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Prosensa to Appoint Michael Wyzga to Supervisory Board

Prosensa Holding N.V. (NASDAQ: RNA), the Dutch biopharmaceutical company focusing on rare diseases with a high unmet medical need, today announced that Michael S. Wyzga has been nominated for appointment to its Supervisory Board at the next shareholders' meeting, scheduled for June 17, 2014.

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Parent Project Muscular Dystrophy Awards UCLA $50,000 Exploratory Grant for Evaluation of Exon Skipping Enhancers in Duchenne

Parent Project Muscular Dystrophy Awards UCLA $50,000 Exploratory Grant for Evaluation of Exon Skipping Enhancers in Duchenne | Duchenne Research | Scoop.it
PPMD announced today that they will award Dr. M. Carrie Miceli and her team at UCLA’s David Geffen School of Medicine and College of Letters and Science, a $5…
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Duchenne muscular dystrophy drug could get OK for U.S. sales in 2016

Duchenne muscular dystrophy drug could get OK for U.S. sales in 2016 | Duchenne Research | Scoop.it
A Northbrook company said it has won fast-track status for approval of a drug that could become the first in the United States to treat Duchenne muscular dystrophy , a fatal muscle disease found most often in boys.
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Sarepta Therapeutics Announces First Patient Dosed in European Phase I/II Study of SRP-4053 in Duchenne Muscular Dystrophy Patients

Sarepta Therapeutics Announces First Patient Dosed in European Phase I/II Study of SRP-4053 in Duchenne Muscular Dystrophy Patients | Duchenne Research | Scoop.it

Sarepta Therapeutics Inc. today announced that it has initiated dosing of SRP-4053 in its first human trial, a Phase I/II study in Duchenne muscular dystrophy (DMD). This multiple-dose study will assess the safety, tolerability, efficacy, and pharmacokinetics of SRP-4053 in DMD patients with genotypes amenable to exon-53 skipping.

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Sarepta Therapeutics Inc - Investors - News Release

Sarepta Therapeutics Inc - Investors - News Release | Duchenne Research | Scoop.it

Sarepta Therapeutics Reports Long-Term Outcomes through 168 Weeks from Phase IIb Study of Eteplirsen in Duchenne Muscular Dystrophy

ParentProjectMD's insight:

“PPMD’s benefit-risk pilot data demonstrated that parents’ highest priority is to slow DMD progression. With this priority in mind, these Phase II data have exceeded our expectations” said Pat Furlong, Founding President and CEO of Parent Project Muscular Dystrophy (PPMD), a nonprofit organization focused on finding a cure for Duchenne muscular dystrophy. “In practical terms, continued ambulation and preservation of respiratory function have immense benefit for individuals with Duchenne. It is important to keep in mind, that individuals participating in this study lived with Duchenne for over 9 years on average prior to receiving eteplirsen, an age when the pathological process that occurs based on the absence of dystrophin is typically well underway. Understanding this, in the eyes of the community these Phase II data are significant and represent an important step toward changing the landscape.”

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Tivorsan Pharmaceuticals Achieves Key Development Milestones under its Parent Project Muscular Dystrophy and Muscular Dystrophy Association Awards

Tivorsan Pharmaceuticals Achieves Key Development Milestones under its Parent Project Muscular Dystrophy and Muscular Dystrophy Association Awards | Duchenne Research | Scoop.it
Tivorsan Pharmaceuticals, Inc. (Tivorsan) today announced that it met its fourth (4th) set of critical milestones under its existing grants from Paren
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PTC Therapeutics Begins Rolling NDA Submission to the FDA for Translarna to Treat Duchenne Muscular Dystrophy (NASDAQ:PTCT)

PTC Therapeutics, Inc. today announced that it has commenced a rolling submission of a New Drug Application (NDA) to the United States Food and Drug Administration (FDA) for Translarna™ for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD). A rolling submission allows completed portions of the application to be submitted and reviewed by the FDA on an ongoing basis. PTC expects to finalize the application in the fourth quarter of 2015 following the completion of the ACT DMD confirmatory Phase 3 clinical trial.

