PTC Therapeutics (NASDAQ:PTCT) Bolsa

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

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Earnings Scheduled For May 6, 2025

PTC Therapeutics, Inc. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/PTCT#NASDAQ">PTCT</a>) on Monday announced results from the Phase 2 PIVOT-HD study of PTC518 (votoplam) in Stage 2 and Stage 3 <a target="_blank" href="https://www.benzinga.com/general/health-care" rel="noreferrer noopener">Huntington&#8217;s disease (HD) patients</a>.
Huntington&#8217;s disease is&nbsp;a progressive neurodegenerative disorder caused by mutation of the&nbsp;huntingtin&nbsp;(HTT) gene, leading to the breakdown of brain cells.&nbsp;It is characterized by uncontrolled movements (chorea), cognitive decline, <a target="_blank" href="https://www.benzinga.com/pressreleases/25/05/n45190339/ptc518-pivot-hd-study-achieves-primary-endpoint" rel="noreferrer noopener">and psychiatric disturbances</a>.
The study met its primary endpoint of reduction in blood Huntingtin (HTT) protein levels (p&lt;0.0001) at Week 12 and favorable safety and tolerability. In addition, the 12-month data from the Stage 2 patients are consistent with the previously reported dose-dependent lowering of HTT protein and dose-dependent trends across clinical scales.
Also Read: <a target="_blank" href="https://www.benzinga.com/general/biotech/25/03/44538151/european-commission-declines-to-renew-marketing-authorization-of-translarna-duchenne-muscular-disorder" rel="noreferrer noopener">European Commission Declines To Renew Marketing Authorization Of Translarna Duchenne Muscular Disorder</a>
&#8220;We look forward to discussions on the next development and regulatory steps, including the potential for accelerated approval as we work to potentially bring the first disease-modifying therapy&#8230;,&#8221; said&nbsp;Matthew B. Klein, M.D., Chief Executive Officer, PTC Therapeutics.
Results from the full 12-month cohort demonstrate dose-dependent lowering in blood HTT levels, with 23% at the 5mg dose level for both Stage 2 and 3 patients and 39% and 36% at the 10mg dose level for Stage 2 and 3 patients, respectively. For Stage 2 patients, there were dose-dependent trends of benefit on clinical scales, including the Composite Unified Huntington&#8217;s Disease Rating Scale (cUHDRS) and Total Motor Score (TMS) subscale.
For Stage 3 patients, trends favored the 5mg dose group relative to placebo but not the 10mg dose group, suggesting that treatment effect may differ in Stage 3 patients relative to Stage 2 patients. It seems investors are reacting to this as the stock is trading almost 20% lower during the premarket session.
PTC518 showed a favorable safety and tolerability profile for all dose levels and disease stages with no treatment-related serious adverse events or neurofilament light chain protein&nbsp;(NfL) spikes.
In addition, 24-month treatment data from the patients on whom data were shared last year (N=21) demonstrate signals of dose-dependent trends on the cUHDRS, Total Function Capacity (TFC), and Symbol Digit Modalities Test (SDMT) subscales when compared to a propensity-matched natural history cohort from the ENROLL-HD Registry.
At Month 24, there was also a dose-dependent lowering of plasma NfL from a baseline of -8.9% (nominal p=0.12) for the 5mg dose level and -14% (nominal p=0.03) for the 10mg dose level.
In April, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) issued a positive opinion on the marketing authorization application for Sephience (sepiapterin) for phenylketonuria (PKU). The opinion includes a broad label that includes <a target="_blank" href="https://www.benzinga.com/pressreleases/25/04/n45007096/ptc-therapeutics-receives-positive-chmp-opinion-for-sephience-sepiapterin-for-the-treatment-of-chi" rel="noreferrer noopener">all ages and disease severities</a>.
Price Action: PTCT stock fell 18.60% to $40.65&nbsp;on Monday.
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Why Did PTC Therapeutics Stock Fall On Monday Despite Mid-Stage Study For Huntington's Hits Primary Goal?

