Our Holdings

0.1%

Achillion Pharmaceuticals

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Achillion Pharmaceuticals

0.1%

Achillion is developing novel complement inhibitors for indications such as paroxysmal nocturnal hemoglobinuria (PNH), C3 Glomerulopathy (C3G), and other diseases where dysfunction of the complement alternative pathway plays a role. The lead compound is ACH-4471, a complement Factor D inhibitor with the potential to provide an oral alternative to Alexion’s blockbuster Soliris, or serve to treat patients who have a suboptimal response to or fail Soliris. Data from an initial Phase I/II trial showed the drug is active, and additional results from ongoing Phase II trials should better determine its potential. In addition, the company is developing ACH-5228, a next-generation Factor D inhibitor that entered the clinic by the end of 2017.

5.4%

Agios Pharmaceuticals

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Agios Pharmaceuticals

5.4%

The two most advanced oncology programs of Agios Pharmaceuticals are targeting mutations in the isocitrate dehydrogenase 1 and 2 (IDH1 and IDH2) enzymes, which are implicated in hematologic malignancies and solid tumors. Data with IDH2 inhibitor Idhifa (AG-221) were compelling and due to the high response rate and well-defined group of patients who benefited, the drug was given an accelerated approval in August 2017. We estimate the worldwide market opportunity for Idhifa at USD 750 mn for acute myeloid leucemia (AML). Celgene has worldwide rights to Idhifa, and Agios will receive milestones and an estimated 15% royalty on sales. Data with IDH1 inhibitor AG- 120 in AML were also promising and an NDA submission was filed end 2017. Results with AG-120 in rare solid tumors were not as compelling as hoped, and we include little revenue potential from these indications despite continued development. Finally, the company is developing AG-348, a novel compound for the treatment of pyruvate kinase deficiency that reported compelling proof-of-concept data and should lead to the start for pivotal trials in 2018.

1.6%

Akcea Therapeutics

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Akcea Therapeutics

1.6%

Akcea was spun out of Ionis Pharmaceuticals and is developing antisense drugs to treat rare and severe lipid disorders. Its lead product is Volanesorsen, which has successfully completed Phase III development for familial chylomicronemia syndrome (FCS), a rare and debilitating disease characterized by extremely high triglycerides. Akcea has filed for approval and expects to commercialize the product globally. Akcea also has a pipeline of next generation lipid products based on its LICA technology which allows for much lower dosing and higher potency. ANGPTL3-Lrx is in a Phase I/II study for rare hyperlipidemias and will also be evaluated in fatty liver diseases such as NAFLD and NASH. Akcea has two LICA programs partnered with Novartis for larger diseases, APO(a)-Lrx and APOCIII-Lrx for patients with elevated risk factors for cardiovascular disease. Ionis remains a majority shareholder.

0.7%

Alder Biopharmaceuticals

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Alder Biopharmaceuticals

0.7%

Alder is a clinical-stage company with a differentiated antibody discovery and manufacturing platform to design and select antibodies that have the potential to maximize efficacy in various therapeutic indications including inflammatory and neurological conditions. Their clinical candidate, eptinezumab, is an antibody that inhibits calcitonin gene-related peptide (CGRP), a well-validated molecular target shown to trigger migraine attacks. Eptinezumab has recently completed Phase III clinical testing for the prevention of both chronic and frequent episodic migraines. Data were highly significant and notable for achieving rapid, robust and durable efficacy. Alder is the only company with an anti-CGRP asset that is developing a durable, intravenous formulation to be administered by neurologists in-office – an infusion that could be given every three months, compared to monthly or biweekly self-administered subcutaneous injections at home. The company expects to apply for FDA approval by the end of 2018. A self-administration strategy for Eptinezumab to be dosed every three months is also being developed in addition to two preclinical programs expected to enter the clinic in the future.

