Alnylam Pharmaceuticals, Inc., the leading RNAi therapeutics company, today provided perspective on the positive complete results from the ORION-11 Phase 3 study of inclisiran, an RNAi therapeutic in development for the treatment of hypercholesterolemia. These results were presented earlier today by Alnylam’s partner The Medicines Company and collaborators at the European Society of Cardiology’s ESC Congress 2019 in Paris, France.
“The ORION-11 results represent a landmark event for Alnylam and RNAi therapeutics. With over 1,600 patients enrolled, ORION-11 is the largest ever randomized, double-blind, placebo-controlled study of any RNA-based medicine. The results from the study demonstrate significant efficacy for a drug administered subcutaneously once every six months and a very encouraging safety profile, with predominantly mild and transient injection site reactions as the only reported drug-related finding,” said John Maraganore, Ph.D., Chief Executive Officer of Alnylam. “These results further support the safety of our RNAi therapeutics platform and provide the largest demonstration to date suggesting that there is no systematic evidence for a platform-specific safety signal. Consequently, these results greatly strengthen our conviction for the future potential of RNAi therapeutics in large population diseases. Moreover, the pharmacology of RNAi therapeutics, as infrequently administered medicines, creates what we believe to be a very attractive profile for the treatment of common diseases. Finally, assuming a positive regulatory review, the significant inclisiran royalties of up to 20 percent will provide yet another source of relatively near-term revenues and will support Alnylam’s transition toward a self-sustainable financial profile for continued and future growth.”
ORION-11 now becomes the third positive Phase 3 study for an RNAi therapeutic and the second in 2019 alone. Assuming ORION-9 and -10 are similarly positive and following regulatory submissions and review, Alnylam expects inclisiran could reach the market around late 2020.
A video of Dr. Maraganore commenting on the implications of the ORION-11 results can be viewed on the Company’s website, www.alnylam.com.
Inclisiran, the first cholesterol-lowering therapy in the RNAi therapeutics class, is an investigational therapy in Phase 3 clinical development to evaluate its ability to lower low-density lipoprotein cholesterol (also known as LDL-C) through twice-yearly dosing. Inclisiran harnesses the body’s natural process of RNA interference to specifically prevent production of the PCSK9 protein in the liver which enhances the liver’s ability to remove LDL-C from the bloodstream, thereby lowering LDL-C levels. In Phase 2 studies, inclisiran provided clinically significant LDL-C reductions greater than 50 percent in addition to the effects of statins and/or ezetimibe, and LDL-C reductions were sustained throughout the six-month dosing interval. The safety and efficacy of inclisiran were evaluated in the ORION-11 Phase 3 study with positive results; these results have not been evaluated by the FDA, the EMA or any other health authority. Inclisiran is not yet approved for use by the FDA or any other regulatory authority. In February 2013 Alnylam granted global rights under a license and collaboration agreement to The Medicines Company to develop, manufacture, and commercialize inclisiran.
RNAi (RNA interference) is a natural cellular process of gene silencing that represents one of the most promising and rapidly advancing frontiers in biology and drug development today. Its discovery has been heralded as “a major scientific breakthrough that happens once every decade or so,” and was recognized with the award of the 2006 Nobel Prize for Physiology or Medicine. By harnessing the natural biological process of RNAi occurring in our cells, a new class of medicines, known as RNAi therapeutics, is now a reality. Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise Alnylam's RNAi therapeutic platform, function upstream of today’s medicines by potently silencing messenger RNA (mRNA) – the genetic precursors – that encode for disease-causing proteins, thus preventing them from being made. This is a revolutionary approach with the potential to transform the care of patients with genetic and other diseases.
