The last time I spoke about BridgeBio Pharma, Inc. (NASDAQ:BBIO), was stated in a Seeking Alpha article titled “BridgeBio Pharma: Acromidis FDA application and first results are known.“The most important What is notable about this article is that I mentioned that the New Drug Application (NDA) for this drug for the treatment of patients with transthyretin amyloid cardiomyopathy (ATTR-CM) has been submitted to the FDA. However, at that time there would be a review period during which the agency would decide whether to accept the submission of this application.

I am pleased to report that the FDA has not only accepted Acoramidis’ NDA application for the treatment of this patient population, but has also set a PDUFA date of November 29, 2024. Even better than that, Acoramidis’ Marketing Authorization Application (MAA) for this patient population was also accepted, making for success on European soil. Speaking of which, another important event that happened since I last spoke about this is that it linked a licensing agreement to Bavarian (OTCPK:BAYRYThis large pharmaceutical company would have licensing rights to sell this drug to this patient population in Europe.

This raised a lot of money, along with the fact that BridgeBio now has a major pharmaceutical partner to commercialize the drug in that area. As for the use of BBP-631 for the treatment of patients with congenital adrenal hyperplasia (CAH), a go/no go update is scheduled for August 2024. That is, a program update will be provided at that time, indicating whether it plans to continue development with this particular program.

Acoramidis review date set with potential to target large ATTR-CM market

As I mentioned above, things are looking very good for BridgeBio, as the FDA has accepted its NDA for Acoramidis for the treatment of patients with ATTR-CM. That said, the PDUFA date on which the FDA will review whether to approve this drug for the treatment of this patient population has been set for November 29, 2024. What’s even better about this set review date is that at the time of acceptance, it was announced that the FDA would not be holding an advisory committee to discuss it.

What’s even better is that there is a possibility to get this drug approved for the European territory as well. That’s because the European Medicines Agency (EMA) has accepted the Marketing Authorization Application (MAA) of Acoramidis for this patient population as well. Both acceptances of these applications for review of this drug bode well for the future of this company. There is even a possibility to expand this drug to other territories through other regulatory submissions planned in the coming years.

As for commercializing Acoramidis for ATTR-CM, BridgeBio is not going it alone. Instead, it has chosen to license the European rights to a partner called Bayer. This large pharmaceutical company will be responsible for marketing this drug in this territory. I believe this is a good deal for BridgeBio and that is because it achieves a couple of things. The first is that it raised a large amount of money. It actually received an upfront payment of $300 million and a short-term loan milestones payment because of it. From there, it qualifies for additional undisclosed sales-based milestone payments and tiered royalties in the low thirties. The second thing is that it allows Bayer to be responsible for the marketing of this drug in the European territories. This gives BridgeBio the ability to focus on U.S. commercialization and other markets.

In order to be able to submit a regulatory filing for Acoramidis, the company had to meet the primary endpoint of the Phase 3 ATTRibute-CM StudyThis late-stage study recruited up to 632 patients with ATTR-CM. Patients were randomized to receive Acoramidis or placebo twice daily. The primary endpoint of this study was all-cause mortality, cumulative rate of CV-related hospitalization, change from baseline in NT-proBNP, and change from baseline in the 6-minute walk test (6MWT). These the primary endpoint was met with statistical significance with a Win Ratio of 1.8 with a p-value of p

The big question is whether this will be enough to counter other competitors in the market. For example, Pfizer (PFE) has its approved drug Vyndamax (Tafamidis)which is approved to treat these patients with ATTR-CM. The point is, it is not clear whether cardiologists will switch patients from the established Vyndamax to Acoramidis. However, such a switch could happen if BridgeBio can continue to emphasize key differences as I mentioned above, namely greater separation of ACM and CVH at month 3. Not only that, but where it could potentially compete with Pfizer is if it can achieve a lower price for Acoramidis.

One disadvantage for BridgeBio is that this treatment needs to be taken orally twice a day, while Vyndamax only needs to be taken once a day. Another competitor would be under the name Alnylam Pharmaceuticals (ALNY) with its drug Amvuttra (vutrisiran), which has already received FDA approval for this drug to treat patients with transthyretin (ATTR) amyloidosis. However, it is also developing this drug to treat patients with ATTR-CM.

