Item 7.01 Regulation FD Disclosure.
On December 17, 2021, Passage Bio, Inc. (the “Company”), issued a press release announcing positive interim safety and 30-day biomarker data for cohort 1 of its global Phase 1/2 clinical trial for PBGM01 (“Imagine-1”) for the treatment of early and late infantile GM1 Gangliosidosis (“GM1”). A copy of the press release is attached as Exhibit 99.1 to this Current Report on Form 8-K, which is incorporated herein by reference.
On December 17, 2021, the Company will also give a presentation describing its positive interim safety and 30-day biomarker data for cohort 1 of Imagine-1. A copy of this presentation is attached as Exhibit 99.2 to this Current Report on Form 8-K, which is incorporated herein by reference.
The information in this Item 7.01, including Exhibits 99.1 and 99.2 to this report, shall not be deemed to be “filed” for purposes of Section 18 of the Exchange Act, or otherwise subject to the liabilities of that section or Sections 11 and 12(a)(2) of the Securities Act. The information contained in this Item 7.01 and in the accompanying Exhibits 99.1 and 99.2 shall not be incorporated by reference into any other filing under the Exchange Act or under the Securities Act, except as shall be expressly set forth by specific reference in such filing.
Item 8.01 Other Events.
PBGM01 Program Updates
The Company announced the following updates with respect to Imagine-1 for the treatment of early and late infantile GM1:
| · | The Independent Data Monitoring Committee (the “IDMC”) recommended proceeding with two additional planned cohorts in the Imagine-1 clinical study. |
| · | Based on the IDMC recommendation, the Company has initiated recruitment in parallel for both a high-dose cohort of patients with late infantile GM1 (cohort 2) and a low-dose cohort of patients with early infantile GM1 (cohort 3). |
| · | Topline interim results from the Imagine-1 cohort 1, consisting of two patients (patient 1 data through eight months and patient 2 data through two months), showed: |
| o | With respect to safety, no serious adverse events were reported, and the reported adverse events were all mild to moderate. All moderate adverse events resolved without intervention and were deemed not to be treatment related. No complications related to intra cisterna magna injection were observed, and there was no evidence of dorsal root ganglion toxicity in nerve conduction studies. |
| o | With respect to beta-galactosidase enzyme activity levels, following PBGM01 administration, beta-galactosidase activity in cerebrospinal fluid (“CSF”) and serum increased in both patients. |
In CSF, for patient 1, enzyme activity at 30 days was 1.5-fold over baseline and the increase was maintained at six months. For patient 2, enzyme activity at 30 days was 4.8-fold over baseline.
In serum, for patient 1, enzyme activity was slightly above baseline at 30 days, 1.7-fold over baseline at three months and the increase was maintained at six months. For patient 2, enzyme activity at 30 days was 1.2-fold over baseline.