ParentProjectMD's insight:

We are excited that PTC will begin their rolling NDA submission to the FDA for Translarna! There is so much hope for 2015 in this community. Thank you to ALL of the companies that remain dedicated to our mission – end Duchenne.

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PPMD To Collaborate With Catabasis Pharmaceuticals, Inc. On CAT-1004 Development

PPMD To Collaborate With Catabasis Pharmaceuticals, Inc. On CAT-1004 Development | Duchenne Research | Scoop.it
Parent Project Muscular Dystrophy (PPMD) announced today a collaboration with Catabasis Pharmaceuticals, Inc. (Catabasis) to assist in a Phase 2 clinical tri…
ParentProjectMD's insight:

We are excited to announce a collaboration with Catabasis Pharmaceuticals, Inc. to assist in a Phase 2 clinical trial of its CAT-1004 product candidate for the treatment of patients with Duchenne. As part of this collaboration, PPMD will provide funding to support participant travel. The trial is expected to begin in the first half of 2015. Click below to learn more about CAT-1004.

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Parent Project Muscular Dystrophy Awards Akashi Therapeutics, Inc. $500,000 Grant

Parent Project Muscular Dystrophy Awards Akashi Therapeutics, Inc. $500,000 Grant | Duchenne Research | Scoop.it
Parent Project Muscular Dystrophy (PPMD) has awarded Akashi Therapeutics, Inc. (Akashi) a $500,000 grant to fund clinical trials to test the safety and effica…
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TREAT-NMD : Message on Carmeseal-MD™ on behalf of UPPMD and TREAT-NMD

TREAT-NMD : Message on Carmeseal-MD™ on behalf of UPPMD and TREAT-NMD | Duchenne Research | Scoop.it
Advancing diagnosis, care and treatment for people with neuromuscular diseases around the world.
ParentProjectMD's insight:

You may have seen the recent announcement from Phrixus about the availability of Carmaseal(TM) in the European Union (http://bit.ly/15SF1GN). UPPMD and TREAT-NMD have released a comment that provides more information about this topic.

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PPMD and Santhera Pharmaceuticals Team Up on New Benefit/Risk Study Focused on Pulmonary Therapies

PPMD and Santhera Pharmaceuticals Team Up on New Benefit/Risk Study Focused on Pulmonary Therapies | Duchenne Research | Scoop.it
PPMD is proud to announce a collaboration with Santhera Pharmaceuticals on a benefit/risk study that will focus specifically on patient and caregiver preferen…
ParentProjectMD's insight:

We are proud to announce a collaboration with Santhera Pharmaceuticals on a new benefit/risk study that will focus specifically on patient and caregiver preferences regarding pulmonary therapies in the disease, and will be based on data from Santhera’s successful phase III clinical trial of idebenone. 

PPMD will be reaching out to the community in the coming months to participate in this new study. Both patients and caregivers will be able to participate, even if you participated in PPMD’s previous benefit/risk study. We hope you’ll add your experiences so that we can continue to provide the FDA with as much information as possible in order to accelerate the decision-making process!

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Catabasis Pharmaceuticals Receives FDA Orphan Drug Designation for CAT-1004 for the Treatment of Duchenne Muscular Dystrophy | Business Wire

Catabasis Pharmaceuticals Receives FDA Orphan Drug Designation for CAT-1004 for the Treatment of Duchenne Muscular Dystrophy | Business Wire | Duchenne Research | Scoop.it
Catabasis receives Orphan Drug Designation for CAT-1004 for the treatment of DMD. CAT-1004, a NF-KB inhibitor, is expected to enter a Phase 2 clinical
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PTC Therapeutics Announces CEO, Dr. Stuart Peltz Recipient of The New Jersey Technology Council's 2014 Legend of Technology Award (NASDAQ:PTCT)

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Capricor Therapeutics Announces Positive Pre-Clinical Data for Cardiosphere-Derived Cells (CDCs) on Duchenne Muscular Dystrophy Cardiomyopathy

Capricor Therapeutics Announces Positive Pre-Clinical Data for Cardiosphere-Derived Cells (CDCs) on Duchenne Muscular Dystrophy Cardiomyopathy | Duchenne Research | Scoop.it
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Rare feat: BioMarin rewrites playbook for small-market drug approvals