By the way, just a few months ago, on June 24, 2024, it announced positive topline results from its Phase 3 HELIIOS-B trial where this drug is being used to treat this patient population. Where this company could have an advantage is that its drug is administered as a subcutaneous injection once every three months. This will be a very competitive area, but if the company can price its drug cheaper compared to the others, plus show an efficacy advantage over these other drugs, then it will still be poised to gain market share. The market for transthyretin amyloidosis treatment is This is expected to reach $11.2 billion in 2032. This market continues to grow and is currently dominated by Pfizer’s Vyndamax. However, I believe that if some competitive advantages can be created with Acoramidis, the company should be able to gain some market share in this space. It believes that it could, by some estimates, be able to gain between 25% and 40% market share for this ATTR-CM market.

Finances

According to the 10-Q SEC FilingBridgeBio Pharma had $587.2 million in cash, cash equivalents, marketable securities and short-term restricted cash as of June 30, 2024. The reason for the cash on hand is because of various financial transactions that it had undertaken to obtain all of these additional funds. Such financial transactions that were undertaken to obtain financing were as follows:

• Net proceeds from term loan under credit facility with Blue Owl of $434 million
• Net proceeds from various equity financings of $314.8 million
• Proceeds from sale of equity investments of $63.2 million
• Special cash dividends received from equity investments of $25.7 million.

The truth is that this biotech is likely to continue to make financial transactions that will likely affect its stock price in some way. Why do I say that? It’s because it states in its 10-Q SEC filing that it believes it is a money trackor at least enough cash in hand for the next 12 months.

Risks to business

There are several risks that investors should be aware of before investing in BridgeBio Pharma. first risk to consider regarding the development of Acoramidis for the treatment of patients with ATTR-CM. The PDUFA date for this drug for the treatment of this patient population is set for November 29, 2024. The risk here is that there is no guarantee that the FDA will approve this drug for marketing in the United States. Additionally, there is no advisory committee scheduled to review this drug at this time. While there is no guarantee that this will happen, it is possible that one could be convened at a later date. Finally, rejection of the drug for FDA approval would mean a loss of an expected $500 million milestone payment. This would be a significant loss in terms of funding.

The second risk to consider with the MAA application of Acoramidis for ATTR-CM to the European Medicines Agency. There is no guarantee that this drug will be approved for marketing in the European territories. If approved, the expected commercial launch of the drug would be in 2025. Even if this drug is marketed in Europe, there is no guarantee that the sales of the drug will be good.

The third risk would be to consider in terms of competing drugs in the ATTR-CM market. As I indicated above, Pfizer is the leading contender with Vyndamax for these patients. In order for BridgeBio to compete with them and other companies for market share, it will need to find competitive advantages. One key area where it could establish such an advantage would be in terms of lower prices. If it can price its drug lower compared to all the others, then it will certainly be in a good position to gain market share. The risk is that there is no guarantee that it can price its drug lower compared to Vyndamax or other competing ATTR-CM treatments.

The fourth and final risk to consider with respect to the development of BBP-631 for the treatment of patients with congenital adrenal hyperplasia, or CAH. A program update for this candidate is planned for sometime in August 2024. Such a program update will determine whether there are plans to continue with this candidate or to remove it from the pipeline. The risk here is that removing this program would result in the loss of an important program. One of which is a significant market opportunity.

Conclusion

I believe it was important to update Bridge Bio’s outlook. This is especially so because not only was Acoramidies’ NDA regulatory submission for ATTR-CM accepted by the FDA, but it also had a PDUFA date of November 29, 2024 set for it.

The global market for transthyretin amyloidosis treatments is a multi-billion dollar market opportunity. If this company can somehow get regulatory approval in the United States and several other territories, it could be in a position to capture a large portion of it. Despite the competition, a conservative estimate is that it could capture between 25% and 40% of ATTR-CM’s market share. Even at the low end of the spectrum of just 25% market share would still be a win in my eyes.

Editor’s Note: This article discusses one or more securities that do not trade on a major U.S. exchange. Be aware of the risks associated with these securities.