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TICKERS: BBIO

New Drug for Bone Growth Disorder Improves Height Velocity
Research Report

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Phase 2 trial data at six months are positive in children with achondroplasia, noted a BTIG report.

Six-month data on BridgeBio Pharma Inc.'s (BBIO:NASDAQ) infigratinib from the PROPEL2 Phase 2 trial, Cohort 5, are consistent with earlier results and look to be significantly more efficacious as an oral drug versus injectable competitors, reported BTIG analyst Dr. Thomas Shrader in a June 19 research note.

"Low-dose infigratinib for achondroplasia continues to look best in class," Shrader wrote. "We continue to believe Part B is promising and should be a positive trial."

The genetic bone growth disorder, achondroplasia, prevents the changing of cartilage to bone, particularly in the long bones of the arms and legs.

Compelling Return on Investment

The potential gain for investors in BridgeBio is projected to be significant, according to the difference between BTIG's price target on the biotech (US$32 per share) and its current share price (US$15.01). The upside from here is an estimated 113%.

BridgeBio is a Buy.

New Results Positive

Shrader summarized the new data from the dose-finding study and compared them to previously reported ones.

Oral infigratinib was shown to significantly increase height velocity in children with achondroplasia, he noted. Cohort 5 patients administered infigratinib 0.25 milligrams per kilogram per day showed a statistically significant (p=0.001) average increase of +3.38 centimeters per year (+3.38 cm/year) in annualized height velocity (AHV) at six months. This result is higher than the AHV reported in March, which was +3.03 cm/year.

In the responder analysis, ten out of 12, or 83% of, children showed at least a 25% increase in AHV, with responders showing a mean AHV of +4.08 cm/year. Also higher this time were dose-dependent mean height z score, or ACH growth curve, changes. Further, a dose-dependent increase in collagen X, a bone growth biomarker, was seen.

Cohort 5 showed a reduction in the mean upper-to-lower body segment ratio at six months. This result was 1.84, and the baseline was 2.06.

Safety outcomes were consistent between the different data sets, with no significant adverse events or treatment-associated adverse events observed.

Upcoming Catalysts

Several events are slated to happen in the remainder of this year that could potentially boost BridgeBio's stock, Shrader noted and presented them.

Regarding infigratinib for achondroplasia, once the Phase 2 trial is completed, BridgeBio will meet with the U.S. Food and Drug Administration and European Medicines Agency.  

As for the biotech's other investigational compounds, Part B topline data are expected in late July from ATTRibute-CM, the global Phase 3 study investigating acoramidis for the treatment of symptomatic transthyretin amyloid cardiomyopathy.

A Phase 3 trial of BBP-418 in patients with limb-girdle muscular dystrophy type 2i is scheduled to start around mid-2023. BridgeBio plans to submit an investigational new drug application for BBO-8520 in H2/23.


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  1. Doresa Banning wrote this article for Streetwise Reports LLC and provides services to Streetwise Reports as an independent contractor.
  2. The article does not constitute investment advice. Each reader is encouraged to consult with his or her individual financial professional and any action a reader takes as a result of information presented here is his or her own responsibility. By opening this page, each reader accepts and agrees to Streetwise Reports' terms of use and full legal disclaimer. This article is not a solicitation for investment. Streetwise Reports does not render general or specific investment advice and the information on Streetwise Reports should not be considered a recommendation to buy or sell any security. Streetwise Reports does not endorse or recommend the business, products, services or securities of any company mentioned on Streetwise Reports.
  3. This article does not constitute medical advice. Officers, employees and contributors to Streetwise Reports are not licensed medical professionals. Readers should always contact their healthcare professionals for medical advice.

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Disclosures for BTIG Research, BridgeBio Pharma Inc., June 19, 2023

Analyst Certification I, Thomas Shrader, hereby certify that the views about the companies and securities discussed in this report are accurately expressed and that I have not received and will not receive direct or indirect compensation in exchange for expressing specific recommendations or views in this report. I, Sung Jun Hong, hereby certify that the views about the companies and securities discussed in this report are accurately expressed and that I have not received and will not receive direct or indirect compensation in exchange for expressing specific recommendations or views in this report.

Company–Specific Regulatory Disclosures BTIG LLC expects to receive or intends to seek compensation for investment banking services in the next 3 months from: BridgeBio Pharma, Inc. (BBIO)

Other Disclosures Additional Information Available Upon Request

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