An Open-Label Trial to Address the Safety of the SmartFlow MR-Compatible Ventricular Cannula for Administering Eladocagene Exuparvovec to Pediatric Subjects

This study will have a trial phase, extension phase, and a long-term extension phase. The primary objectives of the trial phase are to assess the pharmacodynamics (PD) of eladocagene exuparvovec treatment by evaluation of homovanillic acid (HVA) levels and to assess the safety of the SmartFlow® magnetic resonance (MR) Compatible Ventricular Cannula for administering eladocagene exuparvovec to pediatric participants with aromatic L-amino acid decarboxylase (AADC) deficiency. The extension phase is designed to capture additional clinical information for eladocagene exuparvovec through study evaluations, changes in motor development, AADC-specific symptoms, and other PD measures. The long-term extension phase is designed to capture long-term safety and efficacy data from participants treated with eladocagene exuparvovec.

Start Date12 May 2021

Sponsor / Collaborator

PTC Therapeutics, Inc.

100 Clinical Results associated with Eladocagene exuparvovec

100 Translational Medicine associated with Eladocagene exuparvovec

100 Patents (Medical) associated with Eladocagene exuparvovec

Literatures (Medical) associated with Eladocagene exuparvovec

01 Aug 2024·Current Gene Therapy

Potential Therapeutic Approach using Aromatic l-amino Acid Decarboxylase and Glial-derived Neurotrophic Factor Therapy Targeting Putamen in Parkinson's Disease

Review

Author: Goyal, Lav ; Tripathi, Raman Kumar ; Singh, Shamsher

Abstract::Parkinson's disease (PD) is a neurodegenerative illness characterized by specific loss of dopaminergic neurons, resulting in impaired motor movement. Its prevalence is twice as compared to the previous 25 years and affects more than 10 million individuals. Lack of treatment still uses levodopa and other options as disease management measures. Treatment shifts to gene therapy (GT), which utilizes direct delivery of specific genes at the targeted area. Therefore, the use of aromatic L-amino acid decarboxylase (AADC) and glial-derived neurotrophic factor (GDNF) therapy achieves an effective control to treat PD. Patients diagnosed with PD may experience improved therapeutic outcomes by reducing the frequency of drug administration while utilizing provasin and AADC as dopaminergic protective therapy. Enhancing the enzymatic activity of tyrosine hydroxylase (TH), glucocorticoid hormone (GCH), and AADC in the striatum would be useful for external L-DOPA to restore the dopamine (DA) level. Increased expression of glutamic acid decarboxylase (GAD) in the subthalamic nucleus (STN) may also be beneficial in PD. Targeting GDNF therapy specifically to the putaminal region is clinically sound and beneficial in protecting the dopaminergic neurons. Furthermore, preclinical and clinical studies supported the role of GDNF in exhibiting its neuroprotective effect in neurological disorders. Another Ret receptor, which belongs to the tyrosine kinase family, is expressed in dopaminergic neurons and sounds to play a vital role in inhibiting the advancement of PD. GDNF binding on those receptors results in the formation of a receptor-ligand complex. On the other hand, venous delivery of recombinant GDNF by liposome-based and encapsulated cellular approaches enables the secure and effective distribution of neurotrophic factors into the putamen and parenchyma. The current review emphasized the rate of GT target GDNF and AADC therapy, along with the corresponding empirical evidence.

01 May 2024·Journal of Inherited Metabolic Disease

Gene therapy for aromatic L‐amino acid decarboxylase deficiency: Requirements for safe application and knowledge‐generating follow‐up

Review

Author: François-Heude, Marie Céline ; Zeev, Bruria Ben ; Leuzzi, Vincenzo ; Opladen, Thomas ; Pons, Roser ; Pearson, Toni S ; Kurian, Manju A ; Roubertie, Agathe ; Flint, Lisa ; Cazorla, Angels Garcia ; Brennenstuhl, Heiko ; Kiening, Karl ; Roujeau, Thomas ; Willemsen, Michel A ; Kuseyri Hübschmann, Oya ; Hwu, Paul

