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Alnylam Pharmaceuticals (Merged with Ribopharma in July 2003)
John Maraganore, PhD, CEO  Barry Greene, President and COO John A. Schmidt, Jr., MD, CSO Susanna High, VP-Business Planning and Program Management Victor Kotelianski, MD, PhD, VP-Research Jason Rhodes, VP-Business Development Muthiah Manoharan, PhD, VP-Drug Discovery Saraswathy (Sara) Nochur, PhD, VP-Regulatory Affairs Stuart Pollard, PhD, VP-Scientific Business and Strategy Dinah Sah, PhD, VP-Research, CNS, and Oncology
300 Third Street, 3rd Floor
Cambridge, MA 02142 USA
Tel: 617-551-8200 Fax: 617-551-8101
Website: http://www.alnylam.com/
Profile
(Public Nasdaq: ALNY)
Alnylam Pharmaceuticals is engaged in the development of therapeutic RNA interference, a technology that can specifically and potently silence disease-causing genes. The company was founded in 2002 by an international group of scientists that helped discover the phenomenon of RNA interference. Alynylam's scientific founders include Phillip Sharp (Nobel Laureate and Biogen founder), Paul Schimmel (member, National Academy of Sciences and founder of Cubist and Alkermes), and professors Dave Bartel (MIT), Tom Tuschl (Max Planck Institute), and Phil Zamore (UMass Medical School). The company is built around its two operating units, Alnylam Pharmaceuticals of Cambridge, Massachusetts, and Alnylam Europe of Kulmbach, Germany. The company's focus is to discover, develop, and commercialize therapeutic products based on RNAi for a broad range of therapeutic indications including viral, oncologic, metabolic, CNS and autoimmune diseases.
In August 2008, Alnylam Pharmaceuticals formed an exclusive research agreement with the Max Planck Institute of Molecular Cell Biology and Genetics (Dresden, Germany), to investigate and characterize the molecular mechanisms underlying intracellular transport of small interfering RNA, (siRNA), the molecules that mediate RNAi. Alnylam scientists will work closely with the laboratory of Marino Zerial, PhD, whose team is internationally recognized as being at the forefront of the intracellular trafficking field and have the needed expertise and capabilities to investigate cellular uptake pathways in a robust and high throughput manner. Alnylam will have an option on any intellectual property that results from this agreement.
In July 2008, the Glover patent (EP 1230375), which is exclusively licensed to Alnylam from Cancer Research Technology (CRT), completed initial oral opposition proceedings in Europe. Through these proceedings, the Opposition Division of the European Patent Office has overturned the previously granted claims of the patent which covered certain features of RNAi therapeutics. Alnylam intends to appeal any decision which does not result in the patent being maintained in its present form or scope. The Glover patent is one of many issued or granted so-called 'fundamental' patents in Alnylam's broad IP estate for RNAi therapeutics.
In June 2008, Alnylam Pharmaceuticals closed an investment in and completed an expanded agreement with Tekmira Pharmaceuticals as a result of the achievement of Tekmira's business combination with Protiva Biotherapeutics, which occurred effective May 30, 2008. This agreement expands Alnylam's access to key technology and intellectual property (IP) for the delivery of RNAi therapeutics with liposomal delivery technologies. As part of the agreement, Alnylam made an equity investment of $5.0 million in Tekmira at $2.40 per share. Alnylam maintains its exclusive license to Tekmira's rights to the USPTO-issued Semple (atent # 6,858,225) and Wheeler (# 5,976,567 and # 6,815,432) patents, covering cationic liposomal delivery. Alnylam has also obtained expanded rights to IP controlled by Protiva prior to the combination and to new IP generated by Tekmira. Alnylam retains certain rights to sublicense this Tekmira IP to its strategic partners.
Alnylam has also granted to Tekmira InterfeRx(TM) licenses to discover, develop, and commercialize RNAi therapeutics towards seven gene targets. In return for these licenses, Alnylam may be eligible to receive milestone fees and royalties. Alnylam also has the option to co-develop and co-commercialize Tekmira's PLK SNALP program being developed for the treatment of certain cancers. PLK1, the polo-like kinase 1 gene, is one of the seven gene targets for which Tekmira has received an InterfeRx license. It has been shown to be involved in the growth of certain types of solid tumors, and PLK SNALP has been shown in pre-clinical studies to selectively kill cancer cells, while sparing normal healthy cells in the same tissue. Alnylam has the right to exercise its option for this program up until the commencement of Phase II clinical trials.