About Alnylam Pharmaceuticals
Alnylam is leading the translation of RNA interference (RNAi) into a whole new class of innovative medicines with the potential to transform the lives of people afflicted with rare genetic, cardio-metabolic, hepatic infectious, and central nervous system (CNS)/ocular diseases. Based on Nobel Prize-winning science, RNAi therapeutics represent a powerful, clinically validated approach for the treatment of a wide range of severe and debilitating diseases. Founded in 2002, Alnylam is delivering on a bold vision to turn scientific possibility into reality, with a robust discovery platform. Alnylam’s first commercial RNAi therapeutic is ONPATTRO® (patisiran) approved in the U.S., EU, Japan and Canada. Alnylam has a deep pipeline of investigational medicines, including five product candidates that are in late-stage development. Looking forward, Alnylam will continue to execute on its "Alnylam 2020" strategy of building a multi-product, commercial-stage biopharmaceutical company with a sustainable pipeline of RNAi-based medicines to address the needs of patients who have limited or inadequate treatment options. Alnylam employs over 1,200 people worldwide and is headquartered in Cambridge, MA. For more information about our people, science and pipeline, please visit www.alnylam.com and engage with us on Twitter at @Alnylam or on LinkedIn.
Alnylam Forward Looking Statements
Various statements in this release concerning Alnylam's future expectations, plans and prospects, including, without limitation, the potential implications of the ORION-11 results for patients, Alnylam's views with respect to the safety of RNAi therapeutics, its views regarding the future potential of RNAi therapeutics in large population diseases, its expectations regarding the timing of results to be reported by The Medicines Company from the ORION-9 and ORION-10 studies of inclisiran and the regulatory review of an NDA for inclisiran, its expectations regarding the receipt of royalties from The Medicines Company on sales of inclisiran, if approved by regulatory authorities, its plans to achieve a self-sustainable financial profile for continued and future growth, and expectations regarding its “Alnylam 2020” guidance for the advancement and commercialization of RNAi therapeutics, constitute forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995. Actual results and future plans may differ materially from those indicated by these forward-looking statements as a result of various important risks, uncertainties and other factors, including, without limitation, Alnylam's ability to discover and develop novel drug candidates and delivery approaches, Alnylam’s and its partners’ ability to successfully demonstrate the efficacy and safety of its product candidates, including inclisiran, the pre-clinical and clinical results for its product candidates, which may not be replicated or continue to occur in other subjects or in additional studies or otherwise support further development of product candidates for a specified indication or at all, actions or advice of regulatory agencies, which may affect the design, initiation, timing, continuation and/or progress of clinical trials or result in the need for additional pre-clinical and/or clinical testing, delays, interruptions or failures in the manufacture and supply of its product candidates, obtaining, maintaining and protecting intellectual property, Alnylam's ability to enforce its intellectual property rights against third parties and defend its patent portfolio against challenges from third parties, Alnylam’s and its partners’ ability to obtain and maintain regulatory approval, pricing and reimbursement for products, including inclisiran, progress in establishing a commercial and ex-United States infrastructure, successfully launching, marketing and selling its approved products globally, Alnylam’s ability to successfully expand the indication for ONPATTRO in the future, competition from others using technology similar to Alnylam's and others developing products for similar uses, Alnylam's ability to manage its growth and operating expenses, obtain additional funding to support its business activities, and establish and maintain strategic business alliances and new business initiatives, Alnylam's dependence on third parties for development, manufacture and distribution of products, the outcome of litigation, the risk of government investigations, and unexpected expenditures, as well as those risks more fully discussed in the “Risk Factors” filed with Alnylam's most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) and in other filings that Alnylam makes with the SEC. In addition, any forward-looking statements represent Alnylam's views only as of today and should not be relied upon as representing its views as of any subsequent date. Alnylam explicitly disclaims any obligation, except to the extent required by law, to update any forward-looking statements.
the 2019 Asia-pacific pharma IP Leader Summit: http://en.zenseegroup.com/p/404716/ will be held in Beijing on November 14-15, and will attract more than 500 industry experts from domestic and foreign pharmaceutical companies, biotechnology companies, governments, associations, law firms, intellectual property agents and other companies to attend.