4.0%

Alexion Pharmaceuticals

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Alexion Pharmaceuticals

4.0%

Alexion is developing drugs for rare disorders. Its lead product Soliris was approved in the US and Europe in 2007 for paroxysmal nocturnal hemoglobinuria (PNH) and we expect sales in PNH to reach about USD 2.0 bn. Atypical hemolytic uremic syndrome (aHUS) is the next indication for which Soliris gained approval in the US and Europe in 2011. We estimate it adds another USD 2.0 bn market opportunity for Soliris. Other indications such as myasthenia gravis and neuromyelitis optica could add an additional USD 1.0 to 2.0 bn in sales. To maintain its dominance, Alexion is in advanced development with a next-generation Soliris, ALXN-1210, which has an improved dosing profile and should report Phase III results in H1 of 2018. To diversify the revenue base away from Soliris, the company received approval of a novel compound for hypophosphatasia, Asfotase Alfa, in March 2015 and sales to date have exceeded expectations. In addition, Alexion gained Kanuma for lysosomal acid lipase (LAL) deficiency via its May 2015 acquisition of Synageva for USD 8.4 bn, and while the launch has been slow, the product should eventually be a more meaningful contributor to revenue.

3.1%

Alnylam Pharmaceuticals

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Alnylam Pharmaceuticals

3.1%

Alnylam Pharmaceuticals is the market leader in RNA interference (RNAi) therapeutics. This treatment approach selectively blocks the synthesis of specific disease-causing proteins. Alnylam has a broad pipeline of candidates, including five programs that have advanced to the clinical development stage. The furthest along the pipeline currently awaiting regulatory approval is Patisiran which targets TTR amyloidosis, a rare and serious disorder in patients diagnosed with familial amyloidotic polyneuropathy (FAP). Other interesting programs include Fitusiran, which pursues a revolutionary approach in the treatment of hemophilia and rare bleeding disorders, and Givosiran for the treatment of acute hepatic porphyrias. Both RNAi therapeutics are currently in Phase III development. Alnylam continues to support its collaboration with The Medicines Company in their advancement of inclisiran into Phase III studies which investigates RNAi disruption of PCSK9 for the treatment of hypercholesterolemia. Data thus far have been supportive of a once-quarterly and possibly biannual subcutaneous dose regimen which has obvious advantages over recently approved PCSK9 antibody therapies.

0.8%

Argenx

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Argenx

0.8%

Argenx is a Belgian clinical stage small cap biotechnology company developing targeted antibody therapies through its multiple antibody platforms. The company has a variety of mid-to-late stage clinical drug candidates with ARGX-113 being the lead asset. This molecule is currently in the clinics in three IgG-mediated autoimmune diseases with important clinical trial read-outs expected in the coming 12 months. Additionally a second proprietary drug candidate, ARGX-110 is expected to move to proof-of-concept clinical trial in 2018 in in Acute Myeloid Leukemia. A solid balance sheet and experienced management rounds the company’s profile. Argenx can be considered an antibody platform company targeting novel scientific pathways in indications with high unmet medical need with little competition and innovation in the last decades.

1.3%

Avexis

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Avexis

1.3%

Avexis is a clinical stage company using its gene therapy platform to address serious, unmet diseases. Its gene therapy technology utilizes the adeno-associated virus 9 vector (AAV9) to deliver functional genes to cells in order to produce fully function proteins where they are deficient. The company’s lead product is AVXS-101, in Phase III for the treatment of spinal muscular atrophy 1 (SMA 1). SMA is a disease where motor neuron lack a functional, crucial protein called SMN1, leading to severe motor deficiencies, muscle wasting, and also death. AVXS-101 is administered only once, and uses the AAV9 vector to deliver the functional SMN1 gene to the motor neurons in SMA patients. In the ongoing Phase I trial, SMA 1 infants have seen dramatic improvements in their motor scores and functionality. Avexis will meet with the FDA in early 2018 to discuss potential accelerated approval based on the Phase I data. The company also plans on starting a Phase I trial in SMA 2 patients imminently. The company will also leverage its platform to bring additional products into development in the coming quarters.

