PTCT PTC Therapeutics Inc.

47.54
-0.46  -1%
Previous Close 48
Open 48.32
52 Week Low 30.7923
52 Week High 59.89
Market Cap $3,218,610,794
Shares 67,703,214
Float 67,179,439
Enterprise Value $3,094,467,368
Volume 645,759
Av. Daily Volume 859,550
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Upcoming Catalysts

Drug Stage Catalyst Date
PTC857
Healthy volunteers
Phase 1
Phase 1
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PTC-AADC
AADC deficiency
BLA Filing
BLA Filing
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PTC299
COVID-19
Phase 2/3
Phase 2/3
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Drug Pipeline

Drug Stage Notes
Risdiplam (RG7916)
Spinal Muscular Atrophy (SMA) Type 1
Approved
Approved
FDA Approval announced August 7, 2020.
Translarna
Duchenne muscular dystrophy caused by nonsense mutations (nmDMD)
Phase 3
Phase 3
Received a Refusal to File letter February 23 2016. Appeal rejected October 17, 2016. Decision made to resubmit NDA during 1Q 2017 under protest. Further CRL October 25, 2017. NDA to be refiled.
Emflaza
Duchenne muscular dystrophy (age 2-5)
Approved
Approved
FDA Approval announced for label expansion June 7, 2019.
Inotersen (IONIS-TTRRx)
NEURO-TTR - familial amyloid polyneuropathy (FAP).
Approved
Approved
FDA Approval announced October 5, 2018,
Translarna
Nonsense mutation cystic fibrosis
Phase 3
Phase 3
Phase 3 data released March 2, 2017 - endpoints not met. Development to be discontinued.

Latest News

  1. SOUTH PLAINFIELD, N.J., Aug. 7, 2020 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ:PTCT) today announced that the U.S. Food and Drug Administration (FDA) has approved Evrysdi™ (risdiplam), the first at-home, orally administered treatment for spinal muscular atrophy (SMA) in adults and children 2 months and older. Evrysdi showed clinically meaningful improvements in motor function and obtainment of developmental milestones across two trials in patients age two months and above and across all levels of disease severity, including types 1, 2, and 3 SMA. Infants achieved key motor milestones not normally seen in the natural course of the disease, such as the ability to sit without support. Evrysdi also preserved vital functions and improved survival…

    SOUTH PLAINFIELD, N.J., Aug. 7, 2020 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ:PTCT) today announced that the U.S. Food and Drug Administration (FDA) has approved Evrysdi™ (risdiplam), the first at-home, orally administered treatment for spinal muscular atrophy (SMA) in adults and children 2 months and older. Evrysdi showed clinically meaningful improvements in motor function and obtainment of developmental milestones across two trials in patients age two months and above and across all levels of disease severity, including types 1, 2, and 3 SMA. Infants achieved key motor milestones not normally seen in the natural course of the disease, such as the ability to sit without support. Evrysdi also preserved vital functions and improved survival at 12 months. In particular, Evrysdi improved survival without permanent ventilation compared to natural history when measured at 12 and 23 months.

    "Today marks an incredibly important moment for the broader SMA patient community that had been in dire need of safe and effective treatment options," said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics. "We are appreciative of the patients, physicians and others involved with its development, and our partners at the SMA Foundation and Roche. We are proud that the first oral treatment for spinal muscular atrophy patients was discovered through our splicing platform. I believe that this is one of the most innovative products in the pharmaceutical industry and we are gratified to have played a key role in its discovery and development."

    Evrysdi has been studied in a robust clinical trial program in SMA with more than 450 patients and subjects. The program includes infants aged 2 months to adults aged 60 with a range of symptoms and motor function including patients with scoliosis or joint contractures. Patients previously treated for SMA with other medications were also included. The approval is based on results from two clinical studies designed to represent a broad spectrum of patients living with SMA: FIREFISH in symptomatic infants aged 2 to 7 months; and SUNFISH, in children and adults aged 2 to 25 years. SUNFISH is the first and only placebo-controlled trial to include adults with types 2 and 3 SMA. 

    "We started looking for treatments for SMA more than a decade ago," said Loren Eng, President of the SMA Foundation. "PTC was one of the companies that embraced and remained committed to the idea of developing a truly innovative therapeutic for this disease. We are proud to be part of the collaboration that brought the first oral SMA treatment for patients."

    In FIREFISH, 41% (7/17) of infants treated with the therapeutic dose achieved the ability to sit without support for at least 5 seconds as measured by the Bayley Scales of Infant and Toddler Development Third Edition (BSID-III). Additionally, 90% (19/21) of infants were alive without permanent ventilation at 12 months of treatment and reached 15 months of age or older. As described in the natural history of untreated infantile-onset SMA, infants would not be expected to be able to sit independently and only 25 percent of infants would be expected to survive without permanent ventilation beyond 14 months of age. In SUNFISH, children and adults treated with Evrysdi experienced a statistically significant and clinically meaningful improvement in motor function at 12 months (1.55 point mean difference; p = 0.0156) compared to placebo, as measured by a change in baseline on the Motor Function Measure-32 (MFM-32) scale.

