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Spark Therapeutics (ONCE) Unveils Encouraging Hemophilia A Data



8/2/2017 1:22:03 PM

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Spark Therapeutics Reports Second Quarter 2017 Financial Results and Recent Business Progress

Preliminary interim data for SPK-8011 Phase 1/2 dose-escalation clinical trial in hemophilia A show initial human proof-of-concept in three participants

In the two participants at the initial dose, stable factor VIII levels average greater than 12 percent with no spontaneous bleeds

Investigational LUXTURNATM (voretigene neparvovec) Prescription Drug User Fee Act (PDUFA) date set for
Jan. 12, 2018; granted rare pediatric disease designation by FDA

PHILADELPHIA, Aug. 02, 2017 (GLOBE NEWSWIRE) -- Spark Therapeutics (NASDAQ:ONCE), a fully integrated gene therapy company dedicated to challenging the inevitability of genetic disease, announced today financial results for the second quarter of 2017 and recent business progress.

“This has been an unprecedented time at Spark Therapeutics, marked by the achievement of significant regulatory milestones for investigational LUXTURNA™ (voretigene neparvovec). The U.S. Food and Drug Administration (FDA) has accepted for filing the Biologics License Application (BLA) for LUXTURNA, which is the first BLA for a gene therapy for an inherited disease, and we have submitted the marketing authorization application (MAA) to the European Medicines Agency (EMA). Additionally, LUXTURNA has been designated by FDA as a drug for a rare pediatric disease, which will qualify us for a Priority Review Voucher, if LUXTURNA is approved,” said Jeffrey D. Marrazzo, chief executive officer of Spark Therapeutics. “These critical steps all get us closer to potentially bringing the first treatment to patients with vision loss due to confirmed biallelic RPE65-mediated inherited retinal disease (IRD). A natural history study has shown that people with this IRD eventually progress to complete blindness.”

The company also disclosed preliminary initial data from the Phase 1/2 dose-escalation clinical trial for SPK-8011, a novel bio-engineered adeno-associated viral (AAV) vector utilizing the Spark200 capsid and containing a codon-optimized human factor VIII gene, being investigated as a potential one-time therapy for hemophilia A. It is the second investigational hemophilia gene therapy to emerge from Spark Therapeutics’ leading gene therapy platform. Spark Therapeutics is developing SPK-8011 independently and retains global commercialization rights to the SPK-FVIII program.

“The encouraging start of our SPK-8011 clinical trial reinforces the strength of our gene therapy platform, delivers human proof-of-concept in a second liver-mediated disease -- a significant achievement in the gene therapy field -- and positions us well to potentially transform the current treatment approach for this life-altering disease with a one-time intervention,” said Katherine A. High, president and chief scientific officer of Spark Therapeutics. “We are excited about the progress we are making to achieve our goals of our investigational hemophilia A and B programs: to safely achieve predictable, consistent and sustained activity levels that prevent spontaneous bleeding.”

With three participants infused in the hemophilia A dose-escalation clinical trial, there have been no serious adverse events (SAEs) reported to date, including no factor VIII inhibitors and no thrombotic events. As of the Aug. 1, 2017 data cutoff, the first two participants, who have received a single administration of SPK-8011 at an initial dose of 5 x 1011 vector genomes (vg)/kg body weight, have now been followed for 23 weeks and 12 weeks, respectively. During this time, there has been a steady and consistent rise in factor VIII activity levels that have now stabilized at levels of 11 percent and 14 percent of normal, respectively.

Based on these data, the company elected to double the dose and recently infused a third participant at a dose of 1 x 1012 vector genomes (vg)/kg body weight. While the results for this third participant are early, his factor activity level is tracking proportionally higher, consistent with the dose escalation.

At this point in the trial, there have been no immune responses and none of the participants has required use of corticosteroids. There have been no reported spontaneous bleeds.

