On Tuesday, BridgeBio Pharma, Inc. ( BBIO ) released topline results from the Phase 1/2 open-label ADventure study of BBP-631, an investigational adeno-associated virus (AAV) 5 gene therapy, for congenital adrenal hyperplasia (CAH).

CAH is a rare, inherited disorder that affects the adrenal glands and causes a hormone imbalance. The adrenal glands are on top of the kidneys and produce hormones that help the body function. 

Also Read: Why Is BridgeBio Pharma Stock Trading Higher On Friday?

To date, key results from the study include:

Increased endogenous cortisol production was achieved in all patients at higher doses.

At the two highest dose levels, a maximum change from the baseline post-ACTH stimulation test of 4.7 μg/dL and 6.6 μg/dL was observed, respectively, with cortisol levels as high as 11 μg/dL achieved.

Substantial and durable increases in 11-deoxycortisol, the product of 21-hydroxylase, and reductions in 17-hydroxyprogesterone (17-OHP), the substrate of 21-hydroxylase, provide compelling evidence of durable BBP-631 transgene activity. At the highest dose levels, sustained 11-deoxycortisol averaged a 55-fold increase from baseline, with a maximum of 99-fold increase. These represent an average maximum of 23-fold the upper limit of normal.

Robust reduction in 17-hydroxyprogesterone, with most patients reaching a reduction of ≥50%, with a max reduction of 95%.

BBP-631 has been well tolerated, with only mild to moderate treatment-emergent adverse events (TEAEs), and no treatment-related SAEs have been reported.

“While the data to date are not yet transformational, the study showed for the first time that people living with CAH can indeed make their own cortisol, and that gene therapy can be safely administered in this patient population…” said Neil Kumar, CEO and Founder of BridgeBio.

BridgeBio also said it will no longer be pursuing development of BBP-631 for CAH and is seeking partnership opportunities to support future development of BBP-631 or next-generation gene therapies for the treatment of CAH.

Price Action: BBIO stock is down 3.07% at $29.07 at the last check on Wednesday.

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