<h3>Gainers</h3>
<ul>
<li>NuCana (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/NCNA#NASDAQ">NCNA</a>) stock moved upwards by 52.8% to $0.7 during Monday's regular session. The company's market cap stands at $4.2 million. </li>
<li>Immutep (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/IMMP#NASDAQ">IMMP</a>) stock moved upwards by 35.6% to $2.32. The market value of their outstanding shares is at $337.2 million. </li>
<li>INVO Fertility (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/IVF#NASDAQ">IVF</a>) stock moved upwards by 33.33% to $2.4. The company's market cap stands at $11.8 million. </li>
<li>Regencell Bioscience (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/RGC#NASDAQ">RGC</a>) shares moved upwards by 31.91% to $80.8. The company's market cap stands at $1.0 billion. </li>
<li>Alterity Therapeutics (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/ATHE#NASDAQ">ATHE</a>) shares rose 23.54% to $4.25. The company's market cap stands at $62.9 million. </li>
<li>Cyclacel Pharmaceuticals (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/CYCC#NASDAQ">CYCC</a>) shares increased by 23.2% to $0.31. The market value of their outstanding shares is at $4.3 million. </li>
</ul>
<h3>Losers</h3>
<ul>
<li>Integra Lifesciences (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/IART#NASDAQ">IART</a>) stock decreased by 26.4% to $12.4 during Monday's regular session. The company's market cap stands at $957.9 million. As per the news, the Q1 earnings report came out today. </li>
<li>Nexalin Technology (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/NXL#NASDAQ">NXL</a>) shares decreased by 23.47% to $1.37. The market value of their outstanding shares is at $18.2 million. </li>
<li>Impact BioMedical (AMEX:<a class="ticker" href="https://www.benzinga.com/stock/IBO#AMEX">IBO</a>) shares declined by 22.46% to $0.58. The market value of their outstanding shares is at $6.6 million. </li>
<li>AirSculpt Technologies (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/AIRS#NASDAQ">AIRS</a>) stock fell 21.2% to $2.38. The market value of their outstanding shares is at $139.4 million. As per the press release, Q1 earnings came out yesterday. </li>
<li>ImmunityBio (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/IBRX#NASDAQ">IBRX</a>) stock declined by 19.49% to $2.04. The market value of their outstanding shares is at $1.8 billion. </li>
<li>PTC Therapeutics (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/PTCT#NASDAQ">PTCT</a>) stock declined by 17.48% to $41.22. The market value of their outstanding shares is at $3.2 billion. 
See Also: <a href="http://www.benzinga.com/money/best-healthcare-stocks/" title="www.benzinga.com/money/best-healthcare-stocks/">www.benzinga.com/money/best-healthcare-stocks/</a></li>
</ul>
This article was generated by Benzinga's automated content engine and reviewed by an editor.

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Alterity Therapeutics Limited - American Depositary Shares

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12 Health Care Stocks Moving In Monday's Intraday Session

U.S. stocks traded mixed midway through trading, with the Dow Jones index gaining around 0.2% on Monday.
The Dow traded up 0.21% to 41,406.25 while the NASDAQ fell 0.41% to 17,903.64. The S&amp;P 500 also fell, dropping, 0.26% to 5,671.90.
Check This Out: <a href="https://www.benzinga.com/markets/25/05/45192607/jim-cramer-applied-materials-is-an-excellent-company-but-likes-this-tech-stock-more">Jim Cramer Says Applied Materials Is An &#8216;Excellent&#8217; Company, But Likes This Tech Stock More</a>
Leading and Lagging Sectors Industrials shares rose by 0.2% on Monday.
In trading on Monday, energy stocks dipped by 1.9%.
Top Headline
The ISM services PMI rose to 51.6 in April versus a nine-month low level of 50.8 in the previous month March and topping market estimates of 50.6.
Equities Trading UP &nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp; &nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;&nbsp;
<ul>
<li>Hyperscale Data, Inc. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/GPUS#NASDAQ">GPUS</a>) shares shot up 202% to $4.14 after the company issued first-quarter and FY25 sales guidance.</li>
<li>Shares of Skechers U.S.A., Inc. (NYSE:<a class="ticker" href="https://www.benzinga.com/stock/SKX#NYSE">SKX</a>) got a boost, surging 25% to $61.70 after the company agreed to be acquired by 3G Capital for $63 per share.</li>
<li>BioCryst Pharmaceuticals, Inc. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/BCRX#NASDAQ">BCRX</a>) shares were also up, gaining 12% to $10.01 after the company reported a first-quarter sales beat.</li>
</ul>
Equities Trading DOWN
<ul>
<li>Integra LifeSciences Holdings Corporation (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/IART#NASDAQ">IART</a>) shares dropped 29% to $11.94 after the company reported a first-quarter adjusted EPS miss and cut its FY25 adjusted EPS guidance.</li>
<li>Shares of PTC Therapeutics, Inc. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/PTCT#NASDAQ">PTCT</a>) were down 18% to $40.87. The company announced results from the Phase 2 PIVOT-HD study of PTC518 in Stage 2 and Stage 3 Huntington&#8217;s disease patients.</li>
<li>TG Therapeutics, Inc. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/TGTX#NASDAQ">TGTX</a>) was down, falling 16% to $36.53 after the company reported worse-than-expected first-quarter EPS results.</li>
</ul>
Commodities In commodity news, oil traded down 2.2% to $57.00 while gold traded up 2.5% at $3,323.40.
Silver traded up 0.6% to $32.455 on Monday, while copper rose 0.3% to $4.6875.
Euro zone
European shares were mostly higher today. The eurozone's STOXX 600 gained 0.06%, while Spain's IBEX 35 Index gained 0.40%. Germany's DAX 40 rose 0.87% and France's CAC 40 fell 0.58% during the session.
Asia Pacific Markets
Asian markets closed higher on Monday, with India's BSE Sensex rising 0.37%.
Economics
<ul>
<li>The S&amp;P Global services PMI was revised lower to 50.8 in April 2025 compared to a preliminary reading of 51.4.</li>
<li>The ISM services PMI rose to 51.6 in April versus a nine-month low level of 50.8 in the previous month March and topping market estimates of 50.6.</li>
</ul>
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Photo via Shutterstock