    Evrysdi demonstrated a favorable efficacy and safety profile, with the safety profile established across the FIREFISH and SUNFISH trials. The most common adverse reactions were fever, diarrhea, and rash in later-onset SMA. In infantile-onset SMA, the most common adverse events were similar and also included upper respiratory tract infection, pneumonia, constipation, and vomiting. There were no treatment-related safety findings leading to withdrawal from any study. Ophthalmological monitoring is not required.

    Evrysdi will be available in the United States within two weeks for direct delivery to patients' homes through Accredo Health Group Inc., an Express Scripts specialty pharmacy.

    Evrysdi will be marketed in the United States by Genentech, a member of the Roche Group, and received an orphan drug designation from FDA in 2017. Outside the United States, Roche holds global commercialization rights to risdiplam, and the European Medicines Agency (EMA) previously granted PRIME (Priority Medicines) designation to risdiplam in 2018 for the treatment of people with SMA and an orphan drug designation in 2019. The submission of a marketing authorization application (MAA) to EMA for risdiplam is pending. At this time, Roche has submitted applications for approval in Brazil, Chile, China, Indonesia, Russia, South Korea and Taiwan.

    Evrysdi is a product of the SMA collaboration between PTC, the SMA Foundation, and Roche.

    About Evrysdi™ (risdiplam)

    Evrysdi is a survival motor neuron 2 (SMN2)-directed RNA splicing modifier designed to treat SMA caused by mutations in chromosome 5q that lead to SMN protein deficiency. Evrysdi is designed to distribute evenly to all parts of the body, including the central nervous system (CNS). Evrysdi is administered daily at home in liquid form by mouth or feeding tube.

    About the Clinical Studies

    FIREFISH (NCT02913482)

    FIREFISH, an open-label, two-part pivotal study, was designed to assess Evrysdi safety, tolerability, efficacy, pharmacokinetics (PK) and pharmacodynamics (PD) in patients aged 1 to 7 months with Type 1 SMA. Part 1 evaluated several doses of Evrysdi and determined the therapeutic dose of 0.2 mg/kg for Part 2. In Part 1, after 12 months of Evrysdi treatment:

    • 41% (7/17) of infants treated with the therapeutic dose achieved the ability to sit without support for at least 5 seconds as measured by the BSID-III gross motor scale.
    • 90% (19/21) of all infants were alive without permanent ventilation* and reached 15 months of age or older.
    • 81% (17/21) of all patients were alive without permanent ventilation* after a minimum of 23 months of treatment and reached an age of 28 months or older (median 32 months; range 28-45 months)

    *Permanent ventilation defined as tracheostomy or ≥16 hours of non-invasive ventilation per day or intubation for ≥21 consecutive days in the absence of, or following the resolution of, an acute reversible event.

    SUNFISH (NCT02908685)

    SUNFISH, a two-part, placebo-controlled, multicenter pivotal trial, was designed to assess Evrysdi safety, tolerability, efficacy, PK and PD in people with type 2 or 3 SMA aged 2 to 25, including those with scoliosis (67% in Part 2) and joint contractures at baseline. In Part 2, after 12 months, Evrysdi treatment led to:

    • A clinically meaningful and statistically significant improvement in motor function among children and adults, as measured by a change in baseline in the MFM-32 total score (1.55 point mean difference; p = 0.0156), at 12 months as compared to placebo (1.36 points [risdiplam treatment arm -95% CI: 0.61, 2.11]; [placebo treatment arm: -0.19 points 95% CI: 1.22, 0.84]). MFM-32 assesses 32 different motor functions across a wide range of people with SMA.
    • Improved upper limb motor function compared to baseline, as measured by the Revised Upper Limb Module (RULM), a secondary independent motor function endpoint of the study (1.59 point difference; p=0.0028).

    Clinical Trial Safety Data

    The safety profile of Evrysdi was established across FIREFISH and SUNFISH pivotal trials. The most common adverse reactions in later-onset SMA (incidence of at least 10% of patients treated with Evrysdi and more frequently than control) were fever, diarrhea, and rash. The most common adverse reactions in infantile-onset SMA were similar to those observed in later-onset SMA patients. Additionally, the most common adverse reactions (incidence of at least 10%) were upper respiratory tract infection, pneumonia, constipation, and vomiting. 

    About the Evrysdi Clinical Trial Program

    In addition to FIREFISH and SUNFISH, Evrysdi is being evaluated in a broad range of people with SMA, including in:

    • JEWELFISH (NCT03032172): an open-label exploratory trial designed to assess the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) in people with SMA aged 6 months to 60 years who received other investigational or approved SMA therapies for at least 90 days prior to receiving Evrysdi. Recruitment for this study is complete with 174 people enrolled.
    • RAINBOWFISH (NCT03779334): an open-label, single-arm, multicenter study investigating the efficacy, safety, pharmacokinetics and pharmacodynamics of Evrysdi in infants (~n=25), from birth to six weeks of age (at first dose) with genetically diagnosed SMA who are not yet presenting with symptoms. The study is currently recruiting.

    About Spinal Muscular Atrophy (SMA)

    Spinal muscular atrophy (SMA) is a severe, inherited, progressive neuromuscular disease that causes devastating muscle atrophy and disease-related complications. It is the most common genetic cause of infant mortality and one of the most common rare diseases, affecting approximately one in 11,000 babies1. SMA leads to the progressive loss of nerve cells in the spinal cord that control muscle movement. Depending on the type of SMA, an individual's physical strength and their ability to walk, eat or breathe can be significantly diminished or lost.

