Key TakeawaysShares of PTC Therapeutics soared Monday when the pharmaceutical firm announced it struck a nearly $2 billion license and collaboration deal with Novartis.The deal gives Novartis global development, manufacturing, and commercial responsibility for PTC's experimental Huntington's Disease drug.PTC will receive $1.0 billion upfront, and said it would use the money to expand its splicing platform and increase its commercial and development portfolio moves. Shares of PTC Therapeutics (PTCT) soared 1 ...

On Monday, PTC Therapeutics, Inc. PTCT signed an exclusive global license and collaboration agreement with Novartis AG NVS for its PTC518 Huntington’s disease program, which includes related molecules.Under the agreement, PTC will receive an upfront payment of $1.0 billion, up to $1.9 billion in development, regulatory and sales milestones, a profit share in the U.S., and double-digit tiered royalties on ex-U.S. sales.Also Read: FDA Grants Conditional Approval For PTC Therapeutics’ Gene Therapy As First Eve ...

- PTC to receive $1.0B in cash at closing -- PTC is eligible to receive up to $1.9B in development, regulatory and sales milestones -- PTC to share profits in the U.S. and tiered double-digit royalties on ex-U.S. net sales -- Novartis will assume global development, manufacturing and commercial responsibilities following completion of placebo-controlled portion of ongoing PIVOT-HD study -- PTC will host a conference call on Dec. 2, 2024, at 8:30 am EST- WARREN, N.J., Dec. 2, 2024 /PRNewswire/ -- PTC Therape ...

Core Viewpoint - PTC Therapeutics, Inc. has announced an agreement to sell its Rare Pediatric Disease Priority Review Voucher for $150 million, contingent upon the closing of the transaction [1][2]. Group 1: Transaction Details - The Rare Pediatric Disease Priority Review Voucher was granted to PTC on November 13, 2024, alongside FDA approval for KEBILIDI™ for treating AADC deficiency [2]. - The transaction is subject to customary closing conditions, including the expiration of the applicable waiting period under the Hart-Scott Rodino Antitrust Improvements Act [2]. Group 2: About the PRV Program - The FDA awards priority review vouchers to sponsors of rare pediatric disease product applications that meet specific criteria, aimed at encouraging the development of new drugs for rare pediatric diseases [3]. Group 3: Company Overview - PTC Therapeutics is a global biopharmaceutical company focused on discovering, developing, and commercializing clinically differentiated medicines for rare disorders [4]. - The company aims to provide access to best-in-class treatments for patients with limited treatment options, leveraging its scientific expertise and global commercial infrastructure [4].

Core Insights - PTC Therapeutics announced that the Phase 2 CardinALS study did not meet its primary endpoint of slowing disease progression in ALS patients, with a p-value of 0.52 indicating lack of statistical significance [1][2] - Despite showing a modest numerical benefit and correlation with a biomarker (NfL), the study's secondary efficacy endpoints also did not achieve significance [1][2] - Utreloxastat was found to be safe and well tolerated, but due to the lack of efficacy, further development is not planned [2] Company Overview - PTC Therapeutics is a global biopharmaceutical company focused on developing and commercializing medicines for rare disorders, aiming to provide access to treatments for patients with limited options [3] - The company's strategy leverages scientific expertise and global infrastructure to maximize value for stakeholders [3]

Core Points - The FDA has granted accelerated approval for PTC Therapeutics' gene therapy KEBILIDI™ for AADC deficiency, marking the first gene therapy approved for direct brain administration in the U.S. [1][4] - KEBILIDI is indicated for both children and adults, addressing a rare genetic disorder that severely impacts motor function due to dopamine synthesis inability [3][8]. - The therapy is delivered via a minimally invasive stereotactic neurosurgical procedure, with trained surgeons and specialized centers prepared for its administration [7][8]. Company Overview - PTC Therapeutics is focused on developing and commercializing innovative treatments for rare disorders, leveraging scientific expertise and a global commercial infrastructure [14]. - The company aims to maximize value for stakeholders by providing access to transformative medicines for patients with limited treatment options [14]. Product Details - KEBILIDI is a recombinant adeno-associated virus-based gene therapy designed to correct the genetic defect causing AADC deficiency by delivering a functioning DDC gene directly into the brain [6]. - Clinical trials have shown that KEBILIDI leads to the synthesis of dopamine and improvement in motor development milestones [3][4]. Regulatory Milestones - The approval was based on safety and efficacy findings from an ongoing global clinical trial (PTC-AADC-GT-002), with further confirmatory evidence expected from long-term patient follow-up [4]. - A Rare Disease Priority Review Voucher was also granted, which the company plans to monetize [4].

