By Grant Zeng, CFA
The FDA Meeting
Earlier today (Sept. 5, 2017), Oramed Pharmaceuticals Inc. ( ORMP) announced that it has successfully concluded a meeting with the US FDA regarding ORMD-0801, the Company's novel oral insulin formulation.
Here are some highlights from the meeting:
• the FDA gave ORMD-0801 a clear guidance that the regulatory pathway for submission would be a Biologics License Application (BLA).
• Such a pathway would grant a full 12 years of marketing exclusivity for ORMD-0801 if approved. On top of this, an additional six months of exclusivity can be granted if the product also receives approval for use in pediatric patients.
• The FDA also confirmed at the meeting that the approach to nonclinical toxicology, CMC and qualification of excipients would be driven by their published guidance documents, consistent with the Company's expectations.
• The FDA also made specific recommendations for clinical trials designed to provide pivotal data prior to registration.
Since oral insulin may have a positive more physiologic first-pass effect on the liver with less systemic insulin exposure compared to traditional injectable insulin, at the suggestion of the FDA, Oramed plans to initiate a three-month trial in patients with type 2 diabetes to evaluate the effect of ORMD-0801 on HbA1c, the main FDA registrational endpoint, later this year.
In addition, the FDA confirmed the Company's ability to use insulin from different suppliers like HTBT (Hefei Tianmai Biotechnology Development Co., Ltd., Hefei, China) in the Phase III study.
Oramed plans on implementing the FDA's feedback in the coming months which should facilitate the confirmatory Phase III study and registration of ORMD-0801.
Initial Top Line Phase II Data
On May 18, 2016, Oramed announced positive top-line data from the Phase IIb study of ORMD-0801. The study achieved its primary objective: a significant reduction of weighted mean night-time glucose in patients treated with oral insulin ORMD-0801.
This study showed a statistically significant decrease in the primary endpoint, pooled night-time glucose mean percentage change of 6.47% from run-in, between placebo and active cohorts (p=0.0268).
Further, the study demonstrated a good safety profile of ORMD-0801 with no drug related serious adverse events.
On July 28, Oramed reported additional data from the Phase IIb trial. In addition to positive topline data showing the study successfully met its primary efficacy and safety endpoints announced on May 18, the new data indicated a statistically significant lowering of glucose relative to placebo across several endpoints.
Due to technical inaccuracies that can occur in any diabetes study, measuring glucose changes with continuous glucose monitors (CGM), data can include extreme outliers. To reduce variability, trimming of unlocked, fully blinded information was conducted. In the current summary, the 80% trimmed data (data excluding the 10% highest and lowest values) is presented.
In the study, the mean nighttime glucose showed a significant difference in mean change from run-in (13.70 mg/dL for placebo vs. 1.66 mg/dL for the pooled ORMD-0801 arms with a p= 0.0117). ORMD-0801 was safe and well tolerated, with no drug related serious or severe adverse events and no statistically significant differences in laboratory values or vital signs.
Other secondary and exploratory objectives of the study included evaluating the effect of ORMD-0801 on mean 24-hour glucose, fasting glucose, and daytime glucose. The mean 24-hour glucose showed a highly significant difference in mean change from run-in (13.26 mg/dL for placebo vs. -0.32 mg/dL for ORMD-0801, p <0.0001). The mean fasting glucose showed a highly significant difference in mean change from run-in (15.95 mg/dL for placebo vs. -0.41 mg/dL for ORMD-0801, p <0.0001). The mean daytime CGM glucose showed a highly significant difference in mean change from run-in (11.88 for placebo vs. 0.88 for ORMD-0801, p= 0.0010).
With the guidance from the FDA, we believe the Company will accelerate the initiation of a pivotal Phase III study of ORMD-0801.
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