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News Breaks
August 7, 2014
14:05 EDTRHHBY, PTCTPTC Therapeutics reports publication of preclinical study results in SMA models
PTC Therapeutics (PTCT) announced that he journal Science published results of a preclinical study demonstrating that treatment with orally available RNA splicing modifiers of the SMN2 gene starting early after birth is preventing deficits in mouse models of Spinal Muscular Atrophy or SMA. Stuart Peltz, CEO of PTC Therapeutics, said, "Using the experience and expertise in RNA biology we have gained at PTC over the last 16 years, we used our alternative splicing technology to identify and subsequently optimize investigational compounds that target the SMN2 splicing to produce the SMN protein. Our unique partnership with Roche and the SMA Foundation has allowed this project to rapidly move into clinical development." Luca Santarelli, Head of Neuroscience, Ophthalmology and Rare Diseases at Roche (RHHBY), said, "Although still preclinical, these results demonstrate how SMN2 splicing modifiers could correct the molecular deficit that causes SMA. This study represents an important step towards developing a potential therapeutic option for this devastating and currently untreatable condition. Early clinical trials are currently underway to determine the safety and tolerability of this approach."
News For PTCT;RHHBY From The Last 14 Days
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July 2, 2015
13:59 EDTPTCTNHS to wait on NICE to make call on PTC Therapeutics, BioMarin drugs
NHS England has set out its planned investment decisions for certain specialized services as part of its annual commissioning round. NHS England has agreed with the recommendations from CPAG that final funding decisions on the following two treatments should be made after NICE has concluded its Highly Specialized Technology Appraisal process: BioMarin Pharmaceutical's (BMRN) Vimizim for Morquio A Syndrome and PTC Therapeutics' (PTCT) Translarna for Duchenne muscular dystrophy. The decisions on those two drugs came among 39 proposed new investments that the group said were "carefully considered against the principles NHS England follows when making investment decisions." Reference Link
July 1, 2015
08:46 EDTRHHBYExelixis says FDA extends PDUFA action date for cobimetinib NDA
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June 30, 2015
05:30 EDTRHHBYGenentech reports ocrelizumab Phase III studies met primary, secondary endpoints
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June 25, 2015
14:03 EDTRHHBYRoche initiated with a Buy at HSBC
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June 24, 2015
09:07 EDTRHHBYCAR-T cancer drug researchers seen as M&A targets, Bloomberg says
Juno Therapeutics (JUNO), Kite Pharma (KITE) and Bluebird Bio (BLUE) are trading well above their IPO levels as takeovers predictions have spurred interest and share increases for the developers of CAR-T gene therapies, said Bloomberg's "Real M&A" column, quoting Dimo Dimov, a professor at the University of Bath’s School of Management, as having said that companies such as Pfizer (PFE), Merck (MRK), Roche (RHHBY) and AstraZeneca (AZN) are "closely watching" the firms. Cellectis (CLLS), which is working with Pfizer on its own approach to CAR-T therapy, has said it wants to bring its therapy to market and doesn’t anticipate a sale, the report noted. Reference Link
June 23, 2015
07:49 EDTPTCTPTC Therapeutics risk lowered by symposium, says Oppenheimer
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