AbstractThe autosomal recessive defect of aromatic L‐amino acid decarboxylase (AADC) leads to a severe neurological disorder with manifestation in infancy due to a pronounced, combined deficiency of dopamine, serotonin and catecholamines. The success of conventional drug treatment is very limited, especially in patients with a severe phenotype. The development of an intracerebral AAV2‐based gene delivery targeting the putamen or substantia nigra started more than 10 years ago. Recently, the putaminally‐delivered construct, Eladocagene exuparvovec has been approved by the European Medicines Agency and by the British Medicines and Healthcare products Regulatory Agency. This now available gene therapy provides for the first time also for AADC deficiency (AADCD) a causal therapy, leading this disorder into a new therapeutic era. By using a standardized Delphi approach members of the International Working Group on Neurotransmitter related Disorders (iNTD) developed structural requirements and recommendations for the preparation, management and follow‐up of AADC deficiency patients who undergo gene therapy. This statement underlines the necessity of a framework for a quality‐assured application of AADCD gene therapy including Eladocagene exuparvovec. Treatment requires prehospital, inpatient and posthospital care by a multidisciplinary team in a specialized and qualified therapy center. Due to lack of data on long‐term outcomes and the comparative efficacy of alternative stereotactic procedures and brain target sites, a structured follow‐up plan and systematic documentation of outcomes in a suitable, industry‐independent registry study are necessary.

01 Dec 2023·Advances in therapy

Long-Term Outcomes of Eladocagene Exuparvovec Compared with Standard of Care in Aromatic L-Amino Acid Decarboxylase (AADC) Deficiency: A Modelling Study.

Article

Author: Bergkvist, Mats ; Simons, Claire L ; Bennison, Craig ; Simons, Martijn J H G ; Zhang, Rongrong ; Hwu, Wuh-Liang

INTRODUCTIONAromatic L-amino acid decarboxylase (AADC) deficiency is a rare disease with symptoms including movement disorders, developmental delays, and autonomic symptoms starting from birth; further, patients with AADC deficiency are at a high risk of death in the first decade of life. Limited information on the impact of treatment with gene therapy on patients' disease trajectories and survival, quality-of-life, and resource usage benefits are available.METHODA cohort-based model with a lifetime horizon has been developed, based on motor milestones, to estimate the long-term benefits for patients after treatment with eladocagene exuparvovec compared to best supportive care (BSC). The model takes a National Health Service (NHS) perspective using a UK setting. The model comprises two parts: the developmental phase, in which patients with initially no motor function can progress to other motor milestone states, and a long-term projection phase. Efficacy for eladocagene exuparvovec is derived from clinical trial data with a duration up to 120 months. As the incidence of AADC deficiency is low, data for key model inputs is lacking; therefore estimates of survival by motor milestone were based on proxy diseases. A disease-specific utility study provided quality of life inputs and a burden of illness study informed inputs for disease management.RESULTSThe model indicates survival (25.25 undiscounted life years gained) and quality-of-life benefits (20.21 undiscounted quality-adjusted life years [QALYs] gained) for patients treated with eladocagene exuparvovec compared to BSC. Resource usage costs are greater for patients treated with eladocagene exuparvovec, mainly due to the increased life expectancy during which patients accrue additional healthcare resource usage. Scenario analyses indicate robust results.CONCLUSIONThis study assessed long-term outcomes for patients with AADC deficiency. Patients treated with eladocagene exuparvovec were found to have improved survival and quality of life benefits compared to patients treated with BSC.

