Ptc Therapeutics, Inc. · NASDAQ:PTCT
Ptc Therapeutics — PPMD 2017 Connect Conference
23 slides from Ptc Therapeutics's conference presentation, Jun 30, 2017
Slide 1: Accessing EMFLAZA (deflazacort) for Duchenne Muscular Dystrophy
Accessing EMFLAZA™ (deflazacort) for Duchenne muscular Dystrophy PPMD 2017 Connect Conference June 29, 2017; 4:00pm Stuart Peltz, PhD PTC Therapeutics, South Plainfield, NJ
Slide 2: Forward Looking Statements
Forward looking statements within the meaning of The Private Securities Litigation Reform Act of 1995 All statements, other than those of historical fact, contained in this presentation, are forward-looking statements, including statements regarding: the future expectations, plans and prospects for
Slide 3: PTC Therapeutics, Inc.
PTC Therapeutics, Inc. Founded 20 years ago Now has a global footprint with US headquarters located in NJ Focused on discovery, development and commercialization of oral therapies for rare diseases Multiple programs across genetic disorders and oncology
Slide 4: Our Mission
Our Mission To leverage our knowledge of RNA biology to bring novel therapeutics to patients affected by rare and neglected disorders
Slide 5: Commitment to patients with rare and neglected diseases
Commitment to patients with rare and neglected diseases
Slide 6: PTC – A history steeped in commitment to DMD
PTC– A history steeped in commitment to DMD FDA PDUFA IN OCT AdCom Sept. 28 Translarna™ discovery Phase 2a (004) 38 patients Phase 1 62 healthy volunteers Phase 2b (007) 174 patients Initial Natural History Publications 19 years of research and development 1,000+ healthy volunteers / patients expose
Slide 7: DMD Milestones are Irreversible; drugs that slow progression change the disease course
DMD Milestones are Irreversible; drugs that slow disease progression change the course of the disease 0 5 10 15 20 25 30 Years Loss of Ambulation Death Loss of Stair Climb Loss of Stair Descend Early Physical Manifestations Late Physical Manifestations Ventilation Loss of Upper Limb Impaired ability
Slide 8: Targeting the inflammatory component of DMD
Targeting the inflammatory component of DMD DUCHENNE MUSCULAR DYSTROPHY Immune-mediated pathology in Duchenne muscular dystrophy Amy S. Rosenberg,1* Montserrat Puig,1 Kanneboyina Nagaraju,2 Eric P. Hoffman,2 S. Armando Villalta,3 V. Ashutosh Rao,1 Lalage M. Wakefield,4 Janet Woodcock1 Immunological
Slide 9: Acquired in April, 2017
Acquired in April, 2017 Acquisition based on our understanding of the results from the placebo arm from our Randomized Control Trials
Slide 10: EMFLAZA indication is regardless of genetic mutation
EMFLAZA™ indication is regardless of genetic mutation Received FDA approval on February 9, 2017 EMFLAZA™ Improved muscle strength and slowed disease progression We believe that EMFLAZA™ should be available for Duchenne patients as part of their totality of care EMFLAZA™ is the first FDA appr
Slide 11: EMFLAZA (deflazacort)
EMFLAZA™ (deflazacort) EMFLAZA is indicated for the treatment of Duchenne muscular dystrophy in patients 5 years of age and older. Contraindication: Do not use if you are allergic to deflazacort or any of the inactive ingredients in EMFLAZA. Please see Indication & Important Safety Information for E
Slide 12: EMFLAZA strategic rationale
EMFLAZA™ strategic rationale Aligns with mission to bring important therapies to patients with rare diseases Continues PTC’s commitment to DMD patients and families Establishes U.S. commercial footprint for potential ataluren launch if FDA approved A second commercial DMD therapy that is com
Slide 13: Treatment guidelines recommend corticosteroids as standard of care for Duchenne
Treatment guidelines recommend corticosteroids (CS) as part of standard of care for Duchenne CS are the only medication currently available that slows the decline in muscle strength and function in Duchenne muscular dystrophy regardless of mutation CS help reduce the risk of scoliosis and stabilize
Slide 14: EMFLAZACares patient support program
A service program providing personalized support and resources to help you gain access to EMFLAZA
Slide 15: Enroll in EMFLAZACares
Enroll in EMFLAZACares™ Healthcare provider completes the Prescription Start Form, which serves as the prescription Patient/guardian signs the consent section of the Prescription Start form The form is faxed to EMFLAZACares™ with copies of both sides of insurance and prescription benefit cards
Slide 16: Work with an EMFLAZACares Case Manager
Work with an EMFLAZACares™ Case Manager EmflazaCares Case Manage Out-of-pocket Assistance program Bridging program Patient Assistance program Patient foundation support (e.g., Assistance Fund, NORD) Patient financial assistance* *to eligible participants
Slide 17: Prescription Assistance Program
Prescription Assistance Program • For patients with commercial insurance and out-of-pocket costs associated with EMFLAZA who qualify, EMFLAZACares™ offers a copay assistance program • For patients who need additional assistance or are recipients of state or federal insurance and qualify for assistan
Slide 18: Successfully delivering EMFLAZA to patients
Successfully delivering EMFLAZA to patients since May Growing number of prescriptions ~700 patients have received EMFLAZA via either commercial or bridge supply Out-of-pocket Assistance program Bridging program PAP program Patient foundation support (e.g., Assistance Fund, NORD) Patients in 40 state
Slide 19: Prescribing and access takes 4 steps
Fill out the Prescription Start Form with signatures from both the healthcare provider and patient/guardian and fax to EMFLAZACares™ Case manager at EMFLAZACares™ works with insurers and patient assistance programs to get coverage with little to no out-of-pocket cost EMFLAZA is delivered to the pati
Slide 20: Contact EMFLAZACares
A service program providing personalized support and resources to help you gain access to EMFLAZA Contact EMFLAZACares™ Phone: 1-844-EMFLAZA (1-844-363-5292) Fax: 1-844-322-9980 EMFLAZA.com
Slide 21: Important Safety Information
Contraindication: Do not use if you are allergic to deflazacort or any of the inactive ingredients in EMFLAZA. Do not stop taking EMFLAZA, or change the amount you are taking, without first checking with your healthcare provider, as there may be a need for gradual dose reduction to decrease the
Slide 22: Important Safety Information (continued)
Effects on Bones: There is a risk of osteoporosis or decrease in bone mineral density with prolonged use of EMFLAZA, which can potentially lead to vertebral and long bone fractures. Effects on Growth and Development: Long-term use of corticosteroids, including EMFLAZA may slow growth and develop
Slide 23: Contact EMFLAZACares
A service program providing personalized support and resources to help you gain access to EMFLAZA Contact EMFLAZACares™ Phone: 1-844-EMFLAZA (1-844-363-5292) Fax: 1-844-322-9980 EMFLAZA.com