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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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Prosensa Announces 3rd Quarter 2014 Financial Results and Recent Corporate Developments (NASDAQ:RNA)

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Pfizer Myostatin Blocker Trial Set to Launch

Pfizer Myostatin Blocker Trial Set to Launch | Duchenne Research | Scoop.it
At the Action Duchenne meeting in London on Saturday, Pfizer Inc. announced that it has completed a study in healthy volunteers with its antibody-based thera…
ParentProjectMD's insight:

Sharon Hesterlee and Annie Kennedy from the PPMD team recently attended the Action Duchenne Conference in London. At the meeting, Pfizer announced that it has completed a study in healthy volunteers with its antibody-based therapeutic to block the muscle protein myostatin, and is preparing to start recruitment for a phase II study in Duchenne this month. Read Sharon and Annie’s report to learn more.

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Idera Pharmaceuticals' (IDRA) CEO Sudhir Agrawal on Q3 2014 Results - Earnings Call Transcript

Idera Pharmaceuticals' (IDRA) CEO Sudhir Agrawal on Q3 2014 Results - Earnings Call Transcript | Duchenne Research | Scoop.it
Idera Pharmaceuticals, Inc. (NASDAQ:IDRA) Q3 2014 Earnings Conference Call November 7, 2014 08:00 AM ET Executives Jim Baker - Executive Director of Corporate Affairs Sudhir Agrawal - CEO Lou Arcudi - CFO Lou Brenner - CMO Ka
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Capricor Therapeutics Reports Third Quarter 2014 Financial & Business Highlights

Capricor Therapeutics Reports Third Quarter 2014 Financial & Business Highlights | Duchenne Research | Scoop.it
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PTC Therapeutics' (PTCT) CEO Stuart Peltz on Q3 2014 Results - Earnings Call Transcript

PTC Therapeutics' (PTCT) CEO Stuart Peltz on Q3 2014 Results - Earnings Call Transcript | Duchenne Research | Scoop.it
PTC Therapeutics, Inc. (NASDAQ:PTCT) Q3 2014 Earnings Conference Call November 6, 2014 09:00 ET Executives Emily Hill - Director, Investor Relations Stuart Peltz - Chief Executive Officer Shane Kovacs - Chief Financial Office
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Cambridge biotech Idera jumps into Duchenne muscular dystrophy field - Boston Business Journal

Cambridge biotech Idera jumps into Duchenne muscular dystrophy field - Boston Business Journal | Duchenne Research | Scoop.it
The biotech has formed a partnership with Parent Project Muscular Dystrophy to conduct early stage research into a drug which would slow the deterioration of muscles caused by Duchenne.
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GEN | Insight & Intelligence™: Community Organizing Meets the Clinic

GEN | Insight & Intelligence™: Community Organizing Meets the Clinic | Duchenne Research | Scoop.it
Registries connect patients with advocates, clinicians, and drug developers.
ParentProjectMD's insight:

As DuchenneConnect turns 7 this year, we are thrilled to see the way this resource has changed the landscape of patient registries. We often hear from families and companies that DuchenneConnect has helped patients and clinical trials find each other. Not only is this site benefiting the Duchenne community, though, it has created a model that other health registries look to. 

If you have not registered on DuchenneConnect or if you have not updated your information in a while, visit http://DuchenneConnect.org today!

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Sarepta Therapeutics Announces Third Quarter 2014 Financial Results and Recent Corporate Developments

Sarepta Therapeutics Announces Third Quarter 2014 Financial Results and Recent Corporate Developments | Duchenne Research | Scoop.it

- Eteplirsen confirmatory study in ambulatory patients and study in advanced/non-ambulatory patients to begin dosing this month -

- New Drug Application submission for eteplirsen planned for mid-year 2015 based on recent FDA guidance -

- Revised 2014 non-GAAP operating loss guidance lowered to $110-$120 million with cash and other investments of $240 million at quarter end -

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Milo Biotechnology makes headway on reversing disease

Milo Biotechnology makes headway on reversing disease | Duchenne Research | Scoop.it
Al Hawkins thinks he has found a way to reverse muscular dystrophy. It's too early to say if he's right, but new evidence suggests that he could be.
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Translarna™ Phase 2b Data Published in Muscle & Nerve

Data Supports Benefit in Nonsense Mutation Duchenne Muscular Dystrophy Patients
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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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PTC Therapeutics Appoints Dr. Tuyen Ong SVP, Head of Clinical Development and Translational Research (NASDAQ:PTCT)

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PTC Therapeutics Recognized for Outstanding Innovation at the 2014 Rare & Orphan Advocacy and Research (ROAR) Awards (NASDAQ:PTCT)

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

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

Sarepta Therapeutics, Inc., a developer of RNA-based therapeutics, today announced that it has initiated dosing in a clinical study of eteplirsen, the Company's lead exon-skipping therapeutic candidate for the treatment of Duchenne muscular dystrophy (DMD), in patients who are non-ambulant or who have advanced DMD and don't meet a minimum 6-minute walk test score at baseline.