News (Medical) associated with Eladocagene exuparvovec

01 Oct 2024

·www.biopharmadive.com

5 FDA decisions to watch in the fourth quarter

It's not a well-known disease. But transthyretin amyloidosis cardiomyopathy, a rare condition affecting the heart, has become the target of a major drug development effort.One medicine, from Pfizer, is approved and two others, from BridgeBio Pharma and Alnylam Pharmaceuticals, have succeeded in late-stage testing. Deciding which offers the greatest benefit has consumed analysts on Wall Street and top experts in the field. At stake is a market worth billions of dollars.By Thanksgiving, the Food and Drug Administration is expected to decide on approval of BridgeBio's acoramidis, likely kicking off a new phase in the debate. The agency is also set to reach verdicts this quarter on important drugs from AstraZeneca, Vertex Pharmaceuticals, Bristol Myers Squibb and PTC Therapeutics. Here are five FDA decisions to watch:BridgeBios acoramidis for transthyretin amyloidosis cardiomyopathyRarely do biotechnology companies rebound from lackluster Phase 3 trial results. But BridgeBio Pharma did exactly that with acoramidis in transthyretin amyloidosis cardiomyopathy.Like Pfizers Vyndamax the only available treatment and a multibillion-dollar seller acoramidis is supposed to stop a protein implicated in the disease from misfolding. Preclinical data suggested it did so more effectively than Vyndamax, raising expectations it might be superior.But BridgeBio had difficulty proving its case. In 2021, the drug missed the first of two main Phase 3 study goals when people who received a placebo performed better on a walking test than past results suggested. BridgeBio attributed that result, in part, to the better all-around care people with the disease now receive, which in turn means studies may have to run longer to detect a benefit.The company was proven right in 2023, as acoramidis extended lives and kept people out of the hospital in that same trial the type of outcome that led to Vyndamaxs U.S. approval.Acoramidis is now on the verge of joining Vyndamax on the U.S. market, should regulators clear it by a Nov. 29 deadline. If approved, treating physicians could soon be left with difficult decisions to make, as its unclear whether acoramidis is more effective than Vyndamax or a rival therapy from Alnylam Pharmaceuticals the FDA could soon be evaluating. Ben FidlerAstraZeneca and Daiichi Sankyos dato-dxd for lung cancerAstraZeneca and Daiichi Sankyo already have one breakout success in Enhertu, a drug the companies teamed up to develop and that has since changed how certain breast cancers are treated. Their chances at another, with a similar type of drug called datopotamab deruxtecan, could hinge on the outcome of an FDA review due to wrap up by Dec. 20.Like Enhertu, dato-dxd is a targeted medicine known as an antibody-drug conjugate. And as with Enhertu, the partners hope to prove the drug can supplant chemotherapy in multiple cancer settings.But Enhertu has succeeded in a variety of different clinical trials, in some cases producing dramatic benefits. Dato-dxds case to regulators is less straightforward.AstraZeneca and Daiichi originally intended to bring the drug to market for all people whose non-small cell lung cancers express a protein, TROP2, that it targets. But while dato-dxd delayed tumor progression compared to chemotherapy, it didnt meaningfully extend survival. It was later associated with a numerical, but not statistically significant improvement when the companies zeroed in on people with nonsquamous tumors, whom the partners expected to benefit the most.AstraZeneca and Daiichi are now aiming for an approval in nonsquamous non-small cell lung tumors specifically. Theyve also been working to better identify responders to strengthen their case for a drug analysts have projected as a future blockbuster. But the survival findings make us suspicious about a straight approval, and could portend a delayed decision and advisory committee meeting, wrote Stifel analyst Eric Le Berrigaud in a research note last month. Ben FidlerVertexs vanza triple for cystic fibrosisSince 2012, Vertex Pharmaceuticals has won U.S. approvals for four cystic fibrosis medicines, making the lung condition manageable for thousands of people. Along the way, Vertex has turned into one of the industry's most valuable companies and is now worth $120 billion.Vertex could expand its cystic fibrosis franchise further if the FDA decides to approve a combination treatment regimen known as “vanza triple.“ The FDA's deadline is Jan. 2, 2025, but the agency sometimes issues decisions early.Like Vertex's current top-seller Trikafta, the vanza triple uses a compound called tezacaftor as its backbone. But the experimental drug replaces Trikafta's two other components with newer versions the company developed.In testing, the vanza triple was found to be “non-inferior“ to Trikafta on a measure of lung function commonly used in cystic fibrosis studies. It also proved superior to Trikafta on a secondary measure of sweat chloride, which is associated with the function of proteins that are mutated in people with the disease.If approved, the vanza triple would offer patients the convenience of once-daily treatment, rather than the twice-daily dosing of Trikafta. And because it contains two newer compounds, Vertex would owe a lower rate of royalty payments than it does selling Trikafta.Vertex could also enjoy longer patent protection on the vanza triple than for Trikafta, which is covered through 2037 in the U.S. Ned PagliaruloPTC's Upstaza for AADC deficiencyOftentimes, the FDA moves ahead of its regulatory counterparts around the world, approving drugs for the U.S. before they're available elsewhere.That pattern flipped for a gene therapy PTC Therapeutics is developing for a rare genetic nervous system disorder known as AADC deficiency. In May 2022, the European Medicines Agency recommended the therapy, called Upstaza, for authorization, which the European Commission granted later that year.But the regulatory process has taken longer in the U.S., where the FDA has gone back and forth with PTC over data intended to demonstrate the comparability of clinical and commercial drug product. The company could finally have its answer by Nov. 13, when the agency is set to decide on approval.An approval would add Upstaza to a lengthening list of approved gene therapies for inherited diseases. But few have been commercial successes, putting pressure on biotech developers like PTC to prove they can profitably manufacture and sell the complex treatments. So far in Europe, PTC has not generated significant revenue from Upstaza. Ned PagliaruloBristol Myers subcutaneous Opdivo for solid tumorsBristol Myers Squibb has earned tens of billions of dollars from its cancer immunotherapy Opdivo since an initial approval a decade ago. But that revenue could dry up within years, as the expiry of a key U.S. patent in 2028 will pave the way for biosimilar competitors. One way Bristol Myers could protect its profits is by bringing a new version of Opdivo to market. That could happen by Dec. 29, when the FDA is expected to issue a verdict on an Opdivo formulation administered via an under-the-skin injection.Subcutaneous Opdivo is the product of a partnership between Bristol Myers and drug delivery specialist Halozyme. The alliance involves a Halozyme technology capable of making a more convenient version of Opdivo, which is typically given through an intravenous infusion lasting 30 minutes. Other drugmakers, among them Johnson & Johnson and Argenx, have worked with Halozyme for similar reasons. So has Roche, which got Halozymes help in making a subcutaneous version of the immunotherapy Tecentriq the FDA approved last month.Bristol Myers and Merck & Co. are trying to catch up. Merck has a formulation of its drug Keytruda currently in late-stage testing, and initial study results could be coming soon, according to a federal database. Bristol Myers is closer, having already proven its under-the-skin Opdivo is statistically non-inferior to the marketed version. Approval would make the newer version available for all the tumors original Opdivo is cleared to treat.Preserving Opdivos revenue is an essential part of Bristol Myers near-term strategy. The companys top seller, the blood thinner Eliquis, will lose market exclusivity in 2026 and faces pricing pressure from Medicare. Jonathan Gardner '

