Release date: 2017-04-13
Recently, Spark Therapeutics, a company dedicated to gene therapy, released a new phase I/II trial of SPK-9001 for the treatment of hemophilia B at the 2017 HTRS (Study Society for Hemostasis and Thrombosis Research). Consistency and sustained levels of activity. As of the current data, the annual bleeding rate (ABR) has decreased by 96%, and the annualized infusion rate (AIR) has decreased by 99%.
Hemophilia is a rare hereditary bleeding disorder due to the absence of clotting factors. There are approximately 26,000 patients worldwide, of which approximately 4,000 are in the United States alone, and almost all of them are male. People with hemophilia B are deficient in factor IX, a special protein found in the blood. The current standard of treatment for hemophilia B requires patients to undergo repeated intravenous infusion of plasma-derived or recombinant factor IX to control or avoid the occurrence of hemorrhagic events, but this treatment strategy severely limits hemophilia. The patient's activity space, before the emergence of effective therapy, severe hemophilia patients often can not live to adulthood, the average life expectancy is only 11 years. For people with hemophilia, it is clear that they need a new treatment option. Now, because of the emergence of SPK-9001, for patients with hemophilia B, achieving the end of the disease may no longer be a dream.
SPK-9001 is an innovative bioengineered adenoviral capsid that expresses codon-optimized, highly active human factor IX, producing endogenous factor IX, which is also the history of hemophilia treatment. An optimistic approach to gene therapy uses highly optimized gene therapy selected, designed, produced and developed by Spark's proprietary technology platform. In 2014, Spark entered into a strategic partnership with Pfizer to develop hemophilia gene therapy. Under the agreement, Spark is responsible for all Phase I/II clinical studies of all candidate products in the SPK-FIX project. Pfizer is responsible for key clinical research, regulatory issues, and potential global commercialization activities.
In May 2016, SPK-9001 achieved initial success in treating hemophilia. In the Phase I/II clinical study, data from the first three subjects showed that the subject received an intravenous infusion of SPK-9001. A single intravenous infusion of 5x10 (11th power) vg/kg of SPK-9001 successfully achieved a sustained, therapeutic level of clotting factor IX expression that exceeded the level considered to be sufficient to reduce joint bleeding risk and prevent Threshold IX level of sexual infusion of clotting factors.
Among them, 2 subjects without a history of previous liver disease, the level of factor IX in the blood increased continuously during the first 4 weeks after a single infusion of SPK-9001. In the case of data submitted to EHA, 1 case was infused. The latter 8 weeks were stable at 28% of normal IX levels, and the other 1 was stable at 30% of normal IX levels 7 weeks after infusion. The third patient had a history of previous liver disease. After infusion of SPK-9001, blood factor IX levels also continued to increase, and remained stable at 16% of normal IX levels 3 weeks after infusion. Data from a natural history of patients with hemophilia B indicate that when the level of factor IX activity in the patient's blood circulatory system is maintained at a certain threshold (≥12% of normal IX levels), it is considered sufficient to reduce the risk of joint bleeding and prevention The need for sexual infusion of clotting factors.
In the summary submitted to EHA, none of the three subjects received conventional factor IX infusion to prevent bleeding events in a total 28-week observation. Only one subject received a prophylactic infusion for suspected ankle hemorrhage within 2 days after infusion of SPK-9001. In the study, SPK-9001 was well tolerated and none of the subjects required or received immunosuppressive therapy.
In July 2016, the US FDA granted SPK-9001 a breakthrough therapy approval that accelerated the approval process for the drug.
In December 2016, Spark and Pfizer announced the latest I/II clinical results of Spark-9001 at the ASH conference. In this clinical trial, 9 moderate-to-severe patients were enrolled. In the first year after Spark-9001 treatment, exogenous factor IX was no longer needed and no bleeding occurred, but Spark- was injected. In the year before 9001, 98 times of exogenous coagulation factor IX injection and 4 bleeding events occurred. His level of coagulation factor IX is currently maintained at 33%, which is close to normal.
For the 9 patients enrolled in this study, the cumulative treatment time was more than 1,650 days. According to the amount of coagulation factor IX injection in the year before their enrollment, the dosage was reduced by 99% after the treatment, and no IX inhibitor or thrombosis occurred in the patient. . Eight of the patients were no longer required to receive coagulation factor IX from the start of treatment, while the other patient was prophylactically injected with factor IX two days after the start of treatment to prevent joint bleeding, although the coagulation factor IX level was 36%, but still There were 2 suspected knee bleeding events. For 7 patients with a treatment time of more than 12 weeks, the steady-state factor IX level was between 12 and 46%, with an average of more than 28%, and more than 12% could prevent spontaneous bleeding of the joint.
Two of the patients developed an immune response against the viral vector 4-8 weeks after the injection with a transient increase in liver enzymes, with one patient's clotting factor IX level decreasing from 32% to 12%, while another patient used More aggressive corticosteroids control the immune response, reducing the level of factor IX from 71% to 68%. Nonetheless, neither of the two patients had been injected with exogenous factor IX and a bleeding event.
The latest experimental data set adds to the confidence of Spark, and SPK-9001 may be the terminator of hemophilia!
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Spark Therapeutics Presents Updated Preliminary Data from Hemophilia B Phase 1/2 Trial Suggesting Consistent and Sustained Levels of Factor IX Activity at the Hemostasis and Thrombosis Research Society (HTRS) 2017 Scientific Symposium
Source: Medical Valley
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