– Strong revenue performance, supporting increase in full-year revenue guidance to $750-800 million – – Regulatory filings for sepiapterin, Translarna™ and AADC gene therapy under review by FDA –– On track to achieve remaining 2024 clinical and regulatory milestones, including NDA submission for vatiquinone –WARREN, N.J., Nov. 7, 2024 /PRNewswire/ -- PTC Therapeutics, Inc., (NASDAQ: PTCT) today announced a corporate update and financial results for the third quarter ending September 30, 2024. "I am proud of ...

PTC Inc (PTC) reported fourth-quarter fiscal 2024 non-GAAP earnings per share (EPS) of $1.54, up 28% year over year. The figure beat the Zacks Consensus Estimate by 7.7%.Stay up-to-date with all quarterly releases: See Zacks Earnings Calendar.Quarterly revenues soared 15% year over year to $627 million. The Zacks Consensus Estimate for revenues was pegged at $620.5 million. The top line neared the high end of PTC’s guidance, driven by continued demand for its product lifecycle management (PLM) and computer- ...

Financial Data and Key Metrics Changes - In fiscal 2024, free cash flow grew by 25% year-over-year, reaching $736 million [8][34] - Constant currency ARR increased by 12% year-over-year, totaling $2.207 billion at the end of Q4 [8][33] - Operating efficiency improved by 370 basis points to 42% compared to 38% in fiscal 2023 [34] Business Line Data and Key Metrics Changes - In Q4, constant currency ARR growth was 10% in CAD and 13% in PLM, indicating strong performance in these segments [41] - The company reported solid results in areas like APAC and its reseller channel, while Western Europe showed relative weakness [9][41] Market Data and Key Metrics Changes - Organic constant currency ARR growth was solid across the Americas, Europe, and APAC, with growth rates in the low to mid-double digits [42] - The automotive sector in Western Europe is under pressure, affecting deal sizes and timelines, but there is a push for digital transformation [89][90] Company Strategy and Development Direction - The company is focusing on five key areas: PLM (Windchill), ALM (Codebeamer), SLM (ServiceMax), CAD (Creo), and SaaS [11][12] - A new Chief Revenue Officer will be hired to enhance focus and accountability in a vertically oriented go-to-market model [19][21] - The go-to-market structure is being realigned around key verticals to improve specialization and effectiveness [20][22] Management's Comments on Operating Environment and Future Outlook - Management acknowledges macroeconomic headwinds and geopolitical uncertainty but remains confident in the long-term trajectory due to a shift in key verticals [9][10] - There is a strong demand for digital transformation among customers, which the company's offerings are well-positioned to address [12][15] Other Important Information - A $2 billion share repurchase authorization was announced, reflecting confidence in the business and commitment to enhancing shareholder value [10][45] - The company expects free cash flow to grow faster than ARR over the medium term, with operating expenses growing at roughly half the rate of ARR [34][35] Q&A Session Summary Question: Go-to-market disruption and growth rates - Management is not expecting significant disruption from the go-to-market changes and has mechanisms in place to minimize any potential impact [78][79] Question: Market performance and product focus - Strong performance is expected in PLM and CAD, with a focus on expanding Windchill and Codebeamer in key verticals [82][84] Question: Demand in Western Europe - There is pressure in the automotive sector, but this is driving urgency for digital transformation, which could benefit the company [88][90] Question: Timing of go-to-market changes and potential demand recovery - The company is prepared for potential demand increases and is aligning its operations to capitalize on opportunities as they arise [92][94] Question: Performance across other verticals - The federal, aerospace, and defense sectors are experiencing significant demand, while med tech and life sciences are also seeing a need for faster product development [99][100]

Q4'24 AND FY'24 FINANCIAL RESULTS Neil Barua CEO Kristian Talvitie CFO November 6, 2024 FORWARD LOOKING STATEMENTS 2 This presentation includes forward looking statements regarding PTC's future financial performance, strategic outlook and expectations, anticipated future operations, expected effects of strategic investments and initiatives, and share repurchase expectations. Because such statements deal with future events, actual results may differ materially from those projected in the forward-looking stat ...