Spark Therapeutics (ONCE), focuses on the development of gene therapy products for patients suffering from debilitating genetic diseases.
ONCE’s products include voretigene neparvovec, which is in Phase III clinical trial for the treatment of genetic blinding conditions called inherited retinal diseases caused by non-sex-linked, autosomal recessive, or biallelic mutations in the RPE65 gene; and SPK-CHM that is in Phase I/II clinical trial for the treatment of choroideremia.
ONCE'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 a form of Batten disease and Huntington's disease; RhoNova for the treatment of rhodopsin-linked autosomal dominant retinitis pigmentosa; and SPK-LHON for treating Leber hereditary optic neuropathy, as well as preclinical programs in development for the treatment of rare genetic blinding conditions, hematologic disorders, and other neurodegenerative diseases.
ONCE 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.
March 16, 2015
RegMed Monday: sluggish sector reflects past year earnings, spending, clinical data and “runways”
March 13, 2015
RegMed Friday: struggling with skepticism
March 12, 2015
RegMed Thursday: a lot of air coming out of trades and pricing
March 12, 2015
Higher open expected; RegMed's continued concerns
March 11, 2015
RegMed fled the fire but, the water damage soaked the sector
March 10, 2015
RegMed Tuesday: the cold reality, another sell-off
March 6, 2015
RegMed’s frenzied trading, as some earning releases “handle” the news
March 6, 2015
RegMed Friday: get your scuba regulators ready
March 4, 2015
Lower open expected; RegMed, the game of poker and terms reminds one of the sector and market
March 3, 2015
RegMed, what are the company specific drivers for the sector?
35 companies, 1 interpreter!
Insight, foresight and recommendation
Spark Therapeutics (ONCE) -- Opened 1/18 at $53.31, slipped to $50.56 (1/5) ending the month at $56.05 and started February at $56.30 and has been slippin, dipping and retesting lows such as 2/20's $50.23 a believer. ONCE has built a fully integrated company dedicated to discovering, developing and delivering one-time treatments that provide long-lasting, transformative outcomes to patients, families, society and the health care system. In 2018, ONCE is focused on launching LUXTURNA in the U.S. and securing marketing authorization in the EU, advancing its global development program for SPK-8011 in hemophilia A and also continuing to progress its pipeline of other investigational gene therapies. Recent: entered into an amendment to the license agreement for SPK-9001 with Pfizer (PFE), in 11/17; including an initial $10 million cash payment and up to an additional $15 million in potential milestone payments upon completion of certain transitional activities in mid-2018 and also ntered into a supply agreement with PFE in February 2018 to begin production this quarter for one batch of drug substance expected to be used for Phase 3 development; Spark received $7 million up front and will receive up to $7 million upon delivery. Dips are always due to market conditions but, with $540.2 M in cash excludes the $105 million received from Novartis in 1/18 and is eligible to receive an additional $25 million upon approval by European Medicines Agency (EMA) and total of $40 million in aggregate additional milestones on initial sales in multiple ex-U.S. markets
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