Nucala receives self-administration EU approval

PharmaTimes | August 02, 2019

GlaxoSmithKline has announced that the European Commission has granted marketing authorisation for two new methods of administering the companys Nucala mepolizumab - a pre-filled pen and a pre-filled safety syringe. It is the only monthly anti-IL5 biologic approved in Europe that people with severe eosinophilic asthma can take at home, after a healthcare professional decides it is appropriate. The first European launches of the new administration options are expected to take place in August 2019, but the original lyophilised version remains available, giving healthcare professionals a choice of three different administration options to best fit in with their patients’ lives. The marketing authorisation is supported by positive experience data from two real-world open-label, single-arm, phase IIIa studies (NCT03099096 & NCT03021304) that found patients and caregivers were able to successfully self-administer Nucala with the pre-filled pen and pre-filled safety syringe after appropriate training (98% and 100% respectively). Dr Hal Barron, chief scientific officer and president, R&D, GSK, said that making the drug available for patients to take in the convenience of their own home is an "important advance that builds on its proven efficacy, reflecting our ongoing efforts to meet the needs of patients with complex diseases.” First approved in 2015 for severe eosinophilic asthma, Nucala is the first-in-class monoclonal antibody that targets IL-5. It is believed to work by preventing IL-5 from binding to its receptor on the surface of eosinophils, reducing blood eosinophils without completely depleting them.

Spotlight

Mr. Manne Venkanna Chowdary, CMD, AmpleLogic speaks to the audience at IDMA's 21st Pharmaceutical Analysts' Convention – ‘Technology – The Game Changer’ in Mumbai (24th and 25th May 2019) on the topic ‘Digitalization in Pharma with Intelligent Systems, Collaborative Development Approach


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BUSINESS INSIGHTS

NextRNA Launches with $56 Million in Funding to Bring Transformative Non-Coding RNA-Directed Medicines to Patients

NextRNA Therapeutics | March 02, 2022

NextRNA Therapeutics, a biotechnology company unlocking the potential of non-coding RNAs to develop transformative therapeutics, announces its launch with $9.3 million in seed financing and a $46.8 million Series A led by Cobro Ventures and Lightchain Capital, with additional participation from Circle Alternative Investments, Evans Capital, Jefferies, Rivas Capital, and Willett Advisors. Proceeds will be used by the company to augment its target and drug discovery engine, expand its pipeline, and advance lead programs. The vast majority of DNA is copied into RNAs that do not make proteins. These RNA molecules, called “non-coding RNAs,” play essential roles in cells by interacting with and modulating the activities of proteins. These interactions can drive pathogenic processes in multiple disease areas, including oncology, immunology, and neurology. Non-coding RNAs, in particular long non-coding RNAs, and their interacting proteins therefore represent a vast and untapped class of novel therapeutic targets. “NextRNA is uniquely positioned to be the leader in non-coding RNA-directed medicines. We have established a robust target and drug discovery engine to systematically identify disease-relevant long non-coding RNAs and their interacting proteins. By developing selective small molecules to drug these interactions, we plan to translate discoveries of non-coding RNA targets into a robust pipeline of transformative therapies across multiple disease areas.” Dominique Verhelle, Ph.D., MBA, co-founder, chief scientific officer, and interim chief executive officer, NextRNA NextRNA was established based on pioneering work by Carl Novina, M.D., Ph.D., at Dana-Farber Cancer Institute. “By understanding the interactions between long non-coding RNAs and specific proteins, we can decode the function of long non-coding RNAs and apply it to create medicines,” said Dr. Novina, co-founder of NextRNA. Since its founding in January of 2021, NextRNA has established its initial team, built out the target and drug discovery engine, and advanced two small molecule programs in oncology and immunology. “NextRNA is at the forefront of innovation in the field of non-coding RNAs,” said Todd Kaloudis, Managing Director at Cobro Ventures. “There is growing excitement around the potential of non-coding RNA-directed medicines, and we are pleased to have such an experienced team focused on NextRNA’s vision of bringing transformative therapies to patients.” About NextRNA Therapeutics NextRNA is a biotechnology company with a mission to translate pioneering science into transformative therapeutics for the benefit of patients. The company is leveraging its proprietary technology platform to systematically identify disease-relevant non-coding RNAs and their interacting proteins, and to develop selective small molecules to drug these interactions. NextRNA investors include Cobro Ventures, Lightchain Capital, Circle Alternative Investments, Evans Capital, Jefferies, Rivas Capital, and Willett Advisors.

