PTC Therapeutics (PTCT) - NASDAQ
  • Mon, Jul. 25, 11:49 AM
    • The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) informs PTC Therapeutics (PTCT -5.3%) that its renewal assessment procedure for the Translarna (ataluren) marketing authorization will not be completed by mid-year as expected, although the company believes that the current marketing authorization to remain in effect until a decision is announced by the European Commission (EC).
    • The EC initially approved Translarna for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) in 2014 subject to annual renewal and certain conditions.
    • The company is currently recruiting patients in an open-label 10-subject Phase 2 clinical trial assessing Translarna in boys aged 2 to 5 with DMD. The primary endpoint is the number of participants with abnormal laboratory values and/or treatment-related adverse events at week 56. According to ClinicalTrials.gov, the estimated study completion date is October 2017.
    • In Q3 2015, the company submitted a variation to its marketing authorization requesting approval for the use of Translarna in nonsense mutation cystic fibrosis based on a post-hos analysis of its completed Phase 3 study. It no longer expects to receive an opinion from CHMP by mid-year. In addition, there is a substantial likelihood that the results from the ongoing Phase 3 ACT CF trial, expected to be completed in early 2017, will be required for approval.
    • Previously: PTC Therapeutics DMD drug gets conditional nod from European Commission (Aug. 4, 2014)
    | Mon, Jul. 25, 11:49 AM | 2 Comments
  • Thu, Jul. 7, 9:16 AM
    | Thu, Jul. 7, 9:16 AM
  • Thu, Jul. 7, 8:40 AM
    • Micro cap PTC Therapeutics (NASDAQ:PTCT) is up 14% premarket on increased volume in response to its announcement that England's National Health Service (NHS) will provide access to Translarna for ambulatory patients at least five years old with nonsense mutation Duchenne muscular dystrophy (DMD).
    • The Managed Access Agreement (MAA) will be in effect for five years. Final guidance from NHS advisor NICE should be published in a few weeks.
    • Translarna, a protein restoration therapy designed to enable the formation of a functioning protein in patients with genetic disorders caused by nonsense mutation, was approved by the European Commission in August 2014.
    • Previously: Advisor to UK Health Service backs PTC's Translarna; shares up 44% (April 15)
    | Thu, Jul. 7, 8:40 AM | 1 Comment
  • Wed, Jun. 22, 7:21 AM
    • Thinly traded nano cap Pluristem Therapeutics (NASDAQ:PSTI) is up 11% premarket on light volume in response to its announcement of preclinical data that suggest its PLX-PAD cells may be effective in treating Duchenne muscular dystrophy.
    • In a mouse model study, PLX-PAD cells reduced an enzyme called creatine phosphokinase (CPK), a marker of muscle degeneration or injury, by ~50% compared to placebo. In addition, analyses of tissue samples showed reduced levels of inflammation and necrosis (death of most of the cells in an organ or tissue) and evidence of regeneration of muscle tissue.
    • PLX cells are mesenchymal-like adherent stromal cells derived from full term human placentas that may be administered without the need for HLA matching. According to the company, PLX-PAD (peripheral artery disease) cells have demonstrated the ability to stimulate the growth of new blood vessels in preclinical models inducing muscle tissue regeneration and improving muscle function.
    • PLX-PAD is currently under development for the treatment of critical limb ischemia, intermittent claudication (leg cramps caused by the obstruction of arteries), muscle injury, pulmonary arterial hypertension and preeclampsia.
    • DMD-related tickers: (NASDAQ:SRPT)(NASDAQ:BMRN)(NASDAQ:SMMT)(NASDAQ:PTCT)(NYSE:PFE)(OTCQB:MRNA)
    | Wed, Jun. 22, 7:21 AM | 19 Comments
  • Wed, May 25, 8:28 AM
    • PTC Therapeutics (NASDAQ:PTCT) is up 5% premarket on light volume in an apparent ripple effect from the bullish activity in Sarepta Therapeutics (NASDAQ:SRPT). Investors perceive the news that the FDA needs more time to review Sarepta's NDA for DMD drug eteplirsen as a good omen, driving SRPT up 21% premarket on robust volume.
    • PTC's DMD candidate is Translarna, a protein restoration therapy designed to enable the formation of a functioning protein in patients with genetic disorders caused by nonsense mutation. It was approved in Europe in August 2014 but has yet to get the FDA's nod here.
    • Previously: FDA rejects PTC's Translarna NDA; shares plummet 42% premarket (Feb. 23)
    | Wed, May 25, 8:28 AM | 18 Comments
  • Fri, May 20, 12:48 PM
    | Fri, May 20, 12:48 PM | 2 Comments
  • Fri, Apr. 15, 12:52 PM
    • Micro cap PTC Therapeutics (PTCT +43.5%) jumps on a 5x surge in volume in response to its announcement that the UK's National Institute for Health and Care Excellence (NICE), an advisor to Britain's National Health Service on costs and services, has recommended Translarna (ataluren) for ambulatory patients at least five years old with nonsense mutation Duchenne muscular dystrophy (nmDMD) under a Managed Access Agreement (MAA) with NHS England. Patient access is subject to the finalization of NICE's draft guidance, expected next month.
    • The company and NHS England are finalizing an MAA that outlines the financial and clinical details pertaining to the use of Translarna, including a confidential financial arrangement. NICE will review its guidance in five years after the company collects further data on Translarna's efficacy in nmDMD.
