Enterprise Value

4.403B

Cash

286.3M

Avg Qtr Burn

-72.19M

Short % of Float

9.97%

Insider Ownership

2.61%

Institutional Own.

-

Qtr Updated

03/31/23


Drug Pipeline

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Drug & IndicationStage & EventCatalyst Date
Emflaza (deflazacort) Details
Duchenne muscular dystrophy

Approved

Quarterly sales

Evrysdi (riskiplam) Details
SMA (Spinal muscular atrophy)

Approved

Quarterly sales

Translarna (ataluren) Details
DMD (Duchenne muscular dystrophy)

Approved

Quarterly sales

Upstaza (PTC-AADC) Details
Aromatic L-amino acid decarboxylase deficiency

BLA

Submission

Phase 3

Update

Vatiquinone (PTC743) Details
Friedreich’s Ataxia

Phase 3

Update

Vatiquinone (PTC743) Details
Epilepsy, Mitochondrial disease

Phase 2/3

Data readout

PTC518 Details
Huntington's disease

Phase 2

Data readout

Failed

Discontinued