major amendment requiring additional review time.","supersedes":"biib-tofersen-20220726","targetEnd":"2023-04-25","targetPrecision":"exact","targetStart":"2023-04-25","targetText":"2023-04-25","type":"update"},{"correctionReason":null,"effectiveAt":"2023-04-25","firstSeenAt":"2026-09-26T07:20:44Z","id":"biib-tofersen-20230425","ingestedAt":"2026-09-26T07:20:44Z","publishedAt":"2023-04-25","sourceTitle":"Partner Ionis: QALSODY approval announcement","sourceUrl":"https://ir.ionis.com/news-releases/news-release-details/fda-approves-qalsodytm-tofersen-first-treatment-targeting","summary":"Ionis announced that its partner Biogen received accelerated FDA approval for QALSODY in adults with SOD1-associated ALS. The approval used a biomarker endpoint; continued approval could depend on confirmation of clinical benefit.","supersedes":null,"targetEnd":null,"targetPrecision":"unspecified","targetStart":null,"targetText":null,"type":"outcome"}],"trialIds":[]}],"name":"Biogen","ticker":"BIIB"},{"description":"Develops pemvidutide for metabolic diseases. This example follows the Phase 2 obesity readout.","id":"altimmune","milestones":[{"asset":"Pemvidutide","id":"altimmune-momentum-week48","kind":"readout","label":"MOMENTUM Phase 2 48-week results","nextQuestion":"What do the complete 48-week results show about weight change, discontinuations and safety?","records":[{"correctionReason":null,"effectiveAt":null,"firstSeenAt":"2026-09-26T07:20:44Z","id":"alt-momentum-20230511","ingestedAt":"2026-09-26T07:20:44Z","publishedAt":"2023-05-11","sourceTitle":"Q1 2023 update: MOMENTUM timing","sourceUrl":"https://ir.altimmune.com/news-releases/news-release-details/altimmune-announces-first-quarter-2023-financial-results-and","summary":"Altimmune expected the 48-week topline results of the MOMENTUM Phase 2 obesity trial in the fourth quarter of 2023.","supersedes":null,"targetEnd":"2023-12-31","targetPrecision":"quarter","targetStart":"2023-10-01","targetText":"Q4 2023","type":"guidance"},{"correctionReason":null,"effectiveAt":"2023-11-30","firstSeenAt":"2026-09-26T07:20:44Z","id":"alt-momentum-20231130","ingestedAt":"2026-09-26T07:20:44Z","publishedAt":"2023-11-30","sourceTitle":"MOMENTUM 48-week topline announcement","sourceUrl":"https://ir.altimmune.com/news-releases/news-release-details/altimmune-announces-positive-topline-results-momentum-48-week","summary":"Altimmune announced the 48-week MOMENTUM topline results on November 30, 2023. This records publication of the readout, not an ORION efficacy comparison or investment recommendation.","supersedes":null,"targetEnd":null,"targetPrecision":"unspecified","targetStart":null,"targetText":null,"type":"outcome"}],"trialIds":[]}],"name":"Altimmune","ticker":"ALT"},{"description":"Develops medicines using base editing. This example follows BEAM-101 in sickle cell disease.","id":"beam","milestones":[{"asset":"BEAM-101","id":"beam-beacon-initial-data","kind":"readout","label":"BEACON Phase 1/2 initial clinical data","nextQuestion":"How do the abstract and later presentation differ in patient count, follow-up and safety findings?","records":[{"correctionReason":null,"effectiveAt":null,"firstSeenAt":"2026-09-26T07:20:44Z","id":"beam-beacon-20240108","ingestedAt":"2026-09-26T07:20:44Z","publishedAt":"2024-01-08","sourceTitle":"2024 milestones: BEAM-101 first clinical data","sourceUrl":"https://investors.beamtx.com/news-releases/news-release-details/beam-therapeutics-highlights-progress-across-base-editing","summary":"Beam expected initial data from multiple BEACON participants in the second half of 2024.","supersedes":null,"targetEnd":"2024-12-31","targetPrecision":"half","targetStart":"2024-07-01","targetText":"Second half of 2024","type":"guidance"},{"correctionReason":null,"effectiveAt":"2024-11-05","firstSeenAt":"2026-09-26T07:20:44Z","id":"beam-beacon-20241105","ingestedAt":"2026-09-26T07:20:44Z","publishedAt":"2024-11-05","sourceTitle":"ASH