Spark Therapeutics Inc (NASDAQ:ONCE) just announced findings from three post-hoc analyses of data from the Phase 3 clinical trial of LUXTURNA (voretigene neparvovec-rzyl) at the American Academy of Ophthalmology Annual Meeting in Chicago. However, the tape has been brutal to any type of positive news in a speculative name, so it isn’t surprising to see this have little impact.

According to the release, “LUXTURNA is a one-time gene therapy for the treatment of patients with vision loss due to inherited retinal dystrophy caused by confirmed biallelic RPE65 mutations and viable retinal cells. One of these analyses will be the subject of the oral presentation, ‘Visual Acuity Outcomes in the Voretigene Neparvovec Phase 3 Trial’ today at 3:51pm CT in room S405 at McCormick Place in Chicago.”

Spark Therapeutics Inc (NASDAQ:ONCE) bills itself as focuses on the development of gene therapy products for patients suffering from debilitating genetic diseases.

Its products include LUXTURNA (voretigene neparvovec), which is in Phase III clinical trial for the treatment of genetic blinding conditions caused by mutations in the RPE65 gene; and SPK-CHM that is in Phase I/II clinical trial for the treatment of choroideremia.

The company’s products also comprise SPK-7001 that is in Phase 1/2 trial for choroideremia; SPK-9001, which is in Phase 1/2 trial for hemophilia B; and SPK-8011 that is in Phase 1/2 trial hemophilia A.

In addition, its product candidates and development programs include SPK-FVIII program to treat hemophilia A; SPK-TPP1 program for the treatment of CLN2 disease; RhoNova for the treatment of rhodopsin-linked autosomal dominant retinitis pigmentosa; and other neurodegenerative diseases.

The company has collaboration agreement with Pfizer, Inc. for the development and commercialization of SPK-FIX product candidates in its gene therapy program for the treatment of hemophilia B. Spark Therapeutics, Inc. was founded in 2013 and is headquartered in Philadelphia, Pennsylvania.

 

Someday it May Even Matter

We started off by noting that ONCE just hit the wires with the announcement of findings from three post-hoc analyses of data from the Phase 3 clinical trial of LUXTURNA (voretigene neparvovec-rzyl) at the American Academy of Ophthalmology Annual Meeting in Chicago.

This news, while obviously helpful, figures into a tape that has been defined by distribution of late, with shares of ONCE taking a hit in recent action, down about -3% over the past week. It should be an interesting battle in terms of how the stock reacts in coming days.

“These data further deepen our understanding of the biology of RPE65 mutation-associated inherited retinal disease and the Phase 3 clinical trial of LUXTURNA,” said Katherine A. High, M.D., president and head of research & development at Spark Therapeutics. “An important component of clinical research is to complete additional analyses on data to gain further insight into the current understanding of the science. This is particularly important in gene therapy, where there is the potential to restore function in the setting of previously untreatable diseases. We look forward to our continued engagement with the ophthalmology clinical and research communities as a leader in gene therapy research.”

Spark Therapeutics Inc (NASDAQ:ONCE) pulled in sales of $25.2M in its last reported quarterly financials, representing top line growth of 1598%. In addition, the company has a strong balance sheet, with cash levels far exceeding current liabilities ($647.2M against $37.4M).

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