Additional highlights

Advanced investigational LUXTURNA for the treatment of biallelic RPE65-mediated IRD:

  • Progressed regulatory submissions in the U.S. and EU
    - BLA accepted with target PDUFA date set for Jan. 12, 2018
    - Received rare pediatric disease designation for vision loss due to confirmed biallelic RPE65 mutation-associated retinal dystrophy from FDA
    - Submitted MAA to EMA on July 29, 2017
  • Published Phase 3 trial clinical trial data for LUXTURNA in The Lancet, including the intent-to-treat population of all randomized participants, through the one-year timepoint

Reported updated data from investigational SPK-9001 for hemophilia B Phase 1/2 clinical trial at International Society on Thrombosis and Haemostasis (ISTH) 2017 Congress:

  • No SAEs have been reported to date, including no factor IX inhibitors and no thrombotic events
  • Since vector administration, as of the June 5, 2017 data cutoff, among the 10 participants, the annualized infusion rate (AIR) had been reduced by 99 percent and the annual bleeding rate (ABR) was reduced by 96 percent
    - Mean steady-state factor IX activity level post-12 weeks treatment for 10 participants was a consistent and sustained 33 percent
  • Data represent approximately 10 cumulative patient years of SPK-9001 exposure from the start of the trial, with one participant out approximately 18 months post-infusion and four additional participants at least one year post-infusion
    - Only one participant, with severe joint disease, has self-administered precautionary infusions for persistent knee pain
  • Two participants who had experienced an asymptomatic, transient elevation in liver enzymes or a decline in factor IX activity have continued to demonstrate stable factor IX activity levels, 18 and 12 weeks’ post-cessation of steroids, respectively.
    - Neither of these participants has experienced a bleed nor taken factor concentrates.

Financial results for the quarter ended June 30, 2017

Three Months Ended June 30, 2017 and 2016

In the three months ended June 30, 2017 and 2016, we recognized $1.5 million and $1.3 million, respectively, of revenue associated with our Pfizer collaboration.

Our research and development expenses for the three months ended June 30, 2017 were $33.0 million versus $19.6 million for the three months ended June 30, 2016. The $13.4 million increase was due to a $9.8 million increase in internal research and development expenses, primarily due to increased headcount, and an increase of $3.6 million in external research and development. The increase in external research and development was primarily from an increase of $1.7 million in expenses related to LUXTURNA, $1.2 million in our other programs in preclinical development and $0.7 million in our other clinical programs.

Our acquired in-process research and development expense for the three months ended June 30, 2017 was $3.1 million versus zero for the three months ended June 30, 2016. This amount represents a payment related to our license agreement with Selecta Biosciences, Inc., or Selecta.

During the three months ended June 30, 2017, we recorded a non-cash impairment charge of $15.7 million related to acquired in-process research and development, or IPR&D, attained in March 2016. Additionally, we recognized an income tax benefit of $1.0 million related to the reversal of the deferred tax liability associated with the IPR&D during the three months ended June 30, 2017.

General and administrative expenses for the three months ended June 30, 2017 were $26.7 million versus $10.7 million for the three months ended June 30, 2016. General and administrative expenses consisted primarily of salaries and related costs, including stock-based compensation, legal and patent costs, professional fees and other operating costs. The $16.1 million increase primarily was due to an increase of $7.2 million in salaries and related costs, including stock-based compensation, as a result of increased headcount, an increase of $4.0 million in launch preparation activities for LUXTURNA and $4.9 million in legal and patent costs, professional fees and other operating costs.

During the three months ended June 30, 2017, we recorded an income tax benefit of $1.1 million related to our available for sale securities.

Our net loss for the three months ended June 30, 2017 was $74.4 million, or ($2.40) basic and diluted net loss per common share, as compared with a net loss of $28.7 million, or ($1.04) basic and diluted net loss per common share for the three months ended June 30, 2016.

Six Months Ended June 30, 2017 and 2016

In the six months ended June 30, 2017 and 2016, we recognized $2.8 million and $2.6 million, respectively, of revenue associated with our Pfizer collaboration.

Our research and development expenses for the six months ended June 30, 2017 were $65.3 million versus $37.9 million for the six months ended June 30, 2016. The $27.5 million increase was due to a $20.8 million increase in internal research and development expenses, primarily due to increased headcount, and an increase of $6.7 million in external research and development. The increase in external research and development was primarily from an increase of $3.1 million in expenses related to LUXTURNA, $1.8 million in our other programs in preclinical development and $1.8 million in our other clinical programs.

Our acquired in-process research and development expense for the six months ended June 30, 2017 was $3.5 million versus zero for the three months ended June 30, 2016. This amount represents an expense related to our license agreement with Selecta.

To read full press release, please click here.


Read at BioSpace.com


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