    SMA is caused by a mutation in the survival motor neuron 1 (SMN1) gene that results in a deficiency of SMN protein. SMN protein is found throughout the body and increasing evidence suggests SMA is a multi-system disorder and the loss of SMN protein may affect many tissues and cells, which can stop the body from functioning.

    About PTC Therapeutics, Inc.

    PTC is a science-driven, global biopharmaceutical company focused on the discovery, development and commercialization of clinically differentiated medicines that provide benefits to patients with rare disorders. PTC's ability to globally commercialize products is the foundation that drives investment in a robust and diversified pipeline of transformative medicines and our mission to provide access to best-in-class treatments for patients who have an unmet medical need. To learn more about PTC, please visit us at www.ptcbio.com and follow us on Facebook, on Twitter at @PTCBio, and on LinkedIn.

    For More Information:

    Investors:

    Alex Kane

    +1 (908) 912-9643

    Media: 

    Jane Baj

    +1 (908) 912-9167 

     

    Forward-Looking Statements:

    This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. All statements contained in this release, other than statements of historical fact, are forward-looking statements, including statements regarding: the future expectations, plans and prospects for PTC; the timing of commercial availability and potential methods of distribution for Evrysdi; advancement of PTC's joint collaboration program in SMA, including any potential regulatory submissions, regulatory approvals or commercial prospects;  PTC's strategy, future operations, future financial position, future revenues and, projected costs; and the objectives of management. Other forward-looking statements may be identified by the words "guidance", "plan," "anticipate," "believe," "estimate," "expect," "intend," "may," "target," "potential," "will," "would," "could," "should," "continue," and similar expressions.

    PTC's actual results, performance or achievements could differ materially from those expressed or implied by forward-looking statements it makes as a result of a variety of risks and uncertainties, including those related to: the outcome of pricing, coverage and reimbursement negotiations with third party payors for PTC's products or product candidates that PTC commercializes or may commercialize in the future, including Evrysdi; the enrollment, conduct, and results of studies under the SMA collaboration and events during, or as a result of, the studies that could delay or prevent further development under the program, including any potential regulatory submissions and potential commercialization with regards to Evrysdi; the eligible patient base and commercial potential of Evrysdi or any of PTC's other product candidates; and the factors discussed in the "Risk Factors" section of PTC's most recent Quarterly Report on Form 10-Q and Annual Report on Form 10-K, as well as any updates to these risk factors filed from time to time in PTC's other filings with the SEC. You are urged to carefully consider all such factors.

    As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commercialization of new products. There are no guarantees that any product or product candidate will receive or maintain regulatory approval in any territory, or prove to be commercially successful, including Evrysdi.

    The forward-looking statements contained herein represent PTC's views only as of the date of this press release and PTC does not undertake or plan to update or revise any such forward-looking statements to reflect actual results or changes in plans, prospects, assumptions, estimates or projections, or other circumstances occurring after the date of this press release except as required by law.

    Cision View original content:http://www.prnewswire.com/news-releases/ptc-therapeutics-announces-fda-approval-of-evrysdi-risdiplam-for-the-treatment-of-spinal-muscular-atrophy-in-adults-and-children-2-months-and-older-301108489.html

    SOURCE PTC Therapeutics, Inc.

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  2. SOUTH PLAINFIELD, N.J., Aug. 6, 2020 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ:PTCT) is proud to announce that its Senior Vice President of Corporate and Patient Relations, Mary Frances Harmon, has been named to PharmaVOICE magazine's annual list of the most inspiring people in the life sciences industry. Mary Frances is recognized for her inspirational qualities, passion and conviction for the industry, and dedication to improving the lives of patients.

    "Throughout her tenure at PTC, Mary Frances has consistently served as an inspiration to us, the patients, and their families - performing from a place of genuine care and compassion," said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics. "With her long and dedicated experience in patient advocacy, she has been vital in establishing and growing relationships with patients, advocacy groups and caregivers so that PTC can better serve the needs of patients. We could not be prouder of Mary Frances and we are all thrilled that she will receive this well-earned recognition from PharmaVOICE."

    Mary Frances has over 30 years of experience in the pharmaceutical industry and currently oversees strategic and operational leadership for corporate communications, government affairs, advocacy, and patient communications and engagement, which is essential in PTC's commitment to patient families. Her work helps create a unified vision for patient and government relations and implementing programs with patient groups. During her first year at PTC, Mary Frances launched the Strategies to Realize Innovation, Vision, and Empowerment (STRIVE) awards, which supports initiatives benefitting the Duchenne muscular dystrophy patient community. STRIVE drives innovation and awareness within the medical community and has awarded funding to 28 patient advocacy organizations worldwide.

    To learn more about Mary Frances' work at PTC and her PharmaVOICE 100 recognition, please visit the July/August 2020 online digital edition of the magazine: PharmaVOICE July/August 2020

    About PTC Therapeutics, Inc.

    PTC is a science-driven, global biopharmaceutical company focused on the discovery, development and commercialization of clinically differentiated medicines that provide benefits to patients with rare disorders. PTC's ability to globally commercialize products is the foundation that drives investment in a robust and diversified pipeline of transformative medicines and our mission to provide access to best-in-class treatments for patients who have an unmet medical need. To learn more about PTC, please visit us at www.ptcbio.com and follow us on Facebook, on Twitter at @PTCBio, and on LinkedIn.

