BridgeBio Receives FDA’s Regenerative Medicine Advanced Therapy (RMAT) Designation for BBP-812 Canavan Disease Gene Therapy Program

Rhea-AI Summary

BridgeBio Pharma (Nasdaq: BBIO) has received Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA for BBP-812, its gene therapy program for Canavan disease. This designation is based on promising clinical evidence from the CANaspire Phase 1/2 trial, which showed functional improvements in all dosed patients. RMAT status offers benefits like faster and more frequent FDA interactions, potentially accelerating the approval process.

Key points:

• BBP-812 is an intravenous AAV9 gene therapy for Canavan disease
• All patients in the trial showed improvements in key functional areas
• The therapy has been well-tolerated with a safety profile consistent with other AAV9 gene therapies
• BBP-812 also has Orphan Drug, Rare Pediatric Disease, and Fast Track Designations

If approved, BBP-812 could be the first treatment option for this fatal neurodevelopmental disorder.

Positive

• RMAT designation granted by FDA for BBP-812 Canavan disease gene therapy
• Functional improvements observed in all dosed patients in CANaspire Phase 1/2 trial
• Potential for accelerated approval pathway due to RMAT designation
• Well-tolerated safety profile consistent with other AAV9 gene therapies
• Multiple regulatory designations (Orphan Drug, Rare Pediatric Disease, Fast Track) received
• Potential to be first treatment option for Canavan disease if approved

Negative

• None.

Medical Research Analyst

The FDA's granting of RMAT designation for BBP-812 is a significant milestone for BridgeBio's Canavan disease gene therapy program. This designation, based on promising 12-month data from eight patients, could accelerate the development and review process. Key points:

• All dosed patients showed functional improvements in areas like head control and visual tracking
• Reductions in N-acetylaspartate (NAA) levels were observed, indicating potential disease modification
• The therapy has been well-tolerated with a safety profile consistent with other AAV9 gene therapies

This news is encouraging for Canavan disease patients, as there are currently no approved treatments for this fatal neurodevelopmental disorder. The RMAT designation, combined with previous Orphan Drug and Fast Track designations, positions BBP-812 for a potentially expedited path to market.

Biotech Industry Analyst

The RMAT designation for BBP-812 is a positive development for BridgeBio (NASDAQ: BBIO), potentially accelerating their path to market in the rare disease space. Key implications:

• Enhanced regulatory interactions could lead to faster development and reduced costs
• Potential for Accelerated Approval pathway, which could significantly shorten time to market
• If approved, BBP-812 could be the first-to-market therapy for Canavan disease, capturing an untapped market

Investors should note that while promising, gene therapies face challenges in manufacturing and pricing. The potential Priority Review Voucher, if granted upon approval, could provide additional value, either for use or sale. This news strengthens BridgeBio's position in the competitive rare disease and gene therapy landscape.

Financial Analyst

The RMAT designation for BBP-812 is a positive catalyst for BridgeBio (NASDAQ: BBIO), potentially enhancing its market position and financial outlook. Key financial implications:

• Accelerated development could lead to earlier revenue generation and reduced R&D costs
• First-to-market status in Canavan disease treatment could command premium pricing
• Potential Priority Review Voucher upon approval could be worth $100-$200 million if sold

While the immediate financial impact is , this news reduces regulatory risk and strengthens BridgeBio's rare disease portfolio. Investors should monitor progress in the ongoing clinical trial and any updates on potential pricing or market size estimates for this ultra-rare indication. The company's ability to leverage this success across its broader pipeline will be important for long-term value creation.

- Receipt of RMAT Designation is based on preliminary clinical evidence from the CANaspire Phase 1/2 clinical trial, which showed functional improvements in all dosed patients indicating that BBP-812 has potential to address the unmet needs of individuals with Canavan disease

- BridgeBio will leverage the benefits of RMAT designation, including early and more frequent interactions with the FDA, to establish an Accelerated Approval pathway for BBP-812

- If approved, BridgeBio’s gene therapy for Canavan disease could be the first therapeutic option for children born with this devastating and fatal neurodevelopmental disorder

PALO ALTO, Calif., Sept. 10, 2024 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (BridgeBio), a commercial-stage biopharmaceutical company focused on genetic diseases, today announced that the United States Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to BBP-812, an investigational intravenous (IV) adeno-associated virus serotype 9 (AAV9) gene therapy for the treatment of Canavan disease. RMAT designation was granted following the FDA’s review of clinical data from the CANaspire Phase 1/2 clinical trial investigating BBP-812 as a potential therapy to address the unmet medical needs of individuals with Canavan disease.

