Últimas noticias sobre PTC Therapeutics (NASDAQ:PTCT) Bolsa

PTC Therapeutics

PTC Therapeutics Inc is a biopharmaceutical company focused on the discovery, development, and commercialization of orally administered, small-molecule therapeutics. Its target area is RNA biology post-transcriptional control processes of the events occurring in a cell when an RNA messenger is copied from DNA during the transcription process. The discovered products address multiple therapeutic areas, including rare disorders, such as nonsense mutations in Duchenne muscular dystrophy and oncology.

PTC Therapeutics Inc

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.

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PTCT

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

Morgan Stanley analyst Jeffrey Hung upgrades PTC Therapeutics (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/PTCT#NASDAQ">PTCT</a>) from Equal-Weight to Overweight and raises the price target from $45 to $67.

Upgrades

Morgan Stanley Upgrades PTC Therapeutics to Overweight, Raises Price Target to $67

On Friday, Novartis AG (NYSE:<a class="ticker" href="https://www.benzinga.com/stock/NVS#NYSE">NVS</a>) revealed topline results from the APPULSE-PNH Phase 3B study of Fabhalta&nbsp;(iptacopan) in adult patients with paroxysmal nocturnal hemoglobinuria (PNH) who were switched from anti-C5 therapies (Hb ≥10g/dL following treatment with eculizumab or ravulizumab).
Ultomiris (ravulizumab) and Soliris&nbsp;(eculizumab) are commonly used anti-C5 therapies, and they are made by AstraZeneca Plc (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/AZN#NASDAQ">AZN</a>).
Also Read: <a target="_blank" href="https://www.benzinga.com/analyst-ratings/analyst-color/24/10/41634528/positive-outlook-for-novartis-as-company-capitalizes-on-growing-treatment-opportuni" rel="noreferrer noopener">Positive Outlook For Novartis As Company Capitalizes On Growing Treatment Opportunities, Analyst Highlights</a>
PNH is a rare complement-mediated blood disorder. People with PNH have an acquired mutation in some of their hematopoietic stem cells, which are located in the bone marrow and can grow and develop into red blood cells, white blood cells, and platelets.
This mutation <a target="_blank" href="https://www.novartis.com/news/media-releases/new-phase-iiib-data-shows-novartis-fabhalta-improved-hemoglobin-levels-adult-patients-paroxysmal-nocturnal-hemoglobinuria-who-switched-from-anti-c5-therapy" rel="noreferrer noopener">causes</a> them to produce RBCs that are susceptible to premature destruction by the complement system.
After 24 weeks of treatment with Fabhalta, the average hemoglobin (Hb) level improved compared to baseline. The study&#8217;s safety profile of Fabhalta monotherapy was consistent with previously reported data.
Data will be presented at an upcoming medical meeting in 2025.
The FDA recently granted accelerated approval for Fabhalta to reduce proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk of rapid disease progression, and development is ongoing in multiple complement-mediated diseases.
Last month, Novartis raised its mid-term sales guidance, increasing its compound annual growth rate (CAGR) target to 6% from 5% <a target="_blank" href="https://www.benzinga.com/general/biotech/24/11/42115127/novartis-confident-in-growth-as-it-elevates-midterm-sales-forecast-ceo-narasimhan-highlights-robust-pipeline-supporting-long-term-strategy" rel="noreferrer noopener">for 2023-2028</a>.
The upgrade reflects performance from current growth drivers and anticipated launches, many of which are expected to retain U.S. exclusivity into the 2030s.
Earlier this week, PTC Therapeutics (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/PTCT#NASDAQ">PTCT</a>) signed an exclusive global license and collaboration agreement with&nbsp;Novartis for its PTC518 Huntington&#8217;s disease program, which&nbsp;<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">includes related molecules</a>.
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.
<h2 class="wp-block-heading">Is Novartis A Good Stock To Buy?</h2>
Wall Street analysts view Novartis on the whole as a Neutral, given the history of coverage over the past three months. Graham Perry from B of A Securities in Novartis is the most bearish, expecting a 3.7% fall in the stock in the coming year.
But looking at how the market as a whole thinks of the stock, you can reference historical price action for views on whether investors feel strongly about the stock one way or another. In the past 3 months, Novartis fell 12.4%, which indicates that opinion soured on the business and how attractive it is to own based on either its stock price, or underlying fundamentals, like revenue, which rose 8.84% over the past year.
A complete overview of how Wall Street views individual stocks is available <a href="https://www.benzinga.com/analyst-stock-ratings">here</a>, while real time updates on the latest analyst actions will be delivered via <a href="https://pro.benzinga.com">Benzinga PRO</a>. Try it for free.
Price Action: At the last check on Friday, NVS stock was up 0.18% to $102.34.
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Photo by Taljat David via Shutterstock

