February 24, 2015
By Mark Terry, BioSpace.com Breaking News Staff
Rumors that South Plainfield, N.J.-based PTC Therapeutics might be acquired by Shire plc or BioMarin Pharmaceutical Inc. caused the company’s stock to jump by 17.7 percent yesterday. Shares sold for a low of $16.50 on May 14 and are currently selling for $64.70.
Unnamed sources indicate that the company is consulting with financial advisers to work out strategic options. PTC has a market valuation of approximately $2 billion.
The company focuses on modulating protein expression within cells. PTC’s proprietary technology platforms allows its scientists to screen its compound library against targets across numerous therapeutic areas. Its drug, Ataluren (Translarna) is licensed for the treatment of Duchenne muscular dystrophy in Europe and for cystic fibrosis caused by a specific nonsense mutation (nmCF).
The drug has been designated an orphan medicinal product by the European Medicines Agency (EMA) and given orphan drug designation by the U.S. Food and Drug Administration (FDA). Ataluren is the only Phase III-approved treatment for Duchenne muscular dystrophy in the world. It is still awaiting Phase III data results.
The trials have had their problems. Ataluren did not meet its primary endpoints in a Phase IIb trial in 2010. This would seem to have killed any chances for early approval in Europe, but the EMA reevaluated and granted conditional approval based on “some evidence of effectiveness” and the fact there was little if any alternative to the drug.
Another company, Sarepta Therapeutics , also has an experimental drug for Duchenne muscular dystrophy, eteplirsen, which many analysts expect to be approved by the FDA soon. It too has received some questions by the FDA, which the company plans to address by mid-2015. That would push back a regulatory approval—if there is one—to early 2016.
Shire has certainly been on a buying binge. It announced today that it acquired San Diego, Calif.-based Meritage Pharma, Inc. for an upfront $70 million with additional milestone payments possible. It completed the purchase of NPS Pharmaceuticals, Inc. on Feb. 21, 2015. Earlier in 2014 South Chicago-based AbbVie was planning to buy Shire, but terminated the deal in October 2014.
Late in 2014 San Rafael, Calif.-BioMarin Pharmaceutical Inc. agreed to buy The Netherlands-based ProSensa Holding N.V. for about $840 million. As part of that deal, BioMarin picked up Prosensa and its RNA-modulating technology platform used for the treatment of several different genotypes of Duchenne muscular dystrophy and other genetic disorders.
The question remains if PTC stock jump is related to anything more than rumors of a sale. There is no indication of good news pending related to PTC’s drug trials, although hope springs eternal.
BioSpace Temperature Poll
Analyst Mark Schoenebaum, a biotech and pharmaceuticals analyst and medical doctor for ISI Group Evercore, has been running a Best Hair in Biopharma contest for several months now. So far, the candidates are Bristol-Myers Squibb Company‘s John Elicker, Receptos’ Chief Executive Officer Faheem Hasnain, Celgene‘s Vice President of Investor Relations Patrick Flanigan and Acorda Therapeutics’ Ron Cohen.
We want to know what our BioSpace community thinks: Who do you believe actually has the Best Hair in BioPharma?
| Read at BioSpace.com |
