Precision NanoSystems & Alnylam Form New Delivery Collaboration

Precision NanoSystems, Inc. and Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, recently announced the two companies have formed an exclusive collaboration focused on the discovery and development of novel lipid nanoparticles, known as small lipid nanoparticles (sLNPs), using microfluidics technology. Based on their small particle size of approximately 20 nanometers, sLNPs have the potential for broadened biodistribution beyond liver delivery.

“We look forward to working with Precision NanoSystems to support research efforts around the discovery of novel sLNPs that we believe have the potential to significantly improve and broaden biodistribution,” said Kenneth Koblan, PhD, Chief Scientific Officer at Alnylam. “sLNPs represent an exciting and innovative approach in Alnylam’s advancement of proprietary LNPs for RNAi therapeutics.”

“We are excited to have formed this exclusive collaboration with Alnylam focused on the discovery and development of novel sLNPs using microfluidics technology,” added James Taylor, PhD, Chief Executive Officer of Precision NanoSystems. “Alnylam is leading the translation of RNAi technology into human therapeutics, and we look forward to working with them.”

Alnylam is a biopharmaceutical company developing novel therapeutics based on RNA interference, or RNAi. The company is leading the translation of RNAi as a new class of innovative medicines with a core focus on RNAi therapeutics for the treatment of genetically defined diseases, including ALN-TTR for the treatment of transthyretin-mediated amyloidosis (ATTR), ALN-PCS for the treatment of severe hypercholesterolemia, and ALN-HPN for the treatment of refractory anemia. As part of its Alnylam 5×15 strategy, the company expects to have five RNAi therapeutic products for genetically defined diseases in advanced stages of clinical development by the end of 2015. Alnylam has additional partner-based programs in clinical or development stages, including ALN-RSV01 for the treatment of respiratory syncytial virus (RSV) infection, ALN-VSP for the treatment of liver cancers, and ALN-HTT for the treatment of Huntington’s disease.

The company’s leadership position on RNAi therapeutics and intellectual property have enabled it to form major alliances with leading companies, including Merck, Medtronic, Novartis, Biogen Idec, Roche, Takeda, Kyowa Hakko Kirin, and Cubist. In addition, Alnylam and Isis co-founded Regulus Therapeutics Inc., a company focused on discovery, development, and commercialization of microRNA therapeutics; Regulus has formed partnerships with GlaxoSmithKline and sanofi-aventis. Alnylam has also formed Alnylam Biotherapeutics, a division of the company focused on the development of RNAi technologies for application in biologics manufacturing, including recombinant proteins and monoclonal antibodies.

Gilead & MicroDose Therapeutx Announce License & Collaboration Agreement

Gilead Sciences, Inc. and MicroDose Therapeutx, Inc. recently announced they have entered into an exclusive worldwide license and collaboration agreement for the development and commercialization of MDT-637, MicroDose’s inhalable small molecule antiviral fusion inhibitor for the treatment of respiratory syncytial virus (RSV).

Under the terms of the agreement, Gilead will pay MicroDose an upfront payment and provide research funding to support MicroDose’s continued development of MDT-637 through Phase IIa clinical trials. Gilead can assume full responsibility for clinical development following Phase IIa. MicroDose also could receive additional payments based upon the achievement of certain development, regulatory, and commercial milestones, as well as development fees and royalties on future potential net sales.

MDT-637 is a fusion inhibitor that has been shown to block RSV infection in preclinical testing. The product is formulated for pulmonary delivery via MicroDose’s proprietary dry powder inhaler, which allows for rapid delivery to the site of infection (in the respiratory tract). MicroDose plans to file the IND reactivation with the US FDA and to initiate a Phase I study this year with MDT-637.

“This strategic collaboration is a significant milestone in MicroDose’s vision to develop first-in-class therapies for major unmet medical needs,” said Anand Gumaste, President and CEO of MicroDose. “Given Gilead‘s scientific and clinical expertise in virology, this partnership provides a strong validation of the potential for MDT-637 to become an important therapeutic advance for those affected by RSV infection.”

“There is an urgent need to improve upon RSV treatment and care,” added Norbert W. Bischofberger, PhD, Gilead‘s Executive Vice President, Research and Development and Chief Scientific Officer. “We believe this program aligns well with our expertise in both antiviral and respiratory drug development, and we look forward to working with the MicroDose team to advance MDT-637 into clinical testing.”

MicroDose Therapeutx is a private pharmaceutical company dedicated to improving the quality of life for people suffering from serious diseases. The company focuses on developing proprietary products that address large unmet market opportunities, and on pulmonary and oral drug delivery platforms. The company develops its products and technologies independently, as well as in partnership with leading pharmaceutical companies. MicroDose’s current pipeline targets respiratory diseases, such as asthma, COPD, and RSV, as well as IBS-C and constipation.

Gilead Sciences is a biopharmaceutical company that discovers, develops, and commercializes innovative therapeutics in areas of unmet medical need. The company’s mission is to advance the care of patients suffering from life-threatening diseases worldwide.

