Breaking News

BridgeBio Pharma presented positive data from six participants dosed in CANaspire, its Phase 1/2 clinical trial of BBP-812, an investigational intravenous adeno-associated virus serotype 9 gene therapy for the treatment of Canavan disease. As of March 23, 2023, findings from the trial include: All participants showed a rapid and lasting decrease in levels of NAA, a key chemical marker elevated in children with Canavan disease, after dosing with BBP-812; The duration of individual participant follow-up ranged from 1 to 15 months post-treatment; The most recent data continue to show reductions in NAA in all participants and all compartments tested. Across individual participants, percent decreases in NAA from baseline ranged between 70% and 95% in cerebrospinal fluid, 29% and 88% in urine, and 8% and 75% in brain; All participants in the CANaspire trial had urine NAA levels consistent with typical Canavan disease prior to receiving BBP-812. After receiving BBP-812, urine NAA levels in all participants fell to what can be considered less severe Canavan disease as reported in the scientific literature and observed in the Company's natural history study MRI scans indicated the presence of improved myelination, which is essential for brain development, in the brainstem and cerebellum of all participants; These findings have been noted as early as 3 months after BBP-812 administration and were present at post-dosing Month 12 in the participant who has been followed the longest in the trial; In contrast to the natural history of typical Canavan patients in which already-limited functional abilities are lost over time, no trial participants have lost function and there have been incremental improvements in achievement of developmental milestones ; Improvement in the GMFM-88 sitting dimension has been shown to varying degrees in all participants who have been evaluated post-treatment, including one participant who has developed the ability to walk with a mobility aid and has a score on the GMFM-88 sitting dimension consistent with that of unaffected children; BBP-812 has been shown to be generally well-tolerated to date, with a safety profile consistent with other systemically administered AAV9 gene therapies

BridgeBio Pharma announced that on May 9, 2023, the compensation committee of BridgeBio's board of directors granted 12 new employees restricted stock units for an aggregate of 149,400 shares of the Company's common stock. One-fourth of the shares underlying each employee's restricted stock units will vest on May 16, 2024, with one-twelfth of the remaining shares underlying each such employee's restricted stock units vesting on a quarterly basis thereafter, in each case, subject to each such employee's continued employment with the Company or one of its subsidiaries on such vesting dates. All of the above-described awards were made under BridgeBio's 2019 Inducement Equity Plan. The above-described awards were each granted as an inducement material to the employees entering into employment with the Company in accordance with Nasdaq Listing Rule 5635(c)(4) and were granted pursuant to the terms of the Plan. The Plan was adopted by BridgeBio's board of directors in November 2019 and has been amended and restated from time to time.

Cash, cash equivalents, marketable securities and restricted cash, totaled $467.0 million as of March 31, 2023, compared to $466.2 million as of December 31, 2022. The net increase of $0.8 million in cash, cash equivalents, marketable securities and restricted cash is primarily attributable to the net proceeds received of $143.0 million from the follow-on public offering, and proceeds from common stock issuance under ESPP and stock option exercises of $2.0 million, partially offset by net cash used in operating activities of $144.3 million.