Item 1.01. Entry into a Material Definitive Agreement.
On
Pursuant to the terms and conditions of the First Amendment, the parties thereto
have agreed to, among other things: (1) permit the sale of a certain priority
review voucher issued by the
Other terms of the Amended Loan Agreement remain generally identical to those under the Original Loan Agreement.
The above description of the material terms of the First Amendment does not
purport to be complete and is qualified in its entirety by reference to the full
text of the First Amendment, which will be filed, with confidential terms
redacted, as an exhibit to the Company's Quarterly Report on Form 10-Q for the
period ending on
On
Item 2.03. Creation of a Direct Financial Obligation or an Obligation under an Off-Balance Sheet Arrangement of a Registrant.
The information set forth above in Item 1.01 of this Current Report on Form 8-K regarding the Company financial obligations under the First Amendment and the Amended Loan Agreement is incorporated into this Item 2.03 by reference.
Item 8.01. Other Events. Pediatric Review Voucher Sale
On
On
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Recent Developments
Updated Results from Phase 2 Open-label Extension Study of Acoramidis in Transthyretin Amyloid Cardiomyopathy
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• 31 of 47 participants remained in the OLE study; of the 16 discontinuations, adverse events (AEs) with an outcome of death, cardiac transplant or transition to hospice were reported for 11 participants • Acoramidis remained generally well-tolerated with a pattern of AEs consistent with underlying disease, progression of disease, concurrent illnesses, and age of participants. No safety signals of clinical concern were identified • Acoramidis demonstrated near-complete TTR stabilization. Serum TTR levels were sustainably increased from baseline, with mean concentration rising from 21.55 mg/dL at baseline to 30.06 mg/dL at Month 30 (+41%). Near-complete stabilization was verified using established ex vivo assays with mean stabilization of 102.5 ± 8.9% at Month 30 • Median N-terminal Pro-brain natriuretic peptide (NT-ProBNP) were stable or improving in study participants. At Month 30, median change from baseline in NT-proBNP was -437 pg/mL (interquartile range: -950, 316). 68% of participants with available samples at Month 30 (15/22) had NT-proBNP levels below their baseline, suggesting an improvement in their heart failure severity
The Phase 2 OLE data continue to suggest long-term tolerability of acoramidis in ATTR-CM patients and a stabilization of disease progression in treated participants.
Phase 2 Data for Limb-girdle Muscular Dystrophy Type 2i
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• Participants showed an average 0.21 or 43% increase in the ratio of glycosylated ?DG to total ?DG, signifying that the oral therapy has the potential to address both the root cause of LGMD2i and drive functional improvements for patients • Participants showed statistically significant declines in all cohorts for CK, of 70% at day 90 for all cohorts and 77% at day 180 for cohorts 1 and 2. 11 of 12 participants received at least 50% reduction in CK with 75% of participants reaching 2x the normal range, suggesting a reduction in muscle breakdown • All cohorts demonstrated a 0.08 m/sec (3%) increase in 10MWT velocity at day 90 and 0.12 m/sec (4%) increase at day 180 for cohorts 1 and 2. This result is encouraging in correlating the positive biomarker changes to potential clinical outcomes • The 10MWTs were measured at six months and compare favorably to natural history data where the same patients demonstrated a decline of 0.12 m/sec in the 10MWT in the 6-months prior to enrollment in the Phase 2 study • BBP-418 was well-tolerated across a wide range of dose levels with no treatment-related serious adverse events, dose limiting toxicities or discontinuations observed
BridgeBio plans to engage with regulatory health bodies in 2022 to discuss potential paths to approval and subsequently intends to initiate a Phase 3 clinical trial.
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Item 9.01. Financial Statements and Exhibits.
(d) Exhibits. Exhibit Number Description 99.1 Press Release datedMay 13, 2022 , furnished herewith 104 Cover Page Interactive Data File (embedded within the Inline XBRL document).
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