Another rare disease M&A deal? BridgeBio draws buyout interest from Big Pharma: Bloomberg

Rare diseases and cancer are constantly featured in the biopharma M&A landscape. Now, one biotech company with a presence in both areas has reportedly caught Big Pharma’s interest.

Some large pharma companies are evaluating whether to buy BridgeBio Pharma, Bloomberg reports, citing people familiar with the matter.

No decision has been made, and BridgeBio may decide to wait until data readouts from upcoming clinical trials to reap the most value from a deal, the people told Bloomberg.

Still, the prime time for BridgeBio to sell itself passed a while ago. The young biotech took a serious hit in late 2021 from its transthyretin amyloidosis (ATTR) candidate acoramidis, a potential competitor to Pfizer’s blockbuster Vyndaqel/Vyndamax franchise and Alnylam’s Onpattro/Amvuttra duo.

BridgeBio’s stock price plunged after acoramidis’ phase 3 trial in ATTR-cardiomyopathy failed on its primary endpoint thanks to a surprise placebo outperformance. And even with a boost from Bloomberg’s report on Friday, BridgeBio’s stock was nowhere near its high in 2021 before the flop.

Based in Palo Alto, California, BridgeBio develops drugs for genetic diseases and cancers. These include Nulibry, which won an FDA approval in 2021 as the first therapy for an ultra-rare condition called molybdenum cofactor deficiency type A. The company also developed Truseltiq for FGFR-driven cancer, but its partners have since pulled out.

Those two indications represent small assets within BridgeBio’s overall pipeline. BridgeBio hasn’t given up on acoramidis in ATTR-CM, a market that by the company’s estimate could reach $10 billion to $15 billion annually. The company is now awaiting patient outcomes data—including death and cardiovascular-related hospitalizations—from the same phase 3 study in July to potentially support a regulatory filing. If the results turn out positive, the company’s stock price could rally, increasing its value in a potential M&A.

As for Truseltiq (infigratinib), BridgeBio is repurposing a low-dose version for achondroplasia, or dwarfism. Earlier this month, BridgeBio reported positive phase 2 results in annualized height velocity, which SVB Securities analysts said set a “new high water mark” in achondroplasia. The drug’s efficacy appeared better than BioMarin’s newly approved Voxzogo in cross-trial comparisons.

With the phase 2 win, BridgeBio is gearing up for phase 3 testing, targeting a mid-2025 readout, CEO Neil Kumar, Ph.D., said during an SVB investor event, according to a Monday note.

In addition, BridgeBio in late 2022 pushed encaleret into phase 3 testing for a rare genetic condition called autosomal dominant hypocalcemia type 1. Topline data from the pivotal study could also read out by year-end. The company is also working on some AAV-based gene therapies.

On the oncology side, BridgeBio’s early-stage SHP2 inhibitor, BBP-398, attracted Bristol Myers Squibb to a licensing deal worth up to $905 million. Three KRAS cancer candidates are in preclinical development.

With new programs nearing commercialization, BridgeBio appears open to dealmaking but is not necessarily set on a full-on buyout. During his discussion with SVB, Kumar said BridgeBio may seek commercial partnerships or a royalty deal to monetize acoramidis if the upcoming cardiovascular outcomes data are positive, according to SVB’s Monday note. The company is also considering potential royalty transactions on infigratinib in achondroplasia, encaleret or other assets, he told SVB.

BridgeBio hopes to reach $2 billion in annual sales in five years, the company said in a plan shared at the J.P. Morgan Healthcare Conference in January,

Meanwhile, both rare disease and oncology have been hot targets in biopharma M&A. Amgen recently offered around $28 billion to take over Horizon Therapeutics, which owns several rare and autoimmune disease therapies. In two smaller rare disease-focused deals unveiled in January, Chiesi bought Amryt Pharma for up to $1.5 billion, and Ipsen scooped up Albireo for up to $952 million.

In oncology, Pfizer is in the middle of a $43 billion buyout of antibody-drug conjugate specialist Seagen. Last year, Bristol Myers Squibb boosted its cancer pipeline by shelling out $4.1 billion for Turning Point Therapeutics, and GSK snatched Sierra Oncology for $1.9 billion.