8-K: BridgeBio's Attruby Receives FDA Approval for ATTR-CM Treatment, Showing Significant Reduction in Cardiovascular Events

BridgeBio's Attruby (acoramidis) has been approved by the FDA for treating ATTR-CM, demonstrating a significant reduction in cardiovascular death and related hospitalizations.

Summary

• The FDA has approved BridgeBio's Attruby (acoramidis) for the treatment of adults with ATTR-CM to reduce cardiovascular death and cardiovascular-related hospitalization.
• Attruby is the first and only approved product with a label specifying near-complete stabilization of TTR, at 90%.
• The approval is based on the ATTRibute-CM Phase 3 study, which showed a 42% reduction in composite all-cause mortality and recurrent cardiovascular-related hospitalization events relative to placebo at Month 30.
• The study also demonstrated a 50% reduction in the cumulative frequency of cardiovascular-related hospitalization events relative to placebo at Month 30.
• Attruby showed a statistically significant treatment effect at 30 months on the Kansas City Cardiomyopathy Questionnaire and 6-minute walk test.
• The increase in NT-proBNP on treatment was about half that of placebo.
• BridgeBio will provide Attruby free for life to U.S. clinical trial participants.
• The list price for Attruby is $18,759.12 for a 28-day supply.
• BridgeBio will receive a $500 million payment under a royalty funding agreement due to the approval.

Sentiment

Score: 9

Explanation: The document is highly positive due to the FDA approval of Attruby, the significant clinical trial results, and the $500 million royalty payment. The company also plans to expand globally.

Positives

• Attruby is the first and only approved product with a label specifying near-complete stabilization of TTR.
• The drug has shown a significant reduction in cardiovascular death and related hospitalizations.
• Attruby demonstrated the most rapid benefit seen in any Phase 3 study of ATTR-CM to date, with benefits seen as early as 3 months.
• The company is providing Attruby free for life to U.S. clinical trial participants.
• The FDA approval triggers a $500 million payment to BridgeBio under a royalty funding agreement.

Negatives

• The most common side effects reported were diarrhea (11.6% vs 7.6%) and upper abdominal pain (5.5% vs 1.4%) compared to placebo, though most were mild and resolved without drug discontinuation.
• There was a mean increase in serum creatinine of 0.2 mg/dL and a mean decrease in eGFR of 8.2 mL/min/1.73 m2 in patients treated with Attruby compared to placebo at Day 28, though these changes were reversible after treatment discontinuation.

Risks

• BridgeBio has only recently begun establishing its sales force and other commercialization capabilities, which may impact the successful launch of Attruby.
• The company is dependent on third parties for development, manufacture, and commercialization activities related to Attruby.
• Government and third-party payor actions, including reimbursement and pricing, could affect the commercial success of Attruby.
• Competitive products and other changes may limit demand for Attruby.
• Regulatory authorities may require additional studies or data to support continued commercialization.
• Drug-related adverse events may be observed during commercialization or clinical development.
• Global health emergencies, macroeconomic and geopolitical events could impact the business operations and expectations.

Future Outlook

BridgeBio plans to pursue approvals globally, next in Europe, Japan, and Brazil, and to continue exploring the full potential of this treatment. A decision on the European Marketing Authorization Application is expected in 2025.

Management Comments

• Neil Kumar, Ph.D., founder and CEO, stated that they are grateful to each trial participant, their families, and the physicians, scientists and their team at BridgeBio who made this possible.
• Neil Kumar, Ph.D., also mentioned that their journey is not over as they look to pursue approvals globally and to continue exploring the full potential of this treatment.
• Martha Grogan, M.D., of the Mayo Clinic, stated that having a new first line treatment option which provides excellent TTR stabilization and improves outcomes in this disease gives patients more options.

Industry Context

The approval of Attruby is significant in the ATTR-CM treatment landscape, as it is the first and only approved product with a label specifying near-complete stabilization of TTR. This approval provides a new treatment option for patients with this progressive disease, which has a poor prognosis when left untreated.

Comparison to Industry Standards

• Attruby's 90% TTR stabilization is a key differentiator, as it is the first and only approved product with this level of stabilization.
• The 42% reduction in composite ACM and recurrent CVH events at 30 months is a significant improvement compared to placebo, and is a strong result compared to other treatments in the ATTR-CM space.
• The rapid benefit seen as early as 3 months is also notable, as many other treatments take longer to show a significant effect.
• The study's Win Ratio of 1.8 (p<0.0001) on the primary endpoint is a strong indicator of the drug's efficacy.

Stakeholder Impact

• Shareholders will benefit from the FDA approval and the $500 million royalty payment.
• Patients with ATTR-CM will have a new treatment option that has shown significant benefits.
• Clinical trial participants will receive Attruby free for life.
• Employees of BridgeBio will be involved in the commercialization and global expansion of Attruby.

Next Steps

• BridgeBio will share management remarks on key aspects of the Attruby label and important access programs on November 22, 2024 at 8:00 pm ET.
• BridgeBio plans to pursue approvals globally, next in Europe, Japan, and Brazil.
• The company will continue to explore the full potential of this treatment.

Key Dates

Keywords

ATTR-CM, acoramidis, Attruby, FDA approval, cardiovascular death, hospitalization, TTR stabilizer, amyloidosis, BridgeBio, biopharmaceutical