7.7%

Celgene

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Celgene

7.7%

Celgene specializes in oncology and inflammatory diseases and has very strong fundamentals and positive long-term prospects based on products such as Revlimid, Pomalyst, Otezla and its robust pipeline of early-stage products. We expect Revlimid US revenue to continue to grow more than 15% per year, driven by the combined effects of increased prevalence, penetration, duration of treatment. The company’s acquisition of Receptos broadened their immunology and inflammation franchise beyond Otezla by gaining access to ozanimod which we expect to be approved in MS this year and continues to be developed for inflammatory bowel disease (IBD). We expect positive news flow from both Celgene and their partners’ products in a variety of novel cancer combinations and settings over the next two to three years. Celgene now appears to be rapidly moving toward immuno-oncology by recently gaining partial rights to Durvalumab from AstraZeneca for hematologic malignancies as well as their strategic collaborations with Juno and Bluebird to develop T-cell-based therapies for cancer and autoimmune diseases. The company continues to make strategic deals to bolster its pipeline with promising opportunities.

0.2%

Cidara Therapeutics

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Cidara Therapeutics

0.2%

Cidara is a biotechnology company focused on treating severe and resistant microbial infections. Its lead product, Rezafungin (in a Phase II study for candidemia and invasive candidiasis), is from the echinocandin class of antifungals but is dosed as a once-weekly infusion, versus daily for the current echinocandins. This would provide the option of treating patients with the best antifungal on an outpatient basis, thus offering significant advantages to both patients and the healthcare system. Initial Phase I data have demonstrated a strong safety profile and confirmed the once-weekly dosing potential. Data read-out is expected in early 2018. Following a constructive meeting with the FDA, a smaller than expected Phase III study will begin in 2018, along with a prophylaxis study in bone marrow transplant. Finally, Cidara is the only company developing an immunotherapy platform for serious infections.

4.5%

Esperion Therapeutics

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Esperion Therapeutics

4.5%

Esperion Therapeutics is focused on the development of treatments for cardio-metabolic diseases. ETC-1002 is the only clinical asset and has completed multiple clinical trials and has now initiated its complete Phase III program. ETC-1002’s main target ATP citrate lyase is located upstream of where statins work and ultimately reduces LDL cholesterol by upregulation of the LDL receptor. ETC-1002 has shown LDL cholesterol reduction levels of up to 30% as monotherapy and up to 50% in combination with ezetimibe. In contrast to the recently approved subcutaneously administered PCSK9 antibodies, ETC-1002 poses a convenient and more economic once-daily oral solution. To date ETC-1002 has not shown any significant safety signals such as statin-typical myalgia. Primary markets for ETC-1002 will be the statin-intolerant population as well as additional treatment for patients whose LDL cholesterol levels are not sufficiently controlled with a statin. Phase III monotherapy and fixed-dose combination trial read-outs are due from Q2 to Q4 2018 and regulatory submissions are expected by year-end 2018/early 2019.

0.4%

Five Prime

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Five Prime

0.4%

Five Prime has a discovery platform that includes a library of over 5700 human extracellular proteins, generated by a proprietary technology that enables the capture of cDNAs with intact 5 prime ends. I It also has developed a range of proprietary screens and characterization tools, including automated cell-based screening systems and a rapid in vivo protein production system, to take advantage of its library and overcome limitations of traditional protein screening methods. Via this platform, the company has isolated the 700 proteins that drive immune cell interactions. The key drug in development is FPA008, a humanized IgG4 anti-CSF1R (macrophage colony stimulating factor) antibody. The lead indication for FPA008 is pigmented villonodular synovitis (PVNS), a macrophage-driven tumor. The company began a Phase II trial in May 2016 and we expect more data in mid-2018. The larger potential for FPA008 is as a combination partner with other immuno-oncology agents. A Phase I/II trial investigating FPA008 as a single agent and in combination with Bristol-Myers’ Opdivo is ongoing, and first results from the combination portion showed promise in pancreatic cancer. More data in other solid tumor types are expected in 2018. The company is also developing FPA144, a proprietary ADCC-engineered antibody that inhibits FGFR2b. We expect the start of a Phase III trial in gastric cancer in 2018.

