Our Holdings

4.0%

Agios Pharmaceuticals

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Agios Pharmaceuticals

4.0%

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 leukemia (AML). Bristol-Myers/Celgene has worldwide rights to  Idhifa, and Agios will receive milestones and an estimated 15% royalty on sales. Data with IDH1 inhibitor Tibsovo (AG-120) in AML were also promising and the product was approved in July 2018. Results with Tibsovo in a rare solid tumor called cholangio-carcinoma were positive and approval for this indication should be granted by the end of 2020. Meanwhile, development of Tibsovo for low-grade glioma continues. 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 Phase III trials should yield data in 2020.

4.5%

Alexion Pharmaceuticals

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Alexion Pharmaceuticals

4.5%

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 developing a next-generation Soliris, Ultomiris (ALXN-1210), which has an improved dosing profile and is now approved for PNH and aHUS, with additional indications expected to follow. To diversify the revenue base away from Soliris, the company received approval of a novel compound for hypophosphatasia, Asfotase Alfa, in March 2015 and the product has become a meaningful contributor to revenue. In addition, Alexion gained Kanuma for lysosomal acid lipase (LAL) deficiency via its May 2015 acquisition of Syn-ageva. Continuing its aggressive business development efforts, the company acquired Achillion and formed collaborations with Eidos and Stealth in 2019, adding to the 2018 acquisitions of Wilson Therapeutics and Syntimmune.

3.4%

Alnylam Pharmaceuticals

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Alnylam Pharmaceuticals

3.4%

0.7%

Beam Therapeutics

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Beam Therapeutics

0.7%

1.0%

Black Diamond Therapeutics

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Black Diamond Therapeutics

1.0%

0.1%

Cidara Therapeutics

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Cidara Therapeutics

0.1%

3.1%

Crispr Therapeutics

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Crispr Therapeutics

3.1%

2.3%

Esperion Therapeutics

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Esperion Therapeutics

2.3%

4.1%

Fate Therapeutics

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Fate Therapeutics

4.1%

1.5%

Generation Bio

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Generation Bio

1.5%

3.8%

Halozyme Therapeutics

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Halozyme Therapeutics

3.8%

0.4%

Homology Medicines

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Homology Medicines

0.4%

2.5%

Intra-Cellular Therapies

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Intra-Cellular Therapies

2.5%

10.4%

Ionis Pharmaceuticals

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Ionis Pharmaceuticals

10.4%

0.5%

Kezar Life Sciences

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Kezar Life Sciences

0.5%

6.7%

Moderna

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Moderna

6.7%

Moderna is pioneering a new class of medicine made of messenger RNA. Moderna recently gathered a lot of attention through the record-breaking IPO, which raised over USD 600 mn in December 2018. A substantial amount of the USD 3.0 bn of total capital raised since inception in 2011 has been invested in what is now the 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 over 20 development candidates, with 10 of them in the clinic, for mRNA-based vaccines as well as treatments in diverse therapeutic areas. Moderna recently presented key derisking data for their CMV vaccine program as well as for an mRNA encoded chikungunya antibody as a surrogate for rare disease application. In our view, the key programs that will be reading out clinical data within  the mid-term include the rare liver disease MMA and PPA, the proprietary vaccines  in congenital CMV and hMPV+PIV3, the intratumorally injected cytokine cocktail OX40L+IL23+IL36 gamma, the personalized cancer vaccine and early data on the VEGF Phase II during CABG-surgery.

1.3%

Molecular Templates

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Molecular Templates

1.3%

2.9%

Myovant Sciences

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Myovant Sciences

2.9%

1.0%

Nektar Therapeutics

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Nektar Therapeutics

1.0%

6.5%

Neurocrine Biosciences

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Neurocrine Biosciences

6.5%

1.3%

Relay Therapeutics

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Relay Therapeutics

1.3%

3.0%

Sage Therapeutics

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Sage Therapeutics

3.0%

2.5%

Scholar Rock

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Scholar Rock

2.5%

Scholar Rock is a biotech company with a platform based on the understanding of the extracellular activation of growth factors. By targeting the pro and latent forms of  the growth factors with antibodies, not the active/mature factor (given the very high degree of similarity in amino acid sequences in the active sites across the TGF-beta superfamily), the company believes it can avoid the off-target toxicities that have plagued this field historically. Its lead compound is SRK-015, a monoclonal antibody targeting pro myostatin and latent myostatin, is designed to inhibit the activation of myostatin thereby promoting muscle growth and function. The initial indication is later onset spinal muscular atrophy (type 2 and 3) where it can potentially be used in combination with Spinraza and other therapeutics as its mechanism is complimentary, not competitive. Phase II, interim proof-of-concept data were positive with additional data expected in 2020. The platform technology is also focused on TGF-beta 1  in the IO space as well as fibrosis. The fibrosis indications have been partnered with Gilead. The oncology study will begin in 2020.

4.8%

Vertex Pharmaceuticals

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Vertex Pharmaceuticals

4.8%

0.4%

Voyager Therapeutics

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Voyager Therapeutics

0.4%

0.5%

Wave Life Sciences

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Wave Life Sciences

0.5%

All comments as at December 31, 2020 or beginning of investment.

Disclaimer

Any reference to a particular company or security does not constitute a recommendation to buy, sell, hold or directly invest in that company or security.

Latest News

Media releases / 19.02.2021

BB Biotech AG publishes its 2020 annual report