In May 2008, Alnylam Pharmaceuticals and Takeda Pharmaceutical Company formed a strategic platform alliance in RNAi therapeutics in the fields of oncology and metabolic disease with the option to expand to additional therapeutic areas. This collaboration provides Takeda with broad, worldwide, non-exclusive access to and enablement with Alnylam’s RNAi therapeutics platform technology and intellectual property in the fields of oncology and metabolic disease, with the right to expand the number of therapeutic fields in the future. The agreement also includes the transfer of platform technology from Alnylam to Takeda, a collaboration and cross-license of delivery technologies between the two companies, and a drug discovery collaboration on certain RNAi therapeutic targets, subject to certain Alnylam third party obligations. Takeda becomes Alnylam’s strategic partner for RNAi therapeutics over a 5-year period. It has obtained thr right of first negotiation to develop and commercialize Alnylam RNAi therapeutic development programs for the Asian market, excluding Alnylam’s ALN-RSV01 program. In addition, Alnylam obtains opt-in options to co-develop and co-commercialize Takeda RNAi therapeutic programs in the USA market on a 50-50 basis. The partnership includes $100 million in upfront payments and $50 million in near-term technology transfer payments for a non-exclusive license in two therapeutic fields and is valued at potentially over $1 billion in future research and development and commercial milestones, upon successful commercialization of multiple products. At Takeda’s option, the scope of the partnership can be expanded to include additional fields with a $50 million per field expansion payment. Alnylam is also eligible to receive R&D funding related to the drug discovery collaboration, and up to $171 million in development and commercial milestone payments and significant royalties per product.
In September 2007, Merck and Alnylam mutually agreed to terminate their July 2006 amended and restated agreement. As a result, Alnylam has rescinded all grants of its intellectual property related to current and future Merck development programs, including the partnership's former co-development programs.
In September 2007, Alnylam and Isis Pharmaceuticals formed Regulus Therapeutics, a joint venture company, to discover, develop and commercialize microRNA therapeutics for viral infections and cancer. Following an initial $10 million investment by Alnylam to balance venture ownership, Regulus will be jointly funded by Alnylam and Isis.
In August 2007, Alnylam Pharmaceuticals received $38.6 million from the U.S. Defense Threat Reduction Agency (DTRA) to develop a broad spectrum RNAi antiviral therapeutic for hemorrhagic fever. This 33-month contract will also allow Alnylam to extend and leverage its capabilities across its entire proprietary and partnered pipeline.
In July 2007, Roche and Alnylam Pharmaceuticals entered into a major alliance in which Roche obtains a non-exclusive license to Alnylam's technology platform for developing RNAi (RNA interference) therapeutics. The alliance will initially cover four therapeutic areas, oncology, respiratory diseases, metabolic diseases and certain liver diseases. Alnylam and Roche also will collaborate on RNAi drug discovery for one or more disease targets in these therapeutic areas. In addition, Roche will acquire Alnylam's European research site located in Kulmbach, Germany (Bavaria), subject to regulatory approval. This site will become Roche's Center of Excellence for RNAi therapeutics discovery within Roche's global research organization. The alliance could be valued at over $1 billion in consideration of upfront payments, potential product milestone payments for multiple products and field expansion payments, excluding potential royalties on future sales of commercial products. Under the terms of the agreement, Roche will pay Alnylam $331 million in upfront cash payments and equity investment, including 1.975 million shares of Alnylam common stock the Roche Venture Fund agreed to purchase at $21.50 per share, representing just less than 5% of Alnylam's outstanding common stock. Roche will also pay Alnylam milestones on products as they advance in development and commercialization as well as royalties on future sales of commercial products. Furthermore, Roche may make field expansion payments to Alnylam to increase the number of therapeutic areas.
As of April 2007, Alnylam had granted non-exclusive licenses to more than 20 companies, including 13 research product suppliers. The company believes that over 75% of industrial sales of siRNA for research purposes are currently being made under access to Alnylam's IP.
In December 2006, Alnylam Pharmaceuticals closed its public offering of 4,700,000 shares of common stock. Net proceeds to the company were approximately $101 million. The offering was underwritten by Banc of America Securities.
The Department of Defense Appropriations Act of 2007, passed in September 2006, includes $1.1 million of funding dedicated to Alnylam's ongoing development of RNAi therapeutics. This appropriation, combined with earlier funding from the government, supports the goal of Alnylam Biodefense to build a robust platform for developing RNAi therapeutics to combat natural or man-made biological threats that pose a potential risk to public health and national security.
In July 2006, Alnylam and Dowpharma, a business unit of Dow Chemical, entered into an agreement for the manufacture and supply of candidate RNAi therapeutics for Alnylam's lead development programs in age-related macular degeneration (AMD) and respiratory syncytial virus (RSV) infection. Dowpharma will manufacture short interfering RNAs (siRNA), the molecules that induce RNAi, for use in clinical trials. This contract service agreement provides Alnylam with large quantities of GMP-certified siRNA necessary for near term toxicology and clinical trials of RNAi therapeutic candidates. Dowpharma will manufacture siRNA for use in certain Alnylam programs, including those in AMD and RSV.