    For More Information:

    Investors:

    Alex Kane

    +1 (908) 912-9643

    Media:

    Jane Baj

    +1 (908) 912-9167

     

    Cision View original content to download multimedia:http://www.prnewswire.com/news-releases/ptc-therapeutics-mary-frances-harmon-named-one-of-the-most-inspiring-leaders-in-life-sciences-by-pharmavoice-magazine-301107383.html

    SOURCE PTC Therapeutics, Inc.

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  3. SOUTH PLAINFIELD, N.J., Aug. 5, 2020 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ:PTCT) today announced a corporate update and reported financial results for the second quarter ending June 30, 2020.

    "We are pleased with the progress in our pipeline and the strong commercial performance of our global Duchenne franchise," said Stuart W. Peltz, Ph.D., CEO of PTC Therapeutics. "Our growing revenue stream allows us to continue to invest in the development of treatments leveraging our novel technology platforms. We have a robust pipeline and with more than $1 billion in cash are well positioned to advance multiple differentiated therapies for patients living with rare disorders, not only in the near term, but also for many years to come."

    Key

    SOUTH PLAINFIELD, N.J., Aug. 5, 2020 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ:PTCT) today announced a corporate update and reported financial results for the second quarter ending June 30, 2020.

    "We are pleased with the progress in our pipeline and the strong commercial performance of our global Duchenne franchise," said Stuart W. Peltz, Ph.D., CEO of PTC Therapeutics. "Our growing revenue stream allows us to continue to invest in the development of treatments leveraging our novel technology platforms. We have a robust pipeline and with more than $1 billion in cash are well positioned to advance multiple differentiated therapies for patients living with rare disorders, not only in the near term, but also for many years to come."

    Key Second Quarter and Other Corporate Updates:

    • Our Duchenne muscular dystrophy franchise had strong performance in the second quarter. The greater than 30% year-over-year increase in second quarter sales for Emflaza was driven by ongoing improvements in our commercial business. New Duchenne patients continue to be identified in Europe, LATAM and other key markets for Translarna despite the challenges of COVID-19.
    • As part of the annual renewal process for Translarna, the European Medicines Agency (EMA) confirmed its risk/benefit profile for the sixth consecutive year. In addition, PTC previously announced that the Committee for Medicinal Products for Human Use (CHMP) of the EMA recommended to remove the statement "efficacy has not been demonstrated in non-ambulatory patients" from the SmPC for Translarna. This label change enables healthcare professionals to use their clinical judgement to make treatment decisions for their patients on Translarna who have lost ambulation and should support reimbursement agencies granting continued access to Translarna.
    • In the second quarter, PTC strengthened its pipeline and platforms with the acquisition of a late-stage asset for phenylketonuria (PKU). PTC923 is a clinical-stage investigational therapy for inborn errors of metabolism, including PKU and other diseases associated with defects in the tetrahydrobiopterin (BH4) biochemical pathways diagnosed at birth. PTC is currently conducting nonclinical studies to support the long-term dosing for the planned phase 3 trial in PKU in 2021.
    • The U.S. Food and Drug Administration (FDA) PDUFA date for risdiplam is August 24, 2020. In addition, a submission of a marketing authorization application (MAA) to EMA for risdiplam is imminent. The filing of the MAA would trigger a $15 million milestone payment to PTC from Roche. Roche has submitted applications for approval in Brazil, Chile, China, Indonesia, Russia, South Korea and Taiwan.
    • PTC recently announced an agreement to monetize a portion of the rights to the risdiplam royalty stream for a $650 million up front payment from Royalty Pharma plc, providing PTC with significant non-dilutive capital. PTC retains nearly 60% of the risdiplam royalty stream up until a $1.3 billion threshold is reached, after which PTC retains 100% of the risdiplam royalty stream. PTC also retains all economics associated with up to approximately $400 million in remaining regulatory and sales milestones.
    • Launch activities for Tegsedi® continue to focus on patient finding. Pricing discussions in Brazil are ongoing and are expected to be completed by the end of the year.
    • The submission of an application for marketing authorization for Waylivra® in patients with familial chylomicronemia syndrome (FCS) has been filed with ANVISA and a decision is expected in 2021. Patient finding and early access programs are ongoing.
    • PTC continues to develop manufacturing capabilities for its gene therapy products. It has expanded its collaboration with MassBiologics for the manufacturing of the aromatic L-amino acid decarboxylase deficiency (AADCd) commercial supply. In addition, PTC gained occupancy under its lease of ~220,000 square feet at the former BMS site in Hopewell, NJ. Manufacturing of clinical materials for its gene therapy programs at this site is expected to initiate in 2021.
    • PTC recently launched a global internship program, the Talent Pipeline Program (TPP), aimed at providing recent graduates, especially those in minority communities, with real-world experience in the biopharmaceutical industry and related professions, including research, finance, commercial, compliance, quality, legal, information technology, and communications. The TPP will provide approximately 30 interns with a one-year paid program with real-world training experience.