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BUSINESS INSIGHTS

Alnylam Announces 3-Month Extension of Review Period for New Drug Application for Vutrisiran

Alnylam Pharmaceuticals, Inc. | April 05, 2022

Alnylam Pharmaceuticals, Inc. the leading RNAi therapeutics company, announced that the U.S. Food and Drug Administration has extended the review timeline of the New Drug Application for vutrisiran, an investigational RNAi therapeutic in development for the treatment of transthyretin-mediated amyloidosis, to allow for the review of newly added information related to the new secondary packaging and labeling facility. Alnylam recently learned that the original third-party secondary packaging and labeling facility the Company planned to use for the vutrisiran launch was recently inspected and the inspection requires classification for the FDA to take action on the vutrisiran NDA. The inspection observations were not directly related to vutrisiran. In order to minimize delays to approval, Alnylam has identified a new facility to pack and label vutrisiran and submitted an amendment to the NDA for review by the FDA. The updated Prescription Drug User Fee Act goal date to allow for this review is July 14, 2022. No additional clinical data have been requested by the FDA. “We are committed to working with the FDA and the new facility to bring this important treatment option as quickly as possible to patients living with the polyneuropathy of hATTR amyloidosis, an inherited, progressively debilitating, and fatal disease. We are confident in our regulatory application and the body of data supporting the efficacy and safety of vutrisiran.” Pushkal Garg, M.D., Chief Medical Officer & EVP, Development and Medical Affairs Vutrisiran is also under review by the European Medicines Agency the Brazilian Health Regulatory Agency and the Japanese Pharmaceuticals and Medical Devices Agency (PMDA). We aim to minimize the impact of this issue on these reviews through timely resolution of the matter or utilization of an alternative facility for one or more of these regions. Vutrisiran has been granted Orphan Drug Designation in the U.S. and the European Union (EU) for the treatment of ATTR amyloidosis, and in Japan for transthyretin type familial amyloidosis with polyneuropathy. About Vutrisiran Vutrisiran is an investigational, subcutaneously administered RNAi therapeutic in development for the treatment of ATTR amyloidosis, which encompasses both hATTR and wild-type ATTR amyloidosis. It is designed to target and silence specific messenger RNA, potentially blocking the production of wild-type and variant transthyretin protein before it is made. Quarterly, and potentially biannual, administration of vutrisiran may help to reduce deposition and facilitate the clearance of TTR amyloid deposits in tissues and potentially restore function to these tissues. Vutrisiran utilizes Alnylam’s Enhanced Stabilization ChemistryGalNAc-conjugate delivery platform, designed for increased potency and high metabolic stability that may allow for infrequent subcutaneous injections. About Alnylam Pharmaceuticals Alnylam has led the translation of RNA interference into a whole new class of innovative medicines with the potential to transform the lives of people afflicted with rare and prevalent diseases with unmet need. Based on Nobel Prize-winning science, RNAi therapeutics represent a powerful, clinically validated approach yielding transformative medicines. Since its founding 20 years ago, Alnylam has led the RNAi Revolution and continues to deliver on a bold vision to turn scientific possibility into reality. Alnylam’s commercial RNAi therapeutic products are ONPATTRO®, GIVLAARI® (givosiran), OXLUMO® and Leqvio® being developed and commercialized by Alnylam’s partner, Novartis. Alnylam has a deep pipeline of investigational medicines, including six product candidates that are in late-stage development. Alnylam is executing on its “Alnylam P5x25” strategy to deliver transformative medicines in both rare and common diseases benefitingpatients around the world through sustainable innovation and exceptional financial performance, resulting in a leading biotech profile. Alnylam is headquartered in Cambridge, MA.

