Shares of the company spiked 120.75 percent on Wednesday on the back of a Phase 1/2 clinical trial of QR-110 for patients with Leber’s congenital amaurosis 10 (LCA10), an eye disease.
The company announced positive interim results on Wednesday (September 5) from the Phase 1/2 clinical trial of QR-110 for patients with Leber’s congenital amaurosis 10 (LCA10), which is an eye disease.
As evidenced in these results, ProQR intends to begin a Phase 2/3 trial in the first half of 2019.
“The results of this interim analysis are encouraging and met our decision criteria to stop enrollment in this study and progress to a pivotal Phase 2/3 trial,” Dr. David Rodman, executive vice president of research and development at ProQR, said in the press release.
In the results, over 60 percent of the patients had overall improved vision, including the sharpness and mobility of their eyesight. Other objectives of the trial that patients met include improving involuntary eye movements with low vision patients and better colored light sensitivity.
QR-110 was found to be tolerated and safe by the 10 dosed patients and no extreme side effects were reported.
LCA10 is the most severe form and frequent of a group of disease from Leber’s congenital amaurosis (LCA). The genetic disease causes blindness in children, within the first few years of life. There is currently no approved treatment or other products in development for the disease.
There are about 2,000 people that have been diagnosed with LCA10 in the Western world.
The treatment is a RNA-based oligonucleotide intended to meet the underlying cause of LCA10 with the genetic mutation. It works by restoring the normal production of CEP290 protein, which is dosed through intravitreal injections in the eye.
QR-110 has been given orphan drug designation in the US and Europe and fast-track designation by the US Food and Drug Administration (FDA).
ProQR has already signed an agreement to submit a protocol to start the new Phase 2/3 trial called Illuminate. The company expects to enroll up to 40 patients with LCA10 and the p.Cys998X mutation.
Objectives of the trial will be similar to the Phase 1/2 but have a greater focus on the visual mobility, sharpness and others now that the safety and tolerability has been assessed in the first trial.
In other news, the company also issued a new public offering of its shares on Wednesday of over US$11 million. According to that release, ProQR intends to use the funds ongoing research and development activities, among other purposes.
The company has a fairly robust pipeline in ophthalmology, dystrophic epidermolysis bullosa, cystic fibrosis and a few other partnered programs. All are developing medicines for RNA therapies. Most programs are in discovery or preclinical development so this trial for QR-110 represents the most advanced clinical trial the company is working on.
As mentioned, ProQR’s share price saw a dramatic increase on Wednesday. Based off two analyst ratings on TipRanks, the company has a “Moderate Buy” ranking with an average price target of US$14.50, a high estimate of US$20 and and a low of US$9.
H.C. Wainwright analyst Andrew Fein reiterated a “Buy” target for the company on Wednesday with a US$20 price target.
Investors can expect to hear more information about the upcoming Illuminate clinical trial close to the end of the year with the trial is expected to begin in the first half of 2019.
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Securities Disclosure: I, Gabrielle Lakusta, hold no direct investment interest in any company mentioned in this article.