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Novartis Touts Encouraging Data From Late-Stage Study For Fabhalta In Rare Type Of Blood Disorder

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 $26 to $32.

Citigroup Maintains Sell on PTC Therapeutics, Raises Price Target to $32

Goldman Sachs analyst Paul Choi 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 $42.

Goldman Sachs Maintains Sell on PTC Therapeutics, Raises Price Target to $42

UBS analyst Colin Bristow maintains PTC Therapeutics (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/PTCT#NASDAQ">PTCT</a>) with a Buy and raises the price target from $47 to $71.

UBS Maintains Buy on PTC Therapeutics, Raises Price Target to $71

Top Wall Street analysts changed their outlook on these top names. For a complete view of all analyst rating changes, including upgrades and downgrades, please see our <a href="https://www.benzinga.com/analyst-stock-ratings">analyst ratings page</a>.
<ul>
<li>Keefe, Bruyette &amp; Woods raised the price target for Bank of America Corporation (NYSE:<a class="ticker" href="https://www.benzinga.com/stock/BAC#NYSE">BAC</a>) from $50 to $57. Keefe, Bruyette &amp; Woods analyst David Konrad maintained an Outperform rating. Bank of America shares closed at $47.04 on Monday. <a href="https://www.benzinga.com/quote/BAC/analyst-ratings">See how other analysts view this stock.</a></li>
<li>Morgan Stanley cut the price target for Zscaler (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/ZS#NASDAQ">ZS</a>) from $215 to $205. Morgan Stanley analyst Hamza Fodderwala maintained an Equal-Weight rating. Kohl&#8217;s shares closed at $208.51 on Monday. <a href="https://www.benzinga.com/quote/ZS/analyst-ratings">See how other analysts view this stock</a>.</li>
<li>Piper Sandler boosted Fastly, Inc. (NNY FSLY) price target from $8 to $10. Piper Sandler analyst James Fish maintained a Neutral rating. Fastly shares settled at $9.87 on Monday. <a href="https://www.benzinga.com/quote/FSLY/analyst-ratings">See how other analysts view this stock</a>.</li>
<li>Deutsche Bank raised UnitedHealth Group Incorporated (NYSE:<a class="ticker" href="https://www.benzinga.com/stock/UNH#NYSE">UNH</a>) price target from $595 to $625. Deutsche Bank analyst George Hill maintained a Buy rating. UnitedHealth shares closed at $608.52 on Monday. <a href="https://www.benzinga.com/quote/UNH/analyst-ratings">See how other analysts view this stock.</a></li>
<li>Citigroup cut Western Digital Corporation (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/WDC#NASDAQ">WDC</a>) price target from $87 to $85. Citigroup analyst Asiya Merchant maintained a Buy rating. Western Digital shares closed at $73.43 on Monday.  <a href="https://www.benzinga.com/quote/WDC/analyst-ratings">See how other analysts view this stock</a>.</li>
<li>Barclays boosted PTC Therapeutics, Inc. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/PTCT#NASDAQ">PTCT</a>) price target from $45 to $56. Barclays analyst Gena Wang maintained an Equal-Weight rating on the stock. PTC Therapeutics shares closed at $52.07 on Monday. See how other analysts view this stock.</li>
<li>JP Morgan increased the price target for FedEx Corporation (NYSE:<a class="ticker" href="https://www.benzinga.com/stock/FDX#NYSE">FDX</a>) from $350 to $366. JP Morgan analyst Brian Ossenbeck maintained an Overweight rating. FedEx shares settled at $296.69 on Monday. <a href="https://www.benzinga.com/quote/FDX/analyst-ratings">See how other analysts view this stock</a>.</li>
<li>DA Davidson boosted Salesforce, Inc. (NYSE:<a class="ticker" href="https://www.benzinga.com/stock/CRM#NYSE">CRM</a>) price target from $250 to $300. DA Davidson analyst Gil Luria maintained a Neutral rating. Salesforce shares closed at $331.01 on Monday. <a href="https://www.benzinga.com/quote/MSFT/analyst-ratings">See how other analysts view this stock</a>.</li>
<li>Wells Fargo increased the price target for Webster Financial Corporation (NYSE:<a class="ticker" href="https://www.benzinga.com/stock/WBS#NYSE">WBS</a>) from $60 to $70. Wells Fargo analyst Timur Braziler maintained an Equal-Weight rating. Webster Financial shares closed at $60.94 on Monday. <a href="https://www.benzinga.com/quote/WBS/analyst-ratings">See how other analysts view this stock</a>.</li>
<li>Jefferies raised The Kroger Co. (NYSE:<a class="ticker" href="https://www.benzinga.com/stock/KR#NYSE">KR</a>) price target from $54 to $73. Jefferies analyst Rob Dickerson upgraded the stock from Hold to Buy. Kroger shares closed at $60.15 on Monday. <a href="https://www.benzinga.com/quote/KR/analyst-ratings">See how other analysts view this stock</a>.</li>
</ul>
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</ul>