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BUSINESS INSIGHTS

ApiJect Announces Investment by Royalty Pharma and Jefferies

ApiJect Systems Corp. | May 19, 2022

ApiJect Holdings, Inc., the parent company of ApiJect Systems, Corp., a medical technology public-benefit corporation transforming the fill and finish and delivery of injectable vaccines and medicines, announced today that it had completed a $111 million private round of investment. This investment round values ApiJect at approximately $300 million. The new round was led by Royalty Pharma and Jefferies Financial Group (NYSE: JEF), which acquired a revenue interest in addition to equity. Jefferies and ApiJect's other lenders also converted all of their debt into ApiJect equity, such that following this transaction, ApiJect has no debt on its balance sheet. The funds will be used for continued development of ApiJect's proprietary Blow-Fill-Seal (BFS) plastic prefilled injection system, to deploy its equipment and proprietary technologies to licensees, and for general working capital purposes. Royalty Pharma and Jefferies are represented on ApiJect's Board of Directors, along with ApiJect co-founders Jay Walker and Marc Koska, and Hanjin In, the CEO of Tae-Chang Industrial Co., Ltd., ApiJect's South Korean cannula supplier. ApiJect is developing an innovative portfolio of drug delivery devices using high-speed BFS manufacturing, which is recognized by the FDA as an advanced aseptic process that forms, fills and seals a single unit dose in a continuous, automated manufacturing step. BFS technology greatly reduces the risk of contamination and error during one of the most critical steps of drug production. BFS is cost-efficient for short and long runs, and highly suitable to scale quickly in order to meet unexpected spikes in demand such as for a rapid response to population-scale health emergencies. Advancements developed by ApiJect for temperature management now enable BFS manufacturing of a vast array of sterile injectable drugs, including ultra-cold mRNA vaccines. "This is an important day for injection device innovation, and, with that, the future health of billions of people throughout the world. The investment provided by Royalty Pharma and Jefferies supports further development of an injection technology that can scale rapidly and efficiently to meet global demand for injectable medicines." ApiJect Chief Executive Officer Jay Walker Mr. Walker further said: "We welcome Royalty Pharma's commitment to the ApiJect mission. Royalty Pharma has a proven track record in selecting successful partners and in assisting in advancing its partners' goals. We could not ask for a better strategic partner from within the pharma industry. In Jefferies, we have a partner that has been with us from virtually the beginning of ApiJect." "We are delighted to form this win-win partnership with ApiJect," said Pablo Legorreta, Royalty Pharma's Founder and Chief Executive Officer. "The global pandemic highlighted the clear unmet need for a new kind of prefilled syringe that can be rapidly produced to respond to public health threats, and at massive scale that would be capable of delivering hundreds of millions, potentially billions, of doses to people in the U.S. as well as globally. ApiJect is developing a truly innovative technology, that could transform the manufacturing and delivery of life-saving injectable drugs in a way that would also make these medicines safer and more readily accessible for patients around the world." "We are proud of our association with ApiJect and believe strongly that their solutions will be a positive gamechanger in many of the most critical aspects of improving health globally," said Rich Handler, CEO of Jefferies. The first potential device made on the ApiJect Platform is the Prefilled ApiJect Injector, a single-dose prefilled injector designed to efficiently deliver a 0.5mL dose into a patient with a simple squeeze of the BFS container by the healthcare professional. This new type of prefilled injector will be ApiJect's first product submitted for regulatory review and approval. The Prefilled ApiJect Injector and its manufacturing process include An aseptically filled single-dose container, designed to enable efficient and intuitive injection administration. A pen-needle-style hub that turns the BFS container into an injection device. Innovations in temperature management to expand the range of medicines and vaccines suitable for BFS packaging. ApiJect recently launched its Technology Development Center in the greater Orlando area to bring to the U.S. critical capabilities for device design and engineering, and BFS mold development, as well as for small-scale manufacturing of single-dose, prefilled injectors and other injectable devices. Its purpose is to dramatically reduce the time and processes necessary for product development. ApiJect's Technology Development Center supports the company's existing fill-finish lines at its manufacturing partner site in South Carolina, which currently has the capacity to produce up to 540 million single-dose prefilled injectors annually. About ApiJect Systems ApiJect Systems, Corp., is a public-benefit medical technology company working to use our platform to bring more prefilled, single-dose injections to patients everywhere. The ApiJect Platform enables pharmaceutical and biotech companies to design scalable prefilled injectors and efficiently fill-finish them with their injectable drug products. This can be done either on one of their own Blow-Fill-Seal packaging lines with ApiJect-licensed technology, or at one of ApiJect's world-class manufacturing partners.