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Crude Oil Falls Over 2%; ISM Services PMI Rises In April

WIIM

PTC Therapeutics shares are trading lower. The company announced results from the Phase 2 PIVOT-HD study of PTC518 in Stage 2 and Stage 3 Huntington's disease patients.

Study met primary endpoint with dose-dependent blood HTT protein lowering at Week 12 -
- Favorable dose-dependent trends across clinical scales in Stage 2 patients at Month 12 -
- Signals of dose-dependent clinical benefit relative to matched natural history cohort as well as dose-dependent lowering of&nbsp;NfL in Stage 2 patients at Month 24 -
- Continued favorable safety and tolerability profile with no treatment-related NfL spikes -
- PTC will host a conference call on May 5, 2025, at 8:00 am ET-
&nbsp;PTC Therapeutics, Inc. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/PTCT#NASDAQ">PTCT</a>) today announced results from the Phase 2 PIVOT-HD study of PTC518 (votoplam) in Stage 2 and Stage 3 Huntington's disease (HD) patients. The study met its primary endpoint of reduction in blood Huntingtin (HTT) protein levels (p&lt;0.0001) at Week 12 and favorable safety and tolerability. In addition, the 12-month data from the Stage 2 patients are consistent with the previously reported dose-dependent lowering of HTT protein and dose-dependent trends across clinical scales.
"These PIVOT-HD results confirm that PTC518 lowers Huntingtin protein and shows early signals of clinical benefit with a favorable safety profile," said Matthew B. Klein, M.D., Chief Executive Officer, PTC Therapeutics. "In addition, at 24 months, we observed favorable dose-dependent trends on the cUHDRS and the TFC and SDMT subscales relative to natural history as well as dose-dependent lowering of neurofilament light chain protein. We look forward to discussions on the next development and regulatory steps including the potential for accelerated approval as we work to potentially bring the first disease-modifying therapy to those affected by Huntington's disease."

Ptc Therapeutics Announces Results From The Phase 2 Pivot-Hd Study of PTC518 In Stage 2 And Stage 3 Huntington's Disease Patients Study Met Its Primary Endpoint of Reduction in Blood Huntingtin Protein Levels At Week 12 And Favorable Safety and Tolerability

- Label includes full spectrum of PKU patients -
- European launch preparations underway -
WARREN, N.J., April 25, 2025 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/PTCT#NASDAQ">PTCT</a>) announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has issued a positive opinion on the marketing authorization application for Sephience&#x2122; (sepiapterin) for the treatment of children and adults living with phenylketonuria (PKU). The opinion includes a broad label inclusive of all ages and disease severities.

