Onconova (ONTX): Research Note 7 January 

Last week, Onconova announced that it has submitted a Special Protocol Assessment (SPA) request for the FDA for its Phase III study of oral rigosertib combo with azacytidine (Vidaza) for the treatment of adults with treatment-naïve higher risk MDS. Onconova expects its dialogue with the FDA on this SPA submission to conclude in 2019H1. The results of an expanded Phase II trial of oral rigosertib combination therapy with azacitidine were presented at the 2018 ASH Annual Meeting. We have maintained our valuation on Onconova at USD 355 million. In our last update report (September 2018) we increased the potential pricing for rigosertib. The new leukemia drug Vyxeaos from Jazz Pharmaceuticals is priced annually at around USD 150,000. Therefore, we have increased the pricing for rigosertib to USD 100,000 from USD 80,000. The value per share boils down to USD 62.50 per share. 


ProQR Therapeutics (PRQR): Initiating Report 26 April

ProQR Therapeutics is an innovative biopharma company that is developing RNA-based therapeutics for the treatment of severe genetic disorders such as cystic fibrosis, Leber’s congenital amaurosis Type 10 and dystrophic epidermolysis bullosa. The company’s growing pipeline is based on its proprietary technology platform of RNA technologies. Its lead program is eluforsen (formerly QR-010), which is currently in Phase IB for cystic fibrosis. Eluforsen is expected to advance into a phase II study in 2018. We believe the company will aim for a partnership with big pharma in order to focus on its programs in retinal dystrophy. QR-110 is a therapy designed to address the underlying cause of Leber’s congenital amaurosis (LCA) 10. A Phase 1b/2 trial for QR-110 was initiated in 2017Q4, and we expect initial results in 2018. Using our valuation model and taking into account a potential partnership with eluforsen and future revenues of QR-110, the company’s current total value should be USD 350-400 million, or USD 11.00-12.50 per share. 


Pharming (PHARM.AS): Update Report 8 October

Pharming Group is a Dutch based biopharmaceutical company and one of the first publicly traded biotech companies in Europe. In the next months, we expect that the company will initiate new clinical programs to expand the use of RUCONEST® in new indications (like contrast induced nephropathy) as well as start clinical trials new programs like alpha-glucosidase for Pompe Disease. Each of these programs address markets that dwarf the HAE market size. For HAE, focus will lie on the development of better and more convenient administration options of RUCONEST®. We believe that is the key growth driver for the HAE market in the coming years. We have increased our valuation for Pharming based on a further increase of profits and revenues from Ruconest, from EUR 1.6 billion to EUR 2.0 billion. This translates based on the fully diluted number of 657 million shares into EUR 3.11 per share.   

FINANCIAL INSTITUTE IN LIFE SCIENCES 

antonie van leeuwenhoek

(delft, netherlands 1632-1723):

founding father of modern biotechnology


We are proud our firm is named after one of the founding fathers of modern biotechnology: Antonie van Leeuwenhoek. Antonie van Leeuwenhoek was a Dutch scientist that lived in the 17th century: 'The Dutch Golden Age", a period in Dutch history, roughly spanning the 17th century, in which Dutch trade, science, military, and art were among the most acclaimed in the world. He was a Dutch tradesman and scientist and is commonly known as "the Father of Microbiology", and considered to be the first microbiologist.


He is best known for his work on the improvement of the microscope and for his contributions towards the establishment of microbiology. Using his handcrafted microscopes, he was the first to observe and describe single-celled organisms, which he originally referred to as animalculs,and which are now referred to as micro organisms. He was also the first to record microscopic observations of muscle fibers, bacteria, spermatozoa, and blood flow in capillaries (small blood vessels). Leeuwenhoek did not author any books; his discoveries came to light through correspondence with the Royal Society, which published his letters.




 


an unbiased and independent view on companies in the global life sciences sector


Van Leeuwenhoeck Institute (VLI) provides institutional investors and other professional investors with independent, un-biased research on the real value of innovative Life Sciences companies. Many institutional and individual investors will not invest in companies lacking independent third-party research coverage, yet few investment banks cover this sector because it requires dedicated expertise.


In spite of the lack of quality knowledge on an industry-wide basis, there is increased demand for life science-specific market research as investors are seeking information about leading companies in the sector in order to make well-informed investment decisions.

 


Resverlogix (RVX.TO): Update Report 24 July

In the past 6 months, Resverlogix has made important steps towards finalizing its pivotal Phase III BETonMACE trial with its leads product apabetalone in high risk CVD patients with Type 2 diabetes and low HDL. This trial started in November 2015 and exceeded full enrollment of 2,400 patients in March this year. In June, the FDA confirmed that if the Phase III BETonMACE study is successful, it will probably be enough to support the filing and approval of an NDA. The FDA’s feedback is similar to that received previously from European authorities. We have increased our valuation following very encouraging news from the FDA which encouraged us to lower the discount rate and increase the LOA for apabetalone in high risk diabetes and CKD patients. We feel that the company’s current total value should be CAD 3.3 billion, or CAD 19.00 per share.


Addex Therapeutics (ADXN.SW): Report 22 November 

Addex Therapeutics is a Swiss based biopharmaceutical company that is developing innovative oral therapies with a focus on neurological disorders. Addex’ lead product is dipraglurant that successfully completed a phase IIa POC trial in Parkinson’s disease levodopa induced dyskinesia (PD-LID). The drug is scheduled to start a Phase IIb/III pivotal registration study in PD-LID in 2019H2.  The Company’s current cash position has improved dramatically and amounts to CHF 44 million following a successful raise of CHF 40 million and the first payment from its partner Indivior. Based on our NPV based valuation, we believe that Addex is substantially undervalued at the current share price of CHF 2.36. Using our valuation model and taking into account the future revenues from its late stage
clinical pipeline the company’s current total value should be CHF 350-400 million, or CHF 12.25-14.00 per share. Furthermore, recent M&A activity in the Parkinson’s disease field indicates that industry could justify a valuation above CHF500 million.