Highest-materiality recent filing
PTC Therapeutics to acquire ST-920 gene therapy for Fabry disease from Sangamo for $111M upfront
Upfront $111M plus up to $100M milestones: $80M on FDA accelerated approval, $20M on full approval of ST-920 BLA.
PTC wins bankruptcy auction for Sangamo's Fabry gene therapy ST-920; $111M upfront
Deal: $111M upfront plus up to $100M milestones ($80M on accelerated approval, $20M on traditional FDA approval).
PTC Therapeutics Q2 net income $83.5M, raises FY product revenue guidance to $850-950M
Q2 total revenue $361M (product $239M) includes $50M Novartis milestone; net income $83.5M vs net loss $64.8M YoY.
Issued $550M aggregate principal of 0.0% Convertible Senior Notes due 2031, initial conversion price ~$107.48/share.
PTC Therapeutics prices $500M 0% convertible notes due 2031 to refinance 2026 notes
Priced $500M aggregate principal of 0% Convertible Senior Notes due 2031; initial purchasers have option for additional $50M.
Director Alethia Young resigns from PTC board, stays as consultant at $4,167/month
Alethia Young resigned from the Board effective June 8, 2026; no disagreement cited.
PTC Therapeutics shareholders elect four Class I directors and ratify Ernst & Young
Jessica Chutter, Matthew Klein, Stephanie Okey, and Jerome Zeldis elected Class I directors with >83% of votes cast.
PTC Therapeutics Q1 revenue $273M, raises FY product revenue guidance to $750-850M
Q1 2026 total revenue $273M; product revenue $226M, up 47% YoY.
Stage 2 HD participants showed 52% (10 mg) and 28% (5 mg) slowing on cUHDRS vs natural history at 24 months.
PTC Therapeutics appoints biotech banking veteran Jessica Chutter to board
Jessica Chutter appointed to board effective March 24, 2026, as Class I director.
PTC Therapeutics reports FY2025 product/royalty rev $831M, beats guidance; Sephience Q4 rev $92M
Full-year 2025 product/royalty revenue $831M, exceeding guidance; total revenue $1.73B (includes $998M Novartis collaboration).
PTC Therapeutics withdraws Translarna NDA resubmission after FDA cites insufficient efficacy data
Withdrew NDA resubmission for Translarna (ataluren) for nonsense mutation Duchenne muscular dystrophy.
Preliminary 2025 total product & royalty revenue ~$823.4M, exceeding prior guidance; product revenue ~$587.8M.
PTC Therapeutics sells remaining Evrysdi royalty stake to Royalty Pharma for $240M upfront
Sold remaining 9.5111%/16.6666% royalty interest in Evrysdi worldwide net sales for $240M upfront cash.
PTC Therapeutics Q3 net income $15.9M, Sephience launch revenue $19.6M
Total revenue $211.0M vs $196.8M YoY; net income $15.9M vs loss of $106.7M.
FDA denies approval of PTC Therapeutics' vatiquinone NDA for Friedreich's ataxia
FDA CRL states substantial evidence of efficacy not demonstrated; additional well-controlled study needed for resubmission.
PTC Therapeutics Q2 2025: Sephience approved in EU and US; total revenue $179M; net loss narrows
Sephience (sepiapterin) received EC and FDA approval with broad label for PKU across all ages and subtypes; global launch initiated.
PTC Therapeutics pays ~$225M upfront to cancel Censa sepiapterin royalty obligations
Upfront cash payment of ~$225M to former Censa securityholders eliminates 8-12% annual net sales royalties.
FDA approved Sephience for hyperphenylalaninemia in adult/pediatric patients ≥1 month with sepiapterin-responsive PKU.
PTC Therapeutics reports results of 2025 annual meeting; all three proposals approved
Elected Allan Jacobson (60,386,986 for), David Southwell (59,200,375), Alethia Young (54,894,657) as Class III directors to 2028 term.
PTC Therapeutics Q1 net income $866.6M on Novartis deal; guides FY revenue $650-800M
Net income $866.6M vs loss $91.6M YoY, driven by $986.2M upfront from Novartis PTC518 license deal.
PTC518 Phase 2 PIVOT-HD meets primary endpoint with dose-dependent HTT lowering at Week 12
Primary endpoint met: dose-dependent blood HTT protein reduction (p<0.0001) at Week 12.
European Commission refuses renewal of Translarna (ataluren) marketing authorization in EU
EC adopted CHMP negative opinion; renewal of conditional MA for nonsense mutation DMD denied.
PTC Therapeutics reports FY2024 revenue $807M above guidance; cash >$2B after Novartis deal
FY2024 total revenue $806.8M, exceeding guidance; net loss $363.3M vs $626.6M prior year.
Preliminary 2024 total revenue ~$814M, net product revenue ~$601M, and Evrysdi royalties ~$211M.
PTC Therapeutics cuts leased space in half, reduces base rent by $57.7M
Surrendered one of two buildings; premises reduced from 360k to 180k sq ft.
PTC Therapeutics signs $1B upfront license deal with Novartis for PTC518 HD program
PTC receives $1.0B upfront; eligible for up to $1.9B in development, regulatory and sales milestones.
PTC Therapeutics sells Rare Pediatric Disease PRV for $150 million cash
Sale price of $150 million payable in cash upon closing to an affiliate of a large pharmaceutical company.
PTC Therapeutics Q3 revenue $196.8M, raises FY2024 guidance to $750-800M, multiple FDA submissions
Q3 total revenue $196.8M (vs $196.6M YoY); DMD franchise $124M (Translarna $72M, Emflaza $52M).