Phase 3Drug ApprovalClinical ResultImmunotherapy

29 May 2024

·www.fiercepharma.com

As many CDMOs battle post-pandemic sales downturn, Almac's revenue jumps amid global expansion push

The CDMO saw a revenue rise over the second year of its global expansion kick. While several CDMO giants have been weathering an industry slowdown in recent years following the pandemic and longing for better days, Almac Group is busy enjoying a growth spurt. Over a busy year ending on Sept. 30, revenue at the U.K.-based manufacturer rose 14% from last year to 958 million pounds ($1.2 billion). The revenue uptick comes during a major global expansion that was announced in 2021 to the tune of 400 million pounds ($508 million). Just this year, Almac wrapped up work on two new facilities in Craigavon, Northern Ireland. One of the plants makes and packages sachet drug products and the other will more than double the company’s peptide API manufacturing capacity. So far, the expansion drive has created 1,800 new jobs, swelling Almac’s total headcount to more than 7,300. During the financial year, the CDMO helped to develop “hundreds of life-saving drugs spanning more than 20 therapeutic areas,” according to a release Tuesday. Recent products Almac has helped supply include Agios’ rare blood disease therapy Pyrukynd, Sanofi’s diabetes injection Tzield and PTC’s brain-infused gene therapy Upstaza, to name a few. Almac’s revenue growth is somewhat of an outlier in the wider CDMO landscape. Manufacturers such as Catalent, Lonza and AGC Biologics face sliding sales amid lagging funding compared to the pandemic boom. AGC Biologics, for example, laid off just under 4% of its global workforce earlier this month due to “changing economies” and “other external factors” that led the Seattle-based CDMO to decide to “right size and rebalance the resources of our organization,” a spokesperson said at the time. During a recent Evaluate webinar on the state of the manufacturing industry in 2024, CDMO sales lead for Pfizer CenterOne’s North America sector pointed out that much of the funding following a pandemic surge of venture capital interest in small- and mid-sized biotechs has dried up, leaving many CDMOs with fewer contracts. Some, like Lonza, are looking on the bright side. The manufacturer recently flagged “initial signs” of an industry recovery for early-stage work. The Swiss company just inked a $1.2 billion deal to purchase a large biologics facility in California from Roche’s Genentech.

Gene Therapy

25 Apr 2024

·www.prnewswire.com

PTC Therapeutics Provides Corporate Update and Reports First Quarter 2024 Financial Results

