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PTC THERAPEUTICS INC

PTC Therapeutics

PTC Therapeutics Inc is a biopharmaceutical company that discovers, develops and commercializes clinically differentiated medicines that provide benefits to children and adults living with rare disorders. Its diversified therapeutic portfolio that includes several commercial products and product candidates in various stages of development, including discovery, research and clinical stages, focused on the development of new treatments for multiple therapeutic areas for rare diseases relating to neurology and metabolism. The Company views its operations and manages its business in one operating segment: life science.

PTC Therapeutics Inc

WIIM

PTCT

PTC Therapeutics shares are trading lower after JP Morgan cut its price target on the stock from $78 to $75.

JP Morgan analyst Eric Joseph maintains PTC Therapeutics (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/PTCT#NASDAQ">PTCT</a>) with a Overweight and lowers the price target from $78 to $75.

News

Price Target

Analyst Ratings

JP Morgan Maintains Overweight on PTC Therapeutics, Lowers Price Target to $75

The&nbsp;European Commission (EC) adopted the opinion of the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA)&nbsp;not to renew the authorization of PTC Therapeutics, Inc.'s (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/PTCT#NASDAQ">PTCT</a>) Translarna (ataluren) for nonsense <a target="_blank" href="https://www.benzinga.com/general/biotech" rel="noreferrer noopener">mutation Duchenne muscular dystrophy (nmDMD)</a>.
Also Read: <a target="_blank" href="https://www.benzinga.com/general/biotech/25/03/44419721/ptc-therapeutics-reveals-new-sepiapterin-data-from-phase-3-study-in-patients-with-inherited-disorder-fda-decision-expected-in-july" rel="noreferrer noopener">PTC Therapeutics Reveals New Sepiapterin Data From Phase 3 Study In Patients With Inherited Disorder, FDA Decision Expected In July</a>
While this action effectively removes the drug's conditional marketing authorization in the European Economic Area, the EC indicated that individual countries within the European Union could leverage Articles 117(3) and 5(1) of the EU Directive 2001/83 to allow <a target="_blank" href="https://www.benzinga.com/pressreleases/25/03/n44535458/ptc-therapeutics-provides-regulatory-update-on-translarna-ataluren-in-europe" rel="noreferrer noopener">continued use of Translarna</a>.
"We are of course disappointed that after this prolonged period of review the European Commission has decided to adopt the CHMP negative opinion on Translarna," said&nbsp;Matthew B. Klein, PTC Therapeutics CEO in a statement on Friday.
"We look forward to working on a country-by-country basis to provide commercial drug where possible."
In May 2024, PTC Therapeutics announced that the European Commission (EC) has decided not to adopt the CHMP's negative opinion on the annual renewal of Translarna's conditional marketing authorization (ataluren) and has returned the opinion to the&nbsp;<a target="_blank" href="https://www.benzinga.com/general/biotech/24/05/38922172/european-commission-requests-reevaluation-of-opinion-on-ptc-therapeutics-duchenne-dystrophy-drug-" rel="noreferrer noopener">CHMP for re-evaluation</a>.&nbsp;
Translarna's net product revenues were&nbsp;$339.9 million&nbsp;for the full year 2024, compared to&nbsp;$355.8 million&nbsp;<a target="_blank" href="https://www.benzinga.com/pressreleases/25/02/n44024275/ptc-therapeutics-provides-corporate-update-and-reports-fourth-quarter-and-full-year-2024-financial" rel="noreferrer noopener">for the full year 2023</a>.
In 2024, PTC submitted four regulatory approval applications to the FDA, all of which have been accepted for review, including Translarna for nmDMD.
Price Action: PTCT stock is down 1.76% at $54.02&nbsp;at the last check Friday.
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Biotech

Health Care

General

European Commission Declines To Renew Marketing Authorization Of Translarna Duchenne Muscular Disorder