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Bank of America To Rally More Than 21%? Here Are 10 Top Analyst Forecasts For Tuesday

Baird analyst Joel Beatty 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 $52 to $70.

Baird Maintains Outperform on PTC Therapeutics, Raises Price Target to $70

Barclays analyst Gena Wang maintains PTC Therapeutics (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/PTCT#NASDAQ">PTCT</a>) with a Equal-Weight and raises the price target from $45 to $56.

Barclays Maintains Equal-Weight on PTC Therapeutics, Raises Price Target to $56

On Monday, PTC Therapeutics, Inc.&nbsp;(NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/PTCT#NASDAQ">PTCT</a>) signed an exclusive global license and collaboration agreement with Novartis AG (NYSE:<a class="ticker" href="https://www.benzinga.com/stock/NVS#NYSE">NVS</a>) for its PTC518 Huntington&#8217;s disease program, which <a target="_blank" href="https://www.benzinga.com/pressreleases/24/12/n42262890/ptc-therapeutics-enters-into-a-global-license-and-collaboration-agreement-with-novartis-for-ptc518" rel="noreferrer noopener">includes related molecules</a>.
Under the agreement, PTC will receive an upfront payment of&nbsp;$1.0 billion, up to&nbsp;$1.9 billion&nbsp;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: <a target="_blank" href="https://www.benzinga.com/general/biotech/24/11/41984686/fda-grants-conditional-approval-for-ptc-therapeutics-gene-therapy-as-first-ever-with-direct-administration-in-brain" rel="noreferrer noopener">FDA Grants Conditional Approval For PTC Therapeutics&#8217; Gene Therapy As First Ever With Direct Administration In Brain</a>
PTC518 was discovered from PTC&#8217;s validated splicing platform and is currently being studied in the ongoing <a target="_blank" href="https://www.benzinga.com/general/biotech/24/06/39413593/ptc-therapeutics-says-fda-lifts-clinical-hold-on-investigational-study-in-patients-with-rare-weak" rel="noreferrer noopener">Phase 2 PIVOT-HD trial</a>.
Interim results reported in&nbsp;June 2024&nbsp;showed that PTC518 treatment resulted in durable, dose-dependent reduction in blood and cerebrospinal fluid mutant Huntingtin protein levels as well as early signals of dose-dependent benefit on key clinical measurements at 12 months.
PTC518 continues to demonstrate a favorable safety and tolerability profile.
Novartis will assume responsibility for PTC518&#8217;s development, manufacturing, and commercialization following the completion of the ongoing placebo-controlled portion of PIVOT-HD, which is expected to occur in the first half of 2025.&nbsp;
The companies will share U.S. profits and losses on a 40/60 basis (40% PTC and 60% Novartis). The parties anticipate the agreement will close in the first quarter of 2025.
Last week,&nbsp;PTC Therapeutics&#8217; global Phase 2 placebo-controlled CardinALS study of Utreloxastat in amyotrophic&nbsp;lateral sclerosis patients did not meet its primary endpoint of slowing disease progression on the composite ALSFRS-R <a target="_blank" href="https://www.benzinga.com/24/11/42211998/ptc-therapeutics-ends-utreloxastat-development-after-rare-neurodegenerative-disease-trial-misses-goal-analyst-maintains-outperform-with-focus-on-sepiapterin-huntingtons-programs" rel="noreferrer noopener">and mortality analysis</a>.
Price Action: PTCT stock is up 17.1% at $51.38 at last check Monday.
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</ul>
Photo via Shutterstock.