RMAT is an expedited FDA program available to sponsors of regenerative medicine therapies intended to treat, modify, reverse, or cure serious conditions. Benefits of the RMAT designation include all the advantages of the Fast Track and Breakthrough Therapy Designation programs, including faster and more frequent interactions with the FDA to achieve early alignment on critical aspects of the program. FDA granted RMAT designation based on its review of 12 months of safety and efficacy data from the first eight patients with Canavan disease dosed with BBP-812 in the CANaspire Phase 1/2 clinical trial.

“We are honored to be granted RMAT designation for BBP-812 and are eager to work closely with the FDA and the Canavan community with the goal of bringing our therapy to families living with Canavan disease as fast as possible,” said Eric David, M.D., J.D., CEO at BridgeBio Gene Therapy. “We are beyond grateful to the children and their families who are participating in CANaspire, as well as to the study investigators. RMAT will allow us to work more closely with FDA to ensure we are responding to the urgency that families feel.”

To date, results from CANaspire show that all patients dosed with at least one follow-up assessment have demonstrated improvements in functional outcomes in key areas important to caregivers such as head control, sitting upright, reaching for and grasping objects, and visual tracking. All patients dosed with BBP-812 with at least one follow-up assessment have shown reductions in N-acetylaspartate (NAA), both in urine and in the central nervous system, to levels associated with mild disease. BBP-812 has been well-tolerated, with a safety profile generally consistent with that of other AAV9 gene therapy programs.

“Canavan disease is an extremely rare and rapidly progressive neurodegenerative disease that prevents most children from meeting basic developmental milestones, such as crawling, walking, speaking, and even holding their heads up. It is a terminal diagnosis with no approved treatment to date. The news of the RMAT designation, coupled with the preliminary results seen in the clinical trial, provides hope to children worldwide living with Canavan disease and their families,” said Kathleen Flynn, CEO of National Tay-Sachs & Allied Diseases Association, an advocacy organization dedicated to driving research, forging collaboration, and supporting families within the Tay-Sachs, Canavan, GM1, and Sandhoff disease communities.

In addition to RMAT designation, BBP-812 has been granted Orphan Drug, Rare Pediatric Disease (RPDD), and Fast Track Designations from the FDA, as well as Orphan Drug Designation from the European Medicines Agency. With RPDD, if approved, BridgeBio may qualify for a Priority Review Voucher.

About CANaspire
CANaspire is a Phase 1/2 open-label study designed to evaluate the safety, tolerability, and pharmacodynamic activity of BridgeBio’s AAV9 gene therapy candidate, BBP-812, in pediatric patients with Canavan disease. Each eligible patient will receive a single IV infusion of BBP-812. The primary outcomes of the study are safety, as well as change from baseline of urine and central nervous system NAA levels. Motor function and development will also be assessed.

For more information about the CANaspire trial, visit TreatCanavan.com or ClinicalTrials.gov (NCT04998396).

About Canavan Disease
Affecting approximately 1,000 children in the U.S. and European Union, Canavan disease is an ultra-rare, disabling and fatal disease with no approved therapy. Most children are not able to meet developmental milestones, are unable to crawl, walk, sit or talk, and die at a young age. The disease is caused by an inherited mutation of the ASPA gene that codes for aspartoacylase, a protein that breaks down a compound called NAA. Deficiency of aspartoacylase activity results in accumulation of NAA, and ultimately results in toxicity to myelin in ways that are not currently well understood. Myelin insulates neuronal axons, and without it, neurons are unable to send and receive messages as they should. The current standard of care for Canavan disease is limited to supportive therapy.

