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Please watch as PTC Chief Scientific Officer Mark Pykett explains our therapeutic approach to treating rare disease through the innovative science of gene therapy. “Gene therapy provides a natural approach to treating diseases because it uses the natural gene to restore function that’s been lost or compromised. Additionally, for many diseases, including ones of the central nervous system – which is a major focus for PTC, the treatments may be long-lasting because the cells that receive the gene therapy do not divide much, so they will tend to retain the gene for long periods, thereby providing durable treatment.” Learn more.
PTC Chief Development Officer Matthew B. Klein, MD, MS, FACS explains how our “repurposed cancer drug” PTC299 can provide a “dual mechanism of action targeting the two critical elements of COVID-19 (coronavirus)... This novel, dual mechanism of action is the main way that PTC299 stands out from other drugs currently being repurposed or developed against Covid-19 according to Klein. However, this is not where the promise of PTC299 ends." He also explains a key advantage of PTC299 with the potential to reduce burdens on our health care system.
How can PTC299 potentially treat the SARS-COV-2 virus which causes COVID-19 (coronvirus), and how is PTC ready to advance this investigative therapy? Read more from PTC SVP, Head of International Operations Adrian Haigh. "Patients are not only depending on us for a continued supply of their medicines, the world is looking to us to find a solution. I must have been asked a dozen times by friends and family 'when are you guys going to come up with a cure?' "So, last week I was elated when PTC got the green light from the U.S. FDA to start testing PTC299 in patients hospitalized with COVID-19. "PTC299 ticks all the boxes related to repurposing a tried and tested drug to treat COVID-19. Originally developed to treat cancer because it inhibits a cellular enzyme, dihydroorotate dehydrogenase (DHODH), PTC299 offers a novel dual mechanism of action that has the potential to address the two critical elements of COVID-19: viral replication and 'dampening down' the cytokine storm that leads to the severe complications seen with COVID-19."
PTC Therapeutics CEO Stuart Peltz, Ph.D. recently contributed his thoughts on diversity and inclusion to the MassBio blog. For Stu, diversity and innovation in biotechnology go hand in hand. Read more about why we all can (and should!) make a commitment to inclusion.
PTC Therapeutics, Inc. is excited to announce that the U.S. FDA has authorized the initiation of a Phase 2/3 trial to investigate PTC299, a dihydroorotate dehydrogenase (DHODH) inhibitor, as a potential treatment for COVID-19. PTC299 is an oral investigational drug with a novel dual-mechanism of action that has the potential to address the two critical elements of COVID-19: 1) the high viral replication and 2) the uncontrolled inflammatory response that ensues after infection. PTC will initiate an integrated Phase 2/3 study in the U.S. in the coming days with additional sites planned globally. PTC299 is an oral, small molecule tablet that inhibits the cellular enzyme DHODH which is used to produce the RNA building blocks for the production of SARS-CoV-2. PTC299 has demonstrated potent inhibition of viral replication in SARS-CoV-2 cell based assays, which has led to PTC moving rapidly into the clinic.
PTC Therapeutics is excited to announce the launch of the Talent Pipeline Program (TPP), a one-year global internship program aimed at providing recent graduates real-world experience in the biopharmaceutical industry and related professions, including research, finance, commercial, compliance, quality, legal, information technology, and communications. Throughout the immersive internship program, participants will be provided mentoring, job coaching, career counseling, and leadership training. The Talent Pipeline Program will provide approximately 30 interns a one-year program of real-world training experience and will provided a stipend. Certain roles will require participants to live near PTC's research, technical operations, or manufacturing sites in New Jersey and California. Participants working in other roles will be virtually based and can be living anywhere. Successful participants will be provided a Certificate of Completion, a letter of reference, and consideration for future positions at PTC Therapeutics. Apply or learn more about the program online.
We're growing - we're building a patient-centric rare disease company. "(T)he experience of being deeply enmeshed with our patient community has given us perspective on the concept of patient-centered medicine that is irreplaceable. Each step of our journey that we have taken has been with patients, their families and their physicians." Learn more about this approach from PTC CEO Stuart Peltz, Ph.D.
PTC ($PTCT) announced we have entered into an agreement to acquire Censa Pharmaceuticals, a biopharmaceutical company focused on the development of CNSA-001 (sepiapterin). "Results from a Phase 2 clinical trial of CNSA-001 demonstrated significant and clinically relevant reductions in phenylanaline levels compared to current first-line treatment," said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics, Inc. "We believe that CNSA-001 has the potential to address the majority of PKU patients whose condition is not adequately managed by current treatments. We look forward to initiating a Phase 3 study in PKU so that patients diagnosed with this devastating condition can have a new oral treatment option as soon as possible." Read more about how this acquisition diversifies and strengthens our rare disease portfolio.
PTC continues to grow and innovate in the #raredisease space, even during the #coronavirus pandemic. We recently disclosed positive results for risdiplam in both the SUNFISH pivotal study for Type 2 and 3 SMA patients, and the FIREFISH pivotal study in Type 1 SMA #patients. We had strong year-over-year first quarter growth with our Duchenne muscular dystrophy franchise. We also expect to file the biologics license application with the FDA for our aromatic l-amino acid decarboxylase (AADC) deficiency #genetherapy program later this year. Listen to our 2020 Q1 earnings audio recording or view the presentation online for details
Last year, PTC partnered with a local organization in Switzerland to help a young person with community integration through a professional and personal development program at PTC. We are extremely proud of our newly-integrated team member’s achievements, and this person is equally positive about future prospects in this new home country. As we expand our global footprint and grow our team, we continue to grow our cause in rare diseases. And when we challenge ourselves every day to make a positive impact in our local communities, it always starts and ends with people.
