Company Articles and Overviews

ProQR Therapeutics: What's Bigger, Its Pipeline Or Its 2019 Outlook?


ProQR has an extensive leadership committee that has demonstrated a track record of success. With several former CEO’s holding executive level positions in the company as well as a long list of medical professionals and researchers in the RNA fields, the company has a positive outlook. The company has recently diluted shares through a public offering, raising over $100 million in funding and giving the company a cash runway that will hold them through into 2021. Having recently been added to the Nasdaq Biotechnology Index and obtaining a new Chief Medical Officer, the company is poised to continue its march higher in market cap.

The company has several upcoming catalysts in 2019. In January, an agreement was reached with the FDA on the design of the pivotal Phase II/III ILLUMINATE trial which is expected to begin in the first half of 2019. In December 2018, the FDA cleared the IND application for QR-421a for Usher syndrome type 2 and non-syndromic retinitis pigmentosa due to mutations in exon 13 of the USH2A gene. The Phase I/II STELLAR trial is expected to initiate in the first half of 2019, with interim data expected in mid-2019. In October 2018, the company obtained exclusive worldwide rights for QR-1123 from Ionis Pharmaceuticals with first in human Phase I/II clinical trial in adRP patients is expected to start in 2019. The WINGS first clinical trial to evaluate the safety and efficacy of QR-313 in patients that have recessive dystrophic epidermolysis bullosa from mutations in exon 73 of the COL7A1 gene, was initiated in mid-2018 and interim data are expected in Q1 2019 with full results in 2019.

Although treating rare diseases, ProQR is excited with the revenue potential from their extensive pipeline. Epidermolysis Bullosa alone affects over 500,000 individuals globally, let alone the other research fields that the company is involved with. With millions of potential patient’s worldwide that have no current viable treatment options that treat the underlying causes of these genetic conditions, the potential to obtain massive market share in these spaces is exciting.

The company skyrocketed from $7 in September 2018 to over $22 in just a few short days. The share price has had fluctuations over the past few months following the public offering, profit taking, and likely skepticism moving forward with so many clinical trials. However, the current share price around $14 looks like a promising opportunity. Great news seems to move the share price for this company like no other. With the fear of dilution taken out of the equation, interim data and the initiation of clinical trials coming in the first half of 2019 and full data results later in 2019, the company has an excellent timeline of potential great news.

While these products have demonstrated their safety and efficacy early on, these early stage studies will likely reinforce that data, providing positive results. I believe that PRQR has great opportunity for success in the next 2 years while they trials are young and they have the cash reserves to burn. While not a short-term play, a long-term entry here will like prove fruitful. I would however be cautious as the trials begin to enter late stage clinical trials and development, despite having orphan status there is not an extensive amount of data available currently and there is a reason that there are no current treatment options that treat the underlying causes of these genetic diseases. They are difficult to work with and this is a newer concept of treatment, leaving concern on the table.

Collin BeloinComment