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PTC Therapeutics Inks Licensing Pact With Novartis Worth Up To ~$3 Billion For Huntington's Program

U.S. stocks were mixed, with the Dow Jones index falling more than 100 points on Monday.
Shares of XPeng Inc. (NYSE:<a class="ticker" href="https://www.benzinga.com/stock/XPEV#NYSE">XPEV</a>) rose sharply <a href="https://www.benzinga.com/24/12/42260165/li-auto-xpeng-nio-heres-how-november-deliveries-of-chinese-ev-startups-stack-up">during Monday's session</a>.
The company delivered 30,895 vehicles last month, marking an increase of 54% year-over-year and 29% from October.
The Guangzhou-based EV startup said that the company&#8217;s deliveries of the XPeng Mona M03 hatchback coupe alone exceeded 10,000 units for the third consecutive month since launch. Deliveries of the XPeng P7+ sedan, which the company launched last month, also exceeded 7,000 units.
XPeng shares jumped 6.4% to $12.82 on Monday.
Here are some other big stocks recording gains in today's session.
<ul>
<li>NovoCure Limited (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/NVCR#NASDAQ">NVCR</a>) shares gained 29.9% to $26.04 after the company announced its Phase 3 PANOVA-Trial met its primary endpoint and demonstrated a statistically significant improvement in its median overall survival rate.</li>
<li>Super Micro Computer, Inc. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/SMCI#NASDAQ">SMCI</a>) surged 22% to $39.83 after the company announced it completed a review by an independent special committee and stated EY&#8217;s conclusions were not supported by findings.</li>
<li>Fastly, Inc. (NYSE:<a class="ticker" href="https://www.benzinga.com/stock/FSLY#NYSE">FSLY</a>) jumped 17% to $9.92.</li>
<li>Nebius Group N.V. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/NBIS#NASDAQ">NBIS</a>) shares gained 15.5% to $25.40 after the company announced it entered a $700 million private placement financing agreement with institutional investors.</li>
<li>Enovix Corporation (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/ENVX#NASDAQ">ENVX</a>) gained 15.1% to $10.65.</li>
<li>Zeta Global Holdings Corp. (NYSE:<a class="ticker" href="https://www.benzinga.com/stock/ZETA#NYSE">ZETA</a>) jumped 14.6% to $24.41.</li>
<li>PTC Therapeutics, Inc. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/PTCT#NASDAQ">PTCT</a>) surged 14.3% to $50.15 after the company announced it signed a global licensee and collaboration agreement with Novartis for its PTC518 Huntington&#8217;s Disease program.</li>
<li>Dana Incorporated (NYSE:<a class="ticker" href="https://www.benzinga.com/stock/DAN#NYSE">DAN</a>) gained 14% to $11.39 after Barclays upgraded the stock from Equal-Weight to Overweight and raised its price target from $12 to $18.</li>
<li>MINISO Group Holding Limited (NYSE:<a class="ticker" href="https://www.benzinga.com/stock/MNSO#NYSE">MNSO</a>) gained 12.9% to $22.60. The company, on Friday, posted first-quarter results.</li>
<li>Recursion Pharmaceuticals, Inc. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/RXRX#NASDAQ">RXRX</a>) climbed 11.3% to $7.87.</li>
<li>Akso Health Group (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/AHG#NASDAQ">AHG</a>) rose 8.8% to $0.9000.</li>
<li>Abercrombie &amp; Fitch Co. (NYSE:<a class="ticker" href="https://www.benzinga.com/stock/ANF#NYSE">ANF</a>) gained 8.2% to $161.94.</li>
<li>Merus N.V. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/MRUS#NASDAQ">MRUS</a>) gained 8% to $48.43 after the company announced it entered into an agreement to exclusively license the right to Ptx the right to commercialize Zenocutuzumab (Zeno) for Nrg1 Fusion-positive cancer in the US.</li>
<li>The Gap, Inc. (NYSE:<a class="ticker" href="https://www.benzinga.com/stock/GAP#NYSE">GAP</a>) rose 5.7% to $25.63 after JP Morgan upgraded the stock from Neutral to Overweight and raised its price target from $28 to $30.</li>
</ul>
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</ul>