    • The European Commission approved Translarna, a protein restoration therapy designed to enable the formation of a functioning protein in patients with genetic disorders caused by nonsense mutation, in August 2014. Two months ago, though, the FDA rejected the company's marketing application stating it was not complete enough for review.
    • Read now PTC Therapeutics: Discounted To No Enterprise Value Despite Current Sales
    | Fri, Apr. 15, 12:52 PM
  • Fri, Apr. 15, 12:41 PM
    | Fri, Apr. 15, 12:41 PM
  • Wed, Mar. 23, 4:18 PM
    • Prompted by its Translarna regulatory setback in the U.S., PTC Therapeutics (NASDAQ:PTCT) plans to cut its workforce 18%, primarily affecting U.S.-based employees and contractors. It aims to extend its cash runway after the FDA rejected its New Drug Application in late February due to incompleteness. The job cuts should be completed by the end of Q2. The company expects to incur employee severance and benefit costs of ~$2.5M.
    • Previously: FDA rejects PTC's Translarna NDA; shares plummet 42% premarket (Feb. 23)
    | Wed, Mar. 23, 4:18 PM
  • Tue, Mar. 1, 12:48 PM
    | Tue, Mar. 1, 12:48 PM | 3 Comments
  • Thu, Feb. 25, 3:25 PM
    | Thu, Feb. 25, 3:25 PM | 4 Comments
  • Tue, Feb. 23, 12:46 PM
    | Tue, Feb. 23, 12:46 PM
  • Tue, Feb. 23, 9:17 AM
    | Tue, Feb. 23, 9:17 AM
  • Tue, Feb. 23, 9:14 AM
    • PTC Therapeutics (NASDAQ:PTCT) is getting slammed in premarket trading. Shares are down 42% on robust volume in response to its announcement that it received a Refuse to File letter from the FDA regarding its New Drug Application (NDA) seeking clearance of Translarna (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD).
    • Refuse to File means that the application was not complete enough to allow for review. The company is assessing its options.
    • Translarna is a protein restoration therapy designed to enable the formation of a functioning protein in patients with nonsense mutation-caused genetic disorders.
    • This is the second bump in the road for DMD hopefuls. The FDA said no to BioMarin's (NASDAQ:BMRN) Kyndrisa (drisapersen) last month. It's not looking much better for Sarepta's (NASDAQ:SRPT) eteplirsen either. The FDA's briefing document for its upcoming (date unspecified) Ad Comm review is considered overly harsh (bearish) by many observers.
    • Previously: FDA rejects BioMarin's drisapersen for Duchenne muscular dystrophy (Jan. 14)
    • Previously: It ain't lookin' too good for Sarepta's eteplirsen at next week's Ad Comm review; shares down 57% (Jan. 15)
    | Tue, Feb. 23, 9:14 AM | 6 Comments
  • Nov. 25, 2015, 8:02 AM
    • Today will likely be a downer for DMD-related companies in light of BioMarin's (NASDAQ:BMRN) poor showing at yesterday's Ad Comm meeting. In premarket trading, BMRN is down 4% on increased volume, Sarepta Therapeutics (NASDAQ:SRPT) is down 1% on light volume and PTC Therapeutics (NASDAQ:PTCT) is down 12% on light volume (Jefferies just downgraded it to Underperform).
    | Nov. 25, 2015, 8:02 AM | 1 Comment
  • Nov. 24, 2015, 7:22 PM
    • Although it didn't formally vote on whether or not drisapersen should be approved for the treatment of Duchenne muscular dystrophy (DMD), the FDA's Peripheral and Central Nervous System Drugs Advisory Committee was not impressed with the clinical data BioMarin Pharmaceutical (NASDAQ:BMRN) submitted in its New Drug Application (NDA).
    • To the question of whether the issues of discrepant results of the two dosing regimens and lack of statistical proof on the secondary endpoints affect the persuasiveness of Study 1, one committee member voted "yes", nine voted that it "weakened" it and seven voted that it had "no effect."
    • To the question of whether the results of Study 2 strengthened the efficacy evidence of drisapersen (primary endpoint failed to achieve statistical significance and both dosing regimens were numerically inferior to placebo), there were no "yes" votes. Five voted "weaken" and 12 voted "no effect."
    • To the question of whether the results of Study 3 (primary and secondary endpoints failed to achieve statistical significance) undermine or support the findings in Study 1 and Study 2, the vote was zero for "yes", 15 "weaken" and two "no effect."
    • To the question of what impact the dystrophin results (no notable increase observed pre- and post-treatment) have on the interpretation of the clinical results, the vote was zero "yes", six "weaken," 10 "no effect" and one "no vote."
    • With votes like these, there does not appear to be any hope of approval (PDUFA date: Dec. 27). Trading in BMRN was halted all day. When trading resumes tomorrow, expect blood-letting.
    • In after hours trading, Sarepta Therapeutics (NASDAQ:SRPT) is up a fraction while PTC Therapeutics (NASDAQ:PTCT) is down 6%.
    | Nov. 24, 2015, 7:22 PM | 16 Comments
Company Description
PTC Therapeutics, Inc. is a biopharmaceutical company, which is focused on the discovery, development and commercialization of orally administered, small molecule therapeutics targeting an area of RNA biology. The company's preclinical and discovery programs are focused on the development of new... More
Sector: Healthcare
Industry: Biotechnology
Country: United States