Major Chinese biopharmaceutical company Hansoh Pharmaceutical Group Company Limited (Hanmori Pharmaceutical Group Co., Ltd., Hanso Pharmaceutical) and AI ) Atomwise, Inc., a leader in the field, announced the collaboration. The purpose of the collaboration is to design and discover potential drug candidates for a total of 11 private target proteins in multiple therapeutic areas.Atomwise and Hansoh Pharma scientific teams work closely together in each program. The combination of complementary expertise and technology has the potential to dramatically increase success rates and shorten schedules in drug discovery and clinical development.Dr. Aifeng Lyu, President of Jiangsu Hansoh Pharmaceutical Group Co., Ltd., a subsidiary of Hansoh Pharma, said: “Atomwise is the best partner for Hansoh Pharma to use AI technology to innovate and create diverse small molecule pipelines in oncology and other therapeutic areas. I am very impressed with the team and I believe that working together will provide a special opportunity to develop first-in-class and best-in-class therapeutics. "Dr. Abraham Heifets, CEO of Atomwise, said: “It is a great pleasure for us to partner with Chinese biopharmaceutical giant Hansoh Pharma. Hansoh Pharma shares our strong commitment to innovation and our mission to influence patient health globally.”Atomwise's world-class AI platform for structure-based drug design leads the way in finding hit compounds, selecting lead compounds from hit compounds, and optimizing lead compounds. Hansoh Pharma will contribute in its capacity in biological assays and medicinal chemistry, and will lead the subsequent preclinical and clinical development.Subject to the terms of the collaboration, Atomwise will receive revenue based on private technology fees, option exercise fees, royalties, and sublicenses and sales of assets created under the collaboration. Based on historical average sales of small molecule drugs, the overall potential value of this transaction for Atomwise may exceed the potential value of a hit drug if all projects succeed. Hansoh Pharma receives development and commercialization rights in all fields and regions.2019 Asia-pacific pharma IP Leader Summit: http://en.zenseegroup.com/p/404716/ will be held in Beijing on November 14-15, and will attract more than 500 industry experts from domestic and foreign pharmaceutical companies, biotechnology companies, governments, associations, law firms, intellectual property agents and other companies to attend.Official registration and consultation channels:Contact：AnnPhone: 021-65650305Email：Marketing@zenseegroup.comhttp://www.zenseegroup.com/forms/view/22113/
Akari Therapeutics, Plc, a biopharmaceutical company focused on innovative therapeutics to treat orphan autoimmune and inflammatory diseases where the complement (C5) and/or leukotriene (LTB4) systems are implicated, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for nomacopan for the treatment of bullous pemphigoid (BP).“BP, a severe blistering skin condition with no approved treatments, is an exciting therapeutic target for our lead drug candidate, nomacopan. It is also a disease of increasing prevalence due to an aging population and improving diagnosis,” said Clive Richardson, Chief Executive Officer of Akari Therapeutics. “Orphan drug designation for nomacopan is a major step forward for the program, positioning nomacopan for eligibility for an additional seven years of marketing exclusivity in BP if nomacopan is approved by the FDA. This news is in addition to the recent orphan drug designation received for our HSCT-TMA program.”The Company plans to release new safety and efficacy data from an ongoing Phase II trial with nomacopan in patients with BP at an oral presentation by Dr. Christian Sadik at the 28th European Academy of Dermatology and Venereology (EADV) Congress on October 10, 2019. In August, the Company announced new data demonstrating the synergistic benefits of nomacopan’s dual C5 and LTB4 inhibitory activity in pemphigoid disease, generated by Dr. Christian Sadik’s group at University of Lubeck, Germany, and published in the August 2019 edition of JCI Insight [link].Orphan drug designation by the FDA is granted to promote the development of drugs that target conditions affecting 200,000 or fewer U.S. patients annually and that are expected to provide significant therapeutic advantage over existing treatments. Orphan designation qualifies Akari for various benefits, including seven years of market exclusivity following marketing approval, tax credits on U.S. clinical trials, eligibility for orphan drug grants, and a waiver of certain administrative fees.2019 Asia-pacific pharma IP Leader Summit: http://en.zenseegroup.com/p/404716/ will be held in Beijing on November 14-15, and will attract more than 500 industry experts from domestic and foreign pharmaceutical companies, biotechnology companies, governments, associations, law firms, intellectual property agents and other companies to attend.Official registration and consultation channels:Contact：AnnPhone: 021-65650305Email：Marketing@zenseegroup.comhttp://www.zenseegroup.com/forms/view/22113/
