Financial Performance - Translarna generated $70.4 million in net sales during the quarter ended June 30, 2024[179]. - Emflaza achieved $47.3 million in net sales for the same quarter[179]. - Net product revenues for Q2 2024 were $133.2 million, a decrease of $41.4 million, or 24%, from $174.6 million in Q2 2023, primarily due to lower sales of Translarna and Emflaza[234]. - Emflaza net product revenues decreased by $18.4 million, or 28%, to $47.3 million in Q2 2024, driven by the expiration of orphan drug exclusivity[235]. - Translarna net product revenues were $70.4 million in Q2 2024, down $26.1 million, or 27%, compared to $96.5 million in Q2 2023, attributed to the timing of bulk government orders[235]. - Net product revenues for the six months ended June 30, 2024, were $310.8 million, a decrease of $51.3 million, or 14%, from $362.1 million in the same period of 2023[247]. - Net product sales outside of the United States for the three months ended June 30, 2024, were $85.9 million, a decrease of 21.1% compared to $108.9 million in the same period of 2023[212]. - Net product sales in the United States for the three months ended June 30, 2024, were $47.3 million, down 28.0% from $65.7 million in the same period of 2023[212]. Regulatory Developments - The marketing exclusivity period for Emflaza related to DMD in patients aged five years and older expired in February 2024, expected to negatively impact revenue[186]. - Upstaza received approval from the EC for AADC deficiency treatment in July 2022 and the FDA accepted its BLA for review with a target action date of November 13, 2024[189]. - The CHMP issued a negative opinion on the renewal of Translarna's conditional marketing authorization, which is currently under re-examination[182]. - The company plans to submit regulatory applications for sepiapterin in Japan and Brazil later in 2024[195]. - The MAA submission for sepiapterin was validated by the EMA, triggering a $15.0 million regulatory milestone payment[207]. - A BLA for gene therapy for AADC deficiency was submitted to the FDA in March 2024, with priority review granted and a target action date of November 13, 2024[275]. - The MAA for sepiapterin for PKU was validated by the EMA in May 2024, triggering a $15 million regulatory milestone payment[280]. - The company expects to make additional payments of $50 million upon achieving regulatory milestones related to sepiapterin in 2024[280]. - As of June 30, 2024, potential remaining regulatory milestones for Upstaza are estimated at $11.1 million, with potential sales milestones of $50 million[280]. Research and Development - Sepiapterin demonstrated a 63% reduction in blood Phe levels in the primary analysis population during its Phase 3 trial for phenylketonuria[192]. - PTC518 demonstrated a mean 30% reduction in mutant HTT levels at the 10mg dose level during the Phase 2 study for Huntington's disease[196]. - Vatiquinone showed significant benefits on key disease subscales and secondary endpoints in the MOVE-FA trial, despite not meeting the primary endpoint[197]. - The company plans to submit an NDA for vatiquinone in late 2024 based on the MOVE-FA trial data[197]. - Utreloxastat was well-tolerated in a Phase 1 trial, and a Phase 2 trial for ALS is expected to yield topline results in Q4 2024[197]. - Research and development expenses for the three months ended June 30, 2024, were $132.2 million, a decrease of 29.0% from $185.9 million in the same period of 2023[225]. - Total research and development expenses for the six months ended June 30, 2024, were $248.3 million, down 34.8% from $381.0 million in the same period of 2023[225]. - Research and development expense was $248.3 million for the six months ended June 30, 2024, a decrease of $132.7 million, or 35%, from $381.0 million for the same period in 2023[254]. Financial Position and Cash Flow - The company has an accumulated deficit of $3,474.3 million as of June 30, 2024, with a net loss of $190.8 million for the six months ended June 30, 2024[204]. - As of June 30, 2024, the company had cash, cash equivalents, and marketable securities of $1.09 billion[270]. - Net cash used in operating activities was $0.7 million for the six months ended June 30, 2024, compared to $43.6 million for the same period in 2023, primarily due to clinical development and commercial activities, offset by a $100 million sales milestone from Evrysdi[272]. - Net cash used in investing activities increased to $180.9 million for the six months ended June 30, 2024, from $52.7 million in the same period of 2023, mainly for product rights acquisition and marketable securities[273]. - Net cash provided by financing activities rose significantly to $247.0 million for the six months ended June 30, 2024, compared to $22.6 million in 2023, driven by proceeds from sales of future royalties and employee stock purchase plans[274]. Operational Challenges and Future Outlook - The company expects to incur significant expenses related to commercialization efforts and ongoing clinical trials[205]. - The company may seek to expand its product pipeline through in-licensing or acquisitions, which could increase future capital requirements[206]. - The company anticipates ongoing significant expenses related to commercialization efforts in various regions, including the U.S. and EEA, and costs for clinical trials and studies for sepiapterin and other programs[275]. - The company has never been profitable and will need to generate significant revenues to achieve and sustain profitability, potentially requiring substantial additional funding[282]. - If unable to raise additional funds, the company may need to delay or reduce product development and commercialization efforts[283].

