10 by 2018

Spark unveils vision of having 10 clinical-stage gene therapy programs by 2018, including one commercial and two in pivotal trials

Lloyd Dunlap
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PHILADELPHIA—Spark Therapeutics Inc. has outlined a comprehensive three-year vision for expanding the company’s portfolio through 2018 and launching its first commercial product in 2017, doing so most prominently during a recent presentation at the J.P. Morgan 34th Annual Healthcare Conference in San Francisco.
Spark Co-founder and CEO Jeffrey D. Marrazzo outlined plans for advancing 10 total programs across three therapeutic franchises by 2018, including one commercially approved therapy, two product candidates in pivotal-stage trials and at least seven additional programs in clinical proof-of-concept trials. The company’s 2018 vision for its portfolio is fully funded with existing financial resources exceeding $300 million, Marrazzo said.
“The overwhelmingly positive data from our pivotal Phase 3 program for RPE65-mediated blindness, together with the multiyear durability data presented from the same program, provide strong validation of the Spark platform for AAV-based gene therapy that we are deploying across a large and growing pipeline of product candidates,” Marrazzo said. “Our results reflect the power of a true platform that combines proven capabilities across vector selection, design and manufacture, a history of collaborating with regulators to optimize clinical development and develop novel clinical endpoints and our position at the forefront of shaping a patient-centric, commercial model for gene therapies. We are now leveraging this platform through internal innovation and commercialization, partnering and external collaboration to transform the treatment of a wide range of severe genetic diseases in three target tissues—the eye, the liver and the central nervous system (CNS).”
Among the programs unveiled for the first time were two new indications, including Leber hereditary optic neuropathy, an additional program in inherited retinal dystrophy, which affects over 7,500 patients—with a great number of patients at risk for losing their sight in the United States and the five major European markets (EU5). Within the company’s growing CNS portfolio, Spark unveiled its program targeting Huntington’s disease, a hereditary genetic disorder that affects over 60,000 patients in the United States and the EU5.
The company also outlined multiple data readouts and regulatory milestones that it expects over the next 18 months across five separate product candidates, including:
  • SPK-RPE65: A Biologics License Application filing in the second half of 2016 and a Marketing Authorization Application (MAA) filing in early 2017, along with the readout of one-year efficacy data for the Phase 3 crossover subjects, two- and four-year durability data from the Phase 3 intervention group and a comparable cohort of subjects from an earlier Phase 1 trial, respectively;
  • SPK-CHM: Initial safety and efficacy data in the second half of 2016 from the Phase 1/2 trial for choroideremia;
  • SPK-FIX: Initial efficacy data in mid-2016 for the Phase 1/2 trial for hemophilia B;
  • SPK-FVIII: An Investigational New Drug (IND) filing in the second half of 2016 and initial efficacy data in hemophilia A in the first half of 2017; and
  • SPK-TPP1: An IND filing in the second half of 2016 and initial efficacy data for TPP1 deficiency, a form of Batten disease, in the first half of 2017.
Additionally, Spark announced four recent developments relating to its lead program, SPK-RPE65.
In a press release, the company noted that further analyses of the visual acuity data from the Phase 3 trial yielded statistically significant results: using the Lange scale, intervention group subjects improved an average of nine letters versus 1.6 for the control subjects and, in a sub-group analysis that removes subjects that developed cataracts—as recommended by the company’s data safety monitoring board—intervention group subjects improved an average of 10.6 letters versus 1.6 for controls.
The company received positive scientific advice from the European Medicines Agency (EMA) regarding the requirements for its MAA filing including the feasibility of pursuing an approach to the ultimate label that is similar to the approach being pursued with the U.S. Food and Drug Administration (FDA), combining a core clinical manifestation of the disease with the RPE65 genetic mutation.
Spark also converted the license from the University of Pennsylvania, the University of Florida and Cornell University with claims covering the method of treating RPE65-mediated diseases from co-exclusive to exclusive.
FierceBiotech provided an analyst’s perspective when it opined that “After repeatedly batting back doubts about its lead gene therapy, Spark Therapeutics says its pivotal study for SPK-RPE65, an FDA-designated ‘breakthrough’ for sight-blighting inherited retinal dystrophies, came through with positive data. The biotech says that the treatment hit the primary and two of three secondary endpoints in the late-stage study, setting up a biologics license application at the FDA next year.”

Lloyd Dunlap

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