    Updates in PTC's Diverse Product Pipeline

    • In its Bio-e platform, PTC expects to initiate potential registrational trials with vatiquinone, formerly known as PTC743, in refractory mitochondrial epilepsy in the third quarter of 2020 and in Friedreich ataxia in the fourth quarter of 2020.
    • The first subject has been dosed in the Phase 1 trial for PTC857, PTC's second compound from the Bio-e platform. Data from the single ascending dose and multiple ascending dose studies is expected by the end of 2020.
    • PTC recently initiated a Phase 2/3 clinical trial for PTC299 for COVID-19 called FITE-19. PTC expects Stage 1 to be completed in the second half of 2020 and anticipates reporting top-line results from both stages in the first half of 2021. Multiple sites have been opened in the U.S., Brazil, Spain and Australia with additional countries for Stage 2 expected to initiate in the coming months.
    • In the novel splicing platform, the Huntington disease program remains on track for the initiation of first-in-human studies in 2020.
    • PTC expects to initiate submission of the biologics license application (BLA) to the U.S. FDA in the second half of 2020 for its AADCd gene therapy program.
    • Due to COVID-19 related delays, PTC now expects the final opinion from the CHMP on the MAA for the AADCd program in the first quarter of 2021.
    • Given the evolving COVID-19 situation in the U.S., the final study muscle biopsies have not yet been collected from 8 remaining boys in the Translarna (ataluren) dystrophin study. PTC is continuously monitoring the situation to determine when it will be possible to safely obtain the final biopsies and is exploring all potential options in order to have a data read out by the end of 2020. All patients in the study remain on Translarna until they are able to complete the final study visit.

    Financial Highlights:

    • Total net product revenues were $75.2 million across our commercial portfolio for the second quarter of 2020, compared to total net product revenues of $85.5 million for the second quarter of 2019.
    • Translarna net product revenues were $38.6 million for the second quarter of 2020, compared to $57.8 million for the second quarter of 2019. Revenues for the second quarter of 2020 were impacted by the timing of a group purchase order from Brazil, which is the primary driver for the year-over-year decrease, as the second quarter of 2019 included a significant group purchase order from Brazil. Due to the impact of the pandemic, there was an administrative delay by the Brazilian Ministry of Health in receiving the centralized Translarna group purchase order. PTC anticipates a group purchase order from Brazil later this year.
    • Emflaza net product revenues were $36.2 million for the second quarter of 2020, compared to $27.6 million for the second quarter of 2019. Growth in net product revenues was driven by new patient prescriptions and continued operational improvements and efficiencies in our commercial business.
    • Generally accepted accounting principles in the U.S. (GAAP) research and development (R&D) expenses were $176.5 million for the second quarter of 2020, compared to $60.0 million for the second quarter of 2019. The increase in R&D expenses includes one-time charges of $53.6 million related to the acquisition of Censa Pharmaceuticals, and $41.2 million related to the MassBiologics of the University of Massachusetts Medical School agreement for commercial manufacturing of our lead gene therapy program in AADCd.
    • Non-GAAP R&D expenses were $168.0 million for the second quarter of 2020, excluding $8.6 million in non-cash, stock-based compensation expense, compared to $54.5 million for the second quarter of 2019, excluding $5.5 million in non-cash, stock-based compensation expense.
    • GAAP selling, general and administrative (SG&A) expenses were $53.7 million for the second quarter of 2020, compared to $49.2 million for the second quarter of 2019. The increase reflects continued investment to support our commercial activities including our expanding commercial portfolio.
    • Non-GAAP SG&A expenses were $45.3 million for the second quarter of 2020, excluding $8.3 million in non-cash, stock-based compensation expense, compared to $43.8 million for the second quarter of 2019, excluding $5.4 million in non-cash, stock-based compensation expense.
    • Change in the fair value of deferred and contingent consideration was $7.7 million for the second quarter of 2020, compared to $5.3 million for the second quarter of 2019. The change in fair value of deferred and contingent consideration is related to the fair valuation of potential future consideration to be paid to former equity holders of Agilis Biotherapeutics, Inc. (Agilis) in connection with PTC's acquisition of Agilis, which closed in August 2018.
    • Settlement of deferred and contingent consideration was $10.6 million for the second quarter of 2020. The settlement of deferred and contingent consideration is related to a loss upon the settlement of the deferred and contingent consideration liabilities as a result of the rights exchange agreement with certain former shareholders of Agilis, whereby such former shareholders exchanged their pro rata share of specific future cash milestone payments in the aggregate amount of $225 million for a mixture of cash and equity of PTC. Under this agreement, which the former shareholders and PTC entered into on April 29, 2020, PTC has paid $36.9 million in cash and issued 2,821,176 shares of common stock in exchange for the cancellation and forfeiture of the participating shareholders' rights to receive (i) $174.0 million, in the aggregate, of potential milestone payments based on the achievement of certain regulatory milestones and (ii) $37.6 million, in the aggregate, of $40.0 million in development milestone payments that would have been due upon the passing of the second anniversary of the closing of the Merger, regardless of whether the milestones are achieved.
    • Net loss was $181.4 million for the second quarter of 2020, compared to net loss of $41.8 million for the second quarter of 2019.
    • Cash, cash equivalents and marketable securities were $498.9 million as of June 30, 2020, compared to $686.6 million as of December 31, 2019.
    • Shares issued and outstanding as of June 30, 2020 were 67,240,679.