abstracts: first BEAM-101 clinical data","sourceUrl":"https://investors.beamtx.com/news-releases/news-release-details/beam-therapeutics-present-data-across-hematology-franchise","summary":"Beam released initial BEACON findings with accepted ASH abstracts. The preliminary data used a July 2 cutoff. The release also described a patient death judged likely related to conditioning and unrelated to BEAM-101; readers should review the full safety context.","supersedes":null,"targetEnd":null,"targetPrecision":"unspecified","targetStart":null,"targetText":null,"type":"outcome"},{"correctionReason":null,"effectiveAt":"2024-12-07","firstSeenAt":"2026-09-26T07:20:44Z","id":"beam-beacon-20241207","ingestedAt":"2026-09-26T07:20:44Z","publishedAt":"2024-12-07","sourceTitle":"ASH announcement: updated BEACON data","sourceUrl":"https://investors.beamtx.com/news-releases/news-release-details/beam-therapeutics-announces-new-data-beacon-phase-12-clinical","summary":"Beam announced updated BEACON data using an October 28 cutoff, including seven treated participants in the analysis. This was an additional disclosure after the November abstract, not a second initial-data promise.","supersedes":null,"targetEnd":null,"targetPrecision":"unspecified","targetStart":null,"targetText":null,"type":"outcome"}],"trialIds":[]}],"name":"Beam Therapeutics","ticker":"BEAM"},{"description":"Develops gene-based medicines. This example follows the partnered exa-cel sickle cell disease application.","id":"crispr","milestones":[{"asset":"Exagamglogene autotemcel (exa-cel / CASGEVY)","id":"crispr-exacel-scd-us-review","kind":"regulatory","label":"Exa-cel US review for sickle cell disease","nextQuestion":"Which indication and age group were approved, and how does that differ from the separate thalassemia application?","records":[{"correctionReason":null,"effectiveAt":null,"firstSeenAt":"2026-09-26T07:20:44Z","id":"crsp-exacel-20230608","ingestedAt":"2026-09-26T07:20:44Z","publishedAt":"2023-06-08","sourceTitle":"Exa-cel FDA application acceptance","sourceUrl":"https://ir.crisprtx.com/news-releases/news-release-details/fda-accepts-biologics-license-applications-exagamglogene","summary":"CRISPR and Vertex reported a December 8, 2023 FDA target date for the sickle cell disease application. The separate thalassemia application had a different target and is not combined into this milestone.","supersedes":null,"targetEnd":"2023-12-08","targetPrecision":"exact","targetStart":"2023-12-08","targetText":"2023-12-08","type":"guidance"},{"correctionReason":null,"effectiveAt":"2023-12-08","firstSeenAt":"2026-09-26T07:20:44Z","id":"crsp-exacel-20231208","ingestedAt":"2026-09-26T07:20:44Z","publishedAt":"2023-12-08","sourceTitle":"Vertex and CRISPR: CASGEVY US SCD approval","sourceUrl":"https://news.vrtx.com/news-releases/news-release-details/vertex-and-crispr-therapeutics-announce-us-fda-approval","summary":"Vertex and CRISPR announced FDA approval of CASGEVY for sickle cell disease in patients aged 12 years and older with recurrent vaso-occlusive crises.","supersedes":null,"targetEnd":null,"targetPrecision":"unspecified","targetStart":null,"targetText":null,"type":"outcome"}],"trialIds":[]}],"name":"CRISPR Therapeutics","ticker":"CRSP"},{"description":"Develops cancer medicines. This example follows a cabozantinib label expansion for neuroendocrine tumors.","id":"exelixis","milestones":[{"asset":"Cabozantinib (CABOMETYX)","id":"exelixis-cabozantinib-net-us-review","kind":"regulatory","label":"CABOMETYX US review for advanced NET","nextQuestion":"Which previously treated neuroendocrine tumor populations were included in the final