5.2%

Gilead

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Gilead

5.2%

Gilead develops drugs primarily for infectious diseases such as HIV, hepatitis B, and hepatitis C, as well as cancer. The first product, Viread, was launched in 2001 and is now firmly established as a key component in treatment regimens for HIV. Most recently, it launched regimens that include a replacement for Viread with a better longterm safety profile, which should enable it to maintain its leadership when Viread goes generic. The introductions of Hepsera and Viread established Gilead as an important player in the treatment of hepatitis B infection. Gilead acquired Pharmasset in early 2012, which enabled it to become the market leader in the USD 20+ bn hepatitis C (HCV) space. Indeed, sales of its lead products, Sovaldi and Harvoni, reached over USD 12 bn in the first nine months of 2016. However, this was followed by a precipitous decline, and we expect a continued decline in future years due primarily to pricing and competition. To offset the declining HCV sales, the company purchased Kite Pharmaceuticals, a leader in CAR-T therapy, in October 2017. The first product, Yescarta, was approved in October 2017 for diffuse large B-cell lymphoma (DLBCL) and we expect label expansions for other hematologic indications to follow.

4.3%

Halozyme Therapeutics

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Halozyme Therapeutics

4.3%

Halozyme Therapeutics is a biopharmaceutical company with two platforms in its business model. The first is based on partnerships with pharmaceutical companies that use its product rHuPH20 to prepare subcutaneous formulations of intravenous therapies. The company receives a steady flow of royalties from this arm. Partnered products include blockbusters like Avastin and Rituxan as well as newer products such as PCSK9 and Daratumumab. The second platform is PegPH20, which is being tested in the treatment of pancreatic cancer and lung cancer. A Phase III study in pancreatic cancer has started enrollment in the first half of 2016 and is expected to read out on PFS by year end 2018. PegPH20 is also being tested in combination regimens with Keytruda in NSCLC/GC as well as in various tumor types in combination with Roche’s Tecentriq.

0.1%

Idorsia

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Idorsia

0.1%

Idorsia is a Swiss-based biotech company that resulted from the spin-off of the development pipeline of Actelion after the acquisition by J&J. The company has a variety of late-stage clinical assets including a DORA (dual orexin receptor antagonist) for the treatment of insomnia, Clazosentan for reversal of cerebral vasospasms and Cenerimod for lupus. Further the company will be eligible to receive royalties from J&J for the S1P1 inhibitor Ponesimod for the treatment of multiple sclerosis and for a nextgeneration ERA (endothelin receptor antagonist) aimed at treating resistant hypertension. The company is flush with CHF 950 mn in cash following its demerger and listing on the Swiss Stock Exchange, which should give it a runway of two to three years to achieve relevant development milestones. While J&J retains rights to up to 32% of the company through convertible notes, 26% of the company is now under the control of Actelion’s former CEO Jean-Paul Clozel.

8.0%

Incyte

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Incyte

8.0%

Incyte is focused on hematologic disorders, inflammatory disorders, and cancer. Their marketed product is Jakafi, an oral JAK-2 inhibitor, that received approval in 2011 and 2014, respectively. We estimate that myelofibrosis and polycythaemia vera (PV) represent a USD 3+ bn market opportunity in the US and Europe. Phase III trials in graft versus host disease (GvHD) are also ongoing and could add another USD 500+ mn in sales if positive in 2018. In November 2009, Novartis licensed ex-US rights to Jakafi i. A second-generation JAK-2 inhibitor, Baracitinib, posted positive data from several Phase III trials in rheumatoid arthritis in 2015 and we expect launch into this large market by 2019. Incyte will receive royalties from partner Eli Lilly. Progress on other cancer compounds in its pipeline, including IDO inhibitor Epacadostat, also continues. Indeed, encouraging early results with the combination of Epacadostat and Merck and Bristol- Myers’s PD1 inhibitors, Keytruda and Opdivo, in multiple tumor types have been reported. Data from a Phase III trial in melanoma patients are due in H1 of 2018. The company started additional Phase III trials in indications such as lung and head and neck cancer by the end of 2017.