Intraday Update

ENVX

FSLY

MNSO

MRUS

NBIS

NVCR

RXRX

SMCI

XPEV

ZETA

XPeng, NovoCure, Merus, Gap And Other Big Stocks Moving Higher On Monday

WIIM

PTC Therapeutics shares are trading higher after the company announced it signed a global licensee and collaboration agreement with Novartis for its PTC518 Huntington's Disease program.

<ul>
<li>Novartis will assume responsibility for PTC518's development, manufacturing and commercialization, following the completion of the on-going placebo-controlled portion of PIVOT-HD, which is expected to occur in H1 2025. &nbsp;</li>
<li>The companies will share U.S. profits and losses, on a 40/60 basis (40% PTC and 60% Novartis).</li>
<li>The closing of the transaction is subject to customary closing conditions, including regulatory clearance. The parties anticipate that the agreement will close in the first quarter of 2025.</li>
</ul>

PTC Therapeutics Signed An Exclusive Global License And Collaboration Agreement With Novartis For Its PTC518 Huntington's Disease Program, For An Upfront Payment Of $1B, Up To $1.9B In Milestone Payments, A Profit Share In The U.S., And Double-digit Tiered Royalties On Ex-U.S. Sales

PTC Therapeutics, Inc. (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/PTCT#NASDAQ">PTCT</a>) announced today the signing of an exclusive global license and collaboration agreement with Novartis Pharmaceuticals Corporation, a subsidiary of Novartis AG (NYSE:<a class="ticker" href="https://www.benzinga.com/stock/NVS#NYSE">NVS</a>), 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.
"PTC518 is the leading oral disease-modifying therapy in development for Huntington's disease and the economics of this agreement are consistent with the promise of this treatment," said Matthew B. Klein, M.D., Chief Executive Officer, PTC Therapeutics. "This collaboration combines PTC's expertise in developing small molecule splicing therapies with Novartis's expertise in global development and commercialization of neuroscience therapies. We are excited to collaborate with Novartis to accelerate the potential of PTC518 for the hundreds of thousands of HD patients worldwide in need of a therapy designed to be well-tolerated and an effective disease-modifying therapy. PTC will use the proceeds of this transaction to expand our splicing platform as well as to support commercial and development portfolio activities."
"Huntington's Disease is a devastating, fatal, familial disease. This agreement with PTC is intended to bolster our neuroscience pipeline and reflects our strategic focus and commitment to explore new and potentially transformative approaches for neurodegenerative diseases with high unmet needs," said Vas Narasimhan, CEO of Novartis. "We look forward to building on our expertise in neurodegenerative diseases and experience in HD with the intention to advance this potential first in class oral therapy for the HD community."
PTC518 was discovered from PTC's validated splicing platform and is currently being studied in the ongoing Phase 2 PIVOT-HD trial. Interim results reported in June 2024 demonstrated that PTC518 treatment resulted in durable, dose-dependent reduction in blood and cerebrospinal fluid (CSF) mutant Huntingtin protein (HTT) levels as well as early signals of dose-dependent benefit on key clinical measurements at 12 months.1 Importantly, PTC518 continues to demonstrate a favorable safety and tolerability profile.1
Novartis will assume responsibility for PTC518's development, manufacturing and commercialization, following the completion of the on-going placebo-controlled portion of PIVOT-HD, which is expected to occur in H1 2025. &nbsp;
The companies will share U.S. profits and losses, on a 40/60 basis (40% PTC and 60% Novartis).
The closing of the transaction is subject to customary closing conditions, including regulatory clearance. The parties anticipate that the agreement will close in the first quarter of 2025.