China's leading biopharmaceutical company Hansoh Pharmaceutical Group Company Limited (" Hansen Pharmaceutical ") and drug discovery artificial intelligence (AI) Atomwise, Inc. (" Atomwise ") , a leader in the field, announced a collaborative design and discovery of potential drug candidates for up to 11 unexposed target proteins in a variety of therapeutic areas.Atomwise and Hansen Pharmaceutical's scientific team will work closely together in these projects. The combination of complementary expertise and technology is expected to significantly increase success and reduce timelines for drug discovery and clinical development.Dr. Lu Aifeng, President of Jiangsu Hansoh Pharmaceutical Group Co., Ltd., said: Hansen Pharmaceutical is committed to using AI technology to innovate and develop diverse small molecules in the field of cancer and other therapeutics. Atomwise is the right partner for Hansen Pharmaceuticals. We are very impressed with Atomwise's AI platform, capabilities and team. We believe that the two companies will have an excellent opportunity to develop similar and best-in-class therapeutic drugs. ."Dr. Abraham Heifets, CEO of Atomwise, said: "We are very excited to work with Hansen Pharmaceutical, China's leading biopharmaceutical company. Hansen Pharmaceuticals and we all have strong commitment to innovation and a common mission to influence the health of patients worldwide. ."The world-class AI platform for Atomwise structured drug design will guide efforts in hit discovery, accidental discovery to hit-to-lead selection, and optimization of lead compounds. Hansen Pharmaceuticals is committed to biological testing and pharmaceutical chemistry, as well as leading subsequent preclinical and clinical development activities.Under the terms of the partnership, Atomwise will receive undisclosed technology royalties, optional royalties, royalties, and subcontracting authorizations or revenue generated from the sale of the derivative assets. Based on the historical average turnover of small molecule drugs, the potential total value of all successful projects with Atomwise's transaction may exceed potential selling drugs. Hanson Pharmaceuticals will receive development and commercialization rights in all fields and geographies.2019 Asia-pacific pharma IP Leader Summit: http://en.zenseegroup.com/p/404716/ will be held in Beijing on November 14-15, and will attract more than 500 industry experts from domestic and foreign pharmaceutical companies, biotechnology companies, governments, associations, law firms, intellectual property agents and other companies to attend.Official registration and consultation channels:Contact：AnnPhone: 021-65650305Email：Marketing@zenseegroup.comhttp://www.zenseegroup.com/forms/view/22113/
WEDNESDAY, SEPTEMBER 11, 2019I-Mab Biopharma (I-Mab), a China and U.S.-based clinical stage biopharmaceutical company exclusively focused on the discovery and development of potential first-in-class and best-in-class biologics in immuno-oncology and autoimmune diseases announces the signing of a collaboration agreement with Shanghai Junshi Biosciences Co., Ltd (Junshi Biosciences), an innovation-driven biopharmaceutical company to evaluate the combination therapy of I-Mab's TJD5, a proprietary innovative CD73 antibody with Junshi Biosciences' Toripalimab (Trade name: Tuoyi), a recombinant humanized anti-PD-1 monoclonal antibody in patients with cancers in China.Dr. Jingwu Zang, Founder and Chairman of I-Mab, commented, "TJD5 is an innovative CD73 antibody with best-in-class potential that has entered into Phase I trial in the US. We are very pleased to collaborate with Junshi to explore the clinical synergies with Toripalimab which is an innovative drug with distinctive treatment advantages. We are looking forward to bringing more clinical benefits to patients across various cancer types."Dr. Ning Li, CEO of Junshi Biosciences, commented, "As an anti multi-tumor