0.9%

Intercept Pharmaceuticals

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Intercept Pharmaceuticals

0.9%

Intercept Pharmaceuticals is focused on the development of synthetic bile acid analogs for the treatment of cholestatic liver diseases. This disease area primarily includes the highly prevalent non-alcoholic fatty liver disease (NAFLD) and non-alcoholic steatohepatitis (NASH) as well as the orphan diseases primary biliary cirrhosis (PBC) and primary sclerosing cholangitis (PSC). Intercept’s lead product is obeticholic acid (OCA), a firstin- class farnesoid X receptor (FXR) agonist. OCA has been approved in the US and Europe in 2016. As a second and commercially far more attractive indication, Intercept also started a pivotal trial for NASH. Results from this trial are expected to be published in H1 of 2019. NASH, being an obesity and metabolic syndrome-linked disease, has the potential to take on epidemic proportions in western and emerging societies over the coming years. It is projected to be the leading cause of costly liver transplants and liver cancer by 2020. With currently no drug approved, there clearly is an unmet medical and health economic need for new treatments. Intercept’s OCA is the drug furthest in development for NASH and the first to show an anti-fibrotic effect in the liver.

1.2%

Intra-Cellular Therapies

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Intra-Cellular Therapies

1.2%

Intra-Cellular Therapies is a biopharmaceutical company developing treatments for disorders that affect the central nervous system. Their wholly owned lead product candidate is ITI-007, or Lumateperone, a 5-HT2A serotonin receptor antagonist that also modulates dopamine and serotonin transporters, which recently completed two Phase III clinical trials for the treatment of schizophrenia. Lumateperone could prove highly differentiated from other anti-psychotics due to its ability to modulate multiple neurotransmitter pathways simultaneously. This was demonstrated in their first pivotal Phase III trial which showed strong efficacy and placebo-like safety. Tolerability and compliance on current schizophrenia therapies is challenging due to a range of motor and metabolic side effects, which is where Lumateperone has proven to be differentiated. Intra-Cellular is also evaluating Lumateperone in two Phase III trials for the treatment of bipolar depression to be completed during the second half of 2018, while another pivotal study investigating agitation in patients with dementia, including Alzheimer’s disease, will read out by the end of 2018. The company also has a PDE inhibitor, ITI-214, in Phase I trials evaluating its role in Parkinson’s disease and other indications.

9.8%

Ionis Pharmaceuticals

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Ionis Pharmaceuticals

9.8%

Ionis Pharmaceuticals is the leader in the space of antisense, with over 30 compounds in development using this technology. Antisense allows for the control of protein production at the genetic level. Our focus and investment strategy revolve around the technology platform, which demonstrated significant progress in 2017 with both partnered and proprietary compounds across various severe diseases. Spinraza (partnered with Biogen) was approved in late 2016 following two positive Phase III studies in spinal muscular atrophy, and had a very strong launch throughout 2017. Inotersen, for familial amyloid polyneuropathy, produced positive Phase III data and has been filed in the US and EU. Additionally, Ionis’ wholly-owned subsidiary, Akcea, completed a successful IPO and has filed Volanesorsen in the US and EU for familial chylomicronemia syndrome. Our focus going forward is on the company’s next generation technologies such as 2.5 and LICA. Thus, Ionis remains an important and truly innovative investment in our portfolio.

1.7%

Macrogenics

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Macrogenics

1.7%

Macrogenics has multiple compounds in clinical development that were generated using its propriety Fc-optimization technology that simultaneously reduces resp. enhances binding to inhibitory resp. activating FcyRs, thus dramatically increasing antibody- dependent cellular cytotoxicity (ADCC), and its DART (dual-affinity re-targeting) platform. The company believes its DART platform has overcome the challenges of construct instability and short half-lives encountered by other dual-specific antibodies by incorporating proprietary covalent disulfide linkages and particular amino acid sequences that efficiently pair the chains of the DART molecule. This results in a structure with enhanced manufacturability, long-term structural stability, and the ability to tailor the half-lives of the DARTs to their clinical needs. Data from clinical trials with multiple products, including immuno-oncology agent MGA271, are expected through 2018.