 

The open-label study, 4658-204 (Study 204), will include approximately 20 patients treated with eteplirsen who have genotypes amenable to exon 51 skipping and who meet other study inclusion criteria. The study will be conducted at several sites in the United Statesand is designed to evaluate the safety of eteplirsen in DMD patients over 96 weeks of dosing. Patients enrolled in the study will receive once weekly intravenous infusions of 30mg/kg of eteplirsen, and data will be collected across a number of safety parameters and secondary efficacy endpoints.

 

“The initiation of this eteplirsen study represents an important milestone for patients, their families, and the DMD community,” said Edward Kaye, M.D., Sarepta’s Chief Medical Officer. “Expanding the DMD population to include patients who are older and non-ambulant demonstrates our strong commitment to develop eteplirsen for patients at all stages of DMD and will provide additional data to support our planned NDA filing.”

 

Fawn Leigh, M.D., of Mass General Hospital and a principal investigator in the study added, "Eteplirsen is a potential breakthrough treatment for patients with DMD. I am pleased to be able to offer this promising disease-modifying treatment to my patients and to potentially alter the course of this devastating disease.”

 

 

About Eteplirsen

Eteplirsen is Sarepta's lead drug candidate and is designed to address the underlying cause of DMD by enabling the production of a functional dystrophin protein. Data from clinical studies of eteplirsen in DMD patients have demonstrated a broadly favorable safety and tolerability profile and restoration of dystrophin protein expression.

Eteplirsen uses Sarepta's novel phosphorodiamidate morpholino oligomer (PMO)-based chemistry and proprietary exon-skipping technology to skip exon 51 of the dystrophin gene enabling the repair of specific genetic mutations that affect approximately 13 percent of the total DMD population. By skipping exon 51, eteplirsen may restore the gene's ability to make a shorter, but still functional, form of dystrophin from messenger RNA, or mRNA. Promoting the synthesis of a truncated dystrophin protein is intended to stabilize or significantly slow the disease process and prolong and improve the quality of life for patients with DMD. Sarepta is also developing other PMO-based exon-skipping drug candidates intended to treat additional patients with DMD.

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Sarepta Therapeutics' (SRPT) CEO Chris Garabedian on 23rd Annual Credit Suisse Healthcare Conference Call Transcript

Sarepta Therapeutics' (SRPT) CEO Chris Garabedian on 23rd Annual Credit Suisse Healthcare Conference Call Transcript | Duchenne Research | Scoop.it
Sarepta Therapeutics Inc. (NASDAQ:SRPT) 23rd Annual Credit Suisse Healthcare Conference Call November 11, 2014 6:00 PM ET Executives Christopher Garabedian – Chief Executive Officer and President Analysts Jeremiah B. Shepard
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Sarepta’s Safety Study of Eteplirsen to Treat Advanced Stage Duchenne has Initiated Recruitment

Sarepta’s Safety Study of Eteplirsen to Treat Advanced Stage Duchenne has Initiated Recruitment | Duchenne Research | Scoop.it

One site in the U.S. is now actively recruiting for this trial, and Sarepta is working towards the goal of having 9 sites participate in this trial. They plan to continue initiating study sites on a rolling basis between now and early 2015. 

 

The primary objective of this study is to explore safety and tolerability of eteplirsen in patients with advanced stage Duchenne who are amenable to exon 51 skipping. The exploratory objectives are to evaluate the effect of eteplirsen on pulmonary function tests (PFTs) and other functional clinical measures. The study sponsor is Sarepta Therapeutics.