drug, toripalimab has shown good safety and efficacy in clinical trials with mono and combination therapy. We believe, through the cooperation with I-Mab, we could continue to explore the combination potential of toripalimab and innovative drugs to improve the outcomes of immune-oncology therapy, lightening hope for more patients.2019 Asia-pacific pharma IP Leader Summit: http://en.zenseegroup.com/p/404716/ will be held in Beijing on November 14-15, and will attract more than 500 industry experts from domestic and foreign pharmaceutical companies, biotechnology companies, governments, associations, law firms, intellectual property agents and other companies to attend.Official registration and consultation channels:Contact：AnnPhone: 021-65650305Email：Marketing@zenseegroup.comhttp://www.zenseegroup.com/forms/view/22113/
TUESDAY, SEPTEMBER 10, 2019ProBioGen AG, a premier service and technology provider for complex therapeutic antibodies and glycoproteins, today announced the closing of a license agreement with Bayer AG for the GlymaxX® Technology. Under the terms of the agreement, Bayer will leverage the technology to further increase the potency of an undisclosed antibody candidate for oncological indications.ProBioGen’s proven antibody-dependent cellular cytotoxicity (ADCC) enhancing technology GlymaxX® will be applied during cell line development.“We are glad to add Bayer to our list of licensees”, says ProBioGen’s Chief Executive Officer Dr. Wieland Wolf. “The GlymaxX® technology is clinically proven and is a very flexible technology which is liked by all cell lines.”About ProBioGen AGProBioGen is a premier, Berlin-based specialist for developing and manufacturing complex therapeutic antibodies and glycoproteins. Combining both state-of-the-art development services, based on ProBioGen’s CHO.RiGHT™ expression and manufacturing platform, together with intelligent product-specific technologies yields biologics with optimized properties.Rapid and integrated cell line and process development, comprehensive analytical development and following reliable GMP manufacturing is performed by a highly skilled and experienced team.All services and technologies are embedded in a total quality management system to assure compliance with international ISO and GMP standards (EMA/FDA).ProBioGen was founded 1994, is privately owned, and located in Berlin, Germany.About GlymaxX®The GlymaxX® technology, developed by ProBioGen, prevents the cellular synthesis of the sugar “fucose” and hence, in antibody-producing cells, its addition to the N-linked carbohydrate part of the antibody. The absence of fucose is known to greatly enhance ADCC. The GlymaxX® technology is based on the stable introduction of a gene for an enzyme which blocks the producer cells’ fucose biosynthesis pathway. As a unique feature, differentiating it from other approaches, GlymaxX® can be applied to both novel or already existing antibody producer cell lines, and entire antibody expression and discovery platforms, without negatively affecting their productivity or other product characteristics.Furthermore, a single GlymaxX® cell line can be flexibly used to produce differently fucosylated products, depending on the upstream process: In fucose-free medium the antibody is quantitatively afucosylated. The same GlymaxX® cell line grown in fucose-containing medium however, uses the provided fucose and produces fully fucosylated antibody. Thus, a GlymaxX® cell line can by employed to produce different products: For instance ADCC-enhanced GlymaxX® antibodies or wildtype-like, fully fucosylated mAbs, for a parallel Antibody-Drug-Conjugate (ADC) project.Finally, GlymaxX® has been used by biosimilar-developing companies to adjust a specific content of fucose in order to match the originators glycoprofile. Overall, GlymaxX® is simple, rapid, potent, and universally applicable to different CHO hosts and all other eukaryotic cell species.ProBioGen offers its GlymaxX® technology royalty-free and non-exclusively as a service or as an individual license.2019 Asia-pacific pharma IP Leader Summit: http://en.zenseegroup.com/p/404716/ will be held in Beijing on November 14-15, and will attract more than 500 industry experts from domestic and foreign pharmaceutical companies, biotechnology companies, governments, associations, law firms, intellectual property agents and other companies to attend.Official registration and consultation channels:Contact：AnnPhone: 021-65650305Email：Marketing@zenseegroup.comhttp://www.zenseegroup.com/forms/view/22113/