Brainlab & SurgiVision Announce Strategic Alliance

Brainlab AG and SurgiVision, Inc. recently announced a collaboration aimed at integrating SurgiVision’s ClearPoint product line with Brainlab’s iMRI product line, with particular focus on local delivery of drugs and other therapeutic agents to precision targets in the brain under magnetic resonance imaging (MRI) guidance. Brainlab and SurgiVision believe the integration of their technologies will allow patient-specific treatment planning, simplified clinical workflows, and optimal delivery of drugs to the brain.

Under the terms of the agreement, Brainlab also received the right to sell SurgiVision’s ClearPoint product line, including the right to exclusively sell ClearPoint products in the neurological drug delivery field, and Brainlab made an investment into SurgiVision. SurgiVision will continue to sell its ClearPoint product line directly for all applications outside the neurological drug delivery field.

SurgiVision’s ClearPoint system provides guidance for the placement and operation of instruments during neurological procedures performed within the MRI environment. Using the ClearPoint system, a physician sees and selects a neurological target, aims SurgiVision’s targeting device, and watches via MRI as the surgical instrument is advanced to the target and the therapy is delivered. SurgiVision’s ClearPoint system has received both FDA clearance and CE Mark. SurgiVision also recently received FDA clearance for its SmartFlow injection cannula.

Brainlab’s Brainsuite iMRI integrates intra-operative MRI with surgical planning and ceiling-mounted navigation, allowing full utilization of intra-operative data for making informed decisions during the procedure. iPlan Flow enables neurosurgeons to optimize local drug delivery to the CNS, based on patient-specific imaging.

“Direct, image-guided delivery of therapeutic agents into the brain provides the next-generation platform for delivering a wide range of promising therapies for patients suffering from CNS disorders, such as Parkinson’s disease and malignant brain tumors,” said Dr. Krystof Bankiewicz, Vice Chair and Professor, Department of Neurosurgery and Director of the NeuroTherapeutics Delivery Center at the University of California San Francisco. “This collaboration between two important players in the field is welcome news to pharmaceutical companies, biotech companies, and researchers with therapeutic agents that need to be delivered in a targeted, patient-specific, image-guided fashion.”

“MRI-guided interventions is an important, emerging area of medicine. We are excited about the partnership with SurgiVision, the integration of our technologies in functional neurosurgery, and the opportunity to offer these innovative solutions to customers through our global sales network,” added Stefan Vilsmeier, CEO of Brainlab.

“We are very pleased to announce this collaboration with Brainlab, a pioneer and leader in image-guided interventions,” said Kimble Jenkins, CEO of SurgiVision. “Together with our new partner, we are excited about providing to clinicians powerful, new capabilities in targeted drug delivery and other MRI-guided therapies to benefit patients who suffer from CNS disorders.”

Brainlab develops, manufactures, and markets software-driven medical technology that supports targeted, less-invasive treatment. Brainlab technology drives collaboration between hospitals and clinicians from a wide variety of subspecialties – from neurosurgery and oncology to orthopedics, ENT, CMF, and spine & trauma.

Founded in 1998, SurgiVision, Inc. is a leader in the emerging field of MRI-guided interventions, creating innovative platforms for performing the next generation of minimally invasive surgical procedures in the brain and heart. Utilizing a hospital’s existing MRI suite, SurgiVision’s FDA-cleared ClearPoint system is designed to enable a range of minimally invasive procedures in the brain.

Avantor Certifies All Doe & Ingalls Sites Under Certified Excipient Distributor Program

Avantor Performance Materials (formerly Mallinckrodt Baker) recently announced that Doe & Ingalls, a national chemical services provider and Avantor distributor, is the first distributor to have all company sites certified under the Avantor Certified Excipient Distributor (CED) Program.

Avantor Performance Materials manufactures and markets high-performance chemistries and materials around the world under several respected brand names, including the J.T.Baker®, MacroTM (formerly Mallinckrodt®), Rankem, and Diagnova brands. Avantor products are used around the world in a wide range of industries, including biotechnology and pharmaceutical production; electronics and photovoltaic manufacturing; and in research, academic, and quality control laboratories.

Doe & Ingalls, based in Durham, NC, is an Avantor distributor specializing in providing chemical services expertise and high-quality chemicals to the life science and electronics industries. The company sources materials from suppliers who can meet the specific supply chain needs of life science and electronics manufacturers.

In October 2010, the last Doe & Ingalls facility, Riverside, CA, received certification. The 41,600-sq-ft facility includes a fixed modular cleanroom for pre-shipment sampling and dip tubing services. Last year, Avantor also re-certified Doe & Ingalls regional service centers in FL, MD, MA, and NC.

“Avantor has a long history of working with Doe & Ingalls to supply high-quality performance chemicals to the pharmaceutical, biotechnology, photovoltaic, laboratory, and electronics industries,” said Avantor Executive Vice President, Pharmaceuticals and The Americas, Paul Smaltz. “Doe & Ingalls’ business model aligns with our vision of service excellence. Doe & Ingalls has met our rigorous standards time and time again, making it the only distributor to receive this distinctive certification for all sites.”

