(RTTNews.com) - Shares of PTC Therapeutics Inc. (PTCT) have gained as much as 24% so far this month while the iShares Nasdaq Biotechnology ETF index ( IBB ) is up just 8.6% for the same period.
PTC is a biopharmaceutical company focused on the development of drugs for rare disorders.
The Company has two marketed drugs - Translarna and Emflaza - and is expanding its pipeline through in-house innovation and acquisition.
Translarna is approved in the European Union for ambulatory patients aged 5 years and older with Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene. It is not approved in the U.S. yet.
Emflaza is approved in the US for the treatment of Duchenne muscular dystrophy in patients 5 years of age and older.
The Company is also exploring the potential of Translarna in other indications and the results of the following clinical trials are worth watching.
-- A phase II study of Translarna for nonsense mutation aniridia
-- A phase II study of Translarna for nonsense mutation Dravet syndrome/CDKL5
The DMD franchise consisting of Translarna and Emflaza is expected to put up a good showing in the coming years. Revenue from the two products was $174 million in 2017, and is anticipated to be $260 million to $295 million in 2018.
The Company has also sought to expand the indication of Translarna to include ambulatory children aged two to five years with nonsense mutation Duchenne muscular dystrophy (nmDMD). Last month, the Committee for Medicinal Products for Human Use of the European Medicines Agency recommended approval of this expanded indication. The final decision of the European Commission is expected in the coming months.
Another genetic disease of interest for PTC is spinal muscular atrophy (SMA).
SMA is a rare neuromuscular disorder, and is the number 1 genetic cause of infant death. Based on the age of the initial onset of muscle weakness and severity of the disease, spinal muscular atrophy is classified into four types: Type I (Infantile), Type II (Intermediate), Type III (Juvenile) and Type IV (Adult onset).
PTC has a collaboration with Roche and the SMA Foundation for its spinal muscular atrophy program.
The lead drug candidate under the SMA program is RG7916, which is in three clinical studies:
SUNFISH, a trial in childhood onset (Type II/III) SMA patients, and FIREFISH, a trial in infant onset (Type I) SMA patients. An exploratory study assessing safety and tolerability of RG7916 in people with Type 2 and 3 SMA who have previously taken part in a study with another SMN2-targeting molecule, dubbed JEWELFISH is also underway.
Exploring the gene therapy space...
In a bid to expand and diversify its current pipeline, PTC Therapeutics, on July 19, agreed to acquire Agilis Biotherapeutics Inc., a biotechnology company advancing an innovative gene therapy platform for rare monogenic diseases that affect the central nervous system.
The lead gene therapy candidate of Agilis is GT-AADC in treating Aromatic L-Amino Acid Decarboxylase (AADC) Deficiency. PTC plans to submit a Biologics License Application for GT-AADC to the FDA in 2019.
AADC Deficiency is a rare CNS disorder resulting in breathing, feeding, and swallowing problems, frequent hospitalizations, and the need for life-long care. Many patients die in the first decade of life. Currently, there are no treatment options for this disorder.
*Accumulated deficit: $830 million
*Cash on hand: $178.3 million
Shares Outstanding: 46.45 million
Float: 32.12 million
(* As of March 31, 2018).
Shares of PTC have traded in a range of $14.56 to $52.95 over the last 52 weeks. The stock closed Friday's trading at $39.98, up 9.38%.
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