About BridgeBio Pharma, Inc.
BridgeBio Pharma, Inc. (BridgeBio) is a commercial-stage biopharmaceutical company founded to discover, create, test and deliver transformative medicines to treat patients who suffer from genetic diseases. BridgeBio’s pipeline of development programs ranges from early science to advanced clinical trials. BridgeBio was founded in 2015 and its team of experienced drug discoverers, developers and innovators are committed to applying advances in genetic medicine to help patients as quickly as possible. For more information visit bridgebio.com and follow us on LinkedIn, Twitter and Facebook.

BridgeBio Pharma, Inc. Forward-Looking Statements
This press release contains forward-looking statements. Statements BridgeBio makes in this press release may include statements that are not historical facts and are considered forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended (the “Securities Act”), and Section 21E of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), which are usually identified by the use of words such as “anticipates,” “believes,” “continues,” “estimates,” “expects,” “hopes,” “intends,” “may,” “plans,” “projects,” “remains,” “seeks,” “should,” “will,” and variations of such words or similar expressions. BridgeBio intends these forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act and Section 21E of the Exchange Act. These forward-looking statements, including statements relating to the timing and success of BridgeBio’s Phase 1/2 clinical trial of BBP-812 for the treatment of Canavan disease, expectations, plans and prospects regarding BridgeBio’s regulatory approval process for BBP-812, the ability of BBP-812 to be the first therapeutic treatment option for children born with Canavan disease, reflect BridgeBio’s current views about its plans, intentions, expectations, strategies and prospects, which are based on the information currently available to BridgeBio and on assumptions BridgeBio has made. Although BridgeBio believes that its plans, intentions, expectations, strategies and prospects as reflected in or suggested by those forward-looking statements are reasonable, BridgeBio can give no assurance that the plans, intentions, expectations or strategies will be attained or achieved. Furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by a number of risks, uncertainties and assumptions, including, but not limited to, BridgeBio’s ability to continue and complete its Phase 1/2 clinical trial of BBP-812 for the treatment of Canavan disease, BridgeBio’s ability to advance BBP-812 in clinical development according to its plans, the ability of BBP-812 to treat Canavan disease, the ability of BBP-812 to retain Fast Track Designation, Rare Pediatric Drug Designation, Regenerative Medicine Advanced Therapy Designation and Orphan Drug Designation from the U.S. Food and Drug Administration and Orphan Drug Designation from the European Medicines Agency, and potential adverse impacts due to global health emergencies, including delays in regulatory review, manufacturing and supply chain interruptions, adverse effects on healthcare systems and disruption of the global economy, the impacts of current macroeconomic and geopolitical events, including changing conditions from hostilities in Ukraine and in Israel and the Gaza Strip, increasing rates of inflation and rising interest rates, on our business operations and expectations as well as those risks set forth in the Risk Factors section of BridgeBio’s most recent Annual Report on Form 10-K, and BridgeBio’s other filings with the U.S. Securities and Exchange Commission. Moreover, BridgeBio operates in a very competitive and rapidly changing environment in which new risks emerge from time to time. These forward-looking statements are based upon the current expectations and beliefs of BridgeBio’s management as of the date of this press release and are subject to certain risks and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. Except as required by applicable law, we assume no obligation to update publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

BridgeBio Contact:
Vikram Bali
contact@bridgebio.com
(650)-789-8220

FAQ

What is the RMAT designation BridgeBio received for BBP-812?

BridgeBio received Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA for BBP-812, its gene therapy program for Canavan disease. This designation offers benefits like faster and more frequent FDA interactions, potentially accelerating the approval process.

What were the results of the CANaspire Phase 1/2 trial for BBIO's Canavan disease therapy?

The CANaspire Phase 1/2 trial showed functional improvements in all dosed patients. Patients demonstrated improvements in key areas such as head control, sitting upright, reaching for objects, and visual tracking. The therapy was also well-tolerated with a safety profile consistent with other AAV9 gene therapies.

How could RMAT designation impact the development of BBP-812 for BBIO?

RMAT designation allows BridgeBio to have faster and more frequent interactions with the FDA, potentially establishing an Accelerated Approval pathway for BBP-812. This could help bring the therapy to market more quickly for patients with Canavan disease.

What other regulatory designations has BBIO received for BBP-812?

In addition to RMAT designation, BBP-812 has received Orphan Drug, Rare Pediatric Disease (RPDD), and Fast Track Designations from the FDA, as well as Orphan Drug Designation from the European Medicines Agency.