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

Summary

  • PRQR boasts a pipeline of 18 drug candidates, nearly all in early-stage development.

  • Cash runway to last through to 2021 following recent public offering.

  • First half of 2019 catalysts: Phase II/III ILLUMINATE trial, Phase I/II STELLAR trial with interim results mid 2019, Phase I/II adRP clinical trial, WINGS interim data.

  • Share price jumps off positive news. With a pipeline of catalysts coming up in the next 3 months,du share price is poised to take off.

ProQR Therapeutics (PRQR) is one of the many companies focusing its efforts on developing treatments and new medicines for rare disease that are in dire need of therapeutic options through developing RNA therapies to address genetic diseases. ProQR has a unique story where the company was founded in 2012 by CEO Daniel de Boer who was struck with the diagnosis that his newborn son had cystic fibrosis. Fueled by the desire to not only help his own family, but other patients in need of serious treatment, the genesis of ProQR came about with the help of co-founders Dinko Valerio, Gerard Platenburg, and Henri Termeer.

With an experienced executive and leadership team, this company shows great promise for long-term success. Boer has a history of entrepreneurship, having been founder and CEO of RNA Systems, PC Basic and Running IT while also being a co-founder of Amylon Therapeutics, leaving him no stranger to the biotechnology industry. Having a team around him that consisted of the Alison Lawton and his three co-founders, all of which had experience as CEO’s of their own pharmaceutical and biotechnology companies. Backing this extensive leadership team is an extensive team on the scientific advisory board of prestigious PhD’s and MD’s.

Pipeline Overview

ProQR has a large umbrella with many different RNA therapy treatments being developed, a majority still in their infancy under the Discovery and Preclinical Development phases. This umbrella encompasses Ophthalmology with 8 different candidates, Dystrophic Epidermolysis Bullosa with 3 candidates, Cystic Fibrosis with 4 candidates, as well as 3 other drug candidates that the company partially owns in conjunction with partners. 18 different drug candidates is quite an extensive list, even with the research compacted into 3 diseases. It appears the company may be throwing as many darts at the board as they can and hoping a few stick, but is that truly the case?

Recently announced, the company proposed its “ProQR Vision 2023” strategy, focusing on the commercialization of its drug candidates. The company expects that by 2023, it will have a minimum of two commercial products, at least three late-stage development candidates, and seven early-stage programs in the pipeline. This will all run in conjunction with expanding their RNA platform capabilities to expand into additional therapeutic areas as well as branch away from strictly rare diseases. According to ProQR, the company has several key deliverables for this vision:

  • Sepofarsen (formerly QR-110) for Leber's congenital amaurosis 10 (LCA10): Complete pivotal program around year-end 2020 for submission of a New Drug Application (NDA) in the U.S. and a Marketing Authorization Application (MAA) in Europe in 2021

  • QR-421a for Usher syndrome Exon 13: Start the Phase 1/2 STELLAR proof-of-concept clinical trial, with interim data in mid-2019 and initiate an adaptive multiple-dose trial with projected readout in 2021

  • QR-1123 for P23H autosomal dominant retinitis pigmentosa (adRP): Initiate a Phase 1/2 proof-of-concept clinical trial in 2019, with data expected in 2020

  • QR-411a for Usher syndrome PE40: Conduct Investigational New Drug (IND)-enabling work in 2019 to start a proof-of-concept clinical trial in 2020

  • QR-504 for Fuchs endothelial corneal dystrophy (FECD): Complete IND-enabling activities in 2019 to start a proof-of-concept clinical trial in 2020

  • Accelerate discovery: Expand efforts to generate additional programs for inherited retinal diseases amenable to RNA oligo treatment with the goal of establishing at least seven new programs for development

  • Selectively explore and expand platform: Expand platform to develop medicines in new therapeutic areas and create opportunities to build businesses in other therapeutic areas

Ophthalmology

For those who don’t know, Ophthalmology is the field of medicine and therapy that deals with the eye. Mentioned previously, ProQR is developing 8 different drug candidates that revolve around this space, notably for the treatment of Usher Syndrome, Stargardt’s Disease, Leber’s Congenital Amaurosis 10, Autosomal Dominant Retinitis Pigmentosa, and Fuchs Endothelial Corneal Dystrophy. With nearly all candidates in Discovery, Preclinical Development, or just entering Proof of Concept Trials, ProQR does have one late stage development candidate Sepofarsen (QR-110) which it is expected to start its Phase II/III Pivotal Trial in the first half of 2019.