On Thursday, PTC Therapeutics, Inc.&nbsp;(NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/PTCT#NASDAQ">PTCT</a>) shared new data from the Phase 3 APHENITY trial and subsequent open-label extension study at the 2025 American College of Medical Genetics and Genomics (ACMG) <a target="_blank" href="https://www.benzinga.com/general/health-care" rel="noreferrer noopener">Annual Clinical Genetics Meeting</a>.
These data provide further evidence of the potential benefits of sepiapterin treatment for the full spectrum of phenylketonuria (PKU) patients.
Also Read: <a target="_blank" href="https://www.benzinga.com/24/12/42272846/ptc-therapeutics-inks-licensing-pact-with-novartis-worth-3-billion-for-huntingtons-program" rel="noreferrer noopener">PTC Therapeutics Inks Licensing Pact With Novartis Worth Up To ~$3 Billion For Huntington&#8217;s Program</a>
Phenylketonuria is an inherited disorder that increases the levels of a substance called <a target="_blank" href="https://www.benzinga.com/pressreleases/25/03/n44414358/ptc-therapeutics-presents-new-sepiapterin-data-from-ongoing-studies" rel="noreferrer noopener">phenylalanine in the blood</a>.
Untreated phenylketonuria can lead to brain damage, intellectual disabilities, behavioral symptoms or seizures.
&#8220;These new data provide further evidence of the meaningful benefit sepiapterin can provide for children and adults with PKU, including those with the most severe form of the disease,&#8221; said Matthew Klein, CEO of PTC Therapeutics. &#8220;Furthermore, the treatment benefit demonstrated in individuals with mutations not responsive to BH4 and consistent with classical disease phenotype support the potential to penetrate all key patient segments.&#8221;
Treatment includes a strict diet with limited protein.
Key data presented include:
<ul>
<li>Over 97% of subjects participating in the Phe tolerance protocol of the APHENITY open-label extension study demonstrate the ability to liberalize their diet while on sepiapterin treatment, with a mean increase in protein intake of 126%.</li>
<li>66% of subjects participating in the Phe tolerance sub study reached or exceeded the age-adjusted recommended daily allowance of protein intake for an individual without PKU, while maintaining control of blood Phe levels.</li>
<li>Genetic variant analysis of subjects participating in the APHENITY study demonstrates that over 70% had a Genotype-Phenotype Value (GPV) consistent with classical PKU.</li>
</ul>
PTC submitted FDA regulatory approval applications in 2024 for sepiapterin for children and adults with PKU, with a target regulatory <a target="_blank" href="https://www.benzinga.com/pressreleases/25/02/n44024275/ptc-therapeutics-provides-corporate-update-and-reports-fourth-quarter-and-full-year-2024-financial" rel="noreferrer noopener">action date of&nbsp;July 29, 2025</a>.
PTC also submitted several additional marketing authorization applications outside the U.S. for sepiapterin in 2024. The CHMP opinion on sepiapterin MAA is expected in the second quarter of 2025. A regulatory decision in Japan is expected in the fourth quarter of 2025.
Price Action: PTCT stock is down 0.18% at $56.88 on Thursday&#8217;s last check.
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Photo: Shutterstock

PTC Therapeutics Reveals New Sepiapterin Data From Phase 3 Study In Patients With Inherited Disorder, FDA Decision Expected In July

—&nbsp;Results show 97% of phenylalanine (Phe) tolerance study participants were able to increase their dietary Phe intake with a mean increase of 126% —
— Genetic variant analysis of Phase 3 APHENITY trial demonstrates meaningful effect in classical subjects with non-BH4 responsive genotypes —
WARREN, N.J., March 20, 2025 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/PTCT#NASDAQ">PTCT</a>) today shared new data being presented from the Phase 3 APHENITY trial and subsequent open-label extension study at the 2025 American College of Medical Genetics and Genomics (ACMG) Annual Clinical Genetics Meeting. These data provide further evidence of the potential meaningful benefits of sepiapterin treatment for the full spectrum of phenylketonuria (PKU) patients, including significant diet liberalization.
Key data presented include:
<ul>
<li>Over 97% of subjects participating in the Phe tolerance protocol of the APHENITY open-label extension study demonstrate the ability to liberalize their diet while on sepiapterin treatment, with a mean increase in protein intake of 126%</li>
<li>66% of subjects participating in the Phe tolerance sub study reached or exceeded the age-adjusted recommended daily allowance of protein intake for an individual without PKU, while maintaining control of blood Phe levels</li>
<li>Genetic variant analysis of subjects participating in the APHENITY study demonstrates that over 70% had a Genotype-Phenotype Value (GPV) consistent with classical PKU</li>
</ul>