– Strong revenue performance across product portfolio – – Submitted MAA for sepiapterin and BLA for Upstaza – – On target to achieve remaining 2024 clinical and regulatory milestones, including global submissions for sepiapterin – WARREN, N.J., April 25, 2024 /PRNewswire/ -- PTC Therapeutics, Inc., (NASDAQ: PTCT) today announced a corporate update and financial results for the first quarter ending March 31, 2024. "We are off to a strong start in 2024, with outstanding commercial performance and achievement of all planned clinical and regulatory milestones for the first quarter," said Matthew Klein, M.D., Chief Executive Officer, PTC Therapeutics, Inc. "We remain on track to achieve the many planned 2024 clinical and regulatory milestones, including global regulatory submissions for sepiapterin." Key Corporate Updates: First quarter 2024 total revenue of $210 million First quarter 2024 revenue for the DMD franchise was $161 million Translarna™ (ataluren) net product revenue was $104 million, driven by new patients in existing geographies and continued geographic expansion. Emflaza® (deflazacort) net product revenue was $57 million, resulting from new patient starts and high compliance. Key Clinical and Regulatory Milestones: PTC submitted an MAA to the EMA for sepiapterin for the treatment of PKU in March 2024. The company expects to submit an NDA to the FDA for sepiapterin no later than the third quarter of 2024 and to complete regulatory submissions in Japan and Brazil in 2024. PTC submitted a BLA to the FDA for Upstaza™ for the treatment of AADC deficiency in March 2024. Based on FDA feedback, PTC plans to resubmit the NDA for Translarna for the treatment of nmDMD in mid-2024. Based on FDA feedback, PTC plans to submit an NDA for vatiquinone for the treatment of FA in late 2024. The interim data update for the PIVOT-HD trial of PTC518 for HD patients remains on schedule for the second quarter of 2024. This update will include 12-month data on the initial group of subjects for whom data were reported in June 2023, as well as 12-week data on a larger number of stage 2 and stage 3 HD patients. PTC expects to report topline data for the CardinALS trial of utreloxastat for ALS in the fourth quarter of 2024. First Quarter 2024 Financial Highlights: Total revenues were $210.1 million for the first quarter of 2024, compared to $220.4 million for the first quarter of 2023. Total revenue includes net product revenue across the commercial portfolio of $177.6 million for the first quarter of 2024, compared to $187.6 million for the first quarter of 2023. Total revenue also includes collaboration, royalty, and manufacturing revenue of $32.5 million in the first quarter of 2024, compared to $32.8 million for the first quarter of 2023. Translarna net product revenues were $103.6 million for the first quarter of 2024, compared to $115.1 million for the first quarter of 2023, driven by new patients in existing geographies and continued geographic expansion. The decrease was due to the timing of bulk patient orders. Emflaza net product revenues were $57.5 million for the first quarter of 2024, compared to $54.6 million for the first quarter of 2023. These results were driven by new patient starts and high compliance. Roche reported Evrysdi® 2024 year-to-date sales of approximately $400 million, resulting in royalty revenue of $31.2 million to PTC for the first quarter of 2024, as compared to $30.8 million for the first quarter of 2023. Based on U.S. GAAP (Generally Accepted Accounting Principles), GAAP R&D expenses were $116.1 million for the first quarter of 2024, compared to $195.1 million for the first quarter of 2023. The decrease in research and development expenses reflects strategic portfolio prioritization as the Company continues to focus its resources on its differentiated, high-potential R&D programs. Non-GAAP R&D expenses were $107.2 million for the first quarter of 2024, excluding $9.0 million in non-cash, stock-based compensation expense, compared to $179.8 million for the first quarter of 2023, excluding $15.3 million in non-cash, stock-based compensation expense. GAAP SG&A expenses were $73.3 million for the first quarter of 2024, compared to $86.9 million for the first quarter of 2023. The decrease in selling, general and administrative expenses reflects lower employee costs as a result of the reduction in workforce in 2023. Non-GAAP SG&A expenses were $63.9 million for the first quarter of 2024, excluding $9.4 million in non-cash, stock-based compensation expense, compared to $73.4 million for the first quarter of 2023, excluding $13.5 million in non-cash, stock-based compensation expense. Change in the fair value of deferred and contingent