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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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August 27, 2015
10:57 EDTPTCTOptions with increasing implied volatility
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07:51 EDTRHHBYRoche catching up fast to Bristol-Myers in immuno-oncology, says Jefferies
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August 26, 2015
10:36 EDTPTCTOptions with increasing implied volatility
Options with increasing implied volatility: UVXY PTCT VIX SVXY SUNE SQQQ SPXS LPI
August 25, 2015
09:59 EDTPTCTOn The Fly: Analyst Initiation Summary
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08:01 EDTPTCTPTC Therapeutics initiated with a Buy at Citi
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August 24, 2015
15:44 EDTPTCTOptions with increasing implied volatility
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12:01 EDTRHHBYRoche buys nucleic acid purification technology from Lumora
Roche has signed a definitive asset purchase agreement with Lumora for products associated with the patent-protected Heat Elution technology for nucleic acid purification of multiple sample types including challenging formalin-fixed paraffin-embedded tumor samples. Roche plans to explore integrating this technology into its sequencing workflow solution.
10:12 EDTPTCTOptions with increasing implied volatility
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August 23, 2015
12:35 EDTRHHBYBiotech firms could see 'billions' in sales from new cancer drugs, Barron's says
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August 21, 2015
10:36 EDTPTCTOptions with increasing implied volatility
Options with increasing implied volatility: PTCT UVXY VIX VXX SVXXY SRPT TZA UPRO VNR SPXU
09:38 EDTRHHBYUBS global healthcare analysts hold an analyst/industry conference call
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August 19, 2015
09:17 EDTPTCT'Female Viagra' approval seen as positive for Sarepta, BioMarin
The FDA's approval of the world's first drug designed to boost a woman's sexual desire is seen as boding well for companies awaiting critical decisions by the agency. FEMALE VIAGRA: The Food and Drug Administration yesterday approved flibanserin to treat acquired, generalized hypoactive sexual desire disorder in premenopausal women. The drug, which will be sold under the name "Addyi" and is commonly referred to as the "female Viagra," is the first FDA-approved treatment for sexual desire disorders in men or women, the agency prominently stated in its press release. HSDD is characterized by low sexual desire that causes marked distress or interpersonal difficulty and is not due to a co-existing medical or psychiatric condition, problems within the relationship, or the effects of a medication or other drug substance, the FDA explained. Addyi was approved with a risk evaluation and mitigation strategy as well as a Boxed Warning given the risk of severe hypotension and syncope in patients who drink alcohol during treatment. The drug is made by privately held Sprout Pharmaceuticals. ANALYST REACTION: Addyi's approval is "another sign of flexibility" by the FDA, especially for first-in-class therapies, Roth Capital analyst Debjit Chattopadhyay writes this morning in a note to investors. This bodes well for the approval of eteplirsen, drisapersen and Translarna, the analyst contends. Eteplirsen, designed by Sarepta Therapeutics (SRPT), and drisapersen, designed by BioMarin (BMRN), are experimental drugs being developed to treat Duchenne muscular dystrophy. Translarna, designed by PTC Therapeutics (PTCT), is already being sold in Europe as a treatment for DMD and is awaiting approval from the FDA. Duchenne muscular dystrophy is a form of muscular dystrophy affecting around 1 in 3,600 boys, which results in muscle degeneration and premature death. SAVING LIVES: While female sexual dysfunction is a clinical problem, eteplirsen is designed to save lives, Chattopadhyay points out. He has a Buy rating on Sarepta and noted his price target of $45 assumes accelerated approval for eteplirsen in the first quarter of 2016. Shares of the Massachusetts-based biopharmaceutical company closed yesterday at $35.69.
08:29 EDTPTCTFlibanserin approval bodes well for Sarepta, others, says Roth Capital
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August 17, 2015
05:14 EDTRHHBYGenentech reports atezolizumab pivotal Phase II study met primary endpoint
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August 13, 2015
10:53 EDTPTCTOptions with increasing implied volatility
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06:16 EDTRHHBYRoche acquires GeneWEAVE to bolster offerings in microbiology diagnostics
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