Rare feat: BioMarin rewrites playbook for small-market drug approvals | Duchenne Research | Scoop.it
BioMarin Pharmaceutical looks to Europe to save time and money in developing rare-disease drugs.
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Eli Lilly Tadalafil Phase 3 Trial has Completed Enrollment

Eli Lilly Tadalafil Phase 3 Trial has Completed Enrollment | Duchenne Research | Scoop.it
DuchenneConnect serves as a central hub linking the resources and needs of the Duchenne and Becker muscular dystrophy community: young men with Duchenne; their families and caregivers; and the provider community: clinical care providers, policymakers, industry professionals and the medical research fields.âWe offer registered members resources to: assist with early, appropriate and least invasive diagnosis; explore the benefits and limitations of genetic testing; offer access to resources and services, including care and treatment; and assist in understanding and development of new treatment trials.
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Prosensa Announces Shareholder Approval of Certain Matters Relating to the BioMarin Tender Offer (NASDAQ:RNA)

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Capricor Therapeutics Announces $10 Million Private Placement of Common Stock

Capricor Therapeutics Announces $10 Million Private Placement of Common Stock | Duchenne Research | Scoop.it
ParentProjectMD's insight:

"This capital raise will also fuel a clinical development program in our recently announced program to treat Duchenne Muscular Dystrophy (DMD) associated cardiomyopathy with CDCs, which we hope to be in the clinic in 2015. "

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Heart Failure Drugs Slow Deadly Damage in Duchenne, Offering New Hope

Heart Failure Drugs Slow Deadly Damage in Duchenne, Offering New Hope | Duchenne Research | Scoop.it
In a new study led by Dr. Subha Raman of The Ohio State University Wexner Medical Center, researchers were able to dramatically slow the rate of heart damage…
ParentProjectMD's insight:

Early use of available heart failure drugs slows the progressive decline in heart function before cardiac symptoms are apparent in boys and young men with Duchenne, according to a new study published online by The Lancet Neurology. Thank you for helping PPMD fund this promising research!

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Pfizer Initiates Phase 2 Study of PF-06252616 in Duchenne Muscular Dystrophy

Pfizer Initiates Phase 2 Study of PF-06252616 in Duchenne Muscular Dystrophy | Duchenne Research | Scoop.it
Pfizer Inc. announced today enrollment of the first patient in a multicenter Phase II clinical trial of the investigational compound PF-06252616 in boys with D…
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Summit Receives Regulatory Approval to Initiate a Modified Diet Clinical Trial of SMT C1100 in DMD Patients

Summit Receives Regulatory Approval to Initiate a Modified Diet Clinical Trial of SMT C1100 in DMD Patients | Duchenne Research | Scoop.it

Summit announces that it has received approval from the UK Medicines and Healthcare products Regulatory Agency and the Ethics Review Committee to initiate a Phase 1b modified diet trial of SMT C1100. SMT C1100 is an oral small molecule utrophin modulator in development for the potential treatment of all patients with DMD, regardless of the underlying dystrophin fault causing the disease. This new trial aims to increase the plasma levels of SMT C1100 by providing patients with specific dietary guidance intended to improve drug absorption.  The trial will also evaluate the potential impact that SMT C1100 is having on enzyme markers of muscle health.  Top-line data from the Phase 1b modified diet trial are expected to be reported in mid-2015.   

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Prosensa Wins Scrip 'Biotech Company of the Year' 2014 Award (NASDAQ:RNA)

Prosensa Wins Scrip 'Biotech Company of the Year' 2014 Award (NASDAQ:RNA) | Duchenne Research | Scoop.it

Prosensa Holding N.V. (NASDAQ: RNA), the biopharmaceutical company focusing on RNA-modulating therapeutics for rare diseases with a high unmet medical need, is delighted to have won the Scrip 'Biotech Company of the Year' Award for 2014, announced last week in London, UK.

The prestigious Scrip Awards provide an opportunity to acknowledge and applaud the highest achievers across the biotechnology and pharmaceutical industry, recognizing both corporate and individual accomplishments. The award for 'Biotech Company of the Year' is given to the company which has demonstrated the greatest achievements for the year.