    Non-GAAP Financial Measures:

    In this press release, the financial results of PTC are provided in accordance with GAAP and using certain non-GAAP financial measures. In particular, the non-GAAP financial measures exclude non-cash, stock-based compensation expense. These non-GAAP financial measures are provided as a complement to financial measures reported in GAAP because management uses these non-GAAP financial measures when assessing and identifying operational trends. In management's opinion, these non-GAAP financial measures are useful to investors and other users of PTC's financial statements by providing greater transparency into the historical and projected operating performance of PTC and the company's future outlook. Non-GAAP financial measures are not an alternative for financial measures prepared in accordance with GAAP. Quantitative reconciliations of the non-GAAP financial measures to their respective closest equivalent GAAP financial measures are included in the table below.

    PTC Therapeutics, Inc.

    Consolidated Statements of Operations

    (In thousands, except share and per share data)







    Three Months Ended June 30,



    Six Months Ended June 30,





    2020



    2019



    2020



    2019

    Revenues:















    Net product revenue



    $

    75,239





    $

    85,476





    $

    143,435



    $

    138,530



    Collaboration and grant revenue







    46





    63



    575



    Total revenues



    75,239





    85,522





    143,498



    139,105



    Operating expenses:















    Cost of product sales



    5,304





    3,211





    9,389



    5,587



    Amortization of acquired intangible asset



    8,731





    6,575





    16,679



    12,652



    Research and development (1)



    176,525





    59,979





    266,632



    112,544



    Selling, general and administrative (2)



    53,659





    49,215





    111,869



    89,760



    Change in the fair value of deferred and contingent consideration



    7,680





    5,300





    8,580



    26,460



    Settlement of deferred and contingent consideration



    10,613









    10,613





    Total operating expenses



    262,512





    124,280





    423,762



    247,003



    Loss from operations



    (187,273)





    (38,758)





    (280,264)



    (107,898)



    Interest expense, net



    (5,379)





    (2,074)





    (11,021)



    (4,362)



    Other income (expense), net



    11,309





    (183)





    (2,523)



    (292)



    Loss before income tax expense



    (181,343)





    (41,015)





    (293,808)



    (112,552)



    Income tax expense



    (84)





    (774)





    (306)



    (1,350)



    Net loss attributable to common stockholders



    $

    (181,427)





    $

    (41,789)





    $

    (294,114)



    $

    (113,902)



















    Weighted-average shares outstanding:















    Basic and diluted (in shares)



    65,150,780





    55,912,748





    63,769,958



    57,113,141



    Net loss per share—basic and diluted (in dollars per share)



    $

    (2.78)





    $

    (0.75)





    $

    (4.61)



    $

    (1.99)



















    (1) Research and development reconciliation















    GAAP research and development



    $

    176,525





    $

    59,979





    $

    266,632



    $

    112,544



    Less: share-based compensation expense



    8,562





    5,516





    16,741



    10,203



    Non-GAAP research and development



    $

    167,963





    $

    54,463





    $

    249,891



    $

    102,341



















    (2) Selling, general and administrative reconciliation















    GAAP selling, general and administrative



    $

    53,659





    $

    49,215





    $

    111,869



    $

    89,760



    Less: share-based compensation expense



    8,348





    5,404





    15,389



    9,981



    Non-GAAP selling, general and administrative



    $

    45,311





    $

    43,811





    $

    96,480



    $

    79,779



















     

    PTC Therapeutics, Inc.

    Summary Consolidated Balance Sheets

    (in thousands, except share data)







    June 30, 2020



    December 31, 2019

    Cash, cash equivalents and marketable securities



    $

    498,891





    $

    686,563



    Total Assets



    $

    1,476,637





    $

    1,623,782













    Total debt



    $

    315,362





    $

    313,859



    Total deferred revenue



    7,702





    11,657



    Total liabilities



    $

    904,143





    $

    1,029,452













    Total stockholders' equity (67,240,679 and 61,935,870 common shares issued and outstanding at June 30, 2020 and December 31, 2019 respectively)



    $

    572,494





    $

    594,330



    Total liabilities and stockholders' equity



    $

    1,476,637





    $

    1,623,782













    Today's Conference Call and Webcast Reminder:

    Today's conference call will take place at 4:30 pm (ET) and can be access by dialing (877) 303-9216 (domestic) or (973) 935-8152 (international) five minutes prior to the start of the call and providing the passcode 5897698. A live, listen-only webcast of the conference call can be accessed on the investor relations section of the PTC website at www.ptcbio.com. A webcast replay of the call will be available approximately two hours after completion of the call and will be archived on the company's website for 30 days following the call.

    About PTC Therapeutics, Inc.

    PTC is a science-driven, global biopharmaceutical company focused on the discovery, development and commercialization of clinically differentiated medicines that provide benefits to patients with rare disorders. PTC's ability to globally commercialize products is the foundation that drives investment in a robust and diversified pipeline of transformative medicines and our mission to provide access to best-in-class treatments for patients who have an unmet medical need. To learn more about PTC, please visit us at www.ptcbio.com and follow us on Facebook, on Twitter at @PTCBio, and on LinkedIn.