Sepofarsen (QR-110) – Is a drug candidate that is being developed for the treatment of Leber’s Congenital Amaurosis 10 (LCA10) in patients who carry the p.Cys998X mutation in the CEP290 gene. LCA is the most common genetic cause of childhood blindness due to disruption in photo transduction, affecting roughly 15,000 patients in North America and Europe, with 2,000 of those patients having LCA10, the most severe form of LCA. Currently, there are no available options to treat the underlying genetic mutation for the disease. QR-110 is being developed to repair the patient’s RNA defect in order to properly produce a healthy CEP290 protein and effectively halt progression of the disease. Designated an “Orphan Drug” by the U.S. FDA and European Medicines Agency as well as Fast Track designation from the U.S. FDA, the governing bodies recognize the dire need for treatment options. Currently in an ongoing Phase I/II clinical trial, QR-101 has released interim results in September 2018 suggesting that the compound is safe and well tolerated while also producing substantive overall improvement in vision in a majority of patients after 3 months of treatment. We will see side effects and overall safety towards the end of this study. They are also planning to start “ILLUMINATE”, a Phase II/III study on patients with this disease aged 6 years and older in 2019 with the plan to also conduct trials on children younger than 6 years old.

QRX-136 - A drug candidate with little information out for the treatment of an undisclosed LCA mutation. More information hopefully coming out in 2019.

QR-421a –In the early stages of Phase I/II in an ongoing study throughout North America and select countries in Europe, QR-421a is an investigational drug being developed to treat retinitis pigmentosa (RP) in patients that have Usher syndrome type 2 (USH2), a genetic disease that leads to a combination of early onset deafness at birth and later progressive blindness moving forward starting in their 20’s. USH2 is the most common form of Usher syndrome and caused by a mutation on the USH2A gene responsible for forming usherin proteins. The resulting non-functional usherin protein disrupts phototransduction within the rods and cones in the retina, leading to onset RP. QR-421a targets exon 13 mutation specifically, which occurs in roughly 16,000 patients in North America and Europe. QR-421a utilizes a process called “exon skipping”, where in the drug is designed to prevent exon 13 from being utilized from USH2A mRNA, effectively taking the mutations effects out of play. While the resulting protein is smaller and shortened, it is still effective in its functionality. Unfortunately for patients that are seeking this treatment, the administration is done through intravitreal injections (they inject through your eye). Currently, the phase I/II clinical trial named STELLAR is being conducted on 18 patients that have experienced vision loss due to this genetic disease with interim results expected to be released mid-2019. While there is no current treatment for blindness associated with this disease, the deafness can be managed through hearing aids or cochlear implants.

QR-411 – Currently in the preclinical development phase, QR-411 is being developed to treat the vision loss associated Usher syndrome caused by c.7595-2144A>G (PE40) mutation on the USH2A gene. With a small market of roughly 1,000 patients that have this mutation, it is quite a bit smaller but no less of an opportunity for the company to capitalize on the market. Having the orphan drug designation, strong preclinical proof of concept in patient retinal organoids, and a development candidate selected, the company is expecting to have IND-enabling studies starting in 2019. With the work being done on the other trials, it is like to come later in the year. It is also important to note the clinical development of this compound is very similar to QR-421a, which could indicate the process moving forward can be expedited due to familiarity with development.

QRX-461 – This compound is being developed for Usher syndrome on an undisclosed mutation at this time. Hopefully more information to come in the first half of 2019.