consideration was a gain of $0.1 million for the first quarter of 2024, compared to a loss of $2.4 million for the first quarter of 2023. The net loss was $91.6 million for the first quarter of 2024, compared to a net loss of $139.0 million for the first quarter of 2023. Cash, cash equivalents, and marketable securities was $884.8 million on March 31, 2024, compared to $876.7 million on December 31, 2023. Shares issued and outstanding as of March 31, 2024, were 76,653,960. PTC Reaffirms Full Year 2024 Financial Guidance: PTC anticipates total revenues for full year 2024 to be between $600 million and $680 million. PTC anticipates GAAP R&D and SG&A expense for full year 2024 to be between $740 and $835 million. PTC anticipates Non-GAAP R&D and SG&A expense for full year 2024 to be between $660 and $755 million, including expected R&D expense milestone payments of up to $65 million and excluding estimated non-cash, stock-based compensation expense of $80 million. PTC anticipates up to $90 million of payments for full year 2024 upon achievement of potential regulatory success-based milestones from previous acquisitions, of which up to $65 million will be recorded as R&D operating expense. Non-GAAP Financial Measures: In this press release, the financial results of PTC are provided in accordance with GAAP and using certain non-GAAP financial measures. In particular, the non-GAAP R&D and SG&A expense financial measures exclude non-cash, stock-based compensation expense. These non-GAAP financial measures are provided as a complement to financial measures reported in GAAP because management uses these non-GAAP financial measures when assessing and identifying operational trends. In management's opinion, these non-GAAP financial measures are useful to investors and other users of PTC's financial statements by providing greater transparency into the historical and projected operating performance of PTC and the company's future outlook. Non-GAAP financial measures are not an alternative for financial measures prepared in accordance with GAAP. Quantitative reconciliations of the non-GAAP financial measures to their respective closest equivalent GAAP financial measures are included in the table below. Acronyms: AADC: Aromatic l-Amino Acid Decarboxylase ALS: Amyotrophic Lateral Sclerosis BLA: Biologics License Application CHF: Confoederatio Helvetica Francs (Swiss francs) DMD: Duchenne Muscular Dystrophy EMA: European Medicines Agency FA: Friedreich Ataxia FDA: U.S. Food and Drug Administration GAAP: Generally Accepted Accounting Principles HD: Huntington's Disease MAA: Marketing Authorization Application NDA: New Drug Application nmDMD: Nonsense Mutation Duchenne Muscular Dystrophy PKU: Phenylketonuria R&D: Research and Development SG&A: Selling, General, and Administrative Today's Conference Call and Webcast Reminder: To access the call by phone, please click here to register and you will be provided with dial-in details. To avoid delays, we recommend participants dial in to the conference call 15 minutes prior to the start of the call. The webcast conference call can be accessed on the Investor section of the PTC website at . A replay of the call will be available approximately two hours after completion of the call and will be archived on the company's website for 30 days following the call. About PTC Therapeutics, Inc. PTC is a global biopharmaceutical company focused on the discovery, development and commercialization of clinically differentiated medicines that provide benefits to patients with rare disorders. PTC's ability to globally commercialize products is the foundation that drives investment in a robust and diversified pipeline of transformative medicines and our mission to provide access to best-in-class treatments for patients who have an unmet medical need. The company's strategy is to leverage its strong scientific expertise and global commercial infrastructure to maximize value for its patients and other stakeholders. To learn more about PTC, please visit us at and follow us on Facebook, on Twitter at @PTCBio, and on LinkedIn. For More Information: Investors: Kylie O'Keefe +1 (908) 300-0691 [email protected] Media: Jeanine Clemente +1 (908) 912-9406 [email protected] Forward-Looking Statements: This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. All statements contained in this release, other than statements of historic fact, are forward-looking statements, including the information provided under the heading "PTC Reaffirms Full Year 2024 Revenue Guidance", including with respect to (i) 2024 total revenue guidance, (ii) 2024 GAAP and non-GAAP R&D and SG&A expense guidance and (iii) 2024 acquisition related milestone payment