Hans Schikan, CEO of Prosensa, said, "Winning Scrip's 'Biotech Company of the Year' Award is a great honor and a testament to the dedication and hard work of the Prosensa team.  Our drive to succeed is fueled by patients and their families, who continue to inspire us to make innovative therapies available as quickly and efficiently as possible."

The other five finalists were: Ablynx; Horizon Discovery; Isis Pharmaceuticals; Mesoblast; and Silence Therapeutics.

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PTC Therapeutics Announces Launch of Translarna™ (ataluren) in Germany

SOUTH PLAINFIELD, N.J., Dec. 3, 2014 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that Translarna™ (ataluren) is now commercially available to patients in Germany, the first country to launch in the European Union (EU), with first shipments expected to begin this week.  

"The launch of Translarna in Germany marks another exciting milestone for the DMD community," said Stuart W. Peltz, Ph.D., Chief Executive Officer of PTC Therapeutics.  "Through our reimbursed early access programs and commercial efforts, we are working hard to bring Translarna to those who may benefit from this drug. We remain committed to bringing Translarna to patients in Europe, and around the world, as quickly as possible.  We know every day counts for the patients and their families."  

The launch follows the European Commission marketing authorization, which was received in August 2014 for the use of Translarna to treat ambulatory patients with nonsense mutation Duchenne muscular dystrophy who are 5 years and older. A confirmatory Phase 3 clinical trial of Translarna, ACT DMD, in nonsense mutation Duchenne muscular dystrophy patients completed enrollment in September 2014. Top-line data is expected in the fourth quarter of 2015. Currently, Translarna is not approved for use in the United States.

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Sarepta Therapeutics to Host Webcast and Conference Call for the Duchenne Muscular Dystrophy Community on December 2, 2014

Sarepta Therapeutics to Host Webcast and Conference Call for the Duchenne Muscular Dystrophy Community on December 2, 2014 | Duchenne Research | Scoop.it
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BioMarin and Prosensa Holding N.V. Reach Agreement on Intended Public Offer for 100% of Prosensa's Outstanding Stock; Will Add Duchenne Muscular Dystrophy Products to Rare-Disease Portfolio

BioMarin and Prosensa Holding N.V. Reach Agreement on Intended Public Offer for 100% of Prosensa's Outstanding Stock; Will Add Duchenne Muscular Dystrophy Products to Rare-Disease Portfolio | Duchenne Research | Scoop.it

-- Acquisition of Prosensa provides near-term opportunity to commercialize, if approved, its exon-skipping drug candidate, drisapersen, for Duchenne muscular dystrophy (DMD)
-- Drisapersen is currently under a rolling review as part of a New Drug Application process and has Orphan, Fast Track and Breakthrough Therapy designation by the FDA
-- Drisapersen, a potential first-to-market and best-in-class product for treating a large population of patients with a rare, fatal genetic disease represents up to 10,000 DMD patients
-- Follow-on products leveraging Prosensa's same technology platform in the pipeline target an additional 35,000 DMD patients in BioMarin's commercial territories
-- Investor conference call to be held today, November 24, 2014 at 5am PST (8am EST)

 

SAN RAFAEL, Calif., Nov. 24, 2014 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) and Prosensa Holding N.V. (Nasdaq:RNA) today announced that they have entered into a definitive agreement in which BioMarin will offer to purchase all of the outstanding ordinary shares of Prosensa for $17.75 per share, for a total up front consideration of approximately $680 million. In addition, two approximately $80 million contingent milestones are payable for the approval of drisapersen in the U.S. no later than May 15, 2016 and Europe no later than February 15, 2017, respectively.

 

"BioMarin is dedicated to the rare disease community, and the acquisition of Prosensa fits strategically with our mission of delivering therapies that address serious unmet medical needs," said Jean-Jacques Bienaimé, Chief Executive Officer of BioMarin. "We are committed to working closely with regulatory authorities worldwide in bringing a potentially breakthrough therapy to patients with this devastating condition."

 

Mr. Bienaimé continued, "We will leverage our experience at developing rare disease therapies to achieve regulatory approvals and bring drisapersen to market as quickly as possible. Further, if we are successful in advancing drisapersen to early regulatory approvals, we believe this transaction would be accretive to operating and GAAP profitability in 2017."