Core Insights - Analysts project that PTC Therapeutics (PTCT) will report a quarterly loss of $1 per share, marking a 62.4% increase in losses year over year, with revenues expected to decline by 10.1% to $192.12 million [1] - The consensus EPS estimate has been revised upward by 2% in the past 30 days, indicating a reassessment of earnings projections by covering analysts [2] - Changes in earnings projections are crucial for predicting investor reactions, as empirical studies show a strong correlation between earnings estimate trends and short-term stock price movements [3] Revenue Estimates - Analysts estimate that 'Revenues- Net product revenue' will be $134.09 million, reflecting a decrease of 23.2% from the same quarter last year [5] - The consensus for 'Revenues- Royalty revenue' is projected at $57.75 million, indicating an increase of 56.7% year over year [5] - 'Revenues- Net product revenue- Emflaza' is expected to reach $47.55 million, showing a decline of 28% from the prior-year quarter [5] - 'Revenues- Net product revenue- Translarna' is anticipated to be $83.86 million, representing a decrease of 12.6% year over year [6] Stock Performance - Over the past month, PTC Therapeutics shares have experienced a return of -2.3%, compared to a -6.7% change in the Zacks S&P 500 composite [6] - Based on its Zacks Rank 3 (Hold), PTCT is expected to perform in line with the overall market in the near future [6]

Core Viewpoint - PTC Therapeutics has submitted a New Drug Application (NDA) for sepiapterin to the U.S. FDA, aimed at treating both pediatric and adult patients with phenylketonuria (PKU) [1][2]. Group 1: NDA Submission and Clinical Data - The NDA submission is a significant milestone for providing treatment options for PKU patients in the U.S. [2] - The NDA is supported by data from the phase 3 APHENITY trial, which showed a mean reduction in phenylalanine (Phe) levels of 63% in the overall treated population and 69% in subjects with classical PKU [2] - 84% of subjects achieved Phe control under treatment guidelines of <360 µmol/L, and 22% of subjects normalized their Phe levels [2] - The NDA also includes data from the APHENITY open-label extension study, demonstrating the durability of sepiapterin's effects [2] Group 2: Phe Tolerance and Quality of Life - Approximately 60% of subjects in the Phe tolerance sub-study were able to consume protein above the age-adjusted recommended daily allowance while maintaining Phe levels <360 µmol/L [2] - The ability to liberalize diets is expected to enhance patient quality of life and facilitate physician adoption and payer acceptance [2] Group 3: Global Regulatory Status - The European marketing authorization application for sepiapterin is currently under review, with applications for Japan and Brazil expected later in 2024 [3] Group 4: Product Information - Sepiapterin is an oral formulation of synthetic sepiapterin, a precursor to tetrahydrobiopterin, which is essential for the metabolism of various metabolic products [4] - It is considered more bioavailable than exogenously administered synthetic BH4, potentially benefiting a wide range of PKU patients [4] Group 5: Background on PKU - Phenylketonuria (PKU) is a rare inherited metabolic disorder that can lead to severe disabilities if untreated, affecting approximately 58,000 individuals globally [5]

Investors: Kylie O'Keefe +1 (908) 300-0691 [email protected] SOURCE PTC Therapeutics, Inc. WARREN, N.J., July 25, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the company will host a webcast conference call to report its second quarter 2024 financial results and provide an update on the company's business and outlook on Thursday, Aug. 8, at 4:30 p.m. EDT. To access the call by phone, please click here to register and you will be provided with dial-in details. To avoid dela ...

While the sustainability of the immediate price change and future earnings expectations will mostly depend on management's discussion of business conditions on the earnings call, it's worth handicapping the probability of a positive EPS surprise. Estimate Revisions Trend Earnings Whisper A positive Earnings ESP is a strong predictor of an earnings beat, particularly when combined with a Zacks Rank #1 (Strong Buy), 2 (Buy) or 3 (Hold). Our research shows that stocks with this combination produce a positive s ...

NEW YORK, July 09, 2024 (GLOBE NEWSWIRE) -- Attorney Advertising -- Bronstein, Gewirtz & Grossman, LLC is investigating potential claims on behalf of purchasers of PTC Therapeutics, Inc. ("PTC" or "the Company") (NASDAQ: PTCT). Investors who purchased PTC securities are encouraged to obtain additional information and assist the investigation by visiting the firm's site: bgandg.com/PTCT. Investigation Details On June 28, 2024, PTC issued a press release announcing that the European Medicines Agency's Committ ...

PTC Therapeutics (PTCT) announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Commission (EC) once again adopted a negative opinion on the annual renewal of the conditional marketing authorization of Translarna (ataluren) to treat nonsense mutation Duchenne muscular dystrophy (nmDMD). PTC Therapeutics plans to further request a re-examination of the negative opinion regarding Translarna in the EU. Meanwhile, the marketing authorization for Translarna will remain in effect ...

- PTC to submit a request for re-examination - WARREN, N.J., June 28, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the CHMP has issued a negative opinion on the renewal of the conditional marketing authorization of Translarna™ (ataluren) for the treatment of nmDMD. This opinion follows the return of the previously issued negative opinion by the European Commission (EC) for re-review. PTC plans to request re-examination of the opinion. The marketing authorization for Transl ...

This rise in share price is attributable to the company's progress with its pipeline development. Last week, management reported encouraging interim data from the mid-stage PIVOT-HD study evaluating PTC518, its investigational treatment for Huntington's disease (HD). Following 12 months of treatment with PTC518, patients achieved dose-dependent lowering of mutant huntingtin (mHTT) protein in the blood and cerebrospinal fluid (CSF). The FDA also removed a partial clinical hold on this study, which was previo ...

WARREN, N.J., June 21, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that on June 17, 2024, the company approved non-statutory stock options to purchase an aggregate of 1,380 shares of its common stock and 6,260 restricted stock units ("RSUs"), each representing the right to receive one share of its common stock upon vesting, to nine new employees. The awards were made pursuant to the Nasdaq inducement grant exception as a component of the new hires' employment compensation. ABO ...