guidance, and statements regarding: the future expectations, plans and prospects for PTC, including with respect to the expected timing of clinical trials and studies, availability of data, regulatory submissions and responses, commercialization and other matters with respect to its products and product candidates; PTC's strategy, future operations, future financial position, future revenues, projected costs; the extent, timing and financial aspects of our strategic pipeline prioritization and reductions in workforce; and the objectives of management. Other forward-looking statements may be identified by the words, "guidance", "plan," "anticipate," "believe," "estimate," "expect," "intend," "may," "target," "potential," "will," "would," "could," "should," "continue," and similar expressions. PTC's actual results, performance or achievements could differ materially from those expressed or implied by forward-looking statements it makes as a result of a variety of risks and uncertainties, including those related to: the outcome of pricing, coverage and reimbursement negotiations with third party payors for PTC's products or product candidates that PTC commercializes or may commercialize in the future; PTC's ability to maintain its marketing authorization of Translarna for the treatment of nmDMD in Brazil, Russia, the European Economic Area (EEA) and other regions, including whether the European Commission adopts the negative opinion from the Committee for Medicinal Products for Human Use (CHMP) for the conditional marketing authorization for Translarna in the EEA; PTC's ability to use the results of Study 041, a randomized, 18-month, placebo-controlled clinical trial of Translarna for the treatment of nmDMD followed by an 18-month open-label extension, and from its international drug registry study to support a marketing approval for Translarna for the treatment of nmDMD in the United States; whether investigators agree with PTC's interpretation of the results of clinical trials and the totality of clinical data from our trials in Translarna; expectations with respect to Upstaza, including any regulatory submissions and potential approvals, commercialization, manufacturing capabilities, the potential achievement of development, regulatory and sales milestones and contingent payments that PTC may be obligated to make; expectations with respect to the commercialization of Evrysdi under our SMA collaboration; expectations with respect to the commercialization of Tegsedi and Waylivra; the timing of and actual expenses incurred in connection with the discontinuation of PTC's preclinical and early research programs in gene therapy and reductions in workforce, which may be in different periods and may be materially higher than estimated; the savings that may result from the discontinuation of PTC's strategic pipeline prioritization and reductions in workforce, which may be materially less than expected; significant business effects, including the effects of industry, market, economic, political or regulatory conditions; changes in tax and other laws, regulations, rates and policies; the eligible patient base and commercial potential of PTC's products and product candidates; PTC's scientific approach and general development progress; the potential financial impact and benefits of PTC's leased biologics manufacturing facility; PTC's ability to satisfy its obligations under the terms of its lease agreements, including for its leased biologics manufacturing facility; the sufficiency of PTC's cash resources and its ability to obtain adequate financing in the future for its foreseeable and unforeseeable operating expenses and capital expenditures; and the factors discussed in the "Risk Factors" section of PTC's most recent Quarterly Report on Form 10-Q and Annual Report on Form 10-K, as well as any updates to these risk factors filed from time to time in PTC's other filings with the SEC. You are urged to carefully consider all such factors. As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commercialization of new products. There are no guarantees that any product will receive or maintain regulatory approval in any territory, or prove to be commercially successful, including Translarna, Emflaza, Upstaza, Evrysdi, Tegsedi or Waylivra. The forward-looking statements contained herein represent PTC's views only as of the date of this press release and PTC does not undertake or plan to update or revise any such forward-looking statements to reflect actual results or changes in plans, prospects, assumptions, estimates or projections, or other circumstances occurring after the date of this press release except as required by law. SOURCE PTC Therapeutics, Inc.