 

Pat Furlong, President and Founder of Parent Project Muscular Dystrophy said, "BioMarin has a successful track record of developing new therapies for people with devastating disorders and for effectively collaborating with health authorities and patient communities. We look forward to working with BioMarin to bring new treatments to boys with Duchenne and other forms of muscular dystrophy."

 

Under the terms of the definitive agreement, BioMarin will offer to acquire all of Prosensa's issued and outstanding ordinary shares and all ordinary share equivalents in an all cash transaction for $17.75 per share for an upfront purchase price of approximately $680 million. Prosensa shareholders may also receive two regulatory milestone payments of approximately $80 million for receiving approval in the U.S. no later than May 15, 2016 and in Europe no later than February 15, 2017, respectively. In addition, within 5 business days of signing the purchase agreement BioMarin will purchase from Prosensa a $50 million convertible note.  If the transaction fails to close for any reason, the note will automatically convert into 4,395,914 shares of Prosensa's stock.

 

The transaction is expected to be accounted for as a business combination. BioMarin will maintain operations at Prosensa's headquarters, based in Leiden, The Netherlands and integrate Prosensa personnel from that office. 

 

The acquisition will provide BioMarin with worldwide rights to multiple orphan-drug candidates, including drisapersen, which is currently under rolling review as part of a New Drug Application (NDA) with the Food and Drug Administration. Prosensa's pipeline is comprised of several potential products that leverage their proprietary RNA-modulating technology platform for the treatment of various genotypes of Duchenne muscular dystrophy and other genetic disorders.     

 

Hans Schikan, Chief Executive Officer of Prosensa added, "BioMarin has established itself as a leader in rare diseases, characterized by strong management, thorough execution, and a resounding commitment to patients in developing and commercializing treatments where there is a high unmet medical need. This transaction will enhance Prosensa's mission by bringing innovative therapies to patients across the world as quickly and efficiently as possible. The deal also creates shareholder value by positioning Prosensa's strong portfolio of orphan drug candidates for future success with a prominent rare disease company that has the experience and dedication to bring drisapersen and our follow-on compounds to the hands of patients who desperately need them."

 

BioMarin will effect the transaction primarily through a tender offer for all of the issued and outstanding Prosensa ordinary shares (the "Offer") and expect to close in the first quarter of 2015. The commencement of the Offer will be subject to having obtained workers council advice, and the consummation of the Offer is subject to the satisfaction of customary closing conditions for a transaction of this nature, including the tender of at least 80% of the issued and outstanding Prosensa ordinary shares and the receipt of regulatory clearance. Following completion of the Offer, the Supervisory Board of Prosensa will consist of five individuals designated by BioMarin and two individuals who currently serve on the Supervisory Board of Prosensa, who will act as independent directors. The two independent directors will, in accordance with Dutch practice, act as independent supervisory directors to protect the interest of any minority shareholders until BioMarin utilizes certain available reorganization structures available under Dutch law to acquire full ownership of Prosensa's outstanding shares and/or its business. An Extraordinary General Meeting will be convened in connection with the Offer and to adopt, among other things, certain resolutions relating to the reorganization of Prosensa.

ParentProjectMD's insight:

Today, BioMarin Pharmaceuticals announced that it will acquire 100% of Prosensa. As Pat said in today’s press release:

 

“BioMarin has a successful track record of developing new therapies for people with devastating disorders and for effectively collaborating with health authorities and patient communities. We look forward to working with BioMarin to bring new treatments to boys with Duchenne and other forms of muscular dystrophy.”

 

With this news comes question. Pat has requested Biomarin reach out to the Duchenne community to reaffirm their commitment to follow-on exons and shed some light on how/when they will be able to talk about timing and next steps.

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Sarepta Therapeutics Announces First Patient Dosed in Confirmatory Study of Eteplirsen in Ambulant Patients with Duchenne Muscular Dystrophy

Sarepta Therapeutics Announces First Patient Dosed in Confirmatory Study of Eteplirsen in Ambulant Patients with Duchenne Muscular Dystrophy | Duchenne Research | Scoop.it
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