 

Recruitment Criteria:

- Males with a diagnosis Duchenne and a genetic mutation amenable to exon 51 skipping

- Ages 7-21 years old

- Stable corticosteroid therapy for at least 6 months, or no corticosteroid therapy for 6 months

- Non-ambulatory (not able to walk ≥300 meters on the 6-Minute Walk Test)

- Stable pulmonary and cardiac function (predicted FVC equal to or greater than 40% and LVEF equal to or greater than 40%)

- Please see the ClinicalTrials.gov summary for additional inclusion and exclusion criteria

 

The estimated number of participants is 20.  All participants will receive once weekly intravenous (IV) infusions of eteplirsen 30 mg/kg for 96 weeks. 

 

Safety will be regularly assessed throughout the study via the collection of adverse events (AEs), laboratory tests, electrocardiograms (ECGs), echocardiograms (ECHOs), vital signs, and physical examinations. Exploratory efficacy assessments, including PFTs, upper extremity testing, and other measurements of functional status, will occur at functional assessment visits every 12 weeks over the first year of treatment and approximately every 24 weeks over the second year of treatment.

 

 For additional information, including a list of all trial sites, please read the trial summary posted on ClinicalTrials.gov.

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Capricor Therapeutics to Hold Duchenne Muscular Dystrophy Data Presentation Call on November 18, 2014 at 9:30 a.m. ET

Capricor Therapeutics to Hold Duchenne Muscular Dystrophy Data Presentation Call on November 18, 2014 at 9:30 a.m. ET | Duchenne Research | Scoop.it
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Idera Pharmaceuticals and Parent Project Muscular Dystrophy Announce Collaboration to Advance New Treatment Approach for Duchenne Muscular Dystrophy

Idera Pharmaceuticals and Parent Project Muscular Dystrophy Announce Collaboration to Advance New Treatment Approach for Duchenne Muscular Dystrophy | Duchenne Research | Scoop.it
Toll-like receptor antagonism designed to treat muscle inflammation central to disease progressionNew approach offers potential to treat broad population of patients regardless of their genetic mutation
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PTC Therapeutics Reports Third Quarter Financial Results and Provides Corporate Update

PTC Therapeutics Reports Third Quarter Financial Results and Provides Corporate Update | Duchenne Research | Scoop.it
- Confirmatory Phase 3 ACT DMD clinical trial fully enrolled -
- Rolling NDA submission for Translarna in nmDMD planned before year end 2014 -
- European commercial launch activities well underway -
- Initiation of Phase 1b/2a trial for SMA program planned -
- IND application for PTC596 to be submitted before year end 2014 -
- Successful financing raised estimated net proceeds of $117.5 million -
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PTC Therapeutics Expands Access For Translarna™ (Ataluren)

PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that Translarna™ (ataluren) has been approved to be included in the list of drugs reimbursable by the Italian National Health System under Law 648/96. The decision is expected to be formalized through its publication in the official Italian journal, Gazzetta Ufficiale in the coming weeks. A list of drugs reimbursable under this law is published on the Italian Medicines Agency's (AIFA) website.

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PPMD Submits Letter to FDA Regarding Latest Sarepta Update

PPMD Submits Letter to FDA Regarding Latest Sarepta Update | Duchenne Research | Scoop.it
Today’s press release and investor call from Sarepta have sent a ripple of angst and sadness through our community. Moments ago, PPMD sent a letter to the FDA…
ParentProjectMD's insight:

Today’s press release and investor call from Sarepta (http://bit.ly/1xwCbi1) have sent a ripple of angst and sadness through our community.

Moments ago, PPMD sent a letter to the FDA that reflects our organization’s belief that safety and rigor have been demonstrated throughout this process. Further, we believe that any strengthening of data packages that causes delays (as is now being requested by the FDA, as articulated in today’s press release and call) should ensure a streamlined, more expeditious approval process for follow-on exons.

As a community we believe exon-skipping drugs like eteplirsen hold potentially life-altering benefits, and must be moved forward as rapidly as possible. In addition, we believe eteplirsen to be a critical foundational therapy, restoring dystrophin and significantly slowing disease progression. We look forward to the mid-2015 submission and approval of eteplirsen and the consideration of approval of PMO (phosphodiamidate morpholino oligomer) as a class of drugs that will have the ability to slow/halt progression in a great many of the individuals living with Duchenne.

PPMD has also reached out to leadership at Sarepta to support the pathway forward for these trials and to work to ensure the approval and availability of an effective therapy to our Duchenne community at the earliest moment possible. As always we will report back to you with updates on next steps and anything we as a community can do to help this process.

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