QR-1011 – Targeting Stargardt’s disease, a genetic eye disease leading to progressive central vision loss, and more specifically the mutation c.54461-10T>C in the ABCA4 gene ProQR hopes to be the first available therapy for Stargardt’s to help treat the current 7,000 patients in N.A. and Europe. The blindness from this disease begins in childhood and develops into full blindness by mid adulthood. The goal of the company is to repair the mutation, leading to normal ABCA4 protein development which can potentially stop and even reverse progression of Stargardt’s in patients.

QR-504 -Fuchs endothelial corneal dystrophy (FECD) is a genetically inherited disease that can lead to complete blindness and require corneal transplants for correction. QR-504 is targeting a mutation in the TCF4 gene to prevent corneal dystrophy to halt the disease from progression. With over 250,000 patients in N.A. and Europe that have this genetic condition which causes blindness in elderly patients most commonly and has no current therapy, the market is ripe. QR-504 promises rapid delivery to corneal cells and has displayed strong preclinical proof of concept in human cell models.

QR-1123 – Holding exclusive worldwide licensing from Ionis Pharma for the treatment of autosomal dominate retinitis pigmentosa (adRP), ProQR is developing QR-1123 to treat adRP due to the P23H mutation in the rhodopsin (RHO) gene. The goal of this therapy is to block the formation of mutated RHO proteins by binding to RHO mRNA, causing a knock-down of the mRNA which, in theory, should prevent the loss of light detecting cells in the retina and prevent further vision loss. Holding the exclusive license announced in October 2018, the company paid big for the rights and is extremely hopeful for this therapy. ProQR made an upfront equity payment of $2.5 million at a 20% premium of the share price, will make continuing milestone payments to Ionis Pharma, as well as royalties on 20% of net sales in the future of this product. Success in this program would not only lead to a high revenue generating product, but also transition into targeting other autosomal dominant eye diseases. With an accelerated clinical development program and Phase I/II trial expected to start in 20119 after strong preclinical proof of concept in vivo and a majority of the IND enabling activities completed, ProQR is excited about this drug candidate’s potential.

Cystic Fibrosis

Hitting close to home, the CEO of ProQR is all too familiar with Cystic Fibrosis (CF) after his son was diagnoses shortly after birth. CF is a genetic disease affecting more than 75,000 people worldwide where the affected have viscous mucus accumulating in their organs, clogging tubes and organs and essentially shutting down vital processes within the body. One of the most common issues is the mucus accumulating in the lungs, creating an ideal environment for bacteria which can’t be cleared from the lungs from coughing, leading to inflammation and regular infection. Lung failure is the most common fatal result of this disease, with patients lasting an average of 30 years and despite the wide treatment market available, there are limited options on treating the underlying genetic mutations and create a void of unmet medical needs.

QR-010 – Also known as eluforsen, this compound is being developed to target the F508del mutation, the most common mutation in CF patients, affecting more than 85% of people with the genetic disease. Being developed as a regular inhaled therapy, eluforsen binds specifically to defective CFTR RNA to restore functionality to the CFTR protein, treating the underlying cause of CF. With two clinical trials completed by ProQR, eluforsen was observed to be safe and well tolerated for individuals displaying the F508del mutation. Currently planning a Phase II trial, there is no disclosed timeline for the program. The company will likely need to raise funds to start this trial despite recently doing an offering back in September 2018.

QRX-042 for W1282X / QRX-036 for G542X / QRX-052 FOR R553X – These three potential drug candidates are currently in the discovery phase of the development process for the treatment of Class 1 mutations. Each compound is targeting a different genetic mutation which causes CF, and based on the success of eluforsen QR-010, these three candidates can follow its outcome through their clinical trials.

Dystrophic Epidermolysis Bullosa

Epidermolysis Bullosa (EB) is a genetic skin disease and Dystrophic Epidermolysis Bullosa (DEB) is the most severe form of the condition. DEB causes blistering of the skin and mucosal membranes that line structures in the body such as the mouth and esophagus. Resulting in fragile skin, there is a week connection between the dermis and epidermis of the skin, causing constant pain. DEB is the result of mutation in the COL7A1 gene responsible for forming collagen type VII protein forming anchoring fibrils that bind the two layers of skin together. Like a majority of the genetic diseases that ProQR works with, there is no treatment for the underlying cause of DEB, only palliative treatments for wound care and pain management.