PTC Therapeutics: CHMP Of EMA Issues Positive Opinion On Marketing Authorization Application For Sephience For Treatment Of Children And Adults Living With Phenylketonuria

This whale alert can help traders discover the next big trading opportunities.
Whales are entities with large sums of money and we track their transactions here at Benzinga on our options activity scanner.
Traders often look for circumstances when the market estimation of an option diverges away from its normal worth. Abnormal amounts of trading activity could push option prices to hyperbolic or underperforming levels. 
Here's the list of options activity happening in today's session: 
<table>
<thead>
<tr>
<th>Symbol</th>
<th>PUT/CALL</th>
<th>Trade Type</th>
<th>Sentiment</th>
<th>Exp. Date</th>
<th>Strike Price</th>
<th>Total Trade Price</th>
<th>Open Interest</th>
<th align="right">Volume</th>
</tr>
</thead>
<tbody>
<tr>
<td>CRL</td>
<td>CALL</td>
<td>SWEEP</td>
<td>BEARISH</td>
<td>05/16/25</td>
<td>$105.00</td>
<td>$35.3K</td>
<td>866</td>
<td align="right">366</td>
</tr>
<tr>
<td>THC</td>
<td>PUT</td>
<td>SWEEP</td>
<td>NEUTRAL</td>
<td>05/16/25</td>
<td>$115.00</td>
<td>$60.0K</td>
<td>332</td>
<td align="right">193</td>
</tr>
<tr>
<td>VSTM</td>
<td>CALL</td>
<td>TRADE</td>
<td>NEUTRAL</td>
<td>06/20/25</td>
<td>$6.00</td>
<td>$25.6K</td>
<td>5.2K</td>
<td align="right">152</td>
</tr>
<tr>
<td>ISRG</td>
<td>CALL</td>
<td>TRADE</td>
<td>BEARISH</td>
<td>07/18/25</td>
<td>$445.00</td>
<td>$119.4K</td>
<td>1</td>
<td align="right">98</td>
</tr>
<tr>
<td>PTCT</td>
<td>CALL</td>
<td>SWEEP</td>
<td>BEARISH</td>
<td>12/19/25</td>
<td>$49.00</td>
<td>$55.5K</td>
<td>2</td>
<td align="right">67</td>
</tr>
<tr>
<td>SYK</td>
<td>CALL</td>
<td>SWEEP</td>
<td>BEARISH</td>
<td>09/19/25</td>
<td>$390.00</td>
<td>$27.3K</td>
<td>137</td>
<td align="right">46</td>
</tr>
<tr>
<td>JNJ</td>
<td>CALL</td>
<td>SWEEP</td>
<td>BEARISH</td>
<td>06/20/25</td>
<td>$135.00</td>
<td>$27.2K</td>
<td>1.7K</td>
<td align="right">26</td>
</tr>
<tr>
<td>LLY</td>
<td>CALL</td>
<td>TRADE</td>
<td>BULLISH</td>
<td>08/15/25</td>
<td>$700.00</td>
<td>$76.8K</td>
<td>53</td>
<td align="right">10</td>
</tr>
<tr>
<td>TMO</td>
<td>PUT</td>
<td>TRADE</td>
<td>BEARISH</td>
<td>09/19/25</td>
<td>$530.00</td>
<td>$41.0K</td>
<td>119</td>
<td align="right">9</td>
</tr>
</tbody>
</table>