CHMP maintains negative opinion on PTC's Translarna renewal; EC decision awaited in ~67 days
CHMP upheld negative opinion on renewal of conditional marketing authorization for Translarna (ataluren) in nmDMD after re-examination.
PTC Therapeutics Q2 revenue $187M; net loss narrows to $99M; NDAs submitted
Q2 total revenue $186.7M vs $213.8M YoY; DMD franchise $118M (Translarna $70M, Emflaza $47M).
CHMP issues negative opinion on Translarna renewal; PTC plans re-examination
CHMP issued negative opinion on renewal of conditional marketing authorization for Translarna (ataluren) for nmDMD.
PTC518 Ph2 PIVOT-HD shows mHTT lowering and clinical trends; FDA lifts partial clinical hold
PTC518 lowered mHTT in blood by 22% (5mg) and 43% (10mg) at Month 12; similar CSF levels.
EC rejects CHMP negative opinion on Translarna; returns for re-evaluation; 2024 guidance paused
EC decided not to adopt CHMP's negative opinion for Translarna annual renewal; returns to CHMP for re-evaluation.
PTC Therapeutics Q1 total revenue $210M, net loss narrows to $91.6M; pipeline milestones on track
Q1 total revenue $210M (down ~5% YoY); DMD franchise $161M (Translarna $104M, Emflaza $57M).
PTC Therapeutics 2023 revenue up 34% to $938M; guides 2024 revenue $600-680M
Total revenue $937.8M (+34% YoY); net loss $626.6M vs $559.0M in 2022.
PTC Therapeutics' Translarna faces withdrawal in Europe after CHMP negative opinion on renewal
CHMP issued negative opinion on renewal of conditional marketing authorization for Translarna (ataluren) after re-examination procedure.
PTC reports prelim 2023 revenue $946M (+35% YoY); guides 2024 rev $600-850M
Unaudited 2023 total revenue $946M (+35% YoY); net product revenue $661M (+23% YoY).
PTC Therapeutics Q3 total revenues $196.6M (-9% YoY), net loss $133M; updates pipeline timelines
DMD franchise revenue $136M in Q3; full-year 2023 DMD guidance raised to $565-595M.
PTC Therapeutics monetizes Evrysdi royalties for up to $1.5B, pays off Blackstone debt
PTC receives $1.0B upfront for ~38% additional Evrysdi royalty; total ~81% sold to Royalty Pharma.
PTC Therapeutics terminates $302.1M credit agreement, pays $82.1M in prepayment premiums
Terminated Credit Agreement dated Oct 27, 2022; repaid $302.1M principal plus accrued interest.
PTC Therapeutics cuts 25% workforce, targets 20% OPEX reduction
Workforce reduction of ~25% primarily in US early-stage research and gene therapy manufacturing.
PTC Therapeutics removes redactions from $300M credit agreement, disclosing Events of Default terms
Filed unredacted Credit Agreement dated Oct 27, 2022; initial term loan facility of $300M and delayed draw up to $150M.
CHMP issues negative opinion on Translarna full authorization; PTC to seek re-examination
CHMP gave negative opinion on converting Translarna's conditional MA to full MA for nmDMD.
PTC Therapeutics Q2 revenue $214M (+29% YoY); raises full-year guidance to $940M-$1.0B
Total revenue $213.8M vs $165.5M Q2 2022; DMD franchise $162M (+21%).
PTC Therapeutics provides regulatory timeline updates for sepiapterin, Upstaza, Translarna
Expects to submit NDA to FDA for sepiapterin (PKU) in Q4 2023.
PTC Therapeutics appoints Pierre Gravier as CFO effective July 13, 2023
Pierre Gravier, age 38, appointed CFO; previously managing director at Perella Weinberg Partners.
PTC Therapeutics reports positive interim Phase 2 data for PTC518 in Huntington's disease
Interim data from PIVOT-HD Phase 2 study shows dose-dependent lowering of HTT protein in blood, with mean 30% reduction at 10mg dose.
Elected Class I directors: William F. Bell, Jr., Matthew B. Klein, Stephanie S. Okey, Jerome B. Zeldis; each received >58M votes for.
Discontinues preclinical/early gene therapy programs; workforce reduction of ~8%; expects 15% OPEX savings in 2023; severance costs ~$7M.
On June 8, 2026, Alethia Young, a member of the board of directors (the "Board") of PTC Therapeutics, Inc. (the "Company"), notified the Company of her resignation from the Board effective June 8, 2026.
On July 13, 2023, the Board of Directors of PTC Therapeutics, Inc. (the “Company”) appointed Pierre Gravier, age 38, as Chief Financial Officer, effective as of July 13, 2023.
On May 20, 2023, the Company informed Emily Hill, its Chief Financial Officer, that her employment will be terminated effective as of August 20, 2023, and effective immediately she will cease to serve as the Company's Chief Financial Officer.
Dr. Stuart W. Peltz, who provided the Board with notice on March 22, 2023 of his decision to retire as Chief Executive Officer and as a member of the Board, in each case, effective as of March 22, 2023.
the Board of Directors (the “Board”) of PTC Therapeutics, Inc. (the “Company”) appointed Dr. Matthew B. Klein, age 51, as Chief Executive Officer and President and a member of the Board, effective as of March 22, 2023.
On December 5, 2022, Dawn Svoronos, a member of the board of directors (the “Board”) of PTC Therapeutics, Inc. (the “Company”), notified the Company of her resignation from the Board effective December 6, 2022.
Max materiality 0.90 · Median 0.75 · Most common event earnings