Travere Therapeutics, Inc. · NASDAQ:TVTX
Travere Therapeutics — Retrophin — Merger or Acquisition (February 2014)
16 slides from Travere Therapeutics's merger presentation, Feb 13, 2014
Slide 1: Retrophin: Manchester Pharmaceuticals Acquisition
Retrophin Manchester Pharmaceuticals Acquisition February 13, 2013
Slide 2: Forward-Looking Statements
Forward-Looking Statements This presentation contains forward-looking statements, including statements about our prospects, competitive position, regulatory filings and agency actions, and the anticipated development, timing, data readouts and therapeutic scope of programs in our clinical pipeline.
Slide 3: Acquisition — Manchester Pharmaceuticals
Acquisition — Manchester Pharmaceuticals Pre- Phase! Phase Il Phase Ill Market clinical _ Chenodal Gallstones (Chenodal} Cerebrotendinous xanthomatosis Orphan Drug Designation Hypertension Vecamyl Rage Disorders
Slide 4: Overview
Overview * Retrophin to acquire Manchester Pharmaceuticals -Privately-held specialty pharmaceutical company with two FDA-approved products = Chenodal® (chenodeoxycholic acid) = Vecamyl® (mecamylamine) * §$62.5m purchase price - $29.5m paid upfront - Remaining payments to be delivered over 2014 - Ong
Slide 5: Chenodal® (chenodeoxycholic acid)
Chenodal® (chenodeoxycholic acid) e Chenodal (CDCA) is a synthetic bile acid approved for the treatment of gallstones, but... «usage is exclusively in cerebrotendinous xanthomatosis (CTX) - CDCA is the standard of care for CTX - Chenodal is the only FDA-approved formulation of CDCA in the U.S. = Man
Slide 6: Cerebrotendinous Xanthomatosis (CTX)
Cerebrotendinous Xanthomatosis (CTX) e Inborn error (autosomal recessive) of metabolism - Mutation in CYP27A (sterol-27-hydroxylase) = This enzyme converts cholesterol to CDCA = CDCA binds to FXR and downregulates CYP7A1, which generates bile acids from cholesterol = Patients with CTX mutations cann
Slide 7: Cerebrotendinous Xanthomatosis (CTX)
Cerebrotendinous Xanthomatosis (CTX) * CTX is a very difficult diagnosis to make - CTX patients begin life with neonatal cholestatic jaundice and refractory diarrhea = These common, non-critical and non-specific symptoms rarely lead to a CTX diagnosis - Disease progression then occurs with juvenile
Slide 8: CTX Epidemiology
CIX Epidemiology e Due to the underdiagnosis of CTX, epidemiology data are limited - Published speculation of 1/1,000,000 to 1/50,000 prevalence e Retrophin believes there are at least 500 - 1,000 U.S. CTX patients - Currently <5% - 10% are diagnosed / treated « Retrophin believes that many CTX pati
Slide 9: Chenodal replacement therapy is functionally curative for CTX patients
« Chenodal replacement therapy is functionally curative for CTX patients - Measured by serum cholestanol - Healthy patients have little-to-no serum cholestanol - After Chenodal treatment, cholestanol drops ~98% « Chenodal was never subjected to a clinical trial for CTX given its off- label discovery
Slide 10: Chenodal Pricing
Chenodal Pricing « Price per patient per year (PPPY) - Wall Street focuses on this « % of healthcare spend - Insurers focus on this US. Cost to Annual Cost/ % of HMOs Generic Revenue HMO Life Covered drug spend PPPY Alternative? Humira 6,668 1,000 24.40 1.334% YES. Abilify 6,076 911 22.23 1.215% YES
Slide 11: Global Opportunity
Global Opportunity ° Current supply of the only other available chenodeoxycholic acid product is limited to ROW markets and is spotty - Several product availability complaints « Retrophin believes a large international opportunity exists - At least 1,000 international patients undiagnosed, untreated
Slide 12: Intellectual Property
Intellectual Property « Retrophin will seek FDA approval for Chenodal in CTX this year - Expect rapid approval and 7-year orphan status e Centric specialty pharmacy distributor - Closed distribution system does not allow for generics to access product for bioequivalence study = ANDA filings are impo
Slide 13: Other Indications
Other Indications « FXR agonism has become a popular MOA and reaches across several therapeutic areas including hepatology and nephrology « Possible additional indications for Chenodal include primary biliary cirrhosis (PBC) and nonalcoholic steatohepatitis (NASH) Obeticholic acid (OCA) Chenodeoxych
Slide 14: Vecamyl has exhibited strong growth
« Vecamyl has exhibited strong growth since its reintroduction to the market with no marketing - Retrophin is aware of off-label use in rage associated with autism spectrum disorder « Retrophin plans to continue to make Vecamyl available but does not intend to engage in any marketing or promotional
Slide 15: Forecasts
Forecasts © 2014 revenue guidance of $10 - $12 million © 2015 revenue guidance of $19 - $21 million » Manchester EBITDA margins of 75-80% Potential peak sales of $100-$250 million We estimate NPV of acquisition of Manchester of at least ~$10 per Retrophin share 15
Slide 16: Thank You!
Thank You!