“Doe & Ingalls is pleased to have received Avantor’s Certified Excipient Distributor distinction at each of its service centers,” added Doe & Ingalls Chief Executive Officer John Hollenbach. “Doe & Ingalls is focused on building secure chemical supply chains for the life science and electronics industries. We do this by partnering with quality suppliers like Avantor and then offering supply chain services that create efficiencies and manage risk. This certification demonstrates to our customers our commitment to supply chain security.”

Launched in August 2008, the Avantor CED program is designed to provide customers with the assurance that a certified channel partner utilizes fully documented chain of custody and change management procedures. An Avantor CED delivers its regulated products through an optimized supply chain that is compliant with the International Pharmaceutical Excipient Council’s (IPEC) guidelines. In order to receive certified status, a potential channel partner must annually pass a strict quality audit conducted by the Avantor regulatory department. The audit process is designed to ensure that each distributor in the program is in compliance with all IPEC Good Distribution Practices for Pharmaceutical Excipient guidelines.

Baxter Acquires Specialty Pharmaceutical Company

Baxter International Inc. recently announced it has entered into a definitive agreement to acquire privately held Prism Pharmaceuticals, Inc., a specialty pharmaceutical company based in King of Prussia, PA. Prism Pharmaceuticals has developed and received US FDA approval for multiple presentations of NEXTERONE (amiodarone HCl), an antiarrhythmic agent. The NEXTERONE product portfolio, which does not contain polysorbate 80 or benzyl alcohol, includes the first and only ready-to-use premixed intravenous (IV) bag formulations as well as vials and a prefilled syringe.

The terms of the agreement include a total consideration of up to $338 million, consisting of an upfront cash payment of $170 million at closing and up to $168 million in future sales-based milestone payments. The transaction is expected to close in the second quarter of 2011, subject to customary closing conditions and expiration of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act. This transaction is not expected to have a material impact on Baxter’s 2011 financial results.

The NEXTERONE premixed IV formulations were recently approved by the FDA in two ready-to-use dosage forms: 150 mg in a 100-mL flexible container for rapid 10-minute loading infusion and 360 mg in a 200-mL flexible container for subsequent loading and maintenance infusions. These ready-to-use formats require no admixing, eliminating potential medication errors associated with compounding. In addition, NEXTERONE can be stored at room temperature during its 2-year shelf-life and fits in automated dispensing cabinets and crash carts, putting the product in patient care areas ready for use during acute, time-sensitive, and life-threatening situations. Prior to entering into the definitive agreement to acquire the company, Baxter was the contract manufacturer selected by Prism to manufacture the premixed IV bags using Baxter’s proprietary GALAXY container technology and the prefilled syringe.

“NEXTERONE is a great addition to our leading portfolio of premix drugs and solutions for the acute care setting. It offers clinicians a unique, ready-to-use antiarrhythmic agent for critical and time sensitive situations, while also providing convenience to caregivers and value to pharmacists,” said Robert M. Davis, President of Baxter’s Medical Products Business. “Based on our strong hospital relationships and familiarity with the product, Baxter is well-positioned to launch the ready-to-use presentations of NEXTERONE.”

“The development and approval of NEXTERONE has been Prism’s greatest achievement,” added Warren Cooper, CEO of Prism Pharmaceuticals. “We look forward to Baxter’s success in launching this product, which will benefit clinicians and the patients they serve.”

Amiodarone is an antiarrhythmic medication used for ventricular tachyarrhythmias, or fast forms of irregular heartbeat. NEXTERONE Premixed Injection is indicated for initiation of treatment and prophylaxis of frequently recurring ventricular fibrillation and hemodynamically unstable ventricular tachycardia in patients refractory to other therapy.

Gerresheimer Manufactures Insulin Pen for Big Pharma Company

Gerresheimer AG has commenced the commercial production of the ClikSTAR® insulin pens for sanofi-aventis. Medical plastic systems, such as insulin pens, will be increasingly important in the Gerresheimer product portfolio in the future because they incorporate a dosage and application function in addition to being a pure medication packaging.

The industrialization of the insulin pen was prepared in Wackersdorf, the Gerresheimer Plastics Division’s technology center. Gerresheimer’s Pfreimd plant (Germany) is the place where the individual components are manufactured and assembled under clean room conditions.

“We are delighted about this sanofi-aventis production project because it involves a complex product that demonstrates our leadership in the medical plastic systems market to the pharma industry,” said Uwe Röhrhoff, Chief Executive Officer of Gerresheimer AG.

The re-usable ClikSTAR insulin pen has received the Good Design Award. Insulin pens are drug delivery systems in pen format that enable diabetes sufferers to reliably administer their regular insulin doses in a virtually pain-free procedure. Diabetes treatment is gaining in significance from year to year. According to World Health Organization (WHO) estimates, there will be around 350 million diabetes sufferers in the world by the year 2030. ClikSTAR is a registered trademark of sanofi-aventis.

Gerresheimer is an internationally leading manufacturer of high-quality specialty products made of glass and plastic for the global pharma and healthcare industry. Its comprehensive portfolio of products extends from pharmaceutical vials to complex drug delivery systems, such as syringe systems, insulin pens, and inhalers for safe medication dosage and application.