label?","records":[{"correctionReason":null,"effectiveAt":null,"firstSeenAt":"2026-09-26T07:20:44Z","id":"exel-net-20250112","ingestedAt":"2026-09-26T07:20:44Z","publishedAt":"2025-01-12","sourceTitle":"2025 milestones: CABOMETYX NET review date","sourceUrl":"https://ir.exelixis.com/node/26161","summary":"Exelixis reported FDA review of the supplemental application for previously treated advanced neuroendocrine tumors, with an April 3, 2025 target action date.","supersedes":null,"targetEnd":"2025-04-03","targetPrecision":"exact","targetStart":"2025-04-03","targetText":"2025-04-03","type":"guidance"},{"correctionReason":null,"effectiveAt":"2025-03-26","firstSeenAt":"2026-09-26T07:20:44Z","id":"exel-net-20250326","ingestedAt":"2026-09-26T07:20:44Z","publishedAt":"2025-03-26","sourceTitle":"CABOMETYX advanced NET approval announcement","sourceUrl":"https://ir.exelixis.com/news-releases/news-release-details/exelixis-announces-us-fda-approval-cabometyxr-cabozantinib-4","summary":"Exelixis announced FDA approval of CABOMETYX for specified previously treated advanced pancreatic and extra-pancreatic neuroendocrine tumors. The approval announcement preceded the published target date.","supersedes":null,"targetEnd":null,"targetPrecision":"unspecified","targetStart":null,"targetText":null,"type":"outcome"}],"trialIds":[]}],"name":"Exelixis","ticker":"EXEL"},{"description":"Develops medicines including antiviral and oncology therapies. This example follows lenacapavir for HIV prevention.","id":"gilead","milestones":[{"asset":"Lenacapavir","id":"gilead-purpose1-update","kind":"readout","label":"PURPOSE 1 Phase 3 update","nextQuestion":"Was this an interim analysis or final readout, and which population and comparator were studied?","records":[{"correctionReason":null,"effectiveAt":null,"firstSeenAt":"2026-09-26T07:20:44Z","id":"gild-purpose1-20240206","ingestedAt":"2026-09-26T07:20:44Z","publishedAt":"2024-02-06","sourceTitle":"Q4 2023 prepared remarks: PURPOSE 1 timing","sourceUrl":"https://investors.gilead.com/files/doc_financials/2023/q4/GILD-Q423-Summary-of-Prepared-Remarks-6-February-2024.pdf","summary":"Gilead expected a Phase 3 PURPOSE 1 update in the second half of 2024. The presentation described timing estimates, not a guaranteed efficacy outcome.","supersedes":null,"targetEnd":"2024-12-31","targetPrecision":"half","targetStart":"2024-07-01","targetText":"Second half of 2024","type":"guidance"},{"correctionReason":null,"effectiveAt":"2024-06-20","firstSeenAt":"2026-09-26T07:20:44Z","id":"gild-purpose1-20240620","ingestedAt":"2026-09-26T07:20:44Z","publishedAt":"2024-06-20","sourceTitle":"PURPOSE 1 interim topline announcement","sourceUrl":"https://investors.gilead.com/news/news-details/2024/Gileads-Twice-Yearly-Lenacapavir-Demonstrated-100-Efficacy-and-Superiority-to-Daily-Truvada-for-HIV-Prevention/default.aspx","summary":"Gilead published results from a PURPOSE 1 interim analysis and reported a committee recommendation to stop the blinded phase. This interim announcement was earlier than the previously indicated update window; it was not final regulatory approval.","supersedes":null,"targetEnd":null,"targetPrecision":"unspecified","targetStart":null,"targetText":null,"type":"outcome"}],"trialIds":[]}],"name":"Gilead Sciences","ticker":"GILD"},{"description":"Develops RNA-targeted medicines. This example follows olezarsen for familial chylomicronemia syndrome.","id":"ionis","milestones":[{"asset":"Olezarsen (TRYNGOLZA)","id":"ionis-olezarsen-fcs-us-review","kind":"regulatory","label":"Olezarsen US review for FCS","nextQuestion":"Which patients were included in the FCS approval, and how does this differ from the separate severe-hypertriglyceridemia program?","records":[{"correctionReason":null,"effectiveAt":null,"firstSeenAt":"2026-09-26T07:20:44Z","id":"ions-olezarsen-20241106","ingestedAt":"2026-09-26T07:20:44Z","publishedAt":"2024-11-06","sourceTitle":"Q3 