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PHARMACY MARKET

AIkido Pharma Reports Improved Manufacturing Process and New United States Patent for Pancreatic Drug

AIkido Pharma Inc. | March 04, 2022

AIkido Pharma Inc.reported an improvement in the manufacturing process for its pancreatic cancer drug, DHA-dFdC, licensed from the University of Texas at Austin. The Company also reported the issuance of an additional U.S. patent covering the drug, and the filing of a continuation patent application intended to expand patent coverage to other facets of the drug. With respect to manufacturing process, the Company reported the successful development of a new means for the scaled production and isolation of the key intermediate compound in the manufacture of DH-dFdC. The Company has now executed a further contract with its contract manufacturing organization, Parimer Scientific, to employ the new process to produce several thousand milligrams of the drug for use in formulation development and stability studies. The Company also reported that the U.S. Patent Office recently issued new U.S. Patent No. 11,219,633, which provides additional intellectual property protection for the drug compound. The term of the patent is expected to continue until May of 2035 with the payment of required maintenance fees. Prior to the patent issuance, the Company authorized the timely filing of a patent continuation application, U.S. Serial No. 17/539,682, in which additional claims related to various aspects of the drug and formulation will be pursued. "This new process significant step forward in the development of our pancreatic cancer drug and should permit production of the drug on a commercial scale at a lower cost. I am also pleased that we can continue to report expansion of the patent estate." Anthony Hayes, CEO of AIkido Richard T. Pace, Owner and Principal Scientist of Parimer Scientific, stated, "The newly developed manufacturing method is a large step forward for this innovative technology. I believe it will reduce cost per unit and at the same time increase batch production volumes." About AIkido Pharma Inc. AIkido Pharma Inc. was initially formed in 1967 and is a biotechnology Company with a diverse portfolio of small-molecule anticancer and antiviral therapeutics. The Company's platform consists of patented technology from leading universities and researchers, and we are currently in the process of developing an innovative therapeutic drug platform through strong partnerships with world renowned educational institutions, including The University of Texas at Austin and University of Maryland at Baltimore. Our diverse pipeline of therapeutics includes therapies for pancreatic cancer, prostate cancer. We are constantly seeking to grow our pipeline to treat unmet medical needs in oncology. The Company is also developing a broad-spectrum antiviral platform that may potentially inhibit replication of multiple viruses including Influenza virus, SARS-CoV (coronavirus), MERS-CoV, Ebolavirus and Marburg virus.

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Spotlight

Mr. Manne Venkanna Chowdary, CMD, AmpleLogic speaks to the audience at IDMA's 21st Pharmaceutical Analysts' Convention – ‘Technology – The Game Changer’ in Mumbai (24th and 25th May 2019) on the topic ‘Digitalization in Pharma with Intelligent Systems, Collaborative Development Approach

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