ProQR Therapeutics N.V. ( PRQR Quick Quote PRQR - Free Report) recently announced dismal results from its phase II/III Illuminate study on pipeline candidate sepofarsen for the treatment of CEP290-mediated Leber congenital amaurosis 10 (LCA10). Consequently, shares plummeted 75.3%. Sepofarsen (QR-110) is an investigational RNA therapy designed to restore vision in Leber congenital amaurosis 10 due to the c.2991+1655A>G mutation (p.Cys998X) in the CEP290 gene. Leber congenital amaurosis is the most common cause of blindness due to genetic disease in children, and LCA10 is the most frequent and one of the most severe forms. Illuminate, a randomized, sham-controlled trial, enrolled 36 participants aged eight years or older with genetically confirmed LCA10 due to the c.2991+1655A>G (p.Cys998X) mutation in the CEP290 gene, at 14 study sites in nine countries. Participants were randomized to three equal groups (1:1:1) of the target registration dose sepofarsen (160 μg/80 μg loading dose/maintenance doses), a low dose of sepofarsen for masking (80 μg/40 μg loading dose/maintenance doses), or sham procedure, with sepofarsen administered via intravitreal injection and the sham procedure mimicking an injection with no drug or injection given. The study results did not meet its primary endpoint of Best Corrected Visual Acuity (BCVA) at month 12 and no benefit was observed in either treatment arm versus sham. The mean change from baseline in BCVA in the 160/80 μg dose group was -0.11 logMAR, in the 80/40 μg group -0.13 logMAR and in the sham group -0.12 logMAR after a year. There was no difference between the treated and sham groups in the top-line analysis for secondary endpoints full-field stimulus test (FST) and mobility. Shares of ProQR have lost 72.8% in the year compared with the industry’s decline of -40.5%.
Image Source: Zacks Investment Research Investors were disappointed with the failure of the candidate of this clinical-stage company, which has clouded its future at present. The company, however, said that its current cash position provides a runway into mid- to late-2024. On Sep 30, 2021, ProQR held cash and cash equivalents of €156.1 million. Last September, ProQR entered into a licensing agreement with Eli Lilly and Company ( LLY Quick Quote LLY - Free Report) related to ProQR’s proprietary Axiomer RNA base-editing platform. Per the terms of the agreement, ProQR will receive $50 million consisting of an upfront payment of $20 million, as well as an equity investment in its ordinary shares of $30 million. ProQR is eligible to receive up to approximately $1.25 billion in milestones, plus royalties from Eli Lilly. ProQR currently carries a Zacks Rank #4 (Sell). A couple of better-ranked stocks in the biotech sector are Vir Biotechnology ( VIR Quick Quote VIR - Free Report) and Vertex Pharmaceuticals ( VRTX Quick Quote VRTX - Free Report) . While Vir sports a Zacks Rank #1 (Strong Buy), Vertex carries a Zacks Rank #2 (Buy). You can see . the complete list of today’s Zacks #1 Rank stocks here Vir Biotechnology’s earnings per share estimates for 2022 have increased $6.52 in the past 60 days. VIR beat estimates in two of the last four quarters, missing the same on the other two occasions, the average surprise being 12.95%. The consensus estimate for 2022 earnings for Vertex has increased $1.01 over the past 60 days to $14.33. Shares of VRTX gained 16.1% in the past year.