<h2>Explanation</h2>
These bullet-by-bullet explanations have been constructed using the accompanying table. 
• Regarding CRL (NYSE:<a class="ticker" href="https://www.benzinga.com/stock/CRL#NYSE">CRL</a>), we observe a call option sweep with bearish sentiment. It expires in 24 day(s) on May 16, 2025. Parties traded 58 contract(s) at a $105.00 strike. This particular call needed to be split into 10 different trades to become filled. The total cost received by the writing party (or parties) was $35.3K, with a price of $610.0 per contract. There were 866 open contracts at this strike prior to today, and today 366 contract(s) were bought and sold. 
• Regarding THC (NYSE:<a class="ticker" href="https://www.benzinga.com/stock/THC#NYSE">THC</a>), we observe a put option sweep with neutral sentiment. It expires in 24 day(s) on May 16, 2025. Parties traded 70 contract(s) at a $115.00 strike. This particular put needed to be split into 10 different trades to become filled. The total cost received by the writing party (or parties) was $60.0K, with a price of $860.0 per contract. There were 332 open contracts at this strike prior to today, and today 193 contract(s) were bought and sold. 
• Regarding VSTM (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/VSTM#NASDAQ">VSTM</a>), we observe a call option trade with neutral sentiment. It expires in 59 day(s) on June 20, 2025. Parties traded 151 contract(s) at a $6.00 strike. The total cost received by the writing party (or parties) was $25.6K, with a price of $170.0 per contract. There were 5217 open contracts at this strike prior to today, and today 152 contract(s) were bought and sold. 
• Regarding ISRG (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/ISRG#NASDAQ">ISRG</a>), we observe a call option trade with bearish sentiment. It expires in 87 day(s) on July 18, 2025. Parties traded 20 contract(s) at a $445.00 strike. The total cost received by the writing party (or parties) was $119.4K, with a price of $5972.0 per contract. There were 1 open contracts at this strike prior to today, and today 98 contract(s) were bought and sold. 
• For PTCT (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/PTCT#NASDAQ">PTCT</a>), we notice a call option sweep that happens to be bearish, expiring in 241 day(s) on December 19, 2025. This event was a transfer of 50 contract(s) at a $49.00 strike. This particular call needed to be split into 22 different trades to become filled. The total cost received by the writing party (or parties) was $55.5K, with a price of $1110.0 per contract. There were 2 open contracts at this strike prior to today, and today 67 contract(s) were bought and sold. 
• For SYK (NYSE:<a class="ticker" href="https://www.benzinga.com/stock/SYK#NYSE">SYK</a>), we notice a call option sweep that happens to be bearish, expiring in 150 day(s) on September 19, 2025. This event was a transfer of 30 contract(s) at a $390.00 strike. This particular call needed to be split into 9 different trades to become filled. The total cost received by the writing party (or parties) was $27.3K, with a price of $910.0 per contract. There were 137 open contracts at this strike prior to today, and today 46 contract(s) were bought and sold. 
• For JNJ (NYSE:<a class="ticker" href="https://www.benzinga.com/stock/JNJ#NYSE">JNJ</a>), we notice a call option sweep that happens to be bearish, expiring in 59 day(s) on June 20, 2025. This event was a transfer of 12 contract(s) at a $135.00 strike. This particular call needed to be split into 3 different trades to become filled. The total cost received by the writing party (or parties) was $27.2K, with a price of $2270.0 per contract. There were 1737 open contracts at this strike prior to today, and today 26 contract(s) were bought and sold. 
• For LLY (NYSE:<a class="ticker" href="https://www.benzinga.com/stock/LLY#NYSE">LLY</a>), we notice a call option trade that happens to be bullish, expiring in 115 day(s) on August 15, 2025. This event was a transfer of 5 contract(s) at a $700.00 strike. The total cost received by the writing party (or parties) was $76.8K, with a price of $15360.0 per contract. There were 53 open contracts at this strike prior to today, and today 10 contract(s) were bought and sold. 
• Regarding TMO (NYSE:<a class="ticker" href="https://www.benzinga.com/stock/TMO#NYSE">TMO</a>), we observe a put option trade with bearish sentiment. It expires in 150 day(s) on September 19, 2025. Parties traded 4 contract(s) at a $530.00 strike. The total cost received by the writing party (or parties) was $41.0K, with a price of $10250.0 per contract. There were 119 open contracts at this strike prior to today, and today 9 contract(s) were bought and sold. 
Options Alert Terminology 
 - Call Contracts: The right to buy shares as indicated in the contract. 
 - Put Contracts: The right to sell shares as indicated in the contract. 
 - Expiration Date: When the contract expires. One must act on the contract by this date if one wants to use it. 
 - Premium/Option Price: The price of the contract. 
For more information, visit our <a href="https://pro.benzinga.com/blog/how-to-understand-option-alerts/">Guide to Understanding Options Alerts</a> or read <a href="https://www.benzinga.com/markets/options">more news on unusual options activity</a>. 
This article was generated by Benzinga's automated content engine and reviewed by an editor.

Options

ISRG

VSTM

9 Health Care Stocks With Whale Alerts In Today's Session

RBC Capital analyst Brian Abrahams reiterates PTC Therapeutics (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/PTCT#NASDAQ">PTCT</a>) with a Outperform and maintains $65 price target.

Price Target

Reiteration

Analyst Ratings

RBC Capital Reiterates Outperform on PTC Therapeutics, Maintains $65 Price Target

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.

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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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>
Considering buying GEHC stock? Here&#8217;s what analysts think: 
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Upgrades

Pre-Market Outlook

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

Press Releases

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

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

PTC Therapeutics to Host Conference Call to Discuss First Quarter 2025 Financial Results

PTC Therapeutics to Participate at Upcoming Investor Conferences

PTC Therapeutics Receives Positive CHMP Opinion for Sephience™ (sepiapterin) for the Treatment of Children and Adults Living with Phenylketonuria (PKU)

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