QR-313 – Being developed to treat the specific mutation at exon 73 on the COL7A1 gene, the treatment is designed to exclude the exon from COL7A1 RNA, producing exon skipping and generating a shortened C7 protein that brings functionality, anchoring fibrils, and stronger skin to patients. The is a topical hydrogel treatment that is to be applied to wounds. There are approximately 2,000 patients in the U.S. and Europe with this mutation, and with the Phase I/II trial being initiated there will be 8 patients enrolled in the study. Interim analysis data is expected in early 2019. We will focus more on this shortly as it is the nearest catalyst for the company.

QRX-323 for DEB Exon 80 – Working in conjunction with QR-313, will provide proof of concept to develop into preclinical trials and begin further research on exon skipping for DEB patients.

QRX-333 for DEB Exon 3 - Working in conjunction with QR-313, will provide proof of concept to develop into preclinical trials and begin further research on exon skipping for DEB patients.

Partially Owned Programs

In conjunction with the current trials and research the company is taking on through their own R&D, the company is also partnering with outside companies Galapagos and Amylon Therapeutics to develop additional novel therapies for rare and currently non-treatable diseases such as Fibrosis and CNS diseases.

Upcoming Data and Focus

QR-313 is the short term catalyst amongst many that ProQR has in their pipeline. The company is expected to present Phase I/II interim data in the first quarter of 2019. With March newly here, the company is approaching the timeline that they set out.

QR-313 is one of their flagship drug candidates that is an RNA-based oligonucleotide designed to address the underlying cause of dystrophic epidermolysis bullosa (DEB) due to mutations in exon 73 of the COL7A1 gene. Currently, the clinical development of QR-313 is supported with funding from EB Research Partnership and EB medical Research Foundation. QR-313 has been granted orphan drug designation in the United States and the European Union, from which ProQR received $5 million in funding to further their research and clinical development.

DEB leads to a limited life expectancy and blistering of the skin starting at birth.QR-313 as a topical application not only provides the hope that there is a viable treatment option for patients, but also would allow an easy and convenient application that can be done at home without physician supervision.

This compound is currently in the Phase I/II “safety and efficacy study” which the company dubbed WINGS. The name of the study derives from the idea that children with DEB have very fragile skin comparable to that of a butterfly’s wings. WINGS is an ongoing international Phase 1/2, double-blind, randomized, intra-subject placebo controlled clinical trial of QR-313 in patients with (recessive) RDEB due to mutations in exon 73 of the COL7A1 gene. 8 patients will be included in WINGS and during the study, QR-313 or placebo formulated in a gel, will be topically applied to a patient’s wounds two to three times a week for up to four weeks with an eight-week follow-up period. The Company’s primary objectives of WINGS are to test whether QR-313 is safe and tolerated (safety and efficacy study) and to evaluate proof of mechanism by testing, at a molecular level, whether QR-313 actually induces the exon skipping of exon 73 from COL7A1 mRNA and will be assessed by polymerase chain reaction. Secondary objectives are to test the effects on wound healing, skin strength, the presence of collagen type VII protein and anchoring fibrils in the skin as well as systemic distribution of QR-313 after topical administration. Following strong preclinical proof of concept, topical formulation, FDA and EMA orphan drug designation, the company is initiating the clinical trials with full results expected from Phase I/II later in 2019 and interim results in Q1 2019.

After initial screening in QR-313 in vitro evolutional of the compound, a single stranded fully phosphorothioated and 2’O-methylated AON, composed of 21 bases was selected to the target sequence which is optimized for length, sequence, lack of immunogenicity and optimal manufacturability. The company utilized the study to assess exon skip efficacy in multiple cell types, especially when administered through topical gel formulation.