In July 2006, Alnylam amended its two existing RNAi collaboration agreements with Merck and consolidated the efforts into a single ongoing collaboration. The revised terms provide Merck with a more active role in the development of RNAi therapeutic products, and Alnylam with an opportunity to receive accelerated R&D funding at an earlier stage and the potential for significant milestones in addition to royalty payments or profit sharing on any commercialized products resulting from the collaboration. The collaboration was amended to focus on the 9 new therapeutic targets that remain to be nominated by Merck. Under the revised terms, Merck may participate at an earlier stage in the R&D of these 9 targets whereas under the previous agreement, Merck had the right to nominate these new targets for initial development by Alnylam. These new programs are in addition to the existing program directed to the NOGO pathway on which Alnylam and Merck are already collaborating. Also under the revised terms, Alnylam may select three of the 9 new programs as joint development programs, which Merck will co-fund and participate in from the outset, thereby providing Alnylam with accelerated R&D funding for its efforts. Previously, the agreement was structured so that co-funding by Merck would not begin until after the completion of defined preclinical work. In the USA, Alnylam will have the right to co-promote RNAi therapeutic products developed in these three programs. Merck will assume primary responsibility for the remaining 6 programs and Alnylam is eligible to receive milestone payments and royalties on RNAi therapeutics developed and commercialized by Merck in these programs. Specifically, under the revised terms the successful development and approval of three RNAi therapeutic products developed solely by Merck on a worldwide basis would result in milestone payments to Alnylam of over $120 million.
In January 2006, Alnylam Pharmaceuticals priced a public offering of 5,115,961 shares of its common stock at $13.00 per share. All of the shares were offered by Alnylam pursuant to its effective shelf registration statement previously filed with the SEC. Alnylam has granted a 30-day option to the underwriters to purchase up to 767,394 additional shares of common stock to cover overallotments, if any. The company obtained net proceeds of $62.2 million from this public offering.
In June 2004, Alnylam Pharmaceuticals underwriters of its recent initial public offering of 5,000,000 shares of Common Stock have exercised in full their overallotment option to purchase an additional 750,000 shares of Common Stock from Alnylam Pharmaceuticals at the initial public offering price of $6.00 per share, less an underwriting discount of $0.42 per share. Net proceeds to the company from the sale of 5.75 million shares of common stock were approximately $30.0 million.
In June 2004, Merck and Alnylam entered into a new, multi-year collaboration to develop and commercialize RNAi therapeutics for ocular diseases. This collaboration, the second strategic alliance between Merck and Alnylam, will focus on age-related macular degeneration (AMD) and other ocular diseases caused by abnormal growth or leakage of small blood vessels in the eye. Alnylam's existing program to develop a Direct RNAi therapeutic for the treatment of AMD will be incorporated into the new collaboration. Under the terms of the agreement, Alnylam will receive an initial cash payment from Merck and can receive additional cash payments upon the achievement of specified progress milestones. Cash payments to Alnylam could collectively total $19.5 million. In addition, Merck and Alnylam will jointly fund the development of, and share the profits from, any RNAi therapeutics for the USA market that result from the collaboration. Alnylam will also have the option to co-promote these RNAi therapeutics in the USA. Marketing and sales outside of the USA will be conducted by Merck, with Alnylam receiving royalties.
In September 2003, Merck and Alnylam entered into a collaboration to advance RNAi technology to develop RNAi therapeutics that selectively target human diseases. For therapeutics development, Merck will provide Alnylam with a series of proprietary drug targets that have well validated roles in disease and are attractive candidates for a new therapeutic approach using RNAi. Alnylam will develop RNAi compounds against these targets and advance RNAi-based drug candidates through preclinical development. Alnylam and Merck will then decide whether Alnylam, Merck or the two companies together will proceed with the further development and commercialization of each RNAi-based drug candidate. For technology development, Merck and Alnylam will each commit significant resources and expertise to the collaboration. Under the terms of the agreement, Merck will make an upfront and annual cash payments and also make an equity investment in Alnylam. An additional cash payment and equity investment will be triggered by achievement of a pre-specified technology milestone. Merck will also make further payments to Alnylam for each RNAi-based drug candidate for which Merck elects to participate in further development and commercialization. Merck will receive a co-exclusive license to Alnylam intellectual property for use in in vitro and in vivo target identification and validation
In July 2003, Alnylam and Ribopharma merged to become Alnylam Holding (Cambridge, MA). The two companies became operating units of the parent company. Shareholders of Alnylam and Ribopharma exchanged their shares for shares in the merged entity. Alnylam Europe is a wholly owned subsidiary of Alnylam Pharmaceuticals. Ribopharma was founded in June 2000 as the first biotech company worldwide focusing on siRNA as a therapeutic approach, to develop and market pharmaceutical products based on SIRPLEX (Small Interfering RNA Duplex) technology, based on RNA interference (RNAi). In January 2003, Ribopharma obtained a €2 million convertible loan from new investors, Abingworth (London, UK), as the first step in a consortium private financing. In January 2003, Ribopharma obtained a €2 million convertible loan from new investors, Abingworth (London, UK), as the first step in a consortium private financing.
In February 2003, Alnylam Pharmaceuticals filed a registration statement with the SEC relating to its IPO. All the shares of common stock to be offered by the company will be newly issued shares.
In July 2002, Alnylam raised a total of $17 million in series A and B of private equity financing. Polaris Venture Partners, ARCH Venture Partners, and Atlas Venture led the most recent financing. Prior investors included Polaris and Cardinal Partners.
Current as of October 02, 2008
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