    For More Information:

    Investors:

    Alex Kane

    +1 (908) 912-9643

    Media:

    Jane Baj

    +1 (908) 912-9167

    Forward Looking Statements:

    This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. All statements contained in this release, other than statements of historic fact, are forward-looking statements, including statements regarding: the future expectations, plans and prospects for PTC, including with respect to the expected timing of clinical trials and studies, availability of data, regulatory submissions and responses and other matters; expectations with respect to PTC's gene therapy platform, including any potential regulatory submissions and manufacturing capabilities; advancement of PTC's joint collaboration program in SMA, including any potential regulatory submissions, commercialization or royalty or milestone payments; PTC's expected use of proceeds from the agreement with Royalty Pharma; PTC's expectations with respect to the licensing, regulatory submissions and commercialization of its products and product candidates; the timing with respect to orders for PTC's products; expectations with respect to the impacts of the COVID-19 pandemic and related response measures; PTC's strategy, future operations, future financial position, future revenues, projected costs; and the objectives of management. Other forward-looking statements may be identified by the words "guidance", "plan," "anticipate," "believe," "estimate," "expect," "intend," "may," "target," "potential," "will," "would," "could," "should," "continue," and similar expressions.

    PTC's actual results, performance or achievements could differ materially from those expressed or implied by forward-looking statements it makes as a result of a variety of risks and uncertainties, including those related to: the outcome of pricing, coverage and reimbursement negotiations with third party payors for PTC's products or product candidates that PTC commercializes or may commercialize in the future; expectations with respect to PTC's gene therapy platform, including any potential regulatory submissions and potential approvals, manufacturing capabilities and the potential financial impact and benefits of its leased biologics manufacturing facility and the potential achievement of development, regulatory and sales milestones and contingent payments that PTC may be obligated to make; the enrollment, conduct, and results of ongoing studies under the SMA collaboration and events during, or as a result of, the studies that could delay or prevent further development under the program, including any potential regulatory submissions and potential commercialization with respect to risdiplam; PTC's ability to complete a dystrophin study necessary to support a re-submission of its Translarna NDA for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) to the FDA, and PTC's ability to perform any necessary additional clinical trials, non-clinical studies, and CMC assessments or analyses at significant cost; PTC's ability to maintain its marketing authorization of Translarna for the treatment of nmDMD in the European Economic Area (EEA), including whether the European Medicines Agency (EMA) determines in future annual renewal cycles that the benefit-risk balance of Translarna authorization supports renewal of such authorization; PTC's ability to enroll, fund, complete and timely submit to the EMA the results of Study 041, a randomized, 18-month, placebo-controlled clinical trial of Translarna for the treatment of nmDMD followed by an 18-month open-label extension, which is a specific obligation to continued marketing authorization in the EEA; expectations with respect to the commercialization of Tegsedi and Waylivra; the enrollment, conduct and results of PTC's PTC299 clinical trial for COVID-19; expectations with respect to the COVID-19 pandemic and related response measures and their effects on PTC's business, operations, clinical trials, potential regulatory submissions and approvals, and PTC's collaborators, contract research organizations, suppliers and manufacturers; significant business effects, including the effects of industry, market, economic, political or regulatory conditions; changes in tax and other laws, regulations, rates and policies; the eligible patient base and commercial potential of PTC's products and product candidates; PTC's scientific approach and general development progress; PTC's ability to satisfy its obligations under the terms of the lease agreement for its leased biologics manufacturing facility; the sufficiency of PTC's cash resources and its ability to obtain adequate financing in the future for its foreseeable and unforeseeable operating expenses and capital expenditures; and the factors discussed in the "Risk Factors" section of PTC's most recent Quarterly Report on Form 10-Q and Annual Report on Form 10-K, as well as any updates to these risk factors filed from time to time in PTC's other filings with the SEC. You are urged to carefully consider all such factors.

    As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commercialization of new products. There are no guarantees that any product will receive or maintain regulatory approval in any territory, or prove to be commercially successful, including Translarna, Emflaza, risdiplam, Tegsedi, Waylivra or PTC-AADC.

    The forward-looking statements contained herein represent PTC's views only as of the date of this press release and PTC does not undertake or plan to update or revise any such forward-looking statements to reflect actual results or changes in plans, prospects, assumptions, estimates or projections, or other circumstances occurring after the date of this press release except as required by law.

    Cision View original content:http://www.prnewswire.com/news-releases/ptc-therapeutics-reports-second-quarter-2020-financial-results-and-provides-a-corporate-update-301107027.html

    SOURCE PTC Therapeutics, Inc.

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  4. SOUTH PLAINFIELD, N.J., July 22, 2020 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ:PTCT) today announced that the Company will host a webcast conference call to report its second quarter 2020 financial results and provide an update on the company's business and outlook on Wednesday, August 5, 2020 at 4:30 p.m. (ET) after the closing of the market.

    The call can be accessed by dialing (877) 303-9216 (domestic) or (973) 935-8152 (international) five minutes prior to the start of the call and providing the passcode 5897698. A live, listen-only webcast of the conference call can be accessed on the investor relations section of the PTC website at www.ptcbio.com. A webcast replay of the call will be available approximately two hours after completion…

    SOUTH PLAINFIELD, N.J., July 22, 2020 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ:PTCT) today announced that the Company will host a webcast conference call to report its second quarter 2020 financial results and provide an update on the company's business and outlook on Wednesday, August 5, 2020 at 4:30 p.m. (ET) after the closing of the market.

