BioPharma, Pharma

Gearing Up to Challenge Pfizer in a Rare Disease, BridgeBio Secures Up to $1.25B

BridgeBio will receive $500 million in exchange for royalties from sales of acoramidis, a drug currently under FDA review for treating heart complications caused by the rare disease transthyretin amyloidosis. The deal also refinances lending agreements, providing BridgeBio with access to additional capital.

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BridgeBio Pharma is preparing to compete against a blockbuster Pfizer product for a rare disease with few treatment options. A new financing deal provides the company with the capital to support planned commercialization of its drug, which is currently under FDA review.

The agreement announced Thursday calls for Blue Owl Capital and Canada Pension Plan Investment Board to pay BridgeBio $500 million cash in exchange for royalties of 5% from sales of the biotech’s drug, acoramidis. The deal provides the Palo Alto, California-based biotech an additional $450 million in committed capital by refinancing its lending agreement with Blue Owl. The company could also receive an additional tranche of up to $300 million. In total, BridgeBio could receive up to $1.25 billion.

Acoramidis is a potential treatment for transthyretin amyloidosis, or ATTR. The inherited disease leads to abnormal versions of a liver protein called transthyretin, resulting in the buildup of amyloid protein in tissues and organs, including the heart. Acoramidis was developed to treat cardiomyopathy caused by ATTR. The small molecule is designed to bind to transthyretin, stabilizing it. The drug is intended to reduce protein buildup in tissue and organs.

Vyndaqel, Pfizer’s ATTR cardiomyopathy drug, is also a transthyretin stabilizer. The Vyndaqel family of products accounted for $2.3 billion in sales in the nine months ending Sept. 30, 2023, according to Pfizer’s financial reports. That’s a 33.6% increase compared to the same period in 2022. BridgeBio is angling for a piece of that market with a molecule that it believes will be competitive. The company has pointed to lab tests indicating its drug is the better transthyretin stabilizer. Last summer, the company reported statistically significant results from a Phase 3 test of acoramidis. In December, BridgeBio submitted an application seeking FDA approval of the drug.

Under the royalty agreement, the cash payment to BridgeBio is contingent on FDA approval of acoramidis. The deal caps royalty payments at 1.9 times the invested capital, which implies a cap of $950 million. In a securities filing, BridgeBio said the royalty may adjust to a maximum rate of 10% in 2027 under certain conditions that include the sales performance of the drug. The deal also includes a change in control provision that allows either party to terminate the pact prior to FDA approval. In a research note, Leerink Partners analyst Mani Foroohar wrote that this provision allows BridgeBio to keep options open for the drug and the company should a hypothetical acquirer emerge.

BridgeBio has already steered two drugs to FDA approvals, but they haven’t become significant contributors to revenue. In 2021, the agency approved Nulibry, making the BridgeBio molecule the first approved treatment for molybdenum cofactor deficiency type A, a rare metabolic disorder. Later that year, the FDA approved BridgeBio’s Truseltiq for the treatment of cholangiocarcinoma.

In 2022, BridgeBio sold Nulibry’s global rights to Sentynl Therapeutics, which is responsible for manufacturing and commercialization of the product. Truseltiq was partnered with Helsinn. But in 2022, Helsinn notified BridgeBio it would terminate the pact, citing commercial considerations. Helsinn stopped distributing the drug last year.

With limited cash coming in from drug sales, BridgeBio has raised capital by striking deals. In 2022, the company out-licensed to Bristol Myers Squibb a drug candidate that addresses KRAS mutations. That deal paid BridgeBio $90 million up front. The same month, BridgeBio landed another $110 million through the sale of the FDA priority review voucher it had received for the approval of Nulibry.

BridgeBio reported a $505.2 million cash position as of the end of September. The company said the capital from the royalty agreement will be applied toward the planned commercial launch of acoramidis. In addition to competing against the Pfizer drug, BridgeBio might also face competition from Alnylam Pharmaceutical’s Amvuttra, which is approved for treating neuropathy caused by ATTR. Alnylam is conducting clinical trials that could support expanding this drug’s use to ATTR cardiomyopathy. Amvuttra works differently than either the Pfizer or BridgeBio drugs, leveraging a mechanism called RNA interference. Phase 3 data are expected in the first half of this year.

Photo: BrianAJackson, Getty Images