Financial StatementNDA

100 Deals associated with Eladocagene exuparvovec

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Approved

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Indication	Country/Location	Organization	Date
Aromatic Amino Acid Decarboxylase Deficiency	NO	PTC Therapeutics International Ltd.	18 Jul 2022
Aromatic Amino Acid Decarboxylase Deficiency	LI	PTC Therapeutics International Ltd.	18 Jul 2022
Aromatic Amino Acid Decarboxylase Deficiency	EU	PTC Therapeutics International Ltd.	18 Jul 2022
Aromatic Amino Acid Decarboxylase Deficiency	IS	PTC Therapeutics International Ltd.	18 Jul 2022

Developing

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Indication	Highest Phase	Country/Location	Organization	Date
Aromatic Amino Acid Decarboxylase Deficiency	NDA/BLA	US	PTC Therapeutics, Inc.	19 Mar 2024

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


AAN2023	Not Applicable	Aromatic Amino Acid Decarboxylase Deficiency	30	Eladocagene exuparvovec	nqdpbfaaqx(tegziykuso) = xfpzvxwrdp byfjdesxwa (djkmqdignf, partial head control)	Positive	25 Apr 2023
AADCAware (SSIEM2022)	Not Applicable	Aromatic Amino Acid Decarboxylase Deficiency	-	eladocagene exuparvovec	fzwpxiayuy(yklqdidqwv) = ulldkbmmou ywshjdtlds (rfienuisil ) View more	-	30 Aug 2022
AADCAware (SSIEM2022)	Not Applicable	Aromatic Amino Acid Decarboxylase Deficiency	-	Participants receiving eladocagene exuparvovec	fzwpxiayuy(yklqdidqwv) = ltdxxdeczp ywshjdtlds (rfienuisil ) View more	-	30 Aug 2022
4688 (AAN2020)	Not Applicable	Aromatic Amino Acid Decarboxylase Deficiency	26	Eladocagene Exuparvovec	dnyfjuwcue(pefbchshkc) = Dyskinesia was recorded as an AE in 23 patients in the safety population; most events were mild or moderate, occurred within 3 months after eladocagene exuparvovec treatment, generally responded to standard pharmacotherapy, and resolved in all patients by 10 months oxubiqkeyo (gacwyxhzlz ) View more	-	14 Apr 2020

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Bio

Bio Sequences Search & Analysis

Chemical

Chemical Structures Search & Analysis

Eladocagene exuparvovec

Overview

Basic Info

Gene Sequence

Related

R&D Status

Clinical Result

Translational Medicine

Deal

Core Patent

Clinical Trial

Approval

Regulation