PTC Therapeutics Signs Exclusive Global License And Collaboration Agreement With Novartis; PTC To Receive Upfront Payment Of $1B, Up To $1.9B In Development, Regulatory And Sales Milestones

Baird analyst Joel Beatty 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 $48 to $52.

Baird Maintains Outperform on PTC Therapeutics, Raises Price Target to $52

On Tuesday, PTC Therapeutics Inc (NASDAQ:<a class="ticker" href="https://www.benzinga.com/stock/PTCT#NASDAQ">PTCT</a>) revealed data from the global Phase 2 placebo-controlled CardinALS study of Utreloxastat in amyotrophic <a target="_blank" href="https://www.benzinga.com/pressreleases/24/11/n42201952/ptc-therapeutics-announces-topline-results-of-cardinals-trial-of-utreloxastat-in-als-patients" rel="noreferrer noopener">lateral sclerosis patients</a>.
The study did not meet its primary endpoint of slowing disease progression on the composite ALSFRS-R and mortality analysis.
Also Read: <a target="_blank" href="https://www.benzinga.com/general/biotech/24/11/41984686/fda-grants-conditional-approval-for-ptc-therapeutics-gene-therapy-as-first-ever-with-direct-administration-in-brain" rel="noreferrer noopener">FDA Grants Conditional Approval For PTC Therapeutics&#8217; Gene Therapy As First Ever With Direct Administration In Brain</a>
While modest numerical benefit was recorded on the primary endpoint, and a favorable clinical effect was correlated with lowering plasma neurofilament light chain (NfL), a biomarker of neuronal damage, statistical significance was not achieved (p= 0.52). In addition, significance was not achieved on the secondary efficacy endpoints.
Amyotrophic lateral sclerosis (ALS), sometimes referred to as motor neuron disease or&nbsp;Lou Gehrig&#8217;s&nbsp;disease, is a rare neurodegenerative disease that affects motor neurons in the brain and spinal cord.
Utreloxastat was demonstrated to be safe and well-tolerated in the CardinALS trial. However, further development is not planned due to the lack of efficacy and biomarker signal.
William Blair notes that while the CardinALS trial results were disappointing, the program was not a major factor in its investment thesis. As a result, ending the program does not affect the firm&#8217;s Outperform <a target="_blank" href="https://www.benzinga.com/quote/PTCT/analyst-ratings" rel="noreferrer noopener">rating on the stock</a>.
The analyst highlights the potential approval and launch of sepiapterin as a key growth driver over the next year, particularly in treating Phenylketonuria (PKU). William Blair believes the market is undervaluing this opportunity, with a PDUFA decision expected in July.
The analyst also points to growing investor interest in the company&#8217;s Huntington&#8217;s disease program. Given the strong commercial prospects of this small-molecule therapy, positive discussions with regulators about an accelerated approval pathway for PTC-518 could significantly boost the stock. A Type C meeting with the FDA is planned for the fourth quarter to discuss the regulatory pathway.
Additionally, the company aims to file a U.S. marketing application for vatiquinone in Friedreich&#8217;s ataxia (FA) by late 2024. William Blair expects approval based on the drug&#8217;s clinical data and the FDA&#8217;s recent neurology approvals, especially as no treatments exist for pediatric FA patients.
On Wednesday, PTC Therapeutics agreed to sell its Rare Pediatric Disease Priority Review Voucher <a target="_blank" href="https://www.benzinga.com/pressreleases/24/11/n42210840/ptc-therapeutics-enters-into-agreement-to-sell-priority-review-voucher-for-150-million" rel="noreferrer noopener">for $150 million</a>.
PTC was granted the voucher along with the FDA approval of Kebilidi (eladocagene exuparvovec-tneq) for children and adults <a target="_blank" href="https://www.benzinga.com/general/biotech/24/11/41984686/fda-grants-conditional-approval-for-ptc-therapeutics-gene-therapy-as-first-ever-with-direct-administration-in-brain" rel="noreferrer noopener">with AADC deficiency</a>.
Price Action: PTCT stock is down 1.98% at $45.10 during the premarket session at last check Wednesday.
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PTC was granted the PRV on November 13, 2024 along with the U.S. Food and Drug Administration (FDA) approval of KEBILIDI&#x2122; (eladocagene exuparvovec-tneq) for the treatment of children and adults with AADC deficiency, including the full spectrum of disease severity.
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