Item 7.01 Regulation FD Disclosure.
On March 6, 2023, BridgeBio Pharma, Inc. (the “Company”) issued a press release
announcing positive topline Phase 2 Cohort 5 data from its PROPEL2 clinical
trial of the investigational therapy infigratinib in children with
achondroplasia, a copy of which is furnished herewith as Exhibit 99.1 to this
Current Report on Form 8-K. The Company intends to host a conference call and
live webcast to discuss the interim clinical data on March 6, 2023 at 7:30 a.m.
E.T. The Company has made available a slide presentation to accompany the call,
a copy of which is being furnished as Exhibit 99.2 to this Current Report on
Form 8-K. The Company undertakes no obligation to update, supplement or amend
the materials attached hereto as Exhibit 99.2.
The information in this Item 7.01, including Exhibit 99.1 and Exhibit 99.2
attached hereto, is intended to be furnished and shall not be deemed “filed” for
purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the
“Exchange Act”), or otherwise subject to the liabilities of that section, nor
shall it be deemed incorporated by reference in any filing under the Securities
Act of 1933, as amended (the “Securities Act”), or the Exchange Act, except as
expressly set forth by specific reference in such filing.
Item 8.01 Other Events.
On March 6, 2023, the Company announced positive topline results from PROPEL2,
its Phase 2 clinical trial of infigratinib in children with achondroplasia. In
the highest dose level (Cohort 5, 0.25mg/kg once daily), the mean change from
baseline in annualized height velocity (“AHV”) at six months was +3.03 cm/yr (p
= 0.0022) for the first ten children with at least six months of follow-up in
Cohort 5 as of the data cutoff date. The two remaining children who did not yet
have six months of follow-up had a mean change from baseline in AHV of +8.8
cm/yr at three months. Infigratinib demonstrated clear dose-responsiveness when
given as a single daily oral dose and was well-tolerated with no
treatment-related adverse events (AEs) assessed in Cohort 5.
Key results from the clinical trial include:
• At the highest dose level evaluated to date (Cohort 5, 0.25 mg/kg once
daily), the mean increase from baseline in AHV for the ten children that
had six-month visits was +3.03 cm/yr (p = 0.0022). Individual data can be
found in Figure 1 below.
• The baseline AHV for the ten children with six-month visits was in the
expected range for children with achondroplasia at 3.73 cm/yr, increasing
to 6.77 cm/yr after treatment.
• The two remaining children who did not yet have six months of follow-up
had a mean change from baseline in AHV of +8.8 cm/yr at three months. The
mean age for Cohort 5 was 7.24 years.
• 80% of the 10 children with six-month visits were responders, with an
observed change from baseline AHV of at least 25%. Among the responders,
the average change from baseline in AHV was +3.81 cm/yr.
• Preliminary analysis of Collagen X (CXM) levels also showed a
statistically significant increase from baseline in Cohort 5 (p=.03). CXM
is a widely accepted biomarker of chondrocyte-driven growth and further
validates the response to infigratinib.
• Combined with the previously reported Cohort 4 change from baseline in
AHV value of +1.52 cm/yr, the Cohort 5 data demonstrated a strong dose
response for infigratinib.
• Median follow-up across all cohorts was 71.1 weeks. As of the cutoff
date, infigratinib has shown a well-tolerated safety profile, with no
study drug-related treatment emergent adverse events (TEAEs) in Cohort 5.
No serious adverse events (SAEs) or discontinuations due to AEs were
reported in any cohort.
Figure 1
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Based on the positive results to date, the Company has started enrolling
children in the run-in for a Phase 3 trial. Additionally, the Company expects to
initiate clinical development for infigratinib in hypochondroplasia, a skeletal
dysplasia closely related to achondroplasia and similarly driven by FGFR3
gain-of-function variants. The Company previously presented preclinical data for
hypochondroplasia at the ENDO 2022 Annual Conference and the American Society of
Human Genetics 2022 Annual Meeting.
Cautionary Note Regarding Forward Looking Statements
This Current Report on Form 8-K and certain of the materials filed herewith
contain forward-looking statements. Statements in this Current Report on Form
8-K or the materials furnished or filed herewith may include statements that are
not historical facts and are considered forward-looking within the meaning of
Section 27A of the Securities Act, and Section 21E of the Exchange Act, which
are usually identified by the use of words such as “anticipates,” “believes,”
“estimates,” “expects,” “intends,” “may,” “plans,” “projects,” “seeks,”
“should,” “will,” and variations of such words or similar expressions. The
Company intends these forward-looking statements to be covered by the safe
harbor provisions for forward-looking statements contained in Section 27A of the
Securities Act and Section 21E of the Exchange Act. These forward-looking
statements, including statements relating to the clinical, therapeutic and
market potential of the Company’s programs and product candidates, including its
clinical development program for infigratinib in achondroplasia, the timing and
success of its clinical development programs, the progress of its ongoing and
planned clinical trials of infigratinib in achondroplasia and in
hypochondroplasia, including its enrollment of patients in, and plans to
initiate conduct, a Phase 3 trial for infigratinib in achondroplasia and to
initiate clinical development of infigratinib in hypochondroplasia, its planned
interactions with regulatory authorities, the availability of data from its
clinical trials of infigratinib, the duration of the intellectual property
protection available for infigratinib, and the timing of these events, reflect
the Company’s current views about its plans, intentions, expectations and
strategies, which are based on the information currently available to the
Company and on assumptions it has made. Although the Company believes that its
plans, intentions, expectations and strategies as reflected in or suggested by
those forward-looking statements are reasonable, it can give no assurance that
the plans, intentions, expectations or strategies will be attained or achieved.
Furthermore, actual results may differ materially from those described in the
forward-looking statements and will be affected by a number of risks,
uncertainties and assumptions, including, but not limited to, initial and
ongoing data from the Company’s clinical trials not being indicative of final
data, the design and success of ongoing and planned clinical trials,
difficulties with enrollment in its clinical trials, adverse events that may be
encountered in its clinical trials, the FDA or other regulatory agencies not
agreeing with its regulatory approval strategies, components of its filings,
such as clinical trial designs, conduct and methodologies, or the sufficiency of
data submitted, potential adverse impacts due to the global COVID-19 pandemic
such as delays in regulatory review, manufacturing and supply chain
interruptions, adverse effects on healthcare systems and disruption of the
global economy, the impacts of current macroeconomic and geopolitical events,
including changing conditions from the COVID-19 pandemic, hostilities in
Ukraine, increasing rates of inflation and rising interest rates, on its overall
business operations and expectations, as well as those risks set forth in the
Risk Factors section of its Annual Report on Form 10-K for the year ended
December 31, 2022 and its other filings with the U.S. Securities and Exchange
Commission. Moreover, the Company operates in a very competitive and rapidly
changing environment in which new risks emerge from time to time. These
forward-looking statements are based upon the current expectations and beliefs
of the Company’s management as of the date of this Current Report on Form 8-K,
and are subject to certain risks and uncertainties that could cause actual
results to differ materially from those described in the forward-looking
statements. Except as required by applicable law, the Company assumes no
obligation to update publicly any forward-looking statements, whether as a
result of new information, future events or otherwise.
Item 9.01 Financial Statements and Exhibits.
99.1 Press release issued by BridgeBio Pharma, Inc. on March 6, 2023,
furnished herewith.
99.2 Corporate presentation, dated March 6, 2023, furnished herewith.
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