2024 results: olezarsen FCS review date","sourceUrl":"https://ir.ionis.com/node/31826","summary":"Ionis reported a December 19, 2024 FDA target action date for olezarsen in familial chylomicronemia syndrome. Other olezarsen indications and their trials are separate milestones.","supersedes":null,"targetEnd":"2024-12-19","targetPrecision":"exact","targetStart":"2024-12-19","targetText":"2024-12-19","type":"guidance"},{"correctionReason":null,"effectiveAt":"2024-12-19","firstSeenAt":"2026-09-26T07:20:44Z","id":"ions-olezarsen-20241219","ingestedAt":"2026-09-26T07:20:44Z","publishedAt":"2024-12-19","sourceTitle":"TRYNGOLZA US FCS approval announcement","sourceUrl":"https://ir.ionis.com/news-releases/news-release-details/tryngolzatm-olezarsen-approved-us-first-ever-treatment-adults","summary":"Ionis announced FDA approval of TRYNGOLZA as an adjunct to diet for adults with familial chylomicronemia syndrome.","supersedes":null,"targetEnd":null,"targetPrecision":"unspecified","targetStart":null,"targetText":null,"type":"outcome"}],"trialIds":[]}],"name":"Ionis Pharmaceuticals","ticker":"IONS"},{"description":"Develops medicines across several therapeutic areas. This example follows donanemab in early symptomatic Alzheimer’s disease.","id":"lilly","milestones":[{"asset":"Donanemab","id":"lilly-trailblazer-alz2-topline","kind":"readout","label":"TRAILBLAZER-ALZ 2 Phase 3 topline results","nextQuestion":"What did the prespecified analysis show, and how do the full safety results affect interpretation?","records":[{"correctionReason":null,"effectiveAt":null,"firstSeenAt":"2026-09-26T07:20:44Z","id":"lly-donanemab-20230119","ingestedAt":"2026-09-26T07:20:44Z","publishedAt":"2023-01-19","sourceTitle":"Donanemab regulatory update and Phase 3 timing","sourceUrl":"https://investor.lilly.com/node/48206/pdf","summary":"Lilly expected the confirmatory TRAILBLAZER-ALZ 2 topline readout in Q2 2023. The announcement separately described a complete response letter for the earlier accelerated-approval application; those are different events.","supersedes":null,"targetEnd":"2023-06-30","targetPrecision":"quarter","targetStart":"2023-04-01","targetText":"Q2 2023","type":"guidance"},{"correctionReason":null,"effectiveAt":"2023-05-03","firstSeenAt":"2026-09-26T07:20:44Z","id":"lly-donanemab-20230503","ingestedAt":"2026-09-26T07:20:44Z","publishedAt":"2023-05-03","sourceTitle":"TRAILBLAZER-ALZ 2 topline announcement","sourceUrl":"https://investor.lilly.com/news-releases/news-release-details/lillys-donanemab-significantly-slowed-cognitive-and-functional","summary":"Lilly announced Phase 3 topline results and reported meeting the primary endpoint. The release also described serious amyloid-related imaging abnormalities, including deaths; the readout is not a blanket safety claim.","supersedes":null,"targetEnd":null,"targetPrecision":"unspecified","targetStart":null,"targetText":null,"type":"outcome"}],"trialIds":["NCT04437511"]}],"name":"Eli Lilly and Company","ticker":"LLY"}],"coverageNote":"Reviewed historical examples: 10 companies, 10 milestones and 24 source records from 2022–2025. Recorded by ORION on September 26, 2026. This is a selected evidence set, not a complete company history or continuous monitoring. Later company disclosures may change the current picture. Publication dates are not ORION detection dates.","historical":true,"officialHosts":{"alnylam":["investors.alnylam.com"],"altimmune":["ir.altimmune.com"],"beam":["investors.beamtx.com"],"biogen":["investors.biogen.com","ir.ionis.com"],"biomarin":["investors.biomarin.com"],"crispr":["ir.crisprtx.com","news.vrtx.com"],"exelixis":["ir.exelixis.com"],"gilead":["investors.gilead.com"],"ionis":["ir.ionis.com"],"lilly":["investor.lilly.com"]},"reviewedAt":"2026-09-26T07:20:44Z","schemaVersion":"guidance-ledger-v1"}