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.
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.
Parent Project Muscular Dystrophy (PPMD) announced today a collaboration with Catabasis Pharmaceuticals, Inc. (Catabasis) to assist in a Phase 2 clinical tri…
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.
Advancing diagnosis, care and treatment for people with neuromuscular diseases around the world.
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.
PPMD is proud to announce a collaboration with Santhera Pharmaceuticals on a benefit/risk study that will focus specifically on patient and caregiver preferen…
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!
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…
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.
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
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.
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.
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.
-- 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.
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.
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.”
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.
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
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.
- 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.