In vitro transfection of cell cultures Exon skip efficacy of QR-313 was assessed in human primary fibroblasts (HPF), recessive dystrophic epidermolysis bullosa (RDEB) fibroblasts with a mutation in exon 73, and mutated HaCats. Cells were transfected with 3.12- 200 nM QR-313 with polyethylenimine (maxPEI) and subsequently the RNA was isolated and cDNA produced and visually analyzed to estimate skip efficiency through PCR and Bioanalyzers.

Immunofluorescent staining of C7 WT fibroblasts or RDEB fibroblasts were plated and subsequently transfected with QR-313 as described above. 72 hours after transfection, cells were fixed with 4% formaldehyde, blocked and stained with a polyclonal Anti-Collagen VII antibody.

Exon skip measurements in human skin equivalents (HSEs) composed of both a dermal layer containing fibroblasts in a collagen matrix and an epidermal layer containing keratinocytes were created. The HSEs were cultured on filters at the air-liquid interface for 14 days to induce differentiation of the epidermis and form a stratum corneum. Subsequently, QR-313 formulated in a hydrogel was applied onto the wounded area.

The results of this in vitro study produced in the first 24 hours with 25 nM of QR-313 a median skip efficacy measured by PCR of 74% in HPF and 86% in PF, this exon exclusion efficacy is further elevated to a median exon skip of 82% and 87% respectively when the cells were treated with higher concentrations of QR313. After 48 hours the exon 73 exclusion efficacy is further elevated to 100% for all tested concentrations in PF compared to 84% exon exclusion in HPFs treated with 200 nM QR-313.

Using CRIPR, a cell line was created by introducing a homozygous single G deletion in exon 73 of COL7A1, this HaCat cell-line and was assessed for the exon skip efficacy of QR-313 24 hours after transfection at a dose range of 3,125-100 nM QR-313. Results demonstrate that QR-313 efficiently excluded exon 73 from the COL7A1 mRNA cell lines, with skip efficiencies of >50% when the cells were transfected with 25 nM or higher concentrations of QR-313.

The treated cells also showed normal signs of expression of C7 protein when transfected with QR-313, effectively proving the same functionality as healthy HPF.

To assess the efficacy of QR-313 when applied onto HSEs, part of the epidermis was removed to mimic wounded skin before QR-313 was topically. Either 10, 100 or 1000 µg of QR-313 in 200 mg of hydrogel was applied to a wound of 0.8 cm by 2.2 cm. 24 or 48 hours after application the dermis and epidermis were analyzed separately for exon 73 skip. It was observed that with the highest dose QR-313 after 48 hours of incubation all HSEs demonstrated exon skip after 48 hours.

The Company concluded that QR-313 is very efficient in the exclusion of exon 73 from the COL7A1 mRNA in fibroblasts of a patient with a mutation in exon 73. In addition, transfection with QR-313 leads to an increase in C7 protein expression in the patient fibroblasts. Moreover, hydrogel-formulated QR-313 is active in inducing exon 73 skip in COL7A1 in wounded HSEs, which mimics the situation in patient skin. The company also has research which demonstrates that Cy5-labeled QR-313 delivery in vitro, ex vivo, and in vivo models are successful in wounded skin treatment but not with intact skin. This makes the delivery method of the hydrogel formulation being pursued for QR-313 a viable option for wound treatment in patients.

The Phase I/II trial in DEB patients will seek several endpoints. The primary endpoint being safety, tolerability, and the demonstration of exon skipping in the group receiving the topical treatment. Secondary endpoints will be C7 protein detection, anchoring fibrils being formulated, wound healing, and length of time it takes for patients to re-blister. The interim data will provide a proof of concept that exon skipping, C7 protein detection, and anchoring fibrils are all being demonstrated in patients when reported in Q1 2019. Full data on all patients will be available later in 2019. There is a potential for extension of the study in the same patients to test various dosing regimens and administration routes.

There are approximately 2,000 patients in the U.S. and Europe that have the genetic mutation on the COL7A1 gene on exon 73, making the market rather small. But there is a market as demonstrated by the $5 million in funding by the EB organizations.

Recent Events

In December, following the recent success of the company, ProQR Therapeutics was added to the NASDAQ Biotechnology Index (NASDAQ: NBI).