1.8%

Moderna Therapeutics

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Moderna Therapeutics

1.8%

Moderna Therapeutics is a Boston-based private biotech company, which is pioneering a new class of medicine made of messenger RNA. Since their founding Moderna has been able to raise more than USD 2.5 bn in financing from investors and partners. A substantial amount of this capital was invested in what is now a leading mRNA technology platform in order to be able to quickly drive development candidates into the clinic on a broad front of therapeutic and prophylactic applications. Their pipeline now includes 19 development candidates, with 10 of them in the clinic, for mRNA-based vaccines and treatments in diverse therapeutic areas. The in our view key programs that will be reading out clinical data within in the next few years include the rare liver disease MMA and PPA, the proprietary vaccines in congenital CMV and hMPV+PIV3, the intra-tumorally injected cytokine cocktail OX40L+IL23+IL36 gamma, the personalized cancer vaccine and early data on the VEGF Phase 2 during CABG-surgery.

1.9%

Myovant Sciences

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Myovant Sciences

1.9%

Myovant is a biopharmaceutical company with a focus on endocrinology in women’s and men’s health. Its lead candidate, Relugolix, is an oral GnRH antagonist in Phase III development for three indications, endometriosis, uterine fibroids, and advanced prostate cancer. Endometriosis is a condition where part of the endometrium grows outside of the uterus leading to severe pain, painful intercourse, and bleeding. Uterine fibroids is a condition that can lead to painful menstruation and excessive bleeding, and potentially surgical removal of the uterus. Advanced prostate cancer is cancer of the prostate that continues to grow despite castration and/or radiation. Partner Takeda announced positive data from two Phase III trials in uterine fibroids in Japanese women, further validating Relugolix’s mechanism of action. We expect data from all three Phase III trials in the US in 2019. Myovant owns worldwide rights outside of Asia.

7.3%

Neurocrine Biosciences

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Neurocrine Biosciences

7.3%

Neurocrine is a biopharmaceutical company with a focus on women’s health and CNS disorders. Its lead candidate, Elagolix, is an oral GnRH antagonist in development for two indications, endometriosis and uterine fibroids. Endometriosis is a condition where part of the endometrium grows outside of the uterus leading to severe pain, painful intercourse, and bleeding. Uterine fibroids is a condition that can lead to painful menstruation and excessive bleeding, and potentially surgical removal of the uterus. Partner AbbVie has filed for approval in endometriosis. AbbVie is also conducting a Phase III program in uterine fibroids, with data expected in early 2018. Neurocrine received approval for Ingressa (Valbenazine) for tardive dyskinesia in mid-2017 and launched the product in the US. The company has also initiated a Phase II dose-escalation study in pediatric Tourette syndrome with data expected in 2018.

0.5%

Novavax

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Novavax

0.5%

Novavax is a company specializing in the development of novel vaccines. The most advanced program is a vaccine to prevent RSV infections in infants and older adults. Respiratory syncytial virus (RSV) is a respiratory tract infection which may be fatal in infants, older adults, and people with compromised immune systems. In a Phase II study in older adults, Novavax showed that its vaccine results in 44% fewer symptomatic RSV infections and a more than 60% reduction in severe RSV infections. However, in 2016, the company announced that the Phase III study in the elderly failed due to a much lower event rate than expected. In its Phase II study in pregnant women, Novavax showed that theantibodies are transferred effectively from the mothers to their infants. A corresponding Phase III study has been initiated in pregnant women with data expected in H2 of 2018. Novavax also has a seasonal influenza vaccine, an Ebola vaccine, and a pandemic influenza vaccine in its pipeline.

3.6%

Novo Nordisk

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Novo Nordisk

3.6%

Novo Nordisk is a leader in the global diabetes market. Novo’s once-weekly GLP-1 analogue, Ozempic (semaglutide SQ), has been approved in the US in December 2017 for type 2 diabetes and will be a significant growth driver for the company. A clean label and a superior data set should allow for a very competitive profile. Additionally, we expect oral semaglutide to garner more attention as we will see Phase III data start to emerge in 2018. This compound, if successful, would be the most efficacious oral antidiabetic drug ever approved. Tresiba’s launch has been going well and should help drive Novo Nordisk’s long-term penetration in the modern insulin space. Victoza (daily SQ GLP-1) continues to grow. In 2016, Novo refined its long-term growth expectations downward, as the entire insulin market is facing pricing headwinds in the US, which is now reflected in current estimates.