    The call can be accessed by dialing (877) 303-9216 (domestic) or (973) 935-8152 (international) five minutes prior to the start of the call and providing the passcode 5897698. A live, listen-only webcast of the conference call can be accessed on the investor relations section of the PTC website at www.ptcbio.com. A webcast replay of the call will be available approximately two hours after completion of the call and will be archived on the company's website for 30 days following the call.

    About PTC Therapeutics, Inc.

    PTC is a science-driven, global biopharmaceutical company focused on the discovery, development and commercialization of clinically differentiated medicines that provide benefits to patients with rare disorders. PTC's ability to globally commercialize products is the foundation that drives investment in a robust and diversified pipeline of transformative medicines and our mission to provide access to best-in-class treatments for patients who have an unmet medical need.

    FOR MORE INFORMATION PLEASE CONTACT:

    Media:                             

    Investors:

    Jane Baj                           

    Alex Kane

    +1 (908) 912-9167             

    +1 (908) 912-9643

                          

     

    Cision View original content:http://www.prnewswire.com/news-releases/ptc-therapeutics-to-host-conference-call-to-discuss-second-quarter-2020-financial-results-301096391.html

    SOURCE PTC Therapeutics, Inc.

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  5. SOUTH PLAINFIELD, N.J., July 21, 2020 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ:PTCT) today announced that it will host the second webinar in its deep dive series discussing its therapeutic platforms and programs on Friday, July 24 at 10:00 am ET.

    The splicing platform deep dive will provide an overview of PTC's validated splicing platform which leverages its cutting-edge technology to selectively and specifically identify molecules that modulate mRNA splicing to upregulate or downregulate protein production. The deep dive will review the mechanisms underlying the splicing platform obtained over 10 years of development that have driven the platform's progress to date and yielded PTC's discovery of risdiplam. PTC will also provide a clear roadmap for the vast potential of the splicing platform moving forward.

    The webinar can be accessed by dialing (877) 303-9216 (domestic) or (973) 935-8152 (international) five minutes prior to the start of the webinar and providing the passcode 9153632. A live, listen-only webcast can be accessed on the Events and Presentations page under the investor relations section of PTC Therapeutics' website at www.ptcbio.com. A webcast replay will be available approximately two hours after completion of the webinar and will be archived for 30 days following the webinar.

    About the Splicing Platform

    PTC uses its alternative splicing technology to identify molecules that modulate mRNA splicing. Approximately 94% of all human genes undergo splicing. Through alternative splicing, by including or excluding exons, one gene can generate several mRNA products that can have different biological functions. PTC has developed a powerful high-throughput drug discovery technology that enables us to identify small molecule modifiers of pre-mRNA splicing. Using this technology, we have successfully identified oral small molecules to upregulate splicing of a key protein for spinal muscular atrophy. Based on the knowledge and experience from the SMA program, we believe that other small molecule drug candidates can be identified that modify alternative splicing of genes, promote inclusion of specific exons into mRNA or force skipping of undesired exons from the mature mRNA. We believe that this technology has a broad potential applicability to a large number of target genes in all therapeutic areas.

    About PTC Therapeutics, Inc.

    PTC is a science-driven, global biopharmaceutical company focused on the discovery, development and commercialization of clinically differentiated medicines that provide benefits to patients with rare disorders. PTC's ability to globally commercialize products is the foundation that drives investment in a robust and diversified pipeline of transformative medicines and our mission to provide access to best-in-class treatments for patients who have an unmet medical need. To learn more about PTC, please visit us at www.ptcbio.com and follow us on Facebook, on Twitter at @PTCBio, and on LinkedIn.

    For More Information:

    Investors:

    Alex Kane

    +1 (908) 912-9643

    Media:

    Jane Baj

    +1 (908) 912-9167

     

    Forward-Looking Statements:

    This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. All statements contained in this release, other than statements of historic fact, are forward-looking statements, including statements regarding: the future expectations, plans and prospects for PTC, PTC's strategy, future operations, future financial position, future revenues, projected costs; and the objectives of management. Other forward-looking statements may be identified by the words "guidance", "plan," "anticipate," "believe," "estimate," "expect," "intend," "may," "target," "potential," "will," "would," "could," "should," "continue," and similar expressions.

    PTC's actual results, performance or achievements could differ materially from those expressed or implied by forward-looking statements it makes as a result of a variety of risks and uncertainties, including those factors discussed in the "Risk Factors" section of PTC's most recent Quarterly Report on Form 10-Q and Annual Report on Form 10-K, as well as any updates to these risk factors filed from time to time in PTC's other filings with the SEC. You are urged to carefully consider all such factors.

    As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commercialization of new products. There are no guarantees that any product will receive or maintain regulatory approval in any territory, or prove to be commercially successful.

    The forward-looking statements contained herein represent PTC's views only as of the date of this press release and PTC does not undertake or plan to update or revise any such forward-looking statements to reflect actual results or changes in plans, prospects, assumptions, estimates or projections, or other circumstances occurring after the date of this press release except as required by law.

     

    Cision View original content to download multimedia:http://www.prnewswire.com/news-releases/ptc-therapeutics-to-host-deep-dive-webinar-on-splicing-platform-301097046.html

    SOURCE PTC Therapeutics, Inc.

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