- Spark and Pfizer kick out encouraging preliminary data in hemophilia B.
- And Roche had a major publication in hemophilia A!
- Amgen cements its place in the treatment of acute leukemia.
Welcome to another edition of "3 Things In Biotech You Should Learn Today," a daily digest dedicated to helping you keep up to date with recent findings in the fast-moving world of biotech and pharmaceutical research.
What's on the docket for today?
Spark and Pfizer kick out encouraging preliminary data in hemophilia B
Spark Therapeutics (ONCE) and Pfizer (PFE) have generated a lot of excitement for their jointly developed gene therapy solution for hemophilia, specifically hemophilia B, which results from a patient's genetic inability to make sufficient amounts of coagulation Factor IX.
The product in development is called SPK-9001, and it is an adeno-associated virus that delivers genetic material without risk of infection.
ONCE presented interim results at the 2017 International Society on Thrombosis and Haemostasis Congress as part of two presentations.
In abstract OC 13.1, the companies detailed the results of 10 patients enrolled in a phase 1/2 study, noting that all 10 patients had a 99 percent reduction in the rate of annual infusions of Factor IX, down from a mean of 67.5 to 1.0. Furthermore, 9 out of 10 patients had not experienced a bleeding event since the infusion of the vector, and annualized bleeding rate was reduced by some 96 percent.
In abstract PB 1900, analysis of questionnaires from patients enrolled in the phase 1/2 study suggested that their quality of life had improved, including their perception of dependence from Factor IX supplementation and decreased worry about their hemophilia worsening.
Looking forward: Yet another impressive notch for PFE and ONCE with this very interesting gene therapy. It feels increasingly likely that they have a substantially disruptive technology on their hands, with highly favorable tolerability. The only toxicity observed to date has been mild elevation of liver enzymes, which is quite manageable. No serious adverse events have been observed yet, though this is indeed a small patient population.
And Roche had a major publication in hemophilia A!
Despite the encouraging results of gene therapy, other biologics will not be supplanted just yet in hemophilia. Roche's (OTCQX:RHHBF) emicizumab is a "bridging" antibody that is specific for both Factor IX and Factor X, letting it function as a sort of mimic for natural Factor VIII, which is deficient in patients with hemophilia A.
RHHBF recently published the results of the phase 3 HAVEN 1 trial in the New England Journal of Medicine. This study sought to observe a difference in the rate of treated bleeding events when emicizumab was given as a preventative treatment in patients with hemophilia A of any severity.
The results were highly favorable in terms of risk of bleeding events. Patients receiving emicizumab had an 87% reduced rate of annualized bleeding (from 28.3 in the control group to 5.5 in the treated group). This benefit was also observed in patients who had received other therapies as preventative treatments for hemophilia A.
Looking forward: This is an interesting strategy for hemophilia A, bypassing the defective gene altogether and inserting some duct tape to bridge Factors IX and X. It is reminiscent of Amgen's (AMGN) blinatumomab for the treatment of ALL, and I expect that these results are going to be strong incentive when Roche makes its push for FDA approval.
Amgen cements its place in the treatment of acute leukemia
Speaking of blinatumomab, Amgen has not let up on the development of its own bridging antibody, this one grabbing CD3 and CD19 at the same time to bring white blood cells (CD3-positive) near tumor cells that express CD19. This has previously received conditional approval in relapsed/refractory acute lymphoblastic leukemia.
However, AMGN has also shown in the TOWER and ALCANTARA studies that blinatumomab might help patients in a wider variety of settings. Specifically, those who express the Philadelphia chromosome, which confers a very bad prognosis for patients.
AMGN recently announced the approval of its supplemental NDA to incorporate the overall survival improvement seen in the TOWER study, which converts the approval from conditional to full. Moreover, the FDA has approved blinatumomab for the treatment of Philadelphia chromosome-positive ALL.
Looking forward: Conditional approval remains something of a wildcard. Obviously, it's great for a company when an agent appears to have strong enough efficacy that it should be given early rather than wait around to confirm that overall survival is impacted. However, this also carries risk, as sometimes promising surrogates of survival don't translate into clinically meaningful survival, leaving the FDA and the company in awkward positions. This full approval is a great sign, and having another option for Philadelphia-positive ALL is essential, as well. Astute readers will recognize this as the fundamental feature of CML, where imatinib and other kinase inhibitors can give long-term control. This is not often the case in ALL; the kinase inhibitors help, but their responses tend not to be as durable. So more options are gravely needed, and it's good that AMGN has one on tap for these patients.
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