0.4%

Probiodrug

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Probiodrug

0.4%

Probiodrug is a biotechnology company, located in Halle, Germany, focused on the development of innovative small molecule drugs for the treatment of Alzheimer’s disease (AD). The company holds a dominant position in the area of glutaminyl cyclase (QC) inhibition. The role of QC in AD and other inflammatory diseases was discovered, and is comprehensively IP-protected, by Probiodrug. A Phase I study with its lead compound, PQ912, is complete, demonstrating a clean safety profile and initial target inhibition. A Phase II study recently demonstrated target engagement along with other biomarker signals in mild AD patients. The company was founded in 1997, and pioneered the field of DPP4 inhibition for the treatment of type 2 diabetes. Probiodrug sold its DPP4 franchise to OSI Pharmaceuticals in 2004. Probiodrug’s pioneering scientific approach targeting QC in AD has the potential to bring a breakthrough treatment to this therapeutic area of great, unmet need.

0.4%

Prothena Corp.

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Prothena Corp.

0.4%

Prothena is a biotech company focused on the development of antibody-based immunotherapies. Their lead asset, NEOD001, is currently being investigated in a Phase III clinical trial in AL-amyloidosis, a devastating disease characterized by the accumulation of protein plaques in various organs. NEOD001 is an antibody designed to bind these plaques and remove them from the affected organs. The company’s second asset is in earlier clinical development for the treatment of Parkinson’s disease. The company is a spinout of Elan Corporation and their business consists of a substantial portion of Elan’s former drug discovery business platform.

5.5%

Radius Health

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Radius Health

5.5%

Radius Health is a company focused on women’s health and oncology. Its lead product candidate is the subcutaneously delivered Abaloparatide, a synthetic human PTHrP analogue. The faster onset of action and reduction in fractures in nonvertebral sites like the hip and wrist versus Forteo are differentiating and should allow Abaloparatide to capture significant market share. Radius received approval in early 2017, and we expect 2018 to be heavily focused on market access and reimbursement. Importantly, Radius is developing a transdermal patch formulation, which could greatly enhance the outcomes in women with this disease. Transdermal data presented in 2016 showed a meaningful improvement in its profile, and we expect a pivotal study to begin in 2018. Furthermore, the company has RAD1901, a selective estrogen receptor degrader (SERD), in development for estrogen-receptor-positive breast cancer. Following a meeting with the FDA, a potential registrational study will start in 2018.

2.5%

Regeneron Pharmaceuticals

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Regeneron Pharmaceuticals

2.5%

Regeneron is focused on developing monoclonal antibodies. The blockbuster success of Eylea, a VEGF inhibitor indicated for ophthalmic disorders, has been the primary driver of growth for the company. We expect near-term growth to continue in 2018 as Eylea gains broader adoption in wet AMD and expands into DME. Regeneron holds a partnership with Bayer Healthcare for the development, marketing, and sale of Eylea outside of the US. Regeneron also holds a partnership with Sanofi, with whom they have commercialized three products thus far and, more importantly, have a deep pipeline of assets the two partners are co-developing. Praluent for hypercholesterolemia is approved by the FDA for heterozygous familial hypercholesterolemia or clinical atherosclerotic cardiovascular disease patients who need additional lowering of LDL cholesterol. Kevzara and Dupixent have recently been approved for rheumatoid arthritis and atopic dermatitis, respectively. With Teva and Mitsubishi Tanabe, the company is also developing Fasinumab, an antibody to nerve growth factor for pain therapy. Regeneron also has collaboration agreements with Intellia Therapeutics to advance CRISPR/Cas gene-editing technology. 

4.2%

Sage Therapeutics

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Sage Therapeutics

4.2%

Sage Therapeutics is a clinical-stage biopharmaceutical company focused on developing therapies for rare CNS disorders utilizing their GABA-A receptor-targeted proprietary platform. The company’s lead program, Brexanolone, is in Phase III development as an IV treatment for post-partum depression (PPD). Brexanolone has shown rapid and durable efficacy with excellent tolerability, which sets it apart from all classes of drugs currently used in the field of depression and mood disorders. An oral, follow-on version of brexanolone, SAGE-217, has also recently shown significant early clinical success in a Phase II trial in major depressive disorder (MDD), while a Phase II study in PPD is due to read out later this year. SAGE-217 is also being investigated in essential tremor and Parkinson’s disease. Sage also has an NMDA program with SAGE- 718 in Phase I, targeting several orphan neurological indications.