PTC Therapeutics Announces Phase 3 APHENITY Trial Results: Over 97% Of Subjects Participating In The Phe Tolerance Protocol Of The APHENITY Open-Label Extension Study Demonstrate The Ability To Liberalize Their Diet While On Sepiapterin Treatment, With A Mean Increase In Protein Intake Of 126%

JP Morgan analyst Eric Joseph maintains PTC Therapeutics (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/PTCT#NASDAQ">PTCT</a>) with a Overweight and raises the price target from $72 to $78.

JP Morgan Maintains Overweight on PTC Therapeutics, Raises Price Target to $78

Top Wall Street analysts changed their outlook on these top names. For a complete view of all analyst rating changes, including upgrades, downgrades and initiations, please see our <a href="https://www.benzinga.com/analyst-stock-ratings">analyst ratings page</a>.
<ul>
<li>Baird analyst David George upgraded the rating for Capital One Financial Corporation (NYSE:<a class="ticker" href="https://www.benzinga.com/stock/COF#NYSE">COF</a>) from Neutral to Outperform and raised the price target from $190 to $200. Capital One Finl shares closed at $163.87 on Monday. <a href="https://www.benzinga.com/quote/COF/analyst-ratings">See how other analysts view this stock</a>.</li>
<li>B of A Securities analyst Tazeen Ahmad upgraded PTC Therapeutics, Inc. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/PTCT#NASDAQ">PTCT</a>) from Underperform to Neutral and raised the price target from $41 to $55. PTC Therapeutics shares closed at $52.49 on Monday. <a href="https://www.benzinga.com/quote/PTCT/analyst-ratings">See how other analysts view this stock</a>.</li>
<li>Goldman Sachs analyst David Roman upgraded the rating for GE HealthCare Technologies Inc. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/GEHC#NASDAQ">GEHC</a>) from Neutral to Buy and boosted the price target from $85 to $100. GE HealthCare shares closed at $85.36 on Monday. <a href="https://www.benzinga.com/quote/GEHC/analyst-ratings">See how other analysts view this stock</a>.</li>
<li>Evercore ISI Group analyst Durgesh Chopra upgraded CenterPoint Energy, Inc. (NYSE:<a class="ticker" href="https://www.benzinga.com/stock/CNP#NYSE">CNP</a>) from In-Line to Outperform and raised the price target from $36 to $37. CenterPoint Energy shares closed at $34.43 on Monday. <a href="https://www.benzinga.com/quote/CNP/analyst-ratings">See how other analysts view this stock</a>.</li>
<li>Wolfe Research analyst Peter Supino upgraded the rating for Madison Square Garden Entertainment Corp. (NYSE:<a class="ticker" href="https://www.benzinga.com/stock/MSGE#NYSE">MSGE</a>) from Peer Perform to Outperform and announced a $46 price target. Madison Square Garden shares closed at $30.59 on Monday. <a href="https://www.benzinga.com/quote/MSGE/analyst-ratings">See how other analysts view this stock.</a></li>
</ul>
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Upgrades

Pre-Market Outlook

Trading Ideas

GEHC

MSGE

This GE HealthCare Analyst Turns Bullish; Here Are Top 5 Upgrades For Tuesday

B of A Securities analyst Tazeen Ahmad upgrades PTC Therapeutics (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/PTCT#NASDAQ">PTCT</a>) from Underperform to Neutral and raises the price target from $41 to $55.

B of A Securities Upgrades PTC Therapeutics to Neutral, Raises Price Target to $55

Scotiabank analyst Louise Chen initiates coverage on PTC Therapeutics (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/PTCT#NASDAQ">PTCT</a>) with a Sector Perform rating and announces Price Target of $55.

