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Investor Biotech Deep Dive Boston Reflections from Visiting Selected Companies and the Unique Biotech Hub

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Investor Biotech Deep Dive Boston Reflections from Visiting Selected Companies and the Unique Biotech Hub Arctic Aurora LifeScience Investor Biotech Deep Dive Boston Reflections from visiting selected companies and the unique biotech hub Recently, the AALS team invited a group of our core investors to Boston, the absolute center for science and emerging innovative companies in the global biotech and pharmaceutical industry. As a contrast to unstable global markets with worries of trade wars and growth deterioration, the power of innovation and growth creation in the area is astonishing. Aside from two days of company visits, we spent one morning with venture capital backed startups, headed by top class scientists and drug developers with longstanding experience from the global pharmaceutical industry. The purpose of the trip was to give our investors a better view of the biotech sector, experience how the AALS team works and meet key people from our extensive network. Company Visits – RNAi coming to life Alnylam’s clinical pipeline extends to several more potential A special theme this trip was RNAi technology. We had treatments within many therapeutics areas. meetings with the management of Alnylam Pharmaceuticals Dicerna is the new kid on the block with an RNAi technology and Dicerna Pharmaceuticals, both companies focused on platform similar to Alnylam’s. While Dicerna’s pipeline is not RNAi based drug development. as mature as Alnylam’s, a program treating a rare metabolic RNAi or RNA interference utilizes a built-in defense disorder has produced promising trial results to this point mechanism in human cells against foreign genetic material. and may not be far from approval during the following year. When such foreign gene coding molecules enters the cell, Dicerna also has a wide early-stage pipeline where the the molecular RNAi defense system recognizes the foreign company has teamed up with larger pharma companies such material and destroys it. In its therapeutic use, the RNAi as Eli Lilly and Boehringer Ingelheim to address additional machinery is tricked into recognizing one of the body’s own disease areas where RNAi technology can substantially move disease-causing genes as foreign as subsequently stops therapeutic goals posts. those genes from being expressed (see figure below). This Rare disease drug developers, big and small way selected genes can be effectively silenced and this Vertex Pharmaceuticals has become a biotech giant with a biotechnological approach has shown promise in treating a market value over USD 40 billion on the back of building a wide array of different diseases, with the first RNAi drug very successful franchise in treating patients with cystic hitting the market in 2018. fibrosis, a severe and debilitating congenital disorder. Cystic Alnylam is the pioneering company in the development of fibrosis most notably affects the respiratory system and RNAi drugs. The company recently launched the first significantly reduces patient’s breathing capacity. Vertex approved drug in a rare disease, and another drug candidate was the first company to successfully develop a treatment is geared up towards approval soon in another rare and that targeted the underlying biochemical aspects of the debilitating disorder. Adding to that, Alnylam were the disease for a subset of cystic fibrosis patients. The company originators of a drug in late-stage development now run by also showcased its very promising development pipeline to another biotech company treating high cholesterol that replace their first generation drugs and to expand the affects millions of patients worldwide. What’s more, therapeutic use to virtually all patients with cystic fibrosis. In overview, RNAi therapy works by inserting short pieces of genetic information (here, “Antisense DNA oligonucleotide”) which attaches to mRNA. mRNA stands for ‘messenger RNA’ and is the molecule that transfers the genetic information from DNA in the cell’s nucleus so that it can be read by the cell’s molecular machinery (“Ribosome”) that manufactures protein based on the DNA blueprint. With this technology drug developers can create very specific drugs that shuts down the production of protein based on faulty DNA by stopping mRNA to reach its cellular destination. We also met with the CEO of Albireo Pharma. This small cap company primarily develops new treatments for rare liver diseases that often affects children. Albireo has presented strong evidence of effect in a Phase 2 trial in one such liver disorder and are expecting confirmatory trial data to bring in front of regulatory agencies within a year. What’s more, Albireo is building on its initial success by expanding development into more related rare liver diseases that can be targeted by the same approach. Plus, the company is growing an additional pipeline of novel programs to treat other liver-mediated diseases. New ways to fight cancer In the meeting with Agios Pharmaceuticals, we saw an example of the typical way we believe cancer treatments are heading in order to make a real impact for patients. Agios has developed two treatments for leukemia that relies on the cancer’s genetic makeup. If a patient is carrying particular cancer mutations, Agios’ matched treatments has shown considerable therapeutic effect that led to market approval, and now, new clinical trials to show increased effect of the drugs by combining them with other cancer drugs. In addition, Agios are also developing other cancer treatments and drugs to treat other hematological disorders. Opportunities and challenges within central nervous system disorders Sage Therapeutics has been a remarkable story as the company has challenged current treatment standards in depressive disorders, a therapeutic area were very little innovation has happened in the last decades. First, Sage was the first company to ever get approved in treating women suffering from post-partum depression (severe depressive episodes following child birth). Then the company took a swing a major depressive disorder, with an oral pill that has shown impressive effects so far in clinical development. Most importantly the anti-depressive effect of Sage’s drugs have much quicker onset than established drug to treat depression. We also paid a visit to the big biotech company Biogen that are now facing an overhaul in its product portfolio and development pipeline. This follows negative trial outcome for their front-running drug candidate in Alzheimer’s disease, where sadly no new treatment has been able to show noteworthy ability to halt disease progression. Biogen is also facing patent expiries in its large multiple sclerosis business and competition from a new gene therapy to its drug treating the rare muscular disorder SMA. For Biogen now, the key is pipeline hits to rejuvenate the company’s leadership role in multiple sclerosis and effective business development to bring in new programs in order to lead the future growth of the company. A world leading hub for life science innovation re-launched efforts in Rare Disease Discovery and In Cambridge we met with Dr JC Gutierrez-Ramos, member Development and founded the Centers for Therapeutic of our Advisory Board, and CEO of Cogen Therapeutics, Dr Innovation. He also oversaw and enhanced the biologics Lovisa Afzelius, Chief Operating Officer at Cogen platform for the company from early discovery to entry in Therapeutics and Dr. Anthony Coyle, CEO of Pandion manufacturing. Before that, JC was Senior Vice President and Therapeutics. Obviously the combination of rich access to Head of the Immuno-inflammation Center for Drug seed funding and highly educated and skilled people from Discovery at GSK, where he founded entrepreneurial units Harvard Medical and MIT are important pillars when building such as Epinova and Tempero focused in translating novel new companies based on disruptive innovations. Moreover, areas of science into therapeutics. He began his career in the the very important culture with entrepreneurship and drive drug industry at Millennium Pharmaceuticals serving as Vice coupled with a unique infrastructure with large biotech and President of Inflammation Drug Discovery. During his pharmaceutical companies make this area very special. academic career he was part of the Faculty at the Genetics Department of Harvard Medical School and the Basel Dr. Gutiérrez-Ramos has over 20 years of drug discovery and Institute for Immunology in Switzerland. Dr. Gutiérrez- development experience from leading global Ramos holds a Ph.D. in immunochemistry from the biopharmaceutical companies. Previously, he served as Autonoma University in Madrid, Spain. Group Senior Vice President and global head of the BioTherapeutics Research at Pfizer where he was responsible for more than 25 novel programs across the full spectrum of clinical development, Norway Sweden Arctic Fund Management AS Arctic Fund Management AS, P.O. Box 1833 Vika Stockholm Filial NO-0123 Oslo Biblioteksgatan 8 Haakon VIIs gt 5, NO-0161 Oslo SE-111 46 Stockholm Tel +47 21 01 31 00 www.arctic.com/afm facebook.com/arcticfunds @arcticfunds Arctic Fund Management .
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