2.4%

Tesaro

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Tesaro

2.4%

The first marketed product of Tesaro, Rolapitant, is a neurokinin-1 (NK-1) receptor antagonist that completed Phase III trials for the prevention of chemotherapy induced nausea and vomiting (CINV) in 2014. The results were positive and approval in the US was received in September 2015. Niraparib is a PARP inhibitor that had shown promising efficacy in patients with BRCA+ breast and ovarian cancer in early trials. In 2016, the company announced highly positive results from a Phase III trial in platinum-sensitive ovarian cancer and approval with a broad label was granted in 2017. Multiple additional trials designed to expand Niraparib’s potential in ovarian and other cancers are underway, and some data will be available in 2018. Meanwhile, the company inlicensed several compounds that gave them an entry into the immuno-oncology space, and clinical trials with those targeting PD1, TIM-3, and LAG-3 are progressing and should yield results in 2018.

6.3%

Vertex Pharmaceuticals

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Vertex Pharmaceuticals

6.3%

Vertex’s core focus is cystic fibrosis. CFTR potentiator Kalydeco was launched in the US and Europe in 2012 for a subgroup of patients with cystic fibrosis. While the initial market opportunity is limited to around 5% of the patient population, we believe that sales could reach USD 1.0 bn with the inclusion of other small patient populations on the label. Positive Phase III results with the combination of Kalydeco and CFTR corrector VX-809, released in June 2014, enabled Vertex to begin to target the roughly 45% of patients who are homozygous for the most common mutation in the US and Europe in 2015. With this label inclusion, we expect sales of Kalydeco and the Kalydeco/VX-809 combination to reach approximately USD 4 bn. The company is also developing correctors that can be combined with Kalydeco and VX-661 to target the remaining patients who are heterozygous for the mutation. Data from Phase II trials announced in 2017 were highly positive and we expect Phase III trials to start in H1 of 2018 with approval to follow by 2020.

1.0%

Voyager Therapeutics

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Voyager Therapeutics

1.0%

Voyager is a clinical-stage biotech company focused on developing novel genetically targeted therapies to treat CNS diseases. The company’s lead asset, VY-AADC is an AAV-based gene therapy with the objective of increasing the expression of the enzyme responsible for converting levodopa to dopamine (AADC, L-amino acid decarboxylase) in the brains of advanced Parkinson’s disease patients. VY-AADC is currently in Phase Ib with pivotal studies scheduled to begin in 2018. The company is also developing other AAV vectors targeted at increasing expression of a key gene in Friedreich’s ataxia, delivering monoclonal antibodies, or silencing/knocking down genes using microRNA delivery in diseases like monogenic SOD1 familial ALS and Huntington’s disease. Voyager’s discovery engine has generated programs in five CNS indications, and in the next 18 to 24 months, they plan to initiate at least three other clinical programs.

0.9%

Wave Life Sciences

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Wave Life Sciences

0.9%

Wave is a leader in the space of stereochemistry, with an initial focus on antisense oligonucleotides (ASOs) and exon skipping. In simple terms, stereochemistry refers to the three-dimensional structure of a molecule and how this affects its chemical properties. Current ASOs can contain hundreds to hundreds of thousands of various enantiomers (stereomixture), many of which do not contribute to efficacy, but could be causing toxicity. Wave is able to specifically design their individual molecules (stereopure) to contain the desired properties, thus potentially enhancing potency and minimizing toxicity. The company’s lead product is in Phase I/II development for Huntington’s disease and targets very specific point mutations in order to knock down the mutant protein. We expect data in early 2019. Wave’s second program recently entered Phase I development for Duchenne muscular dystrophy (DMD) and acts by skipping exon 51. Initial data are expected in the third quarter of 2018.

All comments as at December 31, 2017 or beginning of investment.

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Media releases / 19.04.2018

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