Financials

The end of 2018 showed that ProQR held cash and cash equivalents of €105.6 million, up more than double what the company held compared to the end of Q4 in 2017. This influx of cash was driven by the recent public offering in September which netted €84.2 million. In the fourth quarter, the company’s operating expenses came to €11.3 million and reached €30.7 for the year in total, with research and development consisting of a majority of the expenses. The company posted a net loss of €13.0 million or €0.33 per diluted share for the fourth quarter and a cumulative €37.1 million or €1.09 per diluted share for the year.

The company has a fairly substantial burn rate. With clinical trials moving forward and potential positive interim data for the QR-313 study as well as other studies they have planned data for in 2019, additional trials are going to be conducted, increasing costs. Comparing the operating costs of 2017 and 2018, they were roughly identical with only a minor difference between them, actually showing operating costs decreasing year over year. I believe that it is safe to assume that for 2019, operating costs will continue to stay at around €30 million for the time being, leaving the company a bit of buffer room for growing operations, initiating new trials, and maintaining investor growth without fear of dilution on the horizon.

Even with increasing expenses and additional trials being initiated, the company appears to have more than two years of cash and cash equivalents on hand to hold them over without the need to raise additional funding. The company states that it now has a cash runway to lead into 2021.

Epidermolysis Bullosa Market

Epidermolysis Bullosa (EB) is both a clinical and genetic group of inherited blisters which affects over 500,000 individuals globally, with 32% of the EB treatment market stemming from the United States according to Technavio. Due to the genetically heterogeneous nature of this disease, the most promising treatment option lies within gene therapy and treating the underlying genetic mutations causing EB. Low prevalence, lack of industry research and exposure, and no current viable treatment options exist for EB causing this disease to be deemed non-curable and can become a cause of cancer.

However, as demand and exposure continue to escalate for this disease, we are seeing an influx of funding into research from the US, Europe, and Asia with the largest push coming from Europe despite Asia having the largest potential market. While Asia will have higher incremental growth than both the US and Europe in this field, ProQR has received the orphan drug designationfrom the U.S. FDA and European Medicines Agency making these two markets the major focus for the company. The US is expected to dominate the market compared globally but will start to lose market share overall as EU and Asia begin larger pushes into the industry.

The global EB market currently sits just north of $1.35 billion in 2018 and is projected to grow at a compound annual growth rate (OTCPK:CAGR) of roughly 5% to approximately $1.65 billion by 2022 according to Technavio.

The steady growth of the market is driven by the increasing funds poured into the research industry regarding this disease as current treatment options have severe side effects, do little to treat the underlying issues, and are inadequately effective. Currently, antibiotics account for roughly 43% of the market for therapeutic treatments of EB. With further development into gene therapy, this treatment option is expected to take over the EB market as the dominating therapy. Gene therapy has become of the powerhouses in the biotechnology field, and is projected by many to begin replacing therapeutic options that are currently commonplace.

At present, a few gene therapies are approved globally, these therapies have proven to be highly effective for the treatment of various indications. This resulted in several companies conducting clinical trials using gene therapy for the treatment of epidermolysis bullosa. Majority of these studies are still in the clinical trial phase. [A senior analyst at Technavio]

Outside of antibiotics, the segment that will account for the highest market share in the EB therapeutics market due to the recent approvals of broad-spectrum combinational antibiotics for the treatment of hospital infections that are easily developed in patients with epidermolysis bullosa, and gene therapy, analgesics are a common therapy option for EB patients suffering from severe pain. While not directly treating the disease, analgesics improve quality of life for patients but will likely phase out as viable long-term treatment options become approved.

Competition

There is an extensive list of competition in various stages of development in the Ophthalmology, Dystrophic Epidermolysis Bullosa, and Cystic Fibrosis markets. While many are working on similar conditions, no company is attacking the RNA field in the way that ProQR is. ProQR is focusing on the underlying cause of these genetic mutations and conditions, rather than treating the condition on the surface.

Some companies that are in the DEB Market there are:

Review

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.