Initiation

Scotiabank Initiates Coverage On PTC Therapeutics with Sector Perform Rating, Announces Price Target of $55

Morgan Stanley analyst Jeffrey Hung assumes PTC Therapeutics (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/PTCT#NASDAQ">PTCT</a>) with a Overweight rating and raises Price Target to $70.

Morgan Stanley Assumes PTC Therapeutics at Overweight, Raises Price Target to $70

JP Morgan analyst Eric Joseph maintains PTC Therapeutics (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/PTCT#NASDAQ">PTCT</a>) with a Overweight and lowers the price target from $74 to $72.

JP Morgan Maintains Overweight on PTC Therapeutics, Lowers Price Target to $72

PTC Therapeutics (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/PTCT#NASDAQ">PTCT</a>) reported quarterly losses of $(0.85) per share which missed the analyst consensus estimate of $(0.66) by 28.79 percent. This is a 254.17 percent decrease over losses of $(0.24) per share from the same period last year. The company reported quarterly sales of $213.00 million which missed the analyst consensus estimate of $232.44 million by 8.36 percent. This is a 30.63 percent decrease over sales of $307.06 million the same period last year.

Earnings

Earnings Misses

PTC Therapeutics Q4 2024 Adj EPS $(0.85) Misses $(0.66) Estimate, Sales $213.00M Miss $232.44M Estimate

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Earnings Scheduled For February 27, 2025

The FDA Has Accepted PTC Therapeutics' New Drug Application For Vatiquinone For Children And Adults Living With Friedreich's Ataxia, Under Priority Review And Assigned A Prescription Drug User Fee Act Target Action Date Of August 19

RBC Capital analyst Brian Abrahams maintains PTC Therapeutics (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/PTCT#NASDAQ">PTCT</a>) with a Outperform and raises the price target from $60 to $63.

RBC Capital Maintains Outperform on PTC Therapeutics, Raises Price Target to $63

Citigroup analyst David Lebowitz maintains PTC Therapeutics (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/PTCT#NASDAQ">PTCT</a>) with a Sell and raises the price target from $32 to $45.

Citigroup Maintains Sell on PTC Therapeutics, Raises Price Target to $45

PTC Therapeutics shares are trading higher after Cantor Fitzgerald raised its price target on the stock from $76 to $113.

Cantor Fitzgerald analyst Kristen Kluska maintains PTC Therapeutics (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/PTCT#NASDAQ">PTCT</a>) with a Overweight and raises the price target from $76 to $113.

Cantor Fitzgerald Maintains Overweight on PTC Therapeutics, Raises Price Target to $113

Cantor Fitzgerald analyst Kristen Kluska maintains PTC Therapeutics (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/PTCT#NASDAQ">PTCT</a>) with a Overweight and lowers the price target from $80 to $76.

Cantor Fitzgerald Maintains Overweight on PTC Therapeutics, Lowers Price Target to $76

PTC Therapeutics Submits NDA To FDA For Vatiquinone As Potential First Approved Therapy For Friedreich Ataxia In Children And Adults, Marking Fourth FDA Application In 2024

Press Releases

PTC Therapeutics Provides Update on Commercial Performance and R&D Pipeline at 43rd Annual J.P. Morgan Healthcare Conference

PTC Therapeutics Reports Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)

Biotech Investing: How PRVs Can Unlock Major Opportunities

PTC Therapeutics to Host Conference Call to Discuss Fourth Quarter and Full Year 2024 Financial Results

PTC Therapeutics Announces FDA Acceptance and Priority Review for Vatiquinone NDA for the Treatment of Children and Adults with Friedreich's Ataxia

PTC Therapeutics to Participate in Upcoming Investor Conferences

PTC Therapeutics Provides Corporate Update and Reports Fourth Quarter and Full Year 2024 Financial Results

PTC Therapeutics Presents New Sepiapterin Data from Ongoing Studies

PTC Therapeutics Provides Regulatory Update on Translarna™ (ataluren) in Europe

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